Session Abstract – PMWC 2022 Silicon Valley


Track Chair:
Keith Yamamoto, UCSF

Patient-centric data – Real-World Evidence (RWE) and Real-World Data (RWD) - is becoming instrumental in the drug development process and for health care decisions in general. This data is not only informative for the process from discovery to new indications, clinical trial design, and drug development, it also can be of value to monitor post-marketing drug safety and for decision support in clinical practice. As the data becomes a decision driver, science companies and medical organizations are increasingly focused on leveraging RWD and RWE to not only better understand the patient populations using their drugs and the respective outcomes, but also to accelerate clinical decision support. This session will focus on the various aspects of integrating RWE and RWD to support drug development and clinical decision support

Sessions:

  • Realizing the Promise of Precision Medicine Using Real-world Evidence (PANEL)
    Session Chair: Atul Butte, UCSF
    - Nancy A. Dreyer, IQVIA
    - Mark Laabs, RCRF
    - Rhonda Cooper-DeHoff, University of Florida
  • Opportunities and Challenges in Using Real World Data (RWD) (PANEL)
    Session Chair: Vivek Rudrapatna, UCSF
    - Joseph Wu, Stanford University
  • How Are Patient Data Revolutionizing Precision Medicine? (PANEL)
    Session Chair: Clara Lajonchere, UCLA
    - Sharon Terry, Genetic Alliance
    - Farid Vij, Invitae
    - Latha Palaniappan, Stanford
  • Regulatory Requirements and Challenges for Using RWE
    Session Chair: Sheila Walcoff, Goldbug Strategies
    - Kathy Hibbs, 23andMe
  • Government Partnerships: California Initiative to Advance Precision Medicine
    Session Chair: Julianne McCall, California Initiative to Advance Precision Medicine
    - William Kim, UCSD
    - George M. Slavich, UCLA
  • Leveraging RWE to Create Value (PANEL)
    Session Chair: Kathryn A. Phillips, UCSF
    - Phil Febbo, Illumina
    - Suzanne Belinson, Tempus
    - Stacey Dacosta Byfield, OptumLabs
  • Computational Social Choice Theory's Impact on the Collection and Understanding of RWD
    - Ian Terry, LunaDNA
  • The Past, Present, and Future of RWE
    Session Chair: Keith Yamamoto, UCSF
    - Matthew Porteus, Stanford

 Session Chair Profile

M.D., Ph.D., Institute Director and Distinguished Professor, UCSF; Chief Data Scientist, University of California Health System

Biography
Atul Butte is the Priscilla Chan and Mark Zuckerberg Distinguished Professor and Director of the Bakar Computational Health Sciences Institute at UCSF, and is also the Chief Data Scientist for the entire University of California Health System, the tenth largest by revenue in the United States, with 20 health professional schools, 6 academic health centers, and 10 hospitals. Dr. Butte is an inventor on 24 patents and has authored over 200 publications, with research repeatedly featured in the New York Times and Wall Street Journal. Dr. Butte was elected into the National Academy of Medicine in 2015, and was recognized by the Obama Administration as a White House Champion of Change in Open Science in 2013. Dr. Butte is also a co-founder of Personalis (IPO, 2019), providing medical genome sequencing services, Carmenta (acquired by Progenity, 2015), discovering diagnostics for pregnancy complications, and NuMedii, finding new uses for drugs using AI.


 Session Chair Profile

M.D., Ph.D., Assistant Professor, UCSF

Biography
Vivek Rudrapatna is a physician and a clinical data scientist. His research group works on developing new methods for analyzing electronic health records and other large datasets to uncover real-world evidence on treatment effects. Vivek is a practicing gastroenterologist and specializes in the treatment of patients with Inflammatory Bowel Disease (IBD). As a clinician, a researcher, and a patient, he is interested in the study of precision medicine as it pertains to this patient population. His group is working on the development of algorithms to predict which patients may be at greater risk for developing the condition and to optimize treatment selection for IBD patients. He hopes that this work may eventually lead to a future where we can prevent IBD altogether. Vivek received his AB from Harvard (2006), his MD/PhD from Mount Sinai (2014), internal medicine residency at Baylor College of Medicine (2016), and gastroenterology fellowship at UCSF (2020).


 Session Chair Profile

Ph.D., Deputy Director UCLA Institute for Precision Health, UCLA

Biography
Clara Lajonchere, PhD is the Deputy Director for the UCLA Institute for Precision Health, Co-Director of the Clinical Neurogenetic Research Program, and Adjunct Professor of Neurology in the David Geffen School of Medicine at UCLA. Dr. Lajonchere’s background reflects a wide range of professional, clinical, and research activities across central nervous system disorders. She has spent her career on cross-cutting issues in psychiatric genetics and translational medicine. Through the UCLA Institute for Precision Health, Dr. Lajonchere is working collaboratively with thought leaders across the state of California to advance precision medicine and improve outcomes for patients. She currently serves as chair of the California Precision Medicine Advisory Council for the Governor’s Office of Planning and Research.


