even though the expectation is phone companies are assembling the metadata if only because you know you get a list of all of your calls at the end of the month. not give-- that did rise to a protected fourth amendment interest. >> even that simple stuff that comes from the pen register is used in courtrooms in the u.s. every day of the week when attorneys get up and say this is a chart of all the times that tospirator a talked conspirator b. you should have heard what they were talking about. here is -- we do not have the content of the phone call but we have a record of the insider at theman sachs talking to

trader. here is a record of the purchase of the stock in question right before the announcement of some new public information. what do you think they were talking about? leakwas proof that was a from the insider. metadata can reveal content. >> the point about the technology that allows analysis leads me back to the point i wanted to make in response moment ago. it is important not to lose sight of all of the other protections that are built acquisition and retention and use of the data the oldis program given production to the government in light of the

technology. that is critical to understand. in addition to being non-content information about the telephone queriedata can only be for counterterrorism purposes and only if there is a reasonable suspicion that the selection turn or number or aceever is associated with this fight foreign terrorist. subsection g requires minimization procedures. it was understood that this tool data be used to obtain

that could relate to a number of u.s. persons. the statute requires that the government proposed and then an element of the program be robust minimization procedures. they spelled out any orders of the fifth but that there would be rejections around the use and dissemination of the data. it would be possible to spell out something in legislation of this program were going to be authorized by congress explicitly that said what made sense and what did not. this as the attorney general has to specify. the record is basically just sign off on what the attorney general asks for. it made more restrict the -- restrictions. that was because of the effort to be more restrictive. they are operating in an expert

take complex. here is what would be better or we think this is what the constitution requires. saysovernment looks and there are things that you should tell us to do. those are approved. this isrn about all of it is fine to say we have this program, whenever ms. use this data and we have rules to -- for its use. that is not the same thing as the government not having that , sitting there. i do not know what mr. stone and could have done. instead of leaking the order he could have linked the database to somebody. when not know what happens some inhabitant of the white house, this one or another one,

has a plumbers unit and decides to let them have access to the database. these are realistic concerns about letting the government have this massive body of data -- i am sureing your answer could he it whoever makes the rules, they could be abused. could get it anyway somehow. there -- they are levels of restriction. it is one thing to have congress adopter program and say this is what it is and we have considered what needs to be done to give protection and one that says "what is relevant but they should be careful how they use it. and then we infer from that this massive program.

enactmentord on the of section 215 and its extensive -- extensive needs -- extensions go past the argument that you articulated. the point in the national security area that clinical ranches are being charged within a range, drawing the lines about what steps are appropriate, something that the supreme court has articulated in the fourth amendment context. draw some ofngress these lines, i am not sure that this was the phrase that was used. some of those are immune to legislative fact-finding about what is -- what trade-offs are appropriate to meet the needs of national security. similarly, in the mcquaid and

cassidy cases, this court and types ofg antiterrorism or counterterrorism activities in connection with the subways and again theted that courts should be reluctant to rest away from the political branches the choices about how these judgments should be made. >> i would be cautious about making a constitutional determination. >> that is why you should evaluate the program that we have. there is obviously a desire to what mightns about arise. given that the supreme court has made clear that [inaudible] you do have smith in the same type of information that was in smith. if you are reaching constitutional issues we are to focus on the program.

i was talking about your preclusion point. that is a regime that congress established and the supreme court has recognized that where that is the case, where congress has not provided the cost of action the idea might be [inaudible] the constitutional claim could the reached. there was not a cause of action available. >> the only way we can achieve constitutional avoidance in this one is by ruling against you something statutory. we are forced to get there anyhow. >> our position is -- prefer you not rule against

this, that is certainly true. with theft constitutional argument. whether you do it at the level of smith versus maryland. and it answers the question about whether it is a fourth amendment search to get the records from the telephone companies and their context. if you go to the special needs in cory my fee look at the program as a whole, not just the initial collection but the act fisc was organized, what would be query and set out in the primary owners reporting that to the fifth. this is not precisely -- this is reflected in the art's opinions

which have been declassified. not just the court accepting whatever the government offered but making determinations according to its own statements that with these procedures the program strikes an appropriate balance with providing the capability -- >> that opposes more restrictions than the government sought at the time? >> i didn't know the answer to that question. my point was that if you look at several of the recent opinions -- >> the procedures there are little different than the procedures that would be in place in a district court or in congress. --terms of having

[inaudible] but to a real debate. >> what i think, what i can say is that although allowed -- these orders are not in the joint appendix. among the declassified materials are opinions reflecting reactions to compliance issues that were identified. -- took theirthe response. it included orders that were not technically proposed but that is a general recollection. >> material has been declassified and should serve to assure us that there is not a special needs problem.

you were pushed to that -- i say this with all due respect. i am not saying that is a bad thing. all of this stuff that we now know and we do not know we do not now, all the stuff we now know is as part of the political reaction to the understanding that this program was in effect. after arguing a good deal from material that was made, that was -- the publicil as a reaction to that. >> of that is in the public realm that had not been public before. this program and this is the critical aspect of the design, was subject to article three review from the beginning by

bodytion of the fisc, a set up to commission that. and others act as a channel for oversight from the congressional side where you are dealing with classified information. >> the whole system would you've won a much more warmer feeling inside. it -- if it was not all ex parte. there was some recreation of the other side. amicus.ono --

i would find what you say, i am now.alking long i find this a lot more reassuring. if it was just subject to an adversary process and it is not. >> among the proposals that are pending would include provisions that allowed for more than one option on the table. the kind of approach that you're talking about. it.n that case i look for >> if you look at the reasonableness inquiry i think and balance the factors the supreme court and this court have said should be balanced. there is an overriding importance in preventing future

terrorist attacks. the intrusion if any subject to the smith argument on the privacy of individuals is -- cabined toand allow findings. use forguards limit the dissemination. there is an oversight system by congress as well as other entities. all of this we submit should leave the court if -- to conclude that the program is reasonable. >> nothing can be constitutionally reasonable if gives us a warm feeling.

