Monday, 16 November
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.
14:30 - 15:00
Break
15:00 - 16:00
Spotlight Session
Scientific 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.
Breakout Session 1
The Meaning of Cure – the Search for What?
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.
Moderators
K. Jack Ishak, PhD
Evidera, St-Laurent, QC, Canada
Panelists
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
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.
Allocating Resources during a Pandemic- To HTA or Not to HTA?
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.
OVERVIEW:
The panel will debate the need for HTA of drugs developed specifically or repurposed for treating COVID-19 prior to recommending them for widespread prescribing and use. Amanda Adler will moderate the panel and provide an overview of the treatments available for managing COVID-19 and the evidence supporting their use. Dalia Dawoud will present the academic perspective and will make the case for using HTA to inform decisions relating to the choice of COVID-19 treatments to recommend for widespread use on a healthcare system level. Mark Chakravarty will present the pharmaceutical industry perspective, making the case for expedited access to all COVID-19 treatments based on clinical efficacy evidence using expedited HTA process. Kalipso Chalkidou will present the perspective of a non-governmental organisation (NGO) perspective and argue for the need for innovative approaches to value assessment and pricing of treatments to be used in pandemic situations similar to COVID-19.
Moderators
Amanda Adler, MD, PhD, FRCP
University of Oxford, Fowlmere, United Kingdom
Panelists
Mark R Chakravarty, BSc (Hons), MBChB, Dip. BA
Novartis Pharmaceuticals, Basel, Switzerland
Kalipso Chalkidou, PhD
Imperial College London, London, United Kingdom
Dalia Dawoud
National Institute for Health and Care Excellence (NICE), London, LON, United Kingdom
Will COVID-19 Accelerate the Adoption of Digital Health Technologies? Perspectives on Evaluating Digital Health Technologies during Global Disease Outbreaks
ISSUE:
Digital health technologies have the potential to improve access to care, empower patients and reduce costs, particularly in a world faced with the COVID-19 pandemic. It is unclear, however, which research designs are best for measuring the value of these innovations. For pharmaceuticals and other treatments, the gold standard is a randomized controlled trial (RCT). In the world of digital, however, RCTs' tightly prescribed protocols, long delays, and high cost may prove problematic for demonstrating real-world value. On the other hand, without robust evidence, the value of digital health innovations will remain unclear. In this issue panel, panelists will provide concrete examples of how the value of digital health innovations has been quantified through recent research, will debate the merits of different methods for quantifying the this value, and discuss how COVID-19 is changing the use and valuation of digital health technologies.
OVERVIEW:
This panel will debate alternative research designs for measuring the value of digital medicine and how digital medicines are likely to evolve as more treatment interactions are conducted remotely due to COVID-19. Dr. Kidholm will provide a summary of the different types of digital health innovations in use as well as an overview of some policies used to incentivize increased uptake of digital medicine. Ms. Kjær Rasmussen will describe how her research has used an RCT approach to measure the value of telemonitoring services. Dr. Shafrin will present his research measuring the value for digital medicine technologies—including chip-in-pill adherence technologies and digital, point-of-care diagnostic tests—in cases where RCT data are not available. Dr. Ossebaard will present findings from a systematic literature review of health technology assessments for eHealth. Presentations will be approximately 8 minutes with 20 minutes for panel discussion and 10 minutes for audience questions.
Moderators
Kristian Kidholm, PhD
University of Southern Denmark, Odense, Denmark
Panelists
Hans Ossebaard, PhD
National Health Care Institute (Zorginstituut Nederland), Diemen, NH, Netherlands
Maja Rasmussen, MS
University of Southern Denmark, Odense, Denmark
Jason Shafrin, PhD
PRECISIONheor, Los Angeles, CA, USA
Clinical Evidence Generation across the Lifecycle of High Risk Implantable Medical Devices
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.
Moderators
Rosanna Tarricone, MSc, PhD
SDA Bocconi School of Management, Milan, Italy
Panelists
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
Is It Time to Find Greater Efficiencies in HTA for Rare Diseases and Cell and Gene Therapies?
ISSUE
: There has been an explosion in the number of new cell and gene therapies and treatments for rare diseases in development. This will result in a heavy demand for health technology assessments. Ideally HTA might look at each treatment individually but demand in the system requires greater efficiency. Many biopharmaceutical
companies developing drugs individually invest in similar and sometimes overlapping work to develop evidence and economic models. Small-sized companies may not have the capabilities or finances to invest in HTA at an early stage. The panel will debate the trade-off between what is efficient and what is optimal to ensure fair HTA appraisals and patient access and how patient-led initiatives might help.
OVERVIEW
: The panel will debate the pros and cons of developing greater efficiencies in HTA for innovative therapies and explore approaches to balancing optimal HTA methods with greater efficiency. Josie Godfrey will moderate the session and describe the challenges of the increase in cell and gene therapies and orphan drugs. She will pose questions including: Will the HTA burden of cell and gene therapies and for rare diseases be too high for current approaches to continue? Small companies may not have the resources to develop evidence for HTA. Should we address this or accept it? Can we identify and agree upon areas where collaboration could help build efficiency in the system? What role is there for patient-led initiates? The panelists will represent different stakeholder and geographical perspectives. Ron Akehurst will represent a HTA perspective arguing that current approaches will continue to be effective without the need for major changes. Fleur Chandler will present a patient perspective arguing for greater collaboration and efficiencies in approach. Sachin Kamal-Bahl will represent an industry perspective that a pragmatic approach is needed to address the increasing demands on the system and identify efficiencies.
Moderators
Josie Godfrey, BA MA MA
JG Zebra Consulting, London, United Kingdom
Panelists
Ron Akehurst, DSc, Hon MFPHM
BresMed Health Solutions, Sheffield, DBY, United Kingdom
Fleur Chandler, PhD
Sanofi, Reading, RDG, United Kingdom
Fleur is an independent health economist, and has been in the pharmaceutical industry for many years, previously leading global health outcomes teams at GSK across many therapeutic areas. Fleur is a recognised industry expert in Health Technology Assessment. In the charity sector, drawing on personal experience in rare disease, and working with leading patient organisations Duchenne UK and PPMD in the US, she conceptualised and leads Project HERCULES, a unique collaboration across industry, academia and government bodies generating a gold standard disease level health outcomes evidence base in Duchenne Muscular Dystrophy.
Sachin Kamal-Bahl, PhD
SKB Consulting Inc., Lansdale, PA, USA
16:00 - 16:30
Break
16:30 - 17:30
GROUP DISCUSSION
COVID-19 and Hospital Procurement- The Role of Value Assessment for Medical Devices & Diagnostics in Pandemic Public Health Situations
Group Discussions are a new, two-part content type for ISPOR events: an on demand, pre-recorded session to be released before the live conference dates, and a follow-up hour-long live discussion during the live conference dates. The expectation for attendees is that they watch the on-demand content before the meeting, and then participate in a live discussion of the on-demand sessions during the conference.
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:
Moderators
Richard Charter, MSc
Alira Health, Basel, Switzerland
Richard is currently the director of Value Based Healthcare at ValueConnected, a company focusing on aligning key stakeholders to ensure adoption of medical technologies via procurement, reimbursement, and risk sharing agreements. Richard is also currently the co-chair of the ISPOR Medical Devices Interest Group, where he also teaches the short course on Market Access for Medical Devices. Richard is also the co-chair of HTAi Medical Device Interest Group, where he is frequently called on as a guest speaker. Richard is currently the industry advisor to COMED, an EU funded consortium measuring outcomes and costs in medical devices. Richard graduated from SDA Bocconi in Milan with a Masters in Healthcare Management, Economics & Policy, as well as from the University of Guelph in Canada, in Management Economics & Public Policy.
