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Composite endpoints are commonly used with an anticipation that clinically relevant endpoints as a whole would yield meaningful treatment benefits. The win ratio is a rank-based statistic to summarize composite endpoints, allowing…

统计方法学 · 统计学 2022-12-14 Di Zhang , Stephen R. Wisniewski , Jong-Hyeon Jeong

Quantifying causal effects in the presence of complex and multivariate outcomes remains a key challenge in treatment evaluation. For hierarchical multivariate outcomes, the FDA recommends the Win Ratio and Generalized Pairwise Comparisons…

统计方法学 · 统计学 2026-03-24 Mathieu Even , Julie Josse

Covariate adjustment can enhance precision and power in clinical trials, yet its application to the win odds remains unclear. The win odds is an extension of the win ratio that counts ties as half a win for the treatment and the control…

统计方法学 · 统计学 2026-04-08 Cyrill Scheidegger , Simon Wandel , Tobias Mütze

Cluster randomization trials commonly employ multiple endpoints. When a single summary of treatment effects across endpoints is of primary interest, global hypothesis testing/effect estimation methods represent a common analysis strategy.…

统计方法学 · 统计学 2025-05-19 E. Davies Smith , V. Jairath , G. Zou

The win ratio offers a flexible approach to incorporate the hierarchy of clinical outcomes into the analysis of a composite endpoint, enabling simultaneous consideration of multiple outcome types, unlike traditional time-to-first-event…

统计方法学 · 统计学 2025-07-22 David Kronthaler , Matthias Schwenkglenks , Felix Beuschlein , Ulrike Held

Ordinal outcomes are common in clinical settings where they often represent increasing levels of disease progression or different levels of functional impairment. Such outcomes can characterize differences in meaningful patient health…

统计方法学 · 统计学 2025-08-29 Zhiqiang Cao , Scott Zuo , Mary Ryan Baumann , Kendra Plourde , Patrick Heagerty , Guangyu Tong , Fan Li

The survey experiment is widely used in economics and social sciences to evaluate the effects of treatments or programs. In a standard population-based survey experiment, the experimenter randomly draws experimental units from a target…

统计方法学 · 统计学 2026-05-11 Pengfei Tian , Jiyang Ren , Yingying Ma

Existing weighting methods for treatment effect estimation are often built upon the idea of propensity scores or covariate balance. They usually impose strong assumptions on treatment assignment or outcome model to obtain unbiased…

机器学习 · 计算机科学 2023-05-09 Dongcheng Zhang , Kunpeng Zhang

Conventional methods for analyzing composite endpoints in clinical trials often only focus on the time to the first occurrence of all events in the composite. Therefore, they have inherent limitations because the individual patients' first…

统计方法学 · 统计学 2022-11-29 Jialu Wang , Yeh-Fong Chen , Thomas Gwise

Randomization, as a key technique in clinical trials, can eliminate sources of bias and produce comparable treatment groups. In randomized experiments, the treatment effect is a parameter of general interest. Researchers have explored the…

统计方法学 · 统计学 2023-12-05 Fuyi Tu , Wei Ma , Hanzhong Liu

This paper considers the problem of design-based inference for the average treatment effect in finely stratified experiments. Here, by "design-based'' we mean that the only source of uncertainty stems from the randomness in treatment…

计量经济学 · 经济学 2025-05-08 Yuehao Bai , Xun Huang , Joseph P. Romano , Azeem M. Shaikh , Max Tabord-Meehan

Researchers often use linear regression to analyse randomized experiments to improve treatment effect estimation by adjusting for imbalances of covariates in the treatment and control groups. Our work offers a randomization-based inference…

统计理论 · 数学 2022-07-08 Hanzhong Liu , Yuehan Yang

This paper studies covariate adjusted estimation of the average treatment effect in stratified experiments. We work in a general framework that includes matched tuples designs, coarse stratification, and complete randomization as special…

计量经济学 · 经济学 2024-07-23 Max Cytrynbaum

In this review, we present econometric and statistical methods for analyzing randomized experiments. For basic experiments we stress randomization-based inference as opposed to sampling-based inference. In randomization-based inference,…

统计方法学 · 统计学 2017-10-26 Susan Athey , Guido Imbens

This paper studies inference in randomized controlled trials with covariate-adaptive randomization when there are multiple treatments. More specifically, we study inference about the average effect of one or more treatments relative to…

计量经济学 · 经济学 2019-01-21 Federico A. Bugni , Ivan A. Canay , Azeem M. Shaikh

Win statistics have become increasingly popular for analyzing hierarchical composite endpoints in clinical trials, because they summarize treatment benefit through pairwise comparisons that respect the clinical importance order among…

统计方法学 · 统计学 2026-04-21 Xi Fang , Guangyu Tong , Yuan Huang , F. Perry Wilson , Patrick J. Heagerty , Fan Li

Treatment effect estimands based on win statistics, including the win ratio, win odds, and win difference are increasingly popular targets for summarizing endpoints in clinical trials. Such win estimands offer an intuitive approach for…

统计方法学 · 统计学 2026-02-13 Kenneth M. Lee , Xi Fang , Fan Li , Michael O. Harhay

What proportion of treated units actually benefited from an experimental intervention? What is the median or the largest individual treatment effect? This paper develops methods for answering such questions about the distribution of…

统计方法学 · 统计学 2026-05-11 David Kim , Yongchang Su , Jake Bowers , Xinran Li

Stratification is commonly employed in clinical trials to reduce the chance covariate imbalances and increase the precision of the treatment effect estimate. We propose a general framework for constructing the confidence interval (CI) for a…

统计方法学 · 统计学 2021-10-26 Yongqiang Tang

We study the estimation of treatment effects using samples stratified by treatment status. Standard estimators of the average treatment effect and the local average treatment effect are inconsistent in this setting. We propose consistent…

计量经济学 · 经济学 2025-04-28 Jinyong Hahn , John Ham , Geert Ridder , Shuyang Sheng
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