Eurordis - Rare Disease Europe

The 20 million people living with a rare disease in the European Union, and the 30 million in Europe, highly anticipate the Revision of the Orphan Medicinal Products and Paediatric Regulations at the end of this year. The EU Regulation on Orphan Medicinal Products has helped transform the lives of many people living with rare diseases and continues to be successful in fulfilling its primary purpose – attracting investment to the development of therapies for life-threatening or debilitating diseases for millions of people who today live without any, or without satisfactory treatment options. Yet major difficulties in accessing approved treatments still exist.

We are looking to the European Union to take the opportunity of the revision of the Orphan Medicinal Products and Paediatrics Regulations to create a robust regulatory framework that will drive innovation and improve access to transformative treatments for this population in the years to come.