Transcripts For CSPAN3 Medical Product Development Innovations 20171206

Chfts approved by congress a year ago this month. This runs about two and a half hours. Order. Chair recognize himself. A monumental achievement. Cures was the product of a by partisan multiyear effort by the energy and Commerce Committee. To brought the laws into a modern era of medicine. It has been one year since cures was signed into law. I remember remarking at that press conference a year ago to imagine a world in which government was not an obstacle but an ally in helping us deliver drugs and devices to patients cures to patients. Todays hearing marks the Health Subcommittee first look into the implementation of the what many in the Healthcare Community called it transformation bill. That would positively impact not only the researchers and the scientists who are developing the latest breakthrough therapy. But physicians seeking treatment for their patients. Giving home to them, their loved ones and advocates. This morning we will hear from two leader responsible for implementing the Drug Development and medical research provision. Included in cures. I want to welcome francis collins. Director of the National Institute of health. And Scott Gottlieb the commissioner of the food and drug administration. All of us know the demands your schedule put on both of you and we appreciate you coming before us. Today. At the time of the energy and Commerce Committee launch of the 21st serge Century Cures Initiative the statement was made that there were 500 cures and treatments to address. 10,000 known diseases. More progress was needed to alleviate the agony of an incurable disease. While the United States has maintained its Global Leadership in innovation. There existed a bridge in the growing divide between the revolutionary advances in science and technology and a less than adequate system for discovering, developing and delivering new therapy. Members of the committee both this and the Senate Health Committee Held numerous public hearings and round tables. And around the country. Bringing together leading scientists and medical experts. Patient and Disease Group advocates and researchers across multiple sectors. The primary objective of these events was to uncover opportunities to strengthen and steam line the process by which cures are discovered and made available to patients. Cures accelerated the cycle of Discovery Development and delivery of new treatments and ensured the United States remained at the helm of medical innovation. At the National Institute of health the cures act authorized resources to support medical research. And reduce add mrtive burdens and provide almost 5 billion in new funding. To support the agencies for Innovation Projects. The Precision Medicine initiative was thordsed for 1. 4 billion for the National Institute of health to be able to National Data set in order to accelerate Health Research and medical breakthrough. The bill also authorized 1. 5 billion for the Brain Research through advancing innovative Neurotechnology Initiative to better understand the brains fizzology. And coordinate efforts across multiple groups to expedite research for decides like alzheimers. Cures also authorized 1 prnt 8 billion for cancer prevention. Diagnosis, treatment and care. Through the moon shot. Finally the Regenerative Medicine Innovation Project was authorized at 30 million. To support Clinical Research in the field of Regenerative Medicine. And coordination with the food and drug administration. The cures act helped the fda modernize the regulation of medical products throughout its life cycle. Established the innovation act and authorized 500 million in funding to implement title three of the law. Including a broad range of deliverables from the fda. These include creating a mechanism for the collection and incorporation of Patient Perspectives in regulatory decision making. Updating the way medical products are reviewed and approved. Advancing new drug therapy through a review path way for bio markers. Other Drug Development tools to shorten time to maintain the same rigorous standard for safety sdp effectiveness. Also required the fda to establish standards and definitions necessary to develop Regenerative Medicine. Before i close, i recognize the cures act also touched upon other Critical Healthcare priorities such as Mental Health and Health Information technology. Both of these areas should have their over separate hearings. Because of their importance to the medical community and those are on the list for the very near future. I want to welcome our witnesses and thank you for being here. I look forward to your testimony. My time is expired. I will yield to the gentleman from texas. Mr. Green. Five minutes for opening statement. Thank you. I want to thank former chairman and congresswoman for being the original cosponsor of the cures. The one Year Anniversary of the cures act. Being signed into law by president obama. And his last public signing. It was a great achievement. Plrly at a time of gridlock. With the work started long before 2016. In 2014 we set out on a mission to do something positive to boost medical research and accelerate the discovery, development and delivery of new cure and treatment. After countless hours the vote at the round tables and hearings. Cures enjoyed by partisan support and endorsements from over 700 ogss. Representing the full spectrum of stake holders. It dedicated 6. 3 billion in new investment to support priorities. The Brain Initiative the medicine initiative. Within the National Institute of health to combat Prescription Drug abuse. It also provides money to the fda to advance the agency mission. Implement the policy. The influx of investment is solving todays complex science problems geing new treatment to the bedside and improving public health. Specifically the nih provided 4. 8 billion in funding to advance cunning Edge Research initiative. Prosided 500 million over ten years to improve the agency medical product review process. Without compromising the safety and effectiveness standards. In addition to this much needed funding there were so many provisions worthy of support from development of new antibiotics the fight against superbugs and yous of modern Clinical Trial designs to foster the next generation of medical research. While some of the technical in nature, the real world impact they could have is not abstract. Patient, families deserve to have elected officials respond to need and this bill was an attempt to do that. Like all negotiations in compromise we didnt get everything we want. Theres always more than can be done. But today is an opportunity hear from the heads of fda on implementation of things like patient focus Drug Development. Improving science and hiring capacity. It has been a year since passage. These things take time. I know folks in the agencies have been hard at work to get new initiatives off the ground and build on past efforts to advance medical research and development. Not the focus of today hearings cures included 1 billion to combat the Prescription Drug abuse and over dose epidemic. The funding was significant but pails in comparison to what needed. There are more americans dying from this than the height of the aids epidemic. I hope the committee and congress will fulfill the responsibility to the people and provide real and needed funding to fight the epidemic. The cures demonstrates what we can accomplish when we work across the aisle. And hope we can do so again. I look forward to hearing from our witnesses about the ongoing implementation of the cures act. And chairman i want to yield the rest of the time to congresswoman. Thank you. Thank you for all of your work that you did on cures. Ill be sorry not to have you as my seat partner. In the next congress. You have done a wonderful job. Many chairman i want to thank you too for all of the work you did on cures. Fred up ton of course is my partner and he was the chairman at the time. We really want to do something bold and big. When we started conceiving of cures. And i think we achieved that. So im looking forward to hearing from our two witnesses today. Doctor Scott Gottlieb your agency was a key partner. And doctor collins was there. Helping us craft the bill. At one point i remember dr. Collins said to me, we just need to let our young researchers go to conferences. And i said if thats all we do, we will have failed. We did that and we did so much more. And so were eager to hear how this bill has had impact in just one yore. And but were even more eager to hear where we can take it next. Thanks for all you do. Thanks to the committee for working together on this bill. I yield back. Chair recognizes the gentleman from oregon. Thanks for having this over sight hearing. Of an important life saving law. It was some of the finest work this committee has done. I know if you havent had anything else on your plate in the last year. I say that facetiously. Youre doing a marvelous job implementing this vast bill and helping move forward to save lives and improve the lives of families, friends and people well never know. And the consequences of the legislation are not confined to this hearing room. Or the United States. The research and the progress that will be made in the sectors will effect everyone in the world. This is world changing. My colleagues on both sides of the aisle have done marvelous work getting this done. My view is always been that once you pass a law thats just starting place. I know how difficult it was to do this. That was starting point. Today we look and say what is it going forward. Hows this working . Are these tools effective. Are there changes that need to be made. We know youre making great progress and we appreciate the terrific work. I also want to recognize a very special guest here with us today. We have many in the room. I want to draw special attention to somebody part of the journey from the beginning. Thats young mr. Max. Were delighted to have you. Youre an extraordinary young man. And we are so very glad that youre here to share in this special birthday appearance of the cures legislation. And its because of people like you that inspire us to do the best that this committee has to offer. The best work and best legislation. We know human lives are at stake. With that ill put [ applause ] ill submit my opening statement. In to the record. And defer the balance of my time to the former chairman of the committee. The chairman on energy now. And i know there are other members who would like to share. With that i yield back. To the champion of the cures and the improvement of peoples lives around the world. My friend from michigan. Thank you. You know when we began the process of crafting the cures four years ago. We began with one goal in mind. That was helping patients and their families. My great partner on the other side of the aisle and i were inspired to act after hearing from folks in the Research Community. Wels patients in their families. About the need for modernization and more