Transcripts For CSPAN3 NIH Officials Testify On FY 2018 Budget Request 20170626

Foreign approval side as weve got an operation in mexico so that deal has to go through that process there. Weve got operations in brazil but theyre not the only ones. So were going through a process at the department of justice right now and, you know, our expectation is, you know, we should be through that process, we should be through, you know, the operational issues that we have to go through in order to be able to close without any license transfers and the foreign approval process and were hopeful, pretty confident that well be able to get through that by the end of the year. Watch it tonight at 8 00 p. M. Eastern on cspan2. Announcer a Senate Appropriation subcommittee heard from the head of the National Institutes of health, dr. Francis collins and other Top Public Health officials on the 2018 budget. This is two hours. Committee will come to order. The appropriations subcommittee on labor, health and human services, education and related agencies will come to order. Before my opening statement, i want to recognize melinda bikini, we had a chance to meet and talk about her cancer fight and the success shes made there when we were out in nih a few days ago and we are glad youre here and i was just told that the youngest of your six children just got their drivers license so good luck with that. Good luck with all of that and we were glad to have a chance to meet with her, glad to have dr. Collins and the other Institute Directors here today, budget of course proposes cuts that i think you can rest assured the committee will find unacceptable. The 7 1 2 billion dollars cut from nih would according to analysts, this funding would cost nearly 90,000 jobs nationwide, result in 15. 3 billion of lost economic activity. In my home state of missouri that equates to a loss of nearly 1,700 jobs and 292 million in economic activity. The cut is one that i think you can rest assured this committee will not take. I certainly fundamentally disagree with the proposed funding reduction, however, this isnt the first president to propose a reduction. President obama proposed a 1 billion cut in his budget last year. We went 3 billion beyond his proposal. Im not sure we could do anything like that this year, but i also mentioned in a hearing earlier this week that when his budget was submitted to the senate, 98 senators voted against it and one senator voted for it. So we have a long history of the congress asserting itself on issues of how to allocate money. I believe this committee will do the same thing this year. In the last two years with significant encouragement from the whole committee and particularly from senator murray, senator alexander, senator durbin and the whole committee. We had nine of our committee out at nih recently. It was the Biggest Senate delegation to ever go to nih. We spent most of the afternoon i hope you dr. Collins and your Institute Directors know how much your work is appreciated. In the last two years weve increased nih funding by a little more than 13 and were going to be listening carefully to your presentations today but before that id like to turn to senator murray for her opening comments. Thank you, very much mr. Chairman. Thank you dr. Collins to you and to awfully your team for being here. We appreciate all that you do to chaming the critical work of nih. Youve been a great partner and appreciate your leadership and i too welcome ms. S bakini. Its good to see you again. And i look forward to having a discussion today about really the devastating impact that President Trumps budget would have on nih. As you all know President Trump has proposed cutting nih by 22 , most of it by arbitrarily capping indirect costs resulting in the lowest funding level for Biomedical Research since 2002. Three months after releasing this proposal we still do not have basic information from the administration about how nih would implement a 7 1 2 billion dollars reduction. Without severe consequences for thousands of Research Facilities and tens of thousands as scientist thats rely on its grants to support their work. Facilities like Fred Hutchinson institute in my home state of washington just to cite one example where scientists have pioneered bone marrow transplants and searching for cures to cancer and hiv vac scenes. They could be forced to dramatically scale back their cures for patients. These cuts are deeply concerning which is why i said repeatedly, i really hope that both parties will once again reject President Trumps Budget Proposal and continue to Work Together as we have to ensure nih is able to carry out its vital work and bootster Economic Growth and competitiveness. Before this discussion can happen i have to note that todays hearing take place in the very midst of Pivotal Moment for our Health Care System as a whole. As we have heard all week our republican colleagues appear to be dead set on jamming their version of trumpcare through the senate in just a matter of days. This is a bill that has been subject to no hearings, no public debate and no expert testimony. Its a bill so secret that apparently until just moments ago, many republicans senators were telling press and constituents they couldnt say what was in the bill. It was a bill so secret even President Trumps Top Health Care advisor, secretary of human and Health Services told us last week that he didnt know what was in it. It has been so seek create and closely guarded that not even the 13 male senators who made up the working group could comment with certainty on what was in that or when it would be brought up or by what procedure. And, in fact, its a bill so secret that even the White House Press secretary couldnt confirm whether anyone in the white house could see the bill which begs the question as i have said this week, what are Republican Leaders so ashamed of . It is becoming very clear now that those who wrote the Trumpcare Bill knew they wouldnt be able to go back home and defend it because based on what we are now learning this bill is going to be the same kind of Trumpcare Bill that went in the house and it will have tremendous impact on patients and families. Higher costs for families especially seniors and people with preexisting conditions, Insurance Companies no longer will be required to cover basic health care, like Maternity Care or mental Health Services and more. Women would lose access to their doctors and to the care they need at planned parenthood and tens of millions of people across the country would see their medicaid coverage taken away. That means people nationwide who are finally getting treatment for Substance Use disorders like opioid addiction or Mental Health care or access to a primary care doctor under medicaid are going to lose that access. So as many of my democratic colleagues have said, this is not a health care bill. Its an attack on Families Health and financial security. Again i just want to reiterate my message to Republican Leadership, it is not too late to dump this Trumpcare Bill. Its not too late to make the right choices and work with us as democrats to fix our Health Care System. As the chairman and others on this committee should know by now, democrats stand ready. As we always have to Work Together to actually make health care more affordable and accessible for patients and families across the country. But we cannot begin that conversation until Republican Leadership reverses course. And lastly, i do have to say that after hearing this week about so many of my republican colleagues feeling frustrated about this bills process who are angered about being shut out are receiving conflicting information or who may be even shocked at the text that was just released, you have the power to do something about it, not just to complain. You can insist on full hearings, open debate, increased transparency, because lets be very clear, paem cross the country are really worried about the approach thats being taken. Theyre watching. Theyre going to be paying close attention and we need to get this right, not what is being jammed through the senate and with that mr. Chairman, ill turn it back to you. Thank you, senator murray. Were pleased to have the chairman of the full committee and Ranking Member of the full committee with us today. Chairman cochran does not have a statement but i think you do, mr. Leahy. I ask that a statement be printed in the record. Thank you. Thank you, chairman blunt, chairman cochran, glad to be here with you and of course with senator murray and i completely agree with her statement. I think this is an important meeting, dr. Collins. Good to have you here and your whole team. Just for a personal note, when my mother, when she was still with us she was first generation Italian American and when she he saw my schedule she thought you say hello to that nice young man. Or she said bon journo. I am worried about the budget. This affects all of us. You have to support the middle las, you have to lift up the most vulnerable. You have to serve our values and interests as a nation and nih has been a shining example of serving our nation, but sequestration has had devastating consequences for both defense and nondefense programs. I think those consequences are going to last for a generation, theyre going to affect my children and grandchildren and this budget makes it even worse. The budget cuts, those have drawn the most bipartisan opposition and you heard what the senator blunt said and others have about this budgets. The most bipartisan criticism has been the reductions for the National Institutes of health and i have want to commend chairman blunt and Ranking Member murray for their efforts to bolster the nih budget in recent years, but this one, this budget that weve been given by the white house turns that progress upside down, it slashz nih resources by 7. 