Estimating the clinical cost of drug development for orphan versus non-orphan drugs

Kavisha Jayasundara, Aidan Hollis, Murray Krahn, Muhammad Mamdani, Jeffrey S Hoch, Paul Grootendorst

Research output: Contribution to journalArticle

8 Scopus citations


Background: High orphan drug prices have gained the attention of payers and policy makers. These prices may reflect the need to recoup the cost of drug development from a small patient pool. However, estimates of the cost of orphan drug development are sparse. Methods: Using publicly available data, we estimated the differences in trial characteristics and clinical development costs with 100 orphan and 100 non-orphan drugs. Results: We found that the out-of-pocket clinical costs per approved orphan drug to be $166 million and $291 million (2013 USD) per non-orphan drug. The capitalized clinical costs per approved orphan drug and non-orphan drug were estimated to be $291 million and $412 million respectively. When focusing on new molecular entities only, we found that the capitalized clinical cost per approved orphan drug was half that of a non-orphan drug. Conclusions: More discussion is needed to better align on which cost components should be included in research and development costs for pharmaceuticals.

Original languageEnglish (US)
Article number12
JournalOrphanet Journal of Rare Diseases
Issue number1
StatePublished - Jan 10 2019



  • Cost of drug development
  • Orphan drugs
  • Rare diseases

ASJC Scopus subject areas

  • Genetics(clinical)
  • Pharmacology (medical)

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