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tv   Discussion on Precision Medicine and the FDA  CSPAN  December 24, 2015 9:37am-10:55am EST

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one of the most productive areas at the manhattan institute and the sense of a developing of a think tank that is doing the work the manhattan institute is doing on this issue. and the question would go to talk about today is one of the most interesting areas in terms of how we think about how to reform the fda. i think people always complain that the fda is too slow in approving innovative new treatments. one of the areas that has come up recently is just this area of precision medicine. what is precision medicine quack?there are these things cad biomarkers. when you go get your cholesterol checked at the doctor's office, that's actually a biomarker. that's what we mean by the term biomarker. a biomarker is a lab test. it's something that we use to correlate something going on in your body with a particular outcome of disease. so when the case of cholesterol has decades of research to indicate that high cholesterol
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levels, particularly low, low density lipoprotein or ldl, idl levels in your bloodstream are highly correlated to heart disease and heart attacks and other problems. that's a biomarker. 50 years ago maybe we had a couple dozen markers. but because of advances in genetic science and the human genome project and all the things we're learning about how different genes in our body regulate and produce different proteins which regulate cellular process and biochemical process, medical processes and diseases, we are on the verge of having hundreds of thousands of these kind of lab tests, effectively biomarkers that can help us determine a lot of things. they can help us advance more quickly drugs to market that affect certain lab tests or biomarkers. they can help us design clinical trials. for example, there are a lot of
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people with lung cancer but lung cancer isn't all the same. different people for different kinds of lung cancer. it may be responsive to different treatments. and so the fda and drug companies and scientists are going to figure out how to tailor their investigation into the individual patient and the individual patient clinical and biochemical and genetic condition. so these are the kinds of things that are going on scientifically in the world, and the private sector. the question is, is the fda doing what it can do to take it to the scientific knowledge into account in the way it regulates the development of new drugs? and so in that light, peter huber, another one of our senior fellows and paul howard, director of the center for medical progress, have put out a new paper which you have in your folders. which argues that the fda is a little behind the curve in
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addressing how these new developments, new scientific developments, can help accelerate the discovery and development, approval and marketing of new treatments for disease. so we are going to hear from both peter and paul today and we brought two additional panels with a soy think can't eliminate a lot of this discussion. we are going to also from the ceo of emerging biotech company called alter genex which specializes in biotech companies, specialist in the development of treatments for ultra rare diseases. before he was at alter genex was with another company that specialized in this area called one of the largest and most successful biotech companies. alter genex has had some success. it went public just the last genuine out as a mark of capital of 2.3 windows which 10 years ago was unheard of but has
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become more common as companies have gotten more sophisticated at how to develop these drugs. emil will talk about some of ofe trials and tribulations he is not in dispute. we will also hear from linda treacy in, the vice president of scientific and medical affairs -- lynn matrisian -- and she's going to tell us at the patient level with some of the challenges have been at trying to work with companies and work with people doing clinical trials of your patients to take advantage of customized medicine, personalized medicine, precision medicine to develop new treatment. and so with the introduction i think we'll just go ahead and start, and maybe get the ball rolling. i just wanted to go through and ask each of you based on your expertise, maybe we start with olympic maybe can tell us about you got a lot of experience in pancreatic world. comes from the patient's point of view what other challenges. how do you think about how
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pancreatic cancer varies between patients and one of the challenges of getting the fda and drug companies to respond to the unique characteristics of each patient's because well, thank you very much. yes, i will tell you about that for a story. so marjorie called our call center, the patient central at the pantry at it cancer action network a few months ago because her husband was diagnosed with pancreatic cancer. and our associates at the call center gave her lots of information about the disease and nutritional information, that type of thing. then they offered her and charlie the opportunity to get his tumor molecularly tested, to do a biopsy and children something about those tasks that would tell us something about
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what was causing his particular cancer and, therefore, how we might treat it. welcome that made marjorie pretty happy because she read the statistics and she knew that only 76% of people, that 76% of people were not going to be a light in one year, and only 7% were going to be alive after five years, so she said well, this is something i can try because their current standard of care are pretty crummy. so they agreed to do this. about a month later they get back a report, and the report has communications, alterations in genes that are underlying charlie's cancer. he's in a lucky, about 55% that we found a pancreatic cancer patients have something that's very interesting based on this molecular profile.
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and so now there's some options that this physician never would've thought of before. some of those are off-label treatments, so there are drugs so there are drugs that have a kelo and other cancers that are for a mutation that charlie has in his cancer. and some of them are clinical trials. and now here's where they get faced with not really medical problems but system problems, and problems with how does charlie get access to these drugs, and how does he decide what's working for him? so there are no real clinical trials in pantry had cancer for any of these drugs. there's 50,000 patients a year, of which 4.4.5% of them go on to clinical trials, and then this mutation is only in two or 3% of
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those 50,000 patients and two or 3% of that, 4.5 is that they're going to clinical trials. so how do we learn about what to treat charlie withthem even though we have this molecular information? and then what happens is that even if he can get on a clinical trial or get off-label treatment, and there's all sorts of insurance issues and being out of network that those kinds of challenges that he's going to face, but even if he can, what usually happens in these clinical trials is that he's treated with a drug and then three or four months later, they take a radiological scan and they say, is this tumor growth or hasn't shrunk. but the overall in point is whether charlie lives or dies, and how long he lives. so in the meantime they are checking what's called a biomarker in his blood, called ca 199 and if it goes up that
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means the tumor is probably growing although we are not 100% sure that's always true but there's good data that says that is often the case. and if it goes down that's a good sign. but again there's no, you can't act on that information. there hasn't been the proper studies to know whether that drug is doing charlie any good or not. and if it's not he could go on another treatment. so i think that's two examples wbut we don't have the information we need to determine what treatments we should get charlie, and we don't, we're not able to act fast enough on a biomarker to determine whether we should change the treatment or not. editing those are both opportunities for the fda to pay attention to a disease which is very life-threatening. charlie does not have a lot of time. survival is measured in months and weeks in this disease.
