Contribution of patient registries to regulatory decision making on rare diseases medicinal products in Europe

Contribution of patient registries to regulatory decision making on rare diseases medicinal products in Europe

Between 2000 and 2021, the European Medicines Agency (EMA) assigned the orphan designation to over 1,900 medicines. Due to their small target populations, leading to challenges regarding clinical trial recruitment, study design and little knowledge on the natural history of the disease, the overall...

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Bibliographic Details
Main Authors: Carla J. Jonker, Elisabeth Bakker, Xavier Kurz, Kelly Plueschke
Format: Article
Language:English
Published: Frontiers Media S.A. 2022-08-01
Series:Frontiers in Pharmacology
Subjects:
patient registry
orphan medicinal product (OMP)
real-world evidence (RWE)
guideline on registry-based studies
orphan designation
Online Access:https://www.frontiersin.org/articles/10.3389/fphar.2022.924648/full

Internet

https://www.frontiersin.org/articles/10.3389/fphar.2022.924648/full

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