Welcome to unscripted the AMCP podcast, a look inside managed care pharmacy. Listen in as experts explore the challenges, innovations, and opportunities shaping healthcare for millions of patients. Welcome to this episode of Unscripted, the AMCP podcast. I'm your host, Fred Goldstein. We continue our rare disease series discussing managed care considerations for incorporating patient value. Today we'll be focusing on the rare disease IgA nephropathy, one of the leading causes of kidney failure.
This is supported by an independent medical education grant from Alexion and AstraZeneca rare disease. Make sure to check the show notes for additional information. It is now my pleasure to introduce our faculty for today's program, Ami Gopalan, senior Vice President of Strategic Content and Growth Optimization at Precision AQ. Welcome Ami. Thank you for having me. And I just wanna say I'm here serving as faculty, uh, but I also have the hat here of, of being a patient.
I was diagnosed with IgA nephropathy five years ago. Thank you for providing that information. Obviously critical information to have to be able to discuss this from both sides of this. So why don't you go ahead and tell us a little bit about IgA nephropathy. Yeah, IgA nephropathy is a rare, progressive autoimmune kidney disease. It's rare as it affects about 200,000 patients in the United States.
Uh, but that diagnosis rate actually does vary sort of across across populations across the world in China and Japan. Biopsy rates, which is typically how IgA nephropathy is diagnosed, up to 40% of biopsies show that patients have IgA nephropathy. Compared to 10% in the United States. So that's why it's rare.
It's a progressive autoimmune disease as what we know about this is that the damage that's being done to the kidneys continues and it's due to the deposition of galactose deficient IgA molecules or complexes in the kidneys. And that deposition sets off a cascade of inflammatory events, which causes the kidney damage, uh, that we see in these patients. Is it typically because of needing that biopsy picked up late? It is.
Most patients do not get diagnosed until they're typically in stage three CKD because they have no symptoms. So they're, you know, they're walking around, things are okay, uh, or they think things are okay, but all the while, um, that kidney damage is occurring and it's not until, you know, they have some of your late stage symptoms that they're typically presenting, um, to to care. And you mentioned stage three, what is the clinical progression of the illness? It is.
So I noted it's, you know, it's a progressive autoimmune kidney disease. What typically ends up is you end up needing dialysis or transplant because of the progressive nature of the disease. And obviously something like that is very expensive, right? Yeah. Uh, it is, it is extremely expensive and I think it's also important to sort of keep in mind for those that.
Transition or, or get, um, a transplant and who are fortunate enough to, you know, find a match and go through that entire process because of its progressive nature, you're being diagnosed, uh, probably in your thirties or forties, about half of patients, um, may end up in end stage renal disease. You know, within their, in their fifties, while Medicare may cover their dialysis and, and that transplant, they may need another one, right? So, uh, IgA nephropathy doesn't go away.
And so just, I think that's just another thing to keep in mind is that this is something that you'll see both under your commercial plan as well as your Medicare plan and, you know, the end all be all in this case is not a transplant because it's very common for it to show up again. So I would imagine, given obviously the intense clinical issues associated with it, the disease burden on an individual and their family must be pretty severe. It, it definitely is.
It, it's one of those, obviously, you know, any, any disease is going to change how a patient and their family needs to. Really live their life. Uh, and this is, this is no different. Um, interesting, you know, because it's not something that people see. There's an intense, you know, invisible burden and a mental burden just in terms of dietary changes that may need to be made.
What that does in terms of, you know, relationships between, you know, family and friends, there is a lot that patients have to deal with. And where are we in terms of treatment for this illness? I would say it's exciting, um, but it can also be causing some concern. IgA nephropathy, I noted as it's, you know, this immune mediated disease. We know a little bit more about how that, uh, progression occurs.
And so a key component of this is the deposition of galactose deficient IgA1 molecules in the kidneys, in the glomerulus specifically, which sets off a cascade of events. And so now we have a couple treatments, or few treatments really, that are, um, on the market. Um, and we have several more in pipeline. And with those in pipeline, I think that's where, you know, we're seeing some really good information and data as it relates to, uh, management of the disease. But at the same time.
There's a lot of new drugs that are coming out as well. And you mentioned the impact, obviously it's on, on patients themselves and then the issues around the broader issues of mental health, et cetera. So where are we in terms of looking at patient reported outcomes and bringing that information in? There's some work that's being done, um, by the IgA Nephropathy Foundation. Um, to collect some of this information and, and really demonstrate some of the, uh, emotional burden, um, that's there.
The, you know, manufacturers that have treatments available here have been, you know, very supportive of the, the foundation and are sort of supporting some of those efforts to really, uh, engage patients. And what are the advocacy groups that are working on this finding and what are they seeing with this area? Patients are are challenged because as a rare disease, they probably haven't met anyone that has this condition.
So trying to find like-minded groups of people to sort of find people that understand what they're going through, that emotional toll as it relates to having a lifelong chronic condition, knowing that there is no cure. Has you know definitely posed challenges for, for patients. And, you know, another thing I think to point out is that this isn't just a disease of adults.
I noted that, you know, most patients may get diagnosed in their thirties or forties, but there's a significant pediatric population and so that can turn entire worlds upside down. And so IgAN Foundation in particular has been really supportive of, of patients, families, and, and caregivers. Given both your background as a clinician as well as a patient, how should managed care be looking at this?
Thank you for letting me answer that question from both the, the patient and the managed care perspective. Let, let's start with the, the managed care impact. IgA nephropathy is a being a rare disease. The treatment options that are available are typically being approved, are being approved through the accelerated. Approval pathway, and that means surrogate endpoints.
And in some rare diseases, the correlation between a surrogate endpoint and you know, that that actual effective endpoint can be, the data might not be that great. Um, in this case, uh, the data's pretty strong to show that the reduction in urine protein levels. Does correlate with the reduction in eGFR decline. So I think that's just an important thing to sort of keep in mind for this rare disease versus, versus others.
That being said, obviously we do wanna see what that difference looks like over, you know, the, uh, for eGFR decline, um, you know, once, uh, full data is available, coverage criteria is another thing. Um, and coverage criteria in this space is, is likely going to be aligned to the, the clinical guidelines. The clinical guidelines were recently updated in October of 2025, and they note a, uh, a management goal of less than 0.5 grams per day, um, of, of protein in the urine.
They also note, ideally, that protein should be managed to less than 0.3 grams per day. And so, you know, as managed care entities, we need to be thinking about, so what are we going to use as our, you know, our cutoff in sort of management of ensuring who has access to these. Really promising new therapies. And so I'm gonna flip that over then to the patient side. As a patient, I really hope that, you know, we're looking for that lower endpoint, right?
0.3 grams per day, which really demonstrates that a patient is in remission because that really offers, uh, patients the opportunity or the greatest opportunity honestly, to maintain their own kidneys. And, you know, for that, that's, I think just something to that I'd wanna make sure folks kept in mind. Well, that's really excellent. I'd like to thank you, Ami, for coming on. Of course. Thank you. And thank you for listening to this episode of Unscripted the AMCP podcast.
Join us for the next part of this series as we dive further into rare diseases.