hope for all. the initiator of this whole program is the chairman of our energy and commerce committee, and i am very pleased to ask our distinguished chairman, chairman upton am a from michigan, for any remarks he would like to make, and then i would like to thank him for taking time. mr. chairman? >> thank you, mr. chairman. it is truly a bipartisan effort. we formally unveiled it this past spring, and it will be bipartisan every step of the way. century cures initiative is, in fact, going to save lives. it is going to impact every family, not only in this country but perhaps around the world. it is going to keep our country the leader in innovations and accelerate the display recycle -- the delivery cycle. there are 7000 known diseases, but we only have treatments for about 500 of them, and that is just unacceptable. we can, and we must do better, and we will. kindnitiatives have this of direct impact on american lives, or, as we think about our family members and close friends who have been impacted by some awful disease. for the past number of weeks, our colleagues have taken this conversation back on, soliciting information from thought leaders and others across the country. chairman, your subcommittee, as he indicated, has had six formal subcommittee hearings on chewers, and we have a number of roundtable discussions not only in washington, that this is the first stop as we go and travel around the country. we will be holding additional hearings when congress returns next month, and your efforts, tireless on this initiative, coupled with bipartisan work on helped manyu have americans, including these in the 16th district of pennsylvania. this committee is responsible for enacting nearly two dozen health measures into law for this congress, a tremendous testament to your leadership. when he looked back at the 113th congress, it will be remembered, perhaps, as the public health congress, and i am pleased to be here, and also vice-chairman and others as we continue to work together. in closing, i want to thank those folks who have contributed to the 21st century cures initiative, and we have received invaluable input and government leaders who are here today, innovators, and researchers. tell you how much we appreciate the thoughtful contributions, especially those of everyday americans, where folks can weigh in and do website cures at cures@mail.house.gov, and we look forward to this, and our goal is to move legislation early next year and get her done. you for the statements. i am very pleased to introduce the ranking member of the health subcommittee, the gentleman from new jersey, frank alone. he and i have worked together tirelessly to get these, i think it is 22 or 24, signed into law so far this year, and he deserves a lot of credit, so, frank. sorry. >> thank you, chairman pitts, and i think you know that i have many times mentioned to you that i wanted to visit -- i was told that i was not pronouncing it right. lancaster. and visit, some this provided the opportunity to come to your beautiful district, and i appreciate all that you have done working on a bipartisan basis, working to, as you say, enact a number of a quite an impressive list of accomplishments we have in the subcommittee for the last two years, during the session. i also want to mention, again, as the chairman said, of the initiative, cures it is obviously a great initiative. and it willtisan, continue to be bipartisan as we proceed, and that is important, because you really do not accomplish anything in congress unless it is done on a bipartisan basis, and this issue and initiative is important for our country, not only in terms of a cure for diseases but also in terms of job creation, and our district is one that has a tremendous amount of pharmaceutical companies, so for all of these reasons, i am pleased to be here today, mr. chairman. thank you. you, frank, and now, i am very pleased to introduce my , dr. michael burgess, from dallas, texas. has beenal expertise of significant value as we consider public policy questions .egarding health care >> thank you, chairman. i thank you for your invitation to come to pennsylvania. i have had a number of opportunities to talk to people, both in the dallas area am where i am from, as well as in the houston area, and last monday, i was at the manhattan institute up in boston, so this exercise has attracted a lot of attention around the country, and i think that is appropriate. always great to be at these roundtables. i learned so much from hearing from people on the frontlines and development, and i would like to know what we can do to make things happen faster, because that is, after all, what the name of the game is, so thank you, mr. chairman, and i will yield back. >> thank you, michael. i would like each of our distinguished guests to take two minutes to introduce yourself, and to explain what you and your company does in the health-care space, and then we will get into the more substantive discussions about what you each think about what can be done to cure patients, so, dr. collins, it is a great honor to have you with us, and we will begin with you. >> well, thank you, chairman pitts, ranking member, vice chairman burgess. it is really wonderful you have pulled this together. us to have this conversation, and i want to thank you and chairman upton for creating a space for these kinds of conversations to happen. i am the director of the national institutes of health, the largest. i am trained as a geneticist, and i have had the privilege of leading the human genome project, which has led to more precision medicine, which we will end up talking about, and i am exhilarated by what i see in terms of the potential of these advances in medical research to to givem our ability people the opportunity to live long and healthy lives, both in terms of prevention and in terms of treatment. i am very grateful though by your interest in asking the question if there are things that can be done to make this whole enterprise more efficient and able to go faster, and certainly, we will have some of those suggestions, and we have already shared some of those with you in other meetings and roundtables that i think would help nih quite a bit. that being said, i think our major concern right now is, in fact, the difficulty we have in terms of providing resources through the best and brightest of our nation who are seeking to break down these barriers, and last year, we awarded $1.4 billion in funds, with at least one grant in your district, so the brain trust in bethesda, maryland, not only, but it is and we aree country, leaving a good deal of wonderful science on the table due to the system,stress on the which is quite understandable given our fiscal constraints, but the case here is so strong in terms of the contributions to our health and economy, that we can find a straight trajectory forward, and that would certainly be a wonderful way to document america's intent to continue leadership in this space. thank you. >> thank you, dr. collins. hamburg, with the u.s. food and drug administration. >> there was huge benefit in getting out of washington and listening to people on the ground with important issues, with various perspectives, and this is a welcome opportunity to do so, in a region that is rich with distinguished research institutions and wonderful health care facilities and systems, so it is very appropriate. noted, the 21st century cures initiative has really given us a chance to come together across disciplines, across sectors, across states, and across party lines in order to address one of the most pressing problems of our air a, which is how do we advance biomedical innovation and really deliver on the promise of science and technology for the people that we all serve in one way or another. it matters hugely for individuals, communities, and our nation. it matters in terms of the health and well-being and the quality of life, but it matters for our health care systems and the health of our economy, and i think we all recognize that over a period of many years now, our nation has been preeminent in the area of biomedical research and important new audit development. we remain in that important role, and i am happy to report that fda, in fact, approves more drugs more quickly than any other country in the world, but we can and must do better in order to strengthen health and also to continue to support our nation's economy, high-value jobs, and a strong export sector that is part of this import enterprise, so it is exciting, and clearly the people around the stable and the vision that is embodied in 21st century that this is all about partnership and collaboration. it really does require an ecosystem working together with clearly a common set of goals, which is to promote and protect the public health. i am a physician trained in internal medicine with emphasis on infectious diseases. most of my career was actually in public service. it was not what i planned when i started medical school, but actually looking at the world and seen the complex array of medical problems that intersected with other social, economic, legal, ethical issues, it really compelled me to step out of the academic medical care system and into public service and public health more broadly, and i have had an approach from the very beginning that reflected the importance of ringing professional expertise whatever therts, setting, and i have had the privilege of serving with both republicans and democrats in my various government service jobs, and i am delighted to be part of this important effort. thank you. >> thank you, dr. hamburg. d, with theo biotechnology industry organization. >> i served with 12 years with chairman pitts, and for the past 10 years, i have been the of adent and ceo biotechnology industry organization. we are the national trade association for biotechnology, and we represent about 900 companies involved in drug discovery. most of them, small startup companies that are trying to make some medical breakthroughs in curing disease. i want to company for three things. first, for taking this kind of initiative, this notion of 21st entry to her's, as a major committee roger. second, for doing it in a completely bipartisan way, which we all know is too rare, and third for doing it right in terms of going out and listening to the people. he'll often, we turn to ideological think tanks, and we think that they have all of the answers, and they don't, so this is the correct way to do that. thank you for that. and in the course of our conversation, i hope to talk 's, and thehree r first is research, getting with making sure that dr. collins at nih is adequately funded, but also a large