Detecting deviations from the efficacy and safety results of single-arm trials using real-world data : The case of a CAR-T cell therapy in B-cell lymphoma
Purpose: Personalized therapies are leading to an increasing number of marketing authorizations based on single-arm trials, which increases the demand for better post-authorization monitoring strategies. The aim of the present study was to estimate the power over time as data accrue in population-based registries for detecting deviations from the expected efficacy/safety of chimeric antigen recept