ArticleStatistics in medicine2021
Optimizing subgroup selection in two-stage adaptive enrichment and umbrella designs.
Article in Statistics in medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers, 1 of them a synthesis that pooled it.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
10 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Design and analysis of umbrella trials: Where do we stand?Frontiers in medicine · 2022Pooled it
- An Adaptive Biomarker-based Umbrella Trial Design Using Bayesian Latent Class Model.Statistics in biopharmaceutical research · 2026Article
- A Seamless Hybrid Phase II/III Design With Bayesian Interim Subgroup Selection.Statistics in medicine · 2025Article
- How to make cardiology clinical trials more inclusive.Nature medicine · 2024Review
- Improving Collection and Analysis of Overall Survival Data.Clinical cancer research : an official journal of the American Association for Cancer Research · 2024Review
- Developing generic templates to shape the future for conducting integrated research platform trials.Trials · 2024Article
- Simulation study for evaluating an adaptive-randomisation Bayesian hybrid trial design with enrichment.Contemporary clinical trials communications · 2023Article
- Innovations in Clinical Development in Rare Diseases of Children and Adults: Small Populations and/or Small Patients.Paediatric drugs · 2022Review
- Enrichment Bayesian design for randomized clinical trials using categorical biomarkers and a binary outcome.BMC medical research methodology · 2022Article
- Optimizing subgroup selection in two-stage adaptive enrichment and umbrella designs.Statistics in medicine · 2021Article
Corrections and comments
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Authors and funding
6 authors.
Funding
Abstract
We design two-stage confirmatory clinical trials that use adaptation to find the subgroup of patients who will benefit from a new treatment, testing for a treatment effect in each of two disjoint subgroups. Our proposal allows aspects of the trial, such as recruitment probabilities of each group, to be altered at an interim analysis. We use the conditional error rate approach to implement these adaptations with protection of overall error rates. Applying a Bayesian decision-theoretic framework, we optimize design parameters by maximizing a utility function that takes the population prevalence of the subgroups into account. We show results for traditional trials with familywise error rate control (using a closed testing procedure) as well as for umbrella trials in which only the per-comparison type 1 error rate is controlled. We present numerical examples to illustrate the optimization process and the effectiveness of the proposed designs.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.