$599.00
A US-based key opinion leader (KOL) provides insights into prescribing habits, key marketed drugs, and late-phase pipeline therapies for hemophilia, including alternative coagulation promoters and gene therapies. Unmet needs are also discussed. The main marketed products covered include Hemlibra, Advate, and BeneFIX, and key pipeline assets highlighted are valoctocogene roxaparvovec (valrox), fitusiran, and concizumab.
Highlights
The advantages, it’s recombinant number one, and with Advate, it has a good mixing system, it’s fairly easy to mix the factor with powder, or mix the factor, the liquid and the powder, and it has a good system for giving the injection. And I think the main thing is that people are comfortable with the company that produced it and the brand, and that’s where the advantage was. The disadvantage is that Advate has to be given 3–4 times a week, and even that it’s just to maintain the trough level of 1%, which is really no longer what we’re satisfied with. So, the main disadvantage of Advate is that it must be given intravenously and multiple times a week, so it imparts a very large treatment burden on patients and
families of children who are using Advate.
…so, that’s how we kind of make the choices categorically. Now, within the categories, honestly in my center we try and share the wealth, because I really don’t think there’s any advantage to any specific brand of half-life factor VIII versus any other specific brand. I know the companies who are marketing, and with marketing materials, they’ll say ours has this, and ours has that, but really at the end of the day what matters is efficacy, and safety, and they’re all equally efficacious, equally safe, and they have basically the same dosing interval within the categories…
Well, I think there is going to be a significant increase of the use of Hemlibra, because there are still countries where it’s just getting licensed. In Europe for example, it’s only available for severe hemophilia, I think that eventually will change. So, the main thing that is going to drive the growth right now still is actually getting it licensed, approved and available in many parts of the world that don’t have for example the availability or the access that we have in the United States… The second big thing is the education part, there are a lot of people who are fearful, it’s something new, they don’t really understand it, there are a lot of physicians who don’t go to conferences all the time, and they’re not keeping up with the latest literature, and don’t have experience with Hemlibra. So, I think there is still a large education gap that needs to be overcome to get prescribers comfortable with Hemlibra, and that’s an issue in the United States… So, across the US you have this large pool of centers that haven’t put hardly any patients without inhibitor on Hemlibra, and other big centers that have put a lot of patients on Hemlibra. So, I think there is an education gap that needs to be overcome as well.
A US-based key opinion leader (KOL) provides insights into prescribing habits, key marketed drugs, and late-phase pipeline therapies for hemophilia, including alternative coagulation promoters and gene therapies. Unmet needs are also discussed. The main marketed products covered include Hemlibra, Advate, and BeneFIX, and key pipeline assets highlighted are valoctocogene roxaparvovec (valrox), fitusiran, and concizumab.
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