Traditional randomized controlled trials often fall short in rare diseases and small population settings. External Control Arms (ECAs) are increasingly used to fill this gap, offering a way to supplement evidence. This webinar will demonstrate how Real-World Data (RWD) can be transformed into regulatory-ready evidence to support ECA strategies.

Key Topics

• Walk through a real-world case study in a rare disease setting, demonstrating the process of building an ECA
• Overview of possible sources for ECA construction
• Integrating RWD with historical data by applying Propensity Score Matching (PSM)
• Addressing common challenges such as heterogeneity, missingness, and standardization
• Key considerations for ensuring data quality, traceability, and fitness for purpose
• Align with CDISC standards such as SDTM and ADaM
• Practical approaches to achieve compliance with regulatory expectations
• Best practices from our example case study for data integration and conformance, to support submission readiness

Register now to gain practical insights and experience-based strategies for implementing ECAs with Real-World Data.

Speakers

Gautham Selvaraj, Associate Director, Statistical Programming, PBS

Ralf Koelbach, Associate Biostatistics Director, PBS