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Abstract Details

Activity Number: 49
Type: Invited
Date/Time: Sunday, July 29, 2012 : 4:00 PM to 5:50 PM
Sponsor: Statistics in Mental Health Research
Abstract - #303603
Title: From Pilot Studies to Confirmatory Studies: Confidence Intervals, Statistical Significance, and Clinical Significance
Author(s): Naihua Duan*+
Companies: Columbia University
Address: 1051 Riverside Drive, Unit 48, New York, NY, 10032,
Keywords: effect size ; feasibility ; mental health ; power analysis ; precision analysis ; psychiatry
Abstract:

Kraemer et al. (2006) and Leon et al. (2011) discussed problems in using estimated treatment effects from pilot studies in power analysis for subsequent confirmatory studies, and recommended that pilot studies be focused on other objectives such as the feasibility of recruitment, randomization, retention, assessment procedures, implementation of the novel intervention, etc. The principles in this literature leave open questions about the implementation of these principles in applications, resulting in disagreements among clinical investigators, statisticians, and grant reviewers. This paper attempts to bridge the gap between principles and implementation in this area, discussing 1) power analysis for feasibility studies; 2) pros and cons for the inclusion of a randomized comparison arm in pilot studies, and merits of unequal allocation between experimental and comparison arms; 3) consideration of confidence intervals instead of statistical significance for the treatment effect of interest, for pilot studies to inform the identification of treatments that are not promising; and 4) practical implications of incorporating clinical significance into the design of confirmatory studies.


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