[inaudible conversations] [inaudible conversations] [inaudible conversations]

.. this committee. have i ever heard testimony such as given by you, and by you.

i want to thank you to the middle east becaus because the e can get good people together and settle everything. you should settle that and then come back and talk to us more realistically but for you you are what is wrong with this country. and the profit motive is good but only if it is aimed at something for the general benefit of the public and that could be stretched a little bit because the public likes to be entertained. i can't say professional basketball is necessary for the existence in america that some people like it so let's go ahead. but i think in your case you don't have that leeway. it's a matter of dollars for the 256% increase in two years in advertising and can yo then youe only for the adult bought the

children when everything else has come out of the hearings is otherwise. i think it's a dreadful. the head of the national institute of health says a lack of steady federal funding is hurting efforts to cure diseases like cancer calls & hiv-aids. in may doctor francis collins testified before the house committee on biomedical innovation. he had other witnesses discussed pharmaceuticals and for new technology, improving patient care, american competitiveness and other subjects. [inaudible conversations]

thanks everybody for coming on time. welcome to the first of what will be a member of roundtables of the 21st century initiative a collaborative bipartisan effort that aims to accelerate the pace of medical breakthroughs in the united states. as my colleague diana degette and i mentioned in the video that we've released last week as part of the bipartisan initiative we are going to spend the next number of months to review the full arc of delivery development and process to determine what steps we need to take as a nation to accelerate cures and treatments and accept america as the innovation capital of the world.

we cannot do it alone for sure. we need the support of and ideas from those of you here today were certainly watching online roundtables in washington and perhaps around the country as well as we hold hearings and the chairman has held subcommittees and we will solicit feedback from experts in the 40s and throughout the country with a lot of peters and questions and we need a lot of answers and we need to listen. no idea is too big or small. the only way we can accomplish the goal is to join together in this conversation and we hope to hear from you during that process. for those of you watching you can e-mail us your idea to cures at mail dot house .gov. today we are going to continue by hosting the first roundtable

of 21st century cures. we are lucky to be joined by some of the nation's greatest thought leaders in medical innovation. they are doctor francis collins director of the national institutes of health, doctor janet woodcock at the center for drug evaluation and research, doctor jeff director of the center for devices and radiological health, doctor james dean of the university of michigan school. doctor joe gray at the night cancer institute. doctor andrew president of the american health initiative in the chairmaandchairman of the pe manhattan institute. margaret anderson executive director, doctor peter senior fellow at the manhattan institute, doctor ellen sigel

and jonathan leff of the deerfield institute. thanks for being with us today. to get most of it like to ask diana degette to say a few words and then introduce eric cantor the majority leader and i would say in advance steny hoyer planned to be here today and had a last-minute conflict but we have the number two republican and number two democrat on board in terms of what we are trying to do to show bipartisan forward mark. >> thank you mr. chairman and i'm delighted to partner -- >> i'm happy to partner with you on this important effort. the 21st century initiative is an exciting new effort that has great potential to positively

impact biomedical research and innovation in this country. the united states has been the leader in the field for decades now i think we are at a crossroads. we can either work together to improvement us and as we hope to do through this initiative or we can fall behind. our effort will bring leaders and policy academia, research and industry to the table to dig into how we can more effectively and efficiently tackle some of the complex challenges in medicine. as fred noted research and innovation runs on a sort of cycle. discovery, development and delivery. it's in those areas we want to focus our attention and seek input like those who are in the panel today. since we launched the effort a week ago we've already seen tremendous interest in the attendance today shows the interest we have. the questions we are focusing on will be the key to "possible solutions to these complex

challenges. first we need to take a look at the current state of biomedical research and innovation in the u.s.. what are the drivers and the barriers? where is the u.s. leading and where are we concerned if we are fallen significantly behind. seconsecond hottest biomedical research and innovation translates to improving patient care and outcomes and better medicine clinics we know that there are pockets of progress and research being conducted every day across the country. what type of patient is benefiting and where can we focus our attention to reach more patients? third are there other countries we can learn from? the u.s. may be in danger of falling behind. what strategies and resources on the other countries employee into exile in the research innovation and finally are there concrete actions that we can

take to help advance biomedical research innovation in the u.s.? does the nih need more tools to harvest research being funded or how can we help modernize the drug approval process to take advantage of the cyclical nature of research innovation that we've mentioned in the discovery development and delivery? although these are important questions but they are also hard questions. i think everybody in the room is up for the challenge. i know we can be productive in the conversations both today and moving forward and i look forward to the discussion and i'm grateful to everybody else on the committee that is here today for joining us on this effort as well as those that are taking their time. thank you very much. i want to first acknowledge the leadership that you are

demonstrating here in bringing us together in a bipartisan way focused on what all of you spend your professional time about which is curing disease. so thank you for that and to my colleagues thank you for the commitment. some of you for decades allocating resources and decisions behind that and affecting the policy to promote what i hope will become common parliaments which is a culture of the cure. we ought to be a country that promotes a culture of cures. i know that all of us are about making sure that there is access to treatment for everyone but i will say that your group pretty much hits the nail on the head. we've got to get better at making it faster to get to the

cure and making it more of a reality. good treatment just isn't enough. thank you for the emphasis. the 21st century initiative by the members will be the tremendous story of accomplishment for the congress and if nothing else but congress should be known for making the right choices in deciding the right priorities and that is saving human life first, promoting the treatment and ultimately i think it is a win-win for everybody. a lot of the discussion on the hill has been about fiscal restraint and how do you deal with limited resources. you and i have had many discussions on this issue and there are all of us here in terms of research and what can

we do to do more? sometimes it is picking the righmaking the rightchoices witd then setting about how we are going to do more so i congratulate you on this and i would just ask and i know that it is imperative on the nature of the commerce committee and a bipartisan to think outside of the box. i think all of us can sit here and talk about breaking down the barriers in terms of promoting a culture of cures. spending $10 billion for taking ten years spending a billion dollars to develop a drug and bring it to market is unacceptable while people are dying so how do we think outside the box? yes it is improving the process and yes it is making choices like the first steps we took in the kid's first research act.

in the scheme of things 123 million isn't the 30 billion that we have protecting the nih but it is a step we are going to make in setting priorities and so i really commend the initiative and look forward to playing an active role however that may be according to the trans- desire to help promote the success and again, thank you all the esteemed panel from all over the country we appreciate your work and honored your honoe present and look forward to the outcome of results on this initiative. initiative. >> thank you very much and we will keep you in the loop there is no question about that. i'm going to ask th the chairman and the ranking member to say a few words in a minut minute ande chairman barton and now the chairman emeritus of the

committee. i want to introduce some of the members that are here. the chairman joe pitts of the committee is the ranking counterpart frank pallone on the subcommittee. the members of thei the leadersp chair of the republican conference cathy mcmorris rogers and members of th member of the, vice chairman and mike burgess from the health subcommittee in texas. ms. castor from florida who joins us and morgan griffith. mr. chairman and witnesses today i think it's important for us to figure out ways to develop secure more rapidly. we spend an enormous amount of money in basic research from which the pharmaceutical companies take advantage and are

able to produce products that are life-saving. we've got to encourage the development of medicines but we also have to make them affordable. we don't do anybody a favor if we have a drug that cannot be bought or that the health care system cannot pay for so as we think about new products and drugs and therapies, we must evaluate them to see if we are adding more to help the american people and of mankind or whether we are just layering new efforts that will cost a lot more and may not add to the therapy that people are so desperate to have. thinking outside of the box is evidence in this kind of meeting and it's interesting -- never seen anything like it in the time i've been in congress and i would look forward to the group

figuring out the recommendatio recommendations. i assumed the committee so we could continue that legislation if it is necessary. we all want to work with you and it's worthwhile to hear from people in a setting that is different because i don't even know if we can make a transcript of the proceedings but anytime that we can exchange ideas it is an ultimate good >> the congressman is just uncomfortable being to my right. [laughter] it's very good to be here. we are going to show our system at its best bipartisan under the leadership with the auspices of mr. waxman.

over the next however many months that the country is going to see america's political system at its best as we work together to decide how to take things out of the laboratory. some of you i've worked professionally but chang upton is an honest broker and he is making this a top priority and you are going to see real results created the american people are going to see real results and i commend you mr. chairman and i look forward to having the dialogue and ultimately coming up with common sense solutions for the cures that make america and the world a better place.

