Advanced Clinical Trials and Clinical Research

Synthetic Control Arms

Synthetic control arms (SCAs) offer an innovative approach to reduce or eliminate the need for traditional control groups, especially in rare diseases or high-risk indications. This session focuses on constructing and validating SCAs using real-world data (RWD), historical trial datasets, and statistical matching techniques like propensity scores and Bayesian models. Attendees will explore the advantages of SCAs, including enhanced patient recruitment, ethical considerations, and regulatory receptivity. The session highlights practical steps for identifying quality RWD sources, assessing comparability, and ensuring transparency in analysis. Discussions will include recent FDA/EMA decisions involving SCAs, challenges in data interoperability, and statistical robustness. Real-life use cases from oncology, gene therapy, and pediatrics will be reviewed. By the end of this session, participants will understand how synthetic controls can complement or replace traditional comparators while maintaining scientific validity and regulatory compliance.