Transcripts For CSPAN3 Medical Product Development And Innov

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 blind. 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 florida, where doctors 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 his drivers license back and that he became an effective advocate for more support for Regenerative Medicine done the proper way, which we included in the 21st century cures act. Earlier this year he gave me the 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 appropriate to be having this hearing today because it marks the one Year Anniversary of the Senate Passing cures. When it became law last december, 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 student suceeds act, that the law is not worth the paper its printed on the its not implemented properly. We began oversight on cures with a hearing in october on Electronic Health records provisions and in addition to todays hearing, well continue our oversight next wednesday on the Mental Health provisions. Today were hearing from dr. Collins and dr. Gottlieb on 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. The legislation included provisions to improve Electronic Healthcare records, resources to fight the Opioid Epidemic 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 ensuring the fda Gold Standard of safety and efficacy. Its not an overstatement to say that the 21st century cures act has the potential to affect virtually every American Family by taking advantage of breath taking advances. At a subcommittee hearing in 2016 you offered bold predictions, you call them for future Major Medical advances if we continue funding the National Institutes of health and ensure that the fda has the tools it needs. One production 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 cost 259 billion a year. 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 antirejection 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 pran pancreas for diabetes patients. 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 for the nih including money for the Precision Medicine initiative and Brain Initiative. Vanderbilt university that the Precision Medicine initiative, the plan to map the genomes of 1 million volunteers tailored to a patients genetics has already gun enrolling patients less than a year after 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 on research and less on administrative tasks. Cure has 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, bennett, hatch, worked on a breakthrough provision for devices, modeled 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 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 sure the funding and authorities for regenre ty medicine are helping to advance this important work, while ensuring that bad actors dont take advantage 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 welcome to our guest today. Thank you for joining us, dr. Collins. Great to have an opportunity to talk to you again. Dr. Gottlieb, welcome to you as well. Im looking forward to your 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, 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 people 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 secured important changes to ensure this money went directly to the states that need 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 coordination between local agencies engaged in crisis intervention. So im looking forward to discussing that work further at a hearing were having next week. Im proud of our work in cures to address head on some of the hardest to treat diseases by providing 4 billion in funding for the nih including support for the beau 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 it a priority to include the inclusion of women and children and over underrepresented populations. 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 science and technology. And im very interested to get an update from you on how youre filling those positions, especially in light of the hiring freeze that this year was mandated by the president. We also made sure cures gave the authority to insure medical devices are safe for patients. And we gave more clarity to developers of drug device combination projects and codified key provisions 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 focussed 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 the strong investments needed, not just to support cures but also to advance 21st century science and innovation. In general im glad that nih an in implementing many of the provisions i 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 century 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 administrations deregulatory approach and making sure cures is being favorite ll lly faithfully implemented. Specifically ill be asking about what the medical devices are safe and effective. Both nih and fda have made important steps this year but we have a lot more deadlines coming up in 2018. Since we know funding remains an issue, i look forward to hearing from each of you to 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 gottlieb, hes commissioner of food and drugs, ensuring our drugs and medical devices are safe and effective. He was formerly Deputy Director of the same agency. He was confirmed on may 9 of this year. Welcome to your witnesses. Dr. Collins, lets begin with you. Good morning. Thank you. It is an honor to be here today with the commissioner of the fda. As you well know aimed to cat liez a very important goal shared by all americans to speed the pace at which discoveries are changed into life saving cures. We had nih greatly appreciate your leadership in passing this bipartisan act by a vote of 954 in the senate exactly one year ago today. This enhances our authorities and resources in a ways that will help us to achieve this goal. Many thoughtful provisions are included in the act such as reducing administrative burdens, expanding our ability to award prizes for exceptionally Creative Ideas and strengthening measures to protect patient privacy. Ive submitted a comprehensive report on how nih has worked quickly to implement the provisions of the act. We are motivated by a sense of urgency. Id like to focus on the Cures Innovation Fund. Among the vital areas of being 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 Research projects. Some of these will develop detailed maps of neural circuits. Others will create a census and others will create powerful tools to modulate brain activity. This will advance efforts to develop new ways of detecting, preventing and treating diseases such as 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 network with a commitment to data sharing and to move Cancer Treatment programs forward more rapidly. 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 cancer immune 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 Research awards covering a Broad Spectrum of science and technology and going well beyond the science provided by the cures act. Including diabetes and vision disorders such as the one that afflicted doug oliver as you alluded to earlier. 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 morning. This conference will inform our future Research Directions by helping us to identify the areas of greatest scientific and therapeutic promise. Finally i want to tell you how thrilled i am that you supported pmi by including an authorization and funding in the cures act. The center piece is the all of us research program, which will enroll 1 million or more americans from every walk of life. These volunteers will contribute their health data many many ways over many years 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 companies to create a participant technology center. Our partners are testing how wearable devices may provide easy ways for all of us volunteers to contribute data on physical activity, 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. Making it possible to move forward with unprecedented speed 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 dress rehearsal before the curtain goes up. Thats what a beta test is. Weve enrolled more than 10,000 people 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 Opening Statement<\/a> 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<\/a> began to have trouble, had his drivers license taken away and declared legally blind. 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<\/a> for a trial. He found a regenerative Clinical Trial<\/a> in florida, where doctors 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 his drivers license back and that he became an effective advocate for more support for Regenerative Medicine<\/a> done the proper way, which we included in the 21st century cures act. Earlier this year he gave me the 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<\/a> 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 appropriate to be having this hearing today because it marks the one Year Anniversary<\/a> of the Senate Passing<\/a> cures. When it became law last december, i said president obama had signed a second christmas had miracle. The first was the every Student Succeeds<\/a> 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<\/a> last year. Ive often said of the every student suceeds act, that the law is not worth the paper its printed on the its not implemented properly. We began oversight on cures with a hearing in october on Electronic Health<\/a> records provisions and in addition to todays hearing, well continue our oversight next wednesday on the Mental Health<\/a> provisions. Today were hearing from dr. Collins and dr. Gottlieb on the provisions related to Biomedical Research<\/a>. 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. The legislation included provisions to improve Electronic Health<\/a>care records, resources to fight the Opioid Epidemic<\/a> and the first update to Mental Health<\/a> programs in a decade. The major purpose was to provide Additional Resources<\/a> and authorities to speed drugs and devices through the research and Development Process<\/a> into Doctors Office<\/a>s and patients medicine cabinets while ensuring the fda Gold Standard<\/a> of safety and efficacy. Its not an overstatement to say that the 21st century cures act has the potential to affect virtually every American Family<\/a> by taking advantage of breath taking advances. At a subcommittee hearing in 2016 you offered bold predictions, you call them for future Major Medical<\/a> advances if we continue funding the National Institutes<\/a> of health and ensure that the fda has the tools it needs. One production 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 cost 259 billion a year. 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 antirejection 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 pran pancreas for diabetes patients. 