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tv   Key Capitol Hill Hearings  CSPAN  September 1, 2015 10:00am-12:01pm EDT

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the power resting in one party. to counter obama. i'm sure that had the race factor in it. they voted in gridlock. thought republicans would do even more when they voted them into the senate. the other factor is race. working-class people who are democrats, republicans, black or white, should all be working together. it is a class war going on. class people are doing worse and the congress has so.s that made itself reagan opened up china -- not reagan, nixon. reagan was working on south
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america. jobs ended up moving down there. they stabilize those places in south america. host: i got you. we are out of time and that's it for the program today. wayher addition comes your at 7:00 a.m. tomorrow morning and we will see you then. host: caller: [captions copyright national cable satellite corp. 2015] [captioning performed by the national captioning institute, which is responsible for its caption content and accuracy. visit] >> here is a look at what's ahead today on c-span. a speech by agriculture secretary tom ville sec will
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speak at the center for american progress. you can watch the remarks live at 1:00 eastern here on c-span. we will show you a hearing later looking at higher education graduation rates posted by the committee.ation after that, we will open up our online to get her thoughts on the issue. the hearing starts at 3:00 p.m. eastern and her phone segment starts at 4:30 p.m. the russian military relations with china is the topic of a discussion at the center for strategic international studies and you can watch that at 2:00 p.m. eastern on c-span two. its congress returns from august recess, where the first items of business will be a resolution of disapproval on the obama administration plus nuclear agreement with iran. starting tonight, we will bring you key statements and hearings that took place after the deal was announced in mid-july. bywill include a speech president obama at american university. house and senate hearings with negotiators and statements for and against the agreement by
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senate leaders. 17gress has until september to pass a resolution. here is a preview. [applause] obama: more sanctions will not produce the results that critics want, we have to be honest -- congressional rejection of this deal leaves any u.s. administration that is absolutely committed to preventing iran from getting a nuclear weapon with one option. another war in the middle east. i say this not to be provocative. i am stating a fact. without this deal, iran will be tough ourion, however rhetoric might be, to steadily advance its capabilities. its breakout time which is already fairly small could shrink to near zero.
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doubt that really the same voices now raised against of this deal will be demanding that whoever is president bomb those nuclear facilities? someone who firmly believes that iran must not get a nuclear weapon and who has wrestled with this issue since the beginning of my presidency, i can tell you that alternatives to military action will have been exhausted once we reject a hard one diplomatic solution that the world almost unanimously supports. words.ot mince the choice we face is ultimately between diplomacy for some form maybe not tomorrow, maybe not three-month from now, but soon. of the was a portion
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iran nuclear agreement related events we will show you this week. our programming gets underway at 8:00 eastern right here on c-span. a signature feature of book tv is our all-day coverage of book fairs and festivals from across the country with top nonfiction authors. here is our schedule -- this weekend, we are lie from the 15th annual book festival capital.nations near the end of september, we are in new york for the brooklyn book festival celebrating its 10th year. in early october, the southern festival of books in nashville. the weekend after that, we are live from austin for the texas book festival and near the end of the month, we will cover 2 book vessels in the same weekend from our nation's heartland, the wisconsin book festival in medicine and on the east coast, the boston book festival. at the start of november, we will be in portland, oregon for word stock followed by the national book awards from new
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york city and a band of november, we are live for the 18th year in a row from florida for the miami book fair international. that is a few of the fairs and festivals this fall on c-span 2's book tv. president obama travel to alaska yesterday and he spoke about the state department sponsored global leadership in the arctic rim known as a glacier. it's part of a climate change anchorage, alaska, this is 35 minutes. >> ladies and gentlemen, to introduce the president of the united states, please welcome the secretary of state. [applause] secretary kerry: thank you very
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much everyone, governor walker, lieutenant governor and senator michalski and senator sullivan. we are so appreciative of all of you and to alaska for an absolutely spectacular welcome here. onhink it is fair to say behalf of all my colleagues who have been part of this day long ascussion that this has been tremendous reception in alaska but importantly, a very constructive and substantive day. i think every delegation here would agree we have covered in a --m us amount of territory an enormous amount of territory and reinforced that every nation that cares about the future of the arctic has responsibility to be a leader in taking action and in urging others to take bold action in order to deal with this challenge. it is immediate and it requires ambitious steps to curb the
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emission of greenhouse gases and deal with methane, coastal erosion, fisheries, a host of challenges that alaska particularly faces. there is no mystery, as we saw reinforced in very dramatic presentations by a number of scientists, no mystery at all about what failure to act would mean. we can already see it. we can already measure it. alaskans are living it every single day. we confirmed today that we cannot afford to wait until someone else moves to implement solutions to the challenges that confront us in the arctic. i am very pleased that through today's glacier meeting, we made progress in a host of areas in our communique will summarize that including addressing the issues of climate change and the impact of it, enhancing resilience and strengthening emergency response and improving
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air quality and promoting renewable energy and household innovations that will increase efficiency and community health at the same time. everyone in this room, those here at the circular table and those in the audience, are connected to the arctic in some way. so are all of the citizens we represent. is note of the region just the responsibility of the arctic. or the arctic states themselves. we agree today it is everyone's responsibility. it is with that in mind that i turned to the next speaker who understands all of what is at stake. that threat holds by changing arctic has long been a top priority for president oh mama. -- president barack obama. he has repeatedly defined climate change as one of the greatest challenges we face in this century.
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and the president has stated clearly what is happening in alaska is not just a preview of what will happen to the rest of us if we do not take action. it is our wake-up call. the alarm bells are ringing, to quote the president. since 2009, president obama has demonstrated he is committed to meeting this challenge before it is too late, not with words but with actions. that is why he put forward a national strategy for the arctic region that establishes a comprehensive and long-term vision for our arctic engagement. that's why he credits the arctic executive steering committee to prepare and enhance coordination of national efforts here. and that is why today thanks to the president's climate action, the united states is well on his way to meeting our international commitments to seriously cut greenhouse gas emissions by 2020 and beyond while bolstering our nation's resilience to ensure
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communities thrive and economies flourish. and that is why he has prioritized so many other things including not a small symbolic step of renaming of a famous mountain and we can say denali never looked better than it does today. [applause] secretary kerry: that is why the president has prioritized working with so many partners because he knows all of us together have to do so much more to meet this threat and have to do it now and it will not be done without our concerted, global commitment. ladies and gentlemen, the president of the united states, barack obama. [applause]
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[applause] president obama: thank you so much. please. it is wonderful to be here in the great state of alaska. [cheers and applause] president obama: i want to thank secretary kerry and members of my administration for your work here today. and thank you to the many alaskans, alaska natives, another indigenous people to the article traveled a long way to -- the arctic who have traveled a long way to share your insights and experiences. and to the foreign ministers and delegations who have come here from around the world, welcome to the united states and thank you for attending this glacier conference. the actual name of the conference is much longer.
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it is a mouthful but at the acronym works. because it underscores the incredible changes taking place here in the arctic that is impacting not only the nations that surround the arctic but have an impact for the entire world as well. i want to saying the people of -- thank the people of alaska for hosting this conference and i love afford to visiting more of alaska over the next few days. the united states is an arctic nation. even if this is not an official gathering of the atlantic council, the united states is -- arctic council, the united states is proud to cheer it for -- chair the next two years. and to all the foreign dignitaries who were here, i want to be very clear, we are eager to work with your nations on the unique opportunities the arctic present at the unique challenges that it faces. we are not going to any of us
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solve these challenges by ourselves. we can only solve them together. of course we are here today to discuss the challenge that will defy the contours of this century more dramatically than any other. and that is the growing threat of a changing climate. our understanding of climate change advances each day. human activity is disrupting the climate. in many ways, faster than we previously thought. the science is stark. it is sharpening. it proves this was distant threat is very much in the present. the arctic is the leading edge of climate change. our leading indicator of what the entire planet faces. arctic temperatures are rising
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about twice as fast as the global average. over the past 60 years, alaska has warned about twice as fast as the rest of the united states. last year was alaska's hottest year on record. just as it was for the rest of the world. the impacts here are very real. destabilizing the earth on which 100,000 alaskans live threatening homes, damaging transportation and imagery infrastructure which can cause billions of dollars to fix. warmer oceans and rivers and migration of entire species threaten the livelihood of indigenous peoples and local economies dependent on fishing and tourism. reducing sea levels. villages unprotected from floods and surges. some are in imminent danger and
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some will have to relocate entirely. in fact, alaska has some of the swiftest shoreline erosion's in the world. one alaska native told me many of our villages are ready to slide off into the waters of alaska. in some cases, there will be absolutely no hope. we would need to move many villages. alaska's fire season is now more than a month longer than it was in the 1950's. at one point, more than 300 wildfires were burning at once. southeast of here, pacific northwest, even the rain forest is on fire. more than 5 million acres in alaska have been scorched by fire this year. an area about the size of massachusetts.
