In today's ACT Brief, we examine how AI enables sponsors to pressure-test protocols before enrollment, HHS's multi-initiative effort to accelerate trial design and execution, and FDA approval expanding heart disease therapy to adolescents.
News|Podcasts|March 3, 2026
ACT Brief: RWD Integration Accelerates, FDA Advances Ultra-Rare Therapies, and Collaboration Shapes the Next Decade
Author(s)Andy Studna, Senior Editor
In today’s ACT Brief, we examine how the Verana Health-COTA merger is evolving real-world data use, the FDA’s new framework for individualized ultra-rare disease therapies, and what a decade of cross-industry collaboration reveals about the future of clinical development.
This is the Applied Clinical Trials Brief—your fast track to the latest insights shaping clinical operations and drug development.
- In a new
episode of the Applied Clinical Trials Podcast, C.K. Wang, MD, chief medical officer at COTA, and Sujay Jadhav, CEO of Verana Health, discuss how their merger is transforming real-world data from a retrospective tool into a prospective driver of trial design and execution. With access to 95 million patient records and 25,000 providers, they explain how machine learning is improving patient identification, trial feasibility, and regulatory-grade evidence generation, with implications for rare disease and pediatric research. - The FDA has issued draft
guidance introducing the Plausible Mechanism Framework to support individualized therapies for ultra-rare diseases. The approach allows sponsors to demonstrate effectiveness using mechanism-based evidence when conventional trials aren’t feasible. FDA leaders highlight regulatory flexibility and streamlined pathways while maintaining scientific rigor for therapies targeting specific genetic, cellular, or molecular abnormalities. - A decade of
collaboration at TransCelerate BioPharma Inc. shows how shared problem-solving can reshape clinical development. In an op-ed on Pharmaceutical Executive, founding member Andy Lee and CEO Janice Chang emphasize aligning on common processes, standardizing data structures, and fostering a culture that absorbs change. These practices have enabled more patient-centric trial designs, smoother operations, and faster adaptation to emerging challenges across the industry.
That’s all for today’s ACT Brief. Join us tomorrow for more updates shaping clinical operations and drug development. Thanks for listening.
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