Gene therapy long-term follow-up studies: Regulatory, Scientific and Operational Requirements, Challenges and Opportunities (webinar)
Cell and gene therapies (CGT) often require long-term follow-up (LTFU) studies of safety and effectiveness once they are approved, with up to 15 years of follow-up per patient. While extension studies of clinical trials can also play a role, real-world data (RWD) are especially relevant to enable the required duration of follow-up and the collection of specific data elements. However, the use of RWD still leaves important challenges for the design and conduct of LTFU studies of cell and gene therapies. Such challenges can be related to sample size, data capture and data quality, among others, potentially more than for other studies involving RWD. This webinar aims to enhance understanding of regulatory, scientific, and operational requirements and considerations for LTFU studies from a global perspective. Examples will be given of challenges with use of RWD and how novel approaches (such as decentralized studies and the use of tokenization) may support capture of crucial data for these studies.
Speaker names: Lisette Hoogendoorn, Jamie Geier, Nick Honig
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Gene therapy long-term follow-up studies: Regulatory, Scientific and Operational Requirements, Challenges and Opportunities