 Session Chair Profile

Ph.D., Professor of Health Economics and Founding Director, UCSF Center for Translational and Policy Research on Precision Medicine (TRANSPERS), Department of Clinical Pharmacy, UCSF

Biography
Kathryn A. Phillips PhD is Professor of Health Economics and Health Services Research, Department of Clinical Pharmacy, University of California San Francisco. She founded and leads the UCSF Center for Translational and Policy Research on Precision Medicine (TRANSPERS), which focuses on developing objective evidence on how to implement precision medicine into health care so that it is effective, efficient, and equitable. Kathryn has published over 150 peer-reviewed articles in major journals including JAMA, New England Journal of Medicine, Science, and Health Affairs. She has had continuous funding from NIH as a Principal Investigator for over 25 years and was recently awarded a 5-year, $5M NIH grant to examine payer coverage and economic value for emerging genomic technologies (e.g., cell-free DNA tests and tests based on polygenic risk scores). Kathryn serves on the editorial boards for Health Affairs, Value in Health, JAMA Internal Medicine, Genetics in Medicine; is a member of the National Academy of Medicine Roundtable on Genomics and Precision Health; and has served on the governing Board of Directors for GenomeCanada and as an advisor to the FDA, CDC, and the President’s Council of Advisors on Science and Technology. She has also served as an advisor to many diagnostics, sequencing, and pharmaceutical companies as well as venture capitalists. Kathryn is Chair of the Global Economics and Evaluation of Clinical Sequencing Working Group, and a member of an evidence review committee for the Institute for Clinical and Economic Review (ICER). Her work has been quoted by the Washington Post, Wall Street Journal, New York Times, CNBC, Reuters, Newsweek, and other major news outlets.


 Session Chair Profile

Ph.D., Co-Director of Precision Medicine, CA Governor's Office

Biography
Dr. McCall oversees cross-sector health policy working groups, research grantmaking, and public interagency efforts, which include serving on Governor Newsom's COVID-19 Testing Task Force and as co-author of the CA Surgeon General's Report. Previously, McCall worked at the California Senate Office of Research and as a Science and Technology Policy Fellow of the California Council on Science and Technology. Prior to state government, she spent sixteen years in neuroscience research labs, including as a Fulbright Fellow. In the community, Dr. McCall teaches Science Policy at UC Davis and UC Riverside, serves on the Editorial Board of the California Journal of Politics and Policy, occasionally directs the International Brain Bee, and is the co-founder of TEDxFulbright, the German Neuroscience Olympiad, and a chapter of the Sustained Dialogue Campus Network for racial justice. She earned a PhD from Heidelberg University, Master's degree from UC San Diego, and Bachelor's degree from Denison University.


 Session Chair Profile

J.D., CEO & Founder, Goldbug Strategies LLC

Biography
Sheila is widely recognized for her skill in integrating her FDA and CLIA regulatory, legislative, and policy expertise in the field of personalized medicine and molecular diagnostics to craft effective FDA strategies and identify novel regulatory pathways for innovators. She leads Goldbug Strategies, a specialized consulting firm focused on helping companies understand evolving FDA requirements for diagnostics, software, and companion therapeutics. Areas of particular expertise are NGS-based molecular tests, artificial intelligence (AI)/machine learning applications, COVID-19 EUAs, Breakthrough Designation for novel diagnostics, DTC requirements, and use of RWE to advance novel Dx and therapeutics.


 Speaker Profile

Ph.D., Chief Scientific Officer, IQVIA; Adjunct Professor, University of North Carolina at Chapel Hill

Biography
Her current work is focused on regulatory use of real-world evidence for decision making. She leads IQVIA’s Center for Advanced Evidence Generation, where they develop and implement innovative approaches to minimally interventional and non-interventional studies. She also directs a large community-based registry designed to study COVID-19. A Fellow of both the Drug Information Association (DIA) and the International Society of Pharmacoepidemiology, she is widely known for her thought leadership, particularly in terms of understanding when real-world evidence is fit for purpose. She received DIA’s 2019 Global Inspire Award for Author of the Year for “Advancing a framework for regulatory use of real world evidence: When real is reliable,” and is an elected member of DIA’s Scientific Advisory. She served for many years as a standing consultant to the National Football League Health & Safety Executive Committee, and continues to lead work on injury analytics for NFL and NBA.

