论文标题
多层模型作为复杂过程临床试验中估算规范的框架
Multistate Models as a Framework for Estimand Specification in Clinical Trials of Complex Processes
论文作者
论文摘要
基于强度的多态模型为表征疾病过程，干预措施的引入，随访的损失以及研究复杂生活历史过程的随机试验引起的其他并发症提供了有用的框架。在此框架内，我们讨论了估算规范所涉及的问题，并根据累积发病率功能回归模型显示了边际过程特征的共同估计值的限制值。当发生交流事件时，我们强调需要仔细定义目标估计值以及避免在现实世界中无法解释的推理目标的重要性。这对分析有影响，还具有临床试验的设计，其中协议可能有助于基于边际特征对估算的解释。
Intensity-based multistate models provide a useful framework for characterizing disease processes, the introduction of interventions, loss to follow-up, and other complications arising in the conduct of randomized trials studying complex life history processes. Within this framework we discuss the issues involved in the specification of estimands and show the limiting values of common estimators of marginal process features based on cumulative incidence function regression models. When intercurrent events arise we stress the need to carefully define the target estimand and the importance of avoiding targets of inference that are not interpretable in the real world. This has implications for analyses, but also the design of clinical trials where protocols may help in the interpretation of estimands based on marginal features.
