Barriers to Treatment in Pediatric Myasthenia Gravis: Jonathan Strober, MD

In the second part of this recent interview with Jonathan Strober, MD, a pediatric neurologist specializing in neuromuscular disorders at the University of California, San Francisco, and Benioff Children's Hospital, he addresses why myasthenia gravis is so rare in pediatric patients and disease triggers in this group. He also explains the importance of regulatory approval and insurance coverage for these patients, explaining that without approval, there may not be access to needed therapies.

Revisit part 1, where Strober discusses extending nipocalimab use to patients as young as 2 years.

This transcript was lightly edited for clarity; captions were auto-generated.

Transcript

With myasthenia gravis being so rare in pediatric patients, what factors are thought to trigger the disease in younger patients?

One is that a lot of kids, for some reason, don't make the antibodies like we see in adults. It’s, again, something we know is working on the antibody level, so we want to make sure the patients who are in the trials have the antibodies that we can watch and make sure they're dropping to make sure it’s effective for that. We do know that in some patients who have myasthenia, who we believe have myasthenia but are antibody negative, what we call seronegative, if we repeat their testing over time, eventually they do develop the antibodies. For whatever reason, the antibody levels get delayed in appearing in the blood.

We're also developing better tests to be able to pick up small amounts of antibodies better. We're using better state-of-the-art testing to be able to confirm. We have other ways of confirming myasthenia in patients who are seronegative by doing electrical testing and testing with different medications to see if it’s something that we believe they truly have, even if they don't have the antibodies. But again, for these studies, we need to make sure we're really doing this study in a patient population that's as similar as possible, and so having those antibodies helps us make sure that we're trying to treat the same condition.

How do insurance and regulatory decisions influence whether patients can actually receive the most appropriate treatment?

I don't know exactly what they go through in Europe vs what we go through in this country. I can tell you that if it's not approved by the FDA, it's really hard to get insurance companies to cover it. Often they say it’s experimental if it’s not approved by the FDA, but even once it's approved by the FDA, because the newer drugs tend to be a little bit more expensive than older drugs, and the older drugs being more generic [and] cheaper, a lot of insurance companies try to push us to use the older stuff, even though those still haven't been approved for this specific condition. It's just that they're older and we've been using them for longer. It's really important for these regulatory agencies to approve them or provide positive feedback so that these drugs can be used and [we] know they're being used safely, but also that they can get paid for our patients.