Transcripts For CSPAN3 Politics Public Policy Today 20140919

People dont know that. Theres no statues of these gangsters. This was the epicenter of 1930s era crime. J. Edgar hoover had this building as their headquarters. Its also the building where those bootlegers were tried, sent to alcatraz and other prisons across america. Its where it began and where it ended. Were standing here at this historic fort, looking over the junction of the mississippi river. St. Paul is located up the river from ft. Snelling. Its intimately constructed. Settlers living on the militarys property. Finally the army had had enough of competing with them for resources and felt they should be removed officially from the military property. Settlers then moved across the river to the other side and farmed what became the nucleus of the city of st. Paul. When you think about the story, the history of this region, you think beyond the walls of ft. Snelling. And thats what we try to do here at ft. Snelling, push people to think more about what does it mean when all these cultures came together . What perspectives did they have on these Historic Events . Watch all of our events from st. Paul saturday at noon eastern on cspan book tv and American History tv on cspan 3. Now, hhs secretary sill via burwell, dr. Francis collins and fda commissioner dr. Margaret hamburg talk about ongoing efforts in the medical community to improve and develop new treatments and cures. They talk to the house energy and subcommittee on help about innovations they see on the horizon. This is about two hours. Again, well get started. Good morning, everybody. Today, this morning, were going to continue our really very strong bipartisan efforts on the 21st Century Cures Initiative with this round table to discuss ideas that the committee has received the last couple of months and look to more specifics about the steps that we can take to accelerate the cycle of cures, embrace Technology Advances and the rise of personalized medicine and keep america, in fact, Innovation Capital of the world. Over august and early this month, our Committee Members, including degette, pitts, and johnson, heard how its providing hope, saving lives and, yes, creating jobs. The 21st Century Cures Initiative affects every american, every one, as they, their families or friends have been affected by disease. Our Committee Members heard during august, hard work remains to find and deliver cures and treatments for the approximately 95 of diseases without cures. Weve seen an outpouring of support, both outside and inside washington to Work Together and to achieve this common goal. So today, to continue that work towards more cures and treatments, we brought together truly another allstar round table. Joining us, we have secretary sylvia burwell, secretary of hhs, dr. Francis collins, director of the nih, dr. Margaret hamburg, commissioner of the fda. Michael milken, chairman of the milken institute, dean kamen, founder deka research, mr. Bill parfet, chairman and ceo of research from michigan. And dr. Dan theodorescu, of the colorado cancer center. We need to hear from our participants as much as possible. Thats why we have this round table today. Were going to listen. I know that my colleagues agree with me that the participants are the expert experts, and thi table is an opportunity to learn. To that end, while we may want to ask the experts some questions to guide the conversation, we want to keep our talking to the minimum. And i would also ask the participants to try to keep their answers brief to so that we can keep the conversation moving knowing that we have a hard stop at noon. In closing, i want to thank these folks who have offered their input on the 21st Century Cures Initiatives. I cant tell you how much we appreciate the thoughtful contributions of Everyday Americans and we intend to release a cures legislation draft early in january 2015. Well look to swiftly move the legislation early in the next congress. If you want your idea considered, you can send it to cures mail. House. Gov as quickly as possible. The more specific the idea, the better that it will be. So now i want to turn to my colleague, Diana Degette from the state of colorado. Thank you very much, chairman. Thank you for convening this last round table. I want to welcome everybody here today. I see the chairman today, in whose shadow we toil. Im glad hes here. I want to thank secretary burwell for coming. I know she has a very busy day and, of course, my homie, dr. Theodorescu, thank you for coming all the way out here. As fred said, weve been working together to hold a big series of round tables and hearings on the 21st Century Cures Initiative and have taken many white papers. Weve had a lot of suggestions, but i think we will both agree that we have a lot of commonality in the themes that were hearing and were eager to sit down and actually start drafting legislation. The round table that we had in denver over the break, we talked about personalized medicine. And we had active engagement and interest from the audience. We heard from the nih, the fda. Weve heard from local universities and Health Centers and private investors, industry and researchers and we really are beginning to hone in on some areas that we can think about how we streamline the process so that we can get from the lab to the clinic. And thats very important to all of us. One of the things we talked about, for example, was the potential time and cost savings of a central r. I. B. Process, something ive opinion working on for a long time. We also talked about the importance of sharing data and information among clinical registries. And these are just some of the topics that fall under this. Today, what were doing is were looking for feedback from participants on what weve learned so far and where we could go. And im really happy to see so many members here. Some of the common topics that weve seen are modernizing Clinical Trials, facilitating data sharing, insent advising drug research, incorporating the Patient Perspective into the research and regulatory process and developing young, emerging scientists as well as making sure we have adequate and Stable Funding for Biomedical Research in this country. And so im looking forward to hear from our experts and our colleagues today about how we can make a positive difference. I just want to echo your thanks for all of the members, all of the participants who have worked so diligently on this. It just really shows that theres a need. I want to leave you with one thought. I went around my district in august and some of you might know, just as happens every two years, in election year, colorados a purple state. So we have shock and awe of campaigning going onjdwn thert now. And every time i talk to somebody, democrat, republican or unaffiliated about 21st century cures, their eyes lit up. They were so excited about the prospect of c3bipartisan legislation, where we would actually be working together in congress to do something, but even more to do Something Big about how we can get those cures to the patients. I called a group last night. The big risk is it falls of its own weight. We cant let that happen. We have to Work Together to make it happen. And so i just really appreciate this effort. Thank you, mr. Chairman. Well start with secretary burwell. Thank you, chairman upton and representi degette for convening this round table and having me here today. I am looking forward to working with all of you. While im sorry i wont be able to stay, dr. Collins and dr. Hamburg, two of our best at hhs will ably represent us. I wanted to become of what you both have said. The importance im here today because we want to be your partner in making something go forward. Im a big believer that if were going to deliver, in fact, we need to start by building those strong relationships. That means listening to the experts we have and listening together and being responsive as we Work Together for meaningful solutions. I first want to thank you, also, for what you all did in terms of bringing everyone together but this committee has also done important work previously in this space with fda and our user fee legislation. These round tables are an Excellent Way to continue making some of the progress that weve already made. When we watch a loved one who is taken by alzheimers or als, we all want the same things, the most effective treatments, the groundbreaking cures and the best medical care possible. And so we take at hhs that very seriously and were committed to keeping us on that cutting edge of innovation, science and technology. And nih has supported most every significant advance of Biomedical Research in the last 50 years. From the human genome project and attempting right now to unlock those secrets of the most complex, mysterious things like the brain. And dr. Collins and his team walk the line, i think, some days between science and Science Fiction in terms of whether its robotic, exoskeletons, nanotechnology developments or personalized medicine and what that can mean. This work isnt just improving our Health Care System. Its hopefully going to transform it. These advancements allow us to shift from keeping up with illnesses to getting ahead of illnesses and they empower patients to engage in their own Health Care Like never before. And new biomedical science and technologies create highpaying jobs and strengthen our economy, another important part of what this work is about. In fact, we have estimates that say for every dollar of nih funding, it generates over a dollar in local Economic Growth. So, at a time when the world is vigorously forging ahead, we must protect our competitive Global Leader and not falling behind. With that said, we have a crucial equilibrium to maintain with the oversight that keeps us safe as a nation. Dr. Hamburg will be speaking to those issues. Fda is committed to reducing the length and soft of medical development. Weve shortened the highrisk device review times by a third since 2009, also working to strength sben en and improve th medical product process. Weve expanding accelerate D Development pathways. With review times as short as 4 1 2 months. Over the last years weve made great progress with support and we appreciate that. We also know theres a lot more to be done in that space and we want to focus on that as part of this conversation. Were committed to partnering with you while committed to maintain a