OBJECTIVES: Tisagenlecleucel, an anti-CD19 chimeric antigen receptor T-cell therapy (CAR-T), was recently approved for the treatment of paediatric and young adult patients up to 25 years of age with B-cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse. The one-time infusion drug cost of tisagenlecleucel is at 320,000 Euros and the economic value has not been evaluated in Greece. The current economic analysis assessed the cost-utility of Tisagenlecleucel in comparison with salvage chemotherapy for the treatment of relapsed or refractory (r/r) paediatric and young adult ALL, from a Greek social security system perspective.

METHODS: A Markov model was used to evaluate tisagenlecleucel in r/r paediatric and young adults ALL from a Greek social security system perspective over 70 year lifetime horizon. Tisagenlecleucel was compared with salvage chemotherapy. The model included three health states: event free survival (EFS), progressive disease and death, and for Tisagenlecleucel it was informed by one pivotal (ELIANA) and two supportive (ENSIGN and B2101J), single-arm clinical trials. Long-term survival was estimated using historical data for pALL survivors. Only direct health care costs have been included. Future cost and effects were discounted at 3.5%. Deterministic and probabilistic sensitivity analyses were conducted to explore uncertainty. Main outcomes were life-years (LYs), discounted lifetime costs, discounted quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER).

RESULTS: Compared to salvage chemotherapy, tisagenlecleucel improved effectiveness by 9.50 LYs and 8.36 QALYs, increasing overall cost by 291,888 Euros. The ICER for Tisagenlecleucel vs salvage chemotherapy was 30,719 Euros per LY gained and 34,917 euros per QALY gained. Deterministic and probabilistic sensitivity analyses supported the base-case findings.

CONCLUSIONS: When compared with salvage chemotherapy, CAR-T therapy tisagenlecleucel shows a potentially acceptable cost-effective option for the treatment of r/r paediatric and young adult B-cell ALL in Greece.