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

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)....

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Autores principales: Caillon, Megane, Brethon, Benoit, van Beurden-Tan, Chrissy, Supiot, Romain, Le Mezo, Antoine, Chauny, Jean-Vannak, Majer, Istvan, Petit, Arnaud
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10333169/
https://www.ncbi.nlm.nih.gov/pubmed/37071263
http://dx.doi.org/10.1007/s41669-023-00411-4
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author Caillon, Megane
Brethon, Benoit
van Beurden-Tan, Chrissy
Supiot, Romain
Le Mezo, Antoine
Chauny, Jean-Vannak
Majer, Istvan
Petit, Arnaud
author_facet Caillon, Megane
Brethon, Benoit
van Beurden-Tan, Chrissy
Supiot, Romain
Le Mezo, Antoine
Chauny, Jean-Vannak
Majer, Istvan
Petit, Arnaud
author_sort Caillon, Megane
collection PubMed
description 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. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1007/s41669-023-00411-4.
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spelling pubmed-103331692023-07-12 Cost-Effectiveness of Blinatumomab in Pediatric Patients with High-Risk First-Relapse B-Cell Precursor Acute Lymphoblastic Leukemia in France Caillon, Megane Brethon, Benoit van Beurden-Tan, Chrissy Supiot, Romain Le Mezo, Antoine Chauny, Jean-Vannak Majer, Istvan Petit, Arnaud Pharmacoecon Open Original Research Article 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. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1007/s41669-023-00411-4. Springer International Publishing 2023-04-18 /pmc/articles/PMC10333169/ /pubmed/37071263 http://dx.doi.org/10.1007/s41669-023-00411-4 Text en © The Author(s) 2023 https://creativecommons.org/licenses/by-nc/4.0/Open AccessThis article is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License, which permits any non-commercial use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by-nc/4.0/ (https://creativecommons.org/licenses/by-nc/4.0/) .
spellingShingle Original Research Article
Caillon, Megane
Brethon, Benoit
van Beurden-Tan, Chrissy
Supiot, Romain
Le Mezo, Antoine
Chauny, Jean-Vannak
Majer, Istvan
Petit, Arnaud
Cost-Effectiveness of Blinatumomab in Pediatric Patients with High-Risk First-Relapse B-Cell Precursor Acute Lymphoblastic Leukemia in France
title Cost-Effectiveness of Blinatumomab in Pediatric Patients with High-Risk First-Relapse B-Cell Precursor Acute Lymphoblastic Leukemia in France
title_full Cost-Effectiveness of Blinatumomab in Pediatric Patients with High-Risk First-Relapse B-Cell Precursor Acute Lymphoblastic Leukemia in France
title_fullStr Cost-Effectiveness of Blinatumomab in Pediatric Patients with High-Risk First-Relapse B-Cell Precursor Acute Lymphoblastic Leukemia in France
title_full_unstemmed Cost-Effectiveness of Blinatumomab in Pediatric Patients with High-Risk First-Relapse B-Cell Precursor Acute Lymphoblastic Leukemia in France
title_short Cost-Effectiveness of Blinatumomab in Pediatric Patients with High-Risk First-Relapse B-Cell Precursor Acute Lymphoblastic Leukemia in France
title_sort cost-effectiveness of blinatumomab in pediatric patients with high-risk first-relapse b-cell precursor acute lymphoblastic leukemia in france
topic Original Research Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10333169/
https://www.ncbi.nlm.nih.gov/pubmed/37071263
http://dx.doi.org/10.1007/s41669-023-00411-4
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