Real-world use of orphan medicinal products (OMPs) in rare disease (RD) patients: A population-based registry study

• Main Authors: Monica Mazzucato (Author), Cinzia Minichiello (Author), Andrea Vianello (Author), Laura Visonà dalla Pozza (Author), Ema Toto (Author), Paola Facchin (Author)
• Format: Book
• Published: Frontiers Media S.A., 2022-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Background: Despite calls for the use of real-world data, the rare diseases (RD) treatment landscape suffers from a scarcity of data referred to orphan medicinal products (OMP) use at the population level.Objectives: We aim to describe the characteristics and patterns of utilization of OMP in a siza...