gottlieb and nih director francis collins, testifying on the cancer moonshot initiative and other medical innovations that are hard on the 21st aimed at combating alzheimer's and parkinson's. the committee is chaired by senator michael burgess of texas. i asked alvar -- i ask all of our guests to take their seats. 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 collins, 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 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. 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 surveillance 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 prevalence, 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, this allows physicians to offer personalized treatment, achieving the potential of precision medicine will require effort to collect health data and research done by our best research investigators. services to carry out the goals of the initiative while assuring confidentiality of the patient's information. the research program is a major piece of the decision medicine initiative and has already engaged in over one million volunteers in the united states. 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 methods are are being put toward finding a cure

and ensuring the infrastructures is in place so that new their piece are accessible to all americans. it requires them to evaluate for adaptiveses of complex and other trial designs. the innovation of promising results of efforts including cure's will provide americans suffering from cancers and other diseases with the opportunity to undergo successful treatments and in some cases beats your. our thanks to the doctors for giving us the update for implementation of this next law. thank you mr. chairman, and dr. collins and gottlieb we are happy that you are here. the all of us research project, and making certain that we anonymize and protect the data that is in that program, and

that privacy to the issuance with data is respected as we move forward with this. dr. gottlieb, following up on the software act, and making certain that the implementation is going well and we welcome you both, we look forward to the hearing. the chairs: recognizes the gentleman from texas, the ranking member of the subcommittee. >> thank you for holding the today's hearing. i want to thank all of our witnesses for being here today, especially dr. collins and dr. gottlieb. this december will mark the two year anniversary of the 21st century cures act being signed into law by president obama. is to advance the discovery and development of new treatments, cures to increased

research and approval process. this up in law supported medical research initiatives like the cancer moon shot initiative, the all of us research program and the brain initiative. it also provides money to fda to advance the agencies mission and implement policies and underlying bills. congressr committee in is putting these investments to work to find cures for our greatest medical issues. the nih was provided for in a billion dollars in new funding and advanced cutting edge research initiatives. the fda was provided $500 million over 10 years to improve medical product reviews. in addition to this much-needed

funding, there are many provisions in this package worthy of support from facilitating the development of new antibiotics to fighting the uses to advancing of modern trial designs, to investing in a next generation of medical research. some of the provisions are worry is note real abstract. parents and families deserve to have elected officials respond to needs. bit --ll was rewritten this bill was written to do just that. about patient law, focus and evolving, improving scientific expertise, and hiring capacity. many members of the committee are interested in getting cures developed and signed into law. act21st century cures demonstrates what we can accomplish when we work across the aisle. i look forward to hearing from

our witnesses about the oncoming implementation. mr. chairman i yield the remainder of my time. thank you very much for yielding. i want to add my thanks to everybody here in particular, dr. collins and dr. gottlieb and all of their staff who have helped us develop this. especially my compositor a fred fred upton whore worked on this every day. the shows the greatness of energy and commerce committee and what we can do when we decide to work together to tackle a serious problem. i want to thank you for having this series of hearings. it is about a year and a half .ince the bill has been signed almost exactly two years since we initially passed it through this committee. we need to make sure that

everything we intended to do into yours is happening -- in cures is happening. if they need additional research ources and the only way to do that as have hearings like this. i have been concerned lately thatng some media accounts say some of the money may be reprogrammed for other purposes issues of theorr kids at the border. we should be able to find the money to do that without taking money away from important biomedical research and drug and device approval of the fda. i hope that is not the case and if it is i hope this committee a bipartisanand in way to make sure the intended money that we authorized in this committee remains. we still have so many bridges to

cross and we are going to need every penny that we authorized. with that, thank you very much for yielding and i yield back. thing the arrival of a chairman, the chair recognizes the ranking member, five minutes. >> i want to thank dr. collins, ottleib for joining us. of course i also want to thank our colleagues, without which cures act would have never happened. it is looking towards the .iscovery of new cure's the all of us research program, and the brain initiative, the

fda included provisions to include the medical product review process and new authorities in funding to assure the agency has the resources to recruit the best talent. at our hearing on this topic last november, you both provided promising updates and what i look forward to your continuing work. important oversight hearings --ow us to learn delectably directly from the administration about how policies are implemented. i am pleased the subcommittee has decided to implement oversight into the cures act. i have asked the majority to schedule hearings on a number of issues that are priorities for democratic members of the committee. on healthearings disparity, gun violence, the indian health service, cosmetics reform, the office of refugee andttlement, drug pricing, marketplace stabilization. these are all different issues

within the jurisdiction and we may have different opinions on many of them, that is exactly why we should at least have a hearing. solutions.ritical weeks after the recess, i hope the chairman will respond and select a few of hearingsues and hold in the short time we have left for this congress. unless someone else wants my time, i will yield back, mr. chairman. >> 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. dr. collins: 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 century cures act. me are other doctors including 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 colon, 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 therapies, but 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 genomic test can identify them quite 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 -- we 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. gottleib: 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 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 asked if we can afford this coming wave of precision guided therapy. 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 s offer 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 complexity can be a barrier to getting timely competition to newly approved branded innovative drugs. one reason we may be seeing higher costs is because it's taking longer to for competitive on to emerge where specialty drugs are targeting medical needs. 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 competition 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 lier 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. they can help us to get more information about safety and effectiveness at the same time. is pioneering a number of advances in clinical trial design. first is protocols, these include basket trials and platform trials. these approaches can sharply increase efficiency and cost. they allow the testing of multiple drugs against one or more diseases or subtypes by using a common trial infrastructure. compresses theh three phases of trials into one continuous trial. new patients, you expand subsequent cohorts using the information you learned about benefits for a new treatments. we will push guidance soon about how products can expand cohorts

as trials progress. a lot of time and cost of clinical development is spent waiting between starting and stopping the three phases of trials. orry american has already will one day face a serious medical diagnosis either personally or through a loved one. we need to reduce the burden of cost. arecal trial reforms we making today will help in short more patients who find themselves in hard situations will find a cure. thank you. thank you dr. gottlieb and thank you to our witnesses for agreeing to be here. we will proceed to the member question-and-answer parts of the hearing. we will yield to chairman walden when he comes in for an opening statement. dr. collins thank you for being

here, thank you for bringing your backup. let me ask you a question on the we tried to cures, identify ways to get regulations for policy that were inconsistent and to give you some flexibility to move past some of these that are overlapping and not necessarily relieving administrative burden. i think the act to ask you to review that, can you pass share and howwhere you are nih has identified some opportunities to relieve the burden on investigators? of having to do that and thanks for making the changes part of this bill. they make a huge difference to us as we look to carry out our mission.

