Advanced Therapy Safety Management: Proactive Strategies for Real-World Risk Detection

Advanced therapies, including cell and gene therapies (CGT), T cell engagers, mRNA-based products, and other novel platforms are transforming the treatment landscape across a wide range of diseases. These modalities while offering promising therapeutic potential, introduce complex safety challenges due to their immune-mediated mechanisms of action and novel manufacturing processes. These include cytokine release syndrome (CRS), neurotoxicity or and other severe inflammatory responses as well as delayed-onset toxicities including secondary malignancies. As such, proactive safety monitoring from Phase 1 through post-marketing is critical to ensure patient safety, accelerate development, and meet evolving regulatory expectations.

Compounding these challenges, advanced therapies are often approved based on small, highly selective clinical trial patient populations, limiting the availability of safety data and its relevance in broader, real-world settings due to the potential for rare and long-latency adverse events. These challenges are compounded by the limited availability of patients with rare diseases, further hindering data collection and signal detection. As a result, regulators mandate enhanced pharmacovigilance efforts, including long-term patient registries, post-authorization safety studies, and the use of real-world evidence, requiring a strategic approach to monitoring patient safety throughout the post-approval period.

This dynamic webinar brings together experts in medical affairs, pharmacovigilance, and project management to dissect the evolving safety landscape of advanced therapies and explore advanced strategies for monitoring emerging safety risks, including the vital role of long-term follow-up through PASS studies and spontaneous reporting. Topics include proactive signal detection approaches, including the use of real-world data, artificial intelligence (AI), and machine learning, as well as the importance of robust traceability systems. Panelists will also share best practices for stakeholder engagement, tailored educational materials, and effective safety communication to promote appropriate use of these therapies.

Watch the webinar to learn how to build a forward-looking pharmacovigilance strategy that ensures the ongoing safe and effective use of these transformative therapies in the post-approval setting.

What you Will Learn

• Clinical insight into the long-term safety monitoring of advanced therapies with case examples of CAR-T and other cell and gene therapies, with a focus on managing immune-related toxicities, interpreting real-world safety signals, and adapting follow-up strategies to diverse patient populations.
• How to design and operationalize effective long-term safety monitoring strategies for advanced therapies, including the use of PASS studies and patient registries.
• Techniques for early identification of emerging safety risks, even in small post-marketing populations, using real-world data and expert clinical insight.
• The role of tailored risk minimization tools and clear safety communication in addressing complex advanced therapy safety profiles — from DHPCs to patient education.
• Key regulatory expectations from EMA, FDA, and MHRA — and how to stay inspection-ready in a field where science evolves faster than guidance

Explore our comprehensive framework and proactive recommendations for long-term safety management challenges in our infographic, Long-Term Safety Strategies for Advanced Therapies: A Proactive Framework