Istore: a project on innovative statistical methodologies to improve rare diseases clinical trials in limited populations

Istore: a project on innovative statistical methodologies to improve rare diseases clinical trials in limited populations

Abstract Background The conduct of rare disease clinical trials is still hampered by methodological problems. The number of patients suffering from a rare condition is variable, but may be very small and unfortunately statistical problems for small and finite populations have received less considera...

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Bibliographic Details
Main Authors: Stefanie Schoenen, Johan Verbeeck, Lukas Koletzko, Isabella Brambilla, Mathieu Kuchenbuch, Maya Dirani, Georg Zimmermann, Holger Dette, Ralf-Dieter Hilgers, Geert Molenberghs, Rima Nabbout
Format: Article
Language:English
Published: BMC 2024-03-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Bias assessment with multiple endpoints
Finite populations
Multiple endpoints
Natural history modelling
Rare disease clinical trials
Similarity of subgroups
Online Access:https://doi.org/10.1186/s13023-024-03103-2

Internet

https://doi.org/10.1186/s13023-024-03103-2

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