Rare disease care in Europe – Gaping unmet needs

Abstract

In Europe, which provides its citizens with the highest level of social protection in the world, the needs of rare disease patients remain fundamentally unmet. The overwhelming majority of rare diseases still have no treatment or cure; infant mortality is unspeakably high; and when a safe and effective rare disease treatment is developed, patient access can vary dramatically from one EU Member State to the next, raising basic questions of health equity. The goal of this article is to advocate for an ambitious and far-reaching reform of the EU’s Orphan Drug Regulation framework, by showing how very difficult things are for Europe’s rare disease patients, in so many different ways, and on so many different levels.

Keywords

• Rare diseases
• Access to care
• Health equity
• Orphan Medicinal Products Regulation
• Regulation 883 on the Coordination of Social Security Systems
• 2011 Cross-border Healthcare Directive