Opinion
Video
Factors Impacting Hemophilia A Care Costs
Russell P. Gollard, MD, FACP, discusses how the rising costs of managing hemophilia A are influenced by drug expenses, treatment efficacy, and adverse events, creating significant financial burdens across health care settings and patient populations.
Russell P. Gollard, MD, FACP: There are a number of different things with emicizumab or on-demand treatment that contribute to the total cost of care. The first thing is the cost of the drugs themselves. These drugs are extraordinarily expensive because they are cloned products. In general, when drugs are developed for rare diseases and they’re not used very often, they are extremely expensive. That’s the first thing. The second thing, when you look at the total cost of care, you look at efficacy. If, in fact, emicizumab is keeping somebody out of the emergency room and you’re not having the development of hemarthroses or severe bleeds, then you’re decreasing the total cost of care, at least theoretically, by keeping somebody out of the acute care hospital and also contributing to their overall quality of life.
The cost of care can differ in different health care settings depending on how the drug is obtained, whether it be through a specialty pharmacy where we can negotiate for prices with the manufacturer. That’s why it’s very important for us as a group with a lot of providers to be able to forecast what our needs may be and then agree by committee on what sort of drug we may be giving and how much of what we may need to give, because that will decrease our overall cost of care. We have the power to do that through looking at things with the lens of a large purchasing agent involved.
The cost of managing administration and related AEs, that is adverse events, and the like in patients with severe hemophilia A can be extremely substantial. On the one hand, many patients have been trained to self-administer some of the prophylactic treatments and they’ve become very, very good at that. On the other hand, when things do go awry, when patients travel or don’t have access to medications, or in some of the younger patients, when perhaps they forget about the severity of their disease, the costs of care can be extremely substantial. These costs can be related to surgeries, such as in joint replacements for people that develop hemarthrosis or bleeds into their hip or elbows. We also occasionally see people who develop subdural hematomas, which require the neurosurgeon to intervene. Any place somebody can get a bleed, including even into the eye, are places where surgeons oftentimes have to be involved and where the intervention does not bring somebody back to the point they were before the adverse event. For that reason, we really want to be able to prophylax people appropriately and not have these adverse events occurring. It’s extremely key.
Transcript is AI generated and reviewed by an AJMC editor.
