OBJECTIVES: Nusinersen is indicated for the treatment of spinal muscular atrophy (SMA) across all ages, administered intrathecally at a dose of 12 mg. Limited evidence exists on real-world healthcare resource utilization (HRU) and costs among patients taking nusinersen. The objective of this study was to evaluate real-world HRU and cost patterns associated with nusinersen use through US claims databases.

METHODS: Patients with SMA treated with nusinersen were identified in the Merative™ MarketScan^® Research Databases from commercial (January 2017 to June 2020) and Medicaid claims (January 2017 to December 2019). Patients who were likely to have complete information on the date of nusinersen initiation and continuous enrollment from 12 months pre- through 12 months post-index (first date of nusinersen) were retained. Number and costs ($US 2020) of inpatient hospitalizations, not relating to nusinersen administration or observational care, were evaluated over 12 months pre- and post-nusinersen initiation and were stratified by age: pediatric (<18 years) and adult (≥18 years).

RESULTS: Overall, 103 individuals receiving nusinersen were identified, of which 59 were pediatric with mean age of 9 (range: 1-17) years, and 44 were adults with mean age of 30 (range: 18-63) years. The mean number of inpatient hospitalizations per patient decreased in the 12 months post-treatment compared with the 12 months pre-treatment for both pediatrics and adults: -0.25 and -0.18, respectively. Average inpatient hospitalization costs per patient decreased from 12 months pre-index to post-index period for the pediatric cohort: $22,903 vs $8,466 and adult cohort: $13,997 vs $2,899. Similar trends were observed for respiratory-related inpatient hospitalization costs for both age cohorts.

CONCLUSIONS: This study demonstrates that nusinersen treatment was associated with reductions in number and costs of inpatient hospitalizations across pediatric and adult patients over a period of 12 months post-treatment initiation compared with the pre-treatment period using US claims databases.