Rare diseases

Rare diseases

Poster

Gergana Veselinska1, Krasimira Kokalarova1, Denitsa Stregarska-Lulcheva1, Emil Hristov1, Kalina Andreevska1, Valemtina Petkova2
1Sofia University "St. Kliment Ohridski", Faculty of Chemistry and Pharmacy
2MU Sofia, Faculty of Pharmacy, Department of Social pharmacy

Objective: To study the regulatory framework for the diagnosis and treatment of rare diseases with the so-called 'orphan medicines'. Materials and methods: Content analysis was conducted and data from regulatory documents were used and analyzed, such as: Regulation (EC) No 847/2000, Regulation (EC) No 141/2000, Regulation (EC) No 726/2004. Results: The history of rare diseases began to be written as far back as 1983 and remains up to date. Rare diseases are diseases that affect less than 5 in 10,000 people and affect between 6% and 8% of the population. "Orphan medicine" is a medicinal product intended for the diagnosis, prevention or treatment of rare diseases. The European Medicines Agency (EMA) plays a central role in facilitating the development and authorization of rare diseases medicines, referred to as' orphan drugs. Authorization of medicinal products as rare diseases medicinal products is carried out in the European Community by the European Commission on the recommendation of EMA's COMP (Committee for orphan medicinal product). The decision shall be published in the Community register on the Commission website. Conclusion: Orphan drugs are one of the most important trends in modern pharmacy, giving hope for longevity and quality of life. In the Republic of Bulgaria, European legislation on orphan drugs and the treatment of rare diseases is currently being implemented.
Keywords: orphan medicines, rare diseases, Committee for orphan medicinal product