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COST-EFFECTIVENESS ANALYSIS OF AXICABTAGENE CILOLEUCEL VS TISAGENLECLEUCEL FOR THE MANAGEMENT OF DIFFUSE LARGE B-CELL LYMPHOMA IN SPAIN
Author(s): ,
Mariana Bastos-Oreiro
Affiliations:
Hematology,Hospital Universitario Gregorio Marañón,Madrid,Spain
,
María Presa
Affiliations:
Pharmacoeconomics,Pharmacoeconomics&Outcomes Research Iberia (PORIB),Madrid,Spain
,
Ana de las Heras
Affiliations:
Pharmacoeconomics,Pharmacoeconomics&Outcomes Research Iberia (PORIB),Madrid,Spain
,
Miguel Ángel Casado
Affiliations:
Pharmacoeconomics,Pharmacoeconomics&Outcomes Research Iberia (PORIB),Madrid,Spain
,
Carlos Pardo
Affiliations:
Market Access,Gilead Sciences,Madrid,Spain
,
Victoria Martín-Escudero
Affiliations:
Market Access,Gilead Sciences,Madrid,Spain
Anna Sureda
Affiliations:
Hematology,Institut Català d’Oncologia-Hospitalet, IDIBELL, Universitat de Barcelona,Barcelona,Spain
EHA Library. Pardo C. 06/09/21; 324888; EP1167
Carlos Pardo
Carlos Pardo
Contributions
Abstract
Presentation during EHA2021: All e-poster presentations will be made available as of Friday, June 11, 2021 (09:00 CEST) and will be accessible for on-demand viewing until August 15, 2021 on the Virtual Congress platform.

Abstract: EP1167

Type: E-Poster Presentation

Session title: Quality of life, palliative care, ethics and health economics

Background
Diffuse large B-cell lymphoma (DLBCL) represents approximately 30% of non-Hodgkin lymphomas, identified as one of its most common and aggressive forms. The recent inclusion of chimeric antigen receptor T-cell (CAR-T) treatments into the therapeutic scheme of DLBCL has been recognised as a change of paradigm in terms of survival and safety. Axicabtagene ciloleucel (axi-cel) and tisagenlecleucel (tisa-cel) are CAR-T therapies approved in Spain for the treatment of relapsed/refractory (R/R) DLBCL after ≥2 lines of systemic therapy in adult patients.

Aims
The aim of the study was to compare the cost-effectiveness of axi-cel vs tisa-cel for the treatment of adult patients with R/R DLBCL after ≥2 lines of systemic therapy, from the Spanish National Health System (NHS) perspective.

Methods
A lifetime partitioned survival mixture cure model, which comprises of pre-progression, post-progression, and death partitioned states, was used in the base case to estimate the accumulated costs and outcomes in terms of life years (LY) and quality-adjusted life years (QALY). Long-term survival with both therapies was estimated through a matching-adjusted indirect comparison (MAIC) that aligned the patient characteristics for the pivotal trials, fitting the patient-level data of ZUMA-1 trial for axi-cel to the aggregate-level data of JULIET trial for tisa-cel (Oluwole et al, Biol Blood Marrow Transplant, 2020). Utility values were extracted from EQ-5D data from ZUMA-1. The analysis was performed from the NHS perspective, thus only direct costs were included. Drug costs were estimated based on ex-factory prices with national mandatory deduction applied. Costs related to administration and monitoring of CAR-T therapy, apheresis, conditioning chemotherapy, disease management, stem cell transplant, hospitalisation and adverse events were derived from local cost databases and literature. A 3% annual discount rate was applied for costs and outcomes. Efficiency was expressed as incremental cost-effectiveness ratio (ICER; incremental cost vs effectiveness measured as LY), and incremental cost-utility ratio (ICUR; incremental cost vs effectiveness measured as QALY). One-way sensitivity analyses (OWSA) were carried out modifying discount rate, unitary costs, utilities, days of hospitalisation and simulating alternative scenarios such as naïve comparison without MAIC and partitioned survival model. A probabilistic sensitivity analysis (PSA) was also developed.

Results
In the base case, discounted results are as follows: axi-cel provides 9.45 LY and 7.47 QALY (7.24 in the pre-progression and 0.23 post-progression states), compared to 6.71 LY and 5.16 QALY (4.87 in pre-progression and 0.29 in post-progression) with tisa-cel. Thus, axi-cel generates 2.31 incremental QALY with an additional cost of €30,135 vs tisa-cel. The higher costs with axi-cel are mainly attributed to a longer progression-free survival, implying higher disease management costs in the pre-progression state. Hence, the ICER and ICUR are €10,999/LY and €13,049/QALY, respectively. OWSA and alternative scenario analyses show that the results are robust. The most influential parameters are the utility in the pre-progression state and the naïve comparison without MAIC. In the PSA, ICUR is ≤€22,000/QALY and ≤€60,000/QALY in 92.3% and 99.2% of the iterations, respectively.

