Investigational New Drugs: FDA Has Taken Steps to Improve the Expanded Access Program But Should Further Clarify How Adverse Events Data Are Used, Report to Congressional Addressees   [open pdf - 8MB]

"FDA's goal for the expanded access program is to allow patients with immediately life-threatening and serious ailments access to investigational drugs when appropriate. Stakeholders have raised concerns that FDA's process is confusing or burdensome, particularly for the entities that have roles in the process--such as physicians, manufacturers, and institutional review boards. GAO [Government Accountability Office] was asked to examine the expanded access program. Among other objectives, GAO examined 1) what is known about the number, type, and time frames of expanded access requests received by FDA; 2) what actions FDA and other stakeholders have taken to improve expanded access; and 3) how FDA uses data from expanded access in the drug approval process. GAO reviewed regulations and FDA documents and analyzed FDA data on the numbers and types of expanded access requests it received from FY2012 through 2015, the most recent at the time of the review. GAO also interviewed FDA officials and other stakeholders including nine manufacturers-- selected to represent large and small companies--and patient and physician representatives."

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Government Accountability Office: http://www.gao.gov/
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