Patient-Focused Drug Development (PFDD) meetings are one of the most powerful advocacy tools available to rare disease communities—but most patients have never heard of them. In this episode of Energy in Action, host Marcy Young sits down with industry expert Marina Kolocha to unpack what PFDD meetings are, why they matter, and how they can help drive real progress in mitochondrial disease research and treatment.
Marina shares insights from her experience moderating over 40 PFDD meetings and breaks down the goals, structure, and long-term impact of these events. Together, they discuss the first-ever PFDD meeting focused on MILAS, scheduled for February 10, 2026, and how patients and caregivers can participate. From voicing daily challenges to shaping future drug development, this conversation offers a rare behind-the-scenes look at a process designed to amplify patient stories at the highest level.
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