Advancing Hemophilia Care: The Evolving Role of Specialty Pharmacists


A panel of experts explore the evolving role of specialty pharmacists in hemophilia care, including gene therapy, adherence strategies, and the transformative impact on patients' lives.

Jonathan Ogurchak, PharmD, CSP: As we see more and more use of gene therapy in hemophilia A, what do you feel like the role of the specialty pharmacist may be when it comes to not only assisting with product selection, product choice, approvals, or even just monitoring once they're on therapies?

Atta Chowdhry, RPh: Unfortunately, there is no defined role of a pharmacist by FDA or CDC at this point in gene therapy. I know that pharmacists have an important role in delivery of the product, patient monitoring, of the wild air on the product, and then even getting the patient education part of the thing. But at this point, I think in gene therapy, the pharmacist's role has not been well defined. It'll be interesting how the treatment centers are, even with other chronic disease states, I think it's interesting that how the pharmacist's role will evolve and get in that process, it'll be interesting to see.

Robert Sidonio Jr, MD, MSc: I think the key thing on this is that, you want to get these, like I said before, a number of patients will have preexisting antibodies that will not make it effective and that will eliminate them from consideration. It makes sense to just get them tested when they come in. And then there's never a rush on this, you're going to test them, you might get the result back in a week or so. Then you have some time to plan this out and make sure it aligns with their lifestyle. Because once you do the infusion, they have to really take it easy. One of the things they have to do is they have to delay family planning. This isn't the time to have a child during this period of time. We certainly think reasonably afterwards they're safe, but I think for just trying to be extra safe, we have this long buffer period. Because things like drinking alcohol can raise your liver enzymes by itself, we want to be a little bit careful of this, so we've asked patients to not drink any alcohol during at least the first 6 months and maybe potentially the 12 months. Some other groups have sort of said longer periods of time, I don't think that's very practical, but I think it's reasonable to sort of, these are things they have to understand and they're going to have to get lab surveillance afterwards. The companies are offering services where they can go to a local LabCorp, for example, maybe it's near the mall, near your house. These are things they'll have to do. There's a little bit of a trade-off, but the potential benefit could be tremendous, like we talked about over 90% reduction infusions. They could go years without needing an infusion if they don't have any surgeries. For some families, that's just incredible for them. It's exciting. We know that it is being started. We're hopeful that a lot more patients will be receiving this in the upcoming year.

Jonathan Ogurchak, PharmD, CSP: Now, we've covered a lot of topics here over the course of our time together today. From therapies to data to specialty pharmacies, I'd just like to get some closing thoughts from each 1 of you before we wrap up.

Atta Chowdhry, RPh: It's an exciting time in hemophilia community as a parent. I hear from the older gentlemen. I joke around 1 of my good friends who has hemophilia. And I think his numbers, T cell numbers were pretty good. Then it got a little bit lower that he had to get on the pill. He was 45 and he calls me up and says, I'm going to be getting on a pill. He was pretty depressed. And I said, how's your labor, that I talked about in general about those. Then I said, how's your cholesterol level? How's your blood pressure? Everything else was fine and he's working out. I said, “Hey, at this point, I have no sympathy for you because you already take in 1 pill only. I don't want to hear from you because everybody who's 45 plus is taking a pill for some reason.” I think that it changed the community so much. I think with all these new therapies, and I think it had to do with socioeconomic background too. If you're, of good economic background and very little educated in that sense that you are maintaining your disease state in a really easy way. At that point, I think you can protect your joints. You can protect your health overall. I think that's the biggest change that I've seen in the last 20, 25 years in this disease state.

Jonathan Ogurchak, PharmD, CSP: Dr Schrade, how about you? Some closing thoughts? –

Lisa Schrade, PharmD: I think there's so many advances that are going on in a very short amount of time. It's our responsibility as healthcare providers to stay well informed about the products that are available because we are the resources that patients go to get the information. It definitely is our responsibility to make sure that we know the side effects, possible coverage for the insurance plans. Again, just encouraging the patients to continue to ask questions. Don't feel like you have to be guarded because understanding this community of individuals has been very guarded with their information in healthcare for such a long period of time that ultimately our goal is to make sure that they have better health outcomes. We just want to make sure that they're successful and lead healthier, happier lives.

Jonathan Ogurchak, PharmD, CSP: Perfect. Dr Sidonio, how about you?

Robert Sidonio Jr, MD, MSc: If you just look back like the 1960s, for example, most of the patients would barely make it to adulthood without dying of some bleed complication. Fast forward to nowadays. When they've looked at this, I mean, patients live about the same length of life. Obviously, there's still patients suffering from HIV and hepatitis. But I mean, there are so many good options now. Just in the last 15 years, just been an explosion of therapies. You see all the innovations that are happening in the pharmacy world and patients with hemophilia are benefiting from this. It really is a golden age of therapy, family. Hopefully, the community and people that work within the community understand that choice is still really important to this patient population. They like having all these options. Hopefully this will still be possible in the future. I think patients, when they have a child with hemophilia, they can look at this and say, "This is a manageable condition." We can really eliminate most of the long-term side effects if it's managed well. I think there's a lot of hope for these patients. Obviously, we really need to do a better job of making sure we're ensuring the mental health of some of our older patients. I think that's something we still need to do a better job of, but it's important, like you were saying, that they really look to their peers, continue to interact with each other. During the pandemic times, you could see some of this happening where they weren't going to meetings. Those are really important for those gatherings. These patients get together and reminisce and look at the hope, and they see the young patients and see how well they're doing, so I think it's really helpful for them.

Jonathan Ogurchak, PharmD, CSP: Well, I'd like to have a special thank you to our panelists here today for sharing all of your insight, and a thank you to our viewing audience at home. I really hope that you found this pharmacy times practice pearl session to be very, very informative.

Transcript is AI-generated and reviewed by an AJMC editor.

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