we think financial conflicts of interest are very important for us to track, the mechanism for doing that tracking is seen as unduly onerous. we are in the process of going through that with our colleagues at hhs. we will make that more of an efficient process. another thing that seems pretty down and the beads but matter to us, the degree to which we need to be monitoring what you call a sub recipient, you give a grant to a particular institution and they give a part of that to another individual. in the past, we had to do it very detailed monitoring, even with sub recipients, we have already moved to simplify that process. financial expenditure reporting, a lot of the report that have been done in the past produced data that not many people look at. at the same time we need to make sure we are being good stewards. we are looking at animal care, and the oversight that is necessary to be sure that we are

dealing with animals in an ethical way. some of those oversight mechanisms are now being -- weep and weep out and got 19,000 comments about how to streamline the process. it was helpful having those features into 21st century cures . think you would find a committee willing to have those discussions. thank you for your comments on the clinical trial reform. i always felt while we were doing the roundtables for cures, that is where the big money was. if we could reduce the time in trial, if a product was going to fail, allow it to be identified and fail early, so we do not spend a lot of time with

something that is not going to pan out, i really like the trial, of the seamless the time between phase one, phase two, phase three clinical trials, i think that is going to have an impact. one of the things that has always concerned me is we have had legislation, but the ability a pair,e do not have but for the researcher, the regulator in the payer to communicate before something comes. i always got the sense that when hepatitis c came down the pipe, the medicaid payers were not ready for what was happening, but they had been prohibited from having does discussions because of current law. i am hopeful we are able to do something. particularly in gene therapy or

gene editing. a one-shot that is going to cure a lifelong problem, that is huge . it is likely to be priced out of the range of most average people's budget. can you comment on that? our ability to communicate higher to approval? -- prior to approval? >> i would say we have fully addressed this issue. your points are well made. i have shared your concerns in the past. i think we have fully addressed this with the guidance we issued earlier this year that was built off a provision related to payer -- communications for purposes of engaging in value-based contracting and other economic kinds of discussions. we have essentially established a safe harbor for those communications. the legaldeciding on

regulation. we would not choose to exercise authority because a matter of public health, we believe there should be discussions for the purposes you suggest, so they can evaluate contracting and other ways to pay for these novels there a piece. >> thank you for that -- novel therapies. next thank you for that. that.thank you for >> let me give you an example of what has happened the last four years. we had an outbreak of ebola. there was no treatment for it. today what we are seeing in the congo, we have a vaccine. what has happened in those four years? can dr. collins or -- share that?

how did that become? we get there? in 2014 there was no vaccine. dr. francis, the vaccine came from him. >> it has to be approved. indexingan working on in the 1990's when no one paid attention, but we were concerned it might become important. there was a fundamental amount of science and work happening for a good 18 years before the crisis that struck in west africa in 2014. that being the case, this was in fact a program that developed the vaccine in record time. still, not in time to have much of an impact on the actual outbreak. by the time the vaccine was being distributed, public health

majors were already resulting in the epidemic waning. vaccine,drc, this manufactured by merck based upon elaboration with nih -- the phase one trials were done in the nih credit -- clinical center -- that was distributed vaccinationring strategy and also giving it to health-care workers. we were happy to see the declaration this epidemic is now over. did the vaccine contribute to that? it is hard to tell. this was a limited outbreak. now tovery well situated deal with an ebola outbreak in the future. it was great to see how quickly the vaccine was available,. -- got distributed.

upfront, i think it is hard to understate what a game changer this is. this is the first time we have a technology available in the setting of ebola to stop the spread with something other than traditional tools of isolation. i think we should make note of the efforts of the many -- the manufacturer in this case. the doses that were deployed in the drc were donated. they shipped 13,000 doses to who. it was delivered under an expanded access protocol. this is very much an altruistic effort. we are working efficiently to try to move to the product in the u.s.. i feel this will be something we can accomplish in the near term. we can have a fully licensed vaccine. >> that is just an example. we had a gentleman from west

africa come to texas in the protocols were not followed. it impacted our own country. paradigm on what we can do for other terrible , inesses that are developing tell people, on any given day we have tuberculosis in the city of houston because it is an international city and you just do not check everybody. we have to be on our toes to do that. yousecurity i think gives those. looking at the ebola vaccine, in four years -- i was trying to explain to folks, more of my constituents will die of the flu because they did not get a flu shot than will ever be exposed to ebola. we want to keep it that way, though. i'm concerned about the growing threat of antibiotic assistance -- resistance. -- tos why i sponsored

meet unmet medical needs. coordinate efforts with respect to monitoring antibiotic resistance and getting drugs approved under the limited population pathway. what steps has the fda taken to coordinate the cdc to support policy that promotes judicious antibiotic use and stewardship? >> we have taken a number of steps. we are going to be taking additional steps to look at antibiotic use in animals. the length and duration of use and the applications in which they are used. we hope to have additional policy steps we should be announcing in the next couple of months to continue to advance what we have already done in to reduce ther -- use of antibiotics in animal route bylimit one

which we are seeing resistance develop. qipd as well as being -- the lpat, we have been able to create new pathways to try to provide incentives and additional pathways to get new drugs to the market that attack some of these drug-resistant pathogens. i think it is still important that we focus on trying to and newwhole incentives ways to reimburse limited use anti-invectives as a way to create incentives for the development of these products. we're working on one such idea with our colleagues to try to move towards a different reimbursement paradigm for drugs that are the kind you want to put on a shelf and never have to use. moving towards a site licensing model as opposed to a pay-per-use model, which is the traditional way we have paid for drugs. >> thank you, mr. chairman.

i yield back the time i do not have. >> the chair recognizes the gentleman from kentucky. five miniature questions. -- five minutes requested. >> there is a lot going on in this country. a lot of times what is happening in this hearing -- a lot of hearings make television. this one probably does not make prime time. but it is important. bipartisan. it makes a difference in people's lives. dr. collins, you and i had a meeting with a member of the band u2. he was talking about somebody who had childhood cancer. i remember walking away because i had a friend of mine passed away when i was 11 or 12. she would be alive today if she had the same disease -- probably would be alive.

the reason i point out u2, i think they being irish really love this country and america inspires the world. people throughout the world to research. nobody compares to what you're doing at nih. what our health care system, our industry, public-private partnerships, we could use a little good news today. there's a lot of good news. a lot of people in this panel, we really appreciate it. i have a couple of questions. call -- codify many practices in the office accommodation products, but also included regulatory process. how is the fda delivering on these efforts in the area of regulation? >> we have put forward a number of additional guidances as well as staff manual guidances on how we approach combination products

in the agency. historically it has been challenging for the agency. i think we have made a lot of progress owing to provisions that flow out of the -- as well. we set up a counsel to try to educate who has jurisdiction over these products. we will put out a guidance this fall into the summer that is going to make further process reforms that is going to make it more efficient for products that sit on that cost to move into the device realm, which i think is the preferred route for a lot of developers if they can get their. we going to look for ways to find the most efficient route while being mindful of our public health obligations. >> i talked just recently on the telephone, several send a letter concerning drug shortages and supply shortages.

i have had an emergency room physician from my district say they do not have simple things they need. tied toome of it was the natural disaster in puerto rico. i think you handled -- he will need to know, there are people in governments trying to make things work. we have issues we need to address. what happened in puerto rico to get pharmaceuticals moving again, what you have done, but i know there are still issues with drug shortages. i hear from anesthesiologist, just basic things. when you talk about a minute and a half what you explain to me on the phone and what you are doing with your drug task force and what is moving forward? >> the challenges here are structural. it is not one thing. we are doing specifically with sterile injectable drugs. those seem to be chronically in shortage. there are structural problems. reimbursement has been driven down so low. many of these drugs are slightly

above cost of goods. the only way to manufacture is at tremendous scale. we have seen under investment in manufacturing because there are not a lot of margins to reinvest. manufacturing these is not trivial. it is one thing to manufacture a small amount of a drug and have a margin above cost. it quite different when you try to manufacture a sterile injectable drug. things go wrong. when things go wrong, if one facility gets shuttered, it can take down 40% of the market. we think there are things we can do. we could define a category of critical access drugs. concerned ily i'm regulatory touch is only going to exacerbate the problem insofar as it will increase costs. but we are trying to do is look at this holistically and bring in error colleagues -- our colleagues to look at regulatory steps to mitigate shortages or

prevent them with potential changes in the reimbursement structure. we talk a lot about drugs that are priced to high. and there are drugs that are very costly. there are drugs also that probably are priced too low relative to their importance in the cost of manufacturing them. we need to take a look at how we reimburse these old sterile generic, off patent drugs. >> my time has expired and i yield back. >> the chair recognizes the gentleman from new jersey. five minutes requested. question for dr. gottlieb. then i want to go to dr. collins. the action plan which strives to encourage more innovation and competition. i believe such action is critical and necessary.

