The challenges of orphan drugs and orphan diseases: real and imagined

Clin Pharmacol Ther. 2012 Aug;92(2):151-3. doi: 10.1038/clpt.2012.72.


The Orphan Drug Act of 1983 in the United States and similar legislation in Europe in 1999 provided incentives for companies to develop and sell medicines for diseases with a small market. In this Commentary, we outline the European position on the regulation of orphan drugs and explain where it differs from the regulation in the United States.

MeSH terms

  • Europe
  • Humans
  • Legislation, Drug / economics*
  • Orphan Drug Production / economics*
  • Orphan Drug Production / legislation & jurisprudence
  • Rare Diseases / drug therapy
  • Rare Diseases / economics*
  • United States