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

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

Kavisha Jayasundara  1 , Aidan Hollis  2 , Murray Krahn  3   4   5 , Muhammad Mamdani  3   6   5 , Jeffrey S Hoch  3   6   5   7 , Paul Grootendorst  3
Affiliations

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

Kavisha Jayasundara et al. Orphanet J Rare Dis. .

Abstract

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.

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Conflict of interest statement

Ethics approval and consent to participate

Not Applicable

Consent for publication

Not Applicable

Competing interests

KJ is currently a full time employee at Hoffmann-la Roche. MM reports receiving honoraria for serving on Advisory Boards for Astra Zeneca, Bristol-Myers Squibb, Eli Lilly and Company, Glaxo Smith Kline, Hoffmann-la Roche, Novartis, Novo Nordisk, and Pfizer. PG, MK, AH and JSH have no conflicts of interest to report.

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Figures

Fig. 1
Fig. 1
Mean number of subjects in each trial phase for non-orphan and orphan groups. Missing = trial phase information not available
Fig. 2
Fig. 2
Study Duration for each trial phase for non-orphan and orphan groups. Missing = trial phase information not available
Fig. 3
Fig. 3
Number of subjects by trial phase for NMEs only. NMEs = New Molecular Entities
Fig. 4
Fig. 4
Study Duration by trial phase for NMEs only
See this image and copyright information in PMC

References

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