press club staff, including the journalism institute and broadcast center for organizing today's event. we are adjourned. [applause] [captioning performed by the national captioning institute] [captions copyright national cable satellite corp. 2014] >> a reminder that you missed any of the event you can watch it anytime in our video library at c-span.org. some political news coming out of mississippi today. mark me feel, a mississippi tea party leader and lawyer facing charges in connection with taking photos of senator thad cochran's wife at her nursing home has died of apparent suicide. weeks agon arrested after those photos of the senator's wife were taken and

used in a anti-cochran political video briefly posted online. won the gop week primary runoff against a rival backed by the departure. -- tea party. journalrow's washington we will look at a decisions handed down by the supreme court as they wrap up this term. and on the one-year anniversary of the court's decision on the afense of marriage act, discussion on what has happened nationally since the rulings and whether the issue could return to the court's docket. also, a detroit reporter joins us to talk about the more the 28 million vehicles that have been recalled this year. we will take your calls and tweets as well. that begins tomorrow at 7:00 a.m. eastern on c-span.

author daniel schulman on the koch brothers. lawsuitwas a massive between the group mothers and the shareholders, and this terminates in a board room showdown. they were trying to expand the size of the board, and this would have ended up opposing charles is the chairman, and they would have taken a greater role in the direction of the company. the end result is bill is tossed of the company, -- >> by the brothers? >> by the brothers. there is a dramatic moment in the book where the board has to sit down and decide bills. bill's fate.

>> the head of the national institutes of health on tuesday said a lack of steady federal efforts tohurting your diseases like cancer, hiv, cure diseases like cancer, hiv and aids. >> thank you everyone for coming. welcome to the first of what will be a number of roundtables about our 21st century cures initiative. to accelerate the pace of cures and breakthroughs in the united states.

as part of this bipartisan initiative we are going to spend 6, 8,xt number of months, review ther, to full arc of delivery development and delivery process we need to improve to keep america as the innovation capital of the world. we cannot do it alone. we need to support of and the of you who aree here today. we will hold roundtables around the country and around washington. we will solicit feedback from interested parties around the questions. a lot of too big, no idea is too small. the only way we will accomplish

our goal is to work together to join this conversation. we hope to hear from you during that process. you who are watching, you can e-mail your s@mail.house.gov. today we host the first roundtable. we have dr. francis collins, direct or of the national , thetutes of health director of the fda center for devices and radiological health, the dean of the university of dr. joe med school, gray, associate director for translational research at the night cancer introduce --

institute. chairman of the project, senior fellow of the manhattan institute, the chair and founder of the friends of cancer or ofrch, the direct the deerfield institute. thank you for being with us today. in order to get the most out of everyone i will keep my remarks very short. i would like to ask diana to get effort to saythis a few words, and then we will introduce eric cantor, majority and steny hoyer planned to be here but he had a last-minute conflict. we have the number two public in and the written number two --

number two republican and the number two democrats on board. >> i'm happy to partner with you in this important effort. the 21st century cares initiative is an exciting effort that has great potential to positively impact biomedical research and innovation in this country. united states has been the fields forhis decades, but we are at across word -- at a crossroads. bring leadersl and policy, academia, industry and research to the table to figure out how we can more effectively and efficiently tackle the complex issues of medicine.

discovery, development, and delivery. it is in those areas that we want to focus our attention and seek input of leaders like the distinguished leaders who are in our inaugural panel today. since we launched the effort a week ago we have already seen tremendous interest, and just the attendance today shows the interest we have. the questions we are focusing on will be key to continuing our work toward these complex challenges. we need to take a look at the current state of biomedical research and innovation in the u.s.. what are the drivers, the barriers? where is the u.s. lead heading, and where we concerned that we are falling significantly behind ? how does this translate to improving patient care at outcome and better health and medicine? we know there are pockets of fantastic cross this -- progress across the country.

where can we focus our attention to reach more patients? are there other countries that we can learn from? of u.s. may be in danger falling behind. what strategies and resources are the other countries importing to excel in biomedical research and innovation? are there concrete, viable actions that we can take to help advance via medical research and innovation in the u.s.? does the nih need more tools to better harness the research being funded? help the fdawe modernize the drug approval process to take advantage of the cyclical nature of research and innovation that we mentioned, this government -- discovery, development, delivery? i think everybody in the room and on this panel today is up for the challenge.

i know we can be productive in our conversations today and moving forward. i look forward to the discussions, and i am grateful to everyone for joining us in this effort as the experts were taking their time. thank you very much. >> thank you very much. i want to first acknowledge the fred andp that both diane are devastating in bringing us together in a bipartisan way, focused on what all of you spend most of your professional time about. curing disease. that, for my colleagues, for the commitment that some of you for decades in have allocated resources and decisions the sign that -- and behind that to affect good policy. what i hope will become carmen politics is a culture of cures

-- common parlance will be a culture of cures. we want to make sure there's access to treatment for everyone. hit the nail on the head in the monitor. at making get better that a reality. good treatment is not enough. thank you for the emphasis here, the 21st century cures , i think it will be a tremendous story of accomplishment for this congress. if nothing else, this congress should be known for making the right choices and set in the right priorities. that is to put saving human life cures andmoting treatment for a better life, and ultimately i think it is a

win-win for everybody. on thef the discussion hill has been about fiscal restraint, about how do you deal with limited resources? dr. collins, you and i have had many discussions on this issue. here, we want us to do more. but sometimes first is to start to make the right choices with what we have, and then set about how we are going to do more. i congratulate you on this. i would just ask for my nine know that it is inherent in the nature of the members on the energy and commerce committee to think outside the box. all of us can sit here and talk about freaking down the barriers the way that diana talked about in terms of promoting a culture of cures. spending $10 billion, taking 10

develop a drug agreed to market is unacceptable, not what people are dying. so how do we think outside the box? it is improving fda processes, it is making choices like the first steps that we took with the kids first research at that is demonstrative of tough choices. scheme of things 123 million is not the 30 billion that we have to protect and grow within the nih, but it is a step. we are demonstrating we are willing to make tough choices and set priorities. i really commend the initiative here and look forward to playing and i don't roll -- an active role. , the esteemed panel that is assembled around

the country, we appreciate your work and we honor your presence and look forward to the outcome of results on this mission. >> thank you. we'll keep you in the loop. i'm going to ask the former chairman and ranking member waxman to say a few words. i want to introduce some of the numbers that are here. joe pitts of the health subcommittee, his ranking counterpart on the health counseling -- subcommittee. burgess from mike the health subcommittee. mr. caster from florida who joins us. leonard lance, a member of the

committee as well. my friend, mr. waxman. >> thank you. i think it is a very important for us to figure out ways to develop cures more rapidly. we spend an enormous amount of rani doing basic research from which the pharmaceutical companies take advantage, and are able to produce products that are life-saving. we have to encourage more development of medicines, but we also to make them affordable. we do not do everybody a favor if we have a drug that cannot be bought, or that the health care system cannot pay for. about new products, new drugs, new therapies, we must evaluate them to see if we are adding more to helping the american people and mankind, or whether we are just layering on

new efforts that will cost a lot more but may not add to the therapy that people are so desperate to have. faking outside the box is evident from this kind of meeting. it is interesting, i've never seen anything like it in the time i've been in congress. i will look forward to this group figuring out the recommendations to the committee so that we can consider that legislation if it is necessary. we all want to work with you, and it is worthwhile to hear from people in a setting that is different. i do not know we can even make a transcript of the proceedings. anytime we can exchange ideas and his all for the good. thank you. justogress man waxman is uncomfortable to being to my right. [laughter]

here.very good to be we are going to show our system bipartisan, under the leadership of chairman upton and diane come and with the otheres of mr. waxman and experts. our system really does work, and over the next however many months, the country will see political system and medical system at its best as we work together to decide how to take things out of the laboratory into everyday life. well,f you i know very some of you i have worked , but chairman upton is an honest broker, and

he is absolutely making this a op p rarity -- trop fire priority. i look forward to having the dialogue, and coming up with solutions that make america and the world a better place. thank you. >> thank you. when we send out the invites we sent a couple of questions to me thought. i would like to see where that takes us over the next couple of hours. the state of biomedical innovation in the united states? biomedical innovation mean for american patients and jobs? u.s. compared to other countries with respect to biomedical innovation and how can we make sure that we lead the way in the 21st century? steps does

congress need to take to accelerate the is covering, development, and delivery cycle? and keep more jobs in the u.s.? that is where we want to go. what family is not impacted by something we can do here? why do we start with the fourth question? what steps can congress begin to take? you, i sat down with you over the last couple of weeks and couple months, and hopefully the ideas and element has been working. i want to remind the listening audience, cures@mail.house.gov is the way to submit.

