Tisagenlecleucel 1.2million-600million cells dispersion for infusion bags

BackgroundChimeric antigen receptor (CAR) T-cell therapy is an area of rapid development, showing the promise of curing blood cancers. While substantial health gains may justify high costs, it is currently unclear the extent to which the overall cost effectiveness of these therapies is driven by i) context-specific factors, such willingness-to-pay thresholds and study perspective, or ii) important subgroups such as line of treatment and therapy product.ObjectiveThis paper aims to critically review published evidence on the cost effectiveness of CAR T-cell therapies and assess the key factors that drive their cost effectiveness.MethodsWe conducted a systematic review using PubMed, Scopus and Ovid (Embase) databases to identify full economic evaluations of CAR T-cell therapies published up to January 2024. One reviewer screened and extracted data from the studies and the second reviewer assessed a sample of the full-text studies against the inclusion/exclusion criteria. Studies were critically appraised using the CHEERS checklist. Cost data are presented in 2022 US dollars.ResultsThe review identified 45 full cost-effectiveness studies of CAR T-cell therapies. These studies considered a total of 92 treatment comparisons, which included tisagenlecleucel (n = 37), axicabtagene ciloleucel (n = 28), brexucabtagene autoleucel (n = 7), lisocabtagene maraleucel (n = 8), idecabtagene vicleucel (n = 6), ciltacabtagene autoleucel (n = 4) and relmacabtagene autoleucel (n = 2). Incremental cost ranged from - US$74,980 to US$714,178 and incremental quality-adjusted life year (QALY) gains ranged from - 0.02 to 10.77. The resulting cost-per-QALY-gained ratios ranged from - US$37,490,000 to US$7,972,845, and the range of willingness-to-pay (WTP) thresholds between US$36,184 to US$317,825. The price of CAR T-cell therapy represented 75% (mean US$391,060) of the total cost of CAR T-cell therapy but was not the sole factor influencing cost effectiveness. Hospitalisation made up 6% of the total cost (mean US$34,152), while adverse events accounted for 9% (mean US$47,350). Regression analysis indicated cost effectiveness did not change according to important clinical or contextual factors.ConclusionsThe findings demonstrate that the cost effectiveness of CAR T-cell therapies is determined by a combination of factors: the relative difference between the cost of the CAR T-cell therapy and comparator, the magnitude of the QALY gains and the WTP thresholds. Their cost- effectiveness does not differ according to therapy product, line of treatment, or country.

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Background and aim: Advanced Therapy Medicinal Products (ATMPs) are innovative drugs based on genes, tissues, or cells that target rare and severe diseases. ATMPs have shown promising clinical outcomes but are associated with high costs, raising questions about cost-effectiveness. Hence, this systematic review aims to analyze the cost-effectiveness and cost-utility profiles of the European Medicines Agency-authorized ATMPs for treating rare diseases. Methods: A systematic review was conducted following PRISMA guidelines. Studies were identified by searching PubMed, Embase, Web of Science, and ProQuest scientific databases. Economic evaluations reporting incremental cost-effectiveness/utility ratios (ICERs/ICURs) for ATMPs were included. Costs were standardized to 2023 Euros, and a cost-effectiveness plane was constructed to evaluate the results against willingness-to-pay (WTP) thresholds of EUR 50,000, EUR 100,000, and EUR 150,000 per QALY, as part of a sensitivity analysis. Results: A total of 61 studies met the inclusion criteria. ATMPs for rare blood diseases, such as tisagenlecleucel and axicabtagene ciloleucel, were found to be cost-effective in a majority of studies, with incremental QALYs ranging from 1.5 to 10 per patient over lifetime horizon. Tisagenlecleucel demonstrated a positive cost-effectiveness profile in the treatment of acute lymphoblastic leukemia (58%), while axicabtagene ciloleucel showed a positive profile in the treatment of diffuse large B-cell lymphoma (85%). Onasemnogene abeparvovec for spinal muscular atrophy (SMA) showed uncertain cost-effectiveness results, and voretigene neparvovec for retinal diseases was not cost-effective in 40% of studies, with incremental QALYs around 1.3 and high costs exceeding the WTP threshold set. Conclusions: ATMPs in treating rare diseases show promising economic potential, but cost-effectiveness varies across indications. Policymakers must balance innovation with system sustainability, using refined models and the long-term impact on patient outcomes.

