Mary Harney (Dublin Mid West, Independent)

The term "rare disease" refers to conditions with a low prevalence of no more than five per 10,000 persons in the population. It is estimated that there are between 5,000 and 8,000 distinct rare diseases affecting in total between 6% and 8% of the population over the course of their lives.

I have met with the Irish Platform for Patient Organisations, Science and Industry, IPPOSI, which represents rare disease patient organisations and industry and the Genetic and Rare Disorders Organisation, GRDO, on issues relating to health care for patients with rare diseases.

Research on rare diseases is scarce and scattered in different laboratories throughout the European Union. Owing to the small market, investment in medicinal products and devices for people with rare diseases is limited. As a result, the price for many of the treatments is such that it is impossible for them to meet conventional criteria for cost effectiveness. Progress has been made on specific rare diseases such as haemophilia. Through the work of the Haemophilia Produce Selection and Monitoring Advisory Board, the cost we are now paying for blood products has reduced substantially in recent years.

I have asked the clinical director of the Health Service Executive, Dr. Barry White, to explore a mechanism whereby a ring-fenced drugs budget could be put in place to specifically cater for people with other rare diseases. Work has also commenced on other elements of rare disease policy such as screening procedures and the organisation of reference centres and clinical networks involved in the diagnosis and treatment of rare diseases.

In view of the low prevalence of rare diseases, there is added value in working collaboratively at EU level and Ireland has supported the EU Council recommendation on an action plan for rare diseases which was adopted in June 2009. Ireland has two years to prepare a national plan.

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