we're really going to start to have a discussion. let's go at it. thank you for being here again. is amazing toit sit here around this roundtable which is really rectangular, and see so many members of congress from both parties represented. i thought to myself that i never seen anything quite like this, but maybe my 20 years were not that informative. when mr. waxman said yet not seen anything like his, i know this is a unique and special moment. for credit to you mr. upton convening us in what will be a series of conversations. to try to answer your question, and not go on too long about it, prospective, what we need to continue the most

successful story in biomedical research is a steady, predictable trajectory of support. we have not seen that over the last 10 years. we have lost more than 20% of our purchasing power come and that has put the system under enormous stress. it has lost jobs because we 138,000 jobs and some of those have been trimmed. most worrisome for the future, tohas caused a great pall fall over the enthusiasm of young investigators who are our future. they need to have the confidence that there is going to be a path for them that their dreams are going to be possible to present -- pursue. they are not lacking in talent, or ideas, this is a unique and are mark will moment in search potential. in a unique situation

where the scientific opportunity is a distinct mismatch with the takety of investigators to risks and go out and do the kind of amazing science that we in america have been famous for and are capable of now. perhaps, an occasion where we are missing out on the kind of return on that investment that might be there. i want to mention that as well because i think your questions reflect an interest in that. analysis of the economic return of the women's health initiative, an effort which was started 20 years ago and which nih but $260 million on. was 40-1ated return and what it taught us about women's health, and how to save lives and reduce cost. in terms of where we are going, the innovation opportunities, we are extremely energized i the

kind of technologies that are haveble and wishing to that unleashed. when you can now do data a chip the on size. kidney placedn into an arrangement of cells, and you can study kidney function in a very reproducible way. things that we are not pursuing at the level we might. we're trying everything we can a try to use this crunch as motivator for creative solutions, working with industry and the exhilarating medicines partner. this brings together the science in the public and private alzheimer's, lupus,

diabetes, and cut down on that 10 year time frame of trying to improve an idea from therapeutic. i did we're pretty bullish about the potential, just going along with what leader cantor said. excited about curing things as you can imagine. we want to cure-all summers -- cure alzheimer's. but the current system is not working. nih was not broken 10 years ago, but 10 years of loss of purchasing power have started to break it. if we have the chance to recognize that this is not that is spending, it is an investment that pays rewards for human health, for the economy, poor everything that the government does well. economistlysis by

say that the most important entrepreneur is the federal government. yet we have not allowed that entrepreneur to be what it could. we could havef the confidence of a stable trajectory for support, that would mean the world to an enterprise that is currently flagging. just as a point of the role consequence of this, i do not know if you saw usa today in the last couple of days. describing a typical situation of a related young scientist -- of a brilliant young scientist who is at the top of his field, and who should in every other era would be fought over by tennis fusions in the u.s. to find this next creative faculty position. he is going to china where he will find himself surrounded by incredible resources as they continue to increase their

support for medical research by 20% a year while we have been decreasing hours deadly over the last five years. we could increase this. it will take all of you and the recognition that this needs to not be an afterthought but a high priority for our nation. losingat risk of something that has been one of our greatest stories. thank you. asi have the privilege serving at the dean of the university of michigan medical school. and actually being involved in all aspects from training, future scientist and physicians, to discovery and application inpatient care. seeing the whole ecosystem.

to theancis mentioned as isst that is us build -- instilled by lack of funding levels for nih is absolutely true. however, there are other things that we could do to be very helpful. that havetory burdens been overlaid on our faculty and staff of the last several years our ever-growing. these are unfunded mandates. basic things like depending upon what institute you're dealing with, what branch of government you are dealing with. there are different conflict of interest regulations. keeping up with that, and being compliant is an administrative word and that has no value added. asking congress to come up with

a uniform approach would be very helpful. similarly, as we look to move our discoveries into thatrcialization, we find there are gaps in the echo system. -- ecosystem. discoverearchers can mechanisms. we can come up with targets, we can actually develop medicines. we can develop devices. that requires a large number of other individuals, partners with industry, partners with government in order to move that forward because we do not have all that expertise in the university. unfortunately, those partnerships are sometimes difficult to develop because of the regulatory burden. -- and a very basic level it would be helpful

that the fda and there would be a checklist. lot of operation from members of the fda that are really trying to help them do their job. at the initial stages, what we had to develop our navigators because the complexities of interacting with these agencies are such that our faculty have to be educated. potentially there are ways to really partner with government in a different way to facilitate that interaction. i think it would be very helpful if congress could help move governmental agencies/a uniform conflict of interest, there are ways that are more systematic and more transparent.

>> i am the head of the center for the drugs that fda. i want to talk about pharmaceuticals in if you look at the ecosystem there are some barriers that academics and developers face when they try to go from a discovery that they make in a laboratory to an actual pride given toproject patients. in order of perhaps ability to deal with this, the clinical trial system we have is not a system. what is done right now is for every product, and they get a clinical trial, and that takes about a year to get together. then you do the trial and then it is shut down, and then if successful they will start another trial and have to do 10 different agreements with 10

different universities, and it takes years. it is exhausting investigators, and much paperwork. do, we are starting to are starting to look a clinical trial networks. to turn the paradigm on its head, you have a network that is funded, and when you get inventions, they contest the invention right away, and you have multiple rugs or drug -- drugs or drug devices. it is much faster, more independent, if i may say so, because the product, whatever it is, from an academic or company, is given to a network, they evaluate it. there is some distance therebetween the evaluators and the inventors, so to speak, but basically it saves a lot of money. comparisons in the network because you are testing

multiple products, as well as approved therapies. that is something that could be advanced, to set up more networks. likes that have done that, cystic fibrosis, that is why they have succeeded in getting products for theiat disease on the market. they have the patients, ready, everybody's ready to go. another ain't you may not be interested in, and it has to do with jobs, is drug manufacturing, a lot of innovation in drug manufacturing. we are having a meeting in a few weeks about this. drug shortagesve that are afflicting the hospitals across this country. we buy many of our drugs from other countries, and if something would happen, we would not be able to get those drugs anymore. hostilities, natural

disasters, and that is another thing that is saved by developers. they have to continue to scale up their manufacturing, and it is the very clinical trial system, outdated and cumbersome. there is now the technologies available to do these continuous manufacturing and make things in situ, they would be made in the united states, and it is the wave of the future where they would be needed. that is another area. under a critical path initialing, and he knows a lot, there is a whole lot of translational research that needs to be done on biomarkers and many other things that would really aid in getting products from that person in the laboratory through and into the clinic, to the clinical development, and that is another thing that really should be worked on as a tremendous

opportunity. chairman, i want to thank you for your leadership and the bringing usn together and tell you what a pleasure it is to be back in this hearing room again. happened a couple of words that have been placed before you, such as ecosystem, and francis diluted to risk -- alluded to risk. it is important to keep in mind that this process of development and delivery is a cyclical one, and it lends itself to tremendous opportunities with regard to acceleration of that process if we pay attention to the issues of what does accelerate it, namely, the investment of intellectual capital as well as financial capital .

