The Economic Burden of Hemophilia B From a Payer Perspective

EP: 1.An Overview of Hemophilia B

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EP: 2.The Economic Burden of Hemophilia B From a Payer Perspective

EP: 3.Defining the Impact of Gene Therapy

EP: 4.Managing Hemophilia B Using Gene Therapy

EP: 5.Predicting the Treatment Evolution of Hemophilia B

Steven Pipe, MD: There have been multiple studies that looked at the economic burden of hemophilia B in the United States for payers. These studies have shown that patients with hemophilia B had significantly higher health care resource use vs match controls. This relates to looking at inpatient admissions, emergency department visits, and outpatient visits. The annual total health care costs per patient among patients with hemophilia B is about 25-fold higher compared with match controls. If we look at annual total healthcare costs per patient in dollars, this increases with severity. For those with a mild disease, this could be in the range of about $80,000–$280,000. If you go up to those with severe disease, this has been determined at over $630,000 per year–$500,000. The findings of these studies highlight the substantial burden of illness that is associated with hemophilia B.

As far as what drives costs in the treatment of hemophilia B, it's the hemophilia-related treatments. There's a European study that is still analogous to what we see in the United States, and this shows that patients averaged at least 2 bleeds per year; nearly half had at least 1 problem joint. The majority reported mild or moderate chronic pain. Mean health-related quality of life measures were low for patients even when they were on factor 9 prophylaxis. You can also see indirect costs related to early retirement or work stoppage because of their illness and reduced work productivity as well, not just for the patients but also caregivers. This has a significant economic impact for parents of children to look after their condition. If we look at the direct costs associated with severe hemophilia B, this is the most substantial and is driven almost entirely by factor 9 treatment. We could also see costs related to pain management, as well as joint assessments, including the need for detailed imaging, such as MRI, and then any joint interventions to deal with chronic pain or improve function in disease joints.

The outcome from regular factor replacement therapy is that you give yourself IV infusion, and you get a peak level right after that infusion. Those levels fall precipitously over the subsequent days after that infusion. How long it takes to reach the trough levels is determined by the pharmacokinetics of the product and individual factors in the patient. We've had some innovations with the recombinant factor 9 products. We now tend to rely on what is called extended half-life factor 9 replacement therapy. These have some advantages because the majority can get by with once-a-week IV infusions. Some people can stretch that out longer. The net effect, however, is still a peak, and a trough can fall all the way down to single digits before their next infusion. It's when they get down to those lower levels where they remain at risk for breakthrough bleeding. They're going to need additional episodic infusions on top of that. When we're communicating with payers, we want them to understand that, yes, we're ordering prophylactic therapy. We're anticipating that they're only going to be dosing once a week. This is a weight-based dosing regimen. However, because of unexpected traumatic injuries, or even breakthrough spontaneous bleeding, they're still going to need episodic infusions on top of that. If they're on good prophylactic therapy as standard of care, we anticipate breakthrough bleeding to be at a rate of between 2–6 times per year. That could be higher if the patient maintains an active lifestyle. We see breakthrough bleeding in the pediatric age group related to accidents and playing, [as well in older adolescents and young adults].

Transcript edited for clarity.