Sat 2 Nov
8:00 - 12:00
SHORT COURSE MORNING SESSION
Tools for Reproducible Real-World Data Analysis
Level: Intermediate
Track: Real World Data & Information Systems
This course will focus on the concepts and tools of reproducible research and reporting of modern data analyses. The need for more reproducible tools in health economics and outcomes research is growing rapidly as analyses of real world data become more frequent, involve larger datasets, and employ more complex computations. This course will cover the principles of structuring and organizing a modern data analysis, literate statistical analysis tools, formal version control, software testing and debugging, and developing reproducible reports. Numerous real-world examples and an interactive class exercise will be used to reinforce the concepts and tools introduced. Participants who wish to gain hands-on experience are required to bring their laptops with R and RStudio installed.
Faculty Member
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.
Rachael Sorg, MPH
Flatiron Health, New York, NY, USA
Rachael Sorg is a Senior Quantitative Scientist at Flatiron Health, where she focuses on using real-world data derived from electronic health records to gain insights into cancer treatment trends and health outcomes. Prior to joining Flatiron, she worked at Analysis Group, where she conducted health economics and outcomes research using clinical trial, administrative claims, and other real-world data sources. Ms. Sorg received her Master of Public Health focusing on Health Policy from the Yale School of Public Health.
New! Why All the Hype? Nordic Data Explained
Level: Introductory
Track: Real World Data & Information Systems
Nordic data has become a hot topic in epidemiology, HEOR, and other observational research in recent years, and for good reason. Denmark, Finland, Iceland, Norway, and Sweden maintain datasets covering the entire Nordic population of over 26 million inhabitants for the duration of their lifetimes. The data includes information on demographics, diagnoses, prescription drugs, socioeconomics, PROs, disease severity, and more. All of this data may all be linked through unique, patient-level social security numbers. The coverage and breadth of data available to researchers in the Nordic countries is globally unparalleled. This course focuses on describing the content and structure of the Nordic data in terms of the variety of ways patients are included, how long they are followed up, what variables are available, and the quality of the data. This leads directly into a discussion about how this data can benefit researchers as well as the key limitations. The course will also consider practical aspects of using Nordic data, including data access from legal and procedural perspectives. Comparisons to other well-known European data such as CPRD will be explored. Key applied examples of analyses using Nordic data will be presented to illustrate the usage and possibilities of this data, including registry-based randomized clinical trials. Note: The applied section of the course will have you assess the feasibility of using Nordic data to address your own research questions, in discussion with course leaders. Please be prepared with a research question. No computer programs are needed for this course.
Faculty Member
Fredrik Borgström, MSc, PhD
Quantify Research and LIME/MMC, Karolinska Institutet, Stockholm, Sweden
Fredrik Borgström holds an MSc in Political Science and Economics from Uppsala University, Sweden and a PhD in Health Economics from Karolinska Institutet, Sweden. He has over 18 years’ experience in providing consultancy services to industry, private, and public organizations and institutions within the field of health economics and outcomes research and market access and reimbursement.
Prior to co-founding Quantify Research in 2011 he held positions such as vice president at i3 Innovus (Managing the Swedish operations) and director at European Health Economics. Fredrik has participated in and managed numerous projects in several disease areas, including osteoporosis, rheumatoid arthritis, osteoarthritis, oncology, multiple sclerosis, menopausal symptoms, and cardiovascular disease.
His research covers methods such as modeling, data collection, and statistical analysis of retrospective and prospective data. Fredrik is an affiliated researcher at the LIME/MMC department at Karolinska Institutet. Fredrik has been lead author and co-author of more than 90 articles published in scientific journals.
Kirk Geale, MSc
Quantify Research AB, Stockholm, Sweden
Ingrid Lindberg, MSc
Quantify Research, Stockholm, Sweden
Ingrid Lindberg is a Sr Research Analyst at Quantify Research, and holds a MSc degree in Economics from Stockholm School of Economics. Ingrid joined the Quantify team in 2017 and has experience in market access, including cost-effectiveness analysis and health economic modelling, as well as observational register-based real-world data studies. Ingrid has primarily worked in the fields of dermatology, and musculoskeletal and inflammatory disease areas in the Nordics as well as select European markets.
Introduction to the Design & Analysis of Observational Studies of Treatment Effects Using Retrospective Data Sources
Level: Intermediate
Track: Study Approaches
Retrospective studies require strong principles of epidemiologic study design and complex analytical methods to adjust for bias and confounding. This course will provide an overview of the structures of commonly encountered retrospective data sources with a focus on large administrative data, as well as highlight design and measurement issues investigators face when developing a protocol using retrospective observational data. Approaches to measure and control for patient mix, including patient comorbidity and the use of restriction and stratification, will be presented. Linear multivariable regression, logistic regression, and propensity scoring analytic techniques will be presented and include examples using SAS code that can later be used by participants. This course is an introductory course designed to prepare participants to take intermediate and advanced observational research courses.
Faculty Member
Nahila Justo, MBA, MPhil, MSc
Evidera, Stockholm, Sweden
Nahila Justo is the Director Nordic Region, Data Analytics, RWE at Evidera. Prior to joining Evidera, she worked for 12 years in the industry where she designed and led numerous outcomes research studies, including retrospective database analyses and chart reviews; literature reviews; health-economics models; strategic consulting; stakeholder engagement and advocacy pieces. Mrs. Justo experience spans across a wide range of therapeutic areas, including but not limited to different cancers (breast, lung, prostate, ovarian, sarcoma), haematological malignancies (different lymphoma and leukaemia indications), cardiovascular diseases (heart failure, diabetes), immune-moderated diseases (psoriasis, psoriatic arthritis, axial spondylitis), and in the central nervous system (dementia, insomnia, Alzheimer’s disease, multiple sclerosis).
Ms. Justo is a PhD Candidate at the Karolinska Institute and holds a Master in Business Administration from the Stockholm School of Economics, an MPhil in European Studies from the University of Salamanca, an MSc in Economics from the Inter-American Development Bank and the University Torcuato Di Tella and a BA and MA in Political Science and International Relations from the National University of Rosario. Her work has been published in peer-reviewed journals including the Oncologist, Value in Health, BMJ Open, Pharmacoepidemiology and Drug Safety, European Radiology, Expert Review in Pharmacoeconomics and Outcomes Research, European Journal of Cancer Care, International Journal of Gynaecological Cancer, and Annals of Oncology.
Bradley Martin, PharmD, PhD, RPh
University of Arkansas for Medical Sciences, Little Rock, AR, USA
Dr. Bradley Martin is currently Professor and was the founding head of the Pharmaceutical Evaluation and Policy (PEP) Division at the University of Arkansas for Medical Sciences College of Pharmacy. The Pharmaceutical Evaluation and Policy Division offers graduate and professional instruction and conducts research in pharmacoeconomics, patient reported outcomes, pharmaceutical economics, and large health claims data base analysis. Dr. Martin received his PharmD from the University of Illinois and earned his PhD in Pharmacy Care Administration from the University of Georgia. Dr. Martin’s research efforts have focused on conducting retrospective observational comparative effectiveness and economic analyses using large administrative data sets and national health surveys. He conducts policy analyses, and develops cost effectiveness models and has contributed to the understanding of the opioid epidemic. Dr. Martin has over 90 peer-reviewed manuscripts published, which have been collectively cited over 4000 times, and his work has been funded by NIH, AHRQ, VA, and a variety of research foundations as well as partnerships with the pharmaceutical industry. Dr. Martin is contributing to national policy research on opioids and health care financing and has recently lead an international effort to improve the conduct and reporting of CER observational research organized by three national associations: ISPOR, NPC, AMCP.
Introduction to Patient-Reported Outcomes Assessments
Level: Introductory
Track: Patient-Centered Research
Patient-reported outcomes (PROs) are widely used to evaluate the impact of health technologies, practice innovations, or changes in health policy from the patients' perspective. This course is designed to familiarize people with the range of PRO domains and the role they can play in evaluating the effects of healthcare interventions. This includes symptoms (including those that arise as side-effects of treatment), functioning, general health perceptions, and health-related quality of life. The faculty will describe the steps that researchers generally go through in order to develop and test a new PRO measure. This will include qualitative concept elicitation work, item generation, cognitive interviewing, testing measurement models (when appropriate), and finally, assessment of validity and reliability. The course will also cover issues such as the role of the recall period and cultural/linguistic translation of measures. This is an entry level course which assumes only a passing familiarity with patient-reported outcomes.
Faculty Member
Theresa Coles, PhD
Duke University, Durham, NC, USA
Dr. Theresa Coles is an Assistant Professor in the Center for Health Measurement in the Department of Population Health Sciences at Duke University. She specializes in the development and psychometric evaluation (reliability, validity, responsiveness, and responder thresholds) of patient-reported outcome (PRO) measures. She is interested in developing tools, methods, and processes to support patients in communication with clinicians about their health status or goals/expectations, and developing methods to improve the interpretability of PRO scores.
Prior to joining the Duke faculty in 2018, Dr. Coles worked in the Patient-Centered Outcomes Assessment group at RTI Health Solutions for almost 10 years, where collaborated with colleagues to develop and evaluate PRO measures for use in clinical trials and clinical practice. Dr. Coles received her PhD in Health Policy and Management (Decision Sciences and Outcomes Research) from the University of North Carolina at Chapel Hill.
Elements of Pharmaceutical/Biotech Pricing
Level: Introductory
Track: Health Policy & Regulatory
This course will give participants a basic understanding of the key terminology and issues involved in pharmaceutical pricing decisions. It will cover the tools to build and document product value including issues, information, and processes employed (including pricing research), the role of health economics, and the differences in payment systems that help to shape pricing decisions. These tools will be further explored through a series of interactive exercises. This course is designed for those with limited experience in the area of pharmaceutical pricing and will cover topics within a global context.
Faculty Member
Renato Dellamano, PhD
MME Europe & ValueVector (Value Added Business Strategies), Milan, Italy
Dr. Dellamano, President of MME Europe and founder and President of ValueVector, had been for seven years (from 1996 to 2003) the Head of Strategic Pricing at the global Headquarters of the Swiss pharmaceutical giant Hoffmann-La Roche. In his consultancy activity and during his tenure as global pricing lead at Roche, Dr. Dellamano was instrumental in analyzing and developing global pricing and reimbursement strategies for products in numerous therapeutic areas. A Health Economist and a former Professor of Hospital Management, Dr. Dellamano combines a solid methodological background with extensive global and local business experience in the areas of strategic planning, business development, pharmaceutical pricing and reimbursement, market access, and health economics.
Jack Mycka
Medical Marketing Economics LLC (MME), Montclair, NJ, USA
As MME’s Global President & CEO, Jack provides critical global support for strategic marketing and pricing decisions to clients in the biotech and pharmaceutical industries. As a recognized expert consultant since 2001, he has successfully completed engagements encompassing many product classes and therapeutic areas and markets, including chronic care, oncology, injectable, and other specialty products all centered on value and its interaction with pricing, payers, reimbursement, and marketing in general at both MME and predecessor organizations. Medical Marketing Economics (MME) is a global leader in the development of value-based strategies and research for health care goods and services. MME’s value-based solutions provide our clients with unique guidance, insights, marketing strategies, and tactics. We identify and address appropriate opportunities, problems, and questions through practical experience, academic rigor, business acumen, and a passion for our work. We help our clients appreciate, understand, and arrive at informed decisions that capture value and thereby succeed. Please visit www.m2econ.com to learn more about our methods and experience. During his tenure at Roche Laboratories Inc., Jack established that company’s Pharmaceutical Pricing and Contracting Department. As the Director of this group, he successfully increased the size and scope of the group’s activities dramatically to cover a multitude of strategic, tactical, and operational issues involving pricing, reimbursement, and discounting in both the United States and other major global markets. Market segments covered globally, include market access/managed care, biologics, hospital, oncology, government, as well as emerging pricing and reimbursement issues. Previously, Jack spent ten years as a commercial banker in corporate lending, managing relationships for customers ranging in size from multibillion-dollar organizations to entrepreneurial firms and not-for-profit organizations. He received a BS in Finance from Lehigh University.
New! Fitting the Structure to the Task- Choosing the Right Dynamic Simulation Model to Inform Decisions about Healthcare Delivery
Level: Introductory
Track: Methodological & Statistical Research
The course aims to provide a basic overview of common simulation modeling methods that can be applied in health care delivery research. This course will first review of traditional questions posed for health economics and the traditional modelling approaches employed to answer these questions. Faculty will then introduce the types of questions that might be posed when assessing health care delivery and the simulation models that can be used to address these questions using the "ISPOR Dynamic Simulation Modeling Application in Health Care Delivery Research Emerging Good Practices Task Force report" as a basis. Faculty will subsequently detail the specifics of three common simulation modeling approaches (system dynamics, discrete event simulation, and agent based models), the types of problems that each can address, and their advantages and disadvantages. A case study will be used to illustrate the advantages and disadvantages of each type of modeling method.
Faculty Member
Elisabeth Fenwick, PhD
Pharmerit International, Oxford, OXF, United Kingdom
Elisabeth Fenwick is a senior director in the Modeling and Meta-Analysis team at Pharmerit International, based in Oxford in the UK.
Liz provides scientific and strategic support to HE projects globally. She has extensive experience in economic evaluation and health economic modeling having worked in the field for over 20 years. She has worked on a variety of projects in a wide range of disease areas including oncology, respiratory, infectious diseases, cardiology, ophthalmology, and orphan diseases.
Liz has also contributed to methods in the field, in particular relating to decision analytic modeling and simulation methods, probabilistic decision analytic modeling and value of information analysis. Liz was a member of the ISPOR joint task force on good research practices in modeling and a co-author on the joint taskforce paper on uncertainty and is currently co-chairing the ISPOR task force assessing emerging good practice in value of information analysis for research decisions. Liz is also a member of the editorial board for Pharmacoeconomics.
Liz has a PhD and MSc in Health Economics as well as an MSc in Operations Research and joined Pharmerit from ICON plc where she led the modeling team for the global HE group. Prior to her consultancy career, Liz spent over 15 years as an academic working at University of York, McMaster University, and most recently University of Glasgow.
Maarten J. IJzerman, PhD
Office of Health Economics, Melbourne, Australia
Maarten J. IJzerman is a VCCC professor and head of Cancer Health Services Research in the Faculty of Medicine, Dentristry and Health Sciences of the University of Melbourne in Australia. He also holds a fractional appointment as a professor of Health Technology Assessment in the University of Twente, where he was the Vice-Dean in the Faculty of Science. Maarten is an active global researcher with multiple research visits and collaborations in the USA, UK and Canada. He is an active ISPOR member, serves in different ISPOR taskforces and committees, and promotes active collaboration between ISPOR and the European Cancer Organisation (ECCO). In 2016, he joined the European Society for Medical Oncology (ESMO) Cancer Medicines working group dealing with the disparities in access to cancer drugs. Maarten is a member of the board of two hospitals in the Netherlands, responsible for quality and safety and strategy/innovation.
Deborah Marshall, PhD
University of Calgary, Calgary, AB, Canada
Deborah Marshall, PhD is a Professor in the Department of Community Health Sciences, University of Calgary, Arthur J.E. Child Chair in Rheumatology Outcomes Research and former Canada Research Chair, Health Services and Systems Research. Her research program focuses on the measurement of preferences, cost-effectiveness analysis, and simulation modeling of health services and interventions. Deborah has over 20 years of research experience in health technology assessment agencies, academic institutions, and industry settings in Canada, US, and Europe. She is a founding co-investigator of the Patient and Community Engagement Research (PaCER) Program at the University of Calgary co-leads the economics and stated preferences research platforms for the Canada-Netherlands Personalized Medicine Network in Childhood Arthritis and Rheumatic Disease (UCAN CANDU). Deborah is an active member of the ISPOR as the past-President of the Board of Directors, and as a member of the Patient Preferences Special Interest Group. She is a co-author on the three ISPOR Task Force Reports for Good Research Practice – Checklist for Conjoint Analysis in Health, Conjoint Analysis Experimental Design and Statistical Methods for the Analysis of Discrete-Choice Experiments. She also chaired the two reports from the ISPOR Dynamic Simulation Modeling Application in Health Care Delivery Research Emerging Good Practices Task Force.
Introduction to Modeling
Level: Introductory
Track: Methodological & Statistical Research
This course gives a brief overview of different decision-analytic model types and provides an introduction to Markov modeling techniques and their practical application in economic evaluation and outcomes research. Faculty will present analytic approaches including deterministic cohort simulation and Monte Carlo microsimulation, and will provide some technical instructions for modelers. Participants learn about the concepts of variability, uncertainty, probabilistic sensitivity analysis (PSA), and cost-effectiveness acceptability curves (CEAC). Additionally, faculty will use the recommendations of the ISPOR-SMDM Joint Modeling Good Research Practices Task Force to explore when and how modeling should be used in economic evaluation and which are the suitable model techniques. This course is designed for those with some familiarity with modeling techniques.
Faculty Member
Uwe Siebert, MD, MPH, MSc, ScD
UMIT- University for Health Sciences, Medical Informatics and Technology, and Harvard Chan School of Public Health, Hall i.T., Austria
New! Health State Utility (HSU) Recommendations for Identification and Use of HSU Data in Cost-Effectiveness Modeling
Level: Intermediate
Track: Patient-Centered Research
This course will be based on the soon to be published Health State Utility (HSU) Good Practices Task Force Report: Recommendations for Identification and Use of HSU Data in Cost-Effectiveness Modelling.
https://www.ispor.org/heor-resources/good-practices-for-outcomes-research/article/identification-review-and-use-of-health-state-utilities-in-cost-effectiveness-models A recent review of economic models in cardiovascular disease indicates that the identification of utilities for the models was not based on formal literature review methods, was not transparent, failed to accurately cite original sources and failed to accurately report the actual values used. In addition researchers sometimes adjusted values without clear justification, assumed no benefit over baseline for comparators, and ignored the impact of adverse events. A recent ISPOR Good Practices Task Force, led by John Brazier, was convened to review these issues and to propose guidance for model developers. The course will describe the main recommendations from the the report including the SpRUCE checklist and will also highlight areas where further methodological work is needed.
This course requires some knowledge of utilities.
Faculty Member
John E. Brazier, MSc PhD
University of Sheffield, Sheffield, United Kingdom
John Brazier is Professor of Health Economics and Dean of the School of Health and related Research (ScHARR) at the University of Sheffield. He has more than 25 years’ experience of conducting economic evaluations of health care interventions for policy maker and published over 200 peered reviewed papers. He has a particular interest in the measurement and valuation of health for economic evaluation where he has published widely. He is perhaps best known for his work in developing a preference-based measure of health for the SF-36 (SF-6D), but with colleagues has further developed and extended these methods to a number of specific condition including measures in mental health (ReQoL), asthma, cancer, overactive bladder, dementia and epilepsy. His research has also examined issues including methods of reviewing measures, mapping between measures, valuation methods (including the use of DCE) and more recently he has been developing ways to incorporate equity concerns such as burden of disease into the weights applied to QALYs. He has been an adviser to NICE on HTA methods and was a member of the NICE Technology Assessment Committee. He is a member of the Euroqol Executive Group.
Currently he is leading a project to develop a new broader generic measure of quality of life for use in economic evaluation. It is funded by the UK MRC and the EuroQoL Research Foundation in collaboration with colleagues at the Universities of Sheffield and Kent, the Office for Health Economics and NICE, together with colleagues in 5 other countries (Australia, Argentina, Germany, USA and Singapore). For more information about the ‘Extending the QALY’ project see: https://scharr.dept.shef.ac.uk/e-qaly/welcome/
Helene Chevrou-Severac, PhD
Celgene International, Boudry, Switzerland
After a PhD in Economics and Econometrics, Hélène Chevrou-Séverac worked for 10 years in the Academic Field as researcher and professor assistant in Health Economics and Applied Econometrics in France and Switzerland. She taught advanced econometric & statistical analyses of real-world data in the field of health economics to PhD students, as well as economic evaluation methods to Master students.
In 2007, Hélène joined the medical industry in Health Economics, Reimbursement & Market Access. She developed a strong experience in health economics, market access, HTA, and reimbursement for medical devices and medical nutrition for patients. In 2014, she joined the pharmaceuticals industry as head of Health Economics & HTA for Takeda, where she developed a Centre of Excellence in HTA. Since 2017, she has been working in Celgene International, as global head of Outcomes Research in the Medical Affairs function.
Since 2014, Hélène has also been involved in IMI projects on real-world data (GetReal) and big data (HARMONY), working closely with patients’ associations, regulators, and HTA members. She was also a scientific external advisor to the French HTA agency (HAS) at the time of the development of the CEESP.
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.
8:00 - 17:00
FULL DAY SHORT COURSES
Introduction to Health Economics and Outcomes Research
Level: Introductory
Track: Economic Evaluation
This course is designed to teach clinicians and new researchers how to incorporate health economics into study design and data analysis. Participants will first review the basic principles and concepts of health economic evaluations, then discuss how to collect and calculate the costs of different alternatives, determine the economic impact of clinical outcomes, and how to identify, track, and assign costs to different types of health care resources used. Different health economic models and techniques will be demonstrated, including cost-minimization, cost-effectiveness, cost-benefit, cost-utility, and budget impact analysis. Decision analysis, sensitivity analysis, and discounting will all be demonstrated and practiced.
Faculty Member
Lorne Basskin, PharmD
Charleston University, Charleston, WV, USA
Lorne Basskin received his Bachelor's degree in business at the University of Toronto and worked as an accountant with a national firm and subsequently developed his own consulting business. He later received his PharmD from the University of the Pacific and completed a post-doctoral residency with Valley Medical Center in Seattle in 1995. Dr. Basskin went on to serve as an Associate Professor in Clinical Practice at two different schools of pharmacy in the United States, and was the Director of Post-Graduate and Continuing Education there for seven years. In 2002, Dr. Basskin started his own Medical Education firm, specializing in education for pharmacists and other health care professionals. From 2005 to 2011 he held various positions with HealthSouth Corporation, a for-profit group of 100 rehabilitation and long-term acute care hospitals. Most recently he served as their National Director of Pharmacy Clinical and Information Services and was involved in implementing the Cerner system of electronic medical records throughout the network. After a two year period with Wingate University College of Pharmacy as their Regional Dean, he moved to Asheville, NC, where he carries on a consulting practice specializing in education about health care technology and outcomes research.
Dr. Basskin has written and spoken extensively on the topics of Pharmacoeconomics and Outcomes Research, and published a book on the topic in 1998. He has over 40 peer-reviewed publications and has made over 120 presentations on both clinical and research related matters. He is on the Editorial Board of several National Pharmacy and Medical publications and serves as a peer reviewer for several professional organizations. He has written continuing education programs including that of diabetes, asthma, and Medicare reimbursement. Additionally, he has conducted over 100 one and two day workshops on the meaning and use of pharmacoeconomics for health care decision makers and health care researchers.
13:00 - 17:00
SHORT COURSE AFTERNOON SESSION
Use of Propensity Scores in Observational Studies of Treatment Effects
Level: Intermediate
Track: Study Approaches
In observational research, issues of bias and confounding relate to study design and analysis in the setting of non-random treatment assignment where compared subjects might differ substantially with respect to comorbidities. No control over the treatment assignment and the lack of balance in the covariates between the treatment and control groups can produce confounded estimates of treatment effect. Faculty will explain how propensity scores can be used to mitigate confounding through standard observational approaches (restriction, stratification, matching, regression, or weighting). The advantages and disadvantages of standard adjustment relative to propensity score-based methods will be discussed. Details of propensity score methodology (variable selection, use, and diagnostics) will also be discussed. The course will also elaborate briefly on risk adjustment models that collapse predictors of outcomes and their use relative to propensity scores. This course is designed for those with little experience with this methodology but some knowledge of observational databases.
Faculty Member
Jeremy Rassen, ScD
Aetion, Inc., New York, NY, USA
Dr. Jeremy A. Rassen, ScD, is co-founder and chief scientific officer at Aetion, Inc., a company that provides software to evaluate the effectiveness, safety, and value of medical treatments. At Aetion, Dr. Rassen leads the scientific effort around designing methodology for obtaining and communicating medical evidence from real-world data.
Dr. Rassen was formerly an assistant professor of Medicine at the Brigham and Women's Hospital and Harvard Medical School, where he focused on methodology for improved validity and reach of pharmacoepidemiology and comparative effectiveness research, including research into propensity score and instrumental variable methods. Before coming to the Brigham and Women’s Hospital, Dr. Rassen worked in Silicon Valley in numerous computer and software companies, including Hewlett-Packard and Epiphany, Inc. His focus was on high-performance software for the creation and analysis of large marketing databases.
Dr. Rassen received his Bachelor’s degree from Harvard College and his Doctorate degree in Epidemiology from the Harvard School of Public Health.
John D Seeger, PharmD, DrPH
Optum, Waltham, MA, USA
Dr. John Seeger is a pharmacoepidemiologist and Chief Scientific Officer at Optum Epidemiology. He is also Adjunct Assistant Professor in Epidemiology at the Harvard School of Public Health. Dr. Seeger has conducted dozens of studies that have addressed regulatory drug safety issues across a wide range of drugs and disease conditions. Most of this work has involved the use of health insurance claims databases as platforms for pharmacoepidemiology, so Dr. Seeger's methodological expertise focuses on research issues encountered in such settings. He has worked extensively with propensity scores and related methods that mitigate confounding by collapsing covariates. Additionally, he is a co-Instructor in courses on propensity scores and is the past President of the International Society for Pharmacoepidemiology (ISPE).
New! US Payers – Understanding the Healthcare System™
Level: Introductory
Track: Health Policy & Regulatory
The US healthcare system is a hybrid system that provides access to care via various channels and means. It can be categorized as: governmental systems (Medicare, Medicaid, Veterans Health Administration, and Department of Defense, etc), private markets (privately sponsored regional and national health plans), and providers assuming responsibility for access and coverage via mechanisms such as Provider Networks, Integrated Delivery Networks (IDNs), Accountable Care Organizations (ACOs), etc. These elements are quite heterogeneous and characterized by different coverage and access mechanisms. This introductory course will cover the roots of the current US system, its evolutionary process, and the consequent independence of these different parts of the system. The focus will be on value and affordability as defined by cost, access, and quality. While all hold value and affordability as important goals, the different stakeholders take various perspectives and define these goals differently. This results in different objectives, incentives, and principles under which they operate. The intent of this course is to better understand characteristics of the different parts of the US healthcare system and the role of various decision makers within it. The course will cover their structure, scope, processes, and perspectives as well as their approach to balancing access, costs and quality. The course will also cover how industry and others produce evidence to support access and reimbursement decisions and how the evidence is used by decision makers. This session will facilitate the increased level of understanding of these disparate systems and emphasize the need to effectively communicate evidence to various access decision makers to support and enhance evidence driven decision making on value and affordability. This course is designed for those having limited experience in understanding the structure of the US Healthcare system, including its various subsystems and how they operate. The course will focus on how these entities differ in perspectives, coverage mechanisms, and the means and evidence they use to define and achieve both value (access and quality) and affordability.
Faculty Member
Finn Børlum Kristensen, MD, PhD
University of Southern Denmark, Hilleroed, 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 organisations and companies.
Dr. Kristensen served on the ISPOR Board of Directors from 2011-13 and has chaired the ISPOR HTA Council since 2013.
Erin Lopata, PharmD, MPH
Precision for Value, Pittsburgh, PA, USA
Erin is currently a Senior Director on the Access Experience Team at Precision for Value. Erin also serves as an adjunct professor at the University of Pittsburgh School of Pharmacy, where she coordinates and teaches a course orienting students to managed care pharmacy principles. Prior to joining Precision, Erin led commercial formulary development and specialty pharmacy management for UPMC Health Plan. Erin also served as the residency program director and a preceptor for the PGY-1 Managed Care Residency program at UPMC Health Plan. Erin received her PharmD from the University of Pittsburgh School of Pharmacy, and her Master in Public Health (MPH) from the University of Pittsburgh Graduate School of Public Health. She completed a postgraduate year 1 (PGY1) managed care pharmacy practice residency at UPMC Health Plan.
James F. Murray, PhD
Eli Lilly and Company, Carmel, IN, USA
Jim Murray, PhD is a research fellow within Lilly’s Global Patient Outcomes and Real-World Evidence (GPORWE) Center of Expertise (COE). The GPORWE COE is a multi-disciplinary group that supports the other functions within GPORWE and Lilly in the development and commercialization of Lilly products. Jim provides expertise and scientific support for the following topics within the COE for Real World Evidence and the use of RWE in Clinical Development, Observational and Pragmatic Study Design, and Health Policy issues.
Jim has been actively involved in ISPOR since its inception. In addition to the development of the US Health System short course, he is actively involved in the development of the ISPOR Competency Framework, the ISPOR Education Council, and the ISPOR Institutional Council. Jim has a Masters in Information Systems and a PhD in Decision Sciences and Operations Research both from the University of Wisconsin Madison.
Laura Pizzi, PharmD, MPH, RPh
Rutgers University, Piscataway, NJ, USA
Dr. Laura Pizzi is professor and director of the Center for Health Outcomes, Policy, and Economics (HOPE) at Rutgers University. For the past 20 years, she has led interdisciplinary teams of outcomes methodologists, statisticians, and clinicians to develop and conduct cost and outcome analyses on pharmacological therapies as well as a variety of non-pharmacological interventions. A particular interest is developing scientific evidence to inform the US translation and implementation of cost-effective interventions to improve the quality of care for older adults. She has testified before the US House of Representatives Committee on Ways and Means on Medicare overspending in beneficiaries with end stage renal disease., co-directed an Agency for Healthcare Research and Quality (AHRQ) evaluation on pharmaceutical quality, and co-edited a text on economic evaluation of health interventions. She is a member of the Editorial Board for the journal PharmacoEconomics, deputy editor of American Health and Drug Benefits, and chair of the ISPOR Faculty Advisor Council. She has published more than 90 peer-reviewed manuscripts and 1110 peer reviewed poster or podium presentations and has over 15 years of teaching and mentoring experience which has been recognized through faculty awards.
