Institute for Medical Technology Assessment, Erasmus University Rotterdam, Rotterdam, The Netherlands.
Institute for Medical Technology Assessment, Erasmus University Rotterdam, Rotterdam, The Netherlands.
Value Health. 2022 Jun;25(6):984-991. doi: 10.1016/j.jval.2021.11.1363. Epub 2021 Dec 15.
The rapid expansion in treatment options for relapsing-remitting multiple sclerosis (RRMS) of the past decade requires clinical decision making on the sequential prescription of these treatments. Here, we compare 360 treatment escalation sequences for patients with RRMS in terms of health outcomes and societal costs in The Netherlands.
We use a microsimulation model with a societal perspective, developed in collaboration with MS neurologists, to estimate the effectiveness and cost-effectiveness of 360 treatment sequences starting with first-line therapies in RRMS. This model integrated data on disease progression, disease-modifying treatment efficacy, clinical decision rules, age-dependent relapse rates, quality of life, healthcare, and societal costs.
Costs and health outcomes were overlapping among different treatment escalation sequences. In our model for RRMS treatment, optimal lifetime health outcomes (20.24 ± 1.43 quality-adjusted life-years [QALYs], 6.11 ± 0.30 relapses) were achieved with the sequence peginterferon-dimethyl fumarate-ocrelizumab-natalizumab-alemtuzumab. The most cost-effective sequence (peginterferon-glatiramer acetate-ocrelizumab-cladribine-alemtuzumab) yielded numerically worse health outcomes per patient (19.59 ± 1.43 QALYs, 6.64 ± 0.43 relapses), but resulted in €98 127 ± €19 134 less costs than the most effective treatment sequence.
Effectiveness estimates of treatments have overlapping confidence intervals but the treatment sequence that yields most QALYs is not the most cost-effective option, also when taking uncertainty into account. It is important that neurologists are aware of cost constraints and its relationship with prescription behavior, but treatment decisions should be individually tailored.
过去十年中，复发缓解型多发性硬化症（RRMS）的治疗选择迅速扩大，这需要对这些治疗方法的序贯处方进行临床决策。在此，我们比较了荷兰 RRMS 患者的 360 种治疗升级方案，从健康结果和社会成本方面进行评估。
我们使用一种具有社会视角的微观模拟模型，与多发性硬化症神经科医生合作开发，以评估 RRMS 一线治疗开始的 360 种治疗方案的有效性和成本效益。该模型综合了疾病进展、疾病修饰治疗效果、临床决策规则、年龄相关复发率、生活质量、医疗保健和社会成本的数据。
不同的治疗升级序列之间的成本和健康结果存在重叠。在我们的 RRMS 治疗模型中，最优的终生健康结果（20.24±1.43 个质量调整生命年[QALYs]，6.11±0.30 次复发）是通过培戈干扰素-二甲基富马酸酯-奥瑞珠单抗-那他珠单抗-阿仑单抗的序贯治疗实现的。最具成本效益的方案（培戈干扰素-醋酸格拉替雷-奥瑞珠单抗-克拉屈滨-阿仑单抗）在每个患者的健康结果方面略逊一筹（19.59±1.43 QALYs，6.64±0.43 次复发），但比最有效的治疗方案节省了 98127 欧元±19134 欧元的成本。
治疗的有效性估计有重叠的置信区间，但产生最多 QALYs 的治疗方案不一定是最具成本效益的选择，即使考虑到不确定性也是如此。神经科医生需要意识到成本限制及其与处方行为的关系，但治疗决策应该因人而异。
