our guests can take their seat. recognize myself five minutes for an opening statement. this morning certainly i want to welcome our witnesses. we are here to conduct oversight

and receive updates on the implementation on one of the most substantial legislative accomplishments in the space of biomedical innovation, the 21st century cures act. cures passed both the house and the senate with wide bipartisan support and was signed into law december of 2016. as with all landmark laws, i think it is critical that the congress, especially the relevant authorizing committee, engage in oversight to ensure that the agencies are implementing the law according with legislative intent. based upon the active leadership and robust activities of both the national institute of health and the food and drug administration, i look forward to hearing from dr. francis clins, director of national institutes of health and dr. scott gottlieb the commissioner of the food and drug administration regarding progress in implementing cures. i thank both -- i thank our witnesses for their willingness

to testify on such an important topic. the 21st century cures act provides hope to those who need it the most, individuals and families suffering from life-altering, often life-threatening illnesses, whether it is cancer or a rare disease or alzheimer's, there are conditions that are costly to americans of all ages and their families. s sadly, we each know too well the financial and human toll that diseases place on our friends and our communities. one of the most impactful positions of the 21st century cures created the nih innovation account in treasury. this account funds projects like those related to precision medicine initiative, the brain research through advancing innovation and neuro technologies initiative, cancer research and regenerative medicine. the base and breadth of biomedical research continues to accelerate and as we now have treatments to cure diseases such as hepatitis c which was once

unimaginable, yet there is still much we do not know especially regarding the neuro genre testify diseases. 21st century cures included a provision for a certificate system. prior to cures there was no requirement or authorization to provide certificate of neurologic disease but this changed thanks to cures, specifically this section of law requires the secretary of the department of health and human services to create such a system by expanding surveillance infrastructure and activities including data collection to determine brief valens, risk factors and diagnostic markers. preliminary results from a multiple sclerosis society study show there are nearly 1 million americans living with ms, more than twice the previously reported number. the surveillance system included in cures will provide us better information so that we can further our understanding of and

eventually cure these diseases. i am especially grateful to see progress in this important policy. this began as stand-alone bill introduced in the previous congress. additionally cures advanced precision medicine which allows physicians to offer their patients truly personalized treatment, achieving the full potential the precision medicine will require effort to collect health data in addition to the research done by our nation's best research investigators. the law cod nice the precision medicine initiative and encourages of secretary of health an human services to carry out the goals of the initiative while ensuring confidentiality of the patient's information. the all of us research program is a major piece of the precision medicine initiative and has already engaged over 1 million volunteers in the united states. i think you are to be congratulated for that. clinical trials play a crucial and necessary role in the drug approval process, while the food

and drug administration's traditional critical trial methods have proven successful they are not always timely or applicable to new types of drugs. the innovation and promising results of efforts included in cures will provide americans suffering from cancer and other diseases with the opportunity to undergo successful treatments and in some cases be cured. so our thanks to dr. collins, dr. gottlieb forgiven us the updates on the implementation of this new law. i yield the balance of my time to the gentle lady from tennessee, ms. blackburn, for a statement. >> thank you, mr. chairman. dr. collins and dr. gottlieb, we are delighted that you are here. two things that i'm going to want to discuss with you all. dr. collins, the all of us research project and making

certain that we anonymize and we protect the data that is in that program. and that the privacy of the patients with data is respected as we move forward with this. and, dr. gottlieb, following up on the software act and making certain that the implementation is going well, and we welcome you both and we look forward to the hearing. yield back. >> thanks to the gentle lady. the chair now recognizes the gentleman from texas, the ranking member of the subcommittee mr. green five minutes for an opening statement. >> mr. chairman, thank you for holding today's hearing on the implementation of the 21 century cures act. i want to thank all our witnesses for being here today, especially dr. collins and dr. gottlieb. you are pretty regulars at that table. this december will mark the two-year anniversary of the 21st century cures act being signed into law by president obama and his last public signing ceremony. the promise of the 21 current ri

cures is to advance the discovery and development of new treatments, cures through increased research and improve drug approval process. this important law dedicated $6.3 billion in new investments to support medical research initiatives like the bo biden cancer moon shot, the brain initiative, the precision medicine initiative within the national institute of health, it also provides money to food and drug administration to advance the agency's mission and implement the policies in the underlying bill. i hope our committee and congress that these investments are being put toward finding a cure and ensuring the infrastructures is in place so that new their piece are accessible to all americans. the in. ih has provided $4.8 billion in new funding and advanced cutting edge research initiatives, the fda was provided $500 million over ten years to improve the agency's medical product review process and expedite patient access to drugs and devices

without compromising standards of safety and effectiveness. in addition to this much needed funding there are many positions in this package worthy of support from facilitating the development of new antibiotics to fight against super bugs through advancing the use of modern clinical trial designs to investing in the next generation of medical researchers. while some of the provisions are technical in nature, the real world impact could not -- could not have to be not abstract. patients and families deserve to have their elected officials respond to their needs. the 21st century cures was w written to do just that. from patient focused drug development, medical device innovation and improving scientific expertise and hiring.

dia the 21st century cures act represents what we can accomplish when we work across the aisle. i will look forward to hearing from our witnesses. mr. chairman, i yield the remainder of into i time to my colleague, congresswoman diana degette. >> thank you, very much, mr. ranking member for yielding. dr. collins and dr. gottlieb and all of their staffs who helped us develop this bill and most especially fred upton who really worked with us every day. but this really was a work product of this entire committee and it shows the greatness of the energy and commerce committee and what we can do when we decide to work together to tackle a serious problem. mr. chairman, i also want to thank you for having this series of hearings. it's about a year and a half since the bill has been signed and it's almost exactly two years since we originally passed

it through this committee. we need to make sure that everything we intended to do in cures is happening and consider the agencies need modifications or changes or additional resources, that we give that all due consideration. the only way we can do that is to have hearings like this. i do want to say i've been concerned lately reading some -- some media accounts that say that some of the cures money may be reprogrammed for other purposes, including for -- for the orr issues of the kids at the border. you know, we should be able to find the money to do that without taking money away from important biomedical research and drug and device approval at the fda. so i hope that's not the case and if it is i hope this committee acts swiftly and in a bipartisan way to make sure that the intended moneys that we

authorized in this committee remain, because we still have so many bridges to cross and we're going to need every penny that we authorized. with that, thank you very much, mr. green, for yielding and i yield back. >> pending the arrival of the chairman of the full committee, the chair recognizes the ranking member of the full committee, mr. pallone of new jersey. five minutes for an opening statement, please. >> thank you, mr. chairman. i want to thank dr. collins, dr. gottlieb for all joining us to discuss the ongoing work at nih and fda to implement the 21st century cures act and i also want to thank our colleagues diana degette and fred upton. the cures act tasks both of your agencies with implementing critical provisions and improving the discovery and development of new treatments and cures. at nih the law provided significant funding for the bo biden cancer moon shot

initiative, the all of us research program and the brain initiative and at fda the law included provisions to improve the medical product review process as well as new authorities and funding to ensure the agency has the resources to recruit the best talent. at our hearing on this topic last november you both provided promising updates and i look forward to your continued work. as i said before, it's important to hold oversight hearings like these that allow us to learn directly from the administration how policies are being implemented. while i'm pleased that the subcommittee has decided to conduct continued oversight of the cures act, there are several topics within the subcommittee's jurisdiction that also deserve hearings and oversight. i've asked the majority to schedule hearings on a number of issues that are priorities for democratic members of the committee, i've asked for hearings on maternal mortality, health disparities, gun violence, the indian health service, cosmetics reform, the office of refugee resettlement,

drug pricing, abuse of the rems program and marketplace stabilization. these are all different issues within the subcommittee's jurisdiction and we may have different opinions on many of them, but that's exactly why we should at least have a hearing. these are critical issues that this committee should be discussing in an effort to find potential solutions. so i hope in the coming weeks after the august recess that the chairman will respond and select a few of these issues to hold hearings on in the short time we have left for this congress. unless someone else wants my time, i will yield back, mr. chairman. >> thanks to the gentleman. the chair will hold the time for the opening statement of the chairman of the full committee pending his arrival. let me thank our witnesses for being here today and taking time to testify before the subcommittee. we're going to give our witnesses an opportunity to give an opening statement, that will be followed by questions from members. today we are going to hear from

the honorable francis collins, the director of the national institute of health and the honorable scott gottlieb, commissioner of the food and drug administration and, dr. collins, it's my understanding you have brought a supporting cast of dr. norman sharpless, the 15th director of the national cancer institute and dr. stephanie davaney who is the deputy director of the all of us research program. i don't know if it's an intention for all to give an opening statement, but we will start with you, then, dr. collins and you are recognized for five statements for an opening statement. >> thank you and good morning, chairman burgess, ranking member green and other distinguished committee members, it's a great honor to appear before you again today along with my colleague fda commissioner dr. scott gottlieb, to give you a progress report on our implementation of the 21st kooecentury cures act. also joining me ned sharpless and dr. stephanie davaney.

