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Clinical trials usually involve sequential patient entry. When designing a clinical trial, it is often desirable to include a provision for interim analyses of accumulating data with the potential for stopping the trial early. We review…

Methodology · Statistics 2023-03-13 Tianjian Zhou , Yuan Ji

The treatment effect in a specific subgroup is often of interest in randomized clinical trials. When the subgroup is characterized by the absence of certain post-randomization events, a naive analysis on the subset of patients without these…

Applications · Statistics 2018-09-12 Baldur P. Magnusson , Heinz Schmidli , Nicolas Rouyrre , Daniel O. Scharfstein

This paper studies inference on treatment effects in panel data settings with unobserved confounding. We model outcome variables through a factor model with random factors and loadings. Such factors and loadings may act as unobserved…

Econometrics · Economics 2023-12-05 Guido W. Imbens , Davide Viviano

Differences-in-differences (DiD) is a causal inference method for observational longitudinal data that assumes parallel expected potential outcome trajectories between treatment groups under the counterfactual scenario where all units…

Methodology · Statistics 2026-05-12 Michael Jetsupphasuk , Didong Li , Michael G. Hudgens

Estimating treatment effects is crucial for personalized decision-making in medicine, but this task faces unique challenges in clinical practice. At training time, models for estimating treatment effects are typically trained on…

Machine Learning · Computer Science 2025-10-31 Yuchen Ma , Dennis Frauen , Jonas Schweisthal , Stefan Feuerriegel

Routinely collected data from electronic health records (EHR) provide opportunities to study effects of longitudinal treatment strategies in real-world clinical settings. A challenge presented by EHR data is that frequency of covariate…

Applications · Statistics 2026-04-14 Leah Pirondini , Karla Diaz-Ordaz , Edward Palmer , Ruth H. Keogh

Principal stratification (PS) is a commonly used approach for understanding the mechanisms through which a treatment affects an outcome. The goal of this work is to extend the PS framework to studies with continuous treatments, which…

Methodology · Statistics 2025-05-20 Joseph Antonelli , Minxuan Wu , Fabrizia Mealli , Brenden Beck , Alessandra Mattei

Taking a rigorous formal approach, we consider sequential decision problems involving observable variables, unobservable variables, and action variables. We can typically assume the property of extended stability, which allows…

Statistics Theory · Mathematics 2020-04-28 A. Philip Dawid , Panayiota Constantinou

In many fields$\unicode{x2013}$including genomics, epidemiology, natural language processing, social and behavioral sciences, and economics$\unicode{x2013}$it is increasingly important to address causal questions in the context of factor…

Methodology · Statistics 2025-06-30 Jenna M. Landy , Dafne Zorzetto , Roberta De Vito , Giovanni Parmigiani

Medical time series datasets feature missing values that need data imputation methods, however, conventional machine learning models fall short due to a lack of uncertainty quantification in predictions. Among these models, the CATSI…

Machine Learning · Computer Science 2024-10-07 Omkar Kulkarni , Rohitash Chandra

Evaluating treatment effect heterogeneity widely informs treatment decision making. At the moment, much emphasis is placed on the estimation of the conditional average treatment effect via flexible machine learning algorithms. While these…

Methodology · Statistics 2021-05-07 Lihua Lei , Emmanuel J. Candès

Given two sets of elements (such as cell types and drug compounds), researchers typically only have access to a limited subset of their interactions. The task of causal imputation involves using this subset to predict unobserved…

Machine Learning · Computer Science 2024-10-29 Marco Jiralerspong , Thomas Jiralerspong , Vedant Shah , Dhanya Sridhar , Gauthier Gidel

How should one assess the credibility of assumptions weaker than statistical independence, like quantile independence? In the context of identifying causal effects of a treatment variable, we argue that such deviations should be chosen…

Econometrics · Economics 2018-05-01 Matthew A. Masten , Alexandre Poirier

In clinical trials, patients may discontinue treatments prematurely, breaking the initial randomization and, thus, challenging inference. Stakeholders in drug development are generally interested in going beyond the Intention-To-Treat (ITT)…

Longitudinal passive sensing studies for health and behavior outcomes often have missing and incomplete data. Handling missing data effectively is thus a critical data processing and modeling step. Our formative interviews with researchers…

Unmeasured confounding presents a significant challenge in causal inference from observational studies. Classical approaches often rely on collecting proxy variables, such as instrumental variables. However, in applications where the…

Methodology · Statistics 2025-01-16 Xiaochuan Shi , Dehan Kong , Linbo Wang

In clinical trials of longitudinal continuous outcomes, reference based imputation (RBI) has commonly been applied to handle missing outcome data in settings where the estimand incorporates the effects of intercurrent events, e.g. treatment…

Methodology · Statistics 2023-10-18 Sean Yiu

We study causal inference in sample selection models where a continuous or multivalued treatment affects both outcome and their observability (eg., employment or survey response). We generalized the widely used Lee (2009)'s bounds for…

Econometrics · Economics 2025-10-29 Ying-Ying Lee , Chu-An Liu

The study of causal effects in the presence of unmeasured spatially varying confounders has garnered increasing attention. However, a general framework for identifiability, which is critical for reliable causal inference from observational…

Methodology · Statistics 2026-02-27 Tommy Tang , Xinran Li , Bo Li

Estimating long-term treatment effects has a wide range of applications in various domains. A key feature in this context is that collecting long-term outcomes typically involves a multi-stage process and is subject to monotone missing,…

Machine Learning · Computer Science 2025-04-29 Qinwei Yang , Ruocheng Guo , Shasha Han , Peng Wu
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