Transcripts For CSPAN2 Medical Product Development And Innov

With the best overall entry. Deadline is january 18. Contest details on our website studentcamp. Org. Next, fda commissioner Scott Gottlieb and Francis Collins testify on medical innovations. They appeared before the Senate Health innovation and Pensions Committee on issues including the Opioid Epidemic, rare disease funding and research into treating addicts nonmedically. This is an hour and 50 minutes. Good morning, the Senate Committee on health and education will come to attention and order. Another bipartisan hearing which is what most of our hearings are which is what bernie and i created on the witnesses. I would say every member of this committee would probably agree on the subject in witnesses today. This is a hearing entitled implementation of the 21st century cures. Progress and the path forward for medical innovation, we will hear from Francis Collins, and doctor Scott Gottlieb, commissioner of food and drugs. We all have as i mentioned a hearing next wednesday on another important part of the cures act which was the first reorganization of our Mental Health laws of a decade which senator cassidy and murphy played a major role in and we look forward to that. Senator murray and i will each have an Opening Statement and introduce our panel of witnesses. Each senator will have five minutes of questions. I expect we will have a large number of senators going to this hearing. 2007. Oliver who is in the front row i believe, a Computer Programmer began to have trouble seeing and after a near accident had his drivers license taken away and was declared legally blind. The culprit was a rare form of macular degeneration. Doug moved to nashville for a doctor who told him while there were no cures, don could Search Online for a Clinical Trial. Dunn found a Regenerative Medicine Clinical Trial in florida and the doctors took cells out of the bonemarrow in his hit , found them in a centrifuge and injected those into his eye. Two days later he began to see. His eyesight eventually improved enough to get his drivers license back and not that he became an effective advocate for more support for Regenerative Medicine. The proper way, which we included in the 21st century tours. Earlier this year, the gave me the pen he used when he was blind and said i dont need it anymore. I treasure it and keep it in my office and i thank him for his advocacy and i hope others have the same advantage. Congress passed the 21st century cures act and hoped to in unleash medical innovation and give americans access to treats and cures so more americans can experience it. Its especially appropriate to be having this hearing today because it marks the oneYear Anniversary of the Senate Passing cures. When it became law last december, i said that president obama had signed a second christmas miracle. The first one in 2015 was the every Student Succeeds, aloft fixing no child left behind which came from this committee. Last year it was the 21st century cures act which the leader mcconnell called the most important legislation risk past last year. Ive often said the every Student Succeeds that the law is not worth the people paper its printed on if itsnot implemented properly and then to ensure cures is implemented properly we began our oversight on jurors with a hearing in october. With Electronic Health records provisions and in todays hearing we will continue our oversight mix wednesday on the Mental Health provisions. Today we are hearing from doctor collins and doctor gottlieb on provisionsrelated to Biomedical Research. We are fortunate to have two such talented leaders who know their agencies and are widely respected in congress and capable of getting results. That doesnt always happen. Sometimes you have capable leaders dont have any money or authority. Sometimes you have authority and money and the leaders dont have a clue what theyre doing. In this case we got new authority, new money and two of the most capable leaders we could have so we are delighted. While the legislation includes provisions to improve healthcare records, resources to fight the Opioid Epidemic in the first update to mental programs in a decade the main purpose was to provide Additional Resources and authorities to do drugs through the process and Doctors Offices and into patients medicine cabinet while ensuring the fda Gold Standard of safety and efficacy. Its not an overstatement to say the 21st century cures act as the potential to affect every american family, taking advantage of breathtaking advances in Biomedical Research. Doctor collins had a Senate Appropriations subcommittee hearing in 2016, you offered bold predictions, you call them for future Major Medical advances if we continued funding the National Institutes of health and ensure the fda has the tools it needs. One prediction is that scientists will find ways to identify alzheimers before symptoms appear as well as how to slow or even prevent the disease, alzheimers causes untold family great and causes 259 billion a year. Another prediction of yours was using a patients own stem cells, doctors could rebuild his or her heart. This persons heart would make transplant waiting risk and antidrug obsolete and put senator bill frist out of business. Doctor collins, youve made other predictions because he was a heart transplant surgeon. Doctor collins, you made other predictions. The developmentof an artificial pancreas. The z vaccine, universal flu vaccine. And hivaids vaccine, all available within a decade. New pain medicines to help patients as we continue to battle the Opioid Crisis that kills 91 americans every day. You said this week and another hearing that might happen as soon as five years. The 21st century cures law put in place policies that will fund Biomedical Research and apply limitless opportunity to help doctor collins predictions the unreality. Today we want to find out doctor collins and doctor gottlieb, those leading the charge how implementation of the law is going. For example, cures included a surge of onetime funding, 4. 8 billion for the nih including money for the position initiative and green initiative. I know from talking with Vanderbilt University that the Precision Medicine initiative, the plan to map the genomes of 1 billion volunteers to help researchers about cures tailored to a patients genetic environment and lifestyle has already begun and rolling patients less than one year after the program began. In addition to Precision Medicine initiatives, im curious about how funding has been spent and when and how you plan to Keep Congress informed of your results. Its part of the process to look at burdensome regulations on researchers id like to hear if this is helping researchers spend more of their time on research and less on administrative cakes. Cure has also put in place policies to move effective treatments and cures through the regulatory process more rapidly and at a lower cost. For example, senators berger, and hatch work on a breakthrough revision for devices modeled after a breakthrough provision for drugs but the first breakthrough approval was last week for an exciting cancer diagnostic. Senators isaacson and casey worked on removing red tape from the review of combination products. I worked with senator murray to make sure the fda had the authority to pay scientists keep up the rate of innovation. 21st century cures 30 million to support Regenerative Medicine and an accelerated pathway for those treatments fda so we can hear more stories like donna olivers. The fda has been implementing those provisions and i look forward to hearing how fda and nih are working together to make sure the authorities are helping to advance this important work while ensuring that bad actors dont take advantage of the exciting field to harm or defraud patients. Its going to be difficult to cover everything today but i look forward to hearing about the process being made to unleash medical innovation and bring new drugs and devices to patients. Senator murray. Thank you so much chairman alexander and welcome to our guests today. Thank you doctor collins, good to have an opportunity this week to talk to you about the work being done in and at my age. Doctor gottlieb, and looking forward to your updates. Next week marks one year since the signing of the bipartisan 21st century cures act and as i said at the time of the passage and ill repeat it now, as much as this law helps to build on americans tradition of leadership in lifesaving Public Health initiatives and medical innovations, cures was first and foremost about providing hope. Hope to the millions of people and families impacted by illness and disease. Hold for communities suffering at the hands of the opioid and like all of my colleagues including our guest today, this is something i think about every day and i visited with communities back home in my home state of washington and have been devastated by addiction. While we can and must do more, im glad that you are is an important first step in dedicating over 1 billion in new funding above and beyond the budget to help states and communities fight back against that crisis and we secured important changes to ensure this money went directly to the states that needed the most. For far too long our Health System has failed patients and families seeking treatment and support. That is why in cures we prioritize expanding access to the quality, care for Mental Illness and Substance Abuse and we strengthen coordination between local agencies engaged in crisis intervention so im looking forward to that hearing we are having this week. On todays topic im proud of our work to address headon of the hardest to treat diseases but i providing nearly 4. 8 billion in funding for nih including support for the bovine cancer moonshot for Precision Medicine and Brain Initiatives. Both of which are important to my home state where we are spearheading much of that new research taking place as a result. Along with these investments we included provisions in the law to encrypt nih with the tools needed to meet the demands of ill medical research in the 21st century. We made it a priority to include the perversion of women in other underrepresented populations in Clinical Research so that the promise of these initiatives by Precision Medicine are extended to all patients. As part of this work i champion the provision that creates a task force to support the evaluation of drugs for pregnant and lactating women and we addressed many of the concerns Holding Back Progress in the Research Community supporting young researchers and reducing the red tape. Cures also made sweeping reforms to fda and one of my Top Priorities was granting the agents greater hiring authority to recruit and retain qualified individuals who understand the latest science and technology and i am interested to get an update from you, doctor gottlieb on how you are filling those positions especially in light of the hiring freeze was mandated by the president. We made sure cures gave the fda to ensure medical devices like goats would cause the outbreak of antibiotic resistant infections in washington are safe for patients and we gave more clarity to developers of drug device combination products and codify key provisions in the Prescription Drug user Fee Agreement including to ensure patients are included in the Drug Development process. So im glad we have the opportunity to talk about the ongoing implementation of cures and i will be very intent 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 investment needed in nih and fda not to support cures but also to advance 21stcentury science and innovation. In general im glad nih and fda have been timely in influencing the law including many of the provisions i talked about. Im encouraged by these efforts and want us to keep moving in the right direction and doctor collins, in light of the extremely concerning direction the Trump Administration has taken when it comes to healthcare especially in regard to Womens Health i want to hear more about what nih is doing to bolster your research to further science over extreme ideology. Doctor gottlieb, during your confirmation hearing you said 21stcentury jurors is a good roadmap for what you hope to accomplish as commissioner. Id like to hear about how you are pushing back against his administrations the regulatory approach and making sure cures is being efficiently and faithfully implemented and specifically i will be asking more about what fda is doing to ensure the medical devices patients rely on our safe and effective. Both nih and fda have made steps in meeting cures deadlines this year but we have more deadlines coming up in 2018 and as we know funding remains an issue, i look forward to hearing from each of you to make sure we are putting patients and Families First so again, welcome to both of you and thank you for being here. Agency, confirmed on may 9 of this year. Welcome again to eyewitnesses, dr. Collins, lets begin with you. Good morning. Thank you, chairman alexander, Ranking Member murray and other distinguished committee members. It is an honor to be a today with my colleague dr. Scott gottlieb. As you well know the 21st century cures act aimed to catalyze a a very important gol shared by all americans to speed the pace at which a scientific discoveries are translated into lifesaving treatments and cures. We at nih actually greatly appreciate your leadership in passing this bipartisan act by a vote of 95four innocent exactly one year ago today. This enhances our authorities and resources