Meta-Analysis & Systematic Literature Review
Level: Intermediate
Track: Study Approaches
Meta-analysis may be defined as the statistical analysis of data from multiple studies for the purpose of synthesizing and summarizing results, as well as for quantitatively evaluating sources of heterogeneity and bias. A systematic literature review often includes meta-analysis and involves an explicit, detailed description of how a review was conducted. This course highlights and expounds upon four key areas: 1) impetus for meta-analysis and systematic reviews; 2) basic steps to perform a quantitative systematic review; 3) statistical methods of combining data; and 4) an introduction to methods for indirect comparisons. The material includes practical examples from the published literature relevant to pharmacoeconomic and PRO research. This course is designed for those with little experience with meta-analysis and includes interactive exercises. Participants who wish to gain hands-on experience must bring their personal laptops with Microsoft Excel for Windows installed.
Faculty Member
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/
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.
Statistical Methods for Health Economics and Outcomes Research
Level: Introductory
Track: Economic Evaluation
This course will provide an introduction to statistical concepts with an emphasis on the use of techniques commonly employed in pharmacoeconomics and outcomes research. Faculty will begin by introducing the concept of random variables and will then proceed to discuss the foundations of statistical estimation and the testing of hypotheses, followed by a discussion of the importance of correlating between variables and the use of regression techniques. The differences between a classical (frequentist) approach to statistics and a Bayesian view of probability will also be outlined. This course is intended for participants with little (or rusty!) statistical training.
Faculty Member
James Lewsey, PhD
University of Glasgow, Glasgow, United Kingdom
Jim Lewsey, PhD, is a reader in Medical Statistics and joined the University of Glasgow in 2007 having previously held posts at the London School of Hygiene and Tropical Medicine (2003-2007), University of Otago (2001-2003), Eastman Dental Institute-UCL (1998-2001), and University of Glasgow (1996-1998). He was awarded Chartered Statistician status from the Royal Statistical Society in 2010 and Chartered Scientist from the Science Council in 2012.
His personal research interests stem from methodological challenges faced when analyzing observational and experimental medical data and have included prognostic model development in the presence of missing data, continuous outcome monitoring of long-term outcomes, modelling dental caries data, and design of cluster randomized trials. His current methodological interests include multi-state survival analysis and modelling of data from natural experiments, and in his applied research he is developing a portfolio of alcohol research. Jim leads the Analysis of Linked Health Data (ALDA) program within HEHTA and is deputy lead of the IHW research theme 'Data Science - Using routine administrative data and record linkage for research'.
Jim is program director and teaches on the MSc in Health Technology Assessment. He also teaches and coordinates a medical statistics course on the Masters in Public Health.
Gerd K. Rosenkranz, PhD
Medical University of Vienna, Vienna, Austria
Gerd Rosenkranz, PhD, studied Mathematics and Physics at the University of Heidelberg (Germany) and worked as a biostatistician with the German Cancer Research Center. After having received his PhD, he was with the pharmaceutical industry for almost 30 years covering preclinical and clinical development of new therapies, mainly with Hoechst (now Sanofi-Aventis) in Frankfurt, Germany and Sandoz/Novartis in Basel, Switzerland.
During his 20+ years in Novartis he led the Statistics Oncology Group, later joined the Statistical Methodology Group and became scientific officer biostatistics. In this role he was representing the statistics function in the review of clinical studies and development plans of Novartis Pharmaceuticals in cardiovascular, immunology, respiratory, neurology, among others. Since beginning of 2016 Gerd is a visiting professor with the Statistics Group of the Medical University of Vienna.
His main professional interests are design and analysis of clinical trials, in particular missing data, subgroups, and quantitative decision making in clinical projects. He is co-editor of Pharmaceutical Statistics.
Alternative Economic Assessment for Expressing Healthcare Value and Informing Resource Allocation Decisions
Level: Introductory
Track: Economic Evaluation
Various stakeholders are involved in the funding, decision-making, policy-making and delivery of effective healthcare. Each of these areas requires different perspectives and constraints that can’t be addressed by applying a single perspective modeling framework such as conventional cost-effectiveness analysis. Increasingly, alternative methodological frameworks are required for informing stakeholders outside of the HTA process regarding healthcare and how to inform decisions considering known constraints including budget. This course will discuss different established economic frameworks for assessing economic value. Firstly, faculty will explore fiscal health modeling (FHM) which reflects the government perspective on population health and investments in medical technologies. Specifically, the course will cover how government can benefit from investments in healthcare based on future changes in tax revenue and reduced transfer costs attributed to changes in health status. The course will also cover constrained optimization modeling which considers that decision-making is often made under specific constraints including budget, logistics, compliance, among other features. Thirdly, faculty will discuss the social accounting matrix (SAM) analytic framework which reflects multiple perspectives simultaneously and is used in developed and developing countries for assessing new budget policies. In the context of health, we apply SAM to understand how money flows through different economic sectors based on investments in healthcare. The course will discuss the strengths and weaknesses of these different frameworks and applications in resource allocation decisions.The course outline will follow the recently completed work of the ISPOR Task Force on Economic Analysis of Vaccination Programs Chaired by Josephine Mauskopf and Baudouin Standaert and published in Value in Health . Participants who wish to gain hands-on experience must bring their personal laptops with Microsoft Excel for Windows installed.
Faculty Member
Mark Connolly, PhD
Global Market Access Solutions, Groningen, GR, Netherlands
Mark Connolly is health economist with over 19 years of experience gained in the pharmaceutical industry, consulting and academia. He has a wide range of pharmaceutical market access, economic modeling and cost-effectiveness experience supporting new product launches and lifecycle management while working in global and regional roles within the pharma industry. His research interests include the economics of healthcare and understanding the broader public economic impact of investments in healthcare applying government perspective frameworks. Dr. Connolly is also the managing director and founder of Global Market Access Solutions with licensed offices in Switzerland, UK and United States. He also holds a guest research position at the University of Groningen Pharmacoeconomics and Pharmacoepidemiology Unit in the Netherlands.
Nikos Kotsopoulos, PhD
Global Market Access Solutions, Geneva, GE, Switzerland
Nikos is a health economist with 16 years of experience in the pharmaceutical industry and consulting. Extensive pharmaceutical reimbursement and health economic experience gained working in Europe and international markets, having held global and regional positions for the pharmaceutical industry and as strategy consultant. Produced diversified health economic analyses for developed and developing countries with over 30 peer-reviewed articles and abstracts published. His research focuses on the development and implementation of novel analytic frameworks that assess the public economic effects of healthcare investments.
Baudouin Standaert, MD, PhD
GSK, Wavre, Belgium
Baudouin “Bo” Standaert was born in Bruges, Belgium. He studied medicine (MD) at the KU Leuven and Tropical Medicine in Antwerp (1982). During the first 7 years of his career Bo had a mixed work assignment in academia (University of Antwerp and UCL in Brussels) and abroad (Suriname, Burundi, Salomon islands, WHO (Geneva)). In 1988 he became director of the Provincial Institute of Hygiene in Antwerp. The Institute had around 160 employees split into lab and field work in collecting basic health and environmental information. During that period Bo was the representative of Belgium in the scientific board of IARC in Lyon, France for 4 years. His interest in economic evaluations of health care emerged with the collaboration with the University of Antwerp in 1990. At that time different pharmaceutical companies were interested in getting HE-expertise in house. Bo took the decision in 1995 to join AMGEN to become their first health economist in Europe. A small team of 8 people was set up in Brussels coordinating all their HE-activities. In 2005, it was time to move to another international group in Belgium and to start working on vaccines. Bo became the head of the global HE-team of vaccines in GSK, Wavre, Belgium. He started with 3 collaborators, first working on Rotarix and Cervarix and later on Synflorix. The group counts now 12 persons. The HE-team has a wide experience in developing different types of economic assessments from simple back-of-the envelope models to more advanced dynamic and macro-economic models for pandemic infectious diseases such as malaria, TB, HIV, Flu. The HE-team explores new economic value evidence (Quality of Care, absenteeism, herd effect) away from the conventional cost-effectiveness analysis including optimisation modelling, return on investment, and fiscal modelling. Bo has numerous publications in the many different fields he worked on. He obtained his PhD on rotavirus vaccination, exploring new ways of economic assessment, at the University of Groningen, the Netherlands, in 2015. He was the industrial representative of the WHO-IVIR-working group for 9 years. He co-chaired the taskforce on economic evaluation of vaccines for ISPOR (2016-2018) and was recently appointed industrial lead for the European IMI-Vital (2018-2022) project on vaccination in elderly.
Oleksandr Topachevsky, MSc, PGDip
Digital Health Outcomes, Kiev, Ukraine
Oleksandr is a health economist with 10 years of experience within pharmaceutical industry, consulting and government positions. He is a founder of Digital Health Outcomes, company providing specialised digital solutions for HEOR, Clinical and Regulatory domains. He is also a member of the National EML Committee, an advisor to the MoH and a World Bank consultant working on the healthcare reform in Ukraine. Following his MSc in financial economics from Kiev Schevchenko University, he also obtained a Health Economics PGDip degree from the University of York, UK.
Introduction to Health Technology Assessment
Level: Introductory
Track: Health Technology Assessment
This introductory course is designed to teach academic researchers, health policy decision makers, manufacturers, and clinicians about the key elements, methods, and language of health technology assessment (HTA). The course provides an overview of basic HTA principles including benefit assessment (biostatistics, clinical epidemiology, patient-relevant outcomes, risk-benefit assessment), economic evaluation (costing, cost-effectiveness analysis, pharmacoeconomic modeling, budget impact analysis, resource allocation), and ELSI (ethical, legal, and social implications). Using real world examples covering both drugs and devices, the course will review the practical steps involved in developing and using HTA reports in different countries and health care systems. Group discussion will focus on the perspectives of different stakeholders and the implementation of HTA in health care decision making. This course is suitable for those with little or no experience with HTA.
Faculty Member
Uwe Siebert, MPH, MSc, ScD, MD
UMIT - University for Health Sciences, Medical Informatics, and Technology, Hall in Tirol, Austria
Prof. Uwe Siebert, MD, MPH, MSc, ScD, professor of Public Health, Medical Decision Making and Health Technology Assessment (HTA), is the chair of the Department of Public Health, Health Services Research and HTA at UMIT - University for Health Sciences, Medical Informatics and Technology in Austria and the Director of the Division for HTA in the ONCOTYROL – Center for Personalized Cancer Medicine in Austria. He is also adjunct professor of Health Policy and Management at the Harvard T.H. Chan School of Public Health and director of the Program on Cardiovascular Research at the Institute for Technology Assessment and Department of Radiology at the Massachusetts General Hospital, Harvard Medical School, Boston. He is the course director of the Harvard summer course on ‘Decision Analysis in Clinical Research’ and he has an adjunct teaching appointment at the School of Public Health and Epidemiology at the University of Munich.
After medical school, he worked for several years as a physician in international public health projects in West-Africa, Brazil, and Germany. He then earned an MPH at the Munich School of Public Health, and completed an MSc in Epidemiology and a ScD in Health Policy and Management with a concentration in decision sciences at the Harvard School of Public Health. Before he started his faculty position at Harvard Medical School, he was the director of the Bavarian Public Health Research and Coordinating Center at the University of Munich, Germany, and completed Visiting Scholarship at the Harvard Center for Risk Analysis.
His research interests include applying evidence-based quantitative and translational methods from public health, epidemiology, comparative effectiveness research, health services and outcomes research, economic evaluation and decision sciences in the framework of HTA as well as in the clinical context of routine health care and patient guidance. His current substantive research focuses on cancer, cardiovascular disease, diabetes, hepatitis C, neurological disorders, and others. His methodological research includes evaluations of public health interventions, prevention/screening, diagnostic imaging procedures, personalized medicine, causal inference from “big” data, decision modelling, and study designs controlling for treatment switching. He has been leading projects/work packages in several EU FP7 and H2020 projects (e.g., ELSA-GEN, BiomarCaRE, MedTechHTA, EUthyroid, FORECEE, MDS-RIGHT, EUREGIO-EFH). He teaches courses in decision-analytic modeling, HTA, economic evaluation, analysis of big data, and causal inference at several universities and for industry in Europe, USA, South America, and Asia.
Prof. Siebert is the past-president of the Society for Medical Decision Making (SMDM), a member of the Latin America Consortium Advisory Committee of ISPOR, member of the ‘National HTA Strategy’ Expert Group of the Austrian Federal Ministry of Health, member of the Oncology Advisory Council of the Federal Ministry of Health in Austria, member of the Advisory Board of the GÖG - National Austrian Public Health Institute, member of the Austrian Cochrane Collaboration Branch, and a member of several national and international Directors Boards (Society for Medical Decision Making; Austrian Society of Epidemiology, German Network of EbM; German Association for Medical Informatics, Biometry and Epidemiology). He has served as vice president of SMDM, and a member of the ISPOR Board of Directors, the Directors Board of the German Competence Network Heart Failure, the International Expert Committee Advising the Institute for Quality and Efficiency in Health Care (IQWiG) on the Methods for Economic Evaluations of Health Care Interventions, the Harvard Flagship Initiative in Comparative Effectiveness Research, the Extended Board of Directors of the German Association of Health Economics, and the Advisory Board of the Ludwig Boltzmann Institute for HTA in Austria.
He is the Clinical Guideline Commissioner for the Association of the Scientific Medical Societies in Germany (AWMF) and the chairman of the Working Groups ‘Health Economics’ and ‘Medical Decision Making’ of the German Society for Medical Informatics, Biometry and Epidemiology (GMDS). He is co-chair of the ISPOR-SMDM Modeling Good Research Practices Task Force, co-chair of the ‘Issues in Methodology Section’ of the SMDM Policy Initiative, and a leadership member of the ISPOR Personalized/Precision Medicine Special Interest Group.
He has worked with several HTA Agencies (eg, DAHTA@DIMDI/Germany, IQWiG/Germany, NICE/UK, ANVISA/Brazil, CADTH/Canada, LBI-HTA/Austria, GÖG/Austria) and he advises government agencies, academic institutions and industry regarding the conduction and impact of health technology assessments on policy and reimbursement decisions. He has authored more than 350 publications including HTA reports, textbook chapters, scientific articles, and editorials, and is Editor of the European Journal of Epidemiology, as well as editorial board member of several scientific journals.
Pharmacoeconomic Modeling - Applications
Level: Intermediate
Track: Methodological & Statistical Research
During this course, students will have hands-on experience in constructing and analyzing a decision analysis tree – including Markov models and one-way, two-way, and probabilistic sensitivity analysis – using TreeAge Pro software. Instructors will provide a series of short lecture-based sessions followed by the opportunity for participants to engage in model-building exercises using the software. Sessions will demonstrate how to build a simple decision tree, extend a decision model to incorporate costs and utilities, and replace terminal nodes with state-transition (Markov) models to represent time-varying events. Other more advanced topics will be covered if time permits. Participants are required to bring their personal laptops equipped with software provided to course registrants.
Faculty Member
Shelby Corman, PharmD, MS
Pharmerit International, Bethesda, MD, USA
Shelby is a Director of Health Economics and Outcomes Research (HEOR) with Pharmerit International, a global HEOR consultancy based in Bethesda, MD. She received her PharmD degree from the University of Pittsburgh School of Pharmacy in 2002, after which she completed a specialty residency in Drug Information at the University of Pittsburgh Drug Information Center in conjunction with the University of Pittsburgh Medical Center (UPMC). After completing this training, Shelby joined the faculty at the University of Pittsburgh and worked in formulary management and drug policy at UPMC, while conducting outcomes research in a variety of therapeutic areas. In 2011, Shelby completed a Master of Science degree in Clinical Research (Health Services Research Track) at the University of Pittsburgh School of Medicine, with thesis work focusing on the cost-effectiveness of new medications in comparison to older, standard-of-care therapies. In 2012, Shelby joined Pharmerit International as a scientist specializing in economic modeling, real-world evidence, and strategic market access. Since joining Pharmerit, Shelby has conducted numerous health economic evaluations, medical chart review studies, claims database analyses, and reimbursement dossiers as a consultant to the pharmaceutical, biotechnology, and medical device industries. Her therapeutic areas of expertise include postsurgical pain, infectious diseases, oncology, and solid organ transplant.
Mark S. Roberts, MD, MPP
University of Pittsburgh Graduate School of Public Health, Pittsburgh, PA, USA
Mark S. Roberts, MD, MPP is Professor and Chair of the Department of Health Policy and Management at the University of Pittsburgh Graduate School of Public Health, and directs the Public Health Dynamics Laboratory, a modeling and simulation group at the University. He also holds appointments as professor of Medicine, professor of Industrial Engineering and professor of Clinical and Translational Science. He obtained a bachelor’s degree in Economics from Harvard College, a Doctor of Medicine from Tufts University, and a Master’s in Public Policy from the Kennedy School. He completed training in internal medicine at the Harvard Medical School, where he was a resident and fellow. Over the past 30 years he has conducted several NIH-funded research programs in the use of mathematics, simulation and decision sciences to improve health care decisions and the delivery of care. He was the founding chief of the Section of Decision Sciences and Clinical Systems Modeling, a research section in the department of Medicine. He has published over 170 papers in academic journals, and been funded on over 40 federally funded grants. He was the 2014 Recipient of the Society for Medical Decision Making Lifetime Achievement Award for contributions in decision sciences. Educationally, he has developed and taught courses in decision analysis and cost effectiveness analysis for several universities, professional societies, pharmaceutical companies and other organizations, both domestically and internationally. He has been the Chair of the education committee for the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), and is a member of several other national societies, including the recent President of the society for Medical Decision Making. For the federal government, he has been a member of several NIH research grant review panels, and for two years was chair of the Health Care Technology Assessment and Decision Science review panel.
Analysis of Longitudinal Data- Fixed and Random Effects Models
Level: Intermediate
Track: Methodological & Statistical Research
Longitudinal data is often encountered by researchers, allowing them to use the time dimension to uncover deeper insights than what is possible with a traditional cross-sectional snapshot. But this advantage comes at a cost: the assumption that each observation is independent is broken due to the fact that patients are measured on multiple occasions over time. Failure to account for this feature when analyzing data can result in bias, and longitudinal methods should be used to account for this problem. Two powerful but simple solutions are the fixed and random effects estimators, both of which have recently become more popular in medical research. A key feature of both is that they model the unobserved differences between patients, and can even control for unobserved confounding. This course will discuss the methods and intuition behind both modelling techniques, alongside practical examples and interactive sessions in STATA. Attendees will gain both knowledge and practical skills in this course. Although not essential, those who have STATA loaded on their laptops are encouraged to bring your laptop in order to participate in interactive sessions.
Faculty Member
Kirk Geale, MSc
Quantify Research AB, Stockholm, Sweden
Alexander Rieem Dun, MSc
Quantify Research, Stockholm, Sweden
Alexander Rieem Dun is a Research Analyst at Quantify Research. He holds a Bachelor of Arts in Chinese and an MSc in Economics from Uppsala University, Sweden. Prior to joining Quantify Research, he worked as a research assistant at Uppsala University at the Department of economics. Since joining Quantify Research, he has worked with real-world evidence studies across a variety of therapies including dermatology and cardiovascular disease. His interest and expertise lies in study design, observational data analysis, analytic methods, and Swedish register data.
New! Digital Real-World Evidence Generation Approaches in Rare Diseases and Oncology
Level: Introductory
Track: Real World Data & Information Systems
This course is designed to provide a good understanding of how to plan and undertake real-world evidence (RWE) studies to collect patient-level data through a digital app and across different countries. During this course, the benefits and challenges of developing digital BYOD apps to collect PRO data will be discussed and analyzed. The focus of the course will be on rare diseases and oncology, and specific case studies will be presented. The methods of analysis of such datasets, as well as their value and use to support HEOR/HTA strategies will also be discussed. The course is open to all -no specific prior experience is required, apart from a basic level of understanding of RWE.
Faculty Member
Mark Larkin, PhD
Vitaccess Ltd, Oxford, 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.
Annabel Nixon, BSc PhD
Chilli Consultancy Ltd, Salisbury, WIL, United Kingdom
Annabel is an expert in patient centered outcomes, with 20 years’ international experience consulting in the pharmaceutical industry in Europe and the USA; specializing in FDA, EMA, and HTA requirements for PROs and other clinical outcome assessments. Annabel was co-chair of the Drug Information Agency (DIA) Study Endpoints community (2012–2016). She has published many research papers and is the lead author of Patient Reported Outcomes: an overview (eBook, paperback). Annabel has provided key PRO support in registry-based studies, including selection of PRO instruments and implementation and analysis strategies.
Jon Spinage, BSc, CITP
Vitaccess Ltd, Oxford, United Kingdom
Jon is director of Technology at Vitaccess. Jon has over 20 years of experience in software development for desktop, web, and mobile, in a variety of industries, including health economics, banking, life insurance, and pensions. He has managed software development projects for a variety of market access applications. Jon is a Chartered IT Professional and a Registered Practitioner in PRINCE2 project management.
Sun 3 Nov
8:00 - 12:00
SHORT COURSE MORNING SESSION
New! Market Access & Value Assessment of Medical Devices
Level: Intermediate
Track: Health Policy & Regulatory
This course is designed for those with an intermediate knowledge of medical devices and their market access pathways. The focus will be on understanding the areas of United States (US) and European Union (EU) healthcare systems relevant to medical devices (eg, diagnosis-related groups (DRG’s), inpatient versus outpatient), implicit value drivers of medical devices, the stakeholder organizations necessary to engage in order to obtain medical device funding/reimbursement and adoption, and the healthcare system pathways through which medical devices can be implemented. The course will conclude with a primer on performance-based risk-sharing agreements. Market access for medical devices is an evolving, multi-faceted, and multi-stakeholder journey that requires dedicated knowledge. Experience in launching pharmaceutical products can present both challenges and opportunities for medical device companies, as the pathways, evidence requirements, and value domains are materially different. This course will attempt to demystify the medical device landscape and help all stakeholders ensure that appropriate patients benefit from innovation in the medical device space through improving participants’ awareness of marketplace trends and needs to demonstrate clinical and economic value.
Faculty Member
Rachele Busca, PharmD, MSc, MBA
W.L GORE, Verona, Italy
Graduated in Chemistry & Pharmaceutical Technology at Bologna University and completed education in Health Economics & Outcomes Research with a Master at the University of Milan and an MBA at the Bologna Business School.
Market Access & Health Economics & Outcomes Research expert / Director level professional with over 10 years of experience in Medical Device, including Health Economics & Outcomes Research, Reimbursement & Access and Value strategies, delivering data and tools to effect reimbursement, funding and Health Technology Assessments at International level.
Currently EMEA Leader Health Economics at W.L GORE – a Global Devices manufacturer known for innovation and a modern, distinctive company culture.
Previously served for over 7 years the Italian Pharmaceutical business in Bayer, Recordati and consultancy group Pbe. Most recently as Director Pricing and Reimbursement Strategy at BTG and Principal Manager at Medtronic assisting several divisions: interventional pulmonology, cardiology, cardiac disease management, structural heart, peripheral products.
Specialties: market access strategies, pricing, HEOR, incorporation of market access requirements into clinical, medical & commercial strategies, new market assessment, due diligence.
Richard Charter, MSc
ValueConnected, 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.
Michael Drummond, MCom, DPhil
University of York, York, 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.
Stephen Hull, MHS
Hull Associates LLC, Rockland, MA, USA
Stephen Hull is President and Founder of Hull Associates LLC, a specialized global reimbursement strategy firm focused on medical device, diagnostic, pharmaceutical and biotech technologies.
Founded in 2007, Hull Associates has market access and reimbursement experts in all major product areas, and is dedicated to helping clients achieve market access and reimbursement success.
With 40+ seasoned partners worldwide, Hull Associates LLC develops and executes strategies for reimbursement and product launches in the US and major global markets, including the Americas; Asia Pacific; Northern & Eastern Europe; The Middle East; and Western Europe.
Stephen has an advanced degree in health policy from the Johns Hopkins Bloomberg School of Public Health, and a bachelor’s degree in international relations and French from Colgate University.
Risk-Sharing/Performance-Based Arrangements for Drugs and Other Medical Products
Level: Intermediate
Track: Health Policy & Regulatory
There is significant and growing interest among both the payers and producers of medical products for arrangements that involve a “pay-for-performance” or “risk-sharing” element. These payment schemes involve a plan by which the performance of the product is tracked in a defined patient population over a specified period of time and the level of reimbursement is tied by formula to the outcomes achieved. Although these agreements have an intrinsic appeal, there can be substantial barriers to their implementation. Issues surrounding theory and practice, including incentives and barriers, will be analyzed along with several examples of performance-based schemes from Europe, the United States, and Australia. A hypothetical case study will be used in an interactive session to illustrate a systematic approach to weighing their applicability and feasibility. This course is designed for those with some experience with health economic analysis.
Faculty Member
Josh Carlson, MPH, PhD
University of Washington, Seattle, WA, USA
Louis P. 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.”
Adrian Towse, MA, MPhil
Office of Health Economics, London, United Kingdom
Professor Adrian Towse is director emeritus and senior research fellow of the Office of Health Economics in the UK. Adrian’s current research includes incentives for new drugs and vaccines to tackle Antimicrobial Resistance, the use of 'risk-sharing' arrangements between healthcare payers and pharmaceutical companies, including value-based pricing approaches; the economics of pharmacogenetics for healthcare payers and the pharmaceutical industry; economic issues that affect both R&D for and access to treatments for diseases prevalent in the developing world; the economics of medical negligence; and measuring productivity in healthcare.
A visiting professor at the London School of Economics and a senior researcher at the Nuffield Department of Population Health at the University of Oxford, Adrian also has been a visiting professor at the University of York. For ten years, he served as the non-executive director of the Oxford Radcliffe Hospitals NHS Trust, one of the UK’s largest hospitals. Adrian was president of ISPOR, for the 2014-15 term.
Adrian joined the OHE in 1993 and served as director for 25 years. He holds an MA (Hons) in Politics, Philosophy and Economics from Keble College, Oxford; an MPhil in Management Studies from Nuffield College, Oxford, and the Oxford Centre for Management Studies; and is a member of the Chartered Institute of Management Accountants.
Using Multi-Criteria Decision Analysis in Healthcare Decision Making- Approaches & Applications
Level: Introductory
Track: Health Technology Assessment
Many health care decisions – such as portfolio optimization, benefit-risk assessment (BRA), health technology assessment (HTA), and shared decision making (SDM) – require a careful assessment of the underlying options and the criteria used to judge these options. This assessment can be challenging given the trade-offs between multiple value criteria. In light of this, many decision makers have begun investigating the use of multi-criteria decision analysis (MCDA) in support of these decisions. This course provides an introduction to MCDA for health care. The course will focus on the use of MCDA for HTA, and will be organised around the following parts: 1) Introduction to MCDA: What is it and how is it being use in HTA?; 2) Implementing MCDA 1: Practical tips when implementing MCDA; 3) Implementing MCDA 2: Methodological options when designing an MCDA; and 4) Using MCDA for HTA. Challenges and possible solutions. These parts are designed to familiarise participants with the steps involved in undertaking an MCDA, the alternative ways of implementing these steps, and good practice guidelines. The course will also review the current MCDA HTA landscape, including current use of MCDA for HTA and the challenges this poses. The course is designed for those unfamiliar with MCDA, but who have a basic understanding of other evaluation methodologies. The course is designed for those unfamiliar with MCDA, but who have a basic understanding of other evaluation methodologies.
Faculty Member
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.
Maarten J. IJzerman, PhD
Office of Health Economics, Melbourne, Australia
Maarten J. IJzerman is a VCCC professor and head of Cancer Health Services Research in the Faculty of Medicine, Dentristry and Health Sciences of the University of Melbourne in Australia. He also holds a fractional appointment as a professor of Health Technology Assessment in the University of Twente, where he was the Vice-Dean in the Faculty of Science. Maarten is an active global researcher with multiple research visits and collaborations in the USA, UK and Canada. He is an active ISPOR member, serves in different ISPOR taskforces and committees, and promotes active collaboration between ISPOR and the European Cancer Organisation (ECCO). In 2016, he joined the European Society for Medical Oncology (ESMO) Cancer Medicines working group dealing with the disparities in access to cancer drugs. Maarten is a member of the board of two hospitals in the Netherlands, responsible for quality and safety and strategy/innovation.
Kevin Marsh, PhD
Evidera Ltd, 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.
Budget Impact Analysis I – a 6-Step Approach
Level: Intermediate
Track: Economic Evaluation
This course will describe the methods used to estimate the budget impact of a new health care technology, and will present six basic steps for estimating budget impact: 1) estimating the target population; 2) selecting a time horizon; 3) identifying current and projected treatment mix; 4) estimating current and future drug costs; 5) estimating change in disease-related costs; and 6) estimating and presenting changes in annual budget impact and health outcomes. Both static and dynamic methods for estimating the budget and health impact of adding a new drug to a health plan formulary will be presented. These six steps will be illustrated using actual budget impact models. This course is designed for those with some experience with pharmacoeconomic analysis.
Faculty Member
Thor-Henrik Brodtkorb, PhD
RTI-HS Nordics, Ljungskile, Sweden
Thor-Henrik Brodtkorb, PhD, is senior director in Health Economics at RTI Health Solutions (RTI-HS). He holds a PhD in Health Technology Assessment from the University of Linköping and has been with RTI-HS for 7 years. He has been teaching courses in decision-analytic modeling at Linköping University as well as presented workshops and short courses on decision-analytic modeling techniques for organizations such as Pharma Industry Sweden, Swedish Agency for Health Technology Assessment and Assessment of Social Services (SBU), and the International Society for Pharmacoeconomics and Outcomes Research (ISPOR).
