Ensuring Access to Pegcetacoplan Requires Updated Guidelines, Education, and Distribution Solutions: Carla Nester, MD, MSA, FASN

Last week, supported by data from the phase 3 VALIANT trial (NCT05067127), the FDA approved pegcetacoplan (EMPAVELI; Apellis Pharmaceuticals) as the first treatment for patients aged 12 and older with C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), 2 rare and severe kidney diseases that can lead to chronic kidney disease.

In part 2 of an interview with The American Journal of Managed Care®, Carla Nester, MD, MSA, FASN, lead principal investigator of the VALIANT trial and director of pediatric nephrology at the University of Iowa Stead Family Children's Hospital, discusses the steps necessary to ensure equitable access to pegcetacoplan and key areas for future research to support long-term treatment success.

Revisit part 1 to learn about the trial's findings and how pegcetacoplan is reshaping treatment for C3G and primary IC-MPGN, especially in pediatric patients.

Transcript

Considering the rarity of these diseases and the potential financial burden of treatment, what steps should be taken to help ensure equitable access to pegcetacoplan?

The issue of equitable access—particularly even globally—is a little bit difficult [to ensure], but I think a simple approach would be to simply update our guidelines. Our guidelines are still suggesting mycophenolate and prednisone, which is fine, and that's what we had at the time, but we do need to move towards a set of expert-based, and even now evidence-based, guidelines. I think it will help the local providers, wherever they are in the world. I think it will help them manage the diseases better if they know that there's a set of guidelines that are available.

Then, I think, along with guidelines comes education. We have to educate people that targeted therapeutics, such as pegcetacoplan, exist so that they can now use them and be more precise about their approach, and that is going to require an educational process.

Finally, access includes not only those guidelines but also education, and it also probably requires solving, literally, how do we get our hands on the drug? Or, if we do get our hands on the drug, what is the burden to the provider or even to the patient to make that happen? I suspect we have some things we need to solve in that area, but that's actually a good problem to have, right? It's good that we actually have something that we are now going to focus on access to.

What unanswered questions are you most excited to see explored in ongoing or future studies involving pegcetacoplan?

Some of the most exciting pieces going forward, I think, I'll start with, is this really a cure? I've stopped short of actually using that terminology because we don't know [if it is a cure], but I would love to know that if my patient responds to pegcetacoplan, are they, in fact, done with their disease? As long as they stay on medication, are they now going to carry their baseline kidney function forward for years and years and years? I need to hear that. I need to see that. That would be wonderful. I think that's one of the most important things for our clinicians.

Also, I would like to see the real-world safety data. Once we've been using these agents for a year or 2 or 3 or 10, for instance, are we still doing a good job safety-wise? I think that's more on the clinician than it would be on the sponsors of any of our complement therapeutics. We just have to watch our patients well and see how that works.

Finally, I think, because I'm half a pediatrician, I want to see these medications taken down even lower so that we can actually be able to see whether pegcetacoplan would work in my 4-year-old or my 8-year-old, for instance. I'm hoping that that's a very near future direction for us to go with this medication.