give me dvd that -- them my name. that is what is happening. i had multiple phone calls from those across the country. it's because they were forbidden to document the force. there are several in that there are many issues. i contacted jeff miller or representative miller's office and got a phone number of someone that said that they would maintain the confidentiality and investigate. and at this point i would tell them that is a problem above my pay grade. >> i have had several company derrick pulley and i share the testimony with the committee and i read some of the statements in the record and i would say though that i know that our failure with the administrative process either the house i would

be more than happy to take the whistleblower complaints to the public. might have grabbed his communications i was able to navigate quicker than most and that is the key thing. it doesn't take everyone to do the same thing. some people might be comfortable just going through the ig and the sun it covetable going to the representative or senator and some light be comfortable going to the press. but there are different levels. you don't have to go as far as we did. in some cases there is an exception that there are different ways that you can get the information out and different people that want to. there's the civil groups, veterans service organizations that would be more than happy to get the information they have direct connections with other leaders in congress. there are different ways that i would tell you this. you feel better when you say something versus holding it in. >> i have no doubt there are

employees listening tonight and seeing the comments and i might add that they are probably out there during all of you down for having the courage to show up. but step forward and model this other office to carry that water for the brave employee like thet was also the appreciate the commitment. >> if i could make a clarification even though i said i wouldn't recommend getting a job at the va i am not looking for a job elsewhere. they have the culture where it stands today. >> there is no doubt in my mind that you are a veteran committed. >> thank you. you are recognized for five minutes. >> thank you mr. chairman. to follow up on that statement, i couldn't help but wondering during your testimony and in the answer to the questions that were asked of you as you

detailed the ostracism that you were being shunted aside when you made problems for the management ending up in the position now that you are doing good but it isn't a position that you signed up for and then i know that you clarified it a earlier saying that you would recommend to somebody thinking about working for the va not now, not until we get accountability and oversight protection for the whistleblowers i couldn't help but wonder why you stayed. >> the work is fulfilling and important. i went back to medical school to be a va physician because i felt there was a great need. everyone knows there are limitations. we are a federal department. the veterans are so grateful for the quality of care. you will see such a wide variety of people at the va and from the physician standpoint to

interesting. in the geriatrics i have a playing field in fact it was urgent medicine at its best it's interesting and fulfilling. i don't always feel so resilient as a physician. i am tenacious i get myself gif that but sometimes there is that's not in the center of your stomach when you just don't want to show up because as much as you love the veterans committee administration really wears you down and you begin to doubt your own professional ability. >> and they answer what you said earlier and for everyone on the panel, we keep asking about the culture that is the most important issue but probably the most difficult task before us as a country turning around but you represent a culture that i think we are looking for and that we want to see throughout the system not just that the provider level at the management and the secretary level on

through this committee and as a country so i want to thank you for that and for the example that you provided. but i also want to follow up on another commente comment that y. you mentioned surviving 16 years of this and these problems didn't just occur under this administration i've been here for a year and a half it is a joint hearing in the committee where we heard from the veterans service organizations and i remember the commander coming before us saying i don't know the number was with a 32nd time that i've appeared and have been coming up for decades and i've been saying the same things over and over again. so, you said that this is a system worth saving but my question to you is is it's salvageable? >> you've got thousands of

employees that are dedicated to the veterans and the welfare of the veterans. i'm discouraged when i hear people say the va is too big to change. you have an entire group of people that are ready for the revolution. and they want this. they want a productive healthcare system delivering good care. the horizontal violence has to stop. that was one of the implications of the whistleblower retaliation is that it affects care because you don't speak up to save the problems are because you are afraid of the repercussions. the corollary to that is that you begin to -- it is a pressure cooker and you pick up to each other gossip, bullying, exclusive we kind of see it on each other because we don't know they are under so much pressure and that needs to stop, to. >> you have given us ideas on that but i do wonder how we are going to be able to do that after so many years and so many

fundamental systemic problems doctor matthews brings up the issue of not being able to trust the integrity of the data that has become obvious to all of us and i commend your efforts to measure those things that are important to the patient care and outcomes in the facility that you worked. we've been trying to do that and in el paso we have seen similar rates of the veterans seeking mental health who cannot get an appointment, just give up and stop trying and we can only write now because we don't have the full story wonder at the outcomes. we introduced the ask veterans act which would integrate wyoming's ea to tell us how they are doing that to ask them what you were trying to measure indoor facility. let me just conclude by thanking you all for what you are doing and we hope the recommendations that you gave us might lead to some of the cultural changes that we all know are essential to turning the va around.

>> thank you. mr. kaufman you're recognized for five minutes. >> thank you all for stepping forward as whistleblowers. i believe the rank-and-file in the veterans administration are in fact employees that care about serving the needs of the nation's veterans and without whistleblowers such as yourself who have had a courage to step forward, we would never know the problems that exist in the veterans administration because none of those problems have ever been self identified by their leadership in the administration. they have always been aware of them simply by the whistleblowers coming forward and sharing with us the reality of what is occurring on the ground with the veterans health administration. one thing i think when we became

aware of the magnitude and the crisis was concerning the patient wait times, and the fraudulent changes in terms of those records often fueled by the drive for bonuses. that's what you're saying is actually the problem was much deeper than simply patient wait times, that they were also denying people inside the system is that correct? >> they didn't get into collecting the applications that i think this is where we have to look at you can only get the appointment if you are enrolled and so we have systemic problems in the enrollment system and to give you some context you may hear this from the next panel the office that helps build of this center is about to start with a call for command center. this is something that they will

assume to be the leadership or perhaps the committee that i want you to understand the change will only come from real solutions at the va. currently this is what i call the gimmicks that go on at the va. we send about making the public and the leadership on the hill. but i will tell you when you look at this document to the communication train people perform the communication training every day. that isn't anything new. the call-center people perform the call center task. this is not going to change anything. the strategy is to take people on the fifth floor and put them in the second floor. this is what constitutes responding to the veteran concerned that the va. following something that works in the business that is the people sign off on the reports they turn into the congress. i can tell you what disappointing to me as a citizen at a va employee is to watch

leader after leader sitting in the chairs and say i don't know. i will get back to you. so and so was supposed to do that. the general council won't let me. that is inefficient and if you are going to be in a leadership position you need to leave for making people sign off on a quarterly report to say that i own the data that i turn and, i owned the enrollment, you doubt very many people in this room there was a 600,000 pending backlog in the va from the iraq and afghanistan veterans of people would have known that was going to happen if the reports have to be signed off on by people in the positions that were held formerly by the doctor this is where the change comes from another you have to document. one of the problems that we have is the first time that you go to make something public they tell you where is your proof, where is the document? most people will not sign the document. i forget to process the applications but this is the

