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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

It is common that in multiarm randomized trials, the outcome of interest is "truncated by death," meaning that it is only observed or well defined conditioning on an intermediate outcome. In this case, in addition to pairwise contrasts, the…

Methodology · Statistics 2016-11-22 Linbo Wang , Thomas S. Richardson , Xiao-Hua Zhou

Various methods have been developed to combine inference across multiple sets of results for unsupervised clustering, within the ensemble clustering literature. The approach of reporting results from one `best' model out of several…

In large observational studies, the case-cohort design is commonly used to reduce the cost associated with covariate measurement. For survival outcomes, literature has suggested that the restricted mean survival time (RMST) be a more…

Methodology · Statistics 2026-05-08 Andy Ni , Wei-En Lu , Bo Lu

Methods for estimating heterogeneous treatment effect in observational data have largely focused on continuous or binary outcomes, and have been relatively less vetted with survival outcomes. Using flexible machine learning methods in the…

Applications · Statistics 2021-07-09 Liangyuan Hu , Jiayi Ji , Fan Li

The number of recurrent events before a terminating event is often of interest. For instance, death terminates an individual's process of rehospitalizations and the number of rehospitalizations is an important indicator of economic cost. We…

Methodology · Statistics 2021-12-30 Willem van den Boom , Maria De Iorio , Marta Tallarita

There have been significant efforts devoted to solving the longevity risk given that a continuous growth in population ageing has become a severe issue for many developed countries over the past few decades. The Cairns-Blake-Dowd (CBD)…

Applications · Statistics 2024-12-30 Ka Kin Lam , Bo Wang

Estimating conditional average treatment effects (CATE) is challenging, especially when treatment information is missing. Although this is a widespread problem in practice, CATE estimation with missing treatments has received little…

Machine Learning · Statistics 2023-04-19 Milan Kuzmanovic , Tobias Hatt , Stefan Feuerriegel

While palliative care is increasingly commonly delivered to hospitalized patients with serious illnesses, few studies have estimated its causal effects. Courtright et al. (2016) adopted a cluster-randomized stepped-wedge design to assess…

In neoadjuvant trials on early-stage breast cancer, patients are usually randomized into a control group and a treatment group with an additional target therapy. Early efficacy of the new regimen is assessed via the binary pathological…

Methodology · Statistics 2022-04-04 Xiaoqing Tan , Judah Abberbock , Priya Rastogi , Gong Tang

Immediately following a disaster event, such as an earthquake, estimates of the damage extent play a key role in informing the coordination of response and recovery efforts. We develop a novel impact estimation tool that leverages a…

Applications · Statistics 2025-01-15 Max Anderson Loake , Hamish Patten , David Steinsaltz

The restricted mean survival time (RMST) model has been garnering attention as a way to provide a clinically intuitive measure: the mean survival time. RMST models, which use methods based on pseudo time-to-event values and inverse…

Methodology · Statistics 2024-06-11 Keisuke Hanada , Masahiro Kojima

In this article, we develop methods for sample size and power calculations in four-level intervention studies when intervention assignment is carried out at any level, with a particular focus on cluster randomized trials (CRTs). CRTs…

Methodology · Statistics 2022-09-07 Xueqi Wang , Elizabeth L. Turner , John S. Preisser , Fan Li

Background: In settings where proof-of-principle trials have succeeded but the effectiveness of different forms of implementation remains uncertain, trials that not only generate information about intervention effects but also provide…

Quantitative Methods · Quantitative Biology 2017-05-16 Guy Harling , Rui Wang , Jukka-Pekka Onnela , Victor De Gruttola

Cluster-randomized experiments are widely used due to their logistical convenience and policy relevance. To analyze them properly, we must address the fact that the treatment is assigned at the cluster level instead of the individual level.…

Methodology · Statistics 2021-08-06 Fangzhou Su , Peng Ding

In both observational studies and randomized trials, post-treatment events such as dropout, nonadherence, and truncation by death occur frequently. In some studies, conditioning on post-treatment variables is a deliberate strategy to…

Methodology · Statistics 2026-04-24 Marco Piccininni , Mats J. Stensrud

Randomized controlled trials (RCTs) are the accepted standard for treatment effect estimation but they can be infeasible due to ethical reasons and prohibitive costs. Single-arm trials, where all patients belong to the treatment group, can…

Background: Mendelian randomization (MR) is a useful approach to causal inference from observational studies when randomised controlled trials are not feasible. However, study heterogeneity of two association studies required in MR is often…

Methodology · Statistics 2021-12-16 Linyi Zou , Hui Guo , Carlo Berzuini

Missing data is unavoidable in longitudinal clinical trials, and outcomes are not always normally distributed. In the presence of outliers or heavy-tailed distributions, the conventional multiple imputation with the mixed model with…

Methodology · Statistics 2022-03-22 Siyi Liu , Yilong Zhang , Gregory T Golm , Guanghan , Liu , Shu Yang

In estimating the causal effect of a continuous exposure or treatment, it is important to control for all confounding factors. However, most existing methods require parametric specification for how control variables influence the outcome…

Applications · Statistics 2020-07-21 Spencer Woody , Carlos M. Carvalho , P. Richard Hahn , Jared S. Murray