>> the director testifies on the cancer initiative and other innovations that were implemented under the 21st century. this took place before the house subcommittee for two hours and 15 minutes. >> if we could take our seats.

we will get five minutes for the opening statement this morning we would like to welcome our witnesses we are here to conduct oversight and receive updates on the mentation of one of the most substantial legislative accomplishments in the states biomedical innovation in the 21st century cures act. it first past the house and the senate it passed with wide partisan support. the relevant authorized -- authorizing committee to make sure that the agency is implementing the law in accordance with legislative intent. >> i think our witnesses for

their willingness to testify on this topic. 20 % provides hope for those who need it the most, individuals and families suffering from life altering and often life-threatening illnesses whether it is cancer, a rare disease, or alzheimer's. there are conditions costly to americans of all ages and their families. sadly, we know too well the toll that these diseases play on our friends and the community. one of the most impactful positions in the 21st century, they created the nih account innovation treasury, this goes to the precision medicine initiative, nero technologies initiative, cancer research, and regenerative medicine. it continues to decelerate -- it

continues to accelerate as we now have treatment for diseases like hepatitis c which was once in a manageable -- unimaginable. cures included a provision to establish a manageable neurological precision -- provision. there was no requirement or authorization to provide surveillance of neurological disease. this changed, specifically, the law that requires the secretary of the department of health and human services to create a system by expanding surveillance of infrastructure and activities including data collection to determine privilege risk factors and diagnostic and progression markers. preliminary results for the ongoing multiple sclerosis society shows that there are nearly 1 million americans with ms. more than twice the

previously recorded number. including cures provided for a better information to further our understanding of and eventually cure these diseases. we expect to see great progress with this policy that was once a stand-alone bill introduced previously in congress. additionally it allows them to offer patients personalized treatment with the full potential of precision medicine which will require efforts to collect health data in addition to research done by the nations task research investigators. the law codifies the provision medicine benefited -- precision medicine initiative while ensuring confidentiality of the patient's information. the research program is a major piece of the precision medicine initiative and has already engaged over 1 million volunteers so they should be graduated for that -- congratulated for that.

the traditional clinical trial method has been successful, they are not always timely or applicable to new types of drugs. the cure requires the food and drug administration to evaluate the issue of guidance for the purpose of incorporating complex and adaptive and other novel trial devices. the promising results of the effort including the cure will provide americans suffering from cancer and other diseases with the opportunity to undergo successful treatment and in some cases, be cured. my thanks to the doctors for giving us the update of the implantation regarding the law. >> thank you mixture chairman -- mister chairman, we are delighted you are here. a few things i would want to discuss with you all, doctor

collins, all of us, the research project, making certain that we protect the data of this in-depth program. and that privacy with data is respected moving forward with this. following up on this act, making certain that the implementation is going well, we welcome you both and look forward to the hearing.>> thank you. the chair recognizes the ranking member of the subcommittee for opening statements.>> mister chairman thank you for holding the hearing for them mentation of this act. i would like to think our witnesses for being here today especially doctor collins and the doctor from the fda. thank you for being here. this will marketeer anniversary

of the act being signed into law by the president and his last public signing ceremony. it's too advanced discovery and development of new treatments, cures through research and improved drug through the process. it dedicated $6.3 billion in new investments. the brain initiative, the precision medicine initiative and the national institute of health and provided the advance of the agency's mission to a movement policies -- to implement policies. i hope that these investments are being put toward finding a cure for our nation's greatest medical priorities. the nih was provided $1.8 billion in new funding. the fda was provided $500 million over six years to

improve the process and expedite patient access to drugs without compromising standards and safety and effectiveness. much needed funding, there are aspects of this package with much-needed support. advancing the use of modern and clinical designs through investing in the next generation of medical research. while some of these provisions -- provisions are technical and research they do not have to be abstract patients and families deserve that elected officials will help meet their needs. this is an opportunity to hear from heads of the fda regarding these provisions.

>> i look forward to hearing from our witnesses about the upcoming mentation. i yield my time to my colleague .>> thank you so much mister chairman for yielding. i would like to add, my thanks to everyone here, in particular doctor collins and the staff. especially my friend fred upton who really worked with us every day. a work product of the entire committed this the committee it shows the efforts in the community and what we can do to tackle this serious problem. mister chairman i would like to thank you for having this hearing. it's about a year and a half

since the deal has been signed. it's almost exact the two years since we originally passed this community. we need to make sure everything we intended to do is happening and if the agency needs modifications or changes for additional resources, that we can give this all of our consideration and the only way to do this is to have hearings like this. i would like to say, i have lately been concerned, i've been reading some media accounts that say some of the money may be programmed for other purposes including issues for the kids of the board and you know, we should be able to sign the money to do that without taking -- find money to do that without taking money from important research. but i hope this is not the case and it this is, hope that the

committee will act quickly in a bipartisan way to make sure that the intended money that we authorized remains because we have so many bridges to cross and will need every penny that we have authorized. thank you very much for yielding and now i will yield back. >> thank you. the arrival of the chairman of the committee recognizes the ranking member of the full committee. five minutes.>> thank you mister chairman i would like to thank the doctors for joining us to discuss the ongoing work at nih and fda to implement the 21st century cures act and think our colleague for without which the cures act would not become a law. if implementing critical provisions to improve the discovery and development of new treatments and cures. it provides significant funding

for the initiative. through this research program and brain initiative, the fda will include a provision for the medical product review process as well as new authorities and funding to ensure that the agency has the resources to recruit the best talent. hearing on this topic last november, providing promising updates, i look forward to your continued work as i said before, it's important to hold oversight hearings to allow us to learn directly from the administration how these policies are being implemented. i'm pleased the subcommittee has decided to conduct continued oversight. the are several topics within the subcommittee's jurisdiction that deserves hearings and oversight. for example i've heard a number of issues that are primary issues for the members of the committee does my for the committee.

the refugee resettlement, the abuse of the rems program, marketplace stabilization. these are different issues within the subcommittee's jurisdiction and we may have different opinions on them which is why we should at least have this hearing. these are critical issues that the committee should be discussing in an effort to find potential solutions so i hope in the weeks to come that the chairman will respond in select to choose to hold hearings in the short time left we have to hold this congress unless there's someone who wants my time, i will yield back mister chairman. >> thank you, the gentleman yields back the chair will hold the time for the statement of the chairman of the full committee pending his arrival. we think our witnesses for being here today and taking time before the subcommittee. we would like to give our

witnesses the opportunity for an opening statement. today we will hear from the honorable francis collins the director of the national institute of health and the honorable scott lieb from the fda. and doctor collins it's my understanding you brought a supporting cast of doctor norman the director of the national cancer institute and doctor stephanie delaney the director of the research program. i do not know if it's intended for all of you to give a opening statement but we will start with you doctor collins and you are recognized for five minutes with your opening statement.>> thank you and good morning distinguished committee members it's a great honor to appear before you again today along with my colleague the fda commissioner to give you a proper support on the mentation of the 21st century cures act and joining me as you just introduced the head of the cans are institute the deputy director of the program doctor

stephanie delaney. i can't emphasize enough to this subcommittee how much we appreciate your bipartisan leadership in passing this act. 51-0 as i recall. speeding the translation of scientific discovery into lifesaving cures, in the written statement i submitted i have outlined a comprehensive report on how we are working swiftly to implement the act of these provisions. especially in your areas of scientific opportunity supported by the activation. today i would like to highlight two of these. the cancer moonshot and the new precision medicine initiative. it aggressively pursuing a very ambitious goal to accelerate advances in cancer prevention, diagnosis treatment and care in

this advance increases immune -- included immunotherapy, to recognize and attack cancer cells and after years of research immunotherapy is one of the dramatic cures of cancer including leukemia, melanoma and lymphoma but some other cancers like:, pancreas, breast and prostate have proven less responsive. i'm thrilled to tell you that some of the barriers are seemingly ready to come down just last month, a team led by doctor stephen rosenberg announced the novel modification of immunotherapy approach that led to a complete regression most likely a cure. in a woman with previously universally fatal forms of a disease. and as always i have told patients the success for solid tumors, it must be replicated

for further study but without doubt, this woman's lifesaving experience represents hope for millions more. as exciting as potential cures like this might be and authorizing this, why we tasking nih with advancing not just cancer therapy for cancer care let me tell you about a funded trial that beautifully illustrates the progress we are making in 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 there faces a difficult decision, whether or not to undergo chemotherapy to improve their odds. thanks to a large clinical trial , we finally have some answers. it turns out that about 70 % of such women actually don't benefit from chemotherapy and a test of tumor tissue identifies them quite reliably.

