Аннотация
Diseases that are life-threatening or chronically debilitating are qualified as rare diseases (RD) in the EU if their prevalence is ≤ 5 per 10.000 (1, 2).One in 17 people in the EU (27 to 36 million people) suffer from one or more of the 6000-8000 rare diseases that exist (2, 3).Many rare diseases are characterized by an early/juvenile onset and a fast and progressive course of the disease, which makes early access to drugs paramount to stop disease progression and prevent irreversible organ and tissue damage (4).The level of access to orphan medicinal products