The Challenges and Treatment of Sickle Cell Disease - Episode 14
Neil B. Minkoff, MD: I’m going to push you guys a little further. The first product you mentioned, voxelotor, was in the HOPE trial. When I asked you whom you’d like to see it in, you basically said as many people as possible, right?
Ahmar U. Zaidi, MD: Absolutely.
Neil B. Minkoff, MD: Yet we talked about the good results that can be achieved by what’s out there if we get the compliance. I’m asking my payers, do we try to push compliance before we open up access to new medication? Does this become a prior authorization process, a step process? How do you look at that when there’s an older effective generic on the market?
Maria Lopes, MD, MS: Well, I also heard that the study was done on top of hydroxyurea.
Ahmar U. Zaidi, MD: That’s right.
Maria Lopes, MD, MS: I don’t think we can just forget hydroxyurea and its benefits, again based on the clinical trial and a center of excellence that is comfortable with the use of hydroxyurea. I would maximize that potential, and then of course add to that if there are intolerance issues. Then we have to think about what the potential is for this drug as monotherapy, but optimally you’d want a patient on hydroxyurea.
John C. Stancil, RPh: And we recognize that these are not replacements to any current therapy. They’re probably going to be an add-on or a combination. But the exciting thing is that it treats the disease upstream as opposed to just being reactive and treating symptoms or manifestations of the progression of the disease. Now we have a chance to actually alter the course of that disease progression.
Neil B. Minkoff, MD: That was the answer I was looking for, but it brings up the follow-up question. And this is something we face in other disease states, right? You have drug A, and drug B is an add-on to drug A, and a lot of people stop taking drug A. Do you continue to go to drug B, or if it’s combination therapy, do you cover only it in combination?
John C. Stancil, RPh: We would cover both. Because that will become the treatment guideline. And so it wouldn’t make sense for the payer to say, “No, we’re going to cover only 1 or the other.”
Neil B. Minkoff, MD: What I’m saying is that if the patient had to be on hydroxyurea to get to voxelotor, would you cover them only if they were used in combination?
John C. Stancil, RPh: I think the clinical trial showed patients who were on hydroxyurea, but I think it also included if maybe there were patients also who were not on hydroxyurea and they had similar significant impact. And not every patient is able to tolerate it. Not every patient responds to hydroxyurea, but we would cover both of them.
Neil B. Minkoff, MD: So you would have to do it as you did before.
John C. Stancil, RPh: Yes.
Maria Lopes, MD, MS: I think we’d probably have language there that would suggest that you have been or are intolerant to hydroxyurea. Again, in line with guidelines or the standard of care, we would certainly make sure that the prior authorization was in line with the thought leader specialty in terms of their approach, but there may be some language in there that gives an out yet reinforces the option of hydroxyurea.
Neil B. Minkoff, MD: And realizing that we don’t have all the data yet, if you’re talking about a medication that’s there as pain prophylaxis, what’s the population for that? And is it something that can be reserved for patients with more severe chronic pain or patients who are going through a crisis? Where could you draw a parameter?
Ahmar U. Zaidi, MD: As far as that drug goes—the crizanlizumab drug, that drug that we’re discussing—for now it definitely is a pain medication. The phase 3 trial is going to look at multiple effects that it may have beyond pain. We’ll see if the indication broadens in the phase 3 setting. I do think you would reserve crizanlizumab for patients who are frequent fliers, so patients who are starting to have pain episodes despite hydroxyurea or patients who are really struggling with staying out of the hospital, having a very tremendous decline in their quality of life. I think each clinic would have to make that decision on a patient level.
Neil B. Minkoff, MD: Is that something you would put a prior authorization or criteria on?
John C. Stancil, RPh: That is possible, but I think it’s a great opportunity for an outcomes-based approach. If they can reduce the number of pain episodes that result in a hospitalization or ED emergency department visits, then we would probably entertain that discussion with the manufacturer.
Neil B. Minkoff, MD: Maria?
Maria Lopes, MD, MS: I think if it’s started in the hospital and it’s allowed to then be continued, that’s usually an easy prior authorization to fulfill. It becomes more of a transition in care opportunity. Whereas for the patient after the hospital going to receive the infusions, how can we connect with the patient? Because again, it is IV [intravenous], so the good news is that we know if it was given or not. The challenge may still be the compliance to be able to get to that site to be able to ensure compliance.
John C. Stancil, RPh: Another thing we have to understand is that what’s driving my medical cost is that ED presentation or hospitalization because of pain crisis. And so I think the patient community, as well as the provider community, is going to be interested in that because of the pain.
Neil B. Minkoff, MD: Maria brought up a term earlier that has become pervasive in the industry in terms of what we’re looking for, and she said disease modifying. I realize that not all the data are in, but just from what we know in the pipeline, from your clinical perspective, do you suspect any of these could actually be disease modifying?
Ahmar U. Zaidi, MD: I do. I absolutely do. I’m very excited as a provider for sickle cell patients. Again, in a field in which we really have not had anything to offer, suddenly we’re going to have all these amazing options. I feel that each medication that we discussed, particularly the first 2—voxelotor and crizanlizumab—are really going to be game changers. I really do feel strongly about that. And I think they will truly make a mark on the footprint that sickle cell currently has in the United States. I have a lot of excitement at this point in time for both agents.
Neil B. Minkoff, MD: And that opens up the channels for you, correct?
Maria Lopes, MD, MS: Oh, absolutely, yes. Indeed, isn’t that our goal, to reduce crises and reduce hospitalizations and ER [emergency department] visits?