Promoting access to innovative medicines
The right to health must be guaranteed to all, even to patients suffering from rare diseases: nobody must be excluded from the progress of science.
A disease is defined as rare in Europe when it affects less than 1 in 2000. There are more than 6000 rare diseases and they are often chronic and life-threatening. 80% of rare diseases patients are children.
However, the research and marketing of orphan medicines are limited as, in view of considerable investments in research and development, expected revenues are very low.
In this field, we are actively involved in international and national collaborative projects aimed to enhance the scientific research on medicines and therapies for rare diseases patients.
We also manage and regularly update EuOrphan, a database collecting regulatory and scientific information on drugs for rare diseases.