Event ID: 616101
Event Started: 11/13/2006 8:18:32 AM ET
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... he had a few ideas in particular, thinking that we ought to transform some of the reports, recommendations into manuscripts. We took this to heart, working on an article on -- talking to some of the people like the general health affairs, others, working hard on that, and we want to have you start to think about places in your domain where you have influence or where you think it would be important for us to get our reports and recommendations out.
I will be very very eager to see that, and our terrific staff team would be happy to help facilitate that. Our committee has a very broad mandate and charter. Guided by a strategic plan that we developed as a committee through a systematic process in March of 04. As you know, every meeting I will pull out this chart, and I am going to do it now. I think it is critical that you keep in front of you what it is we said that we wanted to achieve. We have to keep checking ourselves to see whether or not we are meeting our expectations for our work. Importantly also, I think, time to be thinking about modifying what our a jend a is. I want to be pretty rigorous with reviewing, but quickly, about what we are doing and where we are in our strategic plan.
2004 was a while ago. At some point we will need to revisit the strategic plan. First, public concerns about genetic discrimination have been our highest proirt issue. priority issue. We commissioned a legal analysis of law, significant set of public comments on the issue, almost like a phone book size. Documenting public concerns, we produced a DVD, a summary of our testimonies. We will get an update on the status of congressional activity.
Two, produced a report, nine recommendations on coverage and a good meeting with CMS, they have those evaluations recommendations, evaluating.
The training of health professionals, how that should be enhanced, written several letters to the -- prompted several collaborative efforts among agencies. We heard updates from the working groups formed to address concerns about direct consumer marketing, and in July the efforts culminated in the publication by the Federal Trade Commission on this consumer alert on direct consumer marketing, business in accordation of FDA and CDC. An examples of the agencies working together to solve a problem. You should feel good for highlighting it, and the officials in making it happen. Raising consumer awareness on the in facts about "at home genetic tests", crawgz cautions it's not be evaluated and be ware wary of claims made by those marketing the tests. To consult a healthcare provider and --
We were please pleased and impressed by the collaboration, and the team at FDA, CDC, Linda Bradley, Julie Gesh eding, and her letter, in tab 3, affirms the importance of these efforts.
Earlier we wrote to the secretary to commend has leadership in the advanced infrastructure, vocab Larry, to support the security of genetic data. I will say more about the effort efforts in this area in a moment. A major focus of this meeting, population studies. The t force made extensive revisions based on feedback we received.
We will have an update update from chair Huntington F. Willard and let me be clear, the goal for the session will be to come to closure on the -- come to closure on the report so it can be sent to the secretary in final form.
One thing we will do this this meeting, again, introducing every topic area, you will be very clear about what it is you are supposed to do today, so when you ask the great smart questions, or put the great input you do, it's designed to get us to a goal. The goal on this one is close the report and send it to the secretary in final form.
Gene patents and licensing, moving forward with a study on patient access to genetic technologies. The committee established a task force to define the scope and study plan and they have a detailed scope, work plan and timetable for us to review today. Work will also continue on the priority issue of pharmacogenomics, the task force refined the draft report, recommendations, we will begin a very focussed discussion about it in just a few minutes.
We will be look being closely at looking closely at development of genetic tests, and have a discussion whether SACGHS should take on a role in this area. New to the committee, up to speed about the history, evolved out of an earlier committee, the issue of oversight on genetic tests. We have been able to turn our atongs attention aattention to other issues, and whether we need to be -- an extremely important discussion for us in a few minutes.
The cross-cutting issues of genetic exceptionalism, awareness, integrated into all of our other work. That's where we are with the priority issues. I ask you to keep that in mind, we want to revisit it.
In your briefing books, left hand pocket, is an annual survey we are suppose to do fill out, about your perspectives, our effectiveness, and whether we are meeting our goal and priorities. If you have not done so, you are supposed to do that before we leave. So the thing is, if you don't think we are making the progress we are supposed to make, then, you know, you have to say that. But we have to then figure out what we are not doing to meet the objectives we have. But one thing, at least we are focussed on the work we are to be doing. The foundation -- to assist us, and new work secretary Leaf it would like us to undertake in the future.
I would like to say something about secretary leaf it. I am extremely excited about his leadership. In a way we have not experienced before, attentive in an unusual way to the committee. Talking to me, as your chairman about this. He's made his key staff available to us. We have an opportunity that we've never had before, which is to really get input, guidance from the secretary about his agenda, and how he wants to see us move that agenda forward. I really want us to spend a minute as we pay attention to that agenda, what he's really focussed on is improving quality and cost of healthcare and making that quality and cost transparent to consumers. The president advanced in an executive order, requiring agencies add administering healthcare -- part Drk of they Medicare program, working hard, in a number of meetings with him on planning, preparing for a potential po influenza pandemic.
Taking a systems approach over the next teen 10 years he wants to see healthcare reach new 41 frontiers. Enabling healthcare to become more preventive, predictive and certainly more personalized. The Secretary recognizes jen omics is playing a larger role, and how to incorporate this new information. I met last month with both Sheila and Greg to discuss our work, how we might be helpful in advancing the Secretary's agenda. What we are doing, integrating genomics, particularly in the area of pharmacogenomics is already on the secretary's radar. The effort, called American Health AHEC, aimed at getting the best thinkers across healthcare and those who pay for hilght care healthcare to put together -- health information technology, and data, allowing people to make better choices, decisions, having physicians and healthcare professionals have access to information so they can make better choice and decisions and do it in a way that protects privacy, security within the consims of interoperatable --
Last meeting, a HECK formed a personalized group, very important for us to stay tuned to some of that work. The Secretary asked me to make you aware of a request published in the federal register November 1. A copy of that document was sent to you last week, another is in your table folders. Seeking input from the public and private sectors on plans for developing and using health information technology, genetic and molecular medicine for health outcome evaluation and research. Seeking information on a wide range of topics and we will hear about that in a minute. I encourage you to respond and share with interested colleagues. Due back by the first of next year. With that, again, because this moment of trying to bring together this health information technology, ability to have information about personalized genetics, combined with enhanced consumer decision making, combined with health providers and hospitals having the information, coming together in a new and interesting way, I want to turn to Sheila, see if she can bring us up to date on what is going on, how from the point of view from the secretary's office the committee might be helpful and thank you for joining us.
Sheila: Thank you for the opportunity to join you and the members here today in your important work. The work of this committee is highly relevant to the secretary, one of his top 10 initiatives, accelerating personalized healthcare. Improving the safety, quality and effectiveness of healthcare by leveraging advances in jen omics. A powerful ful force, enabling better clinical outcomes. Focus is on how to improve health in a more patient-centric way. To fully realize the potential we need input from many important stakeholders. At your prior meetings you discussed many of the areas important to science and public policy, we eyingerly await the work of this committee. I brief the Secretary extensively on the work you are doing and he is eager to see the work you produce over the next several in sync months.
What we are working on in the Secretary's office and NHS. He's been highly focussed on using information technology to advance healthcare. At the October meeting, a formal working group established to acknowledge personalized healthcare. Recommendations you can expect from the group include standards on how to incorporate genetic information, genomic test information to personal electronic health record. Other issues this working group may address include inthe greating databases and including genetic test information as part of analysis for clinical decision support. I would like to say we recognize the many hours this committee devoted to these important issues. The list you had up really highlights the focus areas we have been discussing in the Secretary's office over the last several months. We have a count down clock. The Secretary feels a great sense of urgency to accelerate this area. I have it on my desk, we have less than 800 days, to focus on what we can lish in the near term, but to make sure we are on the track, to continue the work beyond his tenure in this important public health area.
On behalf of the Secretary and Dr. we thank you for having us here today. Hope you allow us to come to your next meeting and allow us to give you a briefing on where we are. Look forward to seeing you then
I want to acknowledge Greg as well, the secretary asked Sheila to come back downtown, Greg you have been attending, however long you are going to be here we want to use you, you will follow-up. I know we have a busy agenda and I want to move us forward, but any questions you may have I Sheila or Greg since we have them here, I want to keep centered for you, that 800 days, it's not a countdown to figure out how quick they can go home. The countdown is, they're going to make some changes. From where I sit, you may see the impact from where you sit. This activity they are engaged in, both the AHEC, can President's order, health data, information, transparency of information being fed into quality and performance being fed into physicians, hospitals and consumers, this stuff is transforming the way in which healthcare is delivered. It's a sea change, like with IBM commercial, this changes everything. This is one of those this changes everything. I want you to think a lot, especially with this pharmacogenomics stuff, all of that is connected in here. You really want to be making sure we are in that 800-day agenda.
Sheila: And I want to recognize Dr. Greg Downing, he's the director and done a lot of work in a short period of time; is an incredible force supporting the Secretary in this initiative. It's important for us to keep in mind all of the activities going on in this area, close coordination, transparency of activity, meaningful participation of stakeholder groups is necessary to achieve the objectives, and I thank thank you for supporting us.
We may have naild this, looking to be sure there's no question. I know you have to go Sheila, but Greg, as long as you can stay you are welcome.
A couple of things before we move to the pharmacogenomics session, public comments excuse meed for both days. Individuals who have not signed up should do so. Housekeeping matters relate to do important topics, lunch, dinner. To save time at lunch, I encourage committee members to order a box lunch. Fill out the form in front of you or Abbey Smith will be upset with you. We are having a group dinner tonight, let abbey know if you can attend at the first break.
The commercial from our sponsor, the technical rules about the ethics.
I want to highlight a couple, do this at every meeting, I know you probably know what I am going to say by heart. It's important, bears repeating. As you know, you are appointed to the committee as special government employee, to serve the Secretary and the public. This is a special category, but you are nonetheless subgeek the to subject to the same rules that apply to government employees, ort lined in a large document you received when you were appointed. I will highlight two of those rules. Conflicts of interest: Before every meeting you provide information about personal professional and financial interests. To determine whether you have real or apparent conflicts of interest that could compromise your objective of giving advice in the meetings. We waive for general matters, believe yourability won't be faiveght effected by your interests, we require you to be attentive to issues that could arise, appear to effect your interests. We provided each of you with a list of financial interests, covered relationships that would pose a conflict if it became a focal point of committee matters and if so ask you to recuse yowrdz and yourself and leave the room.
If you lobby in your professional capacity or as private citizen it's important you to keep the activity separate from the activities associated with this committee. Keep in mind we are advisory to the Secretary of Health and Human Service and don't advise the Congress. I thank you for being attentive to these rules and we appreciate your consciousness very much.
Now to the meeting.
As you know, we are in the process of developing a report to the secretary on pharmacogenomics, the opportunities and challenges associated with this integration in healthcare and public health. At the last meeting in June we discussed preliminary straw man recommendations. Following that meeting the loen group prepared a draft report, and staff revised, and -- our colleague kef fitz, the chair Kevin T. FitzGerald will present the work, discussion of the issues identified and the revised recommendations. We will spend about four hours on this important topic. By the end of the session we need to have reached consensus on whether the draft report is ready to be released for public comment or whether further work by the task forces needed. A copy of the draft report is in your tab 4 of briefing book. With that, again, listen to these things, go through systematically, and make a choice about whether or not the report is ready to be released or has to go back for t force, for public comment or back to the t force. With that, Kevin.
Dr. FitzGerald: Really, I am just a spokesperson for Suzanne. I will have the privilege of giving you an update on where we are with the report on pharmacogenomics, and I would like to ask your patience while I begin by giving you a little more background. Reid has give uh some, before we dive swo the report, first, much appreciation for the t force committee. People have worked very hard, given marveleous insight, and as usual there are names missing from who to thank, orderless of how much, Sarah, Suzanne, e vit a refuse. Don't forget them, the incredible work they have done in getting to where we are today
Why are we here? We just heard the great impetus to pursue personalized medicine, and delivering the right drug, right dosage to the right patient at the right time. There a variety of drivers behind this impetus. We have broken these down into research and development, public policy, right out of the Secretary's personalized initiatives, one of the major for the Secretary. The reason is that pharmacogenomics has significant promise. You all have the slides in front of you, the handout from today. Currently on number 5.
You don't have to twist your necks around and pretend to be owls or whatever. Just read off the paper. For our Internet audience out out there who don't have the handouts, in addition to pharmacogenomics, improved safety, efficient use of drugs, there obviously many challenges in order to get pharmacogenomics integrated into the clinical and public healthcare practice. Our role? As indicated, identifying the opportunity and challenges that are ahead of us, advising the Secretary on how the federal government can help to advance the opportunity necessary this field and to address the challenges. In other words, to develop this report, these recommendations specifically for the Secretary.
A little history, as you heart, we had the informational sessions -- you haven't heard this yet. A year and a half ago in June, the approval of report outline a year ago in October. Activities you can find in appendix A. They are extensive, important to be aware of what's going on. This will be part of the challenge of integration. The development of the draft recommendations put forth in June, we took your feedback, responses on those and tried to rework the report integrating those responses, trying to move ahead to develop a report, recommendations for the secretary. Following the June meeting the staff revised the draft recommendations based on discussion and in spite of the guidance of the new task force chair we were able to move ahead and the Loen group, if you need to identify the group, look for the people in the room who look the most harried and dependent on caffeine. That would be that group there in the corner.
We dragged the horse to the middle of the stream to put them on a speedboat, send them down the river. They took our recommendations from that time, put them in the draft report you see before you today.
What had are we here to do today? As we heard, we want to ensure all the major opportunities and challenges have been identified. We want to ensure the draft recommendations address these high priority issues, and we want to ensure that the draft recommendations are the appropriate solutions for addressing the issues. As Reid mentioned, we want to reach consensus on whether the draft is ready for public review. The most important goal, not on the slide, to keep Reid happy. Why we will work efficiently through these hours to a kef achieve consensus on where we are. We want to get to this point so the next plannedded steps might be pursued. Those steps will be to revise the report based on today's input. The Loen group will go out, look for input from 15 federal and non- federal stakeholder ers on various pharmacogenomics issues, scheduled to be done this winter, will seek public comment late winter, into the spring. Love to finalize the report next summer and release the report in fall 2007 so we don't take too many days off the Secretary's 800. Or as few as possible.
The way the report has been structured for today, we took three overarching themes, research and development, who are the gatekeepers facilitating, inhibiting the development of pharmacogenomics, appropriately or inappropriately, to improve outcomes in clinical practice. As far as the research and development piece, clinical research, also the infrastructure enabling research and the -- issues in research and development. In this section we had five subparts, we will go through piece by piece to identify if we have covered the terrain well and have articulated what the recommendations should be. We will then move on to the next section, the geat ceerps, third-party payers and clinical practice guideline developers.
In this, one set of recommendations, 6, that has three subparts. It may appear to be a smaller section, but was identified by the t force as critical to pharmacogenomics moving forward and requiring our direct addressing of these groups. Finally we will look at the implementation of pharmacogenomics, and that will involve education and guidance, information technology, economic implications, the -- issues, and coordination of all the HHS, pharmacogenomics activities, and as you can see, a variety of recommendations here with 14 subparts.
We would like to walk through the three overarching sections one at a time. Go through the issues identified in each section to make sure we hit the major ones, obviously not going to be able to do everything, but we want to hit the big ones, consider the recommendations drafted; consider if they will be adequate to the task of over identifying issues, and finally, since no institution can do everything all at once, the thought is to identify the recommendations of highest priority, not written in stone, but thought it would be useful to give back to the secretary recommendations we consider to be particularly ly high priority.
High priority versus low, a simple high, low identification, the t force identified 12 of the 31, and we have identified those by the little bouncing star on the right.
As we know, stars may appear to be permanent, but they are not. They erk vofl too, we can remove them, place new stars somewhere, it's our chance to play God. We can do that this report. We want your feedback to give to the public and get their feedback. One thing we learn, repetition, repetition. Again, why are we doing this? Issues are does the report cover the major issues, issues that have not been but should be raised? What are of the highest priority. Recommendations, do they as worded sufficiently address a high-priority issue. Any that have not been but should be included? Any that should be deleted because they are a low priority? Not enough of impact on problem or not implementable at this time.
When, the prioritization, to what extent will addressing this issue via this recommendation advanced the goals of pharmacogenomics, is the government in a position to advance this recommendation? After hearing this several times everything is clear. We can start to get into the first section, recere much and development. This breaks down into the four subgroups I mentioned before.
We want to look at the issues at this time. We will get to the recommendations after we have gone through the issues.
Here are the issues: Basic and translational research.
Safety and effectness of drug treatment. More translational research needed to apply the pharmacogenomics technology. Translational research studies, if designed carefully can themselves be a source of data for downstream studies of the clinical validity and utility of pharmacogenomics tests. We identified. When one looks at co-development of pharmacogenomics, drugs and diagnose ticks other -- concern for market segmentation, uncertaint about -- and requires new collaborations between drug and diagnosic industries and development of processes. This can result in expedited FDA approval, fewer label changes, greater likelihood for provider uptake.
What about the oop application of pharmacogenomics drugs, fail in a broad enough population group. A post hock analysis, information available can 2345EUB8 the rescue of abandoned drugs for use by smaller populations. In this area, again, it's important to look at what the incentives are for pursuing Ind cage occasions for -- we have the breakdown, not in the report as structured here, but what we put together. If you look at patent status, for instance, you can see an industry might have more incentive if the drug is still, device is still underpatent. Less incentive if it isn't. If the adverse drug reactions are more severe. No availability of alternate treatment, if there is, less incentive to pursue this application.
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Debt to the research so that across the board providers would have access to at information about which drugs would work best. That, I'd think is gaining some interest in momentum, and I'm just wondering if it should be addressed even if it is in just a small way in this report because it sounds on its face incompatible with the notion of a personalized Madison, but I think there can be placed to reconcile it as well. And I'm wondering if some passing reference to it would be worthwhile. You might want to expand on this because I haven't been participating in that group, but I know that there are several sectors of the health-care industry calling for this and I think each 8S is aware of it.
I don't know if I know a lot more than you. The key issue is, as you have underscored, people really do want to have information about whether this new thing, whatever the new thing is, does it work better than the old thing? And if it does, is it more cost effective when you look at the total management of the condition from diagnostics through the therapeutic implications to the testing cost implications to the safety and convenience. So I think it is right down the middle of the plate because what this optimally is saying for these new personalized pharmacogenomics products is, do these things -- how do they fit into the overall health care landscape in terms of bring out all stock and replacing it with new stock or this synergistic or additive work, initiative or whatever. But if you don't have that information and the health care that is costly like this with 48 million uninsured people, it will be very difficult for the new thing to ever break through.
I also would like to comment on the -- I guess the initiation of some type of comparative analysis in terms of diagnostic tests. I know that we have commissioned Dell looked at various tests for genetic cancer disorders. And one of the things we have asked to take a look at is the effectiveness in terms of not only effectiveness but also the accuracy of the tests looking at measure of accuracy in such things as specificity, receiver, characteristics as well as selected ratios. So in that instance, yes, there are comparative tests which might be applicable in terms of determining whether or not one particular test is more appropriate than another.
Thanks. One of the practical issues with implementing pharmacogenomics in the big U.S. standard of care of medicine is the need for rapid turnaround and results. And clearly many circumstances where one would like to adjust the plan about what the right to prescribe or what goes to prescribe are not well served if it takes a week or more for that test to be returned. So the press Gropper can make that decision. And in fact I think this could be quite a major issue. Until such time as everybody has their entire genome already pre sequenced and sitting in their medical record where it simply becomes a matter of a computer search to get the genotype, we are going to be very much at the mercy of what kind of technologies provide the kind of a point of care, rapid turnaround result. I did not specifically see that flag as a research and development priority, but clearly that could well turn out to be limiting. If we have a wonderful day the killing for instance that in the presence of a particularly burleycue to medical elements a particular pharmacogenomics test would be valuable in terms of eliminating what drug and dose to give, but you can't get that result quickly enough to actually influence that decision. Then people will continue doing what they have been doing. So this notion of coming up with a means of accelerating turnaround time for these kinds of the dean of typing experience is when it comes to pharmacogenomics, it seems to me it is probably not gotten as much attention as it should because until now most genetic tests are done in central laboratories where samples are shut can if it takes awhile for the results to come back that has not been critical, but here it could be. We want to flag that as another potential research need.
Thank you.
I did want to bring up another issue to the community. I sit on one IRB panel N within our institutions. There are four panels that look at this review. One of the things that caught my it isn't going to our current plan draft report is that it is very important as we move forward for translation and research in the clinical research that went testing is point to be dubbed to go back to the patient or to put patients in different categories because you have a certain the genotype, or you are at the point on a specific research from the laboratory that these tests need to be performed in a certified laboratory. So there is good regulation because even research laboratories are not clear if there will report back to result. I'm not sure if all of them throughout the country are really aware of these issues. So either through communication of the office or some other venue we must make all IRB aware of this regulation.
To follow after Doctor Collins point about the practical of the cases of how to get the tests done at the right time, there is the prospect of doing pre symptomatic testing said that you have those results in hand at the time you need them for an acute LS or for acute needs. The military has a little experience in the area of during testing before hand in case you need anti malaria's that may or may not cause problems depending upon what your tests results are. And the same sort of approach to an illness might be the verse to a certain pre-Socratic testing for pharmacogenomics applications.
Would you see that the falling under the same issue?
In terms of practicality, but it does raise a lot of ethical questions when you go down that road, but it will come up. If you are in practice and you want to prescribe a particular medication but no you will run into problems based upon the genetic profile of your patient, then you can know ahead of time that you need to sweat out a particular drug with better benefits.
Great. What I would like to do is just get a sense of the Committee on where we are all these things because nobody can do everything. We can't put everything in the report and all that. So from what I heard so far we have three issues that we can certainly put in. Now let's start with Cynthia this she had the first. Could you restate its and state it to as you see it specifically relevant to the research and development section? Because this is the section it will be going into rather than the application section which people get to later, but if you want to bring it up later it might be applicable.
I was short because I had a couple of issues written down and I was trying to categorize them. I thought this could be other research or coverage. I don't know how to characterize it. It certainly doesn't matter to me where we put it. I know it is out there and is part here get? Maybe when we have someone from ARC perhaps others could inform us a little better.
Okay. I my just guessing that when Michael -- so Francis definitely looking in R&D, how would you specifically phrase that?
That need for a research and rapid turnaround cost effective point of care to pharmacogenomics for -- genotyping for pharmacogenomics.
Great. And Andrea?
Test a sentence or two and I'm not sure how you deal with this issue been aware the secretary can work with OHRP to remind IRBs us throughout the country that we need to have laboratories performing testing when results go back to the pet -- patient either troops putting patients in different [ indiscernible ] or different doses. When testing is back to the patient it must be done in a certified laboratory.
I think what we may have to do is when we get the report more fleshed out people have a good idea where that could slide in.
In terms out adding upon and coming up with specific wording and whether or not to put it in this section or the other section, it might be something like in addition, to looking at the clinical utility and the critical levity of the test also evaluating -- it measures also evaluating accuracy should be taken into consideration.
Okay. All right. Thank you. And is that good what what Francis said for incorporating what you -- okay.
I think so.
One of the areas of research I have noted as I'm trying to implement pharmacogenomics in a health care system setting is cost effectiveness research. What is the cost effectiveness of spending the money to do a test on 100 percent of patients were you know 10 percent of them will have a variant versus, what is that sending you an adverse outcomes. This type of research is really needed to support the clinical implementation of pharmacogenomics testing.
Currently we have that in the third section of the report. We could -- we could put --
So there are research parks in the third?
I'm not sure our it goes. But what do you mean by research? There is certainly the edification of a need for that kind of information. As one tries to apply what the basic science and transitional the debt is going to be to the clinic -- if you think it is important to have a statement about that in the R&D section --
There are public health faculty who wanted to this research but don't have the funding opportunities to support their effort.
Okay. Michael?
In reading the report I think there seems to be some assumptions I want to make sure I understand. The assumptions are, as Mr. Owens was saying, that the tests are accurate. And I want to make everybody to understand that there are only a handful of clinical diagnostic tests that currently have a standard reference materials available. That is out of the 1500 or 2,000 tests that are done all the time. The other part of this is that in order to make that dream a reality of pharmacogenomics and Tech get that some of the basic pieces that you need here as far as gene expression for everyone, Genetics for everyone, to really make this happen, the technology simply is really not there right now. And in order to add to -- in order type, as Doctor Collins said, have a gene sequence for everyone it would still cost about 1 million -- he's gone. Roughly $1 billion per person, where that has the get down to $1,000 or less. The accuracy of that gene expression measurements, that clinical Micro race and things like that, this has a major program in trying to find how to make those tests work better, just a signal transduction problem, a big issue. So there is a great deal of hope and this, but the technology just doesn't exist to really make that happen.
Would you see -- Emily, okay.
I just want to address that comment. I think that technology Ted to rapid genetic testing are coming along and it is actually an RFP out from CDC to develop testing for a ban and funds that based on genetic analysis. And what you will see coming out of that is funding for a number of different technology platforms which could then be leveraged across because you are still during genetic testing whether you are doing infectious diseases and that -- and Texas disease genetics. So one of the things we should do is make sure we are closing the loop between those kinds of activities within CTC and an area which might be perceived as quite different from this. And understand that that same technology but form that CDC is funding is helping to move forward in terms of getting too rapid point of care and molecular testing it can also be applied then and pharmacogenomics. So it is double bang for your buck.
There are a lot of the HMS that have tons of money going into genetic testing and buyout threat agents that could be leveraged against what you are doing, as well. The other part is using this data set to study and learn from it to and implement new discovery. Dr. Got men will tell you, I think, if you look at the FDA would set there have been no new protein bio markers approved by the FDA over the last 10 years. So that system for discovery is a bit broken right now, and I think you have an opportunity right here to change that.
Both the two of you, and the report as it stands now there are a couple of different places where we emphasize the need for -- all call Ed education or public access information, and this could certainly be in their ads -- we certainly want to discuss the hopes and the goals of pharmacogenomics, but also make it realistic. And we want to let people know where it is we stand now. That is already in there. We can be more specific in that regard, but I also get a there is the issue of the cooperation and the interaction of these different groups that are already involved in it. We also try to involve that in the report at some places. We can be more specific in the report as to the issues you raised as that needed to that, but then there was a third thing I was hearing but was actually it sounded to me to be more specific. You were talking about it in particular. They're is a step that needs to be taken that is very concrete. Could you just outlined that again?
I think it was really reiteration of Francis's point that it is wonderful to discover bio markers and validate bile markers, but if you cannot deliver the result back to the patient in a timely way for a position to take action then you have missed the implementation part of it.
Okay. Good. Thank you.
And if it is all right and we do want to keep moving, let's get on to the recommendations because that is where people have the rubber hit the road here. We have now again -- we are looking at the wording of the recommendations, do they sufficiently address what they are intended to address? Are we missing any? And are there some that are there that don't need tepe there? Because it is not a high priority or they won't have a high impact or they are taught not implement the ball at this time. So how will these recommendations advance the goals of pharmacogenomics and is the government in a position to act upon this recommendation? Here is our first one. If you wish, in your Executive summaries starting on page five you have the entire recommendation is spelled out. On some of the slides we have truncated it a little bit obviously due to space limitations. So if you want to follow along, page five of York executive summary you have the recommendations beginning, we will start with the basic and translation of research once and this is 18 that the NIH should invest more resources into the research of bio chemical pathways is a city with drug action Cortines and Balkan these pathways in gene functions related to the safety and development of drug treatments. And I think that is exactly how we have been thwarted in the executive summary. So these two are the same. Any comments on this particular recommendation? Debra?
Well, it is really dead jeans and teen variations. We very often note that scene but may not have the gene variation. So somehow bad gene variation from person to person is key to the recommendation.
Soap you would want to put in bad genes involved in these pathways and Jean variability and function? Or would you just --
You could say it gene variation and functions related to the effectiveness.
Great. Thank you. Everybody else is comfortable? Great. Next. And I it should support more transitional research focused on the development of clinically useful pharmacogenomics technologies. These are sort of boilerplate in one sense, but these are things that certainly came out of the report. There are more, I'm sorry. There is more there in the Executive summary if you want to look at that. Okay. Yes?
Is it appropriate to comment on the philosophies that do these sorts of things? Up to this point a reduction of philosophy has been used of one protein and one gene at a time. There is some broader work being done, but up to this point I think some of these, like I said, they have been bio markers. So are we doing -- I think it may be important for us to an element of the philosophies use up to this point. The nubile markers in 10 years is pretty significant to me. Are we taking the right approach or should we be taking a more systems approach working at the systems medicine or systems biology approach to some of these banks because right now things are moving quite as quickly as we hoped.
Now are you -- we can certainly raise that issue, and I think it is a good thing to bring up in the research and development section two said this is how we have cut into where we are. And in that process this has raised the question that you raised, do we need to be more open than we are currently to a more systems approach? We can do that. Does that translate into a specific recommendation or is it okay to put that into five tepe clear and put that in the issues?
Eye open it to the committee to discuss.
Okay. Yes, Deborah?
I'm concerned about the statement that there haven't been any nubile markers and 10 years because I'm aware of nubile markers being introduced into the clinical testing the every day dearly. So I'm not sure where that statement is coming from.
It is really if you look at the web site and look at that new p.m. A and diagnostic, I'm talking about protein bile markers. There have been some new genetic line markers approved.
What about the [ indiscernible ]? Francis, maybe you can comment as well. I don't want to be hanging in out here on my own.
I'm a little confused by this discussion as well because we are not talking about the whole universe of piled markers. We are talking about to the extent we have identified promising but not fully clinically valid visit of examples of Sagittariuses that are associated with drug response. Take how all of the opportunities and the key 0RC once and all the things we know about tepee empty. I would not say that all we are in a circumstance where there hasn't been progress. What is missing is that next step of full-fledged clinical validity established in perspective trials. But we don't need a systems biology approach to identify the potential candidates to put into those trials. The main step here is the trials themselves.
To follow up on that, but the recommendation 18 and 1C should come before one be.
Okay. You want 1C Tech come before one be.
Or didn't he should put 1A and 1C together. He should collect this information and draw the conclusion, take that next that.
Just a quick question, do you think -- and I think we broke these out, not that they are not related, but to try to emphasize each particular piece. Are you saying it would be more effective to put see together with a?
No, no, no pre.
Test change the order.
Okay. And I have a comment I want to make which will make it more clear.
Again, the order as Suzanne reminded me, is based on how they are discussed in the report. We can certainly look at changing even in the report to just create a better flow. Okay.
Kevin, perhaps one be could be expanded to include Francis's point and Emily's on the pharmacogenomics technologies that are being developed have to be able to provide answers in a clinically timely manner. That is what the purpose it's turnaround time. So that as part of the technology and development.
We could say there development of clinically useful on a timely development of clinically useful pick five. Is that enough or do we need to be more.
We need something more explicit about the need to have technologies that give you more turnaround for pharmacogenomics. But that could fit into that particular recommendation just fine. It just must be fleshed out a little bit.
