Designing Successful Cell And Gene Therapy Trials

Cell and gene therapies (CGTs) are revolutionizing medicine with their potential to cure previously untreatable diseases. However, designing clinical trials for these novel therapeutics presents unique challenges due to the complexity of the products themselves and the evolving regulatory landscape. Here we outline key considerations for successful CGT trials, including establishing a strong preclinical foundation and a robust Chemistry, Manufacturing, and Controls (CMC) framework. We also emphasize the importance of a patient-centric approach to trial design, which includes dynamic informed consent processes, and highlight the need for specialized study designs like adaptive trials and basket trials.

Navigating the complexities of CGT trials requires early engagement with regulators and strategic partnerships with experienced contract research organizations (CROs) to ensure scientific rigor, regulatory compliance, and successful outcomes. Explore strategic recruitment methods and ways to minimize patient and site burden.