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tv   FDA Acting Commissioner Dr. Sharpless on Agency Mission Importance of...  CSPAN  October 2, 2019 6:15pm-6:38pm EDT

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(applause) good afternoon and thank you for that warm introduction. it's great to be here and i want to thank our research america for the support for him to talk about issues that we face in the society today. but also for its long term leadership and i recognize the need for research and improved medicine and health and get familiar for a while now and one of those advocates for those governor castle that was described. i started as an academic researcher and a doctor and i
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took care of patients with blood cancer. and given that background i can't think of any topic more important than producing the best research to provide the best data that's needed to develop treatments for our patients. my experience in this regard occurred between the second third year of school might look at your off to work at the nih and i work on dementia and that experience helped me appreciate the analyzing of my own data and foster the love of science. within a few weeks of starting at the nih they my friends would call me would say, how i like it and i said this is the best you're my life. that's turned out to be true even now in hindsight. (laughs) it's also fostered an opportunity
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for doing good through science and medicine so not surprisingly, when i left a lab i went to school and i trained at the college and that practice and i think that i became frustrated by the limited options that we had. to be clear, taking the privilege is a fun thing to do and i enjoy the practice of medicine. but i also hated that it felt like the recurring nightmare of health and missions with cancer and doing the same treatment over again and watching it fail. it reminds me about the first generation of the vacuum that claimed you're floor and it backed up in your chair and bump into you chair again. he would do that over oregon and that was psychology in the 90s with a few exceptions. we had some scenarios where it worked what is very frustrating and
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drugs it didn't work and was very tough to be an oncologist back then and it was worse for the patients. that sent me back to the lab and i wanted to do better i want to see is to try to improve the forms of cancer so i left to clinical oncology where it decreased my practices so i could become a biologist. and a geneticist in earnest age a mixed emotion with this clinical fellow at harvard and would hardly see you and get your advice on cancer and all of a sudden you go into alive and you make it to use a buffers and a bounce around like a mexican jelly bean and you have to ask the student to how do you make it work and that was a terrible experience but i went through it because at the end of it i was a scientists and it was science
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that was wonderful for cancer patients and that's what it's really all about. i search for reliable data so people could find the answers for our patients that they need to have a cure or an effective treatment artist in some cases the hope for an effective treatment sometime on the horizon. i have to say we, live in a privileged extraordinary time of progress where there's been enormous transformational movement in medicine. i've been particularly lucky in this regard that the progress and cancer research as been particularly impressive and breathtaking. the battle days of the 1990s are rewarded in medical oncology and the pace of progress as really been dramatic and take a disease like malignant melanoma which was in 1995 that disease was about the words cancer imaginable and it was a diagnosis that we saw them to every and it was a grim patient
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that rapidly we came to that which provided no useful benefit for beijing's. then, after a lot of investigating about the biology in that cancer. it doesn't love and, we started to have some breakthroughs. i remember it was 2011 because my father died of melanoma in 2010, about a year before those drug started to work and it was a cruel irony that i studied it my entire career research. with a new biological response to cancer we had several affective fares and rapid succession and a five year remarkable period starting in five or more active drugs and melanoma and it was a disease that had 0% or a 3% long-term care rate and disease now which is 60 or 70% of patients are cured. majority with mist alex melanoma are cured and they get cured with
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sixties disease and go into habitat for humanity houses and that's the kind of progress we have seen i guess one of the most ghastly cancers are manageable. to be clear, it's still a long way to go with cancer as the meeting caught of this in country and there is too many children dying of cancer and i saw a lot when i was in the sea i. but nonetheless, one has to say that the process has the complexion of this disease as a really been remarkable and and it's clear to me that the additional progress of cancer we need more research and what i just said with cancer is true in all areas of medicine and i would argue that even more unity is known as far progress has not been as good as things in the things that begin with the letter a in alzheimer's and als aging and that's just one
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letter in the alphabet where there has been progress and we haven't seen for example with cancer. with brings you back to this session of leveraging dated to accelerate progress. it captures two of the most critical issues we face in the medical research community and the fda specifically. first, what types of data do we need? what it means, when we get those status and in a matter that's efficient and respectful and values patient privacy. second, how can we use these data and other technological advances to have medical products and treatment in how patients lives. in a time of limited resources and scientific challenges. we all make the most of those scientific investments and maximize the payoff of our efforts. really that rings especially true to me in science where it's crucial that not only do we aggravate the
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data and not use the cutting edge novel and the tools that analyze their data. nothing seems must frustrated that you get together and have the variety of what you want in there and there is too large and too cumbersome in too difficult to mine and the pleas what you see is trapped in the data set and using those techniques to solve those problems. by gathering that data and the tools that are developing, we could make scientific progress and find those answers and develop new products and ultimately help more people. few places have higher quality data in the food and drug administration. it's a sport and scientific integration in a meat unique role to help scientists developers to turn their vision into products that are reality for consumers. it helps us to meet our responsibilities to ensure those products are safe
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and effective further intended use. high quality data throughout the life cycle of to be approved but the follow that product that is used for surveillance to make sure the devices are continued as expected in the world views. the responsibilities must be able to integrate the available data for effective regulatory decision-making. the data itself, while useful is not transformative and turned into smart dated to aggregate the reusable. and cutting edge discoveries will real rolled products and solutions and patients lives. the fta, or working with researchers and a number of key areas. where establishing new linkages between accomplished data sets and we are harnessing the real world data and employee these novel approaches in the name of its innovation to provide better information for those
