GAO Report on Expanded Access

The 2017 GAO report on Expanded Access is now available. The GAO conducted a survey and interviews amongst pharmaceutical manufacturers, FDA stakeholders, physicians and patient groups to examine three main areas: 1) ascertain what is known about the number, type and time frames of expanded access requests received by the FDA; 2) what actions have been taken to improve the process of expanded access requests; and 3) how FDA uses data from expanded access in the drug approval process. The primary GOA recommendation is that the FDA should clearly communication how the agency will use AE data when reviewing drugs and biologics for approval.