Slide presentations will be available (subject to speaker approval) at the ISPOR Presentation Database and on the
Virtual ISPOR Europe 2020 Program page beginning 6 January, 2021.
* Program subject to change
Paola Testori Coggi
European Commission, Italian Medicines Agency, Milan, Italy
Natasha Azzopardi-Muscat, MD, PhD
World Health Organization (WHO) Europe, Copenhagen, Denmark
EFPIA, Brussels, Belgium
Politecnico di Milano, Milan, Italy
JW Scannell Analytics Ltd., University of Edinburgh, Edinburgh, United Kingdom
Ian Hudson, OBE, MBBS, MD, FRCP, FFPM
Bill and Melinda Gates Foundation, London, United Kingdom
Peter Marks, MD, PhD
U.S. Food and Drug Administration (FDA), Silver Spring, MD, USA
Pierre Meulien, PhD
Innovative Medicines Initiative (IMI), Brussels, NJ, Belgium
Agnes Saint-Raymond, MD
European Medicines Agency (EMA), Amsterdam, NJ, Netherlands
ISSUE: Recent developments in regenerative and immuno-oncological (IO) therapies, such as CAR-Ts and gene replacement across diverse indications have raised the tantalizing promise of a “cure”. Plateaus have emerged in the progression-free and overall survival curves following IO treatment in some cancers with clinicians interpreting these as indication of cure in some patients. Cure has appeared in statistical analysis and clinical discussions as well as in economic models, yet assumptions around cure fractions remain among the most controversial topics in the HTA assessments. There seems to be no consensus on the definition of cure, nor on how it should be modelled in economic evaluations. In this context the panel would discuss if there should be a standard definition of cure and how it should be defined or shall we continue with definitions from various stakeholders, 2) what are the ways that potential cure can or should be included in economic evaluations and the 3) impact of patient population and disease means when discussing cure.
OVERVIEW: The moderator, Dr Ishak, will introduce the topic and present current statistical methods to estimate cure; he will also challenge the panellists to what extent a consensus can be reached. Dr Latimer, as member of a NICE Appraisal Committee will address how cure has been and could be included in economic evaluations and what impact it has on HTA decisions; Dr Ryll will cover the patients’ and clinicians’ point of view; Ms Benedict will cover the perspective of pharmaceutical companies based on a set of interviews with representatives of companies developing CAR-T, cell and gene-therapies across a diverse set of diseases, impacting different age groups. The moderator will present for 5 minutes and each presenter will present for 10-12 minutes, with 20-25 minutes for panel discussion with audience participation.
K. Jack Ishak, PhD
Evidera, St-Laurent, QC, Canada
Ágnes Benedict, MSc
Evidera, Budapest, LON, Hungary
Nick Latimer, MSc, PhD
ScHARR, University of Sheffield, Sheffield, DBY, United Kingdom
Bettina Ryll, MD, PhD
Melanoma Patient Network Europe, and Past Chair of the Patient Advocates Working Group, European Society for Medical Oncology, Uppsala, Sweden
ISSUE: COVID-19 has spread across the globe in an unprecedented speed, leading to over 10 million cases in just over 6 months. In absence of a treatment, governments had only costly prevention strategies to control its spread. Repurposing drugs approved for other indications as well as developing new drugs are the two main strategies deployed to find an effective treatment. To use any of these drugs on a large scale, however, governments require clear evidence of efficacy and safety. Large-scale randomised controlled trials, for example RECOVERY and SOLIDARITY trials, have so far provided evidence to support some treatments such as low-dose dexamethasone and remdesivir for those with severe COVID-19. However, it will not be clear whether a clinically-effective drug will be cost-effective when used in different countries and settings affected by the pandemic. Nevertheless, many countries have recommended these treatments based on the clinical efficacy evidence alone, without going through the HTA process.
Amanda Adler, MD, PhD, FRCP
University of Oxford, Fowlmere, United Kingdom
Mark R Chakravarty, BSc (Hons), MBChB, Dip. BA
Novartis Pharmaceuticals, Basel, Switzerland
Kalipso Chalkidou, PhD
Imperial College London, London, United Kingdom
National Institute for Health and Care Excellence (NICE), London, LON, United Kingdom
Kristian Kidholm, PhD
University of Southern Denmark, Odense, Denmark
Hans Ossebaard, PhD
National Health Care Institute (Zorginstituut Nederland), Diemen, NH, Netherlands
Maja Rasmussen, MS
Jason Shafrin, PhD
PRECISIONheor, Los Angeles, CA, USA
ISSUE: . COVID-19 emphasized the issue of evidence generation for access to market of medical devices (MDs). In times of urgency and emergency, governments struggled to keep the balance between patients’ and professionals’ needs to access the technologies as quickly as possible and the necessity to assess safety and effectiveness. The final effect was that in many cases governments arrived too late and hospitals had to do it themselves with huge discrepancies across jurisdictions. One interesting stream of research is the one that considers clinical evidence generation across the entire lifecycle of MDs. This is what the new EU Regulations on MDs and vitro diagnostics embrace. Furthermore, if the Commission Proposal for an EU HTA Regulation were adopted, it would provide the possibility for Member States to further strengthen the current HTA systems at national, regional and hospital level and the whole lifecycle of high-risk implantable MDs. The Proposal would enable a comprehensive vision of clinical evidence generation for some high risk MD. This may have an impact in the MedTech ecosystem. Are we ready? There is an urgent need to explore this issue with all stakeholders.
