Program

* Program subject to change

Sun May 712:00 PM - 4:00 PMRegistration Hours1:00 PM - 1:10 PMWelcome1:10 PM - 2:10 PMSession 1From Data Quality to QualitiesLevel: Experienced

This session will examine how data quality is described and measured. It will include a review and discussion of recent data quality frameworks, developments in validation, and measurement of important aspects of quality. It will include presentations from several experts followed by a brief panel discussion and Q&A.

Moderators

Rachele Hendricks-Sturrup, DHSc, MSc, MA

Duke-Margolis Center for Health Policy, Washington, DC, USA

Speakers

Andre Araujo, PhD

GSK, Kennett Square, PA, USA

Andre B. Araujo, PhD is an epidemiologist and is the Head of Real-World Analytics (RWA) at GSK. The RWA team is composed of analysts and data scientists who provide design and analytics to support the generation of high-quality real-world evidence in support of the GSK portfolio. His external engagements include initiatives that focus on best practices in the application of research methods to real-world data in the context of non-interventional study designs.

Jaclyn L. Bosco, PhD, MPH

IQVIA, Cambridge, MA, USA

Dr. Jaclyn Bosco is Vice President and Global Head of Epidemiology at IQVIA. She is responsible for driving real world evidence generation for regulators, clinicians, patients and payers using primary or passive data collection as well as existing data to support the safety and effectiveness of drugs, biologics, and medical devices from early clinical development through the post-approval phase. She sets industry best practices through external thought leadership as an invited speaker and panelist at international congresses and conferences on topics such as generating fit-for-purpose solutions using real-world evidence for regulatory decision-making.

Sebastian Schneeweiss, MD, ScD

Harvard Medical School; Brigham and Women’s Hospital, Boston, MA, USA

2:10 PM - 3:10 PMSession 2Considering Data Qualities in Determining FFP - Can We Converge on an Approach?Level: Experienced

This session will be focused on the principles and realities of how fit-for-purpose decisions are made based on the needs of the study question and the qualities of the data. It will be primarily a panel discussion, considering questions such as “How does the study question interact with data qualities to determine what’s fit-for-purpose”?

Moderators

Marc Berger, MD

Marc L. Berger, LLC, New York, NY, USA

Marc L. Berger, MD, is a semi-retired, part-time consultant and scientific advisor. Until July 2017, he was Vice President, Real World Data and Analytics at Pfizer, Inc. Marc has held senior-level positions in industry including Executive Vice President and Senior Scientist at OptumInsight; Vice President, Global Health Outcomes at Eli Lilly and Company; and Vice President, Outcomes Research and Management at Merck & Co., Inc. He was a temporary employee of CMS from July-December 2022. Marc is an ISPOR Special Advisor for Real World Evidence and a member of the Duke-Margolis Center for Health Policy Real-World Evidence Collaborative. He has written or co-written more than 130 peer-reviewed articles, book chapters, and other publications on a range of topics including health services research, outcomes research, health economics, and health policy. He received the Donabedian Lifetime Achievement Award from ISPOR in 2019.

Speakers

John Concato, MD, MS, MPH

Food and Drug Administration, Silver Spring, MD, USA

John Concato, MD, MS, MPH, is an Associate Director of the Office of Medical Policy, Center for Drug Evaluation and Research, FDA. His responsibilities related to real-world evidence (RWE) include developing internal Agency processes, interacting with stakeholders, coordinating demonstration projects and guidance development, and serving as Chair of the RWE Subcommittee. He joined FDA in 2019 after a career at Yale School of Medicine and the US Department of Veterans Affairs, where he was a clinician, educator, independent investigator, research center director, and Professor of Medicine.

Gracy Crane, PhD

Roche Products Limited, Welwyn, HRT, United Kingdom

Gracy Crane is a cancer biologist by training with a Master in Biomedical Research and a Ph.D. in Cancer Biology. She has also completed post-doctoral training fellowships at Oxford University (UK) and Massachusetts Institute of Technology (USA). She has over 12 years of years of experience in the pharmaceutical industry, in varying roles including medical and scientific affairs, clinical development, health outcomes research and real world data. For the past 5 years, she has worked on leading the real world evidence strategy for molecules within the rare cancer space as part of the global Data Science team at Roche. Currently, she is the RWD policy lead within the Global Regulatory Policy team at Roche. She has authored many publications on this topic and has presented in many international conferences. Gracy is also part of a number of external steering/working groups including the ISPOR RWD transparency Initiative (https://www.ispor.org/strategic-initiatives/real-world-evidence/real-world-evidence-transparency-initiative), the CIOMs working group on RWD/E guidelines, and the Duke Margolis RWE Collaborative. She is also a co-author on the HARPER work: https://onlinelibrary.wiley.com/doi/full/10.1002/pds.5507 This is her LinkedIn Profile: https://www.linkedin.com/in/gracy-crane-374ba120/

