Evidence map›Paper›PMID 31088354›Full record

ArticleBMC medical research methodology2019

Using Bayesian adaptive designs to improve phase III trials: a respiratory care example.

Elizabeth G Ryan, Julie Bruce, Andrew J Metcalfe, Nigel Stallard, Sarah E Lamb, Kert Viele, Duncan Young, Simon Gates

Open access · goldAbstract read
In one paragraph

Article in BMC medical research methodology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.

0numbers the graph read from it
0cells of the map it votes in
22citing papers in PubMed
6.0field-weighted citation impact, top 3% of its field
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

22 citing papers in PubMed, 43 citations in OpenAlex.

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  19. Current status of Bayesian clinical trials for oncology, 2020.Contemporary clinical trials communications · 2020
    Review
  20. Will Evidence-based Medicine Survive the COVID-19 Pandemic?Annals of the American Thoracic Society · 2020
    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

8 authors at 5 institutions in 2 countries.

Elizabeth G RyanWarwick Clinical Trials Unit, Warwick Medical School, University of Warwick, Coventry, CV4 7AL, UK. E.G.Ryan@bham.ac.uk.ORCID 0000-0001-9367-4204
Julie BruceWarwick Clinical Trials Unit, Warwick Medical School, University of Warwick, Coventry, CV4 7AL, UK.
Andrew J MetcalfeWarwick Clinical Trials Unit, Warwick Medical School, University of Warwick, Coventry, CV4 7AL, UK.
Nigel StallardStatistics and Epidemiology, Division of Health Sciences, Warwick Medical School, University of Warwick, Coventry, UK.
Sarah E LambWarwick Clinical Trials Unit, Warwick Medical School, University of Warwick, Coventry, CV4 7AL, UK.
Kert VieleBerry Consultants, Austin, TX, USA.
Duncan YoungNuffield Department of Clinical Neurosciences, University of Oxford, John Radcliffe Hospital, Oxford, UK.
Simon GatesWarwick Clinical Trials Unit, Warwick Medical School, University of Warwick, Coventry, CV4 7AL, UK.
University of Warwick · GBBerry & Associates (United States) · USJohn Radcliffe Hospital · GBNuffield Orthopaedic Centre · GBUniversity Hospital Coventry · GB

Funding

Cancer Research UK 25354Medical Research Council MR/N028287/1
6 · The paper itself

Abstract

backgroundBayesian adaptive designs can improve the efficiency of trials, and lead to trials that can produce high quality evidence more quickly, with fewer patients and lower costs than traditional methods. The aim of this work was to determine how Bayesian adaptive designs can be constructed for phase III clinical trials in critical care, and to assess the influence that Bayesian designs would have on trial efficiency and study results.

methodsWe re-designed the High Frequency OSCillation in Acute Respiratory distress syndrome (OSCAR) trial using Bayesian adaptive design methods, to allow for the possibility of early stopping for success or futility. We constructed several alternative designs and studied their operating characteristics via simulation. We then performed virtual re-executions by applying the Bayesian adaptive designs using the OSCAR data to demonstrate the practical applicability of the designs.

resultsWe constructed five alternative Bayesian adaptive designs and identified a preferred design based on the simulated operating characteristics, which had similar power to the original design but recruited fewer patients on average. The virtual re-executions showed the Bayesian sequential approach and original OSCAR trial yielded similar trial conclusions. However, using a Bayesian sequential design could have led to a reduced sample size and earlier completion of the trial.

conclusionsUsing the OSCAR trial as an example, this case study found that Bayesian adaptive designs can be constructed for phase III critical care trials. If the OSCAR trial had been run using one of the proposed Bayesian adaptive designs, it would have terminated at a smaller sample size with fewer deaths in the trial, whilst reaching the same conclusions. We recommend the wider use of Bayesian adaptive approaches in phase III clinical trials.

trial registrationOSCAR Trial registration ISRCTN, ISRCTN10416500 . Retrospectively registered 13 June 2007.

Indexed as

Research DesignBayes TheoremClinical Trials, Phase III as TopicComputer SimulationCritical CareHumansRandomized Controlled Trials as TopicRespiratory Distress SyndromeSample SizeBayesian sequential designCritical careInterim analysesRandomised controlled trials

Identifiers

PMID31088354
PMCPMC6515675
OpenAlexW2946827877

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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.