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The primary benefit of identifying a valid surrogate marker is the ability to use it in a future trial to test for a treatment effect with shorter follow-up time or less cost. However, previous work has demonstrated potential heterogeneity…

Methodology · Statistics 2022-09-20 Layla Parast , Tianxi Cai , Lu Tian

In vaccine studies, investigators are often interested in studying effect modifiers of clinical treatment efficacy by biomarker-based principal strata, which is useful for selecting biomarker study endpoints for evaluating treatments in new…

Methodology · Statistics 2017-10-30 Yingying Zhuang , Ying Huang , Peter B. Gilbert

Selection bias is a serious potential problem for inference about relationships of scientific interest based on samples without well-defined probability sampling mechanisms. Motivated by the potential for selection bias in (a) estimated…

In industry, online randomized controlled experiment (a.k.a. A/B experiment) is a standard approach to measure the impact of a causal change. These experiments have small treatment effect to reduce the potential blast radius. As a result,…

Econometrics · Economics 2025-05-29 Tanmoy Das , Dohyeon Lee , Arnab Sinha

Recently, Stochastic Variational Inference (SVI) has been increasingly attractive thanks to its ability to find good posterior approximations of probabilistic models. It optimizes the variational objective with stochastic optimization,…

Machine Learning · Computer Science 2022-03-16 Minta Liu , Suliang Bu

The growing availability of observational databases like electronic health records (EHR) provides unprecedented opportunities for secondary use of such data in biomedical research. However, these data can be error-prone and need to be…

Methodology · Statistics 2024-05-28 Sarah C. Lotspeich , Gustavo G. C. Amorim , Pamela A. Shaw , Ran Tao , Bryan E. Shepherd

Observational studies are frequently used to estimate the effect of an exposure or treatment on an outcome. To obtain an unbiased estimate of the treatment effect, it is crucial to measure the exposure accurately. A common type of exposure…

Methodology · Statistics 2024-07-02 Suhwan Bong , Kwonsang Lee , Francesca Dominici

Causal inference methods can be applied to estimate the effect of a point exposure or treatment on an outcome of interest using data from observational studies. For example, in the Women's Interagency HIV Study, it is of interest to…

In clinical studies, the risk of the primary (terminal) event may be modified by intermediate events, resulting in semicompeting risks. To study the treatment effect on the terminal event mediated by the intermediate event, researchers wish…

Methodology · Statistics 2026-05-26 Yuhao Deng , Rui Wang , Tao Zhang , Xiang Zhan

Covariate adjustment is an approach to improve the precision of trial analyses by adjusting for baseline variables that are prognostic of the primary endpoint. Motivated by the SEARCH Universal HIV Test-and-Treat Trial (2013-2017), we tell…

Methodology · Statistics 2025-12-16 Laura B. Balzer , Mark J. van der Laan , Maya L. Petersen

In observational studies, estimation of a causal effect of a treatment on an outcome relies on proper adjustment for confounding. If the number of the potential confounders ($p$) is larger than the number of observations ($n$), then direct…

Methodology · Statistics 2018-10-18 Joseph Antonelli , Giovanni Parmigiani , Francesca Dominici

In many real-world causal inference applications, the primary outcomes (labels) are often partially missing, especially if they are expensive or difficult to collect. If the missingness depends on covariates (i.e., missingness is not…

Machine Learning · Statistics 2024-05-24 Zhenghao Zeng , David Arbour , Avi Feller , Raghavendra Addanki , Ryan Rossi , Ritwik Sinha , Edward H. Kennedy

In adaptive clinical trials, the conventional end-of-trial point estimate of a treatment effect is prone to bias, that is, a systematic tendency to deviate from its true value. As stated in recent FDA guidance on adaptive designs, it is…

Traditional measures of vaccine efficacy (VE) are inherently asymmetric, constrained above by $1$ but unbounded below. As a result, VE estimates and corresponding confidence intervals can extend far below zero, making interpretation…

Methodology · Statistics 2025-12-09 Lucy D'Agostino McGowan , Sarah C. Lotspeich , Michael G. Hudgens

Leveraging external controls -- relevant individual patient data under control from external trials or real-world data -- has the potential to reduce the cost of randomized controlled trials (RCTs) while increasing the proportion of trial…

Methodology · Statistics 2022-07-13 Yanyao Yi , Ying Zhang , Yu Du , Ting Ye

In medical and epidemiological studies, one of the most common settings is studying the effect of a treatment on a time-to-event outcome, where the time-to-event might be censored before end of study. A common parameter of interest in such…

Methodology · Statistics 2024-02-15 Guilherme W. F. Barros , Jenny Häggström

We investigate how a population's natural and vaccine immunity affects the competitive balance between two strains of an infectious disease with different epidemiological characteristics. Specifically, we consider the case where one strain…

Dynamical Systems · Mathematics 2023-12-27 Matthew D. Johnston , Bruce Pell , Jared Pemberton , David A. Rubel

Mislabeled, duplicated, or biased data in real-world scenarios can lead to prolonged training and even hinder model convergence. Traditional solutions prioritizing easy or hard samples lack the flexibility to handle such a variety…

Machine Learning · Computer Science 2023-11-08 Zhijie Deng , Peng Cui , Jun Zhu

In this work, we propose an approach for assessing sensitivity to unobserved confounding in studies with multiple outcomes. We demonstrate how prior knowledge unique to the multi-outcome setting can be leveraged to strengthen causal…

Methodology · Statistics 2023-01-26 Jiajing Zheng , Jiaxi Wu , Alexander D'Amour , Alexander Franks

The positivity assumption is central in the identification of a causal effect, and especially the stochastic variant is an issue many applied researchers face, yet is rarely discussed, especially in conjunction with continuous treatments or…

Applications · Statistics 2026-02-03 Katharina Ring , Michael Schomaker