Senate appropriations subcommittee. This is 40 minutes. Hearing will come to order. I would like to thank the members for being here. Ranking member merkley and certainly want to welcome fda administrator gottlieb. Thank you for being here this morning, we appreciate it. Todays hearing will focus on the food and drug administrations fiscal year 2018 budget request. Thank you again for being here, dr. Gottlieb and obviously this is a Good Opportunity to talk about fdas priorities for the upcoming year. Congratulations on your confirmation. We certainly want to welcome you in your first appearance before the subcommittee and we look forward to working with you. The agency you head has authority over approximately 20 cents of every dollar spent in america. Americans expect that the food they eat and the drugs they take will be safe and effective. Fdas reach is vast. The agency has authority over more than 300,000 foreign establishments, and 185,000 domestic establishments ranging from Food Processing plants, to facilities that manufacture lifesaving medications. In addition to facilities themselves, the fda is tasked with the regulatory responsibility of individual products. And delivering these regulatory responsibilities your privatesector partners expect transparency and certainty from the fda. When i speak to Small Businesses, and ag producers in north dakota, their overwhelming concerns is that often Overly Burdensome Regulations coming out of washington, d. C. Can stifle innovation, and hinder their ability to create jobs. While we all support the fdas mission, we must also be mindful of these concerns. I believe the fda must avoid the trappings of one size fits all solutions and i urge you and your staff to take a commonsense approach in regard to the budget request itself. Im concerned that this request relies on a significant increase in user fees. That is not feasible, and unlikely to gain congressional approval. Im concerned that the proposed cuts to Budget Authority may negatively impact food safety programs and slow the agencys important work on drugs and medical devices. That being said, dr. Gottlieb, i also recognize that these decisions were made before you were confirmed, so i hope you will pledge to work with congress to insure that fda has the resources necessary to meet its critical mission. We have many other issues to cover. So at this point i will turn to senator merkley for his opening comments. Thank you, im going to keep this very brief. Because were hoping to hear your testimony and have a round of questions before we go to the vote at 11 00. The department you head covers products that constitute 20 of what consumers spend. Both in food and drugs, it makes that work very important. And this budget before us very important on everything from scientific research, support for state and local health organizations, blood safety work. Medical device, postmarket surveillance, medical product exams and so much more. So i look forward to hearing your thoughts on the budget. And getting to our inquiries. Thank you and congratulations. I would suggest at this point we go right to dr. Gottliebs statement so we can come right back for questions and then any Opening Statements as well. Other members may have. Dr. Gottlieb . Thank you, mr. Chairman and mr. Ranking member and members of the subcommittee. I appreciate the opportunity to testify today regarding the president s budget. Ive talked to you in the past about the steps that fda is taking on the generic drug side to try to bring more lowcost opportunities to patients when it comes to new drugs and improve access. I want to briefly touch on some things were going to be doing on the new drug side. The most tangible way were going to reduce Health Care Costs is by finding better treatments for a lot of costly diseases. And towards these ends, well be announcing soon a medical Innovation Development plan that will include a broad range of steps to make sure that our own regulatory tools and policies are designed to facilitate the development of potentially breakthrough new treatments. One area of focus of this plan is is going to be on targeted drugs, especially those that affect Rare Diseases or diseases for which there is no effective therapy. Among other things, fda will be updating various guidance documents on the kinds of Drug Development techniques that facilitate the development of drug therapies, guidance on Clinical Trial strategies to improve efficiency and adaptive trial designs to evaluate safety and effectiveness, well be taking a fresh look at policies that support innovation to allow drugs to be targeted only to those patients who most likely to benefit from the medicine and take a broad range of new steps. Among these new actions, well be issuing a new document within the next section months on the clinical evaluation of targeted therapies for rare disease subsets. This policy will address targeted drugs and how we can simplify the development of drugs where diseases all have a similar genetic fingerprint. Even if they have a slightly different clinical expression. One example is the cancer where a drug target as particular molecular subsit of cancer, regardless of where the tumor arises, well clarify where we can give a broad approval to a drug in similar kinds of cancers which are not particular to the tumor being in one specific tissue or organ. In other cases rare subsets may be grouped by lab testing to be studied in a single Clinical Trial this medicine is more common as we understand the genetic basis of disease. Our new policy will describe when we will approach drug review, less by how a disease is expressed and more by how it is expressed by a commonly set of driven factors, rare and orphan diseases, right now we have a backlog of 200 orphan Drug Designation requests where we havent responded to sponsors and whether the drug will receive an orphan