R. Elango

Orphan drug regulation: a global comparison of the United States Food and Drug Administration, European Medicines Agency, and Japanese Pharmaceuticals and Medical Devices Agency approaches.

OBJECTIVE To perform a comparative review of orphan drug policies and designation criteria implemented by the US Food and Drug Administration, the European Medicines Agency, and Japan's Pharmaceuticals and Medical Devices Agency, assessing their influence on development, approval, and access. METHOD A targeted review...