Die „Target Trial Emulation“ zu CAR-T im AMNOG

Abstract

The concept of ‚target trial emulation‘ and propensity score-based adjustment procedures are recommended methods for minimising systematic bias in indirect comparisons without a bridging comparator. Based on the comparison of the single-arm evidence for the CAR-T Ciltacel with data from the LocoMMotion study, which was recognised in the medical community as the best prospective study on the standard of care for multiple myeloma with multiple prior treatments, the application of the concept in the AMNOG is presented. The comparison and the dramatic survival benefit shown with Ciltacel, the validity and clinical significance of which is confirmed by medical experts, contrasts with the complete rejection by the G-BA. This discrepancy is worth looking back on.