FDA simplifies compassionate use process in a big way
- Between 2010 and 2014, the FDA received 5,995 requests for expanded access in which terminally ill patients could receive experimental drugs in the context of clinical trials. The FDA, which must sign off and approve the requests, rejected only 33 of the requests, RAPS' Alec Gaffney reports.
- While the Expanded Access Program has made it possible for terminally ill patients to obtain experimental drug through clinical trials, many physicians have complained that all of the necessary documentation is cumbersome.
- Expanded access programs work either by creating a clinical trial for a patient, or by amending an existing clinical trial to include terminally ill patients—this is called a protocol amendment.
While expanded access/compassionate use is considered a very good thing by everyone involved, traditionally it has included documentation, starting with a cover sheet, which requires extensive information about the physician, the type of protocol, pharma company information. All told, 26 individual pieces of information are needed.
Under the new guidance, only eight pieces of information are needed—and now requests can be made by telephone. There seems to be a consensus here that this is a step in the right direction.
- Regulatory Affairs Professional Society From 100 Hours to 1: FDA Dramatically Simplifies its Compassionate Use Process