Recognizes so for five minutes for purpose of the Opening Statement 21st century chairs sector is a monumental achievement as the product of a bipartisan vote the love effort from the committee to bring the of laws and to the modern era in one year since signed into law i remember remarking at that press conference one year ago to imagine a world that government was not the obstacle but in allied to deliver drugs and devices and cures to patients. Looking into the implementation the transformational bill to give them packed the village to researchers and scientists with the latest breakthrough there peas is seeking treatment to give help to them and their loved ones and advocates for. This morning well hear from those of are responsible for all of the Biomedical Research provisions in the commissioner of the fda coming back to the subcommittee your schedules switched and we appreciate you coming before us today at the time of the launch of the 21st Century Cures Initiative the statement was made repeatedly there 500 jurors santry mens and more progress was needed for the diseases and maintaining that Global Leadership there existed a potential bridge with the revolutionary francis of science and technology and less than adequate system to delivering new therapies. Members of the Committee Committee numerous public hearings around the country for a leading scientists and medical experts and researchers across multiple sectors. The primary objective is to uncover opportunity to streamline the process that pictures are discovered and made available within a cycle of delivery to ensure the United States remains at the helm of biomedical innovation. The 21st century chairs stacked to support Biomedical Research to reduce administrative burdens to provide 5 billion of new funding to support these innovation projects. And was authorized 1. Four hour billion dollars the the National Institute of health to exhilarate held by its research and medical breakthroughs. Also authorize 1. 5 million for Brave Research to advance narrow Technologies Initiative to better understand and to coordinate arts to expedite research and also authorized for through the though biden cancer moonshot and then authorized a 30 landowners with Clinical Research and coordination with the fda the 20th century jurors fact modernize the regulation throughout the life cycle to establish the fda authorization to care authorizing 500 million in funding to implement title three witches a broad range of the live burbles from the fda including a mechanism for the collection for Patient Perspectives of regulatory Decision Making making, the way they are approved and in financing new drug therapies for the aisle markers. Other tools that maintain the same rigorous standard for safety and effectiveness also requires the fda to establish standards necessary to develop Regenerative Medicine. Before i close from recognize the 21st century chairs fact also a touch of critical priorities such as medical Information Technology both have their own separate hearings because of their importance to the medical community and those are on the list for the future and afford to your testimony my time is expired i will yield for five minutes for an Opening Statement. Thanks for being here this morning things to the former chairwoman as the founders of the 21st century cures law does the last public sightings germany by president obama during bipartisanship but tests were still a long before 16 but will set out on a mission to boost medical research and accelerate the discovery and delivery of new cures and treatments around the hearings and drafts we have bipartisan is support endorsements from 700 organizations representing the full spectrum of stakeholders. Dedicated 6. 3 billion to support the of the initiative within the National Institutes of health to combat Prescription Drug abuse also to the fda to implement the policies of the underlying bill this is put towards complex science problems is specifically in was provided 4. 8 billion of new funding to a france Cutting Edge Research and provided 500 million over 10 years to expedite to drugs and devices about compromising the effectiveness. In addition there were so many provisions from field to the super bugs are enhancing but some of those are technical in nature have realworld impact. They deserve to have the our elected officials respond to their needs and this was the effort to do just that of all negotiations we didnt get everything we want there is more that could be done but today is an opportunity to hear from the fda ed nih with patient focus to Drug Development juror improving expertise in hiring capacity. It is only betty your but i know those respective agencies are hard at work to of various medical Research Well not the focus of todays hearing also 1 billion to combat the Prescription Drug abuse funding was a dividend but pales in comparison there are more americans dying from this epidemic them the aids epidemic and then to provide funding to fight the epidemic. The 21st century jurors from when we work across the aisle and hope to do so again in with an ongoing implementation and also remained give the remainder of my time to the congresswoman. Thanks for all of your work you did on 21st century cures act and will be sorry not to have u. S. My partner with issues like this in the next congress you have done a wonderful job. But fred upton of course is my partner and chairman at the time and to we wanted to do something bold and the big and we achieved that. I am looking forward to hearing from our witnesses as agency was a key partner fell 1. I remember very early