OVERVIEW: . Flora Giorgio (10’) will discuss the opportunities the proposed EU HTA Regulation could bring to the systems, including its synergies with the Regulatory framework, from the prospective of the European Commission. Laura Sampietro-Colom (10’) will discuss the challenges of clinical evidence generation across the lifecycle of technologies from the perspective of HTA doers and users. Andrea Rappagliosi (10’) will represent the MedTech industry and discuss whether manufacturers are ready to invest in a long-term, strategic vision that would govern the development of clinical studies across the entire lifecycle. 10’ will be allocated to the introduction of the panel issue and 20’ will be allotted to discussion. The entire MedTech ecosystem would benefit from attending.
Rosanna Tarricone, MSc, PhD
SDA Bocconi School of Management, Milan, Italy
Flora Giorgio, PhD
European Commission, Brussels, Belgium
Andrea Rappagliosi, MSc
Edwards Life Science, Nyon, VD, Switzerland
Laura Sampietro-Colom, MD, PhD
Hospital Clinic Barcelona, Barcelona, B, Spain
Josie Godfrey, BA MA MA
JG Zebra Consulting, London, United Kingdom
Ron Akehurst, DSc, Hon MFPHM
BresMed Health Solutions, Sheffield, DBY, United Kingdom
Fleur Chandler, PhD
Sanofi, Reading, RDG, United Kingdom
Sachin Kamal-Bahl, PhD
SKB Consulting Inc., Lansdale, PA, USA
To participate in this group discussion session, please watch the on-demand session titled COVID-19 and Hospital Procurement: The Role of Value Assessment for Medical Devices & Diagnostics in Pandemic Public Health Situations, which is available to conference registrants here. The on-demand session is presented by the ISPOR Medical Devices & Diagnostics Special Interest Group and the leaders of this group will facilitate this networking session.
Preparation for the pandemic, or even subsequent waves of the existing pandemic, raises several related and pressing questions about the role of breadth, scope, and flexibility of value assessment for Medical Devices & Diagnostics. This discussion can help inform the procurement and distribution of critical medical technologies at the local and international level. Related issues and solutions need to be discussed and assessed using an integrated approach of various key stakeholders. Hence the panelists reflect an international WHO perspective, an HTA agency perspective, and academic / health economic perspective, an industry / manufacturer perspective and a health care provider perspective in order to discuss related key issues including: • How healthcare systems can coordinate, globally, to help ensure that healthcare efficiency has the potential to be maintained even in challenging, emergency situations? Who should have an active role on value assessment of new technologies in emergency situations? • How can non-governmental organizations, national governments, and international pan-national bodies help to provide a framework for more efficient decisions and allocations of resources in a pandemic emergency? How should these organizations receive feedback from local procurement? • How can public and private collaborate and co-design solutions to ensure affordability, sustainability and overall resilience? • How to define strategic procurement strategies, based on the creation of a portfolio of reliable suppliers, in order to avoid the lack of critical goods in case of emergency? • What should be the ideal role of the academics and specifically health economists be in similar future contexts? Which role plays value-based healthcare in this context? • How can the industry and policymakers contribute to address these critical questions? Value-based procurement needs an appropriate place in decision making to ensure organizational and health care systems are prepared for subsequent pandemics in a timely manner.
Richard Charter, MSc
Alira Health, Basel, Switzerland
Abdalla Abotaleb, PhD ,PharmD
World Health Organization, cairo, C, Egypt
Federico Augustovski, MSc, PhD
Institute for Clinical Effectiveness and Health Policy, Buenos Aires, Argentina
Giuditta Callea, PhD
SDA Bocconi School of Management, Milan, MI, Italy
Santiago Hasdeu, MD
National University of Comahue, Neuquén , Q, Argentina
Luqman Tariq, PhD
Stryker, Schiedam, ZH, Netherlands
This symposium provides a forum to discuss what this delay may mean from a humanistic perspective to patients and their caregivers, and how the value of future potential treatments for early AD should consider these critical non-economic elements. Specific considerations for what patients and caregivers or families of those in in early stages of disease value, as it often goes beyond the avoidance of medical resource utilization which is a common payer priority. The critical role and value of early diagnosis will also be discussed.
Samantha Benham-Hermetz, ,
Alzheimer's Research UK, Cambridge, CAM, United Kingdom
Frank Jessen, MD, PhD
University Hospital Cologne AöR, Bonn, Germany
Marc Wortmann, MD, PhD
University Hospital Cologne AöR, Zeist, UT, Netherlands
To participate in this group discussion session, please watch the on-demand session titled Review And Identification Of Criteria For Value Assessment Of Medication Adherence-Enhancing Interventions, which is available to conference registrants here. The on-demand session is presented by the ISPOR Medication Adherence and Persistence Special Interest Group and the leaders of this group will facilitate this networking session.
