Event Update: Patient-Focused Duchenne Muscular Dystrophy Meeting

On March 5th, Parent Project Muscular Dystrophy hosted a new kind of meeting at their annual conference. Parent Project Muscular Dystrophy (PPMD) held the meeting in Washington DC with a focus on patients and community. Keep reading to learn more about this event, or follow the original story here.

PPMD functions as a nonprofit organization with the goal of ending muscular dystrophy. Specifically, the group in many ways spearheads the battle to end Duchenne muscular dystrophy. Every year, PPMD holds their annual Advocacy Conference. This year, they added the Duchenne Patient-Focused Compass Meeting: Navigating our Pathway Forward event to that conference.

The Duchenne Patient-Focused Meeting consisted of a variety of panels and presentations. Many members of the Duchenne community – including both patients and caregivers – attended and shared perspectives. PPMD created the event as an invitation to “explore ‘living with Duchenne’.”

Topics included clinical trials, experiences in and with therapy, and access to services. Community members also shared information regarding support and recognized treatments for Duchenne muscular dystrophy.

The idea for the meeting began after PPMD attended a discussion with key personnel from the Food and Drug Administration (FDA). They sought to create a meeting that would utilize the FDA’s “Patient Focused Drug Development (PFDD) Workshop” format. Members from many other federal health agencies (such as NIH, and CDC) were also invited.

Additionally, PPMD invited all of the Duchenne community foundation partners to join the Community Advisory Board (CAB) during the Duchenne Patient-Focused Meeting. The CAB currently consists of 14 organizations.

PPMD published additional information in advance of the meeting in a white paper which can be read here.

PPMD site several developments stemming from the FDA as reasons for hosting this type of meeting and hosting it now. One of these reasons involves the passage of PDUFA V and the reauthorization of the Patient-Focused Drug Development Initiative. The FDA held a series of meetings with various disease communities, all following the PFDD framework and gained a great deal of information and understanding through this practice.

Another influence came from PPMD’s Duchenne Drug Development Roundtable. This group discussed the research and “regulatory learnings” PPMD endeavored in and encountered in the previous year.

Finally, the FDA published an industry guidance document that seemed to make the timing of the new Patient-Focused meeting all the more important. That document is made available here.

In an effort to ensure as many patients and supporters as possible were involved, PPMD also made the conference available via live stream.


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