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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
     Treatment-related costs are often the focus of atten-tion because of their significant burden. However, inpatient hospitalization for hemophilia care can also have a significant financial impact. Average inpatient costs for patients with hemophilia A are more than 9 times greater than for the average insured member without hemophilia (Table 225). Average annual non-drug claims for non-hemophilia patients were $1065 for inpatient services and $2394 for outpatient services. Comparatively, claims for patients with hemophilia A were $9661 and $7433, respectively. Similar results were seen for patients covered by Medicaid.25

The Hemophilia Utilization Group Study, an analysis examining the annual utilization and cost of hemophilia-related healthcare services, found that patient clinical characteristics and clinician practices predominantly drove the costs of care in hemophilia and that patients with severe arthropathy had greater healthcare costs.26 Treatment-related costs vary substantially based on disease-related complications, such as the presence of inhibitors or HIV or HCV.22,23 Costs also differ when treatment is given on an on-demand basis versus as prophylaxis.14

Cost of Complications

     Costs rise substantially for patients with complications, such as HIV seropositivity, arthropathy, and/or development of inhibitors.21-23 Some estimates claim treatment costs as high as $1 million per year for these patients.21 The majority of these treatment costs are attributed to the cost of clotting-factor concentrates.14 Coinfection with HIV and HCV is associated with significantly greater component costs for clotting-factor concentrates, prescription drugs, and inpatient and outpatient services. Coinfection results in a 59% increase in total annual medical costs (95% CI, 34.8%-82.9%).14 Within the employer-sponsored insurance population in the study, about one-third were infected with HIV or HCV, resulting in 1.5-times higher mean expenditures than with adults with hemophilia without blood-borne viral infections.22 The treatment costs for patients with blood-borne viral infections in the Medicaid population were the same as in the general hemophilia population, but the median was 1.6 times higher.23

Costs increase even more when patients develop inhibitors, which affect 25% to 30% of patients with severe hemophilia A and 1% to 5% with hemophilia B.27 Although treatments (high-dose clotting-factor concentrates, bypassing agents, immune-tolerance induction therapy) do exist for patients with inhibitors, the economic burden associated with the care can be staggering. Patients with hemophilia who develop inhibitors are twice as likely to be hospitalized for a bleeding complication, and treatment costs associated with inhibitors can be 5 times greater than for those without inhibitors.22,23,28

Among patients with employer-sponsored insurance in the study, adults with an inhibitor were more likely to use clotting-factor concentrates (100% vs 74%; P <.01), more likely to visit the ED (63% vs 28%, P <.01), and more likely to require inpatient services (31% vs 12%, P <.05) than those without an inhibitor. Overall, the costs were 5 times higher for adults with an inhibitor than for those without (mean cost: $697,000 vs $144,000, respectively; median costs: $330,835 vs $73,321, respectively).22 Within the Medicaid population, the average costs for males with hemophilia A and an inhibitor were 3.6 times higher than those for individuals without an inhibitor. The majority of expenditures were attributable to clotting-factor concentrates, with or without bypassing agents. Clotting-factor concentrates accounted for 64% of total mean costs for patients with an inhibitor ($287,245) compared with 86% for those without an inhibitor ($106,807).23

Optimizing Outcomes

     Treatment of hemophilia accounts for 80% to more than 85% of the overall cost of treatment. Study results have shown that clotting-factor concentrates make up 86% to 92% of total direct costs in patients without inhibitors.23,29 The high cost of hemophilia poses a definite barrier to patient access. A 2011 survey administered to patients/caregivers and healthcare professionals (HCPs) found that 54% of patients/caregivers and 81% of HCPs felt the economic downturn negatively impacted hemophilia care.30 Most patients made treatment decisions, including modifying treatment and skipping doses and clinical visits, based on their financial situation. HCPs also made decisions based on financial restraints, includ-ing delaying elective surgeries, delaying initiation of prophylaxis, and switching to lower-priced treatment options.

     Fortunately, healthcare reforms have helped change this trend. Twenty-two percent of patients and 58% of HCPs indicated that they expect to initiate treatment decisions they had previously delayed because of the greater affordability resulting from the elimination of lifetime caps. This turn in healthcare reform is perceived as positive for the treatment of hemophilia and may lead to more optimal treatment behaviors.30 Looking at long-term goals for hemophilia is also critical to ensuring long-term cost-effectiveness over short-term cost savings.

