information. i have heard from home, and they feel the larger networks are the wave of the future. you say you do not need additional legislation to continue, but you are having to borrow resources from this and that, so is your are advice to the committee that we need to do more in technology when it comes to improving timelines on clinical trials by focusing on these networks and the electronic health record? >> the networks have much -- the electronic networks have much promise in doing clinical trials. if we could move clinical trials more out into the community and have i'm out in the community, cancer patients, most cancer patients in the u.s. who have diseases that are untreatable are not -- don't get in to trials because they are being treated in places that are not running trials. we need to move this out in to the community, make those folks eligible.

and i'm on the committee in the lung map trial, and i have really urged that we make sure that we are out there in the community so that anyone who has lung cancer has an opportunity to participate in the research and perhaps, have a more effective therapy. so, i think the electronic health records that -- it's a huge different area that we are working on and how to do clinical trials utilizing the infrastructure that is emerging. >> right, thank you very much. >> chair, thanks, gentle lady, and now recognize gentleman from new jersey. five minutes. >> thank you very much, mr. chairman. a focus of your testimony has been focused on fda's efforts on patient engagement. it's my understanding that clinicaltrials.gov was intended to provide clinical study information for patients, health care providers and researchers. but it seems to me that this

site lacks considerable information and has proven to be difficult to navigate. dr. woodcock, would you please comment on the current utility of clinicaltrials.gov is this. >> i think that it has provided along with the requirement of the medical editors of the journals that things be registered before they are going to be published, provide a tremendously more transparency in to what clinical trials are going on in the united states. that has been a big achieve pt. we know that, the issue of publication bias and we know what trials have been done. however, i agree that certainly for patients, i think that initiation of trials, and trials that may be ongoing and available to them has been effective, although, as you said, there are technological

issues that remain. it has made tremendous progress in transparency. >> is there a way that you and we can work together to improve it and i'm not suggesting that you are in any way responsible for the challenges that remain, but moving forward for the better health of the american people, how together can we improve it? >> well the fda amendment act required that regulations be issued around the results. >> yes. >> section, of this. and that they consider whether to require the submission of clinical trial results for unapproved products. much of the lag in getting results in there is that products are still not on the market. nih is the lead for the rulemaki rulemaking. i think they are in the best position and they operate the infrastructure for this database. >> thank you. >> and in another area, in the

past several hearings, we have discussed difficulties of the lack of coordination and that leads toinefficiencies, what methods are being produced to reduce redundancy in clinical trials and what steps are taking to make sure we are not making the same mistakes over and over, and doubling up on research? >> hopefully, host things will eventually come out and be published. certainly in the drug development area, there's interest in more sharing of earlier data sharing of data. this has been a reattractable area for transparency. we continue to work on that. as far as some of the things that were referred to in the prior hearing, which i was able to listen to some, they were talking about some of the

inefficiencies of r.i.bs. you may have 100 r.i.bs, and i believe there's efforts to try and address it. but really, we came out a number of years ago and and say our r.i.bs would be in our trials and then the contractual agreements that take so long to set up, with each specific site is something that has been taken on, they have tried to develop model agreements and so tortfor. but that is something that the standing trial addresses, you sign this contractual agreement once and you do multiple investigative agents. >> are we moving in the direction of the central r.i.bs in your judgment. >> there's a feeling that it's desirable and various universities are naturally concerned, legally concerned

with about their own liabilities. and so forth. >> of course. >> and so there's a push and pull about that. >> i think this is an area that we need to engage in further investigation to make sure that we move forward in a manner that does not result in redundancies, thank you, mr. chairman, i yield back the balance of my time. >> recognize gentle lady from illinois. 5 minutes. >> thank you, mr. chairman. thank you dr. woodcock. i think you are really an excellent witness. i appreciate your answers. >> thank you. >> i wanted to go a little further on the problem that congress woman caps raised about the under representation of women. i know you said you found that women were over represented. recently a meeting was sponsored with leading women heart expert s, both clinical and research

experts. they were concerned that the lack of representation in clinical trials is limiting our ability to treat women effectively with heart disease. and they were focusing in on heart disease. according to the experts for the last 50 years, the women's heart disease research has been based on research on men. what they said, that women make up more than 50% of the u.s. population. that women comprise only 24% of participants in all heart related studies. and additionally scientists from the women health research institute at northwestern. that is in my district. have raised concerns about the disproportionate number of adverse drug effects that are due to the lack of sex based research. you know, the differences in male and females impact the rate

of drug effectiveness. the lack of requirement for drug manufactures to take it in to account and document any sex vary ability early in the drug development pipeline before the drug is released places consumers, especially female parents at increased risk of adverse drug effects. so, i want you to respond to that. many of us were really left with a very disturbing feeling. heart disease is the major killer of women right now. >> well, i think we have to, what are the facts on the ground? all right? one of the reasons for the disparities that they are mentioning is the fact that men suffer heart disease earlier in life than women. >> although let me point out that they also said that the growing number. even though it's lower. is younger women getting heart attacks and heart disease. >> yes, so that, the reason for

maybe mal distribution in the trials is because there's an age cut off at the -- in our survey, we found that 19% of the people in the trials were over 65. which is more than in the general population, obviously. but it is of sick people that still is low representation. right? say with people with heart disease. generally speaking, there's often a cut off, age 75, and we are tryi ining to eliminate tho cut offs of age. we require, to your point, we have always required male and female animals in the toxicology studies. and we require pk, pd early in drug development and we look at

our clinical pharmacologyists, look at blood levels and exposure in men and women. we understand that. that is modern drug development. there's multiple trials that are done that look as exposers, achieved blood level by generde and other factors. we can look at the face -- phase 3 trials. there's been a requirement in the regulations that sponsors submit a gender analysis with this application. >> let me, is this incorrect then? it says, women comprise only 24% of participate amou-- participa in all heart related studies. >> that may be true, it could be including devices. and it could be the age disparity with the on set of disease. >> i hope you will look at that,

it's a great concern. it's a growing problem for women. and let me just give you an example. women, because we have different symptoms of heart disease, if you have symptoms of nausea and dizziness, go to the emergency room and say, i'm having chest pains because without that, you may not get an electrocardiogram, and you may be misdiagnosed. we need to help women. thank you. >> louisiana, 5 minutes. >> thank you. >> i mean it as a big compliment. so, next, real quickly, because i want to talk about manage else, but, does fda, you mentioned that some institutions may be nervous about their liability, if they refer their irb activity to a centralized irb, but many do, so we know

it's a false argument. is there any way that the fda can reassure their institutions. and the gentleman from mayo mentioned it's a cultural issue. >> i heard his testimony in my experience that there are legal -- their concerns of the council, of the -- >> attorneys are always nervous. right? they don't make money if they are not nervous. i hate to be cynical. >> yes. >> any way that you can send, fda can send assura amassuranc >> that's possibly more that we can do to encourage this. >> okay, let me then bring on and go back to, you mentioned something intriguing earlier. that there may be somewhat high risk for disease, therefore they will be more risk tolerant. now, i have a family member nephew with down syndrome, and i am looking at the alzheimers.org

website, they mentioned that adults with downs syndrome will have evidence of the tangles associatesed with alzheimer. what are the issues regarding wow,s that group of kids -- this is a group of kids, adults, who 100% at risk for a terrible condition. >> right. >> but there's other issues involved as well. what are your thoughts about this? how do we make stuff available for folks, incredibly at risk for such a terrible disease? >> yes, well, with alzheimer's, there there's a number of problems. the basic is we do not understand the disease enough, and interventions have been tried in late stage disease, with people already dimented, it failed to work. >> the problem is predicting at an earlier stage those at risk, correct? >> that's correct, if you want

to intervene early. we issued a draft guidance saying that if you want to intervene earlier, we will accept an end point, that is cognitive testing. >> how do you decide which population is at such high risk? if have you a control group, you see what i'm saying, 10% are really at risk. you are with me. this is a really expensive study. >> that's right. so, we advocate techniques called enrichment, which you try to use bio markers or other tests to figure out there are genetic conditions that risk you to alzheimer's disease. >> that would be a example? >> yes, and there would be others. >> if you accept these, are people now using them? >> we need agents to use them. >> agents? >> investigational interventions that we can test in the people. and that is part of the problem, the science of understanding what causes alzheimer's and what

