Pharmacotherapy of rare and ultra-rare diseases in children – clinical and economics aspects

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Mateusz Maciejczyk, Natalia Marcińczyk, Agnieszka Pietrzykowska, Edyta Rysiak, Ilona Zaręba, Iwona Raciborska, Rafał Zadykowicz

3 (44) 2015 s. 194–197
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Fraza do cytowania: Maciejczyk M., Marcińczyk N., Pietrzykowska A., Rysiak E., Zaręba I., Raciborska I., Zadykowicz R. Pharmacotherapy of rare and ultra-rare diseases in children – clinical and economics aspects. Polski Przegląd Nauk o Zdrowiu. 2015;3(44):194–197.

Rare diseases are multisystem, often incurable disorders, which occur with a frequency of not more than 5 cases per 10 000 citizens of the European Union (ultra‑rare disorders are diseases with the incidence of less than 1:200 000). More than half of the cases applies to small children, with approximately 30% of patients die before 5 years of age. Due to a very low number of patients and the lack of widely available diagnostic methods and effective therapeutic strategies, rare diseases are a sphere with a very high „European added value”, and are a particular interest in modern medicine and pharmacy. To this day, is still missing effective organizational and legal solutions, as well as treatment of rare disorders continually needs improvement financing. It is also necessary to introduce the modern classification and registry of rare diseases, increase access to screening and diagnostics tests and innovative medicines, improve the level of healthcare, as well as the promotion of science, education and social support for patients and their families. The study presents important clinical and economics aspects of pharmacotherapy of rare and ultra‑rare diseases in children.

Key words: rare diseases, orphan drugs, pharmacoeconomics.

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