Transcripts For CSPAN3 Hearing On The Future Of Precision Medicine 20240622

Encouraging for them and all of us because these practices not only help Climate Change but produce more food and make and they make themselves more resilient against drought so its one of these amazing winwinwin but very few of us really appreciate it. Thats what anna is so good at getting across and we hope that our new book can help too. Thank you for that great question. [ applause ] tonight, our Washington Journal Program focusing on community policing. Well reair yesterday mornings entire program from richmond, virginia with your cools and comments, the Richmond Police chief and head of the Police Training academy and richmond mayor dwight jones. It starts at 9 00 p. M. Eastern on cspan. Former president jimmy carter held a News Conference this morning in atlanta and briefed reporters about treatments for brain and liver cancer. Well show you the event on cspan. Heres some of what president carter had to say. Spots on the liver and the operation and that same afternoon, we had an mri of my head and neck and showed up it was already in four places in my brain. I would say that night and the next day until i came back up to emery, i thought i had a few weeks left but i was surprisingly at ease. You know, ive had a wonderful life and friends and exciting and adventurous existence. So i was surprisingly at ease, much more so than my wife was. But now i feel you know, its in the hands of god and my and ill be prepared for anything that comes. Former president jimmy carter from earlier today. You can see all of his atlanta News Conference tonight at 8 00 p. M. Eastern. 5 00 p. M. Pacific on cspan. National institutes of Health Director dr. Francis collins updates the Senate Committee on president obamas Precision Medicine initiative. The Initiative Funded through the 2016 budget is a Research Effort designed to improve treatments for diseases like cancer and leukemia by tailering health care to the individual not a one size fits all approach. The Senate Committee on Health Education will come to order. This morning were holding a hearing on continuing americas leadership realizing the promise of Precision Medicine for patients. Well have have an Opening Statement and introduce our panel of witnesses who are getting to be very familiar to us. Were grateful for you for coming. After that well have time to ask five minute rounds of questions or two, depending how many senators are here. Were here to discuss the exciting new direction in our health care called Precision Medicine. What does that mean . Those of us in the room are a good representation of the United States population, centers for Disease Control and prevention estimates nearly one in 10 of us would have diabetes. If doctors could use Precision Medicine, that is if they could look at our individual dna and genetic makeup of our disease, perhaps they could potentially tailor treatments to each individual than to a general category of diabetes. I was visited this morning by the head of philadelphia Childrens Hospital who talked about their work in identifying a defective gene or mutated genetic defect that causes blindness and how they have developed a therapy that restores the sight in a child because of therapy directed for that specific genetic defect. This is happening with cancer treatment, doctors can look at the mutations of the cancer cells and it says how to treat it. This week reports that genetic testing on tumors is already starting to become the norm in the big cancer hospitals. Like sloan kettering, all incoming patients automatically have tumors sequencesed. This is possible because of extraordinary achievement by a great many individuals on sequencing the human genome. None more important that dr. Collins, here today. Today we want to discuss what the National Institutes of health and industry are doing in Precision Medicine and how the food and Drug Administration will regulate those innovations and electronic hgt records can affect the ability for the patient and Health Care System. Its one of the exciting new frontiers in medicine. Senator murray and i are working on a initiative to ensure our federal agencies are equipped to review the medical products and processes produced by this kind of cutting edge medicine so an american patients arent waiting on sidelines. Our Innovation Initiative is not just about Precision Medicine, but Precision Medicine is an important part of our initiative. President obama announced a Precision Medicine initiative in the state of the union this year. Detailed his plans and an event at the white house. I attended that to demonstrate my support for it. The president proposed as part of the plan mapping genomes of 1 million individuals and making the Information Available across the country. We look forward to hearing more about that from our witnesses and similar private efforts under way and imed in hearing about the competition and possible collaboration with Childrens Hospital of philadelphia that i mentioned as 215,000 individual children genome sequenced there. I wonder how useful that would be to the 1 million that dr. Colins is putting together and how this scientific entrepreneurs and philadelphia, other places, how that relates to the president s proposal for a million individuals. I look forward to hearing about the potential cost of Precision Medicine. We know that cost to sequence the human genome have been reduced significantly in the last two decades. Dr. Collins testified, i believe he said 15 years ago it cost us about 400 million to sequence the first human genome and today about 1,000. Very often in Health Care Innovation initially increases our cost, that doesnt mean we shouldnt innovate. Innovative new products can increase costs but in the long term actually decrease Health Care Costs. Take alz zimers which according to the Alzheimers Association cost 226 billion along about other dementias. If we can use Precision Medicine to delay onset or cure that disease, we can save precious dollars in our Health Care Systems and alleviate some of the grief and pain associated with it. The committees also spent some time and spend more on improving the Electronic Health records. The federal government has spent 28 billion to drive the adoption of these record systems and the result is that doctors dont like the systems and many say they disrupt work flow and interrupt the doctor patient relsship. Senator murray and i have begun working to identify the five or six things we can do to help make the failed promise of Electronic Health records something that positions and providers look forward to instead of something they endure. We look forward to working with you on that and with secretary burwell and being able to report maybe early next year some resul results. We have to get to a place where the systems can talk to one another and where doctors particularly smaller physicians offices want to adopt the systems, cant afford the cost, can be confident they are investment will be a value. Dr. Collins has told us and i heard from many others, that a properlily functioning Electronic Medical records system is tremendously important to the president s Precision Medicine initiative. Number one, can help to assemble the genomes of 1 million individuals and if we want to make it useful, this is complex stuff. Its going to take computers that operate easily and with a click of the mouse to help make it possible for doctors to prescribe prescriptions for individual patients. We would like to hear if we know dr. Disalvo, how the 11 billion effort by the Defense Department would relate to the 28 billion we already spent and whether those are compatible. Theres a lot to talk about today. This is a tremendously interesting and important effort and i look forward to the witnesss comments. Thank you, mr. Chairman, thank you to all of our witnesses for being here today. Each of your agencies plays a Critical Role in the topic were going to be talking about. Im grateful to have you all here to share your expertise. Focused on one question, what can congress do to help all patients and families get the safest, most effective treatment and cures more quickly . Our conversation today is the promise of Precision Medicine and it is a crucial and truly exciting piece of the puzzle. Theres no question we are at a critical moment in the medical field. Researchers in medical experts are increasingly finding ways to treat patients not just as the average patient but instead based on their own unique characteristics in history. This is like the difference between getting eyeglasses based on the average prescription and getting eyeglasses based on your own prescription. Its huge. Especially for patients and families across the country who are waiting and hoping for better treatments and cures. Im proud that my home state of washington is home to several institutions that have been pioneers in this area, includes Fred Hutchison Cancer Research center and university of washington using this Precision Medicine technology to tackle Breast Cancer and eye disease and alzheimers disease among others. Im glad we have the opportunity to discuss the ways in which Precision Medicine is changing and improving lives and how congress can help advance this new frontier in biomedical innovation for patients and families. The president proposed making significant investments in Precision Medicine. Fiscal year 2016 budget supports a Bold New Initiative to exploit the advances in Data Management to support the shift away from this one size fits all medicine and towards treatment tailorred to specific individuals. This proposal can do an enormous amount to accelerate the advancement of Precision Medicine. As i discussed with dr. Collins in our appropriations hearing last week, im deeply troubled by the steady erosion of nihs purchasing power over the last decade. Last Congress Democrats and republicans were able to come together to replace the harmful sequestration cuts to the investments in nih and fda and other critical priorities like education and infrastructure and defense. Im really hopeful this year despite the budget proposals put forward by our republican colleagues, we will be able to work across the aisle and find a way to prevent these shortsided cuts from kicking in again. This is absolutely critical to the kinds of investments we need to keep make our families help our families and grow our economy, including Precision Medicine. One of my