Department of Economics, Universidade Federal de Minas Gerais, Belo Horizonte, Minas Gerais, Brazil; School of Business, University of Southern Queensland, Toowoomba, Queensland, Australia.
Department of Economics, Universidade Federal de Minas Gerais, Belo Horizonte, Minas Gerais, Brazil.
Value Health Reg Issues. 2024 Jul;42:100985. doi: 10.1016/j.vhri.2024.02.002. Epub 2024 Apr 26.
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.
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).
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.
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.
本研究旨在系统收集评估治疗 I 型和 II 型脊髓性肌萎缩症技术的成本效益分析数据，并评估其推荐意见。
在 4 个数据库中进行了结构化电子检索。此外，还进行了补充的手动检索。选择了从卫生系统角度评估nusinersen、risdiplam、onasemnogene abeparvovec（OA）和最佳支持治疗（BST）的完整经济研究。将增量成本效益比与各种分析阈值进行了比较。该综述已事先在 PROSPERO（CRD42022365391）中进行了注册。
20 项研究纳入分析。这些研究均发表于 2017 年至 2022 年之间，代表了 8 个国家的推荐意见。大多数研究采用了 5、6 或 10 状态 Markov 模型。一些作者参与了多项研究。共评估了 4 种技术：BST（N=14）、nusinersen（N=19）、risdiplam（N=5）和 OA（N=9）。与 BST 相比，OA、risdiplam 和 nusinersen 被认为是无效的。与 nusinersen 相比，risdiplam 和 OA 通常被认为是具有成本效益的。由于 nusinersen 不是一种具有成本效益的药物，因此无法从该结果中得出任何推荐意见。risdiplam 和 OA 在 2 项研究中进行了比较，这两项研究得出了相反的结果。
nusinersen、risdiplam 和 OA 已在全球范围内被采用作为脊髓性肌萎缩症的治疗方法。尽管如此，药物经济学分析表明，与 BST 相比，这些技术没有成本效益。 risdiplam 和 OA 缺乏对照研究，阻碍了对其直接比较的任何结论。
