Al-Jedai Ahmed, Al-Mudaiheem Hajer, AlSakran AlJohara, Bashiri Fahad A, Ghamdi Fouad, Almuhaizea Mohammad A, AlSamman Abdulaziz, Awad Nancy, Ojeil Rita
Colleges of Medicine and Pharmacy, Alfaisal University, Riyadh, Saudi Arabia.
Ministry of Health, Riyadh, Saudi Arabia.
Cost Eff Resour Alloc. 2025 Aug 5;23(1):40. doi: 10.1186/s12962-025-00647-3.
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).
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.
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.
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.
The online version contains supplementary material available at 10.1186/s12962-025-00647-3.
本研究旨在从沙特阿拉伯卫生部（MoH）的角度评估生存改善和基于绩效的有条件准入协议（PBMEA）对引入创新型脊髓性肌萎缩症（SMA）治疗药物（诺西那生钠、onasemnogene abeparvovec和利司扑兰）以治疗1型、2型和3型SMA的成本影响。
使用通过文献综述获得并经专家委员会验证的总人口、市场份额、中位生存期和资源利用等输入数据创建了一个预算影响模型。该模型以Microsoft Excel作为分析工具，预测了在有和没有这些干预措施的情况下，5年内最佳支持治疗（BSC）的总体成本（药物采购、给药和疾病管理）。
对于1型SMA，引入onasemnogene abeparvovec、诺西那生钠或利司扑兰的总体净预算影响显著，范围为112%至225%。由于生存率较高，2型和3型SMA的影响更大，范围为171%至283%。然而，通过改善临床管理和PBMEA可以减轻预算影响，将1型的预算影响降至77 - 84%，2型和3型降至36 - 117%。
引入这些用于SMA管理的开创性干预措施将增加支付方的总体预算，主要原因是药物采购成本。不过，临床管理的改善和PBMEA可以抵消这一增加。
在线版本包含可在10.1186/s12962 - 025 - 00647 - 3获取的补充材料。
