ArticlePharmaceutical statistics2021
Assessing efficacy in important subgroups in confirmatory trials: An example using Bayesian dynamic borrowing.
Article in Pharmaceutical statistics, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.
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Who cites it
20 citing papers in PubMed.
- Development of Pediatric Dosing for Mepolizumab in Severe Asthma Based on Extrapolation of Data From Adult Patients and a Phase II Open-Label Pediatric Trial.Journal of clinical pharmacology · 2026Trial
- Pharmacometrics-Enhanced Bayesian Borrowing for Pediatric Extrapolation - A Case Study of the DINAMO Trial.Therapeutic innovation & regulatory science · 2025Trial
- Effect of early administration of tetracosactide on mortality and host response in critically ill patients requiring rescue surgery: a sensitivity analysis of the STOPSHOCK phase 3 randomized controlled trial.Military Medical Research · 2024Trial
- Bayesian Dynamic Borrowing to Enhance Evidence for New Therapies.PharmacoEconomics · 2026Review
- Efficacy and safety of as-needed albuterol-budesonide versus albuterol in patients with asthma aged 12 to <18 years: design of the randomised, double-blind, parallel-group phase IIIb ACADIA trial.BMJ open respiratory research · 2026Article
- Bayesian dynamic borrowing in group-sequential design for medical device studies.BMC medical research methodology · 2025Article
- Augmenting external control arms using Bayesian borrowing: a case study in first-line non-small cell lung cancer.Journal of comparative effectiveness research · 2024Article
- Dapagliflozin Utilization in Chronic Kidney Disease and Its Real-World Effectiveness Among Patients with Lower Levels of Albuminuria in the USA and Japan.Advances in therapy · 2024Article
- Using Bayesian Dynamic Borrowing to Maximize the Use of Existing Data: A Case-Study.Therapeutic innovation & regulatory science · 2024Article
- Iptacopan in Idiopathic Immune Complex-Mediated Membranoproliferative Glomerulonephritis: Protocol of the APPARENT Multicenter, Randomized Phase 3 Study.Kidney international reports · 2024Article
- Power priors for replication studies.Test (Madrid, Spain) · 2024Article
- Pharmacometrics enhanced Bayesian borrowing approach to improve clinical trial efficiency: Case of empagliflozin in type 2 diabetes.CPT: pharmacometrics & systems pharmacology · 2023Article
- Statistical Considerations on the Use of RWD/RWE for Oncology Drug Approvals: Overview and Lessons Learned.Therapeutic innovation & regulatory science · 2023Review
- Simulation study for evaluating an adaptive-randomisation Bayesian hybrid trial design with enrichment.Contemporary clinical trials communications · 2023Article
- Article
- Evaluating treatments in rare indications warrants a Bayesian approach.Frontiers in pharmacology · 2023Article
- Reverse-Bayes methods for evidence assessment and research synthesis.Research synthesis methods · 2022Review
- Frequentist rules for regulatory approval of subgroups in phase III trials: A fresh look at an old problem.Statistical methods in medical research · 2021Article
- Assessing efficacy in important subgroups in confirmatory trials: An example using Bayesian dynamic borrowing.Pharmaceutical statistics · 2021Article
- Bayesian Solutions for Assessing Differential Effects in Biomarker Positive and Negative Subgroups.Pharmaceutical statisticsArticle
Corrections and comments
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Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Assessment of efficacy in important subgroups - such as those defined by sex, age, race and region - in confirmatory trials is typically performed using separate analysis of the specific subgroup. This ignores relevant information from the complementary subgroup. Bayesian dynamic borrowing uses an informative prior based on analysis of the complementary subgroup and a weak prior distribution centred on a mean of zero to construct a robust mixture prior. This combination of priors allows for dynamic borrowing of prior information; the analysis learns how much of the complementary subgroup prior information to borrow based on the consistency between the subgroup of interest and the complementary subgroup. A tipping point analysis can be carried out to identify how much prior weight needs to be placed on the complementary subgroup component of the robust mixture prior to establish efficacy in the subgroup of interest. An attractive feature of the tipping point analysis is that it enables the evidence from the source subgroup, the evidence from the target subgroup, and the combined evidence to be displayed alongside each other. This method is illustrated with an example trial in severe asthma where efficacy in the adolescent subgroup was assessed using a mixture prior combining an informative prior from the adult data in the same trial with a non-informative prior.
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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.