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While well-established methods for time-to-event data are available when the proportional hazards assumption holds, there is no consensus on the best approach under non-proportional hazards. A wide range of parametric and non-parametric…

Analysing subgroups defined by biomarkers is of increasing importance in clinical research. In some situations the biomarker is subject to misclassification error, meaning the true subgroups are identified with imperfect sensitivity and…

Methodology · Statistics 2018-01-16 Fang Wan , Andrew C. Titman , Thomas F. Jaki

The Cox proportional hazards model is routinely used to analyze time-to-event data. To use this model requires the definition of a unique well-defined time scale. Most often, observation time is used as the time scale for both clinical and…

Methodology · Statistics 2016-09-09 Prabhakar Chalise , Eric Chicken , Daniel McGee

In two harmonized efficacy studies to prevent HIV infection through multiple infusions of the monoclonal antibody VRC01, a key objective is to evaluate whether the serum concentration of VRC01, which changes cyclically over time along with…

Methodology · Statistics 2018-01-26 Yunda Huang , Yuanyuan Zhang , Zong Zhang , Peter B. Gilbert

Immune checkpoint inhibitor--based therapies often produce heterogeneous survival responses, including early risk, delayed treatment benefit, and durable long-term survival in a subset of patients. In these settings, conventional summary…

Methodology · Statistics 2026-04-28 Yi-Cheng Tai , Weijing Wang , Jedd D. Wolchok , Martin T. Wells

In many clinical contexts, estimating effects of treatment in time-to-event data is complicated not only by confounding, censoring, and heterogeneity, but also by the presence of a cured subpopulation in which the event of interest never…

Methodology · Statistics 2026-02-06 Yuqi Li , Quinn Lanners , Matthew M. Engelhard

This paper develops power and sample size formulas for causal inference with time-to-event outcomes. The target estimand is the marginal hazard ratio: the coefficient of a marginal structural Cox proportional hazard model with treatment as…

Methodology · Statistics 2026-05-19 Chengxin Yang , Bo Liu , Fan Li

In large epidemiologic studies, it is typical for an inexpensive, non-invasive procedure to be used to record disease status during regular follow-up visits, with less frequent assessment by a gold standard test. Inexpensive outcome…

Methodology · Statistics 2022-09-22 Lillian A. Boe , Pamela A. Shaw

In this paper, we introduce a doubly doubly robust estimator for the average and heterogeneous treatment effect for left-truncated-right-censored (LTRC) survival data. In causal inference for survival functions in LTRC survival data, two…

General Economics · Economics 2024-09-04 Guanghui Pan

This paper considers the problem of semi-parametric proportional hazards model fitting for interval, left and right censored survival times. We adopt a more versatile penalized likelihood method to estimate the baseline hazard and the…

Methodology · Statistics 2019-04-16 Jun Ma , Dominique-Laurent Couturier , Stephane Heritier , Ian Marschner

Objectives: Prior event rate ratio (PERR) is a method shown to perform well in mitigating confounding in real-world evidence research but it depends on several model assumptions. We propose an analytic strategy to correct biases arising…

Applications · Statistics 2026-03-03 Xiangmei Ma , Grace Meijuan Yang , Qingyuan Zhuang , Yin Bun Cheung

Randomized clinical trials with time-to-event outcomes have traditionally used the log-rank test followed by the Cox proportional hazards (PH) model to estimate the hazard ratio between the treatment groups. These are valid under the…

Methodology · Statistics 2022-06-07 Jiyu Luo , Ronghui Xu

Evaluation of HIV large scale interventions programme is becoming increasingly important, but impact estimates frequently hinge on knowledge of changes in behaviour such as the frequency of condom use (CU) over time, or other self-reported…

State-of-the-art decentralized learning algorithms typically require the data distribution to be Independent and Identically Distributed (IID). However, in practical scenarios, the data distribution across the agents can have significant…

Machine Learning · Computer Science 2024-03-07 Sai Aparna Aketi , Sakshi Choudhary , Kaushik Roy

Recent years have seen the development of many novel scoring tools for disease prognosis and prediction. To become accepted for use in clinical applications, these tools have to be validated on external data. In practice, validation is…

Methodology · Statistics 2022-12-06 Matthias Schmid , Tim Friede , Nadja Klein , Leonie Weinhold

Bayesian model-averaged meta-analysis allows quantification of evidence for both treatment effectiveness $\mu$ and across-study heterogeneity $\tau$. We use the Cochrane Database of Systematic Reviews to develop discipline-wide empirical…

In this work, we are interested in studying the causal effect of an endogenous binary treatment on a dependently censored duration outcome. By dependent censoring, it is meant that the duration time ($T$) and right censoring time ($C$) are…

Econometrics · Economics 2025-04-04 Gilles Crommen , Jad Beyhum , Ingrid Van Keilegom

Hazard ratios are prone to selection bias, compromising their use as causal estimands. On the other hand, the hazard difference has been shown to remain unaffected by the selection of frailty factors over time. Therefore, observed hazard…

Statistics Theory · Mathematics 2022-11-01 Richard Post , Edwin van den Heuvel , Hein Putter

This paper presents a randomization-based framework for estimating causal effects under interference between units, motivated by challenges that arise in analyzing experiments on social networks. The framework integrates three components:…

Statistics Theory · Mathematics 2018-06-21 Peter M. Aronow , Cyrus Samii

Latent class analysis (LCA) is a useful tool to investigate the heterogeneity of a disease population with time-to-event data. We propose a new method based on non-parametric maximum likelihood estimator (NPMLE), which facilitates…

Methodology · Statistics 2022-02-03 Teng Fei , John Hanfelt , Limin Peng