Transcripts For CSPAN3 Medical Product Development And Innovation 20171208

Good morning. The health labor on education and pension will come to order. This is another bipartisan hearings, which means senator murray and i have agreed on the subject and the witnesses. In falkt i would say every member in the committee would probably agree on the subject and Witnesses Today because this is a hearing entitled implementation of the cures act. The path forward to hear from dr. Frances collins, the National Institutes of health and dr. Gotlieb, the commissioner of food and drugs. Senator murphy will have a hearing wednesday on another reorganization which they played a major role in. We look forward to that. Senator murray and i will each have an Opening Statement and then introduce our panel of witnesses. After, senators will have five minutes of questions. In 2007 doug oliver, who is in the front row i believe, a Computer Programmer began to have trouble, had his drivers license taken away and declared legally bliepd. It was a rare form of macular degeneration. He moved to nashville where they told him while there were no cures, he could Search Online for a trial. He found a regenerative Clinical Trial in california where they took cells out of the bone marrow in his hip, and then injected these into his eye. Three days later he began to see. His eye sight eventually improved enough to get hihis eye sight back and an effective advocate for Regenerative Medicine, done the proper way which we included in the 21st century cures act. Earlier this year he gave me the it cane he used when he was blind, said i dont need it anymore. So i treasure it and keep it in my office and thank him for his advocacy and hope others have the same advantage. When Congress Passed the 21st century cures act we hoped to give americans more access to life changing treatments and cures so more americans could experience medical miracles. Its especially approp retoot have this hearing because ritszer the one year a anniversary of passing cures. I said president obama had signed a second christmas had miracle. The first was the every Student Succeeds act, a law fixing no child left behind. Last year it was the 21st century cures act, which majority leader mcconnell called the most important legislation Congress Passed last year. Ive often said of the every stuntd succeeds act its not worth the paper printed on unless its implemented properly. We began oversight on cures with a hearing in october on Electronic Health records provisions and in addition to today pfsz hearing, well continue our oversight next wednesday on the Mental Health provisions. Were hearing about the provisions related to Biomedical Research. Were fortunate to have agencies capable of getting results. Sometimes you have capable leaders and they dont have any money or new authority. Sometimes you have new authority, new money and the leaders dont have a clue what theyre doing. In this case weve got new authority, new money and two of the most capable leaders we could have. So were delighted with that. It legislation included provisions to improve Electronic Health care records, and the first update to Mental Health programs in a decade. The major purpose was to provide Additional Resources and authorities to speed drugs and devices through the research and Development Process into Doctors Offices and patients medicine cabinets while insuringing the Gold Standard of safety and efficacy. Taking advantage of breath taking advances. At a subcommittee hearing in 2016 you offered bold predictions, you call them for future, Major Medical aed vances if we continue funding the National Institutes of health and insure they have the funds they need. One hope is to identify aulz hiem er hiemers before symptoms occur. Another prediction of yours was using a patients own stem cells doctors could rebuild his or her heart. This personalized heart would make transplant waiting lists and drugs obsolete and put doctors like former senator bill frisk out of business. Dr. Collins you have made other predictions because he was a heart transplant surgeon. You made other predictions equally breath taking. The development of an artificial pankrious. A zika vaccine, a universal flu vaccine, an hiv aids vaccine all available within a decade. New addictive pain medicines to help patients as we continue to battle the Opioid Crisis that kills 91 americans every day. You said this week in another hearing that might happen in as soon as five years. The 21st century cures law put in place policies that will fund Biomedical Research at a time to make dr. Collins predictions a reality. We want to find out from dr. Collins and gotlieb how implementation of the law is going. For example, cures included surge of onetime funding of 4. 8 billion includeling money for the Precision Medicine initiative and Brain Initiative. I know from talking to Vanderbilt University that the Precision Medicine initiative, the plan to map the genoems of one million volunteers taylored to a patients genetics has already begun inrealing patients less than a year before it began. Im curious about how funding has been spent, when and how you plan to Keep Congress informed of your results. It required a process for burdensome regulations on researchers. Id like to know if this is helping them spend more on time and research and less an administrative tasks. They put in measures to move safe and effective cures to move through the regulatory process more rapidly. For example senators burr, bennet, modelled after a breakthrough provision for drugs. The first breakthrough approval was last week for an exciting cancer diagnostic. Senators isaacson and casey worked on the remove of combination products. I worked with senator murray to make sure the fda had had had the authority to hire and pay scientists to keep up with innovation. He also added 30 million to support Regenerative Medicine and an accelerated pathway for those treatments so we can hear more stories like doug olivers. I look forward to hearing how fda and nih are working together to make sthur sure the funding and authorities for Regenerative Medicine are working to enhance this important work while insuring that bad actors dont taked a vantdage of the hope of this exciting field to harm or defraud patients. Its going to be difficult to cover everything today but i look forward to hearing about innovation and bringing new drugs and devices to patients. Senator murray. Thank you chairman alexander and to our guest today. Great to have an opportunity to talk to you again. Dr. Godlieb, im looking forward to your updates. Next week marks the one year since the signing of the bipartisan 21st century cures act and as i said at the time, as much as this law helped to build on americas leadership and life saving Public Health initiatives and innovation, cures was first and foremost about providing hope. Hope to the millions of peepl and families impacted by illness and disease. Hope for our communities suffering at the hands of the Opioid Epidemic, for example and like all of my colleagues, this crisis is something i hear about every day. And i have visited with countless communities back in my home state of washington that have been devastated by addiction. So while we know we can and must do more, im glad that cures took an important step to help fight back. And we insured money went directly to states that needed it the most. For far too long our Health System has failed families seeking treatment and support. Thats why we prioritized expanding access to quality care for Mental Illness and Substance Use disorders and strengthen determination engaged in crisis intervention. So im looking forward to discussing that work further at a haring were having next week. Im proud of our work in cures to address some of the head on disease. Including support for the bo biden cancer moon shot and the Precision Medicine and Brain Initiatives, all of which i would note are very important to my home state where were spearheading much of that research taking place as a result. We included provisions in the law to equip nih with the tools and authorities needed to heat the demands of Biomedical Research in the 21st century. We made a priority to include women, children and other underpopulated so theyre extended to all patients. As part of this work, i championed a provision that creates a task force to better support the evaluation of drugs for pregnant and lactating women and we addressed many of the concerns that had been Holding Back Progress by reducing the red tape. Cures also made sweeping reforms to fda and one of my Top Priorities was granting the agents greater hiring authority to recruit and retain quality individuals who understand the latest sients and technology and to get an update from you on how youre filling those positions in light of the hiring freeze this year mandated by the president. We also made sure they gave the authority to insure medical devices are safe for patients. And we gave more clarity to developers of drug device combination including to the drug Development Process. And i will be very focussed on making sure we are committed to strong congressional oversight rejecting this administrations efforts to role back and undermine patient protections and doing more to provide the strong investments needed, not just to support cures but also to advance 21st century science and innovation. And including many provisions i just talked about. I want us to keep moving in the right direction. And especially regards to womens health. I want to hear more about what nih is doing to bolster your researchers work to further science over extreme ideology. During your conformation hearing you said the 21st severry cures is a good road map for what you hope to accomplish as commissioner. Id like to hear about how youre pushing back against the administration and making sure cures is being faithfully implements and specifically ill be askish about what the medical devices are safe and effective. Both nih and fda have made important