Optimize Long-term Follow-up for CGTs

Cell and gene therapies have incredible potential, but there are distinct considerations to evaluate their safety and efficacy before widespread use. Their unique quality of being single-dose treatments means that almost all CGTs require long-term follow-up (LTFU) studies starting in Phase I clinical trials.

This paper highlights:

• the importance of LTFU studies in continuous benefit-risk assessment
• factors to consider in study design, including regulatory requirements, data collection, patient and site burden, and retention and engagement