in ways that will help us to achieve this goal. Goal. Many provisions are included in the act such as reducing administrative burdens so our scientists can devote more the time to research, expanding our ability toward prizes for exceptional ideas. And strengthening measures to protect patient privacy. In my written statement ive submitted a comprehensive report on at nih has worked quickly to implement the provisions of the act. We are motivated by a sense of urgency to help patients in need of breakthroughs. In my statement of like to focus on the Cures Innovation Fund. Among the vital areas of reported research being a celebrated by this fund are the Brain Initiative, the cancer moonshot, the Regenerative Medicine Innovation Project and the Precision Medicine initiative. Lets begin with the Brain Initiative. This pioneering effort is aimed at revolutionizing our understanding of the most complex structure in the known universe, the human brain. In fiscal year 17 we leverage our Cures Innovation Funding whether annual appropriation to lunch no less than 110 exciting new Brain Research projects pick some of these will develop detailed maps of neural circuits. Circuits. Others will create a synthesis of the cell types in the brain and still others will create powerful new tools to monitor and modulate brain activity. This will advance efforts to develop new ways of detecting, treating and preventing serious Brain Disorders including alzheimers disease, parkinsons, schizophrenic, autism, traumatic brain injury. With help of the Cures Innovation Fund a Second Research area, the cancer moonshot, is aggressively pursuing an ambitious goal to accelerate advances in cancer prevention, diagnosis, treatment and care. To achieve that we must take a variety of innovative steps and that includes enhancing the Research Infrastructure by creating a Clinical Trial network with an unwavering commitment to data sharing and in the Cancer Treatment programs for what more rapidly. In another of these innovative moves and age joined with the fda and with 12 Scott Gottlieb<\/a> and Francis Collins<\/a> testify on medical innovations. They appeared before the Senate Health<\/a> innovation and Pensions Committee<\/a> on issues including the Opioid Epidemic<\/a>, rare disease funding and research into treating addicts nonmedically. This is an hour and 50 minutes. Good morning, the Senate Committee<\/a> on health and education will come to attention and order. Another bipartisan hearing which is what most of our hearings are which is what bernie and i created on the witnesses. I would say every member of this committee would probably agree on the subject in witnesses today. This is a hearing entitled implementation of the 21st century cures. Progress and the path forward for medical innovation, we will hear from Francis Collins<\/a>, and doctor Scott Gottlieb<\/a>, commissioner of food and drugs. We all have as i mentioned a hearing next wednesday on another important part of the cures act which was the first reorganization of our Mental Health<\/a> laws of a decade which senator cassidy and murphy played a major role in and we look forward to that. Senator murray and i will each have an Opening Statement<\/a> and introduce our panel of witnesses. Each senator will have five minutes of questions. I expect we will have a large number of senators going to this hearing. 2007. Oliver who is in the front row i believe, a Computer Programmer<\/a> began to have trouble seeing and after a near accident had his drivers license taken away and was declared legally blind. The culprit was a rare form of macular degeneration. Doug moved to nashville for a doctor who told him while there were no cures, don could Search Online<\/a> for a Clinical Trial<\/a>. Dunn found a Regenerative Medicine<\/a> Clinical Trial<\/a> in florida and the doctors took cells out of the bonemarrow in his hit , found them in a centrifuge and injected those into his eye. Two days later he began to see. His eyesight eventually improved enough to get his drivers license back and not that he became an effective advocate for more support for Regenerative Medicine<\/a>. The proper way, which we included in the 21st century tours. Earlier this year, the gave me the pen he used when he was blind and said i dont need it anymore. I treasure it and keep it in my office and i thank him for his advocacy and i hope others have the same advantage. Congress passed the 21st century cures act and hoped to in unleash medical innovation and give americans access to treats and cures so more americans can experience it. Its especially appropriate to be having this hearing today because it marks the oneYear Anniversary<\/a> of the Senate Passing<\/a> cures. When it became law last december, i said that president obama had signed a second christmas miracle. The first one in 2015 was the every Student Succeeds<\/a>, aloft fixing no child left behind which came from this committee. Last year it was the 21st century cures act which the leader mcconnell called the most important legislation risk past last year. Ive often said the every Student Succeeds<\/a> that the law is not worth the people paper its printed on if itsnot implemented properly and then to ensure cures is implemented properly we began our oversight on jurors with a hearing in october. With Electronic Health<\/a> records provisions and in todays hearing we will continue our oversight mix wednesday on the Mental Health<\/a> provisions. Today we are hearing from doctor collins and doctor gottlieb on provisionsrelated to Biomedical Research<\/a>. We are fortunate to have two such talented leaders who know their agencies and are widely respected in congress and capable of getting results. That doesnt always happen. Sometimes you have capable leaders dont have any money or authority. Sometimes you have authority and money and the leaders dont have a clue what theyre doing. In this case we got new authority, new money and two of the most capable leaders we could have so we are delighted. While the legislation includes provisions to improve healthcare records, resources to fight the Opioid Epidemic<\/a> in the first update to mental programs in a decade the main purpose was to provide Additional Resources<\/a> and authorities to do drugs through the process and Doctors Office<\/a>s and into patients medicine cabinet while ensuring the fda Gold Standard<\/a> of safety and efficacy. Its not an overstatement to say the 21st century cures act as the potential to affect every american family, taking advantage of breathtaking advances in Biomedical Research<\/a>. Doctor collins had a Senate Appropriations<\/a> subcommittee hearing in 2016, you offered bold predictions, you call them for future Major Medical<\/a> advances if we continued funding the National Institutes<\/a> of health and ensure the fda has the tools it needs. One prediction is that scientists will find ways to identify alzheimers before symptoms appear as well as how to slow or even prevent the disease, alzheimers causes untold family great and causes 259 billion a year. Another prediction of yours was using a patients own stem cells, doctors could rebuild his or her heart. This persons heart would make transplant waiting risk and antidrug obsolete and put senator bill frist out of business. Doctor collins, youve made other predictions because he was a heart transplant surgeon. Doctor collins, you made other predictions. The developmentof an artificial pancreas. The z vaccine, universal flu vaccine. And hivaids vaccine, all available within a decade. New pain medicines to help patients as we continue to battle the Opioid Crisis<\/a> that kills 91 americans every day. You said this week and another hearing that might happen as soon as five years. The 21st century cures law put in place policies that will fund Biomedical Research<\/a> and apply limitless opportunity to help doctor collins predictions the unreality. Today we want to find out doctor collins and doctor gottlieb, those leading the charge how implementation of the law is going. For example, cures included a surge of onetime funding, 4. 8 billion for the nih including money for the position initiative and green initiative. I know from talking with Vanderbilt University<\/a> that the Precision Medicine<\/a> initiative, the plan to map the genomes of 1 billion volunteers to help researchers about cures tailored to a patients genetic environment and lifestyle has already begun and rolling patients less than one year after the program began. In addition to Precision Medicine<\/a> initiatives, im curious about how funding has been spent and when and how you plan to Keep Congress<\/a> informed of your results. Its part of the process to look at burdensome regulations on researchers id like to hear if this is helping researchers spend more of their time on research and less on administrative cakes. Cure has also put in place policies to move effective treatments and cures through the regulatory process more rapidly and at a lower cost. For example, senators berger, and hatch work on a breakthrough revision for devices modeled after a breakthrough provision for drugs but the first breakthrough approval was last week for an exciting cancer diagnostic. Senators isaacson and casey worked on removing red tape from the review of combination products. I worked with senator murray to make sure the fda had the authority to pay scientists keep up the rate of innovation. 21st century cures 30 million to support Regenerative Medicine<\/a> and an accelerated pathway for those treatments fda so we can hear more stories like donna olivers. The fda has been implementing those provisions and i look forward to hearing how fda and nih are working together to make sure the authorities are helping to advance this important work while ensuring that bad actors dont take advantage of the exciting field to harm or defraud patients. Its going to be difficult to cover everything today but i look forward to hearing about the process being made to unleash medical innovation and bring new drugs and devices to patients. Senator murray. Thank you so much chairman alexander and welcome to our guests today. Thank you doctor collins, good to have an opportunity this week to talk to you about the work being done in and at my age. Doctor gottlieb, and looking forward to your updates. Next week marks one year since the signing of the bipartisan 21st century cures act and as i said at the time of the passage and ill repeat it now, as much as this law helps to build on americans tradition of leadership in lifesaving Public Health<\/a> initiatives and medical innovations, cures was first and foremost about providing hope. Hope to the millions of people and families impacted by illness and disease. Hold for communities suffering at the hands of the opioid and like all of my colleagues including our guest today, this is something i think about every day and i visited with communities back home in my home state of washington and have been devastated by addiction. While we can and must do more, im glad that you are is an important first step in dedicating over 1 billion in new funding above and beyond the budget to help states and communities fight back against that crisis and we secured important changes to ensure this money went directly to the states that needed the most. For far too long our Health System<\/a> has failed patients and families seeking treatment and support. That is why in cures we prioritize expanding access to the quality, care for Mental Illness<\/a> and Substance Abuse<\/a> and we strengthen coordination between local agencies engaged in crisis intervention so im looking forward to that hearing we are having this week. On todays topic im proud of our work to address headon of the hardest to treat diseases but i providing nearly 4. 