Cost-Effectiveness of Ponatinib in The Treatment of Patients with Acute Lymphoblastic Leukemia with Philadelphia Chromosome Positive (PH+ ALL), Suitable for Allogeneic Stem Cell Transplant, in Greece

OBJECTIVES: To evaluate the cost-effectiveness of ponatinib over induction chemotherapy (IC), for the treatment of patients with Acute Lymphoblastic Leukemia with Philadelphia chromosome positive (Ph+ ALL) who exhibit resistance or intolerance (R/I) to dasatinib, or have the T315I mutation and are suitable for allogeneic stem cell transplant (allo-SCT), in Greece. METHODS: An international Markov model with 3-month cycles was locally adapted from a third-party payer perspective (EOPYY) to reflect the natural progression of patients with Ph+ ALL through different health states over a life-time horizon (50-years). Clinical data for ponatinib arm were retrieved from phase II trial (PACE), whereas for IC arm from LALA-94 trial. In the absence of valuations for Ph+ ALL health states, utilities for blast phase chronic-myeloid-leukemia were used. Resource use for the management of Ph+ ALL patients as well as the distribution of IC schemes used in Greece were based on experts' opinion. The relevant unit costs were obtained from local resources (prices €2017). Outcomes were evaluated in terms of life-years (LY) and quality-adjusted life-years (QALYs), and cost-effectiveness in terms of life-years gained (LYG) and QALYs gained. One-way (OWSA) and probabilistic sensitivity analysis (PSA) were conducted. RESULTS: Patients treated with ponatinib had 0.833 higher life expectancy (3.621 versus 2.788 LY) and gained 0.501 QALYs (2.234 versus 1.733) compared to IC, at an increased cost of €5,465 (€40,743 versus €35,277) per patient.The resulted incremental cost-effectiveness ratios were €6,563/LYG and €10,903/QALY gained. OWSA revealed that treatment costs were the drivers of the results, while the PSA showed that the probability of ponatinib to be cost-effective over IC exceeds that of 97% (willingness-to-pay:€51,000). CONCLUSIONS: Given the assumptions of this analysis, our results suggest ponatinib may offer improved survival and health related quality-of-life to patients with Ph+ ALL R/I to dasatinib, suitable for allo-SCT, at a moderate increase in cost compared to IC.