In a presentation entitled "Hemophilia Management: Collaborating in a New Era to Optimize Cost and Clinical Outcomes in Managed Care," 5 key thought leaders and managed care authorities shared their insights on the changing therapeutic landscape for hemophilia management.
In an afternoon educational session entitled "Hemophilia Management: Collaborating in a New Era to Optimize Cost and Clinical Outcomes in Managed Care,"Jeffrey D. Dunn, PharmD, MBA, of SelectHealth, Inc, began by stating that hemophilia, an inherited bleeding disorder that is consequent to an absent or decreased amount of blood clotting factor, is the most expensive chronic condition currently being treated by any managed care organization. The appropriate management of hemophilia, he noted, is the key to minimizing and preventing the long-term complications and issues that may lead to higher costs. However, as a chronic, complex disease that is less prevalent than other diseases but highly expensive to manage, there are numerous challenges that complicate the movement to improve medical and pharmacy management for hemophilia. Fortunately, organizations such as the National Hemophilia Foundation (NHF) and the unique patient care delivery model provided by hemophilia treatment centers (HTCs) have helped to grow a large and close-knit community that promotes advocacy and drives progress in the management of hemophilia.
Taking the stage afterwards was Marion Koerper, MD, of University of California, San Francisco School of Medicine and medical advisor to the NHF. She began her presentation, "Current Practices in the Treatment of Hemophilia: A Primer for Managed Care Professionals" with an overview of hemophilia epidemiology, noting an estimated US incidence of 20,000 individuals, with approximately 80% and 20% suffering from hemophilia A (HA) and hemophilia B (HB), respectively. There are a total of 10 clotting factors and a deficiency of any 1 factor results in dysfunctional clot formation: HA results from a factor VIII deficiency and HB, factor IX. Koerper emphasized that hemophiliacs do not bleed faster, but longer than normal. With increased severity, the clinical sequelae of hemophilia are tied to substantial morbidity, such as joint and head bleeds. Delving into treatment, Koerper pointed out that the goal of treatment is to replace missing clotting factors with either plasma-derived or recombinant factor replacement products through an episodic or prophylactic therapeutic strategy. Whereas episodic treatment occurs after a bleed has begun, prophylactic treatment, though expensive, is initiated in advance to prevent bleeding. Furthermore, she continued, while primary prophylaxis is continued indefinitely to prevent bleeding episodes, secondary prophylaxis is initiated for a limited duration to rehabilitate joints and stop bleeds. Inhibitors for hemophilia, which are immunoglobulin G antibodies that neutralize the clotting factor, are associated with increased treatment complications and the highest costs. To combat the high clinical and economic burden of hemophilia, a network of 140 HTCs, which are federally recognized centers of excellence that have been linked to improved outcomes in patients with hemophilia, have been implemented throughout the United States. Patients that are managed through HTCs experiencing fewer bleeds, decreased hospitalizations, reduced mortality, and a greater use of home therapy.
Following Koerper was Raulo S. Frear, PharmD, the general manager of Regence Rx, whose presentation was titled "Managed Care Strategies for General Hemophilia and Hemophilia with Inhibitors." Optimal care, he noted, involves the balancing of benefits, risks, and costs, especially because, despite its low relative incidence, hemophilia incurs higher aggregate costs of care. Goals for patients with hemophilia include a reduction of joint disease and serious bleeding episodes, while improving the patient's ability to participate in normal activities and perform academically. Current medical and pharmacy utilization controls, to ensure appropriate use of therapeutic agents, Frear continued, often include prior authorization or precertification for therapies. To further benefit patient outcomes, case management services are often made available to increase access to treatment and improve the quality of care, and may also involve a coordination of care between HTCs and specialty pharmacy providers (SPPs). One such example of cost-containment, according to Frear, was the Utah Hemophilia Factor Project, which was able to cut unnecessary healthcare expenditures while improving the quality of patient care and meeting the medical needs of patients with hemophilia. In addition, there are several resources that are available to help deliver high quality and effective care for patients with hemophilia, such as HTCs and SPPs, which can provide the care coordination, education, and support needed to drive improved outcomes.
Sue Geraghty, RN, MBA, of the Hemophilia and Thrombosis Center of the University of Colorado, Denver was next, with a presentation titled "Applying a Collaborative Approach to Hemophilia Management." She emphasized that while there are many team members in HTCs, the patient is always the most essential, and all processes should be focused on improving the patient's quality of life and outcomes. HTCs, she stated, are models of comprehensive care for hemophilia, providing the individualized education, research, outreach, emotional support, and coordination of care for patients regardless of insurance coverage. Previous studies have shown that patients who received care outside of an HTC experienced a 70% and 40% increase in mortality and hospitalization rates, respectively. Geraghty contended that all patients with hemophilia require individualized care and should have access to all appropriate treatment modalities, since decreased access increases morbidity and costs. To accomplish the goals of increased access to care, improved patient outcomes, and decreased economic burden, she indicated that many opportunities exist to benefit the hemophilia community, most notably, partnerships of HTCs with managed care and specialty pharmacy providers.
The last speaker to take the stage was William D. LaPiana, MT (ASCP), the regional director of Accredo, whose presentation was entitled "Hemophilia Specialty Pharmacy Management Services: Steps for Success." Bleeding disorders, he said, are a low-frequency disease, and as such, there is a limited expertise at many health plans. Furthermore, as the nation becomes more mobile, the continuity of care becomes a more essential facet to improving clinical and economic outcomes. While each SPP offers a different area of expertise, service, and capabilities, as a whole, SPPs play multiple and important roles in patients with hemophilia, helping to provide medication oversight, management of comorbidities, patient support services, education, care coordination, and open channels of communication with all parties involved in care delivery. There are numerous variables that may impact the total cost of hemophilia care, such as disease severity, HTC utilization and compliance, and site of care; a multidisciplinary, continuum-based approach to patient care delivery, LaPiana argued, is needed. Effective disease management can improve outcomes and the total cost of management, and by collaborating with the physician, patient, and HTC, SPPs are able to enhance the continuity of care and ensure optimal patient outcomes. Ultimately, the goal of all initiatives is to minimize the impact of hemophilia and enhance a patient's quality of life.