i can't emphasize enough for this subcommittee how much we appreciate your bipartisan leadership in passing this act. 51-0 as i recall out of energy and commerce. which aims to speed the translation of scientific discoveries into lifesaving treatments and cures. in the written statement i've submitted i've outlined a comprehensive report on how nih is working swiftly to implement the act's provisions one by one that especially includes multi-year support for four specific areas of scientific opportunity, supported by the acts innovation fund. today i'd like to highlight two of these, the cancer moon shot and the bold new precision medicine initiative called all of us. the cancer moon shot is aggressively pursuing a very ambitious goal, to accelerate advances in cancer prevention, diagnosis, treatment and care, such advances include immunotherapy in which a

person's own immune system is taught to recognize and attack cancer cells. after years of research supported by nih immunotherapy is leading to dramatic cures of some cancers like leukemia, lymphoma and melanoma, but some other cancers, particularly solid tumors like colin, pancreas, breast and prostate have proven much like responsive. i'm thrilled to tell you that some of those barriers seemed ready to come down. just last month a team led by nih's dr. steven rosenberg announced a novel modification of an immunotherapy approach that led to a complete regression of widely metastatic breast cancer in a woman with universally fatal form of the disease. as always i have to counsel patients this success story involves very few cases right now and must be replicated in further studies, but without doubt this woman's lifesaving

experience represents hope for millions more. as exciting as potential cures like this can be, in authorizing and funding the cancer moon shot, you wisely tasked nih with advancing not just cancer their piece with you also cancer care. let me tell you about an nih-funded trial that illustrates the progress we are making in had this area. each year as many as 135,000 american women who have undergone surgery for the most common form of early stage breast cancer face a very difficult decision, whether or not to also undergo chemotherapy to improve their odds. now, banks do a large nih-funded clinical trial called trailer-x. we finally have some answers and they are good answers. if turns out that about 70% of such women actually do not benefit from chemotherapy and a genome mick test can ooips

identify them reliably. it's ideal to spare women from the potential toxic side effects of chemotherapy if that's possible and still have a good outcome. on top of that, and this will probably warm your heart because it certainly does mine, this move basically making it not necessary to go through chemotherapy for many of those women will produce a significant cost savings for our healthcare system, maybe up to a billion dollars a year. figuring out what health approaches work best for each individual and why brings me to the goal of another important investment of the cures innovation fund and that is the precision medicine initiative. the centerpiece of this initiative, the all of us research program, will enroll 1 million or more people and we are off to a very strong start. on may 6th, just two months ago, the day we launched national enrollment in seven sites across the nation we reached more than 10,000 people just that day at community events and almost four times that number online.

as of this week over 86,000 volunteers have signed up to contribute their health data in many ways over many years. some are enrolled through health provider organizations, ten of them that are part of our enterprise, and that includes human health centers and the department of veterans affairs. others enroll as direct volunteers who sign up over the internet. all together i'm happy to tell you that almost half are from historically underrepresented racial and ethic groups, which is one of our goals to we can utilize this to look at health disparities. right now anyone who is 18 and older including members of congress, note the url if you are interested in learning more about how to sign up. you can join. next year we will begin enrolling children, with he decided to first start with adults, but next year children will be added in. and in 2019 we plan to open a secure portal to give researchers access to all of this data in a deidentified format with exceptional

security. with every new person enrolled, every biological sample preserved, every electronic health record collected, every survey filled out, this data will hold more and more promise for advancing human health. and with every new scientist mining this data in search of answers to the important biomedical questions the more that promise will be realized. this is ground breaking. this exciting progress along with many other advances in biomedical research is being made possible because of the vision of you and your colleagues. so thank you for your investment in the 21st century cures act as well as your ongoing support of nih. we could not do this without you. my colleagues and i really look forward to your questions. thank you. >> thank you, dr. collins. dr. gottlieb, you're recognized for five minutes for an opening statement, please. >> thank you, mr. chairman. mr. ranking member and members of the subcommittee. almost two years ago the members of this committee held the passage of cures as a potential game changer for patients. i agree. i provided a comprehensive list

of our cures activities in my written statement, but i'd like to focus my remarks on one cross-cutting priorities and that's modernizing clinical trials. fda has embraced en spray testify trial designs in the patient center trials envisioned by the cures act. our aim is simple, innovative advanced evidence generation to ensure the timely availability of safe and effective their piece. the cures act is catalyzing these approaches and catalyzing the development of new precision medical technologies, they are enabling us to target, arrest and cure intractable conditions. these advances aren't cheap. access and cost is a big issue. i know some question whether our market-based system for medical innovation is financially sustainable. they did if we can afford this coming wave of precision guided their piece. i'd say we couldn't sustain our system without them. new advances like regenerative medicine and gene therapy can displays costs by restoring function and reducing reliance on costly medical care doctor

ifrd in hospitals and nursing homes. the best decision it to make it easier to bring new invasions and competition to the market without compromising one bit the gold standard for product regulation. that brings me back to modernizing clinical trials. rising trial costs and complexicomple complexicomple complexicomple complexity can be a barrier to getting timely competition to new a approved branded innovative drugs. one reason we may be seeing higher costs is because it's taking longer to for competitive to emerge where specialty drugs are targeting unmet medical needs. i want to share a snapshot of what we found. we plan to publish the full results really soon. for non-orphan drugs, 41% of the first in class drugs approved between the years of 1991 and 2000 had at least one competitor in the same class within five

years. this rate dropped sharply over the next decade. for the same kind of drugs approved between 2001 and 2010 only 18% had a within class competitor after five years. another way of interpreting the data is to describe the lag in competiti competition. for the drugs approved in 1991 to 2000 nearly a quarter had a competitor within two years. for the cohort of drugs where the first in class medicine was approved between 2001 and 2010 it took an additional five years for there to be nearly as much competition. by year seven competition still lagged with only 22% of the new cohorted drugs having any competitor. we see similar patterns in most rare disease treatments as well. these trends mean that cost clear branded drugs may enjoy longer periods without facing competition from products in the same class and this may increase their pricing power. we need to understand why. part of this has to do with the difficulty of running clinical trials with a second in market drug especially if there's

therapy for an unmet need. it's becoming harder to be second and that's a problem. efficient modern approaches to designing and conducting trials can address some of these challenges and help us get more information about safety and effectiveness at the same time. to advance these and complementary goals the fda is pioneering a number of critical advances in critical trial design. first our master clinical trial protocols, these include basket trials, umbrella trials and platform trials. these approaches can sharply increase trial efficiency and lower costs. they move away from one drug, one disease trials and allow the testing of multiple drugs against one or more diseases or disease subtypes using a common clinical trial infrastructure. another approach is seamless trial designs that come press three phasing into one continuous trial. as you enroll new patients you expand subsequent co-authorities of enrolled patients using the information you learn about the features that predict benefit from a new treatment. we will be publishing a guidance

that lays out how product developers can conduct thighs seamless trials and how to expand co-authorities and the clinical criteria that can be used as these trials advance. a lot of time and cost of clinical development is spent between the starting and stopping of the three phasing of trials. every american has already or will one day face a serious medical diagnosis either personally or through a loved one. we need to reduce the burden and cost of advancing care. the clinical trial reforms we're making today will ensure patients who find themselves in these hard circumstances have a better chance of finding a cure and market competition helps make the treatment accessible to everyone. thanks a lot. >> thank you, dr. gottlieb. again, thanks to all of our witnesses for agreeing to be here. we will now proceed to the member question and answer part of the hearing and will still yield to chairman walden when he

comes in for an opening statement. let me recognize myself five minutes for questions and, dr. collins, again, thinkings for being here, thanks for bringing your backup. so let me ask you a question about on the 21st century cures, we tried to identify ways to get regulation and policies that were inconsistent and give you some flexibility to move past some of these that are overlapping and unnecessarily duplicative to relieve some of the administrative burden. i think the act asked you to review that, so can you perhaps share with us where you are in the review and how nih has identified some opportunities to relieve the burden on investigators? >> yes, i'm happy to do so, and, again, thanks to the committee for making all of those changes part of this bill. some of them may seem kind of bureaucratic and administrative, but they make a huge difference to us in terms of the ability to carry out our mission. we've been asked to look at the

faye in which we've asked our grantees to deal with financial conflicts of interest which we think are very important for us to track but where the mechanisms for doing that tracking were seen as unduly onerous and we are in the process of going that with our colleagues at hhs and i think there will be some changes that will make that more of an efficient process. another thing, again, this sounds pretty down in the weeds but it mattered a lot to us was the degree to which we need to do monitoring of what you would call a subrecipient where you give a grant to a particular institution and then they have a subpart of that to another individual and in the past our need to reach through and do we detailed monitoring even of subrecipients was something we were required to do without a whole lot of reason for that. so we've already moved to simplify that process. financial expenditure reporting, a lot of the reports that have been done in the past produce data that not very many people look at and yet at the same time we need to be sure that we are being good stewards and that is also being simplified.

we are looking right now at animal care and use and the oversight that is necessary of course to be sure we are dealing with animals in an ethical way, but some of those particular oversight mechanisms are being reviewed and we put out an rfi and gotten 19,000 comments back from people who have opinions about how we might steam line this process. those are a few of the examples. again, it was really helpful having those features into 21st century cures to give us the authority to do those things. >> obviously ongoing it is going to be important for us to communicate on this, if there are ways where we can provide additional legislative help on that, i think you could find the committee willing to have those discussions. >> thank you. >> dr. gottlieb, thank you for your comments on the clinical trial reform. i always felt while we were doing the round tables for cures that that is where the -- likely where the big money was. if we could reduce the time in trial, reduce the -- if a product was going to fail, allow

it to be identified and fail early to we didn't send a lot of time chasing something that was not going to pan out, so i really like the concept that you just elucidated about the seamless trial concept and condensing the time between face one, face two and face three clinical trials. i think that is likely to have a significant impact. one of the things that has always concerned me is that we've had some legislation, there's been a little controversy, but the ability for all -- we don't have a payer here, we don't have cms, but for the researcherser, the regulator and the payer to communicate before something comes -- i always got the impression with hepatitis c when savaldi came down the pike the medicaid payers were not ready for what was happening, but they had been prohibited from having those discussions because of current

laws. so i am hopeful that we are able to do something particularly in gene therapy or gene editing, where you have a one shot that's going to cure something that's a lifelong problem, i mean, that's huge, but it's likely to be priced out of the range of most average people's budgets. can you comment on that, on our ability to communicate prior to approval? >> thank you, mr. chairman. i think we -- i would say -- fully addressed this issue and your points are well made, and i share your concerns and have shared your concerns in the past. i think we've fully addressed this issue with the guidance that we issued earlier this year, that was in part built off of a cures provision related to payer communications with product developers for purposes of engaging in value-based contracting and other economic kinds of discussions. where we've essentially established a safe harbor for those kinds of communications

where the fda isn't opining on whether or not it has the legal authority to provide any regulation to that context and some would argue we don't, first amendment grounds, but even if we did we would not choose to exercise that authority because as a matter of public health we believe there should be robust discussions for the purposes that you suggest, so they can engage in value-based contracting. other ways to pay for these novel their piece. >> thank you for that answer. recognize mr. green. five minutes for question. >> thank you, mr. chairman. and i welcome our panel here. let me give an example of what's happened in the last four years. in 2014 we had an outbreak of ebola in the west africa. there was no treatment for it. today what we're seeing in the democratic republic of condo we have a vaccine. what has happened in those four years now cures came on in '16,