Medication non-adherence is prevalent across most clinical conditions causing major medical and economic challenges. Although several studies have demonstrated that medication adherence enhancing interventions (MAEIs) may improve health outcomes, existing evidence on the effectiveness of MAEIs remains of poor quality. Important policy decisions about MAEIs are therefore currently based on inadequate or suboptimal information. The Medication Adherence and Persistence Special Interest Group (MAP-SIG) is conducting a systematic evaluation of the evidence associated with MAEIs by identifying and prioritizing relevant criteria for their value assessment from the perspectives of different stakeholders. Attendees interested in medication adherence and developing consensus-based criteria to assess value of MAEIs are welcomed to join this forum. A brief presentation on the purpose of the initiative and findings of the systematic literature review on criteria for assessing MAEIs will be presented. This will be followed by an open discussion on the criteria. The attendees will be asked to: 1) share their opinions on identified criteria, 2) suggest improvements to criteria presented, and 3) recommend additional criteria. After a final list of criteria is obtained, the audience members will rank the presented criteria in order of importance, via the polling app. Lastly, the group will be asked to share their perspectives and discuss ideas on how these criteria can be used to improve medication adherence studies. Grab your lunch and join us for this informative discussion.
Mickaël Hiligsmann, PhD
Maastricht University, Maastricht, LI, Netherlands
Tamas Agh, MD, PhD, MSc
Syreon Research Institute, Budapest, Hungary
Andrew Peterson, PharmD, PhD, FCPP
University of the Sciences, Philadelphia, PA, USA
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.
European Medicines Agency, Amsterdam, Netherlands
Eurordis, Rare Diseases Europe, Paris, ON, France
Axel Mühlbacher, PhD, MBA
Hochschule Neubrandenburg, Neubrandenburg, MV, Germany
The COVID-19 pandemic has upended the careers and personal lives of millions across the globe. Many HEOR professionals have successfully adjusted and worked through the initial phase of this transition, including establishing a “work from home” routine, managing video meetings, and contributing as a remote team member. But, as the pandemic and its impact stretches on, how does one learn to thrive and advance their careers in this “new normal?” Additionally, early research indicates that the pandemic may be disproportionately impacting working women’s careers compared to their male counterparts.
This panel of highly successful women in HEOR from a diverse array of geographies, work sectors, and career stages will share how they have adapted to the virtual work environment. They will advise on how HEOR professionals can transition to the next phase of this “new normal” with an eye towards long-term career success. Panelists will also share strategies for balancing work and personal wellness in this evolving COVID-19 working world.
This forum will feature considerable discussion and interaction among the panelists and will also include audience Q&A. The session is open to all Virtual ISPOR Europe 2020 registrants—women and men in HEOR.
Julia F. Slejko, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
Olivia Wu, PhD
University of Glasgow, Glasgow, United Kingdom
Blythe Adamson, PhD, MPH
Flatiron Health, New York, NY, USA
Nancy Devlin, PhD
University of Melbourne, Melbourne, VIC, Australia
Ebere Onukwugha, PhD, MS
Louise Timlin, MSc
Eli Lilly and Company, Windlesham, Surrey, United Kingdom
Stefano Centanni, MD, PhD
Università degli Studi di Milano, Milan, MI, Italy
Stefano Del Giacco, MD
Università di Cagliari, Cagliari, MI, Italy
Giorgio Walter Canonica, MD, PhD
Humanitas University, IRCCS Humanitas Research Hospital, Milan, Italy
Eva Dietrich, ,
University of Bonn Pharmaceutical Institute, Lörrach, BW, Germany
Enrico Giorgio Heffler, MD, PhD
Humanitas University, IRCCS Humanitas Research Hospital, Milan, MI, Italy
Claudio Jommi, PhD
Jörg Kleine-Tebbe, MD
Allergy and Asthma Center Westend, Berlin, Germany
Giuseppe Turchetti, PhD
Scuola Superiore Sant'Anna, Pisa, PI, Italy
Novartis Gene Therapies Inc.
Omar Dabbous, MD, MPH
Novartis Gene Therapies, Bannockburn, IL, USA
Michael Drummond, MCom, DPhil
University of York, York, YOR, United Kingdom
Mondher Toumi, MD, MSc, PhD
Aix-Marseille University, Paris, France
Johann-Matthias von der Schulenburg, PhD
Leibniz University Hannover, Hannover, Germany
PURPOSE: Economic evaluation is an important tool to inform health care resource allocation worldwide, and HTA agencies typically develop methods guidance which, in part, sets out expectations on how the economic evidence is developed and appraised. Determining such guidance is, however, never without controversy. This workshop aims to reflect on the experiences of HTA agencies in different jurisdictions in Europe that have recently, or are currently, updating their methods guidance -- EUnetHTA, INFARMED for Portugal and NICE for England -- to identify the most pressing methods and normative issues associated with the updated guidance and main areas of controversy.
