On November 30th, the House Energy and Commerce Committee held a hearing to discuss, “Implementing the 21st Century Cures Act: An Update from FDA and NIH.” A year after 21st Century Cures Act was signed into law, Members of Congress asked Francis Collins, Director, National Institutes of Health (NIH) and Scott Gottlieb, Commissioner, Food and Drug Administration (FDA) to provide updates.
Representative Burgess (R-TX) opened the hearing by commending the bipartisan effort required to pass 21st Century Cures. He then went on to highlight key provisions like the Precision Medicine Initiative, Cancer Moonshot, the FDA Innovation Account, and modern clinical trial designs.
Scott Gottlieb emphasized that the FDA is currently examining how to leverage the agency’s accelerated approval pathway. Accelerated approval would include the authority to require confirmatory evidence to support continued marketing efforts and an expedited withdrawal mechanism if a treatment fails to show benefit. To fully leverage these opportunities, cancer drugs already approved for one indication could be approved for a supplemental application if evidence demonstrates. When Representative Matsui (D-CA) asked for updates on rare disease provisions in Cures, Dr. Gottlieb re-emphasized that the accelerated approval mechanism should be applied to rare disease trials when there is clear clinical benefit at an early stage. The FDA is already making robust use of provisions that allow, especially in the oncology setting, the agency to create supplemental indications without having to do additional clinical trials. This is especially relevant because, as Rep. Matsui pointed out, many rare disease patients take drugs off-label.
Click here to read the press release from the Energy and Commerce Committee.
The Senate Health, Education, Labor and Pensions (HELP) Committee will hold a hearing on the same topic on Thursday, December 7th.