Okay. Thank you. All right. Now we have already talked a little bit about one see, but let's --
I was going to make a specific recommendation. They're is a tool that we have, a mechanism "for clinical trials where we list of ongoing trials. That is -- there have been numerous editorials in The New England Journal for Madison and everyone who does the early funded work which there. Those who do industry support across the want to be published in the bill in the Journal want to be listed there and I think he could encourage its further use to enable collaboration work pharmacogenomics components could be added on or even designed into clinical trials at the outset. So for example the registry could list other materials have been collected and whether DNA has been collected and whether it has been consented for pharmacogenomics studies. I do that as part and parcel of discovering the clinically valid knowledge that later on you want to implement into tests that are available rapidly at the point of care, but you must know what you are doing first and you have to utilize the tiles and studies that are already ongoing by adding a pharmacogenomics component. A mechanism exists to do that if it were upgraded a little bit. And those who run the registry are interested in doing it. They just need the recommendation for government encouragement to do it. It is a matter of collecting the right field and encouraging and enabling research.
That would be great. The more specific we can get is wonderful. So we can certainly -- I have the sense that that could work.
Is that registry you design in your own institution the research and then posted in that registry?
The clinical trials is want of an actual Library of medicine. It is a project and it's self and anybody can post information to it. They make available the deal to do it and then a trowel designer must voluntarily submit that information.
For publication purposes? Her is that ties to publication?
For the journal editors the get together and said we want to promote this kind of sharing of information. If you want to get into our journal, he better be using that registry.
But what we are trying to say here is before you engage in these clinical trials meet with certain people that do research or actually clinical trial saugh develop these in a systematic way that get this the right information so that it can be used for the down the road for applications with the FDA.
I was thinking about the trials. In the context I was presenting things I thought the trials would be important to the discovery, discovering the information. But at their genotypes and medications they are taking and discovered the link that he later what to evaluate through the right kind of outcome or evidence base studies that should be implemented and clinical practice. But I consider the fact that basic discovery of those connections, that knowledge and the first place and that is what I'm seeing up the basic and transitional research recommendations here.
I thought that we also we're looking into increasing some of the values of these early on clinical trials.
We are in total agreement there. Increasing the light of research is already getting done.
Good. Indeed.
I thought it must be useful to that end under the list of research clinical trial outcome of research also cost effectiveness to highlight that as such a key point in this area. The other thing I wonder about this been holed up there is a separate section on Elsie to bring that into this section where you are talking about justice of research has not just a separate type of research but something imbedded in a transitional research preadapted they add and cost-effective studies.
I see. Okay. Cost effectiveness in LC. Now with the Web site, obviously that information doesn't get put in there too. Could that? Is that 85.
That information meeting --
Cost effectiveness LC issues.
I would say no. Think of it as a registry. It simply presents to the world that a group of researchers or a company are planning to do a trial or are doing a trial and this is what we are studying and this is to you contact and these are the enrollment criteria and this is what is being collected. It enables researchers to the ethnic connections and doesn't dictate what kind of research is done. It is not a funding mechanism.
We can put that in but we can make sure that some of it breaks out in one direction and others is there for people to --
What we are trying to say here is that we want to encourage research to coordinate with the outcome research while they are doing study design. What you are talking about is listening what other people are doing so they can communicate with each other and hopefully not repeat some of these studies.
That works. Yes?
Perhaps this is littered but this position is getting into recommendation three which loosest validity. So the cost effectiveness else the issues are certainly part of that also. Are we testing and combine all recommendations or will be keep them in certain places?
We are trying to set the as discreet as possible. On the other hand, we up to make -- we must make sure we do the proper amount of emphasis on various issues. That's why we are going to this now. If we need to jump back and forth a little bit, we can do that in this section. We can also do this in some of these other sections as they regard applications, but that is okay. People to that. [ indiscernible ] they should encourage the best bet is to consult FDA when their research reaches a critical state and they could have the conduct of the flaws least doubt and critical studies making higher party scores to cities that are designed to satisfy F.D.A. quality of evidence standards. Again, this is a recommendation in an attempt to tie things together which we thought was an important thing to do. Every is good with that? All right.
That is just a little bit awkward and that in IA to doesn't actually give priority scores. They symbol review panels that do appear review. I would say some --
Right. Review panels.
Funding decisions should account or give weight to satisfying if the a quality of evidence standards.
I think we just dropped the word scores.
Prior to the scores.
A [OVERLAPPING SPEAKERS]
Hobbs.
Do you think here that maybe education to research is during transitional research and how to conduct some of these statistical studies?
We have some other educational ones.
So workshops or some other venue?
Let me try to figure out the most efficient way to do that and get it . Ises captured and accuracy of testing is to be evaluated. There are only a handful with a standard reference materials.
Well, we talk later. When we get to a recommendation three we talk about the validity and utility and we can certainly add in accuracy at that point. That might fit the concerns that -- yes. Okay. Is that okay?
It is a question which is more a point of clarification, I think, to ask to the NIH. I guess I have always been under the assumption of looking at the weight to these are reviewed priorities are already given to those that are methodologically sound already. I thought that was already in existence and I'm wondering whether that is true. Maybe the way we should be is something along the lines of if there will be integration, that it should be continued to be enforced or I guess remind people. If it is already there it seems a bit unnecessary to say they should do something they are already doing.
I think you are correct that they already give the higher scores to be statistically rigorous and well-designed studies. You are right. I believe and I did not craft this of its own recommendation. There was some intent here that the FDA sought specific and unique means and sometimes they felt that we should lend to funding to types of studies they need to see done. Am I accurate? I think that is what made the effort be a single that made this a different, but you are right, review groups already give the best scores for merit to the most well designed studies.
Right. I think the implication -- we can we were this to make this more clear. What is in the first paragraph is part of what should be the what is considered to be much allows police sound and statistically rigorous. That is not necessarily but to be as strong as the moment.
This is Liz man feels from the FDA. I would just encourage you not to put the cart before the horse here and that studies are typically reviewed and funded prior to having been done. The way this is written it appears they would only seek FDA advised after they had reached a certain point. So I am not sure how review boards could say something was meeting FDA specific studies and hadn't even started yet.
Right. In the second paragraph we have NIH should encourage him this is to consult FTA when their research reaches a pivotal state. Is that what you're --
But then the next section said you would encourage funding to cities that are designed to satisfy it F.D.A. quality of evidence standards. I think those may be somewhat in conflict with each other. As far as I understand you tend to get funding before you reach a pivotal stages.
I agree that you are on the right track. I just think that it will be hard to say, this will meet FDA quality standards before the funding has been given.
Family?
I think what we've or trying to get to is the next level of study beyond the tantalizing early results study. So now you will design a study that really is a validation study and having been into FDA once or twice and been told that one of the things FDA would like to see is some good published studies done independent from the manufacturer indicating that that marker has clinical utility and validity. What we port trying to get to was encourage people to link those studies separate from whatever company might sponsor a device said to those in a rigorous way so that FDA could consider that as a reasonable piece of literature.
That is entirely reasonable. Maybe just a little clarification.
When you read this you are hearing that the third paragraph sort of stood out on its on, and that is what we are getting from both Joe and you. So that third paragraph is leading people away from the first to somehow. Okay. In 28 -- 2A now this is development of pharmacogenomics products and helping with services should provide FDA but let necessary resources to provide guidance documents about best practices for the code development of pharmacogenomics drugs and diagnostics. Diskettes should promote diagnostic -- operation between the drug in diagnostic industries and clarify the review process or code developed process where the drug is subject to FDA review but the laboratory developed companion diagnostic test may not be. This is actually in your incentive summary it is 2D. It was moved up. If the list is actually somehow some kind of proposition, which it isn't. In any case it is now upon 2A.
Can I ask a question here? I thought FDA had advanced on the code development. Has this not being done?
It is actually a white paper right now headed towards balance status.
So this is under way? Did you need to have a recommendation on this?
I got beat -- got the sense of the task force was that this could only help move this process forward and that there was a desire to make sure this was emphasized.
Yes, there is a desire to get the draft guidance out, but it had been previously released as a white paper and not a draft guidance.
Update. Okay. For 2B, at the age shouldn't fight research opportunities related to the development of pharmacogenomics products and should facilitate the research through its critical path initiative. And then 2C HHS should advance the further development of abandoned drugs by facilitating access to information about such drugs. Incentives will be needed to encourage the voluntary submission of proprietary data by pharmaceutical companies. Again trying to address some of that gap problem that we identified in the issues earlier. And then what is now 2D the -- back 2C on terra.
It is so clear where that data will be housed. It says to encourage manufacturers to submit proprietary data to home or where would that be to be housed?
It doesn't say that there either.
It is housed at FE8 right now, right?
Do you have a specific place you want it to be housed or case FDA okay?
The intent is to try and have the data available to others kept take it further. I'm not sure if that will be feasible. So I'm not quite sure what the intent was after the manufacturers release it. If we are still saying FDA is not as accessible to others what it meant the purpose is?
Can you ask for a development of incentives to encourage the eventual release? Because right now there are no plans to release any of that, right?
The voluntary to Numic debt? Had no, I suppose any company that submitted could release it if they wanted to.
How can we get a mike?
Yes, FDA has a voluntary to Numic --
Identify yourself.
[OVERLAPPING SPEAKERS] they have a voluntary submission process but the information that comes into it is confidential. It helps FDA but not the rest of the industry or academia. So I think we are talking about a process for making it public.
Right.
So how would we go about that? That is what the needs to be determined.
And as far as incentives, I think you would want to make some concrete suggestions of what those incentives might be.
Now you want those in this report are we can't just put that forward to the secretary and allow the secretary to make those determinations?
That's up to you.
Can I ask a question here. Are we talking about two different -- it is an abandoned truck will the drug company have done a submission to that FDA? If they have gone through trials -- are we talking about something that would even be submitted to the FDA here? I don't think so.
Drugs can be abandoned at many stages and may have been submitted 2013.
But they may not have.
But they may have been abandoned prior to that in developmental stages.
So we may not be capturing all the abandoned trucks if we are talking about the debt that is submitted to that FDA. So I think we need to incentivize the drug companies to further develop or move for abandon drugs using pharmacogenomics Technologies, and I don't know what those incentives would be. You would have to ask drug companies what would incentivize them to the move drugs board for a smaller market than what they were originally anticipating.
I'm not sure that we need here to -- there are a variety of reasons one could abandon a drug in the development process. That all drugs are abandoned because of a population problem. Do we need to capture all abandoned drugs are we tried to incentivize the ones that might fit this profile of being able to target a smaller sub population?
I think we should be careful about making recommendations feasible or that we have no inkling of how we could ever incentivized because we have a an inkling about how one could and said let's drug companies to make such affirmation public. I'm concerned that we delete our recommendations if we just say, he should do this or we should insist was without any idea but.
In the fact-finding stage that is next week is certainly ask. We could ask what those concrete incentives would be. We can certainly ask industry what they would consider incentives. Ability.
Are there concerns that we need to keep these here?
I think that second paragraph is really valuable because the individuals I know at FDA have all worked hard with industry for the voluntary submission of dynamic data and to reinforce that voluntary submission of proprietary data to an agency that can gain in its balance as it makes decisions about drug approval -- this is not for abandon drugs. This is what things in development now. That is a wonderful thing. Coming from the NIH side of the fence I would like to permit that ultimately being made public. Realistically how many companies will voluntarily submit data to that NIH -- FDA they think they will be forced to make public. That will have a chilling effect on the Hill and submission system. So I understand the competing nature of the issues here. What you have resent is good in that second paragraph. The confusion as at applies to more than a band and drugs. It applies to things in development now.
It certainly could, right.
It does.
The question is could we narrow the scope?
I think your fact finding suggested to the group can't make careful recommendations with a system that already exists is a good idea. You might want to fight to find more or you will drop out to deep and have an unintended consequence. Would you agree?
I think we can certainly in looking for instances discover that. Michael?
If the community is saying is this something that is absolutely needed the move pharmacogenomics along -- and you can't identify any real way of doing it with the existing data, should you also consider another recommendation cadet corps of implementing that? If this is not the only way to do it --
You are saying if this turns out to be one of those things that is up and lamentable then what do we do, right? Let me see. That is definitely a conditional statement. Since we don't know what the if it is true I don't think we can quite get to the then. But that is certainly something we will have to look at. We but have to then address that particular situation. That is a good point.
What is the resolution? The resolution can stand at the moment but we must find what those incentives might be so we can be more specific in recommending to the Secretary, this is what we have discovered from industry or from or fact-finding and not to say that you have to do it this way, but we would at least be able to make some concrete recommendations along those lines. Such as, we are doing some of these recommendations were we say such and such is already under way. We could say, these are things that have been identified as possible incentives. Deborah?
But these seem to be too different recommendations that are bundled together here. One is abandoned drug and encouraging industry to move abandoned trucks Ford if they are abandoned because of an adverse drug reactions in a small population but it shows effectiveness and those that don't have the adverse reaction or other things that pharmacogenomics testing could help with to identify the populace is that would be helped by these abandoned trucks. Then that the incentives part really applies to all drugs. You want the farmer could genetic information for any drug and not just abandoned ones. It seems there are two things here being mixed together and appropriately.
Okay. What we were trying to do was to narrow this down. We could broaden it. If the community thinks this is better you could just Saddam -- HHS should advance the facility of drugs about what incentives would be needed for voluntary submission. We could just drop abandoned. We don't have to have that in there. Emily?
I think the original discussion about this was this whole concept of a drug rusty. There are probably good drugs out there where if you could eliminate a few individuals who had bad reactions to them would be great for the majority of people for whom they are effective. And so I don't want to -- and I think we were trying to take a very narrow defined sub set where we thought it might not be as sensitive hot it. Your truck was abandoned or pulled off the market anyway. So you are making no money off this. Is there some way that pharmacogenetics could help bring that back for the benefit of patients? So I don't think this was ever intended to be a broad recommendation that every drug and every company had to collect this information and review it to the public. It was really in its initial discussion was focused on this small subset of the things that are off the market now for one reason or another. So the stakes are pretty low from the company point of view because they are making no money for them right now, and this might be a way to resurrect something.
She is saying this is the data that would have been submitted ordinarily.
So what do you think of it? The idea was to pick a battleground.
I understand what family is saying which is what I was saying about this first part of a slide 39. By facilitating access to get permission about such trucks -- facilitate information to home to what with is just very vague. What you want to do is encourage the pharmaceutical companies to the move to these drugs for word by using pharmacogenetics. Do they have to give proprietary information to anybody?
My experience is that most companies, once it is done it is done. So the chance for them to resurrect it is not as good. It is an emotional thing within the company. It is much more likely that some other company would take it on and buy the rights for that drug and do these studies and try and show that although the drug was not safe or the total population, if you do this test it then could be used to effectively.
So is this recommendation capturing what needs to be done? I don't think it is before we get too deeply enmeshed in one, I'm going to pull a read and we are going tough like this for just a moment and we will come back to this if we have time at the end but we will say this is a problematic recommendations and we will try and see. If we don't get back to it today people certainly try and rework it in such a way to make it certainly more clear what the intent of the recommendation is and how it addresses the issue. We are running a little behind.
May be what people can do a test to help out our chair person, if they could drop on a little piece of paper what you think it ought to say. Just try to give him the solution to the problem and had set in and he can look at it on the break.
That is good. Going on tasks 2D, this is one that was a flight as a high priority by the task force that FDA should examine the humanitarian [ indiscernible ] so it is extended to pharmacogenomics tests that are intended to be used in conjunction with the drugs. Yes, sir?
FDA again.
I want to hear you.
First of all I want to make sure everyone understands the humanitarian device exemption extends to test that art intended to be run on 4,000 people or less or on a population of 4,000 or less depending upon how you interpret the rules. There is no clinical validity required in order to have a humanitarian device extension, and that is based on the assumption that a potentially flawed test is better than no test. I'm not sure -- I suppose you could read write the regulation to model it differently, but I'm not sure that is where you want to go with orphaned trucks right now. I also think that if you extended it to that 200,000 that orphan drugs are now allowed you would create an extremely unlevel playing field for Genetics courses every other kind of test. That would probably be a somewhat upsetting to the rest of the community.
So now in doing what this says, from what I understand you just mentioned, it apparently would change the rag for tests so that they would have to have clinical utility, right? Because they would be falling under the orphan Drug designation.
I am saying the current humanitarian bright exemption requires an assumption of clinical Lidice, not utility.
Right.
Right. So my feeling on reading this was that he simply wanted to talk up the number of patients who could receive the test under this exemption.
If it is done in conjunction with an or fund drug.
Right. So what I'm suggesting -- suggesting is that as the exemption is written now you are running a test on 50,000 people for which you have no clear political validity with the assumption that the ACT test may be flawed and that a flight test is better than no test.
Thank you. We may have to go back and look at this also.
That is good information.
We will also flagged this to go back to. When we will go down to that whole area of clinical below the pharmacogenetics utility of the pharmacogenomics and the people who mentioned accuracy can recommend wherever they want that to be put in. Here is our first recommendation in this area that HHS should provide 88RQA, CDC and it NIH with additional information to identify pharmacogenomics technologies that are important from a public health standpoint to address gaps in evidence through which clinical validity and utility evidence is lacking. So CTC working group and he genet and 8H part to seek to program may be appropriate mechanisms or models for identifying such technologies and specific evidentiary and research needs. This was applied by the test as a key recommendation.
I'm sorry.
Please.
An addition to political validity and clinical evidence, as I say, we have to somehow incorporate measures of accuracy. And all sober utility evidence, I think we must go further than that. Some people think that pharmacogenomics tests are diagnostic tests. And diagnostic tests will get clinical accuracy and accuracy tests and also at how this test will be used in terms of management of the patient. I think the wording of management will must be incorporated in this because if I'm evaluating a specific technology, not only CMS, but also other insurers -- if tests are lacking in terms of accuracy, at least that should be demonstrated that if a physician uses that test and the result of that test will dictate his or her change in management of the patient, if there is some way we can incorporate management because that is what we look at when we look at a diagnostic test. How does this best alter or continue the management of that patient?
Now the management issue, is that one that we want to put in R&D or could that be later on in the application?
It is also applicable in north seven, but if the studies do not show that the management can be altered -- if the studies cannot be shown that these tests help in the management of the patient then it would be difficult to say how it is applicable in terms of a clinical application. You might even look at a decision tree depending upon the results of the test. Does the position to eight, B, or sea. It is all involved in the management of the patient.
But just wanted to bring up that to address Jams issue about accuracy, the easiest solution here would just be to talk about analytic and clinical validity the way to do in the narrative because that will cover analytic sensitivity and repair disability and fell your rates and all of that accuracy.
Just one second. What page are you on?
Thirty.
Let me read what we have in the report for clarification purposes. If you look on page 30 of the draft report under the section of clinical quality and utility -- I'm not saying that we can still make this more specific, but it says clinical utility -- comical validity refers to accuracy with which a test predicts a given political outcome. Clinical utility refers to the ability of a pharmacogenomics test to inform critical decision making which might include management. Prevent adverse health outcomes and predict outcomes and considered important to individuals and family. You may be aware we are not getting the thing here. We are not getting this into the recommendation. Not everybody understands these terms in the same way. Does that capture --
I was it just going to say the sentence before that was the description of analytical liberty which is a very important in this context.
Okay.
I was just going to say I think clinical utility as it is defined in management seems like it is encompassed in utility.
Maybe would we need to make that clear at the recommendation or is that okay to make that clear in the report to?
When we do technology for example somebody said that it cyber genetic testing. And they submited a lot of articles talking about a specific marker. But they don't connect or they don't blank how the results of that marker is going to result in the management of the patient or change the management of the patient. That is a link which I don't know if we are stressing hard enough. There has to be some kind of link between the results and help the patient will be managed depending upon the results of that particular test.
That is critical utility.
Time to sign if you ask a clinician and clinical utility that is exactly it. It is the management of the patient.
We can work to make sure that is clear in someplace.
The idea would be to add analytical and clinical validity and utility.
The political thing is easy to do predigest put analytical in there with clinical.
Right.
Two things, I support the suggestion. It is the intent of this to focus the political outcomes and not to validity. I'd think we need to capture getting that information. So perhaps you could do that in the first recommendation when you are talking about basic research. There is no mention about analytical validity here. So the intent of this is to focus on the clinical outcomes and to make sure that we don't use that any way in these recommendations. Week could perhaps make it more specific in the first recommendation.
The first being which one?
De NIH should support the basic -- in my reading developing new knowledge about a test, there is the specific language about analytical performance of the test. And so if the intent is to leave the recommendation more focused on the comical of comes and we could be more specific and laying out the political performance in the initial studies.
But so what you are saying it is none of them -- put it in there?
Right.
Following on Reese suggested on right where it would go and give it to us at the break that would be happy to get back to that.
And second, it was I'm speaking here from our perspective. The evidence based practice program is identified, but when we do these reports or technology assessments they are useful and pointing out the gaps in evidence. There is not enough or amenable to creating new knowledge or doing it out comes research to fill in those gaps. And I'm reading through these and only within the you see we have some -- A sub sector where [ indiscernible ]. Here we are making conditional in certain circumstances. So I'm not sure what are the mechanisms for Britain in creating new knowledge for clinical outcomes? We don't really specify that in any of these brief here. We could make more clear by specifying both Denton's, [ indiscernible ]. We would then be identifying mechanisms to fill those gaps. That would be programs like the deciding that work at ARC or the search program at ARC which are more creative for creating new evidence as opposed to appraising existing evidence.
As we did here we are happy to be specific, but would you want that here? That could also probably got here.
It could go anywhere. I did want to raise it that I don't find it.
So we could add that here or over here. It looks like that would be a good place. Thank you. Okay. All right. 3B is one that was passed as the "taskforce as high Purdy. FDA should encourage [ indiscernible ] and post market surveillance. Request manufacturers permission to make this bid available to the public. Manufacturers should disseminate any specific and not specific findings on the validity in clinical utility of pharmacogenomics Technologies through publication in peer review journals. Yes, Emily?
Reading this the gain in isolation which manufactures are we talking about, drug, a device? I think it needs clarification.
We need to clarify.
I'm not familiar and done with the regulations of drugs enough to say, but for devices evidence of clinical utility is currently not a strict requirement. Many companies are performing these tests and would like to get them to market as quickly as possible and it is my assumption that is what the community would like as well. If you delay giving top market by enforcing the provision of a strict clinical utility, you may be working against yourself.
Some supposition of clinical utility is needed, but actual outcome studies and so on generally take a long time and are not traditionally done for devices.
And this one -- short, Deborah. Go ahead. Allen, yes?
Your bike is not working, I think.
A few minor points. You might want to include at the age should encourage a manufacturer to include a pharmacogenetics. Right now is all particle utility data.
On this one -- okay. Deborah, did you have something?
I want to point out that in December seeder develops a table of by markers and tests that actually have an indication of three levels. One is informational and the second is it is recommended and the third is that the testing is required. And it provides information with the drug label and it has references in that table to the studies that have been done to support the pharmacogenetics tests relative to a specific drug. And it was initiated in September and updated in October and I think this community should encourage Cedar to continue to update that table of information because it is extremely useful as a help system. When we found that it actually supported some of the pharmacogenetics implementation stuff that report doing. So in this recommendation the FDA is actually making this information -- I assume cedar is part of the FDA. I have problems of knowing who is what. But it's the cedar of the top of this table thing. So the FDA is actually doing this, and that is great.
We could put that down if it is okay. We have e.g. through publication of turtles or through the table that is being --
Through the Cedar Web sites?
Right. Just a clarification on this to address your issue, Elizabeth. If this said FDA should encourage drug manufacturers to submit, that would take the focus and put it on the drug and we wouldn't have the device issue. Is that correct?
I think so, yes.
That is the specification and then we could do the pharmacogenomics, pharmacogenetics as well as the clinical utility data.
I would also make one other recommendation here. This is to request manufactured permission to make this available to the public. I certainly, encourage it but I'm not sure what you mean by request. When FDA makes a request it is viewed as a regulatory matter. So maybe the wording used to be changed.
Right.
Increase.
Encouraged.
Good. Thank you. We don't want to intimidate anyone. Keep moving. Draft recommendation three c. In certain instances public and private health plans should facilitate the knowledge but pharmacogenomics technologies. To collect data on the clinical validity and clinical utility of pharmacogenomics Technologies. Did I read that correctly? It didn't sound good. Anyway we draft coverage with element initiative which serves as a model for this practice all right. The our good on that one. The.
Am a little concerned because you are mixing payment with clinical research. And is this clinical Research meeting informed consent? And the person doesn't consent they don't get payment for their [ indiscernible ]?
Okay. What we could do is, I guess the lack of clarity here is in certain circumstances. Aha all right. So considering the question you just raised, I think we need to potentially address that problem.
Yes, and whether it is identified data port on identified data, you know, it is kind of --
Lets up like that one. If we put it out here now we have to deal with those issues. In fact, Soviet, if you could just got something done for the break, that would be great. We will see if we can work that around. We move on. Now we are moving on to the research databases. So HHS should work with the private sector to improve data sharing in it dropper ability among research, health record in claims databases. HHS should work with existing organizations to create uniform to Numic data standards and if what ways to harmonize data and analysis methodologies and develop an infrastructure to enable the exchange. Comparable efforts to standardize data is also needed. Again, flag by the task force committee as extremely important. This ties to get at the question we mentioned before about how the different group and databases can talk to one another. Everybody happy with -- yes, this is all right. And then for be, as did his share privacy of patients and research subjects should continue to be of paramount concern and HHS should take steps to ensure that the confidentiality of their data is not compromised, identified by the task force committee in goes back to the balance I mentioned before we are trying to strike.
I have an observation. I don't know the solution, but if privacy is of paramount concern [ indiscernible ].
Yes, it will. That was what I mentioned earlier. It seems to be that --
Clearly this was written to allow a institutions to hold onto as much data as possible when it comes time for them to deposit into databases. This will give them reasons that not want to share their data. Is that exactly the way you want to push it? Please comment.
I'm glad or so but this up because I think this will be an issue for all kinds of issues and pharmacogenomics will be one example. Kelly privacy and confidentiality are absolutely important principle, but if one decides that that is the only possible, then basically you have no research databases at all because somehow they might leak or somebody might get access to show that. This is worded in a way that almost makes it sound like that would be your intention. So I'd be, perhaps, choosing your wording a little more carefully here Ted say that confidentiality and privacy are critical principles and every effort should be made to maintain them while also making certain that research can go forward by providing access to a qualified scientific researchers.
I think that is a very well crafted solution here. I think it is -- and I think the community knows it's obvious we have to push hard on the privacy Paramount attentiveness. I know a lot of this stuff is happening in America's health information community which we talked about earlier and it is really threatened by the concern of the public around this privacy confidentiality deal. In some ways if we are not attentive it makes the whole agenda a nonstarter. I think the way Francis raised it is the way to get at it and if somebody has that language I hope you are writing it, not that you need to say it again.
My only fear is the way that you stated it does the exact reverse. It says that -- just listening to the way you phrased it, this is an important concern, but the research must go forward. The must is the key. It makes it -- I agree with you we must balance this in our language, but I think you all right.
You don't work here.
I think we currently have -- there are regulations to protect the privacy of human subjects that interest up this research that can be used in to these databases are clerical validity except. We use identifying information. So the king is of what is currently in the regulation to these will suffice that we assure some of the privacy of these individuals but then we allowed to continue research.
Go ahead.
I think that was the same point. We will get the demographics out of the shares will still protecting the privacy of individuals.
Sure.
And the other thing, this is a battle but outside of my purview, but I would suggest that you have a couple Edie's on some of these things. There are models that exists like comical networks and that sort of thing that may be considered how they go about doing the data sharing and still protect -- all tamale project privacy. So I will say e.g. along clinical network lines that I think some of the people --
For this is the concern was kind of with the language, were is the bottom line going to be. And I think we will work on that to show this will be a continuing thing. The question is will it be creative or destructive? And hopefully we will make it created. We are scheduled right now -- how many more to give? Fifty-three people 25 and take a break. All right. At the age for guidance population data draft recommendation number five. Race and ethnicity categories should not be used along when analyzing differences and drug response. They said develop guidance that encourages the connection of other biological factors that make the jury finds differences in drug response. When drugs are shown to be effective in certain racial and ethnic Serb populations FDA should require manufacturers to conduct additional studies to identify biological markers that underlie the differential drew response.
The only problem I have with that is the requiring manufacturers to conduct the studies. These are extraordinarily complex reasons that range from environment to genetic factors that may be responsible for different racial and ethnic categories responding differently. And I meant that by demanding an explanation by that when those things are so complex they have proven extraordinarily difficult to work out. I to think that is overstating it. Let me see if we can get at this in some creative way because I understand what is being said. It says we should require manufacturers to conduct additional studies. Doesn't say required manufacturers to identify.
I don't know. I would argue that if you do show in a statistically rigorous way that a certain group be the people with blond hair or people who live in Love Canal respond differently to a different drug. I think it is laudable to look for those, but I'm not sure if requiring those studies is something that makes a lot of sense from the FDA stepped point.
I think I'm not sure either of what this is trying to do. When you pile on all those burdens on the manufacture it release instead me to have a discernible effect of bringing a product to market. And I'm not sure tell what advantage you get here.
The sense I think we were trying to capture here and obviously many of you are aware of this in the literature discussions going on about the potential effects of pharmacogenomics rather than a million rating racial categories and particular disparities in health care delivery that these would actually exacerbate. We could probably take require and put encourage progress I think you address it poorer well and the first two paragraphs and I don't think a third paragraph as much and does make it rather confining. So I think testing at the third paragraph makes it very reasonable.
Alan?
The -- as you know there is a drug out there that currently has proof of that. So I'm not sure what you are exactly recommending. Are you recommending we take it off the market?
No, the recommendation is that we -- and there have been articles addressing this issue. If one did a study of the population that is supposed to target, what would still probably find a spectrum of response to that drug and one might even find a response to the biennial competition outside of that particular group or you have high responders. So the question here is, is using the category African American something that is socially problematic, problematic to a particular undershirt group as it is in such a way as to say this social detriment basis issues that we need to address, not necessarily by taking the drug off the market, but perhaps by better informing ourselves as to what it is that delineates that population for which that particular chart is actually beneficial or significantly beneficial. And that is the intent.
I would make a recommendation -- I would actually make a comment that some of these racial and other criteria are an international agreement and that is just one comment. What you are really aiming for is to encourage us to move forward from a racial to aging comic, pharmacogenomics --
Absolutely. That is the idea.
We might be revised to state that so that race and ethnicity are found to be determining factors, pharmacogenomics should be looked into.
I'm not sure if I getting to where you are going.
I think we will try to get that in the second paragraph foreshore, but in any case -- all right. So one of the recommendations is to cut out paragraph three is what you are saying?
