Economic Burden for SMA Management Continues to Rise

January 21, 2021
Marco Meglio
Marco Meglio

Marco Meglio, Associate Editor for NeurologyLive, has been with the team since October 2019. Follow him on Twitter @marcomeglio1 or email him at

SAP Partner | <b>Cure SMA</b>

The average number of inpatient claims per year was higher for patients with SMA than their matched non-SMA patients, with respiratory illness the most common reason for an inpatient admission among all patients with SMA.

A recently published analysis by both Cure SMA and colleagues at Novartis Gene Therapies has called for efforts to reduce the costs of spinal muscular atrophy (SMA) after results showed a nearly 50-fold higher increase in costs for management of those with SMA compared to matched controls.1,2

Lead author Lisa Belter, MPH, director, research analytics, Cure SMA, and colleagues aimed to characterize direct costs associated with SMA management prior to the first FDA approved therapy for SMA in a retrospective cohort study by matching patients 1:1 to controls by birth year, gender, and geographic region. Patients were categorized as having infantile (n = 58), child (n = 56), or juvenile SMA (n = 279) based on diagnosis at age <1, 1–3, or 3–18 years, respectively.

They found that the mean net payments for inpatient admissions were higher for cases versus controls (infantile: $118,609 vs $58.79; child: $26,940.01 vs $143.56; juvenile: $39,389.91 vs $701.21; all P ≤.01). For patients with child SMA specifically, the mean annualized total net payments for inpatient admission were more than 180-fold higher compared with controls ($26,940 [SD, 64,909] for cases vs $144 [SD, 758] for controls; P = .0031).

Researchers used this study as a way to understand and quantify the impact that new therapies may have on the economic burden of patients with SMA. The results allow researchers to track cost-related changes over time as the new use of new treatments increases.

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"Uniform adoption and implementation of the current standards of care guidelines could result in fewer complications of SMA. In addition, the availability of new treatments and widespread SMA newborn screening has the potential to not only reduce the economic burden of SMA but also to significantly improve long-term clinical outcomes and patient and caregiver quality of life,” Belter et al concluded.

The average number of inpatients claims per year were higher for patients with SMA than their matched non-SMA patients, with respiratory illness the most common reason for an inpatient admission among all patients with SMA. Infantile, child, and juvenile SMA all had annual mean number of inpatient claims of 1.30, 0.62, and 0.66, respectively, compared to 0.01, 0.04, and 0.02 for their matched non-SMA controls.

Belter and colleagues also found that the mean net payments for outpatient services were higher among SMA cases versus controls (infantile: $55,537.83 vs $2047.20; child: $73,093.66 vs $1307.56; juvenile: $49,067.83 vs $1134.69; all P ≥.0002).

The mean outpatient claims per person per year was 234.4 (SD, 226.2) for infantile SMA cases and 25.8 (SD, 18.4) for controls (P <.0001). Among infantile SMA cases, a home health service, such as in-home skilled nursing or physical therapy, was the most common (n = 2219) type of outpatient claim.

The economic burden of SMA was assessed by measuring the direct costs captured through inpatient and outpatient insurance claims from the Truven Health Analytics Marketscan Commercial Claims and Encounters and Medicare Supplemental databases. Inpatient claims covered costs incurred for a patient admitted into the hospital while outpatient claims cover costs for medical care that does not require a hospital admission.

The economic burden can also impact the caregivers of those withSMA. A 2018 report by the National Alliance for Caregiving was included in the statistics, indicating that caregivers for children with rare diseases spend an average of 53 hours per week on caregiving responsibilities, which requires many (52%) to cut back their work hours.

As a result of lost wages and the costs of care, 86% of caregivers for patients with rare diseases experience financial hardship. The study authors noted, “concerns over finances, as well as the everyday stresses and strains of caregiving, can have a profound negative impact on caregivers’ emotional and physical health.”

NeurologyLive recently partnered with Cure SMA for our Cure Connections video program series titled, "Evaluation of Spinal Muscular Atrophy in the Era of Telehealth." To watch all the episodes within the series, click here.

1. Cure SMA, Novartis publish economic burden of SMA analysis in Journal of Market Access & Health Policy. News release. December 14, 2020. Accessed January 20, 2020.
2. Belter L, Cruz R, Kulas S, McGinnis E, Dabbous O, Jarecki J. Economic burden of spinal muscular atrophy: an analysis of claims data. J Mark Access Health Policy. 2020, VOL 8, 1843277. doi: 10.1080/200016689.2020.1843277