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

• Auteurs principaux: Monica Mazzucato (Auteur), Cinzia Minichiello (Auteur), Andrea Vianello (Auteur), Laura Visonà dalla Pozza (Auteur), Ema Toto (Auteur), Paola Facchin (Auteur)
• Format: Livre
• Publié: Frontiers Media S.A., 2022-09-01T00:00:00Z.
• Sujets: article
• Accès en ligne: 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...