TransCelerate and FDA Collaborate to Advance Use of Pragmatic Elements in Clinical Trials
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PHILADELPHIA, Aug. 19, 2025 ~ TransCelerate BioPharma, a nonprofit organization dedicated to improving global health through streamlined clinical research, has released a summary report from a recent tabletop exercise conducted in collaboration with the U.S. Food and Drug Administration's (FDA) CDER Center for Clinical Trial Innovation (C3TI). The organization also announced the publication of a new resource guide titled "Getting Started with Embedded Pragmatic Trials."

The tabletop exercise, held in December 2024, brought together over 30 leaders from TransCelerate member companies and eight FDA offices to discuss ways to scale the use of pragmatic elements in clinical trials. These elements include simplified designs, leveraging routine care settings, and reaching broader patient populations. The goal is to enable more research to be conducted at the point of care and address public health challenges and areas of unmet clinical need.

According to Rob DiCicco, Vice President of Portfolio Management at TransCelerate, collaborative efforts like this are crucial in identifying practical considerations for embedding clinical research into routine care. He also highlighted 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.

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TransCelerate's Embedded Pragmatic Trials initiative aims to accelerate the adoption of these trial designs by building awareness, engaging with regulatory authorities and other stakeholders, and offering practical tools for implementation. Kevin Bugin, Head of Global Regulatory Policy at Amgen and executive sponsor of the initiative, believes that pragmatic trials have the potential to transform how clinical research is conducted by bringing trials closer to where care is delivered and expanding patient access.

During the tabletop exercise, TransCelerate presented a hypothetical case study evaluating a new indication for a post-approval medication. The trial design incorporated several pragmatic elements such as simplified data collection, broad eligibility criteria, and integration with electronic health records (EHRs).

Key themes from the session included 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. There were also discussions on improving data quality by integrating EHR data with quality-by-design (QbD) approaches and clarifying endpoint definitions. The need for aligned expectations regarding endpoint selection, adjudication, blinding, and inspection priorities across multiple FDA offices was also emphasized. Additionally, computational methods to ensure data integrity while preserving patient privacy during inspections were explored.

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In conjunction with the tabletop exercise, TransCelerate has released "Getting Started with Embedded Pragmatic Trials: A Resource Guide," a comprehensive 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.

Moving forward, TransCelerate plans to continue collaborating with global regulators, sponsors, and stakeholders to advance understanding and adoption of pragmatic trials through real-world case studies, tools, and shared learnings. They also aim to clarify operational distinctions between pragmatic and traditional trial models and identify infrastructure-ready geographies to support implementation at scale.

The full report from the tabletop exercise titled "Enabling the Implementation of Pragmatic Elements at Scale" is available on TransCelerate's website. To learn more about TransCelerate's Embedded Pragmatic Trials Initiative, visit their website.
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