Accelerating access to treatments for rare diseases

Nature Reviews Drug Discovery 10, 475-476 (July 2011) | doi:10.1038/nrd3493
Marc Dunoyer

Changes in regulatory policy and legislative incentives to promote the development of drugs for rare diseases — orphan drugs — have led to increases in the number of orphan drug designations, but the rate of such products reaching the market remains frustratingly flat. This article highlights areas in which novel approaches could facilitate regulatory approval and access to treatments for rare diseases.