Challenges and Hurdles to Business as Usual in Drug Development for Treatment of Rare Diseases

Clin Pharmacol Ther. 2016 Oct;100(4):339-41. doi: 10.1002/cpt.422. Epub 2016 Aug 16.


Only 10-15 first-in-class new medicines are approved each year by the global pharmaceutical industry for all diseases, of which less than a third is for rare (orphan) diseases. The drug discovery processes to identify rare and common diseases are similar, suggesting it will be impossible to discover new drugs for even a small fraction of the rare diseases using the current paradigm. Different approaches are required to address this large unmet medical need.

MeSH terms

  • Drug Discovery / methods*
  • Drug Industry / methods*
  • Humans
  • Orphan Drug Production / methods*
  • Rare Diseases / drug therapy*