PPMD’s SVP of Legislation and Public Policy, Annie Kennedy, joined a distinguished panel discussion convened by FasterCures about the impact of Patient Focused Drug Development on the Duchenne community and how it has become a ‘road map’ for PPMD to help develop to tools for patient engagement.
PFDD creates a framework for patient organizations to formally engage with the FDA in a way that wasn’t possible before. As a result, PPMD – with the involvement of our incredible Duchenne community – has:
• Conducted five patient-focused benefit-risk studies
• Published two whitepapers on patient engagement
• Established a patient registr
• Created patient-reported outcomes that reflect the needs of the patient community
• Written a draft guidance for Duchenne muscular dystrophy that the FDA later adopted
The other panelists included a representative from FDA’s Office of Strategic Programs, CDER and a member of Faster Cures leadership and policy team.
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