Paul Melmeyer

“Over the course of the last several years, we've been working on trying to strengthen the FDA’s capacity, expertise, and ability to quickly review gene therapies that come in front of them. The Office of Tissues and Advanced Therapies in the Center for Biologics Evaluation and Research (CBER), who is responsible for gene therapy reviews, has been underfunded and understaffed for years at this point. We are very happy that due to our and many other organizations’ advocacy, we are able to successfully enact provisions within the FDA User Fee Reauthorization... That would greatly expand this office, that would actually elevate this office to be The Office of Tissue Products within CBRE..."

Many investigational gene therapies are currently in development that have the potential to address unmet needs for patients with neuromuscular diseases, including muscular dystrophy. However, bottlenecks, understaffing, and inefficiencies in regulatory processes can hold back the review and approval of these therapies.

, Paul Melmeyer, vice president, Public Policy & Advocacy, Muscular Dystrophy Association (MDA), who recently spoke at the 

 in Washington, DC, discussed the organization’s work with regulatory agencies to help address these issues. He mentioned the specific steps MDA has taken with the FDA regarding the Office of Tissues and Advanced Therapies and provisions pertaining to a patient-focused drug development meeting on gene therapies. Melmeyer also highlighted several additional regulatory and legislative goals he sees as the highest priorities for the patient community. He concluded by mentioning that neurologists and other allied medical professionals can help support the organization’s goals for December.

Videos

The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the organization’s work with regulatory members to advance gene therapies towards review and approval. 

Paul Melmeyer on Addressing Gene Therapy Regulatory Bottlenecks 

“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 

 in Washington, DC. The organization seeks to address unmet needs for the community through advocacy directed at various organizations, including regulatory agencies.

, 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.

The vice president of Public Policy & Advocacy of the Muscular Dystrophy Association discussed the organization’s goals and strategies in educating regulatory decision-makers. 

Paul Melmeyer

Articles

Paul Melmeyer on Addressing Gene Therapy Regulatory Bottlenecks

Paul Melmeyer on Regulatory Advocacy for Patients With Neuromuscular Diseases