steps this year but we have a make sure were truly putting patients and families first. So welcome to both of you. Thank you for being here. Thank you, senator murray. Each witness will have five minutes to give his testimony. First witness well hear from is dr. Frances collins. Hes been director for the National Institutes of health, over seeing the largest supporter of Biomedical Research in the world since 2009. The second witness is dr. Scott gotlieb, insuring our drugs and medical devices are safe and effective. He was formally director of the same agency. He was confirmed on may 9 of this year. Lets begin with you. Other distinguished committee members. It is an honor to be here today with the commissioner of the fda. As you well know aimed to utilize an important goal to speed the pace at which discoveries are changed into life saving cures. By a vote exactly one year ago today. December 7th. This enhances our authorities and resources in a ways that will help us to achieve this goal. So our scientists could devote more time for research. And effect patient privacy. Ive submitted a comprehensive approach. We are motivated id like the focus on the Cures Innovation Fund. Among the vital areas of being acc accelerated by this fund on the Brain Initiative or the cancer moon shot, the Regenerative Medicine project and the Precision Medicine initiative. Lets begin with the Brain Initiative. This pioneering effort is aimed at revolutionizing our understanding of the most complex structure in the known universe, the human brain and fiscal year 2017 we launched no less than 110 exciting new brain and resufrp projects. Others will create a census and others will create powerful tools to modulate brain activity. This will advance efforts to develop new ways of protecting and preventing diseases such as skichizophreni schizophrenia, autism, traumatic brain injury. With the help of the Cures Innovation Fund, the cancer moonshot is aggressively pursuing a very ambitious goal to accelerate diagnoses, treatment and care. We must take a variety innovative steps by creating a Clinical Trials effort with a commitment to move towards data sharing. In another of these innovative moves, they recently joined with the fda and 12 Biopharmaceutical Companies to launch for accelerating cancer pact. This Public Private partnership will initially speed the development of kanlser immuno therapies that enlists the patientss own immune system. Weve seen amazing responses but we need to bring that kind of success to far more people with more types of cancer and do it quickly. The Cures Innovation Fund with the support of this congress is helping to make that happen. The act also provides support for Regenerative Medicine research. It involves the use of cells and other technology such as gene editing to repair damaged cells, tissues and organs. Theyve launched the regenerative innovation project. This recently made eight clinical search rewards covering a Broad Spectrum and provided specifically by the cures act. Including diabetes and vision disorders such as the one that aflicted doug oliver you referred to earlier. While uthers are imaimed at and were hosting a major work shop actually right now starting yesterday and going through today to explore adult stem cells. We spoke at this work shop that beginning of it yesterday mo morning. And helping us to identify the areas of greatest scientific and therapeutic promise and finally i want tale you how thrilled i am had you supported pmi while including an authorization in funding in the cures act. The center piece is the all of us and we must insure more americans in every walk of life. They must contribute health data to create a resource research. This is an ambitious goal and we know they cannot succeed on their own. So all across the nation, theyre teaming up with the veterans administration, Health Provider organizations, data center at vanderbilt and other groups to figure out the best ways to recruit participants, including those underrepresented. Also partnered with five compopies and theyre testing how wearable devices may provide easy ways for all of us volunteers to contribute data on sleep, heart rates and so on. Getting all these partners on board would have been nearly impossible can had not the cures act included ota for pmi. To carry out beta testing all of the many components and we are scheduled to launch fully in the spring of 2018. As someone who grew up in the theater family, i knew the value of a testing but when the full launch does happen you and everyone else who supported the 21st century cures act will deserve applause and if you want to find out more about that, that the website that will keep you posted. That will be not just for all of us but for the many ways cures supports the National Institutes of health or as some have called us, the National Institutes of hope as senator murray recently referred to. Let me conclude with a favorite exeritation from the british poet, peter levi. Hope in every sphere of life is a privilege that attaches to action. No action, no hope. So thank you for your action in enacting the cures act. Id be happy to take your questions. I hope sometimes during the q and a youll have a chance to comment on your belief that the other Transactions Authority would be helpful in other areas to help had us get where we all want to go. Doctor. Thanks a lot. Chairman alexander, ranking mepber murray, members of the committee, thank you for the invitation to testify at this hearing to discuss the implementation of the 21st century cures act. It set fda on a transformative path and set out to modernize how fda overseas technologies. You asked tuse advance more efficiently while maintaining our goal standard. This couldnt come at a better time across multiple fields in science where new technology is creating foundational opportunities to treat the cures disease in ways that werent possible a short time ago. Take for example our recent experience of gene therapy. Weve seen a patients own immune cells to target patients individual cancer. This form of gene therapy represent as whole new paradigm of treating cancer and how we treat tumors. This shows how a fundamental breakthrough of science can combat disease. Thats been the development of vehicles that can deliver genes more efficiently to their target inhadside the body. These are often referred to as vekters and taken the form of viruses specifically engineered for this purpose. In particular theed ad vent of aav vekter was an Inflection Point in this field. I liken it to the development of processes for making antibody drugs and making them identifiable. They represent promising field of breakthrough medicine in the 90s, but for a long time they fell short of their promise. Thats because they were made with antibodies and they were soon rejected by immune systems. And pretty soon we saw many break through drugs result. I believe were at a similar turning point when it comes to gene therapy. Over the next several years well see this approach probably curing a lot of our devastating and retractable illnesses. Were trying to capitalize on the sign and gene therapies might win fda approval from a current pipeline of 932 Development Candidate said. They estimate 45 of therapy drugs are expected to target cancer. I cant aform their estimate. But i can confirm theyre at the early stages of r as a consequence of this new technology and the benefits are likely to accelerate quickly. Yet, theres good examples on their effort to balance safety with scientific promise. Expanding our ability capitalized on breakthrough innovation. Cures provided a pathway to receive expedited review through the designation. We expanded that opportunity. Fda has considered it to be a form of gene therapy since its through a gene product delives s vekter. Serious conditions, fda clarified they would include gene therapyies this would include products bh with they lead to a modification of cells and tissues and deliver a sustainable effect in the body. If a cell has resiliency and maintains presence and delivered a sustainable therapeutic effect. It means gene therapies including next year well begin issuing a sweet of guidance documents on a development of specific therapies. We expect to lay out modern and more efficient puramteres including new clinical measures for the review of gene therapy for different high priority diseases where the platform is being targeted. We plan to focus the first guide. Documents in heme feelia. Others will address clinical areas where theres lot of interest in using the techniques such as common single gene disorders will provide pathways including potential accelerated approval end points. Its one opportunity of transforming medicine. This year they may be on track by logics and drug centers in the entire history. Well also approve the highest number of medicines ever all this year, all at one time the results of these benefits is gene therapy. But ultimately its measured in human impact. I look forward to answering your questions and i want to thank you for your support of our mission. Well now begin a round of five minute questions. I want to have several questions. So let me go through them pretty quickly. Trrs your testimony has more about it. There are a number of senators including the majority leader very interested in it and were trying to get more money and more legislation to have his continued interest. Would each of you, in the next few weeks send to me and senator murray a brief, written report sumerizing what youve done in the area of Regenerative Medicine, the kind of thing that helped doug oliver. Both how you create more hope for people like doug and what youre doing about the bad actors in the field. Id like for it to be the kind of memo i could give to other senators to show that youve taken some action the first year. Absolutely. So thank you for that. Dr. Collins, could you say in about 30 seconds Transaction Authority it allows us to move flexibly in a area of high need and where technology is advancing