Istore: a Project on Innovative Statistical Methodologies to Improve Rare Diseases Clinical Trials in Limited Populations

Stefanie Schoenen,Johan Verbeeck,Lukas Koletzko,Isabella Brambilla,Mathieu Kuchenbuch,Maya Dirani,Georg Zimmermann,Holger Dette,Ralf-Dieter Hilgers,Geert Molenberghs,Rima Nabbout

ORPHANET JOURNAL OF RARE DISEASES（2024）

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Key words

Bias assessment with multiple endpoints,Finite populations,Multiple endpoints,Natural history modelling,Rare disease clinical trials,Similarity of subgroups

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