Licensing of Orphan Medicinal Products-Use of Real-World Data and Other External Data on Efficacy Aspects in Marketing Authorization Applications Concluded at the European Medicines Agency Between 2019 and 2021

• Main Authors: Frauke Naumann-Winter (Author), Franziska Wolter (Author), Ulrike Hermes (Author), Eva Malikova (Author), Nils Lilienthal (Author), Tania Meier (Author), Maria Elisabeth Kalland (Author), Armando Magrelli (Author)
• Format: Book
• Published: Frontiers Media S.A., 2022-08-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Background: Reference to so-called real-world data is more often made in marketing authorization applications for medicines intended to diagnose, prevent or treat rare diseases compared to more common diseases. We provide granularity on the type and aim of any external data on efficacy aspects from...