misleading. sounds like it facester than regular approval. actually if we approve, give regular approval on a surrogate, it is just as fast as accelerated approval but you don't have to do confirmatory studies afterwards because we already buy leave the surrogate. accelerated diseases where there is something missing you may be able to show when you replace that protein in the body you give the activity back to the person, right? so you may not have to show clinical outcomes. that is still a surrogate that we feel is good enough because we understand the problem that something is missing and you deliver an active drug to the site of action of where the problem is and that would be enough. . .

>> this is a hearing about the pat, patient involvement in fda drug approvals, and i think we can agree they deserve a seat at the table. when companies are developing drugs and medical devices within the clinical trial process. i have long been a supporter of the department of defense's congressionally-directed medical research program known as the cdmrp. it funds peer-reviewed research into breast cancer, autism, ovarian cancer, prostate cancer

and other diseases. and since 1993 the patients have been involved and have been attar of cdmrp, and they have a consumer reviewer as part of the peer-reviewed possible to represent the stakeholder community. and it's been very successful in combining patient perspectives and needs with scientific research and bringing those perspectives together. has fda, as you begin to consider improving patient involvement, have you looked at cdrmp to see if there's anything you can borrow from that in the drug approval process? >> uh-huh. we have not, and that's a good suggestion. so we'd be happy to do that. >> okay. you mentioned previously that the patient-focused drug development initiative that was included in pa do you have that -- padufa was designed to

allow fda to hear from patients on how a disease impacts their life. and i understand you're scheduled to hold 0 public hearings -- 20 public hearings. share with us who fda's met with so far, have you started those hearings, and if so, what have you learned already? >> well, we've learned the devastating impact, i think, of the diseases, of these different diseases on people's lives is just incredible. we had one on chronic fatigue syndrome, that was our first one, hiv, lung cancer, narcolepsy, sickle cell disease, fibromial ya, hypertension, and we plan to have 16 of these meetings completed by the end of 2015. but we recognize this is just a drop in the bucket of what people suffer from. so what we're trying to do is really model how people can do this and, hopefully, it could be done more -- not out on by the fda, but by the patient groups

themselves and the medical community that serves them so that they can assemble more of this information and kind of multiply the effect of this. and we're already seeing some of that. nod, for example -- nord, for example, has offered to help with rare diseases, for example, to have more input that way. because our resources are limited. we're not going to be able to cover all the different diseases. >> good. so i expect we'll hear from the patient organizations later this afternoon -- later today and their view on how they can be helpful and we can be effective. i think the wave of the future really is the information we'll be able to gather through the electronic health record. so it's interesting to hear what you've done already with the sentinel initiative. i've heard from research institutes back home that are doing so much in genomics and personalized medicine that they think these larger networks are

the wave of the future. you say you don't need legislation, additional legislation to continue, but you're having to borrow resources from this and that. >> uh-huh. >> so is your 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 also 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 people out in the community -- like cancer patients, most cancer patients in the u.s. who have diseases that are untreatable are not, don't get into trials because they're being treated at places that aren't running trials. we need to move this out into the community, make those folks eligible. and i'm on the steering committee of the lung map trial, and i've 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 this research and perhaps have a more effective therapy. so i think the electronic health records, that there's -- that's a huge, different area that we're working on in how to do clinical trials ute -- utilizing that infrastructure that is emerging. >> thank you very much. >> i recognize the gentleman from new jersey, mr. lance, five minutes for questions. >> thank you very much, mr. chairman. a portion of your testimony has focused on fda's efforts on patient engagement. it is my understanding that clinical trials.gov was intended to be a resource that a provides clinical study information for patients, for health care providers and for researchers. but it seems to me that the 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. >> well, i think that it has provided, along with the requirement of the medical editors of the journals, that things be registered before they're published. it's provided tremendously more transparency into what clinical trials are ongoing in the united states. and that's been a big achievement, all right? so we know that the issue of publication bias and minimized because we know what trials have been done. however, i agree that, certainly, for patients i think that initiation of trials and understanding where there might be a trial that might be ongoing that might be available to them has also been effective although, as you said, there are technological issues that remain. so 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 submission of clinical trial results for unapproved products. because much of the lag this getting results in there is products still are not on the market. so nih is the lead for this rulemaking, and i think they'd be in the best position, and also they operate the infrastructure for this database. >> thank you. in another area, in the past several hearings we have discussed the difficulty of various institutions

communicating one with another and a lack of coordination often leads to inefficiencies. what methods are currently this be place to reduce -- in place to reduce redundancies in clinical trials and what steps can we take to insure we are not doubling up on research and making the same mistakes over and over? >> um, hopefully, most things would eventually become, come out and be published. but certainly in the drug development area there's interest in more sharing of earlier data and sharing of failures. but this has proven to be a very intractable area -- >> yes. >> for transparency. [laughter] >> yes. >> but we've continued to work on that. in the prior hearing they were talking about some of the inefficiencies, say, of irbs where multiple irbs, they

might have to have 100 irbs -- >> yes. >> and i believe there are efforts to try and address this. it's not an fda issue, but really we came out a number of years ago in saying that central irbs would really be preferable in these large, multi-center 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've tried to develop model agreements ask so forth. but that's -- and so forth. but that's something that the standing trial addresses, because you sign this contractual agreement once, and then you can do multiple investigational agents. >> are we moving in the direction of central irws, in your judgment? >> yes. i mean, there's certainly a consensus in the clinical trial sphere, but various universities are concerned -- >> of course. >> -- about their own liability,

so there's a push and pull. >> i think this is an area we should investigate further 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. >> chair thanks the gentleman. gentlewoman from illinois, ms. schakowsky, for five minutes. >> thank you, mr. chairman. thank you, dr. woodcock. i think you're an excellent witness, i appreciate your answers. >> thank you. >> i wanted to go a little bit further on the problem that congresswoman capps raised about the underrepresentation of women. i know you've said that you found that, actually, women or were overrepresented, but recently the congressional caucus for women's issues sponsored a meeting with leading women, heart experts both clinical and research experts, physicians. those experts raised concerns that the lack of representation

from women in clinical trials is limiting our ability to effectively treat women with heart disease. they were focusing in on heart disease. and according to those experts, for the last 50 years women's heart treatment has largely been based on medical research about men. and even today despite that fact what they said is that the 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's health research institute in northwestern in my district have raised concerns about the disproportionate number of adverse drug effects that occur in women due to the lack of sex-based clinical research. and as you know, the biological, physiological, hormonal difference in males and females impact the rate of drug absorption, distribution, metabolism, elimination and, ultimately, affect the drug's

effectiveness. according to those expert, the lack of requirement for drug manufacturers to take this into account and document any sex variability early in the drug development pipeline before a drug has been released places consumers, especially female patients, at adverse -- increased risk of adverse drug effects. so i want you to respond to that. >> certainly. >> many of us were really left with a very disturbing feeling, because 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're mentioning is actually the fact that men suffer heart disease earlier in life than women. >> although let me just point out they also said that the growing number -- even though it's lower -- >> yes. >> -- is younger women getting heart attacks and heart disease. >> yes. so that the reason for maybe

maldistribution in the trials is because there's an age cutoff, and there always has been. in our survey we found that there were 19% of the people in the trial in these 147 studies we looked at were over 65 which is more than in the general population, obviously. but it is, of sick people that's still low representation, right? to say people with heart disease. generally speaking, there's often a cutoff, age 75, and we're trying to eliminate those cutoffs for age and con come plant conditions so that the population will be more representative. but to your -- could i -- to your original point, we required, we've always required male and female animals in toxicology studies, right? we require what we call kpd early in drug development, and we look at our, our clinical pharmacologists look at blood levels and exposure in men and

women, and we understand that usually. that's modern drug development. so there are multiple trials that are done that look at exposures and achieve blood level by gender and other factors -- liver failure, kidney failure and so forth -- and we can look at the phase iii trials to see if they're -- we, there's been a requirement in the regulation since, i think, 1994 that sponsors submit a gender analysis with their application. >> well, let me -- is this incorrect then? it says women comprise only 24% of participants in all heart-related studies. >> well, that may be true. that may also include medical devices. it also may have to do with this age disparity when onset of disease. >> i really hope that you'll look at that, because it's a great concern. it's a growing problem for

women, and let me just give you an example. if you said women, because we have different symptoms of heart disease, if you have some of these symptoms of nausea, dizziness, go to the emergency room but 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, i yield back. >> chair thanks the lady. chairman now recognizes dr. cassidy for phi minutes. >> [inaudible] >> thank you. >> i mean that as a big compliment. so next, real quickly because i want to talk about something else, but do, does fda -- you mentioned that some institutions may be nervous about their liability if they refer their irb activity to a centralized irb. >> correct. >> except so many do, we know that's a false argument. is there any way fda can reassure those institutions, because the gentleman there mayo

suggested it's a cultural issue. he didn't mention anything about legal. thoughts? >> right, yes. i heard his testimony. i think that, in my experience, that there are legal -- there are concerns of the counsel of the -- >> attorneys are always nervous, right? [laughter] i mean, they don't make money if they're not nervous, right? is. [laughter] cynical, but -- >> request yes. >> is there any way the fda can send reassurances for that? >> we have tried in sending guidance, and in this city initiative we had a whole discussion and dissemination of information about central irbs. but possibly there's more that we can do to encourage this. >> okay let me then go back to you mentioned something intriguing earlier, that there may be some at high risk for disease so, therefore, they will be more risk tolerant. >> yes. >> now, i have a family member, a nephew with downs syndrome, and i'm looking on the alzheimer's.org web site, and they mentioned how virtually

