The economic burden of spinal muscular atrophy.
OBJECTIVE
To evaluate the economic burden of spinal muscular atrophy (SMA).


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 (25-75(th) 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)].


CONCLUSIONS
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.
a Strategic Therapeutics , Oro Valley , AZ , USA ;|b College of Pharmacy , University of Arizona , Tucson , AZ , USA ;
Edward P
EP
Armstrong
a Strategic Therapeutics , Oro Valley , AZ , USA ;|b College of Pharmacy , University of Arizona , Tucson , AZ , USA ;
Daniel C
DC
Malone
c Biogen , Weston , MA , USA ;
Wei-Shi
WS
Yeh
d Naval Medical Center , Portsmouth , VA , USA ;
Georg J
GJ
Dahl
e Naval Medical Research Unit , Dayton , OH , USA ;
Rees L
RL
Lee
f Health ResearchTx , Trevose , PA , USA.
Nicholas
N
Sicignano
D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D005260:Female; D005102:Health Expenditures / Q000706:statistics & numerical data*; D006296:Health Services / Q000191:economics* / Q000706:statistics & numerical data*; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D007345:Insurance Claim Review; D008297:Male; D009134:Muscular Atrophy, Spinal / Q000191:economics* / Q000503:physiopathology; D012189:Retrospective Studies; D014481:United States
Muscle disease; Neuromuscular disease; Spinal muscular atrophy