prime time, ronald rockbottom talks about nazi occupied paris in 1902. then an account of the war in afghanistan as told through the lives of throw afghans. and walter isaacson on his new book about the entrepreneurs and creators of the internet. next, house energy and commerce hearing with the heads of nih and fda as well as neuro surgeons and drug developers. they testified on medical cures and innovation. and challenges of conducting research with tightening budgets. this event took plis place at the farm and home center in lancaster, pennsylvania.ace at farm and home center in lancaster, pennsylvania.

good afternoon and welcome. i'm councilman joe pitts. welcome to the 16th congressional district of pennsylvania. for your information, this meeting will be streamed live. c-span is here. so you might want to keep that in mind. but i'd like to welcome everyone here, especially our distinguished panelists. and i have a short introductory comment to set the stage. through the 21st century initiative, congress is taking a comprehensive look at the full arc of accelerating cures. from the discovery of clues and basic science, to streamlining the drug and device development process, to unleashing the power of medicine and social media the at treatment delivery phase. and this cycle of discovery and

development and delivery is what saves lives. and we want this cycle to work effectively and efficiently so there's no gap between the 21st century science and the washington regulatory process. this work will be a collaborative effort which is why we are first listening to experts and involved parties. so that together we can achieve our common goal, accel ratting the pace of cures and keeping america at forefront of medicine and discovery. as chairman of the health subcommittee, we've conducted six hearings and three roundtables. this is the first of the roundtables in the country. and there will be a series in the next month or so of roundtables across the country. but we're eager to hear from interested stake holders. and that's why i'm hosting today's event. i want to welcome all of our

distinguished members. our panelists. our attendees to the 21st century roundtable. the discussion will showcase the innovative work going on in pennsylvania and elsewhere, to help us understand the ideas that can understand the pace for cures for patience. given the advances in molecular medicine, we will examine how the fda is using the existing pathways and if there are new ways to improve the process of developing and approving new drugs and medical devices. advances and our understanding of disease at the genetic level can also help reduce the cost and quicken the pace of clinical trials while also giving patience faster access to the treatments and trials. capturing the impact of new drugs and devices provides new data to share with researchers, care givers and patients, information that can serve to fine tune the discovery process

so treatments can adapt and help even more people in the future. of course, the most important goal is generating better health and saving lives. and achieving these goals will only occur through greater team work and collaboration among nih, fda, private industry, academics, medical professionals, philanthropists, patients and advocates. and it is unclear if the management and organizational structures to achieve this broad and effective team work are in place today or can be improved. but america must maintain its leadership role. in this effort. and this effort accelerate tours will put the nation and our researchers on trajectory to make the predominant diseases of today a thing of the past and provide hope for all. the nish ator of this whole program is the chairman of our energy and commerce committee.

and i'm very pleased to ask our distinguished chairman. chairman upton from michigan, for any remarks he would like to make. then i'd like to again thank him for taking the time to visit lancaster, pennsylvania. mr. chairman? >> thank you, mr. chairman. and i just want to say from the get-go that i certainly appreciate your leadership on this effort. but i also want to say that it is truly bipartisan. something that we have worked on for nearly a year now. we have formally unveiled it this last spring. but in fact we tried to make it, and it'll be boy partisan, every step of the way. this 21st century cures initiative is in fact going to stabilize. it is going to impact every family, not only in this country but perhaps around the world. it is going to keep our country the leader in medical innovations. accelerate the discovery,

development and delivery cycle of new cures and treatments for all patients. panelists are inaugural roundtable way back when understood the starkness of the challenge. voting that there are 7,000 known diseases. but we only have treatments for about 500 of them. and that's just unacceptable. we can and we must do better, and we will. few initiatives have it kind of direct impact on american lives. or as we think about our family members or close friends that have been impacted by some awful disease. the past number of weeks our colleagues have taken this conversation back home. soliciting comments from patients, from families, researchers, advocates and thought leaders all across the country. chairman pitts, your committee held six hearings around 21st century cures. but we've add number of roundtable discussions as well. not only in washington but this is the first stop as we travel around the country.

