Worldwide Health Economics and Outcomes Research, Bristol-Myers Squibb (BMS), Lawrence, New Jersey, United States of America.
Erasmus University Rotterdam, Rotterdam, The Netherlands.
PLoS One. 2018 Oct 5;13(10):e0205013. doi: 10.1371/journal.pone.0205013. eCollection 2018.
The objective of this study was to evaluate current approaches to economic modeling in rheumatoid arthritis (RA) and propose a new conceptual model for evaluation of the cost-effectiveness of RA interventions. We followed recommendations from the International Society of Pharmacoeconomics and Outcomes Research-Society of Medical Decision Making (ISPOR-SMDM) Modeling Good Research Practices Task Force-2. The process involved scoping the decision problem by a working group and drafting a preliminary cost-effectiveness model framework. A systematic literature review (SLR) of existing decision-analytic models was performed and analysis of an RA registry was conducted to inform the structure of the draft conceptual model. Finally, an expert panel was convened to seek input on the draft conceptual model. The proposed conceptual model consists of three separate modules: 1) patient characteristic module, 2) treatment module, and 3) outcome module. Consistent with the scope, the conceptual model proposed six changes to current economic models in RA. These changes proposed are to: 1) use composite measures of disease activity to evaluate treatment response as well as disease progression (at least two measures should be considered, one as the base case and one as a sensitivity analysis); 2) conduct utility mapping based on disease activity measures; 3) incorporate subgroups based on guideline-recommended prognostic factors; 4) integrate realistic treatment patterns based on clinical practice/registry datasets; 5) assimilate outcomes that are not joint related (extra-articular outcomes); and 6) assess mortality based on disease activity. We proposed a conceptual model that incorporates the current understanding of clinical and real-world evidence in RA, as well as of existing modeling assumptions. The proposed model framework was reviewed with experts and could serve as a foundation for developing future cost-effectiveness models in RA.
本研究旨在评估类风湿关节炎（RA）经济建模的现有方法，并提出一种新的概念模型，用于评估 RA 干预措施的成本效益。我们遵循国际药物经济学和结果研究学会-医学决策制定学会（ISPOR-SMDM）建模良好实践工作组 2 的建议。该过程包括工作组对决策问题进行范围界定和起草初步成本效益模型框架。对现有的决策分析模型进行了系统文献回顾（SLR），并对 RA 登记处进行了分析，为草案概念模型的结构提供信息。最后，召集了一个专家小组，就草案概念模型征求意见。拟议的概念模型由三个独立的模块组成：1）患者特征模块，2）治疗模块和 3）结果模块。与范围一致，拟议的概念模型对 RA 中的当前经济模型提出了六项更改。这些更改建议是：1）使用疾病活动的综合指标来评估治疗反应和疾病进展（至少应考虑两种指标，一种作为基础案例，一种作为敏感性分析）；2）根据疾病活动指标进行效用映射；3）根据指南推荐的预后因素纳入亚组；4）基于临床实践/登记数据集整合现实的治疗模式；5）整合与关节无关的结果（关节外结果）；6）根据疾病活动评估死亡率。我们提出了一个概念模型，该模型纳入了当前对 RA 中临床和真实世界证据的理解，以及对现有建模假设的理解。提出的模型框架已由专家进行了审查，并可以作为在 RA 中开发未来成本效益模型的基础。
