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Supplements Incorporating Emerging Innovation in Hemophilia A and B: Tailoring Prophylaxis and Management Stra

Economic Costs of Hemophilia and the Impact of Prophylactic Treatment on Patient Management

Sheh-Li Chen, PharmD, BCOP
Hemophilia is a rare congenital bleeding disorder characterized by spontaneous and potentially life-threatening bleeding episodes. In addition to its clinical burden on the patient, the condition also places a significant economic burden on healthcare payers, patients/caregivers, and society. Hemophilia is associated with staggering direct costs from hos-pitalizations, outpatient visits, and drug treatments, as well as high indirect costs from diminished work productivity and absenteeism from work and school. Additionally, hemophilia incurs tremendous intangible costs, including reduced quality of life, pain and suffering, and the emotional and physical toll on the patient and caregivers.

The evolution of treatment patterns in hemophilia has transformed the once-fatal disease into a chronic but potentially well-managed condition through the use of prophylaxis treatment. However, other complications, such as development of inhibitory antibodies, have added to the complexity of managing the disease and its costs. To ensure optimal treatment outcomes and disease management, there is a critical need to understand the utilization of healthcare resources in the treatment of hemophilia and to educate patients on the importance of treatment adherence and compliance to reduce long-term effects on musculoskeletal health. 

Am J Manag Care. 2016;22:S126-S133

    Hemophilia is a rare X-linked congenital bleeding disorder characterized by bleed-ing manifestations, including spontane-ous bleeding episodes into muscles and joints.1,2 Joint bleeding, which commonly affects weight-bearing joints, such as knees or ankles, can lead to the development of painful, disabling hemophilic arthropathy.3 These patients are also at risk for life-threatening bleeding, including intracranial hemorrhage. Clotting-factor concentrates are used for the treatment and prevention of these bleeds.

    The major complication of the use of clotting-factor concentrates is the development of inhibitors, alloantibodies that neutralize the infused factor. Consequently, bypassing agents have to be used for the treatment of bleeds in these patients. The bypassing agents are not as effective as the clotting factors VIII (FVIII) and IX concentrates, which further inhibits effective management of bleeding episodes; this places patients with hemophilia at greater risk for target joint development (3 or more bleeds in the same joint in 6 months) and progressive arthropathy that can result in permanent disability.4,5 A complication observed in the hemophilia population in the 1980s was the transmission of HIV, along with the hepatitis B virus and hepatitis C virus (HCV), with the use of plasma-derived clotting-factor concentrates. There was a high mortality rate among individuals with hemophilia who developed these viral infections in the 1980s and 1990s. Transmission of these infections, however, has been greatly reduced with the use of recombinant clotting-factor concentrates, the implementation of screening, and effective viral eradication steps in the manufacturing process of the plasma-derived clotting-factor concentrates.

     Over the past 20 years, significant improvements have been made in the management of patients with hemophilia, including advances in acute and prophylactic treatment and the availability of safe and effective clotting-factor concentrates. These advances have resulted in increases in life expectancy.6,7 However, the life expectancy for men with severe disease is still 15 years less than the general population.6,8 Sixty percent of individuals with hemophilia have severe disease, with a median age of 1 month at diagnosis.9 The debilitating joint disease that results from recurrent bleeding episodes, along with the risk of inhibi-tor development, has significant negative impacts on the quality of life (QoL) of these individuals.10-12 The combined factors of ineffective management, pain, potential disability, and reduced QoL translate into a substantial burden to affected individuals, their caregivers, and society.13-15

Economic Burden of Hemophilia

     Although hemophilia affects only a small portion of the population in the United States, it is associated with high aggregate costs and imposes a high financial burden on individuals, healthcare systems, and society in general. Hemophilia is a chronic condition that requires lifelong treatment, with individual costs varying based on disease severity, complications, and treatment regimen.14 The majority of these costs are direct costs, which include antihemophilic medication, clinician visits, hospitalizations, medical and surgical procedures, and laboratory tests.13,16 Indirect costs are those associated with reduced produc-tivity and increased absenteeism caused by hemophilia, its treatment, resulting disability, and death. To maintain perspective, the intangible costs include the impact of the disease on QoL, as well as the emotional and psychological effects of the disease; the pain and suffering resulting from hemophilia are also important to consider.13

Indirect Costs

Complications, such as recurrent bleeding, affect the productivity of people with hemophilia and their caregiv-ers in terms of absenteeism from work and/or school. Those patients with inhibitors are at increased risk for bleeding and associated complications. A survey of 90 caregivers and individuals with hemophilia and inhibitors evaluated the relationship between health-related quality of life (HRQoL) and productivity, as measured by absenteeism.17 Dependent clinical and treatment factors associated with HRQoL included physical, social, and emotional functioning, along with bodily pain, general health, vitality, and mental health.

