Ultra-rare disease: an European perspective

In the past few years, interest in rare diseases has grown, as demonstrated by the agendas of politicians and health authorities, but too little attention is still paid to ultra-rare diseases. Although no legal definition of an "ultra-rare" disease has yet been established, this subcategory was informally introduced by the National Institute for Health and Care Excellence for drugs with indications for diseases that have a prevalence <1 per 50 000 persons [1–3].
Source: European Respiratory Review - Category: Respiratory Medicine Authors: Tags: Pulmonary vascular disease Editorials Source Type: research