Top Priorities on this committee is looking for ways to continue improving the quality of care patients receive and supporting Precision Medicine is essential to that goal. By offering patients and providers more and much Better Health information, patients and consultation with their doctors will be empowered to make informed decisions about their care and our Health Care System will be better equipped to put their needs first. I do want to note the protecting privacy will be an important challenge throughout this process, just in the last few months we have seen serious Security Breaches impacting families, personal Health Information. That is unacceptable. As researchers and providers and patients gather and use more Health Information, we need to be aware data is being created that cyber criminals will want to exploit. That means well need to develop stra strategies to protect privacy that meet todays challenges. Were investigating the certainty state of Cyber Security in the Health Sector and it is clear this needs to be an all hands on deck effort with providers and insurers and government working together. Thank you to all witnesses for being here today and i want to thank chairman alexander for holding this hearing for patients and families in my state and every state across the country. I look forward to working together, mr. Chairman with you and other members of this committee to support this important initiative. Thanks, senator murray. To underscore what senator murray said, this specific topic is a initiative of the president in which the committee and bipartisan way is very interested. We expect to get a result. We have three witnesses now, i ask senator cassidy if he would like to introduce the first. Dr. Karen disalvo know each other from way back. In her honor we made the spread tulane green today so anyway and dr. Disalvo for Health Information technology at the office of the National Coordinator of information technology. The lead Agency Charged with formulating the government i. T. Strategy and coordinating policy standards and programs and investment. Ive been impressed she has come to me personally and theres a friend back home having a lot of problems with her electronic record and she called her and they spoke at length. She clearly recognizes int interoperability as key. She was Health Commissioner for city of new orleans, including and after hurricane katrina. We expect to get to know you pretty well because the Electronic Medical records system and youre on point for that according to secretary burwell so we look forward to that. The other two witnesses are here about every other day. Dr. Collins of the National Institutes of health, largest supporter of Biomedical Research in the world. Been director since 2009. Of course, hes known among other things for his leadership of the International Human genome project, completely sequencing the human genome in 2003. The director of the center for devices and radio logical health and food and Drug Administration more than five years. They are responsible for ensuring the safety and effectiveness and quality of medical devices and sharing saflt of radiation emitting products fostering innovation. In one year that has been detamed to this committee as part of kennedys staff. We welcome him back. If the witnesses would summarize their remarks, in about five minutes, we would appreciate it. We have senators here who want to have a conversation with you. Good afternoon, distinguished committee members, its an honor to appear before you to discuss how to advance Americas Health towards a new era of Precision Medicine. Earlier this year the administration unveiled a Precision Medicine initiative, a bold new Research Effort to revolutionize how we diagnose and treat disease. We believe the time is right for this initiative and nih and fda and onc will work hard to achieve this mission. Historically physicians had to make most recommendations based on the expected response of the average patient. This one size fits all approach works for some patients and some conditions but not others. Precision medicine is an innovative approach that takes into account individual differences in patients genes and environments and lifestyles. The concept is not entirely new. Blood typing for example has been used to guide blood transfusions for almost a center are you. The identification of the b rca 1 and 2 genes made it possible to provide options for women at high risk of breast or ovarian cancer. The gene implicated in cystic fib rose sis discovered in my own laboratory, has led to widespread availability of targeted therapeutics. By the development of powerful and affordable methods for characterizing personal biological information, that includes genomics, the Widespread Adoption of Electronic Health records and recent revolution in mobile Health Technology and computation tools and patients are interested in taking part in research. All of these will help make possible the dream. With this in mind were thrilled to take a lead role in the multiagency Precision Medicine initiative. In the near term, this initiative will focus on cancer. Ak selling rating efforts to develop Precision Medicine strategies for a wide range of adult and pediatric cancers. It includes the analysis of large numbers of individual tumors to see what gene mutations are driving the malignancy and matching that information with available targeted therapeutics provided by partners to optimize responses for the individual. Simple blood tests will be developed that can detect early sponsor resistance to drug therapy. And combinations of targeted drugs will be tested to see how best to achieve not just a remission but a cure. To put a human face on this, id like to paint a forward looking picture for what it could deliver for cancer in a few years. Consider the hypothetical case of lilly, a 52yearold woman asian descent, in 2018 after battling bronchitis and persistent cuff for several months she goes to her doctor and orders a long ct scan along with a must blood test supported by the Precision Medicine initiative. To look for dna and other biomarkers circulating in her blood. The ct scan detects a tiny spot that could be either inflamation from bronchitis or cancer, not clear, but the bottom marker clinchs the diagnose ses revealing a genetic mutation that occurs only in patients with cancer. In 2015, today her prognosis sis from this cancer would be pretty grim. In 2018, that could all change. She is treated with surgery to remove the tumor and the tumor under goes analysis and lilly is treated with a targeted drug originally developed for skin cancer but has the right properties for her cancer and immuno therapy to kill any cells looking in her body. With this treatment a decade later she remains cancer free. That is a hypothetical but quite realistic example of what the cancer component of this initiative could achieve. As a longer term goal of this initiative. Nih will launch a National Research cohort of 1 million or more volunteers. We will play an active role how the medical and genetic and environmental information is used to prevent and manage a broad away of diseases. Participants, some recruited from cohorts and new volunteers, will be centrally involved in the design and implementation of this process. They will be True Partners but the appropriate privacy protections they will be able to share genomic data and biological samples. Participants will have access to their own Health Related information. New approaches for detecting and analyzing a wide array of variables will be nishly tested in small pilot studies, focused on prevention of disease and greater numbers of people over longer periods of time to collect valuable data that will be a great benefit to researchers and patient partners. Let me quickly give an example of how this can benefit a participant in the initiative but provide evidence. Consider the case of 38yearold Precision Medicine initiative participant carla. Its 2020. Carla feels perfectly healthy. She welcomes the chance to tryout a wearable sensor that monitors her pulse and Blood Pressure and physical activity and sleep patterns but discovers her Blood Pressure usually runs 150 over 100, increasing her risk of stroke and heart attack and kidney failure. Nationwide 78 million americans, one out of three adults have high Blood Pressure. Many dont even know it. Whats worse, nearly 50 of those diagnosed with hypertension do not have it under control. Carla consults with her doctor who confirms the need for treatment and suggest she take an inexpensive die youre receiptic drug and she contains a smart bottle connected to her smart phone returning to her Blood Pressure to the normal range avoiding crises. It will also bring many types of Health Care Monitoring into this new century. Current evidence suggests that the annual physical exam and associated lab tests may not be as useful as one would hope but new opportunities for specific indicators of individual health are emerging. This will provide a powerful opportunity to assess such strategies rigorously to see if they provide clinically valid information and more importantly Better Health outcomes for the american people. In closing, let me emphasize that the impact of the Precision Medicine initiative will extend far beyond the individuals who volunteered to participate and it will push the fron tiers of discovery across the entire spectrum, from basic science to translational science intent on moving Research Discoveries into practice for maximum benefit. Given the size of the project and real world nature, evidence of improved Health Outcomes derifd from in initiative will be attractive across u. S. Medical care. With sufficiency resource and scientific and medical and participant communities, the future of Precision Medicine appears very bright. We look forward to working together to make stories like those of lilly and carly a reality. That concludes my testimony. Thank you, dr. Collins. Dr. Disalvo, welcome. Thank you, senator, alexander and Ranking Member murray and others distinguished senators, for the opportunity to be here today with my colleagues. Im the National Coordinator for Health Information technology at the department of health and human services. When i was a medical student at tulane, i could have never imagined that in my career i would see medicine on the front tear of such a significant transformation. As a still practicing doctor it is thrilling for me to know were on