clearly it is ideal that there are women with the potential toxic side effect of chemotherapy if it's possible to still have a good outcome. and on top of that and this will probably warm your heart because it certainly didn't mind, this is making it not necessary to go through chemotherapy for many of these women and will produce a minute -- significant cost savings maybe up to $1 million. to figure out why health approaches work best for each individual why leads to the goal of another important investment of the cures innovation fund which is precision medicine initiative. a centerpiece of this initiative, the research program, will enroll 1 million or more people and we are off to a strong start, on may 6, we watched national enrollment at

seven sites across the nation health and are part of our enterprise, human health centers and the department of veterans affairs. other enroll at volunteer for this sort of a little like look at health anyone with the older

members of, go to the url to learn more about your fear we will enroll children decide the little silver the added plan to open a portal identify enroll in every sample preserved and every electronic health record collected every survey filled out, the data will hold more promise for examining human health and with every scientist mining this data in search of answers, the more the promise will be realized. this is groundbreaking. it is exciting progress along with other advances, it's being made possible because of the provision of you and your colleagues. so, thank you for your investment in the 21st century cures act. we couldn't do this without you. my colleagues and i really look forward to your questions. thank you.>> thank you doctor collins. your recognized for five minutes for your opening statement.>> thank you to the members of the subcommittee. two years ago the members of this committee held this as a

potential game changer for patients and i agree, i provided a conference of list of the carriers act -- cures activity. modernizing clinical trials, the fda has embraced integrative trial design for the patient's trials and provisions. our aim is simple, innovative, evidence, to ensure availability for effective therapy. utilizing the development of provision technology, enabling us to target and these advances are not cheap. access is a big issue. i know it's a question of whether the market based system is financially sustainable. they asked if we could afford this coming waiver of precision guided therapies and i say we could not do this without them. restoring functions and

producing reliance across medical care within hospitals and in nursing homes. making it easy to bring innovations and competition to the market without compromising one bit of these rigorous gold standers for product regulation. rising trial costs and complexity impacts market competition and drug pricing and getting it to newly approved granted. one reason we may see higher costs is because competition emerges in some of these categories, targeting medical needs. we have studied this question with the fda and the data confirms these trends. i would like to share a snapshot today of what we found and plan to publish the full results soon. the conditions affecting larger papers and population -- larger patient population, 41 % between 1981 in 2000 had at least one competitor in the same class in

the last five years. this rate dropped sharply over the next decade, the same kind of drug approved between 2001- 2010, 18 % had it within class competitors for the last five years. another way to interpret the data is to describe the lack in competition. the drugs approved in 1991-2000 nearly 1/4 had a competitor within 3 years. the core haute -- between 2001- 2010, taking an additional five years and there were a nearly as much competition. competition lack by only 25 % of the new corporate drug -- cohort or drug. these trends mean they may long enjoy longer period with products in the same class. this may increase the price in power. this has to do with the difficulty of running clinical trials for the second market

drug especially if there is an available therapy for the unmet need. it's becoming harder and harder to be second which is a problem. conducting trials to address these challenges to give us more information about this at the same time. to advance these and complement your goals, the fda is trying a number of clinical advances in trial design. to master the clinical trial protocols including umbrella trials and platform trials. these approaches could sharply increase the trial base and lower cost moving away from one drug or one disease trial. but disease subtypes using a common trial. trial designed to compress this into one continuous trial. through these approaches you run one continuous trial and as you enroll patients to expand

the cohorts to enroll the patients using the information you learn about the features to help the benefit within the treatment. we will publish guidance very soon that lays out how product developers conduct these trials and how it to extend the trial to progress to expand cohorts as the trial advances. a lot of time and cost development has been waiting between the starting and stopping within these trials to compress this. every american has already or will one day face a serious medical diagnosis. we need to reduce the burden of the cost of advancing care. it will ensure that more patients who find they've got -- a better chance for making these successful for everyone. >> thank you doctor gottlieb and again thank you to our witnesses to be here. proceeding to the member question answer part of the portion of this, we will yield to the chairman when he comes

been -- comes in for the opening statement. i will open for choir of minutes -- for five minutes. thank you for bringing here -- being here. let me ask you a question about the 21st century cures, we try to identify a way to get regulation and policies inconsistent and to give you some flexibility to loop past some of these overlapping and the relief of the administered of burden could you perhaps share with us where you are in the review and how nih has identified some opportunities to relieve the burden on investigators? >> i'm happy to do so and again thank you to the committee for making all of these changes part of this bill. some may seem bureaucratic and administrative but they make a huge difference to us to carry

out our mission. we have been asked for in the's to look at the way we've asked our grantees to deal with financial conflict of interest which we think is very important for us to track with the mechanisms of doing this tracking seen as unduly onerous. we are in the process of going through this with our colleagues at hhs and i think that there will be changes to make this more of an efficient process. this sounds pretty down in the weeds but it matters a lot to us, the degree to which we need to do monitoring of what you might call a sub recipient where you get a grant for particular institutions where they have a sub part of that for another individual and in the past hour need to reach through and do very detailed monitoring with sub recipients which is something we required to do. we have moved to symbolize does make simple by the process. producing data that not many people look at and yet at the same time we need to be sure we

are being good stewards which is being simplified. looking right now at animal care and the oversight that is necessary of course to be sure that we are dealing with animals in a ethical way but some of these particular oversight mechanisms are now being reviewed and we put out a rfi and got 19,000 comments of how we might streamline the process. these are few of the examples and it was helpful to have these features. >> and obviously, it's going to be important for us to communicate on this for ways we could provide additional legislative health on that. i think you could find the committee willing to have these discussions. and doctor gottlieb thank you for your comments. i always felt like while we were doing the roundtables for these cures this is where the big money was and if we could reduce the time in trials and reduce, if a

product was going to fail we would allow it to be identified to see if it will fail early, facing something that was not going to pan out, i would really like the concept about the seamless trial concept. the time between phase i, phase ii, and phase 3 clinical trial i think it's likely to have a significant impact for the things that have always concerned me. with had some legislation, there's been controversy but there's also the ability for, we don't have a payer here. but the researcher and the regulator and the payer, to communicate that for -- before something comes. the payers at the state level certainly, the medicaid payers were not ready for what was happening.

they have been prohibited from having those discussions because of the laws. i'm hopeful that we were able to do some thing, particularly gene therapy or gene editing we have one shot that will cure something that is a lifelong problem. that is huge. it's likely to be priced out of the range of most average people's budgets.>> can you comment on that, our ability to community prior to approval? >> thank you mister chairman. i would say we fully addressed this issue and your point is well made. i share your concern and have shared your concerns in the past. i think we fully address this issue with the guidance we issued earlier this year. that wasn't part built off of the provision related to payer communication with product developers for purposes of engaging in value-based contracting and other economic kinds of discussions where we have essentially established a

safe harbor for those kinds of medications where the fda isn't defining whether or not the -- someone argue we don't. on first amendment grounds. even if we did we would not choose to exercise that authority because as a matter of public health we believe there should be robust discussions between product developers to suggest that they could engage in value-based novel therapies.>> thank you for this answer. >> i would like to welcome our panel here. let me give you an example of what's happened in the last four years. 2014 we had a outbreak of ebola in west africa. there was no treatment for it. what we are seeing today in the democratic republic of condo we

have seen a vaccine. what has happened in those four years now cures came on in 2016. but can doctor collins or doctor gottlieb share that, how did we get there? back in 2014 there was no vaccine. >> i will comment on where this is now. >> we begin working on the ebola vaccine in 1990s when no one paid as much attention. this was a real concern that at some point this could become important it was a fundamental mount of basic science. this works for a good 15-18 years before the crisis struck in 2014. that being the case, this was in fact, a program that developed the vaccine in record time.