we are seeing risks as it relates to the rewards, namely, reimbursements and the challenges coming from that. takes a holistic view of this ecosystem and looks at policy changes, one of the themes will be look at those initiatives that will reduce the risk within this system, and that will promote and enable a greater participation and investment in accelerating the cycle. one of this particular things that needs to be addressed is the transfer of data across this cycle, and the opportunity for greater data sharing and greater data integration. there are data challenges on the front end. as we see the transfer of data from investigators to developers, and the challenges

of even date at sharing among developers is they now need to products,egratived and there are problems on the other end, the need for looking at our clinical trial designs and the way we are dealing with data as it relates to the delivery of these inventions. so you have put in place an enormously important process that will enable us over the next months to step back and take a careful look at all of the components of this ecosystem that need attention and reduce the risk that is currently growing and slowing down the process. thank you so much for inviting us to be part of this. it is an honor and a privilege. i came out of graduates cool and work for the office of technology assessment, and

although mr. waxman, you described this as unusual, it was the normal or a long time, to come together and talk about these critical issues. in terms of answering the question of what can congress do, i think you are doing part of the work, which is putting a spotlight on research, science, and innovation as a national treasure and as a national prior a tv. so if you are prioritizing the research system, i would follow suit with what dr. collins articulated in terms of the resource issue. i do not think you can escape the responsibilities of congress to make sure that these agencies are adequately funded. united for medical research is a group that has come together around the issue of the nih, and the alliance for a stronger fda is a group that i have had the privilege of serving as a past president. these are groups that have a stake in saying that the dollars

that come in are not enough. i think that prioritizing the infrastructure has to happen. it is a critical necessity, and i think if we are not listening -- that they'll being w bell being rung, we run the risk of really losing an enterprise that the united states essentially pioneered. in terms of another priority but i think you are starting to hear about, we had to put patient at the center of this. if you look at statistics, we have 7000 visas, yet -- 7000 diseases, yet we have treatment for 500 of those. the system is doing its work come it is turning through the science, it is getting the outcomes to the doors of the fda, the fda is doing it's work, and then we get approvals and put it out. at the rate we are going i do not think any diseases we are

going to be afflicted with at some point in time really stand a chance of being covered in the near term. i think the speed issue that has been articulated by many of you is of critical importance. as this group does its work, figuring out what are those points to de-risk the system, that becomes a place that i think it is going to be of critical importance for all of these stakeholders you what areto figure out the pressure points. yes even the world at fastercures, we have had the privilege and opportunity to work with a variety of different disease foundations, so the fibrosis foundation, the melanoma alliance, a multitude of these group's. what the system can learn from these groups and what we have

studied is that they are one-stop shopping in terms of understanding what signs do we know, what do we not know, what do we need to be getting prepared for, and how do you bring the patient into the center of all that. so a couple of points that i wanted to make sure that i talked about at that top line is the sacredness of this innovation ecosystem and the real need for that stability to come, and in the putting the patient at the center of it. i want to make sure that as you do your work, we are making sure that happens. thank you. first of all, to thank you for convening -- can you hear me? i want to thank you for convening this important committee, and i want to thank congress for their work. actually, recently, through the reauthorization of the act, we had -- [indiscernible]

i'm trying hard. we had a mechanism that was sponsored by friends of cancer research. it was bipartisan. it was sponsored on the senate side, on the house side, had support of the industry, patients, companies, and the mechanism to get patients drugs faster, but better. where there is evidence and unmet need, this was an extraordinary vehicle, and i know it was sponsored on the house side and it was sponsored by this senators bennett and hatch on the senate side. that is an example of having to come together in a bipartisan way was not just aspirational issues, but really strong evidence can get rugs to patients that work that are better for them. ,here are other opportunities and it is a little bit like a

dickens novel. it is the best of times in the worst of times. we have been extraordinary -- we have an extraordinary biomedical infrastructure, the site has never been better, but it is endangered. we have foundations at the nih and fda that can be empowered and should be and are doing a lot that can be doing more. we are convening now with the public the nih in a -private partnership in lung cancer. over the united states. it is for a deadly disease. it is for long clients are -- lung cancer where their internal tours. it is a partnership between the being convened at

the nih. so there are novel ways we can get there and things we can do, that we cannot take what we have for granted. if we do not get the science there to the patient's, we will .ot benefit - and mostly patients will not benefit. >> thank you for the opportunity to be here today. one of the big inefficiencies and barriers we face, which is acute on the medical device side, is the ability to make optimal use of a that that is collected as part of the real world typical practice. this is the impact that it is having. in a medical device program at the fda, we have been looking at how we can reduce clinical trial burden on medical device innovators and what they need to bring their product to market. by shifting that data collection to the post-market setting. and what we would like to do is reduce that burden on the

post-market side by being able to better rely on data collected by doctors in their clinical practice. two weeks ago we put out two guidances. one proposed a new pathway for high-risk devices for serious conditions that address an unmet medical need. we don't often experiences from the drug program on accelerator prover and -- approval and added other features, which allow moving data into post-market. here is the challenge -- if you look at how we collect data today, in practice a lot of it goes on electronic or otherwise medical records, but it is hard to identify which device was used. either it is not in there, or it is hard to locate. one of the solutions that you directed us to implement is to create a unique device identifier. it is a number that says who was the manufacturer, what is a product, what is the version of the device, because the versions are constantly changing.

and then put that on the labeling of the product. in the next few months, for the first time, devices will start having those numbers, because we issued a rule last year. here's the next that -- it has got to get into electronic health records. what are the incentives that can be in place to build the fields in electronic health records so we can capture that information ever doctors and hospitals to then put it into electronic health records? then we can actually make use of it and you some of that big a that. the other part is sometimes more data than what is actually gathered in practice, but doctors would get it when they are seeing patients. there are challenges for setting them up, so exploring what are those barriers and what are the incentives that can be in place for creating those record -- registries, because we are talking about user data collection and even the case where the manufacturer does not need to get the data, so we recently allowed for expansion of a labeling in the device

where the company and two health care professional societies were doing a clinical trial, and we told them do not do it, do not waste your time. we looked at the registry data and we think it is good enough, ask us to expand labeling, we will do it. you do not need to do it. we can have those kinds of investments in this country, and that kind of infrastructure to allow greater use of it that we collect every day. we think that can have a big impact in reducing burden for products to get to market. i think create critical incentives for more information in medical devices. >> i would like to echo that -- ofent that the color the other panelists. they give a great opportunity. >> hold and a little closer, for the folks listening on -- sarah despres. i would like to follow-up, we have heard from the other

panelists, that clinical trials can be a barrier to development as the leader said, it could be on$1 billion. congress do, and there are opportunities to look at how new tools can be used for clinical trials. -- clinical trial networks, and we know that clinical trials are a barrier to antibiotic development. on the registry side we have a project looking at devices and we have spent a lot of time looking at the promise of registries, and it is interesting that registries have the promise of being able to safety, andct, effectiveness, how health care is delivered. there is a lot of really

interesting ways you could go, and we have registries now doing some good work, but we are not as advanced in this regard as they are in other countries. for example, there was an article in the new england last year about a trial in sweden using an existing clinical trial registry -- i mean, and existing clinical registry, the trial of cardiac intervention. 10,000dy enrolled patients, send them thousand were randomized, and the total cost was $900,000, 50 ask a 7000.t for the you cannot do that in the united states, and you could do about that money.s for there are opportunities with clinical registries to actually help with product development, and i encourage you to look at that as you are thinking about doing forward. you are going to hear a lot