At RTI-HS, Dr. Brodtkorb leads development of cost-effectiveness, cost-utility, cost-consequence, and budget-impact models for pharmaceutical, device, and diagnostic technologies. These models have been used to support reimbursement decisions in more than 15 European countries including NICE in UK, SMC in Scotland, TLV in Sweden, and NOMA in Norway. He has developed models and analyses in the areas of oncology, alcohol dependence, major depressive disorder, Alzheimer’s disease, dermatology, multiple sclerosis, cardiology, orthopedics, and asthma. His research has been presented at professional conferences and published in peer-reviewed journals. He is also a coauthor of the newly published book Budget-Impact Analysis of Health Care Interventions: A Practical Guide.
Stephanie R. Earnshaw, PhD, MS
RTI Health Solutions, Research Triangle Park, NC, USA
Stephanie Earnshaw is Senior Vice President of Health Economics at RTI Health Solutions (RTI‑HS). She received her PhD in Industrial Engineering at North Carolina State University and has been with RTI-HS for over 19 years. She has presented workshops, distance learning, and short courses on decision-analytic modeling techniques for pharmaceutical companies and organizations such as the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), the Academy of Managed Care Pharmacy (AMCP), and the Centers for Disease Control and Prevention (CDC). Dr. Earnshaw currently serves on the ISPOR Board of Directors, and she has held an Adjunct Faculty appointment at the University of North Carolina’s Eshelman School of Pharmacy, Division of Pharmaceutical Outcomes and Policy. She is also one of the lead authors of the newly published book Budget-Impact Analysis of Health Care Interventions: A Practical Guide. Dr. Earnshaw’s research focus is in applying decision-analysis techniques to industry-related issues and health care problems. Her areas of specialization include mathematical programming (constrained optimization), network optimization, and Markov, simulation, and other state transition modeling. She has developed innovative mathematical models using these methods to determine pricing strategy, predict clinical outcomes, and allocate resources. In addition, she continues her support for the pharmaceutical, biotechnology, and diagnostic and medical device industry by developing budget-impact and cost-effectiveness models for their health technologies. Therapeutic areas include cardiovascular disease, gastrointestinal disorders, respiratory disease, transplantation, infectious disease, osteoporosis, vaccines, and oncology. She is a member of the International Society for Pharmacoeconomics and Outcomes Research and the Institute for Operations Research and the Management Sciences. She has presented her work at professional conferences and has published in several peer-reviewed journals.
C. Daniel Mullins, PhD
University of Maryland, School of Pharmacy, Baltimore, MD, USA
C. Daniel Mullins, PhD is a professor and chair of the Pharmaceutical Health Services Research Department at the University of Maryland School of Pharmacy. He is founder and executive director of the University of Maryland PATient-centered Involvement in Evaluating effectiveNess of TreatmentS (PATIENTS) Program, an infrastructure to support patient-centered outcomes research and related training activities. He also serves as director of the Community and Collaboration Core within the university’s Institute for Clinical and Translational Research. Dr. Mullins has received funding as a principal investigator from the NIH/NIA, NIH/NHLBI, AHRQ, the Patient-Centered Outcomes Research Institute (PCORI), and various patient advocacy and industry organizations. He has served as a regular member of AHRQ and NCI Study Sections and has chaired PCORI Study Sections. Professor Mullins is one of two editors-in-chief for Value in Health and is author/co-author of more than 225 peer-reviewed articles. He has received an Outstanding Service Award from the Drug Information Association (DIA) and two Service Awards from ISPOR. In 2007, he received the Dr. Patricia Sokolove Outstanding Mentor Award from the University of Maryland Baltimore campus-wide Graduate Student Association. In 2013, he was the recipient of the Dr. Daniel D. Savage Memorial Science Award, the Association of Black Cardiologists’ most prestigious annual award. Also in 2013, he was awarded a University System of Maryland Wilson H. Elkins Professorship. He was named Researcher of the Year in 2014 and received the Martin Luther King Faculty Diversity Award in 2017 for the University of Maryland Baltimore campus and the ISPOR Marilyn Dix Smith Leadership Award.
Cost-Effectiveness Analysis Alongside Clinical Trials
Level: Intermediate
Track: Economic Evaluation
The growing number of prospective clinical/economic trials reflects both widespread interest in economic information for new technologies and the regulatory and reimbursement requirements of many countries that now consider evidence of economic value along with clinical efficacy. This course will present the design, conduct, and reporting of cost-effectiveness analyses alongside clinical trials based on, in part, "Good Research Practices for Cost-Effectiveness Analysis alongside Clinical Trials: The ISPOR RCT-CEA Task Force Reports". Trial design, selecting data elements, database design and management, analysis, and reporting of results will all be presented. Trials designed to evaluate effectiveness (rather than efficacy), as well as clinical outcome measures, will also be discussed, including how to obtain health resource use and health state utilities directly from study subjects and economic data collection fully integrated into the study. Analyses guided by an analysis plan and hypotheses, an incremental analysis using an intention to treat approach, characterization of uncertainty, and standards for reporting results will be presented. Familiarity with economic evaluations will be helpful.
Faculty Member
Federico Augustovski, MSc, PhD
University of Buenos Aires, Buenos Aires, B, 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.
Shelby D Reed, PhD, RPh
Duke University, 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.
Mapping to Estimate Utility Values from Non-Preference-Based Outcome Measures Part 1
Level: Intermediate
Track: Patient-Centered Research
Mapping is the term used to refer to studies which estimate health state utility values from some non-preference based outcome measures. It is a practice commonly undertaken in HTA, most typically when clinical trials have not included any preference based instrument which would permit the estimation of QALYs in a cost-utility analysis. Mapping uses a different dataset to bridge this evidence gap. This is an introductory level course that will provide instruction to participants on key issues faced either when conducting, interpreting or using the results of a mapping study. It will draw on the ISPOR Good Practice Guide on Mapping that all faculty were members of. The course will introduce the concept of mapping and highlight the types of areas where it has been used. Using real world examples we will provide an overview of the main considerations for mapping including, how to select an appropriate dataset for mapping, key aspects for undertaking the statistical analysis and producing the optimal mapping model, how to report, interpret and use results from mapping in real world cost-effectiveness studies. A mixture of formal presentations, group discussions and illustrated examples will be used with an emphasis on interactive elements between the faculty and participants.
Faculty Member
Monica Hernandez, MSc, PhD
University of Sheffield, Sheffield, NYK, United Kingdom
Monica Hernandez, BSc, MSc, PhD, is an econometrician and health economist with extensive experience in developing complex statistical and econometric models. She has worked extensively on measurement error and latent variable modelling, and the analysis of observational data. On the latter area of research, she has been an author of two technical Support Documents providing guidance and future research recommendations on the use of observational data for submissions to the National Institute for Health and Care Excellence (NICE).
Recently, her research has focused on investigating the properties and policy use of EQ-5D, leading to the development of new methods for analyzing and mapping preference based measures. Monica has developed Stata user-written commands to allow practitioners to use those newly developed methods.
She was a member of the ISPOR Good Practice Guideline Task Force on Health Utilities Mapping on which this short course is based.
Andrea Manca, MSc, PhD
University of York, Heslington, York, United Kingdom
Andrea Manca is an applied health economist with more than 20 years’ experience in the area of economic evaluation for healthcare decision-making. He has evaluated a wide range of technologies (therapeutic medical devices, drugs, diagnostics and non-pharmaceutical interventions) in a number of different clinical areas, including oncology, cardiology and respiratory diseases, diabetes, chronic pain, surgical interventions, obstetrics, and gynecology.
His research interests focus on developing methodological solutions to address the issues that arise when analyzing and interpreting health economics and outcomes research data to inform person-centered healthcare decision-making. This involves the use of advanced statistical and decision modelling techniques for the analysis of cost effectiveness and outcomes data, including quantitative evidence synthesis and health econometrics methods.
Andrea received the ISPOR Award for Excellence in Methodology in Pharmacoeconomics and Health Outcomes Research (2008), and a number of other national scholarships and awards from the BackCare Society (2004), the Wellcome Trust (2007), and the UK National Institute of Health Research (2009).
He is a former co-editor (currently serving on the EAB) of the scientific journal Value in Health and member of the ISPOR Bernie O'Brien New Investigator Award panel. Andrea is deputy chair of the UK NIHR Doctoral Research Fellowship scheme and member of the Technology Appraisal Committee of the National Institute for Health and Care Excellence (NICE) for England and Wales.
Andrea holds a MSc in Health Economics (1998) and a PhD in Economics (2005), both by the University of York, UK.
His full CV can be found here: https://www.york.ac.uk/che/staff/research/andrea-manca/
Allan Wailoo, MA, MSc, PhD
University of Sheffield, Sheffield, United Kingdom
Allan Wailoo, PhD, is a professor of Health Economics in the School of Health and Related Research (ScHARR), University of Sheffield, UK where he has worked since 2000. He directs the National Institute for Health and Care Excellence’s Decision Support Unit and is co-director (with Mark Sculpher) of the UK Department of Health Policy Research Unit for Economic Evaluation.
He is a member of NICE’s Medical Technologies Appraisal Committee and co-editor for the ISPOR journal Value in Health and recently chaired the ISPOR Task Force on Good Practices in Mapping on which this short course is based. He has published numerous papers on mapping methods and has wider research interests in all areas of economic evaluation and related methods.
Transferability and Relevance of Cost-Effectiveness Data between Countries
Level: Experienced
Track: Economic Evaluation
Although the number of countries requiring an economic dossier as part of the submission dossier for public reimbursement of new drugs is growing, the pharmaceutical industry cannot conduct economic evaluations in every potential market. However, national decision makers require country-specific or region-specific data or relevant estimates on health care costs and patient outcomes. More and more, they are only willing to accept foreign or international data when transferable to their own decision-making context. However, little guidance exists on how to do this. This course starts with a discussion of factors that make economic data more difficult to transfer from one country to another than clinical data, and will focus on the report of the ISPOR Good Practices on Economic Data Transferability Task Force. In this respect, faculty will discuss the transferability of health state valuations based on the EQ-5D instrument and the transferability of lost productivity data. Next, faculty will review the methods that have been presented to assess the transferability of foreign cost, effects, and cost-effectiveness estimates and their pros and cons. This topic will be practically covered in a case-study while working in small groups. A stepwise procedure will illustrate how to select a foreign cost-effectiveness model for adaptation to your own decision-making context. Finally, a detailed approach on how to adapt a cost-effectiveness model calculation will be illustrated using the case of breast cancer treatment. During the course, faculty will present transferring issues encountered when assessing model-based economic evaluations.
This course is for those with advanced understanding of economic evaluations of health care programs and experience in the critical assessment of cost-effectiveness studies. Please note : The statistical methods used to analyze multinational trial data and to transfer these data to a specific country are beyond the scope of this course.
Faculty Member
Silvia Evers, PhD, LLM
Maastricht University, Maastricht, Netherlands
Silvia Evers studied Health Sciences (Mental Health Sciences; Health Policy & Administration), Epidemiology, and Law (Labour and Social; Health Law). Since the early nineties, she has been working as a researcher in the field of Economic Evaluations/Health Technology Assessment at the Institute for Rehabilitation Research, the Maastricht University (Epidemiology; Health Economics; Medical Sociology), the National Institute for Public Health and the Environment (Centre for Care Studies), the University of Amsterdam (Pedagogics), and the University Hospital Maastricht (Neurosurgery). Next to research she has been working as a legal (Ethical Commission Psychology) and policy advisor at the faculty of Psychology of Maastricht University, where she headed the Research Institute of the Faculty Board.
Currently she holds a chair on Public Health Technology Assessment at the Maastricht University, Department of Health Services Research. Next to that, she has worked at the Trimbos Institute, the Centre of expertise on mental health and addiction in the field of HTA research since 2013. At the Maastricht University, she is program leader of the program ‘Creating value-based healthcare’ and profile coordinator of the HTA-trace of the Health Sciences Research Master.
She is involved as an HTA-project leader, promotor and supervisor in numerous (clinical) trials funded both nationally (ZonMw, NWO, industry, etc), and internationally (EU, OECD). She is a coordinator and a senior lecturer in HTA and Health Economic courses. Her chief current research efforts are directed towards the methodology of economic evaluation of public health interventions, meta-analysis, and quality of life analysis. She has a special interest in the application of these methods looking at innovative interventions in the field of brain related and mental diseases, public health, youth, and rehabilitation. She is a member of several national and international working groups, editor for several journals, and a referee for various research programs.
Manuela Joore, PhD
Maastricht University Medical Centre+, Maastricht, Netherlands
Manuela A. Joore graduated from Health Policy, Economics and Management (Maastricht University) in 1996. Following her graduation she has worked as a researcher in the field of Health Technology Assessment at the Maastricht University Medical Center and Maastricht University. She got her PhD in Economics in 2002 (Maastricht University) on the topic of health economic evaluation. Since 2006 Manuela has been teaching methods of Health Technology Assessment at Maastricht University and at other organizations. She is the developer and coordinator of the 'Cost-effectiveness modelling methods' course of the Health Sciences Research Master. She is a member of the 'Scientific Advisory Committee' of the National Health Care Institute. She is also a member of the 'Rational Pharmacotherapy' committees of the Netherlands Organisation for Health Research and Development (ZonMw). She is on the HTA board of the Dutch Centre for Personalised Cancer Treatment. Manuela leads a health economics evidence review group for the National Institute for Health and Care Excellence in the United Kingdom.
She holds a chair on 'Health Technology Assessment and decision-making' at Maastricht University. Her research focus is on (mostly model-based) health economic evaluation in a large variety of clinical areas. She uses economic evaluation to inform decision making regarding the (further) development, reimbursement, and use of healthcare technologies. She is (co-)author of over 120 peer-reviewed papers and is/was supervisor of 16 PhD students. She is involved in numerous Health Technology Assessment studies as an HTA-project leader and promotor. The total number of national and international grants obtained as a (co-)applicant adds up to more than 30 with a total sum of more than 13 million Euros.
New! Probabilistic Graphical Models with Openmarkov, an Open-Source Tool
Level: Intermediate
Track: Methodological & Statistical Research
This course will introduce probabilistic graphical models (PGMs), such as Bayesian networks, influence diagrams and decision analysis networks, and discuss their advantages over traditional techniques; for example, influence diagrams and decision analysis networks are equivalent to decision trees containing thousands of branches, Markov influence diagrams can model state-transition problems without multiplying the number of states and decision analysis networks can evaluate large models with unordered decisions. OpenMarkov, an open-source tool, allows to build PGMs for complex problems using a graphical user interface, without writing any code, such as spreadsheet formulas, macros or functions. For beginners, the course will be an introduction to medical diagnosis and decision making. For participants who have already conducted cost-effectiveness analyses it will be interesting to see that building and evaluating a PGM is easier, faster and less error-prone than building and debugging an equivalent model using a spreadsheet, a (Markov) decision tree or a programming language, such as R, MATLAB or C++. Participants are invited to bring their own laptops with OpenMarkov installed.
Faculty Member
Francisco Javier Diez, PhD
Universidad Nacional de Eduación a Distancia (UNED), Madrid, Spain
F. J. Díez is full professor of artificial intelligence at UNED, the largest Spanish university. He built DIAVAL, one of the first Bayesian networks for medicine. He has been principal investigator in several national and international projects and published his work in some the most relevant journals of AI, including Artificial Intelligence and Artificial Intelligence in Medicine, and health decision analysis, such as Medical Decision Making and Pharmacoeconomics. He is the leader of a research group that built OpenMarkov, an open-source tool for PGMs, especially tailored to medicine, which has been used in more than 30 countries. He has been teaching probabilistic graphical models to computer science students for 25 years. He is the director of a modular program that has taught around 2,000 health professionals since 1996.
Understanding Survival Modeling with Application to HTA
Level: Intermediate
Track: Methodological & Statistical Research
Time-to-event (survival) analysis is an important element in many economic analyses of health care technologies. This is particularly true in oncology given the requirement to estimate lifetime costs and outcomes (i.e. extrapolate) beyond the follow-up typically observed in clinical trials. Cost-effectiveness estimates can be sensitive to the methods applied in modelling survival data. Recommendations for selecting a parametric survival model have been recently been published; following a review of extrapolation modelling in National Institute for Health and Care Excellence (NICE) technology appraisals. The purpose of this course is to provide participants with an understanding of the fundamentals of survival analysis and key issues to be considered when comparing alternative survival models for inclusion in cost-effectiveness analysis. This will include an understanding of differences between partitioned survival and Markov-based approaches.
Faculty Member
Andrew Briggs, DPhil
University of Glasgow, Glasgow, 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.
Andrew Davies, MSc
University of Glasgow, Glasgow, United Kingdom
Andrew Davies is senior health economist at the University of Glasgow. He was until recently principal health economist at ICON Health Economics and has over 15 years’ experience of cost-effectiveness modeling in health. He has lead modeling projects in a wide range of clinical areas, including cardiovascular disease, oncology, neurology, and psychiatry.
In recent years he has been closely involved with the UK Risk Share Scheme for Multiple Sclerosis Disease Modifying Therapies. He regularly conducts projects for the development of global cost-effectiveness models with a focus on submissions to bodies such as NICE. These frequently involve analysis of survival data, developing suites of risk equations based on randomized and observational data to support extrapolation and examination of heterogeneity in cost-effectiveness models.
James Lewsey, PhD
University of Glasgow, Glasgow, United Kingdom
Jim Lewsey, PhD, is a reader in Medical Statistics and joined the University of Glasgow in 2007 having previously held posts at the London School of Hygiene and Tropical Medicine (2003-2007), University of Otago (2001-2003), Eastman Dental Institute-UCL (1998-2001), and University of Glasgow (1996-1998). He was awarded Chartered Statistician status from the Royal Statistical Society in 2010 and Chartered Scientist from the Science Council in 2012.
His personal research interests stem from methodological challenges faced when analyzing observational and experimental medical data and have included prognostic model development in the presence of missing data, continuous outcome monitoring of long-term outcomes, modelling dental caries data, and design of cluster randomized trials. His current methodological interests include multi-state survival analysis and modelling of data from natural experiments, and in his applied research he is developing a portfolio of alcohol research. Jim leads the Analysis of Linked Health Data (ALDA) program within HEHTA and is deputy lead of the IHW research theme 'Data Science - Using routine administrative data and record linkage for research'.
Jim is program director and teaches on the MSc in Health Technology Assessment. He also teaches and coordinates a medical statistics course on the Masters in Public Health.
8:00 - 17:00
FULL DAY SHORT COURSES
Bayesian Analysis - Overview and Applications
Level: Intermediate
Track: Methodological & Statistical Research
The first portion of this course is designed to provide an overview of the Bayesian approach and its applications to health economics/pharmacoeconomics and outcomes research. The course will cover basic elements of Bayesian statistics, contrasting briefly with classical (frequentist) statistics, and introduce available statistical packages. The second part of the course is a hands-on workshop where participants will be led through a series of exercises using the free Markov Chain Monte Carlo package WinBUGS. Attendees will have the chance to apply the principles they have learned in the morning session to challenging data analysis problems, including the use of Bayesian generalized linear models (GLM) to analyze cost and outcomes data. This course is designed for those with a limited understanding of Bayesian statistical concepts or for those who want a refresher and more practical experience. Participants are encouraged to bring their personal laptops equipped with software provided to course registrants.
Faculty Member
Keith R. Abrams, PhD, MSc, CStat
University of Leicester, Leicester, United Kingdom
Keith Abrams, PhD, is a professor of Medical Statistics in the Department of Health Sciences at the University of Leicester, having previously held appointments at the London School of Hygiene and Tropical Medicine and King’s College School of Medicine and Dentistry, London, UK. He is a Fellow of the Royal Statistical Society, and a Chartered Statistician.
His research interests are primarily concerned with the development and application of Bayesian statistical methods in Health Technology Assessment (HTA), in particular their application to clinical trials, evidence synthesis, and decision modelling, and Non-Communicable Disease (NCD) epidemiology. This work is primarily supported with funding from EU, Medical Research Council (MRC), National Institute for Health Research (NIHR) and industry.
Professor Abrams has been extensively involved with the UK NIHR Health HTA Programme and UK National Institute for Health & Care Excellence (NICE) appraisal process since their inception. He was a member of the NICE Technology Appraisals Committee for over 8 years until 2015, is a member of the NICE Decision Support Unit and Technical Support Unit, and a NIHR Senior Investigator Emeritus. He has published widely in both substantive and methodological areas including co-Authoring books on Methods for Meta-Analysis in Medical Research, Bayesian Approaches to Clinical Trials and Healthcare Evaluation, and Evidence Synthesis for Decision Making in Healthcare, in addition to co-Editing one of the first texts on Methods for Evidence-based Healthcare.
Christopher S. Hollenbeak, PhD
The Pennsylvania State University, Hershey, PA, USA
Christopher S. Hollenbeak, PhD is Professor and Head of the Department of Health Policy and Administration at the Pennsylvania State University. Dr. Hollenbeak has broad interests in health economics and health outcomes research, with a particular focus on economic issues in surgery. In previous research he has studied modifications to the kidney allocation mechanism, estimated the cost-effectiveness of screening for head and neck cancer, and estimated the burden of surgical site infections after major abdominal and cardiothoracic surgical procedures. Dr. Hollenbeak received his doctorate in Economics from Washington University in St. Louis, where he studied Bayesian econometrics under Ed Greenberg and Siddhartha Chib. He uses Bayesian methods in meta-analysis and to fit nonlinear risk adjustment models for continuous hospital performance measures.
13:00 - 17:00
SHORT COURSE AFTERNOON SESSION
Reimbursement Systems for Pharmaceuticals in Europe
Level: Intermediate
Track: Health Policy & Regulatory
Unlike marketing authorization for pharmaceuticals, mainly regulated at the European level by EMA, pricing and reimbursement decisions in Europe are managed by individual member states. Health care services are generally covered by a single public health insurer operating under the Ministry of Health supervision. As a monopoly buyer, this situation provides a leading position for the public health insurer to set reimbursement conditions. Therefore, based on each country’s set of regulations, processes, and values, wide variations exist in pricing and reimbursement decisions of pharmaceuticals. Using up-to-date governmental regulation sources and the ISPOR Global Health Care Systems Roadmap, this course will discuss health technology decision-making processes for reimbursement decisions for pharmaceuticals in France, Germany, Hungary, Italy, Poland, Spain, Sweden, and the UK. The course will describe these reimbursement systems, as well as compare, and bring into contrast their key characteristics. This course is designed for individuals with intermediate experience within a single health care system wishing to broaden their appreciation of other reimbursement systems.
Faculty Member
Mondher Toumi, MD, MSc, PhD
Aix Marseille University, Marseille, 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.
Advanced Methods for Addressing Selection Bias in Real-World Effectiveness and Cost-Effectiveness Studies
Level: Intermediate
Track: Methodological & Statistical Research
Reimbursement agencies require real-world evidence on the effectiveness and cost-effectiveness of new drugs and medical devices. In many settings, randomised controlled trial (RCT) data is unavailable or insufficient. Where non-randomised data is used to estimate treatment effectiveness and cost-effectiveness, the main methodological challenge is selection bias from confounding by indication. Standard regression or propensity score methods are frequently used to adjust for selection bias, but estimates of treatment effectiveness may be highly sensitive to the chosen parametric form of these models, and evidence that relies on such methods may be viewed as insufficient by reimbursement agencies. While new, more advanced methods for allowing for confounding cannot offer a panacea, they have been shown to provide estimates of treatment effectiveness that are relatively robust. This course offers an in-depth description of ‘cutting edge’ methods for addressing this form of selection bias. These methods include flexible regression which uses machine learning for model selection, propensity score matching with regression adjustment, and Genetic Matching, a recently developed approach that extends propensity score matching. The course introduces the participants to these methods using the R software, through a series of real world data examples. Faculty will also demonstrate sensitivity analyses that convey to decision makers the extent to which the estimates of effectiveness and cost-effectiveness are robust to that assumption of no unobserved confounding. Participants who wish to have hands-on experience must bring their personal laptops with appropriate software installed.
Faculty Member
Richard Grieve, PhD
London School of Hygiene and Tropical Medicine (LSHTM), London, United Kingdom
Richard Grieve is a professor of Health Economics Methodology at the London School of Hygiene and Tropical Medicine (LSHTM). He holds a senior research fellowship from the National Institute of Health Research (NIHR), which aims to improve analytical methods for decision-making.
Since 2002, Richard has led a research programme on the design and analysis of observational studies for establishing relative effectiveness and cost-effectiveness. Richard has provided scientific advice to NICE and has contributed to Decision Support Unit (DSU) Technical Support Documents on the appropriate use of observational data. Richard also advises the Department of Health in England on how to improve the use of evidence from observational studies for policy-making purposes. Richard has published more than 90 articles in peer-reviewed journals, and secured about £25 million in research grants from the research councils, NIHR, and the DoH.
Noemi Kreif, PhD
Centre For Health Economics, York, United Kingdom
Noemi Kreif is a research fellow at the Centre for Health Economics, at the University of York. She holds a PhD in Health Economics from the London School of Hygiene and Tropical Medicine and she has recently held a UK Medical Research Council Early Career Fellowship in the economics of health, on statistical methods to address confounding in economic evaluation. Her methodological work includes translating advanced causal inference methods to health economic evaluation and decision modelling, in the complex settings of time-varying confounding, and more recently, for the economic evaluation of health system level interventions.
Stephen Oneill, MEconSc, PhD
National University of Ireland, Galway, Galway, Ireland
Stephen O’Neill is a lecturer in Economics (Health Economics & Ageing) at the National University of Ireland Galway and a Visiting Research Fellow at London School of Hygiene and Tropical Medicine (LSHTM).
Stephen's primary research interests are in health economics and micro-econometric methods. Stephen is particularly interested in translating methods from other fields within economics and from disciplines such as biostatistics, epidemiology, political analysis, and sociology, to the health economics domain.
Stephen completed a first degree in Economics at University College Dublin followed by a Masters in Economic Policy (Evaluation and Planning) and PhD in Economics at National University of Ireland Galway (NUIG). Along with Prof. Owen O’Donnell (Erasmus University Rotterdam), Prof. Tom Van Ourti (Erasmus University Rotterdam), and Dr. Brendan Walsh (Economic and Social research Institute), Stephen is the author of conindex, a command for the Stata statistical package, which computes a variety of inequality measures, including the concentration index and Gini coefficient.
Multi-Criteria Support Systems for Group Decision Making
Level: Intermediate
Track: Health Technology Assessment
MCDA models combine multiple dimensions of value (“attributes”) into a single metric, hence allowing evaluation of healthcare interventions in comprehensive ways using specific decision-makers’ values. Using different approaches, all MCDA models have two common features: (a) elicitation of decision makers’ values, and (b) transforming the performance of candidates (on multiple dimensions of value) into common scales (“data scaling”). This course emphasizes how to accomplish these key steps when groups (vs. individuals) are the decision makers (or are advising a final single decision maker). Various MCDA models differ substantially on the number of decisions required (by decision makers) to complete the models, a complexity that is exacerbated in settings with group decision making (voting), versus individual decision making. This course will review the virtues and complications of different MDCA models and provide hands-on testing of several voting methods to combine individuals’ preference weights into a group weights. Separately, we will explore various data-scaling mechanisms used in multi-criteria models, the errors they might introduce, and examine ways to simplify these processes. Finally, we will explore methods to create decision cut-offs (maximum willingness to pay) in multi-criteria models, akin to those used in cost-effectiveness analysis (CEA) when multiple factors interact under budget constraints. This intermediate-level course presumes at least an introductory familiarity with multi-criteria decision analysis (MCDA) models. It is designed to supplement (rather than as an alternative to) previous ISPOR Short-Courses that discuss MCDA at an introductory level.
Faculty Member
Guru Madhavan, PhD, MBA
National Academies of Science, Engineering, and Medicine, Washington, DC, USA
Guru Madhavan is a senior program officer and director of studies at the National Academies of Sciences, Engineering, and Medicine, where his portfolio of work has included leading: a global health program on infectious diseases, the creation of a decision support platform to help prioritize new vaccine development, and the analyses for making prescription medicines affordable and toward a national strategy for cancer control in the United States. He has given invited briefings at several state and federal agencies, Capitol Hill, European Commission, and the World Health Organization. He has served as a technical adviser to the US Department of Health and Human Services and has been a strategic consultant for technology startup firms and nonprofit organizations. A systems engineer by background, he received his MS and PhD in biomedical engineering and an MBA from the State University of New York. He has worked in the medical device industry as a research scientist developing cardiac surgical catheters for ablation therapy and advised the development and testing of neuromuscular stimulators for improving blood circulation. He has served two-terms as a vice-president and member of the board of directors of IEEE-USA. His honors include the Association for the Advancement of Medical Instrumentation’s AAMI–Becton Dickinson Award for Professional Achievement, the IEEE–USA Professional Achievement Award, and the Washington Academy of Sciences’ Krupsaw Award for engineering sciences and education. He has also received the inaugural Innovator Award and the Cecil Medal from the presidents of the National Academies of Sciences, Engineering, and Medicine. He was a founding member of the Global Young Academy and has been named a distinguished young scientist by the World Economic Forum. He has co-edited eight books and is author of Applied Minds: How Engineers Think (W.W. Norton) that has been published in multiple foreign markets and languages.
Charles E. Phelps, PhD, MBA
University of Rochester, Gualala, CA, USA
Charles E Phelps, PhD, University Professor and Provost Emeritus of the University of Rochester (Rochester, NY USA), health economist, has published major theoretical and applied research in health economics and particularly in cost-effectiveness analysis since beginning his career at the RAND Corporation, including a founding role in the RAND Health Insurance Experiment. During his time on the active faculty of the University of Rochester (1984 – 2010), he served for five years as chair of the (currently named) Department of Public Health Sciences, and then (for 13 years) as Provost of the University of Rochester. His more recent work has focused on expanding cost-effectiveness analysis using multi-criteria decision analysis (MCDA) models, voting theory (to improve use of MCDA models in group settings), and systems approaches to evaluating health care. His textbook, Health Economics, is in its sixth edition (2017) and (with Stephen T. Parente) published in 2017 a new book entitled “The Economics of US Health Care Policy.” He was elected to the Institute of Medicine (now the National Academy of Medicine) in 1991. Professor Phelps served as a member of the ISPOR Task Force on Value in Health in 2016-2017.
