Investing in hemophilia care: benefits and costs for patients and society

Abstract

Despite the success of recent investments in health technology, inhibitor development still represents a major complication of hemophilia treatment. The introduction of new options, such as immune tolerance induction and the use of bypassing agents, has significantly improved treatment success in inhibitor patients in even the most challenging situations (eg, emergencies, home treatment, and surgery). However, these treatment advances are paralleled by significant increases in the cost of care for these patients, and economic constraints can cause limited access to optimal therapy. In order to optimize the benefits derived from use of the available resources, it is necessary to know and compare both present and future effects and costs of alternative options. Identification of the most efficient option allows implementation of appropriate investments, which must be considered as opportunities to improve patients' health and well-being. Implementing rational investments also may facilitate economic benefits in the future, with potential advantages not only for patients but also for their families and for society as a whole.