TransCelerate and FDA Join Forces to Enhance Pragmatic Trials in Healthcare

Collaboration Between TransCelerate and the FDA

TransCelerate BioPharma, a nonprofit organization focused on revolutionizing clinical research, is making strides to improve healthcare with its recent collaboration with the U.S. Food and Drug Administration (FDA). On August 19, 2025, TransCelerate launched a summary report and resource guide detailing their findings from a tabletop exercise held with members from the FDA's Center for Drug Evaluation and Research (CDER). This exercise, which took place in December 2024, gathered over 30 leaders from pharmaceutical research and development and eight FDA offices, focusing on harnessing pragmatic elements in clinical trials.

What Are Pragmatic Trials?

Pragmatic trials are designed to integrate seamlessly with routine care and engage a broader set of patient populations. They emphasize simplified designs and the utilization of familiar settings to encourage more representative and realistic data collection. By focusing on real-world effectiveness rather than just efficacy, these trials aim to bridge critical gaps in public health challenges.

Rob DiCicco, Vice President at TransCelerate, emphasized the importance of these collaborative efforts: "These exercises illuminate practical strategies for embedding clinical research into everyday care. It's essential for enhancing accessibility to clinical trials and foster generative evidence that reflects real-world scenarios."

Key Findings of the Tabletop Exercise

During the exercise, participants explored several themes and actionable steps that can enable broader adoption of pragmatic trial designs. These key themes included:

1. Design and Oversight Considerations: Discussing hybrid trial methods, the oversight frameworks necessary for investigator and third-party roles, and conducting feasibility assessments to align with regulatory expectations.
2. Data Integrity and Endpoint Alignment: Addressing challenges in data quality through the thoughtful integration of Electronic Health Records (EHRs) combined with quality-by-design approaches, alongside a clear definition of endpoints.
3. Value of Broad Agency Collaboration: The necessity for aligned expectations on endpoint selection and regulatory procedures across various FDA divisions.
4. Operational Efficiency and Privacy: Exploring innovative computational techniques to maintain data integrity while ensuring patient privacy during inspections.

New Resource Guide

Accompanying the findings from the exercise, TransCelerate also published a comprehensive resource guide titled "Getting Started with Embedded Pragmatic Trials". This guide serves as a vital tool for stakeholders eager to understand and engage in embedded pragmatic trial designs. It outlines essential resources to navigate the complexities of parameters such as streamlined data collection, inclusive eligibility criteria, and effective EHR integration.

Looking Ahead

TransCelerate is committed to advancing the adoption of pragmatic trials through continuous collaboration with global regulatory bodies and stakeholders. Future efforts will aim to clarify the distinctions between pragmatic and conventional trial models and identify geographical regions that are equipped to support scalable implementations.

Kevin Bugin, Amgen’s Head of Global Regulatory Policy, and an enthusiastic advocate for TransCelerate's Embedded Pragmatic Trials initiative remarked, "These trials are pivotal in transforming clinical research methodologies, propelling trials closer to patient care settings and expanding the spectrum of patient access to clinical trials."

TransCelerate is poised to build on this critical collaboration with the FDA. Their ongoing initiatives will include sharing real-world case studies to enhance understanding and traction towards pragmatic trial adoption.

For a detailed overview of the "Enabling the Implementation of Pragmatic Elements at Scale" summary report, you can access it here. To learn more about TransCelerate’s commitments to pragmatic trials, visit this link.

About TransCelerate BioPharma

TransCelerate BioPharma aims to foster collaboration across the biopharmaceutical landscape, streamlining clinical trials to expedite the delivery of new treatments. Headquartered near Philadelphia, the organization brings together 20 member companies with the goal of improving connectivity, sharing information, and enabling innovative trial designs in healthcare.