Economic Costs of Hemophilia, Large and Lifelong, Detailed in Report

In addition to its clinical challenges, hemophilia is characterized by a major economic burden that affects patients, care givers, the healthcare system and society. In a recent report, Sheh-Li Chen, PharmD, from the University of North Carolina Medical Center, identifies the most significant areas of healthcare hemophilia management, highlighting the importance of the understanding the distribution of economic resources, and how education can improve patient adherence to treatment.

The report, titled “Economic Costs of Hemophilia and the Impact of Prophylactic Treatment on Patient Management,” was published in The American Journal of Managed Care.

Over the last two decades, the clinical care of patients with hemophilia has improved considerably, with strides in acute and prophylactic therapies, as well as in the availability of safe and effective clotting-factor concentrates. Such advances have resulted in greater life expectancy for this patient population, although men with severe disease are still likely to die 15 years earlier than the general population. Moreover, resistance to treatment and the joint atrophy that develops due to recurrent bleeding episodes have a significant negative impact on the patients’ quality of life (QoL), which, along with potentially disability, pain and ineffective disease management translate into a heavy burden for patients, caregivers and others.

Although hemophilia only affects a small portion of the American population, it has a significant economic impact, as the disease is a chronic condition that requires lifelong treatment, the study reported. Such costs can be divided into two categories, indirect and direct costs.

Indirect costs refer to the toll the disease may have on individual and caregiver productivity due to absenteeism from work or school. Studies have found that the increased number of hemorrhages and on-demand treatment negatively associate with physical ability scores. According to an Hemophilia Experiences, Results and Opportunities (HERO) initiative study, 80 percent of patients with hemophilia reported that their disease negatively affected their employment to some degree. In the same study, 63 percent of parents with hemophiliac children reported their child’s condition negatively impacted their ability to work. According to the initiative, the cost of underemployment due to hemophilia was estimated at almost $4 million per year. 

Direct costs consist of those incurred through hospitalizations, outpatient visits, and drug treatments. According to 2010 Medicare spending records, treatments for hemophilia carry the most costly drug average per beneficiary, largely due to a lifelong need for factor-replacement therapy, either as prophylaxis or as acute (on-demand) therapy. According to the report, “factor replacement therapy makes up over 80% of the total direct expenditures for patients with hemophilia A and B.”

A 2011 survey found that  54 percent of patients or caregivers and 81 percent of healthcare professionals felt the recent U.S. economic downturn negatively impacted hemophilia care, weighing on treatment decisions by patients and caregivers. Healthcare reforms have, however, did help to alleviate this situation, namely by eliminating lifetime caps on insurance coverage for essential health benefits.

The quality and cost-effectiveness of hemophilia care are heavily influenced by the optimization of pharmacy management, and patient involvement and education about their disease and treatment plan. “Cooperation among the key stakeholders — healthcare professionals, patients and their caregivers, and managed care professionals — will help provide individualized treatment strategies for patients with hemophilia, strategies designed to prevent complications and optimize clinical and economic outcomes while enhancing patient [quality of life],” the author concluded.