ACT Brief: Patient-Site Trust Barriers, Rare Disease Evidence Without Registry, and RWD Enrollment Accuracy

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

• In a new Q&A, Andrea Valente, CEO of uMotif, discussed why making technology easier to use is not the same as making patient-site interaction more effective. The real challenge is designing systems around human relationships, educating patients and sites together on how to participate effectively, and mapping the full patient journey from beginning to end as a design input rather than an afterthought.
• In a new contributed article, Susanna Lövdahl, PhD, examined how rare disease evidence engineering begins by defining intended regulatory use upfront, rather than collecting data and reconciling gaps later. When no registry exists, sponsors must design cohort definitions, endpoint selection, harmonization and data provenance as part of the study plan, and build multinational datasets across eight or more countries with submission-grade traceability and independent quality control from the start.
• In a new Q&A from Pharmaceutical Executive, Jen Lamppa, SVP of commercial strategy at Inovalon, explained why self-reported data at enrollment is costly due to collection burden and frequent inaccuracies that drive mis-enrollment and audit risk. Clinical and administrative data layers provide real-time validation of diagnoses, medications and health history before enrollment, while AI accelerates extraction from unstructured clinical notes and imaging to prevent patient mismatches without the manual burden that does not scale.

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