8 billion in funding for nih including support for the bovine cancer moonshot for Precision Medicine<\/a> and Brain Initiative<\/a>s. Both of which are important to my home state where we are spearheading much of that new research taking place as a result. Along with these investments we included provisions in the law to encrypt nih with the tools needed to meet the demands of ill medical research in the 21st century. We made it a priority to include the perversion of women in other underrepresented populations in Clinical Research<\/a> so that the promise of these initiatives by Precision Medicine<\/a> are extended to all patients. As part of this work i champion the provision that creates a task force to support the evaluation of drugs for pregnant and lactating women and we addressed many of the concerns Holding Back Progress<\/a> in the Research Community<\/a> supporting young researchers and 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 qualified individuals who understand the latest science and technology and i am interested to get an update from you, doctor gottlieb on how you are filling those positions especially in light of the hiring freeze was mandated by the president. We made sure cures gave the fda to ensure medical devices like goats would cause the outbreak of antibiotic resistant infections in washington are safe for patients and we gave more clarity to developers of drug device combination products and codify key provisions in the Prescription Drug<\/a> user Fee Agreement<\/a> including to ensure patients are included in the Drug Development<\/a> process. So im glad we have the opportunity to talk about the ongoing implementation of cures and i will be very intent 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 investment needed in nih and fda not to support cures but also to advance 21stcentury science and innovation. In general im glad nih and fda have been timely in influencing the law including many of the provisions i talked about. Im encouraged by these efforts and want us to keep moving in the right direction and doctor collins, in light of the extremely concerning direction the Trump Administration<\/a> has taken when it comes to healthcare especially in regard to Womens Health<\/a> i want to hear more about what nih is doing to bolster your research to further science over extreme ideology. Doctor gottlieb, during your confirmation hearing you said 21stcentury jurors is a good roadmap for what you hope to accomplish as commissioner. Id like to hear about how you are pushing back against his administrations the regulatory approach and making sure cures is being efficiently and faithfully implemented and specifically i will be asking more about what fda is doing to ensure the medical devices patients rely on our safe and effective. Both nih and fda have made steps in meeting cures deadlines this year but we have more deadlines coming up in 2018 and as we know funding remains an issue, i look forward to hearing from each of you to make sure we are putting patients and Families First<\/a> so again, welcome to both of you and thank you for being here. Agency, confirmed on may 9 of this year. Welcome again to eyewitnesses, dr. Collins, lets begin with you. Good morning. Thank you, chairman alexander, Ranking Member<\/a> murray and other distinguished committee members. It is an honor to be a today with my colleague dr. Scott gottlieb. As you well know the 21st century cures act aimed to catalyze a a very important gol shared by all americans to speed the pace at which a scientific discoveries are translated into lifesaving treatments and cures. We at nih actually greatly appreciate your leadership in passing this bipartisan act by a vote of 95four innocent exactly one year ago today. This enhances our authorities and resources in ways that will help us to achieve this goal. Goal. Many provisions are included in the act such as reducing administrative burdens so our scientists can devote more the time to research, expanding our ability toward prizes for exceptional ideas. And strengthening measures to protect patient privacy. In my written statement ive submitted a comprehensive report on at nih has worked quickly to implement the provisions of the act. We are motivated by a sense of urgency to help patients in need of breakthroughs. In my statement of like to focus on the Cures Innovation Fund<\/a>. Among the vital areas of reported research being a celebrated by this fund are the Brain Initiative<\/a>, the cancer moonshot, the Regenerative Medicine<\/a> Innovation Project<\/a> 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. In fiscal year 17 we leverage our Cures Innovation Fund<\/a>ing whether annual appropriation to lunch no less than 110 exciting new Brain Research<\/a> projects pick some of these will develop detailed maps of neural circuits. Circuits. Others will create a synthesis of the cell types in the brain and still others will create powerful new tools to monitor and modulate brain activity. This will advance efforts to develop new ways of detecting, treating and preventing serious Brain Disorders<\/a> including alzheimers disease, parkinsons, schizophrenic, autism, traumatic brain injury. With help of the Cures Innovation Fund<\/a> a Second Research<\/a> area, the cancer moonshot, is aggressively pursuing an ambitious goal to accelerate advances in cancer prevention, diagnosis, treatment and care. To achieve that we must take a variety of innovative steps and that includes enhancing the Research Infrastructure<\/a> by creating a Clinical Trial<\/a> network with an unwavering commitment to data sharing and in the Cancer Treatment<\/a> programs for what more rapidly. In another of these innovative moves and age joined with the fda and with 12 Biopharmaceutical Companies<\/a> to launch the partnership for accelerating cancer therapies. This Publicprivate Partnership<\/a> will initially develop biomarkers to speed the development of cancer immunotherapies, and exciting approach to treatment that unless the patients own immune system. Recently weve seen some amazing responses to cancer immunotherapy 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> which is support of the congress helping to make that happen. The cures act provide support for Regenerative Medicine<\/a> research picked this emerging area of science involves the use of cells and other technologies such as engineered biomaterials and gene editing to repair or replace damaged cells, tissues or organs. As a result, nih as is launchee Regenerative Medicine<\/a> Innovation Project<\/a>. This made eight Clinical Research<\/a> awards covering a Broad Spectrum<\/a> of science and technology and going well beyond the funding specifically provided by the cures act. Some are focus on common diseases including diabetes and vision disorders such as the one that afflicted doug oliver you referred to earlier. While other trained at rare condition such as Sickle Cell Disease<\/a> and idiopathic pulmonary fibrosis. In partnership with the fda we are hosting a major workshop actually right now beginning yesterday going all the way through today, more of the state of Regenerative Medicine<\/a> research involving adult stem cells. We spoke at this workshop at the beginning of it yesterday morning. This comfortable for future Research Directions<\/a> by helping us to identify areas of greatest scientific and therapeutic promises. Finally i want to tell you how thrilled i am you supported the Precision Medicine<\/a> initiative, pmi like including operation and funding. The centerpiece of pmi is the all of Us Research Program<\/a> which will enroll 1 million or more americans from every walk of life. These volunteers will contribute their help of data in many ways over many years to create a Research Resource<\/a> that will catalyze a new era of Precision Medicine<\/a>. This is an ambitious goal and we know nih cannot succeed on its own so all across the nation nih is teaming up with the veterans administration, Health Provider<\/a> organizations, Community Health<\/a> centers, data center at vanderbilt and other groups to think of the best ways to recruit participants especially those that are traditionally underrepresented in Biomedical Research<\/a>. Nih has partnered with five companies to create a Participant Technology Center<\/a> at our partners are testing how wearable devices the ones i went today may provide easy ways for all of us volunteers to Computer Data<\/a> on physical activity, sleep, heart rates and so on. Getting all the partners onboard would eventually impossible at the cures act included other Transaction Authority<\/a>, ota, or pmi making possible for an h to move forward with unprecedented speed and flexibility to carry at beta testing of all the many components and we are now scheduled to launch fully in the spring of 2018. 2018. As someone who grew up in a a theater, and of the value of a dress rehearsal before the curtain goes up. Thats what a beta test is right now. We have enrolled over 10,000 people but when the full launch does happen you enable wilson supported the cures act will deserve applause and if you want to find out more about that, that is the website that will keep you posted. That applause will not just be for all of us but for each of the many, many ways in which cures support the National Institutes<\/a> of health or as some have called the National Institutes<\/a> of hope, and senator murray recent referred to. Speaking of hope let me conclude with a favorite extrication 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. Thank you dr. Collins. I hope sometime during the q a you have a chance to comment on your belief that the other Transactions Authority<\/a> which i have would be helpful in other areas to help us get what i want to go. Dr. Gottlieb. Thanks a lot, chairman alexander, Ranking Member<\/a> murray, members of the committee. Thank you but invitation to testify at this hearing to discuss the implementation of the 20 to cures act. Its a transformative path, set out to optimize our investment in science by how fda oversees technology. He asked us to advance innovation more efficiently while maintaining our Gold Standard<\/a> for protecting patients. This focus on innovation could not come at a better time. Across multiple fields of side we stand at a its election poit in medicine where new technology is creating foundational opportunities to treat and cure disease in ways that were not possible just a short time ago. Take for example, our recent experience with gene therapy. We have seen to recent approvals of therapy for cancer or a patient on are reengineered using the tools of gene therapy to target a patients individual cancer. This therapy represents a whole new paradigm of treating cancer and the early results are changing the way we treat serious tumors. This experience shows how a single fundamental breakthrough insight can open up a whole new way of combating disease. And gene therapy that breakthrough is been the development of vehicles that can deliver genes were efficiently to that target inside the body. These are often referred to as vectors and it taken the form of viruses that specifically engineered for this purpose. In particular, the advent of the specific kind of largely inert virus was an Inflection Point<\/a> in this field. I like in the advent of the vectors to the development of processes for making at the body drugs in making these medicines nearly identical to fully human cells that they were mimicking. They represent a promising field of potentially great breakthrough medicine in the 90s but for a long time these therapeutic drug still short of the promise. That was because these drugs are made with antibodies from mice and the drugs themselves as the rejected by patients immune system. And can besides dehumanize these antibodies so they would more fully mimic the human counterpart. Pretty soon we so many breakthrough drugs result. I hold the field of medicine grew up very fast. I believe were at a turning point when it comes to gene therapy. Over the next several years we will see this approach become a mainstay of treating and probably carry a lot of her most devastating and intractable illnesses. At fda were focused on establishing the right policy framework to capitalize on the scientific opening. Researchers at mit estimated about 40 gene therapies might win fda approval by the end of 2022 from the current pipeline of 932 developing candidates. The estimate 45 of total gene therapy drugs are of total gene therapy drugs are expected to target cancer. I cant do from their estimate but i can confirm the early stages of a transformation medical treatment as a consequences of the technology and the benefits are likely to accelerate quickly. The advances of this field is not riskfree, yet theres good examples about fdas umbrage with the cures act and to build on what congress about is expending our ability to capitalize on this breakthrough innovation. In this case cures provide a pathway for products to receive expedited review by fda. We extended that opportunity. Fda has considered them to be a form of gene therapy since it manipulation of its mate and sells this through gene product delivered by a vector. Fdas new draft guidance on expedited programs for therapies for sues condition fda clarified regions of medicine therapies would include gene therapies the leak of verbal modifications of cells and tissues including genetically modified cells. This would include are two projects with jeans every products they too adorable modification of cells and tissues and, therefore, deliver sustainable effect in the body. For example, if a gene therapy aldus tissue to allow the body to express certain therapeutic proteins or for for car tcells his agency and maintains its present and delivers sustainable through affect we would consider them to be rigid of medicine therapies. By fda taking these decisions it means gene therapies including car key may be eligible for the destination. Next year will building on these opportunities, begin issuing a suite of disease specific guidance document on a development of specific gene therapy products. We intend to lay layout modernd more efficient parameters including new clinical measures for the evaluation and approval and review of gene therapy for different high priority diseases where the platform is being targeted. We plan to focus the first guidance document on use of gene therapy in hemophilia. Other document will address clinical areas where theres a lot of interest in using these techniques such as certain more common single gene disorders will provide innovators with advice and Development Pathways<\/a> including potential excellent approval in point. Gene therapy is one opportunity transforming medicine. This year fda may be on track to put the highest or secondhighest number of novel medicines across a combine biologics and drug centers in fdas entire history. We will approve the highest number of Generic Medicines<\/a> ever and were on pace to approve eyes number of novel medical devices in our modern history. All this year, all at one time. The result of these beneficiaries measured in some important highly novel medicines like the gene therapies but its measured in its human impact. I look forward to answering your questions and on that might call he said