2 billion, 21 , lowest nih budget since 2002. You know far better than i do, you cant medical research you cant say well just turn off for a few years those studies and trials and all, but well come back five years from now and pick it back up again. You just cant do it. You dont hit pause on studies. You cant have ups and downs based on the whimz of an antiscience group. That hurts the medical search field. I think not only would it do to you, a cap on research around the nation, universities and other entities. Im not being parochial but i mentioned the university of vermont. They received millions of dollars of nih grants each year. They enrolled recently to director mulvaney and noticed its unlikely they could continue their research on this budget and theyve been doing Cutting Edge Research in cancer, among other things. And id ask for full copy of the university of vermonts letter be placed in the record, mr. Chairman. Without objection. Thank you. So i think as vice chairman of the Overall Committee im committed to getting money back in. Science, this is where we can lead the world but not only that, we can make life so much better for our own people and people around the world. So i will fight for every dollar that i can and thank you very much, mr. Chairman. Thank you, senator leahy. Director collins, were leesed your here and look forward to your Opening Statements and when youre through with that well go to questions. Let me introduce the folks at the panel table with me. Over on my left, your right, dr. Richard hoe dus the director of the National Institute on aging, next to him dr. Vol could have on drug abuse, next to me doug lowie whose the acting director of National Cancer institute, joshua gordon, and youve already recognized in one comment at least dr. Tony falcci. And at the far left on your side, gary gibbons who is the director of heart lung and blood institute. We brought the a. Team today so its a pleasure and an honor that we were able to host many of you on our campus earlier this month and its a great honor to be here with you today and im privileged to continue in that role as nih director. I want to thank all of you for your sustained commitment to nih, a commitment that will ensure that our nation remains the Global Leader in Biomedical Research with all that means for human health. Today id like to highlight several areas of exceptional scientific opportunity. Introduce you to a few patients and also talk about some young searchers who are working hard to make these dreams come true and ill show a few images over there on the screen. Lets start with an opportunity that shows the transformational power of investing in nih basic science. Imagine that you could determine the price molecular targeted by pharmaceuticals and see how they interact with each drug. This is starting to happen. Thanks to a new technology called cryoem. This image caused the structure of a promean channel that regulates salt and water balance in the lungs. This very protein is misfolded in sis tick fibrosis our nations most common fatal genetic disease. The genetic mutation was discovered in my own research lab many years ago, but only now is new structural information allowing us to design better drugs to help chris tick fibrosis patient like ma honey. She has not been easy. She required surgery. Shes doing pretty well now. Just a few decades ago she probably wouldnt have made it beyond her teens, no longer. Today we have two targeted drugs for cf and more to come all building on nih research. Were not done. The goal is to turn cf into a 100 curable disease for that we need the next generation of scientific talent, among the early stage investigators tackling this challenge is steven aller of the university of alabama and birmingham trained in both Computer Science and biology, steven plans to transform how we design and deliver drugs for all kinds of conditions. In fact, we need that. Treatments only exist today for about 500 of the 7,000 diseases for which a molecular cause is now known. Among those in desperate need of a breakthru is sickle cell he disease a life threatening disorder in which red blood cells are deformed in a way that clogged small blood vessels. Its caused by a genetic misspelling and that was essentially understood 60 years ago, even today the only way it can be cured is by a bone mar roe transplant from an unaffected donor. That can work really well for some patients like chris sweet shown here with his family. Chris received a transplant at the Nih Clinical Center six years ago and is now essentially cured. Most dont have a well matched don donor. What if we could correct that sickle misspelling in the patients own blood cell . A few years ago i wouldve said thats pretty unlikely but thats all changing. Nih is seeking to use a new gene editing system called chris per to modify bone mar roe stem cell. The goal is to fix the underlying genetic defect and make the persons own cells healthy. What might that do for thousands of others still awaiting a cure . Now imagine you could thats what our Brain Initiatives aims to do. I know that each member of this subcommittee is aware of the enormous toll that Brain Disorders are taking upon our nations. Alzheimers disease alone has an Economic Cost of 259 billion this year and is projected to exceed 1. 1 trillion by 2050. Lets imagine that using tools and technology created by the Brain Initiative you could easily identify someone at risk for alzheimers and use such advanced warning to apply effective ways of preventing this disease thats touched so many, including singer Glenn Campbell who was hill on the with his family to advocate for alzheimers disease research. Young investigator rose of Massachusetts General Hospital is among those taken on this challenge. Her work is focused on the largest known group of people in the world with enher itsed alzheimers an extended Family Living in the mountains of columbia. In the effort to capture subtle changes in the brain, her team is using pet scans of the brain of family members with those who do not clearly fighting alzheimers wont be easy but other young researchers have the talent and drive to make it happen. And you all are part of this. Your emphasis on alzheimers and related Dementia Research in fiscal 2016 and 2017 is enabling progress toward our mutual goal of preventing and effectively treating these devastating conditions. All of us here are motivated today by a sense of urgency to help patients in need of breakthrus. The next generation of innovative and passionate young researchers will be the critical part of achieving that bright future. Two weeks ago nih announced the next initiative a focused approach to bolster support to early and midcareer investigators like the three you see here. Our Nations Health and wellbeing depend on your strong support for them. So thank you, mr. Chairman, my leagues and i welcome your questions. Were glad youre here. I was pleased with the president s decision to continue your leadership at nih and many of us that advocated for that and were pleased to see it happen. Your team and what your team does is inspiring to all of us. Well have a five minute round of questions. I think there are a lot of competing things going on today so well try to keep that as close to five minutes as we can starting with me. A couple of areas i want to ask specifically about in the president s proposal, one was to eliminate the foe gerty International Center, i think particularly with the zika process were going through and have gone through that center was pretty actively involved and your thoughts may be about what the center does as opposed to commenting on necessarily the president s view of this. Appreciate the question. Ill ask dr. Falcci to Say Something about this. The foe gerty International Center is truly integral to all that we do, both directly and indirectly, internationally and domestically because particularly in the arena of Infectious Diseases as we know and have testified before this committee so often, that Infectious Diseases know no borders. Some examples of how the training of International Scientific colleagues has benefited the United States of america and the two that is in most recent memory are the Ebola Outbreak and zika outbreak not to mention the longstanding commitment for hiv and aids that are being done in africa, almost all of whom these trials are led by International Scientist who were trained by foe gerty grants. With regard to ebola, you might be recall that in west africa in liberia and sierra leone they had 28,000 case and 11,000 deaths. If that had gone beyond those borders to places like mali or nigeria it could have been a greater catastrophe and when affected individuals went to mali and went to nigeria it didnt make much press because it was a success, but the investigators in those countries were able to handle, identify, isolate, Contact Trace and stop it in those countries before it started particularly in nigeria which is the most populus country in africa. Each of those individuals were people who were trained at the foe gerty International Center. We look upon them as our true colleagues. I referred to them and i mean that sin seller as our brothers and sisters in the battle of infectious disease. If this were a met for of a military thing they would be our line of scrimmagest allies. They wear maybe a different uniform but they are our allies. The impact of the training has been extraordinary and we really need to continue. Another area, the establishment of the National Institute for research on safety and equality, the agency for Health Care Research and quality would be consolidated into a new i believe they propose a new institute in your organization, one if you want to comment on that generally and two, if that was going to happen are there alternative ways for those things to be moved into nih without creating a new institute . So the agency for health Research Quality does Complimentary Research to nih particularly focused on Health Safety and Health Quality looking how to prevent Health Problems such as hospital readmissions, such as infections from cage terz. We have strong interest in the work they do and have close correction connections with them. We look to make sure were being complementary and not dupe ca live. We would figure out how it is that we could make the best of that circumstance in order to keep that Important Research going forward. Your other question is would there be other models to achieve this other than a new institute . Certainly, we could consider other models such as having the portfolio thats in nhrq distributed around who do Similar Research and incorporating the staff thats part of ahrq into the nih staff without having them to set up a separate entity. That would be another option. I might point out in my last 30 seconds here to something i shouldve mentioned earlier and follows up on senator murrays view. What weve done at nih in the last two years there was no new money to do that so it was truly a prioritization that eliminated programs and consolidated programs and these were programs we had to determine werent doing everything wed hope they would do so that priority could be placed on what youre doing and this is one of those times when Congress Really has chosen to make decisions that for a lot of members were really hard and some members were almost impossible to make but the case youre making is an important case for us to be able to understand and talk about. Senator murray. Thank you, again dr. Collins to you and all your team. As you have been hearing there is tremendous concern among the Research Community about President Trumps proposal to cap indirect costs. What can you tell us about the status of efforts to release the existing longstanding practice for calculating indirect cost with a 10 flat fee . Are your staff participating in those discussions . So indirect costs may be say somewhat unfortunate term because its hard to know whats being referred to and another alternative is f a. Those indirect costs are about 28 of the grant awards and the extra community but it varies from institution to institution. It covers such things as the facilities, the building, the United States, the supply of light and water. The operations of various Administrative Services such as human subjects oversight, animal care oversight and so on. Those are not things that can be ascribed to a single grant but theyre