kind of conversation where you ggo in to talk -- >> let me put it this way. if you all could comment on th this. the veterans administration is so dysfunctional )-right-paren in terms of its leadership and in terms of the culture as well. so having the new secretary come in they are still there. i hope the secretary ca can take the appropriate change but it's going to be difficult. should there be an entity outside of the veterans administration for what they report and let me go down to the rest of the physicians here. >> it is inherited if you really want the new change in the whistleblower environment where people come forward you have to take the policing power outside of the va. >> i agree no one trusts them to handle their own problems that we have reported it to them. >> i agree. if we acknowledge the problem

these are expected to fix it. the position is that there is no problem. we have the numbers to prove it. >> thank you for being here. i realize that we need to go outside the va that is under the point that we have to make the change because of nine of the positions including the secretary are vacant right now so if we can bring in a new leadership team and impress upon them the need for the accountability that we have heard repeated every hearing whether it is on the backlog or bonuses or the whistleblowers this is the message that maybe we are at a point where we can start to make a difference. i'm sorry that mr. mcdonald can't come in here and hear what we are hearing.

i know sloan gibson is scheduled to come that we need to get the new secretary and as soon as we can because he needs to hear the kind of thing so that we can move this in a new direction. we have a new hospital. i met with some of the emergency room doctors at my invitation they were scared to come. they are not as brave as you all are and they want to be sure that they knew that i invited them because they feared retaliation. you travel in small circles. have you had any contact with people at the hospital were for me or any whistleblower proble problems. we have had a significant number of the staff to relocate but i haven't had a whistle blower problem. >> i'm glad to hear that.

one of the things i wanted to ask you, you mentioned that the first sponsor to the whistleblower is to try to impugn their integrity. one of the reasons you mentioned is that you often say they are just a disgruntled employee because you didn't get the bonus that you wanted. can you talk about the nexus between the bonuses and whistleblowing. are people getting paid to be quiet? >> i don't know that people are getting paid to be quiet, but i do think that there is a tendency to try to generate the motive for why someone is coming forward to when the truth and reporting wrongdoing. and it's often associated with somehow personal gains for the whistleblowers but i tell you there is no personal gains of being a whistleblower even as you go through the litigation you ultimately when, you know,

there is no financial incentive whatsoever. >> i'm sure of that. i'm thinking of just the opposite you keep people pat down and not speaking up if you give them regular bonuses and it keeps that culture of silence that you mentioned. >> the bonuses are among the chief of staff and higher-ups. >> doctor mathews or doctor mitchell? >> my experience in the st. louis va have productivity data for every psychiatrist as to the number of patients being seen. and i know that there is only one who perhaps didn't get the full performance based for what could consider to be the bonus and that's me because actually for their own reasons they were correct. you could accomplish what i set

out to do but just do what you want to do it you will not get into trouble for not working. the only reason i think one can get in trouble is by identifying problems and then coming forward. so that has to change. and i think it is a complex issue with the college culture, that the fix can be very simple and demanding the data and holding people accountable. you know, that once that starts to happen and some senior positions, not people that redesigned again, they have high integrity that they re-sign because they have integrity, but the people who don't care, those people need to b be fired be fit it sends a message that this is not -- this cannot be tolerated anymore. so i would say that your right to the people that get the

bonuses are the ones that keep quiet and keep doing what they are doing. >> doctor mitchell? >> the difference between the performance bonus, the performance measure though this is what you get if they've got the performance. most o of us that are eligible e quite frustrated because the facility never ha have the resources to make the performance measures. so there is a bonus just nowhere near where we want to be reworded for the work we do in the proficiency that they are actually how he would perform through the year on our own personal merit and those are subjected. if the administrators like us they can rate as high or low and they don't have to give a reason why. most say quiet not because there is any benefit one way or the other at least at my level. >> thank you mr. chairman. >> you're recognized for five minutes.

>> as i sit here and listen to your testimony one of the things that comes to my mind is somewhere along the line through your parents were somewhere, someone taught you about doing the right thing. and about being able to look in the mirror at the end of the day and know that you are doing the right thing and i applaud you for that. and know that you are respected by those that matter. and those that don't have their own issues and i appreciate that. i served in iraq as a doctor, and we have something that you mentioned tonight with a sense of mission. we have a shared sense of mission and everyone was on the same page. in the reserve unit we come from private practice. there is no reason for slacking and the patients were the first priority and you work through the night if you have to. and you take shifts sleeping.

wouldn't you love to be able to practice in the environments like that every day? they've provided for the others that are veterans today. and it is really sad for me to hear that there is a need for an agency with a higher integrity than the va. which was said tonight. that's the people would be willing to accept that they need someone to watch over them because of their lack of integrity and we've brought up the mortality and wha when did e and the peer review. i needs to is you had people from the same specialty reviewing the charts and people that know they are familiar with the procedures that you're talking about and you do that to try to make things better and if someone is feeling that they have to go because the reputation is on the line.

it's not fair to be punitive but to make everything better as far as care. so my question as far as whistleblowing is there any chance for the providers and put such as have too much administrative responsibility we don't get to see the patients, such as i need another clinical assistant in here i need a medical assistance then i can see by times more patients or do you have the opportunity to say so and so is a poor performer in the clinic and it's slowing my time down with my patients and i don't get to see as many. is that available? and i will start with you, doctor mitchell. >> in a particular form is not available. in section four and five i talk about the retaliation tactics against providers and one of them is failing the ancillary service so the providers are stretched thin and there's another they overloaded the

patient panels that there is no way that they can link through. we are not at the level to be able to communicate equally with our administration. we are far below. and anyone that speaks out is retaliated against the don't have the freedom to speak freely and advocate for the patients in ourselves. >> ..

>> yes, by the providers. basically the provider side they that they would no longer practice this in this area and they were responsive to that. so now it is a certain amount of perception. and it also took a lot of experience for the providers for those who as a group spoke up and said this is not right. so i do think that this is appropriate. and i think a lot of times resources are placed in areas where there are too many resources and things. so again, it involves a solution

