Cost of Untreated Spinal Muscular Atrophy is Tremendous, Study Suggests

Inpatient and outpatient costs directly associated with spinal muscular atrophy (SMA) averaged more than $100,000 annually per patient from 2012 to 2016, according to a study published in the Journal of Market Access & Health Policy.

“Direct costs of SMA are tremendous, often more than 50-fold higher compared with matched controls,” wrote researchers from Cure SMA and Novartis Gene Therapies. 

To gauge costs associated with the rare genetic neuromuscular disease during a 4-year span preceding approval of the first pharmacotherapy for SMA, researchers compared claims data for 58 patients with infantile SMA (diagnosed younger than 12 months), 56 patients with child SMA (diagnosed between 12 months and 36 months), and 279 patients with juvenile SMA (diagnosed between 36 months and 18 years) with claims data for age- and gender-matched control subjects from the same geographic region.  

Inpatient claims were much more frequent for patients with SMA. Rates were 60.3% for participants with infantile SMA compared with 1.7% among controls, 35.7% for those with child SMA compared with 3.6% for controls, and 47% for juvenile SMA compared with 4.3% for controls, according to the study. Average net payments for inpatient admissions were also higher for participants with SMA: $118,609 for participants with infantile SMA compared with $58.79 for controls, $26,940.01 for participants with child SMA compared with $143.56 for controls, and $39,389.91 for those with juvenile SMA compared with $701.21 for controls. 

Average net payments for outpatient services, too, were higher when SMA was present: $55,537.83 among participants with infantile SMA compared with $2047.20 for controls, $73,093.66 among those with child SMA compared with $1307.56 for controls, and $49,067.83 for those with juvenile SMA compared with $1134.69 for controls. 

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

—Jolynn Tumolo 

Reference: 

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;8(1):1843277. Published 2020 Nov 8. doi:10.1080/20016689.2020.1843277