Cost-effectiveness of Tisagenlecleucel vs Standard Care in High-risk Relapsed Pediatric Acute Lymphoblastic Leukemia in Canada

Furzer, Jill; Gupta, Sumit; Nathan, Paul C.; Schechter, Tal; Pole, Jason D.; Krueger, Joerg; Pechlivanoglou, Petros

doi:10.1001/jamaoncol.2019.5909

Cited by 37 publications

(48 citation statements)

References 44 publications

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“… 37 Two publications were added following the update search. 38 , 39 The publication by Walton et al 37 presented results from the Incremental Cost-Effectiveness Ratio (ICER) HTA report and is therefore included in the following summary instead of the HTA report. Most studies focused on pALL patients, while 3 publications 40 , 41 , 45 studied relapsed/refractory (r/r) DLBCL as indication.…”

Section: Resultsmentioning

confidence: 99%

Health Economic Aspects of Chimeric Antigen Receptor T-cell Therapies for Hematological Cancers: Present and Future

et al. 2021

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Since 2018, 2 chimeric antigen receptor (CAR) T-cell therapies received approval from the European Medicine Agency, with list prices around 320 000 Euro (€) (EUR) per treatment. These high prices raise concerns for patient access and the sustainability of healthcare systems. We aimed to estimate the costs and budget impact associated with CAR T-cell therapies for current and future indications in hematological cancers from 2019 to 2029. We focused on the former France, Germany, Spain, Italy and the United Kingdom (EU-5) and the Netherlands. We conducted a review of list prices, health technology assessment reports, budget impact analysis dossiers, and published cost-effectiveness analyses. We forecasted the 10-year health expenditures on CAR T-cells for several hematological cancers in selected European Union countries. Nine cost-effectiveness studies were identified and list prices for CAR T-cell therapies ranged between 307 200 EUR and 350 000 EUR. Estimated additional costs for pre- and post-treatment were 50 359 EUR per patient, whereas the incremental costs of CAR T-cell therapy (when compared with care as usual) ranged between 276 086 EUR and 328 727 EUR. We estimated market entry of CAR T-cell therapies for chronic mantle cell lymphoma, follicular lymphoma, chronic lymphocytic leukemia, multiple myeloma, and acute myeloid leukemia in 2021, 2022, 2022, 2022, and 2025, respectively. Cumulative expenditure estimates for existing and future indications from 2019 to 2029 were on average 28.5 billion EUR, 32.8 billion EUR, and 28.9 billion EUR when considering CAR T-cell therapy costs only, CAR T-cell therapy costs including pre- and post-treatment, and incremental CAR T-cell therapy costs, respectively. CAR T-cell therapies seem to be promising treatment options for hematological cancers but the financial burden on healthcare systems in the former EU-5 and the Netherlands will contribute to a substantial rise in healthcare expenditure in the field of hematology.

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Section: Resultsmentioning

confidence: 99%

Health Economic Aspects of Chimeric Antigen Receptor T-cell Therapies for Hematological Cancers: Present and Future

et al. 2021

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“…Using a multi-state survival model, Furzer et al (55), using data of 192 patients from three pooled clinical trials and 118 patients from the cancer registry of Canada, quantified the value of tisa-cel compared with current standard care for eligible pediatric patients with relapsed ALL. Even though the authors were unable to use a randomized clinical trial dataset due to unavailability, they incorporated sensitivity analyses to demonstrate the robustness of their cost utility analytic results.…”

Section: Pharmacoeconomic Modelsmentioning

confidence: 99%

A Systematic Review of the Efforts and Hindrances of Modeling and Simulation of CAR T-cell Therapy

Nukala

Messan

Yogurtcu

et al. 2021

AAPS J

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Chimeric Antigen Receptor (CAR) T-cell therapy is an immunotherapy that has recently become highly instrumental in the fight against life-threatening diseases. A variety of modeling and computational simulation efforts have addressed different aspects of CAR T therapy, including Tcell activation, T-and malignant cell population dynamics, therapeutic cost-effectiveness strategies, and patient survival analyses. In this article, we present a systematic review of those efforts, including mathematical, statistical, and stochastic models employing a wide range of algorithms, from differential equations to machine learning. To the best of our knowledge, this is the first review of all such models studying CAR T therapy. In this review, we provide a detailed summary of the strengths, limitations, methodology, data used, and data lacking in current published models. This information may help in designing and building better models for enhanced prediction and assessment of the benefit-risk balance associated with novel CAR T therapies, as well as with the data collection essential for building such models.

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“…39 We used published literature to determine the healthcare qualityof-life utility experienced by pediatric patients with ALL receiving therapy, in remission, and with relapsed disease. 36,[40][41][42] We estimated that hospitalization for hypersensitivity reactions decreased utility by 20%. The disutility associated with death was calculated based on life expectancy and with remaining life years discounted by 3% per year to determine discounted life expectancy lost due to death.…”

Section: Model Parametersmentioning

confidence: 99%

Premedication prior to PEG‐asparaginase is cost‐effective in pediatric patients with acute lymphoblastic leukemia

McCormick

Lapinski

Friehling

et al. 2021

Pediatric Blood & Cancer

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Background: PEG-asparaginase is critical in pediatric acute lymphoblastic leukemia (ALL) therapy but is highly immunogenic. Severe allergic reactions lead to substitution of further PEG-asparaginase with Erwinia. Erwinia is associated with more frequent dosing, increased expense, and limited availability. Premedication may reduce rates of allergic reactions.Procedures: This Markov model evaluated the cost-effectiveness of three strategies: premedication plus therapeutic drug monitoring (TDM), TDM alone, and no premedication or TDM. We modeled two scenarios: a standard-risk (SR) B-ALL patient receiving two asparaginase doses and a high-risk (HR) patient receiving seven asparaginase doses. The model incorporated costs of asparaginase, premedication, TDM and clinic visits, and lost parental wages associated with each additional Erwinia dose. We incorporated a five-year time horizon with a societal perspective. Outcomes were Erwinia substitutions avoided and differences in quality-adjusted life years (QALYs). Probabilistic and one-way sensitivity analyses evaluated model uncertainty. Results:In both scenarios, premedication was the least costly strategy. In SR and HR scenarios, premedication with monitoring resulted in 8% and 7% fewer changes to Erwinia compared with monitoring alone and 3% and 2% fewer changes compared with no premedication/monitoring, respectively. Premedication resulted in the most QALYs gained in the SR patients. Individual variation of model inputs did not change premedication/monitoring favorability for either scenario. In probabilistic sensitivity analyses, premedication/monitoring was favored in >87% of iterations in both scenarios. Conclusion:Compared with other strategies, premedication use and asparaginase level monitoring in children with B-ALL is potentially cost-saving.

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Cost-effectiveness of Tisagenlecleucel vs Standard Care in High-risk Relapsed Pediatric Acute Lymphoblastic Leukemia in Canada

Cited by 37 publications

References 44 publications

Health Economic Aspects of Chimeric Antigen Receptor T-cell Therapies for Hematological Cancers: Present and Future

Health Economic Aspects of Chimeric Antigen Receptor T-cell Therapies for Hematological Cancers: Present and Future

A Systematic Review of the Efforts and Hindrances of Modeling and Simulation of CAR T-cell Therapy

Premedication prior to PEG‐asparaginase is cost‐effective in pediatric patients with acute lymphoblastic leukemia

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