New EU Orphan Regulation evaluation study findings

A new study to support the evaluation of the European Union Orphan Regulation (Technopolis, July 2020) and the accompanying Staff Working Document (SWD) has been released.

The study finds that the “EU Orphan Regulation has contributed to important strides in the field of rare diseases and development of orphan medicines. Since the Regulation was introduced more products have come on the market. There is also a promising pipeline of products under development, that may bring real value to patients for whom currently no treatment options exist.”

Responding to this, the European Federation of Pharmaceutical Industries and Associations (EFPIA) says it strongly believes that the evidence in both the study and SWD supports keeping the existing legislation on orphan medicines in place, while underlining the need to co-create vehicles to drive research to areas of unmet medical need and address issues around access to new treatments.