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Estimating causal effects from observational data is inherently challenging due to the lack of observable counterfactual outcomes and even the presence of unmeasured confounding. Traditional methods often rely on restrictive, untestable…

Methodology · Statistics 2025-04-07 Li Chen , Xiaotong Shen , Wei Pan

Estimating average causal effect (ACE) is useful whenever we want to know the effect of an intervention on a given outcome. In the absence of a randomized experiment, many methods such as stratification and inverse propensity weighting have…

Machine Learning · Computer Science 2019-07-11 Rathin Desai , Amit Sharma

The primary endpoint in oncology is usually overall survival, where differences between therapies may only be observable after many years. To avoid withholding of a promising therapy, preliminary approval based on a surrogate endpoint is…

Methodology · Statistics 2022-05-25 Samuel Kilian , Johannes Krisam , Meinhard Kieser

Many benchmarks for automated causal inference evaluate a system's performance based on a single numerical output, such as an Average Treatment Effect (ATE). This approach conflates two distinct steps in causal analysis: identification -…

Artificial Intelligence · Computer Science 2026-05-15 Ayush Sawarni , Jiyuan Tan , Vasilis Syrgkanis

Matched case-control studies are commonly employed in epidemiological research for their convenience and efficiency. Analysis of secondary outcomes can yield valuable insights into biological pathways and help identify genetic variants of…

Methodology · Statistics 2026-02-24 Shanshan Liu , Guoqing Diao

In this paper, we introduce a new causal methodology that accounts for the rarity and frequency of events in observational studies based on their relevance to the underlying problem. Specifically, we propose a direct causal effect metric…

Artificial Intelligence · Computer Science 2025-02-28 Usef Faghihi , Amir Saki

This dissertation focuses on modern causal inference under uncertainty and data restrictions, with applications to neoadjuvant clinical trials, distributed data networks, and robust individualized decision making. In the first project, we…

Methodology · Statistics 2023-01-24 Xiaoqing Tan

Post-randomization events, also known as intercurrent events, such as treatment noncompliance and censoring due to a terminal event, are common in clinical trials. Principal stratification is a framework for causal inference in the presence…

Methodology · Statistics 2023-01-19 Bo Liu , Lisa Wruck , Fan Li

A common practice in clinical trials is to evaluate a treatment effect on an intermediate endpoint when the true outcome of interest would be difficult or costly to measure. We consider how to validate intermediate endpoints in a…

Methodology · Statistics 2022-11-30 Emily K. Roberts , Michael R. Elliott , Jeremy M. G. Taylor

This work was motivated by observational studies in pregnancy with spontaneous abortion (SAB) as outcome. Clearly some women experience the SAB event but the rest do not. In addition, the data are left truncated due to the way pregnant…

Methodology · Statistics 2017-08-24 Yuan Wu , Christina D. Chambers , Ronghui Xu

In neoadjuvant trials on early-stage breast cancer, patients are usually randomized into a control group and a treatment group with an additional target therapy. Early efficacy of the new regimen is assessed via the binary pathological…

Methodology · Statistics 2022-04-04 Xiaoqing Tan , Judah Abberbock , Priya Rastogi , Gong Tang

Selection bias is pervasive in observational studies. For example, large scale biobanks data can exhibit ``healthy volunteer bias'' when respondents are healthier and of higher socio-economic status than the population they are meant to…

Methodology · Statistics 2026-05-14 Yiwen Qiu , Filip Kovacevic , Shimeng Huang , Peter Spirtes , Francesco Locatello

The average treatment effect (ATE) is a common parameter estimated in causal inference literature, but it is only defined for binary exposures. Thus, despite concerns raised by some researchers, many studies seeking to estimate the causal…

Methodology · Statistics 2026-02-06 Kaitlyn J. Lee , Alan Hubbard , Alejandro Schuler

Quasi-experimental causal inference methods have become central in empirical operations management for guiding managerial decisions. Among these, empiricists utilize the Difference-in-Differences (DiD) estimator, which relies on the…

Methodology · Statistics 2026-05-13 Mingxuan Ge , Dae Woong Ham

Motivated by a potential-outcomes perspective, the idea of principal stratification has been widely recognized for its relevance in settings susceptible to posttreatment selection bias such as randomized clinical trials where treatment…

Applications · Statistics 2011-11-08 Corwin M. Zigler , Thomas R. Belin

Understanding how the causal effect of a treatment evolves over time, including the potential for waning, is important for informed decisions on treatment discontinuation or repetition. For example, waning vaccine protection influences…

Methodology · Statistics 2025-11-25 Eni Musta , Joris Mooij

In this paper we study the problems of estimating heterogeneity in causal effects in experimental or observational studies and conducting inference about the magnitude of the differences in treatment effects across subsets of the…

Machine Learning · Statistics 2022-06-08 Susan Athey , Guido Imbens

Understanding causal mechanisms is crucial for explaining and generalizing empirical phenomena. Causal mediation analysis offers statistical techniques to quantify the mediation effects. Although numerous methods have been developed for…

Methodology · Statistics 2026-05-12 Jiawei Fu

When dealing with right-censored data, where some outcomes are missing due to a limited observation period, survival analysis -- known as time-to-event analysis -- focuses on predicting the time until an event of interest occurs. Multiple…

Machine Learning · Statistics 2024-10-23 Julie Alberge , Vincent Maladière , Olivier Grisel , Judith Abécassis , Gaël Varoquaux

Platform trials allow treatment arms to enter and exit over time while maintaining a shared control arm, yielding concurrent and non-concurrent controls (NCC). Pooling NCC is often motivated as a strategy to improve statistical efficiency,…

Methodology · Statistics 2026-03-12 Antonio D'Alessandro , Samrachana Adhikari , Michele Santacatterina
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