Statistical challenges in assessing potential efficacy of complex interventions in pilot or feasibility studies

Early phase trials of complex interventions currently focus on assessing the feasibility of a large randomised control trial and on conducting pilot work. Assessing the efficacy of the proposed intervention is generally discouraged, due to concerns of underpowered hypothesis testing. In contrast, ea...

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Bibliographic Details
Published in:Statistical methods in medical research Vol. 25; no. 3; pp. 997 - 1009
Main Authors: Wilson, Duncan T, Walwyn, Rebecca Ea, Brown, Julia, Farrin, Amanda J, Brown, Sarah R
Format: Journal Article
Language:English
Published: England 01.06.2016
Subjects:
Bayes Theorem
Clinical Trials, Phase II as Topic - methods
Feasibility Studies
Humans
Pilot Projects
Research Design
Sample Size
feasibility
phase II
pilot
sample size
Complex interventions
ISSN:1477-0334
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Summary:Early phase trials of complex interventions currently focus on assessing the feasibility of a large randomised control trial and on conducting pilot work. Assessing the efficacy of the proposed intervention is generally discouraged, due to concerns of underpowered hypothesis testing. In contrast, early assessment of efficacy is common for drug therapies, where phase II trials are often used as a screening mechanism to identify promising treatments. In this paper, we outline the challenges encountered in extending ideas developed in the phase II drug trial literature to the complex intervention setting. The prevalence of multiple endpoints and clustering of outcome data are identified as important considerations, having implications for timely and robust determination of optimal trial design parameters. The potential for Bayesian methods to help to identify robust trial designs and optimal decision rules is also explored.
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ISSN:1477-0334
DOI:10.1177/0962280215589507