One year after the 21st Century Cures Act was signed into law, two Congressional committees held hearings on its implementation. During hearings with the U.S. House Energy and Commerce Subcommittee on Health, and the Senate Health, Education, Labor, and Pensions (HELP) Committee, National Institutes of Health (NIH) Director Francis Collins, MD, and Food and Drug Administration (FDA) Commissioner Scott Gottlieb, MD, provided updates on how their respective agencies have been implementing the legislation.
The Cures Act contained several provisions giving FDA new authority to advance the development of treatments. During the hearings, Commissioner Gottlieb stated that FDA is using its new authority to consider new steps to move drugs through the development and approval process more quickly. Director Gottlieb also noted that Cures set FDA on a transformative path and has allowed the agency to optimize investments in science by modernizing how FDA oversees breakthrough technologies.
- FDA is working on a proposal to allow cancer treatments already approved for one indication to be given approval for a second indication based on a supplemental application that relies on more targeted data. FDA intends to issue guidance clarifying the data extrapolation and when this type of supplemental application is appropriate.
- FDA is working on the implementation of novel trial designs. The agency plans to hold a public meeting on March 20, 2018 to discuss these clinical trial designs. FDA also expects to issue guidance on novel trial designs following the meeting.
- FDA is working to build on recent advances in gene therapy by releasing a series of disease-specific guidance on the development of specific gene therapy products.
In addition, Commissioner Gottlieb discussed FDA’s creation of the Regenerative Medicine Advanced Therapy (RMAT) designation program, which aims to provide more timely access to potentially life-saving therapies by expediting review of innovative RMATs. As of October 31, FDA has granted 11 RMAT designations.
During the hearings, Director Collins discussed NIH’s recent award of 12 grants aimed at answering questions about how to measure the usefulness of data sets, where shared data should be stored, how to achieve interoperability, and other critical questions to enhance data sharing. NIH has also worked to establish the necessary privacy protections to support data-sharing.
Commissioner Gottlieb also highlighted FDA’s partnership with CancerLinQ, which will use real-world, aggregate, de-identified patient care data to examine the real-world use of emerging and newly approved cancer therapies.
As part of NIH’s implementation of the Cures Act, the agency is investigating approaches to expand participation in clinical trials for children and seniors, women, and racial and ethnic minorities. The agency is beginning to collect inclusion data on every study, and next year, NIH will report on this data for the first time. NIH will hold an Advisory Committee meeting from December 14-15, 2017, where they will discuss additional approaches to expand participation in clinical trials.
In his testimony, Director Collins addressed the decline in NIH’s purchasing power, stating that “As a consequence of NIH’s loss of more than 20 percent of its purchasing power from 2003 to 2015, researchers were forced to vie for limited resources, leading to a hypercompetitive environment. With success rates below 20 percent, many highly meritorious applications continue to go unfunded. This has too often resulted in misaligned incentives and unintended consequences for talented researchers at all career stages who are trying to succeed and stay in science.” He discussed NIH’s new Next Generation Researcher Initiative as a way NIH is working to encourage early- and mid-career researchers to remain in the field.
Cancer Moonshot Initiative
Director Collins spoke briefly about his agency’s work to continue to implement the Cancer Moonshot Initiative, and stated that in Fiscal Year (FY) 2017, NIH made 142 Cancer Moonshot awards, including efforts to leverage advances in immunotherapy, understand drug resistance, and develop new technologies to characterize tumors and test therapies.
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