Fournier Joey, Boussat Bastien, Dervaux Benoit, Gaudin Philippe, Romand Xavier
Department of Epidemiology and Medical Evaluation, CHU Grenoble Alpes, Grenoble, France.
Department of Epidemiology and Medical Evaluation, CHU Grenoble Alpes, Grenoble, and Laboratory TIMC, UMR 5525, CNRS, Joseph Fourier University, Grenoble, France.
Clin Exp Rheumatol. 2025 Jan;43(1):62-69. doi: 10.55563/clinexprheumatol/3f60yv. Epub 2024 Oct 3.
This study aimed to evaluate the cost-effectiveness of introducing tofacitinib in second-line therapies after methotrexate failure for rheumatoid arthritis in France.
Using a Markov model, we simulated a cohort of 10,000 patients based on literature data to compare various treatment strategies. The reference strategy included the four classes of biologics commonly used in France (TNFi, tocilizumab, abatacept, rituximab). The trial strategies additionally included tofacitinib at different introduction positions. The cycle duration was set at 6 months, and the time horizon was a lifetime. The data for severe adverse effects were sourced from the ORAL Surveillance study.
Compared to the reference strategy, introducing tofacitinib is a dominant strategy, regardless of its introduction position. Introducing it as the first-line treatment results in the greatest cost savings (€1,679 per patient) while increasing quality-adjusted life years (QALYs) by 0.29. According to the one-way sensitivity analysis, the discount rate and the cost of TNFi were the two variables that most influenced costs, while the change in HAQ score and the discount rate were the two variables that most influenced QALYs.
Our study represents the first assessment of the cost-effectiveness of tofacitinib in France and incorporates the latest adverse effects reported in the literature. It reinforces previously obtained results from other countries. Our study has some limitations, mainly related to the use of data from clinical trials. Our analysis is limited to severe adverse effects, and their cost is extrapolated from the average hospitalisation cost. The estimated costs are therefore underestimated for chronic diseases such as cancer.
本研究旨在评估在法国，对于甲氨蝶呤治疗失败后的类风湿关节炎患者，在二线治疗中引入托法替布的成本效益。
我们使用马尔可夫模型，基于文献数据模拟了10,000名患者的队列，以比较各种治疗策略。参考策略包括法国常用的四类生物制剂（肿瘤坏死因子抑制剂、托珠单抗、阿巴西普、利妥昔单抗）。试验策略还额外包括在不同引入位置使用托法替布。周期时长设定为6个月，时间跨度为终身。严重不良反应的数据来源于ORAL监测研究。
与参考策略相比，引入托法替布是一种占优策略，无论其引入位置如何。将其作为一线治疗引入可实现最大成本节约（每位患者节约1,679欧元），同时质量调整生命年（QALY）增加0.29。根据单向敏感性分析，贴现率和肿瘤坏死因子抑制剂的成本是对成本影响最大的两个变量，而健康评估问卷（HAQ）评分的变化和贴现率是对QALY影响最大的两个变量。
我们的研究是法国对托法替布成本效益的首次评估，并纳入了文献中报道的最新不良反应。它强化了之前在其他国家获得的结果。我们的研究存在一些局限性，主要与临床试验数据的使用有关。我们的分析仅限于严重不良反应，其成本是根据平均住院成本外推得出的。因此，对于癌症等慢性疾病，估计成本被低估了。
