Availability, accessibility and delivery to patients of the 28 orphan medicines approved by the European Medicine Agency for hereditary metabolic diseases in the MetabERN network
The European Medicine Agency granted marketing approval to 164 orphan medicinal products for rare diseases, among which 28 products intended for the treatment of hereditary metabolic diseases. Taking advantage of its privileged connection with 69 healthcare centres of excellence in this field, Metab...
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| Main Authors: | , |
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| Format: | Article (Journal) |
| Language: | English |
| Published: |
06 January 2020
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| In: |
Orphanet journal of rare diseases
Year: 2020, Volume: 15, Pages: 1-10 |
| ISSN: | 1750-1172 |
| DOI: | 10.1186/s13023-019-1280-5 |
| Online Access: | Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1186/s13023-019-1280-5
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| Author Notes: | Jean-Michel Heard, Charlotte Vrinten, Michael Schlander, Cinzia Maria Bellettato, Corine van Lingen, Maurizio Scarpa and the MetabERN Collaboration Group ; Jean-Michel Heard, Charlotte Vrinten, Michael Schlander, Cinzia Maria Bellettato, Corine van Lingen, Maurizio Scarpa, Gert Matthijs, Marie-Cécile Nassogne, François-Guillaume Debray, Dominique Roland, Teodora Chamova, Viktor Kozich, Jesina Pavel, Martin Zenker, Martin Zenker, Christina Lampe, Anihb Martin Das, Julia Hennermann, Stefan Kölker, Natalie Weinhold, Klaus Mohnike, Sarah Gruenert, Allan Meldgaard Lund, Montserrat Morales-Conejo, Mireia del Toro-Riera, Luis Aldámiz-Echevarría, Maria-Teresa Garcia-Silva, Manuel Schiff, Laurent Gouya, Pascale de Lonlay, Nadia Belmatoug, Dominique P. Germain, Aline Cano, Dries Dobbelaere, Simon Jones, Charlotte Dawson, Patrick Deegan, Saikat Santra, Suresh Vijay, Danijela Petkovic Ramadza, Ivo Barić, Tamara Žigman, György Pflieger, Katalin Szakszon, Rita Kaposta, Serena Gasperini, Alberto Burlina, Giancarlo Parenti, Pietro Strisciuglio, Giovanni Ceccarini, Antonio Federico, Alessandro Simonati, Birute Tumiene, Hidde Huidekoper, Francian van Spronsen, Annet Bosch, Maria-Estela Rubio-Gozalbo, Gepke Visser, Trine Tangeraas, Aasne Aarsand, Beata Kieć-Wilk, Ana-Maria Simões Mendes Gaspar, Dulce Quelhas, Elisa Leao-Teles, Olga Azevedo, Esmeralda-Maria Ferreira Rodriges Silva, Luísa-Maria de Abreu Freire Diogo Matos, Esmeralda Martins, Svetlana Lajic, Niklas Darin, Urh Groselj, Mojca-Zerjav Tansek, the MetabERN collaboration group |
| Summary: | The European Medicine Agency granted marketing approval to 164 orphan medicinal products for rare diseases, among which 28 products intended for the treatment of hereditary metabolic diseases. Taking advantage of its privileged connection with 69 healthcare centres of excellence in this field, MetabERN, the European Reference Network for hereditary metabolic diseases, performed a survey asking health care providers from 18 European countries whether these products are available on the market, reimbursed and therefore accessible for prescription, and actually delivered in their centre. |
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| Item Description: | Gesehen am 07.04.2020 |
| Physical Description: | Online Resource |
| ISSN: | 1750-1172 |
| DOI: | 10.1186/s13023-019-1280-5 |