resources. To move quickly bring life saving treatments to market. In all of us had inspiration in the district for me it was two sisters the kennedy sisters. Who have a rare disease called spinal muscular atrophy. Cures provided the fda with billions of dollars in much needed resources so the nations best and brightest could work and finding cures for diseases that impact virtually every sick single family. Whether cancer. Diabetes or Rare Diseases. This hearing is a great thing for lots of reasons. A remirnd of how republicans and democrats came together to get a monumental piece of legislation signed into law despite our divided time. She worked with me on this as we worked for years. And listened and worked to craft the language that would become law. The hearing is reminder we have a lot of work to do. The kennedy girls and max in the front row along with millions of patients and families are counting on us. For that reason i im glad to welcome both dr. Collins and dr. Scott gottlieb on how the law is being implemented and what we can do to help the process along and improve it. I yield to the general lady from tennessee. We do welcome our witnesses. And we take this as an to thank you each. For the help and the guidance that you provided. In what were then your roles and what are now your roles. As we implement 21st century cures. It is so appropriate that we do this hearing. Because as youve heard there was so much more that went into this. Than just saying lets have people go to conferences or lets try. This was a way to change and reform the review and approval process. So that it more adequately meets the innovation that is taking place. And Healthcare Delivery systems. So we welcome you. We welcome max and his by partisan friends. Who have joined him this morning. What a great reminder max they have a reserved seat. Right there on the front row in sharing the success of the day. Mr. Chairman i thank you for the hearing i yield back. The gentleman from new jersey. Five minutes please. Thank you. I want to welcome dr. Collins and dr. Got leeb. To discuss the implementation. While the law addressed several different issues such as the Opioid Epidemic and Mental Health. Were focusing on the ongoing work to implement the provisions of the law. Aimed at improving the discovery and development of new cures. Im proud that the law included funding for the biden cancer moon shot. This Initiative Aims to accelerate Cancer Research and youve the ability to prevent and detect cancer early on. In the hope that one day we might find cures for the different cancer such as pancreatic. Im interested in hearing how theyre working to achieve the goal. New fund in the Brain Initiative and Precision Medicine initiative. The Research Program the funds important rerge r research on Brain Disorders such as alzheimers. Epilepsy and traumatic brain injury. To gather data from one Million People that will lead to the development of Personalized Therapy rather than one size fits all treatment. The cures act aim ts to bolster the medical product review process in order fo get treatment to patients faster while also maintaining fda goal standard for safety and effectiveness. For example the law granted aided thor authority to development and utilize new tools to facility Drug Development. Provide flexibility in the Clinical Trial process and support the development of Continuous Manufacturing. The use of Patient Experience data in the review process and provided with 5 up million in new funding to insure the agency has the necessary resources to recruit the best and brightest scientists. I look forward to hearing about the Progress Agency has made to date. And lastly the cures act marked an important step towards the development of new treatment and cures. Able to Work Together on a basis last congress to pass the monoyoumtal law. I want to thank the chief sponsors. Its critical that with hold hearings like this to insure the law is working as it should and achieving its goals. I look toward to hearing from the witnesses today. Id like to yield the rest of my time to representative. I thank the chairman and Ranking Member for this hearing today. I tharng the witnesses for their attendance. We worked together to pass the addiction and recovery act. Its fair to say it was a compromise. During the debate i pushed and have continued to advocate for more funding and resources it address the deadliest drug crisis in american history. We came together and advanced legislation to o provide 1 billion over two years to strengthen the response to the crisis. Still cures act two year funding window creates planning problems for state and local government. The uncertainty in funding to hire staff or plan beyond two years makes it difficult for people on the ground to do the work were trying to empower them to do. We must do more. Thats why i introduced legislation to extend funding to combat the Opioid Epidemic for five years. I ask members to add their voices to the effort. Lets Work Together to find Common Ground and move this. Because this drug crisis is tearing apart the fabric of Community Across the country. We must Work Together to ensure that this important funding doesnt expire. Too many people are suffering without access to meaningful support systems. We must step up our prevention eft. One long time avenue is the development of safe and effective nonaddictive opioids. We need to move Forward Research and treatments that stop the craving of opioids and alcohol. Ty communicated with your office on this matter and i understand the fda is working on a series of meetings to Facility Development of nonaddictive pain treatments. I sent a letter on this issue. You responded by answering a few of the questions. Not all of them. Ill be sending letter again with expectation of more thorough answers. And responses to all of the questions and ill be submitting them into the record. Thank you for holding the hearing. I yield back. This actually concludes member Opening Statements. And the chair would remind members pursuant to rules all members Opening Statements will be made part of the record. I want to thank and welcome the witnesses for being here today. And taking time to testify before the subcommittee. Each witness will have the opportunity to give an opening statement. Well follow that with questions from the members. Well hear from dr. Scott gottlieb. The commissioner of the fda and francis collins. The director of the National Institute of the health. We appreciate you being here today. Your recognized five minutes please. Thank you. Thank you for the opportunity to testify today. On the anniversary of the cures act. And to update you on the progress in the provisions of the landmark legislation. The cures act gave broad new set of authority and resources to adapt the policy and organization truckture to make sure the efforts are modern as the medical products were seeing. Im proud that my colleagues have worked hard to meet the commitment under this statute. I want to commit that timely implementation of the legislation is one of my highest priorities. The cures act is the defining element of my own policy planning. When i arrived seventh months ago i remarked i couldnt imagine a better time to lead lt agency. The opportunity offered by new science and opportunity. Gene and cell therapy. Targeted drugs. Regenerative medicine. Digital health tools and materials. Offer the potential for better and curative therapy for many disorders. The Second Opportunity provided by congress. The reauthorizization of the cures act. Offers new platform to fashion the scientific advances and practical traemt for patients. If i came before congress five years ago and said that within the next five years we might have a cure for sicklecell or cancer. Such predictions would have been unrealistic. In fact we should expect these opportunities. While these scientific advances wont be risk free. These and equally opportunities are before us. The cures act inspired a new approach to the work. A direction from congress that you want us to think differently. When it came to breakthroughs to transform human health. At the same time you asked us to look for ways to make the approach more scientifically modern and efficient. To meet the urgent needs of patients. We have taken spirit of cures and set out to extend across our policy making and planning. To build on what you asked us to do. Well release a document to make sure were continuing to expand what congress set out to achieve. I want to share with you one such effort. With the targeted medicine were sometimes able to observe earlier and some cases benefits. This is especially true when it comes to oncology. These situations are compelling us to facility and expedite the development and review out products. For example were currently examining approaches to better expedite review and approval by leveraging the existing programs. That showing a benefit is based on the drugs effect on a surrogate end point. In these cases that end point like tumor shrinkage is drugged to be reasonably likely to predict clinical benefit. A targeted drug introduced. Where the benefit is seen in the small trial where we need more evidence to fully understand how to best use the drug in clinical practice. We might want to approve a product earlier and require post market confirm study to validate the finding similar to an accelerated approval approach. Even though the observed benefit is on a clinical end point. And not on a surrogate measure. We believe using an accelerated approach could be valuable. Congress clarified our authority under to grant accelerated approval based on clinical end points. We want to better define whats meant by end point to ensure Product Developers can full advantage of the provision. As a mechanism of diseases like cancer become clearly defined. And drugs tailor to the under line biology of disease. Well see more such case. Situations where a new drug offers a survival benefit. One reason we want to consider accelerated approval is it would include authority to require confirm tear evidence. If that evidence fails to confirm the benefit. To fully leverage the opportunities and keep with the cures working on a similar proposal. The cancer drugs already approved for one indication. We intend to issue guidance further clarifying the circumstances in which this is appropriate. This maybe suitable when theres a treatment effect. For example a targeted drug approved for a third line use. Cures refashion and modernize updates enabling new technology to reach patients more efficiently. Continuing to build on the frame work. I look forward to discussing the plans and answering questions. Chair recognizes the director of the dr. Collins. Good morning. Its an honor to be here. With my colleague. Dr. Scott gottlieb. We were cheering a year ago today. When the cures act passed the house of representatives. 