OBJECTIVES: Cushing's disease (CD) is a rare debilitating disease that incurs substantial healthcare costs when untreated or inadequately controlled. This study assesses the incremental budget impact of introducing pasireotide long acting release (LAR) for patients with CD from a Finnish societal perspective.

METHODS: The target population is adult patients with CD for whom surgery is not an option or surgery has failed. The model adopts a 5-year time horizon and includes direct costs (i.e. costs for drugs and medical procedures, drug administration, monitoring, and adverse events) and indirect costs. Model input parameters (prevalence, market share projections, resource use, and costs) were obtained from published literature or via interviews with a Finnish clinical expert. Alternative model inputs (varying prevalence, market share of pasireotide LAR, dosage of drugs, and the inclusion of costs associated with comorbidities) were used to evaluate the robustness of results.

RESULTS: A total of 89 patients are eligible for pasireotide LAR treatment in the year 2018 (first year) in Finland and are projected to increase to 91 patients by 2022 (fifth year). The introduction of pasireotide LAR is estimated to lead to an incremental budget impact of €45,247 in 2018, and up to €231,318 by 2022. This corresponds to a budget impact of 2% of total expenditure in year 1, increasing to only 9% in year 5. A higher treatment cost associated with the introduction of pasireotide LAR is partially offset by lower indirect and adverse event costs. Through sensitivity analyses, alternative model inputs showed the budget impact results to be robust.

CONCLUSIONS: With a low prevalence for this rare disease, the inclusion of pasireotide LAR in the Finnish national formulary for the medical treatment of CD will have a minimal impact on the total budget.