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

• Main Authors: Francesca Gorini (Author), Michele Santoro (Author), Anna Pierini (Author), Lorena Mezzasalma (Author), Silvia Baldacci (Author), Elena Bargagli (Author), Alessandra Boncristiano (Author), Maurizia Rossana Brunetto (Author), Paolo Cameli (Author), Francesco Cappelli (Author), Giancarlo Castaman (Author), Barbara Coco (Author), Maria Alice Donati (Author), Renzo Guerrini (Author), Silvia Linari (Author), Vittoria Murro (Author), Iacopo Olivotto (Author), Paola Parronchi (Author), Francesca Pochiero (Author), Oliviero Rossi (Author), Barbara Scappini (Author), Andrea Sodi (Author), Alessandro Maria Vannucchi (Author), Alessio Coi (Author)
• Format: Book
• Published: Frontiers Media S.A., 2022-05-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...