longitudinal study which we could initiate. the second being the regulatory pathway, and that involves 'sking sure that dr. hamburg organization is adequately funded so she has the tools and the personnel, and that means choices, and it can be expensive but well worth it, and also to make sure we continue to push the envelope in terms of accelerate ways to the review and the approval of products so we can get them to patients, and then the third r has to deal with reimbursement, and that is to recognize that this is a highly risky, highly expensive proposition, and if we are to get the investment, small, medium, and large, we have to have a reimbursement system that adequately rewards those risks and also at the same time makes certain that our products are affordable to all the patients who need them. thank you. >> thank you, jim. of, mr. matthews, the head reading hospital. >> chairman, thank you for the opportunity to be with you today. i am the president and ceo of reading health system, in reading. those who have the opportunity to use the products, the devices, the drugs that are developed for the curious. a 730 five-bed hospital. we have got 37 sites in the county. we care for approximately 420,000 people. are a tertiary level facility. we see 130 7000 visits in our emergency department alone on an annual basis, the most within the commonwealth of pennsylvania. we cover stroke, cardiac, psychiatric care. procedures, tavern which is the aortic valve replacement. t-var and the aortic repair, and these were developed to care for people with vascular disease. you have been in our hybrid rooms, congressman pitts, where we bring together the neurovascular neurosurgeons as well as the neurologists, so we have a surgeon and a clinician working on the same patient at the same time. surgeons,thoracic cardiothoracic surgeons, working with the interventional cardiologists at the same time, in order that we were able to decrease the impact on the through procedures that are not as invasive as others. we participate -- we are the only system within pennsylvania that participates with johns hopkins and gold research network. we are proud to be part of that. our focus is not only on caring for patients but also educating and participating in clinical research. so it is a pleasure to be here with you. >> thank you. next, the vice president and head of global regulatory affairs, dr. hanlon. >> thank you, and as you said, my name is dr. bill hanion with u.s.-based company, a commercial services partner here in pennsylvania and in new jersey as well as elsewhere in the u.s. and around the world. broadest range of industrial solutions for our clients to help them bring innovative medicines to market sooner. ce has more than 12,000 employees and delivers these services in several countries. ofare in all of your phases development, with preclinical research, as well, and we do health economics outcomes research. in addition, we operate the largest critical trial laboratory in the world, generating more clinical trial data than any worldwide. has its own internal effort right now under way to reduce costs of clinical trial development, and we believe that this effort, along with our breadth and depth of pharmaceutical development experience provides us with a knowledgeable experience to be here today to contribute to the 21st century chewers escutcheon. as a scientist and drug developer, i have spent money seven years in the pharmaceutical development, most of those in new jersey. with my 10 years spread equally between drug discovery and development of new molecules. it is exciting to be here and to be engaged in such a pioneering effort to simplify clinical trial development, and it is also a privilege to be here and share in this discussion. thank you. >> thank you, dr. hanon. -- maron, and now we go to dr. otti, the head of the lancaster regional medical center. >> thank you, members of congress, fellow panelists, ladies and gentlemen. it is truly an honor and a privilege to be invited to participate in this hearing and a very humbling experience to be among such esteemed members of the health care community. by way of background, i spent 18 years of my life at yale university. it was there that i received a bachelor of science degree in biology, the master of philosophy in neuroscience, a phd in neuroscience, and an m.d.. i completed my training in neurological surgery at yale at the hospital, and during my residency, i completed a post doctorate fellowship at fiber -- pfizer pharmaceuticals and then did a fellowship in spine surgery at harvard. for the past 5.5 years, i have been a private practice neurosurgeons here in lancaster, and part of a multidisciplinary practice that incorporates neurosurgery, orthopedics, neurology, a management, and physical therapy. our practice takes an integrative approach to treat patients with diseases of the nervous system, spine, and peripheral joints. fortune tothe good see health care multiple perspectives throughout my career. tower, big former, small-town country doctor, and i hope to draw from a very experiences to be able to contribute something valuable to this discussion today. thank you. >> thank you, dr.. uer, who iseuba chairman and ceo of braun medical. >> thank you, mr. chairman. i want to thank you for initiating this roundtable discussion, and on behalf of the industry, the medical device industry, this is an important initiative to keep the united states in the forefront of technology and innovation. assure you,, i can supports your call to action. neubauer, androll i am the chairman and ceo of b. held in the sixth generation of a family dedicated to health care. >> will be microphone closer to you. >> i will be glad to. with approximately $7 million in revenue and over 50,000 employees worldwide spread all over populated continents, b. braun is one of the largest companies in the world. we have around $1.5 billion in revenue, and five point 5000 employees in the u.s., and the majority of our products are made in the u.s. and sold in the u.s., and currently, we see our organization and our industry with great challenges. the hurdles towards innovation in our industry are the expenses and the burden to get products approved currently in the united states. it is getting more and more expensive. it is taking more and more time, and, unfortunately, it is becoming less preventable to get products approved in the united states. nations in the medical technology innovation cycle has been an ecosystem which the commission referred to that was working well, and it is in trouble and under severe stress right now, and we appreciate the focus on it to get this ecosystem back into order to keep this country back on the forefront of medical technology. and i am losing my voice, and i am sorry. however, i do want to recognize that we are addressing these issues. fda, with thehe medical device user fee act, and we're already seeing an acceleration of approvals. again it is working. fta and the industry are working together. it is also important to note the cooperation under the medical device innovation consortium under a doctor who is also working well, it is promising. and it is not enough, and we are hoping and willing to work with you to address these issues with all constituents to bring this cycle and this ecosystem back to work. there is a lot of work. and, oh, one thing i should not forget, another large threat to the medical device innovation. taking funds from small and large companies all over the country that they need to put into research and development to find the cures of tomorrow. and this will not show up tomorrow, ladies and gentlemen, but for years to come if these funds do not return to the industry and are put into work to find the cures for tomorrow. we are here to offer our help in any way we can. our organization and industry are conflict your initiative. >> thank you. and now we go to the physician and chief in charge of children's hospital, philadelphia. >> thank you. i appreciate the opportunity to participate in this roundtable discussion. as mentioned, i am the chair of pediatrics at children's hospital in philadelphia. i am a pediatrician. i am trained, as well, in pediatric infectious diseases, drawn to this area because of the fact that our middle son developed a bacterial meningitis at an early age. as many of you know, it is the oldest children's hospital in the country. it operates the largest pediatric network in the country and is ranked number one in pediatric research in the united states. like other major children's hospitals across the country, we care for children with common diseases, like asthma and and also forutism children with rare diseases and children with special health-care needs who require the expertise and thatdisciplinary it care an academic center can provide. we are also responsible for training the next generation of nutrition's, pediatric subspecialists, pediatric investigators. despite the fact that children are our future, much adult disease has its origin during childhood. the investment in pediatric research in this country is relatively limited. is nih budget portion devoted to pediatric research is roughly 10% or so. the investment by the pharmaceutical industry is relatively limited, reflecting the fact that it is uncommon to develop a blockbuster drug for pediatric care. as i think about needs for child health and pediatric surgery in this country, certainly continued investment in research is critical. the national pediatric research network act was recently signed into law in november of this past year, but at this point is unfunded. graduateon, the medical education program will remain essentail in terms of our ability to train the next generation of pediatricians and pediatric investigators. , likewiseelopment essentail as we think about the workforce to produce the work that will result in increased child health. thank you. >> thank you, dr.. to dr. chi van dang, at the university of adelaide -- pennsylvania abramson cancer center. cancer centermson at the university of pennsylvania, and i am also here on the half of a doctor at a cancer research center, which is overyzing interests with 30,000 members, and i just want to reflect on three areas. first is the u.s. commitment to invest in innovation so we can have an impact on patient lives and the economy. urge the believe -- we u.s. congress to have a plan for predictable, sustained, nih funding so we can keep a competitive edge, which is byding suddenly but tangibly china investment in their research enterprise. only through this investment can we continue to innovate, and i can give you an example of innovation that has already touched