candidate service as a soldier, legislator and statesman. watch live at 3:00 p.m. eastern here on c-span 3. c-span's washington journal, live every day with news and policy issues that impact you. coming up wednesday morning, democratic congressman bobby scott of virginia and republican congressman jason lewis of minnesota discuss bipartisan legislation they've introduced to reform the criminal justice system. and we're live in raleigh, north carolina with attorney general josh stein will be our guest aboard the bus. be sure to watch c-span's washington journal live at 7:00 eastern wednesday morning. food and drug administration scott gottlieb announced a new

process to approve new drugs. this is 35 minutes. >> thanks a lot. thanks for the opportunity to be here today. we had a dialogue over many years, and i appreciate the relationship we had talking about policy issues. i wrote a subsequent article saying what's taking you so long, josh. so i've been waiting for him to return the favor, and i just got a copy of it. so i appreciate it very much. thanks for the opportunity to be here today. thanks john hopkins for hoestin this, and pew for providing space. transparency can be a powerful

tool for innovation. new platforms for visualizing large complex data sets present important new opportunities for the research and development community for regulators and for clinicians. it allows people to harness data to advance the safe and effective and efficient development for medical needs and to communicate information supporting regulatory decision making in ways that engender greater public confidence in those decisions. the fda's goal is to advance transparency without reducing incentives to innovate. this can be accomplished by meeting our statutory responsibility for protecting confidential, commercial information, trade secrets and private data while disseminating

more information gnat supports and informs more regulatory decisions and patient care when ones products are approved for use. with these safe guards in place, transparency is a tide that can lend to all boats. to identify areas requiring additional post-market research and to generate the data necessary to meet fda's gold standard for assuring product safety and efficacy. and fda's modern transparency strategy began with the doctor's leadership at the transparency task force and subsequent recommendations in 2010. and we continue to expand fda's commitment to transparency today. but stakeholders continue to request greater transparency in the drug approval process and greater access to usable information on those approvals. and i believe they're right. we need to look for ways to be

more forthcoming with the information we have within the boundaries off our statute and your obligations. much of our information concerns products that are integral to people's family, the food they eat, the pets they love, and the medicines they use. people deserve to have as much information about these products as we can possibly eliabreliabl provide. we have a public health obligation to keep people fully informed of the safety and benefits of the products they use to improve their lives, and we're taking some new steps to fulfill these commitments. today i'm announcing two initiatives i hope will provide greater transparency and efficiency in review. together these initiatives will

give patients and researchers new insight into the data and decision making behind the approval process. first beginning this month our center for drugs is launching a pilot to evaluate whether disclosing certain summaries of clinical information called clinical study reports or csr's following the approval of new drug applications can improve the public's access to drug information. to start the pilot can include up to nine drug applications to sponsor and volunteers to participate. the pilot is going to test the process for selecting, redacting, and posting csr information on a public website drugs@fda that fda uses to provide approval information on new drugs. applications to be included in the pilot will be selected based on criteria including their novelty, scientific interest and

whether or not the drug is new molecul molecular entity. a csr is portion of a drug file related to a clinical trial that contains a detailed summary on the methods and results of the trial. csr is a scientific document addressing the efficacy and safety. we suggest making it available after approval will provide stakeholders on more information on clinical evidence fda's decision making process. it can help those interested in that detailed data. for example, academic researchers who want to study a specific drug and need access to summaries of bottom line information in order to inform that research. this will be the first time that fda is proactively disclosing the reports to the public. it will include study report bodies, the study protocol and

amendments and statistical analsy plans for each pivotal study. clinical summary reports will be posted on a new fda web page describing the program in addition to new drugs appearing on the fda. with the drug information alongside the time it's approved. while fda does provide access to drug application action packages that include all discipline reviews after a drug has been approved, thia tilt is sometimes limited by their complexity. while these new summaries provide a window into the basis for our approval decisions, they're packaged into a format that at times can make a difficult to decipher the key clinical evidence. we believe by posting key

portions of the csrs publicly in response will both comply with our existing obligations to make sure the summary material is publicly available on approval and make that material significantly more practical and use it friendly for key collaborators. at the same time we're taking another new step to make it easier for patients and clinicians to track new therapies as they advance through product development and different regulatory check points. right now the job of privately and publicly supported trials arn the world register at the national institute of health's database. this website provides easy access to information on studies on a wide range of diseases and conditions. ultimately some of these new conditions provide new drugs.

