Transcripts For CSPAN2 Medical Product Development And Innov

Good morning. The cynic in me to help education letter pensions will please come to order. This is another bipartisan hearing which is what most of our hearings are which means senator murray and ive agreed on the subject and on the witnesses. In fact, i would say every member of this committee would probably agree on the subject and a witnesses today. Because this is hearing entitled implementation of the 21st century cures act, progress and the path forward for medical innovation to from dr. Francis collins, the director of the National Institutes of health, and dr. Scott gottlieb, the commission of food and drugs. Well have some mention as senator murphy will have a hearing next wednesday on another important part of the cures act which was the first reorganization of our Mental Health laws in a decade which senator cassidy and senator murphy played a major role in. We look forward to that. Senator murray and i will each have an Opening Statement and then well introduce our panel of witnesses. After our witnesses each senator left five minutes of questions. I expect we will have a large number of senators coming and going to this hearing. In 2007, doug oliver, who is in the front row i believe, a computer programmer, begin to have trouble seeing, and after a near accident had his drivers license taken away and was declared legally blind. The culprit was a rare form of macular degeneration. Doug moved to nashville, where his doctor at the Vanderbilt Eye Institute told him that while there were no cures, doug could Search Online for a Clinical Trial. Doug found a Regenerative Medicine Clinical Trial in florida, where doctors took cells out of the bone marrow in his hip, spun them in a centrifuge, and then injected those into his eye. Three days later he began to see. His eyesight eventually improved enough to get his drivers license back, and enough that he became an effective advocate for more support for Regenerative Medicine, which we included in the 21st century cures act. Earlier this year, doug gave me the cane hed used while he was blind. He said i dont need anymore. So i treasure it and the keep it in my office and i think it for his advocacy and we hope others have the same advantage. When Congress Passed the 21st century cures act, we hoped to unleash medical innovation and give americans more access to lifechanging treatments and cures, so more americans could experience medical miracles. It is especially appropriate to be having this hearing today because it marks the oneyear anniversary of the Senate Passing cures. When it became law last december, i said that president obama had signed a second christmas miracle. The first one in 2015 was the every Student Succeeds act, the law fixing no child left behind which came from this committee. Last year, it was the 21st century cures act, which majority leader mcconnell called the most important legislation Congress Passed last year. I have often said of the every Student Succeeds act that a law is not worth the paper its printed on if it is not implemented properly. I intend to ensure cures is also implemented properly, and this committee began our oversight hearings on cures with a hearing in october on the Electronic Health records provisions and, in addition to todays hearing, we will continue our oversight with another hearing on the Mental Health provisions next wednesday. Today we are hearing from dr. Collins and dr. Gottlieb on the provisions related to Biomedical Research. We are fortunate to have two talented leaders who know their agencies, are widely respected in congress, and capable of getting results. It doesnt always happen. Sometime ship capable leaders and they dont have any money or they dont have new authority. Sometimes your new authority, new money, and the leaders of have a clue what theyre doing. In this case weve got new authority. Weve got new money and we got to the most capable leaders we could have. So were delighted with that. While the legislation included provisions to improve Electronic Health records, resources to fight the Opioid Epidemic, and the first update to Mental Health programs in a decade, the major purpose of the legislation 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 ensuring the fda Gold Standard of safety and efficacy. It is not an overstatement to say that the 21st century cures act has the potential to affect virtually every American Family by taking advantage of breathtaking advances in Biomedical Research. Dr. Collins, at a Senate Appropriations subcommittee hearing in 2016, you have offered bold predictions for future Major Medical advances if we continue funding nih and ensure fda has the tools it needs. One prediction is that scientists will find ways to identify alzheimers before symptoms appear as well as how to slow or even prevent the disease. Alzheimers causes untold family grief and costs 259 billion a year. Another prediction is using a patients own stem cells, doctors could rebuild his or her heart. This personalized heart would make transplant waiting lists and antirejection drugs obsolete. And put doctors like former senator bill frisk out of business. Dr. Collins, you have made other predictions that are equally breathtaking. The development of an artificial pancreas for diabetes patients a zika vaccine, a universal flu vaccine and an hiv aids vaccine available within the decade. New nonaddictive pain medicines to help patients as we continue to battle the Opioid Crisis that kills 91 americans every day. You said this week and 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 of limitless opportunity to help make dr. Collins predictions a reality. Today, i want to find out find out from dr. Collins and dr. Gottlieb, those leading this charge, how implementation of the law is going. For example, cures included a surge of onetime funding of 4. 8 billion for the National Institutes of health, including money for the Precision Medicine initiative, cancer moonshot, and Brain Initiative. I know from talking with Vanderbilt University that the Precision Medicine initiative, the plan to map the genomes of 1 million volunteers to help researchers develop treatments and cures tailored to a patients genetics, environment, and lifestyle, has already begun enrolling patients less than one year after the program began. In addition to pmi, i am curious how the funding has been spent, and when and how you plan to Keep Congress informed of your results. Cures required a process to look at burdensome regulations on researchers. I would like to hear if this is helping researchers spend more of their time and money on research and less on administrative tasks. Cures also put in place policies at the fda to move safe and effective treatments and cures through the regulatory process more rapidly and at a lower cost. For example, senators burr, bennet, and hatch worked on a breakthrough provision for devices, modeled after a breakthrough provision for drugs. The first breakthrough approval was just last week for an exciting cancer diagnostic. Senators isakson and casey worked on removing red tape from the review of combination products. And i worked with senator murray to make sure fda had the authority to hire and pay scientists to keep up with the rapid rate of innovation. 