Priscilla Forrester, 35, was diagnosed with myasthenia gravis (MG) in March 2020 after undergoing a thorough medical history, blood work, and a nerve conduction study and electromyography, which measures electrical activity in muscle when it's been stimulated by a nerve. In MG, an autoimmune disease, antibodies disrupt the connections between nerves and muscles, causing weakness, especially in muscles that control the eyes, mouth, throat, arms, and legs.
Since her diagnosis, Forrester has controlled her condition through medication. She and her doctor also are considering removing her thymus gland. Evidence suggests that the gland promotes the production of antibodies that interfere with the muscle messenger acetylcholine, so removing it may help those with high levels of immunoglobulin G antibodies, which block the receptors on the muscle surface that receive the acetycholine signal from the nerve. Most patients continue to take prednisone after the surgery, although some can taper to lower doses. Thymectomy combined with prednisone is more effective at reducing symptoms than prednisone alone, according to a 2016 study in the New England Journal of Medicine.
Thanks to several research advances over the past decades, Forrester and other people with MG have more options than ever to manage their disease.
Treatment depends on how patients' immune systems attack the communication between nerves and muscles, says Nicholas J. Silvestri, MD, FAAN, professor of neurology at the Jacobs School of Medicine and Biomedical Sciences in Buffalo, NY. Most people with MG produce immunoglobulin G antibidoes. When acetylcholine binds to the receptors on the muscle surface, it signals the muscle to generate a contraction.
One way to treat MG is with anticholinesterase inhibitors, which prevent the breakdown of acetylcholine. Another way is with immunosuppressants, which tamp down the immune system to prevent the creation of antibodies that disrupt the nerve-muscle communication pathways.
Corticosteroids, such as prednisone, also prevent the creation of antibodies that attack the nerve-muscle connection. Prednisone is typically prescribed for mild cases of MG in gradually increasing doses until improvement is observed. Prednisone is often prescribed in combination with other drugs.
Patients who have antibodies that target the acetylcholine receptor are often prescribed monoclonal antibodies. These newly approved treatments, which are administered intravenously or by injection, include eculizumab (Soliris), efgartigimod (Vyvgart), ravulizumab (Ultomiris), rozanolixizumab (Rystiggo), and zilucoplan (Zilbrysq). They are likely to cause fewer side effects than older medications, but they are very expensive.
For example, eculizumab, a drug approved by the U.S. Food and Drug Administration in 2017, costs $653,100 a year for an initial series of four weekly infusions followed by infusions every two weeks. Even older treatments, like immunoglobulin therapy and plasmapheresis, can be prohibitively expensive. During immunoglobulin therapy, patients are infused with antibodies purified from the blood of healthy donors, which reduces symptoms for a few weeks. Plasmapheresis, which takes a few hours, involves removing blood from the body and stripping the antibodies that cause MG, then transferring the altered blood and a blood product called albumin back into the body. Most people need several treatments over the course of a few days to see improvement that usually lasts a few weeks.
In comparing the price of some of the new drugs with the benefits expected, the Institute for Clinical and Economic Review (ICER), a nonprofit organization that analyzes the value of medical treatments, estimated that eculizumab should cost 98 percent less to be worth the price. The prices of efgartigimod and ravulizumab are $225,000 and $458,000 a year, respectively. ICER estimated that efgartigimod should cost $18,300 to $28,400 a year based on its potential effects.
Until the prices come down, everyone with MG, not just those who are uninsured, faces challenges in paying for treatments, says Joan Wincentsen, executive director of Conquer Myasthenia Gravis, a patient organization. Some insurance plans have deductibles of several thousand dollars, she notes. Also, when people switch insurance companies because of job changes or other reasons, they may find that their new insurers won't cover their prior medications. People may have to switch to less costly therapies and fail on them before their insurance companies will consider covering a more expensive treatment.
“‘Failing’ is not pretty,” says Wincentsen. “Symptoms may get worse, and it can take that much longer to recover from a flare-up before getting a medication that works.”
There are programs that cover not only medications but also home care, transportation to doctors' appointments, and other needs, says Richard Sagall, MD, a former family medicine physician who is now president of NeedyMeds, an organization that connects people with more than 30,000 assistance programs.
These suggestions from Wincentsen and Dr. Sagall may ease the financial burden.
Comparison-shop
Drug costs can vary from store to store and even from month to month. Check prices at different retailers to see where you get the best price. “I've seen hundreds of dollars' difference in prices from pharmacy to pharmacy,” Dr. Sagall says.
Apply to PAPs
Most drug companies have patient assistance programs (PAPs) that provide free medications to people who meet certain eligibility requirements—sometimes even people who are far above the federal poverty level. Most of the pharmaceutical companies that make drugs for MG have information about their PAPs, including 800 numbers, online. If the person you speak with doesn't provide satisfactory assistance, call again, suggests Dr. Sagall. “If you feel you come close to qualifying, there's always an appeal process, and appeals rarely get turned down,” he says. Your neurologist may be able to help with the appeal.
Look for discounts
NeedyMeds provides a discount card for up to 80 percent off the price of prescription and over-the-counter medications. Or call individual companies to ask for coupons.
Visit low-cost clinics
Some health care providers will charge patients based on what they can pay. NeedyMeds has a list of more than 18,000 free or low-cost clinics on its site.
Find MG–specific programs
Conquer Myasthenia Gravis has a Direct Patient Assistance Program that provides up to $1,000 per person per year for costs related to MG care. The Myasthenia Gravis Foundation of America has a list of assistance programs on its site, and a group called the Assistance Fund helps patients cover out-of-pocket costs. The National Organization for Rare Disorders has a program called RareCare that provides financial help for medications, insurance premiums and co-pays, diagnostic testing, and travel for clinical trials or consultation with disease specialists.
Advocate for lower prices
Patients for Affordable Drugs is a national nonprofit group established to encourage policy changes and reduce the cost of prescription drugs.