you can intervene in that would actually or delay or prevent the disease is not mature enough, and we have approved a couple of imaging agents for alzheimer's, but they are not 100% and you would maybe be kind of -- >> for example, i know hyper insulinemia is considered a factor. and actos may give benefit. it would be a nonmetabolic syndrome for the use of acto stmp s, fair statement? >> hm-mm. >> i ask to the agree to much that has been -- again, what is the current state of using that sort of thing? >> the current state, if somebody decided to do a trial and i believe there's been intervention trials at high risk and higher risk people. they may identify people they felt were at high risk for one

reason or another, randomized them to this intervention or not and then we would allow use of neuroc neurocognative testing and if the treatment group did better than the placebo group, we could give accelerated approval. >> you have the frame work, but it's a question of are hahaving someone come forward to complete the study. how long do you think that will happen, 20 years? >> no, but we need better measurements. like a subtle cognitive function, us and others are working on this. because, the earlier you can intervene, if you have a very targeted test that can identify people early, they don't have any symptoms, you can tell that their brain is not working as well as it should, and it will decrease over time. prevention is difficult.

there you want to intervene on people who are well and treat them for a long time, and expose them to something, with the hope that at the end of the day. they are not going to get whatever bad outcome. >> we are out of time. thank you very much. >> i will recognize the gentleman from virginia. 5 minutes. >> thank you, dr. woodcock, as others are have said today and also what i have heard in the informal conversations that you not only do a good job as a witness, but you are doing a good job overall. so, i appreciate that. thank you so much for being here today. i will tell you, you and doctor had a conversation about lawyers. some lawyers are always nervous, other lawyers are always looking for a way to find a way to solve the problem and so maybe we need to get some of those lawyers on your team and the corporate teams and solve a problem and figure out how to make these things work. i do think it's important. as you probably know, i'm one of those that advocates that we try

to move a little quicker in those areas that we have problems that we don't have solutions for currently and also, favor what is known in some states and state laws is right to trial when you have a situation where doctors have tried everything and folks are given a diagnosis they have months to live. i'm one of those people that believes they should be able to try anything that they want to try to do. the fda is not going to protect you if you are going to die from something that may kill you. it's going to happen one way or the other, you should have the right to try something. i know there's a lot of issues surrounding that. i don't know if we have time for that discussion today. and i know there's another panel and i want to hear from the patients as well. they are involved in this process. so respecting you greatly, i yield back my time. >> chair, thanks the gentleman and recognizes gentle lady from north carolina. 5 minutes for questions.

>> thank you, mr. chairman. and thank you woodcock for being with us again today. this is such an important issue, you -- as you know, we had our panel on wednesday and seemed to me that it was a general consensus that everyone is looking for ways, you know, to expedite this and to make it more efficient and get those drugs to market sooner so we can take care of our patients more effectively. in your testimony, and in the discussions that we have had today, you have touched on the bio markers, and targeted drug development for disease ed populations, obviously. as all of our representatives here, we all have constituents with rare diseases, heartbreaking, especially right now in my community i have a very good friend with als. and as i'm learning more and

realizing we have had a number of our members of our community sdi diagnosed with als, so it's very important to me right now. and i'm just looking at the idea, as far as the target approval process being appropriated and applied through the fda, it seems to be that we are looking at cancer and hiv, where do some of the rare diseases fall within that? and you know you had mentioned and there was discussion about the master protocol and that seems to be applied more to cancer, hiv, where can some of the rare diseases fall in there and what we can do to help make that happen? >> well, any rare disease would be a great candidate for a standing panel. where they are ready to evaluate any therapy that advances

through the early, the nonhuman stages. so they could pick that up right away and test it quickly. and that, in the meantime, until that happens, they can get what we call natural history. which i know sounds very wonky, people are asking just now, mr. cassidy, how long does alzheimer's progress? we need to know had that so we can design the trial correctly. and in rare diseases, they are more difficult. nobody knows. and usually they get experts together and they say, it takes this long. and they are usually wrong, because they have only seen a few people. so, we are encouraging these natural history studies. these networks. first they look at the people and they look at the bio markers too. what changes in als? what can we machieasure? >> can we measure something that gives us indication that treatment is working. as soon as a therapy becomes available, you can rapidly get people in a trial and there

would be no delays. because there's no delaying an als -- >> right, exactly. and that's, you know, part of the concern. and ernl, i agree with my colleague in talking about right to try. you know, had this would be a perfect example of decisions that families and patients can make. i do want to talk about, you know, you had also mentioned listening carefully to parents and families. >> yes. >> and you know, do you consider and give more weight, that is one of our questions. how much weight are you giving the patients and families? and there again, from our perspective in congress. what can we do, you know, as we have heard everyone agreeing, that we need to make a difference here and we can move things forward. how open is the fda to this possibility and what can we do, right now, to make this happen? >> well, as i said in my

testimony in the beginning, medical culture has changed over the years. now it's patient centric and the fda culture is a medical culture, so that has changed at the same time. but slowly. we have been working though, very diligently with patient groups and so forth to try to get the patient point of view more central to the evaluation of benefit and risk and what it means to the person who has the disease, is going to take the drug. to answer your question, what can be done. i think a lot of it needs to be done out in the community. the patient groups need to get organized and develop these, some of them are working with pacori, and trying to use that mechanic niche to get more information available and so forth. we have gotten draft guidances from different groups, including muscular distrophy, a at the

same time saying this is what we care about and this is what we value and we will pay close attention to those, and those are very valuable. >> thank you. we are voting on the floor, we have ten minutes left in the vote. we have three more questioners. mr. guthrie, recognized for five minutes for questioning. >> thank you, i will try to be brief. i will echo about your testimony, appreciate it. since we started the 21st century cures, both sides, i have heard from a lot of graups and i have heard several times that the oncology divisions that everyone likes to work with. and some of the other divisions is not worked well with as much. within your agency, you are

having wonderful feedback and wonderful programs and some not so much. my question is, is there impediment, is there any impediment taking what is happening and using it in other agencies. is there something congress can do to make it easier? >> ten years ago, i heard a lot of negative comments about the oncolo gmp oncology group, and now they are really on fire. they see for their patients they took care of, they are all oncologist, that these new treatments would really have made a difference for those people. so they are doing everything that they can to get the treatments out. we need the same kind of inspiring therapies in the other areas, they are doctors, and physicians and they care about patients in their disease area. this, this is break through, i

don't know if you can see it. other disease areas are coming up and we are designating, in neurology and psychiatry, this type of thing will respect help. of course, we try to are have a management structure, multiple mechanisms where by we have consistency and uniformity of our approach and procedures. i think we do well in our procedures. i think, underlying, sort of the attitude may have something to do with, the underlying science. we had a war on cancer, it's starting to pay off and we need to really expedite that. >> i have a bill particularly to put the same status for professional budget judgment status for alzheimer's, which which would, we were going to spend in 2058, a trillion

dollars. so that is when my children and grandchildren will be taking care of it. hopefully we can have inspiration in dealing with it. >> if there are promising treatments for alzheimer's, we would jump right on them. >> appreciate that. thank you very much, and i yield back. >> chair recognizes gentle lady from tennessee. >> thank you so much, dr. woodcock, i have basically one question that i do want to get to. look at the quidp, and the moving forward of that, pardon me. it can give up to five years of additional data exclusivity, bipartisan effort, we were all for it. what i want to know from you is how many quidps has the fda designated to date, how many products have actually been approved to date and do you believe that the quidp is an important designation?

