ArticleClinicoEconomics and outcomes research : CEOR2020
Cost-Effectiveness Analysis of Tisagenlecleucel in the Treatment of Relapsed or Refractory B-Cell Acute Lymphoblastic Leukaemia in Children and Young Adults in Spain.
Article in ClinicoEconomics and outcomes research : CEOR, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers, 5 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
12 citing papers in PubMed, 5 syntheses or guidelines pooled it, 27 citations in OpenAlex.
- Cost-Effectiveness of Chimeric Antigen Receptor (CAR) T-Cell Therapy for Blood Cancers: An Updated Systematic Review.PharmacoEconomics - open · 2026Pooled it
- Evolving Evidence-Based Value Assessment of One-Time Therapies: Tisagenlecleucel as a Case Study.Applied health economics and health policy · 2024Pooled it
- A systematic review on the appropriate discounting rates for the economic evaluation of gene therapies: whether a specific approach is justified to tackle the challenges?International journal of technology assessment in health care · 2024Pooled it
- Economic Evidence on Potentially Curative Gene Therapy Products: A Systematic Literature Review.PharmacoEconomics · 2021Pooled it
- Applying State-of-the-Art Survival Extrapolation Techniques to the Evaluation of CAR-T Therapies: Evidence from a Systematic Literature Review.Advances in therapy · 2021Pooled it
- Pharmacoeconomic Profiles of Advanced Therapy Medicinal Products in Rare Diseases: A Systematic Review.Healthcare (Basel, Switzerland) · 2025Review
- Review
- Healthcare costs of cancer among children, adolescents, and young adults: A scoping review.Cancer medicine · 2024Article
- Multidimensional Results and Reflections on CAR-T: The Italian Evidence.International journal of environmental research and public health · 2023Article
- The Evolution of Chimeric Antigen Receptor T-Cell Therapy in Children, Adolescents and Young Adults with Acute Lymphoblastic Leukemia.Biomedicines · 2022Review
- Article
- Critical Reflections on Reimbursement and Access of Advanced Therapies.Frontiers in pharmacology · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 4 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
purposeTisagenlecleucel, 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
methodsA 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.
resultsThis 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.
conclusionTisagenlecleucel would represent a cost-effective intervention for the treatment of children and young adults with r/r ALL in Spain.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.