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Inference in hierarchical nonlinear models needs careful consideration about targeting parameters that have either a conditional or population-average interpretation. For the special case of mixed-effects nonlinear sigmoidal models we…

应用统计 · 统计学 2017-07-11 Daniel Gerhard , Christian Ritz

Estimating the effects of continuous-valued interventions from observational data is a critically important task for climate science, healthcare, and economics. Recent work focuses on designing neural network architectures and…

When considering the effect a treatment will cause in a population of interest, we often look to evidence from randomized controlled trials. In settings where multiple trials on a treatment are available, we may wish to synthesize the…

统计方法学 · 统计学 2023-09-08 Nicole Schnitzler , Eloise Kaizar

In experimental and observational data settings, researchers often have limited knowledge of the reasons for missing outcomes. To address this uncertainty, we propose bounds on causal effects for missing outcomes, accommodating the scenario…

统计方法学 · 统计学 2026-03-19 Max Rubinstein , Denis Agniel , Larry Han , Marcela Horvitz-Lennon , Sharon-Lise Normand

Health economic evaluations face the issues of non-compliance and missing data. Here, non-compliance is defined as non-adherence to a specific treatment, and occurs within randomised controlled trials (RCTs) when participants depart from…

应用统计 · 统计学 2019-02-26 Karla DiazOrdaz , Richard Grieve

Baseline covariates in randomized experiments are often used in the estimation of treatment effects, for example, when estimating treatment effects within covariate-defined subgroups. In practice, however, covariate values may be missing…

统计方法学 · 统计学 2023-08-29 Gauri Kamat , Jerome P. Reiter

Missing data is unavoidable in longitudinal clinical trials, and outcomes are not always normally distributed. In the presence of outliers or heavy-tailed distributions, the conventional multiple imputation with the mixed model with…

统计方法学 · 统计学 2022-03-22 Siyi Liu , Yilong Zhang , Gregory T Golm , Guanghan , Liu , Shu Yang

Mediation analysis has been comprehensively studied for independent data but relatively little work has been done for correlated data, especially for the increasingly adopted stepped wedge cluster randomized trials (SW-CRTs). Motivated by…

统计方法学 · 统计学 2025-04-03 Zhiqiang Cao , Fan Li

Multiple outcomes, both continuous and discrete, are routinely gathered on subjects in longitudinal studies and during routine clinical follow-up in general. To motivate our work, we consider a longitudinal study on patients with primary…

应用统计 · 统计学 2013-04-17 Arnošt Komárek , Lenka Komárková

In most medical research, the average treatment effect is used to evaluate a treatment's performance. However, precision medicine requires knowledge of individual treatment effects: What is the difference between a unit's measurement under…

统计计算 · 统计学 2022-08-30 Mingyang Cai , Stef van Buuren , Gerko Vink

This paper studies inference in cluster randomized trials where treatment status is determined according to a "matched pairs" design. Here, by a cluster randomized experiment, we mean one in which treatment is assigned at the level of the…

计量经济学 · 经济学 2025-08-14 Yuehao Bai , Jizhou Liu , Azeem M. Shaikh , Max Tabord-Meehan

Causal identification of treatment effects for infectious disease outcomes in interconnected populations is challenging because infection outcomes may be transmissible to others, and treatment given to one individual may affect others'…

统计方法学 · 统计学 2021-05-11 Xiaoxuan Cai , Eben Kenah , Forrest W. Crawford

The analysis of randomized controlled trials is often complicated by intercurrent events (IEs) -- events that occur after treatment initiation and affect either the interpretation or existence of outcome measurements. Examples include…

统计方法学 · 统计学 2026-04-07 Sizhu Lu , Yanyao Yi , Yongming Qu , Huayu Karen Liu , Ting Ye , Peng Ding

The micro-randomized trial (MRT) is an experimental design that can be used to develop optimal mobile health interventions. In MRTs, interventions in the form of notifications or messages are sent through smart phones to individuals,…

统计方法学 · 统计学 2022-02-14 Shuangning Li , Stefan Wager

Typically, trials investigate the impact of either an individual-level intervention on participant outcomes, or the impact of a cluster-level intervention on participant outcomes. Factorial designs consider two (or more) treatments for each…

统计方法学 · 统计学 2026-05-04 Rhys Bowden , Rebecca Walwyn , Jessica Kasza , Andrew Copas , Fan Li , James Wason , Andrew Forbes

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

To test scientific theories and develop individualized treatment rules, researchers often wish to learn heterogeneous treatment effects that can be consistently found across diverse populations and contexts. We consider the problem of…

统计方法学 · 统计学 2026-05-01 Yi Zhang , Melody Huang , Kosuke Imai

Missing exposure information is a very common feature of many observational studies. Here we study identifiability and efficient estimation of causal effects on vector outcomes, in such cases where treatment is unconfounded but partially…

统计方法学 · 统计学 2020-02-04 Edward H. Kennedy

A multivariate mixed-effects model seems to be the most appropriate for gene expression data collected in a crossover trial. It is, however, difficult to obtain reliable results using standard statistical inference when some responses are…

统计方法学 · 统计学 2023-09-12 Savita Pareek , Kalyan Das , Siuli Mukhopadhyay

A central obstacle in the objective assessment of treatment effect (TE) estimators in randomized control trials (RCTs) is the lack of ground truth (or validation set) to test their performance. In this paper, we propose a novel…