Speakers
Abdalla Abotaleb, PhD ,PharmD
World Health Organization, cairo, C, Egypt
Federico Augustovski, MSc, PhD
Institute for Clinical Effectiveness and Health Policy, Buenos Aires, Argentina
Federico Augustovski, MD, MSc, PhD, is the current director of Health Economic Evaluations and Technology Assessment at the Institute for Clinical Effectiveness and Health Policy (IECS), an independent non-profit organization affiliated to the University of Buenos Aires, a CONICET (National Scientific and Technical Research Council) center, and one of the few INAHTA Health Technology Assessments agencies in Latin America. Federico is the director of the WHO Collaborating Centre in Health Technology Assessment and Economic Evaluations at IECS. He is also the founding editor-in-chief for Latin America section of Value in Health Regional Issues, the ISPOR peer-reviewed journal for Latin America, Asia, and Central & Eastern Europe, Western Asia, and Africa. He is the director of the PAHO affiliated PROVAC Center of Excellence for decision making in vaccines. Federico leads a multidisciplinary team devoted to clinical and economic evaluations of new and existing preventive, diagnostic, and therapeutic technologies that provides research, education, and technical support with public and private health decision makers in Latin America. He is a professor of Public Health at the School of Public Health of the University of Buenos Aires, where he teaches courses for graduate and postgraduate students in Decision Sciences; Patient-Reported Outcomes Development in Health, as well as Health Economic Evaluations.
Federico earned his MD with honors at the University of Buenos Aires and is a specialist in family medicine. He practiced family medicine and was a staff physician for more than 20 years at the Family and Community Medicine Division of the Hospital Italiano de Buenos Aires. He received his MSc in epidemiology (Harvard School of Public Health). He was a European Union Scholar in health economics at the Centre for Health Economics at the University of York in the UK. His research production concentrates in health technology assessments and health economic evaluations methods and applications. He has published more than 70 PubMed-indexed papers.
Federico has served and serves ISPOR in several capacities during the past 10 years. Among other commitments, he was the first Latin American director on the board of directors, the founder and first president of the Argentine local chapter, the first chair of the Latin American Consortium, chair of the Research Excellence Award, president of Buenos Aires 2013 Regional meeting, member of the Health Science and Policy Council and Vision 2020 teams, as well as several Task Forces.
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
16:30 - 18:00
Posters and Exhibits Viewing
Use this time to browse the latest research through our virtual Posters and find solutions for your challenges with our Exhibits.
Educational Symposium
Insights Into Potential Patient and Caregiver Benefits of Changing Disease Course in AD- A Discussion on the Non-Economic Values of Future Potential Early Intervention
The overall burden of Alzheimer’s disease (AD) is immense, and is known to be experienced more intensely as the disease progresses. With disease progression comes an increased need for support from others to complete everyday tasks and activities, and what some call “a loss of self”. But preceding these large steps in disease advancement are more subtle changes occurring earlier, and these subtle changes themselves can have a profound impact on early stage patients and their families. Future potential treatments for use in early AD that delay progression to later stages will be valued as they can allow for more time to be spent in earlier, less burdensome, segments of the disease.
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.
Sponsor
Biogen
Moderators
Samantha Benham-Hermetz, ,
Alzheimer's Research UK, Cambridge, CAM, United Kingdom
Speakers
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
18:15 - 19:15
Social Hour
Meet and chat with ISPOR leaders during our nightly social hours. Attendance is limited, so be sure to join early to ensure you have a spot in our Social Hours!
GROUP DISCUSSION
Review And Identification Of Criteria For Value Assessment Of Medication Adherence-Enhancing Interventions
Group Discussions are a new, two-part content type for ISPOR events: an on demand, pre-recorded session to be released before the live conference dates, and a follow-up hour-long live discussion during the live conference dates. The expectation for attendees is that they watch the on-demand content before the meeting, and then participate in a live discussion of the on-demand sessions during the conference.
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.
Moderators
Mickaël Hiligsmann, PhD
Maastricht University, Maastricht, LI, Netherlands
Speakers
Tamas Agh, MD, PhD, MSc
Syreon Research Institute, Budapest, Hungary
Andrew Peterson, PharmD, PhD, FCPP
University of the Sciences, Philadelphia, PA, USA
Tuesday, 17 November
13:00 - 14:30
PLENARY SESSION
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, ON, 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 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.
14:30 - 15:00
Break
15:00 - 16:00
WOMEN IN HEOR SESSION
Adapting to the ‘New Normal’
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.
Moderators
Julia F. Slejko, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
Olivia Wu, PhD
University of Glasgow, Glasgow, United Kingdom
Olivia Wu, PhD is director of the Health Economics and Health Technology Assessment (HEHTA) Research Unit and William R Lindsay chair of Health Economics, at the University of Glasgow. She is also director of the Complex Reviews Support Unit, a national methods support unit for evidence synthesis funded by the UK National Institute for Health Research (NIHR). Olivia has expertise in a broad range of health technology assessment (HTA) methodologies and is interested in adapting and applying HTA methodologies in context. She has a particular interest in evidence synthesis and economic evaluations. Her research spans across a wide range of clinical areas and different types of health technologies (eg, pharmacological treatments, medical devices and diagnostic tests). Her work has informed clinical guidelines and health policy decisions, both at national and international levels. In addition to her research, Olivia has been a long-standing member of the NICE Technology Appraisal Committee and advisor to Healthcare Improvement Scotland. She has also been advisor to HTA agencies in Brazil, China, Taiwan and Thailand.
Speakers
Blythe Adamson, PhD, MPH
Flatiron Health, New York, NY, USA
Blythe Adamson is a Senior Quantitative Scientist at Flatiron Health. She uses health economics, math, epidemiology, and data science to research and identify high-value medicines in development. Her research has included dynamic transmission modeling of infectious diseases, novel microsimulation modeling methods in oncology, and cost-effectiveness studies to inform policy. Dr. Adamson received her PhD in Pharmacoeconomics and Masters in Public Health in Epidemiology from the University of Washington in Seattle. Before joining Flatiron, she worked on the development of HIV vaccines at Fred Hutchinson Cancer Research Center and informed Gates Foundation investing decisions with the Institute for Disease Modeling at Global Good.
Nancy Devlin, PhD
University of Melbourne, Melbourne, VIC, Australia
Nancy J. Devlin is director of the Centre for Health Policy at the University of Melbourne in Australia. Previously, she was director of research at the Office of Health Economics in London. Professor Devlin has more than 30 years’ experience in health economics and health outcomes research and serves as an advisor to international healthcare organizations, both in the public and private sectors. She is past-president of the EuroQol Group. She has published more than 100 peer-reviewed journal articles and a number of books in the field. She served as a director on the ISPOR board of directors from 2015-2017 and is now serving as the Society’s president for the 2019-2020 term.
Ebere Onukwugha, PhD, MS
University of Maryland School of Pharmacy, Baltimore, MD, USA
Louise Timlin, MSc
Eli Lilly and Company, Windlesham, Surrey, United Kingdom
15:00 - 16:30
Posters and Exhibits Viewing
Use this time to browse the latest research through our virtual Posters and find solutions for your challenges with our Exhibits.