we are going to be holding additional hearings when congress returns next month and your efforts tireless on this initiative coupled with bipartisan work on medicare have helped millions of americans, obviously here in the 16th district. pennsylvania. this subcommittee, the one you chair is responsible for enacting nearly two dozen public health measures into law this congress. tremendous accomplishment, testament, to your leadership. as i said before when we looked back at the 113th congress, it is going to remember perhaps as the public health congress and i'm pleased to be here not only with you but ranking member of the subcommittee and vice chairman burgess as we continue to work together. closing i want to thank those folks who have contributed to the 21st century cures initiative. we have received invaluable input from patients, government leaders, such as dr. collins and dr. hamburg who are both here today.

innovators and researchers, especially everyday americans where folks with weigh in on the website on cures at mail.house.gov. we look forward to having a number of these discussions in the weeks ahead. and our goal is to move -- begin to move legislation early next year and get her done. and i yield back. thank you. >> thank you for those statements. i'm pleased to introduce the ranking member of the committee. he and i have worked tirelessly, i think 22 or 24 bills now sign need law. so far this year. and he deserves a lot of credit. so frank, you're welcome to make any opening statements you like. oh, yeah. sorry. >> thank you. thank you, chairman pitts.

i think you know that many times i mentioned to you that i wanted to visit -- i had to watch how i said it. because i was told i wasn't pronouncing it right. is it lancaster? i know i'm not pronouncing it right. but i wanted to come and visit so this provided me the opportunity to come to your beautiful district. and i appreciate all that you've done working on a bipartisan basis to, as you say, enact a number, quite impressive list, of bills and accomplishments that we've had in the subcommittee for the last two years during session. i also wanted to mention that again that it is the chairman upton said, the 21st century cures initiative is obviously a great initiative. it has been bipartisan and i hope we will continue to be boy partisan as we proceed. and that is very important because you don't accomplish anything in congress unless it is done on a boy partisan basis and obviously this is a -- this issue and this initiative is

very important for our country. not only in terms of a cure in diseases but also in terms of our economy and job creation. you know my district is one that has a tremendous amount of pharmaceutical companies. but for all these reasons, i'm pleased to be here mr. chairman. thank you. >> thank you, frank. now i'm very pleased to introduce my vice chairman, dr. michael burgess from dallas, texas. his medical expertise has been of significant value, as we consider public policy questions regarding health care and it is great to have you, if you would make a few comments. >> thank you. i thank you for the invitation to come up from pennsylvania. i've add number of opportunities to talk to people. both in the dallas area where i'm from as well as down in the houston area, texas medical center. last monday, i was at the invitation of manhattan institute at boston.

so this is -- this exercise has attracted a lot of attention around the country. and i think that's appropriate. for me it is great to be at the roundtables. we are hearing from people on the front lines if the development and to know what we can do to make things happen faster. because that is, after all, what the name of the blame is. thank you mr. chairman. i yield back. >> thank you, michael. next i would like to have each of our distinguished guests to take two minutes to introduce themselves. tell us who you are. a little bit of your background. and talk about what your health care does in the health care space and we will get into more discussions about what each of you thinks can be done to accelerate cures for patience. so dr. collins, a great honor to have you with us and we will begin with you fold by dr. hamburg. >> well, thank you, chairman

pitts, chairman upton, ranking member and it is great that you pulled this session together. to allow us to have this conversation and i just want to thank you and chairman upton for creating a space for these kinds of conversations to happen. i'm the director for the national institutes of health. i'm trained as a physician, as geneticist. i had the pleasure of leading the human genome program. and i'm exhilarated by what i see in terms of a potential of these advances in medical research to transform our ability to give people the opportunity to live long and healthy lives. both in terms of prevention and treatment. i'm very grateful by your

interest in asking the question of what can be done to make this wholeant price more efficient and able go faster and certainly we will have questions, and have shared some with you in other meetinges a not roundtables that i think could help nih quite a bit. that being said, i think our major concern right now is in fact the difficulty that we have in terms of providing resources to the best and brightest of our nation who are seeking to break down the barriers and they are all over the country, including here in pennsylvania. last year, we awarded $1.4 billion worth of nih funds to the state including at least one in your district. franklin martial. so the brain trust is not in bethesda, maryland. it is all over the country. and the brain trust is struggling now and we are leaving a good deal of wonderful sighs on the table because the current stress on the system, which we understand, given our federal fiscal constraints but we remain hopeful.

