Spotlight Sessions

* Program subject to change

Monday, 16 November15:00 - 16:00Spotlight SessionScientific Collaboration in the Face of a Pandemic

Given the medical emergency presented by COVID-19 and the importance of international cooperation to tackle this global crisis, FDA, EMA, and the European Commission intensified their collaboration efforts under the umbrella of the International Coalition of Medicines Regulatory Authorities (ICMRA) - an international executive-level coalition that brings together key regulators from every region of the world to facilitate access to safe, effective, high-quality products. Sharing of experience and common challenges in facilitating the development, review and availability of COVID-19 vaccines; review and availability of COVID-19 vaccines, collaborating on observational research in COVID-19 as a model, specifically on vaccines surveillance; building international cohorts; and the use of medicines in pregnant women with COVID-19 are among the current priority topics of common interest. What does this mean for outcomes researchers as they look to add and aid in the developing evidence in the pandemic space? Join this spotlight session to learn about the latest progress in joint efforts by national regulatory authorities and discuss what more we could and should be doing to ensure robust scientific evidence for COVID decision making.

Moderators

Ian Hudson, OBE, MBBS, MD, FRCP, FFPM

Bill and Melinda Gates Foundation, London, United Kingdom

Dr Ian Hudson practiced as a paediatrician for a number of years before working in research and development at SmithKline Beecham for 11 years. Subsequently, in 2001, he joined the MHRA as Director of Licensing and was the UK delegate to EMA’s scientific committee, CHMP, latterly its Vice Chair. In 2013 Dr Hudson became CEO of the MHRA, also a member of the EMA management Board and part of the Heads of Medicines Agencies Management Group. He was also Chair of the International Coalition of Medicines Regulatory Authorities between 2016 and 2019. Dr Hudson joined the Bill and Melinda Gates Foundation as Senior Advisor, Regulatory Affairs, Integrated Development in September 2019. He was awarded an OBE for Services to Healthcare in the New Year’s honours 2020.

Speakers

Peter Marks, MD, PhD

U.S. Food and Drug Administration (FDA), Silver Spring, MD, USA

Peter Marks received his graduate degree in cell and molecular biology and his medical degree at New York University and completed Internal Medicine residency and Hematology/Medical Oncology training at Brigham and Women’s Hospital in Boston. He has worked in academic settings teaching and caring for patients and in industry on drug development. He joined the FDA in 2012 as Deputy Center Director for CBER and became Center Director in January 2016.

Pierre Meulien, PhD

Innovative Medicines Initiative (IMI), Brussels, NJ, Belgium

Pierre Meulien is executive director of the Innovative Medicines Initiative (IMI), a €5 billion public-private partnership between the European Union and the European pharmaceutical industry. At IMI, he is responsible for the overall management of the program, which works to improve and accelerate the drug development process by facilitating collaboration between the key players involved in health research. Previously, Dr Meulien was president and CEO of Genome Canada, where he raised money and oversaw the launch of novel projects and networks in the field of genomics-based technologies. Prior to that, he was chief scientific officer for Genome British Columbia and was the founding CEO of the Dublin Molecular Medicine Center. Dr Meulien also worked with the French biotechnology company Transgene and with Aventis Pasteur (now Sanofi Pasteur). He has a Ph.D. in molecular biology from the University of Edinburgh and carried out a postdoctoral fellowship at the Institut Pasteur in Paris.

Agnes Saint-Raymond, MD

European Medicines Agency (EMA), Amsterdam, NJ, Netherlands

Agnes is an MD and qualified French Paediatrician. She worked as a paediatrician in a paediatric Hospital in Paris, France, then in pharmaceutical industry, and then moved to the French Medicines Agency. In 2000 she joined the European Medicines Agency (EMA) and was responsible for Orphan Medicines, Scientific Advice, the Small & Medium-sized Enterprises Office, and Paediatric Medicines Sector, implementing the EU Regulation on paediatric medicines. In 2013, she became Head of the Portfolio Board, providing oversight of projects for EMA. Since November 2016, she is also the EMA Head of the International Affairs Division.

Tuesday, 17 November17:00 - 18:00Is Credible, Robust Real-World Evidence Just a Myth?

It is becoming very clear that real world evidence (RWE) is a necessary tool in our armament of evidence-based medicine and the pandemic has shown that clinical data if often the first line of defense in a public health emergency. However, we also know that there many decision makers are still skeptical that RWE is reliable and credible. This session focuses on issues regarding RWE and why there are still perceived and real credibility hurdles including data curation and trust of the underlying data or the ability for the study design and planned analysis to make valid causal inferences, and how to ensure that reviewers are well placed to evaluate the quality of RWE studies. This panel will discuss the issues from each their various viewpoints and what they see as ways to improve the perception and credibility of RWE.

