because the expertise is being developed as we speak. there's a tension about what might emerge in the next few years. efb is looking ahead to see what's coming. talking about things, some of which will never come, some of which will come in two weeks. ro lots of conversation about interdisciplinary lives. the line between the inspired and the insane is very difficult to discern. it's either crazy or is quite brilliant. very hard to know. a lot of talk about definitions, sort of fundamental definitions. there is a gee wiz quality to it. i use that as a compliment. you know, these days, if you go to an internet law conference, you go to a copyright law conference, there's a lot less gee wiz and a lot more oy vey,

is the way i look at it. the lines are drawn. i thought of that last night. you know, the lines are drawn, the winners and losers are clear. if you two this way, here are the party who is go -- everybody knows what everybody else is going to say. it's all been laid out. and this is reminding me of what it was like in 1995 in cyber law when that was not true. and this is a real opportunity. it's a fabulous opportunity. just as it was in 19 t 5, you're behind the vail of ignorance. so you can think about rule making and law without the winners and the losers piling in and fighting over it purely as a sort of economic battle ground. it is about the ideas. and it is about the issue. and i think that's what he's talking about. he wants to take that energy

here. i don't think cyber law structure is the right one. i think cyber law process is the right one. he talks about interdisciplinary prague mattism. the attention to metaphor. it's ault about how people were thinking about it that could be transplanted in a way. i thought, when i was talking to aaron, just to break a while back here, he had a good metaphor. cyber law is the boat, the last boat that just sailed a while back into these seas of, you know, uncharted seas confronting the new disruptive technology. so we're looking at cyber law. robotics should emulate where it was sailing but not how it was sailing.

i think it also includes a deep distrust of experts, a willingness to entertain experts, no matter how crazy they are. i believe it was impossible to document, but i do think it was a very important part of the early cyber law with community that helped us figure [expletive] out, as they say. two more quick comments about his part three arguementeds and then we'll open it up. one of the things that made cyber law distinctive is what the hell are we talking about? it was a focus of much discussion in debate early on. does the subject matter really

exist? a lot of time with international law scholars these days. they should do more of that kind of thinking. they could use some of that thinking. we thought a lot about it in the context of this troep. since i got your attention, i'll tell you the story. anyone who's familiar with cyber law knows what i'm talking about. many of come from different communities and may not. 1999 was one of the first c conferences on cyber law. a lot of the people who were then interested in internet law, cyber law were there. it was an important moment, i think, for the field. university of chicago law schools, an important

institution and was sorts of a validating of sorts. in a way, the first discussion was frank easterburg. adjunct professor at university of chicago law school, very much identified with the university of chicago law school. it's like he's going to, okay. it's such an important figure would come and kick off this new cyber law conference. this is the moment. of course, it's not exactly what happened. first speaker tells a roomful of aspiring cybers and academics, there's no such thing as cyber law. he begins with the story the dean of this law school, he says, is always proud to say we don't offer a course in the law of the horse.

there are lots of legal issues about horses, about medical treatment for horses. but nothing is gained by bringing them all together into a course on the law of the horse. there's no illumination of either horses or of the law generally will be gained by bringing them together. and cyber space law, easterburg says, is like that. it will not illuminate the law and it will not illuminate cyber space i don't remember, michael, if you were there. he just picks up his paper and walks out, which he does, at some point. he just walks out. you know, talk about a buzz kill. welcome to the conference. go home. now, easterbrook was wrong. it was about time somebody came through.

