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.
With over 300 million people affected by rare diseases, timely access to effective therapies is critical. Despite strong alignment in regulatory expectations, patients in many regions face years of delays highlighting the need for improved international coordination.
New EMA ICH M14 guidelines sets common international standards for non‑interventional real‑world data studies to strengthen medicine safety assessment.
