Adaptive trial designs in diagnostic accuracy research
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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.
In: STAT MED, Vol. 39, No. 5, 28.02.2020, p. 591-601.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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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 - 2020/2/28
Y1 - 2020/2/28
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.
U2 - 10.1002/sim.8430
DO - 10.1002/sim.8430
M3 - SCORING: Journal article
C2 - 31773788
VL - 39
SP - 591
EP - 601
JO - STAT MED
JF - STAT MED
SN - 0277-6715
IS - 5
ER -