necessary for the institution to be able to conduct research. Certainly the proposal to reduce the amount that would go from nih to indirect costs from 28 roughly on the average to 10 has attracted a lot of attention in the community, because universities who do this work see that as potentially quite dangerous for their ability to keep going forward. We are looking at every possible way that we might be able to assist in this conversation by identifying areas in which various administrative burdens that we ask institutions to follow could be reduced because maybe theyre not as necessary as. Were looking at our current regulation, about conflict of interest, animal care. We might be able to identify a way to reduce somewhat the burden. I dont think it would add up to an enormous difference in what were currently asking our grantee institutions to do and theyre the ones that do the work. What would happen to your intermurial Research Program if the costs were capped at 10 . Our program which you visited recently, much it on the nih campus is about 11 of the overall budget. Calculating indirect cost is complicated. Its a purely government facility. When that was recently looked at it was about 30 . It was lar to what we see happening in the extra mural community. There would be no other potential source of funds. It would make it rather hard for me or anyone at this table to imagine what we would do if that was required to drop to 10 given that we have the buildings and the electric, power and so on. It would be pretty devastating . Im having a hard time imagine how we would manage that. This proposal assumes that state or ben factor will come in and step in and cover the lions share. Takes lot to assume that our states would pick up whats long been a federal responsibility but many private institutions dont have access to state resources or large trust funds that could cover the difference. What would likely happen to those research places like fred hutch or or script if theyre forced to result their indirect cost to 10 of their grant. Its difficult to speculate exactly what would happen but institutions such as the Fred Hutchinson Cancer Research center have been central to the advances that we have made in Cancer Research. For example, thomas received the nobel prize for the research that he conducted with his colleagues at the Fred Hutchinson. More recently, paul wynn led the effort to do research on use of immu noe therapy for patients with merkel cell cancer and this has head through a breakthru. Its not as common as melanoma but it is the skin cancer that has the highest mortality rate and recently the fda approved a cancer immune check point inhibiter for treatment of this disease, the first advance in many years at the Scripts Institute for example, theyre performing breakthru research on trying to inhibit a particular gene called mic that is involved in at least 50 of human cancer but we dont yet have interventions that are successful for that. So these are just some examples of the breakthru treatments that are being developed at these institutions. That would have an impact if the direct impact was cut, it would be hard for them to continue that . Yes. Certainly they would have difficulty to continue their rate of progress. Okay. Thank you very much. Chairman cochran. Mr. Chairman, let me join you in congratulating our panel here in the participation youre bringing to this challenge. Dr. Collins, how is the nih working that minorities are represented at Alzheimers Research activities . So we are very interested in making sure that the resources Congress Gives us are utilized in an optimum way for Health Disparity Research Across all the fields represented at people at this table. Im going to ask dr. Hodus to address that. Thank you very much for the question. In fact, in our efforts is attention to disparities both because all americans have a right to the fruits of research but also the best understanding would underlies the processes is going to come by understanding how it affects individuals of different racial, ethnic and social backgrounds. A great deal of discovery has shown that genetic risk factors differ. We have active recruitment effort that are broad. We work through collaboration with some of the existing resources. Some of the populations who have been studied through nhlbi including those in your own state are being harmonyized. Thank you. Thank you, chairman. Senator durbin. Let me first acknowledge dr. Lowie. Thank you for coming to chicago last october and meeting with the directors of illinois designated comprehensive cancer senators. And university of Chicago Medicine Comprehensive Cancer Center and thanks for your years of service as the acting director of the nci i understand youll be stepping aside to allow Ned Sharpless to takeover. Your work personally has helped to develop a vaccine against cervical cancer. It is saved and will continue to save countless lives. Our nation and beyond are forever in debt to you. I hope reports that you are going to continue on at nih are true. I just want to thank you on behalf of this committee and the people who have been benefited by your great work over the years. Thank you dr. Lowie. Thank you, senator durbin. Let me ask you a question, dr. Collins. If i were to ask you whether or not nih research was part of the development of a certain pharmaceutical drug or part of the development of a certain medical device, could you trace the lineage of the research so you could tell me a yes or no if nih was involved . I think usually we could and in about 75 or so the questions of that sort you would ask the answer would be yes, nih played a role, not necessarily a direct proximal role to the ultimate product but discoveries that happened at an earlierpoint leading to ideas about mechanisms, leading to ideas about possible therapeutics, you can connect those dots and those dots almost always involve nih research in some way. Well, let me tell you why i ask you that question. Were all sitting here wearing clothing and suits and such and some where theres a label inside where its made. And we go out to the market and we byproducts and we have and identity of the producer and contents and i think its about time that the nih had a label, that is applied to pharmaceuticals and medical devices where you can connect the dots. I think its time the American People came to hear of the National Institute of health being referred to on a regular basis as part of the sourcing of the great things that are happening. You have so many miraculous stories to tell and i dont know if you did a survey across america how many people would be able to identify what the letters nih stand for, i think its time we do something about it. We pass laws about the labels in my suit and the labels on products and id like to ask the chairman and others to think about joining me in a bipartisan effort to make sure that credit is given where its due so that americans come to appreciate how youre at the heart of basic research that really makes their lives an awful lot better. Im going to work on something and work with you to make sure we do it in the right way. Senator, i really appreciate your emphasizing the importance of this being more widely recognized. It is true if you ask people on the streets do you know what nih is the last survey it was less than 20 . Much higher recognition for nasa than there is for nih and yet obviously a lot of the taxpayers dollars going into what were trying to do here. I would give a quick plug that in august there is a series on discovery called first in human which follows a series of families that have been treated at the Nih Clinical Center which you all visited. It is powerful. It is three subsequent weeks, two hour segments and you will follow and see what happens to these patients and their families as they go through what are clearly circumstances that are quite desperate and thats how people come to us because all other options have been ruled out. That would be a very good way for america to get a sense about what we do and why it matters so much. Thank you. Were in the midst of an opioid heroin crisis. Some places hit harder than others including the home state of my colleague, senator shaheen. It is an incredible crisis generated by the production of 14 billion opioid tablets a year in the United States of america. Enough for every adult, every adult to have a one month prescription to opioids. That production number is approved by the federal government each year. The department of Drug Enforcement administration and then of course it gets into the general population through scripts written by doctors, cdc has warned these doctors dont overdo it except in extreme cases. I know youre looking closely at this but would you comment on the fact that at least at this moment the United States government is come plisity in the overproduction of opioids . Well i think that in the Health Care System we have to recognize our involvement in creating the opioid crisis. It was result of our well intentioned initiative to treat those patients that were suffering from chronic pain, but without the proper education or understanding about the tools that were seeing to actually treat those patients which was predominantly relying on opioid medications, and as a result of that there was an overprescription that were currently leaving that led to diversion, abuse and then transfer into heroin and synthetic opioids. What were doing from the nih perspective is taking a multipronged approach to address it. Recognizing that the needle patients was initial driver, we are aiming to actually create Public Private partnerships and this is for energying the development that are not going to be addictive. Thats one of them. The other one is again incentivizing the pharmaceutical through this Public Private partnerships to the development of alternative relations for the medication that we currently have for treating opioid use disorders as well as to developing alternative targets for the treatment of addiction of opioids and finally, again, to Public Private partnership with industry to develop prevention interventions for overdose as well as to develop even more efficient reversal medications such as narcam, to address the opioid crisis. Thank you. Thank you, senator durbin. Thank you, welcome to you and your colleagues at nih. Its a pleasure to hear from you. I join my colleague in expressing the gratitude of the work that you do. As well as helping us while we have this Health Care Debate going on, its always been my belief if we can devote Additional Resources into finding treatments, its one of the suggestions ive made for a long time we can do a significant benefit to the cost of health care in the United States and as we have a debate going on about how to pay for health care, i hope we never forget the opportunity to look at why Health Care Costs so much in the first place. Who pays the different question . Thats a challenge for lots of folks, if we can reduce the cost to everyone, all will benefit and i hope that we can spend a lot of attention on that including the continued support for nih and