Hemophilia: Patient Burden, Innovation, and Managed Care Strategy

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Welcome to Unscripted, The AMCP Podcast, a look inside managed care pharmacy. Listen in as experts explore the challenges, innovations, and opportunities shaping health care for millions of patients. Welcome to this episode of Unscripted, The AMCP Podcast. I'm your host, Fred Goldstein. In this episode, we continue our discussion on rare diseases and how managed care can incorporate patient value.

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Today, we'll be focusing on hemophilia, a disease with a high burden on patients and their families. This podcast is supported by an independent medical education grant from Genentech, Inc. and Pfizer Inc. It is now my pleasure to introduce our faculty for today's program, Janna Evans, Director of Pharmacy Sales Support at Blue Cross and Blue Shield of Texas. Welcome, Janna.

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Thank you for having me today. A pleasure to get you on and really looking forward to this discussion. So why don't we start—give us a background on hemophilia? Yeah, actually, hemophilia is something I've had to learn about because of the costs associated with it. And I think a lot of people in the health plan setting, in the payer space that I work in, end up learning a lot because of the cost of the medication.

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This is a clotting disorder. So people have either a defect or deficiency of factor VIII or factor IX, depending on the type of hemophilia. It causes a lot of complications. Depending on how much active clotting factor an individual has, it could be very severe, where they spontaneously bleed in joints, and it can be very painful for the patient.

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And there can be complications, such as inhibitors, where the body develops autoantibodies to the clotting factor, or bleeding episodes that are prolonged and potentially life-threatening. So it's very difficult and a lot of burden from a patient perspective to have this condition, regardless of severity, but definitely more so with greater severity. In addition to some of those physical symptoms and the complications I mentioned, mental health and psychological concerns can be very prevalent in people who have hemophilia.

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You think about it: if you know that you're going to bleed more—or in response to some injury that's more mild—you'll be more cautious about what you're doing. It can have a lot of mental health impacts for that reason, just because people are afraid and very vigilant about their activities and bleeding. I would say in the employer space I work in, in the commercial space within the health plan, it's very concerning from an employee perspective too.

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It may even limit the jobs that someone might take if they have hemophilia, to prevent the risk of injuries where they would have those bleeding episodes. So it's really an impact on quality of life. I would assume that for each person, it's sort of individualized based on how they decide to live with that disease. Yeah. So patient decision-making and that shared decision-making framework with the provider is very important,

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because the goals for each individual who has the condition can be different. So they do have those quality-of-life impacts. But there are a variety of different treatments that come out, standardly. We really looked at things like infusions of clotting factors, either plasma-derived or recombinant technologies. We've been able to extend those. Those were either used on demand for a bleed if there's less severity, or even preventive in prophylaxis to prevent bleeds in those who have more severe forms of hemophilia.

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So that can be very difficult for a patient to receive that type of treatment because it's constant infusions. They may be able to learn to infuse at home, but there are complications potentially that come with that. And they may need to have a port or some kind of venous access. There is a risk of infections with that. So lots of worries that come with this type of condition.

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Now we have seen in the treatment space quite a bit of innovation. So we've seen products that aren't clotting factor, but mimic clotting factors to help with that clotting cascade that's not working properly in an individual with hemophilia. We've also seen some gene therapies, and that's the correcting of those genes that are getting in the way of the clotting cascade, come out as potential solutions to this.

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So lots of innovation happening in the space. We have very efficacious treatments still, certainly, and a lot of burden in terms of receiving the treatments that exist today. And given these various treatments and obviously the cost associated with them, how do health plans typically look at this? I mentioned, you know, I learned about hemophilia because I work at a health plan.

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And when you've got someone with severe hemophilia, that's always on your radar—both in terms of the complexity of the patient and the costs associated with that. So being that the treatments are largely on the pharmacy benefit, and drug therapy is, we're monitoring that very closely. From a health plan perspective, we want to minimize risk to the payer, either from an insured perspective or from a commercial perspective for an employer group.