>> the test is if it is a means of a composting be interest. unless my colleagues have questions i think we understand your argument. unless there's something you think you have not gotten to that is critical. we will consider that based on the brief. he will not be able to pick it up in her bottles. rebuttals. the -- he is relying on his brief but i do not think we need to year more about any of these things. unless there's something critical they have not gotten to, we have given it probably

more time than you will get in the supreme court. let alone the regional argument. >> if and when you get to the supreme court you will not see two of these. >> thank you, your honor. rebuttal. hear you on we have had a very thorough discussion. i hope you'll be able to be relatively brief and respond only two points that you have not had an opportunity to address so far. ahead.at, go >> the first is their response to the discussion of ratification. the question is always whether there is an official interpretation of statute that congress was aware of and legislated and that is not the case here. many members of congress were

not aware of the program. those who were were not provided legal analysis. they were not able to discuss it with colleagues or constituents in a way that the supreme court has pointed to in past cases of ratification. second point is an exchange that you had relating to efficiency. could usement targeted demands and and nearly instantaneous way if it structured its arrangement with the telecommunications companies in a certain way and congress could provide for that mechanism. the fact that congress has not yet provided for that mechanism is no bar to this court ruling. the supreme court ruled that the government could not wiretap individuals without a war and and it led to the enactment of title iii, the supreme court ruled that for an

intelligence surveillance, it had to be individualized. smith is different from this case from a lot of reasons. it is not just the government is acquiring certain types of information. government is acquiring information about millions of individuals and not just one but also that it is requiring -- acquiring information definitely and not a few years after smith's. when the government scales up a surveillance operation, the constitutional balance is different and needs to be addressed differently. you were exactly right. the court is to assess the expectations of this program and not just of these -- the supreme court decided. a quick related point.

the minimization procedures would be constitutionally superfluous. if smith governed this case, they could collect the records without any of those protections in place and they could store all of them indefinitely and clear it for any reason or no reason at all and they can build the dossiers with no constitutional restriction. the government tries to explain why it is asking for a narrow ruling of this court but the -- this is a roadmap to a world in which the government routinely elects vast quantities of information about americans who have done nothing wrong. it is not the world that the framers envisioned when they crafted the fourth amendment. if there are no questions. >> thank you. very much appreciate the arguments of both sides which careful andly

learned. we will take them under .dvisement and render a decision. thank you. that is the last case on the calendar. we will adjourn. >> court stands adjourned. >> a look at the militarization of police forces. our guest is kara dansky. we will discuss the threat of the islamic militant group isis. your comments are welcome by facebook and twitter. live on c-span every day at 7 a.m. eastern. the committee field hearing.

include the heads of the national institutes of health and the fda as well as neurosurgeon's and drug developers. this is two hours. >> welcome. this meeting will be streamed live. c-span is here so you might want to keep that in mind. i would like to welcome everyone here especially our distinguished panelists and i have a short introductory comment to set the stage create through the 21st century cures initiative, congress is taking a comprehensive look at the full

arc of accelerating cures from the discovery of clues and basic science to streamlining the drug and device development process to unleashing the power of digital medicine and social media at the treatment delivery phase. in this cycle, discovery and development and delivery is what saves lives. we want this cycle to work effectively and efficiently so there is no gap tween the 21st-century science and the washington regulatory process. this work will be a collaborative effort which is why we are first listening to experts and involved parties so that together, we can achieve our common goal, accelerating of the pace of cures and keeping america at the forefront of medicine and discovery. as chairman of the health subcommittee, we have conducted six hearings and three

roundtables. this is the first of the roundtables in the country and there will be a series in the next month or so of roundtables across the country. we are a here to hear from interested stakeholders and that is why am posting today's event. it want to welcome our distinguished members and panelists and presenters and attendees to this 21st-century cures roundtable. the discussion will showcase the innovative work going on in pennsylvania and elsewhere to help us understand the ideas that can accelerate the pace of cures for patients. in molecularances medicine, we will examine how fda is using the existing pathways and if there are new ways to improve the process of developing and improving new drugs and medical devices. advances in our understanding of disease at the genetic level can

help reduce the cost and quicken the pace of clinical trials while also giving patients faster access to the treatments and trials. capturing the impact of new drugs and devices provides new researchers, with caregivers, and patience. information that can serve to find 10 the discovery process so treatments can adapt and help even more people in the future. the most important goal is generating better health than saving lives and achieving these goals will only occur through greater teamwork and collaboration along with nih, fda touch, medical professionals, patients, and advocates. it is unclear if the structures are in place today or can be improved. america must maintain its leadership role.

in this effort. the effort will put the nation in -- and our researchers on a trajectory to make the predominant diseases of today a thing of the past and provide hope for all. wholeitiator of this program is the chairman of our energy and commerce committee. to ask ourleased distinguished chairman, chairman upton for any remarks he would like to make and then i would like to again thank him for taking time to visit pennsylvania.