rapidly. It meejz we can bring partners together without taking the whole year it often takes to issue an opportunity for grants to be submitted and make the rewards. Plus gives us the flexibility when we can just cut them off without any further to do. This is something has used. Were happy to have all of this for our Precision Medicine. Where do you want it that you doint have it . We could use it in the Opioid Crisis. We have a big plan we want to move forward quickly. This would be a great advantage as one of the tools. I believe they have for the third Consecutive Year reported out 2 billion increase in nih funding. Can you sums are in less than a minute why you believe that as we appropriate money for opioids, that some of that money ought to go for research into nonaddictive pain medicines . The critical solution for the future is to have alternatives for opioids, for people who suffer from chronic pain and there are 25 million in the United States right now that have pain every day. We need have better alternatives. There are ways to get there with exciting new drug targets that have emerged working with industry, with a lot of contributions from nih. We believe we can accelerate that process to help us. And to be able then to have available for those people who have that kind of pain, answers and youre talking about using additional appropriations and private money in a Public Private partnership to accelerate that . Exactly. And we would expect businesses to put their money in this too just like we have parkinsons it takes sometimes 10 12 years to take a breath taking new medical innovation from idea to the Doctors Office. The whole goal of cures is to shorten that period and shorten that cost and still do it in a way thats safe and effective. What are you doing can you give me specific examples of how fda and nih and cms. Which has to do with funding are working together so we dont make a lot of progress in research, for example. Have it sit on the shelf while fda works on Something Else and have it sit on the shelf. Doctor. I think a lot of our ability to make the Development Process itself more efficient and lower costs is trying to develop better tools, better scientific tools to evaluate products being brought through the Development Process to ascertain their safety and effectiveness in more efficient ways, ways that are faster, that can be done at a lower cost. That is a process of developing better science, better regulatory science for evaluating technology and theres a lot of work were doing in concert, a couple of initiatives i point to you are the tissue on a chip initiative for developing better tools for toxicology and looking at issues of safety and effectiveness and the partnership for cures. Ill close by saying i think if we can can develop this better science, this is a case where we could have the best of both worlds. But also is going to give us a better measure of safety and effectiveness. The ability to determine those puramteres. Im out of time but as we continue our oversight and im sure youre going to do this. We need to think of this as a seamless process and we need to go from idea to the Doctors Office to the patient. And we need to get through the research, into the fda and to make these things work and i hope youll pay a great deal of attention to that. Thank you very much. Let me start with you. We had some deadly outbreaks of antibiotic resistant infections linked to my home state and actually across the country and i championed the provision in the 21st century cures that provided fda with additional tools to make sure the safety of the safety of reprocessed medical devices. I really appreciated the fda meeting its guideline to publish the list of devices for which the Agency Requires evidencebased cleaning procedures. Can you update on how many products have these validated cleaning protocols because of this law . Thank you, senator and ill preface by saying we think this is a very important authority and plan to make robust use of it. Are there have now been 14 products that have gone through the process outlined in this legislation. And i can get you a fully up to date number because its evolving but about 14. I really appreciate it and i want to stay updated because i think we need to respond quickly and appropriately when problems occur with these medical devices. Patients need to trust whats being used. So i appreciate that. I was very proud to champion provisions in the cures act to improve the inclusion of historically underrepresented pop yls populations as i mentioned. Into clinical research. One of the provisions was a task force to improve the evaluation of drugs for pregnant and lactating women. They are in a very difficult position often today whether its treating morning sickness or chronic condition because theres so Little Research today on how medication might impact them. And we all know if they dont get an answer from the doctor they go online and that, just to me is unacceptable in todays world. I want to ask you how do you plan to integrate the Task Force Work into the broader efforts to Impact Clinical Research . Thank you for the question. We welcomed that recommendation in the legislation and weve moved swiftly to set up this group chaired by cathy, whos a very respected natologis. Theyve mest twice and going to meet again and by may of 2018 will have a series of recommendations, which we inhad tend to take with great seriousness. As you understand very clearly this is an issue we want to be sure were carrying out the appropriate Clinical Trials to understand about efficacy and safety during pregnancy and lactation and we know those are sensitive times so getting the balance just right is what this committee is wrestling with. Theyve gotten very quickly engaged. I think youre going to see something substantial come out of this. M ier hearing from women very concerned about the direction our country is going today whether its Family Planning or allowing employers to deny acstees birth control. So theyre looking for good information and i know one of the things we did in cures was strike a balance between members of the federal government and external stake holders on the task force and today the nonfederal members have not been hired by the task force and have ntd been able to fully participate. And i want to ask how youre insuring that nongovernmental voices that congress required to be included are heard from and incorporated fully. We want those voices to be critical. They have been attending all of the meetings and are speaking. The problem is getting them officially appointed, which means going through ethics clear ngss. Im assured by february they will have gone through that process and be fully installed and not just present but devote. Thats really important. Can you keep me updated oen that . Just wanted to ask. Got a few second said left. But were living in a new digital age and some of the products are low risk. Some have a big impact on patients and their safety. People are wearing wearables. Were seeing a lot of different apps that use camera phones to diagnose diseases. These are promising and we have to make sure they work and are backed up by hard evidence. To me its less clear how fda is going to insure that the sheer quantity of products on the market are being validated and wanted to ask what ass fda is taking to make sure patients and providers can have faith in Digital Health products . And if there was a situation we received an adverse event reported, even one we excluded from regulation, that would be a reason for us to bring that product back under regulation through the provisions in cures because if some event or issue with the soft ware is leading to an adverse event, that tells us it shouldnt have been excluded in inthe first place. Okay. We want to stay updated. Thats going to take a lot of work in the future. Thank you. Thank you, senator murray. Senator roberts and senator young have deferred to senator cassidy. Senator cassdy, we have our hearing next wednesday on oversight of Mental Health law you and senator murray worked on so that will be here. Im supposed to be at the white house and so i thank you for your this is not an Appropriations Committee but its a broad topic. Dr. Collins, again, Great Respect for you and your institute. But as ive pointed out in the past we dont seem to have an nih which targets funding relative to Disease Burden. So when i look at nida, their budget only went up 2 and still remains far smaller than other instuitutes in which there is fr less mortality flowing from those diseases. So if were going to address opioids or Mental Health, it seems there has to be a greater shift in where our funding is going towards these disease conditions. In the past youve suggested that well organically grow but when i look at it, all the institutes seem to be growing at about the same pace. So i guess my question for you it doesnt seem as if nih is making it a priority if you look at the relative funding increase of those institutes. Youre a very thoughtful person. So please give me your thoughts on that. You point to a very important issue in how do we make decisions. I have to point out that its the congress that assigns a budget. Its a line item every year to each institute as the nih director i dont get to set those numbers and so we follow what the Congress Tells us ought to be the appropriation for a given year and we try to be sure when had there is a Public Health need because of course we have a big opportunity there in terms of our understanding of how the brain works, the Brain Initiative is directly relevant. The Neurology Institute has an enormous investment in pain as does the center for integrative had health. So one shouldnt look at our structure and say the money fits precisely into those buckets. We have lots of ways that we can mix i accept that. If i may because i have limited time. Youre saying if we want more money to go to the National Institute of drug addiction, we have to line itemal it in our budget . Thats the only way