Real-world cost-effectiveness in chronic myeloid leukemia: the price of success during four decades of development from non-targeted treatment to imatinib

Abstract

Imatinib has revolutionized the treatment of chronic myeloid leukemia (CML). We evaluated clinical outcome and cost-effectiveness, using Swedish registry data based on patients with CML diagnosed 1973–2008. Outcome from three time periods (I: 1973–1979; II: 1991–1997; III: 2002–2008) associated with symptomatic treatment, interferon-α/stem cell transplant and implementation of imatinib, respectively, were compared and a lifetime cost-effectiveness model developed. Survival data from population registries, estimated resource use from clinical practice and quality of life estimates were employed. Substantial health gains were noted over time, paralleled by increased treatment costs. Median survival was 1.9, 4.0 and 13 years during the respective time periods. The incremental cost-effectiveness ratio (ICER) between periods III and II was €52 700 per quality-adjusted life year (QALY) gained. An estimated 80% price reduction of imatinib, related to patent expiry, would reduce this ICER to €22 700. Our data from four decades reveal dramatically improved survival in CML, paralleled by ICER levels generally accepted by health authorities.

Keywords::

• Chronic myeloid leukemia
• survival
• population-based
• costs
• imatinib
• lifetime simulation

Acknowledgements

This work was supported by grants from the Swedish Chronic Myeloid Leukemia Group; Adolf H. Lundin Charitable Foundation; Swedish Cancer Society; the regional agreement on medical training and clinical research between Stockholm County Council and Karolinska Institutet; and the Swedish Center for Business and Policy Studies.

The authors thank Professors John Goldman and Bengt Jönsson for critical review of the manuscript and very helpful comments.

Potential conflict of interest

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Supplementary material available online

Additional supporting information including: Supplementary Table I, resource use by treatment and year; Supplementary Table II, unit prices for resources, yearly costs for medicines in chronic phase and average costs in advanced phase; Supplementary Table III, expected health gains and costs by age group, and corresponding ICER per QALY gained by health-sector and societal perspectives (life years were undiscounted while lifetime costs and QALYs were discounted at 3% per year); Supplementary Figure 1, sensitivity analysis: ICER in health-sector and societal perspectives when varying time horizon, discount rate, utility weight and costs of stem cell transplant