ACT Brief: Data Strategy, AI Integration, and Rare Disease Milestones Drive Clinical Innovation

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.

• We begin with a look at the persistent challenges slowing patient recruitment and extending trial timelines. In a new video interview with Applied Clinical Trials, Matt Holms, vice president of sales at Citeline, explained how site performance variability, investigator scarcity, and rising protocol complexity continue to stall enrollment. Holms noted that more than 85% of studies undergo major amendments—often without improving recruitment outcomes—and that the shortage of qualified principal investigators has reached a critical point. He emphasized that only 4% of US healthcare providers participate in clinical research. To address these barriers, Holms called for broader use of AI and tokenization to improve patient–protocol matching and for sponsors to adopt data-driven oversight models that enhance feasibility and expand patient access.
• Next, the FDA has approved UCB’s Kygevi—the first treatment for thymidine kinase 2 deficiency (TK2d)—based on a multi-study clinical program demonstrating significant survival and motor gains. In the pivotal Phase II study, Kygevi reduced the risk of death by up to 94% in patients with early symptom onset and enabled 75% of participants to regain at least one lost motor milestone. Sixteen percent of patients reduced or discontinued ventilatory support after therapy. No major safety concerns were reported, and the most common adverse event was mild gastrointestinal discomfort. This approval marks a breakthrough for patients with TK2 deficiency, a rare mitochondrial disease with no previous FDA-approved treatment options.
• Finally, in a recent video interview with our sister publication, Pharm Exec, Raj Indupuri, CEO of eClinical Solutions, discussed why robust data strategy and infrastructure are essential to unlocking AI’s full potential in pharma. Indupuri emphasized that without strong, governed data foundations, AI adoption at scale cannot succeed. He noted that while the technology’s accessibility has improved, responsible AI still depends on establishing proper controls, safety practices, and data guardrails to ensure that generated insights are both accurate and actionable.

Join us again tomorrow for more insights shaping the future of clinical operations and drug development. Thanks for listening.