if you want to help you for the support of our mission. Thank you, dr. Gottlieb it will begin around the fiveminute questions. My five minutes, i have several questions so let me go through it pretty quickly. Both of you talked about Regenerative Medicine<\/a>. Your testimony has more about it. You are a number of senators including the majority leader who are very interested in it and it helps were trying to get more money and more legislation to have his continued interest. Would each of you in the next few weeks said to me and to senator murray a brief written report summarizing what you have done in the area of Regenerative Medicine<\/a>, the kind of thing i mentioned that help doug oliver . Both about how you create more hope for people like doug but also what youre doing about the bad actors in the field. Id like for it to be the kind of numb i can give two other senators who are interested in this to show that youve taken some action in 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 Transactions Authority<\/a> has been helpful to you . Other Transactions Authority<\/a> allows us to move very flexibly in an area of high need and where technology is advancing rapidly. It means we can bring partners together without taking a whole year that it often takes to issue an opportunity for grants to be submitted for us to do the reviews and make the award plus it 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. This is something darpa has used it were delighted to have for our common fund and for all of us the position medicine, it makes things possible much faster. Where do you want that you dont have it . We could use this in the Opioid Crisis<\/a>. We have a big play because want to move forward quickly. This would be a a great advante to have as one of the tools. Senator murray and senator blunt i i believe that for the third consecutive 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 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. There are 25 million of them in the United States<\/a> right now that happening every day. We need a 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 particularly with ota to help us. And to be able to didnt have available for those people who had that kind of pain, answers and youre talking about using additional appropriations and additional private money and a Publicprivate Partnership<\/a> to accelerate that, correct . Exactly did we would expect companies to put their funds in this just as well as other partnerships with diabetes and parkinsons and alzheimers. Dr. Gottlieb, it takes sometimes ten, 12 years to take a breathtaking new medical innovation from idea to the Doctors Office<\/a>. The whole goal of the cures is to shorten that time and cost. And still do in a way that saves data safe and effective. What are you doing, can you give me some specific examples of how fda and nih and cms, which has to do with funding, are working together so that we dont make a lot of progress in research, for example, have it sit on the shelf while fda works of Something Else<\/a> to make a lot of progress at the fda and have sit on the shelf cms deciding whether to fund it. Dr. Gottlieb. A lot of our ability to make the Development Process<\/a> itself more efficient and lower cost is trying to develop better tools, better scientific tools to evaluate products that are being brought to the Development Process<\/a> to ascertain their safety and effectiveness in more efficient ways, in 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. Theres a lot of work were doing in college with nih in collaboration with doctor francis, a couple of initiative i would point you are the tissue on a chip initiative which is an initiative for developing better tools for toxicology and looking at issues of safety and effectiveness of the partnership for excellent cures which is another collaboration were developing these tools. I think if we can develop this better sides this is the case we can have the best of both worlds, the process of lowcost and more efficient but will give us a better measure of safety and effectiveness, the ability to determine those parameters. I 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. We need to get through the Research Come<\/a> into the fda, through cms and, to make these things work and i hope youll pay a great deal of attention to that. Senator murray. Thank you very much. Dr. Gottlieb, as you know we had some deadly outbreaks of antibiotic resistant infections that were linked to contaminated scopes of my home state and across the country and i champion a provision in the 21st century cures that provide fda with additional tools to make sure the safety of the safety of reprocessed medical devices. I really appreciated the fda meeting its got like to publish the list of devices for which the agency now requires valid evidencebased cleaning procedures. Can you update us on how many products have the validated cleaning protocols because of this law . Thank you, senator. Ill preface my answer by saying we think this is a very important authority and plan to the grow bust use of it. As you make it we put in place the provision of august 2013. There have been deemed products i believe to date that of going to the process outlined in this legislation with many more were having discussions with that i can get shuffled up to date number evolving but its about 14. I want to stay updated on this because i think we do need to respond quickly and appropriately when problems occur with these medical devices patients need to trust whats being used so i really appreciate that. Dr. Collins, i was very proud to chip in provisions in the cures act to improve the inclusion of historically underrepresented populations as i mentioned women, racial and ethnic minority, children, seniors into Clinical Research<\/a>. One of the provisions was a task force to improve the evaluation of drugs are pregnant and lactating women. They are in a very difficult decision often today whether its treating morning sickness or chronic condition because theres so Little Research<\/a> today at how medication might impact them. We all know if they dont get an answer from the doctor they go online. That just to me is unacceptable in todays world. I want to ask you how do you plan to integrate the task force worked into the broader efforts to advance innovation and impact Clinical Research<\/a> . Thank you for the question. We welcome that recommendation in the legislation and moved swiftly to set up this group, chaired by doctor kathy who is a very respected neonatologist and ob she what an expert at nih. That group has met twice and will meet again twice more in the next coming months, and by may of 2018 will have a series of recommendations which we intend to take with great seriousness. As you understand clearly this is an issue where we want to be sure that were carrying out the appropriate Clinical Trial<\/a>s to understand about efficacy and safety during pregnancy and lactation but we also know theres a very sensitive time in terms of not treating risk as a getting the balance just right is what this committee is wrestling with. They have gotten very quickly engaged. I think youll see something substantial come out of it. Good. Im hearing from a lot of women who are concerned about the direction our country is going today whether its familyplanning or allowing employers to deny access to Birth Control<\/a> so they are looking for good information, and then the one of the things we did in cures was strike a balance between members of the federal government and external stakeholders on the task force. Today the nonfederal members have not been hired by the task force and have been able to fully participate and wanted to ask you how you are ensuring that the nongovernmental voices that congress required to be included in this heard from advance and corporate fold into this. We very much want those voices to be critical. There are probably the most critical parts. They have been attending all of the meetings and are speaking. The problem is getting them officially appointed which means going to the ethics clearances. I am assured by the february meeting they will all it got to the process and will be fully installed and able to not just be present but also to vote. Thats really important. Can you keep me updated on that . Id be happy to. Dr. Gottlieb, just wanted to ask, a few seconds left but were living in a new digital age, been mentioned several times and some of the problems are low risk, some of them have big a big impact on patients and their safety. People are wearing wearables that were seeing a lot of different things, apps that use camera phones to diagnose diseases. These are really promising but we have to make sure they work. Their claims are backed up by hard evidence and the note the agency is working to speed that up now. But to me its less clear how fda is going to ensure that the sheer quantity of products now on the market are being validated. Wanted to ask you what action fdas take you to make sure and providers can put their trust in the Digital Health<\/a> product . Quickly, we continue to receive adverse report even for Software Products<\/a> and Digital Products<\/a> that we might exclude from regulation based on the parameters outlined in the cures statute. If there was a situation we received an Adverse Event Reports<\/a> allege a recall, that would be a reason for us to did bring the product back under regulation through the provision in cures because if some adverse event or issue with the software is leading to an adverse event that tells us he shouldnt have been excluded in the first place. Place. We continue to monitor the products we are scoping out over active regulation. 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 diverted senator cassidy. Senator cassidy, we have our hearing next wednesday on oversight of Mental Health<\/a> law that you and senator murphy worked on so that will be here. Wonderful. Im supposed to be at the white house so thank you. This is not an Appropriations Committee<\/a> but that the broad and safety dont mind, dr. Collins, again Great Respect<\/a> for you and for institute but as i pointed out in the past we dont seem to have an nih which targets funding relative to Disease Burden<\/a>. When i look at nida, the budget or the went up last year and a story made are smaller than other institutes in which there is far less morbidity and mortality from those disease conditions. So if we are going to address the issues of opioids for Mental Health<\/a>, both of which are playing into this, it seems like there has to be a greater shift in where our funding is going at in age towards these disease conditions. In the past you suggested what will organically grow, this one a stable and allow this winter grow, but when a look at all to institutes seems to be growing at about the same pace. I question for you is, it doesnt seem that nih is making these are priority if you look over of the conditions, if you look at the amount of funding increase of those institutes. You are a very thoughtful person, so please give me your thoughts on that. Senator, you point a very important issue but how do we make decisions. I happened to point out that its the congress that assigns the budget, its a line item every year in the appropriations process to each of those institutes as an agent director leiter get to set those numbers. We follow what the Congress Tells<\/a> us ought to be the appropriation for a given you. And then we work with great flexibility to try to be sure that when there is a Public Health<\/a> need as it is now for instance, with opioids which i think youre referring to, because of course we have a big opportunity there in terms of our understanding about the brain works, the Brain Initiative<\/a> is relevant. The Neurology Institute<\/a> has an enormous investment in understanding pain as does the National Center<\/a> for coffee maker integrated health. One shouldnt should look at or Organization Structure<\/a> and save the money its precise into those markets. We have lots of ways that we can mix speech i except that if i may. Ive limited time. So youre saying if we want more money to go to National Institute<\/a> of drug addiction we need to line item in our budget. That the only way it happens. That said, the flexibility to seem as if it should be flexing towards things like nida. Pain is important but ultimately paid translate into addiction. Thats the final common pathway. So does seem as if we should be flexing towards them is that when a flexing is taking place . I think thats what i did is to ship with the Party Opportunities<\/a> we have mama more funds in that space because we recognize this this is a terrie Public Health<\/a> emergency. Let me ask a different topic again trying to stay on time. There is a move afoot to suggest marijuana usage can be used in lieu of opioids that states that have legalized marijuana more liberally have lower incidence of opioid addiction. Thats right. But then i read about the brain being pretty plastic up until age 25, and so well know the pothead, the kid that is a Motivational Center<\/a> make you will. So theres been at least one suggestion i read that we should make a recommendation the 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. There is published data although it still controversial that heavy use of marijuana beginning in adolescence does have permanent consequences in terms of intellectual performance. 