we unveiled this this last spring and it will be bipartisan every step of the way. this 21st-century cures initiative is in fact going to save lives. it will impact every family not only in this country but perhaps around the world. it will keep our country the leader in medical innovation and accelerate this -- the discovery and development and delivery cycle of cures and treatments for patients. panelists at our roundtable underscored the starkness of the challenge. diseases 7000 known but will have treatments for about 500. that is unacceptable. we can and we must do better and we will. few initiatives have this kind of direct impact on american ours or as we think about family members and close friends that have been impacted i some awful disease. thisolleagues have taken

conversation back home. your subcommittee helped six formal subcommittee hearings but we have had a number of roundtable discussions as well. not only in washington but this is the first stop as we travel around the country. we will be holding additional hearings when congress returns next month. , tireless on this initiative coupled with the bipartisan work on public health and medicare have already helped millions of americans and those here in the 16th district. who isb to many responsible for enacting 2000 public health measures this year. when we look back at the 113th congress it will remember perhaps as a public health

conference and i am pleased to be here not only of you but with the ranking member and vice chairman burgess as we continue to work together. i want to thank those folks who have contributed to the 21st-century cures initiative. we have received invaluable input from patients, government anders such as dr. collins dr. hamburg who are both here today. innovators and researchers create i cannot tell you how much we appreciate the thoughtful contributions especially those of everyday americans where folks can win on .he website we look forward to having a number of these discussions in the weeks ahead and our goal is to move legislation early next down and it her yield back. thank you. click thank you. am very pleased to introduce the ranking member of the health

subcommittee. we 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. i wanted to come and visit, so 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 21st century cures initiative, it is obviously a great initiative. it has bipartisan, and it will 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. >> thank you, frank, and now, i am very pleased to introduce my vice-chairman, dr. michael burgess, from dallas, texas. his medical expertise has been of significant value as we consider public policy questions regarding 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. it is 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. it allows 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 transform our ability to give 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 all over the country, and we are

leaving a good deal of wonderful science on the table due to the current stress on the system, 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. dr. 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. as was 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 cures recognize 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 to those efforts, whatever the 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.

mr. greenwood, with the biotechnology industry organization. >> i served with 12 years with chairman pitts, and for the past 10 years, i have been the president and ceo of a 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 about the three r's, and the 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 making sure that dr. hamburg's organization is adequately funded so she has the tools and the personnel, and that means some hard choices, and it can be expensive but well worth it, and also to make sure we continue to push the envelope in terms of getting new ways to accelerate 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. now, mr. matthews, the head of 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.

we have a 730 five-bed hospital. we have got 37 sites in the county. we care for approximately 420,000 people. we 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. we do have a tavern procedures, which is the aortic valve replacement. the 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. also, the thoracic surgeons, cardiothoracic surgeons, working with the interventional cardiologists at the same time, in order that we were able to decrease the impact on the patient 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 covance, a 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. we offer the broadest range of industrial solutions for our clients to help them bring innovative medicines to market sooner. covance has more than 12,000 employees and delivers these services in several countries. we are in all of your phases of 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.

covance 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. -- hanlon, and now we go to dr. marotti, 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. i have had the good fortune to see health care multiple perspectives throughout my career. ivory 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.. next, mr. neubauer, who is 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. our industry, i can assure you, supports your call to action.

my name is carroll neubauer, and i am the chairman and ceo of b. braun medical, 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. formerly 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. innovation, and i am losing my voice, and i am sorry. however, i do want to recognize that we are addressing these issues. congress and the fda, with the 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.

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 obesity and autism and also for

children with rare diseases and children with special health-care needs who require the expertise and interdisciplinary it care that 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. the nih budget portion is 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. in addition, the graduate medical education program will remain essentail in terms of our ability to train the next generation of pediatricians and pediatric investigators. career development, likewise essentail as we think about the workforce to produce the work that will result in increased child health. thank you. >> thank you, dr.. next, we will go to dr. chi van dang, at the university of adelaide -- pennsylvania abramson cancer center. >> at the abramson cancer center

at the university of pennsylvania, and i am also here on the half of a doctor at a cancer research center, which is catalyzing interests with over 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. first, we believe -- we urge the u.s. congress to have a plan for predictable, sustained, nih funding so we can keep a competitive edge, which is eroding suddenly but tangibly by 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 immune 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 fda, so we really thank the fda for really watching over this. this example makes an economic impact because it allows for up public harvard partnership. that wants to take this technology and get it out to treat other cancer patients that have no other options. i want to appeal to you and the u.s. government to invest in research so we can maintain our innovative edge and make an

impact. for every dollar you invest in research there is two dollars of return with over 20,000 people in the state employed is of research. thank you very much for the opportunity. , dr. norman barton. >> i am delighted to be here. as indicated with shire pharmaceuticals. it is a large company that has a very, very big presence in rare disease. it certainly has other activities in narrow science and various other disorders. 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.

too much capital at risk therefore i will not go there. i, at a personal level, as 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 span of time where rare disease 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 disorders. the work of the 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 more investment in it. for example, the work i do right now is in a program in extremely prematurely born infants. that is a risky space in the kinds of dialogues and conversations that are necessary

to move those types of programs for it really require a broader 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 big 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. next, we will go to the senior 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 touche so many. i am a 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. it aims to do along the lines of what you are trying to explore.

the novel approaches to making new medicines. the company is a major international pharmaceutical company. we have 17,000 employees in the united states, 5000 and the commonwealth of pennsylvania, including one in marietta. we focus on consumer health care products. words that provide an umbrella.