>> very quickly. >> to quickly outcome i was trying to institute a position with what is being provided. so we had those two accurate measures, we can know which facility we are putting end, 15 hours or whatever, and the answer exists with more resources. and this includes the psychiatrist that i was monitoring and so there is a solution there that has more efficiency and it's not just more resources. it. >> thank you very much. i appreciate it. >> sir, you are recognized for five minutes. >> thank you, mr. chairman. i am grateful to all of you as well. it's interesting. mr. davis coming you said

something i can relate to. but i have been here 18 months as well. a typical pattern of how this investigation is working out to get the health care that we promised them when they fall for liberty and freedom. so typically a panel comes in and the reference desk. and it tells us unbelievably shocking stories, back to your comment, that are so shocking and disappointing and disappointing to me representing veterans of our district. and there's nothing celebratory about it. and especially we need to commissar mentions a good future. and representative walden alluded to this as well. you will walk out of here there

have been dozens of be a panelist are going to get us to answers, either that you are not telling the truth we don't have other questions to answer and that's going to happen again tonight and if it doesn't happen tonight, i will be shocked. but there are dozens and dozens of high-ranking members and specifically doctor mitchell, this has kind of been in phoenix, the phoenix facility has been part of this reaction time. one of the things that has floored me is the lack of urgency on the part of the va that there is a five alarm fire and no one is rushing to put it out. i'm thinking that if i was in an among the phoenix va, and if i was responsible for any of this stuff from the minute that this hit the fan nationally i would be trying to get us out doubletime and make sure that my facility is the standard and that we have reset the record

and we are an example for the rest of the country. in the three months of this has been part of it, the american people so they will not tolerate this and this committee says that we will not tolerate this either. we will drill this down to where we can provide the best health care of our veterans. but doctor mitchell, have you sent anything in phoenix that says, wow, what a turnaround? people have been fired, there's a ton of accountability and the people demanding accountability. have you seen that in phoenix in a. >> the turnaround that i have seen has to do with scheduling. because the backlogs have been reduced. i put in a consult and we got a phone call during my appointment and they have certainly done tremendous work in the veterans process. but the problem is they only fix the problem in the media and they haven't fixed the patient care problem although they are certainly working on that in the psychiatry department.

so there are administrators they are the refused addresses that was directly impeding care for those patients in the emergency room and there was a meeting or five or six full-time positions we're part of the chain of command and he said flat out that we will not investigate this against doctor mitchell. >> we had the inspector general here a couple of weeks ago who said that the issue of ruling out corruption is not going to stopcoming it is still actively going on in your corroborating that's actually going on against you. until somebody does a prison and people are fired and that there's actual tangible action being taken and that includes the veterans for health care and in third, your colleagues that you work with as well feel like your backs are covered. how long do you see if it took a national urgency, but it took a

resilience on the part of the chairmen chairman and the ranking member actually go after this issue to try to reset it. even if we keep pressure up, even if a new secretary comes in, if we don't root out the corruption, how long do you see that it will take to turn this around if we keep up the same amount of pressure? >> i am not sure i'm in the best position to judge that. but the media paid attention to the scheduling issues and i think all of a sudden that that is completed within 10 minutes. they need to pay attention to the lack of ethics. >> absolutely. >> i would like to say that i think that we have to do two things. we do need a separate group and one a new secretary comes in, he or she will have to deal with the health care issue first. it will probably not have time to become the chief of police

for the va and make the health care reforms. so you're going to need some assistance with this operating authority only acting for a period of years until you get the va under control. so the next thing we have to look at his performance standard for leadership. unlike those who work at medical facilities, they may have some legitimate researchers for their challenges. and our in our organization, the primary function is to enroll the veterans and health care and that is not just about the politics of the law but the va having a public affairs division here and a national veteran outreach here and it helps us with the communication outfit and that was sent down to atlanta for the sole purpose of a senior executive at a performance goal and had nothing to do with anything else. so what you need this agency to look at us? we have 600,000 and that rivals

the number of people who roll in the va in a given year. so imagine a years worth of applications sitting in a pending status. put this in context of talking about a bank. 600,000 deposits go in on monday and we never hear about them for another year or two. so do you think the walls of the bank would still be standing here today? and yet the men and women who sacrificed his country have to deal with us. and not because we don't have the resources. the focus on the insurance program and marketing materials for delta dental and metlife, yet we need to let people know if you had a pending application in these years, contact these numbers. the same that we put in getting the bonuses would attaching

ourselves to a high-profile projects. so the same tension into the veterans. so if nothing else, make sure that we move to a system that has more data integrity and this includes the people that sit on these panels to sign off on information that they turn into congress. this way when they come back they cannot say that the report was done by someone else. that is the only way, to hold them accountable and do it in public and do it when the cameras are on. >> thank you, mr. chairman. >> sir, you are recognized for five minutes thank you, mr. chairman. doctor, what is the relationship between ucla and the l.a. va hospital? >> like many of our institutions we have an affiliation agreement and a institution and a member of them have joint appointments with both of these within the university and also with the va.

so i ask because this is a very egregious case and i shared my colleagues comments and it was a case ultimately against ucla board of regents. >> that is correct. >> so the settlement was with this and not with the va? >> well, it is a complicated case in with my case we both satisfactorily agreed to part ways. but if you have noticed, there is tremendous overlap in a federal component two. >> so here is my question. trying to distinguish this. the incident he referred to was the june 2006 party of one of these parties.

so was it strictly ucla party? or was it also the va institutional involved in our? >> at a particular party of there were a number of members who were employed as physicians at the va. a component of that was directly related to that investigation occurred at the va. >> the facts of the case led to a settlement with the board of regents in california, do they also support a claim against the va and is it a habit you from filing some kind of legal action against the va? or was it specific ucla and not the va? >> i cannot comment on the state component of this. >> here is my question. because it is a significant

case. the facts solely lead you to litigation against ucla and at what is the prohibits somebody in your position from seeking redress from the va? >> it allows me to seek this from the va and there is a federal component. >> but your settlement is on the stateside? >> yes. >> are you familiar with this program? >> it's something that i have seen and has been highlighted by my local va hospital is a program that every bacs from the cost of meals staff to others, if they see something that interferes with the delivery of patient care at any level, it is an imagery, if you will, that any employee has the ability to

stop operations immediately out of concern for something they might see. it has been adopted in a number of facilities and none of you are aware of this? remapped i certainly am not. [laughter] >> i will be honest with you. it was promoted to me as an effort by the va to encourage every employee to step up and say that there is a problem. but each of you have already supported a whistle blower capacity and yet have no knowledge of the program that says perhaps that is not as promoted as internally as somewhat just say has been. >> i would say during this long process, i find that very distinguished. >> thank you, mr. chairman. i yield back. >> thank you, members.

i like to go head to the next panel, unless someone has a burning question that they would like to ask. thank you very much the witnesses. we do all appreciate the courage it took to come here tonight. we will be watching and rest assured if any of you contact us, we will all jump to protect you from any fu screeria -- nig-

nasa engineer behind the mars rover curiosity talks about space exploration. next the house commerce subcommittee on health lacks at how prescription drugs are approvaled for general use. members heard testimony from pharmaceutical company ceos and the center for drug evaluation and research who discussed way the agency has streamlined the approval process. this two-hour hearing is chaired by congressman joe pitts.