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 reduce incidence of opioid overdoses and deaths. One has to be careful. Thats a correlation and that message is a causation. I would not want to leap to that. That. I would go to the point that we are increasingly studying the cannabinoid receptor pathways in the brain as a potential whether we might come up with alternatives that would be affected for managing pain and depression and anxiety, not marijuana itself using that pathway. But for the two of you is it reasonable Public Policy<\/a> consideration perhaps the age of Legal Marijuana<\/a> for those dates that are legalizing should be 25 and above . Were giving it a difficult policy territory. I will simply say the concerns about marijuana exposure to the developing brain would have to be strongly considered in anything that might access to adolescence more readily available because we do have that concern. And the brain is developing at least 2h25 . You could say 22, 25. I dont know if i have my wife says its 60 but im out of time. I should yield back. I apologize. Thank you, senator cassidy. Senator casey. Mr. Chairman, thank you very much. I do want to add my words of commendation to you and the Ranking Member<\/a> for the work you did to get this legislation passed. It is hard to believe its a year, and innumerable ways to go with regard to implementation but in a place and in an institution where there are not many days where you have, not just bipartisanship, but i 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. Collins, i wanted to start with you on a question regarding 503b confounders. While we primarily been focusing in the hearing on getting new drugs to patients, im also concerned about maintaining the supply of drugs that are already in the market. We know that since Hurricane Maria<\/a> hit puerto rico, ive heard it from pennsylvania regarding shortage of iv fluid and amino acids for injection of critical product for patients who must receive nutrients intravenously. And while puerto ricos 1500 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 and facilities on the eyes and for the sole source of the party for the entire country. When you mentioned Continuous Manufacturing<\/a> and how they can help reduce drug shortages, i wanted to ask you, could 503 the outsourcing facilities also in the case of drug shortages . The short answer is yes. We remain uncertain about the shortage situation in puerto rico, taken as a relates to the iv kludge reference, the amino acids. We think the steps weve taken in concert with the manufactures and authorities in puerto rico was not to alleviate the shortage situation quite a connection but we still have to get through december and we stuff is another hard month what is going to be challenges getting access to adequate supply but we do expect this to continue critical and forward. The issue with the compound is, they theoretically can compound the spotted pig who have authority to do that. One of the issues is the parts that arent shortage are not just the iv solutions themselves but the actual physical plastic bags to put the iv solutions in. So the 503 503 ba compounder ss a challenge getting access to the plastic bags a need in order to compound the product. Those are also mentioned in puerto rico. That has been a challenge and where she wouldve expected the 503b facility to step into someone more of this market i think theyve been limited in their ability to do that. Wanted to ask you as well, i know i have limited time, but with regard to the pediatric review voucher program, we know an estimated one in ten people in the United States<\/a>, twothirds of whom are children, have rare disease according to the National Organization<\/a> for rare disorders, despite the need five companies are less likely to pursue new therapies for rare disease because it requires making an investment in products that will likely not recoup the high cost associated with the Research Development<\/a> marketing and distribution. You will remember i i work 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 rare pediatric diseases. When we last spoke, the fda were still working on implementing changes that were made to the program. Most informally, the definition of what constitutes quote rare pediatric disease, unquote. Can you give us the sense of the product you have made on the update of that definition . Are you reading the statutory requirements to respond to request for Companies Seeking<\/a> designation for the products within 60 days . I know thats a lot. Im proud to say we are, senator, and we put forward some guidance. Theres Additional Guidance<\/a> coming out in permitting the prv rocher. We continue to look at this as a potential opportunity. We are awaiting the gao report as are others to better anybody these. I will just close by saying theres a lot of other things that we can and are doing to try to create additional efficiencies to address some of the challenges that you outlined with respect to pediatric Drug Development<\/a> including one we took yesterday. I want to put a plug in for policy yesterday trying to outline a more efficient pathway for drugs targeted for very rare pediatric diseases. Thanks doctor. Thank you, senator casey. Senator young. Doctors, i have a lot to cover in five minutes so i i ak that you try and be concise to these questions so thank you for your presence here today. Try for you mention in your chest when the pledge of any limiting your recount competitions eureka competition ensures that price program. When i was in the house of representatives put together an amendment to cheers, a variant cheers bivariate, the eureka prize provision i know some of this contributed to that effort as well. It was inspired by a neighbor of mine who is a latterday thomas edison. He develops medical devices in his garage. I consulted with eminent economist, healthcare professionals, researchers, venture capitalists, angel investors, and all sorts of individuals in developing my form of prize. The objective was to ensure that we have objective rather than highly subjective criteria with respect to what those prizes are offered for. My hope, something particular by one of my colleagues here today, was that we would target funding according to Disease Burden<\/a>. And when my colleagues consult 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 used to determine what areas you are going to be funding . With acknowledgment that we funding alzheimers now. That strikes me as meritorious but what criteria do you use . Appreciate the question. By the way, at every center displays this booklet which is published this week will walk you through a lot of responses to that, how do we side decidee 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. When you see an opportunity that stickley right for investment we dont want to miss that. With the price mechanism we just put out a request for ideas about prizes for alzheimers disease. Talk about it and i Disease Burden<\/a> to go looking for to see what that would look like. Enterprise right now thats waiting for responsive and a string of a lot of interest. At the microbrewery assistant, came up with a testable tell you within four hours to all the criteria are located in the book you just held up, is at the case . To go through quite a lot of it. If you need more information i can supply you. I thank you for that, for publishing that information. You include all you 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 panel, especially for High Technology<\/a> efforts repopulate them from travis sector who have that expertise. We want to see what people think in all sectors about an alzheimers prize. And lastly do you allow in your prize competitions or have any plans to offer inkind benefits . And by that aiming the ability for a garage investor or researcher to use lab space at 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 weve done and a couple of the small projects, prizes where we are asking students, undergraduates have a great idea how to be able to apply and they may not have access to the equipment. We are trying to very flexible on that. Thank you. It sounds as if the career in h folks that were telling members of congress that are ideal is not thought well enough, perhaps a bit on the right track. So, dr. Gottlieb, pharmaceutical Development Today<\/a> prettily involves whats been called shooting in the dark, making key decisions that impact the Development Program<\/a> success with very limited data. For example, decisions including indication selection, and points and inclusionexclusion criteria. You might say ive made some optimally because the data that informs such decisions lies in an accessible silos. At least an accessible to most stakeholders this contributes significantly to hire r d costs and longer timelines. The fda already has much of this data, this otherwise siloed information. So my question for you is has the fda taking any steps in aggregating healthcare data 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 for the scientific insight back to the Research Community<\/a> to accelerate in the risk . I appreciate the question is a complex one and a delighted to followup with you on it, but the bottom what is there are situations, first of all well be taking steps very soon to make more data from clinical portion of the review to improve that where they can to aggregate into the data set you speak of. There are situations where we develop our own proprietary data sets of data weekly to make decisions particularly about drug safety but those data sets remain proprietary to the fda because that information is commercially confidential. We are looking at how we could make these data sets public without appropriating anyones intellectual property rather outdated because my belief is if we are making decisions on the basis of aggregator data that is acceptable acceptable to the public that something we should try to address. This is a complex that were actively working on. Suffice to say where trying to move in the direction that you suggest. There are legal complexities. You are out of time. 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 one Year Anniversary<\/a> of one more reason to be happy to be on this committee. Trent lott i would if you give the committee an update on where you are with the implementation of the med tech act, bill senator hatch and i worked on together . I appreciate the question and i will try to be briefing we Just Announced<\/a> major components of implementation of some of those provisions today. We continue to move forward with this on schedule. I think its a good example what were trying to do in this space. Its a good example of what return to generally with cheers where we are taking privations that congress outlined and try to take the spirit of what our set out to achieve and try to go beyond that. So, for example, conversation out to create a more sufficient process for review or to exclude certain decisions support tools used by clinicians and the announcement we make today we are also going to exclude certain decisions to support tools, Digital Tools<\/a> used by patients as well as long as they meet certain parameters. Thats an attempt by fda to try to take the spirit of what congress about to do and extend it a little bit. What sort of things you expect to see sooner rather than later in terms of new devices for patients . One area of development we havent seen as much of is in the area of Decision Support<\/a> tools where there is tools i could take information can help support decision without making a decision for the clinician of the patient. In part theres a lot of reasons why we have seen as much innovation in that space that you wouldve thought but ambiguity probably played a wel for a time. Fda always intend to exercise Enforcement Discretion<\/a> in this space. Congress expressly outlined that in tears as of this provides us the opportunity to put out guidance that great some really bright lines in primary care what is, what does and what does it cost a lot and what is the obligation, even those were excluded from active regulation to make sure into unit to look at the sake of the products and collect information with can go wrong. I am hopeful we will see more innovation in the space, tools they considered top top of Electronic Health<\/a> records and help physicians make decisions on that information. I appreciate your focus on it. I also noticed that unrelated to that, the fda approved the fit breakthrough designated diagnostic test that detects genetic mutation for cancer. That was an approval to came to i think the new breakthrough, the breakthrough device section that we wrote for the bill in this bill last year i was interested to see at the same time that happened there was 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 ridges and whether we can expect to see this sort of coordination in the future . I think you can and this is an example of a test that voluntarily came through the regulatory process. We are able to review it in six months under the breakthrough designation first as you said, breakthrough designated products receive fda approval. We worked with cms to have in place simultaneous coverage at the same time. We think Going Forward<\/a> this is a panel, cancer margins 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 now and what cms said in the policy and a let them articulate it more clearly is that panels that come through fda for voluntary approval now will automatically receive coverage of National Coverage<\/a> determination. Its a powerful incentive i believe