Future-Proofing Long-Term Follow-Up in Cell and Gene Therapy

This white paper discusses the complexities and challenges associated with long-term follow-up (LTFU) studies in cell and gene therapies (CAGTs). It outlines the necessity of these studies as a regulatory requirement to ensure the sustained efficacy and safety of therapies over time. The paper identifies key challenges such as patient engagement over extended periods, geographic dispersion of patients, fragmented data collection, and the evolving health status of patients. It emphasizes the importance of a patient-centered and data-driven approach to address these issues. The authors present strategies to overcome these challenges, including leveraging real-world data and technology, designing adaptable study protocols, and integrating trial and post-commercial follow-up efforts. The overarching theme is the need for innovative methodologies that ensure the protection of patients while advancing scientific understanding and maintaining operational efficiency in LTFU studies.