DESCRIPTION: The workshop will start with an introduction to the topic (Mark Sculpher, 7 minutes). The panellists will then identify challenging aspects of three recent methods guidance updates, describing changes implemented. The three guideline updates are:
Mark Sculpher, PhD
Marta Soares, MSc, PhD
University of York, York, YOR, Great Britain
Conor Teljeur, PhD
Health Information and Quality Authority, Dublin, D, Ireland
Ian Watson, MSci PGDip
National Institute for Health and Care Excellence, London, United Kingdom
Hareth Al-Janabi, PhD
University of Birmingham, Birmingham, United Kingdom
Andrew Lloyd, DPhil
Acaster Lloyd Consulting Ltd, London, United Kingdom
Noemi Muszbek, MSc
Visible Analytics, Reading, RDG, United Kingdom
Edit Remak, PhD
Visible Analytics, Oxford, OXF, United Kingdom
Anja Schiel, PhD
Norwegian Medicines Agency, Oslo, Norway
Nancy Dreyer, MPH, PhD
IQVIA, Cambridge, MA, USA
Robert Golub, MD
JAMA, Chicago, IL, USA
Xavier Kurz, MD PhD
ISSUE: Pricing and reimbursement policies play an important role in determining how the value generated by new branded pharmaceuticals is shared between manufacturers and patients served by health systems. They also send a signal to pharmaceutical companies, biotech companies and others involved in R&D investment decisions about the value of future investments and are, therefore, expected to influence the number and nature of new products brought to market. However, limited attention has been paid to how value is currently shared and how it should be shared taking in to account implications for health system sustainability and pharmaceutical innovation.
OVERVIEW: Danny Palnoch will provide a 10-minute introduction and overview of key issues for discussion. Beth Woods will present a framework for estimating how value is shared and show that the share of value accruing to manufacturers is higher than previously estimated. Jens Grueger will discuss how the costs of innovation are shared across countries and whether all countries can be considered to be contributing appropriately to the development of new pharmaceuticals. Finally, Patricia Danzon will consider how value should be shared when a proprietary technology is developed using publicly funded R&D. Patricia will use COVID-19 vaccines as a topical case study that also raises issues of affordability due to high volumes and the significant economic impact of COVID-19. Each panellist will speak for 10 minutes and Danny Palnoch will then chair a 20-minute discussion and debate. This session will be of broad interest to the pharmaceutical industry, health care decision makers, and researchers.
Danny Palnoch, BSc
NHS England, LON, United Kingdom
Patricia Danzon, PhD
University of Pennsylvania, Philadelphia, PA, USA
Jens Grueger, PhD
University of Washington, Boston Consulting Group, Freiburg, BW, Germany
Beth Woods, MSc
University of York, Heslington, York, YOR, United Kingdom
ISSUE: Rising spending on pharmaceuticals has caused a shift toward value-driven healthcare. Multiple initiatives have addressed the measurement of value of health technologies; notably ISPORs Initiative on US Value Assessment Frameworks. Among the challenges is how to integrate patient preferences into HTA. Agencies express interest in receiving patient preference data. But it often remains unclear precisely how such data should be combined with other evidence when assessing value. Should patient preference data be incorporated into economic evaluations? If so, how can this be achieved in a manner consistent with existing valuation frameworks? If not, how should patient preferences be considered alongside economic evaluation and how can it impact assessments?
OVERVIEW: Mendwas Dzingina will outline the challenges associated with measuring the value of technologies from a sponsors’ perspective. Using the ISPOR value flower, he will illustrate how patient preferences could enhance HTA.
Esther de Bekker-Grob, PhD
Erasmus University Rotterdam, Rotterdam, Netherlands
Jacoline Bouvy, PhD
Mendwas Dzingina, PhD
Pfizer, Tadworth, United Kingdom
Kevin Marsh, PhD
Evidera, Newport Pagell, BKM, Great Britain
Kristian Thorlund, MSc, PhD
Cytel, Hamilton, ON, Canada
Danielle Bargo, MSc
Edward J Mills, PhD, FRCP (Edin)
McMaster University, Hamilton, ON, Canada
Mark Shapiro, PhD
xCures, DURHAM, NC, USA
To participate in this group discussion session, please watch the on-demand session titled Effect Of Public And Private Partnership In Healthcare Financing To Improve Health Outcomes, which is available to conference registrants here. The on-demand session is presented by the ISPOR Arabic Network and the leaders of this group will facilitate this networking session.
The cost to deliver healthcare in developing countries has been rising exponentially. Governments around the world are searching for alternative mechanisms to reduce costs while increasing the medical capacity of the system with significant investments in infrastructure. Governments especially in Europe, have increasingly used private sector involvement in developing, financing and providing public health infrastructure and service delivery through public–private partnerships (PPPs). Use of PPPs in MENA is a relatively new phenomenon. Policy makers and governments are very much interested in studying and understanding the relationship between health care expenditure and health outcomes. Public healthcare systems may not be technically and allocative efficient due to several challenges, a push for private funding is usually needed. The goal of this forum is to explore motivations, challenges, and lessons learned during PPP partnerships: engagement, formation, operations, financing and knowledge management. The forum objectives is to engage in forward-looking dialogue on the evolving models of partnerships for improving health outcomes in MENA region; to explore opportunities for identifying and applying lessons learned from experiences of success and failure; and to discuss how the evolving model of partnerships and lessons learned can impact the future of the healthcare system in MENA, help meet developmental challenges, and sustain improved outcomes.
