OBJECTIVES: Spinal muscular atrophy (SMA) is a rare, genetic neuromuscular disease. Nusinersen is the first pharmacologic therapy approved for treatment of SMA. More research is needed to understand the real-world economic benefits of nusinersen treatment. Healthcare utilization and costs of patients treated with nusinersen were investigated using US hospital-based administrative data.

METHODS: Patients who were likely to have complete information on the date of first nusinersen dose (index admission) between 01/01/2017 and 03/31/2021 were included from the PINC AI™ Healthcare Database. Patients were followed in their index hospital system from index date to last discharge date up to 09/30/2021. Healthcare utilization and costs were reported for patients with ≥ 24-month follow-up over three time periods: index-to-6-months, 6-to-12-months, and 12-to-24 months (normalized to 6-month values). Number, length-of-stay (LOS), total cost ($US 2021) of inpatient hospitalizations and outpatient hospital visits were evaluated, excluding those related to nusinersen. Results were stratified by age: pediatric (0-17 years) and adult (18+ years).

RESULTS: Patients with ≥ 24-month follow-up (N = 56) were on average 22 years old (SD = 18), with 27 considered pediatric (mean age 6) and 29 adult (mean age 36). The mean cost of inpatient hospitalizations per pediatric patient decreased over time: $37,945 (index-to-6-months) to $13,787 (6-to-12-months) and to $8,780 per 6 months (12-to-24 months). Mean LOS per pediatric patient decreased from 8 days (index-to-6-months) to 3 days (6-to-12-months) and to 2 days per 6 months (12-to-24 months). The mean cost of outpatient hospital visits per pediatric patient also decreased over time: $4,020 (index-to-6-months) to $2,594 (6-to-12-months) and to $2,087 (12-to-24 months). Similar decreasing trends were observed for adults.

CONCLUSIONS: The results showed a trend in which healthcare utilization and costs decreased over time after initiation of nusinersen treatment using US hospital-based administrative data, suggesting observed economic benefits of treatment.