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Background and objectivesLarge B-cell lymphoma (LBCL) is a common and aggressive non-Hodgkin lymphoma (NHL) characterized by abnormal proliferation of mature B lymphocytes, with a 10-year prevalence of up to 45 cases per 100,000 individuals in European populations and a continuing upward trend. Axicabtagene ciloleucel (Axi-cel) and Tisagenlecleucel (Tisa-cel) are the two most established chimeric antigen receptor T-cell (CAR-T) therapy products for LBCL, yet a systematic comparison of their efficacy and safety in European populations has been lacking. This systematic review and meta-analysis aims to address this gap by comprehensively evaluating differences in treatment efficacy and adverse events between Axi-cel and Tisa-cel in European patients with LBCL.Materials and methodsWe searched PubMed, Cochrane Library, Scopus, and other databases for studies published between January 2020 and February 2026, ultimately including eight European cohort studies with a total of 2,178 patients. Efficacy and survival outcomes comprised 3-month overall response (OR), 3-month complete response (CR), 12-month progression-free survival (PFS), and 12-month overall survival (OS). Toxicity outcomes included 12-month non-relapse mortality (NRM), all-grade and grade ≥ 3 cytokine release syndrome (CRS), all-grade and grade ≥ 3 immune effector cell-associated neurotoxicity syndrome (ICANS), all-grade and grade ≥ 3 neutropenia, thrombocytopenia, and anemia, as well as tocilizumab use and ICU support. A random-effects model was used for pooled analysis, with sensitivity analyses performed for outcomes exhibiting substantial heterogeneity.ResultsRegarding efficacy, Axi-cel was associated with significantly higher 3-month OR and 3-month CR rates compared with Tisa-cel, whereas 12-month PFS was lower in the Axi-cel group. In terms of toxicity, the incidences of all-grade CRS, all-grade ICANS, and grade ≥ 3 ICANS were significantly higher with Axi-cel, and tocilizumab use was more frequent. No statistically significant differences were observed for the remaining outcomes.ConclusionsIn European patients with LBCL, Axi-cel demonstrated superior short-term efficacy compared with Tisa-cel, but showed inferior performance on certain intermediate-term efficacy endpoints and was generally associated with greater toxicity and higher healthcare resource utilization. Clinical selection of a CAR-T regimen should be individualized, with careful consideration of efficacy, toxicity, and cost. Future high-quality studies with longer follow-up are needed to evaluate long-term outcomes and to validate the findings of this analysis.

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BackgroundAdvanced cell therapies often face high parameter uncertainty at launch, prompting calls to collect further data about long-term effectiveness and safety. However, the value of collecting these data to support resource allocation decision-making is not known. Therefore, this study aimed to appraise all published value of information analyses for advanced cell therapies.MethodsA systematic review (PROSPERO: CRD42023446874) identified value of information analyses for advanced cell therapies between inception and 14 May 2025 (databases: Medline; Embase). Included studies reported the expected value of perfect information (EVPI), expected value of partial perfect information (EVPPI), expected value of sample information (EVSI) or expected net benefit of sampling (ENBS). Study design and value of information results were summarised in a narrative synthesis. Quality of reporting was assessed using the Consolidated Health Economic Evaluation Reporting Standards Value of Information (CHEERS-VOI) checklist.ResultsThree published value of information analyses were identified: tisagenlecleucel for relapsed/refractory acute lymphoblastic leukemia; tisagenlecleucel for relapsed/refractory diffuse large B-cell lymphoma, and tumor infiltrating lymphocyte cell therapy for advanced melanoma. The beneficiary populations were 6, 36, and 400 individuals per year, respectively. All studies reported EVPI; two studies reported EVPPI. Estimated base case population EVPI was: €314,455, €0, and €2,250,000, respectively. Estimated EVPPI indicated that input parameters for survival extrapolations were the most valuable targets for further research specifically during scenario analyses that explored a lower cost of treatment acquisition.ConclusionsValue of information analyses will help decision-makers, analysts, and manufacturers understand whether long-term data collection is worthwhile to reduce decision uncertainty for advanced cell therapies at launch. Current estimates indicated that the value of further research is likely to be low. The value of collecting additional data will likely increase if future advanced cell therapies are priced such that their corresponding incremental cost-effectiveness ratio aligns with a relevant cost-effectiveness threshold and if they are indicated for larger beneficiary populations.

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BackgroundIndividuals diagnosed with relapsed or refractory large B-cell lymphoma (R/R LBCL) typically exhibit a dismal prognosis when treated with conventional therapeutic modalities. CD19-targeted chimeric antigen receptor T (CAR-T) cell therapy has brought about a paradigm shift in the treatment paradigm of this disease. Nevertheless, a comprehensive assessment of the efficacy and safety profiles of diverse CAR-T products (e.g., axicabtagene ciloleucel (axi-cel), tisagenlecleucel (tisa-cel), and lisocabtagene maraleucel (liso-cel)) is imperative.ObjectiveThis systematic review aims to systematically summarize and evaluate the efficacy and safety of CAR T-cell therapies for R/R LBCL through systematic review and meta-analytic approaches.DesignThis is a systematic review and meta-analysis.Data sources and methodsRelevant studies were identified by systematically searching PubMed and Web of Science until November 29, 2023. Cohort studies and clinical trials were incorporated, with the inclusion of single-arm studies. CAR T-cell therapies are involved in tisa-cel, axi-cel, and liso-cel. Proportions and their 95% confidence intervals were calculated using standard meta-analytic approaches.ResultsThirty-seven studies were included in meta-analyses, liso-cel demonstrated equivalent overall survival rates (70.3%) to axi-cel (65.6%) at 12 months post-treatment, whereas tisa-cel was 48.0%. Objective response rate for liso-cel was comparable to axi-cel (79.0% vs 76.8%, p meta-regression = 0.74), both of which were notably higher than those observed for tisa-cel (58.3%, both p meta-regression ConclusionBased on the available evidence, liso-cel has shown promising efficacy and a manageable safety profile in patients with R/R LBCL, when compared to axi-cel and tisa-cel. However, real-world data on liso-cel are limited.

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