the cusp of being able to customize treatment for the patient in front of me based on genetics preferences and Key Information instead of having to treat them as the average patient. Precision medicine is not just a theory, its all right changing practice and saving lives in the u. S. We wouldnt be on this cusp but for Health Information technology, which is foundational to the president s Precision Medicine initiative. The office of the national koord ator is the onc responsibility to advance the health i. T. Infrastructure for a sixth of the u. S. Economy health care. We do this through a minimum of programs and assistance and seek to spur and support innovation to help address and cordon advancements like Precision Medicine. At the same time, we want to provide clear and steady direction. Onc has responsibility to ensure that all consumers are engaged and their interests protected. Congress created significant momentum when it passed the high tech act in 2009. The act provided funding to support adoption of Electronic Health records and technical supports for hospitals and doctors on front lines as they made the transition to use them. As a result of High Tech Program and the hard work of providers, we are bringing health care into the digital age and we have reached a Tipping Point. The Strong Foundation of health inasmuch as Technology Makes it possible to brill to the bedside personalized treatment through Precision Medicine. The data and Electronic Health records married with advanced anl lit tick and information from mobile Health Devices and other sources of data, will provide the picture of a Persons Health and needs. This picture is necessary to identify the right prevention and treatment and not only the most effective but also most desired by the patient. This is not just a vision about what might come but a reality already in places like tennessee and maryland, nebraska, florida, i could go on. Thanks to tools built into the Electronic Health record, doctors are able to tailor treatment today. The university of Florida Health where a parents cardologist can order a test to see if they carry a particular variant of a gene. This will help them know if they are using the best medicine to prevent a future clot in the patients heart. This means at a critical time in someones life a doctor is choosing the right life saving blood thinner specific for them. Though this kind of treatment it exciting, it is indeed only the beginning. We have much wosh to do ahead, to see this is available to everyone in this country and as part of routine care and onc stands ready to undertake this work. To get there we need to stay the course in adoption to see every american has an Electronic Health record and go beyond the pockets of data exchanged and achieve true inneroperability. And establish standards for most fundamental clinical information shared by all. We will also need to establish standards for new data necessary for Precision Medicine, including genomic saxt and environmental exposures. Well build a trust framework that respects individual privacy and establishes Strong Security protections. We will work with the private sector to establish openly available apis which are doorways to unlock data. Well remember what i hear consistently from consumers who are a principle customer. They want to be able to access and share their Health Information, including the scientists if they wish, without blocking or delay. The president s Precision Medicine initiative is one of the most exciting ways we can bring the right care, the right presengs to the right patient. Only imagined a few years ago. Because of advances like this and the Underlying Technology that we are on the cusp of realizing better care and health for anyone. Onc stands ready to help further precision colleagues with our colleagues and congress. Thank you. We look forward to your questions. Accurate reliable and clinically meaningful tests. Its the results of the test which determine which patients get which drugs or treatments and whether or not they get them. In precise medicine, results from bad tests, you have miss diagnosis and get the wrong treatment or no treatment at all, as a result patients get harmed and health care koflt costs go up. Fdas role in the initiative is primarily focused on advancing technology called next Generation Sequencing or ngs. Ngs tests can sequence long segments of a patients dna or entire genome. As a result, were moving away from the model one test, one disease. But a test that can identify one of many different diseases or even the risk of developing that disease. But today there are significant barriers in place for advancing that technology and affect the research and development and affects our ability to use this optimally in health care. Let me tell you what some of the barriers are and what were doing about it. If youre making a test and want to know, is it accurate, reliable and clinically meaningful . One, does it accurately measure what youre trying to measure . In this case, genetic var yans, you identify the right variance. And secondly, you want to know, is there a Good Relationship between what you measure and a particular disease. A test for Breast Cancer, we call that clinic cal validity. Thats difficult to do for next Generation Sequencing. Heres why. Think about the human genome, you have 3 million variants and there are 3 billion base pairs. Now you want to know how accurate this next Generation Sequencing test to measure all of that. Normally you look at each of the variant. You cant access the accuracy of 3 million variants but they are good standards so people are struggling to make sure are the tests accurate. Then you want to know is it clinically meaningful . You have data for that. Many of these variants are uncommon. Its hard to get a lot of clinical data. Its difficult to do clinical studies and it tends to be sigh lowed in the institutions doing the testing. So in december, we proposed an entirely different framework for the oversight of next Generation Sequencing tests, tests weve regulated for a long time and we developers struggled on what to do with it. For an lit cal abit built, we need subsets of genetic variants, if you can show accuracy, its reasonable to infer youre good at measuring the other variants, we gave 2 million to the National Institute for standards and technology to work with the Scientific Community to come up with a very first reference standard for the genome and they just released that last week. Under this initiative and with additional funding support well continue to work with nist in the Scientific Community on developing additional reference standards and developers wouldnt have to come to the fda to show they are analytically valid. The clinic cal validity, we need to leverage data bases where we get all of that Genetic Information and make sure its standardized and of sufficient quality. Were partnering with francis and his team to take advantage of a database, clin bar and clin gen and working with them and Scientific Community, develop standards and best practices for having data bases and doing curation and having clinical interpretation. You know what happens today, you can send your blood to different labs and you can get different results. Thats what happens. So it may be because you missed the particular genetic var yans or interpreted it differently. With those standards in place, we can now have consistent accuracy in testing and consistent clinical interpretation and reduce the time and cost to spur research to advance Technology Developments and ultimately achieve Better Health outcomes. Thank you. Thank you, dr. Shuren. Well have a round of five minutes questioning. In 1980 when i was governor of tennessee, i had the big idea that all 8th graders would become computer literate. I met with steve jobs and bought enough mack computers, big tall things then to put in all of the middle schools. It was a great idea, sounded good. Forgot something. I forgot teacher training. So nobody really knew how to do it. I didnt think it all the way through to the end. I think we have something of the same problem with our Electronic Health care record system. We spent 28 million, its a great idea, holds great promise but its not working the way its supposed to. The current standards for meaningful use arent clear. Upgrades are expensive, the systems dont work well enough to share the data. We hear its expensive to share the data because of the relationship between vendors and doctors. Some of the doctors call this date at a blocking. You just released a report on data blocking describing these concerns. Senator murray and i have set up a Group Working Group to work on this because of the larnl amount of interest in our committee on the subject. My question, will you work with us, this committee, to identify the five or six steps we could take to get our Electronic Medical records system functioning well enough so that it supports not just Precision Medicine effort that we have but so that it functions into something physicians and providers can look forward to using instead of enduring . Yes, senator, very much look forward to working with you all in identifying ways that we can make this work for doctors and others on the ground, on front lines, thats where it really matters where the work flows are sometimes not the way they ought to be. And so we look forward to that and you can count on our participation actively. We have efforts under way as the senator is likely aware, weve been working through our rules for meaningful use for certification to blocking report and other strategies but we know theres more work to be done. We look forward to that. Good, what im talking about here is again, getting results. Identifying the five or six steps we should take, you should know them better than we really, although the doctor has personal experience in this hell give us im sure. Going step by step to get them down and you can do them by administrative order, terrific. If we need to do anything, well include them as part of the initiative and begin to do what we ought to do. Dr. Collins, 1 million genomes, i was advice itded by the philadelphias Childrens Hospital, they have 250,000 sequenced genomes. I have one question, how many of these are already out there . I mean, you want to assemble a million, they have 250,000 in philadelphia. Dr. Bitener wants to assemble a million. Can you get the million simply by going to the philadelphia Childrens Hospital