Onasemnogene Abeparvovec Gene Therapy and Risdiplam for the Treatment of Spinal Muscular Atrophy in Thailand: A Cost-Utility Analysis.

IF 3.1 4区医学 Q1 ECONOMICS Applied Health Economics and Health Policy Pub Date : 2024-09-27 DOI:10.1007/s40258-024-00915-y

Sarayuth Khuntha, Juthamas Prawjaeng, Kunnatee Ponragdee, Oranee Sanmaneechai, Varalak Srinonprasert, Pattara Leelahavarong

{"title":"Onasemnogene Abeparvovec Gene Therapy and Risdiplam for the Treatment of Spinal Muscular Atrophy in Thailand: A Cost-Utility Analysis.","authors":"Sarayuth Khuntha, Juthamas Prawjaeng, Kunnatee Ponragdee, Oranee Sanmaneechai, Varalak Srinonprasert, Pattara Leelahavarong","doi":"10.1007/s40258-024-00915-y","DOIUrl":null,"url":null,"abstract":"Objectives: Caring 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.Methods: A 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.Results: For 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.Conclusions: 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.","PeriodicalId":8065,"journal":{"name":"Applied Health Economics and Health Policy","volume":" ","pages":""},"PeriodicalIF":3.1000,"publicationDate":"2024-09-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Applied Health Economics and Health Policy","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s40258-024-00915-y","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"ECONOMICS","Score":null,"Total":0}

引用次数: 0

Abstract

Objectives: Caring 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.

Methods: A 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.

Results: For 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.

Conclusions: 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.

查看原文

微信好友朋友圈 QQ好友复制链接

本刊更多论文

泰国用于治疗脊髓性肌肉萎缩症的 Onasemnogene Abeparvovec 基因疗法和 Risdiplam：成本效用分析》。

目标：脊髓性肌萎缩症（SMA）是一种罕见的遗传性疾病，为脊髓性肌萎缩症患者的护理工作带来了巨大的挑战。本研究评估了泰国治疗 SMA 的 onasemnogene abeparvovec-xioi 基因疗法和 risdiplam 的成本效用：方法：采用马尔可夫模型分析这些疗法与标准疗法相比，治疗有症状的 1 型和 2-3 型 SMA 的终生成本和疗效。1型SMA患者接受onasemnogene或利钠嗪治疗，2-3型SMA患者接受利钠嗪治疗。有关疾病进展和医疗费用的数据来自医院数据库，而治疗效果则基于临床试验。对患者和护理人员的访谈提供了非医疗费用和公用事业的数据。我们进行了基础病例成本效益分析和敏感性分析，增量成本效益比（ICER）以每质量调整生命年（QALY）收益美元（USD）计算，支付意愿阈值为每质量调整生命年收益 4444 美元：对于 1 型 SMA，onasemnogene 和 risdiplam 的 ICER 分别为 163,102 美元和 158,357 美元/QALY。对于 SMA 2-3 型，利斯地普仑的 ICER 为 496,704 美元/QALY gained：onasemnogene和risdiplam超出了泰国医疗保健系统的性价比门槛，但在所有获批药物中，它们的QALY收益最高。对于使用昂贵药物的罕见病，政策制定者应将各种证据与成本效益结果结合起来。此外，成本效益结果还有助于价格谈判和替代性财政资助，使政策制定者能够根据泰国全民医保的原则寻求解决方案，确保患者能够获得药物。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文去求助

来源期刊

Applied Health Economics and Health Policy Economics, Econometrics and Finance-Economics and Econometrics

CiteScore

6.10

自引率

2.80%

发文量

期刊介绍： Applied Health Economics and Health Policy provides timely publication of cutting-edge research and expert opinion from this increasingly important field, making it a vital resource for payers, providers and researchers alike. The journal includes high quality economic research and reviews of all aspects of healthcare from various perspectives and countries, designed to communicate the latest applied information in health economics and health policy. While emphasis is placed on information with practical applications, a strong basis of underlying scientific rigor is maintained.