Rita Karam, PharmD, PhD
Lebanese University, BEIRUT, Lebanon
Sherif Abaza, BPharm, MBA
Syreon Middle East, Cairo, C, Egypt
Sarine Aderian, PHARM D
Levant and Egypt, Abbvie biopharmaceuticals. Member of ISPOR Lebanon chapter, Beirut, Lebanon
Kasem Akhras, PharmD
Astrazeneca, Dubai, United Arab Emirates
Mahmoud Elmahdawy, PharmD
Novartis, Singapore, 01, Singapore
Yacine Sellam, Pharm.D, Ph.D
Algerian Society for Regulatory Affairs & Pharmacoeconomics (SAARPE), ALGER, 16, Algeria
Sylwia Bujkiewicz, PhD, MSc
University of Leicester, Leicester, United Kingdom
Stephen Palmer, PhD
Uwe Siebert, MD, MPH, MSc, ScD
UMIT- University for Health Sciences, Medical Informatics and Technology, and Harvard Chan School of Public Health, Tirol, Austria
To participate in this group discussion session, please watch the on-demand session titled Expanding COA In 2020: Guidance In RWE And Improving Communication Between Different Stakeholders, which is available to conference registrants here. The on-demand session is presented by the Clinical Outcome Assessment Special Interest Group and the leaders of this group will facilitate this networking session.
The Clinical Outcomes Assessment (COA) Special Interest Group’s focused this past year on harmonizing COA nomenclature and value as well as align guidance for COA generation in real world (RW) studies which are often presented to regulatory and HTA agencies. Bryan Bennett and Lynda Doward will discuss the results of a membership survey which identifies how different stakeholders value PRO data in their professional decision-making based on their current and projected use of COA data . Additionally as part of the key project, they will present the preliminary findings from a review of HTA terminology used for COA from different countries that will help to inform our understanding with an aim to a degree of harmonize the way Health Economic and Outcome Research scientists and COA specialists describe COA value. Angela Rylands and Ana Maria Rodriguez would like to involve COA experts attending ISPOR in aligning next steps in their proposal of new guidelines and best practices for COA in real world evidence (RWE) studies. They will share a draft manuscript framework, which aims to summarize the output from the virtual expert meeting and international survey with industry, regulatory and HTA/payer agencies hosted at the end of 2019 which collected views on the use of COA in RWE.
Katja Rudell, PhD, MSc
Parexel International, Chittering, United Kingdom
Bryan Bennett, PhD
Bristol Myers Squibb (BMS), Uxbridge, Middlesex, BKM, United Kingdom
Lynda Doward, Master of Research
RTI Health Solutions, Manchester, United Kingdom
Ana Maria Rodriguez, PhD, MSC, PT
IQVIA, Madrid, Spain
Angela Rylands, PhD, CPsychol
Kyowa Kirin International plc, UK, Marlow, BKM, Great Britain
The scale up of pharmaceutical innovation is facing an even more challenging environment than imagined. Within this context some initiatives have arisen across EU with the willingness to combine an always complex equation: early access to innovation for the patients that may benefit the most at a fair price, health care expenditure cost control, and incentives to pharma and biotech innovation for health solutions.
While these therapies have been able to gain accelerated access via adaptive regulatory pathways in various countries, existing HTA processes prove more challenging. Positive reimbursement recommendations are scarce and usually limited to specific indications. Potential ways to overcome the HTA hurdles include better alignment of evidence sources and endpoint requirements, acceptability of post-authorisation data collection models and innovative payment models. Yet, while the challenges for histology independent treatment are quite similar in various countries, solutions will likely have to be diverse and include methodological advancements as well as policy-makers’ willingness to adopt new approaches.
This Educational Session will 1) provide insight in the current regulatory and HTA landscape for histology independent therapies in various countries in North-America, Europe and Asia; and 2) discuss how to move forward from here. Global experts on histology independent therapies will provide their insights and discuss how to make a positive change from a patient representative perspective, a medical perspective and an HTA and policy perspective.
With an increasing amount of histology independent therapies coming towards market, there is a need to find solutions today. This session will sharpen your thinking and inspire to move beyond the unsustainable status quo.
Office of Health Economics
Lotte Steuten, PhD, MSc
Office of Health Economics, London, LON, United Kingdom
Rosa Giuliani, MD
The Clatterbridge Cancer Center, Liverpool, ON, United Kingdom
Entela Xoxi, PhD PharmD MSc
Catholic University of Rome, Roma, RM, Italy
PURPOSE: This workshop will give attendees insights into designing preference studies and communicating results to inform public-health decision making.
DESCRIPTION: Background: Recent controversies regarding the timing and extent of pandemic-related public-health measures have highlighted common challenges facing officials across a wide range of policy contexts. Even with accurate information on policy consequences, elected officials and public-health experts often lack crucial data on societal values necessary to inform the difficult decisions they are facing. Decision makers need objective information about the public’s willingness to accept tradeoffs among health, economic impacts, and competing priorities for limited budgets. In the absence of such information, decision makers implicitly apply their own personal values which can be influenced by unrepresentative stakeholder perspectives, stories in the press or on social media, or public-opinion surveys. Attendees will learn how to identify policy-relevant questions and how to design, execute, and disseminate results to inform health policy decisions. Discussion leaders will share lessons learned on the impact of several studies, what researchers can do to improve uptake, and what design tradeoffs could be necessary to produce results quickly enough for time-sensitive decision making. Successful policy-relevant stated-preference research includes identifying preference-sensitive policy problems, quantifying heterogeneity in policy-relevant populations, including demographics, geography, and political views, and communicating results effectively to decision makers. Discussion leaders: Reed Johnson will share lessons learned from a stated-preference study designed to prioritize expenditures on health and non-health programs. Shelby Reed will discuss challenges in producing policy-relevant benefit-risk preference data under time and resource limitations at the onset of the pandemic in the U.S. Axel Mühlbacher will review how stated-preference data has informed regulatory decision making in Germany. Marcel Jonker will describe a highly publicized study in the Netherlands of public preferences for a COVID-19 contact-tracing app.
