PCN170 - COST-EFFECTIVENESS OF TISAGENLECLEUCEL IN PEDIATRIC AND YOUNG ADULT PATIENTS WITH RELAPSED OR REFRACTORY B-CELL ACUTE LYMPHOBLASTIC LEUKEMIA: A CANADIAN SOCIETAL PERSPECTIVE

Abstract

Tisagenlecleucel, a CD19-directed genetically modified autologous T-cell immunocellular therapy is pending regulatory approval for the treatment of pediatric and young adult patients with relapsed or refractory (r/r) B-cell acute lymphoblastic leukemia (pALL). The current study assessed the cost-utility of tisagenlecleucel in comparison with salvage chemotherapy for the treatment of r/r B-cell pALL from a Canadian societal perspective. The model included three health states: event free survival (EFS), progressive disease, and death. Using the partitioned survival model approach, the proportions of patients in each health state were determined by the area under the curves based on the fitted overall survival (OS) and EFS trial curves. The model used literature of ALL long-term survivors to inform the mortality risk after year 5 and conservatively assumed no difference in mortality risk across treatments. A 70-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 Tisagenlecleucel was associated with incremental life year (LY) gains of 13.46 years, incremental quality-adjusted life years (QALYs) gains of 11.74 years and an incremental cost of $135,758 when compared to salvage chemotherapy. The incremental cost-effectiveness ratio (ICER) for tisagenlecleucel vs. salvage chemotherapy was $11,567 per QALY gained. Deterministic and probabilistic sensitivity analyses supported the base-case findings and contributed to the robustness of the economic case. Tisagenlecleucel represents a clinically significant advancement in the management of r/r B-cell pALL and fulfils the unmet needs for this difficult-to-treat patient population. Compared with the current standard of care, tisagenlecleucel greatly prolonged survival, EFS, improved quality of life and is a cost-effective option for the treatment of r/r B-cell pALL.