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A Regulatory Overview About Rare Diseases

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Rare Diseases Epidemiology

Part of the book series: Advances in Experimental Medicine and Biology ((AEMB,volume 686))

Abstract

Rare diseases attract very little interest for drug development. To create more favourable conditions, incentives for development (scientific advice, research grants) and marketing of medicines (market exclusivity, regulatory fee reductions) are offered by several orphan legislations. These incentives have proven to be a valuable stimulus for research and development for new products for treatment, prevention and diagnosis of rare diseases. In the US almost 2000 products have been designated as orphan medicines and about 340 have received marketing authorisation. Rare diseases have also gained attention from regulators in the last years. Nowadays it is acknowledged that rare diseases deserve specific attention and individual regulatory guidance. Also, regulatory authorities have developed different mechanisms to put products on the market considering specific limitations of data availability (conditional marketing authorisation, exceptional circumstances authorisation). In the future more initiatives will have to address the need for networking scientific knowledge and research capabilities to address the difficulties to generate data in rare diseases.

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Authors and Affiliations

  1. European Medicines Agency, Canary Wharf, London, E14 4HB, UK

    Jordi Llinares

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  1. Jordi Llinares
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Correspondence to Jordi Llinares .

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Editors and Affiliations

  1. , Instituto de Investigaciñon en Enfermeda, Instituto de Salud Carlos III, Monforte de Lemos 5, Madrid, 28029, Spain

    Manuel Posada de la Paz

  2. , Office of Rare Diseases Research, National Institutes of Health, Executive Boulevard 6100, Bethesda, 20892-7518, USA

    Stephen C. Groft

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Llinares, J. (2010). A Regulatory Overview About Rare Diseases. In: Posada de la Paz, M., Groft, S. (eds) Rare Diseases Epidemiology. Advances in Experimental Medicine and Biology, vol 686. Springer, Dordrecht. https://doi.org/10.1007/978-90-481-9485-8_12

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