100% of adults with downs syndrome by age 40 will have evidence of the tangles associated with alzheimer's. >> uh-huh. >> now, what are the issues regarding -- wow, this is a group of kids, adults who are at risk, 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 credibly at risk for such a terrible disease? >> yes. well, with alzheimer's there are a number of problems. the basic rob is we still don't -- problem is we still don't understand the disease well enough and the interventions that have been tried when people are already demeanted have failed to work. >> as i gather, though, the problem is predicting at an earlier stage those at risk, correct? >> that's correct. if you want to intervene early. we recently issued a draft guidance saying, okay, if you

want to intervene earlier, we would accept an end point that is cog thinktive testing. >> i accept that, but how do you decide which population is such at high risk? if you had a control group, only 10% are really going to be at risk. >> right. >> you're with me. this is a really expensive study. >> that's right. and so we advocate techniques called enrichment which you try to use mile markers or other tests to figure out -- there are genetic conditions that increase your risk for alzheimer's disease. >> so speaking of downs syndrome is one example. >> that would be one example. there are others. >> and you give us the progress of that? so if you accept these, are people now using these? >> well, we need agents to use them in. so that's -- >> i'm sorry, agents -- >> i'm sorry, investigatal interventions that we can test in the people. and that's part of the problem. the science of understanding what causes alzheimer's and what you can intervene in that would actually delay or, you know,

prevent the disease is not mature enough, and we don't have really -- we've approved a couple imaging agents for alzheimer's, but they aren't 100%. and you would maybe be kind of -- >> for example, i know hyperi said anemia is thought to be a potential risk factor, and i think there are some studies suggesting that actose might give some benefit. presumably, it would be a nonmetabolic indication for use of actose, fair statement? >> uh-huh. >> now, there's at least some of that. i guess i pose that to ask to the degree to which that has been, again, the current state? i'll go back to what is the current state of using that kind of thing. >> right. so the current state we would, if someone decided to do a trial -- and i believe there have been some intervention trials in actose, earlier intervention at high risk and higher risk people -- they might identify people they felt were high risk for one reason or

another, randomize them, and then we would allow use of neurocock anitive testing even if they had symptoms. and if the treatment group did better than the placebo groom, we could -- group, we could give accelerated approval. >> so how long would such a study, do you imagine, take to complete its course? 20 years? >> no. no, but we need to have better measurements that get to these biomarkers and other measurements like of subtle cognitive function. the nih 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, but you can tell their brain isn't working as well as it should. and it'll decrease over time. so that's kind of the rate-limiting step. but i agree, prevention is very difficult because 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're not going to get whatever bad outcome. >> we're out of time. thank you very much. >> chair thanks the gentleman. recognize the gentleman from virginia, mr. giffords, five minutes for questions. >> thank you very much, mr. chairman. dr. woodcock, as others have said today and also what i have heard in some of our informal conversations is you not only do a good job as a witness, but you're doing a good job overall. >> thank you. >> i appreciate that, thank you so much forking here today. you and dr. cassidy had a consideration about lawyers. some lawyers are looking for a way to solve the problem, and so maybe we need to get some of those lawyers on your team and some of the corporate teams to solve the problem, figure out how we can make these things work because i do think it's important. as you probably know, i'm one of those who advocates that we try to move a little quicker this those areas where -- in those areas where we have problems that we don't have solutions for

currently and also favor be what is known -- favor what is known in some states, state laws as right to try where you have a situation where doctors have tried everything and folks are given a diagnosis they've got, you know, months to live or their condition is going to be fatal. i'm one of those people who believes that we ought to let them go ahead and try whatever it is they're willing to pay to do, because the fda can't protect you if you're going to die from something that might kill you. >> right. >> i mean, it's going to happen one way or the other. you might as well have the right to try something. that being said, i know there are a lot of issues surrounding that. i'm not sure we have time for that today, for that discussion today, and i know that there's another panel, and i want to hear from the antibiotics as well because they're -- patients as well because they're involved this the process. so respecting you greatly, i yield back my time. >> chair thanks the gentleman and recognizes the gentlelady from north carolina, ms. ellmers, for questions.

>> thank you, dr. woodcock, for being with us again today. you know, this is such an important issue. as you know, we had our panel on wednesday, and it 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 that we can be taking care of our patients more effectively. in your testimony and in the discussions that we've had today, you've touched on the biomarkers and targeted drug development to benefit disease populations, obviously. you know, as all of our representatives here, we all have constituents with rare diseases, heartbreaking -- >> yes. >> especially right now in my community we have, i have a very good friend with als, and i'm -- as i'm learning more and realizing we've had a number of

our members of our community diagnosed with als. so this is something that is very important to me right now. i'm just looking at the idea as far as the target approval process being appropriated and applied through the fda, it seems to me that we're looking at cancer and is hiv. where do some of those rare diseases fall within that? and, you know, you had mentioned 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 there, and what can we do to help make that happen? >> well, any rare disease would be a great candidate for a standing network, a network of experts. and i think you may hear more about this from the next panel where they're ready to evaluate any therapy that advances through the early, the nonhuman stages. so they can pick that up right

away and test it quickly. and that, and in the meantime, until that happens, they can get what we call natural history which i know sounds very wonky. but, you know, people were asking just now, mr. cassidy, how long does alzheimer's progress from presymptomatic to symptomatic. well, we need to know that so we can design the trial correctly. in rare diseases, even more difficult because nobody thoughs. usually they get experts together and they say, well, in my opinion, it usually takes this long. and they're usually long i because we've only seen a few people. we're encouraging this natural history studies. so what changes this als? what can we measure? can we measure something that gives us an indication that treatment might be working? and then as soon as a therapy becomes available, then you can rapidly get people into a trial, and there would be no delays because there's no delaying in als. >> right, exactly.

and that's, you know, obviously part of the concern. certainly, i agree with my colleague in talking about right to try, you know? 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 careful, carefully to patients and families. >> yes. >> and, you know, do you consider and give more weight -- i mean, that's one of our questions is, you know, how much weight are you giving to the patients and families? what -- there again from our perspective in congress, what can we do, you know, as we have heard everyone agreeing that we these to make a difference here, and we can move things forward. how open is the fda to if this possibility -- to this possibility, and what can we do right now to make this happen? >> well, as i said in my testimony in the beginning, you know, the medical culture has changed over the years. it used to be very

doctor-centric, and now it's patient-centric, and the fda culture is a medical culture. so that has changed at the same time, but slowly. so we've been working 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 actually has the disease, is going to take the drug. to answer your question what can be done, i think a lot of this needs to be done out in the community. the patient groups need to get organized and develop these. some of them are working with we curry in trying to use that mechanism to get more information available and so forth. we have gotten draft guidances from different groups including muscular dystrophy that really are a statement of this is what we care about, this is what we value, this is what we want you to look at, and we will pay extremely close attention to

those, and those are extremely valuable. >> thank you, dr. woodcock, and i yield back. >> chair thanks the gentlelady. we are voting on the floor. we have ten minutes left in the vote. we have three more questioners. mr. dwutly, reck knewed -- recognized five minutes for questioning. >> i'll echo what others said about your testimony, appreciate it. everybody's excited, both sides trying to, how we can do this better. and i've heard from a lot of groups, and i've heard from them several times that the oncology division seems to be one -- people really like to work with, and it works well. some of the other divisions are not as well to work with. and and i've always believed jack kemp used to say don't study failures and and point out the problem, let's look at successes and see how it can be replicated. so within your own agency you're having wonderfully successful rams, at least according to the feedback i've gotten, and some not as -- i mean, as the ability to work with. so i guess my question is, is there any impediment to saying,

