This initiative is also highly relevant from a rare diseases perspective, as paediatric cancers are rare conditions where small patient populations make robust non‑clinical proof‑of‑concept data essential for responsible and ethical trial initiation.
For rare diseases, where research often relies on limited animal models and small development teams, the regulatory acceptance of virtual control groups could help streamline early non‑clinical studies while maintaining a strong focus on patient safety.
The European Medicines Agency has begun a formal review of Tavneos (avacopan) after emerging information raised questions about the integrity of key clinical trial data supporting its EU authorisation, with potential implications for adults living with rare autoimmune vasculitis.
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.