safe and accurate product data and information for people. With that, we look forward to working with you all. This kind of session where a conversation can occur, i think, is an important part to moving forward. And the committee process, which we want to engage in support. So, thank you for having me. I just want to say that you and i talked about this, literally, the first week you became secretary of hhs. And you indicated then you would be very supportive. Were delighted for your participation today and all the way down the line. So, we really appreciate you being here, knowing that you still have to leave a little bit early. But thank you. Had you. Peggy, do you want to Say Something . Sure. Well, thank you so much for including me in this discussion and importantly for the leadership that chairman upton and congressman degette have brought to this 21st century cures and all of you on the committee. And it is true, as secretary burwell said, this committee has done great work to support science and the advance of biomedical Product Innovation and most recently with the passage of fadaz, i think we really accomplished something thats important and in a bipartisan way. This goes to the heart of what matters to us at the fda. Our mission is to deliver on the promise of science for the public, to do our part not just in assuring the most streamlined review times, but also in helping to support the most effective and Efficient Research anD Development pathways for promising new products, and to be able to provide information and oversight throughout the lifespan of a product, not justify in the development and review stage but also when its in the marketplace, being used by people when we have the opportunity to learn more about what works and for whom and why. And, you know, we think that the discussion so far has been enormously productive. In the identification of some of the critical area that is congresswoman degette noted very much align with our priorities and perceived needs. We want to work with all of you, to continue to reduce the length and cost of medical Product Development, to improve medical product review. Of course, improving business processes and systems but also to bring new tools and perspectives to bear. More advance science and technology, Patient Perspectives and, of course, increasing the predictability and transparency and partnership in what we do. We think that there are a lot of lessons to be learned from the work thats been going on with accelerateD Development, including the breakthrough designation that we were able to move forward with, with the passage of fadazia. The more the fda engages with stakeholders and more importantly with companies as they develop products to make sure that the right studies are done, we can reduce the time for research anD Development. We can certainly dramatically decrease the time for review. And, most importantly, we can give the American People the products that they want and stryker korpcorporation, a devi Company Located in our district. Im humbled by what i dont know, but excited about what we can do here today as the legislation moves forward. I hope during the two hours that we have here today, what i consider to be the critical success factors for our success going forward, talking about such things as a collaborative role between private sector, industry and government, including regulations that are required and can be a real companion to what were doing, including the changing role of the patient. We have to have a changing role for the patient, including the consequences of risk, which are sometimes becoming so ownerous that risk takers dont want to step up to the plate with really innovative ideas and also looking at the role of government in terms of helping out some of these smaller biotech that is have great ideas but dont have the Funding Sources and finally making sure we can energize our young people to be rigorous scientists. We clearly are the best we have the best Health Care System in the world. We have the best ability to innovate in this world and we want to unleash, unshackle some of the impediments that, over time, have allowed us to be the best we can possibly be. Im pleased to be here and thank you for the opportunity. Im dean kamen. Im not 67, but i spent a lifetime designing and building medical equipment over the last few decades, as everybody in this room is aware. Im concerned that the pace at which technology is developing is increasing. Were in the 1st centus 21st ce accelerating, the pace at which the systems around us are moving makes us feel like were trapped in a 19th century model trying to deal with 21st century opportunities. Over the next few hours, i would like to talk about a few of them. But a list of things that i think should be noncontroversial that should help things go better since, as Michael Milken has said more than once, the whole issue is time and money. We have to shorten the time and reduce the cost. If cms, fda and all the other agencies that are part of the chain of events could Work Together so that we could, for instance, in parallel do all the work we need to do in this industry to deal with cms and reimbursement at the same time as we go through the fda process, things would get faster and simpler and cheaper. Another issue that i hope is noncontroversial, no business i know could run if it lost 25 of its people every year. We have had multiple projects come through the fda and the reviewer changes. One of our most recent projects, the reviewer changed four times. Each time you sought to do a restart. Its expensive, takes time and costs money. We have to find a better way to keep continuity on the government side of this process. The third, and im glad that the congresswoman brought it up, is irbs. You go it you a pretty detailed, extensive process of getting an internal review board to approve it. Thats a necessary thing. It affects the people and the company. You have to do it in three or four medical centers and have to go through theach and every one separately. Thats crazy. International competition. Its not just that they compete sort of in the marketplace. These days, most of my major partners, the Biggest Companies in the world that want to take our products out, look at the situation and because of the lack of certainty in how long it will take to get a product approved or the lack of certainty of what the reimbursement will be for it, almost almost universally now, we transition to a world where our biggest clients would prefer to go and launch a product in europe or japan than in the United States. And that hurts on many levels. It makes the product less accessible in the United States. It has a lot of bad implications. We should be competing to be the most favorable place to attract innovation. Finally and maybe its just a small one to some of you. The device tax, which does nothing but make things more expensive but its really kind of a slap in the face to this industry. You could say its only a few percent. But a few percent is a pretty big number to people that are trying to make a new product. And particularly because its uniquely a medical device tax, as a matter of public policy, we learn that you tax tobacco, alcohol, have luxury taxes. Theres certain tacxes that the government puts out trying to encourage or discourage certain behavior. I dont know why people trying to make lifesaving equipment should be taxed the way you Tax Industries that youre trying to say its a luxury or its a sin tax. I dont like to be lumped with that crowd. And i think its bad policy. Thank you. Dr. Theodorescu . [ inaudible ] k close, and the green light on. Its a privilege to be here. And a little bit about myself. Im director of the university of colorado comprehensive cancer center. In that role, i am charged to the synergies of Cancer Research at the university of colorado, a consortium cancer center, which means we encompass three universities as part of our consortium. I also have a very vibrant laboratory thats funded by the federal government with multiple nih grants. And our area of interest is doing personalized medicine, establishing genomic tests to assign therapeutics as well as doing drug discovery. How we do it is in our moment in time. Thanks to dr. Collins division, its built an incredible genomic system intestine fra structure and resulted in technology to really push forward molecular. It would be a shame not to capitalize on that. We have a great opportunity. This initiative is fundamentally important, i think both from a Health Perspective and doing right and doing good by our citizens, but our International Leadership and also for the economy. And we have discussed the numbers of things about the irbs, et cetera. There are tangible things that we can do. One of them, for example, is aligning the reimbursement process with the regulatory process for biomarkers, really the part and parcel companions to targeted agents. They work hand in hand. We need to establish the guidelines for biomarket development. Biomarket sphere is reimbursed more than Drug Development but yet its fundamental to precision medicine. We could really think how we have parallel tracks so companies, small bioteches, Small Businesses can really develop biomarkers and have a possibility, an expectation of return on investment to grow this economy. The other sort of vignette that i like to bring out is in trying to protect our patients, patients are basically the heart and soul of translational medicine. We rely on them. When we have consent forms for drug trials 40 pages long, even though theyre written at the eighth grade level, perhaps my children, in eighth grade, were not as smart. I can tell you, they were really not prepared to read 40 pages. I think we need to visit those n trying to protect people, we are actually dissuading them from participating and hurting them. We really need to rethink that. Thats a vignette of a practical thing we could do to the central irbs and supporting our young investigators. That support can be in the form of facilitating the translation. People who want to do Translational Research have to have a clear career path and spending their time with forward motion, getting trials done, getting patients on trial. If the barriers to accruing patients on Clinical Trials are so high, well get progressively fewer young investigators wanting to embark on those careers. Its not a money thing. Its basically