hey, oncology is what we hear about more -- not that the others aren't -- is there any impediment to transferring to other agencies? is there something congress can do to make it easier, or is it just learning and moving forward? >> you know, let me tell you that ten years ago, you know, i heard a lot of negative comments about oncology, oncology group, right? now we have therapies that are so effective, they're just, they're really on fire. they see that for their patients they took care of, they're all oncologists, doctors that these new treatments would really have made a difference for those people. and so they're doing everything they can to get those treatments out. and i think what we need, we need the same kind of inspiring therapies in these other areas. and i do think the doctors, they're doctors, they're physicians. they care about patients in their disease area. and be this, this breakthrough -- i don't know whether you can see it here, but you see that other disease areas are coming up, and we are

designating in neurology and anti-infectives and psychiatry, we're designating potential breakthroughs, and so this type of thing will really help. also we, of course, we try to have a management structure, multiple mechanisms whereby we have consistency and uniformity of our approach and our procedures. and i think we do quite well in our procedures. but 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. >> well, thanks, and i have a bill particularly to put the same status for professional budget judgment status for alzheimer which would, we're going to spend in 2050 a trillion dollars. that's not just loss of income, loss of productivity, that's a trillion dollars spent on that disease. >> right. >> that's when i'm 86. so that's when our children and

grandchildren will be taking care of it, so hopefully we can have the same inspiration particularly in alzheimer's -- >> i can assure you, if we found -- if there are promising treatments for alzheimer's, we would jump right on them. >> appreciate that. >> chair thanks the gentleman, chair recognizes the gentlelady from tennessee, ms. blackburn, for five minutes. >> thank you so much, dr. woodcock. i have, basically, one question that i do want to get to looking at the qidp 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 qidps has the fda designated to date? how many products have actually been approved to date? and do you believe that the qidp is an important tez nation? >> it's absolutely important.

>> okay. >> we have granted 50 designations for 34 unique molecules, and in the last several weeks we've approved the first two medications that are designated, the first two antimicrobials. >> that's a lot. >> so that's 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'd be interested in that. >> well, we are very interested in the pathway that people call limited population, antibacterial drugs or other streamlined pathways for development that would be matched with some sort of symbol or logo that would enable doctors and other prescribers to recognize that it was from 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 tennessean, dr. marshall sommer,

who is going to be speaking on behalf of the national organization of rare diseases, so welcome, we're delighted you're here, and i will yield back. >> chair now recognizes the gentleman from florida, mr. bill rack -- bilirakis, five minutes for questions. >> thank you, mr. chairman, i appreciate it. i ask these questions a few months ago, and i didn't get a response, so i'm going to see if i can get a response this time. appreciate it if you can answer. can you tell me how many treatments were approved with notable biomarkers used for the first time? is. >> no. [laughter] i don't have that -- >> could you get that information? >> i would be happy to. it's a very interesting question. >> and then next question, have any accelerated approvals occurred with a novel biomarker a never-before-treated disease? >> oh, yes, all the time. and i can get that for you. i don't have it, again. >> how many new biomarkers did

the fda use in the last five years? can you get that for me? >> certainly. absolutely. >> thank you very much. i know we don't have a lot of time, so i'll yield back. >> thank you. there are two minutes left in the vote on the floor, so we're 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've been a terrific witness. >> thank you. >> members, we'll have follow-up questions, we'll send them to you, we ask that you please respond. thank you, and thank you for your patience. the subcommittee stands in recess. [inaudible conversations] >> so this house hearing taking break thousand as members are joining their colleaguing in the

house chamber for votes on a bill that's making changes to current business equipment depreciation regulations. you can see live coverage as those votes take place over on c-span. we plan to return to live coverage of this hearing when those votes are done. it'll where about 45 minute -- it'll be about 45 minutes or so. while the committee stands in recess, we'll show you a congressional gold medal ceremony giving the award posthumously to raoul wallenberg, credited with saving the lives of nearly 100,000 hungarian jews during the holocaust. house members make remarks. [inaudible conversations] >> please welcome our horned guest -- honored guests, members of the united states house of representatives, members of the united states senate and the speaker of the united states house of representatives. [applause]

>> ladies and gentlemen, the speaker of the united states house of representatives, the honorable john boehner. [applause] >> ladies and gentlemen, good afternoon and welcome to the united states capitol. we're honored to be joined today by members of the diplomatic corps, former ambassadors, representatives from the state department and the u.s. mint and leaders of the jewish community throughout our country. shortly before christmas in 1944, raoul wallenberg did something many of us have had to do; told his mother he wouldn't be home for christmas or home

for the holidays. i must send you my best wishes for christmas by this means, he wrote. i hope the peace so longed for is not so far away. the bottom of the note he added: lots of kisses to nina and her little girl. well, that nina, of course, was raoul's sister who is with us today as are several members of the family. thank you all for being here. [applause] by the time he sent that letter, wallenberg had saved more lives than we can count. he had done much of the work on his own, and his daring was so dumbfounding that the nazis aimed their guns above his head. they were perpetrating what churchill called the most horrible crime ever committed in

the whole of history. but before this man, they cowered. you could say he was like a comet across a dark sky, seen once in a lifetime. yet we know he burns on which is why we are all here today. in america's history only seven individuals have been made honorary citizens. the first was churchill. wallenberg was the second. to this honor we add the congressional gold medal, a tradition that began with george washington himself. this medal is a tribute to a citizen of the world, but it is really more than that. it's a commitment to honor the family and his memory and to tell his story and to always seek the truth. that is not too much to do. in fact, the least we can do.

for his deeds may be beyond our capacity, but his lessons are not. the answer to fear is always courage. none of god's children, not one, is alone. and, of course, honoring your mother. i hope you all enjoy today's program. [applause] >> ladies and gentlemen, please stand for the presentation of the colors by the united states armed forces color guard, the singing of the national anthem and the retiring of the colors. [background sounds]

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[background sounds] >> ladies and gentlemen, please remain standing as the chaplain to have united states senate, dr. barry black, gives the invocation. >> let us pray. lord god almighty, judge of all the earth, you are the sovereign lord of history. as we meet today to honor with a

congressional gold medal an incomparable humanitarian, raul wall p berg -- raoul wallenberg, we praise you, o god, for using him as an instrument of your mercy. we're grateful that you equipped him with the requisite skills and talents to become the right person in the right place at the right time. thank you for using him to organize and negotiate in order to save 100,000 jews from nazi extermination. today as we celebrate the contributions of a man who became a captive so that people

could live free, make us fit to become a liberating force in your world. lord, save us from the slavery of negativity and from the bondage of selfishness. purify our hearts and guide our purpose that your will may become our will. we pray in your sovereign name, amen. >> amen. >> ladies and gentlemen, united states representative from the 5th district of new york, the honorable gregory meeks.

>> mr. speaker, leader reid, distinguished members at the dais, my colleagues, all distinguished guests, i am delighted to join with all of you this afternoon for the congressional gold medal ceremony honoring the life of raoul wallenberg. it is my distinct honor to have played a role in the recognition of one of history's most unheralded heroes. i offer my deepest gratitude to ezra freelander, the international raoul wallenberg foundation and the many individuals and organizations as well as my congressional colleagues -- especially nan hay worth, my cosponsor of this bill who supported the legislation -- for bestowing this congressional gold m.d.ial upon raoul wallenberg. i did not learn of the

remarkable acts of raoul wallenberg in my elementary school or middle school or high school or college or each law -- even law school, for that matter. it was through entities like the international raoul wallenberg foundation, the american jewish joint distribution committee and other institutions and individuals that i became aware of the courage, resourcefulness and -- [inaudible] of this great man. the more i learned about raoul wallenberg, the more i was convinced that congress needed to acknowledge his legacy in an enduring way. in such a way that we could inspire future generations of americans by what wallenberg accomplished. the overwhelming bipartisan passage of the legislation reflects the undeny undeniable

