目的:本研究旨在系统地收集有关成本效益分析的数据,以评估治疗I型和II型脊髓性肌萎缩症的技术并评估其建议。
方法:在4个数据库中进行结构化电子搜索。此外,进行了补充的手动搜索。评估Nusinersen的完整经济研究,risdiplam,无性子基因(OA),并从卫生系统的角度选择了最佳支持治疗(BST)。将增量成本效益比与各种阈值进行比较以进行分析。该审查在PROSPERO(CRD42022365391)中事先注册。
结果:分析中包括20项研究。它们都在2017年至2022年之间发布,代表了8个国家的建议。大多数研究采用5、6或10状态马尔可夫模型。一些作者参与了多项研究。评估了四种技术:BST(N=14),nusinersen(N=19),risdiplam(N=5),和OA(N=9)。OA,risdiplam,与BST相比,nusinersen被认为效率低下。与nusinersen相比,Risdiplam和OA通常被认为具有成本效益。因为Nusinersen不是一种具有成本效益的药物,没有建议可以从这个结果。在2项研究中比较了Risdiplam和OA,结果相反。
结论:Nusinersen,risdiplam,和OA正在世界范围内被采用作为脊髓性肌萎缩的治疗方法。尽管如此,药物经济学分析表明,与BST相比,该技术的成本效益不高.缺乏对利司普坦和OA的对照研究阻碍了关于其面对面比较的任何结论。
OBJECTIVE: This study aims to systematically collect data on cost-effectiveness analyses that assess technologies to treat type I and II spinal muscular atrophy and evaluate their recommendations.
METHODS: A structured electronic search was conducted in 4 databases. Additionally, a complementary manual search was conducted. Complete economic studies that evaluated nusinersen, risdiplam, onasemnogene abeparvovec (OA), and the best support therapy (BST) from the health system\'s perspective were selected. The incremental cost-effectiveness ratios were compared with various thresholds for the analysis. The
review was registered a priori in PROSPERO (CRD42022365391).
RESULTS: Twenty studies were included in the analyses. They were all published between 2017 and 2022 and represent the recommendations in 8 countries. Most studies adopted 5, 6, or 10-state Markov models. Some authors took part in multiple studies. Four technologies were evaluated: BST (N = 14), nusinersen (N = 19), risdiplam (N = 5), and OA (N = 9). OA, risdiplam, and nusinersen were considered inefficient compared with the BST. Risdiplam and OA were generally regarded as cost-effective when compared with nusinersen. Because nusinersen is not a cost-effective drug, no recommendation can be derived from this result. Risdiplam and OA were compared in 2 studies that presented opposite results.
CONCLUSIONS: Nusinersen, risdiplam, and OA are being adopted worldwide as a treatment for spinal muscular atrophy. Despite that, the pharmacoeconomic analyses show that the technologies are not cost-effective compared with the BST. The lack of controlled studies for risdiplam and OA hamper any conclusions about their face-to-face comparison.