realize thel better importance of bio similars. years have passed. only three bio similars are marketed despite the fda having approved 11 of them. you noted competition is anemic. the high costware of prescription drugs including biologics continues to be a barrier for many patients. you revealed a recent analysis has found in all fda approved by a similar's were available to american consumers, savings could be realized. could you discuss further fda's analysis and the savings potential bio similars could offer? >> i appreciate the question. we going to be publishing full results. we look to the experience in the european market, where we saw product introductions and extrapolated that to what could have happened in the u.s. market

if the same product have been introduced in the u.s.. we effectively took the competitive landscape from europe, made some corrections to the fact that the dynamic is slightly different than the dynamic in the u.s. market, but look to the price reductions when four bio similars enter the market. when we extrapolated those back to the u.s. market and assumed every bio similar that was approved have launched, we extracted that additional $4.5 billion would have been saved in 2017. the savings are significant. we probably err on the side of underestimating. the savings, when you look at the european experience, are not of the same magnitude. but they are significant. they start to approach the small molecule type of savings want to see 45 introductions. -- four or five product introductions.

>> thank you. a question is, how can congress work with the fda as an implementer to facilitate greater competition in the bio similars? >> we are going to look at some of these more difficult scientific questions. i think they could facilitate more competition like interchangeability. one in particular we are going to be looking hard at is being able to use the european product as a reference standard. a bioituations where similar might be manufactured in the same facility and distributed in the u.s. and europe. our knowledge of that constitutes confidential information. we have to require an entrant to run the study against the u.s. reference even though the european product says the same thing and might be easier to source.

have are opportunities to savings if you could source a reference product globally. i will add that half the cost right now of developing a bio similar -- it is highly variable -- on average, half the cost is the cost of acquiring the branded biologic to run the comparative trial. the savings could be significant. >> let me quickly gets dr. collins. he mentioned nih's currently building the standard resource which will help researchers study begin a collected from participants and connect on a large data set, making this available to a broader range of researchers. obviously it has pros and cons. the major issues are security and privacy. given that nih's asking participants to share details about health and lifestyle, how do you ensure data privacy?

are there protocols to share updates with participants? >> i asked the deputy director, who is all over these issues, to respond to your question. >> thank you for having us here today. with where wed have come. we have over 86,000 participants . as we try to engage a community of diverse participants up to one million or more, we need to gain their trust. privacy is one of our most important priorities. encrypted anda is goes into a secure cloud environment. once we open access to researchers, they will have to abide by it a code of conduct. no data will be allowed to be downloaded. briefly, i want to thank the committee here for giving us strong privacy protections, including certificates of confidentiality which allow us to provide robust protections. >> thank you, thank you mr.

chairman. >> the chair recognizes the gentleman from michigan. the author of the cures legislation. -- i want tothank remind everybody here this is a great committee process that passed 51-0. everyone in the committee in the last congress had a piece of this. staff was terrific. so were the agencies. you helped lead us to where we wanted to go. the senate. we had all the disease groups. we had lots of players. we appreciated that input. based on the hearing today and what we thought was going to happen, it has fulfilled our expectations. we have a lot of time left to play. it is a game changer. way back when, i was one of the waxmanpants with henry for the nih.

four of us were the sponsors of that bill to get it done. it was a very important element that we actually increase the money for the nih knowing we had a terrific steward in dr. collins. i can or member sitting upstairs, mr. pallone, to get my partner and a few others to talk about how much money we wanted to add to that baseline. we got $45 billion increase over a 10 year span. leadership demanded we have worked, we came up with those not once, but twice. comments made earlier and might be more important to have someone from hhs here, but i was alarmed lest week when i read the story that hhs was moneyramming some of the

we had done a dollar for dollar a year by year table to make sure that went for the nih. i do not know dr. collins, if you have knowledge, there is some truth to the published reports that money was being taken away from what we were able to do and put to something else? if it is happening, how do we get back? >> i am not aware of the report your mentioning. there is, i believe, and we should check this, late which in 21st century cures prohibits funds for any other purposes. i think this is nicely designed so those funds are intended to go for your original plan. >> that is good to hear. i want to talk about all of us. i think the privacy protections are important. important, there are

people that are going to have to worry about that data being stolen. as we found from our roundtables, people were -- individuals were more than willing to share what their own iterience was, knowing that was going to protect someone else from the same ailment. it is exciting to hear that 87,000 people have already signed up in a couple weeks. for me, particularly within privacy protections, we would like to set up a caucus. i would encourage members and staff to participate. 25,000 people worked here on the hill. maybe we can get a percentage to come down and figure that out. i know that researchers are going to have access to this information in a year or so. how quickly do you think that

data might be able to be utilized and figure out the for some real important research to be promised? weeks, notuple having chemo for breast cancer was very important. heralded around the country, nicely. how fast do we expect some of this information might be able to be used by the researchers with concrete results? >> it's a great question. we are excited to get the brightest minds around the country accessing the data. 86,000 participants, the data is going to get richer as we add more participants. i would imagine once we get access to researchers we would start to see significant

findings. the data gets richer with more participants, but over the long term as well. we see people who are healthy, stay healthy, how they respond to therapies. >> quickly, how long does it take for someone to sign up to do this? what is the time element to participate? >> the online enrollment process takes about 45 minutes to go through the consent and authorize your health record. we have the in person visit where people donate blood and go through a physical measurement that takes between 45 minutes and now are. over time we will ask participants to do other things digitally and maybe come back for in person visits. >> the chair recognizes the gentlelady from california. >> thank you mr. chairman. thank you doctors, it is an honor to have you here. i cannot help but think that

while most people do not know our names or what we do, they are all counting on you. i think you represent part of the real genius of our country, and i always say nih stands for national institute of hope. you give us even more hope this year with this report. dr. gottlieb, i know that you are aware the legislation that my wonderful colleague congresswoman brooks and i wrote , strengthening public health emergency response, 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 by a defense medical countermeasures to qualify for the prb would dramatically increase the number awarded. the first product was just approved earlier this month. does the fda still have concerns about the number of products that qualify for the prv? can you tell us if there has been on the part of companies to apply for it? briefly -- >> i appreciate the question, and i think we all appreciate the effort of this committee to provide if it -- provide incentives. the first product that qualified for one of these was just approved by the fda. it is potentially a very important product.

a treatment for smallpox. the holy grail was always to have a treatment in the event of smallpox being used as a bio weapon. i do not know the position of my colleagues before me, but i would not argue we have too many medical countermeasures coming into the agency and too many prv's. if anything it is the opposite. we would like to see more development in this realm. >> i appreciate that. i do not know whether dr. -- pancreatic cancer is still a death sentence. agod legislation some years regarding it. can you give us an update of if the needle has actually moved? i know what i did the

legislation, i cannot remember the year, that the needle really has not moved in 40 years. can you give us a quick update on that? and then i would like to go to dr. devaney for a question. >> the ansell is yes -- answer is yes. the needle has begun to take up -- tick up. i think we have seen really good stuff. get a recession, those patients are doing well. it is a minority of those patients, but that group is growing. we had a randomized trial with a significant survival bid for those patients. patients who do not have the mutations, which maybe 10%, some of those are responding to --

