European regulation on orphan medicinal products: 10 years of experience and future perspectives.
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DOI: 10.1038/NRD3445
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摘要: In 2000, regulation on orphan medicinal products was adopted in the European Union with aim of benefiting patients who suffer from serious, rare conditions for which there is currently no satisfactory treatment. Since then, more than 850 drug designations have been granted by Commission based a positive opinion Committee Orphan Medicinal Products (COMP), and 60 drugs received marketing authorization Europe. Here, stimulated tenth anniversary COMP, we reflect outcomes experience gained past decade, contemplate issues future, such as catalysing development large number diseases that still lack effective treatments.
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