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个体随机实地试验中常用估计量隐含的疫苗效力目标量AI 解读

We review vaccine efficacy (VE) estimands for susceptibility in individual randomized trials with natural (unmeasured) exposure, where individual responses are measured as time from vaccination until an event (e.g., disease from the infectious agent). Common VE estimands are written as $1-θ$, where $θ$ is some ratio effect measure (e.g., ratio of incidence rates, cumulative incidences, hazards, or odds) comparing outcomes under vaccination versus control. Although the ratio…

利用疫苗无关感染去偏随时间变化的风险率型疫苗效应AI 解读

Understanding how vaccine effectiveness (VE) changes over time can provide evidence-based guidance for public health decision making. While commonly reported by practitioners, time-varying VE estimates obtained using Cox regression are vul- nerable to hidden biases. To address these limitations, we describe how to leverage vaccine-irrelevant infections to identify hazard-based, time-varying VE in the pres- ence of unmeasured confounding and selection bias. We articulate…

基于替代终点的临时批准因果路线图AI 解读

For many rare diseases with no approved preventive interventions, promising interventions exist, yet it has been difficult to conduct a pivotal phase 3 trial that could provide direct evidence demonstrating a beneficial effect on the target disease outcome. When a promising putative surrogate endpoint(s) for the target outcome is available, surrogate-based provisional approval of an intervention may be pursued. We apply the Causal Roadmap rubric to define a surrogate…