its mission. Let me ask a process question i guess to dr. Collins, one of the things that has been brought to my attention is the way that nih spending occurs in grant support and i raise this with secretary price when he was in front of our Committee Last week, but its my understanding that some Research Institutions make and particularly universities make the salaries of their researchers contingent upon receiving an nih grant. And instances in which some researchers receive up to 80 of their salaries as a result of a grant. And the question i raise is, is there thought about trying to focus the nih grant dollars on the research as compared to the salaries of the researchers, such that more money goes into i know you cant have Research Without the scientists, but i also know that universities and others institutions in my view should be supporting those individuals more where grant dollars go to the research, am i missing something, dr. Collins . No, i think youve accurately stated the facts of the situation. Since world war ii, the government through nih and other funding agencies for science has sought to try to encourage institutions to do Scientific Research and is basically then contracted with those universities to provide the full cost of that research being done. And that has included in that calculation then the percentage of time of a faculty member thats going towards a Research Project as an allowable request when the Grant Application is submitted. And many of the times in some places where faculty are essentially doing almost all of their work in research, their percent effort over various grants can climb up to be in the neighborhood of 50, 60 or 80 . On reality its generally much less than that because most researchers have other activities as well. We do not pay for other activities of researchers. It looks as if roughly between 5 and 10 of the nih budget currently goes to pay the salaries for those faculty members who are our grantees who are doing that Research Institutions would obviously be alarmed at the idea that that would no longer be allowable. I would point out that there are caps on the level of salary that we will pay. A salary cap has been applied by the congress in most of the last many years, but in terms of a actual effort to reduce the total percentage that is not previously been tried. I would also say, of course, that we are willing to pay the salaries of the post doctoral fellows, the technicians who are working in projects and some of those would be at 100 level because thats all their doing is doing the research. If you add up all the salary coverage its in the neighborhood of 35 to 40 of our grant extra mural funding but in a way thats our most critical resource. Theyre the ones doing the work. Theyre the ones we have the hopes and dreams of them making the discovery. This has seemed so far consistent. Thank you for your answer. I may explore with you additional thoughts i have on this topic outside the hearing. Let me ask dr. Gordon, i chair the subcommittee that funds the department of veterans affair. Its a new assignment to me and the appropriations process. Whats the relationship one of the things i think in which the v. A. Faces some of its greatest challenges is in the care and treatment for those who served in our military and the consequential effects on their Mental Health. Too many veterans slip through the cracks and its in my view, its in the Mental Health arena. What is it that takes place in research at the department of Veterans Affairs that is different or in addition, how is there coordination between what happens at nih and the v. A. . Thank you for the question, senator. We have at the nimh a long standing relationship of collaboration with our colleagues at the v. A. , many of our investigators work with the v. A. Investigators, many work with us. Specific programs we focused on recent times include programs in suicide prevention, and in ptsd research. Suicide prevention we played an integral part in helping the v. A. Develop research to identify those veterans who are at highest risk of suicide, so that we can help them get into treatment and reduce the suicide rates among veterans. Thats an Ongoing Program implemented throughout the Veterans Administration that will hopefully help save lives in the near future. We also are engaged in Research Programs as i mentioned, particularly around ptsd, in general around the Mental Health of veterans. One resource the Veterans Administration constructed, the million veterans program, an area were engaged trying to help our investigators that we fund to use that data to bet ter health of veterans and really all americans. Thank you, doctor. Thank you. Senator shaheen. Thank you. Dr. Collins, thank you for hosting the members of the committee who came to nih two weeks ago and thank you to the chairman for arranging that. I learned a lot. It was impressive to see your work firsthand at nih and we appreciate everything you all do. Doctor, thank you for coming to New Hampshire and for seeing the challenge that we face with the heroin and opioid epidemic, you joined me at Catholic Medical Center and we heard a compelling presentation how they were dealing with seven patients all with an overdose at one time in the emergency room and what kind of challenges that presented. Certainly, as we look at whats happening in New Hampshire we have got to do more, weve got to provide more resources, more help. And i know you responded to senator durbin about some of the things that youre working on, but is there something that you think holds the most promise that we ought to be focused on, or is this trying to better coordinate everything that were doing . I mean, we need to in order to solve the crisis we need an i want grated approach working with other agencies and multiproblem strategy so the one that i was describing was specifically targeted toward development of medications. But in the meantime, we need to actually act rapidly and one of the big challenges particularly certainly in New Hampshire but not unique to New Hampshire is the lack of treatment programs that can accommodate for patients with oyp yoyd abuse disorders. In the limit of this research one of our priorities has been to take advantage of the fact that they are systems throughout the United States so how do we integrate the Health Care System into being actively involved in the screening, treatment, and followup of patients with opioid disorder. In New Hampshire we were interested in addressing for example involvement of Emergency Departments on treatment, involvement on and many of these models are starting to emerge in the states like New Hampshire and trying to evaluate them so we translate that into other places. This area of i want police departmentation research, new models for treatments of opioid disorders is fundamental. Absolutely. I visited many in New Hampshire. One of the things that were cites how to respond has been the expansion of medicaid and get medicaid dollars and we have many hospital, community Mental Health centers that have been able to hire people that are looking at expanding and expanding treatment because they are able to count on those medicaid dollars that are available through the Affordable Care act. As we look at what happens with health care here thats one of the real challenges i think we face. I want to go, im not sure dr. Collins who to direct this to, one of the real chronic diseases that we are challenged with in this country is diabetes. I have both a personal and a policy interest in type 1 diabetes, and i think there are many people in the country that dont understand there is a difference between type 1 and type ii diabetes, that type 1 has nothing to do with lifestyle, it has nothing to do with what youve eaten, it has nothing to do with where you live, sadly, but it also is increasing at rates that will make it hard in the future to afford treatment to make sure we can address this disease by i think the statistic is by 2050, 1 in 3 americans will have diabetes, either type 1 or type 2. Can you talk about what research is happening at nih to address type 1 diabetes and type 2 . Id be happy to do that. My research lab on the campus is primarily focused on type ii diabetes. Were learning a lot about both type 1 and type 2 in terms of what the risk factors are, using the cools of again owe micks to identify the path ways that seem to be conferring susceptibility. For type 2 coming on later in life, so much of a problem with juvenile obesity pointing us to new ideas about therapeutics, Exciting Program we have is a partnership with industry t accelerating immediate sips which focused on type ii diabetes and brought both sectors together, with a lot of really interesting ideas about new drug targets. For type 1 diabetes were learning more about the genetics. We dont mow the environmental trigger. Not everybody gets the disease but an exciting advance is the development of artificial pancreas that would make it possible for kids and adults with diabetes to have the way of managing their glucose and insulin without the finger sticks and injections which dont work as nicely as one would like. Just last year when i spoke to this panel, i predicted that we might actually find in the next 10 years an artificial pancreas reaching fda approval. Well it happened six months later. I was going to say its going to happen sooner than 10 years. There you go. In november 2016, fda approved the first one of these, its basically a feedback loop that samples glue dose coast and delivers the appropriate insulin without having to do the calculation or stick their finger. We believe this could be even better if it was made not from this kind of machinery but from your own cells. Using the opportunities were learning about with stem cells to take your skin cells and turn them into cells that make insulin that maybe arent doing that any more but could be convinced to do so. Its a very exciting area. It is, and thank you very much for the work thats ongoing. Thank you. I think we may have a few more colleagues come, if we dont, senator cochran and senator mo ran and i may have a second, third and fourth round while you are all here and wed be fortunate to have that. Dock terks in march we had a hearing on funding and cancer, one of our witnesses doctor tim errorline runs the Cancer Center in st. Louis, and you know, he was talking about the importance of Precision Medicine, one of the things he thought might be possible to determine was whether 80 of the women that currently have chemotherapy after Breast Cancer surgery would have to have that, i think there is some active discussion that probably only about two out of ten women benefit from that but how to define that. But on that topic and on generally your immu 0 therapy advances would you talk about individual cancers and individual people and how they fight those cancers, youre working to find ways to define what treatment they need and also ways to figure out how they can better, from their own unique makeup, fight that cancer. Thank you, senator blunt. I had the pleasure of visiting dr. Eberline and his colleagues last