I think what we really are here is that when you make an interesting observation that there ought to be efforts that other facilitates the further research into what is actually going on or the recommendation is that you are trying to make sure that there is a database made available to support such research -- and it looks like you are trying to use that to the team as a way of facilitating access to the data that researchers can then use downstream, and the question ultimately becomes -- I think if I here the people speaking at everyone would be -- would say that it is a good thing to learn more about what this observation means and we ought to say yes. But the second question is, can you put on the back of the manufacturer a requirement to do that? And I think we are rejecting that in this in per referee. The second question is can you put that on the back of the FDA to supply some mechanism for that to occur, and it seems to me that FDA is saying that might be problematic as well and I'm not sure what your answer is. What we think we are left with is this is an important thing to study and people must pay attention to get in in the best of all worlds we should go after it that smart people would decide to think about it.
If we put HHS in instead of FDA, that takes the burden of of the specifically but it allows greater breadth.
So let's not undercut this too severely. Many people do believe that the ideal experience was an unfortunate one. I happen to be one of those buried the re-education of the racial categories in a decision about to get this drug war that truck both does a disservice to the public health because it substitutes and imperfect proxy for what may be much more specific information that this wasn't collected that might be too would respond and. And has the other possibility of applying to the public that race is something that is biologically determinate and because the FDA has approved this drug for African Americans they must be somehow different which is a vast overstatement of the biological facts of the matter. So I think it is highly inappropriate in this set of recommendations to put something into discourage that kind of occurrence again and I think FDA, appropriately, should be asked to develop ads just as your second paragraph says here to encourage manufacturers who are putting forward this kind of a test to do better next time.
I have somewhat of a conflicting opinion. I think that race and ethnicity are certainly very imperfect surrogates. On the other hand, to be want to say if that is the only surrogate you can come up with, forget it. You don't have a drug. I'm not trying to say he said that. I'm saying my opinion.
Joe?
Yes, knowing that this is more than -- I'm looking the wording they are using which is -- I think surrogate and proxy were the words used. A really what is the meaning behind this and what is the intent to do and what I'm sensing is along the lines that there are areas at which disparity's occur through which there are unevenness of ways this is done. Maybe the point here at the first two paragraphs but then to refer to add put something in a low but stronger language in your Elsie the section to be able to do this. We then get at the intent as well. You can start with the intent and leave it at that with the first two paragraphs. I would agree with my colleague here. Then gutted the issue blessed to help spur the which seems to be something that the community and everybody is in agreement as to be addressed. But maybe you can make a stronger case or that. That is something that came up in our group as well, we could find a better place or something like this. That would be my recommendation because I think why try to -- I think it is very confusing many many times to use the proxy or use the other terms being used. Plainly this is what the intent is and I would recommend that.
And we do have in the third area some of those. We could job the paragraph out here and make sure it is emphasized.
You could always say this is the recommendation for the intent here. I think you do need to address the tenth.
I would just like to emphasize where Joe is and take Francis's point. I think what we need to state -- I need people are reading to different things here and so I think we ought to describe what your concern is. And Francis T. it up very well. Because you don't want to see a misuse. However, in the more positive activity there be a step be the attitude to encourage research. So at think if you did what your -- because nobody else knows what you are talking about here. So you need to declare what your anxiety is.
Okay. All right. We are a little late for our break, but let's do it right here if that is okay with everyone. It is 11:00 now. We are supposed to have a 15 minute break. If you are not here at 11:15 a.m. all well will be fall you. And we will have the cameras turned to your response so all of America will know you are not here.
[15 minute break.]
Thank you all for resuming. We appreciate it. Right on time for -- as always. What a great crew you are. We are at a critical part of this discussion. I cannot talk in the latter. We are going to shoot to Anna. She didn't hear me. Well, I mean to tell you the there are so few people with whom will will be fall. It is amazing. Mr. Chairman, let's keep going.
All right. First of all, I did want to thank everybody for the comments and the insights. They are greatly appreciated, however I did overstate the case did little bit earlier when I said this was our opportunity to realign the universe and move stars around. The one thing I brought to tell you is we cannot miss with time. It just keeps going. So we must keep going. If we hadn't can be more insistent and targeted in our comments that would be greatly appreciated. But I do not wish to cut you off from making comments. So we bought move on to gatekeepers. It is important to understand what we mean by this term, and these were the groups that we're identified as those that can enable, ," or redirect the course of pharmacogenomics technologies. And therefore they affect the integration in the piece it access. We divide these entities into four groups, industry, the FDA, CMS and other third-party payers and clinical practice guideline developers. Again, these were the ways that we put it out and thought this was perhaps the most constructive way to do it, but we are willing to hear from you on that issue. And looking at these groups the points of our discussion covering the major issues and is there anything we are missing and what are the high priorities. So looking at the role of industry observers manufacturers perceptions of risk and return on investment, and floods, whether and how far could Numic products are developed and marketed. So there are disincentives to develop pharmacogenomics products. It can lead to a segmented market which can lead to decreased profitability. It can cause additional responsibility involving coordinating code developed products. Then there is the role of that FDA, FDA approval defects manufacturing practices, conduct of clinical trials, post marketing surveillance, access Tech pharmacogenomics products and their use in clinical practice which raises questions about the adequacy of genetic test regulations which we built also get into this afternoon so we don't have to solve all those issues here. The extent to which genetic data submissions will be required, premarket review of product and labeling of pharmacogenomics products. The goal of CMS and other third-party payers, ability to obtain coverage in federal reimbursement critical to manufacturers' willingness to involved in R and D of new products and the challenges here include the fact that Medicare does not cover preventive services and private plan coverage may be difficult to obtain, especially because of limited chemical LSD and utility information. Reimbursement may not be adequate and uncertainty about the region and plans evidence expectations. And then we have the role of the clinical practice developers. So the availability of the guidelines affect the coverage of products and their uptake by health providers and evidence based practice guidelines for pharmacogenomics products are indeed lacking. So looking at this, are these the major issues, have we missed anything, are these the things of highest priority and I open it to your comments. We haven't got into the recommendations, it just the issues. Everybody seems all right. This is good. We like this brevity. Let's look at the recommendations. Do they work as they are currently worded? Is there anything we are missing and should some be deleted. In looking at these regulations this is 68 which was flagged by the task force as being of higher priority. CMS [ indiscernible ]. He did. I looked at you and then I looked away.
It is true that CMS looks at certain pharmacogenomics test as being diagnostic and the patients who have signs or symptoms of a specific disorder. It does not look at pharmacogenomics test as bank diagnostics in patients who don't have signs or symptoms of a particular disease. Again, going to the point of, we don't cover preventive services and for a person to have a predisposition for a specific genetic disorder even though he or she may not have signs or symptoms of it, for that reason it would not be covered under that specific scenario, but if a patient did have signs or symptoms, then it would be covered. And I think this is one of the recommendations which was made that we made earlier. And I think that is purely -- the secretary is looking at whether or not Congress can give us a designation for a prevention category. But at the current time we don't have that.
And if this were to stay at is -- as it is written it would be supporting that change. You would have a pension category.
Correct.
Correct.
Said the question is do we want to support that change?
We should make it a recommendation to support that change so the undersurfaces are being met.
Pardon?
Could we make a recommendation to support that change ticket.
Well, this does, I think in essence, do that. You want to be more specific and say, for example recommending that --
You could say for example in addition, to covering patients who have signs and symptoms of a particular disorder we are proposing that patients who have a predisposition for genetic disorder, even though they don't have signs or symptoms, the genetic test should be -- they should be allowed to --
And as I have just been informed that would make us consistent with the coverage report we have already sent along. I think that specificity is fine to put in there.
Yes?
And I think another way to say it, if James think this is the title, this should be primary and secondary prevention and it is bidding at the heart of the matter, primary prevention when individuals are a symptomatic and don't have the signs and symptoms, Medicare would not pay for it. But increasingly there is secondary Branson when there are signs that Medicare would potentially before.
I guess I'm a low confused about signs and symptoms of we talk about pharmacogenomics. If somebody comes in who has a diagnosis, signs and symptoms of an illness and they need a treatment, the treatment would be optimized if a pharmacogenomics test was first done to assist with this was the right drug at the right dose. Would that be acceptable under Medicare's definitions of when they will cover this kind of test?
Yes, because the patient would have signs and symptoms and/or signs or symptoms of the disorder. But they wouldn't get have signs or symptoms of an adverse drug reactions, you are not requiring that?
No.
That's good. But this would say is the earlier conversations we had about done prospective pharmacogenomics testing is for instance with ARC PD in the military would not be something Medicare would currently cover. You would have to come in with a bag possible signs and symptoms containing illnesses for which drug therapy is needed before a -- Medicare would cover the cost of doing that pharmacogenomics test.
At the current time that is correct.
Obviously I would agree then if it is time to expand the universe of opportunities to the perspective, that would be a good thing and it is consistent with what ADDS is currently recommending.
Okay.
I don't know if we need a recommendation on this or not, but I know that CN asked as employed the device or approach of the least costly alternative and in the area of pharmacogenomics perhaps that doesn't apply or is more difficult to apply because you can't just say here are two drugs that are comparable and we are going to pay for the cheapest one. They're is budget reason for that and I'm not saying it is invalid, but as we drove down deeper on the science developed such that some people could not use the least costly alternative, perhaps there is room for at least acknowledging in the body of the report. I'm not certain it rises to the level of a recommendation, but as long as we are at the CMS section I thought I would bring it up. I'm not certain what the recommendation would be. With.
As part as this recommendation goes that would still be in place even if we extend this recommendation to a preventive mode as well as a diagnostic one where signs and symptoms of already present, is that correct?
Well, this is --
Test.
And the other would be more once you have the test would there be with you use.
All right. You got that? All right. Thank you. All right. I think we are definitely following gems and Francis's comments. We will definitely make this more specific and make it more clear that it is consistent with our earlier coverage report. Okay. 6B, health insurance plans should be more transparent in terms of how they make recommendations for pharmacogenomics Technologies by developing guidelines that define the type, quality and standards of evidence that might be bad for pharmacogenomics, technologies to be covered. Whenever a specific pharmacogenomics is denied coverage because it does not meet these evidence theory standards, health insurance plans should inform the test developer what additional evidence is needed. Yes, there?
I mean the skunk at the party. Topeka and I don't know if it goes there and I think these mechanisms are in just fine. The question that I miss -- and I don't know if it rises to the level of a recommendation or suggest simply be touched on briefly in the report. That is the impact of pharmacogenomics on the development and use of health plan formularies. Formularies are used in an aggressive way to help figure out what their piece our best, how can we manage cost, and this is something that is quite extensive in the private sector -- sector and of course Medicare is subjected to that as well. There is a difficult tension between figuring out what drugs and their piece you are going to have on your formulary and reimburse for and pharmacogenomics because you may have a certain drug on your formulary which you will pay for, but some person could not benefit from that drug or therapy because of a particular genetic issue or marker. So I'm wondering if it is worth considering a recommendation about not making when we have evidence like that, concrete evidence, not making that individual go through a rigorous appeals process, you know, the standard thing you have to do if you are going to go off formulary. I don't know what the recommendation would look like. I haven't thought enough about it.
What about -- I don't want to jump to the next one before anybody comes in, but the next is about addressing evidenciary gaps, and that is pretty broad, but it would sound like to me in one sense you are addressing in evidence theory gap.
I'm saying is that when the evidence is out there already, how do we manage the tension between health plans to use up formularies and making sure that people have access to the therapies that they need that may not be on the formulary. We don't want to eliminate a formulary, but we need to somehow reconcile the two.
Read?
I am going to be careful here because I'm from that industry. I don't have a conflict. But the use of the word more, they should be transparent. It implies that they are not and I don't think that helped spur a.
I see.
Rather than --
We are just trying to get all the flaws out of the class, that's all. Mark?
All right. Thank you, Cynthia come on that. Do you think we need a recommendation directly to that? A formulary recommendation?
If I had one I would but it out, and I don't. I think that a minimum it should be acknowledged.
Okay.
As an issue.
But perhaps someone else has an idea.
If you do later you can always drop a line. Okay. HHS should provide resources to relevant agencies to address evidentiary gaps identified by health insurance plans. That is mentioned. We are good.
I mean, I'm just trying to be -- I want to -- I don't want to be the start of the party here either.
We are getting so many that it doesn't much matter.
To say that the secretary should provide resources so felt evidentiary gaps, the budgeting process and the prioritization, are we getting ready to come back with a large pot study or there is a lot of stuff going in play here. I think it is kind of tough to make a serious recommendation that HHS should provide -- it is pretty definitive year that we are saying this is more important than some other things. I'm not sure how to handle this.
Of Cauvery's point and maybe Doctor Dunning can't comment as well, I'm not sure if this recommendation what Ford of the secretary's office would know what to do with it. There is more in the briefing prep package, but it is a bit broad --
I'm just making sure it is the same at it is in here.
The problem I have with it is as much as from a manufacturer's point of view I want to say this is all on HHS shoulders. I don't really think it is. I think there is an obligation for both parties that manufacturers, drugs and devices to play a part in closing the gap of evidence so that HHS can move into normal clinical practice and it is not just a HHS activity.
So would you want to delete or be right or --
Well, you know, I don't know. I'm not in the delete mode, but I think you need to definitely say, encourage public-private partnerships or something in Baird said --
Okay.
I am in the delete mode. I think this is so broad as to be meaningless.
But you don't have any specification you would give to it to make it meaningful?
No.
I'd just want to go back for a second. Different health insurance plans will help to their make a judgment call on standards of evidence and whether or not something merits inclusion in their and their services. So there may well be that different insurance plans will have different interpretations of this and offered different pharmacogenomics coverage. That will be a marketplace issue and within the consumers to go out there and say I like this insurance company because it provides me with this service. Is that the intent of putting a burden on the insurance plans to sort of make that judgment?
Should we at least be transparent about what they are doing?
Sure. Or even be in that will and the first place. So each insurance plan will then have expertise on making judgments about pharmacogenomics. Right to give.
I think this takes us back to the general conversation about genetic conceptualism and so forth. At the end of the day all pharmaceutical and technological issues are very clearly -- all health plans follow a pretty rigorous and pretty standard way of viewing the evidence for any of these new things. A lot of it is based on a CMS Bennett's first of all and so see him as is enormously important in this and then we all have various ways of doing it. So I don't think there will be any Super special thing about pharmacogenomics per say. I think it is just simply, is it in the literature, does it state [ indiscernible ]. And then what is the stuff that Cynthia and Debra commented on in terms of the availability of cost effectiveness kinds of information so you can to the pharmacogenomics and so forth. So the point is I think is that this will be handled the way that everything else is handled having appropriate research and literature assessment available.
Okay. Thank you.
I'm wondering if this is not overlapping with 11 be which is also talking about research and coordination done by the HHS. To me it seems to be speaking to the same issue.
Okay.
So my sense what this is we may have hit a delete for the most part of this because it is rather broad.
Okay. I don't see anybody dying in this trench. We will let that one go. On to the next. So again the next part here is the implementation section. And this is taking information that is developed and the research and gone to the gatekeepers in putting this out into clinical practice. This would involve education and guidance and information technology in pharmacogenomics and economic implications, ethical legal social issues and the coronation of the HHS activities. Ability so again which are the major issues? Are these adequate and sent -- should we get rid of some which we have actually done. For education and guidance, genetic education and guidance by health professionals and regulators is insufficient. The limited information is available through labeling and practice guidelines about how to interpret pharmacogenomics test results and how to use them to inform treatment decisions. And these are the issues, Genetics education is needed to help consumers make informed treatment decisions. Direct access to the pharmacogenetics testing through over the counter sales or direct the consumer marketing may increase in the corporate use of these tests which could lead to increased health care costs potential from misinterpretation of has results and misinformed decisionmdecisionm aking and adverse health consequences. The uptick of electronic health records is still in its early stages with no consensus yet on how genetic information could be stored in these records that he should have access to the stored data. Obviously lack of harmonize standards for storage and extent of genetic data and need for pharmacogenomics decision support tools and reminder systems. Economic implications, the use of these technologies will likely add to health-care costs, at least in the short term. They need to examine the benefits and costs of investment in these technologies and there is little research. We have heard this before. What are some of the issues that we haven't raised it? Financial barriers to pharmacogenomics to all the that has been raised now. No insurance could result in excess disparities and concerns about genetic discrimination which we talked about and liability risk of the provider fails to administer recommended test. In the coordination area there are lots of activities that are ongoing. Be have a list in appendix A. There may be more there but as you can see it is an already extensive list with 23 pages and there is no single coordinated framework or action plan to address pharmacogenomics talent is or shared information about activities among the federal agencies. These are the issues that we have highlighted. Everybody could with these issues? Anything we have missed or that you think is an appropriately highlighted?
Me eye test, I do think there are some activities on behalf of professional organizations, professional medical organizations and coalitions. Slide 65 is sort of dismissing everything as insufficient. There are activities going on to educate practicing physicians to incorporate into medical curricula among the genetic counselors, among human genetic testing groups. So it is a little bit --
I particularly want to tell you the intent is not to dismiss everything. The intent is to acknowledge even in spite of what you mentioned it is not sufficient. We don't want to stop here. We don't want to say where we are is a good place. We can try to make that --
[OVERLAPPING SPEAKERS]
How about we say it is currently insufficient. Is that it?
Okay. We don't want to downplay anything because everything going on now is certainly needed, but we need more.
Attend just to touch on this point and slide 68 this implication that farmer cut genomics will add the health care costs, well maybe not of what happens there is you reduce the incidence of adverse drug reactions which cost a huge amount, both in terms of health care economics and in terms of human suffering. Somebody that is a little too strongly worded. It is not a definite uptick in the overall medical expense. I would argue that to be the case because we do have the word lightly. If you had to guess which way it would go, which we do you think it will go?
I don't know.
That is the point.
The way you could say it is that pharmacogenetics did analysis are an additional backup cost to the health care system or a new technology. The use of the pharmacogenetics is a new development in the health care system. And the need to examine the benefits and cost of investment or use of technologies is your second bullet. Is new. So it is not something that is currently being done. But in the balance the second point is the question. Is it going to be cost-effective and save on length of stay or at a press reactions such that the cost of 300 hour test out ways the savings.
Remember, these are issues, not recommendations. We are raising these as bases. And in the discussion where we look at the economic implications we do in a sense -- this is bulleting what is in here which does say, let me just read you, the rapidly increasing cost of health care is a concern in the United States with to got to go being among the drivers of those cost. It is a new technology. While new technologies may improve the blank and quality of life or be cost-effective they will most invariably increase total cost.
No, but I think --
Practices point is right.
So you just don't know?
There should be an acknowledgement in this first bullet that they may add to health-care costs, but on the other point might actually reduce cost which is different from cost effectiveness.
Cost effectiveness is it is worth the money.
It is conceivable that pharmacogenomics will save money.
That analysis has already been done. It is incorporated right now based on what we know, testing you would save money over all for the health care system because of those address events that you would predict and prevent.
When you look at that from the infectious disease when we implement testing you increase the cost of taking care of that particular patient, but the overall cost of health care has significantly been reduced. So maybe that is how we can phrase it that we and tested that patient and the overall cost would be significantly less saving.
Sent the ticket.
Another thing we should factor in, and again I don't know -- I think we could craft a bad condition on this. In the real-world application we understand the cost savings over all downstream. Federal programs have to pay attention to what the Congressional Budget Office would say. And so any changes, legislative changes in federal program coverage and other statutory changes are all going to be dependent upon whether CBO besides there are cost savings or not. And traditionally CBO does not recognize downstream savings for avoiding hospitalizations or avoiding and verse drugs. They simply say, what is the cost of the therapy and how many people would benefit from the therapy and multiplied that an ad in a few additional numbers for the woodwork effect and suddenly that is the cost. And it is some 55 frustrating for everyone and health policy because we know in the real world we can achieve savings, but in the world that federal programs have to pay attention to the can't get CBO to acknowledge those savings. If there is some way we can craft the recommendation whether it be pushing for some dynamic scoring or if that is a bad word, call it something else that would put CBO to at least consider these types of data that would help make the case. And it will lead to enhanced coverage at least in federal programs. It is a real problem that we face.
I think that would be a tremendously important idea. I didn't realize that about CPL perspective which seems unbelievably limited. And maybe that should be a separate recommendation. Take out that first bullet and say something like pharmacogenomics may increase costs ultimately in an overall sense it may decrease cost and add something about how CBO perspective --
Just for this issue if everybody would look -- it is on page nine of your executive summary. Number nine is the economic value of our Mycogen Numic -- pharmacogenomics. And that is another realization we have come to. We seem to be getting a little bit of an overlap here. So one way to deal with that would be to either change this first bullet and deal with the issue in the number nine, deal with it here. We have to just figure where we want to go with this. This first bullet could say, currently there is concern that the use of PGX technology will likely add to health-care costs or me add to health-care cost. There is concern.
Or you could just drop the first bullet. And.
And pick it up in nine, right.
I am just a little confused because these are not the recommendations we are looking at.
[OVERLAPPING SPEAKERS]
This is an issue. Nine is where we pick it up. These are the issues we are talking about here. And that goes to nine.
But then let me then argue that the text you read peace treaty because it overstates what the consequences will be pre.
Thank you. Right. So we could say there is currently the reality of concern. Some I think we can say that report that apparently we can say.
Okay.
At think everyone is kind of worried about looking at this one statement orders of magnitude and higher importance of what it needs. By taking it out are reconnecting it they can't --
They can't see this statement. This is an issue that is in the report, but we can be work that part of the report that I did read.
But the critical piece is it will cost a lot of money to develop these technologies, but the goal is to lower health care.
Understandable. All right. Good. All right. We can go back in that section and a Greek word that. Habib any other issues that we covered that are raising red flags? Okay. Then let's get to the recommendations.
I actually had one point. I am a broken record on this point for those in the other worker. The last point is one that has an argument to be made for NIH entire budget. And I hate once again to create an exceptional argument for this area of technology and would hope that the discussion and reported to acknowledges that this is not specific for this technology or this application. It is not a centralized and anyway.
To be honest and not quite sure how that comes out that the report but I will look at that and make sure that is not the case. This was flagged by the task force as a high priority. As evidence of chemical validity and utility for pharmacogenomics accused, HHS should approve the support of Technology says was summarizing the evidence base. These analyses and assess this should be disseminated to professional organizations to facilitate their development of clinical practice guidelines which gets back to something Rochelle mentioned earlier about the way people are trying to get up to speed on this. Comment?
You see a lot of analysis looking at trials as far as case control studies. It is extremely rare that you find an analysis of diagnostic studies simply because of the receiver operated characteristics as well as the changing in point. I would suggest instead of using the word match analysis use the word system reviews and you can say systematic reviews and looking at how test results produced in the management of patients or something like that. Again it is reiterating the word management, but seeking out [ indiscernible ].
This should be including this and others.
Thank you. HHS agencies should collaborate with federal, state, and progress is to develop, Kellogg and disseminate case studies and practice models and the use of pharmacogenomics Technologies. Anybody to do that good. All right. H8S should provide resources to professional organizations that will help enable their membership to meet and establish compasses on the appropriate use of these technologies. Began trying to facilitate what is already on going. We did this to be a good thing.
The drug companies and the domestic companies do a lot of this as far as working with physicians and laboratories and reference laboratories and try to teach them how to use their products. What he suggest maybe industry be included in this Cuban because we have asked industry to do a lot and maybe we can help.
Maybe we should collaborate with federal, state, industry and private organizations.
When you say private organizations does that include industry?
I think.
Update the Bucks -- if it is included in everyone is comfortable.
Providing resources to professional organizations, we are good with that. I'm sorry, said the it is not.
I throw this out to the group to find out if you think that there is a certain element of reporting that we would want to ask providers to engage in. What I tried to do is think about if there is a way to weave performance -- we've pharmacogenomics into the performance concept that HHS might be moving towards which is to incentivize positions to of physicians and other providers by paying them a little bit more to do certain things. And down the road the idea would be for quality measures, but initially I think it will start out as reporting. So if they report certain date in they will get enhanced brought Medicare reimbursement. Is there some recommendation the talk about alter preliminary that what we've been reporting of data, what kind of data that could be incorporated in the paper performance approached. I don't know if the science is still too new and we are not there yet, but if there is a step that physicians would have that would be useful if we what incentive does them to report that data somewhere then that could be moving into the paper performance approach.
We have to make sure they are covered first.
I think this is, Cynthia, right down the middle of the place for what we started the meeting off on when she was here regarding the American Health Information community and so forth. This is the essence of what that is trying to do, to find a way to connect the information about clinical practice that derives from the physicians office records and elevate that up in a more convenient way to larger activities. So I think we should try to find a way to connect that in to the activity. I think that is what we are getting at. With regard to the specific thing here I'm still struggling with this one. I think that if we are saying that HHS should work with professional society to facilitates the continuing professional development of their members, I mean that is fine. But the idea that government is somehow or another going to write a check to professional society to help them do a better job on this area, then you get their radiology imaging committee coming forward and saying, all right, where is my check for that? And it goes on and on and gives absurd. At the end of the day this is what professional societies do. That is what they are supposed to be doing. And so the idea of the government will subsidize those societies to do that, without delivering it there must be some things we can all do to help them to do their job. So we would be working with them to facilitate the continued professional development of the physician.
Soak right. Right there you are saying using the word provide resources everybody will just think money.
[OVERLAPPING SPEAKERS] expedite good point joke.
I was just thinking, because I don't know if this exists already, the effort to either provide a mechanism for coordination or to coordinate and assist professional organizations and coordinating the effort because that would mean that you have a cross organizational or even a collaborative if you will group that has representation of organizations that will probably continue to work on what they are working on, but would have a number of the things built and which is the transparency issue that we needed to talk about, accountability issues that will be there, as well as having an up today real-time assessment of the assessments that are going on. So that is if I am recommending that instead of correlation you provide [ indiscernible ] but the idea would be that you make a recommendation. And this may be a recommendation that is there that this is to be tweaked a bit. That would be a very cost-efficient way. Along the lines of what real was saying but I would say that would actually be a better thing. I think that is terrific because it reminds me Ted nuance my comments. On the one hand I am a legitimately concerned about the idea that HHS would be sending public Monday that is in short supply and even shorter to the society's to accomplish this. On the other hand, the society's get very freaked out if government is going to try and coordinate their efforts to tell them how to practice medicine. I and you must be -- I think the idea of facilitating a rational efforts where people are trying to work together, but the government certainly should try to coordinate medical societies in terms of how they will practice their profession. That is their expertise, but they must be supported. The way he phrased it was good. I just realized I need to give the other half of that balance nuance here.
What do we have here?
How about to facilitate the ongoing professional development of their members oppose that will enable their membership to establish competencies'.
Facilitate sounds -- I know this is -- can you say it works with, work with professional organizations because you sure don't want to imply that the government is going to be coordinating or facilitating could also mean giving money.
I understand that facilitation been difficult because it is what to do, but it -- it's sent to be there are models. And I guess my point is that whatever way the wording comes out it really needs to be a joint collaborative efforts with the HHS and professional organizations.
What we can say is the HHS should work with and then along the lines of and if you can give us examples of those models -- you don't have to do it now but you can use this as an example of how we should go.
Okay.
In discernible.
Once again, industry has a lot of activity in this area.
Right. We could use those models.
What you want is some universal resource, some database or teaching tool that everyone can use. That is not just professional societies, but that would help industry as well to do what they do because there is a significant activity that industry undertakes in working with their customers.
So we are back in 70 again?
No, for both. Use a professional organizations, but --
You are St. Professional organizations and industry, but how does that help enable their membership to be established competencies'?
You will have to change it a little bit call but the role of industry in educating their customers and making the resources available to industry, the goal is to improve health care and industry pays a large part in the teaching.
I've just tried to make sure we don't have the summer else down the road here. Okay. We will flag industry and then we will see if we have it down the road. Thank you. Anybody else?
I have a few concerns about that last suggestion. The motivations of industry for providing information to practitioners, there are a variety of motivations. I would want the subcommittee to examine all the aspects of that suggestion.
Okay.
I would just say that the Virginia has published two reports of this issue through the National Center for Ethics and health care and national ethics committee.
Okay. Thank you. All right. This was one which began the task force flight. The FDA should continue to work with drug and diagnostic manufacturers to provide adequate labeling information so that conditions can make testing decisions based on farmer economic test results. The labeling should clearly described the test critical validity and provide testing guidelines based on test results. So we got the elegant in here anyway. Yes?
I'm thinking of saying you might not want to box yourself into dozen decisions, just decisions. Sometimes a test will come out that tells you which drug to use.
I see.
You could just to make that progress that they could make decisions based on, right.
As somebody who encounters a lot of confusion among questions on how to use these things, it may sound trivial, but I would put specific guidelines based on test results. In other words physicians may be very unfamiliar with these types of things and will need very specific guidance. And I think we should emphasize that precursor to take out dosing and put in specific?
Well, you may want -- you may want to say specific deadlines or recommendations our web. That is such as dosing or Doug selections parabolic.
Concrete is good. Sure.
To size this even finer I think you might want to say what level you are talking about. The dynastic the party has analytical and clinical validity of the test but the drug label does not as far as I'm aware. So you might want to point toward what level you are talking about three.
So instead of saying both drugs and diagnostic --
I think we discussed this at the task force -- and I don't know why that tends to be made. But the drug labeling provides the dosing kind of information and the diagnostic is the one that should have the performance characteristics.
Because you can do do the typing which will be used for many drugs parabolic.
So we need to disqualify which parts of that are for the diagnostic per.
What we want to do is tie drug and testing and digest and selection?
Diagnostic will have all the analytical characteristics that you are talking about, analytical and clinical validity per.
So everybody is comfortable with the general thrust of this? And Ali, can you report that a little bit?
Sure.
Good. Just so we make that -- and put in those other suggestions at the end that I think Deborah was talking about. Okay. FT eat a should continue their efforts to provide up-to-date real-time transcription drug level / package insert them permission. The project currently under way will be right breeching but to ensure that all sectors of the public access to this information. These agencies to find other ways to reach members of the public to may not have or use access Paribas back again? Good. 7F, the Office of National coordinator for health information technology should promote the incorporation of to pharmacogenomics test confirmation into health records as well as decision support Systems and tools that can notify providers about pharmacogenomics tests and labeling information that could help them make appropriate treatment and testing decisions. [ indiscernible ]. And this may also add to the working with professional organization. All right. Great. Now information for the public, HHS should fund studies of a public awareness of the benefits, risks and limitations of pharmacogenomics Technologies and I think this got to an earlier issue we were talking about, I'd versus what is the reality? Where are we with the technology. So this would be part of that. Everyone is on board. Great. 8B, a gain one plied by the task force, HHS should ensure that educational resources are widely available through federal Web sites and other appropriate media to inform decisions about the use of pharmacogenomics Technologies. This is just public awareness. HHS said the key resources to public consultation activities to gauge the public's receptiveness to and concerns about these technologies and their willingness to participate in clinical research studies involving pharmacogenomics here I to believe resources should include funding. Joe?