people's lives. this is the future of medicine. at the university of pennsylvania, our researchers are also able to harness your ,mmune cells and fuse them back where leukemia has no other options to kill these kenya cells, and i can tell you, this impact is clearly tangible, he gets one of the patients i have seen, and this has an impact also in children, now treated over 90 patients in a clinical trial that just received a breakthrough from the this allows us for a private-public partnership with a pharmaceutical company that wants to take this technology and get it out to treat other cancer patients who have no other options. i just want to will peel to you and the u.s. government, the congress to really invest in research so we can maintain our innovative edge and make an impact. than i do that for every dollar you invest in research, there is at least two dollars in return for economic impact with over 20,000 people in this state employee because of research. thank you very much for the opportunity. >> thank you. next, dr. norman barton, vice president of the global leader of shire pharmaceuticals. >> i am absolutely delighted to be here. shire is a large company that has a very, very big presence in rare disease. it certainly has other activities in euro science -- in uroscience. shire is a company that is made very substantial investments in places where others have not gone because the road to approval of the types of compounds that are under investigation is perceived of as too risky. riskuch capital at therefore i will not go there. level, asrsonal training as a physician and scientist -- for 35 years, i have worked in rare disease. 17 of which were at the nih. the remainder in the biotech sector. that 35 year period to me has been the most absolutely amazing diseasetime where rare 35 years ago was really rare and out there by itself. it was not paid attention to. now, it has come to more the mainstream event thankfully because of all of the scientific understanding that has emerged in these kinds of d isorders. fda, congress, the institutes in preparing a framework that allowed organizations to take a deep breath and say yes, we will invest in this. so, it really is a great honor to be here today. to have the opportunity to explore how might this enterprise move forward and deliver more on its promise with investment in it. for example, the work i do right in extremelyrogram prematurely born infants. that is a risky space in the kinds of dialogues and conversations that are necessary to move those types of programs require a broaderld acquir type of interaction. a type of interaction with the agency in which complicated issues can get talked about frequently. first in class development. what is the pathway? how do we do this? there was this need really to be able to collaborate in a very b ig way and hear all of the voices, the voices of the little people, the mothers, the fathers, the underserved. the voices of the scientists, the regulators, every voice. it is important because that is how we get down the road to deliver that therapy where none previously existed. to me, a great pleasure to be here. thank you. >> thank you. the seniorll go to vice president of alternative development. >> mr. chairman, members of the committee, thank you very much for the opportunity. to join this. i think it is terrific and really important for you to provide a forum to listen to a diverse set of voices that can touches s so many. --m a genesis by training geneticist by training. i have had an academic career in both the united kingdom and the united states. university of oxford in the u.k. and the university of washington-seattle here. do along the lines of what you are trying to explore. the novel approaches to making new medicines. majormpany is a international pharmaceutical company. we have 17,000 employees in the united states, 5000 and the commonwealth of pennsylvania, marietta.one in m we focus on consumer health care products. we have five sites in new jersey. we are delighted to be there with you. today, i would like to highlight three themes. i heard a few words here that really provide an umbrella over the themes. those words were opening teamwork. i heard collaboration, partnerships. that is the umbrella for all the things we would like to discuss today. in particular, those three items -- one is the importance of the digital era and electronic health records and how we can maximize and harness the potential of that type of data. power of theth the biological information that is coming out of the genome revolution. that will take partnership. providing as sustainable framework for the national institute of health and a way to draw that work that they are doing internally and externally with their funding in a more seamless fashion to translate into new medicine. note that you to partnerships with ebola vaccines. finally, we would like to very much underscore the importance of antimicrobial resistance research and the importance of that worldwide. .> thank you finally, we have mrs. jennifer westdyke. she is representing the pediatric foundation. -- is a parent of a child >> thank you for having me here today. i am honored to be a voice for my son who is eight years old and the other patients who were born with or develop an illness that is incurable. placed when shunt he was three days old and all too often as a parent, you sit back and wonder -- it is not if it will fail, it is more when it will. this is a 50 plus-year-old technology that has a very high failure rate. my son has been very fortunate and had not too many revisions. he has had three but there are children his age who have had 40 which means 40 bring surgeries. this certainly is having an impact on their lives. you operatees can on a child's head without there being some sort of brain damage on top of the fluid building up in the brain. it is scary and it is scary for any parent who has a child with any illness without a cure. i am so honored to be a part of ideasnd to hear all the and the breakthroughs. hopefully, this will have such a positive impact on my son's aslth and his future as well all the children that suffer from any kind of illness. i am honored. thank you so much for having me here. >> it is an honor to have you here. i would like to thank all the panelists for their remarks. in the remaining time, there will be a free-flowing dialogue between the panelists. we hope to allow time at the end for a few questions from the audience. most of the panelists will remain after the event to answer questions. i would like to begin today's discussion with this question. dr. collins, i was asked you to address this. what steps can be taken to accelerate the discovery delivery cycle to foster innovation, bring new treatments that cures patients and keep more jobs in the united states? >> thank you for the question. what a wonderful panel. it is wonderful hearing these introductions and i think we are very much coming from a similar perspective and very appreciative of the chance to have this conversation. we thought a lot about this in light of the 21st centure cures initiative that you all have been leading. i think there are a lot of things that congress can do to accelerate this discovery development delivery cycle. maybe you can break them down into three major areas. one is to enhance the discipline protection -- participant protection and increased transparency. in that regard as a specific example -- as the personalized medicine revolution is coming forward quickly and as sequencing machines have gone from being the size of phone booths to being the size of postage stamps and the cost has come down from hundreds of millions of dollars to close to $1000, patients are increasingly interested in having this information, being able to take advantage of it for their own personal care. but, we need to be sure we have adequate prohibition against unauthorized re-edification of individuals based upon genomic information which currently is not properly prevented. furthermore, we think there should be a prohibition against surreptitious or nonvoluntary genetic testing with perhaps some exception to law-enforcement. there is a need there as well to provide confidence that this information will be used to the benefit and now potentially in some other way. that is an example of areas involving patient protection, increasing transparency of data we produce. a second area would be the facilitation of scientific collaboration and innovation. while it isrd, understandable that the congress has had considerable concerns about conferences and expenditures on travel, particularly given there has been some egregious examples of misuse of funds. the gsa extravaganza in las vegas for infants. the way that is now being implemented it provides a very heavy burden upon those of us that are trying to organize scientific conferences and make sure scientists have the ability to attend them. i can tell you for my 30 years of experience as a physician and scientist, many times and some of the most exciting ideas that have arrived in mine own career has been on the basis of those conversations that happen at these meetings where you rub shoulders with people who were not at your own institution. andght bulb goes off another idea get started and something important for the future gets to occur. i must tell you the way in which oversight of scientific conferences is being applied at nih has caused us to spend 13 set -- urging $.7 million last year on the enterprise and involve more than 150 employees to try to track, maintain some kind of record they could be scrutinized by anybody that wants to see. all the conferences we want people to go to. we were to really lose money because it is so complicated when you have to put together people. you optimistic early registration opportunity. it is not a system that is working right now. if there was a way to get relief from that, that would be an enormous pat on the back for the whole enterprise because people are deeply troubled about this set of bureaucratic oversight which i think accomplishes almost nothing and yet costs time and money. reduce administrative burden. one example -- the ironically named paperwork reduction act which has caused more paperwork than anything in my experience will prevent a researcher in our integral program who is interested in public attitudes towards personalized medicine and wants to carry out a well-designed survey that has been reviewed by an institutional review board and is gone through all the appropriate oversight. they cannot do that if they have to want to ask more people a question because otherwise it has to go through a clearance which is going to be