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In nutritional and environmental epidemiology, exposures are impractical to measure accurately, while practical measures for these exposures are often subject to substantial measurement error. Regression calibration is among the most used…

Methodology · Statistics 2026-01-27 Zexiang Li , Donna Spiegelman , Molin Wang , Zuoheng Wang , Xin Zhou

In epidemiology, obtaining accurate individual exposure measurements can be costly and challenging. Thus, these measurements are often subject to error. Regression calibration with a validation study is widely employed as a study design and…

Methodology · Statistics 2026-02-24 Zexiang Li , Donna Spiegelman , Molin Wang , Zuoheng Wang , Xin Zhou

When assessing causal effects, determining the target population to which the results are intended to generalize is a critical decision. Randomized and observational studies each have strengths and limitations for estimating causal effects…

Methodology · Statistics 2022-10-21 Irina Degtiar , Sherri Rose

Data analysis based on information from several sources is common in economic and biomedical studies. This setting is often referred to as the data fusion problem, which differs from traditional missing data problems since no complete data…

Methodology · Statistics 2022-04-07 Wei Li , Shanshan Luo , Wangli Xu

Despite the general consensus in transport research community that model calibration and validation are necessary to enhance model predictive performance, there exist significant inconsistencies in the literature. This is primarily due to a…

Methodology · Statistics 2023-09-18 Samson Ting , Thomas Lymburn , Thomas Stemler , Yuchao Sun , Michael Small

Observational studies can play a useful role in assessing the comparative effectiveness of competing treatments. In a clinical trial the randomization of participants to treatment and control groups generally results in well-balanced groups…

Generalizability and transportability methods have been proposed to address the external validity bias of randomized clinical trials that results from differences in the distribution of treatment effect modifiers between trial and target…

Many lifestyle intervention trials depend on collecting self-reported outcomes, like dietary intake, to assess the intervention's effectiveness. Self-reported outcome measures are subject to measurement error, which could impact treatment…

Methodology · Statistics 2020-04-06 Benjamin Ackerman , Juned Siddique , Elizabeth A. Stuart

Clinical machine learning applications are often plagued with confounders that can impact the generalizability and predictive performance of the learners. Confounding is especially problematic in remote digital health studies where the…

In multi-center clinical trials, due to various reasons, the individual-level data are strictly restricted to be assessed publicly. Instead, the summarized information is widely available from published results. With the advance of…

Methodology · Statistics 2021-01-05 Jing Qin , Yukun Liu , Pengfei Li

The generalizability of empirical findings to new environments, settings or populations, often called "external validity," is essential in most scientific explorations. This paper treats a particular problem of generalizability, called…

Methodology · Statistics 2015-03-06 Judea Pearl , Elias Bareinboim

Transporting findings from a study population to a target population is central to evidence-based decision-making in real-world settings. Most existing methods require individual-level data from both populations to account for covariate…

Methodology · Statistics 2026-03-04 Ying Sheng , Yifei Sun , Chiung-Yu Huang

Generalization methods offer a powerful solution to one of the key drawbacks of randomized controlled trials (RCTs): their limited representativeness. By enabling the transport of treatment effect estimates to target populations subject to…

Methodology · Statistics 2025-05-20 Ahmed Boughdiri , Clément Berenfeld , Julie Josse , Erwan Scornet

Augmenting the control arm in clinical trials with external data can improve statistical power for demonstrating treatment effects. In many time-to-event outcome trials, participants are subject to truncation by death. Direct application of…

Methodology · Statistics 2025-06-24 Zehao Su , Helene C. W. Rytgaard , Henrik Ravn , Frank Eriksson

Many scientific questions in biomedical, environmental, and psychological research involve understanding the effects of multiple factors on outcomes. While factorial experiments are ideal for this purpose, randomized controlled treatment…

Methodology · Statistics 2025-12-03 Ruoqi Yu , Peng Ding

Evaluation of clinical prediction models across multiple clusters, whether centers or datasets, is becoming increasingly common. A comprehensive evaluation includes an assessment of the agreement between the estimated risks and the observed…

Methodology · Statistics 2026-04-22 Lasai Barreñada , Bavo D. C. Campo , Laure Wynants , Ben Van Calster

Learning about causal effects in target populations and their subsets may be facilitated by combining information from multiple sources. One major class of study designs that combine information involves appending an index study with data…

When estimating an effect of an action with a randomized or observational study, that study is often not a random sample of the desired target population. Instead, estimates from that study can be transported to the target population.…

Data from both a randomized trial and an observational study are sometimes simultaneously available for evaluating the effect of an intervention. The randomized data typically allows for reliable estimation of average treatment effects but…

Methodology · Statistics 2021-12-01 David Cheng , Tianxi Cai

There is increasing interest in combining information from experimental studies, including randomized and single-group trials, with information from external experimental or observational data sources. Such efforts are usually motivated by…

Methodology · Statistics 2026-04-23 Lawson Ung , Guanbo Wang , Sebastien Haneuse , Miguel A. Hernán , Issa J. Dahabreh
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