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<\/a> 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<\/a> 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 for the nih including money for the Precision Medicine<\/a> initiative and Brain Initiative<\/a>. Vanderbilt university that the Precision Medicine<\/a> initiative, the plan to map the genomes of 1 million volunteers tailored to a patients genetics has already gun enrolling patients less than a year after it began. Im curious about how funding has been spent, when and how you plan to Keep Congress<\/a> 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 on research and less on administrative tasks. Cure has 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, bennett, hatch, worked on a breakthrough provision for devices, modeled 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 the authority to hire and pay scientists to keep up with innovation. He also added 30 million to support Regenerative Medicine<\/a> 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 sure the funding and authorities for regenre ty medicine are helping to advance this important work, while ensuring that bad actors dont take advantage 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 welcome to our guest today. Thank you for joining us, dr. Collins. Great to have an opportunity to talk to you again. Dr. Gottlieb, welcome to you as well. Im looking forward to your 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, as much as this law helped to build on americas leadership and life saving Public Health<\/a> initiatives and innovation, cures was first and foremost about providing hope. Hope to the millions of people and families impacted by illness and disease. Hope for our communities suffering at the hands of the Opioid Epidemic<\/a>, 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 secured important changes to ensure this money went directly to the states that need it the most. For far too long our Health System<\/a> has failed families seeking treatment and support. Thats why we prioritized expanding access to quality care for Mental Illness<\/a> and Substance Use<\/a> disorders and strengthen coordination between local agencies engaged in crisis intervention. So im looking forward to discussing that work further at a hearing were having next week. Im proud of our work in cures to address head on some of the hardest to treat diseases by providing 4 billion in funding for the nih including support for the beau biden cancer moon shot and the Precision Medicine<\/a> and Brain Initiative<\/a>s, 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<\/a> in the 21st century. We made it a priority to include the inclusion of women and children and over underrepresented populations. 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<\/a> by reducing the red tape. Cures also made sweeping reforms to fda and one of my Top Priorities<\/a> was granting the agents greater hiring authority to recruit and retain quality individuals who understand the latest science and technology. And im very interested to get an update from you on how youre filling those positions, especially in light of the hiring freeze that this year was mandated by the president. We also made sure cures gave the authority to insure medical devices are safe for patients. And we gave more clarity to developers of drug device combination projects and codified key provisions including to ensure patients are included in the drug Development Process<\/a>. Im glad we have the opportunity today to talk about the ongoing implementation of cures and i will be very focussed 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 the strong investments needed, not just to support cures but also to advance 21st century science and innovation. In general im glad that nih an in implementing many of the provisions i 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 century 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 administrations deregulatory approach and making sure cures is being favorite ll lly faithfully implemented. Specifically ill be asking about what the medical devices are safe and effective. Both nih and fda have made important steps this year but we have a lot more deadlines coming up in 2018. Since we know funding remains an issue, i look forward to hearing from each of you to 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<\/a> of health, over seeing the largest supporter of Biomedical Research<\/a> in the world since 2009. The second witness is dr. Scott gottlieb, hes commissioner of food and drugs, ensuring our drugs and medical devices are safe and effective. He was formerly Deputy Director<\/a> of the same agency. He was confirmed on may 9 of this year. Welcome to your witnesses. Dr. Collins, lets begin with you. Good morning. Thank you. It is an honor to be here today with the commissioner of the fda. As you well know aimed to cat liez a very important goal shared by all americans to speed the pace at which discoveries are changed into life saving cures. We had nih greatly appreciate your leadership in passing this bipartisan act by a vote of 954 in the senate exactly one year ago today. This enhances our authorities and resources in a ways that will help us to achieve this goal. Many thoughtful provisions are included in the act such as reducing administrative burdens, expanding our ability to award prizes for exceptionally Creative Ideas<\/a> and strengthening measures to protect patient privacy. Ive submitted a comprehensive report on how nih has worked quickly to implement the provisions of the act. We are motivated by a sense of urgency. Id like to focus on the Cures Innovation Fund<\/a>. Among the vital areas of being accelerated by this fund on the Brain Initiative<\/a> or the cancer moon shot, the Regenerative Medicine<\/a> project and the Precision Medicine<\/a> initiative. Lets begin with the Brain Initiative<\/a>. 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 Research<\/a> projects. Some of these will develop detailed maps of neural circuits. Others will create a census and others will create powerful tools to modulate brain activity. This will advance efforts to develop new ways of detecting, preventing and treating diseases such as schizophrenia, autism, traumatic brain injury. With the help of the Cures Innovation Fund<\/a>, 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 Trial<\/a>s network with a commitment to data sharing and to move Cancer Treatment<\/a> programs forward more rapidly. In another of these innovative moves, they recently joined with the fda and 12 Biopharmaceutical Companies<\/a> to launch for accelerating cancer pact. This Public Private<\/a> partnership will initially speed the development of cancer immune 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<\/a> with the support of this congress is helping to make that happen. The act also provides support for Regenerative Medicine<\/a> 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<\/a>. This recently made eight Clinical Research<\/a> awards covering a Broad Spectrum<\/a> of science and technology and going well beyond the science provided by the cures act. Including diabetes and vision disorders such as the one that afflicted doug oliver as you alluded to earlier. 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 morning. This conference will inform our future Research Directions<\/a> by helping us to identify the areas of greatest scientific and therapeutic promise. Finally i want to tell you how thrilled i am that you supported pmi by including an authorization and funding in the cures act. The center piece is the all of us research program, which will enroll 1 million or more americans from every walk of life. These volunteers will contribute their health data many many ways over many years 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<\/a> organizations, data center at vanderbilt and other groups to figure out the best ways to recruit participants, including those underrepresented. Also partnered with five companies to create a participant technology center. Our partners are testing how wearable devices may provide easy ways for all of us volunteers to contribute data on physical activity, 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. Making it possible to move forward with unprecedented speed 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 dress rehearsal before the curtain goes up. Thats what a beta test is. Weve enrolled more than 10,000 people 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<\/a> of health or as some have called us, the National Institutes<\/a> of hope as senator murray recently referred to. Let me conclude with a favorite exhortation 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<\/a> would be helpful in other areas to help had us get where we all want to go. Dr. Got league. Thanks a lot. Chairman alexander, Ranking Member<\/a> 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 oversees break through technologies. You asked us to advance more efficiently while maintaining our goal standard. This couldnt come at a better time across multiple fields in science we stand at an Inflection Point<\/a> in medicine where new technology is creating foundational opportunities to treat and cure 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 experience shows how a single, fundamental break through in science can open up a whole new way of combatting disease. Thats been the development of vehicles that can deliver genes more efficiently to their target inside the body. These are often referred to as vectors and theyve taken the form of viruses that are specifically engineered for this purpose. In particular the advent of an aav vector was an Inflection Point<\/a> in this field. I liken it to the development of processes for making antibody drugs and making them nearly identical to fully human cells they were mimicking. 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. A whole new field of medicine grew up very fast. 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 intractable illnesses. At fda were focused on establishing the right policy framework to capitalize on this scientific opening. Researchers estimate that about 40 gene therapies might win approval by the end of 2022. They estimate 45 of therapy drugs are expected to target cancer. I cant affirm their estimate. But i can confirm theyre at the early stages of a transformation in medical treatment as a consequence thof new teof this 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 extended that opportunity. Fda has considered it to be a form of gene therapy since its through a gene product delivers by a vector. Fda clarified that Regenerative Medicine<\/a> therapies would include gene therapies 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 gene therapy alters tissue or if a cell has resiliency and maintains presence and delivered a sustainable therapeutic effect. It means gene therapies including cart may be eligible for the designation. Next year well begin issuing a suite of guidance documents on a development of specific therapies. We expect to lay out modern and more efficient pramts parameters 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. Well provide innovators with advice and development pathways. Approval end points. Its one opportunity of transforming medicine. This year they may be on track to approve the highest number of novel medicines in the entire history of the fda. Well also approve the highest number of medicines ever all this year, all at one time the results of these benefits is gene therapy. The result of these benefits is measured in product approvals. 