inintent of congress to keep his legacy alive. i have had many moments of quiet reflection on the wallenberg story. as we look to tackle the violent conflicts and unthinkable oppression around the world today, i am reminded that even in the midst of the most grotesque acts of inhumanity one person can make a difference. regardless of any differences that distinguish us from each other, the moral courage of one person is sometimes enough to make all the difference for all of humankind. today raoul wallenberg's voice still echoes across the generations. his actions reverberate through time summoning us to muster the moral courage to do in our time what he and his colleagues did in their time. raoul wallenberg's legacy

challenges us to come together, to collaborate, to cooperate for humanity's sake and against today's threats to human dignity, human rights and human life. i'm honored to be one of the respond -- sponsors, the lead sponsor, to honor this great human being. thank you, god bless you. [applause] >> ladies and gentlemen, united states senator from new york, the honorable kristin gill brand. [applause] >> today we are proud to honor raoul wallenberg with the nation's highest civilian award, the congressional gold medal of honor. during world war ii, raoul wallenberg chose to leave his life of ease in sweden for a

diplomatic assignment in hungary which was then an ally of nazi germany. his assignment was the result of a recruitment effort by the united states war refugee board and the office of strategic services to try to save the remaining hungarian jews from the holocaust. in this effort mr. wallenberg succeeded beyond any reasonable expectation. he provided swedish passports to thousands of jews which literally made the difference between life and death. will wallenberg -- mr. wallenberg rented 32 buildings in budapest, raised a swedish flag and deplayered them -- declared them protected by diplomatic immunity. he housed, protected and saved almost 10,000 precious lives. mr. wallenberg's bravery and his will to act are an example to

all of us. according to an eyewitness, mr. wallenberg once climbed onto the roof of a train with jews departing for auschwitz, handing protective passes through the doors. amid threats from the guards, he then marched dozens of those the with passes to safety in a diplomatic convoy. as the nazi front was collapsing and adolf eichmann moved to kill all the remaining j well, s in -- jews in budapest, mr. wallenberg threatened hungarian leaders with the promise of hanging for war crimes if they carried out their plot. sadly and selflessly, mr. wallenberg was taking prisoner when the soviet army liberated hungary from the nazis and is presumed to have died in prison. when we look up hero, a

legendary figure often of divine strength, a warrior, one who shows great courage. while the word "hero" is sometimes used gratuitously, raoul wallenberg truly personifies it. his willingness to risk his own life for others exemplifies his outstanding spirit, dedication to humanity and the responsibility for all of us to speak out against atrocities. his enduring legacy lives on in the countless descendants of those he saved. i want to just close by reflecting on dr. barry black's area to open this ceremony. raoul wallenberg was placed there for a time such as this. all of us are placed where we are in our lives for a time such as this. we should never forget it. [applause]

>> ladies and gentlemen, mr. ben olander. [background sounds] ♪ ♪ mt. midst of the black storm storm -- [inaudible] ♪ came a breeze of cleanest air, air -- ♪ -- gave life so that --

[inaudible] ♪ and no one could crawl behind words like -- [inaudible] ♪ this solitary hand amongst us -- man amongst us now with glory to his name. ♪ became our gleam of hope in a world of guilt and shame. ♪ like a cliff in churning waters, he stood firm to lead the way. ♪ so that no one could -- or from truth go far away. ♪ the deserted, he took his last

stand. ♪ no shield upon his arm, no sword in his hand. ♪ but his words bit like steel on the slayers of -- [inaudible] ♪ and with cunning tricks, he snatched the victims out of their bonding ties. ♪ this solitary man amongst us, now with glory to his name; ♪ became our gleam of hope in a world of guilt and shame. ♪ like a cliff this churning waters he stood firm to lead the way. ♪ so that no one could deny, or from truth go far astray.

♪ ♪ when the light poured in, this freedom's sacred name -- ♪ a twin-headed dragon, he was stolen without shame. ♪ [applause] >> ladies and gentlemen, the democratic whip of the united states house of representatives, the honorable steny hoyer. [applause] >> nina, as you've noticed, i am not nancy pelosi. [laughter] she is out of the state, but she

wanted me to give you her very best and her great respect. mr. speaker, leader reid, leader mcconnell, my dear friend, the majority leader of the house of representatives, eric cantor, kirsten gillibrand, gregory meeks who has sponsored this legislation, foreign minister bilt, speaker westerberg, members of the raoul wallenberg foundation and ladies and gentlemen, i am pleased to be here. unfortunately, as i've said, leader pelosi could not be with us this afternoon, but she asked me to convey her greetings to all of you and to express her gratitude, nina, to you and the entire wallenberg family along with her congratulations on the presentation of this gold medal. thethe talmud teaches, and i qu:

whoever destroys a single soul, it is considered as if he destroyed an entire world. and whoever saves a life, it is considered as if he saved an entire world. raoul wallenberg saved 100,000 worlds, and the descendants of those whose lives were spared, because of his courage and conviction, number like the stars. today's gold medal ceremony is not only a moment to reflect on his heroism and his role as one of the most consequential, righteous among the nations. it is also an opportunity for all of us here to remember the lesson he taught us all through his example, a lesson as applicable today as it was amid the horrors of the second world war and the holocaust. and that lesson is for us never

to be indifferent. never to be a bystander in the face of injustice. never to say someone else will do the right thing so i don't have to. like his countrymen in sweden and those who have paid tribute to him in israel, in hungary and around the world, americans honor raoul wallenberg because we see in the man and in his incredible act of resistance a reminder of the same values that led our nation to fight for the liberation of europe during the second world war and to support movements for human rights, self-determination and democracy ever since. congress honored him by making him an honorary citizen in 1981, as the speaker has pointed out, and accepting a bust of his

likeness that now greets visitors in emancipation hall. today we continue the work of celebrating his life and his heroism for which millions continue to give thanks. and on a personal level, i am grateful to your brother, raoul wallenberg, for making possible the years of close friendship i was so very fortunate to share with an extraordinary american, an extraordinary hungarian, tom lantos, and still today with annette. and be all -- and all americans are indebted to him for tom's irreplaceable service to this country and support for human rights around the world. as a result of the many survivors like tom and annette who have worked to combat the forces of bloodshed and intolerance that marred their

early years, raoul wallenberg's works of saving lives continues to this day and will, we pray, continue for generations to come. .. someone who was the gold

standard, with the proposition that we are our brothers keepers. [applause] >> ladies and gentlemen, the majority leader of the united states house of representatives, the honorable eric cantor. [applause] >> mr. leader, leader reid, to my colleagues, to nina lagergren, other members of the walberg family, to all guests. it is an honor to join you today to recognize one of history's gentle heroes, and a remarkable man, raoul wallenberg. history has taught us that war and an overzealous quest for power can bring out the worst of mankind. throughout world war ii we saw the advancement of tyranny and

terror along with the destruction of cities across the globe. we also witnessed the greatest tragedy of modern times, the holocaust. like so many of you, i have visited and walked among the ruins and ashes of the death camps of auschwitz and birkenau. while there, i was dumbfounded by how evil can overtake humankind. the scale of horror was undeniable, which is why history demands that we are here and proclaim, never again. the same history has taught us that through suffering and sorrow we can find the best of mankind, the one man that we honor here today, raoul

wallenberg, provided strength and showed fearlessness while saving the lives of thousands of innocent people. he would eventually give his life to prevent men, women and children from entering the nazi death camps. those he saved were people he had met, and people he had never known. in the jewish faith we believe that god works through messengers. i truly believe raoul wallenberg was one of history's great messengers for freedom and peace. in the spirit of redemption, it is written in isaiah, the people that walk in darkness have seen a great light.

to those who were rescued by raoul wallenberg, he was their light that shines in their darkness. is gift to mankind, and for the peace of the world, will continue to be immeasurable. and the united states of america will remain forever grateful. thank you. [applause] [inaudible conversations] >> the time of recess has expire. we will reconvene at the

subcommittee on health and introduce our second panel, and on her second panel we have five witnesses. i'll introduce them in order of their presentation. first miss pat furlong, founding president and ceo of the parent project muscular dystrophy. secondly, mr. robert beall, president and ceo of the cystic fibrosis foundation. third, esther richard pops, chairman and ceo of alkermes. fourthly, dr. leonard lichtenfeld, deputy chief medical office, american cancer society. finally, dr. marshall summar, director, scientific advisory committee national organization for rare disorders. thank you all for coming. we'll each have five minutes to summarize your testimony. your written testimony will be placed in the record. ms. furlong, we will start with you. you are recognized for five minutes for your opening statement. >> good morning, chairman pitts, and members of the committee.