trying to overcome obstacles. After a while, you just get tired and you go and do other things. We need to remove those barriers and that will promote our young investigators and also the entire translational infrastructure of Clinical Trials. Thank you. Commend the work of both you and representative degette have done in those efforts. I am here today not as the chairman of the milken institute. Im here today not as the chairman founder of faster cures. Im here today not as the chairman and founder of the Prostate Cancer foundation. Im here today not as one of the founders of Melanoma Research alliance or adviser to the focus ultrasound foundation. Im here today as an american who lost ten relatives to cancer and representative of the men and women in america, one in two men, and one in three women, that will be diagnosed with cancer. And i am the happiest person to be with you today, mr. Chairman in that they gave me 12 to 18 months to live when i was diagnosed with cancer 21 years ago. And im here to remind the committee that 50 of all Economic Growth in the last 200 years has come from bioscience and public health. That the greatest achievement in the history of the world has been the increase in worldwide Life Expectancy from 31 years of age in 1900 to over 70 today. It took millions of years for average Life Expectancy to go from 20 to 31. And a little more than a century weve risen average Life Expectancy from 31 to 70. Im here today to remind all of us that it was 20 years ago that a woman had more than a 90 chance of passing hiv or aids on to her children. And today with modern science, its easy to understand Economic Growth when in subsahara, Life Expectancy has increased 50 in a generation with twothirds of everyone living with hiv or aids. Living in Subsahara Africa today and its no surprise as a result that today six or seven of the Fastest Growing economies in the world in this century are in Subsahara Africa due to the great benefits of u. S. Medical science, possibly the greatest contribution of the United States to the world has been the benefits of this medical science that is available to so many people today. Those countries that lead in the biosciences will lead the world in this century. Were not just talking about health. Were talking about agriculture, environment, energy, air, water, et cetera. And so were of the dawn of the golden age, this enormous investment that the American Public has made with the leadership both in the administration and the senate and the house, approving in 98 an increase in funding, which now exceeds over 200 billion. Were about to reap those benefits. Whether those benefits will accrue to the United States of america or to some other country will be determined by the leadership and the commitment of this committee and others today. There are five easy lessons we could talk about today. One, putting the patients at the center of discovery, development and delivery process, more efficient Clinical Trials, meaningful working relationships between patients and disease specific organizations. One of the greatest advancements of the past two decades has been the strengthening of the research group. Two, incentivise collaboration across the r d process, public, private partnership. Look at history books and see those partnerships between ford and kaiser and the government that allowed us to have victory in world war ii as we moved our forprofit Industrial Companies quickly to produce ships and other products we needed during world war ii. Three, change the risk return ratio. As an example, mr. Chairman aushlgs we have to do is look at acid rain. A major issue in the latter part of the 1980s in america. Movies, public hearings, et cetera, the loss of life to acid rain. The creation of a Financial Market for sulfur index, to reduce emissions, to buy credits, eliminated 95 of all the acid rain in america. Strengthening the basic Translational Research infrastructure depended heavily on the next generation of young scientists. Stem education is the rage for men and women, boys and girls in america. After 15 years of study iing ph, mds, internships, residency, we have little to promise them in the future. And today, america is losing our best and brightest students to the rest of the world and we run the risk of losing generations of young scientists either to other countries or to other fields in the world today. Fifth, assure sustainable and predictable funding for our science agencies, nih, fda, nci, cbc, cms, et cetera. Its impossible to build an organization or recruit the talents you need when, on a yeartoyear basis you dont know if you can fund them. The probability of a young scientist today being funded is the lowest its been in more than four decades. This golden age is in front of us. We think about the cost and to do no harm. In many ways by length time, we are losing lives. By not providing access to the state of the art treatments today, by not incorporating science. In many ways, science today is running in the 21st century. But as dean has pointed out, its running on tracks from the 19th and 20th century. We wonder why today in america that the average train, symbolic of science, travels at the exact same speed it traveled at 100 years ago. It has nothing to do with science or the ability of the train to move. It is running on tracks that cannot afford to have trains running at those speeds. We look at china and around the world whose trains run two to three times faster than ours. Its not that they have different technology. Its that theyve allowed different infrastructure, 21st century infrastructure to allow those. One of the things im quite confident on is that this committee will help us to build that 21st century infrastructure. Im Francis Collins, director of the National Institutes of health and mike milken has, i think, wonderfully articulated both the promise and the challenge of living up to that promise. I want to thank chairman upton and representative degette for convening this whole series of discussions. And many members of this committee who have participated by convening similar hearings and round tables in various parts of the country. I was here for the first round table may 6th in this room. I was two weeks ago in lancaster, pennsylvania, when representative pitts convened a very Interesting Group of us around that table. It is enormously inspiring and encouraging to have this kind of focused attention on the opportunities that now lie in front of us for developing these 21st century cures. As director of the nih i have the opportunity on a daily basis to see things that are impossible, things i would not have dreamed would happen in my own lifetime. At the same time i also preside over an effort that, as mike has laid out, is under the greatest stress that it has seen in four decade decades with young scientists who see it increasingly difficult to see a path forward to lay out their visions. So, it is welcomed, indeed, that this group has come together to try to see what could be done to cat lies greater progress and those cures that are still being waited for, only 500 of which currently have a therapy thats available. The gemone project helped us a lot into stepping into a new space here. Now we now have technologies that allow us to look into the intricate details of how a cell does what it does and how things go wrong in the presence of disease. And yet were not capitalizing on that opportunity at the level that we might. My own area of genomics, i cant help but point out that while we, the United States, led the human genome project and, from that, derived, its 1781, that derived from the leadership of that effort. If you ask me where is the largest investment in genomics in the world it is not in the United States. It is in china. They read our playbook, saw an opportunity and took advantage of that. Over the 11 years since the genome project was completed in 2003 have seen a steady decrease in our ability to support research with a 23 cut in our purchasing power for a Biomedical Research since 2003. And the results of that is the subsequent loss of thersfawjui leadership thpa was clearly ours at that point. Dont get me wrong. I celebrate what other countries are doing but why would we, the United States, with all the talent and creativity and vision here decide to exit the stage just when it was getting interesting . We clearly have a Critical Role to play here. I really appreciate the way in which this 21st century cures effort is asking very specific questions of what could be done by the congress in legislative terms that would assist us in terms of our ability to go faster. And we will be supplying i mentioned in other sessions specific examples of that sort. Its getting to be quite a long list. Some are big ideas and some of them seem kind of small, but they would help us a oog dna sequence would be an important step forward. Second area is to facilitate collaborations and innovation. Weve already heard some of those issues, as relates to central irbs, something that we also feel quite strongly about and a common rule, the way in which we have overseen Human Subjects Research for 20 years is desperately in need of an upgrade. A lot of work has been done on that. It has not come across the finish line. A simple area that, in one way, is getting in our path, our new National Center for advancing translational sciences. For reasons that i think reflect anxieties about whether this would be competitive with the private sector is legislatively prohibited from pursuing clin Clinical Trials beyond phase 2a. Something at the present time makes very little sense. Nobody would support the rationale for that, given we have worked very hard to be sure worry in no way competing with the private sector. And the third category, reduce Administrative Burden and increase efficiency. The famous paperwork reduction act has been for nih, the source of the greatest amount of paperwork that you can imagine when it comes to individuals who are trying to run simple studies that we wish to actually do a survey. This happens many ways for trainees in our program. You may not know that you cant run a survey to ask people about their opinions about precision medicine, for instance, if youre going to ask more than nine people, unless you go to the omb and get approval for that. That takes about nine months, at which point the trainee is about ready to leave. Clearly, theres helps