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

Author

Listed:
  • 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)

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.

Suggested Citation

  • Anirban Basu & Simu K. Thomas & Richard H. Chapman & Jason Spangler, 2025. "HTA Evidence in Rare Diseases: Just Rare or Also Special?," PharmacoEconomics, Springer, vol. 43(11), pages 1271-1279, November.
    • Handle: RePEc:spr:pharme:v:43:y:2025:i:11:d:10.1007_s40273-025-01538-4
    DOI: 10.1007/s40273-025-01538-4
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