Drug Designation from the fda. Were committing that 90 days well eliminate this backlog of requests and provide an answer back to the sponsors. To help eliminate the backlog weve created a special orphan designation s. W. A. T. Team and well never again develop a backlog. Going forward were committing that every orphan drug application will receive a response from the fda within 90 days, to enable more Efficient Reviews and timely responses to sponsors who are also implementing a new streamlined orphan designation review template. These are some things were working on. I look forward to discussing with you how fdas budget and this committees can support all agencies key priorities, including food safety and enable consumers to improve their lives, thanks a lot. Thank you. And well start with the questioning. I have concerns about the administrations proposal to essentially double user fees. And it doesnt appear that the Health Committee will move forward with that proposal and instead congress will likely pass the previously negotiated user fee so my question is, based on the fy 17 appropriation, are you confident that youre going to be able to meet the Program Needs . Based on your current appropriate yated level . Fy 17. Senator, thanks for your question. The bottom line is we can always do more with more. When it comes to the resources that the agency has. Im confident that we have been able to be efficient in everything we do. I think theres still places that we can look within the agency to try to improve our operational efficiency. We recently made an announcement with respect to a realignment when it comes to the field activities, i think were going to continue to look for places to improve our operational efficiency. We appreciate very much the resources that weve gotten from this committee. In particular, the resources on the food safety side and the resources weve gotten under that. I think its dramatically improved the stature and the base of resources for food safety. Its a very Different Agency today than the one that i left ten years ago in that regard. You have about 1,000 vacancies, hiring freeze has been lifted for your agency. Are you moving forward and where are you in that process of filling positions . Thanks for the question. Thats right. We negotiated the lifting of the hiring freeze about two weeks ago i believe. And were starting to move forward with filling those vacancies. I put out a notification recently to the office of the commissioner as well as the different senators in terms of process for moving forward. Theres already activity with filling some of those existing open slots. What is the fdas role in addressing the Opioid Crisis . And what are you doing . Well its multifaceted. This is in my view the biggest challenge facing the agency. I think one of the biggest Public Health crises facing this country. Theres a lot of different components where the fda is going to play a role. You look at medically assisted therapy where the agency plays an Important Role trying to address the current addiction problem. Trying to get better opioids on the market, that are more tamperresistant. We can play a particularly Important Role is on the new addiction aspects of this crisis. We know that most people who are going to become addicted to opioids are first expokesed to opioid drugs in the clinical setting through legitimate prescription. Certain pesage of patients who are exposed to opioids in the clinical setting will go on to develop an addiction. I think its incumbent upon all of us to make sure that only properly indicated patients are being prescribed opioids and when they are prescribed them, theyre prescribed them for a duration comports with the clinical circumstance for which the prescription was writ innocent first place there are things that the fda can do to try to address these aspects of the problem. Thats going to be a particular area of focus of ours. S we recently developed a Steering Committee made up of all the Center Leadership and senior clinicians within the agency to look at trying to see how we can think differently about this problem. The other place where ive tried to focus some policymaking attention is looking at the risk in the illicit setting we traditionally have looked at the risk of illicit use as a component of how we evaluate the risk and benefit of opioids overall. But i twoont make sure we have a proper framework in place for doing this. And were looking not just at the risks associated with these drugs in their labeled indication, but also the risks associated with how they might be abused and misused and diverted and used illicitly. Weve recently taken an action, that baked into it, a consideration of how the drug was being used in the illicit setting. What about approval of drugs that actually help wean people off some of the opioids . I know theres development in this area. Right. This is the medically assisted therapy. We need to continue to develop good drugs in this area. One of the challenges, though, has been reimbursement. While its outside of my mandate, getting these drugs to patients is an important step as well. So were looking at things we can do to help facilitate the development of Clinical Trials that move these drugs into different clinical settings to see how we might better study them in realworld settings and hopefully well have more to say on that soon. Last week the fda announced a delay for compliance to the Nutrition Facts Panel regulations and i know youre limited on what can you say until its published in the federal register. But given my comment regarding one size fits all solutions, will fda use a commonsense approach towards those nutrition regulations . Thanks a lot. I am confident we will, senator. We announced the delay in part to provide Additional Guidance to sponsors on how to interpret aspects of the new nutrition facts label. This is a