TransCelerate BioPharma announced the release of a comprehensive summary report from a collaborative tabletop exercise with the U.S. Food and Drug Administration's CDER Center for Clinical Trial Innovation (C3TI), marking a significant step toward scaling pragmatic clinical trials in routine healthcare settings. The December 2024 exercise brought together more than 30 pharmaceutical R&D leaders from TransCelerate member companies and representatives from eight FDA offices to identify actionable opportunities for implementing pragmatic trial elements at scale.
Advancing Clinical Research Integration
The collaborative effort focused on enabling more research to be conducted at the point of care through pragmatic trials, which offer simplified designs, leverage routine care settings, and reach broader, more representative patient populations. According to Rob DiCicco, Vice President of Portfolio Management at TransCelerate, "Collaborative efforts like this help surface practical considerations for embedding clinical research into routine care. It also highlights the value of FDA's Streamlined Trials Embedded in clinical Practice (STEP) initiative in supporting innovative trial designs that can improve patient access and generate reliable evidence from real-world care settings."
The exercise utilized a hypothetical case study evaluating a new indication for a post-approval medication, incorporating several pragmatic elements including simplified data collection, broad eligibility criteria, and integration with electronic health records (EHRs). This approach demonstrates the potential for pragmatic trials to address public health challenges and areas of significant unmet clinical need.
Key Implementation Considerations
The tabletop exercise identified four critical themes for successful pragmatic trial implementation:
Design and Oversight Frameworks: Participants emphasized the value of hybrid trial designs and fit-for-purpose investigator and third-party oversight frameworks. Early feasibility assessments were highlighted as essential for ensuring data readiness and regulatory alignment before trial initiation.
Data Integrity and Endpoint Optimization: The session explored opportunities to improve data quality by integrating EHR data with quality-by-design (QbD) approaches and clarified endpoint definitions. This integration represents a crucial step toward leveraging real-world data sources effectively.
Cross-Agency Collaboration: Discussions revealed the need for aligned expectations regarding endpoint selection, adjudication, blinding, and inspection priorities across multiple FDA offices, highlighting the complexity of regulatory coordination for innovative trial designs.
Operational Efficiency and Privacy Protection: The exercise explored computational methods to ensure data integrity while preserving patient privacy during regulatory inspections, addressing a key concern for healthcare systems and patients.
Resource Development and Industry Impact
Alongside the tabletop exercise findings, TransCelerate released "Getting Started with Embedded Pragmatic Trials: A Resource Guide," designed to build understanding of embedded pragmatic trials and provide practical implementation guidance. The guide addresses key pragmatic elements such as simplified data collection, broad eligibility criteria, and EHR integration.
Kevin Bugin, Head of Global Regulatory Policy at Amgen and executive sponsor of TransCelerate's Embedded Pragmatic Trials initiative, emphasized the transformative potential: "Pragmatic trials have the potential to transform how clinical research is conducted – bringing trials closer to where care is delivered and expanding patient access to clinical trials. This exercise represents a critical step toward enabling this vision, and we are grateful for the FDA's partnership and engagement."
Future Collaboration Framework
TransCelerate outlined plans for continued collaboration with global regulators, sponsors, and stakeholders to advance pragmatic trial adoption. The organization will focus on advancing understanding through real-world case studies, tools, and shared learnings while clarifying operational distinctions between pragmatic and traditional trial models. Additionally, efforts will identify infrastructure-ready geographies to support implementation at scale.
The initiative represents part of TransCelerate's broader mission as a nonprofit organization with 20 member companies focused on transforming connectivity, enabling information sharing and reuse, and driving innovative trial designs. The organization aims to simplify clinical trials and help bring new treatments to patients faster, safer, and more efficiently through collaborative industry efforts.