     The study found that among employed patients, physical HRQoL deficits contributed to substantial work time missed or compromised. On-demand treatment and the increased number of hemorrhages were negatively associated with physical component scores (P <.05), which were themselves negatively associated with missed work or school days (P <.001). Over a 12-month period before the start of the study, patients with hemophilia reported an average of 20.2 low productive days and 25.7 missed days at work/school. Caregivers reported an average of 19.1 low productive days and 19.1 missed days at work/school. It was concluded that reduction in the number of hemorrhages in patients with hemophilia would likely improve physical functioning and productivity.

     The Hemophilia Experiences, Results and Opportunities (HERO) initiative was developed to bring about greater understanding and awareness of the psychosocial issues facing individuals with hemophilia.18 The study found that 80% (n = 537) of patients with hemophilia reported either a very large, moderate, or small negative impact of hemophilia on their employment. Forty percent (n = 243) selected their job/training to take into account the needs relating to their hemophilia, and 22% (n = 132) believed that they had lost a job in the past because of their hemophilia. Furthermore, 63% (n = 351) of parents of children with hemophilia reported that they felt that having a child with hemophilia had a negative impact on their employment. Although 35% felt that their child’s current treatment allowed them to work in most situations, 29% said they selected their job/training to take their child’s hemophilia and his needs into account. Seventeen percent had voluntarily left their job in order to care for a child with hemophilia.19 In a more recent study of 474 individuals with hemophilia, 25% of part-time participants and 30% of unemployed participants attributed their employment status to hemophilia. Nineteen percent of parents who had a child with hemophilia were underemployed; underemployment was significantly more likely in adults with severe hemophilia and parents of children with severe hemophilia. Overall, the cost of underemployment due to hemophilia was estimated at almost $4 million per year.20

     HRQoL among individuals with hemophilia is considerably impaired, mainly due to the pain and disability associated with hemophilic arthropathy. However, the HRQoL of untreated patients with inhibitors is even poorer.11,12 The HERO study showed that those with hemophilia have poor levels of HRQoL, in terms of mobility, pain/discomfort, self-care, usual activities, and anxiety/depression (Figure 111). In fact, 89% (n = 598) of patients with hemophilia reported that pain had interfered with their daily life in the past 4 weeks, and 301 patients with hemophilia (50%) reported constant pain.11

     Psychosocial issues may also prevent individuals with hemophilia from living the life they desire. The HERO study found that psychological or psychiatric conditions were the most common group of comorbidities that were both related and unrelated to hemophilia.18 Thirty-five percent of parents in the study felt that hemophilia had influenced their son’s relationship with them, 46% felt it influenced his relationship with friends at school, and 25% said it made him feel isolated at school.11

Direct Costs

     There are several variables that make estimating the cost of care for hemophilia challenging, including the different types of hemophilia, disease severity, frequency of bleeding episodes, presence of inhibitors, type and frequency of treatment, and payer source (private insurance vs Medicare).21 Published estimates suggest that mean healthcare costs for patients with hemophilia in the United States reach upward of $140,000 per year in the absence of inhibitors.22,23 Based on 2010 Medicare spending, treatments for hemophilia are the most costly drug average per beneficiary.24

     Data from 2002 to 2008 were collected through the Market-Scan Commercial and Medicare Research Databases to estimate mean and median medical expenditures during 2008 of 1164 males with hemophilia enrolled in employer-sponsored insurance plans. Thirty-three percent of the patients had at least 1 emergency department (ED) visit during the 11-month enrollment period, with an average of 2.8 admissions. Fourteen percent of patients had at least 1 hospital admission during the enrollment period, with an average of 1.4 admissions (Table 123).22 The overall annual average cost per patient for males with hemophilia was $155,136 (median $73,548), with higher costs for those with hemophilia A ($162,054 [median $78,598]) than those with hemophilia B ($127,194 [median $55,220]; P = .06) (Figure 222,23).22,23 Eighty percent of the patients included in the final analysis had hemophilia A.22

    Using the same database, the cases of 435 males with hemophilia who were enrolled in Medicaid for at least 11 months between 2004 and 2008 were identified.23 About one-third of all patients with hemophilia in the United States are covered through their state’s Medicaid program,21 and most with hemophilia qualify based on disability. In the Medicaid population, the average expenditure in 2008 was $142,987 (median $46,737) per patient, similar to patients in the employer-sponsored insurance group. The incidence of admissions to a hospital and visits to the ED were significantly higher for patients in the Medicaid group than for patients covered by an employer-sponsored insurance program (Table 123).23

    Lifelong treatment with factor-replacement therapy, either as prophylaxis or as acute or on-demand therapy, is the mainstay of hemophilia management. In fact, spending on factor replacement therapy makes up over 80% of the total direct expenditures for patients with hemophilia A and B.21-23 Similar results were seen in a broader cost analysis, where data were collected from the PharMetrics Patient-Centric database, which was derived from at least 73 US health plans, covering more than 43 million beneficiaries.14 The annualized costs, in 2004 dollars, was similar to other studies discussed,22,23 with anti-hemophilic medication accounting for more than 80% of the annual cost of treatment, even in patients without inhibitors (Figure 314).14

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