year. And they and many other nci designated Cancer Centers at the forefront of this area of Precision Medicine. The overall goal as you point out, is to deliver the right medication for the right patient at the right time. And not to overtreat people because we know that overtreatment can sometimes have serious side effects. Recently, for example, with nci supported research it was reported at the meeting that giving people with Colorectal Cancer less treatment they did better than people with more treatment. An important part of the Precision Medicine initiative which thanks to your committee and congress has been strongly supported, we have been conducting research that is trying to understand better how the molecular abnormalities with Breast Cancer and other cancers can have implications for what treatments should different patients be obtaining as we try progressively to refine those opportunities and those interventions. Doctor, i think there is new research out that would suggest that having the stroke more than doubles the risk of dementia. Could you talk about that a little bit and also in that area, you might further define as people get older they a stroke may not even be apparent but what is cumulative impact. I guess my question is stroke as it relates to dementia. Sure. Well, its important question youre bringing out, senator, in which we now appreciate that there are many forms and pathways to promote dementia. You all are very familiar with alzheimers. But there are also another category often called vascular dementia, that can affect maybe one in five of individuals affected by that debilitating condition. And indeed, that vascular dimension can be insidious in which it may be cumulative effects of vascular disease that leads to cognitive decline. Promoted by risk factors such as high Blood Pressure and other risk factors for not only Heart Disease but that compromises brain health. So, this is an active investigation, it is actually one area where women appear to be particularly predisposed in terms of the cause of dementia, as well as africanamericans. Again, it relates to earlier question by senator cochran as to why in the jackson heart study we were interested in looking at the study of africanamericans, the causes of dementia, may give us a particular insight in that regard. We also in collaboration with our colleagues at the National Institutes of aging and stroke are engaged in the sprint trial, in which were looking at the effect of lowering Blood Pressure in terms of preserving agonytive function. So this is an active area of research. You know, i would think also thats another verification of generally my view and i think the committees view, that while prescribing funding in some areas is frankly helpful to get these funds increased and to make the case as in alzheimers weve gone from i think 400 million to 1. 3 billion, in two years. But one, youre better at knowing where the pathways are than we are. And two, theres more than even chance i believe that youll find something somewhere differently than you are looking for it and theres lots of research that suggests thats the case. Senator moran. Thank you. Doctors, perhaps this is for dr. Hotis. We had a number of senators raise topics about dementia and alzheimers. What is the current state, what is the one of the things i think, i told chairman blunt this and were in agreement, that one of the opportunities this subcommittee has is to highlight to americans ways in which they can change their lifestyle, dietary, intake, whats the status of the research in regard to dementia and alzheimers today that we would want every american to know in regards to their activity and behavior and nutrition and diet. Its an important question. All of us individually and societally want to do all we can to decrease the risk. In fact its important enough that nih commissioned a study on just this to ask what the level of evidence is for the interventions that might have the reduced risk. It was released. Some may have been briefed yesterday about it. The key words were that there are a great deal of encouraging but in most cases inconclusive evidence for what we can do. I can identify three critical areas and the implications what we need to do, one of them was control of hypertension. So there is very Strong Association between hypertension and its control and the risk of alzheimers disease and dementia as dr. Gibbons mentioned. One study, sprint was designed to look at the impact of aggressive control of the Blood Pressure on multiple outcomes, a case as described our institutes working together and using some of the alzheimers funding have s supplemented that. Those studies are on going. Its an area of great promise, trials such as splint may give us evidence. We have to point out the evidence for the importance of controlling Blood Pressure and decreasing stroke and cardiovascular disease is so critical it must be part as we communicate. Another area is that of physical activity and exercise, shown to decrease the risk of death and where again there is suggestive it may play a role in decreasing risk of demeng sha. A number of studies at university of kansas we had a chance to discuss there, that are looking directly and randomized trials to see what forms of physical activity may make a difference in lowering the risk of cognitive decline or dementia. The other area is that of cognitive training and one study supported some years ago showed that cognitive training was able to induce sustained improvements in cognitive function in areas as individuals aged, that kind of study needs to be extended furd to see if it will have impact on dementia. What would whats the example of cognitive training . What is it that suggests one ought to do. There was a specific study that called active, that looked at older americans, men and women, who had normal cognitive function and trained them in either speed, reasoning or memory. Then studied them over years to see the impact of the training. It was an extremely positive outcome. Those individuals were trained in those areas, did have improved function over 5 up to 10 years in that due main. What this recent report stresses, however, is that theres not yet compelling evidence for any of the other activities including computer driven games to identify whether they provide similar outcomes. There were a number of studies. Another thanks to the increased funding for Alzheimers Research able to look to see what kind of intervention as well as others may play a long term role in reducing cog mifftive decline. Thank you in response to the suggestion that nih ought to receive more credit for its work. Certainly want that to occur. One of the ways that americans would relate to nih is here are the things that if you do, the chances of your Health Improving or being maintained, there is a consequence to this behavior and it is a way i think you can tie nih to Everyday Americans and their lives, their worry and their families. Let me tie another topic that i care a lot about, Downs Syndrome. Weve had conversations in the past which Downs Syndrome and alzheimers perhaps theres a connection, whats the status of our research in Downs Syndrome. I dont know who i should look at. Ill look to my right. Whats the status of that research and what do we know about the relationship . Its a very important area of research. Begins with a genetic basis as you know, Downs Syndrome is an extra copy of one chromeosome which happens to be the chromosome on which the gene protein and pep tied lie and reflective of that is that as we have seen an increase in Life Expectancy with Downs Syndrome, over 30 years now, average Life Expectancy not 30 but 50 and 60, that individuals at a relatively early age in high proportion are developing alzheimerslike dementia in terms of symptom and brain function and structure. Both because this population is a special high risk deserving attention and studying this population is likely to form all we know, we have in collaboration with the Child Health Institute and again using some of the funding that of has been provided over the last fiscal years, initiated studies in individuals over range of age with down syndrome to study what is happening in the brain through imaging and biomarkers to better understand and have a basis for intervening in these people a highly vulnerable population as well as learning more about the more general problems around alzheimerstype dementia. Thank you very much. Thank you for speaking in language and terms that are reasonably upsable to me. Thank you. Senator alexander. Thank you, mr. Chairman. Let me thank you for what you do for our country and were all excited about the future of the National Institutes of hope as dr. Collins calls it. Were delighted the president has asked dr. Collins to lead that. And we look forward to your implementation of the 21st century curtis act which we all worked on, voted for and even in effect asked nih what can we put in to make it easier for you to succeed. I want to salute senator blunt, senator murray, senator cochran, senator durbin, senator moran for bipartisan support of increasing Biomedical Research, theres very little thats happened in terms of technological change in our country since world war ii that hasnt had some Government Research as part of it. Were obviously leading the world in Biomedical Research and we want to accelerate it, not slow it down. Dr. Collins, am i correct that its a goal of the Trump Administration to keep more american jobs in the United States . Yes, thats my understanding as well. Yeah. And is it we have about 50 Major Research universities, 17 national laboratories, nowhere in the country has anything like them. Is it true that around those we spend a lot on them. The office of science supports 5. 