Orphan drugs

Juan Manuel Fontanet Sacristán, Josep Torrent-Farnell

Research output: Contribution to journalArticleResearchpeer-review


© 2018 CSIC. Orphan drugs are intended for the treatment of diseases of low prevalence, also known as rare diseases. Research and development into new therapies for this heterogeneous group of diseases presents a number of well-recognized difficulties. One of these is that the small number of patients affected jeopardizes the economic return on the investment made by the pharmaceutical industry. For this reason, in the year 2000 the European Union brought out a specific European Regulation to promote and encourage the development of these therapies. After more than a decade, the results reveal a positive effect of the approval of that regulation. Currently, more than 1300 compounds have been designated orphan drugs, of which over 90 have already obtained marketing authorization. The voice of the patient has been key to producing this change and programs and research consortia promoted by the EU promise an even brighter future.
Original languageEnglish
Article numbera466
Issue number789
Publication statusPublished - 1 Jul 2018


  • Designation
  • Incentives
  • Investigation
  • Orphan drug
  • Rare disease

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