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Related papers: Multiply robust estimators in longitudinal studies…

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This article investigates the model-robustness of fixed-effects models for analyzing a broad class of longitudinal cluster trials (CTs) such as stepped-wedge, parallel-with-baseline and crossover designs, encompassing both randomized (CRTs)…

Methodology · Statistics 2026-04-10 Kenneth M. Lee , Fan Li

In biostatistics and medical research, longitudinal data are often composed of repeated assessments of a variable (e.g., blood pressure or other biomarkers) and dichotomous indicators to mark an event of interest (e.g., recovery from…

Applications · Statistics 2019-09-13 Sezen Cekic , Stephen Aichele , Andreas M. Brandmaier , Ylva Köhncke , Paolo Ghisletta

The difference-in-differences (DiD) design is a quasi-experimental method for estimating treatment effects. In staggered DiD with multiple treatment groups and periods, estimation based on the two-way fixed effects model yields negative…

Methodology · Statistics 2026-03-05 Yuhao Deng , Le Kang

Estimating causal effects in a target population with unmeasured confounders is challenging, especially when instrumental variables (IVs) are unavailable. However, IVs from auxiliary populations with similar problems can help infer causal…

Methodology · Statistics 2025-08-06 Wei Li , Jiapeng Liu , Peng Ding , Zhi Geng

In the absence of data from a randomized trial, researchers often aim to use observational data to draw causal inference about the effect of a treatment on a time-to-event outcome. In this context, interest often focuses on the…

Methodology · Statistics 2021-06-15 Ted Westling , Alex Luedtke , Peter Gilbert , Marco Carone

Missing data is a systemic problem in practical scenarios that causes noise and bias when estimating treatment effects. This makes treatment effect estimation from data with missingness a particularly tricky endeavour. A key reason for this…

Machine Learning · Statistics 2023-02-27 Jeroen Berrevoets , Fergus Imrie , Trent Kyono , James Jordon , Mihaela van der Schaar

This research was motivated by studying anti-drug antibody (ADA) formation and its potential impact on long-term benefit of a biologic treatment in a randomized controlled trial, in which ADA status was not only unobserved in the control…

Methodology · Statistics 2021-01-13 Shengchun Kong , Dominik Heinzmann , Sabine Lauer , Tian Lu

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…

Methodology · Statistics 2020-02-04 Edward H. Kennedy

Target trial emulation (TTE) enables causal questions to be studied with observational data when randomized controlled trials (RCTs) are infeasible. Yet treatment-effect methods often address causal estimation, missingness, and temporal…

A platform trial is an innovative clinical trial design that uses a master protocol to evaluate multiple treatments, where patients are often assigned to different subsets of treatment arms based on individual characteristics, enrollment…

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…

Methodology · Statistics 2023-09-08 Nicole Schnitzler , Eloise Kaizar

We propose a joint model for a time-to-event outcome and a quantile of a continuous response repeatedly measured over time. The quantile and survival processes are associated via shared latent and manifest variables. Our joint model…

Methodology · Statistics 2014-04-07 Alessio Farcomeni , Sara Viviani

This paper develops methods for uncertainty quantification in causal inference settings with random network interference. We study the large-sample distributional properties of the classical difference-in-means Hajek treatment effect…

Methodology · Statistics 2025-11-11 Matias D. Cattaneo , Yihan He , Ruiqi Rae Yu

When multiple measures are collected repeatedly over time, redundancy typically exists among responses. The envelope method was recently proposed to reduce the dimension of responses without loss of information in regression with…

Methodology · Statistics 2021-03-25 Yuyang Shi , Linquan Ma , Lan Liu

Replicating causal estimates across different cohorts is crucial for increasing the integrity of epidemiological studies. However, strong assumptions regarding unmeasured confounding and effect modification often hinder this goal. By…

Methodology · Statistics 2024-09-23 Roy S. Zawadzki , Daniel L. Gillen

Randomized experiments are the gold standard for estimating treatment effects, yet network interference challenges the validity of traditional estimators by violating the stable unit treatment value assumption and introducing bias. While…

Methodology · Statistics 2024-09-02 Xin Lu , Hongzi Li , Hanzhong Liu

Mean-based estimators of causal effects in randomized experiments may behave poorly if the potential outcomes have a heavy tail or contain outliers. An alternative estimator proposed by Rosenbaum (1993) estimates a constant additive…

Methodology · Statistics 2026-02-09 Aditya Ghosh , Nabarun Deb , Bikram Karmakar , Bodhisattva Sen

Interventional effects have been proposed as a solution to the unidentifiability of natural (in)direct effects under mediator-outcome confounders affected by the exposure. Such confounders are an intrinsic characteristic of studies with…

Methodology · Statistics 2022-03-30 Iván Díaz , Nicholas Williams , Kara E. Rudolph

Multi-regional clinical trials (MRCTs) play an increasingly crucial role in global pharmaceutical development by expediting data gathering and regulatory approval across diverse patient populations. However, differences in recruitment…

Methodology · Statistics 2024-04-15 Kaiyuan Hua , Hwanhee Hong , Xiaofei Wang

Missing data and confounding are two problems researchers face in observational studies for comparative effectiveness. Williamson et al. (2012) recently proposed a unified approach to handle both issues concurrently using a multiply-robust…

Methodology · Statistics 2020-07-22 Katherine Evans , Isabel Fulcher , Eric J. Tchetgen Tchetgen