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Intercurrent events, such as treatment switching, rescue medication, dropout, or truncation by death, frequently complicate intention-to-treat analyses in randomized clinical trials. Existing causal inference frameworks typically target…

Methodology · Statistics 2026-03-12 Georgi Baklicharov , Kelly Van Lancker , Stijn Vansteelandt

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

Medication adherence is essential to ensure treatment effectiveness, but too often in routine care non-adherence compromises the desired outcome. We explore longitudinal causal modelling using observational data to estimate the time-varying…

Methodology · Statistics 2026-03-10 Xiaoran Liang , Deniz Türkmen , Jane A H Masoli , Luke C Pilling , Jack Bowden

Inverse classification uses an induced classifier as a queryable oracle to guide test instances towards a preferred posterior class label. The result produced from the process is a set of instance-specific feature perturbations, or…

Machine Learning · Computer Science 2018-02-15 Michael T. Lash , Qihang Lin , W. Nick Street

Digital monitoring studies collect real-time high frequency data via mobile sensors in the subjects' natural environment. This data can be used to model the impact of changes in physiology on recurrent event outcomes such as smoking, drug…

Methodology · Statistics 2022-04-15 Walter Dempsey

Estimating the individual treatment effect (ITE) from observational data is meaningful and practical in healthcare. Existing work mainly relies on the strong ignorability assumption that no hidden confounders exist, which may lead to bias…

Methodology · Statistics 2020-12-16 Ruoqi Liu , Changchang Yin , Ping Zhang

The estimand framework provides guidance on handling intercurrent events, such as treatment discontinuation, in the analysis of clinical trial responses. Under ICH E9(R1), the treatment policy (TP) strategy incorporates post-discontinuation…

Methodology · Statistics 2026-04-07 Myeongjong Kang , Sangyoon Yi

Composite endpoints that combine recurrent non-fatal events with a terminal event are increasingly used in randomized clinical trials, yet conventional time-to-first event analyses may obscure clinically relevant information. We compared…

Methodology · Statistics 2025-12-16 Adrien Orué , Derek Dinart , Laurent Billot , Carine Bellera , Virginie Rondeau

Interference occurs when the treatment (or exposure) of one individual affects the outcomes of others. In some settings it may be reasonable to assume individuals can be partitioned into clusters such that there is no interference between…

Methodology · Statistics 2018-06-21 Lan Liu , Michael G. Hudgens , Bradley Saul , John D. Clemens , Mohammad Ali , Michael E. Emch

In the analysis of observational studies, inverse probability weighting (IPW) is commonly used to consistently estimate the average treatment effect (ATE) or the average treatment effect in the treated (ATT). The variance of the IPW ATE…

Methodology · Statistics 2020-11-25 Sarah A. Reifeis , Michael G. Hudgens

There is growing interest in developing causal inference methods for multi-valued treatments with a focus on pairwise average treatment effects. Here we focus on a clinically important, yet less-studied estimand: causal drug-drug…

Methodology · Statistics 2022-09-20 Di Shu , Peisong Han , Sean Hennessy , Todd A Miano

Studies intended to estimate the effect of a treatment, like randomized trials, may not be sampled from the desired target population. To correct for this discrepancy, estimates can be transported to the target population. Methods for…

Weighting procedures are used in observational causal inference to adjust for covariate imbalance within the sample. Common practice for inference is to estimate robust standard errors from a weighted regression of outcome on treatment.…

Methodology · Statistics 2025-07-29 Erin Hartman , Chad Hazlett , Arisa Sadeghpour

Treatment-induced confounders complicate analyses of time-varying treatment effects and causal mediation. Conditioning on these variables naively to estimate marginal effects may inappropriately block causal pathways and may induce spurious…

Applications · Statistics 2018-08-24 Geoffrey T. Wodtke , Zahide Alaca , Xiang Zhou

We provide guidance on multiple imputation of missing at random treatments in observational studies. Specifically, analysts should account for both covariates and outcomes, i.e., not just use propensity scores, when imputing the missing…

Methodology · Statistics 2025-01-23 Joseph Feldman , Jerome P. Reiter

While recurrent event analyses have been extensively studied, limited attention has been given to causal inference within the framework of recurrent event analysis. We develop a multiply robust estimation framework for causal inference in…

Methodology · Statistics 2025-09-30 Benjamin R. Baer , Trang Bui , Daniel Mork , Robert L. Strawderman , Ashkan Ertefaie

Imputing missing potential outcomes using an estimated regression function is a natural idea for estimating causal effects. In the literature, estimators that combine imputation and regression adjustments are believed to be comparable to…

Statistics Theory · Mathematics 2023-01-20 Zhexiao Lin , Fang Han

There are now many options for doubly robust estimation; however, there is a concerning trend in the applied literature to believe that the combination of a propensity score and an adjusted outcome model automatically results in a doubly…

In clinical trials, multiple outcomes of different priorities commonly occur as the patient's response may not be adequately characterized by a single outcome. Win statistics are appealing summary measures for between-group difference at…

Methodology · Statistics 2025-06-04 Ying Cui , Bo Huang , Gaohong Dong , Ryuji Uozumi , Lu Tian

When estimating causal effects from observational data with numerous covariates, employing penalized covariate selection can improve the estimation efficiency. Outcome-oriented covariate selection, which involves selecting covariates…

Methodology · Statistics 2025-01-14 Wataru Hongo , Shuji Ando , Jun Tsuchida , Takashi Sozu