timelimited delay. This is not a suspension of the regulation and were not reopening the regulation. We are just using this time to develop Additional Guidance documents that will be issuing to help inform how people can comply with the new, with the new labelling. Senator merkley . Thank you, mr. Chairman. And welcome, dr. Gottlieb. And the question first question i have for you is, is a number of news reports have discussed the administrations directive to federal agencies, not to respond to requests from minority members of the senate. That is completely contrary to the very long and positive bipartisan history, both on this subcommittee and in our dealings with the fda under both republicans and democrats. I think dialogue with the executive branch for all members results in stronger bills, more likely to have support. And clearly benefits the fda and the American Public to have that dialogue. Can we count on you to be engaged in that dialogue and respond to inquiries and requests for the majority and minority . Absolutely, senator, i responded to all the requests for new information that i got during my confirmation process from the minority. I am prioritizing timely responses equally from both the majority and the minority. We have reached out to many offices, including your own. I had the pleasure to meet with you twice. And so we will not pick sides in how we, how we provide information to congress. I respect congress and were going to make sure we, were providing you the information you need. Thank you very much. And your outreach has been appreciated and i appreciate your commitment to continuing on that course. During your confirmation process, you described the staggering human consequences of opioid addiction and characterized that epidemic as the biggest crisis facing the agency. Now we have prediction that the trump, the cdc engaged in various other experts, that the trump care bill coming out of the house, we dont know what version well see in the senate yet, would cut billions of dollars from treatment for Mental Health and Substance Abuse disorders. And also the millions of people would lose their insurance which makes very likely that they would be seeking medical care in the first place. Were concerned that this, this budget would damage the ability to take on opioid addiction with its enormous daily toll in lives. As someone who is leading the charge on this epidemic, who has presented it as a top priority, do you have concerns about the loss of access to health care by millions of americans, if this bill is enacted . Senator, im very focused on what were doing at fda right now. To address this crisis. As i know you can, you appreciate. And youre going to continue to see a series of activity out of the agency to address this crisis in you know, different ways that hopefully start moving us to a posture where were getting ahead of the problem instead of always being one step behind. I have not focused a lot of attention on the various legislation moving through respect to the Affordable Care act. Im focused on what im doing at the fda which is more than a fulltime job. Which i know can you appreciate and well continue to talk to you about those priorities. There are 13 senator who is are holding a series of closed meetings to work to prepare a version of the bill that would come before the senate probably next week. Have those individuals or as a group, brought you into get your consultation and insights on the Opioid Epidemic . Ive had, i dont know what group youre talking about. But i have had conversations with individual members about the Opioid Epidemic. I met with probably 60 members in the runup to my confirmation process. And i would say that this issue came up in most of those meetings. And since ive been in this position, weve taken a lot of additional meetings with both democratic and republican members in the house and the senate and this issue comes up a lot. So i have consulted widely on this issue. Including with you, senator. And appreciate all the dialogue ive had with congress. The group im referring to are the 13 republican senators who have been delegated the authority to prepare a bill to move out to the floor, one that will have no public input. Thats the group. Has that group asked you to come and share your expertise on this issue . Ive had no dialogue with any group working on legislation as a group, no. Well i imagine just knowing your background that you probably share my concerns, that such issues get the insight from experts and also that the public has a chance to weigh in so that we get in this we the people, republic, a full opportunity to make sure we get kind of policy right, if you will. That hassan impact on so many people. Thank you. Senator. Thank you. Senator collins. Thank you, mr. Chairman. Mr. Chairman, i have an Opening Statement that i would request be submitted for the record. Without objection. Thank you. Doctor, we have previously discussed the need for improved Health Care Provider education with regard to the prescribing of opioids. Medicaid beneficiaries are described pain relievers at a higher rate than those with other sources of insurance and they also not surprisingly, given the higher rate, have a higher risk of overdose from prescription opioids, heroin, and fentanyl. What opportunities do you see for greater collaboration among the fda, cms, state medicaid directors, medical societies, and other parties in order to address this problem of appropriate prescribing of opioids . Right. I appreciate the question, senator, i would also add dea to that, because there might be things we can could had in conjunction with our partners at the justice department. As part of the Steering Committee, were currently having discussions about what steps we can take to improve provider education and maybe take a look at packaging as well as a way to help make sure prescriptions