Advancing Myasthenia Gravis Treatment Access via Guideline-Directed Expertise

There are key systemic factors crucial for ensuring broad access to new myasthenia gravis treatments, including guidelines and research, improved insurance coverage, inclusivity in clinical trials, and advocacy and education, explained Beth Stein, MD, chief of neurology, St. Joseph’s Health, and Ratna Kiran Bhavaraju-Sanka, MD, the John H. Doran, MD, FACP, Endowed Chair in Peripheral Neuropathy. They also noted that advocacy at the legislative level is vital to support "tiered therapies."

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

Transcript

What are key considerations for ensuring access to myasthenia gravis treatments?

Beth Stein, MD: I think that what's really important is the advent of new guidelines and clinical trials indicating their need. Once it's indicated and we show that it works, we'll be able to get more improved insurance coverage for these patients in order to attain these targeted therapies. Without appropriate guidelines, without that appropriate research that's showing that there is benefit to these patients, we're unable to meet the needs of the patients and get these costly therapies.

Ratna Kiran Bhavaraju-Sanka, MD: I agree with Dr Stein that we need better guidelines. A lot of the insurance organizations and payers are looking at our trials. It's also important to include different populations in our trials because a lot of times if we only choose a super-refractory patient, they're only going to approve this therapy for a super-refractory patient. I think we need good guidelines, access to trials, [and] better inclusion of varied populations in the trials.

We need to also include advocacy, advocacy at the level of legislature that you can be doing tiered therapies. There are certain patients who respond to certain therapies, and they don't need to fail a certain therapy to be able to get the medication that we believe, as their physicians, works for them.

I think it's important to do advocacy [and] education, and be super inclusive with our trials of different patient populations.

Stein: As we move more from this paradigm of escalation therapy to more proactive disease management within the guidelines, we'll have improved insurance coverage. It'll limit the financial burden both on the economic health system but also on the patients themselves. Improved insurance coverage is really key, and I think we're going to get that as we move to a more proactive disease management state, as guided by our guidelines.

Bhavaraju-Sanka: Then to add on, sometimes though these therapies can be financially burdensome early on, their long-term benefits and on the economics we are putting patients or people into the workforce. We are making them more breadwinners. We are making them take care of their families, not get admitted to the hospitals for crisis or not have the chronic complications of some of the conventional therapies, and not getting diabetes or hypertension or cardiac disease. I think these data are important and that also impacts our health care system of the burden of improper therapies and side effects from some of these therapies.

Stein: Also, if we move toward more upstream medications, if we can lead to better control earlier, while it may seem like a huge burden initially with the treatment, ultimately they'll need less medication over a longer period of time, and therefore maybe the ultimate burden on the financials on the health care system would be less.