Educational Symposium
Characterizing the Value in Coexisting Type 2 Inflammatory Diseases
The symposium will focus on Type 2 inflammation, which is at the basis of various diseases such as atopic dermatitis, asthma, nasal polyposis, allergic rhinitis, eosinophilic esophagitis, etc., all linked to the same pathway. Very often, patients experience more than one of these conditions which results in increasing the disease burden. Respect to that, it is essential to picture challenges to be overcome and opportunities of the patient pathway in order to develop new strategies for enhancing the therapeutic experience and investigate its economic aspects.
Sponsor
Sanofi
Moderators
Stefano Centanni, MD, PhD
Università degli Studi di Milano, Milan, MI, Italy
Stefano Del Giacco, MD
Università di Cagliari, Cagliari, MI, Italy
Speakers
Giorgio Walter Canonica, MD, PhD
Humanitas University, IRCCS Humanitas Research Hospital, Milan, Italy
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.
Enrico Giorgio Heffler, MD, PhD
Humanitas University, IRCCS Humanitas Research Hospital, Milan, MI, Italy
Claudio Jommi, PhD
SDA Bocconi School of Management, Milan, Italy
Jörg Kleine-Tebbe, MD
Allergy and Asthma Center Westend, Berlin, Germany
Giuseppe Turchetti, PhD
Scuola Superiore Sant'Anna, Pisa, PI, Italy
Gene Therapies- Is Europe Ready to Value and Reimburse the Next Medical Frontier?
Gene therapy is an innovative approach designed to address the genetic root cause of monogenic diseases. The lack of standardized evaluation methodologies for gene therapies pose a challenge for its medical and health technology assessment. The limits of the current reimbursement landscape in Europe, which has been set up for chronic therapies and incentivizes their development, have also led to an affordability challenge for single-dose gene therapies. One objective of this symposium is to provide an overview of the fundamentals of gene therapy, including its clinical application to treat rare diseases. Innovative solutions, such as the value-based assessment of gene therapies and use of novel reimbursement models, which can be used to overcome the challenges posed by gene therapies due to limits in the current system, will also be reviewed and discussed during this symposium.
MED-CON--00021-EMA 10/2020
Sponsor
Novartis Gene Therapies Inc.
Moderators
Omar Dabbous, MD, MPH
Novartis Gene Therapies, Bannockburn, IL, USA
Speakers
Michael Drummond, MCom, DPhil
University of York, York, YOR, United Kingdom
Michael Drummond, BSc, MCom, DPhil is professor of Health Economics and former Director of the Centre for Health Economics at the University of York. His particular field of interest is in the economic evaluation of health care treatments and programmes. He has undertaken evaluations in a wide range of medical fields including care of the elderly, neonatal intensive care, immunization programmes, services for people with AIDS, eye health care and pharmaceuticals. He is the author of two major textbooks and more than 650 scientific papers, and has acted as a consultant to the World Health Organization and the European Union. He has been President of the International Society of Technology Assessment in Health Care, and the International Society for Pharmacoeconomics and Outcomes Research. In October 2010 he was made a member of the National Academy of Medicine in the USA. He has advised several governments on the assessment of health technologies and chaired one of the Guideline Review Panels for the National Institute for Health and Care Excellence (NICE) in the UK. He is currently Co-Editor-in-Chief of Value in Health and has been awarded 3 honorary doctorates, from City University (London), Erasmus University (Rotterdam) and the University of Lisbon.
Mondher Toumi, MD, MSc, PhD
Aix-Marseille University, Paris, France
Professor Mondher Toumi is a MD by training and holds two MSc in Biostatistics, and in Biological Sciences (option pharmacology) and a PhD in Economic Sciences. He is professor of Public Health at Aix-Marseille University. After working for 12 years as a research manager in the Department of Pharmacology at the University of Marseille, he joined the Public Health Department in 1993. In 1995, he entered the pharmaceutical industry and worked there for 13 years.
Mondher Toumi was appointed global vice president at Lundbeck A/S in charge of health economics, outcome research, pricing, market access, epidemiology, risk management, governmental affairs, and competitive intelligence. In 2008, he founded Creativ-Ceutical, an international consulting firm dedicated to support health industries and authorities in strategic decision-making.
In February 2009 he was appointed professor at Lyon I University in the Department of Decision Sciences and Health Policies. The same year, he was appointed director of the chair of Public Health and Market Access. He launched the first European University Diploma of Market Access (EMAUD) an international course already followed by more than 350 students. Additionally, he recently created the Market Access Society to promote research and scientific activities around market access, public health and health economic assessment. Since 2009, he also chaired the Annual Market Access Day, a purely academic event. He is editor in chief of the Journal of Market Access and Health Policy (JMAHP), which is PubMed index.
Since September 2014, he joined the research unit EA3279 of the Public Health Department, at Aix-Marseille University (France) as full professor. Mondher Toumi is also visiting professor at Beijing University (Third Hospital).
He has been an active member of ISPOR from its creation. He served as co-chair of the Research Review Committee ISPOR Europe in 2012 and 2013. He contributed to Short Courses in ISPOR Asia and Europe. He is member of the Editorial Board of Value and Outcome Spotlight. From 2010 he contributed to ISPOR conferences with more than 196 posters and podium presentations and 11 issue panels and workshops. He co-Chaired two consecutive SIGs on orphan drugs. He also supported ISPOR African and Nigerian Chapter.
He is a recognized expert in health economics and an authority on market access and risk management. He has more than 200 scientific publications and oral communications and has contributed to several books. He just finished a book on Market Access soon to be available.
Johann-Matthias von der Schulenburg, PhD
Leibniz University Hannover, Hannover, Germany
16:30 - 17:00
Break
17:00 - 18:00
Breakout Session 2
Methodological and Normative Issues Arising in Recently Updated National Guidelines for Economic Evaluation
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:
. the Portuguese guidelines (
Marta Soares , 12 minutes) that changed recommendations on the perspective of analysis and explicitly defined principles for re-appraisal and further evidence requests, amongst others; . the EUnetHTA guidelines (
Conor Teljeur , 12 minutes) that identifies problems with the selection of comparators when not working on the efficiency frontier and considers issues with the use of surrogate outcomes, amongst others; and . the ongoing review of the NICE methods guide (
Ian Watson , 12 minutes), which is considering a wide range of topics, including exploration of uncertainty, factors and evidence considered in decision-making, issues for emerging technologies, and others. The panellists will reflect on the motivations, and controversies, associated with each topic area, drawing from the comments received from stakeholders (e.g. at public consultation), where available. The reflections made will consider who the guidelines are for, the process for identifying and formulating updates/revisions, and the process whereby stakeholders can input into the changes. The workshop will end with a structured interactive discussion (
Mark Sculpher, 17 minutes ).
Discussion Leader
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.
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
Carer Utilities in Cost-Utility Analyses (CUAS)- Crucial Information or Additional Burden of Uncertainty?
PURPOSE
: Informal carers’ burden is disproportionately high in some diseases. The inclusion of carer utilities in CUAs in these cases seem justified, however there are questions and uncertainties around their measurement, interpretation, and method of incorporation into the evaluation.