those words were in opening team heard from mr. chairman and i heard partnership and collaboration. that's the umbrella for all the things that we'd like to discuss today. in particular those three items i'd like to emphasize. the importance of the digital era and electronic canth records and how we harness the potential of that type of data. power of that with the the buy logical information that's coming out of the genome revolution. providing as sustainable framework for the national institutes of health draw that work that they are doing internally and ex-termly with their funding in a more seamless fashion to translate into new medicines. and we are pleased to note you may have seen today a partnership, working with the national institute of allergy

theinfectious diseases on ebola vaccine. to verywe would like much underscore the importance of antimicrobial resistance and the importance of that worldwide for this global challenge. you. >> thank you. we have mrs. jennifer westdike, she is representing the pediatric foundation, she's a child with -- havingk so you much for me here today. i'm honored to be a voice for my and foris 8 years old all the other patients who were born with or developed an is incurable. my son had a shunt placed when three days old and all too often as a parent you sit not if thender, it's shunt will fail it's more along

the lines of when it will fail. this is a 50 plus-year-old tech has a high failure rate. my son has been fortunate and only had three remission -- revisions. but some children have this 40 means 40, which surgeries. this certainly has an impact on their lives. can you operate on a child's head without there brain damage,t of and the fluid building up in the scary.it's it's scarry for any parent. so i'm so honored to be part of ideasnd to hear all the and breakthroughs, and hopefully this will have such a positive impact on my son's health and his future as well as all the from anythat suffer kind of illness and i'm just

honored. so much for having me here. >> thank you, it's an honor to have you here. i'd like to thank all the panelists for their remarks. in the remaining time there will free flowing dialogue between the panelists and we for to allow time at the en questions from the audience. most of 'panelists will remain event and answer questions. i'd like to begin with this question. tot steps can be taken accelerate the discovery, development, delivery cycle and innovation, bring new treatments and cures to patients and keep more jobs in the united states? >> thank you, mr. chairman for the question. panel, it'srful wonderful hearing these introductions, and i think we are all very much coming from a similar perspective and very toreciative of the chance have this conversation. we thaw a lot about this in the

21st century cures initiative that you all have been leading. is thate a lot of this congress can do to accelerate this discovery, development, cycle.y and maybe you could break them down into three major areas. to enhance participant protection and increase transparency. in that regard, just as a specific example, as the personalized medicine revolution is coming forward quickly and as gonencing machines have from being the size of phone booths to being the size of the cost hass and come down from hundreds of millions of millions of dollars to a thousand dollars, patients are increasingly interested in having this information, being able to take advantage of it. be sure that we have adequate prohibitions there against unauthorized reidentification of individuals

based upon genomic information, curbly is not properly prevented. therer more we think should be a prohibition against testing.ary but there is a need there as well to provide confidence to kind ofhat this information will be used for their benefit and not 'some way. so that's an example of areas patient protection. a second area two facilitation of scientific collaborations and innovation. in that regard, while it is understandable that the congress has this consider kbl concerns conferences and --enditures on krafl travel, particularly since egregiousen some examples of that. the way that is now being implemented provides a very

heavy burr upon those of us who are trying to organize conferences and make sure that scientists have a chance to attend them. own 30ell you from my years of experience as a physician scientist, many times the most exciting ideas that have arrived in my own career have been on the basis of conversations that happen at these meetings where you rub arelders with people who not at your own institution and newght bulb goes off and a idea gets start and something important for the future gets to occur. the way in which swrefer sight of scientific conferences is being applied at i.h. caused us to spend $ on thellion last year enterprise and involved more than 150 employees to try to track, maintain some sort of record that could be scrutinized to see.dy who wants because it's so

complicated when you have to ofl together a total list attend dees. working a system that's right now. if there's a potential way to that wouldfrom that, be an enormous pat on the back for the whole enterprise, deeply people are troubled about this set of ireaucratic oversight, which think accomplishes nothing yet costs a operate deal of time and money. is to reduce administrative burden and increase the efficiency of the enterprise. the ironically named paperwork reduction act, which as far as i can tell has caused more paperwork than anything in my experience, will prevent a researcher in our program who is attitudes in public towards personalized medicine and wants to carry out a well designed survey that's been reviewed by an institutional review bore and gone through all oversight.iate they can't do that if they want

to ask more than nine people a question because otherwise it has to go through o.m.b. clearance, which i guarantee you by thatmany month and time the trainee has gone onto something else. i'm sure that authorize of the reduction act did not have this in mind, but it's way.y getting in our so those three areas, enhancing participant protection, facilitating scientific collaboration, reducing administrative burden. that we haveas many suggestions. i'm happy to have the chance to provide more examples are as part of this process. you dr. collins. would any members like to ask a question? frank? >> one of the concerns that i ite and i think we can use in terms of hydrosef alus, but

probably applies in general is that often you talk about the shunt, you have to have multiple surgeries. and i do worry, and i know you've ebbs pressed it to my about the whole issue of the family of insurance and coverage. to think of,nd need forhere's more research, particularly when you deal with pediatric research, that's a problem and our focus is on cures. worry, like you talk about your son, that the initial but then is covered, maintenance of the shunt, having repeated operations, the whole issue of access for this of care. did you just want to comment on

bit?a little i know you do talk about it not only for yourself but for the association. course.of i'm sure that nobody here doesn't understand how the insurance industry works, and they don't cover much as far as routine checkups. of times an mri is declined saying my child doesn't checked once a year. but our children are certainly impacting the cost of health repeateduse of their surgeries, their need for ct scans and mri's and shunt series. insurance just doesn't pay for it. so it's coming out of our pockets. costly. very

to --ying >> i know you represent the hydrocephalus association. have they talked about some way of remedying this in general? not only in terms of research in terms of access as well and followup. aboutt just talking insurance, although i know that's an important part of it. have they made any recommendations? our whole point in having these roundtables is to think of things that we can do toislatively or otherwise make a difference. >> well, certainly funding a big deal.at's hydrocephalus is underfunded. i don't think that many people about it. one in 500 children are born with it. probably numbers are much higher because that doesn't include the children that develop it after they're born. so there's some different

things, and the adults that acquire it. is severelys underfunded, which leads to very changeresearch to anything. and that's really our mission, pediatric hydrocephalus foundation is trying to help information about this, and so people know that the far higher than they think. tolet me ask, i'm not trying monopolize, but when i listen to dr. st. gem, you talked about fact that when you talk about a pediatric research in seemsof cures that that to be even more underfunded than anything. it always shocks me, but i know it's true that when we deal with children, whether it's research funding, their care, oftentimes it does seem to fall cracks.the do you want to talk about that

