Skip to main content

New study to establish evidence for myasthenia gravis treatments

Tuesday, August 22, 2017
By Will Alexander
Sanders

For the first time, patients and clinicians may have empirical evidence for the best treatments for myasthenia gravis, thanks to a newly funded research project led by Donald Sanders, MD, and Pushpa Narayanaswami, MD (Beth Israel Deaconess Medical Center/Harvard Medical School), and funded by the Patient-Centered Outcomes Research Institute (PCORI).

A variety of treatments exist for this rare but debilitating, potentially fatal condition. However, clinicians still don’t have strong evidence for how these treatments compare in alleviating symptoms or long-term management of the disease. In addition, the ideal treatment for an individual varies based on that person’s disease severity, age, gender, and other factors. The $2.5 million PCORI contract will allow Sanders and Narayanaswami to follow roughly 200 patients from across the United States and Canada for two years each.

“This study is the culmination of many years’ work with myasthenia gravis patients and their physicians, and will bring together members of an informal network of neurologists who share the common goal of improving the lives of their patients,” said Sanders. “It provides a unique opportunity to help patients--and their physicians--answer the question, ‘Given my diagnosis and disease severity, which treatment(s) will be the most effective for me, with the least side effects?’”

The prospective, observational study may also help myasthenia gravis patients receive coverage for the costs of their health care. Because no treatments for myasthenia gravis have FDA approval, insurance companies are sometimes reluctant to reimburse patients for treatment. Stronger evidence for the benefits of treatments may make insurers more likely to cover those costs, Sanders said.

Narayanaswami said that the study will have two specific approaches. "First, the project will help clinicians to directly compare two commonly used  treatments to see if either is more effective than the other, or if both are equally effective. Secondly,  it will test the validity of the international consensus guidance statements for myasthenia gravis sponsored by the Myasthenia Gravis Foundation of America research funding and published last year," she said. "At the end of the three-year study period, researchers will be able to analyze whether patients receiving treatment meeting this guideline did better than patients who did not."

The Duke Clinical Research Institute (DCRI) will act as the primary research facility and coordinate the results from the various centers collecting data. In addition, other researchers, clinicians, patients with myasthenia gravis, advocacy groups such as the Myasthenia Gravis Foundation of America, and other stakeholders will be involved in the implementation of the study and dissemination of the results.

The award has been approved pending completion of a business and programmatic review by PCORI staff and issuance of a formal award contract. PCORI is an independent, nonprofit organization authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers and clinicians with the evidence-based information needed to make better-informed healthcare decisions. For more information about PCORI’s funding, visit www.pcori.org