Opportunities and challenges associated with the evaluation of chimeric antigen receptor T cells in real-life.

ABSTRACT

PURPOSE OF REVIEW With the approval of the first chimeric antigen receptor (CAR)-T cell products on the market, the European Medicines Agency (EMA) required market authorization holders (MAHs) to monitor the long-term efficacy and safety of CAR-T cells for 15 years after administration. In 2019, the cellular therapy module of the European Society for Blood and Marrow Transplantation (EBMT) registry received a positive qualification opinion from the EMA indicating that the registry fulfills the essential needs to capture such data. We investigated its broader implication. RECENT FINDINGS: Since 2020, the cellular therapy module of the EBMT registry captures data to support postauthorization studies for MAHs and EMA. The process toward a positive qualification opinion has attracted interest from many other stakeholders, such as scientists and Health Technology Assessment bodies, and was the spin-off for a stimulating development which defined the need for a registry to comply with regulatory requirements, and also inspired ways to deal with CAR-T cell programs in terms of center qualifications and educational standards for professionals. SUMMARY: The positive qualified opinion of the EBMT registry by EMA to monitor long-term efficacy and safety of commercial CAR-T cells created opportunities and challenges and was serving as linking-pin to launch a novel CAR-T cell community.