Medicines For Rare Diseases And Children

Orphan Medicinal Products (OMPs) are medicines which are developed to provide solutions for the diagnosis, prevention or treatment of rare diseases. A rare disease is defined as a life-threatening or chronically debilitating condition affecting not more than 5 in 10,000 persons within the European Union. It is also true that a significant number of rare diseases begin in childhood or are prevalent at birth.

Research and development of OMPs is a complex, costly, and time-consuming undertaking, more so than for more general medicines. This is due to several factors, such as the small number of patients, chronicity or severity of the disease, or the lack of scientific knowledge due to the limited number of people suffering from the condition.

EuropaBio’s Working Group on Rare Diseases focuses on identifying ways to encourage investment into Research & Development into rare diseases currently suffering from lack of treatment or unmet need.
The WG also places particular attention to working with public authorities to discuss patient access to the treatments created by the research completed thus far.

The WG is acting as a constructive partner in the discussions with the European Commission around the evaluation of the Orphan Medicinal Products and Paediatrics Regulations.