Managing hemophilia requires intensive, integrated, coordinated, and individualized medical and pharmacy care. Best practice approaches to managing hemophilia incorporate these elements to achieve optimal outcomes and reduce the cost of care. Employers need to consider treatment and site of care options.
Treatment and Site of Care
The care and treatment of members with hemophilia is typically tied to traditional benefit plan design, carrier networks, and related provider sites of care. Where care is directed has an important impact on outcomes for members and costs for employers. Most individuals with hemophilia self-infuse at home and get their medications from a specialty pharmacy or Hemophilia Treatment Centers (HTCs). Both provide support services with some differences and distinctions.
Specialty Pharmacies
FDA-approved factor replacement therapy falls in the specialty drug category. Since these factor replacement medications require special handling and storage, they are provided by specialty pharmacies.
Unlike retail or traditional pharmacies, specialty pharmacies provide a range of support services targeted at patient education, care coordination, clinical management, adherence programs and disease management. Examples include independent specialty pharmacies, pharmacy divisions owned by national retail chains, specialty pharmacies owned by carriers and pharmacy benefits managers (PBMs) and Hemophilia Treatment Center (HTCs) 340B pharmacies.
Hemophilia Treatment Centers
Because hemophilia is a complex and rare disorder, many providers do not have the expertise to treat and manage the condition effectively. In the mid-1970s, the Federal Office of Maternal and Child Health of the Health Resources and Services Administration (HRSA) established a network of federally designated centers of excellence, known as Hemophilia Treatment Centers (HTCs).
HTCs are required to follow guidelines for treatment which were developed by the National Hemophilia Foundation’s Medical and Scientific Advisory Committee (MASAC) and include a multi-disciplinary team approach to integrated and evidenced-based care. Today, there are approximately 150 federally recognized HTCs across the U.S., most of which are embedded in academic centers or hospitals. View the Hemophilia Treatment Center Directory here. Though not every state contains a hemophilia treatment center, all areas of the country can access best in class care through outreach clinics which include HTC staff traveling to outside areas to provide care closer to where the patients live. The telemedicine movement has created a new avenue to connect patients with treatment centers, allowing patients anywhere in the country to conveniently access this gold standard of care.
In the HTC model, a multi-disciplinary team establishes a personalized plan for each patient that includes a hematologist, nurse coordinator, physical therapist, and social worker. Other care providers are available onsite or via established partnerships to support the unique needs of people with hemophilia, including dental professionals, genetic counselors, orthopedists, women’s health providers, infectious disease specialists, and pharmacists. HTCs offer specialty pharmacy services, providing medications specific to the treatment of bleeding disorders. This integrated pharmacy model allows real time coordination between the clinical care team to address adherence issues and/or medication efficacy concerns.
Research shows HTCs are effective at managing outcomes and the cost of care for patients with hemophilia, including fewer lost workdays, decreased complications—reducing unnecessary visits to the emergency department (HTC patients are 47% less likely to use the ER)—decreased number of bleeds, reduced number of infusions, increased proper dosing of medication, and improved adherence to therapy.