DESCRIPTION
: Noemi Muszbek will introduce the importance of carers in disease management, the current use of carer utilities in CUAs and implications for health technology assessments (HTAs). The various barriers to their use and the uncertainties in measurement, interpretation and inclusion will be outlined (8 minutes). Hareth Al-Janabi will discuss indirect elicitation methods, including the potential issues with validity and responsiveness of various preference-based quality of life measures in informal care and will compare the relative performance, potential value, and limitations of carer specific vs. generic instruments for HTAs using the example of four highly prevalent conditions (12 minutes). Andrew Lloyd will describe the elicitation of carer utilities with vignette studies in contrast to generic measures, considering their potential pitfalls and discussing recent examples. Vignettes studies also provide the opportunity to elicit post-death values, however their interpretation and significance can raise additional questions (12 minutes). Lastly, Edit Remak will discuss the ethical and methodological issues around the implementation of carer utilities, including the use of post-death values and the risks of conferring survival benefit to the carers from patients. A conservative method of implementation will be offered using an oncology example (12 minutes). The workshop will use real-time polling on the experience of audience members in the use of carer utilities, and the challenges and opportunities they see before and after the presentation. They will be invited to discuss the inclusion of carer utilities in CUAs, their implications for HTAs regarding the definition of value and the current thresholds, the suitability of methods and their impact on uncertainty.
Discussion Leader
Hareth Al-Janabi, PhD
University of Birmingham, Birmingham, United Kingdom
Andrew Lloyd, DPhil
Acaster Lloyd Consulting Ltd, London, United Kingdom
Andrew Lloyd is the Director of Acaster Lloyd Consulting Ltd, an outcomes research consultancy. Andrew has previously been the VP and Practice Lead at ICON PLC for 4 years, and a Director at Oxford Outcomes and United BioSource Corporation. Prior to 2001 Andrew had academic positions in Leicester, Oxford, and Aberdeen. His main research interests are in the assessment of patient-reported outcomes. Andrew’s work is concerned with assessing HRQL and patient preferences to support economic submissions for reimbursement to bodies like NICE. Andrew sits on the Executive Committee of the EuroQol group. He undertakes teaching at ISPOR and the University of York. He is a co-Editor at Value in Health. Andrew has undertaken work in a broad range of therapeutic areas including oncology, diabetes, asthma, and vascular disease. These projects have included assessments of quality of life; utility estimation; determination of patient preferences through discrete choice experiments; and development and validation of novel patient reported outcomes. He completed his DPhil in the Psychology Department at the University of York in 1997. Andrew has published over 85 peer reviewed articles in journals including: Medical Decision Making, Lancet, Value in Health, British Journal of Cancer and others. He has been keynote speaker at national and international conferences. He was the research co-Chair at the ISPOR European Conference in 2009. Andrew has supervised PhD students in the UK and US.
Noemi Muszbek, MSc
Visible Analytics, Reading, RDG, United Kingdom
Edit Remak, PhD
Visible Analytics, Oxford, OXF, United Kingdom
Spotlight Session
Is 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.
Breakout Session 2
How Should Pharmaceutical Companies and Patients Served By Health Systems Share the Value Generated By New Medicines?
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.
We will address three questions: (1) How is the value of branded medicines currently shared between pharmaceutical companies and patients served by health systems? (2) Is this appropriate given its expected effects on global R&D? and (3) Should value sharing differ when publicly funded R&D has contributed to product development?
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.
Moderators
Danny Palnoch, BSc
NHS England, LON, United Kingdom
Panelists
Patricia Danzon, PhD
University of Pennsylvania, Philadelphia, PA, USA
Jens Grueger, PhD
University of Washington, Boston Consulting Group, Freiburg, BW, Germany
Jens Grueger is a Director and Partner at the Boston Consulting Group based in Zurich Switzerland. He provides expert advice to companies in the life science industries on pricing and market access. Since July 2019, he is an Affiliate Professor of Health Economics at the CHOICE Institute at the University of Washington School of Pharmacy in Seattle, USA. He is the 2019-2020 President Elect for ISPOR, the leading professional society for health economics and outcomes research.
Prior to this, Jens had a distinguished career in the pharmaceutical industry. From 2011-2019, he was the Senior Vice President and Head of Global Access for F. Hoffmann-La Roche based in Basel, Switzerland. Under his leadership, Roche created rapid, broad and sustainable patient access for 12 new medicines and expanded access to essential cancer medicines in lower- and middle-income countries from 29 to 60%. Before this Jens was the Vice President and Head of Global Market Access Primary Care at Pfizer, based in New York and London (2009-2011), Head of Global Pricing & Health Economics at Novartis Pharma in Basel, Switzerland (1999-2009) and Director of Health Economics at the German affiliate of SmithKline Beecham Pharma in Munich (1994-1997). He founded Diversified Health Systems, a start-up company providing internet-based disease management services to physician networks in Europe (1997-1999).
Jens holds a MSc (Diplom) in Medical Statistics and Theoretical Medicine, and a PhD in Mathematical Statistics from the Technical University of Dortmund, Germany. He has authored more than 40 publications in biometrics, cancer epidemiology, and health economics.
Beth Woods, MSc
University of York, Heslington, York, YOR, United Kingdom
Integrating Patient Preference into Health Technology Assessment- Can Patient Preferences be Incorporated into the ICER?
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.
Following a recent publication outlining how patient preferences might support NICE’s committees, Jacoline Bouvy will elaborate on the role that patient preference data have in the HTA process from the perspective of an HTA body, and how these might sit alongside the evidence on the cost-effectiveness of a technology. Given the lack of a reliable method for integrating patient preferences into HTA, Kevin Marsh will outline a promising approach that uses patient preference studies to estimate value in terms of ‘QALY equivalents’. One of the critical components of this approach – the consistency of valuations across patient groups – will be assessed based on a review of published patient preference studies. The discussion leaders will use real-time polling to collect the audience’s views on alternative methods for integrating patient preferences into HTA.
Moderators
Esther de Bekker-Grob, PhD
Erasmus University Rotterdam, Rotterdam, Netherlands
Panelists
Jacoline Bouvy, PhD
National Institute for Health and Care Excellence, London, United Kingdom
Mendwas Dzingina, PhD
Pfizer, Tadworth, United Kingdom
Kevin Marsh, PhD
Evidera, Newport Pagell, BKM, Great Britain
Kevin Marsh, PhD, is Executive Director at Evidera in London, UK. He specializes in the use of preference data and decision analysis to inform health decisions, including pipeline optimisation, authorisation, reimbursement, and prescription decisions.
Dr Marsh’s research interests include stated and revealed preference methods, decision modelling, and MCDA. He has applied these and other research techniques for a range of organisations, including both regulatory and industry clients. He actively contributes to the methodological development of these techniques. He is currently co-Chairing the ISPOR Task Force on the Use of MCDA in Health Care Decision-Making, and is a co-Convenor of the Campbell and Cochrane Economic Methods Group.
Dr Marsh completed his PhD at the University of Bath, specialising in economic valuation techniques. After a year at Oxford University, he joined the Matrix Knowledge Group in London, before joining Evidera in April 2012.
Reflecting on the Failures of Real World Data in COVID-19 – Should Global Health Learn from Oncology or Are the COVID-19 Real World Data Challenges Too Unique?