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So cost containment is a big deal. It's tricky to get it right—to get the right access to medications that are going to help on an individual basis for that person, and also make sure they have access to the newest treatments. You'll see things like working with preferred products depending on the PBM or health plan involved.

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There's a lot of opportunity for care management and coordination of care that are kind of ancillary to managing the drug itself that are very important for an individual as well. And sometimes we see less-than-optimal types of management when we think about the patient perspective. So we do have some payers that may want to carve out this particular therapy,

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take it off of the benefit just because of the costs of that, or put in prior authorizations that can be burdensome to the patient. I think prior authorization is pretty standard, but the requirements for it vary between different payers and different health plans. That can be a difficulty in itself for patients: knowing what their particular plan will cover, which benefit it covers, what are the requirements?

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There's some degree of step therapy sometimes—so try a lower-cost medication, that's what some plans will do, before they get to a newer medication. Those can get in the way sometimes of patient access to treatment if the workflows aren't smooth, if the requirements are very stringent for bleeding, for instance, as a measure of whether that drug is accessible or whether the patient has tolerated the drug.

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And so in a lot of ways, the management of hemophilia is equally as complex when we try to factor in the very patient-specific needs with the disease. I would guess this is also an area where you might be looking to establish centers of excellence as a health plan. Yeah, that's one of the ways that some health plans have worked through it.

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There's been a lot of collaboration in the industry with hemophilia treatment centers, for instance. So it's kind of that patient-centered place to navigate the patient to the right treatments, to potentially dispense the medication. Certain pharmacies may be considered a center of excellence based on the support that they provide for the member: navigating and paying for these very costly drugs.

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They're expensive for the member just as much as they are for the payer. So making sure that that's affordable to the person that needs the life-saving treatment associated with hemophilia. Also coordinating between different providers—there tends to be a hematologist prescribing medication—but the centers of excellence can really bring together multidisciplinary teams in a way that's not necessarily standard within our health system.

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And you touched upon the issues that the patients face and the individualization of those. When you think about this and you're in your plan, what sort of data would you like to get that would help you make better decisions as you're seeking to help these individuals? So oftentimes what we have access to from a health plan perspective is claims data, right?

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We see the fills of the medication for factor products. Sometimes, in collaboration with the pharmacy, we can see bleeding logs or those measures of outcomes. So that's very helpful. And I don't often see things like patient-reported quality of life or those measures that show that disease burden and show what it means to the member to live with this condition, and what their treatment means to them.

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So I think that's an area of opportunity for potential expansion, and definitely something in the industry that regulatory agencies are working on, and different advocacy groups are working on—really bringing in that patient perspective. And with the ways that she and her committee works, representing that patient voice, there's little elements in there right now. We can see the claims, we can see how much factor is being dispensed,

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we can see bleeding logs, but continuing to bring in the humanistic parts of that is very helpful to understand the outcomes and benefits of the treatment for the patient. And I would assume one of the other areas that would be important is the impact that it has on the individuals and their ability to work and function at work, and presenteeism and absenteeism as well.

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Yeah. And I would say that both from the patient perspective and the caregiver perspective. So when I work with employer groups, that is a big concern for them. Do they have an employee that can come to work safely, or do they have an employee that has to leave work to care for a child with hemophilia, for instance? And so the bleeding episodes can be very disruptive.

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People that have hemophilia are more likely to use the emergency room or have an outpatient or inpatient stay, depending on those episodes. So that is very disruptive when we think, from an employer's perspective, to have an employee that may not be able to be present or risk their safety for certain types of jobs when they have this condition.

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Well, I'd like to thank you, Janna, for joining us. Absolutely. And thank you for listening to this episode of Unscripted, The AMCP Podcast. Join us for the final episode of this Rare Disease series, where we'll discuss another blood-related disease.