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so we need to act faster. we need to get, to change the system so that people like charlie can benefit from the science that we have around cancer treatments of these days. >> great. let me properly introduced peter. peace official tiger is senior fellow but i like to call them the resident genius of the manhattan institute. he has a ph.d in mechanical engineer from mit and a law degree from harvard and writes about energy policy, legal policy, tech policy and, of course, is the author of the cure and the code, how 20th century law is undermined 21st century medicine. so he is i disagree equipped to talk about this topic for today. how do we take that everyday patient example and translating -- translate that into better policy speak was the first thing to understand what example lynn just walk through, there's going to be a test or device to check for these oscillations in this patient's bloodstream and what it's telling us about the
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efficacy of the director doesn't get to market without the fda say so. i've had a number of oncologists tell me if they were not about to prescribe off-label which they do come oncology which pretty much shut down. i think the drug has been approved for something but not for what you want to use of corporate labels as you can use it for kidney cancer but not bladder cancer or this event. i don't know what the off-label drug was in your case but once again the fda and its drug approval process at the very least is that capturing every possible use of every drug. your life depends on people who do not believe that. hope for a lot of off-label work because it's happening all over the place. cancer can look completely different in different parts of the body and they can have the same underlying mechanisms, repurposing the drug and the, prescribing drugs based on their molecular effects and the ideology, the molecular etiology of the cancer is widespread in this area.
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it's been used in other areas as well but visibly see the most of it. if a drug company walks around and says do this for your panic or to cancer, if the owner of the drug tells you, they will probably get prosecuted, probablprobably criminally. people so they face a civil suit, and there will be false claims action. these are billion dollar issues for drug companies. it's a pity because they know on an awful lot about these. they are compiling this data but it doesn't fit. the fda hasn't gone after them although they very well may. they went after 23 enemy when it after 23 enemy when it became not just getting a genetic profile that sang this gene probably means that the fda says who? you know, 23 later by the fbi hasn't certified it to be true so, therefore, it isn't true. i had to shut down have other business in the states. the fda has in short plenary
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power over how much of his biomarker information you used in the drug in the device approval process. you have to persuade them. this is especially painful because the best people at the fda, and i would start with doctor janice woodcock is ahead for the the center of the drug develop an and evaluation research, know exactly what's right. janet has give an excellent presentation 10 years ago. you can still read your powerpoint on the web relating to them. to coder verbatim she said i'll markers are the foundation of evidence-based medicine. they determine what happens, what we should prescribe it we should prescribe to, outcomes happen to people, not populations. there is an indictment of what the fda has been doing for 50 years because fda clinical trials our population-based trials, overwhelmed with based just looking for empirical correlations. if you've got pancreatic cancer, let's throw this drug at you, if you do better, good.
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but, of course, is what you define as a disease is 25 different diseases, you fail to cure. there is a core problem with the fda existing trial protocols which statisticians call the reference class problem. if each of you represents 1000 patients, the simple question is, let's to pancreatic cancer, that's her favorite cancer today. this row here, if around you all of which are pancreatic cancer, 15,000 or so and test the drug in all of you, who is to say your predictive of the rest of the people in this room? if pancreatic cancer is an absolutely identical disease, that's a good sample. we know today modern genomics issue and there's enormous amounts of variations in genes and, therefore, the proteins that they code for. the saddest part of this to my mind is the fda issued its first
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guidance as they called for far more genomic data in late 2003 public or possibly early 2004, the nih is one of the agencies in washington, our major health agency that does a lot of genomic research. found comments on this draft guidance and said nice work, friends, down the street. that please tell us what you mean by validating a biomarker. could you give us standards? you are funding all this research. and by the way, we are doing all this research and we have set up internal standard for what will accept is good biomarker fund. this is from nih. they filed as with the fda. as far as i can tell it sank without a trace. by your letter the fda did issue its critical path report saying that biomarkers of something we've really got to do and to set up an institution called, what is it -- what is it called? the critical path institute
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which got some public and private funding for researching this. good people saying th the right things can never have enough funds. meanwhile, we have an enormous amount of taxpayer and health care money going into sequencing to what the outcome doctors are treating patients like george we're getting in sync with the. eric ward critical data which means for developing huge databases they say we see this profile associate with these affect the you can do basically epidemiology for biomarkers. this is how isolated the links between micro and a diseases. now you can begin working out molecules the molecules are way more complex. there's not one to one correlation. they work in very complicated networks. we have fantastic computing power today, and no less intellect. google knows this, that's where they getting into the business and apple knows this and that's what they're getting into this
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business. hasn't reached washington yet. they have incredible computing to give them an affidavit and they can unravel pathways. last one because it's just too good not to see. why would agencies be some interesting counterparts the issued a big mechanism noticed a couple of years ago saying if we do get overly complex data set, it's an ingenious technology for working out also mechanisms within the data set and by the way, signaling systems. why is darfur of doing this? is cancer the main killer of our army trucks know it is not that the nsa has huge databases. you want to have good causal mechanism analysis. if i blow up this person, we will stop having these terrorist. i'm quite sure the nsa would not want to share its database with everyone in the country so they said those are for cancer and we have terrific news for oncology
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at a have got it exactly right. the fda has lead the analysis we would be in good shape. we would because these people know. is what it's all about speed i think about a political platform for the next presidential candidates. with the nsa in charge of the drug development. >> just validating. target acquisition and they're very good at it. >> emil, let me ask you get what i take away from both of these discussions, lynn's point, very interesting, there are these patients like two to 3%, these small subpopulations within a larger population of people with cancer who may have unique genetic signature that that's a tiny number of patients. it's hard to show statistically in a tiny number of patients that your drug is having an effect, that it's not just noise, a placebo effect. so how does the drug company ceo