392 to 26. And as you well know this act aimed to cat liz a an important goal shared by americans to speed the pace at which scientific discoveries are translated into life saving treatments and cures. We greatly appreciate your leadership. In passing this by partisan act one year ago. That enhances the authorities and resources and ways that will help us achieve this goal. Many thoughtful provisions are inclouded in the act. Such as reducing administrative burdens so the scientists can devote time to research. Expand the ability toward prizes for exceptionally creative inside and strengthen measures to protect patient privacy. When individuals are involved in research. I submitted a report on how we quicked quickly to implement the provisions of the act. In my oral statement just now id like to focus on the Cures Innovation Fund. Among the vital areas being accelerated by the fund, are the brain initiatetive. The cancer moon shot. The Regenerative Medicine Innovation Project. And the Precision Medicine initiative. Im delighted to have max here rep represent the most important audience. Id like to recognize my friend doug oliver. An effective spokesperson for the importance of investing in medicine. The Brain Initiative. This effort is aimed at revolutionizing our understanding of the most complex structure in the known universe. The human brain. In 2017 we leveraged our funding with the annual appropriation to launch no less than 110 exciting new Brain Research products. Some will develop detailed maps of neurocircuits. A census of cell types in the brain. And others will create powerful new tools to monitor and brain activity. This will advance efforts to develop new ways of detecting treating and preventing many serious Brain Disorders such as alzheimers. Schizophrenia. Autism. Drug addiction. Epilepsy and traumatic brain injury. With the help of the cures fund a Second Research area. The cancer moon shot. Is aggressively pursuing an ambitious goal. To accelerate diagnosis, treatment and care. In collaboration with the colleagues. To achieve that goal, we must take a variety of innovative steps. These include enhancing the Research Infrastructure by creating a Clinical Trial net work. With an unwaivering trials netw an unwavering commitment to data sharing, to move Cancer Treatment programs forward rapidly. In another innovative move nih recently joined with the fda and 12 pharmaceutical companies to launch the partnership for accelerating cancer therapies, or pact. This Publicprivate Partnership will initially develop bio markers to speed the development of cancer immuno therapies and an exciting approach to treatment that enlists a patients own immune system. Recently we have seen some amazing responses from immuno therapy. But we need to bring that kind of stus to far more people with more types of cancer and do it quickly. The Cures Innovation Fund with the support of this congress is helping to make that happen. The cures act also provides support for Regenerative Medicine research. This emerging area of science includes the use of cells and other technologies such as engineered biomaterials and gene editing to repair or replace damaged cells, tissues, or even whole organs. As a result of the cures act n. I. H. Has launched the Regenerative Medicine Innovation Project. This project recently made eight Clinical Research awards covering a Broad Spectrum of science and technology and going well beyond the funding specifically provided by the cures act because we found it to be so compelling. Some are focused on common disease including diabetes and vision disorders while others are aimed at rarer conditions such as sickle cell disease, which scott has already mentioned as a very exciting time of potentially moving forward a cure in as little as five years, and a condition like Idiopathic Pulmonary Fibrosis and many others. Also in partnership with the fda were going to be hosting a workshop next week which is going to explore the state of Regenerative Medicine research involving adult stem cells. This conference will inform our future Research Directions by helping us to identify areas of greatest scientific and therapeutic promise. Finally i want to tell you how thrilled i am that you supported the Precision Medicine initiative, pmi, by including an authorization and funding in the cures act, the centerpiece of pmi is the all of us Research Program, which will enroll 1 million or more americans from every walk of life. These volunteers will contribute their health data in many ways over many years to create a Research Resource that will catalyze a new area of Precision Medicine. This is truly an ambitious goal and we know nih cant succeed on its own. So all across the nation nih is teaming up with the veterans administration, Health Provider organizations, Community Health centers and other groups, recently libraries across the country, to figure out the best ways to recruit participants, especially those that are traditionally underrepresented in Biomedical Research. Nih also partnered with five companies to create a Participant Technology Center and our partners are testing how wearable devices like the ones im wearing today and many of you are probably wearing something like, this how can we use these to provide ways to contribute data on physical activity, sleep, heart rates, environmental exposures and so on . Getting all these partners on board would have been nearly impossible had not the cures act included something called other Transaction Authority for pmi. Making it possible for nih to move forward with unprecedented speed and flexibility to carry out beta testing among the many components and now a planned launch in the spring of 2018. As someone who grew up in a theater family i know the value of a dress rehearsal before the curtain goes up. Thats what a beta test is. But when it does go up you and everyone else who supported the 21st century cures act will deserve applause. Not just for all of us but for each of the many, many ways in which cures supports the work of the National Institutes of health. Or as some have called us, the National Institutes of hope. Specking of hope, let me conclude with a favorite exhortation from the 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 cures. Thank you. Ill be happy to answer your questions. Thanks to both of our witnesses for testimonies. We will move into the member question portion of the hearing. I also want to recognize the chairman of the full committee, mr. Walter morgan, for five minutes. I thank the chairman. And again, thank you both. Not only for your good work but also your terrific testimony here today. I also want to thank dr. Gottlieb for his excellent efforts to make sure that our war fighters have access to cutting edge medical devices and medicines that are both effective for them and safe for them. We appreciate the work you did with us and our friends at the Armed Services committee and the pentagon to get that done. Dr. Gottlieb, the fda Oncology Center of excellence was created in cures as a model of how collaboration in science among and within Government Agencies should be done in the future. This is a new model, and i know we were hoping here in congress that this would succeed. And in your testimony you referenced the role of o. C. And the approval of two cell based gene therapies that are indicated for treatment of cancer patients. Can you speak a little more about how groundbreaking these two treatments are and the role oce played in their approval . Thank you, mr. Chairman. I just want to make one brief comment about the legislation that this committee helped craft and crafted with respect to the war fighter. I think its going to give us a profound opportunity to expedite the approval of products destined for the battlefield setting and to help protect and promote the health of war fighters in the battlefield setting. We look forward to early implementation of that and robust implementation of that. Well try to make an effort early on to put out specifications on how we plan to make full use of that. I think its going to provide profound opportunity for our war fighters, and i thank the committee. With respect to the Oncology Center for excellence the products you refer to with gene therapy products that we think are going to represent sort of a transformative opportunity as a class of products for the treatment of patients with a range of conditions including cancer, these were carty products in which cells are genetically altered to attack cancer and personalized to the patients individual cancer. With reblth to the Oncology Center for excellence it was instrumental in the review of these products. We believe the orientation in the future across the entire agency is to try to consolidate the clinical portion of the review among the agencys various medical products centers. We divide medical products into different centers. But the clinical aspects of the review are the same, even if the product features are different. So trying to consolidate that clinical portion of the review provides a lot of efficiency, rigor and also helps quicken the process. Standing up this new Oncology Center we think is crucial to these future classes of products. Thank you, sir. Dr. Collins, over the last several years nih has been acting to address a Biomedical Research workforce thats tilted toward frankly late career investigators. Population of grant recipients is also highly concentrated with 10 of nihfunded investigators receiving over 40 of nih funding. Analyses conducted by your agency and others have shown that a more diverse population of nih grant recipients would be beneficial to Biomedical Research. Cures require the nih to develop strategies to promote and facilitate the next wave of nung researchers, and in your testimony you talk about the next Generation Researchers Initiative. Can you further elaborate on the multipronged approach you plan to take into crease the number of nihfunded early stage and midcareer investigators . Thank you for the question. This is an area of great and high priority for us and we appreciate it very much, the way in which the cures bill called this out and gave us additional encouragement to think boldly about how we can ensure this next generation of researchers are getting their start as independent investigators with all the energy and creativity they bring to it. We could look at our own demographics and see that we were increasingly seeing an aging of our workforce, and while we have many investigators who were highly productive as senior investigators we were worried the next generation was having a tough time coming on board. So over the course of this past year since the cures act passed and guided by many conversations before that, we have come up with an approach which is going to provide Additional Resources for those who come to us for the first time with a grant that has not previously been funded by nihp but this is their start. And to provide additional opportunities for those individuals if they fall in the top 25 of applicants to be able to receive funding. We made this decision fairly late in fiscal year 17 but we were determined to go ahead and implement it. We are