>> i'll say it. he's wrong about cyber rule. the university of chicago does not have a course called cyber law or the law of the internet.

a lot of courses about horses. so here's my final. so ryan asks the law of the horse question. in a sense. what makes 5 technology, whether it's internet or robotics or railroad or radio or vaccines. what makes it exceptional in the law what makes it worthy of study on its own. what makes it not the law of the horse, in a sense. and the office's definition, when it's introduction into the mainstream, requires a systematic change of law in order to reproduce or, if necessary, to displace. he looks at rate of railroads. ziszmatic change to the law. he looks at the railroads. railroads categoryize the uptake of negligence as the liability rule. an exceptional response. radio formation of the fcc.

vaccines. necessary to keep the law doing what the law does when it works. so these are exceptional in a sense that they require these major league calibrations. so i'm with them all the way. yes, it's really interesting. and then he turns to the internet and says no, not so much. i don't see any information in cyber space. unlike radio u he says the internet did not lead, for example, to a new agency. i think as you begin this discussion, i think you have this completely back wards. in a sort of magnificent way, by the way. it's maybe harder to maybe write back wards.

that there's no testament to inception. there's no new agency because nobody can figure out what the hell this agency would look like. and how it would do what it's supposed to do. the fcc will not know what it's about. how do i figure out how to get its hands about. all the discussion about whether it is or is nots governing the internet. it reflects how exceptional this is. not that it is not exceptional.

if i have reason to believe somebody in belgium, an twerp, has committed a crime, i go to get a search warrant from the duly authorized belgium authorities to search doing whatever i need to show to them. and to search his e-mail inbox. the data is in the cloud. what do i do? a cross border search requires permission from the sovereign in his territory. the search proceeds. it's like an invasion. it's like the belgium police

come and i'd like to see the records. i have a search warrant. the bank is not going to let them in. that's not how it works. you need permission from the sovereign. whose territory is being invaded. whose permission do i need to get. they had no idea what that entailed. this was very smart. people who deal with this law every day are very sophisticated understanding of their obligations. it was hard. 2014, we still have not figured out the answer to that question.

the legal system has not been. it's true there's been no systematic change in a legal doctrine. but that does not make it unexceptional. did not if i can your out the answer yet. it's because internet technology is so exceptional that we don't -- we have not yet figured out what the systematic change is going to do. okay. it's a little bit about cyber law. so go build your boat. and build this field up. i don't think you need the umbrella of cyber law. i'm not sure, in fact, it will help you get where you ooir going.

>> oh, i forgot. so i'm not going to say -- i'm not going to say much. except for a couple of quick things about it. first of all, thank you very much. it really is an immense honor to have you commenting on this paper. and, by the way, this is not -- i mean, you can see that heavily into the draft, this is not just sitting next to me. i really do have to recommend david's book. it's really very interesting in terms of basically teeing up for what the on going questions are giving you history and then analogizing to a previous moment in history. anyway, that's a really, really great read. so as to the substance of your

comments, it was a little bit hard to pay attention. that's helpful, yeah. so, anyway, i just want to say two very, very small things before opening it up. i really just want to hear about my community. so, for instance, the notion of emergency, there would be things that would be unpredictable by design. uselessly unpredictable.

and so i did mean to make a point about the law. i just wanted to sort of note that. it shows the dichotomy between instrument and person that you sort of see in law over and over. and i think that's right. i think that the social meaning aspect of robotics is to see that. last thing i'll say is by a new cyber law chrks really goes to thehearted of your claims, i really tend to agree with you -- you're right about what i'm saying. you're right about what i'm saying at one level. so i agree that it is more the procedure that i'd like to copy.

that said, it is extremely difficult to overlook the fact that so many of the meaningful contributors to the robotics law come from the cyber law community. and i just want to say that my decision to sort of characterize it initially as a new cyber law is strategic in two senses. one unfortunate, i guess and the other more fortunate. the unfortunate one is it's just hard to cite, as you've ill lal to. >> you can't say the conference in 1996 was a really cool conference. but that doesn't mean it's not important. it's a true thing about the world. and it's hard to accept.

the other thing that's strategic was i have to say that respect to technology, cyber law is kind of the big game in town, you know. and if you warant to get smart technologies and lawyers together, where am i going to post it if not on cyber law for now. >> and so i win in that way, right? so with that again, with my deep thanks, i just sort of want to open it up to other people's questions. >> i really did not mean to suggest that the study of robotics is the law of the horse.

