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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AI 解读

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…

混合对照试验的功效与样本量计算AI 解读

Hybrid controlled trials (HCTs) augment randomized controls with external controls (ECs) to address practical challenges in randomized controlled trials (RCTs) and improve statistical power in settings such as rare diseases, oncology, and pediatrics. However, prospective sample-size determination is challenging because the required RCT sample size depends on the comparability of ECs with RCT controls, which are unavailable at the planning stage. We propose a 5+3 design for…

AMP 艾滋病预防试验中地区特异性生存效应的定向数据融合AI 解读

The Antibody Mediated Prevention (AMP) trials opened a new scientific frontier by showing that passively administered monoclonal broadly neutralizing antibodies (bnAbs) could prevent HIV-1 acquisition. Conducted across multiple geographic regions, including the United States, Brazil, Peru, Switzerland, and sub-Saharan Africa, the AMP trials revealed substantial regional heterogeneity in treatment efficacy. These differences, together with privacy and regulatory limits on…

利用辅助外部数据识别治疗受益者的共形筛选框架AI 解读

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…

多区域临床试验中协变量不匹配下的区域治疗效应推断AI 解读

Multi-regional clinical trials (MRCTs) are central to global drug development, enabling evaluation of treatment effects across diverse populations. A key challenge is valid and efficient inference for a region-specific estimand when the target region is small and differs from auxiliary regions in baseline covariates or unmeasured factors. We adopt an estimand-based framework and focus on the region-specific average treatment effect (RSATE) in a prespecified target region,…

因果推断的设计型理论AI 解读

Causal inference, as a major research area in statistics and data science, plays a central role across diverse fields such as medicine, economics, education, and the social sciences. Design-based causal inference begins with randomized experiments and emphasizes conducting statistical inference by leveraging the known randomization mechanism, thereby enabling identification and estimation of causal effects under weak model dependence. Grounded in the seminal works of Fisher…

COADVISE：随机对照试验中结合变量选择的协变量调整AI 解读

Adjusting for covariates in randomized controlled trials can enhance the credibility and efficiency of treatment effect estimation. However, handling numerous covariates and their complex (non-linear) transformations poses a challenge. Motivated by the case study of the Best Apnea Interventions for Research (BestAIR) trial data from the National Sleep Research Resource (NSRR), where the number of covariates (p=114) is comparable to the sample size (N=196), we propose a…

分层随机实验中的锐方差估计与因果自助法AI 解读

Randomized experiments are the gold standard for estimating treatment effects, and randomization serves as a reasoned basis for inference. In widely used stratified randomized experiments, randomization-based finite-population asymptotic theory enables valid inference for the average treatment effect, relying on normal approximation and a Neyman-type conservative variance estimator. However, when the sample size is small or the outcomes are skewed, the Neyman-type variance…

随机区组与再随机化实验中 Lasso 调整的设计型理论AI 解读

Blocking, a special case of rerandomization, is routinely implemented in the design stage of randomized experiments to balance the baseline covariates. This study proposes a regression adjustment method based on the least absolute shrinkage and selection operator (Lasso) to efficiently estimate the average treatment effect in randomized block experiments with high-dimensional covariates. We derive the asymptotic properties of the proposed estimator and outline the conditions…

个体数与时间长度均较大时的面板 ARMA-GARCH 模型推断AI 解读

We propose a panel ARMA-GARCH model to capture the dynamics of large panel data with $N$ individuals over $T$ time periods. For this model, we provide a two-step estimation procedure to estimate the ARMA parameters and GARCH parameters stepwisely. Under some regular conditions, we show that all of the proposed estimators are asymptotically normal with the convergence rate $(NT)^{-1/2}$, and they have the asymptotic biases when both $N$ and $T$ diverge to infinity at the same…