Details

Autor(en) / Beteiligte

• Jalali, Ali
• Rothwell, Erin
• Botkin, Jeffrey R.
• Anderson, Rebecca A.
• Butterfield, Russell J.
• Nelson, Richard E.

Titel

Cost-Effectiveness of Nusinersen and Universal Newborn Screening for Spinal Muscular Atrophy

Ist Teil von

• The Journal of pediatrics, 2020-12, Vol.227, p.274-280.e2

Ort / Verlag

United States: Elsevier Inc

Erscheinungsjahr

2020

Link zum Volltext

Quelle

Elsevier ScienceDirect Journals Complete

Beschreibungen/Notizen

• To evaluate the cost-effectiveness of nusinersen with and without universal newborn screening for infantile-onset spinal muscular atrophy (SMA). A Markov model 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. 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. Universal newborn screening for SMA provides improved economic value for payers and patients when nusinersen is available.