still 2349 process of identifying all of those investigators who were reviewed in fiscal year 17 that otherwise missed the can the but otherwise we believe we can reach down to and find funds for. Were very concerned about those who are at risk of losing all of their funding. They got started in the competing path. We are also seeking to identify those individuals and give them an additional boost. That money has to come from somewhere, and that means we may not be able to be quite as generous in other areas of research including some labs that are extremely well funded. And as you can imagine, not everybodys been excited about that part. But we do believe its the right thing to do. This is the future. If our mission is to try to find every place that we can to use the dollars that the congress provides us to get the maximum benefit, those young investigators just Getting Started are a critical part of that. Thank you. Thank you both for the good work youre doing and being here today before committee. With that, mr. Chairman, i yield back. The gentleman yields back back. Chair recognizes the gentleman from texas mr. Green for five minutes of questions, please. Thank you, mr. Chairman. Over the last decade theres been a growing recognition in the u. S. And abroad that antibiotic resistance poses a serious and growing problem to our health. Without the important medical advances such as chemotherapy and surgeries become very risky because of the possibility of infection. Addressing this threat requires a multiprong approach including reducing the inappropriate use of num antibiotics in agricultu agriculture. We know there are a number of challenges in discovering and developing new antibiotics. Dr. Collins, my first question, there are basic scientific barriers which impede new antibiotic discovery and development. Can you tell us what the nih is currently doing and will be doing to address these barriers . We have a very Important Role to play so thank you for the question. Yes, there have been challenges in terms of keeping this pipeline of discovery and Development Going for antibiotics in part because some sort of looked at this as a bit of a market failure because of the expectation that new antibiotics would have potentially a very limited market for a while, youd want to save them for those circumstances where you really needed them. So nih has an even larger role to play in this space in terms of the discovery part and in moving the new discoveries along the. Imline, closer to commercialization to derisk those projects so antibiotics will be seen by the commercial sector as something theyre ready to pick up and go. And we 59 nih particularly through the National Institute of allergy and Infectious Disease led by tony fauci have a very significant amount of funding invested in this. One is using new technologies to discover natural antibiotics created by soil organisms that we didnt know there because we cultured them in a lab. Theres a whole new generation of ideas coming from there. This is not a solved problem. Its going to take the full effort of the public and private sector to ensure we are using the maximum energy in this space because we have a ticking clock here for a significant number of individuals being found with infections for which none of our antibiotics currently would be ready to work. We will not be able to succeed in the goal of developing antibiotics without a strong bench of scientists. What is nih doing to ensure that these young scientists are pursuing careers in the antibiotic discovery and development . This ties into the answer i gave a moment ago to chairman walden about the things we are doing to try tone courage our firsttime investigators to come on board and be able to get successfully funded. In fact, many of those investigators are in this area of Infectious Disease. As we are lifting all the boats for that category of investigators were also helping in this space. But the National Institute of allergy and irvin effect shous disease also because this is a high priority, issues special funding announcements, including recruit investigators that recognize theres people out there that might work on Something Else but knowing theres a funding opportunity would raise their hands and say lets work on this. Dr. Gottlieb, this committee has taken the threat of antibiotic resistance very seriously. In 2012 Congress Passed the generating antibiotics gain act, which our former colleague phil gingrey, whos here today, came out of this committee and gave exclusivity to treat serious and lifethreatening infections. Just last year in cures we passed the adapt, which created a new regulatory pathway for antibiotics that treat serious and lifethreatening infections and meet an unmet need. I thank congressman ship kuss for picking up that cosponsor. Can you give us the status of implementation of adapt . It continues to move forward. I will comment on a couple of things if i may, congressman. To pick up on an earlier comment. Weve taken steps to reduce antibiotic use in veterinary animals. Were going to look at continued steps we can take to address some of the prevention claims in those labels and build on the good work that was begun by my predecessor dr. Hamburg. Another important provision, you mentioned the gain act. Another important provision is obviously the lpad designation created by the cures act 37 were going to put out guidance on that this summer. Weve had a large number of limited number but a robust number given the early days of that provision. Preind meetings with sponsors looking to take advantage of that provision as a way to accelerate the approval of products targeted to resistant organisms. I want to thank the committee for the collective good work youve done through all of this legislation. This has been immensely important to the agency in giving us a new set of tools to address these issues. Thank you. I dont have any more time left. But thank you for that effort. And thank both of you for being here and the work youre doing. Obviously as a committee we want to continue to partner with you. Thank you. The gentlemans time has expired. Ill recognize myself for questions. Thank you for for being here. When im home a lot of times doing town halls or whatever, a lot of times most of the things i talk about is whats happening from this committee, subcommittee, in the Health Care World and the research. Its fascinating stuff thats going on. As dr. Gottlieb said, were talking about hopefully being able to be on the cusp of curing diseases i never thought about. Around this time the round table where Roger Daltrey was here, he was talking about teenage cancer. We also had a young man talking about Cystic Fibrosis. And i have a friend would lost his son in his mid 20s to Cystic Fibrosis. I was sitting there thinking hes my daughters age and a few years ago hed probably have a few years left to live. Depending on a lot of circumstances. But they talked about he may live a full life expectancy. That is really whats happening with the research nih, whats happening in the private sector. And so for me what made 21st century cures exciting and all of us have these experiences, i have a constituent, a father, that has deshins muscular dystrophy who wa wo come to the office and say theres in promising trial, my sons not in the trial, it doesnt improve you but it prevents flu regressing, were racing against time for his son not to get into a wheel chair because his goal is his son not on ney wheel chafr. Another one his son was on trial for the afshlg pancreas and then the trial was over. Of course it was in a lab setting so they couldnt it home. They said my son has never felt this good since he was diagnosed and now we have to give it back. They have it now because hes approved. I is had a k450i8d with a little childhood issues. And parents immediately become experts in that childhood disease. It drives your life. I can tell you that. We hear from a lot of people. And what we want to be able to say is the research, money, we want to make sure the fda is doing everything to get these because if youre a parent and youre not in the Clinical Trial but youre hearing this is well, this is for a small bisis but im not sure i can extrapolate along the whole population. You want it for your child if you can have it. But understand the safety and efficiency that you guys have. What we wanted to do, my first of 21st century was how do we give you the tools and in your reach and in your approval process to make sure people in those situations are confident that its coming as fast as possible, if we have the accelerated approval process and those things moving forward and so on, excited for this overview. It is something that drives all of us in washington because we all have experiences personally or with our constituents. One area i focussed on was the continuous background, thats kind of my background so i appreciate you being here dr. Gottlieb and understand the development of Continuous Manufacturing systems could be some of the most Significant Development in pharmaceutical industry in the next decade and im happy to hear the fdas taken steps to still tate progress in this arena so our country can recognize the benefits of this faster and a more reliable way to manufacture pharmaceuticals. Can you speak to the next steps and lu be providing additional grants . How do you envision in Technology Improving in the field . I thank you for the question. Congressman. This committee provided us a good head start. Providing grants for the development of tools that will help this Technology Continue to advance. We allocated one such grant of i believe a Million Dollars. We have 4 million left to allocate. Were going to do that. To look at other programs, mostly in academic institutions that can help facilitate the development of the regulatory tools that well use to better evaluate and allow this technology to advance. This is very important. You mentioned to allowing more efficient, maybe lowercost development or manufacturing. It also is very important to trying to address drug shortages because of the nature of Continuous Manufacturing. You dont have as much risk of discontinuities in the traditional manufacturing process as you would through traditional manufacturing. The final point is by using Continuous Manufacturing you require a much smaller, less expensive footprint. So i think that the Rapid Deployment or the further deployment of this technologys going to lend itself to potentially reparticipate rie yailting some of the manufacturing weve seen go offshore, come back to the United States. The final thought is this technology is going to be important to some of the new complex products we see in Development Like gene therapy. We think of Continuous Manufacturing. Its also being adopted with respect to biologics. Race against time. So speed is important but the regulatory side important too as i understand that as well. My time has expired. Id like to recognize the Ranking Member of the full committee, mr. Polone, for five