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Suppose we have a binary treatment used to influence an outcome. Given data from an observational or controlled study, we wish to determine whether or not there exists some subset of observed covariates in which the treatment is more…

统计方法学 · 统计学 2016-03-22 Alexander R. Luedtke , Mark J. van der Laan

Long-term causal inference has drawn increasing attention in many scientific domains. Existing methods mainly focus on estimating average long-term causal effects by combining long-term observational data and short-term experimental data.…

机器学习 · 计算机科学 2025-03-04 Weilin Chen , Ruichu Cai , Junjie Wan , Zeqin Yang , José Miguel Hernández-Lobato

The hypothesis of homogeneous treatment effects is central to the instrumental variables literature. This assumption signifies that treatment effects are constant across all subjects. It allows to interpret instrumental variable estimates…

计量经济学 · 经济学 2023-04-17 Jad Beyhum , Jean-Pierre Florens , Elia Lapenta , Ingrid Van Keilegom

There has been a growing interest in covariate adjustment in the analysis of randomized controlled trials in past years. For instance, the U.S. Food and Drug Administration recently issued guidance that emphasizes the importance of…

统计方法学 · 统计学 2023-06-12 Kelly Van Lancker , Frank Bretz , Oliver Dukes

In this paper, we address the issue of estimating and inferring distributional treatment effects in randomized experiments. The distributional treatment effect provides a more comprehensive understanding of treatment heterogeneity compared…

计量经济学 · 经济学 2025-01-15 Tatsushi Oka , Shota Yasui , Yuta Hayakawa , Undral Byambadalai

Although combination antiretroviral therapy (ART) is highly effective in suppressing viral load for people with HIV (PWH), many ART agents may exacerbate central nervous system (CNS)-related adverse effects including depression. Therefore,…

统计方法学 · 统计学 2020-04-14 Wei Jin , Yang Ni , Leah H. Rubin , Amanda B. Spence , Yanxun Xu

For mental disorders, patients' underlying mental states are non-observed latent constructs which have to be inferred from observed multi-domain measurements such as diagnostic symptoms and patient functioning scores. Additionally,…

机器学习 · 计算机科学 2020-11-03 Yuan Chen , Donglin Zeng , Tianchen Xu , Yuanjia Wang

There is increasing interest in combining information from experimental studies, including randomized and single-group trials, with information from external experimental or observational data sources. Such efforts are usually motivated by…

统计方法学 · 统计学 2026-04-23 Lawson Ung , Guanbo Wang , Sebastien Haneuse , Miguel A. Hernán , Issa J. Dahabreh

Non-significant randomized control trials can hide subgroups of good responders to experimental drugs, thus hindering subsequent development. Identifying such heterogeneous treatment effects is key for precision medicine and many post-hoc…

统计方法学 · 统计学 2024-01-24 Valentine Perrin , Nathan Noiry , Nicolas Loiseau , Alex Nowak

In many social, behavioral, and biomedical sciences, treatment effect estimation is a crucial step in understanding the impact of an intervention, policy, or treatment. In recent years, an increasing emphasis has been placed on…

统计方法学 · 统计学 2024-10-10 Xinhai Zhang , Xingye Qiao

Comparative binary outcome data are of fundamental interest in statistics and are often pooled in meta-analyses. Here we examine the simplest case where for each study there are two patient groups and a binary event of interest, giving rise…

统计方法学 · 统计学 2018-06-12 Rose Baker , Dan Jackson

Analyzing data from multiple sources offers valuable opportunities to improve the estimation efficiency of causal estimands. However, this analysis also poses many challenges due to population heterogeneity and data privacy constraints.…

统计方法学 · 统计学 2025-10-23 Rong Zhao , Jason Falvey , Xu Shi , Vernon M. Chinchilli , Chixiang Chen

The standard paradigm for confirmatory clinical trials is to compare experimental treatments with a control, for example the standard of care or a placebo. However, it is not always the case that a suitable control exists. Efficient…

统计方法学 · 统计学 2024-10-29 Thomas Burnett , Thomas Jaki

Laparoscopic surgery has been shown through a number of randomized trials to be an effective form of treatment for cholecystitis. Given this evidence, one natural question for clinical practice is: does the effectiveness of laparoscopic…

统计方法学 · 统计学 2023-11-09 Matteo Bonvini , Zhenghao Zeng , Miaoqing Yu , Edward H. Kennedy , Luke Keele

Not only does mobile health technology enable researchers to track changes in multiple longitudinal outcomes of interest and to record the occurrence of health-related events over time, but it also allows for the delivery of repeated…

Heterogeneous treatment effects, which vary according to individual covariates, are crucial in fields such as personalized medicine and tailored treatment strategies. In many applications, rather than considering the heterogeneity induced…

统计方法学 · 统计学 2025-08-26 Peng Wu , Pengtao Zeng , Zhaoqing Tian , Shaojie Wei

Cluster-level dynamic treatment regimens can be used to guide sequential, intervention or treatment decision-making at the cluster level in order to improve outcomes at the individual or patient-level. In a cluster-level DTR, the…

统计方法学 · 统计学 2016-07-15 Timothy NeCamp , Amy Kilbourne , Daniel Almirall

We provide sufficient conditions for the identification of the heterogeneous treatment effects, defined as the conditional expectation for the differences of potential outcomes given the untreated outcome, under the nonignorable treatment…

统计方法学 · 统计学 2019-01-15 Keisuke Takahata , Takahiro Hoshino

A key aspect of patient-focused drug development is identifying and measuring outcomes that are important to patients in clinical trials. Many medical conditions affect multiple symptom domains, and a consensus approach to determine the…

统计方法学 · 统计学 2025-10-14 David S. Robertson , Thomas Jaki

The empirical literature on program evaluation limits its scope almost exclusively to models where treatment effects are homogenous for observationally identical individuals. This paper considers a treatment effect model in which treatment…

统计方法学 · 统计学 2019-02-20 Jason Abrevaya , Haiqing Xu