>> when we send out the invitations, you send a couple of questions. i would like to see where that takes us over the next couple of hours. and we asked these questions. one, what is the state of biomedical information in the united states? number two, what does biological information in for the american patients and jobs. three come out as the u.s. compared to other countries with respect to biomedical integration and how can we make sure that we lead the way in the 21st century and last, what steps does the congress needs to take to accelerate the discovery development and delivery cycle in the u.s. to foster integration and bring new treatments, and as we know to keep more jobs in the u.s.. a nice afterthought and where we need to go. what family isn't impacted by something that we can do here. whether it's this country or

someplace else. hopefully the ideas and elements of that is what remained of the listening audience that way people can weigh in and look at those as we begin to process. we want to sit here at the roundtable that is rectangular and see so many of the members represented here. i thought to myself when i walked in i've never seen

anything like this but i thought maybe my 20 years were not that informative. but when he said he's never seen anything like this, i know that this is a unique and special moment and began, much credit to you and what i hope will be a series of conversations. so to try to answer your question and not to go on for too long about it, certainly from the nih perspective of most desperately needed in order to continue what is the most successful story in the biomedical research the world has ever seen is a steady predictable trajectory of support. we have not seen that over the last ten years. we have lost more than 20% of our purchasing power. and that has put the system under enormous threat. it's cost. could cost jobs because we support the jobs and those have been trimmed as a result of this but maybe most importantly and most worrisome for the future,

it is caused by young investigators who are our future. they want to see the innovation continue at a level that it could need to have the confidence that there's going to be a path for them that their dreams are going to be possible to pursue. we are not lacking enough talent. we are not lacking in their ideas. this is a remarkable moment in terms of the research potential. but we are in fact in a situation paradox of enough where this is in a distinct mismatch with the ability of investigators to take risks and go out and do the kind of amazing science that we in america have been famous for and are capable of now. it is also perhaps the location where we are missing out on the return on the investment that might be there and i want to mention that as well because your questions reflect an interest in that.

i don' don't like you saw the analysis of the economic returns of the women's hope initiative, the effort was started 20 years ago which the nih started. the estimate is the return was 140-1 in terms of what it taught about women's health, the ability to put that into practice and to save lives and reduce cost. pretty amazing kind of consequence of that. in terms of where we are going, the innovation opportunities we are extremely energized before the kind of technologies that are possible in wishing to have been unleashed. but again being held back when it comes to things like personalized medicine where you can do the dna sequencing on the chip the size of a postage stamp and very proud with me an i prot that is joined with fda a human kidney made up of human embryonic stem cells, sorry that

are placed into a particular arrangement using three d. printing that you can basically study human kidney function the right from an individual in a very reproduced way. extremely exciting technology. but again things we are not pursuing at the level we might. we are trying everything we can to try to use this as a motivator for creative solutions working with industry in the accelerated medicine partnership put things together for science in the public and private sector on alzheimer's and diabetes and rheumatoid arthritis and looking in other ways to cut down on the-to go from an idea to an end proved therapeutic and we are bullish about the potential just going along with what the leaders that i think it is fair to say we are just as excited as you could imagine.

we want to cure cancer and hiv and those are all potentially within reach for the current system frankly isn't working. nih wasn't really broke in ten years ago but ten years of the loss of purchasing power started to break. if we had a chance to recognize this is an investment that pays rewards for human health for the economy for everything the government does well to recent analysis indicating that may be the most important entrepreneur in this system is to government yet we haven't about the entrepreneur to be what it could so the bottom line is if we could have the confidence of a stable trajectory for support that would mean the world to an enterprise. finally it is a point of the consequence i don't know if you

saw usa today in the last couple of days describing a typical situation of a brilliant young scientist in michigan who is at the top of his field and who should be fought to find this next creative faculty position and because of the squeeze that hasn't happened and he is going to china where he will find himself surrounded by incredible resources as china continues to increase its support by 20% a year while we have been decreasing. we can fix this but it will take all of you and the recognition that this needs to be thought an afterthought but a priority because we are at risk of losing something that has been one of america's greatest stories our success in biomedical research.

>> i have the privilege as the dean of the university of michigan medical school, of serving as the dean of the university of michigan medical school to actually be involved in all aspects from training of the future scientists and physicians to discovery of application and patient care associate and the whole ecosystem and what francis mentioned is that is instilled by a lack of reliable funding levels is absolutely true. however there are other things congress could do that would be very helpful. the regulatory burdens that have been overlaid on the faculty and

staff over the last several years are growing. these are unfunded mandates very basic things like depending upon what institute arguing with, what branch of government you're dealing with there are different conflicts of interest regulations. keeping up with that and being compliant is an administrative burden that has no value added into so asking the congress to help come up with a uniform approach would be very helpful. as similarly as we move our discoveries into the commercialization, we find that there are gaps in the ecosystem and researchers can discover mechanisms and come up with

targets and develop medicines but then it requires a large number of individuals and partners in the government to move that forward because we don't have all the expertise and the university and unfortunately those partnerships are sometimes difficult to develop because the regulatory burden. at a very basic level it would be helpful if in the fda there would be a checklist. the facult faculty as they moves for would have found a lot of cooperation from the members that are trying to help them do their job in the initial stages with have had to develop our navigators because the complexities of interacting with

these agencies are such that the faculty have to be educated and potentially there are ways to partner with the government in a different way to facilitate the interaction. i think it would be helpful if congress could help move governmental agencies for word as it is a uniform conflict of interest and ways that we could access bureau in a systematic way that is more transparent. hispanic audience ahead of the center for drugs fda. if you step back and i wanted to pharmaceuticals in particular and not some of the other innovations but if you step back and look at the whole ecosystem i think there are some major barriers that academics and

developers face when they go from a discovery to a product that is given to patients and in order to deal with this the trial system we have isn't a system. right now for every product they get a clinical trial and that takes about a year than they do the trial and then they shut it down and then if it is successful maybe they will start another tryout and they have to do ten different agreements with different universities and transfers and it takes years and it's exhausting and much paperwork and lawyers. what we are starting to do is look at clinical trial networks then when you get inventions

they test it right away and you have multiple drugs or diagnostics. it's faster and more independent if i may say so because the product is given to the network and they evaluate if so there is distance between the evaluators and the inventors but basically it saves a lot of money and you get to do comparisons because you are testing multiple products as well as therapy at the same time so that is something that could be advanc advanced. groups that have done that is where they succeeded in getting products for the disease on to the market because they have been ready.

another thing you may not be interested in but i'm going to bring it up because it has to do with jobs and so forth is the drug manufacturing there's a lot of innovation in drug manufacturing. right now we have drug shortages that are afflicting the hospitals across the country. we buy in many drugs from other countries and if something were to happen, we wouldn't be a bitt them anymore. that is another thing saved by the developers they have to scale up their manufacturing index like the clinical trials estimates updated and very cumbersome. there is now the technology available to do these continuous and yoveterans to make things ie united states. they wouldn't have to be all over the place.

