2017 FDA Science Forum Mission Possible: FDA Research Enhancing Public Health May 31 - June 1, 2017 • Great Room www.fda.gov Contents A Message from FDA’s Commissioner . 4 A Message from FDA’s Acting Chief Scientist . 6 Keynote Speaker . 17 Speaker Bios and Abstracts Session 1: Identification and Evaluation of New Biomarkers 18 Session 2: FDA Response to Urgent Public Health Needs 23 Session 3: Microbiome and Human Health 29 Session 4: Advanced Manufacturing and 3D Printing 34 Session 5: Omics Technologies at the FDA 40 Session 6: Patient and Consumer Engagement and Communication 45 Session 7: Computational Modeling and Simulation at FDA 50 Session 8: Current Progress in Nanotechnology Research at FDA 56 Poster Session 1 (Day 1, AM) Scientific Topic: Identification and Evaluation of New Biomarkers 61 Scientific Topic: FDA Response to Urgent Public Health Needs 81 Poster Session 2 (Day 1, PM) . 102 Scientific Topic: Additive Manufacturing and 3D Printing 102 Scientific Topic: Microbiome and Human Health 112 Scientific Topic: FDA Response to Urgent Public Health Needs 122 Poster Session 3 (Day 2, AM) Scientific Topic: Omics Technology at FDA 140 Scientific Topic: Patient and Consumer Engagement and Communication 155 Scientific Topic: FDA Response to Urgent Public Health Needs 170 Poster Session 4 (Day 2, PM) Scientific Topic: Computational Modeling and Simulation at FDA 176 Scientific Topic: Current Progress in Nanotechnology Research at FDA 200 Acknowledgements . 214 A Message from FDA’s Commissioner Scott Gottlieb, MD Commissioner, FDA Addendum letter will be posted. School of Medicine and of Wesleyan University, in Middletown, Connecticut, where he studied Bio of Scott Gottlieb, MD Economics. Dr. Scott Gottlieb was sworn in as the 23rd Commissioner of Food and Drugs on May 10, 2017. Dr. Gottlieb is a physician, medical policy expert, and public health advocate who previously served as the FDA’s Deputy Commissioner for Medical and Scientific Affairs and before that, as a senior advisor to the FDA Commissioner. He also worked on implementation of the Medicare drug benefit as a Senior Adviser to the Administrator of the Centers for Medicare and Medicaid Services, where he supported policy work on quality improvement and the agency’s coverage process, particularly as it related to new medical technologies. In 2013 Dr. Gottlieb was appointed by the Senate to serve on the Federal Health Information Technology Policy Committee, which advises the Department of Health and Human Services on healthcare information technology. Dr. Gottlieb was previously a Resident Fellow at the American Enterprise Institute, and a Clinical Assistant Professor at the New York University School of Medicine in Manhattan, where he also practiced medicine as a hospitalist physician. He completed a residency in internal medicine at the Mount Sinai Medical Center in New York, New York and is a graduate of the Mount Sinai 2017 FDA Science Forum 4 Enhancing Public Health 2017 FDA Science Forum 5 Enhancing Public Health A Message from FDA’s Acting Chief Scientist Luciana Borio, MD Acting Chief Scientist, Office of the Chief Scientist, OC, FDA We live in an age of speed, distinguished by increasingly cleared through FDA’s Center for instant access to a raft of products, the push Devices, FDA bioengineers have positioned button economy, and communication in 140 themselves at the forefront of knowledge characters. Some may wonder why this and research about this process, conducting characteristic of our times, which has reshaped research into patient matching, imaging, and other sectors of our economy, has not led to phantoms. With our proactive posture, FDA is equally rapid medical product development. paving the way for safe and effective innovation And so it bears noting that the app we use to hail that will usher in life-saving advanced a cab or order a book is, in fact, the outcome treatments for patients. of many long hours of scientific research, Or take the growing use of nanomaterials in collaboration, and investment. FDA-regulated products. Silver nanoparticles The 2017 FDA Science Forum gives the are now used in wound dressing for their American public the chance to understand and antimicrobial properties. And liposomal appreciate the unique scientific research and nanoparticles are used as drug carriers to collaborative efforts of our 11,000 scientists, reduce toxicity and increase circulation time who investigate and apply new science and in the blood. Characterizing these complex emerging technologies to inform FDA’s nanomaterials is challenging, and well regulatory decision-making and foster safe developed methods are not available. But FDA innovation. research has produced analytical methods for characterizing nanomaterials in