my name is pat furlong. 20 is ago i joined other pairs to form parent project muscular dystrophy two and duchenne from one of the many forms of martial distant and those common lethal this order back to solid. in 1984 i received the horrific diagnosis on my two sons, christopher and patrick, above of my sons are gone now. i waged his crusade in their honor. much has happened over the past 15 years to transform the duchenne landscape. and much of this is the direct result of the action by congress and this committee notably the enactment of the child health act in 2000 the muscular dystrophy care act one year later. since they can act was enacted we've seen about 10 years added to the life with duchenne. there's been an improvement in quality of life driven largely by the development and dissemination of caregivers so that all patients can be diagnosed accurately and as early as possible and provide with the highest quality of care. the act also transform the

duchenne landscape. what was just 12 years a mere barren field has involved into a robust area of research where therapies are in clinical testing and several others are in early stages of development. despite these advancement duchenne remains a disease without any fda approved therapies. most end up in wheelchairs, and only a few live beyond their late '20s. our community needs therapy and we need them fast. to achieve this goal, ppmd is like groundbreaking efforts over the past year to address two major impediments in our quest to into duchenne. one is a lack of regular understanding of patient and. perspective on benefit risk, and a lack of clear guidance or direction to the biopharmaceutical companies designed to meet clinical tough. ppmd partner with johns hopkins university to conduct the first ever scientific survey on benefit risks perspective. the survey involved 120 parents

of duchenne children, validated what we had known anecdotally for years, because duchenne is 100% fatal at a young age, many patients and families are willing to accept higher levels of risk in return for the prospect of potential benefit. date has been shared and was published in an academic journal. now the fda must ensure its readers apply this evidence to their decision-making process. another impediment to drug development, particularly in rare diseases, is the absence of a clear guidance from fda when it comes to designing clinical trials. small patient population, limited knowledge about the condition, a lack of accepted or validated biomarkers are some of these challenges. at the invitation of the fda, ppmd let a comprehensive six-month effort to convene key stakeholders, patients, parents, clinicians, researchers and industry to write documents that would address trial design and many other issues. this was submitted to the fda

last month marking the first time a patient group has led the development of such a project. the fda must step up to review the draft the average stakeholder input and issue a guidance document under the agency's name. while each of these projects is focused on duchenne, each also offers a model that could be applied to other diseases or other conditions, particularly rare diseases. and of other organizations will take on similar programs. so what can congress and federal agencies to moving ahead? worst you can make sure the patient perspective on benefit risk and other issues is considered by reviewers at the fda. one way to do so could be by establishing a non-burdensome step where reviewers would disclose how they did or did not take such information into account making their decisions on a drug application. this would shed light on for what many consider a mistress process and to be done in a very simple manner. second, i suggest a given greater focus on regulatory

science the the fda keep space with innovation. specifically nih could bolster support for regulatory science research and infuse it into people -- awards. in corporate and regulatory perspectives earlier in the pipeline and maximize the likelihood that candidate therapy will be ready for the rigor of the fda. finally, i would encourage greater flexibility in clinical trials, particularly rare fatal conditions like duchenne that have small populations. business as usual trial design simply cannot hit the mark when working with these populations. addition energy has traveled a great distance over the past 15 years thanks in significant part to the leadership of this very committee. leadership that continue on monday with action by the full committee on the md care act amendment. far too many families, my own included, this journey has not been fast enough. we stand ready to work with your committee to make sure the 21st century cures initiative and duchenne and someone other rare diseases.

thank you spent the chair thanks the gentleman you. mr. beale, you are recognized for five minutes. >> thank you for this invitation to present this test will. the story of cystic fibrosis is a story of determination of hope and optimism. the progress we're documented in our submission shows what is possible when a system works well when patience, stakeholders and errata the agency collaborate to develop life-changing treatment. cystic fibrosis is clearly a life-threatening genetic disease that affects about 30,000 individuals in the united states. there's been tremendous progress in life expectancy over the decades, in the 50s people with cystic fibrosis barely lift a elementary school but there are people that are living today with cystic fibrosis in their 30s and '40s, and some are even going beyond. we still is too many patients at very young ages. the increase in life expectancy is due in large part to

groundbreaking advancements and treatments made possible because of the cystic fibrosis foundation, our patient community, and our industry collaborators. two years ago the fda approved collide ago, the first drug to treat the underlying causes of cystic fibrosis in a small subset of people with the disease. hailed as a game changing it is transform the lives of those taking this drug. there's a perfect example of personalized medicine. i might mention that the fda approved this drug near record time, three months before the prescribed -- and most -- just to be suicide another breakthrough in cystic fibrosis. it happen when the positive data from a phase three clinical trial for a new therapy that is targeted at 40% of the cf population that this data was released by a pharmaceutical company. these products would not have been possible without the

breakthroughs that have taken place in basic research, and all the efforts that are foundation has made over the years. the cf community was thrilled to learn that the trapper just been showed a significant improvement in lung function, weight gain, in 30 to 40% reduction in exacerbation. that's the time to dedicate to the hospital or have id infections. this is clearly a game changer for these patients. vertex glances met the new drug application to fda by the end of the year for the treatment. what is exciting about this progress is that these drugs would not of been possible were it not for the foundation and our committee. our commitment -- is not the root of our success. it hasn't been easy and it hasn't occurred overnight. in 1965 we created the first patient registry in the united states, now a model for chronic disease.

because of this registry we have a documented natural history of cystic fibrosis. we have the mutation analysis on most of these patients as dr. woodcock referred to this morning. we have the ability to post marketing follow-up on these new drugs as they are introduced to the committee. the same year, 1965, we create a care center network. 90% of all patients in the united states are seen at the cff accredited and funded care centers. in 1989 through our support, 12 years before the human genome was completed. in 1998 we established a clinical trials network, the first network founded solely by a nonprofit institution, organization 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

philanthropy model to derisked companies from investing in cf research and drug development. it was our initial investment of $42 million a small biotech company in san diego at all different to kalydeco. vertex would not a veteran 12 and the other drugs announced last week were it not for the cystic fibrosis foundation. the cf foundation spirited collaboration across all sectors and the same collaborative spirit extends to the foundations strong partnership with food and drug administration. the fda come with the fda we are committed to collaboration in bringing strong data to the table. has often been stated, the cf foundation comes with data, not demand. just last week we met with fda officials to discuss strategies for clinical research design that may not occur until five years from now. however, curing the disease is never easy, and even more risky is the approval of drugs without sufficient data to assure efficacy and safety.

if this happens can you please patients immediately at risk and to risk losing the opportunity to test drugs that could have a real impact beneficial effect. so in closing what can congress do for us? congress should make sure patients have a seat at the table, as was just refer to the august must provide the necessary resources so the fda can attract the best and the brightest, and congress must provide the nih and fda sufficient resources for regulatory sites as also mentioned. finally, congress, look at the seat -- the network of care centers that are funded by the nih and see how they might use these to be able to facilitate clinical trial networks and the development of patient registries and other rare diseases. so once again, thank you for this opportunity to add our perspective to this discussion. >> chair thanks the gentleman. mr. popps, you're recognized for your opening statement.

[inaudible] >> i just want to thank chairman upton and congresswoman gets -- [inaudible] >> is your mic on? >> now it is. thank you. i'd also like to express my respect for and appreciation for the focus on the panel and for dr. woodcock. we'll all partners in this together and it's an incredibly important mission. the simple and powerful concept of incorporating insights and patients is important to the future of the nation's health care system. it's also one of the great opportunities us to have a transformative impact. i have said at this is ceo of alkermes for over 20 years. our company develops medicines for people living with chronic, debilitating diseases such as opiate, and depression but our approach is dependent upon considering the patient perspective early and consistently throughout the

process. i serve on the boards of both bio and pharma, and deeply involved in the five negotiations as well as the preparations ongoing for the douches six where elevating patients voice is a key theme for the initiative. today i'd like to propose a new framework for patient involvement in developing medicine which requires engagement from all three of the major parties involved, innovative companies like ours, fda, and the patience to stand to benefit from these medicines. the framework is based on three core principles. first, interaction must be data-driven. based on science, other than come and separate from powerful and passionate advocacy missions that patient groups otherwise deliver. second and engagement framework should be actionable not theoretical. it should improve the overall efficiency of the process rather than adding new steps in a process that is already incredibly complicated. this is particularly more for young biotechnology companies

