Cost-Effectiveness Models for Tisagenlecleucel in Relapsed or Refractory Pediatric B-Cell ALL


Key Points

  • Cost for QALY gained differed substantially according to assumptions of 5-year relapse-free rate with tisagenlecleucel and need for HSCT.
  • Cost reduction would improve cost-effectiveness even in the context of modest long-term effectiveness.

As reported by Lin et al in the Journal of Clinical Oncology, cost-effectiveness modeling of treatment with the anti-CD19 chimeric antigen receptor T-cell therapy tisagenlecleucel (Kymriah) in relapsed or refractory pediatric B-cell acute lymphoblastic leukemia (ALL) showed that price reduction would result in meeting acceptable thresholds under numerous assumptions of clinical outcomes.

The one-time infusion cost of tisagenlecleucel is estimated at $475,000.

Study Details

Markov modeling was used to evaluate tisagenlecleucel cost-effectiveness in pediatric relapsed or refractory ALL from a U.S. health payer perspective over a lifetime horizon. Modeling was based on findings in recent multicenter single-arm clinical trials. Using a range of plausible estimates of long-term effectiveness, tisagenlecleucel was compared with other currently available treatments, consisting of blinatumomab, clofarabine in combination with etoposide and cyclophosphamide, and clofarabine monotherapy.

Cost-Effectiveness Estimates

On the assumption of a 40% 5-year relapse-free survival rate, tisagenlecleucel was associated with an increase in life expectancy of 12.1 years, with a cost of $61,000 per quality-adjusted life-years (QALY) gained. On the assumption of a 20% 5-year relapse-free survival rate, treatment was associated with an increase in life expectancy of 3.8 years, with a cost of $151,000/QALY gained. On the assumption of a 0% 5-year relapse-free survival rate with treatment serving as a bridge to hematopoietic stem cell transplantation (HSCT), tisagenlecleucel was associated with an increase in life expectancy of 5.7 years, with a cost of $184,000/QALY gained. It was estimated that a reduction of price of tisagenlecleucel to $200,000 or $350,000 would allow it to meet $100,000/QALY or $150,000/QALY willingness-to-pay thresholds in all scenarios considered.

The investigators concluded, “The long-term effectiveness of tisagenlecleucel is a critical but uncertain determinant of its cost-effectiveness. At its current price, tisagenlecleucel represents reasonable value if it can keep a substantial fraction of patients in remission without transplantation; however, if all patients ultimately require a transplantation to remain in remission, it will not be cost-effective at generally accepted thresholds. Price reductions would favorably influence cost-effectiveness, even if long-term clinical outcomes are modest.”

The study was supported in part by a Veterans Affairs Office of Academic Affiliations fellowship and a National Center for Advancing Translational Science Clinical and Translational Science Award.

John K. Lin, MD, of the Center for Primary Care and Outcomes Research, Stanford University, is the corresponding author for the Journal of Clinical Oncology article.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.