Pioneering SMA therapies for all types: survival gains, cost dynamics, and performance-based agreements
Abstract 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, fo...
Saved in:
| Main Authors: | , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
BMC
2025-08-01
|
| Series: | Cost Effectiveness and Resource Allocation |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s12962-025-00647-3 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| _version_ | 1849763467218649088 |
|---|---|
| author | Ahmed Al-jedai Hajer Al-Mudaiheem AlJohara AlSakran Fahad A. Bashiri Fouad Ghamdi Mohammad A. Almuhaizea Abdulaziz AlSamman Nancy Awad Rita Ojeil |
| author_facet | Ahmed Al-jedai Hajer Al-Mudaiheem AlJohara AlSakran Fahad A. Bashiri Fouad Ghamdi Mohammad A. Almuhaizea Abdulaziz AlSamman Nancy Awad Rita Ojeil |
| author_sort | Ahmed Al-jedai |
| collection | DOAJ |
| description | Abstract 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. |
| format | Article |
| id | doaj-art-3629c63552cd4c76a9afcf487f84631c |
| institution | DOAJ |
| issn | 1478-7547 |
| language | English |
| publishDate | 2025-08-01 |
| publisher | BMC |
| record_format | Article |
| series | Cost Effectiveness and Resource Allocation |
| spelling | doaj-art-3629c63552cd4c76a9afcf487f84631c2025-08-20T03:05:24ZengBMCCost Effectiveness and Resource Allocation1478-75472025-08-0123111210.1186/s12962-025-00647-3Pioneering SMA therapies for all types: survival gains, cost dynamics, and performance-based agreementsAhmed Al-jedai0Hajer Al-Mudaiheem1AlJohara AlSakran2Fahad A. Bashiri3Fouad Ghamdi4Mohammad A. Almuhaizea5Abdulaziz AlSamman6Nancy Awad7Rita Ojeil8Colleges of Medicine and Pharmacy, Alfaisal UniversityMinistry of HealthKing Faisal Specialist Hospital and Research CentreKing Saud UniversityKing Fahad Specialist HospitalColleges of Medicine and Pharmacy, Alfaisal UniversityNational Neuroscience Institute, King Fahad Medical CityCarexsoCarexsoAbstract 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.https://doi.org/10.1186/s12962-025-00647-3Spinal muscular atrophy (SMA)Budget impact model (BIM)Health technology assessment (HTA)Kingdom of saudi arabia (KSA)Saudi ministry of health (MoH)Performance-based managed entry agreement (PBMEA) |
| spellingShingle | Ahmed Al-jedai Hajer Al-Mudaiheem AlJohara AlSakran Fahad A. Bashiri Fouad Ghamdi Mohammad A. Almuhaizea Abdulaziz AlSamman Nancy Awad Rita Ojeil Pioneering SMA therapies for all types: survival gains, cost dynamics, and performance-based agreements Cost Effectiveness and Resource Allocation Spinal muscular atrophy (SMA) Budget impact model (BIM) Health technology assessment (HTA) Kingdom of saudi arabia (KSA) Saudi ministry of health (MoH) Performance-based managed entry agreement (PBMEA) |
| title | Pioneering SMA therapies for all types: survival gains, cost dynamics, and performance-based agreements |
| title_full | Pioneering SMA therapies for all types: survival gains, cost dynamics, and performance-based agreements |
| title_fullStr | Pioneering SMA therapies for all types: survival gains, cost dynamics, and performance-based agreements |
| title_full_unstemmed | Pioneering SMA therapies for all types: survival gains, cost dynamics, and performance-based agreements |
| title_short | Pioneering SMA therapies for all types: survival gains, cost dynamics, and performance-based agreements |
| title_sort | pioneering sma therapies for all types survival gains cost dynamics and performance based agreements |
| topic | Spinal muscular atrophy (SMA) Budget impact model (BIM) Health technology assessment (HTA) Kingdom of saudi arabia (KSA) Saudi ministry of health (MoH) Performance-based managed entry agreement (PBMEA) |
| url | https://doi.org/10.1186/s12962-025-00647-3 |
| work_keys_str_mv | AT ahmedaljedai pioneeringsmatherapiesforalltypessurvivalgainscostdynamicsandperformancebasedagreements AT hajeralmudaiheem pioneeringsmatherapiesforalltypessurvivalgainscostdynamicsandperformancebasedagreements AT aljoharaalsakran pioneeringsmatherapiesforalltypessurvivalgainscostdynamicsandperformancebasedagreements AT fahadabashiri pioneeringsmatherapiesforalltypessurvivalgainscostdynamicsandperformancebasedagreements AT fouadghamdi pioneeringsmatherapiesforalltypessurvivalgainscostdynamicsandperformancebasedagreements AT mohammadaalmuhaizea pioneeringsmatherapiesforalltypessurvivalgainscostdynamicsandperformancebasedagreements AT abdulazizalsamman pioneeringsmatherapiesforalltypessurvivalgainscostdynamicsandperformancebasedagreements AT nancyawad pioneeringsmatherapiesforalltypessurvivalgainscostdynamicsandperformancebasedagreements AT ritaojeil pioneeringsmatherapiesforalltypessurvivalgainscostdynamicsandperformancebasedagreements |