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Innovative Patient-Focused Models for Accelerating Rare Drug Development

June 17, 2025
Breakout Session
Orphan and Rare Disease
We are witnessing a revolution in rare disease drug development. Traditional models fall short due to small patient populations and limited disease understanding. However, regulatory agencies are now embracing innovative, patient-focused approaches. Collaborations between patient groups, tech companies, philanthropy, and biopharma are driving this change. Rare diseases are becoming the testing ground for a new era of precision medicine development, potentially transforming the entire pharmaceutical landscape. This session will highlight novel models and successful case studies where patient communities, researchers, technology companies, philanthropies, and the biopharma industry have collaborated to fast-track translational research and therapeutic development. Attendees will gain practical strategies for engaging patient communities as co-creators in research and utilizing novel technology platforms to transform the traditional drug development process to better meet the needs of rare disease communities.
Moderator
Cara Altimus, PhD
Managing Director, Science Philanthropy Accelerator for Research and Collaboration
Milken Institute
Speakers
Nasha Fitter, MBA
Co-Founder & Chief Business Officer
Citizen Health
Sunitha Malepati, JD
Founder & CEO/VP
Buffalo Initiative/CACNA1A Foundation
Justin West, MD
Co-Founder, President & Director of Clinical Medicine
KCNT1 Epilepsy Foundation

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