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or biotech company ceo -- evidence that you found a correlation between your drugs ability to target this particular genetic subpopulations? and then do peterson point, then how do you take this big data idea that is revolutionizing the rest of the economy, and how to convince the fbi -- the fda to put that into own thought process speak with window we don't but we would like to. the challenge i think imprecision medicine is the challenge we deal with in rare diseases every day. we are always dealing with small subsets and we've been struggling with this for a long time. these biomarkers are one of the most important ways to gain power in understanding what's going on in a very precise and accurate way which allow you to determine what you were trying to do in a very small population. the challenge has been, and our
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thousands of rare diseases, many of them which you wouldn't know how to spell them or how to save them, you never heard of them but 10% of u.s. population is affected by a rare disease and all of you probably know someone with a rare disease, and only 5% have treatments. the truth is that we should be doing more translate of science we do have and in able to get the power to determine what's going on in these patients with biomarkers is one of the critical things we believe. i am the president of the foundation which is focused on where to these issues, and would focus on how to accelerate biomarker and we believe the biomarkerbiomarkersbiomarkers fe that will help where disease well as patients with pancreatic cancer and stories that peter has been talking about. so i can tell you this though. the facts are that since the beginning we saw approval which started in 1992 after the hiv/aids crisis and when it was first promulgated in that time
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is only then really in a rare disease, genetic disease every only to disease where a new biomarker has occurred has been used in those whatever 23 years. i was involved in one of them, and the other one was -- the only two and all the time. there have been a few other biomarkers used but all of them have been ones that were approved in the early '90s. so there hasn't been anything you. the question is with all the genetic information we have and all the rare diseases out there and all the signs that has been done, that we know and understand what's going on, how can it be that there are no biomarkers-based approval for more diseases that could require them? those options have happened because i'm working on a program for disease called ps seven and -- type seven. that disease is a slight
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disease. there's only 20 patients in the united states, perhaps, with the disease. but yet we have the treatment. the treatment has been around for 20 years no one and works beautifully, not translated because we can't detect, t did a study with 10 patients improved that it is working. if you use a biomarker come how much better they have in their body, we could do it immediately. we've gotten the dma to agree to that. the fda has not agreed, but with a biomarker we can run a study with 12 patients and get the study to work. based on all the science we have show that that's a reasonable treatment and get these kids treatment. what is the point of all the nah research women funding from the billions of dollars we been spending i it when we find punditry but we don't turn into product for someone to get? that's been the tragedy and i've made it my mission to get through the process to show why it should be done and make the process better.
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.. it's great to travel these tests that may correlate to better clinical outcomes, longer lives, better performance on
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disease than they would have otherwise, but we don't necessarily know that. we don't necessarily have conclusive proof that the biomarker is correlated to better disease outcomes and longer lives in typical situations. so, pushback on that and tell me why the fcc shouldn't be cautious and conservative and say yes this biomarker sounds like it is a good thing to test but there is no definitive evidence that means a patient who does better on the test is actually going to have a longer life or do better on that disease. >> one dimension of the problem is the industry would say don't make it use, tell us what the standard is validating that the marker or context of use and we will go out and we will develop
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the evidence and fund the studies that do that. in the biomarker space the agencies concerned is something like this. the nih funded research or runs a study and discovers the marker and publishes an article in science or technology and declares victory and moves on. if you look at that and say that's not a biomarker, it hasn't been reproduced. how do we know that is good science, what is the control that would prove that you have had something? that isn't exactly what we are talking about here but there's a really interesting study done a year or two ago. what they did is they took an expression profile and looked at
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classes of drugs already approved by the fda and then compared them across two different types and they found a match between the genes that were hit by a 50-year-old class of antidepressants, some of the first antidepressants that came online in a class of long cancer, about 20% of cancers and it's also a class called the endocrine tumor. they say wow this is interesting. the pathway of the drugs inhibit. these are the same ones implicated for this class particularly in the small so long cancer -- lung cancer. they looked at models where they grew the true -- the two tumors.
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so now those drugs are in testing for small cell lung cancer for a efficacy. they haven't been used very much but it's a more recent class of drugs we have lots of data so physicians can look back and see how many patients who had been taking lithium or some other class where the rates are slower than they have been for lung cancer or others do they have a better response rate so there is a range of data that can be used to validate that expression profile data and observational data so that there is a biomarker that can be used in a clinical trial for a particular
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class or if you can get down to the level of knowing in this case but it is exactly causing those cells to self-destruct then we can use that as an endpoint so i think what we are asking them to do is paying off that of the develop and which they have said they are not equipped to do or they were reluctant to do for the people that were capable of doing it which is the research community. >> would that be like a particular professional society or would they deputized certain academics? how would that process go? the people there or or be spending billions a year and doing it pretty well they characterize them as genomic variations that have medical impacts. they do not just say stand in and they and i might add in contrast to one database that i know they actually want where they invite people from all over the country to send in the ports
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of adverse effects of drugs. it is so poorly monitored by this what is up this up into the reporter that tested it says that the vaccine transformed him into the incredible hulk. so that's the one you should be worrying about. apparently he got a call saying well you please withdraw this. [laughter] first of all, people talked about drugs as a biomarker. they are drugs changing the level of sob biomarker. if you were going to do a molecular weight to understand the complete molecular pathway which will often be a series of interactions. alzheimer's is probably hundreds of proteins in a chain one or another. it can be any number of them and each will have the same effect because it is a chain effect and the link is what causes the disease.
