Composite endpoints, including patient reported outcomes, in rare diseases.

Johan VerbeeckMaya DiraniJohann W BauerRalf-Dieter HilgersGeert MolenberghsRima Nabbout

Published in: Orphanet journal of rare diseases (2023)

Generalized pairwise comparisons is a promising statistical methodology for evaluating any type of composite endpoints in rare disease trials and may allow a better evaluation of therapy efficacy including patients reported outcomes in addition to outcomes related to the diseases signs and symptoms.

Keyphrases

patient reported outcomes
end stage renal disease
ejection fraction
newly diagnosed
peritoneal dialysis
stem cells
type diabetes
metabolic syndrome
skeletal muscle
adipose tissue
bone marrow
physical activity
insulin resistance
cell therapy