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The identification of surrogate markers is motivated by their potential to make decisions sooner about a treatment effect. However, few methods have been developed to actually use a surrogate marker to test for a treatment effect in a…

Methodology · Statistics 2024-09-17 Layla Parast , Jay Bartroff

Given the long follow-up periods that are often required for treatment or intervention studies, the potential to use surrogate markers to decrease the required follow-up time is a very attractive goal. However, previous studies have shown…

Methodology · Statistics 2016-08-12 Layla Parast , Tianxi Cai , Lu Tian

In modern clinical trials, there is immense pressure to use surrogate markers in place of an expensive or long-term primary outcome to make more timely decisions about treatment effectiveness. However, using a surrogate marker to test for a…

Methodology · Statistics 2025-04-22 Rebecca Knowlton , Layla Parast

Surrogate markers are most commonly studied within the context of randomized clinical trials. However, the need for alternative outcomes extends beyond these settings and may be more pronounced in real-world public health and social science…

Methodology · Statistics 2025-04-23 Rebecca Knowlton , Layla Parast

Surrogate markers are often used in clinical trials to evaluate treatment effects when primary outcomes are costly, invasive, or take a long time to observe. However, reliance on surrogates can lead to the surrogate paradox, where a…

Methodology · Statistics 2025-06-17 Emily Hsiao , Lu Tian , Layla Parast

Evaluating treatment effects is critical in clinical trials but sometimes involves lengthy, invasive, or costly follow-up procedures. In these cases, surrogate markers, which provide intermediate measures of the long-term treatment effect,…

Methodology · Statistics 2026-03-24 Sarah C. Lotspeich , P. D. Anh. Nguyen , Layla Parast

When evaluating the effectiveness of a treatment, policy, or intervention, the desired measure of effectiveness may be expensive to collect, not routinely available, or may take a long time to occur. In these cases, it is sometimes possible…

Methodology · Statistics 2022-11-10 Denis Agniel , Layla Parast , Boris Hejblum

Motivated by increasing pressure for decision makers to shorten the time required to evaluate the efficacy of a treatment such that treatments deemed safe and effective can be made publicly available, there has been substantial recent…

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

Recent work has focused on nonparametric estimation of conditional treatment effects, but inference has remained relatively unexplored. We propose a class of nonparametric tests for both quantitative and qualitative treatment effect…

Methodology · Statistics 2026-04-07 Oliver Dukes , Mats J. Stensrud , Riccardo Brioschi , Aaron Hudson

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

Estimating the long-term effects of treatments is of interest in many fields. A common challenge in estimating such treatment effects is that long-term outcomes are unobserved in the time frame needed to make policy decisions. One approach…

Methodology · Statistics 2024-08-23 Susan Athey , Raj Chetty , Guido Imbens , Hyunseung Kang

This paper provides asymptotically valid tests for the null hypothesis of no treatment effect heterogeneity. Importantly, I consider the presence of heterogeneity that is not explained by observed characteristics, or so-called idiosyncratic…

Econometrics · Economics 2023-04-04 Jaime Ramirez-Cuellar

Surrogate markers offer the potential to reduce the burden of data collection by replacing costly or invasive primary outcomes with more accessible measurements, provided that they can faithfully indicate the effectiveness of a treatment.…

Methodology · Statistics 2026-04-15 Silvaneo V. dos Santos , Layla Parast

Many studies include a goal of determining whether there is treatment effect heterogeneity across different subpopulations. In this paper, we propose a U-statistic-based non-parametric test of the null hypothesis that the treatment effects…

Methodology · Statistics 2020-12-08 Maozhu Dai , Hal S. Stern

We propose a framework for testing the homogeneity of conditional average treatment effects (CATEs) across multiple experimental and observational studies. Our approach leverages multiple randomized trials to assess whether treatment…

Econometrics · Economics 2026-02-25 Ana Armendariz , Martin Huber

In many experimental and observational studies, the outcome of interest is often difficult or expensive to observe, reducing effective sample sizes for estimating average treatment effects (ATEs) even when identifiable. We study how…

Machine Learning · Statistics 2024-10-11 Nathan Kallus , Xiaojie Mao

Mediation analysis is a useful tool to evaluate surrogate endpoints in clinical trials. We propose a novel method, the M-survival learner, for estimating heterogeneous indirect treatment effects in the presence of censored outcomes. The…

Methodology · Statistics 2026-04-16 Xingyu Li , Qing Liu , Xun Jiang , Hong Amy Xia , Brian P. Hobbs , Peng Wei

A surrogate marker is a biomarker or other physical measurement used to replace a primary outcome in clinical trials to evaluate a treatment effect when the primary outcome of interest is costly, invasive, or takes a long time to observe.…

Methodology · Statistics 2026-04-24 Emily Hsiao , Layla Parast

The method of surrogate data provides a framework for testing observed data against a hierarchy of alternative hypotheses. The aim of applying this method is to exclude the possibility that the data are consistent with simple linear…

Chaotic Dynamics · Physics 2007-05-23 Xiaodong Luo , Jie Zhang , Junfeng Sun , Michael Small , Irene Moroz

In oncology the efficacy of novel therapeutics often differs across patient subgroups, and these variations are difficult to predict during the initial phases of the drug development process. The relation between the power of randomized…

Methodology · Statistics 2025-06-05 Boyu Ren , Federico Ferrari , Sandra Fortini , Steffen Ventz , Lorenzo Trippa
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