A question of clarification, and maybe this is for your group. The issue of literacy in terms of the use of public [ indiscernible ]. I assume that was something that was taken into account in terms of how information was given out because this one is related. I was waiting for this. So it does make a difference if it is understood before you can actually comment.
Right. My understanding is literacy would be involved across the board and in the projects that have been done so far and maybe France's can tell us what the situation is currently whiff there consultation, but I to believe and some of the things that have been done and that was taken into consideration. How to engage people at these meetings that were held to get public in Asia. Is that also --
That was focused primarily on the large-scale study. So of the extent you can map across those reactions into this area, there might be some information to be cleaned there. It is not certainly asking the specific questions.
Is literacy and is to take into consideration when addressing that?
I think it is across the board.
Right. We.
I was wondering if we might consider deleting 88 because I could go out on a limb to say that there is great bustle public awareness. I don't know that HHS each step fund a study about public awareness. My guess is they don't know anything and we should does move right into the summit they don't know what they need to know and that they should provide resources to help educate the public.
He could be -- hang on one second. Let me make sure this is accurate. Public consultation -- I think this was a preliminary pact 8C, but you are right, we could -- if you are doing public consultation you will presumably find out what the public knows and does not know. Joe?
What I would suggest is that under HHS if you look at what is going on in tercet which is focused on access there are projects that are funded past, current in in process or in the beginning related to this issue of public awareness. And that may have a recommendation of using existing mechanisms to enhance the benefit of just the selling that they either don't exist or whatever. So I would focus on that. Again it is using what is already there.
HHS should consent to -- continued to fund studies and then said the ones that you mentioned.
They already have in place commitment to find not studies, but awareness projects.
That is the education piece. That would be right. I'm just wondering if we need three. We have awareness, education and public consultation. Are we saying we can fold 88 and 8C? Is that the general sense here? We could always say awareness in consultation. That would help because we can get rid of one. Pulled 88 and eight c.
As you continue to go through this I try to see if we can find correct text comments. I think it is important to connect with this recommendation if the community is willing to whatever it is that this person was held agenda is that HHS is already drawing. Is that in a different section 22I think given that -- HHS is pretty well telegraphed to us where they are going to spend their money and where the energy is. So I'm thinking if you know there is a train leaving the station with lots of gas you might want to jump on board the train. Anything else is sort of listening as the train goes by. And I think in our discussions with Greg one of the reasons we try to pick some for higher priority rather than lower is because this is where the secretary's purse the last initiative was already --
So that helps me that you have already done -- at least you are putting it there because you know there is a train. What I am saying is you might want to cast 309 out about to Philadelphia.
We know there is a light at the end of the tunnel. This time we are hoping it is a train.
May I follow up on that to end this is an observation, reflecting back on slutty if referring --
I'm sorry?
Eighty, 70 km it is referring to electronic health record as though there universal. And they are not right now.
No, it says until.
So right now for example an HMO's there are electronic health records but they are different from organization to organization and reflecting on what you are saying one of the goals here is getting these in a varsity to do studies across groups. And you might need to be more --
[OVERLAPPING SPEAKERS]
So we to have a thing where we said we need to put this data bases together. They have to talk to one another. In the meantime we have to also continue to not let this fall to the cracks. Joe?
You are up three.
Just the model that you recommend with what our existence and health -- what is part of the recommended where at least the list of things that health plans should be working on? This comes out of in CHP, just a point of reference because this is or what one?
For the recommendation earlier, British is up to --
As additional resources?
Yes. They will be combined.
Great. Thank you. And then there was a comment back here. Is there a microphone back here somewhere.
You get it.
I think it has to come on. Try it.
And I am with the Maryland Office of Minority. I was trying not to [ indiscernible ] I really like 883C as they are. And I think there is a very important distinction between education and awareness been engaging perceptions of what groups about this technology and their desires and needs and how they can be met. So I would try not to combine or merge any of those initiatives that are in the visually distinctive.
Okay. Be.
When the what I would do is build on that and talk about -- maybe I'm going back and forth. I agree that we don't necessarily need studies on awareness, but we definitely do on perceptions and opinions and beliefs. So maybe using that specific language the ease of perceptions rather than awareness would keep a clear and separate.
So you are saying 488 HHS should fund studies of public perception and belief of the benefit address and the limitations of pharmacogenomics technologies. Is that correct?
I am by nature the leader. So I would put the word public perception in there.
Got it.
Right. Well, to gauge the public receptiveness. So we put perceptions. Okay. All right. Thank you. We are talking about just a little but I think we are good. Now we are on time. This is again applied by the task force as something important since it has come up several times. This is about the economic value of pharmacogenomics. HHS should determine the economic value of investments in pharmacogenomics research and development relative to its investments and other health and not held related areas. This assessment should analyze the effect on society as a whole as well as each individual stockholder. This goes back to that discussion we were having before, is it going to effect an individual and how it will it affect society and how will it fit into the zero larger picture. Deborah?
I'm just concerned by the emphasis of value of the research and development as opposed to the use of pharmacogenetics technology.
Okay. I think that could easily go in, research development use.
Well, do you want to assess the economic body of their research and development to the Secretary has already, and said he is investing in this. This is a high priority. Every going back and asking him to assess the first three or whatever research and development recommendations to say the cost effectiveness of those or the value of those or are we talking about really the clinical use of pharmacogenetics which is the part that this year. That is the part we are and, the section of the recommendation we are in. This is the clinical use of this.
My sense was a fight are correctly from our meeting that it was difficult titties these all part. I think he's was supposed to be in here anyway.
Could it not be rewarded that they should determine the economic value of pharmacogenomics brought it to investment?
Right just put investments and pharmacogenomics Delta to investments and not run into the problem.
It seems to me in this area it might be the most appropriate place, but they. Discussion like what some bit -- said the pot up excessively saying that pharmacogenomics does hold the possibility of lowering health care Cosby's and that this should be looked at in some kind of global sense and not a limited sense so we encourage the CBO.
I think this is what we have then that second paragraph.
I'd think it should be.
Said the effect on society as a hobo -- rebook it out bad Paribas bad do we need the CBO example?
That is kind of legislative limited, write to.
It pertains to federal programs. The private sector is not bound by what CBO does. It is also an awkward unrealistic world, CBO, but it is something that we have to face. I think it probably means it's all bull recommendation.
Recommendation or just put it in the report tubes.
Well to start out by putting it in the report, if we are going to weave it into this same recommendation we could maybe direct HHS to the Pearl down a little bit more at the types of data that CBO might be receptive to by examining. Maybe we can get at CBO and directly that way and not have a new recommendation, but amend this current one.
Could you write up a possibility for that? Okay. I think it could fit in a such as looking at this particular issue back.
I'm just trying to make sure that we dealt with stuffed the point Gabriel was trying to make earlier about outcomes research and that we allowed for funding for investigators to look at the economic benefit or all of health care. So have we covered that in the first part of research and development or should be at in this economic value that maybe HHS should find some of this research?
I think what we decided here was to -- we are to sign the economic value of investments in pharmacogenomics in business relative to others. Because it is inclusive. Abandon.
But determining economic value, we will that provide funding for investigators to do outcome research? Is that part of that?
That was added back in the other research and development research recommendations.
Okay. Of back in the first section? Do we have the?
We are looking at that right now.
All right. As we look at that, anything else on nine? Okay. We move to attend. Elsie research, HHS it should fund more research on the social implications of pharmacogenomics. The gaps in current knowledge into questions about whether integration of pharmacogenomics is critical and public health practice will exacerbate health and health care disparity and led access to or decrease the quality of health care and increase medical liability or result in genetic discrimination. Steps should be taken by its it as to identify any problems identified through this research. Does this captured the -- yes?
This is the notion that we are after all and a zero sum at the present time as far as in I it budget. So if you say more research the implication is you will do less research of something else. If that is what you mean then okay. But if what you really mean is that in I aid should continue to encourage research on the ethical legal and social implications of pharmacogenomics that might be a little easier to fold into all of the other needs out there. Because there are plenty of them.
I will also endorse encouraging high quality applications and they will get funded through the private system where we need to start new programs.
I hope we aren't necessarily implying starting a new program. What is the level of funding right now but?
About $20 million per year.
What is the percentage?
About 5%. And there is research going on in other institutes as well not captured and that number. So we could look at this and say should continue to fund.
Sure.
In to ensure or encouraged --
It should encourage funding.
We need to encourage people to apply with high-quality applications. Habits we need to promote, encourage, help, and assist development of.
I'm going to add something to what is being said because the reality is you have more than one mechanism within HHS that is looking at these issues. And it seems to me it is a very straightforward process and is something depth was actually done a lot to long ago which was to do some work and looking at across agencies that already exist to collaborate on high-quality research in this area. And that way -- because you are supporting an effort that exists and are reinforcing an effort that exists and not adding anything to that beyond having this reinforced which is a good thing him. That is already there. And either actually should continue.
Right. Now my question is, does that fall under the next recommendation which is trying to coordinate all the HHS pharmacogenomics activities which I presume would include Elsie I don't want to duplicate recommendations if we don't have to. I presume that includes Chelsea 40 you think we must emphasize -- or what we could do is in the one-bedroom give an example such as the Elsie endeavors that are ongoing at various institutions, something like that.
I think if you are quite to make a recommendation such as this given the current environment now -- given the current environment and what we anticipate to be the environment you must make recommendations that will be looked at as being realistic. And that's all I'm really saying. So I leave it up to you to decide if carried to me the whole idea of coordinating and clever rating is always an effort is a big emphasis and the recommendation will be there for something to be done. That is the direction it would go. I did not want to delete something, but I'm just saying that you're 10 and 11, to me, go together to form something a little stronger and more realistic.
How about for that first bullet is that instead you would something like a and I hate sitting courage high-quality research on the ethical, legal and social effects of pharmacogenomics in collaboration with other agencies.
Great. That's good. But is moving on, I hope, the assertive seen so -- I'm not sure if the agency knows what to do with that. It seems unbelievably fraud and I'm not in favor of leaving things and debate that art -- maybe you can explain. But missed the last conference.
I think the idea here was to say whatever is discovered in the research should be followed up on. You are right. In one sense one can assume that would be done. I think the idea was other than some, state. Back.
The problem may not be what HHS.
But if you have a better way of --
I'd just struggling with it because it seems so fraud.
And I'm just not sure defibrillate.
And the solution may not be something that HHS can't do anything about either Perry Edberg.
I think that is a very important point. One of these tests actually does decrease health care quality. It is not that HHS will have the magic bullet to solve that.
Okay.
All of our recommendations have the implication that they aren't just going to live there but by.
I mean you could probably add that to every recommendation.
Exactly precooks so why do it here? Send the one other thing I ask is in the LC recommendations it seems to me from previous discussion disband that the possibility of litigation blooms very large as a driver for the adoption of pharmacogenomics and the medicine. I'm just wondering if that should summer be explicit in the LC Todd carried there is a unique or a very powerful relationship between the legal issues.
So increasing medical liability is --
Maybe just acknowledging that that is likely to be or seen by many to be a real driver of this adoption prepare.
To am I think there is a section in the report --
We tested and have that and the recommendation because we weren't sure that was anything HHS really had control over.
That's fine.
Debra?
As we moved through the recommendations their is a lot where we are asking NIH to support research on various things. I would recommend that the subcommittee pull all those together and think how much we our recommendation -- recommending NIH to spend more money that doesn't exist and how to prioritize those because that will probably be something that HHS will do and we are not giving any relative priority to these things. And so I think that might be useful, maybe as we go on.
As you go on to the ones we did fight -- the ones with the stars were the ones we are saying were up higher priority.
The point out all the ones for funding and looking at them as a group because we just kind of go through if -- money would be nice and money would be nice and more cities would be nice. But what are our priorities to the Secretary?
Okay. Above.
And so pull this out sending. Did you have a format?
I me pull it out to look at it and see what you are recommending as an overall thing and see if you want to prioritize those in any way. I don't know if that many are incorporated into the recommendations, whether there is a little paragraph that says of all the funding recommendations, car or a party would be this. I don't know how you want to do that. There are a lot of recommendations with for funding and no per positions for the funding.
The appendix at the back where it talks about what all the agencies are doing already by think is a reflection of current funding. So it is not like we are going from zero to something. We are just saying these are the areas that we feel funding should continue to be applied to. I want to go on not sure we are making any recommendations to increase funding, but just to ensure that these five or seven areas are addressed.
But has there been an assessment of whether or not their gaps in the current things being done all relative to our recommendations where there are real gaps that need to be filled as opposed to continued emphasis on things that are ongoing.
I think that is a grid segue into action number 11.
We can do that. Says -- let's just does that then. Draft recommendation 11. An interagency working group should be established to review HHS recommendations and assess whether art and helped implement them and monitor the department's progress and report back. At the request of the agency's the work group could also serve as a forum for discussion of specific activities. [ indiscernible ] and obviously the Secretary knows better than we the resources and can do some of that per testing. When the?
I was just wondering if this was something I would support, but whether you need to be a little more specific their -- in other words what comes to mind when I read this or when areas where the we do need some additional funding, for instance, post market surveillance tests that are entering practice. Out comes Resources has already been mentioned. This might be a place to reach that and also to resolve the identify gaps in knowledge that will come out of these different processes of looking at the evidence. So this might be a place to specify some of those things.
One of the things we wrestled with of course is how specific you get and what did you tell the secretary to do. In a situation like this it is completely legitimate to put in a list of for instance where such as. If you would like to read that list, we would be happy to give those as examples. Again, we don't want to necessarily dictate to the Secretary to do this rather than that. This is our mandate.
On 11 a the test.
May want to consider adding a little bit of language they're saying the department should consider inviting participation of other federal agencies as appropriate. There may be areas where other agencies --
And actually we didn't. Because I think we will get that round a little bit in the meeting. If you think that is a good thing, we're okay. Good. Peeper sort of on the fence about whether that was good or not. Excellent. Okay. We do have time. All right. Yvette have some of the ones that we have applied to come back to the Casbah we were wrestling with how to do them. Do you have that list? Why don't we start with number two. Let's see how fast we can go through this. Which one?
To a.
Which slide, do you know?
Recommendation number two a.
Of Cape Friedberg HHS should provide FDA with the necessary resources to provide guidance the estimates for the code development of the pharmacogenomics which should provide collaboration during the drug and divested industries. That's right. We did it backwards. My fault. PG-13 should amend the humanitarian device exemption regulation so that incentives for the development of orphan drugs are extended to pharmacogenomics tests that are intended to be used in conjunction with the orphan drug. And this is one where the subject bad blood lead to on anticipated and undesirable consequences. And so is there a way that we can rework this ticket to the poll that we are trying to get to? Elizabeth?
I might suggest that you don't recommend amending specific things but looking at ways to encourage pharmacogenomics testing and Carol rather than just saying, take this rule and change it.
So instead of trying to be specific as we try to be, to back off and be a little more generic. FDA should investigate --
Can I make a suggestion? What I was thinking is what we really want to recommend here is that FDA -- at the same incentives apply to or from the trucks as with their big campaign in diagnostic.
Next. HHS should advance the further development of an abandoned trucks by facilitating access to information about such drugs with incentive incurred to development the proprietary data bytes from sickle companies. We get hung up your by some people seeing this as two separate things. One suggestion during the break was that after the term proprietary data, we add end of by pharmaceutical companies making both paragraphs specific to the subset of the whole thing being abandoned trucks and leaving the broader incentives issues which Debra because I think this was her think --
I still want to know who it is being submitted to.
And to get around that we can put sharing, agreed to the sharing of data.
Increased the sharing of proprietary data. Now you would want voluntary sharing, and presuming?
Yes, forced.
Forced sharing, right. Okay. The voluntary sharing of proprietary data. Deborah, to whom?
Well, if it is sharing then it is of submitting.
Okay. That's good. Excellent. Next to kebab off? Forty-three? Okay. This is the comical validity. In certain circumstances private and public health plans should facilitate the ballot by funding the pharmacogenomics technologies on makeup by test developers to collect data on the cultural validity and utility of pharmacogenomics technology. We have dropped coverage with evidence development and initiative may serve as a tip -- Development may serve as an initiative for this product. We will adhere analytic, I believe it. But and then maybe sharpen.
I think one of the problems we had focused around the issue of, to decide clinically utility? And conditioning payment by based on the studies of clinical easily, I think there is a certain contract -- conflict of interest there. Obviously insurers want to see political utility. But I think also it is up to the people practicing medicine tough figure out whether this demonstrates clinical utility. It sounds almost like ultimate arbiter about. Conditioning pen-based on their clinical assessment.
Could we hear from CMSQI am curious to see that you already I assume do this. I'm not sure what this does parabolic this is other than again argue for having the knowledge, the research, the data that tells you whether or not these criteria are being met.
We currently do determine payment based what we perceive as what is considered effective even though physicians as well as others may feel a particular technology might be helpful. Our premise is we take a look at the totality of all the data currently available and make a determination of whether or not it is determined reasonable and necessary for a specific condition. And based upon that -- and I'm sure that some people would say we should not do it this way. We easily dictate what gets paid for and what does not breed so what is currently being requested falls into the realm of what we do.
I'm sorry.
I wonder if it would be useful to remove ourselves, a discussion about the specific perspective because what we are trying to get at and this recommendation is a mechanism to identify if there is a gap. That is what -- and desirable. Could be other entities. We are looking at the broader issue of how we assess enough evidence and when there is not how do we create enough evidence. But the mechanisms for that. This is one element of this profession.
Kevin, I do wonder whether the recommendation and number 6B above actually covers the direction at this discussion is going. That is ensuring that when coverage decisions are made they are made on a transparent basis and the reasons for that -- those coverage decisions are provided back to the various manufacturers. And so it really gets at the same point. This is in some ways without putting the circumstances of conditioning payment on you blowing upon --
Okay.
Does that capture the larger issue that you were discussing ticket.
I think it has captured the issue, but also sought to separate the erection. We are thinking there should be some collaborative efforts between different entities and there could be one perspective. There could be other things to focus on. Everything is on programs and I to give some examples of programs. For example barf effect of the way we look at this is we are talking about creating new mechanisms and it would be on funding one grabs and funding collaborative clobbered of agreements such as this program or the network. But the you see would be a different program where we are [ indiscernible ] be fully published evidence.
So what I think we are doing here is I think what we are saying is that given that public and private purchasers make the coverage decisions or pan decisions based upon demonstration of the day that like, but utility and critical validity and the others that we talked about a breakup we urge that mechanisms occur that have a government working with various entities to facilitate that knowledge being developed, that information be developed so that these functions can be achieved. So without trying to be read it, when you go back in the subcommittee -- whips up will be generally testing as it reminds you all and the beginning of a we are charged to do with this discussion is whether you will let this go forward to public comment which I won't bust 42. I would say what he will find is several recommendations that all speak to the same idea of facilitating the collection of knowledge, the SEC, studies and so forth by a variety of entities. And this just becomes one of those. The way you phrase it is in recognition of the fact that people are going to need this data to make decisions. Let's work together to try to get it.
That sounds good. You want to read that one up?
Quick comment.
I would like to reiterate a point.
It is true that CMS has the development for those promising technology. Now promising to policy is one where there is insufficient evidence selling it would be effective and that they be due to the fact that there are only a limited number of studies out there, but the studies out there to look promising. Unfortunately they are not sufficient for us to say it is reasonably necessary. So we do have a number of projects that do fall under coverage development. But they must be promising. But as I say unfortunately they don't have enough justification to show that they are effective. Whether or not any of the former coach -- pharmacogenomics test would fall under this category is something that will have to be determined by CMS or some other pair if they chose to go to that route.
Does that fall under your general conceptualization?
[ indiscernible ]
I would like to see that. Next let's let read -- that's it? Excellent. This is great. What I would like to do now is to turn our attention to where we want to go. Obviously we have done wonderful input today on a lot of these. Very helpful. Very insightful and brief which is wonderful. Now the question is what we do to revise the report and recommendations based upon what we have received and how we look forward to give a am not sure exactly of the range of options here, but one thing I can suggest is the possibility that we take the various recommendations we have received on the recommendations and work the San. We could e-mail people those recommendations in their revised form. If we don't hear back from you to veto the process then we will go forward with the recommendations as they are phrased. If you come back to us with a veto then we will try to engage that process and eventually we will speed these banks along so that we can move forward with the lemon group during the stakeholder and expert interviews very much on schedule. How does that sound to people?
I have just a minor editorial thing that doesn't merit full group discussion taking up time. Is there a process --
You been in the report?
Should we just tear this out and hand them?
Sure.
That will be bought waste people's time.
There is information that you think would be good to get in the report. Let us know. We have already indicated some things. We can rework the report absolutely. Does that process sound reasonable to everyone? If so I think that is what people do. We will work as quickly as we can. I began to see -- okay. All right. We will be very, -- I will be very unpopular, but people try to get those back to you. We will tell you how much time we need to have your responses. And then we can pretty much of have and will try to do that so the group can get going on the stickle the interviews and we can seek public comment. If that is okay that is what people try to do. And I believe that pretty much wrapped up what we were told to do today.
Isn't he could?
Yes.
Masterful, masterful. It is time for lunch. Now I need support. I ask them and hope that the box want deal that circulated. I hope that we did not stop the process. The other thing is that what time do we come back?
And the people who are not in the box lunch group know that there is a terrific restaurant right next door.
There is the cafeteria.
And there is something cord. You can turn left or right. And food will be there waiting. And we come back at exactly 145. So we will see you that 1:45 p.m. at the box what is where? Appear in this room.
[Lunch until 1:45 p.m.]Please stand by for realtime captioned text.
[CAPTIONER IS TRYING TO DIAL INTO AUDIO LINE. PHONE LINE RINGING BUSY].
Et you all very much. We will now turn our attention to the issue of gene patents and licensing practices. We will now turn our attention to the issue of gene patents and licensing practices, Joseph. In March of 2004, the committee identified this is a high priority issue because we have some concerns about adverse effects to genetic tests and services. At that time, however, the National Academy of Science had just begun a study on gene patents for the NIH. We decided to postpone a decision on whether to take our own in-depth study until the academy's work was complete. It was called reaping the benefits of to Numic and inestimable research was published in the fall of 2005. In March of this year, Deborah and Emily, the team we have appointed to reduce that study gave its recommendation sufficiently addressed intellectual property concerns in the research, but did not fully examined the impact of patents and licensing practices on patient access. Very different. In June we had more information on this topic before reaching a conclusion that we needed to embark on an in-depth study on gene patents and licensing practices on patients. We have a scope for the study, it had several investigational- investigational-and tapped, drafted, hijacked Jim Evans to serve as the chair. On do work of the task force and the detailed work plan. We have two hours for this session. For test is to discuss the task force recommended approach and give them and the committee some clear direction and marching orders so that he can lead the group forward. As return it to you, I want you to be real clear about what it is you want from us. We can give you marching orders to leap forward, but we can give you marching orders that lead you up Mount Everest, and you will not get there. Have specific do you want from us? What do you want to achieve by the end of this two hour session session?
Well, thanks. This is an extraordinarily complex topic, and it is a topic that I think more so than any other item that the committee is taking on, with the exception of genetic discrimination, it rarely elicits passions in people. What we are after today is really three things. We would like people to weigh in on the scope, which we have spent a lot of time trying to determine what the scope of our investigation is going to be. We would like in a point on the study questions that we have defined, and we would like input from everyone on how we have proposed to go about this. None of these things are written in stone. This is an opportunity, early on on, for people to really change what we are doing. I certainly do not hold myself up as an expert on the gene patenting. We need lots of input from people. This is the membership of the task force as it currently exists. Let me introduce a couple of people. One is not a hero and that is [indiscernible]. She has a role in the committee, but in addition, Brian Stanton who is sitting to my right is with the NIH Office of Technology transfer. Raise your hand. I think that most people know the other members of the committee. The activities today, to summarize the very briefly, in March of 04, it was really defined as one of the priority issues. In October of 05, Denver letter contained a small group to look at these issues, and then in March, 06, many of you remember that at that point, we were able to evaluate the National Academy report. It was bought before that report came out, that perhaps it would really have done our work for us us. That really is not the case. The National Academy report was quite heavy and looking at the research implications of the gene patents. They were extraordinarily light on the issue that we feel is most important in the secretary's committee, and that is, ultimately, patient access. Because we see a patient access as our salient interest, we felt more work needed to be done, and there was a role for this committee to take up issues of patenting that were not addressed by the National Academy report. In June 06, we decided to move ahead with an in-depth study, discuss the scope and work plan and established the task force that is reporting to you now. The first task force meeting was was, as you can see, quite recently. We have come up with a scope and study proposals. The goal of today's session is to try to reach consensus on that scup, on the study questions and on the way forward forward. Now, I am going to go through a few things, and then we will have a chance to talk about these in detail. People will want to wait and on this. I will hold off for a moment on the proposed scope statement. We may want to modify it. One of our goals is to be very balanced. Everyone recognizes that gene patenting provides both benefits to this whole endeavor of genetic and medicine, but it also has certain downside. Our ultimate goal is to guide things so that we can affect that balance to the favorable extent on getting-you can follow the scope in the folders that you have. Okay. So, we also wanted to define for you some of our terminology. I have referred a couple of times to a patient access. You will see the term classical access used throughout our discussions. It does so that everyone knows, what we mean by that, patient access is pretty self explanatory. We want patients to have full access to emerging technologies and things that will benefit them. When we said clinical access, we are also trying to capture the idea of the developments of tests and the integration of the genetic testing, for example, into patient care. This also assumes issues that relate to reimbursement and cost cost. And other words, patient access hinges on a lot of upstream types of things. We want to try to capture that flavor. I would also keep in mind as we go through this that we are often times looking at proxy's for patient access. When we look at the affects of patents, for example, we heard a lot of information from Deborah, from Mildred, about, for example example, availability of clinical laboratories to roll out new tests and offered genetic testing. On or charge at a committee is not really to look out for the welfare of molecular biology- biology-court charge is to look out for patient access. That may be the best proxy we can get for those things. Keep in mind the fact that we are oftentimes going to look at perhaps imperfect proxies' to judge patient access. The study questions are the following. Again, we will have a chance, momentarily, to go through these in detail. I will give you the 30,000-foot overview. What are the overall effects of testing on clinical access? What are the quantitative and qualitative data for the positive and negative effects of gene patenting and licensing practices. That is an extremely important part of this. If there are problems, where do those problems of? Are they in the development stage? Are they in the reimbursement state? Are they in the integration stage? I think we need to think seriously about current licensing practices for two reasons. They are obviously very important in patient access, in the ability to grow up predictive tests, etc.. It also may represent an area where we can have some influence influence, because, of course, patents, ultimately patent policy is based in the U.S. Constitution, we are not going to recommend amending the Constitution. On the other hand, of licensing is something that may prove a more attractive to all if we identify problems. We want to think about the solutions. The effects on cost is extremely important. That it's directly to patient access when tests are prohibitively expensive. That is a problem. What we would like to assets is, are there of data that address the affect of patents on the ultimate cost of genetic tests, for example. Of their economic data that analyzed the contributions of patents to these things? We would like to look at the effects on the development tests tests. Do patents and licensing practices as currently seen create barriers to the development and in addition of clinical tests? This is 1I think is up for some discussion as to whether it even belongs on our plate in this task force, and that is the issue of quality of testing. It has been argued that when there is a patent on a particular test, it hampers the ability to engage in independent verification of test results and has a deleterious impact on the quality of testing. That may not fall within our purview, but it is something to discuss. We want to, quickly, go through the study approaches. What are the types of things that can be employed to assess the direct effect of gene patents and licensing practices on patient access? If those studies do not exist, what are they. What alternative models that aren't practical? We are not interested in going off on a tangent as trying to climb Mount Everest. We are interested in practical models that might relieve any problems that are found without harming the good things, be beneficial affect of current patent and licensing practices. With that preamble, we can now get to the discussion. The first thing to discuss, before we get to study questions is the scope. We came up with the scope. I will read it to you. I have already heard some very legitimate criticisms that we might be able to improve it by incorporating. While recognizing the benefits and importance of packaging and innovation and--SACGHS will explore whether current gene patenting and licensing practices are having adverse affects aand ultimately on the public's health. I will throw it open and let people make comments about that scope.
Thank you everybody. I am glad to have the opportunity. When the emails were going back and forth about how to define a fairly substantial problem, my concern was that the scope of the study question can influence the way people approach, the pursuit of the answer to the questions. My thought was, my preference would be that we not use the word adverse right in the sculping document of the question. Rather, we leave it a little more open and it and use the study questions themselves. Rather than say adverse affects, that the working group to explore the affect or what affects gene patenting and licensing practices are having on patients' access in genetic tests and technology, thereby leaving it open to looking at positive and negative. The reason I suggest that is as we explore this, what we are going to find from my own experience are two things. One on the gene patenting side, sometimes Patton's get in the way, and sometimes the quality of the patent in the White. It is not necessarily the testing. When it comes to licensing practices, and similarly, that is something we have an hour or controls. What the affect is, where it is good and where it is bad. How we can-what factors pushed the balance in one direction or the other then we can look at operative outcomes do see where the system needs to be tweaked without changing the constitution, which I don't know necessarily need to pull off the table, but it is probably more cumbersome.
Would probably need three or.
That you very much.
So, we could, for example, and then is to say that SACGHS will explore the positive and negative balance, which exist in the current key gene patenting and licensing practices. With an eye towards identifying how best to enhance that balance balance, something along those lines. Other people, suggestions, comments?
If you want to keep it from neutral, you can keep the first part of it and say that SACGHS will explore whether current gene patenting and licensing practices are having an affect on the patient access.
I am sure they are having an affect, right? I guess what we would want to do is follow it up and say, are there things that we can suggest suggest, practical issues that we can suggest, that would enhance that balance or something, right? I think-other suggestions? I think if we start with while recognizing, starting with the wild means there is something coming, something negative coming. Would people be comfortable with the statement that says that really starts with that second clause that will explore current dean practices in order to determine the balance, the positive and negative effect on patient access with an eye toward suggestions that could further enhance, or enhance the positive side of that balance? Brian, do you --
That would address my issue.
Update. Yes?
Your positive balance is the public help, is that right?
Yes. We should continue to emphasize that our role, that we see our mandates as ultimately coming down to patient access or the public's help. You may notice the '. We are talking about the public's health. It may sound nit picky, but it is an important distinction. Okay. Moving along.
Just a question. In the current freezing here, if you are focusing on patient access, and ultimately on public health, are we losing the affect on the patient outcomes?
No. I would think that when you are talking about the public's health, that inherent in that would be out comes, the kind of outcomes we desire. I maintain that that is inherent to any idea of patients health.
Just to make my comment more clear, the reason I bring this up is if we are assuming that because of that cannot be shared, and knowing that is useful to determine how good the test is. And apparently, there would be about the outcomes, not just the test. I do not know if that counts it has been in the scope here.