3 billion in the laboratories. The energy department, 28 billion goes through your agency to universities for research. Is it true that around these universities like stanford, university of oklahoma, university of kansas, tennessee, missouri grow complexes of industries who are attracted by the research and who as a result create jobs around these centers of research . Thats absolutely true. If you look at the geography of where those places have sprung up, it is very much attached oftentimes to a university that is a generator of a lot of ideas and critical mass. Is it also true that china is making an extraordinary investment in new Research Even though they have one fourth our Gross Domestic Product they may exceed us soon in the amount of total funding for research and that as a result a number of the chinese scholars that our graduate schools and universities are being attracted back home . That is also true. Recent paper published pointed out that in 2000 china spent 12 of what the u. S. Does on Biomedical Research. By 2015 it was 75 . But spendable dollars on track to surpass us in the next three or four years. One of the more hairbrained recommendations in the budget that came up to us which is that we lower the amount of indirect costs thats allowable for Research Grants from an average twof 8 to pen . This came up when i was education secretary 25 years ago, produced a huge uproar. Most universities and colleges and universities said to us at the time look, we spend a lot more than the average 28 on research and the net effect of taking that from 28 to 10 or anything like it would be Less Research, and Less Research we reminded ourselves means more jobs moving overseas around the research wherever it is in the world. I just did a quick look, university of tennessee could lose nearly 10 million if that new policy went into place. University of missouri 15 million, kansas 4 million. People think of harvard and stanford when they think of indirect cost. They are rich enough to handle it people say. Tennessee, missouri, kansas might not be. The only way i can think of to make that money up is either higher tuition or Less Research. Less research is not our goal because it means to me more jobs overseas. I understand that you may be asked because of the 27 or 8 billion that goes through your agency to the universities for the kind of research that were trying to increase, not decrease. To report to secretary price and the office of management and budget which is i have great suspicions that this originate there. What the effect of this would be, so may i ask that you, that you put in your report the following. One, if there is to be any change in this Congress Wants to be involved. And im going to get in the middle of that and ill bet i can get a Bipartisan Group up to make sure we are. Two, will you please ask the universities especially the state universities, how much they would lose in funding, how much they contribute to their own administrative facility funding for the money you give them and whether there would be more or Less Research as a result of this policy, include that all in your report. And if its appropriate let us know about that as well. I hope we can nip this idea in the bud. I hope its one of those ideas in the president s budget that is just out there to stir up conversation. But it is a thoroughly awful idea, bad policy, it would not do what i know the president wants to do is create more american jobs, not fewer. More research, not less. This would be less. My time is up but if you want a constructive way to get more money out of nih, look at the National Academy, may i take 60 seconds just to finish up . The National Academy, two reports says more than 40 of a researchers time is spent on administrative tasks. My guess is most of those come from the office of management and budget too. So if they want to reduce some of those administrative tasks, and free up more money for Research Grants, that would be a good area to work on. So maybe i could ask you to be prepared to talk to us about that at some time in the future. I dont want to put you on the spot now, my 5 is up but i wanted to register my strong concern about the indirect cost policy and ask that you be specific in polling universities around the country about how much would you lose, how much do you put into research yourself and would there be more or Less Research as a result of this policy. Thank you. Id be happy to work with you on that. Thank you. Your extra 60 seconds led into my first 60. Thats where i was beginning. Let me say first i appreciate all your hospitality to get a chance to see whats going on. Were all looking forward to quite a bit of what we saw in research and initial levels and in clinicals, getting a chance to see that. Everyone would benefit from things like universal flu vaccines, the zika vaccine that is progressing, the aids vaccine getting closer. Those are remarkable discoveries and will help a tremendous number of people. We very much appreciate the Ongoing Research as it benefits families. Let me tap into what senator alexander was mentioning about about administrative costs. Many areas have been addressed. One is how grants are done and how they are approvaled. I want to talk specifically between coordination. When i talk to grant recipients they tell me i apply at nih and anywhere i think is close and they are just going after money wherever they can get to the do their research. It is up to the entities to determine weve got this one, this is closer to what were going to do. The people is when we deal with Law Enforcement they can tell you the lane that dea has, atf have, they know. And the Research Area thats tougher to do. So what im trying to figure out the grant recipients eager to get the grant money. From our end now do we coordinate that. But that were prioritizing and how do we get more Research Done by reducing the administrative costs. At 40 percent of the cost whats happening with the research dollars. Thats being completed hats not helping us get to greater discovery. What can we do to reduce the burden for the researcher but make sure that were coordinating this and being strategic . Senator, two great questions. In terms of reducing the burden were intensely interested in looking at ways. Senator alexander mentioned this 42 number that came out of the national study. 22 of that may be the point that we really want the investigators to do. Thats writing the grant and makings sure they have great ideas an putting them forward and giving us annual progress reports. But that leafs a lot of other time. We have some levers we can pull. University or some of the overside. We need to Pay Attention to that. Some of those we dont hold, that would require other ways to do those. How do we discover those so we can help in that . I think it would be fine for you all to ask us a summary of what the administrative responsibilities are and where those are decided. We have a pretty good list of those. Terrific. That would be useful and helpful to others. The National Academy pub lushed a report on it that goes through a good deal of that information as well. We could consolidate it. Not to increase your burden but need the help. In terms of the real concern about not having duplication but different funding agencies we do everything we can to try to nip it in the butt if its starting to happen. We have much better schools for whats in the portfolio or the department of defeps or department of energy. Were looking at those with increasing intensity to be able to identify if there are unintended duplications. Sometimes its good to have efforts in different ways. Certainly we have a lot of areas where we specifically coordinate. The Brain Initiative, for instance, is one where nih and national parka and a few others have a big role and were meeting regularly to make sure thats going to way its supposed to. In some areas we have a good swim lane. The usda does agricultural plants and animals. Nih those relevant to human health. We ask every investigator they have to list their other support. If we give them the grant we want to make sure doesnt sound familiar into what they are asking us to do. Well ask them if thats the case. So i think in the most for the most part we dont have a lot of unintended overlaps. These new tools may be an opportunity to do a better job of that. Love to follow it up with you. I understand the grant recipients have to list where they are getting other funding, thats the area i see as the weak suppose. Wruv got this and we have to figure out whos got what lane. We need the Research Done. Thank you. Senator shelby. I was in another committee. Sorry. Dr. Collins, how much money overall in the u. S. By not just nih but all of the governmental agencies, v. A. , has spent on Biomedical Research and how much is spent out of the private sector. Whatever it is its not enough. I know that. So, the nih budget you all have overseen and thank you for your strong support and a very pipe way, this has now led us to 34 billion. The v. A. Part i dont have the number in front of me. Furnished some of that for the record. Happy to do that. The private sector outspends government supported by a factor of 2 or maybe more than 2. 21, 31 in that space. Were talking about an overall span in the neighborhood of 100 billion a year. How much of that is complimenta complimentary, do you record nate sum, the guess vers in the private sector and other government aus there is a lot of overlap there. Its encourage the collaborative, and this project how do we do that . We have a model that is working pretty well, which is called the accelerating medicines partnership. This gets the scientists and the leaders from the Public Sector mostly fund i by nih, and the private sector and we get around the table and say what are the needs neither of us can do ourselves getting treatment to patients. Those need to be in the precompetitive stakes. They dont want competition. Theres a lot of that we have a project on diabetes, on alzheimers, a project on loop us, and we started a new one on parkinsons, part of this effort, and we put money into it from both places. Theres money on the table and skin in the game. These projects are actually i think touching out to be successful and talking about hundreds of millions. How much coordination is it regarding investigating various challenges we have internationally with our International Friends or competitors and so forth . In a private and Public Sector. Well, happy to say what youre looking for is a cure for this. Yes indeed. Happy that science has always been a Pretty International enterprise and most scientists have collaborators all over the world. When it comes to industry interaction europe has an effort call nd innovative metd sins initiative. We worked closely which is now imi 2. What is the complimentary with what they are doing. I served as the heads of Research Organizations which brings together all of the world. About 95 . Able to work better with each other. Dr. Collins, we discussed research i have with you privately and here many times involving Cystic Fibrosis and some auto immune disease such as lupus. I know nih has been involved in research. You made a lot of progress. Could you bring us sup to date on Cystic Fibrosis. Where are we going . Looking for a cure i know. We are and i think were pold enough to say were getting there. The sCystic Fibrosis cause, whe i was at michigan at 1989 but now we are here at this moment in tift. Seeing the development of targeted drugs. The first only treated about 5 of patients who had a particular misspieling in the gene. The wex one 50 feet, you look at whats happening now and example of Academic Research to leading to advances in the private sector. The ceo was bold enough to say were on the footh curing 100 of patients with Cystic Fibrosis. You extended the life of a lot of children. In the last year or so. Thats a milestone. And a hat tip to the foundation who have been incredible supporters from a flilen tlopic perspective. Absolutely. How many children or people, because they become adults more and more, are affected by Cystic Fibrosis. In the u. S. About 30,000. World side and people of Northern European background. Lupus. Its difficult i know. Its difficult. This project i mentioned has a focus on lupus. What they have been doing which is very advanced the look at kidney biopsies with its one of the major consequences is affecting the kidney. And what they are doing is looking at the im,500 sells to