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if you add the fires across canada and siberia, we are talking 300 million acres. an area about the size of new york. this is a threat to many communities but also an immediate threat to the men and women who put their lives on the line to protect us. three firefighters lost their lives fighting a fire in washington state. another has been a critical condition. we are thankful to each and every firefighter for their heroism including the canadian firefighters. who have helped fight the fires in this state. the point is climate change is no longer some far off problem. it is happening here and now. climate change is already disrupting our agriculture and ecosystems. our water and food supplies.
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our energy, our infrastructure, human health, human safety. now, today, climate change is a trend that affects all trends. economic trends, security trends, everything will be impacted. it becomes more dramatic with each passing year. already it is changing the way alaskans live. and considering the arctic's unique role in affecting the global climate, it will accelerate changes to the way we all live. since 1979, the arctic has decreased by more than 40%. a decrease which is dramatically accelerated over the past two decades. one new study as a method that
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-- estimates that the glaciers lose all 75% gigatons, 75 billion tons of ice each year. to put that in perspective, one scientist described a gigaton as a block the size of the national mall in washington. from congress all the way to the lincoln memorial, four times as tall as the washington monument. imagine. that's what alaska's glaciers alone lose each year. 75 of them. the pace of melting is only getting faster. it is now twice what it was between 1950 and 2000. twice as fast as it was just a little over a decade ago. it is one of the reasons why sea levels rose about eight inches over the last century and why they are projected to rise
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another 1-4 feet this century. consider as well many of the fires burning today are actually burning through the permafrost in the arctic. this permafrost stores massive amounts of carbon. when the permafrost is no longer permanent, when it thaws or burned, these gases are released into our atmosphere. over time and that coming to -- could mean that the arctic might become a new source of emissions that further accelerate global warming. if we do nothing, temperatures in alaska are projected to rise between six and 12 degrees by the end of the century, triggering more melting and more fires, more thawing of the permafrost. a negative feedback loop, the
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cycle, warming leading to more warming. we do not want to be a part of. and the fact is climate is changing faster than our efforts to it. -- to address it. that must change. we are not acting fast enough. i have come here today as the leader of the world's largest economy to say the united states recognizes our role in creating this problem and we embrace our responsibility to help solve it. and i believe we can solve it. that is the good news. even if we cannot reverse the damage we have already caused, we have the means, the scientific imagination, and
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technological innovation to avoid irreparable harm. we know this because laughter -- last year for the first time in our history the global economy grew and global emissions stay flat. we are making progress, we are now making it fast enough. -- we are just not making it fast enough. here in the united states where trying to do our part. -- we are trying to do our part. since i took office 6.5 years ago, the united states has made ambition investments in clean energy. we've now harness three times as much electricity from wind and 20 times as much from the sun. alaskans lead the world in the development of wind systems from remote grids. it is expanding solar and biomass resources. we have invested in energy efficiency and every imaginable
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way. our buildings, our cars, our trucks, our homes, even the appliances inside of them. we are consumers billions of dollars along the way. here in alaska, more than 15,000 homeowners have cut their bills by 30% on average and that collectively saves more than $50 million each year. we have helped communities build infrastructure to prepare for the impacts of climate change we can no longer prevent. earlier this month, i announced the first set of nationwide standards to end the limitless dumping from our power plants. the second most important step the u.s. has taken on climate change. over the course of the coming days, i intend to speak more about the particular challenges facing alaska and the united states as an arctic power.
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i intend to announce new measures. -- intended to announce new to address them measures. we are working hard to do our part to meet this challenge. in doing so, where proven that does not have to be a conflict between a sound environment and a strong economic growth. but we are not moving fast enough. none of the nations represented here are moving fast enough. let's be honest. there is always been an argument against taking action. the notion is it will curb our economic growth and at a time when people are anxious about the economy, that is an argument often times for inaction.
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we do not want our lifestyles disrupted. in countries where there remain significant poverty including here in the united states, the notion is can we really afford to prioritize decisions. the irony is few things will disrupt our lives as profoundly as climate change. few things can have as negative impact on our economy as climate change. on the other hand, technology is advanced to the point where any economic disruption from transitioning to a cleaner more efficient economy is shrinking by the day. clean energy and energy efficiency are not just proven cost-effective, but also cost saving. the costs of solar are coming down rapidly.
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we are still under investing. men of america's biggest businesses recognize the opportunity and are seizing them. they are choosing a new route. and a growing number of american homeowners are choosing to go solar every day -- it works. all told, america's economy has grown 6% over the past 20 years -- 60% over the last 20 years but our carbon emissions are roughly back to where they were years ago. we know how to use less dirty fuel and grow our economy at the same time. but we are not moving fast enough.
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more americans everyday are doing their part thanks to their efforts, america will reach the emission target i set six years ago. we will reduce our carbon emissions in the range of 17% by 2020. and that is why last year i said a new target. america is going to reduce 26% to 28% by 10 years from now. that was part of a historic joint announcement made last year in beijing. america will double the pays in china committed for the first time because of the world's two largest economies came together. we are seeing other nations stepping up aggressively as well. i am determined to make sure american leadership continues to drive international action. we cannot do this alone. even america and china alone cannot do it. all the countries represented around here cannot do it alone.
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we have to do it together. this year in paris has to be the year that the world finally reaches an agreement to protect the one planet we have got while we still can. let me sum up. we know human activity is changing the climate. that is beyond dispute. everything else is politics. people are denying the facts of climate change. we could have a legitimate debate about how we are going to address this problem, we cannot deny the science. we also know the devastating consequences if the current trend continues. that is not deniable. and we are going to have to do some adaptation and we are going
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to help communities be resilient because some of these we were not been to stop on a dime. we are not going to stop tomorrow. if the trend continues the way they are, there's not going to be a nation on this earth not impacted negatively. people will suffer. economies will suffer. entire nations will find it themselves under severe, severe problems. more drought. more floods. rising sea levels. greater migration. more refugees. more scarcity, more conflict.
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that is one path we can take. the other path is to embrace the human ingenuity that can do something about it. this is within our power. it is a solvable problem. if we start now. and we're starting to see enough consensus is being built internationally and within each of our politics that we may have the political will finally to get moving. the time to heed the critics and cynics and deniers has passed. the time to plead ignorance is surely passed. those who want to ignore the science, they are increasingly
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alone. they are on their own shrinking island. so -- [applause] president obama: let's remember beyond the climate benefits of more resilient energy, the byproduct of it is we make our air cleaner. and a safer for children to breathe. we are also making our economies more resilient to energy shocks on global markets. were making our countries less reliant on unstable parts of the world. we are gradually powering a planet on its way to 9 billion humans in a more sustainable way. these are good things. this is not simply a danger to be avoided.
