SC07 - Cell and Gene Therapy: Introduction and Overview of Important Regulatory, Statistical, and Operational Considerations

Room: Salon C 

As the science of advanced therapy medicinal products continues to evolve, the impact of cell and gene therapies (CGT) on patients around the globe is increasing. 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 on cell and gene therapy concepts and current industry status, as well as highlight important regulatory, statistical (e.g., dose finding, single arm and randomized designs, using historical information in clinical trials, estimand, etc.), and operational considerations during the clinical development of CGT. The course will also touch upon the impact of data science, digital technologies and artificial intelligence (AI) on current developments and in the future of cell and gene therapies. These may include AI for understanding potential drug targets, better patient identification and recruitment, decentralization of long term follow up, and dissemination of results. The course will conclude with two case studies covering a real example as well as a hypothetical scenario to engage the attendees.

Outline
• Introduction to cell & gene therapy
• Status of regulatory programs & guidance
• Considerations for study designs & protocols
o Dose escalation
o Single arm vs. randomized trials
o Estimands
• Long term follow up
• Benefit-Risk evaluation
• RWE and registry studies
• Operational Considerations (CRF, analysis considerations, etc.)
• Potential for Artificial Intelligence in CGT
• Case Study #1 (approved product)
• Case Study #2 Interactive hypothetical example

CGT

cell therapy

gene therapy

CAR

NK 

Clinical Trial Design (e.g., Innovative/Complex Design, Estimands, Master Protocol)

ASA Biopharmaceutical Section Regulatory-Industry Statistics Workshop 2024