Principles of Sample Size Calculation


5. What Drives a Sample Size Calculation?

5.1. The Design of the Clinical Trial

A trial with only one or several experimental treatments arms in a Phase II setting will require a different approach from a randomised comparative Phase III trial. Furthermore, the sample size needed for a study depends on the assumption of the size of the difference expected between the two treatments being studied. In a study where a large difference between the treatments is assumed, the difference should be observable in a smaller sample, whereas a larger sample size is needed to detect a small difference between two treatments. The situation becomes more complex when more than two treatment groups are planned since there is no longer one single clear alternative hypothesis. A test strategy must be defined upfront and adequate measures applied to maintain the overall type I error.