Cost-effectiveness of ponatinib in chronic myeloid leukemia in Italy: Valutazione di costo-efficacia di ponatinib nella terapia della leucemia mieloide cronica in Italia

An area-under-the-curve Markov model was designed to evaluate the cost-effectiveness of ponatinib as a third line treatment of Chronic Myeloid Leukemia-Chronic Phase (CML-CP) with reference to Italy. As for current guidelines, comparators were dasatinib, nilotinib, bosutinib, allogeneic stem cell transplantation (SCT), hydroxyurea. The economic perspective was the Italian National Health Service's (NHS), where costs for treatment drugs, monitoring and follow-up, adverse events, SCT procedure were considered on a lifetime span. Costs (mainly based on current tariffs in Italy) and benefits (QALYs) were discounted at a 3.5% annual rate. Ponatinib resulted dominant versus SCT. The lowest ICER was €13,090 (ponatinib vs hydroxyurea); the highest was €22,529 (ponatinib vs dasatinib). Sensitivity analysis – both deterministic (one way) and probabilistic – was focused on the comparison between ponatinib and dasatinib. The deterministic analysis showed that the most critical parameter in the model was ponatinib price, a 20% increase of which would imply a 63% increase in the base case ICER (from €22,529 to €36,871). In the cost-effectiveness plane, ponatinib consistently resulted more expensive but also more effective than dasatinib. Assuming as a threshold value the range indicated by the Health Economics Italian Association (€25,000-€40,000), the acceptability curve showed that ponatinib would be cost-effective with about a 50% probability when compared to the lower value (90% probability when compared with the higher). This analysis suggests that treating CML-CP with ponatinib provides a substantial clinical benefit as compared with current alternatives, with an incremental survival of 3-4 QALYs and at reasonable costs from the perspective of the Italian NHS. Sensitivity analysis confirmed the robustness of these results.