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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

Learning the Individual Treatment Effect (ITE) is essential for personalized decision-making, yet causal inference has traditionally focused on aggregated treatment effects. While integrating conformal prediction with causal inference can…

统计方法学 · 统计学 2025-01-23 Chenyin Gao , Peter B. Gilbert , Larry Han

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…

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…

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

In statistical inference, it is rarely realistic that the hypothesized statistical model is well-specified, and consequently it is important to understand the effects of misspecification on inferential procedures. When the hypothesized…

统计方法学 · 统计学 2025-09-01 Beomjo Park , Sivaraman Balakrishnan , Larry Wasserman

We study the problem of model selection in causal inference, specifically for conditional average treatment effect (CATE) estimation. Unlike machine learning, there is no perfect analogue of cross-validation for model selection as we do not…

机器学习 · 计算机科学 2024-04-30 Divyat Mahajan , Ioannis Mitliagkas , Brady Neal , Vasilis Syrgkanis

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

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

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

This dissertation focuses on modern causal inference under uncertainty and data restrictions, with applications to neoadjuvant clinical trials, distributed data networks, and robust individualized decision making. In the first project, we…

统计方法学 · 统计学 2023-01-24 Xiaoqing Tan

What is the difference of a prediction that is made with a causal model and a non-causal model? Suppose we intervene on the predictor variables or change the whole environment. The predictions from a causal model will in general work as…

统计方法学 · 统计学 2024-04-27 Jonas Peters , Peter Bühlmann , Nicolai Meinshausen

Identification of causal effects is one of the most fundamental tasks of causal inference. We consider an identifiability problem where some experimental and observational data are available but neither data alone is sufficient for the…

人工智能 · 计算机科学 2019-03-13 Santtu Tikka , Juha Karvanen

Surrogate models are statistical or conceptual approximations for more complex simulation models. In this context, it is crucial to propagate the uncertainty induced by limited simulation budget and surrogate approximation error to…

机器学习 · 统计学 2026-01-27 Philipp Reiser , Javier Enrique Aguilar , Anneli Guthke , Paul-Christian Bürkner

Surrogate modelling techniques have seen growing attention in recent years when applied to both modelling and optimisation of industrial design problems. These techniques are highly relevant when assessing the performance of a particular…

统计方法学 · 统计学 2024-03-14 Nicolau Andrés-Thió , Mario Andrés Muñoz , Kate Smith-Miles

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

The goal of causal inference is to understand the outcome of alternative courses of action. However, all causal inference requires assumptions. Such assumptions can be more influential than in typical tasks for probabilistic modeling, and…

统计方法学 · 统计学 2016-10-31 Dustin Tran , Francisco J. R. Ruiz , Susan Athey , David M. Blei

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

In causal inference, interference occurs when the treatment of one unit may affect the outcomes of other units. The goal of this work is to serve as a guide to the use of linear outcome modeling for estimating causal effects in settings…

统计方法学 · 统计学 2026-04-01 Eric Tong , Salvador V. Balkus
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