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Ordinal data in a repeated measures design of a crossover study for rare diseases usually do not allow for the use of standard parametric methods, and hence, nonparametric methods should be considered instead. However, only limited simulation studies in settings with small sample sizes exist. Therefore, starting from an Epidermolysis Bullosa simplex trial with the above‐mentioned design, a rank‐based...
To optimize the use of data from a small number of subjects in rare disease trials, an at first sight advantageous design is the repeated measures cross‐over design. However, it is unclear how these within‐treatment period and within‐subject clustered data are best analyzed in small‐sample trials. In a real‐data simulation study based upon a recent epidermolysis bullosa simplex trial using this design,...