Kuzelová M, Kubácková K, Palágyi M, Smíd M
Katedra farmakológie a toxikológie FaF UK, Bratislava, SR.
Cas Lek Cesk. 2006;145(4):296-300.
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 research development and marketing of appropriate medicine for tretment of rare disease, named "Orphan" medicinal products. EU "Orphan" medicinal products legislation which entered into force in April 2000 is described. Definition of "Orphan" medicinal products as well as the procedure of designation and placing the products into the Community register is presented. Those incentives to industry are described, which are already five years very well implemented oh the European level mostly on the pre-authorisation phase of "Orphan" medicinal products development, but also in the registration process as well as the post-authorisation phase. Finaly, the first twenty "Orphan" medicinal products, which have been given positive opinion in the Community for the grant of a marketing authorisation till April 2005 are mentioned in this work. The real availability of "Orphan" medicinal products in the particular EU member states is analysed.
罕见病被定义为在欧盟每10000人中患病人数少于5人的疾病。本文旨在介绍一些活动，这些活动有助于推动针对罕见病治疗的适宜药物（即“孤儿”药品）的研发和市场推广。文中描述了2000年4月生效的欧盟“孤儿”药品立法。介绍了“孤儿”药品的定义以及产品指定和列入共同体登记册的程序。阐述了对制药行业的激励措施，这些措施在欧洲层面已实施五年，主要体现在“孤儿”药品研发的授权前阶段，也包括注册过程和授权后阶段。最后，本文提及了截至2005年4月已在共同体获得上市许可积极意见的首批20种“孤儿”药品。分析了“孤儿”药品在欧盟各成员国的实际可及性。