>> subcommittee will come to order. we're going to have early votes so we're going to have to start. we understand minority members are on their way. the chair will recognize himself for an opening statement. today's hearing provides us with an opportunity to examine perhaps one of the most important aspects of the 21st century cures initiative. what does medical innovation or faster cures mean for patients? keeping our work centered on the patient and understanding the patient perspective will bring much-needed focus on results for patients who may lack adequate treatment options. remember there are only effective treatments for 500 of

the 7,000 known diseases impacting patients today. while fda has developed an enhanced structured approach to benefit risk assessment in regulatory decision making for human drug, device and buy logic products, the committee recognizes the value of considering patients in decision making about therapy, development and access. assessment of a drug or device's benefits and risk includes an analysis of the severity of the condition treated and the current treatment options available and getting the patient's unique perspective should be a part of that assessment. one of our witnesses today of the parent project muscular dystrophy, and i might say pat is accompanied by mary bono mac, a former member of this committee. welcome, mary.

and pat will explain how this organization was founded to create opportunities for families waiting for therapies to develop muscular dystrophy from claiming young lives. to quote pat fur long, patient focused drug development acknowledges the need of input from patients and caregivers to create a more complete assessment of the benefit risk equation encouraging predictability and increased flexibility within the review process. the clock is tick iing for patit who is need and deserve access to promising therapies, end quote. i would like to applaud her tireless work drafting guidance ppmd recently released that quantifies patient priorities and preferences. this guidance will serve the community and every other patient community because it provides a path for other patient groups to follow. this was an enormous undertaking

and i'm confident it will make a substantial contribution to the entire medical community. i want to welcome our witnesses today and look forward to learning more about the assessment of benefits and risks central to development, regulations and health care decision making and the tradeoffs between desired benefits and tolerable risk. any member on the majority side seeking recognition? chair recognizes vice chairman dr. burgess. >> thank you for joining us again. it's always good to see you. always good to have you as a witness. you always provide valuable testimony and our second panel representatives also want to acknowledge just as the chairman did many of the patient organizations that you represent that have worked well with our office and myself over the last several years.

mr. chairman, the goals of the 21st century initiatives and they are indeed allotable. we have to remember at the end of the day it's all about patients. doctors want to heal, that's why we entered the profession. no doctor ever wants to tell a patient there is nothing more we can do. the good news is that the golden age of medicine is really right around the corner. the doctors of tomorrow will have tools at their disposal unlike any before in human history, the ability of tomorrow's doctor to alleviate human suffering is going to be unmatched in history. every day that goes by that these tools are not realized is a day that patients and their families have to struggle through the pain and suffering of their condition. every day counts for these americans and their families, for those who struggle with rare diseases, their struggle is only compounded by the lack of research. for those patients it's difficult to see over the

horizon. we have much work to do on this committee and we have done a lot in the past. we particularly celebrate the two-year anniversary of the food and drug reauthorization act that was just a few days ago. that was a good template. it was a good method for moving forward and i appreciate that the cures initiative is following that template, but there's no doubt we can do much more. i welcome the testimony and yield back my time. >> the chair thanks the gentleman. now recognize the ranking member of the full committee r for an opening statement. >> thank you very much, mr. chairm chairman. this hearing is a fitting followup to wednesday's hearing on clinical trials. after all, it is patients who live with the diseases and conditions for which treatments are being sought and this hearing which is called 21st century cures incorporating the patient perspective illustrates that we should take every opportunity to understand their experience. congress has a long history of listening to concerns of

patients. that's what i did in 1983 when i wrote the organ drug act. that law came up when i heard from a constituent adam who had a rare disease called toour ets syndrome. he was forced to take a drug he could only get from canada. there were no effective treatments available in the united states. when his drugs were seized at the border, his mother made a desperate call to my office begging me to do something. i set out to figure why there were no drugs in the u.s. for adam's condition. we discovered that adam was not alone. there were 134 drugs for rare diseases, but only 10 had come to market solely as a result of industry. we knew we had a problem ourn hands and we set out to solve it. the organ drug act has been a resounding success. today there are over 400 drugs for rare szs and i want to welcome the national

organization for rare diseases here today and look forward to their testimony. i'm telling this story about the organ drug act not only as an example of how congress has listened to the concerns of patients and acted on them, i tell it because it's an example of appropriate use of legislation. in the case of rare diseases in the early '80s, there was clear evidence of a market failure in need of congressional action. in the context of the 21st century cures initiative, we need to assure that both fda and the drug and device companies are listening to the concerns of the patients. fda has a long history of engaging with patients both in the context of advisory committees and in its review of drugs and devices in the 2012 fda and safety act congress pushed fda to do even more to hear patients' concerns. and i look forward to hearing more from fda today.

from what i can tell, the agency has taken that mandate seriously and has engaged extensively with the patient community. we should ask today whether fda has adequate resources to continue to do this work. as i mentioned on wednesday when we had our last hearing, when it comes to legislating in complicated scientific areas like the conduct of clinical trials, we need to proceed with great caution. for example, one issue in the area of clinical trials that's likely to come up today is how to incorporate so-called patient reported outcomes. as i understand it, this is an area that is multifaceted and scientifically complex. congress should ensure that fda has the flexibility and authority to make use of these outcomes, but not dictate how and when that occurs. i hope fda will tell us about how it is applying other novel

approaches to clinical trials in their regulation of drugs and devices. i would also like to know whether the agency believes it has the authorities necessary to adopt new approaches and whether other new statutory powers are necessary. mr. chairman, thank you for holding this hearing. i look forward to the witnesses' testimony. i must say in advance, there's another subcommittee scheduled at the very same time as this one, so i will try to be back and forth to participate in both of them. thank you, and i yield back my time. >> now recognize mr. upton for five minutes for an opening statement. >> i yield back my time. i'll just submit my statement. >> chair thanks the gentleman. we have two panels today. on our first panel, janet woodcock, u.s. food and drug administration. thank you for coming again today. and you'll have five minutes to summarize your written

testimony. so at this time, the chair recognizes dr. woodcock for five minutes for opening statement. >> thank you. we are here to discuss how drug development better meets the needs of patients. decades ago health care was very physician centric and very paternalistic. we all recognize that today. it was kind of the doctor knows best, don't ask any questions, right? today the model is collaboration between a patient and a health care team. these changes, though, have evolved slowly in our society and thinking in drug development has slowly changed in parallel. the fda safety and innovation act of two years ago took significant steps in this direction of patient-centric development. it contained agreements that fda would sponsor at least 20 patient-focused, disease-focused

meetings over five years. eight of these meetings have been held to date and have been very impactful. the first was chronic fatigue syndrome. we have issued a draft guidance on drop development in this area, a very serious medical need. under were agreement s s to adve the development and use of patient-reported outcome measures. these are measures that the patient can fill out to say from their point of view how well they are feeling, how well the treatment is working, what adverse events they are experiencing. we are having an expert meeting next week and we are continuing to work on slcollaboration for patient-reported outcomes. this is very important to really scientifically inkorcorporate t patient's perspective into clinical trials. additionally, fda was to advance the development and use of a

