Closing the Real-World Evidence Gap: Pragmatic Clinical Trials & Observational Studies

December 4, 2019 8:00 AM - 10:00 AM

MassBio, 300 Technology Square 8th Fl, Cambridge, MA 02139

Add to Calendar 12/4/2019 8:00:00 AM 12/4/2019 10:00:00 AM Closing the Real-World Evidence Gap: Pragmatic Clinical Trials & Observational Studies

Clinical trials are performed at specialized sites, with entry criteria that selects subjects likely to show efficacy but who are not necessarily representative of the broad population of patients. There is increasing concern on the part of regulators that explanatory clinical trials, which confirm a clinical hypothesis, tend to overestimate the effectiveness and underestimate the safety of medicinal products and devices. The use of real-world evidence to support regulatory decision making is discussed in the FDA final guidance for medical devices (2017) and draft guidance for drugs and biologics (2019). The guidances outline prospective pragmatic clinical trials (PCTs) and retrospective observational studies as the means to bridge the gap between explanatory trials and adoption evaluation. In this forum, our panel of experts will discuss the evolution and future of PCTs and observational studies as important tools for integration of real-world evidence in medical product development.

Sponsored by the Biostatistics, Data Management & Clinical Trials (BSDMCT) Forum Working Group. MassBio, 300 Technology Square 8th Fl, Cambridge, MA 02139
Vice Chancellor for Clinical and Translational Research, Duke University
Robert M. Califf, MD, MACC, is vice chancellor for health data science and director of Duke Forge, the Center for Actionable Health Data Science at Duke Health, Donald F. Fortin, MD Professor of Cardiology in the Duke University School of Medicine, Chair of the Board of the People Centered Research Foundation, and an advisor for Verily Life Sciences, a member of the Alphabet family of companies formed by Google. He served in the administration of President Barack Obama as Deputy Commissioner for Medical Products and Tobacco in the U.S. Food and Drug Administration (FDA) from 2015-2016, and as Commissioner of Food and Drugs from 2016-2017. Prior to joining the FDA, Dr. Califf was a professor of medicine and vice chancellor for clinical and translational research at Duke University. He was founding director of the Duke Clinical Research Institute. A nationally and internationally recognized expert in cardiovascular medicine, health outcomes research, healthcare quality, and clinical research, Dr. Califf has led many landmark clinical trials and is one of the most frequently cited authors in biomedical science, with 1,250 publications in the peer-reviewed literature. Dr. Califf is a member of the National Academy of Medicine (formerly the Institute of Medicine [IOM]). Dr. Califf has served on numerous IOM committees, and was a member of the FDA Cardiorenal Advisory Panel and FDA Science Board's Subcommittee on Science and Technology. Dr. Califf has also served on the Board of Scientific Counselors for the National Library of Medicine, as well as on advisory committees for the National Cancer Institute, the National Heart, Lung, and Blood Institute, the National Institute of Environmental Health Sciences, and the Council of the National Institute on Aging. Dr. Califf is a graduate of Duke University School of Medicine. He completed a residency in internal medicine at the University of California, San Francisco and a fellowship in cardiology at Duke.