what i've heard in the past 24 hours sort of deepens me in the authority and the responsibility and my ability that are going to be illuminated through this discussion. but i think it's on is own. i wish it well. maybe i'll even come visit, yes. >> michael, from the university of miami. david says i was there as he was. but i think he might have taken it to form the memory.

i've been teaching signer law for a long time. easterbrook's comments, the prediction that the cyber law, certainly for practice, for how we think about it, that cyber law would be coming a sub field of everything else rang truer and truer. then, it's still not all. but this year, i told my dean that its days are numbered. i got a little nervous about it intellectually. it's disconnected. it no longer has much of a common theme of a network. an that's an intellectual problem. there's still thicks i can do, but they're not as coheernt ren

they used to be. one thing that occurs to me is if robotic social security a new cyber law, does it have the same arc? it's very useful and sensible to understand the technology and therefore can make sensible rules about it in the beginning. i kept thinking i've seen this movie before. standards are going to get ridden and then make waves that the lawyers could have told you are going to cause untold grief and you'll get sued. probably not going to work this time, either, but that's the dream. maybe there's an arc, too, because there's a piece of

insurance law. so the only thing that i can think of that makes it different in the remake are the issue that is we talked about in john roberts. to its simulation becomes so good that it becomes increasingly difficult to make it emotionally and intellectually. and then having ugly flash backs of slavery and all the rest of it. other than that, and maybe that's so far away that that's not even relevant, is it the same movulovie or not.

i think it's interesting that we disagree about the law of the horse. this trout that we've been bouncing around for 20 years. i continue to think that there are unifying principles about cyber law. along the lines of what ryan was talking about. we're not going to resolve that here. what i think, and i meant to include this in the talk, and i'm not sure that i did. it's the met question that's interesting.

easterbrook did us a great favor. and, again, you know, after 20 years, we kind of know where we stand, we're not going to be hashed that way. we're going to have different views about it. but that process was and, again, i think that's what ryan was trying to initiate here. thinking about what made that last panel a robo panel as opposed to a cyber. and then there's some special things that focuses on it.

>> honestly, i lack the context to really answer this question well. the ways that the law will change probably systematically there a new technology that strikes me as interesting enough on its own term that is i don't worry too much about how the movie ends, in a sense. so that's my newfound answer. >> so, ryan, i saw your paper come over the transom some mo s months or weeks back. thank you so much for adding to the thought fro cess process.

but my mental art of thinking about it is well expressed by david. and i'm more in david's camp, which i don't think cyber law is maybe the proper way of terming it. and maybe you didn't even mean that. but what i think about is back in the 1990s, as a younger lawyer, i was thinking cyber law. we need to be looking at this intermedian liability. and the funny thing happened about cyber law is i didn't really practice it too much. but i did practice information technology law. and i ended up working as the second in-house lawyer and i was doing secure electronic commerce, going to michael's point. what i noticed is there are a lot of commonalities. we do recognize that internet

plays an important role. but there are a lot of legal issue that is that draw from a non-network computer to the pod to walk away with it. so i was worried about these kinds of things. so there are all of these kinds of strange strange differences.

what makes robotics unique, in my mind, is the potential for autonomous behavior. that really adds a new twist to thing that is we didn't talk a lot. what does it mean to say an autonomous device is defective from a legal perspective. i don't think we've really come to grips with that. when does it come off the rails from a legal perspective. i think that's the interesting part in my mind. ultimately, if we look back and say the process is right, welt, what can we cite to?

michael was at the beginning of something really great. when we called it cyber law, that was just the beginning. it grew far beyond that. part of the vehicle was the american science and technology law where it was a pioneer in terms of creating interdisciplinary publications, international in scope and those are the things that you can cite to. ryan was a co-chair of the section. this is one of the places you can cite to. >> so, first of all, that's really helpful. especially everything you said, what you said just now. maybe you and i can work together about generating those siets. let me at least say, to me, i haven't thought about what cyber law is or what it means to define a field.