but can dr. collins or dr. gottlieb share that, how did that become -- i know merck has the vaccine now, but how did we get there? because like in '14 there was no vaccine. >> well, i will let francis talk about that because it came from him and i will comment where it is now. >> it has to be approved. >> exactly. >> nih actually began working on an ebola vaccine back in the 1990s when nobody had really paid that much attention to this but we were concerned that this might at some point become important. there was already a fundamental amount of basic science and work towards a vaccine happening for a good 15 to 18 years before the crisis had struck in west africa in 2014. that being the case, this was, in fact, a program that developed a vaccine in record time, but still not, in fact, for west africa in time to have much of an impact on the actual

outbreak because by the time the vaccine was being distributed good public health measures were already resulting in the epidemic, waning rather quickly, which was a good thing. with the drc, this vaccine, which is manufactured by merck, but based upon a close collaboration within nih, in fact, the phase one trials for this very vaccine were done in the nih clinical center in bethesda showing that it was in fact safe and seemed to generate good anti-body titers. that was distributed in drc in a ring vaccination strategy and also giving it to healthcare workers. we were happy to see yesterday the declaration that this epidemic is now over. did the vaccine contribute to that? it's a little hard to tell because fortunately this was a limited outbreak, drc moved quickly, but, again, we are very well situated now to deal with an ebola outbreak in the future. in this instance it was great to see how quickly the vaccine was available, got distributed and

was made available to those who needed it. >> i will just comment just to build off of dr. collins' comments, i make two points up front, first i think it's hard to understate what a game changer this is. this is the first time that we now have a technology available in the setting of ebola to intervene to stop the spread with something other than just traditional public health tools of isolation. i also think we should make note of the efforts of the manufacturer in this case, merck, the doses that were deployed in the drc were donated. they shipped about 13,000 doses to w.h.o., w.h.o. handled the transportation to the drc. so this was very much an altruistic effort that helped in that setting. i will say we are working efficient will i to try to move towards a licensed product here in the u.s. obviously i need to be cautious what i say, but i feel optimi optimistic that this will be something that we can accomplish

in the near term and we can have a fully licensed vaccine. >> that's just an example because ebola did -- we had a gentleman from west africa come to dallas, texas, and the protocols weren't followed and so it impacted our own country, but using this as a paradigm on what we can do for other terrible illnesses that are developing and i tell people on any given day we have -- you know, we have tuberculosis in the city of houston because it's an international city and you just don't check everybody. so we have to be on our toes to be able to do that in the 21st century cures act i think gives you some of those focus, but looking at the ebola vaccine, in four years we went from people worried it's going to get -- and i was trying to explain to folks more of my constituents will die of flu because they didn't get a flu shot than will ever be exposed to ebola and we want to keep that way, though. i'm concerned about the growing

threat of the antibiotics resistance, that's why i sponsored a provision for the antibiotics that meet unmet medical needs. the provisions directed fda and cdc to coordinate efforts with respect to monitoring antibiotic resistance and any other drugs approved under the limited population pathway for antibiotic and anti-fungal drugs. dr. gottlieb, what steps is the fda taking to coordinate the cdc to -- with the cdc to support policy that promote judicious antibiotic use and antibiotic stewardship? >> we've taken a number of steps. we are going to be taking some additional steps to look at antibiotic use in animal feed. the length of the duration of use and indications in which they are used and plan to have some additional policy steps that we should be announcing within the next couple of months to continue to advance what we've already done in that regard to reduce the use of antibiotics in animal feed and, you know, limit one route by

which we're seeing the resistance develop. as well with the provisions in the qdap also the lpad that was enabled in legislation passed by this committee, we've been able to create new pathways to try to provide additional incentives and additional efficient pathways to get new drugs to the market that attack some of these multi-drug resistant pathogens. i would close by saying i think it's still important that we focus on trying to develop new poll incentives and new ways to potentially reimburse limited use anti-inn if he can tiffs as a way to create incentives for the development of these products. we are working on one such idea in conjunction with our drugs at cis to develop a paradigm for drugs that are the kinds that you want to just put on a shelf and never have to use moving towards a site licensing model as opposed to a pay peruse model

which has been a traditional way we have approved for drugs. >> i know i'm way over time and yield back the time i don't have. >> chair recognizes the gentleman from kentucky mr. guthrie. five minutes for question. >> i have a couple questions but first i want to say i know there is a lot going on in this great city and this great country moving forward and a lot of times what's happening in this hearing, a lot of hearings make television, this one probably doesn't make a lot of prime time television, but it's important. it's extremely important what happened when a bill like the 21st century cures based by a voice vote out of this committee, overwhelmingly bipartisan and really makes a difference in people's lives. dr. collins, you and i had a meeting the other day with a member of the band u2, the edge, he was talking about somebody who was close with him who had had childhood cancer. i had a friend of mine that passed away when i was 11 or 12 and she would be alive today

given if she had the same disease this time -- or probably would be alive today given -- and the reason i point it out u2 is because i think they really being irish really love this country and they really put out that american inspires the world and does things throughout the world. people throughout the world do research but nobody compares to what we're doing as a country, what our healthcare system, our industry, and it's just a shame that there is a lot of good news and a lot of it's happening in what people in this panel is doing and we really appreciate it very much. i do have a couple questions for dr. gottlieb. one is specific on the cures act it codified many practices in the office of combination products, but also included provisions to clarify regulatory requirements, improve processes. can you explain how the fda is delivering on these efforts and the complex area of regulation? >> we've put forward a number of

additional guidances as well as staff manual guidances on how we approach combination products in the agency. i mean, historically it's been challenging for the agency, i think we've made a lot of progress in recent years. i think we've made a lot of progress into some of the provisions that flow out of the cures act as well. we set up a combinations council to try to adjudicate who has primary jurisdiction over these products and we will be putting out a guidance sometime probably this fall, end of the summer, early fall, that's going to make some further process reforms that i think is going to make it more efficient for products that sit on that cusp to move into the device realm, which i think is the preferred route for a lot of product developers if they can get there. so we are going to look for ways to find the most efficient route while being mindful of our public health obligations in these circumstances. >> thank you. and also you and i talked recently on the telephone and several of us here sent a letter

concerning drug shortages and drug supply short annals. i know i have had an emergency room physician from my district say that sometimes they don't have just the simple things they need, they have to be more creative, they have to figure out other things to move forward. i know some of it was tied to, you know, the natural disaster in puerto rico, i think you guys really went in and handled -- that's what people need to know. there are people in government that are trying to make things work. we have issues we need to address, but what happened this puerto rico to get pharmaceuticals moving again, i mean, what you guys have done -- but i know there's still issues with drug short ans, i heard from ems, emergency room physicians. just basic i think so this. could you talk about a minute and a half what you explained to me on the phone and what you're doing with your drug task force and what's pressing and what's moving foort sfoord. >> the challenges are structural in my view. we're dealing specifically with sterile drugs, sterile injectable drugs, those are the ones that seem to be chronically in shortage.

i think there are structural problems. reimbursement has been driven down so low many of the drugs and manufacturers are slightly above the cost of goods. the only way to profit clee manufacture them are to do it in great scale. we've seen underinvestment in manufacturing because there aren't a lot of margins and manufacturing these isn't trivial. it's one thing to manufacture a small molecule drug and have a margin that's slightly above cost of goods, quite different when you're trying to manufacture a sterile inject i believe drug. things go wrong and when things do go wrong if one facility gets shuttered it could take down 30, 40% of the market. we think there's things we can do applying additional regulatory touch to some of these critical drugs, if we can design a cot gore which are critical access drugs we don't want to go into shortage but i'm concerned that my regulatory touch will only exacerbate the problem insofar as it will increase the costs. what we're trying to do is look at this holistic clee and bring

in our colleagues from the va and cms and look at how we might couple regulatory steps to mitigate shortages or try to prevent them with potential changes in the reimbursement structure. we talk about drugs that are priced too high and there are drugs that are very costly and probably, you know, exceed the value that they are delivering, but there's drugs also that probably are priced too low relative to their importance and the costs of manufacturing them and i think we need to take another look at how we reimburse these old sterile parental drugs, off patent drugs. >> thank you very much and my time is expired and i yield back. >> chair recognizes the gentleman from new jersey, mr. pallone, the ranking member of the full committee. five minutes for questions, please. >> thank you, mr. chairman. first i have a question for dr. gottlieb and then i want to go to dr. collins for a second. last week fda announced the release of its bio similar action plan which strives to encourage more innovation and

competitive in the biologics market. if patients are to realize the full benefits of bio similars, while eight years have passed since the biologics price competition and innovation act only three are marketed in the u.s. despite fda having approved 11 of them. so have you noted yourself, dr. gottlieb, competition is anemic. i have two questions, first, you are well aware that the high cost of prescription drugs including biologics continues to be a barrier for many patients. as a part of the action plan release you revealed that a recent fda analysis found if all fda approval bio similars were available to american consumers that significant savings could be realized. could you suggest further fda's analysis and the savings potential that bio similars could offer? >> i appreciate the question. we're going to be publishing the full results of that analysis soon but what we did is we looked at the experience in the european market where we saw

product introductions and extrapolate that had experience to what could have happened in the u.s. market if the same products had been introduced in the u.s. market. so we effectively took the competitive landscape from europe, made some corrections for the fact that the dynamic in the european market is slightly different than the dynamic in the u.s. market, but looked at the percentage price reductions when one, two, three, four bio similars entered the market and when we extrapolated those findings back to the u.s. market and assumed if every bio similar that was approved in the u.s. market had launched, we extracted an additional $4.5 billion would have been saved in 2017. so the savings are quite significant. if anything we probably erred on the side of underestimating them by being conservative in how we did our analysis. the savings aren't -- when you look at the european experience the savings aren't of the same magnitude in the small molecule world but they are quite significant. they start to approach the small molecule savings once you see four or five product

