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Safety analyses in terms of adverse events (AEs) are an important aspect of benefit-risk assessments of therapies. Compared to efficacy analyses AE analyses are often rather simplistic. The probability of an AE of a specific type is…

Applications · Statistics 2021-05-20 Regina Stegherr , Claudia Schmoor , Michael Lübbert , Tim Friede , Jan Beyersmann

We consider the problem of estimating the causal effect of a treatment on an outcome in linear structural causal models (SCM) with latent confounders when we have access to a single proxy variable. Several methods (such as…

Computational Engineering, Finance, and Science · Computer Science 2023-06-13 Yaroslav Kivva , Saber Salehkaleybar , Negar Kiyavash

Risk management is particularly concerned with extreme events, but analysing these events is often hindered by the scarcity of data, especially in a multivariate context. This data scarcity complicates risk management efforts. Various tools…

Methodology · Statistics 2026-01-15 Nisrine Madhar , Juliette Legrand , Maud Thomas

Given the long follow-up periods that are often required for treatment or intervention studies, the potential to use surrogate markers to decrease the required follow-up time is a very attractive goal. However, previous studies have shown…

Methodology · Statistics 2016-08-12 Layla Parast , Tianxi Cai , Lu Tian

Practitioners are interested in not only the average causal effect of the treatment on the outcome but also the underlying causal mechanism in the presence of an intermediate variable between the treatment and outcome. However, in many…

Methodology · Statistics 2016-02-04 Peng Ding , Jiannan Lu

A delay between the occurrence and the reporting of events often has practical implications such as for the amount of capital to hold for insurance companies, or for taking preventive actions in case of infectious diseases. The accurate…

Applications · Statistics 2021-06-24 Roel Verbelen , Katrien Antonio , Gerda Claeskens , Jonas Crevecoeur

In situations with non-manipulable exposures, interventions can be targeted to shift the distribution of intermediate variables between exposure groups to define interventional disparity indirect effects. In this work, we present a…

Methodology · Statistics 2023-05-18 Helene C. W. Rytgaard , Amalie Lykkemark Møller , Thomas A. Gerds

Background: A common intercurrent event affecting many trials is when some participants do not begin their assigned treatment. Many trials use a modified intention-to-treat (mITT) approach, whereby participants who do not initiate treatment…

Methodology · Statistics 2023-12-04 Brennan C Kahan , Ian R White , Mark Edwards , Michael O Harhay

Competing risk is a common phenomenon when dealing with time-to-event outcomes in biostatistical applications. An attractive estimand in this setting is the "number of life-years lost due to a specific cause of death", Andersen et al.…

Methodology · Statistics 2025-02-04 Simon Christoffer Ziersen , Torben Martinussen

It is of substantial scientific interest to detect mediators that lie in the causal pathway from an exposure to a survival outcome. However, with high-dimensional mediators, as often encountered in modern genomic data settings, there is a…

Methodology · Statistics 2024-08-14 Tzu-Jung Huang , Zhonghua Liu , Ian W. McKeague

In this article, we consider models for time-to-event data obtained from experiments in which stress levels are altered at intermediate stages during the observation period. These experiments, known as step-stress tests, belong to the…

Applications · Statistics 2018-07-04 Nandini Kannan , Debasis Kundu

Suppose one wishes to estimate the effect of a binary treatment on a binary endpoint conditional on a post-randomization quantity in a counterfactual world in which all subjects received treatment. It is generally difficult to identify this…

Methodology · Statistics 2019-11-12 Alex Luedtke , Jiacheng Wu

A parametric expression for causal natural direct and indirect effects is derived for the setting of a binary outcome with a binary mediator. The proposed effect decomposition does not require the outcome to be rare and generalizes the…

Methodology · Statistics 2021-04-13 Marco Doretti , Martina Raggi , Elena Stanghellini

In longitudinal studies where units are embedded in space or a social network, interference may arise, meaning that a unit's outcome can depend on treatment histories of others. The presence of interference poses significant challenges for…

Methodology · Statistics 2025-08-26 Ye Wang , Michael Jetsupphasuk

Natural direct and indirect effects are mediational estimands that decompose the average treatment effect and describe how outcomes would be affected by contrasting levels of a treatment through changes induced in mediator values (in the…

Methodology · Statistics 2022-05-10 Kara E. Rudolph , Ivan Diaz

Semi-competing risks data arise when both non-terminal and terminal events are considered in a model. Such data with multiple events of interest are frequently encountered in medical research and clinical trials. In this framework, terminal…

Methodology · Statistics 2022-11-21 Fatemeh Mahmoudi , Xuewen Lu

We study the conditional expert Kaplan-Meier estimator, an extension of the classical Kaplan--Meier estimator designed for time-to-event data subject to both right-censoring and contamination. Such contamination, where observed events may…

Methodology · Statistics 2026-01-06 Martin Bladt , Kristian Vilhelm Dinesen

Estimands can help to clarify the research questions being addressed in randomised trials. Because the choice of estimand can affect how relevant trial results are to patients and other stakeholders, such as clinicians or policymakers, it…

Causal effect moderation investigates how the effect of interventions (or treatments) on outcome variables changes based on observed characteristics of individuals, known as potential effect moderators. With advances in data collection,…

Methodology · Statistics 2024-11-26 Soham Bakshi , Walter Dempsey , Snigdha Panigrahi

Construction of just-in-time adaptive interventions, such as prompts delivered by mobile apps to promote and maintain behavioral change, requires knowledge about time-varying moderated effects to inform when and how we deliver intervention…

Methodology · Statistics 2022-12-06 Jieru Shi , Zhenke Wu , Walter Dempsey