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Public policy-makers use cost-effectiveness analyses (CEA) to decide which health and social care interventions to provide. Appropriate methods have not been developed for handling missing data in complex settings, such as for CEA that use…

Methodology · Statistics 2012-06-27 Karla Diaz-Ordaz , Michael G. Kenward , Richard Grieve

Unknown constraints arise in many types of expensive black-box optimization problems. Several methods have been proposed recently for performing Bayesian optimization with constraints, based on the expected improvement (EI) heuristic.…

When drawing causal inferences about the effects of multiple treatments on clustered survival outcomes using observational data, we need to address implications of the multilevel data structure, multiple treatments, censoring and unmeasured…

Methodology · Statistics 2022-02-18 Liangyuan Hu , Jiayi Ji , Ronald D. Ennis , Joseph W. Hogan

Analysis of observational studies increasingly confronts the challenge of determining which of a possibly high-dimensional set of available covariates are required to satisfy the assumption of ignorable treatment assignment for estimation…

Methodology · Statistics 2022-03-23 Chanmin Kim , Mauricio Tec , Corwin M Zigler

Cost-Effectiveness Analyses (CEAs) alongside randomised controlled trials (RCTs) are increasingly often designed to collect resource use and preference-based health status data for the purpose of healthcare technology assessment. However,…

Applications · Statistics 2016-07-22 Andrea Gabrio , Alexina Mason , Gianluca Baio

We overview Bayesian estimation, hypothesis testing, and model-averaging and illustrate how they benefit parametric survival analysis. We contrast the Bayesian framework to the currently dominant frequentist approach and highlight…

Methodology · Statistics 2022-09-13 František Bartoš , Frederik Aust , Julia M. Haaf

In causality, estimating the effect of a treatment without confounding inference remains a major issue because requires to assess the outcome in both case with and without treatment. Not being able to observe simultaneously both of them,…

Machine Learning · Computer Science 2021-12-09 Celine Beji , Florian Yger , Jamal Atif

Quantifying causal effects of exposures on outcomes, such as a treatment and a disease respectively, is a crucial issue in medical science for the administration of effective therapies. Importantly, any related causal analysis should…

Methodology · Statistics 2024-09-04 Federico Castelletti , Laura Ferrini

Inferring treatment effects on a survival time outcome based on data from an observational study is challenging due to the presence of censoring and possible confounding. An additional challenge occurs when a unit's treatment affects the…

Methodology · Statistics 2025-08-13 Chanhwa Lee , Donglin Zeng , Michael Emch , John D. Clemens , Michael G. Hudgens

Meta-analysis is a powerful tool for assessing drug safety by combining treatment-related toxicological findings across multiple studies, as clinical trials are typically underpowered for detecting adverse drug effects. However, incomplete…

We consider the linear regression problem under semi-supervised settings wherein the available data typically consists of: (i) a small or moderate sized 'labeled' data, and (ii) a much larger sized 'unlabeled' data. Such data arises…

Methodology · Statistics 2018-07-02 Abhishek Chakrabortty , Tianxi Cai

To investigate whether treating cancer patients with erythropoiesis-stimulating agents (ESAs) would increase the mortality risk, Bennett et al. [Journal of the American Medical Association 299 (2008) 914--924] conducted a meta-analysis with…

Applications · Statistics 2010-10-11 Rui Wang , Lu Tian , Tianxi Cai , L. J. Wei

Estimating causal effects from observational data is inherently challenging due to the lack of observable counterfactual outcomes and even the presence of unmeasured confounding. Traditional methods often rely on restrictive, untestable…

Methodology · Statistics 2025-04-07 Li Chen , Xiaotong Shen , Wei Pan

Trial-based cost-effectiveness analyses (CEAs) are an important source of evidence in the assessment of health interventions. In these studies, cost and effectiveness outcomes are commonly measured at multiple time points, but some…

Methodology · Statistics 2022-03-30 Andrea Gabrio , Catrin Plumpton , Sube Banerjee , Baptiste Leurent

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

Complex data features, such as unmodelled censored event times and variables with time-dependent effects, are common in cancer recurrence studies and pose challenges for Bayesian survival modelling. Current methodologies for predictive…

Methodology · Statistics 2026-01-12 Saku Suorsa , Aki Vehtari

Bayesian nonparametric methods are a popular choice for analysing survival data due to their ability to flexibly model the distribution of survival times. These methods typically employ a nonparametric prior on the survival function that is…

Methodology · Statistics 2022-02-22 Edwin Fong , Brieuc Lehmann

Estimates of the effects of treatment on cost from observational studies are subject to bias if there are unmeasured confounders. It is therefore advisable in practice to assess the potential magnitude of such biases. We derive a general…

Applications · Statistics 2014-01-09 Elizabeth A. Handorf , Justin E. Bekelman , Daniel F. Heitjan , Nandita Mitra

There has recently been a growing interest in the development of statistical methods to compare medical costs between treatment groups. When cumulative cost is the outcome of interest, right-censoring poses the challenge of informative…

Methodology · Statistics 2017-05-25 Andrew J. Spieker , Arman Oganisian , Emily M. Ko , Jason A. Roy , Nandita Mitra

Usual parametric and semi-parametric regression methods are inappropriate and inadequate for large clustered survival studies when the appropriate functional forms of the covariates and their interactions in hazard functions are unknown,…

Methodology · Statistics 2024-11-12 Durbadal Ghosh , Debajyoti Sinha , Antonio R. Linero , George Rust