ACT Brief: Master Protocol Design Considerations, Early Predictive Data Integration, and Real-World Evidence as Regulatory Standard

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

• In part three of her video interview, Mwango Kashoki, MD, MPH, senior vice president and global head of regulatory strategy at Parexel, examined biological, dosing, and population-level considerations sponsors must get right from the start when using basket and master protocol designs. Success depends on thoughtful target selection, dose optimization across conditions, and realistic patient population assumptions before enrollment begins.
• In a new Q&A, Jenna DiRito, PhD, COO and co-founder of Revalia Bio, discussed why traditional go/no-go models are breaking down and how human-relevant data generated earlier in development reduces uncertainty. Teams are answering questions about mechanism, safety, and biodistribution in human systems before clinical trials rather than waiting for late-stage failures to expose gaps.
• In a new contributed article from Pharmaceutical Executive, FDA's shift toward single-trial approvals supported by real-world evidence represents recognition that AI analytics can now convert fragmented RWD into regulatory-grade evidence. The transition requires building organizational capabilities to unify disparate data sources and apply rigorous study design before approvals, moving RWD integration from late-stage consideration to foundational development planning.

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