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相关论文: Robust Evaluation of Longitudinal Surrogate Marker…

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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 clinical trials where long follow-up is required to measure the primary outcome of interest, there is substantial interest in using an accepted surrogate outcome that can be measured earlier in time or with less cost to estimate a…

统计方法学 · 统计学 2024-12-19 Xuan Wang , Jie Zhou , Layla Parast , Tom Greene

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…

Estimating the effects of long-term treatments through A/B testing is challenging. Treatments, such as updates to product functionalities, user interface designs, and recommendation algorithms, are intended to persist within the system for…

计量经济学 · 经济学 2025-12-30 Shan Huang , Chen Wang , Yuan Yuan , Jinglong Zhao , Brocco , Zhang

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

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 study the identification and estimation of long-term treatment effects when both experimental and observational data are available. Since the long-term outcome is observed only after a long delay, it is not measured in the experimental…

统计方法学 · 统计学 2024-09-04 Guido Imbens , Nathan Kallus , Xiaojie Mao , Yuhao Wang

Long-term outcomes of experimental evaluations are necessarily observed after long delays. We develop semiparametric methods for combining the short-term outcomes of experiments with observational measurements of short-term and long-term…

计量经济学 · 经济学 2023-08-21 Jiafeng Chen , David M. Ritzwoller

Estimating long-term causal effects based on short-term surrogates is a significant but challenging problem in many real-world applications, e.g., marketing and medicine. Despite its success in certain domains, most existing methods…

机器学习 · 计算机科学 2023-11-22 Ruichu Cai , Weilin Chen , Zeqin Yang , Shu Wan , Chen Zheng , Xiaoqing Yang , Jiecheng Guo

Causal mediation analysis seeks to investigate how the treatment effect of an exposure on outcomes is mediated through intermediate variables. Although many applications involve longitudinal data, the existing methods are not directly…

应用统计 · 统计学 2021-02-24 Shuxi Zeng , Stacy Rosenbaum , Elizabeth Archie , Susan Alberts , Fan Li

Long-term outcomes are often unavailable in randomized clinical trials, although short-term surrogate outcomes are commonly observed. External observational data may contain the long-term outcome, but causal comparisons based on such data…

统计理论 · 数学 2026-05-15 Ziyang Liu , Niwen Zhou , Peng Wu , Xu Guo

In many randomized experiments, the treatment effect of the long-term metric (i.e. the primary outcome of interest) is often difficult or infeasible to measure. Such long-term metrics are often slow to react to changes and sufficiently…

统计方法学 · 统计学 2024-06-18 Nilesh Tripuraneni , Lee Richardson , Alexander D'Amour , Jacopo Soriano , Steve Yadlowsky

Screening and surveillance are routinely used in medicine for early detection of disease and close monitoring of progression. Biomarkers are one of the primarily tools used for these tasks, but their successful translation to clinical…

We consider linear regression model estimation where the covariate of interest is randomly censored. Under a non-informative censoring mechanism, one may obtain valid estimates by deleting censored observations. However, this comes at a…

应用统计 · 统计学 2017-10-24 Folefac Atem , Roland A. Matsouaka

The use of observational time series data to assess the impact of multi-time point interventions is becoming increasingly common as more health and activity data are collected and digitized via wearables, social media, and electronic health…

统计方法学 · 统计学 2020-12-01 Roy Adams , Suchi Saria , Michael Rosenblum

The onset of several silent, chronic diseases such as diabetes can be detected only through diagnostic tests. Due to cost considerations, self-reported outcomes are routinely collected in lieu of expensive diagnostic tests in large-scale…

应用统计 · 统计学 2015-09-15 Xiangdong Gu , Yunsheng Ma , Raji Balasubramanian

A very classical problem in statistics is to test the stochastic superiority of one distribution to another. However, many existing approaches are developed for independent samples and, moreover, do not take censored data into account. We…

统计方法学 · 统计学 2023-06-19 Dennis Dobler , Kathrin Möllenhoff

The primary analysis of clinical trials in diabetes therapeutic area often involves a mixed-model repeated measure (MMRM) approach to estimate the average treatment effect for longitudinal continuous outcome, and a generalized linear mixed…

应用统计 · 统计学 2022-08-03 Lingjing Jiang , Michael Rosenblum , Yu Du

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