OBJECTIVES: To evaluate the cost-effectiveness of ponatinib compared to bosutinib, allogeneic stem cell transplantation (allo-SCT) and hydroxycarbamide in the treatment of adult patients with accelerated or blast phase - Chronic Myeloid Leukemia (AP/ΒP-CML) whose disease is resistant/ intolerant to dasatinib or nilotinib 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 was locally adapted from a third-party payer perspective over a 50-year time horizon. Efficacy, safety and utility data were extracted from relevant clinical trials and the literature. Resource consumption data were obtained from local experts and were combined with unit costs (in €2017) obtained from official sources. Primary outcomes were patient quality-adjusted life years (QALYs), total costs and incremental cost-effectiveness ratios (ICERs) per QALY gained. All the future outcomes were discounted at 3.5% per annum. A probabilistic sensitivity analysis (PSA) was conducted.

RESULTS: Total life time cost per patient in BP-CML was estimated at €29,895, €16,038, €42,893, and €8,609 for ponatinib, bosutinib, allo-SCT and hydroxycarbamide, respectively. In terms of health outcomes, ponatinib was associated with 0.96, 0.48 and 1.05 increment in QALYs compared with bosutinib, allo-SCT, and hydroxycarbamide respectively, resulting in ICERs of €14,481 and €20,288 per QALY gained versus bosutinib and hydroxycarbamide. Similar results were found in AP-CML with ICERs reaching at €679 and €13,878 per QALY gained, respectively. Moreover, ponatinib was a dominant alternative over allo-SCT in both AP/BP-CML. PSA revealed that the probability of ponatinib being a cost-effective option at the predetermined threshold of €51,000 per QALY gained was higher than 95% versus all available comparators in both AP/BP- CML.

CONCLUSIONS: The results indicate that, ponatinib seems to be a cost–effective option compared to other alternative therapies in the treatment of AP/BP-CML patients in Greece.