Evidence used for FDA decisions “varies widely,” study claims

The quality of clinical trial evidence used by the US Food and Drug Administration as the basis for recent approvals of novel therapeutic agents varies widely, with important implications for patients and physicians, claims a new report in the Journal of the American Medical Association (JAMA).

Researchers analyzed publically available data from FDA approvals from 2005 to 2012. Pivotal efficacy trials were classified according to the following design features: randomization, blinding, comparator, and trial end point. Surrogate outcomes were defined as any end point using a biomarker expected to predict clinical benefit. The number of patients, trial duration, and trial completion rates were also determined.

More than a third approved after just one trial