Economic Evaluation for the US of the Novel Tyrosine Kinase Inhibitor Ponatinib Versus Reference and Generic Imatinib in Front-line Management of Philadelphia Chromosome–Positive Acute Lymphoblastic Leukemia

Abstract

Objective: Ponatinib, a third-generation tyrosine-kinase inhibitor (TKI) demonstrated greater efficacy compared to imatinib, a first-generation TKI, in the PhALLCON trial. This study aimed to conduct an economic evaluation of ponatinib compared to reference imatinib (Gleevec®), the average cost of several imatinib generics and the weighted cost of imatinib respectively, in the treatment of patients with Philadelphia chromosome-positive Acute Lymphoblastic Leukemia (Ph+ ALL) from the US payer perspective. Methods: Cost-effectiveness and cost-utility analyses were performed using a partitioned survival model with three mutually exclusive health states—progression-free, progressed disease, and death. A time horizon of 60 months and a 3% discount rate were applied. The incremental costs per 1% gain in probability of progression-free survival (PFS) and overall survival (OS) at 60 months were estimated. Results: The exponential distribution was deemed the best fit for both PFS and OS in both treatment arms. LYs and QALYs were higher for ponatinib (3.74, 2.15) than imatinib (3.52, 1.68). Using Gleevec® as a reference, ponatinib had cost savings of $56,024. However, ponatinib had an incremental cost of $147,147, an ICER and ICUR of an additional $668,150 and an additional $314,646 per LY and QALY gained respectively compared to average generic imatinib. For weighted imatinib, ponatinib had an incremental cost of $128,861, yielding ICER and ICUR of $585,122 and $275,546 per LY and QALY gained respectively. The incremental cost of ponatinib per patient for a 1% increase in the probability of remaining in the PFS state was -$6,883 compared to Gleevec®, $18,077 compared to the average generic imatinib and $15,831 compared to the weighted imatinib. Conclusion: Ponatinib demonstrated cost savings relative to Gleevec®, though its incremental costs were substantially higher when compared to both average and weighted imatinib. Given that 91% of prescriptions are generic medications, payers and policymakers must weigh the clinical benefits of ponatinib in improving survival against its financial implications.