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 have several questions. So let me go through them pretty quickly. Both of you have talked about Regenerative Medicine<\/a>. 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 summari summarizing what youve done in the area of Regenerative Medicine<\/a>, 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 or 45 seconds what you mean when you say other Transaction Authority<\/a> has been helpful to you . It allows us to move flexibly in an area of high need and where technology is advancing rapidly. It means 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 to decide when a particular partner is not meeting milestones and we can just cut them off without any further to do. Were delighted to have that for our common fund. Where do you want it that you dont have it . We could use it in the Opioid Crisis<\/a>. 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<\/a> reported out 2 billion increase in nih funding. Can you summarize 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<\/a> 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<\/a> partnership to accelerate that . Exactly. And we would expect businesses to put their money in this too just like we have with other partnerships. It takes sometimes 10 12 years to take a breath taking new medical innovation from idea to the Doctors Office<\/a>. 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<\/a> and have it sit on the shelf. I think a lot of our ability to make the Development Process<\/a> itself more efficient and lower costs is trying to develop better tools, better scientific tools to evaluate products being brought through the Development Process<\/a> 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 parameters. 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<\/a> 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<\/a> 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. 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 populations as i mentioned. Into Clinical Research<\/a>. 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<\/a> 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<\/a> into the broader efforts to impact Clinical Research<\/a> . 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 neo theytologina. 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 Trial<\/a>s to understand about efficacy and safety during pregnancy and lactation and we know those are neo they theyve gotten very quickly engaged. I think youre going to see something substantial come out of this. Im hearing from a lot of women who are very concerned about the direction our country is going today whether its Family Planning<\/a> or allowing employers to deny access to 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 not 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 are probably the most critical parts of this. They have been attended all of the meetings. The problem is getting them officially appointed, which means going through the ethics clearances. Im assured by february they will have gone through that process and be fully installed and not just be present, but also to vote. Thats really important. Can you keep me updated on 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. I know the agency is working to speed that up right now. 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 you what actions the fda is making sure patients and providers can put their faith in dibgital health products. 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 adverse event or some issue with the software itself is leading to an adverse event, that tells us it shouldnt have been excluded in the 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 cassidy, we have our hearing next wednesday on oversight of Mental Health<\/a> law that 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<\/a> but its a broad topic. Dr. Collins, again, Great Respect<\/a> 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<\/a>. So when i look at nida, their budget only went up 2 and still remains far smaller than other institutes in which there is far less mortality flowing from those diseases. So if were going to address opioids or Mental Health<\/a>, 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. Senator, you point to a very important issue about 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 of those institutes as the nih director, i dont get to set those numbers. And so we follow what the Congress Tells<\/a> us ought to be the appropriation for a given year. And then we work with great flexibility to try to be sure that when there is a Public Health<\/a> need, as there is now for instance, with od pioids, which think youre referring to, because we have a big opportunity in terms of our understanding of how the brain works, the Brain Initiative<\/a> is relevant here. The Neurology Institute<\/a> has an investment in understanding pain. One shouldnt look at our organizational structure and say that the money actually 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. So youre saying that if we want more money to go to the National Institute<\/a> of drug addiction, we need to line item it in our budget. Thats the only way it happens. That said, the flexibility does seem it should be flexing toward things like nida. Pain is important, but it translates to addiction. Thats a common pathway. It seems as if we should be flexing towards them. Is that where the flexing is taking place. Were trying to shift the priority opportunities we have more funds into that space. We recognize this is a terrible Public Health<\/a> emergency. Let me ask on this, a different topic, trying to stay on time, there is a move afoot to suggest that marijuana usage can be used in lieu of opioids, that states that have legalized marijuana more liberally have lower incidents of opioid addiction. Thats right. But then, i read about the brain being pretty plastic up until age 25, and so, and we all know the pothead, the kid that has amotivational syndrome, if you will. So theres been at least one suggestion that we should make a recommendation that legalization of marijuana should be restricted to those 25 and above. And i say this not because im an expert but to get the thoughts of those such as the two of you who are so expert. Well, there is publishedidaty, although its still controversial, that heavy use of marijuana beginning in adolescence does have permanent consequences, that iq points get lost in those individuals who have been exposed a lot to marijuana starting in adolescence. It is also true, as you said, there seems to be a statistical relationship between the states that have legalized marijuana and a reduced incidence of opioid overdoses and deaths, but one has to be careful there. Thats a correlation, and not necessarily a causation. I would not want to leap to that. I would just go, though, to the point that we are increasingly studying the can bunoid receptor as a way to come up with alternatives for managing pain and depression and anxiety, not marijuana isself. For the two of you, is it Public Policy<\/a> that perhaps the legal age for marijuana for those states legalizing should be 25 and above. Were getting into difficult Public Policy<\/a> territory. I will simply say the concerns about marijuana exposure to the developing brain would have to be strongly considered in anything that made access to adolescences considered. And the brain is developing through 25 . You could say 22, 25. My wife says it 60, and dr. Gottlieb, im out of time. I should yield back. I apologize. Thank you. Thank you, senator cassidy. Senator casey. Mr. Chairman, thank you very much. I do want to add my words of commendation for you and the Ranking Member<\/a> for the work you did to get this legislation passed. Its hard to believe its a year. I know we have a ways to go with regard to implementation, but in a place and an institution where there arent many days where you have not just bipartisanship but a bipartisanship that undergirds a substantial matter of Public Policy<\/a> of this importance. Its especially great to be able to celebrate this passage and to continue to Work Together<\/a>. Dr. Gottlieb, i wanted to start with you on a question regarding 503b compounders. While weve primarily been focused in the hearing on getting new drugs to patients, im also concerned about maintaining the supply of drugs already in the market. We know that since Hurricane Maria<\/a> hit puerto rico ive heard from pennsylvania hospitals regarding shortage of iv fluid and amino acids for injection. A critical product for patients who must receive nutrients intravenously. And while puerto rico is 1,500 miles away, providers in pennsylvania now face product shortages. Due to challenges facing Major Medical<\/a> Product Manufacturing<\/a> facilities located on the island. The shortages are especially damaging because in the case of the iv fluid and amino acids, the facilities on the island were the sole source of the product for the entire country. So when you mention a Continuous Manufacturing<\/a> and how that can help reduce drug shortages, i wanted to ask you, could 503b outsourcing facilities also help in the case of drug shortages . The short answer is yes, senator. We remain extremely concerned about the shortage situation in puerto rico, particularly as it relates to the iv fluids you referenced and the amino acids. We think that the steps we have taken in concert with the manufacturers and authorities in pk prk was thought to alleviate the shortage situation next year, but we still have to get through december and we still face another hard month where theres challenges getting access to adequate supply. We expect this to continue to improve Going Forward<\/a>. The issue with the 503b compounders, they can compound this product, they would have the authority to do that. One issue is the products in shortage arent just the iv fluids themselves but actual physical bags to put the solutions in. So the 503b compounders also face a challenge getting access to the plastic bags they need in order to compound the prukd, because those were also manufactured in puerto rico. That has been a challenge, whereas you would have expected the facilities to step in to supply more, they have been limited in their ability to do more. I wanted to ask you as well, and we have limited time, but with regard to the pediatric priority review voucher program, we know that an estimated 1 in 10 people in the United States<\/a>, two thirds of whom are children, have a rare disease. According to the National Organization<\/a> for rare disorders. Despite the need, private companies are