were developing the first drug unlimited resources. third the approach should preserve and enhance fda gold standard of safety and efficacy which is really one of the more great national treasures. i believe deeply personally that increase patient input can coexist with efficiency and highest level of rigor. so from industries perspective there's currently no consistent way to incorporate patient generated input. this input would have a meaningful impact on a 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. woodcock mentioned at the fda, engagement is not a new concert. provisions included in pdufa as well as fdasia, fda has been open to and taken initial steps to include patient input into the reviews and we can build on

this. this proposed framework i'm considering would build on all of these things. the historical paradigm of drug regulation in the bilateral process between fda and industry is outdated. science and society have continued to advance their patients are organizing new ways and the critical role in driving innovation is becoming more the rule than the exception. if 20th century regulatory framework for 21st century drug development. to include the increasingly -- to tackle these increase the complex scientific or regulatory issues as we look to treat and cure complex diseases, all three parties can work together. these would include new clinical trial designs, more efficient clinical trial involvement, advancing fda's evaluation of risk and benefits, and more sophisticated post-market data collection. these are exciting areas for future consideration. we will need to evolve the way

we work together, all the different parts, recognizing our shared responsibility and our accountability to assure that medicines are safe. there will be a number of challenges and these could include establishing a, threshold for date and the scientific rigor, shared by patient and industry groups come on to find existing regulations of framework. protecting intellectual property and data, which is essential to enabling innovation. and maintaining the gold standard of safety and efficacy. the next that i propose for congress, it is, fda, patient groups come together to develop and implement this new framework. building on existing patient focused decisions at pdufa intrinsics. -- and fdasia. in conclusion the concept of a new and comprehensive patient inclusive framework is both ambitious and at the same time it's quite modest. is ambitious as it could result

in a dramatic change in the way we discover and develop medicine. it's modest because it's not a new regular pathway or authority and it builds on existing foundations. we at trent 11 and all of our colleagues are standing by to help in that effort. thank you very much for your leadership. >> the chair thanks the children. dr. lichtenfeld, you are recognized for your opening statement. >> good morning, chairman pitts, recommender below and members of the subcommittee. i'm dr. leonard lichtenfeld, deputy chief medical officer for the american cancer society. the american cancer society is pleased to contribute to the dialogue around the committee 21st century cures initiative. today i'd like to focus on three critical areas for the committee's consideration. one is the need for greater investment in research. secondly, expedited approval processes that can do to ensure safety and efficacy of approved

drugs, and third, making patients active partners in all aspects of research, development and regulation of new therapies. we are fortunate and blessed today with 14 million cancer survivors in the united states. it's a remarkable number, due to more screening tools have been made possible through research. we must continue and expand our steadfast commitment to research and we must continue to support researchers working on finding the next generation of tears. just as importantly must ensure that expedited approval process for drugs and devices are properly safe, effective, and accessible to patients. the goal of the 21st century cures initiative is to accelerate the development, approval of new medical treatments. there are few other areas that can match the research and develop an activity in the field of cancer. it is, in fact, and has been a model of innovation.

the fda's office of hematology and oncology products has aggressively used the tool provided by congress to speed new drugs to patients, has encouraged drug company's to be innovative in clinical trials. in the past eight months cancer drugs been approved using the accelerate pathway. won approval is based on the trial of 111 patients. an example of research and approvals happening faster and smaller clinical trials as in the case in the best. small sized trials accelerate approvals do have drawbacks. they may not include a diverse population which may yield an incomplete picture of how the drug might work in a broader population. small trials accelerated approval came to be seen as deadlier cancers where there are no other good therapeutic options. i want to stress that the risk benefits tolerance of a cancer patient they see a poor prognosis may be much different than for those with other of able treatment options. and, therefore, the same

acceptance of reduced dat data n which today' to base fda approvy 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 innovation act requires greater patient involvement throughout the drug and device approval process. acs can champion provisions to expand the fda's patient representative program to maximize patient input during the drug development process. we need to continue to build on the progress. patients can provide important perspective at first stages of medical 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 reinforced and

supported, and to this end the fdasia provisions that attended patient involvement must be fully implemented. we urge the committee to consider examining opportunities for providing greater funding, just for the fda patient representative program as well as broader continued engagement with the patient community. another importantly patient's perspectives can't inform the government of therapies, is the design and use of patient reported outcomes. measures of cancer therapy affected sometimes the functional status, pain, or quality of life measures but these may be reported by the physician rather than by the patient. quality of life measures like pain or knowledge of should come from patients themselves, and patients should help prioritize the importance of these side effects and the overall response to disease and the associated treatments. when quality of life outcomes are rigorously measured and supported by the fda, they should be included in a drug

labeling. they should be considered for a drug's approval. the fda should also be encouraged to work with industry and researchers to incorporate self-reported symptoms measurements as a regular part of clinical trials. in closing, we appreciate the opportunity to contribute the dialogue around the committee's 21st century cures initiative, and look forward to working with the committee and its staff. i'm happy to take any questions. >> the chair thanks the gentleman. dr. summar, you are recognized for five minutes for your opening statement. >> good morning, chairman pitts, ranking member hits to my name is marshall summar. i the good fortune to be chief of genetics and metabolism of the children's medical center in washington, d.c. washington, d.c.. been working the fields of red diseases the last 29 years and than it did in my capacity as a member of the board of directors of the national organization for rare disease and chair of the scientific advisory committee. on behalf of the made 39

individuals with rare diseases, a nine thank you for your continued strong support of the rare disease committee to give me a huge difference for us. nord's unique federation over 200 patient advocacy groups, clinicians from research competitive helping people with rare diseases. nord provides resource and research advocacy, education and infrastructure support to the rarities committee a small individual organizations cannot. nor does sound and making a trip lead active role the passage of the orphan drugs act which possesses us a model of identifies the government of treatment that saves lives. data show years of life lost to red diseases decline declined in array of 3.5% after the orphan drug acted to development of new treatment. without these new drugs and take them out of the equation the number of years lives lost should increase at about 1% rate per year. so it's made an impact on our patients. speaking personally without the

statement, many of my patients would not be here. i thank you for what you've already done. these efforts represent -- there's much we can do to improve the lives of our patients in order to use the 21st cures initiative as a great way to do that. nord's long involvement of patients in the drug develop an process, we appreciate that commitment by many of the fda to increase patient involvement but believe much more needs to be done make patients feel their partners in the process. nord will continue to work with fda to advance patient role in the development and approval process. we develop a series of recommendations we believe will advance not only the developing of new orphan drugs and devices but non-orphan ones as well. we look forward to discussing these ideas with the committee. to offer recommendations, first we support the establishment of a commission and national plan to determine priorities, methods, resource needs and consistent agenda on very disease registries and natural history studies.

we have a lot of variation. they tend to be all over than that. assess the drugs ever to win information on the existence, frequency and severity of clinical findings. this information is needed or trial can begin to encourage the creation of minutes of programs to great, curate and standardize registries natural history study which can generate the needed data. this to be one of the most important accelerators of the treatment development in the marketing process. registries can be used imposed approval process as well. this isn't a where patients can have a major and causing impact on the process. patients enter data has shown to be accurate and useful when collected properly. created hybrids using physician-patient and other health professional collected data can greatly speed the understand, discovery, approval and monitoring process. in collaboration with the and h. and fda nord is built and is in the process of testing rare disease patient driven registries natural history program. the nih registered clinical disease network has been

stripped the benefits of this approach. in the register i've been involved with we have had approval of three dogs over 10 years with only 700 patients. it is accelerate the approval process for us. the patients and outcomes research institute is developing this is just the methods and models to use data from rare disease patient studies that will further refined this process to their involving patient driven cornet ambition working with nor did -- nord. all of these efforts will help patients but a national plan would help prevent duplicated effort and resources. this is what we truly need. the other thing we abdicate significant reform the institutional review board system. i've been working with a system for the last 30 years so i pretty smart with all of its manifestations. currently all clinical trust for new treatment with a drug come by logic or medical device must receive approval from an irb.

