The orphan drug act restoring the mission to rare diseases

Michael G. Daniel, Timothy M. Pawlik, Amanda N. Fader, Nestor F. Esnaola, Martin A. Makary

Research output: Contribution to journalArticlepeer-review

47 Scopus citations


The Orphan Drug Act has fostered drug development for patients with rare cancers and other diseases; however, current data suggest that companies are gaming the system to use the law for mainstream drugs. We identify a pattern of pharmaceutical companies submitting drugs to the Food and Drug Administration (FDA) as orphan drugs but once approved, the drugs are used broadly off-label with the lucrative orphan drug protections and exclusivity benefits. Since the law was passed, the proportion of new FDA-approved drugs that were submitted as orphan drugs has increased with a peak last year of 41% of all FDA-approved drugs approved as orphan drugs. On the basis of the current data, we suggest that patients with rare cancers and other diseases may suffer due to dilution of the incentives and benefits. We propose reform to increase submission scrutiny, decrease benefits based on off-label use, and increase price transparency.

Original languageEnglish (US)
Pages (from-to)210-213
Number of pages4
JournalAmerican Journal of Clinical Oncology: Cancer Clinical Trials
Issue number2
StatePublished - 2016


  • Health policy
  • Orphan Drug Act
  • Orphan drugs
  • Rare diseases

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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