Talk
Real-world Evidence Offers a Path to Precision Medicine
Advances in precision medicine will come from capturing and aggregating vast amounts of data about molecular markers, treatments and outcomes that can be used to shape insights about comparative treatment effectiveness and dosing schedules, but how do we know what evidence we can trust and when?


 Speaker Profile

Founder and Chairman, Rare Cancer Research Foundation

Biography
Mark Laabs is an entrepreneur who is a founder, advisor, and/or builder of a wide range of organizations which seek to address critical social and environmental problems on five continents. Mark, an ocular melanoma survivor, founded the RCRF to accelerate innovation in rare cancer research by creating shared infrastructure that drives scale and catalyzes research in these understudied and underserved areas. One of its key initiatives, www.pattern.org, enables patient-powered engagement with cancer research through the donation of fresh tissue vital to create disease models necessary to more fully understand each cancer and to identify potential new treatments. Mark is also focused on enabling precision medicine and trial enrollment opportunities for rare cancer patients regardless of location. Mark serves as Managing Partner of Modern Energy Group, with businesses focused on energy efficiency and resources in the United States and Brazil. Previously, he was COO of Soligent Holdings Inc., helped build and later sell Climate Bridge, a clean energy finance company, and was a consultant at McKinsey & Company. Mark graduated as a Robertson Scholar from the University of North Carolina at Chapel Hill and Duke University with a dual degree in International Studies and the Dynamics of Social Change


 Speaker Profile

Pharm.D., Associate Professor, University of Florida

Biography
Rhonda M. Cooper-DeHoff, Pharm.D., M.S., is a graduate of the University of California, San Francisco School of Pharmacy. Dr. Cooper-DeHoff’s area of research interest include hypertension and cardiovascular disease pharmacogenetics, as well as the factors that influence outcomes with antihypertensive and cardiovascular treatments. She also has interest in the area of antihypertensive drug use and adverse metabolic effects including diabetes, as well as the area of dietary supplements. In total, she has over 200 publications and many have been of high impact, helping to define demographic, clinical, metabolic, metabolomic and genetic and pharmacogenetic factors associated with responses to antihypertensive drugs and cardiovascular outcomes with antihypertensive therapy. She is currently conducting research in the area of ‘big data’ science including the use of EHR data from multiple health systems to conduct real world longitudinal surveillance of important blood pressure control and process metrics, as well as preemptive pharmacogenetics testing in multiple therapeutic areas.

Talk
Tracking BP Control Performance and Process Metrics in 25 US Health Systems
BP control (<140/90 mmHg), calculated using real-world data from 25 health systems across the US, averaged 62%, and there was substantial variation by health system (range 44%-74%). A new class of antihypertensive medication was prescribed in only 12% (range 0.6%-25%) of patient visits where BP was uncontrolled BP. When a medication intensification event occurred, subsequent SBP was 15 ± 20 mm Hg lower on average (range 5-18 mm Hg). Major opportunities exist for improving BP control and reducing disparities.


 Speaker Profile

CEO, Genetic Alliance

Biography
Sharon F. Terry is President and CEO of Genetic Alliance, an enterprise engaging individuals, families and communities to transform health. Genetic Alliance works to provide programs, products, and tools for ordinary people to take charge of their health and to further biomedical research. As ‘just a Mom’ with a master’s degree in theology, she cofounded PXE International, a research advocacy organization for the genetic condition pseudoxanthoma elasticum (PXE), in response to the diagnosis of PXE in her two children in 1994. She is the author of 150 peer-reviewed papers, of which 30 are clinical PXE studies. Her story is the topic of her TED Talk and TED Radio Hour. She has created people-centered technologies, tools, and resources, and holds patents. She was instrumental in getting the Genetic Information Nondiscrimination Act passed. She has many awards, including from the White House and the National Academies of Science, Engineering and Medicine.

Talk
Not Just Data: People as Drivers
Biomedical research has consistently ignored the people and been centered on their data. It is critical that people be not only at the center, but also driving the processes with other stakeholders. This radical shift is overdue. I will describe the solution Genetic Alliance, in partnership with Luna, has created.


 Speaker Profile

M.D., Professor of Medicine, Stanford University

Biography
Latha Palaniappan, MD, MS is Professor of Medicine and Scientific Director of Precision Genomics and Pharmacogenomics in Primary Care at Stanford. She is the faculty lead for the Stanford Biobank, a population based biobank designed to accelerate genetic and other -omics discovery.

Talk
How Are Patient Data Revolutionizing Precision Medicine?


