Hemophilia and Managed Care: Partnering to Achieve Cost-Effective Care

Exploring how health plans and hemophilia treatment centers can better work together to deliver high-quality care and manage costs associated with hemophilia.
Published Online: September 30, 2011
Amy Shapiro, MD; Steven W. Pipe, MD; Sue Geraghty, RN, MBA; Edmund J. Pezalla, MD, MPH; James A. Jorgenson, RPh, MS, FASHP; Celynda G. Tadlock, PharmD, MBA; Karen Wulff, RN; Robert Kaminsky, MBA; and Craig Kessler, MD
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This activity has been planned and implemented in accordance with the Essential Areas and Policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint sponsorship of Medical Education Resources (MER) and the National Hemophilia Foundation, the P&T Society, and MedSpan. MER is accredited by ACCME to provide continuing medical education for physicians.

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The content and views presented in this educational activity are those of the authors and do not necessarily reflect those of Medical Education Resources, the National Hemophilia Foundation, the Pharmacy & Therapeutics Society (PTS), MedSpan, and/or Novo Nordisk. The authors have disclosed whether there is any discussion of published and/or investigational uses of agents that are not indicated by the US Food and Drug Administration in their presentations. The opinions expressed in this educational activity are those of the faculty and do not necessarily represent the views of Medical Education Resources, the National Hemophilia Foundation, PTS, MedSpan, and/or Novo Nordisk. Before prescribing any medicine, primary references and full prescribing information should be consulted. Any procedures, medications, or other courses of diagnosis or treatment discussed or suggested in this activity should not be used by clinicians without evaluation of their patient’s conditions and possible contraindications or dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities. The information presented in this activity is not meant to serve as a guideline for patient management.
In late 2010, the Pharmacy & Therapeutics Society, with significant contribution from the National Hemophilia Foundation (NHF), convened a series of 6 educational webinars for payer executives. The primary objective of the webinars was to explore how health plans and hemophilia treatment centers (HTCs) can better work together to deliver high-quality care and manage costs associated with hemophilia. Each webinar’s faculty comprised an HTC hematologist, an HTC nurse, and a managed care executive. Each webinar was part of an accredited continuing education program. These webinars were made possible by an unrestricted educational grant from Novo Nordisk.

Most of the 66 webinar attendees represented the medical and pharmacy departments from commercial, Medicaid, and Medicare health plans located across the country. The health plans were diverse, ranging from national plans with multiple lines of business to small regional plans and state Medicaid agencies. A few attendees represented pharmacy benefit managers (PBMs) and specialty pharmacies. This article is based on the educational materials presented and the discussions that transpired during the webinars.

Hemophilia is a chronic, rare disease with significant individual patient cost. Over the last few decades, with the introduction of new treatment modalities, the management of pediatric and adult hemophilia patients has progressively improved outcomes and resulted in increased longevity and decreased morbidity of disease. However, the transmission of viral blood-borne infections via replacement products (largely prior to 1990), clotting factor deficiency–associated arthropathy, and spontaneous and traumatic bleeding episodes have affected a significant segment of the hemophilic population. Overall care for bleeding-disorder patients is complex and requires an expert, dedicated multidisciplinary team. As hemophilia is a rare disorder, health plans often do not invest as many resources to evaluate formulary and coverage issues as they might for higher prevalence diseases with greater associated patient numbers and total cost.

Many webinar attendees suggested that their health plans do not fully understand the comprehensive medical and support services that HTCs provide and the positive impact those services have on patient quality of life and cost of care. Also, many webinar attendees reported that they have not fully evaluated the complexities of hemophilia and their impact on medical and pharmacy benefit design, formulary development, therapy acquisition, and patient management. Many webinar attendees believed that payers and HTCs might not have fully explored appropriate avenues to productively work together.

As this article demonstrates, a closer working relationship and better mutual understanding between health plans and HTCs could improve outcomes for hemophilia patients while providing a more favorable cost-to-benefit ratio. Treatment of rare diseases like hemophilia is unlikely to result in cost offsets that are greater than or equal to the drug costs. Managed care’s goal should be to optimize care and outcomes in a way that minimizes the cost of treating the rare condition. Managed care also should keep in mind that the model of care for hemophilia, as described below, can be a model of care for many other congenital diseases with costly treatments, such as those treated with enzyme-replacement therapies.


