Mon 16 Nov
13:00 - 14:30
PLENARY SESSION
“New Deal” for Healthcare Systems: Mission Inspirational, Impossible, or Inevitable?
Scientific advances in the understanding of disease biology and healthcare technologies have led to an increasing number of technological innovations, but currently most healthcare systems are facing challenges in funding these innovations. While most healthcare systems accept the principle of value-based healthcare, many have had issues transparently and consistently operationalizing a definition of “value” while managing the affordability of new health technologies and incentivising the development of high-priority new pharmaceuticals and devices for the future. This has never been more apparent than right now given the economic uncertainties across the globe. A key consideration in policy deliberations related to this issue is how value should be shared between society and manufacturers to appropriately incentivize healthcare innovation while equitably addressing current healthcare needs. The concept of sharing also needs to reflect the variation in countries’ available resources and its effects on affordability. How should we prioritize areas for sustainable innovation and implement it in policy? Is there a case for greater public-sector involvement in the development of health technologies to better align the availability of new therapies with population health needs and public priorities? How can the huge variation in resource availability between countries be addressed in such policies? These are some of the questions that a panel of health economists, policy makers, and industry experts will address to consider the concept of sharing the value of innovation.
Moderators
Paola Testori Coggi
European Commission, Italian Medicines Agency, Milan, Italy
Paola Testori Coggi has been President of the Committee for price and reimbursement of the Italian Medicines Agency from 2015 to 2018.
Previously she has done her career within the European Commission where she entered in 1983 in the environmental sector and held various posts in the area of health, food safety, consumer protection and research.
She has been Director General for health and consumer until 2014; among her achievements, the legislation on cross-border health care and on pharmaceuticals and medical devices, and the promotion of health as an investment for growth, the White Paper on food safety, the creation of the European Food Safety Authority.
Paola Testori Coggi is a biologist from the University of Milan, Italy, with a Master degree in Ecotoxicology.
Speakers
Natasha Azzopardi-Muscat, MD, PhD
World Health Organization (WHO) Europe, Copenhagen, Denmark
Dr Natasha Azzopardi-Muscat is the Director of the Division of Country Health Polices and Systems at WHO Regional Office for Europe. She is the Past President of the European Public Health Association. Dr Azzopardi-Muscat qualified as a medical doctor and proceeded to specialize in public health and health service management. Her transdisciplinary research bridging health policy, European studies and small states studies led to her PhD entitled “The Europeanisation of health systems: a small state perspective”. She has authored several publications in public health and European health policy.
Nathalie Moll
EFPIA, Brussels, Belgium
Nathalie Moll joined the European Federation of Pharmaceutical Industries and Associations (EFPIA) as Director General in April 2017.
EFPIA represents the pharmaceutical industry operating in Europe. Together with its direct membership 40 leading pharmaceutical companies, 33 national associations representing over 1,900 companies and in collaboration with health and research players, EFPIA's mission is to create an environment that enables our members to innovate, discover, develop and deliver new therapies and vaccines for people across Europe, as well as contribute to the European economy.
Prior to joining EFPIA, between 2010 and 2017 Nathalie was the Secretary General of EuropaBio ranked as the most effective European Trade Association in Brussels in 2013. She spent over 20 years working for the biotech industry at EU and national level in associations and corporate positions and held the position of Chair and Vice Chair of the International Council of Biotech Associations (ICBA).
In 2013, Nathalie won the Technovisionaries Women Innovation Award organised by Women & Technologies® while in 2009, Nathalie and the Green Biotech Team of EuropaBio were presented with the Leadership and Excellence in Advancing Ag-Biotech and Food Issues Award. Nathalie was also named one of the 15 leading women in biotech in Europe in 2017.
Fabio Pammolli
Politecnico di Milano, Milan, Italy
Jack Scannell
JW Scannell Analytics Ltd., University of Edinburgh, Edinburgh, United Kingdom
Jack Scannell studies biomedical R&D from both economic and scientific perspectives. He works as an independent consultant to investors, drug and biotech companies, and the public sector. He is an honorary fellow in the School of Social and Political Sciences at Edinburgh University. Jack worked in drug and biotech investment at UBS and at Sanford Bernstein. He was head of Discovery Research at e-Therapeutics PLC, an Oxford-based biotechnology firm. He started his career as an academic neuroscientist. He has a Ph.D. in physiology from Oxford University and a degree in medical sciences from Cambridge University.
Tue 17 Nov
Patient and Public Involvement in Healthcare Decision Making: Are We Maximizing Opportunities?
