Statistics in medicine
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Statistics in medicine · Jul 1996
Comparative StudyA method for determining the size of internal pilot studies.
The assessment of sample size in clinical trials comparing population means requires a variance estimate of the main efficacy variable. When this variance estimate has a low precision, it may be appropriate to use the data from the first patients entered in the trial ('internal pilot study') to estimate the sample size. We suggest a method for determining the size of internal pilot studies, which aims at ensuring that this size is as large as possible, but not larger than 'the optimal size' of the planned study. Advantages and limitations of the method are discussed.
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Statistics in medicine · Jul 1996
Maximum likelihood estimation of the kappa coefficient from bivariate logistic regression.
We propose a maximum likelihood estimator (MLE) of the kappa coefficient from a 2 x 2 table when the binary ratings depend on patient and/or clinician effects. We achieve this by expressing the logit of the probability of positive rating as a linear function of the subject-specific and the rater-specific covariates. We investigate the bias and variance of the MLE in small and moderate size samples through Monte Carlo simulation and we provide the sample size calculation to detect departure from the null hypothesis H0: kappa = kappa 0 in the direction of H1: kappa > kappa 0.