it's environmentally friendly and it is the way of the future how drugs should be made where they are needed. so i think that is another area i have been under the clerk david cook critical path initiative there is a whole lot of translational research that needs to be done on the biomarkers and other things in the laboratory through and into the clinic and through the clinical development and that is another thing that really should be worked on is a tremendous opportunity. >> i want to thank you for your leadership and tell you what a great pleasure it is to be in the hearing again. there've been a couple of words that have been placed before you such as the ecosystem and francis eluted to the risk.

i think it's important to keep in mind process of the discovery development and delivery is now very cyclical and it really lends itself to tremendous opportunities with regards to acceleration of the process if we pay attention to the issues of what it does accelerate mainly the investment of intellectual capital and financial capital. and francis has already yielded some of the risk that our now associated and that investment and there are the risks with regard to the regulatory components of this and we are seeing even on the other end of the spectrum risks as it relates to their reward in the reimbursement and the challenges coming from that. so as the congress takes a holistic view of the ecosystem and looks at policy changes one of the general themes will be to

look at the initiative that would reduce the risk within the system that would enable a greater participation and investment in accelerating the cycle. if one of those particular things that needs to be addressed is the transfer of data across the cycle and the opportunity for the greater sharing and intervention. there are challenges on the front end as we see the transfer from investigators to developers and the challenge of the data sharing as they now need to create integrated products such as the combination of diagnostic therapeutics and there are problems on the other end as eluted to the need for looking at the clinical trial designs and the way that we are dealing with the data as it relates to the delivery of the interventions.

so you have put in place and enormously important process that would enable us over the next months to step back and take a careful look at all of the components of the ecosystem that need attention and reduce the risk that is currently growing and slowing down the process. hispanic thank you for inviting us to be part of this. it is an honor and a privilege. i came out of graduate school and worked for the office of technology assessment and i think this is -- although you described this as unusual and certainly i think was the norm for a long time to come together and talk about these critical issues so in terms of answering the question of what can congress do, you are doing part of the work which is putting a spotlight on research, science and innovation as a priority so

i would follow suit with what the doctor articulated. i don't think you can escape the responsibility that they are adequately funded. united for medical research has come together on the issue of nih and the alliance for a stronger fda is a group that i had the privilege of serving as a past president on wednesday fothe eve ofthe different stakes coming together to say the bowlerdollars are extremely vale and they are not enough. i think that's prioritizing the infrastructure has to happen and it's a critical necessity and i think if we are not listening to that though then i think in terms of the leadership function we run the risk of losing an enterprise that the united

states ine or. in terms of another priority you are starting to hear a little bit about the have to put patients at the centethepatientu get this did ask have 7,000 diseases yet we have treatment for 500 of those so the system is doing its work and a churning through the science and getting the outcomes to the door of the fda and then we get some approval and we put that out into the system but at the rate we are going i don't think that any of the diseases we are going to be afflicted with at some point in time stand a chance of being covered in the near term. so i think the speed issue that has been articulated i articulaa critical importance and so as this group does its work figuring out what are those planes as articulated it becomes

a place that will be of critical importance for the different stakeholders you gathered here today and going forward to come to some agreement about what are those pressure points. we have seen in the world at faster cures we had the privilege and the opportunity to work with a variety of different foundations to the cystic fibrosis foundation was noted. mike milken created the prostate foundation for the melanoma research alliance. there is a multitude of groups. but the system can learn and what we have studied is they are one-stop shopping in terms of understanding what do we know and what don't we know, what do we need to get prepared for, where is it going to go and how do you bring the patient into the center of all of that so a couple of points i want to make sure i talk about is the sacred innovation ecosystem and the

need for that stability to come into putting the patient at the center and i want to make sure as you do your work we are making sure that happens. hispanic first i want to thank you -- can you hear me? i want to thank you for convening this important committee and i want to thank the congress for their work actually recently through the reauthorization of the act. [inaudible] [laughter] we had a mechanism sponsored by friends of cancer research that was bipartisan and is sponsored on the senate side and the house side that supports the industry and patients, companies and a mechanism to get patients drugs

faster but better where there is evidence. i know diana degette sponsored on the house side so that is an example of how coming together on a bipartisan way isn't just aspirational issues but strong evidence to get drugs to patients that work and that are better for them. it is like a novel it is the best of times and worst of times and we have an extraordinary and biomedical infrastructure and we are doing incredible things. things. at the science has never been better but it is endangered and there are things that we can do better. we do have foundations at the nih and the fda that can be and

howard. we are convening a. we are putting the companies into it it's for lung cancer where there are no cures. its next agenda sequencinit's nd it is a partnership between the fda of mci, companies so there are ways we can get there and there are things we can do but we can't take what we had for granted and if we don't get the science fair tha then we are not benefiting. >> first, thank you for the opportunity to be here today.

one of the big inefficiencies and i would say barriers that we face which is particularly on a medical device side is the ability to make the use of the data that is collected as a part of the real-world clinical practice and this is the impact it's having so in the medical device program we've been looking at how we can reduce clinical trial burdens on medical device innovators and what they need to bring the product to market in appropriate cases shifting the data collection to the post market setting and what we would like to do is reduce the burden on the post market side by being able to rely on the data collected by doctors in their clinical practice. two weeks ago we put out to guidance is that proposed a new pathway for high-risk devices and a series conditions that address an unmet medical need and in that we build on experiences on the program on accelerated approval and add other features which allow for moving the data pay market and

post market but here is the challenge. if you look at how we collect the data a lot of it goes in electronic or otherwise medical records but it's very hard to identify which device was used. either it isn't fair or it's hard to locate someone of the solutions you directed was to create a unique device identifier that says who is the manufacturer. they will start having the numbers because we issued a rule last year. to build the fields in electronic health records to put it into the electronic records.

then we can make use of the big data. sometimes you need to data that is gathered in the clinical practice and those are the device registries and the challenge is setting them up so what are the barriers and incentives that can be in place creating the registries because when they are there we are talking about the easy collection and even in the casee the manufacturer doesn't need to get the data we allow the expansion on the device where the company and professionals decided we are going to do a clinical trial and we told them don't do it, don't waste your time. we looked at the data and we said we think it's good enough ask us to expand the labeling. so if we can have those kind of investments in the country and that kind of infrastructure to allow for greater data that we

collect every day we think that could have a big impact on reducing the burden for the products to get to the market and create clinical incentives for the devices. >> i would like to echo the sentiments of the other panelists and thank you very much for the opportunity. it's been accorded a little bite bit closer to the folks listening -- >> i represent the trust and i'm delighted to be able to participate and i would like to follow up on something doctor sharon said. we heard from the panelists that other vertical trials can be a barrier to the development as it can be a billion dollars or ten years to get a product to market and so the question is what can the congress do and there's opportunities to look at how technology can be used to support clinical trials, tools for the clinical trails.

in the area of antibiotics and we know clinical trials are a barrier to the development. we've spent a lot of time looking at the registries and it's interesting that they do have the promise of being able to access the products safety and effectiveness in how healthcare is delivered with interesting ways you can go. we have the united states doing some good work but for example there was an article in the journal about a trial in sweden using an existing clinical trial registry, i mean an existing registry for the trial of the cardiac intervention.