about the challenges of trials and answer is not to get rid of the trial, we needed at the end of the day. the regulators need to data in order to assist product. the clinicians need the data to know if the product system and the patients needed data to know if it is the right product for them. the question is how to gather that data more efficiently. a key. -- thank you. >> thank you, and i want to thank this committee for taking on this extremely important issue in this way. i testified to front of this committee three years ago and talked in my capacity as a venture capital investor about the trajectory of venture investment in innovative life-sciences companies come and i presented data showing that that trajectory was in an alarming state of decline is one indicator of the state of investment in biomedical innovation in this country. and i attribute that phenomenon

at the time to a relentless increase in the time and cost of developing new drugs and medical products, not just over the last two years, but over the course of several decades to the point where we now all talk about takesstatistics that it up to 15 years and cost s $1 billion to develop drugs. it is three years later and looking at the same metrics, in terms of venture capital investment in new and innovative biotechnology companies, the picture is strikingly different. -- it appearster much better than it did just a few years ago, and really over ae past year he has seen resurgence in interest and a resurgence in the dollars being invested in this industry from a venture capital point of view. so maybe to answer the question of what we can do to build on that and further improve, part

of the answer is to think about how is it that we have gotten better or at least on this one measure of the last two years? i would point to two factors that have driven the increase in venture investment in the sector. science,e breakthrough the kinds of treatments, the kind of chores that we are now investing in and developing and ringing to patients are transformative in areas like cancer, genetic diseases, and many others, that we are seeing an impact on disease that really was -- is remarkable and would have been hard to predict a few years ago. that is a function of the caves of investment in basic research and in fundamental understanding of elegy and the mechanisms of disease over the course of decades. that is number one, breakthrough science. number two is a dramatically improved drug development environment and regulatory environment by which i really all a renewed focus by

parts of this community led by fda, but also patient groups, industry, the medical community, to work together to address this problem, recognizing nobody is happy when it takes up to 15 years to develop drugs to patients are waiting too long, and it's striving investment away. when you look at these to highlight the point about the breakthrough therapies designation, that is emblematic of a number of initiatives that has led to a different kind of dialogue about how we develop drugs and how we regulate drugs and how we need to think about while always insuring that we get good products that are safe and effective to patients, that we do so as quickly as possible and efficiently as possible, especially for those drugs that make a dramatic impact on serious diseases, recognizing the amount of uncertainty and the amount of risk that patients are willing to take in accessing those drugs. so those two things have in my

mind driven the resurgence of venture capital and biotech, so i would say from a big picture perspective, let's focus on those things. reiterating the call to make have consistent funding for biomedical research for nih and fda, these are the investments we make in the public sector that drive the opportunities to create these break through chores, and let's continue what we started over which isseveral years, a real mature dialogue about --ulatory process about just process, about drug development, at out risk about how we can responsibly accelerate getting therapies to patients. x thank you. -- >> thank you. and i dooe gray, appreciate the attention you are giving to this critically important area. i come to this discussion with a background in --

>> just put the mike down. >> i come to this discussion with a background in physics engineering, and somebody who has tried today attention -- try to pay attention to the technologies to understand how we can best explore this discovery development delivery cycle. and i would encourage us to think about this as perhaps a four-step cycle. one of the things that is limiting our ability to move drugs effectively into the clinic is the fact that we do not take advantage, take as much advantage of the trials that we are conducting as we might. the technologies that we have an enormouse us amount of information about the behavior of the biological systems that we are try to manipulate therapeutically. the reason that these drugs and treatment strategies are so expensive to develop is that most of them do not work as we

expect them to work. what that means is that the model systems that we are using are not as informative as we might like them to be. and i think today with the technologies that we have in hand we have the opportunity to learn in great detail why it is that the strategies that we are failing on do so, and to use that information to go back and improve the model systems that we are using to guide the development of these treatments. that requires that we change clinical trials so that we make sure that every -- that we learn canmuch as we possibly from every patient. means we have to get the samples to analyze them, that means that we have to broadly distributed the analytical technologies that we need to understand how they differ from the model systems, thate need to bring now individuals from the basic sciences to help us understand why it was that our models did

not teach us as much as we needed to know. and so i think there are two things that i would suggest here. one of them in today's community, the basic science faculty, if you will, researchers, are being drug farther and farther toward the translational side of the house, and sort of away from unfettered basic science discovery. one of the things our technologies are teaching us is the complexity of the biological systems that we are trying to manipulate. they really are much more competent than we thought a decade ago, but we have the technologies that understand now these molecular machines of life. this is going to require we invest in some pretty basic science to understand how these molecular machines work. so i would call first for clinical trials that are designed to be as informative as we can get them, investment in substantialesearch,

investment in basic research to understand what we do not know about our systems, and then competent than wefinally, as ise that we continue to invest in analytical technologies in this country. genomeans microscopes, analysis capabilities, advanced computer systems that are designed to securely manage the unprecedented amount of data that we are generating. we are really generating in a response of data that have to be managed securely. it will take a new kind of computer industry to actually do that. but i think one of the problems that we have in this country today is that we are falling behind in our technical infrastructure. it is difficult to acquire the instruments. it is difficult to replace them when they go obsolete. and this is something that happens pretty much in 18 months and weo-year cycle, cannot sustain them with service contracts. we are going to have to keep the

infrastructure and technical infrastructure up so it becomet cease to competitive. mr. chairman, on the white paper that you circulated with your invitation, for which i am is grateful as everyone else here, it started i believe or early on with a quotation from a gulft of a perceived between the basic science here and her translation of this science interworking drugs. i have read quite extensively, and i think this view is widely shared. cancer is one of the most generously treated areas by the fda. there is a 2010 report from the biomarker confesses collective, i think, was the title, which i -- ive representatives believe included representatives from industry and maybe conclusion.

i think you can break -- i am so sorry -- >> just speak a little closer. >> you can break down the essence of the fantastic resolution -- revolution we are witnessing into four categories. for the first time in medical history we are able to see absolutely everything down to the molecular bottom. there's nothing remotely comparable to this revolution since the advent of germ theory 150 years ago. it has transformed medicine. that led quickly to vaccines and antibiotics. is cost of doing that dropping, not rising. we're putting that on chips. we can see the proteins, we are

really good at seeing what is done there, and that is very useful. down at the molecular level, that is where the action happens. are having extremely good, if you show a biochemist at target today, and say if i can get my drug to the target, can you destroy it, they're really very good at that these days. this is not the oend of the store, so we have drug design prizethere was a nobel for in 1988. we have entered by these -- we have antibodies which target drugs. we are doing remarkable things with live cell therapies. stem cells which can be genetically engineered and return to the patient and sort of sibling group of therapies using white blood cells, extracted from the patient and

engineered to attack cancers. some stunning results coming. this is the newest area. and you look at how the people involved are engineering b cells, and it is as logical as writing software for cell phone. they're taking a gene or turning one off. they are dispatching it to do something. the nimble genius of this is just stunning, and the other two areas i am confident that we still lead the world, employee interesting to see the therapies we keep up. a lot of other countries are more willing to break every uleulatory role and move -- r and move forward. we will see. this new technology and the new ability to see everything poses -- has presented us with a scientific and policy question. the scientific part was alluded to by dr. gray. we are finding things are messier than we might have hoped, more than they looked

when there was one bacterium and we could just go after it with tetracycline or something and mission accomplice. with these complex webs, there are large numbers of variations in the structure of molecules that propel diseases. then we are increasingly seeing the molecular etiology of active disease. firste in front of this with the advent of hiv in the late 1980's, and they are comics and slow. this means that at least using traditional fda trials, the process is slow and it is complex. we often do not get the right results from trials unless we are thinking in these terms. the economics of drug development has been cited, a large center of that the government is on the process, so you have politicking and all your upfront investment in the trials are expensive to conduct and the clock is ticking on your path.

