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相关论文: On the Evaluation of Surrogate Markers in Real Wor…

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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 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

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

Objective: Traditionally validation of surrogate endpoints has been carried out using RCT data. However, RCT data may be too limited to validate surrogate endpoints. In this paper, we sought to improve validation of surrogate endpoints with…

应用统计 · 统计学 2021-12-17 Lorna Wheaton , Anastasios Papanikos , Anne Thomas , Sylwia Bujkiewicz

In biomedical studies, estimating drug effects on chronic diseases requires a long follow-up period, which is difficult to meet in randomized clinical trials (RCTs). The use of a short-term surrogate to replace the long-term outcome for…

统计方法学 · 统计学 2023-06-27 Wenjie Hu , Xiaohua Zhou , Peng Wu

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…

Study populations are typically sampled from limited points in space and time, and marginalized groups are underrepresented. To assess the external validity of randomized and observational studies, we propose and evaluate the worst-case…

机器学习 · 统计学 2022-02-04 Sookyo Jeong , Hongseok Namkoong

We propose a novel multi-task neural network approach for estimating distributional treatment effects (DTE) in randomized experiments. While DTE provides more granular insights into the experiment outcomes over conventional methods focusing…

机器学习 · 计算机科学 2025-07-11 Tomu Hirata , Undral Byambadalai , Tatsushi Oka , Shota Yasui , Shingo Uto

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

Estimating the total treatment effect (TTE) of a new feature in social platforms is crucial for understanding its impact on user behavior. However, the presence of network interference, which arises from user interactions, often complicates…

应用统计 · 统计学 2024-08-09 Yiming Jiang , Lu Deng , Yong Wang , He Wang

Augmenting randomized controlled trials (RCTs) with external real-world data (RWD) has the potential to improve the finite sample efficiency of treatment effect estimators. We describe using adaptive targeted maximum likelihood estimation…

统计方法学 · 统计学 2025-01-30 Sky Qiu , Jens Tarp , Andrew Mertens , Mark van der Laan

We consider the problem of estimating the effects of a binary treatment on a continuous outcome of interest from observational data in the absence of confounding by unmeasured factors. We provide a new estimator of the population average…

统计方法学 · 统计学 2020-08-04 James Robins , Mariela Sued , Quanhong Lei-Gomez , Andrea Rotnitzky

Randomized Controlled Trials (RCT) are the current gold standards to empirically measure the effect of a new drug. However, they may be of limited size and resorting to complementary non-randomized data, referred to as observational, is…

统计方法学 · 统计学 2025-06-11 Ahmed Boughdiri , Julie Josse , Erwan Scornet

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

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

Policy makers typically face the problem of wanting to estimate the long-term effects of novel treatments, while only having historical data of older treatment options. We assume access to a long-term dataset where only past treatments were…

计量经济学 · 经济学 2022-02-11 Keith Battocchi , Eleanor Dillon , Maggie Hei , Greg Lewis , Miruna Oprescu , Vasilis Syrgkanis

The weighted average treatment effect (WATE) is a causal measure for the comparison of interventions in a specific target population, which may be different from the population where data are sampled from. For instance, when the goal is to…

统计方法学 · 统计学 2018-04-17 Yebin Tao , Haoda Fu

The term `surrogate modeling' in computational science and engineering refers to the development of computationally efficient approximations for expensive simulations, such as those arising from numerical solution of partial differential…

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

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
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