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While the inverse probability of treatment weighting (IPTW) is a commonly used approach for treatment comparisons in observational data, the resulting estimates may be subject to bias and excessively large variance when there is lack of…

Methodology · Statistics 2024-02-13 Zhiqiang Cao , Lama Ghazi , Claudia Mastrogiacomo , Laura Forastiere , F. Perry Wilson , Fan Li

In causal inference, estimating the average treatment effect is a central objective, and in the context of competing risks data, this effect can be quantified by the cause-specific cumulative incidence function (CIF) difference. While…

Methodology · Statistics 2026-03-27 Yifei Tian , Ying Wu

Estimands using the treatment policy strategy for addressing intercurrent events are common in Phase III clinical trials. One estimation approach for this strategy is retrieved dropout whereby observed data following an intercurrent event…

Precision medicine involves developing individualized treatment regimes (ITRs) which allow for treatment decisions to be tailored to patient characteristics. Naturally, the identification of the optimal regime, that is, the rule which…

Methodology · Statistics 2025-09-30 Misha Dolmatov , Erica E. M. Moodie , David A. Stephens , Dipankar Bandyopadhyay

When drawing causal inferences about the effects of multiple treatments on clustered survival outcomes using observational data, we need to address implications of the multilevel data structure, multiple treatments, censoring and unmeasured…

Methodology · Statistics 2022-02-18 Liangyuan Hu , Jiayi Ji , Ronald D. Ennis , Joseph W. Hogan

Interval censoring occurs when event times are only known to fall between scheduled assessments, a common design in clinical trials, epidemiology, and reliability studies. Standard right-censoring methods, such as Kaplan-Meier and Cox…

Methodology · Statistics 2025-09-04 J. T. Korley

In this paper, we expand the methodology presented in Mertens et. al (2020, Biometrical Journal) to the study of life-time (survival) outcome which is subject to censoring and when imputation is used to account for missing values. We…

Methodology · Statistics 2021-05-06 Bart J. A. Mertens

Optimizing survival outcomes, such as patient survival or customer retention, is a critical objective in data-driven decision-making. Off-Policy Evaluation~(OPE) provides a powerful framework for assessing such decision-making policies…

Methodology · Statistics 2026-03-25 Kohsuke Kubota , Mitsuhiro Takahashi , Yuta Saito

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

Unmeasured confounding is one of the major concerns in causal inference from observational data. Proximal causal inference (PCI) is an emerging methodological framework to detect and potentially account for confounding bias by carefully…

Methodology · Statistics 2025-04-24 Kendrick Li , George C. Linderman , Xu Shi , Eric J. Tchetgen Tchetgen

Estimating the causal effect of a treatment or health policy with observational data can be challenging due to an imbalance of and a lack of overlap between treated and control covariate distributions. In the presence of limited overlap,…

Methodology · Statistics 2025-03-24 Martha Barnard , Jared D. Huling , Julian Wolfson

We propose a restricted win probability estimand for comparing treatments in a randomized trial with a time-to-event outcome. We also propose Bayesian estimators for this summary measure as well as the unrestricted win probability. Bayesian…

Methodology · Statistics 2024-11-06 Michelle Leeberg , Xianghua Luo , Thomas A. Murray

With the advancement in drug development, multiple treatments are available for a single disease. Patients can often benefit from taking multiple treatments simultaneously. For example, patients in Clinical Practice Research Datalink (CPRD)…

Applications · Statistics 2018-04-17 Muxuan Liang , Ye Ting , Haoda Fu

Longitudinal studies could be complicated by left-censored repeated measures. For example, in Human Immunodeficiency Virus infection, there is a detection limit of the assay used to quantify the plasma viral load. Simple imputation of the…

Applications · Statistics 2007-05-23 Rodolphe Thiébaut , Hélène Jacqmin-Gadda

Dynamic Treatment Regimes (DTRs) provide a systematic framework for optimizing sequential decision-making in chronic disease management, where therapies must adapt to patients' evolving clinical profiles. Inverse probability weighting (IPW)…

Methodology · Statistics 2026-03-26 Chloe Si , David A. Stephens , Erica E. M. Moodie

We propose an empirically stable and asymptotically efficient covariate-balancing approach to the problem of estimating survival causal effects in data with conditionally-independent censoring. This addresses a challenge often encountered…

While right-censored time-to-event outcomes have been studied for decades, handling time-to-event covariates, also known as right-censored covariates, is now of growing interest. So far, the literature has treated right-censored covariates…

Methodology · Statistics 2024-09-10 Jesus E. Vazquez , Marissa C. Ashner , Yanyuan Ma , Karen Marder , Tanya P. Garcia

Adaptive experimentation enables efficient estimation of causal effects, but existing methods are not designed for survival data with censoring, where event times are only partially observed (e.g., overall survival in cancer trials but with…

Machine Learning · Computer Science 2026-05-19 Yuxin Wang , Dennis Frauen , Jonas Schweisthal , Maresa Schröder , Emil Javurek , Stefan Feuerriegel

Introduction In analysis of time-to-event outcomes, a mixture cure (MC) model is preferred over a standard survival model when the sample includes individuals who will never experience the event of interest. Motivated by a cohort study of…

Applications · Statistics 2026-04-02 Changchang Xu , Laurent Briollais , Irene L Andrulis , Shelley B Bull

Clinical decision support using data mining techniques offers more intelligent way to reduce the decision error in the last few years. However, clinical datasets often suffer from high missingness, which adversely impacts the quality of…

Machine Learning · Computer Science 2020-11-20 Xuetong Wu , Hadi Akbarzadeh Khorshidi , Uwe Aickelin , Zobaida Edib , Michelle Peate