introductions. >> thanks a lot, really. second question, i always want to commend you for your continued dedication to curbing -- to delay or impede competition. the question is how can congress work with the fda as you implement the bio similars action plan to help facilitate greater competition in the bio similar space? >> i appreciate the question. congressman, we are going to look at some of these more difficult scientific questions that i think, you know, could facilitate more competition like interchangeability, but one of them in particular that we're going to be looking very hard at is being able to use the european product as a reference standard. we have situations where bio similar might be manufactured in the same facility and distributed in both the u.s. and europe. we know that, but our knowledge of that constitutes commercial confidential information so we have to require a bio seminar entrant to run the study against the u.s. reference product even

though the european product is the same thing and the european product might be cheaper and easier for them to source. so there are opportunities, i think, to have cost savings if you could source a reference product about half the costs right now of developing a bio similar -- it's highly similar, but on average it's the cost of acquiring the branded biological to run the trial. to run the comparative trial. so the savings could be significant. >> let me get to dr. collins. you mentioned in your testimony that nih is building the data resource which will help researchers study the data collected from participants and collect the other large data sets making it available to the broad range of researchers. data security and participant privacy comes to mind. given that nih is asking

participants to share intimate details how do you plan to ensure data security and are there protocols to share updates or study results with participants? >> i'm going to ask dr. deveiny who is all over the issues to respond to your question. >> thank you for the question. so we're thrilled about where we are -- where we have come. we have over 86,000 participants. we know as we try to engage diverse participants up to a million or maybe more we need to regain their trust. so we are all of the day that comes into the database resource is encrypted and identified and goes into the secure cloud environment. once we open up access to researchers next year they have to abide by a code of conduct and no date will be allowed to be downloaded from the environment. in addition and just really briefly i want to thanking the committee forgiving us strong privacy including

confidentiality and am lows -- allows us to provide to our confidentiality. >> thank you. >> chair thanks the gentleman. mr. upton, the author of the cures legislation. five minutes for questions. >> thank you, mr. chairman. i want to remind everyone here that this was a great committee process that passed 51-0. and i want to say that everybody that was on the committee in the last congress actually had a piece of this. because we listened to everybody here. staff was terrific. that's for sure. but so are the agencies as you helped to lead us where we want to go. the administration, the senate. we had all the disease groups, we had lots of players and we appreciated that input. i think based on the hearing today and what we really thought was going to happen is fulfilled our expectations. we got a lot of time left to play. it is game changer. i have to say way back when i

was one of the participants with john mccain and paul wellstone and doubled the money for the nih back in the '90s. we sponsored the bill to get it done. it was an important element that we increased the money for the nih knowing we had a terrific steward in dr. collins who would lead us there. i remember sitting upstairs with mr. poll loan and a few others to talk about how much money we wanted to add to the base line we added up to $45 million. question came up with -- we came up with the pay forces as they were stolen the first time. and a comment that was made earlier might be more important to have maybe someone from hhs here but i was too alarmed last week when i read a story that

hhs was perhaps reprogramming some of the money that we had done a dollar for dollar year by year table to make sure that that money went for the nih. i don't know if dr. collins if you're able to -- if you have knowledge, this is some truth to the published reports that money was being taken away from what we're able to do and put to something else. i don't know if that's happening. if it happening, how do we get it back? >> so i'm not aware the published report you're mentioning. i believe we should check this language in 21st century cures that prohibits the transfer of the funds that are allow cased in the innovation fund by the secretary for any other purposes. i think this is nicely designed so that those funds are intended to do for your original plan. >> good to hear.

i want to talk about all of us. i think privacy protections are important. it's very important. people know about them and they have to worry about that data being stolen. you know, as we found from marley from our roundtables people were -- individuals were more than willing to share what their own experience was. knowing that it was going to protect someone else from having that same ailment. and so it's exciting to hear that 87,000 people have already signed up really in a couple of weeks. i know for me, particularly with a privacy protection we'd like to set up a caucus around it can be -- and it can be bipartisan. we have 25,000 people who work on the hill. maybe we can get a good percentage that will come down to the gold room around the corner to figure that out. how quickly -- i know that

researchers are going to have access to this information in about a year or so. how quickly do you think some of that data might be able to be utilized and figure out the right pathway for some real important research to be promising? obviously the news in the last couple weeks about the chemo and not having chemo for breast cancer was very important. heralded around the country. but what -- how fast do we expect that maybe some of this -- all of this information might be able to be used by the researchers with some concrete result of a positive -- >> yeah, it's a great question. we are really excited to get some of the brightest minds around the country actually accessing the data. as you mentioned 86,000 participants it will get richer

as we get more electronic health data in. once we give access to researchers we would start to see some significant findings but of course it gets richer with more participants as well and we see people who are healthy, stay healthy, how they get disease and all that. >> real quickly, how long does it take for someone to sign up to do this? what's the time element for them to participate? >> so it takes 45 minutes to authorize your elect frontic health record and people doe nate flood that takes between 45 minutes and an hour. we'll asking the participants to fill out surveys and maybe even come back for in person visits. >> i yield back. >> okay. thanks, gentlemen. the chair recognizes the gentle lady from california.

>> thank you. good morning, doctors. it's an honor to have you here and i can't help but think that while most people in the country don't know our names or what we do, they're all counting on you. so thank you for what you do. i think you represent the real genius of our country. and i always say nih stands for the national institutes of hope. you gave us even more hope with your report, dr. collins. dr. gottlieb, i know that you're aware that the legislation that my wonderful colleague congresswoman susan brooks and i wrote the strengthening public health responsibility act was included in the 21st century cures act. amongst other provisions the bill established a priority review voucher to encourage the

development of medical countermeasure drugs and vaccines at fda. when we developed the bill the fda at that time expressed concerns that allowing countermeasures to qualify for the prv would dramatically increase the number of prvs awarded. the first product was just approved earlier this month. so does the fda still have concerns about the number of products that will qualify for prv under the legislate? can you tell if that's been good interest, you know, on the part of companies to apply for it. can you just maybe briefly inform us about that? >> i appreciate the question, congresswoman. i think we all appreciate the effort of this committee to try to provide incentives.

as you noted the first product qualified for one of the prvs was approved by the fda. it was treatment rather than purely a vaccine for smallpox. the holy grail was to have a treatment in the event of smallpox being used as a bio weapon. i don't know the position, my colleagues who are here before me, but we don't have too many prvs being issued. i think if anything it's quite the opposite. we would like to see more drug development in this realm. >> i appreciate that. i don't know whether it's dr. collins or dr. sharpless. pancreatic cancer is still a death sentence.

i did legislation some years ago regarding it. can you give us an update of if the needle has actually moved? i know when i did the legislation i can't remember the year. that the needle really hadn't moved in about 40 years. so can you give us just a quick update on that and then i'd like to go to have deveiny for a question. >> sure. i think the answer is yes, the needle has begun to tick ever so slowly on pancreatic cancer. i was speaking to the group who do great work and i think we have some good stuff. if you can resect pancreatic cancer they're doing very well. a minority of patients but they're growing. we had a positive trial with a

significant survival. the patients who are about 10% some are responding to novel inhibiters -- >> i think i'd like to follow-up with you. because i'm not a doctor anything you just said. but i know that you can break it down for me if we have a conversation or meeting. i appreciate it. dr. deveiny, thank you for your wonderful report. one of the places where the program is taking place is in my congressional district at the palo alto v.a. can you give me just a quick update if you know about the veterans that signed up to participate, how your experience working with the palo alto v.a. i'm very proud of it. it's quite an enterprise there. i have heard from many constituents who were interested who are interested in participating in all of us and i'd like to know how you're advertising to the general public. >> sure.

start with the v.a. we have -- thank you for that by the way. we have a really great partnership with the v.a. and they have been running the million veteran program. they have 670,000 veterans or more already involved. the p.i. at palo alto has been a great partner. they have had great success getting veterans enrolled into our program which has been really -- it's a really important population for us because of of course the health outcomes that are so important there. we have the v.a. site in boston. we're excited to get more launched from around the country. >> great. how are you advertising? >> we're using a number of different tactics. how do we engage a study that's national. we use digital marketing. we are doing in person engagement. the health care provider including the v.a. site are critical for us because they have the district relationships with the patients.

>> thank you. i yield back. thank you to all of you. >> recognize the gentleman from ohio. >> thank you, mr. chairman. thank you very much for the panel being here. i really appreciate it and the testimony you have provided. it's important for this country and also the gentleman from michigan is not here right now. but i want to thank him for the leadership on 21st century cures. dr. collins, if i can start with you, you talk about the brain initiative and you talked about that, you know, it will lead to increased understanding for brain health and a means of preventing brain disordering such as parkinson's, autism, drug addiction and draw mattic injury. i see that there's about $1.5 billion that's been allocated over the next ten years for the studies that's

going on. i know that when i have been at ohio state and seeing what they're doing with parkinson's and their studies, like the one area i want to focus in today is on alzheimer's. because as we look across the nation today, we are seeing what's happening. the reporting from the alzheimer's association report they're looking at the cost could rise as high as $1.1 trillion by the year 2050. one in ten americans over 65 will have it. we have about 5 million americans living with alzheimer's today. one-third are afflicted with it. when you look down the road, people over the age of 35 they're 14 million people afflicted. where do you see the studies going right now with alzheimer's? >> this is an area of intense

interest as a major challenge for the world and certainly that's very true of the united states. you have quoted the frightening numbers of individuals who are affected and the cost that will apply to the health care system. and the personal tragedies that happen every time a diagnosis appears and affects the caregivers and we are all in trying to come up with an aggressive study to identify a path way towards prevention of the disease. and congress what are r has been giving -- has been giving us greater resources to work on alzheimer's and we're working with the industry. in something called the accelerating medicines partnership and the science we're doing on the brain, what the earlier signs of alzheimer's gets translated quickly.