structured benefit risk framework in the drug approval decision. in this work is underway and it provides for considering the burden of disease, the impact of current therapies, both for good and for ill, and the tolerance of risk from the patients point of view. this is extremely important set of factors that need to go into the benefit risk decision, but we need to do this in a scientific manner and we are rolling out this structured benefit risk framework. now for people we know for people with very serious diseases who may lack good therapy or lack any therapy, access to new treatments is their number one priority. and that's why expediting drug development programs in these areas is so important. if you look at the diagram we

have here that was provided these data and the diagram developed by the pharma organization, it shows the drug development process. and starting on the left, it shows you start with many thousands of compounds, up to 10,000 compounds at one end, the beginning, and after 9 to 13 years, you may end up with one safe and effective drug on the market. the clinical development phase, which is the gray phase, the middle phase on this diagram, is the longest and by far the most expensive phase. in contrast, the fda review phase of which much attention has been paid to is actually the very small slice there, the white slice toward the end of the process right before the drug gets on the market and is typically at this time less than a year in duration. so fda has made strenuous

efforts to help reform and modernize the clinical development phase of drug development because that is the major bottleneck. we not only is it expensive and long, many products fail in this phase and there's a tremendous opportunity cost there where other treatments could have been developed. now it included several innovations to this process and the most striking being the breakthrough therapy designation program, so if we could have the next diagram, thank you. this was mandated by congress and was specifically directed at that clinical development phase so that we could help when therapies were particularly promising and were designate d, we could help move them through that phase more quickly. the designation has been enthusiastically described. we have had over 160 requests in

the two years since the legislation was passed. we have actually, and this is the really surprising part, we have granted 52 designations. so what dr. burgess said we're on the verge of a new era of therapeutics is reflect ed by this. we would not have seen this a decade ago. we have approved six products, four new products and two new indications. now it's too early to judge really the impact of the breakthrough designation program. is it really going to be able to speed up drug development? however, i will say the four products we approved, their clinical development time was 4.5 years. so much shorter than what i showed in the earlier diagram. also where clarifications of accelerated approval and we issued a final expedited guidance in may that includes in

response to the stake holders request examples of rare diseases and includes more information on the use and rare diseases. however, it is clear much more needs to be done to modernize the clinical trial process. that's the big bottleneck of giving discoveries to patients. this can't be done by fda alone. we don't this process. and i think the series of hearings that have been held in the 21st century initiative can help provide the framework for significant reform in this proce process. >> chair thanks the gentle lady. i will begin the questioning and recognize myself for five minutes for that purpose. dr. woodcock, what is fda's plan to advance biomarkers and the use of patient reported outcomes data during the drug development process and post market setting? >> many years ago, a decade ago, we recognized that there was no

structured scientific process to provide the evidentiary basis for use of a biomarker in decision making. so doctors and biomedical researchers would float new biomarkers, but there was no rigorous process to see if they were really useful. so we actually established a process for this. it's not really in our mission, but we established it. it's called the biomarker qualification process. and we also worked with the european medicines agency and the japanese regulators so this would be a worldwide activity. and con sor sha can come into the fda and propose a new biomarker and we'll give them advice on what needs to be done. and also for patient reported outcomes. and if that evidence is developed, then we'll publish a letter that's public and so will the ema if they accept it.

and then any developer can use that biomarker or measure in a development program and will rely on it for the context of use. we have 79 projects by different con sor sha. >> good. describe your plans for implementation of the structured benefit risk framework you mentioned that transparent to the public and the sponsor so that the assessment of data from critical trials can be better understood and acted upon. >> this is an iterative process. we have had public meetings. then we went back and were piloting this in a different review division and having the medical officers work through this framework that we have

developed and see what the results are. then when we have that, those results, we'll go back through a public discussion and talk about how i get input on how this can be improved. so this is not something that can happen overnight. it it is a scientific process and actually we feel that we don't have the tools right now. they exist out in society and science, but we haven't applied them. these rigorous to the benefit risk decision. so we have had workshops on this, scientists come in and advise us. we will have a public process once we have gathered more experience. >> i have been hearing a lot about fda's efforts to improve the quality of pharmaceutical manufacturing. where do u.s. drug manufacturers currently stand when it comes to producing quality medicines? can you tell me a little bit about your plans in this area? >> well, i think the major

problem here is many of our central drugs are not made in the united states. and they are made all around the world. and sometimes they may only come from a single source. and this is, i think, a real vulnerability to medicine. in addition we used to be a manufacturing power house in drug manufacturing, but those jobs have moved offshore. and i think now we have an opportunity with new modern manufacturing methods such as continuous manufacturing to actually build a high-tech industry in the united states that actually will make the drugs we need here in this country. and fda has been collaborating with the community, manufacturing community to help bring this about. we are very interested in seeing this happen. >> we have recently heard a lot about lung map, the lung cancer protocol trial.

there are other examples of several trial designs like i spy for breast cancer. what else needs to happen before these types of trials are no longer front page stories? >> that's a good question. we also have been advocating for this for many years and it's wonderful to see it start to become a reality. the concept, i think, in drug development needs to be turned on its head in clinical development. instead of for each investigational drug, there's a whole clinical trial program developed that take very long time, as you heard on wednesday, to get started and so forth, that there are networks that are available that investigational drugs can be plugged into. this will provide independence of assessment, but really decrease the time and expense of assessing whether these drugs are safe and effective. but what needs to happen, i think, is we need to expand this

to more diseases. the nih is very interested in antimicrobials and setting up a network and other groups are looking into this. i think you may hear today from some patient groups say cystic fibrosis has successfully set up the infrastructure to have cystic fibrosis drugs rapidly evaluated once they reach the clinic. >> chair thanks the gentle lady. now recognizes the ranking member for five minutes for questions. >> since i just got here -- >> you want to yield to green? >> yeah. >> mr. green? >> thank you, mr. chairman. welcome back. i want to thank our chairman and ranking member and dr. woodcock for testifying. in time we have an opportunity to target specific patient populations, advanced

personalized medicine and transform how we approach the prevention and treatment of disease. one of the things worthy of exploration is personalized medicine, which a patient may be able to receive more tailored drugs and treatment suited to his or her certain condition. our understanding the human genome is a key to that goal. researchers tell me and other pieces the potential for research and developers to discuss these drug and device innovations with patients during the development phase. dr. woodcock, can you give us your views of the up sides and down sides of any increasing per misblt of communication between patients and developers during the clinical trial phase? >> it's a very interesting question. we have seen from the 1990s where only 5% of drugs were targeted in 2013, 45% of the drugs were targeted in some way. there are barriers to locating

