HTA Evidence in Rare Diseases: Just Rare or Also Special?

Anirban Basu, Simu K. Thomas (), Richard H. Chapman and Jason Spangler
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Anirban Basu: University of Washington
Simu K. Thomas: Alexion, AstraZeneca Rare Disease
Richard H. Chapman: Center for Innovation and Value Research
Jason Spangler: Center for Innovation and Value Research

PharmacoEconomics, 2025, vol. 43, issue 11, No 2, 1279 pages

Abstract: Abstract Manufacturers of orphan drugs face several obstacles in meeting health technology assessment requirements because of poor availability of natural history data, small sample sizes, single-arm trials, and a paucity of established disease-specific endpoints. There is a need for specific considerations and modified approaches in health technology assessments that would account for the challenges in orphan drug development. Multistakeholder collaborations can benefit patients, their families, and the broader society and reduce the inequity faced by patients with rare diseases.

Date: 2025
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DOI: 10.1007/s40273-025-01538-4

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