Li Nannan, Majoie Marian, Evers Silvia, Rijkers Kim, Gubler Felix, Rouhl Rob, Lazeron Richard, Klarenbeek Pim, Laskier-Owens Vicki, Hiligsmann Mickaël
Department of Health Services Research, Care and Public Health Research Institute (CAPHRI), Maastricht University, Maastricht, The Netherlands.
Department of Research and Development, Epilepsy Center Kempenhaeghe, Heeze, The Netherlands.
J Med Econ. 2025 Dec;28(1):114-123. doi: 10.1080/13696998.2024.2443338. Epub 2025 Jan 2.
The objective of this study was to explore the financial consequences of adopting cenobamate as a treatment alternative in epilepsy patients with drug-resistant focal onset seizures (FOS) from a societal perspective in the Netherlands.
A previous budget impact model with a 5-year time horizon was adapted to the Dutch setting accounting for the eligible population, real-world market shares, treatment effectiveness and resource use in two scenarios: cenobamate with constant market share versus cenobamate with linearly increased market share up to 20%. Clinical inputs included treatment response, seizure reduction and adverse events. Costs consisted of drugs, medical and non-medical costs. One-way sensitivity analysis and scenario analysis were conducted to test the robustness of our results.
14,723 patients were eligible for cenobamate in 2022. Although cenobamate adds a gross budget impact of €12,686,30, the displacement of other drugs yields a total impact on the drug budget of €3,722,596 over 5 years. Adopting cenobamate resulted in a medical cost savings of €13,499,498 due to less resource use, and non-medical cost savings of €22,144,054 due to reduced productivity losses. Overall, savings generated at medical and non-medical cost level offset the gross drug budget impact of cenobamate, resulting in a saving of €31,920,955 over 5 years. Results were robust in the sensitivity/scenario analyses.
Treatment with cenobamate is associated with both medical and non-medical cost savings, which offset the increase in drug budget and result in a significant potential budget saving. The higher the market share of cenobamate, the larger the budget savings. We acknowledge several limitations; Complex scenarios such as drug interactions, stopping/switching drugs, and multiple drug use were not taken into account. The long-term efficacy and safety of cenobamate and its comparators remains uncertain. Future real-world data are needed to confirm our findings.
本研究的目的是从荷兰社会角度探讨在耐药性局灶性发作（FOS）癫痫患者中采用司替戊醇作为治疗选择的财务后果。
一个先前的5年预算影响模型根据荷兰的情况进行了调整，考虑了符合条件的人群、实际市场份额、治疗效果以及两种情景下的资源使用情况：市场份额恒定的司替戊醇与市场份额线性增长至20%的司替戊醇。临床输入包括治疗反应、癫痫发作减少情况和不良事件。成本包括药物、医疗和非医疗成本。进行了单向敏感性分析和情景分析以检验我们结果的稳健性。
2022年有14723名患者符合使用司替戊醇的条件。尽管司替戊醇增加了1268630欧元的总预算影响，但其他药物的替代在5年内对药物预算产生的总影响为3722596欧元。由于资源使用减少，采用司替戊醇导致医疗成本节省13499498欧元，由于生产力损失减少，非医疗成本节省22144054欧元。总体而言，医疗和非医疗成本层面产生的节省抵消了司替戊醇的总药物预算影响，在5年内节省了31920955欧元。结果在敏感性/情景分析中具有稳健性。
司替戊醇治疗与医疗和非医疗成本节省相关，这抵消了药物预算的增加并带来显著的潜在预算节省。司替戊醇的市场份额越高，预算节省就越大。我们承认存在一些局限性；未考虑药物相互作用、停药/换药以及多种药物联合使用等复杂情景。司替戊醇及其对照药物的长期疗效和安全性仍不确定。需要未来的真实世界数据来证实我们的发现。