but still, in fact a program that was developed, a vaccine was being distributed, good health measures were resulting in the epidemic, waning rather quickly. which was a good thing. this vaccine which is manufactured by merck, the phase 1 trials for this very vaccine were done in the clinical center showing that it was safe to generate antibodies. this was distributed at drc in a ring vaccination strategy and giving -- and given to health workers. we were happy to see the declaration that this epidemic is now over. if the vaccine contributed to that it's hard to tell because fortunately, this was a limited outbreak. drc moved quickly with traditional measures, i think

again, we are very well situated to deal with a ebola outbreak in the future and in this instance, it was great to see how quickly the vaccine was available and was distributed and made available to those who needed it.>> i will comment to build off of doctor collins is comment. i make the point up front, it's hard to understate what a game changer this is. the first time we now have a technology available to intervene to stop of spread of something other than just traditional public-health tools of isolation. i think we should make note of the efforts of the manufacturing company merck, the doses that were employed in the drc, they were donated. they shipped about 13,000 doses and handle the transport. delivered under a extended active protocol. this was very much a altruistic effort that helped in that setting. we are working efficiently to dry to move toward the license products here in the u.s. obviously we need to be cautious of what we say but i

feel optimistic that at this point, it's something we could accomplish in the near term. >> well and, that is just an example because a bullet did, we had a gentleman from west africa come to dallas texas and the protocols were not followed and so, this impacted our own country. using this as a paradigm on what we could do for other terrible illnesses that are developing, i tell people on any given day, we have tuberculosis in the city of houston. it is a international city and you just don't check everyone. we have to be on our toes to be able to do that. i think this gives you some of those points of focus. looking at the ebola vaccine in four years we went from people worrying, i tried to explain to people, four of my constituents might die of the flu because they did not get the flu shot but would never be exposed to ebola but we want to keep it

that way. i'm concerned about the growing threat of the antibiotic resistance which is why sponsored the provision in the 21st engine does make 21st century cares provision. it directed the fda and cdc to coordinate efforts with respect to monitoring antibiotic resistance and any other drugs approved under the limited population pathway, the antibiotic and antifungal drugs. doctor gottlieb what steps is the fda taking to coordinate the cdc to support policy to promote traditional and demonic use and antibiotic stewardship? >> we've taken a number of steps. to look at the antibiotic use in animals, the links of duration. planning to have additional policy steps that we should announce within the next couple of months. to continue to advance what we have already done with regards to the use of antibiotics.

limiting one room by which we are seeing the system develop. with both the provisions, the provisions as well as the steps, i was enabled within a steps committee to create new pathways to provide additional incentives and additional efficient pathways to get new drugs to the markets to attack some of these pathogens. i think it is important that we focus in trying to develop full incentives and new ways to potentially reimburse some of these limited anti-infectives to create additional incentives. we are working on one such idea in conjunction with our colleagues to try to move force -- forward for the drugs that are the kinds that you would want to put on the shelf and

never have to use moving toward the site licensing model as opposed to a pay-per-use model which is the traditional way we take the drug.>> thank you terman, i know i'm way over time and i will yield back the time.>> the vice chairman of the subcommittee, five minutes requested.>> i've got a couple of questions. i know there's a lot going on in this great city and great country moving forward, a lot of times what's happening, this probably doesn't make a lot of primetime television. it is important, it's extremely important. when the bill like the 21st century cure has a voice on the midi, overwhelmingly bipartisan, it makes the difference in people's lives. the doctor and i had another meeting with a member of the group. i would like to point out, he was talking about someone close to him who had cancer. i remember walking away, i had a friend who passed away when i

was 11-12. she would be alive today if she had the same disease this time, probably would be alive today, the reason i point this out. i think they really love this country. people throughout the world do research. no one compares to what you are doing at nih or what we are doing as a country or what our healthcare system or as an industry, public-private partnerships, it's a shame, there's a lot of good news. with what this panel is doing. we appreciate this very much. i had a question for gottlieb regarding the cures act. including provisions to clarify regulatory requirements including processes, could you explain how the fda is delivering on these efforts to

make the complex area of regulation?>> we've put forward a number of additional guidances as well as staff manual guidances on how we approach combination products within the agency, historically it has been challenging for the agency. i think we've made a lot of progress in recent years with some of the provisions within the cures act as well setting up a combination counsel to try to dedicate who has -- adjudicate who has primary jurisdiction over these cures. will put this out sometime at the end of the summer and early this fall that will make some for the process reforms to make it more efficient for products that sit on the cusp to move within the device realm which is a preferred route for a lot of product developers if they could get there. we will look for ways to find the most efficient route while being mindful of the public health obligations within the circumstances.>> well thank you, and we spoke briefly,

concerning drugs sorted is and drug supply shortages. sometimes they don't have just the simple things they need, they have to figure out other things to move forward, i know some of this, the natural disaster in puerto rico i think, you guys really went into handle this. there are people in government that are trying to make things work. we've got issues we need to address about what happened in puerto rico, getting pharmaceuticals moving again. i know there are issues, emergency room physicians, geologists, basic things, could you talk about a minute and a half of what you explain to me on the phone and what you are doing with your drug task force and what's pressing and moving forward?>> the challenge here it's not just one thing today it will be one thing tomorrow and we are dealing specifically with

be chronically in shortage. there are structural problems and that, it's been driven down so low, these are slightly above the cost of goods so the way to properly manufacture them on the tremendous scale we've seen consolidation based and we've seen underinvestment in manufacturing because there aren't a lot of markets to reinvest in manufacturing. manufacturing is not trivial. it's one thing to manufacture and have a drug slightly above the mark but it's different when you try to manufacture sterile injectable drugs. when things go wrong, if one facility gets shut down it takes away 30 % of the market and we think that there are things we could do to define a pathway. quite frankly, i'm concerned my regulatory touch will only exacerbate the problem in regards to increasing cost. we are trying to do -- we are trying to do is

look at this holistically to look at how we might couple some additional regulatory steps to mitigate shortages or prevent them with potential changes and reimbursement structures, we talk a lot about drugs that are priced too high. there are drugs that are very costly, the value that they are delivering. there's also probably a price too low relative to their importance and the cost of manufacturing. i think we need to take another look at how we embers -- we reimburse the problems regarding these patent drugs. >> thank you all for being here.>> the ranking member of the full committee, five minutes for questions please.>> thank you mister chairman.

first, i've got a question for doctor gottlieb than i would like to go to doctor collins for a second. last week the fda announced the release of the similar action plan which tries to encourage competition in the biologics market which i believe is necessary with the patients realizing the pull does make the full benefit. only 3 bio similar markets having approved 11 of them. as you've noticed yourself doctor gottlieb, you're well aware that the high cost of prescription drugs including biologic continues to be a barrier for many patients, the cause of the action plan revealed in a recent analysis found that is all fda approved bio similars were available or could you discuss further the fda analysis and savings potential that they could offer? >> i appreciate the question,

we will polish the full results of the analysis but essentially what we did is we looked at the experience in the european market where we saw product, we extrapolated what could have happened similarly to what we had seen in the u.s. market. we saw the competitive landscape from europe made some corrections to the fact that the dynamic between the european market was slightly different than the dynamic in the mac market. looking at the percentage price reduction from one, two, 3, four % of the market. and when we extrapolated those findings back to the mac market and assumed that of every bio similar was approved in the market was launched we extracted that $4.5 billion that would have been saved in 2017. it is quite significant. by undressing -- underestimating, looking at this to the same magnitude, it's quite significant. they start to approach this type of savings with the

product introduction.>> thanks a lot, really. the second question, i was wanting to commend you for your continued dedication. regarding the competition. how could congress work with the fda as you implement the bio similar action plan to help facilitate better competition in the bio similar? >> i appreciate the question, you know, we are going to look at some of these more difficult scientific questions, i think you know, they could facilitate more competition and changeability. one in particular that we are going to be looking hard at is being able to use the european product as a reference standard. we have situations where a bio similar might be manufactured in the same facility and distributed in the u.s. and europe. we know that, but our knowledge

, we have to require biro similar entrance to run a study against the product even though the european product is the same thing. the european product may be cheaper and easier for them to source. so there are opportunities to have cost savings if you could source a reference product globally and i will add, half of the cost right now, developing the bio similar, on average the cost is the cost of acquiring the biologic to run the trial. so the savings could be significant.>> thank you.>> let me quickly get to doctor collins you mentioned in your testimony that nih is currently building data resource which could ultimately help research similarly the data collected by the data sets. it obviously has pros and cons. let me quickly put two questions into one. given that they are asking for

the participants to share about the lifestyle, how do you plan to ensure privacy and data security and are there protocols in place to share updates or study results for the participants? >> i will ask the deputy director regarding these issues to respond to your question. >> thank you for the question and thank you for having us here today. so, we are thrilled about where we are with the program, we have 86,000 participants, we know as we try to engage as a community of diverse participants up to 1 million or maybe even more, privacy is one of our most important priorities. all of the data comes into the data resource is encrypted and to be identified going into a secure place of research. they will have to provide a code of conduct and no data will be allowed to download it. i would like to think the committee for giving strong privacy protection within the act.

including the certificate of confidentiality to allow us to provide robust protection to our participants of confidentiality. >> thank you, we recognize mister upton. five minutes requested, please.>> thank you mister chairman. i would like to remind everyone that this was a great committee in process, 51-0. i want to everyone who is done the committee with congress has had a piece of this. the staff was terrific for sure. so were the agents these, you helped lead to where we wanted to go, the administration, the senate, we had all of these groups with a lot of players, we appreciated the inputs, i think based on the hearing today and what we really thought would happen, it's fulfilled our expectations. we have a lot of time, it's a game changer. i have to say, way back when, i

was one of the participants with john mccain and paul wellstone, they doubled the money in the nih back in the 90s. and for the sponsors of that bill, they got it done. it was a very important element that we had to increase the money for the nih knowing that we had a terrific store amma conductor collins, able to lead us there. river sitting upstairs, i managed to get my partners and a few others to talk about the amount of money that we wanted to add to the baseline and we ended up getting $45 billion added over a 10 year span. we came up with those not once but twice. a comment made earlier, it's important to have maybe similar from hhs, i was alarmed when i

read the story that hhs was perhaps reprogramming some of the money that we had done, a dollar for dollar year-by-year to make sure that the money went for the nih, i don't know if doctor collins if you are able to acknowledge if there is some truth to this public report that money was being taken away from what we were able to do and put it to something else. i don't know if this is happening and if it is, how do we get it back?>> so, i am not aware of the published report you are mentioning. there is, i believe, some subjective language that prohibits the transfer of funds that are allocated in the innovation funds about the secretary for any other purposes. so i think this is nicely designed so that those funds are intended to go for the original plan.>> that is good to hear.

