Designing a pilot sequential multiple assignment randomized trial for developing an adaptive treatment strategy.

Abstract:

: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.

journal_name

Stat Med

journal_title

Statistics in medicine

authors

Almirall D,Compton SN,Gunlicks-Stoessel M,Duan N,Murphy SA

doi

10.1002/sim.4512

subject

Has Abstract

pub_date

2012-07-30 00:00:00

pages

1887-902

issue

17

eissn

0277-6715

issn

1097-0258

journal_volume

31

pub_type

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