EU rare disease action plan

This own-initiative legislative procedure calls for an EU rare disease action plan. The amendments seek to reduce diagnostic delay (a one-year target and newborn screening), strengthen European Reference Networks, interoperable registries built on FAIR principles, and access to orphan and advanced therapy medicinal products including a cross-border hospital exemption, while addressing health technology assessment, clinical trials, the European Medicines Agency, HERA, patient organisations and the gender dimension of care.