>> i would like to talk about all of us and i think, privacy protection, where it is important, it's very important, people know about them, they tend to worry about the data being stolen, as we found particularly from the roundtable, people or individuals who are more than willing to share what their own experience was, knowing that who could protect someone else from maybe the same ailment and so, it is exciting to hear that 87,000 people have already signed up really in just a couple weeks. i know for me, particularly with privacy protection, we would like to maybe set up the cost this year in the caucus which could be bipartisan to encourage members of the staff to participate, we have about 25,000 people who work here on the hill. and they have a fixed percentage to figure that out.

how quickly, i know that researchers will have access to the information in about a year or so, how quickly do you think some of the data might be able to be utilized or figure out the right pathway for some real important research to be promising? obviously the news over the last couple of weeks about chemo and not having chemo for breast cancer that was really important, how fast do we expect that maybe we may be able to use the information by researchers with some concrete result? >> yes this is a great question. we are excited to get some of the brightest minds around the country accessing the data.

and as you mentioned, 86,000 participants, they will get richer and richer as we had more participants. i would imagine, once we give access to researchers, we would shortly begin to see some pretty significant findings. the data gets richer with more participants and over the long term as well as we get to see people who are healthy and remain healthy and they respond to therapy and all of this will be.>> how long does it take for someone, if they sign up to do this, what is the time element for this?>> the online enrollment process takes about 45 minutes to go through the consent and to authorize your electronic health record and we have the visit were people donate blood and going to the physical measurement that takes somewhere between 45-minutes to an hour. will continue to fill out surveys and do other things essentially to make -- to maybe even come back for these visits. >> thank you.

>> thank you mister chairman and good morning doctors, it is an honor to have you here, i can't help but think that while most of the people in the country don't know our name for what we do, they are all counting on you so thank you for what you do. i think that he represent part of the real genius of our country. i always say, the nih stands for our national institute of hope. you gave us even more hope with your report doctor collins. doctor gottlieb, i know that you are aware that legislation of my wonderful colleague, congresswoman stephen brooks and i, we wrote the public health emergency response act which was included in the 21st century cares act.

the bill established a priority reviews voucher to encourage the development of the medical countermeasure for the drugs and vaccines of the fda. when we develop the bill, the fda at that time expressed concerns allowing about offense countermeasures qualified for the ferc which dramatically increased the number of those awarded. the first product was improved -- approved earlier this month. the fda -- do they have concerns about the number of products qualified under the legislation, could you tell us if there has been good interests , you know, on the part of the companies who purchase for it? could you maybe briefly inform us about that?>> i appreciate the question congresswoman and we appreciate the efforts of the committee -- of the

committee to provide incentives. the first product that qualify for one of these was approved by the fda. it was essentially an important product, a treatment rather than just purely a vaccine for smallpox. the holy grail was always to have treatment in the event of smalltalk's -- smallpox being used as a bio weapon, i do not know as my colleagues were here before me, i certainly wouldn't argue that we've got too many countermeasures coming to the agency, if anything, it's probably quite the opposite, we would like to see more drug development in this realm with economic challenges to develop the medicine.>> i appreciate that. i do not know whether it is doctor collins or doctor sharp, pancreatic cancer, it is still. i did legislation some years ago

regarding this. could you give us a update of if the needle has actually moved. i know what i did legislation and i can't recall the year, the needle really had moved in about 40 years, could you give us a quick update on that? then i would like to go to for question.>> the answer is yes, the needle has begun to take slightly. >> marvelous. >> i think we have seen some really good stuff. if you could respect pancreatic cancer, those patients are doing really well. respectful to pancreatic cancer, the group is growing and we had a positive trial. and so for the significant arrival of therapy with these patients, for those who don't

have the mutations, some of these patients are responding to novel kinds of inhibitors.>> i think i would like to follow up with you because i'm not a doctor so i do not understand this. but i know that you could break this down for me if we had a conversation or meeting so i would appreciate that. thank you for your wonderful report, one of the places where the program is making -- taking place is within my congressional district in palo alto da. could you give me a quick update .

>> think you know we have five minutes for questions. >> thank you mr. chairman. thank you very much for being here and we do appreciate the testimony you provided. is important for this country and also, he's not here right now but i want to thank him for his leadership on 21st century and getting us where we are today. >> i have a question for you in your testimony you talked about the rain and initiative. especially at the end of the testimony talking about reagan increase understanding for brain health and a means of preventing brain disorders such as alzheimer's disease, autism, traumatic brain injury. i know i also see is also $1.5

billion that has been allocated over the next 10 years for the studies that are going on. i know that when i have been at the house and see what they are doing with parkinson's in their studies, the one area i would like to focus is on alzheimer's. because as we look across the nation today and seeing what's happening with the alzheimer's association reports, you are looking at costs can rises high as $1.1 trillion. fifth relating cause of death, one in 10 americans over 65 will have it. we have about 5 million americans living with alzheimer's. one third over 85 are afflicted with a. but unfortunately looking down the road over the next almost 30 years people over the age of 65 there will be 14 million people afflicted. where do you see the studies going right now? with alzheimer's?

>> this is an area of intense interest and is a major challenge for the world and certainly that's very true for the united states. you have quoted the frightening numbers of individuals that are projected to be affected and the cost is going to be apply to our healthcare system. even more than that the personal tragedies that happen every time a diagnosis of this appears. it affects not only the individual but the family and the caregivers. we are trying to find a aggressive strategy and pathway. we are grateful to congress over the course of the last three years congress has progressively given us greater resources. there also during that in a very visionary, productive, partnership with industry. something called accelerating medicine partnership to try to be sure that basic science we are doing on the brain, earliest signs of alzheimer's, is translated quickly into therapies.

one of the things we are learning is alzheimer's begins long before the first symptom and corms -- terms of cognitive function. we need to find people at risk at the earliest possible moment and not wait until they are already in the circumstance of already losing neurons that will be hard to get back at that point. i am guardedly optimistic that we are on the right path with some of the trials underway right now. we have the opportunity to treat people before they have any symptoms. but we know there on a dangerous pathway and can watch and see what's happening. we are also learning a lot more about roles that play hand-in- hand and what happens with alzheimer's and the immune system. we hadn't really realized before, inflammation seems to be important in this condition. all of the focus that 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

on all this and i'll stop now because of the timetable. but i do want to reassure you that between basic and clinical science working with industry and close collaboration with advocates, we are pushing this as fast as we possibly can. >> i would just add if i may, we recently issued a new guidance document updating agencies guidance on clinical trial points in the study and particular disease study. historically there was perception had to show improvement in both functional status and cognition with approval, slow the decline in cognition. that is challenging. by the time the functional status declines as an alzheimer's patient you probably had a pretty significant decline in cognition. now that we are able to identify patients earlier with more sensitive tools also patients at high risk of developing alzheimer's.

we want to intervene earlier when there are slight changes in cognition to try to arrest the process. so what the agency has set in the right circumstances, showing improvement in cognition alone that could essentially qualify improvement for a drug that could slow alzheimer's in the right population, properly identified patients. so we are trying to work closely with nih as well. >> that's really important to have that. >> chairman can i ask member of congress come up and sit in the ranking chair as a cosponsor of the chair -- bill.

