The accelerated approval regulations of the US Food and Drug Administration
(FDA) allow pharmaceutical manufacturers to offer patients treatment for life-threatening
diseases. This may occur when early evidence suggests that the agent is likely
to improve survival or reduce symptoms, before confirmatory studies show whether
the compounds are fully safe and efficacious.
However, while this concept is embraced by many clinicians and patients,
recent evidence suggests that its execution leaves much to be desired.
Mitka M. Accelerated Approval Scrutinized. JAMA. 2003;289(24):3227-3229. doi:10.1001/jama.289.24.3227