OBJECTIVES: A phase 3 trial demonstrated superior outcomes for CPX-351 versus conventional daunorubicin/cytarabine (3+7) in patients aged 60–75 years with newly diagnosed secondary AML (high-risk AML). Understanding the economics of CPX-351 in the substantial fraction of this population aged <60 years is important. A systematic literature review (SLR) of differences in clinical outcomes in younger high-risk patients was employed, and findings were used to assess the cost-effectiveness of CPX-351 versus 3+7 among patients aged <60 years in the UK.

METHODS: An SLR was conducted to identify potential effects of age on clinical outcomes (response, transplant rate, overall survival, progression-free survival) of patients with 3+7 treatment. The findings from this review were incorporated into an existing economic model of CPX-351 versus 3+7 treatment for high-risk AML, assuming the effects of age applied equally regardless of treatment, to project lifetime health and cost outcomes. A survival-partition approach was used, with distinct survival curves for cohorts defined by treatment pathways (response and transplantation). Model outputs included survival, quality-adjusted life-years (QALYs), and costs.

RESULTS: Limited evidence was identified in the literature for an effect of age. Four studies reported a difference in response rate by age, with the largest and most relevant reporting an OR of 1.9. Applying an age-dependent response rate and age-adjusted background mortality in the model resulted in a higher predicted transplant rate in the younger versus older populations (56% vs 50% among responders receiving CPX-351) and longer mean survival over the course of the model (4.1 vs 2.6 years with CPX-351). Consequently, a more favorable incremental cost-effectiveness ratio (ICER) was computed for CPX-351 versus 3+7 in patients with high-risk AML aged <60 years (£30,450/QALY) versus >60 years (£51,421/QALY).

CONCLUSIONS: An economic model of CPX-351 versus 3+7 for high-risk AML estimates a lower ICER among younger versus older patients.