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.
Prophylaxis for Prevention
Prophylactic use of clotting factor or substitution therapy forms the foundation of care for patients with hemophilia. These preventive treatment regimens are given through intravenous (IV) infusions or subcutaneous injections (needle is injected under the skin, between the skin and muscle). They help to prevent bleeding episodes by maintaining factor levels in the blood or replacing the function of missing clotting factors.
Frequency of treatment can vary significantly depending on the severity of the disease, the time it takes for the activity level of the infused factor to drop by half (half-life), and type of medication used. The average patient treated with clotting factor replacement therapy infuses three times per week. Substitution therapy, administered through subcutaneous injections, is offered with multiple dosing options (weekly, every two or four weeks).
Prophylaxis 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. There are a variety of prophylaxis treatments available. The Institute for Clinical and Economic Review (ICER) compared different prophylaxis treatments for Severe Hemophilia A and Hemophilia A with Inhibitors.
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.
FDA-approved blood clotting factor or substitution therapies fall in the specialty drug category. Since these 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) 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 patient 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. HTCs 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.
Employer and patient benefits also include:
- Access to the 340B Drug Pricing Program; may offer competitive and/or lower average pricing per unit
- Integrated medical/pharmacy coordination
- 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
- 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
- Dose and adherence management
Additional patient benefits include:
- Annual meeting with the integrated care team to establish a proactive comprehensive care plan
- 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 National Hemophilia Foundation’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 the same. HTCs must adhere to specific guidelines so it’s imperative to know if your specialty pharmacy is following these guidelines. It’s also important to ensure they are consistent in practices related to disabling auto-shipping and dose checking. Your specialty pharmacy:
- Should not auto ship medications
- Should contact the patient monthly
- Should verify inventory on hand before shipping additional doses
If these practices are not in place, employer costs can dramatically increase.