is the scientific challenge. you got to work out, you can see all the stuff done that, you can modulate some do, but you have tried to work out what to modulate. i have to say, since your passage of today can and i may be dreaming, but i think nih has launched a whole series of project a beginning around 2013, they go back earlier than that. thenhad projects and since the one very interesting study going back to single charles of the past that have failed and going to work out. why did they fail, and they are plucking out the responders, this is terrific stuff. the regret is we were not doing it all along. i'm not sure why we are not, but is an that program absolute paradigm of what we should be doing to get this right. now we move into the drug and into the biomarkers. target selection is important, but also prognostic biomarkers.

those are important because they see directly into the fda approval process. rule,celerated approval which we know about or we ought to, invented in the late 1980's, but added to the federal regulations in the 1990's, let's regulate, and give accelerator approval on the base of circuit and points, and that hinges on our gnostic biomarkers of one kind or another. and i h is to my mind doing exactly what ought to be done, and i do not know if this is a russian, but they are doing it exactly right. important thing we can do i believe to really seriously accelerate drug approval and lower the cost is to get the fda -- the fda's at the table for a number of these initiatives. the fda is at the table and giving input.

others can tell me how detailed it is. we should have one process that makes these calls, not do them multiple times. we want to bring in other stakeholders. if we could get that process moving, we would substantially accelerate the approval of drugs , lower the costs, and face less talent and competition from other countries that are willing to do this quite possibly less well than us, but are willing to cut a lot of corners. >> thank you, all. let me say, and i will let a colleague asked some questions here. ask will let my colleague some questions here. you are just bound to speak of the questions we have asked. you have been waiting, you are excited, and suzy este, and it is about time someone actually allowed you to answer some of the questions. just one, ite ask

seems like many of you have covered all four. i am not going to take the next three questions, but instead, i know a number of my colleagues here would like to ask some questions, and we will do a sum-uip at the end. we will try to and buy 5:30. -- by 5:30. we will be very informal here. no one will have unlimited time. if we could limit ourselves to a question or two, and that each of you respond, and help us. >> thank you for all your presentations today. it just seems that the day sick investment ine research that is needed for the whole process to work, and if you ask anybody in the public what they think the best investment government can't, it is at the national institute of

health. yet we have heard the term revenue to have more predictable levels of hunting. i am concerned we have spending caps, dr. collins, in place with non--defense is eric, by as much as which would cut as much 15% in 2016 under the ryan budget that was adopted by the house, and that would jeopardize critical federal funding as a result undermining the discovery process. we have also had sequestration, which was supposed to never happen, what did happen, and nih was severely affected by that. ,o i am interested in reliable certain, and adequate funding levels for the nih. that is my question to you, and for dr. woodcuts, this seems to be an underlying assumption that all the time it takes to get drugs approved is a problem at the fda. and i want to know if that is acurate, or could you give us

more transparent understanding of why it takes a long time and should we lower the standards for approval of drugs without knowing whether they are working or not? if you would both respond. question.ou for the there are various ways in assessing what is the state of health of biomedical research funded by nih. one can look at the trajectory over the last 40 or 50 years, and you can see that in fact for supportthat time nih was pursuing a fairly stable trajectory, about inflation, about4%, that was to for 1970 up until 1992. there was a doubling of funding fornih which was beneficial ramping up the ability to do things faster and in a more risk-taking way.

which was bipartisan. waxman-upton bill in the house. >> i should have been more explicit. >> it was actually up to waxman. >> i think the expectation of everybody was that i was going to sort of set a new basic. at the end of that doubling, essentially nih's budget standoff, and any remain with inflation gradually eating away at that purchasing power. the sequester added amazingly to that pain, cutting $1.5 billion through fy 2013. we are still below where we were in 2012. the consequence of that is in terms of purchasing power. nih lost more than 20%. if you look at nih budget as a gdp, and if you

look at what other countries are doing, it is in the opposite direction since 2003. they have been growing as we have been shrinking, and the consequences are quite clear. in terms of what the average investigator experiences, and this is the thing that -- >> dr. collins, my colleagues are going to start to hate me. if you take too long, and i only get to ask a question, we do not have adequate funding, and we have a budget that calls for severe decreases. do you think that makes sense, or does anybody here that makes sense for system where we are trying to find cures? >> why is the health of our nation's missionary? -- why is the health of our nation discretionary? from my perspective this is one of the most important investments a country can make. every other country in the world seems to think it that way. >> thank you, so i think that is

an important point, and the way the house is already operated is to put in place cuts that are going to take effect later. dr. woodcock, it take so long for these drugs to get approved. >> there are standards and force by fda. after a drug is discovered, companies have to figure out whether it is worth investing in, so they do studies to see whether they think it is going to work before they put it into people. that is a good business decision, not driven by the fda. there is also toxicology studies that have to be done in animals make sure it is safe enough to be put in the first human volunteers. there are people who will say you should not do that come actually, if you're going to be one of those volunteers. have a very good record of many thousands of personal ind's that are very safe. effects, but they

can be managed, and so the clinical phase is the most expensive, and that is where the most arguments are about. it costs a lot are patient in the united states to do that, and those studies are done to meet fda standards to show that embodied to make sure that the drug is effective and it works. at the end of that day, the benefit risk analysis, so if it is a terrible disease, you do not have to be as safe, basically, ensor. -- basically, and so forth. the fda park is a blip on the end. we approve trucks as fast as 45 days or perhaps three months. specified time frames that are under a year for the fda review of all that information. >> thanks. >> i would like to address the

orue of fda, is it too fast too slow. the answer it is just about right. ago, i went tors the largest cancer meetings in the world, and companies said everything is faster in europe. we embarked on a study, because in fact patients were having in europethe drugs faster than the united states. this was an issue. research showed the opposite. it showed the opposite that everybody said they would never believe you unless you publish it, and in fact we were faster. we were substantially faster. what is important is that we have to be that are. what really is important is that these treatments have to work for patients. so having out faster, a drug that does not work, that is toxic come has no benefit for a patient or for a company.

yes, we have a lot of issues in the clinical trial system, issues about how we designed trials, but at the end of the day, what a patient wants is a treatment that works for then. -- for them. thank -- i do not know what to call you guys, panel, advisers, participants, whatever, patriots -- for your opening remarks. i do not think it is a surprise that dr. collins once a more stable and increased funding system. it would be news if he did not. i do not think it is a surprise that the ranking democrat on the committee agrees with him. i mean, that is not a news flash. >> and everybody else. >> and everybody else. there are financial constraints

we have to operate under. i was gratified to read a lot of comments on folks that have been speaking about regulatory reform, unfunded mandates. dr. woodcock mentioned something about a clinical trial network in which i think has merit. aboutchenbach talked reducing the risk in the system. dr. shuren talked about we improve electronic health records. ms. despres talk about a clinical registry. mr. gray was talking about greater investment in what he called analytical technology. and mr. huber talked about process form. ideas.ave lots of one thing that was not mentioned, mr. chairman, that i would like dr. woodcock to comment on, i have a number of small medical vice any fractures in my district -- medical device

districturers in my ever getting approvals from the fdi for devices in applications with they do interactive their electronic devices, with in therapy, and also in diagnosis. cms approves them, but the medicare will not give them a code or does not know where to fit within the current system. how do we handle something like technology it is a or a therapy that has been approved, they know it will help people, then we get out down in bureaucracy of trying to code it and try to figure out how to reimburse through medicare. dr.you need to opask shuren that. >> if he is the man, i'm willing

to listen. >> dr. woodcock throws me under the bus, so i will it's running under the bus. [laughter] what a great country we have. i will apologize by dancing and little bit around it, because i have my colleagues at cms, but you're raising an important point which is if i'm a developer avid technology, what i need is predictability, not just to get the market, but to get reimbursed. as a physician, but i care about is also patient access. we know patients to not have real access in many cases unless there is also reimbursement for the technology, particularly if we deal with more extensive technologies or part of an expensive procedure. many are disenfranchised because they would not have the coverage and money for it. we need to think about others circumstances were we can provide that more streamlined pathway and more guarantees if you're getting actually through fda, are there circumstances where the you should get paid for. where yous atie-in