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an opportunity to be seized. but we have to keep going. we are making a difference but we have to keep going. we are not moving fast enough. if we abandoned our course of action and stop trying to build a clean air economy and do nothing to keep glaciers from melting faster in oceans for rising faster and storms from growing stronger, we will condemn our children to a planet beyond their capacity to repair. abandoned cities, fields no longer growing and indigenous people who cannot care of -- carry out traditions that stretch back millennia. entire industries of people who cannot practice their livelihood. desperate refugees seeking the sanctuary of nations not their own. political disruption that can trigger multiple conflicts. that is not a future strong
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economic growth not where freedom and human rights are on the move. any leader willing to take a gamble on a future like that, any so-called leader who does not take this issue seriously or treats it like a joke, is not fit to lead. on this issue of all issues, there is such a thing of being too late. that is why we are here today. that is what will convey tar -- to our people, tomorrow, the next day and the day after. that is what have to do with we meet in paris later this year. it will not be easy. there are hard questions to answer. i am not trying to suggest that there are not going to be
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difficult transitions we all have to make. but if we unite our highest aspirations and if we make our best efforts to protect this planet for future generations -- we can solve this problem. and when you leave this conference center, i hope you look around. hope you have a chance to visit a glacier. or just look out of your airplane window as you depart and take in the god-given majesty of this place. for those of you flying to other parts of the world, do it again when you are flying over your home countries. remind yourself that there will come a time when your grandkids and mine, if i'm lucky enough to have some, want to see this. they want to experience this
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just as with gotten to do in our own lives. they deserve to live lives free from fear and want and peril. ask yourself if you are doing everything you can to protect it? are we doing everything to make their lives safer and more prosperous? let's prove that we care about them and their long-term futures. not just short-term political expediency. i had a chance to meet with some native peoples before i came in here. and they described for me villages that are slipping into the sea. and the changes taking place. changing migratory patterns.
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the changing fauna so that was used to feed the animals that they would in turn hunt or fish are beginning to vanish. it is urgent for them today, but that that is the future for all of us if we do not take care. your presence here today indicates your recognition of that. but it is not enough to just have conferences. it is not enough to just talk the talk. we have got to walk the walk. we've got work to do. and we have got to do it together. thank you. and may god bless all of you and your countries and thank alaska for your wonderful hospitality. thank you. [applause]
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>> ladies and gentlemen, please remain in your seats until announced delegation departures. [captions copyright national cable satellite corp. 2015] [captioning performed by the national captioning institute, which is responsible for its caption content and accuracy. visit] [no audio] [no audio]
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>> here is a look at what's ahead today on c-span. there will be a speech by agriculture secretary tom vilsack at the center for american progress at 1:00 p.m. eastern time. later today, a look at higher education graduation rates hosted by the senate education committee. we will open our phone lines afterwards to get your thoughts on the issue and will have a hearing starting at 3:00 p.m. eastern and we will start taking your calls at 4:30 p.m. on c-span two, live in washington, d.c., a look at the russia military relations with china at the center for strategic and international studies and that will start at 2:00 p.m. eastern on c-span two. when congress returns from its august recess, one of the first items of business will be a resolution of disapproval on the obama administration's nuclear agreement with iran another world powers. we will bring you key statements
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tonight and some of the hearings that took place after the deal was announced in mid-july including a speech by president obama at american university. house and senate hearings with negotiators and statements for and against the agreement by senate leaders. congress has until september 17 to pass that resolution. here is a preview of tonight's coverage. >> when i was in college, i wasn't a particular good student for the first part of college i was interested in sports and the latter part i was interested in working. i learned one thing -- i learned about the critical path method and went to things all over the country and i learned you can start with something like this and you lay out a vision and you build it out and you begin with the end in mind and you put first things first. it's the critical path. what i have seen our secretary do -- i know he has developed a tremendous warmth with the
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iranian foreign minister and talks about it often. what i think you have actually done in these negotiations is codify a perfectly aligned pathway for iran to get a nuclear weapon just by abiding by this agreement. i look at the things they need to do and the way it's laid out and i don't think you could more perfectly laid out -- lay it out. from my perspective, mr. secretary, i'm sorry. guest thata hotel leaves only with a hotel bathrobe on his back, i believe you have been fleeced. one of the events related to the iran nuclear agreement that we will show this week. it gets underway at 8:00 eastern right here on c-span. >> a signature feature of book
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tv is our all-day coverage of book fairs and festivals from across the country with top nonfiction authors. this is our schedule -- fromweekend we are live the 15th annual national book festival from our nations capital. near the it of september, we are in new york for the brooklyn book festival celebrating its 10th year. in early october, the southern festival of books in nashville. the weekend after that, we are live from austin for the texas book festival. near the end of the month, we will cover2 book festivals on the same weekend. heartland,tion's that wisconsin book festival in madison and on the east coast, the boston books festival. at the start of november, we will be in pearland, oregon for word stock followed by the national book awards from new york city and at the end of november, we are live for the 18th year in a row from florida for the miami book fair international. that is a few of the fairs and festivals this fall on c-span
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2's book tv. we will take a look now at fda approval of new treatments that use precision medicine. the manhattan institute put out a paper earlier this year saying where behind the curve on approving these new methods. along with the authors of the report, we'll hear from cancer patient advocate any medical geneticist. fda officials were invited to join the panel but declined. this runs about one hour, 15 minutes. [no audio] [no audio]
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>> hi, everybody. we will go ahead and get started . thanks to all of you for being here today. i'm the senior fellow at the manhattan institute and the new editor at forbes. we are here to discuss bridging the gap between precision medicine and the fda. me as a lot of you know the obamacare entitlement reform guy and that's what i worked on in my time at the manhattan institute.what you may not know is before i came to manhattan, i spent a dozen years on wall street as an investor where i looked at clinical trial data and fda regulatory actions to figure out which companies we should invest in based on development in medicine and science. this is an area i have long been interested in. i am glad to have this opportunity to help moderate
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this discussion today. foralk about how to pay health care and how much it costs. we don't spend enough time isking about how innovation transforming the quality of life for millions if not hundreds of millions of people in america and around the world. the manhattan institute has been a leader in this area. we have this: to to and called project fda which is centered around fda reforms. that has probably been one of the most productive areas at the manhattan institute in the sense that i don't know of any other think tank doing the kind of work the manhattan institute is doing on this issue. the question we will talk about today is one of the most interesting areas in terms of how we think about how to reform the fda. people always complain that the fda is too slow in approving innovative, new treatments.
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comef the issues that has up recently is this area of precision medicine. there are these things called biomarkers. when you go get your cholesterol checked at the doctor's office, that is a biomarker. that's what we mean. a biomarker is a lab test. it is something we use to correlate something going on your body with a particular outcome of disease. in the case of cholesterol, there is decades of research that indicate the high cholesterol levels, particularly a low density level protein or are highlylevels correlated to heart disease and heart attacks and other problems. that is a biomarker. 50 years ago, maybe we had a couple of dozen biomarkers. because of advances in genetic and all things we are
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learning about how different genes in our body regulate and produce different proteins with regulating the biochemical process in the medical processes and diseases, we on the verge of having hundreds of thousands of these kind of lab tests were biomarkers. they can help us determine many things. they can help us advance more quickly drugs to market that affect certain i'm tests or biomarkers. they can help us design clinical trials. for example, there are many people with lung cancer but it is not all the same. different people have different kinds. it might be responsive to different treatments. companies areug trying to figure out how to tailor their investigation into the individual patient and the clinicall patient's and biogenetic position. these are the kinds of things
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that are going on scientifically in the world in the private sector. dothe fda doing what it can to take this scientific knowledge into account in the way it regulates this development of new drugs? huber andght, peter out a newd have put paper which you have in your folders there. it argues that the fda is a little behind the curve in how these new developments, these new scientific developments can help accelerate the discovery and development approval and marketing of new treatments for disease. andill hear from both peter paul today and what brought to additional panelists with those who i think can illuminate a lot of this discussion. ckusill hear fromemile ka
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who is the ceo of ultra-genex who specializes in the development of treatments for ultra-rare diseases. was with was there, he another company that specialized in this area calledbio-marin. ultra-genex has had some success. it went public last january and has a market cap of two point $3 billion which 10 years ago was unheard of but has become more common as companies have gotten more sophisticated at how to develop these drugs. he will talk about the trials and tribulations he has had in this field. we will also hear from lynn mat the pancreatic action network. she will tell us at the patient level what some of the challenges have been in trying to work with companies and work
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with people doing critical trials of your eight -- doing clinical trials if you are a patient to develop new treatments. with that introduction, i think we will go ahead and start and maybe get the ball rolling. i want to go through that each of you based on your expertise -- maybe we will start with lynn -- you have had experience in the pancreatic cancer world. from the patient point of view, what are the challenges? how do you think about cancer varies between patients and what are the challenges in getting the fda and drug companies to to the unique characteristics of individual patients? >> thank you very much. yes, i will tell you about that through a story. center, called our call the patient central at the
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networkic action because her husband was diagnosed with pancreatic cancer. the calliates at center gave her lots of information about the disease and physicians who saw a lot of pancreatic cancer and nutritional information and that type of thing. charlie offered her and the opportunity to get his tumor molecularly tested and do a aopsy and do something about test that will tell us something about what was causing his particular cancer and therefore how we might treat it. that made marjorie pretty happy because she read the statistics of she knew that only 76% people were not going to be alive in one year and only 7% were going to be alive after five years. she said this is something i can try.
