The economic burden of spinal muscular atrophy

AbstractAim: To evaluate the economic burden of spinal muscular atrophy (SMA).Materials and methods: This study used Department of Defense Military Healthcare System (MHS) data from 2003–2012. Healthcare costs were determined for patients with at least one inpatient or three outpatient claims with a diagnosis of SMA before 18 years of age and who had ≥ 6 months of data after first SMA diagnosis or expired within 6 months of initial diagnosis. A comparator cohort was selected using a 3:1 match based on age and gender.Results: A total of 239 individuals with SMA diagnosis met the inclusion criteria along with 717 matched comparator patients. More patients with SMA had hospitalizations (69.5%) compared to the comparator cohort (17.2%, p < 0.001). Median total expenditures across all years of data for patients with SMA were $83 652 (25–75th percentile = $29 620–228 754) vs the comparator group of $4329 (25–75th percentile = $1229–10 062 (p < 0.001)) over an average (SD) of 6.9 ± 3.6 years. The annualized mean...