The PATH (Predictive Approaches to Treatment effect Heterogeneity) Statement was developed to promote the conduct of, and provide guidance for, predictive analyses of heterogeneity of treatment effects (HTE) in clinical trials. The goal of predictive HTE analysis is to provide patient-centered estimates of outcome risk with versus without the intervention, taking into account all relevant patient attributes simultaneously, to support more personalized clinical decision making than can be made on the basis of only an overall average treatment effect. The authors distinguished 2 categories of predictive HTE approaches (a "risk-modeling" and an "effect-modeling" approach) and developed 4 sets of guidance statements: criteria to determine when risk-modeling approaches are likely to identify clinically meaningful HTE, methodological aspects of risk-modeling methods, considerations for translation to clinical practice, and considerations and caveats in the use of effect-modeling approaches. They discuss limitations of these methods and enumerate research priorities for advancing methods designed to generate more personalized evidence. This explanation and elaboration document describes the intent and rationale of each recommendation and discusses related analytic considerations, caveats, and reservations.

中文翻译：

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治疗效果异质性 (PATH) 声明的预测方法：解释和阐述。

PATH（治疗效果异质性预测方法）声明的制定是为了促进临床试验中治疗效果异质性 (HTE) 的预测分析的实施并为其提供指导。预测性 HTE 分析的目标是提供以患者为中心的干预与不干预结果风险估计，同时考虑所有相关患者属性，以支持比仅基于总体平均值做出的更加个性化的临床决策治疗效果。作者区分了 2 类预测 HTE 方法（“风险建模”和“效果建模”方法），并制定了 4 套指导声明：确定风险建模方法何时可能识别有临床意义的 HTE 的标准、方法论风险建模方法的各个方面、转化为临床实践的考虑因素以及使用效果建模方法的考虑因素和注意事项。他们讨论了这些方法的局限性，并列举了旨在生成更多个性化证据的先进方法的研究重点。本解释和阐述文件描述了每项建议的意图和基本原理，并讨论了相关的分析考虑因素、警告和保留。