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who need medical choices. an example of this set is worth pointing out is the devices of logic how at the faa which is a private partnership of the national evaluation of the technology or nest. their sets of health systems to allow the devices in the real world views because of the surveillance and is my opinion a good idea even the diversity of products teams. for continuing to speak development by promoting the clinical trials. its platform trials and attractable trials and randomized controlled trials and things that the fda is providing guidance on how to consider such data in the decision-making. these can be more efficient and can be cruel and lower costs. they're trying to make full use of the expedited excel ready approval pathways that we've been able
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to traffic pack and review. these are congress mandated and are used to help speed development for therapies for medical need. it's not to say were abandoning they researches and fda trials. but were building on hierarchy and strengthening so that we can use these new forms of evidence as the methodology approved. of course, increasing the input of patients and they've done a good job of incorporating the patient towards our decision-making. or building disciplinary structures across the fta to support more efficient decision-making and the center of excellence. which has climate cancer activity in the incentives to allow more efficient cancer applications. that's more of the famous fda initiatives which was related to the art of universal
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intelligence and manufacturing people. the key technical solution and will use cloud will cross the agency we and is in quite a large way and using technology in the creative ways and they're using data sets in realtime. this is related to the topic of information informed infrastructure and storage uses for the challenges and i'm pleased to say that in the next few weeks, will be announcing a new fda plan for fda technology infrastructure that will allow us to use this more efficiently so we can use topics of data sets or across the agency. as you can see the, faa is committed to its core approaches to hammering data. but our one final point is to roll the fda plays and between
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leverage and the speedy developments of products while ensuring the integrity of data in the process. everyone here understands the fda has been pushed to approve newer and better and safer products and do so as quickly as possible. they key of course is that were asked to maintain legal standards and safety as medical products. this requires striking a balance. a balancing point is the health and well-being at the patient. unfortunately, explaining it to the public and the times be problematic. on one hand, it's rare to find anyone was seeking a new tribute and will complaint that the fda is doing this too fast. on the other hand, these faster approvals an expert-ites must involve a lower standards for approval products. i wanna state, i don't want to be anything further for the truth in this regard. we can have it both
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ways. both faster and nimble approvals but at the same time having exactly always farce six patients. it's the basic understanding of cancer with clinical research. so it's much more now today by the new treatments. take another example from cancer. why sorry practicing, randomized trials where a common thing and were a standard way of telling the drug abuse efficacious and they did not like this of randomized trials and we had to do it but we felt at the time there were no credible alternatives. over the years, we learned a great deal about biology and the face of cancer and he subset-ing of cancer. we developed a whole new paradigm and we came to understand, this is what they control and usually we can make a lot of progress in cancers
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without you can see bows and the guidance on this topic that we will see in cancer trials. there's a few places where it's reasonable that can support oncology. but it's rare for therapy to go through the panel. i would say we, found processors with other patients and no one who has pancreatic cancer want to be randomized protesting. it's also more efficient for pediatric cancers and don't have the patience so the use of these larger settings is hard and so these more elevated trials have these sorts of diseases two and as i have alluded there's alarmists progress but we had similar stories worth cancer and breast cancer and leukemia. the goal now is, to replicate that success to some degree over
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other areas that we've seen less there be groggeries and other diseases where the biology is still very complex and without for the result of understanding of these eases that will help greatly. which brings me back to i started which is the critical importance and the need for more biomedical research. and or critical to the agency's work. let me dwell hear from moment on what i mean by good as opposed to bad data. every day, there is research that offers great products and some of rapid desire controls and for taking a short cut and collecting good data and bad data and submitting is bad data to us for example. but they could be back for certain reasons. certain causes are
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that it's a sloppy, slip shot research it and it can be difficult to take this apart because it's in terms of intent. but in one sense it didn't do much to the agency and had a decision where we can make a decision on those ways. let me comment on the problem when, i was an editor of the investigation, we decided we wanted to add the primary data from the offers and i thought this was an unnecessary endeavor and we rapidly appreciated many offers were submitted and frankly fabricated and has agreed that really surprise and we had not expected. later, when i was at the national cancer institute i saw that people would create applications. it's probably not surprising that if someone will
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use bad data to get their paper published or their grant funding or their billion dollar medical product. we simply cannot tolerate the deception behind the fta. i don't want to imply that these are often but they're rare cases and we have no data to suggest in those cases should become more rare. we still see data then we have expected in the agency. i would argue, that they are rare, they are significant because even a few examples of the public confidence in the approval process. think about today's moderate or, liberal fta that everyone desires which requires that the applications be truthful. when that doesn't happen the whole social compact breaks down. if someone comes to the fta whether it's
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accurate and see an application that contains a false claim, it undermines the search for a cure and violate the public trust. but he races costs, exposes patients to needless therapies and give science a bad name and most importantly it hurts patients. at fda, we don't have the sources to check every aspect in this bit of research. we have a lot of checks on data equality but at some level, we have to trust the spotters. we have to be doesn't vigilant in every review. we will use the full range of authorities to address this in the criminal penalties. i would argue that new data is really -- we can't really enforce our way out of this. it's part of the good culture and good culture in science it's the product of vibrant and scientific community. it's a
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place that research america could continue their work to put a scientific culture and values data and integrity to protect patients. i know this is an area where we all care deeply and i look forward to working with this group in the future on this topic. thank you for the information to speak today. i look forward to hearing more. (applause)
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