Paradigm Shifts in the Management of Chronic Myeloid Leukemia and Health Policy Challenges in the Era of High Cost Anticancer Drugs

Chronic myeloid leukemia (CML) therapy has evolved from survival-oriented management into the pursuit of a “functional cure,” characterized by treatment-free remission (TFR). Achieving a deep molecular response (DMR) is a critical prerequisite for TFR and recent clinical advances have introduced novel therapeutic options that enable this goal to be reached more efficiently. asciminib, the firstin- class specifically targeting the ABL myristoyl pocket inhibitor, has demonstrated superior efficacy compared to conventional ATPcompetitive tyrosine kinase inhibitors (TKIs). In a pivotal phase 3 ASC4FIRST trial, asciminib showed significantly higher rates of major molecular response (MMR) and DMR (MR4) compared to investigator-selected TKIs. Furthermore, the early molecular response (EMR) rate was 89.6% at 12 weeks. Such rapid and sustained molecular responses are known to be predictors for successful TFR. A successful TFR improves patient quality of life by eliminating drug-related toxicities and leads to a structural reduction in long-term socioeconomic healthcare costs. This study evaluated the clinical impact of these new therapeutic paradigms. It also highlights the need to standardize high-sensitivity molecular monitoring and reform the reimbursement guidelines to support the safe implementation of TFR in Korea.