Unscripted - Sickle Cell Disease: Measuring Burden, Managing Pain, and Improving Care

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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. We are wrapping up our final episode of this rare disease series discussing managed care considerations for incorporating patient value.

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Today, we'll be focusing on sickle cell disease, a disease that has a huge impact on the quality of life for both patients and their families. This podcast is supported by an independent medical education grant from Agios Pharmaceuticals, Inc. It is now my pleasure to introduce our faculty for today's program, Angela Luong, Senior Clinical Consultant for Pharmacy Strategy Group, or PSG.

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Welcome, Angela. Hi, Fred. Thanks for having me today. It's fantastic to get you on. Why don't we begin—give us a little bit of background on sickle cell disease. Sure. Sickle cell is one of those inherited diseases. So if you have it in your family, typically then it's passed down. Most Americans hear about it, but they don't actually know the full impact it has on someone who actually has it.

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There's different variations of it that have different severities too, that could affect someone's life. And when somebody has this disease, how is the typical progression? Sure, the progression for sickle cell disease could start in patients as young as six months old. They can start feeling pain. That's usually the telltale sign of having sickle cell disease.

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And then as you grow older, there's other complications that come with the progression, such as anemia, which is pretty common. And into adulthood, this pain actually is about 95% of your days. So if you could imagine, right, having a great quality of life—having sickle cell and having pain every day—I think that's pretty severe, too. It could affect your own mental, behavioral, and overall quality of life with sickle cell.

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So when someone is living with this disease, how is the treatment normally? Well, I think a lot of times, right, the treatment with sickle cell for pain can get pretty confusing because I've heard a lot of times when they're in pain, they could be seen as opioid-seeking or pain medication–seeking, because that's really the forefront of controlling sickle cell disease: opioids and pain medications.

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And a lot of times they're going to be using doses higher than usual because of that tolerance over time. And so obviously that's impacting the patient and their life in taking medications like that. So how do you incorporate that kind of thinking into your therapy around what they're taking and how it's impacting them?

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Yeah. Unfortunately, for sickle cell disease, there are limited treatment options, which is part of the issue, Fred. There are only three disease-modifying treatments available, two of which are oral—so we'll see that in pharmacy claims—and then one that is an IV infusion. So it's difficult, I think, for these patients to really seek out therapies because there isn't much available.

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And obviously, as we talked about, there's this profound impact. What sort of patient-reported outcomes would be important for a clinician to understand or measure when they're working with individuals with sickle cell disease? I think with sickle cell disease, it's really important to use data, right? Coming from a managed care perspective, we don't see the patient frequently, but what we can use is data.

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And the important part of this is not just drug claims or what they're filling, but also how often they're going into the ER. Those are, I think, the most profound outcomes. If they're frequently going into the ER for their crises—which could even be as severe as having a stroke or a heart attack—using that and seeing how frequently they're going in, I think patient-reported outcomes may be a little bit difficult because of that pain tolerance that develops over time.

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But we have to use a combination, right—of seeing what they're using for drugs in terms of pain, how often they're using their pain meds, and how often they're having hospitalizations and ER visits. And you've talked about data, and obviously that's critical to managing this disease. And one of the things I noticed is we were discussing this previously is your dashboard.

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Can you talk about what sort of data you have on your dashboard that you're using? Sure. What I use on a daily basis integrates pharmacy and medical data all into one. So in terms of having patient management in sickle cell disease, we're able then to look at a patient holistically, right? So if you're in a provider office you're seeing them regularly, hopefully.

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But you don't see how often they're filling their drug prescriptions. So are they adherent? What else are they seeing? For instance, are they seeing another provider? Are they getting opioids from somebody else? Are they actually filling their hydroxyurea, which can help prevent this pain? I think on top of that, right, our dashboard is able—our dashboard that you can develop—

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you're able then to put both their medical history with hospitalizations. Are they going into the hospital and they're just not telling you, right? And even how long they're in the hospital, right? Was it a day? Was it a week? Was it two weeks? Did they have an adverse event and they just didn't tell you? I think being able to have that holistic view and using data would be helpful.

