ACT Brief: Trial Transparency and Synthetic Controls, Readiness as Leading Risk Indicator, FDA Streamlines Biosimilar Pathway

This is the Applied Clinical Trials Brief—your fast track to the latest insights shaping clinical operations and drug development.

• In part two of his video interview, Marc Buyse, founder and CEO of IDDI, addressed operational gaps undermining data quality and reliability. Complex designs require transparency and explainability; synthetic controls and real-world evidence demand clear documentation of how comparator groups were constructed, as unexplained differences between arms cannot be attributed to treatment alone. Bayesian approaches add genuine value in specific cases—such as pediatric extrapolation from adult data—but each application requires weighing advantages against added complexity.
• In a new column, ACT EAB member Brian S. McGowan, PhD, Chief Learning Officer at ArcheMedX, examined why risk-based monitoring relies too heavily on lagging indicators that arrive after problems surface. Measuring site readiness during training—assessing actual performance capability, not just completion—provides a leading indicator weeks before enrollment. Sites demonstrating high readiness become fast-track candidates; those with low readiness receive targeted support or remediation, allowing trial teams to prevent deviations rather than react to them.
• The FDA issued draft guidance streamlining biosimilar development by eliminating certain clinical pharmacokinetic studies when scientifically justified. The change could save developers up to $20 million per program and builds on prior efforts to reduce unnecessary comparative efficacy studies, signaling sustained FDA commitment to lowering regulatory burden without compromising safety or efficacy standards.

That's all for today's ACT Brief. Join us next time for more updates shaping clinical operations and drug development. Thanks for listening.