the study enrolled at 10,000 patients an10,000patients and 7e randomized and the cost was $300,000. $50 a patient for the 7,000 that are randomized. you can't do that in the united states. a two-day trial like that you could dcan do about for patient. so, there are definitely opportunities with the registries to actually help with product development, and i would encourage you to look at that as you are thinking about moving forward for the challenges of the trial the answer isn't to get rid of the trial as the gold standard. the regulators need the data to assess the product and the clinicians need the data to know that it is safe and effective and patients need to know if it is the right product for them answer the question is how to gather the data more

efficiently. thank you. >> i want to thank the committee for taking on this issue in this way. i testified in front of the committee three years ago and talked in my capacity as an investor about the trajectory of the venture investment and life science companies and presented the data showing that it was in an alarming state of decline as one of the investment in the country and i attributed that to the relentless increase in the time and cost of developing the drugs and medical products not just over the last few years but several decades to the point that we now talk about the statistics that take ten to 15 years and cost a billion dollars and we feel like that is an unacceptable price to be.

it's now three years later and looking at the same that tricks the venture capital investment into the innovative biotechnology companies that picture is different. it's much better it appears than just a few years ago and over the past year we have seen a resurgence in interest and the dollars being invested in the venture capital point of view so to answer the question of what we can do to build on that and improve product answer is to think about how is it that we've gotten better at least on this one measure over three years and i would point to the two factors that have driven the increase in the investment in the sector. one is the breakthrough science into the kind of treatment and cure that we are now investing in and developing and bringing to patients that are transformative in areas like

cancer, genetic diseases and others that is remarkable and would have been hard to predict a few years ago but is a function of the decades of investment in basic research and fundamental understanding of biology and the mechanisms of disease so that's number one breakthrough science. number two is a genetically improved drug development environment and regulatory environment by which i mean a focus and a patient groups and the medical community to work together to address the problem recognizing nobody is happy when it takes ten to 15 years to develop drugs that patients are waiting for too long and it drives away from the critical sector so when you look at things to highlight the plaintiff at the breakthrough

that's emblematic of a number of initiatives that has led to a different dialogue about how we develop drugs and regulate and think about ensuring that we get good products as quickly as possible and efficiently as possible especially for those that make a dramatic impact on serious disease and recognizing the amount of uncertainty and risk the patients are willing to take in accessing those drugs. so those two things have in my mind driven the resurgence of the venture capital and biotech so i would say in the big picture perspective with focus on those couple things reader rating the call to make sure that we have consistent funding for biomedical research in the nih and fda. they drive the opportunities to create these breakthroughs and

second, let's continue with research over the last several years which is a real mature dialogue about the regulatory process of drug development and a benefit and risk and how we can responsibly accelerate bringing therapy to patients. the patients. >> thank you. go ahead. >> i appreciate what you are giving to this critically important area. i come to this discussion with a background -- >> bring the microphone down. >> i come with a background of physics into somebody that has tried to pay attention to the technologies that are bringing to bear to understand how it is that we can best explore this discovery development on the delivery cycle and i would encourage us to think about this

as perhaps a four step cycle rather than three-step cycle. in the trials are conducted as we might, the technologies provide an enormous amount of information about the behavior of the biological systems that we are trying to manipulate therapeutically. most of them don't work as we expect them to work. that means the model systems that we are using are not as informative as we might like to be. and i think today with the technologies that we have in hand, we have the opportunity to learn a great detail why it is the strategies that we are failing on to guide the

development of the treatment that requires that we change clinical trials to make sure that we are as much as we can possibly learn from every patient that means that we have to get the samples to analyze them and that means that we have to have broadly distributed analytical technologies and we need to now bring back individuals from the basic sciences to help us understand why it was that of the models didn't teach us as much as we needed to know and so i think there's two things we would suggest here. one of them is in today's community the basic science faculty if you will researchers are being dru drug further and further towards the translational side of th the hoe and the sorhouseand sort of awae unfettered basic science

discoveries. one of the things they are teaching us is the complexity of the biological systems that we are trying to regulate. they are much more complicated than we thought a decade ago. but we have the technologies to understand how these molecular machines of life. this is going to require that 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 the true basic research to understand what we don't know about our systems and then finally this is going to require that we continue to invest in analytical technologies in this country. this means microscopes, advanced computer systems that are designed to securely manage the unprecedented amounts of data

that we are generating that have to be managed securely going to take a new kind of computer industry to actually do that. but i think one of the problems that we have in the country today is that we are falling behind in our technical infrastructure. it's difficult to acquire the instruments and replace them when they go obsolete and this is something that happened pretty much within 18 months to two year cycle these days and we can't even sustain them with service contracts, so we are going to have to keep the infrastructure in the country and the technical infrastructure of. other countries are doing a better job making the history instruments of the science available. >> on the white paper you circulated for which i'm as grateful as anybody else here started i believe they quote

from the report of the perceived basic science here into the translation of the science. i've read quite extensively and i think the view is widely shared its one of the most generously treated areas by the fda. there is a 2010 report from the biomarker consensus collective i think was the title of which i believe included representatives from the fda and the private industry and simply said make this exact same point in considerable detail and no uncertain language that somehow or another the basic research was moving far ahead of our ability to transform it into working drugs. ..

that led very quickly to vaccines and antibiotics. we are putting in genome sequencing curves. we can see the proteins. we are really good at seeing what's down there and this is very useful because drugs do their thing at the molecular level. that is where the action begins. doesn't end there but if you could see it all that puts you in a completely different world from the one that has been known for most of medical history. we are getting extremely good if you show a biochemist at target today and say one-on-one if i could get my job to this target

kill it and destroy it they are really very good at that area this is not the end of the story so nobody should interrupt me at that we have got structure-based drug design which we won two nobel prizes for in 1988. we have got my antibodies that are targeted drugs and we are doing or mark about things now with live cell therapies and stem cells which can be drawn from a patient and genetically engineered and returned to the patient in a sibling group of therapies using white blood cells that extract from the patient and engineered for example to attack cancers with stunning results coming. this is the newest area and we look at how the people involved are engineering the cells as logical as writing software for cell phone. they take a gene and turning one often sticking it in the cell and dispatching it to do something.

it's the sheer nimble genius of this is just stunning and the other two areas i am fairly confident we do still lead the world and will be interesting to see whether these live cell therapies we keep up. a lot of other countries are much more willing to break every regulatory rule and move forward. we will see if that ends up in disaster or try and sooner or later. i think this new technology and visibility to see everything poses has presented us with a scientific policy. the scientific part was alluded to us by dr. gray. we find they are made -- we messier and complex when it was one bacterium and to go after with tetracycline and mission accomplished. we have these complex webs. there are large numbers of variations in the structure of molecules that propel diseases. the networks are complicated. they are redundant and that we are increasingly seeing the like

in the of slow-moving diseases. we confronted the first of the avanda of hiv in the mid-80s and the mid-1990s. they are complex and they are slow. this means that at least using traditional fda trials the process is slow at the complex. we often don't get the right results from trials unless we are thinking in these terms. the economics of drug development has been cited and a large portion essential fundin funding -- centered on the time process. you have that click -- clock ticking and the clock is ticking on your back. we have some extension after that. so that is the scientific challenge. you can see all this stuff and modulate something that you tried to work out what to modulate. i have to say censure passage of the days of the best i can tell him there may be dreaming but i think nih has launched a series of projects beginning around

2013. actually they go back earlier than that. they have the 1000 genomes project and since then one interesting study going back to clinical trials of the past that have failed and going to work out why they failed. they are plucking out the exceptional responders and this is terrific stuff. the regret is we weren't doing it all along. i'm not exactly sure why we weren't but anyway they are -- the program is absolutely a paradigm of what we should be doing to get to the science. now we move into the biomarkers target selection very important but also prognostic biomarkers and those are extremely important because they feed directly or ought to feed directly into the fda approval process. the accelerated approval rules which we all know about or we ought to invented and actually the late 1980s but actually caught attitude the code of federal regulations and early