these trial drugs are often of interest to patient research communities. yet tracking a specific trial in its active state on clinical tria tria trials.gov can be very challenging at times. in response we plan to increase transparency around clinical research posted on clinical tria trials.gov will use to link fda communications about those specific drugs including in the product labeling and even in our advisory committee materials. this new number will be called an mct number. members of the patient and academic and scientific communities can use this number to follow the development through key regulatory mile stones. this number will make a easier to coordinate the listings to

fda communications about specific drugs including product labeling and even our advisory committee materials. including this number on fda's materials could greatly benefit all those interested in following the progress of specific clinical research. we're also continuing to explore whether it would be possible as we discussed at the outset under existing statutory authority with changing the governoring law to release additional information from complete response letters related to the efficacy and safety that could have significant public health value. releasing all csrs would be challenging with the likelihood we would continue to redact certain proprietary information from these letters. for example, many letters primarily relate to manufacturing shortcomings with new drug applics that are eventually resolved. but some of the letters do contain information that could

be directly relevant to patient. we're evaluating whether there's a subset in response letters, whether there's public health reasons to redact and release these letters. this could include letters that have safety findings that could help with the profile of already marketed drugs. releasing this information could enhance patient safety by reducing the number of potentially futile trials and spare patients exposure to potential risks without the likely benefit. we're committed to enhancing transparency throughout the work we do at fda. this is especially true when it comes to these efforts to have the potential to foster further research and discovery across the scientific community and clinical care. and we'll continue to seek additional opportunities to foster greater access to key scientific information and clarity around regulatory decisions wherever appropriate.

the powerful synergy between high power data sets has been effect for over 50 years, long before the advent of the desktop commuter. arguably two of the most important and impactful initiatives undertaken in the post-war era. in the launching of the study in 1948 and the surgeon general's report on smoking in 1964. over the ensuing decades subsequent analysis new generations of participants, spouses and children helped revolutionize our understanding of the risk factors of card

cardiovascular disease. the combination of better science and a better informed public led to a dramatic decline in mortality from all forms of cardiovascular disease including heart stroke, and coronary disease. the national institute of health reports that the death rate from the disease peaked just before the surgeon general's 1964 report on smoking and started falling. from 1968 to 2010 the age of death rate from the disease has declined by nearly 70%. today the study has expanded to include bio specimen banks. technology no matter how powerful is always just a tool.

it's our commitment to the transparent, responsible and science based use of those tools that gives them their potential to save and improve lives and build many more practical framingham studies for the 21st century based on a growing number of open sourced data tools and data sets. to reach their potential open data, and advance trial designs will all have to be validated through transparent processes that gain the trust of stakeholders. not by inventing new values but by recommitting ourselves to fda's centurylong commitment to its gold standard for evaluating effectiveness and safety. thanks a lot. thanks for your time today. look forward to your questions.

>> so i think people should have index cards. and if you can put questions down there and just raise your hand with them, we'll collect them. and i'll get them and we'll be able to ask questions. i'm going to come over and get started. mr. gottlieb, thank you very much. it did not disappoint with some transparency of the news at the agency. i want to start with a general question and start with some specifics about the announcements that you made. some people think of fda as a black box, that the agency sits out there like a white oak and makes decisions. and that's the important thing.

