FDA Lecture: The FDA BEST System: Leveraging EHR data and Innovative Approaches for Surveillance of Biologic Products

RSVP

Steve Anderson, PhD, MPP
Director, Office of Biostatistics and Epidemiology
CBER, U.S. Food and Drug Administration

Abstract

In 2016 the FDA Center for Biologics Evaluation and Research (CBER) launched the BEST (Biologics Effectiveness and Safety) Initiative to build an active surveillance system better able to evaluate the safety and effectiveness of biologic products including vaccines; blood and blood products; and advanced therapeutics. BEST is part of the Sentinel initiative and advances beyond FDA’s current claims data-based systems. It offers new electronic health record (EHR) data sources, uses new innovative approaches such as artificial intelligence (AI), natural language processing (NLP) and automation. At its core, BEST’s primary function is to conduct routine surveillance activities such as queries and epidemiological studies for more than 100 million patients that address timely regulatory questions concerning biologic product safety and effectiveness. To enhance BEST’s basic operational capabilities, we are in the early phases of using NLP and AI to improve, expedite and semi-automate medical chart review for use in outcome validation. FDA also considers other sources of data such as passive or spontaneous reporting in regulatory decision-making concerning safety. However, passive reporting of adverse events (AE) for biologics is voluntary and usually suffers from underreporting. To address these and other limitations BEST is beginning to use NLP and AI to identify and mine biologic product AE information from EHRs, with the goal of populating the fields of an AE report form and automatic submission to FDA. Automated reporting is expected to supplement current reporting and is in its very early stages. But it has the potential to transform the current landscape by providing additional, potentially higher-quality real-world evidence to inform regulatory decision-making. This presentation will provide context for the development of the BEST system, discuss relevant legislative and regulatory mandates, some preliminary findings and future directions for the program.

Speaker Bio

Steve Anderson, Ph.D., M.P.P., is currently the Director of the Office of Biostatistics and Epidemiology (OBE) at the FDA Center for Biologics Evaluation and Research (CBER). He provides leadership for all CBER statistical, epidemiological and benefit-risk assessment programs and has led numerous important benefit-risk assessment and epidemiological studies of vaccines, tissues, and blood products using the FDA Sentinel and CMS data. Most recently Dr. Anderson’s office established the Biologics Effectiveness and Safety (BEST) system, CBER’s new postmarket risk identification and analysis system that engages new data sources, tools and experts to further enhance biologic product effectiveness and safety surveillance.  Dr. Anderson earned a Master’s Degree in Public Policy (MPP) at Georgetown University and while there developed the first quantitative risk assessment for antimicrobial resistant pathogens in livestock. Dr. Anderson received his PhD from the University of Cincinnati where he worked on biochemistry, drug resistance and ion pumps, pathogenicity and genomics of unique tropical disease pathogens. He has published a number of articles in biologic product safety, risk assessment, epidemiology, infectious diseases, biologics safety, and genomics.