1990s lets you regulate not conditional and not for forever give accelerator approval on the basis of surrogate endpoints for immediate and points in that hinges on prognostic biomarkers of one kind or another. the nih to my mind is doing exactly what up to be done and i don't know if this is a reaction or not but they are doing it exactly right. the most important thing we can do and believe to seriously accelerate drug approval and lower the cost is to -- the fda is at the table by the way for a number of these initiatives. i don't know if it's everyone of them them but the fda is at the table and giving input. others can tell me how detailed it is. i think we should have one process. that makes these calls and not do them multiple times with different agencies. yes we want to bring in the other stakeholders. if we could get that process moving wealthy would substantially accelerate the approval of drugs. we would lower the cost and

faceless challenge and competition from other countries that are willing to do this by possibly less well than us and willing to cut a lot of corners. >> thank you all and that may just say and i will let my colleagues asked some questions here. i almost feel as though you are just bound to speak on the questions that we ask. you have been waiting. you are excited. your enthusiastic and it's about time someone actually allowed you to answer some of the questions. even though we asked just one of seems like many of you have covered really all for so i am not going to take the next three hours to do the next three questions but instead i know a number of my colleagues here and we would like to ask him questions. what we will do us some of the band and finish somewhere between 5:00 and 5:30 or even before 5:00.

we will see where it takes but henry will be informal here. no one will be limited to time but if we can limit ourselves to a question or two and then let you all respond maybe not each and every one of you but help us. >> thank you for all of your presentations today. it seems that the basic is the research come investment of basic research that is needed for the whole process to work and if you ask anybody in the public what they think the best investment of government funds is if at the national institutes of health. yet we have heard the term that we have to have more stable and predictable levels of funding. i'm concerned that we have safety caps dr. collins in place for non-defense discretionary by as much as cutting as much as 15% in 2016 under the ryan budget which 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 that did happen. the nih was severely affected by that. so i'm interested in the reliable and adequate funding levels for the nih. that's my question to you and for dr. shuren 11 there seems to be an underlying assumption that all the time it takes to get drugs approved, it is a problem at the fda and i want to know if that's accurate or could you give us a 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 do both respond. >> thank you for that question mr. waxman. there are various ways of

assessing what is the state of health of biomedical research funded by nih. one can look at the trajectory of the last 40 or 50 years and you can see in fact for most of that time nih support was pursuing a fairly stable trajectory of inflation plus 4%. that was true from 1970 until 1998. in 1998 there was this wonderful development at nih which many of you around the table had a role in which is enormously beneficial for ramping up the ability to do things faster and in a more risk-taking way. which was bipartisan. >> it was the waxman upton bill in the house. >> i should've been more explicit when i sat around the table. >> it was actually upton waxman. [laughter] >> i think the expectation of everybody was it that was going to sort of set a new base but in

fact what happened was at the end of that doubling essentially nih's budget flat and often it remains so with inflation gradually eating away at the purchasing power. the sequestered and immeasurably to the pain of that experience cutting $1.5 billion halfway through fy13 which has not been fully recovered and at by 14. we are still below where we were in 12th. the consequence of that in terms of purchasing power that nih lost more than 20%. if you look at the nih budget as a% of gdp another way of looking at it. we have lost that same% relative to 2003. if you look at what other countries are doing it's been exactly the opposite direction since 2003. they have been growing as we have been shrinking and the consequences of that are quite clear. in terms of what the average investigative experiences. >> dr. shuren 11 my colleagues are going to start to hate me.

you take too long i'm the only one that gets asked a question. we don't have adequate funding and we have a budget that calls for severe decreases. do you think that makes sense and does anybody here think it makes sense for trying to find cures? this is one of the most important investments the country can make. every other country sees that to be the case. >> thank you so much because i think that's an important point and the way it's operating is to put in place. dr. woodcock it takes to long to get these drugs approved. it's about fda's fault? >> there are standards enforced by the fda in most parts of the development.

after druggist discovered companies have to figure out whether it's worth investing in so they do studies to see whether they think is going to work before they put it in to people. that's a good business decision. that's not driven by the fda but there are also toxicology studies that have to be done in animals to make sure it's safe enough to put into those first human volunteers. there are few people and view either be's who say that you shouldn't do that actually if you are going to be one of those volunteers. we have a very good record of many thousands of commercial i ind's that are very safe. there are side effects that they can be managed and so that's phase. the clinical phase is the most expensive and that is where the most arguments are. it costs a lot per patient in the united states to do this and those studies are done to meet the fda's standards to ensure a statutorily embodied to show the drug is safe enough and it has

effectiveness that works. at the end of the day at the benefit risk analysis. if you have a terrible disease you don't have to be a safe basic lan so forth. the review time is really just a little blip. we approved drugs as fast as sometimes 45 days or perhaps three months. the pdufa goals that you passed in the desai specified timeframes that are a year or under for the fda review of all that information. >> thanks. >> i would like to address the issue of fda and if it's too fast or too slow. the answer is it's just about right. about three years ago i went to the largest cancer meetings in the world and companies were telling us everything is faster in europe. this is for cancer and we are really worried. we embarked on a study because

in fact drug patients were having access to drugs in europe faster than the united states and this was an issue. our research assured as a matter of fact to show 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 better. what really is important is that these treatments have to work for patients. so getting out faster a drug that doesn't work that has no benefit is not benefit for a patient or for a company. yes we have a lot of issues with their clinical trial system and issues about how we design trials but at the end of the day when a patient wants is a treatment that works for them. >> i want to thank and honor what to call you guys, the

panel, advisers participants whatever. patriots for your opening remarks. i don't think it's a surprise dr. collins wants a more stable and increased funding system. it would be news if he didn't. i don't think it's a surprise that the ranking democrat on the committee agrees with him. that is not a newsflash. and everybody else. but having said that there are financial constraints that we have to operate under. i was gratified that there were a lot of comments by the folks that have been speaking about regulatory reform, unfunded mandates. dr. woodcock mentioned something called an uncle trial network which i think has merit. dr. von eschenbach talked about reducing risk within the system.

dr. shuren asked about improving electronic health records. ms. despres come is that the way to save? talk about something called a clinical registry. mr. gray was talking about greater investment in what he called analytical technology. and dr. huber talk about process reform. so we have lots of ideas. one thing that wasn't mentioned mr. chairman that i've liked -- would like dr. woodcock to comment on, i have a number of small medical device manufacturers in my district who are coming up and getting approved by the fda, new devices and medical applications where they do interactive with their electronic devices with their doctor in therapy and also in diagnoses.

fda approves them but the cms, medicare won't give them a code or doesn't know where to fit within the current system. how do we handle something like that where it's a technology or a therapy that has been approved and they know will help people. we get down in bureaucracy and trying to determine how to reimburse through medicare. >> you may wish to ask dr. sub -- this doctor shuren that. he is head of the device center. >> i'm willing to listen to you. >> dr. woodcock throws me under the bus so i can throw them under the bus is. what a great country we have. i apologize by dancing a little bit around it because i do have my colleagues at cms that you are facing an important point which is if i'm a developer of the technology what i need is the ability not just to get it

to market it to be reimbursed. as a public-health official position what i care but also as patient access. no patients don't have real access in many cases unless there is also reimbursement, the technology particularly if we deal with more expensive technologies or an expensive procedure. many are disenfranchised and don't have that money for it. we need to think about it. their circumstances where we can provide a more streamlined pathway and more guarantees if stances where itactually threw should be paid for or there is for or there's a tie-in where you are paid for and there is that additional data collection. cms will sometimes do that. there's something called coverage with evidence development where they say we will take it as it comes but there's additional information. at least you are getting reimbursed for it. i think that is a critical area to be looking at how we can provide that greater payment predictability. >> thank you mr. chairman.

