Bill frist and had the opportunity to watch firsthand governor alexander work was a pretty rowdy Democratic Legislature and tennessee that he came forth with education reform, with infrastructure, investments, industrial recruitment. I think regular order is back in the senate and will be a better country for that. Is fortune is going have a good partner in the house, someone for the good fortune to settle the Energy Commerce committee with, diana degette, and diane reminds me, when i think of her i think of a story my grandmother may read the of the detention a good. I think i can, i think i can. I know i can. Thats diana. Come forth with a major bipartisan vote in the house. Senator alexander come to the good partner and diana, tell us whats going on with you. [applause] thanks, part. Im so happy to be here. They dont invite us to come over from the house very often so its really a treat for us. Im so happy, lamar, to hear your reflections on some of these issues, because to some of you know, chairman upton and i worked really hard. When youre talking about all the hearings we had, we had hearings, we have roundtables, we had white papers, we had union. We worked for about 18 months trying to get consensus, and many other people in this room attended multiple of those hearings. So you know what were talking about. I was really, really pleased when i found out that senator freshman congressman gordon were heading out this effort because they really found meaning areas where we could make substantial updates to bring new treatments and cures to patients. The good news is that this is similar to the consensus chairman upton and i found in the house, and i think what it shows is that there really is a large amount of consensus about what we need to do to the regulatory process but also the resources that we need to bring to bear on expediting Biomedical Research at the nih and bring it to approval at the fda. Across the medical and research fields, as fred and i found, experts from different backgrounds and all different sectors really agreed about the improvements that we can make it todays report that was released, i havent read it in detail yet to read the executive summary, and it offers us a lot of the same proposals that echoes a lot of the things that we heard in the house and then acted on in h. R. Six. I just want to highlight a few of the similarities in this report to what weve seen in our house investigation. For example, in the report that our recommendations to more effectively include the Patient Perspective throughout the drug and Device Development process. H. R. Six has several provisions that strongly echo this policy. In addition there are several recommendations in the report to strengthen publicprivate partnerships. This is something again that we highlighted in the 21st century. Further there is a recommendation to accelerate the development and approval of antibiotics. This is a provision thats been a little bit controversial in the press, especially since h. R. Six past the house. But when crafted in a very careful way, and in hand with the agency giving us technical assistance, we know that this is something really important, specifically to do with those cases of antibiotic resistance. This is also included in h. R. Six. I could go on and on but you be glad to know im not going to. Im under the house rules today, but what i want to say is that i think that we can find a great deal of this consensus. One thing i do want to talk about very briefly is the importance of new resources for our flagship medical research and development institution. Fred and i as part of our quest went all around the country. I was in san francisco, michigan, fred was in denver. We were all over the country at biomedical institutions. And whether wer we are talkingt the fda or the nih, they are were given multiple priorities in a very stifling budget climate and the need to have the ability to carry out the ideas that are circling around right now. I want to keep you an example. We were having one of our bipartisan roundtables over in energy and commerce room, and Francis Collins turned to me and he whispered, he said, diane, if we had had full funding for the nih and we had not been under the budget cut, then we probably wouldve had a vaccine for ebola i know. And i said, francis, youve got to tell the whole crew. He did. He said it on the record. What he meant was because of a lot of the budget constraints, what happened was many worthwhile programs Like Research on ebola vaccines were put on the back burner in favor of diseases that are naturally in this case seemed to be much more of a threat. So really with all of the great breakthroughs we have going on now, we need to also be able to research the innovation. As most of you know, we passed 21st century and house at the beginning of the month. We passed it 34470. You cant pass a journal without by the way. So we were really gratified by our efforts but we know, and we hope, that this bill will just be a resource to the senate as the senate embarks upon its very important investigation this fall. Im also really encouraged that our efforts have been joined by serious discussions and working groups at the senate h. E. L. P. Committee. The Bipartisan Policy Center initiative really adds the substance expert analysis and further momentum for the conversation. We have an opportunity to make important updates and reforms to our Biomedical Research and development systems. After the last century of remarkable medical discoveries, we want to make sure that in the 21st century the pace of breakthroughs, treatment and cures accelerate to meet the challenges of our time. Some of you know fred and i did traveled around together or 18 months, and so i would be remiss if i didnt make the fred upton point right now, which