>> absolutely, it's important. we have granted 50 designations for 34 unique molecules, and in the last several weeks we have approved the first two medications that are designated. first two anti-microbials, so that is making a difference. we do feel that probably more needs to be done. >> and in that more needs to be done, give me a couple of examples of what you think the next step should be? i would be anded einterested i that? >> we are interested in the path way, that would be matched with some sort of symbol or logo, that would enable doctors and other prescribers to recognize that it was a limited program. we think that would also allow us to streamline the development program. >> excellent. and i just, for the second

panel. want to welcome a fellow tennesseean, dr. marshal summer. who is going to speak on the national organization of rare diseases. welcome, we are delighted you are here and i will yield back. >> chair now recognizes -- thank you, mr. chairman. i thank you for your testimony, dr. woodcock. i asked these questions, a few months ago and did not get a response. i want to see if we can get a response this time. appreciate if you can answer. can you tell me how many treatments were approved with bio markers used for the first time? >> no. i don't have that in the -- >> can you get that information? >> i would be happy to, it's a very interesting question, yes. >> and then next question, have any act he -- accelerated approval occurred in a never before treated disease? >> yes. all the time. i can get that for you.

i don't have it again. >> how many new bio markers did the fda accept for the first time use in the last five years? >> certainly. >> can you get that for me? >> absolutely. >> very good, thank you very much. i know we don't have a lot of time, i will yield back. >> thank you. two minutes left in the vote on the floor. so, we are going to recess, there are two votes, as soon as we have the second vote we will come back and reconvene with our second panel. again, dr. woodcock, thank you for coming, you have been a terrific witness. members will have follow-up questions, we will send them to you, and we ask that you please respond. thank you and thank you for your patience. the subcommittee stands in recess.

time of recess having expired, we will reconvene, subcommittee on health and introduce our second panel, and we have five witnesses on the second panel. i will introduce them in order of their presentation. first, ms. pat furlong. founding member and ceo of the parent project of muscular dystrophy, and third, mr. richard pops, chairman and ceo -- fourthly, dr. leonard lichtenfeld, and finally dr. marshal summer. director of scientific committee. and thank you all for coming. you will each be given five minutes to summarize your testimony.

your written testimony will be placed in the record. we will start with you. you are recommegnized for five minutes for the opening statement. >> thank you, good morning, my name is pat furlong, twenty years ago, i joined other paren parents in the group. in 1984, i received who diagnosis on my two sons and woe both of my sons are gone now. i wage the crusade in their honor much has been done to change the landscape. and much has been the direct result of congress and this committee. since the md-care act was enacted we have seen ten years added to the life span of patients. there's been an improvement in

quality of life, driven largely so that all patients can be diagnosed a diagnosed accurately. the md care act transform onned the research landscape, what was just 12 years a ago, a near barren field, where multiple therapies are in clinical testing and several others are in early stages of development. despite this, it's still a fatly disease. both boys end up in wheelchairs in their teens and only a few live beyond their 20s. our community needs therapies and need them fast. to achoef the goal, ppmd has led ground breaking therapies. one is a lack of regulatory understanding of patient and parent perspectives on benefit risk and two, a lack of clear guidance or direction to the bio

pharmaceutical companies that are designing the clinical trials. approxima ppmd conducted the first surveys, it involved 120 parents of the children. it validated what we have known anecdotally for years. it's 100% fatal at a young age, many patients and families are willing to accept higher levels of risk for the return of a potential benefit. data has been shared with the fda and was published in an academic journal. and now they must ensure that it apply this to their drug development. small patient populations. limited knowledge about the condition. these are some of the challenges. at the invitation of the tda, ppmd led a comp hency six-month effort to convene key

stakeholders, parents and clinicians and researchers and industry to write a draft guidance document that could address trial design and other issues. this was submitted to the fda last month, marking the first time that a patient groups that led the development of such a product. now the fda must step up to review the draft, and issue a guidance document under the agency's name. while each of the projects is focused on this disease, each offers a template or model for other diseases. particularly rare diseases and i hope that other organizations will take on similar programs. what can congress and federal agencies do moving ahead? first, make sure that the patient perspective on benefit risk is considered by the reviewers of the f tda. one will be how they will disclose how they did or did not take such information in to account making their decision on

a drug application. this would shed light on what many consider a mysterious project. and a greater focus on regulatory science, so that they keep pace with the break neck speed of innovation. incorporating perspective earlier in the pipeline can maximize the likelihood that candidate therapies will be ready for the rig or of the fda, and finally, greater flexibility in clinical trials. including diseases that have small population. business as usual trial design does not hit the mark when working with these population. leadership will continue on monday with action by the full committee on the md care act

amendments. far too many tamfamilies my own included, they do not act fast enough. thank you. >> you are recognized for 5 minutes. >> thank you for the ability to present the testimony. the story of cystic fibrosis is a story of hope and optimism. the information we have documented in our submission shows what is possible when a system works well. when patients, stakeholders and the regulatory agencies collaborate to develop life changing treatments. is cystic fibrosis is clearly a life threatening genetic disease that effects about 30,000 individuals in the united states. there's been tremendous progress in life expectancy. there are people living today with cystic fibrosis in their

30s and 40s and some beyond. but we still lose too many patients at very young ages. the increase in life expectancy is due in large part to ground breaking advancements and treatments made possible because of the cystic fibrosis foundation, our patient community and our industry collaborators. two years ago, the fda approved colidico, the first drug to treat the under lying causes of cystic fibrosis in a small subset of people with the disease. held as a game changer, it has transformed the lives of those taking this drug. here's a perfect example of personalized medicine. i might mention that the fda approved this drug, in near record time. three months where the prescribed padufa, and -- two weeks ago, we saw another break through in cystic fibrosis, it happened when the positive data from a phase 3 clinical trial for a new therapy that is

targeted at 40% of the cf population. it was released by vertex pharmaceutical company. these things would not possible if not for the break lthroughs, the cf community was thrilled to learn that the trial participants showed a significant improvement in lung function, weight gain. and 30 to 40% reduction in exaserbations, this is clearly a game changer for the parents. obviously they plan to split the now drug application by the end of the year for this treatment. what is exciting about the progress, these drugs would not have been possible, were it not for the foundation in the patient community. our commitment to scientific drug development is at the root of our success. but it has not been easy and it

has not occurred overnight. and in 1965, we created the first patient registerry in the united states. now a model for chronic disease. because of the registry, we have a documented natural history of cystic fibrosis. we have mutation analysis on most of the patients as dr. woodcock referred to this morning. and we have the ability to have post marketing phase 4 follow-up as they are introduced into the community. the same year, in 1965, we created the care center network. 90% are seen at the fubdnded ca centers. the cf jean was discovered. and in 1998, we established a clinical trials network. the first clinical trials network. founded by a nonprofit institution. it is a critical component of

our ability to collect, to conduct cf clinical trials efficiently and effectively. in 1999, the cf foundation pioneered a successful venture model to derisk companies from investing in cf research drug development. it was a $42 million investment in a small company this san diego that led to colidico, vertex would not have colidico and the other drugs announced last week, were it not for the cystic fibrosis foundation. this same collaborative spirit extends to the foundation's strong partnership with the food and drug administration. the fda, with the fda, we are committed to collaboration. just last week, we met with fda officials to discuss tragics for clinical research design that

may not occur until five years from now. over curing a decide is never easy. and more risky is the approval of drugs without sufficient data to assure efficacy and safety. if this happens, you place ashts immediately at risk and you lose the opportunity to test drugs that could have a real impact. beneficial event. in closing, what can congress do for them? >> congress has to make sure that the patients have a seat at the table. and they must provide the necessary resources. and congress must provide the nih, and fdh sufficient resources for regulatory sciences. finally, congress, we are encouraged that they look at the cts-a program, the network of care centers that are funded by the nih and see how they may use them to facilitate clinical trial network and the development of patient registries and other rare

diseases. once again, thank you for the opportunity to add the cf's perspective to the discussion. >>. [ inaudible ] now it is. thank you. and i would like to express my respect for and appreciation for the folks on this panel and for dr. woodcock, we are all partners in this together and it's an incredibly important mission. the simple and powerful concept of incorporating insights from patients is centrally important to the nation's health care system and it's a way for us all to have a impact. i have served for 20 years, in our company. our company can develops medicines for those living with

chronic addictions, our approach is dependant on considering the patient perspective early and consistently throughout the drug development process. i also serve on the boards of both bio and pharma and was involved in the negotiations as well as the preparations ongoing for padufa 6, where elevating the patient voice has already emerged as a key theme for that initiative today, i would like to propose a new way to develop drugs. and that is to have all the parties involved. and the frame work is based on three core principals. first, is that interactions must be data driven. based on science, other than the -- and separate from powerful and passionate advocacy messages that patient groups otherwise deliver. and the frame work should be

