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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

Randomized trials are considered the gold standard for estimating causal effects. Trial findings are often used to inform policy and programming efforts, yet their results may not generalize well to a relevant target population due to…

Concerns over reproducibility in science extend to research using existing healthcare data; many observational studies investigating the same topic produce conflicting results, even when using the same data. To address this problem, we…

Applications · Statistics 2018-03-30 Martijn J. Schuemie , Patrick B. Ryan , George Hripcsak , David Madigan , Marc A. Suchard

When analyzing data from randomized clinical trials, covariate adjustment can be used to account for chance imbalance in baseline covariates and to increase precision of the treatment effect estimate. A practical barrier to covariate…

Methodology · Statistics 2023-07-04 Chia-Rui Chang , Yue Song , Fan Li , Rui Wang

Randomized controlled trials (RCTs) with binary primary endpoints introduce novel challenges for inferring the causal effects of treatments. The most significant challenge is non-collapsibility, in which the conditional odds ratio estimand…

Methodology · Statistics 2024-03-01 Yunfan Li , Arman Sabbaghi , Jonathan R. Walsh , Charles K. Fisher

Many scientific questions in biomedical, environmental, and psychological research involve understanding the effects of multiple factors on outcomes. While factorial experiments are ideal for this purpose, randomized controlled treatment…

Methodology · Statistics 2025-12-03 Ruoqi Yu , Peng Ding

Combining experimental and observational follow-up datasets has received a lot of attention lately. In a time-to-event setting, recent work has used medicare claims to extend the follow-up period for participants in a prostate cancer…

Methodology · Statistics 2022-04-12 Gang Cheng , Yen-Chi Chen , Joseph M. Unger , Cathee Till , Ying-Qi Zhao

The era of big data has witnessed an increasing availability of observational data from mobile and social networking, online advertising, web mining, healthcare, education, public policy, marketing campaigns, and so on, which facilitates…

Machine Learning · Computer Science 2023-03-06 Zhixuan Chu , Ruopeng Li , Stephen Rathbun , Sheng Li

For randomized controlled trials (RCTs) with a single intervention being measured on multiple outcomes, researchers often apply a multiple testing procedure (such as Bonferroni or Benjamini-Hochberg) to adjust $p$-values. Such an adjustment…

Methodology · Statistics 2023-05-17 Kristen Hunter , Luke Miratrix , Kristin Porter

Causal inference in modern largescale systems faces growing challenges, including highdimensional covariates, multi-valued treatments, massive observational (OBS) data, and limited randomized controlled trial (RCT) samples due to cost…

Methodology · Statistics 2026-02-27 Yuxi Du , Zhiheng Zhang , Haoxuan Li , Cong Fang , Jixing Xu , Peng Zhen , Jiecheng Guo

This study introduces a data-driven, machine learning-based method to detect suitable control variables and instruments for assessing the causal effect of a treatment on an outcome in observational data. Our approach tests the joint…

Econometrics · Economics 2026-05-20 Nicolas Apfel , Julia Hatamyar , Martin Huber , Jannis Kueck

We study causal effect estimation from a mixture of observational and interventional data in a confounded linear regression model with multivariate treatments. We show that the statistical efficiency in terms of expected squared error can…

Clinical trials usually target average treatment effects, but treatment decisions are made for individuals. This tension motivates a common criticism of evidence-based medicine: a treatment that is beneficial on average may be inappropriate…

Applications · Statistics 2026-05-29 Zach Shahn , Mats Stensrud

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

The use of information from real world to assess the effectiveness of medical products is becoming increasingly popular and more acceptable by regulatory agencies. According to a strategic real-world evidence framework published by U.S.…

Methodology · Statistics 2022-03-22 Jianghao Li , Yu Du , Huayu Liu , Yanyao Yi

Unmeasured confounding presents a common challenge in observational studies, potentially making standard causal parameters unidentifiable without additional assumptions. Given the increasing availability of diverse data sources, exploiting…

Methodology · Statistics 2023-09-18 Shanshan Luo , Yechi Zhang , Wei Li

Methods that infer causal dependence from observational data are central to many areas of science, including medicine, economics, and the social sciences. A variety of theoretical properties of these methods have been proven, but empirical…

Methodology · Statistics 2021-07-08 Amanda Gentzel , Purva Pruthi , David Jensen

The theocratical properties of the power of the conventional testing hypotheses and the selection bias are usually unknown under covariate-adaptive randomized clinical trials. In the literature, most studies are based on simulations. In…

Statistics Theory · Mathematics 2021-05-04 Li-Xin Zhang

This project considers how one might augment a limited amount of data from randomized controlled trial (RCT) with more plentiful data from an observational database (ODB), in order to estimate a causal effect. In our motivating setting, the…

Methodology · Statistics 2018-10-23 Evan Rosenman , Art B. Owen , Michael Baiocchi , Hailey Banack

Randomized Controlled Trials (RCTs) represent the gold standard for causal inference yet remain a scarce resource. While large-scale observational data is often available, it is utilized only for retrospective fusion, and remains discarded…

Machine Learning · Statistics 2026-03-05 Erdun Gao , Liang Zhang , Jake Fawkes , Aoqi Zuo , Wenqin Liu , Haoxuan Li , Mingming Gong , Dino Sejdinovic