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Since the release of the ICH E9(R1) addendum on estimands, its application in non-inferiority trials has received far less attention than in superiority settings. A key conclusion from Lynggaard et al. was that the "choice of…

Applications · Statistics 2026-03-12 Tobias Mütze , Helle Lynggaard , Sunita Rehal , Oliver N. Keene , Marian Mitroiu , David Wright

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

Researchers are frequently interested in understanding the causal effect of treatment interventions. However, in some cases, the treatment of interest--readily available in a randomized controlled trial (RCT)--is either not directly…

Methodology · Statistics 2025-09-29 Lan Wen , Aaron L Sarvet

In clinical trials with recurrent events, such as repeated hospitalizations terminating with death, it is important to consider the patient events overall history for a thorough assessment of treatment effects. The occurrence of fewer…

Methodology · Statistics 2025-09-08 Alessandra Ragni , Torben Martinussen , Thomas Scheike

Many interventions are both beneficial to initiate and harmful to stop. Traditionally, to determine whether to deploy that intervention in a time-limited way depends on if, on average, the increase in the benefits of starting it outweigh…

We focus on the problem of generalizing a causal effect estimated on a randomized controlled trial (RCT) to a target population described by a set of covariates from observational data. Available methods such as inverse propensity sampling…

Methodology · Statistics 2023-02-27 Imke Mayer , Julie Josse , Traumabase Group

Missing data are often dealt with multiple imputation. A crucial part of the multiple imputation process is selecting sensible models to generate plausible values for incomplete data. A method based on posterior predictive checking is…

Computation · Statistics 2026-05-14 Mingyang Cai , Stef van Buuren , Gerko Vink

Panel data consists of a collection of $N$ units that are observed over $T$ units of time. A policy or treatment is subject to staggered adoption if different units take on treatment at different times and remains treated (or never at all).…

Methodology · Statistics 2025-08-14 Eric Xia , Yuling Yan , Martin J. Wainwright

The current COVID-19 pandemic poses numerous challenges for ongoing clinical trials and provides a stress-testing environment for the existing principles and practice of estimands in clinical trials. The pandemic may increase the rate of…

Methodology · Statistics 2023-02-02 Yongming Qu , Ilya Lipkovich

This project was motivated by a dialysis study in northern Taiwan. Dialysis patients, after shunt implantation, may experience two types ("acute" or "non-acute") of shunt thrombosis, both of which may recur. We formulate the problem under…

Methodology · Statistics 2017-07-07 Bowen Li

Estimating heterogeneous treatment effects is central to data-driven decision-making, yet industrial applications often face a fundamental tension between limited randomized controlled trial (RCT) budgets and abundant but biased…

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…

Methodology · Statistics 2023-10-17 Yong Wu , Mingzhou Liu , Jing Yan , Yanwei Fu , Shouyan Wang , Yizhou Wang , Xinwei Sun

Researchers increasingly leverage movement across multiple treatments to estimate causal effects. While these "mover regressions" are often motivated by a linear constant-effects model, it is not clear what they capture under weaker…

Econometrics · Economics 2018-04-19 Peter Hull

The problem of missing data, usually absent incurated and competition-standard datasets, is an unfortunate reality for most machine learning models used in industry applications. Recent work has focused on understanding the nature and the…

Machine Learning · Computer Science 2022-01-25 Spyridon Mouselinos , Kyriakos Polymenakos , Antonis Nikitakis , Konstantinos Kyriakopoulos

We investigate the problem of calibration and assessment of predictive rules in prognostic designs when missing values are present in the predictors. Our paper has two key objectives which are entwined. The first is to investigate how the…

Methodology · Statistics 2018-10-12 B. J. A. Mertens , E. Banzato , L. C. de Wreede

We study the problem of a decision maker who must provide the best possible treatment recommendation based on an experiment. The desirability of the outcome distribution resulting from the policy recommendation is measured through a…

Econometrics · Economics 2022-04-06 Anders Bredahl Kock , David Preinerstorfer , Bezirgen Veliyev

Joint modeling technique is a recent advancement in effectively analyzing the longitudinal history of patients with the occurrence of an event of interest attached to it. This procedure is successfully implemented in biomarker studies to…

Methodology · Statistics 2021-01-08 Gajendra K. Vishwakarma , Atanu Bhattacharjee , Souvik Banerjee

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…

Methodology · Statistics 2026-04-23 Lawson Ung , Guanbo Wang , Sebastien Haneuse , Miguel A. Hernán , Issa J. Dahabreh

Bipartite experiments arise in various fields, in which the treatments are randomized over one set of units, while the outcomes are measured over another separate set of units. However, existing methods often rely on strong model…

Methodology · Statistics 2025-04-16 Sizhu Lu , Lei Shi , Yue Fang , Wenxin Zhang , Peng Ding

In both observational studies and randomized trials, post-treatment events such as dropout, nonadherence, and truncation by death occur frequently. In some studies, conditioning on post-treatment variables is a deliberate strategy to…

Methodology · Statistics 2026-04-24 Marco Piccininni , Mats J. Stensrud
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