actionable. it should improve the overall efficiency of the rouse, rather than adding new steps in a process that is already incredibly complicated. this is particularly important for new drug companies. and the approach should preserve and enhance the gold standard of on safety and efficacy. which is one of the great national treasures. i believe deeply and personally that increased patient input can go-exist with efficiency and the highest level of scientific rig or. there's no consistent way to generate patient generated income. it would have an impact on the range of critical decisions specific to particular product candidates and certainly to the way we design clinical trials and implement them around the world. this is an important missing link. as dr. wooddock mentioned, patient engagement is not a new

concept. it has resulted in meaningful new expansions of patient engagement. f fda has been open to and has included patient reviews in their information. the proposed frarm work i'm considering would build on all of these things. the historical paradigm of drug regulation as a bila t ampbilat process. patients organize in new ways. if their critical role in driving innovation is becoming the rule and not the exception. we have 20th century regulatory frame work for 21st century drug development. to tackle these increasely complex scientific and regulatory issues, as we look to are treat and cure complex diseases, all three can work together, so that existing regulatory frame work. >> this includes more clinical trial designs and enrollment

methods. advancing fda's evaluation. these are exciting areas for future consideration. we will need to evolve the way we work together. and our accountability to ensure that the -- there will be a number of challenges to this. this could include establishing a common threshold for data and scientific are rigor. modifying existing regulations to assist the frame work. protecting intellectual property and data. and maintaining the standard of financial safety. the groups will come together and implement the new frame work, building on the existing frame works. we should analyze statutes and regulations to identify

impediments and opportunities. in conclusion, the concept of a new and comprehensive patient ininclusive frame work is am ambitious us and modest. it is modest because it can -- is not a regulatory path way and builds on an existing foundation. we are standing by to help you in that effort. and thank you very much for your leadership. >> chair, thanks gentleman, you are recognized 5 minutes for your opening statement. >> good morning chairman and members of the committee. i'm deputy chief medical officer for the american cancer society and truly appreciate the opportunity to be with you today to testify. the cancer society is pleased to commit to the dialog. today, i would like to focus on three critical areas for the

committees consideration. one is the need for greater investment and research. consecuti secondly, and aefficacy of approve drugs and make the parents be vou s involved in al. we must continue and expand our stead fast commitment to research and continue to support researchers, working on finding the next generation of cures. just as important, we must ensure that the expedited approval products are appropriately safe. effective. and accessible to patients. the goal of the 21st of new

medical treatments. there are few other areas that can match the field of development activity in the field of cancer. the fda's office of hematology and oncology products have aggressively used the tools provided be congress to speed products to patients. in the past eight months three cancer drugs have been approved. one approval was based on a trial of 111 patients an example of research and approvals happening faster and with smaller clinical trials than in the past. it does have drawbacks. they may not include a diverse population which may yield an incomplete picture of how a drug might work. small trials tend to be seen in deadlier cancers where there are no other good therapeutic

options and the risk benefit tolerance of a cancer patient facing a poor prognosis may be much different than those with other available treatment options and the same acceptance of reduced day they may not be appropriate in other fields or for other diseases. finally i want to stress the importance of researchers, pharmaceutical companies and the fda in engaging widely and meaningfully with patients. the food and drug administration safety and administration act requires acs can championed provisions to expand the patient representative program to maximize patient input during the drug development process. we need to continue to build on that progress. patients can provide important perspectives in various stages of product development and regulation. they know more than anyone what

is most important to patients to themselves. symptom reduction, risk tolerance and design elements that might affect trial recruitment or retention. this kind of patient involvement should be supported. and requiring fda to address challenges must be fully implemented. we urge the committee to consider examining opportunities for providing greater funding to support the program as well as broader, continued engagement with the patient community. another important way patients perspectives can inform development of therapies is through the design and use of patient reported outcomes. measures of cancer therapy effectiveness sometimes includes functional status, pain or quality of life measures but these may be reported by the physician rather than by the patient. it should come from patients themselves and patients should help prioritize the importance

of these side effects and the overall response to a disease and associated treatments. when quality of life outcomes are rigorously sported by the fda they should be included in a drug's labels and should be considered for a drug's approval. the fda should be encouraged to work with industry and researchers to report self reported as a part of clinical trials. we appreciate the opportunity to contribute to the dialogue around the committee's initiative and look forward to working with the committee and its staff. i'm happy to take any questions. thank you very much for this opportunity. >> chair thanks the gentleman. dr. summar you are recognized for five minutes. >> thank you for inviting me today. my name is marshall summar i'm the chief of genetics and metabolism at children's national medical center in

washington, d.c. i'm here today in my capacity as a member of the board of directors of the organization of rare disease and the chairman of the scientific advisory committee of nord. nord thanks you for your continued strong support of the rare disease committee. you have made a huge difference for us. nord is dedicated to helping people with rare decides. riding research and infrastructure support to the rare disease committee that small organizations cannot. it's the nature of rare disease. they're small. nord played an active role in the active drug act. data show that years of life lost to rare diseases declined at an annual rate of 3.3% after the orphaned drug act.

without these new drugs the number of years lives lost should have increased 1% per year. speaking personally without these treatments many of my patients would not be here. i thank you for what you have already done. these efforts represent a good beginning but there is more we can do to improve the lives of our patients. and this is a great way to do this. nord's long advocated including the patients in the drug development process. we believe much more needs to be done to make patients feel they are partners in the process. nord will work with the fda and advance the patient role we have developed the -- we look forward to discussing the ideas with the committee as the 21st cures initiative continues.

first we support the establishment of a commission and national plan to determine priorities, methods resource needs and a consistent agenda on rear disease registersies and natural history studies. to assess a drug's efficacy we need to know the frequency and severity of clinical findings. this information is need before a clinical trial can begin. we would like to standardize registersies in natural history studies. this could be one of the most important accelerators of the treatment, development and monitoring process. these registries can be used in the post approval process as well. this is an area where patients can have a major and cross saving impact on the process. patient entered data is accurate and useful when created properly. hybrids can speed the understanding, discovery and approval of monitoring process.

in collaboration with the n.i.h. and fda nord is creating a development, the nih has demonstrated the benefits of this approach. we have had approval of three drugs over a ten-year period with 700 patients. it has definitely slerlted the proce process for us. we are developing statistical models to further refine this process. they are also involved in patient driven registries and will work with nord on our program. all of these efforts will help our patients but a national plan and standards would help revent dupe kate effort and resources. the other thing we advocate is a change to the review board system. i have been working with this

system for the last 30 years. currently all clinical trials for new treatments whether a drug, buy logic or medical device must receive approval. each institution requires its own approval. this is one of the greatest impediments and cost to clinical trials. nord recommends to derisk the process and foster the creation of a -- the derisking of the process and the encouragement requirements between institutions receiving federal funding would save cost and time while accelerating the clinical research process greatly. it will allow greater patient participation. these are just two of our representations. my written testimony includes the rest and on behalf of nord thank you for allowing us to

testify today. >> thank you for your opening statements. we'll now begin questioning. i'll recognize myself five minute first that. mr. furlong we'll start with you. do you believe your guidance collaboration with industry is a scaleable model that can be used in other conditions, specifically, where there are unique factors that make duchene muscular dystrophy -- >> thank you for the pitts for the question. i think this methodology is exportable to other rare conditions. how we initiated the guidance is we had patients, academia as well as industry. from there the steering committee identified several areas, seven working groups of things that they felt were relevant to include diagnosis,

biomarkers, clinical trial design and benefit risk and we further developed a cab which is the community advisory board. so that would incorporate the entire voice in any patient group that wanted to contribute to the development of the guidance. this standardization for the guidance is that it would be a reference document and include documented evidence that was published or send for publication by the end of july. this methodology is exportable. it was an investigation and thorough thoughtful look at the community of duchene but most diseases could do the same. their progress might be different but it is certainly exportable. >> thank you, dr. beal. communication with patients to make sure they can make informed decisions is critical. how does the cystic fibrosis community communicate with

