Important tool in our rare disease toolbox: hybrid retrospective-prospective natural history studies serve well as external comparators for rare disease studies

Important tool in our rare disease toolbox: hybrid retrospective-prospective natural history studies serve well as external comparators for rare disease studies

Natural history studies (NHS) can support regulatory decision-making at different stages of the drug product life cycle and are especially important in the context of rare diseases, which are associated with not only delayed or erroneous diagnoses but also a lack of approved treatments. Real-world e...

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Bibliographic Details
Main Authors: Chinenye Ugoji, Julien Heidt, Joan Largent, Emily Bratton, Laura Hester, Sareh Keshavarzi, Stuart Turner, Christina Mack
Format: Article
Language:English
Published: Frontiers Media S.A. 2024-12-01
Series:Frontiers in Drug Safety and Regulation
Subjects:
rare disease
natural history studies
real-world data
hybrid design
external comparators
clinical development
Online Access:https://www.frontiersin.org/articles/10.3389/fdsfr.2024.1418050/full
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