2019 FDA Science Forum Transforming Health : Innovation in FDA Science September 11 & 12, 2019 • 2019 FDA Science Forum Transforming Health: Innovation in FDA Science Wednesday, September 11, 2019 & Thursday, September 12, 2019 FDA White Oak Campus, Great Room www.fda.gov/scienceforum • Table of Contents A Message from FDA’s Acting Commissioner, Ned Sharpless, MD . 6. A Message from FDA’s Chief Scientist, RADM Denise Hinton . 8. Opening Remarks from Amy Abernethy, MD, PhD, FDA Principal Deputy Commissioner, Chief Information Officer. 10 Keynote Speaker: Francis Collins MD, PhD, Director, National Institutes of Health FDA and NIH: Partners in Transformation . 11. 2019 FDA Science Forum Agenda . 12. Speakers Bios and Abstracts | Concurrent Session 1: Precision Health Regulatory Perspective on Digital Health for Precision Medicine . 20 Sex and Gender Differences in Health and Disease . 21 Clinical Trials In 200 Microliters – Extending Approval in Rare Diseases Using In Vitro Data. .23 Genomic Biomarker Use in Cardiovascular Disease Clinical Trials . .24 Learning Healthcare Systems and Big Data: Advancing the Goal of Precision Pain Medicine . 25 Speakers Bios and Abstracts | Concurrent Session 2: Advanced Techology Accelerating Innovation in Manufacturing Technology for Biomanufactured Products: Manufacturing U.S. and NIST . 27 MetagenomeTrakr Pilot Program for Rapid Foodborne Pathogen Detection . 29 The Promise of Microbial Genomics: How Microbiology is Standing Up to the Many Challenges of a 21st Century Food Supply . .30 Editing the Genome without DNA Breaks. .31 Computational Modeling for Medical Devices . 32 Avian Influenza A Susceptibilities to Pulmonary Surfactant Protein D: Confirmation of N-glycan sub-type as a Pathogenic Factor in Influenza . 33 Panel Q & A, Advanced Tehcnology at FDA: Potential Utility and Requlatory Challenges . .35 Speakers Bios and Abstracts | Concurrent Session 3: Product Accessibility, Integrity, and Security Violent Non-State Actor Use of Food as a Delivery System: Comparing ideological and Non-Ideological Perpetrators . 36 Product Availability: A Drug Shortage Perspective . 37 Bio-Terrorism Regulations and Food Security . 38 FDA Food Defense Efforts – A Preventive Approach to Food Terrorism. .40 On the ‘Cyber-Securability’ of Medical Devices . 42 Speakers Bios and Abstracts | Concurrent Session 4: Predictive Tools Developing Digital Measures from Person-Generated Health Data . 43 MRI In Nonclinical Safety Assessment . 44 The VICTRE Project: The First All-In-Silico Imaging Clinical Trial . 45 Use of The MHC Associated Peptide Proteomic Assay to Understand the Immunogenicity Risk of Therapeutic Proteins . 46 Cardiac and Hepatic Cellular Systems to Model Human Drug Effects . 47 C. elegans for Rapid Developmental Neurotoxicity Assessment of Mixtures . .48 Determination of Seafood Decomposition by Mass Spectrometry with Sensory-Driven Modeling . 49 Speakers Bios and Abstracts | Concurrent Session 5: Advancing Digital Health and Artificial Intelligence Assessment of Devices that Rely on Artificial Intelligence/Machine Learning . 51 Deep Learning for Polypharmacy and Drug Repurposing . 52 FDA’s Real-World Evidence Program – Technology and Innovation as a Cornerstone . 54 Assessment of Devices that Rely on Artificial Intelligence/Machine Learning . .56 AI at FDA: Potential Utility and Regulatory Challenges . .57 Speakers Bios and Abstracts | Concurrent Session 6: Outbreak! Innovation in Science: Protecting People from Emerging Infectious Disease Threats . 59 Foodborne Outbreak Investigations in the Whole Genome Sequencing Era . 60 Immune Responses to Zika Infections . 62 Tracking antibiotic resistance in Salmonella: The role of the National Antimicrobial Resistance Monitoring System . 63 Emerging & Pandemic Threat Preparedness . 64 Strengthening Regulatory Science to Support the Development of Medical Countermeasures for Emerging Infectious Diseases. .65 Speakers Bios and Abstracts | Concurrent Session 7: Addiction Drug Abuse in the U.S. 67 FDA Response to the Opioid Crisis . 68 Assessing the Structural and Pharmacological Similarity of Newly Identified Drugs of Abuse to Controlled Substances Using PHASE . 69 Preclinical Pharmacology of Novel Synthetic Opioids Appearing in Clandestine Drug Markets . 70 FDA Assessment of the Abuse Potential of Drugs, Including Opioids . 71 Speakers Bios and Abstracts | Concurrent Session 8: Impacting Public Health Through Electronic Media: Empowering Consumers, Patients, and Other Stakeholders Tobacco Regulatory Science – Understanding the Role of Flavor in E-Cigarette Marketing. .72 Using Content Analysis to Understand Tobacco Industry Use of Technology to Engage Consumers . 73 Consumers’ Use of Personal Electronic Devices in the Kitchen . 