patients about the various options and how do you think we can best translate your good practices into the cures initiative? >> thank you very much. first of all we have -- because 90% of our c.f. patients are seen in a network of care centers and we have a clinical trial network there is a very close relationship between our physicians and the patients that are involved. and that's critical toward the recruitment of patients in the clinical trials it's critical important for showing them the value and the risk of participating in clinical trials. and it's that close association between the physician and the patient and the recruitment process in a very closed network that is critical. that's why i think that clinical trial networks are important. so we also have established in our clinical trials network a data safety monitoring board made up independent of the cystic fibrosis foundation but

made up of experts. that provides a degree a of away shurns that there is somebody looking out for their continuing interest and in risk that may be inherent in any single trial. plus we have worked very hard to try to create a culture of participation and responsibility that when you have a small patient population needs to participate in the process. >> launch'ing thank you. mr. pops what stage of drug development could most use the assistant of patient insight about benefit, expectation and risk tolerance. >> thank you for the question. it's the most exciting part of the opportunity is it is ever stage from identification of new drug candidates all the way through to determination of the value of the medicine after the completion of pivotal clinical trials. and i think that's the whole idea of this frame work is

creating a structure where we can get that input on a continuous basis and it could transform the way we approach these development programs. >> thank you, dr. lichtenfeld you have discussed examples of cancer drugs that have been recently successfully approvedly the the fda through an accelerated approval process. are there best practices that we can learn from cancer and how fda is expediting the approval process for particular drugs? >> thank you, mr. chairman. when we talk about best practices the question came up with the dr. woodcock earlier today. what is the oncology community doing that is different than other communities. we have research that has been building for decades that is producing exciting results that is actionable and companies are standing up to create drugs for

the targets that we're finding for the immune therapies for the genetic therapies, the genetic markers. we are interesting and turning point kind of place. but important relevant to your question, the office of hematology oncology products has also stepped up to the plate. and as was mentioned earlier, the oncology community appreciates the efforts to reach out to the patient community, to reach out to the pharmaceutical community, to reach out to those who do clinical trials. lung map was cited several times. the american cancer society is grateful to con tribute to that. but the fda has become an active partner with the process. so i don't know if that is a best practice or a best example but it's that source of communication. but let's not forget it's also the opportunity. because we're now in a place

that we only dreamed of just a short while ago. >> chair thanks the gentleman. chair recognizes the ranking member five minutes for questions. >> my questions are of mr. beall. the cystic fibrosis foundation has done great collaborative work that has resulted in successful marketing of -- and recent positive complimentary drug that may extend treatment to half the patients with cf. but i want to ask you about a couple of points in your testimony. you talk about the cf foundation's strong relationship with the fda and the important of bringing good data to the table when consulting with the fda which i know is true. i want to hear more about that relationship. we are hearing about the need for the fda to seek and

incorporate patient input into the review process. can you tell us more about the cf foundation's interaction with the fda and are there lessons that can be learned by other disease groups? >> i can give you a perfect example. on wednesday of this week we had three officials including dr. robert temple who is in the drug division at our offices talking about the development of clinical trials protocols of drugs that might not into clinical trials three to five years from now. this is an example of this ocean discussion because we have a natur natural history of the disease. with you know the drugs and the mechanism of action. we have a safety profile and now we can start to talk about the future. and i think it's that kind of example. and that goes back many, many years. soon as we discovered the cf gene we talked about gene therapy and we had extensive dialogues with the fda not only

with manufacturers but the fda and the foundation. we have always had a wonderful collaboration. we have data. we have natural history of the disease because of the patient religi registry and we come with data and the experience and the networks that can make things happen. that was the example. >> we are hearing about the various expedited drug review processes at fda. and it is clearly a push by many to get agency to use these path ways more frequently and more disease areas. i think we need to be cognizant of the risk that could accompany that speed and we have to be concerned about the risks if we require more of these expedited path ways through approval. you also described that these

approvals could hinding the progress of other treatments. can you just o'lab rate on those concerns. >> when you are dealing with a small population when you are talk about personalized medicine with 25 patients who may be approachable by the therapeutic opportunities for that drug. if those patients were introduced a drug that was less than effective, what happens when the next drug that could be effective, how do you do the clinical trial? i think that is really very critical. we want to make sure our first introduction are drugs that are efficacious and move to the next level. if you don't have safe drugs of developing good drugs and effective drugs that could move us above the therapeutic options that we have. i think that's the critical thing that we have faced.

there is always a risk but it's now we are dealing with small populations, personalized medicine. i think you have to be particularly critical on that issue with rare diseases. >> okay. yeah, i just wanted to echo another point you made in your testimony about the importance of resources. and i couldn't agree more that as you say, fda needs resource so they can rely on the best regulatory science available and they need adequate resources to enable them to basically -- to meaningfully engage with the patient community. and you know, we have this 21st century cures initiative which is progressing now. we have had larger meetings and now hearings. and my colleagues always ask what request congress do? and i think that the most effective thing we can do is

provide adequate resources to make sure that fda as well as nih have the resources to fulfill the expectations we have for both agencies. i hear not only from the agencies but also from my constituents that, you know, they don't have enough resource. so i think that's -- i just want to echo again what you said. thank you, mr. chairman. >> chairman thanks the gentleman. now recognize the vice chairman of the sub committee. >> can i make a unanimous consent request? >> proceed. >> i would like to move that the committee make people aware that if they wish to contact the cures initiative it is cures.mail.gov. >> without objection, so ordered. >> very well. i knew they wouldn't deny me. let me ask you, mr. furlong, you

were kind enough to mention the work on the md care act. thank you for. that we will likely be marking that up next week. that is a big milestone. can you talk about how the md care act needs updating and type of updating that this committee has pursued? >> certainly. than thank you for the question. the md care act was the solid foundation that set duchene and the muscular dystrophys really galvanized their progress. so the md care act was passed and enacted in 2001 and reauthorized in 2008. and the amendments are really to look at what we have learned in the meantime. so the cardiac issues are real and they have to be tackled in order to answer the question as you look at these therapies that potential therapies that were hopeful to be approved they extend function.

will they protect or have a negative effect on the heart. it's the gaps that we need to really look at with the amendments. in addition to the fact that when this legislation was enacted in 2001, young boys with duchene didn't live to be adults. now we have an adult population and we have to address those adults in terms of their medical care and how to make sure they have long and independent lives. i think the md care act is look with the muscular dystrophy committee from the nih and other agencies. the research plan has to be updated and the amendments to be incorporated so we can achieve the full effect. >> and mr. beall will also acknowledge that the population of patients is changing because of some of the successes that has happened over the past several years. and in both of those illnesses, both cystic fibrosis and

muscular dystrophy, it is important that we keep pace with the way the patient population is change. we want people to live longer and fuller lives with their conditions and at the same time we don't want the legislation to stymie that. it's my opinion, an important step forward. dr. beall, you talked about the development of mutation specific therapies and the next evolution in precision medicine. and you can see the cystic fibrosis example impacting the way we address other serious illnesses. is there something more you would like to add to that. >> we are in the age of personalized medicine. fortunately with the completion of the human genome. we understand the genetic diseases it is a critical time for us. dr. collins downstairs, he is excited because he was one of

the discoverers of our cf gene. we live in a unique age. more and more therapies are going to be developed towards specific mutations. that's why we have to have patient registries. when they felt they had a drug that could work on one mutation we were able to tell them in the united states we have 1100 of those patients within five minutes after they asked us because of a patient registry because we have a documented history of the disease. i think that's why it is important to have personalized medicine therapies and the options for that. but we have to be able to document the patients that can participate in the trials. >> it is very powerful. in 1965 you didn't know we were going to know about the sequence of the human genome 30 years

later. >> but today we cover -- we have 26,000 patients whose data is provided to our patient registry every single year. >> let me ask you a question. i'm going to run out of time quickly. the world is different now. and you have people that are, perhaps, lucky enough to enter into a clinical trial and likely to have friends with the same condition. in the old days, randomized clinical trial. you don't know what arm you are an domized. who is getting the study drug or who was getting an older therapy. but now people communicate with facebook and twitter. how is that going to impact the ability to have a blinded randomized clinical trial? are people likely to communicate with each other? i'm getting better on this stuff. how about you? i haven't seen a thing?