>> i think i would like to follow up, because i am not a doctor, so i do not understand what you just said. but i know you can break it down for me if we have a conversation or a meeting. i appreciated. dr. devaney, thank you for your report. one of the places where the program is taking place is my congressional district. it is palo alto. can you give me a quick update on if you know about the veterans that signed up to participate, how your experience working with the palo alto v.a. -- i'm brave proud of it. it is quite an enterprise. i heard from many constituents who are interested -- you are interested in participating. i would also like to know how your advertising to the general public. >> we have -- thank you for that. we have a really great partnership with the v.a..

they have been running the veterans program and they have thousands already enrolled. the pi at palo alto has been a great partner. they had great success getting veterans enrolled into our program, which is then -- it is a really important population for us. the health outcomes are so important. we also have v.a. in boston. we are excited to get more sites launched across the country. >> how are you advertising? >> using a number of different tactics. learning how we engage across a study that is trying to be national. we are different marketing techniques. digital marketing, in person engagement, the health care provider organizations are critical for us. the have direct relationships with their patients. >> thank you chairman, i yield back. >> the chair recognizes the gentleman from ohio. >> thank you mr. chairman.

thank you, panel, for being here. countryportant for this and also the gentleman from michigan who is not here, but i want to thank him for his leadership on 21st century cures and getting us to the point where we are. dr. collins, if i could start. you talked with regards to the brain initiative. , it will lead to increased understanding for brain health and a means of preventing brain disorders such as alzheimer's disease, drug addiction, schizophrenia, and traumatic brain injury. i also see it has been $1.5 billion allocated over the next 10 years for the study going on. at ohiohen i have been state and seen what you are doing with parkinson's and their i wouldut the one area

like this focus is on all timers. -- all timers disease. mer's.hei the cause could rise as high as $1.1 trillion by the year 2050. the sixth leading cause of death in the united states. one in 10 americans over 65 have it. we have about 5 million americans with alzheimer's today. unfortunately, when you start looking down the road over the years, the people by the age of 65, we have talked about 14 million people afflicted, where do see the study going right now with alzheimer's? >> this is an area of intense interest as a major challenge for the world, certainly that is true of the united states. you have quoted the freighting

number of individuals and the cost that is going to apply to our health care system. more than that, the personal tragedies that happen every time a diagnosis appears that affects not only the individual, but their families and caregivers. we are all in on an aggressive strategy to identify pathway to prevention and treatment of a disease. we are grateful to congress because congress has been progressively giving us greater resources to work specifically on alzheimer's. we are doing that in a very, and i think visionary partnership with industry in the accelerating medicines partnership to be sure the basic science we are doing on the brain, trying to tell what the earliest signs of alzheimer's, gets translated quickly into therapeutics. we are learning alzheimer's begins long before the first symptom in terms of cognitive function. if we are going to be successful in delaying or preventing it, we

need to find risk at the earliest possible moment and not wait until they are already in a circumstance of having lost a lot of the neurons. they are hard to get back at that point. i am guardedly optimistic we are on the right path with trials underway right now. we have get virginity to treat people before they have any symptoms. we know they are on a dangerous pathway and can watch and see what happens. we are also learning more about what happens in the immune system, which we had not realized before. inflammation seems to be important in this condition. thethe focus we have had on proteins that build up in the brain are important. but that may not be the whole story. i could give you a long lecture and i will stop because of the timetable, but between nih basic and clinical science working with industry and with close collaborations with the advocates, we are pushing this as fast as we possibly can.

wei would add if i may, worked closely with dr. collins and we recently issued a new guidance document updating guidance on critical -- clinical trial endpoints. historically there was a position you had to show improvement on functional status and cognition to win approval or show you had slowed the decline in functional status. that is challenging because by the time functional status declines, you have probably had a significant decline in cognition. now that we are able to identify patients earlier with more sensitive tools to detect changes, also prespecified patients at high risk, what we want to do is intervene earlier when there are slight changes in cognition and try to arrest that process. for the agency has said is in the right circumstances, if you can show improvement, that could qualify for approval for a drug that would slow the progression

of alzheimer's in the right prespecified population. we are trying to work closely with nih. >> that is really important. again, thank you very much. i yield back. i ask forirman, could the next member to come and sit as a cosponsor. >> thank you so much. i just thought mr. greene wanted a break. [laughter]

that was very nice of him to recognize me. i want to add my thanks to all four of our witnesses for their extraordinary help, not just in hatching this bill, but also implement. it warms our hearts to see how much progress we have made in such a short time. thinko encourages us to how much more we can make. eache a few questions for of our agencies that are here today. i will start with the fda. dr. gottlieb, the biomedical community has really made great strides in developing clinical therapies from unlocking the potential of stem cells to creating therapies tailored to the unique genetic fabric of a patient. one of the things we have been concerned about for a long time is that overseeing and regulating these therapies

requires an experienced, well-trained public health workforce. cures, we had a provision for new hiring authorities. i'm wondering if you can talk to curesut how the hr authority has helped the agency. >> we are in the process of implementing that. i think it is going to be transformative for the agency in terms of our ability to recruit people with specialized skills. have identified 38 occupations that meet requirements for the alternative pay system. hires. the first two both deputy senate directors. to briefly pick up on your point, we are talking about new treatments like gene therapy and cell-based regenerative medicine. the clinical characteristics of the products relate very closely to the product features.

the product features change quickly. the underlying technology of the product itself -- these are not all pills anymore. used to be as you had expertise in a scientific clinical field, you could adjudicate all the different kinds of drugs. now it requires expertise in the products themselves. the regenerative medicine platforms. there is a lot of novel technology you have to have engineering skills. that is where this is going to be helpful. trying to help find people with discrete skills. >> you cannot hire someone straight out of grad school. these are people with specialized areas of knowledge. >> that is right. they are often employed in the private sector where their highly paid in skilled. we are competing against others for a small pool of talent. reporthe fda's june 2018 , you talked about this work of

-- patchwork of hiring authorities. are you starting to address that? >> we have had historical challenges with the overall hiring framework at fda going back 15 years or longer. some of the same challenges him grappling with now i saw commissioners grappling with in 2003 and 2007. we are addressing it very directly. we are trying to do a top-down wholesale change of the hiring process to make it more efficient. we started a pilot, which dramatically streamlined the boarding process, the hiring process. we focus that. what we have decided to do is take the structure of that pilot and apply it to the whole hiring process. not just bifurcate one set of hiring -- >> i hope to -- i hate to cut you off but i cannot ignore dr. collins. i have about 10 minutes of questions for you. you are going to have one minute

to answer. let me just ask, what kind of research is going on over in your agency on alternatives to opioids for pain management? the opioid crisis is one of the big issues that is really facing this committee last year or two. >> thank you for raising this. this is incredibly important. we are grateful to congress that additional $5 million was put into our budget to work on the opioid crisis. a significant fraction is being devoted to the thing you are raising. nonaddictiveevelop pain medicines for 25 million americans who suffer from chronic pain everyday, for whom opioids is not the answer. it actually is harmful in many instances. this goes all the way from basic science to discovering new targets to working with an industry to help actually free up ideas that have been on the slow boat and can be sped up working in a collaborative way

and setting up a clinical trials network so we can quickly test and see whether new therapeutics that are nonaddictive work. and in what setting to the work? all back pain may not be the same as the pain you get from neuralgia. we need to have the kind of clinical trial work that does not exist. >> this might be a good subject for a separate hearing. thank you. dr. gottlieb, had questions about rules pending about shock therapy. we do not have time to have you answer those questions. i'm going to be contacting your agency to find out why those rules have not been approved, pending since 2016. i promised my constituents i would ask about this. >> i would be happy to discuss. >> thank you, mr. chairman. click thank you, mr. chairman. i appreciate you holding this

hearing. i want to thank all the witnesses and the men and women you are speaking on behalf of who do amazing work in the area of health care in america. the legislation was passed, medical innovation, reforms to america's mental health services , today was a really important they and important hearing to continue our work to legislate and evaluate and evaluate inseam what else we need to do going forward. what is working, what is not, what do we need to change. i have a lot of roundtable discussions in hospital for rooms and community centers with people of my district who are very pleased with the investments we have made in medical research and in turn the work your scientists are doing to return that money and research into reality and medicine and cures that goes from one end of this table to the other.

