FAR-BENE Study: pharmacoeconomics and well-being in hemophilia. Monitoring outcomes in light of pharmaceutical expenditure with focus on patients undergoing therapeutic switch.

Hemophilia, a rare bleeding disorder, raises significant pharmacoeconomic challenges, particularly with the emergence of innovative therapies. This study evaluates clinical and patient-reported outcomes and expenditures in hemophilia patients focusing on those who underwent therapeutic switches between 2017 and 2023. This retrospective study included severe hemophilia patients. Expenditure was analyzed based on clotting factor consumption, while clinical well-being was assessed using ABR, NRS for pain, joint health scores (HJHS, HEAD-US) and trough plasma levels. Sub-analysis was conducted by applying different therapeutic groups. Statistical significance of differences between paired measures (pre [T0] vs post [T1]) was examined using the Wilcoxon Sum Rank test. Seventy patients were included. Total expenditure was € 12,947,580.44 in 2017 and € 12,967,576.92 in 2023. Among 45 patients who switched therapy, the median number of infusions/year significantly decreased from 156 to 91 (p<0.001), ABR from 1 to 0 (p<0.001) and NRS from 3,5 to 0 (p<0.001). Joint health did not show improvement with a slight but not statistically significant increase (HJHS from 10 to 15; HEAD-US from 7 to 8). Median trough increased from 3 UI/mL to 5 UI/mL (p=0.181). Patients were grouped for analysis based on homogeneous treatment regimens. This study is the first effort to conduct comprehensive analysis of the impact of therapeutic choices on well-being and cost of care in hemophilia Center. The study underscores the need for value-based approach to hemophilia care, integrating expenditure and clinical outcomes. Adopting validated economic weight outcome in future pharmacoeconomic evaluations will be essential for decision making.