The economic burden of spinal muscular atrophy

Edward P. Armstrong, Daniel C. Malone, Wei Shi Yeh, Georg J. Dahl, Rees L. Lee, Nicholas Sicignano

Research output: Contribution to journalArticlepeer-review

27 Scopus citations


Abstract: Aim: 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 costs of total healthcare expenditures were significantly higher for the SMA cases than the comparison cohort, $47 862 ± 88 607 compared to $1861 ± 6374, respectively (p < 0.001). The sub-group of patients with early diagnosis (n = 45) had 4.3 ± 2.9 years of observation with a median cost of $167 921 ($53 349–678 412). Mean age (SD) at first observed SMA diagnosis was 7.5 ± 6.4 years. Mean (SD) duration of follow-up after initial SMA diagnosis was 4.8 ± 3.3 years, with a median post-diagnosis cost of $60 213 ($18 229–192 559). The major costs for all patients were outpatient visits [median = $53 152 ($23 902–136 150)], followed by inpatient costs [median = $11 258 ($0–51 987)] and total prescription costs [median = $3167 ($943–13 283)]. Limitations: The analysis is limited to the data available and may under-estimate the total cost of SMA. Conclusions: Individuals with SMA have a high degree of morbidity, particularly those diagnosed during infancy. SMA patients have significant medical expenditures and high utilization of healthcare services.

Original languageEnglish (US)
Pages (from-to)822-826
Number of pages5
JournalJournal of medical economics
Issue number8
StatePublished - Aug 2 2016


  • Muscle disease
  • Neuromuscular disease
  • Spinal muscular atrophy

ASJC Scopus subject areas

  • Health Policy


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