Randomized reverse marker strategy design for prospective biomarker validation. (18th March 2014)

• Record Type: Journal Article
• Authors: Eng, Kevin H.
• Is Part Of: Statistics in medicine. Volume 33:Number 18(2014)
• Page Start: 3089
• Page End: 3099
• Publication Date: 2014-03-18
• Subjects: Medical statistics -- Periodicals; Statistique médicale -- Périodiques; Statistiques médicales -- Périodiques; 610.727
• DOI: 10.1002/sim.6146
• Languages: English
• ISSNs: 0277-6715

Abstract

<abstract abstract-type="main" id="sim6146-abs-0001"> <title> <x xml:space="preserve">Abstract</x> </title> <p id="sim6146-para-0002">We describe a novel study design for validating marker‐based treatment strategies meant to select among possible therapeutic options using a biologic marker. Studying existing designs in realistic scenarios, we demonstrate that this design is more than four times more efficient for testing the interaction between a marker and its intended treatment. Our analysis employs a simple parametric framework that uncovers systematic biases in currently proposed designs and suggests how they may be accommodated or enumerated. In the context of markers for choosing a treatment for recurrent ovarian cancer, our proposal requires sample sizes on the order of recently completed phases II and III studies making validation studies for this clinical decision scenario viable. © 2014 The Authors. Statistics in Medicine Published by John Wiley &amp; Sons Ltd.</p> </abstract>