by comparison to research and funding in general? and maybe tell us why that's ise, if it is, or if it true. that's what i hear all the time. i made comments about a introductorymy remarks, namely that the portion i.h. budget devoted to pediatric research is 10% or less. and some might argue that that's appropriate because there's much more disease in the adult population. but as i tried to emphasize, we're learning increasingly that its origins inas pediatrics, during childhood. know thatition we children represent the future of country. so from my own perspective is that it makes sense to at least increase the investment in pediatric research, at least increase the fraction of the budget that's devoted to

pediatric research. an approachghlight that i think underscores the point that dr. collins made team work, partnership, collaboration. inwe think about an area pediatrics that has arguely been model, bothcessful in pediatrics and as an example generally, the children's oncology group, which represents a network of cross thecenter as country with over 200 centers rrkablyating, has been successful in bringing together investigators and bringing together populations of patients from across the country to study those patients and to develop approaches to treatment. and the consequence has been in outcomes.vances in most or at least many pediatric cancers. that model is a model that we should think about for inerally, more generally

pediatrics and more generally in medicine and biomedical research broadly speaking. i again highlighted the national network act,earch which essentially proposes to create consortium that would be like the children's oncology for other disease processes him. to add something? >> yes. i just wanted to build on the raised.t issues just one is the importance of really at how do you incentivize research and product critical areas that are underaddressed and are unlikelyforces to move the dial. and i think that we have not actually done enough analysis the incentives that are out

there and which ones work and which don't. we've got areas that have proven their value including the orphan drug act, which has led a huge expansion of research and development and new products. last year a very large percentage of the new entities were to address rare diseases, and i think that reflects an incentive for a framework that has been working. i think we're beginning to see that arena.t in i also wanted to underscore in ms. westdike was talking about, we're seeing a new focus on bringing the pain into both research and theuct development and regulatory process as well.

i think it's very important that feedback, on the patient experience a disease, what matters, what's lacking in the current products that's then in terms of our regular willer to review how can we really look at some of the measures that matter that might not be the measures that we as thinkian scientists would of but that are really important in terms of the patients experience of the disease and symptoms, and begin to integrate those patient reported into the regulatory assessment of a product as well. there's a lot of interesting and important activities, some of which are under way and need expanded, some of which are still in need of more analysis and development. heard that same emphasis roundtable, for patient feedback. i want to does you, dr. barton, representative of industry,

pediatricl that the priority review voucher program incentive tove encourage -- >> thank you very much for that question. i absolutely believe and we as an organization believe it is a incentive.ant it puts a spotlight on research in children, and it derisks it at a certain level, because an incentive. and that becomes a very important issue as companies look across their portfolios and to makehere investments, to better the nation.n our so that in my mind is a big deal. how it could be made better? it is a provisional or

trial.ry it would be a stronger statement was permanent. you know, we the people, we want and make incentives and opportunities for research children. while i have the mic, there's one other thing a like to on, if i may. i am particularly impressed with that dr. hamburg made about the voice of the patient. colleaguesdo many my within shire and across the industry, in the rare disease the voice of the patient or the patient organization is hugely informative. fact, oftentimes patients and organizations who represent them know more about their

disorder and what really matters any of us. you know, highly schooled physicians, regulators or scientists or who ever we are very very well informed and sophisticated. engages withy f.d.a.,rs, with the bringing that voice to the time, but inery theain situations where pathway has never been down, the has never been laid out before, that voice is just particularly in the selection of end points, et cetera. the efforts that f.d.a. and congress have gone through to enable dialogues between the agency are

commendable. think, i think they are superb. special assessments, all of this much but they don't incorporate that voice. personally as do my colleagues that it would, it would really facilitate moving rapidly.little more >> thank you. >> thank you, i appreciate all the comments and you've done an outstanding job of getting that we can listen to and obviously at the en of the ay we want to end up with good product and that's what this is all about. dr. collins, your comments are right on. want to help with you, help you on those as we proceed. i'm interested in exploring a little bit on the genomic education issue that you talked about. we made great strides in the

last 30, 40 years as we moved that.d on and i had what i would consider good conversation with jip greenwood several months ago as we talked privately about where we should go with the 21st ken try cures. an idea that really the federal government would be the only one that would be able you'd this, and that is to really come up with a times ofe study, all demographics, and then be able by interfacing that with the advances that we saw on genomics to really help industry theseaster cures for all things. or maybe if i might yield to you to not only describe it in better detail essence tot then in get the comments from those of us at the table as it relates to as to howcular side that can be useful or

constructive. >> thank you, mr. chairman. so as an entryway to this description, think about alzheimer's, and everyone knows the tsunami of suffering heading us, particularly as my generation ages out. hundredstry has spent and hundreds of millions of withoutand almost exception very expensive clinical trials have failed. comes atsunami still us. part of the reason we have failed is because by the time patients develop symptoms of alzheimer's disease, they have such neurological damage that it's almost impossible to reverse. so what's clear is that in that disease, as an example, we need need tobio markers, we under at a much earlier date, we the slight and suits changes in the body's chemistry, enable us toat can predict the vulnerability

and thenlzheimer's intervene sooner with a drug. by, in order to do that, and the way this is applicable to basically any disease you can think of. do that kind of thing, what we need, and i've talked to a lot of folks about a very large and very long longitude natural study, on other of magnitude of differentople in groups by age. and a 21st the framinghamf study which was starred in 1948, sequence the genomes of all these people. we would amy every test to in their bodies. we would probably do an mri. might do cognitive and functional tests for these people over time. then what happens is that when people do, in the instance of alzheimer's, dole the symptoms,