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the current standards of care are pretty crummy. they agreed to do this. about a month later, they got has a report and it mutations and alterations in jeans that are underlying charlie's cancer. luckier than 55% of pancreatic cancer patients. based on this molecular profile. there are some options that his position would never have thought of before. some of those are off label treatments so they are drugs that have been developed and are for aers that mutation that charlie has in his cancer. some of them are clinical trials. here is where they were faced with not really medical problems but system problems and problems
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with how does he get access to these drugs and how does he decide what is working for him? there are no real clinical trials in pancreatic cancer for any of these drugs. there are 50,000 patients per year of which 4.5% of them go on to clinical trials. 2%n this mutation is only in or 3% of those 50,000 patients. 3% of that going to clinical trials so how do we learn about what to treat charlie with human that we have this molecular information? happens is that even if he can get on a clinical trial or get off label treatment and there is all sorts of insurance issues and being out of network and those kinds of challenges
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that he will face -- even if he can, what usually happens in these clinical trials is that he is treated with the drug and then three or four month later, they take a radiological scan and they look at the tumor if it has grown are shrunk. the overall result is whether charlie lives or dies and how long. he lives. in the meantime, they are checking what's called a biomarker in his blood 9 which if it goes up, the tumor is probably growing but we are not 100% sure that is always true but there is good data that says that is often the case. if it goes down, that's a good sign. act on that information. there has not in the proper studies to know whether the drug is doing charlie any good or not. if it's not, he could go on another treatment.
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i think that is two examples where we do not have the information to determine what treatment we should give charlie and we are not able to act fast enough i biomarker to determine whether we should change that treatment or not. i think those are both opportunities for the fda to pay attention to a disease that is life-threatening. charlie does not have a lot of time. survival is measured in months and weeks and this disease. when he to act faster and we need to change the system so people like charlie can benefit from the science that we have around cancer treatment these days. >> let me introduce peter. he is the senior fellow but i like to call them the resident genius of the manhattan institute. he has a phd in mechanical engineering from m.i.t. and a writesree in harvard and
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about energy policy and legal policy and tech policy and is cure andr of the the coding of 20th-century law is undermining 21st-century medicine so he is equipped to talk about this topic today. how do we take that every day patient example and translate that into better policy? >> the first thing to understand is the example that lynn went through. there will be a test for a device that will check for these oscillations in a patient's bloodstream and what it tells us about the drug. it does not get to the market without the fda say so. i have a number of oncologists that tell me if they were not allowed to describe off label which they do, oncology would shut down. guest: >> explain what that is. >> that means the drug has been up for something but not for what you are prescribing it for. i don't know what the off label drug was in your case. is not capturing every
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possible use of every drug. your life depends on people who do not believe that. there is hope for a lot of off label work because it is happening all over the place. it can look different in different parts of the body and have the same underlying mechanisms and repurpose the drug and prescribing drugs based on their molecular effects and the ideology -- the etiology of the cancer is widespread in this area. it has been used in other areas but this is where it would be most effective. if a drug company walks run and says it does this for pancreatic cancer and if the owner of the drug tells you, they will probably get prosecuted. they will certainly face a civil suit. these are billion-dollar issues for drug companies.
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they known off awful lot about this and are interested in their drugs. they are compiling the data. doctors who share this can and the fda has not gone after them but they very well may . fda questions these uses. it has not certified it to be true so it wasn't true and they had to shut down half of their business in the state. has plenary power over how much of this biomarker inormation you use in a drug the approval process print you have to persuade them that this biomarker is good and valid and can be used. this is especially painful because the best people at the fda and i would start with dr. woodcock who is the head of the agency, they know exactly what is right here.
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there are excellent presentations that were given 10 years ago. foundation of the evidence-based medicine. they determine what happens and what we should prescribe to home and outcomes happen to people not populations. there is an indictment of what the fda has been doing for 50 years. the fda clinical trials are population-based trials and are overwhelmingly based and looking for imperial cult -- for empirical correlations. if what you have defined as the is 25 different diseases, the drug does not get through. there is a core problem with the fda and their existing trial protocols which statisticians call the reference class problem. represents 1000
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patients, let's do pancreatic , this row here, if i round you up, 15,000 or so and it think we had a cancerous identical disease, then that is a pretty good sample. we know today that june on the have shown in august amounts of variations in genes and the proteins that they code for. the saddest part to my mind is the fda issued its first guidance, as they call it, for possibly early 2004, ok? the nih is one of the agencies in washington that does a lot of genomic research. they said, well, nice work. helle tell us what the you mean by validating a
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biomarker. folks, we have set that internal standards for what we accept as good funds. tell, it iscan without a trace, ok? the fda dider, issue its critical path report saying that biomarkers are something that we really have to do and they said that an institution called -- what is it called? cpac, the critical path institute which got private funding to research this. meanwhile, we have an enormous amount of taxpayer and health care money going into sequencing tumors. every time doctors are treating patients like us, we are getting new sequences. they are firing clinical data at the same time which made your developing huge databases that they receive these molecular profiles and are associated with
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these effects and you can basically do epidemiology for these biomarkers. this is how we isolate between microbes and many diseases. you can begin working out molecules. molecules are way more complex, there is not one-to-one correlations, they work in very complicated networks. we have fantastic competing they haveay and decreed and stated publicly and he is right and google knows this and that is why they're getting into business and apple knows this and that is why they're getting into business and all of silicon valleyand has not reached washington you. the have incredible computing tools. you give them enough an update and they can unravel pathways. to say, whyod not would darfur, then advanced research projects agency, suddenly be interested in cancer? they actually issued a big mechanism notice a couple of beers ago saying that if we give you a really complex data set, get some ingenious technology working out mechanisms within
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that data set, and vitally, the nsag systems, too, has this huge database [indiscernible] person, wep this will stop having terrorist incidents, i'm quite sure the nsa would not want to share its database of every risk of the country. same goes for cancer, and we have terrific oncology and they haven't exactly right. the fda has the lead analysis and affiliated to the nsa, we would be in really good shape. this is what it is all about. political you found a platform for the next president. put the nsa in charge of drug development. [laughter] peter: just validating power. acquisition. >> let me ask you, i think what
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i have taken away from both of these discussions, from lynn's point, which i think is interesting is that there are these patients, two to three per -- 2% to 3%, small populations within a large population of people of cancer who may have a unique genetic signature, but that is a tiny number of patients. it is hard to show statistically in a tiny number of patients that the drug is actually having an effect and not just a noise, a placebo effect, so how was the drug company feel ortho biotech company navigate that scientific problem of genuine -- of generating genuinely statistical evidence that you found a correlation between your drug's ability to target this particular genetic subpopulation? to peter's point, how do you take this big data idea --t is revolutionary revolutionizing the rest of the economy, and had to convince that the a to adapt some of that
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technology into their own thought process? >> we don't, but we would like to. that welenge i think in deal with rare diseases every day are these small subsets and we have been struggling for a long time. these biomarkers are the most important ways to gain power in understanding what is going on in a precise and accurate way that allow you to actually determine what you are doing in a small population. the challenge has been, and there are thousands of diseases -- where diseases, thousands of which you do not know how to spell, save them, or never have heard of them, but 10% of the american population is affected with the rare disease and probably all of the miss some of the rare disease and 25% have treatments. we should be doing more and being able to get the power to determine what is going on in these patients at time markers is one of the
