ACT Brief: Bayesian Designs Gain Ground, Data Priorities Take Shape, and Rare Disease Strategy Expands

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

• In a new contributor article tied to Rare Disease Day, Billy Amzal of Phastar highlights how Bayesian trial designs are emerging as a practical alternative to traditional randomized controlled trials in small populations. With new draft guidance from the FDA, these approaches are gaining regulatory momentum by enabling more flexible designs that incorporate prior data and reduce sample size constraints.
• In the final part of his interview, Jonathan Andrus, co-CEO of CRIO, outlined key priorities for 2026, emphasizing the need to design source systems alongside downstream environments and strengthen governance across the data lifecycle. He also pointed to growing use of AI to automate elements of study design and workflow configuration, helping teams improve efficiency while reducing site burden.
• In an interview with Pharmaceutical Technology, Elisabeth Gardiner of Tevard Biosciences explained that rare disease research continues to face scientific and financial challenges, but also offers critical insights into broader drug discovery. Approaches that can target multiple diseases tied to shared genetic mechanisms may help expand impact while improving the long-term viability of development programs.

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