Many gene therapies promise life-changing effects, but without long-term data it remains to be seen how long the benefits last.
Despite the long-term benefits of gene therapies, there still requires additional treatment and follow-up to ensure that disease remains under control, especially because most data are short term with less known about the long-term effects of these treatments, explained Michelle Booth, PharmD, senior director of specialty clinical solutions, and Andy Killpack, PharmD, director of specialty clinical solutions, both of Magellan Rx Management.
How should payers and providers balance the benefits of the largely one-shot nature of gene therapies—and their long-term benefits and reduced treatment burden—with the high cost of treatment?
Booth: I can see where there is a tradeoff. First and foremost, if I were a provider, I would want to assure that the right patients are getting the right medication. There really shouldn't be any wiggle room, and I do feel that providers should be responsive to what the study population was, as much as possible. Obviously, there are outliers that may be appropriate for the medication—I understand that that happens. But I really do feel that they need to be mindful of the study population.
From the perspective of continuing to see the patient, I would imagine they can't fall off and [think] “I don't have hemophilia anymore.” They’re still obviously going to need to be monitored, and I feel like it is potentially a different type of monitoring at this point: looking at not necessarily what you can see easily, like symptomatically, but what are we not seeing? What are the factor levels? Are those responding appropriately? Did they continue to respond appropriately? Are we starting to drop off? At what point are we going to need to start being concerned that somebody's going to need factor replacement?
I feel like it's uncharted waters. I’m really kind of unsure of how we're going to proceed with these patients, because we're at the forefront, and these are all new, and replacing somebody's gene and actually fixing them is a new territory. So, I think that we certainly are going to need to approach the patients that we're treating with gene therapies very differently—regardless of the vector and across the board. We are going to need to approach this a little bit differently.
Killpack: Michelle summarized it well. We're really navigating uncharted waters. We've explored this new space of treatment types, and we have great hope that they're really going to work, they're going to have this durability, they're going to have long-term great effects. But we don't know that yet. We have some short-term data that look really good. We have some life-changing treatments for a lot of these diseases. But how long are they life-changing? That's the question that remains unanswered that we're trying to navigate.
Then tied to that is: What if they are and what if they aren't? Should it wear off over time, and you have to go back to the standard of care today, what does that mean for that treatment that was administered? What does that mean for the payer? What does that mean for the patient? What does that mean for the manufacturer? And those are questions that are unanswered today. Those are the things that ongoing discussions are going to have to take place. Some kind of agreement should be decided upon between the parties involved. And, as of yet, we don't have that. But those are the questions that are out there that we need to be talking about and looking to answer as more time comes on and more information is available with this real-world evidence.
Theoretically, should payers find these therapies attractive despite the high price tag because they lead to avoidance of other costs?
Booth: Yeah, because we're taking into consideration [emergency department] visits, hospitalization, home care. I'm envisioning a child with SMA [spinal muscular atrophy] and all the ancillary services. Caregivers coming in, overnight nursing or medical assistants coming in. Yeah, I agree. It certainly is something that would be attractive to insurers.
It's almost like you're paying up front. It's not a bad thing. It's just a lot of money. So, you worry about how am I going to pay for this? It's a budget buster.
But yes, I certainly agree that it's all encompassing. We’ve got to get out of the pharmacy silo. The budget goes beyond just, how much does the drug cost? I agree completely with that.
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