Rare Disease Day: 28 February 2011

News 25/02/2011

The European Medicines Agency supports Rare Disease Day, which is taking place on Monday 28 February.

Rare Disease Day is held every year on the last day of February. Started in 2008, it seeks to raise awareness of rare diseases, and to improve access to treatment and medical representation for individuals with rare diseases and their families. It is co-ordinated by the European Organisation for Rare Diseases (EURORDIS).

In 2011, the day's theme is 'rare but equal'. It is focusing on the differences between and within countries for patients with rare diseases, and compared to other segments of society. It is advocating for equal access for these patients to healthcare, social services, basic social rights, medicines and treatments.

About 30 million people living in the European Union (EU) suffer from a rare disease. The EU offers incentives to companies developing 'orphan' medicines for conditions affecting fewer than 5 in 10,000 people. These include reduced fees for marketing-authorisation applications and protection from market competition once the medicine is authorised. These incentives aim to bring more medicines for rare diseases to the European market.

The Agency plays a central role in the development and authorisation of these medicines: the European Commission grants orphan designation to medicines on the recommendation of the Agency's Committee for Orphan Medicinal Products (COMP), and designated orphan medicines are assessed centrally on a European level by the Agency's Committee for Medicinal Products for Human Use (CHMP), rather than in each Member State separately. To date, the European Commission has granted 838 orphan designations, and authorised 65 orphan medicines.

The Agency works closely with its international partners on the designation and assessment of orphan medicines. In particular, it collaborates with the United States Food and Drug Administration (FDA), by sharing information on orphan medicines under their confidentiality arrangement. The two authorities have also developed common procedures for applying for orphan designation and for submitting annual reports on the status of development of designated orphan medicines.

The Agency also works with organisations representing patients with rare diseases through EURORDIS.

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