are more such test to try to come through the process. We can provide i hope a greater assurance of the effectiveness of it. I think thats a big step forward so i can tell dr. Collins degrees. Dr. Gottlieb, last question, only a minute left. Senator rubio and i worked on the race act for pediatric cancers and i know you guys put out some guidance recently on developing drugs for rare pediatric diseases. I wonder if you could expand a little bit about that . We did and will have more to say on this area but what we did yesterday was finalize parameters and guidance that we had in draft form that tries to look at very rare pediatric catchers. Pediatric diseases, excuse me. Trying to outline a more efficient process for developing products that we might have life as much on placebo trials, might use simulation 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 accommodations are important in the setting is because its hard to enroll patients with these very Rare Diseases<\/a> or you might have dozens of patients who have disease were eligible to be enrolled in Clinical Trial<\/a>s. Mr. Chairman, im out of time but i didnt think the witnesses for their for the Great Service<\/a> to this country, and i deeply appreciate it. Thank you, senator bennet. Senator collins. Thank you, mr. Chairman. Dr. Collins, i am always glad to see you and i still cling as my cousin regardless of the facts. I accept. Thank you. The state of maine is doing some really exciting medical research, and i visited a lot of the labs and medical institutes that we have in our state. Maine Medical Center<\/a> Research Institute<\/a> is contacting cuttingedge work that researchers in maine have underway to develop medical treatments for chronic diseases. And in september 1 of the researchers with whom i met, doctor oxford, and his team at maine Medical Center<\/a> received an award to the nh, Regenerative Medicine<\/a> Innovation Project<\/a>. Using adult stem cells, doctor oxford is working to develop novel therapies for Chronic Kidney Disease<\/a> 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 come to congress on the state of maine and group only 11 miles from where i did and he shared his compelling story about losing and regaining his site through regenerative therapy. Mdi biological laboratory in maine is linked 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 applications to Regenerative Medicine<\/a> as we are gathered this morning as an intense discussion going on at a hotel in bethesda sponsored by fda at nih jointly on this topic of Regenerative Medicine<\/a>, several hundred in the room talking about applications to kidney disease, heart disease, the aging come to such things as Rare Diseases<\/a>, diseases, polanski. It is our most exciting to see a that is taking place when i spoke yesterday i even forecasted if we do this in a way i think we could we cant cure, not just treat the cure a disease like sickle cell in the course of the next five years. With regard to the question that aging, we are learned about itt the process is. I will need to an example of the whites are a long lecture. That is that as you age is a certain category of cells that are no longer able to keep going, they dont completely exit the stage. They are still there. It turns out they are not good actors. They are making a a healthy ces are about the not so healthy. If we could figure out as weve done in mice have those who basically told okey, you are done now, it could vary significantly slow down the normal process of aging with all that that entails entrance of frailty and chronic disease. This is an exciting new development. I think it is also, when i remember visiting Harvard University<\/a> in talking with researchers and their about alzheimers disease, and they are 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 did know about before by very careful system biology approaches to understanding ad networks in the brain work in normal individuals and those are at risk for alzheimers. We have partnership with industry you may part about called the accelerating Medicine Partnership<\/a> which is advancing this at a pace i would not affect possible for a 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 where really pushing one is biomarkers. We really need to find out what are the indications that therapies working without having to wait ten years to find out whether it was, in fact, protected against the development of cognitive decline. Its a lot going on with various types of imaging for windows proteins involved in this. The other part is the biology where were trying to step away from what we know already and ask the question what else is going on in the alzheimers brain, and how can we use to develop the nextgeneration of therapeutics taking advantage of all of the areas we are now learning about to come up with interventions that are going to work. Then apply those early before people have begun to succumb to the illness. Weve got to start early if you want to get a good effect. Thank you, mr. Chairman. And thank you, dr. Collins. Thank you, senator collins. Senator murphy. Thank you very mu ch, mr. Chairman. Thank you both for your service. I know it comes with great sacrifice. Dr. Collins, as a connecticut resident i know the sacrifice you make your computer dr. Collins, wanted to talk to you more about the all of Us Research Program<\/a>. This is a program that designs to build a diverse biomedical data net for the Precision Medicine<\/a> initiative at an interest in it because one of the participating Health Centers<\/a>, Community Health<\/a> centers that is gathering data is based in Middletown Connecticut<\/a> and they i think is great that you got out and were quick unity Health Centers<\/a> because the centers, you typically serve a Patient Population<\/a> that is often underserved in Biomedical Research<\/a>. I just want to put that question to you, which is if you could talk about the importance of having this as part of your initiative and whether there are plans to expand the on the current six Health Centers<\/a> that are part of this initiative, Community Health<\/a> center in connecticut, its very please with a partnership with you on this and would love to see more like them get involved. [inaudible] opening remarks i put up this visual image about how it is that we are seeking to enroll participants in this very bold largest ever prospective study of health and illness in the United States<\/a>. We are inviting people to participate my upright of needs. One of them is to be Community Health<\/a> centers here include in the Health Provider<\/a> organizations because we have a specific goal of having at least 50 of the participants in all of us being individuals that are underrepresented traditional in medical research programs. In the Community Health<\/a> centers that provide care, particularly to those in lower socioeconomic issues are very excited about being our partners and we are excited about having to miss partners. Were glad about middletown as a place that that is clued been y 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 we are in a beta test. Weve enrolled about 12,000 individuals. We are learning over the weekend about how these moving parts can Work Together<\/a> and is looking very good. Dr. Gottlieb, want 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 Call Center Team<\/a> to Work Together<\/a> on it right of products to treat cancer. Just an update on what you have learned thus far from this model, and what do you take into account when youre thinking about creating other disease specific offset . I appreciate the shout out. This is an extremely important effort on the part of the agency. I appreciate your asking the question the Oncology Center<\/a> of excellence what we try to do is consolidate the clinical portion of the review of products into one combined center. This was instrumental in our ability to expedite the review by consolidating the complex, more official look at some of the biologics. We had challenges fully stand up and on all candidate related to fully funding it. We believe this is the future of the agency, try to get these consulted programs in place and looking to other therapy areas we can do this. I think before we can progress of the therapeutic areas we need to make it work in the oncology setting or when looking for to continuing to try to move this program forward. Great. Another reminder with got to give you the funds to implement this act. Quickly back to you, dr. Collins, one more question, that is on another part of the point for 20 for signatures act senator isakson and i worked on a provision that allows for the cdc to collect information on the incidence and prevalence of neurological diseases if this wasnt advancing research for neurological diseases act that senator alexander at senator murray helped us include in this legislation. Talk about the importance of having this data on neurological diseases to Research Done<\/a> at nih. Is incredibly helpful to know what incidence and prevalence is for neurological conditions, particularly a condition like parkinson but their many other disorders as well. It is challenging and expensive to do that analysis and to do it effectively and to keep it updated. As i understand it while cdc was given the charge to do this, they do not appear to have the funding to carry it out. Great. Another advertisement to put the money behind the legislation we pastor thank you very much, mr. Chairman. Thank you, senator murphy. Senator hatch, former chairman of this committee, welcome. Thank thank you so much, tryi appreciate. I certainly appreciate you to judgment. I think youre both some of the best Public Servants<\/a> ive seen in all of my 40 plus years in the United States<\/a> senate and im proud of both of you and i hope youll just keep doing what you were doing. So i i want to thank you both e today. Dr. Gottlieb, last congress i join senator bennet and authoring the past act which was included in the 21st century cures and will help advance the department of urgently needed new antibiotics for multi drug resistant infections by allowing them to be studied in smaller more rapid Clinical Trial<\/a>s and, therefore, approved more quickly for the patients who need them the most. As you fda was tasked with developing guidance for industry regarding this limited population pathway, or help add. Can you please detail the ways in which Stakeholder Input<\/a> is being solicited and incorporated into the guidance . Thank you for the question senator. With an issue the guidance that you referenced in the spring and weve been meeting with stakeholders and other interested parties in developing of that kind guidance. Consistent with good guidance practices so we will have stakeholder meetings and development of any guidance document. I will just say we think this is an excellent important pathway and prevent multiple meetings with sponsors and believe thisll be a robust vehicle for trying to trade new pathways for drugs targeting significant unmet medical needs. Dr. Collins, i was pleased to see as result of the 21st century cures and h was able to start a Regenerative Medicine<\/a> Innovation Project<\/a> that several of the first awards from this initiative had gone to research on Rare Diseases<\/a>. Ive been a champion of the Rare Disease Community<\/a> for quite some time and i applaud you for also recognize how Vital Research<\/a> in this area truly is, strides in emergency may prove to be transformational in the way we understand possibly even treat Rare Diseases<\/a>. What barriers do you foresee an expanding research into this area as to Regenerative Medicine<\/a> Innovation Project<\/a> continues . Thank you, senator for the kind remarks at the beginning of your time and also for the question. I think this is an enormous exciting time for Rare Diseases<\/a> because we have tools that are starting to work. Dr. Gottlieb refer to go to a gene therapy. I couldnt help but point out today in the new england journal of medicine really dramatic advance in hemophilia using gene therapy with a viral vector to deliver the gene to deliver a mentor affected. Its because its a condition was really remarkable benefit over the course of many weeks. Thats just a single example of whats possible in the space. It results in children being born who seem to be okay and then develop paralysis and often dont live past their first year. With the Clinical Trial<\/a> recently published, some are making it too kindergarten making it pretty good due to a gene editing strategy. I think the big barrier is to just push the science as hard as we can. 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, working with scott and his colleagues who have also made rare disease a very high priority. There is this issue of investment. Companies may not be so interested in putting a lot of money into a very rare disease because the market will be small which means they havent Even Stronger<\/a> responsibility to do risk those projects and push them as far down the road as possible. One more thing i would say that you did for us and the cures bill is to 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. Thank you. One of the provisions of the 21t century cures was designed to improve opportunities for young and emerging researchers. Ive heard from the university of utah which received around 150 million in grants and they have seen an improvement in this area. For that 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 senator hatch. Senator baldwin. Thank you. I want to add my words to the others of congratulations of the bipartisan work you lead mr. Chairman and Ranking Member<\/a> murray on the 21st century cures act and think both of you, doctor collins for your service. During the Committee Deliberations<\/a> on 21st century cures, i regaled the committee a number of times about being the granddaughter of nih funded scientists and how that influenced my champion strengthening our Research Enterprise<\/a> and support for the nih. In my first year in the United States<\/a> senate in 2013 i had a particularly powerful and inspirational meeting with the young man, a high school senior. His name is ian and he is a bone cancer survivor from wisconsin. Ian told me cancer research, no doubt, help save his life, and thats why he wanted to grow up to be a scientist, to help others with his disease. 