Conclusion
Considering the frequently assumed willingness-to-pay thresholds in Spain, axi-cel is likely to be a cost-effective treatment vs tisa-cel for adult patients with R/R DLBCL in Spain. 

Keyword(s): CAR-T, Cost effectiveness, Diffuse large B cell lymphoma

Presentation during EHA2021: All e-poster presentations will be made available as of Friday, June 11, 2021 (09:00 CEST) and will be accessible for on-demand viewing until August 15, 2021 on the Virtual Congress platform.

Abstract: EP1167

Type: E-Poster Presentation

Session title: Quality of life, palliative care, ethics and health economics

Background
Diffuse large B-cell lymphoma (DLBCL) represents approximately 30% of non-Hodgkin lymphomas, identified as one of its most common and aggressive forms. The recent inclusion of chimeric antigen receptor T-cell (CAR-T) treatments into the therapeutic scheme of DLBCL has been recognised as a change of paradigm in terms of survival and safety. Axicabtagene ciloleucel (axi-cel) and tisagenlecleucel (tisa-cel) are CAR-T therapies approved in Spain for the treatment of relapsed/refractory (R/R) DLBCL after ≥2 lines of systemic therapy in adult patients.

Aims
The aim of the study was to compare the cost-effectiveness of axi-cel vs tisa-cel for the treatment of adult patients with R/R DLBCL after ≥2 lines of systemic therapy, from the Spanish National Health System (NHS) perspective.

Methods
A lifetime partitioned survival mixture cure model, which comprises of pre-progression, post-progression, and death partitioned states, was used in the base case to estimate the accumulated costs and outcomes in terms of life years (LY) and quality-adjusted life years (QALY). Long-term survival with both therapies was estimated through a matching-adjusted indirect comparison (MAIC) that aligned the patient characteristics for the pivotal trials, fitting the patient-level data of ZUMA-1 trial for axi-cel to the aggregate-level data of JULIET trial for tisa-cel (Oluwole et al, Biol Blood Marrow Transplant, 2020). Utility values were extracted from EQ-5D data from ZUMA-1. The analysis was performed from the NHS perspective, thus only direct costs were included. Drug costs were estimated based on ex-factory prices with national mandatory deduction applied. Costs related to administration and monitoring of CAR-T therapy, apheresis, conditioning chemotherapy, disease management, stem cell transplant, hospitalisation and adverse events were derived from local cost databases and literature. A 3% annual discount rate was applied for costs and outcomes. Efficiency was expressed as incremental cost-effectiveness ratio (ICER; incremental cost vs effectiveness measured as LY), and incremental cost-utility ratio (ICUR; incremental cost vs effectiveness measured as QALY). One-way sensitivity analyses (OWSA) were carried out modifying discount rate, unitary costs, utilities, days of hospitalisation and simulating alternative scenarios such as naïve comparison without MAIC and partitioned survival model. A probabilistic sensitivity analysis (PSA) was also developed.

Results
In the base case, discounted results are as follows: axi-cel provides 9.45 LY and 7.47 QALY (7.24 in the pre-progression and 0.23 post-progression states), compared to 6.71 LY and 5.16 QALY (4.87 in pre-progression and 0.29 in post-progression) with tisa-cel. Thus, axi-cel generates 2.31 incremental QALY with an additional cost of €30,135 vs tisa-cel. The higher costs with axi-cel are mainly attributed to a longer progression-free survival, implying higher disease management costs in the pre-progression state. Hence, the ICER and ICUR are €10,999/LY and €13,049/QALY, respectively. OWSA and alternative scenario analyses show that the results are robust. The most influential parameters are the utility in the pre-progression state and the naïve comparison without MAIC. In the PSA, ICUR is ≤€22,000/QALY and ≤€60,000/QALY in 92.3% and 99.2% of the iterations, respectively.

Conclusion
Considering the frequently assumed willingness-to-pay thresholds in Spain, axi-cel is likely to be a cost-effective treatment vs tisa-cel for adult patients with R/R DLBCL in Spain. 

Keyword(s): CAR-T, Cost effectiveness, Diffuse large B cell lymphoma

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