Young advocates met in Paris from 23–25 April 2026 for the second ERDERA Training for Young Advocates for Rare Diseases, focused on advocacy, clinical research, patient rights and partnership in paediatric rare disease research.
Published on 14 December, this WHO technical document maps global trends in registered clinical studies using human genomic technologies from 1990 to 2024, including patterns of inclusion and equity.
Following the 11 December 2025 political agreement, EU institutions are moving towards formal adoption of a new Directive and Regulation that will reshape how medicines—including orphan and paediatric medicines—are developed, authorised and supplied across the European Union.
The RealiseD project launched a new multi-stakeholder survey that invites the rare disease community to share insights and help improve clinical trial enrolment
Open for consultation from 12 December to 12 April, this draft ICH guideline sets out how to design and submit robust patient preference studies to inform regulatory decision‑making across a medicine’s lifecycle.