Reed Johnson, BS
Duke Clinical Research Institute, Durham, NC, USA
Marcel Jonker, PhD
Erasmus University Rotterdam, Rotterdam, ZH, Netherlands
Shelby Reed, PhD, RPh
PURPOSE: To introduce practitioners to a new, generalized cost-effectiveness analysis (CEA) that incorporates risk-aversion and diminishing returns to preferences for health and their considerable implications for practice.
DESCRIPTION: Standard CEA methods assume constant returns to health in determining the optimal CEA threshold and measuring QALY gains, even though the bulk of theory and evidence implies that returns to health diminish. We introduce diminishing returns to health-related Quality of Life (QoL). This leads to three important modifications in the conduct of CEA: (1) Overall willingness to pay (WTP) thresholds decline compared to conventional approaches. (2) WTP measures increase non-linearly as severity of illness increases. Our preliminary estimates suggest that the combined effects of these two changes will reduce optimal reimbursement levels per QALY for mild illness (perhaps by a factor of 2 or more) while increasing optimal reimbursement per QALY for very severe illnesses (perhaps by a factor of 5 or more). (3) In contrast to current CEA practices, which discriminate against disabled people (and the use of which is banned in the US Medicare program), the new model shows that improving the quality of life (QoL) for disabled people has greater (not less) value than for comparable non-disabled people.
Darius Lakdawalla, PhD
University of Southern California, Los Angeles, CA, USA
Charles Phelps, PhD
University of Rochester, Pittsford, NY, USA
ISSUE: The current pandemic has brought into sharp relief a need to further understand and quantify the broader societal benefits of vaccines, including benefits to patients, caregivers, health systems, and nonhealth impacts on education, economic development, and other macroeconomic effects. Further, countries who may benefit the most from these medical advances may also be those least able to afford them. How can innovators be rewarded for developing life-saving technologies and be incentivized for future development in an affordable and sustainable fashion?
OVERVIEW: This panel will discuss the need to fully quantify the health, nonhealth, and broader societal benefits of innovative vaccines. Dan Ollendorf will moderate the session and will briefly (5 minutes) present findings of recent research on the degree to which social impacts of vaccines have been quantified as well as whether and how inclusion of these elements changes cost-effectiveness calculations. Each panelist will then be given 12 minutes to speak. Carlo Giaquinto will discuss the benefits of life-saving therapies to children and other vulnerable populations, with a focus on how these benefits extend beyond the patients themselves. Raymond Hutubessy will discuss the state of research in broad economic impacts of vaccination and the recently updated WHO guidelines for economic evaluation of vaccines. Craig Roberts will provide a private sector perspective, which seeks to recognize value derived from the broad benefits that accrue from vaccination, while recognizing the global nature of infectious disease and aspiring to support affordability for countries with different abilities to pay.
Daniel Ollendorf, PhD
Tufts Medical Center, Boston, MA, USA
Carlo Giaquinto, MD
University of Padova, Padova, PA, Italy
Raymond Hutubessy, PhD
World Health Organization, Geneva, Switzerland
Craig Roberts, PharmD, MPA, MBA
Merck & Co., Inc., North Wales, PA, USA
ISSUE: Covid-19 is indirectly affecting patient access to innovative treatments in countries, potentially exacerbated by the effects of IRP. Evidence suggests that IRP may indirectly or directly limit patient access to innovative medicines. Covid-19 constrains resources and budgets for other healthcare needs and for innovations to address them. Potential IRP implementation in the US adds to this global dynamic. How would this new constellation affect payers, policy makers, industry, and patients in large EU and lower-income countries? Who would benefit? Eventually, could the negative effects of IRP “go viral,” limiting patient access in more and more countries?
OVERVIEW: This panel will debate how IRP and Covid-19 together may compound the effects of overt and covert patient access limitations in different types of European markets and what the additional impact of a potential US implementation of IRP could be. What have been the benefits and unintended consequences of IRP pre-Covid-19 and how is the dynamic changing for payers, policymakers, industry, and patients—with the panelists presenting different country perspectives, reflecting their academic and industry experiences as well. Andras Incze will moderate the panel and begin with an overview of the evolving IRP dynamic affecting patient access in Europe. Then, he will pose key questions for the panelists to debate: Which countries in Europe and which stakeholders benefit from IRP and what is the emerging effect of Covid-19 on access through interaction with IRP? Who would be affected negatively and over what time horizon? How is industry in Europe affected by the current trend? What could be the add-on effect of the US eventually implementing IRP?
