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Linearly divergent treatment effects in clinical trials with repeated measures: efficient analysis using summary statistics
Author(s) -
Frison Lars J.,
Pocock Stuart J.
Publication year - 1997
Publication title -
statistics in medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.996
H-Index - 183
eISSN - 1097-0258
pISSN - 0277-6715
DOI - 10.1002/(sici)1097-0258(19971230)16:24<2855::aid-sim749>3.0.co;2-y
Subject(s) - statistics , repeated measures design , clinical trial , medical statistics , econometrics , computer science , medicine , mathematics
In many randomized clinical trials with repeated measures of a response variable one anticipates a linear divergence over time in the difference between treatments. This paper explores how to make an efficient choice of analysis based on individual patient summary statistics. With the objective of estimating the mean rate of treatment divergence the simplest choice of summary statistic is the regression coefficient of response on time for each subject (SLOPE). The gains in statistical efficiency imposed by adjusting for the observed pre‐treatment levels, or even better the estimated intercepts, are clarified. In the process, we develop the optimal linear summary statistic for any repeated measures design with assumed known covariance structure and shape of true mean treatment difference over time. Statistical power considerations are explored and an example from an asthma trial is used to illustrate the main points. © 1997 John Wiley & Sons, Ltd.