Presentation (CTI)

OBJECTIVES: To assess the economic implications of metyrapone versus osilodrostat in adults with endogenous Cushing’s syndrome (CS), a rare endocrine disorder with limited pharmacological treatment options, from the perspective of the Polish public payer, the National Health Fund (NFZ).
METHODS: Based on clinical evidence, including PROMPT and MOSKETEER studies, indicating comparable efficacy and safety of metyrapone and osilodrostat, a cost-minimization analysis was conducted in line with Polish HTA requirements. A global Markov cohort model was adapted to the Polish healthcare setting, using a 54-year lifetime horizon, 12-week cycles, and 5% annual cost discounting. Analyses were performed from the NFZ perspective across five CS etiologies: Cushing’s disease, adrenal adenoma, adrenocortical carcinoma, ectopic ACTH syndrome, and other or unknown etiologies of CS. QALYs and life-years were modelled as equal-outcome checks. Deterministic and probabilistic sensitivity analyses assessed robustness.
RESULTS: Metyrapone was associated with equivalent health outcomes and lower total costs versus osilodrostat across all assessed etiologies. Discounted lifetime per-patient NFZ savings ranged from PLN 258,098 in adrenocortical carcinoma to PLN 892,657 in adrenal adenoma; savings in Cushing’s disease were PLN 856,037. Savings were driven mainly by lower drug acquisition costs, with additional reductions in adverse-event management costs. Results remained robust in deterministic and probabilistic sensitivity analyses.
CONCLUSIONS: From the Polish public payer perspective, metyrapone was associated with consistent lifetime cost-savings versus osilodrostat across assessed CS etiologies, with equivalent clinical outcomes assumed based on available evidence. These findings suggest that metyrapone may offer an economically favorable option within the Polish treatment landscape of CS.