less likely to pursue new therapies for Rare Diseases<\/a> because it requires making an investment in products that will likely not recoup the high costs associated with their research, development, marketing, and distribution. As you will remember, i worked with senator isakson, chairman alexander and Ranking Member<\/a> murray to extend this review program at fda, and this particular Program Provides<\/a> important incentives to companies to invest in new therapies for pediatric diseases. When we last spoke, the fda was still working on implementing changes made to the program, most importantly the definition of what constitutes, quote, rare pediatric disease, unquote. Can you give us a sense of the progress that you have made on the update of that definition . And are you meeting the statutory requirements to respond to requests for Companies Seeking<\/a> designation 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. I think theres some Additional Guidance<\/a> coming out on implementing the program. We continue to look at this as a potential opportunity. Were awaiting the jo report as are others to better evaluate these. I will close by saying i think theres a lot of other things we can and are doing to try to create additional efisaenlss to address some of the challenges you outlined with respect to pediatric Drug Development<\/a>, including one we took yesterday. I want to put a plug in for policy we put out yesterday trying to outline a more efficient clinical pathway for drugs targeted to very rare pediatric diseases. Thanks. Thank you, senator casey. Senator young. Doctors collins and gottlieb, i have a lot to cover so i ask you try to be concise in these questions. Dr. Collins, you mentioned in your testimony the plans you have in implementing the eureka provisions 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 economists, health care professionals, researchers, venture capitalists, angel investors, and all sorts of individuals in developing my form of prize, and the objective was to insure that we have objective rather than highly subjective criteria with respect to what those prizes are offered for. My hope, something articulated by one of my colleagues today, was that we would target funding according to Disease Burden<\/a>. And when my colleagues consulted with the nih, they were told, no, they dont want to be directed. And 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 . With due acknowledgment that were funding alzheimers now. That strikes me as meritorious on first blush, but what criteria do you use . Appreciate the question. By the way, at every senators place, this booklet which is published this week, will walk you through a lot of the responses to that, how do we decide where the priorities are. Certainly, Disease Burden<\/a> is a huge part of that, let me be very clear about that. At the same time, scientific opportunities dont always happen at the same pace in the same areas, and when you see an opportunity thats particularly ripe for investment, we dont want to miss that. With the prize mechanism, we just put out a request for ideas about prizes for alzheimers disease. Talk about an area of high Disease Burden<\/a>. Were looking forward to seeing what that will look like. We have a prize right now waiting for responses and stirring up a lot of interest in antimicrobial resistance, coming up with a test to tell you within four hours so all the criteria are located in the book you held up, is that the case . It will go through quite a lot of it. If you need more information, ill supply it. Ill be skrultinizing that, and i thank you for publishing that information. Do you include or plan on including the private sector or outside stakeholders like some of those i mentioned as we move forward in reviewing proposals and in designing prize competitions . Absolutely. Yes, in both cases. Our review panels, especially for High Technology<\/a> efforts, we populate those with people from the private sector who have that expertise, and especially in prize competitions. We want to see what people think in all sectors about an alzheimers prize. Lastly, do you allow in your prize competitions or have any plans to offer inkind 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<\/a> to those that need it. Yes, i think we have done that, and actually in a couple of the small project prizes where were particularly asking students, undergraduates who have a great idea, to be able to apply, and they may not have access to the equipment. Were trying to be very flexibility on that. Thank you. It sounds as though the career nih folks that were telling members of congress that our idea was not thought well enough, perhaps maybe theyre on the right track. So dr. Gottlieb, pharmaceutical Development Today<\/a> frequently involves whats been called shooting in the dark. Making key decisions that impact a Development Programs<\/a> success with very limited data. For example, decisions including indication selection, dosage end points, and Inclusion Exclusion<\/a> criteria. You might say are made suboptimally because the data that informs such decisions lies in inaccessible silos. At least inaccessible to most stakeholders. This contributes significantly to higher r d costs and longer timelines. Now, the fda already has much of this data. This otherwise siloed information. So my question for you is, has the fda taken any steps in aggregating Health Care Data<\/a> across silos to improve the pharmaceutical Development Process<\/a> . And if not, would the fda be willing to analyze the data it collects to provide further scientific insight back to the Research Community<\/a> to accelerate and derisk biomedical innovation . I appreciate the question, senator. Its a complex one. I would be delighted to follow up with you on. The bottom line, first, were going to be taking steps very soon to make more data from the clinical portion of the review from approved applications public where they can be aggregated into the data sets you speak of. There are situations where we develop our own proprietary data sets from data we glean across applications to make decisions particularly around drug safety. But those data sets remain proprietary to the fda because that information is commercially confidential. Were looking how we can make those public without appropriating anyones intell intellectual property around their own data. If were making regulatory decisions on the basis of aggregated data thats not accessible to the public, thats something we should address. This is a very complex area were actively working on. Suffice to say were trying to move in the direction you suggest. There are legal complexities associated with it. I look forward to working with you. Thank you, senator young. Senator bennet. Thank you, mr. Chairman. I want to also congratulate you and senator murray on this oneYear Anniversary<\/a>, one more reason to be happy to be on this committee. Dr. Gottlieb, i wonder if you could give the committee a little bit of an update on where you are with the implementation of the medtech act, a bill that senator hatch and i worked on together. Well, i appreciate the question. And ill try to be brief. We Just Announced<\/a> major components of implementation of some of those provisions today. So we continue to move forward with this. On schedule. I think that its a good example, what were trying do in this space is a good example of what were trying to do with cures where were taking the provisions that congress outlined and trying to take the spirit of what congress tried to achieve and go beyond that. Congress set out to create a more efficient process for the review or to exclude certain Decision Support<\/a> tools used by clinicians. In the announcement today, were also going to exclude tools used by patients as well as long as they meet certain parameters. Thats an attempt 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 that we havent seen as much of is in the area of Decision Support<\/a> tools where theres tools that could take information, help support decisions without making a decision for the clinician or the patient. And i think in part, theres a lot of reasons why we havent seen as much innovation in that space as you would have thought, but regulatory ambiguity probably played a role for a period of time. Fda intended to exercise regulatory in that space. This provides us the opportunity to put out guidance that creates some really bright lines and some parameters around what is and what does and doesnt cross the line and what are the obligations of sponsors, even though who are excluded from active regulation to make sure were continuing to look at the safety of the products and collect information where things could go wrong. So i am hopeful that were going to see more innovation in this space. Tools that could sit on top of the Electronic Health<\/a> record, for example, and help physicians make decisions from that information. I appreciate your focus on it. I also noticed that unrelated to that, the fda approved the first breakthrough designated diagnostic test to detect genetic mutations for cancer. That was an approval that came through, i think the new breakthrough not breakthrough therapies, breakthrough device section that we wrote for the bill in this bill last year. And i was interested to see that at the same time that happened, there was a simultaneous decision by cms to also cover the diagnostic test. I wonder if you could share with the committee how you and cms conducted these parallel reviews and whether we can expect this sort of coordination in the future. Well, i think you can, senator. And this is an example of a test that was a Laboratory Developed<\/a> test that voluntarily came through the regulatory process. We were able to review it in six months. Under the breakthrough designation, the first, as you said, breakthrough designated product to receive fda approval. And we worked with cms to have in place simultaneous coverage at the same time. And we think Going Forward<\/a> this is a panel of cancer markers that will help guide the treatment of Cancer Patients<\/a> and help guide the prescription of therapy. This is an area of a lot of innovation right now, and what cms said in their policy, and ill let them articulate it more clearly is that panels that come through fda for voluntary approval now will automatically receive coverage, National Coverage<\/a> determination. Its a powerful incentive, i believe, for more such tests to try to come through the regulatory process. We can provide, i hope, a greater assurance of the effectiveness of that. I think thats a big step forward. I can tell dr. Collins agrees. Dr. Gottlieb, i only have a minute left. Senator rubio and i last year worked on the race act for pediatric cancers. I know you guys put out some guidance recently on developing drugs in rare pediatric diseases. I wonder if you could expand a little bit about that. We did, senator, and were going to have more to say on this area, but what we did yesterday was finalize some parameters, guidance that we had in draft form that tries to look at very rare pediatric cancers. This is pediatric diseases, excuse me, trying to outline a more efficient process for developing those products. We might not have to rely as much on placebo trials. Might be able to use modeling assimilation to represent the experience of the placebo arm, and also allow sponsors to collaborate to try to test multiple drugs in the same Clinical Trial<\/a>. The reason why these kinds of accommodations, i think, are important in this setting is because its very hard to enroll patients in these very Rare Diseases<\/a> where you literally might have just dozens of patients who have a disease who are eligible to be enrolled in trials, looking at ways you dont have to randomize some of those patients to placebplacebo. Im out of time, but i didnt thank the witnesses for their Great Service<\/a> to this country, and i deeply appreciate it. Thank you, senator bennet. Senator collins. Thank you, mr. Chairman. Dr. Collins, always glad to see you. And i so claim you as my cousin regardless of the facts. I accept. Thank you. The state of maine is doing some really exciting medical research. And i have visited a lot of the labs and medical institutes that we have in our state. The Maine Medical Center<\/a> Research Institute<\/a> is conducting cutting edge work that researchers in maine have under way to develop medical treatments for chronic diseases. And in september, one of the researchers with whom i met, dr. Oxberg, and his team at Maine Medical Center<\/a> received an award through the nih im sorry, Regenerative Medicine<\/a> Innovation Project<\/a>. 