patients and joining up patients who have specific conditions subsets with appropriate velgs therapy. and as diseases are fragmented into smaller and smaller subsets, it's harder and harder to find these people who might be eligible for giving therapy. the lung map trial is one way of doing that. it has multiple investigational trials and people can be spread out, but there's great interest with more patient activism in using social media and other ways to actually match up the right patients with the right investigational drugs. and i think this is one of the challenges right now of the clinical trial enterprise. >> well, increasing patient involvement in da's decision making surrounding drugs and devices is a significant yet challenging endeavor. can you provide your suggestions on how mechanisms need to be developed to measure what

meaningful outcomes for patients are both in the clinical outcomes and the quality of life? can we do that? >> yes, that's what i was referring to with chairman pitts is that there's a science of measurement. patient reported outcomes is one science. how do you measure how a patient feels from their point of view and there are ways to do this, but these measurements have to be developed. we approve many drugs based on their impact on quality of life. so that is completely possible. but what needs to be done is the science needs to be developed. we are participating in that. we have an expert meeting next week on patient-reported outcomes. >> patient involvement and process has to be data driven to improve the efficiency of drug development and maintain fda standards of safety skpef skpefbtiveness. how can congress support the fda in incorporating decision making

in a way that helps deliver that innovative medicines to the patients sooner? do you need statutory authority or do you think you already have it? >> to my knowledge, we have the authority to do this. and i think you will hear from the next panel, for example, how patient groups can develop graft guidance, they can run processes that actually incorporate all their points of view and those of the expert scientists. so more of that needs to be done, but i don't know that it needs more statutory authority. >> can you do it within current resources? because again, you're specializing instead of a broad brush. i assume it costs more when you're doing individual. >> yes, well when you have 7,000 diseases that need good treatments and most of them don't have them, it would be very difficult for fda alone to develop the standards for a patient-reported outcomes much

less the clinical outcomes. so much more participation of the medical and patient community is needed in drug development and we need to find better ways to do that. but i'm not sure that it's through legislation. >> okay. without a doubt, greater resources, but our committee has worked over the years to try to provide those resources to the fda and look forward to working with you. thank you, mr. chairman, i yield back. >> i recognize myself for five minutes for the purposes of questions. dr. woodcock, good to have you back in the committee. so we've talked about how the fda routinely works with sponsors to apply flexibility including the use of biomarkers, nontraditional trial designs and other available tools to expedite the development of products to treat both common and rare diseases. with respect to the common diseases, how is the fda working

with sponsors to apply these innovative development and review methods? >> well, for example, hyper tension is a common disease. we approve drugs based on a surrogate measure, blood pressure that's very well accepted, and for a number of years ago, we look at automated blood pressure monitoring. we decided it was unbiassed and so we decided that you really didn't need a control group in the same way that you would for most other diseases because you have an unbiassed measure. so we issued new approaches to studying in a hyper tensive medicines. so that's an example. >> what could happen so that the fda could use this more frequently? >> well, 45% of the drugs that we approved over the last several years used a surrogate end point. so we do use that when it's appropriate and it is available.

for many diseases we don't know what the right surrogate is. that's why many of the accelerated approvals have been confined say to cancer and hiv is because the great deal of science has been driven in those conditions and we understand the biomarkers. but for other diseases, there needs to be more scientific development. that's why we're using this biomarker qualification process to try to get more biomarkers developed that we can use. but we can't just dream them up and use them. >> i thought that was your job. let me ask you this. are there situations where a majority of the scientific or research community believes that a biomarker predicts a clinical outcome but the fda has hyet to accept that? >> there may be. there's a lot of controversy over use of these. you heard some of that on

wednesday. there are two sides to this. if you rely upon a surrogate often, especially when it isn't well validated, there's more uncertainty about whether the drug is going to work or not. so there are different points of view. and as we have all been saying, the community, the patient community really ought to have a lot of input into how much uncertainty should be tolerated given the circumstances of that disease. so there are situations where there's disagreement amongst various parties, external and internal, about the use of a surrogate. >> are there any -- are you able to give us any examples of that, of a surrogate that the fda may not right now be willing to accept? >> well, for example, raising good cholesterol, we had a series of trials on that. everybody thought raising good cholesterol would be really good

and in fact, it turned out to be either neutral or in one case it actually increased mortality. so we no longer accept that surrogate. that's the kind of example where there are many others like that in bone density. for osteoporosis, estrogens do very good job and decrease fractures, although they have other liabilities. some other agents were tried and they increased bone density, but they also increased fractures. so we have to be careful when we use these surrogates to make sure we're getting the intended result. >> thank you for that. let me ask you a question that's a follow-up from when we visited in april. do you have an update on the status of the fda's guidance on biosimilars naming and when the guidance will become final? >> well, i certainly would like to get that guidance out as soon as possible. we are working diligently on

that. i don't have any further update. >> but that was submitted as a question in april and we are awaiting an answer. now also along with that, i asked if anyone in the administration outside of the fda had provided the agency with suggestions or recommendations with respect to this guidance. if the answer to that is yes, can you provide us with the names of those individuals? >> we'd have to get back to you on that. >> we anxiously await your answer. my time is expired. i'll recognize the gentleman from new jersey, the ranking member, for five minutes for questions. >> thank you. mr. chairman, you asked a lot of my questions so i'm going to have to move on to other things. but dr. woodcock, we heard a lot at wednesday's hearing about the accelerated approval program. as you know, the program allows for earlier approval of drugs

that treat serious conditions and feel an unmet medical need and are approved on surrogate end points, which we also learned about on wednesday, and the critical requirement is that companies conduct studies to confirm the benefits suggested by the surrogate end. point and these studies are called phase four confirmatory trials. so a critical part -- i wanted to ask about the phase four trials. what challenges has fda faced with respect to phase four tr l trials? do sponsors complete them in a timely manner? >> it is sometimes difficult to complete these trials. the reason is that if you had a serious and life threatening disease and we approved a treatment for it, you probably would be somewhat reluctant to enter a trial where you had maybe 50% chance of not kbeting the treatment. so what we often do is ask that trials be conducted in a different stage of disease or something where it hasn't been

studied yet so we can get the results and that might take time. so i think in the early years of the program, it didn't track this as well as we should and we did have a lot of trouble getting these trials completed. but in the current era, we are on top of this and generally speaking, the sponsors are diligent in trying to get them completed, generally, but they have difficulties sometimes enrolling patients in these trials. >> another important component of the program is when surrogate end points don't show the clinical benefit, fda could be phased with needing to remove the indication or take the drug off the market and i imagine that's no easy task. can you describe what's involved with taking a drug off the market and what challenges does the fda face there? >> generally if the confirmatory trial fail to show benefit shs

the first thing we ask is the sponsor to withdraw the indication from the market. it's only if the sponsor does not agree to do that then we go into a long administrative process, which includes hearings and findings and so forth. this can take a long time if the sponsor contests our finding that the drug isn't effective. >> now just a couple years ago, we included some provisions to improve upon the approval. for example, the law made it clear that fda could rely upon evidence developed using biomarkers when tools in assessing end points. can you describe what impact those changes had on the program and are there any other changes you feel are necessary to allow you to make full use of the most recent developments with respect to surrogate end points?

