Economics of prophylactic treatment

Summary.  Clinical studies have shown significant improvement in outcomes with the use of prophylactic treatment, as well as apparent gains in health‐related quality of life. Given the high cost and still limited availability of factor concentrates, physicians, insurers and governments require cost‐effectiveness data to evaluate competing treatments (eg prophylactic vs. on‐demand treatment) and to set priorities within the context of each country's financial resources. To date, the few published studies of the cost‐effectiveness of haemophilia prophylaxis have generally focused on the reduction in frequency of bleedings as an intermediate outcome, and not on long‐term health outcomes, such as the development of arthropathy and its social and economic costs. Since all prophylactic measures are investments for the future, long‐term economic consequences need to be carefully studied and evaluated.