are paid for and there is additional data collection. they're sometimes called coverage with evidence development, so we will take it as it comes, but there is additional information but at least you are getting reimbursed for it. to that is a critical area be looking at, how can provide that later payment or disability. >> thank you, mr. chairman. >> thank you. let me thank chairman upton and for puttingdegette this together, and it is beneficial for everyone participating. i just want to throw a general session, maybe dr. collins, mr. gray, or anyone could answer it. was inantly hear -- it the white paper, dr. collins mentioned and mr. waxman mentioned about funding. in other words, obviously there

is not as much money available to nih as there used to be. a researcher who went to china because he thought betterre -- there were opportunities there. mr. gray mentioned the technical infrastructure that we are falling behind. i'm just trying to get a handle. we are constantly told we are falling behind in the sense we are not keeping up. is it because of lack of federal funds? is it because we are not investing in labs or technical infrastructure? china, it is hard for me to figure out what that means because china is a communist state-run, here we have money for research, or research being done by private companies, state ticking in, universities kicking in,. is the advantage that china has to click that their national government or their government is spending more money, or does it go beyond that? does it have to do with the fact

that they organize all this in a very dictatorial fashion? -- when trying to get we say what we can do, obviously we can't spend more money, but is it just a question of federal dollars, or does it go beyond that in the sense that it deals with the infrastructure or it is the way they get together to do their research in the way that we do not? >i know it is a general questio. >> i hear it all the time, so it is difficult to see that comparison with china because it is such a different type of political structure. >> and china is not the only example. to answer your question about why they're able to do disk so quickly, they have a decision process that is much more top can, and they basically make such a decision and implement it quickly, and may have been increasing their support of medical research about 22% per year now for several years now. within the next few years they

will outstrip the united states in absolute dollars spent on medical research. that is their goal. they have been very clear about that. i have a system that basically allows that happen once that decision is made. it is not just china that has read our playbook. singapore, south korea, brazil. look at europe, which is not an easy economic time. germany or the united kingdom an recognizing the medical research is a strong component of the success of their economies. they have protected that and have continued to increase support even at a time when other things are being cut cap. they have looked at america's story and are trying to learn from that. my greatest concern is not about the way in which this is affecting anything in the way of nuts and bolts or bricks and mortar. most important resource we have, which are the scientists, the biomedical research nudity, particularly the next generation. they're spending 70% of their time just writing grants because

their success rate has dropped to one out of six. most of the time it is another failure. they are increasingly taking less risk because risky science does not seem like it has much of a chance. we're coming up with all sorts of ways to stimulate their innovative instincts, that they are fighting a bit. if we really want to see our future, we have to have that resource supported. that is what wakes me up at night. we are leaving about half the signs on the table that this next generation of science wants to do and would have been supported in the past and now is not. you really have placed the question right dead center. to my way of thinking, it is not about just investment or funding. is about creating opportunity. and that is a much larger set of issues, and it has a lot to do with some of the other things that go beyond funding, like

changing the infrastructure, for example. at the core of it is the point that everyone around the recognizes that this explosion in science and technology is not just changing medicine, but all the rest of the life sciences. in fact, it is not just about nih biomedical research. as dr. gray pointed out, we are seeing a convergence now of the physical sciences along with the biological sciences and the emergence of material sciences, nanotechnology, for example, asputational science, is equally important this opportunity for this country. so we should be paying attention even the agencies beyond nih which are normocytic important. are economically important. what committee has laid before

us is a challenge get beyond just the question of are we funding, but more importantly, are we creating the opportunities and changing the ecosystem in a way that really will keep us at the forefront. that, thely build on creativity happens in large part in universities where a job but somebody like me is to --ilitate severed to the serendipity, because on time the innovations that are the boundaries between traditional disciplines. so one of the things we're doing at michigan is how do we consciously bring engineering and the life sciences together? biomedicalnged the engineering department from being solely in the college of engineering now to being joint in the medical school and the college of engineering.

is that human-human interaction with the creativity occurs. there are critical facilitators, finances, technology. much of the breakthroughs in medicine in our understanding our technology-dependent. so what we are trying to figure out is how do we give real problems to the engineers so that they can help us make advances in understanding, not just of disease, but also of actually is the ultimate goal for where we are. so it is looking at the total ecosystem, not just one piece, that is really critical to maintain a creative advantage and the lead that we have had for arguably over the last century. >> just to continue this dialogue, i want to come back to

one of the consequences of the decline in the real spending dollars that is happened over the last few years, and one of the things that has happened is the sciences has become hyper competitive. dr. collins mentioned that people are spending a large writingf their time grants. they are spending a large amount of their time writing nonproductive grants, that will not get funded, so you have taken the thinking time away from your scientific community that would be used to innovate and are forcing the community to devote that to just pushing paper around. and that is really a killer. so one of the reasons that we are calling for stable funding -- maybe it doesn't have to be vastly increased funding, but --ategies did deploy funding strategies to deploy funding to ensure that our most productive scientists are spending their time thinking about science and not writing grants.

if we could aspire to have long-term funding goals in this country, perhaps with achievable, granular objectives, then i think people would feel confident in saying i can't take this, try some new things. a job not be out of because somehow i took on a risk and fail. we have to enable ourselves to break out of this ever competitive cycle and get back to thinking about science. we can do this by defining where we are doing and what money is going to take to get there and identifying the scientists that we need to get us there. aboutt to echo the point it not just being china, i also want to punch with the idea that even the european community has really banded together because they see this as such an amazing growth opportunity. fastercures -- how do different

to solvecome together problems. --ere are a lot of co consortia happened in united states. the number is going up. i look at it is of is the u.s. declining and more of his everybody catching up? perhaps if you can be thinking about it in terms of what is it that we can use to keep that competitive edge, to keep the secret sauce of the american tookuity, innovation, what place at the beginning of the biotech revolution. there are a unique attributes in terms of how the american scientific surprise opportunities -- scientific enterprise operates that we do not want to lose, because what it has produced thus far and what we expect it to produce. at the. is still looked

place where everyone to model after. they are modeling after the gold standard of the food and drug at administration. they are modeling after an ih and the other site agencies that the doctor articulated. the question and the challenge in front of you all is what is that model going to look like in 10 years, 15 years. we are talking about bring in different disciplines can bring in a collaborative spirit, teen science. i have a daughter that will be simulating the westward expansion through the fields in virginia. she at 11 understands teamwork in an entirely different way that any of us were schooled in. if we want to entice kids like that to go into science careers, to have to be this sense that there something for them to be stepping into, and i think if we lose that opportunity, all of this work going on, whether china, europe, lots of other

places, it is this going to keep going, because all those countries have prioritized this and said this is something that we need to be paying attention to. >> dean kaman is going to be one person we're going to rely on. of course, that's his focus in life. sarah? >> thinking of this as an eco system is absolutely the right way to look at and it you've talked about a continuum of discovery development and delivery and it raises the question of how you're going to make sure there's actually a feedback. we've been hearing about the need to invest in research and that's critical, but there's also the need to ensure that we have the tools to collect the data and disseminate the data.

i saw a trial in sweden, very expensive and they're able to do that because they have the electronic house records. you think of what needs attention in the united states that we can be competitive in terms of innovation, i urge you to keep in mind the need to make sure that we have the data systems that will allow for researchers to bring the products to market more quickly and also to understand how these products are being used in the real world. it's one thing to see them in a clinical trial but in the real wofford it's a different game and what happens in the real world experience can inform the next generation of products. the data collection is a critical component of this as well. >> thank you, mr. chairman. thank you for convening this group. i think it's extremely important that we have this discussion.

