Payer Education Is Key to Early DMD Therapy Access

Timely initiation of care is crucial for patients with Duchenne muscular dystrophy (DMD), a rare, inherited, degenerative muscle disease with limited treatment options. But while the disease-modifying treatment armamentarium has been expanding in recent years, access to the latest approved therapies can be a challenge for patients and providers, Aravindhan Veerapandiyan, MD, a pediatric neuromuscular specialist at Arkansas Children’s Hospital, told The American Journal of Managed Care^® (AJMC®).

Aravindhan Veerapandiyan, MD | Image Credit: Arkansas Children's Hospital

Veerapandiyan, an associate professor of pediatrics at University of Arkansas for Medical Sciences, and colleagues authored a commentary published in Annals of the Child Neurology Society that outlined challenges associated with access to the latest DMD therapies.¹ In part 1 of this interview, Veerapandiyan spoke to the latest therapies for DMD, challenges in accessing those therapies, and the importance of timely therapy initiation for patients.²

Here, he discusses the benefits and limitations of the FDA’s accelerated approval pathway and how to overcome therapy postponements and denials.

This transcript has been lightly edited for clarity.

AJMC: What are the benefits and limitations of the FDA’s accelerated approval pathway when it comes to DMD treatments?

Veerapandiyan: With the accelerated pathway, the approval is based on a surrogate end point that can reasonably predict the functional outcome. The positives would be early access to therapy—I think that's important. If there is a therapy that has the potential to change the trajectory of the disease, it's critical that it gets out sooner. So, earlier access to therapy is the main benefit, or main positive, with the accelerated pathway.

For limitations, I think sometimes we tend to forget that phase 3 confirmatory data are still required for a full approval. I think we need to keep that in mind, and I think we all have to work collectively with our industry partners to have that provided to the FDA.

Another limitation is educating our payer partners. A lot of them look at accelerated approval as still investigational, and they don't consider this an approved drug for the particular indication. That's a limitation we need to look at from that perspective.

AJMC: Your paper noted that payers’ use of prior authorization, or peer-to-peer review, can drive treatment postponements and denials. How might this issue be addressed?

Veerapandiyan: What I get, from a payer standpoint, is that these are expensive therapies. So one of my recommendations, or tips, would be that I typically do the discussion part more proactively. As soon as we have a therapy that's approved, or even in the pipeline, I'm reaching out to my contacts on the payer side—it could be a state payer, it could be a commercial payer. I'm reaching out to them and seeing what conversations we can have. So that's a tip.

In terms of, let's say, if you're starting the prior authorization process, there's a denial, and you're going to do a peer-to-peer review, the first question that you have to ask is, does the person on the other side have the ability, after the discussion, to overcome that denial? Most of the time, you have someone who will probably end up saying, “Hey, I get it, but I just can't overcome the denial.” Then, it becomes, why am I wasting my time doing a peer-to-peer review? At the start of the discussion, I would ask, “What’s your specialty?” If you're not a pediatric neurologist or neuromuscular specialist, I don't think it's worth doing the peer-to-peer review. And if they do not have the capability or authority to change and overcome the denial, then I wouldn't do the review; I would move on to the next step.

Along those same lines, there's also something called external review. With some of the private payers, if you go through the process of prior authorization, there is a process called external review. You can also request to have a pediatric neurologist or neuromuscular specialist do an external review.

References

1. Veerapandiyan A, Connolly AM, Mathews KD, et al. Access to novel therapies for Duchenne muscular dystrophy—Insights from expert treating physicians. Ann Child Neurol Soc. Published online June 11, 2024. doi:10.1002/cns3.20076

2. McNulty R. Access to novel DMD therapies remains challenging despite regulatory approvals. AJMC. August 5, 2024. Accessed August 28, 2024. https://www.ajmc.com/view/access-to-novel-dmd-therapies-remains-challenging-despite-regulatory-approvals