Cost-Effectiveness of Blinatumomab in Pediatric Patients with High-Risk First-Relapse B-Cell Precursor Acute Lymphoblastic Leukemia in France

Megane Caillon, Benoit Brethon, Chrissy van Beurden-Tan, Romain Supiot, Antoine Le Mezo, Jean-Vannak Chauny, Istvan Majer, Arnaud Petit, Megane Caillon, Benoit Brethon, Chrissy van Beurden-Tan, Romain Supiot, Antoine Le Mezo, Jean-Vannak Chauny, Istvan Majer, Arnaud Petit

Abstract

Background: Based on the results of the phase III randomized 20120215 trial, the European Medicines Agency granted the approval of blinatumomab for the treatment of pediatric patients with high-risk first-relapsed Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (ALL). In France, blinatumomab received reimbursement for this indication in May 2022. This analysis assessed the cost effectiveness of blinatumomab compared with high-risk consolidation chemotherapy (HC3) in this indication from a French healthcare and societal perspective.

Methods: A partitioned survival model with three health states (event-free, post-event and death) was developed to estimate life-years (LYs), quality-adjusted life-years (QALYs) and costs over a lifetime horizon. Patients who were alive after 5 years were considered to be cured. An excess mortality rate was applied to capture the late effects of cancer therapy. Utility values were based on the TOWER trial using French tariffs, and cost input data were identified from French national public health sources. The model was validated by clinical experts.

Results: Treatment with blinatumomab over HC3 was estimated to provide gains of 8.39 LYs and 7.16 QALYs. Total healthcare costs for blinatumomab and HC3 were estimated to be €154,326 and €102,028, respectively, resulting in an increment of €52,298. The incremental cost-effectiveness ratio was estimated to be €7308 per QALY gained from a healthcare perspective. Results were robust to sensitivity analyses, including analysis from the societal perspective.

Conclusions: Blinatumomab administered as part of consolidation therapy in pediatric patients with high-risk first-relapsed ALL is cost effective compared with HC3 from the French healthcare and societal perspective.

Keywords: Acute lymphoblastic leukemia; Blinatumomab; Cost-effectiveness; Economic evaluation; France; Pediatric; Societal perspective.

Conflict of interest statement

Istvan Majer, Megane Caillon and Jean-Vannak Chauny are employees of Amgen. Chrissy van Beurden-Tan and Antoine Le Mézo were employees of Amgen at the time this work was conducted. Benoit Brethon reports receiving honoraria from Amgen SAS. Arnaud Petit was an investigator for the 20120215 trial but received no honoraria for his participation. Romain Supiot has no conflicts of interest to declare.

© 2023. The Author(s).

Figures

Fig. 1
Fig. 1
Modelled event-free survival curves for blinatumomab and HC3. (a) Short-term fit; (b) long-term extrapolation. EFS event-free survival, HC3 high-risk consolidation chemotherapy
Fig. 2
Fig. 2
Modelled overall survival for blinatumomab and HC3. (a) Short-term fit; (b) long-term extrapolation. HC3 high-risk consolidation chemotherapy, OS overall survival
Fig. 3
Fig. 3
Deterministic sensitivity analysis results. Since vial sharing between bags or between patients is not considered in the base-case analysis, the change from a dose of 14.25 μg/day to 13.20 μg/day for blinatumomab therefore has no impact. Regarding the bag change frequency of blinatumomab, the two bounds tested in the DSA (3.5 and 4.0) are higher than the value retained in the base-case analysis (3.0), which explains why the ICER decreases in both cases. This is because changing bags less frequently limits wastage and reduces administration costs. OS overall survival, mg microgram, ICER incremental cost-effectiveness ratio, QALY quality-adjusted life-year, HSCT hematopoietic stem cell transplantation, HC3 high-risk consolidation chemotherapy
Fig. 4
Fig. 4
Probabilistic sensitivity analysis results. (a) Incremental cost-effectiveness plane; (b) cost-effectiveness acceptability curve. QALYs quality-adjusted life-years, PSA probabilistic sensitivity analyses, HC3 high-risk consolidation chemotherapy

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