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.
Lasse Hjort Kyneb JakobsenTorbjörn CallréusMaurizio SessaMats JerkemanMorten AndersenTarec Christoffer El-GalalyPublished in: Pharmacoepidemiology and drug safety (2021)
Projections of the statistical power for detecting important deviations in efficacy/safety from that reported in pivotal clinical trials(s) provide critical information about the expected performance of post-authorization monitoring programs.