In today’s ACT Brief, we examine what it will take to scale EHR-to-EDC connections for real-time trials, how the FDA is reimagining rare disease drug development, and why oncology real-world evidence still faces persistent data barriers.
News|Podcasts|September 21, 2026
ACT Brief: Patient-Site Trust and AI Role, Platform Approach to Scale, Gene Therapy for Pediatric Disease
Author(s)Andy Studna, Senior Editor
In today's ACT Brief, we examine what patient-site relationships need most at DPHARM, how platform-based AI scales across workflows, and FDA's approval of a gene therapy for a rare childhood syndrome.
This is the Applied Clinical Trials Brief—your fast track to the latest insights shaping clinical operations and drug development.
- In a
video interview prior to DPHARM 2026 , Andrea Valente, CEO of uMotif, shares what she expects to be at the center of the conversation, from building trust between patients and sites to where AI may and may not have a role in that relationship. Understanding that distinction—where human connection matters most versus where technology can help—is critical to genuine patient engagement. - In a
contributed article , Jason Makuch of IQVIA examined how only one in five life sciences organizations are scaling AI and seeing measurable value, because disconnected AI tools create silos instead of reducing them. A platform-based approach connects AI across workflows and governance, bringing AI into the tools employees already use rather than requiring them to switch between systems. - The
FDA approved Fayuvi as the first therapy for Sanfilippo syndrome type A , a rare genetic disease that progressively damages children's brains and nervous systems. Administered as a single infusion using gene therapy to deliver a missing enzyme, Fayuvi-treated patients in clinical trials maintained or improved cognitive function compared to an untreated historical comparison group, diverging from the expected disease course of decline during early childhood.
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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