Patients and caregivers have a unique and personal perspective on rare disease. For a long time, this valuable perspective was not a primary focus within research and drug development and regulatory review. From 2012 through 2017, the Food and Drug Administration (FDA) held 24 disease-specific Patient-Focused Drug Development (PFDD) meetings, including several for rare disease, to ensure patient perspectives are included in regulatory efforts. Patient organizations are now encouraged to organize meetings using the PFDD process as a model. In this session, leaders from patient organizations who are planning or who have conducted PFDD meetings will lead a discussion on the process, challenges and benefits of engaging in this effort to help ensure the understanding of the patients’ disease experience and their treatment priorities are included in drug development and review.
Speaker: Joy Aldrich, Charcot-Marie-Tooth (CMT) Advocacy Director, Hereditary Neuropathy Foundation
Speaker: Allison Moore, Founder and Chief Executive Officer, Hereditary Neuropathy Foundation (HNF)
Speaker: Jill Jarecki, PhD, Chief Scientific Officer, Cure SMA
Day 1, Track 2