>> thank you so much i just thought mr. green wanted a break. there was very nice of him. again i want to think i want to add my thanks to all four of our witnesses appear for their extraordinary help not in just passing this bill but also in implementing it. it really warms our heart to see how much progress we have made in such a relatively short time. it also encourages us to think how much more we can make. i just have a few questions for each of our agencies that are here today. i think i'll start with the fda. dr. gottlieb, the biomedical community has really made great strides in developing clinical therapies from unlocking potential 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 cutting edge therapies really requires an experienced, well-trained, robust, public work help force. we had a provision for new hiring authorities. i'm wondering if you can talk to us about how that hiring authority has helped the agency recruit and retain talent? >> thank you, we are in the process of implementing that. i think is going to be transformative for the agency in terms of our ability to recruit people with specialized skills. we have identified 38 occupations that meet the requirement for the alternative pay system. we have made the first two hires, both deputies and directors. i think this is going to be very important to the agency. just to briefly pick up on your point we're talking about new

treatments, the clinical characteristics of the product relate very closely to the product features. the products features change very quickly. we underline technology with the product itself. these are not pills anymore. it used to be if you had expertise in the scientific field and clinical field you could adjudicate all different kinds of drugs. but now it requires parties in the product itself. there's a lot of novel technology you have to have engineering skills on that specific technology. that's where this is going to be particularly helpful. try to find people with specialized skills. >> these are people you can't just hire right out of grad school, these are people with very specialized areas? >> that's right, they are often employed in endeavors in the private sector where they are highly paid and highly skilled. so we are competing with others for the same multiple talent.

>> in the fda june 2018 report you talked about the past work of hiring authorities that have created challenges. are you beginning to be able 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 i'm grappling with now grappled with 2003 and again 2007. we are addressing it very directly. we're trying to do it top-down. wholesale change of the hiring process, make it more efficient. we started a pilot which dramatically's streamlined the on boarding, hiring process. we focus that pilot on several issues. we decided to take the structure of that pilot and apply to the whole hiring process.

>> i hate to cut you off but i can't ignore dr. collins. and i have about 10 minutes of questions for you. but you have a minute to answer them. let me just ask you, what kind of research is going on over in your agency on alternatives to opioids for pain management? because the opioid crisis is one of the big issues is really been facing this committee for the last year. >> thank you for raising this this is an incredibly important issue. we are grateful to congress with the additional $500 million put into our budget to work on the opioid crisis. a significant fraction of that is being devoted to the need to develop effective and nonaddictive pain medicine for those 25 million americans who suffer from chronic pain every day. and for whom opioids is not the answer it's actually quite harmful in many instances. this goes all the way from basic science to discover new

targets, to working with industry to help free up some ideas that have been on the slow boat and now can be speeded up. we are working in a collaborative way. we are setting up a clinical trials network so we can quickly test and see therapeutic that are non- addictive. all pain is not the same, low back may not be the same as you get from trigeminal to neuralgia. we need to get ready to go with the type of clinical program that does not exist. >> one less thing dr. gottlieb. i have some questions about roles that have been pending about therapy and the fda. we don't have time to answer those questions right now. but i'm going to be contacting your agency to find out why those rules haven't been approved they've been pending since 2016. and i promise my constituents i would ask about this. >> i would be happy to discuss that with you moving forward.

>> thank you very much. >> thank you mr. chairman we appreciate you holding this hearing. we want to thank all of our witnesses today. and the very talented men and women that you're speaking on behalf of in your agencies who do amazing work in the area of healthcare in america. the legislature passed, curious, reforms to mental health services and i thought today was a really important day, an important hearing to continue our work to legislate and evaluate. and see what else we need to do going forward. what is working, what's not, what do we need to change? i know i've had a lot of roundtable discussions in hospital boardrooms and community centers, town halls with people in my district. people who are very pleased with the investments we have

made in medical research and in turn the work that your scientists are doing. to return that money in research into reality and medicine and cures. it goes from one end of this table to the other. so we appreciate the work that you are doing. i know you will hear that every day. very excited about precision medicine that cures -- the cures that are out there, waiting to be found in the work that you are doing. dr. gottlieb in your testimony today you suggested a correlation between lowering radical product costs and promoting modern clinical trials. can you elaborate on that? we are all concerned about the health care and medicine costs. clinical research is and always efficient as it could be. biomarkers in drug development tools how did it make that process more efficient?

>> clinical trials directly translate into how a product. based on what the product -- market is going to bear. we know clinical trials to factor into the rising cost of overall development and competitive nature of this landscape. when i talked about in particular today is the complexity of conducting clinical trials especially in areas of unmet medical needs when there is already available therapy is my -- in my view delaying the market second and third and class drugs. specialty drugs that target unmet medical needs are enjoying monopolies for long period of time. if you believe the competition jaded a data shows competition comes in market prices go down. we now have data to demonstrate this we will be publishing that soon. i think there are things we can do to try to facilitate more efficient root markets for second and class and third in

class drugs. at the same time increasing being effective not sacrificing anything. those are the type of development we are focusing on. >> trials for diseases can be difficult to conduct especially if there's a fda approved treatment already in the market. how do you focus your clinical trials on rare diseases? >> one thing in particular is looking at natural history models, model the behavior of the control arm or model simulation. you'll have to enroll placebo trials because if there is already therapy people don't want to use placebos. they want to use an active drug. this is the place we could benefit from more disease models. we should be able to create those. there is a request of the president's budget for about $20 million. i know we have support from this committee and others in

congress. we are gravely grateful for that. i think we all need to be working together. >> i appreciate that is a good reminder, well done, well played. dr. collins, and i saw the nih prostate cancer men of african ancestry to find genetic markers and structural stress. a study funded by 20 century cancer moonshot initiative will investigate environmental and genetic factors of prostate cancer in african-american men? can you tell us more about this important study? >> thank you for the question this is alluding to a trial just announce that will be the largest trial on this topic in our history. is going to take 10,000 patients to try to analyze genomes and

look for disparities. african-american males are more likely to be diagnosed and die of prostate cancer. understanding this important disparity in our country is a really crucial question. >> anything else anyone wants to add? >> the point again to the study is a poor form that we will be able to utilize going forward for answering many questions about sort. we have this goal and we aim to reach it, about 50% of the participants and all of us are going to come from traditionally underrepresented groups, racial and ethnic groups. if you want to really examine a health disparity circumstance you're going to have 1 million participates who are highly motivated to take part in research. we have taken a great deal of data and we're going to learn a lot once we have that platform up and going. >> thank you all we appreciate your continuing involvement with our committee has been

positive and effective i think for the american people. we look forward to doing more together. >> thank you mr. chairman, welcome to all of our witnesses today. i share your enthusiasm over the future of the cancer moonshot and precision medicine. heart of my enthusiasm stems from the fact that back home in tampa, i represent moffitt cancer center, designated center in the state of florida. i love meeting routinely with the young scientists and 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 and competes with all the other needs. now they feel like 21st-century here are these new investments the cancer moonshot gives them hope for all the research and

the cures they are providing. the moffitt cancer center has been a national leader in building large data sets and tissue samples and working with other institutions, especially for those that have underrepresented communities. you have given us a bit of an outline on protecting privacy for people who participate. can you tell us a little bit about the protocol going forward for researchers, will they be required to share their research? when will that happen? will those research results be available to all other researchers? >> thank you for that we have been thinking a lot about the fair point about young investigators and diversity of researchers they can also access the data. making sure the platform where

building is open to everyone. and feel responsible for sharing things that we learn on the date of the list are generally provided by our participates is returned to the scientific community. we are developing policies ensuring that researchers who access the data acquired follow a code of conduct and share the results within a specific amount of time. we are still finalizing these policies in anticipation of opening of the data next year. >> dr. collins in the past it has been kept researchers held a closed. what does the future holds? >> i appreciate the question, i appreciate 21st-century cores gave me as the nih director that i did not have before to require data access for studies that we support.

