FDA's new efforts to strengthen expanded access program

FDA Commissioner Scott Gottlieb, MD, discussed Thursday steps the agency has taken to strengthen the expanded access program, which aims to facilitate access to promising investigational medical products for individuals with serious or immediately life-threatening conditions who are unable to access products via clinical trials.

FDA Commissioner Scott Gottlieb, MD, discussed Thursday steps the agency has taken to strengthen the expanded access program, which aims to facilitate access to promising investigational medical products for individuals with serious or immediately life-threatening conditions who are unable to access products via clinical trials. According to Gottlieb, FDA has authorized more than 9,000 expanded access applications across drugs, biologics, and devices in the past 5 years, authorizing about 99% of all requests it has received, for all application types. An independent assessment of the expanded access program last year "found overall support for FDA's program, but as we hoped in pursuing this analysis, also identified new steps we can take to improve upon our efforts," Gottlieb said. Based on the feedback from the analysis, FDA announced Thursday that the agency's expanded access webpages will be updated to increase usability through streamlining of content and a more user-friendly organization. In addition, FDA plans to formally establish an agency-wide Patient Affairs Staff and Health Care Providers Affairs Program to "enhance our engagement with these important external stakeholder groups," he added. FDA has also formed an agency-wide Expanded Access Coordinating Committee, which Gottlieb says "facilitates cross-Center communication and promotes discussion to rapidly address cross-cutting issues related to expanded access to promote consistency and best practices."