how the decisions get made, not so important. how would you think of, what's the metaphor you would like people to think of fda? >> i think it's very important that people understand exactly how the decisions get made and the processes that inform how we go about analyzing data, making decisions and also the values that inform those decisions. because ultimately a lot of the decisions that we do make are policy calls. and you can scientifically define the risks and the benefits of a product related decision. but ultimately a decision on how you balance those risks against those benefits involves a lot of policy decision making. and i think we need to be more transparent and engage the public on how we ultimately do that balancing. and i think we do do that. i think we do a good job of

that, and it's gotten a lot better over time. it provides a lot of transparency. we hold a lot of public workshops. we engage the public. we try to post online guidance and through other vehicles detailed documentation on how we make those decisions. and there's more of an impetus i think than ever before for the career staff to engage the public as much as possible through speaking engagements and other kinds of workshops to articulate our philosophy. i think ultimately, though, one of the challenges we face is that the complexity of what the agency does sometimes makes it hard to access. and i think that's something we struggle with historically. and that's something that i think we're trying to address through, for example, engaging patients more actively in the mechanics of the review process, trying to elevate patient

involvement in some of the regulatory decision making on the advisory committees. but i think that's been a historical challenge. i don't think it's just a question of making more information available. and i think the issue is how do we sort of make the philosophy and that policy balancing more transparent. >> yeah, let me ask you about the csr announcement you just made, the complete study reports. these are pretty complex documents. they're big. they have a lot of data in them. what are the kinds of benefits do you see from this pilot program? >> i think, first of all, it's going to make the review process more efficient. and we're doing it as a pilot program, but we think over time a lot of manufacturers are going to see the inherent value of making these csr reports public. because a lot of these memos right now are cutting and posting from the review documents and the bottom line

clinical information on forms to review isn't very accessible on those documents. i think when we're able to link to the csrs, the bottom line information on the csr is going to make the bottom line review document much more efficient, more accessible and could provide a lot of efficiency to the review document itself. this is part of the broader reforms under way. we're trying to come up with a more consolidated review memo. a lot of documents made public at the time of product approval are different documents related to the different consultative aspects of the review process, so different components of the agency and the consultants, so you have multiple memos. we want to work in the direction of coming up with a more consolidated memo so you have a more team based approach and everyone's working together to

consolidate that explains the agency's filus fae where you have one document where you can see the consensus. having the csr available, being able to link to it makes it much more feasible to come up with a consolidated document. just to finish, so that's one virtue of doing it. and that's sort of an efficiency. but the other is that the csr is one level above patient specific data. it is more bottom line data, and these can run hundreds of pages. i think that having it available is going to allow third parties to do more analytics around our decision making and do more analysis. and so you'll have research better informed by actual data. right now some drug companies release summaries of the csr,

but they don't actually release the csr, so this would be the whole csr. >> so two questions. one, should people be worried patient level data will be disclosed or there'll be approaches to making sure there's not a confidentiality -- >> so this isn't patient level data, and we're very mindful to make sure, and we're going to have to go through it to make sure there's no way to -- we saw that experiment with the kol, the way they looked at searches and were able to go to people's homes and who were doing searches by decoupling the data. that's a concern, obviously. you want to make sure the csr data that someone who is a researcher can't somehow correlate in a way they can get down to patient level data.

and so that's one of the things we're very focused on. we feel pretty confident that that's not going to be the case. the csrs are released, i don't know all the specifics around it. in europe the csrs are available but they're not readily available. they're available on a proprietary database i think that's maintain bide the ema where certain researchers can get access to it. so there is some precedent for allowing certain qualified researchers access to the csrs. we want to do it on a more whole sale fashion, but starting as a pilot we're working through more legal issues. we're reaching out to companies with products we think this could be particularly relevant and could allow for a more efficient process, and then we'll see where it goes.

