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 develo