Clinical trials for rare diseases are reaching a critical inflection point as traditional designs struggle to meet the demands for speed, efficiency, and patient access. External Control Arms (ECAs) are gaining traction as sponsors look to leverage real-world data to optimize evidence generation. Yet key questions remain: What does it truly take for an ECA to gain regulatory acceptance, and how are expectations evolving?

This webinar examines how regulatory bodies, including the FDA, EMA, and MHRA are shaping frameworks for ECAs in rare disease programs — and what sponsors must demonstrate to align with emerging standards. Our experts will draw on recent approvals, regulatory interactions, and real-world implementation experience to offer a practical roadmap for adoption.

Key Topics

• How regulatory expectations for ECAs in rare disease trials are evolving across FDA, EMA, and MHRA
• Evidence requirements and methodological rigor needed for credibility and acceptance
• Key design, data quality, and statistical considerations for successful implementation
• Lessons from recent submissions and real-world programs using ECAs
• How to integrate ECAs into ongoing development strategies without delaying timelines

Watch the on-demand webinar to understand where the regulatory bar is moving, what constitutes credible evidence, and how to position your strategy for the next era of rare-disease clinical development.