minutes of questions. Thank you, mr. Chairman. You already asked one of my questions. So i have to cut that out. But let me start with dr. Collins and again ill go to dr. Got lieb about Continuous Manufacturing if i could. Dr. Collins, during the 21st century cures kabt debate we had a lot of discussion. While im glad were able to Work Together to advance policies that support the development of the next generation of researchers i am concerned about reports on how the tax bill could thwart such efforts. As you know, a fundamental element in pursuing careers in Biomedical Research is obtaining a graduate degree. Unfortunately the house tax bill could put such education out of retch for students. Akoshding to my own russ gerz University President dr. Robert barchi, the provision of the house tax bill that would tax as income tuition that schools waive for graduate studentsing it working as teaching or Research Assistants would impose, and this is a quote from the rutgers president , would impose an especially heavy burden on our graduate students, many in stem fields. Other College Leaders have said the change will make graduate education unaffordable, lead to fewer graduate students at a time when the u. S. Needs more students earning advanced degrees in the stem fields to remain competitive. I just want to ask you are you worried that making tuition waivers taxable for students would harm our efforts to create the next generation of scientists and how you might such a result harm our ability to advance the discovery of new treatments and cures which of course was the galvanizing force behind 21st century cures . If you could. Congressman, thank you for the question. And it ties in with what i was saying a few minutes ago responding to chairman walden about the next Generation Researchers Initiative which we are putting a lot of time and effort into trying to make sure it becomes a high priority. Certainly graduate students as the path towards those independent investigators of the future are absolutely krital and we want to have all the best and brightest who are interested in pursuing those careers to have the opportunity to do so and anything that represents a major impediment in that regard is something we should take with great seriousness. I am not an expert in tax reform or in the particular provisions of any of the bills under consideration, but certainly i think we can all agree that given that science has driven our economy and this country by most estimates more than 50 of our growth since world war ii has been on the basis of science and technology, this is a very important area for continued investment and anything that would dim nirk the interest in the talent of the next generation in joining that workforce is something we should be very cautious and careful about. Let me go back to dr. Gottlieb. Mr. Guthrie talked about the Continuous Manufacturing issue and you mentioned dr. Gottlieb you awarded the first Continuous Manufacturing grant in this fiscal year i guess to the university of connecticut to build the manufacturingplan platform for complex dosage forms. What i wanted to ask, though, is will you discuss further how many additional grant awards the agency intends to offer and what criteria the agency is considering when awarding the Continuous Manufacturing. Thank you for the question. I mentioned we had 5 million to allocate. Weve allocated a Million Dollars of it and were going to continue to allocate the other four. Im not quite certain how many different grants well give but there will be a number of grants awarded and theres a number of academic institutions doing good work in this area including one in my hometown of Rutgers University that has a program of looking at this. A criteria we look at are programs that are developing regulatory tools that serve the basis for how were going to evaluate this technology when sponsors bring in applications when theyre employees of Continuous Manufacturing. Because its so novel it requires us to think differently about how we apply the oversight to the manufacturing process and thats going to require us to develop new methodologiey s. O. P but new tools to evaluate the safety and reliability of the manufacturing process. Were looking for institutions that help develop those tools. As i mentioned theres a number of them including one in my hometown but uconn also was doing this. All right. Thank you so much. Thank you, mr. Chairman. The chair recognizes the gentleman from michigan, mr. Upton, the primary sponsor of the cures bill. Five minutes for your questions, please. Thank you, mr. Chairman. I appreciate all the kind words here but i want to remind everyone on this committee as we passed it 510, we had wonderful staff who worked plenty of weekends the rest of the year. We had leadership on both sides of the aisle, we had administration, and we had the appropriators. Together we did this. It was a great victory for sure. I know a number of us who were at the ken burns dinner earlier this week, and im very proud to say that he is working on a documentary on the nih that hell be unveiling i believe next year through pbs. I talked with dr. Collins earlier in the week. I know theyve done some extensive filming already, and i think that its important for the American Public to see in a nonbiased way the great work that the nih has done and is going to do. And obviously this bill is this legislation is going to find the cure so many families desperately want. I want to start off just by asking dr. Collins to explore a little bit more all of this project. I know a little bit about it. I know the unveiling is scheduled for next spring. I have some concerns about the privacy element of it in terms of what the individuals themselves will experience or some of the protections that might be there. How can we help and tell us a little more about it in what its going to be able to do. Glad to. And i appreciate your strong support and that of this entire committee for the concept were trying to pursue here which is the largest ever contemplated longitude nall cohort study of individuals across a wide range of ages, ethnicity, societyio economic status, race, and so on. And this is going to be a plarm for discovery for almost everything you want to know about allowing people to stay healthy and what happens when imness strike. I appreciate your mention of the ken burns film. And i hope members of the committee had a chance to see earlier this year the first in humans series that was about what its like to be involved in a Clinical Trial and what goes through all of those experiences in terms of trying to find answers for untreatable diseases. It was inspiring and emotionally powerful. How long does this beta test take for the individuals when they come in . Its a blood sample. Its a series of fairly simple physical measurements. Its answering a whole series of questions in a questionnaire at your own convenience. And it is of course a detailed consent process so that people know what in fact theyre getting into. You asked about privacy. Everybodys worried about that and we are as well. This is a program that has to maintain the highest standards of privacy and security in order to be credible. And we are working with partners that are top of the market in terms of doing that. One of our major partners is verily and google. All of the patientfight identifiers are stripped off before any of the data is moved into a location where researchers have access to it. And everything is encrypted end to end. Weve already been doing a series of penetration tests and hackathons to see whether there are weak sfots in this enterprise and so far its looking really good but were not going to do a full launch until were absolutely convinced all those parameters have been taken care of. So that volunteers participates in the program, how often will you come back to that individual and what information will they continue to transmit over the rest of their lifetime . Thats critical. We want people to feel this issing . Theyre proud to be part of pf redemption is critical over decades. They will be getting information back about themselves in terms of blood tests, their dna analysis which is going to get started sometime next year, as well as giving them information about how they fit in with the rest of this million strong people. So well be in touch with them at least every couple of months, seeking constantly to hear from them, what they like, what they dont like. They are really at the table here in designing this with us. So a lot of us are very familiar with the group, the private group 23 and me. People actually send saliva. Is this going to be somewhat similar to that . Is it going to be more extensive . So 23 and me as a commercial operation which many of us including myself have taken advantage of does give people Genetic Information back. And weve learned a lot from them in terms of how they do their educational materials to explain things that can be a little complicated in a sensible fashion that people can absorb. But were going to give more than that i know my time is rapidly expiring but i know your predecessor we worked with at the fda with 23 and me to make sure this could be launched in a successful way. So i presume that youll be working very closely with the fda on this to make sure it meets all the proper requirements. Fda has worked very effectively in this space. If i can speak for my colleague here in terms of figuring out how to do the right balance between protecting consumers against fly by night genetic test thats are giving you inaccurate information versus those where people are really interest sxid think we got the balance just right. Weve taken a firm based approach to the regulation of these kinds of consumer genetic testing technologies and announced that about two weeks ago where were going to allow the test platforms themselves to iterate and regulate the firm itself to make sure it has good s. O. P. S in place and lieu to go to mark with iterations of their tests the psalm way we approach digital health. Thanks. The gentleman yields back chair recognizes the scentll laidy from illinois miss schakowsky five minutes for questions, please. Thank you so much. I appreciate dr. Collins and dr. Gottlieb for being here today. One of the most critical components for the 21st century cures act was providing the nih with 4. 