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 too author the next generation researchers act along with my colleague senator collins to fight to improve nih opportunities for new and earlystage 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 osteosarcoma and he is now working with the scientists at Huntsman Cancer Institute<\/a> in Salt Lake City<\/a> utah on pediatric cancers. Doctor 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 to issue more awards to investigators in the earliest parts of their independent Research Careers<\/a> to help them sustain or achieve research independence. I would like it, doctor 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. Thank you for the question. You are touching on a personal passion that i feel and many of the other Institute Directors<\/a> do as well. We actually just published, about a month ago, myself and mike lauer and larry, a description of what this policy is and how we are 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. With the loss in purchasing power sustained between 2003 and 2015, those first time earlystage investigators were having a hard time Getting Started<\/a> and we cannot lose them and we were starting too. Each one of the institutes depending on where they fit, they can find their flexibilities and enough 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 bunch of awards that otherwise would not have happened and were not done yet because we are 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 he would be interested in because it has graduate students and junior faculty, not just the gray eminence but people who are really involved in us and we will have a deep discussion about whether we have the right plan in order to make this particular priority really happen. Im running out of time, but i just want to note, you estimate 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 ensure that it fulfills promise and continues to advance all other critical nih effort. Very quickly, we are prioritizing this within our existing resources but of course we can do more of it if the resources are available. Before we go to senator warren, let me recognize senator murray. Thank you very much but i want think both of you for your answers. I will be summoning more questions to the record. I have another commitment i have to make but this has been excellent. I really appreciate it and we have another one next week on the Mental Health<\/a> cures. I think its important for all of us and i appreciate your efforts and look forward to working with you. Thank you. Senator warren. Thank you, mr. Chairman. We been talking but the cures act. It has a lot of bipartisan provisions and im really glad they became law. During our recent hearing on jean editing we discussed new genetic protections for Research Participants<\/a> that the senator and i have worked on together. Right now massachusetts Biotech Companies<\/a> are benefiting from a provision that senator bennett and senator burns and hatch and i wrote to try to clarify the fda authority and we are waiting for gene therapy. They fell short in a really big way and thats on funding. I blame them caps on this committee and 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. Doctor collins, let me go through this a little bit. Does the nih funded most of the grant applications that it receives from scientists . No, we certainly arent able to do that. We fund about 19 because thats the way it comes out after we do the priority scoring to see how much money we have. So out of 100 applications you get, you are funding about 19 of them. 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 around 2001, we were funding about 30 , maybe even 35 because funds were more available. We looked at those to see, did a grant that scored at the 25th percentile turn out to be less productive than one that scored at the 15th percentile . The answer was no. We couldnt tell a difference up to about the 30th percentile. Theyre trying to draw a distinction but its tough to do so in the top third. So roughly, if we doubled the number of grants we were able to fund you think theyre still a lot of good science. I think there would be a lot of good science. That is very important. I want to follow up on the point that senator baldwin made in the discussion you made about researchers in the early part of their career. 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 they have to leave academic medicine and go somewhere else. I just want to ask more about the details. Where are we right now on early career researchers. What percentage of the grants are they able to get . Bubeginning in 2008 we actually instituted a policy so those applicants who came to us for the first time got a bit of a boost. They competed against each other opposed to the most experienced ones which meant effectively they got a few extra points, but thats not good enough. We are still losing lots of those in their success rates for those earlystage investigators are still well below what you would want to see. Ultimately, we think it would be most healthy if at least 25 of those applications were going to get funded and thats what were trying to do with this new initiative, the next Generation Initiative<\/a> which is named specifically for the words that were used in the bill. Thank you senator baldwin for that encouragement. I understand right now weve been at about 16 . Is that right . At a minimum youre saying we ought to boost that to about 25 . And we looked closely at every Institute Success<\/a> and tried to figure how we can get there with this new policy. I know that nah has done what it can in this area, but nih funding is still down about 15 of where was a decade ago. That was back when we had a 50 higher success rate for the proposals that were coming across reviewers desks. The cures acted not solve this problem. It did not even come close. Thats why today we are reintroducing the national Biomedical Research<\/a> act which provides 50 billion in new funding for the nih and for the fda. I see you are setting up straighter there. This legislation is cosponsored by senators sanders, casey, bennett, white house, murphy, kane and hasson, all members of this committee as well as several of our democratic colleagues were 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. Thank you, mr. Chairman. I will add my thanks to you and the Ranking Member<\/a> of this hearing and for your work on the cures act two doctors collins, thank you so much for being here. Along with us, thinking the two of you for your service and leadership, please think the women want 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>. I wanted to start with a question or two for you. We had discussed the devastating impact that the Opioid Epidemic<\/a> is having a New Hampshire<\/a> and all across the country. One of the tools in combating the epidemic is medication assisted treatment like. [inaudible] and plays an Important Role<\/a> in recovery along with access to other services in sports. Both of you also 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 becomes addicted. The 21st century cures act took some important steps on patient focused Drug Development<\/a> including requiring guidance on Patient Experience<\/a> data. Can you discuss how patient focus Drug Development<\/a> initiatives could be used in relation to developing new medication assisted treatment and the development of new nonaddictive pain treatment . I appreciate the question. The bottom line is they can be a very important tool in the provision in the cures act would help inform how we go about thinking about the parameters we use to encourage the development of products. Both kinds of products. In particular, medication that can assist people to live lives of sobriety. We have committed to provide new guidance documents for outlining the pathway for the development of medical assisted therapy. We want to create incentives for sponsors to develop those products and part of that is making sure we have very clear guidelines. As part of that we will be looking at alternative endpoints that we havent looked at in the past. For example cravings. We know craving is a factor that leads people to continue to use opioids and if that can be an endpoint in the trials that we used to assess new products and might provide a more efficient pathway. That certainly has informed by the work we do with patients and looking at patient informed endpoints. Thank you. Doctor collins, i wanted to talk to you too but you made a comment in response to senator alexander about the impact of the other Transaction Authority<\/a> that you now have. Can you comment a little bit about how that could be helpful in the opioid space bar you said it would be but how specifically would it be useful to the work in doing opioid Disorder Research<\/a> . So the plan that we have put in place which is going to be flushed out in more detail next week in an intense meeting will involve a lot of rapid action both to come up with new ways to treat addiction because we knew 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 will require partnerships between academic institutions with the appropriate science skills with industry that may have formulation capabilities or Drug Development<\/a> pathways come if we have to build that on the process of issuing grants and contracts and the traditional way, it is going to take a long time. Other transaction Authority Allows<\/a> us to do something in maybe six weeks that might have otherwise taken nine months. Weve learned how to use this effectively and responsibly for the program for the common fund and we think that would be a goo great place were to be available. We will be careful about how to use it but it could help us a lot. Thank you. Finally, i wanted to talk a little bit about our new generation of scientific researchers because both senators and baldwin and warren were talking about that. Cures took important steps for the next generation of researchers by helping to make it more affordable first Prospective Students<\/a> to pursue their graduate degrees. Nih has been responsive to these efforts and in september announced a new policy for implementing funding considerations for the students. In recent weeks ive heard from a number of graduate students who are fearful of how the republican tax legislation, particularly the house version of it could impact their ability to pay back their student debt and whether it could even force them to drop out of their programs. One particular provision in the house bill would require that graduate waived tuition is taxed with their stipends. A change that ive heard from Phd Candidates<\/a> could really impact, they wrote this would triple my taxable income and increase my yearly taxes by more than 10000 credit would force me too drop out of school because i wouldnt be able to afford rent or groceries while earning below the poverty line. Can you comment, doctor collins, on what you think this provision would mean for the future of our researchers. These are our future graduate student centered learning to be the next generation of leaders in science across many disciplines but certainly lifesciences needing those individuals to be the ones to make the next breakthrough. 