Evidera Inc., San Francisco, CA, USA.
Agaplesion Markus Hospital, Frankfurt/Main, Germany.
J Med Econ. 2021 Jan-Dec;24(1):279-290. doi: 10.1080/13696998.2021.1881323.
Tofacitinib is an oral, small molecule Janus kinase (JAK) inhibitor for the treatment of ulcerative colitis (UC). This study assessed the cost-effectiveness of tofacitinib versus other available treatments for patients with moderate to severe UC following an inadequate response to conventional treatment and who are either naïve to or have failed previous biologics in Germany.
A Markov cohort model was developed to evaluate the differences in long-term costs and outcomes between tofacitinib and its comparators from the perspective of German statutory health insurance (SHI) for patients either naïve or exposed to biologics. Tofacitinib was compared to infliximab, infliximab biosimilar, adalimumab, adalimumab biosimilar, golimumab, vedolizumab, ustekinumab, and conventional therapy. Health states modeled were remission, treatment response, active UC, and post-colectomy. Patients not responding to treatment could switch to a different treatment. Treatment efficacy for induction and maintenance phases were assessed by a systematic literature review (SLR) and network meta-analysis (NMA). The model included costs associated with drug administration, adverse events, and medical resource use. Extensive deterministic and probabilistic sensitivity analyses (DSA and PSA) were conducted.
Over a life-time horizon, patients treated with tofacitinib gained 0.035-0.083 quality-adjusted life-years (QALYs) and had direct cost savings to the SHI of €4,228-€17,184 compared to biologic treatments other than adalimumab biosimilar. When compared to adalimumab biosimilar, treatment with tofacitinib resulted in an incremental cost-effectiveness ratio (ICER) of €17,497 per QALY gained and can be considered a cost-effective alternative. Compared with conventional therapy, tofacitinib resulted in a lower ICER than all other biologics. The DSA showed that the model results were most influenced by differences in treatment efficacy. The PSA suggested confidence in the base-case results considering uncertainty around parameters.
The results of this economic model suggest tofacitinib is a cost-effective treatment option for patients with moderate to severe UC in Germany.
托法替布是一种用于治疗溃疡性结肠炎（UC）的口服小分子 Janus 激酶（JAK）抑制剂。本研究评估了托法替布对比其他可用治疗方案在中重度 UC 患者中的成本效益，这些患者对常规治疗反应不佳，且无论是否对生物制剂初治或既往治疗失败，均在德国。
采用 Markov 队列模型，从德国法定健康保险（SHI）的角度评估了初治或既往接受过生物制剂治疗的患者中，托法替布与其他对照药物（英夫利昔单抗、英夫利昔单抗生物类似药、阿达木单抗、阿达木单抗生物类似药、古塞奇尤单抗、维得利珠单抗、乌司奴单抗和常规治疗）之间的长期成本和结局差异。评估了诱导和维持阶段的治疗疗效，评估方法为系统文献综述（SLR）和网络荟萃分析（NMA）。模型纳入了药物管理、不良事件和医疗资源使用相关的成本。进行了广泛的确定性和概率敏感性分析（DSA 和 PSA）。
在终身时间范围内，与生物制剂治疗相比，除阿达木单抗生物类似药外，托法替布治疗的患者获得了 0.035-0.083 个质量调整生命年（QALY），并为 SHI 带来了 4228-17184 欧元的直接成本节约。与阿达木单抗生物类似药相比，托法替布治疗的增量成本-效果比（ICER）为每获得 1 个 QALY 增加 17497 欧元，可被视为一种具有成本效益的替代方案。与常规治疗相比，托法替布的 ICER 低于所有其他生物制剂。DSA 表明，模型结果受治疗效果差异的影响最大。PSA 表明，考虑到参数的不确定性，对基础病例结果有信心。
该经济模型的结果表明，托法替布是德国中重度 UC 患者的一种具有成本效益的治疗选择。
