Cost Effectiveness of the Third-Generation Tyrosine Kinase Inhibitor (TKI) Ponatinib, vs. Second-Generation TKIs or Stem Cell Transplant, as Third-Line Treatment for Chronic-Phase Chronic Myeloid Leukemia

Carsten Hirt, Sergio Iannazzo (), Silvia Chiroli, Lisa J. McGarry, Philipp Coutre, Leif Stenke, Torsten Dahlén and Jeffrey H. Lipton
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Carsten Hirt: Greifswald University Medical Center
Sergio Iannazzo: SIHS Health Economics Consulting, SIHS SRL
Silvia Chiroli: Incyte Biosciences International Sàrl
Lisa J. McGarry: ARIAD Pharmaceuticals, Inc., a Wholly Owned Subsidiary of Takeda Pharmaceutical Company Limited
Philipp Coutre: Charité, University of Medicine Berlin
Leif Stenke: Karolinska University Hospital and Karolinska Institutet
Torsten Dahlén: Karolinska University Hospital and Karolinska Institutet
Jeffrey H. Lipton: Princess Margaret Cancer Centre

Applied Health Economics and Health Policy, 2019, vol. 17, issue 4, No 11, 555-567

Abstract: Abstract Background and Objectives Third-line treatment options for patients with chronic-phase chronic myeloid leukemia include tyrosine kinase inhibitors and allogeneic hematopoietic stem cell transplantation (alloHSCT). The objective of this study was to develop a Markov model with a lifetime time horizon to assess the cost effectiveness of ponatinib for third-line chronic-phase chronic myeloid leukemia vs. second-generation tyrosine kinase inhibitors (dasatinib, nilotinib, bosutinib) or alloHSCT from the public healthcare system perspective in Germany, Sweden, and Canada. Methods Clinical outcomes were derived from the literature, and from patient-level data (phase II PACE trial) for ponatinib. Resource use included drugs, alloHSCT, monitoring and follow-up, adverse events, and end-of-life care; costs were based on national tariffs. Quality-adjusted life-years (QALYs) were calculated using chronic myeloid leukemia health-state utilities from an international time–trade-off study. Costs and benefits were discounted at 3% per annum for Germany and Sweden, and 5% for Canada. Results Ponatinib yielded more discounted QALYs than any second-generation tyrosine kinase inhibitor/alloHSCT in all three countries, mainly owing to better response rates and longer durations of response. Incremental cost-effectiveness ratios for ponatinib vs. second-generation tyrosine kinase inhibitors were US$21,543–37,755/QALY in Germany, $24,018–38,227/QALY in Sweden, and $43,001–58,515/QALY in Canada. Ponatinib was dominant over alloHSCT in Germany, while incremental cost-effectiveness ratios for ponatinib vs. alloHSCT in Sweden and Canada were $715/QALY and $31,534/QALY, respectively. Conclusions Ponatinib may improve outcomes (mainly because of higher response rates and longer response durations) at an acceptable cost level compared with other third-line treatment options for chronic-phase chronic myeloid leukemia in Germany, Sweden, and Canada; however, the lack of an indirect comparison is a limitation of our study.

Date: 2019
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DOI: 10.1007/s40258-019-00489-0

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