The new issue of our scientific journal Rare Diseases and Orphan Drugs is now online. The issue contains 8 publications on various topics. The editorial is “Regulation (EC) № 141/2000 for orphan medicinal products – 20 years later”.
This year marks the 20th anniversary of the entry into force of the Regulation (EC) № 141/2000 on orphan medicinal products. This legislation determines the criteria for the designation of orphan medicinal products for the prevention, diagnosis and treatment of rare conditions, as well as provides incentives for their research, development and marketing. Although rare diseases appear in some earlier documents of the European legislation, this regulation is the first to specifically and exclusively address the problems of people with rare diseases and their families in Europe.
Twenty years later, it is time for reconsideration. In this context, the European Commission conducted a series of public consultations with various stakeholders, followed by a study of the effect and results of Regulation (EC) № 141/2000. There are many reasons for satisfaction, but the identified problems are not few. However, on the most important issue – adequate access of patients with rare diseases to therapy with orphan drugs – the success is partial. Yes, the number of orphan drugs with a marketing authorization in the EU is significant. But at the same time, there are huge inequalities in access to them across member states. A significant part of these therapies remain inaccessible to Eastern European patients. Read the whole article here.