OBJECTIVES: To analyze the financial burden associated with the management of chronic myeloid leukemia (CML) patients who failed treatment with two or more tyrosine kinase inhibitors (TKI) through the Brazilian private market’s perspective.

METHODS: An economic model was developed to estimate total cost of treatment in later lines of CML. Target population was obtained through an epidemiological approach considering adult patients diagnosed with CML in chronic phase, who were previously treated with at least 2 TKIs. Percentage of failure (75%) of patients switching 2^nd generation TKI (2^nd TKI) as third line treatment approach was obtained from Akard et al. 2013. Resource usage in 3^rd line and time of treatment was based on Atallah et al., 2022. Patients who failed 3^rd line treatment and were ineligible to hematopoietic stem cell transplantation (HSCT) were treated with ponatinib. Drugs were selected based on regulatory-approved labels in Brazil. Drug costs reflect list prices from Câmara de Regulação do Mercado de Medicamentos (CMED), considering ex-factory prices. Cost of HSCT has considered data from Funke et al., 2022 study, and estimation of monitoring cost considered the Brazilian table: Classificação Brasileira Hierarquizada de Procedimentos Médicos (CBHPM).

RESULTS: 375 patients were estimated to be analyzed. Total treatment expenses in later treatment lines resulted in a total of BRL 265.3 million. Drug acquisition was the most significant economic variable, representing from 75% to 88% of total cost in all treatment lines. Patients who underwent HSCT represented the highest per capita disbursement in the analysis.

CONCLUSIONS: CML patients in later lines demonstrate relevant disease management-related expenditures. Although HSCT is associated to an elevated budgetary impact, only a low proportion of patients are eligible to undergo the procedure. Significant costs regarding failure of second line treatment highlight the medical unmet need for new efficient technology in CML landscape, especially in later lines.