each study site to put requires approval and vertical adjustment by its own irb. with a margin of -- is one of the greatest impediments and cost to clinical trials. nord recommends congress develop legislation and foster aggression of an ip system that portable across all institution. the encouragement requirement other alliance agreement between institutions receiving federal funding would save cost and time while accelerating the clinical trials and clinical research process greater. this will increase the pool study site and allow greater patient participation. these are just two of our recommendations. my written test that includes the rest and am half of nord i think the committee for allowing us to testify today. >> the chair thanks the gentleman. thanks all the witnesses for your opening statements. we will now begin questioning. i recognize myself five minutes for the purpose. ms. furlong, start with you. do you believe your guidance, collaboration with industry is a

scalable model that can be used in other conditions? specifically, were their unique factors that make addition muscular dystrophy guidance a special case -- make douche in -- that you led with encouragement? >> i sort of think that this methodology and process is affordable to other recommendations. how we started the guides are initiate the guidance with development steering committee that was represented both stakeholders and academia as well as industry. from their distinctly identified several areas, seven working groups of things that they felt were relevant to include diagnosis, biomarkers can clinical trials designed to match a hash and benefit risk. and then we further develop a cap which is committee advisory board, so that would be incorporating the voice any

individual or patient group that wanted to contribute to the development of the guidance. the standardization for the guidance was it would be a reference document and it would include documented evidence that was published or accepted for publication by the end of july. we felt and we're writing the methodology that this methodology is -- a thorough, thoughtful reason look at the community and the nuances of to shin, but i believe most were diseases could do the same. they are issues may be slightly different and the progress to date my beside different but it certainly is affordable. >> thank you. dr. bill, can engage with patients to make sure they can make informed decisions about clinical trial participation is critical. now there's -- how does the cystic fibrosis community communicate with patients the option to naddi think we can best translate your good practices into the cures initiative? >> thank you very much. first of all because 90% of all

of our cf patients are seen in a network of care centers and we also have a clinical come network, there's a very close relationship between our physicians and patients that are involved. that's critical for the recruitment of patients in the clinical trials. it's critically important for showing them the value and the risk of participating in clinical trials. and that close association between the physician and patient and the recruitment process in a very close network that is critical, that's why the clinical trod networks are critically important. so we also have established within the clinical trials network and monitoring board made up of them independent of cystic fibrosis foundation but it's made up of experts. that provides a degree of assurance that every single patient that there's somebody looking out for their continued interest and for any risk that may be inherent in any single

trial. so i think all of these things, plus we have worked very hard to try to create a culture of participation in a responsibility at each patient when you have a small patient populations needs to purchase the in the process. so i think that reassurance is so important. >> thank you. mr. popps, what stage of drug development could most use the assistance of patient insight about benefit expectations and risk tolerance? >> thank you for the question to its the most exciting part of the whole opportunity that's at every stage. from identification of new drug candidate, all the way through to determination of the value of the medicine at the completion to the pivotal clinical trial and i think that's all i get of this framework is creating a structure would begin get input on a continuous basis. i think it could transform the way we approach these development programs.

>> thank you. dr. lichtenfeld, you've discussed examples of cancer drugs that have recently been successfully uprooted by fda through an accelerated approval process. on their best practices that we can learn from cancer and out fda's expediting the approval process for particular drug? >> thank you, mr. chairman. when we talk about best practices i think the question came up was with dr. woodcock earlier today. what is the oncology community doing that is different from others? let's understand it's a complex process in the sense we have research that has been building nearly for decades that has produced very exciting result, actionable and companies are standing up to create drugs for the targets were funding for the new and new therapies for genetic disease, what have you, genetic markers. so we are in a sense interesting and turning point kind of place.

but important relevant to your question, the office of hematology oncology projects has also stepped up to the plate. as was mentioned earlier, the oncology committee appreciates the effort of the fda staff to reach out to the patient team unity, to reach out to the pharmaceutical kennedy, to reach out to those who do clinical trials, to be active participants get the table, london map was cited sometimes, the american cancer society's grateful to go to contribute to that effort among many other organizations. but the fda has become an active partner with the process. so i don't know if that's a best practice or best example. that's for such an occasion but let's not forget it's also the opportunity because we are now in a place that we only dreamed of just a short while ago. >> the chair thanks the gentleman. my times expire. chair recognizes the ranking member for five minutes.

>> thank you, mr. chairman. my questions are of mr. beall. the cystic fibrosis foundation has done some great collaborative work that is resulted not only in successful marketing of kalydeco but also the recent positive test result of the company to drug that makes treatment of nearly half of all patients with cf and, of course, i commend you for your efforts. i wanted to ask about a couple of points in your test with the you spoke about the cf foundation strong relationship with the fda and the board to bring good data to the table when consulting with the fda, which i know is true, and i like her more about that relationship. we are hearing a lot today about the need for fda to do more to seek and incorporate patient input into its review process. the basic question, can you tell us more about the cf foundation's interaction with the fda? are there any lessons that can be learned by other disease groups?

>> i can give you a perfect sample because of wednesday this week with three officials, including doctor temple who's in the drug division at our office is talking about the development of clinical trial protocols of drugs that might not enter into clinical trials three to five years in the. so that's a perfect example of this open discussion. because we have a natural history of the disease, we know that the drugs were tested that are treating the basic defects, we know the action. have a safety profile, and now we can start to talk about the future. i think it's that kind of example and that goes back many, many years, soon after we discovered the cf gene we started talking about gene therapy i would extensive dialogue with the fda. not only with manufacturers of the fda and the foundations. so i could just say we have always had a wonderful collaboration. we have data, natural history of the disease because of patient

registry, and again, we come with the date and we come with experience, and we come with the networks that can make these things happen. so i just give you an example. that was an example -- >> we are hearing a lot about the various expedited drug review process is at fda, and the clerk ambushed by me to get the agency to use these pathways more quickly. and i share the goal. i think we need to be cognizant of the risks that can accompany that speed and we need to be concerned about those risk if we are ultimately thinking about somehow requiring more frequent use of these expedited pathways through legislation but i know you share that concern. it has been mentioned the health risk that could result from a proven therapies based on early data that needs more rigorous studies. you describe the possibly these kinds of recruits could be major progress towards the development of other treatments. can you just elaborate on both of those concerns, if you would?

>> well certainly come when other things when you're dealing with a small population, and then we're talking a personalized medicine we may only have 25 patients with a certain genotype that may be approachable, therapeutic opportunities for that particular 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? so i think that's really very critical because we want to make sure that our first introduction is drugs that work and we before to the next level but because then we are really are depriving if you don't have safe drugs developing good drugs and effective drugs that could move us about the therapeutic options that we have. so i think that that's the critical thing that we always face, there's always a risk but now we're dealing with small population, personalized medicine. maybe there will only be a thousanthousand patients. i think at the particularly critical on that issue with rare

diseases. >> okay. 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 resources to ensure that they can rely on the best regulatory science available and they need adequate resources to enable them to basically, you know, to meaningfully engage with the patient community. we have this 21st century cures initiative, which is progressing now. we've had some sort of larger meetings announcing hearings. and my colleagues always ask what can congress do. i think that the most effective thing we can do is provide adequate resources to make sure that fda as well as nih has the resources to fill the expectations we have are both agencies. and i know, i hear not only from the agencies but also from my

constituents that, you know, they don't have enough resources. so i think, i swear to echo again what you said. thank you, mr. chairman. >> the chair thanks the gentleman. now recognized the vice chairman of the subcommittee, dr. burgess, five minutes for questions. >> before my time start start sn and maintenance consent request? >> proceed. >> i'd like to move that the committee make people aware that contact or committee with the cures initiative, it's cures at mail at house.gov. many people watching who think i like it or out with committee staff so that's the way to do it. >> thank you. without objection, so ordered. >> very well. i knew they wouldn't deny me. let me ask ms. furlong, your kind enough to mention the work on the md care act, and thank you for that. as you know we will likely be marching that up next week so that's a big milestone. can you talk about how the md

care act needs updating and type of updating this committee has pursued? >> certainly. and thank you for the question, dr. burgess. the md care act was a solid foundation that set duchenne and the muscular dystrophy, really galvanized their progress. of the md care act was passed, enacted in 2001 and reauthorized in 2008. right now the amendments are really to look at what we've learned in the meantime. so the cardiac issues in duchenne and mustard you in duchenne and mustard you should operate and have to be tackled in order to answer the question as you look at these therapies, potential therapies that are helpful to be approved in the next months and years. extend function. will they protect or have a negative effect on the heart. so it's the gaps we need to look at with the amendments. in addition to the fact that when this legislation was in active and 2001, young voices of

duchenne didn't live to be a does. would now have an adult publishing and we need to address those adults in terms of their medical care and also to incentivize and understand how, how to treat them, have encroachment of long, independent lives as they become adults and reach for their dreams. i think the md care act is now looking with the macho dystrophy committee from the nih and other agencies, research plans to be updated and these amendments to incorporate so that we are really achieving the full effect that the intake air act was initiated for. >> thank you. i think will acknowledge the population of patients is changing because some of these successes that has happened over the past several years 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 changing. we want people to live longer and fuller lives.