Risk-Sharing/Performance-Based Arrangements in Central & Eastern Europe- Implementation of Managed Entry Agreements
Level: Intermediate
Track: Health Policy & Regulatory
During the recent years, Managed Entry Agreements (MEAs) have become instrumental in ensuring the access of the innovative medicines. This course is designed for health care professionals (including public decision-makers, academia and industry) involved in pricing and reimbursement decisions who are wishing to understand the applicability and technical aspects of managed entry agreements (MEAs) in countries with severe economic constraints and explicit cost-effectiveness criterion. The topic will be introduced with key features of pricing and reimbursement systems in Central-Eastern European countries to understand why special methods are needed to facilitate evidence-based reimbursement policies of new health technologies. Faculty will present an economic model to explain the methodology and implications of managed entry agreements in cost-effectiveness and budget impact analysis. Participants will then have the opportunity to apply what they have learned through a hands-on exercise on making pricing and reimbursement decisions. A decision algorithm will be presented to support evidence and value based policy decisions of high-cost new technologies in CEE countries. A series of password protected economic models will add more and more complexity to a pragmatic case study on a new pharmaceutical product in oncology. To close the course faculty will lead a discussion on the applicability of a pragmatic decision tool illustrating the pros and cons of different managed entry agreements and their usefulness in CEE settings. Participants who wish to gain hands-on experience must bring their laptops with Microsoft Excel for Windows installed.
Faculty Member
Rok Hren, PhD, MSc
University of Ljubljana, Ljubljana, Slovenia
Rok Hren has more than 15 years of commercial experience in pharmaceutical industry, with more than 12 years on a board level in both (i) line management, which has included full P&L responsibility for operations in Slovenia and Romania, and (ii) leadership regional functions in Central and Eastern Europe. He regularly presents on the topic of pharmaceutical economics and policies at conferences in Europe and is well experienced in healthcare media business.
He received his PhD in Physiology and Biophysics from Dalhousie University, Canada and MSc in International Health Policy (Health Economics) with Distinction from London School of Economics and Political Science, UK while he was a post-Doctoral Fellow at Nora Eccles Harrison Cardiovascular Research and Training Institute, University of Utah Medical School, USA. He is also a professor at the University of Ljubljana and past president of ISPOR Slovenia Regional Chapter. In total, his publications gathered 300/394 citations (excluding self-citations) in WoS/Scopus (as of September 10, 2016).
Zoltan Kalo, PhD
Semmelweis University and Syreon Research Institute, Budapest, 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.
Katarzyna Kolasa, PhD
Kozminski University, Warszawa, Poland
Katarzyna Kolasa has more than 20 years of academic and business experience in the healthcare sector. She holds a PhD degree in health economics and is an author of more than 60 publications. Katarzyna Kolasa reviews manuscripts submitted to Health Policy, Value in Health, Expert Review of Pharmacoeconomics & Outcomes Research, International Journal for Equity in Health and International Journal of Technology As-sessment.
Katarzyna Kolasa heads the Health Economics & Healthcare Management Division (HeM) at the Kozminski University. She is the leader of the International Master Pro-gram Health Economics & Big Data at the Kozminski University financed by the re-search grant of the National Center for Research and Development which was listed as the best project in the POWER 2018 call.
In the past, she worked at both global and regional Health Economics & Outcomes Re-search (HEOR) functions at AstraZeneca and BiogenIdec being based in Sweden and Switzerland respectively. At Bristol Myers Squibb and Lundbeck she was leading Mar-ket Access teams in Central Eastern European (CEE) and Nordic Region. Her practical skills in the field of health economics were developed at the Kalmar County Council in Sweden as well.
For the last five years, she has been developing experience with the pricing &reimbursement challenges in the field of medical devices. Since 2016, Katarzyna has been representing Straub Medical as Global Market Access Lead being the External Principal Consultant for that Swiss family owned company. Before that, Katarzyna was employed as a Senior HEOR Director at GE Healthcare.
Her extensive knowledge in the field of health economics was acquired at University of York, University of Lund, and University of Bergen as well as during International Doc-toral Courses in Health Economics and Policy organized by the Swiss School of Public Health.
Katarzyna was nominated by the Ministry of Science to represent Poland in the COST Action CA17117, COST Association „Towards an International Network for Evidence-based Research in Clinical Health Research”. Since January 2019, she is working on the establishment of Global Special Interest Group for Digital Health at ISPOR.
Budget Impact Analysis II- Applications & Design Issues
Level: Intermediate
Track: Economic Evaluation
This course covers the concrete application of the six-step approach for developing budget impact analyses and provides hands-on learning with two different budget impact models programmed in Excel. The course will review the basics of budget impact analysis, interpretation of results, simplicity versus accuracy and face validity, and how budget impact analyses are used by payers and other decision makers. Technical topics will include static versus dynamic budget impact models, considerations for device and diagnostic technologies, and realistic features such as patient copayments and use of generics. The instructors will walk through two different budget impact analyses programmed in Excel (one static and one dynamic) and work with participants on hands-on exercises to enhance these models. The instructors will also review good practices for building budget impact models and provide a number of Excel tips. The Excel-based budget impact models used for the course will be provided to participants in advance of the conference. This course is designed for those who have basic knowledge of budget impact analyses and desire exposure to these analyses in Excel. Participants who wish to gain hands-on experience must bring their personal laptops with Microsoft Excel for Windows installed.
Faculty Member
Thor-Henrik Brodtkorb, PhD
RTI-HS Nordics, Ljungskile, Sweden
Thor-Henrik Brodtkorb, PhD, is senior director in Health Economics at RTI Health Solutions (RTI-HS). He holds a PhD in Health Technology Assessment from the University of Linköping and has been with RTI-HS for 7 years. He has been teaching courses in decision-analytic modeling at Linköping University as well as presented workshops and short courses on decision-analytic modeling techniques for organizations such as Pharma Industry Sweden, Swedish Agency for Health Technology Assessment and Assessment of Social Services (SBU), and the International Society for Pharmacoeconomics and Outcomes Research (ISPOR).
At RTI-HS, Dr. Brodtkorb leads development of cost-effectiveness, cost-utility, cost-consequence, and budget-impact models for pharmaceutical, device, and diagnostic technologies. These models have been used to support reimbursement decisions in more than 15 European countries including NICE in UK, SMC in Scotland, TLV in Sweden, and NOMA in Norway. He has developed models and analyses in the areas of oncology, alcohol dependence, major depressive disorder, Alzheimer’s disease, dermatology, multiple sclerosis, cardiology, orthopedics, and asthma. His research has been presented at professional conferences and published in peer-reviewed journals. He is also a coauthor of the newly published book Budget-Impact Analysis of Health Care Interventions: A Practical Guide.
Anita Brogan, PhD
RTI Health Solutions, Manchester, LAN, United Kingdom
Anita Brogan is Head of Decision Analytic Modeling within the Health Economics division at RTI Health Solutions (RTI-HS). She holds a PhD in Operations Research from the University of North Carolina and has been with RTI-HS for 16 years. She has presented workshops, distance learning courses, and short courses on decision-analytic modeling techniques in a variety of venues, including meetings of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and the Academy of Managed Care Pharmacy (AMCP). Dr. Brogan has held an adjunct professorship in the Fermanian School of Business at Point Loma Nazarene University. She is also a coauthor of the book Budget-Impact Analysis of Health Care Interventions: A Practical Guide.
In her role at RTI-HS, Dr. Brogan uses analytical techniques to assess and present the clinical and economic value of emerging pharmaceutical and biotechnology products. She leads development of user-friendly and transparent cost-effectiveness, cost-utility, and cost-consequence models; budget-impact models; resource allocation models; and infectious disease transmission models programmed in Microsoft Excel and other platforms. She has experience with numerous types of modeling procedures, including Markov and other stochastic models, simulation, regression, linear and nonlinear programming, and various types of sensitivity analysis. Dr. Brogan has developed models and analyses in the areas of HIV, hepatitis C, RSV, influenza, cystic fibrosis, spinal surgery, mental health, women’s health, oncology, osteoporosis, chronic pain, age-related macular degeneration, bone healing, hospital-acquired infection, financial portfolio optimization, and vehicle routing. Her research has been presented at various professional conferences and published in peer-reviewed journals.
Stephanie R. Earnshaw, PhD, MS
RTI Health Solutions, Research Triangle Park, NC, USA
Stephanie Earnshaw is Senior Vice President of Health Economics at RTI Health Solutions (RTI‑HS). She received her PhD in Industrial Engineering at North Carolina State University and has been with RTI-HS for over 19 years. She has presented workshops, distance learning, and short courses on decision-analytic modeling techniques for pharmaceutical companies and organizations such as the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), the Academy of Managed Care Pharmacy (AMCP), and the Centers for Disease Control and Prevention (CDC). Dr. Earnshaw currently serves on the ISPOR Board of Directors, and she has held an Adjunct Faculty appointment at the University of North Carolina’s Eshelman School of Pharmacy, Division of Pharmaceutical Outcomes and Policy. She is also one of the lead authors of the newly published book Budget-Impact Analysis of Health Care Interventions: A Practical Guide. Dr. Earnshaw’s research focus is in applying decision-analysis techniques to industry-related issues and health care problems. Her areas of specialization include mathematical programming (constrained optimization), network optimization, and Markov, simulation, and other state transition modeling. She has developed innovative mathematical models using these methods to determine pricing strategy, predict clinical outcomes, and allocate resources. In addition, she continues her support for the pharmaceutical, biotechnology, and diagnostic and medical device industry by developing budget-impact and cost-effectiveness models for their health technologies. Therapeutic areas include cardiovascular disease, gastrointestinal disorders, respiratory disease, transplantation, infectious disease, osteoporosis, vaccines, and oncology. She is a member of the International Society for Pharmacoeconomics and Outcomes Research and the Institute for Operations Research and the Management Sciences. She has presented her work at professional conferences and has published in several peer-reviewed journals.
New! Mapping to Estimate Utility Values from Non-Preference-Based Outcome Measures. Part 2
Level: Intermediate
Track: Patient-Centered Research
Mapping is the term predominantly used to refer to studies that employ individual patient level data to estimate health state utility values from some non-preference based outcome measures. It is a practice undertaken in HTA, usually but not exclusively, when clinical trials have not collected preference-based instruments (a requirement to be able to derive QALYs in a cost-utility analysis). Using a mixture of lectures and practical exercises, this course will teach participants how to analyses individual patient level data to derive estimates of health state utilities from non-preference based outcome measures.This is an intermediate level course that will help participants to conduct mapping studies consistent with ISPOR Good Practices. It is a practical, computer-based course that requires participants to be familiar with the concept of mapping, associated good practices, and have a reasonable degree of competency with statistics and the use of the software Stata. Faculty will provide instruction on how to organize the data, pre-modeling checks, and detailed teaching on the implementation of methods that are recommended for mapping. These include both direct methods (where the health utility scores are the dependent variable) and indirect methods (where responses to the descriptive system of a preference based instrument are modeled). Computer code and pre-programmed Stata commands will be the basis for much of the practical instructions. Participants are encouraged to bring their personal Laptop computer with Stata 14 installed for use during the practical exercises. Attendees who do not own a copy of Stata can purchase an educational version (lasting 6 months) here http://www.stata.com/order/new/edu/gradplans/student-pricing/nodl/ for a small cost.
Faculty Member
Monica Hernandez, MSc, PhD
University of Sheffield, Sheffield, NYK, United Kingdom
Monica Hernandez, BSc, MSc, PhD, is an econometrician and health economist with extensive experience in developing complex statistical and econometric models. She has worked extensively on measurement error and latent variable modelling, and the analysis of observational data. On the latter area of research, she has been an author of two technical Support Documents providing guidance and future research recommendations on the use of observational data for submissions to the National Institute for Health and Care Excellence (NICE).
Recently, her research has focused on investigating the properties and policy use of EQ-5D, leading to the development of new methods for analyzing and mapping preference based measures. Monica has developed Stata user-written commands to allow practitioners to use those newly developed methods.
She was a member of the ISPOR Good Practice Guideline Task Force on Health Utilities Mapping on which this short course is based.
Andrea Manca, MSc, PhD
University of York, Heslington, York, United Kingdom
Andrea Manca is an applied health economist with more than 20 years’ experience in the area of economic evaluation for healthcare decision-making. He has evaluated a wide range of technologies (therapeutic medical devices, drugs, diagnostics and non-pharmaceutical interventions) in a number of different clinical areas, including oncology, cardiology and respiratory diseases, diabetes, chronic pain, surgical interventions, obstetrics, and gynecology.
His research interests focus on developing methodological solutions to address the issues that arise when analyzing and interpreting health economics and outcomes research data to inform person-centered healthcare decision-making. This involves the use of advanced statistical and decision modelling techniques for the analysis of cost effectiveness and outcomes data, including quantitative evidence synthesis and health econometrics methods.
Andrea received the ISPOR Award for Excellence in Methodology in Pharmacoeconomics and Health Outcomes Research (2008), and a number of other national scholarships and awards from the BackCare Society (2004), the Wellcome Trust (2007), and the UK National Institute of Health Research (2009).
He is a former co-editor (currently serving on the EAB) of the scientific journal Value in Health and member of the ISPOR Bernie O'Brien New Investigator Award panel. Andrea is deputy chair of the UK NIHR Doctoral Research Fellowship scheme and member of the Technology Appraisal Committee of the National Institute for Health and Care Excellence (NICE) for England and Wales.
Andrea holds a MSc in Health Economics (1998) and a PhD in Economics (2005), both by the University of York, UK.
His full CV can be found here: https://www.york.ac.uk/che/staff/research/andrea-manca/
Allan Wailoo, MA, MSc, PhD
University of Sheffield, Sheffield, United Kingdom
Allan Wailoo, PhD, is a professor of Health Economics in the School of Health and Related Research (ScHARR), University of Sheffield, UK where he has worked since 2000. He directs the National Institute for Health and Care Excellence’s Decision Support Unit and is co-director (with Mark Sculpher) of the UK Department of Health Policy Research Unit for Economic Evaluation.
He is a member of NICE’s Medical Technologies Appraisal Committee and co-editor for the ISPOR journal Value in Health and recently chaired the ISPOR Task Force on Good Practices in Mapping on which this short course is based. He has published numerous papers on mapping methods and has wider research interests in all areas of economic evaluation and related methods.
New! Creating Natural, Flexible Models with DICE Simulation
Level: Introductory
Track: Methodological & Statistical Research
DICE simulation is emerging as a standard approach to designing and building models for health technology assessment (HTA) because it is very transparent, simple to use and yet flexible enough to implement everything from cohort Markov to discrete event simulation in plain Excel. This course will provide a detailed understanding of the DICE concepts, its components and how it works. A discrete event simulation and a Markov model will be constructed live to illustrate how quick and easily DICE models are implemented and modified and highlight the advantages and disadvantages of DICE. This course is designed for those with some familiarity with modeling. For this course, participants are required to bring a fully charged personal laptop equipped with Microsoft Excel installed, a version 2007 or later. It needs to be a Windows version so if it is on a Mac, a Windows emulator and Windows Excel is needed.
Faculty Member
J. Jaime Caro, MDCM, FRCPC
Evidera, Waltham, MA, USA and University McGill, Canada, Waltham, MA, USA
J. Jaime Caro, MDCM, FRCPC, FACP, is chief scientist at Evidera where he advances Evidera’s leadership in developing and applying novel techniques in modeling, health economics, comparative effectiveness, epidemiology, and outcomes research. Dr. Caro is also adjunct professor of Medicine, Epidemiology and Biostatistics at McGill University, and professor in Practice at the London School of Economics. He also lends his teaching ability to other academic institutions such as Thomas Jefferson University School of Population Health.
Dr. Caro continues to pioneer new methodologies. In an effort to provide an alternative to the well-known cost per QALY technique and avoid many of the latter’s problems, he is working on a broader approach to valuing health benefits. He is also further developing DICE, the unified approach to health economic modeling that he has created. Working with health technology assessment agencies and academic groups, he is formalizing this innovation to enable rapid, standardized and less error-prone development of decision-analytic model. Previously, Dr. Caro adapted an engineering technique – discrete event simulation – to model diseases and their treatment and extended it further to simulate the design of clinical trials and other types of studies, something which has been particularly effective in helping design pragmatic clinical trials. He has also applied the technique to provide comparative effectiveness information in the absence of head-to-head trials in a new method called Simulated Treatment Comparison. In response to increasingly frequent requests by authorities to provide data to support the value of new interventions, Dr. Caro has applied simulation techniques to make post-marketing registries more feasible and efficient – an approach called SAVES.
On behalf of the German health technology assessment agency, he proposed an innovative approach to the assessment of health technologies, involving the efficiency frontier. As part of his work with governments, he has helped the World Bank Institute and the InterAmerican Court for Human Rights address the growing problem of Supreme Courts overriding health care system decisions and ordering them to provide treatments that had been considered unwarranted. After leading the Quality Assurance for Modeling Studies Task Force, jointly sponsored by ISPOR, Academy of Managed Care Pharmacy and the National Pharmaceutical Council, and the ISPOR-SMDM Good Modeling Practices Task Force, endorsed by the Society for Medical Decision Making, Dr. Caro was recently named co-chair of the ISPOR Science and Research Committee and has been awarded the Marilyn Dix Smith Leadership Award.
Jörgen Möller, MSc
Evidera, Hammersmith, United Kingdom
Jörgen Möller, MSc, is Vice President, Modeling Technologies with Evidera in London, UK. He specializes in simulating complex systems and played a key role in simulating the re-design of the Swedish national postal distribution system, the extension of Stockholm-Arlanda Airport, and the utilization of surgical theatres at Eskilstuna Hospital. Intrigued by the dearth of simulation in pharmacoeconomics and outcomes research, Mr. Möller joined Caro Research in 2003 as a specialist in discrete event simulation (DES) and has been instrumental in implementing DES as a modeling tool in medicine. His focus has been on translating methods from operations research to pharmacoeconomics and on developing guidelines for this type of modeling. Mr. Möller has created more than a dozen DES models in the areas of devices and pharmaceuticals. He also conducts advanced training courses in DES and the ARENA-software. Mr. Möller received an MSc-degree in mechanical engineering at Lund University, Sweden.
Value of Information (VOI) Analysis
Level: Intermediate
Track: Economic Evaluation
Value of Information (VOI) techniques provide the analytic framework to estimate the value of acquiring additional evidence to inform a decision problem. VOI analysis is increasingly used to inform research prioritization decisions as it allows evaluating the extent to which new evidence might improve expected benefits by reducing the level of uncertainty in the current evidence base and compares that improvement with the cost of conducting the research. The course will provide a thorough understanding of the concepts and methods used in VOI analysis for participants with a working-knowledge of model-based cost-effectiveness analysis and its role in healthcare decision making. The course is based on the recommendations of the ISPOR Value of Information Task Force Reports and will 1) provide participants with an introduction to the fundamentals of VOI; 2) explain why VOI is important to decision makers; 3) identify the types of healthcare decisions that can be supported by VOI, as well as its limitations; 4) describe how the methods should be used and how the results should be interpreted, and 5) explain how VOI can support decision making in different contexts. Participants are provided with the opportunity to engage with concrete applications of the six-step approach for VOI analyses, including identification and characterization of different sources of uncertainty, calculation of the Expected Value of Perfect Information (EVPI), Expected Value of Perfect Parameter Information (EVPPI), Expected Value of Sample Information (EVSI) and Expected Net Benefit of Sampling (ENBS) and interpretation / presentation of results. To help participants engage with the course content, the instructors will walk through each step of the VOI analyses using worked examples (e.g. on VOI of next-gen sequencing studies, immunotherapy trials, and other emerging technologies). They will guide participants through hands-on exercises in Excel and R, show available online VOI tools, and lead discussions on the topic. This course is designed for beginners in VOI analysis that have some experience with probabilistic decision modelling. Participants are required to bring a laptop with Excel and/or R installed (software choice depending on their own preference).
Faculty Member
Elisabeth Fenwick, PhD
Pharmerit International, Oxford, OXF, United Kingdom
Elisabeth Fenwick is a senior director in the Modeling and Meta-Analysis team at Pharmerit International, based in Oxford in the UK.
Liz provides scientific and strategic support to HE projects globally. She has extensive experience in economic evaluation and health economic modeling having worked in the field for over 20 years. She has worked on a variety of projects in a wide range of disease areas including oncology, respiratory, infectious diseases, cardiology, ophthalmology, and orphan diseases.
Liz has also contributed to methods in the field, in particular relating to decision analytic modeling and simulation methods, probabilistic decision analytic modeling and value of information analysis. Liz was a member of the ISPOR joint task force on good research practices in modeling and a co-author on the joint taskforce paper on uncertainty and is currently co-chairing the ISPOR task force assessing emerging good practice in value of information analysis for research decisions. Liz is also a member of the editorial board for Pharmacoeconomics.
Liz has a PhD and MSc in Health Economics as well as an MSc in Operations Research and joined Pharmerit from ICON plc where she led the modeling team for the global HE group. Prior to her consultancy career, Liz spent over 15 years as an academic working at University of York, McMaster University, and most recently University of Glasgow.
Erik Koffijberg, PhD
University of Twente, Enschede, Netherlands
Dr H. (Erik) Koffijberg, PhD, MSc, is Associate Professor in the field of Health Technology Assessment at the University of Twente, the Netherlands.
Dr Koffijberg has published > 90 scientific papers in peer reviewed journals, and secured > 4M€ in funding for research on methods, and on applications, in the field of health technology assessment. He has performed applied research in this field, such as cost-effectiveness, risk-benefit, and budget impact analyses. He also contributed to methodological research in this field, amongst others on the topics of (early) HTA of new tests and clinical prediction rules; optimizing screening strategies, and prioritizing research, based on systematic reviews and meta-analyses. His work focuses foremost on impact assessment of diagnostic technologies on healthcare and healthcare delivery, in the medical fields of cardiology, neurology and oncology.
Dr. Koffijberg teaches BSc, MSc, and postgraduate (external) courses on topics such as Health Technology Assessment, Health Economic Modeling, Discrete Event Simulation in Healthcare, and Advanced Diagnostic Research. He has been/is PI in several large (international) research consortia: HEART-Impact trial (Effect of Using the HEART Score in Patients With Chest Pain in the Emergency Department); CREW study (Dutch National Consortium to Promote Cardiovascular Healthy Aging in Women); TANGO study (Systems modeling of the nation-wide organization of WGS in the Netherlands); RED-CVD study (Reviving Early Diagnosis of CardioVascular Disease).
Dr. Koffijberg is member of the Dutch Society for Health Economics (VGE), the ISPOR taskforce group on VOI analysis, and the multi-stakeholder Value of Diagnostic Information Platform.
Lotte Steuten, PhD, MSc
Office of Health Economics, London, WA, United Kingdom
Professor Lotte Steuten is Vice President and Head of Consulting of the Office of Health Economics, and honorary visiting professor at City, University of London (UK).
Lotte’s research interest focuses on the development and application of health economic analysis and health technology assessment (HTA), with the aim to accelerate patient access to high value health care services and treatments. She specializes in quantitative methods for estimating and comparing the expected health and economic benefits of new approaches and interventions in disease prevention, diagnostics and treatment, and prioritizing data collection to efficiently build the evidence for new interventions.
After obtaining her PhD she has acquired >15 years of international expertise in leading health economics and HTA projects to inform a wide range of decision problems, working effectively with various academic and non-academic stakeholders including patients, care providers, payers, policy makers, innovators and manufacturers.
Prior to joining OHE, Lotte was an associate member at the Hutchinson Institute for Cancer Outcomes Research (HICOR) at the Fred Hutchinson Cancer Research Center, and an affiliate associate professor in the Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute at the University of Washington, Seattle. She co-founded Panaxea bv, a health innovation consultancy firm with offices in the Netherlands, Belgium and Germany, and served as its Chief Executive and Scientific Officer for nearly 10 years.
Lotte has published nearly 100 original peer reviewed journal articles and is a frequent presenter and panellist at conferences on health innovation and economics worldwide.
Network Meta-Analysis in Relative Effectiveness Research
Level: Intermediate
Track: Study Approaches
For several medical questions of interest, many treatment options exist for the same indication. These treatments may have been compared against placebo or against each other in clinical trials. Knowing whether one specific treatment is better than placebo or some other specific comparator is only a fragment of the big picture, which should incorporate all available information. Ideally, one would know how all the treatment options rank against each other and the level of differences in treatment effects between all the available options. Network meta-analysis provides an integrated and unified method that incorporates all direct and indirect comparative evidence about treatments. Based in part on the ISPOR Task Force Reports on Indirect Treatment Comparisons, the fundamentals and concepts of network meta-analysis will be presented. The evaluation of networks presents special challenges and caveats, which will also be highlighted in this course. The material is motivated by instructive and concrete examples. The ISPOR-AMCP-NPC questionnaire for assessing the credibility of a network meta-analysis will also be introduced. This course requires at least a basic knowledge of meta-analysis and statistics.
Faculty Member
Sarah Goring, MSc
SMG Outcomes Research, Vancouver, BC, Canada
Sarah Goring has more than 15 years of experience in health economics and outcomes research (HEOR). Previously a Principal at ICONplc, Sarah is now an independent consultant, providing scientific leadership on a broad range of HEOR projects including epidemiological and burden of illness studies, health services research, systematic reviews, and evidence synthesis. Sarah has a particular interest in methodological innovations; she has applied and developed novel methods relating to fractional polynomials, simulated treatment comparisons, matching adjusted indirect comparisons, and issues in network structure and disconnected networks. Sarah has a B.Sc. in mathematics from the University of Victoria and an M.Sc. in health care and epidemiology from the University of British Columbia. Sarah is a co-editor of a Springer textbook on Health Services Research and has co-authored more than 50 research contributions.
Jeroen P Jansen, PhD
Precision Xtract, Oakland, CA, USA
Jeroen P. Jansen is the chief scientist – Evidence Synthesis and Decision Modeling at Precision Xtract and adjunct professor of Health Research & Policy (Epidemiology) at Stanford University. In addition, he is lead scientific advisor - Open Source Value Project (OSVP) at the Innovation and Value Initiative (IVI) where he oversees the development of open-source cost-effectiveness models. He has 15 years of research experience in the areas of evidence synthesis and economic modeling. He was an associate editor of the Research Synthesis Methods Journal; and is co-author of a textbook on network meta-analysis for decision-making. Dr. Jansen has published extensively on both applied and methods research in his areas of expertise. Notable contributions are the development of guidance for consumers and producers of network meta-analysis studies (ISPOR, PRISMA), as well as the development of methods for network meta-analysis of time-to-event data, repeated measures, and integration of patient level data with study level data. Previously, he was a founder of Redwood Outcomes, a health economics and outcomes research firm that was acquired by the Precision Medicine Group.
17:30 - 18:30
EDUCATIONAL SYMPOSIUM
HOW HTA METHODOLOGY AND EVALUATION FRAMEWORK SHOULD ADAPT TO ENSURE PATIENT ACCESS TO CELL AND GENE THERAPIES?
There have been significant advancements in the development of cell and gene (C&G) therapies, such as gene targeting or stem cell-based therapies, but HTA frameworks have been slow to adapt. C&G therapies offer transformative benefits to patients but present a challenge to current HTA decision making systems because they are typically reviewed when clinical efficacy data are limited and there is uncertainty around the long-term durability of outcomes. This symposium aims to answer the question:
“How should HTA methodology and frameworks be adapted so that patients can benefit from the rapidly evolving science of C&G therapies”?
How can we better assess durability of outcomes from new C&G therapies with limited data on long-term clinical effectiveness?
The potential magnitude of long-term positive health outcomes that C&G therapies may provide highlights the importance of including additional elements of value into HTA assessment. We will discuss what the most important additional value elements are and how they could be incorporated into HTA decision making.
Cost-effectiveness thresholds can make decision making dichotomous. The panel will discuss whether additional value drivers should be reflected quantitatively into weights or thresholds, or whether a deliberative approach is preferred.
The potential for huge health gains from C&G therapies will be reflected in high value-based prices however the financial benefits may not be shared with society in the same way the current system enables. Alternative models for surplus sharing have been proposed and the panel will discuss the feasibility of implementing these.
The panel and audience will debate potential solutions that could enable HTA methodology to make C&G therapies available to patients faster.
Sponsor
Novartis
Moderators
Mark Ratcliffe, PhD
PHMR Ltd, London, United Kingdom
Speakers
Louis P. 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.”
Stephen Palmer, Prof, PhD
University of York, Heslington, York, United Kingdom
Jie Shen, MSc, MBA
Novartis, Basel, Switzerland
Kun Zhao, MD, MHSci, PhD
China National Health Development Research Center, National Health Commission (NHC), Beijing, China
18:45 - 19:45
INDICATION BASED PRICING- ALIGNING INCENTIVES AND VALUE TO IMPROVE PATIENT ACCESS
Clinical development and medical practice in the past decade has witnessed an increase in the number of new drugs approved for multiple indications. Yet, a “one price for one drug” rule dominates global pharmaceutical pricing approaches. A single price for a single drug (if the drug has multiple indications) creates a disconnect between price and value. The symposium will present the most up-to-date synthesis of current thinking about the concept of Indication Based pricing (IBP). The session will discuss the rationale for IBP and what it means for patients, industry and payers. The symposium will review how various countries currently deal with the challenge of pricing medicines with multiple indication, and how these practices differ from an theoretically optimal model of IBP. The symposium will showcase interim results of the ongoing academic research on the effects of the lack of a formal IBP policy by the London School of Economics and Political Sciences. The symposium will provide a floor for a pluralistic debate about pros and cons of IBP, versus status quo, and explore practical ways of making IBP work for everyone.