>> i think the legislation was very helpful. we have taken it quite seriously. we have issued guidance, final guidance on expedited programs. probably the biggest change that the legislation brought about was its focus on intermediate clinical end points. we had to have quite an internal discussion about what that means. i think you will see us approving more products under accelerated approval based on these intermediate clinical end points. >> well, thanks. it's clear to me that this is an extremely complicated area and one that's not conducive to further legislation. but i wanted to ask last about the master protocol. at the hearing on wednesday, some panelists describe some of the inefficiencies that exist in the way that clinical trials are currently conducted and one of the suggestions for addressing those inefficiencies is to create a master protocol. i wanted to ask first can you tell us more about this?

what's the master protocol? how would it improve? has fda been involved in the development of a master protocol and are there dezs that the protocol is more appropriate for than others? are there other areas it might be expanded? >> master protocol is one version of using clinical trial networks or standing clinical trials to evaluate investigational therapies. it isn't just for one therapy. it's for any therapy for that disease. so a master protocol has to be disease specific. you can't just have a general overall master protocol. it has to be focused on one disease. for example, the lung map trial is on cancer of the lung that is advanced. but five different agents right now are being studied all at once within that protocol. and that's a huge efficiency.

but there are other versions of standing trials or trial networks that also could be used in other diseases. as you said, the cystic fibrosis foundation has a network of clinical excellence where they have actually sequenced the genome of all their patients. so they are ready when a targeted therapy comes along. they are ready. they can put those patients into the protocol and that tremendously improves the efficiency. so it's a long conversation that probably can't be had in five minutes, but i have long advanced this concept and tried to push this concept because the current clinical trial paradigm is not sustainable. >> thank you very much. >> the gentleman's time is expired. the chair recognized dr. murphy for five minutes for questions. >> thank you and good morning, doctor. . let me start by asking it is

important for the medications and research to advance, but also for those that have approve ed. so let me ask you, we had passed the laws awhile ago. certainly mr. griffith just moved between me. that helped -- supposed to help us get more generic drugs. what's happened is we have 1.5 billion over five years. but what's happened is approval times have gone up and there are fewer approvals even though the law was supposed to reduce those. can you give me indication of what's going on? >> certainly. we're well aware of these issues. in june we received 625, i believe, generic drug applicati applications. so the rate of submission is well above what was projected in the negotiations that we held. however, on october 1, the deadlines kick in for timelines for review of generic drugs.

we are fully prepared to meet those timelines as well as deal with this large backlog of pending. we had to hire a large number of people and totally revise our process processes, reorganize the review, offices and conduct many other changes and that's what we have done over the past two years in preparation for the deadlines coming into effect on october 1. >> thank you. another question here about some labeling issues. the abbreviated new application that would allow generic manufacturers, this is a proposal for fda, to change the label without fda's prior approval but come back later on. as the fda recognized kinlt labeling will ensure health professionals and consumers that a generic drug is as safe as its brand name counterpart. but there's a concern that allowing the changes to take

place and go back fill them later on can cause a lot of confusion in studies that have asked. i'm wondering where this issue stands in clarifying this. >> we have received kmen ed com the proposed rule and received many comments or analyzing the comments and subsequent to that we'll have to go forward with a rule making process. the proposed rule contemplated that e we would actually have less despairties of labels in the marketplace on this because of this proposal. because we would put up a website and also require conform mans of labels, which we cannot carry through right now given the current systems. >> but the committee in january has to meet with others about this. i'm not sure those things have taken place yet. so i hope this gets expedited and these issues are addressed.

it still leaves some confusion. i'm not clear yet in understanding why this proposed rule was set up to allow this individuals to change the label and come back later and ask permission. >> well, currently generic labels do not always match the innovator and do not change their label in a timely manner. so there will be labels out there for quite a bit of time even with serious safety issues like new box warnings that don't conform to the innovator label. so we are trying to address the situation and also as generics are now 85% of all drugs dispensed to consumers that they should have the opportunity since their drugs are the ones that people are being exposed to to submit their findings of adverse events and suggest label changes, proprosed label changes and execute them. >> i hope you'll meet with staff members and companies to help

clarify this because it's stimnot clear to me why this will be allowed. i think it would be confusing. i want to bring up one last thing while you're here. i had sent a letter a few weeks ago to dr. ham burg, i'm sure you didn't see this, but one of the things out there is complications that are reported in the media about caffeine. whether it's sometimes toxic levels people take through over the counter things, whether it's pure caffeine or supplements for athle athletes. yet it's also in everything from chocolate to coffee we promote all the time. i'm hoping fda can also give some recommendations in terms of individual levels per drink, per dose, per day, for male, female, weight, age, whatever that is because it's still pretty confusing whatever those products are that they can be beneficial.

i hope you'll expedite that. >> thank you. >> i yield back. >> chair recognizes gentle lady from california for five minutes. >> thank you for holding this hearing to our chairman and ranking member. thank you. dr. woodcock, for your testimony. this is an issue very dear to me. as you know, i'm concerned by our nation's history of excludeing minority groups, especially women, from all levels of medical research from the lab rats to the most advanced clinical trials. reports have shown even when these groups are included, there are too few participants in the groups to analyze the effects on them or the analysis are simply not run or reported. i'm sure you're familiar with the case of ambien, a medication that had its label changed because it metabolizes differently in women than men meaning women had been receiving an inappropriately high dose of this drug for over 20 years. in addition, this spring a report entitled "sex specific medical research" was released,

which provides evidence for the further inclusion of sex and gender in scientific research and the fda's own august 2013 report, which was initiate d by the inclusion of my heart for women act in the legislation show that there's much work to be done to make sure it's available. i know the fda is continuing to work on an action plan to address these despaisparities. so can you give us an update on where the agency is on this? >> certainly, i would expect that that would be released, we would be timely in its release. i believe there was a statutory deadline or there was some expectation. so we're working diligently on the action plan, yes. but i will say for drug development, which was what we were discussing here, that we did a study, for example, of the

