Statistical Considerations in Cell and Gene Therapy Development

The impact of cell and gene therapies (CGT) on patients around the globe continues to expand in the face of rapidly growing scientific discoveries and new approvals. In this half-day short course, presenter(s) from the American Statistical Association's Cell and Gene Therapy Scientific Working Group will provide an overview of statistical considerations in cell and gene therapy development including important concepts such as dose finding, application of the estimand framework, and confirmatory trial design, etc. The course will first provide an introduction of CGT products and the regulatory considerations. It will then focus on dose escalation methods in developing CGT, estimands and intercurrent event strategies for clinical trials as well as long term follow up studies, and trial design aspects including implications of single-arm or randomized studies and the application of real-world data. In addition, the course will also touch on statistical operational aspects of trial conduct and future directions in CGT, such as use of artificial intelligence. The course also plans to have interactive case studies to engage the attendees.