In regard to the-I do not is in our purview to really decide on a case by case basis what tests are of benefit to people, et cetera. I think it is fair for us to assume going into this that there will be technology that is subject to patent law and licensing type practices that does, indeed, have a beneficial affect on the outcomes, et cetera. I think we need-my own bias is that we need to assume that we are talking about test that are found to be legitimate. That is not our charge here to look at how you determine what tests have a good outcome, etc.. We are going to assume for purposes of this that those tests exist and they are subject to patent law. Am I answer your question?
I and is trying to verify the scope of all.
That actually gets to a discussion in a minute about ensuring the quality of tests, which I am not sure be bonds in our scope. When we get to that maybe we should discuss it. I can go back to the study questions one by one. I should probably go ahead and do that. To remind you, the first and question is the overall effects then the effect on development of a test, the location of possible problems, impact of licensee in and then further study and alternative models. I think what I am going to do now is just step back here to oppose any questions. And we can take those one by one.
Before we get into the detailed questions, I think there is a good job as you will see in a moment, as I have looked internationally at the question of access in genetic testing, the context of the national system in which the question is being asked sometimes can bring different answers. I have a question as to whether or not we need to put it in the beginning of the document as part of the purpose or whether we need to contextualize each individual question to stay within a given national system. There are different constraints within different systems. The thought to put on the table for this group, whether or not we want to limit it to the U.S. system and its reimbursement or where--what we want to globalize it.
I think the assumption of the task force has been we are dealing with U.S. practices on the effect within our system. That is not to say that we, as you'll see in a minute, that we can get very useful information by talking to people from other countries and different systems. I think it is implicit in all of this that we are talking about- about-We know the Secretary works for, right? It is not another country, right right? The first one is really the overarching issue of the overall effects of packaging and clinical access. What is the quantitative and qualitative evidence on clinical access. That is the lead off study questioned at in a way summarizes the broadest terms what we are looking at. Comments? Suggestions?
Are you looking at economic quantitative and qualitative?
Not necessarily.
You may want to spell that out a little more and define what specific but benefit you're looking at.
What we are getting at is, are their data out there that directly or indirectly SS the impact of current practices on patient access? It could be economic, but it could be the inability, for example, Ford Laboratories to develop tests, etc.. On their data? What are the data out there for determining the current impact of patent and licensing practice practice? Do you find it too broad?
I just want to know what you mean by impact?
Well, on things that are enhancing or limiting patient access. And world met goal is to look at patient access. When we are talking about having an impact on patient access, our patients able to reap the benefits of these technologies.
Will you be looking at the benefits of having a patent for companies as far as protecting the acceleration of that technology as well as --
That could certainly tight into are there any data that show that because patents were enclave in place, because licensing practices, this test was able to be accelerated and get out there, whereas it wouldn't have happened without current patent practices. But would fall into that.
I think it is-it's fairly broad. I think we need to drill and, such as. If you'd say it-1st of all, I'd think you should split between the two. What are the positive issues and what are the negative issues, because it is so broad. It you start thinking-defining in clear terms at the beginning of the report what you mean by access and the question that Mike is raising on the premise of the entire activity. You have to be really, really clear on what access means. This question then becomes, what would be the level of evidence that you would require for being significant? Would it be-I think what you are asking is things like, would it be the numbers of people, would it be the price of the drug, would it be the price of the licensing? There are certain elements, I think that is what people are asking, giving examples of what qualitative and quantitative would mean.
Okay. We could have some some point there. We can split it into positive and negative and have examples of things that we are talking about on impact of patient access. Okay. So, the location of possible problems in the healthcare system are barriers, if those exist, for example in the development of tests and the reimbursement of testing. Trying to, in this sense, figure out where those problems exist, if they are found. In the first study question, what are the data? Where in the healthcare system to those exist? Some of those things have been addressed by the National Academy report, but again, that is focused primarily on extremely upstream types of stuff. We want to keep this boat is on patient access.
I think here, what would be useful would be if this question could be accompanied by the chain of evidence, let's start here. Not saying by laying it out, this is the menu of possibilities. It would advance everyone if you could say A then to the debt into C.
That is a great idea. We could show the flow from initialed basic research which might not fall within our purview. We can mention that all the way to the patient being able to get reimbursed for this test.
Just wondering about the chain of evidence. We usually also have some in the chain of evidence. Are we thinking that in this case, the comparison would be to non patent diagnosticians to patent [indiscernible].
You get to one of the difficult problems in here, which is, what is your control? What is your control group? If you are going to say what is the affect on patient access, which you would like to do is compare that with no patent. That is very difficult to do with gene patents now, because the way to do then, the one where you can envision is seen happen is what happens when things go off patent. There has not been enough time for that. That raises one of the things that we struggle with as we talk about it, which is, how do you want to tabes defects? I do not have an easy answer. I do not think there is an easy answer. There might be in 20 years when things have gone off patent. One example would be, perhaps in looking at other systems were the pectins is not as restrictive. Your question is a really good one. It is one we do not have a good answer for. That is one of the things we struggle but as we get to our methods for trying to carry this out.
I think you can go beyond patent or non patented. There are a variety of licensing procedures that are used. There is broad licensing at a reasonable royalty rate versus exclusive licensing pursing proprietary testing per so you can look at the relative effects of those, although they are comparing want us to another test. You are also comparing apples and oranges in that the tests are not the same across the different licensing practices, but you can look at the different licensing practices.
What I also suggest is this question and 34, the development and survival. This question is focusing on the health care system and to refine that we might say, where within the healthcare system and what elements of the IP Spectrum are affecting the provision of clinical --
--
In other words, in the research phase once you get the patent delivered and you're going to deliver the test there will be licensing issues. You have as components be aware in the IP Spectrum all the way down to licensing and delivery or not. There is the issue as well and the different components of the healthcare system. That looks at which problem or solution may be present and which component of the delivery system and three questions down we ask now that we have identified which component of the IP system affects which part of the healthcare system, I guess, then we can go ahead and find out okay, now that we note the convergence between what part of IP and delivery stream, we ask if there is a solution. Planning the concerns of those two components may clarify this question and bring it to the third one forward.
Okay. Moving on. I think that something that has been mentioned a lot here is that we are emphasizing on the impact of licensing practices. Or licensing practices affecting the ability to develop accessible genetic technologies. What role does technology what are the downstream of facts of licensing practices on clinical access? I think that licensing issues probably deserved a lot of attention, because of the possibility of the possibility of that these are things that can be more readily changed been the aspects of patents in practice. Comments?
This is very much like the earlier one of what are the data sets? How would you know, what would be your-what would be the criteria to determine that licensing practices had an effect? How would you segregate out that piece of the puzzle? Is it possible, I have no idea, I am just signed to play around with ideas, would it be that the relative proportion of expense of a hot ultimate test / the licensing fee was less than 30%?
Or, do you factor in the reimbursements, the percentage of plans that reimbursed for this test or that is, what the cost is, etc.?
People outside of that is again playing this out of 100 manufacturers interviewed or developers interviewed, 3% said without a licensing fee, I would not do this, it is not worth my time, therefore, there would be the test. This is where I think the real work-this is pretty exciting. It would be to try to figure those things out. What would be the Mets'? I'd like that.
We get to that in a moment on ways to address that. The affect on cost is obviously integrally related to patient access. When some of these tests are extraordinarily expensive, and there is all kind of debate about just how expensive they should be. What we would like to try to figure out is the quantitative, and I suspect we will also have to deal with qualitative data on licensing practices, Gene patents, ultimately on the pricing of genetic tests and some of these pieces of data and they come from the economic world that to the ultimate cost and ultimately ultimately, how does that affect patient access? That is what we are most interested in. Comments? Questions? Okay. The affect on development of tests.
Sorry. The way this is worded it is a yes / no question. Maybe-I don't mean toward Smith, but we should say what is the quantitative and qualitative- qualitative-does any qualitative or quantitative evidence indicate adverse --
Address the magnitude.
Right.
I think we probably all agree --
I do not know, Brian, if you want adverse out of their.
I was probably going to do that offline. The question of going to prejudice the answer. Let's make it neutral. I would like to foster, when I had a conversation with the patent office last week, if we, when I was there, would have done a better job, then maybe we would not have had some of the issues. Where the trigger point arc card hard to define and a neutral matter.
For all should be to look at these things and let the chips fall. There is no reason we need to go into it with preconceptions just because there are popular preconceptions.
James.
Yes.
The only fair way to look at this is if you look at the genetic tests, the world of genetic testing, and you look at the value that has been created by the ability of a company to patent and get protection and ordered to find or afford the tests, and compare that and identify specific examples of how limited licensing practice of having a patent has specifically kept a product or a test from getting to the clinic-.
Will once it is in the clinic is kept from patients being able to get access to it, which exists now.
I am talking about in clinical practice.
So am I.. There are lots of tennis out there that many of our patients cannot get, because, for example example, they are so expensive. We have to look at the whole gamut. Does that make sense?
Yes. That gets back to insurance issues as well.
Right.
I think it is critical to be fair.
I could not agree more. Believe me, I understand there is a certain default kind of feeling among many people that gene patents are bad. We do not want to, I think, come across the gene patents are bad. We need to take a very balanced approach. You are right, there are incentives that are very good in bringing this to fruition that might never have come to fruition. We also, we cannot shy away from when they do limit patient access. At is one of the reasons we are here to look at it.
I would agree with keeping its balance and neutral but not try to neutralize language too much. We are representing the Haitian side of it. We are not looking at the other bodies looking for the positive effects, we do not need to do that here. My recommendation would to keep the words positive and negative in there. It is and will.
I think that is good to constantly keep in mind that we are looking at both the positive and negative effects. We do not want to take this on the ankle that this is rosy or evil about patents and licensing practices. Fair enough? Okay. So, the effect of development on tests. This is one of those upstream issues. Perhaps, we can-we should say that dependence and or licensing practices enhance or create barriers to the development and barriers of clinical tests. One could argue that they can enhance as well. We could probably be more balanced in this.
You do not want something that is going to give you a yes / no answer, write? You want to somehow get into the details of --
How do routine patents, what.
A [indiscernible]. In what ways, or how do gene patents-okay. This one, I am not sure if this belongs within our purview. The concern, let me read it, is the quality of genetic testing having an adverse affect on the independent verification of test results? The genesis of this concern rally stemmed from a recommendation 13 of the National Academy report, which said, okay, there could rise situations in which only a single laboratory is doing a test, and therefore there would not be any quality control. I am not sure that this falls within our purview. Perhaps these falls butter in CMF. Telework concern is with patient access. I am kind of presuming the-we are dealing-that we are concerned with the test that make a difference, the quality test. It is not within our to decide on quality. What do people think?
Did, can I ask a clarifying question? I know that this question came out of the second part of that question, that is no independent the addition of the us. In net for statement about quality, is the following hypothesis included or not in quality. That is, in one laboratory, there are seven different genetic changes that are responsible for certain conditions. One Lab owns three and one owns four. They have decided not to pursue them, but is not giving anyone else access to be able to use those. I would say that is not a high-quality test if you can only look at four of seven. Does that included or excluded from the as?
That is a very good point. I have not thought about that particular spin on the idea of quality. In any way, that is assumed under patient access to a genetic testing if people, because of a patent are not able to get three of the that can be responsible for it, that would be an access issue and not so much a quality issue. I understand how it can be spun that way.
I hear what you are questioning whether this fits in here, but I do not know how you can separate the idea patient access to test and they being meaningful. There is a serious issue here and the National Academy, while highlighting it and making suggestions, it is not clear to me that anything will happen with that. To leave this untouched would be unfortunate. There is a real issue when you have the tests that have been exclusively licensed to a single provided that there is no natural way and objective outside evaluation assess the quality outside of what will get done by other government oversight which is now a bit unclear. It belongs on your list.
I am certainly okay with keeping it on there. You both have good points.
If you look at the broader charge to SACGHS, the quality of genetic tests is what got this whole committee started to begin with.
[SPEAKER/AUDIO NOT CLEAR]
Right. I think this is smack in the middle of what SACGHS is supposed to be doing.
Great.
Hi, Francis, how are you doing. I agree 100% that quality is an issue. The only reason I would suggest that it is not for this predictor task force is the following reason. I think Jim said it very well, if we look at the broader question of where is the conversions of intellectual Property and licensing on the ability to have the test in the first place, then the derivative question is how does that affect the quality of that product? And we cannot get to the first base of I cannot get the teams together to do the test or looking at it prostate test where they needed seventeens to do it and they only had access to six. There is a qualitative questions which, which is probably seductive from a medical perspective, nothing is an objective, but there is a question of how does IP get there, and once we are there if we have all the genes, do you need them or not? I wonder if that is a separate question for this committee but not for the study. The quality of the test itself isn't the charge here although it is an important question. Can you get to the genes in the first place so that doctors can determine how to put the test together?
It goes beyond that. Sometimes you cannot do the test test. You end up with a sole provider of a test, because they are exclusively enforcing their right to do that test. They decide the national standard of how that test will be done, the testing method, and so, you are not getting be brought medical community doing the test and developing a consensus on standards. It affects the quality of testing beyond whether you can get the license to use all the genes necessary for a particular test. This is a single gene tests that you cannot do.
I would submit this as one that could fall through the cracks. I you're you are saying by an as this thing on the periphery of the focus of this study. At the same time, this would be on the periphery they would probably not pay that much attention. [Overlapping speakers]
If it is going to be captured captured, why not capture it now now.
I tend to agree with that. I am going to argue what I said against before. Just to show how open-minded I am. As someone who orders genetic tests all the time, I do lament the fact that there are certain tests that I can only get from a particular laboratory that I have been far from impressed by. That is an impact on patient access to good genetic testing. I think there is a reasonable consensus.
not only access to the test but something that was mentioned earlier, if you to offer we can improve the sensitivity that we can provide, not only for the laboratories, but also for the manufacturers also.
I think the issue is very persuasive to me that this could fall through the cracks in another committed since it can be seen as peripheral to others. Okay. So, study approaches. What quantitative and qualitative approaches can be employed? On one hand we are asking, what are the data out there for the affective patents and licensing practices on these issues. Here, we are asking what are the approaches that could be employed to assess the direct on patient access and genetic technologies if those do not currently exist in? We are going to talk a little more about this. It is a bit of a departure from what SACGHS has done in the past past. I would suggest that perhaps, unless they are driving questions, we hope is for a second discussion until we get to that aspect of our study plan plan.
Could we to say what additional quantitative and qualitative-should yes, that would make it more clear.
Not identified by the above.
That is right. That sounds good. Okay. Of their feasible alternative models and innovations the could be applied to the packaging system to [indiscernible]? I think feasible is an extraordinarily important part of this statement. This is such a thorny issue. There are so many stakeholders and a lot of constraints on what we can do and what the secretary can do, that we have to focus-it just on this one, Jim, I understand the reasons behind focusing on the U.S. situation. And this one might we want to look more broadly to see if there is something out there globally that might be useful?
Yes. We could add something to the effect that we would like to cast a wide net. We are continuing to keep it feasible but look at a wider --
When you are trying to do the study, will you provide a list of the constraints-and there are going to be things that we cannot get by. You have to build your story based with in that context.
I think that what we envision envision, as you will see in a minute here, we would like to convene a roundtable in order to start to approach some of these models and will have to take into account, giving into feasible, what those constraints are.
For instance, you can probably do a lot, SACGHS, can do a lot into NIH. It is going to be more difficult for industry. That will require was. Also, it kind of lies in the face of administration policies to promote the industry.
Right. Also, we certainly have, correct me if I am wrong, the committee has not been shy, necessarily, where we see a need about encouraging the Secretary to promote certain types of legislation with Congress. Look at the genetic dimmest discrimination issues. We know the Secretary cannot mandate genetic discrimination. That has to be done through Congress. It is not unreasonable if we identified alternative models, even if they are not in the direct abilities of the Secretary, that the secretary, he / C could have an internal.
While we have taken out adverse and all the other things and make it positive and negative, can we do the same thing to this, that it be applied to mitigate any negative effects while preserving its inherent incentives?
It sounds good to me.
Because --
We want balance the whole way way.
Right.
Okay. Let me go through here. We have gone through the study question topics. We come now to the proposed study plan, which has four components. We feel that an in-depth study is in order to figure out what are the data that are out there now that address these questions questions, that address the issues that ultimately get to patient access in the current environment. But on a positive and negative standpoint. We feel that a public consultation process is especially important with this topic. We can talk about that in just a minute as we go through each of these things. Again, I am continually amazed at how both and what little knowledge is out there among the fairly knowledgeable members of the public about gene patents and the fact that they exist. I think that is in a combination with the fact that people get very strong feelings from people when they learn about these things. It is very important that we have an extremely open process that elicits public perceptioperceptio ns, feelings and, ultimately, the public goals. That is an important part of this and can be compared with the process we pursued when discussing genetic discrimination. That was something the public had strong feelings about and a strong stake in.
Yes. However, I want to be careful in my mind, I remain open. If you say, and you did not mean this, but I am being deliberately provocative, their public, do you realize that people make money on licensing these things, and its elevator cost of access to these things. Looking for someone to kill. On the other hand, phrased a different way, it is going to be extremely important that the public consultation process the engaged ones there is a clear set of facts, information, reasonable mythological data derived fax, and sort of saying, therefore, then there are some choices and issues that need to be addressed. The work is really on the front and to define if there is a problem, the magnitude of the problem, the elements of the problem before the public gets brought into the.
Yes?
Part of what is public consultation process is to define it is is there a patient access problem that is happening individual patient by individual patient that we do not know about and would never find out about by any studies that have done to update. Kind of like the genetic discrimination public comment. Is anyone --
Do we now know enough to know of that how much of that is now because of licensing independent of any other factor? That is the question I am asking asking?
I do not think we know. For example, the families brought a lawsuit against the holder of the patent patent. There is an asset on an individual pet level, at least for that one, there may be others that we are not aware of where to these organizations have been brought a lawsuit. That is the qualitative evidence that we may be able to get to that others cannot, if we do ask this question of the public.
So, the way in which-I am following with you. Excuse me, and Jim. I am breaking every rule that I have established here. It would then, I think Deborah you mean, that you have enough ability to raise the question to the public that would say, do you believe that you have been denied access because of patenting or licensing issues, and here are criteria that you can use to determine whether it was simply not affordability, but it was patented or licensing licensing, given that so much of it is below the iceberg, below the water? Or maybe perceive, the patient may not perceive it, they may interpret it as it is probably in there, I have been denied because things cost a lot.
The same is true for genetic discrimination. Their experiences were not necessarily what happened, and yet it seemed like an important issue.
Just one thing on this. It might be critical to decide in this process is Reid is a question. Who are experts and who is the public in the sense that I would consider some of the people that are thinking about our physician as a group people to be an expert in some sense. Bay are expert in exactly that type of information. Are there people are being bought because of patent them license? Ed there are people engaged in lawsuits or something like that? If those are the people we are thinking about, absolutely, we have got to hear from them before we can establish the language we are going to use to do a more broad public.
When we did the genetic nondiscrimination public comment comment, we heard from patients, genetic ulcers, physicians, it was at a number of levels. You could do that process similarly with the.
I will back off with this. the language to describe whether or not you feel you have been harmed in this regard.
[SPEAKER/AUDIO NOT CLEAR] [Overlapping speakers] That is where the art of it is is in the description.
I am going back to the earlier ones where I was happy to see the emphasis on qualitative and quantitative research. I would think he would address issues just like as. I am wondering, is it really consultation or records qualitative research trying to get at these questions. Are you trying to consult with individuals or gathered the data in the research phase? I could see it in my mind as the research phase as a separate consultations waves.
I think that is a good point. One of the things that drove us to consider this whole issue of a more informal and a more elaborate public consultation as to what happens every time the report goes out, which is the public sees it after it has been drafted, I think one of the things that drove that is how difficult we all realize the task is of figuring out the impact of the gene patents, etc. on patient access. We are not, probably, going to have pristine quantitative research that demonstrates in a controlled fashion the effects. We are going to have to rely, to some extent, on qualitative research, qualitative experiences and comments of various stakeholders.
Going back to also in-depth research studies and the public consultation process, maybe we need to propose the to research studies being performed in a systematic way. When you go back to in-depth research studies you review the constraints because of current patent practices. I think you will find a very different environment five years ago versus what you see today. You will see, for example, five years ago I would have said no. If you have made today, I would say in the last four months, I was not able to bring six different us into of laboratory. It is a different environment. In also charge some of these --
Exactly. That is something we get to in a minute.
What you have then, if you go back to the points that I heard from all three is the public consultation process includes concentrating advocacy organizations, the public at large and people like you, so that we get all three of these constituencies coming forward.
Maybe it could be called a public data gathering process or information gathering process.
Sure.
Because we are really using the literature, number one is getting whatever information we can from published studies and literature out there, second is experiences of different groups who might have been affected to indicate whether there is a problem. The International perspective, these are just three waves. I did not mean to jump ahead. Those three would lead into the development of a comprehensive report to the Secretary where we could provide what information we found, but we could also say whether the problems are and whether studies are needed, whether certain things need to be addressed by policies, these kinds of things.
Going ahead to the International perspective, I think that as we deliberate, as we put this together, we all realize that perhaps we could learn something from the models in the other countries. Obviously, we are going to have to be very careful to keep such comparisons to remain and feasible. We exist in very different political physician government will situations. We are not going to be able to transplant practices from another country into the you as. However, we might be able to learn something that has been implemented from other countries countries. It is crazy to ignore the world experience with this. The development of a comprehensive report. Going in-depth in these things, the in-depth research, we want to refine the study topics for review. In the relatively near future start a contract for that literature review. We will commission not and then see what types of gaps, by the spring of 07 exist. At that point, then, what we have envisioned is a roundtable that would identify what gaps exist that are quite amenable to further and data in a fairly near term type up perspective. What we had envisioned, if there are discrete types of questions that do not have an answer, simply because the data is not there, the study has not been done and it is relatively straightforward, at that point we can commission limited studies in order to address those gaps. If we find, on review of the literature that all the questions are answered and do not need that, the questions are not answered but there is nothing feasible, then we would not go on to this. If there are practical types of things that could be commissioned that would help fill those gaps, we would have an opportunity to do that through this committee.
What sort of things do you envision finding in that literature?? It seems that people are not going to talk a lot in their literature about the failures?
In the literature now, if you look at Minter it Mildred and Debra Leonard's work, looking at the ability to certain clinical tests, those data already a little old. It could be, for example what Andrea said a minute ago is that the landscape is changing quickly, would it be wise to commission an update to look at how is the field changing, how is the landscape changing? I think that most of these things will be proxy's. Most of these will be proxy's for patient access that can on the ability to develop a test and offer a test, cost issues. I could imagine that there may well be a body of Economic Literature that has used modeling to look at aspects of the Altman affects of access that gene patents and licensing of that.
What about a parallel study that potentially, like a questionnaire or circuit that could be administered through the College of American pathologists that would pass the people who are actually using the tests would have a pretty good handle on, like you said Andrea, what the limits are and what they have not been able to --
What. Those things are too old or not out there. That is the kind of thing that we need to commission that would be affordable. We can not commissioned-it has to be affordable and durable and a relatively short period of time.
Jim, as I listen, I am finding one gap here. That is the benchmark against which we are going to measure our results. When I hear that someone cannot deliver a genetic test, because- because-my ears prick up. What is the standard of care? Where are we going to gathered the data or gather the opinion as to what should be the standard of care so that we have a benchmark which to measure what should be provided. What is the ultimate goal and given the economics, or tangible situation? If we do not have that somewhere somewhere, maybe when we consult with the experts, we have some input summer about what an abrupt is standard is?
An inappropriate level of access as?
Yes.
Are you talking about access or whether that test is medically necessary?
For patient Management?
Well, I guess I will have to leave it to the other side of the table to figure out the answer to that. In the abstract, the patient does not have access, the question that came to my is always going to the methodology, is if they should have access to X versus Y and in medical care, where does that come into play. If we do not-ask that question as to where it fits into the care process, then we are going to have this study --
I think that could be addressed, for example, by a service, something-a survey could not be administered to State Laboratory directors but to genetics counselor, to geneticists who say, have you had experiences in which there was a limitation because of the current licensing practices?
Okay. This really gets this issue of commission studies. I think we did not work is a very well. We are not interested here in expanding the literature, per say. That is not our intent. I think we should at it this a little bit. What I think this should be taken to mean is we do not want to reinvent the wheel. We want to look at where there are fallible gaps that we can commission a study in order to address. In that sense, it would make a contribution. Our real goal is not to make a contribution to the literature but to answer specific questions and recommendations that we can make.
And Molly?
I am just concerned that-what I have none too concerned. One is that if you do not go out in a very neutral way and do what I would call market research, you are likely to get a biased outcome your. I think that some of the previous studies had a hypophysis in mind. They did a study that supported their hypophysis. Good or bad, that is what it is all about in the general terms. And this case, what we are trying to understand is what really is the scope of the problem. The other thing is, if you only talk to the Laboratory directors directors, you will never know why companies make a decision to do or not do. You should make sure that you are looking at that side as well well. I am just thinking, because this is the job I do at my company is due in and out licensing. Depending on how someone asks me questions, I could tell you just the horror stories or I could tell you the whole story of 90% of the time it is okay, 5% it is a pain and 5% I cannot get the license that I want. I think it is important that whatever we commission to do this had a script that is not biased by the way it is an.
There is a refrain your. Reid was saying much the same thing very appropriately about the public consultation process. We do not want a bias on the results by asking questions in the wrong way. That is something we definitely need to keep in mind.
I would also like to see that we get also a non modest but look at it from the two perspectives from academic and independent laboratories and the what has been the issue of from individuals has id delete access to the testing? Sometimes you have to wait a year because there is some litigation or patents already. Not only being able to offer the test, but also have you been delayed in offering the test because of organizations, a year year, six months?
I think that is an appropriate caution about-who we address and how we asked them these questions.
Will there be a prospective aspect to this report as well? Are you just looking at what has happened in the past? I think what what I would like to see, purslane for the future, health care in the United States is some sort of plan or vision for what we can do to help get around some of these barriers as we move forward and if there is any change in the patent laws. Also, within the constraints of what the lawyers on and what we have to live with, what specific things we can do to get around some of these things and make them more accessible to the patient.
What I would see the is that as far as the specific studies that we Commission are going to be very modest enzymes and Scott Scott. I can envision and our final report really encouraging that type of thing on a prospective basis. It would look to the future. Okay. The public consultation process.
Jim, could I just.
Yes.
On the last point, the a and office of the director can wait in any time whether we ask them or not. I realize they are currently about what NIH can do on testing and licensing, I am not sure in what context. Idol of the response to the report are if they are in their minds at the moment. I don't know if the Community officially asked the NIH if they had this particular questions that they ensured were in the development of the scope.
No, I do not think so.
Brian, do you want to speak to it. Both of you are serving on the working group.
The percent access part of it it. They may speak to that, or I guess the variety of options on the table that may be presented to Dr. that could either-1 of what might support the work that this committee is doing. I think they have not finished cogitating completely.
I would ask that some of the discussions it is okay, this committee is doing some work so we do not have to do it. I guess the question is there needs to be a conversation at some point between the two committee chairs and simply say how are we going to split up some of the questions. We are looking at the other and waiting for them to finish.
You should do all the hard work and we should get all the credit.
I like the. Okay. We have covered this ground and the take-home message, I think from the discussion about the public consultation process is that we have to be very careful how we word these issues, and we have to try to target the appropriate public. Some of them will be experts in their own right because of their own experiences with these diseases, some of the home will have other interests but we were thinking that we would solicit public comment over a two month. And that would be in early next year. We would invite key stakeholders to the SACGHS meeting next summer and then ultimately develop a final product that documented these public comments comments. With regard to the International perspective, we will identify and invite international experts that have written on these topics or have some physician in which they deal with these things as part of their bread and butter and develop questions for international roundtable sessions that I think, again, needs to keep as a primary focus the whole time is being the practical side of things that we can learn and not just a pipe in the sky type of issue. Next fall we will have the roundtable session as far as developing a comprehensive report. By late spring of 07 and I'm going to develop a first draft then solicit public comment on the report by late summer and by sometime in mid all a have an actual report that we could submit to the Secretary. What I would like to get feedback from today is do you feel that the components of this approach will achieve the steady bowls? What do you think about the timetable? Specifics about the methods that should be used to solicit from the interested stakeholders, and help and patience and consumers who have been affected by Dean patent issues be identified? It is a very difficult situation situation, because like in the discrimination cents, and knows whether I was denied this because of patent issues or I was full it discriminated against. That said, involving the public is an important aspect of this. It is something that people feel strongly about. Comments? Much as I do not like to say the timetable isn't too short, I think the timetable would be fine if you are not going to go out and get new information program, as you are going to do that in parallel with the literature, I think it is a with a little ambitious.
I agree. As I was going through it now, I was asking [indiscernible].
Yes, is the answer.
Exactly. I think part of it is true. We can pursue some of these aims in Palo. I agree with you that we may have to revise this to be more realistic sense we are doing two things that are in and of themselves time-consuming. One is the public consultation. The other is being if we are looking to generate new data if needed.
Just thinking as a little bit about what has gone back and forth on this about getting input from the public and the experts who work in the area. We know that it is very difficult to determine exactly the reasons why access was denied. If I was writing a scope of work for a contract, I might include an option in the contract to do some case studies. following the consultation. So that you could further explore what you got out of it to go back to the companies, to go back to people and say, we heard about this. We would like to find out more what that is about and all the reasons? You might have the option later, if you found that there were some issues you wanted to explore more that you had the flexibility to do some case studies.
I'd like that. You are not just left hanging. You could get some interesting things. I might, as less--next steps. We will revise the scope, we will revise the timetable and move forward and come back to you with our progress.
Jim, since you are really at the end of this, first of all, this is terrific. I really think the methodology here is going to push this thing forward in a way that I had not thought of and just the way that you get at it and the way you try to answer the question of significance of the criteria is going to be very interesting. I am starting to wonder whether or not there are some expertise or consultation and advice that is different from what would normally be it in this sort of demesne that you may have to draw on, waves of trying to crack a problem of how much increase-I'm trying to use examples that I am not trying to stick by, but what percentage of licensing fee versus total cost is a significant number? How do you think about that? Of their regular economists that this is how they make their living? Not what you bring up is exactly what. This is a really difficult issue issue. Trying to quantitate and characterize the impact of patents and licensing on something else downstream as a patient access is immensely difficult, because we know, you can come up with all kinds of theoretical pluses and minuses to the whole thing. Not to be too much of a pessimist, I think it is entirely possible that we will come up with we do not know, that we will not come up with definitive type of data at address them. We have to try. It is clearly important and we have to be creative in the people we ask and the methods we use to try to get at those things, because we are not going to give direct answers. A lot of it will be by proxy. I agree with you that the economists may have things to offer, etc.. It is very top.