2010, the class of 2010, the products that we approved. we found that 45% of the participants were male. and we found that almost all the submissions included the required gender analysis which is required for drugs for 20 years. because i oversaw that when i first joined the center for drugs in 1994. so by regulation. so we do have these, but i think the transparency of the information is the problem. we are working on that. i really am committed to making that information more transparent so people understand what we know and what we don't know. >> i u think you put your finger on something. i want to highlight a bipartisan letter i led urging this agency to include clear and actionable strategies, and i think what you

said about transparency and the reporting in the action plan is a way to address this issue once and for all. at wednesday's hearing this week, i also asked the panel about the tools fda is developing that could supplement our knowledge base, especially in the light of robust clinical trial designs. the fda sentinel system, which is making progress, if slowly, to conduct post market passive surveillance of drugs and devices could help spot issues like adverse drug interactions more quickly. the program holds great promise, and that's why i work to get assurance for effective devices act included to continue progress on the program and ensure that we design for both drugs and devices. could you update us on the development of the sentinel program please and what other resources or authorities do you

need to get the system up and running to protect consumers more effectively and expeditiously? >> i think use of electronic health data, which is rapidly becoming available in the electronic health records and so forth, has tremendous promise for actually finding out what happens in the real world for medical products, both that are approved reelt recently and those that have been on the market a long time. that's what the sentinel system is intended to do. we have run the network for five years, that was between drugs and biologics. we paid for that out of the money that we have. we are recompeting that to put up the sentinel system. that is out on the street and we hope to establish the real sentinel system, which will be a large scale system for surveillance. now as far as medical devices, we require in a unique identifier or some kind of

identifier in the identifier or some type of identifier in the medical record, so we can capture that, the sentinal system uses the electronic systems to get the information and i will repeat for everyone, that it does not take any personal information and move it to some central database, it is strictly running those analysis within the health care systems and then the results only are combined. so, that has tremendous promise. we feel very good about that. we actually are piloting running active surveillance on there. when we approve a drug and have a question about it, we can watch over time and see what actually happens. as who and who people get on electronic health records we can have more insight in what is happening. that is where we are in that. it is resource limited. i have to pull resources from other activities to fund that. i believe strongly that this is the future. >> thank you, i appreciate that.

>> chair, thanks, gentle lady recognizes the doctor from georgia. five minutes for questions. >> thank you, mr. chairman. and thank you dr. woodcock for appearing, did good to see you as always. i understand that a number of calendars that have contributed to the cost of clinical trials are are outside of the fda's purview. that being said, clinical trials are used to generate evidence to get approval from the fda, how often do you typically talk to the companies, to discuss their trial design before the investigation of a new drug application is submitted? >> well, we have agreements that

prescription drug user fee program. say they are testing in a disease that does not have treatment. companies can come in and have a preimd meade i preimd meeting. that meeting is before the clinical trials, and we talk about the development program so they can start thinking about how that is going to be done. >> we do have information, preliminary, butting looking at our information, it seems that companies that have more interactions with the fda, are able to get their products through more quickly. through the entire clinical trial process than companies that have not had interaction with the fda during the development process. but there are formal meetings that are held at different times under the user fee program. and those minutes are tracked and we track the meetings and so forth. so, there's quite a process for interaction during drug development. >> so, you as a manager would be

maybe at that particular time, you make sure that your reviewers are not requesting overly burdensome data, that is not necessary so that the process can be speeded up. >> there's always a push and pull, scientists of all stripes want more data. scientists in the companies and scientists in the fda, so we have to walk that path between you know, getting more data and actually the costs that is generated, and we have made a number of efforts under the city collaboration that we do with duke university and many, many, many other partners, to try and figure out how to streamline clinical trials as far as data collection, for example. but it is, it is difficult. we have 1600 meetings a year. under the paduf ama, and when w meet with the companies the senior medical officials are

also at the meetings. >> that's the whole purpose of 21st century curesing, as we get to the second panel and we hear about the associations and from the tammys, i'm sure they are going to talk about how we can speed this process up. let me, the last question, our first 21st century cure. we heard that only 19 drugs outside of cancer and hiv space have have been approved by the accelerated approval path way since 1992. and i understand that you wrote a blog post after that hearing, how a number of drugs that were being considered under a accelerated approval, received traditional roo aal approval an statistics were somewhat misleading. can you provide examples of when it occurred? >> for certain rare diseases, we may decide for example, that the surrogate is fine. okay, and it correlates with clinical benefit.

then, the term accelerated approval is a little misleading. it sounds like it's faster than regular approval. but, actually, if we approve. we give regular approval on a surrogate, it's just as fast as accelerated approval, but you do not have to do confirmative studies afterwards. because we already believe the surrogate. for the rare deefficiency diseases, you can show when you replace the protein in the body you give the activity back to the person, right, so you may not have to show clinical outcomes, it still a surrogate that we feel it's 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. so, in many cases, we are able to do traditional approval with a sursurrogate, meaning the patients and sponsors do not

have to go through confirmtory trials, i described the difficulty of that when you have a serious disease and you pro approve a drug and you ask people to be randomized for testing. >> 5 minutes for questions. >> well, thank you very much, thank you dr. woodcock for everything that you are doing to assure safe and effective drugs are available for the american public and those with health challenges. patient, this is a hearing about the 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 trying process. i have long been a sporter of the directed medical research programs, known as the cdmrp,

they fund peer research in to you a simple, cancer, and other diseases. and since 1993, the patients have been involved, have been a part of cdmrp, they have a consumer reviewer, as part of a panel to represent the stakeholder community. and it has been very successful in combining patient perspectives and needs with scientific research. bringing those perspectives together. has fda, are have you begun to consider improving patient involvement? have you looked at cdrmp to see if there's anything that you can borrow from that in the drug approval process? >> yeah, we have not. and that's a good suggestion. we would be happy to do that. >> okay. you mentioned previously that the patient focused drug development initiative that was

put in padufa was designed to let the fda hear from patients on how a disease impacts their life. and i understand you're scheduled to have 20 public hearing. have you started the hearings and if so, what have you learned from them? >> we have learned the devastating impact of the diseases of these different diseases on people's lives. it's just incredible. we have had one on hiv, lung cancer is, sickle cell disease, hypertension and we planned to have 16 of these meetings completed by the end of 2015. but we recognize this is just a drop in the bucket of the -- of what people suffer from. so, what we are trying to do is really model how people can do this. and hopefully could be done

more, not put on by the fda, but by the patient groups themselves and the medical community that serves them. so, that they can aassemble more of the information and kind of multiple the effect yof this an we are seeing some of that. we have had help offered with rare diseases, for example, to have more input that way. because we are -- our resources are limited, we are not going to be able to cover all the different diseases. >> good, so i expect to hear are from the patient organizations later today on -- in their view on how they can be helpful and we can be we can be effective. i think the information we will gather through the electronic health record. it's interesting to hear what you have done with the sentinal

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