and using some of theirs. Thats a great question, senator. Were trying to come up with every possible way to assemble this million strong cohort by taking advantage of things that have already been done instead of having to start from scratch. We will in fact have a major meeting in your state, may 27, 28 with a number of those who have been managing these large scale cohorts gathering together to see if there is a way to put them together in a way that can you estimate the number of agagenomes that have been sequenced. Some people are referring to a sampling of some of the base pairs in the genome, a snip chip. Some are talking about sequencing the parts that code for protein and we call that the exome. To do a whole genome sequence is substantially more expensive but it is becoming quite affordable. Most of the couhorts out there have not done whole genome sequencing and the group in philadelphia is doing that with some patients but not many. Dr. Ventener has the ability of doing a lot of whole agagen ome sequencing. Senator murray has stressed the importance of making sure a Representative Group of everyone is included in this. What about children . The suggestion was made this morning that sequencing genome of a child for a genetic defect leading to a particular disease can be less complicated than for an older person who may have a more complex disease. Will you include children in and the single gene therapy treatment as part of what you do . That is an active area of investigation by our working group. Let me explain, we have assembled a group of both public and private expert on this whole question of this million strong cohort. They have met once last week, they will meet again to specifically to talk about what should be the constitution of this cohort. Should children be included . What should we do about individuals that may not otherwise be asked to participate . We want to be sure this covers diversity of our population as well. Some of the cohorts already out there may not be as diverse as what we need. Well figure this out. Theres a desire, however, senator tofr this be something that represents the broad swath of our country. There will be a strong motivation for many people to include children. Thank you, senator murray. Thank you for being with us. Dr. Collins, weve heard a lot about how Precision Medicine is revolutionizing the practice of medicine, allowing for development of targeted cures for individuals. But im also interested in the Economic Impact of this work. We know that Precision Medicine is not only about treatment but about prevention. We have a lot to learn about how environment, nutrition, impact individual Health Outcomes. How might these discoveries Impact Health care costs . We all degree Health Care Costs need to be brought in contr control. If we had the opportunity to focus more on prevention instead of waiting for illness to strike, we would both improve the health of the nation and save money. The Initiative Aims with this million strong cohort to focus on prevention and find out what works. I mentioned in my Opening Statement something about the fact that our annual physicals which many sign up for probably dont collect the kind of data that ultimately you would like to have that might be a tipoff to something that needs attention. And the opportunity to begin to use many of these new tool as well as these wearable sensors reporting on the environmental exposures and bodys performance under various situations should put us in a much better situation to monitor individual health before any illness strikes. I dont want to overpromise the value that this will result in as far as cutting Health Care Costs and bending that curve we want to see start downward again because its a longer term initiative. I would think over the course of time, this is one of the best opportunities well have to cut our Health Care Costs. Okay, one of the exciting things about Precision Medicine is that its empowering patients and people to participate and be full partners in discovery of new treatments. You mentioned the new mobile and Wearable Health technologies that are out there that allow researchers to collect data on how participants behaviors impact the health outcome. I know all fl your agencies are working hard to find new ways to engage patients on their own health in their own health and sustained participation among people that are involved in research studies. What are the best practices in Patient Engagement to ensure sustained participation throughout these Precision Medicine studies . Another great question. So certainly there are a number of cohorts that have already engaged a lot of patients that were going to be consulting with about what the experience has been and kiser permanenty has a large cohort and mayo clinic has one and geisinger in pennsylvania. All done a lot of work to find out what people are looking for if they are going to participate. I think an important part of what were trying to do with this initiative, not to think of the individuals that take part as patients, they are really partners. They are participants, we want them at the table. Well have a workshop july 1st and 2nd, which was focused on trying to get input from individuals about what they are looking for. What we can already say, people expect if they are going to be part of this, their organization is going to help people. Theres a lot of al truism involved and they would like information back about themselves in terms of whats been learned about their own state of health. What has the study led to in terms of broader discoveries that might not have happened otherwise. They want to be included and informed and be at the table. We promise that is the attitude well bring to this. Very good. And let me ask you, dr. Desalvo. Weve seen several high profile sophisticated attacks on Health Care Organizations in the last few months. Americans expect that Health Care Providers and researchers are taking the necessary precautions to protect their data. Thats why i mentioned working with senator alexander on current state of Cyber Security in the health care industry. Can you tell us what steps onc is taking to help researchers keep the large amount of genetic and other Health Information that they are collecting secure . Yes, thank you for the question. We agree its a maj ir issue and something on top of our minds every day. The step we have taken most recently, for example, are to require an Electronic Health records that data is encrypted at rest and in motion. And data is moving across systems and also needs to be secure and encrypted. We are working with, for example, department of Homeland Security with the National Security council and others to ramp up the additional security expectations because again as data begins to be more liquid, theres more opportunity for there to be Security Issues so its a top priority. We have taken actions and we have additional ones on the way. Dr. Colin, what is nih doing . We already initiated a genome data sharing policy in place for several years because we have been conducting studies to try to understand genetic contributions to alzheimers and schizophrenia and Heart Disease. The conditions under which that data can be shared is carefully overseen. You have qualified researchers and we think much is gained by having it accessible but it has to be overseen in a way to make sure the individuals looking at the data are appropriately signing on for various restrictions such as not sharing with third parties and acknowledging where it came from. That has been very successful over several years, we have a pretty good framework there. We think there are things needed in order to protect genetic privacy and make sure it is not acquired by individuals who do not have the right to do so and there ought to be some way to avoid serp tishs without their consent. Senator hatch and then senator franken. We appreciate the work youre doing. Been following it for many years and in utah we have a big data base too and i wonder if that could be part of because we have as i recall, lets see, the data base is the Worlds Largest repository of computerized family histories and linked with more than 22 million public hekt and clinical records. The scientists at the university of utah have used this to identify dozens of genes responsible for diseases. The utah genome project is harnessing the power of utahs large families and discovered new disease causing genes for under lying conditions, diabetes, obesity and cancer. These large families accelerate the pace of genetic discovery by magnifying our ability to identify disease causing genes and harnessing these advantages, it seems to me, gains for using these large families and cohorts, i think our folks in utah can make significant contributions to what youre trying to do here. And i would just like to know if you think they would be useful and if i can play a role in getting the university of utah and you to i think you are already working together but i think you need to work with that group. We are indeed. Senator, thank you for the question. Utah has been in a wonderful place as far as the ability to do remarkable search in genetics over many decades, that i personally benefited from collaborating with over decades of my own research career. And youre right, you have an unprecedented level of depth in terms of family collections. One of the things that we are going to be wrestling with a bit in terms of this cohort is exactly what ought to be the involvement of multigenerational ped degrees. It brings considerable strength to the effort and that will be a topic of discussion later this month in nashville. I would say that the intermountain Health Care System which involves lots of folks in utah, i should have mentioned it on my list a minute ago of those already generated cohorts, they have a very strong presence in this as well. Im quite sure when the dust all settles this Precision Medicine initiative will have a utah connection. I saw that wonderful piece this morning in the news talking about some of this and i think theres a lot of excitement across the Scientific Community in what this might lead to. I appreciate youre volunteering to help us. We do want to help you and university of utah as a Genetics Department good as anybody can have. Weve irritated harvard to death by enticing a number of very top research to utah, they like the mountains and skiing as much as anything but they wouldnt leave once they