that the case is so strong in terms of contributions to our health and keen my that we can have a stable trajectory forward and certainly that would be a wonderful way document america's intent to continue leadership in this space. thank you. >> thanks you, dr. collins. dr. hamburg? >> thank you. i'm thankful to be in your district and with distinguished colleagues. there is huge value as is noted in getting out of washington and listening to people on the ground dealing with important issues from varying perspectives and this is a welcome opportunity to do so. and in a region that is rich with distinguished research institutions and wonderful health care facilities and systems. but very appropriate as was noted, the 21st century cures

initiative has given us a chance to come together across disciplines, across sectors, across states. and across party lines. and in toward address one of the most important pressing problems our jeeryy most important pressing problems our jeer era and promise techno to people that we serve and it matters hugely for individuals, liberties and our nation. and it matters that in terms of the health and well-being and quality of life. but it matters in terms of the health care systems as well as the health of our economy. and i think we all recognize that over a period of many years now our nation has been preeminent in the area of biomedical research and important new product, development. we remain in that important role. and i'm happy to report that fda

in fact approved more drugs more quickly than any other country in world but we can and must do better and we must do it in toward strengthen help and also to continue to support our nation's economy. high will have u job and the strong export sector that's part of this important enterprise. so it is exciting and clearly the people around this table and the visions that is embodied in 21st century cures recognizes this is all about partnership and dlab racicollaboration. there is no one entity that can advance it there is an ecosystem working together with a clearly a common set of goals which is to promote and protect public health. i'm a physician trained in internal medicine with a focus on infectious diseases. most of my career has been in public service. not what i planned when i started medical school but actually looking at world and seeing a complex array of medical problems that

intersected with other social economic legal, ethical issues. it really compelled me to step out of the academic medical care setting and into public service and public health more broadly. and i've had an approach from the beginning that reflected the importance of bringing professionals to those efforts. whatever the setting and have had the privilege of serving for both republicans and democrats. and in my various government service jobs and i'm delighted to be part of this important effort. thank you. >> thank you, dr. hamburg. mr. jim greenwood. president ceo of biotech industry organization. >> thank you mr. chairman. thank you for inviting me. . thank you all four of my former colleagues for putting this form on by way of introduction. i'm jim greenwood. i served for 12 years with chairman pitts in pennsylvania legislature and for 12 years in the united states congress and for the past 10 years, president and ceo of biowhich is the

biotechnology industry organization and we are the national trade association and we represent about 900 companies involved in drug discovery. most of them small start-up companies that are really trying to make some medical breakthroughs in curing disease. i want to compliment you for three things. first, for taking this kind of initiative, this notion of 21st century cures as a major committee project. secondly, for as i said doing it it in a completely bipartisan way. which is the only way we can succeed in this and third for doing it right in terms going out and listening to the people. too often we turn to ideological think tanks they think they have all of the answers and they don't spop this is the appropriate yit way do that. thank you for that. of course in the conversation, i hope to talk about the three rs that i think are important to advancing cures. the first of those is research.

beginning with making making sure that dr. collins nih is adequately funded but i also have an idea about a very large longitudal study that i think the committee could investigate. second is regulatory pathway. making sure that dr. hamburg's fda is adequately funded so she has tools and personnel and that means making hard choices about allowing her to have the tools to hire the right people. and which can be expensive but well worth it. but also trying to make sure that we continue to push the envelope in terms of getting new ways to accelerate the review and the approval of products so we can get them properly to patient and then the third are has do with reimbursement and that is to recognize that developing these products is a highly risky, highly expensive proposition and if we are going to attract the private investment into these companies, small medium size and large we have to have reimbursement of

the system. that adequately reward the risks and also at the same time makes certain that our product are affordable to all of the patient who need them. thank you. >> mr. clinton matthews. >> thank you for the opportunity to be with you today. i'm president and ceo of redding health system in west redding, pennsylvania. i represent the end users. the people who actually lay hands on patient. who lay hands on patients. who use the products, the drugs, that are developed in toward have the cures. we have a 735-bed hospital. 37 sites in burks county. we care for approximately 420,000 people. we are a trish areary level facility. we see 130,000 visits in our

emergency department on an annual basis. the most within the common wealth of pennsylvania. we cover stroke, cardiac, psychiatric care. we do have the procedures, the trans catheter aortic valve replacement. the thoracic and vascular aortic repair. these are for people with vascular disease. you've been in our hybrid rooms, congressman pitts, where we bring together the endo vascular so you have a surgeon and clinician working on the same patient at the same time. also, the tho rracic surgeons, working with the intervention yl cardiologist at the same time. in order that we are able to

