On 11 August 2020, the European Commission published its evaluation of the EU’s orphan drug and pediatric medicine legislations. This is the first and only comprehensive evaluation of the Orphan Regulation and Pediatric Regulation since their adoption. Both have been evaluated together due to the overlap between rare diseases and diseases affecting children.

Among other aspects, the evaluation concludes that the Regulations have fostered the development and availability of medicines for patients with rare diseases and for children. Conversely, it also highlights that both regulations have not adequately managed to support development in areas where the need for medicines is greatest. In addition, the evaluation finds that incentives remain relevant to encourage the development of medicines for rare diseases but questions the merits of the 10-year market exclusivity for such products.

Based on the outcome of the evaluation, the Commission reported that it will examine the impacts of possible future policy options to update the legal framework. A preliminary impact assessment is expected to be published for public feedback this autumn.