75 Assessment of Patient Perspective on Risks and Benefits Associated with High Intensity Focused Ultrasound (Hifu) for The Ablation of Prostate Tissue in Men With Localized Prostate Cancer . 76 Clinical Outcome Assessments in Medical Product Development . 78 Collect Once, Use Many Times: Challenges and Opportunities for the Use of Real-World Evidence to Improve Healthcare. .79 Poster Session 1 | Topic: Precision Health (Day 1, A.M.) . 80. Poster Session 1 | Topic: Advanced Technology (Day 1, A.M.) . .99 . Poster Session 2 | Topic: Advanced Technology (Day 1, P.M.) . 131. Poster Session 2 | Topic: Product Accessibility, Integrity, and Security (Day 1, P.M.) . 159 Poster Session 2 | Topic: Predictive Tools (Day 1, P.M.) . 175 Poster Session 3 | Topic: Predictive Tools (Day 2, A.M.) . .183 . Poster Session 3 | Topic: Advancing Digital Health and Artificial Intelligence (Day 2, A.M.) . 228. Poster Session 4 | Topic: Advancing Digital Health and Artificial Intelligence (Day 2, P.M.) . 234. Poster Session 4 | Topic: Outbreak! (Day 2, P.M.) . .237 . Poster Session 4 | Topic: Addiction (Day 2, P.M.). 269 Poster Session 4 | Topic: Empowering Consumers, Patients, and Other Stakeholders (Day 2, P.M.) . .272 Poster Guidelines . 285. Schedules. 286 Acknowledgements . .287 . Map of Building 31, Great Room . 291. A Message from FDA’s Acting Commissioner Norman E. “Ned” Sharpless, MD Acting Commissioner, FDA I’m honored to welcome you to FDA’s 2019 efficient risk-based monitoring. Science Forum. The Science Forum gives our As these transformative technologies are outstanding scientists an opportunity to share developed, FDA is increasing our expertise with the public the critical scientific activities and research in these areas to enable optimal and research that we perform daily to protect, review and regulation of products incorpo- promote, and advance public health across rating from cutting-edge technology, and to our entire regulatory portfolio, from medical help us leverage these tools, where appro- products and foods to tobacco products. priate, to maximize our public health impact. As we all know, the pace of scientific innova- FDA’s research is critical for developing the tion is accelerating. In just the past few translational science strategies and regula- years, we’ve seen transformative technolo- tory standards needed to assess innovative gies like artificial intelligence, whole genomic technologies that might not fit into older sequencing, and cell- or gene-based therapies regulatory approaches. Our research is result in FDA-approved or cleared products advancing our understanding of these novel that are advancing precision medicine--deliv- technologies and helping create the modern ering the right intervention to the right patient, regulatory approaches needed to assess their at the right time. safety and performance. What may not be as well appreciated is that I’d like to mention just a few of FDA’s ground- many of these technologies are transforming breaking scientific research efforts highlighted the way FDA conducts our mission, as they in our two-day Science Forum: create new opportunities for us to do our work more effectively and efficiently. For instance, Researchers in FDA’s Center for Food Safety new digital ledger technologies offer the and Applied Nutrition (CFSAN) are developing potential to help FDA determine the source or high-resolution forensic tools to help link origin of foodborne illnesses by tracking and foodborne illnesses that would have previ- tracing food from farm to table in seconds ously gone undetected. These tools, including instead of days or weeks. Predictive artificial whole genome sequencing (WGS), enable the intelligence algorithms can help our investiga- comparison of complete pathogen genomes tors to better target products for examination to better establish commonalties of illnesses and potentially stop unsafe food and medical and assist with determining the source of food products, including illegal drugs, offered for contamination, including individual farms or import at the border, before they reach our facilities and specific geographic locales. communities; identify facilities that should To support this effort, FDA created Genome- be prioritized for inspection; or to predict and Trakr, an integrated network of state and analyze risks in clinical trials to support more federal laboratories that uses whole genome 2019 FDA Science Forum 6 Transforming Health: Innovation in FDA Science sequencing to enhance traceback of foodborne always grab headlines like these other efforts, pathogens. GenomeTrakr comprises several but it is equally vital, and this Science Forum government food safety agencies and nearly gives us an ideal opportunity to show you three-dozen state, academic, and international