FDA, NIH create common vocabulary for real-world evidence
The US Food and Drug Administration (FDA) and National Institutes of Health (NIH) have jointly published a glossary of real-world evidence/real-world data (RWD/RWE) terms intended to improve communication in interventional and noninterventional studies with innovative trial designs and data sources. It applies to studies of drugs, devices, and biological products.The glossary of 40 clinical research terms appeared in a JAMA Network Open article published 17 June by Donna Rivera, associate director of pharmacoepidemiology, RWD, and RWE at the FDA Oncology Center of Excellence, and colleagues from both agencies.
“The clear use of these terms is intended to allow for easier assessment and comparison of studies across the clinical research enterprise and improve communication about research opportunities,” the authors wrote. “By applying aligned consensus definitions, the research community can increase their ability to accurately characterize clinical research, potentially spurring further innovation.”
The glossary reflects the thinking and work of epidemiologists, statisticians, clinicians, pharmacologists, engineers, and policy experts at both agencies, as well as public review and input, the authors noted. The effort was led by the FDA-NIH Modernizing Research and Evidence (MoRE) Glossary Working Group (MGWG).
Modernizing evidence and harmonizing terminology has been a scientific priority for leadership at the agencies, to “foster a shared understanding across the clinical research ecosystem,” the authors explained (RELATED: Mark McClellan: More alignment needed on RWD terminology, Regulatory Focus, 21 February 2024). Novel scientific approaches have expanded, but communication has not always kept pace, the authors suggested.
“Gaps in our common vocabulary to describe modern clinical research methods introduce variation in how those terms are used across the clinical research ecosystem, especially around descriptions of innovative interventional and noninterventional study designs,” Rivera et al. wrote.
The MGWG focused on clinical research terms that most needed clarification and consensus definitions. To develop the glossary, the team reviewed federal regulations, FDA guidance, and publicly available clinical research resources. The public review led the group to consider adding new terms.
“Overall, the feedback from the public comment period improved the definitions, provided an opportunity to add additional reference terms, and recommended ideas for potential future efforts,” the authors explained.
The glossary includes terms such as data curation and harmonization, pragmatic clinical trial, selection bias, and synthetic data. The group had a special focus on describing complex innovative trial designs.
A pragmatic clinical trial is defined as, “A clinical trial designed to efficiently inform decision-making on the benefits, burdens, and risks of health interventions in representative populations by including pragmatic elements that (1) are partially or fully integrated into routine clinical practice and/or (2) streamline trial design and conduct.”
Pragmatic elements are defined as, “Design features that can be integrated into a clinical trial, including but not limited to, [less than or equal to] 1 of the following elements: broad eligibility criteria, simplified recruitment and follow-up, flexibility in delivery of the intervention (e.g., community settings), flexibility in assessment frequency, streamlined data collection, and measurement of outcomes relevant to the population.”
The glossary also defines pragmatic trials, noninterventional studies, observational studies, cluster randomized trials, N-of-1 trials, sequential multiple assignment randomized trials (SMART), stepped-wedge cluster-randomized trials, and umbrella trials.
“Due to the complexities of describing these designs, misperceptions are common in the clinical research community, and the consensus definitions are intended to bring clarity,” Rivera and colleagues explained. “For example, the definitions clarify that pragmatic trial designs include elements along a continuum that often also incorporate randomization, and that RWD denotes a data source rather than a study design, which can be prospectively or retrospectively collected.”
The authors noted that standardized terminology will help with descriptions in study protocols, regulatory submissions, grant applications, and institutional review board reviews. It will also help improve clinical trial efficiencies and dialogue after products are on the market.
“As innovative study design terminology is evolving, the clinical research community can continue to exercise diligence in defining emerging terms as well as where shared processes may be needed for re-evaluating these foundational current terms together as part of building an iterative practice to benefit greater shared understanding,” they wrote.
Special Communication paper
×
Welcome to the new RAPS Digital Experience
We have completed our migration to a new platform and are pleased to introduce the updated site.
What to expect: If you have an existing login, please RESET YOUR PASSWORD before signing in. After you log in for the first time, you will be prompted to confirm your profile preferences, which will be used to personalize content.
We encourage you to explore the new website and visit your updated My RAPS page. If you need assistance, please review our FAQ page.
We welcome your feedback. Please let us know how we can continue to improve your experience.