Real-world data and evidence in the development of cell and gene therapies

Cell and gene therapies (CGTs) are rapidly growing fields in regenerative medicine with the potential to tackle the most difficult-to-treat diseases. Despite their transformative potential, the development of CGTs is inherently complex, resource-intensive, and associated with high costs, posing substantial barriers in development and commercialization. As a feasible approach to address these challenges, real-world data (RWD) and real-world evidence (RWE) are gaining increasing recognition as powerful tools to potentially support and accelerate CGT development. This manuscript aims to identify the potential role of RWE and RWD in CGT development, with a focus on its application in regulatory licensure to date. Furthermore, we discuss current challenges, limitations, and evolving regulatory landscape pertaining to the use of RWE in regulatory decision-making.