decrease the impact on the patient through more -- through procedures that are not as invasive as others. we participate with only system within pennsylvania that participates with johns hopkins clinical research network. we are proud to be part of that. our focus is not only in caring for patients but also in educating and participating in clinical research. so, it is a pleasure to be here with you. >> thank you. >> next, dr. hanlon. vice president of regulatory affairs. >> thank you, chairman. my name is dr. bill hanlon. head of global regulatory affairs. a pharmaceutical development and commercialization services partner here in pennsylvania and new jersey as well as elsewhere in the u.s. and around the

world. we offer the broadest range of industrial solutions for our clients to help them bring innovative medicines to market sooner. more than 12,000 employees globery and delivers services in over 60 countries. we conduct clinical trials in all four phases of development. we perform preclinical research as well and we do health economics and research. in addition we operate the largest clinical trial central laboratory in the world. generating more clinical trial data than any other entity worldwide. it has its own internal innovation effort under way right now to cycle cost of clinical trial development and believe that this effort alone with our bred j and depth of pharmaceutical development and commercialization, with a knowledge position to be here

today and add to the 21st century discussion. with my tenure split equally between drug discovery and the development of new molecules. it's exciting to be here and engage in such an effort and be here in clinical trial development and it is a privilege to be here and share in this discussion. thank you. >> thank you, dr. hanlon. now to dr. marotti. lancaster regional medical center. >> thank you. members of congress, fellow panelists, ladies and gentlemen, it's truly an honor and privilege to be invited to participate in this hearing. and a humbling experience to be with the esteemed members of the health care community. by way of background, i spent 18 years of my life at yale

university. it was there that i received a bachelor of science degree in psychology, and an md. i completed training in neurological surgery at yale new haven. i did a fellowship in spine surgery at harvard. for the past 5 1/2 years i've been a private practice neuro surgeon here in lancaster. i'm part after multidisciplinary practice that includes neur ro surgery, orthopedics, neurology, pain management and physical therapy. our practice takes an integrative approach to the treatment of patients with diseases of the nervous system, spine and peripheral joint. i have had the good fortune to see health care for multiple perspectives for my career.

ivory tower, big pharma, small town country doctor. i hope to be able to contribute something valuable to the discussion today. thank you. >> thank you, doctor. next, under new bauer, chairman and ceo of new medical. >> thank you mr. chairman. i want to thank you for initiating this roundtable discussion on behalf of the industry i represent that is the medical device industry. this is an important and necessary initiative to keep the united states on the forefront of medical technology and innovation. our industry, i can assure you, is coming back here. >> hold the mic a little bit closer. >> i would be glad to. >> thank you. >> with approximately 7 billion

in revenue, dollars that is, and over 50,000 employees worldwide spread over all populated continents, brawn is one of the largest medical device companies in the world. u.s. organization i represent has nearly 1.5 billion dollars in revenue and sales generated by 5.5,000 employees in the u.s. currently we see our organization and our industry for great claels. challenges. the hurdles for our organization, our industry, are expenses and burden to get products approved currently if the united states. getting more and more expensive. it is taking more and more time. and becoming less predictable to get products approved in the united states. the formal leading nation in the medical technology innovation cycle, there's been an ecosystem

which commissioner hamburg referred to that was working well and it's in trouble and under severe stress right now and we appreciate the focus on it to get this ecosystem back into order, to keep this country back op the forefront of medical technology. innovation. which actually would help me right frou as i lose my voice. sorry. however, i do want to recognize we are addressing these issues. congress and fda, with the medical device user fee act we are seeing acceleration of approval again. it is working. fda and industry again are working together. also important to note, the cooperation of the agency and industry under the medical device innovation consortium under the initiative of dr. sherman which is also working well it is promising. so while these initiatives are certainly a recognition of the issue, they are unfortunately not enough. and we are hoping and willing to work with you to address these issues with all constituents to

bring this cycle and this ecosystem back to work. there is a lot of work. and one thung i shouldn't forget, another large threat to the medical device is the medical device. it is taking fund from small and large from all over this country to fine the cures of tomorrow. and this will not show up time, ladies and gentlemen, but for years to come, if these fund aren't put back into the industry. we are here to offer help any way we can. our organization and industry will help any way we can. thank you very much. >> thank you. now to the physician and chief of children's hospital, philadelphia. >> thank you. appreciate the opportunity to participate in this roundtable discussion. as mentioned, i'm the chair of pediatrics and physician chief at children's hospital philadelphia, commonly referred

to as chops. i'm pediatrician, trained in pediatric infectious diseases. drawn to the fact that our middle son had general injietis at 12 hours of age. chops operates the largest pediatrics network in the country and ranked number one in pediatric research in the united states. like other major children's hospitals across the country, at chops we care for children with common diseases, like asthma, and obesity, and autism. and also for children with rare diseases and children with special health care needs who require the expertise and interdisciplinary care that an academic pediatric center can provide. we are also responsible for training the next generation of pediatricians.