ISSUE
: CoViD-19 research has been subject to an unprecedented pace of clinical research efforts. With this also comes a high frequency of prolific failures, that diminishes the public’s confidence in research and slows the path from scientific results to action. Arguably, the most prolific research failures in CoViD-19 have come from studies using Real World Data (RWD). In areas such as oncology, RWD has been used with much success for over a decade and underlying know-how and technologies have evolved as researchers have learned from their mistakes. It is particularly surprising and concerning to see many of the CoViD-19 RWD research efforts make the same mistakes that were made in oncology this past decade. There is a wealth of experience and technology available for Real World studies that many CoViD-19 researchers may not be aware of, and it will be crucial for the world’s health to ensure that this capacity is adequately shared globally.
OVERVIEW
: This Panel will address the current shortcomings of Real World studies in CoViD-19 and how the experience and technologies already available in areas like oncology may aid to ensure the feasibility, validity and relevance of future CoViD-19 Real World studies. Key questions to be addressed are: What are the key challenges of logistics and data validity that currently exist in academic, non-for-profit, and commercially available CoViD-19 Real World Data; What are the technologies and processes that have or are being developed to address these issues and what are the realistic timelines? In this unprecedented research pace, what is the appropriate balance between retrospective and prospective data collection? Which technologies already exist for oncology Real World Data that have not adequately been utilized in CoViD-19 and what are the major reasons for this?
Moderators
Kristian Thorlund, MSc, PhD
Cytel, Hamilton, ON, Canada
Panelists
Danielle Bargo, MSc
Flatiron Health, New York, NY, USA
Edward J Mills, PhD, FRCP (Edin)
McMaster University, Hamilton, ON, Canada
Mark Shapiro, PhD
xCures, DURHAM, NC, USA
18:15 - 19:15
Social Hour
Meet and chat with ISPOR leaders during our nightly social hours. Attendance is limited, so be sure to join early to ensure you have a spot in our Social Hours!
GROUP DISCUSSION
Effect Of Public And Private Partnership In Healthcare Financing To Improve Health Outcomes
Group Discussions are a new, two-part content type for ISPOR events: an on demand, pre-recorded session to be released before the live conference dates, and a follow-up hour-long live discussion during the live conference dates. The expectation for attendees is that they watch the on-demand content before the meeting, and then participate in a live discussion of the on-demand sessions during the conference.
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.
Moderators
Rita Karam, PharmD, PhD
Lebanese University, BEIRUT, Lebanon
Speakers
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
Wednesday, 18 November
13:00 - 14:30
PLENARY SESSION
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, Tirol, 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.
14:30 - 15:00
Break
15:00 - 16:00
GROUP DISCUSSION
Expanding COA In 2020- Guidance In RWE And Improving Communication Between Different Stakeholders
Group Discussions are a new, two-part content type for ISPOR events: an on demand, pre-recorded session to be released before the live conference dates, and a follow-up hour-long live discussion during the live conference dates. The expectation for attendees is that they watch the on-demand content before the meeting, and then participate in a live discussion of the on-demand sessions during the conference.
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.
Moderators
Katja Rudell, PhD, MSc
Parexel International, Chittering, United Kingdom
Speakers
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
ISPOR Chapter Meeting
ISPOR Spain Chapter Meeting
New and detailed proposals from the Pricing and Reimbursement Advisor Committee (CAPF) has been published in Spain. These proposals will be the basis for an open discussion about the implications of these measures in the future of the Pricing and Reimbursement processes and negotiations in Spain.
This Chapter Meeting will be presented in Spanish only. Simultaneous interpretation will not be available.
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.
15:00 - 16:30
Educational Symposium
Bridging the Gap- Pathways for Regulatory and Health Technology Assessment of Histology Independent Therapies
Histology independent therapies (also known as tumour agnostics) are a new class of medicines that target a genomic alteration within a tumour regardless of where it is in the body. These novel treatments face significant methodological and policy challenges related to evidence development and acceptance, value assessment and reimbursement pathways, and diagnostic infrastructure availability.
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.
Sponsor
Office of Health Economics
Moderators
Lotte Steuten, PhD, MSc
Office of Health Economics, London, LON, United Kingdom
Speakers
Rosa Giuliani, MD
The Clatterbridge Cancer Center, Liverpool, ON, United Kingdom
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.
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.
Entela Xoxi, PhD PharmD MSc
Catholic University of Rome, Roma, RM, Italy
Posters and Exhibits Viewing
Use this time to browse the latest research through our virtual Posters and find solutions for your challenges with our Exhibits.
16:30 - 17:00
Break
17:00 - 18:00
Breakout Session 3
Stated-Preference Research in the Real World- Making a Difference in Public-Health Policy Making
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.
Discussion Leader
Reed Johnson, BS
Duke Clinical Research Institute, Durham, NC, USA
Marcel Jonker, PhD
Erasmus University Rotterdam, Rotterdam, ZH, Netherlands
Axel 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.
Shelby Reed, PhD, RPh
Duke Clinical Research Institute, Durham, NC, USA
Shelby D. Reed, PhD, RPh, is professor in the Departments of Population Health Sciences and Medicine and the Duke-Margolis Center for Health Policy at Duke University. Dr. Reed has 20 years of experience in economic evaluation, health services research, and health policy. Dr. Reed has extensive expertise in designing and conducting trial-based and model-based cost-effectiveness analyses of medical diagnostics and interventions in numerous therapeutic areas. She was a member of the ISPOR Task Forces that published recommendations for Good Research Practices for Cost-Effectiveness Analysis Alongside Clinical Trials in 2005 and 2015. She has led a wide range of economic and epidemiological studies using secondary data from healthcare claims, clinical trials, surveys, and disease registries. In her evaluations of health policy issues, she has developed computer models to analyze the potential economic impact of trends in clinical trial design, changes in reimbursement policies, financial incentives and the regulatory process in the development of orphan drugs, and the societal value of alternative approaches to identifying drug safety problems. Dr. Reed currently leads the Center for Informing Health Decision at the Duke Clinical Research Institute. Dr. Reed received her pharmacy and doctoral degrees from the School of Pharmacy at the University of Maryland and completed her training at the University of Washington.
Aligning Cost-Effectiveness Analysis Methods with Real World Patient Preferences
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.
The new approach requires estimates of new preference parameters (the health-elasticity of utility, risk aversion in health, and new time-tradeoff (TTO) estimates of WTP for QoL gains in terms of reduced life expectancy (LE). We will estimate these parameters in interactive parts of our workshop (see separate section). Leaders will each speak approximately 15 minutes to describe the new model and its implications, leaving 30 minutes for audience discussion and interactive participation. The presentation should interest academics, HEOR practitioners from industry, public policy, patient advocacy, and clinically oriented organizations (i.e., most of ISPOR).
Discussion Leader
Darius Lakdawalla, PhD
University of Southern California, Los Angeles, CA, USA
Charles Phelps, PhD
University of Rochester, Pittsford, NY, USA
How Can We Balance Quantifying the Broad Societal Benefits of Vaccines with Paying for Them?
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.
Moderators
Daniel Ollendorf, PhD
Tufts Medical Center, Boston, MA, USA
Panelists
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
Could International Reference Pricing “Go Viral” after COVID-19 and Limit Patient Access to Innovative Medicines?
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?
Moderators
Andras Incze, PhD, MBA
Baden-Wuerttemberg Cooperative State University Germany & Akceso Advisors AG, Basel, BS, Switzerland
Panelists
Jaime Espin, PhD
Andalusian School of Public Health, Granada, GR, Spain
Lou Garrison, PhD
University of Washington, Seattle, WA, USA
Lou Garrison, PhD, is professor emeritus in The Comparative Health Outcomes, Policy, and Economics Institute in the School of Pharmacy at the University of Washington, where he joined the faculty in 2004.
