In his keynote address, Christof von Kalle reflected on the evolution and current landscape of cell and gene therapy, highlighting both the remarkable progress and the persistent challenges facing the field. Von Kalle began by acknowledging the complexity inherent in these therapies, noting that treatments are often highly individualised and multifactorial, with products ranging from cell-based to gene-based interventions, or combinations thereof. This complexity extends from diagnosis through to product development, creating significant hurdles for both clinical and regulatory pathways.
Von Kalle observed that traditional clinical trial models are frequently unsuitable for these therapies, given the rarity and specificity of many conditions treated. As a result, there is a growing emphasis on real-world evidence and the use of health system data to support regulatory and reimbursement decisions. He pointed out that across Europe, national programmes vary widely in their strengths, with some excelling in basic research and others in clinical development or market access. However, this diversity has led to a fragmented landscape, with a patchwork of strategies that can be difficult to navigate and often lack harmonisation.
Regulatory and investment challenges remain significant. Von Kalle cited overregulation, duplication of effort, and insufficient private investment as barriers to innovation, particularly when compared to the United States and Asia. He stressed the need for more risk-based, flexible regulatory approaches and greater transparency in resource allocation.
Germany’s recent efforts to develop a national strategy for gene and cell therapy were presented as a case study. Von Kalle described a multi-stakeholder approach, coordinated by the Berlin Institute of Health, which brought together industry, academia, patient organisations, and policymakers to identify key areas for action. This process resulted in a comprehensive set of proposals aimed at strengthening the entire value chain, from talent development to market access.
Von Kalle concluded by advocating for greater European collaboration, suggesting that a united, needs-driven approach would allow the continent to build on national strengths and foster international partnerships. Ongoing initiatives, such as the creation of transparent resource maps and the integration of artificial intelligence and health data, are seen as crucial steps towards a more cohesive and innovative future for cell and gene therapy.