then using all of that data you can go back and correlate some these early genetic and bio then learnursors and from that how to intervene. thought with as i'mers in mind, but it applies parkinson's, cancer and cardiovascular. is, as you mentioned, a project of such magnitude that the federal government could do it, and i would argue that the federal government should do it. i have readthat dr. collins' mind on the subject every time we've conversed and says in one way or another, as soon as you take care of the rest of my needs, we'll talk about the next big science project. so as i said to both mr. upton and mr. pitts earlier, we need an advocate of meeting academic needse as well. having said that, i call this

the mother ship, this is a big project of data collection, and interprices -- enterprises, academic, could dock into this mother ship and upload their the datanrich the to base and then download data from this set to enhance their abilities to further their research projects, whatever they may be. not a project that would i think cost project, probably order of magnitude you're talking about $100 million a year, but of course when you think about the trillions of dollars of costs avoided be eventually just with alzheimer's, let alone other diseases, would pay for itself. and the people who participate this project would also learn how to better ten though their own health, there by saving the care system dollars. i would argue that in not the

few, thatnce everybody will be doing this. everyone will have the genome sequenced at birth, everyone will have these early diagnostic tests done. benefit.ty are >> dr. collins, would you like to elaborate? >> i would, and i'd like to throw my arms around my colleague's appropriate. jim and i have talked about this several occasions and i do believe that while this would require an up front investment of time it would more than pay for notice terms of providing a platform for all research thatcal currently we have to set up as one offs and here you would have infrastructure that would allow you to pose all sorts of onto thisg questions --g also it has the potential of in thepatients right center of how this was

organized. the patients would basically be empowered as being part of this to learn a lot more about their own health care. are so many gadgets around today, but i'll wave this ekg that'ss an attached to the back of my iphone. it will it on my heart give a readout as to my electrocardiogram. mobile healthr applications. you can imagine that this kind a those kind ofing applications and a lot of private sector producers of some applications bow -- would be very interested in having them tested in this space. that you could in addition to genomics have monitoring of on a real time basis, which allows peep to themselves.about this could be transformative. is only thing i would say wrong with jim's proposal is that 100,000 is too small and really if you with a to have is in numbers and

when you start to see the reduction in cost that is happening with things like genomics i think we should go million. >> anyone else like to respond? ueekg on my heart, now it's fluttering. >> we're going to let dr. burgers weigh in on that. >> i would like to say that is that's where it's going. as as where we need to go country. embracing something of that when it's time to do it right is something that could set science to a new level. very exciting proposal there. i thought i would take an and a more mondayn way. some of the barriers that we're facing today with innovation, with new breakthroughs for new treatments in medicines are things that fostere type of

innovation in this country, very sets, different providers, different health care settings, they don speak to one data aren't the interoperable at all and we have access to different groups who are taking advantage allhe potential of bringing that together. there are cultural challenges, there are incentive differences. we've got to find a way to bring data thatarge sets of can be mined most productively respectfully. such an endeavor that was just proposed would provide a sohanism where we have to. those problems along the way or else the project wouldn't succeed. and i think that by a great benefit to society. >> i'm enthusiastic about this proposal as well, but it's reminiscent of the national children's study. and i'm interested in hearing dr. collins' comments about the national children's study which has now stalled, and wonder

weut where this fits in as prioritize federal funding for research and funding for awards,al investigator versus studies like this. >> so very appropriate question, the national children's study, which was authorized by the year 2000 hase had a challenging period of design and redesign and re-redesign over more than a decade with the goal basically follow 100,000 children, ideally, right from the the pregnancy so they could assess what events might have happened during a prenatal life that would be important to know about for future health, and then age 21.g these kids to it has had lots of challenges in terms of exactly what would be included, how would the sampling be done. most recently the institute of medicine asked by the congress to do a review of the current plan raised questions about plan would achieve the necessary goals, which is

why we decided it wasn't a good to actually push start on what could be a very long and expensived and 21-year program. so i have a special working advisory committee to the director chaired by phil a retired dean at stanford and peed yeah altmanlar an, and russ to really look tat what the opportunities might be. ofsense is that the design the children's study could take greater advantage now of things that have happened more particularly the advent of technologist like genomics and electronic health records. but i take your point that when about large studies, there are all kinds of ways you can stub your toe. are also ways you can be

quite successful. framingham has been mentioned as good example, and there are others, we need to learn 'those lessons. we are building on greenwood's vision here at an exceptional moment where we have for the first time the ability to design such a cohort that into electronic health records, which are now available who couldndividuals then participate with much less trouble in terms of getting information about their medical their experiences with health care. and we have technologist like to becs which used prohibitively expensive, which are now becoming remarkably cheap. i think we have a lot of public momentum behind this and thatgument to be made ultimately while this is hard, it could save us money in the run. put all those things together and it seems like we would not kind of leadership we should without taking that seriously. by the way, other countries, china for instance, gets this

and are making investments of sort. but i don't think we're going to learn everything we want to know health are waying to see what the chinese results sure thatand are not they will be made available. effort thatve an teaches us about our own population. >> so this is an exciting proposal, and studies like this a large scale could produce a tremendous amount of data to the new bioanalyze markers. but it's also important that we don't lose sight of what we've already accomplished on the data are already existing from the medicines already approved that are contained in the data bases at f.d.a. the u.s. is the only country, agency thathe on requires primary data sets would with nda's, where

countries on require -- so we are sitting on an enormous amount of da that that is not accessible. congress could expand the freedom of information act to include the primary data from studies. in addition to that, there many studies that are done every year of the process, from molecules that ever make to it drug, so inapproved other words failed studies. but we argue my have more to our failed studies than from our studies that have succeeded. access to this data in a way that the scientist community can mine, would add insights on study design, the appropriateness of well they have twail provided to the assessment of benefit reive for new drugs through the review. so i applaud the idea, but we shouldn't forget the enormous