>> i think it's a real question. it's a real issue. and you can't pretend it's not going to happen. it is already happening especially with large cohorts of patients in multiple countries. they are all communicating. it is important we are regular rouse in maintaining the blind to the extent we can. >> and we have to embrace the fact that the information is being communicated and further enhance it. >> let's take advantage of it. let's do more in the after market and collect this information and get a more nuanced view of the drug's used in the real world and turn it to our advance. >> and in some cases will make it easier for clinical trials. when you is the large networks and they can report patient reported outcomes and things like that. i think it's a disadvantage sometimes but we ought to turn it to an advantage. i think it can expedite the ability to do clinical trials as

we move forward with the technology. >> and post talk analysis. in a clinical trial of 12 months you might not see the effects of the drug. so we can understand the full impact on the patient's life. >> gentleman now rec mr. schakowsky, five minutes for questions. >> thank you, thank you very much it i had a question for dr. lichtenfeld. and actually for anyone on the panel that wants to comment on this. this is about quality of life outcomes. i mean, obviously, if this is a known life threatening disease you want to do everything you can to make sure that the therapies match the disease. but there are you say when these

quality of life outcomes are rigorously measured and supported by the fda they should be included in the drug's labels and be the basis for a drug's approval. i certainly know people who have suffered so much from side effects of drugs and i just wonder in the whole process of drug approvals, how much are these quality of life issues really -- really looked at as abe sis for approval or just as abe sis of whether or not they're used? >> thank you for your question, in fact it's a work in progress. let's understand that quality of life is a buzz word today but it wasn't a buzz word very recently. so as we look at the issues of pal yative care or quality of life issues that the american cancer society and many others have been involved in it's relatively new to the table.

having said that there have been issued recently with a drug -- one particular drug, where the question really centered around was the -- even though the drug may not have met the fda standard and this was about two years ago, even though it had not met the fda standard did it improve the quality of life -- happened to be a breast cancer drug, for the women who took it. that would have been an important consideration. and unfortunately the quality of the data measuring quality of life was inadequate. so going forward, cancer patients have enough on their plates as do the -- everyone represented at this table as do patients throughout this country. we need to be aware the quality of life is an important part of the treatment process. and we need to have tools in place that are not uniform yet. they're not as good as we would like but they have to be in place to measure quality of life and that has to be considered.

and patient reported outcomes are a part of that process. >> go ahead, doctor. >> i want to make a comment as it relates to patients with chronic diseases as well. we have talked about cancer and rare diseases. we work where patients are taking medicines for a long period of time. and simple things that may seem pro stayic to the researcher like nausea, fatigue, these are really important inputs we want to hear from patients about. >> is that part of the process? >> it's less part of the approval process today than i think it will be in the future. certainly part of the utilization process. patients say which medicine do i want to stay on for years and months but it's not incorporated in the approval. >> especially where all things might be equal in effectiveness,

whether or not something causes nausea. >> that's right. >> fatigue. >> could be really important. >> particularly for launching a new medicine into a large category where there might be generic drugs that might be safe and effective but not hit all the parameters for subsets of patients for a long period of time. from the patient perspective there are differences between the medicines. >> i also -- dr. lichtenfeld, wanted to ask you about small-sized trials. and you mentioned one of the drawbacks. i had talked about the extent to which women aren't considered. and i would just be concerned -- i understand the plus. i do. but if we rely too heavily on them, isn't there the real risk of excludeing important populations. >> yes, there is a real risk. in fact when you talk about

women in heart disease i remember back in the 1990s when the article came out talking about the absence of women in clinical trials for the treatment of hyper tension and heart disease. but let's talk about the other side of the coin. and that is when you're sitting -- and i have sat in the presentations at asco at the oncology meetings and you see a presentation of 80 patients and you see what we call a waterfall spot the responses in survival that occur and suddenly 70% of those patients are having significant responses. i don't think -- in a disease where there was no treatment before, i don't think one asks the -- they ask the question in followup but not at the moment. and what has happened and what is exciting to me is i'm now sitting in those presentations every june, and i see -- i actually wrote about it. it took a year for one of the

drugs to go from clinical trial to approval because it was that effective. that's spectacular. that's new thinking and a new approach. we have learned more as time as gone on. yes. does it mean we stop learning as was mentioned before with cystic fibrosis. but when you see moments like that, no one would want to hold back. develop the data but don't hold back the opportunity. a phase i trial that was reported at asco in june. the asco was willing to put it into use. that is a change in the way that we have seen cancer drugs move through the pipeline. >> thanks . >> dr. lichtenfeld let's pick up there. that compassionate use is

something we have to figure out how we can make it more effective and do it faster at the federal level. you talked about in your testimony that patients needed to be involved both on saying what kind of nausea they had and what the pain level were but particularly when you had no treatment patients need to be involved in that too. and when there isn't a treatment, you're much more willing to take those risks than you would be if there is some other treatment out there that might work but this might be more comfortable. and i want to give you an opportunity to address that further. >> i appreciate the question. i know you mentioned it earlier. is it a complex issue. it is not a new issue. it's been around for some time with the number of drugs that have gone through -- through the pipeline which seem to show some opportunities. there -- and i know it is

various state legislatures are involved. i'm sitting here today, both as a representative of the society but also as an individual. and understand that there are discussions on both sides of that issue and they're complicated. we need to understand what drugs work when they work. and patients need access to promising drugs as soon as possible. companies make decisions on how they handle that process. the fda approved restroom all the applications they received and we have to have those discussions about how to address that issue. >> and what i would say is whatever we can do, i think, i speak for a lot of the members of the committee i'm more out there than some. but whatever we can do to help by changing the law to expedite that process we will do. >> we'd be glad to have those

discussions. >> and did you want to make a comment about risk assessment? when your boys were sick you probably would have taken anything that had any promise of hope. >> i can telling you stories of looking in china for tea that you might be appalled about. this is really up to the companies. fda has always to my knowledge in the duchene and other fields being been willing to talk about compassionate use. for rare disease community this really talks about and gets us back to trial design. in general, trials are designed to test a small subset of patients. the six-minute walk test is the standard outcome measure. as a child with duchene you have to walk for six minutes and it's in a very narrow subset of people within that six-minute walk test leaves a great numb of

people outside the trial. the trials have to be inclusive and welcoming of people that live with the spectrum of the illness very young as well as adults with limited functional ability so we can test those in those populations and provide labels that are broad and have access to all. >> look forward to working with you all. i'll yield my time to dr. burgess. >> i wanted to free throllow up you. your speciality has been involved in this longer than any branch of medicine going back to 1955 when developmental therapeutics program was put into place. with that lodge tuesday -- that breadth of experience in your speciality are there things you want to share with others? >> what we did back in 1965 or

whenever was a lot different than what we are doing today. i don't want to take the time to go into it. you may be aware of it. but here's the message. it didn't happen overnight. it took 40 years of research to get us to the tipping point where we understood the genome and had the opportunity to take advantage of that and move forward. immunetherapy the same story. i don't want to underestimate the value of research investment where suddenly we look like we have so much to offer and to do. i also comment with regard to my co-panelists that finding the patients where they are is critically important. we have to understand not only the clinical trial membchanism u but the medical practice system. that my friends have opportunities to get these drugs

in clinical trials and be a part of that process. there is a lot of work to do. >> chair thanks the gentleman. now recognize the gentleman from new york mr. engel. >> thank you mr. chairman and mr. pallone for holding this hearing. i'm pleased to have this opportunity to further consider how patient perspectives can best be incorporated into a therapeutic development process. as the author of the als registry act and the amendments of 2008 and 2013 along with dr. burgess i have worked to be a voice with those with rare and orphaned diseases. i'm encouraged with the advancements we have made as well as our progress toward treatments but obviously we still have a long way to go. one of the most striking gains is for those with duchene muscular dystrophy. our efforts have added an