alzheimer's begins long before the first symptom. if we're going to be successful in delaying or preventing it, we need to find people at risk and not wait until they're already in a circumstance of having lost a lot of the neurons because they'll be hard to get back at that point. i am guardedly optimistic we are on the right path with some of the trials under way right now. where we have the opportunity to treat people before they have any symptoms but we know they're an dangerous pathway and can watch and see what's happening. we can learn about roles that play significant hand in what happens with alzheimer's and the immune system. an inflation seems to be important in this condition. all of the focus we have had on am alloyd and the two proteins. i can give you a long lecture on this but i'll stop now. between nih basic and clinical

science working with industry and with close collaborations with the advocates we are pushing this as fast as we can. >> and i would just add if i may, we work closely with dr. collins on these efforts. we updated the guidance on the setting in this particular disease setting. historically there was a perception you had to show improvement in functional status and cognition to win approval or show you slow the decline. that's challenging because by the time you -- your functional status declines as an alzheimer's patient you had a pretty significant decline in cognition. now that we can identify patients earlier with more sensitive tools to detect changes in cognition and what we want to is intervene earlier when there are slight changes in cognition, try to arrest that process. what the agency has said, if you

show an improvement on cognition alone, that could potentially qualify for approval for a drug, to properly identify patients. so we are trying to work closely with nih as well to help facility this innovation. >> thanks, scott. that's important to have that as an end point. >> thank you very much for the panel and mr. chairman i yield back. >> thank you. >> could i ask for the next american of congress to sit up in the ranking chair as a cosponsor of the bill? >> no objection. >> the lady is recognized for

five minutes. >> thank you so much. i just thought mr. green wanted a break. that was very nice of him to recognize me. and again, i want to thank -- i want to add my thanks to all four of the witnesses up here for their extraordinary help not just in passing this bill. but also in implementing it. it really warms our heart to see how much prospective we have made in a relatively short time and it encourages us to think how much more we can make. so i just have a few questions for each of our agencies that are here today. i think i'll start with the fda. so dr. gottlieb, the biomedical community has made great strides in developing clinical therapies from unlocking the potential stem cells to creating therapies tailored to the unique generic fabric of a patient. one of the things we have been

concerned about for a long time overseeing and regulating the cutting edge therapies is -- it requires an experienced well trained robust public health workforce. and so in 21st century cures we had a provision for new hiring authorities known as hr cures. i'm wondering if you can talk about that, how the hr cures authority has helped the agency recruit and retain talent. >> thank you. we're in the process of implementing that. i think it will be transformative in terms of our ability to recreate people, and we have identified 38 occupations for the alternative pay system. we have made the first two hires under the new cures authority. both center directors but i think this is important for the agency and to brief pick up on your point. when talk about new treatments like cell therapy and even the

things on the medical side of the house the clinical characteristics relate to the product features and the product features change very quickly. the underlying technology of the product itself. these are not all pills anymore. if you had expertise, it requires it in drugs as well. that's lot of novel technology that you have to have engineering skills on that specific technology. that's where it's helpful. trying to find those people -- >> these are people you can't hire someone straight out of grant school. these are people with specialized areas -- >> that's right. they're often employed in the private sector where they're highly paid and skilled. we're competing against others.

>> in the fda's june 2018 report called the workforce plank, you talked about the patch work of hiring authority that have created challenges. can you address that? >> we have had historical challenges with the overall framework going back 15 years or longer. some of the same changes i'm grappling with now, we are addressing it very directly. we have -- we are trying to do a top down, you know, wholesale change of the hiring process to make it far more efficient. we started a pilot which dramatically streamlined on boarding process. the hiring process. and we focused an the user fee slots. what we decided to is take that structure and apply it to the whole hiring process and not bifurcate it off -- >> i hate to cut you off but i

can't ignore dr. collins. i have about ten minutes of questions for you. but you have a minute to answer them. let me ask you what kind of research is going on in you agent -- in your agency for opioids for pain management? the opioid crisis has been facing this committee for the last year or two. >> thank you for raising this. this is an incredibly important issue. we're graceful to congress and the $5 billion was put in to work on the opioid crisis and the need to develop effective and nonaddictive pain medicines for the 25 million americans who suffer from chronic pain every day. this goes from basic science to working with industry to help

free up some ideas that has been on the slow boat and now can be speeded up. working in the collaborative way and setting up the clinical tries network to see if they work and in what setting do they work because not all pain is the same. low back pain is not the same as other pain. we need to already to go the kind of network that currently doesn't exist. >> i think mr. chairman this might be a good subject for a whole separate hearing. one last thing. i had some questions about some rules that have been pending about shock therapy and we don't have time to answer those questions right now. but i'm going to be contacting your agency to find out why those rules haven't been approved. they have been pending since 2016. i promised my constituents i would ask you about that. >> i would be happy -- >> thank you.

>> thank you, mr. chairman. i appreciate you holding this hearing. i want to thank all of our witnesses today and the very talented men and women that you're speaking on behalf of in the agencies that do amazing work in the area of health care in america. cures. the legislation that was passed, medical innovation. reforms to america's mental health services and this is why i thought today was an important day, an important hearing to continue our work to evaluate and legislate and evaluate and see what else we need to do going forward. what's working, what's not. what do we need to change? i know i had a lot of round tables and discussions, town halls with people in my district. people are very pleased with the investments we have made in medical research and in turn the work that your scientists are

doing to turn that money and research into reality and medicine and cures that goes from one end of this table to the other. so we appreciate the work you're doing and a friend -- carol said cures was a great step in making it possible to find the cures to ms. i know you hear that every day. people are excited about the cures that are out there waiting to be found and the work that you're doing. dr. got leash, in your testimony today you suggested a correlation between lowering medical product costs and plo -- promoting medical consistent designs. we are concerned about the cost of medicines and clinical research isn't always as efficient as it can be and other tools help to make that process more patient centered.

>> i think while the clinical trial don't directly translate into how the product is priced they priced on what the market can bear. it's in the rising cost of the competitive nature of the landscape. what i talk about today is the complexity of conducting clinical trials when there's already favorite -- is delaying the third and in class drugs. so they're enjoying monopolies for a long period of time and if you know the -- you believe the subsequent competition the prices aren't coming down and we have data to publish this. i think there's things we can do for in the second place drugs

and increasing our safety, not sacrificing it one bit. those are the kind of development reforms we are focused only. >> trials for rare diseases can be difficult to conduct especially if that's -- there's an fda program. >> one i think in particular in rare diseases looking at natural history models to model the behavior of the control arm so you didn't have to enrolle in the placebo trials. they want to use an active drug. this is a place that we could benefit from more disease models. that's a request in the president's budget to help facility that. i know we have congress from those here so we're graceful to

that. >> appreciate that. a good reminder, good promotion for the president's budget. send that to the white house. dr. collins and deveiny and sharpless i saw the nih call respond or research on prostate cancer and men of african ancestry. so that study funded in part by 21st century cures moonshot will investigate the african-american men. can you tell us more about this important study and how it will combine science? >> yes. this is alluding to the trial just announced from the nci that will be the largest trial on this topic in our history. so it's going to take 10,000 patients newly collected to try

to analyze the gee gnomes and african-american men are more likely to be diagnosed and die of prostate cancer. understanding in important disparity in our country is crucial. >> initiate want to add? >> i think i just have to point again to the study as a platform that we'll be able to utilize for answering many questions of that goal. we have about 50% of the participants who will come from underrepresented groups so if you want to look at the health disparity you have a million who are highly motivated. on whom we have greated a great deal of data. we'll learn a lot about all diseases once we have that have platform up and going. >> excellent. it's been positive for the american people.

we look forward to doing more together. >> the chair recognizes if gentle lady from florida. five minutes for questioning. >> welcome to all the participants today. i share your enthusiasm over the excitement of the cancer moonshot. part of the enthusiasm stems from the fact that back home in tampa i represent the moffett cancer center. the nci center in the state oh florida. i love meeting with the young scientists and researchers who feel we have given them a new commitment where they were concerned in the past on the future of nih funding. now they feel like 21 century

cures. the moffett cancer center had been building large data sets. tissue samples and working with other institutions. especially for those in -- that have -- underrepresented communities. you have now given us a bit of outline on the importance of protecting privacy for the people who participate. can you tell us about the protocols going forward for researchers? will they be required to share their research? when would that happen? the research results be available to all for other researchers to build upon? >> great question. to your point about young investigators the diversity of the investigators and fuel responsible for learning the things we learn on the data so

generously provided is returned to the scientific community and to our participants in. we are developing policies around the researchers who are required a code of conduct and share results within a specific amount of time. we're still finalizing the possibilities and possibilities around returning results back to participants in participate in this study as well. >> dr. collins, in the past it's been siloed. if certain researcher held it close, what does the future hold, do you think? >> i appreciate the question. i appreciate the 21st century cures gave me the thortdz i did 23409 have -- authority i did not have before to require data access for studies that we support. i could cajole in the past. i could try to embarrass people but i didn't have the clout to say this is a requirement.

you are required to make your day accessible. that's been a very useful tool. for a wide varieties of study we have done it particularly for genomics where we have a well worked out data sharing policy. we are working that out for imagining and electronic health information and so on. we hate the silos too. we have been having a good time knocking them town is. >> great. look forward to that. from sharpless your predecessor another nci dr. doug lowey was well known for his research on hpv and the vaccine. the part of the cancer moonshot isn't just the future research, but what nci can do to help prevent cancers and detect them. over the past few years, a lot of the nci -- there has been new

nci focus on making sure that there is greater uptake of the hpv vaccine across the country. do you intend to continue with that? if we discovered the cure for cancer there'd be parades in the streets. i think it's important, can you committed to continuing those initiatives? >> they'rer have much -- i should mention that dr. lowey is continuing and it's challenging so i really appreciate doug. his work is wonderful. yeah, we have a robust, huge portfolio. one of the most important trials is one or two doses of the vaccinate. r -- vaccine. that could be game changing for subjects in the united states as

well as globally where cervical cancer is is a bigger problem. >> i guess it's a reminder of back to school time. this -- it's important for boys and girls, middle schoolers to get the hpv vaccine is that correct? >> yes. a number of vaccinations, hepatitis "b" and they work into the community is a real challenge for us. in terms of the information science to make it more available. >> thank you very much. i yield back. >> the chair recognizes the gentleman from new jersey, mr. lance. >> thank you, mr. chairman. good morning to you both and thank you for your enormous public service. for dr. got leeb, the orphan drug act has created a market base system to develop new medicines, most of whom are

