M.M. Verhoeven, PhD student, J. Tekstra, MD, PhD, J.M. van Laar, MD, PhD, J.W. Bijlsma, MD, PhD, J.W. Jacobs, MD, PhD, F.P. Lafeber, PhD, P.M. Welsing, PhD, Rheumatology & Clinical Immunology, University Medical Center Utrecht, Utrecht, the Netherlands;
M.M. Verhoeven, PhD student, J. Tekstra, MD, PhD, J.M. van Laar, MD, PhD, J.W. Bijlsma, MD, PhD, J.W. Jacobs, MD, PhD, F.P. Lafeber, PhD, P.M. Welsing, PhD, Rheumatology & Clinical Immunology, University Medical Center Utrecht, Utrecht, the Netherlands.
J Rheumatol. 2021 Apr;48(4):495-503. doi: 10.3899/jrheum.200067. Epub 2020 Aug 1.
Our study aimed to evaluate the cost effectiveness of initiating tocilizumab (TCZ) ± methotrexate (MTX) versus initiating MTX as treat-to-target treatment strategies over 5 years in early disease-modifying antirheumatic drug (DMARD)-naïve rheumatoid arthritis (RA).
Data on resource use were collected with questionnaires at baseline, 3, 6, 12, and 24 months, and yearly thereafter, and were converted to costs using Dutch reference prices. Quality-adjusted life-years (QALY) were calculated using the EQ5D5L, with utility based on Dutch tariff or estimated by the Health Assessment Questionnaire. To account for missing cost data and QALY data and for sample uncertainty, first bootstraps (10,000 samples) were obtained. Second, single imputation using chained equations nested within these bootstrap samples was performed. An economic evaluation was performed for TCZ + MTX and TCZ, compared to MTX, as initial treatment in a treat-to-target strategy from a healthcare and societal perspective over 5 years. Several sensitivity analyses were performed.
Mean differences in QALY were small and not significant (TCZ + MTX vs MTX: 0.06, 95% CI -0.02 to 0.13; TCZ vs. MTX: -0.03, 95% CI -0.05 to 0.11). Limited savings in indirect nonhealthcare costs and productivity loss costs (for TCZ only) were observed, but these did not compensate for the higher medication costs. Sensitivity analyses did not materially change these findings, although lower-priced TCZ, or reserving TCZ as initial therapy for prognostically unfavorable RA patients, improved cost effectiveness considerably but did not individually lead to a strategy being cost effective.
Based on our analyses, early initiation of TCZ + MTX is not cost effective compared to MTX initiation in a step-up treat-to-target treatment strategy over 5 years in early RA patients.
我们的研究旨在评估在早期疾病修饰抗风湿药物（DMARD）初治类风湿关节炎（RA）患者中，与起始甲氨蝶呤（MTX）单药治疗相比，起始托珠单抗（TCZ）±MTX 作为达标治疗策略，在 5 年内的成本效果。
使用问卷在基线、3、6、12 和 24 个月以及此后每年收集资源使用数据，并使用荷兰参考价格转换为成本。使用 EQ5D5L 计算质量调整生命年（QALY），效用基于荷兰关税或使用健康评估问卷估计。为了考虑缺失的成本数据和 QALY 数据以及样本不确定性，首先进行了 10,000 个样本的 bootstrap 抽样。其次，使用嵌套在这些 bootstrap 样本中的连锁方程进行单个插补。从医疗保健和社会角度出发，在 5 年内，对 TCZ+MTX 和 TCZ 与 MTX 相比，作为达标治疗策略的初始治疗进行了经济评估。进行了几种敏感性分析。
QALY 的平均差异较小且无统计学意义（TCZ+MTX 与 MTX：0.06，95%CI-0.02 至 0.13；TCZ 与 MTX：-0.03，95%CI-0.05 至 0.11）。观察到间接非医疗保健成本和生产力损失成本（仅针对 TCZ）的节省有限，但这些成本并未弥补更高的药物成本。敏感性分析并没有改变这些发现，尽管较低价格的 TCZ 或将 TCZ 保留为预后不利的 RA 患者的初始治疗，可以显著提高成本效果，但没有一个策略可以单独实现成本效果。
根据我们的分析，与 MTX 起始相比，在早期 RA 患者中，5 年内采用 TCZ+MTX 早期起始作为达标治疗策略并不具有成本效果。