on dr. Collins said we need to let our young researchers go to conferences and a set of that is all we do we will have failed for kobe did that and so much more so we hear eager to hear how this bill has impacted but even more where we go next. Thanks for working together on this bill. I yield back. Thanks for having this oversight hearing of an incredible savings lifesaving law passed in a bipartisan way the finest work this committee has ever done. I never did anything else on your plate over the last year, i have said that facetiously because you have a lot yet you seem to be doing a marvelous job passing this bill to move forward to save lives to improve the lives of families and friends and the consequences they are not confined to this hearing room where the district and the Research Made in these sectors will offend and then to do marvelous work but my view that once you pass a law it is the starting point i know how difficult it was to get everybody to do this but that was the starting point. Are these tools effective or changes that could be made . We know youre making progress by one to recognize a very special guest better in the room to draw special attention that this part of the journey from the beginning. Youre delighted to have you as an extraordinary and me and were so glad you are here to share in this special birthday appearance of the 21st century cures act people like you inspire us to do the best this committee has to offer because we know human lives are at stake. [applause] so with that i will submit my eloquent we written opens statement into the record and give to the former chairman on energy and is there other members have like to share so i yield to the champion of 21st century cures act my friend from michigan. To be given the of process for years ago we began with one goal in mind in to be inspiring to act after hearing from those in the Research Community with the need of modernization of both the nih rand fda to move quickly to bring lifesaving treatments to market it to have inspirations the sisters have day rared janet yellen negative genetic diseases it provided the nih with tens of billions of dollars of resources were those that impact every single family. Cancer or Rare Diseases but most notably a reminder how republicans and democrats came together in to sign into law and diana worked with me on this but millions are counting on us and i am glad to welcome on how that law is implemented and what we could do. We do welcome our witnesses said and taking this opportunity to thank you for the health the help and guidance what was your role bin in as we implement 21st century cures act. It is so appropriate lead to this hearing because you have heard what went into this bin to say having people go to conferences or lets try. It is a way to change and reform the approval and if it leads the innovation with the Health Care Delivery system we will come back san his bipartisan friends and then to have a reserve see right there in the front row mr. Chairman thinks for a the hearing and i yield back want to welcome dr. Collins here today with the implementation of the 21st century cures fact those with the Opioid Epidemic and Mental Health focusing on the ongoing work at nih to implement the provisions of the law aimed at improving the discovery and development of treatments and cures for 21st century cures act with Cutting Edge Research with the bowl by bin Moonshot Initiative to a salary Cancer Research to improve our ability to detect cancer early on in the hope one day redefined a cure for those such as pancreatic affecting patients today her cry interested to hear n. I. H. Is achieving the goal also investing the precision that this initiative of the Brain Initiative and as in those leaving to pursue their fees and then to get the patients faster while maintaining the steward for effectiveness. In the sense of for it the development and encouraging the use of patient but with 500 million in funding to ensure the agency has the resources to recruit the best and brightest and implement the laws of afford to hearing were about the progress the agency has made on these issues. Last the 21st century cures act marks the important step for treatments we could work on of a fundamental basis with those chief sponsors it is critical to hold hearings that it is working as should and looking at further implementations of the of lot. So taking the remainder of my time savings for organizing this hearing today and as congress to Work Together to pass a comprehensive addiction in recovery act but everybody had everything they wanted the during the debate i continue to advocate for more funding and resources to address the deadliest of crisis in American History coming together to provide 1 billion to strengthen the response to a the crisis with the twoyear funding window to creates planning problems for state and local governments to plan beyond two years makes it difficult for those on the ground to do the work we were of powering them to do so now we will extend the cures funding to combat additional five years that extension is the minimum we should be doing i am grateful to the members of this committee who have cosponsored this bill and have added their voices to the effort to find Common Ground in because the drug crisis turns those communities across the country we must Work Together to ensure it does not expire jiminy your suffering without access to meaningful support systems. One longterm advocate with the development of safe and effective non addictive hope your aides that stop the craving so dr. Grundy by communicating with your office on this