GAO: FDA Must Clarify How Expanded Access Data Will Be Used

The FDA needs to clearly communicate to drugmakers how the agency will use adverse event data gleaned through the expanded access use process, according to a new GAO report.

The FDA simplified the expanded access process and offered more in-depth guidance last year, but its communication with manufacturers “lacks clarity and specificity,” GAO researchers found, in a report titled Investigational New Drugs -- FDA Has Taken Steps to Improve the Expanded Access Program but Should Further Clarify How Adverse Events Data Are Used.

The lack of clarity was a common concern raised by stakeholders, some of whom said they may be more likely to deny expanded access requests for fear of any subsequent adverse events delaying drug development.