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Randomized trials are considered the gold standard for making informed decisions in medicine, yet they often lack generalizability to the patient populations in clinical practice. Observational studies, on the other hand, cover a broader…

Methodology · Statistics 2026-04-14 Piersilvio De Bartolomeis , Javier Abad , Konstantin Donhauser , Fanny Yang

The statistical efficiency of randomized clinical trials can be improved by incorporating information from baseline covariates (i.e., pre-treatment patient characteristics). This can be done in the design stage using stratified (permutated…

Methodology · Statistics 2025-02-04 Zhiwei Zhang

In randomized controlled trials without interference, regression adjustment is widely used to enhance the efficiency of treatment effect estimation. This paper extends this efficiency principle to settings with network interference, where a…

Methodology · Statistics 2025-02-18 Xinyuan Fan , Chenlei Leng , Weichi Wu

Rerandomization systematically reduces chance imbalance and can improve the efficiency of the average treatment effect estimator in randomized experiments. While the asymptotic properties of finite-dimensional M-estimators under…

Methodology · Statistics 2026-04-28 Xinyuan Chen , Fan Li

There has been a split in the statistics community about the need for taking covariates into account in the design phase of a clinical trial. There are many advocates of using stratification and covariate-adaptive randomization to promote…

Methodology · Statistics 2011-02-21 William F. Rosenberger , Oleksandr Sverdlov

Randomized Controlled Trials (RCTs) may suffer from limited scope. In particular, samples may be unrepresentative: some RCTs over- or under- sample individuals with certain characteristics compared to the target population, for which one…

Methodology · Statistics 2024-03-15 Bénédicte Colnet , Julie Josse , Gaël Varoquaux , Erwan Scornet

Tuning parameters are parameters involved in an estimating procedure for the purpose of reducing the risk of some other estimator. Examples include the degree of penalization in penalized regression and likelihood problems, as well as the…

Statistics Theory · Mathematics 2026-03-31 Ingrid Dæhlen , Nils Lid Hjort , Ingrid Hobæk Haff

In unsupervised combinatorial optimization (UCO), during training, one aims to have continuous decisions that are promising in a probabilistic sense for each training instance, which enables end-to-end training on initially discrete and…

Machine Learning · Computer Science 2025-06-23 Fanchen Bu , Kijung Shin

Estimating heterogeneous treatment effects is central to data-driven decision-making, yet industrial applications often face a fundamental tension between limited randomized controlled trial (RCT) budgets and abundant but biased…

Randomized controlled trials (RCTs) are the gold standard for estimating heterogeneous treatment effects, yet they are often underpowered for detecting effect heterogeneity. Large observational studies (OS) can supplement RCTs for…

Machine Learning · Computer Science 2026-04-07 Amir Asiaee , Samhita Pal

Estimating the causal effect of a treatment or health policy with observational data can be challenging due to an imbalance of and a lack of overlap between treated and control covariate distributions. In the presence of limited overlap,…

Methodology · Statistics 2025-03-24 Martha Barnard , Jared D. Huling , Julian Wolfson

Baseline covariates in randomized experiments are often used in the estimation of treatment effects, for example, when estimating treatment effects within covariate-defined subgroups. In practice, however, covariate values may be missing…

Methodology · Statistics 2023-08-29 Gauri Kamat , Jerome P. Reiter

Nonignorable missingness and noncompliance can occur even in well-designed randomized experiments making the intervention effect that the experiment was designed to estimate nonidentifiable. Nonparametric causal bounds provide a way to…

Statistics Theory · Mathematics 2020-10-13 Erin E. Gabriel , Arvid Sjölander , Michael C. Sachs

In variable selection, most existing screening methods focus on marginal effects and ignore dependence between covariates. To improve the performance of selection, we incorporate pairwise effects in covariates for screening and…

Methodology · Statistics 2019-02-12 Siliang Gong , Kai Zhang , Yufeng Liu

Covariate adjustment aims to improve the statistical efficiency of randomized trials by incorporating information from baseline covariates. Popular methods for covariate adjustment include analysis of covariance for continuous endpoints and…

Methodology · Statistics 2025-05-09 Zhiwei Zhang , Ya Wang , Dong Xi

The ability to generalize experimental results from randomized control trials (RCTs) across locations is crucial for informing policy decisions in targeted regions. Such generalization is often hindered by the lack of identifiability due to…

Econometrics · Economics 2021-12-10 Xinkun Nie , Guido Imbens , Stefan Wager

Randomized controlled trials (RCTs) are the benchmark for causal inference, yet field implementation can drift from the registered design or, by chance, yield imbalances. We introduce a remote audit -- a preregistrable, design-based…

Methodology · Statistics 2025-10-21 Connor T. Jerzak , Adel Daoud

Causal decomposition analysis aims to assess the effect of modifying risk factors on reducing social disparities in outcomes. Recently, this analysis has incorporated individual characteristics when modifying risk factors by utilizing…

Machine Learning · Statistics 2025-09-16 Soojin Park , Suyeon Kang , Chioun Lee

Methods to correct class imbalance, i.e. imbalance between the frequency of outcome events and non-events, are receiving increasing interest for developing prediction models. We examined the effect of imbalance correction on the performance…

Methodology · Statistics 2022-02-21 Ruben van den Goorbergh , Maarten van Smeden , Dirk Timmerman , Ben Van Calster

The gold standard for identifying causal relationships is a randomized controlled experiment. In many applications in the social sciences and medicine, the researcher does not control the assignment mechanism and instead may rely upon…

Applications · Statistics 2016-11-22 Johann Gagnon-Bartsch , Yotam Shem-Tov