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European Pharmaceutical Journal

Acta Facultatis Pharmaceuticae Universitatis Comenianae (formerly)

2 Issues per year


CiteScore 2016: 0.28

SCImago Journal Rank (SJR) 2015: 0.195
Source Normalized Impact per Paper (SNIP) 2015: 0.202

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2453-6725
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Orphan Dugs in EU / Lieky na zriedkavé choroby v EÚ

T. Foltánová / M. Mazág
Published Online: 2013-04-20 | DOI: https://doi.org/10.2478/afpuc-2013-0005

Medical and scientific knowledge about rare diseases is minimal or lacking, thus making research difficulties for pharmaceutical industry. Orphan drugs in EU are under supervision of European Commission, European medical agency (EMA) and Committee for orphan medicinal products (COMP).

The presentation provides a brief review of all supportive incentives in the field of orphan medicinal products as: the European orphan medicinal product (OMP) regulation, Guideline on Clinical Trials in Small Populations and Commission Regulation (EC) No 2049/2005 / support of small and medium enterprises (SMEs). It also introduces the concept of Clinical added value of orphan medicinal products, as one of the key instruments to increase the availability of orphan medicinal products in the member states. Separately it stresses the necessity of Health technology assessment implementation in whole process of orphan medicinal product development as well as the implementation of the Europlan indicators into the Slovak National plan

Výskum v oblasti terapeutických možností zriedkavých chorôb je náročný. Najväčšou prekážkou sú chýbajúce informácie o patogenéze tejto skupiny ochorení. Situáciu výrazne komplikuje nízka prevalencia. Príspevok prináša prehľad aktivít zameraných na podporu vývoja liekov na zriedkavé choroby na európskej úrovni. Tiež sa venuje vysoko aktuálnej problematike riešeniu problému rozdielnej dostupnosti liekov na zriedkavé choroby v jednotlivých členských štátov. Osobitne zdôrazňuje význam HTA pre sprístupnenie liekov na zriedkavé choroby v členských štátoch. Riešenie tohto problému je potrebné implementovať HTA do celého procesu podpory vývoja liekov na zriedkavé choroby od designácie až po registráciu.

Keywords : orphan drugs; policy in the field of orphan drugs; European orphan medicinal product (OMP) regulation

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About the article

Published Online: 2013-04-20

Published in Print: 2013-03-01


Citation Information: Acta Facultatis Pharmaceuticae Universitatis Comenianae, ISSN (Print) 0301-2298, DOI: https://doi.org/10.2478/afpuc-2013-0005.

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