OBJECTIVES : Patients with chronic myeloid leukemia (CML) having high Sokal score treated with imatinib demonstrated lower overall survival at 10 years compared to those having lower risk scores. This study aimed to estimate the budget impact of incorporating nilotinib as an option for the first-line treatment (1L) of patients with CML in chronic phase, with high-risk Sokal score in the Brazilian private healthcare system (PHS).

METHODS : A budget impact model was developed to estimate the total incremental cost associated with the use of nilotinib. To determine the eligible patient population, an epidemiological approach was adopted. Incidence of leukemia (5.16 per 100,000), CML rate (15%), diagnosis rate (90%) and proportion of patients with high-risk Sokal score (28%) were the filters applied in the total beneficiaries’ population of PHS (~47 million). The chosen comparator (imatinib) reflected the current reimbursement scenario. Progression to accelerated or blastic phase (AP/BP) data was obtained from ENESTnd study. After progression to AP/BP, patients did not receive any therapeutic drug and were only clinically followed until end-of-life. Only direct medical costs were considered. Drug acquisition were based on official published prices (CMED list/March 2019), and costs of progression and end-of-life were calculated from official sources from PHS. The incorporation scenario assumed a 100% market share to nilotinib.

RESULTS : Incorporation of nilotinib demonstrated an initial impact of USD 30,885 (1 USD = 3.97 BRL) in the first year, and USD 156,517 in five years accumulated.

CONCLUSIONS : The incremental cost per year showed the incorporation of nilotinib in 100% of eligible patients represented an additional cost less than 0.5% in comparison to the current scenario with only imatinib. Considering the severe condition of patients with CML with high-risk Sokal score, nilotinib demonstrated to be more effective than imatinib associated with low incremental impact to the PHS.