Khuntha Sarayuth, Prawjaeng Juthamas, Ponragdee Kunnatee, Sanmaneechai Oranee, Srinonprasert Varalak, Leelahavarong Pattara
Mahidol University Health Technology Assessment Program, Mahidol University, Bangkok, Thailand.
Siriraj Health Policy Unit, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand.
Appl Health Econ Health Policy. 2025 Mar;23(2):277-290. doi: 10.1007/s40258-024-00915-y. Epub 2024 Sep 27.
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.
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.
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.
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.
照顾患有脊髓性肌萎缩症（SMA）的个体，这是一种罕见的遗传性疾病，对经济和医疗保健系统构成了巨大挑战。本研究评估了onasemnogene abeparvovec-xioi基因疗法和利司扑兰在泰国治疗SMA的成本效益。
使用马尔可夫模型分析这些治疗方法与有症状的1型和2-3型SMA标准治疗相比的终身成本和结果。1型SMA患者接受onasemnogene或利司扑兰中的一种治疗，而2-3型SMA患者接受利司扑兰治疗。疾病进展和医疗成本数据来自医院数据库，而治疗效果基于临床试验。对患者和护理人员的访谈提供了非医疗成本和效用的数据。进行了基础病例成本效益和敏感性分析，计算了每获得一个质量调整生命年（QALY）的增量成本效益比（ICER），以美元（USD）为单位，支付意愿阈值为每获得一个QALY 4444美元。
对于1型SMA，onasemnogene和利司扑兰的ICER分别为每获得一个QALY 163,102美元和158,357美元。对于2-3型SMA，利司扑兰的ICER为每获得一个QALY 496,704美元。
虽然onasemnogene和利司扑兰超过了泰国医疗保健系统的性价比阈值，但它们在所有获批药物中产生了最高的QALY增益。政策制定者应将各种证据与昂贵药物治疗罕见病的成本效益结果结合起来。此外，成本效益结果对于价格谈判和替代资金筹集很有用，这使政策制定者能够寻求解决方案以确保患者能够获得治疗，符合泰国全民健康覆盖原则。