get there. But we love to be of great assistance to you. I personally would appreciate all three of you and know a little bit about what each of you do and have taken a great interest in what you do over these many years. As im chairman the of finance committee, i havent had as much time to spends in this committee, which i used to chair, but i want to compliment the distinguished chairman and vice chairman for the good work they are doing. They are terrific, terrific leaders in the United States senate. I want to personally express that. I appreciate all three being here and appreciate the work youre doing. Thank you, senator. Thanks, senator. Go ahead. Thanks, senator hatch, almost had to give senator warren equal time there. The order im calling on senators is based on who was here at the time the gavel went down and based on the republican side, cassidy and colins are next and democratic side, franken and bennett and murphy are next. Senator franken. Thank you, mr. Chairman. Thank you to the Ranking Member for holding this hearing Precision Medicine is extremely exciting. Those of us in minnesota thank you, dr. Collins because mayo and the university of minnesota have been doing a genomics project funded by you. Very smart on your part. This is an exciting because theres been a paradigm shift in the way we think of health care in this country and in some part due to the Health Care Reform law and Health Care Providers and insurers are moving more towards person centered care and im talking about coordinated care, medical homes, acos that provide incentives and information that help doctors tailor their practice, their treatments and their therapies to meet the needs of individual patients. And thats of course what Precision Medicine is all about, making sure the right patient gets the right treatment. The right treatment doesnt as you were saying, dr. Collins, doesnt necessarily mean it isnt treating people when they are sick. Its about health care not sick care. And using personalized medicine should improve prevention and so we are doing health care and not sick care. Dr. Desalvo, i do want to talk to you about, what you said, which were at a Tipping Point in the adoption of medical health records. And Electronic Health records. And i just want to talk about that for a second because we had a hearing on that not so long ago. And this there are some barriers to adoption by certain medical providers, some resistance, some because of doctors who feel like i got 20 minutes with this patient and i dont want to spend eight of it inputting data. And but on the other hand, we are this is where were going. So what are you doing to address that and what are some good models . Ive heard of things like having a scribe whos job, like a medical student whos there with you and taking down the information. Where we need to get there. What are we doing to get there . Well, senator, thank you for raising the voice of a lot of doctors in this country. I hear similar things when i travel and talk from my own family members and including my husband, that there are its been a great advancement, Going Forward and folks want to go there, however, the systems are not a part of the work flow in the way that we want or expect in clinical practice. The opportunities there include giving more time for providers to be able to implement the systems on the front line, so the senator may be aware in the last year we put forward some rules with cms to provide additional flexibility in timing of the adoption of records or upgrading to new ones and also to propose in this last set of rules, getting doctors the option of more stream lined approach to the kinds of ways they have to report. Reducing the burden or expectation on the amount of clicks that they must do to show they are functionally using the records. Were working towards a goal of a shared expectation that this is going to be an enabler and really support them and im committed to continue in that path. Your point about successes on the ground and tools that doctors have used in their Office Practice and otherwise is really important. We collect those. Our fellows as an examle and share though. It vary about doc and office whats going to work for them. Sometimes using a dictation system and having assistants transcribe over it and sometimes a scribe. Sometimes working with the Electronic Health record, they had so much time theyve been able to make the systems as seam also as possible for them. A state like minnesota is so far advanced in health i. T. As im sure youre aware and many more years to make sure systems are working. I dont mean to interrupt you, thank you. We can talk about this for a long time. I want to i have some want to get one quick question in dr. Shuren, how the tests that fda about the tests the fda is going to be assessing. One thing that concerns me that some tests may get quite expensive. I want to make sure that i understand how they and the highly Personalized Care that Precision Medicine can provide will benefit everyone. And wont contribute to Health Disparities in our country. So my questions are, will these tests be considered dyiiagnosti or preventive tests and whos going to be paying for them . Well, in the case of next Generation Sequencing, they can be used potentially for both diagnosis and for predicting, in for prevention purposes. It all depends do you have the data to show that particular test can perform in such a way. In terms of reducing costs, theres the opportunity to reduce costs for those technologies to be developed. We have the standards i talked about and databases of information. It will be a lot less expensive to have the sign. In the past you do a clinical study, does your test actually predict or diagnose that disease . When database you might be able to point to the data. Its the Clinical Community is crowd sourcing the evidence. We just did that recently with two years ago about a test Cystic Fibrosis where first we approved that mgs test based upon a subset of variance and secondly, they were able to use data in a database at Johns Hopkins that was supported by the Cystic Fibrosis foundation and didnt have to do a clinical study, dramatically reduced the cost of bringing that test to market. Who pays for it . Hopefully the insurers will pay for it at the end of the day. If you have good technology, its of no value to patients if they dont have access and if they cant afford it, they wont have access. Theres no question that medicine can bring down costs and in certain ways i just dont worry about a brave new world where certain people have access to certain things. I would just im out of time and i would just ask unanimous scent to submit a statement from senator cloeb aschaar to the hearing record. Thank you, senator franken and senator cassidy. Aqo2s  a elgaodw9n report in dod3lcxiiikf must do much kwoc hmw;[m on something that the va is not operable with. Please tell me that im absolutely wrong. Senator, the dods acquisition of a new Electronic Health record, you are correct, is one of the most important things thats going to happen on the i. T. Landscape. We are intimately involved in that. The department of defense for example has seen that were communicating and the department of defense has agreed to lead the way in pointing to the standards. The senator asked me earlier what are the steps we should take. If i could for a second only have three and a half minutes. One of the most important things is to move away from proprietary standards which is getting in the way. Theres an open source bidder, epic is one of them but they are not open source, correct . Those vendors will have to agree to use the standards that the department of defense wants to use which are the ones that onc has published. So we are very pleased that were all moving in a direction to have a core set of standards that everyone will agree with. The va or the local hospital will be able to share records with the dod . Theyre a separate issue because they have a different kind of technology, but yes, sir, the goal is that that not only becomes exchanged but interoperability. I talk to medical students all the time and i dont mean to offend but when you mention the goal, i accept it as a goal but how likely is it to happen because it seems like you left some wiggle room that the va may not communicate with the dod. They have found a solution to Exchange Information so if youre at the bedside with the patient you can see the records from the va and the dod so theyve taken that first step. The integrating of the data requires having the same core Data Elements so theres a Technology Issue which is solvable. Theres also a policy and a culture issue which honestly is generally the harder one in what were facing in circumstances like information blocking which is one of the things getting in the way. You spoke of the open source though. That seems kind of by definition not to include information blocking. So who is blocking info . Information blocking can happen sometimes from technology but what were seeing commonly is that the vendor systems will charge weve had those hearings about how the vendors are blocking so lets take it back where we started. My fear is that the very vendors who are blocking data are the ones bidding, but then you mentioned its open source but you returned to the fact that there could be blocking. So i guess im not clear, will the final 11 billion project be something that i at our lady of the lake in baton rouge can access data, or will there be a problem with vendor blocking . I would need the dod to confirm the answer. However, i would share with you that since we have ascribed blocking and put out the report, the vendors have begun to pull down the fees to make this go away. I do not think the work is done. I apologize. Im almost out of time. I guess, is it part of the initial rfp that they cannot block and that the va has to be able to share as does the Community Hospital . Let me ask it that way. That is the intention of the dod, yes, thats correct. Is it the intention but is it part of the rfp . Yes, thats where theyre going. That is what i understand from what we have recommended as onc to the dod. I would have to defer to. Dod. Can you . I sometimes find the recommendations are not adopted. We can certainly get back with you on that. Im almost out of time. I yield back. Thank you. Thank you, senator cassidy. Senator bennett . Thank you, mr. Chairman and i thank the witnesses for your testimony. Youre fortunate