it happens. Flexibility does seem as though it should be flexing towards nida. Pain translates into addiction. Thats a common pathway and so it does seem we should be flexing towards them. I think thats what were trying to do is shift with the priority tubt opportunities we have more funds into that space. Because we recognize this is a Terrible Health emergency. A different topic, again trying to stay on time. There is a move afoot to suggest marijuana usage can be used in lieu of opioids. States that have legalized marijuana more liberally have low lower incidents of opioid addiction but then i read about the brain being pretty plastic up until age 25 and we all know the pot head that has arks motivational syndrome if you will. So theres been one suggestion we should make a recommendation that legalization of marijuana should be restricted to those 25 and above. I say this to get the thoughts of those such as the two of you who are so expert. Well, there is published data although its still controversial that heavy use of marijuana beginning inned a lessance does have permanent consequences, that iq points get lost in those individuals being exposed a lot to marijuana starting in ad lessance. It is also true there seems to be a statistical relationship befween the states that have legalized marijuana and reduces in opioids addiction and death. Thats not necessarily a causation. We are increasingly studying the cabbanoid receptor as a potential way we might come up with alternatives that would be effective for managing depression, pain and ang zipy. But for the two of you is it reasonable Public Policy consideration that perhaps the legal age of marijuana should be 25 and above . Were getting into difficult Public Policy. I would say the uncancerns to the developing brain would have to be strongly considered in anything that made access toed a lessance more readily available. And the brain is developing at least through age 25. You could say 22, you could say 25. My wife says its 60. And im out of time. I should yield back. I apologize. Thank you. Thank you, senator cassidy. Senator casey. Thank you very much. I do want to add my words of commonidation to you and the Ranking Member for the work you did to get this legislation passed. It is hard to believe its a year and i know we have a ways to go to with regard to implementation. But in a place and in an institution where there arent many days where you have, not just bipartisanship, but a bipartisanship that undergurds a substantial matter of Public Policy of this importantance, its especially great to continue to Work Together and celebrate this passage. But i want to talk to you on a question regarding 503 b compounders. While weve primarily been focussed in the hearing im getting new drugs to patients. Im also concerned about maintaining the supply of drugs already in the market. We know that since Hurricane Maria hit puerto rico ive heard from pennsylvania hospitals concerning shortage of iv fluid and amino acids for injection. A critical product for patients who must receive nutrients intervenusly and while puerto rico is 1500 miles away, providers in pennsylvania now face product shortages due to challenges facing Major Medical Product Manufacturing facilities on the island. The shortages are especially damaging because in the case of amino acids, the facilities on the islands were the sole source for the entire country. So when you mention a Continuous Manufacturing and how that can help reduce drug shortages, i wanted to ask you could 503 b out sourcing facilities help in the case of drug shortages . Yes, senator. We remain extremely concerned about the situation in puerto rico, specifically as it relates to the iv fluids you mentioned. We think the steps weve taken in concert with those in puerto rico will alleviate the shortage for next year but we still face another hard month getting access to adequate supply but we expect this to continue to improve going forward. It issue is they theoretically can compound this product. They would have authority to do that. The products in shortage arent just it iv solutions themselves but the actual physical plastic bags to put the solutions in. So the compounders also face a challenge getting access to the plastic bags in order to compound the product. So that has been a challenge. And whereas you would have expected the 503 b facility to supply more, i think theyve been limited in doing that. I know we have limited time but with aregard to the voucher program, we know an estimated 1 in 10 people, 2 3 of whom are children have a rare disease, according to the National Organization for rare disorders. Despite the need private companies are less likely to pursue new therapies for Rare Diseases because it requires making an investment in products that will likely will likely not recoup the high cost in marketing and distribution associated. I work with senator isakson, chairman alexander and Ranking Member murray to extend this review program at fda. This particular Program Provides important incentives to companies to invest in new therapies for rare pediatric diseases. When we last spoke, the fda was still working on implementing changes made to the program, most importantly the definition what constitutes quote rare pediatric disease unquote. Can you give us a sense of the progress made in that definition and are you meeting the statutory requirements for Companies Seeking destination for their products within 60 days . I know thats a lot. Im proud to say we are, senator and we have put forward some guidance. Theres Additional Guidance coming out implementing the prv program. We continue to look at this as an opportunity and awaiting the jail report. I would close by saying theres a lot of things we can and are doing to try to create additional efficiencies to the challenges you outline including one you took yesterday. I want to put a plug in to outline a more Efficient Development pathway for drugs targeted for very rare pediatric diseases. Thanks, doctor. Thank you, senator casey. Senator young. Doctors collins and gottlieb. I have a lot to cover and i ask you to try to be precise. You mentioned the plans you have in implementing the eureka competitions for vision and cures, the prize program. When i was in the house of representatives i put together an amendment to cures, a variant of this became the eureka prize provision. I know some others contributed to that effort as well. It was inspired by a neighbor of mine who is a latter day thomas edison. He develops medical devices in his garage. I consulted with imminent economist, healthcare provision, researchers, venture capitalists and investors and individuals developing it. The objective was to have objective rather than highly subjective criteria with respect to what those prizes are offered for. My hope, something articulated by a colleague today is we would fund it according to Disease Burden. When my colleagues consulted with the nih, they were told, no, they didnt want to be directed so it effectively killed this effort which i frankly find more meritorious than the current provision. What criteria do you or others within nih use to determine what areas youre going to be funding be due acknowledgement were funding alzheimers now. That strikes me as meritorious on first blush. What criteria do you use . I appreciate the question. Every senators place, this book published this week, will walk you through a lot of responses to that, how do we decide where the priorities are. Certainly, Disease Burden is very huge, let me be clear. At the same time, scientific opportunities dont always happen at the same pace in the same areas. When you see an opportunity particularly ripe for investment we dont want to miss that. With the prize mechanism we put out ideas for prizes for alzheimers. We have a prize right now waiting for responses and stirring up a lot of interest inanity microbial resistance coming up with a test that will tell you within four hours. So all the criteria are in that book you just held up . It will go through a lot of it and if you need more information i can supply it. Thank you for that and publishing that information. Do you include or plan on including outside stakeholders as we move forward in reviewing proposals and designing price competitions. Absolutely, yes in both case, our review panels especially High Technology efforts we populate them from the private sectors who have that expertise and private competitions we want to see what people think in all sectors about alzheimers. Do you allow in your prize competitions and have any plans to offer in kind benefits . By that i mean the ability for a garage investor or researcher to use lab space at the nih . Or to use equipment that we the taxpayers have paid for, or to offer Technical Assistance to those that need it . Yes, i think weve done actually, in a couple small project prizes were particularly asking students, undergraduated who have a great idea to apply and may not have access to the equipment we are trying to be very flexible on that. Thank you. It sounds as though the career nih folks telling members of congress their idea was not thought well enough of perhaps were on the right track. Dr. Gottlieb. Pharmaceutical Development Today frequently involves shooting in the dark, making key decisions that impact a Development Programs success with very limited data. Decisions including indication selection, dosage end points and inclusionexclusion criteria, you might say are made suboptimally, because the data that forms such decisions lies in inaccessible silence. At least inaccessible to most stakeholders. This contributes significantly to higher costs and longer timelines. The fda already has much of this data, otherwise siloed information. My question for you, has the fda taken any steps aggregating healthcare data across silos to improve the pharmaceutical Development Process, and if not, would the fda be willing to analyze the data it clektsz to provide further scientific insight back to the Research Community to