Orphan Drugs and Their Impact on Pharmaceutical Development

Trends Pharmacol Sci. 2018 Jun;39(6):525-535. doi: 10.1016/j.tips.2018.03.003.


High levels of productivity, with an increasing number of approvals for new molecular entities (NMEs) by the FDA during the past decade, have coincided with the emergence of innovative drugs for treatments of rare diseases that have utilized the FDA orphan drug program. Since 2000, NMEs with orphan designation encompass a significant portion of approved drugs and constitute about 80% of the approved drugs that have established novel human genome-encoded products in recent years. Biological approvals are also expanding, with 40% of the approved biological agents having orphan designation. This trend illustrates a pivot within the pharmaceutical industry: from research programs that focus on canonical blockbuster indications and targets, towards the establishment of new treatments for rare and difficult to treat diseases.

Keywords: biologicals; drug targets; innovative therapies; orphan drugs; rare diseases.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Antineoplastic Agents / classification
  • Antineoplastic Agents / therapeutic use*
  • Clinical Trials as Topic
  • Drug Discovery / economics
  • Drug Discovery / trends*
  • Humans
  • Neoplasms / drug therapy*
  • Orphan Drug Production / classification*
  • Orphan Drug Production / economics
  • Rare Diseases / drug therapy*


  • Antineoplastic Agents