many months and by that time the trainee has gone on to something else. i am sure the authors of the paperwork reduction act did not have this in mind but it is really getting in our way. to have those kinds of relief would help. enhancing participant protection, facilitating collaboration, reducing administered a burden -- those are areas that we have many suggestions. chancery happy to have a to provide more examples as part of this process. thank you. >> thank you, dr. collins. any members would like to ask a question? >> thank you. thank you, mr. chairman. what arented to ask the concerns -- i think we can use it in terms of general. often -- you talk about the shunt. how a lot of times you need to have multiple surgeries. i do worry and i know you've expressed it to my staff about the whole issue of, you know, cost to the family and insurance and coverage because we tend to clearly, there is more need for research and i know we need to deal with pediatric research. cures.us is on i do worry like you talk about your son -- the initial operation is covered but then maintenance of the shunt, having these repeated operations, but whole issue of access for this type of care. do you want to comment on that? i know you did not really do that and i know you talk about it a lot for the association. >> of course. closer,ou pull that up please? heream sure that nobody does not understand how the insurance industry works. they do not cover much as far as routine checkups. a lot of times an mri has been declined saying my child does not need to be checked once a year. our children are certainly impacting the cost of health care because of their repeated surgeries, their need for cat s.ans and mris and shunt the insurance doesn't pay for it. it is coming out of our pockets. it is very costly. >> i know you represent the association. have they talked about some way of remedying this in general, not only in terms of research, but in terms of access as well and follow-up? i am not just talking about insurance overall. have they made any kind of recommendation? theseole point of having roundtables is anything we can do legislatively or otherwise to make a difference. >> funding research, that is a big deal. association is underfunded. i don't think many people think about it. one in 500 children are born with it. those numbers are probably much higher because that doesn't include the children that develop it after they are born. thingsre some different and the adults that acquire it. it is severely underfunded which leads to very little research to change anything. that is really our mission. the foundation is trying to help provide information about this and so people know the numbers are far higher than they think. >> i am not trying to monopolize. i know when i listen to the about -- you talked pediatric research in terms of cures. that seems to be even more underfunded than anything. it always shocks me but i know it is true that when we deal with the children whether it is research funding, care. oftentimes if alter the cracks. -- it falls through the cracks. do you want to talk about comparing it to researching funding in general and maybe tell us if it is true? that is what i hear all the time. >> i made comments about a few facts in my introductory remarks. namely that a portion of the nih budget that is dedicated to research is less than 10%. it isight argue that appropriate because there is much more disease. emphasize, we are learning increasingly that adults that origins in pediatrics during childhood. in addition, we know that children represent the future of this country. my own perspective is that it makes sense to at least increase the investment in pediatric research, increase the fraction of the budget that is devoted to pediatric research. approach highlight an that underscores the point that dr. collins made about teamwork, partnership, collaboration. area inink about an pediatrics that is arguably been bothost successful model in pediatrics and as an example for medicine more generally, the children's oncology group which represents a network of pediatric centers across the country with over 200 centers has beenpating remarked -- participating has been successful in bringing together investigators and bringing together populations of patients from across the country to study those patients and develop new approaches to treatment. the consequence has been remarkable advances in outcomes in most pediatric cancers. i think that model is a model we should think about more generally in pediatrics and in medicine and biomedical research broadly speaking. again, i highlighted the national pediatric research act which essentially proposes to create consortia that will be like that oncology group for other disease processes. >> dr. hamburg, you wanted to add something? i just wanted to build on the important issues just raised in terms of opportunity for 21st century cures moving forward. one is the importance of really looking at how you incentivize research and product development in critical areas that are under addressed. to really move the dial. i think we have not actually done enough analysis into the incentives that are out there and which really work and make a difference and which don't. we have got areas that have proven their value, including the orphan drug act which really led to a huge expansion of research and development and new products in the orphan drug arena. last year, the new entities we approved -- a very large percentage were in fact to address orphan or rare diseases. it partly reflects an incentive framework that has been working. a new incentive program to try to stimulate pediatric therapeutic development. i think we are beginning to see some movement in that arena. i also wanted to underscore in relation to what ms. westdyke was talking about. we're seeing new focus in bringing the patient voice into both research and product development and the regulatory process as well. i think that is very important that there is feedback -- the patient experience of the disease, what really matters, what is lacking in the current products that is needed. then, in terms of our regulatory review, how can we really look at some of the measures that matter at that might not be the measures that we as scientists with think of but are really important for the patient experience. and integrate those patient reported outcomes into the regulatory assessment of a product as well. i think there are a lot of interesting activities, some of which are under way and need to be expanded and some of which are still in need of more development. thing atrd that same our last roundtable. patient feedback. i want to ask you dr. barton, do you and shire feel the pediatric priority review voucher program is an effective incentive to promote treatment for rare pediatric diseases? is there anything that can be done to make it more effective? >> thank you for that question. i believe, we as an organization believe it is a very important incentive. to put the spotlight on research in children. it de-risks it at a certain level because there is an incentive. that becomes a very important as companies look across their kerf oreos -- their portfolios and decide where to make investments to better the health in our nation. that, in my mind, is a big deal. how it could be made better -- right now, it is a provisional or temporary trial. it would be a stronger statement if it was permanent. invest andle want to make incentives and opportunities for research with children. while i have the microphone, one other thing i would like to comment on if i may -- i am impressed with the comments of that dr. hamburg made about the voice of the patient. i think, as do many of my colleagues within shire and across the industry, in the rare disease world, the voice of the patients or the patient organization is usually informative. patientsoften times and organizations who represent them know more about their disorder and what really matters than any of us. highly schooled physicians or regulators, scientists or whoever we are -- they are very well informed and sophisticated. withdustry engages regulators, with the fda, bringing that voice to the table -- not every time but in certain --uations where the pathways the road has never been laid out before, that voice is just critical particularly in the effortsn of -- the the fda and congress have gone through to enable dialogues between industry and the agency are commendable. i think they are superb. fast-trackrograms, special protocol assessment, all of it. they don't incorporate that voice. i think, personally and as to my colleagues, it would really facilitate moving things a little more rapidly. >> thank you. we will go on this side of the table. chairman upton. >> thank you. i appreciate all the comments. you have done an outstanding job of getting some witnesses that we can really listent o to. we want to make sure we end up with a good product. dr. collins, your comments are right on. we want to help you on those as we proceed. i am interested in exploring a little bit on the genomic education issue that you have talked about. we have made great strides in the last 30 or 40 years as we move forward on that. consider -- i had a very good conversation with jim greenwood as we spoke privately about where we should go with the 21st century cures. really theidea that federal government would be the only one that would be able to pursue this and that is to really come up with a nationwide demographicspes of and then use that by interfacing it with advances we saw on genomics to really help industry find faster cures. you not onlyeld to thenscribe it more, but get the comments from those of us at the table as it relates to ide as to how that can be useful or constructive. >> thank you. the entryway to this description -- think about alzheimer's. everyone knows it is a tsunami of suffering. it is typically might generation. the industry has spent hundreds and hundreds of millions of dollars and almost without exception, very expensive clinical trials have failed. part of the reason we have failed is because by the time patients develop symptoms of alzheimer's disease, they have such neurological damage it is almost impossible to reverse. what is clear in that disease as an example -- we need earlier oh biomarkers. we need to understand at an earlier date. we need to see this light and subtle changes of the body's chemistry that can enable us to predict the vulnerability towards alzheimer's and intervene sooner with a drug. in order to do that -- by the way, this is applicable to any disease you can think of -- in order to do that kind of thing what we need is a very