The current price for tisagenlecleucel is approximately $475,000 USD. A study published in JAMA Oncology found that, at this price, tisagenlecleucel for relapsed or refractory pediatric acute lymphoblastic leukemia (ALL) had an incremental cost per quality-adjusted life-year (QALY) gain greater than estimated willingness-to-pay thresholds; however, cost-effectiveness was highly dependent on the assumed cure rate and reached acceptable ranges with product discounts.
Researchers explained that for tisgenlecleucel-eligible patients in Canada to receive the treatment and be formally reimbursed for the cost, an assessment of whether the value of tisgenlecleucel justifies the cost compared with current standard of care is necessary.
To conduct the cost utility analysis of tisagenlecleucel compared with the current standard of care, the team leveraged data from a Canadian population-based registry of pediatric patients with ALL. Data collection and analysis took place in September 2017 and December 2017, respectively. The investigators pooled data from 3 single-arm tisagenlecleucel clinical trials to create a treatment arm and from the provincial pediatric cancer registry to create a population-based control arm.
Simulation modeling was used to estimate relative healthcare costs, survival gains, and QALYs between tisagenlecleucel and the current standard of care. Because the long-term effectiveness of tisagenlecleucel is uncertain, the model included long-term cure rates ranging from 10% to 40%.
The treatment arm included 192 patients while the control arm included 118 patients; the mean age for both arms was similar at 11 years and 10 years, respectively.
Treatment with tisagenlecleucel was associated with an increased 24-month survival rate compared with the standard of care (0.44 vs 0.23). Post-treatment life expectancy was also improved with tisagenlecleucel (7.19-14.90 years, depending on the assumed cure rate) relative to the control arm (5.05 years), translating to an additional 2.14 to 9.85 life years or 1.68 to 6.61 QALYs compared with the standard of care.
At an assumed cure rate of 40%, the mean cost of tisagenlecleucel was $442,098 USD. The mean cost of the standard of care was $86,597 USD. When taking into account the total discounted cost over a patient’s lifetime, the incremental cost of tisagenlecleucel was $53,933 USD.
“Tisagenlecleucel cost-effectiveness would fall below $50,000 [USD per] QALY with a long-term cure rate of over [40%] or a price discount of 49% at its currently known effectiveness,” wrote the researchers. “These results provide a real-world benchmark for what product discount would make tisagenlecleucel cost-effective.”
1. Furzer J, Gupta S, Nathan PC, et al. Cost-effectiveness of tisagenlecleucel vs standard care in high-risk relapsed pediatric acute lymphoblastic leukemia in Canada [published online January 23, 2020]. JAMA Oncol. doi:10.1001/jamaoncol.2019.5909