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Statistical documentation for multi-disease, multi-domain platform trials: our experience with the Staphylococcus
Robert K Mahar1,2,3, Anna McGlothlin4, Michael Dymock5,6
1Centre for Epidemiology, Melbourne School of Population and Global Health, University of Melbourne, Parkville, Australia. robert.mahar@unimelb.edu.au.
This study presents a framework for operationalizing platform trials, using the Staphylococcus aureus Network Adaptive Platform (SNAP) trial as a template. It offers guidance on statistical documentation and governance for adaptive trial designs.
Area of Science:
- Clinical Trials
- Biostatistics
- Epidemiology
Background:
- Platform trials are increasingly used but lack standardized operational guidelines.
- The Staphylococcus aureus Network Adaptive Platform (SNAP) trial exemplifies a complex adaptive design.
Purpose of the Study:
- To provide an informal template for platform trial statistical documentation and processes.
- To guide researchers and reviewers in operationalizing adaptive platform trials.
Main Methods:
- Summarizing key statistical documents and governance structures from the SNAP trial.
- Outlining the roles of core protocol, statistical appendices, simulation reports, and DSMC reports.
- Describing a transparent governance model with separate blinded and unblinded statistical teams.
Main Results:
- The SNAP trial's documentation and governance provide a practical model for platform trials.
- A comprehensive set of statistical documents and transparent oversight are crucial for adaptive trial success.
Conclusions:
- The SNAP trial's operational framework can inform future platform trial development.
- Establishing clear guidelines and consensus for platform trial statistical documents is needed.
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