 Speaker Profile

M.D., Chief Medical Officer, SVP, llumina

Biography
Dr. Febbo is responsible for developing and executing medical strategy to drive genomic testing into healthcare practice. Before joining Illumina, Dr. Febbo served as CMO of Genomic Health. Prior to his years at Genomic Health, Dr. Febbo was a Professor of Medicine and Urology at the UCSF, where his laboratory focused on using genomics to understand clinical behavior of prostate cancer, and his clinical practice focused on genitourinary oncology. Dr. Febbo holds an M.D. from UCSF. Completing his internal medicine residency at the Brigham and Women’s Hospital and fellowship in oncology at the Dana-Farber Cancer Institute. While an Attending Physician at Dana-Farber and Instructor at Harvard Medical School, he was a post-doctoral fellow at Dana-Farber and the Whitehead Institute Center for Genomic Research of MIT. In 2004, Dr. Febbo joined Duke University Medical Center’s Institute of Genome Sciences and Policy until his return to UCSF.


 Speaker Profile

Ph.D., VP, Commercial Markets, Tempus Inc.

Biography
Suzanne Belinson, PhD, MPH, is Vice President of Commercial Markets at Tempus Labs Inc. Tempus is a technology company building the world’s largest library of clinical and molecular data and an operating system to make that data accessible and useful, starting with cancer. Her role focuses on commercial growth for Tempus through the development and execution of partnerships with organizations aiming to improve healthcare value and outcomes by leveraging molecular and clinical data.


 Speaker Profile

Ph.D., M.P.H., VP Health Economics and Outcomes Research, OptumLabs at UnitedHealth Group

Biography
Stacey DaCosta Byfield currently leads the Genomics Health Economics and Outcomes Research (HEOR) Team focused on conducting observational research studies using administrative claims, EHR/medical record data and other data sources, including laboratory results data.  Stacey joined UnitedHealth Group (UHG) in 2009 in the OptumInsight Life Sciences HEOR group where she started as a Senior Researcher focusing on oncology studies.  She later served as VP, Research of the Life Sciences HEOR group before moving to OptumLabs were she led the Data Scientist team and most recently the Oncology Focus Area Research Team. Prior to joining UHG, she worked in the Department of Drug Use Policy and Pharmacoeconomics at the University of Texas M.D. Anderson Cancer Center as a Pharmacoeconomics Research Specialist. In this capacity, her main responsibilities included developing research designs to evaluate clinical, economic and humanistic outcomes of pharmaceutical products and services. She holds a PhD in Tumor Biology from Georgetown University and completed a basic science fellowship at the National Cancer Institute where her work focused on identifying targets of pharmaceutical intervention for metastatic breast cancer. Stacey also received her MPH with a concentration in clinical effectiveness from the Harvard T. H. Chan School of Public Health and has completed the Program in Cancer Outcomes Research Training (PCORT) Fellowship, a jointly sponsored program by the Massachusetts General Hospital and the Dana-Farber Harvard Cancer Center in Boston, Massachusetts.


 Speaker Profile

M.D., Ph.D., Professor of Pediatrics, Stanford University School of Medicine

Biography
Matthew Porteus is the Sutardja Clark Professor of Definitive and Curative Medicine in the Department of Pediatrics and Institute of Stem Cell Biology and Regenerative Medicine at Stanford. He is the co-Director for the Stanford Center for Definitive and Curative Medicine (CDCM). His primary research focus is on developing genome editing as an approach to cure disease, particularly those of the blood and immune system (including sickle cell disease) and other organ systems. His goal is to combine his research and clinical interests to develop innovative curative therapies. His dream is to one day develop gene editing so that patients are cured of their disease before they or their parents even knew they had it. He served on the 2017 National Academy Study Committee of Human Genome Editing and currently serves on the Scientific Advisory Board for WADA on Cell and Gene Doping and the NIH NExTRAC advisory committee evaluating the emergence of new technologies.


 Speaker Profile

Ph.D., Professor of Psychiatry and Biobehavioral Sciences, UCLA

Biography
Dr. Slavich is a leader in the conceptualization, assessment, and management of life stress, and in psychological and biological mechanisms linking stress with poor health. His current research uses methods from precision health and multi-omics to reduce disparities in human health and opportunity caused by stress. In addition to directing the Laboratory for Stress Assessment and Research at UCLA, Dr. Slavich is Director of the California Stress, Trauma, and Resilience Network, Co-chair of the National Task Force on Stress Measurement in Primary Care, Director of the Global Belonging Collaborative, and Senior Program Director for the UCLA/UCSF ACEsAware Family Resilience Network, which is supported by a $41.5 million grant from the State of California. His research has been published in leading scientific journals including Nature Medicine, Nature Communications, and JAMA Psychiatry, and has been covered by many media outlets such as The New York Times, Washington Post, The Guardian, The Atlantic, Scientific American, and others.

