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We study identifying and estimating the causal effect of a treatment variable on a long-term outcome using data from an observational and an experimental domain. The observational data are subject to unobserved confounding. Furthermore,…

We introduce a statistical procedure that integrates survival data from multiple biomedical studies, to improve the accuracy of predictions of survival or other events, based on individual clinical and genomic profiles, compared to models…

Applications · Statistics 2020-07-20 Steffen Ventz , Rahul Mazumder , Lorenzo Trippa

Observational studies can play a useful role in assessing the comparative effectiveness of competing treatments. In a clinical trial the randomization of participants to treatment and control groups generally results in well-balanced groups…

A key challenge in causal inference from observational studies is the identification and estimation of causal effects in the presence of unmeasured confounding. In this paper, we introduce a novel approach for causal inference that…

Methodology · Statistics 2022-10-17 Ying Zhou , Dingke Tang , Dehan Kong , Linbo Wang

Data fusion techniques integrate information from heterogeneous data sources to improve learning, generalization, and decision making across data sciences. In causal inference, these methods leverage rich observational data to improve…

Methodology · Statistics 2025-06-02 Quinn Lanners , Cynthia Rudin , Alexander Volfovsky , Harsh Parikh

How should researchers analyze randomized experiments in which the main outcome is latent and measured in multiple ways but each measure contains some degree of error? We first identify a critical study-specific noncomparability problem in…

Econometrics · Economics 2026-01-13 Jiawei Fu , Donald P. Green

Most causal inference methods focus on estimating marginal average treatment effects, but many important causal estimands depend on the joint distribution of potential outcomes, including the probability of causation and proportions…

Methodology · Statistics 2025-10-16 Zach Shahn , David Madigan

Under what circumstances is it a threat to the parallel trends assumption required for Difference in Differences (DiD) studies if treatment decisions are based on past values of the outcome? We explore via simulation studies whether…

Methodology · Statistics 2022-08-02 Zach Shahn

We provide a novel characterization of semiparametric efficiency in a generic supervised learning setting where the outcome mean function -- defined as the conditional expectation of the outcome of interest given the other observed…

Methodology · Statistics 2025-04-22 Harrison H. Li

Data from both a randomized trial and an observational study are sometimes simultaneously available for evaluating the effect of an intervention. The randomized data typically allows for reliable estimation of average treatment effects but…

Methodology · Statistics 2021-12-01 David Cheng , Tianxi Cai

Opioids are an effective analgesic for acute and chronic pain, but also carry a considerable risk of addiction leading to millions of opioid use disorder (OUD) cases and tens of thousands of premature deaths in the United States yearly.…

Quantitative Methods · Quantitative Biology 2024-03-27 Sybille Légitime , Kaustubh Prabhu , Devin McConnell , Bing Wang , Dipak K. Dey , Derek Aguiar

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

Real-World Data (RWD), with its large sample sizes and rich clinical detail, offers a compelling alternative to randomized controlled trials (RCTs) for studying treatment effects in diverse and complex patient populations. However, its…

Applications · Statistics 2026-05-26 Yifei Xu , Hwiyoung Lee , Zhenyao Ye , Yezhi Pan , Jingsong Zhou , Yun Yang , Chixiang Chen , Shuo Chen

Integrated IPD-AD analysis, which combines individual participant data (IPD) with aggregate data (AD), is increasingly recognized as an effective strategy for generating more reliable and generalizable inferences from heterogeneous studies.…

Methodology · Statistics 2026-03-03 Ming-Yueh Huang , Jing Qin , Chiung-Yu Huang

Motivated by two case studies using primary care records from the Clinical Practice Research Datalink, we describe statistical methods that facilitate the analysis of tall data, with very large numbers of observations. Our focus is on…

Methodology · Statistics 2018-05-14 Kirsty Rhodes , Rebecca Turner , Rupert Payne , Ian White

Semiparametric inference on average causal effects from observational data is based on assumptions yielding identification of the effects. In practice, several distinct identifying assumptions may be plausible; an analyst has to make a…

Methodology · Statistics 2025-10-07 Tetiana Gorbach , Xavier de Luna , Juha Karvanen , Ingeborg Waernbaum

Suppose we have individual data from an internal study and various summary statistics from relevant external studies. External summary statistics have the potential to improve statistical inference for the internal population; however, it…

Methodology · Statistics 2026-02-06 Wenjie Hu , Ruoyu Wang , Wei Li , Wang Miao

Causal estimands can vary significantly depending on the relationship between outcomes in treatment and control groups, potentially leading to wide partial identification (PI) intervals that impede decision making. Incorporating covariates…

Methodology · Statistics 2025-06-02 Sirui Lin , Zijun Gao , Jose Blanchet , Peter Glynn

Probabilities of Causation (PoC) play a fundamental role in decision-making in law, health care and public policy. Nevertheless, their point identification is challenging, requiring strong assumptions, in the absence of which only bounds…

Artificial Intelligence · Computer Science 2023-10-13 Numair Sani , Atalanti A. Mastakouri

Augmenting the control arm in clinical trials with external data can improve statistical power for demonstrating treatment effects. In many time-to-event outcome trials, participants are subject to truncation by death. Direct application of…

Methodology · Statistics 2025-06-24 Zehao Su , Helene C. W. Rytgaard , Henrik Ravn , Frank Eriksson
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