OBJECTIVES : Pediatric B-cell precursor acute lymphoblastic leukemia (BCP-ALL) is an extremely rare disease, and while it has a high cure rate with first-line therapy, it will relapse in 15% to 20% of patients and a further 2% will have refractory disease. Current treatment options for pediatric relapsed or refractory BCP-ALL are extremely limited, and the disease is considered to be life-threatening and associated with high unmet need. The main aim of this study was to perform cost-utility model comparing blinatumomab with standard of care (SoC) for treating paediatric patients with relapsed or refractory Philadelphia chromosome negative B-precursor acute lymphoblastic leukaemia (R/R Ph- BCP-ALL) in Russia.

METHODS : Health outcomes were expressed in terms of life-years (LYs) and quality-adjusted life-years (QALYs) gained. Cost estimates were presented as aggregated total costs, costs by components (i.e. medication costs (blinatumomab and SoC), inpatient and outpatient costs, allo-SCT-related costs, subsequent treatment costs, adverse events costs, terminal care costs and indirect costs). Incremental cost-effectiveness ratios (ICERs) were calculated and expressed as Incremental cost per LY gained and Incremental cost per QALY gained, by combining health outcomes with cost data for competing treatment alternatives. The analysis was conducted from the social perspective including direct and indirect costs. The evaluation uses a partitioned survival model with states defined on the basis of response to treatment, relapse, and death. The model was programmed in Microsoft Excel.

RESULTS : Blinatumomab was both more effective (greater life-years and QALYs) and less costly than SoC. Total costs were estimated to be $6518 lower with blinatumomab than with SoC chemotherapy. The resulting ICER was $1857 per QALY gained and $1640 per LY gained. Consequently, blinatumomab is dominant (i.e. less expensive and more efficacious) (rate for June 2019).

CONCLUSIONS : Using blinatumomab for treating R/R Ph- BCP-ALL was effective and economically justified treatment option in Russia.