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Real-World Data (RWD), with its large sample sizes and rich clinical detail, offers a compelling alternative to randomized controlled trials (RCTs) for studying treatment effects in diverse and complex patient populations. However, its…

Applications · Statistics 2026-05-26 Yifei Xu , Hwiyoung Lee , Zhenyao Ye , Yezhi Pan , Jingsong Zhou , Yun Yang , Chixiang Chen , Shuo Chen

Multiple imputation (MI) has been widely applied to missing value problems in biomedical, social and econometric research, in order to avoid improper inference in the downstream data analysis. In the presence of high-dimensional data,…

Methodology · Statistics 2023-05-04 Zhiqi Bu , Zongyu Dai , Yiliang Zhang , Qi Long

In cluster-randomized trials (CRTs), missing data can occur in various ways, including missing values in outcomes and baseline covariates at the individual or cluster level, or completely missing information for non-participants. Among the…

Methodology · Statistics 2025-11-06 Bingkai Wang , Fan Li , Rui Wang

The problem of monotone missing data has been broadly studied during the last two decades and has many applications in different fields such as bioinformatics or statistics. Commonly used imputation techniques require multiple iterations…

Machine Learning · Computer Science 2020-09-25 Thu Nguyen , Duy H. M. Nguyen , Huy Nguyen , Binh T. Nguyen , Bruce A. Wade

Estimating heterogeneous treatment effects with machine learning has attracted substantial attention in both academic research and industrial practice. However, the two communities often evaluate models under markedly different conditions.…

Machine Learning · Computer Science 2026-05-26 George Panagopoulos

We evaluate the performance of targeted maximum likelihood estimation (TMLE) for estimating the average treatment effect in missing data scenarios under varying levels of positivity violations. We employ model- and design-based simulations,…

Methodology · Statistics 2026-05-12 Christoph Wiederkehr , Christian Heumann , Michael Schomaker

Public policy-makers use cost-effectiveness analyses (CEA) to decide which health and social care interventions to provide. Appropriate methods have not been developed for handling missing data in complex settings, such as for CEA that use…

Methodology · Statistics 2012-06-27 Karla Diaz-Ordaz , Michael G. Kenward , Richard Grieve

Physics education researchers (PER) commonly use complete-case analysis to address missing data. For complete-case analysis, researchers discard all data from any student who is missing any data. Despite its frequent use, no PER article we…

Physics Education · Physics 2019-02-20 Jayson Nissen , Robin Donatello , Ben Van Dusen

While machine learning (ML) methods have received a lot of attention in recent years, these methods are primarily for prediction. Empirical researchers conducting policy evaluations are, on the other hand, pre-occupied with causal problems,…

Machine Learning · Statistics 2019-03-04 Noemi Kreif , Karla DiazOrdaz

When analyzing incomplete data, is it better to use multiple imputation (MI) or full information maximum likelihood (ML)? In large samples ML is clearly better, but in small samples ML's usefulness has been limited because ML commonly uses…

Methodology · Statistics 2017-03-24 Paul T. von Hippel

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

Machine learning iterative imputation methods have been well accepted by researchers for imputing missing data, but they can be time-consuming when handling large datasets. To overcome this drawback, parallel computing strategies have been…

Applications · Statistics 2020-04-24 Shangzhi Hong , Yuqi Sun , Hanying Li , Henry S. Lynn

Background: Multiple imputation is often used to reduce bias and gain efficiency when there is missing data. The most appropriate imputation method depends on the model the analyst is interested in fitting. Several imputation approaches…

Methodology · Statistics 2022-11-29 Matthew J. Smith , Matteo Quartagno , Edmund Njeru Njagi

In applications of linear mixed-effects models, experimenters often desire uncertainty quantification for random quantities, like predicted treatment effects for unobserved individuals or groups. For example, consider an agricultural…

Methodology · Statistics 2022-10-19 Nicholas Syring , Fernando Miguez , Jarad Niemi

Missing data is prevalent in tabular machine learning (ML) models, and different missing data treatment methods can significantly affect ML model training results. However, little is known about how ML researchers and engineers choose…

Machine Learning · Computer Science 2025-03-24 Wanyi Chen , Mary Cummings

In pharmacoepidemiology, safety and effectiveness are frequently evaluated using readily available administrative and electronic health records data. In these settings, detailed confounder data are often not available in all data sources…

Complete randomization allows for consistent estimation of the average treatment effect based on the difference in means of the outcomes without strong modeling assumptions on the outcome-generating process. Appropriate use of the…

Methodology · Statistics 2021-08-03 Anqi Zhao , Peng Ding

Machine learning (ML) models are increasingly being used in metrology applications. However, for ML models to be credible in a metrology context they should be accompanied by principled uncertainty quantification. This paper addresses the…

Machine Learning · Computer Science 2024-05-09 Andrew Thompson

The estimands framework outlined in ICH E9 (R1) describes the components needed to precisely define the effects to be estimated in clinical trials, which includes how post-baseline "intercurrent" events (IEs) are to be handled. In…

Methodology · Statistics 2023-08-28 Thomas Drury , Juan J Abellan , Nicky Best , Ian R. White

We propose a procedure for imputing missing values of time-dependent covariates in a survival model using fully conditional specification. Specifically, we focus on imputing missing values of a longitudinal marker in joint modeling of the…

Methodology · Statistics 2024-03-29 Havi Murad , Nirit Agay , Rachel Dankner