>> thank you. let me thank chairman upton and congressman to get for putting this together. i think it's very beneficial and everyone who has participated. i just want to ask a general question. i guess mr. dr. collins or any one really can answer it. i constantly hear and listen away paper and dr. collins mentioned it. and mr. waxman mentioned about funding. obviously there is not as much money available here to nih as it used to be. you mentioned a researcher that went to china because he thought they were better opportunities fair and mr. gray mentioned the technical infrastructure you know that we are falling behind. i'm just trying to get a handle on we are constantly told that

we are falling behind in the sense that 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? we used china's hard for me to figure out that really means because china is a communist state run. here we have money for research and research being done by private companies. states kicking in and universities kicking in. is the advantage that china has strictly that they are national government or spending more money? is that the fact that they organize this in a dictatorial fashion. i'm just trying to get, when we say will we continue is that this question of federal dollars or does it go beyond that in the sense that it's deals with the infrastructure?

i know it's kind of a general question. i hear this all the time and is so difficult to see that comparison with china because it's such a different political structure. >> china is not the only example but to answer your question about why they are able to do it so quickly clearly they have a decision process that is much more top down and they basically can make such a decision and implement it quickly. they have been increasing their support by 22% per year for several years. it will outstrip national dollars. it's their goal and they been clear about that. it's not just china that is read our playbook. singapore south korea brazil. goodness look at europe which has not had an easy economic time either.

they protected that and continue to increase support even at a time are other things -- like they have looked at america's story and trying to learn from that. my greatest concern is not the way in which this is affecting anything in the way of and bolts or bricks and mortar. it's the most important resource we have. the biomedical research committee particularly this next generation. they are under serious threat. there has been 60 or 70% of their time spent writing grants because their success rate has dropped to one out of six of most of the time it's another failure. they are getting discouraged and increasingly thinking less risks because risky science doesn't seem like it's got much of a chance. we are coming up with all sorts of ways to try to stimulate their innovative instincts that they are lighting up in that regard. if you really want to see her

future we have to have that resource supported. that's what wakes me up at night. we have half the great science on the table and this next generation of scientists want to do which generally would have been supported supported in the past and now it's gone. >> you answered the question dead center and to my way of thinking it's not about just investment are funding. it's about creating opportunity. that's a much larger set of issues and it has a lot to do with some of the other things from changing the infrastructure for example. but at the core of it is the point that mr. leff made. everyone is recognized this explosion in science and technology is changing not just medicine but all the rest of the sciences and in fact it's not just about nih or just about 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 science of nanotechnology. for example computational science is as equally important to this opportunity for this country. so we should be paying attention even to the agencies and the government beyond the nih such as the department of energy and our national laboratories which are in arm wrestling important part of this future frontier. i think what the committee has laid before us is the challenge to get beyond just the question of are we funding but more importantly are we creating an opportunity in changing the ecosystem in a way that really will keep us at the forefront? >> to really build on that, creativity happens in large part

to universities where a job of somebody like -- is to facilitate serendipity because oftentimes the innovations are at the boundaries between traditional disciplines. so one of the things we are doing is how do we constantly bring engineering and the life sciences together? we have changed the biomedical engineering department from being solely in the college of engineering now to being joint with a medical school and the college of engineering. because it's that human, human interaction where the creativity occurs. there are critical facilitators, finances, technology. much of the breakthroughs in medicine in our understanding our technology dependent, and 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 it dances in our understanding not just of disease but also of health, which actually is the ultimate goal for where we are. so it's looking at that total ecosystem, not just one piece, that's really critical to maintain our creative advantage and the lead we have had for arguably the last century. >> just to continue this dialogue i wanted to come back to one of the consequences of the decline in real spending dollars that has happened over the last few years. one of the things that has happened is that science has become hypercompetitive. dr. collins mentioned i think that people are spending a large amount of their time with grants. they are spending a large amount

of their time writing nonproductive grants. these are grants that are not going to be funded so you have basically taken the thinking time away from the scientific community that can be used to innovate and are forcing the community to devote back that to just pushing paper out. that is really a killer. one of the reasons we are calling for stable funding maybe doesn't have to be increased, vastly increased funding that strategies to deploy funding that insurers that our best scientists, are most productive scientists are spending their time thinking about science and not spending their time writing grants. if we could inspire to have long-term funding goals in this country perhaps with an achievable granular objective, then i think people would feel confident in saying okay i can try some new things. i won't be out of a job because somehow i took on a risk and

fail. so we have to enable ourselves to break out of this hypercompetitive cycle and get back to thinking about science. we can do this by defining where we are going and what money it's going to take to get there and identifying the science that we need to get us there. >> just to echo the point about it not just in china i also want to punch away this idea that even the european community has banded together because they see this is such an amazing growth opportunity. a study of the consortia phenomenon. it's element of what we are talking about here. it's how the different entities come together to solve problems. their loss of consortium to happen in the united states. some arguments are by the administration of the nih or they have participation that there are a whole heck of a lot of them in europe right now. the numbers of these consortia are going up. so i look at it as a little bit

less of is the u.s. declining and a little bit more up as everybody else 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 ingenuity, the innovation, what took place at the beginning of the biotech revolution, there are unique attributes i think in terms of how the american scientific enterprise operates that we don't want to lose for a whole lot of reasons. for what it has produced let -- thus far and what we expected to produce printed think the u.s. is still looked at as that place for everyone to model after. they are modeling after the gold standard of the food and drug administration. they are modeling after the nih and other science agencies that dr. von eschenbach articulated but the question in the challenge in front of you while is what's that model going to look like in 10 years, in 15 years?

we are talking about bringing in different disciplines, bringing in a collaborative spirit, teen science. i have an 11-year-old daughter who's on the team tomorrow to simulate the westward expansion expansion so we'll be hiking five miles with their little red wagon through the fields in virginia. she had 11 understands teamwork and an entirely different way than i think any of us for school then. i think if we want to entice kids like that to go into science careers there has to be the sense of their something for them to step into. i think that if we lose that opportunity, all of this work going on whether it's china, europe, lots of other places, it's just going to keep going because all those countries have prioritized this and said if this is something we need to be paying attention to. >> before i let sarah get the last word and then we will move to mr. burgess, dean kamen will be one that we rely on and of

course that is a focus in life. sarah. >> so thinking of this as an ecosystem from our standpoint is the will right way to look at it and you talked about kind of a continuum of discovery development and delivery. it raises the question of how you were going to make sure it's actually a feedback loop. we have been hearing about the need to invest in research and as critical but there's also the need to ensure that we have the tools to collect the data and disseminate the data. i spoke about a trial in sweden by american standards, very efficient. a lot more expensive and they were able to do that in large part because they had the interoperable electronic health records. so i should think about the kinds of what needs attention in the united states so 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 world it's a different game. what happens in the real world experience can inform the next generation of products. so the data collection is a critical component of this as well. >> thank you mr. chairman and thank you for leading this group. i think it's extremely important that we have this discussion. i have an observation that i have a question to some on the panel. the observation would be it was in this very room the last thing we did in calendar year 2006 was reauthorized national institute of health. we reauthorized the database level of $31 billion to increase by 5% every year. we were eviscerated at the time because that number was woefully

small and no subsequent session of congress republican democrat or divided government actually came anywhere close to 5%. it has been as dr. collins pointed out much less than that so perhaps we should consider the reauthorization that is a done some years ago and perhaps we should consider reauthorizing nih. dr. collins i would just point out to you that all the buildings on your fine campus and i've been there many times but they are only appropriators because the operators who are your friends here not the appropriators. the question i want to shift gears for a little bit and then i have to go another hearing but we hear a lot these days about the right to try for people who have serious or terminal illnesses who they have found there's an availability of a clinical trial they can get on. can any of you address address that? i address that? i think the cold war institute is doing work on this and is one of those things that pulls on

