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.
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