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Interval-censored data analysis is important in biomedical statistics for any type of time-to-event response where the time of response is not known exactly, but rather only known to occur between two assessment times. Many clinical trials…

Methodology · Statistics 2019-06-12 Weichi Yao , Halina Frydman , Jeffrey S. Simonoff

In this paper, we expand the methodology presented in Mertens et. al (2020, Biometrical Journal) to the study of life-time (survival) outcome which is subject to censoring and when imputation is used to account for missing values. We…

Methodology · Statistics 2021-05-06 Bart J. A. Mertens

We propose a novel methodology to quantify the effect of stochastic interventions on non-terminal time-to-events that lie on the pathway between an exposure and a terminal time-to-event outcome. Investigating these effects is particularly…

We study the identification and estimation of long-term treatment effects when both experimental and observational data are available. Since the long-term outcome is observed only after a long delay, it is not measured in the experimental…

Methodology · Statistics 2024-09-04 Guido Imbens , Nathan Kallus , Xiaojie Mao , Yuhao Wang

Often in Phase 3 clinical trials measuring a long-term time-to-event endpoint, such as overall survival or progression-free survival, investigators also collect repeated measures on biomarkers which may be predictive of the primary…

Methodology · Statistics 2022-11-30 Abigail J. Burdon , Lisa V. Hampson , Christopher Jennison

Applied researchers in biomedicine and related fields are often interested in estimating the causal effect of a treatment or intervention. Although randomized clinical trials are considered the gold standard for establishing causal effects,…

Many medical decisions involve the use of dynamic information collected on individual patients toward predicting likely transitions in their future health status. If accurate predictions are developed, then a prognostic mode can identify…

Methodology · Statistics 2018-02-22 Aasthaa Bansal , Patrick J. Heagerty

In survival analysis it often happens that some subjects under study do not experience the event of interest; they are considered to be `cured'. The population is thus a mixture of two subpopulations: the one of cured subjects, and the one…

Statistics Theory · Mathematics 2017-01-16 Valentin Patilea , Ingrid Van Keilegom

Consider a survival time T that is subject to random right censoring, and suppose that T is stochastically dependent on the censoring time C. We are interested in the marginal distribution of T. This situation is often encountered in…

Methodology · Statistics 2021-04-15 Claudia Czado , Ingrid Van Keilegom

Lung cancer remains one of the leading causes of cancer-related mortality, yet most survival models rely only on baseline factors and overlook posttreatment variables that reflect disease progression. To address this gap, we applied Cox…

Applications · Statistics 2025-10-03 Varun Vishwanathan Nair , Victor Miranda Soberanis

How should researchers analyze randomized experiments in which the main outcome is latent and measured in multiple ways but each measure contains some degree of error? We first identify a critical study-specific noncomparability problem in…

Econometrics · Economics 2026-01-13 Jiawei Fu , Donald P. Green

Time plays a fundamental role in causal analyses, where the goal is to quantify the effect of a specific treatment on future outcomes. In a randomized experiment, times of treatment, and when outcomes are observed, are typically well…

Methodology · Statistics 2016-01-19 Guillaume W. Basse , Alexander Volfovsky , Edoardo M. Airoldi

Estimating the long-term effects of treatments is of interest in many fields. A common challenge in estimating such treatment effects is that long-term outcomes are unobserved in the time frame needed to make policy decisions. One approach…

Methodology · Statistics 2024-08-23 Susan Athey , Raj Chetty , Guido Imbens , Hyunseung Kang

Adaptive subgroup enrichment design is an efficient design framework that allows accelerated development for investigational treatments while also having flexibility in population selection within the course of the trial. The adaptive…

Methodology · Statistics 2022-10-21 Liwen Wu , Qing Li , Mengya Liu , Jianchang Lin

Survival analysis plays a crucial role in estimating the likelihood of future events for patients by modeling time-to-event data, particularly in healthcare settings where predictions about outcomes such as death and disease recurrence are…

Machine Learning · Computer Science 2024-10-01 Muhammad Ridzuan , Numan Saeed , Fadillah Adamsyah Maani , Karthik Nandakumar , Mohammad Yaqub

Observational cohort data is an important source of information for understanding the causal effects of treatments on survival and the degree to which these effects are mediated through changes in disease-related risk factors. However,…

Methodology · Statistics 2026-05-20 Saurabh Bhandari , Michael J. Daniels , Juned Siddique

In observational studies of survival time featuring a binary time-dependent treatment, the hazard ratio (an instantaneous measure) is often used to represent the treatment effect. However, investigators are often more interested in the…

Methodology · Statistics 2020-05-21 Yun Li , Douglas E. Schaubel , Kevin He

Randomized trials are considered the gold standard for making informed decisions in medicine, yet they often lack generalizability to the patient populations in clinical practice. Observational studies, on the other hand, cover a broader…

Methodology · Statistics 2026-04-14 Piersilvio De Bartolomeis , Javier Abad , Konstantin Donhauser , Fanny Yang

A very classical problem in statistics is to test the stochastic superiority of one distribution to another. However, many existing approaches are developed for independent samples and, moreover, do not take censored data into account. We…

Methodology · Statistics 2023-06-19 Dennis Dobler , Kathrin Möllenhoff

Clinical trials or studies oftentimes require long-term and/or costly follow-up of participants to evaluate a novel treatment/drug/vaccine. There has been increasing interest in the past few decades in using short-term surrogate outcomes as…

Methodology · Statistics 2024-12-19 Xuan Wang , Tianxi Cai , Lu Tian , Layla Parast