Planning Real-World Studies for Cell and Gene Therapy

This white paper discusses the planning of real-world studies aimed at supporting evidence generation for cell and gene therapies (CGTs). It outlines the increasing number of CGTs entering clinical trials and the regulatory landscape, including definitions from the U.S. FDA and EMA. The document highlights the complexities associated with CGT studies, such as patient recruitment challenges, the need for direct comparators, and long-term follow-up commitments. It emphasizes the importance of incorporating real-world data (RWD) and real-world evidence (RWE) to address these challenges effectively. The paper details strategies for optimizing patient recruitment through RWD sources and constructing historical control datasets. It also discusses the role of registries in regulatory decision-making and the integration of data from various sources to enhance evidence generation. Furthermore, it addresses the significance of decentralized trial approaches in adapting to patient needs and ensuring robust data collection over long-term commitments.