Laurie Lambert, PhD

Canadian Agency for Drugs and Technologies in Health, Ottawa, ON, Canada

Laurie Lambert completed a Master of Public Health in the epidemiology and biostatistics stream at the University of Western Australia. She then received a four-year Australian National Health and Medical Research Training scholarship to pursue her PhD. From 2004 to 2020, Laurie was the scientific coordinator of the Cardiovascular Evaluation Unit at INESSS where the focus was on evaluations of new technologies in the real-world context. In August, 2020 Laurie began a new chapter in her career as Lead of Real World Evidence at CADTH.

Daniel Leaver Morales

EMA, Amsterdam, Netherlands

3:10 PM - 3:30 PMBreak3:30 PM - 4:30 PMSession 3Transparency in RWE: Ensuring Credibility and ConfidenceLevel: Experienced

This session will cover areas of good conduct for RWE studies that help decision-makers have more confidence in the credibility of their results. They include use of standardized protocol templates, pre-registration of study protocols, and moving towards master protocols, with reference to specific tools that are available or in development.

Moderators

Richard Willke, PhD

ISPOR, Lawrenceville, NJ, USA

Dick became ISPOR’s first chief science officer in April 2016, following nearly 25 years in the pharmaceutical industry with Pfizer and its legacy companies. In his CSO role at ISPOR, Dick’s responsibilities are to develop, lead, support, and direct strategic initiatives related to research, scientific, and content priorities to accomplish the organization’s mission to promote health economics and outcomes research excellence to improve decision making for health globally. While with Pfizer, his final position was Vice President, Outcomes & Evidence, lead for Cardiovascular /Metabolic, Inflammation & Immunology, the last in a succession of HEOR group lead roles. He received a PhD in economics from Johns Hopkins University in 1982, concentrating in econometrics and labor economics. Prior to joining Pfizer’s legacy company Upjohn in 1991, he was a member of the economics faculty at Ohio State University as well as a senior economist at the American Medical Association Center for Health Policy Research. Dick has served on the ISPOR Board of Directors (2007-09), was chair of the ISPOR Institutional Council in 2010, and was co-chair of the ISPOR Good Research Practices Task Force on Cost-Effectiveness Analysis in Randomized Clinical Trials in 2003-2005 as well as its 2014-15 reprise to revise and update that Report. He has co-taught many ISPOR short courses on this topic as well as on “Transferability of Cost-Effectiveness Data between Countries.” He was also a member of the Health Outcomes Committee of PhRMA from 1998-2009, having been its chair from 2002-2004. He has served as a co-editor for Value in Health, on the editorial board for Farmeconomia, on AHRQ, NIH, and PCORI project review study sections, and is a member of the Ohio State University Economics Advisory Board. Prior to joining industry, Dr Willke served as department director in the Center of Health Policy Research at the American Medical Association and held research and teaching positions at The Ohio State University. Dr Willke earned a PhD and MA in economics from Johns Hopkins University. He has authored more than 80 scholarly publications that examine the science and methodologies of health economics and outcomes research.

Speakers

Adam Aten, MPH, MSc

Duke-Margolis Center for Health Policy, Bethesda, MD, USA

C. Daniel Mullins, PhD

University of Maryland School of Pharmacy, Baltimore, MD, USA

C. Daniel Mullins, PhD is a professor at the University of Maryland School of Pharmacy. He received his BS in Economics from M.I.T. and his PhD in Economics from Duke University. His research and teaching focus on comparative effectiveness research (CER), patient-centered outcomes research (PCOR), pharmacoeconomics, and health disparities research. He directs the University of Maryland PATient-centered Involvement in Evaluating effectiveNess of TreatmentS (PATIENTS) Program, which received a University of Maryland Baltimore Champion of Excellence Award. He has received funding as a Principal Investigator from the NIH/NIA, NIH/NHLBI, AHRQ, and the Patient-Centered Outcomes Research Institute (PCORI) and was the Shared Resources Core Director for the NIH-sponsored University of Maryland Center for Health Disparities Research, Training, and Outreach.

