Cost-Effectiveness of Nusinersen and Universal Newborn Screening for Spinal Muscular Atrophy.
Cost-Effectiveness of Nusinersen and Universal Newborn Screening for Spinal Muscular Atrophy.
复制标题
DOI：
10.1016/j.jpeds.2020.07.033
复制
发表时间：
2020-12
期刊：
影响因子：
--
通讯作者：
Nelson RE
中科院分区：
文献类型：
--
作者：
Jalali A;Rothwell E;Botkin JR;Anderson RA;Butterfield RJ;Nelson RE
目的：评估在进行新生儿普遍筛查和不进行新生儿普遍筛查的情况下，使用努赛生治疗婴儿型脊肌萎缩症(SMA)的成本-效果。利用临床试验数据和美国的流行病学和成本数据建立了马尔科夫模型。研究的主要干预措施是筛查环境中的努西那森治疗，非筛查环境中的努西那森治疗，以及标准护理。分析是从社会角度进行的。与不筛查和不治疗相比，接受筛查的努西那森的增量成本-效果比(ICER)为每节省一个无事件生命年(LY)330,558美元。未经筛查的Nusinesen治疗的ICER为每个无事件LY节省508,481美元。为了让带有筛查的Nusinesen以每节省50,000美元的支付意愿(WTP)门槛具有成本效益，价格需要为每剂23,361美元，不到目前价格125,000美元的五分之一。来自FORTURE试验的初步数据表明，与我们的基本结果相比，预期节省的LYS提高了85.7%。在概率敏感性分析中，在500,000美元的WTP门槛下，93%的情况下Nusinesen和筛查是首选策略。当Nusinesen可用时，SMA的普遍新生儿筛查为付款人和患者提供了更好的经济价值。
To evaluate the cost-effectiveness of nusinersen with and without universal newborn screening for infantile-onset spinal muscular atrophy (SMA). Markov model using data from clinical trials with U.S. epidemiologic and cost data was developed. Primary interventions studied were nusinersen treatment in a screening setting, nusinersen treatment in a non-screening setting, and standard care. Analysis was conducted from a societal perspective. Relative to no screening and no treatment, the incremental cost-effectiveness ratio (ICER) for nusinersen with screening was $330,558 per event-free life year saved (LY). The ICER for nusinersen treatment without screening was $508,481 per event-free LY saved. In order for nusinersen with screening to be cost-effective at a willingness-to-pay (WTP) threshold of $50,000 per event-free LY saved, the price would need to be $23,361 per dose, less than one-fifth its current price of $125,000. Preliminary data from the NURTURE trial indicated an 85.7% improvement in expected LYs saved compared with our base results. In probabilistic sensitivity analysis, nusinersen and screening was a preferred strategy 93% of the time at a $500,000 WTP threshold. Universal newborn screening for SMA provides improved economic value for payers and patients when nusinersen is available.
登录
查看更多内容
影响因子：
2.8
作者：
Eagle, M;Baudouin, SV;Bushby, K
通讯作者：
Bushby, K
影响因子：
3.3
作者：
Lochmuller, Hanns;Evans, David;Wilson, Petra
通讯作者：
Wilson, Petra
影响因子：
8
作者：
Chung, BHY;Wong, VCN;Ip, P
通讯作者：
Ip, P
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.
影响因子：
9.8
作者：
Little, Sarah E.;Janakiraman, Vanitha;Caughey, Aaron B.
通讯作者：
Caughey, Aaron B.