large and very long longitudinal study. the magnitude of a hundred thousand people in differentiated by age. a 21st century version of the study which was started in 1948. we would sequence the genomes of all of these people. we would apply tests to fluids and their bodies. we would probably do an mri. we might do cognitive and functional tests for these people over time. what happens is that when people do, in the instance of alzheimer's, develop the symptoms, you are using all of that data and can go back and correlate of these early genetic and other biomarker precursors and then learn from that. thought with alzheimer's in mind but it applies to als and parkinson's and cancer. this is a product of such magnitude that the private sector cannot do it, only the federal government can do it. i would argue they should do it. inave read dr. collins' mind this subject every time we converse. takeys -- as soon as you care of the rest of my needs, we will talk about the next big science project. as i said to both mr. upton -- we need to be an advocate of meeting his needs and the academic needs as well. think this that, i is a big project of data collection. so many other enterprises -- academic, commercial, private could dockstigators into this mothership and upload their data to enrich the total database and download data from this set to enhance their abilities to further their research projects, whatever they may be. this is not a project that would be a low-cost project. you are talking about $100 million a year. when you think about the trillions of dollars in costs that can be avoided just with alzheimer's, let alone other diseases, it would pay for itself in spades. the people that participate in this project but also learn how to better tended to their own health and thereby saving the health care system dollars. in the verye not distant future, everybody will be doing this. everybody will have the genome sequence at birth, everyone will have this kind of early diagnostic tests done. the whole society will benefit. >> dr. collins, if you would like to elaborate. and it will like to throw my arms enthusiastically around my colleagues proposal. we have talked about this. i do believe wall this would require an upfront investment, it would more than paper itself in terms of providing a platform for all kinds of clinical research that currently we have to set up and here you would have an infrastructure that would allow you to pose all sorts of interesting questions at a much lower cost. i also think this has a real potential if it is set up in the way that many of us would like to see -- having patience right in the center of how this was organized. patients would basically be empowered as being part of this to learn a lot more about the road health care. there are so many gadgets. i will waive this one which is an ekg that is attached to the back of my iphone. it will give a readout if i put it next to my heart. there so more mobile -- there so many mobile health applications. you can imagine -- providing those kinds about locations, i think a lot of private sector produces would be very interested in having them in additionu could, to genomic, yet monitoring of individuals in a real-time basis. the quantified self movement which allows people to learn about themselves in a real-time way. this could be transformative. the only thing that i say is wrong is that 100,000 is too small. really, if you want to a power in numbers, when you start to see the production of cost for things that are happening. , i think we should go for one million. >> anybody else like to respond? >> i put your ekg on my heart and it is fluttering now. doctor? >> that is the future. that is where it is going. that is where we need to go as a country. something ofcing that magnitude when it is time to do it right is absolutely something that could set science to a new level. very exciting proposal. i thought i would take and endorsed that in a more monday way. -- mundane way. some of the barriers we are facing today with new breakthroughs for new treatments are really practical. the types of things that foster innovation in this country -- very desperate data sets, providers, different health-care settings. they do not speak to one another so the data is not connected at all. we have different access for different groups. technical, there are cultural challenges, incentive differences. we need to find a way to bring together large sets of data in a way that can be mined most productively and respectively. such an endeavor that was such proposed would provide a mechanism that causes us to solve the problems along the way. that by itself would be a great benefit to society. i am very enthusiastic about this proposal as well. i think it is reminiscent of the national children's study. i am very interested on hearing dr. collins' thought about the study. and wonder about how this fits in as we prioritize federal funding for research and individual investigators versus studies like this. question.appropriate the study that was authorized by the congress back in the year 2000 has had challenging periods of redesign over the course of more than a decade. with the goal basically to follow 100,000 children ideally rifled the beginning of the pregnancy so we could assess what the events might have happened during prenatal life that would be important to know about her future health and then following these kids do a 21. it has had lots of challenges in terms of what would be included, how can the sampling be done. oft recently, the institute medicine asked by the congress to do a review of the current plan raised questions about whether the plan would achieve the necessary goals. that is why we decided it was not a good moment to actually push start on what could be a very long and complicated and expensive 21 year program. i had a special working group of my advisory committee to the deantor who is a retired of stanford and a pediatrician to try to really look at what the opportunities might be. my sense is the design of the children study could in fact a greater advantage now with things that happened more recently, like the advent of various technologies like genomics and electronic health records. there may be ways to do this kind of study which i am totally committed to making sure we invest in in a way that would be more o efficient. i take your point. about large studies, there are all kinds of ways you can be quite successful. birmingham is a good example. we need to learn from those lessons. anhink we are at exceptional moment where we have for the first time the ability to design such a cohort that does tap into electronic health records which are now available for many individuals who could then participate with much less trouble in terms of getting information about their medical histories and their experiences with health care. we have technologies like genomics which used to be relatively expensive. i think we have a lot of momentum of an argument that we made that while this is hard, it will save us money in the long run. but all those things together and it seems like you would not be doing the kind of leadership we should without taking that seriously. other countries, china for instance, gets this and are making investments of this sort. i do not think we will learn everything we want to know about american health by waiting to see what the chinese results look like. i am not even sure they are necessarily going to be made immediately available. we should have an american effort of this sort to teach us about our own population. this is an exciting proposal. studies like this on a large scale could produce a tremendous amount of data to really allow us to analyze aspects that have been mentioned. i think it is important that we don't lose sight of what we have already accomplished on the data sets that are already existing for medicines already approved, that are contained in in the databases at the fda. the u.s. is the only country and the fda's the only agency that requires data sets to be submitted where other other country only requires clinical study reports. enormoustting on an amount of data that is not accessible to the scientific community in any arrogant way that aggregate way -- in any aggregate way. in addition to that, there are many studies that are done every year as part of the process of molecules that never make it to becoming approved drugs like failed studies. we have more to learn from our failed studies than we do from our studies that have succeeded. having a way to have access to this data in a way that the scientific community can mine will add tremendous insights on study design, the appropriate endpoints. of lookinghe idea forward but we should not forget the enormous amount of data that already exists that we are not mining in any meaningful way. i was going to be more pessimistic about the ability of a large federal government run trial. you touched on something that i wanted to ask dr. collins about we came upu showed -- to nih several months ago and you showed us some of the things you are doing with repurpose and molecules from failed drug trials. you actually had a couple of things that were intriguing. could you speak to that a little bit? >> i certainly will. thank you for the question. moleculeshundreds of that get tested for therapeutic benefit in a particular disease over the course of a decade that do not turn out to provide the benefit that everybody hopes for. the failure on the basis of lack of efficacy is still over 50%. those are compounds for which there has been enormous investment already made in terms of understanding that they are safe, knowing what target they hit in the human body, knowing what goes to give appropriate tissue levels and so on. when you hit that failed phase three, thatephase is usually the end of the line. they could have great value if you can identify a particular disease that this particular drug hits the right pathway that you know has been involved in that disorder. as we're learning more and more about molecular pathways and those 7000 diseases that were mentioned, you can start to play matchmaker and identify a new use for an old drug. we set up a program which many people thought was going to be pretty difficult because of the issues but ultimately it was successful. to invite companies to open their freezers and make available compounds that have but face to and phase three never achieved fda approval because they didn't get efficacy or there was a change in the -- 58 suchan compounds were made available by a different companies and we are now about a year and two months into the effort of seen whether those will in fact turn out to be of great benefit to other diseases based on crowd sourcing the idea on these new applications and writing people and universities and small businesses to say that compound may work for this disease, let's try it. i don't know what the home runs will be. trying to build on this profound investment that has been made in these molecules and see if new use can be found is going to be very helpful. there is one hitch that i might talk about because of the intention of the 21st century cures. that as many of these compounds are near the end of their patent life. if we were successful in finding a new use, and might be difficult to convince the company to do the trial for that new use because where are they going to recoup that investment ? some way to do what that would use patterns but it is certainly an area which some creativity might be thought about in terms of providing an even greater incentive for this kind of repurpose them to succeed. -- mr.a chairman chairman, thank you. just on the concept of the large and i know wetudy have had discussions with dr. collins and dr. hamburg before -- there are private sector entities that are working on the same problems. 