i have an observation and then a quick question. the observation would be, the last thing we did in calendar year 2006 was reauthorize the national instudents of health. at a base level of $31 billion, to increase by 5% every year. we were eviscerated every time because that number was woefully small and then no subsequent session of congress, republican, gat or divided government came anywhere close to 5%. and it has, as doctor collins pointedous, much less than that. programs we should consider a reauthorization that had been passed some years ago. perhaps we should consider reauthorizing n.i.h. dr. collins, all the buildings on your fine campus are all named aceh appropriate rators. it's the authorizers who are your friends here, not the a proto-- aprohm rators.

you hear a lot these days about the right to try for people who are serious or terminal illnesses who may find there's the availability of a clinical trial that they can't get on. i think the goldwater institute is doing some work on this. can we talk about that a little bit? >> i can. as far as for pharmaceutical. generally speaking, we have a very -- policy about people getting on the what people call compassionate use but basically treatment protocols with that -- if they can't get into a trial for an investigatal drug. we had 1,000 requests. we only turned down two in a year. we get about that number of requests each-year. however, companies, for a

variety of reasons are sometimes not willing to provide drugs that are investigational drugs to patient under treatment protocols and they have a variety of reasons. shortage. they often say they're concerned that a side effect will be found out in these patients that actually wasn't in the clinical trials and it will delay their development program. never happened but that's often cited as a fear and then there's a financial cost to providing the drug, although you can do cost recovery, you can't sell the drug at a profit in a treatment trial. and so there are many people who have been unable to obtain drugs under treatment protocols from a company. that does happen. >> is there anything that we can do? this is a round table about solutions -- anything we can do as far as providing companies the certainty or the liability protection that they might sneed

are there places we can go with that? >> i think you could ask the companies. from the f.d.a. standpoint, we'd be very reluctant to have you pass something since we can't like -- look at the treatment experience because that would be unfair to the other patients. but otherwise -- >> this issue of expansion is something we're looking at. we're working with cancer research in brooksings and asco. there were interesting proposals on how we may approach that problem and get data that's what's missing often. often we have no data an exactly what happened. so there is a very vehicle -- unique, very interesting proposal we are looking at in our meeting in november and it will be interesting to see if there is going to be a legislative need to do something

on it. it is a very legitimate issue but at the end of the day we really need to get data. we want that known what happened, if the patient had access to it. did it work, did it not, if not, why? capturing data is going to be important. >> trying to alternate between republicans and democrats -- let me go to kathy and joe and leonard. >> touch. thank you to the chairman for leading this sexevert thank everyone here on this panel and thank you for devoting your professional careers to improving the lives of american families. it is an exciting time for biomedical research in the u.s. and across the globe. i have two questions, two points, mainly for dr. collins but for anybody else that wants to comment. many of you have already stated how much you value our scientists and researchers and

it's just fantastic to me a lot of these young, talented researchers, for example, at the cancer center in tampa. the university of tampa at florida, all of the research universities across the country and they are not shy in talking to policymakers about the fear they have for the future. that they have -- they are passionate. they believe in what they're doing, they're making progress. they see all the technological advancements but they're very fearful that based on inconsistencies and uncertainty in funding levels that this isn't the career for them and i hate to hear that from them. so i think that congress right now has a very important responsibility to make a new founled commitment to the n.i.h. and to biomedical research in america. the bunt this year, i offered a

committee to move n.i.h. funding from discretionary to mandatory. i think this is something that we should discuss. maybe we do it with dr. graves' suggestion that there are certain benchmarks that have to be met or certain requirements. or if you could discuss discuss that and there are other budgetary solutions. number two, we don't have all of the resources in the world to spend. are you confident that moneys are going to the right place in biomedical research? for example, when you look at much of the dat i-, a the aging population, it would seem like we have to do a whole lot more in brain research, in alzheimer's. but when you look at the threats to the health of america, boy, that's staring us right in the face. so number one, shifting to mandatory funding and two, are we making the investments in the

right place? >> i appreciate the question. in fact, i appreciate this whole panel discussion. again, i'm not a budget guy so i'm not going to be a clever respond end to this notion of shifting the dollars from one place to the other, but what matters for biomedical research is to find a path forward that has this stability. we've lost ground. to have a confident sense that we're not going to be a -- on a roller coaster ride to feast or ma fin. if the mandatory fund was a way to achieve that then i would stand up and cheer. i'm not sophisticated enough to know how those options might look like to get there. with regard to how we are using the dollars we have. you're right to ask that question. i'm always glad to have that question asked. we have to have the ability to respond to any querrey about whether the dollars that are

being allocated by the congress are being widey used and we are looking every day at ways to achieve new efficiencies, by partnering up with other agencies like we have with the acceleration partnership or working with darfa as we are on this toxicity chip. working on the brain initiative, which is going to be between darfa and a number of private industries and philanthropies, trying to make sure these kinds of interdisciplinary efforts are tapped into in every way. e are even looking at our peer review system. while we claim it's the best in the world, with -- are we sure about that? are we sure it's reflecting the scientific opportunities of 2014 is -- and knot some earlier stage and do we need to thing about the way those review

panels are established. we're pretty bullish about that. another thing we're doing -- this reflects something that joe gray said -- to try to see if there are opportunities to put some of our grand funding into programs where you give stable support to investigator over perhaps a period of five years with the expectation that once you have one of those awards you're going to get that down to science and not spend all of your time writing and rewriting. this is a program at n.i.h. called pioneer awards that started this. it's been very successful. we're looking to expand that into other parts of n.i.h. and do some pilot experiments. i think that will be a possible way to deal with this terrible waste of people's time in grant writing. but there's no magic that you

can snap your fingers and say now it's fine when you've lost 25% of your purchasing power and people see the current circumstance, you're not going to be able to really fix that without getting back on that stable tradget rip. whatever we can do to fix that that's got to be job one. >> i think one of the most important points of this effort is the fact that you've exited to a long-term look at the problem and i think there are some significant issues that need to be addressed beyond just staple mechanism of funding. for example, are there new resources that can be tapped that would support the kind of investment that we need? for example, looking at moneys that could be repatriated but done in a way that were directly related to funding, what created those opportunities and dollars for the via in the first place. the other thing that's a quite difficult issue to take on but i think would be an important

consideration in how we're spending the dollars is to really look at incorrect costs and the issue of funding infrastructure versus funding ideas. one needs to actually roll up our sleeves and begin to take a deep look at the problem if we're going to create an entirely new way of sustaining it for the future. it's not just a matter of a mechanism for more money but also of where we're getting the money and how we spend the money. >> thank you. thank you, mr. chairman and diane for this new initiative. is it on? ok. and this is just the first of a three-year process and the first step is discovery. so you're the first briefing. we will have other briefings and hearings as the health he and

and we will translate the -- subcommittee and we will translate the information given to us to other members here. if i were doing another d, i would add diagnosis. early medical diagnosis of the disease because the faster you can get the cure to the disease the better, the least costly. i have a couple of questions. dr. woodcock, you mentioned something called clinical trial network. would you mention barriers? do you need legislation or what are the barrer yers now that prevent us from having clinical trial networks? and how can we use our technology platforms to accelerate the cures to bring researchers and patients and innovators together. a couple of questions. > well, these are all related.