despite the fact that children are our future, that much adult disease has its origin during childhood, the investment in pediatric research in this country is relatively limited. the nih budget portion that's devoted to pediatric research is roughly 10% or so. the investment by the pharmaceutical industry is relatively limited. reflecting the fact that it's uncommon to develop a blockbuster drug for pediatric care. as i think about need for child health and for pediatric research in this country, certainly continued investment and research is critical. the national pediatric research flet work act was recently signed into law in november of this past year. but at this point is unfunded. in addition, the children's hospital medical education program is essential and will

remain eseb shl in terms of our ability to train the next generation of pediatricians and pediatric investigators. training grants, career development award are like wise essential as we take a look at the work force to produce the work that will result in improved child health. thank you. >> thank you, doctor. next we go to dr. chi van dang, abramson cancer center. >> thank you very much, representative pitts and mr. chairman. i'm chi dang. from emmerson cancer center, university of pennsylvania. i'm also here on behalf of dr. margar margea margaret body from cancer research interest with over 30,000 members. i just want to reflect on three areas. first is the u.s. commitment to invest in innovations that we've

made an impact on patient slide and economy. first we believe in investment, that we urge the u.s. congress to have a plan for predictable sustained increase in nih funding so we can keep a competitive edge. which is eroding slowly but tangibly by china's major investment in their research enterprise. only through this investment can we continue to innovate. i can give you an example of innovation that touched people's lives. at university of pennsylvania researchers are able to harness your immune cells and engineer them and fuse them back with the patients with leukemia that has no other options to kill the leukemia cells. can i tell you the impact is clearly tangible because one of the patients sitting in this audience and there is also made impact in children at chop hospital we have now treated over the 90 patients on this

clinical trial that just received a break through by the fda. we are really thanking the fda for watching over this to get this innovation. i also have to mention at this particular example has also made an economic impact because it allows us for a private public partnership with novartis pharmaceutical company that want to take the technology and get it out to treat other cancer patients who have no other options. i want to appeal to you and the u.s. government that congress to really invest in research so we can on pain innovative edge. and make an impact. you know better than i do that for every dollar you invest in research, there's at least $2 in return on economic impact with over 20,000 people in this state. employed because of research. so thank you very much for the opportunity. >> thank you. >> next, dr. norman barton vice

president of global clinical development team leader of shire pharmaceuticals. >> yes, good afternoon. i'm absolutely delighted to be here as indicated with shire pharmaceuticals. shire is a large company that has a very, very big presence in rare disease. it also certainly has other activity in neuro science and various other disorders. shire is a company that made various substantial investments in places where others have not gone. because the road to approval of those types of compounds that are under investigation is perceived of as too risky. too much capital at risk, therefore i will not go there.

i at a personal level am trained as a scientist and for 35 years worked in rare disease. at the national institute of health and the remainder in the biotech sector. and that 35-year period to me has been the most absolutely amazing span of time where rare disease 35 years ago, was really rare and out there all by itself. and it wasn't paid attention to. now it has come to more of the mainstream event. thankfully because of all of the scientific understanding that is emerged in these kind of disorders, the work of the fda, work of congress, the work of the institutes in preparing a

framework that allowed organizations to take a deep breath and say, yes, we will invest under this. so it really is a great honor to be here today. to have the opportunity to explore how might this enterprise move forward and deliver more onity promise with more a repetitive risk so there is more investment under it. for example, the work that i do right now is in a program in extremely prematurely born infants. that is a risky space. and the kinds of dialogues and the conversations that are necessary to move those types of programs forward really require a broad collaborative type of interaction. the type of interaction with the

agency in which complicated ush issues can get talked about frequently. first in class development, what's the pathway. how do we do this. so there's in need really to be able to collaborate under a very, very big way and hear all of the voices, the voices of the little people. of the mothers, the fathers. the underserved. the voices of the scientists, regulators. every voice. it's important because that's how we get down the road to deliver that therapy where none previously existed. and so, to me, a great pleasure to be here. thank you. >> thank you, dr. barton. next we go to dr. lon cardon.