Shirley Wang, PhD, MSc

Brigham and Women’s Hospital, Harvard Medical School, Boston, MA, USA

Dr. Wang is an Associate Professor at Brigham and Women’s Hospital, Harvard Medical School. She has led 2 joint task forces between ISPOR and the International Society of Pharmacoepidemiology (ISPE) focused on real-world evidence for healthcare decision-making. Dr. Wang directs the REPEAT Initiative, a non-profit program with projects aimed at improving transparency, reproducibility and robustness of evidence from healthcare databases and co-leads RCT-DUPLICATE, a series of projects designed to inform when and how real-world data analyses can draw causal conclusions to inform regulatory decision-making.

4:30 PM - 5:30 PMSession 4Navigating the RWE Landscape - Successes, Struggles, and the Path ForwardLevel: Experienced

This session will include review of specific cases of RWE use for regulatory or HTA purposes, with consideration of both data and analytic issues. The discussion will What has worked, what the challenges have been, and what lessons can be drawn for the future.

Moderators

Nancy Dreyer, MPH, PhD, FISPE

IQVIA Real World Solutions, Newton, MA, USA

Nancy Dreyer is Adjunct Professor of Epidemiology at the University of North Carolina at Chapel Hill, a Trustee of Brandeis University and Chief Scientific Officer Emerita at IQVIA Real World Solutions. Well-known for her thought leadership, she is a Fellow of both the International Society of Pharmacoepidemiology and the Drug Information Association. She has helped advance the use of real-world evidence for regulatory purposes, influencing the content of recent guidelines by regulators in the US, Europe, and China, each of which cite one or more of her publications. Her substantial executive and field experience have helped hone her pragmatic views about real-world evidence.

Speakers

Stephen Duffield, PhD, MD

NICE, Liverpool, United Kingdom

Stephen is an Associate Director at NICE. His role involves the continuing development of NICE’s real-world evidence (RWE) framework, collaboration on RWE demonstration projects, and helping to transform NICE’s use of real-world data across guidance products. He is also involved with upskilling individuals within and externally to the organisation, contributing to training workshops and technical forums. Stephen has a degree in medicine and a PhD in public health. Previously, he worked as a clinical doctor and a guideline developer in NICE Centre for Guidelines

Wim Goettsch, PhD

National Health Care Institute (ZIN); Utrecht University, Division of Pharmacoepidemiology and Clinical Pharmacology, Diemen, Netherlands

Wim Goettsch, PhD is currently Special Advisor Health Technology Assessment (HTA) at the Dutch National Health Care Institute. Since December 2022, he also has a position as a Professor and Chair on HTA of pharmaceuticals at Utrecht University (NL) where he is leading an H2020 consortium with fifteen partners around Europe, called HTx, new methods for Health Technology Assessment (2019-2024). He was the Director of the EUnetHTA JA3 (2016- 2020) Directorate and Chair of the Executive Board of EUnetHTA between June 2016 and March 2018. He is iinvolved many activities on real world evidence (RWE) such as the GetReal Institute, the ISPE-ISPOR taskforce on RWE transparency, CIOMS WG XII on RWE and the EU stakeholder Advisory Board on DARWIN-EU. He has more than 110 publications in peer-reviewed international journals.

Ashley Jaksa, MPH

Aetion, Inc, Boston, MA, USA

Ashley Jaksa is Market Access Scientific Strategy Lead at Aetion. She focuses on collaborating with decision-makers to enhance their use of RWE and setting methodological guidance for RWE generation. She also works with Aetion’s biopharma clients on incorporating RWE in their global market access strategy. She has presented her research at numerous international conferences including ISPOR, HTAi, and AMCP, and published her findings in various peer-reviewed publications. She holds a bachelor's degree from the University of Michigan-Ann Arbor and a MPH from Yale University.

Tae Hyun Jung, PhD

Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD, USA

Tae Hyun (Ryan) Jung, Ph.D., is a Senior Statistical Reviewer in the Office of Biostatistics at CDER, FDA. He has comprehensive review experience in phase III reviews including RWE efficacy and phase IV registry/database safety reviews. In addition, he works on clinical research that leverages CMS data and leads RWD projects using machine learning in causal inference and in synthetic data. Tae Hyun joined the FDA in October 2017 after completing his Ph.D. in Biostatistics from Yale University.

5:30 PM - 5:40 PMClosing Remarks5:40 PM - 6:30 PMNetworking Reception

ISPOR Real-World Evidence Summit 2023

May 7, 2023

Program

ISPOR–The Professional Society for
Health Economics and Outcomes Research