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主方案试验：从目标量定义到治疗效应的稳健推断AI 解读

Master protocol trials use a single overarching protocol to evaluate multiple interventions, diseases, or disease subtypes, where individuals are often randomized to different subsets of intervention arms based on individual characteristics, enrollment timing, and intervention availability. While offering increased flexibility, this constrained and non-uniform intervention assignment poses two fundamental inferential challenges: the precise definition of treatment effects…

阶梯楔形集群随机试验中的模型稳健标准化AI 解读

Stepped-wedge cluster-randomized trials (SW-CRTs) are widely used in healthcare and implementation science, enabling all clusters to receive the intervention through a staggered rollout. Traditional model-based methods, including generalized estimating equations and mixed models, yield estimates that depend on implicit weighting schemes and parametric assumptions, and therefore may target ambiguous estimands under model misspecification. In this article, we propose a…

通过高斯过程稳健、自适应地整合平台试验非同期数据AI 解读

A platform trial is an innovative clinical trial design that enables simultaneous and continuous evaluation of multiple treatments within a single master protocol. Existing robust methods restrict analyses to concurrently randomized participants due to concerns that including nonconcurrent data may introduce bias from temporal trends. However, this exclusion represents a missed opportunity to improve efficiency. We propose a Gaussian process framework for incorporating…

用线性混合模型检测整群随机试验处理效应异质性：样本量与功效教程AI 解读

Cluster-randomized trials (CRTs) are a well-established class of designs for evaluating community-based interventions. An essential task in planning these trials is determining the number of clusters and cluster sizes needed to achieve sufficient statistical power for detecting a clinically relevant effect size. While methods for evaluating the average treatment effect (ATE) for the entire study population are well-established, sample size methods for testing heterogeneity…

主方案试验中的纵向患者历史与重复入组AI 解读

A master protocol trial uses a single overarching protocol to test multiple therapies, often across several diseases or subtypes. Although such trials offer considerable flexibility and efficiency, their constrained and non-uniform treatment assignment raises two core challenges: precisely defining treatment effects and conducting robust, efficient inference. These challenges intensify when participants can re-enroll to receive additional eligible therapies over time. To…

处理效应随时间变化时阶梯楔形试验检验功效的影响因素AI 解读

Stepped wedge cluster randomized trials (SW-CRTs) have historically been analyzed using immediate treatment (IT) models, which assume the effect of the treatment is immediate after treatment initiation and subsequently remains constant over time. However, recent research has shown that this assumption can lead to severely misleading results if treatment effects vary with exposure time, i.e. time since the intervention started. Models that account for time-varying treatment…

处理效应随暴露时间变化时的阶梯楔形整群随机试验分析AI 解读

Stepped wedge cluster randomized controlled trials are typically analyzed using models that assume the full effect of the treatment is achieved instantaneously. We provide an analytical framework for scenarios in which the treatment effect varies as a function of exposure time (time since the start of treatment) and define the "effect curve" as the magnitude of the treatment effect on the linear predictor scale as a function of exposure time. The "time-averaged treatment…