<p>Cost-Effectiveness Analysis of Tisagenlecleucel in the Treatment of Relapsed or Refractory B-Cell Acute Lymphoblastic Leukaemia in Children and Young Adults in Spain</p>

Abstract

Purpose: Tisagenlecleucel, 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 Methods: A 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. Results: This 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. Conclusion: Tisagenlecleucel would represent a cost-effective intervention for the treatment of children and young adults with r/r ALL in Spain.

Tisagenlecleucel for relapsed/refractory acute lymphoblastic leukemia in the Irish healthcare setting: cost-effectiveness and value of information analysis

“…This was based on clinical opinion. This approach has been accepted by the National Institute for Health and Care Excellence (NICE UK) for reimbursement decision making (8) and has been employed in the literature (14). Mixture cure models are an alternative approach to model long-term survival; this approach was not used here due to the high degree of censoring towards the end of the trial follow-up periods.…”

Costs, effectiveness, and safety associated with Chimeric Antigen Receptor (CAR) T-cell therapy: Results from a comprehensive cancer center

“…The therapeutic value of axi-cel and tisa-cel, accessed through cost-effectiveness analyses of health outcomes gained, has been widely recognized by health systems of several countries [11,17,29]. In Spain, cost-effectiveness analysis showed a greater benefit of 10.10 life-years gained and 8.97 quality-adjusted life-years (QALYs) gained of tisa-cel compared to salvage chemotherapy, associated to an additional 258 378€ total cost of tisa-cel, in relapsed/refractory B-cell acute lymphoblastic leukemia [40]. In the US, in patients with B-cell lymphoma, cost-effectiveness estimates of axi-cel ranged from $58 000 to $289 000/QALYs gained and estimates of tisacel ranged from $168 000 to $223 000/QALYs gained, depending on model assumptions relating to cost and long-term survival [17,29,41].…”

Critical Reflections on Reimbursement and Access of Advanced Therapies

“…This makes it more difficult to appraise the value of advanced therapies given that the cost-effectiveness of these products tends to be sensitive to parameters such as the time horizon, assumptions regarding the duration of health gain generated by the advanced therapy, the number of years of patient follow-up in single-arm trials and survival extrapolation techniques ( Hao et al, 2017 ; Marchetti et al, 2018 ; Roth et al, 2018 ; Armoiry et al, 2019 ; National Institute for Health and Care Excellence, 2019c ; FiNoSe, 2019 ; Johnson et al, 2019 ; Malone et al, 2019 ; South et al, 2019 ; Walton et al, 2019 ; Whittington et al, 2019 ; Zimmermann et al, 2019 ; Autorité de Santé, 2020 ; Cook et al, 2020 ; Ribera Santasusana et al, 2020 ; Thielen et al, 2020 ; Wakase et al, 2021 ). In light of such clinical uncertainties, the French High Council for Health ( HAS, Haute Autorité de Santé ) for example has demanded recurrent annual economic evaluations of Kymriah® and Yescarta® incorporating the most recent data ( Jorgensen and Kefalas, 2021 ).…”