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Cost-effectiveness analyses (CEAs) compare the costs and health outcomes of treatment regimes to inform medical decisions. With observational claims data, CEAs must address nonrandom treatment assignment, administrative censoring, and…

Methodology · Statistics 2026-01-22 Esteban Fernández-Morales , Emily M. Ko , Nandita Mitra , Youjin Lee , Arman Oganisian

Healthcare decision-making often requires selecting among treatment options under budget constraints, particularly when one option is more effective but also more costly. Cost-effectiveness analysis (CEA) provides a framework for evaluating…

Econometrics · Economics 2025-07-29 Jonas Esser , Mateus Maia , Judith Bosmans , Johanna van Dongen

Patient-level health economic data collected alongside clinical trials are an important component of the process of technology appraisal, with a view to informing resource allocation decisions. For end of life treatments, such as cancer…

Applications · Statistics 2020-11-24 Andrea Gabrio

Researchers are often interested in predicting outcomes, conducting clustering analysis to detect distinct subgroups of their data, or computing causal treatment effects. Pathological data distributions that exhibit skewness and…

Methodology · Statistics 2020-08-24 Arman Oganisian , Nandita Mitra , Jason Roy

Causal effect estimation (CEE) provides a crucial tool for predicting the unobserved counterfactual outcome for an entity. As CEE relaxes the requirement for ``perfect'' counterfactual samples (e.g., patients with identical attributes and…

Machine Learning · Computer Science 2024-11-19 Hechuan Wen , Tong Chen , Guanhua Ye , Li Kheng Chai , Shazia Sadiq , Hongzhi Yin

Bayesian modelling for cost-effectiveness data has received much attention in both the health economics and the statistical literature in recent years. Cost-effectiveness data are characterised by a relatively complex structure of…

Statistics Theory · Mathematics 2013-07-22 Gianluca Baio

Adaptive experimentation enables efficient estimation of causal effects, but existing methods are not designed for survival data with censoring, where event times are only partially observed (e.g., overall survival in cancer trials but with…

Machine Learning · Computer Science 2026-05-19 Yuxin Wang , Dennis Frauen , Jonas Schweisthal , Maresa Schröder , Emil Javurek , Stefan Feuerriegel

Conventional survival analysis approaches estimate risk scores or individualized time-to-event distributions conditioned on covariates. In practice, there is often great population-level phenotypic heterogeneity, resulting from (unknown)…

Machine Learning · Statistics 2020-03-03 Paidamoyo Chapfuwa , Chunyuan Li , Nikhil Mehta , Lawrence Carin , Ricardo Henao

We evaluated the implications of different approaches to characterize uncertainty of calibrated parameters of stochastic decision models (DMs) in the quantified value of such uncertainty in decision making. We used a microsimulation DM of…

The solution here proposed can be used to conduct economic analysis in randomized clinical trials. It is based on a statistical approach and aims at calculating a revised version of the incremental costeffective ratio (ICER) in order to…

Applications · Statistics 2013-06-19 C. D'Urso

Statistical agencies and other institutions collect data under the promise to protect the confidentiality of respondents. When releasing microdata samples, the risk that records can be identified must be assessed. To this aim, a widely…

Applications · Statistics 2015-06-03 Cinzia Carota , Maurizio Filippone , Roberto Leombruni , Silvia Polettini

Datasets containing large samples of time-to-event data arising from several small heterogeneous groups are commonly encountered in statistics. This presents problems as they cannot be pooled directly due to their heterogeneity or analyzed…

Machine Learning · Statistics 2016-12-05 Alexandre Piché , Russell Steele , Ian Shrier , Stephanie Long

Causal mediation analysis in cluster-randomized trials (CRTs) is essential for explaining how cluster-level interventions affect individual outcomes, yet it is complicated by interference, post-treatment confounding, and hierarchical…

Methodology · Statistics 2026-03-25 Yuki Ohnishi , Michael J. Daniels , Lei Yang , Fan Li

Modern epidemiological analytics increasingly use machine learning models that offer strong prediction but often lack calibrated uncertainty. Bayesian methods provide principled uncertainty quantification, yet are viewed as difficult to…

Machine Learning · Statistics 2025-11-18 Debashis Chatterjee

Unmeasured confounding bias threatens the validity of observational studies. While sensitivity analyses and study designs have been proposed to address this issue, they often overlook the growing availability of auxiliary data. Using…

Methodology · Statistics 2024-10-22 Jie Kate Hu , Dafne Zorzetto , Francesca Dominici

We address causal estimation in semi-competing risks settings, where a non-terminal event may be precluded by one or more terminal events. We define a principal-stratification causal estimand for treatment effects on the non-terminal event,…

Methodology · Statistics 2025-06-27 Karina Gelis-Cadena , Michael Daniels , Juned Siddique

A Bayesian non-parametric framework for studying time-to-event data is proposed, where the prior distribution is allowed to depend on an additional random source, and may update with the sample size. Such scenarios are natural, for…

Methodology · Statistics 2025-05-06 Martin Bladt , Jorge González Cázares

In the field of population health research, understanding the similarities between geographical areas and quantifying their shared effects on health outcomes is crucial. In this paper, we synthesise a number of existing methods to create a…

Applications · Statistics 2023-11-27 Wala Draidi Areed , Aiden Price , Helen Thompson , Reid Malseed , Kerrie Mengersen

In cancer epidemiology, the \emph{relative survival framework} is used to quantify the hazard associated with cancer by comparing the all-cause mortality hazard in cancer patients to that of the general population. This framework assumes…

Applications · Statistics 2024-11-05 Piyali Basak , Antonio R. Linero , Camille Maringe , F. Javier Rubio

Bayesian paradigm takes advantage of well fitting complicated survival models and feasible computing in survival analysis owing to the superiority in tackling the complex censoring scheme, compared with the frequentist paradigm. In this…

Methodology · Statistics 2021-09-10 Chong Zhong , Zhihua Ma , Junshan Shen , Catherine Liu
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