Lessons Learned and Challenges in the Future of CGTs

Because autologous CGT products may involve multiple study phases, including receipt of bridging therapies to maintain stable disease, this creates an interesting challenge in identifying the research question of interest and in analyzing the data. Are we interested in the effect of the CGT treatment as an entire regimen or the effect of receiving the product itself? In addition, allowing a CGT product as a crossover option after failure of standard of care is different than changing a drug within a typical SOC regimen for example, and this brings its own challenges in presenting safety and efficacy data after crossover. In this presentation, a regulatory clinician will present lessons that have arisen in past reviews of CGT products and discuss how they can be applied to the future.

CGT

cell and gene therapy

cell therapy

gene therapy

future directions

lessons learned