Table 1 Two-sided (one-sided) type I error and power of the single and composite GPC analyses in 5000 permuted samples of the original EBS trial. The blister outcome is treated as a standardized difference of the number of blisters \(\left(\frac{\text{N}\text{u}\text{m}\text{b}\text{e}\text{r } \text{o}\text{f } \text{b}\text{l}\text{i}\text{s}\text{t}\text{e}\text{r}\text{s } \text{a}\text{t } \text{b}\text{a}\text{s}\text{e}\text{l}\text{i}\text{n}\text{e}-\text{N}\text{u}\text{m}\text{b}\text{e}\text{r } \text{o}\text{f } \text{b}\text{l}\text{i}\text{s}\text{t}\text{e}\text{r}\text{s } \text{a}\text{t } \text{w}\text{e}\text{e}\text{k } 4}{\text{N}\text{u}\text{m}\text{b}\text{e}\text{r } \text{o}\text{f } \text{b}\text{l}\text{i}\text{s}\text{t}\text{e}\text{r}\text{s } \text{a}\text{t } \text{b}\text{a}\text{s}\text{e}\text{l}\text{i}\text{n}\text{e}}\right)\)

From: Composite endpoints, including patient reported outcomes, in rare diseases