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. Thank you. Senator whitehouse. Thank you very much chairman. I joined my colleagues in congratulating you and Ranking Member<\/a> murray on the success of a year ago. I hope we have more to come. Doctor collins, one of the things we got into the cures act was that a request that you reduce the burden of what we call low risk sub recipient monitoring. I just left the University Director<\/a> of research a moment ago and this was also the university of Rhode Islands<\/a> request, you get things Like Brown University<\/a> working together on a project and the supposed obligation to monitor their subcontractors now applies to each of them. So, it theyre both monitored. [inaudible] can you help reduce the bureaucratic and reporting burden to your sub recipients . Senator, we dont particularly enjoyed doing that kind of lotus monitoring, and we have, in fact, put together a proposal of a way that we might reduce the amount of low risks of recipient monitoring. There are certain situations that dont quite meet that but many of them do. Will you include us in that cycle so we can be helpful in giving advice and making sure that goes forward at a good pace. Will be happy too. The cures act was very good at getting you, the fda to connect the device and the drug side a little bit better and youve done a lot of training to try to connect the two. The original testimony that we had from fda from both the drug and the device side was that what you really needed was a third pathway for drug device, nations. Would you assure us that if it turns out that the highbred 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 time for us. We will be implement in those provisions and really have some guidance pretty soon that we think will articulate a better pathway. If it doesnt resolve the challenges that youve identified in the past i would absolutely be delighted start to at alternative. Just so long as we are in the loop and not wasting time so long as we can continue to be productive. There have been some everyday moments that are very significant. Back in 1876, a gentleman called his assistant watson to come into the room and in 2012. [inaudible] it was a pretty minor thing except for the technology involved. Ms. Hutchinson picked up the coffee and brought it to her lips through what at Brown University<\/a> they call the brain gate which is electrodes in the brain so that by mere thought she could goin control the robotic arm. The Brain Technology<\/a> that non nihs funding is traffic. Are you adequately connecting with them to make sure everyone is going in the same direction. Yes, we have worked quite closely with darpa in this whole idea with how you could begin for people who are paralyzed to control a robotic arm to carry out sophisticated activities including the ones you mentioned that this is a big part of what the Brain Initiative<\/a>, which involves nih and darpa and nsf as well as the department of energy aims to do. They want to figure out how those 86 billion neurons between your ears do what they do in these complicated circuits which we are beginning to take apart and understand further. We want to really understand it so well that you know exactly where to place electrodes to get the maximum sophistication of the move of that robotic arm. Last topic, weve just done some good Bipartisan Legislation<\/a> on plastics in the ocean here in the senate. The un has just issued a unanimous statement of concern about plastics. Plastic, as you know doesnt biodegrade, it just breaks down of the smaller and smaller and smaller point. Then microscopic creatures can consume it and begin to track up the food chain. Never in Human History<\/a> have we had to experience that kind of plastic loading into our diet. It is now found in cap water, its found all over the place. Its obviously going to be in our food. Is this something the National Institute<\/a> of health should begin doing basic research on so that if this turns out to be a problem, its certainly a new experience for humankind to have to digest and process that kind of microscopic plastic in the food chain that you are alert to it. Absolutely. The National Institute<\/a> of health sciences, one of our 27 institutes but the one thats located in North Carolina<\/a> is already looking at the sprint i can give you a summary of where we currently stand in terms of the research being conducted. I would appreciate that. We can do that offline answer your staff. Thank you chairman. Thank you senator whitehouse. Thanks to both of you for being here today. I hope you will follow up with the memo on Regenerative Medicine<\/a> in the next few weeks that i can give to senator mcconnell and other senators interested in that. I know you have quite a story to tell. Also, will be interested as we look fo forward to the second anniversary of the cures act, especially taking that ten or 12 years between idea and something in the medicine cabinet, something in the Doctors Office<\/a> and howard compressing that in setting priorities and how fda, nih, cms are working together to do that. I salute you for the step severity taken, but that would be a very promising way to do things. Really, you have to do that more than we. We can do some things in legislation, but that one we really cant do as well. We can order you to do it but that wouldnt make you do it. You have to do what youve already started to do which is to Work Together<\/a> to speed that up and do it in a way that provides safety. Without getting you in the middle doctor collins, i sometimes worry that the messages that come out of washington art heard by the graduate students and the researchers across country and they worry that 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, nobody knows it doesnt happen. I remember when president obama last year, he actually reduced recommended funding, discretionary funding for the National Institutes<\/a> of health and he recommended a big increase in mandatory funding for the National Institutes<\/a> of health. This isnt too hard for people to understand. This is the authorizing committee. We dont appropriate money usually. We have another committee which senator murray is actually the Ranking Member<\/a> of and that is the money committee. So, for us to appropriate 50000 for the National Institutes<\/a> of health is a wonderful aspiration, but thats not what we do. We decide, for example, whether the doctor should have a new breakthrough path for medical devices. If the Appropriations Committee<\/a> were to decide that he should have a breakthrough device, we would be very upset because we would say thats our job. If we were to try to appropriate another 50 billion for you, the Appropriations Committee<\/a> would be very upset because they would say thats our job. Let me ask you this. We have discretionary funding, that the preparations committee. We have mandatory funding which this committee can do, what is the size of the nih budget this year. What is your total funding. 34 billion. , to that is Biomedical Research<\/a> be met virtually all of it. To be clear, more than 80 of that goes out to all those institutions all of the country that do that. , to that 34 million is mandatory funding. I think theres 150,000 in their. So almost all of it is money that is supplied. [inaudible] through the subcommittee that the senators do. Now, two years ago, if im correct they recommended a 2 billion increase in funding. Last year they recommended in congress agreed and they signed another 2 million, and if im not correct, i believe the committee has recommended another for the third year in a row the congress has not backed it. The way we normally do things here is once we put something in the budget we counted for ten years of the first years 2 billion, thats 20 billion over ten years. The second is to billion over ten years and the third one if we do it another 20 billion over ten years. Thats not guaranteed, but thats usual what we do and we put it in the base and we expect to do it again. The priorities we have on this committee and the Appropriations Committee<\/a>, i expect that to be the case. Then in addition, we did something very unusual. It was very hard to do. We took 4. 8 billion, created a hybrid funding that created all kind of. [inaudible] with the house of representatives representative. 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 been doing a tremendous job over the past few years. I say that, im a member of it myself but ill give senator blunt and senator murray great credit for squeezing out the budget sources which are good deal larger than in the last few years. Nothing wrong with aspiring for more. Theres nothing wrong with looking back 15 years and seeing if we had gone up at the same rate we had gone up it would be even higher today. I think its important for researchers across the country to know that this congress, in a bipartisan way, for the past three years has been extraordinarily committed to significant increases in funding for the National Institutes<\/a> of health. I think it has made a difference. Senator, i really appreciate you taking the time to explain all that because i think there are people watching, wondering how these decisions get made and where we are, and certainly there have been a lot of anxieties in the Biomedical Research<\/a> about whats going on and whats the likelihood of a pathway. You pointed out theres a great deal of reasons to be excited and optimistic about a career in Biomedical Research<\/a> first while because the science is so incredibly promising and exciting, but second of all because the support has been on the upward trajectory. We have two years already with this 2 billion increase happening and theres significant promise but that may happen for the third. That would be about a 20 increase for nih over the course of three years which is a very encouraging sign. Again, i am grateful that in this era was so few things seem to be bipartisan, medical Research Continues<\/a> to be so and much credit to you and your colleagues for keeping them alive. Our last one last question. With all of this excitement in these breathtaking medical biomedical advances, do you see any, as you talk to high school or College Students<\/a> to see any interest in biology . I dont talk to high school and College Students<\/a> that much. My girls are eight and four. I think there is an increasing excitement among those training in medicine right now about what the future holds but i will affirm that. I have a lot of contact with residents and medical students and i see an increased excitement about what they will be able to accomplish over the course of their careers that i couldnt accomplish over the course of my medical career. Certainly when i got to university to give a talk and meet with students and i always do that, thats the best part of any visit to an Academic Center<\/a> to say i want a chance to meet with the graduate students and i dont want anybody else because i want to hear from them. I have heard a lot of anxiety in the past couple of years and im trying to reassure people that things are looking much brighter than they think they are. The sense of their trouble is probably beyond what it may be considering the path that we are on, but boy do i hear excitement about the scientific opportunities. If you can quickly switch to that, then it goes into the conversation in the eyes light up in 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 that a single student can do right now that might have taken a team of ten people five years to do but technology has come along so quickly that the ability to use big data and computational approaches, this is the golden era. If anybody wants to really have a great time in science they should come join us. I hope ian will be one of those as soon as possible because we need the spread this is our future. We want the best and brightest to join us so this can happen. Thank you. The record will remain open for ten days. You can submit Additional Information<\/a> within that time if you would like. The committee will meet again next week tuesday december 12 at 10 00 a. M. For a hearing titled the cost of Prescription Drug<\/a>s, and examination of the national cavities of science, 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 and 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 stands adjourned. [inaudible conversations] tonight on cspan2, book tv in prime time with a look at some recent bestsellers starting at 8 00 p. M. Eastern, historian David Mccullough<\/a> on his book the american spirit. Former secretary of state Condoleezza Rice<\/a> whose new book is titled democracy. The National Book<\/a> Award Winning<\/a> offer examines race, the Obama Presidency<\/a> and the 2016 election in his book we were eight years in power. Also, conservative radio host rediscovering americanism, and we close with feminist writer roxanne on her memoir hunger. Book tv in prime time all week on cspan2. Ive been attacked by everybody. Ive been attacked by the rightwing, by the russians, by the trump campaign, by the Sanders Campaign<\/a> and now i can add to that list the clinton campaign. Sunday on cspan q and a former Democratic National<\/a> Committee Chair<\/a> Donna Brazile<\/a> talks about her life and politics and her memoir. I was here at tenth and g street, not far from here. Hillary was very excited. She had met this young state senator who was running. She has roots in illinois. She met this young state senator and she told my good friend, we were on the third floor and she said youve, she knew barack obama. I didnt know barack obama. I knew a lot of other people in chicago politics but i hadnt heard of him. We met him that spring of 2003, let me just say this, the rest is history. Q a sunday night at eight eastern on cspan. The second session of the 11h Congress Starts<\/a> next week. The senate is back on january 3 and will welcome two new democratic lawmakers. Alabama doug jones and tina smith. The house of representatives returned a few days later on january 8. In the new year, Congress Needs<\/a> to consider a Government Spending<\/a> bill because current government funding runs out on january 19. Also on the calendar, this years state of the union address, House Speaker<\/a> paul ryan has invited President Trump<\/a> to address a joint session of congress on january 30. When congress is back in session, the senate is live on cspan2. The house is live on our companion network cspan","publisher":{"@type":"Organization","name":"archive.org","logo":{"@type":"ImageObject","width":"800","height":"600","url":"\/\/ia800403.us.archive.org\/11\/items\/CSPAN2_20171228_144300_Medical_Product_Development_and_Innovation\/CSPAN2_20171228_144300_Medical_Product_Development_and_Innovation.thumbs\/CSPAN2_20171228_144300_Medical_Product_Development_and_Innovation_000001.jpg"}},"autauthor":{"@type":"Organization"},"author":{"sameAs":"archive.org","name":"archive.org"}}],"coverageEndTime":"20240630T12:35:10+00:00"}