Important Considerations to Optimize Long-term Follow-up Studies for Cell and Gene Therapies
Cell and gene therapies (CGTs) have shown promise in treating various diseases like cancer, genetic syndromes, autoimmune diseases, and neurological disorders.
Gene therapy corrects disease-causing gene defects, while cell therapy introduces functioning cells to treat diseases. Both approaches offer long-term benefits, but safety and efficacy must be thoroughly evaluated before widespread use. As a result, CGTs usually require long-term follow-up (LTFU) studies starting in Phase I clinical trials. The number of CGTs is increasing, with the gene, cell, and RNA therapy pipeline growing by 6% according to the latest report.
This white paper emphasizes:
The importance of LTFU studies for continuous benefit-risk assessment
Considerations for study design, including regulatory requirements, data collection, patient/site burden, retention, and engagement.
