Rachel Smith | Vice President, Rare and Genetic Diseases
Parexel International LLC

Rachel has more than 10 years of experience in all development phases of rare disease and cell and gene therapy clinical trials. Rachel is an ultra-rare disease subject matter expert with oversight of 30+ orphan programs in APAC, Europe and North America. She is a specialist in clinical development strategy, novel and adaptive trial design, decentralized trials and creative solutions for rare disease and products with a non-classical route to market. Prior to joining Parexel, Rachel was VP European Clinical Operations, Global Head of Cell & Gene Center of Excellence and Portfolio Director in Rare Disease at Veristat LLC.

Appearances:

Day 1 - World Orphan Drug Congress USA 2026 @ 10:10

Future outlook of the US rare disease regulatory landscape – What can we expect?

Rare disease drug development has entered a new era of possibility. Advances in genomics, AI-enabled diagnostics, real-world data, and innovative trial designs are unlocking therapies for some of the most complex and least understood conditions. Yet this progress which is hard won in the face of extremely small, geographically dispersed patient populations and inconsistent standards of care depends on unprecedented global coordination. The World Health Organization’s recognition of rare diseases as a global research priority underscores the need for harmonized approaches, while recent initiatives from the U.S. Food and Drug Administration, including the Rare Disease Evidence Principles and Rare Disease Innovation Hub, reflect renewed regulatory focus. In an increasingly complex global environment, the question is not whether innovation is possible but whether the global research community can stay aligned enough to sustain its momentum. This panel will explore how regulators, patient advocates, and industry leaders can drive global alignment across regulatory pathways, responsible use of real-world data, and next-generation trial methodologies to ensure rare disease progress continues, faster, smarter, and more equitably across borders.