and actually, i angsted over that immensely. what does it feel? what are the methodologies? is it the structure that david mentioned? it's not trivial. fr me, what's important to bring either out of there to export and within the framework of cyber law. one of the things in the mix, because you braugts it up, steven, is what i tend to be so

powerful about cyber law is its emphasis on interdisciplinary practices. one of the things that judge easter brook was obviously going about were computer scientists themselves and they weren't in any danger of winning a nobel prize for science. so that idea of doing it all together is the role of metaphor, analogy. and it's this whole tool kit. it's whether you call it

information technology or cyber law that grew up with the internet and maybe is applied more widely. i'm hoping that that gives you some sense of thinking. >> the section of science technology law is part of this overall mission of the section to bring different disciplines together to whatever the field has to be. >> this is a good phrase. i think it means something.

i think cyber law may have been the first -- maybe i'm wrong about this, but there was certainly a sense and a much-discussed sense, that you kind of have to know something about the technology. you could not talk intelligently about the issues. if you really knew nothing about it. to an extent, you were hoping to emulate that. i think that was a critical part. it was a big part of it.

kate darling, mit. i probably souldsn't say this, but screw cyber law. predoctorate about your paper and he says for the life of me, i can't figure out a legal principle that would apply to the robot that wouldn't be useful for the computer and vice versa. i feel like your paper offers a lot of counter argument. early drafts is called keys to

row molt exceptionalism. i don't think there's many left. corey has been an immenseful help on this. he's a very smart guy. what could he say again? i'm not sure i got the full impoort of it. >> he says a lot of things. h talks about how there's a distinction and we're just talking about software.

>> so this is just software and computers and all of that stuff? yeah. i think you have to draw lines. and even as we talk about software, we're drawing lines for reasons. so i'm going to leave it there. and, yeah, i thank you for bringing that up. >> i love the paper and i think david's exple case of it is the single most entertaining over the last two days.

cyber law agenda is the law of the horse chlts they ooir all the settled. privacy is one area and robotics is another. a lot of people might say what's a list serve? it's this old sort of telegraphic mechanism like a mimiagraph or something to that metaphor. i think this is what ryan is

saying. cyber law, as a spirit, as a set of tools, as a methodology, as an openness to new ideas to interdisciplinary sharing and clap ration is called privacy. i think that's what we can take in the last panel. that the law falls behind.

it's what we call cyber law. but, that said, easter brook was right in the long term. >> no, we have one of those. >> we've had hundreds of those. and what makes this one -- and, right, there was privacy is obviously sort of a core challenge in this domain. we had a conversation in privacy and lots of different -- not just cyber law, but obviously lots of places in the law. the challenge is to arctic late what makes it different when it's a robot. we have an answer to that.

it is -- something is different about it when that happens. this is not the same old privacy question. it's been new on the table when it's a semiautonomous agent. they group together, if at all. it's hard to ignore the fact that some of these people, are, without consciously saying so, grappling with the exact set of issues.

that is a question for sort of us. and people like henry and many others sort of noodling through the infringement and ownership consequences of machines creating copyright. it's like these are cyber law folks. part of what an mates are now grappling with a different set of issues.

>> in the 19th century, there was the field of corporate and commercial law. so these things have a life cycle. robotics, not only has it not been used, robotics 1 a subset of cyber log. it's a state of mind. we're making due with the tools that are there to try and mog you will through problems.

