Navigating EU Regulatory Challenges With Cell And Gene Therapies

Biopharma companies developing cell and gene therapies for the European Union face a rapidly evolving regulatory environment. Recent overhauls to EU pharmaceutical legislation are reshaping how advanced therapy medicinal products (ATMPs) are reviewed and approved. The integration of specialized committees into broader regulatory bodies raises questions about maintaining domain expertise, while expanded definitions of gene therapy now encompass products previously classified differently, triggering new requirements and risk assessments.

Platform technologies offer promising efficiencies but demand well-characterized submission packages. The new Joint Clinical Assessment process shifts health technology assessment from national to multinational review, requiring companies to predict their evidence parameters up to two years before marketing authorization submissions. Meanwhile, navigating scientific advice procedures differs substantially from FDA interactions, with written guidance replacing face-to-face discussions.

For detailed recommendations on preparing for scientific consultations, adapting to regulatory restructuring, understanding definitional changes, and building robust JCA strategies, review the complete guide.