Adaptive trial designs in diagnostic accuracy research

Research output: Contribution to journalArticle

Authors

  • A Zapf
  • M Stark
  • O Gerke
  • C Ehret
  • N Benda
  • Patrick M. Bossuyt
  • J Reitsma
  • T Alonzo
  • T Friede

Colleges, School and Institutes

External organisations

  • Academic Medical Centre, University of Amsterdam
  • University Medical Centre Hamburg
  • Odense University Hospital, Odense, Denmark.
  • ROCHE DIAGNOSTICS GMBH
  • Federal Institute for Drugs and Medical Devices
  • University Medical Center Göttingen
  • University Medical Center Utrecht
  • University of Southern California Keck School of Medicine

Abstract

The aim of diagnostic accuracy studies is to evaluate how accurately a diagnostic test can distinguish diseased from non-diseased 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 pre-planned 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 non-academic research.

Details

Original languageEnglish
Pages (from-to)1-18
Number of pages18
JournalStatistics in Medicine
Publication statusAccepted/In press - 26 Oct 2019

Keywords

  • Diagnostic studies, diagnostic accuracy, adaptive designs, group sequential designs, sample size re-estimation