RESUMEN
OBJECTIVE: This study aims to estimate the budget impact of luspatercept reimbursement as an adjuvant to the standard management of ß-thalassaemia major in Cyprus, from a societal perspective, and assess the financial feasibility of its inclusion in the ß-thalassaemia armamentarium. METHODS: A 5-year horizon budget impact model was developed to determine the budget impact of reimbursing luspatercept for the management of ß-thalassaemia major in Cyprus. Two treatment discontinuation scenarios were elaborated. In the first scenario, luspatercept is reimbursed complementary to best supportive care, and a dropout rate of 40% is assumed based on published real-world data, while for the second scenario a dropout rate of 25%, is assumed as per the clinical trial data. Input parameters were retrieved from the phase III clinical trial of luspatercept, literature, and expert opinion consensus. One-way sensitivity analyses were conducted for both scenarios. RESULTS: The addition of luspatercept to the standard management of ß-thalassaemia major in Cyprus imparted an incremental budget impact ranging from 21,300,643 to 25,834,368, depending on the drop-out rate scenario assumed. Results were sensitive to the number of eligible patients and dose per patient. CONCLUSION: The potential reimbursement of luspatercept will wield a substantial impact on Cyprus total pharmaceutical expenditure and it is therefore imperative to affix a reimbursement framework that will allow the payer to mitigate uncertainty stemming out of the scarce clinical data and the inherently complex therapeutic landscape of ß-thalassemia management.