The Cost-Effectiveness of Frontline Tyrosine Kinase Inhibitors for Patients With Chronic Myeloid Leukemia: In Pursuit of Treatment-Free Remission and Dose Reduction.

Objectives: The management of chronic myeloid leukemia (CML) now includes dose reduction (DR) and treatment-free remission (TFR). Evaluating the cost-effectiveness of lifelong-prescribed expensive tyrosine kinase inhibitors (TKIs) for CML is crucial. Prior cost-effectiveness evaluations state that imatinib is the favorable frontline TKI. Some of these evaluations address TFR, but not DR, nor aging and second-generation (2G)-TKIs upcoming patent expirations. This study evaluates the cost-effectiveness of frontline TKIs for CML patients including these factors.

Methods: This Markov model evaluates the cost-effectiveness of frontline TKIs for newly diagnosed patients with CML using 17 health states. Transition probabilities, costs, and utilities were derived from literature data. Incremental cost-effectiveness ratios were calculated. Sensitivity analysis and model validation were conducted.

Results: Nilotinib is most effective (20.13 quality-adjusted life-years [QALYs]) and imatinib is least effective (17.25 QALYs) for the model including TFR and DR. Imatinib was favored over dasatinib (89.80%), nilotinib (62.70%), and bosutinib (78.40%), at a willingness-to-pay threshold of €80 000 per QALY. Without TFR and DR, fewer QALYs were generated. For patients at the age of 70 years, imatinib has a high probability of being more cost-effective than dasatinib, nilotinib, and bosutinib. With 50% 2GTKI cost reductions, nilotinib is considered more cost-effective compared with imatinib (98.40%), dasatinib (94.80%), and bosutinib (68.90%).

Conclusions: The findings indicate that 2GTKIs are more effective in generating QALYs, including for older (age >70 years) patients. Given the current TKI prices, imatinib remains cost-effective. Including DR and TFR in CML management generates more QALYs. Cost reductions from expected 2GTKIs patent expirations will greatly increase their cost-effectiveness. Results may inform 2GTKIs cost discussions after patent expiration, potentially broadening global availability. The findings also emphasize the importance of aiming for TFR and DR in CML management.