senior vice president of glaxosmithkline. >> i think it is trichic for you to have a forum for a set of voices that can ultimately touch so many. we greatly appreciate that. i'm lon cardon. i'm a geneticist by training. statistical geneticist to be specific, but that usually stopts most conversations. i've had an academic can reer in the united kingdom and in the united states. university of oxford in the uk and university of washington in washington. now i'm with glaxo splij cline that aims to do what you are trying to explore. novel ways and novel approach. we have 17,000 employees in the united states. 5,000 in the common wealth of pennsylvania across seven sites.

including one in mr. chairman's district, in marietta. on vaccines we focus on pharmaceuticals. vaccines and consumer health care products. we also have a site can be congressman, or five sites, i believe within in new jersey. we are delighted to be there with you. today i would like to highlight three themes and will continue do but in setting those up, i heard a few word here that really provide an umbrella over the themes that they would like to emphasize. those words in opening team work i heard from you on mr. chairman and i heard partner shup and collaboration. and that's actually the umbrella for all of the things we would like to discuss today. and in particular, those three items that i would like to emphasize is one, the importance of the digital era. and electronic heal willing records and how we can maximize and harness the potential of that type of data.

and merge that with the power of the boy logical information coming out of the genome revolution and how we can bring those together and that will take partner shup. >> the the second is providing a sustainable framework for the national institute of health and way to draw that work that they are doing internally and externally with their funding in a more seamless fashion. translate into new medicine. and we are pleased to note you may have seen today a partnership of is up working with the infectious diseases on the ebola vaccine, and participating with the international endeavor. and finally, we would like to very much underscore the importance of anti-microbial resistance research and the importance of that worldwide for this goebal clael in front of us. thank you. >> thank you, dr. cardon. and finally, we have mrs.

jennifer westdike. she is representing the pediatric foundation, a parent of a child with -- >> thank you so much for having me here today. i'm honored to be a voice for my son, who eight years old, and for all of the other patient who have were born with or developed an illness that is incurable. my son had a shunt placed when he was three days old. and all too often as a parent you sit back and wonder, it's not if the shunt will fail, it is more along the lines of when it will fail. this is a 50-plus-year-old technology with a very high fail rate. my son has been lucky, he hasn't had too many revisions. he has had throw. however, there are children his age who have had 40 revisions. which means 40 brain surgeries. this has an impact on their lives. how many times can you operate

on a child's head without there being some sort of brain damage on top of the hydroreceiveless, the fluid building up in the brain. it is scary. and it is scary for any parent who has a of any illness without a cure. so i'm so honored to be a part of this and to hear all of the ideas and breakthroughs and hopefully this will have such a positive impact on my son's health and his future as well as all of the children that suffer from any kind of illness and i'm just honored. thank you so much for having me here. >> thank you. it is an honor to have you here. i would like to thank all of the panelists for their remarks. in the remaining time, there is a free-flowing dialogue between the panelists and we hope to allow time at end for two or three questions from the audience. most of the panelists will remain after the event to mingle and answer questions. i would like to begin today's

discussion with this question and dr. collins, i will ask you to address this. what steps can be taken to accelerate the discovery development site over foster innovation, brain new treatments that cures patients and keeps more jobs in the united states. >> thank you, mr. chairman. for the question. and what a wonderful panel. wonderful hearing the introductions. i think we are all very much coming from a similar perspective and appreciate the clans to have this conversation. we thought a lot about this in the light of the 21st century cures initiative that you all have been leading over the past several months. i think there are a lot of things that congress can do to accelerate this discovery development delivery cycle. and maybe you could break them down into three major areas. one is to enhance participant protection and increase transparency. in that regard, just as a

specific example, as the personalized medicine revolution is coming forward quickly and sequencing machines have gone from being the size of phone booths to being the size of postage stamps and the cost has come down from hundreds of millions of dollars to close to a thousand dollars, patients are increasingly interested in having this information being able to take advantage of it for their own personal care. but we need to be sure that we have adequate prohibition there against unauthorized reidentification of individuals based upon genome information. which currently is not properly prevented. we think there should be prohibition sense nonvoluntary genetic testing with perhaps some exception for law enforcement but there is a need there as well to provide confidence to people that this kind of information will be used for their benefit and not potential in some oerther way. that's an example of areas with

