This study investigates possible measures that could be taken at EU level to address these challenges. It finds significant European added value in harmonising coordination and access across the 27 Member States, mainly in terms of improved diagnostic tools and availability of medical treatment, better health outcomes, particularly lower infant mortality, and improved well-being of family members and caregivers.
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.
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.
Gathering key stakeholders, including EU policymakers, industry leaders, patient advocacy groups, researchers, and healthcare providers. Its goal is to identify concrete actions and incentives to enhance innovation across the rare disease ecosystem.
New EMA ICH M14 guidelines sets common international standards for non‑interventional real‑world data studies to strengthen medicine safety assessment.