accelerate and derisk biomedical innovation. I appreciate the question, senator, a complex one and id be delight to follow up on it. We will be taking more steps soon to make more data from approved applications public to being be aggregated into the sets you speak of. The data we glean across our applications to make decisions particularly around drug safety but they remain proprietary to the fda because theyre commercially confidential. We are looking how to do this without appropriating anyones intellectual property. If were making regulatory decisions on data not accessible to the public thats something we should try to address. It is a very complex area were actively working on and sufficient to say we should move in the area you suggest. Theres legal complications associated with it. Thank you. I want to congratulate you and senator murray on this one year anniversary, one more reason to be happy to be on this committee. I wonder if you can give an update on the med tech bill senator hatch and i worked on together. I appreciate the question. Ill try to be brief. We Just Announced major components implementation of some of those provisions today. We continue to move forward with this on schedule. Its a good example what were trying to do in this space, a good example what were trying to do with cures with what congress outlined and what congress set out to achieve and go beyond that. Congress set out to create a more efficient process of review or exclude certain support tools by clinicians, in the announcement we made today we will exclude certain support tools used by patients as well as long as they meet certain parameters and thats an attempt by fda to take the spirit of what congress set out to do and extend it a little bit. What sorts of things do you expect to see sooner rather than later in terms of new devices for patients . I think one area of development we havent seen as much of is in the area of support tools that could take information and help support decisions without making a decision for the clinician or patient. I think in part there is a lot of reasons we havent seen as much innovation in that space as you thought. Regulatory ambiguity played a part. We always intended to have this in space and congress emphasized that and this provides guidance in bright lines and parameters what does and doesnt cross the line and what are the obligations of sponsors including those excluded and continuing to look at those products and collect information where things can go wrong. I am hopeful we can have innovation on tools to set up making decisions based on that information. I appreciate it. I also noticed unrelated to that, the fda approved the first break through diagnostic techs. The first break through device section we wrote for this bill last year. I was interested to see at the same time that happened, there was a simultaneous decision by cms to cover the diagnostic test. Im wondering if you could share with the committee how you and cms conducted these parallel reviews and how we can see this in the future. I think you can. This was Laboratory Developed test that voluntarily came through the regulatory process and we were able to do it in six months under the break through designated product to receive approval and we work to have simultaneous coverage at the same time. Going forward, this is a panel of cancer markers that will help guide the prescription and therapy. This is a lot of innovation right now. What cms said in their policy, panels that come through the fda for voluntary approval will receive coverage. We can provide i hope a greater assurance of the effectiveness of it. I i think thats a big step forward and can tell dr. Collins agrees. Thank you. I only have a minute left. Senator rubio and i last year worked on the act for pediatric cancers and you put out guidance on developing drugs in rare pediatric diseases. Could you expand a little more about that . We did and will have more to say about this area. We will finalize parameters we had in draft form that tries to look at very rare pediatric cancers diseases, excuse me, trying to outline a more official process for developing those products. We might not have to rely as much on placebo trials and modeling assimilation for the placebo. It allows sponsors to collaborate and test multiple drugs in the same Clinical Trial. The reason why these combinations are important in this setting because its very hard to enroll patients with these Rare Diseases where you might have a dozen patients trying to enroll and looking ways not to randomize those patients to placebo can facilitate more Efficient Development. Im knew to this and didnt thank the citizens for their Great Service to this country and deeply appreciate it. Thank you, senator bennet. Senator collins. Thank you, mr. Chairman. Always glad to see you and i still claim you as my cousin regardless of the facts. I accept. Thank you. The state of maine is doing some really exciting medical research. I have visited a lot of the labs and medical institutes we have in our state. The Maine Medical Research Institute is conducting cutting edge work that researchers in maine have under way to develop medical treatments for chronic diseases. In september, one of the researchers with whom i met, dr. Oxberg and his team at Maine Medical Center received an award through the nia Regenerative Medicine innovation project, using adult stem cells, dr. Oxberg is working to develop novel therapies for chronic Kidney Disease, which is very exciting. Regenerative medicine also holds Great Potential for understanding aging and reversing diseases like macular degeneration. I understand the chairman has already introduced doug oliver. He, too, is from the state of maine and grew up only 11 miles from where i did, and he shared his compelling story about losing and regaining his sight through regenerative therapy. Mdi biological laboratory in maine is leading the development of such therapies that hold significant potential for slowing the changes that occur with aging and the diseases of aging. What especially can we do to support research into biological aging. What a great question and thank you for pointing out the number of applications to Regenerative Medicine as were gathering this morning an intensive discussion going on at a hotel in bethesda responsible shored jointly. Several hundred people in the room talking about applications from Kidney Disease to Heart Disease to aging and Rare Diseases and the whole landscape. It is enormously exciting to see how thats taking place. I said yesterday if we forecast how i think we could, we could cure a disease like cyclical secyclical sickle cell. I will say one thing otherwise i will go into a long statement. They dont completely exit the stage but still there. Theyre not good actors and making the healthy cells around them not so healthy. If we could figure out, as we have already done in mice, say, okay, youre done now, it could significantly slow down the process of aging with all that entails with aging and chronic disease. This is a pretty significant development. I think so. I remember visiting Harvard University and talking about alzheimers disease and theyre looking at turning proteins back on to restore cells that have been lost, neurons that have been lost. Enormous excitement about all those alternatives as well as identifying drug targets we didnt know about by careful biology approaches to understand how it works and those at risk for alzheimers. We have a partnership you may have heard about, accelerating partnerships and at a pace i wound have thought possible a couple years ago. About that program, which aspects of the accelerating medicine projects do you find most promising . For alzheimers, there are two areas were really pushing. One is biomarkers. We have to find out if therapy is working without waiting 10 years to find out it is protective against the development of cognitive decline and one of the proteins involved in this. The other part is the systems biology where were trying to step away from what we know already and ask the question what else is going on and how to use that in the next generation of their pu ticks and taking advantage of interventions. And to apply those earlier before people succumb to the illness. You have to start early if you want a good effect. Thank you very much. Thank you, mr. Chairman. Thank you. Senator murphy. Thank you very much. I thank you for your service and i know you were a resident and it was a sacrifice to all of you. This is a program that designs to build a diverse biomedical data seth for the medicine initiative. Im interested because one of the Community Health Centers Currently gathering data is based in middletown, connecticut. I think its great you worked in the Health Centers that particularly serve an area understood served in biomedical medicine. If you could talk about the important of having this as part of this initiative and whether there are plans to expand beyond the six Health Centers a part of this. Community Health Center in connecticut is very pleased with their partnership with you and wed like to see more get involved. In our opening remarks i put up this visual image how we are seeking applicants in this larger study of health and illness in the United States. We are inviting people to part by a variety of means. One is the Community Health center. We have a very specific goal of having at least 50 of participants being individuals underrepresent tent. They provide care to a great number of people, particularly those in lower economic situations are very excited being our partners. Were starting at the six and going to middletown to see if that is a success but this is a pilot to have many many more. Were in a beta test. Were learning everything we can to how the moving parts can Work Together. Its looking good. I want to switch topics to the establishment of the fda encology center for excellence creates a cross center team to Work Together on a variety of products to treat cancer. An update what you learned