patient protection increasing transparency of data that we produce. a second area would be the facilitation of scientific collaborations and innovation. in that regard while it is understandable that congress has considerable concerns about connens conferences and expenditures on travel particularly that there have been egregious examples of misuse of funds of that sort the gsa extravaganza in las vegas for instance, the way in which that is now implemented provides a very heavy burden upon those of us who are trying to organize scientific conferences and make sure that scientist have a chance to attend them. i can tell you from my own 30 years of experience as a physician scientist, many times some of the most exciting ideas that have arrived in my own career have been on the basis of those conversations that happened at these meetings where you rub shoulders with people who are not at your own institution and a light bulb goes off and new idea gets

started and something important for the future gets to occur. >> all right. now i must tell you the way in which oversight is being applied at nih caused us to spend $13.7 million last year on the enterprise and involved more than 150 employees to try to track, maintain some sort of records that could be scrutinized by anybody who want to see all of the connen froms that we want people to go to. and we routinely lose money because it is complicated when you have to pull together a total list of attendees across the government. you often miss early registration opportunities and you have to pay more to go. it is not a system that's working right now. and if there is a potential way to get relief from that, that would be an enormous pat on the back for the whole enterprise because people are deeply troubled about this set of bureaucratic oversight which i think accomplishes almost nothing and yet cost a great deal of time and money.

a third area is to reduce administrative burden and increase the efficiency of the enterprise. give you one example there. eye ironically named paperwork reduction act, which as far as i can tell, caused more paperwork than anything in my experience, will for instance prevent a researcher in the ipt mural program clo is interested in public at tutds towards personalized medicine and wants to carry out a well designed sur i have that's been reviewed by an institution yl reviewed board and gone through all of the oversight. tle can't do that if they want to ask more than nine people a question because otherwise it has go through omb clearance which i guarantee you will be many months and by that time the trainee has gone on to something else. i'm sure that ujors of the paperwork reduction act did not have this in mind when that came along. but it is really getting in our way. so to have those kind of relief will help us. ep hansing participant perfection, scientific

collaboration, those are areas we have many suggestions i give you an example of each of them but happy to have the chance to provide for examples. >> thank you. >> thank you dr. collins. anyone like to ask a question? >> thank you. >> thank you mr. chairman. >> i just wanted to ask ms. wesley because she is from edison and i asked her to come, what one of the concerns i have, and i think we can use this in terms of hydroreceiveless but probably applies in general is that often, you know, you talk about the shunt a lot of times you have to have multiple surgeries. and i do worry and i know you have expressed this to my staff, about the whole issue of you know, cost to the family and

insurance and coverage because we tend to think of, you know, clearly there's more need for research and i know particularly when you deal with a pediatric research, i think the doctor mentioned that it a problem and our focus is on cures. but i do worry, as you talk about your son, that initial operation is covered but then you know maintenance of the shunt, having the repeated operations, the whole issue of access you know for this type of care, did you just want to comment on that a little bit? because i know you didn't really do that. and i know you do talk about it a lot. not only yourself but for the association. >> yes, of course. >> mics are not very sensitive. can you pull that up closer, please? >> sure. >> i'm sure everyone here

understands how the insurance works and they don't cover much as far as the routine check-up a lot of times and an mri has been declined saying that my child doesn't need to be checked once a year. but our children are certainly impacting the cost of health care because of their repeated surgeries. their need for cat scans and nris and shunt series and the insurance just doesn't pay for it. so it is coming out of our pockets and it is very costly. >> i hope you represent the hydrosyphilis association. have they talked about, you know, some way of remedying this in general. not only in terms of research but in terms of access as well and follow-up? i mean, i'm not just talking about insurance, though i know that's an important part of it,

have they made any kind of recommendation -- our point of the roundtables is things that we can do legislatively or otherwise to make a difference. >> well certainly, funding research, that's a big deal. i don't -- i think hydroreceiveless is underfunded. i don't think that many people think about it. 1 in 500 children are born with it. and those numbers are probably much higher because that doesn't include the children that develop it after they're born. so there's from different things and adults that acquire it. hydrosyphilis is very funder funded which leads to little research to change anything. and that's really our mission, pediatric hydrosyphilis foundation is trying to help provide information about this and so people know that the

numbers are are far higher than they think. >> let me ask, i know -- i'm not trying to monopolize it, i know when i listen to -- if i'm pronouncing it properly, you talk about pediatrics in terms of cures, that is more underfunded than anything. and it always shocks me but i know it's true that when we deal with children, whether it is resear. funding, care often times it does seem to fall through the cracks. is there -- do you want to talk about that by comparison to, you know, research and funding in general. or and maybe tell us why that's true, if it is. or if it is true. that's what i hear all the time. >> yeah. i made comments about a few facts in my introductory remarks, namely that portion of the nih budget devoted to research is 10% or less than