23 and me is an example of that. -- fulls closure disclosure, i am a full participant in 23 and me. when they first started e-mailing me and filling out the survey, i thought what a nuisance. everyday a few minutes filling out one of their surveys and you realize that over time you are making a lot of information available. now, you were filling in some of the background for them. dr. collins, when you mentioned about the protection for the data, i would also offer that people are concerned about privacy but it has to be a two-way street. we are willing to give up all kinds of information on twitter and facebook. my constituents do it all the time. give upre willing to information as long as it is a two-way street. the thing that concerns me about a governmental structured study would be that it would be almost out.sible to get data back whatever happens, it has to be -- the interface has to be extremely user-friendly to allow the participants to actually also be recipients of the information that could be critical to them. >> i totally agree with your point that this will not succeed unless participants are really full participants and have some benefit from this in terms of being able to get access to the data that is generated upon them. we have talked about this. it might well be we went for one million individuals that many of them will be recruited from 23 and me because they have artie had a positive experience with this kind of connection with their genomic information. i don't mean to suggest that this would be some heavy-handed lockbox of federal information at all. i think one of the advantages of this being run by the government might well be an insistence upon open data access because that would be the model we would have to follow. that is the norm for everything nih is doing. i grant you that is not going to be trivial and people may be a little skeptical about the idea that the government is running this enterprise and it might make more sense to contract it out. somewhere, somehow somebody have to get it started. i think in our long history of successes at medical research hiv-aids in terms of treating people in the developed world successfully, the government's role has been successful and it needs to be here as well. with partners, of course. >> cms, probably one of the holders, it is almost impossible for the average person and average physician and hospital to interface with and get information back out. just to respond t o dr. burgess. only the federal government can afford to do this. i think it would probably be to great advantage if we can figure out how to set this up in a way that avoided the bureaucracy induced problems of government in terms of time frames and so forth. so i think a private public partnership is probably in order. i also think that there are ways to partner in the cost. users of the data could be charged to use the data and they could pay for that data either with dollars or they can pay with contributions o their own data into the system. and in terms of it's already being done and so forth, those are microcosmic by comparison. they're not systematic. nobody gave me an m.r.i. . body's testing my fluids this is a mothership in which all these other projects can dock and trade data. and that's why think it exclusively has to be funned at least and managed to some regard by the federal government. >> i was actually originally oing to comment on dr. anlin comment. this one addition i wanted to existing it's an data, it's critical that we can what questions need to be asked and design the study that we get meaningful answers. that's so important. and there's a lot going on now in the area of clinical trial design and really trying to be able to ask and answer questions in ways that require smaller numbers and -- and shorter trial designs using bio markers or adaptive kinds of approaches and also using existing data. and the point i was going to originally make in the position in the repurposing effort that we've talked about, that as the sciences advance an we know at diseases are not as homogenius as we once thought. there are existing data bases once we know more to go back and reexamine and get important new answers, you no, studies that might have looked at a given condition with a mixed population even though any technically had the same disease. you might wash out your effect. but if you have a bio marker that tells you that you have subset of people that are more likely to respond or more likely to suffer serious adverse affect then you can star to tease it out and take advantage of work -- data that all right exists to get new important information. and i also, you know, real write think that -- really think that the importance of being able to learn from failures is so important. and it's frustrating to me and i've spoken to this on many occasions. that sometimes we working with and work with legislations and companies need to be part of this process. i think that dr. carson's company has been at the forefront of making this clinical trial data more publically available. but there obviously has been reluctance in some sectors to do that. but this is an important time to find the barriers and what are the opportunities to existing data. >> i just wasn't to comment on one of the barriers for these very large studies which i felt alzheimer or cted pediatric. >> if you want to know when a drug goes awry you can go to norway and get this done. because there's harmonization of electronic records. we don't have them. even within the same health record, there are disparate records. if there's a way to harmonize electronic health records that's the only way we're going to achieve some of these big goals. >> excellent. >> turn to the ranking member for questions. >> thank you, plch, it's hard -- thank you, mr. chairman, it's hard for me to not get back to the issue of resources and my resources are cut at the federal level. you always mention, i always talk about keeping up with china. i guess i wanted to ask, and i can star with you and dr. collins and others mentioned that, how bad does it get -- in other words when we talk about uts at the federal level and maybe end the cuts at the state level or at the private level, i'm not just talking about the tral government here. but overall, how bad is it in terms of us keeping up with china vs. other countries? do we continue to deteriorate looking at not just the federal government for also collectively u.s. expenditures on research. >> thank you very much for the questions. i can tell you that -- this our cutting edge is getting a little bit more dull tangibly. just to give you an anecdote. a previous trainee who went back to china as a professor actually called me and said, you know, work with me because there's more money for research here. and that's just within the last month. so the sense of the chinese investigators being very well funded is really there. and they really are getting people to go back to really build up the research empire. our country has been built on a . mber of immigrants we have to make sure that we have resources, attract that len, retain talent. and the other issue that we are confronting in the academy are young people. we have a lot of good, young ientists who are now discouraged with cuts being imposed. and many of the talents are going to go somewhere or are going to do something else. many ideas in these young people will be gone. i gave you the idea that the innovation in terms of the person ells and the that was a young investigator, that idea would have been gone and not implemented. i think that short term fixes will have long-term consequences. i think that we really need to continue to informs in our research enterprise. >> ranking member, i thank you for that question and adding the private sector to this issue. >> the highest respect to the n.i.h. and cutting their funding is going to hurt medicine in the future and well as cuing funding for private enterprises. companies have just again to the medical device tax lost funds that would go in large part to the medical research. getting a product approved in the united states costs much, much more than any other country. those are also funds that are not available for pickup truck research and development. we are working here many the united states sometimes on first generation pickup trucks that the europeans are already using in -- and they're second nd third generation. go to europe first. do your clinical trials there. earn money there. get reimbursement there and when you have the cash flow then go to united states. that is a trend that really concerns me. it is taking us off the forefront of medical device innovation. we have to work on those. these basic things, i love these dreams that hopefully will turn into reality, dr. collins and mr. greenwood. i sure do. but we have some basic thanges we will work on that i hope will find the initiative's attention. >> and that tax, 2.3% is on total revenues, no just on profits? >> yes. >> it's money you're still paying. >> even if you don't make money you pay this tax, mr. chairman. it's truly off revenue. >> doctor? >> i just want to emphasize the point that i think we are at risk of losing a generation of investigators in bio medical research, building on the point that the doctor made about young faculty. we are seeing an increasing loss of junior faculty as they're unable to make the transition from a mentor career development toward an independent investigator award because of the challenges at