Vice-President, Medical World Federation of Hemophilia La Jolla, United States
Status of Global Inequities of Treatments in Persons with Bleeding Disorders
Despite the remarkable technological advances in treatment of bleeding disorders over the past 60 years, most of the world has been left far behind. Advances in hemophilia A and B treatments, from extended half-life products to non-factor replacement therapies, have benefited primarily patients in high income countries, with some uneven penetration into upper middle income countries. Lower income countries purchase variable, but small amounts of coagulation products and some receive WFH humanitarian aid, which can include newer, more effective products. However, taken together, the treatments are mostly insufficient to provide 2025 standards of care. The 2020 WFH Treatment Guidelines called for troughs of at least 3-5%, and newer products can easily surpass these levels.
However, as technology continues to advance, a new FDA-approved rebalancing agent is priced at over $800,000 USD and licensed hemophilia A and B gene therapies are priced in the millions of USDs. If these prices are indications of trends in future licensed products, there will likely be no access in lower income countries. Thus the health equity gap, which is huge, is becoming a larger chasm as new technologies are introduced. This is so obviously the wrong direction.
A second area of disparity and health inequity is women with bleeding disorders. Symptomatic carriers of the FVIII or FIX genes have hemophilia, a fact that continues to go unrecognized by many affected persons and health care providers alike. As newer products are introduced, these may be of particular benefit to affected women, but outreach, education, and treatment are impeded by preconceived biases.
A third area of disparity and health inequity includes persons with rare bleeding disorders, including von Willebrand disease (VWD). Most of the lack of progress in new treatments in these disorders is the direct result of insufficient markets to provide a return on investment by pharmaceutical companies. This is slowly changing, as rebalancing agents and non-factor products are being recognized as having therapeutic benefit in some of these disorders (e.g., Glanzmann’s thrombasthenia, some forms of VWD, other factor deficiencies).
Thus while we embrace the technology advancements that bring us closer to lower treatment burdens and improved outcomes, we struggle with the growing health inequities to which they contribute. Viable solutions are needed through collaboration of all stakeholders, including the pharmaceutical industry and governments.
Learning Objectives:
Technology advancements have been extraordinarily in bleeding disorders. But most of the world is left far behind. Health inequities are growing, not shrinking in our global community. Determined actions are urgently needed to increase access to newer technology products for all.
