Long-Term Follow-Up In Gene Therapy Trials: Ensuring Patient Engagement & Regulatory Compliance

By Dr. Susanne Schmidt, Senior Medical Director, Medical Management, and Hanna Wide, Director, Program Delivery, Rare Diseases & Pediatrics

Gene therapy products are typically designed to achieve therapeutic effect through long-acting or permanent changes in the human body. Due to this extended period of exposure, patients who receive gene therapies may be at increased risk of delayed adverse events. To understand and mitigate the risk of these delayed adverse events, participants in gene therapy trials may be monitored for a long-term follow-up (LTFU) period, which may be as long as 15 years. During this period, sponsors are challenged with navigating complex regulatory requirements as well as finding innovative ways to keep patients engaged for a decade or more.