For the first 13 years of his career, Dr. Garrison worked in non-profit health policy at Battelle and then the Project HOPE Center for Health Affairs, where he was the Director from 1989-1992. Following this, he worked as an economist in the pharmaceutical industry for 12 years. From 2002-2004, he was vice president and head of Health Economics & Strategic Pricing in Roche Pharmaceuticals, based in Basel, Switzerland.
Dr. Garrison received a BA in Economics from Indiana University, and a PhD in Economics from Stanford University. He has more than 150 publications in peer-reviewed journals. His research interests include national and international health policy issues related to personalized medicine, benefit-risk analysis, and other topics, as well as the economic evaluation of pharmaceuticals, diagnostics, and other technologies.
Dr. Garrison was elected as ISPOR President for July 2016-June 2017, following other leadership roles since 2005. He recently co-chaired the ISPOR Special Task Force on US Value Frameworks. He was selected in 2017 by PharmaVOICE as being among “100 of the Most Inspiring People” in the industry. He recently received the PhRMA Foundation and Personalized Medicine Coalition 2018 Value Assessment Challenge First-Prize Award as lead author on a paper on “A Strategy to Support the Efficient Development and Use of Innovations in Personalized and Precision Medicine.”
Zoltan Kalo, PhD
1) Semmelweis University; 2) Syreon Research Institute, Budapest, ON, Hungary
Zoltán Kaló is a professor of Health Economics at the Center for Health Technology Assessment of Semmelweis University in Budapest, Hungary. Before moving to Semmelweis University in July 2019 he was the founder and co-director of an international master program in Health Policy, Planning, and Financing at Eötvös Loránd University (ELTE).
Dr. Kaló is also the founder and leader of Syreon Research Institute, an international research corporation specializing in health policy, health economic modeling, and technology assessment.
He has 25 years of international experience in academia and industry, specializing in health systems design, HTA implementation, health economics and outcomes research, patient access, and pricing policies of healthcare technologies.
Dr. Kaló serves as a policy advisor to public decision makers and global healthcare corporations. He is a Scientific Committee member of the Innovative Medicines Initiative 2 Joint Undertaking (IMI2 JU). He was a director of ISPOR between 2012-2014, and the chair of ISPOR Central and Eastern European Network Executive Committee between 2013-2015.
Multi-National HTA- Does IT Really Benefit Public Health and Promote Access to Innovation?
ISSUE
:
Do multi-national HTA and procurement processes such as EUnetHTA, FINOSE and BeNeLuxAI improve access to innovation and benefit public health or are they merely additional barriers to access? Multi-national HTA (MNHTA) processes exist across Europe but perspectives differ on their utility and efficiency. Do MNHTA and procurement processes such as EUnetHTA, FINOSE and BeNeLuxAI improve access to innovation and benefit public health or are they additional barriers to access? OVERVIEW
:
Bringing together one of the founders of EUnetHTA, a pharmaceutical innovator with a track record of engaging in multiple MNHTA processes and a consultancy conducting fresh research into MNHTA activity in Europe, this issue panel will provide varied, informed perspectives on what multi-national HTA in Europe has achieved to date and how MNHTA should evolve to enable access and positively impact public health. Presentation will include fresh research on European HTA body frequency of participation in a variety of multi-national processes (EUnetHTA, FINOSE, BeNeLuxAI), drawing conclusions on which HTA bodies participate most and in what therapy areas/modalities HTA bodies focus. Equal time is allotted to panellists’ opening remarks and audience debate and discussion. HTA policymakers and influencers, HTA decision-makers and value and access strategists will especially benefit from this session.
Moderators
Gavin Outteridge, MA
AESARA Europe, London, United Kingdom
Panelists
Finn Børlum Kristensen, MD, PhD
University of Southern Denmark, Hilleroed, 84, Denmark
Finn Børlum Kristensen is an international strategic consultant in HTA and HEOR implementation and management and is a professor in Health Services Research and HTA at University of Southern Denmark since 1999. He is also external lecturer at Copenhagen Business School. He headed the Coordinating Secretariat of the European Network for HTA, EUnetHTA (www.eunethta.eu) from its inception in 2006 and was chairman of the EUnetHTA Executive Committee until 2016. Dr. Kristensen directed the Danish Centre for HTA (DACEHTA) from its establishment in 1997 until 2009. His PhD is in Epidemiology, and he is a medical specialist in public health. He also worked as a primary care physician for several years. He publishes frequently in scientific journals and was editor of a Health Technology Assessment Handbook. He is now a consultant to public and private organizations and companies. Dr. Kristensen served on the ISPOR Board of Directors from 2011-2013 and chaired the ISPOR HTA Council 2013-2019.
Vanessa Schaub, PhD
F.Hoffmann-La Roche, Switzerland
18:15 - 19:15
Social Hour
Meet and chat with ISPOR leaders during our nightly social hours. Attendance is limited, so be sure to join early to ensure you have a spot in our Social Hours!
Thursday, 19 November
13:00 - 14:00
Breakout Session 4
Social Cost Value Analysis- Do We Need an Alternative Health Economic Evaluation Paradigm?
ISSUE:
: Conventional cost effectiveness analysis is broadly used in the context of formal Health Technology Assessments (HTAs), but rests on a set of problematic assumptions and conventions. It cannot capture citizens’ preferences for health care resource allocation beyond individual gains in length and quality of life and the associated uncertainty. This is particularly relevant for increasingly important areas such as the evaluation of orphan medicinal products and precision (or “personalized”) medicine.
OVERVIEW:
: Our starting point is the assumption that Health Technology Assessment (HTA) should inform decision makers acting on behalf of citizens covered by a collectively financed health scheme. Accordingly, their decisions should respect the social norms, preferences, and value judgments of the population. However, the currently prevailing conventional health economic evaluation approaches, in particular cost effectiveness analyses (CEAs) relying on the ICER (incremental cost effectiveness ratio) metric, are based on a reductionist efficiency criterion and do not capture the full social value of health technologies. This may result in recommendations contradicting social norms and preferences, which have been demonstrated by the “empirical ethics” project. As an example, the limitations imposed by the linearity assumptions underlying the ICER metric are illustrated by the inadequacy of the method to reflect the size of programs and the prevalence (or “rarity”) of a health problem, with potentially far-reaching implications not only for the evaluation of rare and ultra-rare disorders. Thus, measuring social preferences may require a payment vehicle different from the (purely selfish) consumers’ perspective applied in most stated preference studies. Empirical studies using a citizens’ perspective (resting on the budget impact of programs) will be presented. Social cost value analysis will be discussed as an emerging concept that may capture citizens’ social preferences better than conventional CEAs. Methodological challenges, policy implications and further research needs will be debated.
Moderators
Michael Schlander, MD, PhD, MBA
German Cancer Research Center (DKFZ), Heidelberg, BW, Germany
Panelists
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
Use of Health Technology Assessment in a Global Pandemic; How Will the Development of a COVID-19 Vaccine Affect Reimbursement and Value Based Price in Local Markets?
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.