We are all going to have to help. We have a lot of smart people to reach out and think of another white. Faugh this reminds me of then and assignable committee that I was on on the consequences of insurance. When you start to try to calculate the economic cost of insurance on a community, it is a not traditional help stuff when you get into a whole variety of its downstream things that almost brought in the need to invent a methodology for thinking about that. This is terrific. Jim has still got the steering wheel for the closing.
Any questions on the work plan? Any suggestions.
Jim, quick question. Is there any hypothesis that we have here or given that it is research, that we are not trying to couch it in one way or another?
I think that the hypotheses will probably have to wait until we see what the gaps are. If we identify gaps that are fallible, once we look closely at the literature, I guess we could formulate a hypothesis. It is important to maintain a large degree of neutrality about these things and say, here is the question. Let's see if there is an answer. This really isn't, in my mind, hypothesis driven.
When ever this concludes in 2008, 2009, is the end product is a report or recommendation?
What I really hope for, and I do not know if we can accomplish this, with the actual recommendations. The real dream here is we identified some things that are good and some that are bad. We say, look, here are three or four discrete things that the secretary can work towards that would enhance the ultimate positive, negative ratio of the pluses and minuses to Gene patents on patient access. I would hope that we would have some tangible recommendations. I do not know if we are going to to. I do not know if we can get the kind of concrete data that would allow for discrete recommendations. I certainly think that is what we should shoot for.
Thanks.
how are we defining the testing? Is it a genetic test, or is it more broadbased?
I think in a way, what we are talking about here doesn't matter so much on the modality of testing as it does on what 13 patents of that debt, but? So, if people on doing as we all know, you can do genetic tests without doing--looking at 18. If gene patenting does not have an impact, I think it is outside of our purview. I would try to keep in focus the fact that we are interested in how gene patents affect testing. Usually, I think that will rely on looking either at the gene or its expression in a fairly proximal cents. I think that would be the answer answer.
The only reason I am asking that is I don't know what it are you going to be exploring back or not? Not say that again.
I am not an expert here, you are not looking at specific genes, but a platform or methodology.
Right. Let me think. I have not thought about it in those terms. I do not think we are talking here about the patenting of technologies and platforms. I really think, and please wait and if this is not what you will perceive, I think we are interested in how the taking out of patents on genes, on specific genes have an impact on all of this. That is different from, say, patenting and technique for sequencing or patenting and technique for analyzing and expression,. Would you agree with me on not? Yes.
You said something earlier that I agreed with and now you have raised it differently. Clarification is useful. When I think of a gene patent, you said Gene patents and it's close derivatives, which is the gene, what does that encompass? One of the things we will have to do when we start this road is define what we mean by that patent family. I will share the experience that two years ago my office tried to ask the question of the effect of NIH's licensee and be metrics that we should use. You cannot use a single one. If we look at the entire family of patents, we would not be able to get any answers. The person we should do is sit down and mean what we mean by Gene patents. That will lead us into our case studies so but they-limit our questions with very concrete --
I agree. Your question brings up a good one. We obviously do need to define that. The check comes into how far downstream from the gene do you go? We would all agree that a New sequencing technology would not be something within our purview. I think we have to decide if it goes anything beyond the patenting of a specific sequence or [indiscernible].
I will give you a specific example of how a gene that can affect other testing. When you own the rights to a gene sequencing, you are authorized to use, to make, to have made, to have used, etc.. In any case where someone wants to develop a protein test, and they have to express that protein predominately, they can not use that that unless they have a license. It is pretty. If you aren't --
Those are people who owned the patent on that, the sequence of that gene.
That is the whole point of Iraq that would be important for us to analyze the impact of rock it goes beyond just a test to see if someone has a specific snap.
Exactly. When someone else has a patent on a sequence, they owned the whole shebang.
Just one other wriggled to that. You also have to take into consideration the fact that some of the cancer tests that might be developed, you could have a gene expression changes that are not due to any synthetic or a beautician but a in difference or something. You would not want to be quite so near to demand.
The question is, what those types of things be covered by someone who owned a patent on the sequence of that Jane?
No, not necessarily. They could presumably that sent the information involved in the methylation testing. Therefore, you still have something that would be acting like gene tests or genetic tests the way you are defining it.
What we are thinking about here is just on changes, expand it to variances and also changes that cause cancer, which is a genetic disease.
Again, what this gets back to is if someone owns a patent on a gene sequence and it is derivatives, if that is used in their in-or in someone else's andResearch, a diagnostic test, it is covered by the.
If it can be used to block from someone doing something that could help a patient.
Right.
Help me out here. I am not sure I understand how this will apply to commuter it has. Proteins or peptides.
I do not think it would, unless they used in their technology, the sequence, just like you described. If they need to create a peptide from the gene that you have planted it, then it would have an impact. As far as a technique that uses NNR to look at products and the serum to diagnose something, that would not be related. That would not fall under the issue. We are talking about-there is some confusion about what a genetic test and what is not. That is legitimate. What is a gene patent? We can come to a concrete definition for that. It is the packaging of the sequence and its [indiscernible].
Jim, just to remind everyone, it is in the statement that you have in your scope of work, but the charter for the committee covers those. Gene patents and access to genetic technology. You still have to define --
Gene patents and the impact on access to technologies.
Genetic technologies. It says genetic.
It started with Pat and.
I see it as overlapping circles. You are only doing that which is colored in the center.
Right. I think that is in and of itself a huge bite to do off Perot I think we need to focus on the impact of patented gene sequences and a [indiscernible]. That would make sense.
However, everything that has gone on with the gene patents is likely to occur or further down the road with proteins and [indiscernible]. It is more than to genomics. Whatever recommendations are made regular to Gene patents could easily be translated and applied at an earlier phase and hopefully prevent less of the assets was a petite and clinical practice on the.
That is a good point. I think we are a little ahead of schedule.
For which you get an extra cookie.
Good job. Thank you. Give him some applause.
This is going to be a fun one. Public testimony. No, he is not getting a project. This is public testimony before.
[SPEAKER/AUDIO NOT CLEAR]
There was not a break scheduled? Why don't you take five.
We will start at 25 of. People want to go, earlier. Of these people are saying that they want to go home. So, 25 of.
Okay. We are reconvened. One of our critical functions is to serve as-I knew there would be trouble over there with the caffeine. We are a public forum for the deliberations on issues raised by the use of genetic technologies. We greatly value the input we received from the public. We set aside to hear from members of the public, and we welcome and appreciate the views that they have with us. In the interest of our schedule, we ask them to keep their remarks to five minutes. We have copies of your statements which will be made as part of the meeting record. Today we will be heard from Dr. Dr. that works with the Maryland Department of Health, but is speaking on her own today. Good afternoon and thank you for letting me be here today. And the understanding the genetic basis for life as an overall impact on the ideological flames within people feel themselves, their actions and others and society. a new paradigm shift is starting students to more common afflictions such as diabetes, hypertension and asthma, estimable 60% of the population. Scientists are turning to genetically simplified population [indiscernible]. Particularly troubling. In my research I identified that we can learn a lot by focusing on this population that have gone through the experience of population based studies. The in and the perceived willingness of Jewish families to participate and genetic testing. was a very costly. On my other population studied for to gnomic testing, Jews carry the scars of the Holocaust Holocaust, the scientific notion that Jews are members of an inferior race. They are concerned that the focus and lead to stigmatization and discrimination of this group. Of American Jews in they've received little attention in the literature as other religious groups like Hindus and Muslims. In spite of the racial, cultural and social of a fluid, well-connected and well educated people. Even when social studies are done on in related to genetic testing, no distinction is made to the various subgroups that make up the Jewish community and conclusions tend to be penalized to the entire U.S. population. As an example, the American Public Health, they operate as an advantaged group and a son of a and conservative estimates suggest that more than 600,000 Jews are recent immigrants to the U.S.. Invisible American today is some is the reverse with respect to religious the denomination's. The majority of U.S. Jews define themselves 40% of conservatives and 11% are Orthodox. The various branches and some Orthodox Jews strictly in assembled importantly the joyous population shows wide economic social economic disparities while the average U.S. household is well over the national average, when six of Jewish households fall in the lowest income category. It has been well documented the in pick-up have inadvertently led to discrimination and stigmatization of this group. Moreover, my dissertation which focused on the impact of it identified serious issues related to it especially related to marriage prospects, which largely utilizes arranged marriages. An alarmingly growing segment of the population, especially women women, in this community, and an ability to marry and procreate [indiscernible]. This inestimable with respect to its genetic endowment. For example, some of these studies already underway to identify genes for common disorders, include inherited mental disorders such as schizophrenia and bipolar disease, autism and Alzheimer's disease. Putting such an identifiable prejudices will be defined and biological terms. And as hon. as we spoke about earlier the emphasize a host of important social and environmental constructs and the and treatment. widening health disparities. In summary, I recommend that when studying public perceptions researchers expand definition of the population. They should include all stakeholders, especially those groups already every boy's account. Seven, it might be beneficial to have the federal offices charged with eliminating health disparities take part in this important deliberation. Thank you.
Thank you very much and thank you for taking the time to do that. Let me ask if there are any questions? I think you were very specific about how you wanted us to consider your comments. I wonder if there are any questions or comments?
Yes?
It is not a question but thank you for bringing us some specific examples of the phrases that we throw around easily year and we forget what those phrases mean. Thank you for doing the.
Thank you very much.
Dr. Deborah of Leonard is presenting on behalf of the Association of molecular. Molecular pathology.
[SPEAKER/AUDIO NOT CLEAR] Good afternoon. I am changing hats from a previous member of the SACGHS. I am not representing the Association for molecular pathology. I want to make clear that I am representing them here. I will take the hat off so that you do not think this is totally totally. Them for issues relevant to the charge of the SACGHS. First, to draft guidances' from the FDA on [indiscernible]. AMP is finalizing comments to the FDA on the draft guidances' that raised concerns for the membership of the AMP. The first of guidance commercial it distributed the Cali asked questions defines a much narrower interpretation of the AS R. Bass. Ha AMP limit the availability of which laboratory developed test under clear regulations. Has ASR become more limited in availability either let the Tories will find other sources that are of poor quality such as research reagent of the laboratories will stop performing many tests that are currently standard of care. This could lead to decreased patient access to a minute to testing services to the the second death in divines the FDA's regulatory approach to complex and multi variable tests but while AMP agrees that the tests interpreted the buildings that are not transparent and able to be manipulated by the Laboratory warned that the a interpretation interpretation, the use of an Internet of problem is your team and medical practice and should not in and of itself raise specific concerns with the FBI. The purpose of raising these concerns is to ensure this advisory committee is aware of these draft guidances' and I was not aware at the time that these were being presented right after the public comment. And their potential impact on genetic testing experience as. One AMP finalizes there the letter can be provided to Jim, if that would be of interest. My second area of concern is regulations. AMP is concerned by the recent decision not to incorporate a genetic specialty into clear regulations. Frank, we are mystified by this action which follows years of favorable comments from the clinical laboratory and genetics communities in response to the initial CDC from making. The current regulations that have not changed in almost 20 years divined genetic testing in terms of classical side of genetics only. Dividing testing and explicitly would allow for a prepared regulation and oversight of these tests by the agency tested and legally mandated to regulate what for is performing these tests, these clinical tests. Define a genetic specialty within the clear regulations would promote provide better oversight of a genetic tests and reassured the public and members of Congress about the quality of genetic testing performed in clear certified laboratories. The members of speethirtspeethirt een strongly urge SACGHS and encourage the aggression of a genetic specialty within the clear regulations. The third AMP numbers performed genetic tests and other types of molecular tests with the management of patient care. We remain concerned that the codes and reimbursement levels set for these codes are less than the cost to perform these tests. While the SACGHS on coverage and reimbursement issues made recommendations that speed development plan, but 13 is not aware of any action taken today on this issue. AMP applied SACGHS for its recommendations and asks SACGHS to follow up and determine if action will be taken to correct be an adequate but levels for these codes. Finally, the gene patent issue. AMP asks that SACGHS give full consideration to the negative impact of exclusive licensing and enforcement practices of dependence on the future of genetic testing. We understand that SACGHS said it had a high priority issue and is formulating an approach to on patient access to molecular test. AMP wants to ensure SACGHS that the gene patent enforcement continues to limit the tests and perform for their patients. We urge SACGHS to develop a plan of recommendations to the SACGHS to address the critical impact of these issues. AMP remains available to the SACGHS to assist with or provide additional information for your thoughtful deliberation and important work. On behalf of AMP, I think for the opportunity to speak with you today.
With that have still on, are there any questions to follow up up? Private. You will be available-since we are about to turn to the key recommendations you may, which is around the oversight issues, --
You also going to hear about the guidances and about the decision not to have a genetic specialty.
Okay. We are getting ready to get into the oversight sought.
I did not know that you were hearing about those.
Perfect. We will have a chance to follow up specifically. Very well done. Okay. Let me refer all of you-But, by the way there are written public comments also on the International Society of nurses and genetics endured tabled folders. There are others in the audience that would like to make comments, we have another public comment scuttle tomorrow. Please sign up at the registration desk if you would like to speak at that session. We will turn to the session on genetic testing laboratories. This is an issue brief on oversight on tab six of your briefing books. I think it is important that you take a chance at at some point to a study that. To set the stage for our presentations and discussions, I am going to review its debt of the background on this important issue, particularly for the members of the committee that are new. Oversight of genetic tests have been a public policy concern for over a DEC wait for. Both FDA and CMS have responsibility for regulating a genetic test and for regulating genetic testing laboratories. Currently, genetic tests developed in-house by individual labs are subject to less regulation and commercial it distributed genetic tests. Most genetic tests that are currently available are in house developed tests. By the way, we are using the word and House, because we have been banned to use the word and gone.
It is still not a house. It is what it really develop.
Today, it is in house. We will keep Pan it is not home brewed.
At least to stop the growing, but at least I am still working from home.
With that modification put an addition, as we know from Judy's presentation in June, regulations on critical laboratory, the amendment's author currently lacking specific provisions for laboratories performing genetic tests. And assuring the quality and of the genetic tests have always been a high priority for our committee and for our predecessor committee, the best- best-D a has concluded that a critical gap has happened in genetic tests relating to clinical validity and that a new multi faceted a framework was needed. There were three components of the a recommended for more. Per first, the FDA should be responsible for the review, approval and labeling of all new genetic tests that have moved beyond the basic research and waves. The level of review applied by the FDA should correlate with the level of scrutiny warranted by the test. Second, CLIA should provide prisons for providing the quality of the laboratories conducting genetic tests. Board, a collaboration between the public and private sectors coroneted ICDC in discernable on the clinical validity and utility of a genetic test. And 2,001 then Secretary of State one accepted these recommendations and indicated that HHS would proceed in a stepwise what do and enhance --
In 2003, requested from CMS and FDA about the status FDA said that they had no plans to regulate laboratory developed test. At the time, there were questions about whether the FDA had statutory authority to regulate laboratory developed test. CMS said there were plans to add a genetic specialty to the the the.edu regulations. At our meeting in March of this year, we heard public comments questioning why CMS has not moved forward with the proposed rule amending the CLIA roles. In June we invited Judy to update us on those plans. She indicated that the rule making process was proceeding, that proposed regulations had been developed and where we incurrence with CMS and that of all went well we might see proposed amendments by early 2007. At the end of the meeting in June we decided to revisit the oversight issue at this meeting to have a more in-depth discussion about whether any gaps in oversight process, and I would indicate any gaps and over cypresses that we think are important enough for us to be attentive to. Today will be updated first by CMS and then by the FDA and then have a pope is discussion on whether SAGET should take further activity in this area. What I mean by that is should we move beyond a monitoring role and undertake some the birth fact-finding and analysis? Let's be clear program what we are saying that we want you to do is listen carefully to whether or not there are significant gaps and oversight, but not just to discover that there might be dealt, but to discover whether or not those gaps in our role as representing the interests of the public, whether those gaps deserve further attention. It is not dissent there is a gap gap, it is that GATT will be of further attention by this committee? Those are the two things that we are going to do. Let's start with Thomas Hamilton and duty. They will be presenting-Steve is going to go first?
We want Steve to go first?
[SPEAKER/AUDIO NOT CLEAR]
Indicates, we are going to turn to Dr. [indiscernible]. You may recall his last presentation was in March when he talked about the FDA critical in and cutting edge medical products to the marketplace. He described FDA guidances' released in February 06. Today he will clarify the current regulatory paradigm related to oversight of genetic tests and tell us about some additional guidance is that the FDA has issued on this topic. Dr. Steve, please proceed.
FBI has been embroiled in the medical devices in general and of in vitro diagnostic devices. That is our code term for an test is in bed together since the annulment of 1976 preco those amendments introduced in the first time a variety of general controls for laboratory tests including requirements for companies making them to research and lest their products to follow good manufacturing processes and to report adverse offense put the as a result of those general controls, for the first time in our history, we had a menu of tests on the market. We have mechanisms for ensuring that companies produce those tests consistently falling good manufacturing processes overtime and we have mechanisms for insuring that companies that experience or identified post marketed as a first offense would report those advanced to have DNA and hopefully, we would work together to resolve what had gone wrong. That insider predatory framework was based on a brisk pace regulatory process. The 1976 law also introduced for the first time in the U.S., in fact for the first time on this planet a requirement for premarket review before a new medical advice could enter the marketplace. Although that review occurs in very different kinds of administrative packages, there is a comment interest. That interest is demonstrating that the test is analytically reliable. Until analytical performance is locked in, it you are playing in a sandbox with no sand. For some tests, it might stop there. For hemoglobin, we do not ask that you do is demonstrate him clinically that hemoglobin is associated with anemia. We might be quite interested in understanding what the clinical meaning of that signal would be. Poured all products, we are very interested in labeling and showing that there are adequate instructions, but there is a clear intended use, but there is a clear performance and the limitations, but I have yet to see a test that doesn't have some either political or biological limitations. FDA regulations read that common to market to try and keep the rabbit, I will try to save its laboratory developed test rather than in the House or home brewed test. These are diagnostic tests graded in a single Web for use at the specific laboratory. They have been called and House. The use of a laboratory develop test is that that they're well established practice, but there has been a broad menu of tests yielded from this particular practice it is not an entirely trouble-free practice in that there are differences between the regulatory route for the product and for the product that does not visit FDA. Some of the differences are that for the product that is developed as a laboratory developed test, there is no requirement for discreet premarket review. There is no specific the operation of segmentation between the research phase and the clinical phase of product use. There is no prohibition against demonstrating clinical politically, but there is no explicit requirement for political validity. The outcome of those differences revolt in some very cold but players. Anyone who has not looked at the recent GAO report might want to go on the Internet and look at those and see at one extreme some of the very colorful players. In 1997, the FDA published and in role and defined-it was viewed as a us by and for mental change in the laboratory developed tests, although it was defined as a down regulation, since by default, and we define those to provide an actual comprehensible and was the definition. I would characterize them as the building blocks of the laboratory developed tests. We define those as tests that were subject to regulation and the regulation was a requirement for general controls, such as registration and listing, good manufacturing practices, adverse reporting, but we drew back requiring blanket premarket review this was not an accident. The rule was deliberately developed to create a stapler for laboratory develop test, to create clarity, to about his practice to go on unabated but to go on with some increased quality because we were trying to care for the building blocks of themselves and to increase transparency and that there was some labeling requirements. The FDA does not have a lot to do with how rude to us. It is crafted a little differently, but that is what they are saying. It you go back to the preamble, it codifies the fact that in spite of the ASR rule, the FDA did, and it has always had, although in some cases it has been rather silent, it has always had the belief that laboratory developed tests are in fact, within the definition of medical devices and laboratories creating those tests are within the definition of sponsors and manufacturers. These are all subject to FDA and jurisdiction under the 1976 act. ASR role was a very will. I was one of many authors who tried to get it right. It had some surprising and disturbing unintended consequences. That was a follow up to that rule that was either inadvertent or in some cases deliberate abuse of the role and that what people began to do is take what were either it is or large pieces of carrots and call them ASR I when I say a kit I call it an ASR in the clothing. To make decisions about test of precision that were intended to fall within the purview of the laboratory, companies began to do that and to make implicit claims program again, either inadvertently or deliberately, the ASR role went right over a cliff. The guidance in 2006 was intended to do nothing more or nothing less to into clarify whether you follow over the glove. It may not publicly and privately clarified it, but we are very intent on not having people take gets a, call them ASR and market them outside the construct of U.S. law. We are very interested in getting that right. The intent of this document was to better clarified the definition of a ASR, the limitations on marketing and ASR ASR. From four prospective there is nothing different in the meaning meaning. There may be something new in examples. This document is not intended, this is neither good or bad news, it is not intended to-it is not a frontal attack on home testing. It is an interesting dialogue on what FDA might construe as home testing. The laboratories have the same responsibility than they did when the rule was published that they have to step to the plate and say that I developed, I designed, I validate, I stand behind this test. That is the intent. That is the spirit of the ASR guidances. Comment on those items as close on the first week ended Dec.. We will have the possibility of aid and one month extension. It is very important to note that the ASR rule does, in fact, provide a class one exempt status to the building blocks of of, but it did not go further than that. It did not help or hurt. It did not provide benefits to the laboratory test itself. The ASR did not exempt laboratory developed tests. The FDA action not to regulate laboratory tests remains grounded in the same history, in the same thread that is a matter of enforcement discretion, not a matter of legal exemption. This may be some people outside of the agency, or within the agency, confused by the. Laboratory developed tests cannot use the ASR exemption. They can appeal, applied, benefit from the FDA expert use of enforcement. In fact, if you see anything in the next client, the FDA is appreciating changes and laboratory developed tests that makes it the gap for some products more disturbing that it has been in the past. The lack of premarket review or lack of research to clinical threshold, the lack of parity, as a matter of fairness to some in the industry, the lack of discreet evaluation and the lack of clinical evaluation for some particular cutting edge and high risk in in this wonderful world of 2006 are of interest and the FDA, in all honesty is trying to reassess and do some soul-searching in this area. Pat, I guess is startling to Baroness constituencies in part because there has been a periods of silence on the agency. I did in fact probably say how all the things at a previous SAGET meeting. It is startling because it is an expression of a party. The document was hardly the most dramatic expression of a party. If you want that, you should go to the minutes on the congressional hearing on the aging where it assembles started to express this authority. The bad news is that this is actually not new stuff. There is nothing under the sun. That is what I try to tell my daughter, and she tells me what is new and what I do not understand. I must tell you, and I have stolen this line from Dr. Mansfield and of official FDA brake that this is Gary much like a Dickens novel. You have seen the first chapter and you have not seen the last chapter, assuming that FDA is working with Charles, at the last chapter has not been written. I assure you that there is plenty of time for people to put great ideas on the table about what the FDA should do. There is plenty of time to say what the FDA should not do. Better to put on the table what the FDA is worried about. To speak to any group in any sector that are addressing those issues, that is good news for FDA. The multi buried dozens is a specific example of a diamonds that is fueled by the FDA concern that it is not such a great idea to regulate all laboratory developed devices and that the blanket application of enforcement discretion is not a particularly brilliant public health move for all laboratory- laboratory-developed devices. The a have been over red. While it is clearly a signal, it is a much narrower signal than the laboratory community or the community in general has appreciated the when we read looking about the that we were worried about and interested and and, we were thinking about one dozen or two dozen products that might be on the market and this might be an incredible market in the few years. We are not thinking of hundreds of thousands of we were worried about a growing category of tests that seem to not fit Judy's mold, be the kind of being an average and Specter, whether the bid or working for would-be able to access and understand these are tests that produced novelty with new safety and effectiveness concerns and to us that we thought were very poor for its for enforcement discretion. We thought of this. We still think of this. Maybe we did not communicate it right and maybe it was misled. We were looking at a narrow area of the devices. Those devices that were devices that have the use of software driven and software to ride, and I am talking about complex software, rhythms or formulas, that they would take information and create a patient specific score or index and that a normal trade pathologist on colleges or cardiologists or neurologist would look at the index and say what does that mean? It would not know what that meant unless they contacted the sponsor and color it in for them to explain what it meant it would not be able to second-guess it it a life depended on it. That is what we intended on.
[CAPTIONERS TRANSITIONING].
[Captioner attempting connection to audio line. Need the line to be hung up so I can dial in.]
And if the answer is yes you won't have trouble with that FDA. Our deal is to promote public health by getting good product out quickly and to protect public health by either keeping bad products off the market. We don't actually have the strongest was in the world. We take very blessed tests and we label them. We do believe in transparency. If you want to see where our regulatory process is like, go to will and type oivd. We make all of our regulatory decisions eparch of public record. You want to know what we did with cystic fibrosis to the process or with the latest PS8 test all you must do is go on our web site. Everything we do is all a matter of public record, and you are always free, everyone is free. And in fact lots of people aren't shy about telling us what we are doing well and what we are doing poorly in the wake of review. We do like to think that our mantra is good science and that if our science is dead, of our science is focused you can get rid of us. You can get us out of the picture, but you can't get rid of the pesky questions we are wanting to put on the table. Thank you.
The key. I noticed that our colleagues from cl S are here. And we will turn to them and a second. Let me just ask you one thing and then we will get to them and open this whole thing up for discussion. I thinking I am still -- I guess I just want you to be as simple and straightforward as possible.
That is hard for me.
No it isn't. You are the best person to ask this question. So if we are sitting here as a community trying to make sure that we are being attentive to our responsibility for thinking about issues that protect the public in this regard and we are trying to figure out whether there is a gap that we should be attentive to, now you and FDA -- you get paid. That is what you do is protect the public in this regard. So this is all you guys to all day long.
We dream about this stuff.
You dream about this. So the question becomes when you boil down your presentation about good science and good public health, at the end of the datebook do you consider from your perspective there to be a gap or not that anybody should be worried about? Is the sum of your reports saying all is well, return to your homes? Or are you saying something different?
Yes, the problem is that there is a partial gap because the sector that we don't regulate and the sector that Judy does regulate is very heterogeneous, and so there are entities that do it very very well and there are people in that community that I would turn to if I had a question that I didn't know the answer to. If I had a heart submission I would be wanting to call them for it fice. So when it is good it is very very good and when it is mediocre it is kind of mediocre and when it is bad it is really awful. So there isn't a uniform gap. There is unequivocally a gap. There are a variety of ways to address that. FDA is the only way. I think -- is not a bad way.
Steve, so that we can -- people who are not smart about this like you are -- and people have this walk right up here, would you please with your expertise define the gap. What is this gap from your point of view?
From my point of view there are three things. It is good as ," as good as D.C. 88 are. They can't go in in a credible way in one and two and even a three days and actually figure out what is going on in the data. I have seen too many -- Judy has been too generous with me in terms of assuring to bodice and packages and information from multiple and points. And I have seen a lot of bad data when a bullet in odd ways. This is from people who who do it because they don't know and people who do it in spite of the fact that they do now. So there is -- one there is a gap. There is some value to implement this that the assessment and labeling. They're is a value added to that. Secondly this is a very competitive universe, and I understand that both labs and companies are not like me. They are not against public health, but their bottom line is their stockholders are with less and hospital administrators. There are economic pressures that make their decision making process different than ours, but I would say that there are -- there are -- there are differences in decision making and in the laboratory community there it's a capacity that does not enlist [ indiscernible ] community which is after the first publication you can't bring to life as a non Research test. All you need is that first publication. The third gap is that I do think and you can ask Judy or you can ask her boss -- I do think that there are differences in the law itself, and the bank itself in the ability to go after clinical validation. And I believe that we have that as a core principle and I believe that we have this as a basic limitation.
Thank you. You say there or nearby. And we are going to turn to Tom Hamilton, director of survey and certification a group for CMS and Judy is director of labs and to care services for the Center for the case of services. Tom, I think you are going first, is that right to give so both you and to the -- however you we'd get into what you are going to present can you please also highlight when you get to that part of your presentation what you see as the gap? And help us to understand whether you're on the -- well, let me just leave it at that. So you understand what the question is in the context of this question for us who are not as sharp as you are about this. Given that your job is to protect the public make it simple for us to tell us whether all is well and we should return to our homes or whether there is something really important that we should attend to. But we just need you to be clear about all of this after you have made the formal presentation which I'm sure will be clear.
The expert much and bank you for inviting us. I'm Thomas Hamilton. I'm the director of the survey certification group and easy yes you all now heads up our division of clinical laboratory eminent. By what we are going to do is switch off a little bit and walked through the power point. She will start with a the background of the clinical Laboratory Improvement amendments. In order to put this into context because there is some misunderstanding about what Clea can and cannot do and about some of our priorities. So we will start with the back ground-based and then I will come in in the middle and talk a little bit about one of the controversies of the day which is whether or not there will be a genetic specialty regulation promulgated and then Julie will pick up again and talk a the but more about Clea and then we will open it up if that is all right with you Perrysburg.
