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 lessen the cost of care.
Prophylaxis for Prevention
Prophylactic use of clotting factor forms the foundation of care for patients with hemophilia. This preventive treatment regimen, given through regular infusions, is based on condition severity and helps to maintain enough clotting factor in the blood to prevent bleeds. It is now the standard of care for those with severe hemophilia and is associated with decreased physical pain, improved joint outcomes and better health-related quality of life.
Frequency of prophylactic infusions can vary significantly depending on the severity of the disease, activity level, patient-specific metabolism (half-life) and product used. Like other chronic conditions, compliance to this therapy can be challenging. New therapies have been developed that have longer half-lives, thus potentially requiring fewer weekly infusions. There is now at least one product on the market that requires a subcutaneous injection (needle is injected under the skin, between the skin and muscle) as opposed to an intravenous infusion, potentially improving adherence and reducing the need for frequent infusions.
Site of Care Treatment Options
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 Center. Both provide support services with some differences and distinctions.
Individuals with hemophilia or other inherited bleeding disorders rely on FDA-approved blood clotting factor therapies that fall in the specialty drug category. Since these drugs 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 insurers and pharmacy benefits managers (PBMs), and Hemophilia Treatment Center 340B pharmacies.
Hemophilia Treatment Centers
Because hemophilia is a complex and rare disorder, many providers do not have the experience or 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), to help address this. The objective was to improve expertise, access to care and outcomes for people with hemophilia.
HTCs are required to follow guidelines for treatment which were developed by the National Hemophilia Foundation’s Medical and Scientific Advisory Committee (MASAC) that promote evidence-based care and a multi-disciplinary team approach to integrated comprehensive care. Today, there are over 140 federally recognized HTCs across the US, most of which are embedded in an academic center or hospital. View the Hemophilia Treatment Center Directory.
In the HTC model, the team establishes an annual proactive comprehensive care plan for each individual that includes a hematologist, nurse coordinator, physical therapist, and social worker. Other care providers including dental professionals, genetic counselors, orthopedists, infectious disease specialists and pharmacists are either available onsite or via established partnerships to support the unique needs of people with hemophilia. Hemophilia Treatment Centers offer specialty pharmacy services, providing medications specific to the treatment of bleeding disorders.
Research shows that HTCs are effective at managing outcomes and the cost of care for patients with hemophilia, including decreased complications, reducing unnecessary visits to the ER (HTC patients are 47% less likely to use the ER), decreased number of bleeds: reduced number of infusions, proper dosing of clotting factor and adherence to therapy.
Program benefits also include:
- Annual meeting with the integrated care team to establish a proactive comprehensive care plan for the coming year
- Multiple points of contact throughout the year with providers, follow-up visits, coordination of care with all providers, surgical consultations and updates to the treatment plan
- Access to the federal drug discount program; may offer competitive and/or lower average pricing per unit
- Integrated medical/pharmacy coordination
- Unbilled, ancillary services such as nursing/telephone triage, care coordination, physical therapy, dental care, genetic counseling, nutrition counseling and assistance from social workers are available as part of integrated comprehensive care
- Monitoring of bleeding episodes with real time provider access if interventions are required
- Dose and adherence management
- Home delivery of clotting factor and home visits when needed
- Patient education and on-demand consultation
- Travel document planning and preparation
- Hospital support and training when in-patient services are required
- For students, annual school individualized education program (IEP) planning
- 24/7/365 telephone support and follow-up
HTCs are tailored to meet the needs of their patient population, with variation in characteristics and capabilities in terms of funding, geographic area covered, available provider specialties and patients served. For example, in Illinois there are outreach clinics where the HTCs take their clinic team on the road to eight different cities. This reduces the need for patients to travel long distances to receive care. This same approach is used by many HTCs throughout the country. View the Hemophilia Treatment Center Directory.
Understanding the Difference
It is important to make sure that specialty pharmacy providers serving the hemophilia and bleeding disorders community meet minimum standards. Ensure your specialty pharmacy is adhering to the NHF’s Medical and Scientific Advisory Council’s MASAC 188: Recommendation Regarding Standards of Service for Pharmacy Providers of Clotting Factor Concentrates for Home Use to Patients with Bleeding Disorders.
Not all specialty pharmacies are consistent in practices related to disabling auto-shipping and dose checking. Specialty pharmacies:
- Should not auto-ship medications used to treat hemophilia
- Should require the patient be contacted by phone, email or text message to refill a prescription
- Should require doses on hand be verified by the patient before shipping or patient pick-up