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critical things that we believe. the president of a foundation that is focused on where diseases and we focus on how to use biotech -- biomarkers and we believe that is one of the key pieces that will help her diseases as well as patients of pancreatic cancer, and the stories that peter has been talking about, so i can say this , the facts are that since the beginning of approval in 1992 after the hiv and aids crisis, when it was first [indiscernible] time, in the rare disease genetic area, only to diseases where new biomarker has occurred has been used in those whatever 23 years. i was involved in one of them and the other one was have her son. -- all of them have been ones that were approved and drive and
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in the early 1990's, so there hasn't been anything new. the question is with all the genetic information we had and all the bread diseases out there and the science that has been done, that we know and understand what has been going on. how can it be that there are no biomarkers based approvals for more diseases that could require them? them becauses had i working on a program for disease and it -- type seven, that disease is a slight disease. only 20 patients in the united states perhaps with this disease. yet, we have a treatment that has been around for 20 years. and not beautifully translated because we cannot detect or do a study with 10 patients and prove it is working. biomarker, how much material they have in their bodies, we could do it immediately. we have got bma to a great and
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fda has not agreed at this point. with a biomarker, we can run the study with 12 patients and get the study to work. based on all the signs we have, showed that is a reasonable treatment and get these kids treatment. what is the point of all the nadh research we have been funding? the billions of dollars we have been spending and if we find the treatment, we do not turn them into products for someone to get? nps has been a tragedy for seven. to show why it should be done and how it should be better. we continue to work on access and policy and legislation. it has been challenging and i my manhattan students are tied to the got away to make some financial happen on the biomarker front. >> a meal, even if the case with their company, altered genex, withg to study disease
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clinical trials is physically impossible because you cannot ethically do a study in where you let the patients died. you have to treat them in the way that you know how to treat them. the only way to measure progress is through the biomarker or lab test. if janeter problem is woodcock were here or representative of the fda, she or he would say, well, it is great that you have all these biomarkers, these test that may correlate to better clinical outcomes, better performance on disease or measurement of they probably would have otherwise had, but we do not necessarily have that. we do not necessarily have a inclusive proof that the biomarker is correlated to better disease all caps and longer lives in statistical situations, so push back on that . tell me why the fda should not be cautious, should not be scientifically conservative and say, you know what?
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yeah, this biomarker sounds like it is a good thing to test, but there is no definitive evidence that it actually means a patient who does bad on a lab actually going to have a longer life or do better on a disease. >> i think one dimension of that problem, if the industry would say, it doesn't make it easy, you can make it hard, but tell us exactly what the standard is invalidating a particular biomarker for the context of use. we would go out and we would develop these weapons states or find studies that do that. concernede agency is to something like this -- funded researcher runs a study, discovers a biomarker for cancer of the diseases, publishes an article in science, declares victory and moves on -- the agency would look at that and
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say, gosh, that is not a biomarker, it has not been reproduced, how do we know that is good science? how do we know it has been validated? what is the reference class control that would prove he would hit something that is really important for that particular disease or indication thatw abuse question mark -- that indication? i think one year or two years ago, researchers out at stanford , what they did was to achieve expression profile, looked at classes of drugs that were already approved by the fda and then compared them to cancer expression profiles across a number of different tumor types. what they found was a match that were hitnes by a 50-year-old class of drugs of antidepressants, some of the first i came online, and a very rare class of lung cancer called small cell lung cancer, about
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20% of cancers. i think it was called the classine tumors, a small of pancreatic tumors. they said this was interesting. the pathway that these drugs inhibit, it seems they are implicated for this class, particularly in small cell lung cancer but other cancers we know endocrine.are let's do the science, so they ran and looked at tumor cell lines, they looked at knaus models where they grew tumors in mice and then they hit them with the drugs, they hit them with other drugs. it was a neat experiment, and they said, you know what? we are to launch a clinical trial. so now 15-year-old drugs are in lungng for small cell cancer and face to be trial cells for efficacy. as a really interesting things, but unfortunately, these rugs are 50 years old and have not been used very much. this is a more recent class of
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drugs. we have lots of data on observational data, so we will look at these positions and see how many patients have been taken lithium or other classes with rates of brain cancer response or better no endocrine tumors for lung cancer or other cancers and do they have good response rates? there is a range of other dad is taken years to valid -- there is a range of other dad as they can use. it is observational data. they can be used to enrich a clinical trial for a particular class. if you can get down to a little of knowing and what it is exactly that is causing those cells to self-destruct, billy can use that as a circuit in point. i think what we are asking the fda to do is hand out that evidence of development which they have said they are not equipped to do, or have been reluctant to do, to the people who are capable of doing it, which is the research community. >> would that be a particular
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professional society over that the -- or what they deputized certain academics? how that process go? computer: how about getting it to the people who are spending billions a year on taxpayer money? the nih has characterized them as characterizing genomic variations that have medical impacts. not just say to anybody who would like and send it in. one database that i know that they monitor is where they invite people from all of the country descendent reports of adverse affects on drugs. it is important to monitor and i googled this and i kid you not, they tested that they have done vaccines that transformed into lk, so that is the one you should be worrying about an apparently got a call sometime later saying, oh, that is strong. first of all,
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people talk about drugs doing a biomarker. certain endpoints are not drugs pointing toward a biomarker, they are drugs changing the level of some biomarker, ok? certain endpoints are about understanding. if you're going to do a collective, it is understanding the pathway which will often be a whole series of interactions and alzheimer's is probably dozens or hundreds of proteins in a chain. it can be any number of them and each one will have the same effect because it is a chain effect and the weakest link is what causes the lead. you have to understand these mechanisms, that is why i am glad dartmouth is looking, they deserve a nobel or something. you are talking about mechanisms of action. if you contact those down in much smaller data sets, in fact, you don't really have to use of statistics at all. you can simply wake up and this is leading to that, leading to that and these are a lot of experience. i would also add that the tumor
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typically for the more common effects you can find 10,000 mutations in a cancerous and they can go crazy. they mutate in every possible direction. they just do it wildly. these analyses are sophisticated methods. these tumors have -- they call it days and network analysis, i have some of them in my book, they look like this huge spidery web with links between them and you can quantify the links and it is important to note that these are hierarchical networks. almost clearly important all variations and others are playing alongside try they map our system systematically and have four time, they major categories, depending -- i think there are four major but more recently become of the database is [indiscernible] statistics -- that
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non-basics as his six, they do have these large databases and it does it very well. the drugs are developed to be oriented and homework. with theobody can number that is collectible from a molecular perspective. associatediseases oftene effects that are one to one. sometimes you have to double up get better effects. once you have it narrowed down, you can do very good studies on and it is a very long chain reaction and more difficult. that is of modern computing is. >> i think two takeaways from your paper, actually, that i thought were very interesting was one, first call, the fda is
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very restrictive in the ability to talk to drug companies and biotech companies because of conflict of interest that prevent them from talking. even the national institute of health, which is the major governing agency that runs a good -- academic biomedical research, even nih does not have a lot of cross channel abouttion with the fda some of these advances and maybe as you said, that they could the with nih and use nih as intermediary to help develop some of these standards. the second thing that you talked about in the paper that was very interesting was the fact that their european medicines agency, the em ea, european equivalent of that day, actually spent more forward thinking on some of these issues relative to the u.s. fda i thought, paul, maybe could talk about a little bit , the reason we have this $2.3 billion market cap is because the em ea has been more forward thinking on some of
