Session Synopsis: Drug development programs will look different in the era of precision medicine. Already, we are seeing new types of trials—master protocols, basket trials, umbrella trials—intended to cope with the complexities of biomarker-defined disease subsets and target- rather than disease-focused therapeutic interventions. More attention is now focused on the performance of diagnostics as they become central to therapeutic decision-making. One of the biggest challenges in development is “lumping” versus “splitting”: what groups to incorporate into a trial, using what criteria. As extrapolation beyond studied subgroups becomes a necessity, the use of real world evidence to fill in gaps will become a compelling alternative.
Session Chair Profile
M.D., Director, the United States Food and Drug Administration/Center for Drug Evaluation and Research
Janet Woodcock is the director of the Center for Drug Evaluation and Research (CDER) at the Food and Drug Administration (FDA). Dr. Woodcock has shown dedication to personalized medicine by fast-tracking individualized treatments through the FDA approval process and by encouraging collaboration between the regulatory and industry arenas. During her tenure, both of the individualized treatments Xalkori and Zelboraf received FDA approval paired with companion diagnostics. Dr. Woodcock has led many of FDA’s drug initiatives. She introduced the concept of risk management in 2000 as a new approach to drug safety. Since 2002, she has led the “Pharmaceutical Quality for the 21st Century Initiative,” FDA’s highly successful effort to modernize drug manufacturing and its regulation. In 2004, she introduced FDA’s “Critical Path” Initiative, which is designed to move medical discoveries from the laboratory to consumers more efficiently. Most recently, Dr. Woodcock launched the “Safety First” and “Safe Use” initiatives designed to improve drug safety management within and outside FDA, respectively. Dr. Woodcock previously served as FDA’s deputy commissioner and chief medical officer. She also led CDER as director from 1994–2005. Prior to joining CDER, Dr. Woodcock oversaw approval of the first biotechnology-based treatments for multiple sclerosis and cystic fibrosis in her position as director of the Office of Therapeutics Research and Review in FDA’s Center for Biologics Evaluation and Research. Dr. Woodcock received her medical degree from Northwestern University Medical School, and her undergraduate degree from Bucknell University. She has held teaching appointments at Pennsylvania State University and the University of California at San Francisco. She joined FDA in 1986.