Towards a value-based approach in hemophilia: indications from the FAR-BENE study

Abstract

The significant improvements in hemophilia management and treatments observed in the last decade changed expectations of hemophilia community in terms of achievable health status but also raised concern on economic implications associated with these new approaches. In this context the need of new indicators for the desired health outcomes made achievable by the transformation of treatment has been developed and suggested in the literature, providing the basis for the development of value-based healthcare in hemophilia. The FAR-BENE (Pharmacoeconomic and Well-Being in Hemophilia) study, recently published, represents an important and timely effort to bridge this gap. Even with limits, the FAR-BENE study offers a clear signal of the need and possibility to move beyond the measure of factor consumption and bleeds and implementing a value-based approaches that integrates economics aspects with comprehensive, clinical and patient-centric outcomes. A further effort is now required by the healthcare system to create the appropriate conditions to adopt and implement standardized value-based approaches, ensuring the possibility of improving the patients’ health by maximizing the healthcare resources available.