title: European regulation on orphan medicinal products: 10 years of experience and future perspectives.
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.
authors.forename: Kerstin
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authors.lastname: Westermark
authors.forename: Birthe Byskov
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authors.lastname: Söderholm
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authors.lastname: Wilk-Kachlicka
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authors.lastname: Borvendég
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authors.lastname: Krieviņš
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authors.lastname: Matulevičiene
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authors.lastname: Metz
authors.forename: Albert Cilia
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authors.lastname: Vincenti
authors.forename: Albertha
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authors.lastname: Voordouw
authors.forename: Bożenna
authors.initials: B
authors.lastname: Dembowska-Bagińska
authors.forename: Ana Corrêa
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authors.lastname: Nunes
authors.forename: Flavia Mirela
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authors.lastname: Saleh
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authors.lastname: Foltánová
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authors.lastname: Možina
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authors.lastname: Torrent i Farnell
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authors.lastname: Beerman
authors.forename: Segundo
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authors.forename: Marie Pauline
authors.initials: MP
authors.lastname: Evers
authors.forename: Lesley
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authors.lastname: Greene
authors.forename: Sigurdur
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authors.lastname: Thorsteinsson
authors.forename: Lars
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authors.lastname: Gramstad
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authors.lastname: Mavris
authors.forename: Fabrizia
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authors.forename: Chantal
authors.initials: C
authors.lastname: Belorgey
mesh_terms: D017277:Drug Approval; D015195:Drug Design; D005062:European Union; D006801:Humans; D007880:Legislation, Drug; D009965:Orphan Drug Production / Q000331:legislation & jurisprudence*; D035583:Rare Diseases / Q000188:drug therapy; D014481:United States