Endo-ERN in its fifth year: a pinch of care, science, curiosity and new horizons

Abstract

Rare diseases are complex health entities with low prevalence (by the European definition less than 1 in 2000 people); some are so rare that only a few patients suffer from the condition in a whole region. In Europe, it is estimated that more than 7000 distinct rare diseases affect 6–8% of the population. Thus, only specially constructed and nationally endorsed combined efforts could address the challenges regarding that type of special health care management. During the past years, with the active involvement of clinical scientists as well as that of patients’ advocates, the European Commission (EC) has recognized the unmet long-term needs of patients with rare diseases. The EC adopted the 2011/24/EU directive on cross-border health care and launched large-scope programs aiming at standardized care and improvement in research and education (1).