When developing a long-term follow-up strategy for gene therapy trials, safety is the main concern, closely followed by the patient perspective. Digital technologies and monitoring have proven essential to this, easing the burden on the patient, and providing accurate, time-saving methods of data management.
Momentum is increasing in the field of gene therapy, with over 20 products approved and more than 2,000 clinical trials underway worldwide (1). By the year 2025, it is expected that the FDA will be approving 10 to 20 gene therapies per year (2). While gene therapy offers the potential for a cure, it also affects long-acting or permanent changes in the human body. This prolonged period of exposure can increase the risk of delayed adverse events. To understand and mitigate this risk, sponsors may be required to monitor participants of gene therapy trials for a long-term follow-up (LTFU) period of up to 15 years. During this extended period, sponsors will be challenged with not only navigating regulatory requirements and covering the cost of ongoing monitoring but also keeping patients engaged with their studies.