Andras Incze, PhD, MBA
Baden-Wuerttemberg Cooperative State University Germany & Akceso Advisors AG, Basel, BS, Switzerland
Jaime Espin, PhD
Andalusian School of Public Health, Granada, GR, Spain
Lou Garrison, PhD
University of Washington, Seattle, WA, USA
Zoltan Kalo, PhD
1) Semmelweis University; 2) Syreon Research Institute, Budapest, ON, Hungary
Gavin Outteridge, MA
AESARA Europe, London, United Kingdom
Finn Børlum Kristensen, MD, PhD
University of Southern Denmark, Hilleroed, 84, Denmark
Vanessa Schaub, PhD
F.Hoffmann-La Roche, Switzerland
Michael Schlander, MD, PhD, MBA
German Cancer Research Center (DKFZ), Heidelberg, BW, Germany
Søren Holm, Professor of Bioethics
University of Manchester, Cheadle Hulme, United Kingdom
Maarten J. Postma, PhD
University of Groningen, University Medical Center Groningen, Groningen, Netherlands
Jeff Richardson, Professor emeritus
Monash University, Clayton, Australia
ISSUE: The current COVID-19 pandemic has brought about unprecedented cooperation between biopharma, governments and cross country collaboration. Plans have been set to share research, research expenses, manufacturing risks and fast track regulatory approval. However, we don’t know how this will affect health technology assessment (HTA) and reimbursement for these vaccines. Considering there is the possibility that the vaccine will be multi-sourced with different manufacturing processes and mechanisms of action. How will a value based price be determined in this scenario. In addition, add on that the COVID-19 vaccines will be available simultaneously across the globe, will the process and methodology used to determine value-based price and ultimate reimbursement recommendation follow the standard approaches?
OVERVIEW: This interactive session will invoke audience discussion around how multi-sourced, multi country roll out of the novel COVID-19 vaccine will affect HTA methodologies and this will invoke discussions on areas of consensus and disagreement. The program will include viewpoints from experts in HTA within Europe and the pharmaceutical industry. The session will encourage audience participation to generate a robust discussion regarding their perceptions on HTA assessment in the midst of a global pandemic and permit discussion of points not raised by the panel using periodic interactive polling questions. Overall, better understanding of how value-based price and reimbursement decisions are affected by the current COVID-19 pandemic, will allow for better understanding an insights into future multi sourced products, in particular with respect to a global development plan and roll-out. The goal is to leave the audience with a more informed opinion and a better appreciation of the complexities associated with HTA assessment conducted during a global pandemic.
Richard Stanford, PharmD, MS
AESARA, Chapel HIll, NC, USA
Andrew Briggs, DPhil
London School of Hygiene & Tropical Medicine, London, LON, United Kingdom
Anna Kaltenboeck, MA
Memorial Sloan Kettering Cancer Center, New York, NY, USA
Raquel Aguiar-Ibáñez, MSc
Merck Sharp & Dohme Ltd, Haarlem, NH, Netherlands
Gianluca Baio, PhD
University College London, London, SRY, United Kingdom
Dawn Lee, MMath, MSc
BresMed Health Solutions Ltd., Sheffield, DBY, United Kingdom
PURPOSE: To introduce the Digital Endpoints Ecosystem and Protocols (DEEP) initiative, outline its purpose, and gain feedback from the ISPOR audience by pressure-testing key DEEP service specifications from different stakeholder perspectives.
DESCRIPTION: This workshop assumes some knowledge of digital endpoints and contexts of use. It is aimed at people with experience of, or interest in, the development of innovative endpoints for clinical trials. The workshop will present DEEP, a collaborative initiative to define protocols and processes for the development and implementation of digital endpoints in clinical and real-world studies and guide the setup of standardized services that will accelerate adoption and expand usage.
Kai Langel, BSc
Janssen, Titusvile, NJ, USA
Mark Larkin, PhD
Vitaccess Ltd, Oxford, LON, United Kingdom
Sheela Upadhyaya, Dip
Independent HTA perspective, London, United Kingdom
Marc Walton, MD,PhD
Janssen, Ashton, MD, USA
PURPOSE: To provide participants with the knowledge to implement the SIR model in evaluating the healthcare system’s demand and, thus, better allocate the medical resources.
DESCRIPTION: This workshop introduces participants to the basic principles and use of the SIR and SEIR model, especially in evaluating the demand of the healthcare system to allocate medical resources. A novel coronavirus pneumonia, initially identified in Wuhan, China, has surged in the world. In anticipation of substantial burdens on the healthcare system following this human-to-human spread, advance and precise prediction of the demand can provide the time window to mobilize the resources to prevent a possible chasm in the healthcare system. The SIR model was found to serve as a feasible tool for better scrutinizing the currently available information and evaluating the burden of healthcare systems during the pandemic, and thus it provides practical suggestions on reducing the spread on a large scale and helps authorities formulate effective control measures to combat this emerging viral outbreak. The workshop proceeds in three sessions in which we introduce the SIR model, incorporate the SIR model in evaluating the demand of the healthcare system, and critique in small groups. Based on problem-based and case-based learning, the workshop will use real-time demonstration with an audience discussion of the implementation of the SIR model to predict the demand for medical resources due to the outbreak of COVID-19 using real data and cases in January 2020.
Zonglin He, MBBS
Jinan University, Guangzhou, 44, China
Jian Huang, PhD, MPH
Imperial College London, London, LON, United Kingdom
Wai-kit Ming, MD, PhD, MPH, MMSc, EMBA
Jinan University, Guangzhou, China
Casper Zhang, PhD,MPH
The University of Hong Kong, Hong Kong, 91, China
PURPOSE: To introduce the concept of surrogacy and outline the levels of evidence and analytical techniques used to evaluate the surrogate relationship. To describe the current status of using surrogate endpoints in regulatory and health technology assessment (HTA) submissions and outline the international HTA agencies’ methodological guidance this area, providing an update on the changes currently being made to NICE methods guide relating to their use in submissions.
