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Random-effects meta-analyses are used to combine evidence of treatment effects from multiple studies. Since treatment effects may vary across trials due to differences in study characteristics, heterogeneity in treatment effects between…

统计方法学 · 统计学 2017-07-10 Tim Friede , Christian Röver , Simon Wandel , Beat Neuenschwander

Multi-regional clinical trials (MRCTs) enable efficient global drug development by assessing treatment effects across regions within a single protocol. While powered for overall efficacy, MRCTs are typically not designed to provide…

This paper reviews and compares methods to assess treatment effect heterogeneity in the context of parametric regression models. These methods include the standard likelihood ratio tests, bootstrap likelihood ratio tests, and Goeman's…

应用统计 · 统计学 2026-02-06 Yao Chen , Sophie Sun , Konstantinos Sechidis , Cong Zhang , Torsten Hothorn , Björn Bornkamp

Case-mix heterogeneity across studies complicates meta-analyses. As a result of this, treatments that are equally effective on patient subgroups may appear to have different effectiveness on patient populations with different case mix. It…

应用统计 · 统计学 2021-08-25 Tat-Thang Vo , Raphael Porcher , Anna Chaimani , Stijn Vansteelandt

The treatment effects of the same therapy observed from multiple clinical trials can often be very different. Yet the patient characteristics accounting for these differences may not be identifiable in real world practice. There needs to be…

统计理论 · 数学 2015-03-20 Tinghui Yu , Yabing Mai , Sherry Liu , Xiaofei Hu

Multidimensional heterogeneity and endogeneity are important features of models with multiple treatments. We consider a heterogeneous coefficients model where the outcome is a linear combination of dummy treatment variables, with each…

计量经济学 · 经济学 2021-11-23 Whitney K. Newey , Sami Stouli

Treatment effect heterogeneity occurs when individual characteristics influence the effect of a treatment. We propose a novel approach that combines prognostic score matching and conditional inference trees to characterize effect…

Randomized controlled trials play an important role in how Internet companies predict the impact of policy decisions and product changes. In these `digital experiments', different units (people, devices, products) respond differently to the…

应用统计 · 统计学 2015-12-21 Matt Taddy , Matt Gardner , Liyun Chen , David Draper

Randomized trials typically estimate average relative treatment effects, but decisions on the benefit of a treatment are possibly better informed by more individualized predictions of the absolute treatment effect. In case of a binary…

统计方法学 · 统计学 2021-08-20 J Hoogland , J IntHout , M Belias , MM Rovers , RD Riley , FE Harrell , KGM Moons , TPA Debray , JB Reitsma

The causal effect of a treatment can vary from person to person based on their individual characteristics and predispositions. Mining for patterns of individual-level effect differences, a problem known as heterogeneous treatment effect…

机器学习 · 计算机科学 2019-09-04 Christopher Tran , Elena Zheleva

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

One size fits all approaches to medicine have become a thing of the past as the understanding of individual differences grows. The paper introduces a test for the presence of heterogeneity in treatment effects in a clinical trial.…

Estimating the joint effect of a multivariate, continuous exposure is crucial, particularly in environmental health where interest lies in simultaneously evaluating the impact of multiple environmental pollutants on health. We develop novel…

统计方法学 · 统计学 2025-01-06 Heejun Shin , Antonio Linero , Michelle Audirac , Kezia Irene , Danielle Braun , Joseph Antonelli

This article proposes different tests for treatment effect heterogeneity when the outcome of interest, typically a duration variable, may be right-censored. The proposed tests study whether a policy 1) has zero distributional (average)…

统计方法学 · 统计学 2020-02-19 Pedro H. C. Sant'Anna

Identifying heterogeneity in a population's response to a health or policy intervention is crucial for evaluating and informing policy decisions. We propose a novel heterogeneous treatment effect estimator in the difference-in-differences…

统计方法学 · 统计学 2021-08-24 Xinkun Nie , Chen Lu , Stefan Wager

Investigators often use multi-source data (e.g., multi-center trials, meta-analyses of randomized trials, pooled analyses of observational cohorts) to learn about the effects of interventions in subgroups of some well-defined target…

统计方法学 · 统计学 2024-02-06 Guanbo Wang , Alexander Levis , Jon Steingrimsson , Issa Dahabreh

The use of patient-level information from previous studies, registries, and other external datasets can support the analysis of single-arm and randomized controlled trials to evaluate and test experimental treatments. However, the…

统计方法学 · 统计学 2025-10-23 Gopal Kotecha , Daniel E. Schwartz , Steffen Ventz , Lorenzo Trippa

We propose a test-based elastic integrative analysis of the randomized trial and real-world data to estimate treatment effect heterogeneity with a vector of known effect modifiers. When the real-world data are not subject to bias, our…

统计方法学 · 统计学 2022-11-30 Shu Yang , Chenyin Gao , Donglin Zeng , Xiaofei Wang

Individuals often respond differently to identical treatments, and characterizing such variability in treatment response is an important aim in the practice of personalized medicine. In this article, we describe a non-parametric accelerated…

统计方法学 · 统计学 2017-06-22 Nicholas C. Henderson , Thomas A. Louis , Gary L. Rosner , Ravi Varadhan

Empirical studies using Regression Discontinuity (RD) designs often explore heterogeneous treatment effects based on pretreatment covariates, even though no formal statistical methods exist for such analyses. This has led to the widespread…

计量经济学 · 经济学 2025-07-08 Sebastian Calonico , Matias D. Cattaneo , Max H. Farrell , Filippo Palomba , Rocio Titiunik