Good afternoon as well. This is near the end of the day, but this is a really exciting topic. Again, I think as Thomas indicated these are the things we are going to walk through. Also we want to provide you an update of what happened at that hearing beginning director consumer testing earlier this summer. We have also been asked to provide a little synopsis of what the New York state's genetic testing program does under their state license or law as well and then people move forward. Just as read so aptly did he provide you some background on the history. And I think you can't go forward without knowing what that is. But I think before I began -- and I think it is reported to say that CMS goal and administering Cleo is to ensure the highest quality testing for all types of testing because as many of you know 70 percent of all health-care decisions are based on a laboratory results. The final clean-air regulation for public back in '92 and cover all testing in the United States including genetic testing. A task force was convened by the end I8 and Department of Energy back in 97. They released a report that did not provide specific recommendations, but did say that Cleo should be augmented. Following that recommendation and at the request of the Secretary CMS and FDA and CDC work offline together to discuss how we would implement genetic testing oversight under new regulation and what tasks were in front of us and how we could work together to accomplish them. Based upon that recommendation however the community which is our advisory community did convene a work group of experts who subsequently provided a whole listing of recommendations to us regarding the changes that should be made for genetic testing. And of course FAC T 8CGE which is the precursor of you're committing did also support those recommendations. And subsequent to that CDC did publish a notice of intent. What that notice of intent did was to list those recommendations and to also list the existing requirements that correspond. That is so individuals could then comment as to whether that particular improvement might be needed. Interestingly enough the comments to that notice of intent were pretty mixed. Some people work for changes and others were not. But as a result collected revise its recommendations into does one. Following that CMS actually published final regulations so even though he said nothing has been done in 20 years, actually we published a rather comprehensive final quality control regulation in 2003 which actually strengthened quality control for all testing. And it also included some of the recommendations from Clea as well. Some of those things that we're added to that two-seat regulation from the act included quality control for PCR testing, unit directional workflow to prevent specimen contamination, enhanced confidentiality requirements as well as enhanced result recording requirements. As a result of that notice of intent there were still a number of open issues, some of which are still unresolved. Also the recommendations will be on the scope of Korea. They included such thing as patient consent which of course is not included under clear. And because the field is relatively new and dynamic and I think we all heard that today, there was concern internally and externally about writing prescriptive requirements that could, in fact, prevent the lavatory or limit them from using the tests and new technology. And I would also like to because I think I did myself that this service. When I first presented this to yourself and other committees as far as the summer and overview I promised I would not go into excruciating detail, but I would like to show you a little bit more of the scope because I don't think I have really done that. A have to say that this audience is relatively unique and that your folks to want regulation, most of the people I talk to our people who don't want us there and never want to see us again. And so the minimal burden that we can provide is all they want to hear about. So in that respect I have always been relatively conservative about providing an overview of the regulation. So today I would like to give you just a little more detail. Not to describe it, but to show it to you at least. The first and foremost requirement up like to talk about its quality control. Quality control is easy to describe. It is a real time evaluation of test quality. Because it is something you do every day, you want to check that your test is working before you report your results, but that is all we ever really talked about. We didn't really say that quality control includes all of these things. It is not just the fact you are running something every day to check the test, but you must do all these other procedures under clean up partly in order to show that the test is working. And so it is very important to mention that. And the last thing on the list is the specialty requirements. I think it is also important to note that these are actually created to correspond to the Medicare payment coats. That was the reason the laboratory specialties were created in the first place. Things like hematology and microbiology and so forth. But as easy as we go on through this presentation you will see that they only are a very small piece of the regulations. Sometimes there will be a specific quality control requirements before something like under hematology for coalition tests because they are unique. And so a certain type of D.C. is required for this test. So they are very limited in scope. Curly there is no genetic testing specialty as was previously stated. However, tests that are considered genetic are actually currently dispersed throughout all the different laboratories specialties that exist. Although because we did strength and quality control and 2,003 we made it from anything the manufacturer could call a quality-control to something very specific and very stringent, to levels of external QC of testing. We felt that the need to have as many specialties as previously existed was no longer necessary because we had strengthened everything over all. And so the number of specialties has actually instead of adding which we have never done, they have actually been reduced over time. In addition, I think I would like to also mention that we found some information that indicated there were laboratories that did not have specialty certification. We went through the clear database and actually discovered that there are no laboratories existing currently that do not have some type of specialty certification and are doing now on with types of testing. And the interesting factor is that clear, as you know is user fee funded. Therefore the fees that the labs are assessed under the cleanup program to pay for its cost actually use that specialty information to calculate the fees. So there is an absolute need for us. We need our money to operate this program and so there by every laboratory has a specialty certification. Again, Quality Control tells you if the test is working each day before you report your patient results. It should monitor the test operator. It should monitor the actual analysis, the procedure that the that is doing, and also the environment where the test is being performed. For example if the lab is too hot many test systems don't work and provide an correct results. And so it is important to perform that quality control. The next regulation or requirement for Standard I would like to talk about is proficiency testing. Proficiency testing is a measure of long term accuracy of the laboratory testing. It is not something that you can use on a daily basis because you don't get your keep the results from your provider for three months. So those test results went out and you didn't do any quality control. It won't help you here. It will just tell you from afar that the test is working on an ongoing basis. And you don't need to have a specialty in order to perform proficiency testing. All Monde with laboratories have to perform some fashion of proficiency testing. Currently in the regulations there are 83 tests listed that must have formal PT performed. However, there are at least 1,000 different tests that a laboratory might wish to perform. And they have a corollary requirements for those types of tests because twice a year the laboratory has to do a test to insure the accuracy of those tests. So you must do one or the other. You can't escape from PT, but you can see that for the most part most tests do not have a formal PT required in the regulations currently. In addition, our surveyors look at that. They look to see what the lab is actually doing on that twice a year. They are just looking at the results you get from the program. They are looking to see what the that is actually doing and if they are doing it right or are doing it at all, they said does. I actually pulled some data and was cited quite frequently. So they are clearly looking.
Let me to stick sure of one thing. Are these genetic tests or are these not genetic?
This is Clea over all.
I'm just asking. At it want to specifically know if this relates to genetics.
No.
Thank you.
I need to provide you a context of where you are going. Also we checked with the College of American pathologists because they jointly have a proficiency testing program currently for 46 categories of genetic testing, proficiency testing right now and there are approximately 16 or 300 laboratories in rolled in those programs. Me and stand that there are about 600 laboratories and the United States that do genetic testing currently. However again because there are no formal PT required for genetic testing right now in the clear regulation the law passed to perform the twice yearly evaluation. So a statement that a certain percentage of labs are general the NPT has no relevance for compliance with clean up because it is not required. Also we saw that there was a recent article in which potential errors were considered to be possible problems in genetic testing. However, in evaluating the data more closely it indicates that most of the problems that were self reported and certainly qualified as being a potential or actually in the pre and post analytic face of testing, thereby specific requirements for genetic testing would not have affected these errors at all. The requirements that would apply would be the pre and post analytic requirements already in place and Clea. Pre and post are both things that we search as investment collection and processing and result reporting. In addition, you can see there are also requirements for record keeping, confidentiality, Sebastian and integrity and LeBlanc and for handling of complaints. Because most genetic tests are considered high complexity there are a number of required positions in the laboratory for all high complexity test currently. The laboratory director who has the overall responsibility to ensure the quality of testing. In addition, to responsibilities under Clea there are educational, experiential and training requirements for each position listed under Clea as well. The responsibilities correspond for the laboratory director to all of the Clean Air Quality Standards. In many cases the laboratory director is started as a deficiency during a survey if there are significant quality program to the problems in the laboratory. The chemical consultant is responsible for ensuring that the protests are ordered and result interpretations are correct. The technical supervisor has the responsibility for the scientific and technical aspects of the laboratory testing. And that also is the selection of a protest. The general suppressor is responsible for day-to-day oversight in the laboratory. Also each individual and the laboratory who manages and does testing must have a competency checked once each year and when a new test is at it that is twice per year. So competency is not proficiency testing. Last but most importantly, under clear that there is quality assurance. This is an ongoing mechanism, an overall plan that the laboratory must have to assess the quality of its own testing to solve its own problems and communicate with its clients, it's patience, it's staff. It encompasses all of the clean air quality standards. I guess you would call it a package deal and that is why I have the package there. If you take quality control and proficiency testing and personnel requirements and record-keeping and wrap those in a bow, that provides your quality assurance. All laboratories that perform non wave tests including genetic tests are surveyed every two years. If there is a complaint alleged against a laboratory that is followed up immediately. The survey process this outcome oriented and utilizes an educational approach. That is any laboratory which does not meet a requirement has a deficiency said it. However the surveyor will clarify that requirement for the laboratory and also resources to facilitate the compliance. The goal here is to get the lab to do the right thing. Then the laboratory has the opportunity to correct the problem. If all else fails after several attempts Clea does have a section that can and will be opposed against Laboratories based upon the seriousness of the problem and the scope of the problem. They range from fines from $10,000 a day to loss of Medicare reimbursement to losing your certificate. Clea has an unusually high success rate and that the proportion of proposed sanctions when we won the laboratory that people to this if they don't to impose sanctions is about 10%. Again the survey process is very effective. It looks at outcomes and test results. It is interactive. We talked of the personnel and the laboratory here do the testing and manage the laboratory. We observe testing throughout all phases and reviewed records as well. The QA program at the laboratory does encompass all of the quality standards that is the pivotal piece that really tells us, that is the clue of how well this laboratory is doing. Now Thomas Hamilton will address the answer to the question that you have all been waiting for today.
Let me begin by the question posed. Is there a problem? I have to confide in you that I am probably not the best person to pose that question to because survey and certification groups, we are the Grand Central Station were reports of medical misadventures from all over the country to arrive daily. So we have a very distorted view of the American health care system. We are is possible for the on-site service for just about every type of Medicare certify provider, hostels the Syncom's dialysis centers hospices and so on. And everywhere we look their is a problem. So it is not so much is their problem, but rather what are the most effective ways to go about identifying and addressing the problems that to arise. As we looked at the topic of whether or not there should be a regulation establishing a new genetic testing specialty and put a fair amount of work into that when we take a rule through what is called our clearance process, it is an internal deliberation process involving the major agencies in the department. And we ask ourselves a number of questions, we are obliged to establish positive answers to make the process a little simpler. There are three questions. First this is there an absolute benefit to this particular rule? And we might ask is there a problem for which the proposed rule is a remedy? Is it a significant problem and does the rule if it is a significant problem effectively address that problem and how strong is the evidence that suggests that the proposed rule will indeed effectively address the problem that is identified? And this is where we first ran into problems in terms of this particular issue and the regulation. To what extent is there evidence that there are problems that are not only soluble, but currently unattended. And when we look at this one of the strongest arguments I have think is the argument in favor of proficiency testing. Genetic testing specialities will not magically make proficiency tests available. And the way that clear is set up, NIH is not directed or authorized or funded to go out and create these tests. So I see statements at various communications saying CMS is not established to test and is falling down on the job. The way the clear law is structured it is the professional societies, the professions that establish those tests and come forward and the CMS approves those. So right now if there is more than a thousand genetic tests there are only a few proficiency tests available as to describe. We ran into this problem and this will illustrate the issue well I think. In 1988 the version of Clea cytology proficiency testing for gynecological examination, post prescreening was required. It was only and tell -- it was not until 2005 that CMS was actually successful at getting that mandated nationwide. Why was that? The reason was nobody made the investment and came forward with an approval paps merit screening proficiency testing program. And there was a fair amount of controversy over this when we did implement it. And basically we found ourselves and CMS at the mercy, if you will, of the private sector coming forward with an acceptable and approvable test that met the requirements of the law. And there has been a fair amount of controversy, and ironically we haven't heard a whole lot from the advocacy organizations. I don't think this advisory body took up that issue, but for the first time in history in 200,512,007 technologists and pathologists were individually tested for proficiencies reading tests Pap smears. We think that was a very significant advance and very much needed when we began to look at the results. When we got those results and we found that individuals had by regulation up to four opportunities to pass the test. Approximately 7% of the cytotechnologists passed the test -- fail to pass the test on the first strike or to put it in reverse 93% passed and 7 percent did not. For those who took the test the second time, only 3 percent fail. So overall those results weren't too bad. 7 percent failing is still a problem, it is not an overwhelming one. However, when we look at the failure rate of a pathologist who worked without a sudden attack the failure rate on the first test was 33%. And on the second test it was 29%. It did not go down very much. We think that is a problem. And we are devoting considerable energies to addressing the educational and the testing aspects of this so that in pre performance piece of an improved performance can be achieved going forward. But in the process we had significant opposition to that. In fact one post of Congress passed a bill that would have if it had been passed by the other side has suspended the testing. So I raise this as an example of an area in which there has been a significant problem and has had assertive action on our part to try to rectify the problem. But you can't see the gap between 1938 and a 2005, the gap that persists it because no organization came forward with a statutory the approval proficiency testing for Pat May screening. So we could establish a genetic testing specialty, but it simply does not make those proficiency tests appear. The second burden of any proposed regulation is not just the absolute benefit, but how does the benefit compare against the cost to the benefits exceed cost and to the benefits -- de the benefits outweigh alternative approaches that might be less costly and more effective or faster to address the problem. When we look at this area, the issues are laboratories are already covered by clinical Laboratory Improvement amendments and that includes debt the testing laboratories. There is also some objections we received as we look at this because there are a set of existing specialities and some specialties and the genetic testing would be to be teased out of that. So that was a concern to some people. And we also announced at going through the rule making process is a long endeavor. We first proposed the notice of proposed rulemaking according to the federal administrative procedures Act and have to respond to Casillas and respond to every comment and only then does the final will become published. So we ask ourselves, what can we do that would be faster and what could we do to it may be just as or more effective. But those are the three major test that any proposed rule would have to satisfy. And that is where the genetic testing proposed regulation runs into problems. If you think otherwise we certainly invite your comments and thoughts in evidence with regard to each of those issues. What we have been doing is going back and trying to identify how the existing clear regulations and what can be used as effectively as possible to address any issues that show up. And there are issues. We find some laboratories doing genetic testing that did not think they were subject to Clea. And we have been screening does and identifying those and working with that FDA and CDC to pool our collective surveillance whenever we find a laboratory that does not have a clear certificate or has a clear certificate but not for the to the testing. Then we directed the state survey agencies to get out there and we have numerous examples where we found some of those laboratories and have gone out on-site. If they refuse entry or refuse to apply for a clear certificate we threaten them with the appropriate -- appropriate sanctions and move forward. We also benefited from some of the reconnaissance and discussions that have occurred. The Canucks Policy Center at Johns Hopkins did a survey and we looked at the survey and the survey found that 8 percent of the laboratories that were not doing proficiency testing work also not during the alternative quality-control. The portions of the regulation that she was setting that to apply to a genetic testing is the requirement that every laboratory have a quality system. And other the code of regulations for 93.801 it says that for those tests performed by the laboratory but we're not required to have proficiency testing they must establish and maintain the accuracy of its testing procedures. The responsibility is on the laboratory to make sure that it has appropriate internal controls to insure the accuracy of the testing. Another part of the regulation goes on to set at least twice annually the laboratory must verify the accuracy of any test or procedure that is not subject to proficiency testing. And we can do this through various methods such as sample exchanges with other laboratories. To the extent that tactic testing laboratories are not fulfilling those responsibilities that are required right now on the regulation, we want to identify those laboratories and make sure that they are brought on board and the full compliance with the regulation. The third burden that we must satisfy is even if a regulation is important in its own right, even if it has comparative advantages compared to other regulations, how does it fit the overall scheme of a priority as we look at all the different regulations and are in the process of trying to get out be patient rights regulation for all hospitals, millions and millions of people and struggling to meet the new timeline that Congress has established before all regulation disappears, the three-year limit that Congress established. And so when the department and CMS is looking at all the potential regulatory changes that we could affect that we have been studying and developing the agency has to make a set of prioritization decisions. And when see them as has gone through the process of looking at the evidence and looking at the urgency and looking at the extent to which we might be able to address whatever issues are there through current regulations, then the final decision on the part of the agency was that we would put our efforts into applying and strengthening as much as possible the existing regulations and in contrast to a proposed regulation those actions can be done immediately. So I hope this is useful in terms of some of the logic and the process that CMS engage in. If we didn't think the is -- it is not that we didn't think the issues work on important. We go through the process of looking at evidence and in developing alternative strategies that hopefully can get to the same or better of a destination. And the next slide provides an example of some of the things that we have been doing. We are very open and interested [ indiscernible ] there are quite a few tools in the existing regulation that are quite useful. And I think at this point Judy was going to pick up on the conversation about the direct consumer testing, but let me pause at this point and see if we have any questions related to what I've discovered because I know that this was a topic of great and publicity if not concerned.
Let me to ask from a process point of view -- thank you by the way. Judy, what will you do with the remainder of your time again?
Very little. I'm going to provide an update on the GAO investigation on direct to consumers testing and the suspect customer hearing that took place this summer. And a brief overview of the New York State to edit program under their state law.
Okay. So the New York state law thing, I'm thinking we ought to just deal with the questions on the table and come back to that New York State deal and the GAO deal. The key for that. So my colleagues, I think it is time for you to ask questions. And I think where I am still struggling and as you all start to -- I'm looking for what's to come on and has to go up as you all will make this make sense. This is where I'm caught still here. First of all, there is one thing I think is important. You were very wise to put a dichotomy out there. I don't think this community -- and I know I am not interested in regulation for regulation's sake. I am not interested in having more regulations. That doesn't interest me. What I think we are trying to feel out is, does their need to be regulation, not should you just have more regulation. But I don't think anybody likes regulation. So the question continues to be, what -- with what FDA does there are some laboratories who produce things that are done to the American people it seems that do not have to have that thing, that test go through the FDA process. There are some things that slide -- and I'm asking this rhetorically because I'm trying to a understand both of your comments. They're is a set of tests that slide past FDA and go into -- and deal with people, human beings. Clea people say that we evaluate how good the laboratory it is and evaluate a specialty and you check to see whether there are bugs running around in the room. And that is important. But it doesn't speak that I'm hearing to the same things that Steve does in terms of that test itself. So it seems like what I keep trying to get from you guys is, is there a set of things that slide by both of you. You don't deal with it and you don't deal with it and it still goes to my mother. And what I'm trying -- doubt their is a second order issue here as to whether that is okay. I'm not dealing with whether that is fine and it doesn't matter because nobody gets hurt and it doesn't have -- and there is nobody ever reporting anything bad or it is another order and not the poor folks who are trying to the do this, the bubbly never toward people who are working night and day for $2.22 and can't have any more regulation on top. If they did they would produce another test ever again. So leave them alone. That is a different issue. Is there a set of tests that go to my kids that slide by FDA and/or you guys can give that is all I've got to know. And I just want to get the answer. I have been asking this forever.
I understand and Lord knows you know. I am asking my government.
There are certain things that Clea does, particularly the analytical validity. So are you going to get accurate results does not address the critical validity of, should this test be done in the first place. Is this really the right test for this kind of -- it is just going to focus on whether or not a physician ordered the test. In is the test being done accurately. So there are a lot of issues. This came up heavily in the direct to consumer testing. There are probably five or six different functions you can look at there, the advertising of the test, the sales, the clinical validity the analytic validity the interpretation of results and the interpretation of those results for the consumer. Of all of those functions clear itself is just one, analytic validity. So it is important when you ask that question and we challenge ourselves with regard to the most effective system, it is important that the system be comprehensive. And this means that be all have to work together. And that FDA has emboldened the critical validity for the test kits, but the home brews, I'll let steep speak to that particular question.
Well I think the answer is yes. The corollary answer is maybe it doesn't matter and maybe it matters a lot and maybe sometimes it matters and FDA is the reason. But the answer is yes.
I will get off the soap box and now I am just pure moderate. Deborah was first and we will go around.
I kind of like that. Can I answer your question?
You are up.
Yes, there is a category of test that doesn't go through pre-market or 510K of FDA that are called laboratory developed tests. And make their regulations are weak in the evaluation of those. So Clea as perfume which is my understanding over tests that are currently being done, but that premarket review of tests, if you will, making sure they are okay before you start doing them is not necessarily what Clea test. CHP in response to as ACT P added a whole list of questions for the genetic tests that have to be answered by the inspector. They must look at validation data and cannot be done effectively. Depends upon the quality of the inspector and educating and training of that particular inspector on whether or not they can make an assessment of the delegation process that tests winter. But every two years every test that has been brought on line has to be reviewed by the inspector for genetic tests. So and not the setting to make a genetic test specialty or genetics specialty, I think there are things that are falling through the cracks. That it is possible that through creating a general rules in the cadet that look at test validation for laboratory developed test you can get at this. You are shaking your head. Do you look at the laboratory developed tests and the validation data of those?
In this novel.
Because if CAP added.
To their check list they basically have paralleled the questions that FDA now uses for its evaluation process because I was partly involved and that.
CAP does have a specific checklist that asks questions regarding to lead testing. However Clea always had a requirement for every test, the laboratory must validate the analytic validity. Clea does not cover anything regarding clinical validity. That is the distinction. But Claire it does always ask is it accurate, is it precise what is your reference range for bat test and what is the reportable range for that particular the laboratory. And if it is a new test it has to meet the sensitivity and specificity and we clearly recognize that in some cases our surveyors may not have the expertise to look at that data and to evaluate it. So we have been talking with but CDC and FDA do have graciously agreed that if we collect the stuff on site they will evaluate it to look at it. But it is just the laboratory and get the right answer, not was that test plan to diagnose or any kind of a disease or a malfunction. That is the distinction.
So you could turn this around and say if we had a genetic testing specialty could we all go home? What all the problems be solved? And I don't think so. So we go back to the two parts of the regulations that I can't provide overarching authority and responsibility for laboratories. Title 42 with the code of federal regulations, 4281 a double I, each laboratory must establish and maintain the accuracy of its testing procedures. That applies to the home test as well as the test kits. So even though the FDA may not be approving the home brews, the laboratory is still responsible when it mixes up and produces its home brew. Then it is still responsible for the accuracy. And then when we go on to title 42, 492.1236C at least twice annually the laboratory must verify the accuracy of any test that is not subject to proficiency testing. That also applies to the home brews. So all of the quality control requirements in Clea to apply to the home brews to establish the analytic validity. But again just to stress if we are concerned about whether or not the tests are really measuring the thing that you would hope they are measuring to the clinical validity and that is a different topic.
So not this is terrific. We are going to keep this discussion going. We have three people in the docket. Joe, Emily and Andrea and I will look for other hands.
I does want to reiterate regarding -- I just want to restate that you said something about laboratory developed tests as not being regulated by FDA. But the process of analysis is being regulated by Clea. So if there is any way that the laboratory developed asset be regulated by somebody.
Well, if the essay is not producing accurate results then that falls under the analytical detail area and is subject to Clea. So when we go back to the responsibility of laboratories to establish the accuracy of its testing and we find that a laboratory as inaccurate testing them that is a deficiency under the clinical Laboratory Improvement amendment.
Again, just to tell where we are at here. If I understood your earlier comment it is sounding like we keep cutting through this breed this general provision that says if it isn't specifically noted then you have to just have to the procedures and processes or else we are going to yell at you. And hopefully somebody is actually testing that. The problem this that there is no requirement around the assessment of clinical levity. And that is what you I think are also saying. There is no clinical validity. So it is a terrific test. You have great processes and are very sharp. By the way, the test is applied to the ballot, and that is what I think they are also saying. We have family and then Andrea.
You know, I think we have to accept that this is an imperfect system because if I ask Andrea or dab how long a CAP inspectors spent in your laboratory reviewing your validation data on a new test and I asked Steve Gutman how long the at the A. Spence reviewing an IDD said mission on a needs test and regain that data, I'm willing to bet that the FDA spends more time per test and reduce things in more depth then this inspection system can support. So I think what we have to struggle with is some kind of balance between, is there a system that works for the volume test and brand new test and at what point do we say that a test is so well established that it doesn't I sense any more to have 200 molecular labs each manufacturing their own. And it makes sense to have six or eight companies manufacturing. I don't think because we are in a field where things are emerging all the time that we don't want to get rid of lab developed test and we don't want to get rid of a IDs. But we have to understand there is a continuum and there is also some point when those things need to change over. I don't see any way that we are going to ever bring that developed test up to the bigger of manufacturing developed test.
So as we go to Andrea I want to keep in the minds of people to be thinking about and some people can start to answer, does it matter? Is this much to do about nothing? Maybe that is what we will come up to. If you can't ever get at it and it is such a thorny issue, who cares? Is it a big deal? We will get to that in a moment, but those who no the answer to that should start to help us with that.
I think you for all your presentations. I was taken by part of what he said about the efficiency. The proficiency testing for psychology that took so many years. I look at it a different way. Where now where I got to have my best marathon I feel very confident that if I have technologist at this point that the results will be more accurate or you will have a result. Before that results from this proficiency testing even though it took so many years to come along, we didn't have that. At the same time we have to address issues that we need to go back and continue education out this section of the individual provided. So I think we have learned a lot of neat things about testing being done broadly. It is taking so long to come up with that proficiency testing program -- I agree it is a major issue, but I will also put it that not because it is not easy it should not be done. The second question that I have or comment that I have is we have discussed a lot about processes and quality control and pre analytical and analytical. But I would like to see if you would mind elaborating a little more about the specific requirements for individuals performing a genetic testing. And specifically we inherited these orders. If you are a high complexity Clea certified laboratory director that as microbiology or chemistry and so forth and you decide today to start offering an FDA product for 65, could you do that? That is what all the quality control. Is there any specific personal requirement that would assure them that it would be interpreted with somebody with adequate training that would assure me a valid interpretation with that the clinical context?
Currently the way that we is structured, because there are not specific requirements for genetic testing personnel the laboratory director obviously has the overall responsibility for hiring the right people to do the job. So that is the part of the process. And in that case if you are going to add a test where it takes the very specific expertise to do that, then the laboratory director is responsible for either retaining contract or hiring individuals who can perform and oversee that particular type of testing and be sure the information that is provided to whomever the authorized person has is accurate and reliable. So there is a responsibility there that there may not be a specific requirement, but there is clearly a responsibility because we say Clea personnel requirements are essentially minimal, even though for high complexity for the type of test you are describing we are talking about a physician or a Ph.D. with board certification and a number of years of experience plus specific training. And so with all of those three things place on that individual the responsibility to ensure they hire the right people that not only do the job but to oversee the job so if you need a technical supervisor to help with the interpretation of those results, then that laboratory director is responsible for making sure that that does occur.
And is that good enough? In other words, every doctor that is graduating from school, every professional in the business, you are supposed to do right. Now to read. Is that enough? Does CMS believe that is enough?
I have to say that what we do, we have a very strong practice. Our practice is that if we find quality problems in the laboratory such as this, you have a test and you don't have the appropriate individuals with appropriate training or experience to be able to perform the testing, guess what we do? We said the lab director and that gets their attention. Trust me. It gets their attention because they know that they are ultimately responsible and if not that laboratory can lose its certificate to do testing.
I have an undergrad and genetics. I have been going to the genetics, and haven't had to do this. That is where I am going to.
Clear is a package deal. You saw that it is a series of checks and balances. You must do your quality control at a regular basis to make sure that the test results are correct. You also must have the right people tough match and do the testing. You have to do an audit system, it recordkeeping system to follow the process the route. You must to the proficiency testing such as Thomas describe whether it is a formal proficiency testing or whether it is the informal, but either one is mandated. You must do all of those pieces plus you have to be able to it demonstrate that you are providing accurate and reliable results for all the tests that you are provided, not just your genetic tests because there are a lot of other tests that are very complicated to perform and interpret and you must be able to provide that information. You have to look at this not in the narrow scope but in the broader scope of everything a that can do. Someone said this morning 2,000 test that a laboratory could possibly do. I put 1,000 up there just as a conservative estimate of how many tests on the market that a lab might possibly perform. They can tell me I don't know what is on your menu, how many tasks. It is very Spence of, and so you must be responsible for every one of those and you have to be able to under those circumstances. So it is not just one thing or another that will guarantee that the system will work. It is all those pieces working in concert.
Let me just make sure [ indiscernible ]
[OVERLAPPING SPEAKERS]
Just to make sure because what you just said is important. Can quest get a test to the American people no matter how many they have on there without going through FDA?
Sure.
They can? They get them out without going through FDA? So they are in the same deal.
Can I just add one more, to what I was saying to do it is important to put this in context. I'm not saying every lap is perfect because plea is here. We have the good and the bad and the ugly. Every once in awhile there are things that nobody could have anticipated that we go and say, oh my God what have we been doing all these years. For the most part we had that Bell curve and those labs are doing a good job.
And that continues to come back to the question, does anybody have any data for those who are doing a good job. Has anything that happened to Kim.
[ indiscernible ] in the discussion I have been hearing today I am not sure that even if you were to give an ideal thing of actually doing tests of way you think it should be done that is not sufficient to improve public health and change outcomes. So we are just talking about the first part in what the downstream consequence of the tests are, whether they show control contests or there is a arms and beneficial. The treatment and how we balance that has everyone improved on the public health. So I think is well for us to get a better handle -- handle on during the test well. I haven't heard any discussion about clinical utility or the outcomes of testing and that hasn't been a part of anything I heard from CMS or FDA. So I'm not sure that even if we were to get it done perfectly by the labs it would solve the problem.
Grade. I understood what he said and that makes sense. I didn't think we had to go. I would have jumped her and line. They are trying to job and see if they can catch her. Other questions from the committee that will help out here. I guess that me to ask the community if you don't have questions, to yell feel like you understand any of this so you are prepared to make some recommendations here?
I am confused about one thing. Is one of the problems that create inspectors don't have the expertise to evaluate the in house developed just to give is that a problem?
As a solution it has an identifiable solution. We didn't really get to talk about the best of things that we are planning on doing. And one of those is to probably do something creative as far as looking at test. Because we realized we may not and because most of the labs that we look at are not the type of clubs that will do this type of sophisticated testing. So we put either create a corps of folks who would go round and develop the expertise and train them and get the technical and investigative Training in place, and they could go around and look at all the laboratories in the country, rather than the local surveyors. Or we could have a contract with someone who already does that and already possesses that expertise and just pay them. So there are a number of ways we can get at this. I don't think that is -- I would take that as an irresolvable problem the get is is something that can be addressed and something we have already been evaluating. Actually way back when in 1997 when the recommendations were made for the task force we knew we probably would need to look at something because the technology was exploding and the would be able to keep up with it in house.
Related to the lack of the specialty of these genetic testing?
I think at all. Now, it is not. It is assisted technology but that will happen any place because you will have my career race or other types of technology that will come into place that we can't even anticipate at this point in time and will be in the same position. But don't also forget that those folks that we trained, we trained with a very detailed specific investigative skills. How to collect information and how to add to the people and what to look at. It doesn't matter what kind of technology they are using, as well compliant -- find the problem if a problem exists. They know what to ask them where to look. They will have the lab explain the technology if they don't understand it. So that isn't a limitation. It is actually an advantage.
Another way to get around this issue of CMS and the specialty is that -- CDC is here? It is my and the Standing that Cleo is actually going to look at these specific issues on the genetic specialties. Is that correct?
I can't speak to that. I'm not involved with Clea.
Okay.
It is clear actually which is the advisory T CTC and CMS will look at these specific issues and maybe we can wait for them to see what they recommend. Now the other thing we can do is they got to that report have somebody from that group come back to this community and report on what their recommendations are. And then we see what that group which are most of the laboratories actually feel.
Would you hold that recommendation for just a minute and the researchers said when we get to what we want anything to do as a community. I think you read something that is tangible. Let me ask men to finish the part which had some important stuff in terms of what New York is doing. And one of the things I think that to be emphasized will hear this certainly getting my attention and this whole discussion and it came up with the sample also with quest. And that is, how many of these tests are rolling down the road to and so if you put every single test through a pretty serious trouble that might be almost impossible regulatory burden. So that is important to think about and I am thinking about it and try to figure out what that means. And now New York has done some stuff that gives us a real case scenario for some of this. Can you share with us the New York bank to give.
