Both the FDA and EMA have made statements that support the concept of using real world evidence (RWE) for regulatory decision-making. Historical control arms represent a way to take a safe and effective leap forward into real-world evidence. But there is much heterogeneity in the lexicon (eg. external, historical, synthetic) and the tools used (eg. natural history cohorts, disease registries, placebo arms of clinical trials, simulated cohorts, artificial intelligence). During this presentation, the speakers will present a case for a common language and the need for a robust toolkit that supports planning and design of external control arms.
- The context for external control arms – regulatory/HTA
- A discussion of key terms related to external control arms and available tools
- Key design considerations for external control arms and suggestions to harmonize/clarify language and methods
- Demonstrate a “toolkit” approach with the use of case studies