Evidence map›Paper›PMID 38594809›Full record

ArticleStatistics in medicine2024

A Bayesian platform trial design with hybrid control based on multisource exchangeability modelling.

Wei Wei, Ondrej Blaha, Denise Esserman, Daniel Zelterman, Michael Kane, Rachael Liu, Jianchang Lin

Abstract read
In one paragraph

Article in Statistics in medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

2 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

7 authors.

Wei WeiDepartment of Biostatistics, Yale School of Public Health, New Haven, Connecticut, USA.ORCID 0000-0003-1263-5620
Ondrej BlahaDepartment of Biostatistics, Yale School of Public Health, New Haven, Connecticut, USA.ORCID 0000-0003-3665-3276
Denise EssermanDepartment of Biostatistics, Yale School of Public Health, New Haven, Connecticut, USA.ORCID 0000-0003-1326-9618
Daniel ZeltermanDepartment of Biostatistics, Yale School of Public Health, New Haven, Connecticut, USA.
Michael KaneDepartment of Biostatistics, Yale School of Public Health, New Haven, Connecticut, USA.ORCID 0000-0003-1899-6662
Rachael LiuStatistical & Quantitative Sciences, Takeda Pharmaceuticals, Cambridge, Massachusetts, USA.ORCID 0000-0001-6033-4510
Jianchang LinStatistical & Quantitative Sciences, Takeda Pharmaceuticals, Cambridge, Massachusetts, USA.ORCID 0000-0002-9123-0690

Funding

Yale Clinical and Translational Science Award (U Component)UL1TR001863 · NCATS · YALE UNIVERSITY · PI John H. Krystal, LUCILA OHNO-MACHADO · 2016 to 2026
$102.9M
Yale Pathology Tissue Services Shared ResourceP30CA016359 · NCI · YALE UNIVERSITY · PI Eric P. Winer · 1985 to 2026
$85.0M
Yale Head and Neck Cancer SPORE: Overcoming Treatment Resistance in Head and Neck CancerP50DE030707 · NIDCR · YALE UNIVERSITY · PI BURTNESS, BARBARA · 2020 to 2024
$11.8M
NCATS NIH HHS UL1 TR001863NCATS NIH HHS UL1TR001863NCI NIH HHS P30 CA016359NIDCR NIH HHS P50 DE030707NIH HHS P30-CA16359NIH HHS P50-DE030707
6 · The paper itself

Abstract

Enrolling patients to the standard of care (SOC) arm in randomized clinical trials, especially for rare diseases, can be very challenging due to the lack of resources, restricted patient population availability, and ethical considerations. As the therapeutic effect for the SOC is often well documented in historical trials, we propose a Bayesian platform trial design with hybrid control based on the multisource exchangeability modelling (MEM) framework to harness historical control data. The MEM approach provides a computationally efficient method to formally evaluate the exchangeability of study outcomes between different data sources and allows us to make better informed data borrowing decisions based on the exchangeability between historical and concurrent data. We conduct extensive simulation studies to evaluate the proposed hybrid design. We demonstrate the proposed design leads to significant sample size reduction for the internal control arm and borrows more information compared to competing Bayesian approaches when historical and internal data are compatible.

Indexed as

Bayes TheoremComputer SimulationModels, StatisticalRandomized Controlled Trials as TopicAdaptive Clinical Trials as TopicHumansResearch DesignSample SizeBayesian adaptive designshistorical datainformative priorplatform trialrare disease

Identifiers

PMID38594809
PMCPMC11325877

What OpenQuestion holds

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Registered trials

None linked

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.