The office is the investigative arm of congress. They were requested to look at director of consumer testing, and also to a follow up on the status of the proposed rule at CMS. And just to get back with you, they did identify a number of laboratories that were providing the true economic testing, evaluation of lifestyle. And as Thomas indicated earlier most of the issues surrounding that are not pleased issues. We do not deal with advertising and marketing and chemical validity and interpret it utility of results by a provider. But we are responsible for oversight of laboratories. That is if the test is covered. In this case some of the Senate for economic tests as they are called, evaluate lifestyle. They look at your diet and your nutrition and whether you smoke. They look at how much you sleep at all those kinds of things. Obviously it is only if it is a health assessment it is covered by clip. And thereby it would be subject to clear requirements. In this case these are clearly laboratory developed tests. Just me. And so in that case we are closely monitoring. I think it is important for you to know that the labs that have been identified, there are a whole pile of others because once you find one that are like little bunnies, they multiplied because they are all associated with one another. They are all in cahoots with one another. And so you find one and there is another and one is marketing for the other and one is during the test and one is interpreting the test. One is in London and one is in Denver, same lab. So you just have to unweave the Web constantly to get it by these facilities and we are doing that. We are working very closely with the CDC and with the FDA of this process. But let's go to the New York program because it is probably the ultimate and oversight of genetic testing, I have to say. Clearly it is -- they are over all in New York the most stringent state laboratory standards and the United States. They also have a wonderful infrastructure to share. And it is the difference between the economy and CMS where we are well funded and a laboratory program that has very high fees, very good resources and revenues to support it. Under this program there are two types of tests, FDA approved and everything else. The research only, the investigative use, the in-house developed a up ARS and so forth. In those cases those tests must be approved by the laboratory program before the lab can offer the test. Currently Steve, you are correct. About 12 tests are proved. So everything else under the sun that is a genetic test has not been hampered by the FDA. They're is a typo on this, they'd do it to actually they have done up to a foreign entity reduce over all, not just this year. But the review includes both analytic and political liberty. They also provide the laboratory data is on the information they need to provide in order to do this review. But there is an interesting factor here and that all the reference laboratories and the country probably have a site somewhere in the state of New York because if you understand the New York program it has pedicles because any testing on any New York resident regardless of where it takes place is covered under the New York state law and thereby must submit their test to the state in order to be approved. So if you extrapolate that considering that all the major reference laboratories are there and all of the -- and the major facilities that are there, it is estimated that probably 75 percent of the genetic testing in the United States is already such technique York State over said. The program in New York is -- and there is a representative here from the program. Says she will be happy to jump up if I say something off. And she has provided all this information. So I didn't make it out. There is broken at the segments. There is such methods and all of this information, political but patient consent and confidentiality and specimen retention turn around time and very prescriptive and very detailed requirements are for the information that needs to be submitted. In addition, there are requirements for reports to be signed by a set of geneticists and that there be an interpretation suitable for a not geneticist was is just fantastic. Also, prenatal and pre implantation outcome verification, we are even looking at utility here. And of course the lab is subject to the New York State PT Program. It is the same thing for genetic testing. There have very similar requirements as you can see. There are very detailed. There is, an analytical validity and confidentiality and so forth. In this case they have the same thing that we does essentially because where there is not peaty material available, particularly for rare diseases in the laboratory is subject to external PT if there is BT available or the twice per year just as unclear. Let me go back to that. It is also interesting to note that even that there is a very detailed definition of genetic tests under the state law, there are also tests that are disbursed to the laboratories just as unclear. There are some tests and microbiology, immunology and pathology. So not everything is collected into the one area. And that is basically a summary. This affects about a hundred and 70 facilities. The said Genetics piece has been in place since 1972 and the genetic peace since 1990.
Thank you very hot. All right. Well I first of all want to really think Steve, Tom, and Thomas and Judith for not only their presentations but for putting up with much pain in the neck questions. And I really appreciate the way you have handled all of it. As the community now tries to think about this as to what you think, any next steps that you may decide or make just let it lay here, I wish I had never brought this than not. There are some things that it seems to me that he take for granted. When it comes to clinical interventions, whether they be diagnostic or therapeutic, one Assams that there is a government that is appropriately looking out for your interest. And you to sort of take that for granted. I do hope that that is the case here. And mentee it is. I also know that you sure don't want to be piling on me this regulation on the backs of Industry that would therefore resolved and extra health care costs that deny access to the very life saving diagnostics and their peaks that you started out with in the first place. And you definitely don't want to pile on a bunch of regulations on to the laboratory in an academic center to barely gets an indirect cost permission from their team to keep the lights on. So you darn sure don't want to do that. So somewhere in the middle of all of this is a right answer. And I'm trying to make sure that there is an ethical obligation that this community has for having even raised it for our predecessor for having raised it -- shame on them except I was on that community as well. Once you raise it you better doggone well deal with it. Because it is at the end of the day that means that people are getting -- or their health could be compromised because of a lack of vigilance. That is a pretty bad thing to have happen and just decide it is too complicated or wherever and just walk away from. So I really regret ever having asked the question. So with that, you need to figure out what you want to do. And also how you want to do it and you need to determine first here, is there a problem? Is the problem significant? Do you know enough to answer either of those questions, if you don't know then what do you want to do if anything to find out the answer to, is there a problem or if the problem this significant. And I look now for guidance. Thank you. I think it would be a great place to start to answer the question, many what the response has been and the fact of the New York law on industry and consumers.
And if I could amend that to what is it that prompted to your to act in the first place?
Right.
I am a doctor and Billy I am the director of policy for the research center which is the public health lab in the state of New York. I have been responsible for the subtle genetics oversight program since 1979 and for the implementation of the tax program in 1990. What prompted the York State is a New York state statute that dates to 1964 and predates Cleon 1967 which requires the state of New York to oversee the practice of Laboratory Medicine for the testing of all specimens derived from the human body for all purposes. So when cytogenetics, the examination of the human chromosomes became a practice of Laboratory Medicine it was an area that required a percent. When the biochemical genetics at a phase where enzymes or PK you were DNA markers for genetic became the practice of Laboratory Medicine, it was required that we establish appropriate good science based standards for laboratory practice. The statute requires that all tests performed by a primitive laboratory must be either generally accepted by definition, FDA cleared is an in vitro diagnostic device and approved by the department. That demand -- me technically we could say that to a test that the FDA said yesterday. I don't know that we have ever done that, but for any test that is not cleared by the FDA. So it is not to limit availability of this general practice of lab based test development. We had to have some basis by which we but approve those tests that didn't go to the FDA. Our process for validation Review of non FDA cleared tests is not unique to genetics. It applies to any laboratory test. Could be clinical chemistry or it could be microbiology or virology or genetics. That is only one area. The Standard but the but the jury has to submit adequate data for Genetics is usually very small numbers of cases. So that will be [ indiscernible ]. And their validation and their critical validity, there must be some nonclassical Association with the genetic marker. So for example a lab that says they can find snaps analytical validity is absolute and can't find every step in the gym until now and can print out the CDs and stamp profiles $4,000. If they cannot establish clinical outcomes associated disease state for each step, they cannot offer that snip and New York state. What is the response? Consumers are not well informed about how we oversee laboratory tests. Physicians ought only slightly better informed. Many of them are told where to order their test by the insurance that is paying for the services for the patient. So the laboratories themselves, 95 percent or better of all Lapps are good labs. I think it was Steve it said if you don't know that the test you are offering provides good results, what are you doing offering the test? And 95 percent or better, that will be the case. If they forgot to submit their validation package hopefully it is really already on the shelves and they can correct the deficiency within a matter of days. They send us the package and we review it. We are slow. They're ought only to technical persons on staff who actually review all of the submissions. I review all of the said its index. And these that require individual review, all of the genetic testing is reviewed by another individual who is a to assist. We get behind -- and that is the biggest deduction from industry. It takes time. They have done work and we have to do the review. It is also an educational process. We would like to streamline it and see every application come to us with the table of contents and everything on the same page. That is not the way it happens. But it works. In the meantime if a laboratory had a critical comical need to offer a test for a clinical referral reason, we will issue what we call a non permit Lab approval. So that patient for that purpose for that time only. And we tell us that when they get to 50 requests we began to get really unhappy. In our surveyors do not attempt to review the technical data at the time of service. They go in and ask the lapse what test to offer, if it is a home protest that is not FDA-approved with the package insert that says it is an IDD. They ask to see the letter of approval from the Department of health. If a lead cannot produce the letter of approval in the that is cited for offering a test without approval and if the deficiency is corrected by submission of the delegation did the. There are bad out there offering clinicallclinicall why useless tests that are having dire consequences for fetuses and patients.
I have been waiting for three hours to hear somebody say that. Now by the way, why would -- so -- first of all, thank God for what you all are doing in New York. It does a lab -- and given what Judy told us so clearly about the interconnections between New York and the rest of the country, with a lap have to submit to you and then to other -- I mean wouldn't this be burdensome for a lab to have to jump through your group as well as the Clio hoops and the FDA hopes?
New York state is Clea exam to for those lab is located in New York State by beating New York state requirements. They do not have to meet clean-air requirements. Four laps outside New York State they will get their Clea certificate from their local state entity. However to my knowledge most of these labs -- nobody else is repairing the actual validation data. Cap may be at the time of survey on site. We would not discourage that, and they may find things that we don't find. The more times you look the more helpful comments and constructive criticisms we can offer. Right now we are not in competition because nobody else is doing it.
How much money is --
I'm sorry. Cynthia had her hand up. Mentee, you had the floor and in some deal is next.
I did, thank you Paramount. I'm just wondering, what do you do about these letters that are offering test?
For New York State we simply tell them that must cease and desist and if they continue we touch them to a thousand dollars per day.
Are they operating in other states even if --
New York state has no jurisdiction in any other state.
Right.
Okay. I am an attorney as well as a geneticist. We have Santayana and Emily.
What I think I heard was that there was not a particular problem that prompted this. This was a statute that existed a long time ago and it wasn't specific to kinetics or [ indiscernible ] or any of that. And so because of the regulatory scheme in New York State to the six fell into it if I am interpreting.
Well there was a terrible problem or if he said the same assessment to clinical labs you get two different answers. The hypothesis is the same potential problem in every area that has clinical laboratory medicine if you don't put appropriate oversight in place.
And so what I'm wondering is maybe there are things that New York does that could be done at the federal level with some tweaking of whatever. But I'm not totally convinced that genetics is unique and that whatever these problems are they could apply to many tests and many labs. And so I'm struggling a the a bet with why is it we need to focus on the genetics component for Clea or whatever because it seems to me there are other tests or things that could fall through the crack.
Can we ask you specifically, given that you are going there. In your it pending with your experience and we appreciate your stepping up and helping us here. Is there a special need? Week raffled with this and it is on our list of activities for all those things we are supposed to be doing. Is there a need for conceptualism or is this just diagnostic laboratory medicine.
I have made geneticist but I have a personal bias. I believe this is good for all Laboratory Medicine and the success of the New York state program is because it does not create genetics differently. It makes no exception for genetics. But it simply holds it to the same standards.
Emily and then Andrea.
I have a question regarding FDA cleared tests. Once they are cleared, is there any incentive for New York State license laps to switch over to a crude is it over them the fact that they don't have to create a dossier for you to give.
It is of concern. We believe is some of the vendors. If they get to the trouble of producing an ITT and getting clearance this is true the genetics. I can't speak to other areas. But a lab will use the FDA cleared IDD as their gold standard against which they will validate their in-house developed [ indiscernible ] perhaps because the in house is cheaper. So yes there would still be an incentive for a map to do a home developed as a if they thought it was cheaper than the IB T. But the state doesn't have any mandate that says you have to it.
A [OVERLAPPING SPEAKERS]
You have to use it.
But it is probabilistic if there is a cassette. That is the standard against which we would expect the lead to validate. That may put them in a high standard that the board be able to me.
Okay. I had a question for Judy. I actually was shot up to just to look at this molecular surveillance. In a tuxedo to stability in East and art full process controls, ought there any plans to work with CAPE to develop full process control to fortunate 62.
Specifically know.
What do you mean by that?
Well, it doesn't go through the isolation purification part like a clinical sample would. It comes as a purified to Numic DNA. For the proficiency test.
There are major issues of how to manufacture us the support.
I understand that. ITT didn't understand how it medically is required.
Actually because that PT is not approved by a CMS. The only peaty we look at when we approved proficiency testing providers are for the economic and lives that I mentioned that are in the regulation. Everything else is voluntary for the never choice to perform or in the case of certain accredited organizations where they require every analyst have peaty, formal PT. One of the difficulties again is the fact of the shortness of materials. There are not materials available that are easily transportable or in the case of a where -- rare disease where there are so few materials that it is impossible to create P T believe we are used to seeing traditional PT. So there augmentations to what can be done with that. And I think that is part of the process.
And that is the problem you have with Scientology as well. That is one of the EC's.
It was early on and was one of the problems we had, that there were not enough available.
That was significant effort from the CTC to develop control for materials. It is for a significant amount. Many debt could be a venue for materials that could be used enough to have enforcement. Estrada believe there has to be some enforcement for our laboratory.
You are absolutely right and there has been major efforts by CTC and that area but NPT and D.C. for genetic tests. And we had been working with the CTC to support those efforts. People -- we are paying for most of them anyway. But it is important to know that in addition, to working with them we hope to develop educational materials for genetic testing laboratories that would encourage the use of those types of materials. So we would get at it from that direction. We really didn't go through my list of detail, but that is one of the things we talked about.
A question for the new lady.
He mentioned that he'd have FDA after the product "when the larger is bring them on my the don't have to select for these reviews or the that by using it. He's an SRE still to a mature adult process and have to submit for the review by the New York State Health department. Is that correct?
I'm sorry. I didn't hear the second part.
When you use an ASR --
Yes, and he is an ASR there is a component of an in-house developed asset. And we greatly appreciate the FE any recent clarification but when you buy a single component you are buying an ASR. So when you put them together you Arkin -- your control program targets probe and make up an asset, then alt in house developments using a as RS required departmental approval whether it is genetics or microbiology.
We have gone back and forth. About be within clear there is no need to show clinical validity for the testing we bring. This is not just for genetic testing. If this were all laboratories. So we are trying to fit 9 feet into six. And this is at the core. Who does the oversight Board New York State have or other issues. The problem that currently the regulation does nothing for demonstration of political validity. The CP molecular pathology checklist as significant about how questions put into that program to deal with this issue. It is two days or three days like last month became with an unspoken [ indiscernible ] there is a different issue. And again these are your peers looking at what you are bringing on line. But it would be that issue that there is none in the Clea regulation for the critical levity.
And I think that we all get what that means. Is to discuss open how there could be or there are in some cases people who are purchasing a test people cheered -- people are paying that may not work. It may not tell you any information that would be useful or it could be that a commission is using that information to make a diagnosis that leads to a therapeutic interventintervent ion that may not be appropriate. And that is where the? , the importance of the? Arises. If I understand the discussion.
And discernible.
The reason New York is exempt from Clea is it already had a program according to Clea and with the second part of that question, this is hypothetically a dangerous question to answer. Supposing another stiff comes up with another program similar to that New York, but it also become potentially exempt from clear teetoo.
States have to apply in order to become exam to, and then we do an excruciating review of their standards to ensure that they actually are at least the equivalent. They could be more stringent, but they must be at least equal wants to clean up. Please understand that we are talking about opposites here. This is a continuum of laboratory oversight and this is one end of the spectrum, and we are the other. Clea has put in place minimal standards for Laboratory Quality with the certain death again that the lab director as that overall responsibility and that should pick up where it needs to in the event that they can. And the laboratory as a choice about who is going to inspect them under Clea. They have the choice of either the state Department of Health who uses the Clea standard or an approved accrediting organization, many of you also have stringent standards. Because the equivalent doesn't stay added. That is the way the program was developed grade it was told also that there would essentially dovetail into FDA because they didn't want to petition of effort. Clear it would look at the answer and FDA would decide if it was a useful test. And so it makes sense if you put all the pieces together.
All right. Here we are. Fifteen minutes left. Doctor, you are terrific and I test can't thank you enough.
Can I add one market to the ranch?
First of all, what you get to do is add one monkey to the thing, but what you also get to do is to start so formulate the beginning of a consensus of what if anything we wanted to. You get to do two things.
[ indiscernible ]
After the, this morning there are a number of [ indiscernible ]. We reviewed a lot of particles that actually are clinical trial protocols and where some of the cultural particles propose to do, for example, pharmacogentics testing of individuals who are placed in a specific arm of the clinical trial. A certain drugs are given to that patient. In talking to my members and sank to is doing the testing for these clinical trials? And nobody looked at me. And I started bringing the issue of research testing that is being used to make decisions on how a patient is treated in clinical trials. And my understanding is this this in the clear per view. But it seems to be that these banks connect between what actually I art be in different areas of the country are as knowledgeable about these specific issues. So one of the [ indiscernible ] is also the research laboratories responsibility to regulate testing.
Let me try this, thank you by the way. Let me give you an option to issue that because I'm looking for somebody smarter than me to say it and figure it out. What I'm thinking is, is that we say to the secretary in a letter that we have a chance to discuss this and learn a lot about that and that we have certain principles that we have come to understand , principal number one is that the public should be protected -- should be protected and there should be over said. Not even protected, and that's negative. The public should expect that there is appropriate oversight and place for diagnostic and corporate interventions that they receive. Member to come ahead -- number two, this transcends kinetics, but because our focus, our mission is on genetics we recognize that Genetics fits into the larger continuum of critical care and we come at this from the point of view of specifically looking at the genetics. Number three, that as our principal understanding that in an -- inappropriate regulations to no good and we have a report that is out around our concern about access to kinetic technology that could be compromised by unnecessary burden some rules and regulations that are not helpful. But that as a principal so weakly get on record as not looking to be a lot of irresponsible not been running around talking about regulating everybody and everything. Number four, that we heard a good testimony has all agencies that leaves this community with some questions as to the adequacy of oversight of a genetic testing. And finally, because of the implications -- the potential implications of a lack of adequate oversight on the side of the public that it would behoove him to bring his people together, FDA, CDC, CMS and they should report to him and give him an answer. That is as to whether or not there is a significant problem and what the remedies might be to be able to address. And hand him the transcripts of our testimony as record of all we have gone through and simply say, this is on you. This is what you get paid to do and all these smart people and government get paid to do and the FDA, that is all they exist for ankle yet, that is all they exist for and the CDC exists in part for it. Figure it out. Altus put that up there for you to shoot at.
Has that already been done to the extent that -- Thomas, you went through the laundry list or the process or approach that you take when you analyze, is there a problem, and if so is the proposed solution necessary to correct that problem. The whole thing he outlined for us. Was that done just at CMS? Was that done with input from FDA and others? I'm wondering if we have already done what you just kind of outline.
Again, we aren't looking at the entirety of a genetic testing. We were looking at the question of the proposed rate that would establish a genetic testing specialty. So it wasn't our focus within CMS -- we obviously discussed it with a CDC and the FDA, but our current process was with an CMS.
I'm just wondering if we could be more specific in the letter to try to the give the secretary a bit more concrete focus and mention the fact that in this process which you mentioned with these principles that you mentioned one of the things that came up that Andrea also mentioned, this fact that there is a difference between a plan like York's and what is currently federal regulation, and it focuses around clinical utility. And so the question is to say about that Federal has to be the same as New York, but as we heard the York is one end of the spectrum. Should we move more in that direction and if so then how? And to what extent? So I would say you can break it down into fairly specific sorts of issues.
I would agree with post you and Kevin, but also take into account, I think, what has been said might as -- it is simple. Hopefully it seems like that issue is broader and bigger than just our community as a whole. And I guess what we can do in terms of the strongest thing we can do this year what is the bigger issue of clarity and then CN determine based upon even listed presented and also I think because there is the community coming up to say this is our piece to build a bigger solution. And that is the most we can do. That would be the thought.
Thank you.
But we heard earlier from CMS genetic testing is currently regulated under Clea. They think the specialty will produce a more stringent or define the criteria for this testing. But I think maybe letting Clea come up with their recommendations specific if there is a need to increase oversight of to a testing by the creation of a specialty or specialty within Clea might not be a bad idea and just wait for that group to look at these and report back to us. That is before we make --
[OVERLAPPING SPEAKERS]
Under, one quick question, and that process will even the issue of clinical utility ever come up? Because it wouldn't normally, right? Even when the new specialty. Right.
Your guess is it will come up? So this will be a first? Do you have critical utility --
It has been coming up and that the new also.
Andrea, I think first of all the committees to do what the consensus is and I think we must keep raising consensus. What my concern is, and I think first of all we have gotten, well meaning and get testimony from but Steve, Thomas and Judith and clearly they are honorable people serving the public's interest. At the end of the day the natural tendency inevitably for people and government agencies has to be to tell us that things are moving along and doing fine. I think we need to put it on the secretary and ask him in the quiet of his all deliberations whether or not this is going as it should because I think there are things that can be explored in private that can't be explored as well in public. That is just a bias.
In discernible.
I just think whenever we send to the secretary should more clearly defined what the problem is because we are hearing that 75 percent of the labs are regulated by New York state. We also know that health plans to a large extent tell doctors which labs to use so that something is reimbursed and cats are regulated by FDA. Is that correct? So if some consumer just on his own consulting -- so he is protected -- he or she is protected to a certain extent. So I want to hone in on, what is the actual problem? To is the unprotected and what are the problems? What are the gaps? And I am not certain I have a good understanding.
Can I make one technical -- 75% of the testing is regulated by New York state. That is because of the very large commercial labs all being regulated by York. The reality is the majority of tests are still bound by those large labs, not 75 percent of the labs.
And by the way, you may want to -- Cindy, again the committee in this section of the briefing book on page eight there is a set of one, two, three, four identified gaps which are part of it. But I think -- so the challenge we have based on what Cindy has said -- and I was sort of expecting that is where we also are here. There are still despite the information we have gotten, still some and clarity's on the basic data base in the background. So Cindy, you could -- let me to ask you your preference here, Cindy, based on what he said. You could postpone writing a letter to the secretary and try to discover some more of these things and make it more clear what you want the right in terms of the problem and the issue and we could come back at it and work on this in the interim between the meeting and finalize it at the next meeting or you could just simply say we have all this stuff and are on certain and urge you to take a look at it. So it is really a matter of sequencing as to how you think you may want to proceed.
I have read the briefing book. I just come to this discussion don't know precisely all of these problems, what the scope of these problems are, is there a universal agreement, are there others that weren't mentioned here. I don't have any objection to if people know what that is -- and this is my expertise. There are others here who deal with this on a daily basis to would be able to articulate what better than I what the problems are if there are truly concrete issues and problems and gaps. I to think we shouldn't fight it in a letter, and I don't object to doing the letter right away if we are able to put our arms around the nature of the problem immediately. If not then I would suggest that we postpone the letter until we can get that bill down.
Other points of view as we try to determine what your wishes are in this regard?
Just a quick question -- and again if you would help me to clarify this a little. Perhaps we can look at it this way. Rather than listing all the specific problems there might be my question would be, is there an approach or solution that would pretty much address all that list of problems, like critical utility.
I don't think there is one solution, but I think there are many actions. One is graded use of the FDA and one is changing clear authority for all testing to include political validity, heaven forbid. One is something specific for genetics. There isn't one solution.
I'm not saying there is. NBI didn't phrase that properly. What I'm asking is -- and there are a whole list of things that may be wrong. Is there a solution, not want a solution, a solution or maybe several different solutions all of which as you mentioned could address the list of problems. Is the list of problems I tried to get that. Do we need to list all the problems or are there a set of solutions that would pretty much take care of all the problems? Whether it be one in the SAT or many.
I don't have the best.
Okay.
I guess I would want to I know how rigorous it is to determine the accuracy of the test and then what is in terms of to the patient how accurate the tests are in terms of their percentage of accuracy and how certain we can be of how accurate that assessment is.
Accuracy of the test is a very specific scientific definition that is not hard to establish. That is if you have a specimen that has a target. How often do you find the target or how often do you get a negative answer when you should have got positive and possibly he should have gone negative. I don't think that is the accuracy you mean. I think you mean the predictive value and the particular clinical circumstance of that patient.
Marty, is that what he meant?
In a laboratory I can tell you that side of genetics laboratories their accuracy rate is 99.9%. One out of 1,000 case will get the wrong answer. That doesn't mean much to a patient where the productive value depends on why the test was ordered by that position in that circumstance. And I the laboratory can't tell you that without knowing exactly the comical resources of the physician or the test. --
In New York State we ask laboratories to confirm prenatal and pre implantations genetic diagnoses results for all provinces. The only way that lab can do quality assurance on outcomes. But that doesn't give them predictive value. Again and the comical circumstance it gives them test accuracy.
Here is a hypothetical, number of pregnant women are told they have Down syndrome, their top has Down's syndrome. They give birth and then there is a determination that how many of those were actually accurate. 40 percent of the children did not actually have done some, can you then conclude that perhaps that test is 40 percent inaccurate or does that give you reason to --
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It would give you analytical accuracy. It would be how you would establish analytical accuracy. That is how you would establish predictive value. There are two different measures.
By the way, Steve, would you remind us again of this post marketing survey -- surveillance that we are getting at here? Who handles that again just so we have the right information.
Well, I can't state for New York state. There is obviously an entire program being reenergize. Those were put out last week about the new contours of that program. We are sort of responding to the issues and trucks which is that New York State and not CMS and not FDA looking at a pre market package can't predict the picture of when to extrapolate into the huge numbers of youth. So it is always an estimate. And the reason we want to attract is just to see the truth. We are back to decisions.
The decision is between you standing in the body being picked up to go to dinner at 7:00 -- and if you are late, God help you because you won't eat. Cynthia has -- great, Phyllis, save us.
It is dinner, but I do have a third option. Somewhere between a letter to the Secretary saying there is an issue that he needs to get his smart people working together on between during what sounds a bit more like an initial report really hiding the issues that are at stake here would be to read a letter. But include in it an illustrative example that highlights a gap that an individual could fall into. It seems to me that might be a way of getting a busy person to notice that there is a real personal element here that he can make it effective.
That is actually very [ indiscernible ] recommendation. If the community were to adopt that you would have to have a few people who would volunteer to a sort of put that together and then we would float that by the full community in time to send that forward. What you have done because obviously you are an active listener, you have summarized the three options better than the chair person ever could. So community, which of those three options that have been laid out do you enjoy first? Which of those seem to make sense? Phyllis, start with the first one. We are going to raise your hands for those that like one, two and three.
The first one is suggesting that the Secretary get his hands together and try to address the problem that we superficially highlighted.
And the second one was?
Ted to a it short report somewhat to option one but to include a case study, an example if you will highlighting an example that would illustrate the gap we have discussed today -- A gap we have discussed say.
That was the second one -- that was the third one.
No, that was the --
Here we go again. I am an active listener to. The first one was to send a letter to the Secretary send it to our people together and figure this out. Number two was doing something that is essentially a report, what we put together and really go through, some of the things that were applied by send each very good recommendation. And the third is to send a letter signed, but he indicate in there an example of somebody falling through the cracks because you want to illustrate what is going on.
Fourth, to wait until the cleanup report of the specific issue of genetic specialty and see what clinical Laboratory Improvement advisory community has to say.
Now you are assuming it would be for the clippers to me that whenever they come up with is actually the solution to the problem. Give that or you are saying it will help inform your situation.
So we will do it in reverse order. All of those -- I'm not good accounting, but.
One point of clarification, a sense of when the input from it would be forthcoming.
That is the right question to ask.
And discernible.
So we would build that into --
One could also take one of the three after the Clea recommendations.
Gut, I love you all. Here we go.
This is sense of the community including the [ indiscernible ]. So we are going to let everyone because you have all worked hard and deserve the right to vote -- so we will ask you with a sense of the community, you raise your hand. Number one, we don't do diddly-squat until February after the Clea folks meet. How many of you all like that idea? So we have one, two, three, four, five. I love this. Okay. The next one is -- I just want to do them out of order. The next one is you are going to send a letter to the Secretary today and say get people to get there and figure it out knowing that the cleanup people are meeting in February. Who wants that one? All right. That is out. One. Good for you, courage of your conviction. The next one is --
[ indiscernible ]
Rate. So letter possum area. He said that? To 3:00. One, two, three, four, five. Don't give me a tie.
You know you really should got my vote also for the ledger plus the example because the key here is the ledger. And maybe I shouldn't vote because of where I am, but I would count my vote there.
So he is with the last one.
Note to give.
No.
A [OVERLAPPING SPEAKERS]
Take one.
Mind is wait for Clea but start working of the ledger with examples so we can get it.
This is fine. This is great. We are almost done. You have two choices, it is all down teetoo.
[ indiscernible ]
Wait for Khalil while working on the ledger with examples in and out right away.
So here we have a compromise. Our people excited by that? Those that did not vote to wait for Clea, would he be happy to jump on the bandwagon of wait for Clea and start working the report? That sort of makes everybody happy. Who is upset with that?
I'm just wondering if getting the letter out that might help influence the Clea meeting. And that is why am wondering if not getting at the letter now might be the idea precooks so we have two hands up. But we have an interesting point of view here which is nice. This is simple. We can do this. Do you send out the letter now and therefore urges up the tension around the Clea deal?
My very humble advice, you all have identified clearly a very important issue and a potential gap. And whatever form you all feel comfortable, get a letter out. Get a letter out, in addition, to that is wonderful. But don't lose the momentum and don't wait for the perfect thing. Get a letter out and let or continue.
And is there anything that Clea might give us that will materially alter what we want to say or get a curveball?
So I'm going to be built. Let me phrase the consensus on options. Number one, get a letter out and get it to the Secretary so it can be started working. In your letter others to the fact that Quebec is by Debbie meeting in February among other things that are relevant for the secretary's staff to review. Option to is wait and so clear yet beats and February and simultaneous to that continue to gather information and facts find and so forth such that by the time you come back after the February quick thing he have some work done. Now, we are calling to show hands again. Option one, I want to see what you feel for sending a letter to the secretary now that tells him that there is an area of concern and among other things in that letter noting Clea meeting and if at Yuri. How many wanted to that strategy? And we have one, too, three, four, five, six, seven, eight. The alternative, how many for wait until cleome and simultaneously began to do some fact-finding? And we have one, to -- one, two, three, four, five, six. So eight to six. At me ask the out liars, would he be terribly -- terribly unhappy with doing it the other way? Are you violently opposed? Yes?
I would be imposed if we were just going to send a letter to it just stayed on the issue and really doesn't clearly and identify the specific problems, not only added by the problems, but how or genetic technologies differ from all complex tests. And I feel we read to -- need to really nail that down and if we can I don't object to the letter, but I am sure we are there yet.
Good. How about we do this then, Joseph, are you in that ballpark?
Eye in this ballpark because I agree we need to have as clear and strong of an evidence information.
All right. What I would like is volunteers for the letter writing effort who will put something together and work, not as a permanent Subcommittee, but just a single issue, not a bureaucratic subcommittee to put something together which we will circulate to our colleagues with the exhortation that people have sent this letter to the Secretary on or before the January 1. So are there some people who would like to help the draft such a letter with the sandy Barry modification? Will work on it they the bad. Who would like to help on of that other than me to give.
I can't take the lead, but I can review whatever you like.
We get Andrea. Scott is an. Sandy is on it. Said the rights well.
You all will be part of the review.
And did a and b? And is gone. I think we might want to consult with an. We will certainly consult with FDA and CMS of that as well. We have a community. That me to say first of all again Judy, Thomas, Steven, we go you a debt, especially putting up with the terrible people. So you are terrific. Committees, good work. We are in the lobby at 7:00 and feel bad about the fact that you guys can reach consensus about such complex issues. The hotel lobby, hotel lobby. 7:00. It 30 tomorrow, we start at 830 tomorrow. Pay no attention to the man behind the curtain. Leave your things here. No, they are making new tables. Don't leave your things here. Take them with you.