History of Orphan Drug Regulation-United States and Beyond

Clin Pharmacol Ther. 2016 Oct;100(4):342-3. doi: 10.1002/cpt.426. Epub 2016 Aug 26.


The US Orphan Drug Act, passed in 1982, was the first orphan drug legislation in the world. It is a law based on economic incentives making it financially possible for pharmaceutical firms to develop products for small patient populations. Since passage, many additional countries have developed orphan drug programs and many pharmaceutical firms have developed around the orphan program. Today, more than 500 drugs for rare diseases have been developed in the United States.

Publication types

  • Historical Article

MeSH terms

  • Drug Approval / legislation & jurisprudence*
  • Drug Industry / economics
  • History, 20th Century
  • Humans
  • Legislation, Drug / history*
  • Orphan Drug Production / history
  • Orphan Drug Production / legislation & jurisprudence*
  • Rare Diseases / drug therapy*
  • United States