Accelerating Rare Disease Access Through Global Alignment

Rare disease innovation is advancing rapidly, but progress can stall without global alignment. This also reinforces the central theme of the panel before highlighting the barriers to faster patient access. Yet scientific progress alone is not enough to accelerate patient access. Sponsors continue to face fragmented regulatory expectations, inconsistent real-world data standards, limited natural history data and development strategies that may be accepted in one region but challenged in another.

Rare disease drug development has entered an extraordinary period of possibility, fueled by advances in genomics, AI-enabled diagnostics, real-world data and innovative trial designs. Yet scientific innovation alone is not enough. Success depends on global alignment across regulatory expectations, real-world data standards and development strategies. By fostering collaboration among regulators, sponsors, researchers and patient communities, the industry can reduce duplication, streamline development pathways, generate evidence that supports broader acceptance across markets and ultimately bring new therapies to patients sooner.

Learn how global alignment, harmonized real-world evidence standards and coordinated regulatory approaches can help sponsors reduce development friction, accelerate patient access and bring life-changing therapies to patients sooner.