Pioneering SMA therapies for all types: survival gains, cost dynamics, and performance-based agreements.

BACKGROUND

The purpose of this study was to assess the impact of survival improvements and performance-based managed entry agreements (PBMEAs) on the cost implications of introducing innovative spinal muscular atrophy (SMA) treatments, nusinersen, onasemnogene abeparvovec, and risdiplam, for managing SMA Types 1, 2, and 3 from the perspective of the Saudi Ministry of Health (MoH).

METHODS

A budget impact model was created using inputs such as total population, market share, median survival, and resource utilization obtained through literature review and validated by expert committees. The model projected the overall cost (drug acquisition, administration, and disease management) for best supportive care (BSC) with and without these interventions over a 5-year period using Microsoft Excel as the analytical tool.

RESULTS

For SMA Type 1, the overall net budget impact of introducing onasemnogene abeparvovec, nusinersen, or risdiplam was significant, ranging from 112 to 225%. The impact was even greater for SMA Type 2 and 3, ranging from 171 to 283% due to high survival rates. However, the budget impact could be mitigated by improved clinical management and PBMEAs, reducing it to 77–84% for Type 1 and 36–117% for Types 2 and 3.

CONCLUSION

the introduction of these pioneering interventions for SMA management would raise the overall budget for the payer, primarily due to drug acquisition costs. Nevertheless, this increase could be offset by improvements in clinical management and PBMEAs.

SUPPLEMENTARY INFORMATION

The online version contains supplementary material available at 10.1186/s12962-025-00647-3.