论文检索

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…

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,…

融合随机试验与真实世界右删失数据的异质处理效应推断AI 解读

The heterogeneous treatment effect plays a crucial role in precision medicine.There is evidence that real-world data, even subject to biases, can be employed as supplementary evidence for randomized clinical trials to improve the statistical efficiency of the heterogeneous treatment effect estimation. In this paper, for survival data with right censoring, we consider estimating the heterogeneous treatment effect, defined as the difference of the treatment-specific…

删失与隐藏混杂下高维随机试验和真实世界数据的整合分析AI 解读

In this study, we focus on estimating the heterogeneous treatment effect (HTE) for survival outcome. The outcome is subject to censoring and the number of covariates is high-dimensional. We utilize data from both the randomized controlled trial (RCT), considered as the gold standard, and real-world data (RWD), possibly affected by hidden confounding factors. To achieve a more efficient HTE estimate, such integrative analysis requires great insight into the data generation…

带阈值识别的生物标志物引导自适应富集生存试验设计AI 解读

Biomarker-guided designs are increasingly used to evaluate personalized treatments based on patients' biomarker status in Phase II and III clinical trials. With adaptive enrichment, these designs can improve the efficiency of evaluating the treatment effect in biomarker-positive patients by increasing their proportion in the randomized trial. While time-to-event outcomes are often used as the primary endpoint to measure treatment effects for a new therapy in severe diseases…

多区域临床试验中基于限制平均生存时间的处理效应及区域修饰因素推断AI 解读

Multi-regional clinical trials (MRCTs) play an increasingly crucial role in global pharmaceutical development by expediting data gathering and regulatory approval across diverse patient populations. However, differences in recruitment practices and regional demographics often lead to variations in study participant characteristics, potentially biasing treatment effect estimates and undermining treatment effect consistency assessment across regions. To address this challenge,…

利用个体数据与限制平均生存时间回归开展生存结局网络荟萃分析AI 解读

Restricted mean survival time (RMST) models have gained popularity when analyzing time-to-event outcomes because RMST models offer more straightforward interpretations of treatment effects with fewer assumptions than hazard ratios commonly estimated from Cox models. However, few network meta-analysis (NMA) methods have been developed using RMST. In this paper, we propose advanced RMST NMA models when individual participant data are available. Our models allow us to study…

将随机试验的生存处理效应推广至目标总体的双重稳健估计AI 解读

In the presence of heterogeneity between the randomized controlled trial (RCT) participants and the target population, evaluating the treatment effect solely based on the RCT often leads to biased quantification of the real-world treatment effect. To address the problem of lack of generalizability for the treatment effect estimated by the RCT sample, we leverage observational studies with large samples that are representative of the target population. This paper concerns…