The National Hemophilia Foundation (NHF) is the nation’s leading advocacy organization for individuals with bleeding disorders. Our mission is to ensure that individuals affected by hemophilia and other bleeding disorders have timely access to quality medical care, therapies and services, regardless of financial circumstances or place of residence.
Hemophilia and related bleeding disorders are rare, complex genetic conditions for which there are no known cures. Individuals with inherited bleeding disorders experience spontaneous and prolonged internal bleeding into their joints and soft tissues. To effectively manage these disorders, patients require life-long infusions of clotting factor therapies that replace the missing or deficient blood clotting proteins. These products must be infused intravenously within 3 hours to prevent debilitating and life-threatening internal bleeding. While today’s therapies are safer and more effective than ever, they are also more costly than other types of medication. For example, cost of a single dose for an adult with severe hemophilia can be $3000 or more.
Clotting factor therapies are biological products either derived from human blood plasma or else produced using recombinant technology; there are no generic equivalents. Moreover, these therapies are neither pharmacologically nor therapeutically equivalent because of variables such as how they are manufactured (i.e., recombinant or plasma-derived), purity, and half-life, among other factors.1 Collectively, these characteristics mean that an individual’s response and tolerability is unique for a specific product. Switching from one product to another may result in the formation of an inhibitor, which is an immune response to the clotting factor that neutralizes it and renders it ineffective; thus the bleeding cannot be stopped. Eliminating a patient’s inhibitor necessitates an extremely costly protocol. For these reasons, NHF’s Medical and Scientific Advisory Council (MASAC) recommends that individuals retain access to the specific FDA-
approved clotting factor products prescribed for them.2
We recognize the challenges that hospitals face when dealing with a low prevalence, high-cost, chronic condition such as hemophilia. The high cost of these therapies and the limited number of these patients that many Emergency Departments encounter means that many hospitals do not have rapid access to the full range of FDA-approved clotting factor products. Because of the high cost and the large number of different products available, many hospitals cannot afford to stock these products in their pharmacies. Thus many hospitals permit patients to bring their prescribed product to the Emergency Department in order to facilitate their being treated there in a timely manner. This practice allows patients to remain adherent to the treatment regimen established by their physician and minimizes possible costly complications, including developing an inhibitor due to a change from their usual clotting factor product and having a lengthy inpatient hospital stay due to a delay in treatment because the appropriate clotting factor concentrate is not immediately available in that hospital’s pharmacy.