期刊
JOURNAL OF PEDIATRICS
卷 227, 期 -, 页码 274-+出版社
MOSBY-ELSEVIER
DOI: 10.1016/j.jpeds.2020.07.033
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类别
资金
- Utah Center for Excellence in ELSI Research (UCEER)
- National Human Genome Research Institute of the National Institutes of Health [RM1HG009037]
Objective To evaluate the cost-effectiveness of nusinersen with and without universal newborn screening for infantile-onset spinal muscular atrophy (SMA). Study design A Markovmodel using data from clinical trials with US epidemiologic and cost data was developed. The primary interventions studied were nusinersen treatment in a screening setting, nusinersen treatment in a nonscreening setting, and standard care. Analysis was conducted from a societal perspective. Results Compared with no screening and no treatment, the incremental cost-effectiveness ratio (ICER) for nusinersen with screening was $330 558 per event-free life year (LY) saved, whereas the ICER for nusinersen treatment without screening was $508 481 per event-free LY saved. 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. Conclusion Universal newborn screening for SMA provides improved economic value for payers and patients when nusinersen is available.
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