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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

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 drugs desig...

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Détails bibliographiques
Publié dans:Orphanet J Rare Dis
Auteurs principaux: Miller, Kathleen L., Fermaglich, Lewis J., Maynard, Janet
Format: Artigo
Langue:Inglês
Publié: BioMed Central 2021
Sujets:
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC8191002/
https://ncbi.nlm.nih.gov/pubmed/34107994
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-021-01901-6
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