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251 Economic burden of spinal muscular atrophy type 1 (SMA1)
  1. Omar Dabbous1,
  2. Jennifer Seda2,
  3. Imran Kausar1,
  4. Marcus Droege1,
  5. Douglas M Sproule1
  1. 1AveXis, Inc
  2. 2SSI Strategy


Background SMA1 is a rapidly progressing disease resulting in muscle weakness, respiratory failure, and early death. This retrospective analysis estimated the economic burden of SMA1 using QuintilesIMS’s PharMetrics Plus Health Plan Claims Database.

Methods SMA1 infants (N=119) were matched (1:1) with a random sample of infants by age, gender, index year, and Charlson Comorbidity Index. Healthcare resource utilization (HCRU) and costs incurred during the post-index/follow-up period (≥30–360 days, February 2011–November 2016) were compared.

Results Significantly more SMA1 patients had ≥1 all-cause HCRU claims vs. matched patients (98.3% vs. 54.6%, P<0.0001). Mean per-patient-per-month (PPPM) all-cause HCRU was higher for SMA1 infants: pharmacy (1.43 vs. 0.37 prescriptions); outpatient (14.10 vs. 2.17 services); inpatient (0.23 vs. 0.003 admissions) (all, P<0.0001). Mean PPPM hospital admissions (0.23 vs. 0.003), length of hospital stay (6.93 vs. 0.09 days), procedures/admission (1.49 vs. 0.03), and readmissions (0.04 vs. 0.00) were also significantly greater for SMA1 infants (all, P<0.0001). Pharmacy, outpatient, and inpatient costs PPPM were greater in SMA1 infants ($371 vs. $20; $4,192 vs. $232; and $22,500 vs. $22 [all, P<0.0001]), resulting in extrapolated all-cause total annual costs of $324,751 (SMA1 cohort) vs. $3,294 (matched cohort).

Conclusions The economic burden of SMA1 is substantial.

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