利斯地普兰与诺森森治疗中国脊髓性肌萎缩症的成本-效果分析。

IF 1.5 4区医学 Q3 HEALTH CARE SCIENCES & SERVICES

Expert Review of Pharmacoeconomics & Outcomes Research Pub Date : 2025-07-01 Epub Date: 2025-05-08 DOI:10.1080/14737167.2025.2499720

Yueyang Huang, Hongmei Yuan, Zhe Huang

{"title":"利斯地普兰与诺森森治疗中国脊髓性肌萎缩症的成本-效果分析。","authors":"Yueyang Huang, Hongmei Yuan, Zhe Huang","doi":"10.1080/14737167.2025.2499720","DOIUrl":null,"url":null,"abstract":"Background: As a rare disease with the largest number of cases in China, spinal muscular atrophy (SMA) causes severe motor deficits as well as multi-system organ damage. Some potent drugs for SMA, such as Nusinersen (NU) and Risdiplam (RI) represented by targeted survival motor neuron gene therapy, have become a new trend in the treatment of SMA. RI has gradually attracted attention due to its ease of administration, safety, and efficacy.Objective: The study was to analyze the cost-effectiveness of RI and NU for the treatment of SMA in China from the perspective of the healthcare system.Methods: We constructed a five-state Markov model based on the different stages of SMA. The willingness-to-pay threshold was chosen to be 1-3 times the gross domestic product (GDP) per capita. The base-case analysis was used to calculate the incremental cost-effectiveness ratio (ICER), and one-way and probabilistic sensitivity analyses were performed.Results: The ICER value was ¥132,402.10/QALY, which was in the range of 1-3 times GDP per capita. Sensitivity analysis results showed the cost of RI and NU had a significant effect on ICER as well as proving the stability of results.Conclusions: RI is a cost-effective option compared to NU in SMA treatment.","PeriodicalId":12244,"journal":{"name":"Expert Review of Pharmacoeconomics & Outcomes Research","volume":" ","pages":"943-953"},"PeriodicalIF":1.5000,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Cost-effectiveness analysis of Risdiplam and Nusinersen for the treatment of Chinese patients with spinal muscular atrophy.\",\"authors\":\"Yueyang Huang, Hongmei Yuan, Zhe Huang\",\"doi\":\"10.1080/14737167.2025.2499720\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"Background: As a rare disease with the largest number of cases in China, spinal muscular atrophy (SMA) causes severe motor deficits as well as multi-system organ damage. Some potent drugs for SMA, such as Nusinersen (NU) and Risdiplam (RI) represented by targeted survival motor neuron gene therapy, have become a new trend in the treatment of SMA. RI has gradually attracted attention due to its ease of administration, safety, and efficacy.Objective: The study was to analyze the cost-effectiveness of RI and NU for the treatment of SMA in China from the perspective of the healthcare system.Methods: We constructed a five-state Markov model based on the different stages of SMA. The willingness-to-pay threshold was chosen to be 1-3 times the gross domestic product (GDP) per capita. The base-case analysis was used to calculate the incremental cost-effectiveness ratio (ICER), and one-way and probabilistic sensitivity analyses were performed.Results: The ICER value was ¥132,402.10/QALY, which was in the range of 1-3 times GDP per capita. Sensitivity analysis results showed the cost of RI and NU had a significant effect on ICER as well as proving the stability of results.Conclusions: RI is a cost-effective option compared to NU in SMA treatment.\",\"PeriodicalId\":12244,\"journal\":{\"name\":\"Expert Review of Pharmacoeconomics & Outcomes Research\",\"volume\":\" \",\"pages\":\"943-953\"},\"PeriodicalIF\":1.5000,\"publicationDate\":\"2025-07-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Expert Review of Pharmacoeconomics & Outcomes Research\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1080/14737167.2025.2499720\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2025/5/8 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q3\",\"JCRName\":\"HEALTH CARE SCIENCES & SERVICES\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Expert Review of Pharmacoeconomics & Outcomes Research","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1080/14737167.2025.2499720","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/5/8 0:00:00","PubModel":"Epub","JCR":"Q3","JCRName":"HEALTH CARE SCIENCES & SERVICES","Score":null,"Total":0}

引用次数: 0

摘要

背景：脊髓性肌萎缩症（spinal muscular atrophy， SMA）是一种罕见的疾病，在中国发病率最高，可导致严重的运动障碍和多系统器官损害。以靶向生存运动神经元基因治疗为代表的Nusinersen （NU）、Risdiplam （RI）等治疗SMA的强效药物已成为治疗SMA的新趋势。国际扶轮因其易于管理、安全和有效而逐渐引起人们的注意。目的：从卫生保健系统的角度分析RI和NU治疗SMA在中国的成本-效果。方法：基于SMA的不同阶段，构建五态马尔可夫模型。支付意愿的门槛被定为人均国内生产总值（GDP）的1-3倍。基本情况分析用于计算增量成本-效果比（ICER），并进行单向和概率敏感性分析。结果：ICER值为132,402.10元/QALY，在人均GDP的1 ~ 3倍范围内。敏感性分析结果显示，RI和NU的成本对ICER有显著影响，证明了结果的稳定性。结论：与NU相比，RI在SMA治疗中是一种具有成本效益的选择。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

查看原文本刊更多论文

Cost-effectiveness analysis of Risdiplam and Nusinersen for the treatment of Chinese patients with spinal muscular atrophy.

Background: As a rare disease with the largest number of cases in China, spinal muscular atrophy (SMA) causes severe motor deficits as well as multi-system organ damage. Some potent drugs for SMA, such as Nusinersen (NU) and Risdiplam (RI) represented by targeted survival motor neuron gene therapy, have become a new trend in the treatment of SMA. RI has gradually attracted attention due to its ease of administration, safety, and efficacy.

Objective: The study was to analyze the cost-effectiveness of RI and NU for the treatment of SMA in China from the perspective of the healthcare system.

Methods: We constructed a five-state Markov model based on the different stages of SMA. The willingness-to-pay threshold was chosen to be 1-3 times the gross domestic product (GDP) per capita. The base-case analysis was used to calculate the incremental cost-effectiveness ratio (ICER), and one-way and probabilistic sensitivity analyses were performed.

Results: The ICER value was ¥132,402.10/QALY, which was in the range of 1-3 times GDP per capita. Sensitivity analysis results showed the cost of RI and NU had a significant effect on ICER as well as proving the stability of results.

Conclusions: RI is a cost-effective option compared to NU in SMA treatment.

求助全文

通过发布文献求助，成功后即可免费获取论文全文。去求助

来源期刊

Expert Review of Pharmacoeconomics & Outcomes Research HEALTH CARE SCIENCES & SERVICES-PHARMACOLOGY & PHARMACY

CiteScore

4.00

自引率

4.30%

发文量

审稿时长

6-12 weeks

期刊介绍： Expert Review of Pharmacoeconomics & Outcomes Research (ISSN 1473-7167) provides expert reviews on cost-benefit and pharmacoeconomic issues relating to the clinical use of drugs and therapeutic approaches. Coverage includes pharmacoeconomics and quality-of-life research, therapeutic outcomes, evidence-based medicine and cost-benefit research. All articles are subject to rigorous peer-review. The journal adopts the unique Expert Review article format, offering a complete overview of current thinking in a key technology area, research or clinical practice, augmented by the following sections: Expert Opinion – a personal view of the data presented in the article, a discussion on the developments that are likely to be important in the future, and the avenues of research likely to become exciting as further studies yield more detailed results Article Highlights – an executive summary of the author’s most critical points.