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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…

Bivariate meta-analysis provides a useful framework for combining information across related studies and has been utilised to combine evidence from clinical studies to evaluate treatment efficacy on two outcomes. It has also been used to…

应用统计 · 统计学 2022-05-20 Tasos Papanikos , John R Thompson , Keith R Abrams , Sylwia Bujkiewicz

In meta-analytic modeling, the functional relationship between a primary and surrogate endpoint is estimated using summary data from a set of completed clinical trials. Parameters in the meta-analytic model are used to assess the quality of…

统计方法学 · 统计学 2025-08-07 James P. Long , Abhishikta Roy , Ehsan Irajizad , Kim-Anh Do , Yu Shen

The use of valid surrogate endpoints is an important stake in clinical research to help reduce both the duration and cost of a clinical trial and speed up the evaluation of interesting treatments. Several methods have been proposed in the…

统计方法学 · 统计学 2025-02-13 Quentin Le Coent , Virginie Rondeau , Catherine Legrand

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

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…

统计方法学 · 统计学 2026-04-16 Xingyu Li , Qing Liu , Xun Jiang , Hong Amy Xia , Brian P. Hobbs , Peng Wei

We introduce in this paper an extension of the meta-analytic (MA) framework for evaluating surrogate endpoints. While the MA framework is regarded as the gold standard for surrogate endpoint evaluation, it is limited in its ability to…

统计方法学 · 统计学 2025-09-03 Florian Stijven , Peter B. Gilbert

A common practice in clinical trials is to evaluate a treatment effect on an intermediate endpoint when the true outcome of interest would be difficult or costly to measure. We consider how to validate intermediate endpoints in a…

统计方法学 · 统计学 2022-11-30 Emily K. Roberts , Michael R. Elliott , Jeremy M. G. Taylor

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…

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

As data collection and simulation capabilities advance, multi-modal learning, the task of learning from multiple modalities and sources of data, is becoming an increasingly important area of research. Surrogate models that learn from data…

机器学习 · 统计学 2026-05-13 Ian Taylor , Juliane Mueller , Julie Bessac

Candidate binary endpoints are often considered as surrogates for time-to-event (TTE) clinical endpoints, primarily because they can be assessed at earlier time points. To be submitted for regulatory approval candidate binary endpoints need…

应用统计 · 统计学 2026-03-23 Renee Y. Ge , Azadeh Shohoudi , Malini Iyengar , Quefeng Li , Judy Li

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

Adaptive designs are increasingly used in clinical trials and online experiments to improve participant outcomes by dynamically updating treatment allocation as data accumulate. In practice, experimenters often consider multiple candidate…

统计方法学 · 统计学 2026-04-08 Wenxin Zhang , Aaron Hudson , Maya Petersen , Mark van der Laan

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

Random-effects meta-analyses are very commonly used in medical statistics. Recent methodological developments include multivariate (multiple outcomes) and network (multiple treatments) meta-analysis. Here we provide a new model and…

统计方法学 · 统计学 2017-08-16 Dan Jackson , Sylwia Bujkiewicz , Martin Law , Richard D Riley , Ian White

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

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

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…

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