Rewriting the Rules: Scaling Innovation for Rare Diseases

Historically, the rare disease community has met persistent barriers when forced to follow clinical and regulatory pathways designed for common diseases. Such barriers are reinforced by a fractured funding environment and a challenging investment case. With more than 10,000 rare diseases identified, applying one-disease-at-a-time models is prohibitively slow, costly, and ultimately unsustainable. Breakthroughs in genetic therapies and AI are now enabling a fundamental reimagining of how rare diseases are diagnosed, studied, and treated. Panelists will examine how innovators are rewriting the rules—from AI-powered diagnostics and drug-repurposing screens to platform-based therapies and batch regulatory-approval models that address multiple diseases at once. Together, these approaches will facilitate more rapid, cost-efficient, and scalable progress for the global rare disease community.