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We study problems with multiple missing covariates and partially observed responses. We develop a new framework to handle complex missing covariate scenarios via inverse probability weighting, regression adjustment, and a multiply-robust…

Methodology · Statistics 2021-11-04 Daniel Suen , Yen-Chi Chen

Case-cohort studies are conducted within cohort studies, wherein collection of exposure data is limited to a subset of the cohort, leading to a large proportion of missing data by design. Standard analysis uses inverse probability weighting…

This paper studies inference in cluster randomized trials where treatment status is determined according to a "matched pairs" design. Here, by a cluster randomized experiment, we mean one in which treatment is assigned at the level of the…

Econometrics · Economics 2025-08-14 Yuehao Bai , Jizhou Liu , Azeem M. Shaikh , Max Tabord-Meehan

Noncompliance and missing data often occur in randomized trials, which complicate the inference of causal effects. When both noncompliance and missing data are present, previous papers proposed moment and maximum likelihood estimators for…

Methodology · Statistics 2014-09-04 Hua Chen , Peng Ding , Zhi Geng , Xiao-Hua Zhou

We often seek to estimate the impact of an exposure naturally occurring or randomly assigned at the cluster-level. For example, the literature on neighborhood determinants of health continues to grow. Likewise, community randomized trials…

Methodology · Statistics 2021-07-08 Laura B. Balzer , Wenjing Zheng , Mark J. van der Laan , Maya L. Petersen

Return-to-baseline is an important method to impute missing values or unobserved potential outcomes when certain hypothetical strategies are used to handle intercurrent events in clinical trials. Current return-to-baseline approaches seen…

Methodology · Statistics 2021-11-19 Yongming Qu , Biyue Dai

Joint modeling technique is a recent advancement in effectively analyzing the longitudinal history of patients with the occurrence of an event of interest attached to it. This procedure is successfully implemented in biomarker studies to…

Methodology · Statistics 2021-01-08 Gajendra K. Vishwakarma , Atanu Bhattacharjee , Souvik Banerjee

Boosting techniques from the field of statistical learning have grown to be a popular tool for estimating and selecting predictor effects in various regression models and can roughly be separated in two general approaches, namely gradient…

Methodology · Statistics 2019-12-16 Colin Griesbach , Andreas Groll , Elisabeth Waldmann

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

Trial-based cost-effectiveness analyses (CEAs) are an important source of evidence in the assessment of health interventions. In these studies, cost and effectiveness outcomes are commonly measured at multiple time points, but some…

Methodology · Statistics 2022-03-30 Andrea Gabrio , Catrin Plumpton , Sube Banerjee , Baptiste Leurent

Evaluating heterogeneity of treatment effects (HTE) across subgroups is common in both randomized trials and observational studies. Although several statistical challenges of HTE analyses including low statistical power and multiple…

Methodology · Statistics 2024-07-10 Noorie Hyun , Abisola E. Idu , Andrea J. Cook , Jennifer F. Bobb

Missing covariates in regression or classification problems can prohibit the direct use of advanced tools for further analysis. Recent research has realized an increasing trend towards the usage of modern Machine Learning algorithms for…

Machine Learning · Statistics 2022-03-23 Burim Ramosaj , Justus Tulowietzki , Markus Pauly

Missing data arise in most applied settings and are ubiquitous in electronic health records (EHR). When data are missing not at random (MNAR) with respect to measured covariates, sensitivity analyses are often considered. These post-hoc…

Methodology · Statistics 2023-07-11 Alexander W. Levis , Rajarshi Mukherjee , Rui Wang , Heidi Fischer , Sebastien Haneuse

When outcomes are missing for reasons beyond an investigator's control, there are two different ways to adjust a parameter estimate for covariates that may be related both to the outcome and to missingness. One approach is to model the…

Methodology · Statistics 2008-12-18 Joseph D. Y. Kang , Joseph L. Schafer

Although randomized experiments are widely regarded as the gold standard for estimating causal effects, missing data of the pretreatment covariates makes it challenging to estimate the subgroup causal effects. When the missing data…

Statistics Theory · Mathematics 2014-01-08 Peng Ding , Zhi Geng

Health economic evaluations based on patient-level data collected alongside clinical trials~(e.g. health related quality of life and resource use measures) are an important component of the process which informs resource allocation…

Applications · Statistics 2020-05-25 Andrea Gabrio , Rachael Hunter , Alexina J. Mason , Gianluca Baio

When the difference between treatments in a clinical trial is estimated by a difference in means, then it is well known that randomization ensures unbiassed estimation, even if no account is taken of important baseline covariates. However,…

Statistics Theory · Mathematics 2014-07-22 J. N. S. Matthews , Nuri H. Badi

External information, such as prior information or expert opinions, can play an important role in the design, analysis and interpretation of clinical trials. However, little attention has been devoted thus far to incorporating external…

Applications · Statistics 2013-04-24 Minge Xie , Regina Y. Liu , C. V. Damaraju , William H. Olson

We investigate the problem of calibration and assessment of predictive rules in prognostic designs when missing values are present in the predictors. Our paper has two key objectives which are entwined. The first is to investigate how the…

Methodology · Statistics 2018-10-12 B. J. A. Mertens , E. Banzato , L. C. de Wreede

Misclassification in binary outcomes is not uncommon and statistical methods to investigate its impact on policy-driving study results are lacking. While misclassifying binary outcomes is a statistically ubiquitous phenomena, we focus on…