A new study published in the journal Advances in Therapy concluded that spinal muscular atrophy (SMA) is associated with substantial economic burden. The pharmacy costs of patients being treated with onasemnogene abeparvovec are greater than that of those being treated with nusinersen monotherapy, but their healthcare resource utilization and medical costs are lower, the study also reported.

To describe and compare the real-world healthcare resource utilization and costs of patients with SMA treated with disease-modifying treatments in the US, a team of researchers conducted a retrospective analysis of a US claims database from January 1, 2017, to March 31, 2021.

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During this period, the team analyzed data from 74 patients. Of these, 62 received nusinersen, 9 received onasemnogene abeparvovec monotherapy, and 3 switched to onasemnogene abeparvovec after nusinersen treatment.

The analysis showed that patients treated with nusinersen had more inpatient and emergency department visits per year than those treated with onasemnogene abeparvovec. More precisely, the number of mean inpatient visits for patients treated with nusinersen was 5.3 compared to 1.8 for patients treated with onasemnogene abeparvovec, and their mean number of emergency department visits was 3 compared to 1.5 for those being treated with onasemnogene abeparvovec.

Patients treated with nusinersen also had more disease-related medical costs per year, with a mean of $78,446 compared to $29,438 for those treated with onasemnogene abeparvovec. 

However, patients receiving onasemnogene abeparvovec incurred more pharmacy costs compared to those treated with nusinersen, with a mean of $2,241,875 compared to $693,191.

“Our study indicates the greater medical costs among patients receiving nusinersen were largely driven by invasive procedures, such as tracheostomy and gastrostomy, that required hospitalization,” the researchers said.

However, they added the exact reason why patients treated with nusinersen had greater healthcare resource utilization and cost was not clear and needed to be explored further.


Toro W, Yang M, Georgieva M, et al. Health care resource utilization and costs for patients with spinal muscular atrophy: Findings from a retrospective US claims database analysis. Adv Ther. Published online August 16, 2023. doi:10.1007/s12325-023-02621-y