to be on the cutting edge of all this stuff. Dr. Shuren as i know you know, there is a thriving movement of innovation in Molecular Diagnostics under way thanks to the human genome project and investment in the past decade, there are a number of Colorado Companies which are developing remarkable advanced diagnostics in areas like ebola, cardiovascular disease and lung cancer and the fda released a draft work to regulate Lab Developed tests. As i wrote in a letter to you a couple weeks ago, as always, we need to balance both innovation and safety to ensure that we create a fair and workable process. Theres some concern that the draft framework could require the fda to register to improve thousands of labs or a minimum thousands of tests. I wonder if you can speak to this, given the size and scope of the issue, do you intend to propose more formal regulations in this space or are you open to congressional action here . Would that be useful . How do we get a handle on this and create predictability for the people that are doing this work . First let me say that Laboratory Developed tests play an Important Role in our Healthcare System today and our goal here is not shutting down Laboratory Developed tests but in fact making sure that we are both facilitating innovation and that those tests are accurate, reliable and clinically meaningful. We try to strike that balance and i dont know that well actually receive thousands of tests. What weve heard from the Lab Community is that a lot of the tests they make are to address unmet needs. One of the things that we put in our proposal is to say if youre making a true Lab Developed test, the healthcare facility is doing this, and there isnt a test like that that the fda has approved, you dont come in the door. If subsequently someone has that test, they send us the data, and it turns out we know this test works, then our expectation is other people who are making that test do the same because we now have data that that test, in fact, is accurate, reliable and clinically meaningful. We received a lot of comments on the proposal. Were working on it, and we will be making changes before we have the final policy. If there is legislative work that needs to be done around this, i hope youll let the committee know. Im sure that you will. I wanted to return to one other topic around innovation. Dr. Collins mentioned earlier how important mobile technologies have become here, that really in the blink of an eye this is all changing the way doctors Practice Medicine and patients monitor their own wellbeing, and as you know, probably senator hatch and i reintroduced the med tech act yesterday to ensure the lower Risk Software mobile of apps are not regulated by the fda. I think we share the same goals on this, and i want to thank your team for giving us Technical Advice all the way through. Can you talk a little bit about fdas thinking in this area . First let me thank you and senator hatch and your staffs for the opportunity to Work Together on the med tech act. We agree as we looked at this space we were looking at functions, device functions that we had been regulating for a long time and now some of them are being put on mobile platforms. What we found as we looked at it is that some of these lower risk functions we may better serve by no longer actively regulating them and spur a little bit more innovation but theyre sufficiently low risk. We can instead focus on higher risk medical device functions in this space. That is kind of a nice balance on that facilitate innovation but still assure good Patient Safety. Thank you. I dont know, dr. Collins, if you have anything you would want to add. Only that i think your point about mobile Health Technologies is extremely well taken. The proliferation of really exciting opportunities is happening all around us and we certainly see this initiative as a great opportunity to test those out. You not only want to have an application thats kind of cool and gives you interesting information, you want to know does it actually improve health, does it change outcomes. If we have a million individuals who are excited about participating and research who are essentially volunteering to become users of these kinds of technologies, whether its the next version of a watch that measures all kinds of aspects of your bodys physiology or something thats detecting in the air around you what kind of composures youre having, this would be a great opportunity to find what works and what ought to be extrapolated in medical care across the whole country. Thank you, mr. Chairman. Thanks, senator bennett. Senator collins . Thank you very much, mr. Chairman. Dr. Collins, just this morning i met with some advocates were maine who were pushing for more research into brain cancers such as glee yoe blass toem mas. You mentioned that oncology is the clear choice for enhancing the nearterm impact of prediction medicine and that important advances have already been made in this area, so i very much look forward to sharing your testimony with this group of people from maine who are concerned about such devastating brain cancers as glee yoe blass toem mas. Im wondering if you also see a role for Precision Medicine in neurodegenerative diseases like alzheimers, parkinsons, als. Are investments in these important areas also being considered as part of the Precision Medicine initiative . Thank you, senator collins, for that question. Absolutely. We are learning that disorders like parkinsons and alzheimers and other neurodegenerative conditions do, in fact, have multiple contributions, whether they happen or not, to a given individual. For alzheimers disease we know of 35 individual places in the genome where variations place an individual at higher risk and we know one or two where individual variations are protective which is a more potentially actionable finding because you would like to understand that in order to develop the next generation of preventative strategies for people who werent so lucky as to inherit that genetic variation. Especially for any disease that is common enough that youre going to have thousands of individuals in your one million strong cohort, an opportunity to study those at a scale that has not previously been possible and to try to put together all of technology would make that possible and the time is now. Weve reached a remarkable inflection the point and we should not let this moment pass. I could not agree more. Truly its so exciting. Doctor, despite the federal support that you mentioned, i continue to hear from medical providers about the barriers that they face with Electronic Health record implementation. Just yesterday i met with a physician from bangor, maine who shared with me that putting in place a comprehensive electronic Data Collection system for his small practice was going to cause in excess of 230,000. This was just for the software, not for the hardware. Thats no small amount, particularly for a smaller, independent practice that is not hospitalowned. 3. A to access information about individuals to improve diagnosis, treatment, and prevention of diseases, you discuss the Important Role, the absolutely essential role of Health Information technology and interoperability in standards that are going to be needed. As you work to build these systems for Precision Medicine, how can we assure that were not leaving out Rural America, smaller practices, rural hospitals, Health Clinics because of the cost . Senator collins, thank you for the question and for particularly spotlighting the challenges of small practices and rule. As the santa anitaer may be aware, that was a particular focus that we had early on in the joining of the administration but that team wanted to see that Rural America was into the left behind and there was Great Success actually in the adoption of many of those communities across the country in partnership with the usda as an example. Theyre facing a challenge of upgrading technology and its one of the reasons last year because of challenges that they were having that we put out that flexibility rule. The cost that youre describing sounds fairly exorbitant to see if we can understand whats happening there. You are exactly correct. Its critical that we get this. Its critical that nobody is left behind and that we find a way to make it successful for everyone. Thank you very much. Im going to take you up on that offer. Thank you. Thank you, mr. Chairman. Thanks, senator collins. Senator warren. Thank you, mr. Chairman. The president s prediction Medicine Initiative could be a big step forward for more that is targeted effective therapies for any number of conditions. I think its a great idea and we should have started years ago. Dr. Collins, you first advocated for a National Genetic study to examine how peoples genes contribute to diseases over a decade ago, 2004. Is that right . Thats exactly right and it landed with a thud. Thats the article on the screen that i published in 2004. In retrospect, this was probably a bit ahead of its time because we didnt have the technology at the point where this would have been affordable or practical but it is now. Im glad to hear that it is now although if we had started pushing and funding back then, we can only wonder how much further we would be ahead right now. Congress didnt make those investments and in fact, over the past decade nih funding hasnt even kept pace with inflation, and that means we are years behind in doing this work. If we are serious about speeding up biomedical innovation, about improving health, about reducing longterm costs, we start by investing in nih. Now, the house has a proposal called 21st century cures that is supposed to accelerate biomedical innovation. When it was first released by the republicans a few months ago, it didnt include a single dime of new nih funding for congress. Last weeks new bipartisan draft of this bill very much seems to be moving in the right direction. It has 2 billion in new mandatory funding for the nih every year for five years. I applaud the House Republicans for acknowledging what so many of us, including Newt Gingrich and the drug industry, have been saying for years. Nih funding is critical to accelerating cures. But lets be clear. A few billion dollars in temporary funding will not solve a decade of neglect, much less build the future that we need. Dr. Collins, in the late 1990s, congress doubled the budget of nih and then Agency Funding was left to shrink back down. If congress had never doubled