An urgent need exists for a more holistic and meaningful approach to citizen involvement in healthcare decision making that includes both those with (ie, patients) and without (ie, the general public) disease-specific interests. Several decision-making bodies state that they involve the general public, but the role of the public’s participation is often not clearly and/or transparently defined, and the impact of this input is often not enumerated in publicly available documents. Patient engagement to inform a specific intervention for a particular disease has been practiced and extensively discussed. However, patient or general public involvement in assessing the implications of funding decisions across technologies, including opportunity costs, is not widely practiced or addressed. There is inadequate insight into how shifts of perspectives associated with the change in health status (eg, healthy to sick) impact value judgements or h ow normative judgements about the health of certain groups (eg, the young, or people with severe or rare diseases) are given additional ‘weight’ in technology adoption decisions. What are society’s underlying—and often not explicit—value assumptions that direct resource allocation? How can we make these assumptions more explicit and transparent? And how generalizable are these assumptions? Should non-health outcomes be considered and, if so, how should they be traded-off against health outcomes?
A panel of experts, including patient organization representatives, academics, and decision makers, will discuss opportunities to think differently and explore options beyond currently practiced patient/public involvement, not just in the one-off technology decision-making process, but at a higher level of resource allocation in healthcare.
Moderators
Bettina Ryll, MD, PhD
Melanoma Patient Network Europe, and Past Chair of the Patient Advocates Working Group, European Society for Medical Oncology, Uppsala, Sweden
Dr. Ryll holds a medical degree from the Free University of Berlin, Germany, and a PhD in Biomedical Sciences from University College London, UK. After losing her husband to Melanoma, she founded the Melanoma Patient Network Europe and developed a special interest in patient-centric clinical research, innovative trial designs and novel drug development concepts, such as MAPPS (medicines' adaptive pathways to patients). Lately, she focuses on patient-relevant outcomes and sustainable healthcare models ensuring access to innovation for patients. Dr. Ryll is involved in numerous initiatives promoting evidence-based advocacy. She is fascinated by the enormous potential and capacity of patient networks to both educate and support patients as well as to capture data at the primary data source, the patients themselves, to generate evidence at a granularity level non-accessible to outsiders. Between 2015 and 2018, Dr. Ryll chaired the ESMO-PAWG, the first time this position was held by a non-oncologist and was instrumental behind the development of the advocacy track at annual ESMO conferences.
Speakers
Nathalie Bere
European Medicines Agency, Amsterdam, Netherlands
Nathalie has been working with the European Medicines Agency since 1998 and is currently responsible for its engagement with patients and consumers. Patients bring specific knowledge and expertise from their real-life experience of the condition and its treatment to the scientific discussions, and their contributions have led to better regulatory outcomes.
Over the years, she has been dedicated to testing and implementing opportunities for the inclusion of the patient’s voice in all aspects of the regulatory lifecycle of a medicine. As a steady increase in patient engagement at EMA has been observed, Nathalie will look to further strengthen these activities and ensure they are fit for purpose in an evolving health landscape.
Nathalie holds a Master in Public Health from the University of East London (UEL) and has a Bachelor of Science in Psychology.
Yann LeCam
Eurordis, Rare Diseases Europe, Paris, France
Yann Le Cam is a patient advocate who has dedicated over 30 years of professional and personal commitment to health and medical research NGOs in France, Europe and the United States in the fields of cancer, HIV/AIDS and rare diseases.
He holds an MBA from HEC Paris. He has three daughters, the eldest of whom is living with cystic fibrosis.
Yann Le Cam was one of the founders of EURORDIS-Rare Diseases Europe in 1997 and has been the organisation’s Chief Executive Officer since 2000.
Axel C Mühlbacher, PhD, MBA
Hochschule Neubrandenburg, Neubrandenburg, MV, Germany
Axel Mühlbacher is professor of health economics and health care management at Hochschule Neubrandenburg. As a secondary appointment he is an adjunct professor in the Duke Department of Population Health Sciences, and a CHPIR Senior Research Fellow in the Center for Health Policy and Inequalities Research at the Duke Global Health Institute, Duke University (USA). He serves as a member of the scientific advisory board of the Institute for Quality Assurance and Transparency in Healthcare (IQTiG) and a board member and chair of the International Academy of Health Preference Research (IAHPR). He currently serves as associate editor of Value in Health and on the editorial advisory board of the Journal of Choice Modelling (JOCM), PharmacoEconomics, Int. Journal of Integrated Care (IJIC) and The Patient – Patient Centered Outcomes Research. Axel Mühlbacher is author of several books and more than 100 peer-reviewed articles.