10%. and some might argue that that's appropriate because there's much more disease in the adult population. but as i try to emphasize, we're learning increasingly that adult disease has its origins in pediatrics, during childhood, and in addition we know that children represent the future of this country. and so from my own perspective is that it makes sense to at least increase the investment in pediatric research. at least increase the fraction of the nih budget devoted to pediatric research. i want to highlight an approach that i think underscores as well, the point that dr. collins made about team work, partnership, collaboration. as we think about an area in pediatrics that has arguably been the most successful model, both in pediatrics and as an example for medicine more generally, the children's oncology group which represent a

network of immediate at trick centers across the country with over 200 centers participating, has been remarkably successful in bringing together investigators and bringing together populations of patient from across the country. to study those patients and to develop new approaches to treatment and the consequence has been remarkable advances in outcomes and most or at least many pediatric cancers and i think that model is a model that we should think about more generally. more generally under pediatrics and more generally in medicine and biomedical research, broadly speaking. and i again, highlighted the national pediatric research network act which essentially proposes to create con soar sha that would be like the children's oncology group for other disease processies. >> dr. hamburg, you wanted to add something? >> yes. >> you can pass the mic down.

>> well i just wanted to really build on the important issues just raised in terms of opportunities for 21st century cures moving forward. one is the importance of really looking at how do you innocent advise research and critical areas and market to really move the dial.innocentadvise researc critical areas and market to really move the dial. and i think we haven't done enough analysis on what works and which don't. but we have areas that have proven tlir value and the drug act which has led to a huge expansion of research and development and new products. in the orphan drug arena last year. the new molecular entities that we approved a very large percentage in fact to address orphan or rare diseases and i think that is partly reflects an

incentive framework that has been working. and including actually, a new incentive program to try to stimulate pediatrics therapeutic development and i think we are beginning to see some movement in that arena. i also wanted to underscore in relation to what miss westdyke, in we are seeing the patient's voice in research and product development and the regulatory process as well. and i think it is very important that there is feedback on the patient experience of a disease. what really matters. what is lacking in the current product that is needed. and then in terms of ourvi, how really look at some of the measures that matter that might not be the measures that we as physicians scientist would think of but that are important to think of the patient's experience of the disease.

and symptoms. and begin to integrate those patient reported outcomes into the regulatory assessment of a product as well. so i think that is lot of interesting and important activity some of which are under way and expand and some of which are still in need of more analysis and development. >> we heard that same emphasis at you're last roundtable in-patient feedback that we put in the bill. i want to ask you, dr. barton, as representative of industry, do you and shire feel that pediatric priority review voucher program is an effective incentive for rare pediatric diseases? anything that can be done to make it more effective? >> thank you very much for that question. i absolutely believe and we as an organization believe it is a very important incentive. it puts a spotlight on research

and children and it derisks it at a certain level. because there's an incentive. and that becomes a very important issue as companies look across their portfolios and decide where to make investments to better the health in our nation. and so, that, in my mind, is a big deal. how it could be made better right now, it is a provisional or temporary trial. it would be a stronger statement if it was permanent. you know, we the people, we want to invest and make incentives and opportunities for research with children. while i have the mic, there is one other thing i would like to comment on, if i may. i particularly impressed with

the comment that dr. hamburg made, about the voice of the patient. aep i think that as do many of my colleagues, within shire and across the industry, in the rare disease world, the voice of the patient or the patient organization, is hugely informative. and in fact, often times patients and organizations who represent them know more about their disorder and what really matters than any of us. you know, highly schooled physicians or regulators or scientists or whoever we are, they are very, very well informed and sophisticated and so as industry engages with regulators, with the fda, bringing that voice to the

table, not every time, but in serp certain situations, where the pathways never been down or the road has never been laid out before, that voice is just critical. pathways never been do road has never been laid out before, that voice is just critical.