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用于处理效应估计的预测驱动数据融合AI 解读

Randomized controlled trials (RCTs) identify treatment effects without confounding but are often small, whereas observational studies (OBS) are large but may be confounded. Many estimators combining a small RCT with a large OBS have been developed for the average treatment effect (ATE) and the conditional ATE (CATE). However, existing ATE estimators either make assumptions on the OBS or do not borrow enough power from them. The CATE has been studied less than the ATE.…

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…

多暴露与多结局空间回归的谱混杂调整AI 解读

Characterizing social vulnerability is fundamental to disaster response planning. Numerous vulnerability indicators have been developed, but they are typically not validated for their predictive power over the outcomes of interest. As with many environmental health studies where interventions are impractical or unethical, validation of social vulnerability against public health outcomes is observational and relies on spatially-dependent data. Observational studies are…

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…

整合随机试验、真实世界数据、人工智能与统计学：下一代证据综合AI 解读

Randomized controlled trials (RCTs)have been the cornerstone of clinical evidence; however, their cost, duration, and restrictive eligibility criteria limit power and external validity. Studies using real-world data (RWD), historically considered less reliable for establishing causality, are now recognized as an important source of real-world evidence (RWE). In parallel, artificial intelligence and machine learning (AI/ML) are increasingly used throughout the drug…

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…

含未观测混杂因素的贝叶斯结构化中介分析AI 解读

We explore methods to reduce the impact of unobserved confounders on the causal mediation analysis of high-dimensional mediators with spatially smooth structures, such as brain imaging data. The key approach is to incorporate the latent individual effects, which influence the structured mediators, as unobserved confounders in the outcome model, thereby potentially debiasing the mediation effects. We develop BAyesian Structured Mediation analysis with Unobserved confounders…

偏好性采样下的空间因果推断：海洋保护区影响评估AI 解读

Marine Protected Areas (MPAs) have been established globally to conserve marine resources. Given their maintenance costs and impact on commercial fishing, it is critical to evaluate their effectiveness to support future conservation. In this paper, we use data collected from the Australian coast to estimate the effect of MPAs on biodiversity. Environmental studies such as these are often observational, and processes of interest exhibit spatial dependence, which presents…

总体异质性下双样本孟德尔随机化的稳健框架AI 解读

Mendelian randomization is a powerful tool for causal inference in observational studies. The two-sample summary-data design, which estimates genetic associations with exposures and outcomes in separate cohorts, is the most widely used Mendelian randomization approach in large-scale genomic studies. However, this approach relies on a strong assumption of population homogeneity across the two samples. In practice, available samples often differ in ancestry, demographics,…

通过自适应借用外部对照改善试验中的处理效应估计AI 解读

Randomized controlled trials (RCTs) often suffer from limited inferential efficiency in estimating treatment effects due to their small sample sizes. In recent years, incorporating external controls (ECs) has gained increasing attention as an effective way to augment small RCTs and thereby enhance estimation efficiency. However, ECs are not always comparable to RCTs, and direct borrowing without careful evaluation can introduce substantial bias and, paradoxically, undermine…

样本分割回归 SREG：高维调查数据的稳健估计量AI 解读

Model-assisted regression estimation is fundamental in survey sampling for incorporating auxiliary information. However, when the auxiliary dimension grows with the sample size, the standard Generalized regression (GREG) estimator can exhibit non-negligible bias under informative sampling, even when the working model is correctly specified. This failure stems from the double use of sampled outcomes simultaneously for fitting the regression and for forming the residual…

多暴露与多结局的空间因果张量补全：PFAS 污染健康效应应用AI 解读

Per- and polyfluoroalkyl substances (PFAS) are typically encountered as mixtures of distinct chemicals with distinct effects on multiple health outcomes. Estimating joint causal effects using spatially-dependent observed data is challenging. We propose a spatial causal tensor completion framework that jointly models multiple exposures and outcomes within a low-rank tensor structure, while adjusting for observed confounders and latent spatial confounders. This method combines…

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,…

从分类视角利用自适应 LASSO 估计可解释的最优个体化治疗规则AI 解读

Real-world data (RWD) gains growing interests to provide a representative sample of the population for selecting the optimal treatment options. However, existing complex black box methods for estimating individualized treatment rules (ITR) from RWD have problems in interpretability and convergence. Providing an interpretable and sparse ITR can be used to overcome the limitation of existing methods. We developed an algorithm using Adaptive LASSO to predict optimal…

平均处理效应的增广匹配加权估计量AI 解读

Propensity score matching (PSM) and augmented inverse propensity weighting (AIPW) are widely used in observational studies to estimate causal effects. The two approaches present complementary features. The AIPW estimator is doubly robust and locally efficient but can be unstable when the propensity scores are close to zero or one due to weighting by the inverse of the propensity score. On the other hand, PSM circumvents the instability of propensity score weighting but it…

利用效应度量可迁移性的异质性感知联邦因果推断AI 解读

Federated learning of causal estimands offers a powerful strategy to improve estimation efficiency by leveraging data from multiple study sites while preserving privacy. Existing literature has primarily focused on the average treatment effect using single data source, whereas our work addresses a broader class of causal measures across multiple sources. We derive and compare semiparametrically efficient estimators under two transportability assumptions, which impose…

通过张量补全推断序贯处理的因果效应AI 解读

Marginal Structural Models (MSMs) are popular for causal inference of sequential treatments in longitudinal observational studies, which however are sensitive to model misspecification. To achieve flexible modeling, we envision the potential outcomes to form a three-dimensional tensor indexed by subject, time, and treatment regime and propose a tensorized history-restricted MSM (HRMSM). The semi-parametric tensor factor model allows us to leverage the underlying low-rank…

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…

加权平均处理效应的速率双重稳健估计AI 解读

The weighted average treatment effect (WATE) defines a versatile class of causal estimands for populations characterized by propensity score weights, including the average treatment effect (ATE), treatment effect on the treated (ATT), on controls (ATC), and for the overlap population (ATO). WATE has broad applicability in social and medical research, as many datasets from these fields align with its framework. However, the literature lacks a systematic investigation into the…

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…

用于帕金森病生物标志物发现的稀疏因果结构学习：惩罚化 FCIAI 解读

Parkinson's disease (PD) is a progressive neurodegenerative disorder that lacks reliable early-stage biomarkers for diagnosis, prognosis, and therapeutic monitoring. While cerebrospinal fluid (CSF) biomarkers, such as alpha-synuclein seed amplification assays (alphaSyn-SAA), offer diagnostic potential, their clinical utility is limited by invasiveness and incomplete specificity. Plasma biomarkers provide a minimally invasive alternative, but their mechanistic role in PD…

迈向连续处理的 R-learnerAI 解读

The R-learner is widely used in causal inference due to its flexibility and efficiency in estimating the conditional average treatment effect. However, extending the R-learner framework from binary to continuous treatments introduces a non-identifiability issue, as the functional zero constraint inherent to the conditional average treatment effect cannot be directly imposed in the R-loss under continuous treatments. To address this, we propose a two-step identification…

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…

存在试验期间事件的外部对照试验：双重稳健综合敏感性分析AI 解读

Externally controlled trials are crucial in clinical development when randomized controlled trials are unethical or impractical. These trials consist of a full treatment arm with the experimental treatment and a full external control arm. However, they present significant challenges in learning the treatment effect due to the lack of randomization and a parallel control group. Besides baseline incomparability, outcome mean non-exchangeability, caused by differences in…