Moderators

Anja Schiel, PhD

Norwegian Medicines Agency, Oslo, Norway

Anja Schiel has studied Biology at the Johannes Gutenberg-University, Mainz, Germany. She received her PhD from the Free University in Amsterdam in 2006 and worked several years as Post-Doc on a range of subjects focusing on oncology, immunology and molecular biology, first at the University of Leiden and later at the University of Oslo, before starting at the Norwegian Medicines Agency (NoMA) in 2013. At NoMA she is working as special adviser/Statistician/Methodologist both on regulatory (EMA) and HTA projects. She has been Chair of the Biostatistics Working Party 2017 - 2019 and is currently the Chair of the Scientific Advice Working Party at EMA. In addition, she has been heavily involved in EUnetHTA activities, with focus on parallel EMA-HTA scientific advices as Norway is member in EUnetHTAs Early dialogue working party. This explains her particular interest in use of methodology and statistics in pharmaco-economic evaluations. She is furthermore involved in several IMI projects as member of their Scientific Advisory boards.

Speakers

Nancy Dreyer, MPH, PhD

IQVIA, Cambridge, MA, USA

Nancy Dreyer is Senior Vice President and Chief Scientific Officer for Real World Solutions at IQVIA, and Adjunct Professor of Epidemiology at the University of North Carolina at Chapel Hill. She leads the IQVIA Center for Advanced Evidence Generation. A fellow of both the International Society for Pharmacoepidemiology and DIA, her current work is focused on COVID-19 along with other issues of regulatory and public health importance. Recent recognition includes the Red Jacket honor from PharmaVOICE in 2020 and DIA’s Global Inspire Award for Author of the Year in 2019. She is also celebrating recent publication of the 4th edition of the popular book “Registries for Evaluating Patient Outcomes: a User’s Guide.”

Robert Golub, MD

JAMA, Chicago, IL, USA

Robert M. Golub, MD, is Deputy Editor, JAMA. His roles include oversight of the JAMA scientific content and managing the peer review process; he is also responsible for directing JAMA educational activities. He is Associate Professor of Medicine at the Feinberg School of Medicine at Northwestern University, with academic appointments in the Division of General Internal Medicine and the Department of Preventive Medicine. He served as chair of the Northwestern University Medical School Curriculum Committee. Areas of research are in medical decision making (decision analysis, cost-effectiveness analysis, psychology of decision making, and assessing patient preferences). He has served on the Board of Trustees for the Society for Medical Decision Making and as visiting faculty for the Stanford University Faculty Development Program and the University of Buenos Aires Program in Clinical Effectiveness. Dr. Golub received his undergraduate degree from Princeton University, and his MD from Columbia University College of Physicians and Surgeons. He completed his internship and residency at Northwestern University School of Medicine/Northwestern Memorial Hospital, where he also served as chief resident. He is board certified in internal medicine.

Xavier Kurz, MD PhD

European Medicines Agency, Amsterdam, Netherlands

Xavier Kurz graduated in 1982 as a Medical Doctor at the University of Liege, Belgium. He specialised in Tropical Medicine and worked for several years in public health projects in Africa and Asia. He obtained a MSc (1991) and a PhD (1997) in Epidemiology and Biostatistics at McGill University, Montreal, Canada. He then joined the Department of Pharmacology of the University of Liege and the Belgian Centre for Pharmacovigilance (Ministry of Health) as scientific expert. He joined the European Medicines Agency (EMA) on 1st September 2005. He is currently Head of Data Analytics within the Data Analysis and Methods Task Force.

Thursday, 19 November14:30 - 15:45Novel Methods for Modeling Treatment Sequencing

This 75-minute session will present new methodological research for the modeling of sequential treatment decisions. Actual clinical practice often involves sequential treatment decisions that create modeling considerations more challenging than those typically encountered in more standard single pairwise comparison models. For example, the sequential decisions make cohort Markov modeling very difficult because they introduce problems in developing the controlling equations to take proper account of the conditionalities involved. Also, propagating uncertainty and exploring its impact in PSA cannot follow standard approaches. There are also methods issues relating to estimating parameters that are appropriate to a place in the sequence or pathway. These research presentations will be more in-depth than typical research podium sessions and will include time for moderated audience discussion.