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And you really lay this out in a linear approach that allows you to see changes or adjustments over time and say, hey, something's happening here, we need to look to intervene—or something is going well and we can continue moving on? Yeah. And even like I've looked at some patient profiles, and it's hard being a pharmacist in managed care really understanding that patient level.

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But I've viewed sickle cell patient profiles in which they were seeing five different providers, right? They're getting drugs through like three different facilities, are going to two different community hospitals. So being able to really put that information together and say, okay, how can we best manage them? Can we just refer them to a specialist, right?

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Are they comfortable going somewhere? Can we provide additional services? Are they having issues going to see a provider nearby or a specialist? So I think being able to use that and then to adjust, right, to provide the additional support—especially for sickle cell disease—is important. And one of the areas I assume would be important in this is using centers of excellence or groups that really have a lot of experience treating sickle cell.

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Yes, for sure. I think in any of these rare diseases, being able to partner with Centers of Excellence, having a care management program that creates a linear patient experience and a patient journey, I think also helps, right? Providing additional support—not just like, okay, did you get a prescription? Are you filling a prescription? But what else do they need help with?

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In sickle cell, there's a lot of data out there, and there's a lot of studies that show there are some health disparities in this population. So having that additional support, such as behavioral and mental health capacity, transportation, food—I think food access and transportation are two areas that's been highlighted as well in sickle cell disease.

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Yeah. And you've touched on these social determinants of health issues. And given the fact that a large number of these individuals are on Medicaid, that's obviously something to make sure you have integrated in your program. Yeah, I think the difficulty, too, is patients jumping on and off Medicaid programs, right? So having that full view and creating a transition, too—right, if they're on this month, but two months later they come back—what happened?

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Being able to have an open line of communication and trust, I think, in the health care system is important. You touched on the behavioral aspects of this illness. Obviously, it has a pretty profound impact on individuals having to live with the pain, etc., and taking the medications. So they really should be looking to bring in, as a team, possibly behavioral health expertise.

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Yes, for sure. I mean, I think that goes into part of pain management as well. I mean, if you have pain 95% of your days, imagine how much that's really going to affect your ability to function—employment, going to school, just even going to the grocery store. So a lot of that, I think, would be helpful in terms of when we talk about disease management.

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Right. It's not just the drug management. I think it's overall holistic well-being. That's a really great way to put it. And then you mentioned transitions and that idea of transitions on and off Medicaid. I also understand the issue of transition from pediatric to adult care can be a difficult point as well. Yeah, there are some studies out there, too, for sickle cell disease patients in that transition between pediatric care.

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Right. As when you're younger, that pediatric care is actually substantial. You have an amazing staff, you have everybody walking you through. And then as you transition into adulthood and maybe your coverage changes, your health plan changes, and your providers change, right—because pediatric care changes as you grow—there's that gap that's not filled anymore. You don't have a robust team anymore.

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You don't have, you know, a dedicated nurse or you don't have a dedicated behavioral health specialist. So there is that gap between pediatric care and adulthood in sickle cell disease management. And as you think about this from a closing perspective, if you're a managed care entity, what sort of points would you just highlight again that they should be considering as they work with individuals living with this disease?

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I think with sickle cell in particular, it hasn't been on a lot of payers' and managed care organizations' radar until recently because gene therapies were approved, right? And these gene therapies are costing $2 million to $3 million. I think because prior to this, hydroxyurea is a generic drug. I mean, you're not going to have high-cost patients.

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So it wasn't on a lot of managed care organizations' radar. But as we are moving forward, I think being able to really understand holistic care, being able to create care management programs around these rare diseases that are hereditary, right—you’re not just going to have one patient; you may have a whole family. So being able to create a care management program, and also partnering with the Centers of Excellence, as you mentioned earlier, would be beneficial too, because then you know that your patients are having the best quality care and they know where to go.

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They're not just going to any ER to get their pain treated, right? They're not just staying at home and waiting till it gets really bad, but they're preventing the disease from progressing any further. Well, I'd like to thank you, Angela, for joining us today. Yeah. Thank you, Fred, and thank you for listening to this episode of Unscripted, The AMCP Podcast.

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We hope you've enjoyed this series on rare diseases.