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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 indicates if a subject has a 40% reduction in blisters

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

 

Type I error

Power

 

Unmatched GPC

Single blister

0.0692 (0.0216)

0.5904 (0.7202)

Single QoL

0.0514 (0.0486)

0.8642 (0.9302)

Multi Prioritized

0.0514 (0.0510)

0.9594 (0.9812)

Multi Non-prioritized

0.0490 (0.0524)

0.9886 (0.9716)

 

Matched GPC

Single Blister

0.0348 (0.0632)

0.4751 (0.6029)

Single QoL

0.0422 (0.0610)

0.7044 (0.8650)

Multi Prioritized

0.0260 (0.0258)

0.5824 (0.8210)