Characteristics of rare disease marketing applications associated with FDA product approvals 2006–2010

New drug and biologic product marketing applications submitted to FDA's Center for Drug Evaluation and Research (CDER) between 2006 and 2010 were analyzed to identify rare disease application characteristics associated with higher approval rates. The results show that approval rates were similar for rare and common disease applications. Larger company size, prior regulatory experience and priority review designation were associated with higher approval rates. The study findings show that rare disease product development is feasible, and increased interactions between product developers and FDA in early investigational phases can facilitate product development.

► We analyze rare disease marketing applications submitted to FDA from 2006 to 2010.
► We examine characteristics associated with rare disease drug approvals.
► Rare disease products have similar approval rates to those for common diseases.
► Company size, priority review and regulatory experience correlate with approvals.
► Increased interactions between developers and FDA may support product development.