Efficacy assessment in trials of complex and rare diseases: a comparison between the meta-analytic global statistical test and co-primary analysis

IntroductionChoice of the primary outcome can be problematic in 1) diseases with heterogeneous signs and symptoms, 2) trials of disease-modifying treatments (DMTs) that are expected to affect all aspects of the disease, 3) in complex and rare diseases with minimal data on clinical trial outcomes. In...

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Bibliographic Details

Published in:	Frontiers in Medical Technology
Main Authors:	Samuel P. Dickson, Abe Durrant, Caleb W. Dayley, Joshua R. Christensen, Craig H. Mallinckrodt, Suzanne B. Hendrix
Format:	Article
Language:	English
Published:	Frontiers Media S.A. 2026-06-01
Subjects:	clinical trials complex disease co-primary endpoints Global Statistical Test (GST) rare disease
Online Access:	https://www.frontiersin.org/articles/10.3389/fmedt.2026.1819463/full