CERGAS Seminar "Use of Real-World Data and Evidence for Medical Product Evaluation: Promise and Perils."

The U.S. Food and Drug Administration (FDA) has oversight over the medical product industry. Among its authorities is establishing the evidentiary standards required to demonstrate the safety and effectiveness of novel drugs and medical devices in order to secure FDA approval, usually clinical trials. However, there remains important uncertainties for all newly approved drugs and medical devices, issues that are likely to be important to patients and clinicians, regulators and industry. It’s unlikely that each remaining uncertainty can be addressed through clinical trial evaluation. Instead, efforts are needed to leverage real-world data to generate real-world evidence – data that comes from the routine practice of medicine. These data are increasingly being used as part of life-cycle evaluation efforts, repeated evaluations of products over the course of their market life. These efforts hold promise, but are also fraught with perils, especially when isolated to observational research studies. The future is likely to make greater use of pragmatic clinical trials.

Speaker:

Joseph S. Ross, MD, MHS, is a Professor of General Internal Medicine at the Yale School of Medicine, of Health Policy and Management at the Yale School of Public Health, an Associate Physician of the Center for Outcomes Research and Evaluation at Yale-New Haven Health System, and Co-Director of the National Clinician Scholars Program at Yale. With expertise in health services and outcomes research, his research examines the use and delivery of higher quality care, how clinical research is translated into practice, and issues related to pharmaceutical and medical device regulation, evidence development, postmarket surveillance, and medical product adoption. Dr. Ross co-directs the Yale-Mayo Clinic Center for Excellence in Regulatory Science and Innovation (CERSI), the Yale Open Data Access (YODA) Project, and the Collaboration for Regulatory Rigor, Integrity, and Transparency (CRRIT) and leads efforts at Yale-New Haven Health System in collaboration with the National Evaluation System for health Technology (NEST). He has published more than 700 articles in peer-reviewed biomedical journals, co-founded the preprint server medRxiv, and is currently a Deputy Editor at JAMA.