Sponsor
London School of Economics and Political Science
Moderators
Panos Kanavos, PhD
London School of Economics and Political Science, London, United Kingdom
Speakers
Niklas Hedberg, MSc, Pharmacy
The Dental and Pharmaceutical Benefits Agency (TLV), Stockholm, Sweden
Paula Lorgelly, BSc(Hons), PhD, PGCAP
University College London, London, United Kingdom
Mon 4 Nov
7:00 - 8:15
ISPOR FORUMS
ISPOR MEDICATION ADHERENCE AND PERSISTENCE SPECIAL INTEREST GROUP- METHODS FOR MEASURING MULTIPLE MEDICATION ADHERENCE; RESULTS AND FUTURE WORK
Attendees interested in medication adherence, the management of patients with multiple medical comorbidities, or the health and economic challenges of polypharmacy are welcome to join this forum of the Medication Adherence and Persistence Special Interest Group. A brief presentation on the main findings of the recently released systematic literature review on the methods for measuring multiple medication adherence will be followed with an open discussion on the proposed future projects of the Special Interest Group. Topics related to adherence in patients receiving polypharmacy, such as how to validate multiple medication adherence measurements using real world data and how to evaluate adherence enhancing interventions along multiple criteria will be discussed with the audience. Attendees will have the opportunity to contribute their perspectives, discuss ideas for future studies and volunteer to help activities of the Special Interest Group. Grab your lunch and join us for this informative discussion.
Speaker
Tamas Agh, MD, PhD
Syreon Research Institute, Budapest, Hungary
Mia Malmenas, Msc, MBA
ICON plc, Stockholm, Sweden
ISPOR AUSTRIA CHAPTER- USING DIGITAL DATA TRANSFORMATION FOR STRATEGIC PATIENT EMPOWERMENT
Amplifying the voice of the patient alone does not lead to better care. Strategic patient empowerment (PATEMP) is the prospective collection of patient voices with a mechanism for implementation of findings. Strategic PATEMP makes use of digital data to communicate patient values to stakeholders at different levels. This enables them to make patient centered decisions for the efficient and timely delivery of effective healthcare services in the most appropriate setting. This forum describes PATEMP tools and how different stakeholders can use them strategically. Stakeholders will discuss: government collected patient reported outcomes and experience measures, digital patient-generated health data, patient engagement in HTA and decision analysis, pharmaceutical product development and clinical trial design, and point-of-care level wearables, mobile devices, and online platforms. Presentations will be followed by a discussion open to the audience and moderated by a patient representative.
Moderators
Ernst Leitgeb
Bundesverband Selbsthilfe Österreich - Austrian Association of Patient Advocacy and Support, Vienna, Austria
Speakers
Gottfried Endel, Dr.
Main Association of Austrian Social Security Institutions, Vienna, Austria
Ansgar Hebborn, PhD
F. Hoffmann-La-Roche AG, Basel, Switzerland
Beate Jahn, Dipl.Math. oec. Dr.rer.soc.oec
UMIT – University for Health Sciences, Medical Informatics and Technology, Hall i.T., Austria
Noemi Kiss, MSc
Medical University of Vienna, Vienna, Austria
Günther Zauner, Dipl.Ing
dwh GmbH, Vienna, Austria
7:15 - 8:15
EDUCATIONAL SYMPOSIUM
CELL AND GENE THERAPIES - VALUE AND ACCESS
Recent advances in cell and gene therapies are rapidly evolving care for many life-threatening conditions with high unmet need. The introduction of durable and potentially curative new treatment options is changing the prospects for providers, patients, and their families. Regulatory agencies are accounting for these innovative treatments with new programs and guidelines. Under the FDA Cures Act, cell and gene therapies can receive a regenerative medicine advanced therapy (RMAT) designation, which accelerates access to market. Similarly, the EMA has developed a number of scientific guidelines specific to gene therapy products to keep pace with clinical and scientific innovations.
As regulators put policies in place to prepare for the coming wave of cell and gene therapies, significant challenges exist to assess long-term value using conventional metrics. Lack of effective comparators and heterogeneity of the patient population in clinical trials introduce uncertainties when assessing the value of these medicines. High upfront costs and limited evidence of long-term outcomes also highlight the need for new reimbursement models. A new value assessment framework is needed to incorporate broader elements of value into decision-making. In light of this landscape, presenters will discuss common considerations and potential alternative approaches to assess value-based pricing for cell and gene therapies. They will facilitate a discussion on how to prepare the healthcare system for the advancement of cell and gene therapies, from the perspectives of manufacturers, policy makers, and payers.
Sponsor
Analysis Group
Moderators
Eric Q. Wu, PhD
Analysis Group, Inc., Boston, MA, USA
Speakers
Omar Dabbous, MD, MPH
AveXis, Inc., Bannockburn, IL, USA
Michael Drummond, MCom, DPhil
University of York, York, 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.
Jenny Zhou, PHD, MS
Analysis Group, Boston, MA, USA
8:30 - 10:30
WELCOME AND FIRST PLENARY
CONFERENCE PROGRAM OVERVIEW FROM PROGRAM COMMITTEE CO-CHAIRS
Speaker
Dorte Gyrd-Hansen, PhD, MSc
University of Southern Denmark, Odense, Denmark
Dorte Gyrd-Hansen is Director of the Danish Centre for Health Economics, DaCHE, and Professor of Health Economics at Department of Public Health, University of Southern Denmark, and at University of Tromsø. Her research interests lie in the intersection of health economics and behavioral economics, with a particular focus on citizens’ preferences for health gains, and patients’ and health professionals’ behavior. She uses register data, survey data an conducts experiments in order to improve our understanding of policy relevant behavioral mechanisms. She is president-elect of the European Health Economics Association, and has previously held professorships at University of Queensland and Copenhagen Business School.
Carole Longson, PhD
Association of the British Pharmaceutical Industry (ABPI), London, United Kingdom
Hans Severens, PhD
Erasmus University, Rotterdam, Netherlands
PRESIDENTIAL ADDRESS
Speaker
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.
WELCOME FROM ISPOR CEO/EXECUTIVE DIRECTOR
Speaker
Nancy S. Berg
ISPOR, Lawrenceville, NJ, USA
Nancy S. Berg is chief executive officer (CEO) and executive director of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), the leading global scientific and educational organization for health economics and outcomes research. Ms. Berg has over 30 years of experience in health care and scientific/technical association leadership, and has been an entrepreneur and business consultant to both commercial and nonprofit organizations. At ISPOR, Ms. Berg is responsible for the Society’s global strategic direction and leadership of the association. She has led the organization, in concert with the Board of Directors, to design and implement a new strategic plan, mission, and vision to guide the Society into the future.
Previously, Ms. Berg led the International Society for Pharmaceutical Engineering (ISPE) as president and CEO. ISPE is the world's largest professional society fostering dialogue and education on technical, scientific, and regulatory best practices in the pharmaceutical and biotech industries. During her tenure with ISPE, Ms. Berg was the architect of the Society's Strategic Plan, focusing ISPE's energies and expertise on high-value initiatives related to drug shortages, quality metrics, global manufacturing quality, facility and supply chain issues, and patient experiences in clinical trials. Prior to her role at ISPE, Ms. Berg served as executive director and CEO of the Society of Manufacturing Engineers (SME). She also previously founded a strategic and business development consultancy company.
Ms. Berg has been engaged in education and business issues at national and international levels. She has served on many government initiatives involving business, community, development, growth, revitalization, and labor issues. She is a supporter of the Leukemia and Lymphoma Society’s Team in Training Program cycling events and a patron of many other charities. Ms. Berg is a graduate of the University of Michigan-Flint and is married to Timothy Jackson.
FIRST PLENARY SESSION- HEALTHCARE DIGITALIZATION- INSTANT, ON DEMAND, AND ALWAYS CONNECTED
Rapid change is characterizing health care systems now. New technologies, discoveries focusing on ensuring integrated and personalized care, and patient empowerment are influencing the way we prevent, diagnose and treat diseases. Experts say that in the years to come major changes will relate not only to technology (robotics, continuous on- and in-body health sensoring, man-made organs, and AI in healthcare decision support), but also to the way care is organized and delivered (telemedicine, hospital redesign, patient empowerment). In addition, the paradigm of healthcare services is shifting from treating illness to sustaining well-being of individuals for whom values, preferences, and expectations vary, making individualized care even more relevant. Within this wide range of developments, digitalization is considered to be the main driver for the way healthcare systems evolve, even at this very moment. Not only will digital technologies influence communication and collaboration within health care systems, but we will learn and discover from data that will be continuously and instantly available. The panel will explore what the objectives, incentives and aspirations of the new players in digital transformation of healthcare are, how the roles of traditional stakeholders in healthcare are changing, and what needs to be done to strengthen the effectiveness of the change efforts in delivering desired results.
Moderators
Bogi Eliasen
Copenhagen Institute for Futures Studies, Copenhagen, Denmark
Bogi Eliasen is a knowledge broker whose expertise lies in combining various fields of knowledge. He initiated the first population genome project, FarGen 2009 and works with bridging digital health and genomics in the context of the future of personalized health for which he in 2019 received the HIMSS Global Achievement Award.
Speakers
Elena Bonfiglioli
Microsoft, Brussels, Belgium
As regional leader for Microsoft Health and Life Sciences’ business, Elena is responsible to drive go-to-market strategy, strategic partnerships and business development with public health organizations, payors and pharma, working together with a community of over 150+ professionals across the EMEA region.
She is very passionate about the opportunity to enable the transformation of the health sector through new digital health experiences powered by Cloud and AI. She is an active advocate and public speaker, invited to represent the approach of Microsoft for Healthcare – see recent interview with HIMSS Media.
Elena has been working in the health sector for 15+ years, first in health policy at EU level and then in business development. In July 2017, Elena was elected to the HIMSS Europe Governing Council.
Mobilizing a core group of industry innovators, Elena started-off the European Cloud in Health Advisory Council, a vendor-neutral C-level forum aimed to foster cloud-first innovation in the health sector.
In 2013, working with a group of leading e-Health partners, Elena led two industry-wide go to market and advocacy positions: the Manifesto for a Healthier Europe and Healthier Cities.
From 2007-2012, Elena held the role of Senior Director Health and Education Policy in Europe, when she led the creation of the Employability Alliance aimed to empower 20 Mil people with Skills for New Jobs.
Elena started at Microsoft in 2003 as Director of Corporate Affairs and Corporate Responsibility. Before joining Microsoft, Elena worked for CSR Europe as Director of Corporate Programs - in charge of stakeholder engagement and CSR initiatives across member companies; actively contributing to the development of the CSR agenda in Europe. Elena co-founded the European Academy of Business in Society where she served as interim Executive Director. She is a co-founder and Board Member of the Women in Leadership (WiL) Network.
Elena started her career as policy advisor for the Italian government and the university of Modena on matters of fiscal policy, socio-economic impact of fiscal reform. She co-developed the model and software used to run fiscal reform simulations at national level.
Elena is a founding member of the Centre for Evolutionary Management, a network organization aimed to support executive development programs through meditation.
Alexandra Goncalves, MD, PhD, MMSc, FESC
Philips Healthcare, Cambridge, MA, USA
Alexandra Goncalves is the Philips Chief Medical Officer, Strategy, M&A and Partnerships, a position she has held since June 2019.
As an academic cardiologist with a business acumen, she provides functional leadership for clinical innovation and clinical strategy for Philips. Alexandra works closely and collaboratively with business, research and functional leaders across the organization, as well as exploring inorganic growth opportunities.
Alexandra has joined Philips in 2016 and worked three years as Sr. Medical Director for the Ultrasound business. She was instrumental for the Cardiovascular Ultrasound business success by leading the evaluation of medical trends, by providing clinical insights and strategic direction, and by developing impactful partnerships through fostering relationships with clinical, scientific, regulatory and marketing leaders worldwide.
Alexandra started her career as an academic cardiologist in Portugal and Spain and her past experience includes research in epidemiology and clinical trials in the Cardiovascular Department at the Brigham and Women’s Hospital/ Harvard Medical School (HMS). She earned a Master in Medical Sciences from HMS and while working as a clinical cardiologist, she earned a doctorate degree (PhD). Her work was a driving force towards the refinement of the use of echocardiography in transcatheter structural heart interventions.
Alexandra is an internationally recognized thought leader with business acumen and an extensive record of multi-disciplinary medical collaborations, as educator, committee leader, author, and editor. She serves as Affiliate Professor of Medicine in Portugal and has been awarded several prizes, including the prestigious Senior Clinical Research and Career Development Award from HMS and Portuguese Foundation for Science and Technology.
Executive Profile
Julian Isla
Microsoft, Foundation 29, Madrid, Spain
Julian Isla is the Chief Scientific Officer of the European Dravet Syndrome Federation, and European organization of patients with Dravet Syndrome. Julian Isla founded Dravet Syndrome Foundation seven years ago. Dravet Syndrome Foundation is committed to find new treatment for Dravet Syndrome, an epileptic encephalopathy having long lasting seizures refractory to treatment as severe developmental delay as main symptoms. Julian is the father of Sergio, a young boy six years old who has Dravet Syndrome. Julian is software engineer by training and he works for Microsoft as full time employee. Despite of not having a neuroscience or medical background he gained the skills to be part of the Orphan Drug Committee at European Medicines Agency (EMA) as patient representative. Julian is also part of the Therapeutic Advisory Group for Eurordis, the biggest organization of rare diseases in Europe. In Spain he is ambassador of the Spanish Rare Diseases Federation and member of Ciberer (Spanish Network for research on rare diseases) scientific advisory group”
Peter Knox, MPP
The Life Raft Group, Wayne, NJ, USA
Peter is responsible for oversight of all research activities at the Life Raft Group (LRG), a non-profit that has been advocating for a rare form of cancer (Gastrointestinal Stromal Tumor, or GIST) for nearly 20 years. In particular, Peter and the LRG have been involved in the production and analysis of Real World Data (RWD) and Real World Evidence (RWE) through a patient registry, web-based side effect management platform, and other data products aimed at both patients and clinicians/researchers, and possess significant experience working with various HEOR professionals in the pharmaceutical industry. Prior to working in patient advocacy, Peter worked for various technology and web firms where he gained valuable knowledge on both the user perspective and how technology can be applied to the current digital health paradigm. A holder of a Masters in Public Policy with a concentration in quantitative methods, economics, and health policy from Rutgers University, Peter is especially proud to be working with ISPOR’s newly formed RWE special interest group, where he will enthusiastically offer his knowledge of the patient and caregiver perspective.
Ernst Kuipers, MD PhD
Erasmus MC University Medical Center, Rotterdam, Netherlands
Ernst trained in Internal Medicine and Gastroenterology. He became professor of Medicine and chair of the Departments of Gastroenterology and Hepatology (2000), Internal Medicine (2006), and Surgery (2012) of Erasmus MC University Medical Center in Rotterdam, the Netherlands. His clinical and research interests included early neoplastic gastrointestinal conditions, and colorectal cancer screening. He is fellow of the American Gastroenterology Association and the World Endoscopy Organization’s regional lead on colorectal cancer screening for Europe and the Middle East. He organized and contributed to various international guidelines on cancer screening. He serves since 2013 as CEO of Erasmus MC, a university medical institute that includes a medical school, research facilities and hospitals. It has 4.500 biomedical students, 17.000 employees and an annual budget of 1.7 billion euro. It is the largest university medical center in the Netherlands. In addition, he chaired the board of the Dutch Federation of University Medical Centers, a group of 8 UMCs with 67.000 employees. He also chairs the Dutch national emergency medicine network, and is member of the Dutch national committee for capacity planning of medical specialists and the Dutch National Health Council.
Rebecca Miksad, MD, MPH
Flatiron Health, New York, NY, USA
Rebecca Miksad is a medical oncologist and health outcomes researcher who serves as senior medical director at Flatiron Health. In this role, Rebecca focuses on generating real-world evidence, establishing regulatory-grade quality standards and contributing to the development of clinically-relevant methods to real-world data evaluation and analyses. Rebecca maintains a clinical practice at Boston Medical Center. A nationally recognized clinician and researcher, Rebecca regularly publishes in leading medical journals, serves on national committees and lectures internationally. Prior to joining Flatiron Health, Rebecca was an assistant professor at Harvard Medical School, senior scientist at the Institute for Technology Assessment at Massachusetts General Hospital, and director of gastrointestinal oncology and gastrointestinal oncology clinical trials at Beth Israel Deaconess Medical Center (BIDMC). Rebecca’s academic research focused on improving cancer treatment decision-making through better characterization of patient outcomes and increasing relevance of clinical trial endpoints. Rebecca earned a BA cum laude in economics from Harvard University and an MD with honors in research from Cornell University. She was a resident in internal medicine at Cornell University/New York-Presbyterian Hospital, and completed hematology and medical oncology fellowships at Harvard Medical School/BIDMC. Rebecca earned a MMS from Harvard Medical School and an MPH in Clinical Effectiveness from the Harvard School of Public Health in clinical effectiveness (with an emphasis on decision science). She also completed a post-doctoral fellowship in the Dana-Farber/Harvard Cancer Center Program in Cancer Outcomes Research Training (PCORT).
10:30 - 14:00
RESEARCH POSTER SESSION 1
10:30 - 19:30
EXHIBIT & POSTER HALL HOURS
11:00 - 12:00
BREAKOUT SESSION 1
TRANSLATING ONCOLOGY CLINICAL TRIAL ENDPOINTS TO REAL WORLD DATA FOR DECISION MAKING
PURPOSE
: To explore challenges and solutions in translating oncology clinical trial endpoints into endpoints that can be captured and/or measured in real world data (RWD).
DESCRIPTION
: The majority of new oncology therapy regulatory approvals are based on non-mortality endpoints such as response and progression-free survival (PFS), and overall survival may not be available for HTA decision making. Response to therapy and disease progression within a randomized clinical trial (RCT) are defined using criteria that cannot be directly translated to routine clinical practice due to differences in clinical care processes and external or structural influences. With the increased interest in the use of real world data (RWD) to inform the clinical effectiveness of new therapies (including long-term benefit outside clinical trials), the use of endpoints developed specifically for RCTs introduces a hurdle in translating results from the RCT setting to real world clinical practice. To enable RWD to inform relative clinical effectiveness of new medicines, it is essential that multiple types of endpoints are readily available and accepted from data collected in routine clinical practice. The utility and acceptance of such endpoints varies across regulators and payers. Keith will introduce the session and moderate the discussion. Sylwia will provide an overview of current non-mortality endpoints used in HTA decision making, methods for surrogate endpoint validation and limitations of use of RCT data (15 minutes). Jessica will present on non-mortality endpoints abstracted from electronic health records (15 minutes). Federico will present two case studies that demonstrate the use of non-mortality endpoints from RWD to enable more informed predictions of OS. Attention will be drawn on the systematic differences observed in non-mortality time-to-event endpoints between real-world and trial settings (20 minutes). The audience will be polled to gain live feedback about each topic with a live discussion to close out the session.
Discussion Leader
Keith R. Abrams, PhD, MSc, CStat
University of Leicester, Leicester, United Kingdom
Keith Abrams, PhD, is a professor of Medical Statistics in the Department of Health Sciences at the University of Leicester, having previously held appointments at the London School of Hygiene and Tropical Medicine and King’s College School of Medicine and Dentistry, London, UK. He is a Fellow of the Royal Statistical Society, and a Chartered Statistician.
His research interests are primarily concerned with the development and application of Bayesian statistical methods in Health Technology Assessment (HTA), in particular their application to clinical trials, evidence synthesis, and decision modelling, and Non-Communicable Disease (NCD) epidemiology. This work is primarily supported with funding from EU, Medical Research Council (MRC), National Institute for Health Research (NIHR) and industry.
Professor Abrams has been extensively involved with the UK NIHR Health HTA Programme and UK National Institute for Health & Care Excellence (NICE) appraisal process since their inception. He was a member of the NICE Technology Appraisals Committee for over 8 years until 2015, is a member of the NICE Decision Support Unit and Technical Support Unit, and a NIHR Senior Investigator Emeritus. He has published widely in both substantive and methodological areas including co-Authoring books on Methods for Meta-Analysis in Medical Research, Bayesian Approaches to Clinical Trials and Healthcare Evaluation, and Evidence Synthesis for Decision Making in Healthcare, in addition to co-Editing one of the first texts on Methods for Evidence-based Healthcare.
Sylwia Bujkiewicz, PhD, Msc
University of Leicester, Leicester, United Kingdom
Jessica Davies, MPH
Roche Products Ltd, Welwyn Garden City, United Kingdom
Federico Felizzi, PhD
F. Hoffmann La Roche, Basel, Switzerland
RISK OF BIAS IN SYSTEMATIC REVIEWS WITH COST AND COST-EFFECTIVENESS OUTCOMES
PURPOSE
: Explore how the risks of bias in systematic reviews with cost or cost-effectiveness outcomes can be addressed in review design and assessed in its evaluation
DESCRIPTION
: A dramatic increase of health economic studies has led to a consequent proliferation of systematic reviews summarizing their results. The diversity of methods and outcomes of these reviews (eg, methodologic or synthesizing reviews, including trials or models, reporting absolute costs or cost-effectiveness ratios) make it difficult for the reader to use this summarized information most effectively. Systematic reviews are generally perceived as a “lens of evidence”. However, the reviews themselves are subjected to biases. Thus, understanding the risk of bias is necessary to transform the systematic reviews into a valuable tool for decision makers. Speakers will discuss:
Planning the review and literature search (10 min) Literature selection and validation (10 min) Literature extraction and synthesis (10 min) Presentation and reporting (10 min) We will compare how the recommendations for systematic reviews reporting cost outcomes align or disagree with the recommendations for clinical reviews, eg, the Overview Quality Assessment Questionnaire (OQAQ), Assessing the Methodological Quality of Systematic Reviews (AMSTAR), and Risk of Bias in Systematic reviews (ROBIS)). More specifically, the following issues will be discussed:
How specific should be objectives of cost-related reviews? What are the relevant grey literature sources reporting cost outcomes in systematic reviews? Should social networks be used as a source of cost data? Does artificial intelligence increase or minimize the risk of bias in systematic reviews? What is the best method for data synthesis in reviews with cost components (ie, applying a quantitative synthesis versus narrative or graphical syntheses)? When synthesizing the findings, how does one reach a threshold between consolidated conclusion and transferability of the results? This workshop will benefit decision makers, researchers, and producers of health technologies .
Discussion Leader
Salah Ghabri, PhD
French National Authority for Health (HAS), Saint-Denis La Plaine, France
Lena Mandrik, PhD
Sheffield University, Sheffield, YOR, Great Britain
J. L. Hans Severens, PhD
Erasmus University Rotterdam, Rotterdam, Netherlands
Torbjorn Wisløff, MSc, PhD
University of Tromsø, Tromsø, Norway
FAKE OR NOVEL ELEMENTS OF VALUE?
ISSUE: The ISPOR Special Task Force on U.S. Value Assessment Frameworks argued for the inclusion of novel elements of value that could augment conventional cost-effectiveness analysis (CEA). In addition to core elements of value assessment (e.g. QALYs and cost offsets) and common used elements (e.g. productivity and adherence), several novel elements to consider were identified: severity of disease; value of hope; insurance value; real option-value; reduction in uncertainty; reduced fear of contagion; equity; and scientific spillovers. In this issue panel, panelists will debate whether the proposed novel elements are relevant for healthcare decision-making; whether these are truly novel or already implicitly captured with conventional CEA; whether these elements should be formally included in an augmented CEA or considered as “qualitative factors” in the appraisal; the methodological and data challenges; and the implications in terms of opportunity costs and the cost-effectiveness ceiling ratio.
OVERVIEW: Ms Goring will provide an overview of the proposed novel elements of value. Prof. Garrison will outline the arguments in support of incorporating novel elements of value in a CEA from both a payer and society perspective. Prof. Briggs will outline concerns with incorporating some of the proposed novel elements of value from a conceptual and decision-making perspective. Dr Jansen will provide an overview of recent attempts to incorporate novel elements of value in cost-effectiveness models for rheumatoid arthritis (i.e. insurance value) and non-small-cell lung cancer (i.e. value of hope), outlines the encountered data and methodological challenges, and impact on estimates of value. The moderator will present for 5 minutes and each presenter will present for 10-12 minutes, with 15 minutes for panel discussion and 5-10 minutes for audience questions.
Moderators
Sarah Goring, MSc
SMG Outcomes Research, Vancouver, BC, Canada
Sarah Goring has more than 15 years of experience in health economics and outcomes research (HEOR). Previously a Principal at ICONplc, Sarah is now an independent consultant, providing scientific leadership on a broad range of HEOR projects including epidemiological and burden of illness studies, health services research, systematic reviews, and evidence synthesis. Sarah has a particular interest in methodological innovations; she has applied and developed novel methods relating to fractional polynomials, simulated treatment comparisons, matching adjusted indirect comparisons, and issues in network structure and disconnected networks. Sarah has a B.Sc. in mathematics from the University of Victoria and an M.Sc. in health care and epidemiology from the University of British Columbia. Sarah is a co-editor of a Springer textbook on Health Services Research and has co-authored more than 50 research contributions.
Panelists
Andrew Briggs, BSc (Hons), MSc, DPhil
London School of Hygiene & Tropical Medicine (LSHTM), London, United Kingdom
Louis P. 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.”
Jeroen P Jansen, PhD
Innovation and Value Initiative, Alexandria, VA, USA
EVALUATING DIGITAL HEALTH TECHNOLOGIES- WHY, WHAT AND HOW?
ISSUE
: It is recognised that digital technologies hold potential to help tackle some of the key health-related challenges faced across Europe. But supply is currently far outpacing demand for digital health apps. Healthcare professionals are not ready to make recommendations and commissioners are rarely confident to invest. In this busy space, there‘s an increasing need for standardised, evidence-based approaches to encourage implementation of the most innovative and effective digital technologies. However, traditional methods for evaluating health technologies may be ill-equipped to assess digital health technologies. Challenges include difficulties in performing RCTs, issues of interoperability and user friendliness, variability between settings, the iterative development of innovations and optimum time in the development process for evaluations. Other aspects important to digital technologies not well addressed by traditional HTA methods include access, equity, patient empowerment or goal-orientation.
OVERVIEW
: Chris Chesters will moderate, providing an overview of the landscape, including NICE’s efforts to define what ‘good evidence’ looks like for digital technologies. Key questions for the panelists to debate: What should be evaluated and when? At a centralised or decentralised level? What value frameworks should be used? Megan Coder will share her insights working in an international organisation for developers of complex digital health technologies. She will bring an international industry perspective into how digital products are being reviewed and outline the key challenges and opportunities for the industry. Dr Panos Stafylas will discuss his involvement in establishing an eHealth HTA framework as part of the Joint Action to support the eHealth Network. He will discuss the current situation and challenges ahead for the use of eHealth in Europe. Bettina Ryll will highlight the importance of collaboration when developing digital health tools, particularly the engagement of patients and the public in this process. Time will be allotted (at least 15 minutes) for audience discussion and debate.
Moderators
Chris Chesters, PhD
National Institute for Health and Care Excellence (NICE), Manchester, United Kingdom
Panelists
Megan Coder, PharmD, MBA
Digital Therapeutics Alliance, Arlington, VA, USA
Bettina Ryll, MD, PhD
Melanoma Patient Network Europe, and Past Chair of the Patient Advocates Working Group, European Society for Medical Oncology, Uppsala, Sweden
Panagiotis Stafylas, MD, MSc, PhD
HealThink, Thessaloniki, Greece
IS DELAYED ACCESS DUE TO ISSUES OF HTA CAPACITY A NECESSARY REALITY OR AN UNACCEPTABLE PROBLEM?
ISSUE: The volume of health technology assessments (HTAs) is expanding dramatically, driven by increasing numbers of multi-indication therapies and pressure to minimise delays between regulatory approvals and HTA recommendations. There are signs that HTA bodies are consequently struggling to conduct appraisals in a timely manner, placing patients at risk of 'procedural' delays to access, which may be a difficult pill to swallow. The reality is that HTA bodies have finite capacity and must maintain rigorous processes. However, it can also be argued that there is potential to alleviate capacity issues by reducing duplication, repetition and waste within individual HTA processes and across the global HTA system. In this context, should we accept 'procedural' delays in patient access as an inevitability of the HTA system or must we look more critically at whether stakeholders are doing all they can to address capacity concerns?
OVERVIEW: Matt Griffiths will present the issue, moderating discussion and challenging panellists to debate the extent to which capacity-borne delays are avoidable and how this can be achieved. Eric Low will provide insight into the real-world impact of HTA capacity issues on patients and present his views on the roles of different stakeholders in avoiding 'procedural' delays to patient access. Michael Drummond will present a critical view on whether we have the correct balance between ensuring robust, locally-relevant HTA and avoiding unnecessarily delayed access. Lesley Tilson will argue that HTA bodies have already made adaptations in response to the rising challenge to HTA capacity and provide her perspective on the benefits and risks of further streamlining appraisal processes and methods. Following presentations, audience members will be presented with the proposals for how different stakeholders could address the issue and asked to vote on what is most achievable. The floor will then be opened for discussion and audience suggestions.
Moderators
Matthew Griffiths, MA, MSc
Costello Medical, London, United Kingdom
Panelists
Michael Drummond, MCom, DPhil
University of York, York, 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.
