Josep Maria Ribera Santasusana,1 Alejandra de Andrs Saldaa,2 Nuria Garca-Muoz,3 Joana Gostkorzewicz,2 Diana Martnez Llins,3 Cristina Daz de Heredia4

1Clinical Hematology Department, Catalan Institute of Oncology - Hospital Germans Trias i Pujol, Barcelona, Spain; 2Health Economics and Outcomes Research, Novartis Farmacutica S.A., Madrid, Spain; 3Oblikue Consulting, S.L., Barcelona, Spain; 4Paediatric Oncology and Hematology Department - Hematopoietic Stem Cell Transplantation, Hospital Universitari Vall dHebron, Barcelona, Spain

Correspondence: Diana Martnez LlinsOblikue Consulting, S.L., C/Comte dUrgell, 240, 2-D, Barcelona 08036, SpainTel +34 93 252 1377Fax +34 93 737 9984Email diana.martinez@oblikue.com

Purpose: Tisagenlecleucel, a chimeric antigen receptor T-cell (CAR-T) therapy, is a promising alternative for the management of children and young adults with relapsed and refractory B-cell acute lymphoblastic leukemia (r/r ALL). The aim of this study was to determine whether treatment with tisagenlecleucel is a cost-effective intervention compared with salvage chemotherapy in paediatric and young adult patients with r/r ALL in Spain.Materials and Methods: A partitioned survival model of monthly cycles with three health states was used (event-free survival, progressive/relapsed disease and death). A lifetime time horizon and the Spanish National Health System perspective were adopted. During the first 5 years, permanence in the different health states was determined according to the results in the clinical studies. In successive years, mortality tables of the Spanish general population adjusted by standardized mortality rate for survivors of childhood cancer were used. Clinical, economic, and quality of life parameters were drawn from clinical trials and the literature. Only direct health costs (pharmacological costs and the costs derived from health resource use) were included. The robustness of the results was evaluated in a sensitivity analysis.Results: This cost-effectiveness analysis showed a greater benefit (10.10 and 8.97 life-years gained [LYGs] and quality-adjusted life-years [QALYs] gained, respectively) and a higher cost ( 258,378.40) for tisagenlecleucel compared to salvage chemotherapy. The resulting incremental cost-effectiveness and cost-utility ratios were 25,576.80 per LYG and 28,818.52 per QALY gained, respectively. In the sensitivity analysis, all the results were below 50,000/QALY.Conclusion: Tisagenlecleucel would represent a cost-effective intervention for the treatment of children and young adults with r/r ALL in Spain.

Keywords: ALL, cost-effectiveness, tisagenlecleucel, acute lymphoblastic leukaemia, Spain, CAR-T

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

See the original post:
Cost-Effectiveness Analysis of Tisagenlecleucel in the Treatment of Re | CEOR - Dove Medical Press