Keywords: Kymriah, CAR-T, Estimand
The recent surge of CAR-T cell therapy has revealed a great potential of such revolutionary cancer treatments over standard chemotherapy and the big challenges of appropriately designing and estimating the treatment effect in clinical trials. The scientific question of interest the trial is aimed at must be carefully thought through. In the context of a randomized study design, the existence of personalized manufacturing prior to patients receiving the CAR-T infusion add another layer of complexity when specifying the estimands of interest, selecting the most appropriate method to quantify and interpret the size of the corresponding relative treatment. In this presentation, we illustrate our thoughts using an example of CAR-T Phase III clinical trial.