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In classical study designs, the aim is often to learn about the effects of a treatment or intervention on a single outcome; in many modern studies, however, data on multiple outcomes are collected and it is of interest to explore effects on…

统计方法学 · 统计学 2017-06-15 Edward H. Kennedy , Shreya Kangovi , Nandita Mitra

Understanding treatment effect heterogeneity has become increasingly important in many fields. In this paper we study distributions and quantiles of individual treatment effects to provide a more comprehensive and robust understanding of…

统计方法学 · 统计学 2026-03-31 Zhe Chen , Xinran Li

The predictions from an accurate prognostic model can be of great interest to patients and clinicians. When predictions are reported to individuals, they may decide to take action to improve their health or they may simply be comforted by…

定量方法 · 定量生物学 2019-09-10 Michael C Sachs , Arvid Sjölander , Erin E Gabriel

Despite their cost, randomized controlled trials (RCTs) are widely regarded as gold-standard evidence in disciplines ranging from social science to medicine. In recent decades, researchers have increasingly sought to reduce the resource…

统计方法学 · 统计学 2026-02-24 Guilherme Duarte

Assessing causal effects in the presence of unobserved confounding is a challenging problem. Existing studies leveraged proxy variables or multiple treatments to adjust for the confounding bias. In particular, the latter approach attributes…

统计方法学 · 统计学 2023-10-17 Yong Wu , Mingzhou Liu , Jing Yan , Yanwei Fu , Shouyan Wang , Yizhou Wang , Xinwei Sun

Scientists regularly pose questions about treatment effects on outcomes conditional on a post-treatment event. However, causal inference in such settings requires care, even in perfectly executed randomized experiments. Recently, the…

统计方法学 · 统计学 2026-02-19 Chan Park , Mats Stensrud , Eric Tchetgen Tchetgen

Data from both a randomized trial and an observational study are sometimes simultaneously available for evaluating the effect of an intervention. The randomized data typically allows for reliable estimation of average treatment effects but…

统计方法学 · 统计学 2021-12-01 David Cheng , Tianxi Cai

When evaluating the efficacy of social programs and medical treatments using randomized experiments, the estimated overall average causal effect alone is often of limited value and the researchers must investigate when the treatments do and…

应用统计 · 统计学 2013-05-27 Kosuke Imai , Marc Ratkovic

When engagement with a randomized trial is driven by factors that affect the outcome or when trial engagement directly affects the outcome independent of treatment, the average treatment effect among trial participants is unlikely to…

统计方法学 · 统计学 2019-06-27 Issa J. Dahabreh , James M. Robins , Sebastien J-P. A. Haneuse , Miguel A. Hernán

Treatment effect estimation is essential for informed decision-making in many fields such as healthcare, economics, and public policy. While flexible machine learning models have been widely applied for estimating heterogeneous treatment…

机器学习 · 计算机科学 2025-09-29 Pascal Memmesheimer , Vincent Heuveline , Jürgen Hesser

A fundamental limitation of causal inference in observational studies is that perceived evidence for an effect might instead be explained by factors not accounted for in the primary analysis. Methods for assessing the sensitivity of a…

统计方法学 · 统计学 2018-09-14 Colin B. Fogarty

Evaluating the treatment effects has become an important topic for many applications. However, most existing literature focuses mainly on the average treatment effects. When the individual effects are heavy-tailed or have outlier values,…

统计方法学 · 统计学 2023-05-11 Yongchang Su , Xinran Li

Randomized experiments are a powerful methodology for data-driven evaluation of decisions or interventions. Yet, their validity may be undermined by network interference. This occurs when the treatment of one unit impacts not only its…

统计方法学 · 统计学 2024-10-08 Sadegh Shirani , Mohsen Bayati

Meta-analysis, by synthesizing effect estimates from multiple studies conducted in diverse settings, stands at the top of the evidence hierarchy in clinical research. Yet, conventional approaches based on fixed- or random-effects models…

The purpose of this paper is to investigate and develop methods for analysis of multi-center randomized clinical trials which only rely on the randomization process as a basis of inference. Our motivation is prompted by the fact that most…

应用统计 · 统计学 2008-07-28 Lu Zheng , Marvin Zelen

The Causal Roadmap outlines a systematic approach to asking and answering questions of cause-and-effect: define the quantity of interest, evaluate needed assumptions, conduct statistical estimation, and carefully interpret results. To…

统计方法学 · 统计学 2024-05-30 Nerissa Nance , Maya L. Petersen , Mark van der Laan , Laura B. Balzer

The development of high-throughput sequencing and targeted therapies has led to the emergence of personalized medicine: a patient's molecular profile or the presence of a specific biomarker of drug response will correspond to a treatment…

应用统计 · 统计学 2020-05-27 Jonas Béal , Aurélien Latouche

Randomized experiments are considered the gold standard for estimating causal effects. However, out of the set of possible randomized assignments, some may be likely to produce poor effect estimates and misleading conclusions. Restricted…

统计方法学 · 统计学 2025-08-28 Maggie Wang , René F. Kizilcec , Michael Baiocchi

Randomized experiments can provide unbiased estimates of sample average treatment effects. However, estimates of population treatment effects can be biased when the experimental sample and the target population differ. In this case, the…

统计方法学 · 统计学 2022-11-10 Wenqi Shi , Xi Lin

For obtaining causal inferences that are objective, and therefore have the best chance of revealing scientific truths, carefully designed and executed randomized experiments are generally considered to be the gold standard. Observational…

应用统计 · 统计学 2008-11-12 Donald B. Rubin