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<\/a> for understanding aging and reversing diseases like macular degeneration. I understand that the chairman has already introduced doug oliver, but 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 a number of the applications to Regenerative Medicine<\/a>. As were gathered here this morning, theres an intense discussion going on at a hotel in bethesda sponsored by fda and nih jointly on this topic of Regenerative Medicine<\/a>. Several hundred people in the room talking about applications to kidney disease, to heart disease, to aging, to such things as Rare Diseases<\/a>, common diseases, the whole landscape, and its enormously exciting to see how thats taking place. When i spoke yesterday, i even forecasted if we do this in the way that i think we could, we could cure, not just treat, but cure a disease like sickle cell in the course of the next five years. With regard to the question about aging, were learning a lot about what that process is. Ill just mention one example. Otherwise ill go into a long lecture. That is that as you age, theres a certain category of cells that are no longer able to keep going. They become senescent, but they dont completely exit the stage. Theyre still there. It turns out theyre not good actors. Theyre making the healthy cells around them not so healthy. If we can figure out, as we have already done in mice, how those senescent cells can be told, youre done now, it could slow down the normal process of aging with all that entails with terms of frailty and chronic disease. This is an exciting new development. I think it is also. I remember visiting Harvard University<\/a> and talking with researchers there about alzheimers disease and theyre also looking at the possibility of turning proteins back on to restore cells that have been lost and neurons that have been lost. Enormous excitement about all of those alternatives, as well as identifying drug targets that we just didnt know about before, by very Careful Systems<\/a> biology approaches to understanding how networks in the brain work in normal individuals and those who are at riv for alzheimers. We have a partnership with industry that you may have heard about called the accelerating medicines partnership, which is advancing this as a pace that i wouldnt have thought possible a couple years ago. And that brings me to my question for you about that program. Which aspects of the accelerating medicine projects do you find most promising . For alzheimers, there are two areas that were really pushing. One is biomarkers. We really need to find out what are the indications a therapy is working without having to wait ten years to find out if it was protective against the development of cognitive decline. Theres a lot going on there with various types of imaging, for instance, one of the proteins involved in this. The other part is the systems biology, where we step apart from what we know already and ask the question, what else is going on in the alzheimers brain and how could we use that to develop the next generation of therapeutics taking advantage of all the areas were now learning about to come up with interventions that are going to work, and then apply those early before people have already begun to succumb to the illness. We have to start early if we want a good effect. Thank you very much, mr. Chairman, and thank you, dr. Collins. Thank you, senator collins. Senator murphy. Thank you very much, mr. Chairman. Thank you both for your service. I know it comes at great sacrifice, in particular, dr. Gottlieb as a connecticut resident, i know the sacrifice that you make to serve our country. Thank you to both of you. Dr. Collins, i wanted to talk to you a little more about the all of us research program. This is a program that designs to build a diverse biomedical data set for the Precision Medicine<\/a> initiative, and im interested in it because one of the participating Health Centers<\/a>, Community Health<\/a> centers, that is currently gathering data, is based in middletown, connecticut. And i think its great that you have gone out and worked with Community Health<\/a> centers because these centers, typically serve a Patient Population<\/a> that is typically underserved in Biomedical Research<\/a>. I want to put that question to you, which is if you could talk about the importance of having fqhcs as part of this initiative and whether there are plans to expand beyond the current six Health Centers<\/a> that are right now part of this initiative, Community Health<\/a> center in connecticut is very pleased with their partnership with you on this, and we would love to see more like them get involved. My opening remarks, i put up this visual image about how it is that were seeking to enroll participants in this very bold, largest ever prospective study of health and illness in the United States<\/a>. And we are inviting people to participate by a variety of means. But one of them is through the Community Health<\/a> centers here, included in the Health Provider<\/a> organizations because we have a very specific goal here of having at least 50 of the participants in all of us being individuals that are underrepresented traditionally in medical research programs. And the Community Health<\/a> centers who provide care to a great number of people, particularly those in lower socioeconomic situations, are very excited about being our partners and were excited about having them as partners. Were starting with the six and glad about middletown as a place that is clearly been very successful in getting this up and going. But that is a pilot to see if we can in fact enroll many more. We aim to launch this project fully next spring. At the moment, were in a beta test. We have enrolled about 12,000 individuals. Were learning everything we can about how the moving parts can all Work Together<\/a>, and its looking very good. Great. Dr. Gottlieb, i wanted to switch topics to another Exciting Development<\/a> out of the cures act. This is the establishment at the fda of the Oncology Center<\/a> for excellence. Which creates a cross center team to Work Together<\/a> on a variety of products to treat cancer. Just an update on what you have learned thus far from this model and what are you taking into account when youre thinking about creating other disease specific offices . Having gone to college in middle town, connecticut, i appreciate the shoutout. Right. This is an extremely important effort on the part of the agency. I appreciate you asking the question. The Oncology Center<\/a> for excellence, what we have tried to do is consolidate the clinical portion of review of Oncology Products<\/a> into one combined center. This was instrumental in our ability to expedite the review of the products by consolidating the clinical expertise, able to more efficiently look at some of the biologics. We have had challenges fully standing it up, and in all candor, its related to fully funding it. We believe that this is the future of the agency, though, trying to get these consolidated programs in place. Were looking to other therapeutic areas we can do this. I think before we can progress to other areas, we need to make it work in the oncology setting. Were looking forward to continuing to try to move this program forward. Great. Another reminder to us that weve got to give you the funds to implement this act. Quickly back to you, dr dr. Collins. One more question, and that is on another part of the 21st century cures act. Senator isakson and i worked on a provision that allows for the cdc to collect information on the incidence and prev lnls of neurological diseases. This was the advancing research for neurological diseases act that senator alexander and senator murray helped us include in this legislation. Talk for a second about the importance of having this data on neurological diseases to Research Done<\/a> at nih. Well, its incredibly helpful to know what incidents and prevalence is for neurological conditions, particularly a condition like parkinsons, but there are many other disorders as well. It is challenging and expensive to do that kind of analysis and do it effectively and to keep it updated. And as i understand it, while cdc was given the charge to do this, at the present time, they do not appear to have the funding to carry it out. Great. All right. Another you have another advertisement to put the money behind the legislation we passed. Thank you very much, mr. Chairman. Thank you, senator murphy. Senator hatch, former chairman of this committee. Welcome. Thank you so much, mr. Chairman. I appreciate it. I certainly appreciate you two gentlemen. Youre some of the best Public Servants<\/a> i have seen in all of my 40plus years in the United States<\/a> senate, and im proud of both of you, and i hope you just keep doing what youre doing. So i want to thank you both here today. Dr. Gottlieb, last congress i joined senator bennet in authoring the path act, which was included in the 21st century cures, and will help advance the development of urgently needed new antibiotics for multidrug resistant infections by allowing them to be studied in smaller, more rapid trials and approved more quickly for patients who need them most. As you know, fda was tasked with developing guidance for industry regarding this limited Population Pathway<\/a> or lpad. Can you detail the ways in Stakeholder Input<\/a> is being solicited and incorporated into the guidance . Thank you for the question, senator. We plan to issue the guidance that you reference in the spring. We have been meeting with stakeholders and other interested parties in the development of that guidance. Consistent with our good guidance practices. We will have stakeholder meetings in any development document. We think this is an extremely important pathway and we have had multiple ind meetings with sponsors and believe this will be a robust vehicle for trying to create new pathways for drugs targeting significant unmet medical needs. Thank you. Dr. Collins, i was pleased to see as a result of 21st century cures, the nih was able to start the Regenerative Medicine<\/a> Innovation Project<\/a> that several of the first of words from this initiative had gone to research on Rare Diseases<\/a>. And as you may know, i have been a champion of the Rare Disease Community<\/a> for quite some time, and i applaud you for also recognizing how Vital Research<\/a> in this area truly is. Strides in Regenerative Medicine<\/a> may prove to be truly transformational in the way we understand and possibly even treat Rare Diseases<\/a>. What barriers do you foresee in expanding research into this area, as the Regenerative Medicine<\/a> Innovation Project<\/a> continues . Well, thank you, senator, for the kind remarks at the beginning of your time, and also for the question. I think this is an enormously exciting time for Rare Diseases<\/a> because we have tools now that are starting to work. Dr. Gottlieb referred a little while ago to gene therapy. Today in the new england journal of medicine, really dramatic advance in heme feel yeah using gene therapy to develop the gene to the liver of men who are affected. With really remarkable benefit over the course of many weeks. Thats just a single example of what is possible in this space. I mentioned earlier my hopes we could treat Sickle Cell Disease<\/a> by taking out the bone marrow cells, putting them back. Its a transplant to yourself, a gene editing preach which we also find exciting. Recently, this disease called spinal muscular atrophy, in children who are born, seem to be okay, then develop paralysis, now with a Clinical Trial<\/a>, some of these kids are making it to their prekindergarten and looking pretty good, again, by a genetic change thats been introduced using either a virus or a gene editing strategy. I think the big barriers right now is just to push the science as hard as we can, and the cures bill helps us by giving us that kind of inspiration. Im glad to say the barriers with regulation are not the issue right now wurxing with scott and his colleagues at fda who also have made rare disease a very high priority. Of course, there is this issue about investment. Companies may not be so interested in putting a lot of money into a very rare disease because the market is going to be small, which means nih has an Even Stronger<\/a> responsibility to derisk those projects and push them as far down the road as possible. One more thing you did for us in the cures bill is make it possible for us to run phase three trials for Rare Diseases<\/a> in the therapeutics and neglected Disease Program<\/a> which we had not previously had the privilege of doing and which we will be using aggressively for this purpose. Dr. Collins, one of the provisions of the 21st century cures was designed to improve opportunities for young and emerging researchers. I heard from the university of utah which received about 150 million in nih grants annually that they have seen an improvement in this area. For that, i would like to thank you and your staff and your work in this area. My time is up, so ill just thank you. And tell you how much i appreciate your willingness to serve in this government. And how much you really mean to People Like Us<\/a> who have worked all these years in these areas. Thank you, senator. Thank you. Thank you, senator hatch. Senator baldwin. Thank you, and i want to add my words to the others of congratulations of the bipartisan work you led, mr. Chairman and Ranking Member<\/a> murray, on the 21st century cures act. And thank both of you, dr. Collins and dr. Gottlieb for your service. During the Committee Deliberations<\/a> on 21st century cures, i regaled the committee any number of times about being the granddaughter of nih funded scientist and how that influenced my championing, strengthening our Research Enterprise<\/a> and support for the nih. But in my first year in the United States<\/a> 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 fond du lac, wisconsin. Ian told me that Cancer Research<\/a> no doubt helped save his life. And thats why he wanted to grow up to be a scientist. To help others with his disease. But he was concerned that 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 Research<\/a>ers act. Along with my colleague senator collins. 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<\/a> the genetics of ostio sarcoma and he is now working with a scientist at Huntsman Cancer Institute<\/a> in salt lake city, utah, on pediatric cancers. Dr. Collins, i am encouraged that the nih has begun to robustly implement the next Generation Researchers Initiative<\/a> to help support future scientific leaders like ian. As they discover cures for cancer and pursue other lifesaving research. You announced a new policy this year to issue more awards to investigators in the earliest parts of their independent Research Careers<\/a> to help them sustain or achieve research independence. Id like it, dr. Collins, if you could please describe how each institute and center will prioritize awards for these early and midcareer investigators, and how this will help improve and stabilize opportunities for our next generation of researchers. Well, thank you for the question. Youre kutouching on a personal passion that i feel and that many of the other Institute Directors<\/a> do as well. We actually just published about a month ago, myself and mike lower and larry, a description of what this policy is and how were going to implement it. Because we passionately agree with you that the next generation is really critical for our future, and it has been a tough period with the loss in purchasing power that nih sustained between 2003 and 2015. Those firsttime early stage investigators were having a hard time getting started. And we cannot lose them. We were starting to. So each one of the institutes depending on where they fit, think they can find their flexibilities, are freeing up dollars to make it possible for those earlystage investigators who otherwise would just miss the pay line to actually get funded. We started this quite late in fiscal year 17, but we were able to make a whole bunch of awards that otherwise would not have happened, and were not done yet because were actually trying to see if we can reach back into some of those fiscal year 17 reviews and fund a few more of those. And we will have a meeting next week of my Advisory Committee<\/a> where we have a working group, which you would be interested in because it has graduate students and postdocs and junior faculty, the people who are really involved in this. We will have a deep discussion about whether we have the right plan here in order to make this particular priority really happen. I can see im running out of time, but i want to just note, maybe you can answer for the record, that you estimate that this new effort to prioritize more awards for early investigators will be about 210 million in the next year and 1. 1 billion over the next five years. Does nih need Additional Resources<\/a> for this policy to insure that it fulfills its promise and continues to advance all other critical nih efforts in. Very quickly, we are prioritizing this within our existing resources, but of course, we could 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 just want to thank both of you for your answers, and i will be submitting more questions for the record. I have another commitment i have to make, but this has been an excellent hearing. As chairman, i really appreciate it, and we have another one next week on the Mental Health<\/a> part of cures. Its really educational for all of us, and i appreciate both of your efforts and look forward to continuing to work with both of you. Thank you, senator murray. Senator warren. Thank you, mr. Chairman. So we have been talking about the cures act. It has a lot of bipartisan provisions that im really glad became law. During our recent hearing on gene editing, we discussed new genetic privacy protections for Research Participants<\/a> that senator enzi and i had worked on together. Right now, massachusetts Biotech Companies<\/a> are benefitting from a provision that senator bennet and senator burr and senator hatch and i wrote to try to clarify the fdas authority relating to gene authorities. But cures also fell short in a really big way, and thats on funding. I led democrats on this committee in calling for an extra 50 billion for the nih and the fda. And cures did not send one single new dollar to these agencies. Instead, it only said that future congresses might spend about 10 of that amount on nih and fda. And 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 the nih. So dr. Collins, let me just go through this a little bit. Does the nih fund most of the grant applications that 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 and see how much money we have. Out of every 100 applications you get, youre funding about 19 of them. Now, is that because the other 81 would have been bad investments that would not have helped us make biomedical breakthroughs to advance science . If we look back in history, say back around 2000, 2001, we were funding about 30 , maybe even 35 , because the funds were more available. We have looked at those to see, did a grant that scored at the 25th percentile turn out to be less productive than one at the 15th percentile . The answer is no. We cant tell a difference up to about the 30th percentile. Its hard to do so in the top third. So in other words, if we roughly, just using the numbers you had here, if we doubled, for example, the number of grants that we were able to fund, you think theres still a lot of good science to be had out there. I think there would be fantastic science. Okay, thats powerfully important. I want to follow up, too, on the point that senator baldwin made and the discussion you all had about researchers in the early part of their career. You know, getting that first nih grant can make or break an academic 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. So i just want to ask more about the details here. Where are we right now on early career researchers . What percentage of the grants are they able to get . Beginning in 2008, we actually instituted a policy so that those applicants who came to us for the first time as a Principal Investigator<\/a> got a bit of a boost. They competed against each other essentially as opposed to against the most experienced ones which meant effectively in priority score terms they got a few extra points, but thats not good