Center for Health Policy and Technology Evaluation, Peking University Health Science Center, Beijing, 100191, China.
Orphanet J Rare Dis. 2024 Oct 28;19(1):400. doi: 10.1186/s13023-024-03381-w.
Edaravone has been widely used in amyotrophic lateral sclerosis (ALS) treatment, and a sublingual (SL) tablet has been developed to offer a more convenient alternative for injection. We present a cost-utility analysis to comprehensively evaluate the costs and health outcomes of oral and intravenous edaravone for the treatment of ALS in Chinese medical context.
Cost-utility analysis of SL tablets of edaravone versus intravenous edaravone at home was performed by constructing a 20-year Markov model of ALS stage 1-4 and death. The data were extracted from the literature with model assumptions. Typical sensitivity analysis and scenario analysis for administering SL tablets at home versus intravenous tablets at the hospital were performed.
In the base case analysis, with SL tablets and intravenous injections both at home, the model estimated an additional cost of ¥12,670.04 and an additional 0.034 QALYs over 20 years (life time) of modeling analysis, and the ICER was ¥372,648.24 per QALY. However, in the scenario of intravenous administration at the hospital, SL tablet was demonstrated dominance to intravenous injection.
Using 3 times the GDP per capita of China in 2023 as the threshold, the SL tablet edaravone was not cost-effective in the context of home treatment for both formulationst, but was dominance to intravenous injection in hospital treatment. The results highlighted the importance of treatment context for health economic analysis.
依达拉奉已广泛用于肌萎缩侧索硬化症（ALS）的治疗，现已开发出口服（SL）片剂，为注射提供更便捷的替代方案。我们进行了一项成本效用分析，以全面评估口服依达拉奉和静脉内依达拉奉在中国医疗环境下治疗 ALS 的成本和健康结果。
通过构建 1 至 4 期 ALS 及死亡的 20 年 Markov 模型，对 SL 片剂和静脉内依达拉奉进行了成本效用分析。数据取自文献，并进行了模型假设。对在家中使用 SL 片剂和在医院使用静脉内片剂进行了典型敏感性分析和情景分析。
在基础案例分析中，在家中同时使用 SL 片剂和静脉内注射，模型估计在 20 年建模分析期间增加了 12670.04 元人民币的额外成本和 0.034 个质量调整生命年（QALY），增量成本效果比（ICER）为 372648.24 元人民币/QALY。然而，在医院静脉内给药的情景下，SL 片剂表现出对静脉内注射的优势。
以 2023 年中国人均国内生产总值的 3 倍作为阈值，在家中治疗两种制剂时，SL 片剂依达拉奉均不具有成本效益，但在医院治疗时，SL 片剂优于静脉内注射。结果强调了治疗环境对健康经济分析的重要性。
