EU Regulatory Playbook: Expert Guidance For EU Gene Therapy And Companion Diagnostic Trial Regulations

By Mesfin James, VP & Head of Regulatory Affairs, and Lakshmi Guduri, Senior Director, Project Management with TFS HealthScience

This playbook provides a concise yet comprehensive roadmap for navigating Europe’s multifaceted regulatory environment when conducting gene therapy trials involving genetically modified organisms (GMOs) and companion diagnostics (CDx). It underscores the critical need to classify each study component accurately—distinguishing between CTA submissions under the Clinical Trials Regulation (EU No. 536/2014), GMO authorizations governed by Directive 2001/18/EC or Directive 2009/41/EC, and CDx performance studies under the In Vitro Diagnostic Regulation (EU 2017/746)

By detailing three parallel submission tracks, the guide illustrates how misalignment among these processes can delay site activation and increase costs. It offers practical tools, including flowcharts and templates, to synchronize timelines, harmonize documentation, and anticipate regulatory queries. The playbook also highlights evolving trends such as the phased EUDAMED rollout, country-specific review efficiencies in Belgium, the Netherlands, and Denmark, and strategic risk-mitigation techniques. Sponsors are encouraged to engage early with EMA and national authorities to secure parallel reviews and streamline approvals. Finally, it emphasizes operational readiness, selecting sites with appropriate biosafety infrastructure, training staff, and establishing logistics for GMO handling and CDx specimen management—thereby ensuring that once regulatory clearances are in hand, clinical activities can commence without delay.