Orphanet report

Patient registries and databases constitute key instruments to develop clinical research in the field of rare diseases (RD), to improve patient care and healthcare planning. 

Registries of patients treated with orphan drugs are particularly relevant as they allow the gathering of evidence on the effectiveness of the treatment and on its possible side effects, keeping in mind that marketing authorization is usually granted at a time when evidence is still limited.

The Orphanet report gathers the information collected regarding systematic collections of data for a specific disease or a group of diseases in all EU countries.

For the report click here.