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Updated: Jan 23, 2026
Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
Published on: September 18, 2013
Cost Effectiveness of the Third-Generation Tyrosine Kinase Inhibitor (TKI) Ponatinib, vs. Second-Generation TKIs or
Carsten Hirt1, Sergio Iannazzo2, Silvia Chiroli3,4
1Department of Internal Medicine C, Hematology and Oncology, Greifswald University Medical Center, Greifswald, Germany.
Insights
Ponatinib offers improved outcomes for chronic-phase chronic myeloid leukemia patients in third-line treatment. This tyrosine kinase inhibitor demonstrates cost-effectiveness compared to other options across Germany, Sweden, and Canada.
Area of Science:
- Hematology
- Pharmacoeconomics
- Oncology
Background:
- Third-line treatment for chronic-phase chronic myeloid leukemia (CML) involves tyrosine kinase inhibitors (TKIs) or allogeneic hematopoietic stem cell transplantation (alloHSCT).
- Assessing the cost-effectiveness of novel treatments is crucial for healthcare systems.
Purpose of the Study:
- To evaluate the cost-effectiveness of ponatinib as a third-line treatment for chronic-phase CML.
- Comparison against second-generation TKIs (dasatinib, nilotinib, bosutinib) and alloHSCT from a public healthcare perspective in Germany, Sweden, and Canada.
Main Methods:
- A lifetime horizon Markov model was developed.
- Clinical outcomes were sourced from literature and the PACE trial; resource use and costs were based on national tariffs.
- Quality-adjusted life-years (QALYs) were calculated, with costs and benefits discounted at 3% or 5% depending on the country.
Main Results:
- Ponatinib demonstrated superior QALYs compared to second-generation TKIs and alloHSCT in all studied countries.
- Incremental cost-effectiveness ratios (ICERs) for ponatinib vs. second-generation TKIs ranged from US$21,543/QALY to $58,515/QALY.
- Ponatinib was dominant over alloHSCT in Germany and showed favorable ICERs in Sweden and Canada.
Conclusions:
- Ponatinib presents a potentially cost-effective option for third-line chronic-phase CML treatment in Germany, Sweden, and Canada.
- Improved outcomes are attributed to higher response rates and longer response durations.
- The lack of indirect comparisons is noted as a study limitation.
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.
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