School of International Pharmaceutical Business, China Pharmaceutical University, 639 Longmiandadao Avenue, Nanjing, 211198, China.
Department of Rheumatology, Renji Hospital, School of Medicine, Shanghai Jiao Tong University, Shanghai, China.
Pharmacoeconomics. 2020 Dec;38(12):1345-1358. doi: 10.1007/s40273-020-00961-z. Epub 2020 Sep 15.
Patients with moderate-to-severe rheumatoid arthritis have a heavy financial burden. The cost-effectiveness of introducing tofacitinib to the current treatment sequence for patients with moderate-to-severe rheumatoid arthritis who have inadequate response or intolerance to conventional synthetic disease-modifying antirheumatic drugs (csDMARDs-IR) in China remains unknown.
The objective of this study was to assess the cost-effectiveness of introducing tofacitinib into the current treatment sequence in China for patients with moderate-to-severe rheumatoid arthritis who have csDMARDs-IR.
A Markov model was constructed from the perspective of the Chinese healthcare system to compare treatment sequences with and without first-line tofacitinib for patients with rheumatoid arthritis with csDMARDs-IR. The treatment sequence without tofacitinib included adalimumab, etanercept, recombinant human tumor necrosis factor receptor-Fc fusion protein, infliximab, and tocilizumab. Costs were derived from publicly available sources. Clinical trials, network meta-analysis, and real-world data were used to generate quality-adjusted life-years (QALYs), transition probabilities, and the incidence of adverse events. Mortality probabilities were estimated from rheumatoid arthritis-based, Chinese all-cause mortality data. One-way and probabilistic sensitivity analyses were conducted to verify the robustness of the model. In addition, the cost-effectiveness of adding tofacitinib as second- and third-line treatment options was evaluated in our analyses. Costs and effects were discounted at 5% per anum.
Compared to the current treatment sequence, adding tofacitinib as first-line treatment led to a cost-saving of $US880.11 (2018 values) and incremental QALYs of 1.34. Sensitivity analyses showed the results to be robust. Adding tofacitinib at second-line therapy was also a cost-saving option with a cost saving of $US653.65 and incremental QALYs of 1.34, while the incremental cost-effectiveness ratio of adding tofacitinib at third-line therapy was $US5588.14 per QALY gained.
Using the WHO-recommended ICER acceptability threshold of ≤ 1-time per capita Gross Domestic Product (GDP), our analysis suggests that the introduction of tofacitinib into the current treatment sequence for moderate-to-severe RA patients with csDMARDs-IR in China was a cost saving option as first- and second-line treatment, and cost-effective as a third-line treatment option. Of note, use of tofacitinib as first- and second-line treatment post-csDMARDs-IR appeared to be cost saving.
中重度类风湿关节炎患者的经济负担沉重。对于在中国对常规合成改善病情抗风湿药物（csDMARDs）反应不足或不耐受的中重度类风湿关节炎患者，引入托法替布（tofacitinib）加入当前治疗方案的成本效益尚不清楚。
本研究旨在评估在中国引入托法替布加入中重度类风湿关节炎 csDMARDs 反应不足患者当前治疗方案的成本效益。
从中国医疗保健系统的角度构建 Markov 模型，以比较中重度类风湿关节炎 csDMARDs 反应不足患者采用和不采用一线托法替布的治疗方案。不采用托法替布的治疗方案包括阿达木单抗、依那西普、重组人肿瘤坏死因子受体 -Fc 融合蛋白、英夫利昔单抗和托珠单抗。成本来自公开来源。临床试验、网络荟萃分析和真实世界数据用于生成质量调整生命年（QALYs）、转移概率和不良事件的发生率。死亡率根据基于类风湿关节炎的中国全因死亡率数据进行估计。进行了单因素和概率敏感性分析，以验证模型的稳健性。此外，我们还评估了将托法替布作为二线和三线治疗选择的成本效益。成本和效果按每年 5%贴现。
与当前治疗方案相比，将托法替布作为一线治疗可节省 880.11 美元（2018 年价值）和 1.34 个 QALYs。敏感性分析表明结果稳健。将托法替布作为二线治疗也是一种节省成本的选择，可节省 653.65 美元，增加 1.34 个 QALYs，而将托法替布作为三线治疗的增量成本效益比为每增加一个 QALY 需花费 5588.14 美元。
根据世界卫生组织推荐的成本效果比可接受阈值≤1 倍人均国内生产总值（GDP），我们的分析表明，在中国，将托法替布纳入中重度类风湿关节炎 csDMARDs 反应不足患者的当前治疗方案作为一线和二线治疗是一种节省成本的选择，作为三线治疗选择也是具有成本效益的。值得注意的是，将托法替布作为 csDMARDs 反应不足后的一线和二线治疗似乎具有成本效益。
