Bielefeld University, Faculty of Public Health, Universitätstr. 25, 33615, Bielefeld, Germany,
Health Econ Rev. 2014 Dec;4(1):18. doi: 10.1186/s13561-014-0018-2. Epub 2014 Sep 16.
Rheumatoid arthritis (RA) is a chronic, inflammatory disease with severe effects on the functional ability of patients. Due to the prevalence of 0.5 to 1.0 percent in western countries, new treatment options are a major concern for decision makers with regard to their budget impact. In this context, cost-effectiveness analyses are a helpful tool to evaluate new treatment options for reimbursement schemes.
To analyze and compare decision analytic modeling techniques and to explore their use in RA with regard to their advantages and shortcomings.
A systematic literature review was conducted in PubMED and 58 studies reporting health economics decision models were analyzed with regard to the modeling technique used.
From the 58 reviewed publications, we found 13 reporting decision tree-analysis, 25 (cohort) Markov models, 13 publications on individual sampling methods (ISM) and seven discrete event simulations (DES). Thereby 26 studies were identified as presenting independently developed models and 32 models as adoptions. The modeling techniques used were found to differ in their complexity and in the number of treatment options compared. Methodological features are presented in the article and a comprehensive overview of the cost-effectiveness estimates is given in Additional files 1 and 2.
When compared to the other modeling techniques, ISM and DES have advantages in the coverage of patient heterogeneity and, additionally, DES is capable to model more complex treatment sequences and competing risks in RA-patients. Nevertheless, the availability of sufficient data is necessary to avoid assumptions in ISM and DES exercises, thereby enabling biased results. Due to the different settings, time frames and interventions in the reviewed publications, no direct comparison of modeling techniques was applicable. The results from other indications suggest that incremental cost-effective ratios (ICERs) do not differ significantly between Markov and DES models, but DES is able to report more outcome parameters.
Given a sufficient data supply, DES is the modeling technique of choice when modeling cost-effectiveness in RA. Otherwise transparency on the data inputs is crucial for valid results and to inform decision makers about possible biases. With regard to ICERs, Markov models might provide similar estimates as more advanced modeling techniques.
类风湿性关节炎(RA)是一种慢性炎症性疾病,对患者的功能能力有严重影响。由于西方国家的患病率为 0.5%至 1.0%,因此新的治疗选择是决策者关注的主要问题,因为这涉及到他们的预算影响。在这种情况下,成本效益分析是评估新的治疗选择以用于报销计划的有用工具。
分析和比较决策分析建模技术,并探讨它们在 RA 中的应用,以了解它们的优缺点。
在 Pubmed 中进行了系统的文献回顾,并分析了 58 篇报告卫生经济学决策模型的研究,以了解所使用的建模技术。
从 58 篇综述文献中,我们发现有 13 篇报告决策树分析,25 篇(队列)马尔可夫模型,13 篇关于个体抽样方法(ISM)的出版物和 7 篇离散事件模拟(DES)。因此,有 26 项研究被确定为独立开发的模型,32 项模型为采用模型。所使用的建模技术在其复杂性和比较的治疗选择数量方面存在差异。本文介绍了方法学特征,并在附加文件 1 和 2 中给出了成本效益估计的综合概述。
与其他建模技术相比,ISM 和 DES 在涵盖患者异质性方面具有优势,此外,DES 能够对 RA 患者的更复杂治疗序列和竞争风险进行建模。然而,为了避免 ISM 和 DES 练习中的假设,需要有足够的数据可用性,从而避免产生有偏的结果。由于在综述文献中存在不同的设置、时间范围和干预措施,因此无法对建模技术进行直接比较。来自其他适应症的结果表明,马尔可夫和 DES 模型之间的增量成本效益比(ICER)没有显著差异,但 DES 能够报告更多的结果参数。
在有足够的数据供应的情况下,当对 RA 进行成本效益建模时,DES 是首选的建模技术。否则,关于数据输入的透明度对于获得有效结果和告知决策者可能存在的偏差至关重要。关于 ICER,马尔可夫模型可能会提供与更先进的建模技术相似的估计值。