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

Orphanet J Rare Dis. 2019 Jan 10;14(1):12. doi: 10.1186/s13023-018-0990-4.


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.

Keywords: Cost of drug development; Orphan drugs; Rare diseases.

MeSH terms

  • Drug Costs / statistics & numerical data
  • Drug Development / economics*
  • Health Expenditures / statistics & numerical data
  • Humans
  • Orphan Drug Production / economics*
  • Rare Diseases / drug therapy
  • Rare Diseases / economics