TransCelerate and FDA Collaborate to Advance Use of Pragmatic Elements in Clinical Trials

New Summary Report and Resource Guide Highlight Opportunities to Integrate Research into Routine Care and Enable Broader Trial Participation

PHILADELPHIA, Aug. 19, 2025 /PRNewswire/ -- TransCelerate BioPharma (TransCelerate), a nonprofit organization dedicated to improving the health of people around the world by accelerating and simplifying clinical research, today announced the release of a summary report from a tabletop exercise conducted with the U.S. Food and Drug Administration's (FDA) CDER Center for Clinical Trial Innovation (C3TI), along with the newly published resource, Getting Started with Embedded Pragmatic Trials: A Resource Guide.

Held in December 2024, the exercise convened more than 30 pharmaceutical R&D leaders from TransCelerate member companies and eight FDA offices to identify actionable opportunities to scale the use of pragmatic elements in clinical trials to enable more research to be conducted at the point of care. Pragmatic trials offer simplified designs, leverage routine care settings, and reach broader, more representative patient populations—making them well-suited to address public health challenges and areas of significant unmet clinical need.

"Collaborative efforts like this help surface practical considerations for embedding clinical research into routine care," said Rob DiCicco, Vice President of Portfolio Management at TransCelerate. "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."

TransCelerate's Embedded Pragmatic Trials initiative aims to accelerate the adoption of these trial designs by building awareness, engaging in productive dialogue with regulatory authorities and other stakeholders, and offering practical tools to guide implementation.

"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," said Kevin Bugin, Head of Global Regulatory Policy at Amgen and executive sponsor of TransCelerate's Embedded Pragmatic Trials initiative. "This exercise represents a critical step toward enabling this vision, and we are grateful for the FDA's partnership and engagement." 

As part of the informal, non-binding exercise, TransCelerate presented a hypothetical case study evaluating a new indication for a post-approval medication. The clinical trial design incorporated several pragmatic elements, including simplified data collection, broad eligibility criteria, and integration with electronic health records (EHRs).

Key themes from the session include:

• Design and Oversight Considerations: The value of hybrid trial designs, fit-for-purpose investigator and third-party oversight frameworks, and early feasibility assessments to ensure data readiness and regulatory alignment.
• Data Integrity and Endpoint Alignment: Opportunities to improve data quality by integrating EHR data with thoughtful quality-by-design (QbD) approaches and clarified endpoint definitions.
• Value of Broad Agency Collaboration: The need for aligned expectations regarding endpoint selection, adjudication, blinding and inspection priorities across multiple FDA offices.
• Operational Efficiency and Privacy: Exploration of computational methods to ensure data integrity while preserving patient privacy during inspections.

In parallel with the tabletop exercise, TransCelerate has released Getting Started with Embedded Pragmatic Trials: A Resource Guide, a robust tool designed to build understanding of embedded pragmatic trials and provide practical guidance for successful implementation. The guide identifies resources to help stakeholders navigate the design and implementation of trials that incorporate pragmatic elements such as simplified data collection, broad eligibility criteria, and EHR integration.

Looking Ahead
TransCelerate will continue to collaborate with global regulators, sponsors, and stakeholders to:

• Advance understanding and adoption of pragmatic trials through real-world case studies, tools, and shared learnings.
• Clarify operational distinctions between pragmatic and traditional trial models.
• Identify infrastructure-ready geographies to support implementation at scale.

The full report, Enabling the Implementation of Pragmatic Elements at Scale: Summary Report from an FDA and TransCelerate Tabletop Exercise, is available here: https://www.transceleratebiopharmainc.com/wp-content/uploads/2025/06/FDA-Tabletop-Public-Summary.pdf

To learn more about TransCelerate's Embedded Pragmatic Trials Initiative, visit: https://www.transceleratebiopharmainc.com/initiatives/embedded-pragmatic-clinical-trials/

About TransCelerate BioPharma Inc.
TransCelerate BioPharma (TransCelerate) is a nonprofit organization that fosters collaboration across the global biopharmaceutical research and development community to simplify clinical trials and help bring new treatments to patients faster, safer, and more efficiently.
Headquartered in the Philadelphia area, TransCelerate has 20 member companies and a robust portfolio of initiatives focused on transforming connectivity, enabling information sharing and reuse, and driving innovative trial designs. For more information, please visit transceleratebiopharmainc.com.

SOURCE TransCelerate BioPharma