Cost effectiveness of nusinersen for patients with infantile-onset spinal muscular atrophy in US.

ABSTRACT:

Background

Patients with infantile-onset spinal muscular atrophy (SMA), a rare, genetic neuromuscular disease, do not achieve key motor function milestones (e.g., sitting) and have short life expectancy in the absence of treatment. Nusinersen is a disease-modifying therapy for patients with SMA.

Objective

The aim of this study was to estimate the cost-effectiveness of nusinersen compared to best supportive care (BSC) in patients diagnosed with infantile-onset SMA in the US.

Methods

A de novo economic model was developed with the following health states: "permanent ventilation", "not sitting", "sitting", "walking", and "death". Short-term data were sourced from the pivotal clinical trials and studies of nusinersen (ENDEAR and SHINE). Motor function milestones achieved

SUBMITTER: Thokala P 

PROVIDER: S-EPMC7539471 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature