So doctor, pharmaceutical Development Today frequently involves what is called shooting in the dark, making key decisions can with very limited data. For example, decisions including indication selection, dosage end points and Inclusion Exclusion criteria. The data that informs such decisions lies in inaccessible silence at least to most stake holders. It includes to hire longer time lines. Now the fda already has much of this data, this otherwise siloed information. So my question for you is has the fda taken any steps in ago ri gaiting to improve the process and if not would the fda be willing to analyze the data to accelerate and derisk . I appreciate it. The bottom line is there are situations. Well be taking steps to make it improve it where they can be ago gri ka there are situations we provide from data we use to make decisions particularly around drug safety. The information is confidential. We are looking at how to make it public without because my belief is if we are making regulatory decisions that isnt accessible to the public thats probably something we should try to address chl address. This is a very complex area. There are legal complexities. We are out of time here. I look forward to working with you. Thank you, senator young. Thank you. I want to also congratulate you and senator murray on this. I wonder if you could give a little bit of an update on where you are that we worked on together. Well, i appreciate the question. Ill try to be brief. We announced major components of some of those today. We continue to move forward with those. I think its a good example of what we are trying to do. Or to exclude used by clinicians. We will also exclude certain Decision Support tools that are used by patients as long as they meet certain things. What do you expect to see in tomorro terms of new devices . I think in the area of Decision Support tools. There are tools that could take information and help support decisions before the clinician or the patient. I think in part there are a lot of reasons we havent seen as much as you would have thought. It probably played a role for a period of time. Fda always intended to dpexerci enforcement. So this provides us the opportunity to put out guidance that creates per am ters on what does and doesnt cross the line. I appreciate your focus on it. I also noticed that the fda approved the first breakthrough designated diagnostic test to detect genetic mutation. It came through the breakthrough device section we wrote last year. I was interested to see that there is a decision to also cover the diagnostic test. I wonder if you could share with the committee how you conducted these reviews. I think you can, senator. We work to have there place coverage at the same time. We think Going Forward this is a panel of cancer markers that will help guide the prescription thera therapy. This is a lot of innovation. Ill let them articulate it more carefully is that panels that come through fda for voluntary approval will automatically receive coverage determination. It is for more such tests to come through. We can provide greater assurance. I think thats a big step forward. Thank you. T the last question i have, senator rubio and i last year worked on the race act for pediatrics cancers. I know you put out guidance on developing drugs and rare pediatrics diseases. I hope you can expand on that. Thank you. What we did yesterday is final ease guidance that we had. It tries to look at very rare pediatrics diseases. Trying to outline a more efficient prosecess. It is to represent the experience of the placebo and allows sponsors to collaborate to try to test multiple drugs in the same Clinical Trial. I think why they are important is because it is very hard to enroll patients in these rare diseases. They are trying to allow them to look at ways you dont have to can facilitate more efficient development. I didnt thank them for their service to this country and i deeply appreciate it. Thank you sflaenator bennett. Senator collins. Thank you. Always glad to see you. I claim you as my cousin regardless of the facts. I accept. Thank you. The state of maine is doing some real loi exciting medical research. I have visited a lot of the labs and medical institutes we have in our state. The main Medical Research Center is conducts cutting edge work to develop medical treatments for chronic diseases. In september one of the researchers with whom i met and his team at Maine Medical Center received an award through the nih im sorry, regenerative medication process. Using adult stem cells he is working to develop novel therapies. Regenerative medicine holds great phone shl for understanding ageing and reversing diseases like mac lar degeneration. I understand that the dharm has already introduced oliver. He too is from the state of maine and grew up only 11 miles from where i did. He shared his compelling story about losing and regaining sight through regenerative therapy. What especially can we do to support research into biological ageing is this. What a great question. Thank you for pointing out a number. As we are gathered here this morning with an intens discussion several hundred people talk about applications to kidney disease, ageing and such things as diseases and the whole landscape. I even forecasted if we do this in the way i think we could we could cure disease like sickle cell. We are learning a lot about what that process is. I mentioned one example. That is that as you age theres a certain category of cells that are no longer able to keep going. They dont completely exit the stage. They are still there. It turns out they will not good actors. They are making the healthy cells around them not so healthy. As we could figure out how they could be told okay, youre done now it could slow down the normal process of ageing. There is a new exciting development. And i remember visiting Harvard University and talking are researchers there. They are also looking at the possibility of turning proteins back onto restore cells that have been lost. We have a partnership with industry