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Comparative binary outcome data are of fundamental interest in statistics and are often pooled in meta-analyses. Here we examine the simplest case where for each study there are two patient groups and a binary event of interest, giving rise…

Methodology · Statistics 2018-06-12 Rose Baker , Dan Jackson

Covariate adjustment and methods of incorporating historical data in randomized clinical trials (RCTs) each provide opportunities to increase trial power. We unite these approaches for the analysis of RCTs with binary outcomes based on the…

Methodology · Statistics 2022-12-21 Alyssa M. Vanderbeek , Jessica L. Ross , David P. Miller , Alejandro Schuler

In the statistical literature, a number of methods have been proposed to ensure valid inference about marginal effects of variables on a longitudinal outcome in settings with irregular monitoring times. However, the potential biases due to…

Methodology · Statistics 2021-12-23 Janie Coulombe , Erica E M Moodie , Robert W Platt

In recent years, there has been a growing interest in the prediction of individualized treatment effects. While there is a rapidly growing literature on the development of such models, there is little literature on the evaluation of their…

Methodology · Statistics 2023-12-22 J Hoogland , O Efthimiou , TL Nguyen , TPA Debray

We consider the problem of variance reduction in randomized controlled trials, through the use of covariates correlated with the outcome but independent of the treatment. We propose a machine learning regression-adjusted treatment effect…

Machine Learning · Statistics 2022-01-07 Yongyi Guo , Dominic Coey , Mikael Konutgan , Wenting Li , Chris Schoener , Matt Goldman

In light of newly developed standardization methods, we evaluate, via simulation study, how propensity score weighting and standardization -based approaches compare for obtaining estimates of the marginal odds ratio and the marginal hazard…

Methodology · Statistics 2023-10-10 Harlan Campbell , Julie E Park , Jeroen P Jansen , Shannon Cope

Higher-Order Influence Functions (HOIF), developed in a series of papers over the past twenty years, are a fundamental theoretical device for constructing rate-optimal causal-effect estimators from observational studies. However, the value…

Methodology · Statistics 2025-11-19 Sihui Zhao , Xinbo Wang , Lin Liu , Xin Zhang

Binary classification rules based on covariates typically depend on simple loss functions such as zero-one misclassification. Some cases may require more complex loss functions. For example, individual-level monitoring of HIV-infected…

Machine Learning · Statistics 2019-05-14 Yizhen Xu , Tao Liu , Michael J. Daniels , Rami Kantor , Ann Mwangi , Joseph W. Hogan

Covariate balance is crucial in obtaining unbiased estimates of treatment effects in observational studies. Methods based on inverse probability weights have been widely used to estimate treatment effects with observational data. Machine…

Methodology · Statistics 2021-04-08 Michele Santacatterina

We often seek to estimate the causal effect of an exposure on a particular outcome in both randomized and observational settings. One such estimation method is the covariate-adjusted residuals estimator, which was designed for individually…

Methodology · Statistics 2019-10-28 Stephen A. Lauer , Nicholas G. Reich , Laura B. Balzer

The paper considers the problem of out-of-sample risk estimation under the high dimensional settings where standard techniques such as $K$-fold cross validation suffer from large biases. Motivated by the low bias of the leave-one-out cross…

Methodology · Statistics 2020-02-12 Kamiar Rahnama Rad , Arian Maleki

Randomized clinical trials typically aim to estimate a marginal treatment effect. While covariate adjustment can improve precision, it may change the estimand in nonlinear models due to noncollapsibility, leading to conditional rather than…

Methodology · Statistics 2026-05-25 Leticia Wuethrich , Torsten Hothorn

Covariate adjustment is widely recommended to improve statistical efficiency in randomized clinical trials (RCTs), yet empirical evidence comparing available strategies remains limited. This lack of real-world evaluation leaves unresolved…

Applications · Statistics 2026-02-03 Yulin Shao , Liangbo Lyu , Menggang Yu , Bingkai Wang

A simple device for balancing for a continuous covariate in clinical trials is to stratify by whether the covariate is above or below some target value, typically the predicted median. This raises an issue as to which model should be used…

Methodology · Statistics 2025-10-24 Stephen Senn , Franz König , Martin Posch

In observational studies, propensity scores are commonly estimated by maxi- mum likelihood but may fail to balance high-dimensional pre-treatment covariates even after specification search. We introduce a general framework that unifies and…

Methodology · Statistics 2017-03-22 Qingyuan Zhao

We consider a randomized controlled trial between two groups. The objective is to identify a population with characteristics such that the test therapy is more effective than the control therapy. Such a population is called a subgroup. This…

Methodology · Statistics 2021-12-06 Shintaro Yuki , Kensuke Tanioka , Hiroshi Yadohisa

Inferring the causal effect of a treatment on an outcome in an observational study requires adjusting for observed baseline confounders to avoid bias. However, adjusting for all observed baseline covariates, when only a subset are…

Methodology · Statistics 2021-02-04 Wen Wei Loh , Stijn Vansteelandt

Cluster-randomized trials (CRTs) involve randomizing entire groups of participants -- called clusters -- to treatment arms but are often comprised of a limited or fixed number of available clusters. While covariate adjustment can account…

Methodology · Statistics 2022-11-29 Angela Y. Zhu , Nandita Mitra , Karla Hemming , Michael O. Harhay , Fan Li

Covariate adjustment is desired by both practitioners and regulators of randomized clinical trials because it improves precision for estimating treatment effects. However, covariate adjustment presents a particular challenge in…

Methodology · Statistics 2023-07-20 Yunfan Li , Jessica L. Ross , Aaron M. Smith , David P. Miller

Covariate shift relaxes the widely-employed independent and identically distributed (IID) assumption by allowing different training and testing input distributions. Unfortunately, common methods for addressing covariate shift by trying to…

Machine Learning · Computer Science 2018-01-02 Anqi Liu , Brian D. Ziebart