Hemophilia is an X-linked recessive bleeding disorder that affects approximately 1 of 5000 live male births.1 Currently in the United States, approximately 400 to 500 babies are born with hemophilia each year, and there are a total of approximately 30,000 affected individuals. Males are almost exclusively affected, yet females who carry the genetic mutation may be symptomatic, depending on the clotting factor level.2

Hemophilia A, characterized by decreased activity and/or levels of Factor VIII, affects 80% to 85% of hemophilia patients. Hemophilia B, characterized by decreased activity or levels of Factor IX, affects 15% to 20% of hemophilia patients.2

As shown in the Table, most hemophilia patients are classified as having severe disease and have levels of Factor VIII or IX of less than 1%. They experience approximately 2 to 4 bleeding episodes per month, many of which are spontaneous (ie, there is no apparent inciting cause). The remaining affected patients have a less severe form of the disease, with Factor VIII or IX activity levels between 1% and 40%.

Hemarthrosis: A Common Complication of Hemophilia

Patients with severe hemophilia frequently experience spontaneous intra-articular hemorrhages, mainly in the ankles, knees, and elbows. Over the long term, repeated episodes of hemarthrosis may cause irreversible damage to the joint, leading to hemophilic arthropathy, which is characterized by joint stiffness, chronic pain, and a severely limited range of motion.3

In a longitudinal 6-year study performed during the 1980s and 1990s, 90% of people with severe hemophilia experienced chronic degenerative changes in 1 to 6 joints by the age of 25 years.4 In another study, 42% of patients with severe hemophilia were restricted in their physical activities because of arthropathy.5

A critical factor for avoiding the occurrence of hemophilic arthropathy is the prevention of articular hemorrhages.3 However, despite regular infusions of antihemophilic concentrate at an early age, recurrent joint bleeding and the possibility of hemophilic arthropathy still persist in some patients.6 Innovative strategies for the preventionand treatment of this common and serious complication are therefore required.

Several treatment regimens are utilized for the care of patients with hemophilia. One regimen, on-demand therapy, is defined as the administration of clotting factor concentrates at the time of an acute bleed. Another regimen, prophylaxis, consists of the administration of clotting factor concentrate in advance of active bleeding with the intention of preventing bleeding events and associated subsequent damage to the affected area. Prophylaxis can be primary (ie, prevention of bleeding events starting early in childhood before the development of a hemarthrosis) or secondary (ie, prevention of bleeding events in those patients with a history of previous bleeding events). Prophylaxis has been documented to improve the joint health and quality of life for many patients with severe hemophilia. Pain also may be averted or mitigated by prophylaxis strategies.7

Available data demonstrate that prevention of bleeding episodes from an early age enables hemophilia patients to avoid or reduce the clinical impact of joint disease and improve psychosocial development and quality of life.8 For example, young patients on prophylaxis are better able to participate in physical activities, regularly attend school, and achieve their academic and personal goals.

Hemophilia and Inhibitors

One of the most serious and costly complications of hemophilia is inhibitor formation.9 As many as 20% to 30% of severe hemophilia A patients10 and approximately 1.5% to 3% of those with severe hemophilia B11 develop a neutralizing antibody—called an alloantibody inhibitor—directed against the exogenously administered factor replacement therapy required to treat or prevent bleeding episodes. Most inhibitors develop early in life at a median age of 1.7 to 3.3 years and after a median of ~9 exposure days (ie, the number of days the patient was exposed to any clotting factor replacement therapy).12

When inhibitors develop, the administration of normal replacement therapy to achieve hemostasis may fail, requiring the use of “bypassing therapies.” For patients with high titer inhibitors, examples of bypassing therapies include rFVIIa and an activated prothrombin complex concentrate. When inhibitors develop, there is a decreased ability to perform needed or elective surgery and an associated increase in morbidity and mortality.13

While hemophilia patients with inhibitors comprise a minority of an HTC’s patient population, they often require many times the hours of care and volume of services utilized by their counterparts without inhibitors. Patients with inhibitors require intensive treatment interventions with frequent telephone interaction, education, emotional support, and appropriate plans for pain management.

The total cost of care for patients with alloantibody inhibitors is much greater than that for patients without inhibitors. In addition to requiring more costly therapies, hemophilia patients with inhibitors are often hospitalized at a higher rate. Also, hospitalizations for inhibitor patients may be more prolonged or complicated.14

Regardless of the severity of disease and presence of inhibitors, the treatment of hemophilia is complex and requires expert evaluation and knowledge of the disease and individual patient. Therapies are costly and dosing varies from patient to patient. An experienced physician’s medical direction is required for appropriate care.


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