Moderators

Mark Sculpher, PhD

University of York, York, YOR, United Kingdom

Mark Sculpher is professor of Health Economics at the Centre for Health Economics, University of York, UK where he leads the Centre’s Programme on Economic Evaluation and Health Technology Assessment. He is also co-director of the Policy Research Unit in Economic Evaluation of Health and Care Interventions, a seven-year programme, run collaboratively with the University of Sheffield and funded by the UK Department of Health. Mark has worked in the field of economic evaluation and health technology assessment for over 30 years. He has researched in a range of clinical areas including heart disease, cancer, diagnostics, and public health. He has also contributed to methods in the field, in particular relating to decision analytic modeling and techniques to handle uncertainty, heterogeneity, and generalisability. He has over 250 peer-reviewed publications and is a co-author of two major text books in the area: Methods for the Economic Evaluation of Health Care Programmes (OUP, 2015 with Drummond, Claxton, Torrance and Stoddart) and Decision Modeling for Health Economic Evaluation (OUP, 2006 with Briggs and Claxton). Mark is an emeritus member of the UK National Institute of Health Research (NIHR) College of Senior Investigators. He has also been a member of the National Institute for Health and Clinical Excellence (NICE) Technology Appraisal Committee and the NICE Public Health Interventions Advisory Committee. He currently sits on NICE’s Diagnostics Advisory Committee. He chaired NICE's 2004 Task Group on methods guidance for economic evaluation and advised the Methods Working Party for the 2008 update of this guidance. He has also advised health systems internationally on HTA methods including those in France, Ireland, Japan, Singapore, Germany, Portugal, and New Zealand. He has been a member of the Commissioning Board for the UK NHS Health Technology Assessment Programme, the UK NIHR /Medical Research Council’s Methodology Research Panel, and the UK Department of Health’s Policy Research Programme’s Commissioning Panel. He served as President of ISPOR for the 2011-12 term.

Speakers

Maiwenn J Al, PhD

Erasmus University Rotterdam, Rotterdam, Netherlands

Dr. Maiwenn Al is associate professor of Modelling in HTA at Erasmus School of Health Policy & Management, Rotterdam, the Netherlands. Her research focus is on modelling studies and methods for dealing with uncertainty in economic evaluations. Together with Ben van Hout, Maiwenn developed the cost-effectiveness acceptability curve, now widely used to describe uncertainty around ICERs. She was the 2006 recipient of the ISPOR Health Economics Outcomes and Research - Methodology Award, for the paper she wrote with Jan Oostenbrink on the analysis of incomplete cost data due to dropout. From 2012=2016, Maiwenn was co-editor of Value in Health. Until recently, Maiwenn was a member of the Scientific Advisory Board of the National Health Care Institute (ZIN) in the Netherlands. She was also on the committee for the revision of the Dutch guidelines for economic evaluations in healthcare. Maiwenn has been health economic lead to many technology appraisals for NICE in the UK. Besides this work for NICE and various teaching activities, her current work targets the use of real-world evidence for the health economic assessment of medical devices (H2020 project COMED).

Huajie Jin, MBBS, MSc

King’s College London, London, United Kingdom

Dr Huajie Jin (Lily) has been a Senior Health Economist at King’s College London since 2013. Her role at King’s involves designing and conducting economic evaluations for a range of policymakers and research organisations, including the NHS England, the National Institute for Health and Clinical Excellence (NICE), the National Institute for Health Research (NIHR), Medical Research Council (MRC), as well as voluntary sector and commercial organisations. Before joining King’s, Lily used to work on the National Institute for Health and Clinical Excellence (NICE) clinical guidelines for three years. Between the Year 2011-2013, Lily was an honorary Research Fellow of Cardiff University, and an Honorary Research Associate of Swansea University. Lily’s research focuses on health economic modelling, trial-based economic evaluations and systematic reviews. She has conducted economic evaluations for a range of healthcare interventions, including screening tests, diagnostic tests, drugs, radiotherapies, haemodialysis and service-level interventions, such as training for GPs, and liaison modes between primary care and secondary care services. Her work has been published in journals such as “The Lancet Psychiatry”, “JAMA Network Open”, “PharmacoEconomics” and “PLOS One”. She was the principal health economist of three NICE clinical guidelines and three NICE Medtech innovation briefings (MIBs).

James Robins, MD

Harvard T.H. Chan School of Public Health, Boston, MA, USA

Virtual ISPOR Europe 2020

16-19 November 2020

Spotlight Sessions

The Professional Society for
Health Economics and Outcomes Research