Onasemnogene Abeparvovec Gene Therapy and Risdiplam for the Treatment of Spinal Muscular Atrophy in Thailand: A Cost-Utility Analysis
While onasemnogene and risdiplam exceeded the value-for-money threshold of the Thai healthcare system, they yielded the highest QALY gains among all approved medications. Policy-makers should incorporate various pieces of evidence alongside the cost-effectiveness results for rare diseases with costly drugs. Additionally, cost-effectiveness findings are useful for price negotiations and alternative financial funding, which allows policy-makers to seek solutions to ensure patient access, aligning with universal health coverage principles in Thailand.
Authors
- Sarayuth Khuntha (ORCID: https://orcid.org/0000-0002-6430-0630)
- Oranee Sanmaneechai (ORCID: https://orcid.org/0000-0002-4557-0387)
- Varalak Srinonprasert (ORCID: https://orcid.org/0000-0001-5311-7657)
- Juthamas Prawjaeng (ORCID: https://orcid.org/0000-0003-3356-7890)
- Pattara Leelahavarong (ORCID: https://orcid.org/0000-0002-9584-0293)
- Kunnatee Ponragdee (ORCID: https://orcid.org/0000-0001-5130-7545)
Institutions
- Siriraj Hospital (TH)
- Mahidol University (TH)
Publication Details
- Journal
- Applied Health Economics and Health Policy
- Published
- 2024-09-27
- DOI
- https://doi.org/10.1007/s40258-024-00915-y
- Citations
- 3
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 1.31
Funders
- Mahidol University
- Faculty of Medicine Siriraj Hospital, Mahidol University