i could cajole in the past i could try to embarrass people but i didn't really have the clout to say this is a requirement. if you're getting a grant from nih you are required to make your data accessible. you all gave me that in the has been a very useful tool. we are very engaged in the process of trying to establish exactly what that needs to look like for a wide variety of studies. we've done it specifically for genomic we have a very worked out data sharing policy. where are in the process of working that out for things like imaging and electronic information. this is a very high priority. we hate those silos to an been having a good time knocking them down. >> your predecessor was well known for his research on hpv and the vaccine. what can nci do to help prevent cancers, detect them? over the past years a lot of the nci,

there has been new focus on making sure there is greater uptake of the vaccine across the country. do you intend to continue with that? i mean if we discovered the cure with cancer there would be praise in the streets but here is an actual vaccine to help prevent cervical and a whole host of other cancers. i think it's important. are you committed to continuing those initiatives? >> very much, dr. lloyd continues as my deputy director. he's incredibly valuable in that regard. entering the federal an academic is challenging. we have a robust portfolio in this area. i think one of the most important studies is a trial of doses of vaccine. right now they are required three doses that's a big implementation of

the committee. if one dose works well that could be game changing for subject united states as well as globally. >> i guess it's a good reminder , it's important for boys and girls, middle school to get the vaccine to prevent cancer in the future. is that correct? >> yes there are number of vaccines things that work we need to assimilate them in the communities that the real challenge for us. >> thank you very much. >> thank you mr. chairman. good morning to you both and thank you for your enormous public service. for dr. gottlieb, the drug act has helped to create a market- based system create a new development of new medicines for people living with rare diseases most of whom are

children. in exchange innovators are granted seven-year monopolies in specific disease area. the act was established in 1984, medicines have been brought to market for only rare diseases as you both know. there are no medicines at all. many of these diseases however, there has been zero second generation, nearly enervated medicines brought to market for patients. multiple disorders for example there have been enzyme replacement therapies for eight different rare diseases. the cost on averages nearly $500,000 per patient for the year. however with the exception of disease not a single second- generation therapy has been approved for any of these diseases. the first generation art

approvals and most of those date back at least a decade. in your view, dr. gottlieb, have we created monopolies in many rare diseases especially in areas i have discussed? what are the barriers to moving innovation forward versus competition and innovation to bring newer, better medicines to patients with rare diseases as rapidly as possible? >> thank you for the question, this cuts to some of the other issues we discussed here today. it's a very relevant question. i do believe that there are studies where it's harder to bring second to market competition in certain categories. i would argue this is one of them. particularly the case when you have drugs to target medical needs for small populations. it's hard to run clinical trials when only one therapy is available. typically the subsequent drugs would have to be studied on top

of the available therapy. you would have to show improve efficacy with combination, it's hard to run head-to-head studies one already effective therapy is available. people don't want to forgo effective treatment when you're dealing with the child. i think there's a lot of things we can do. earlier this year we published in conjunction with our counterparts in europe and massive protocol and how you can use multiple drugs in the same clinical trial structure. we talked earlier about natural history models, the behavior of patients who go untreated. we should know how these diseases progress. we know how they affect patients. we should have really robust natural history models to be able to model that information to enroll so we don't have to enroll patients in placebo trials. that will make trials easier to

conduct. there's a whole host of clinical trial reforms i think we can pursue to try and speculate on the market which is critical in these errors -- areas? >> is there something statutorily we should be doing here in the legislative branch, doctor? >> i would love to have that discussion with you there a lot of things we can do and are trying to and will do. i think one thing that i would just confirm i have this discussion with the chairman about natural history models. we allocated $6 million earlier this year to develop six natural history models for multiple diseases. but we did in our budget this year request additional funds to pump up the additional 20 natural history models that will focus on super orphan diseases we will focus on resources there. >> thank you i look forward to

working with you as we have in the past. i have been the chair of the rare disease caucus for quite some time. this is completely bipartisan in nature and we look forward to continuing the discussion. dr. collins is always a pleasure to see you. mr. chairman i notice in the audience john crowley who's been very much involved in the rare disease and his daughter megan. there are residents of new jersey. there is no one who is fighting for progress in the rare disease face and my friend mr. crowley. thank you mr. chairman. >> thank you very much mr. chairman. i do appreciate it. i appreciate you all being here. june 8, 2018 the committee sent fda bipartisan oversight request for information about the fda increasing criminal enforcement at ports of entry to combat the opiate epidemic.

an initiative that you have championed and we support. the letter has only six requests. some of the requests were first posed by an email to the fda office of legislation on january 30 of this year. it is now late july and the fda continues to tell committee staff letters and clarence. commissioner, did you help expedite clearance of this letter so the committee has fda's response before the end of july? >> i absolutely will it is and clarence. and in an effort to try to improve transparency get information out publicly i did make most of the information in the letter available publicly and some remarks i gave in a form we had to try to work with intimate stakeholders. we address opiates -- opioid sales online. i can make those remarks available to you. we may then widely available. i recognize the importance to get a formal response back to congress. i'm on it.

>> you have so much to do and i really thank you for the good work that you are doing. there's a lot of different areas of weep have been working out together. i appreciate that very much. what further improvements will the fda make to the expanded access program to ensure the process is effective and efficient for providers and patients, particularly now that the federal right trial legislation has passed. i like the house version better than the senate version. give me an update on that? >> have a process underway that we are looking at what's best we should take to facilitate facilitation it's going to be a pathway it's alongside extended act. are still going to operate our extended access program. we did bring in an expert group to take a top-down look at our

extended access program and make recommendations on how we can improve it. i'm going to make that public soon to provide transparency around what they found. but we think there are additional processes that we can make it easier for people who aren't as sophisticated or physicians who haven't done this before, to access that. we are also working with cancer research and udall foundation to create a platform to have a one side of entry, if you will. get info from information what the extended access program still exists. the challenge still remains, difficulty, sometimes reluctance but sometimes difficulty sponsors to make drug available especially when we're dealing with things like biologics. the costs aren't trivial. when you're a small company you barely have enough product for the clinical trains. i've been on the other side of this. i know how that is. i think we still need to look for ways we provide proper incentives, try to make sure we have really promising therapies.

we might be able to make that available to patients. >> i appreciate that. i've been an active proponent for many years. if people are facing death they will take that hail mary pass and take whatever they need to if they can. let us know what we can do to assist you and matt as well. >> dr. collins am going to ask you couple questions to give me an update. i do have a partial answer i found on the internet. that is with lou gehrig's als. i understand you had a breakthrough this spring. but i'm also interested in huntington's korea. also ask i have members that are afflicted with both of those. >> those are both terribly important and often tragic neurological diseases. with als as you mentioned there has been encouraging developments of a new therapeutic approach this spring. still very much in this process of being evaluated. huntington's disease i will reflect a bit on 25 years ago my own laboratory was involved

in the discovery of the gene that is responsible for that condition. it has been very gratifying to see in the course of this -- just the last year or two to build a model. very encouraging information to use a genetic approach to try and lock the production of the toxic roti. is now in human clinical trial which we are waiting for the full data to be rolled out. is sounding encouraging. a similar possibility to utilize molecular therapy into spinal fluid that gets to the brain may be able to provide benefit. goodness knows we've waited a long time for that to happen. we have a long way to go before we can say we really have the answer. but this is a lot different than saying we have nothing to offer. >> i appreciate that. >> thank you very much i

appreciate the opportunity to openly discuss witnesses progress we made. about every day the united states congress actually pass laws that we looked at and said i think we did something good. i want to thank all of the implementers for doing good work . my first question has to do with the diversity and research of subjects. so thank you for, i was excited to read in your testimony about 85,000 individuals have already started the enrollment process for the research program. 70 to 75% from communities have been historically underrepresented in biomedical research and almost 50% specifically from rachel and ethnic groups have not been included in previous research. as you all know, diversity in medical research has long been an issue. i believe one way to diss

decrease health disparities in this country is to ensure we are including individuals of all races and ethnicities in medical research. with that my question is, what is nih doing to ensure that all of us research program recruits a diverse group of participants that is racial and ethnically diverse? >> we track that week by week. i personally am asking for that information every week to see how the enrollment is going. but i'm going to asked dr. devaney to see how this is being done. it's unprecedented to have this level of diversity. >> thank you for the question. this is perhaps the strongest priority of the program. understanding a lot of the data scientists are using today is not applicable to all communities. we have a number of different ways we are continuing to reach out to diverse communities and

go into it with the understanding that we are going to try things and is not going to work and we are going to have to shift and try new things. one of the things i'd like to highlight is working with community partners. we have a number of community engagement partners across the country that are hope helping us build relationships with their communities at a local level. our chief engagement officer at the nih has really been working with those groups in a very robust way. we have about 30 partners international that are helping us build awareness with other communities. >> you're talking about groups that are more local and might have a propensity for working with a particular population? whether is the immigrant or hispanic population or black population etc., working with local groups already have relationship with those communities? >> that's right. >> when we did the launch may 6 several different places i was in new york at the abyssinian baptist church with an african- american community. they were totally revved up in the opportunity to be included in this time.