i do believe, whether making this voluntary or mandatory going forward, we'll work it out with the pilot. but i am making a pitch, and i made it at the outset for the fact i think that when you look at what ware able to do in making the csr publicly available in terms of how it changes the nature of how the review memo gets to arrive and changes how the review process works, i think people are going to see it creates a lot of efficiencies. because the creation of that -- those reviewer memos and how people formulate their opinions does drive a lot of the structure of how the review process itself works. to the extent you can develop a more consolidated review opinion that more easily surfaces areas of agreement, disagreement, it's all in one document, it lends itself to a more team based approach, which is the direction

i want to move in which i've already talked about. where people are part of a dawn solidated team, the memo itself needs to be a team-based effort. >> so in theory that would encourage more manufacturers to participate. >> we think it's going to create a more efficient review process with greater input from people who have expertise in different disciplines. they're part of consolidated review team, and we think manufacturers are going to recognize that's more efficient. >> i see. and that would encourage. >> yes. >> and moving onto the clinical trial number, which is another recommendation that's in the blueprint for transparency, so much appreciated. one question there. it has been the case that some researchers sometimes don't register their trial appropriately or don't, you know, do the linkages themselves. so obviously there's one aspect

of it which is just getting the information to people so they can see where there's a trial that's been registered with nih so they can look it up. companies that don't do that, have you thought if they don't provide the information -- i don't mean companies, researchers actually, don't tell you which trial they are correctly or maybe they tell you some and not others. do you see this could be an area that lends itself to some expectations that fda has? >> we'll have to see how it goes. in terms of creating requirements, we'd have to do that through some kind of common process. i think we're going to see how this works, and then there's questions of, you know, some of the issues that you're alluding

to would be issues we would address. >> what about devices? do these have implications for devices or right now are you starting with drugs? >> right now we're starting with drugs. i'm not sure what the analogous piece would be to the device side. it's different. but the broader goals on the medical device side are similar in that we're trying to move towards a more team-based approach in the review process. and to do that we want to have more consolidated types of communications, so it's easier to read the narrative of what the agency's thinking was. that is challenge right now. when you look at a review package in terms of looking at what the different opinions were, you have to read a lot of different memos. and we think sort of a more consolidated narrative will lend itself to more transparency

among the agency considerations. >> and it sounds like what you're saying it's clearer for people to understand still allows room for disagreement. >> oh, absolutely. >> but allows to connect to some of the original data in a way that's different from now. instead of saying here's five different memos and three data sets, you're saying here's the memo and here's the data that will be referring, so it's a bigger approach. >> right now if you look at the memos they're long, but a lot of it is cutting and posting information out of the package that the sponsors provided. and so sometimes it's not very accessible. if you can provide a memo that focuses more on agencies thinking and provides linkages to the underlying data because you now have this electronic available and that document pertain tuesday the data. so you're looking at the

analytics and their bottom line conclusions. that's a more accessible document for lay people because it's more focused on the agency's philosophy. and i think it's more accessible for researchers because now they can link back to the detailed data sets we use to derive some of the conclusions. >> got it. last couple of questions on complete response letters. you knew that would come up. in 2010 a wise person wrote the public zurdeserves to see the agency's decisions. the complete response letters were ways they couldn't improve the product. and that could include some proprietary manufacturing issues but could include challenges with the safety and effective of the product. and there's been some research by fda showing that while companies will release some

information why their drugs weren't approved, they often don't release other information like some safety concerns. you made a comment you're most focused on the public safety issue. can you answer on the issue of where you see public play coming into -- for example, if someone says if the drug's never going to be approved at all why is it necessary to let people know why it failed? >> well, it's hard to draw a conclusion that a drug's never going to be approved at all. if you look at the statistics most drugs that make it to the mba state are eventually approved. i think it's upwards of 90%. you know it as well as i do. sometimes the basis for the

decision issue of complete response will be, you know, a safety related issue where the agency is asking for more information about a drug because of a particular concern that it has where that concern might be relevant to similar drugs in the category, in the class. and that's highly relevant. or, you know, information that can inform different classes of drugs where the agency sort of has an overall philosophy about a therapeutic space or drug interactions. so those are situations where i think that making that information available to inform clinical practice and public health. i think what we don't want to do, and you've been around this issue and the challenges have been the resource intensive nature of redacting and making these letters public. and i want to see if there's a way to make an easy cut -- not easy but something that can be

implemented as a matter of policy between letters that have that kind of public health value and those that have less value, might be more interesting to investment but not have public value. we have someone working right now where we've pulled 100 crls, and we have someone going through them to see if there are identifiable things. when you do a clear response letter they'll have different issues, but there's only two that are driving decisions. so is there a way we can kind of extract it or a way to identify it in this part of the letter or contain this kind of information, we're going to make

