Pharmaceutical Efficacy: The Illusory Legal Standard

The author argues that despite lengthy and expensive drug development processes, the FDA’s efficacy standard under the Federal Food, Drug, and Cosmetic Act permits approval of drugs with minimal effectiveness, as it requires only “substantial evidence” of an effect without establishing any meaningful threshold for the magnitude of that efficacy. The author concludes that FDA approval should not be interpreted as assurance that approved medicines, particularly new and expensive drugs, possess efficacy meaningfully greater than no effiacy at all, with the responsibility for determining acceptable efficacy levels delegated to market forces.