Proposed Outcome Measures

MGNet Seeks Public Comments on Proposed Outcome Measures

The NIH-supported Rare Disease Clinical Research Consortium (RDCRC) for myasthenia gravis (MGNet), which is part of the Rare Diseases Clinical Research Network (RDCRN), views standardization of myasthenia gravis (MG) outcome measures as a critical need for clinical trials. To address this issue, MGNet convened an outcome measure symposium and a working group charged with refining specific MG outcome measures and their instructions for administration, based on consensus opinion. The goal was to improve the clarity of instructions and scoring and reduce outcome measure variability.

Six outcome measures were included in this effort. Please view the draft measures at the following links:

Draft Quantitative Myasthenia Gravis Score (QMG) and Quantitative Myasthenia Gravis-Revised Score Instructions
Draft MG Activity of Daily Living Scale (MG-ADL)
Draft MG Quality of Life-15 (MG-QOL15 and MGQOL15 revised)
Draft MG Composite
Draft MG Impairment Index (MGII) and MG Impairment Index (MGII) Patient Questionnaire

Ultimately, implementing these recommendations is expected to lead to less data variability, greater efficiency, and fewer errors at clinical sites.

As the next step in the process, MGNet is seeking public comments to help ensure that the views of all stakeholders are heard. We would value your (or your organization’s) input on the proposed revisions.

Click here to provide feedback in a Qualtrics survey

The public commentary period will begin May 9th, 2022 and end Jun 24, 2022.

Following the public commentary period and final revisions, MGNet intends to develop a comprehensive set of training materials and case report forms for each outcome measure that reflect the recommended changes. MGNet anticipates holding training sessions for study teams at future meetings and offering initial and renewal certification processes for clinical trial raters.

Thank you for your input on this important effort to improve clinical trial conduct in MG.

Sincerely,

Jeffrey Guptill, MD
Michael Benatar, MD, PhD
On behalf of the MGNet Clinical Trial Outcome Measure Working Group

The Myasthenia Gravis Rare Disease Network (MGNet) is part of the Rare Diseases Clinical Research Network (RDCRN), which is funded by the National Institutes of Health (NIH) and led by the National Center for Advancing Translational Sciences (NCATS) through its Division of Rare Diseases Research Innovation (DRDRI). MGNet is funded under grant number U54NS115054 as a collaboration between NCATS and the National Institute of Neurological Disorders and Stroke (NINDS). This website is hosted by the network’s Data Management and Coordinating Center at Cincinnati Children’s Hospital Medical Center, which is funded by NCATS and NINDS under grant number TR002818.