International Rare Diseases Day – 12 new orphan medicines authorised in the EU over the past year
Over the past year, a total of twelve medicines for the treatment of rare diseases were recommended for marketing authorisation by the European Medicines Agency’s (EMA) Committee for Human Medicinal Products (CHMP). These medicines are known as "orphan” medicinal products, and these new medicinal products include three medicines for the treatment of rare cancers, three for multidrug resistant tuberculosis and two for pulmonary arterial hypertension. The marketing authorisations of these medicines became valid in Croatia from the date of the accession to the EU, thus providing affordability of these medicines to Croatian patients. You may read more about the newly authorised orphan medicinal products on the European Medicines Agency webpages or here.
Rare diseases are defined as potentially fatal conditions or disease leading to a chronic weakness, which is a condition that does not affect more than 5 in 10,000 people. It is estimated that 30 million people in the EU suffers from a rare disease.
International Rare Disease Day is held every year on the last day of February. It aims to raising awareness of rare diseases, and to improve access to treatment for patients. The Agency for Medicinal Products and Medical Devices (HALMED) joined this year the Croatian Association for Rare Diseases in manifesting this day. Within this manifestation, the HALMED representatives held workshops on the importance of adverse reactions reporting in Osijek, Rijeka, Pula, Split, Dubrovnik, Bjelovar, Križevci. A lecture was held within the 3rd Croatian Symposium and National Conference on the Rare Diseases on the HALMED’s role in medicines affordability after the accession to the EU. More about the HALMED’s participation in manifesting the International Rare disease Day, you may read here.