the heartstrings of constituents.com. can we talk about about that a little bit? >> i can as far as pharmaceuticals. generally speaking we have a very -- policy about people getting onto what people call compassionate use but basically treatment protocols. if they can't get into a trial for an investigational drug. i think i testified we have thousands of requests and we only turned down two and a year so we get about that number of requests each year. however companies for a variety of reasons sometimes are not willing to provide drugs under investigational, investigational drugs to patients under treatment protocols. they have a variety of reasons, shortage. they often say they are concerned that a side effect will be found out in these patients that actually wasn't in the clinical trials and a later

development program. it has never happened but that is often cited as a fear. then there is a 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 are unable to obtain drugs under treatment protocols from a company. that does happen. >> this is a roundtable about solutions. is there anything we can do as far as preventing companies with the certainty that they would need four or the liability protection that they might need? >> i think you could ask the companies. i think from the fda standpoint we would be very reluctant to agree to pass something since we can't look at the treatment experience because i would be unfair to the other patients. >> so this issue of access or

expanded access to something we are actually looking at. we are working with brookings, cancer research in brookings and we are working on it. they're actually interesting proposals on how we may approach that problem and actually not only get access to the patients that get data that support and to what's what's missing often limited compassionate use as we have no data on exactly what happened. there is a very unique, very interesting proposal. we are looking at it in our meeting in november and it will be interesting to see if there is going to be a legislative need to do something odd. but it is a very legitimate issue. at the end of the day we really need to get data. we want to know what happens if the patient did have access and if it didn't work why so captioned it is going to be incredibly played in. this is something people are thinking about. >> trying to alternate between republicans and democrats.

let me go to kathy and then joe and then leonard. >> bank chairman upton and -- and thank everyone here on this panel. thank you for devoting your professional careers to improving the lives of american families. i do think it's an exciting time for biomedical research in the u.s. and across the globe. i have two questions and two points mainly for dr. collins but mainly for anybody else who wants a comment. many of you have already stated how much you value our scientists and researchers. it's just fantastic to me a lot of these young talented researchers, for example at home at the moffitt center in tampa and all other research universities across the country. they are not shy in talking to policymakers about the fear they

have for the future. they are passionate. they believe in what they are doing. they are making progress. they see the technological advancements that they are very fearful that based on inconsistencies and uncertainty and funding levels that this isn't the career for them. i hate to hear that from them. i think congress right now has a very important responsibility to make a newfound commitment to the nih and to biomedical research in america. the budget this year, i offered an amendment in the budget committee to move the nih funding from discretionary to mandatory. i think this is something that we should discuss. maybe we do it with dr. gray's suggestion that they are certain benchmarks that have to be met for certain requirements. if you could discuss that or

there are other budgetary solutions. number two we don't have all of the resources in the world to spend. are you confident that monies are going to the right place in biomedical research? for example when you look at much of the data of the aging population would seem like we have got to do a whole lot more in brain research and alzheimer's. when you look at the threats to the health of america, boy that is staring us right in the face. number one shifting to mandatory funding into cop are we making the investments in the right place? >> i appreciate the question and in fact i appreciate this whole panel discussion. again i'm not a budget guy so i'm not going to be a clever responded to this notion of shifting their dollars from one place to another but what matters for biomedical researchers to find a path forward that has this predictable stability and again we have lost ground and many to

catch up but then to have a competence and so we are not going going to be on a rollercoaster ride of feast and famine which is the most destructive thing you can do to the research enterprise. if a mandatory source of funds with a way to achieve that than i would stand up and cheer but i'm not sophisticated enough to know what those options might look like to get you there because i understand how complicated it can be to achieve that kind of special mandatory funding stream. with regard to how we are using the dollars we have your right to ask that question. am i is glad question. i'm iceclad to that question asked. we have to have the ability to respond to any query about whether the dollars that are being allocated by congress are being widely -- wisely use. we are looking every day at ways to achieve new efficiencies by partnering with other organizations like they have with the accelerating medicines partnership and we are working with darpa with fda on this toxicity chip. working on the brain initiative, new efforts which is going to be

focused between nih and nsf and darpa and a number of private industries and philanthropies trying to make sure that these kinds of interdisciplinary efforts to jim woolliscroft was talking about her caps-on two. we are asking whether, we claim it's the best in the world. are we really sure about that and are we sure that peer-reviewed system is reflecting the scientific opportunities of 2014 and not some earlier stage and do we need to rethink about the way in which this peer review panels are established as far as what disciplines they represent? we have the tools now with various kinds of text mining to look into what's going on in science that they might want to made adjustments for. we are pretty bullish about that. another thing we are doing it again this reflects a something that joe gray said is to make sure -- see if there are opportunities to put more of our grant funding more than we do into programs we give stable

support to investigators with the expectation that once you have one of those awards you'll actually get that down to a science and not spend all of your time writing and rewriting it getting discouraged with rejections. this is a program at nih called pioneer awards have started this. it's very successful. we are not proposing to expand that beyond the common into other parts of nih and to do some pilot experiments. i think there will be a possible way to deal with some of this terrible waste of peoples time when they are spending so much of their time in a productive grant writing and just getting frustrated. not that will really suffice. there's no magic you can snap your fingers and say now it's fine when you've lost 25% after purchasing power and people see the current circumstances. you are not going to be able to fix that without getting on that stable trajectory so whatever we can do to achieve that that's got to be job number one. >> i think one of the most important point to this effort is the fact that you are committed to a long-term look at

the problem. i think there are some significant issues that need to be addressed beyond just the stable 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 monies that could be repatriated but done in a way directly related to funding what created those opportunities for the industry in the first place. the other thing that is quite difficult issue to take on but i think would be an important consideration in how we are spending the dollars is to really look at indirect costs and the issue of funding infrastructure versus funding ideas because one needs to actually roll up our sleeves and begin to take a deep look at the problem if we are 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 money. the mechanism also of where we getting the money and how were we spending the money? >> thank you mr. chairman. and for this new initiative -- is that on? okay. this is just the first of the three-year process and the first step is discovery. you are the first briefing. we will have other briefings and hearings as a health subcommittee and we will translate the information you have given us today to other members who are not here so that all of our members can participate in this. and if i were doing another t i would add diagnosis. early medical diagnoses of the disease because the faster you can get the care to the disease that better the least costly.

i have a couple of questions. dr. woodcock you mentioned something called clinical trial network. what do i do not buy any barriers that you see and do you need legislation to set this up or what are the barriers now that prevent us from having clinical trial networks? how can we use our technology platforms to accelerate the cures to bring researchers and patients and innovators together a? a couple of questions. >> there are related. i think a lot of the barriers have been cultural and we have done things in the past and this is how we have always done them so this is how we will do them. the availability of electronic health record, the availability to reach out to clinicians all around the country, to join them together and then to two trials

utilizing that infrastructure and training could provide all sorts of networks. it could very rapidly answer questions which is really what device development is about. we need to ask questions. does does this work? as if they have? you can rapidly answer those questions and that those products into patients and test them and get the answers out. the network is simply a group of trained people who are trained and are trained in writing of the protocols in place to evaluate something. maybe many things and registries are where you identify patients and then you follow them. you can to trials in that registry. you you can randomize people in that registry 212 manure and other in and follow them. these things are all linked together. this type of infrastructure doesn't have too much in the united states except we are driven by patient groups who have gotten us set up to advance their disease. i think it's a tremendous

