ACT Brief: EHR-to-EDC Connectivity, FDA’s Rare Disease Framework, and Barriers to Oncology RWE

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

• In a contributed article, Nick Frenzer, general manager of Veeva site solutions, argues that real-time trials depend on scaled eSource-EDC-EHR integration, but progress stalls because competing data standards lack regulatory enforcement. Frenzer points to USCDI’s mandated FHIR connections as a model for industry alignment, and notes that 67% of patient consents are still completed on paper. He also flags unresolved questions about who owns de-anonymized data under current privacy frameworks.
• The FDA launched its Rare Disease Framework Initiative, led by the agency’s Rare Disease Innovation Hub, with a public meeting set for October 28. The effort addresses a gap in which only about 5% of recognized rare diseases have an approved treatment, and it aims to make regulatory flexibility applied more consistently across review divisions. Planned deliverables include potential regulatory revisions and updated guidance on endpoints, statistical approaches, and trial design.
• In the final part of a video interview, Saamir Pasha and Carole Berini, PhD, of Ontada, identify three persistent barriers to consistent oncology real-world evidence. These are inconsistent data capture for key clinical concepts, structural data limitations that modeling alone cannot fix, and the need for earned generalizability through deliberate multi-site validation rather than direct model export across settings.

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