the n.i.h. level and younger individuals see that. so we have a forward stream of individuals again to bio me cal research so the bottom line is we have a decreasing number of young investigators. s we think about the future of biomedical research that's a critical issue. he n.i.h. has made significant efforts to accommodate junior investigators by making certain allowances. but the bottom line is we do not have enough money that's been allocated to the n.i.h. and if we want to support ongoing research, if we want to have a future of biomedical research in this country, we need to think more about junior investigators and how to support that group. >> ok. we've got three. let's go down the line. dr. collins? where? we have another one over here. go ahead, dr. collins. >> so just some numbers to back up the conversation which is clearly the one that wakes me up in the middle of the night. if you look at n.i.h.'s spending potential, our purchasing power for research which is basically the appropriation and the effects of bio medical research, the bird pie as we call it, compared to 2003 to right now, n.i.h. has lost 23% of its purchasing power, almost a quarter. the consequence of that again just numbers that reflect why this is such a tough time to be a young scientist or a mid career scientist, it relates to what your chances are if you relate your best ideas in a grant proposal and ask our best peer review system and ask what priorities you assign to it and are you going to get funded or not? carl june who set up proposal in the past would have been considered innovative and ressky. might have gotten a score that eflected some ambiguity to the peer review people. but he would have gotten funded. he did get funded. your chance of getting funded has shrunk down to 1-6. it's about 16% is the success rate. where as we're virtually all of the last 50 years until 2003 that success rate ran about 30%. so i've looked at grants that scored in the 20th percentile or the 10th percentile or the 25th percentile. i can't tell the difference between them and neither can anybody else. we've done that experiment. what does that say? we're leaving half the good science in the united states on the table. and the people who have proposed it go away discouraged and some of them give up. 18% of young scientists were considering leaving the united states to go to another country. and other countries as you've heard are increasing their support in some instances dramatically, china going up by 20%. compound that and you can imagine our predictions are in another four year china will spend more on biomedical research than the united states not as a percentage g.d.p. but in absolute dollars and the consequences of that will be very grave. >> this is a great discussion and we are listening well. i just want to report to you that during this august break sent a number i of the members over to china. i don't know if you've talked to joe barton. joe -- all right. i don't know if that's a good or bad thing that you don't get texas radio in michigan. but i look forward to hearing back from joe and some of their staff that went with them to find out precisely what are the chinese are doing that we're not. and that will be part of the equation. one of the reasons that we deemed it a priority. >> to respond to the congressman's question resources and to echo some of the previous speakers, the pathway to curing a sick person begins with the basic research that congress funds and that dr. conse' organization takes into academia. it goes into some $37 billion. 23% needs to -- there's not a number that i wouldn't support in terms of the n.i.h. then comes the $50 billion a year that my industry spends to take that basic knowledge and to con ver it into real medicine. and where does that money come from? that money comes from two sources, sales of products -- existing products. and investment in venture capital and by people who buy stocks in the open market. those investors don't make those investments for the most part because they want to cure disease, they do it for return on investment. this enterprise is among the most risky enterprises there is. most projects fail over an over again. the expenditures of money is enormous. most of the pickup trucks that dr. hamburg's organization never even return their investment. and so in order to continue to attract all of those resources into the process, we have to make sure that those investors have confidence that this is a good product. it will be approved in a timely fashion and that it will be reimbursed in a reasonable fashion and if you look at the -- i hate to do that but if you look at the case of sovaldi that cures hepatitis c, it will save american society enormous ams of numbers of dollars. it's going to cost to pay for that drug but in the long run it is because of successes like that investors will say yes, let's put more money in this. >> thank you, chairman. i just want to bring it back to the idea of medical records. this idea ha multiple layers of benefit to health care and the development of new medicines. network to truly electronic medical system in the u.s., it will not only benefit from following forms of veg lens and safety of patients but it would also be a network for health care providers to find patients in terms of demographics and stages of disease to collaborate in terms of treatment and health care and finding out what has worked. what hasn't worked? and how can that be done in a more systemic way? according to lay bedtime and also not according to label to see what kind of adverse events are related to things that they were intended more or otherwise with the way physicians are treing their patients. but if addition it could also benefit the cost toward clinical trials. right now, you have your medical record whether it's electronic or not. it could be paper based. and you have a data collector that pharmaceutical companies use to collect data for the trial. data is entered separately and then it has to be entered to verify this source document verification. if the medical record became the primary source of the initial data industry, there would be no need for that. data would be easily uploaded. there would be no need to clean data which is part of a process to look for data that is missing. this would have tremendous health care but also on cost of clinical trials. >> just before we move completely off the question of the n.i.h. funding, i thought i would raise one point from an industry perspective. basically every major farmer has reduced their d.n.c. and all of those farmers are trying to find better ways to work with academic investigators so that really both sides play to strengths in a collaborative way. chemistry and clinical trials enter acking with regulators work together with those academic investigators to bring out basic science. among those creative ways are emerging some threeway partnership with the venture capital community. so we're creating jobs together with those basic sciences that are n.i.h. funded. to the ex-theand that goes away we go from job creation to job reduction because those creator noss longer have an outlet. so everyone loses in this case. the pharmaceutical system the pashes with the innovation that comes with creative partnerships an young investigators have fewer outlets to go forward. >> thank you. dr. hamburg. >> dr. moratti. > we went over this at length. >> let's see if we can reach. >> i want to chime in. funding for young investigators because i've basically lived through the se nar yes that's been described. the federal government spent considerable amount of money to train me as a physician scientists. it was very difficult to secure funding early in my career. i went in an entirely different direction. certainly there were a number of colleagues who i thought were extraordinarily talented who also chose to pursue different career paths because of difficult with securing funding early on in their careers. i just wanted to bring that oint home again. while we're talking about funding one dread that i keep hearing through all of the discussion is cost, the cost of about on we can talk electronic health records, the available of data for that for both comparative as well as predict of analytics. but the hipa law is going to restrict access to that. it started out as the health for the portability purpose of being able to move from one insurance to the other an protect that and so that the information was available, the regulation that has come from hipa is strangling. research as well as the able to compare the health an it will -- analytics there. it's like the patient protection affordable care act. a little over 2,000 pages. it has spun more than 700 regulations and the regulations exceed 2,100 pages at this point and i would asthume continues to grow. so when we think about funding also think about the ability to reduce cost so that we are able to use the dollars better than what they have been in the past. >> dr. thank you. thank you, dr. hamburg. i wanted to ask a question. are there currently any devices? bout the question is how can we insure that truthful, nonmisleading information about real world safety in this reach is doctors and patients as soon as possible. you know, what role do you see for companys that have dealt these projects or products playing in the process? >> well, first, let me comment on how important the ability to monitor products in the postmark setting is and whether it's a drug or a device the able to really have that lifespan approach has been enormously valued to the f.d.a. and congress has worked with us to give us the tools and the authority for a more robust post market individual lens approach and we appreciate that. many of the comments have been made about the inner openerbility of the electronic health records obviously speaks to the able to do post market surveillance in as effective a way as possible. but we do have systems in place stronger now on the drug side than the device side to be able to monitor many pashe lives an experiences with pickup trucks in the post market setting and -- with products in the post market settings and we're in the midst of expanding those ables. as you're looking at the premarket approval, if you know you'll be able to follow in the post market setting where you really learn more about the product than you possibly can with a limbed se of clinical trials even if they're large clinical studies, they don't give you what the real world experience does. so that's very, very important. and we, you know, really central ability to do that monitoring and sometimes ask companies to do studies in the real world. in terms of having to