we could use here. All those reports, if you start adding up the reports that are congressionally required. As far as i can tell that nobody reads but requires many hours of busy peoples times, it would be great to go down the list and throw out a lot of those that, if they once served a purpose, no longer do so. Again, i just want to say how critical it is that we look at this issue from a holistic perspective, of all the ways that our enterprise could be more vigorous and more healthy. Particularly, as mike milken has said and others, Diana Degette mentioned it as well. Theres a great sense now of instability, of insecurity, of a trajectory that nobody can be quite sure of. Thats the worst thing you could do if youre trying to encourage innovation. To find that path forward would make a huge difference. It will make a difference in very concrete ways of saving lives. One other story, which is very much on my mind and then ill yield up. Here we are, all of us, deeply troubled by what we see happening, the number of deaths from the Ebola Outbreak now over 2,000 and with no end in sight because of the way in which this terrible disorder is spreading rampantly like wildfire in liberia, sierra leone and guinea. We have steadily pursued the effort over the last 13 years, hoping that by the time a big outbreak occurred, we would be ready for it. That vaccine has now been in animal trials, shown to be highly effective, but as of this summer, when the outbreak really began to catch fire, had not yet quite reached the point of a phase one Clinical Trial. We worked in record time with enormous assistance from our colleagues at fda to speed up the process and last week the first volunteers at the Nih Clinical Center were injected with this vaccine in a phase one trial to determine whether, in fact, it is going to be safe. And in another two or three months, we will know that and then hopefully be in a position to begin a phase two trial in the places where its most needed in africa. But i have to tell you, if weed wee had not gone through this tenyear decline in this support of Biomedical Research, we would be a year or two ahead of where we are now. Think of the difference that would make. Had we, in 2014, been in a position to distribute rapidly tens of thousands of doses in collaboration with our colleagues of this vaccine, how much different would this be and how many lives would we have saved . The consequences of whats happened in the last ten years is not just that its been tough on some scientific researchers, although it really has been, not that its just damaged our ability to take advantage of Economic Growth that otherwise would have happened. Its also that lives are not being saved that could have been. The importance of this conversation could hardly be greater. I thank you for the chance to be part of it. Thank you all. Great experts in your fields and i didnt mention at the beginning that, you know, not only is this Committee United in working on this together, but we also have the commitment of leadership on both sides. Steny hoyer was there, testified that first day may 6th and kevin mccarthy, our new majority leader, is on board as well. Lets open this up for a discussion. Frank . Thank you. Again, i want to say to both you and diana how valuable i think that these round tables have been. And we had one, i guess it was was it only a week ago . Seems like two weeks ago with chairman pitts in his hometown in lancaster, pennsylvania. I thought it was very good. I wanted to follow up on something that commissioner hamburg mentioned that day. You had talked about engaging patients. And i think you were responding to some extent to the woman who was from my district, in edison, with the hydrocephalous foundation. And i just wanted to talk a little bit more about that, and how we can engage patients. I was particularly referencing these as part of these foundations like that. So, if you could, just elaborate a little more. Mention something about the advisory panels and patient participation. Well, thank you. Its such an important question. This is a critical time, as were really rethinking not just within the fda more broadly about how to really put patients at the center of what were doing and really include the Patient Perspective and reflect the patient needs in the work that were doing. It goes far beyond engagement on an advisory panel. We do have patient representatives on advisory panels. But it really is to sit down with patients and patient groups in a systematic way, to better understand their experience and their perspectives on the products that theyre using, whether drugs or medical devices, and what they actually need to better understand how they are experiencing their disease and what are the critical symptoms of their disease that they want addressed and then how we can what, of course, are the unmet needs and how can we actually incorporate that into Product Development and product review . For example, biomarkers was mention mentioned with respect to personalize medicine, look at genetic traits and linking it to a therapy. We also want to be able to use patient reported outcomes like biomarkers, in order to make sure we are assessing and mike milken. We increasingly work with them as well as with the Research Community and product sponsors to identify critical needs and opportunities. And then together think about how to shape the in particular, the Clinical Trials that need to be done. But other aspects of Data Collection so that the research anD Development and review process can be as accelerated as possible. One good example is the work that was done with the Scientific Community and the Cystic Fibrosis foundation to develop a new drug clidaco, a new drug for a subset of patients with a specific genetic marker but the first drug that treats the underlying cause of illness and not just the symptoms of this dove stating disease, Cystic Fibrosis, building on research that Francis Collins did a while back. But, you know, this has been transformative in terms of the lives of these patients. The Cystic Fibrosis foundation really made this work possible because they helped to create the Patient Community necessary to rapidly move into Clinical Trials. To get the tissue as well as the patients that could create a foundation for understanding the opportunity and moving quickly on it and put up critical resources. Not just patients as well as dollar resources to help move the dollar forward so theres a lot of important activity in this area. And i think we need to look at whats being done. And its incredible support and success and build on it. And i think this effort 21st century cures can put a major focus on this and help us advance this work. Thank you. Mike burgess. Thank you, chairman and all the witnesses being here. Ill be one of your test subjects on ebola vaccine. You rejected me on age. I thought that was discriminatory i had to reject myself on the same grounds. I still stand ready to offer my services. We had a good with Manhattan Institute we had a very good round table and i think faster cures was parter of that up in boston. And the concept of adaptive licensing came up. Currently, of course, Clinical Trials, someone is approved or not approved. Once its approved, its assumed to be safe and effective until proven otherwise. The adaptive licensing pathway that some are using really did seem to have appeal because we live in the information age. So the rapidity to which something could come through partial approval, the feedback thats now possible with the Information Systems we have, it seems this would be a reasonable direction in which to proceed. I hope its something we consider when we write our legislation. I also ask the administrator at the fda, is this something you all are looking into or acceptive in the realm of adaptive licensing . Its very much a topic of discussion and we have been involved deeply with counterpart regulatory authorities in other countries that are also looking at this as well as the Scientific Community to look at what are the opportunities to get robust Scientific Answers from a range of innovative approaches in terms of Clinical Trial design that can underlie licensure and also innovative regulatory pathways. At the frequent time the fda actually has quite an array of tools and regulatory flexibility things to our work with congress and the designation. Of course, our approaches are not binary in the sense that accelerated example, using biomarkers as surrogate end points enables us to move a product with promise into the marketplace, but then collects data in a continuing way to actually then assure the final approval if the product really works and provides an overall benefit for patients. But this is an area rife with opportunity to explore. How can we streamline, modernize and best adopt our regulatory pathways to make a difference. I think secretary burwell mentioned that half of the drugs that made a huge difference. Breakthrough designation is proving to be a real benefit. I would have to tell you, one of most important things to emerge in recent years in terms of all this work, what makes the most difference in actually reducing times from discovery to the bedside is really the ability for the researchers and companies and fda to all Work Together early and in a continuing way to shape the Development Process, to identify what is the data thats needed to move us as swiftly as possible over the finish line. And i think, you know, that is really where the most important work needs to be done to make the most difference for patients. Well, it just you know, i have to add then the comment was made this morning about the 25 turnover. Someone is working with someone at the fda. Theyre getting to a point where perhaps the steps are go ahead to be taken and, boom, have you to go back to square one. I hear that all the time. Thats a huge concern for us. And, you know, it reflects, unfortunately, that people are stretched too thin. They work too hard. They burn out. The conditions are challenging. And theyre very competitive in other sectors. So, i think we need to address those issues. The user fee legislation has enabled us to begin a process of hiring on more people and begin to create the training and management symptoms that we are intending to help us do a better job at recruiting and retaining people. But that is an essential issue. And it can undermine all of the best