Upton, McCaul request GAO compassionate use study

Reps. Fred Upton (R-Mich.) and Michael McCaul (R-Texas) have requested a Government Accountability Office study on compassionate use. In a Sept. 17 letter, they asked GAO to report on the number of requests for expanded access FDA has received in recent years and the outcomes of those requests. FDA calls access to unapproved investigational therapies "expanded access."

The letter also asked GAO to investigate what is known about "the rates at which pharmaceutical companies accept or deny patient requests for expanded access," the burdens to companies in responding to such requests, and the time it takes for patients and physicians to navigate corporate expanded access programs. ...