Wed 18 Nov
Much Ado About Little- Dealing with Limited RCT Evidence for Early HTA and Reimbursement Decisions
It has become more common recently for some innovative drugs to be approved with little or no randomized controlled trial (RCT) evidence, such as those for rare diseases with clear unmet treatment needs. However, given the resulting evidence gaps and uncertainty without RCT evidence, this presents challenges for health technology assessment (HTA) and for payers in making initial value assessments and coverage decisions. Can these gaps be alleviated prospectively in the trial program or real-world data collection planning? If not, what other approaches might be used prior to, instead of, to help inform further evidence development, outcomes-based agreements, or similar arrangements? This plenary will explore several methodological approaches that can help inform those initial decisions. The range of potential methods includes sequential trial planning and quasi-experimental approaches, scenario and hierarchical modeling, causal inference, and value of information techniques, among others. Presentations will include actual applications of those methods.
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
Sylwia Bujkiewicz, PhD, MSc
University of Leicester, Leicester, United Kingdom
Sylwia Bujkiewicz is an Associate Professor of Biostatistics and joint director of the MSc course in Medical Statistics, based in the Biostatistics Research Group at the University of Leicester.
Sylwia leads a team of researchers in advanced methods of evidence synthesis. Her recent research, funded by the Medical Research Council and the National Institute for Health Research, is in methods development and application of Bayesian multi-parameter evidence synthesis for combining data from diverse sources, including multivariate meta-analysis (of multiple outcomes including surrogate endpoints), network meta-analysis and meta-analysis of data from mixture of study designs. She has particular interests in the application of these methods to HTA decision-making. She is a member of NICE Decision Support Unit and Associate Editor for the Journal of the Royal Statistical Society A.
Eva Dietrich
University of Bonn Pharmaceutical Institute, Lörrach, BW, Germany
Professor Eva Susanne Dietrich is a pharmacist with more than 20 years professional experience in reimbursement, health politics, and drug evaluation.
She graduated at the University of Heidelberg, obtained her PhD in the field of pharmacoeconomic methodology from the University of Tuebingen and received a Master’s degree on Health Technology Assessment and Management from the University of Barcelona.
Professor Dietrich served as a deputy member of the Federal Joint Committee (G-BA) which specifies for almost 90 percent of the German population the services in medical care that are reimbursed. In parallel, she led the Department of Drugs, Remedies and Aids at the German National Association of Statutory Health Insurance Physicians (KBV) which concludes contracts with health insurance funds and other parties of the health care sector on the part of the 140,000 office-based physicians and psychotherapists in Germany. In the following years, she organized a scientific institute for Techniker Krankenkasse, one of the largest health insurance funds in Germany, and was responsible for the Management Division Health Sciences. Thereafter, she managed a scientific consulting firm in Basel for eight years and founded the Institute of Evidence-based Positioning in the Healthcare Sector in 2018.
An important emphasis of her professional and academic activities is the critical review of the evidence of new drugs, their classification in the existing care context and, more specifically, early benefit assessments and the German AMNOG process.
Since 2000, Eva Susanne has been teaching at the department of clinical pharmacy at the University of Bonn, where she holds a honorary professorship. Apart from this she taught pharmacoeconomics, evidence based medicine, and benefit legislation e.g. at the universities of Hamburg, Berlin and Marburg.
Stephen Palmer, PhD
University of York, York, YOR, Great Britain
Stephen Palmer is a Professor at the Centre for Health Economics (CHE), University of York. CHE was one of the world’s first research institutes dedicated to the study of the economics of health and health care and has established a leading international reputation. Stephen has over 25 years’ experience of health economic evaluation, regulatory and reimbursement processes. He has published over 175 peer-reviewed publications. His principal areas of expertise relate to the methodology and application of decision-analytic modelling and Bayesian approaches to Health Technology Assessment. He has worked closely with policy makers throughout his career and led a programme of work at CHE supporting the National Institute for Health and Clinical Excellence (NICE) between 2005 and 2019. He was also a member of the NICE Technology Appraisal Committee for 10 years and is currently a member of the NICE Decision Support Unit.
Uwe Siebert, MD, MPH, MSc, ScD
UMIT- University for Health Sciences, Medical Informatics and Technology, and Harvard Chan School of Public Health, Hall i. T., 7, Austria
Univ.-Prof. Uwe Siebert, MD, MPH, MSc, ScD is Professor of Public Health, Medical Decision Making and Health Technology Assessment (HTA), Chair of the Department of Public Health, Health Services Research and HTA at UMIT, and Adjunct Professor of Health Policy and Management at the Harvard Chan School of Public Health. His research interests include applying evidence-based quantitative, causal and translational methods from public health and medicine in the framework of medical decision making, patient guidance and HTA. His research focuses on cancer, neurological disorders, cardiovascular disease and others. He is Past-President of the Society for Medical Decision Making (SMDM), member of the Oncology Advisory Council of the Federal Ministry of Health and the COVID-19 Prognosis Consortium of GÖG Austria, and several Boards of Directors. He advices several HTA and government agencies. He has authored more than 400 publications (H index>50) and is Editor of the European Journal of Epidemiology.