efforts. Adaptive licensure will not fix that problem. We need to address the issue of recruitment and retainment, i think, in a very targeted and forceful way because it is essential and it has so many critical ramifications. And crosstraining within the institutions was not up to one individual. Thank you very much, mr. Chairman. I want to commend these conferences, roundtable discussions. Theyve been very valuable. What has come home to me in the number of meetings weve had, is the promise of a lot of new innovations in the health care area. Its an exciting time in medicine. I also have been struck by how dependent this whole future is on the government in terms of nih and fda having the ability to do their work in a new fashion. We gave fda a lot of authority to be more nimble, to be flexible, to work more collaboratively with those who are developing pharmaceuticals and medical devices. And ive heard a lot of praise for that. And with the nih, theyre so clear how stable its going to be on funding for Biomedical Research. If we had a tenyear decline in nih, its not just people that work at or for nih. Its the whole Health Care System that pays a very heavy price. So, if were determined to do anything, and i think reflecting the will of the American People overwhelming is lets adequately fund nih research and the food and drug administration. Im embarrassed, although i accept it already, that we have to fund the work of fda through user fees. This is a government function. Those who are trying to develop new breakthroughs have to figure out how to pay the user fees. Thats just look, i author the law, set up the user fees, but we do what we have to do. We shouldnt see the constant decreasing or just flattening out of these funds, which are erode over the years. I am also proud to have my constituent and friend mike milken here. Particularly the 2011 discussions about barrier that prevent progress. I was struck by the fact that you talked about the cap and trade program for the acid rain to get rid of acid rain, which i authored. It seems to me we need to do that for carbon as well, which is to unleash Market Forces. Dont think the government can do it. We need to give the right incentives. We need to step aside and let Market Forces dictate the infrastructure to accomplish the goals we want. We need to do it more in the environmental area. We especially have to do it in the health area. Part of that infrastructure must be the must be the the maintaining and Stable Funding of the government areas. Now, it to whomever on the panel that wants to address this, legislation is a blunt instrument. We heard about the paperwork reduction act, which has caused more paperwork, that records all the other things we think are going to make things easier. Instead, they make them harder. I think we would welcome legislation to reform that area but if we come in with a blunt instrument, suddenly recreating the fda authorities or mandating things on the fda or looking for the department of health and Human Services to do things theyre not equipped to do, and then of course we dont fund them to do it, i think we have to be cautious about the legislation that may be proposed. More than anything we want government to be there, to work collaboratively, but we still have to recognize the safety and efficacy, which American People and most people here would certainly think is necessary for a new product. But we want those products out as quickly as possible. I remember the days when we had the first evidence of the aids epidemic and the people that brought home the reform at fda to get some of these th3mvp groups know more about those dieses than anybody else because necessity worked so hard to try and understand whats at stake. Pressing the government to be as flexible as possible, i think, is important for the infrastructure for the 21st century cures. I just want to say, i would be a little cautious thinking we could solve big problems with legislation with all the unforeseen consequences that can we can sit at a panel here ten years from now we can bemoan. I thank my colleagues and constituents and mike milkin for all he has done. Joe pitts. Thank you, mr. Chairman, for convening this roundtable and thank you for the expert witnesses for your insights. Every time we have one of these hearings and roundtables, we learn a lot. We had an excellent roundtable with dr. Collins, dr. Hamburg, mr. Burgess was there, and it was very meaningful and important. Dr. Collins, let me ask you, you forcefully made the case its imperative for congress to should examine as it attempts to find federal dollars to help advance the next generation of cures. Your thoughts . I dont envy the position youre in, in at the same know you have to figure out how not to have a circumstance where we bury our heads in the sand as the deficit grows so youre faced with very many difficult situations. One thing weve been trying to do, and it has helped a bit s to identify other ways to support medical research other than the traditional nih appropriation. One that has just gotten started about six months ago is a partnership between nih and ten Large Pharmaceutical Companies called accelerated medicines partnership, which is really unprecedented in its scope. The goal here is to try to identify the next generation of drug therapies we know is waiting to be discovered given all the breakthroughs waiting to happen, which will get here sooner or later, depending on how organized we are are about it and where the expertise doesnt reside in any sector alone. Putting them together, academic researchers could be exciting. Companies recognizing that, and much to their credit, are willing to engage in a partnership with nih where all the data is immediately made accessible. Its not one hidden behind a curtain. So, this partnership which is being equally supported financially by both the companies and the nih, 50 50 split here n an unprecedented way is moving forwards on diabetes o alzheimers disease and on Rheumatoid Arthritis and lupus. My colleagues in big pharma watching this closely want to make sure it doesnt run off the ralsz are quite gratified how far its come. We ruls aworking in every way we can to identify partnerships with other parts of the government. Darpa where we have a number of exciting initiativesing including some that aim to put cells on a chip to allow you to investigate their behavior. I have a kidney here on a chip, by the way. I got a lung over here. These are basically amazing technologies, bringing together the engineering skills of darpa with biology skills of nih investigators to move things forward in a fashion that could be great for identifying drug toxicitieses as well as effectiveness. Youre asking about other places to go and look. Im probably not in a great position to be able to say, of all the various parts of government support, where are there opportunities. You mentioned pekori, im on the board, by statute the board of governor includes nih governors. And theyre working hard to identify which interventions work and which dont work for patients in the real world. Obviously, issues we need to know about it. Its a hard working organization thats only been around two or three years. I dont know how to balance thosetic kinds of decisions people are struggling to make. I do want to promise this whole group that were not just at nih saying, give us money and were going to keep doing things the way we always have. I get it. We have to be creative. We have to make reforms in the way in which we administer our own funds that you all through the public give to us. We are looking at every nook and cranny to come up with ways to be more efficient with what weve got. I promise you, this is not just researchers with their hands out saying, leave us alone. We understand the obligation to be incredibly efficient, creative about what we do and productive. Were willing to put ourselves under the microscope about whether were doing that right or not. Mr. Chairman, can i just suggest a couple of one, i would say scoring. When we look at how things are scored, we need to totally reevaluate that. I quickly cite one example. Fellow board member and friend of mine for years, ward trip casell was diagnosed 13 years ago with cancer and given a short period of time to live. He engaged in every Clinical Trial, in this case for Prostate Cancer. He recovered and over the next 11 years, i would say 10, extremely productive. He led two tours of duty to iraq where he led our medical efforts in iraq. One in afghanistan while he was taking chemotherapy. Became the assistant secretary of defense in charge of the health of everyone in the d. O. D. When we had issues at walter reed, he was called on to solve the problems at walter reed. Founded two bioscience companies, was previously the doctor for both president bush as a cardiologist and hired,000 employees. In the scoring, everything he accomplished in those 11 years, not the least of which was 11 years more that his Young Children had with him. Who are very young, 3, 4, 6 at the time. All of them will remember their father. Their lives will be different. Their childrens lives will be different. His score was zero of any benefits of those treatments he got. So, we just count the cost, we dont count the benefits of this Great American patriot. And the 11 additional years he got every person and cancer dies 16 years before their normal life span. What would those things have been . I would say scoring. The first gulf war supported by Henry Kissinger and other efforts. United states put up 80 of the money, other countries 0 of the money and the u. S. Managed 100 of the money. If we focused as the state department and other international efforts. Other countries are per capita basis are far wealthier than United States today. Norway, for example, many years ago set up a fund for the north sea. Its kind of a rainy day fund. Their goal, 500 million u. S. That fund today approaches 900 billion. Theyve overshot that fund. And the United States that would be like if you had a fund of 60 trillion sitting there. They can play as many other nations can an active role in activities, if we had the structure here to get Financial Support from them today. Third, young scientists. Many things have been created to encourage young to work in the field of teaching. If you go and get teaching degrees from universities, if you work in inner cities or difficult areas, we forgive