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
    Session Chair: Atul Butte, UCSF
    - James Wall, Stanford
    - Nancy A. Dreyer, IQVIA
    - Mark Laabs, RCRF
    - John Concato, FDA
    - Rhonda Cooper-DeHoff, University of Florida
  • Opportunities and Challenges in Using Real World Data (RWD)
    Session Chair: Vivek Rudrapatna, UCSF
    - Kathy Giacomini, UCSF
  • How Are Patient Data Revolutionizing Precision Medicine?
    Session Chair: Clara Lajonchere, UCLA
    - Sharon Terry, Genetic Alliance
    - Farid Vij ,Ciitizen
    - Latha Palaniappan, Stanford
  • Regulatory Requirements and Challenges for Using RWE
    Session Chair: Sheila Walcoff, Goldbug Strategies
  • Government Partnerships: California Initiative to Advance Precision Medicine
    Session Chair: Julianne McCall, California Initiative to Advance Precision Medicine
    - Pablo Tamayo, UCSD
    - George M. Slavich, UCLA
  • Leveraging RWE to Create Value
    Session Chair: Kathryn A. Phillips, UCSF
    - Phil Febbo, Illumina
    - Suzanne Belinson, Tempus
    - Stacey Dacosta Byfield, OptumLabs
  • The Past, Present, and Future of RWE
    Session Chair: Keith Yamamoto, UCSF
    - Matthew Porteus, Stanford

 Session Chair Profile

Ph.D., Vice Chancellor for Science Policy and Strategy, UCSF; Director, UCSF Precision Medicine; Professor, Cellular & Molecular Pharmacology, UCSF

Biography
Dr. Yamamoto is a leader in science and public policy. He has made an indelible impact by simultaneously advocating for Precision Medicine across the .edu, .gov, .com and .org sectors. As Chair of the National Academies Board on Life Sciences, he appointed and served on the study committee that produced “Toward Precision Medicine: Building a Knowledge Network for Biomedical Research and a New Taxonomy of Disease”, the report that enunciated the precision medicine concept. He helped to stimulate President Obama’s interest, which led to the Precision Medicine Initiative, as well as Gov. Jerry Brown’s launch of the California Initiative to Advance Precision Medicine. He also promoted a precision medicine approach to Vice President Biden’s Cancer Moonshot, provoked broader participation by the corporate and nonprofit sectors, and directs UCSF Precision Medicine, an institution-wide imperative. In addition, Dr. Yamamoto has led or served on numerous federal or national committees focused on public and science policy, public understanding and support of biological research, research funding and peer review, and science education and the biomedical workforce; he chairs the Coalition for the Life Sciences, and sits on the National Academy of Medicine Council and Executive Committee, and the National Academy of Sciences Division of Earth and Life Studies Advisory Committee. He is a member of the Advisory Board for Lawrence Berkeley National Laboratory and the Board of Directors and Executive Committee of Research!America. At UCSF, Dr. Yamamoto is vice chancellor for science policy and strategy and professor of cellular and molecular pharmacology. He is a leading researcher, investigating transcriptional regulation by nuclear receptors, which mediate the actions of essential hormones and cellular signals; he uses mechanistic and systems approaches to pursue these problems in pure molecules, cells and whole organisms. He is an elected member of the National Academy of Sciences, the National Academy of Medicine, the American Academy of Arts and Sciences, and the American Academy of Microbiology, and a fellow of the American Association for the Advancement of Science.


 Session Chair Profile

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

Biography
Dr. Butte is 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 medical schools, 6 academic health centers, and 10 hospitals. Dr. Butte has been continually funded by NIH for 20 years, 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 in 2013, he was recognized by the Obama Administration as a White House Champion of Change in Open Science for promoting science through publicly available data. Dr. Butte is also a founder of three investor-backed data-driven companies: 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 through open molecular data.


 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

Biography
Clara Lajonchere is the Deputy Director for the UCLA Institute for Precision Health, Co-Director of the Clinical Neurogenomics Research Center 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. Prior to UCLA, Dr. Lajonchere held faculty appointments at the USC Keck School of Medicine where she served as Principal Investigator and Director of the NIH Center for Genomic and Phenomic Studies in Autism and conducted research on models for inclusion of underserved populations in biomedical research. She also served as VP of Clinical Programs for Autism Speaks for over a decade where she oversaw a diverse portfolio of basic science and clinical programs. She currently consults for several tech start-ups in the autism space focused on AI, robotics, and targeted therapeutics.


