Rare Disease Body Wants EU Legislative Overhaul To Address Orphan Definitions & Incentives

Eurordis has welcomed some parts of the leaked proposals for overhauling the EU medicines legislation, but it has a number of concerns, including the future role of patient representatives if the European Medicines Agency’s committee structure is slimmed down as planned.

Painted hand shows concept hologram Regulation on his hand. Drawn man in business suit with future technology screen and modern cosmic background — Orphan drug regulations are among those being overhauled in the EU • Source: Shutterstock

The European rare disease organization Eurordis has called on the European Commission to rectify what it sees as a number of shortcomings in the proposed revision of the pharmaceutical legislation in areas such as disease incidence, ultra-orphan diseases, and the definition of “significant benefit” in relation to orphan drugs.

The non-profit alliance, which represents more than 1,000 rare disease patient organizations, also wants reassurance that patients will be properly...