Paul Melmeyer on Regulatory Advocacy for Patients With Neuromuscular Diseases

“Individuals in our community understand the urgency of acting as quickly and as soon as possible with whatever might be effective. And it's that urgency that they bring to conversations with the FDA, to conversations with biotechnology organizations, with any decision-maker that may not feel the same urgency because they're not living with a neuromuscular condition or they’re not a loved one of an individual with a neuromuscular condition."

Many unmet needs remain for patients with neuromuscular diseases. An issue facing the community is the disparity in perspectives on urgency between decision-makers in regulatory bodies such as the FDA and patients when it comes to evaluating and approving new treatment options. Multiple gene therapy candidates are currently under investigation for indications such as muscular dystrophy and patients hope to see some of these come to market as soon as possible.

Paul Melmeyer, vice president, Public Policy & Advocacy, Muscular Dystrophy Association (MDA), recently spoke at the 2022 NORD Breakthrough Summit in Washington, DC. The organization seeks to address unmet needs for the community through advocacy directed at various organizations, including regulatory agencies.

In an interview with CGTLive, Melmeyer discussed the MDA’s mission and the challenges it faces in pursuing its goals. He highlighted the necessity of patients with neuromuscular diseases and their families representing themselves in advocacy directly. He also discussed the importance of helping regulatory decision-makers understand the perspective of patients, specifically when it comes to understanding factors such as the risks that the patients are willing to take and what a clinical benefit truly means to them when it comes to investigational treatments. Melmeyer also spoke about what he sees as the points of unity in the diverse patient community, and how this relates to the organization’s priorities in advocacy.