| Literature DB >> 21532564 |
Kerstin Westermark, Birthe Byskov Holm, Mirjam Söderholm, Jordi Llinares-Garcia, Frida Rivière, Stiina Aarum, Florence Butlen-Ducuing, Stelios Tsigkos, Agnieszka Wilk-Kachlicka, Cinzia N'Diamoi, János Borvendég, David Lyons, Bruno Sepodes, Brigitte Bloechl-Daum, André Lhoir, Mariana Todorova, Ioannis Kkolos, Kateřina Kubáčková, Heidrun Bosch-Traberg, Vallo Tillmann, Veijo Saano, Emmanuel Héron, Rembert Elbers, Miranda Siouti, Judit Eggenhofer, Patrick Salmon, Maurizio Clementi, Dainis Krieviņš, Aušra Matulevičiene, Henri Metz, Albert Cilia Vincenti, Albertha Voordouw, Bożenna Dembowska-Bagińska, Ana Corrêa Nunes, Flavia Mirela Saleh, Tatiana Foltánová, Martin Možina, Josep Torrent i Farnell, Björn Beerman, Segundo Mariz, Marie Pauline Evers, Lesley Greene, Sigurdur Thorsteinsson, Lars Gramstad, Maria Mavris, Fabrizia Bignami, Annie Lorence, Chantal Belorgey.
Abstract
In 2000, regulation on orphan medicinal products was adopted in the European Union with the aim of benefiting patients who suffer from serious, rare conditions for which there is currently no satisfactory treatment. Since then, more than 850 orphan drug designations have been granted by the European Commission based on a positive opinion from the Committee for Orphan Medicinal Products (COMP), and more than 60 orphan drugs have received marketing authorization in Europe. Here, stimulated by the tenth anniversary of the COMP, we reflect on the outcomes and experience gained in the past decade, and contemplate issues for the future, such as catalysing drug development for the large number of rare diseases that still lack effective treatments.Entities:
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Year: 2011 PMID: 21532564 DOI: 10.1038/nrd3445
Source DB: PubMed Journal: Nat Rev Drug Discov ISSN: 1474-1776 Impact factor: 84.694