Sample size considerations for n-of-1 trials

Abstract

N-of-1 trials are trials in which patients are treated with two or more treatments on multiple occasions. They can have many different purposes and can be analysed in different frameworks. In this note, five different criteria for planning sample sizes for n-of-1 trials are identified, and formulae and advice to address the associated tasks are provided. The basic design addressed is that of randomisation to treatment and control within cycles of pairs of episodes and the model assumed is that of a Normal–Normal mixture with variance components corresponding to within-cycle within-patient variation and treatment-by-patient interaction. The code to accomplish the tasks has been written in GenStat®, SAS® and R® and the application of the approaches is illustrated.