ACT Brief: AI in Clinical Research, FDA Gene Therapy Approval, and High-Performing Hospital Partnerships

This is the Applied Clinical Trials Brief—your fast track to the latest insights in clinical research operations. In under three minutes, we’ll recap top stories, highlight expert perspectives, and keep you current on what’s moving the industry.

• A new feature article from Norman Goldfarb explores how AI agents, AI teammates, and integrated intelligence are poised to reshape site-level operations in clinical research. Administrative workflows—including site closeout, document review, and task tracking—are prime candidates for partial or full automation, while humans retain responsibility for judgment-intensive and relationship-driven tasks. Investigative sites that leverage AI effectively may reduce timelines, improve consistency, and lower operational costs, offering more value to sponsors and freeing study staff to focus on higher-value clinical work.
• The FDA has approved Itvisma, the first gene replacement therapy for adolescents, teens, and adults with spinal muscular atrophy (SMA) who carry SMN1 mutations. Approval was based on Phase III STEER and Phase IIIb STRENGTH studies demonstrating motor function improvements and a favorable safety profile. This milestone expands access to gene therapy for a broader SMA population and signals opportunities for clinical operations teams to engage adult neurology and community practice sites in upcoming late-stage and post-marketing trials.
• Another new feature article highlights strategies for pharmaceutical companies to partner effectively with hospitals to grow clinical trial programs. By addressing operational, financial, and organizational barriers—such as long start-up timelines, high staff turnover, and misaligned research support—pharma can work with hospital leadership to implement enterprise-wide improvements. Case studies, including Henry Ford Health, show that structured partnerships, executive engagement, and optimized research infrastructure can accelerate accrual, expand study capacity, and strengthen hospital-based clinical research operations.

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