amount of data that already exists that we're just not mining in any meaningful way. >> dr. burgess? >> i was going to be somewhat more pessimistic about the a large federal government run trial. but there. hanlon, you touched tosomething that i wanted ask dr. collins about, because iu showed eric cantor and came up to the n. i.h. several months ago and you showed us doing with you were repurposing molecules from youed drug trials and actually had a couple of things that had been quite intriguing. that a littlek to bit? >> i certainly will, thank you, question --for the dr. burgess for the question. there are hundreds of molecules tested to therapeutic benefit in a particular disease of a decade that

don't turn out to provide the benefit that everybody hopes for much the failure rate in face 2 still over 50% those are compound for which an vep has already been made. knowing what target they hit in to body, knowing what dose give, and so on. and yet when you hit that failed phase 2 and phase 3, oftentimes that's the end the line. compounds, however corks potentially have freight value, if you could identify a disease for which this particular drug happens to hit exactly the right network or that you know has been involved in that disorder. and as we're learning more and past ways molecular and those 7,000 diseases that were mentioned, you can start to matchmaker here, and identify a new use for an old drug. a program which many people thought was going to be pretty difficult because of the p. issues but ultimately was

successful. to invite companies to open their freezers and make available compound that had been 3, were known and to be safe but didn't achieve approval because there wasn't prove of efficacy or there was a change in the much and we are now about a year and two months boo the effort of seeing whether in fact turn out to be of great benefit for other based on crowd sourcing the idea of these new applicationings, and writing universitieses and small business toes say, hey, that compound might work for try it.ease, let's i don't know what the home runs will be and i hope there will be at least one or two. idea here, i'm trying toed by on this profound very much that's been made in these molecules. and see if some new use could be found. there is one hitch here that i float, because of the intention by the 21st attury cures to look obstacles and that is many of

these compound are near the end so if wepatent life. were successful in fining a new use, it might be difficult to to actuallyompany then do the definitive phase three trial for that new use going tohere are they recoup that investment going downstream with the patent already expired. may be some way to deal with that, but it is certainly area which some creativity might be thought about in terms of providing eve than greater incentive for this kind of repurposing to succeed. >> thank you for that. is intriguing. the largee concept of i knowental study, and we've had these discussions with dr. hamburgand before, i mean there are private sector entities that are working same problems, and 23 and me is an example of that. full disclosure. i'm a par is the pan in 23 and

startedn they first e-mailing me and say willing you fill out this survey i thaw what nuisance. but you spend a few minute every day filling out one of their you realize that over time you are making a lot information, the genomic information and some of the become ground. you mentioned about the protection for the data, but i would also offer that people are concerned about privacy, but way kind of got to be a two street. we're willing to give up all kind of provide sit on twitter and facebook. my constituents do it all the time, i hear from them, that's their primary method of communication. so people are willing to give up information two-ways it is a street. so the thing that concerns me about a governmental structured study would be that towould be almost impossible get data back out.

has got toppens, it be the interface has to be extremely user friendly to allow the participants to actually also be recipients of the beormation that could critical to them. >> i totally agree with your this will not succeed unless participants are someparticipants and have benefit from this in terms of being able to get access to the generated upon them. it we've talked about this, might well be that if we went million individuals, that had a positivedy experience with this kind of connection with their genomic information. i don't mean to suggest that this would be some heavy landed information.ederal one of the advantages of this being run by the government insistence bond upon open data abscess, because

that would be the model you'd have to follow. that's the norm for anything doing. i.h. is i grant you, people may be skeptical about the idea that the government is running this enterprise, and it might make more sense to contract it out. but somewhere somehow, somebody get it started. and i think in our long history successes of medical research from framingham down through ofving h.i.v. aids in terms being able to treat people in the developed world successfully, the government's been essential and it would need to be here as well, with partners of course. here today so i can pick on them, but c.m.s., probably one of the largest repositories of data out there, is almost impossible for the average person, the average the average hospital, health plan, network, to getrface with and information back out. may? i just to pond to dr. burgess for

saident, first off, when i that only the federal government could do this, that only the federal government could afford to do this. is not something the private sector could afford. having said that i think it be to greatly advantage if we could set this up in a way that avoided some of bureaucracy induced problems of government in terms of time forth.and so so a private-public partnership order.ably in i also think that there are ways to partner in the costs. the data could be charged to use the data and they eitheray for that data with dollars or they could pay for that data in in kind dataibutions of their own into the system. and in terms of it already being done in 23 and me and so forth, cosmic by copo parson, they're not as , i'm a member of 23 me too, nobody is testing my

blood fluids and so forth. and as i said this is a mothership in which all these projects can dock and trade data, and that's why i to beit's exclusively has funded at least and managed to some regard by the federal government. >> dr. hamburg? >> thank youism was originally on dr. hanlon's comment, but obviously there's been a lot of rich discussion. that i wouldion make to what's already been said more broadly is that whether big huge longitude smallerstudy or a study, what's critical is that we know what questions need to we design the study in such a way that we get meaningful answers that really need to know.e i think that's so important and there's a lot going on now in of involuntary native clinical trial design and trying inask and answer questions ways that require smaller

shorter trial using bio markers or adaptive kinds of approaches. there are huge opportunities data bases once we know more to go back and reexamine and get important new answers. looked that might have at a given condition with a mixed population, even though they tech any chicago have the

same disease, you might wash out your effect. but if you know there's a bio marker that tells you that there set of people more respond or suffer adverse events, then can you take to tease it out and advantage of data that already importantget new information, and i also really that the importance of being able to learn from important. so it's frustrating to me, and i've spoken about this on many occasions, that sometimes we company knowthe why something hasn't worked, but able to figure that information more broadly and it's something that with work withme legislation and also companies to be part of

this process. and i think that dr. cardon's company has been at the forefront of making some of its clinical trial data more publicly available. but there obviously has been reluctance in some sectors to do that. to this is an important time look at what are the barriers to are transparency and what the huge opportunities for more transparency and access to data.ng >> i just want to comment on one of the barriers for these large studies which are not restricted pee yacht --mer or as i'mer or pediatric. if you want to know when a drug goes awry, can you go to to author way and get this done because there's health record there.

