Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases

Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases

Abstract Background Orphan drug designations are a useful proxy to investigate trends in rare disease drug development. Drug developers must receive a designation before they are eligible for the economic incentives of the Orphan Drug Act in the United States. We created a database of all orphan dru...

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Bibliographic Details
Main Authors: Kathleen L. Miller, Lewis J. Fermaglich, Janet Maynard
Format: Article
Language:English
Published: BMC 2021-06-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Oncology
Orphan drugs
Orphan Drug Act
Orphan drug designation
Pediatric
Rare disease
Online Access:https://doi.org/10.1186/s13023-021-01901-6

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