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but you have to understand these mechanisms. that's why they deserve a nobel or something like that but they come out and win the competition and deserve that. you're talking about mechanisms of action. you can track those down in much larger data sets. you don't have to use the of statistics at all. you can simply wake up this is leading to that and this is leading to that area i would also add a single tumor or the more common effect can find 10,000 mutations into the cancers go crazy in every possible direction. they do not think they just do it wildly. the analyses are sophisticated modern methods. these tumors have built in the network and analysis. analysis. i have some of them in my book. they look like a huge spider web of links between them and you can quantify a doubling as and they are hierarchical networks
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that are clearly important in almost all the variations and there are others that are playing alongside. they've mapped the mouth systematically. one of the most studied as breast cancer. for a long time they broke the disease up into four major categories depending on four major receptors or combinations of the receptors. the databases grew the modern statistics -- if you are not into this stuff whether it is your phone and computer and everything else it does it very well and we know this because the drugs were developed towards the staircase into the work area they are rare diseases. a number of them are actually quite simple from the molecular perspective. you get these strong correlations with it the diseases associated often 1-1. sometimes you have to double enough to get an effect.
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once you have it narrowed down you can do good studies on the mechanisms. there are good studies on whether a drug will go in and reverse it or block it. if it is a long chain reaction it's more difficult but we can know how to deal with complexity >> i think to take away from your paper that i thought were interesting word first of all the fda is restricted in its ability to talk to drug companies because of conflict of interest rules that prevent them from talking but even the national institute of health which is a major government agency that funds academic biomedical researchers. even the nih doesn't have a lot of cross channel communication with the fda about some of these advances and maybe as you said they could work with the nih and use them to help develop some of the standards. the second thing that you talked
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about in the paper that was very interesting was the fact that the european medicines agency for the european equivalent of the fda actually has been more forward thinking on some of these issues relative to the u.s. fda and i thought maybe you could touch on that a little bit and then there's a reason you have this market because they have been more forward thinking perhaps on some of these issues then the fda has been so maybe you can touch on that as well. first, paul. >> is there any other group that you can put at the table just to look at one of them and say there's the foundation and there's the biomarkers consortium which is a private not-for-profit foundation that can accept funding and actually works with academic researchers and drug companies and they have an interesting partnership we talked about in a paper copy of the paper copy of defensive
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medicine partnership looking at four or five of these. at least a are alzheimer's to do what we are talking about, go through the science can't validate the markers and develop the evidence. so that is a big step forward and then i think they have an initiative called the innovative medicine initiative which is also they can use a biomarker validation and one of its key projects spending hundreds of millions of dollars compared to a few tens of millions of dollars a year and that could be a breakthrough but the key is getting this outside expertise the agency is effectively walled off from in setting these critical standards so that people in the academic medical community and the drug companies would sit at the table and say this is all the evidence we have. this is how we can validate it and we should go about validating so that we can check things off the list and move science forward. >> i think what i would like to
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say first of all about eugenics market value isn't the eugenics market value isn't all based on market values and i don't own it all to be clear. we do have one program that is looking to use the biomarker approval but i think in general what we can say about the ema and the agency the last few years, they've been more scientifically based in a little bit less regulatory based so they are willing to look at what you've got and make an intelligent decision about it. the case makes it look like we've shown them the data and we had an organized plan on how we present the data and it's based on a vacation criteria that we just published. we created a very thorough and thoughtful set of criteria on how to mollify a biomarker and we presented the data in a format to them and we had enough animal mechanistic data pieces put together to explain that it
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would be impossible that the marker isn't correlated with disease activity and wouldn't predict benefit to them but it's also all of the drug approvals that make sense to us with the bases and what they told the basis in the meeting was this is the only way you can do this study. that's what they said the only way you can do the studying this disease is what the urinary measurement. there is no other way to do it and so their view is this is the only way forward and we will accept the plan because we think that it's reasonable to do given the rarity of this disease and i think that's the basic fact is that at some point in their the rules into the regulation of the policy that we have created for the country we have to make it practical or realistic otherwise things won't get done and to say i want a clinical endpoint addressing and i went walking tests and this test and got dressed a standard you need 100 patients or something to do the study and there's only 20 in the united states you are basically
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saying i figured out a way that we are never going to apply a science these patients will never be treated if we want them all to die rather than risk getting treated with a disease approved with a biomarker endpoints and that's fundamentally is no one else that would agree with that sort of the ultimate practical outcome of the decision. that's why we need to change. there are thoughtful ways to do to do this if you propose to them with academic and foundation people developed criteria written. it's not trivial, it's not quick and dirty, it is thorough and comprehensive and the key and the key thing is it is an acquired things possible. it will require you to know the outcome in the disease that has less than 100 patients ten years from now. look what the law says. they had a piece on the approval
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to qualify the biomarker is using the pharmacological criteria where other types of data in other words clinical outcome data was impossible or impractical to collect and so it really struck that you need to be practical in your choices we are using the science best we can and not creating unrealistic barriers that will cause the disease month to get treated. so i think that is one of the differences that you may see in the scientific side and i don't think that they are looking at a press event and concerns and the concerns come from the fact that a couple of drugs and a heart arrhythmia area didn't play out and people were hurt by that and those big fancy cases of drugs and a big market. it didn't play out are causing
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them to move away from diseases where we know a lot more of what's going on in the very specific biomarker that is clear to that condition and it's just not the same situation as the others failed in the past. they need to get over that story. i love bob temple and he's a great guy .-full-stop telling that story and let's get on with the new science and where we are going now which is much more soundly based and mechanistically sound. >> when i introduced him earlier i neglected to mention you were president of the national association of cancer research which is the most important academic professional society and she also worked at the institute of the national institute as a phd in molecular biology from arizona. so i give you the floor now. please address all these points you've wanted to mention.