>> let me -- identify yourself? >> you're supposed to identify yourself. >> oh, yes, sorry. ken anderson. let me speak on behalf of those who were not around for any of this in 1985. not because i was too young, but because i'm not sure what i was doing. i just remember, vaguely, coming into this law. this is like this cool area, jamie boyle, a very close friend and it seemed like he was intellectually playful and too cool for anything that i did. you know the sense of gee wizness. this is, in somece sense, a ver

strange feel that has a certain sense about staking ownership intellectually in the next camp. rhymyan, you know i'm the biggest fan of this paper. but i believe if we get outside of the conversation with the people who are part of the cyber conversation from before, which notably, everything here is really focused on, i think that the part of the pher that's going to be the most lasting in terms of generating the most basic ideas is your identification of embodiment, emergence and social meeting chlgt i would actually suggest separating out those two conversations. and what grabbed me so much about this paper was precisely your articulation of the

features that made this different from cyber. that made this different from automation, that made this different from these other areas and said something to me about kwha's going to make this different about the area because they're coming into a social life are going to wind up addressing the social, regulatory, legal, all of those. you were in junior high school. did you have to get per pigs? sorry, that was uncalled for. i think cyber, at least to

me and looking backward, that this was an empty untamed, shapeable, and metaphors would be the sort of thing that is plan themselves and all of that stuff. i think the biggest difference is going to be for the reasons you articulate anything that's done, that's going to be the case for em o bodying machines coming into people's homes. into all of these facilities that we have a robotics state and it's not a sort of unclaimed space out there. moreover, it is not an unclaimed space in which we kind of, by fiat, decided that it would be governed by contract principles of an enormous sort of

privileged space for consent. and no damages. so i think it's largely about a space that's already occupied. i mean, half way through it. his suggestion about the part two is really an important contribution to sort of help set the am xxs, if you will. or at least begin the conversation i'm not sure about

that. they grow. and they matter. law of the horse was one of those for cyber law, code is law, was another one for cyber law. they summarize and set the path. everybody knows and talks about them. i think you brought up a really interesting point. i teach media law. for a long time, i taught social media law. and now, the more i say that word, the less it makes sense. michael made this point very el

kwently. it reminded me of a quote by weird al yankovich. someone asked do you ever feel you'll be unpopular. he said no. i teach pop culture. so i wondered. if -- >> no need to feel. >> right. right. so i wonder if there's a new framing that we've been missing in all of this. because it seems as though everyone is right here in a certain sense. so there are unique things about cyber law. inevitably, they will become assimilated. is it possible the new framing could be spg like law and the assimilation of technology? that's really what we're talking about. and that way, it goes on forever. so whatever we're grappling with, you know, pop culture and weird al yankovich, we can

always be assimilating new technologies. >> like the bourge, right? >> right. right. exactly. that's a framing that we can call upon to keep this going. and maybe we keep from talking past each other also. >> yeah, and sort of in a totally underdeveloped part of the paper, i try to suggest something like this by saying is cyber law the law of the internet or is it a way by which to absorb new technologies. i like your idea of framing the whole thing that way. . so, i even say for a second, maybe we suggest a methodology to do that. the methodology is what are the essential qualities of this new technolo technology, what new experiences does it create and how does that differ? i like that frame and rather than just refer to it in

passing, i might shape part three that way. >> i want to throw a monkey wrench into this. i have the opposite opinion of what woody just said. i think we need to be a little more critical about the monikers we throw out there for potential self-interests. i'm not saying that you did that, but as a community. and think about institutions we need in order to advance the thoughts that come out of that area. so incredibly important, but have been immersed into pretty much existing fields. so institutional purposes or do

we want something to advance the fields and the understanding. so writing on what i did mostly is autonomous weapons systems. everything that's been styled a robot at this conference doesn't strike me as what i would think of as robots. so i would just throw that monkey wrench in addition and say is maybe one framing device autonomy. i think i heard you say that, right? and to me, robots are nice, but a lot of the things that was on the questionnaire yesterday threw a lot of stuff together. i know this was hopefully done, and i'm sure it was done on purpose, but what do we think of? do we mean the iphone and the switch on the wall to turn the

light on, or do we mean something different? that i would like to hear more in the paper discussed and fleshed out maybe in that way. historically speaking, i don't think we've talked about car law in the same way. it was looked at as a way of looking at the fields that were most relevant. i think i'll leave it there as food for thought. >> and i'll take that as very good food for thought. i do struggle with how do we define this stuff and why should we. i do think at one level sort of like a lot of what we do as academics falls into one of two categories. it's like show how two things that really look the same are different or show how two things that look different are the same. so you sort of pick which one you're going to do. or you do both.