we appreciate the work you are doing. i have a friend who is an ms advocate and said cures is a great step toward finding a cure. about are pretty excited precision medicine, the cures that are out there waiting to be found in the work you are doing. suggestedeb, you there is a correlation between lowering product costs and promoting closer trial designs -- clinical trial designs. where are all concerned about the cost of medicines and you know clinical research is not always as efficient as it could be. biomarkers help to make them more efficient. >> i appreciate the question. well clinical trials do not translate to how products are priced, we know clinical trial

the risingctor into costs of the overall development and the competitive nature of this landscape. when i talk about today is the complexity of conducting clinical trials especially in areas of unmet needs when there is already available therapy is to market delaying second and third class drugs. specialty drugs are enjoying monopolies for longer periods of time. if you believe that subsequent cup titian shows this -- competition shows this, prices are not coming down. i think there are things we can do to try to facilitate more efficient routes to market second class and third class drugs while increasing effectiveness, not sacrificing it. those are development reforms we're focused on.

>> and trials for rare diseases can be difficult to conduct. how do you spur additional innovation and competition for rare diseases? this gets to changes in clinical trial designs. one thing is in rare diseases looking at natural history models, you do not have to enroll placebo trials. if there is already available therapy, patients are not going to want to go onto a placebo. they're going to want to use an active drug. this is a place we could benefit from more disease models. there is a request in the president's budget for $20 million which would help facilitate that. i know we have support for some of those resources. we are grateful for that. i think there are things we can do working together. >> well then, well played.

collins, i saw the nih recently announced an effort called respond, research on prostate cancer in men of african ancestor -- ancestry. that study funded by 21st century cures cancer moonshot initiative will investigate the environmental and genetic factors of prostate cancer in african-american men. can you tell us more about this study and how it will combine molecular approaches and environmental science? >> thank you for the question. this is alluding to a trial announced that will be the largest trial on this topic in our history. it's going to take 10,000 trial analyze the genomes that contribute to this disparity. african-american men are more likely to be day -- diagnosed with prostate cancer and die of it. understanding this is a crucial

question. >> anything else? i have to point again to the study as a platform we will be able to utilize going forward for answering many questions of that sort. we have this goal and we aim to reach it. all of us are going to come from judicially underrepresented groups, racial and ethnic groups. if you want to examine health disparities are ghost dance is, you are going to have a million participants who are highly motivated on whom we have collected a great deal of data. we are going to learn a lot about this health disparity about all diseases once we have that platform. >> we appreciate your involvement with our committee. we look forward to doing more together. mr. chairman, i yield. >> the chair recognizes the gentlelady from florida. >> welcome to all of our

witnesses today. dr. collins, i share your enthusiasm over the future of the cancer moonshot and precision medicine. part of my enthusiasm stems from the fact that back home i represent the moffitt cancer center community, the only designated conference -- comprehensive cancer center in florida. i love meeting routinely with the young researchers who now feel like we have given them a new commitment where they were very concerned in the past on the future of nih funding as it competes with all the other needs. now they feel like 21st century cures, these new investments for the cancer moonshot, gives them hope for their research in the care they are providing. the moffitt cancer center has been a national leader in building a large data set, tissue samples, and working with

other institutions, especially for those in underrepresented communities. you have given us a bit of an outline on the importance of protecting privacy for people who participate. can you tell us about the protocol going forward for researchers? will they be required to share their research? will the research results be available to all for other researchers to build upon? >> great question. would have been thinking a lot about your point about young investigators. the diversity of researchers also, making sure the platform is open to everyone. and feel responsible for ensuring the things we learn on the data that is provided by our participants is returned to the scientific community and our participants. we are developing policies around ensuring researchers who

required toata follow a code of conduct and share results within a specific amount of time. we are finalizing those policies. and policies around returning results back to participants. >> in the past it has been kind of siloed. certain researchers kept it, held close. what does the future hold? >> i appreciate the question. and i appreciate that 21st century cures give me authority i did not have before to require data access for studies we support. i could cajole and the past. -- in the past. but i did not have the clout to say this is a requirement. if you are getting a grant, you are required to submit your data. you gave me that in that has been a useful tool. we are very engaged in the

process of trying to establish what that needs to look like for a wide variety of studies. in particular for genomics. we have a very well worked out genomic sharing policy. we are in the process of working that out for imaging and electronic information. this is a very high priority. we hate those silos, too. >> we look forward to that. , your predecessor was well-known for his research on hpv. part of the cancer moonshot is not just future research. it is what can be done to prevent cancers, detect them. over the past few years, a lot of the -- there has been new focus on making sure 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 would be parade in the street. here is a vaccine to prevent a whole host of cancers. are you committed to continuing those initiatives? >> very much. the doctor continues as my deputy director and has been invaluable. i appreciate doug. his work has been wonderful. we have a robust portfolio in this area. one of the most important studies is a trial of one versus two doses of the vaccine. the recommended is three doses. that's a big issue in terms of -- and limitation. if one does works well, that could be a game changer globally. >> it is a good reminder at back-to-school time. this is important for boys and

girls, middle schoolers, to get the hpv vaccine to prevent cancer in the future. >> right. thatber of vaccines prevent hepatitis b and the dissemination of those things is a real challenge for us. >> i yield back. >> the chair recognizes the gentleman from new jersey. >> thank you, mr. chairman. good morning to your both and thank you for your public service. , the drug act has helped to create a market-based system to encourage the development of new medicines for people living with rare diseases. i am the chair of the rare disease caucus in the house. innovators are granted seven your monopolies in specific disease areas.

