Patients With SMA Have Medical Costs Over 50 Times Higher Than Those of Healthy Patients

For infants, the main driver of these increased costs came from inpatient visits vs outpatient visits among children and juveniles.

A recent analysis is shining a spotlight on the high costs associated with spinal muscular atrophy (SMA), with researchers suggesting that direct costs of the condition run 50 times higher than those of matched controls. The results have the researchers urging for standardizing care to reduce costs.

The cost estimates come from 5 years’ worth of claims data on over 300 patients with at least 2 SMA-related medical claims at least a month apart. Most (279) patients were juvenile, while 58 were children and 56 were infants.

“Results of these analyses show that the economic burden of direct inpatient and outpatient costs associate with untreated SMA is tremendous and significantly higher than for age-, gender-, and region-matched controls,” wrote the researchers, who pointed out that these high costs were particularly pronounced for infants with SMA, which is the most severe form of the disease.

The main driver of these increased costs for infants came from inpatient visits, typically from respiratory infection and/or respiratory failure. Compared with controls, the length of an inpatient stay was 4 times longer for infants with SMA, with net mean payments for these admissions surpassing $100,000, drastically higher than the approximately $59.00 for controls. Similarly, outpatient costs were significantly higher for the infants with SMA, totaling $55,000 compared with $2000 for controls.

Child and juvenile SMA were also associated with significantly higher costs compared with controls, albeit less pronounced than those for infants. For these 2 groups of patients, outpatient costs were the main drivers of heightened costs associated with SMA, particularly those associated with in-home services.

For children, those impacted by SMA had inpatient costs of nearly $27,000 and outpatient costs of $73,000, compared with $140 and $1300, respectively, for controls. For juvenile patients, inpatient costs neared $40,000, and outpatient costs totaled $49,000 compared with $700 and $1100, respectively, for controls.

According to the researchers, their findings are in line with previous research.

“Uniform adoption and implementation of the current standards-of-care guidelines could result in fewer complications of SMA,” they wrote. “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.”

Highlighting the latter, the researchers cited a 2018 report, which showed that caregivers for children with rare diseases dedicate over 50 hours per week on caregiving responsibilities, requiring more than half to cut back on their work hours and resulting in the majority (86%) experiencing financial hardship, as well as the everyday stresses of caregiving.


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. Published online November 8, 2020. doi:10.1080/20016689.2020.1843277