their Student Loans theyve had. Almost all noble prizes have been issued for ideas within five years of school up. Might have been 60 years old when you received a noble prize but the idea was generated when you were 25 and 30. Today the average age, im assuming, francis, is low, the first time an individual received a grant. How discouraging can that be for a person with new ideas to receive a grant. Weve all had children grow up, go to college and leave. Intellectually challenged when our teenagers leave the house, to go to college, to find out how something works. This is the same technology thats available in science today but we havent found a way to encourage people to stay in science, to stay in research, to stay with the nih. And one of them might be easily to forgive their Student Loans they accrued, being a doctor, scientist, et cetera, if they work in these fields. So theres benefit the same as a teacher in the inner city. Lastly, matching. Challenge the American Public. Challenge philanthropies. We have funded over 500 young scientists. Its amazing how little the money 100 young scientists for the fda. 100 young scientists for the nih and others around the country. Lets challenge them where you need matching grants for those young scientists and i think we could accrue billions of dollars in matching grants to encourage those young people and further their careers. Thank you. I have 11 people on their list. Gene. I want to thank you all again for those who serve on the hill subcommittee, its easy to have these kind of exchanges. Dr. Hamburg, i want to thank you for the gain act we passed last session. We have a followup, adapt act that would give some more support for the future of antibiotics and hopefully well do that. But my main question, dr. Collins, july 30th and july 31st joint fda nih meeting on new antibiotics you mentioned the u. S. Government should launch a new Public Private partnership for antibiotic development which would work to master Clinical Trial. This is similar to what mr. Mr. Milken talked about. The new drug for bad bugs, initiative in europe, they like ak crow anymores there, too, ndb 4 were crucial for bringing together Key Stakeholders in academia to tackle the scientific challenges facing antibiotic development. Smaller Public Partnerships although nih and berda have important components. Dr. Collins can you tell us more about the private Public Partnerships and the master protocols. I certainly can. Thank you for the question, congressman. The fda and the nih and industry all got together at that meeting july 3031. Peggy hamburg and i worked together through our joint Leadership Council to work through this. Its a very important issue because you see the way in which increasingly infekdzs, particularly in hospitals, partially in patient who is are very sick in icus turn out to be from organisms that resist all known antibiotics. We are on a trajectory thats quite frightening. The delivery of new antibiotics through the pipeline has been quite slow, or mostly, for financial reasons. The development of new antibiotic is not seen by most pharmaceutical companies as necessarily something that will be profitable. You have a short market, a lot of liability concerns. Here we are in a circumstance where the bacteria are increasingly through evolutionary processes after beibe ing exposed to drug prescribing going on in hospitals and agriculture are developing these resistances and we dont have, at the present time, a fully effective strategy. One of the things that we need as part of that strategy, and this was the main topic of that meeting, was to have a preexisting network of Clinical Trial sites so that if a company says we now have a promising new antibiotic, you want use that on people who are infected with these highly resist ant organisms, but you have to found them. Fortunately you dont find them anywhere. They tend to happen in outbreaks. If you dont have a preexisting network with staff on the ground, consent forms, approval, the company has the drug but theres no way to check it out to see if it works in am, am months. Nih through our National Institute of allergy and Infectious Disease in order to provide industry with that kind of a platform. Its a great example of a partnership where we all have a lot in common, a lot of needs here. There will be, by the way, in the relatively near future, a lot more information about plans that weve been cooking up over many months now about how to take a systematic look at this problem of antimicroabal resistance. There be coming from the president ial council advisers. We hear a lot about ebola. The only two doctors who were able to get something. But mrsa is in every hospital in the country, where we have our loved ones and our constituents. Thank you for that effort and hopefully we can move forward with it. 30 seconds. [ inaudible ]gnnr bill, i had the opportunity to go through your wonderful facility a number of times this last year and see the advances of what you have done as relates to animal testing that can then help the drug industry. Tell us a little about you know, as we think about trains traveling at the same speeds but the tracks are the same, lets talk about changing those tracks and what youve been able to do with the science to, in fact, improve the testing and expedite the approvals. You were there when we launched our moleculeal ability. In many cases, on a regulatory basis, the regulations havent kept up with this science. Thats not a criticism. Believe me. Its not a criticism at all. No. But this isnt one. But, for example, theres no fda imaging approved for biomarkers for early stage. And yet when our clients go through the archaic wakes of working through toxicology and efficacy and says we know youre not required to do imaging, do you have any . What that says to me, its our fault that were not keeping pace with changing technology. And so, i think in the case of the Regulatory Environment in which we live that its so hard for regulations to keep pace. Its so hard for regulations to really help allow for new innovative methods to be tested and used. Maybe theres some way to have emerging issues task force or something along those lines. Im sure you thought of this before where, when new technologies are available or being used, perhaps not in the mainstream of regulatory approval, but are being used to supplement insight into drugs. Again with imaging in particular for Clinical Trials, its incredible what kind much insight has been approved as a result of looking at biomarkers and absorption, et cetera. If there were emerging issues task force, where in this case in a collaborative way, the prepare the sponsors in this case, researchers, innovators could work with regulators, the subject experts so we could quickly allow some of this to happen. I know this is litigious world we live in and breaking new science. But what you saw there is what is its not theres a lot more advance technology and science than just imaging, but its coming of age today. It can clearly add significant insight, improve cost, reduce speed, as you know. And i would encourage that part of your legislation would allow us in a collaborative way to allow us to Work Together to be allowed to experiment and allow to work let these new technologies help us meet the goals and objectives you define. Just a quick comment on that. Very important point and it raises two different issues in my mind. One speaks to, i think, a broader opportunity that builds on this notion of the importance of Public Private partnership and Collaborative Research in critical areas. There are things, biomarkers, . H various kinds of computer models or simulations or other kinds of approaches where no one company is going to necessarily want to take the risk of trying to develop it and characterize it and get it validated because theres a lot of risk and a lot of cost. But if these activities could be undertaken and we could identify these new research and regulatory review tools, whole classes of products would benefit and Many Companies would benefit. Find ways to take some of those Research Dollars and make sure that they are targeted to these critical areas that are gaps in Current Research funding that requires the kinds of new Public Private partnerships as well so you get the best minds around the table working on these projects that often are really complicated and evolve emerging technologies and how best to use them. And weve seen the benefit already in the biomarkers area, for example, and the biomarkers consortium. These are really underdeveloped models for private Public Market and Collaborative Research and really underresourced in terms of Research Funding at the present time. I think its a critical need and its one where we know that if we develop it, it will make a huge difference. The other thing is, you know, the regulatory process is really cumbersome. You know, congressman waxman noted congress recently gave us new strategies and authorities to be more nimble and flexible, but, you know, putting regulations into place takes years and the notice and comments process is an important one for getting input but its lengthy, et cetera. You know, thinking about what are some of the models for how to work in more responsive, more collaborative and more flexible ways in these kinds of situations where an emerging technology and an emerging opportunity is there, i think, is a challenge. I dont know if it can be fixed by legislation, per se, at the present time, but i i think its an area we need to delve more deeply into because it really matters. [ inaudible ] in the end i know my years in the pharmaceutical industry one of the Major Concerns is we have to do everything ourselves. Its our pry pry tear information, were not going to share it with others. You dont see that anymore. Theres not a drug that gets developed today that hasnt been developed by someone crosslicense with somebody else. There is that collaboration. So, i think the movement is underfoot. We just have to provide the framework that encouraging it to happen so we can, indeed, get these 21st century cures to the public more than we do. I think its all there. But collaboration is clearly the key. Thank you. Congress should be doing. That is number one. Its followed shortly with shortage of residents but we have this issue as well with how medicare pays for physician residents and theres a shortage in a number of