see whats going on. Not such a bunch together but one sell at a time. This is brand new. This is single sell biology. They are discovering types of immune cells. They are rather rare, you look one at a time you can find them. Right now i cant tell you but this is another example where industry working with academia doing the project has everybody excited what this could lead to. Is the money spent overall for all, so many things are related to our auto immune system. Does it overlap . You discover things in basic research . I should ask the doctor to come with in as the person because i think the answer is yes but please, tony. There is a major effort coming from put mel inside on this but there are others that are doing the same thing in the conduction of immune tolerance. Auto immune disease is an inappropriate reaction of your own bodies against tissues of your body. Depending, is it rheumatoid arthritis, is it thyroiditis. What were trying to do is go back in the development of an individual and try and get the cells that normally would have been either deleted or suppressed from the time you were developing as a fetus. When you develop from the first part of the fetus you have cells that have the capability of attacking all of your tissues. What you do in a normal development is that those either get suppressed, deleted. They dont recognize or they dont reresponsibility in appropriately. We have this network which is working by a number of mechanisms to determine how you can shut off the inappropriate response without suppressing the appropriate response. Because right now today, the crude approach to auto immunity is to suppress the entire immune system which unfortunately leads to complications like insections and other adverse devents. Exactly. Is kidney failure one of the big things dealing with lupus . There are other things but thats the big one . Its a multisystem disease as you well know and senator, youve shared your wifes trug struggle. She is doing well. Thanks to medical research. Thats wonderful. As you know in fact joints, kidneys, can affect other parts of the body including difficulty with the rain. We need to get on top of this and figure out. What ask the best way. What role does the dna play. Absolutely everything. But it would happen to be true in this case. I asked you the question. Youre the father. So clearly, there are genetics susceptibilities to lupus, again they are not at the present time sufficient for but there must be a trigger that happens on top of that. Lupus is common in africanamerican women. And we dont entirely understand that. Some of that may be genetic but some may be environmental. Likely both. Which is often the case when you have diseases that have a genetic predisposition, thats one of the things we see in all diseases of auto immunity have some sort of either very strong or maybe a little bit more modest relationship to some gentlem genetic predisposition. There is a link. It is certainly a link. Senator shelby took his two fiveminute rounds at once. I thank the chairman. Were getting a chance to ask multiple questions. Might have been asking those same. No, those were good questions. Senator moran. Thank you. Senator shelby, while the chairman realized you did that i didnt even notice. Perhaps my final question is this. I dont think we have the director here for this topic particularly but let me hear from you. I heard from parents, advocates of Childrens Hospital directors the difficulties in improving pediatric care. Treatments and new drugs are often developed with adults rather than children in mind. Establishing pediatric Clinical Trials is a challenge. So, id like to ask these questions. Considering the difficulties facing this Patient Population what work is nih doing onpedic issues, and what standing between nih and Pediatric Research and affect your results in what were looking for. You are correct that the director of the National Institute for child health and development is not at the table. Although it would be great for you to have a chance to meet her. She is recruited from tufts in boston. But every institute at this table has investments inpedic research in the way little kids become adults but they are not the same as miniature adults and need special attention. The focus on pediatrics has been a long tradition of what nih has done. I might ask two of the folks at the table to Say Something about this, maybe dr. Louie aboutpedic cancer and dr. Gourder about autism. Thank you, i would like to highlight three new programs which the the nci is initiating to support pediatric Cancer Research. As you may know, in the last 15year period, there has been a 20 decrease in mortality rates from children with answer. But there are two serious problems. First, some children who get cancer arent helped and second, the long term side effects for children who are treated can be devastating. So, one program that we are initiating is part of the use of the cancer moon shot funds from the 21st century cures bill which is develop new treatments for children with cancer and as dr. Collins mentions, children, childhood capser is not just adult cancer in the small sense but its different. It requires a Different Research effort et cetera. Thats one. Number two, with our regular appropriation that congress has been generous in giving to nci, we are also doing what we are calling the Provocative Questions Initiative to deal with vexing problems in pediatric Cancer Research and three, this is with the Precision Medicine initiative, oncology devilling the pediatric match trial opening in the next few week, building on the acof6 success of the adults it brings the treatment to the patient rather that the treatment to the patient. Second, based on molecular abnormalities of the patient. I realize i should have put into my Quick Response something about the echo program which is a new initiative that we are excited about. It stands for environmental influences on child health outcomes, this is a transient effort to try to collect information on more than 50,000 children in terms of environmental exposure, risk factors, following them over the course of time bringing together cohorts established but not connected to this standardized assays because there are many things we dont understand about the role of the environment, maybe even in prenatal arena, maybe especially as well as Early Childhood how that affects asthma, obesity, Neurological Development and basically how it affects whether the child is healthy or not. We want to not just study illness but health. So echo which is now just about a year on, which is involving a large number of components. A network to be basically able to do Clinical Research is our big investment thats new on the scene to try to answer this kind of ay question. You want to Say Something about autism. Thats okay so. The autism is not just a transnih initiative but its transgovernmental. I chair the inner Agency Coordinating commit that involves colleagues from really across the government from the d. O. D. , department of education, of course from the nih, and our goal is to try to understand and formulate Strategic Plan for research around autism. I would say the three really exciting on going things that were trying to do. First were trying to understand what makes is the difference in terms of interventions and how that translates into long term outcome. Were trying to get a better handle on screening, particularly the younger that we can identify people at high risk for autism, so that we can intervene earlier and also we can learn more about what happens in these early ages, and the third is really recent developments in genetics and environmental influence that suggest things are going on actually in the womb, thats really where things are starting. So were learning we have to understand Early Development so we can understand the risks for autism. And do something about them. Thank you for answering in a timely fashion such that senator shelby still owes me. I would conclude. By saying this. One of the light bulbs that went off as you were describing this new initiative such as echo, one of the things that suggests to me that increasing funding for nih research theres always science brings the new knowledge which then causes tuesday look in a new area and a new initiative which suggests that the resources are growing, the need for resources continue to grow, very few things do we get to put the check in the box and say woe no longer need to look at this but researchers say oh, here is a new way, a new opportunity for us to kind information through research and science that didnt exist so its lots of reasons i advocated for increased funding that never occurred just your success in finding information breeds the need for more resources to find more information. So doctor gordon, befr we go to senator kennedy, 19 places including Washington University that are looking at the adolescent Brain Cognitive Development and wonder if you give us an update on what were seeing in those 19 locations. Id be happy to but thats an initiative that dr. Wolcut initiated. That would be great. Yes, im very much, and thanks, josh. The idea of the abcd study was the concept that now we have the technologies that allow us noninvasively to examine the human brain in its structure and function. This is an uninvasive technology that is widely available across Different Centers in the country. We wanted to we now have the capabilities to don ter how the human brain develops from childhood to add less septembers to adult who had. If you can get the standards like we are do in pediatrics you have a norm that tells you whether a child is growing faster or slower, we should be able to get what are the normal distribution of Human Brain Development . So when a parent comes to the physician with complaining that the child who has problems you can determine the extent to which the brain needs changing. This will allow us to understand the eeffects in brain development. Very relevant as pates are legalizing marijuana. It woyl allow how Mental Illness emerges. Can we detect that early on on the bases of brain changes. Allow us to understand how physical trauma like sports may negatively influence the development of the brain, how alcohol, how tobacco, did influence the iversty. This will be a perspective charn ages 9, 10, until adult who had. And evaluate not just their brain but cog if itive performance, at schools, social immediate works. Any investigator will be able to analyze and extract data. Thank you. Senator kennedy. Thank you, mr. Chairman. And thanks to all of you doctors. I have two brothers in the medical field. I heard them talk for years with respect and admiration about nih so i was especially pleased to be able to attend the meetings that we had at nih a few weeks ago that senator blunt organized. Very, very impressed. Heres my question. Lets suppose as you often do, that nih or one of the institutes, develops a new pharmaceutical drug or a new vaccine. And obviously its developed with your creativity and intelligence and experience but the money comes from the american taxpayer. And you develop it, its successful, it works, at some point that vaccine or that new pharmaceutical drug is turned over to the private sector to develop further and market. When that happens how much in terms of royalty or shared compensati compensation, does nih and more to the point the american taxpayer get . So, the connection between nih research and the development of successful drugs, vaccines and devices is very strong. Can certainly point to nih funded scoffries in the majority of such instances upon which the Research Track as travel it. In some it was for basic science but made a light bulb go on. In other instances nih carries the discoveries further down the line to something that is patentable and then can be licensed out to a company to develop a product. If that is done by one of our grantees, and one of those institutions that we support. 