-- however, i think a lot of the barriers have been cultural and the way we've done things in the past. this is how we've always done them so this is how we'll do them. but the availability of the electronic health record. the availability to reach out to clinicians all around the country, to join them together and to do trials utilizing that infrastructure and training could provide all sorts of link networks. it could very rapidly answer questions. we ask questions, does this work, is it safe? you can rapidly answer those questions. you can get those crossed in a patient, test them and get the answers out. so a network is simply a group of trained people who are eager and ready and have the protocols in place to evaluate something, maybe many things and registries are where you identify patients

and then you follow them. you can do trials in that registry. you were randomize them in that registry, one treatment or another and then follow them. this type of infrastructure we don't have too much in the united states. except we're driven really by patient groups who have gotten us set up to advance their disease. what ellen was talking about was driven by a patient group. lung cancer trial. that trial right now is going to start out with probably five different investigative agents that be be evaluate sod that every person with that type of lung cancer, if they're screened, they can be part of that trial. >> will clinical trial networks work with medical devices? >> it would. actually, we -- back in 2007 had talked about how you might better identify centers of excellence. clinical trial sites that can do

a better job and put out should there be a voluntary certification, not something government runs but market could drive that. that's for innovators to say we know these centers are really good in these areas. some institutions might be good at heart surgery, some on bowel surgery. that could help. those networks in place, you might be able to rely more on data collection and -- to market because those networks are there. they'll be using those technologies. they're already geared to gather that information. we'd love to see something like that here in the u.s. >> i just want to respond to the clinical trials that janet woodcock just mentioned, lung cancer that will start at the end of the month. not only drugs but c.b.h., we're

using sequencing. we're use agnew and interesting platform. it's going to be done at every cancer center, every community in the united states, maybe even canada and it's public/private. so we will learn quickly -- first of all, no patient will be refused access to a drug. everyone will be screened. 100% of everybody on the trial will be screened in sequencing. if we don't have a specific fire marker for that patient they will get therapy. we'll learn more. it's efficient, much cheaper than doing a normal clinical trial and it is truly public-private. it is the way of the future. n.i.h.ave cbrh drugs and at the table at this thank you, mr. chairman and

this is among the most interesting discussions in which i've been involved in in this room and i want to thank both of you for it. there will be a test. that's why i've chose on the stay, diane. i favor stable funding, dr. collins. you and i have discussed this and i think that the roller coaster has been unfortunate and the roller coaster should be con find to disney world or coney island. however, from my perspective, i'd be perfectly willing to discuss in a cordial fashion how we move forward on budgets long term. perhaps we couldn't be as generous as would be optimal. but of course, we need a partner in the senate that passes a budget and i hope we have a partner moving forward and we can have an honest discussion as how best to fund n.i.h.

appropriately in the national interests and perhaps one of the ideas that have come out of this discussion this afternoon, and dr. burgess raised it is somehow reauthorizing n. himplet and i think that is a very interesting idea and i'm all for naming a building for dr. burgess. we have a discussion about china. and i think it's informative but i don't think it's dispositive. and to the panel in general, as i understand the system in china, it's quite different from the system here. here we rely on governmental funding, on the nonprofit sector, so ably represented by a great unfortunate and a great charitable trust and we rely on the for-profit sector, including the drug companies, many of which are headquartered in the district i serve. nd we rely on venture combat lis. so i would ask mr. leff, the

three-legged stool, what should we be doing, recognizing we have a greater responsibility with more stable funding for the n. h., recognizing regarding various universities we need to make the system better so the fact that only one in six grants is awarded should change. we -- regarding the for-profit sector, chashed we be doing? repatriating profits from abrood perhaps but what else should we do to encourage the for-profit sector in a system which is quite different from china's? >> that's a great question and thank you for that question. we've talked a lot about investment and n.i.h. and this country invests about $30 billion a year in n.i.h. funding

for medical research but private enterprise, private companies and investors put about $80 billion a year into the r.t. the. >> precisely. >> hopefully it's obvious to everyone here that this 30 billion and the 80 billion are deeply intertwined and in order for the private sector to make those nevertheless -- investments there has to be raw material coming out of the basic research. in order for that investment in basic research to create new cures there has to be that investment in private sector or to develop those scientific discoveries into cures for patients. talking about that, that 80 billion is important and it also got the benefit of not being a zero sum game we have to come up with in order to solve the problem. in order to enhance that

investment, that 80 billion. it's obvious that if that goes up, we'll get more cures and treatments coming out the other end. >> we'll be saving more lives. not only here but across the demrobe and china. >> absolutely. if that goes down then the opposite will be true. so i would encourage this committee has it thinks about this issue, to treat an economic problem with economic solutions. that is to say that to look at the decision that investors and companies make about whether to fund biomedical r. and d. and which moneys to put into it is fundamentally economic. we all want to save lives but ultimately investors will put their capital where the returns are. if returns in biomedical r. and

d. is less attractive than eelectronic devices or whatever it may be, capital will go elsewhere. when you look at the recent history of policies coming out of congress and this committee and elsewhere, you can see that impact and you can see the kinds of things that have been done from a public policy point of view that have enhanced investment in biomedical r. and d. i would point to the or fan drug act as an example, where it was recognized that credit for funding in orphan drugs ensure that there is a better economic equation for making nose investments and we have seen dramatic increases and huge productivity in investment of orphan drugs. i think we've seen already some of the fruits of the gain act, which was part of the f.d.a.

safety and innovation act in 2012 where exclusivety was provided for anti-beauties that resist -- antibiotics that resist infections. those are a couple of examples. what are some specific examples of what we can do going forward? one is to continue to enhance this dialogue about empowering the regulators with the tools to help speed development of the knost important new therapies. it really was f.d.a. who came forward and said we need breakthrough designation as a tool and that's worked out extraordinarily well in lowering the time and cost and developing certain therapies and attracting nevpl. most recently, the f.d.a. has come forward and said we really

need a use tour. ometimes callled the l-pad and there's new legislation introduced. i believe it's called the adapt act in the house, to implement that. that's a great step forward in that it's providing the consensus and the will of congress, it will of this country, to give f.d.a. the tools it needs to appropriate flexibility to accelerate the development of certain kinds of drugs. i think we need to try harder to give the f.d.a. what it asks for there. where this tool is not just applied to new tools but anything this could help accelerate when it comes to parents. certainly when we talk about tax reform and economic incentives these things matter and they matter to the biomedical

industry and to investment. one other dimension to mention here is how we pay for these new and innovative therapies when they come to market and this is a really difficult issue. we awe all no that expensive therapies cost money, cost the budget money. at the same time, if we're not prepared as a society to pay for the real innovations that provide real value, real cures to patients, then investors won't make those investments. it's providing, i think, a dialogue which recognizes the value of innovation and recognizes that it's appropriate for new therapies to have a period of exclusivety in which they can recoup their investment and for the ones that can provide real value for patients and really save lives, we ought to do everything we can to figure out how to get them to

market and how to pay for them in a manner that creates the cycle of further private investment. >> thank you. let me say that this is an issue that deeply interests me. my bill modern cures is involved as are other bills. and i hope we can work collaborative on a bipartisan capacity on what is a three-legged stool in this country. mr. chairman and others who are leaders, i think we'll have to, as well, work with the ways, means, and ways committee. there's tax policy involved. thank you, mr. chairman. >> thank you. i know margaret has a quick thing and jim. i just want to say too, it's my understanding that we were able get through permanent tax credits on the floor -- and i expect that on the floor soon.

shouldn't be too much longer. i'll close things out. >> on the front end of the funding conundrum, the work of dr. andrew lowe has received sort of star status as far as the creation of a cancer megabond. the idea is how do we aggregate risks in terms of funding that can go to companies doing development of products? i think that is certainly one that the group should be looking at just in terms of the themes that he's articulated and then on the other side that was just raised by jonathan, the issue of how we're going to pay. there was a panel discussion on this that was extremely insightle. if you build it will they pay? now we have to worry about what do we do with this innovation? can we afford it in the larger health care system? i think there's an exciting opportunity to close the circle of it.

if we can prevent disease, please, let's prevent it. if we need cures, let's figure out how to fix that part of the system and get the funding to do that so that we can have a robust economy. but we have to pay attention to the other end too, which is how are we valuing this evaluation and how are we as a society being able to take in what's coming through? in years past, jonathan and his work and much of the work presented here, when we were all at meetings on innovation, the bogeyman you always heard, the f.d.a. oh, the f.d.a. is responsible for all the problems. not true, obviously and not what you hear now. i emailed some colleagues at f.d.a. recently and said what's coming up is the specter of not being able to have products be reimbursed. i think that's one everything