Proposed reform of the EU pharmaceutical legislation: New regime for orphan medicinal products under the proposed regulation

As part of the revision of the EU pharmaceutical legislative framework, a new regulation is proposed, which will replace, among others, Regulation (EU) 141/2000 on orphan medicinal products. According to the European Commission, the current legislation on orphan medicinal products no longer meets the needs of both patients and the pharmaceutical sector, with 95% of the 6000+ recognised rare diseases having no treatment option. Below we set out the main changes in the proposed regulation.