opportunity. what alan was talking about is driven by the cancer trial and error couple of others going on. that trial is going to start out with probably five different investigational agents that can be evaluated so every single person with that type of lung cancer if they are screened for that trial. >> would clinical trial networks work with medical devices? >> it would. actually we bachan 2011 talked about how you might better identify centers of -- centers and clinical trials and should there be a voluntary certification. then that is open to innovators to say we know the centers are good at these areas. devices wouldn't be one-size-fits-all. some institutions might be good at surgery but that can help and i think not only in the data collection for something going

prior to marketing to think about it, those networks in place you might be able to rely more on data collection after it goes to market because those networks are there. they will be using those technologies. they are our dig hearing to gather that information. we would love to see something like that. >> i just want to respond to the clinical trial that mr. woodcock reference that will start at the end of the month. first of all fda is at the table not only with drugs that cdr h.. we are using nextgen sequencing on that so we are using a very new and very interesting platform. nih is at the table. it's going to be done at every cancer center in every community in the united states amid deep in canada and is public-private. we will learn very quickly first of all medication will be refused access to a drug. everyone will be screened, 100%

of everyone on the trial will be screened with nextgen sequencing. if we don't have the biomarker for that patient they will go to immunotherapy. it's much cheaper than doing a normal chronicle trial and companies have been eager to join it. it is truly public-private. we do have cd rh drugs and an r. h. at the table with this. >> thank you mr. chairman and this is among the most interesting discussions that i have been involved with in this room and i want to thank both of you for it. there will be a test. that is why i have chosen to stay. [laughter] i favor stable funding dr. collins. you and i have discussed this and i think the rollercoaster has been unfortunate and the

rollercoaster should be confined to disney world or coney island. however from my perspective i'm perfectly willing to discuss in a cordial fashion how we move forward on budgets long-term. perhaps we couldn't be as generous as we would be optimal but of course we need a partner and the senate that passes the budget and i hope that we have a partner moving forward and we can have an honest discussion as to how best to fund nih appropriately in the national interest and perhaps one of the ideas that has come out of this discussion this afternoon and dr. burgess raced it is somehow reauthorizing nih. i think that is a very interesting idea. i am all for naming a building for dr. burgess. now we had a discussion about

china. think its informative but i don't think it's to sponsors. to the panel in general as i understand china is quite different from the system. here we rely on governmental funding. we rely on the nonprofit sector so it would be represented by a great university university and a great charitable trust and the rely on the for-profit sector including the drug companies many of which are headquartered in that district i serve. and we rely on venture capitalists so i would ask mr. leff on that for-profit sector where should we be doing recognizing the other greater responsibility with more stable funding for the nih, recognizing regarding various universities we need to make the system better so that in fact only one

in six grants was awarded should be change. we have to make a system better. regarding the for-profit sector which is an indispensable part of a larger whole, what should we be doing, repatriating profits from abroad but what should we be doing and encouraging for-profit sector which is very different after all from china's. >> this is a great question and thank you for that question. we have talked a lot about the investment in nih and this country putting numbers on its investment about $30 billion a year in nih funding and for medical research but private enterprise by the companies and investors put about $80 billion a year into r&d. >> precisely. >> i think it's obvious to everyone here that this 30,000,000,080,000,000,000 are deeply intertwined and in order

for the public-private sector to make those investments there has to be rum until coming out of the basic research. we have talked a lot about that. in order for that investment in basic research to create new cares there has to be that investment in private sector r&d to develop those signed to pick discoveries into -- patients. talking about that it's really important and it also has the benefit of not being the zero-sum game that we have to come up with government funding in order to solve the problem. in order to enhance that investment, that 80 billion which it's obvious that goes up, all other things being equal we would get more cures and treatments coming up. >> and we will be saving more lives. not only here but across the globe in putting china. >> absolutely increasing more jobs in this country as well as saving more lives. of course the opposite will be

true so i would encourage this committee as it thinks about that issue to treat an economic problem with economic solutions. that is to say the decision that investors make and companies make about whether to fund biomedical r&d and which projects to fund an homage monday to to put into it is fundamentally economic. we all want to do it to save lives but ultimately investors will put their capital where the returns are. investment in a medical r&d is less attractive than investing in social networks are new electronic devices or natural resources or whatever. we believe the capital would go elsewhere so as it's an economic equation. i think when you look at the recent history of policies coming out both congress and this committee and elsewhere you can see that impact. you can see the kinds of things that have been done from a

public policy point of view that up and danced -- advance biomedical r&d and i would point to the orphan drug act is a great example where was recognized that r&d credits for investing in orphan drugs as well as exclusivity for developing orphan drugs ensures that there is a better economic equation for making those investments. we have seen dramatic increases in huge productivity of investment in orphan drugs as a great example. we see already some of the fruits of the game act which was part of the fda safety innovation act in 2012 where exclusivity was provided for antibiotics that address the resistant infections and we have seen a resurgence of private investment in an extremely important area. those are just 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 most important new therapies and it really was fda who came forward and said we need breakthrough designation as a tool to do what we all want to do here. that is worked out extraordinarily well in lowering the time and cost of developing therapies and attracting investment. more recently the fda has come forward and said we could really use a new tool which is sometimes called the special medical use path also sometimes called the el paso limited antibiotic legislation act. that is a great step forward that is providing the consensus on the will of congress and the will of this country to give fda

the tools it needs to apply the appropriate degree of flexibility to accelerate development of certain kinds of drugs. i believe and many believe we have to try harder that this tool should apply not just to do an about expect any therapies of any category work and help accelerate the important innovations. that is one example. certainly when we talk about tax reform and we talk about economic incentives, these things matter and they matter to the biomedical industry and they matter to investment. the one other dimension to dimension here is how we pay for these new and innovative therapies may come to market. this is a really difficult issue because we all know expensive therapies costs money. they cost the budget money and they create challenges for access to the patient to need the drugs.

at the same time if we are not prepared as a society to pay for the real and the nations that provide the real value and real ckiwers to patients that investors won't make those investments. providing i think a dialogue which recognizes the value of the patient and recognizes that it's appropriate for new therapies to have a period of exclusivity in which they can recoup their investment for the ones that provide real value to patients and really save lives we have to do everything we possibly can to figure out how to accelerate time-to-market and then have a consensus about the way in which we pay for them in a manner that creates a cycle of further prime investment. >> thank you. let me say this is an issue that deeply interesting. i hope that we can work collaboratively in a bipartisan capacity on what is a three-legged stool in this

country. and mr. chairman and to the others who are leaders in this i think we will have to as well work with the ways & means committee because there is a fundamental tax policy involved as well as the wonderful work we do on this committee. thank you mr. chairman. >> thank you. one quick thing i want to say too is my understanding that dave camp will shoot through r&d permanent tax and i expect that on the floor soon. i will let my colleague in crime here say a few things that i will close. it shouldn't be too much longer. >> two good things on the economic. the front end of the funding conundrum the work of dr. andrew lowe has achieved star status in terms of the creation of a cancer make upon any can insert any disease dadar pathway. the idea is basically how do we

aggregate risk in terms of funding that can go to companies that are doing development of products? i think that is certainly one that should be looked at in terms of the themes that he has articulated. then on the other side that was just raised by jonathan this issue of how are you going to pay. the global conference competin competing -- convened a panel discussion on this that was extremely insightful. if you build it well they pay tax now we have to worry about what we do with this innovation? kimmie afford it and afford it and the larger health care system? i think there is an exciting opportunity here to post the circle of it. if we can prevent disease and please let's prevent it and boy do we have a lot of prevention work that needs to happen. if we need cares let's figure out how to fix that part of the system and get the funding so we can do that and we have a robust economy. but boy do we have to pay attention to that other and too which is how are we valuing do we valuing dissemination and how are we as a society going to sort of be able to take in what's coming throh?