25,000 Maltese citizens suffer from a rare disease

25,000 Maltese are affected by one of around 8,000 rare diseases, leading the Maltese Presidency of the Council of the European Union to bring together a number of organisations and institutes to explore synergies within and across the research and healthcare rare diseases communities, as well as gaps and existing challenges.

A high level conference is being organised today in Malta to discuss the development of medicinal products for rare diseases, which affect 30 million people across the EU.

This conference will bring together the European Organisation for Rare Diseases (EURORDIS), EU Health Ministers, experts from the Committee of Orphan Medicinal Products (COMP) and the members of the Innovative Medicines Initiative (IMI).

COMP is responsible for reviewing applications from people or companies seeking ‘orphan-medicinal-product designation’, issued in the case of medicines to be developed for the diagnosis, prevention or treatment of rare diseases that are life-threatening or very serious.

In the European Union (EU), a disease is defined as rare if it affects fewer than 5 in 10,000 people across the member states. COMP, which meets at the European Medicines Agency (EMA) in which representatives of all EU member states participate, is also responsible for advising the European Commission on the establishment and development of a policy on orphan medicinal products in the EU.

The experts will be contributing to drafting up guidelines on matters relating to orphan medicinal products.

In a workshop on Monday, Malta’s Minister for Social Dialogue, Consumer Affairs and Civil Liberties Helena Dalli said that such open debate would go a long way to creating awareness and would help to dissuade any inequity towards people with rare diseases

The workshop on Integrated Research and Healthcare for Rare Diseases was organised by the EURORDIS and supported by the National Alliance for Rare Diseases chaired by Michelle Muscat, Spouse of Prime Minister Joseph Muscat and honorary patron of EURORDIS.