2500 of them out in this country because of the act that institution holds the property and if there are royalties that come forward they return back to that. Pardon me to interrupt but were limited by time. Im talking about when, its not one of our grantee, its nih itself. Who negotiates the deal, do you have a law firm you use . Do you use inhouse counsel . What is the standard royalty, how do you know youre getting a good deal . Im not suggesting youre not. Give me the details about how you do it. If its a discovery made by one of our own we have an office of tech transfer which regularly is looking around to see if people made such discoveries to make sure they claim them appropriately if they have. Then a patent gets filed. If it seems like its legitimate it might ultimately be worth something. Then the office of tech transfer working with the scientists try to figure out who would be considered in licensing that. And an industry that steps forward and says were interested then a negotiation begins. I think our negotiators are pretty good in terms of figuring out in house . Yes. We have our own legal staff in house. We do i will tell you employ consultants, to help us if we have a special area of we have a strong staff. That has resulted roughly each year in the return of about 90 million to the nih from those discoveries that did get licensed and which royalties are flowing back. Again im sorry to interrupt. Im down to 1 06 and i want to keep in my time. Have you ever had you can take extra time. Thank you. Much more disciplined than your colleagues i think. Thank you. Have any of you ever had the development of a pharmaceutical drug or a procedure or a vaccine that you looked at and you said this works, its a lead pipe cinch. Maybe you havent completed all of the different phases that you need to confins the fda but based on your intelligence which is considerable and your experience i know this is it. You have had one of those . Oh, yeah. All the time. Have we ever thought in those instances, ill call them hed pipe cinches, i dont know where that phrase came from, a certainty that might be more senatorial. Youve got a certainty. Have you given thought instead of farming it out to someone, lets do it ourselves . Yes. The trick there, senator, is weather the manufacturing process is something that we want to take on. We dont make pills. Could you sub that out . Instead to have a fa pharmaceutical company. I suppose thats a possibility. Ill ask tony whether he knows of an example where that has been done. The times where we might do that would be if its a rare disease or neglected where there isnt industrytry. We try not to step into the territory where the private sector is most capable. We dont want to mess with their success. If they are not, well tony, can you . We have discussed this, senator. Its an excellent question and something that has you know, crossed our minds multiple time. As it turns out, the scientific and Technical Capability of putting i deal mostly with vaccines, to get a vaccine production capability outside of the already established pharmaceutical industry is almost impossible to do. Its just not out there. And our sperps has been that it just makes sense in the scenario you developed if we develop is very early stages of the vaccine which are actually in the process of doing it right now with zika and we did with it ebola. Where you do the concept development, you do it in an animal predinical, in a phase one trial and as you say youre never sure, but there are some that you kind of have a feeli i going to work. At that stage you have to develop a relationship with the pharmaceutical company. Our experience is when we try to get into the pharmaceutical type of approach, the expertise of on going at the end of the day its much more economic to essentially license it to them. Bet the kinds of royalty that dr. Collins has spoken about. We have considered your model but it doesnt work. Well, i want to thank you, i dont i dont doubt for a moment that anyone of you could go into the private sector and quadruple your income. A and, you know, moneys not everything. I think you live that. I just want to thank you. I was so impressed with the tour and talking to your researchers and your physicians and to the patients who were willing to talk to us and i just want to thank all of you. Thank you, senator. Chairman. Thank you, senator. Senator shelby. It will be your third round. Ill try to be this slide here, basic science, can you put that back up, Cystic Fibrosis drugs are working, shows the outside the cell, inside the cell, you know, on the im afraid i cant you cant do it . But youre very familiar with that. Yes, we showed that in the opening is this the lung . That diagram on the left, what is that . That is actually a photograph at a very high magnification using a technique. Its a protein. That is the protein that is responsible for cf. Its there in the lining cells of the lung. As a channel that moves chloride and salt around. Until a few months ago, we didnt quite know what it looks like and now we do. And this is where youre talking about targeting things rather than perhaps rather than just do the shotgun approach. Exactly. It gives you the opportunity to be much more sophisticated in your drug design than just hoping youre going to hit something that was going to work. Dr. Gibbons, youre involved in the lung, heart, lung and blood. Do you overlap into Cystic Fibrosis and dealing with the lung there . Yes, we do, senator. Would you like to comment on this . I hadnt given you a chance but were certainly, again, dr. Collins clearlys the pioneer in this area, but its also critical that there have been other elements of the research. For example, he alluded to the fact that certain misspellings changed how that protein traffics in the cell. A lot of that involved basic Cell Biology Research of those epthialial cells and where they got stuck and why and that was instrumental in making a target that would change the trajectory or trafficking. Theres always this back and forth between not only understanding the mutation but how it affects the cell, the cell function and, therefore, the organ function and effect on the patient. Moreover, there are other elements of the disease, unfortunately, how much microbial agents invade the lung. The progeneration of the mucus thats so important as a barrier function. And so there are other treatment strategies of mucus in the lungs correct . Yes, sir. How do you prevent it . Exactly. In addition to influntsing how the channel works, there are other strategies that can be taken to help the patient. As you target the cells there, youre talking about, what are you i guess youre trying to suppress the mucus some way or get rid of it, i dont know what you do. Its a great question. You try every tact you can. I mean, one of the drugs thats turned out to be most useful in Cystic Fibrosis but now 15 years ago is actually something called dna switch which makes that muse cuss more able to be removed. Its not so thick and sticky when you have that making it more fluid. Thats one approach. Of course theres infections that happen in Cystic Fibrosis so antibiotics are critical, including those that can be inhaled as an aerosol and go right to where the problem is. I think all of us primarily are looking to the time where you have a drug thats just not treating the symptoms, the consequences, but actually treating the fundamental problem. The underlying cause. Thats exactly right. When i talked about this, i have to point out that the investigator that i highlighted in my conversation briefly about cf was from the university of alabama at birmingham who is one of those folks who is doing this amazing work to look at the structure of the protein at atomic level. I think you went to med school at lsu though. Might be, i dont know. Dr. Gibbons, if you what kind of time line do you see as far as i dont know, its hard to say. Its going to be three years, two years, or what. But so much time youve made so many breakthroughs in Cystic Fibrosis. Now you made a big one you think, right, going to the fundamental thing. And if you can target that, that will change the game, would it not . Oh, absolutely. Change the lives we should say of so many promising young people. Absolutely. It also is perhaps a forerunner for other Rare Diseases that we understand the genetic cause of. Ones that high priority for us is sickle cell disease. So were excited about the emerging new technologies that existed where we can actually modify that misspelling and turn it back into the correct spelling with this Gene Editing Technology thats emerging. And thats really something thats was really more just a glimmer before, but now i think were on the threshold of. So were looking forward to Rapid Advances related to correcting at a fundamental level these genetic disorders. Thank you, both. Thank you, mr. Chairman. Thank you, senator shelby. You can see lots of continued interest in everything youre doing and were glad youre here. Do you have a five comment . I just want to say thank you, mr. Chairman, to you and the members of this committee for your support of what all of my colleagues and i believe is at a remarkable moment in history and i wanted to take the moment, if youll forgive me, to allow me to introduce to you the future senator from michigan, my granddaughter, bailey fraccer, whos sitting back there, so bailey, stand up. [ applause ] wonderful. I dream of a day where she doesnt have to worry about the health of herself or her children or maybe even her grandfather if hes still interested in playing his guitar and riding his motorcycle 20 years from now. I want to say thank you. Thank you for being here. We hope youre still playing your guitar and riding your motorcycle with enthusiasm 20 years from now. Thanks to the directors for joining you today. The record will stay open one week for additional questions. The subcommittee will stand in recess until june 27th at 1 30 p. M. And live now to remarks from senator tom cotton. Hell be speaking about the future of Missile Defense at the center for National Interest here in washington, d. C. And then following his remarks, a panel of experts will discuss the pros and cons of increased Missile Defense efforts and spending. Live coverage here on cspan 3