Contribution of patient registries to regulatory decision making on rare diseases medicinal products in Europe
Sign Up to like & getrecommendations! Published in 2022 at "Frontiers in Pharmacology"
DOI: 10.3389/fphar.2022.924648
Abstract: 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… read more here.
Keywords: patient registries; registries regulatory; medicinal products; contribution patient ... See more keywords