Orphan Dugs in EU / Lieky na zriedkavé choroby v EÚ

T. Foltánová 1  and M. Mazág 2
  • 1 Faculty of Pharmacy, Comenius University, Odbojárov 10, 832 32 Bratislava
  • 2 State Institute for Drug Control, Bratislava, Slovakia

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

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  • Regulation (EC) No 141/2000 of the European parliament and of the council. Off J Eur Commun, 2000, 43, L18:1-5.

  • Regulation (EC) No 847/2000, Off J Eur Commun, 43, 2000, L103, p. 5-8.

  • European Commission guideline ENTR/6283/00. (http://www.ema.europa.eu/ema/index.jsp?curl=/pages/medicines/landing/epar_search.jsp&mid=WC0b01ac058001d124). Accessed February 20 2013.

  • Guideline on Clinical Trials in Small Populations. (http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003615.pdf). Accessed February 20 2013.

  • Commission Regulation (EC) No 2049/2005. (http://ec.europa.eu/health/files/eudralex/vol-/reg_2005_2049/reg_2005_2049_en.pdf) Accessed February 20 2013.

  • Rare Diseases: Europe’s Challenges. (http://ec.europa.eu/health/ph_threats/non_com/docs/rare_com_en.pdf). Accessed February 20 2013.

  • The Council Recommendation on a European Action in the Field of Rare Diseases, adopted by the Health Council on 9 June 2009. (http://eurlex.europa.eu/LexUriServ/LexUriServ.do?uri=OJ:C:2009:151:0007:0010:EN:PDF) Accessed February 20 2013.


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