Аccess to medicines for rare diseases in European countries

Abstract

Orphan drugs are drugs or vaccines suitable for the treatment and subsequent prevention of patients with rare diseases. Their availability and access to them are important for reducing the morbidity and mortality of those affected. Almost all patients with proven such disease need specific treatment. This is where the need to develop strategies, policies and procedures for creating, disseminating and accessing effective and advanced therapies arises. According to the principles of human rights, every patient should have equal access to the necessary treatment, regardless of the severity and specificity of his disease. Patients with rare diseases should not be excluded from the opportunity to improve their health, given the proven technological and medical advances in healthcare. It is on this basis that many governments and authorities build legislation, regulations and policies to promote and improve research and access to modern, effective and timely treatment for patients with rare diseases.