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Patient-Focused Drug Development in Rare Diseases: Applying Guidances and Case Studies from the Field
Session Chair(s)
K. Kimberly McCleary
Founder and Chief Executive Officer
The Kith Collective, LLC, United States
FDA guidances from the three product centers (CDER, CBER and CDRH) provide direction for how to involve patients and caregivers in medical product development and to integrate their perspectives and preferences into across the lifecycle. A team of subject matter experts from life science companies and patient advocacy organizations convened by the EveryLife Foundation and partners has workshopped the guidances to apply them to stages of product development of innovative therapies for rare diseases. This session features learnings from this activity, as well as case studies from the field.
Learning Objective : Describe FDA guidances that can be applied to patient-focused medical product development; Identify how these guidances can be applied across the development lifecycle for an innovative drug, gene/cell therapy, or medical device intended to treat a rare disease; Discuss using case studies of patient involvement in therapy development for rare diseases.
Speaker(s)
Project Leader
Annie Kennedy
EveryLife Foundation for Rare Diseases, United States
Chief of Policy, Advocacy, and Patient Engagement
Steering Committee Member
Pujita Vaidya, MPH
Novartis Pharmaceuticals Corporation, United States
Director, US Patient Engagement
Panelist
Kristin Van Goor, PhD
Vertex Pharmaceuticals, United States
Regulatory Policy and Intelligence, Global Regulatory Affairs
Patient-Focused Drug Development in Rare Diseases: Applying Guidances and Case Studies from the Field
Jill Jarecki, PhD
Cure SMA, United States
Chief Scientific Officer
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