enough. Were still losing lots of those. Their success rates for those early stage investigators, were still well below what you would want to see. Ultimately, we think it would be most healthy if at least, say, 25 of those applications were going to get funded. And thats what were trying to do with this new initiative, the next Generation Research<\/a> initiative, which is named specifically for the words used in the bill. Thank you, senator baldwin, for that encouragement. But i understand right now, we have been at about 16 . Is that right . And youre saying at a minimum, we ought to be boosting that to about 25 . And we have looked closely at every institutes successes and tried to figure out how we can get there with this new policy, yes. I know that nih has done what it can in this area. But nih funding is still down about 15 of 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, frankly, it didnt even come close. And thats why today were reintroducing the national Biomedical Research<\/a> act, which provides 50 billion in new funding for the nih and for the fda. I see youre sitting up straighter there, dr. Gottlieb. This legislation is cosponsored by senator sanders, casey, franken, bennet, whitehouse, baldwin, murphy, kaine, and hassan, as well as several of our democratic colleagues who are not on the committee. Families across this country are waiting for medical breakthroughs. And researchers are waiting for the money to fund their work so they can make those breakthroughs. Its time for us to step up and put more money into nih. Thank you. Thank you, mr. Chairman. Thank you, senator warren. Senator hassan. Thank you, mr. Chair. And ill add my thanks to you and the Ranking Member<\/a> for this hearing and for your work on the cures act. To doctors collins and gottlieb, thank you for being here. Would you please, along with us thanking you for your service and leadership, please thank the women and men who work with you every day in your agencies for the extraordinary work they do on behalf of the people of the United States<\/a>. Dr. Gottlieb, i wanted to start with a question or two for you. We have discussed the devastating impact that the Opioid Epidemic<\/a> is having in New Hampshire<\/a> and all across the country. One of the tools in combatting the epidemic is medication assisted treatment like bup anephrine. It plays an Important Role<\/a> in recovery along with access to other services and supports. And we also both of you, i think, mentioned in your opening comments the possibility that we will develop nonaddictive pain products, which would help prevent addiction when for example, someone gets in an accident and is prescribed an opioid and then becoming addicted. The 21st century cures act took some important steps on patient focused Drug Development<\/a>, including requiring guidance on Patient Experience<\/a> data. So dr. Gottlieb, can you discuss how patient focused Drug Development<\/a> initiatives could be used in relation to developing new medication assisted treatment and the development of new nonaddictive pain treatments . I appreciate the question. The bottom line is they can be a very important tool. And i think the provisions that are in the cures act are going to help inform how we go about thinking on the parameters we use to encourage the development of products, both kinds of products, nonaddictive alternatives to some of the opioid products, but in particular, medication that can assist people to live lives of sobriety. We have committed to publish new guidance documents for outlining the pathway for the development of medically assisted therapy. We want to create incentives for sponsors to come in and try to develop those products. Part is making sure we have very clear guidelines. As part of that, were going to be looking at alternative end points we havent looked at in the past. For example, issues like craving. We know craving is a factor that leads people to continue to use opioids. If that can be an end point in the trials that we use to assess new products, it might provide a more efficient pathway. That certainly is informed by the work we do with patients and looking at patient informed end points. Thank you. And dr. Collins, i wanted to talk to you, too. You made a comment in a response i think to senator alexander, about the impact of the other Transaction Authority<\/a> that you now have. And can you comment a little bit about how that could be helpful in the opioid space . You said it would be, but how specifically would ota be useful to the nih in its work in doing opioid Disorder Research<\/a> . So the plan that we have put in place, which is going to be fleshed out in more detail next week in an intense twoday meeting, will involve a lot of rapid action, both to come up with new ways to treat addiction, because we need more options to treat overdoses that dont respond as well as they might to fentanyl, and to develop these new nonaddictive pain medicines that everybody agrees. Thats going to require partnerships between academic institutions that have the approperate science skills, with industry that may have formulation capabilities or Drug Development<\/a> pathways. If we have to build that on the process of issuing grants and contracts and the traditional way, its going to take a long time. Other Transaction Authority<\/a> allows us to do something in maybe six weeks that might have otherwise taken nine months. We have learned how to use this effectively and responsibly for the all of us program, for the common fund. We think this would be a great place for it to be available. We will be careful about how to use it, but it could help us a lot. Well, thank you. And finally, i wanted to talk a little bit about our new generation of scientific researchers, because both senators baldwin and warren were talking about that. Cures took important steps for the next generation of researchers by helping to make it more affordable for perspective students to pursue their graduate degrees in science and engineering. Nih has been responsive to these efforts and in september announced a new policy for implementing special funding consideration for these graduate students. In recent weeks, i have heard from a number of graduate students, dr. Collins, who are fearful of 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 even force them to drop out of their programs. One particular provision is in the house bill, would require graduate waived tuition is taxed with their stipends. A change i have heard from ph. D. Candidates could really impact, one wrote, this would triple my taxable increase and increase my yearly taxes by more than 10,000. It would force me to drop out of school because i wouldnt be able to afford rent and groceries while earning below the poverty line. Can you comment, dr. Collins, on what you think this provision would mean for the future of our young researchers and Biomedical Research<\/a> . Very quickly, because of the time, these are our future graduate students that are learning to be the next generation of leaders in science across many disciplines, but certainly, i think of life science needing those individuals to be the ones who are going to make the next breakthroughs. Anything that happens to discourage the best and brightest from taking that track or to feel that they cant afford to do so is something we should approach with great concern. Thank you very much. And thank you. Thank you, senator hassan. Senator whitehouse. I join my colleagues in congratulations you and Ranking Member<\/a> murray on the success of a year ago. Hope we have more to come. Dr. Collins. One of the things we got into the cures act was a request that you reduce the burden of what we called lowrisk subrecipient monitoring. I just left brand universitys director of research, who was in the interim a moment ago. This was also the university of Rhode Islands<\/a> request. You get things Like Brown University<\/a> and university of rhode island working together on a project. And the supposed obligation to monitor the subcontractors now applies to each of them. So you have uri monitoring brown, brown monitoring uri. Can you help reduce the bureaucratic and reporting burden to your subrecipients . Senator, we dont particularly enjoy doing that kind of lowrisk monitoring either. And we have in fact, thank you to the cures bill, put together a proposal of a way that we might reduce the amount of lowrisk subrecipient monitoring. There are certain situations that maybe dont meet that. Please include us in that cycle so we can be helpful in giving advice and making sure that goes forward at a good pace. Dr. Gottlieb, the cures act was very good at getting you to fda to connect the device and the drug sides a little better, and you have done a lot of training to try to connect the two. But the original testimony that we had from fda from both the drug and the device sides was what you really needed was a third pathway for drug device combinations. Would you assure us that if it turns out that 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 a challenging area for us over a period of time. We think the provisions give us a lot of new tools to address it. Well be implementing the provisions and rolling out guidance soon that we think will articulate a better pathway. If it doesnt resolve the challenges you have identified in the past, i would absolutely be delighted to talk to you about alternatives. Just so long as were staying in touch and in the loop and not wasting time if it turns out not to be productive. Dr. Collins, there have been some very everyday moments that are very significant. Back in 1876, a gentleman called his assistant watson to come into the room. And in 2012, a lady named Cathy Hutchinson<\/a> took us for coffee, pretty minor things, except for the technology involved. Come here, watson, i need you, was Alexander Graham<\/a> bell first using the telephone to summon his assistance, and ms. Hutchinson picked up the cup of coffee and brought it to her lips through what at Brown University<\/a> they call a brain gate, which is elect roads in the brain so that by mere thought, she could control the robotic arm. The Brain Technology<\/a> that nih is funding is terrific. Are you satisfied that youre adequately connected with the brain gate funding to make sure that everybody is pulling in the same direction . We are enormously excited about that as well. Yes, we have worked quite closely with darpa in this whole yie idea about how you could begin for people who are paralyzed to control a robotic arm to carry out fairly sophisticated activities including the one you mention said. This is a big part of what the Brain Initiative<\/a>, which involves nih and darpa and as well as the department of energy, as well as International Partners<\/a> aims to do, to figure out hout the 86 billion neurons between your ears do what they do in these complicated circuits, which were beginning to take apart and understand even better, because we would like to go from where we are now, which was sort of an empirical effort to get this