Most rare diseases are life-threatening and chronically debilitating conditions, and the vast majority of them are genetically determined. Their individually low prevalence requires special combined efforts to address them so as to improve diagnosis, care and prevention. Though it is difficult to develop a public health policy specific to each rare disease, it is possible to have a global rather than a piecemeal approach in the areas of scientific and biomedical research, drug research and development, industry policy, information and training, social benefits, hospitalisation and outpatient care. In the recent past, several initiatives at EU and Member States levels have been taken and proved efficient in developing suitable solutions which are now having a positive impact on the quality of life of patients. These initiatives are presented here. They include the establishment of Orphanet, a database of rare diseases and orphan drugs providing an encyclopedia of rare diseases and a directory of associated expert services, the funding of research networks to boost the collaboration between research teams, as well as the funding of networks of clinical centres of reference to better serve the patients and contribute to developing clinical research.