Decision Analysis on the Cost-Effectiveness of Sequential Treatment Strategies for Patients with Chronic Myeloid Leukemia in the United States

Abstract

A639 ment regimens for chronic myeloid leukemia. If further research were funded, studies should examine a combination of natural history, treatment, and quality of life parameters, especially the effectiveness of first-line TKI treatment.Objectives: The first goal was to adapt an existing Austrian decision-analytic model for chronic myeloid leukemia (CML) treatment to the US-American health care context. Secondly, we updated the model with new data and further treatment strategies to identify the most effective and most cost-effective strategy for the treatment of CML patients with different sequential tyrosine kinase inhibitors (TKIs). MethOds: We evaluated 18 different treatment strategies within the US-American setting in terms of survival, quality-adjusted survival and costs. For model parameters, data from literature, a US-American expert survey, the Utah Cancer Registry, and economic data from a US-American database were used. Evaluated treatment strategies included imatinib, dasatinib, nilotinib, bosutinib, ponatinib, stem-cell transplantation and chemotherapy. The Markov state-transition model was analyzed as a cohort simulation over a lifelong time horizon, a third-party payer perspective was adopted and a discount rate of 3% was used. Additionally, several deterministic and probabilistic sensitivity analyses were conducted. Results: Imatinib without second-line TKI resulted in an incremental cost-utility ratio (ICUR) of $148,700/QALY gained (incremental cost-effectiveness ratio (ICER) of $128,800/Lys) compared to baseline strategy 'chemotherapy'. Imatinib with second-line nilotinib yielded an ICUR of $217,100/QALY gained (ICER $242,200/ LY) compared to imatinib without second-line TKI. Imatinib followed by secondline bosutinib had an ICUR of $331,300/QALY gained (ICER $265,100/LY) compared to imatinib followed by second-line nilotinib. Imatinib with second-line dasatinib produced an ICUR of $343,200/QALY gained (ICER $279,600/LY) compared to imatinib with second-line bosutinib. All remaining strategies were excluded due to dominance. ICURs and ICERs obtained from the probabilistic sensitivity analysis deviated up to 6.5% (2.5%) compared to base-case ICURs (ICERs). cOnclusiOns: Based on our analysis and current treatment guidelines, we recommend imatinib followed by second-line nilotinib as the most cost-effective treatment strategy. Our model results may support clinicians and patients in CML treatment decision making.

Oncotyrol – Center for Personalized Cancer Medicine: Methods and Applications of Health Technology Assessment and Outcomes Research

“…[20] Additionally, the model was applied to the U.S. health care context. [21] Furthermore, the project team implemented a software tool for the electronic collection of QoL data (CHES -Computerbased Health Evaluation System) and conducted several QoL surveys at the University Hospital Innsbruck, which are currently being analyzed. The final step of the project is to adapt the model in order to analyze a more personalized approach to treatment decisions for CML patients.…”

Analysis of the cost-effectiveness of treatment strategies for CML with incorporation of treatment discontinuation

“…The cost-effectiveness of CML treatment has been of particular interest for physicians and researchers in many countries. [8][9][10][11] As a solution to this problem, Padula et al compared the costeffectiveness of an "imatinib-first" approach, in which physicians start treatment with imatinib, switching to dasatinib or nilotinib only in cases of resistance or intolerance, to that of a "physician's choice" approach, in which they initiate treatment with any 1 of 3 TKIs. 8 Their Markov model analysis clearly showed that the imatinib-first approach was associated with lower cost but similar quality-adjusted life years (QALYs) compared with the physician's choice approach.…”