Orphan Drug Use in Patients With Rare Diseases: A Population-Based Cohort Study

Orphan Drug Use in Patients With Rare Diseases: A Population-Based Cohort Study

Background: Orphan drugs are used for the diagnosis, prevention and treatment of rare diseases that, in the European Union, are defined as disorders affecting no more than 5 persons in 10,000. So far, a total of around 800 orphan medicinal products have been approved by the European Medicines Agency...

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Main Authors: Francesca Gorini, Michele Santoro, Anna Pierini, Lorena Mezzasalma, Silvia Baldacci, Elena Bargagli, Alessandra Boncristiano, Maurizia Rossana Brunetto, Paolo Cameli, Francesco Cappelli, Giancarlo Castaman, Barbara Coco, Maria Alice Donati, Renzo Guerrini, Silvia Linari, Vittoria Murro, Iacopo Olivotto, Paola Parronchi, Francesca Pochiero, Oliviero Rossi, Barbara Scappini, Andrea Sodi, Alessandro Maria Vannucchi, Alessio Coi
Format: Article
Language:English
Published: Frontiers Media S.A. 2022-05-01
Series:Frontiers in Pharmacology
Subjects:
orphan drug
rare disease
registry
population-based
prescription
Online Access:https://www.frontiersin.org/articles/10.3389/fphar.2022.869842/full

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https://www.frontiersin.org/articles/10.3389/fphar.2022.869842/full

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