so far on this model. What do you take into account thinking about other disease specific offices. Having gone to college in middletown, i appreciate the shoutout. This is an extremely important question. The Oncology Center for excellence tried to combine it into one fine center. This was example to expedite the products and we can look at biologic business. We have challenges fully standing it up. In all candor related to fully funding it. We believe this is the future of the agency. We can do this. We really need to make it work in the oncology setting and looking forward to move this forward. Another great reminder we have to give you the funds. Back to you, dr. Collins. In another part of the cures act, we worked on a proisk ject worked on the neurological diseases act senator alexander and senator murray helps include. Talk about the having this Data Research done at nih. Its helpful to know what the prevalence is. Its challenging and expensive to do that kind of analysis and do it effectively and keep it updated. While cdc was prepared to do it they dont have the funding. Another advertisement to put the money behind the legislation we passed. Thank you, senator murphy. Senator hatch former chairman of this committee. I think youre both some of the best Public Servants ive seen in my 40 years in the United States senate. Im proud of both of you and hope you keep doing what youre doing. Im going to thank you both here today. Dr. Gottlieb, i joined senator bennet authoring the path act in the 21st century cures and eventually needing new antibiotics for multidrug resistant infections by allowing them to be studied in smaller rapid Clinical Trials and approved for the patients we need the most. Fda was tasked for guide dances fordry in pathway or elpad. Can you detail the ways in which stakeholder information is being imported for the guidance. Thank you for your question. We plan to issue the guidance in the spring and weve been meeting with stakeholders and other interested parties consistent with good guidance practices. We will have a guidance document. We have had multiple ind meetings with sponsors and believe this will be a robust vehicle for drugs targeting significant unmet medical needs. Dr. Collins i was pleased to see as a result of many 21st century cures the nih was able to start the Regenerative Medicine innovation project. It had gone to research on Rare Diseases. As you know, ive been a champion of the Rare Disease Community quite some time and applaud you for recognizing how Vital Research is. It may be truly transformational in the way we understand and treat Rare Diseases. What barriers do you foresee expanding research into this area as the regenerative innovation medicine project continues. Thank you, senator, for the kind remarks at the beginning of your time and the question. I think this is an enormously exciting time for Rare Diseases because we have tools now starting to work. Dr. Gottlieb pointed out to gene therapy, a dramatic advance in hemophilia using it with a variety vector to deliver the gene to the liver of men affected, its men, a remarkable benefit over the course of many weeks. Thats an example of whats possible. And my hope we could cure Sickle Cell Disease by directing the bone marrow and implanting them back, a transplant to yourself, a gene editing approach we find to be very exciting. This sma disease results in children that are born seem to be okay and develop paralysis and then dont live past their first year, some are making it to prekindergarten and looking pretty good by making a genetic change by virus or gene strategist. The big barrier is push the science as hard as we can and the cures bill help us by giving us that kind of inspiration. Im glad to say the barriers for regulation are not an issue working with scott who has made it a high priorities. Companies may not be so interested putting money in a very rare disease because the market is even smaller and we have a responsibility to push them as far down the road as possible. One thing i would say you did for us to make it possible to run phase three trials for Rare Diseases in their pu ticks and end caps we had not been doing and will be aggressivesively doing for this purpose. Thank you. One of the provisions for 20th century cures was to improve opportunities for american researchers. I heard there were about 150,000 in nih grants annually and have seen an improvement in this area. I would like to thank you and your staff for your work in this area. My time is up and i want to say thank you for your willingness to serve in this government and how much you really mean to People Like Us who have worked all these years in these areas. Thank you, senator. Thank you. Thank you, senator hatch, senator baldwin. Thank you. I want to add my words of congratulations to the bipartisan work you made mr. Chairman and murray and thank both of you, dr. Collins and dr. Gottlieb for your service. During the Committee Deliberations on 21st cures, i regaled the committee any number of times about being the granddaughter of nih funded scientists, how that influenced my championing strengthening our Research Enterprise and support for the nih. In my first year in the United States senate in 2013, i had a particularly powerful and inspirational meeting with a young man, a high school senior. His name is ian. And he is a bone cancer survivor from phondy lack, wisconsin. Ian told me Bone Cancer Research no doubt helped save his life. Thats why he wanted to grow up to be a scientist. To help others with his disease. He was concerned it wouldnt be possible for him to break in as a new researcher due to his awareness of nih funding cuts at the time. Ian inspired me to author the next generation researchers act, along with my colleague, senator col lines. To fight to improve nih opportunities for new and early stage researchers. I am proud to report that ian recently graduated from college, where he was helping a researcher at the university of Minnesota Study the genetics of osteosarcoma and he is now working with a scientist at Huntsman Cancer Institute in salt lake city, utah, on pediatric cancers. Dr. Collins im encouraged the nih has begun to robustly implement the next Generation Researchers Initiative to help support future scientific leaders like ian, as they discover cures for cancer and pursue other life saving research. You announced a new policy this year to issue more awards to investigators in the earliest parts of their independent research careers, to help them sustain or achieve research independence. Id like it, dr. Collins, if you can describe how each institute and center can prioritize awards for these early, midcareer investigators and how this will help stabilize opportunities for our next generation of researchers. Thank you for the question. Youre touching on a personal fashion i feel and many of the other Institute Directors do as well. We actually just published about a month ago, myself and mike lawyer and larry dayback, what this policy is and how we will implement it. We passionately agree with you the next generation is really critical for our future. It has been a tough period with the loss in purchasing power nih sustained between 2003 and 2015. Those first time early stage investigators were having a hard time getting started. We cannot lose them. We were starting to. Each one of the institutes, depending where they fit think they can find their flexibilities are freeing up dollars to make it possible for early stage investigators who otherwise just miss the pay line to get funded. We started this quite late in fiscal 17 and able to make a bunch of awards that would not have happened. Were not done yet because were looking to see if we can reach back into those reviews and have more of them. We will have a working group you would be interested in because it has graduate students and post docs and junior faculty, not just the gray emanaces but the people involved in this and see if we have the right plan to make this priority really happen. Im running out of time. I want to note, maybe you can answer for the record the effort to prioritize awards for early investigators would be about 210 million in next year and 1. 1 billion over the next five years. Does nih need Additional Resources for this policy to make sure it fulfills its promise and continue to advance all other critical nih efforts. Very quickly we are prioritizing this within existing resources but can do more of it if the resources are available. Before we go to senator warren, let me recognize senator murray. Thank you very much. I want to thank both of you for your answers. I have questions i will be submitting. We have another hearing next week on the Mental Health part of cures. Its very educational for all of us and look forward to continuing to work with you. Thank you, senator murray. Senator warren. Thank you, mr. Chairman. Weve been talking about the cures act. It has a lot of bipartisan provisions im really glad became law. During our recent hearing on gene editing we discussed new genet genetic privacy protections or research the senator and i worked on together. Massachusetts biotechs are benefitting from a provision tourist bennett and bird and senator hatch and i wrote to try to clarify the fdas authority relating to gene therapies for Rare Diseases. I could go on. Its a long list. Cures also fell short in a really big way. Thats on funding. I led democrats on this committee calling for an extra 50 billion for the nih and fda. Cures did not send one single new dollar to these agencies. Instead, it only said that future Congress Might spend about 10 of that amount on nih and fda. Im glad that so far congress has been increasing nih funding but i dont think its time for us to pat ourselves on the back yet over where we are in funding nih. Dr. Collins, let me go through this a little bit. Does the nih fund most of the grant applications it receives from scientists . No. We certainly arent able to do that. We fund about 19 of those because thats the way it comes out