the act was established in 1984, medicines have been brought to market for only some rare diseases. there are many where there are no medicines at all. these, there has been zero second-generation newly innovative medicines brought to market for patients. lysosome storage disorders, for example. cost on averages nearly $500,000 per patient per year. with the exception of catcher disease, not a single second-generation therapy has been approved for any of these diseases since these first-generation approvals. most of those date back at least a decade. gottlieb, haver. we created perpetual monopolies in rare diseases, especially the

areas i have discussed? what are the barriers to moving innovation forward to spur competition and innovation, and to bring newer medicines to patients with rare diseases as rapidly as possible? >> thank you for the question. i think this cuts to the other issues we discussed here. i do believe there are settings where it is harder to bring second to market, composition -- competition. i would argue this is one of them. when you have drugs that target smallrative diseases, populations, it is hard to run clinical trials once one therapy is available. typically subsequent drugs will have to be studied on top of available therapy and you will have to show improved efficacy with combination there be as opposed to monotherapy. it is hard to run head-to-head

comparative studies when effective there a fee is available. people do not want to go -- forgo an effective treatment. there are a lot of things we can do. earlier this year we published in conjunction with our counterparts in europe a master protocol for how you can study multiple drugs within the same clinical trial structure. we talked about natural history models to model the behavior of patients who go untreated. we know how these diseases progress. we know how they affect patients. we should have robust natural history models so we do not have to enroll patients and see what trials rely as much on placebos. there are a whole host of reforms we can pursue to facilitate second to market innovation, which is critical. >> is there something more we should be doing statutorily in

the legislative branch? >> i would be happy to have that discussion with you. there are a lot of things we can do, that we are trying to do and will do. hadthing i would just -- i this discussion with the chairman about the natural history models. we allocated $6 million to develop six natural history models. unnatural history model for the sickle cell disease. 20 natural funds for history models. super orphaned diseases. >> i look forward to working with you. i have been the chair of the caucus for some time. is completely bipartisan in nature and we look forward to

continuing the discussion. in the audience, i noticed john crawley who has been involved in the rare disease space. they are residents of new jersey. there is no one fighting for progress more. i yield back the balance of my time. >> the chair recognizes the gentleman from virginia. five minutes for questions, please. >> the letter only has six requests. some proposed in an email.

fda continuesthe to tell committee staff the letter is in clearance. could you help expedite the clearance so the committee has the response before the end of july? it is in clearance. transparency,ove i did make a lot of the information if not most of it, available publicly. a forum we had to try to work with internet stakeholders to read weopioid sales to can make them available. ofecognize the importance the informal response. i am on it. >> you have so much to do. worknk you for the good you are doing everything are a lot of areas we have been

working on together to read what further improvements will fda make to the program to ensure the process is efficient? theicularly now that federal right to trial legislation has passed? we have the senate version. give me an update. >> we have a process. the work group is looking at what steps any we should take. to be a pathway that sits alongside our program. an outside group read an expert group to take a top-down look. make some recommendations to the agency how we can improve it. i'm going to make that public soon. we think there are additional processes that we can take to make it easier for people who are not as sophisticated.

not dones who have this before. we are working with friends of cancer research to create a platform to have one site of injury to get information about what programs exist. exists, thee difficulty, a lot of times difficulty of sponsors making things available. -- you barely have clinicaloducts for the trials. toneed to look for ways provide incentives about this. might be able to have supply available, too. activeve been an proponent for many years. if people are facing death, they

will take that pass and whatever they need. let us know what you need to i have a partial update, i have found on the internet. i understand you had a breakthrough. i have friends who have benefited with those. >> they are important and often tragic diseases that result in neurodegeneration with there has .een encouraging developments very much in the process of being evaluated. huntington's disease, i will reflect a bit on. my only between was involved in the discovery. tohas been very gratifying see, in the course of the last

year or two. very encouraging information about using a genetic approach. clinical trial, which we are waiting for, the data to be rolled out. it is sounding encouraging. a molecular therapy injected into spinal fluid may very well be able to provide benefits. we have waited a long time for that kind of thing to happen. go beforeong way to we can say we really have the answers, but it is different than saying we have nothing. >> the gentleman before him. >> thank you very much. i appreciate the opportunity to discuss with witnesses the progress we have made. pass lawsday do we where we can say, we think we

did something good. i want to thank the implementers for doing good work. withrst question has to do the diversity and research of subjects. thank you for testifying today. 85as excited to read about thousand individuals have started the enrollment process. 70-75% are from communities that have been historically underrepresented. from groupsifically not included. diversity in medical research has long been an issue. i believe one way to decrease disparities is to ensure we are including individuals of all races and at the city's. is, what is nih doing to ensure all of us, the

research recruits a diverse participants? >> we track that, week by week. i personally am asking for that to make -- to see how the enrollment is going. this is unprecedented. >> thank you for the question. our strongests priority. ultimately, a lot of the data using is not reflective of the diversity. what we are learning is not applicable. we have a number of ways we are attempting to reach out to communities and go into it. we are going to try things. one of the things i would like to highlight is working with community partners. we have a number of community engagement partners helping us build trusting relationships.

her chief officer. been working with those groups in a robust way. we have about 30 plus partners that are national helping us to build awareness. >> you are talking about groups that are more local. whether it is an immigrant population, hispanic. with local groups that have relationships of those communities? >> that is right. >> when we did the launch, seven different faces, i was in new york. with an african-american community that was wrapped up about the opportunity to be included. focus on inclusion. you have given us some additional tools. be ahis is turning out to shift.

>> this opportunity for us to remind ourselves diversity in research, subjects in particular communities, they ave different reactions, etc.. correct? >> absolutely. one should not assume when you see a health disparity, that is eadily understandable. usually these are a combination of environmental exposures, stress levels. one should not over interpret that part. we are all awfully similar at the dna level. you have these comprehensive studies. figure out how to intervene. that is more guesswork.

>> we just heard from the doctor a few minutes ago about this extent he, starting with african-american prostate cancer. we don't understand why men with prostate cancer who are black have a higher likelihood of dying and aggressive disease. >> what are some of the hallenges building this? do we have some findings? things we are hoping to ackle? maybe you learn? >> we would love to follow up and talk about this more. we have community advocacy groups. participant advisory groups. they are helping us to understand what is working and what is not working. even things like parking. it is too hard for me to make the appointment time. we have been learning a lot.

i will be happy to share as i earn more. >> as an engineer, adjusting is important. you are doing that? thank you. > thank you, mr. chairman. >> she is my cochair. also to fred upton. their tireless work. one more shout out to them. my youngest daughter, when she was 25 years old, was diagnosed with hodgkin's lymphoma. daughte was 25 years old, was diagnosed with hodgkin's lymphoma. years,elopments over the

have been phenomenal. i want to thank everyone. theere happy to support cancer and shot. 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? >> it is tough. it is a tough law. we talked about the response. the rare tumorut initiative. rare cancers are difficult to study. it is a good place to study those things. we can have patients get care. the paradigm works. the moonshot is trying to build on that. ,n the area of the disease

different mutations activate it, a driver protein, they generally present with childhood tumors. recent trial we just had presented. slide, one of our scientists. a trial. a deficiency. my executive summary was, the kids feel better and the drugs seems safe. a couple said, my child was on this. this is after treatment. you can see, the windpipe is not being compressed. of going totress school with a region like this. this is a mutation, a cancer gene. this is a great thing for the

nci to take on. says -- can you they are hoping to transform infrastructure? the new funding from congress has made it possible to increase the success rates for new nci, we setthin the aside enough funds to increase that by 25%. we are trying to lengthen the purity of the award. that will allow scientists to focus on their science. we are testing out a grant. we are thinking about the training opportunities we are providing. both in the postdoctoral and faculty setting. they data, something we are

training in. >> thanks to the cure. the next generation researcher initiative. we have put in place a better chance for investigators coming with their first grant application. having sustainable support. will fund thewe largest number of investigators. we have made this shift and you help us with that. >> i had a chance to meet with early career scientists. i was very impressed with the excitement and passion they have. cancer patient, the survivor, it means a lot to me. thank you for being here.

i yield back. >> the gentleman yield back. >> thank you for coming today. reminded of the tremendous responsibility you have heading the organizations you do. i want to thank you for your work to implement the cures. which, as you know, is a point of pride for this committee. the commissioner, i wanted to ask. i know there have been some discussions about the action plan. i commend you for that. i have introduced in the last few days something we are calling a competition act. to try to get to some of these delay agreements. in a similarating

space, in the same way we have ftc touthority to the police that conduct. drugs and generics. been consequential for sure. the authority is there. whether they are fair and appropriate. vis-a-vis the consumer or not. there are tremendous savings to be had there. is, the brand names of the drugs produced 75% of the still cost out there. i think that includes the .iologics file similar distinctions as well. can you speak to whether you think it is a good idea to have this authority?

