Case Studies in Big Data and Analysis NAS WORKSHOP ON BIG DATA AND ANALYTICS FOR INFECTIOUS DISEASE RESEARCH, OPERATIONS, AND POLICY May 10, 2016 Luciana Borio, MD Acting Chief Scientist Food and Drug Administration

FDA Mission FDA is responsible: Protecting the public health by assuring the safety, efficacy and security of human and veterinary drugs, biological products, medical devices, our nation’s food supply, cosmetics, and products that emit radiation. Advancing the public health by helping to speed innovations that make medicines more effective, safer, and more affordable and by helping the public get the accurate, science-based information they need to use medicines and foods to maintain and improve their health. Regulating the manufacturing, marketing and distribution of tobacco products to protect the public health and to reduce tobacco use by minors. Fostering the development of medical products to respond to deliberate and naturally emerging public health threats.

Food Safety

Food Safety Food enters commerce Routine disease reporting Astute clinicians observations Digital disease detection Clinical & environmental isolates WGS + Env Food enters commerce Source of contamination identified from few cases 3 Environmental Positive Samples by FDA in 2/2014 2 Environmental Positive Samples by FDA in 7/2014

Antiviral Drug Resistance

Antibacterial Resistance

Designing Better Clinical Trials – HABP/VABP HABP/VABP trials difficult to conduct Very low enrollment rates Expensive Acutely ill patients with urgent need to start treatment CTTI performing an observational study to identify patients at high risk for developing HABP/VABP Identifying risk factors for HABP/VABP may allow for “pre-consent” of high-risk patients enroll patients into trial earlier after diagnosis of HABP/VABP adjust inclusion / exclusion criteria to enroll more patients After learning from observational study – incorporate “new” approaches in one arm of an RCT to see if improves enrollment rates compared to “old” approach