Cost and access to treatment products
The biggest barrier to long-term prophylaxis is the cost of treatment. Prophylaxis is only possible if significant resources are allocated to hemophilia care. However, it is cost-effective in the long-term because it eliminates the high cost associated with subsequent management of damaged joints and improves quality of life.
It is very important to collect and provide scientific evidence that justifies the high cost of treatment, even in countries that have traditionally invested significantly in hemophilia treatment and where prophylaxis is well-established as a standard of care.
In countries where access to clotting factor concentrates is limited, prophylaxis is not possible for most patients. However, some countries are now starting low-dose prophylaxis in children. Follow-up data of these patients will be very important in confirming the effects on their joints. Patients and healthcare providers must work together to advocate for a national hemophilia care program and the purchase of sufficient clotting factor concentrates.
Adjustments to the prophylaxis protocol, such as administering lower doses of factor concentrates more frequently, may also be an effective option. Cost-efficacy studies designed to identify minimum dosage are necessary to allow access to prophylaxis across the world.
Prophylaxis requires frequent injections and it can be difficult to find suitable veins in very young children with hemophilia.
An implanted venous access device (e.g., Port-A-Cath) can make injections much easier and may be required for administering prophylaxis in young children. However, there are risks involved with the use of these devices, including the risk of local infection and the formation of blood clots that may cause blockage. These risks need to be weighed against the advantages of starting intensive prophylaxis early. In 70% of children early prophylaxis is possible without venous access devices. An important option is starting prophylaxis once a week as it does not require the implantation of a Port-A-Cath which makes the treatment easier for patients and families to accept.
Adherence to (or compliance with) a treatment plan is generally defined as the extent to which patients take medications as prescribed by their healthcare providers. According to the World Health Organization, rates of non-adherence with any medication treatment may vary from 15% to 93%, with an average estimated rate of 50%.
Adherence to a prophylaxis protocol is critical to its success. Prophylaxis is most effective if factor levels are continuously maintained above the target level. Missing or skipping a dose can cause clotting factor levels to fall below this target, which increases the risk of bleeding. Bleeding that occurs while a patient is on prophylaxis is called “breakthrough bleeding.”
Patients and healthcare providers must work together to ensure that the protocol is manageable for the person with hemophilia and their family. A patient’s adherence to the protocol should be assessed regularly during clinic visits and strategies to improve adherence, including changes to the protocol, should be explored wherever possible.