Systematic literature review of the economic burden of spinal muscular atrophy and economic evaluations of treatments.
Systematic literature review of the economic burden of spinal muscular atrophy and economic evaluations of treatments.
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DOI：
10.1186/s13023-021-01695-7
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发表时间：
2021-01-23
影响因子：
3.7
通讯作者：
Hiligsmann M
中科院分区：
文献类型：
--
作者：
Dangouloff T;Botty C;Beaudart C;Servais L;Hiligsmann M
脊髓性肌萎缩症（SMA）是一种罕见且具有破坏性的疾病，新的疾病缓解疗法最近已获得批准。鉴于经济因素在医疗保健决策中的重要性日益增加，本综述总结了评估 SMA 成本的研究和治疗的经济评估。根据 PRISMA 指南对截至 2020 年 9 月 15 日的 PubMed 和 Scopus 文献进行了系统回顾。九项研究报告了 SMA 患者的年度护理费用，并对 SMA 治疗的成本效益进行了六项评估。 SMA1 是 6 个月前出现症状的最常见和最严重的形式，根据不同的研究，其平均每年费用为 75,047 美元至 196,429 美元。晚发型 SMA2、SMA3 和 SMA4 的年度费用通常汇总在医疗费用估计中，变化较大，从 27,157 美元到 82,474 美元不等。治疗成本效益评估比较了 nusinersen 治疗与标准护理 (n = 3)、两种治疗（nusinersen 和 onasemnogene abeparvovec）相互比较和无药物治疗 (n = 1)、nusinersen 与 onasemnogene abeparvovec (n = 1) 以及护理标准与 nusinersen 有或没有新生儿筛查(n = 1)。与 SMA1 治疗标准相比，nusinersen 的增量成本效益比 (ICER) 范围为每个质量调整生命年 (QALY) 210,095 美元至 1,150,455 美元，onasemnogene abeparvovec 的增量成本效益比 (ICER) 范围为 32,464 美元至 251,403 美元。对于出现症状前的患者，ICER 值范围为 206,409 美元至 735,519 美元。晚发型 SMA（2、3 和 4）的 ICER 更加多样化，范围从 275,943 美元到 8,438,049 美元。本次审查证实了 SMA 患者标准护理的巨大成本负担，以及按当前价格批准的药物在症状后患者中使用时的高成本效益比。由于迄今为止进行的研究很少，因此需要对症状前和症状后的患者进行进一步的前瞻性和独立的经济研究。
Spinal muscular atrophy (SMA) is a rare and devastating condition for which new disease-modifying treatments have recently been approved. Given the increasing importance of economic considerations in healthcare decision-making, this review summarizes the studies assessing the cost of SMA and economic evaluations of treatments. A systematic review of the literature in PubMed and Scopus up to 15 September 2020 was conducted according to PRISMA guidelines. Nine studies reporting the annual cost of care of patients with SMA and six evaluations of the cost-effectiveness of SMA treatments were identified. The average annual cost of SMA1, the most frequent and severe form in which symptoms appear before the age of 6 months were similar according to the different studies, ranged from $75,047 to $196,429 per year. The yearly costs for the forms of the later-onset form, called SMA2, SMA3, and SMA4, which were usually pooled in estimates of healthcare costs, were more variable, ranging from $27,157 to $82,474. The evaluations of cost-effectiveness of treatment compared nusinersen treatment against standard of care (n = 3), two treatments (nusinersen and onasemnogene abeparvovec) against each other and no drug treatment (n = 1), nusinersen versus onasemnogene abeparvovec (n = 1), and standard of care versus nusinersen with and without newborn screening (n = 1). The incremental cost-effectiveness ratio (ICER) of nusinersen compared to standard of care in SMA1 ranged from $210,095 to $1,150,455 per quality-adjusted life years (QALY) gained and that for onasemnogene abeparvovec ranged from $32,464 to $251,403. For pre-symptomatic patients, the ICER value ranged from $206,409 to $735,519. The ICERs for later-onset forms of SMA (2, 3 and 4) were more diverse ranging from $275,943 to $8,438,049. This review confirms the substantial cost burden of standard of care for SMA patients and the high cost-effectiveness ratios of the approved drugs at the current price when delivered in post-symptomatic patients. Since few studies have been conducted so far, there is a need for further prospective and independent economic studies in pre- and post-symptomatic patients.
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影响因子：
2.8
作者：
Dangouloff, Tamara;Burghes, Arthur;Servais, Laurent
通讯作者：
Servais, Laurent
影响因子：
3.2
作者：
Darba, Josep
通讯作者：
Darba, Josep
影响因子：
9.9
作者：
Finkel, Richard S.;McDermott, Michael P.;De Vivo, Darryl C.
通讯作者：
De Vivo, Darryl C.
DOI：
10.1016/j.jpeds.2020.07.033
发表时间：
2020-12
期刊：
The Journal of pediatrics
影响因子：
--
作者：
Jalali A;Rothwell E;Botkin JR;Anderson RA;Butterfield RJ;Nelson RE
通讯作者：
Nelson RE
影响因子：
2.8
作者：
De Vivo, Darryl C.;Bertini, Enrico;Farwell, Wildon
通讯作者：
Farwell, Wildon