those public to start. and that's where we're looking right now. i think there might be a way to get there, to find the subset that's really important from a public health standpoint. i'm just speculating. places where we're asking for an additional clinical trial related to safety issue or a drug where there's other marketed drugs that have some relevancy. that might be an easily identifiable subset. those are the things we want to look at. what i don't want it to become, i don't want to use this kind of initiative where, you know, i'm going to be eating a lot of resources to redact these documents just for purposes of market efficiency. this has to have some bottom line applications. >> so two last questions. this is really interesting. one more on the complete response letters. when we've thought about the public health value, complete

response letters, those are for drugs already on the market for unapproved uses is pretty high up. because sometimes there's a llt bit of data out there and maybe some intense interest on a company's part for a drug to be used, but when the chips are down and fda is really doing its review they don't see evidence of the effectiveness. and because that drug is on the market and because there may be even be articles in the medical they're saying, hey, why not try this drug for this reason, the fda's view of that could be quite influential coming from the perspective the fda is the neutral agency to decide whether it's a strike. can you see the potential health value there? >> crls related to already marketed drugs. yeah, i would make two points.

in terms of making a cut around that, that's a pretty small subset. so we might be trying to loo lo little farther than that. and that information generally is made public. now, that said, there could be a lot of value doing that. so i'm not by any means saying i don't think that's a good idea. i think we're trying to look a little more broadly than that, a little bit of a broader subset. and that could be certainly scoped into what we do. but a subset also where the information might not diffuse as readily but for our ability to release the crls. that could certainly be part of a policy. >> one of your predecessors, he really lights up on the issue of transparency is the agency correcting on this information out there.

and you just alluded to this possibility. and i think he was incredibly frustrated when people would say things he knew not to be true and he felt sort of policies and procedures at the agency made it really hard for him to just go out there, was not it case. do you feel this is an area the agency may be willing to jump in depending on the -- >> i feel that frustration every day. there's usually about 15 or 20 tweets that i craft that my media staff stopped me from sending out. but that's a challenge. i mean it's a challenge for the agency because we have to be very careful about disclosure information. and to appreciate that, in order to do our job we get access to a lot of confidential information and trade secret information. and people at the agency take that responsibility very seriously.

they take it seriously because there's criminal sanction that attaches to the inadvertent disclosure information. but they know how important it is to do be able to do that public health job, be confident in information sharing at the agency. so there's a concern that if we demonstrate that we are either, you know, not careful to safeguarding information or will to pursue our own policy prerogatives, sort of change our posture easily with respect to how we handle information, it could make sponsors more reluctant to disclose their information and to fight us on it when we need it. and that could be a real challenge. i come up against that all the time, where, you know, sort of for example with the shortage situation, we want to disclose more information about what we're seeing in the marketplace and inpendimpending shortages. but at the same time we need to

be careful. so it's a constant challenge. the problems with sort of correcting stuff in the market is oftentimes to correct stuff you have to put out stuff. and sometimes the stuff you're putting out is also confidential information. you know, we do make referrals, as you know. when it's in the context of, you know, security markets. but that's not the same as trying to correct things in realtime on a public health basis. >> well, thank you so much. i'm just going to make a prediction, which is what you've announced today is going to be very well received. that transparency has a constituency that really cuts across politics. and i think you -- people can see the, you know, or think about what potential barriers are out there. but i i think you're going to and i hope it develops more

momentum and it will lead to more and your ability to feel more confident about doing more things that help people understand the agency, that help others develop more effective medical products based on information the agency has. so congratulations on the steps you're taking today. thanks so much for coming today and your leadership. >> thanks a lot. thanks for your input. the forum on transparency continuewise the discussion on the effective transparency on investors. during this portion of the event we hear from jonathan left, chair of the deerfield institute. it's 25 minutes. >> thank you so much for coming. chairman of the deerfield institute, he joined this company in