21st century cures also added 30 million to support Regenerative Medicine and an accelerated pathway for these treatments at fda, so we can hear more stories like doug olivers. Fda has begun implementing these provisions and i look forward to hearing how fda and nih are working together to make sure the funding and authorities for Regenerative Medicine are helping to advance this important work, while ensuring bad actors do not take advantage of the hope of this exciting field to harm or defraud patients. It is going to be difficult to cover everything today, but i look forward to hearing about the progress being made to unleash medical innovation and bring new drugs and devices to patients. Senator murray. Thank you so much, chairman alexander and welcome to our guests today. Thank you for joining us, dr. Collins. Good to have another opportunity this week to talk to you again about the work in debt at an age. Dr. Gottlieb, welcome as well. Im looking for updates from fda. Next week marks the one year since the signing of the bipartisan 21st century cures act. And as i said at the time of the passage and i will repeat it now, as much as this law helped to build on americas tradition of leadership and lifesaving Public Health initiatives in medical innovation, cures was really first and foremost about providing hope. Hope to millions of people and families who are 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 including our guests today this crisis is something i hear about everyday. And i visited with countless communities back home 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 youre cs took an important first step and dedicated over 1 billion in new funding above and beyond the budget caps to states and communities by back against that crisis. We secured important changes to ensure this money went directly to states that need it the most. For far too long our Health System has their patients and families seeking treatment and support. That is why in cures we prioritized expanding access to quality care for Mental Illness and Substance Use disorders. We strengthened coronation between local agencies, gates in crisis intervention. So i looking forward to discussing that work further at a hearing we are next week. On todays topic im proud of her work in cures to address headon some of the hardest to treat diseases by providing nearly 4. 8 4. 8 billion in fug for the nih, including support for the beau biden cant a man shot and a Precision Medicine and Brain Initiative. All of which i would note are very important to my home state that we are spiriting much of that new research taking place as a result. Along with these investment we included provisions in the law to equip nih with the tools and authorities needed to meet the demands of Biomedical Research in the 21st century. We made it a priority to improve the inclusion of women and children and other underrepresented populations in Clinical Research so that the promise of these initiatives like Precision Medicine are extended to all patients. As part of this work i championed a provision that creates task force to better support the evaluation of drugs for pregnant and lactating women, and we addressed many of the concerns that have been Holding Back Progress in the Research Community by supporting young researchers and 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 qualified individuals who understand the latest science and technology. Im interested to get an update from you dr. Gottlieb on how you are filling the positions, especially in of the hiring freeze issue is mandated by the president. We also made sure cures give fda the authority to ensure medical devices like are safe for patients. We gave more clarity to developers of drug device combination products and codified key provisions in the Prescription Drug user fee agreement, including to ensure patients are included in the drug Development Process. Im glad we have the opportunity today to talk about the ongoing implementation of cures and i will be very focused on making sure we are committed to strong congressional oversight, rejecting this administrations efforts to roll back and undermine patient protections, and doing more to provide strong investment needed at nih and fda not just to support cures, but also to advance 21st century science and innovation. In general im glad nah and have been active and timely in the permitting the law including many provisions i just talked about. Im encouraged by these efforts. I want us to keep moving in the right direction. Dr. Collins, in light of the extremely concerning direction the Top Administration has taken when it comes to health care, especially in regards to womens health, i want to hear more about what in ages and to bolster your researchers work to further science over extreme ideology. Dr. Gottlieb come during her confirmation hearing you said the 21st century cures act is a good roadmap for what you hope to accomplish as commissioner. I would like to hear about how you fda are pushing back against this administrations the regulatory approach and making sure cures is implemented. Specifically, i will be asking more about what fda is doing to ensure the medical devices patients rely on are safe and effective. Again, both nih and fda have made important steps in implementing and beating cures deadlines this year but we have a lot more deadlines coming up in 2018 and since we know funding remains an issue under 40 hearing from each of you on how we can make sure that were truly putting patients and families first. So again 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 will hear from is dr. Francis collins. Hes been director of the National Institutes of health overseeing the work of the larger support of Biomedical Research in the world since 2009. The second witness will hear from is dr. Scott gottlieb. Is commissioner of food and drugs, ensuring our drugs and medical devices are safe and effective. He was formerly Deputy Commissioner of the same agency. He was confirmed on may ninth of this year. Welcome again to our witnesses. Dr. Collins, lets begin with you. Good morning and thank you, chairman alexander, Ranking Member murray and other distinguished committee members. It is an honor to be today with my college dr. Scott gottlieb the commission of the fda. As you one of the 20 futures act aimed to catalyze a very important goal shared by all americans, to speed the pace at which scientific discoveries are translated into lifesaving treatments and cures. We at nih actually really appreciate your leadership in passing this bipartisan act by a vote of 95 4 innocent exactly one year ago today, december 7. This enhances authorities and resources in ways that will help us to achieve this goal. Many thoughtful provisions are clear in the act such as reducing administrative burdens so our site is can do but more of the time to research, expanding our ability to award a prize for exceptionally creative ideas. And strengthening measures to protect patient privacy. In my written statement i submitted a comprehensive report on at nih has worked quickly to limit the provisions of the act. We are motivated by sense of urgency to the patients in need of breakthroughs. In my oral statement i would like to focus on the cures innovation fund. Among the vital areas of ni h supported research being accelerated by the Brain Initiative, the cancer moonshot, the Regenerative Medicine Innovation 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. In fiscal year 17 fiscal yeard our cures innovation funding with her annual appropriation to launch no less than 110 exciting new Brain Research projects pick some of these will develop detailed maps of neural circuits, others will create a synthesis of the cell types in the brain and still others will create powerful new tools to monitor and modulate brain activity. This will advance efforts to develop new ways of detecting and treating and preventing miniseries Brain Disorders including alzheimers disease