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>> there's lots of ways i could do that but what occurs to me that we are really saying is that the fda looks like one of those human resource departments to you roll your eyes at that says one size fits all. you've got to do it, these are the standards, everybody has to fit into these groups and you know how for those of you that have been in that position where you just say, on we have a mission here and there is something we need to accomplish and there needs to be some flexibility around this. so i think for historical purposes, they have this one-size-fits-all, not a real big government agency and in order to gain some efficiencies, everything has to fit in their individual little site was and i think that is just what has to change, this idea that not all diseases are the same and that yes they have a very important
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role in protecting us as a society and protecting us from drugs that are out there that thousands and thousands of people take our politically healthy, blood pressure control medicine, those types of things. so it's a very different story than rare diseases or deadly cancers. and it's that ability to be flexible enough and yet stick to the principles of some sort of scientific validation and overlaying exactly as you are saying the principality of it and what's required. in a population we are addressing what is the risk tolerance? i can tell you pancreatic cancer patients or in general much more willing to take risks with their treatment because what they are facing is virtually certain that otherwise. so it's overlaying all of that into the decision-making that
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goes into what's approved and what is the level of validation we need for those biomarkers that seems to be the mindset that has to change within the agency. >> how good is the science? there was a paper that was published yesterday in fact arguing or suggesting or indicating that sometimes when we do these genomic profiles say a tumor in a particular patient that we are not actually getting good data because if you don't benchmark that to the baseline genetic profile maybe you're not actually getting accurate information about how the tumor is distinctive and so there is a lot of commentary out there that okay yes it all sounds great in theory to do all this general network that at the very basic analytical level are we at the right level, are we mature enough with those tools that we can actually say okay we are measuring accurately the genetic profile of the patient and being able to make good decisions on the basis.
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>> it comes down to don't throw the baby out with the bathwater and it comes down to the research component and the practical patient treatment component. so, right now yes it's not 100% certain that a mutation means you will respond to the receptor and in a disease like pancreatic cancer. i have no evidence of that divides the network and other cancers. this patient has very little option. i see no reason to say i ought to check their genomic dna to make sure that this is right. what is -- it's standing in the way of the good and moving forward. now in a research setting, yes we should be doing that. we should be sequencing both the tumor as well as the normal dna
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understanding what is specific for the tumor versus what is a normal background for that person. that is all absolutely true in the research setting and that will eventually end form the treatment setting. give us a little bit of time to move things from the research lab into the clinic but for right now the thing that would scare me about that kind of statement is people would throw up their hands and say we shouldn't be doing any of this because it's not good enough. but it's a whole lot better than shutting your eyes and treating the patient exactly like you treat every other pancreatic cancer patient and you know you're only giving them a couple months to live. so it's a whole lot better than that. >> just to follow on that once you have clear evidence he or he standards of the industry which traditionally has gone through the new drug approval they have a biomarker in the product because they see okay i know how
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the process works, we know what to expect. but if there is clear evidence he or he standards and timelines for the qualification of the biomarkers suddenly the industry will say we can reduce the cost of the development and the time it takes to bring the new products to market and we are going to invest in bringing the academic research and other tools of the at a level that is needed to meet at so i think that is another half of it is when there is a clear pathway as there is for the accelerated approval for cancer and hiv and some other drugs that basically 90% of the approvals which are used as surrogate endpoints are three classes of drugs for cancer and hiv and anthrax so we need to move beyond that and part of that is setting up a pathway that drives the investment that will raise the level of science science the way the wind is suggesting. >> regulatory clarity in other words. we are going to go to questions and i know there will be a microphone circulating around
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back there. when you get the microphone, make sure you get your name and affiliation because this is going to be on c-span i believe. while we are getting the microphone moved around for whoever raises their hand, let me ask one question before. so there's something going on in congress right now called the 21st century cure century cure initiative through the house energy and commerce committee led by the chairman fred upton and they are trying to develop some of these ideas into a concrete congressional bill that would push the fda in the right direction. i would open up to the floor have you all been following the process and how do you feel about going and are they doing the right things you want them wants them to do and are they currently thinking about it? >> my foundation is working on part of it which is the opening act which is an exclusivity provision that allows companies of the big markets to repurpose them for diseases and gives an incentive for more of the science we've already created.
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the bill is extremely long, 400 pages and a lot of sections. it is a very -- it's effort and i think what fred upton was trying to do was get the best ideas he could, put them in and come up with something that was going to change the way things get done. on the house side or something else being put together that will be comparable but there's their things on patient input in the process. there's incentives and a number of other aspects that could improve and i think it's a good discussion to have because it gets different state court first outside to start talking about fixing and improving things as we need to. >> we wouldn't be having this conversation without the chairman and the congresswoman leadership. we focus on the biomarker piece
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of it and i think that is moving in a good direction because it is recognizing that you are right, the clear standards for the particular context are used for the biomarker would really incentivize bringing in those other experts to convene the groups making sure the patients are at the table and academic communities to develop standards to assure everybody if they do the science correctly the fda is going to recognize it. >> i might add it's quite stunning how bipartisan they've been with the equal number of democrats and republicans and i might also add the council advises the technology issues a report two years ago which gets this right as far as i can tell. there's an awful lot of people who seem to believe that science is about saving lives and medicine and its hard to -- some of us are more skeptical than others but this is what is politically wonderful.
