Unidade de Avaliação de Tecnologias em Saúde, Hospital Alemão Oswaldo Cruz, São Paulo, SP, Brasil.
Universidade Federal do Rio Grande do Sul, Porto Alegre - RS, Brasil.
Value Health Reg Issues. 2024 Mar;40:108-117. doi: 10.1016/j.vhri.2023.11.004. Epub 2024 Jan 5.
This study aimed to evaluate the cost-effectiveness of the onasemnogene abeparvovec in relation to nusinersen and risdiplam in the treatment of spinal muscular atrophy type 1 from the perspective of the Brazilian Unified Health System.
A Markov model was built on a lifetime horizon. Short-term data were obtained from clinical trials of the technologies and from published cohort survival curves (long term). Costs were measured in current 2022 local currency (R$) values and benefits in quality-adjusted life-years (QALYs). Utility values were derived from type 1 spinal muscular atrophy literature, whereas costs related to technologies and maintenance care in each health state were obtained from official sources of reimbursement in Brazil. Deterministic and probabilistic, as well as scenario, sensitivity analyses were performed.
Compared with the less costly strategy (nusinersen), the use of onasemnogene abeparvovec resulted in an incremental cost of R$2.468.448,06 ($975 671.169 - purchasing power parity [PPP]) and a 3-QALY increment and incremental cost-effectiveness ratio of R$742.890,92 ($293 632.774 - PPP)/QALY. Risdiplam had an extended dominance from other strategies, resulting in an incremental cost-effectiveness ratio of R$926.586,22 ($366 239.612 - PPP)/QALY compared with nusinersen. Sensitivity analysis showed a significant impact of the follow-up time of the cohort and the cost of acquiring onasemnogene abeparvovec.
Over a lifetime horizon, onasemnogene abeparvovec seems to be a potentially more effective option than nusinersen and risdiplam, albeit with an incremental cost. Such a trade-off should be weighed in efficiency criteria during decision making and outcome monitoring from the perspective of the Brazilian Unified Health System.
本研究旨在从巴西统一卫生系统的角度评估依洛硫酸酯酶纳在治疗 1 型脊髓性肌萎缩症方面相对于那西妥珠单抗和利司扑兰的成本效益。
在终身时间范围内构建马尔可夫模型。短期数据来自技术的临床试验和已发表的队列生存曲线（长期）获得。成本以 2022 年当地货币（雷亚尔）的当前价值衡量，效益以质量调整生命年（QALY）衡量。效用值来自 1 型脊髓性肌萎缩症文献，而每种健康状态下与技术和维持护理相关的成本则来自巴西的官方报销来源。进行了确定性和概率性分析，以及情景和敏感性分析。
与成本较低的策略（那西妥珠单抗）相比，使用依洛硫酸酯酶纳导致增量成本为 24684480.66 雷亚尔（975671.169 美元-购买力平价 [PPP]）和 3 QALY 增量和增量成本效益比为 74289092.92 雷亚尔（29363277.47 美元-PPP）/QALY。利司扑兰相对于其他策略具有扩展的优势，与那西妥珠单抗相比，增量成本效益比为 92658622.22 雷亚尔（366239612 美元-PPP）/QALY。敏感性分析表明，队列随访时间和依洛硫酸酯酶纳获取成本对结果有重大影响。
在终身时间范围内，依洛硫酸酯酶纳似乎比那西妥珠单抗和利司扑兰更有效，尽管成本有所增加。在巴西统一卫生系统的决策和结果监测的效率标准中，应该权衡这种权衡取舍。
