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Multiple imputation (MI) has become popular for analyses with missing data in medical research. The standard implementation of MI is based on the assumption of data being missing at random (MAR). However, for missing data generated by…

Methodology · Statistics 2019-01-03 Tra My Pham , James R Carpenter , Tim P Morris , Angela M Wood , Irene Petersen

The ICH E9(R1) Addendum (International Council for Harmonization 2019) suggests treatment-policy as one of several strategies for addressing intercurrent events such as treatment withdrawal when defining an estimand. This strategy requires…

Methodology · Statistics 2023-08-28 Suzie Cro , James H Roger , James R Carpenter

Multiple imputation (MI) is a method for repairing and analyzing data with missing values. MI replaces missing values with a sample of random values drawn from an imputation model. The most popular form of MI, which we call posterior draw…

Methodology · Statistics 2019-11-18 Paul T. von Hippel , Jonathan Bartlett

Missing confounders are common in observational studies and present fundamental challenges for causal effect estimation by weakening identification and increasing sensitivity to model misspecification. Within the missing-indicator…

Methodology · Statistics 2026-04-23 Md. Shaddam Hossain Bagmar , Hua Shen

Inverse probability of treatment weighting (IPW) has been well applied in causal inference to estimate population-level estimands from observational studies. For time-to-event outcomes, the failure time distribution can be estimated by…

Methodology · Statistics 2025-05-13 Yuhao Deng , Rui Wang

In precision medicine, one of the most important problems is estimating the optimal individualized treatment rules (ITR), which typically involves recommending treatment decisions based on fully observed individual characteristics of…

Methodology · Statistics 2025-10-15 Yue Zhang , Shanshan Luo , Zhi Geng , Yangbo He

This paper provides clear and practical guidance on the specification of imputation models when multiple imputation is used in conjunction with doubly robust estimation methods for causal inference. Through theoretical arguments and…

Methodology · Statistics 2025-12-19 Lucy D'Agostino McGowan

Missing data is a systemic problem in practical scenarios that causes noise and bias when estimating treatment effects. This makes treatment effect estimation from data with missingness a particularly tricky endeavour. A key reason for this…

Machine Learning · Statistics 2023-02-27 Jeroen Berrevoets , Fergus Imrie , Trent Kyono , James Jordon , Mihaela van der Schaar

In observational research treatment effects, the average treatment effect (ATE) estimator may be biased if a confounding variable is misclassified. We discuss the impact of classification error in a dichotomous confounding variable in…

Methodology · Statistics 2019-12-13 Linda Nab , Rolf H. H. Groenwold , Maarten van Smeden , Ruth H. Keogh

Contrasting marginal counterfactual survival curves across treatment arms is an effective and popular approach for inferring the causal effect of an intervention on a right-censored time-to-event outcome. A key challenge to drawing such…

Methodology · Statistics 2022-04-29 Andrew Ying , Yifan Cui , Eric J. Tchetgen Tchetgen

Missing data is a pervasive problem in epidemiology, with multiple imputation (MI) a commonly used analysis method. MI is valid when data are missing at random (MAR). However, definitions of MAR with multiple incomplete variables are not…

Methodology · Statistics 2025-04-14 Paul Madley-Dowd , Rachael A. Hughes , Maya B. Mathur , Jon Heron , Kate Tilling

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

Recurrent events are common and important clinical trial endpoints in many disease areas, e.g., cardiovascular hospitalizations in heart failure, relapses in multiple sclerosis, or exacerbations in asthma. During a trial, patients may…

Methodology · Statistics 2026-04-08 Jiren Sun , Tobias Mutze , Richard Cook , Tianmeng Lyu

Propensity score methods are an important tool to help reduce confounding in non-experimental studies. Most propensity score methods assume that covariates are measured without error. However, covariates are often measured with error, which…

Methodology · Statistics 2017-06-08 Hwanhee Hong , David A. Aaby , Juned Siddique , Elizabeth A. Stuart

Survival outcomes are common in comparative effectiveness studies and require unique handling because they are usually incompletely observed due to right-censoring. A ``once for all'' approach for causal inference with survival outcomes…

Methodology · Statistics 2021-12-21 Shuxi Zeng , Fan Li , Liangyuan Hu , Fan Li

Our work was motivated by a recent study on birth defects of infants born to pregnant women exposed to a certain medication for treating chronic diseases. Outcomes such as birth defects are rare events in the general population, which often…

Applications · Statistics 2017-02-24 Ronghui Xu , Jue Hou , Christina D. Chambers

Propensity scores are commonly used to reduce the confounding bias in non-randomized observational studies for estimating the average treatment effect. An important assumption underlying this approach is that all confounders that are…

Methodology · Statistics 2022-08-02 Youfei Yu , Jiacong Du , Min Zhang , Zhenke Wu , Andrew M. Ryan , Bhramar Mukherjee

Missing data is inevitable in longitudinal clinical trials. Conventionally, the missing at random assumption is assumed to handle missingness, which however is unverifiable empirically. Thus, sensitivity analysis is critically important to…

Methodology · Statistics 2022-03-18 Siyi Liu , Shu Yang , Yilong Zhang , Guanghan , Liu

In many observational studies in social science and medicine, subjects or units are connected, and one unit's treatment and attributes may affect another's treatment and outcome, violating the stable unit treatment value assumption (SUTVA)…

Methodology · Statistics 2024-06-25 Zhaonan Qu , Ruoxuan Xiong , Jizhou Liu , Guido Imbens

With multiple outcomes in empirical research, a common strategy is to define a composite outcome as a weighted average of the original outcomes. However, the choices of weights are often subjective and can be controversial. We propose an…

Methodology · Statistics 2025-09-17 Wei Zhang , Qizhai Li , Peng Ding
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