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Balancing Aspects That Influence Hemophilia A Treatment Decisions


Diverse factors can influence treatment decisions for patients with hemophilia A, such as the evolving landscape of insurance coverage, challenges related to vein access and medication storage, and mode of administration.

Kimberly Epps, PharmD, CSP: In my experience with patients with hemophilia A, it varies whether they prefer one mode of treatment to another. It’s been 5 or 6 years since they’ve even had an option for mode of administration. Now that we have a subcutaneous form of treatment, the majority of the products are IV [intravenous]. It depends on where the patient is in their life, what transitions of care they have, whether they’re going to college and if they’re younger. Of course, the parents prefer a subcutaneous mode of administration. It’s a lot easier to give to a young child, who’s a little squirmier, a subcutaneous injection over IV injection because this is a continuous treatment; it’s lifelong. Most patients who are severe and quite a few who are moderate will have to be treated at least weekly for the rest of their lives.

I haven’t found many access issues due to preferential coverage by payers. We’re just now seeing a few payers dip their toes into excluding some of the options that patients with hemophilia have. In general, patients with hemophilia have had a lot of control over their treatment and the treatment options, and we’re seeing payers start to narrow that down. In the near future, you could see payers exclude options like emicizumab, which are subcutaneous. As those patients get older, patients who’ve accessed their veins two or three to seven times a week for the entirety of their lives are going to experience scarring and some vein access issues.

Most of the approved agents are refrigerated medications. Even for the ones that are OK at room temperature, most pharmacies continue to use cold chain and allow the patient to make that room temperature vs refrigerated decision. Part of our counseling as a pharmacy is to let the patient know the expiration date at room temperature vs refrigeration. That’s something we do cover with the patients.

For a lot of patients with hemophilia A, it’s a little less common to have as many storage issues because there are a lot more options. But for some other bleeding disorders and some patients who are on unusual dosages of hemophilia A products, they might experience storage issues depending on the size of their family. They’ve got to put [these larger boxes] into a refrigerator. They may not have space. These boxes are kits in most instances, so they have a diluent and the active drug. Some have a syringe and all the supplies they would need to infuse. They aren’t small boxes. If you need more than one box per dose, storage would become a problem with larger boxes. So yes, storage becomes an issue. As a specialty pharmacy, we’re required to manage that. If a patient has storage issues, we have refrigeration options that we can help the patient with, providing extra dorm-size refrigerators to make sure they can store their products at the refrigerated temperature they prefer.

There are many challenges with infusions. As a layperson, if you think about going to the doctor, having your blood drawn or having to get an infusion yourself, there’s a trained nurse on the other end of that infusion who knows how to find your vein. You don’t have to worry about that thought process going through your head sticking your own veins. There’s a whole mental challenge to overcome the thought of, “I’ve got to stick a needle into my vein. I’ve got to cause myself pain.” That’s not a natural thing to do. Ultimately, when you’re requiring someone to infuse two or three to seven times a week, they’re going to have to learn to infuse themselves because it’s not an option for a nurse to come into your home every day and infuse your medication. For most payers, these medications are considered self-infused for that very reason. They expect the patients to infuse themselves or have a caregiver who can infuse them, or they expect them to go into the HTC [hemophilia treatment center] to get infused. That creates a lot of challenges, especially with younger children. The sooner you can start training them to infuse themselves, the easier that transition is. There are options such as ports and PICCs [peripherally inserted central catheters], but those options come with their own risk for infection. There’s a lot to consider in that infusion realm, and a lot of issues come up.

Transcript edited for clarity.

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