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washington has an excellent agency that works really well on the project that we wrote about recently in an op-ed some weeks ago. this might be a little bit mean that they are doing a series approach prospective studies on the clinical cancer drugs have failed on the fda standards and and their going back and re- analyzing the tissues involved. this is just truly wonderful. >> the 21st century cure is a step in the right direction getting a lot of perspectives at the table. the double is in the details and it is owing to be an awful lot of working through the details as we go forward but it certainly is delightful to see the effort being put into that. >> are never your name and affiliation >> i am a lawyer here in new
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york city and i would like to ask the panel listening to the discussion i'd heard compliments about nih but i have to ask to what extent has the fda outlived its usefulness. i heard you say that they do some things that remain useful but are we better off limiting the jurisdiction and narrowing it on a macro level and permitting the market to take this on and move with it? >> to me i would assume that is a step backwards in terms of safety. i think we'd rely on somebody we've rely on somebody to determine the things that we can buy and get prescribed for us that we understand the safety parameters anyways. in my mind it is a matter of tiling in the flexibility that
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we need to be able to look at specific situations as opposed to the ones for all. >> speaking as a wall street investors i can tell you the biotech investors i know pretty much every single one they would tell you that getting rid of the fda would be a disaster because the fact is companies sometimes by. sometimes they do studies pretty don't always disclose what went wrong with the patient or that may maybe the baseline characters were different and save the new england journal of medicine doesn't have the regulatory authority to audit this patient records. they have to rely on the honesty of the people that submit those studies to the new england journal of medicine or the fda goes through with a fine tooth comb and maybe they go too far. if you are able to do things where they analyze the clinical trial data. for me personally a lot of the people in the investment would say there's an appropriate role for the fda and it will
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modernize and that's why we are convening this meeting. >> i think the update is absolutely necessary. there are some areas they need to help recruit more people and connect to the academic world as they were when they had more people at sieber to help them be more creative and connected to the science and i think it is easier to help them get their. i do think they need more funding. they need to figure out better how to organize and recruit the kind of people that can keep connected with what is going on and drive the science back to the review level. the initiatives on the programs where they hire people need to give money to the nuts and bolts of the qualities that people involved in the appropriate standards for things like biomarkers and other things.
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it definitely wouldn't work well without them. >> i don't think there is a major drug company that would say [inaudible] they are well aware on whether or not the fda is getting it right and the government review is important to them. i would add the driving the lecture of medicine is progressively moving power away from the fda. and i don't know if president obama was aware of this when endorsing medicine and the state of the union address, but in the off label world, the doctors are going it alone now. they said it's okay to prescribed these cancer drugs, but the problem is getting them to the market. ..
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that's where the fda wants to approve the drug and loses control of the story, which is why they become more conservative in approving drugs because they worry about a know once the cat is out of the barn they don't have that complete control anymore, so-- >> they have more than you suggest.
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as many of you know, that was a drug that increased birth defects. but, they put on a bunch of conditions and certainly bit of power that doctors can have. they look to regulate 23 and me. ibm watson is doing that for hospitals in new york and computers are elicited to the diagnosed is. there doing it with more data more specific recommendations. technically the federal drug law is written so broadly that every computer, every docket-- database is technically a medical device that is telling doctors and patients to treat it with that.
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they have not dare go after any of them and i hope they hold their fire and if they do i think they will lose on first amendment grounds. >> lets get to another question. >> i am a recently retired oncologist here now sinai in new york and i want to quote what paul howard said, i could not have practiced for 40 years if i knew with fda approved and didn't and this goes back to the 1969, when we had a four drug, nation approved to cure hodgkin's disease and cured 60% of the people. the drug we use we get a six or nine month remission and the fpa never approved, nation of drugs, but we went ahead and did it and we we still laugh about it some of the older guys years later and you very well discuss the mutation and various diseases that cross over to diseases, but the key issue i think has to be
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addressed is that when we went down to the american society of clinical oncology to lobby congress ted kennedy told us there was a senator that was against us and he said get some patients and sit them in front of his office or in front of his lawn and they cannot argue with cancer patients. when you come down here they think you armada-- months of guys that want to make money for yourselves, so my question is i am so glad to see that doctor monty is here. what more can we do to get patients involved because it's difficult dude do it especially with pancreatic patients, but for people who let gotten involved, the aids community in breast cancer patients have seen funding and people listen to them and we need a patient approach because the congress of people think one way and what's sad to see john dingell sitting next to president obama. he was one of the people who was most against this. he put bernie fisher, the hero of breast cancer under tremendous scrutiny and now
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sitting next to the president when he approved to something for healthcare, so we a patients involved and please tell me what you are doing. >> almost everyone can be a patient at some point in their life and as these new technologies like 23 and me i think only does a couple hundred genes, but as the cost of the genome test plummets i think it's about a thousand dollars now, but the prices plummeted to the point where it will get into the clinic and at some point they will prick newborns for it when they are born and take a drop of blood and looking at it and saying we think we see a predisposition for heart disease or diabetes or: cancer and that level at which everyone subtly becomes a patient advocate or potential patient advocate i think is a game changer. >> added to the an important role in what you're talking about about, using the power of the people, power of the patient in order to influence change and i know more specific example, the recalcitrant cancer research
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act was passed into law of january 2013, as a result of the efforts that started out as the pancreatic cancer research act and we could get 600 patients, not usually patients, but family members of patience of a family's the had lost a someone to the disease dressed in purple on capital year-- hill you're after you're saying this is important to us in the end of result was asking that and ci to come up with a scientific framework for the deadliest cancers, which are eight of the major cancers with five-year survival rate of 50% or less and that is now something that and the ci is acting on in terms of looking at that from a more strategic viewpoint as to what could be done in the disease, so i think those are the types of things. they take a long time and you need a lot of organization in terms of making sure the message
