OBJECTIVES: Tisagenlecleucel, a CD19-directed genetically modified autologous T-cell immunocellular therapy is pending regulatory approval for the treatment of adult patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL). The current study aimed to assess the cost-utility of tisagenlecleucel in comparison with salvage chemotherapy for the treatment of r/r DLBCL from a Canadian societal perspective.

METHODS: The model included three health states: progression-free survival (PFS), progressive/relapsed disease (PD/RL), and death. The estimation of patients occupying each health state was derived directly from the observed cumulative survival probabilities of PFS, overall survival (OS), and extrapolated using parametric functions after the observed periods. Conservatively, the model assumed no additional survival benefit for tisagenlecleucel and considered the same mortality rate for both arms after the end of trial periods. A 20-year time horizon was used to capture all the relevant costs and benefits associated with the treatments. Both costs and benefits were discounted at 1.5% per year. The model comparator was salvage chemotherapy. Costs, health state utility and treatment-related disutility inputs were obtained from Canada-specific databases and literature. The benefit endpoints evaluated were quality-adjusted life years (QALYs) and life years (LYs).

RESULTS: Tisagenlecleucel was associated with 2.81 LY gains, 2.98 QALY gains and an incremental cost of $307,371 when compared to salvage chemotherapy. The incremental cost-effectiveness ratio (ICER) for tisagenlecleucel vs. salvage chemotherapy was $109,461 per LY gained and $103,112 per QALY gained. Deterministic and probabilistic sensitivity analyses supported the base-case findings and contributed to the robustness of the economic case.

CONCLUSIONS: Tisagenlecleucel represents a clinically significant advancement in the management of r/r DLBCL and fulfils the unmet needs for this difficult-to-treat patient population. Compared with the current standard of care, tisagenlecleucel greatly prolonged survival, PFS, improved quality of life, and likely is a cost-effective option for treatment of r/r DLBCL.