is the appropriation under 21st century cures begins in fiscal year 2016, so thats october 1. Anyway, we are glad to be here. Were glad to be of any resources that we can be. We are thrilled the senate is taking up, and we are thrilled to be here at this update. Thank you very much. [applause] thank you very much. Good afternoon to my name is janet. Im glad you all made it to be. We had a bit of a shower coming into our event. I served as director of Health Innovation of the Bipartisan Policy Center. On behalf of bpc, welcome all of you today. So the going to spend all a bit of time going through the report and you should have it if you walked in. And with that we are delighted to you from our cochairs, and if i could ask you to come to the table and hard by the committee. While theyre doing some going to take just a minute to introduce each of them. You able to meet with senator bill frist the open us up to date. Is a nationally recognized health and Lung Transplant surgeon. Imagine the also a former Senate Majority leader. On his left the also, this is congressman gordon, representative gordon, former congressman who served as the chairman of the help committee on science and technology. During his tenure in congress focus a great deal of his time on improving americas competitiveness. And weve got a lot of that can report and well hear about that during our panel discussion. And lets see. All the way to your, lets see, to your very left, youre very right im delighted to introduce patrick soonshiong, just look in your from los angeles, hes a physician, surgeon and scientist and has pioneered for both diabetes and cancer, published over 100 scientific papers and has over 95 issued patents a groundbreaking advancements standing newt of fields. And then final im delighted here on my left to introduce mark boutin who ive known for a long time. Mark is the chief executive officer of it or position called the National Health council, a really amazing organization. Brings together, provides a unified voice for more than 1339 people in our country, nations and individuals, those who are sick and those who have disabilities. And has been a real strong support for medical innovation. Im sure that our third Advisory Committee member, mark mcclellan, who serves as a senior fellow and director of Health Care Innovation and got initiatives at the center for Health Policy at the Brookings Institution couldnt be with us in person today. So im going to move over here so we can get into the panel discussion. [inaudible] there we go. Our first question, i thought senator, folks have just seen the executive summary, the longer letter is on the website, pretty long, 80 pages. I wonder if my able to to folks in the room sort of a broadbrush around whats in the report. Janet, thank you. Again the website makes it available but for those of you who are here, the report is hot off the press. The final finishing touches this morning. Easily digestible but let me point out a couple of things. The first area of the report is focused on, we have four areas, and its easy to think about it this way. First area is on improving the medical Product Development process. The article budget issues covered but to give you sense of those with the highest priority but their circle is the following. We call for congressional action to increase in the generation and use of realworld evidence. All of this data is accessible today but its hard to get into the fda in the consideration process. How to take advantage of all this data of information, technology is generating on us underground devices about drugs . Not only postmarket surveillance, which is critical, but also approval of the new indications for existing products. Ultimately, into the Clinical Trials themselves that are used for regulatory review. We also call for actions that enable the dissemination of scientific information to support that whole clinical decisionmaking regarding something thats misunderstood i think generally but for me as a heart, lung, transplant surgeon, that is what the offlabel use. Very confusing to people but it has to be addressed up front. More than one in five outpatient prescriptions, one in five are written for offlabel therapies today. It is critical, fundamental, lifesaving. All of that is to get the approval process is set up to get approval for a new indication, drug or device has got to go to an entire Clinical Trial process. That doesnt mean for drug you know works and has been shown scientifically to work should not be given to patients in need. The fda doesnt regulate the practice of medicine. Nothing prohibits physicians from prescribing drugs offlabel that you know will help them. Manufactures interestingly are generally restricted, they can share that information, good, Accurate Information offlabel use of the product. That inability to share is a huge barrier into effective and safe use of those drugs. In order to balance the risk and benefits of offlabel uses, physicians, that doctors have to have that information, that i could information that right now is being denied them. Nobody is intended to deny. As an example of an example of an antiquated structure very good for the times, right now for the today, scientist and so fast, needs to be reform. We also call for harmonization of International Requirements and standards. I think bart, you come to that because anyone of our discussions we know america is great, the best, the most innovative but theyre good things happening around the world in a scientific way they need to be assimilated into our thought processes and databases. You also see, i dont have the pages before you right now, but we address the whole issue of interoperability, improved interoperability of Health Information technology. Lamarr implied it, you did imply it, with all these