states. We were supposed to go and look at nationwide the Health Workforce and we havent done that yet. I think hopefully it could be incorporated into what were doing. Id like to follow on the last exchange on the biomarkers is this a problem that yall are hearing about . Maybe dr. i; genomix, et cetera, when the big Data Revolution and then in the middle of a health care revolution. So, i think i like to answer your question with two sort of two answers. One is that i think in terms of public and private partnerships, i think we need to enhance by really providing a coalesced data set of all Clinical Trials that are federally supported. That will make mining that data by said companies and in the examples that dr. Collins made, would make the cooperation of the federal government a lot more attractive. Thats how we can enhance things by biomarker work as well as industry government partnerships. The other issue that would facilitate these biomarkers is, as i was saying earlier, is to support often the Smaller Companies that cannot really afford to do clinical biomarkers in hhs. Thats a good way to potentially cover some of these biomarkers as were gaining the knowledge through registries to see their effectiveness. Also, as you probably know, moffett has total cancer care, which we have very good relations with bill dalton there. Were about to launch a joint venture with them. And so the concept of moving those biomarkers through the orion system or through the total cancer care where theres these large consortia to pull together the discovery markers, it comes down to managing and leveraging big data across multiple institutions to really not only develop new biomarkers but also validate them and part of that is the coverage with evidence development. If that could be expanted, it would help a lot. As well as supporting a central repository from federally funded Clinical Trials that would also be a boone to industry partnerships and making those very attractive. Thank you. Bill cassidy. First, let me just thank you all for your good work. I am struck, though, i think its a little ironic because i think congress has created a lot of problems we have here, at least the federal government has. Im struck the 3 tax you speak of is obviously a part of the Affordable Care act. You know, an example. Now, i guess i have two questions, but along that theme, im a fellow that did clinical research. Ive gone through those 40page documents. We wrote ours in sixth grade, not eighth grade. I was always told it was the office of Human Research protection, which part of hhs that required this. Whenever i asked about centralized rib, i was told, we are required to have a local representative. Although i know theres a central r. I. B. In denver, for whatever reason my institution did not allow it because they felt like the federal government required, and im looking at you dr. Hamburg. So im told this 40page document is washington, d. C. Said you had to have it. Dr. Collins, you and i have spoke about this in committee. We spoke about how nih needs more funding but theres an article out of ucsf, speaking about how theres a very poor correlation in nih funding between Disease Burden, however defined, and where nih puts its money. Now, this fellow wrote, and he said ten years ago, this was a credit te critique, but as he reviewed data there was no change. You mentioned a little wiggle room in terms of moving dollars around but what im most familiar with at cdc, at one point they were spending a billion on hiv and like 60,000 on hepatitis, even though the number of deaths related to Liver Disease was as great. So there seems to be a lousy correlation between where were spending our dollar and Disease Burden. So two questions away from things, frankly, weve had a great deal of success with. So, perhaps you first. First, i think that you have raised a set of really important issues were all working on and certainly francis and i have spent a lot of time discussing the value of central r. I. B. S and more broadly the experience of reducing the time, cost and cumbersomeness of doing critical trials in this country. And anything we can do to reduce that and make Clinical Trials more accessible to patients, because that is another barrier to advancing Clinical Trials and ultimately the delivery of opportunity and science into a real world product. So, i think that this is a really important issue. I think one ultimately this effort, the 21st century cures will likely take on some way on another. I applaud that. And i think that it is an area where it has to be done right. Where there are Things Congress can do to actually make the situation better, not worse, big picture, there are many others than fda that need to be engaged in that discussion. Youre right, theres a Critical Office in hhs thats involved in this. Work continues. Theres a smaller scale congress could do quickly that would enable Clinical Trials and device area to be more efficient and effective, and that is, that the food, drug and cosmetic act actually has a requirement in it right now that mandates review of a Clinical Trial on a device by a local r. I. B. We think that if that could be amended and, you know, i think its a Pretty Simple fix, we could do some good. Done. Done. Francis, yeah, go ahead. So, congressman, appreciate both of those very significant questions. Just quick coda to what peggy said. There has been an effort under way for five years to revise the rule. Have their own r. I. B. Tinkering with the consent form, adding another paragraph, changing the tense of the verb and all that stuff that ends up costing you a year of time and a lot of pages that get generated along the way. It is irrational. We need to stop this whole approach. Nci, the Cancer Institute for their large scale cancer, are using largescale r. I. B. Those are largely been overcome. We can do this. We just need to get on with it and i appreciate you raising the issue. In terms of this correlation between funding support and specific diseases, we look at that a lot. Thats one of the Jobs Institute directors and i talk about every week when we gather around the table. Actually, if you look at the current plot, and id be glad to send it to you, its not that wildly out of whack from what you might think if you look at various measures of disease, whether youre talking about deaths or dailiys and qualies, actually that quufcurve is a reasonable. Mags wi with the exception of aids. [ inaudible ] im saying aids is an outlier. There are scientific reasons. Namely, this is not just a disease in this country. Its a threat to the entire global circumstance. Your relative funding of alzheimers relative to that is miniscule. Again, my dad dies of it. You know, we all have a parent. And i got to say, when i look at your funding for alzheimers, it seems miniscule relative to the Disease Burden and future expense. The near future expense to medicare, medicaid, et cetera. Im deeply concerned about alzheimers, congressman and have spent some of my personal efforts of trying to do readjustment in the way current portfolio. This is a current threat to the nation and the world and an enormous burden to families stricken by that disease. I take your point we need to see that as a particularly high priority right now and that has been, in fact, what we have been trying to do at nih over the last two or three years. The other thing i want to say, and i dont to want take up too much more time, is that we have to be careful with the way in which science has developed over the last four or five years, particularly coming from insights from the genome. We discover there are connections between diseases we never knew about. It would not surprise me if the next breakthrough were looking for in alzheimers disease came from a research who at the present time is not working on that disease but working in some other area. This connection of pathways has continually surprised us. I do think we have to be kale not to be so targeted that we miss out on the unexpected where somebody worging in one field actually comes up with the aha moment. But the billions spent on aids has paid great dividends. It seems like on some of this were hoping for sarin dipty. I think we need both. Targeted research is critical when we can see a pathway towards finding solution to a disease. Dont know if i have the mike on or not. Is the mike on . Yes. Good. I want to thank all the members of the panel. Youve done a fantastic job. I do apologize for coming in a little late and missing an important aspect of it. Representative degoete, chairman upton, these roundtable discussions have been absolutely invaluable there are a number of physicians, of course, on our committee. There are a number of people on our committee that have knowledge far beyond my limited medical knowledge but it has been a great effort as we go forward in the 21st century. Dr. Hamburg, im going to address my question to you. Representative green, my colleague on the committee from texas and coauthor of the gain act, we are now, fction as you know, working on the adapt act to take that a step further for a limited population and a antibiotical drugs and you didnt get a chance to he diverted his question back to dr. Collins, so you didnt get a chance to really discuss that. But i would like for you to spend a little time talking about the adapt act and what that model maybe can be an example for other efforts and bringing other drugs to market and medical devices in these highly specialized need areas respect resist ant antibiotics, limited populations. Thank you for quet. Thank you for your leadership on the critical issue of how can we ensure an adequate pipeline in terms of new Drug Development in the antibiotic arena, which is ever more important as were seeing Drug Resistance spread. You know, i think dr. Collins did address a critical part of why the gain act was important in terms of needing to create new focus and incentives for Drug Development in this area. It has been a hard area in terms of companies wanting to really invest because Market Forces dont make it irresistible in terms of return on risk. Have i to be honest, fda didnt make it irresistible either because we were really asking for very elaborate, large Clinical Trials that were timely, costly and hard to recruit patients for all the reasons we talked that dr. Collins mentioned in terms of the complexity of doing these studies and being able