Lin JK, et al. - Researchers compared the cost efficacy of tisagenlecleucel (anti-CD19 chimeric antigen receptor T-cell therapy recently approved to treat relapsed or refractory pediatric acute lymphoblastic leukemia) to that of currently available treatments, given that, at a one-time infusion cost of $475,000, tisagenlecleucel is currently the most expensive cancer therapy. For determining cost effectiveness, long-term efficacy was a critical but uncertain factor. At its current price, the value of tisagenlecleucel is reasonable if it keeps a considerable number of patients in remission without needing transplantation; however, it won’t be if patients ultimately require transplantation. Regardless, price reductions would positively effect cost effectiveness.

Methods

• From a US health payer perspective, tisagenlecleucel was assessed in pediatric relapsed or refractory acute lymphoblastic leukemia over a lifetime horizon by using Markov modeling.
• Data from recent multicenter, single-arm clinical trials was incorporated in the model.
• Comparison of tisagenlecleucel (under a range of plausible long-term effectiveness) with blinatumomab, clofarabine combination therapy (clofarabine, etoposide, and cyclophosphamide), and clofarabine monotherapy was carried out.
• The uncertainty was explored using scenario and probabilistic sensitivity analyses.
• Life-years, discounted lifetime costs, discounted quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (3% discount rate) were assessed as main outcomes.

Results

• Increase in life expectancies by 12.1 years and cost $61,000/QALY gained were expected with tisagenlecleucel, with an assumption of a 40% 5-year relapse-free survival rate.
• Life-expectancies were found to be more modest (3.8 years) and expensive ($151,000/QALY gained) at a 20% 5-year relapse-free survival rate.
• Tisagenlecleucel resulted in increased life expectancies by 5.7 years and cost $184,000/QALY gained at a 0% 5-year relapse-free survival rate and with use as a bridge to transplant.
• In all scenarios, a $100,000/QALY or $150,000/QALY willingness-to-pay threshold would be met by reduction of the price of tisagenlecleucel to $200,000 or $350,000.