OBJECTIVES

Estimate the budget impact of nilotinib therapy in chronic myeloid leukemia (CML) considering treatment-free remission (TFR) under the Brazilian private healthcare system (BPHS) perspective.

METHODS

A 10-year budget impact model was developed to evaluate the impact of TFR in CML patients treated with nilotinib in first (1L) and second line (2L). Patient population was obtained through an epidemiological approach, considering total number of beneficiaries in BPHS, Brazilian leukemia and CML incidence rates (n=329). All eligible patients were analyzed in two different treatment pathways: (1) 1L nilotinib and 2L dasatinib, (2) 1L imatinib and 2L nilotinib. 2L was adopted in cases of disease progression or failed molecular response. An additional comparison of 1L imatinib scenario was performed, considering only dasatinib or nilotinib in 2L. TFR eligibility was only considered for nilotinib therapy and was based on patients in use of tyrosine kinase inhibitors for 3 years, who maintained deep molecular response (MR4.5) for the last 12 months. Clinical data such as time to and duration of MR4.5, treatment-free survival and event-free survival were obtained from ENESTnd, ENESTfreedom and ENESTop studies. Cost of treatment considered drug acquisition and monitoring, which were calculated based on recommendations by national label and factory prices from Brazilian official list. qPCR test cost was obtained from official table of Brazilian private medical procedures.

RESULTS

By the end of 10-year analysis, 115 patients remained in 1L imatinib and 165 patients in 1L nilotinib, and 138 patients were in TFR. Total cost resulted in BRL423.9 million and BRL434.3 million in 1L nilotinib and imatinib scenarios, respectively. The additional 2L analysis resulted in a total cost of nilotinib and dasatinib of BRL229.8 million and BRL289.8 million.

CONCLUSIONS

TFR of nilotinib in 1L and 2L scenarios offers potential savings to BPHS, demonstrating its importance in both clinical and economic perspectives of long-term CML treatment.