i think i understand from the put forward, you look for ways to cooperate with an arecy to make sure we getting these drugs at the price point they should be at. -- i haven'ton looked specifically but i will. of myoutside the scope authorities. tonging cases related delaying tactics that might involve what we can help provide information about. areher the practices anti-competitive. toerally speaking, we like approve safe and effective bile similars. we like to see patients benefiting. culturally, we like to see drugs

marketed. most have not been marketed for various reasons. of moleculesber are approved but the drugs are never marketed. i think we need to keep a close eye on that. competition may be declining. >> we will try to get this into the toolkit. the government has to make sure we are protecting consumers. let's switch gears. to you, dr. collins. i know you got a question. alzheimer's research. some of the clinical trials associated with it. there was an article that pointed specifically to the challenge resented by trying to find people to participate.

there are a lot of trials underway. forh would suggest the need 25,000 participants. some of them could be breakthrough. i gather this is a real problem, a challenge trying to find enough for disciplines. can you speak to what you know about that? >> that was based on comments made by murray bernard. this is a challenge. we are at the point you're trying to recruit individuals who do not have cognitive decline. some of them

who are motivated. we are arguing, that is the best time to intervene. one of the dreams i have we have been talking about, one million participants who are preapproved. whether it is diabetes, hypertension. at the moment, where we do not have that platform, we have we're pulling out all the stops. and again e. the congress having made alzheimer's such a high priority for us, we have resources in order to do that kind of recruitmentment but it is not simple because it's a different model than what people are used to and you get approached about a clinical trial when you already have the diagnosis. here we're approaching people who don't have that diagnosis trying to figure out how to prevent it. that, trying to fige out how to prevent it. >> the gentleman yield back. five minutes for questions, please.

is a pleasure. dr.s at an event with sharp. the work that he has done over expresss, can't even how much of an impact he has had. you've said high risk. hugh is high risk for alzheimer's? or 90.ody at 85 if your mom hasn't, your debt has it? -- dad has it? maybe if one of your family members has it, you are high risk. >> there are three ways we are identifying such folks. you have a strong family history. those families. if you are in one of those circumstances, we can track the

gene and figure out who has it. another way, genetic risk factor. if you have two copies of that, fold.s up 15 those people are very high risk. the third way, a pet scan. it starts depositing probably 20 years before the first cognitive decline symptoms. if people are worried about this, we made the able to say, you ought to get into this. >> somebody out there is watching c-span. i wonder if i am high risk. testsrstand the technical and things. people need to say, my mom had it so i am high risk. >> that is one of them. try tore other ways, to be more precise. if people are wondering, should i take part? we heard how difficult it has

been. the place to go, clinical trials. trials.sts the you can find out who is doing the trial and what are the enrollment criteria. all of this we do very closely. >> my wife is an anesthesiologist. she is practicing medicine. she did not know you were here. she said, can you check into the fentanyl shortage? me -- texted me. she is practicing medicine. we have demonized it. it is common. apparently there is going to be a lot -- long-term backorder. you don't need to answer the question. what you are doing on drug shortages, real everyday -- you know this -- clinical

applications. uses everyts she day. fentanyl is a new one. she just texted me that one. thank you for your work on that. further comments, i have one other question for you. these are structural problems. beuarantee it is going to like this. it impacts patient care. we are working hard on this. >> personalized medicine. cancer, ies such as know your staff has told you i might ask about this.

they have not been updated and's 1988. have released this. we have had a discussion draft. we appreciate your input. to laboratories, patient groups, others. i know you are familiar with the issue. as well as the last hearing. you recently gave us a narrative on what the fda provides us. reformto get diagnostic done. we are waiting for the red line from fda. i want to know if you have insight. >> we have completed most of our work. i would be happy to brief you on

it. i think we are very close. you have been very helpful and i am appreciative. we are hopeful to get this accomplished. the chair recognizes the gentlelady. >> thank you all for your service to our country. all the incredible innovations. you could be doing different things in our country, and yet you are here, working on behalf of citizens. like to ask, in your testimony you mentioned there are provisions that provide direction from congress. i am particularly interested in childhood disease and cancer is. having heard from constituents and lost a child to cancer

are fighting the disease, we know and i age is focused on improvements for children. an area lacking in the past. can you elaborate on the implementation of the recently passed childhood cancer star at? innovation are we focused on? >> i.s. dr. sharp. >> it is important to say two things. it is true, we have made tremendous progress. we are still having too many children died. kids,e are able to cure we leave them with toxicity. that is one of the principal issues. therapies for kids with cancer. the burden of survivorship some of the patients incur.

we are interested in this topic. this was a personal research interest of mine. i know firsthand the pain many of the patients go through. i think we have a focus on new research efforts. getting new voices to make sure we are advised. thank you for your leadership. >> thank you very much. >> thank you very much. ,s my colleague talked about one of the things that has come up in various discussions is the importance of platform technology, the use of platform technology when it comes to innovation with vaccine. can you talk about that to some

extent? because there has been frustration on this committee vaccines versus platform technology. appreciate the question very much, and we have a proposal in the president's budget for additional funding to try to support alternative manufacturing, and we think this is the direction we want to hidden. when you have closed manufacturing platforms, particularly with recovery technologies, we could sets that youve basically plug into the platform and allow continuous manufacturing of the vaccine. if you want to modify the vaccine, you can close in another cassette that codes for a different permutation of the same that same. we think this is really the solution or a solution for influenza -- seasonal flu -- to be able to scale up vaccination more quickly and put his vaccinations closer to flu

seasons and you can guess the stream better. in an ideal circumstance, we would not have a mothballed vaccine that degrades over time if not stored correctly and takes up space but platforms that allow quick vaccination should we need it. >> the idea of having to build rings in alex -- build things in eggs is so much yesterday's technology. flu vaccineth the having been so ineffective, turns out the virus had mutated in the process of being grown in not to beo turned out a particularly effective vaccine. these new platforms allow you to build a vaccine in a much more rapid and much more directed fashion.

once you have the platform going, you can very quickly adapt it to many different pathogens, and that is something we are working on now to develop this universal influenza vaccine which is a very high priority in congress that has given us additional funds so we would not have to have the yearly effort to try to guess right. you would have a vaccine that works against virtually all strains and would also be nexttive against that 1918-style pandemic, which we are all worried about. >> thank you. we did include a pan flu differentiation in the legislation. back. gentlelady yields the chair recognizes the gentleman from florida. , mr. chair. i appreciate it. i thank the panel for their testimony today as well. the first question i wanted to ask is -- we talked about alzheimer's disease, of course. what about parkinson's? you know, how do you

know if you are a candidate for parkinson's disease to produce a paid in these clinical trials? there are no symptoms at early stages. if you could answer that question, i would appreciate it. >> there are parallels here that are notable. there are genetic risk factors for parkinson's disease, which is interesting because when i was in training and asked my professor if there were any diseases that do not have genetic contributions, he said everybody knows parkinson's is always totally random and sporadic, well, he was very wrong. we are beginning to, therefore, be able to identify people at high risk and invite them to be able to take part in prevention trial. another thing that has happened in parkinson's disease in the last six months is the four -- the formations of a partnership within the last six months.

fda is a critical partner in this as well, and really figuring out how we could learn from a large amount of data out there that had not been brought together, what are the next generation of drug targets for parkinson's disease and how we accelerate the process of getting there. we had treatments that have been around a long time, but we do not have things that prevent progression or treat the symptoms. we believe we can do better at that. i should also say the brain initiative, which is this very bold effort is learning things about the wiring diagram of the brain that will be very relevant to some of the things being done for parkinson's disease with direct brain stimulation where you actually put an electrode into the brain to try to take care of some of the motor problems. what we do right now is kind of clunky. diagram,rn the wiring we can be much more precise and effective about that. >> thank you very much. anyone else want to add something with regard to that? ok, thank you.

