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.
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Acknowledgements
The Chairperson of the COMP would like to specifically acknowledge the invaluable input of COMP colleagues who actively contributed to this work by coordinating the discussions of subheadings and coordinating the participation of the entire committee, namely B. Sepodes (COMP member, nominated by the EMA's recommendation); J. T. i Farnell (COMP member, Spain); F. Rivière, J. Llinares-Garcia and S. Aarum (EMA COMP Scientific Secretariats), L. Greene and B. B. Holm (COMP patients' representatives);and M. Mavris (COMP General Observer, EURORDIS). The views expressed in this article are the personal views of the authors and may not be understood or quoted as being made on behalf of or reflecting the position of the EMA or one of its committees or working parties.
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The Committee for Orphan Medicinal Products and the European Medicines Agency Scientific Secretariat. European regulation on orphan medicinal products: 10 years of experience and future perspectives. Nat Rev Drug Discov 10, 341–349 (2011). https://doi.org/10.1038/nrd3445
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DOI: https://doi.org/10.1038/nrd3445
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