after we do the priority scoring to see how much money we have. Out of every 100 applications you get youre funding about 18 of them. Is that because the other 81 would have been bad investments a that would not have helped us make biomedical breakthroughs in science . We look back to history, 2001, we were funding 35, 35 because funds were more available. We looked at those to see did a grant that scored at that percentile change . The answer is no we cant tell the difference. Even though peer review is trying to draw distinctions, its very hard to do so in that top third. In other words, if we roughly, using the numbers you have here, if we doubled the number of grants, you say theres a lot of science to be had. I think there would be fantastic science. Thats powerfully important. I want to follow up with what senator baldwin made and discussion about researchers in the middle part of their career, getting that first nih grant can make or break an economic career. It can be the difference between whether the scientist stays in the fight or whether the scientist has to leave academic medicine and go somewhere else. I just want to ask more about the details here. Where are we right now in early career researchers . What percentage of the grants are they able to get . Beginning in 2008 we actually instituted a policy so applicants who came to us for the first time as a Principal Investigator got a bit of a boost. They competed against each other opposed to the most experienced ones which meant effectively they got a few extra points. Thats not good enough. We are still losing lots of those and success rates still well below what we want to think. Ultimately we think it would be more healthy if 25 of those applications were going to get funded. Thats what we do with this Research Initiative named specifically for those named in the bill. Thank you, senator baldwin for that encouragement. Weve been at about 16 , is that right . Youre saying at a minimum we ought to be boosting it at 25 . Weve looked closely to see how we can get there with this new policy, yes. I know nih has done what it can in this area. Funding is down about 15 where it was a decade ago, back when we had a 50 higher success rate for the proposals that were coming across reviewers desks. The cures act did not solve this problem and didnt even come close. Thats why today were reintroducing the national Biomedical Research act which provides 50 billion in new funding for the nih and fda. I see youre sitting up straight there, dr. Gottlieb. This legislation is cosponsored by senator sanders, casey, franken, bennett, white house, baldwin, murphy, kaine and hassan, as well as several of our democratic colleagues not on the committee. Families across this country are waiting for medical break through and researchers waiting to make those break thyougs. Its time for us to step up and put more money into nih. Thank you, mr. Chairman. Thank you, senator warren. Senator hassan. Thank you, mr. Chairman. Ill add my thanks to the Ranking Member and the cures act members. Would you please along with us thanking the two of you for your leadership and Service Please thank the women and men that work for you in your agencies for the work they do on behalf of the people of the United States. Dr. Gottlieb i wanted to start with a question or two for you, we discussed the devastating impact the Opioid Epidemic is having in New Hampshire and across the country. One of the tools is medicated resist stiff treatment that plays an Important Role in recovery along with other access to services and supports. I think both of you mentioned in your opening comments the possibility we will develop nonaddictive pain products which would help prevent addiction when for example somebody gets in an accident and is prescribed an opioid and becomes addicted. The 21st century cures act took important steps on patient focused Drug Development including requiring guidance on patient experienced data. Dr. Gottlieb, can you discuss how patient focused Drug Development initiatives could be used for developing new medication assistive treatment and development of new nonaddictive pain treatments . I appreciate the question. Bottom line they can be a very important tool and the provisions in the cures act are important how we think about the parameters we use to encourage the development products, nonaddictive to some opioid and in particular medication that can assist people that live lives of sobriety. We have committed to publish develop new guidance documents for outlining the pathway for the development of medically assistive therapy and want incentives for sponsors to develop those products and part of that making sure we have clear guidelines. As part of that were looking at alternative end points. Issues like craving. Craving is a factor that leads people to continue to use opioids. If that can be an end point in the trials to assess new products that could be an efficient pathway. That is from the points we use from parents and patients informed end points. Dr. Gottlieb, you made a response to alexander about the impact of the Transaction Authority you now have. Can you comment a little bit about how that could be helpful in the opioid space . You said it would be. How specifically would ota be useful to the nih and its work doing opioid Disorder Research . The plan we have put in place, which will be fleshed out in more detail next week in an intense twoday meeting will involve a lot of rapid action, both to come wake up new ways to treat addiction because we need more options, treat overdoses that dont respond as well as they might and new nonaddictive pain medicines, that will require partnerships between economic institutions with science skills and industries with formulation capabilities and Drug Development pathways. If we have to build that on the process of issuing drugs and contracts, it will take a long time. Other transactional authority allow us to do something in six weeks that might have taken nine months. We learned how to use this responsibly and we think this would be a great place to be available. We will be careful how to use it but it could help us a lot. Thank you. Finally, i want to talk about our new generation of scientific researchers because both senators baldwin and warren were talking about that. Cure took important steps for the next generation of researchers making it more affordable for students to pursue their graduate degrees. In september, announcing a new policy for special funding for graduate students. In recent weeks i heard from a number of graduate students, dr. Collins, who are fearful how the republican tax legislation, particularly the house version of it could impact their ability to pay back their student debts and whether it could force them to drop out of their programs. One particular provision in the house bill would require graduate waived tuition is taxed with sty pend that i heard from ph. D. Candidates say this would triple my taxable in come and increase taxes by more than 10,000 and i would have to drop out and cant afford rent and groceries while living below the poverty line. Can you comment what you think this provision would mean for the provision of young researchers and Biomedical Research . Very quickly. These are our future, graduate Students Learning to be the next generation of leaders in science across many disciplines. Certainly, i think of life science being those individuals to make the next breakthroughs. Anything that happens to discourage the best and brightest making that track or feel they cant afford to do so is something we should approach with great concern. Thank you. Thank you, senator hassan. Senator whitehouse. Thank you and i join my colleagues congratulating you and mr. Murray on your success. And hope for more to come. The requests that you go into the burden of subrepresentative monitoring. I just left Brown Research an hour ago and this is the university of Rhode Islands request. You get Brown University and university of rhode island working together on a project and the supposed obligation to monitor their subcontractors now applies to each of them. You have your eye monitoring brown and brown monitoring uri and theyre both monitoring umb, can you help reduce the bureaucratic reporting burden to your su your subrecipients . We dont enjoy dog that low risk monitoring and put together a proposal to redue the amount of low risk for recipients. Some dont meet that but many do. Could you include us in that cycle so we can be helpful giving advice and making sure that goes forward at good pace . Be happy to. It was helping you connect the device side with cure and youve done a good job. The original testimony from the fda is what you really needed was a third pathway for drug device combinations. Would you assure us if it turns out the hybrid effort that the cures act established isnt doing the job, that you will let us know so we can take a second crack at it legislatively . Absolutely. This has been challenging. We think the provisions in the cures act gives us the opportunity to address it and we will have guidance soon to articulate a better pathway. If it doesnt resolve the challenges you talked about in the past id be delight to talk about alternatives. Just so were in the loop and not wasting time if it turns out not to be productive. Dr. Collins, there have been everyday moments very significant. Back in 1876, a gentleman called his assistant watson to come into the room and in 2012 lady named Kathy Hutchinson took us out for coffee, pretty minor things except for the technology involved. Watson saying i need you to Alexander Graham bell for the telephone and miss hutchinson picked up a cup of coffee and brought to her lips through what is called a brain gape, electrodes in the brain the mere thought she could control the robotic arm. The brain therapy nih is funding is terrific. Are you fully knowledgeable of darpa to make sure everybody is pulling in the same direction . We are enormously excited and we have worked quite close widarpa