308aottlieb, section clarifies fda has the authority to grant emergency use authorization for animal drugs allowing approval of gmo zikaitoes for florida's problem. would you provide an update on the implementation of section 38? >> i can get back to you with a more detailed update on that, congressman, but i will tell you this been discussion about the nexus of authority with epa for some of these products, but we did provide guidance earlier this year, i believe, that addressed some of these issues. i can get back to you with more specifics on where that stands. >> i appreciate that very much. as a longtime champion and supporter of providing a deeper level of patient engagement in

the therapy development processes, i'm pleased with the progress the agency has made under your leadership. congratulations and we appreciate all you do. including the fda's moving ahead to implement the patient-focused impact assessment act provision of cures requiring fda to disclose how patient engagement data informed a review of any approved products. inre is the agency presently implementing this provision, particularly efforts to standardize the inclusion for such information in the record where it isdrugs excess and understandable. issued one of four guidances we intend to relate to the patient-focused drug development, and we have standardized a format for the presentation of patient-related

.nformation in clinical trials when a clinical trial is submitted to us, there's a very explicit section for patient-focused information. on the medical device side of our house, we've done similar things. we are seeing a very high rate of the use of patient-focused pro'sation and p aro -- in the development of medical devices as well. patientjust developed a affairs office inside the

efforts to provide a focus of access for patient crews, but also a focus of policymaking with its cross-agency policy making around these issues. >> thank you very much. i yield back, mr. chairman. question on yields back. chair calls on the -- >> the gentleman yields back. sure calls on the general and from georgia. >> a feeling this is some of the best legislation we have passed in congress in quite a while. dr. collins, i will start with you. particularlysk about one of the initiatives of 21st century cures, and that was to really review the regulations and policies respect to research and laboratory animals. as i understand it, you are working with usda and the fda to try to complete a review of that, and i just wanted to ask if you could tell us the current status of that review and when you anticipate the completion of that review. >> i appreciate the question. we are very seriously engaged in this.

in terms of if the oversight we apply to animal experimentation is sufficient or if it has areas that are overly bureaucratic has been a concern. we are deeply concerned about maintaining ethical as possibilities in terms of how we take care of animals subjected to various experimental approaches from which we learn a great deal that has led to many medical advances. we got 19,000 responses to that request, and they are currently being sort at the moment. we would expect, therefore, to half, based upon those draft sets of recommendations about animal care and use some time in september. we will then need to ask responses to that. we would hope to have a final earlyn by december or 2019. >> great. great. any opportunities you have identified thus far that may help you? there are concerns some of the requirements be 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 those things in place when they submit an application because that can add a lot of time and effort. our concern is if we're going to actually 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 and we are seeing .hose in those 19,000 responses we're trying to come down and what we think is a fair and balanced approach. >> let me switch gears and talk it -- and talk about something that is important to me. i see great opportunities for us in health care with genomics, but i'm concerned. we get all this data -- we are struggling already with our electronic health records. how are we going to handle this? i'm going to ask you that another like to ask dr. gottlieb as well. >> i will say a word.

in looking invested at this in the fashion that is cutting edge as far as dealing with very large data sets. how it can be applied to unprecedented data sets to glean the maximum amount of information out there while at the same time maintaining the confidentiality and security system that participants are going to expect. >> one of the datatypes that i think will be the most important is health workers. we have direct partnerships with ,any health care organizations but we are also working on other to work on making the data transmission much more seamless across provider lines

whennto the program authorized. >> this is an important development in drug data as well. >> i was just taking a step up the continuing to try to make effective use at the data. this is where some of the issues we published earlier comes in. we have the ability with seamless clinical trial design to effectively bias in moment in the trials for some of the genomic information and predictive information that is likely to predict who is more likely to benefit from a treatment and who is less likely to experience a side effect. we think if we can use this information in that way to structure trials and enrollment, we could end up with much more information about who is likely to benefit from a drug and more rightto make sure the drug gets to the right patient at the right time.

>> i can sense the excitement in your voice, and is exciting for me as a health care professional as well. i'm looking to the future of this, and i just think, wow, what we've got to look forward to. thank you and i yield. >> chair thanks the gentleman. the gentleman yields back. numbers'bjection, all opening statements will be made part of the record. the chair asks if the gentleman from texas has any consent requests. were you going to ask additional information be forwarded to the committee about alzheimer's? >> if you could, just send us to the committee. >> happy to do that. >> thank you. i forgot what i was going to ask. [laughter] with like tochair make the observation, dr.

collins, you started this hearing with the remark about the immunotherapy and some of the dramatic things that have occurred, and i just -- it theated our work, but former president of the united states in july 2015 with the information that he had metastatic melanoma to the brain and liver, and remembering my time in medical school, my "we willhought was, not have this individual with us by labor day," but it has really yeardramatic to see him a later delivery speech at the democratic convention. a year and a half, he was present at the inauguration in january 2017. i do not know what his clinical status is now, but it was truly dramatic, and 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. again, i felt obligated to make mention of that milestone. seeing that there are no further members wishing to ask questions, i do want to thank our witnesses for being here today. pursuant to committee rules, i remind members they have 10 business days to submit additional questions for the record. i ask witnesses to submit their responses in receipt of those questions. without objection, subcommittee is adjourned. [captions copyright national cable satellite corp. 2018] [captioning performed by the national captioning institute, which is responsible for its caption content and accuracy. visit ncicap.org]

announcer: one of the witnesses at this hearing was stephanie devaney, deputy director of the all of us research program. she will be a guest on our friday morning washington journal to talk about precision medicine. you can call in with your questions and comments at 8:45 a.m. eastern here on c-span.

>> hey, hey, oh, oh -- >> one of the most qualified nominees ever picked for the supreme court. and he's contributed a great deal to his community and the legal profession besides being an outstanding judge on the d.c. circuit court of appeals. >> judge kavanaugh has a special obligation to make his views on this topic clear given the president's litmus test. that he would only appoint judges who would overturn roe. on that obligation, judge kavanaugh fails spectacularly. >> i look forward to watching judge kavanaugh's confirmation hearing and after conducting a thorough and objective review of his nomination, i'm confident that judge kavanaugh will be an excellent addition to our nation's highest court. >> watch day one of the senate confirmation hearing for

supreme court nominee brett kavanaugh. live, tuesday, september 4, on c-span3. tch any time on cspan.org or listen for free on the c-span radio app. >> the senate foreign relations committee considered several nominees to be ambassadors to morocco. bangladesh and uzbekistan. senator bob corker chairs the committee. >> good morning. this hearing of the senate foreign relations committee will come to order. i want to thank senator merkley for helping to convene this hearing today and i'm grateful for our continued partnership on so many issues, senator. and i would also like to thank the senior senator from indiana, senator donnelly, as well as senator peters of michigan. the purpose of today's hearing is of course to