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本库首次提交范围:2004-06-23 — 2026-10-14。
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Robust Estimation and Inference in Hybrid Controlled Trials for Binary Outcomes: A Case Study on Non-Small Cell Lung Cancer

二元结果混合对照试验的稳健估计与推断:非小细胞肺癌案例

Hybrid controlled trials (HCTs), which augment randomized controlled trials (RCTs) with external controls (ECs), are increasingly receiving attention as a way to address limited power, slow accrual, and ethical concerns in clinical research. However, borrowing from ECs raises critical statistical challenges in estimation and inference, especially for binary outcomes where hidden bias is harder to detect and estimands such as risk difference, risk ratio, and odds ratio are of…

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A Conformal Selection Framework for Individual Treatment Beneficiaries with Auxiliary External Data

利用辅助外部数据识别治疗受益者的共形筛选框架

Identifying patients who are likely to benefit from a treatment is central to precision medicine and can guide follow-up trials, enrichment designs, and individualized decisions. Although randomized controlled trials (RCTs) provide evidence on efficacy, they are usually powered to estimate average treatment effects rather than patient-level benefit. Meanwhile, artificial intelligence and machine learning methods offer flexible tools for estimating heterogeneous treatment…

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Variance Estimation for Weighted Average Treatment Effects

加权平均处理效应的方差估计

Common variance estimation methods for weighted average treatment effects (WATEs) in observational studies include nonparametric bootstrap and model-based, closed-form sandwich variance estimation. However, the computational cost of bootstrap increases with the size of the data at hand. Besides, some replicates may exhibit random violations of the positivity assumption even when the original data do not. Sandwich variance estimation relies on regularity conditions that may…

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