for people who are paralyzed to carry out robotic control. This involved nih and darpa and nsf and the department of energy and some International Partners aims to do, to figure out how those 86 billion neurons between your ears do what they do and these complicated circuits were trying to take apart and understand better. We would like to go from where we are now, sort of an empirical effort to get this to work to know exactly where the place those electrodes to get the maximum sophistication of the move of that robotic arm. Last topic, we have done got bipartisan effort on plastic is in the ocean. Issuing a unanimous statement of concern about plastics. Plastic doesnt biodegrade and just breaks down in smaller and smaller points and microscopic creatures can consume it and it begins its track up the food chain. Never in Human History have we had to experience that kind of plastic loading into our diet. Its now found in tap water and found all over the place. It will obviously be in our food. Is this something the National Institutes of health should do basic research on so if this turns out to be a problem, certainly a new experience for human kind to digest and process that kind of microscopic plastic in the food chain, that youre alert to it . Absolutely. The National Institute of environMental Health science, one of our 27 institutes, located in north carolina, is already looking at this. I can give you summary where we stand in terms of the research being conducted. Id appreciate that. We can do that offline and through your staff. Thank you. Thank you, chairman. Thank you, senator whitehouse. Thank you both for being here today. I hope youll follow up on Regenerative Medicine in the next few weeks i can give to senator collins and other senators interested. You heard that interest from other senators today and you have quite a story to tell, i would think. And we look towards the second anniversary of the cures act in a year especially taking that 10 or 12 years between idea and something in the medicine cabinet, Doctors Office, how were compressing that and setting priorities, and fda and nih and cms are working to do that. I salute you for the steps youve already taken. That would be a very promising way to do things. Really, you have to do that more than we. We can do some things in legislation but we really cant do as well. We could order you to do it, that wouldnt make you do it. You have to do what you already started to do, Work Together to speed that up and still do it in a way that provides safety and efficacy without getting you in the middle, dr. Collins, i sometimes worry the messages coming out of washington are heard by the graduate students and the researchers across the country and theyre worried were not funding their work if the president s budget, for example, says one thing and everybody pays a lot of attention to it and that really doesnt happen. Nobody knows it doesnt happen. I remember in president obamas last year he actually reduced discretionary funding for the National Institutes of health and recommended a big increase in mandatory funding for the National Institutes of health. This isnt too hard for people to understand. This is the authorizing committee. We dont appropriate money usually. We cant appropriate money. We have another committee senator murray is actually the Ranking Member of and senator blount the chairman of, thats the money committee. For us to appropriate 50 billion for new money for the National Institutes of health is a wonderful aspiration but thats not what we do. We decide for example whether dr. Gottlieb should have a new break through path for medical devices. If the Appropriations Committee were to decide dr. Gottlieb should have break through medical devices wed be very upset because wed say thats our job. If we were to try to appropriate another 50 billion for you the Appropriations Committee would be very upset because theyd say, thats our job. Let me ask you this. We have discretionary fund, the Appropriations Committee and mandatory funding this committee could do. Whats the size of the nih budget this year . Whats your total funding . 34 billion . How much is Biomedical Research . Virtually all of it. To be clear, more than 80 of that goes out to all those institutions that do that research. How much of that 34 billion is mandatory funding . I think theres 150,000 in there. Almost all of it is money 150 million. Through the subcommittee senator blount and senator murray do. Two years ago, if im correct, senator blount and murray recommended, Congress Approved a 2 billion increase in funding for nih. Yes. Last year, they recommended and congress agreed, the president signed another 2 billion increase for nih. Thats correct. If im not correct i believe the committee has recommended another 2 billion. For the third year in a row. Congress hasnt acted. The way we normally do this once we put something in the budget we count it for 10 years. The first year is 2 billion, 20 billion over 10 years, the third, three billion, third one if we do it this year, another 20 billion over 10 years. Thats not guaranteed but thats usually what we do. We put it in the base and expect to do it again with the priorities we have in this committee and Appropriations Committee, we expect that to be the case. We did something very unusual in the cures bill, took 4. 8 billion and created a hybrid funding creating consternation with the house of representatives and Appropriations Committees and did specific funding with cancer medicine and the moon shot and brain initiate dif and other things. The chances of that happening again are close to zero. The real money supposed to go to National Institutes of health and nih comes through the Appropriations Committee. Theyve been doing a tremendous job over the last four years. I say that, im a member of that myself. I give them tremendous credit for squeezing out the budget. Nothing wrong with aspiring for more or looking back the last 15 years to say if we had continued to go up it would be even higher today. Its important for people across the country this congress in a bipartisan way has been extraordinarily committed to significant increases in funding to the National Institutes of health and it has made a difference. Senator, i appreciate you taking the time to explain all that. I think there are people watching wondering how these decisions get made and where we are. Certainly, there have been a lot of anxieties in the Biomedical Research community particularly young investigators, whats going on and what is the likelihood of the pathway. You pointed out there is a great deal of reason to be optimistic, first of all because the medicine is exciting and promising and it has been on the upward trajectory with the 2 billion increase happening and significant promise it to happen for the third year and if that were the case, that would be about a 25 increase, which is a very encouraging sign. Im grateful where in this era so few things seem to be bipartisan, medical research is and much credit to you and your colleagues for keeping that alive. I will ask one last question of dr. Gottlieb with all this excitement and breathtaking advances, as you talk to high school or College Students, do you see any increase in biology . I dont talk to high school and College Students that much. I will tell you that my girls are 8 and 4 i think there is an increasing excitement among those training in medicine now in what the future holds. I will affirm that. I have a lot of contact with medical residents and i see excitement they will cover over the course of their medical career i couldnt accomplish over my medical career. Dr. Collins. Certainly, when i go out to university and give a faulk and meet with students give a talk and i always do that. Thats the best part of academics saying i want to be with the graduate students and post docs because i want to hear from them. I have heard a lot of anxiety in the last couple of years and im trying to reassure people things are looking much brighter than they think they are. That sense of trouble is probably beyond what it looking brighter. But, boy, do i hear excitement about the scientific opportunities. If you can quickly switch to that topic, then the spark goes into that conversation and the eyes light up. And this graduate student tells you the things they can do. If anybody really wants to have a great time in science, they should come and join us. I hope ian, will join us. The hearing record will remain open for ten days. Members may submit Additional Information for that record if they would like. Our committee will meet again next week on tuesday, december 12th, 10 00 a. M. Entitled the cost of prescription drugs and sciences, engineering and medicines report. This is the third in our hearing on drug prices, which has been requested by both democratic and republican members of our committee. And then next wednesday we have our oversight hearing on the Mental Health aspects of the cures act. Thank you for being here today. The committee will stand adjourned. Cspans washington journal live every day with news and policy issues that impact you. Coming up friday morning, politico budget and appropriations reporter sarah ferrous will join us to discuss ongoing negotiations ahead of the government funding deadline and possible shutdown. Be sure to watch live at 7 00 eastern friday morning. Join the discussion. Friday on cspan, at 8 00 eastern, this Weeks Supreme Court oral argument in the case of the Colorado Civil Rights Commission on whether a baker can refuse to make a wedding cake for a same sex couple based on religious believes. And then at 9 30 p. M. An interview with the counselor to the president , kellyanne conway. Im not hear to think about myself. Im here for something so much bigger than me. Ive often said publicly and certainly privately that there are only two people who work here who were elected to anything. And their names are donald j. Trump and michael r. Pence. 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