Brandon Michael Henry, MD is a physician-scientist and Medical Director of the Cell and Gene Therapy Center of Excellence at IQVIA, where he leads medical strategy for rare disease programs across cell, gene, and RNA therapeutic modalities. He is internationally recognized for his contributions to rare disease research and clinical development, with over 400 peer-reviewed publications and recognition among the world’s top 2% of scientists (Stanford University, 2021–2024) and Clarivate’s Highly Cited Researchers list (2024). Dr. Henry brings 12+ years of experience spanning basic, translational, and clinical research across cell, gene, and RNA therapy, with deep expertise in biomarker-driven trials, adaptive trial design, and mitigating risks of immunogenicity. His work focuses on accelerating the development of novel therapeutics for rare diseases through innovative, data-driven approaches to trial design and execution. He serves as a scientific and medical advisor to both internal and sponsor teams and is an appointed member of multiple international professional body committees and journal editorial boards. At the World Orphan Drug Congress USA 2025, Dr. Henry will share insights on clinical strategy and development innovation for long term follow-up in rare diseases, drawing from his experience at the intersection of cutting-edge science and global trial execution.
We will discuss unique needs and challenges associated with the design and delivery of LTFU studies in rare disease. We will focus on how we can address these challenges by bringing together digital tools, with behavioral science, with instrument science / Patient Experience Data, and decentralized operations to drive long-term engagement and retention. We will review case studies, which provide lessons learned, and illuminate the possibilities of what can be achieved when you bring together these unique disciplines and deploy them through digital tools
Sposnored by IQVIA.