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

Kavisha Jayasundara, Aidan Hollis, Murray Krahn, Muhammad Mamdani, Jeffrey S. Hoch and Paul Grootendorst; Orphanet Journal of Rare Diseases 201914:12 doi:10.1186/s13023-018-0990-4

Using publicly available data, we estimated the differences in trial characteristics and clinical development costs with 100 orphan and 100 non-orphan drugs. 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. More discussion is needed to better align on which cost components should be included in research and development costs for pharmaceuticals.

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