you may have heard about. It is advancing this at a pace i wouldnt have thought possible a couple of years ago. That brings my question to you about that program. Which aspects do you find most promising . For alzheimers there are two areas. We really need to find out what are the indications that are therapy is working without finding out whether it was protective. Theres a lot going on there with various types of images. One is involved in this. The other part is this systems biology where we are try to go st trying to step away to find out whats doing on and how could we use that to develop this next generation of therapeutics taking away all of the general rations and apply t early. We have to start early if we want to get a good we if he cef. Thank you. Thank you senator collins, senator murphy. Thank you both for your service. I know it comes with great sacrifice. I know the sacrifice that you make to serve our country, thank you to both of you. I wanted to talk to you more about the all of us research program. This is a program that designs to build a medical data set for Precision Medicine initiative. Im interested in it because one of the participating Community Health centers is based in middleton, connecticut. I think its great you have gone out and worked with Community Health centers. They tipedly serve a Patient Population that is often under tps served it is currently part of this Community Health center. Very pleased with your partnership on this and we would loouf love to see more like this involved. We are seeking to enroll participants in this in the United States. We are inviting people to participate by a variety of means. One is through the Community Health centers. We have a very specific goal here of having at least 50 being individuals that are un r underrepresented. They provide care to those in situations are very excited about being our partners. We are starting with tin gettin up and going. That is a pilot to see if we can enroll many more. At the moment we are in a beta test. We are learning everything we can about highwow the moving pa can Work Together. It is looking good. Wanted to switch topics to another exciting development. This is the establishment at the fda of the Oncology Center for excellence which creates a cross center team to Work Together on a variety of products to treat cancer. Just an update on what you have learned. What do you take into account when you think about creating other disease specific offices. I appreciate it. This is an extremely important effort. I appreciate you asking the question. We consolidate into one combined center. So this was instrumental. The products by consolidating were able to look at some of the buy logi b biologics. We are looking to other areas we can do this. I think before we can progress onto other areas we want to make it work. We are looking forward to try move this program forward. Another reminder we have to give you the funds to implement this. Quickly back to you, one more question and that is on another part of the 21st century we worked on a provision that allows for the cdc to collect information ton incidence of neurological diseases. Talk for a second act the importance of having this data. It is important to know what it is for neurological conditions. It is challenges and expensive to do that kind of analysis and to do it effectively and to keep it updated. Thank you very much. Thank you. Senator hatch, welcome. Thank you so much mr. Chairman. I think youre some of the best Public Servants i have seen there all of my 40 plus years of the United States senate. Im proud of both of you. I hope youll keep doing what youre doing. I want to thank you both here today. I joined senator bennett in the act included in the 21st century cures for new antibiotics by allowing them to be studied in smaller more rapid Clinical Trials. As you know developing guidance regarding this limited population. Can you detail ways in which it is being incorporated into the guidance . Thank you. We plan to issue the guidance and we have been meeting with stake holders in the development of that guidance in the development. We think it is an extremely important pathway. We believe it will be a robust vehicle to create new pathways for significant medical needs. Thank you. I was pleased to see the nih was able to start a project that several from this initiative and as you may know i have been a champion for quite some time. It may prove to be what do you think as the project continues . Thank you for your question. I think there is an enormously deciding time because we have tools now that are starting to work. He referred a little bit ago to therapy. Really dramatic events using gene therapy that delivered the gene. It is with really remarkable benefit over the course of many weeks. Thats a single example of whats possible. My hopes we could yur circle cell disease by correcting the mutation and putting them back. It is a transplant to yourself. Rekrentdly s plrks a whi now Clinical Trial some of those kids are make it to prekinder garden a prekindergarten. I think it is to push the science as hard as we can. It gives us that kind of inspiration. Im glad to say the barriers are not an issue right now working with scott and his colleagues that are also made it a very high priority. There is an issue about investment. Countries may not be so interested interes interested because it will be so small. It has a stronger responsibility to push hthem as far down the road as possible. One thing you did is make it possible for us to run phase 3 which we had not previously had the privilege of doing and which we will be using aggressively for this purpose. One was designed to improove opportunities. My time is up to thank you for your willingness to serve and how much you mean to people like us. Thank you. Thank you senator hatch. Senator baldwin. Thank you. I want to add my words to the others of the bipartisan work you had. It is on the 21st century cures act. I thank you for your service. During the Committee Deliberations i have any number of