AML is a rapidly progressing and life-threatening blood cancer that disproportionately affects older adults. The treatment paradigm in AML has seen little change in the past 4 decades, with intensive chemotherapy with a 7-day continuous infusion of cytarabine plus an anthracycline such as daunorubicin on Days 1-3 (ie, “7+3” regimen) remaining the standard-of-care induction therapy for AML. CPX-351 is a dual-drug liposomal encapsulation of cytarabine and daunorubicin that delivers a synergistic drug ratio into leukemia cells. In a phase 3 study (NCT01696084), CPX-351 demonstrated superior overall survival (9.56 vs 5.95 months; HR = 0.69; P= 0.005) and significantly improved complete remission rate (38% vs 26%; P= 0.040) compared with 7+3 in the treatment of elderly patients with newly diagnosed tAML or AML-MRC (Lancet JE, et al. ASCO 2016. Abstr 7000). Additionally, CPX-351 is administered as a 90-minute infusion on Days 1, 3, and 5 (induction cycle 1 only), and offers the potential for outpatient administration. The aim of this study was to estimate the budget impact associated with the introduction of CPX-351 for the treatment of patients with tAML or AML-MRC from a US payer perspective.

A decision analytic model was developed to estimate the budget impact of using CPX-351 compared with 7+3 for induction and consolidation treatment of AML patients with tAML and AML-MRC. The decision algorithm was developed based on clinical practice guidelines from the National Comprehensive Cancer Network and validated by an oncologist. Epidemiology estimates were from SEER 2017. Clinical outcomes (response rate and incidence of adverse events [AE]) and site of care were from the CPX-351 phase 3 pivotal trial. In that study, CPX-351 was administered in the outpatient setting for 51% of patients in consolidation cycle 1 and 61% in cycle 2, compared with 6% and 0% of 7+3 patients in consolidation cycles 1 and 2 (Kolitz JE, et al. ASCO 2017. Abstr 7036). Administration costs were from CMS for Medicare and InGauge Healthcare Solutions for commercial costs. Hospital costs were from the CMS Limited Data Set. H-CUP National Inpatient Sample was used to estimate the AE costs by payer type. Drug costs were wholesale acquisition costs. The model did not include costs for hematopoietic cell transplant.

For a hypothetical 1 million-member plan, it is estimated that there are approximately 12 patients with tAML and AML-MRC each year. Increasing the use of CPX-351 from year 1 to 3 (assumption of year 1 = 20%, year 2 = 50%, and year 3 = 80%) resulted in cost increases compared with 7+3 (year 1 = $7,471 to year 3 = $29,884, or <$0.01 per member per month increase in each year). Over the same time period, the estimated number of responders increased from 3.06 with 7+3 to 4.19 with CPX-351. The increased level of responses with CPX-351 reduced the cost/responder from $358,695 with 7+3 to $269,106 by year 3 following introduction of CPX-351. Of note, outcomes are derived from a phase 3 randomized, controlled trial of CPX-351 versus 7+3, the model did not account for potential regimen changes, and costs may not be generalizable across all settings

This model predicted that the use of CPX-351 may be associated with improved patient outcomes and limited budget impact from the payer perspective.