Decisions In Cell And Gene Therapy Development

By Tanya Partridge, Head, CAGT Global Site Network and Recruitment solutions, CAGT Center of Excellence, IQVIA; Natalie Sacarakis, Head, CAGT Logistics, CAGT Center of Excellence, IQVIA; Jessica Knight-Perry, Medical Strategy Lead, CAGT Center of Excellence, IQVIA; and Diego Correa, Vice President and Global Head, CAGT Center of Excellence, IQVIA

Cell and gene therapies (CAGTs) represent a transformative approach to treating previously untreatable diseases. As of Q3 2023, there are 3,866 CAGTs in various stages of development, with 53% being gene therapies, 25% RNA therapies, and 22% non-genetically modified cell therapies. Currently, eleven CAGTs are approved for 16 indications in the US, and significant launches are expected in 2024. The global market for these therapies was valued at $22.7 billion in 2023, projected to grow at a CAGR of 28.7% through 2030. Clinical trials for CAGTs involve rare patient populations, complex protocols, high patient and site burden, and long-term safety monitoring, all impacting successful development and commercialization decisions.

This white paper explores critical decisions across clinical trial design, patient recruitment, site selection, logistics, manufacturing, and long-term follow-up, aiming to guide stakeholders through the intricate landscape of CAGT development with foresight and precision.