当前,药品真实世界医保综合价值评估（以下简称“医保价评”）正逐步成为药品价值评估和医保决策支持的重要工具。然而,与随机对照试验相比,真实世界研究更易受到选择偏倚、混杂因素、信息偏倚及缺失数据等因素影响,需要通过科学合理的研究问题和研究设计来保障研究结果的可靠性。现阶段,医保价评在研究设计环节仍存在不足,影响了所产生证据的科学性和决策应用价值。本研究通过梳理医保价评研究方案,归纳该评估中的关键问题,发现当前研究设计的关键问题在于其与该评估的核心内涵之间存在脱节。在此基础上,本文提出针对性优化路径,以促进医保价评规范开展,为医保科学决策提供更高质量的证据支撑。

Currently, real-world evidence-based comprehensive value evaluation of medicines in medical insurance (hereafter referred to as “R-VALUES”) is gradually becoming an important tool for medicine value assessment and medical insurance decision-making support. However, compared with randomized controlled trials, real-world studies are more susceptible to factors such as selection bias, confounding factors, information bias, and missing data. Therefore, scientific and appropriate research questions and study designs are essential to ensure the reliability of research findings. At present, there are still deficiencies in the study design phase of R-VALUES, which may affect the scientific validity of the generated evidence and its value in decision-making applications. By reviewing research protocols for R-VALUES, this study identifies and summarises the key issues in current evaluation practices. The findings indicate that the central challenge in current research design lies in a disconnect between the design of these studies and the core objectives of R-VALUES. On this basis, this study proposes targeted approaches for optimisation to promote the standardised conduct of R-VALUES and provide higher-quality evidence to support evidence-informed medical insurance decision-making.