the budget of nih and had simply kept pace with prior investments, where would the nih budget be today . Senator, i keep a graph in front of me all the time about this very question and ill put it up on the screen because this is a documentation of the problems that we are now facing. What youre seeing on that screen there, the yellow line is basically what nih has had as far as our purchasing power for research, so its the appropriation, but as adjusted by the effects of inflation. The dotted green line is the trajectory that nih was on going back to 1970, until 1998 when we had that wonderful doubling but then weve been getting undoubled ever since. If you followed the dotted green line and we had stayed on that smoothie trajectory we would be in the neighborhood of a little over 40 billion. Just to get back on track and to reverse the damage of the last decade, nih, if im reading this right, would need more than 12 billion . Just the first year and the house proposal doesnt even put that much in over the space of five years. So let me just ask, based on what youve got here, in your expert judgment, whats the annual rate of increase that nih needs to get back on track on its funding . First let me say we were thrilled also to see whats in the 21st century cure as the 2 billion a year of mandatory gave a great jolt of excitement and some relief to a community thats been really quite stressed over the past 12 years as weve been losing ground. But to get back on a stable trajectory that would result in a healthy Biomedical Research ecosystem which our country has depended on with Great Success over 50 years, i would estimate in my professional judgment we would need to be in the space of inflation plus four or five percent per year. Thats pretty much that dotted line was inflation. That was a healthy way to be sure that all the talent and capabilities of this country in terms of Biomedical Research where we have led the world for decades could be sustained, encouraged, and innovation could go forward in all the ways that i think we want it to. Thank you, dr. Collins. 2 billion a year for five years is certainly better than nothing, but lets not pretend that a small temporary investment that falls billions of dollars short of what were going to need to do the job, there is a gaping hole in our nih budget and we need a serious plan to fix it. There are many ways to make that happen. I have a medical innovation act for example that could add another 6 billion a year, wouldnt cost taxpayers a dime. But whatever we do, this committee has to get serious about medical innovation and that means we have to do better than the house proposal on this. Thank you, mr. Chairman. Thank you, senator warren. Senator whitehouse . Thank you, mr. Chairman. Let me start by echoing the chairmans interest in having a review of where we are on Health Information technology. I read with interest the wall street journal piece by your predecessor, david braylar and i think it makes a lot of sense and provides, i hope, some bipartisan foundation for us to work forward. I think that the Meaningful Use Program has become obsolete and needs to be tuned up to meet the new challenges that the progress over the last years since it was passed now present to us. So thank you for agreeing to work with the chairman and the committee on the four or five key goals that we should be achieving and i would urge you to think big in accomplishing that. Lets not tweedle around the edges. Lets get this right. Dr. Collins, to follow up a little bit on what senator warren was saying, im interested in what youve been able to document by way of consequences for failures to adequately fund our scientific and medical research. It strikes me that you could probably tell me that theres a return on investment from the research that we do and if we dont fund the research, we lose that return on investment. It strikes me also that you probably have examples of human benefits from the Scientific Research which if foregone become human costs, and you probably also pay some attention to the countrys Global Competitiveness in this field. So new could comment specifically in those three areas on what you think is the payback for investment in Scientific Research or if you want to put it con traerl, the cost of not funding Scientific Research. I very much appreciate the opportunity to talk about some of those consequences. Ill just put up another graph which in many ways reveals the difficulties that are present in this country beginning back in 2003. What im showing you there is the opportunity that an investigator who comes to nih with their best ideas has of actually getting funded. Most Biomedical Research done in this country and our nations finest universities and institutes is supported by nih. So this is the main place where this work gets done. From most of our history that has been about one in three success rate. Thats not easy. That means twothirds of the people are sent away. But now its about one in six, and thats very unhealthy. We have looked at what happened in the past when we could fund up to 30 and theres a lot of great science that falls in that space between the 16th percentile and the 30 percentile, about half of what we should be reporting in historical trends is left on the table so we dont know what were missing in that regard. The next great idea about cancer might have been one of those things that didnt quite make the cut. In terms of your question about medical consequences, im a physician. The reason i love being at nih and love what we do is the hope that this is going to change things for the better for Peoples Health and our track record there is striking in terms of whats happened in terms of longevity and prevention of diseases. It is frustrating that we are going more slowly. I promise you the Institute Directors and i when we sit around the table and try to figure out what to do, we still prioritize and still try to push forward but were just going more slowly. We need more advances in cancer. We need a universal influenza vaccine but we could be going faster. In terms of the financial return on investment, thats been documented over and over again. One dollar of nih grant money returns about 2. 20 in the first year to the local economy because of the goods and services that are generated as a result. We support about 400,000 jobs directly across the country in all 50 states on the basis of the grants that we give out. Those are high quality jobs. You asked about Global Competitiveness. We were the unquestioned leader of the world in Biomedical Research until recently. That is no longer to be taken for granted. When you see our losing ground and we see countries like china and india and singapore and south korea upping their investment sometimes in double digits we are losing that. Quick question on that. When they are making those increases in investments, do they have an eye on us as a target to try to beat us, or are they doing this in a general way . Its a little of both. They basically read our playbook from 20 years ago and they saw what it did for americas economy and for the spinning off of Small Businesses that come out of this effort and they want to do what we did. So i dont know if you would say theyre gunning for us, but theyre basically trying to learn from our experience and recreate that in their environment. One statistic that i think particularly renders this a very serious last year, china filed more patents in bioscience than the United States did. That was not even a close competition a couple of years ago and they have now jumped ahead of us. Those patents resulted in intellectual property claims that are going to ultimately spin off new businesses. We have to take that with seriousness. Thank you, chairman. Does that mean china will start respecting patents more . I think i better not comment on that. Dont get the man in trouble. Senator, thank you. Senator baldwin . Thank you, mr. Chairman. I very much appreciate you and Ranking Member holding this hearing, giving us the opportunity to learn more about the administrations Precision Medicine initiative. Were excited about it because its lifesaving potential. Were excited about it because its breakthrough potential. Im excited about it having the honor of representing a state thats been a leader in setting the stage for some of the things were talking about today in Precision Medicine from the isolation of the first embryonic stem cells to the discovery of short tandem repeat polly morphisms at marsh field clinic which is a major discovery that has had i think a big impact on the study of human genetics. The clinic has since developed a very significant genetic bio bank, one of the larger ones with information from over 20,000 Central Wisconsin residents. Dr. Collins, i know youve been asked some it ragss of this question before by my colleagues on the committee, but if you have more to add, i would like to hear more about how youll utilize the existing data like the data that ive just described that was collected by the marsh field clinic and real world clinical data. How will you use those and share those in new ways to create personalized therapies . Thats a great question. Yes, marsh field is a wonderful leader in this enterprise. Ive visited there myself several times and dr. Murray brilliant is the person who is overseeing their large cohort, their Precision Medicine effort, is somebody that i think we are all looking to for his experience to share with us. He was at the white house when the president announced this on january 30th. In this workshop were going to hold at the end of this month at vanderbilt, we will really look hard at the ways in which marsh field, mayo, intermount and kaiser permanente, gisin ger, could be assembled into a synthetic cohort, not doing the work from scratch but making the whole greater than the sum of the parts. This kind of initiative really builds power by numbers and thats one of the reasons were excited about being able to say that word, million, which would not have been in the vocabulary of the people planning these things until recently. We want to take the opportunity to build on the experience. One of the things theyve found is people, looking at their dna sequence, ought to be sick but theyre not. Theres something about them that we need to understand because they have a resistance to disease that we perhaps could learn more about and figure out how to share with other people by development of new