communicate with patients and health care providers that's a bigger question in many ways because it really does involve the partnership of our having the information and making it many ble and includes others helping to get the information out. i would say one thing that is porn to it is that f.d.a. has increasingly realized our critical role and rather than operating sort of in isolation really trying to work more closely with our stakeholders and partners to get information out whether it's patient groups or hale care providers or professional societies or the media, but that has to be, the communication issue has become, i think, really ones where there has to be this two-way communication. to eed to -- to be able provide the best information, and using all the tools of the media that are now available to us. we also need to get that feedback back to us about where the problems are, where the gaps in understanding about appropriate use because you can have the best drugs possibly and if they're not effectively used they don't provide benefits and they can even provide harm. >> jim greenwood. >> i took your question to be off label -- >> i didn't. >> it may not have been this question but i'm going to run with it anyway. [laughter] >> when a drug is approved, it has a label and it's clear ha the company who makes that drug is permitted to promote it patients consistent with the label and not to push -- because the product has not been approved for other use. to use a drug off the labor and beyond the layable. so what happens is sometimes companies are -- and with sales forces commit violations. and for that they are frequently fined and fined in huge numbers billions and billions of fines have resulted from these cases. have a system where and a former employee can blow a whistle. they can get a lawyer. and then under these type of situations they can make the lawyers and the whistle blowers can take huge percent of what could be a billion dollar fine. then the company litigates and loses, they can no longer have their drugs reimbursed by the government program. so that's death a company. they settle. something -- we need to protect against bad behavior here. but i think we need more clarity from the congress and from the f.d.a. as to where the lines reasonably should billion drawn to avoid both ills that drive from an appropriate promotion and the ills that are derived from overpenalizing behavior by some portion of a company. >> and just to elaborate, not speaking from a legal point of view off label use is a reality and in many areas in forms of fed cal information, dr. deng could probably speak to on off-label use. if we could really move towards the vision we've been talking out, more integrated uses of record. it will be helpful to advance medical practice and the way the system currently works, it's usually not in the company's interest to do the kind of data collection and studies to bring a product that's already approved for one use for the f.d.a. but in fact, it is very important from your protective medical care to know as much as we can for those drugs an their use in other medical treatment ttings than just what it originally. a no no word in my world. let's me go back for the patients, i think the f.d.a. is doing the best job in the world. and their warning systems with or without post surveillance. post surveillance is appropriate. and a new tool to be used in the matter. but i think the f.d.a. is doing an excellent job. >> venture capital going over to the u. -- empleumple. with's their record? >> it depends on how they measure it. they have a different recall system, but all in all, i would claim to say that i don't have any scientific data to support that. that we are not rice k here in the european union. i can assure you that's where the company -- for the industry i wouldn't have data to support that. my ideal things is when i look at these two, the products that are already approved will have a fast track approval situation here in the united states an vice versa. we're working on for example lipids. we're three generations behind. and we're bringing and in stead of working on the next generation, we're working backwards. if there would be a way to fast track and use that data in the experience of 20 years to get these products faster approved. that would make a dream come true for me. >> i you know, the defense world is different than the drug world in terms of -- you know, approval b and, you know, we're first in the world to prove that's happening. on the drug side it really is the united states. and we are the models that other people strive for. devices will be different. and i would say that in europe as you well know after some very prominent safety issues there have been some discussion about there, regulatory framework. but i think the goal is not to sort of, you know, look at who's system is better but what is the best system we can prove towards overall. we work closely with our european colleagues. i just came back from a meeting, you know, where i had an opportunity to be with many of them. ou know what's crucial is that we really look at the science that needs to drive decision-making and wendy's need clinical data. and wednesday don't use -- you mentioned about the device consortium which is a private partnership. you don't have to have more expensive and potentially risky clinical trials in humans. you can do some of that testing, using various other kinds of marker. there's a lot, i think that's going on -- but i do think that you also have to look at the reimbursement framework. this is a discuss that we should have more off line. they approve first in europe i'm not being reembered data yes, is colleged and we often do use data from your approval here. but it's, you know, it's a different -- it's a different -- >> the reimbursement is quite different. i'm glad to hear what you're ayingle -- saying. >> working together and using the knowledge to get the projects faster and safer. >> we have a comment. and think progress is being made. i agree with you. >> yeah. i've been asked to say that for those folks watching on c-span, we want to hear from you. lease e-mail your ideas to cures@mail.gov. this has been a tremendous forearm. ome quffle -- some wonderful enlightening. and interesting discussions. forum lgs be discussions like this all over the country. and when we go get washington we will meet together. share the insights that we received at the round table. i'd like to thank all the members lost a couple. i'd like to thank eve of you for taking time to be here and we look forward to working with you in a collaborative effort tomorrow on this. so thank you and this round table is adjourned. [applause] >> poth bama is on his way to europe from meeting with leaders of estonia, latvia and lithuania to discuss security issues. mr. obama will head to the nato summit in wales on thursday and friday before returning home. our coverage of the p's trip includes a trip to the students. you can see that tomorrow morning on c-span2 at 9:00, 10:00 eastern. our coverage of the debate leading up to the midterm election continues tomorrow evening at 7:00 p.m. eastern. the democratic incumbent kay go up against thom tillis. >> we'll talk to a reporter leading the race. >> washington has completely lost touch work american. the federal budget's a joke. i'm tom tillis, came up in the real world. . the senate could use more people for a living. few of the politicians who made this metsdz. lote's make this right. >> i'm kay hagan, one of the things that i love about north carolina is that unless you're talking basketball you don't have to pick a team. that's how i get results from frontier at home. or republican, for democrat. i approve this message because a as so proud with nonpartisan national senator, not too far left. not too far right. just right, north carolina. >> we turn now to jim morrow with the charlotte openeror. the first debate between state house speaker tom tillis the republican, senator kay hagan. that's happening at 7:00 p.m. what does each side need to do? and who has more to gain or lose? >> that's a good question, john. i think that both sides have a will the to gain and to lose. this is really their first head-to-head matchup. tillis has been on the sidelines. in fact, ever since he won the nomination in may. he's the house speaker in north carolina. and he's had to -- and so he said that stay off the trial and that's the last few days. it's really sort of the first chance for a lot of people to see him, to be the primary and for hagan it's a chance to, you know, turn the agenda off of esident o -- obama which the democrats are trying to pin him on. a very tight race between illsi and hagen. that's according to the average of all polls. 7:00 p.m. you can watch it on c-span. we'll be showing i. but mr. morrow, what's the strategy in debate saturday ni this debate?t. is it a matter of trying to avoid a major mistake? trying to do that. the broader strategy for him is to tie hagan to president obama to washington. bama's popularity is at 41% in north carolina. which is slightly below his average. her strategy is going to be to going tillis to what's on in raleigh, which is also unpopular. think a poll has shown the north carolina legislature was controlled by the republicans to unpopular here too. host: asking viewers this morning what races they're 2014.ing for campaign love to hear from viewers in north carolina. the phones are open for viewers from around the country to talk about the races they're watching. president obama, mr. morrill. he visited north carolina last week, an appearance to make some announcements about veterans' sides frame both that trip by the president to north carolina? >> she was quick to disstance herself from the president about that. she's been very supportive of veterans generally. to speak to e down the american legion convention. and all of the trouble that the experienced over the last year has been particularly in the last few months, it was speech for him. distanced herself after the appearance was announced if the minute aviation had done enough. tillis jumped on her for not oing more for veterans and letting the situation devolve to what it has. host: can you talk about the importance and the focus on both sides on women in north carolina nd how both sides are working to turn out the vote among women voters. >> sure. hagan, very important. women skewed democratic anyway. of about 18nder gap points in one poll recently. planned parenthood is planning to hold a little rally tonight, the row night, outside of debate. and then about $3 million in support of her.

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