DESCRIPTION: Using surrogate endpoints has become common in trials; especially those informing regulatory decisions. This means that many drugs, particularly those for cancer and rare diseases, are increasingly being approved based on an evidence-base that lacks direct evidence on their actual impact on final outcomes that are important to patients, clinicians and HTA agencies – including overall survival and quality of life.
Oriana Ciani, PhD
Huseyin Naci, PhD
London School of Economics and Political Science, London, United Kingdom
PURPOSE: Contact tracing has been used as an early COVID-19 containment measure in combination with physical distancing measures. Although the precise impact of contact tracing on the epidemic is unknown in most countries, there is a need to understand what combination of measures - including novel digital tracing approaches and less intensive physical distancing - are effective and provide least burden to the society. Comparative effectiveness of strategies can be evaluated using infectious disease modeling (IDM) as recommended by the ISPOR-SMDM Good Modeling Task Force. However, outcomes may vary depending on model characteristics and regional context. We aimed to provide an overview of estimated reduction in transmission under different control measures in Austria, Poland and Germany. In this workshop, participants will get insights on two different agent-based COVID-19 models (ABM) applied in the three countries to inform decision makers. Participants will understand how these models were developed, how they helped understanding disease dynamics, providing outcome measures including distributions of effects in the population, communication and guiding policy decisions.
DESCRIPTION: Ass.-Prof. Jahn will start with a brief introduction on IDM approaches, perceived (dis)advantages and the application of ABM. Next, two independently developed ABMs and their role in COVID-19 policy decision making in Austria, Poland and Germany will be discussed. Niki Popper will present the Austrian model, contact tracing outcomes and a critical reflection on the unique modular structure and challenges in model calibration . Subsequently, Prof. Krüger will present the model developed for Poland/Germany and evaluations on contact tracing based on 13.000 linked Polish patient data, focusing on communication of results. Finally, Dr Bock will provide insights in contract-tracing evaluations for Germany with the specific focus on acquisitions and quality of input date. The audience will be actively engaged in a 15 min Q&A session and life polling/chat to foster discussions.
Martin Bicher, PhD
TU Wien, Vienna, Austria
Wolfgang Bock, PhD
Technical University Kaiserslautern, Kaiserslautern, Germany
Beate Jahn, Dipl.Math. oec. Dr.rer.soc.oec
UMIT - University for Health Sciences, Medical Informatics and Technology, Institute of Public Health, Medical Decision Making and Health Technology Assessment, Department of Public Health, Health Services Research and Health Technology Assessment, Hall i.T., Austria
Tyll Krueger, PhD, Prof.
Wroclaw University of Science and Technology, Wrocław, Poland
PURPOSE: The availability of data to support health economics and outcomes research (HEOR) is increasing at an exponential rate. In the age of big data, cutting-edge machine learning algorithms, which have been tremendously successful in transportation and retail industry (e.g., Uber and Amazon) can play a promising role in HEOR. The objective of this workshop is to introduce the basics and key concepts in machine learning (ML), how machine learning can influence the field of HEOR, what are some of the potential pitfalls of machine learning and applications from health economics outcomes research that utilize machine learning.
DESCRIPTION: In this workshop, we will introduce the current landscape in HEOR through the lens of the state-of-the-art modern machine learning techniques. In particular, we will start with reviewing basics and key concepts of modern machine learning techniques for descriptive, predictive, prescriptive analytics. We will proceed with discussing how ML differs from statistical learning and modeling and provide an overview of selected ML algorithms such as deep neural networks, and random forests. We will then illustrate state-of-the-art applications of ML to HEOR– this will include use of ML for augmenting health economic modeling for cost-effectiveness analysis, developing ML-driven Apps or online tools for running complex models in real time, and synthetic data generation using ML. We will conclude by discussing potential pitfalls of ML in health outcomes research and promising future research directions. This workshop will be primarily geared towards the interested audience from the pharmaceutical industry concerned with health economic evaluation.
Turgay Ayer, Ph.D.
Georgia Institute of Technology, Atlanta, GA, USA
Jag Chhatwal, Ph.D.
Harvard University, Boston, MA, USA
Selin Merdan, Ph.D.
Value Analytics Labs, Boston, MA, USA
Grace Hsu, MSc
Cytel Canada Health Inc, Waltham, MA, USA
Michael Zoratti, MSc, PhD(c)
Cytel Canada Health Inc, Burlington, ON, Canada
Bami Oshinowo, BSc, MSc
CBPartners, London, United Kingdom
Mark Joinnides, MSE
COVAXX, Dallas, TX, USA
Hernan Rosenberg, PhD
University of Chile & Universidad de la Frontera, Santiago, MD, Chile
Antonia Morga, PhD
Astellas Pharma Ltd, Addlestone, United Kingdom
Neil Hawkins, PhD, MSc, MBA, CStat
Martin Scott, MSc
Numerus, Tübingen, Germany
Maiwenn J Al, PhD
Huajie Jin, MBBS, MSc
King’s College London, London, United Kingdom
James Robins, MD
Harvard T.H. Chan School of Public Health, Boston, MA, USA