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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Autori principali:	Chinenye Ugoji, Julien Heidt, Joan Largent, Emily Bratton, Laura Hester, Sareh Keshavarzi, Stuart Turner, Christina Mack
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	Frontiers Media S.A. 2024-12-01
Serie:	Frontiers in Drug Safety and Regulation
Soggetti:	rare disease natural history studies real-world data hybrid design external comparators clinical development
Accesso online:	https://www.frontiersin.org/articles/10.3389/fdsfr.2024.1418050/full
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