OBJECTIVES: To evaluate the cost-effectiveness of ponatinib compared to bosutinib, allogeneic stem cell transplantation(allo-SCT) and hydroxycarbamide as treatment of adult patients with Chronic Phase -Chronic Myeloid Leukemia (CP-CML) who failed prior therapy with dasatinib or nilotinib for reasons of resistance/intolerance to, and for whom subsequent treatment with imatinib is not clinically appropriate, or who have the T315I mutation in the Greek healthcare setting.

METHODS: A markov model with three-month cycle was locally adapted from a third-party payer perspective to reflect the natural progression of patients with CP-CML through different health states over a life-time horizon (50-years). The clinical inputs as well as utility values were extracted from relevant clinical trials and published studies. Resource consumption data of CP-CML patients were obtained from local experts and the relevant unit costs from local resources(in €2017). Primary outcomes were patient quality-adjusted life years (QALYs), total costs and incremental cost-effectiveness ratios (ICERs) per QALY gained. Both costs and outcomes were discounted at 3.5% per annum. A probabilistic sensitivity analysis (PSA) was conducted to account for uncertainty in the model.

RESULTS: The analysis showed that ponatinib was associated with 4.21, 4.09 and 5.99 increment in QALYs compared with bosutinib, allo-SCT, and hydroxycarbamide, respectively, at additional cost of €160,679, €74,482 and €180,014, respectively. The corresponding ICERs were €38,161, €18,226 and €30,050 per QALY gained versus bosutinib, allo-SCT, and hydroxycarbamide, respectively. At the predefined willingness to pay threshold of €51,000 per QALY gained (3 times of Greek GDP, based on WHO), PSA estimated that treatment with ponatinib had a probability of 80% to be a cost-effective option compared to bosutinib and 94% compared to allo-SCT and hydroxycarbamide.

CONCLUSIONS: The results indicate that ponatinib provides substantial clinical benefit as compared with current treatment alternatives at a reasonable cost. Hence, ponatinib may represent a cost–effective treatment option for patients with CP-CML in Greece