Adaptive trial designs in diagnostic accuracy research.
Antonia Zapf1, Maria Stark1, Oke Gerke2
1Department of Medical Biometry and Epidemiology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
Statistics in Medicine
|November 28, 2019
Summary
Flexible designs, like those used in intervention studies, can improve the efficiency of diagnostic accuracy trials by allowing for early stopping or modifications. This article reviews current methods and advocates for future development in adaptive diagnostic accuracy research.
Area of Science:
- Biostatistics
- Clinical Trials Methodology
- Medical Diagnostics
Background:
- Diagnostic accuracy studies evaluate a test's ability to differentiate between diseased and non-diseased individuals.
- Flexible designs, including group sequential and adaptive designs, are common in intervention studies but less so in diagnostic accuracy research.
- Limited prior knowledge in diagnostic trials often necessitates design modifications during the study.
Purpose of the Study:
- To summarize the current status of methodological research and applications of flexible designs in diagnostic accuracy studies.
- To advocate for the future development and interdisciplinary application of adaptive design methodology in diagnostic accuracy trials.
Main Methods:
- Review of current methodological research on flexible designs in diagnostic accuracy studies.
- Discussion of specific features of diagnostic accuracy studies impacting sequential and adaptive designs.
- Exploration of interdisciplinary viewpoints on adaptive design methodology.
Main Results:
- Flexible designs offer opportunities to enhance diagnostic trial efficiency through pre-planned interim analyses.
- Adaptive designs can allow for early stopping for efficacy or futility, or modifications to study elements.
- Specific characteristics of diagnostic accuracy studies may necessitate adaptations in statistical methods for flexible designs.
Conclusions:
- Flexible and adaptive designs are crucial for improving the efficiency and rigor of diagnostic accuracy studies.
- Further interdisciplinary research and development are needed to fully leverage adaptive designs in this field.
- Adoption of flexible designs in diagnostic accuracy research aligns with established practices in intervention studies.
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