children. i'm the rare disease caucus in the house. in exchange innovators are granted seven year monopolies. since the act was established in 1984 there are many rare diseasing as you both -- diseases as you both know. for many of the diseases, however, there has been zero second generation brought to market for patients. in the field of lice asew mow storage disorder, the cost on average is nearly $500,000 per patient per year. however, with the exception of goucher disease, not a single second generation has been approved since the first generation ert approvals. most of those date back a

decade. in your view, dr. gottlieb, have we created perpetual monopolies especially in the areas i have discussed and what are the barriers to moving inknow vax forward and to bring newer, better medicines those with rare diseases as rapidly as possible. >> thank you for the question, congressman. i think this cuts to some of the other issues today so it's a relevant question. i do believe that there are settings where it's harder to bring second to market competition into certain categories. i would argue this is one of then. when you have drugs that tabernacle degenerative diseases, one therapy is available. typically the subsequent drugs have to be studied on top of the

available therapy. you have to show improved ever facasy and hard to run head to head studies. people want to forego the effective treatment especially when you're dealing with a child with degenerative disease. earlier this year we published with in conjunction with the european counterparts and how you can look at the structure for gau shays. we have talked about the natural history models. we know how the diseases progress. we know how they affect patients. we should have the robust history models so we don't have to enroll players on placebo

trials. i agree with you it's critical in these areas. >> is there something more we should be doing statutorily in the legislative branch of government? >> i think there's lot that things that we can do and that we are doing. one i think in -- i affirmed,el with alocated six natural history model for the effects of sickle cell disease on the kidney. we did in our bjts -- budget request up to 20 natural history models. they would be focussed on the resources in. >> thank you. i look nrdz of -- forward to

working with you. this is completely bipartisan in nature and we look forward to continuing the discussion. dr. collins, a pleasure to see you. mr. chairman i note in the audience john crawly and his daughter megan. thank you are residents of new jersey and no one is fighting for the rare disease more than crawly. >> i american you being here today. the committee dr. gottlieb sent fda increasing criminal enforcement at ports of entry. an initiative that you have championed and that we support.

some of the requests were first posted on january 30th of this year. it's late july and they continue to say the staffs are in clearance. could you help expedite the clearance of this letter so the committee has the fda response before the end of july? >> i absolutely will. it is in clearance. and we -- in an effort to try improve transparency i did make most of the information available publicly in some remarks i gave in the forum to try to work with intimate stakeholders to address opioid sales online. i can make those remarks available to you. but i recognize the informal response getting back to congress. i'm on it. >> okay. some of the challenges -- >> well, you have so much to do.

really i thank you for the good work you're doing. there's a lot of different areas we have been working on together. i appreciate that very much. what further improvements will the fda make to ensure the -- particularly now that the federal rights of trial legislation has -- you're in on that? >> well, we have a process under way, we're looking at what steps if any we ought to take to facility the proper implementation of the right to try measure. it's sitting alongside the expanded access program. we did bring in an expert group to take a top down group to make some recommendations and i'll make that public soon. but we think there are additional process steps we can

take to make it easier for people who aren't as sophisticated or physician haven't this done before to access that. and we're creating a platform to have sort of a one site of entry if you will to get information about what expanded access programs exist. i think the challenge still remains the difficulty. sometimes reluctance, but a lot of times difficulty to make the drugs available. especially when we're dealing with things like the cell based therapies. when you're a small country, you barely have enough products fore the clinical trials. i think we need to look for ways to try to make sure that when we have really promising therapies we might be having that

available to patients. >> i have been an active proponent of right to die and if people are going to die, they'll take that hail mary pass. let us know what you we do to assist you. dr. collins, an update. one i have a partial answer that i already found or the internet and that is with lou gehrig's als. i understand you had a breakthrough this spring. i have friends who have been afflicted with different diseases. >> with als as you mentioned there's encouraging development of a new therapeutic approach this spring, still in the process of being looked at. huntington's disease, my own

laboratory was involved in the gene that is responsible for that condition. it's gratifying to see, very encouraging information about using a genetic approach and now in the human chin tall trial, it's sounding encouraging. utilizing a molecular therapy injected into the final fluid may be able to provide benefit. we have waited a long for it to happen. we have a long way to go before we have the answers. but this is a lot different than saying we have nothing to offer. >> i appreciate that very much and i yield back. >> the chair recognizes the yes marn from -- gentleman from california. >> thank you very much.

thank you for the progress that we have made and not every day do we as the united states come pass -- congress pass laws that say, we did something good. i want to thank all the implementers for doing good work. my first question is the diversity in research of subjects. so thank you for testifying today. i was excited to read in your testimony, dr. collins, 85,000 have started the process and 70 to 75 are from communities who are underrepresented and almost 50 % are from racial and ethnic groups who have -- not been included in previous research. i believe one way to decrease health disparities in this country is to ensure that we're

including all ethnicities. with that, what is nih doing to ensure that we recruit a group of people that areth in nickly city verse? >> aim asking -- i'm asking for that every week and i'll ask deveiny about how this is done. this is unprecedented to have that level of diversity. >> yeah. understanding that ultimately a lot of the data scientists are using today is not reflective of the diversity of the country and what we're learning is not applicable to all communities. we have a number of different ways that we are attempting to reach out the diverse communities and kind of go in and shift to new things. i want to highlight working with community partners.

we have a number of community engagement partners who are helping us to build trusted relationships on a local world and our chief doctor really working with those group z -- groups in a robust way. and they're helping us to build away. >> so you're talking about working for a propensity with the lie cal population, like a black population, et cetera. working with local groups that have relationships with those communities? >> absolutely. >> when we did the launch i was in new york with an african-american community that was revved up to be included this time. i want to focus on inclusion, you have given us additional

tools and in many different projects, but all of this is turning out to be a flagship in the most visionary way. >> so this opportunity for us to remind ourselves that diversity and research when it comes to subjects, et cetera, because they all genetically have different reactions, et cetera. different propensities when it comes to certain diseases and cause and effect, correct? >> absolutely. one should not assume when you see a health disparity that's something readily understandable only the basis of looking at one thing. these are environmental exposures of stress leveling, is of only genetics in there one shouldn't overintermet that market. you need the studies figure and

having to intervene. >> that's more guess work -- >> you want the data on the people. we heard from dr. sharpless about the big study. they're starting with african-american prostate cancer. we don't understand that men to have prostate cancer are black and have a higher likelihood of the aggressive disease. >> what are some of the challenges that are building a cohort? are we hoping to tackle in the near future that you venture into the diversity effort than ever before? >> we would love to follow-up up with your office as well. we want to see what's working, what's not working as part of --

even things like your parking isn't close enough to the clinic and therefore i can't -- i can't make my appointment time and make the feedback. >> having a good feedback system and adjusting all the way is important. so you're doing that? >> yes. >> thank you very much, mr. chairman. >> the chair recognizing the gentleman from new jersey, billy lodge. >> thank you. i want to give a shout-out to diana cadet. we travel to japan together every year and for fred upton on this 21st century cures. they did yeoman's work. i want to give a shout-out to

them. my daughter was diagnosed with hodgkin's lymphoma. and she's three years past the chemo. i want to thank everyone. dr. sharpless, we were happy to support the cancer moonshot finding the cures and are looking forward to hearing about progress for patients. can you tell us about the most exciting thing that is being supported in the cancer moonshot? >> well, that's really tough. i think this is a very meaningful bill, law for patients with cancer. i think it's impacting many many ways -- we talked about the response. maybe i'll talk about the rare tumor initiative which is an exciting initiative. we mentioned rare diseases. many rare cancers are hard to study for some of the reasons that came up. they're rare and hard to do clinical trials. we can have them come to the nci

and this works and the moonshot is trying to build on that experience. for example, in a scanner is drug, different mew substitutions in ras, they generally present with childhood tumors. and there was a recent shroud present on the left is is andrea gross showing the results of the trial in the ras on athy patients. the tumor shrinks, the kids seem better and parents say my child has been on this trial and it's marvelous. so pretreatment on the level. you can see the wind pipe is not compressed by the tumor anymore. and he has not the social stress

of going to school with a big lesion like that. ras, the cancer gene. this is a great thing for the nci to take on because of the intramural program. >> okay. and the testimony says we must transform the way we share results and the way we get d discoveries into patients' care. can you talk about that, how they're meeting the goals? >> the virginia scientists? >> yeah. the new funding from congress has made it possible to increase the success rates for new scientists applying for the first grants. nci we set aside enough funds and trying to lengthton period of the award. we think that will allow them to focus on the science and not writing the grants. we are testing out a five year award and not a seven year award.

we're waiting for the training opportunities both in the postdoctoral setting. >> if i may, just across all of nih and thanks to the 21st cure and the initiative which is part of this bill, we have mutt into -- put into place the efforts to provide a better chance for early stage investigators coming here with their first major grant application so they have a career. this year fy 128 we are funding we'll make this shift in priorities and you helped us with that. with 21st century cures. >> thank you. i met with the cancer research and i was very pleased with the excitement and passion that they have in helping cancer patients. once again as a father of a

cancer patient that is a cancer patient survivor, it really means a lot to me. thank you for being here today. so i yield back. >> thank you. chair things mr. sarbanes. >> thank you all for coming today. whenever you testify i'm reminded of the tremendous responsibility that you have heading the organizations that you do and i want to thank you for your work to implement the 21st century cures which is a point of real pride for the committee so thank you for that. commissioner gottlieb, i snow there have been dis -- i know there have been discussions about the bisimilar action plan that you've released. i commend for you that. i just have introduced in the last few days the bio similars

act to get to pay for delay agreements that are operating in the biological, bio similar space in the same way that we have given authority to the ftc to kind of police that conduct with respect to brand name drugs then generics. it has been consequential for sure that the authority is there. that they can look at these agreements. they can judge whether they're fair and appropriate, vis-a-vis the consumer or not. and there are tremendous savings to be had there. i think the figure is the brand names occupy about 20% of the drugs that are produced every year, but still 75% of the cost that's out there. so i think that includes the