Eric Low, NA
N/A, East Lothian, United Kingdom
Lesley Tilson, BSc(Pharm), PhD
National Centre for Pharmacoeconomics (NCPE), Dublin, Ireland
ARTIFICIAL INTELLIGENCE STUDIES
AI3: DEVELOPMENT OF A DECISION-AID FOR PATIENTS WITH DEPRESSION CONSIDERING TREATMENT OPTIONS- PREDICTION OF TREATMENT RESPONSE USING A DATA-DRIVEN APPROACH
11:30AM - 11:45AM
Kan K 1 , Jörg F2 , Wardenaar KJ2 , Blaauw FJ3 , Visser E2 , Buskens E2 , Mulder H4 , Cath D5 , Doornbos B5 , Schoevers RA2 , Feenstra T3 1 University of Groningen, University Medical Center Groningen, Groningen, GR, Netherlands, 2 University of Groningen, University Medical Center Groningen, Groningen, Netherlands, 3 University of Groningen, Groningen, Netherlands, 4 Wilhelmina Hospital Assen, Assen, Netherlands, 5 GGZ Drenthe Mental Health Institute, Assen, Netherlands
OBJECTIVES : Many different treatment options for major depressive disorder (MDD) are proven effective. However, it remains a challenge to determine the optimal choice of treatment for each individual patient. Studies show that patients need treatment (response) information to enable shared decision-making (SDM). We developed a decision-aid that predicts treatment response stratified by patients’ personal characteristics and informs about patient relevant outcomes. METHODS : Focus group interviews with patients and semi-structured interviews with clinicians were carried out to identify gaps in clinical practice, relevant components of the decision-aid, preferences regarding treatment outcomes, and preferences regarding the interface. All interviews were audio recorded, transcribed verbatim, and analyzed using the thematic approach. Additionally, a literature search identified predictors of treatment response. Data from GGZ Drenthe, a regional mental health care institution, served as a training set for developing prediction models. It contained 143 potentially predictive variables on patient characteristics, clinical variables, treatment data and outcome data. Data of 449 patients were used for variable selection and model development. A SuperLearner algorithm was developed, fitting and weighting different machine learning models (e.g., lasso, random forest, gradient boosting) to obtain an optimal hybrid prediction model. This was done for all relevant outcomes (e.g. clinical recovery, social functioning) identified in the qualitative research. Analyses were performed in R. RESULTS : A weighted lasso (0.59) and random forest (0.41) model showed an overall AUC of 0.90 for the outcome of remission (OQ45<55) in the validation set (n=112). Similar models for all outcomes were combined with the qualitative results and literature review to develop the decision-aid. The decision-aid was then integrated in an existing electronic health record system. CONCLUSIONS : A decision-aid for depression, based on statistical learning to show personalized predicted treatment response, has the potential to assist patients and clinicians in personalized treatment choice for MDD, simultaneously enhancing SDM.
AI2: GEO-SPATIAL RISK ASSESSMENT OF ASTHMA HEALTH RESOURCE USE AT THE MUNICIPAL LEVEL IN THE GREATER HELSINKI METROPOLITAN AREA OF FINLAND
11:15AM - 11:30AM
Navaratnam P 1 , Friedman HS2 , Tammi I3 , Navaratnam A4 1 DataMed Solutions LLC., New York, NY, USA, 2 DataMed Solutions, New York, NY, USA, 3 Ubigu Ltd, Tampere, Finland, 4 Sygeny Ltd, Helsinki, Finland
OBJECTIVES To characterize and compare asthma risk associated with select municipalities in the greater Helsinki metropolitan area (GHMA) of Finland, utilizing a unique geo-informatics risk assessment tool. METHODS The geo-informatics Risk Assessment (RAS) tool links relevant data sources by using a common geographical marker (e.g. municipal code), which is available in publicly accessible data (such as public health data, census data, environmental data, etc.) to compute asthma risk based on asthma medication use intensity across the municipalities of GHMA in Finland. Asthma risk was represented as a total risk score, which is a composite aggregation of three components: demographic risk (age, sex, etc.); socio-economic risk (income, unemployment rate, etc.); and health behavior risk (smoking, obesity, etc.). These risks scores were derived from a statistical analysis, followed by a machine learning predictive analytics approach using the asthma medication intensity risk as the outcome measure. The composite risk score as well as the component scores were rescaled to a range of 0-100. These risk scores were computed for each municipality in the GHMA and displayed as heat maps for better visualization. RESULTS GHMA municipalities [Etelä-Vuosaari (EV) and Helsinki Keskusta/Etu-Töölö (HKET)] with similarly sized populations were chosen to demonstrate the differences in asthma risk. EV (pop. 22,616; average age:40.0; 46.3% males) had a total asthma risk score of 33.0, whereas HKET (pop. 18,035; average age: 40.0; 47.3% males) had a total asthma risk score of 9.7. The national average is 43.5. EV total asthma risk was 3.4 times that of HKET. It appears that the % highly educated persons [21.90% (EV) vs. 52.20% (HKET)] and average income [22,321 Euros (EV) vs. 35,952 Euros (HKET)] may be driving this difference in risk. CONCLUSIONS The RAS tool provides deep insight into asthma risk, by utilizing relevant data sources linked by a common geographical marker.
AI1: PREDICTORS OF HIGH HEALTHCARE UTILISERS AMONG PATIENTS WITH CHRONIC HYPOPARATHYROIDISM AND APPLICATION OF MACHINE LEARNING METHODOLOGY
11:00AM - 11:15AM
Chen K 1 , Gao W2 , Wang Y3 , Swallow E2 1 Shire Human Genetic Therapies, Inc., a member of the Takeda group of companies, Lexington, MA, USA, 2 Analysis Group, Inc., Boston, MA, USA, 3 Analysis Group, Inc., Los Angeles, CA, USA
OBJECTIVES: To identify patient characteristics that predict high healthcare utilisers among adult patients with chronic hypoparathyroidism. METHODS: A retrospective cohort study of patients with chronic hypoparathyroidism was conducted using a US claims database (Q1 2007-Q2 2017). Patients who had received parathyroid hormone were excluded. The index date was a randomly selected date of hypoparathyroidism diagnosis ≥6 months after the initial hypoparathyroidism diagnosis to follow patients at various hypoparathyroidism disease duration and stages. A decision tree model and a balanced random forest model were built to identify patient baseline characteristics that may predict the highest 10% of healthcare utilisers in the year following the index date. For both algorithms, the candidate patient characteristics entered into the models included demographics, comorbidities, and treatments during the year before the index date. The area under the receiver operating characteristics curve (AUC) with 10-fold cross-validation was estimated. RESULTS: Among the 4,674 patients included in the study (mean±SD age 59.3±16.18 years; 77% women), 461 represented the highest 10% of healthcare utilisers (age 62.2±14.73 years; 71% women). The decision tree model selected kidney diseases (chronic kidney disease, dialysis, infection), cardiovascular diseases (atrial fibrillation, heart failure, hypertension, tachyarrhythmia), sepsis, respiratory infection, type 1 diabetes, bone fracture, hypocalcemia, cataracts, and gout as patient characteristics predictive of high healthcare utilisers (AUC=0.68). The random forest model ranked patient characteristics predictive of high healthcare utilisation by importance as kidney diseases, cardiovascular diseases (coronary artery disease, hypertension, heart failure, and peripheral vascular disease), sepsis, cerebrovascular disease, diabetes (type 1 and type 2) and bone fracture (AUC=0.71). CONCLUSIONS: Recent studies showed that chronic hypoparathyroidism is associated with increased risk of infections, renal or cardiovascular complications, or diabetes. Improved treatment may reduce risks of these complications predictive of high healthcare utilisation that may lead to high medical care expenditure in the coming year. Funding: Shire, a Takeda company
AI4: GENERAL PAIRWISE COMPARISONS TO SUPPORT QUANTITATIVE MULTIPLE CRITERIA DECISION ANALYSIS
11:45AM - 12:00PM
Chiem JC 1 , Cantagallo E2 , Kicinski M3 , Saad E4 , Buyse M4 , Ciani O5 1 International Drug Development Institute (IDDI), Ottignies, WBR, Belgium, 2 European Organisation for Research and Treatment of Cancer (EORTC), Brussels, Belgium, 3 European Organisation for Research and Treatment of Cancer (EORTC), Brussels, WBR, Belgium, 4 International Drug Development Institute (IDDI), Ottignies-Louvain-la-Neuve, WBR, Belgium, 5 SDA Bocconi University, Milan, MI, Italy
Multiple Criteria Decision Analysis (MCDA) refers to a collection of formal methods which seek to take explicit account of multiple criteria in helping individuals or groups exploring decisions in health-related matters. Quantitative MCDA models involves measurement to determine weights for criteria. Recently, generalized pairwise comparisons (GPC) have been proposed as a flexible statistical method to analyze multiple outcomes simultaneously for the comparison of a treatment group and a control group (e.g. in randomized trials). One specific implementation of GPC requires outcomes to be prioritized, which circumvents the difficulty of defining weights for the outcomes of interest (Buyse M, Stat Med 2010). The outcomes can be of any type (binary, categorical, continuous, or time to event), and can include efficacy, toxicity, quality of life and cost-related data. All pairs of patients are formed by taking one patient from the treatment group and one patient from the control group. Each of these pairs is a “win” if the prioritized outcomes favour the patient in the treatment group, a “loss” if the reverse is true, and a “tie” if the prioritized outcomes favour neither. In this talk we discuss the advantages of GPC to support quantitative MCDA: (1) the number of outcomes is unlimited; (2) the outcomes can be arbitrarily complex; (3) prioritizing outcomes is easier than assigning weights to them; (4) sensitivity analyses can be carried out with different (possibly patient-centric) choices of priorities and (5) the measure of treatment effect called “net benefit” is easy to interpret as the net probability of a better outcome on treatment than on control (Péron et al., JAMA Oncology 2016). We will present examples of benefit/risk analyses in patients with advanced cancer, where combination chemotherapies often improve progression-free survival or overall survival at the cost of severe acute and/or long-lasting toxicities.
PATIENT REPORTED OUTCOMES & QUALITY OF LIFE STUDIES
PR3: HOME-BASED ASSESSMENT OF PATIENT REPORTED OUTCOME MEASURES USING A SMARTPHONE APP PLATFORM- A FEASIBILITY STUDY.
11:30AM - 11:45AM
Badawy S 1 , Alward Z2 , Zolkowski G2 , Barrera L2 1 Lurie Children's Hospital of Chicago / Northwestern University Feinberg School of Medicine, Chicago, IL, USA, 2 Lurie Children's Hospital of Chicago, Chicago, IL, USA
OBJECTIVES : (1) To evaluate the feasibility and acceptability of the assessment of health-related quality of life (HRQOL) at home using smartphones with PROMIS® measures, computerized adaptive testing (CAT) approach, integrated into a sickle cell disease (SCD) app; (2) To examine the effect of the frequency of HRQOL assessments on participants' completion rate over 24-week period, every 2-weeks (Group-A) vs. 4-weeks (Group-B); and (3) To explore participants' experience and preferences with the process and the frequency of HRQOL assessment at home. METHODS : In this pilot randomized trial, participants were randomly assigned to either Group-A (every 2-weeks) or Group-B (every 4-weeks) HRQOL assessment. At enrollment, participants completed demographics and technology comfort questionnaire. At the end of the study, participants completed a semi-structured interview as well as usability and acceptability questionnaires. RESULTS : Forty-two patients participated (57% males, 91% Black, age 15.7±3 years old) with 94% enrollment rate. Overall, HRQOL assessment completion rate was 65% for patients and 47.9% for parents (P =0.13). Completion rates were significantly higher in Group B [every 4-weeks] compared to Group-A [every 2-weeks] among patients only (71.7% vs. 59.3%, P =0.005) and all participants [patients/parents] (65.4% vs. 45.5%, P <0.001), respectively. Similar findings were seen among parents with trend towards significance (Group-B [58.3%] vs. Group-A [37.5%], P =0.09). Participants who completed assessments using iPads had significantly higher completion rates compared to iPhones (100% vs. 45.2%, P <0.001), respectively. Similar findings were seen among participants who installed SCD-app at home compared to those who did so in clinic (83.3% vs. 47%, P <0.001), respectively. Acceptability and usability scores were high among participants (86-100%). CONCLUSIONS : The completion of HRQOL assessments at home using PROMIS®-CAT measures integrated into SCD-app is feasible and acceptable. Completion rates were significantly higher with less frequent HRQOL assessment and using iPads. Future longitudinal studies to integrate routine HRQOL assessments in patients with SCD care are warranted.
PR2: FATIGUE- ITS USE ACROSS TRIALS FOR MULTIPLE CONDITIONS- REGULATORY SUCCESS
11:15AM - 11:30AM
Skaltsa K 1 , Meyers O2 , Sánchez Trullàs K1 1 IQVIA, Barcelona, Spain, 2 IQVIA, Beachwood, OH, USA
OBJECTIVES : Fatigue is of significant concern for people with cancer, but also other diseases, like hypertension, heart failure or depression among others. Fatigue is known to be a multidimensional construct and several instruments are currently available for measuring it and have been used in clinical trials. In the context of new medical product development, it is important to understand under what circumstances regulatory claims for improvement in fatigue have been granted. This review focuses on FDA labels including fatigue data. METHODS : A search in Mapi’s PROLABELS database was performed searching for “fatigue” and filtering for agency “FDA”. Label text from the FDA website was then searched for each record. The drug, indication, instrument, endpoint and threshold used when relevant, were abstracted and summarised. RESULTS : We identified 26 labels from the screening process, out of which 14 were considered as stating a fatigue benefit. Three records on anemia specifically mentioned that the drugs were not demonstrated to improve fatigue, thus were excluded from the review. Indications included acromegaly, influenza, inborn genetic diseases, paroxysmal hemoglobinuria, myelofibrosis, pulmonary hypertension and SCLC. Several instruments were identified: two used the FACIT-fatigue, one used a PROMIS fatigue short form, seven used a study/indication-specific symptom-scale, and four did not report the instrument used. Endpoints also varied, with change from baseline used in five labels, improvement mentioned in five labels, and time to alleviation of symptoms, including fatigue, was used in the three influenza drug labels. CONCLUSIONS : Fatigue is an important symptom relevant to patients in several therapeutic areas. Most of the labels reviewed were approved after the publication of the 2009 FDA PRO Guidance and there is psychometric support for several fatigue-specific instruments. However, PRO efficacy data in labels remains quite limited in terms of the numbers of claims granted and the detail provided in the labels.
PR1: THE BURDEN OF MILD TO MODERATE ATOPIC DERMATITIS IN EUROPE- ANALYSES OF THE NATIONAL HEALTH AND WELLNESS SURVEY
11:00AM - 11:15AM
Neary M 1 , Myers DE2 , Romero WA3 , Gruben D4 , Smith TW5 , Cha A5 1 Pfizer Inc., Princeton, NJ, USA, 2 Pfizer Inc., Collegeville, PA, USA, 3 Pfizer Ltd., Surrey, UK, 4 Pfizer Inc., Groton, CT, USA, 5 Pfizer Inc., New York, NY, USA
OBJECTIVES : Atopic dermatitis (AD) is a relapsing chronic inflammatory skin disease characterized by pruritus/eczematous lesions. The objective was to describe clinical and humanistic burden of mild to moderate (M2M) AD in Europe. METHODS : Data from the 2017 National Health and Wellness Survey, a global cross-sectional self-reported survey, from France, Germany, Italy, Spain, and UK were evaluated. Adults self-reporting AD/eczema (AD/ECZ) were compared with respondents without AD/ECZ using propensity score matching. Demographics and health characteristics were evaluated using Chi-square tests and one-way analysis of variance (ANOVA) for categorical and continuous variables, respectively. Variables which differed between groups (p<0.10) were entered into a logistic regression model to predict AD/ECZ status. Differences across severity (mild, moderate, severe) vs. matched controls, for comorbidities, quality of life, work-related outcomes and psychosocial burden were assessed using Chi-square/ANOVA tests. Similar evaluations were performed using Dermatology Life Quality Index (DLQI) severity bands. Self-reported data provided patient-centered assessments of burden; however, a study limitation was not having clinician-confirmed diagnosis or severity assessment. RESULTS : A total of 4,208 AD/ECZ patients, mean age 45.6, 68.1% female were included. Among 4,039 with self-reported M2M severity, considerable comorbidity burden was observed, most commonly allergies (40.0% mild-48.2% moderate vs. 22.2% control), pain (31.6% -35.1% vs. 22.4%, and allergic rhinitis (26.1%-25.0% vs. 22.6%). Increased burden was consistently observed for: impact/severity of sleep difficulties, Short Form-36 (SF-36) (Mental Component Summary Score, Physical Component Summary Score, 8 domain scores), EuroQOL (EQ-5D) index, work/activity level (absenteeism, activity impairment). Increased frequency of self-reported anxiety (41.4%-45.7% vs. 32.2%) and for moderate/severe depression (23.8%-31.3% vs. 20.6%) was observed. Increased moderate/severe sleep difficulties were also reported (23.1%-34.5% vs. 18.9%). CONCLUSIONS : Clinical and humanistic burden was consistently observed across multiple self-reported assessments in M2M AD respondents in Europe compared to matched controls. This highlights the importance of improving disease management in this population.
PR4: TREATMENT SATISFACTION QUESTIONNAIRE FOR MEDICATION (TSQM - VERSION 1.4)- CEILING AND FLOOR EFFECTS, RELIABILITY AND CONSTRUCT VALIDITY
11:45AM - 12:00PM
Sauer Liberato AC 1 , Landaas E2 , Wongchareon K3 , Rodrigues RCM4 1 University of Freiburg, Freiburg, BW, Germany, 2 University of Washington, Seattle, WA, USA, 3 Naresuan University, Phitsanulok, Thailand, 4 University of Campinas, Campinas, Brazil
OBJECTIVES : Evaluate the acceptability, ceiling and floor effects, reliability and construct validity of the Brazilian version of the Treatment Satisfaction Questionnaire for Medication (TSQM - version 1.4) in patients with hypertension. METHODS : The sample consisted of 300 hypertensive patients with undergoing treatment in an outpatient department. The acceptability was evaluated from the items not answered in TSQM. The reliability of the TSQM was estimated thought Cronbach’s alpha. Construct validity was evaluated by known group analysis to test if TSQM discriminates satisfaction between in different stages of hypertension, diagnoses of Left Ventricular Hypertrophy (LVH), medication adherence and occurrence of side effects and Convergent validity between patient satisfaction and medication adherence measures. RESULTS : TSQM had high acceptability (99% of participants answered all items). A ceiling effect was found in the Side Effect domain (89%), even though this might be a characteristic of the scoring for this subscale. Reliability evaluated with Cronbach’s alphas showed values >0.76 in all domains. Known groups validity was supported with a statistically significant difference in patient satisfaction effectiveness between the hypertension groups and on those without-LVH. Regarding medication adherence, the TSQM was able to discriminate satisfaction in the Side Effect and Global Satisfaction domains. A significantly higher satisfaction was found in all domains of the TSQM on those patients without side effect. Convergent validity was partially supported between TSQM and adherence measures with significant negative correlations of weak magnitude with the Side Effects, Global Satisfaction and Convenience domains. CONCLUSIONS : TSQM is reliable and valid questionnaire to be used in Brazilian hypertensive population.
ISPOR FORUMS
ISPOR STUDENT RESEARCH SPOTLIGHT
This revamp of the Student Research Showcase will allow for increased participation and interaction as it will feature 6 of the top student outcomes research studies submitted for the conference. Attendees of the event will rotate to each presenter and listen to a 3-5 minute elevator pitch of the summary of the research study, the methods, challenges, the so what, conclusions, and the relevance of the research to the conference theme
12:00 - 13:30
LUNCH IN THE POSTER & EXHIBIT HALL
12:00 - 14:00
EXHIBITS & RESEARCH POSTER PRESENTATIONS VIEWING - SESSION 1
12:30 - 13:30
EDUCATIONAL SYMPOSIUM
CAPTURING MULTI-STAKEHOLDER PERSPECTIVES ON ASSESSING THE LONG-TERM CLINICAL BENEFIT OF CANCER IMMUNOTHERAPY
We developed a checklist capturing multi-stakeholder perspectives on assessing the long-term clinical benefit of cancer immunotherapy. The checklist used input from multi-country advisory panels into the key issues to be addressed by both manufactures and payers.
Immunotherapy represents a significant breakthrough in the treatment of cancer across multiple tumor types and patient populations. Immunotherapies differ from traditional cancer therapies as they do not target cancer directly, but instead target the body’s immune system. This changes the mechanism of survival in treated patients, as well as the shape of the survival curve and smoothing estimators of the hazard function. This is the effect known as the ‘plateau’ or ‘tail of the curve’: the flattening of the overall survival Kaplan-Meier curve typically seen after 2 years of follow-up. How best to demonstrate and convey the longer-term health gains of immunotherapies at both regulatory and HTA filing offer is widely contested and impacts access for patients worldwide.
In our research to be presented, an international, multi-stakeholder steering committee was formed comprising 9 experts: payers, economists, and clinicians from the US, UK, France, Italy, and Sweden. Following a review of published literature and technology assessments, we identified key issues and challenges in measuring the long-term clinical benefit specific to cancer immunotherapy. Double-blinded, country-level, multi-stakeholder panels were then convened in the US, UK, France, Germany, and Sweden to refine the key issues and how to address them.
The challenges identified relate to three areas (1) mechanism of action (underlying biology, pseudo progression); (2) limited data at launch (immature overall survival, response, surrogate endpoints); and (3) the analytic methodology (non-proportional hazards and model structure, the plateau in the survival curve, heterogeneity in treatment effect and patient outcomes). To focus evidence generation on addressing those issues systematically, we developed a checklist that manufacturers can use to produce structured, multi-stakeholder evidence that addresses the key challenges for HTA of cancer immunotherapies. Applying a consistent approach to evidence generation, HTA submissions and assessments, will help to improve estimation and consistency, and reduce uncertainty, around the long-term clinical benefit of cancer immunotherapies.
All work relating to the development of the source guidance document and current position paper was funded by Bristol-Myers Squibb. The sponsor was not involved in the analysis or interpretation of the guidance generated.
Sponsor
Bristol-Myers Squibb
Moderators
David Sykes, MSc
PRMA Consulting, Fleet,, United Kingdom
Speakers
Louis P. 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.”
Philip McEwan, PhD
Swansea University, Cardiff, CRF, United Kingdom
Gustav Ullenhag, PhD
Consultant and Associate Professor in Oncology, Uppsala University Hospital, Uppsala, Sweden
12:30 - 13:45
ISPOR FORUMS
ISPOR MEDICAL DEVICES AND DIAGNOSTICS AND PERSONALIZED/PRECISION MEDICINE SPECIAL INTEREST GROUPS- VALUE DEMONSTRATION AND HTA OF NEXT GENERATION DIAGNOSTIC TESTING APPROACHES- CURRENT STATE AND FUTURE NEEDS FOR DRIVING PRECISION MEDICINE E ...
There is an explosion in availability and utilization of next generation testing (NGT) approaches that include multiple genes/genomic biomarkers and require novel value demonstration strategies. NGT approaches include those covering an array of diseases areas and many such tests cover multiple indications in a single assay. Substantial data is being generated through routine clinical NGT use, and potentially available for evaluating real-world clinical utility in health technology assessment (HTA) decision-making. This forum based on a collaborative project of the Medical Devices & Diagnostics and Precision Medicine SIGs aims to outline and debate the following: Key differences between NGT approaches and more ‘traditional’ diagnostics; How these differences challenge NGT value demonstration and HTA; Addressing challenges with clinical and economic value demonstration from the NGT developer/ manufacturer perspective; The critical role of NGT in progress towards health system-wide precision medicine, and related policy issues; NGT assessment challenges from the HTA perspective.
Moderators
Daryl S. Spinner, PhD, MBA
Evidera, Morrisville, NC, USA
Speakers
Eric Faulkner, MPH
Evidera, Bethesda, MD, USA
Joshua Ransom, PhD
AcornAI, a Medidata Company, Boston, MA, USA
Brock Schroeder, PhD
Illumina Inc, San Diego, CA, USA
Uwe Siebert, MPH, MSc, ScD, MD
UMIT - University for Health Sciences, Medical Informatics, and Technology, Hall in Tirol, Austria
Prof. Uwe Siebert, MD, MPH, MSc, ScD, professor of Public Health, Medical Decision Making and Health Technology Assessment (HTA), is the chair of the Department of Public Health, Health Services Research and HTA at UMIT - University for Health Sciences, Medical Informatics and Technology in Austria and the Director of the Division for HTA in the ONCOTYROL – Center for Personalized Cancer Medicine in Austria. He is also adjunct professor of Health Policy and Management at the Harvard T.H. Chan School of Public Health and director of the Program on Cardiovascular Research at the Institute for Technology Assessment and Department of Radiology at the Massachusetts General Hospital, Harvard Medical School, Boston. He is the course director of the Harvard summer course on ‘Decision Analysis in Clinical Research’ and he has an adjunct teaching appointment at the School of Public Health and Epidemiology at the University of Munich.
After medical school, he worked for several years as a physician in international public health projects in West-Africa, Brazil, and Germany. He then earned an MPH at the Munich School of Public Health, and completed an MSc in Epidemiology and a ScD in Health Policy and Management with a concentration in decision sciences at the Harvard School of Public Health. Before he started his faculty position at Harvard Medical School, he was the director of the Bavarian Public Health Research and Coordinating Center at the University of Munich, Germany, and completed Visiting Scholarship at the Harvard Center for Risk Analysis.
His research interests include applying evidence-based quantitative and translational methods from public health, epidemiology, comparative effectiveness research, health services and outcomes research, economic evaluation and decision sciences in the framework of HTA as well as in the clinical context of routine health care and patient guidance. His current substantive research focuses on cancer, cardiovascular disease, diabetes, hepatitis C, neurological disorders, and others. His methodological research includes evaluations of public health interventions, prevention/screening, diagnostic imaging procedures, personalized medicine, causal inference from “big” data, decision modelling, and study designs controlling for treatment switching. He has been leading projects/work packages in several EU FP7 and H2020 projects (e.g., ELSA-GEN, BiomarCaRE, MedTechHTA, EUthyroid, FORECEE, MDS-RIGHT, EUREGIO-EFH). He teaches courses in decision-analytic modeling, HTA, economic evaluation, analysis of big data, and causal inference at several universities and for industry in Europe, USA, South America, and Asia.
Prof. Siebert is the past-president of the Society for Medical Decision Making (SMDM), a member of the Latin America Consortium Advisory Committee of ISPOR, member of the ‘National HTA Strategy’ Expert Group of the Austrian Federal Ministry of Health, member of the Oncology Advisory Council of the Federal Ministry of Health in Austria, member of the Advisory Board of the GÖG - National Austrian Public Health Institute, member of the Austrian Cochrane Collaboration Branch, and a member of several national and international Directors Boards (Society for Medical Decision Making; Austrian Society of Epidemiology, German Network of EbM; German Association for Medical Informatics, Biometry and Epidemiology). He has served as vice president of SMDM, and a member of the ISPOR Board of Directors, the Directors Board of the German Competence Network Heart Failure, the International Expert Committee Advising the Institute for Quality and Efficiency in Health Care (IQWiG) on the Methods for Economic Evaluations of Health Care Interventions, the Harvard Flagship Initiative in Comparative Effectiveness Research, the Extended Board of Directors of the German Association of Health Economics, and the Advisory Board of the Ludwig Boltzmann Institute for HTA in Austria.
He is the Clinical Guideline Commissioner for the Association of the Scientific Medical Societies in Germany (AWMF) and the chairman of the Working Groups ‘Health Economics’ and ‘Medical Decision Making’ of the German Society for Medical Informatics, Biometry and Epidemiology (GMDS). He is co-chair of the ISPOR-SMDM Modeling Good Research Practices Task Force, co-chair of the ‘Issues in Methodology Section’ of the SMDM Policy Initiative, and a leadership member of the ISPOR Personalized/Precision Medicine Special Interest Group.
He has worked with several HTA Agencies (eg, DAHTA@DIMDI/Germany, IQWiG/Germany, NICE/UK, ANVISA/Brazil, CADTH/Canada, LBI-HTA/Austria, GÖG/Austria) and he advises government agencies, academic institutions and industry regarding the conduction and impact of health technology assessments on policy and reimbursement decisions. He has authored more than 350 publications including HTA reports, textbook chapters, scientific articles, and editorials, and is Editor of the European Journal of Epidemiology, as well as editorial board member of several scientific journals.
ISPOR HEALTH SCIENCE POLICY COUNCIL'S TASK FORCE REVIEW COMMITTEE- YOU, TOO, CAN INITIATE AND DEVELOP AN ISPOR GOOD PRACTICES TASK FORCE - THE 'HOW TO' GUIDE TO NAVIGATING THE PROCESS
ISPOR’s Good Practices Task Force Reports are highly cited, multi-stakeholder perspective, expert consensus guidance reports that set international standards for HEOR and its use in healthcare decision making. Despite the prominence and relevance of these task force reports to the HEOR field, many at ISPOR are unfamiliar with them. Our goal is to explain task force proposal development, evaluation, recommendation, and final approval by the ISPOR Board of Directors. In addition, the steps to initiate the task force and the consensus review process that the report undergoes will be described. Finally, speakers will answer questions and share their experiences participating in task forces and leading an ISPOR Good Practices Task Force. We hope to encourage participation in and proposal submissions for task forces from a diverse range of experts in and outside ISPOR. Any active interested and knowledgeable ISPOR member can participate as a task force report reviewer.
Moderators
Deborah Marshall, PhD
University of Calgary, Calgary, AB, Canada
Deborah Marshall, PhD is a Professor in the Department of Community Health Sciences, University of Calgary, Arthur J.E. Child Chair in Rheumatology Outcomes Research and former Canada Research Chair, Health Services and Systems Research. Her research program focuses on the measurement of preferences, cost-effectiveness analysis, and simulation modeling of health services and interventions. Deborah has over 20 years of research experience in health technology assessment agencies, academic institutions, and industry settings in Canada, US, and Europe. She is a founding co-investigator of the Patient and Community Engagement Research (PaCER) Program at the University of Calgary co-leads the economics and stated preferences research platforms for the Canada-Netherlands Personalized Medicine Network in Childhood Arthritis and Rheumatic Disease (UCAN CANDU). Deborah is an active member of the ISPOR as the past-President of the Board of Directors, and as a member of the Patient Preferences Special Interest Group. She is a co-author on the three ISPOR Task Force Reports for Good Research Practice – Checklist for Conjoint Analysis in Health, Conjoint Analysis Experimental Design and Statistical Methods for the Analysis of Discrete-Choice Experiments. She also chaired the two reports from the ISPOR Dynamic Simulation Modeling Application in Health Care Delivery Research Emerging Good Practices Task Force.
