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Speaker: Dr. Hong Yang (FDA, Center for Drug Evaluation and Research)

Abstract:

Medical product decision-making at FDA is a complex process involving large, multi-disciplinary teams, voluminous streams of scientific information, regulatory requirements, and diverse external stakeholders. Focusing on Agency decision-making at the time of initial marketing authorization, this session will provide an overview of the principles of benefit-risk assessment used by the three medical product centers and demonstrate how benefit-risk assessment, a subset of decision analysis, informs regulatory decision-making. Illustrative case examples showing the spectrum of decision analysis approaches used will be provided as well as information useful to product sponsors. Dr. Lackey from the Center for Drug Evaluation and Research (CDER), will provide an overview of the descriptive Benefit-Risk Framework for new drug approval decisions as well as the Benefit-Risk Guidance for Industry. This talk will present a case study showing how regulators approached the approval decision, how the principles in the Guidance are applied, and explore some key areas of concern and describe how those were resolved. Dr. Yang, from the Center for Biologics Evaluation and Research (CBER), will present a case example from a recent vaccine approval. This example will explore regulatory challenges and how the FDA Benefit-Risk Framework, real-world data and quantitative benefit-risk modeling are used to support the decision making. Dr. Gebben, from the Center for Devices and Radiologic Health (CDRH), will discuss how patient preference information is utilized by the Center as well as guidance for industry on collection and submission of preference information. This talk will highlight how patient preferences are integrated into regulatory decision-making, including the use of preference techniques (discrete choice experiments, threshold technique, etc.) to gain insights. The session will close with a panel discussion.