Unmet Needs and Future Directions in Myasthenia Gravis


James F. Howard Jr., MD, and Nicholas J. Silvestri MD, FAAN, deliver parting thoughts on the state of the myasthenia gravis treatment, focusing on current deficits and future prospects.

Dr. James F. Howard: Where are the unmet needs?

Dr. Nicholas J. Silvestri: I think that thankfully as time goes on, hopefully, those unmet needs really shrink. But as I think of unmet needs, I think about our patients that are serum negative, that maybe don't have access to some of the therapies we recently discussed, like complement inhibitors and FcRn antagonists. Hopefully, over time, that changes. I think patients with severe ocular disease, like you mentioned before, Dr. Howard, those that maybe we don't treat aggressively enough, there's an unmet need there. And certainly, in those patients that don't respond to any of the therapies that are currently available. Though we are getting good response rates with complement inhibitors and FcRn antagonists. Not every patient is going to be helped by those. There are going to be a minority of patients where other options are necessary. Those are the unmet needs in my mind.

Dr. James F. Howard: Yeah. In addition, I think we need more data in our pediatric populations. We clearly have trials going on with eculizumab and trials will be starting with efgartigimod in the pediatric population, ravulizumab, zylupaplan once it's approved. The EMA, European Medicines Agency mandates small trials in the peds populations, which is something we didn't have decade, two decades ago. The other area I see that we need info in is pregnancy and the safety of these drugs. Complement inhibition eculizumab, primarily in PNH pregnancies appears to be relatively safe. We don't have any good data in myasthenia. I don't think there should be much of a difference perhaps, but clearly, that's an area where more information is needed. The other, as you said, zero negative. To me, that's a big one. While it represents a very small portion of the total population, for your practice and my practice, it represents the bulk of the patients we see. Because no one knows what to do with them. They send them to you. And so, we need something there. The other area I think that is really lacking though is starting to change is info and health economics. We know the cost of medication, we know the cost of hospitalization, but we don't have good handles on what's societal cost. What does the cost, if I give you diabetes because you're on tacrolimus or prednisone, and what's the lifetime cost now of treating your diabetes or osteoporosis that's severe, or the mood disorders that you get into because you've been on high-dose prednisone, etc.? In many of the agencies or the companies that look at health, economics don't factor any of this in. What's the impact on employment? And if 58% of folks in Thailand lose their job because of MG or in Japan, more than half have a reduction in work hours and wages, that's a huge cost and that we don't take into account. And so, my hope is that these are areas that will be forthcoming.

Dr. Nicholas J. Silvestri: I agree. Yeah, absolutely agree.

Dr. James F. Howard: Well, this has been great. And I want to thank you for watching this Neurology Live Peers and Perspectives. If you enjoyed the content, subscribe to the eNewsletters to receive upcoming opportunities of Peers and Perspectives and other great content right in your inbox. Again, thank you for watching. We hope you learned something and enjoyed it.

Transcript Edited for Clarity

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