i got to give credit about this focus on inclusion. you've given us additional tools there with many different projects across the board. but this is turning out to be a flagship here on how to do this in the most visionary way. >> this opportunity for us to remind ourselves there is diversity in research when it comes to subjects. they all genetically have different reactions and different propensities when it comes to certain diseases and affects, correct? >> absolutely. one should not assume when you see a health disparity that that something that is readily understandable on the basis of looking at one thing. usually these are combination of environmental exposures, stress levels, genetics and there but one should not over interpret that part because we are awfully similar to the dna level. if you really want to understand the health disparities you have to have comprehensive studies that collect data from all those

sections. >> is better to have research rather than having to extrapolate out that's more guesswork does not necessarily scientific. >> you want to have the data on the people whom you need to offer answers. we just heard from dr. sharp about a big study stop with african-american prostate cancer. we don't understand why men who have prostate cancer who are black have a higher likelihood of dying and likelihood of aggressive disease. we need to know that. >> but what are some of the challenges of building a unit that is ethnically and racially diverse, with hoping to tackle some things in the future that maybe you learn to venture further into this diversity effort than ever before? >> we are just at the beginning. we would love to follow up with your office and talk about it more. we have community advocacy groups and participant advisory boards at all of the healthcare

organizations that are part of our program. they are helping us to understand what is working and what is not working. even things like parking is in close enough to the clinic and therefore is too hard for me to make the appointment. to come in and donate my samples. we have learned a lot from participant feedback. we will be happy to share with me learn more. >> having a good feedback system and adjusting all the way is important so you're doing that? >> yes. >> thank you very much mr. chairman. thank you very much for the generosity of your time. >> thank you mr. chairman i'd like to get one more shout out, diana is my cochair on the congressional study group on japan. we are trying to spend together every year. also to fred for their tireless work on the 21st century cures. they just did yeoman's work a lot of you know. want to give them one more shout out to them. my youngest daughter when she was 25 years old was diagnosed

with hodgkin's lymphoma. today she is 29 years old she's three years since chemo. the developments over the year have been phenomenal. i want to thank everyone. dr. we were happy to support the cancer moonshot with 21st- century occurs. we're looking forward to hear about progress her patient. can you tell us about the most exciting things that are being supported in the cancer moonshot? >> there is so much good stuff. it's very meaningful law for patients with cancer. i think it's going to impact them in many ways. we already talked about response maybe i'll talk about the rear tumor initiative which is very exciting. we mentioned red diseases, many rare cancers are very hard to study for some other reason. too rare to make clinical trials. nci is a good place to study

those things. we can have patience with these diseases come to the nci. this paradigm works and i think the moonshot we can build on that experience. for example, area of the disease , different mutations that activate driver protein in cancer. those patients present with childhood tumors. the nci now has the recent trial that we just presented, shown here on the left is a growth showing one of our sentai us trial in these patients. this is the patient has one deficiency. my executive summary was the tumors shrink kids feel better, drug seems safe. it appeared a few parents contacted me. my child was on this trial and the responses have been marvelous. showing us the picture of the child pretreatment on the left and after treatment on the right. you can see the windpipe,

airway is not being compressed by the tumor anymore. he not the social stress of going to school at the big lesion like that. so this is sort of a pre-cancer syndrome. this kind of study is a really great thing for the nci to take on. >> okay. we must transform the way we conduct research the way we share results on the way we get discoveries in patient care. can you discuss how nih is helping transfer infrastructures needed to help meet these goals? >> the new funding that congress has made it possible to increase the success rate for new scientists applying for their first grants. within nci we didn't have enough funds we increase that by 25%. we are simply trying to allow scientists to concentrate on science and not as much

bureaucracy to writing grants. we're testing out a seven-year reward is post a five-year reward. and were also thinking about training opportunities we are providing for young scientists. make sure they get the right skills. we really need more training and big data. >> if i may thanks for the 21st century cure next-generation research initiative which is part of this bill. we have put into place efforts to provide better chance for early-stage investigators coming to nih with the first major grant application to get funded and that have sustainable support so they know they have a career. this year we are predicting we will fund the largest number of first-time investigators ever at nih because we have made the shift in priorities. and you helped us with that. >> thank you. i was able to meet with two early scientists from early cancer research. i was impressed with the excitement of pets that passion they have for careers in

research and helping cancer patients. once again as the father of the counselor survivor it really means a lot to me. thank you for being here. >> thank you all for coming today. whenever you testify am reminded of the tremendous responsibility that you have in the organizations and things that you do. we thank you for your work to implement 21st century cures. obviously you know is appointed real pride for this committee. so thank you for that. commissioner gottlieb i wanted to ask if there's been discussions about action plan that you are released i commend you for that. i have just introduced the last few days something we are calling bio similars

competition act which is try to get some of these pay for delay agreements that are operating in the biologic similar space in the same way we have given authority to kind of place that conduct with respect to drugs and generics where it has been consequential for sure. the authorities there, people look at these agreements. they can judge whether they are fair and appropriate regarding the consumer or not. and there is tremendous savings to be had there. i think maybe the figure is, brand names occupying about a percent of the drugs produced every year but still 75% of the cost. i think that includes biologic,

bio similar distinction as well. can you just speak to whether you think it's a good idea to have this authority and i think i understand from the plan that you put forward in an almost generally, that you look for ways to cooperate with agencies like the ftc around this kind of thing. to make sure that we are getting drugs at the price point that they should be. >> i have my own equities and trying to work with them. try to see how we can facilitate their interests and bring cases related to delaying tactics that might involve rams that we can help provide information prompted to take a look whether those practices have been compare that has been a big bugaboo of mine. i will say generally speaking you like to improve safe and

effective bio similars. we want to see patients benefiting from them. culturally we like to see drugs marketed. does the fact that most bio similars that have been approved had not been marketed for various reasons. also the fact that a growing number and have been approved but the drugs are never marketed because of the changing economics of that market as well. i know we can need to keep a close eye on that because competition may be declining in the small generic drug as well. >> thank you, we will put that in the toolkit peer we want to make sure we protect consumers in that space. let me switch gears. do you, dr. collins, know you got a question earlier i believe about alzheimer's research, generally. and clinical trials associated there with.

there was an article in the new york times yesterday that pointed specifically to the challenge that is presented by trying to find sufficient people to participate in these trials. there is a lot of trials that are underway sort of on deck that would suggest the need up to 25,000 participants in these trials. some of them could be really breakthrough. i gather that this is a real problem trying to find enough participants. can you speak to what you know about that? and what can be done about it? >> give those based on comments made by dr. murray bernard was the deputy director of the national institute on aging. this is a challenge. because we are now at a point where we are trying to recruit individuals who don't yet have cognitive decline but who are high risk. by increasingly accurate means we have some using engineering

-- imaging and genetics. we need a lot of participants to do that. but now we are reaching out to people who may not be that motivated to take part in research because they're fine now. we're arguing that is the best time to intervene. but it's also easy to enroll. i will say one of the dreams i have is the program we've been talking about this morning. you have 1 million participants who are pre-consented, recontacted with research protocols. that be a fantastic group to be able to enroll participants in studies like this for, disease. whether it's diabetes, hypertension, or alzheimer's. the moment we don't have that platform, we are having a challenge trying to convince people that this is something they want to take part in. we are pulling out all the stops. again congress having made alzheimer's such a high priority for us. it is not simple. different model than what they're used too. you get approached about clinical trial when you are to have the diagnosis. here we are approaching people who don't have that diagnosis

trying to figure out how to prevent it. >> thank you very much. >> thank you mr. chairman. thank you for being here. it's a pleasure for me to be here as a physician i never thought i'd get a chance to talk to you all. i was at an event with dr. sharp and steve rosenberg recently and the work that he's done over the years i can't even express how much of an impact he has had. >> dr. collins you just at high risk. who is high risk for alzheimer's? >> anybody who gets to be 85 or 90. >> i'm saying people that don't know they have it if your mom has that your dad has it your grandma? i think that would be an area, maybe if one of your family members has that you are high risk i don't know? >> there are three ways we are currently identifying such

folks. one is you have a very strong family history almost inherited in a dominant fashion. there are those families if you're in one of those circumstances we can track the gene in the family and it wasn't long before anyone has any symptoms. another is a genetic risk factor are. if you have one copy your risk goes up to fold if you have two copies those people are very high risk. the third way is using a pet scan that picks up alloys. amyloid start depositing in your brain probably 20 years before the first cognitive decline symptoms. so people are willing to do a scan we may be able to say hey you are one of those that ought to get in a clinical trial. >> so somebody out there watching c-span right now. they are sitting there wondering if i am high risk. i understand the technical tests and things that you do.

but if you say my mom had a dime high risk? >> is indeed that is one of them. there are other ways to try to be more precise about it. if people are watching c-span and wondering if i should take part in that we just heard how difficult it is been to get the word out there. a place to go is clinical trials.gov which posts the trials on alzheimer's disease and everything else where you can find out who is doing the trial what is the enrollment criteria, you contact to learn more about it. all of this we contribute with the fda as a contributor as a partner. >> my wife just texted me she's a doctor out practicing medicine. she said can you check into the sentinel shortage. i'm not kidding. she just texted me. she does and she's practicing medicine. we have demonized the illicit forms but this is a very common anesthetic agent. apparel it is going to be a long-term backorder on fentanyl

which is going to be a huge problem. you don't need to answer the question i just want to point out what you are doing on drug shortages has real, everyday, clinical implications in the practice of medicine. she said so friend, other common paralytic agents that she uses daily in anesthesia practice are really in short supply. fentanyl is a new one on me. she just texted me on that. thank you for your work on that and your response. if you have any further comments. i have one other question for you. >> i will just say, this is a structural problem as i mentioned before. whatever is it shortage something different i grapple this impacts we are working very hard on this. >> 21st-century cure this is a proprietary question here.