Talk
Cells-to-Society: A Precision Health Approach to Mitigating Toxic Stress


 Speaker Profile

Ph.D., Assistant Professor, University of California San Diego

Biography
For almost 20 years, Dr. William Kim has been leveraging the power of machine learning with functional genomics approaches to contribute to a deeper understanding of cellular circuitry underlying cancer initiation, maintenance, and progression. He has helped pioneer several novel computational framework to better functionally characterize cancers to identify appropriate therapeutic strategies towards realizing precision cancer medicine. He is currently the co-leader of a California Initiate to Advance Precision Medicine (CIAPM) Project sponsored by the State of California Office of Governor’s Planning and Research which aims to target Triple-Negative Breast Cancers to help reduce health disparities.


 Speaker Profile

J.D., Chief Legal and Regulatory Officer, 23andMe

Biography
Kathy is globally recognized for her effectiveness, having successfully pioneered the first ever FDA clearance for over-the-counter genetic testing, overcoming one of the most publicized FDA warning letters and enabling the relaunch and expansion of the company’s core products. She has successfully managed complex teams under regular and crisis conditions and has negotiated numerous transactions and private and public financings totaling more than $500m. She is active in key industry groups and is a frequent speaker on genomics, privacy and regulation. She sits on the board of Sophia Genetics, Inc. and served on the Board of Decipher Biosciences, Inc. prior to its merger with Veracyte, Inc. During her 20 years’ experience as an executive officer and corporate secretary for two public biotechnology companies, Genomic Health, Inc. and Monogram Biosciences, Inc., Kathy developed her passion for personalized medicine and the power of innovation to improve the lives of patients.


 Speaker Profile

General Manager, Data Business, Invitae

Biography
Farid Vij is General Manager for Invitae's Data division where he leads strategy, product and operations for the business. His core focus is building a two-sided platform that connects patients, providers and researchers through patient-consented data to help patients find better care options and advance research through real world evidence generation. Prior to Invitae, Farid was co-founder and COO at Ciitizen, a company focused on providing patients with access to their complete medical records. At Ciitizen, he drove the company's operations and go-to-market strategy, before leading its acquisition by Invitae in September 2021. Before Ciitizen, Farid held various commercial and operational roles at ZL Technologies, a big data company focused on harnessing unstructured information to drive critical privacy decisions for large organizations. Farid holds a BS in Economics from the Wharton School at the University of Pennsylvania. He enjoys angel investments in mission-focused companies and supporting education-focused non-profit organizations.


 Speaker Profile

M.D., Ph.D., Professor & Director, Stanford Cardiovascular Institute, Stanford University

Biography
Dr. Wu's works on cardiovascular genomics and induced pluripotent stem cells (iPSCs). The main goals are to (i) understand basic disease mechanisms, (ii) accelerate drug discovery and screening, (iii) develop “clinical trial in a dish” concept, and (iv) implement precision medicine for patients. Dr. Wu has published >450 manuscripts with H-index of 110 on Google scholar. He is listed as top 1% of highly cited researchers by Web of Science for past 4 years (2018, 2019, 2020, 2021). Dr. Wu serves on the FDA Cellular, Tissue, and Gene Therapies Advisory Committee. Dr. Wu is an elected member of Association of University Cardiologists (AUC), American Institute for Medical and Biological Engineering (AIMBE), American Association for the Advancement of Science (AAAS), American Association of Physicians (AAP), and National Academy of Medicine (NAM).


 Speaker Profile

Senior User Experience Researcher, LunaPBC

Biography
Focusing on developing participant-focused research methods, surfacing priorities, needs, hierarchies, and categories through the mental models of people directly affected by clinical and diagnostic research. Ian has a long history in Human-centered research and advocacy, diagnosed with a rare disease at 5 years old he has worked his whole career to bring together the shared experience of participants and the proven methods of product development.

Talk
Computational Social Choice Theory's Impact on the Collection and Understanding of Real-World Data
The fields of Computational Social Choice, UX Research, and product development have vastly outpaced the medical field in their ability to collect, categorize, and understand real-world data points to improve the lives of their "users." By implementing these lessons into the clinical landscape we can leap-frog years of systems growth and evolution without having to re-invent the wheel. For RWE to be effective, it must be representative of people's real lives, in their living rooms and around their dining tables, growing at the same pace as real life. How exactly can this be done? Case studies and an explanation of novel methodologies will pave the way.