Moderators
Richard Stanford, PharmD, MS
AESARA, Chapel HIll, NC, USA
Panelists
Andrew Briggs, DPhil
London School of Hygiene & Tropical Medicine, London, LON, United Kingdom
Andrew is a professor of Health Economics and the London School of Hygiene & Tropical Medicine. Previously, he held the William R Lindsay Chair in Health Economics at the University of Glasgow. Following a sabbatical at Memorial Sloan Kettering Cancer Center, New York in 2016/17 he remains a visiting investigator, collaborating with Dr. Peter Bach on value frameworks for oncology medications.
Andrew has expertise in all areas of health economic evaluation -- he has published over 200 articles in the peer-reviewed literature. He has particularly focused on statistical methods for cost-effectiveness analysis. This includes statistical methods for estimation of parameters for cost-effectiveness models as well as statistical analysis of cost-effectiveness alongside clinical trials. He also has a more general interest in epidemiological methods, in particular the use of prognostic scoring methods for predicting health outcomes and the relationship with heterogeneity in cost-effectiveness.
Andrew took a leadership role as co-chair of the Joint Society for Medical Decision Making (SMDM) and ISPOR Task Force on Modelling Methods. The Task Force, which was responsible for producing a set of seven papers covering all aspects of modeling methods applied to medical decision making and health technology assessment. He is also the author of two successful textbooks, one published by OUP entitled Decision Modelling for Health Economic Evaluation, and another published by Wiley entitled Statistical Methods for Cost-Effectiveness Analysis.
In addition to his academic activities, Andrew is also director & principal of Avalon Health Economics, a small consultancy company based in New Jersey, USA, which focuses on providing support to companies bringing products to market in both US and ex-US.
Anna Kaltenboeck, MA
Memorial Sloan Kettering Cancer Center, New York, NY, USA
How to Model the Cost-Effectiveness of Histology-Independent Oncology Therapies within Health Technology Assessments (HTAs)
PURPOSE
: To discuss the methodological and data challenges faced when developing cost-effectiveness models for histology-independent oncology therapies, and learnings derived so far from HTAs.
DESCRIPTION
: The recent regulatory approval of histology-independent oncology medicines has seen a paradigm shift in the classification of cancers, from a focus on tumour location to assessment based on the presence of specific tumour biomarkers. The evidence generated for these histology-independent medicines poses particular challenges: single-arm basket trials are commonly designed, which evaluate surrogate endpoints (response-based rather than time-based) and include different tumour types with relatively small samples per type. These trials have considerable potential for heterogeneity in prognosis and standard-of-care therapies across different tumour types, which further complicates comparability and cost-effectiveness assessments. This workshop will offer participants the current HTA experiences on histology-independent oncology therapies.
Raquel Aguiar-Ibáñez will provide an overview of the landscape related to histology-independent therapies and the challenges these present in terms of data availability and comparability. Dawn Lee will discuss the modelling approaches and types of data analyses that have been presented when assessing the cost-effectiveness of histology-independent therapies (e.g. response-based analyses, previous treatment line analyses and Bayesian hierarchical models), identifying the limitations. Gianluca Baio will discuss key considerations when building a model, including the need to explore heterogeneity and uncertainty related to treatment response and effectiveness. Jacoline Bouvy will provide recommendations for modelling and data analyses to meet HTA requirements, identifying what additional evidence may need to be generated and how HTA bodies might view evidence submissions for histology-independent therapies.
Discussion Leader
Raquel Aguiar-Ibáñez, MSc
Merck Sharp & Dohme Ltd, Haarlem, NH, Netherlands
Gianluca Baio, PhD
University College London, London, SRY, United Kingdom
Jacoline Bouvy, PhD
National Institute for Health and Care Excellence, London, United Kingdom
Dawn Lee, MMath, MSc
BresMed Health Solutions Ltd., Sheffield, DBY, United Kingdom
The Digital Endpoints Ecosystem and Protocols (DEEP) Initiative- A Collaborative, Multi-Stakeholder Approach to Defining and Developing Standards for Digital Endpoints
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.
Digital technology, including smartphones, wearables and other mobile sensors, has the potential to deliver new insights and provide richer, more patient-relevant measures of the impact of disease and interventions. Digital endpoints are becoming more sophisticated and are on the cusp of wide use – for clinical development, and presenting evidence to regulators, payers, healthcare providers, and patients. Marc Walton will use use-cases, both actual and conceptual, to present how digital endpoints differ from non-digital, including contexts of use and richness of the acquired knowledge. He will also identify challenges that need to be overcome to achieve the potential for digital endpoints. [10 min] Kai Langel will then present DEEP. DEEP engages and connects expertise across industry, consulting, patient-centered measurement science, academia, digital health, product design, and regulatory agencies. [10 min] Mark Larkin will run the interactive workshop, eliciting feedback and input on DEEP from the audience. This will be based around the draft framework and strategic design of DEEP, using live polling and text-based responses to pressure-test key components of the draft frameworks from stakeholder perspectives. [20 min] Finally Sheela Upadhyaya will provide a HTA perspective and anchor a panel Q&A session and then address audience questions. [15 min]
Discussion Leader
Kai Langel, BSc
Janssen, Titusvile, NJ, USA
Mark Larkin, PhD
Vitaccess Ltd, Oxford, LON, United Kingdom
Mark is the chief executive office & founder of Vitaccess. He has more than 20 years of experience in consulting, specializing in European P&R for the last 10 years. Mark founded Vitaccess to harness innovative smartphone and dashboarding technology to build the Company's multi award winning MyRealWorldTM platform, offering real-time digital RWE research. Mark has led all Vitaccess' international RWE studies, across oncology and rare/orphan diseases, and led partnerships with patient advocacy organizations (now >30). Mark has a BA and MA in Natural Sciences and a PhD in experimental psychology.
Sheela Upadhyaya, Dip
Independent HTA perspective, London, United Kingdom
Marc Walton, MD,PhD
Janssen, Ashton, MD, USA
Prevent the Breakdown of the Healthcare System- How to Use SIR Model to Allocate Medical Resources Better
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.
Discussion Leader
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
The Use of Surrogate Endpoints in Regulatory and HTA Submissions- Methodological Considerations, Current Status and Best Practice Recommendations
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.
In this workshop, Sylwia Bujkiewicz will present the methodologist perspective, introducing the concept of surrogacy, outlining the levels of evidence used to validate the surrogate endpoints and the latest analytic techniques proposed by NICE Decision Support Unit (DSU) for their evaluation (12 minutes). Huseyin Naci will outline the regulatory context, describing the rationale for and extent of using surrogate endpoints in regulatory approvals, the limitations of the evidence used to support the surrogacy relationship in recent approvals and the societal impacts of the current regulatory context (12 minutes). Oriana Ciani will describe the HTA context, providing an overview of the use of surrogate endpoints in HTA submissions, and how the HTA context differs from the regulatory one, discussing the impact of their use on decision-making (12 minutes). Dalia Dawoud will present the proposed update of NICE methods in relation to the use of surrogate endpoints, comparing it with other international HTA agencies’ requirements (12 minutes). The workshop is relevant to attendees from various backgrounds including academics, regulators, HTA agencies and pharmaceutical companies.
Discussion Leader
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.
Oriana Ciani, PhD
SDA Bocconi School of Management, Milan, MI, Italy
Dalia Dawoud
National Institute for Health and Care Excellence (NICE), London, LON, United Kingdom
Huseyin Naci, PhD
London School of Economics and Political Science, London, United Kingdom
14:00 - 14:30
Break
14:30 - 15:30
Breakout Session 5
Contact-Tracing Policies for SARS-COV-2- Lessons Learned from Austrian, German and Polish Modeling Studies Informing Decision Makers
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.
