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Collecting multiple longitudinal measurements and time-to-event outcomes is a common practice in clinical and epidemiological studies, often focusing on exploring associations between them. Joint modeling is the standard analytical tool for…

Methodology · Statistics 2024-12-10 Taban Baghfalaki , Reza Hashemi , Catherine Helmer , Helene Jacqmin-Gadda

Dropout poses a significant challenge to causal inference in longitudinal studies with time-varying treatments. However, existing research does not simultaneously address dropout and time-varying treatments. We examine selective…

Methodology · Statistics 2025-03-18 Zhichao Jiang , Eli Ben-Michael , D. James Greiner , Ryan Halen , Kosuke Imai

In observational studies, covariates with substantial missing data are often omitted, despite their strong predictive capabilities. These excluded covariates are generally believed not to simultaneously affect both treatment and outcome,…

Methodology · Statistics 2024-02-23 Shanshan Luo , Mengchen Shi , Wei Li , Xueli Wang , Zhi Geng

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

Learning about causal effects in target populations and their subsets may be facilitated by combining information from multiple sources. One major class of study designs that combine information involves appending an index study with data…

Matching estimators for average treatment effects are widely used in the binary treatment setting, in which missing potential outcomes are imputed as the average of observed outcomes of all matches for each unit. With more than two…

Methodology · Statistics 2019-04-29 Anthony D. Scotina , Francesca L. Beaudoin , Roee Gutman

In this paper, we introduce a unified estimator to analyze various treatment effects in causal inference, including but not limited to the average treatment effect (ATE) and the quantile treatment effect (QTE). The proposed estimator is…

Methodology · Statistics 2025-03-31 Kuan-Hsun Wu , Li-Pang Chen

We consider the conditional treatment effect for competing risks data in observational studies. While it is described as a constant difference between the hazard functions given the covariates, we do not assume specific functional forms for…

Applications · Statistics 2021-12-28 Denise Rava , Ronghui Xu

In randomized trials, repeated measures of the outcome are routinely collected. The mixed model for repeated measures (MMRM) leverages the information from these repeated outcome measures, and is often used for the primary analysis to…

Methodology · Statistics 2023-07-20 Bingkai Wang , Yu Du

In various data situations joint models are an efficient tool to analyze relationships between time dependent covariates and event times or to correct for event-dependent dropout occurring in regression analysis. Joint modeling connects a…

Methodology · Statistics 2018-10-25 Colin Griesbach , Andreas Mayr , Elisabeth Waldmann

Intensive longitudinal data, characterized by frequent measurements across numerous time points, are increasingly common due to advances in wearable devices and mobile health technologies. We consider evaluating causal mediation pathways…

Methodology · Statistics 2025-06-26 Tianchen Qian

Causal inference from observational data requires assumptions. These assumptions range from measuring confounders to identifying instruments. Traditionally, causal inference assumptions have focused on estimation of effects for a single…

Machine Learning · Statistics 2019-03-04 Rajesh Ranganath , Adler Perotte

Mixed Models for Repeated Measures (MMRMs) are ubiquitous when analyzing outcomes of clinical trials. However, the linearity of the fixed-effect structure in these models largely restrict their use to estimating treatment effects that are…

Methodology · Statistics 2023-01-23 Lars Lau Raket

The primary analysis of clinical trials in diabetes therapeutic area often involves a mixed-model repeated measure (MMRM) approach to estimate the average treatment effect for longitudinal continuous outcome, and a generalized linear mixed…

Applications · Statistics 2022-08-03 Lingjing Jiang , Michael Rosenblum , Yu Du

Increasing evidence suggests that variability in longitudinal biomarkers, in addition to their mean trajectory, carries prognostic information for time-to-event outcomes. However, standard joint models typically capture only the expected…

Methodology · Statistics 2026-05-08 Felix Boakye Oppong , Dimitris Rizopoulos , Thierry Gorlia , Nicole Erler

Joint modelling of longitudinal observations and event times continues to remain a topic of considerable interest in biomedical research. For example, in HIV studies, the longitudinal bio-marker such as CD4 cell count in a patient's blood…

Methodology · Statistics 2024-07-19 Srimanti Dutta , Arindom Chakraborty , Dipankar Bandyopadhyay

In many important applications of precision medicine, the outcome of interest is time to an event (e.g., death, relapse of disease) and the primary goal is to identify the optimal individualized decision rule (IDR) to prolong survival time.…

Methodology · Statistics 2022-04-11 Yu Zhou , Lan Wang , Rui Song , Tuoyi Zhao

When treatment effects are naturally expressed as ratios -- as in medicine, pricing, and marketing -- the ratio-based CATE $\tau(x) = E[Y|W=1,X=x] / E[Y|W=0,X=x]$ is the appropriate estimand. Yet existing estimators either impose a…

Machine Learning · Statistics 2026-05-27 Michael Fuchs , Dominik Kreiss

In clinical trials, the observation of participant outcomes may frequently be hindered by death, leading to ambiguity in defining a scientifically meaningful final outcome for those who die. Principal stratification methods are valuable…

Methodology · Statistics 2025-09-01 Jiaqi Tong , Chao Cheng , Guangyu Tong , Michael O. Harhay , Fan Li

The generalized g-formula can be used to estimate the probability of survival under a sustained treatment strategy. When treatment strategies are deterministic, estimators derived from the so-called efficient influence function (EIF) for…

Methodology · Statistics 2022-02-10 Lan Wen , Julia Marcus , Jessica Young