RPTR FORADORI EDTR CRYSTAL IMPLEMENTING THE …

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RPTR FORADORI EDTR CRYSTAL

IMPLEMENTING THE 21ST CENTURY CURES ACT: AN UPDATE FROM FDA AND NIH THURSDAY, NOVEMBER 30, 2017 House of Representatives, Subcommittee on Health, Committee on Energy and Commerce, Washington, D.C.

The subcommittee met, pursuant to call, at 10:03 a.m., in Room 2123, Rayburn House Office Building, Hon. Michael Burgess, M.D. [chairman of the subcommittee] presiding.

Present: Representatives Burgess, Guthrie, Upton, Shimkus, Blackburn, Lance, Griffith, Bilirakis, Long, Bucshon, Brooks, Mullin, Hudson, Collins, Carter, Walden (ex officio), Green, Engel, Schakowsky, Matsui, Castor, Sarbanes, Lujan, Schrader, Cardenas, Eshoo, DeGette, and Pallone (ex officio).

Staff Present: Ray Baum, Staff Director; Karen Christian, General Counsel; Kelly Collins, Staff Assistant; Zachary Dareshori, Legislative Clerk, Health; Paul Edattel, Chief

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Counsel, Health; Adam Fromm, Director of Outreach and Coalitions; Caleb Graff, Professional Staff Member, Health; Jay Gulshen, Legislative Associate, Health; Ed Kim, Policy Coordinator, Health; Bijan Koohmaraie, Counsel, Digital Commerce and Consumer Protection; Katie McKeogh, Press Assistant; Alex Miller, Video Production Aide and Press Assistant; Mark Ratner, Policy Coordinator; Kristen Shatynski, Professional Staff Member, Health; Jennifer Sherman, Press Secretary; Danielle Steele, Counsel, Health; Hamlin Wade, Special Advisor, External Affairs; Greg Zerzan, Counsel, Digital Commerce and Consumer Protection; Jeff Carroll, Minority Staff Director; Waverly Gordon, Minority Health Counsel; Tiffany Guarascio, Minority Deputy Staff Director and Chief Health Advisor; Jessica Martinez, Minority Outreach and Member Services Coordinator; Samantha Satchell, Minority Policy Analyst; Kimberlee Trzeciak, Minority Senior Health Policy Advisor; and C.J. Young, Minority Press Secretary.

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Mr. Burgess. The subcommittee will now come to order. The chair will recognize himself for 5 minutes for the purpose of an opening statement. The 21st Century Cures Act was a monumental achievement. Cures was the product of a bipartisan, multiyear effort by the Energy and Commerce Committee that brought our laws into a modern era of medicine. It has been nearly 1 year since Cures was signed into law. I remember remarking at that press conference a year ago to imagine a world in which government was not an obstacle but an ally in helping us deliver drugs and devices to patients and cures to patients. Today's hearing marks the Health Subcommittee's first look into the implementation of what many in the healthcare community called a transformational bill that would positively impact not only the researchers and the scientists who are developing the latest breakthrough therapies, but physicians seeking treatment for their patients, giving hope to them, their loved ones, and their advocates. This morning we will hear from two leaders responsible for implementing the drug development and biomedical research provisions included in Cures. I want to welcome Dr. Francis Collins, the Director of National Institutes of Health, and Dr. Scott Gottlieb, Commissioner of the Food and Drug Administration, both back to this subcommittee. All of us know the demands your schedules put on both of you, and we appreciate you coming before us today. At the time of the Energy and Commerce Committee's launch of the 21st Century Cures initiative, the statement was made repeatedly that there were 500 cures and treatments to address 10,000 known diseases. More progress was needed to alleviate the agony of an incurable disease.

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While the United States was maintained its global leadership in biomedical innovation, there existed a potential bridge in the growing divide between the revolutionary advances in science and technology and a less-than-adequate system for discovering, developing, and delivering new therapies.

Members of the committee, both this committee and the Senate HELP Committee, held numerous public hearings, forums, roundtables in Washington, D.C., and around the country bringing together leading scientists and medical experts, patient and disease group advocates, and researchers across multiple sectors. The primary objective of these events was to uncover opportunities to strengthen and streamline the process by which cures are discovered and made available to patients.

Cures accelerated the cycle of discovery, development, and delivery of new treatments and ensured that the United States remained at the helm of biomedical innovation. At the National Institutes of Health, the 21st Century Cures Act authorized resources to support biomedical research and reduce administrative burdens and provided almost $5 billion in new funding to support the agency's four innovation projects.

The Precision Medicine Initiative was authorized for $1.4 billion for the National Institutes of Health to build to a national biomedical dataset in order to accelerate health research and medical breakthroughs.

The bill also authorized $1.5 billion for the Brain Research through Advancing Innovative Neurotechnologies Initiative to better understand the brain's physiology and to coordinate efforts across multiple Federal and private groups to expedite research for diseases like Alzheimer's.

Cures also authorized $1.8 billion for cancer prevention, cancer diagnosis, cancer treatment and care through the Beau Biden Cancer Moonshot.

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Finally, the Regenerative Medicine Innovation Project was authorized at $30 million to support clinical research in the field of regenerative medicine in coordination with the Food and Drug Administration.

The 21st Century Cures Act helped the Food and Drug Administration modernize the regulation of medical products throughout its lifecycle. It established the FDA Innovation Account and authorized $500 million in funding to implement Title III of the law, which included a broad range of deliverables from the Food and Drug Administration.

These include creating a mechanism for the collection and incorporation of patient perspectives in regulatory decisionmaking, updating the way medical products are reviewed and approved, and advancing new drug therapies through a review pathway for biomarkers and other drug development tools to help shorten the development time while maintaining the same rigorous standard for safety and effectiveness.

It also required the Food and Drug Administration to establish standards and definitions necessary to develop regenerative medicines.

Before I close, I recognize the 21st Century Cures Act also touched upon other critical healthcare priorities, such as mental health and health information technology. Both of these areas should have their own separate hearings because of their importance to the medical community, and those are on the list for the very near future.

I again want to welcome our witnesses and thank you for being here. I look forward to your testimony.

My time has expired, and I will yield to the gentleman from Texas, Mr. Green, the ranking member of the subcommittee, 5 minutes for an opening statement, please.

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