Emicizumab's Impact on the Hemophilia Treatment Landscape


With particular deference to health care costs and clinical data, a panel of experts considers how emicizumab has affected the treatment landscape of hemophilia.


Peter L. Salgo, MD: First of all, what is the availability of emicizumab now? Did you have it in your pharmacy?

Lacey Chapman, RPh: At first, it was only certain pharmacies, and we were one of the first ones to get it. I don’t know how available it is now. I believe it’s starting to be rolled out, and it’s very common.

Tim Boonstra, RPh: I think you can buy it.

Peter L. Salgo, MD: If it’s commonly available and there’s some stock here, how is this changing the entire landscape of hemophilia therapy?

Robert F. Sidonio Jr, MD: Oh, it’s radical for every person and pharmacist, everybody. It’s changing everything. You’re talking about a drug where you don’t typically have to monitor its effect. It changes the route, so you don’t need as many nurses going out to infuse the patient. They still have to go out for bleeding treatment, but you’re talking about a medication that’s going to significantly reduce bleeding. It definitely is going to change things. As you mentioned, it’s not available at every specialty pharmacy, and they’ve actually put in some restrictions to make sure that who they’re working with is reputable and has experience in this. Some of the mom-and-pop pharmacies may not be around, which isn’t always a big deal to us. It’s important for patient access. We’re not worried about that. But it’s going to change the way we take care of patients. If the patient has an inhibitor, we have a choice to treat it or put them on emicizumab, and we’re looking at whether you can do both at the same time off-label. It just opens up another set of medications that patients have.

Tim Boonstra, RPh: We have 5 patients currently on it, and it has been a game changer for those patients with inhibitors.

Peter L. Salgo, MD: Really?

Robert F. Sidonio Jr, MD: Yes, that’s not an exaggeration.

Lacey Chapman, RPh: It’s part of cost savings as well. It’s dramatically cheaper. Not only is the medicine cheaper, but the nursing as well. It’s subcutaneous, so you don’t need as much nursing care or instruction. That’s a big cost savings as well.

Peter L. Salgo, MD: It occurs to me that when we look at cost, we come to this whole silo issue. “Well, I don’t have to pay for this.”

Robert F. Sidonio Jr, MD: We’re all paying for it.

Peter L. Salgo, MD: But somewhere in the global health care delivery system, we’re all paying for somebody. If you were to lump the meatball, if you will, of health care per year for somebody with hemophilia, without emicizumab, what would that be? What would that number be?

Robert F. Sidonio Jr, MD: You might have a better idea.

Lacey Chapman, RPh: It depends on the patient’s age because of the factor dose per kilogram. Some patients get $100,000 of factor every month.

Peter L. Salgo, MD: Every month?

Robert F. Sidonio Jr, MD: Yes.

Lacey Chapman, RPh: That’s the medication alone.

Peter L. Salgo, MD: That’s $1,200,000. I did that in my head.

Lacey Chapman, RPh: Yes. Hemlibra [emicizumab] is maybe $2,000 a month. I only have 1 patient on it and he’s a child, so he only gets a very small amount.

Robert F. Sidonio Jr, MD: You get a very small vial.

Tim Boonstra, RPh: It’s drastically different between an adult and a pediatric patient. I just did an analysis of a patient who was coming to our center who was not very compliant or adherent, and they’re potentially considering switching him to emicizumab instead of his normal factor VIII therapy. It would have been $30,000 to $35,000, if he was treating appropriately. If we treat him with emicizumab, it’s going to be about $60,000. It’s different.

Peter L. Salgo, MD: It is. If you take everything—if there are fewer bleeds, fewer emergency department visits, and fewer surgeries, and if you can control the bleeding—you were talking 7 figures, but now we’re talking six 6 figures, maybe high 5 figures. There’s a difference.