over-the- Just as industry focuses on product counter FDA-regulated products. This will help development research and academia focuses us with assessing risk, developing industry on the scientific foundation, FDA research guidelines for characterizing nanomaterials, concentrates on creating test methods and postmarket surveillance, and determining shelf developing a knowledge of processes to make life of nanomaterials in consumer products. sure that our products are safe and effective for consumers and patients--and that the harm In the area of food safety, FDA has contributed from regulated tobacco products is reduced. to enhancing antimicrobial resistance The science we do at FDA is critical to product monitoring in a collaborative effort with our quality and safety, most especially because it’s sister agencies. Genomics studies conducted seldom undertaken by industry or academia. by FDA scientists have demonstrated that we can use the emerging technology whole For example, as the emerging technology of genome sequencing as an effective tool for additive manufacturing is put into use and predicting antimicrobial resistance of certain medical devices, produced by 3D printing, are foodborne pathogens. 2017 FDA Science Forum 6 Enhancing Public Health The robust science that allows us to do all commissioner for counterterrorism policy and these things--from 3D printing to omics director of the Office of Counterterrorism and technologies—also promises to speed access Emerging Threats (OCET) in the Office of the to safe and effective medical products, and Chief Scientist at FDA. In this capacity, Dr. enable a swifter and more efficient monitoring Borio provided leadership, coordination, and of food safety. But not without the strong oversight for FDA’s national and global health research that brings this vision to life. And the security, counterterrorism, and emerging urgency of developing medical products rapidly threat portfolios and led the MCMi. and successfully has never been greater as Dr. Borio has been instrumental in coordinating infectious disease outbreaks like Zika become FDA’s response to the 2009 H1N1 influenza the new normal and put us all at risk. pandemic and continues to oversee FDA’s Over the next two days we hope you gain a preparedness and response activities for deeper understanding of the cutting-edge emerging threats, such as the avian influenza A science we do at FDA to protect and promote (H7N9) virus and the West Africa Ebola epidemic. the public health. And we look forward to Before joining FDA as a medical reviewer in sharing with you some of the exciting advances 2008, Dr. Borio served as a senior associate we’re making with our partners in the scientific at the University of Pittsburgh Medical Center community. – Center for Biosecurity, assistant professor of medicine at the University of Pittsburgh, Bio of Luciana Borio, MD and advisor on biodefense programs for Dr. Luciana Borio is FDA’s acting chief scientist. the U.S. Department of Health and Human In this capacity, she is responsible for leading Services. Dr. Borio received her MD from the and coordinating FDA’s cross-cutting scientific George Washington University, and continues and public health efforts. to practice medicine at the Johns Hopkins Hospital. The Office of the Chief Scientist works closely with FDA’s product centers, providing strategic leadership and support for FDA’s regulatory science and innovation initiatives, including the Advancing Regulatory Science Initiative, the Critical Path Initiative, scientific professional development, scientific integrity, and the Medical Countermeasures Initiative (MCMi). Since 2011, Dr. Borio has served as the assistant 2017 FDA Science Forum 7 Enhancing Public Health FDA Science Forum Agenda (Day 1: May 31, 2017) 8:30 – 8:40 AM Introduction Bernadette Johnson-Williams, MEd, Senior Advisor for STEM, Office of the Chief Scientist 8:40 – 8:50 AM Welcome FDA Acting Chief Scientist, Luciana Borio, MD 8:50 – 9:05 AM Remarks and Introduction of keynote speaker FDA Commissioner, Scott Gottlieb, MD 9:05 – 9:45 AM Frontiers in Biomedical and Regulatory Science Keynote Speaker: Eric Lander, PhD, President and Founding Director of the Broad Institute 9:45 – 10:55 AM Poster Session 1 and Break Topics: 1. Identification and Evaluation of New Biomarkers 2. FDA Response to Urgent Public Health Needs 10:55 – 12:40 PM Concurrent Sessions 1 & 2: Concurrent Session 1: Identification and Evaluation of New Biomarkers Great Room Section B Session Chair : Lisa Meier McShane, PhD, Chief, Biostatistics Branch, Biometric Research Program, National Institutes of Health /National Cancer Institute 10:55 – 11:25 AM FDA/National Institutes of Health Interactions and BEST Lisa Meier McShane, PhD, Chief, Biostatistics