to have the Clinical Trial ready to go when the patient appears sometimes deathly ill with an infection that needs to be treated. So i think what were trying to think about now is how, especially in the context of antimicrobal resistance, which is such an urgent problem, how can we really design regulatory pathways that enable us to get meaningful answers as quickly as possible and reduce the risk for developers and a sort of special population approach enables us to look at the highest risk end of a spectrum of patients that might be treated and narrow the focus so we can ask and answer questions where the risk benefit falls out as clearly as possible and enable us to move forward more swiftly. And i think that thats really what were thinking about there. It applies in antimicrobal resistance and new antimicrobal development. It applies in other areas important to public health, including a very heterogeneous disease category like obesity where if you can narrow the indication, the Development Process can actually be sped. Thank you. Joe barton. Thank you, mr. Chairman. Joe brought his own expert with him. Yeah. I didnt think we had enough firepower here with the head of nih and hhs and one of the billionaires whos put a lot of money into medical research, so i brought my own. This is my volunteer staff assista assistant. Dr. Yomoto, university of Californiasan Francisco who calls dr. Hamburg peggy. He said, oh, peggy is down there. I want to say hi to her. You can call me peggy, too. Im very impressed, mr. Chairman, with the panel you put together today. Obviously, to have these imminences here is just amazing. My question is going to be similar to mr. Pitts but with a little different twist. You know, we brought the deficit down from a trillion and a half to half a trillion but its still half a trillion. And with all the good intentions in the world, we and ive introduced nih reform bills and double the funding of nih back in the gingrich era. I introduced an authorization bill that increase funding for nih every year. But the reality is, youre going to get what you get, plus a little bit. Thats just so, given that, i want to ask mr. Milken, is there anything we can do to incentivize the private sector to give a match, some sort of tax break for medical research or perhaps even some sort of a government match if the private sector puts up so much, the government will match it dollar for dollar or maybe one for two or something, because we obviously i think the chairman is with me on this. In a perfect world, we would want to increase across the board but in the real world we have to find ways to do better with what we have right now. So are there some things we can do next year in terms of giving incentives to the private sector that might actually result in, you know, measurable increases in private funding in these areas . Thank you. I want to thank you for your work that you do. And im excited to see my good friend, keith yomimoto today. As hes aware, there are a number of efforts right now on the potential for a would go to citizens, foundations, corporations and the government would challenge them by offering to put up a dollar if they were matched for a dollar in the f l philanthropy standpoint. In the United States today, you are limited on your deductions to you can use 30 of your income for public foundations and 20 for private. In some other countries they have allowed you to have a Tax Deduction up to 50 if its for medical research, science, etc. I think as we saw in the efforts in acid rain and so2, that you can direct funds. I think a risk we are aware of is a number of the people that have accumulated wealth are not as focused on inheritance taxes because theyre planning to give their money when they pass away to foundations. The opportunity to accelerate that. One example i might give you is iras and 401 ks, but when iras, when you are 75 and a half you pull money out and are potentially taxed. Many people if we afforded the opportunity to give money to charity now instead of waiting 20 years or ten years would give the money in their ira and 401ks. There was an experiment where could you give up to 100,000 without tax out of your ira. It ran for two years. I think 140 million was given. But if you took the limit of 100,000 off and tried it for a couple years, you might be surprised how much of private sector money is willing to go. If you decided it needed to go into medical philanthropy or whatever the decisions were, i think you could tap that. Third, i think today the issue of young scientists i think if we had a formal program initiated, you would have a lot of funding Foreign Policy individuals, whether its through their own academic institutions that they felt loyalty or they went to, to support these young scientists. We have spoken to dr. Collins and also commissioner hamburg about the possibility through their private foundations could we help augment the funding. Many years ago i was touring the nih and saw a young scientist. There wasnt going to be funding for the scientist. And septembnt a check to cover funding for the scientist. Nine months later, they figured out how to cash the check. Were better now. But told us not to ever do that again. I told them there was another agency, the irs it bounced. They are getting better, as dr. Collins said. I think we have not appealed to the American Public enough to incentivize them. We see initiatives such as recently with a. L. S. And putting an ice bucket on your head that raised 100 million and they used to raise 2 million. In the area of men cancer, the growing of a mustache in november, worldwide now raises 150 million a year. How do you communicate one of the challenges has been, how do you get small amounts of money from large numbers of people . This has been a challenge. Companies like safeway, through their checkout program, probably have raised a half a billion dollars for different diseases, breast cancer, Prostate Cancer, muscular dystrophy and others. Average donation, 2. If you send a check in to a medical Research Foundation it would probably cost us 15 to process it. So these other mechanisms, which safeway did at the checkout counter rounding up, allow you to get millions of people involved with small contributions. I think you can, through some policy changes, run some tests here and maybe run them for a year or two and see what happens. I think the risk is in an area i spoke of early, scoring. Theyre going to tell you that its going to cost, you know, billions of dollars in future taxes in the next 40 years. But i come back to the statement that, 50 of all Economic Growth has come from advances in extension of life and increasing the quality of life. I think by accelerating the giving, we have young people in our country today that are in their 20s that are worth 30 billion. Should they wait until they are 70 or 80 years old or should we try to tap into their intellect and their funds today by ins incentivizing them to support it today . Theres a number of areas that we could test. Theres trillions of dollars in these iras. Should we wait . If you wait until you pass away, you can give it to your Foundation Tax free. No estate tax. Why dont we encourage them today to participate . I think theres a number of things we could do with private industry. I think theres another factor. We have surveyed cancer patients. We have set up so many regulations and collection of data and accessing of data. We are protecting people that dont want to be protected. 600 Million People on this planet go to facebook every day. They put information and secrets up on line that anyone in the world can access that you cant even believe that theyre saying that or putting it up on their website. More than 70 of all cancer survivors will be willing to make all their data public, all their tests public, so that any graduate ph. D. Md student could access that data. How do you waive hippa . How do you give up the rights to protect information you dont want to protect . I think theres a lot of work that could be done in Data Collection. When i was at berkeley in the 1960s and was focused on how do we access capital, for small and medium businesses, as a student with little to no money, i was able to access free the chris tapes which summarized Financial Transactions in markets under the guidance of the university of chicago covering the previous 50 years at no cost. To try to create these data sets sometimes could cost 100 million. Who has access to this data . We could provide enormous data. But i do think one of the elements is, how do you waive your privacy rights if you dont want that privacy . Well said. That buzzer means its 12 00. We are going to have a hard stop at 12 00. So we are. I want to really thank really thank everybody here that was involved in the thoughtful discussion. Not only today but in recent months as well. Your ideas have triggered us to move. I want to commend our staff. [ applause ] were not done. This is the last formal meeting this month because were getting out of session i think next week. But i know that during the next couple weeks, i know this weekend im going to oregon and Washington State and were going to have an event. I think theres an event or two in texas, in florida and other states. I would encourage our members to do that, to reach out, to continue to listen, because when we do come back, were going to start writing. Were going to try to do our best to do it right. As i said at the beginning, our goal is to move this early next year. Have a draft proposal done early next year. In all likelihood it wont be done this year. We will miss you dr. Gingrey. We really do want to do this right. Lots of shoulders were going to be leaning on to make sure that it happens. Were most grateful for the administrations support, too. Peggy, you have done a good job. You realize constructively where we need to go. Sylvia burwell today confirmed the administrations support for this. Were reaching out to the senate. Dr. Collins has been a super star not only in this room but traveling to different parts of the country as well. Of course, michael, your work, again, we stole a lot from you over the last couple years. Just outstanding work that you have done and committed in the foundation and the organization that you have has truly helped us. Great entrepreneurs, last two two weeks ago in colorado but now were very grateful for that assistance. We are goito