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these issues than the fda has been, so maybe you could touch on that as a. first paul and then emil. paul: just a look at one of them and say, there is a foundation for the nih. there's biomarkers consortium which is a private profit hat worksn t because academic researchers and are companies have an interesting partnership that we talk about called the advancing medicine's partnership looking up for diseases or five diseases. they do exactly what we are talking about, you go through, validate biomarkers, and debunked evidence. i think it is a big step forward. and then i think the emea has something called innovative medicine initiative which is making biomarkers one of the key projects and sending hundreds of
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millions of dollars compared to a few tens of millions of dollars a year and that could be doubt they, too, but i think the key is getting this outside expertise that the agency is usually walled off from, involving critical standards so that people like lynn and emil and academic medical community and the drug companies can say, this is all the evidence we have. this is how it can validated and we should go about validating so we can affect and check things off the list. i think what i would like the market is it is not all based on biomarker -- biomarkers. [laughter] and i do not own it all, to the clear. i think in general what we can and thet the ema agency, the last few years, they have been more scientifically regulatoryss
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precedent base. they're willing to look at what you've got to make intelligent decisions about it. in the case of mps-7, we showed them the plan and how we present the data and that is based on false the kitchen criteria that for qualifiedhed biomarkers. we create a very thorough, thoughtful set of on how to qualify a biomarker and we present it in that format to them. they were convinced we have a lot of cash and updated pieces put together to explain that it would be impossible that the marker is not correlated with disease activity in the patient saidould not predict, as i to them, and there is also the drug approval that made sense. thisthey told us is that is the only way you can do this study, that is what he said, the only way to do the study in the disease is of the urinary substrate measurement and there is other way to do it, so their view is this is the only way
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forward and we will accept your plan because we think it is reasonable to do given the rarity of this disease. i think that is the basic fact, at some point in the rule and regulation policy in the country, we have to make it practical realistic. otherwise, things will not get done. to say that i want a clinical driven and i went walking test than this test and that test, unit 100 patients or something to do this study and there are only 20 in the united states, you're basically saying that i figured out a way that we are never going to find the science for the patients to retreat and that we want them all to dive rather than risk getting treated with a biomarker endpoint. that fundamentally is no one us would agree with that. that is sort of the ultimate outcome of that decision and that is what we need to change. i think there are ways to do this in a the way and we proposed them.
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some of the low group of industry, academic and foundation people, developed the criteria and wrote a paper on it. it is not trivial, it is not quick and dirty, it is there a, , and the key thing is it does not require things that are impossible. there is little to require you to know the cardiovascular outcome in the disease that has less than 100 patients 10 years from now. you're never going to get that data. what about the data you do and how good is that? what the law said, and this is important to member, they had a piece on approval that said it was to include ways to qualify a biomarker endpoint by using pathological and pharmacological criteria were other types of data, in other words, outcome data, was impossible or impractical to collect. so it really struck me that you are hearing your choices.
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you are not creating unrealistic and if possible barriers that will cause the disease is not to get treated. i think that is where the differences. you may see the practical and scientific side and i don't think that they did -- theft is a can of precedent and concerns. i think their concerns come from the fact that a couple of drugs and the harder area and circuits did not play on people were hurt by that. these big fancy cases of drugs in the big market area that did not play out. it is causing them to flinch and move away from diseases where we know a lot more about what is going on with a specific biomarker and the clear genetic condition. it is just not the same situation where biomarkers have failed in the past. we need to get over that story. i love bob temple, he is a great guy, he keeps telling that story and needs to stop telling that story. know,to get on with, you
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new science and where we're going now which i think is much more soundly based. >> when i introduced lynn, i neglected to mention that you are president of the national cessation of cancer research which is the most important academic society in cancer research and the country and she also worked at the national cancer and the national institute of health as a phd in molecular biology from the university of arizona. and it islk about obvious points that you have been hearing. lynn: well, there are lots of ways that i could do that, but what occurs to me is that what we are duly saying is that the fda looks like one of those human resource departments that you roll your eyes at that says, one-size-fits-all. you've got to do it, these are the standards, everybody has got to fit into these groups. you know -- and for those of you who have been in that position
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where you just say, come on, we have a mission here and there is something we need to complex and there needs to be some flexibility around, so i think for historical purposes, the fda has this one-size-fits-all. big not -- not a real government agency and in order to gain efficiency, everything has to fit in the individual little silos. i think that is what has to change. this idea that not all diseases are the same, they have a very important role in protecting our society. and protecting us around drugs that are out there that thousands and thousands of people take who are relatively healthy. blood pressure controlled medicine, those types of things, that is a very different story than rare diseases or deadly cancers. to bethat ability flexible enough and yet stick to the principals of some sort of
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scientific validation and overlaying exactly, as you are the principality of it. what is really required here. in a population that we are addressing, what is their risk tolerance? i confided that pancreatic cancer patients are, in general, much more willing to take risks with their treatment because what they are facing is really virtually certain death otherwise. of thaterlaying all into the decision-making that goes into what is approved and what is the level of validation that we need for those biomarkers that seems to be the mindset that has to change within the agency. roy: how good is the science or, lynn? a paper came out of johns hopkins, suggesting or indicating that sometimes when and a tumorprofiles in a particular patient that we are not actually getting good data because if you do not
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benchmark that to the baseline genetic profile of that patient, maybe you are not getting accurate information about how that tumor is distinctive. there is a lot of commentary out there that, ok, yes, it sounds great in theory to do this genomic work and biomarker work, but at the very basic analytical level, are we at the right level? enough windows analytical tools that we can actually say, ok, we are actually measuring accurately the genetic profile of the patient and being able to make the decisions on that basis? don'tit comes down to throw the baby out with the bathwater. it comes down to the research component of it and the practical patient treatment component of it. not what, yes, it is hundred percent certain that it will respond to a receptor like
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pancreatic cancer. i have no evidence of that, but i have seen it work in other cancers. this patient has very little options. i see no reason to say, well, i really have got to check their genomic dna and they should that this is right. perfect, it is the standing in the way of the good and moving forward. wea research setting, yes, should be doing that. in a research setting, you should be sequencing both the tumor as well as the normal dna, looking at the difference, understanding what is specific for that tumor versus what is normal background for that person. that is all absolutely true in the research setting, but -- and that will eventually inform the initial -- the patient 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 the
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statement is that people would still up their hands and say, well, we should not be doing any of this because it is not good enough. it is 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 are only giving them a couple months to live. it is a whole love better than that. have clearyou standards of the, the industry -- paul: once you have clear standards of that, the industry has a biomarker with their product because they see, i know how the process works, we know what to expect. if there is clear evidentiary standards for the qualification of biomarkers and the industry will suddenly say, we can reduce the cost of development, the time it takes to bring new canucts to market, and they invest in bringing academic research and other tours up to the level needed to meet that, so i think that is another half.
