Sickle Cell Disease: Cost and Access to Therapy in an Evolving Paradigm

Neil B. Minkoff, MD: Traditionally, payers hear about a rich pipeline like this that involves a bunch of new compounds or a bunch of new molecules—especially some that are biologics, some that may look like they’re about to lead to more combination therapy—and have somewhat of a reaction to that. But we’ve also been talking about the unmet needs here. I’d like to get the 2 of you to weigh in on what you just heard about the pipeline, how you’d be thinking about integrating those into your treatment guidelines, where they’d fit maybe between pharmacy and medical benefit, or whatever you think is appropriate.

John C. Stancil, RPh: Certainly infusions are probably going to be covered more over on the medical benefit, physician-administered drug program. But we’re going to look to partner with providers or the centers of excellence to establish those treatment guidelines and then make sure that we ensure access. One of the other things, and this fits to Maria’s point earlier, is that there are these new emerging therapies, and we’re exploring outcomes-based approaches. If they’re promising a certain result, we’re looking to engage the manufacturer or contract with the manufacturer for those end points. And if they don’t achieve them, then we don’t pay the whole price; we get some type of a rebate back. Certainly seeing this is encouraging those approaches.

Maria Lopes, MD, MS: I think it’s exciting. It’s an exciting time when we had such few options to work with, to see potentially the spectrum of additional treatments that may come to market, including the opportunity even for a cure. I completely agree that with gene therapy, there’s great promise. But there’s also the need with very new treatments to ensure the durability of the effect and that, indeed, as we sink extraordinary costs, we are achieving the outcomes.

Neil B. Minkoff, MD: Let’s hold that thought and come back to gene therapy for a second. Let me ask you, when you’re looking at, for example, the first 2 products we discussed, and 1 of the outcomes is pain, do you as a payer view that differently from other outcomes?

Maria Lopes, MD, MS: Yes. I think with the aspect of disease modification, you can really change the course of the disease, the complications that ensue versus just symptomatic management, and there’s also the setting of care. I heard that with 1 of these options, it may be more on the hospital side where, at the time that you’re already inpatient, you have a vaso-occlusive crisis. As a payer, you may not get into the management of what happens inpatient until discharge occurs. If it’s a chronic therapy, then we typically get involved. I think also in looking at them, some are IV [intravenous], and some are oral. Obviously, the oral is pharmacy benefit, and the IV is medical benefit. But I think there’s more and more of a concerted effort to manage both medical and pharmacy as we look at what the options are and how we sequence them. What do we bring together in terms of potential combinations that have different mechanisms of action to really come back to the personalization aspect that adds value to the outcomes?

Neil B. Minkoff, MD: Let’s say, for the sake of argument, the biologic wasn’t infused. It was self-injected, but it’s pain based instead of other forms of outcome based. Do you have a similar response?

John C. Stancil, RPh: Well, for several drugs that are available as an infusion or an injection, we actually have covered those in both pharmacy and the medical benefit. Because there are some smaller providers, or providers who don’t want to stock or buy and bill that drug, we allow it to be provided from a specialty pharmacy or a pharmacy to be sent to the treatment center to be infused or injected that way.

Neil B. Minkoff, MD: Obviously we don’t know cost of any of this, right? They’re all pipeline drugs. But based on what you’ve heard, do you think specialty pharmacy is a reasonable option in terms of distribution?

Maria Lopes, MD, MS: I do. I think 1 of the aspects we’re also looking at, in terms of controlling medical spending, is the site of care. Where can the same agent be infused that hopefully minimizes and maybe even improves compliance if it’s convenient for the patient? In terms of where they reside, where they can access this, including the home I would say. But yes, specialty pharmacy becomes a venue. They can drop ship to a physician’s office or an infusion center with a goal of hopefully moving to a total lower cost of care.

John C. Stancil, RPh: Sometimes with specialty pharmacy, even with the oral agents, they all have patient programs that are committed to adherence or improving adherence where they’re calling the patient—making sure they’re taking their medication, finding out if there are any adverse effects, and redirecting them to their primary care physician or their treatment-prescribing physician for issues—rather than showing up or presenting to the ED [emergency department] or the hospital.

Neil B. Minkoff, MD: As a clinician, dealing with this population, you mentioned earlier your hydroxyurea example. For example, compliance was not what you would like it to be, either. Are those sorts of clinical programs pushing adherence and compliance something that you welcome as adjunct? Or do you find them to be an interference between your relationship with your patient?

Ahmar U. Zaidi, MD: That’s a difficult question. But the answer is that I think that some providers do probably require a little of the need to be pushed on the adherence issue. I think it is helpful to sometimes push that adherence issue. But there are situations in which I feel like, with this personalized medicine arena that we’re in with this population, open lines of communication are so necessary in really determining the plan for every single patient. Sometimes that becomes the biggest issue: the lack of communication among the involved parties that are helping determine care for every patient.

John C. Stancil, RPh: That’s what we’re seeing now. It really is a team approach to the treatment of this disease or any disease. It can’t be siloed with the physician doing his thing, the pharmacy provider doing their thing. It really needs to be a team approach on how we care that will give the best outcome.

Ahmar U. Zaidi, MD: Absolutely.

Maria Lopes, MD, MS: I’d also like to add that you mentioned before, this can act with the psychosocial. And if that’s a gap on your side, how can the payer—through either care management or social workers—integrate with understanding the patient needs and maybe even the dynamics, because compliance is very personalized. There are many drivers of noncompliance. And we really need to understand that and then develop solutions to get to that 90% that we’re perhaps seeing outside the US. I think that is a huge opportunity to work out.