Robert F. Sidonio Jr, MD: Yes. As you mentioned, it varies. In a young child, they may spend somewhere between $150,000 to $400,000 depending on their weight and their factor consumption for standard factor VIII and IX products. Some of the extended half-life products are more expensive, of course. It’s going to be fairly close, somewhere in the $300,000 range, depending on how much per year. But when you look at inhibitor patients, as we were talking about, the costs are just dramatic. We had patients who were spending $3 [million] or $4 million a year trying to get rid of their inhibitor, and that’s 1 teenage patient. For that patient who I was referring to, we weren’t able to get rid of the inhibitor, so we probably spent $10 [million] or $15 million and we weren’t successful.

Peter L. Salgo, MD: Those are national debt numbers.

Robert F. Sidonio Jr, MD: That’s not sustainable, and we’re seeing this in the community and there’s pressure on those patients. It’s not their fault that they have this, right? It’s a shared cost. For the inhibitor patients, there’s no doubt that there’s a cost savings. It’s not even a contest. It’s more for the non-inhibitor patients where it’s going to be more of a discussion.

Peter L. Salgo, MD: But it sounds like there’s cost saving there too, in all likelihood.

Robert F. Sidonio Jr, MD: Yes, there’s definitely that, but hospital cost savings, exactly.

Peter L. Salgo, MD: For the inhibitor patients, getting rid of the inhibitors is an issue.

Robert F. Sidonio Jr, MD: Yes.

Peter L. Salgo, MD: Right? We don’t care if you’re there anymore, we’ll just give you something that chunks you away.

Robert F. Sidonio Jr, MD: Sure.

Peter L. Salgo, MD: We like this. Now, you mentioned trials. There’s HAVEN 1 and HAVEN 2. What are those trials?

Robert F. Sidonio Jr, MD: The HAVEN 1 trial was looking at this use in adults. In clinical trials, you always start with 18-years-or-older patients. They looked at patients with inhibitors who were 18 years or older, and they wanted to look at the ability to reduce the bleeding rate. There was a dramatic response—it was in the upper 80% range—of reduction of bleeds compared to not doing anything. The great thing about these studies, HAVEN 1, 2, and 3, is they had these noninterventional arms. The biggest criticism about studies is that you’re measuring it differently than I measure at home, when I count bleeds at home. You count it differently on a trial than I measure it when I’m at home by myself.

And so, they took patients as a lead-in, measured their bleeding events the same way in the trial, and what they saw in HAVEN 2 and 3 is that even when people are on factor VIII prophylaxis, there was a reduction in bleeds when they switched to emicizumab. It wasn’t a direct comparison, but it was a comparison afterwards. The median annual bleeding rate for some of these kids was less than 1. These kids were having 1, maybe 0 to 1, bleeding events per year that required treatment.

Peter L. Salgo, MD: That’s dramatic, right?

Tim Boonstra, RPh: Dramatic.

Robert F. Sidonio Jr, MD: Just dramatic.

Peter L. Salgo, MD: Was it a powerful enough study to take these results and expand them and apply them?

Robert F. Sidonio Jr, MD: Yes.

Tim Boonstra, RPh: I think it certainly is powerful. Our center is still philosophizing on how they might use it in some of their noninhibitor patients. It’s a difficult question. How do you know if they’re bleeding or not?

Peter L. Salgo, MD: How do you know?

Tim Boonstra, RPh: Do you just wait and see and say, “Call me tomorrow and see what it looks like,” or do you use ice and compression and elevation and those kinds of nonpharmacological approaches?

Peter L. Salgo, MD: Don’t take 2 aspirin and call me.

Robert F. Sidonio Jr, MD: Yes.

Tim Boonstra, RPh: Exactly.

Robert F. Sidonio Jr, MD: Bleeding events are more of a discussion. Before, if we told them if their joint felt warm or tingly, go ahead and treat it. We’re never going to say you did something wrong, right? Now we’re saying, “Why don’t you wait a little bit, see what happens. If it progresses, then treat it; maybe it’s your arthritis, it might not be a bleeding event.” That’s the challenge in adult patients, certainly. It’s very difficult to discern arthritis between an acute joint bleed. Now we’re having discussions about individual bleeding events.

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