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When the primary outcome is difficult to collect, surrogate endpoint is typically used as a substitute. It is possible that for every individual, treatment has a positive effect on surrogate, and surrogate has a positive effect on primary…

统计方法学 · 统计学 2017-12-27 Linquan Ma , Yunjian Yin , Lan Liu , Zhi Geng

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…

统计方法学 · 统计学 2025-06-17 Emily Hsiao , Lu Tian , Layla Parast

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…

机器学习 · 统计学 2024-10-11 Nathan Kallus , Xiaojie Mao

Inferring causal effects on long-term outcomes using short-term surrogates is crucial to rapid innovation. However, even when treatments are randomized and surrogates fully mediate their effect on outcomes, it's possible that we get the…

统计方法学 · 统计学 2023-11-09 Aurélien Bibaut , Nathan Kallus , Simon Ejdemyr , Michael Zhao

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…

统计方法学 · 统计学 2024-08-23 Susan Athey , Raj Chetty , Guido Imbens , Hyunseung Kang

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.…

统计方法学 · 统计学 2026-04-24 Emily Hsiao , Layla Parast

A surrogate endpoint S in a clinical trial is an outcome that may be measured earlier or more easily than the true outcome of interest T. In this work, we extend causal inference approaches to validate such a surrogate using potential…

统计方法学 · 统计学 2022-02-04 Emily Roberts , Michael Elliott , Jeremy M. G. Taylor

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…

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…

统计方法学 · 统计学 2016-08-12 Layla Parast , Tianxi Cai , Lu Tian

Surrogate endpoints play an important role in drug development when they can be used to measure treatment effect early compared to the final clinical outcome and to predict clinical benefit or harm. Such endpoints are assessed for their…

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…

统计方法学 · 统计学 2022-09-20 Layla Parast , Tianxi Cai , Lu Tian

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…

混沌动力学 · 物理学 2007-05-23 Xiaodong Luo , Jie Zhang , Junfeng Sun , Michael Small , Irene Moroz

Clinical trials or studies oftentimes require long-term and/or costly follow-up of participants to evaluate a novel treatment/drug/vaccine. There has been increasing interest in the past few decades in using short-term surrogate outcomes as…

统计方法学 · 统计学 2024-12-19 Xuan Wang , Tianxi Cai , Lu Tian , Layla Parast

When direct measurement of a clinically relevant primary endpoint in a clinical trial is infeasible, a surrogate endpoint may be used instead to infer treatment effects. Trial-level surrogates predict the average treatment effect on the…

统计方法学 · 统计学 2026-05-06 Arthur Hughes , Rodolphe Thiébaut , Layla Parast , Boris P. Hejblum

This paper introduces the necessary and sufficient conditions that surrogate functions must satisfy to properly define frontiers of non-dominated solutions in multi-objective optimization problems. These new conditions work directly on the…

人工智能 · 计算机科学 2015-12-21 Conrado Silva Miranda , Fernando José Von Zuben

Surrogate endpoints are used in place of long-term outcomes in randomized experiments when observing the real outcome for a large enough cohort is prohibitively expensive or impractical. A short-term surrogate is good if the result of an…

In many decision-making problems, the primary outcome is expensive, time-consuming, or difficult to observe, so individualized treatment rules (ITRs) may be instead learned from surrogate endpoints. However, a surrogate that is highly…

统计方法学 · 统计学 2026-04-13 Zeyu Xu , Xiaojie Mao , Hao Mei , Yue Liu

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…

统计方法学 · 统计学 2022-09-20 Xuan Wang , Layla Parast , Lu Tian , Tianxi Cai

For many rare diseases with no approved preventive interventions, promising interventions exist, yet it has been difficult to conduct a pivotal phase 3 trial that could provide direct evidence demonstrating a beneficial effect on the target…

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,…

统计方法学 · 统计学 2026-03-24 Sarah C. Lotspeich , P. D. Anh. Nguyen , Layla Parast
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