times about being the granddaughter of nih funded scientists and how it influenced my championing and strengthening report for the nih. But in my first year in the United States gnsenate i had an inspirational meeting with a young man, a high school senior. His name is ian. He is a bone cancer survivor from wisconsin. He said it helped save his life. Thats why he wanted to grow up to be a scientist, to help others with his disease, but he was concerned that it wouldnt be possible for him to break in as a new researcher due to his awareness of nih funding cuts at the time. He inspired me to author the next generation researchers act along with senator collins to fight to improve nih opportunities. I am proud to report that ian recently graduated from college where he was helping a researcher study the genetics. He is now working with a scientist in salt lake city, utah on pediatrics cancers. I am encouraged that the nih has begun to robustly the initiative to help support future scientific leaders like ian. As they discover cures for cancer and pursue other life saving research. You in the earliest parts of their Research Careers to help them sustain or achieve research independence. I would like it if you could please describe how each institute and center will prioritize awards and how this will help improve and stabilize opportunities for our next generation of researchers. Thank you for the question. Youre touching on a personal passion that i feel and many of the other Institute Directors do as well. We actually just published about a month ago a description of what this policy is and how we are going to implement it. We passionately agree that it is really critical for our future. It has been a tough they sustained between 20303 and 2015. They were having a hard time getting started. We cannot lose that. We were starting to. So each one of the institutes depending on where they think they can find their flexi flexibilities are freeing up dollars to make it possible for those that would just miss the pay line to just get started. We started this quite late but we were able to make a buncho o awards. We are trying to see if we can reach back into some of those years and fauund a few more of those. It is not just the gray imnan imminences and people that are involved in this. It will make this priority really happen. Im running out of time but i want to just note you estimate this new effort will would be about 210 million in the next year and 1. 1 billion over the next five years. Do they need it to ensure that it fulfills its promise and continue to advance all other critical kefefforts . We can do more if the resources are available. Before we go to senator warren let me recognize senator murray. Thank you. I will be submitting more. This has been an excellent haerg. Hearing. It is really educational for all of us and look forward to continuing to work with you. Thank you senator murray. Senator warren. Thank you. We have been talking about the cures act. Im really glad became law. During our recent hearing we discussed Research Participants that they had borworked on together. Right now they are benefitting from a provision that senator bennett wrote to clarify the authority relating to gene therapies. Its a long list. Cures also fell short in a really big way. Thats on funding. Im glad democrats on this committee and calling for an extra 50 billion and cures did not send one single new dollar to these agencies, instead it only said that future congresss might spend about 10 of that amount on nih and fda. Im glad so far congress has been increasing funding but i dont think its time for us to pat ourselves on the back yet. So dr. Collins, let me go through this a little bit. Duds the nih fund most of the grant applications if. No. We certainly arent able to do that. So out of every hundred applications you get youre funding about 19 of them. Is that because the other 81 would have been bad investments that would not have helped us make bio medical breakthroughs to advance science . If we look back around 2000 and 2001 we were funding because funds were more available. We looked to see did it actually turn out to be less productive . The answer is no. We cant really tell the difference even though they are trying to draw distinctions it is very hard to do so. So in order if we doubled the number of grants we were able to fund you think theres still a lot of good science . I think there would be fantastic science. Okay. Thats powerfully important. I want to follow up on the point that senator baldwin made and researchers in the early part of their career. Getting that first nih grant. It can be the difference on whether or not the scientist stays in the fight or whether the scientist has to leave academic medicine and go somewhere else. I want to z more. Where are we on early career researchers . What percentage of the grants we were able to get. Beginning in 2008 we with instituted a policy so that they got a bit of a boost. They come peelted against each other as opposed to the most experienced ones which meant terms that got a few extra points. Thats not good fluff. We are still losing lots of those. They were well below what you would want to see. We think it would be most healthy if at least 25 of those were going to get funded. Thats what we are trying to do with this new initiative which is named specifically for the words that were used in the bill. Thank you senator baldwin for that encouragement. I understand right now we have been at about 16 . Is that right . Youre saying we ought to be boosting that at about 25 . We looked closely to try to figure out how to get there with this new policy, yes. I know nih has done what it can. Nih funding is still down about 15 of where it was a decade ago back when we had a higher success rate for the proposals that were coming across reviewers desk. It didnt even come close and thats why today we are reintroducing research angt which providing funding for the fda. I see youre sitting up straighter there. This legislation is cospo sonso by all of the members