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Activity Number:
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418
- SPEED: Biostatistical Methods, Application, and Education, Part 2
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Type:
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Contributed
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Date/Time:
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Tuesday, July 30, 2019 : 2:00 PM to 2:45 PM
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Sponsor:
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Mental Health Statistics Section
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Abstract #307835
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Title:
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Sample Size Calculations in Single-Case Designs
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Author(s):
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Jiabei Yang* and Christopher Schmid and Jon Steingrimsson
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Companies:
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Brown School of Public Health and Brown University and Brown University
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Keywords:
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Personalized medicine;
N-of-1 trials;
Fixed-effects analysis;
Random-effects analysis;
Power;
Precision
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Abstract:
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Randomized controlled trials are the gold standard for estimating average treatment effects in a population, but are less effective in describing treatment effect heterogeneity. Single-case designs, trials carried out in individuals, are able to estimate efficacy for individuals directly; combining single-case studies increases precision for individual as well as population average treatment effects. We derive sample size formulae according to whether the scientific interest lies in individual-specific or population average treatment effect under both fixed- and random-effects analysis, assuming various randomization schemes and correlation structure among measurements taken on individual patients. We present figures to inform designs balancing the relative effects of: 1) number of crossover periods and number of measurements taken per period in individual trials and 2) number of individuals and number of measurements per individual in a series of N-of-1 trials.
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Authors who are presenting talks have a * after their name.
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