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forear pathway as there is approval of cancer and hiv and some other drugs that have basically 90% of the approvals which circuit are three classes of drugs for cancer, hiv, and anthrax. move beyond that empire part of that is setting up a path that drives investment rather than raising the levels of going away. : we are going to go to questions. there is a microphone circulating around. when you get the microphone, give your name and your creation because this is going to be on c-span, i believe. while the get the microphone ited around and deadly hands to other raises their hands, let me ask one question to the floor -- there is something going on in congress rental call the 21st century cures initiative. it is through the house, energy by conference committee led chairman upton. they're planning to develop some
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of these ideas into a concrete congressional bill that would push the fda in the right direction. i opened it up to the floor, have you all been following that process, how do you feel that is going, are they doing the right thing? are the things you want them to do that they are not currently thinking about? emil: my foundation is working on one part of it, the open act, which is a provision which allows companies to repurpose them for rare diseases as an incentive to get more of our signed that we have already created for rare disease patients. the goal is extremely long. i think it is more than 400 pages and a lot of sections. it is a very ambitious effort and i think what fred upton was trying to do was get all the best ideas speaker, put them in, looked through them to come up with something that would really change the way things get done. on the help side, there is something that spain put
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together that will be comparable, but i think there are things with patient input into the drugs process. there are incentives and a number of other aspects of it that i think could improve. i think it is a good discussion to have because it gets different stakeholders outside of the fda to start talking about fixing and improving things as we need to. would not be we having this conversation without chairman upton and the commerce .eant for leadership we are focusing on the biomarker piece of it and i think that is a good direction because it is recognizing that evidentiary stands for particular context for use of a particular biomarker and that would incentivize investment and bringing in those other expert bodies can be those groups and make each of the patients are at the table, industry at the table, to develop standards that ensures everybody the science is done correctly and the fda will recognize it. : i might add it is quite
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stunning how bipartisan this has been. they have equal numbers of democrats and republicans and i might add taking obama's count through technology two years ago which gets all this right through, as far as i can tell. a lot of people believe that this stuff is science about saving lives. there is a lot of political attitude. think this is politically wonderful about this. it looks really well on this and the responder project that and iric -- that paul wrote about several weeks ago, as far as i can tell, they are doing retrospective studies of clinical trials of cancer drugs that fails on fda standards and --ng back to reanalyze that
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for drugs that can in fact work well. i think it is truly wonderful. cures is acentury step in the right direction, getting perspectives at the table, the devil is in the details, and there will be an awful lot of working through the details as to go forward, but it is certainly -- it is delightful to see the effort being put into that. avil: let's go to queue and date. remember your name and affiliation. yes, sir? >> i'm a lawyer in new york city. i just would like to ask the , listening to the discussion, i have read complement about darfur and nih, but i have to ask, to what extent has fda outlived its usefulness? they do some things that remain useful what are we better off limiting the jurisdiction at a macro level and permitting the
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market to take this on and move with it? me i would assume that is a step or could be a step backwards in terms of safety. i think we rely on somebody to determine the things that we can buy and get prescribed for us. that we understand what those anyway.arameters are in my mind, it is a matter of diving in flexibility. believe that we need in the specific situation as opposed -- the flexibility we do in situation as opposed all. emil: if you ask any of the biotech investors are i know, pretty much everyone, they would say that getting rid of the fda would disaster. the fact is, companies are i sometimes. sometimes they do studies are of a time not disclose what went wrong with the patient or a be
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the base like your district for patients with different. the journal of medicine does not have the regulatory authority to audits those patient records. they have to rely on the on the of people who submit those instead to the new england journal of medicine, let's say, where the fda goes through with a fine tooth comb. maybe they go too far, but they are able to do things in terms of the rigor in which they analyze clinical trial data that civilian institutions are not able to do. for me personally, and a lot of people in the investment committee would say, there is an appropriate role for the fda but should the fda role be modernized? i think that is why we're competing at the meeting. l: the fda is actually necessary. : i think they need to help recruit more people that are connected to the academic world as they were when they had more which would help them be more creative, connected to the
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science than they are now. the easiest thing to do to help them get better, i do think they need more funding. i think they need to figure out how to better organize and look for people that can keep connected to what is going on, drive the science down to the review level. i think they need fewer political in the shares on programs where they hire people and need to get more money into their drug review and the quality of people involved and appropriate as for biomarkers and other things. are ways to make it work and i definitely think it would not work well without them. peter: i don't think there is a single major drug company that would say yes. it would be a stockholm syndrome problem. [laughter] they are well aware that a lot depends on confidence and social miss. -- and social miss. i will add that the rise in medicine is progressively moving
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power away from the fda. i do know if obama was aware of this when he endorsed medicine in the state of the union it,ess, but it darfa can do the fdally the world, has not said it is ok to prescribed is canceled drugs off label, but they will be described as off label. the problem is getting them to market. once it is in market, that they lost grip on it. have a drugsay you identity has approved for breast-cancer, once the drug has actually been approved by the fda for any disease, a doctor has the liberty to prescribe it for anything. in fear he, the dark that has been approved for breast-cancer, your doctor can prescribe it to you for high cholesterol. that would be stupid and super malpractice, but he has the legal authority to do that. peterappens a lot is what refers to, doctors actually have a certain amount of liberty once
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the evidence is out there in the public domain to say, yes, this is out there. there is evidence that it may work for your pancreatic cancer or your lung cancer or your high cholesterol, so i'm just going to prescribe it to you for that. once is where the fda wa it approves a drug, loses the story which is light has become more conservative in cases of approving drugs because they are worried. they know that once the cat is out of the barn, they do not have the complete control anymore. peter: they have more than you are suggesting. assumeredible courage, i you know about the drug from the 1960 that caused birth defects across europe and australia, it was a licensed drug in the united states but they put on a lot of conditions. i might add it scare them even more. [indiscernible]
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looked at your jeans and said it predisposes you to this, that, and other things. people are being alyssa to do the diagnostics and they are ofng it on the huge scale much more data and specific recommendations. lawside, the federal drug is written so broadly that everything google sets up in the country is a technical and medical device, telling doctors to treat patients with that. to holde not dared their fire. if they do, [indiscernible] acvik: let's get to another question. recently retired oncologist. i want to talk about what mr. howard said, this goes back to had the -- 1969 when we
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combination of approved ticket hodgkin's disease, to cure 62% of the people, the single drug that we used was assisting nine-month remission. we discussed it at that day never approved combinations of drugs but we went ahead and did it. he never got bothered and we still laugh about it years later. was reallyly -- it well to has the mutations caused by other diseases, but the key issue that has to be addressed is that when we went down to the american society of clinical oncology toronto congress, they told us that just get some patients, put it in front and they cannotnd, argue with cancer patients. when you come down here, they think your budget money grabbing has you want to make a lot of money and i'm sure the same goes for biotech people. my question is -- i'm so glad to see you, what more can we do to get patients involved because it
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is very difficult to do, especially with pancreatic cancer, but the people who have gotten involved, the breast-cancer patients, people listening to them, we need a patient approach because congress people think one way and it was sad to see john against president obama. he was one of the people must against it. david and that is sitting next to the president when he approve something for health care. we need patients involved and please, tell me what you guys are doing? l: almost everyone is going to be a patient at some point. with new technologiesp -- in line l: i think it is about $1000 now, but the price is prevalent to the program that it
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will get at the clinic and at some point, they will get pricked, and look at it and say, we see predisposition for heart disease, diabetes, or potentially colon cancer. that level at which everyone suddenly becomes a patient advocate or potential patient advocate i think is a game changer. lynn: the advocacy organizations have a very important moral and exactly what you are talking about. using the power of the people, the power of the patient in order to influence change. thatw for specific example cancer research act was passed into law of january 2013 as a result of the efforts that started out as pancreatic cancer research act. we could get 600 patients, not usually patients but family members of patients of families who had lost someone to the disease, all dressed in purple on capitol hill year after year,
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so this is important to us. the end result was asking the nci to come up with a scientific framework for the deadliest cancers which are the major cancers with five-year survival -- 50% or0% or less less. that is something the nci is acting on in terms of looking at that from a more strategic viewpoint as to what can be done with what disease. i think those are the types of things that take a long time. you need a lot of organization in terms of making sure the message is consistent. that you know that what you are asking for is what you want. a lot of those things, but that is really one way to affect the kind of change. emil: we actually organize the rare disease community and we have about 80 rare disease groups that are behind our campaign what we call the cure the process campaign. every year, we bring about 200
