Tutorial in Biostatistics
Designing a pilot sequential multiple assignment randomized trial for developing an adaptive treatment strategy
Article first published online: 22 MAR 2012
Copyright © 2012 John Wiley & Sons, Ltd.
Statistics in Medicine
Volume 31, Issue 17, pages 1887–1902, 30 July 2012
How to Cite
Almirall, D., Compton, S. N., Gunlicks-Stoessel, M., Duan, N. and Murphy, S. A. (2012), Designing a pilot sequential multiple assignment randomized trial for developing an adaptive treatment strategy. Statist. Med., 31: 1887–1902. doi: 10.1002/sim.4512
- Issue published online: 16 JUL 2012
- Article first published online: 22 MAR 2012
- Manuscript Accepted: 9 DEC 2011
- Manuscript Revised: 3 NOV 2011
- Manuscript Received: 6 APR 2011
- dynamic treatment regimes
There is growing interest in how best to adapt and readapt treatments to individuals to maximize clinical benefit. In response, adaptive treatment strategies (ATS), which operationalize adaptive, sequential clinical decision making, have been developed. From a patient's perspective an ATS is a sequence of treatments, each individualized to the patient's evolving health status. From a clinician's perspective, an ATS is a sequence of decision rules that input the patient's current health status and output the next recommended treatment. Sequential multiple assignment randomized trials (SMART) have been developed to address the sequencing questions that arise in the development of ATSs, but SMARTs are relatively new in clinical research. This article provides an introduction to ATSs and SMART designs. This article also discusses the design of SMART pilot studies to address feasibility concerns, and to prepare investigators for a full-scale SMART. We consider an example SMART for the development of an ATS in the treatment of pediatric generalized anxiety disorders. Using the example SMART, we identify and discuss design issues unique to SMARTs that are best addressed in an external pilot study prior to the full-scale SMART. We also address the question of how many participants are needed in a SMART pilot study. A properly executed pilot study can be used to effectively address concerns about acceptability and feasibility in preparation for (that is, prior to) executing a full-scale SMART. Copyright © 2012 John Wiley & Sons, Ltd.