therapeutics. Thats one kind of insight that theyve got a start on, but if you had a million people, you could find a lot more. I want to follow up on a discussion you were just asking about the Research Work force. You were talking about funding and reducing percentage of Research Grant applications that are actually funded. Im curious to know what impact this initiative may have on the changing nature of the Research Work force. It strikes me that doctors have happened on discoveries of novel therapies in the course of treating patients, but others may not know that their patients unique reaction to a treatment holds the potential for a breakthrough in this field. What opportunities for new and nontraditional researchers are presented through this initiative . Senator, thats another great question and im glad you brought it up. This prediction Medicine Initiative will not reach its full potential if it doesnt lure and recruit all kinds of people from different disciplines to get together to work on this. I think a parallel with the human genome project that i had the privilege of leading, it was such a historic opportunity that people who never thought of themselves as working in that space decided to make it their passion. I think the same can happen here. We would want to have a computational experts. This is the world of big data in the best way. We would want to have Technology Developers of all sorts who could figure out ways not only to look at your dna sequence but what about all of those met tab lights that are floating around in your system. We could look at hundreds of those in a given situation. All the technologies about mobile health, theres lots of opportunities there as well. And physicians who can begin to figure out how to take this kind of data and implement that in a real word setting in order to improve Health Outcomes. I have to say when i look at the way we Practice Medicine today compared to when i was a resident in medicine in 1979 or 80, its not that different. We have such a long way to go here in terms of really incorporating all the new technologies that are coming along. This is going to be a wonderful laboratory for all kinds of people to get involved and figure out what kind of discoveries can be made and what use can be put to them. So i hope its going to be filled with that kind of innovative talent. Appreciate that. Mr. Chairman, with your indulgen indulgence, one quick question, you can answer this for the record. For dr. Shuren, theres incredible potential in what were talking about, but it also strikes me that theres incredible potential for fraud, for folks, as this develops, offering selling fraudulent interventions that claim to be personalized medicine. I would like again, it can be after this hearing to hear from you more about how the fda will work to prevent fraud and ensure Patient Safety in the age of Precision Medicine. Ill take a quick moment just to if its okay. Sure. Just to say senate or murray and i would like to know the answer too. Thinking beyond fraud, the whole point of having a test out there that really doesnt work and its being sold. This is one of the impetus of our deciding to actively regulate that subset of in Vitro Diagnostics Laboratory Developed tests. There are great tests out there but there are some bad tests out there. Let me give you an example of one thats in the Precision Medicine space and there are several out there. Its called kip six, for people having coronary Heart Disease and their response to staten treatment. There was a med analysis of 19 clinical studies, test doesnt work. Then they performed a randomized p test. The test doesnt work. Over 150,000 people got that test. And that test is Still Available today. That doesnt serve patients well and it doesnt serve Precision Medicine because it undermines our effort to make sure we get accurate and reliable, meaningful tests out there and we get the right treatment to the right patient. Senator, murray, do you have any further remarks . How are consumers supposed to know that . Right now they cant. They dont. You dont have that oversight for some of these tests for knowing, are they, in fact, good tests. Do you not have oversight . We do. Years ago when we set up the program, Laboratory Developed tests tended to be very simple. They were used locally. So in setting up the program we said we would exercise enforcement discretion. Youre subject to our requirements, were not enforcing them. Over time, particularly without being there, these tests z become more complex, theyre being used nationally. Theyre increasingly more important for being relied on for healthcare decisions. As a result of that and seeing some of the bad tests out there is why we moved forward to regulate them. This question has come up since the 1990s. We had nih back then, the department of energy saying the fda needs to get involved. The institute of medicine came out to say that. Two Advisory Committee to the secretary of health and human services. Weve been trying to move forward. In 2007 we put out a guidance to start regulating a subset. We heard from the Lab Community, dont chip away. Put out an overarching framework, make it risk based. We held a Public Meeting and got that input. The framework we proposed in late last year was our attempt to do that, try to balance innovation with Patient Safety and phase it in over a period of time. Right now were addressing comments. Were still, wind working with community on that policy. Youre engaging providers and patient groups as you work through that . Yes. The American Cancer Society has said they have seen tests where theyre incorrect. Patients are getting diagnosed with cancer when they dont have it and people who have cancer are getting told they dont have cancer. Theyve said, too, we need better oversight here. We need to make sure we have accurate tests and the tests do what they claim to do. Important question. I just have one more question. Dr. Collins, why is one million the right number . Well, thats a great question. Theres nothing magic about one million except its a nice round number that we could aim for. I actually will admit that i would love if we could go beyond that. As i said a minute ago with a conversation with senator baldwin, this is all about numbers. Thats where you get the power of the analysis to find out what works. A million is very ambitious. It seems like a goal we could set for ourselves to try to achieve. Given the fact that we already know there are cohorts out there which collectively have enrolled more than a million people, if we can figure out how to do this, maybe well do better. Just listening to the committee members, i think every one of our states apparently has we have fred hutch in my state that is developing a database. I dont know how youre going to work through all of this to get your cohort, but again, diversity, making sure that we represent everybody is really important. Im going to look forward to hearing how you do that. Totally agree with you on that. If we just tack together the existing cohorts, i dont think we would have the kind of representation that we need of the country. Thank you. Dr. Shuren, on your following up from senator baldwin and senator murray, this was the high risk low risk difference you were talking about in these laboratory tests. Youre focusing your attention on the high risk areas, is that right . What we were talking about earlier was on mobile technologies. But here, too, we try to put a focus on when we implement this to focus first on the higher risk tests as we phase that in. As you phase it in . As we phase that in. Is any of your enforcement is it all prior approval or is it where you might be acting on a complaint . In other words, to let the marketplace run for a while on the lower risk items and police it in effect. For the lower risk tests we would not enforce requirements on them other than tell us what you are and if there are problems, report it. We wouldnt enforcement requirements on tests for Rare Disorders and again some of these tests for unmet needs as well. This has been very useful. I said to senator murray this is kind of like going back to college. Its actually very interesting and were very privileged to be students in a classroom with such distinguished teachers and witnesses on a subject thats so important and one that the president , the house of representatives and this committee are all committed to working on. Its my hope and senator murray and i will work out exactly how we will do this but it was my hope that we can finish our work on our Innovation Initiative this year and report it to the full senate early next year so it can be acted on. Some schedule like that. Weve got some other things we have to do as well. But the Precision Medicine proposal by the president would be incorporated within that, so it will be a part of all of it. We didnt talk about privacy today. Whos going to figure that out . Very important question. Onc and nih and the white house ostp have been engaged in this. We will have a deep conversation about this july 1st and 2nd with the participant workshop thats coming forward. If youre going to have a million participants or more and you use all their data, youve got to figure out some way to protect that. Yes. And we are deeply serious about doing that in the most high tech, thoughtful, capable way, again with a fair amount of experience to build on, but we have to take that with great seriousness. Evenii think you heard from variety of senators our interest in helping you figure out what the steps are to actually improve the Electronic Medical record system, see some real results, coordinate properly with whatever the Defense Department is doing. Its a lot of work to do there. Were not trying to catch anybody here. Were just trying to fix a problem, and we would like to work with you to do that and to do it soon because it affects many, many physicians, many, many hospitals. As weve heard today in a couple of important ways its absolute absolutely essential to the Precision Medicine initiative. So theres no other ive got a final page im supposed to read. The hearing record will remain open for ten days. Members may submit Additional Information for the record within that time if they would like. I want to thank senator murray again for the way she has conducted helped us do this in a bipartisan way. We learn a lot more that way. The next hearing will be tomorrow on higher education. The committee will stand adjourned