Speakers
C. Daniel Mullins, PhD
University of Maryland, School of Pharmacy, Baltimore, MD, USA
C. Daniel Mullins, PhD is a professor and chair of the Pharmaceutical Health Services Research Department at the University of Maryland School of Pharmacy. He is founder and executive director of the University of Maryland PATient-centered Involvement in Evaluating effectiveNess of TreatmentS (PATIENTS) Program, an infrastructure to support patient-centered outcomes research and related training activities. He also serves as director of the Community and Collaboration Core within the university’s Institute for Clinical and Translational Research. Dr. Mullins has received funding as a principal investigator from the NIH/NIA, NIH/NHLBI, AHRQ, the Patient-Centered Outcomes Research Institute (PCORI), and various patient advocacy and industry organizations. He has served as a regular member of AHRQ and NCI Study Sections and has chaired PCORI Study Sections. Professor Mullins is one of two editors-in-chief for Value in Health and is author/co-author of more than 225 peer-reviewed articles. He has received an Outstanding Service Award from the Drug Information Association (DIA) and two Service Awards from ISPOR. In 2007, he received the Dr. Patricia Sokolove Outstanding Mentor Award from the University of Maryland Baltimore campus-wide Graduate Student Association. In 2013, he was the recipient of the Dr. Daniel D. Savage Memorial Science Award, the Association of Black Cardiologists’ most prestigious annual award. Also in 2013, he was awarded a University System of Maryland Wilson H. Elkins Professorship. He was named Researcher of the Year in 2014 and received the Martin Luther King Faculty Diversity Award in 2017 for the University of Maryland Baltimore campus and the ISPOR Marilyn Dix Smith Leadership Award.
Praveen Thokala, MASc, PhD
School of Health and Related Research (ScHARR), The University of Sheffield, SHEFFIELD, United Kingdom
Praveen Thokala joined the School of Health and Related Research (ScHARR) at the University of Sheffield after completing an MASc from the University of Toronto and a PhD from the University of Southampton.
During this time, he has completed several health economics projects including single technology appraisals (STA), multiple technology appraisals (MTA), and diagnostic assessment reports (DARs) for NICE. He has also recently led a modeling project for Institute for Clinical and Economic Review (ICER) in the US. His research interests include health economic modeling, multi-criteria decision analysis (MCDA), discrete event simulation modeling, and optimization. He co-supervised four PhD students to completion and currently co-supervises four PhD students.
In terms of MCDA, he has worked with the National Institute of Health and Care Excellence decision support unit (NICE DSU) in the UK on exploring the applicability of MCDA in HTA. He co-chaired the ISPOR Task Force on the use of MCDA in healthcare decision making and has been involved in several MCDA studies including supporting priority setting and benefit-risk analysis. He also co-edited a book titled, Multi-Criteria Decision Analysis to Support Healthcare Decisions.
ISPOR CLINICAL OUTCOME ASSESSMENT SPECIAL INTEREST GROUP - NEW FRONTIERS- VALUING COA DATA AND GUIDING PRINCIPLES FOR RWE
The Clinical Outcomes Assessment (COA) Special Interest Group will update ISPOR membership on its goals, aims and progress of the group’s first initiatives with EU ISPOR attendees.
The two initiatives of the group include: a key project focusing on the value of COA data to external stakeholders, and a membership engagement project investigating approaches to standardizing COA for real world (RW) studies. The key project aims to update the terminology used by COA specialists and to publish this in the updated ISPOR book of terms. The work will involve the review of HTA terminology used for COA from different countries and to harmonize the way HEOR scientists and COA specialists describe COA value. The COA SIG membership engagement project will explore the standardization of COA in RW studies. A virtual roundtable meeting will be conducted with industry colleagues to discuss topics, such as the operationalization and scientific integration of COA in in the RW.
We will discuss the available results and the next phases of our two initiatives during this forum.
Moderators
Katja Rudell, PhD
Parexel International, Chittering, United Kingdom
Speakers
Bryan Bennett, PhD
Bristol-Myers Squibb Pharmaceuticals Ltd, Uxbridge, United Kingdom
Lynda Doward, Master of Research
RTI-Health Solutions, Manchester, United Kingdom
Ana Maria Rodriguez, PhD MSc BScPT BSc
IQVIA, Madrid, Spain
Angela Jane Rylands, PhD, CPsychol, BSc
pH Associates (an OPEN Health company), Marlow, United Kingdom
ISPOR REAL-WORLD EVIDENCE SPECIAL INTEREST GROUP- INITIATIVES, FEEDBACK, AND HOW TO PARTICIPATE
Increasingly healthcare decision makers are simultaneously looking for efficiency and a more comprehensive evidence base. Real-world evidence (RWE) is the spotlight as a viable solution to these challenges. We are forming a new RWE SIG dedicated to increasing awareness, understanding, rigor and utilization of RWE as an important part of the wider evidence base for healthcare decision making. In order to be successful, ISPOR needs you, its members, to support this initiative. This open SIG meeting will present a range of RWE-specific topics to ascertain which concerns require immediate action, whether ISPOR is best placed to contribute to the solution and to start defining how we collectively might address them. During the meeting, attendees will have the opportunity to contribute their perspectives, suggest alternative topics and volunteer to help advance the future SIG activities, including an initiative on research transparency and developing definitions for the ISPOR revised book of terms.
Speaker
Suzanne Belinson, PhD, MPH
Tempus, Inc., Chicago, IL, USA
Peter Knox, MPP
The Life Raft Group, Wayne, NJ, USA
Peter is responsible for oversight of all research activities at the Life Raft Group (LRG), a non-profit that has been advocating for a rare form of cancer (Gastrointestinal Stromal Tumor, or GIST) for nearly 20 years. In particular, Peter and the LRG have been involved in the production and analysis of Real World Data (RWD) and Real World Evidence (RWE) through a patient registry, web-based side effect management platform, and other data products aimed at both patients and clinicians/researchers, and possess significant experience working with various HEOR professionals in the pharmaceutical industry. Prior to working in patient advocacy, Peter worked for various technology and web firms where he gained valuable knowledge on both the user perspective and how technology can be applied to the current digital health paradigm. A holder of a Masters in Public Policy with a concentration in quantitative methods, economics, and health policy from Rutgers University, Peter is especially proud to be working with ISPOR’s newly formed RWE special interest group, where he will enthusiastically offer his knowledge of the patient and caregiver perspective.
Elisabeth Oehrlein, PhD, MS
National Health Council, Washington, DC, USA
Maartje Smulders, MS MPH
Kubadili, Severna Park, MD, USA
ISPOR CENTRAL & EASTERN EUROPE CONSORTIUM- PROBLEMS AND SOLUTIONS FOR IMPLEMENTATION OF RISK SHARING SCHEMES IN THE COUNTRIES OF CENTRAL AND EASTERN EUROPE
Risk-sharing mechanisms have been known and used for many years in developed countries, but it is still very difficult at the initial stage of concluding an agreement to foresee all the “pitfalls” that may arise in the process of its implementation both on the payer and in the manufacturer sides. However, the payer's desire to provide patients with innovative drugs, as well as the manufacturer’s desire to sell its drugs, stimulates the introduction of risk-sharing schemes and is especially important in countries with limited resources. Therefore, it is interesting to understand what kind of “pitfalls” appear in the countries of Eastern and Central Europe and how they manage to develop their own approach to the implementation of risk sharing agreements in their current regulatory framework and cultural environment.
Moderators
Tatiana Kurnosova, MSc
State Budgetary Institution, Research Institute for Healthcare Organization and Medical Management of Moscow Healthcare Departme, Moscow, MOW, Russia
Speakers
Malwina Holownia-Voloskova, MPharm, PhD(cand.)
SBI Research Institute for Healthcare Organization and Medical Managment of Moscow Healthcare Department, Moscow, MOW, Russian Federation
Zoltan Kalo, PhD
Semmelweis University and Syreon Research Institute, Budapest, 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.
Roman Topór-Mądry, Dr
Agency for Health Technology Assessment and Tariff System, Warsaw, Poland
ISPOR WOMEN IN HEALTH ECONOMICS AND OUTCOMES RESEARCH INITIATIVE- UNLEASHING THE LEADER WITHIN YOU
The vision of the ISPOR Women in Health Economics and Outcomes Research (HEOR) Initiative is to support the growth, development, and contribution of women in HEOR; to serve as a catalyst for women’s leadership in the field; and to offer a platform for ISPOR women to collaborate, network, share, and mentor each other. Please join us in this session as keynote speaker, Sabine Hutchison of Seuss Consulting provides strategies on how to embrace your inner leader and enhance your inherent leadership skills and growth opportunities. Leaders of this initiative will outline the initiative’s work to date and provide the opportunity for networking with colleagues who seek to advance women’s leadership in HEOR. This session is open to all attendees (women and men) interested in the advancement of women in the field of HEOR.
Moderators
Shelby D Reed, PhD, RPh
Duke University, 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.
Speakers
Sabine Hutchison, BA
Seuss+, Hamburg, NC, Germany
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.
13:00 - 14:00
POSTER AUTHOR DISCUSSION HOUR - SESSION 1
Attendees have the opportunity to interact with poster presenters during this hour-long session.
ISPOR BOOTH EVENT - CAREER CENTER - MAXIMIZE YOUR ONLINE EXPERIENCE
ISPOR Career Center—Maximize Your Online Experience
14:15 - 15:15
BREAKOUT SESSION 2
REIMBURSEMENT & ACCESS POLICY RESEARCH
RE4: IS MARKET ACCESS FOR ONCOLOGY TREATMENTS EASIER IN ENGLAND THAN FOR NON-ONCOLOGY TREATMENTS? AN ANALYSIS OF NICE SINGLE TECHNOLOGY APPRAISALS FROM 2017-2019
3:00PM - 3:15PM
Chunara F 1 , Wilson C1 , Foxon G2 , Craddy P3 1 Remap Consulting, Alderley Edge, CHE, UK, 2 Remap Consulting, Macclesfield, CHE, UK, 3 Remap Consulting, Zug, Switzerland
OBJECTIVES : The National Institute of Health and Care Excellence (NICE) undertakes clinical and cost-effectiveness assessments of pharmaceutical products. Products assessed by NICE can be either recommended, recommended with restrictions (i.e. optimised) or not recommended. Oncology products may also be recommended for use within the cancer drugs fund (CDF). This study compares the outcomes for oncology and non-oncology appraisals conducted by NICE. METHODS : NICE single technology appraisals (STAs) published between January 2017 and January 2019 were identified. For each appraisal, outcomes of draft appraisal consultation documents (ACDs) and final appraisal determinations (FADs) were extracted. Outcomes of oncology and non-oncology appraisals were then analysed. RESULTS : A total of 98 appraisals were identified, of which 66% and 34% were for oncology and non-oncology indications, respectively. ACDs were published for 74% of oncology appraisals and 48% of non-oncology appraisals. Nearly all oncology ACDs were “not recommended” (96%), however non-oncology ACDs were either “not recommended” (56%) or recommended with restrictions (44%). Nevertheless, at FAD, a similar proportion of oncology and non-oncology appraisals received positive recommendations (89% vs. 88%, respectively). However, a greater proportion of non-oncology indications than oncology indications were optimised (55% vs. 26%). Of the positive oncology recommendations, 32% were recommended for use within the CDF. In terms of financial agreements, 69% of positive non-oncology appraisals were subject to patient access schemes (PAS) or complex arrangements whilst 31% had no financial agreement. In contrast, all but one (2%) of positive oncology appraisals were subject to a financial agreement. CONCLUSIONS : Overall, oncology and non-oncology appraisals receive similar rates of positive recommendations. However, whilst positive oncology recommendations generally apply for all eligible patients, non-oncology appraisals are more likely to be recommended for only a subset of the labelled population. Nevertheless, although oncology appraisals are achieving greater access, they are more likely to be subject to financial arrangements.
RE1: REIMBURSEMENT OF INNOVATIONS IN GERMAN HOSPITALS - FIRST ANALYSIS OF HOSPITAL CLAIMS DATA ON THE DEVELOPMENT OF NEW EXAMINATION AND TREATMENT METHODS
2:15PM - 2:30PM
Krumm A 1 , Irps S2 , Jaeger C3 , Kersting T4 , Berghoefer A5 1 Charité - Universitätsmedizin Berlin, Berlin, BE, Germany, 2 IMC clinicon GmbH, Berlin, Germany, 3 German Hospital Federation, Berlin, Germany, 4 Technische Universität Berlin, Berlin, Germany, 5 Charité - Universitätsmedizin Berlin, Berlin, Germany
OBJECTIVES: Medical innovations are usually not covered by the German Diagnosis Related Group (DRG) system. In order to address this issue an application system for so called new examination and treatment methods (NUB) has been implemented. Hospitals annually submit NUB specific data to the Institute for the Hospital Remuneration (InEK) to explain why the current DRG remuneration is insufficient. Based on a positive decision by the InEK, an individual fee can be negotiated between hospitals and the statutory health insurances (SHI). Negotiation results are confidential and data is only partly publicly available. This study represents the first analysis of real world data on NUB remuneration. METHODS: Hospital claims data were used to detect all individual fees for NUB billed to SHI between 2013 and 2017 and matched with the annually published InEK database. An analysis of negotiated fees concerning level, quantity and revenue was executed. Analysis also includes a differentiation on the type of NUB (pharmaceuticals, medical devices, medical procedures). RESULTS: In total, applications for 1,082 NUB were filed to InEK between 2013 and 2017. Individual fees were ultimately billed only for 207 (19.1%) NUB. Of these, 54.1% accounted for pharmaceuticals, 37.2% for medical devices and 8.7% for medical procedures. In 78.4% of all NUB the invoice was based on a positive assessment (status 1) by the InEK. In contrast 38 NUB were invoiced to the SHI based on other legal claims. The total NUB based revenue increased between 2013 and 2017 from 73.3 million to 233.5 million euros. This translates to a 0.2% increased share of the annual SHI expenditures for inpatient services. CONCLUSIONS: Data indicates that just for a low number of NUB individual fees are negotiated and billed to the SHI. Due to the high and rising fees SHI expenditures for NUB increased in the period under review.
RE2: THEMES IN THE DESIGN OF PHARMACEUTICAL VALUE-BASED CONTRACTS IN THE UNITED STATES AND EUROPE
2:30PM - 2:45PM
Adimadhyam S1 , Weckstein A2 , Patrick A3 , Mattox P1 , Jaksa A 4 1 Aetion, Inc, Boston, MA, USA, 2 Aetion, Inc, BOSTON, MA, USA, 3 Aetion, Boston, MA, USA, 4 Aetion, Inc., Boston, MA, USA
OBJECTIVES: To evaluate themes in contract structures of value-based contracts (VBC) for pharmaceutical products across the US and Europe. METHODS: VBCs in the US and select European nations (UK, Italy, Netherlands, and Spain) were extracted from the University of Washington Performance-Based Risk Sharing Agreement database (1997-2019). Contracts were categorized by the terms of reimbursement (rebate on product, reimbursement for disease sequelae, or coverage with evidence development), and endpoints (clinical, economic, or humanistic (i.e., QALY) with or without patient compliance). Preliminary results for the US and UK are reported. RESULTS: There were 40 US-based and 21 UK-based VBCs. Most common therapeutic areas for US-based VBCs were endocrinology (32.5%), cardiology (25%), and oncology (20%). The majority of UK-based VBCs were in oncology (48%) and neurology (24%). The majority of US-based contracts were rebates (88%), whereas the majority of UK-based contracts were coverage with evidence development (52%). Outcomes-based assessments were stipulated in the majority of US-based contracts (90%), but not in the UK (42%). Of these, the most frequently utilized endpoints were clinical in the US (62.5%) and both clinical and humanistic (20%, respectively) in the UK. Humanistic outcomes were not mentioned in US-based contracts. 25% of US-based contracts also stipulated components of patient compliance, compared to 1 UK-based contract. CONCLUSIONS: In an environment of rising prices, VBCs are becoming a cost containment strategy. However to date, only 61 VBCs were noted in the US and UK over 2 decades, which could be driven by lack of contracts and/or transparency in reporting. The lack of contracts could be driven by the challenges of data collection and analysis, which will likely be mitigated by the development of RWE methods for VBCs. More research and transparency is needed on the structure and successes of VBCs and how RWE can improve VBCs.
RE3: A PROPOSED, SCALABLE APPROACH TO IMPLEMENTING INNOVATIVE PAYMENT AGREEMENTS
2:45PM - 3:00PM
Woolmore A 1 , Belcher MD2 , Chin-A-Young L2 , Drage EP2 , Wiinberg L2 , Ogley RJ2 1 IQVIA, Courbevoie, France, 2 IQVIA, London, UK
OBJECTIVES: Interest in Innovative Payment Agreements (IPAs) that link product value or risk sharing to real-world practice e.g. by indication or Duration of Therapy (DoT) is increasing. Despite this and the existence of many real-world data (RWD) sources, no standard methodology for designing and implementing IPAs using RWD currently exists. The European Oncology Data Network (ODN), established by the Collaboration for Oncology Data in Europe (CODE), led by IQVIA, is a collaborative near-real time RWD sharing platform designed to provide information on anti-cancer therapy usage and support the negotiation and implementation of IPAs. In parallel to ODN development and in anticipation of its use supporting IPAs, this study aimed to describe a scalable approach to the design and implementation of IPAs using RWD. METHODS: A simulated RWD set was prepared based on the ODN dataset, cleaned, and used to model two agreements: DoT and Indication Based Payments (IBP). Working with CODE members, nine steps were identified to design, measure, forecast and reconcile agreements. National and sub-national surveillance cohorts (50%, 25%, 12% of total population) tested the variation in agreements’ outcomes. RESULTS: Complexities of using existing RWD sources not designed to support IPAs were revealed, including analytical choices (e.g. preparing and cleaning the data), evolving interpretation of the data (e.g. variations in DoT or indication over time) and requirements for measuring contextual variables (e.g. volume, new patients or indications) throughout the agreement. Variations based on surveillance cohort size, were quantified in the context of the risk associated with RWD measurement vs reliability of forecasting the future performance of the agreement. CONCLUSIONS : This study highlighted the challenges inherent in using RWD for IPAs and proposed an approach to address them. It reinforced the need and benefit of using a ‘tailormade’ infrastructure with highly structured, near-real time data to develop and monitor IPAs.
TIME FOR CHANGE? HAS THE TIME COME FOR THE PHARMA INDUSTRY TO ACCEPT MODEST PRICES?
ISSUE
: Payer pushback on the prices of new therapies is impacting companies’ bottom lines, their reputation and patient access. Drug companies have had to make drastic cuts to the prices of novel therapies in Europe and the U.S. for important indications -- and others that have failed to secure coverage in important European countries. Pricing pressures are also weighing on the quarterly earnings results. Has the time come for the pharma industry to accept more modest prices in order to deliver long-term growth? Or would this infuriate investors who want high returns for high-risk investments? Balancing the needs of patients, healthcare systems, drugmakers and investors has never been trickier, but failure to do so could have disastrous consequences for everyone
OVERVIEW
: This panel will present contrasting views on how to ensure the pharma industry delivers maximum return on investment for investors without compromising patient access. Grueger will argue that the benefits of a medicine should dictate the price of a medicine and failure to capture this will result in drugmakers leaving money on the table that could be reinvested in future R&D activity. Schroeter will argue the old model of banking on innovation to drive sky-high prices has expired. He will make the case that payer pushback is leading to access restrictions that are hitting both the top- and bottom-line of companies. Schroeter’s solution: a holistic access strategy focused on getting the price equation right from the get-go. Garner will look at the question of sustainability from the public health perspective and will propose fresh ideas on how companies can reap the rewards for their innovations and still make sure patients benefit from them. Moderated discussion (Dion) will explore these views and conclude the panel with a clear overview of actionable outcomes.
Moderators
Kate Dion, MA
3D Communications, Dollar, United Kingdom
Panelists
Sarah Garner, PhD
World Health Organization (WHO), Copenhagen, Denmark
Jens Grueger, PhD
F. Hoffmann-LaRoche, Division Pharma, Basel, Switzerland
Michael Schröter, PhD
Viopas Partners, Zürich, Switzerland
SPOTLIGHT SESSION 1
TRANSPARENCY IN RWE - TIME FOR A UNIFIED APPROACH
This session will provide an overview of the history and goals of the RWE Transparency Initiative and why it is timely, as well as its work to date, and the key points of its recent white paper. It will cover fundamental aspects of this issue including: potential registration sites, issues of credibility and transparency, characteristics of studies recommended for registration (ie, hypothesis-evaluating treatment effectiveness studies), and the importance of reporting for replicability and totality of evidence considerations.
Moderators
Brigitta Monz, MD, MPH, MA
Roche, Freiburg, Germany
Panelists
Wim Goettsch, PhD
The National Healthcare Institute (ZIN), Utrecht University, Diemen, Netherlands
Sebastian Schneeweiss, MD, ScD
Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA
Massoud Toussi, MD, PhD, MBA
IQVIA, Courbevoie, France
BREAKOUT SESSION 2
PRO DATA ARE RELEVANT ENDPOINTS FOR HTA ASSESSMENT- CUI BONO?
PURPOSE: We will share a variety of examples on how Patient-Reported Outcomes (PROs) are included in the HTA submission and are evaluated by regulators, to better understand the current place of PROs in the HTA decision process in Europe.
DESCRIPTION: EunetHTA guidelines recently acknowledge the central value of Patient-Reported Outcomes (PROs) as patient-relevant endpoints in the HTA process. Moreover high level representatives of HTA Agencies in Europe do publicly request PRO data in dossiers submitted. However, from the industry perspective, the real place of PRO in defining the value and the added value of a new health product in the HTA decision process seems still disappointing and its consideration remains heterogeneous across the different European HTA agencies.
We will discuss about the 1/ potential reasons about this discrepancy of expectation between industry and regulators and we will present 2/ variety of real dossier’ submissions to HTA Agencies in Europe to better understand how PRO data were submitted by applicants and how (if) they were reviewed by regulators. Finally, in order to eventually reconcile the different perspectives (i.e. industry vs. regulators), we will identify approaches for improvements in the measurement of PRO (how to best capture patient experience with high quality especially when measured in real-life and/or observational studies), in order 1/ to achieve high quality studies and clinical study reports incorporating PRO assessment which in turn may mitigate the hesitancy of some HTA regulators to base part of their decision on these endpoints and 2/ to tend to a better harmonization of review by the different HTA agencies. There will be polling asking the audience their beliefs and expectations about the place of PRO in HTA process, approaches to improve the quality of PRO data submitted and its review by regulators, and an opportunity for questions at the end.
Discussion Leader
Olivier Chassany, MD, PhD
Patient-Centered Outcomes Research, University Paris-Diderot, Paris, France
Lynda Doward, Master of Research
RTI-Health Solutions, Manchester, United Kingdom
Katarina Halling, MSc
AstraZeneca, Molndal, Sweden
Niklas Hedberg, MSc, Pharmacy
The Dental and Pharmaceutical Benefits Agency (TLV), Stockholm, Sweden
DEMYSTIFYING MACHINE LEARNING- WHY SHOULD WE BE OPEN TO IT?
PURPOSE: Machine learning is gaining in momentum among stakeholders in the healthcare ecosystem with the advent of Big Data and new possibilities for evidence generation to support patient access to novel therapeutics. However, machine learning remains a mysterious term for many stakeholders. The workshop will describe common machine learning methodologies and will focus on real applications of these techniques in the health economics and outcomes research (HEOR) area.
DESCRIPTION: ‘Machine learning’ is a generic term to describe algorithms and methodologies, which includes cluster analysis, decision trees, artificial neural networks, Bayesian networks, and others. These techniques, while used before in other fields, are becoming relevant in healthcare with the influx of new types and/or volume of data, as well as the new possibilities to consolidate datasets from several origins.
Helene Karcher will start by presenting the principles of machine learning, the main techniques, and the data needs for a few staple techniques with examples. Christoph Gerlinger will outline how machine learning techniques, ie, cluster analysis, time series analysis, and random forests, were used to develop a patient-centered prediction model for the menstrual bleeding for women who start a new hormonal intrauterine device for contraception. Gorana Capkun will discuss the use of machine learning for patient identification, its role in optimizing chart reviews and improving referrals for multispecialty diseases with difficult diagnostic patterns and no existing therapy. Jackie Vanderpuye-Orgle will present applications and case studies of machine learning in oncology and pulmonary hypertension for patient identification, clinical decision support, and prognostic tools. Finally, the presenters will discuss with the audience the proper place of machine learning techniques in the HEOR area.
Discussion Leader
Gorana Capkun, PhD
Novartis Pharma, Basel, Switzerland
Christoph Gerlinger, PhD
Bayer AG, Berlin, Germany
Helene Karcher, PhD
Parexel Regulatory and Access Consulting, London, United Kingdom
Jacqueline Vanderpuye-Orgle, PhD
Parexel, Glendale, CA, USA
14:30 - 15:00
ISPOR 2019 EXHIBITOR HEOR THEATER
Economic Modelling Considerations for Rare Diseases
Presenters:
Chris Knight , MSc, Senior Director in Health Economics, RTI Health Solutions
Isobel Pearson , DPhil, Director in Health Economics, RTI Health Solutions
When seeking orphan drug approval, demonstrating value for therapies can be a challenge. The limited amount of data available affects the feasibility of developing models that meet good practice recommendations for health technology assessment submissions. We will discuss different approaches for developing a robust model structure that can help you with orphan drug reimbursement.
Sponsored by RTI Health Solutions
The HEOR Theater presentations are not an official educational offering of ISPOR Europe 2019 and are not sponsored, endorsed or accredited by ISPOR.
14:30 - 15:30
ISPOR FORUMS
ISPOR NEW PROFESSIONAL EVENT - CAREER ADVICE ACROSS THE GLOBE- "UNFORTUNATELY THIS CONTENT IS UNAVAILABLE IN YOUR REGION- WHAT IS THE IMPACT OF GENERAL DATA PROTECTION REGULATION (GDPR) ON THE HEOR COMMUNITY"?
The New Professional Event, Career Advice Across the Globe, provides both New Professional and soon-to-graduate Student members with the opportunity to hear first-hand experiences from established ISPOR members around the world on various career related topics.
Moderators
Elisabeth Oehrlein, PhD, MS
National Health Council, Washington, DC, USA
Speakers
Carl V. Asche, PhD
University of Illinois College of Medicine at Peoria, Peoria, IL, USA
Jacco Keja, PhD
IQVIA, London, United Kingdom
15:30 - 19:00
RESEARCH POSTER SESSION 2
15:45 - 16:45
SPOTLIGHT SESSION 2
GENOMICS IN THE FUTURE OF HEALTH – THE ROAD TO BETTER OUTCOMES?
The accelerating development in digital possibilities opens for better understanding of our biology where the progress in the field of genomics (and multiomics) has opened for new insight and understanding of health and disease. Personalized health is built on genomics and is seen as a necessary change for future health systems. At the same time, a host of questions need to be addressed to ensure that the new knowledge and tools bring about better outcomes. This session will bring together the two worlds of HEOR and clinical genomics to explore how implementation of these transformational technologies can be better guided by evidence. The current achievements of clinical genomics and approaches to assessing the costs and benefits of these emerging technologies will be reviewed followed by discussion on how health economics and outcomes research can be better used to influence how genomics is rolled out in the clinic.
Moderators
Bogi Eliasen
Copenhagen Institute for Futures Studies, Copenhagen, Denmark
Bogi Eliasen is a knowledge broker whose expertise lies in combining various fields of knowledge. He initiated the first population genome project, FarGen 2009 and works with bridging digital health and genomics in the context of the future of personalized health for which he in 2019 received the HIMSS Global Achievement Award.
Steven McAdam
DNV-GL, Oslo, Norway
Panelists
Lars-Åke Levin, PhD
Linköping University, Linköping, Sweden
Rajji Mehdwan
Roche, Amcham, Oslo, Norway
Rajji Mehdwan is General Manager for Roche Pharmaceuticals in Norway. Rajji is passionate about the next era of personalized medicine and advances we can make to provide even better and targeted healthcare to Norwegian patients. She is excited about the nexus of technology and science that will/is transforming how we deliver and experience healthcare and believes meaningful health data at scale will be key pillar of making personalized medicine a clinical reality in everyday life of patients. Rajji has taken concrete steps to invest in personalized approaches in Norway leveraging Roches strong expertise in Genomics, Diagnostics, Medicines and Health Data. She is an advocate for public private partnerships and is looking forward to meeting and learning from other colleagues at the event to generate ideas on how we can ‘Co-Create’ to benefit our communities in Norway & globally.
Rajji joined Roche / Genentech in 2010 and has held various leadership roles in US, Switzerland and Denmark prior to Norway. Prior to Roche Rajji has worked at Johnson & Johnson, Management Consulting and a Healthcare start up. Rajji holds a M.B.A from the Wharton School of Business, University of Pennsylvania and is a Registered Nurse having trained at the Royal Free Hospital NHS Trust in England
Kjetil Tasken
Oslo University Hospital, University of Oslo, Oslo, Norway
Kjetil Taskén (born 1965) received his M.D. in 1991 and did his Ph.D. in molecular and cell biology (1994) at the University of Oslo (UiO). He was appointed Professor of Medicine in 2001 and serves as Head of the Institute of Cancer Research, Oslo University Hospital (OUH) since 2018. He has been the national Director for Norway of the ESFRI infrastructures EU-OPENSCREEN (academic chemical biology and screening) and EATRIS (translational medicine) and in charge of the corresponding national infrastructures. He is a partner in the K.G. Jebsen Centres for Cancer Immunotherapy and B cell malignancies. He serves on several evaluation panels, SABs and Editorial Boards, including the IMI Scientific Committee, ERC Panel and CRUK New agents committee.