matter in your working with nih to facilitate that development of non addictive pain treatments i sent you a letter you responded ready answering a few of the questions but not all of them. I will send the letter again with a more thorough answer and responds to all of the questions in submitting into the record. Mr. Chairman takes for holding the hearing. And then to be made part of the record ben to testify before the subcommittee he will have a the Opening Statement and this morning well hear from the commissioner from the fda and in darter collins to rush your from the National Institutes of health. Youre recognized for five minutes. Thank you for the opportunity to do testify for the 21st century chairs stacked to update on the provisions of this landmark provision giving a broad set of authorities with the organizational structure to be transformative congress wanted us to have policies to enable the American People to capitalize on the break through science transforming medicine. And i want to commit the implementation of this legislation lectures fact is a defining element when i arrived seven minutes of your remarks i cannot imagine a better time with new opportunities for the first offered by a science and Technology Digital health and the potential to be dramatically better in the second provided by congress with those user fees to offer a new platform with those practical treatments for patients if i said that in the next five years with a cure for hemophilia or sickle cell it would not have been realistic such discussions are no longer prudent these equally profound are before us and the cheers fact inspired a new approach the one in restituting different and transform human health. We remain steadfast but at the same time asking us to make our approach more scientifically the modern and efficient. We have taken in the spirit of jurors for a policymaking and planning and then take were continuing to expand with congress set up to achieve. With more targeted medicines the we could observe earlier , this is especially true with the field of oncology to explore new ways to facilitate the review of these products and for example, currently examining for purges by leveraging the existing expedited programs accelerated approval has been granted were earlier small datasets show those on many the that is the benefit from the surrogate endpoint and then to predict clinical benefits. What you do with the targeted drug that has a benefit for overall survival or a deadly cancer as a small child to have more evidence to understand how to best use the drug in clinical practice to approve earlier to validate the finding similar to the accelerated approval approach. Even though the benefit was an early look at survival we believe in the so rigid approval approach could be valuable. Congress clarify our authority to be based on intermediate and points we want to better define what is mean meant for other promising drugs to take full of vintage in a broader range of such settings. For those to be more carefully tailored we will see more pieces and situations of those benefits in a smaller stage Clinical Trial. It would have the evidence to support the marketing if that fails to confirm the benefits and also to exploit the policy work we are undertaking per from and working on similar proposals in the approval concerns the never really relying on a more targeted data set we will issue guidance budget in clients their poorer new concerns but and then to enable new technologies to reach patients in then to ruth of course, and build on framework for from of afford to answer your questions. No the director, of mr. Collins. Good morning and other distinguished we were cheering one year ago today when it passed 392 26 as it a into a cannibalize day gold that the pace of scientific discovery has been translated in night and talk to you today have a dream is coming true. We appreciate your leadership passing this bipartisan act when year ago with the resources in ways to help achieve this goal. Those included to reduce of illustrated of burdens and expanding our ability and his trephining missions in my written statement i submitted a comprehensive report out nih works quickly we are motivated by a sense of urgency and i would like to focus on the chairs Innovation Fund in the procedural medicine in the most important audience which is those patients waiting for answers in the light to recognize my friend at the end of the furrow a very effective spokesperson for the folks to avestan Regenerative Medicine. Beginning with a Brain Initiative this pioneering effort aimed at a revolutionary in revolutionizing the bills complex structure in the known universe, the human brain we leveraged our funding with appropriation with 110 bright Brain Research projects to create a census of self but to have new tools to monitor and modulate this will advance efforts to develop new ways of detecting and treating disorders such as alzheimers disease drug addiction epilepsy and dramatic brain injury finding a Second Research area to aggressively pursue a very ambitious goal in collaboration with our colleagues that the fta we must make steps to enhance the infrastructure to create a network to move Cancer Treatment programs for word and in another innovative move joining with the fda in 12 pharmaceutical companies to launch the partnership for accelerating Cancer Therapies the Publicprivate Partnership will develop aisle markers to speed the development of Cancer Therapies in the exciting approach using the patients own immune system recently we have seen amazing responses of immunotherapy to bring the success t