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

Gorini, Francesca; Santoro, Michele; Pierini, Anna; Mezzasalma, Lorena; Baldacci, Silvia; Bargagli, Elena; Boncristiano, Alessandra; Maurizia Rossana Brunetto,; Cameli, Paolo; Cappelli, Francesco; Castaman, Giancarlo; Coco, Barbara; Maria Alice Donati,; Guerrini, Renzo; Linari, Silvia; Murro, Vittoria; Olivotto, Iacopo; Parronchi, Paola; Pochiero, Francesca; Rossi, Oliviero; Scappini, Barbara; Sodi, Andrea; Vannucchi, ALESSANDRO MARIA; Coi, Alessio

doi:10.3389/fphar.2022.869842

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, however the utilization profile of orphan drugs has yet to be explored. This study aimed at assessing the utilization profile of orphan drugs authorized for marketing by the Italian Medicines Agency using population-based data

Orphan Drug Use in Patients With Rare Diseases: A Population-Based Cohort Study / 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. - In: FRONTIERS IN PHARMACOLOGY. - ISSN 1663-9812. - ELETTRONICO. - 13:(2022), pp. 0-0. [10.3389/fphar.2022.869842]