Table 2 Original EBS trial data analysis of the composite blister and QoL outcomes with the prioritized, non-prioritized and matched GPC. The blister outcome is treated as a standardized difference of the number of blisters \(\left(\frac{\text{N}\text{u}\text{m}\text{b}\text{e}\text{r } \text{o}\text{f } \text{b}\text{l}\text{i}\text{s}\text{t}\text{e}\text{r}\text{s } \text{a}\text{t } \text{b}\text{a}\text{s}\text{e}\text{l}\text{i}\text{n}\text{e}-\text{N}\text{u}\text{m}\text{b}\text{e}\text{r } \text{o}\text{f } \text{b}\text{l}\text{i}\text{s}\text{t}\text{e}\text{r}\text{s } \text{a}\text{t } \text{w}\text{e}\text{e}\text{k } 4}{\text{N}\text{u}\text{m}\text{b}\text{e}\text{r } \text{o}\text{f } \text{b}\text{l}\text{i}\text{s}\text{t}\text{e}\text{r}\text{s } \text{a}\text{t } \text{b}\text{a}\text{s}\text{e}\text{l}\text{i}\text{n}\text{e}}\right)\). NTB = Net Treatment Benefit, CI = Confidence Interval

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