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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…

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

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

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…

统计方法学 · 统计学 2025-04-22 Rebecca Knowlton , Layla Parast

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.…

统计方法学 · 统计学 2026-04-15 Silvaneo V. dos Santos , Layla Parast

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…

统计方法学 · 统计学 2026-04-07 Oliver Dukes , Mats J. Stensrud , Riccardo Brioschi , Aaron Hudson

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

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…

统计方法学 · 统计学 2022-11-10 Denis Agniel , Layla Parast , Boris Hejblum

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

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

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…

The development of statistical methods to evaluate surrogate markers is an active area of research. In many clinical settings, the surrogate marker is not simply a single measurement but is instead a longitudinal trajectory of measurements…

统计方法学 · 统计学 2024-02-28 Denis Agniel , 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…

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

Surrogate markers are often employed in clinical trials to replace primary outcomes that may be difficult, expensive, or time-consuming to measure directly. These markers can accelerate the evaluation of new treatments, provided they…

统计方法学 · 统计学 2026-03-17 Pietro Carlotti , Layla Parast

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

The primary analysis in two-arm clinical trials usually involves inference on a scalar treatment effect parameter; e.g., depending on the outcome, the difference of treatment-specific means, risk difference, risk ratio, or odds ratio. Most…

统计方法学 · 统计学 2022-04-25 Anastasios A. Tsiatis , Marie Davidian

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

When estimating treatment effects, the golden standard is to conduct a randomized experiment and then contrast outcomes associated with the treatment group and the control group. However, in many cases, randomized experiments are either…

统计方法学 · 统计学 2023-06-08 Kevin Han

We consider the problem of testing for treatment effect heterogeneity in observational studies, and propose a nonparametric test based on multisample U-statistics. To account for potential confounders, we use reweighted data where the…

统计方法学 · 统计学 2021-03-30 Maozhu Dai , Weining Shen , Hal S. Stern

This article proposes different tests for treatment effect heterogeneity when the outcome of interest, typically a duration variable, may be right-censored. The proposed tests study whether a policy 1) has zero distributional (average)…

统计方法学 · 统计学 2020-02-19 Pedro H. C. Sant'Anna

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
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