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Applied Health Economics and Health Policy
Published in: Applied Health Economics and Health Policy 4/2019

01-08-2019 | Chronic Myeloid Leukemia | Original Research Article

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

Authors: Carsten Hirt, Sergio Iannazzo, Silvia Chiroli, Lisa J. McGarry, Philipp le Coutre, Leif Stenke, Torsten Dahlén, Jeffrey H. Lipton

Published in: Applied Health Economics and Health Policy | Issue 4/2019

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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.
Appendix
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Metadata
Title
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
Authors
Carsten Hirt
Sergio Iannazzo
Silvia Chiroli
Lisa J. McGarry
Philipp le Coutre
Leif Stenke
Torsten Dahlén
Jeffrey H. Lipton
Publication date
01-08-2019
Publisher
Springer International Publishing
Published in
Applied Health Economics and Health Policy / Issue 4/2019
Print ISSN: 1175-5652
Electronic ISSN: 1179-1896
DOI
https://doi.org/10.1007/s40258-019-00489-0

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