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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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Detalles Bibliográficos
Autores principales:	Jonker, Carla J., Bakker, Elisabeth, Kurz, Xavier, Plueschke, Kelly
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2022
Materias:	Pharmacology
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9386590/ https://www.ncbi.nlm.nih.gov/pubmed/35991868 http://dx.doi.org/10.3389/fphar.2022.924648

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