Adaptive trial designs in diagnostic accuracy research

Antonia Zapf, Maria Stark, Oke Gerke, Christoph Ehret, Norbert Benda, Patrick Bossuyt, Jon Deeks, Johannes Reitsma, Todd Alonzo, Tim Friede

Publikation: Bidrag til tidsskriftTidsskriftartikelForskningpeer review

Resumé

The aim of diagnostic accuracy studies is to evaluate how accurately a diagnostic test can distinguish diseased from nondiseased individuals. Depending on the research question, different study designs and accuracy measures are appropriate. As the prior knowledge in the planning phase is often very limited, modifications of design aspects such as the sample size during the ongoing trial could increase the efficiency of diagnostic trials. In intervention studies, group sequential and adaptive designs are well established. Such designs are characterized by preplanned interim analyses, giving the opportunity to stop early for efficacy or futility or to modify elements of the study design. In contrast, in diagnostic accuracy studies, such flexible designs are less common, even if they are as important as for intervention studies. However, diagnostic accuracy studies have specific features, which may require adaptations of the statistical methods or may lead to specific advantages or limitations of sequential and adaptive designs. In this article, we summarize the current status of methodological research and applications of flexible designs in diagnostic accuracy research. Furthermore, we indicate and advocate future development of adaptive design methodology and their use in diagnostic accuracy trials from an interdisciplinary viewpoint. The term "interdisciplinary viewpoint" describes the collaboration of experts of the academic and nonacademic research.

OriginalsprogEngelsk
TidsskriftStatistics in Medicine
ISSN0277-6715
DOI
StatusE-pub ahead of print - 27. nov. 2019

Fingeraftryk

Diagnostic Accuracy
Adaptive Design
Research
Medical Futility
Group Sequential Design
Sequential Design
Routine Diagnostic Tests
Diagnostic Tests
Sample Size
Prior Knowledge
Statistical method
Design Methodology
Efficacy
Diagnostics
Planning
Design
Evaluate
Term

Citer dette

Zapf, A., Stark, M., Gerke, O., Ehret, C., Benda, N., Bossuyt, P., ... Friede, T. (2019). Adaptive trial designs in diagnostic accuracy research. Statistics in Medicine. https://doi.org/10.1002/sim.8430
Zapf, Antonia ; Stark, Maria ; Gerke, Oke ; Ehret, Christoph ; Benda, Norbert ; Bossuyt, Patrick ; Deeks, Jon ; Reitsma, Johannes ; Alonzo, Todd ; Friede, Tim. / Adaptive trial designs in diagnostic accuracy research. I: Statistics in Medicine. 2019.
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Zapf, A, Stark, M, Gerke, O, Ehret, C, Benda, N, Bossuyt, P, Deeks, J, Reitsma, J, Alonzo, T & Friede, T 2019, 'Adaptive trial designs in diagnostic accuracy research', Statistics in Medicine. https://doi.org/10.1002/sim.8430

Adaptive trial designs in diagnostic accuracy research. / Zapf, Antonia; Stark, Maria; Gerke, Oke; Ehret, Christoph; Benda, Norbert; Bossuyt, Patrick; Deeks, Jon; Reitsma, Johannes; Alonzo, Todd; Friede, Tim.

I: Statistics in Medicine, 27.11.2019.

Publikation: Bidrag til tidsskriftTidsskriftartikelForskningpeer review

TY - JOUR

T1 - Adaptive trial designs in diagnostic accuracy research

AU - Zapf, Antonia

AU - Stark, Maria

AU - Gerke, Oke

AU - Ehret, Christoph

AU - Benda, Norbert

AU - Bossuyt, Patrick

AU - Deeks, Jon

AU - Reitsma, Johannes

AU - Alonzo, Todd

AU - Friede, Tim

N1 - © 2019 The Authors. Statistics in Medicine Published by John Wiley & Sons Ltd.

PY - 2019/11/27

Y1 - 2019/11/27

N2 - The aim of diagnostic accuracy studies is to evaluate how accurately a diagnostic test can distinguish diseased from nondiseased individuals. Depending on the research question, different study designs and accuracy measures are appropriate. As the prior knowledge in the planning phase is often very limited, modifications of design aspects such as the sample size during the ongoing trial could increase the efficiency of diagnostic trials. In intervention studies, group sequential and adaptive designs are well established. Such designs are characterized by preplanned interim analyses, giving the opportunity to stop early for efficacy or futility or to modify elements of the study design. In contrast, in diagnostic accuracy studies, such flexible designs are less common, even if they are as important as for intervention studies. However, diagnostic accuracy studies have specific features, which may require adaptations of the statistical methods or may lead to specific advantages or limitations of sequential and adaptive designs. In this article, we summarize the current status of methodological research and applications of flexible designs in diagnostic accuracy research. Furthermore, we indicate and advocate future development of adaptive design methodology and their use in diagnostic accuracy trials from an interdisciplinary viewpoint. The term "interdisciplinary viewpoint" describes the collaboration of experts of the academic and nonacademic research.

AB - The aim of diagnostic accuracy studies is to evaluate how accurately a diagnostic test can distinguish diseased from nondiseased individuals. Depending on the research question, different study designs and accuracy measures are appropriate. As the prior knowledge in the planning phase is often very limited, modifications of design aspects such as the sample size during the ongoing trial could increase the efficiency of diagnostic trials. In intervention studies, group sequential and adaptive designs are well established. Such designs are characterized by preplanned interim analyses, giving the opportunity to stop early for efficacy or futility or to modify elements of the study design. In contrast, in diagnostic accuracy studies, such flexible designs are less common, even if they are as important as for intervention studies. However, diagnostic accuracy studies have specific features, which may require adaptations of the statistical methods or may lead to specific advantages or limitations of sequential and adaptive designs. In this article, we summarize the current status of methodological research and applications of flexible designs in diagnostic accuracy research. Furthermore, we indicate and advocate future development of adaptive design methodology and their use in diagnostic accuracy trials from an interdisciplinary viewpoint. The term "interdisciplinary viewpoint" describes the collaboration of experts of the academic and nonacademic research.

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