Registries for orphan drugs: generating evidence or marketing tools?

Orphanet J Rare Dis. 2020 Sep 3;15(1):235. doi: 10.1186/s13023-020-01519-0.


Independent disease registries for pre-and post-approval of novel treatments for rare diseases are increasingly important for healthcare professionals, patients, regulators and the pharmaceutical industry. Current registries for rare diseases to evaluate orphan drugs are mainly set up and owned by the pharmaceutical industry which leads to unacceptable conflicts of interest. To ensure independence from commercial interests, disease registries should be set up and maintained by healthcare professionals and patients. Public funding should be directed towards an early establishment of international registries for orphan diseases, ideally well before novel treatments are introduced. Regulatory bodies should insist on the use of data from independent disease registries rather than company driven, drug-oriented registries.

Keywords: Disease registries; Orphan drugs; Real world data; Registries.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Drug Approval
  • Drug Industry
  • Humans
  • Marketing
  • Orphan Drug Production*
  • Rare Diseases* / drug therapy
  • Registries