Sample Size in Obesity Trials: Patient Perspective Versus Current Practice

David B. Allison, Mai A. Elobeid, Mark B. Cope, David W. Brock, Myles S. Faith, Stephanie Vander Veur, Robert Berkowitz, Gary Cutter, Theresa McVie, Kishore M. Gadde and Gary D. Foster
Additional contact information
David B. Allison: Department of Nutrition Sciences and the Clinical Nutrition Research Center, University of Alabama at Birmingham, dallison@uab.edu
Mai A. Elobeid: Department of Biostatistics, University of Alabama at Birmingham
Mark B. Cope: Department of Pharmacology and Toxicology, University of Alabama at Birmingham
David W. Brock: Department of Biostatistics, , University of Alabama at Birmingham
Myles S. Faith: Department of Pharmacology and Toxicology University of Alabama at Birmingham
Stephanie Vander Veur: Center for Weight and Eating Disorders, University of Pennsylvania, School of Medicine, Philadelphia
Robert Berkowitz: Department of Pharmacology and Toxicology, University of Alabama at Birmingham
Gary Cutter: Department of Biostatistics, University of Alabama at Birmingham
Theresa McVie: Department of Biostatistics, University of Alabama at Birmingham
Kishore M. Gadde: Center for Obesity Research and Education, School of Medicine, Temple University, Philadelphia, Pennsylvania
Gary D. Foster: Center for Weight and Eating Disorders, University of Pennsylvania, School of Medicine, Philadelphia

Medical Decision Making, 2010, vol. 30, issue 1, 68-75

Abstract: Objective. To evaluate patient opinions on acceptable risks in exchange for a given degree of weight loss and their implications for sample size determination in obesity randomized clinical trials (RCTs). Design. Survey of patients entering RCTs for weight loss in a university-based clinical research setting and power calculations based on their responses. Participants. Men (n = 8) and women (n = 66) between 24 and 73 years of age with body mass indices ranging from 26.8 to 40.5 kg/m 2 . Measurements. Survey responses to questions assessing the added risk of serious adverse events (SAEs) or death one is willing to assume for a given degree of weight loss. Results. For 5% and 10% weight loss against risk for death per se, the mean acceptable risk tended to be about 3.5%, but the median (0.00) and mode (0.00) suggested that for most individuals, only a risk of ≤ 1% would be acceptable. Figures, estimated dropout rates, and base rates of SAEs (including deaths) from recent obesity trials indicate that 1-year 2-group obesity RCTs would need tens of thousands of participants per group to have 80% power to detect risks that are meaningful to patients at the 2-tailed 0.05 α level. Conclusion. Patient education is needed to explain which risks are realistically detectable in RCTs so that patients may provide truly informed consent, or RCT standards should be modified to meet patients’ implicit expectations.

Keywords: obesity; randomized clinical trials; risk; weight loss. (Med Decis Making 2010; 30:68—75) (search for similar items in EconPapers)
Date: 2010
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Persistent link: https://EconPapers.repec.org/RePEc:sae:medema:v:30:y:2010:i:1:p:68-75

DOI: 10.1177/0272989X09340583

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