average of ten years to life expectancy of boys with duchene and now we have challenges in finding effective therapies. patient community brings an important perspective and understanding to this process and i'm interested to see how we can best use that knowledge to assist medical researchers with therapy development. let me ask you this, i'm particularly interested in the way the duchene patient community is engaged with the fda to inform the benefit risk determinations made by agency reviewers and the duchene community guidance document you referred to. can you please comment on how you hope to see these efforts affect the pipeline and the stake holders who are a part of that pipeline. >> the benefit risk origin theyed out of discussions with the fda. in our early discussions we were telling anecdotal stories that

the benefit risk was different than in a more common disease and the fda suggested they agreed but didn't have anything they could rely on. any quantified evidence based document to help them make those decisions. we agreed to go out with the pilot on 120 parents and their priority was disease stabilization and they were willing to accept a great deal of risk and living with a great deal of risk. the fda has asked us to expand that study to a greater number of patients and to ask these questions of the young men with the disease. we hope they will incorporated into the review process and demonstrate how they use it and when they don't and what makes sense to them as they make their decisions. >> thank you for your advocacy and hard work. it's appreciated. dr. summar can you talk about the role you think that patient

perspective should play in developing therapies for diseases like als and muscular dystrophy that have limited treatment options and for which quality of life is obviously and especially important factors to patients. how can the fda best consider the views of patients and families when examining the benefit risk calculus for these diseases? >> this really expands across the entire field of rare diseases but your question cell vent for those two groups. patients often tell us about things they wish were better than we never thought of. when we ask our patients what is the worst part of this diseases, a lot of times it's parents in the case of pediatric patients. sometimes the things we thought were most important are nine or ten or the lists. when we look at therapeutic targets and quality of life targets for these diseases,

patient and family input is a huge factor. and i think it's something we can incorporate better than we have. when we are designing our pivotal trials, clinical trials looking at what end points are i think they will be more and more important. and the other thing is the small group sizes. it's hard to pick one outcome that you can achieve. the smallest study i have been involved with is five parishes for an approval process. getting one target for that. the effect of the drug was massive. but it had been a milder, i might have needed more than one outcome variable. so i think families can help us determine what is important there. they can help us also as we talked about what risk is tolerable in those situations. it's different. there are 7,000 different rare diseases. each one of these is unique in its own regard but there are commonalties like.

that. >> thank you. and thank you for your comments and also thank you for your interests and i want to thank the panel for very interesting discussion. thank you mr. chairman. >> chair thanks the gentleman, now recognize the gentleman from florida, mr. bilirakis. >> i appreciate it and thank the panel for their testimony today. i know we've been talking about this and you've had an opportunity. i want to give you more of an opportunity to respond on this. on wednesday i asked one of the witnesses about his statement in including patients in the clinical trial process but i want to make sure you have every opportunity to respond to this. if patients had a greater role in clinical trial design and you touched about this. if trials measured equal davive data from patients like how do you feel? is it less painful, what have you, how would things be definite and what would you like to see?

we'll start with -- >> i'd like to start. first of all, the patient reported outcomes i think has been part of every clinical trial in cystic fibrosis for the last 10 or 15 years. some of the tools are not the best at this point but we are working to refine them. we have spent a large effort to look at patient reported outcomes the specific validated tool for cf. and it's going to be submitted to the fda and go through a validation process. in the past we used one that is generally f generally for lung disease but not specific. a decade ago or 20 years ago pros were not incorporated. it's a science that is evolving. it has be to be evolved at the fda and with the sponsors too. they have to be willing to incorporate those into the clinical trial process. i'm encouraged by the process.

but i will tell you in this last trial we had where the lung function went up and the sbeser bagss went down we didn't have a significantly improvement in the patient outcomes. when co-you are starting to treat the basic defect you are treating the whole disease process and the patients may not feel that from day-to-day. but over years you may have a tremendous impact on those patients. so it's a tool that can be used but shouldn't be used exclusively. >> thank you very much. >> so i agree with dr. beall. patient reported outcomes are important. but this is where involving the patients in the design and conduct of clinical trials is going to be important. how do we measure energy and endurance? how do we know that turning over in bed is important as opposed to the six-minute walk test. i think things are important to patients with a ripple effect in their lives.

if a boy can text at the age of 18 that gives him independence. if a child can walk up a single step they can enter buildings. if a child can roll over in bed that makes the family's quality of life much better. including the patient voice in the discussion about what the clinical trial looks like and what the measurements are is going to be incredibly important. >> thank you very much. mr. pops do you have a comment? >> i think these outcomes are so critical in the world we are developing drugs in which is in psychiatry, schizophrenia, the hard end point of the clinical trial is asking people how do you feel? and that is in a validated scales. but those doesn't incorporate how they feel over time. the perfect example might be in an opioid dpengs when a critical

question is my craving going to go down. it may keep me from drinking but is my craving going to change? that is something we couldn't incorporate in the label but it is essential in the perception of the disease. >> doctor? >> at asco they showed a picture of a lady who was 96 years old who had received a phase i drug whose cancer completely resolved and on the bottom end of the before and after picture. on the after picture you saw a trace of a little smile. i noticed the smile. i tweeted it. and the lecturer said yes that is really a smile in front of 2,000 people. what i'm trying to say by that example is that is what we have to be able to measure and

aggregate in a scientific way to show that the treatments make a difference. one example of one lady in an unusual situation, but something that i think all of us would agree. is so critical to understanding and particularly in oncology world what we do and how we do it and the goal that we have to have of improving quality of life. >> thank you. dr. summar? >> just another example too. we had a new medication we're looking at. most people with rare diseases they are on the medicines for life. day in and day out and the care is complex and affect the whole family. the new drug looked promising with a little bit better efficacy and control but five times a day instead of two or three times a day and the families are like why would we add three more times of day of dosing for this small effect and no one bothered to ask them that

before we started. so i think there is all these things that get the patient input early on is going to make a different. >> thank you so much. can i ask one more question? >> you may proceed. >> dr. bilirakis, the cf foundation's venture has produced incredible results. your breakthrough drug was found when it helped translate some of the early research through the value of death and now you have the --. it sounds similar -- i'm sorry. how are you able to establish this program and how can other groums adopt a similar model? >> well, it's a willingness to take risk and that's what you have to do in drug discovery. and we were frustrated by the fact that companies were not getting involved in the orphan diseases. so the concept was to take the risk out of biotech companies to

get engaged in cf research. we spent $42 million to start a high throughput screening that led to the drug. and what is great fight. ms. furlong was in my office a number of years ago. what we are seeing is so many other organizations are feeling the same impatience that our foundation felt 14 years ago. and adopting this. when i talked about venture flanthpy, we had ten people in the audience. and now it is inherent in what many voluntary health organizations are doing. faster cures has been an organization and is central to making some of that happen. there are law firms that specialize in it. we share ideas all the time. and it's been very gratifying to our community that we happen to be fortunate enough to be able to start it because we had the sources. bill and plen da gates gave us

money to make that initial investment. >> can you tell us how you established the registry? >> as i say it goes back a long time. but the doctor was here several years ago when he was the head of the nih and he said one thing about the cf community it's a community with a culture of research. and every patient who goes to one of our care centers is asked do you want to participate in a patient registry. and 99% of the patients say yes, i do. it's part of the culture the organization creates and the physicians and the relationships and the recognition that it's important part of being -- having a disease because we can't cure this disease without their involvement. >> very good, thank you very much. appreciate. i yield back.

>> chair thanks the gentleman. dr. summar, on wednesday we heard an idea thrown out there that there are vast amounts of data available that are not being utilized. and we all know what an organ donor is. the idea was that we have data donors. now, how would this play and you mentioned the irb system. what is your reaction to that? >> this is something we talk about when we're having coffee a lot. there are data sets all over the place. most of them are usually lost when someone's computer is recycled. we had a physician lose 15 years of data because his excel spread sheet didn't update to. find a way that balances, obviously people's design for confidentiality versus the irreplaceable amounts of data

that are out there we have to find that balance. i would love to find a way forward with. that that one's going to -- you can see a lot of sides to that question. but i think it's worth looking at. and what we find is a lot of patients are like, put it out there. i'm fine with that. there will be a small core that won't. you can take account for that. but most folks if you ask them, would you be okay if your data is out there, they would be fine. you can see people who had their genome sequencing published with their health history. >> i did that. that is a real thing that is happ happening right now. the voluntary relinquishing to the greater good is something we ought to encourage -- or not stand in the way. and unfortunately and i can't

say that we don't always respect that that we shouldn't stand in the way. but enough about that. >> all right. the chair thanks the gentleman. that concludes the questions of the members who are here. on exciting and informative haerg. thank you for coming. members will have follow up questions and we'll send those to you. we ask that you please respond promptly. i remind members they have ten business days to submit questions for the record. and that means members should submit their questions by the close of business on friday july 25th. i have a uc question, a statement for the record from the national health council. without objection that will be inserted into the record. without objection the subcommittee's adjourned.

