Evidera Inc, 500 Totten Pond Road, 5th Floor, Waltham, MA, 02451, USA.
Takeda Pharmaceutical Company Ltd, 1-1, Nihonbashi-Honcho 2-chome, Chuo-ku, Tokyo, 103-8668, Japan.
Pharmacoeconomics. 2020 Jan;38(1):69-84. doi: 10.1007/s40273-019-00841-1.
Vedolizumab (VDZ) was approved by the Japanese Ministry of Health, Labor and Welfare in 2018 for the treatment of patients with moderate-to-severe active ulcerative colitis (UC). The comparative cost-effectiveness of VDZ compared with other biologics is unknown in Japan. This information could be useful for decision makers at the time of repricing biologics for the treatment of patients with moderate-to-severe UC.
The aim was to assess the cost-effectiveness of VDZ versus other branded biologics for the treatment of patients with moderate-to-severe UC who were anti-tumor necrosis factor (TNF)-naïve, from the Japanese public healthcare payer perspective.
A hybrid decision tree/Markov model was developed to predict the number of patients who achieved response and remission at the end of the induction phase and sustained it during the maintenance phase, translating this into quality-adjusted life-years (QALYs) and costs. Treatment-related adverse events, discontinuation and surgery, and their impact on QALYs and costs were also modeled. A systematic literature review and network meta-analysis were conducted to estimate the comparative efficacy of each treatment versus placebo. Rates of adverse events, surgery, surgery complications, and utilities were from the literature. Costs (2018 Japanese yen) were obtained from the Japanese National Health Insurance drug price list and medical fee table and local claims databases. Clinical and economic outcomes were projected over a lifetime and discounted at 2% annually.
Over a lifetime, VDZ yielded greater QALYs and cost savings compared with golimumab and was cost-effective compared with adalimumab and infliximab (incremental cost-effectiveness ratios ¥4,821,940 and ¥4,687,692, respectively). Deterministic and probabilistic analyses supported the robustness of the findings in the base-case analysis, indicating that VDZ was either dominant or cost-effective in most scenarios and replications. The main limitations of this analysis include excluding tofacitinib and infliximab biosimilar as comparators, health-state utility estimates were obtained from population studies in the United Kingdom, and the impact of subsequent (i.e., second-line) biologic treatment was not evaluated.
Our analysis suggests that VDZ is dominant or cost-effective compared with other branded biologics for the treatment of anti-TNF-naïve patients with moderate-to-severe UC in Japan.
2018 年，日本厚生劳动省批准维得利珠单抗（VDZ）用于治疗中重度活动溃疡性结肠炎（UC）患者。在日本，尚不清楚 VDZ 与其他生物制剂相比的成本效益。在重新定价用于治疗中重度 UC 患者的生物制剂时，这些信息可能对决策者有用。
本研究旨在从日本公共医疗保健支付者的角度评估 VDZ 与其他品牌生物制剂治疗抗 TNF 初治中重度 UC 患者的成本效益。
采用混合决策树/马尔可夫模型预测诱导期结束时达到缓解和缓解的患者数量，并在维持期保持缓解，将其转化为质量调整生命年（QALYs）和成本。还对与治疗相关的不良反应、停药和手术及其对 QALYs 和成本的影响进行了建模。系统文献综述和网络荟萃分析用于估计每种治疗与安慰剂的比较疗效。不良事件、手术、手术并发症和效用的发生率来自文献。成本（2018 年日元）来自日本国家健康保险药品价格清单和医疗费用表以及当地索赔数据库。临床和经济结果在一生中进行预测，并按每年 2%贴现。
在一生中，VDZ 产生的 QALYs 更多，成本节省超过戈利木单抗，与阿达木单抗和英夫利昔单抗相比具有成本效益（增量成本效益比分别为¥4821940 和¥4687692）。确定性和概率分析支持基础分析结果的稳健性，表明在大多数情况下，VDZ 要么具有优势，要么具有成本效益，并且复制了结果。该分析的主要局限性包括排除托法替尼和英夫利昔单抗生物类似物作为对照药物，健康状态效用估计来自英国的人群研究，并且未评估后续（即二线）生物治疗的影响。
我们的分析表明，在日本，与其他品牌生物制剂相比，VDZ 治疗抗 TNF 初治中重度 UC 患者具有优势或具有成本效益。
