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

ABSTRACT:

Objective

To evaluate the cost-effectiveness of nusinersen with and without universal newborn screening for infantile-onset spinal muscular atrophy (SMA).

Study design

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.

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

SUBMITTER: Jalali A 

PROVIDER: S-EPMC7686158 | biostudies-literature | 2020 Dec

REPOSITORIES: biostudies-literature