OBJECTIVES : Lymphangioleiomyomatosis (LAM) is a rare, progressive, systemic neoplastic disease currently without cure of unknown etiology that mainly affects young women in reproductive age. The objective of this study is to evaluate the budget impact of sirolimus, in the treatment of lymphangioleiomyomatosis in the perspective of Brazilian public health system.

METHODS : A budget-impact analysis was developed, comparing sirolimus with the current symptomatic care, considering a five-year time horizon and including only medicines direct costs. The current symptomatic care is an approach to treat the condition effects using alternatives and/or actions to monitor patients, without a specific medicine to treat the disease progression. Global epidemiological data from literature was used to estimate the eligible population. This data was applied to the Brazilian population projection within a five-year period.

RESULTS : Epidemiological data extrapolation presented a cohort of approximately 159 patients eligible for sirolimus treatment on year 1, up to 217 patients on year 5. Budget impact analysis resulted in an incremental budget investment of approximately BRL 896,2 thousand in the first year of incorporation, reaching BRL 1,22 million in the fifth year. Over the five-year period, the accumulated incremental budget was approximately BRL 5,3 million.

CONCLUSIONS : The inclusion of Sirolimus in the Brazilian public health system would grant access to patients with LAM to the only specific treatment for this condition presently. Considering only direct drug costs, the treatment of patients with LAM utilizing sirolimus was associated with a low impact investment considering the total budget of Brazilian public health system.