and at the same time we don't want the legislation to stymie that. so it's my opinion an important step forward. doctor beal ca, we talked with u talked about development of mutation specific therapies the next evolution in precision medicine. and you can see the cystic fibrosis example. is there something more good like to add to that? >> again, we are clearly in the age of personalized medicine. fortunately, with the human genome and we understand the genetics of so many diseases and genetic diseases that it is a very critical time for us. i just saw doctor -- am i on? dr. collins downstairs and he's excited, he was one of the discoverers of our cfg. we live in a unique age, and i think more and more therapies are going to be directed to specific communications. that's one of the reasons that

we have to have these kinds of patient registries so we can start to identify those communications. windber text felt they had a drug that might work on a certain mutation, small but became a, we were able to tell them in the united states we have 1100 of those patients. within five minutes after the ask this. because of a patient registry because we have it documented history of the disease. i think that's why it's very important to personalized medicine therapies and the options for that but it's also, we have to be up to document the patient's back and participate in the trial. >> it is there a powerful and, of course, you reference the 1965 registry. in 19 cities but you did know that we're going to know about the sequence of the human genome 30 years later. >> well, but we been able to document. today we currently cover 26,000 patients whose dated is provided to our patient registry every single year.

>> let me ask you a question, i'm going to run out of town quickly, this is it for mr. deal or mr. pops. the world is different now. you have people that are perhaps like enough to end into a clinical trial and they are likely to perhaps have friends with same condition. so in the old days randomized clinical trial, you would know which arm, who was getting the target, the study drug, who was getting either an older therapy or no therapy. now people communicate, facebook, twitter. they are likely to be facebook friends. how is that going to impact the ability to have a blind, randomized clinical trial? i'm getting a lot better on this stuff, how about you? spent i think it's a real question, a real issue. you can't pretend it's not going to happen to its already happening, particularly if you get large cohorts of patients and randomized studies and

multiple countries. they are all communicating. i think it's important we be regress in maintaining to the extent we can. >> you probably need to embrace the fact that information is out there and being communicate and to the extent they can further enhance what we're doing. >> let's take advantage of the. let's do more in the aftermarket. let's been approved drugs and get a more nuanced view in the real world and turn it to our advantage. >> and in some cases to make it easier to do clinical trials, when you can have large networks that exist out there when they to report patient reported outcomes and things like that. so i think it sometimes look at it as a disadvantdisadvant age but we are determined it as mr. pops just said, we are to turn into an advantage because i think you can expedite the ability to do clinical trials as we move forward with the technology spent and should expedite analysis of it and see the long-term effects because clinical trial of 12 months, plus or minus, you might not see the full effect of the drug that

is multi-stemmed. so it will enable us to understand the full impact on the patient's. >> thank you. thank you, mr. chairman. yield back. >> ms. schakowsky, five minutes. >> thank you very much. i had a question for dr. lichtenfeld. actually, for anyone. on the panel that wants to comment on this. this is about quality of life outcomes. obviously, this is known life-threatening disease. you want to do everything you can to make sure that the therapies match the disease. but when the squalid of life outcomes are rigorously met, measured and supported by the fda they can and should be included in drug labeling and can't by themselves be a basis for a drug approval.

i survey know people who have suffered so much -- i certainly know people have suffered so much in side effects from drugs, and i just one in the whole process of drug approvals, how much are these code of life issues really looked at as a basis for approval, or just as a basis of whether or not they are used? >> thank you for your question. in fact, it's a work in progress. let's understand that quality of life is a buzzword today but it wasn't a buzzword very recently. so as a look at the issues shall we say palliative care, quality of life issues with the american cancer society many others have been involved, it's a relatively new to the table. having said that, there have been issues recently with a drug, one particular drug where the question really centered around was, you can even though the drug may not admit the fda

stated, this was about two years ago, even though it's not make it fda stated, did you meet quality of life standard? did improve the quality of life -- breast cancer drug, for the women who took it? that would've been an important consideration. and, unfortunately, the quality of the data, measuring quality of life was inadequate. so going forward, i mean, cancer patients have enough on their plates come as do everyone representing at this table as to patients throughout this country. we need to be aware of quality of life is an important part of the treatment process, and we need to have tools in place that are not uniform yet, 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 very much a part of that process. >> go ahead. >> i did want to make a comment as it relates to patients with chronic diseases.

we talked a lot about cancer and orphan, small disease. we work in the field of chronic disease, schizophrenia, depression, addiction. patients taking medicine for long periods of time, and simple things that may seem prosaic to a researcher, nazi, propensity to get addicted or dependent on a drug is a really important, we might want your from patients about. >> is that part of the process speak with its less part of the approval process today that it will be in future. it's part of the utilization process as patients make a determination what medicine don't want to stay on for years and months. i think that's a critica critict of it but it's not incorporate in the consideration of the approval spent a special were all thinks might be equal and effectiveness, whether or not something causes nausea, fatigue. could be really important -- >> particularly launching a new medicine into a large category weather might be an abundance of generic drugs that are safe and

effective but might not hit all those parameters for certain along predetermined. just want people to be sensitive of the fact from the patient's perspective there are differences. >> dr. lichtenfeld, i want to ask you about small size trials. and you mentioned one of the drawbacks. i have talked about the extent to which women are not considered, and i would just be concerned. i understand the plus, i do, but if we will rely too heavily on them, isn't it the real risk of excluding important parts of the publish? >> yes. in fact, when you talked about women and heart disease i remember back in the early 1990s when the article came out talking about the absence of women in clinical trials for treatment of hypertension and heart disease. it's an issue i'm aware of.

but let's talk about the other side of the coin, and that is when you're sitting -- i have sat in the presentations at the oncology meetings, and you see a presentation of 80 patients, and to see what we call waterfall plots, basically the responses in survival that occur, and suddenly 70% of the patients are having significant responses. i don't think, in the disease where there was no treatment before, i don't think one ask -- asks the question in follow-up but not at the moment, and what has happened and what's exciting to me is i'm now sitting in those presentations every june, and i actually wrote about it, took the year for one of the drugs to go from clinical trial to approval. because it was that effective and the disease, there was no other treatment available. that's pretty spectacular. that's new thinking, a new approach.

we've learned more as time is going on, yes. it doesn't mean we have stopped learning. as we mentioned for with cystic fibrosis that when you suddenly see moments like that, no one would want to hold back, develop the data, yes, but don't hold back the opportunity in fact even a phase one trial presented at this meeting in june, the company, not the company but the press release indicates the government was willing after phase one talk to put it into compassionate use. that's pretty, again, pretty amazing how a major change in the way that traditionally we have seen cancer drugs move through. >> thank you. i've overstayed my time. thank you. >> recognized the children from virginia for five minutes. >> dr. lichtenfeld, just pick up there because i think that compassion is a something we really need to be figured out how we can make it more effective and that we can do it faster at the federal level. you talked about in your testimony that patients need to

be involved, what kind of nausea debt and with the pain levels were and so forth. and i agree with that. i also think when you have no treatment, patients need to be involved in that. as ms. furlong said earlier, when it isn't a treatment, you are much were willing to take those risks than you would be if there some other treatment out there that might work but this might be a little more comfortable. i wanted to both you and yours for long enough to to address that for the -- ms. furlong. spent i appreciate the quick ad i know you mentioned it earlier. it is a complex issue. it is not a new issue, as you may well be overcome it's been around for some time with the number of drugs that have gone through the pipeline which seem to show some opportunities. there are a substantial, and also noticed in the legislators are involved. i'm sitting here today both as a representative of society but

also as an individual, and understand that the our discussion on both sides of that issue and there are complicated. bottom line is that we need to understand what drugs work when they work. we need to understand that patients did have access to promising drugs as soon as possible. companies make those decisions as to how they're going to handle that process. the fda has approved almost all the applications they received, and we need to have those discussions to come to a better resolution about how to address that issue. >> what i would say is that whatever we can do, i think i speak for a lot of the members of the committee, i'm probably a little more out there than some, but whatever we can do to help by changing the law to expedite that process we will do. >> we would be glad of those discussions. >> ms. furlong, did you want to make a comment about risk assessment? it is obvious and when your boys were sick, it would've taken anything