 Session Chair Profile

Ph.D., Professor of Health Economics and Founding Director, UCSF Center for Translational and Policy Research on Personalized 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
Goldbug Strategies is the leading boutique FDA/CLIA professional services company retained by the most innovative clinical laboratories, CDx drug developers, software & digital health companies, and biotech investors. As an FDA regulatory strategist and LDT/IVD policy expert with over 25 years of experience, Ms. Walcoff is widely recognized for her skill in integrating her regulatory, legislative, and policy expertise in the field of personalized medicine and molecular diagnostics to identify novel regulatory pathways for innovators. Ms. Walcoff is a former Associate Commissioner at the FDA and served as the Senior Counselor for Science and Public Health Policy to former HHS Secretary Michael O. Leavitt. Ms. Walcoff was a partner in the law firm of McDermott Will & Emery LLP and served as majority counsel to a US House of Representatives Committee. She earned her law degree from Georgetown University Law Center.


 Speaker Profile

M.D., Associate Professor of Pedaitric Surgery, Stanford University

Biography
James Wall is an Associate Professor and Pediatric Surgeon at Stanford University who focuses on minimally-invasive approaches to children’s surgery. He is a physician entrepreneur who has developed multiple health technologies. James teaches and enables life sciences innovation as the co-director of the Stanford Biodesign Innovation Fellowship and the UCSF-Stanford Pediatric Device Consortium.


 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

M.D., M.P.H., Associate Director for Real-World Evidence Analytics, Office of Medical Policy, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

Biography
Trained as an internist and epidemiologist, Dr. Concato is Associate Director for Real-World Evidence Analytics in the Office of Medical Policy (OMP) at the Center for Drug Evaluation and Research (CDER), US Food and Drug Administration. In seeking to enhance policies related to drug development and regulatory review in CDER, his responsibilities include a focus on real-world evidence (RWE) and involve work developing internal Agency processes, interacting with external stakeholders, supporting RWE demonstration projects and guidance development, and serving as the Chair of the RWE Subcommittee. Prior to joining FDA, his career focused on generating research as an independent investigator and research center director at Yale University School of Medicine and the U.S. Department of Veterans Affairs (VA), including serving as one of two founding principal investigators of the VA Million Veteran Program genomic mega-biobank. He received M.D. and M.S. degrees from New York University and an M.P.H. degree from Yale University.


 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

Ph.D., Professor, UCSF

Biography
Kathy Giacomini, a professor at the University of California, San Francisco, is a leader in the field of membrane transporters with a focus on genetic polymorphisms. She cloned, characterized and discovered the endogenous role of the human xenobiotic transporter, OCT1 (SLC22A1), and recently de-orphaned SLC22A24 and SLC22A15, discovering physiologic and pharmacologic substrates of both transporters. Together with others, she co-founded the International Transporter Consortium, which has published highly impactful papers informing regulatory policy. She is the Co-Principal Investigator of the UCSF-Stanford Center of Excellence in Regulatory Sciences and Innovation, funded by the Food and Drug Administration. She has received numerous awards including an honorary doctorate degree from Uppsala University, and is an elected member of the National Academy of Medicine.

Talk
Use of Real World Evidence in Regulatory Sciences
I will describe the UCSF-Stanford Center of Excellence in Regulatory Science and Innovation, which provides a forum for collaborative research between faculty and FDA scientists. I will focus on research that makes use of RWE to monitor safety and efficacy of diverse medical products including drugs and biologics.


 Speaker Profile

Co- founder, President & 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, the National Academies of Science, Engineering and Medicine.


 Speaker Profile

Founder, Ciitizen

Biography
Anil Sethi is the CEO of Ciitizen, a Silicon Valley startup helping patients collect, organize, and securely share all their digital health data—converting text narratives, into computable data.  acquired Sethi’s consumer Health Records company Gliimpse in 2016. Apple released its Health Records feature in 2018. Anil served as a Director of Apple Health until leaving in 2017 to take care of his little sister Tania, who died soon after from Metastatic Breast Cancer. Amongst his previous five healthcare ventures, he’s had one failure, three exits (WebMD, Citrix, Apple) and a Nasdaq IPO.


 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., MPH, Vice President Commercial Markets, Tempus Inc.

Biography
In her current role, Suzanne focuses on commercial strategy and growth for Tempus developing and executing partnerships with organizations to improve healthcare value and outcomes by leveraging molecular and clinical data. Value creation for commercial payers, benefit managers and group purchasing organizations is where she focuses. Dr. Belinson is especially interested in applications of data to power solutions for the transition to and management of value based care. Prior to Tempus, Dr. Belinson served as Executive Director, Clinical Markets, at the Blue Cross Blue Shield Association where she led the sales and market development as well as day to day operations of Evidence Street. Dr. Belinson received her bachelor’s degree from Cleveland State University, a Masters in Public Health from the University of Pittsburgh, and a PhD in Epidemiology from the University of North Carolina at Chapel Hill.


 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.