The Economic Burden of SMA Prior to Treatment Approvals: Lisa Belter, MPH


The director of research analytics at Cure SMA discussed her research on the economic burden and costs patients with spinal muscular atrophy and their caregivers face.

"When we started to go into the literature, we realized that very little had been studied on this. That really started to get the ball rolling, and that it would be interesting to look at what the cost of having SMA is prior to these new therapies now being approved, with more coming down the pipeline. This way, we can provide a baseline data set of what the costs have been, and then moving forward, how those costs have changed."

At the conclusion of 2020, researchers at Cure SMA and Novartis Gene Therapies collaborated to analyze the direct costs associated with spinal muscular atrophy (SMA) management prior to the first FDA approved therapy for SMA. They found a nearly 50-fold increase in costs for this management compared to matched cohorts, as well significantly increased mean net payments across all inpatient admissions.

Lead author Lisa Belter, MPH, director, research analytics, Cure SMA, and colleagues evaluated infantile, child, and juvenile SMA, and aimed this study as a way to understand and quantify the impact new therapies may have on the economic burden of patients with SMA over time. To Belter’s knowledge, there had been a limited amount of literature produced on this specific burden.

In an interview with NeurologyLive, Belter discussed how her study came about and the impact these new therapies may have. She also mentioned why the study was originally focused on the prevalence of SMA but changed gears to this economic focus.

Cure SMA, Novartis publish economic burden of SMA analysis in Journal of Market Access & Health Policy. News release. December 14, 2020. Accessed March 3, 2020.
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