Summary of US Food and Drug Administration Chimeric Antigen Receptor T-Cell Biologics License Application Approvals From a Statistical Perspective
Cet article passe en revue les produits biologiques à base de lymphocytes CAR-T ayant été autorisés par la "Food and Drug Administration" avant août 2021 puis présente les différents facteurs à prendre en compte pour la conception et l'analyse statistique des essais cliniques évaluant ce type de traitements
The approval of tisagenlecleucel and axicabtagene ciloleucel in 2017 marked a milestone in the development of oncology therapies. Since 2017, the breakthrough in treatment or even cure of previously intractable diseases represented by this new class of cancer treatments has continued with subsequent chimeric antigen receptor T (CAR T)-cell approvals. To date, the US Food and Drug Administration has approved five autologous CAR T-cell products for seven indications. A feature of autologous CAR T-cell products that differentiates them from traditional oncology drugs is that they need to be manufactured specifically for each patient. This feature has implications in study design, statistical analyses, and interpretation of study results. In this article, we share our experiences in the statistical review of CAR T-cell products and provide considerations for the design and statistical analyses of CAR T-cell trials. We also describe how the newly adopted estimand framework for clinical trials can help clarify nuanced issues in CAR T-cell trial design.
Journal of Clinical Oncology , résumé, 2021