Evidence map›Paper›PMID 34755388›Full record

Trial reportStatistics in medicine2022

A group-sequential randomized trial design utilizing supplemental trial data.

Ales Kotalik, David M Vock, Brian P Hobbs, Joseph S Koopmeiners

Abstract readRandomized Controlled Trial
In one paragraph

Trial report in Statistics in medicine, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
–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

4 citing papers in PubMed.

  1. Article
  2. Article
  3. Article
  4. 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

4 authors.

Ales KotalikBiometrics, Late-stage Development, Respiratory and Immunology (R&I), AstraZeneca BioPharmaceuticals R&D, Gaithersburg, Maryland, USA.ORCID 0000-0003-2981-3884
David M VockDivision of Biostatistics, School of Public Health, University of Minnesota, Minneapolis, Minnesota, USA.
Brian P HobbsDell Medical School, The University of Texas at Austin, Austin, Texas, USA.ORCID 0000-0003-2189-5846
Joseph S KoopmeinersDivision of Biostatistics, School of Public Health, University of Minnesota, Minneapolis, Minnesota, USA.ORCID 0000-0001-6147-3168

Funding

Women's CancerP30CA077598 · NCI · UNIVERSITY OF MINNESOTA TWIN CITIES · PI Timothy C. Hallstrom · 1998 to 2026
$100.4M
VERY LOW NICOTINE CIGARETTES IN SMOKERS WITH SCHIZOPHRENIAU54DA031659 · NIDA · WAKE FOREST UNIVERSITY HEALTH SCIENCES · PI DONNY, ERIC CHRISTIAN · 2011 to 2022
$64.7M
Innovative Statistical Methods for Evaluating the Impact of Tobacco Product StandardsR01DA046320 · NIDA · UNIVERSITY OF MINNESOTA · PI Joseph S. Koopmeiners, David M. Vock · 2018 to 2026
$2.4M
Training in Biostatistics for Heart, Lung and Blood Disease ResearchT32HL129956 · NHLBI · UNIVERSITY OF MINNESOTA · PI NEATON, JAMES DENNIS · 2016 to 2020
$1.1M
SMART-ACT: Spatial Methodologic Approaches for Risk Assessment and Therapeutic Adaptation in Cancer TreatmentR01CA214825 · NCI · UNIVERSITY OF ILLINOIS AT CHICAGO · PI CANAHUATE, GUADALUPE, FULLER, CLIFTON DAVID · 2017 to 2019
$1.1M
QuBBD: Precision E –Radiomics for Dynamic Big Head & Neck Cancer Data R01CA225190 · NCI · UNIVERSITY OF ILLINOIS AT CHICAGO · PI CANAHUATE, GUADALUPE, FULLER, CLIFTON DAVID · 2017 to 2019
$776k
Innovative Statistical Methods for Detecting and Accounting for Non-Compliance in Randomized Trials of Very Low Nicotine Content CigarettesR03DA041870 · NIDA · UNIVERSITY OF MINNESOTA · PI KOOPMEINERS, JOSEPH S., VOCK, DAVID M. · 2016 to 2017
$229k
NCI NIH HHS P30 CA077598NCI NIH HHS R01-CA214824NCI NIH HHS R01 CA214825NCI NIH HHS R01 CA225190NHLBI NIH HHS T32 HL129956NIDA NIH HHS R01 DA046320NIDA NIH HHS R03 DA041870NIDA NIH HHS U54 DA031659
6 · The paper itself

Abstract

Definitive clinical trials are resource intensive, often requiring a large number of participants over several years. One approach to improve the efficiency of clinical trials is to incorporate historical information into the primary trial analysis. This approach has tremendous potential in the areas of pediatric or rare disease trials, where achieving reasonable power is difficult. In this article, we introduce a novel Bayesian group-sequential trial design based on Multisource Exchangeability Models, which allows for dynamic borrowing of historical information at the interim analyses. Our approach achieves synergy between group sequential and adaptive borrowing methodology to attain improved power and reduced sample size. We explore the frequentist operating characteristics of our design through simulation and compare our method to a traditional group-sequential design. Our method achieves earlier stopping of the primary study while increasing power under the alternative hypothesis but has a potential for type I error inflation under some null scenarios. We discuss the issues of decision boundary determination, power and sample size calculations, and the issue of information accrual. We present our method for a continuous and binary outcome, as well as in a linear regression setting.

Indexed as

Research DesignBayes TheoremChildComputer SimulationHumansSample Sizeborrowing strengthdata aggregationexchangeabilitygroup-sequential trial

Identifiers

PMID34755388
PMCPMC8795487

What OpenQuestion holds

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Read underepoch 390

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.