The DUPLICATE initiative is building an empirical evidence base for using longitudinal insurance claims prospectively to achieve large-scale replication of randomized controlled trials. Dr. Wang, a codirector of the initiative, is an associate professor of medicine at Harvard Medical School and associate epidemiologist in the Division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women’s Hospital.
In the latest episode of the NIH Collaboratory Grand Rounds podcast, Dr. John Concato and Dr. Richard Platt continue their discussion about the FDA draft guidance on real-world evidence.
John Concato, MD, MS, MPH
Associate Director for Real-World Evidence Analytics
Office of Medical Policy (OMP)
Center for Drug Evaluation and Research (CDER)
Food and Drug Administration (FDA)
Keywords
Big data; Real-word evidence; Real-world data; 21st Century Cures Act; FDA Draft Guidance
Key Points
Big Data, a term first used in the 1990s, leverages modern technology to increase the quantity, forms, speed, and capability to manipulate large-scale data. Real-world data (RWD) is a term with specific regulatory implications referring to health care data routinely collected from a variety of sources. Real-world evidence (RWE) is clinical evidence derived from analysis of RWD regardless of study design.
Terminology is important in research work, and we should strive to be as precise as possible with the terminology we use.
With the 21st Century Cures Act of 2016, the FDA established a program to evaluate the potential use of real-world evidence to support new indications for drugs and satisfy post-approval study requirements.
In 2021, the FDA issued 4 draft guidance documents for Real-world data and Real-world evidence intended to guide the selection and management of data sources to appropriately address the study question and support decision-making for drug and biological products.
Discussion Themes
– Could real-world data sources be certified and preclude the need for submission of source data on a study specific basis? From the FDA point-of-view, while reliability can be more readily evaluated and would tend to be more stable, the relevance to a particular study could not be determined as easily.
– While there can be a reflex that says we can never be sure about major confounding, it should not be the miasma of the 21st century. A thoughtful approach that considers the characteristics that matter is the best approach.
SACHRP provides recommendations on human subjects protection to the Secretary of the U.S. Department of Health and Human Services and reviews activities of the Office for Human Research Protections (OHRP). Other topics to be covered during the two-day meeting include informed consent comprehension, the return of research results to human subjects, and ethical/regulatory issues in interventional social media research.
The meeting is available to the public and will be videocast live.
View the meeting agenda
View the live webcast (available July 21-22, 2014)
A link to materials from the meeting will be provided in an update to this post when available.
In the latest episode of the