TransCelerate BioPharma and the US Food and Drug Administration (FDA) have released new resources highlighting ways to embed pragmatic elements into clinical trials, with the aim of bringing research closer to routine care and increasing patient participation.

The summary report follows a tabletop exercise held in December 2024 between the FDA’s Center for Clinical Trial Innovation (C3TI) and more than 30 pharmaceutical R&D leaders from TransCelerate member companies. The exercise explored how pragmatic trial designs could be scaled to allow research to take place directly at the point of care.

Pragmatic trials are designed to simplify trial processes, use routine healthcare settings, and include broader patient populations. These features make them well-suited for tackling public health challenges and unmet medical needs.

“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.”

As part of the non-binding exercise, participants reviewed a hypothetical trial design for a new indication of an already approved medicine. The design incorporated pragmatic elements including simplified data collection, broad eligibility criteria, and integration with electronic health records (EHRs).

Kevin Bugin, head of global regulatory policy at Amgen and executive sponsor of TransCelerate’s Embedded Pragmatic Trials initiative, said: “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.”

Key themes identified from the exercise included:

• Design and oversight: hybrid trial models, early feasibility checks, and oversight frameworks tailored to pragmatic elements.

• Data and endpoints: use of EHR data, quality-by-design approaches, and clear endpoint definitions.

• Agency collaboration: alignment across FDA offices on endpoints, blinding, adjudication, and inspection focus.

• Efficiency and privacy: computational approaches to safeguard data integrity while protecting patient privacy.

Alongside the report, TransCelerate published Getting Started with Embedded Pragmatic Trials: A Resource Guide, a practical tool to help stakeholders design and implement pragmatic trials. The guide covers trial features such as simplified data collection, wide eligibility criteria, and EHR integration.

TransCelerate said it will continue to work with regulators, sponsors, and stakeholders to expand the use of pragmatic trials, share real-world case studies, and identify regions with infrastructure to support these trial models at scale.