Skip to main content
main-content

01.12.2019 | Research | Ausgabe 1/2019 Open Access

Orphanet Journal of Rare Diseases 1/2019

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

Zeitschrift:
Orphanet Journal of Rare Diseases > Ausgabe 1/2019
Autoren:
Kavisha Jayasundara, Aidan Hollis, Murray Krahn, Muhammad Mamdani, Jeffrey S. Hoch, Paul Grootendorst
Wichtige Hinweise

Electronic supplementary material

The online version of this article (https://​doi.​org/​10.​1186/​s13023-018-0990-4) contains supplementary material, which is available to authorized users.

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.
Zusatzmaterial
Literatur
Über diesen Artikel

Weitere Artikel der Ausgabe 1/2019

Orphanet Journal of Rare Diseases 1/2019 Zur Ausgabe