Pediatria pre prax 5/2007

DOES EUROPEAN „ORPHAN“ MEDICINAL PRODUCTS LEGISLATION HELP TO TREAT CHILDREN SUFFERING FROM RARE DISEASES?

Rare diseases are defined as those affected less than five in every 10 000 person in European Union. The purpose of this paper is to present activities, which make possible to stimulate development of appropriate medicine for treatment of rare disease, named “orphan” medicinal products. EU ”orphan” medicinal products legislation which entered into force in April 2000 is described in this article. Definition of “orphan” medicinal products as well as the procedure of designation and placing the products into the Community register is presented. More than 50% of designated ”orphan” medicinal products are intended for children population. The first thirteen of “orphan” medicinal products, which have been given pricing and reimbursement till July 2007 in the Slovak Republic, together with the restrictions of indication to children hospitals, are mentioned in this paper. Finally, possibilities how to achieve an increased availability of “orphan” medicinal products to rare disease patients in the Slovak Republic are mentioned.

Keywords: “Orphan” medicinal products, rare diseases, designation, European Union.