CAR-T cell therapy: Contrasting the evidence from pivotal trials with the real world evidence (RWE)

Project Lead: Claudia Wild
Auhors: Andrea Titieni-Schuhmann, Anja Panhuber, Gregor Götz
Publication: October 2022
Language: English (with German summary)

The two CAR-T cell therapies Kymriah® and Yescarta® were approved by the EMA in 2018 for patients with B-cell acute lymphoblastic leukaemia (B-ALL), diffuse large B-cell lymphoma (DLBCL) or primary mediastinal B-cell lymphoma (PMBCL) in third line. Treatment is often associated with high expectations, but the evidence on efficacy and safety of these novel therapies is still uncertain. The aim of this systematic review was to contrast the results of patient characteristics as well as selected efficacy and safety endpoints from the pivotal trials with those from real-world evidence studies. In addition, the results and conclusions of other HTA reports were summarised regarding the available evidence on efficacy and safety.

 

AIHTA Project Report No. 146