Patient-reported outcome claims in European and United States orphan drug approvals

• Ngā kaituhi matua: Szymon Jarosławski (Author), Pascal Auquier (Author), Borislav Borissov (Author), Claude Dussart (Author), Mondher Toumi (Author)
• Hōputu: Pukapuka
• I whakaputaina: Taylor & Francis Group, 2018-01-01T00:00:00Z.
• Ngā marau: article
• Urunga tuihono: Connect to this object online.

Purpose: We aimed to evaluate the rate of usage and the kind of patient-reported outcome (PRO) claims in orphan drug approvals from the European Medicines Agency (EMA) dated between 1/1/2012 and 31/12/2016 and to compare them to those from the US Food and Drug Administration (FDA). Methods: Orphan d...