[inaudible conversations] half way through the month of july, the house comes back

for a full week next week starting with reauthorizing the highway bill, the ways and means republicans are tweeting about the debate on that bill. they say we just approve hr 5021 in a bipartisan way. we are joined by lauren french. so on that highway bill how bipartisan is it and how similar or dissimilar is it to what the senate proposing? >> it's not very bipartisan. you have republicans in the house back it. it is a temporary extension of the highway bill. it will give about eight to nine months of extension for those funds. and you see nancy pelosi the minority leader saying it would put america in a rut. democrats are not thrilled is it a temporary extension instead of a permanent solution to the funding crisis. and you have seeing differences between the senate and the house version. the house version is temporary and extend it through next may.

you will see the two sides hammering out their differences even though the house is scheduled to vote next week. >> you are writing about the rules committee meeting on authorizing a lawsuit against president obama. here's the headline, determines tap witnesses in possible boehner lawsuit. what will that rules session look like? >> you will see both sides harshing out the argues of why they can or cannot sue the president. you have the democrats who are bringing in high profile witnesses, a former assistant attorney general, you have simon lazarus and they are going to argue this suit has no standing and the house republicans should not be allowed to go forward. and you is two noted constitutional experts. jonathan turley and elizabeth foley who is a professor in

florida and they will argue why the house does have standing to sue the president over his executive actions on the employer mandated obamacare. >> they are churnging through the spending bill. what is ahead? >> the irs appreciations is going to come up. this is a chance for house republicans to punish the irs for that tea party targeting controversy and other controversies. so decrease funding there is ahead for the irs. >> a headline shows john cornen and texas gop to obama, don't mess with us. this is part of the statement that the senator was down in texas meeting with governor perry and other. when will they hold a hearing on that? >> that's still to be determined. you have republicans saying they're not thrilled with that amount, the house appreciations committee chairman thinks that

number is house committee chair says he thinks that number is too high and a number of republicans says they don't feel the rush to fund that as it is. they will look to see what has already been allocated. what needs to be allocated immediately and what could be aallocated next year. even if the $3.7 billion figure is right or whether it is too high. so that's going to continue to be a huge source of debate between democrats and republicans next week. >> one more issue, the democratic leaders unveiled the response to the supreme court's hobby lobby decision. this is the tweet from senator reed who said that next week we'll vote on legislation. >> the house d.c.s will overrule the supreme court's decision which said that for profit businesses with religious exemptions do not need to

provide firms of birth control for women. for profit businesses no matter the religious affiliation have to provide or account for all birth control options in their health care plans. >> lots to cover in the coming week. lauren french covering congress. you can read your work, she's also on twitter at lauren french. thanks for the update. >> on the next washington journal, the american civil liberties union on the class action lawsuit filed on behalf of unaccompanied minors facing deportation. one recent supreme court rulings regarding freedom of region. former home land security inspector general on efforts to improve security at oversea airports with direct flights to the u.s. begins live at 7:00 a.m. eastern on cspan.

>> baseball does strike me. i don't want to get meta physical about this. i'm the anti-meta physical school the baseball but it's a good sport to be the national past time of a democratic nation because democracy is about compromise and settling. you don't get everything you want. baseball is like that. there's a lot of losing in baseball. every team that goes to spring training know it's going to win 60 games. knows it's going to lose 60 games. you play the whole season to sort out the middle 42. if you win 10 out of 20 games you're mediocre. if you win 11 out of 20, you got a good chance to play in october. it's the sport of a half loaf as it democracy. sunday night at 8:00 eastern and pacific on cspan's q and a. >> now you can keep in touch

with current events from the nation's capitol using any phone any time with cspan on audio now to 202-626-8888. every weekday listen to a recap of the day's events at 5:00 p.m. eastern on washington today. you can also hear audio beginning sundays at noon eastern. cspan radio on audio now. 202-626-8888. long distance phone charges may apply. >> this week whistle blowers who spoke out against mismanagement at the department of veteran affairs talks about the retaliation that they received from supervisors and coworkers. the va says they are investigating employees who reports health and safety lapses. this hearing is two hours and 45

minutes. this hearing will come to >> good evening everybody. this hearing will come to order. i want to welcome everybody to tonight's hearing entitled va whistle blowers. exposing inadequate service provided to veterans in insuring appropriate accountability. i would also like to ask unanimous consent that representative tom price from the great state of georgia bea l allowed to join us. without objection, so ordered. tonight we will hear from a representative sample of the hundreds ever whistle blowers who have contacted our committee seeking to change the va to improve patient safety and

better serve veterans who have served our great nation. we will also hear from the office of special counsel regarding its work protecting whistle blowers and the vital information they provide. representatives of va will also be hear to answer for the department's reprisals against whistle blowers and its continuing failure to protect employee rights to report waste, fraud, abuse and mismanagement to the inspector general, to the counsel, to congress, and to this committee. it's important to emphasize that the national scandal regarding data manipulation of appointment scheduling did not spring forward out of thin air at the department of veterans affairs. deceptive performance measures that serve as window dressing for automatic bonuses have been part of the organizational sest

pool for many years. the focus instead of being on veterans has been on serving the interest of the senior managers in charge. the manipulation of data to gain performance goals is a wide spread cancer within the va. we've also heard that va is a data rich environment. when it's exposed as vulnerable to manipulation, it cannot be data that is trusted. until recently va would continue to trout out the tired saying that patient satisfaction exceeds the private sector. that may be true at a few select va centers, however as our colleague demonstrated, such results have been overgeneralized. more over during the course of the past year, this committee has held a series of hearings showing a pattern at va of

preventible patient deaths across the country from pittsburgh to augusta to columbia to phoenix. va's satisfaction results are refuted by these tragic outcomes. every one of these locations, whistle blowers played a vital role in exposing these patient deaths at the department. whistle blowers served the essentially function of providing a reality check on what is actually going on at the department. at great risks to themselves and their families, whistle blowers dare to speak truth to power and buck the system in va designed to crush dissent and thereby alter the truth. tonight we're fortunate to have three distinguished physicians testify as to regard with their experiences with the va. we will also here from a conscience program manager who will show that the disease of data manipulation may have

spread to the initial eligibility determination for medical benefits. none of these whistle blowers lost sight of the essentially mission of the va. the mission to owe serve veterans. they understand that people are not inputs and outputs on a central office spread sheet. they understand that metric and measurements mean nothing without personal responsibility. unlike their supervisors, these whistle blowers have put the interests of veterans before their own interests. unfortunately what they all have in common is the fear of reprisal by the department. they will speak of the many different retalatory tactics to keep them in line. it is time that va embraces their integrity and recommitted itself so to provide quality

health care to vert ans. i have asked my staff to improve whistle blower protections for va employees and i invite all the members of this committee to work with us towards the end. with that, i now yield to my good friend and ranking member for any opening remarks that he may have. >> thank you very much mr. chairman. this committee has held many hearings over the last years with problems with va health care. problems with disclosed and the va promised to improve. little has changed. va is widely known to have a culture of denying problems and not listening to feedback by it from congress, veterans or its own employees. the department of veterans administration has had a

representation as being intolerant of whistle blowers. so far in this fiscal year, nearly half of the matters transferred to agency heads, seven out of 15 involved the va. according to the osc, it currently has 67 active investigations into retaliation complaints from va employees and has received 25 new whistle blower retaliation cases from va employees since june 1st of 2014. a recent new york times article stated that within the va there was a culture of silence and intimidati intimidation. acting secretary gibson recently stated that he was deeply disappointed not only in the substantiation of allegations raised by the whistle blowers but also in the failures