8 billion in new funding and these collars are certainly critical in advancing research sxm meaningful initiatives like Precision Medicine and the cancer moonshot as you mentioned dr. Collins but we must have a serious conversation about drug prices and we need to do more to address this growing problem. If we are spending billions to incentivize the development of new drugs i think we also have to make sure patients can afford those drugs. The development of new drugs and devices is meaningless unless the discoveries are affordable to patients. Its almost cruel to find a cure and then have it priced so high that a patient cant afford it. I hear from my constituents that the cost of the drugs they pay for that they need is far too high and theyre frustrated theyre paying twice for their Prescription Drugs, once in taxpayer dollars funding for drug discoveries and then again at the pharmacy. So dr. Collins, hoerz my questi question. I foe scientists dedicated their life, your life to making discoveries that make the world a better place. As nih is Funding Research that will lead to the development of therapies, do you think that patients should be able to afford the drugs that result from your nihfunded research and that hard work . So this is obviously a topic on many peoples minds. The designate for hhs secretary yesterday said yes, we do have a problem with drug pricing. Everybody agrees this is a serious issue. Nih has some roles to play but not to the degree that perhaps the public wishes or you might wish we do. What we can do is try to be sure were doing the front end of drug discovery, which is to identify the right targets and then to develop a pathway toward turning those into therapeutics as efficiently and accurately as possible so that the failure rate for Drug Development is not so incredibly high as it currently is. One of the reasons that drugs are so expensive is because the industry has to compensate for all the failures which is are over 95 depending on how high you count. If we had a success rate of 50 instead of 5 you can imagine how the equation would look a lot different our goal and the National Association for translation of sciences n cats is to fry to do 3we9er in terms of identifying ways to be more efficient ways we could do toxicology more cheaply and other things such as that but when it comes to actually having a role in determining cost, the price of a drug once it has left nihs hands its been commercialized which it needs to be we dont make pills, we dont really have any levers to pull in that situation. We depend on other places to do so. Let me ask you this. First of all, you mentioned a kind of calculation, how many drug how many failures there a are. We do not know that. Weve asked for transparency of how much is spent to develop. Wed like to see that data. Has the nih ever exercised what i think is its right under these licensed to ensure that publicly funded drugs are reasonably priced . I believe youre referring to the march in rights which are a component of the buy dole act. Weve looked at that and been asked on a couple of occasions to see whether that would apply in a case where a drug price seems to be unduly high and nih has played a role in its early development. But if you look at the language of the bill it really intends to cover a circumstance where a drug is simply not available to the public under any circumstances and then nih is entitled to step . This is a little different when its available but at high cost. Our legal experts dont feel the law puts us in a position to step in. I thank you for that i understand its outside of nihs purview to make su tced. But really i think we need to be partners in figuring out this piece because i believe some of the calculations and some of the prices really do say that many people are not going to be able to access the cures that are available that are shortening their lives. Glad to work with you in any way we can. I yield back. The chair recognizes the gentleman from illinois mr. Shimkus for fine minutes of questions, please. Welcome. Were glad to have you here p kind of exciting day and its fun otalk about this. What im enjoying about the hearing is hearing my colleagues on both sides address issues weve been been working on either separate at some time, then jointly. Gene green and i have picked up phil gingreys work and worked on the adapt act. My first question kind of deals were to commissioner gottlieb. Are there additional policies we might be able to do to even help in the guise of economic incentives that would help move on this antibiotic resistance attack and be able to get drugs quicker to the market if needed. Congressman, id be happy to work with you on thinking through what additional steps we can take. We do have a platform now and a tailwind of some really extraordinary legislation thats just been passed in recent years. As you know, the gain act did provide additional incentives through exclusivity for the development of antibiotic thats were targeted to unmet medical needs. The kinds of situations youre talking about. And were still in the early days of implementing lpat and the adapt act. Were going to put out guidance as i mentioned this summer sketching out the framework for how we intend to implement that. And we have had multiple preind meetings with sponsors. We think this is going to grow into a robust tool for trying to get earlier, more expedited approval of drugs targeted to these special situations. I think there are some things we could do to think about how we reimburse these kinds of products in the marketplace. To the extent were asking sponsors to help, antibiotics that tha are going to be used on an emergency basis or a very limited basis a reimbursement model where you pay for use might not be the most appropriate way to provide an appropriate incentive. Site licenses, these are things that have been considered in the past where hospitals might pay a licensing fee. That might provide more of an incentive. Thats obviously outside of my scope let me jump in. One hurdle we havent overcome we were told earlier in the prs ive been involved with was the issue of charitable vouchers which i didnt get across the finish line. Oso my colleagues understand theres a need, and that may not be the venue. If theres Something Else we can do that might get us to the table so we can send another signal. We dont have to talk about now. This is the time to raise that issue. Let me go on the same line of questioning about antibiotic resistance and talk about just where were at on the rapid diagnostic ability, rapid diagnostic tests to be able to identify quicker so we can intervene earlier. Any comments on that . As dr. Collins will probably opine as well, this technology is becoming more and more available at the point of care. We used to rely on blood cultures that would take days to grow out organisms and we would give Broad Spectrum antibiotics until we could tell what they were now you have the ability to sequence organisms or gaining the ability to sequence them at the point of care. Were doing things with respect to generation of next generation sequencing. Its going to be prnt to make these opportunities available. Were running a prize competition right now and again 21st century has had a specific call outs out to us to do prizes using the eureka part of the bill. For amr were basically asking xeef competitors to come up with the means within four hours of being able to determine whats the infection and does it have multiple Drug Resistance in the case of urinary tract infection or nooim pneumonia or sepsis. That would be a dramatic game changer. There are a lot of competitors out there. Theres 20 million bucks out there for the one who wins this nih from barda and i think that could be a pretty exciting moment if we could get the technology to that point. Yeah, and thank you very much. Im going to end on, this which is still a positive note. Im also very excited about the all of us campaign, university of illinois is involved with it and thats kind of part of my area. Its exciting. Same issue. We had a telecommunication subcommittee hearing yesterday ruling on big data, algorithms, all this stuff. Then i segued into my visit with Washington University which is close to my home, i live in the st. Louis metropolitan area. So i know that university well and i know the associated hospital that they work in conjunction with. They have been so excited about the passage of the 21st century cures act because in their research and i toured them just last week during the break and did alzheimers, new technologies that really drill down to the cellular structure. Antibiotics, which is one of the worlds in which i focus on individually. And they just reiterated the importance of consistency. And of course sometimes weve been inconsistent and the 21st century cures has established consistent streaming and commitment to what were doing in the healthrelated field. I want to thank you on behalf of the university of illinois and Washington University. I may in one sentence say thank you for what you did and the Innovation Fund for providing consistent support over the course of ten years for these projects which clearly are going to need that kind of sustained funding in order to be successful and its often difficult to see a path for sustained funding in the year by year appropriations. So thank you. The gentleman yields back. Chair recognizes the gentle lady from california miss matsui. Thank you, mr. Chair for the hearing and dr. Gottlieb and dr. Collins to be here today as we talk about the implementation of the 21st centuries cures act. As we Work Together on these bill, patients were always the center of our conversations and as we move forward patients are still at the center as we implement this bill. Im particularly concerned with research and Drug Development that affects patients with Rare Diseases because with small populations of patients its often very hard to get drugs and treatments through the approval process. I just cant tell you how many individuals come to me with their concerns in wheelchairs and with their stories. Because finding cures for Rare Diseases is not only important to their patients with Rare Diseases and their families but to all of us because you never know where a cures going to come from and often research and Drug Development on one disease may create results on another. We need to leverage all the tools that we have. Id like to hear about some updates of provisions i worked on in cures that were aimed at encouraging innovation for patients with diseases. Section 3012 and 16 of the law were designed to encourage the development of targeted drugs for Rare Diseases including allowing manufacturers to leverage data from previously approved applications. For a new indication. We see that all of the time with Rare Diseases as many patients use drugs off label as their oj options, drugs proven safe and effective but not for their specific condition. Dr. Gottlieb, can you provide an update on implementation of these provisions . If i may, congresswoman, i want to build on what you said. I appreciate your comments and your commitments to these efforts. To the extent there are challenges associated with drugs and Rare Diseases sometimes its difficult to enroll these trials. Also what we are seeing are situations where because the biological basis is so well established with some of these drugs and we can select which patients likely derive a clinical benefit, were seeing clinical benefit very early in the Development Process and that was the point of trying to see how we can apply the accelerated approval mechanism to achieve what you outlined, the ability to expedite these products to