Modernizing Research and Evidence Consensus Definitions
Donna R. Rivera, Tracy L. Cutler, Lisa M. McShane, Ann Meeker-O’Connell, Hilary D. Marston, Robert M. Califf, Monica M. Bertagnolli, FDA-NIH MoRE Glossary Working Group, Larissa Avilés‐Santa, Kassa Ayalew, Ryan Bayha, Joshua Chetta, John Concato, Michelle Culp, Lori E. Dodd, Natasha Facey, Richard A. Forshee, Andrew N. Freedman, Philipa Friedman, Elisa Golfinopoulos, Cheryl Grandinetti, Hannah Jarman-Miller, Lyric A. Jorgenson, Alyson Karesh, Lydia Kline, Paul G. Kluetz, Stefanie Kraus, Jennifer J. Lee, Catherine C. Lerro, Mark Levenson, Jie Li, Yun Lu, Céline Moore, Anna E. Ordóñez, Gail E. Potter, Sheila A. Prindiville, Yves Rosenberg, Gisele Sarosy, Stephanie Shapley, Emily R. Smith, Anne Talley, Robert F. Tamburro, Charles J. Viviano, Charlie Yongpravat
Abstract
Importance: The US Food and Drug Administration (FDA) and National Institutes of Health (NIH) share a mutual interest in facilitating efficient, well-designed clinical studies of drugs, devices, and biological products. Recent advances in science and technology, as well as innovative approaches to research design and methodology, provide opportunities to enhance efficiency in medical product development and improve participant engagement in clinical trials. Recent initiatives across the FDA and NIH focus on evidence modernization approaches. Fostering appropriate use of novel designs and sources of evidence, such as real-world data (RWD) to support marketing authorizations and satisfy postapproval study requirements, may be enhanced by using consensus terminology for innovative study designs. Objective: To facilitate effective communication within the scientific community, FDA and NIH formed an interagency collaborative initiative to define clinical research terms related to innovative study designs, with a focus on studies using RWD, for FDA-regulated medical products or broader research and foster a shared understanding of terms across the clinical research ecosystem. Evidence Review: The FDA-NIH Modernizing Research and Evidence (MoRE) Glossary Working Group (MGWG) was initiated in April 2023 to evaluate terms inadequately defined within the clinical research community that would benefit from development of a consensus definition. The MGWG conducted a landscape evaluation of common innovative design terminology that may lack clarity or concordance. Subsequently, the MGWG reviewed whether and how existing regulations, guidance, and policies use or define such terms. Following the landscape evaluation, the MGWG engaged in rigorous review to seek consensus definitions. In addition, federal agencies sought public input via a request for information before publishing the included terms and definitions. Findings: The MGWG developed the MoRE Consensus Definitions, comprising 40 clinical research terms and definitions related to innovative clinical study designs that support scientific, patient, clinical, and regulatory decision-making. Conclusions and Relevance: The MoRE Consensus Definitions are intended to facilitate effective communication about clinical research and enable transparency around innovative clinical study designs. This publication makes available the glossary developed through this collaboration and serves as an accessible resource for the clinical research enterprise. Furthermore, as clinical research is continuously evolving, additional efforts may focus on emerging new vocabulary and evolving use of current terms to benefit medical product development.