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It is becoming increasingly common for researchers to consider incorporating external information from large studies to improve the accuracy of statistical inference instead of relying on a modestly sized dataset collected internally. With…

Methodology · Statistics 2021-07-20 Tian Gu , Jeremy M. G. Taylor , Bhramar Mukherjee

A full parametric and linear specification may be insufficient to capture complicated patterns in studies exploring complex features, such as those investigating age-related changes in brain functional abilities. Alternatively, a partially…

Methodology · Statistics 2024-02-07 Jia Liang , Shuo Chen , Peter Kochunov , L Elliot Hong , Chixiang Chen

Background: External validation is essential for assessing the transportability of predictive models. However, its interpretation is often confounded by differences between external and development populations. This study introduces a…

Contemporary sample size calculations for external validation of risk prediction models require users to specify fixed values of assumed model performance metrics alongside target precision levels (e.g., 95% CI widths). However, due to the…

Applications · Statistics 2026-02-13 Mohsen Sadatsafavi , Paul Gustafson , Solmaz Setayeshgar , Laure Wynants , Richard D Riley

Suppose we have individual data from an internal study and various summary statistics from relevant external studies. External summary statistics have the potential to improve statistical inference for the internal population; however, it…

Methodology · Statistics 2026-02-06 Wenjie Hu , Ruoyu Wang , Wei Li , Wang Miao

Databases of electronic health records (EHRs) are increasingly used to inform clinical decisions. Machine learning methods can find patterns in EHRs that are predictive of future adverse outcomes. However, statistical models may be built…

Machine Learning · Statistics 2018-12-04 Andrew C. Miller , Ziad Obermeyer , Sendhil Mullainathan

In cluster analysis, it can be useful to interpret the partition built from the data in the light of external categorical variables which were not directly involved to cluster the data. An approach is proposed in the model-based clustering…

A new method, with an application program in Matlab code, is proposed for testing item performance models on empirical databases. This method uses data intraclass correlation statistics as expected correlations to which one compares simple…

Motivated by two case studies using primary care records from the Clinical Practice Research Datalink, we describe statistical methods that facilitate the analysis of tall data, with very large numbers of observations. Our focus is on…

Methodology · Statistics 2018-05-14 Kirsty Rhodes , Rebecca Turner , Rupert Payne , Ian White

Consider the problem of estimating average treatment effects when a large number of covariates are used to adjust for possible confounding through outcome regression and propensity score models. The conventional approach of model building…

Statistics Theory · Mathematics 2018-01-31 Zhiqiang Tan

We present a general framework for using existing data to estimate the efficiency gain from using a covariate-adjusted estimator of a marginal treatment effect in a future randomized trial. We describe conditions under which it is possible…

Methodology · Statistics 2021-05-03 Xiudi Li , Sijia Li , Alex Luedtke

One fundamental statistical question for research areas such as precision medicine and health disparity is about discovering effect modification of treatment or exposure by observed covariates. We propose a semiparametric framework for…

Methodology · Statistics 2020-08-04 Muxuan Liang , Menggang Yu

In modern data analysis, information is frequently collected from multiple sources, often leading to challenges such as data heterogeneity and imbalanced sample sizes across datasets. Robust and efficient data integration methods are…

Methodology · Statistics 2026-01-06 Facheng Yu , Zhen Qi , Yuqian Zhang

A prediction model is most useful if it generalizes beyond the development data with external validations, but to what extent should it generalize remains unclear. In practice, prediction models are externally validated using data from very…

Machine Learning · Computer Science 2023-04-11 Yilin Ning , Victor Volovici , Marcus Eng Hock Ong , Benjamin Alan Goldstein , Nan Liu

In machine learning, the selection of a promising model from a potentially large number of competing models and the assessment of its generalization performance are critical tasks that need careful consideration. Typically, model selection…

Machine Learning · Statistics 2023-02-06 Pascal Rink , Werner Brannath

We consider a general statistical estimation problem involving a finite-dimensional target parameter vector. Beyond an internal data set drawn from the population distribution, external information, such as additional individual data or…

Methodology · Statistics 2025-07-31 Guorong Dai , Lingxuan Shao , Jinbo Chen

Randomized clinical trials are the gold standard for analyzing treatment effects, but high costs and ethical concerns can limit recruitment, potentially leading to invalid inferences. Incorporating external trial data with similar…

Methodology · Statistics 2024-09-09 Yujia Gu , Hanzhong Liu , Wei Ma

We consider the problem of estimating personalized treatment policies that are "externally valid" or "generalizable": they perform well in target populations that differ from the experimental (or training) population from which the data are…

Econometrics · Economics 2025-11-10 Christopher Adjaho , Timothy Christensen

Machine learning (ML) models deployed in healthcare systems must face data drawn from continually evolving environments. However, researchers proposing such models typically evaluate them in a time-agnostic manner, splitting datasets…

Machine Learning · Computer Science 2023-07-21 Helen Zhou , Yuwen Chen , Zachary C. Lipton

Practitioners in diverse fields such as healthcare, economics and education are eager to apply machine learning to improve decision making. The cost and impracticality of performing experiments and a recent monumental increase in electronic…

Machine Learning · Computer Science 2023-08-01 Fredrik D. Johansson , Uri Shalit , Nathan Kallus , David Sontag