sort of biologicals distinction as well. can you speak if it's a good idea for the ftc to have this authority and you look for ways to forward in your comments that you look for ways to cooperate with an agency like the ftc around this kind of thing, to make sure we're getting these drugs at the price point that they should be at. >> i think that the question, congressman, i haven't looked specifically at the measure, but i will. it's a little outside the scope of my authorities. i have my own equities with ftc and trying to work with them to try to see how we can facilitate their interests and potentially bringing cases related to delaying tactics that might involve, for example, the rems where we can help provide information to take a look at whether those practices are anti-competitive. that's been a big bugaboo of mine. we like to provide safe and

effective bio similars and see the market benefiting from them. we like to see drugs marketed. it is a fact most of the bio similars have not been marketed for various reasons. the biggest reason has been patent delays. a growing number of small molecule drugs are being approved but never marketed because of the changing economic dynamics of that market as well. we need to keep a close on that because the competition may be declining in the small molecule market as well. >> thank you. we'll try to get this tool into the tool kit that the government has, broadly, to make sure we're protecting consumers in that space. let me switch gears real quick to you, dr. collins. i know you got a question earlier, i believe, about alzheimer's research generally and some of the clinical trials they're associated with. but there was an article in "the

new york times" yesterday that presented the challenge finding people to participate in these trials. there's a lot of trials that are under way or sort of on deck that would suggest the need for up to 25,000 participants in these trials. and some of them could be really breakthrough. but i gather this is a real problem, challenge trying to find enough participants. can you speak to what you know about that and what can you dob about it? >> yes. thank you. that was based upon comments by dr. murray bernard. at the major alzheimer's meeting in chicago. we're at the point trying to recruit individuals who don't have cognitive decline but at high risk for alzheimer's by increasingly accurate means we have, some using imaging, some using genetics. we need a lot of participants in order to do that. but now we're reaching out to people who may not be that motivated to take part in

research because they're fine right now. we are arguing that that is the best time to intervene but it's not so easy to enroll. i will say one of the dreams i have of the all of us program we've been talking about where you have a million participants who are preconsented for recontacted for research protocols that will be a fantastic group to be able to enroll participants in studies like this for common disease, whether it's diabetes or hypertension or alzheimer's. but right at the moment, where with he don't have that platform, we really are having a challenge trying to convince people this is going to be something they want to take part in. we're pulling out all the stops. again, the congress having made alzheimer's such a high priority for us, we have resources in order to do that kind of recruitment, but it is not simple because it's a different model than what people are used to where you get approached about a clinical trial when you already have the diagnosis. here we're approaching people that don't have the diagnosis, trying to prevent it. >> thank you very much. yield back. >> chair recognizes the gentleman from indiana, dr

dr. bishon. >> thank you. as a physician, i never thought i'd get to talk to the -- you all. and i was at an event with dr. sharp, with steve rosenberg and diana de gat recently, i can't even express how much of an impact he has had. dr. collins, you just said high risk. who's high risk for alzheimer's? >> well, anybody who gets to be 85 or 90 is high risk. >> i'm saying, people that don't know they have it, if your mom has it, your dad has it, your grand month? because i think that would be an area that i -- you know, where maybe if one of your family members has it, you're high risk. i don't know. >> there are three ways in which we are currently identifying such folks. one is you have a very strong family history. almost inherited in a dominant

fashion. in fact, it is a dominant fashion. there are those families and if if you're in one of those circumstances, we can track the gene. another way is to look at genetic risk factor calls apo-e-4. if you have one copy of that your risk goes up three-fold. if you have two copies it goes up 15. third way is scanning using a p.e.t. scan that picks up amyloid because it starts depositing in your brain probably 20 years before the first cognitive decline symptoms. if people are worried, we can do a scan and say, you may be one that should get in the clinical trials. >> if someone is watching c-span and they're going, i wonder if i'm high risk, i understand the technical tests and things you do, but, you know, to -- people need to say, my mom had it so i'm high risk. you know what i'm saying? >> indeed, that is one -- >> that's one of them, right. >> but there are other ways to try to be more precise about it.

if peach are watching c-span out there and wondering, hey, should i take part in this, we just heard how difficult it is to get the word out there. the place to go is clinicaltrials.gov which posts trials on alzheimer's disease and everything else. you can find out the enrollment criteria, who do you contact to find out more. all of this we do closely with the fda. >> thank you. dr. gottlieb, my wife is an anesthesiologist. she's out in the field today practicing medicine. she just texted me. she didn't even know you guys were here and she says, hey, can you check into the fentanyl shortage because there's -- i'm not kidding. she just texted me. she said -- >> she's not watching c-span? >> no, she doesn't. she's actually practicing medicine. we demonized fentanyl, the illicit form, but this is a common anesthetic agent and apparently there's going to be a long-term back order on fentanyl. you don't need to answer the question.

i just want to point out that what you're doing on drug shortages has real, everyday -- and you know this, but for everyone else out there -- clinical implications in the practice of medicine. z zofran, other common medicines she uses every day. fentanyl is a new one on me. she just texted me that. and thank you for your response on that. if you have any other further comments as i have one other question for you. >> you know, i will just say, this is -- these are structural problems, as i mentioned before. whatever is in shortage today, six months from now i guarantee it's going to be something different. i've glrappled with this myself. this impacts patient care. we're working hard on this. >> 21st century cures, this is a little proprietary question, for diseases such as cancer that relies on diagnostics. i'm sure your staff told you i

might ask this. the current diagnostic framework for laboratory tests, lvts and diagnostic test kits, haven't been updated since 1976 and 1988. that's why dyi diane and i have intruced how to get those introduced. we have a draft out there. we appreciate your input. and we've talked to laboratories, patient groups, providers and others and we need reform now. i know you're familiar with the issue and i brought it up in the past. as well as the committee's last hearing. we're -- you recently gave us a narrative on what the fda provided us earlier this year in response to that, but i want to get diagnostic reform done this congress, hopefully. so, we're waiting for the red line of the bill from fda and i wanted to know if you have any insight in that progress. >> it'ses very close,

congressman. we've completed most of our work. i hope to have it to you very soon. i'd be happy to come into your office and brief you on it as well. but we're very close. >> i want to reiterate, you've been very helpful. i'm appreciative of that. we're hopeful to get this accomplished this congress. thank you, mr. chairman. i yield back. >> chair thanks the gentleman. chair recognizes the gentle lady from indiana, mrs. brooks, five minutes for question. >> thank you, mr. chairman. thank you all for your service to our country and for all of the incredible medical innovation. just want to thank you because you all could be probably be doing different things in our country and yet you have -- are here, you know, working on behalf of all citizens. not just here but actually around the globe. dr. collins, i'd like to ask, in your testimony you mentioned that there are provisions that provide direction from congress to ensure specific subgroups of patients and specifically include clinical and research trials. i'm specifically interested in childhood disease and cancers

and having heard from constituents who have lost a child to cancer and are currently fighting the disease, we know that nih is focused on improvements for children. an area that's been lacking in the past. can you elaborate on the implementation of the recently passed childhood cancer star act, which was signed into law in june. and what kind of innovation are we focused on for childhood cancers and disease. >> i asked dr. sharp to respond to that. >> thank you. >> thank you for the question on childhood cancer. it's important to say two things about childhood cancer. while it's true we've made tremendous progress, if your children are dying of cancer, we're also having true too many children die of cancer. in particular, even when we're able to cure kids of cancer, we often leave them with life-long toxicity so they have survivorship problems.

star act tried to deal with the burden of survivorship some of these patients endure through curative therapy. the nci is interested in this topic. this was a personal research of mine when i was in academics. i know firsthand the pain these patients go through, the suffering this entails. we have a focus on new research efforts related to survivorship by a specimen acquisition and getting new voices into nci to make sure we're advised from the community. thank your leadership for that. >> dr. gottlieb, as my colleague talked about on papa reauthorization, which it's critically important we get that reauthorized by september, one of the things that's come up in various discussions with dr. cadillac and others is importance of platform technology when it comes to

innovation with vaccine. can you talk about that to some extent because there's been frustration in this committee about ed-based vaccines versus platform technology. or if anything would like to comment. i'm starting with you. >> i appreciate the question very much. we have a proposal in the president's budget for additional funding to try to support continuous manufacturing, alternative manufacturing. we think this is the direction we want to head in where you are closed manufacturing platforms with technologies where you can effectively have like cassettes you basically plug into the platform and can allow continuous manufacturing of a vaccine. if you want to modify the vaccine, you can literally plug in another cassette that codes for different permanent you tags of the same vaccine. we think this is really a solution or a solution for

influenza seasonal flu, to produce vaccine closer to flu season to guess the strains better and have a platform available in the case of a pandemic flu. these technologies can be used to scale up other vaccines. ideal circumstance what we would have isn't mothballed vaccine that degrades over time if it's not stored correctly and is costly but manufacturing that provides quick manufacturing of vaccines, if we need it. >> dr. collins quosworks very closely in this swas. the idea of having to build things in eggs is so much yesterday's technology. the concrete example this past year with the flu vaccine having been surprisingly ineffective. the virus mutated in the process of being grown in the eggs and, therefore, turned not to be particularly effective vaccine for something we didn't have control over. these new plat forms that allow you to build vaccines in a much

more rapid fashion, dna-based vaccines, once you have that platform going, you can very quickly adapt it to many different path agains. that's something we're working on now to develop this universal flu vaccine so we don't have to try to guess right. you would have one to work against all strains and against the 18, 19 style pandemic which we're worried about and overdue. >> thank you. we did include a pan flu provision in the legislation passed out of this committee. with that i yield back. thank you for your work. >> thank you for that. >> chair thanks the gentle lady. yields back. chair recognizes the gentleman from florida, mr. bilirakis. >> thank you. we talked about alzheimer's

disease, of course. and what about parkinson's, as far as, you know, we're talking -- maybe how do you know if you're a candidate for parkinson's disease to participate in these clinical trials, if there are no symptoms? it may be at early stages. and if you could answer that question, sir, i'd appreciate it. >> there are parallels here that are notable. there are genetic risk factors for parkinson's which is interesting because i said, is there any disease that has any genetic? he said, parkinson's. he was wrong. if you have lurc 2, it goes up. we are beginning to, therefore, be able to identify people at high risk and invite them to take part in prevention trials. another big thing that's happened in parkinson's disease in the last six months is the

formation of a partnership with industry called the accelerating medicines partnership for parkinson's disease with fda as a critical partner in this as well. and really figuring out how we could learn from a very large amount of data that's out there but hadn't been brought together. what are the next generation of drug targets for parkinson's and how do we accelerate the process of getting there? we have treatments. eldopa has been around for a long time but we don't have anything to treat the progression. they treat the symptoms. the brain initiative, which is this very bold effort, supported by 21st century cures and the innovation fund, is learning things about the wiring diagram of the brain. that it's going to be very relevant to some of the things being done for parkinson's disease with direct brain stimulation where you put an electrode into the brain to take care of the motor problems. what we do right now is clunky. as we learn the wiring diagram, we could be much more precise and effective about that.

