U.S. Food and Drug Administration (FDA) Commissioner Scott Gottlieb, MD, testified before the House Energy and Commerce Committee on how the FDA has worked to implement provisions from the 21st Century Cures Act, which includes major changes to how the agency regulates drugs, devices, and biologics.
Dr. Gottlieb told the committee that the FDA is working on a proposal whereby cancer treatments already approved for one indication might be able to more rapidly gain approval for a second indication. Supplemental approvals “can sometimes appropriately rely on a more targeted data set, like a single-arm study,” he explained. “We intend to issue guidance further clarifying the circumstances in which this is appropriate.”
Dr. Gottlieb noted the agency will soon produce other guidance on how to secure approval for biomarker-targeted therapies.
The agency also wants to better define intermediate endpoints. Since the passage of the Food and Drug Administration Safety and Innovation Act, the FDA is authorized to grant accelerated approval to candidates based on these types of endpoints, but better definitions are needed “to ensure that product developers with promising drugs take full advantage of this provision and can consider it in a broader range of such setting,” Dr. Gottlieb added.
Source: Endpoints News, November 30, 2017; FDA statement, November 30, 2017.