to work, to understand it so well that you knew exactly where to place the electrodes to get the movement of the arm. Last topic. We have just done some good bipartisan work on plastics in the ocean. The u. N. Has issued a unanimous statement of concern about plastics. Plastic, as you know, doesnt biodegrade. It just breaks down to smaller and smaller points. And then microscopic creatures can consume it, and it begins its track up the food chain. Never in Human History<\/a> have we had to experience that kind of plastic loading into our diet. Its now found in tap water. Its found all over the place. Its obviously going to be in our food. Is this something that the National Institutes<\/a> of health should begin doing basic research on so if this turns out to be a problem, it is certainly a new experience for human kind to have to digest and process that kind of microscopic plastic in the food chain that youre alert to it . Absolutely. The National Institute<\/a> of environMental Health<\/a> sciences, the one located in north carolina, is already looking at this. I can give you a summary of where we stand in terms of the research being conducted. I would appreciate that. We can do that offline and through your staff. Be happy to. Thank you, chairman. Thank you, senator whitehouse. Thanks to both of you for being here today. I hope youll follow up with the memo on Regenerative Medicine<\/a> some time in the next few weeks that i can give to senator mcconnell and other senators who are interested in that. You heard several senators mention that today, and you have quite a story to tell, i think. Also, well be interested as we look toward a second anniversary of the cures act in a year to especially at our were taking years between idea and something in the medicine cabinet, how we are compressing that and setting priorities and how fda, nih and cms are working together to do that. I salute you for the steps you have already taken but that would be a very promising way to do things. And really you have to do that more than we. We can do some things in legislation but that we cant do, as well. We can order you to do it. You have to do what you already started to do which is to Work Together<\/a> to speed that up and still do it in a way that provides safety and efficacy. Without getting you in the middle i sometimes worry that the messages that come out of washington are heard by the graduate students and the researchers across the country and they worry we are not funding their work. If the president s budget says one thing and everybody pays a lot of attention to it and that really doesnt happen and nobody knows it doesnt happen. I remember in president obamas last year he actually reduced recommended discretionary funding for the National Institutes<\/a> of health and recommended big increase in mandatory funding for National Institutes<\/a> of health. This isnt too hard for people to understand. This is the authorizing committee. We dont appropriate money usually. We cant appropriate money. And we have another committee which senator murray is the Ranking Member<\/a> of and senator blunt is the chairman of and that is the money committee. So for us to appropriate, say, 50 billion in new funding for National Institutes<\/a> of health is a wonderful aspiration but that is not what we do. We decide, for example, whether dr. Gotlieb should have a new break through path for medical dwi devices. We would say that is our job. We were to try to appropriate another 50 billion to you the Appropriations Committee<\/a> would be very upset because they say that is our job. So let me ask you this. We have discretionary funding that is the Appropriations Committee<\/a>. We have mandatory funding which this committee can do. What is the size of the nih budget . What is your total funding . And to be clear all of that goes out to institutions. Almost all of it is money that is supplied by the through subcommittee that senator murray now, two years ago if im correct recommended and Congress Approved<\/a> 2 billion increase in funding for nih. Last year they recommended and congress agreed another 2 billion increase for nih. Once we put something in the budget we counted for ten years. The first year is 2 billion. Thats 20 billion over ten years. The second year is 2 billion. Thats another 20 billion over ten years. If we do it this year another 20 billion over ten years. Now, thats not guaranteed but that is usually what we do. We put it in the base and we expect to do it again with the priorities we have on this committee and on the Appropriations Committee<\/a> i expect that to be the case then in addition we did something that was very unusual in the cures bill, very hard to do. We took 4. 8 billion and created a hybrid funding that created consternation with the house of representatives and gave special funding to Precision Medicine<\/a>, Brain Initiative<\/a> and some other things. The chances of that happening again are close to zero. The real money that is supposed to go to the National Institutes<\/a> of health comes through the Appropriations Committee<\/a>. They have been doing a tremendous job over the last few years. I will give senator murray great credit for squeezing out of the budget the sources which are a good deal larger than in the last few years. Nothing wrong with aspiring for more. Nothing wrong with looking back 15 years and saying that if we had gone up at the same rate we had been going up it would be even higher today. I think it is important for researchers across the country to know that this congress in a bipartisan way has been committed to funding. I really appreciate your taking the time to explain all of that because i think there are people watching wondering how the decisions get made and certainly there have been a lot of anxieties particularly from young investigators about what is going on and what is the likelihood of pathway. You pointed out that there is a great deal of reason to be excited and optimistic because the science now is so promising and exciting. With two years already with this 2 billion increase already happening. Significant promise that it may happen for the third. It is a very encouraging sign. Again, im grateful that in this era where so few things seem to be bipartisan. Medical Research Continues<\/a> to be so. I will ask one last question. With all of this excitement and breath taking bio medical advances do you talk to High School Students<\/a> and see increase in interest in biology . I will tell you that my girls are 8 and 4. I think that there is an increasing excitement among those training in medicine right now about what the future holds. I have a lot of contact with residents and medical students and i see increase in excitement. Dr. Collins . Certainly when i go out to university to give a talk and meet with students and i always do that, that is the best part of a visit is to say i want a chance to meet with the graduate students and i dont want anybody else in the room because i want to hear from them. I have heard a lot of anxiety in the last couple of years and i am trying to reassure people that things are looking much brighter than they think they are, the sense out there of trouble is probably beyond what it maybe ought to be considering the path we are on. Boy, do i hear excitement about the scientific opportunities. If you can quickly switch to that topic then the spark goes into the conversation and the eyes light up and this graduate student tells you about the experiments they are doing that week and how excited they are about what they can do. There are things a single student can do now that might have taken a team to do five years ago. This is the golden era. If anybody really wants tohave a great time in science they should come and join us. I hope ian, whoever that is senator baldwin was talking about will be one of those as soon as possible. We want the best and brightest to join us. The hearing record will remain open for ten days. Members may submit Additional Information<\/a> for the record within that time if they would like. Our committee will meet again next week on tuesday december 12 at 10 00 a. M. For a hearing titled cost of prescription drugs and examination of the National Academies<\/a> of sciences, engineering and medicine report making medicines affordable. This is the third in our hearing on drug prices which has been requested by both democratic and republican members of our committee. Next wednesday we have our oversight hearing on the Mental Health<\/a> aspects of the cures act. Thank you for being here today. The committee will stand adjourned. The senate is back this afternoon, as well for work on a judicial nomination. Watch the house live on cspan and senate on cspan 2. Off the floor work continues on tax Reform Efforts<\/a> discussing reconciling the two bills that were passed separately out of each chamber. We will have live coverage for you. Online at cspan. Org and with the free radio app. This week on the communicators two advocates talk about Net Neutrality<\/a> and regulating the internet like a public utility. Number one, it deregulates broadband. It basically takes broadband oversight out of the fcc entirely and gives it to the federal trade commission which does not have as strong tools. It eliminates the rules against Internet Service<\/a> providers like comcast and at t blocking internet traffic and allowing for what is called fast lanes. In other words, charging online providers to get to the consumer faster and better quality of service. Thirdly and this is really important, it preempts or prohibits the estates from protecting consumers and competition with similar rules. There are three acts, at least three federal statutes that protect consumers and entrepreneurs here. They are the sherman act, clayton act and federal trade Commission Act<\/a> and there are state protections. The order says it preempts the states from being miniature fccs and trying to regulate economics of broadband. There are Consumer Protection<\/a> laws at the state level. Watch the communicators tonight at 8 00 eastern on cspan 2. This past friday the Fourth Circuit<\/a> court of appeals heard oral argument in the case of National Refugee<\/a> Assistance Project<\/a> v. Trump. This latest edition places certain restrictions on nationals from eight countries, chad, iran, libya, north korea, syria, somalia, venzalia and yemen. The Supreme Court<\/a> issued an order allowing the ban to take full effect. This is about two hours. Hours. [inaudible] [inaudible] [inaudible] [inaudible]","publisher":{"@type":"Organization","name":"archive.org","logo":{"@type":"ImageObject","width":"800","height":"600","url":"\/\/ia800400.us.archive.org\/24\/items\/CSPAN3_20171211_160800_Medical_Product_Development_and_Innovation\/CSPAN3_20171211_160800_Medical_Product_Development_and_Innovation.thumbs\/CSPAN3_20171211_160800_Medical_Product_Development_and_Innovation_000001.jpg"}},"autauthor":{"@type":"Organization"},"author":{"sameAs":"archive.org","name":"archive.org"}}],"coverageEndTime":"20240630T12:35:10+00:00"}