>> thank you very much. anybody else want to add something with regard to that? okay. thank you. dr. gottlieb, sections 3088 clarifies that fda has the authority to grant emergency use authorization for animal drugs, allowing the agency to approve the gmo mosquitos for, again, florida's zika problem. we're planning ahead. would you provide an update on the implementation of section 3088, specifically as it relates to the approval of the gmo mosquitos. >> i can get back to you with a more detailed update on that, congressman. i will tell you there's been some discussion about the nexus of authority with the e. . a for some of these products. we provided guidance earlier this year, i believe, that addressed some of these issues. i can get back with specifics on where that stands. >> i appreciate that very much. dr. gottlieb, second question,

as a long-time champion and supporter of policies to support deeper level of patient engagement in the therapy development processes, i'm pleased with the progress the agency has been -- has made under your leadership, so congratulations. we appreciate all you do, including the fda's moving ahead to implement the patient focus impact assessment act, provision of cures. that requires fda to disclose how patient engagement data informed a review of any approved product. where is the agency presently in implementing this provision, particularly efforts to standardize the inclusion of such information in the record of approved drugs so that it's accessible and understandable? >> we've implemented -- we issued one of four guidances we intend, and we have standardized

a format for the presentation of patient-related information in clinical trials. so, when a clinical trial is submitted to us, there's a discreet, very explicit section for patient-focused information. on the medical device side of our house, we've done some similar things. we're seeing a very high rate of the use of patient-focused information and pros in the development of medical devices as well. this is a cross-agency effort, across all of our medical product centers. we also stood up a patient affairs office inside the office of the commissioner reporting into the principal deputy that's going to help advance some of these policies, really a coordinating office to provide a focus of access for patient groups, but also a focus of policy-making when it's cross-agency policy-making around these issues. >> okay. thank you very much. i yield back, mr. chairman. >> chair thanks the gentlemen. the chair recognizes the

gentleman from goernl goereorgi mr. carter. >> thank you for being here and all your efforts. implementing 21st century cures, which i feel is some of the best legislation we've passed in congress in quite a while. we're very proud of particularly coming out of this committee. dr. collins, i'll start with you. i wanted to ask you about -- particularly about one of the initiatives of 21st century cures. that was to really review the regulations and policies with respect to research in laboratory animals. and as i understand it, you're working with usda now and the fda to try to complete a review of that. i just wanted to ask you if you could tell us the current status of that review and when do you anticipate the completion of that review. >> i appreciate the question. we are very seriously engaged in this. again, 21st century cures gave us some clear guidance about what we ought to engage in. we put out an rfi back in march

to ask for comments in this area in terms of whether the oversight we currently apply to animal experimentation is sufficient or whether it has areas that are overly bureaucratic, which has been a concern. obviously, we're deeply kerpd about maintaining our ethical responsibilities in terms of how we take care of animals subjected to various experimental approaches, for which we have learned a great deal. we got 19,000 responses to that request. and they're currently being sorted at the moment. we would expect, therefore, to have based upon those a draft set of recommendations about animal care and use some time probably in september. we'll then need to have responses to that. so, we would hope to have a final version of this by december or early in 2019. >> great. great. any opportunities you've identified thus far that may help you? >> i think there are concerned that some of the requirements we

put on grant applicants in terms of animal care and use could be delayed until the award is actually made as opposed to asking them to have all of those things in place when they submit an application, because that can add a lot of time and effort. and, obviously, our concern is if we're going to make the award, we want to be sure the animal care is being done in the best possible way. that is one area. obviously, there are differences of opinion here. we're seeing those, in those 19,000 responses. and at some point, we have to try to come down in what we think is a fair and balanced approach. >> and i'm sure you'll reach that. i'm very confident. let me switch gears and talk about something that's very exciting to me as a pharmacist, and that's precision medicine. and that's something i see genomic testing is something i see as the wave of future and great opportunities for us in health care. but i'm concerned, when we get all of this data in, we're struggling already with our electronic health records. how are we going to handle this? i want to ask you this and then

i would like to ask dr. gottlieb as well. >> i'll say a word and ask mrmr. mr. devaney to say more. we're interested in this, cutting edge dealing with large data sets, putting the data into the cloud. we just had an all-day workshop on artificial intelligence. at the same time maintaining the confidentiality, the security systems that the participants in this are going to expect about their identified data. >> i would like to add to that. one of the data types that's going to be the most essential to precision medicine or one of is information from electronic health records. this is one of the largest challenges for our program. we have direct partnerships as many health care organizations and we're getting that data continuously from the partnerships and we're working on other strategies including one in partnership with four of

the largest vendors right now to work on making the data transmission much more seamless across provider lines and into the program when a participant authorizes it. >> great. dr. gottlieb, i know this is important in drug data development as well. >> i appreciate the question. i would just take a step up the continuum to try to make an effective use of the data because there's so much information, how can we make effective use of the data in ways to translate to clinical benefits to patients? i think this is where some of the issues we talked about earlier with clinical trial innovation comes in, where you have the ability to seamless clinical trial design to effectively bias enrollment in the trial for some of the genomic information and predictive information that's likely to predict who's more likely to benefit from a treatment and less likely to experience a side effect. if we can use this information in that way to structure trials and enrollment, we can end up with much more information about who's likely to benefit from a drug and more tools to make sure that the right drug gets to the

right patient at the right time. we now have -- we talked about this for decades. we now have that technology at hand. >> and, you know, i can sense the excitement in your voice. and it's exciting for me as a health care professional as well. i just look into the future of this and just think, wow, what we've got to look forward to. thank you. and i yield. >> chair thanks the gentlemen. the chair needs to state that without objection, all members' opening statements will be made part of the record. the chair asks if the gentleman from texas has a unanimous consent request? concerning alzheimer's? were you going to ask additional information before to the committee about alzheimer's? >> if you could, just send it to the committee on some of the information you couldn't have given us today because of the time limits. >> happy to do that. >> thank you. >> and to -- >> i forgot what i was going to ask.

>> and i also -- the chair would like to make the observation, dr. collins, you started out this hearing with the remark about the immunotherapy and some of the dramatic things that have occurred. and i'd just -- it predated our passage of the cures bill, but former president of the united states in july of 2015 went public with the information that he had metastatic melanoma to the brain and liver. and remembering my time in medical school, my initial thought was, we will not have this individual with us by labor day. but it has really been dramatic to see him a year later deliver a speech at the democratic convention, a year and a half then he was present at the inauguration in 2017 -- in january 2017. i don't know what his clinical status is now, but it was truly dramatic. again, all of you are to be

congratulated for making that possible. and hats off to former president carter for going public with the information and entering a clinical trial, because that is the way information is gathered and learned. so, again, i felt obligated to make that -- make mention of that milestone. seeing there are no further members wishing to ask questions, i want to thak our witnesses for being here today, pursuant to committee rules i remind members they have ten business days to submit additional questions for the record, ask witnesses to submit their responses upon receipt of those questions. without objection, the subcommittee is adjourned.

we'll return live to capitol hill a little later today here on c-span3. secretary of state mike pompeo will be testifying about north korea, president trump's trip to the nato summit and his meeting with russian president vladimir putin. second pompeo will start at 3:00 p.m. eastern. you'll be able to watch it live here on c-span3. tonight the hearing with fda commissioner scott gottlieb and nih director francis collins, that just wrapped up. you'll be able to see that hearing on medical research programs in its entirety tonight starting at 8:00 p.m. eastern on c-span3. join washington journal friday live from baltimore, maryland, as we spotlight the opioid epidemic. during the program we look at how city and state agencies work

with city residents who are addicted to opioids. on the program baltimore city health department commissioner of health dr. lena winn on the challenges cities face with opioid abuse. and director clay stamp on efforts the state is making to counter opioid use. maryland democratic congressman cummings talks about his effort, along with senator elizabeth warren, to direct over $100 billion in federal funds to curb opioid addiction. and baltimore's acting police commissioner, gary tuggle and niles ford discuss the opioid crisis from the perspective of first responders. watch washington journal spotlight on the opioid crisis live friday morning starting at 7:00 a.m. eastern on c-span. sunday night on

"afterwords," sean spicer discusses his book "the briefing: politics, the press and the president." he's interviewed by former republican national committee chairman michael steele. >> and ronald reagan and donald trump are about 180 degrees apart from each other. and yet here we are in this space. how did you navigate that? because we're both reagan conservatives in that regard. so, it -- >> that's right. >> was it a little bit of a, you know, dance every once in a while or how did you do it? >> with respect to the president himself, look, there's no question. he is not traditional in terms of how he speaks. he has his own vernacular but he also connects to people in a way that most politicians never have. and he talks very bluntly in his own style. but i don't think he would have won the presidency, he wouldn't have won the nomination if it wasn't for that style. and i think there's always this balance with elected officials which is they say all the right things but they don't

necessarily get things done. in the case of trump is he's getting all these things done and people say, i didn't like this tweet or i didn't like how he interacted in this thing. i'm a results-oriented people. i look the at how our country is doing, are people making more money, is the country safer? net/net i think most people would generally agree that if we can get the right things done for the country, then that is a better place than someone just talking about the right things to get done. >> watch "after words" sunday night at 9:00 p.m. eastern on c-span2's booktv. the senate energy and natural resources committee held a hearing yesterday on oil prices, the role of the opec oil cartel and suggestions from iran it could disrupt trading. lisa murkowski of alaska chairs the committee.