the market when he do observe an extraordinary clinical smons in an early stage trial knowing well be able to get the confirmatory evidence. Building on those two provisions you mentioned were going to be releasing very soon guidance that was first announced probably three four months ago we were developing which is a target therapies guide. Its going to outline very specifically how sponsors can get approval for products that are targeted to biological markers rather than certain diseased tissue states if you will. So tissue inagnostic drugs. A best example might be a cancer that might appear in multiple organ systems but be driven by the same biological market. If you can zrit a drug targets the biological mechanism you can get approval across all those different indications. Were also to the point where were making robust use in my opinion especially in the oncology setting of the provision that allows us to give supplemental indications more easily based on existing data in the Public Domain or references to literature rather than having to in many cases rep qulaikt the new Clinical Trials zplz indications where you have a very strong biological rationale to know the drug works there and that was the other point of my opening testimony stayed the ability to extend approvals in other settings that are proportionate to where the original profl was given. You approve the drug in a second light oncology situation and making it easier to extend it into a front line indication when the evidence starts to accr accrue. Thank you very much for that update. Dr. Collins, how can nihs Precision Medicine Initiative Benefit rare disease patients . Precision medicine as a concept is trying to get away from one size fits all to identifying the individual characteristics that are going to lead to better prevention and treatment. While the Precision Medicine initiative flagship called all of us is not particularly well designed to deal with Rare Diseases because even with a Million People there may be relatively few with a truly rare disease. The whole rare disease field is very attached to the interprets igs medicine idea. You can see whats happened with Cystic Fibrosis which was mentioned earlier where we now have therapeutics that are specific or the particular kind of misspelling that that individual has in the Cystic Fibrosis gene. Thats a good example. And we want to see much more of that because there are at least 7,000 of these Rare Diseases for which we know the genetic mutation but we dont yet have a treatment. We at nih are working hard with our colleagues at fda on trend which is part of the advancing center for advancing sciences because there are some of these disorders that are so rare that industrys not interested at least initially in investing in them theres more interest now than there used to be in industry and i think were making real headway in something the 21st century cures bill did was to give trend the ability to run phase 3 trials on those disorders which we did not have at ncats before and were grateful for that. Thank you very much and i yield back. Chair thanks gentle lady. Yields back. Recognizes the gentleman from new jersey mr. Lance, five minutes for questions, please. Thank you, mr. Chairman. And good morning to you both. Dr. Gottlieb from middlesex county, is that right . Very good. Dr. Gottlieb, throughout 291st century cures dialogue we heard about a number of innovative Treatment Companies were pursuing that would target specific genetic patients with diseases. Im the republican chair of the disease caucus in the house. This is of course encouraging but weve also heard there could be multiple genetic subtypes of each rare disease and that could complicate Drug Development and clinical testing in already challenging circumstances. Tone sure as many patients can benefit from these new technologies as possible and as quickly as possible as you know, section 301. 2 orders the fbi to rely on tata from a Company Previously submitted for drugs that use a same or similar technology. Could you elaborate a little further . I know youve been discussing this in ways in which the fda has utilized this authority to date and what we should be doing more perhaps here at the congressional level. If you, congressman. The provision you built into 21st century cures that youre referencing really anticipated the future and what youre seeing. The truth of the matter is its still early days in terms of the drugs were seeing that are targeting in many case thats are inherited disorders where you have a genetic change that drives a disorder but you have multiple subtypes that all produce the same clinical circumstance and the request becomes if you study one genetic subtype when is and how do you extend the approval into the other substiepz without requiring the sponsor tone roll in a Clinical Trial each one especially when each one might be only a handful of patients. We are currently having discussions with sponsors around this very principle. I think what congress built into the law is giving us the latitude that we need to be thoughtful about how we can think about this and extend approvals across the range of subtypes that drive a common phenotypes, and i think youll see us exercising that authority in some upcoming approvals and we also plan to address this it some extent in the targeted therapies guidance well be releasing soon. Thank you, doctor. And how does fdas familiarity with an Underlying Technology affect subsequent product applications and the supporting data the agency expects to be included . I think the our ability to understand how the products works and how it intervenes in the molecular basis for a disease drives our ability to make these extensions youre talking about and give us confidence that a drug that works in one setting is going to have the same clinical performance in another setting where there might be a slight genetic variation but at least to the same phenotype. What you referenced is instrumental in our ability to make these determinations. I was pleased language was included in the bill authorizing grant funding. The study and expansion of Continuous Manufacturing. New jersey has been a leader in this area including our State University rutgers, i know this as well. Bringing together Research Institutions and industry to advance technology. What steps are being taken by the fda to carry out the language included in the act regarding what i just discussed . This has been a very high priority for the agency trying to facilitate a platform for Continuous Manufacturing. Were going to continue to give grants to institution thats are going to continue to develop the tools that enable to us continue to move this forward. We think Continuous Manufacturing represents the future. Its going to provide a much more robust way to manufacture predicts, especially some of the newer products were seeing. We think it provides certain safeguards from potential drug shortages and i think it also might help us repatriate manufacturing back to the United States. That is driven by High Technology theres lends itself to domesticating that process as opposed to outsourcing. Im hoping this will also help us build up the industry. I encourage repatriation. Congratulations on your appointment and confirmation. Dr. Collins, i look forward to being with you again at nih particularly on rare disease day. Mr. Chairman, i yield back eight seconds. The chair thanks the gentleman. The chair recognizes the gentle lady from florida miss castor. Five minutes of questions. The 21st centery cures acts funded nih to 23r50i6d support for Biomedical Research in the nih Innovation Fund. This focused on four Vital Research priorities address some of the greatest challenges in disease prevention and treatment. Back home in tampa were home to the only nci designated Cancer Center in florida, the moffett Cancer Center, and just in my short time in congress ive been floored at the progress that weve made in treatments and cures for cancer, and yet theres so much more to be done. And i think the beau biden cancer moonshot thats part of 21st century cures, it accelerates screening and treatment for cancer. Can you discuss some of the research that the Beau Biden Cancer Moonshot Initiative is funding and how it may contribute to addressing the burden of cancer across the country . Yes, id be happy to. We convened a Blue Ribbon Panel of some 28 individuals who were the most visionary folks we could identify to figure out what would be the best way to take Additional Resources coming forward from 21st century cures and do things we otherwi wiswis wouldnt be able to do. And they came up with a set of ten different wrarz that were ripe for further investment. Ill just mention one because its so much on everybodys mind right now as a source of great excitement and that is the area of cancer immunotherapy. This, which for 40 years has been labored by a very smaum gruch of people, particularly dr. Steven rosenberg at the nci, has arrived in the last few years. As the most Exciting Development in Cancer Treatment in a very long time. Weve had surgery. Weve had chemotherapy. Weve had radiation. That was sort of it. And now we have a fourth modality which when it comes is capable of taking somebody with widely met astatic disease from mel noechla or somebody with advanced luckemia luke yooemia and not just providing a response, providing what appears to be a cure. When you see that it is enough to make you believe we should put every bit of energy in it to make it work for all cancers. Thats what the moonshots making it possible for us to do. Working with industry and this partnership we Just Announced a month ago were trying to figure out why doesnt it work when it doesnt and what could we learn from that . Why doesnt it work for pancreatic cancer . Why doesnt it work for most cases of Prostate Cancer or Breast Cancer . It seemed to work for a certain subset. But the immune system ought to be able to recognize those cancers too. What can we do to find those answers . Working closery with our colleagues on fda with this. You have probably heard the first socalled car t cell approaches to leukemia and are being approved. So thank you to the whole congress for recognizing this was one of those areas that was ready for a big boost and the 300 million i share your excitement for immunosome therapy. Ive heard it directly from my researchers at home and from families now that they have additional hope in their lives. How about alzheimers disease . Give us the same sketch for hope and promise now under 21st century Cures Innovation Fund for alzheimers. 