EL-PFDD Reports

On June 20, 2023, the SADS Foundation hosted an Externally-Led Patient-Focused Drug Development (ELPFDD) meeting to share with officials at U.S. Food and Drug Administration (FDA) and other stakeholders (including industry and research institutions) the perspectives of people living with ARVC (Arrhythmogenic Right Ventricular Cardiomyopathy), its impact on their daily lives, and their expectations and priorities for current and future treatments for ARVC.

The subsequent “Voice of the Patient: ARVC ” report is a compilation of the testimonies and survey responses of individuals affected by ARVC from all around the world and is the first-ever report of its kind for inherited heart conditions.

The Adjunct Scientific Session Report is a summary of the Scientific Session held after the EL-PFDD for ARVC, and summarizes the findings of expert clinicians and researchers about the unmet needs, gaps in knowledge, and areas where research can have the most significant impact on the quality of life of those with ARVC.

The SADS Foundation would like to gratefully acknowledge the many individuals who participated in this meeting, including caregivers, affected individuals, family members, representatives from industry, clinicians and researchers and leaders from the FDA. The 2023 ARVC PFDD and the Voice of the Patient report represent important milestones for our community; it is our hope that this effort and Voice of the Patient Report will inform and influence both drug development and regulatory efforts so that the significant unmet therapeutic needs of people living with ARVC is better understood.