OBJECTIVES : To evaluate the cost-effectiveness of ponatinib compared to other second-line tyrosine kinase inhibitors (TKIs,), including dasatinib, nilotinib, and bosutinib, in the treatment of adult patients with chronic myeloid leukemia (CML) who failed or were intolerant to first-line TKIs in the United States (US).

METHODS : A Markov state transition model was conducted to compare cost-effectiveness of second-line TKI for treatment practice of CML patients who failed or were intolerant to first-line TKI using a US commercial payer perspective over a life-long time horizon. Transition probabilities, safety and utility data were obtained from clinical trials and literature. Medical costs on incident CML treatment was estimated using the Medical Expenditure Panel Survey data between 2013 and 2016. Resource utilization included study drugs, monitoring, follow-up, adverse events and allogeneic stem cell transplantation (allo-SCT). Primary outcomes were patient quality-adjusted life years (QALYs), total costs and incremental cost-effectiveness ratios (ICERs) per QALY gained with discount rate 3% per annum. Univariate sensitivity analyses and a Bayesian multivariable probabilistic sensitivity analysis were conducted using Monte Carlo simulations.

RESULTS : Total life time medical costs including follow-up care after allo-SCT per CML patient was estimated at $2,226,616, $2,272,596, $2,362,797, and $2,457,005 for nilotinib, dasatinib, bosutinib and ponatinib, respectively. In the economic evaluation, dasatinib resulted in an ICER of $79,114.19/QALY compared to nilotinib. Ponatinib yielded an ICER of $176,301.6/QALY compared to dasatinib. Ponatinib yielded an ICER of $141,588/QALY compared to nilotinib. All remaining strategies were excluded due to dominance of the superior strategies. Sensitivity analysis showed the model was robust to plausible changes in input parameters and had good face validity.

CONCLUSIONS : Based on current treatment guidelines, this analysis indicates that dasatinib and ponatinib can be considered cost-effective options and provide substantial clinical benefit compared to other TKIs for treating patients with CML, depending on willingness-to-pay, from the perspective of the US commercial payer.