     Prophylaxis is recognized as the standard of care for the treatment of patients with severe hemophilia. The World Federation of Hemophilia recommends starting primary prophylaxis before the second clinically evident large joint bleed, and before 3 years of age, to prevent future bleeding episodes and the resulting complications.1 Recommendations such as these, along with vast clinical trial data demonstrating the long-term effective-ness and superiority of prophylaxis over on-demand use, have shifted the debate from whether to use prophylaxis to optimize strategies for cost-effective use.31-33 Although this may result in increased costs in the short term, a vast body of clinical trial data has demonstrated the long-term effectiveness of prophylaxis over on-demand treatment in patients with hemophilia—preventing con-ditions leading to severe or permanent impairment, improving clinical outcomes (preventing bleeding epi-sodes, minimizing articular damage), and reducing long-term total costs compared with on-demand treatment.16

Cost-Effectiveness of Prophylaxis

     Data from short-term analyses of 1 year of healthcare utilization and 2 years of clotting-factor concentrates dis-pensing records of patients with hemophilia A in the United States showed that the use of prophylaxis in patients with severe hemophilia significantly reduced the numbers of ED visits and bleeding episodes compared with on-demand treatment.29 The cost of treatment, as expected, was higher for prophylaxis use. The mean direct medical costs for patients with severe hemophilia were $184,518 for those receiving on-demand treatment and $292,525 for those receiving prophylactic treatment (P = .009). Clotting factor accounted for 92% of these costs ($170,037 and $289,172, respectively). However, other direct costs were lower for patients receiving prophylaxis ($3353) than the potential reduction in long-term repercussions of the disease, namely, hemophilic arthropathy.16 These results are only from a short-term perspective (1 to 2 years).29

Prophylaxis has also been shown to be beneficial in decreasing potential long-term disability and adverse outcomes. In a 6-year longitudinal uncontrolled study, the bleeding episodes, X-ray exams, and treatment regimens of patients younger than 21 years with severe hemophilia A were analyzed.34 The results showed that high doses of FVIII did not necessarily result in improved orthopedic outcomes. However, full-time prophylaxis significantly reduced the rate at which joints deteriorated, both on physical (P = .02) and X-ray examination (P ≤.001). Patients on prophylaxis also had significantly fewer days lost from work or school, as well as fewer days spent in a hospital (P ≤.01).

    Cost-effectiveness of prophylaxis is also an important issue in managed care practice. The use of primary prophylaxis with FVIII concentrate versus secondary prophylaxis, treatment on demand, and a “hybrid” form of therapy (primary prophylaxis followed by on-demand treatment) was evaluated in patients with severe hemophilia A in a study by Colombo and colleagues.35 Performed with a Markov model using different sources of clinical and cost-utility data, the results demonstrated that the incremental costs per quality-adjusted life years (QALY) gained for patients receiving primary and secondary prophylaxis were substantial (€40,229 to €40,236) versus on-demand treatment. Another study assessing cost-effectiveness of primary prophylaxis in the United Kingdom found that patients on prophylactic treatment could expect 55.9 QALYs, compared with 41.1 QALYs for patients with on-demand treatment. However, the associated costs were sensitive to the type of hemophilia, amount of factor used, and the price per unit of the factor.36

Optimizing Prophylaxis—Individualized Care

     The rationale for prophylaxis is based on studies demonstrating that patients with moderate hemophilia (clotting-factor activity level >1 IU/dL) rarely experienced episodes of spontaneous bleeding. These patients also had better preservation of joint function.1 This observation led to the theory that artificial elevation of plasma levels of specific factors could change the phenotype of a severe patient to that of a moderate patient. However, a variety of aspects influence bleeding patterns. These include the pharmacokinetic profile of the patient, musculoskeletal status of the underlying joint at the time prophylaxis is initiated, potency of the dose and the factor product, and the relationships of these aspects to each other.16,37 As such, it is important that treatment be individualized for each patient. Higher doses of factor-replacement product may not harm the patient, but when lower doses are optimal, excess doses will result in unnecessary costs.     

     The presence of treatment-induced inhibitors are significant variables in cost-utility models, and ensuring optimal pharmacokinetic dosing is the most valuable tool for cost-effective therapy.38 Assay management by way of tighter pharmacy standards is one tool that can enable cost-effective factor prescribing. Utilization management programs that encourage frequent HCP–patient interaction can also help. These programs ensure that prophylactic treatments are reassessed and adjusted based on each patient’s individual needs and circumstances. The frequent interaction also enables greater patient education regarding the need for and importance of treatment adherence.16

Optimizing Care

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