diseases such as cancer which rely on diagnostics. i know yourself probably told you might ask about this. the correct diagnostic works for laboratory developed test and in vitro diagnostic. this we've had a discussion out there you all have had that and we really appreciate your input. and we have talked of laboratories and patient groups of providers and we really need to reform now. so i know you're familiar with the issue. i brought it up in the past as well as the committee's last hearing. he recently gave us a narrative on the fda provided us earlier in this year i want to get diagnostic reform done in this congress hopefully. so we are waiting for the redline of the bill from fda and i want to know if you had

any insight into that progress? >> thank you we have completed most of our work and i hope to have it for you very soon. i like to come into your office and brief you on it as well. i think we are very close. >> i just want to reiterate where very appreciative of that we are hopeful that thank you mr. chairman i yelled back. >> thank you mr. chairman. thank you all. for your service to our country. all the incredible medical innovations. i just want to thank you probably doing very very different things. in our country and yet you are going to be working on behalf of all citizens not just here but actually run the girl globe. i want ask this testimony you mentioned specific groups

research and clinical trials. i'm particularly interested in child disease and cancers. having heard from constituents that lost a child to cancer and are currently fighting the disease. we know that nih's focus on improvements for children. an area that's been lacking in the past. can you elaborate on the implementation of the recently passed childhood cancer star act which was signed into law in june. and what kind of innovation are we focused on for childhood cancer and disease? >> is important to say two things about childhood cancer. it's true we've made tremendous progress. your children are dying of cancer more than ever. in particular when were even able to cure kids of cancer we often leave them with lifelong toxicity.

one of these principle issues the star act tried to address, this is of novel therapies also the burden of survivorship some of these patients incur. the curative therapy. as has really interested in the topic. i know firsthand the pain that many of these patients go through suffering. i think we have a focus on new research efforts. by specimen acquisition getting new advices new voices make sure we are quickly advise talked about in our focus on reauthorization get that reauthorized by the end of september. one of the things that has come up in various discussion 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's been frustration in this committee about egg-based vaccines versus platform technology. if anyone would like to comment, i'm starting with you. >> i'd like to start, if i can. i appreciate the question very much. we have additional funding from the agency to try and support alternative manufacturing. we think this is the detection we want to head in where you have closed continuous manufacturing platforms particularly with recombinant technology you can plug into the platform and allow continuous manufacturing of the vaccine. if you want to modify the vaccine, you clearly plug in another cassette that codes for

a different permutation of the same vaccine. we think this is a situation for influenza available flu so you can get the strain better as well as have a platform available in the event of a pandemic flu and these same technologies continue to try to scale up manufacturing of other vaccines. in an ideal circumstance what we would have isn't mothballed vaccine that degrades over time if it's not stored correctly and takes up a lot of space and it's costly, but platforms that allow quick manufacturing of vaccines should we need it. >> if tony fauci was here, he would go into this in some detail, the idea of having to build things in eggs is so much yesterday's technology. the concrete example this past year with the flu vaccine having been surprisingly ineffective turns out the virus mutated in the process of being grown in the eggs. so therefore it turned not to be a particularly effective vaccine for something that we didn't have control over. these new platforms which allow

you to build vaccines in a much more rapid fashion and much more directed fashion, dna- based vaccines, rna-based vaccines. once you have that platform going you can very quickly adapt it to many different pathogens. that's certainly something we're working on now to develop this influenza vaccine and congress has given us additional funds so we wouldn't have to have the yearly effort to try to guess right. you'd have a vaccine that works against virtually all strains and would also be effective against that next 1918 style pandemic which we were all worried about and which is overdue. >> we did include a pan flu legislation in possible legislation passed out of this committee. i yield back. >> thank you for your work. >> the clair recognizes the governor from florida -- chair recognizes the governor from florida. >> thank you, mr. chairman. i appreciate it. thanks to the panel for their

testimony today as well. the first question i wanted to ask, we talked about alzheimer's disease, of course, and what about parkinson's? as far as we're talking maybe how do you know if you're a candidate for parkinson's disease to participate in these clinical trials if there are no symptoms, maybe early stages? if you could answer that question, sir, i'd appreciate it. >> there are parallels here that are notable. there are genetic risk factors to parkinson's disease, which is interesting, because when i was in training and i asked my professor are there any diseases that don't have genetic contributions and he said oh, yeah. everybody knows parkinson's is always totally random and sporadic. he was really wrong. if you have a gene called lurk 2, your risk of parkinsons goes up. we are beginning to therefore be able to identify people at high risk and invite them to take part in prevention trials. another big thing that's

happened in parkinson's disease in the last six months is the formation of a partnership with industry called the accelerating medicines partnership to parkinson's disease with fda a critical partner in this as well and really now figuring out how we could learn from a very large amount of data that's out there, but it hadn't been brought together what are the next generation of drug targets for parkinson's disease and how do we accelerate the process of getting there because we have treatments, but we certainly don't have things that actually prevent progression. they more treat the symptoms. we believe we could do better with that. the brain initiative, which is this very bold effort, reported by 21st century cures and the innovation fund is learning things about the wiring diagram of the brain, that it's going to be very relevant to some of the things that are being done for parkinson's disease with direct brain stimulation where you 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. as we learn the wiring diagram, we can be much more precise and

effective. >> thank you very much. anyone else want to add something with regard to that? okay. thank you. dr. gottlieb, section 308a clarifies that fda has the authority to grant emergency use authorization for animal drugs allowing the agency to approve the gmo mosquitoes, florida's zika problem. we're planning ahead. would you provide an update on the implementation of section 308a specifically with regard to the mosquitoes? >> i can get back to you with a more detailed update, congressman, but there's been some discussion about the nexus of authority with epa about some of the these products, but we did provide guidance earlier, i believe, to give direction with some of these issues, but can i get back to you. >> i appreciate that very much.

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

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

i yield back. >> the chair recognizes the gentleman from georgia, mr. carter, five minutes for questions, please. >> thank you, mr. chairman and thank all of you for implementing your efforts. i personally feel like it's some of the best legislation we passed in congress for quite a while. dr. collins, i'll start with you. i wanted to ask you particularly about one of the initiatives of the 21st century cures and that was to really review the regulations and policies with respect to research in laboratory animals. as i understand it, you're working with usda now and the fda to try to complete a review of that. i wanted to ask if you could tell us the current status of that review and when do you anticipate completion of that review? >> i appreciate the question. we are very seriously engaged in in and again 21st century cures gave us some clear guidance about what we ought to engage in. we did put out an rfi in march

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

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

how are we going to handle this? i'm going to ask you that and i'd like to ask dr. gottlieb as well. >> i'm say a word and ask dr. mulvaney to fill in more as deputy director of the program. we are very invested in looking at this in the fashion of cutting large dealing with very large data sets, putting the data into a cloud. just had a very interesting all day workshop monday on artificial intelligence and machine learning and how that can be applied to these unprecedented data sets that glean the maximum amount of information and while maintaining the confidentiality and security systems that the participants will expect about their deidentified data. maybe dr. devanei will want to say more? >> one of the data types that will be the most precision is information from health records. this is one of the largest challenges for our program. we have direct partnerships with many healthcare organizations and we're getting those data continuing from those partnerships, but we're also working on other

strategies including one in partnership with four of the largest hr vendors right now to work on making the data transmission much more seamless across provider lines and into the program when a participant authorizes it. >> dr. gottlieb, i know this is important in drug data development as well. >> i appreciate the question. i was taking a step up the continuum to try to make effective use of the data because there's so much information, how can you make effective use of the data in a way it can translate to clinical benefit in patients. i think this is where clinical trial innovation comes in where you have the ability now to effectively bias enrollment in the trials for some of the genomic information and predictive information to predict who is more or less likely to benefit from treatment and experience side effects. we can use the information in that way to structure trials and enrollment and end up with much more information about who is likely to benefit from a drug and more tools to make

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

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

dramatic and again allful you are to be congratulated -- 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 going forward with the clinical trial because that is the way information is gathered and learned. so 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, members have 10 business days to submit additional questions for the record, ask the witnesses to submit their responses in receipt of those questions. without objection the subcommittee is adjourned.

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