Study: Orphan Drug Trial Costs are Half of Non-orphan Drugs

A new study on drug development costs has found that clinical costs per approved orphan drug were half that of a non-orphan drugs. Appearing in the 1/10 issue of the Orphanet Journal of Rare Diseases, the study examined the differences in trial characteristics and clinical development costs with 100 orphan and 100 non-orphan drugs.

The University of Toronto researchers said that the orphan drug group had a “higher proportion of Phase 2 trials while the non-orphan group had the highest proportion of trials in Phase 3. This implies that Phase 2 trials are used as pivotal trials for orphan drugs and some orphan drugs may not even be tested in a Phase 3 setting.”

The researchers acknowledged that more research and discussion are needed to “better align on which cost components should be included in research and development costs for pharmaceuticals.”