21st century cures funded the Brain Initiative, which is an incredibly ambitious effort to understand how those 86 billion neurons between your ears do what they do, each one of them with maybe 1,000 corrections. Thats going to provide them with information about neuroscience we just have not had. Theres an effort directed at alzheimers disease and congress has been increasing our funding it hasnt been enough in the past. And its been going up wonderfully well. Were now in a position to take both the basic science coming from the Brain Initiative and the Clinical Applications possible flu the regular appropriation and really turbo charge the efforts. We need those answers as all of us us know who look at the 5 Million People that are already affected and look at whats going to happen in the next few decades w9 aith the aging of ou population if we dont come up with a solution. Were on the path to figure out what to do to prevent these disease in those who are at high risk before it even strikes. How can the public monitor progress here . You might go online and do a Google Search but that wont get to the heart of the matter of what is happening over the coming years because of these investments. So we try our best through nih to make Public Information available but we dont think its appropriate for us to be out there marketing what we do. We are educators but were not necessarily doing the best job of communicating to people who rirnted. We count on the media or we count on interested advocates to get w0rd out, particularly the Alzheimers Association and other advocates like that. And i do think the consciousness of the public has been raised about this but in terms of tracking whats happening month by month we need better opportunities to do that. I agree with you. Thank you very much. I yield back. Chair thanks the gentle lady. Gentle lady yields back the chair recognizes the gentleman from florida for five minutes, please. Thank you for your testimony. Dr. Collins, as one of the cochairs of the congressional kauk caucus i was proud we included a neurological Surveillance System as part of cures. Its estimated that 1 in 6 people suffer from neurological disorders. This neurological Surveillance System would gather information about patients including inside dennises, prevalence and also demographics and outcome measures. I know the cdc would run the Surveillance Program but nih has the experts that would use the data. How will having this Information Available to nih assist Biomedical Research at the agency and in the Research Community at large . Thank you for the question. That feature of 21st century cures which as you point out is assigned to cdc to develop this neuroscience assessment of prevalence and incidence of neurological conditions is certainly something that if the data were available we would find it quite useful. I think at the present time because of the funding issues cdc has not been able to act upon that. We are certainly deeply invested in parkinsons Disease Research including working with industry on something called the accelerating medicines partnership. Its possible 2459 athat the als program thats going tone roll millions of americans over the next three or four years will provide some useful information because some of those folks will have parkinsons quite a few in fact when you consider how frequent the illness is and the fact were talking about a million medium but it wont quite substitute for what you asked cdc to do. I think this is a circumstance where ability to get the information is not trivial. It takes a lot of resources, a lot of time, and heres where cdc as i understand it is having trouble figuring out how to actually do what cures act asked them to do. Dr. Gottlieb, during my cures round tables in my district i heard from a woman who had a child with duchesnes muscular dystrophy. She stalked about two hurdles the challenge of acceptable biomarkers and the need to incorporate patientreported data. In cures we had a provision dealing with patientreported data. You mentioned in your testimony theres a new section on Patient Experience data. Can you update us on when that came online, how the fda will incorporate that data in the review process, and what does fda hope this type of feedback will lead to . Were starting to do that right now. Congressman, cures decided to build in better measures of the Patients Experience as a measure of how we look at efficacy for purposes of approval. I think the opportunities that were going to have that im most excited about is better opportunities to look at things like physical performance. You talked about duchesnes muscular dystrophy. One of the objective end points we used in measuring outcomes in that setting is traditionally a walk test. Look at whether or not new therapies are approving physical function or slowing the rate of decline in that clinical setting. What if we had a tool that al louz a patient to wear a device, maybe its a watch that measures their physical performance in routine daily living . That might be very preferable to trying to do it in an artificial setting of a Clinical Trial where youre doing it in a sort of random fashion when a patient comes into a Doctors Office for an evaluation or checkup if youre able to look at a patient in their daily life that might provide a much more objective measure of how a drug might be impacting their life. These are the kinds of opportunities that i think we have with new technology. Theyre the kinds of opportunities i think that this legislation is giving us the legal basis to make better use of. And this is what im looking to the future for. Very good. Thank you. Again, dr. Gottlieb, the other issue that was brought up in my round table was a challenge of acceptable biomarkers. This has been an issue brought up in prior hearings. Can you update us on changes fda has made and how can we encourage the greater use of bio markers particularly for rare disease patients where traditional Clinical Trials may be too hard for them to theyre limited in population as you know. So if you could answer that id appreciate it. Thank you, congressman. Here again cure has provided us with new opportunities sorry, that was my watch talking to me. The legislation did provide us an opportunity for the incorporation of Drug Development tools into our regulatory process. We have a bio marker Qualification Program now. We have eight bio markers that are under consideration all by various consortia. We also have another program that allows us to develop sort of other kinds of measures that can measure efficacy or performance of patients in clinical settings and were going to qualify the first for major depressive disorder very soon, a new survey tool that looks at outcomes for patients who are suffering from mdd in the clinical setting. Again, this is a qualification process that was created by cures as well. So these are moving forward. Were seeing a lot of interest in these kinds of opportunities and we think this is going to provide a very important framework for the future. Excellent. Thank you very much. I yield back, mr. Chairman. The chair thanks the gentleman. The chair recognizes the gentle lady from colorado five minutes for questions, please. Thank you very much, mr. Chairman. One of the proudest aprochlts we had in 21st century cures i thought was the establishment of the Oncology Center of excellence at the fda. Dr. Gottlieb, as you mentioned in your testimony, this first of a kind center enhances coordination between the fdas drug device and biologic centers to leverage the agencys expertise on cancer im hoping the oce model will be successful that we can use for other diseases. Can you tell us what the center is already doing to advance the working Cancer Treatment . So weve already been able to use the center to do consolidated clinical assessments on a range of products. I think the most profound sort of manifestation of the opportunity that such a center affords us is what we saw with respect to the approval of two gene therapy products targeted to some rare cancers that i think do provide a meaningful opportunity and perhaps a profound opportunity for patients to get more advanced and potentially more curative therapy that settings where there wasnt very good available therapy prior to the approval of these products. The essential point is that the center allows us to consolidate the clinical review, take a more multidisciplinary approach to how we look at the evaluation of efficacy and safety around these products. And we do think that this kind of center approach represents the future of how we want to approach other therapeutic spaces for other diseases. Immunology. A center for neuroscience. These are things were contemplating. Now, its very important that we get it right in the setting of oncology since this is our test case and our first model for this. And can you do more if you get full funding for this center . Well, we appreciate the what congress tried to do in appropriating funds to the center through nih. As dr. Collins will attest, theres been some challenges associated with transferring those funds to fda, some legal challenges. We look forward to continuing to have discussions about how we could how we could fund this. It hasnt been funded to date in part because of the challenges associated with how the money was allocated to nobodys fault. Its a frustration for us too. So if theres something we can do to help let us know. I just have i have a couple of more quick questions. I want to ask about the irb provisions. You guys know that for about ten years i worked on a protection for patients and research act that would streamline the irb process and i was really happy to get some of that signed into law as part of cures. I know thatment of the irb provisions in cures have not been implemented yet but im hoping maybe you can talk to us about how the secures provision thats streamline the irb process will help reduce administrative barriers for scientific research. Well start with you, dr. Collins. Quickly. I think it has been very helpful to have those features in the cures act. One thing that we are now insisting upon is that multisite trials which used to have multiple different irbs, each of which might have some opinions about the wording of the consent form, we no longer think thats the right way to do things and having a sing male irb for multisite trials has become the norm and basically if thats not to be the case we need to understand why. And your support for that has been really helpful because we generally lost many months in the process of we lost many months and we lost many millions of dollars every time we did a research study. Indeed. So this makes a lot of sense and we appreciate the tints to do that. In sort of a related issue, and thats the Clinical Trials. The cures provisions established processes at the fda to qualify bio markers and real world evidence into trials. The committee recently worked on the cures provision in the fda reauthorization act. What more can we do to improve the way and modernize the way were doing Clinical Trials . We can start with you, dr. Gottlieb, on that one. I think theres a lot we can do. And here again, cures gives us a platform for doing it. This is one place where i think were trying to take the spirit of what congress did and wanted of what congress did and wanted us to do and extend it. Captions Copyright National cable satellite corp. 2008 captioning performed by vitac