A systematic review of real-world evidence (RWE) supportive of new drug and biologic license application approvals in rare diseases

A systematic review of real-world evidence (RWE) supportive of new drug and biologic license application approvals in rare diseases

Abstract Background Real-world evidence (RWE) generated using real-world data (RWD) presents the potential to contextualize and/or supplement traditional clinical trials for regulatory approval of rare diseases (RDs). This systematic review evaluated the use of RWD for non-oncologic RD therapies wit...

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Bibliographic Details
Main Authors: Shailja Vaghela, Kaniz Afroz Tanni, Geetanjoli Banerjee, Vanja Sikirica
Format: Article
Language:English
Published: BMC 2024-03-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Biologic license application
New drug application
Orphan drugs
Rare diseases
Real-world data
Real-world evidence
Online Access:https://doi.org/10.1186/s13023-024-03111-2

Internet

https://doi.org/10.1186/s13023-024-03111-2

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