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is consistent, but you know what you're asking for is what you really want. a lot of those things, but that is clearly one way to affect that kind of change. >> that every life foundation, we actually organize the patient in the community and have about 180 groups that are behind our campaign, like what we call the cure the process campaign. every year we bring about 200 patient advocates and we go through our legislative conference to tell them what's going on. we have lobbyists train them how to talk to congressmen and staffers and we have a company that sets up meetings with their particular congressman or saffer we send them to the hill input as an politico and make it a rare disease week and we have premiere movie and do a rare disease caucus had make a big event out of it and we do it every year now and they know we are coming and actually, congressman love the rare
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disease issue and feel like these are good win for them. we work both sides of the island they are very aware and frankly some the 21st century to her stuff is basically rare disease focused. so, we agree with you it is not just the company. definitely has to be the patients affected by these rare disease telling their stories on the hill and we do it in an organized way with enough firepower so that they feel the presence of those groups by having attacked the hill on one day every year in february. >> any other questions out there? yes. >> i teach at the university of pennsylvania. i'm wondering if one possible way through the whole ind thicket and it is a very slow fekete would be for that fda to have something like the recalcitrant disease program. just carveout specific diseases with a dismal prognoses, a lot
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of them would be cancer and created much freer, much looser regime for treatment protocols. i can see one obstacle to that is just this kind of conceits that existing treatments work and every thing has to be compared to existing treatments. i think that is probably pernicious for diseases like pancreatic cancer and lung cancer and the like, but it might create tensions to say, nothing really works, so we should just try anything. >> i mean, i think that is it exactly on the lines of trying to open it up so it isn't one size fits a and a sane and even if they have to lump some diseases into those that are particularly difficult that are really recalcitrant, there's nothing to do for them, that
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might be easier for them to do than to really think about it each disease individually. although, at some level that needs to come into play. >> next question. >> network for excellence in health innovation. can you comment or if you are doing any work or have any opinions about the payment side of this. so, certainly another reason why there has not been lot of activity in the biomarker of spacers and i think if you get something approved by the fda you go through another three to my five-year odyssey before you determine what you'll get paid. it tends to be very depressing for the number of projects that get finance. so, with the exception of a drug companies absolutely positive it will get its drug approved and has an absolutely essential requirement that the test go along the drug, have you looked
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at all at what the incentives are for product developers, with -- once they get to the fda? >> i think it's a major economic problem both for reasons slightly broader than you suggested. i know of only two ways to modernize-- monetize your brain's of discovery. if you come up with a surrogate endpoint for alzheimer's, a good one that the fda approves, it will be a billion-dollar discovery, at least. getting an alzheimer drug through conventional fda trials would be expensive. it's too slow, the disease and by the way, in response to an earlier question, people across united states oh a huge debt of gratitude with the gay community with their activism in that late '80s and 90s that created the accelerated approval, which is basically the primary regulatory matrix for the positive-- possibility of introducing biological and basically molecular level stuff, but the two tools modernizing
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these discoveries are the diagnostic devices. if you're going to have a targeted drug that will only be prescribed in these conditions you will have to have a diagnostic with it. the fda actually required companion licensing of the device. now, the problem with this it is if you are a good pioneer and you find a new target and better still you find a way to accelerate the approval and now you have got a lubricated pathway for rapid approval by the fda, is easy for all the followers of to steal your invention. they work out the mechanism of action of your drug, conduct a few studies and they write on it. the classic story on this are statins. a japanese researcher worked out the basic chemistry and discovered the first step. he didn't work out the chemistry at all, but his grandfather was interested in aromatics and they produced these medicines and figured maybe some microbes might have thought of a way to disrupt cholesterol because
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cholesterol is glue used through nature. so, he worked this out and meanwhile brown and goldstein in the united states were working out the molecular pathway got a nobel for that and the call at the most decorated molecule in history. these people did incredibly valuable work, the whole staten industry store-- sort of hinges on what they did. none of them profited at all. when british humvee came up with an early commercial staten, but it did not go far and so far there are about to drugs later with lipitor, which was a 40 billion-dollar your drug. we have no way to monetize this research in clip-- molecular chemistry. i have proposed to some. @they want everything shared for free with everyone, but this stuff is expensive and you have some incentive.
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>> think your peers talk about this, but some kind of effectively what would be a compulsory license to say whatever .5 revenue that any product approved using a validated biomarker would flow money back into diagnostics industry, back into the medical community and potentially also encourage countries to share data. they say we could pool our data and revenue and allows to get the biomarkers validated that much faster, so much the better. in. >> any other questions? we are done? okay. one more? no? okay, let's do one more. i am getting mixed signals from my governors over here. >> sorry to hold everyone up. i'm from the parkinson's disease foundation and i enjoyed your discussion about precision medicine and the focus on cancer. i represent neurological space, parkinson's and others like alzheimer's and i think it's important to recognize that one
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of the panelists said this is a rising tide that can raise all boats and we are focused on how the genomic era really helped people and we need recognize something as simple as mri scans or pet scans can really help these other neurological editions, which currently represent barriers for drug approval. there is a scan for parkinson's to indicate if someone has or does not have parkinson's, but people who are negative for the scan so had to be included in clinical trials even though they don't have parkinson's, the fda one allow it. how do we increase the sox know the that you talk about in order to get fda to say we recognize these people don't have parkinson's and we can exclude them from your trial and therefore, ensure that drugs which can help slightly will actually get approval to help live-- people live with the disease they had today? >> a big chunk of biomarker research, we didn't discuss much now, is actually located in the sort of siege and that starting point of disorder.
