Li SiNi, Li JianHe, Peng LiuBao, Li YaMin, Wan XiaoMin
Clinical Nursing Teaching and Research Section, The Second Xiangya Hospital, Central South University, Changsha, 410011, China.
The Xiangya Nursing School, Central South University, Changsha, 410013, China.
Rheumatol Ther. 2021 Jun;8(2):863-876. doi: 10.1007/s40744-021-00308-w. Epub 2021 Apr 24.
A phase 3 (RA-BEAM study) clinical trial reported that baricitinib (BCT) + methotrexate (MTX) had clinical improvement compared with adalimumab (ADA) + MTX as a first-line strategy in patients with rheumatoid arthritis (RA) who had inadequate responses to MTX monotherapy. However, from the perspective of the Chinese healthcare system, the cost-effectiveness of introducing BCT into current treatment for patients with RA unresponsive to MTX remains unclear.
A patient-level microsimulation model was used to extrapolate the lifetime incremental cost per quality-adjusted life-year (QALY) and other outcomes. This study compared treatment sequences with or without first-line BCT with current treatment sequences, including adalimumab, etanercept, tocilizumab, and palliative care. Effectiveness and physical function were assessed using the American College of Rheumatology (ACR) 20/50/70 response and Health Assessment Questionnaire (HAQ). The input parameters of the model, comprising patient characteristics (sex and age) and treatment efficacy (ACR responses and HAQ score), were derived from a phase III clinical trial and network meta-analysis. The total cost estimation included direct costs and indirect costs. Probabilistic and univariate sensitivity analyses were performed, as were a series of scenario analyses.
The lifetime analysis revealed that adding BCT as a first-line treatment resulted in a QALY gain of 2.66 years; this gain would cost an incremental $26,662, leading to an incremental cost-effectiveness ratio of $10,036/QALY per patient compared with the current treatment sequence. Sensitivity and scenario analyses showed the results to be robust.
From a Chinese payer perspective, the introduction of BCT into the current treatment sequence is projected to be a cost-effective option as first-, second-, third-, and fourth-line treatment for patients with moderate-to-severe RA.
一项3期（RA - BEAM研究）临床试验报告称，对于甲氨蝶呤（MTX）单药治疗反应不足的类风湿关节炎（RA）患者，作为一线治疗策略，巴瑞替尼（BCT）联合甲氨蝶呤（MTX）相较于阿达木单抗（ADA）联合MTX有临床改善。然而，从中国医疗体系的角度来看，将BCT引入当前对MTX无反应的RA患者治疗中的成本效益仍不明确。
使用患者水平的微观模拟模型来推断每质量调整生命年（QALY）的终身增量成本及其他结果。本研究将有或无一线BCT的治疗序列与当前治疗序列进行比较，当前治疗序列包括阿达木单抗、依那西普、托珠单抗和姑息治疗。使用美国风湿病学会（ACR）20/50/70反应和健康评估问卷（HAQ）评估有效性和身体功能。模型的输入参数，包括患者特征（性别和年龄）和治疗效果（ACR反应和HAQ评分），来自一项III期临床试验和网络荟萃分析。总成本估计包括直接成本和间接成本。进行了概率和单因素敏感性分析以及一系列情景分析。
终身分析显示，添加BCT作为一线治疗导致QALY增加2.66年；这一增加将花费增量26,662美元，与当前治疗序列相比，每位患者的增量成本效益比为10,036美元/QALY。敏感性和情景分析表明结果是稳健的。
从中国支付方的角度来看，将BCT引入当前治疗序列预计对于中重度RA患者作为一线、二线、三线和四线治疗是一种具有成本效益的选择。
