TransCelerate BioPharma (TransCelerate), a nonprofit organization dedicated to improving the health of people around the world by accelerating and simplifying clinical research, has officially announced the release of a summary report from its tabletop exercise conducted with the U.S. Food and Drug Administration’s (FDA) CDER Center for Clinical Trial Innovation (C3TI).
According to certain reports, the company also took this opportunity to highlight results from its newly published resource i.e. Getting Started with Embedded Pragmatic Trials: A Resource Guide.
More on the given summary report would reveal how it includes a survey of 30 pharmaceutical R&D leaders from TransCelerate member companies and eight FDA offices, with the whole effort geared towards identifying actionable opportunities to scale pragmatic elements’ use in clinical trials and conduct research at the point of care.
For better understanding, pragmatic trials make up a concept which offers simplified designs, leverages routine care settings, and reaches broader, more representative patient populations, translating to an outcome ideal for addressing public health challenges and areas of significant unmet clinical need.
 TransCelerate’s Embedded Pragmatic Trials, in that regard, are effectively designed to accelerate the adoption of these trial designs, something which is done on the back of building awareness, engaging in productive dialogue with regulatory authorities and other stakeholders, as well as offering practical tools to guide implementation.
Anyway, during the course of its exercise, TransCelerate would go on to present hypothetical case study, focused on assessing a new indication for post-approval medication. You see, this clinical trial design packed together several pragmatic elements, including simplified data collection, broad eligibility criteria, and integration with electronic health records (EHRs).
“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.”
Talk about the whole thing on a slightly deeper level, we begin the session’s design and oversight considerations. This showcases how value of hybrid trial conceives fit-for-purpose investigator and third-party oversight frameworks, along with early feasibility assessments, so to ensure data readiness and regulatory compliance.
Next up, there is the prospect of data integrity and endpoint alignment. Both the given factors here are evident in opportunities to improve data quality by integrating EHR data with thoughtful quality-by-design (QbD) approaches and clarified endpoint definitions.
Another detail worth a mention is rooted in TransCelerate’s bid to reinforce value of broad agency collaboration. The idea behind that is to generate greater understanding around the need for aligned expectations regarding endpoint selection, adjudication, blinding, and inspection priorities across multiple FDA offices.
Rounding up highlights would be an aspect revolving around operational efficiency and privacy. The stated aspect is understood to include exploration of computational methods to achieve data integrity, while simultaneously preserving patient privacy during inspections.
Turning our attention towards TransCelerate’s newly-published resource, it would be a robust tool purpose-built to enhance knowledge in the context of embedded pragmatic trials, as well as providing practical guidance for successful implementation.
Not just that, this particular guide also arrives on the scene bearing an objective to identify resources, and therefore, help stakeholders navigate the design and implementation of trials that incorporate pragmatic elements.
Founded in 2012, TransCelerate BioPharma’s rise up the ranks stems from fostering collaboration across the global biopharmaceutical research and development community to simplify clinical trials and bring new treatments to patients faster, safer, and more efficiently.
The organization’s excellence in what it does can also be understood once you consider it enjoys, at the moment, more than 20 member companies and a robust portfolio of initiatives centered on strengthening connectivity, facilitating the exchange of information sharing and reuse, and achieving innovative trial designs.
“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.” .