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is there a profile that tells us hopefully rather advance that yes, you are susceptible to this disease. there are quite a number of many of them rare diseases where that is clear and you can scan for huntington's and for another-- number of other things. from what i've read, we do call parkinson's neurological and a lot of those are thought to be very complex, molecular perspective because you complex change they are actually propelling signals, nerves signals and so on and multiple molecules in those change in variations in any number of them can lead to the same disruption that causes the same clinical symptoms. i did see-- and the national institute for medical health is recently tossed overboard all of the standard definitions of neurological diseases and said
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we're not setting that any more. we are not fighting that. and they actually use the analogy precision no logical medicine. we will isolate pathways in the brain that affect cognition and pain any motion and so on, but some finite number presumably and work on those as much as possible. a major use of biomarkers is what the fda endlessly calls trial enrichment. we should be testing and dropping someone who does not have the disease. i might add a second problem is there are quite a few people have resilience genes than having to be really good at not succumbing to a disease even if they are infected with microbes. there are certain people who get infected with hiv, but it never progresses in their body. is a small number, but they are the elite controllers and there are other markers like prostrate cancer and so on and you don't want to test drugs against people who are not six-- six. it can mess up your travels
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completely in the best i can give you on that one. to make great. i think we have covered a lot of ground here and i want to thank all of our panelists for being here. [applause]. >> on to thank you for being here and went to take the supporters of the manhattan institute for helping us invest in this very important and often neglected area of public policy, so thank you very much. [applause]. [inaudible conversations] [inaudible conversations] [inaudible conversations] [inaudible conversations] tran 11. [inaudible conversations] [inaudible conversations] [inaudible conversations]
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[inaudible conversations] [inaudible conversations] [inaudible conversations] [inaudible conversations] [inaudible conversations] [inaudible conversations] [inaudible conversations] >> coming up on c-span 2, google's chief economist talks with the twitter's former chief counsel about privacy and security on the internet. after that, scientists try to answer the question, can there be life on mars, a congressional committee looks into the nasa finding a possible water on mars. then, the head of the smithsonian institute talks
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about the future of the museum and research group. later, afghanistan's interior minister visit to the us and his comments on the future of the country's stability. during this holiday weekend we are showing you encore presentations of q&a, each night at 7:00 p.m. eastern on c-span 2. this evening weekly standard senior editor andrew ferguson talks about the potential republican contenders for the 2016 presidential election as well as president obama, cost overruns at the planned eisenhower memorial and the growing number of journalists and pundits in washington dc. each night at 8:00 p.m. eastern book tv primetime, tonight history books starting with the first ladies, presidential historians on the lives of 45 iconic american women from c-span's first ladies series. also, historian david mccall on his book the wright brothers. erik larson, author of dead
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weight, the last crossing of the lusitania. >> abigail fillmore the first first lady to work outside the home. teaching a private school, she successfully lobby congress for funds to create the first white house library. her hairstyle and love of pink created fashion sensations. it was marketed as a color and stores sold clip on banks to women is good to replicate her style. jacqueline kennedy was responsible for the creation of the white house asarco association and nancy reagan as a young actress saw her name mistakenly on the black list of suspected communist sympathizers and she appealed to screen actor guild had ronald reagan for help. she later became his wife. the stories and more are featured in c-span's book: first ladies. the life-support five iconic american women. the book makes a great gift for the holidays, giving readers into the look of personal lives of every first lady in american history. of stories a fascinating women
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and how their legacies resonate today. shall the stories of america's first ladies for the holidays. defense book, first lady is available as a hardcover or e-book from your favorite bookstore or online bookseller. be sure to order your copy today. >> google's chief economist and twitter's former chief counsel recently joined government regulators on a panel discussing connected consumer products, privacy and security. from the annual technology policy institutes aspen for them, this is an hour. >> think you. thank you. will come of this is the panel of privacy and big data, all very hot issues. a recent mckinsey report stated
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that internet has the potential to fundamentally shifted the way we interact with our surroundings and that cancer when extended to include the effects of big data. they had differing views as to whether this is a good thing or bad thing. nevertheless, virtually everything transportation, energy, medicine, healthcare, politics and government, content and virtually every business is being affected. we have a great panel to discuss these issues. i will introduce them alphabetically. shawn dubravac is the chief economist, senior editor of research for the consumer electronics association and the author recently of, digital destiny, how the new age of all transform, work and comedic a. to his left is alexander macgillivray, who has been the
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deputy chief technology officer at the white house for a little less than a year and his portfolio is the focus on internet policy and electrical property policy and the intersection of big data technology and privacy and before joining the government he served as general counsel and head of public policy at twitter. terrell mcsweeny is the commissioner of federal trade commission which he has been april 2014. prior to that she served in senior positions in antitrust division and deputy assistant to the president's domestic policy adviser to vice president biden. paul nagel, for the commerce committee. and how therein is the chief economist at google where he has been starting as a consultant in .


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