different standards, to improve the outcomes for patients. This is a foundation i think for where we are today, not on improving the medical Product Development process from idea to clinical delivery, also for the broad aspects of health care which lamarr did mention, including delivery, getting into the field, payments for the strategy for empowering the patient who has all this information today that we didnt have 10 years ago. We talk about the increasing regulatory clarity. I am going to what all that means that is what regulations actually mean, who is responsible for what, who is in charge and to get clarity. Very, very confusing today. Congressional action is going to be needed to approve the regulatory clarity and the violation of things like combination products, where you are putting different things, different stuff together. Who is responsible for regulating that . Some of the most Innovative Products unlike when dad was practicing medicine 40 years ago is the ability to combine drugs and devices in ways that heretofore have been impossible. This whole field. In my own field of heart specialty, an example would be a drug to begin about 15 years ago for the treatment of coronary artery disease, which mechanically opens up the coronary artery. Insulation devices instead of injecting for the management of diabetes, patches for the treatment of early parkinsons disease are all examples. Theres a lack of inter center, between center elimination of the roles and responsibilities. For those of you who follow the fda closely know what im talking about when you look, who is really responsible for them to lead on it to be clear for the clinical investigator, you need to know who is responsible. That is the rules of the road. Uk conflicting feedback, lack of clarity, chaos which slows down the field as people try to rectify that. A Regulatory Framework can do that for approval and delivery, rejection. There is little consistency and predictability in the decisions made by the office of a combination of products. Since the nonleague center if you centers, if a nonleague center received no user fees for the work, they dont get paid for it yet their part of the process that becomes very confusing. There is little incentive for a time and review of data and products by the nonleague center. Another example, clarity regarding regular effort to associate with the laboratory develop test, its very confusing. Many of us have expressed with directly but when it comes down to Laboratory Tests, diagnostic tests, trying to figure out what is good, bad, what works, its not a big part of spending the e Laboratory Test due to a vatican clinical decisions made. So he was approving and who is responsible is very important. We call for congressional clarification, including clinical software, the things we carry on our iphones around, i. T. Software should not be regulated as a medical device. That software cant be because the medical device is a device, its a thing. When we have Regulatory Framework, it just doesnt work it slowed the innovation. That uncertainty keeps people from innovating. Also clarity is needed uncertain regenerative cell therapy. Great example, adult stem cells is exploded in my own field of Heart Disease and orthopedics and as all of us page you tend to Pay Attention to those things. Right now we are trying to approve these regenerative cell therapies and an antiquated system designed 20, 30 years ago when it didnt exist there. Stem cells, other genetically modified cell therapies argument hold the most promise for curing many of todays insurmountable untreatable diseases that are out there. Additional therapy is needed to assure fdas regulatory, or recognition of unique nature of the stem cell therapeutics, in particular oncologist and similar therapies. Different world when i was in the senate, all embryonic. Adult stem cells got locked down there. More work needed Oversight Framework of come as lamar mentioned, the president s Precision Medicine, what exactly is that and what is a Regulatory Framework. Third area we talk about is strengthening the fdas ability to carry out this mission, its culture. We have so many great people, smart people working every day but working with an outdated framework. We focus on the nation. Mark mcclellan could not be with us, but a lot of time talking about how important that is. We have a number of recommendations in the report relating to the fdas human resources, how do you hire people today, many of these will require elaboration with hhs and the office of personnel management. We all know in government the hiring of a process, hiring and firing and recruiting process, happens in the legislative branch, it needs to be addressed. That whole human resources. Recall of the need for fda to expand the effect effectiveness of publicprivate partnerships. Finally, ive gone on too long, with a section on improving access to medical products for unmet Public Health needs. It is a Government Role and responsibility to look at the unmet needs that may not have a financial incentive to go into. The Biggest Drug Company would amount to make a lot of money just to go to the scientist do not concentrate. Researchers will not be there but theres a Huge Population of Public Health needs and that is a specific role of government that the fda has a very important obligation and responsibility. Lastly let me just say another area is the need to accelerate the development of something, again, that affects everybody in the room that is use of antibiotics. We see a lot in the press today. We know superbugs are an issue. How can we, we know theres a problem, window people are dying from it and it is created by our use of other antibiotics. We need to celebrate to treat, teacher and the attitude. We have to give special attention to the approva