even the same system we have des par at health records. to inscent's a way different private sectors to oh niceether to harm public health records, that's the only way we can achieve these goals. mr. chairman. it's hard for me to spend a day without getting back to the issue of resources and my concerns over cuts at the level. and of course comparison with other countries. dr. dang, you mentioned of course i always bring upkeeping up with china. what i wanted to ask, and i can start with you, but dr. collins and others mentioned it, how bad is it getting? we talk words, when about cuts at the federal level, and maybe cuts at the state level or maybe cuts at the private level, i'm not just

talking about the federal but overall,re, how bad is it in terms of our china versusth other countries. do we continue to deteriorate, looking at not just the federal government but also collectively expenditures on research and cutting end, if you will. the question. i can tell you that this, our end is getting a little dull. a previous trainee of mine, who went back oh china as a professor call called me and said, you know, work with me because there's more money for here.ch and this is just within the last month. chineseense of the investigators being very well funded is really there and they really are getting peel to go back to really build up the research empire. been built on a

number of imgrant, actually i came from vietnam. and that's how we can keep the competitive end. we have young scientists who are getting discouraged because of the type of funding cuts that are beg from posed, 'many of the talent will go somewhere else or do something else. many ideas that are buried in young people will be gone. for example, i gave you that idea that the innovation in of the immune cells, again the person was once a young investigator, if he didn't stick with it, that idea would have again -- short-termo much the fixes we have right now will have long-term consequences.

so we need to continue to invest research enterprise. their funding is going to hurt medicine in the as cut funding to private enterprises. they lost funds that would go to a large part into medical research. getting a product approved in the united states costs much in any othern than country. those were also funds that are for producte research and development. we are working her in the united sometimes on first generation products that the europeans are already using in their second and third generation. i've been on so many panels and startuphers tell companies not to start the

united states with your new product and your product go to europe first, do your clinical trials there. there.ney get reimbursement there. cash flowou have the that you do not depend on investment funding, then go to the united states. a trend that really concerns me. it is taking us off the medical device innovation. we have to work on these basic things. love these dreams this hopefully will return into reality, i sure do. some real basic things to work on that i hope will find the initiative's attention. >> and that taxis on total profits.not just >> it's on sales. tax.a real excise even if you don't make money, mr. children,ax, it's truely off revenue. to emphasize the

point that i think we are at generation of a investigators in biomedical research. thating on the point dr. dang made about young faculty, we are seeing an loss of junior faculty as they are unavailable to make the transition from a mentor to an independent investigator award because of the funding challenges at the n. i.h. level, and younger individuals see that, so we have individualseam of into biomedical research. so the bottom line is we have a decreasing number young investigators, and as we think about the future of biomedical research that is a critical issue. the n. i.h. has made significant efforts to accommodate junior investigators, by making special allowances in terms of scores on first independent investigator award. but the bottom line is we do not beenenough money, and it's allocated to the n. i.h.

if we want to support ongoing we want to have a future of biomedical research in this country, we me to think about junior investigators and how to important that group. >> thank you. got three. let's go down the line. dr. collins. >> so, just some numbers here to the conversation, which is clearly the one the wakes me night.he middle of the if you look at n. i.h.s spending powerial, our purchasing for research, which is basically the appropriation plus the biomedicalof research inflationary index, comparing 2003 to right now, n. has lost 23% of its purchasing power, almost a quarter him the consequence of again, just numbers, that reflect why this is such a tough scientist oryoung a mid career scientist, relates

if you your chances are send your best ideas to n. i.h. our grant proposal, and ask best peer review system in the world to review and it decide what priority to assign to it and then are you going to get not.d or carl, who sent us a proposal in the past, would have been considered risky, might have gotten a priority score that reflected such am big you'dity work, but he this would have gotten funded. of this loss of purchasing power, your chance of getting funded has shrunk down six.e out of so it's about 16% is the success rate. all ofs for virtually the last 50 years until 2003, aroundccess rate ran 30%. so i've looked at grants that 20th percentile or

the 10th percentile or the 25th percentile, i can't tell the conference between them and neither can anybody else. that experiment. so that says we're leaving half the good sigh thens the united table.on the and the people who have proposed it go away discouraged and some basically give up. 18% of young scientists the a considering were leaving the united states to go to another country. and other countries, as you've heard, are increasing their support in some instances bymatically, china going up 20% per year compound that and predictions are in another four years china will spend more on biomedical research tonight united states, not as a percent of g.d.p. but in absolute dollars. of thatconsequences will be very clear. >> this is a great discussion, are listening well. i just want to report to you this august break that we're out, i said a number

of members from our committee on a bipartisan basis over the china. if you, i don't theo barton, all right, backk forward to hearing from joe and some of our staff him to find out precisely what are the chinese doing that we're not and how we can make up for this gap. and be part of our equation and one of the reasons priority to a a send a >> 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. collins' organization distributes into academia. it then goes -- and that's 30-some billion dollars. and i think 23% shortfall needs to be made up and i don't think

there's a number i wouldn't support in terms of funding the nih. then comes the $50-some billion to take that basic knowledge and convert it into real medicine that can save a real human life. where does that money come from? essentially from two sources. sales of product, existing roducts, and then investment and people who buy stocks on the open market. those investors don't make those because they want to cure disease. they do it for return on investment. this is among the most risky enterprises there is to most projects fail over and over of money expenditures is enormous and most of the dr. hamburg's