Discussion Leader
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
Introduction to Machine Learning for Health Economic and Outcomes Research
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.
Discussion Leader
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
The COVID-19 Trial Tracker- A Framework to Provide Real-Time Updates on Emerging Evidence
PURPOSE
: This workshop will describe the benefits of clinical trial trackers as an alternative to traditional systematic reviews. We will illustrate this in the context of the COVID-19 pandemic, a period defined by high uncertainty and rapidly shifting research priorities. Our discussion will present the tracker as a framework which is readily adapted across the clinical spectrum. The workshop will particularly highlight practical challenges and solutions to setting up trial trackers based on lessons learned with the global CoViD-19 Trial Tracker (
https://www.covid19-trials.com ).
DESCRIPTION
: In response to the COVID-19 pandemic, clinical trial research of candidate interventions began emerging at an unprecedented rate. Indeed, by early June 2020, over 1400 trials had been registered internationally. The logistics of managing this wealth of emerging evidence presented researchers and policymakers with a unique but pressing challenge. In response, we established the COVID-19 Trial Tracker (
https://www.covid19-trials.com ). The functionality of this platform is amenable to a number of research contexts. We will discuss means of adapting this platform through a generalized framework to improve systematic reviews or, in essence, any exercise that provides regular monitoring and access by cross functional teams to a dynamic body of data. In this workshop, we will review the logistics of establishing our tracker and discuss key lessons learned for future implementations. Kristian Thorlund will discuss the purpose and motivation of the tracker as a means to catalogue and organize trial data for decision makers, investigators, and the general public. Michael Zoratti will present our approach to amalgamating evidence from key sources, including national and international databases, and the iterative means by which we categorized evidence. Grace Hsu will provide an in-depth look at the functionality we developed to disseminate information and the technical requirements to implement these solutions.
Discussion Leader
Grace Hsu, MSc
Cytel Canada Health Inc, Waltham, MA, USA
Kristian Thorlund, MSc, PhD
Cytel, Hamilton, ON, Canada
Michael Zoratti, MSc, PhD(c)
Cytel Canada Health Inc, Burlington, ON, Canada
The SAR-2-COV Vaccine- What Does an Optimal Vaccination Strategy Look like
ISSUE
: Amidst the ongoing COVID-19 pandemic, a SAR-2-COV vaccine will likely be one of the most effective solutions to ease the spread of the virus. However, historical challenges with vaccination strategies for global pandemics (e.g. the 2009 H1N1 pandemic) come into focus. In preparation for the coming SAR-2-COV vaccination, there is a critical need for public health systems to collaborate with the industry and think through an optimal and cost-effective strategy that supports the sustainability of the healthcare system.
OVERVIEW
: This issue panel will bring together stakeholders from public health bodies and industry to discuss the optimal SAR-2-COV vaccination strategy approach for public health systems. Bami Osinowo will moderate the panel and provide an overview of the key challenges and learnings from the 2009 H1N1 influenza vaccine rollout as well as pose key questions for the panelists to debate: How can public health systems approach their SAR-2-COV vaccination strategy? How do government agencies ensure effective coordination of the SAR-2-COV supply chain? How can government agencies measure the impact of the SAR-2-COV vaccine post-launch? What kind of data, if any, should we collect to assess the effectiveness of the vaccinations and inform future decisions? What will be the most cost-effective initial strategy between vaccinating the entire population and vaccinating high-risk groups (e.g. the elderly) or groups more likely to spread the disease (e.g. <25 years of age)? How do external factors influence the cost-effectiveness of each strategy (e.g. supply chain issues, current economic crisis, etc.)? Mark Joinnides will represent the manufacturer perspective, offering insights on the unique challenges of developing and scaling a SAR-2-COV vaccine during the COVID-19 pandemic. Hernan Rosenberg will represent the global public health perspective and discuss the vaccination strategies that have been historically successful / unsuccessful as well as any proposals currently being developed for the SAR-2-COV vaccination strategy.
Moderators
Bami Oshinowo, BSc, MSc
CBPartners, London, United Kingdom
Panelists
Mark Joinnides, MSE
COVAXX, Dallas, TX, USA
Hernan Rosenberg, PhD
University of Chile & Universidad de la Frontera, Santiago, MD, Chile
Is Intention-to-Treat Analysis Still the Gold Standard, or Should All HTA Authorities and Regulators Now Embrace the ICH E(9)R(1) Addendum on Estimands and Sensitivity Analyses?
ISSUE
: The ICH E(9)R(1) addendum raises the question of whether estimating treatment effects on the basis of the intention-to-treat (ITT) principle should always be seen as the “gold standard” when establishing a causal relationship between intervention and outcomes in a clinical study. The design of appropriate Estimands, and the identification of suitable strategies for “intercurrent events”, such as treatment switching or rescue medication, are brought to the fore in the ICH E(9)R(1) addendum, and seen as a way to bridge across multiple stakeholders’ interests and needs. In addition, the addendum appears to widen the focus beyond the issue of missing data when addressing post-randomization events that may be related to treatment and outcomes. Against this context, it becomes critical to consider the question of what HTA considerations should be taken into account when designing Estimands and agreeing on strategies for intercurrent events
OVERVIEW
: The topic will benefit a wide range of participants, from decision makers, HEOR, HTA and regulatory experts, statisticians, clinical development and patients. The panel will debate the pros and cons of the ICH E(9)R(1) addendum with a specific focus on implications for HTA bodies and considerations for Regulatory agencies. Antonia Morga will introduce and moderate the discussion, and pose questions for the panellists to debate, including: Is a multiplicity of Estimands the best way to reconcile conflicting evidence needs from different stakeholders? Does the addendum introduce further divergence between Regulatory and HTA objectives? What does this all mean, ultimately, for patients? Each panellist will speak for 12 minutes, allowing at least 15 minutes for audience debate and discussion.
Moderators
Antonia Morga, PhD
Astellas Pharma Ltd, Addlestone, United Kingdom
Panelists
Neil Hawkins, PhD, MSc, MBA, CStat
University of Glasgow, Glasgow, United Kingdom
Neil is a Professor of Health Technology Assessment (HTA) at the University of Glasgow. He was previously Vice President leading the global Health Economics practice at ICON PLC following its acquisition of Oxford Outcomes Ltd, where he was a member of the Board of Directors.
Over the previous fifteen years, he has participated in methodological and applied research regarding the relative and cost- effectiveness of health technologies. In addition to participating individual evaluations, Neil has published articles discussing methods for network meta-analysis, cost-effectiveness modeling, value-based pricing, and the placebo effect. More recently, he has become interested in application of HTA “thinking” during the process of technology development and the development of conceptual models of value.
Neil received Master’s Degrees in Health Economics (York) and Applied Statistics (Sheffield Hallam), a BSc and PhD in Pharmacology (University of Bristol), and an MBA (University of Oxford). He is also a Chartered Statistician for the Royal Statistical Society, UK.
For more details please see: https://neilhawkins.wordpress.com/
Nick Latimer, MSc, PhD
ScHARR, University of Sheffield, Sheffield, DBY, United Kingdom
Martin Scott, MSc
Numerus, Tübingen, Germany
14:30 - 15:45
Spotlight Session
Novel 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