but i need to address that better and i appreciate the comment for sure. >> i think from the clock handler, this will be the last question at least goes to you. >> very nice. i'm ian kur from the university of ottawa. i came up here to make remarks largely echoing what ended up being kenneth ander son's remarks about locating key features here that really merit what david described as having our own boat. and i really think that that is an essential contribution. it will be a lasting contribution that comes from this paper no matter what the final format of it was. but since kenneth had already talked about that, i was reflecting autobuy graphically because so many of us have been stemming from the law of the

horse comment and moving forward and i remember as a fairly young academic in 1998 and '99 being commissioned by one of canada's law reform bodies to write a study on whether software bottoms could enter into contracts. that was a question which was unanswerable at the time. and i think that the law reform commission was correct in sort of deciding to give it to somebody who was working on what i was working on in those days rather than a contract law scholar. i think if a classical contract law officer, my mentor who writes the books on contract law in canada, had been given that question he would have fundamentally gotten it wrong for whatever else the rest of us struggled through. that paper incidentally tweeted about because it has to do with thinking about intermediaries as

agents. which was part of our discussion today. i guess the autobiographical in thinking through this is at the time i remember i had just moved from the university of western ontario to join my incredible colleague michael geist who we had been talking about our careers in our role in this discussion in this space. he chose to call his course e-commerce law. i chose to calamine internet law for preshies cystoly the reasons his course would be commerce. and even from the moment i was asked that question about the software bottoms, i guess i considered that while michael was teaching cyber law, i was teaching siborg law and that's something about the merger of humans and machines, which is how i saw the issues that i was dealing with, suggested

distinctions from what cyber law was trying to do that raised new and emerging questions. i think very beautifully characterized by the kinds of descriptions that ryan gave particularly in part too. so mine is more of a comment since ken took my original point, which is to say i tend to agree with david that there may be a value in thinking of two different boats. but the question regardless, especially for people in the room who aren't law scholars and so don't worry about what sub fields in law are, we all know that categories matter. and there's important things that hinge on how these things ultimately become understood. so for that reason, i wanted to commend ryan on a beautiful paper. >> thank you.

>> so as the chair of this conference, i can't resist making one last comment. that is if you look at your program, we have this lovely graphic. you also have a line under there which tems us what we're about. which is legal and policy issues relating to robotics. for those who are not lawyers and wondering what the hell this was all about, i want to encourage you that the policy issues have equal place and we'll see that in the conversations this afternoon where we'll be talking about automated law enforcement and the civilian regulation of drones, and also this is the law professor talking, if you want to make policy that sticks and can be enforced, you have to do if in conversations with your lawyers. maybe you tell them what you want to achieve, but you need them for your agents. we'll have an hour for lunch. thank this great panel.

40 years ago, the watergate scandal led to the only resignation of an american president. throughout this month and early august, "american history tv" revisits 1974 and the final weeks of the nixon administration. this weekend hear the supreme court oral argument united states v. nixon as the watergate special prosecutor contests the executive privilege over his oval office recordings. >> now the president may be right in how he reads the constitution, but he may also be wrong. and if he is wrong, who is there to tell him so? and if there's no one, then the president is free to pursue his course of erroneous interpretations.

what then becomes of our constitutional form of government? >> watergate, 40 years later, sunday night at 8:00 eastern on "american history tv" on c-span 3. tonight on c-span 3, a hearing on speeding up the development and approval of prescription drugs. at a hearing, whistleblowers described the retaliation they faced at the v.a. and the head 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