Innovation and Value Initiative, 11100 Santa Monica Boulevard, Suite 500, Los Angeles, CA, 90025, USA.
Division of Clinical Immunology and Rheumatology, University of Alabama at Birmingham, Birmingham, AL, USA.
Pharmacoeconomics. 2019 Jun;37(6):829-843. doi: 10.1007/s40273-018-00765-2.
The nature of model-based cost-effectiveness analysis can lead to disputes in the scientific community. We propose an iterative and collaborative approach to model development by presenting a flexible open-source simulation model for rheumatoid arthritis (RA), accessible to both technical and non-technical end-users.
The RA model is a discrete-time individual patient simulation with 6-month cycles. Model input parameters were estimated based on currently available evidence and treatment effects were obtained with Bayesian network meta-analysis techniques. The model contains 384 possible model structures informed by previously published models. The model consists of the following components: (i) modifiable R and C++ source code available in a GitHub repository; (ii) an R package to run the model for custom analyses; (iii) detailed model documentation; (iv) a web-based user interface for full control over the model without the need to be well-versed in the programming languages; and (v) a general audience web-application allowing those who are not experts in modeling or health economics to interact with the model and contribute to value assessment discussions.
A primary function of the initial version of RA model is to help understand and quantify the impact of parameter uncertainty (with probabilistic sensitivity analysis), structural uncertainty (with multiple competing model structures), the decision framework (cost-effectiveness analysis or multi-criteria decision analysis), and perspective (healthcare or limited societal) on estimates of value.
In order for a decision model to remain relevant over time it needs to evolve along with its supporting body of clinical evidence and scientific insight. Multiple clinical and methodological experts can modify or contribute to the RA model at any time due to its open-source nature.
基于模型的成本效益分析的本质可能导致科学界的争议。我们提出了一种迭代和协作的模型开发方法，通过为类风湿关节炎（RA）展示一个灵活的开源模拟模型，使技术和非技术最终用户都能够访问。
RA 模型是一个具有 6 个月周期的离散时间个体患者模拟。模型输入参数是根据现有证据进行估计的，治疗效果是通过贝叶斯网络荟萃分析技术获得的。该模型包含 384 种可能的模型结构，这些结构是基于先前发表的模型得出的。该模型由以下组件组成：（i）可在 GitHub 存储库中修改的 R 和 C++源代码；（ii）用于进行自定义分析的 R 包；（iii）详细的模型文档；（iv）一个基于网络的用户界面，用于完全控制模型，而无需精通编程语言；（v）一个面向大众的网络应用程序，允许那些不是建模或健康经济学专家的人与模型交互并为价值评估讨论做出贡献。
RA 模型的初始版本的主要功能是帮助理解和量化参数不确定性（概率敏感性分析）、结构不确定性（多种竞争模型结构）、决策框架（成本效益分析或多准则决策分析）以及视角（医疗保健或有限的社会）对价值估计的影响。
为了使决策模型随着时间的推移保持相关性，它需要随着其支持的临床证据和科学洞察力而发展。由于其开源性质，多个临床和方法学专家可以随时修改或为 RA 模型做出贡献。
