The introduction of BCR-ABL1 tyrosine kinase inhibitors (TKIs) to the treatment of chronic myeloid leukemia (CML) dramatically changed long-term outcomes.Now, there are several TKI options, and optimal treatment selection can be challenging given differences in patient characteristics and cost of therapy. 

“While successful therapeutic outcomes and long-term survival are clearly the main goal of TKI treatment, patients are also faced with lifelong management of the financial aspects of being diagnosed with CML and affording treatment,” Giuseppe Saglio, MD, of the San Luigi Hospital in Turin, Italy, and Elias Jabbour, MD, of The University of Texas MD Anderson Cancer Center, Houston, wrote.1

Imatinib, a first-generation TKI that was approved by the US Food and Drug Administration (FDA) in 2002, dramatically improved survival compared with the previous standard of care, which was the combination of interferon-α and cytarabine. The 10-year overall survival (OS) with imatinib is estimated at 83% compared with a 5-year OS of 68% with interferon-α plus cytarabine.1

Since then, the second-generation TKIs have emerged — dasatinib was first approved by the FDA in 2006 and nilotinib in 2007. Both agents can induce faster and deeper treatment responses compared with imatinib, but the safety profiles differ, so must also be considered, particularly when selecting first-line therapy.Cost-effectiveness should also be measured, as expenses increase for both the patient and the payer as CML becomes a chronic disease.  

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Cost-effectiveness analyses have been conducted that compare the TKIs to each other, but these results will change as generic formulations become available. In 2016, Gleevec (imatinib) lost its exclusivity, resulting in the introduction of the generic formulation of imatinib.1Dasatinib and nilotinib will lose their exclusivity in the coming years, which will further change the cost-effectiveness of these agents. 

Cost-effectiveness of TKIs for CML treatment is, therefore, a complex topic that requires consideration of multiple factors — not just apparent costs to payers.