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through ago conference to tell them what is going on. have lobbies, and train them to know how to talk to congress and staffers and we set up meetings with their particular congressman or staffers. we send the knot on the hill, we put ads in "politico," and we make it a big where disease week and we have a premier movie where we do a rare disease caucus and you make a big event out of it and we do it every year now. they know we are coming. actually, congressman with rare disease issues feel like these are good events for them. we work both sides of the aisle. they are very aware and some of the 21st century cure said is basically where disease focused work. it is not just the company. it definitely has to be the patients that are affected by these for diseases telling their stories on the capitol hill and we do it in an organized way
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with enough firepower so that they can feel the presence of those groups by having them contact the hill on one day every year in february. avik: any of the questions? amy and i teach at the university of pennsylvania. i am wondering if one possible way for the whole ind ticket and it is a slow ticket would be for the fda to have something like a recalcitrant disease program geoff hart about specific dismal a lot of them would be cancer and create a much freer, much looser regime for treatment protocols. i can see one obstacle to that is this kind of conceit that existing treatments work and everything has to be compared to existing treatments. i think that is probably punish us for diseases like pancreatic cancer and lung cancer -- but it
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might create some tension to say that the really works. so we should just try anything. i think that is exactly along the lines of trying to aen it up so it is one-size-fits-all. even if they have to lope some diseases into those that are particularly difficult and really recalcitrant and nothing to do for them, that might be easier for them to do then to really think about it each disease individually, although it -- at some level, that needs to come to play. avik: next question. jonathan: i'm jonathan, network for excellence and help innovation, can you comment on if you are doing any work or have any feelings about the payment side of this? certainly in another reason why
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do has not been a lot of activity in the biomarkeris i do not think you could even get something approved by the fda. you have to go through another three-year to five year odyssey before you determine what you are going to get paid. it tends to be very depressing for number of projects that get financed. with the exception of a drug company with absolute positivity that it will get its drug approved and has an essential trial onnt for a test the door, have you looked at all what the incentives are for product developers and what they get to the fda? peter: i think it is a major economic problem for reasons slightly that you suggested. i know there are only two ways to monetize that can serve in an point. first of all, let's put it this, if you come up to an end point for all timers, a good one that fda approves, it would be $1
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billion discovery at least. definite all summer drugs with conventional fda trials would be [indiscernible] too slow of the disease. it is interesting in response to an earlier question, people across the united states and huge debt of gratitude to the gay community with their activism in the late 1990's and my team night -- and 1980's which is physically the primary military -- primary way of introducing from logical and molecular stuff. but the two tools for monetizing these discoveries are the diagnostic devices. if you're going to have a targeted drug that is prescribed in these conditions, and you will have to have a diagnostic look. i'm not sure if they still do it but the fda still -- required a companion licensing of the device. the problem is that if you are good pioneer and you find a new target and you find a way to accelerate the approval, so now
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you have a pathway for rapid approval by the fda, it is easy to follow up and someone to steal your invention. they work out the mechanism of actions of your drug, the conduct of studies, and they arrived on it. statins.ic story are the japanese researcher worked at the basic chemistry and discovered the first but did not work out the chemistry but his grandfather looked at the wonders of nature that produce all these medicines and he figured, they be some microbes might have found a way to disrupt cholesterol because cholesterol is also used in nature, so meanwhile, in the united states, they were working out the molecular cluster populate and they got no buffer that. they caught the most decorated molecule in history, anyway, -- [laughter] these people did incredibly valuable work. the whole staffing industry sort of hinged on what they did.
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one british company came out with another patent but it did not go far. so pfizer ends up with about three or four drugs later doing lipitor which is a billion-dollar debt. we have no way to monetize this expensive research into chemistry. we need some kind of intellectual property and researchers want everything shared with everybody, but this stuff is expensive and you have to have some incentives. paul: you could say like .5 of revenues that would be any product that is approved using a validated biomarker would flow money back into diagnostics industries, back into the academic medical community and would encourage companies to share data. if they say, we could pull our data and pulled revenues and that would allow us to get biomarkers validated that much faster, so much the better. avik: any other questions?
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one more? we done? let's do one more. mix signals from my governor's over here. >> sorry told everyone up. i am genvec from the parkinson's disease foundation and i really enjoyed your discussion today and it focused a lot on cancer. i represent urological parkinson's disease and other diseases like ms and alzheimer's. i think it is important to recognize that this is a rising tide which can raise both, and we focus on the genomic area and how it helps people to recognize that something as simple as mri scans or pet scans can really help these other neurological conditions which currently represent barriers for drug approval. there is a scanned for parkinson's to indicate whether someone has or does not have parkinson's, but people who are
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negative for the scan still have to be included in clinical trials even though they do not have parkinson's disease. the fda won't allow it, so how do we increase this in order to get fda to say, we recognize these people do not have parkinson's disease, you connect with them from your tiles, therefore, in short that drugs which can help will actually get to approval to help people living with the disease they have today? paul: a big chunk that we did not discuss is actually located in the slips these and starting point. i mean, is there profile that tells us hopefully well in advance, yes this is subject to both of this disease. peter: there are quite a number of rare diseases where the profile is crystal clear. you can scan and a number of other things. ofm what i have read, a lot -- would you call parkinson's ideological disease? they are very complex from
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molecular perspective because you have changed propelling the signals and numerous signals and so on and be multiple molecules in those chain and variations in any number can lead to the same disruption that causes the same clinical symptoms. and the institute for dental health recently -- for mental health recently tossed out standard definitions of neurological disease as we are setting that anymore, we are not funding that. they use the analogy for urological medicine that we will isolate the pathways in the pain, emotion,ct and some finite numbers and they will work on those as much as possible. the use of biomarkers, it was the fda and the city is tooting its own name, [indiscernible] of course, we should not be
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testing someone who does not have the disease. the second problem is that there are quite a few people with the genes but they happen to be really good and not succumbing to a disease even if they have been affected. there is a micro-fraction of people that get infected with hiv but it never progresses. a rather small number but they have control. there are other markers like this with cancers and so on. you do not want to test drugs on people who are not sick because it stacks the deck wrong and makes the medical trials incomplete. that's the best i can give you on that one. avik: i think we have covered a lot of ground here and i want to thank all of our panel for being here. the particular emil. [applause] i want to thank the reporters at the manhattan institute for helping us invest in this important and neglected area of policy, so thank you very much. [applause]
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[indistinct chatter] ♪ >> this he spent cities tour, working with our cable affiliates and visiting cities across the country. this weekend, we are joined by charter communications to learn more about the history and literary life. the mining of a certain mineral had an important part in this town of colorado. >> all over the colorado plateau and in mesa county outside of
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grand junction, we are surrounded by rock. we find a lot of dinosaur bones, fossils, and that is really intriguing site is for a long time. another thing we also find in the morrison is a mineral, a rock called incarnate type, it contains three different elements and contains radium, it is radioactive and used by marie curie to help fight cancer and it also contains a mineral used to strengthen steel. up to world war ii and during world war ii, it was of extreme value. andlso contains uranium uranium, as we know, is one of the best sources for atomic power and atomic weapons. >> colorado congressman wayne aspinall was largely responsible for this area is agricultural development through his body legislation. >> he fought the battle to reserve water for western that we by making sure
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got our fair share. how did he do that? well, beginning in his state career and then going on to his federal career, he climbed up the ladder of seniority and was i think moreise power than you might normally have. certainly in the united states congress, where he was able to make sure colorado and western colorado would be treated fairly in any divisions of water, his thet major success was passage of the colorado river storage project in 1956. programs fromour grand junction saturday at 7:00 p.m. eastern on c-span [applause] the tv.-- on c-span2 here is a look at what is
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ahead on c-span in about one hour or so. vilsack will speak at the center for american progress to talk about child nutrition programs. you will be able to watch his remarks until then, it is a discussion on immigration policy. the american bar association hosted this panel. it featured dozens of legal and national security analysts. this portion is about one hour. >> get situated. we have picked the topic after lunch that is totally noncontroversial, which is the united states immigration policy and laws. as we move towards this


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