Cost-Effectiveness of Anti-CD19 Chimeric Antigen Receptor T-Cell Therapy in Pediatric Relapsed/Refractory B-cell acute Lymphoblastic Leukemia.

INTRODUCTION: In several studies, the chimeric antigen receptor T-cell therapy tisagenlecleucel demonstrated encouraging rates of remission and lasting survival benefits in pediatric patients with relapsed/refractory (r/r) acute lymphoblastic leukemia (ALL). We assessed the cost-effectiveness of tisagenlecleucel (list price: 320,000 EUR) among these patients when compared to clofarabine monotherapy (Clo-M), clofarabine combination therapy (Clo-C), and blinatumomab (Blin) from both a health care and a societal perspective. We also assessed future medical and future non-medical consumption costs. METHODS: A three-state partitioned survival model was used to simulate a cohort of paediatric patients (12 years of age) through different disease states until the end of life (lifetime horizon). Relevant outcomes were life years, quality-adjusted life years (QALYs), health care costs, societal costs and the incremental cost-effectiveness ratio (ICER). Uncertainty was explored through deterministic and probabilistic sensitivity analyses as well as through several scenario analyzes. RESULTS: Total discounted costs for tisagenlecleucel were 552,679 EUR from a societal perspective, which was much higher than the total discounted costs from a health care perspective (i.e. 409,563 EUR). Total discounted societal costs for the comparator regimens ranged between 160,803 EUR for Clo-M and 267,259 EUR for Blina. Highest QALYs were estimated for tisagenlecleucel (11.26), followed by Blina (2.25), Clo-C (1.70) and Clo-M (0.74). Discounted societal ICERs of tisagenlecleucel ranged between 31,682 EUR/QALY for Blina and 37,531 EUR/QALY for Clo-C and were considered cost-effective with a willingness-to-pay (WTP) threshold of 80,000 EUR/QALY. None of the scenarios exceeded this threshold and more than 98% of the iterations in the probabilistic sensitivity analysis were cost-effective. DISCUSSION: At the current price and WTP threshold tisagenlecleucel is cost-effective from both a health care and a societal perspective. Nevertheless, long-term effectiveness data is needed to validate the several assumptions that were necessary for this model.