ArticleApplied health economics and health policy2025
Onasemnogene Abeparvovec Gene Therapy and Risdiplam for the Treatment of Spinal Muscular Atrophy in Thailand: A Cost-Utility Analysis.
Article in Applied health economics and health policy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers, 1 of them a synthesis that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
4 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Economic evaluations of disease-modifying therapies for spinal muscular atrophy: a systematic literature review.Orphanet journal of rare diseases · 2025Pooled it
- Addressing the Global Disparities in Access to Treatment in Spinal Muscular Atrophy.Muscle & nerve · 2026Review
- Beyond Narrative and Tabular Synthesis: Introducing an Approach-Rationale-Note (ARN) Framework for Thematic Evidence Synthesis in Economic Evaluation Reviews.PharmacoEconomics · 2026Article
- Therapeutic advances in spinal muscular atrophy: a review of clinical, safety, and economic considerations.Frontiers in pharmacology · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
Abstract
objectivesCaring for individuals with spinal muscular atrophy (SMA), a rare genetic disorder, poses tremendous challenges for the economy and healthcare system. This study evaluated the cost-utility of onasemnogene abeparvovec-xioi gene therapy and risdiplam for SMA in Thailand.
methodsA Markov model was used to analyze the lifetime costs and outcomes of these treatments compared with standard of care for symptomatic SMA types 1 and 2-3. SMA type 1 patients were treated with one of either onasemnogene or risdiplam, while SMA types 2-3 patients received risdiplam. Data on disease progression and medical costs were sourced from hospital databases, while treatment efficacy was based on clinical trials. Interviews with patients and caregivers provided data on non-medical costs and utilities. Base case cost-effectiveness and sensitivity analyses were conducted, with the incremental cost-effectiveness ratio (ICER) calculated in US dollars (USD) per quality-adjusted life year (QALY) gained, against a willingness-to-pay threshold of 4444 USD/QALY gained.
resultsFor SMA type 1, the ICERs for onasemnogene and risdiplam were 163,102 and 158,357 USD/QALY gained, respectively. For SMA types 2-3, the ICER for risdiplam was 496,704 USD/QALY gained.
conclusionsWhile 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.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.