Modifying the Criteria for Granting Orphan Drug Market Exclusivity

Mariana P. Socal, Sonal Parasrampuria, Gerard F. Anderson

Research output: Contribution to journalArticlepeer-review

1 Scopus citations


Objectives: To examine policy options to deny orphan drug exclusivity after drugs exceed a target population of 200 000 across all orphan indications (combined prevalence threshold) or once drugs receive a nonorphan approval (market approval threshold). Methods: Retrospective analysis of drugs with 2 or more orphan approvals from 1983 to July 01, 2017 examining prevalence of orphan indications and approval years of orphan and nonorphan indications. Characteristics of drugs crossing either threshold are described. A budget impact analysis of Medicare and Marketscan® claims databases estimated potential savings from generic or biosimilar entry as a result of foregone market exclusivity periods determined by these policies. Results: Out of 86 drugs with 2 or more orphan approvals, 21 drugs would be denied orphan drug exclusivity periods under the prevalence threshold and 18 drugs would be denied orphan drug exclusivity periods under the market approval threshold. Drugs with orphan approvals after 2010 were more likely to be denied orphan drug exclusivity. In 2017, Medicare could have saved about $2 billion on 8 drugs under the prevalence threshold policy and $1.3 billion on 12 drugs under the market approval threshold policy). Private insurers could have saved $814 and $919 million, respectively. Over half of the savings would come from 9 drugs that first entered the market for a nonorphan indication. Conclusions: Modifying the criteria for granting orphan drug exclusivity would affect a small number of orphan drugs but could generate large savings through increased competition. Other incentives such as grants or tax credits for clinical trials could be explored to incentivize research for new orphan indications for drugs that crossed either threshold.

Original languageEnglish (US)
Pages (from-to)1470-1476
Number of pages7
JournalValue in Health
Issue number11
StatePublished - Nov 2020


  • biologics
  • budget impact analysis
  • generics
  • medicare
  • orphan drug exclusivity
  • private insurers

ASJC Scopus subject areas

  • Health Policy
  • Public Health, Environmental and Occupational Health


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