Taskén received the Anders Jahre Medical Prize for younger scientists in 2002 (Nordic award), and won the King Olav V’s Prize for Cancer Research (national life-achievement award by the Norwegian Cancer Society) in 2016. He was elected to the Norwegian Academy of Science and Letters in 2005. Taskén is author of more than 275 scientific publications and inventor of more than 20 patents. He is cited over 12,000 times with an h-index of 58.
Terry Vrijenhoek, PhD
University Medical Center Utrecht, Utrecht, Netherlands
L’ADN fait n’importe quoi (DNA does whatever). That was the opening statement of the thesis of Terry Vrijenhoek. It refers both to the unpredicatble way in which our DNA acts, and to the unlimited possibilities it has for research, diagnostics and care. Terry is the Faculty and Staff Advisor of the Department of Genetics at UMC Utrecht, the Netherlands. As such, he prepare the management team and staff members for future developments in clinical genetics. At the same time he involves a wide variety of stakeholders in his mission to develop a strategy for the ‘Genetics Clinic of the Future’.
Terry obtained his Ph.D. on the genetic background of schizophrenia at the Radboud University Nijmegen Medical Centre (RUNMC), the Netherlands. He obtained his M.Sc. in Animal Genetics at Wageningen University and Research Centre (WUR).
Terry’s career has been entirely devoted to bringing genetics as close to society as possible. He has been largely involved in networking and career development for young scientists. As chairman of the Genomics Network for Young Scientists (GeNeYouS) he has contributed to the development of a platform for young life scientists. He has been involved in many public engagement activities, and has experimented with many tools and settings to provide applications of genetics in society. Together with 15 other young scientists he now co-authors SciencePalooza, a popular science blog which is also regularly published in other media, such as newspapers and popular science journals.
BREAKOUT SESSION 3
NEW FRONTIERS IN ONCOLOGY COST STUDIES
ON1: HEALTHCARE RESOURCE UTILIZATION AMONG PATIENTS RECEIVING TREATMENT FOR CHRONIC LYMPHOCYTIC LEUKEMIA- REAL-WORLD DATA FROM A LARGE HEALTHCARE PROVIDER
3:45PM - 4:00PM
Weil C 1 , Chodick G2 , Shalev V2 , Kan I3 , Afik R3 , Cohen R3 , Sail K4 1 Maccabi Healthcare Services, Tel Aviv, TA, Israel, 2 Maccabi Healthcare Services & Tel Aviv University, Tel Aviv, Israel, 3 AbbVie Inc., Hod-Hasharon, Israel, 4 AbbVie, Inc., North Chicago, IL, USA
OBJECTIVES: Chronic lymphocytic leukemia (CLL) is associated with a heavy economic burden. This study aims to evaluate healthcare resource utilization (HCRU) in the changing treatment era of CLL. METHODS: A retrospective database study was conducted in Maccabi Healthcare Services, a 2.2-million-member health fund in Israel. Included were CLL patients (defined by diagnosis, pharmacy and laboratory data in 1999-2017) initiating treatment in 2009-2017 (index=first line, L1). HCRU was measured annually pre- and post-index (Yn ) through 31/3/2018. Drivers of costs were investigated (per-patient-per-year direct medical costs estimated using Israeli Ministry of Health 2018 data). RESULTS: Among 411 included patients (mean±SD age: 63.8±11.5y; 63.7% male), 19.5% received L1 fludarabine/rituximab (primarily with cyclophosphamide [FCR]), 12.2% bendamustine/rituximab (BR), 11.4% obinutuzumab±chlorambucil (G-Clb), 22.3% chlorambucil monotherapy (Clb), 27.5% rituximab±chemotherapy not-elsewhere-classified and 7.1% other. Age (mean±SD) was lowest for L1-FCR (58.6±8.0y) and highest for Clb (75.0±8.9y) and G-Clb (74.8±6.9y). In Y1 , 54% were hospitalized (median=9 days [FCR: 52%, median=6 days]) and the median number of outpatient hematologist and primary-care-practitioner visits was 16 (FCR: 23) and 20 (FCR: 17), respectively. From Y1 to Y2 , there was no observed increase in hospitalization and outpatient visits and the ratio of hospitalization-to-outpatient-visit costs remained similar (1.6 vs. 1.4). CONCLUSIONS: This real-world study suggests that the economic burden within the first years of CLL treatment initiation is largely driven by hospitalization, similar to European reports.
ON2: VARIATION IN METHODOLOGICAL APPROACH TO PRODUCTIVITY COST VALUATION- DISTRIBUTIONAL EFFECTS IN THE CASE OF PROSTATE CANCER
4:00PM - 4:15PM
Hanly P 1 , Maguire R2 , Drummond F3 , Sharp L4 1 National College of Ireland, Dublin, Ireland, 2 Maynooth University, Maynooth, Ireland, 3 University College Cork, Cork, Ireland, 4 Newcastle University, Newcastle, UK
OBJECTIVES Standardised integration of productivity costs into health economic evaluations is hindered by equity and distributional concerns. Our aim was to explore the distributive impact of productivity cost methodological variation by socio-demographic characteristics, describing the consequences for different groups. METHODS 527 prostate cancer survivors (2-5 years post diagnosis) completed questions on work patterns since diagnosis. Productivity loss, categorised into temporary/permanent absenteeism, reduced hours and presenteeism, was costed in €2012. Valuation approaches included -the human capital approach (HCA), the friction cost approach (FCA) and the wage multiplier approach (WMA). Both national and self-reported wages were used. Costs were compared across socio-demographic and economic characteristics using non-parametric tests. RESULTS The estimated base case (HCA, using national wages) total productivity cost was €44,201 per prostate cancer survivor. Permanent absenteeism accounted for the largest cost (€18,537), followed by reduced work hours (€11,130), presenteeism (€8,148) and temporary absenteeism (€6,386). Productivity costs estimated according to the alternative approaches deviated widely from the base case, ranging between -89% (FCA: €4,625) to +49% (WMA: €65,764). Higher productivity costs were estimated compared to the base case using self-reported wages (€53,678); this finding was consistent across all valuation approaches. Statistically significant differences in productivity cost were found across four of the six survivor socio-demographic and economic characteristics by valuation approach, despite no significant difference in their physical unit equivalents. CONCLUSIONS Our results indicate that the distributional impact of productivity costs varies by socio-economic and demographic characteristic, in addition to valuation approach. We advocate that: productivity loss should be reported in physical units where possible; cost estimation should be subject to sensitivity analysis; and only where this is not feasible, that the HCA and national wages be used to value productivity loss where equity concerns are paramount.
ON3: HOSPITAL COSTS BEFORE, DURING, AND AFTER DIAGNOSIS OF FIVE CANCER FORMS IN DENMARK DURING 2014
4:15PM - 4:30PM
Green A 1 , von Arx LB2 , Hornbak M3 , Bliddal M1 1 University of Southern Denmark, Copenhagen N, Denmark, 2 ApHER, Copenhagen, Denmark, 3 AstraZeneca A/S, Copenhagen S, Denmark
OBJECTIVES: As part of The CEDAR Study (Cancer Impact in Denmark) aiming to characterize the epidemiology and impact of five cancers using health care registers (HCR), we present preliminary estimates of costs incurred at Danish hospitals before, around, and after diagnosis of cancers of lung, ovary, breast, bladder, and prostate during 2014. METHODS: Individual data on all patients living with the selected cancer forms between 2006-2015 from the Danish cancer registry were linked at individual level with other nationwide HCR providing data on pathology examinations, hospital activities, and their costs (in Diagnosis Related Grouping tariffs), and civil status. Costs related to year 2014 were aggregated according to whether the costs were incurred from -12 to -1 months before diagnosis; the last month before diagnosis through 1 year after diagnosis; 2, 3, 4, 5 or >5 years after diagnosis. Costs (EUROs) for each time interval were expressed per patient-year. RESULTS: The study covered totally 128,119 patient-years during 2014. For the 113,982 patient-years covering time from one month before diagnosis and afterwards, the overall mean costs ranged from 7,938 (breast cancer) to 26,877 (lung cancer) EUROs per patient-year. Costs per patient-year were highest around diagnosis and during the first year after diagnosis, ranging from a 16 time increase (breast cancer) to a 6 time increase (prostate cancer) compared with the interval -12 to -1 months before diagnosis. CONCLUSIONS: Cancer costs are particularly high around and during the first year after diagnosis and vary according to cancer form. In-depth analyses will investigate correlations between costs and comorbidity, stage at diagnosis as well as the modalities of initial treatment. When combined with epidemiological forecasts of future incidence and prevalence of various cancer forms, such information will provide valuable new information for the planning and dimensioning of current and future cancer care.
ON4: THE ECONOMIC BURDEN OF RENAL CELL CARCINOMA (RCC) IN CANADA USING REAL-WORLD EVIDENCE; A SOCIETAL PERSPECTIVE.
4:30PM - 4:45PM
Nazha S1 , Yanev I2 , Le F3 , Tanguay S1 , Dragomir A 1 1 McGill University Health Centre, Montreal, QC, Canada, 2 McGill University, Montreal, QC, Canada, 3 Faculty of Pharmacy, University of Montreal, Montreal, QC, Canada
OBJECTIVES: Kidney cancer is placed third in urologic cancers in Canada, right behind prostate and bladder cancer. Many new therapeutic options are being developed in the metastatic phase mainly, but these innovations are being presented with high costs. The objective of the current study is thus to establish clinical and economic outcomes of the current practice in RCC treatment in Canada post-nephrectomy. METHODS: A Markov model with microsimulation was developed to estimate the cost of follow-up and treating patients from post-nephrectomy up to diagnosis of metastatic RCC and death from any cause. The model included 5 health states: Active Surveillance, Local recurrence, mRCC, death from RCC or death from other causes. Probabilities were adjusted by taking in consideration patient characteristics and most estimate were extracted from real-world evidence studies assessing the survival of RCC and mRCC patients. Costs were extracted from available literature. Deterministic sensitivity analysis was conducted to account for uncertainty on different parameters by varying parameters by 25%. RESULTS: Mean survival (± SD) was evaluated to be 15.56 ± 5.69 life years (LYs) for T1 tumours, 13.22 ± 5.68 LY for T2, 12.22 ± 5.52 LY for T3 and 14.85 ± 5.71 LY for the weighted average of the 3 stages. The weighted mean and median total cost of the disease amounts to 107 811.22$ and 48 992.33$ respectively over a 20-year time horizon. In the weighted average scenario, the mRCC state costs represented the main burden, at around 40.3% of total cost. The local recurrence, active surveillance, death and kidney cancer related death states respectively represented 27.2%, 23.8%, 8.6% and 0.1%. CONCLUSIONS: The economic burden of mRCC is increasing with the severity of the disease. The results given in the present work are preliminary and constitute a groundwork for future studies that need to be done integrating newer treatment option.
DO INNOVATIVE TECHNOLOGIES REQUIRE INNOVATIVE APPRAISAL TECHNIQUES?- CASE STUDIES FROM RECENT HTAS IN THE UK, US AND JAPAN
PURPOSE: While innovative medicines, such as gene therapy, offer the potential for important health gains in many diseases, these innovations raise concerns about health care spending. As a result, health technology assessment (HTA) is gaining traction in countries where payers traditionally have not used HTAs for pricing negotiations. Because the need to adapt HTA processes and methods to overcome emerging challenges is likely similar across countries, discussion of the challenges and solutions surrounding how new technologies are appraised in different markets can provide valuable insights. This workshop will discuss how HTAs affect pricing and coverage decisions in the United Kingdom, the United States, and Japan and how pricing and market access are granted to drugs that may not be considered cost-effective using conventional methods. Recent case studies and application of novel HTA approaches can provide ideas on future directions in HTA methods and processes.
DESCRIPTION: The first section of this workshop will focus on understanding the relationship between HTAs and pricing and market access in each of the three markets (20 minutes). Next, speakers will discuss whether and how pricing and market access is granted to drugs that may not be considered cost-effective using conventional methods. The process and rationale for each of the three markets will be presented, illustrated by recent appraisals. Specifically, examples of patient access schemes, managed access schemes, and outcome-based contracts will be introduced (20 minutes). Speakers will discuss the feasibility and downstream implications of each scheme in addressing challenges faced in other markets. (10 minutes). There will be polling throughout the workshop for the audience to share their experiences and challenges in conveying the value of innovative medicines within their market’s HTA framework and to discuss the advantages and disadvantages of the approaches presented. Finally, there will be an opportunity for audience questions (10 minutes).
Discussion Leader
Ataru Igarashi, Ph.D.
The University of Tokyo, Tokyo, Japan
Jeanette Kusel, MSci, MSc
National Institute for Health and Care Excellence, London, United Kingdom
Isobel Pearson, DPhil
RTI Health Solutions, Manchester, United Kingdom
Naoko Ronquest, PhD
RTI Heatlh Solutions, Durham, NC, USA
IS INDICATION BASED PRICING FEASIBLE AND/OR BENEFICIAL FOR SOCIETY?
ISSUE: Feasibility and Desirability of Indication Based Pricing (IBP)
OVERVIEW: With this panel, we want to ignite a sound debate on whether IBP can be implemented as an alternative pricing mechanism. Also, we want to discuss whether IBP could be beneficial for society and, if so, under which conditions it may be.
We bring together three views: that of someone who believes it may work, that of someone who is not convinced and that of someone knowledgeable of the payer's perspective. This discussion promises to be well attended and of high interest for all stakeholders: payers, industry, patients and policy makers.
Moderators
Mireia Jofre-Bonet, PhD
Office of Health Economics, London, United Kingdom
Panelists
Andrew Briggs, DPhil
Health Economics & Health Technology Assessment, Institute of Health and Wellbeing, University of Glasgow, Glasgow, Scotland, United Kingdom
Andrew Briggs, DPhil, has extensive experience conducting economic evaluation analyses globally, and developing cost-effectiveness models across health systems. Andrew has a DPhil in Health Economics and MSc in Applied Statistics, both from the University of Oxford. He currently holds the William R Lindsay Chair of Health Economics at the University of Glasgow and has acted as Advisor to the UK Department of Health, the UK National Institute of Health & Care Excellence (NICE), and a number of pharmaceutical companies. He has published extensively on the topic of cost-effectiveness analysis and economic evaluation with over 200 publications in the peer-reviewed literature. He has served on numerous committees on economic evaluation methodology and reporting, including five ISPOR Task Forces. He is an Editor at the journal Health Economics, and has previously served on the editorial boards of Value in Health and Medical Decision Making. He is also lead author of the widely cited book Decision Modelling for Health Economic Evaluation (with Karl Claxton and Mark Sculpher; Oxford University Press 2006). Following a sabbatical at Center for Health Policy & Outcomes, Department of Epidemiology & Biostatistics, Memorial Sloan Kettering Cancer Center (MSKCC), with Dr. Peter Bach, Andrew continues to work half-time at MSKCC as a Visiting Investigator.
Adrian Towse, MA, MPhil
Office of Health Economics, London, United Kingdom
Professor Adrian Towse is director emeritus and senior research fellow of the Office of Health Economics in the UK. Adrian’s current research includes incentives for new drugs and vaccines to tackle Antimicrobial Resistance, the use of 'risk-sharing' arrangements between healthcare payers and pharmaceutical companies, including value-based pricing approaches; the economics of pharmacogenetics for healthcare payers and the pharmaceutical industry; economic issues that affect both R&D for and access to treatments for diseases prevalent in the developing world; the economics of medical negligence; and measuring productivity in healthcare.
A visiting professor at the London School of Economics and a senior researcher at the Nuffield Department of Population Health at the University of Oxford, Adrian also has been a visiting professor at the University of York. For ten years, he served as the non-executive director of the Oxford Radcliffe Hospitals NHS Trust, one of the UK’s largest hospitals. Adrian was president of ISPOR, for the 2014-15 term.
Adrian joined the OHE in 1993 and served as director for 25 years. He holds an MA (Hons) in Politics, Philosophy and Economics from Keble College, Oxford; an MPhil in Management Studies from Nuffield College, Oxford, and the Oxford Centre for Management Studies; and is a member of the Chartered Institute of Management Accountants.
Inneke Van de Vijver, MSc
National Institute for Health and Disability Insurance, Brussels, Belgium
ARE WE THERE YET? USING RWD FOR REGULATORY-GRADE EXTERNAL CONTROL ARMS.
ISSUE: Regulatory agencies have accepted external controls with RWD in certain cases when evaluating drug approvals and indication expansions. However, with focus on RWE, will external control arms become more acceptable? Questions frequently arise around the use RWD and analytic methods to develop sound external controls in lieu of a randomized-controlled trials (RCTs).
OVERVIEW: Moderator and panelists representing the pharmaceutical industry, academic institutions, regulatory policy organizations, and RWE analytics providers will discuss data and methods considerations for developing external control arms. The moderator will begin by reviewing and defining key terms - e.g., single arm trials, external controls with RWD. Panelists then will discuss recent examples where the EMA, FDA, and other regulators have accepted submissions using external controls, and where there are similarities or differences to note across these examples. Industry and academic panelist(s) will discuss some successes and challenges they have experienced with RWD vs. RCT data, and what their organizations are doing to advance the approach of RWD control arms. We also will provide a deeper dive into key analytical and statistical methods that can be used to effectively match patients for external control analyses, and will discuss regulatory and policy methods and feedback. Finally, panelists will discuss ways to ensure analysis quality when using external controls. Moderator will facilitate questions from the audience and panel discussion.
Moderators
Yin Ho, MBA MD
Aetion, Inc., New York, NY, USA
Panelists
Gregory Daniel, PHD, MPH
Edwards Lifesciences, Washington, DC, USA
Jeremy Rassen, ScD
Aetion, Inc., New York, NY, USA
Dr. Jeremy A. Rassen, ScD, is co-founder and chief scientific officer at Aetion, Inc., a company that provides software to evaluate the effectiveness, safety, and value of medical treatments. At Aetion, Dr. Rassen leads the scientific effort around designing methodology for obtaining and communicating medical evidence from real-world data.
Dr. Rassen was formerly an assistant professor of Medicine at the Brigham and Women's Hospital and Harvard Medical School, where he focused on methodology for improved validity and reach of pharmacoepidemiology and comparative effectiveness research, including research into propensity score and instrumental variable methods. Before coming to the Brigham and Women’s Hospital, Dr. Rassen worked in Silicon Valley in numerous computer and software companies, including Hewlett-Packard and Epiphany, Inc. His focus was on high-performance software for the creation and analysis of large marketing databases.
Dr. Rassen received his Bachelor’s degree from Harvard College and his Doctorate degree in Epidemiology from the Harvard School of Public Health.
Christophe Segalini, PhD, MBA
Idorsia Pharmaceuticals Ltd, Allschwil, Switzerland
16:00 - 16:30
ISPOR 2019 EXHIBITOR HEOR THEATER
A Vision for Sustainable Growth and Commercial Success in Cell & Gene Therapy
Presenters:
Andrew Hobbs , MD, Managing Director, Huron Consulting Group
Santanu Das , MD, Managing Director, Huron Consulting Group
Mapping the challenges and key considerations for cell and gene therapy success across the value chain and the need for cross-functional alignment and integrated solutions to drive commercial success
We are experiencing the first wave of cell & gene therapies in the market, however full adoption of a broader range of these technologies across many more indications will require significant change across health systems.
Based on this early experience a number of challenges have been identified and some of the conditions for long term sustainability are starting to emerge in terms of innovative commercial models to ensure products are sustainable and accessible even in conditions with large patient populations.
Huron Life Sciences will explore some of the current assessment frameworks, value benchmarks and uncertainty management and financing challenges that payers and payer advocates are currently experiencing and how these may shape future contracting solution sets for high-cost durable cell & gene therapies.
Sponsored by Huron Consulting Group
The HEOR Theater presentations are not an official educational offering of ISPOR Europe 2019 and are not sponsored, endorsed or accredited by ISPOR.
16:00 - 17:00
ISPOR BOOTH EVENT - MAKE THE MOST OF YOUR ISPOR EXPERIENCE - UPDATE YOUR PROFILE
Make the Most of Your ISPOR Experience—Update Your Profile
17:00 - 18:00
BREAKOUT SESSION 4
ADHERENCE, PERSISTENCE AND COMPLIANCE STUDIES
AD2: DYNAMICS OF SWITCHING, ADHERENCE, AND PERSISTENCE OF SODIUM GLUCOSE CO-TRANSPORTER 2 INHIBITORS USE- AN AUSTRALIAN PERSPECTIVE
5:15PM - 5:30PM
Ofori-Asenso R 1 , Liew D1 , Lalic S1 , Magliano D1 , Ademi Z2 , Bell JS1 , Ilomaki J1 1 Monash University, melbourne, VIC, Australia, 2 Monash University, Melbourne, VIC, Australia
OBJECTIVES: Poor adherence to diabetes medications is associated with adverse clinical outcomes. The aim of this study was to characterise the patterns of switching, adherence and persistence of Sodium-glucose co-transporter 2 inhibitors (SGLT2is) use in Australia. METHODS: Using Australian nationwide Pharmaceutical Benefits Scheme (PBS) data, we identified 11,981 adults aged ≥18 years who initiated SGLT2is (5993 dapagliflozin; 5988 empagliflozin) between September 2015 and August 2017. Adherence was measured via the proportion of days covered (PDC), persistence was defined as continuous use of SGLT2i until a gap of ≥90 consecutive days without medication on hand, and switching was defined as the first change from dapagliflozin to empagliflozin or vice versa. Generalised linear models were used to compare the adherence (PDC=continuous), logistic regression models were used to compare the likelihoods of being adherent (PDC≥0.80) and Cox proportional hazards regression models were used to compare the likelihoods of switching or being persistent between people prescribed empagliflozin and dapagliflozin over 12 months. RESULTS: Overall, 65.8% of people dispensed SGLT2i were adherent (PDC≥0.80) and 72.1% were persistent at 12-months. The mean PDC over the 1-year was 0.79±0.27. In comparison to dapagliflozin, the use of empagliflozin was associated with higher adherence (PDC=continuous) (odds ratio [OR], 1.04, 95% confidence interval [CI] 1.03-1.05), being adherent (OR 1.39, 95% CI 1.29-1.51) and persisting for 12-months (hazard ratio [HR] 1.14, 95% CI 1.06-1.22). Just 4.3% of people switched between the SGLT2i. Compared to dapagliflozin, empagliflozin was associated with a lower likelihood of switching (HR 0.46, 95% CI 0.38-0.55). CONCLUSIONS: Real world Australian nationwide data suggest that a considerable proportion of people prescribed SGLT2is are non-adherent or non-persistent. However, empagliflozin was found to be associated with better adherence and persistence as well as a lower likelihood of switching compared to dapagliflozin. Interventions to improve adherence among people prescribed SGLT2is are needed.
AD1: PERSISTENCE OF BIOLOGIC THERAPIES FOR THE TREATMENT OF PLAQUE PSORIASIS - A NATIONAL LONGITUDINAL OBSERVATIONAL POPULATION STUDY IN SWEDEN
5:00PM - 5:15PM
Stelmaszuk-Zadykowicz NM1 , Apol E 2 , Hansen JB3 , Freilich J4 1 PAREXEL International, Stockholm, Sweden, 2 LEO Pharma, Ballerup, Denmark, 3 LEO Pharma A/S, Ballerup, Denmark, 4 PAREXEL International, Stockholm, AB, Sweden
OBJECTIVES Recent observational studies suggest that persistence of biologic therapies in plaque psoriasis (psoriasis) differs from what is observed in clinical trials. The objective of this study is to assess the persistence of biologic therapies for the treatment of psoriasis in clinical practice in Sweden. METHODS A longitudinal observational population study was carried out using individual-level data from the Swedish National Patient Register, Prescribed Drug Register, and Cause-of-Death Register. Included patients were adults diagnosed with psoriasis, treated with a biologic between 2010-2018. Median treatment persistence and 1-year predicted persistence probabilities were estimated from Kaplan-Meier curves. Sub-group analysis was conducted with biologic-naïve vs. biologic-experienced patients. Biologic therapies with <20 patients and biologics used off label are not reported. RESULTS 2,258 patients with 2,975 treatment periods were included (adalimumab: n=1,046, etanercept: n=974, ustekinumab: n=488, secukinumab: n=394, ixekizumab: n=50, and certolizumab pegol: n=23). Mean age at psoriasis diagnosis was 42.1 (SD 14.2); 62% were male; 78.7% had concomitant psoriasis medication; and 61% were biologic naïve (range: 12-93%) Overall median drug persistence was 23.8 months (95% CI: 21.6-26.2). For biologics reaching 50% drug continuation, median persistence ranged from 49.3 months (95% CI: 38.0-59.1, ustekinumab) to 9.3 months (95% CI: 6.4-18.1, certolizumab pegol). Secukinumab and ixekizumab did not reach the 50% drug continuation probability threshold. The proportion of persistent patients after 1 year was highest for ixekizumab (81.3%), followed by ustekinumab (79.9%), secukinumab (75.9%), adalimumab (64.6%), and etanercept (57.8%). Biologic-naïve patients had higher drug persistence than biologic-experienced patients for all treatments except certolizumab pegol and ixekizumab, both limited by low patient numbers. CONCLUSIONS In this observational study in Sweden, median persistence was around 2 years for biologic therapies in psoriasis in clinical practice. The study indicates that modern biologic therapies may have higher persistence than traditional anti-TNFs, though potential confounders need to be explored further.
AD4: ESTIMATING THE RELIABILITY OF THE MEDICATION ADHERENCE REASONS SCALE (MAR-SCALE) IN ASTHMA AND CHRONIC OBSTRUCTIVE PULMONARY DISORDER AND IDENTIFYING THE REASONS FOR NON-ADHERENCE
5:45PM - 6:00PM
Unni E1 , Gupta S 2 , Sternbach N2 1 Touro College of Pharmacy, New York, NY, USA, 2 Kantar, New York, NY, USA
OBJECTIVES: Chronic respiratory diseases accounts for 6.3% of global Years Lived with Disability (YLDs); COPD contributing to 29.4 million YLDs and asthma, 13.8 million YLDs. Literature reports adherence to asthma medication between 30-70% and that to Chronic Obstructive Pulmonary Disorder (COPD) medication at <50%. Knowing the prevalence and reasons for non-adherence to asthma/COPD medications are beneficial in developing both patient and population level adherence improvement interventions. This calls for the need to develop a reliable self-reported adherence measure so that reasons for non-adherence can be understood. The objective of this study was to establish the reliability of the Medication Adherence Reasons Scale (MAR-Scale) in measuring non-adherence in asthma/COPD and to describe the reasons for non-adherence. METHODS: Data from the 2018 National Health and Wellness Study, a self-administered, annual, internet-based cross-sectional survey of US adults was used. Respondents who self-reported taking daily prescription medication(s) to treat asthma/COPD were given the MAR-Scale, a 20-item comprehensive scale. The scale has 19 items/reasons for non-adherence and one global item, that measures non-adherence “in the past 7 days”, on an 8-point scale ranging from 0 days to 7 days. Scale reliability was estimated using Cronbach’s alpha and confirmatory factor analysis. Frequencies were used to identify the reasons for non-adherence. RESULTS: The Cronbach’s alpha for the MAR-Scale in asthma (N = 2,810) was 0.880 and 0.932 in COPD (N = 1,632). The Goodness of Fit Index (0.982, 0994) and Standardized Root Mean Square Residual (0.048, 0.036) was acceptable for asthma and COPD respectively. The medication non-adherence rate in asthma was 38.4% and 28.4% in COPD. The most common reasons for non-adherence for both conditions were “simply missing” and “skipping medication to see if still needed”; and cost for COPD. CONCLUSIONS: The MAR-Scale demonstrated acceptable reliability with both asthma and COPD medications and provided an overall estimate for non-adherence.
AD3: ORAL ANTICOAGULANT PRESCRIPTION TRENDS, PROFILE USE AND DETERMINANTS OF ADHERENCE IN PATIENTS WITH ATRIAL FIBRILLATION
5:30PM - 5:45PM
Perreault S 1 , de Denus S2 , White-Guay B3 , Côté R4 , Schnitzer M3 , Dubé MP5 , Dorais M6 , Tardif JC5 1 Université de Montréal, Montreal, QC, Canada, 2 Institut de Cardiologie de Montreal, Montreal, QC, Canada, 3 University of Montreal, Montreal, QC, Canada, 4 Montreal General Hospital, Montreal, QC, Canada, 5 Montreal Heart Institute; Faculty of medicine, Université de Montréal; Université de Montréal, Beaulieu-Saucier Pharmacogenomics Centre, Montréal, QC, Canada, 6 StatSiences Inc,, Notre-Dame-de-l'Ile-Perrot, QC, Canada
OBJECTIVES: There is limited data on oral anticoagulants (OAC) uptake and pattern of use. Real life data in patients with atrial fibrillation (AF) is important for understanding patient exposure. A cohort study of new OAC users was built to assess trends of drug use from 2011 to 2017, persistence rate, adherence level and its predictors. METHODS : We built a cohort using the Quebec RAMQ and Med-Echo administrative databases of new adults OAC users within the 1-year following hospitalization with a diagnosis of AF. New users of OAC were defined as no OAC claims in the 1-year prior to cohort entry. Trends of OAC use, persistence rate defined as gap between refills of 30 days, and adherence level defined as the proportion of days covered (PDC) were assessed over a 1-year period following the initiation. Predictors of non-adherence (PDC<80%) were analysed using logistic regression models. RESULTS: The cohort consisted of 33,311 incident OAC users. The proportions of warfarin claims decreased from 77.9% to 12.7% (2011-2017) of total OAC claims, with direct OACs (DOACs) accounting for 86% of claims for which apixaban and rivaroxaban accounted for 60.1% and 23.4%, respectively. After OAC initiation, the persistence rates ranged from 49.1% to 70.6%; the highest rate was with the high dose of apixaban and the lowest one with warfarin. Approximately 75% of incident OAC users were considered ‘adherent’ (PDC ≥ 80%) with mean PDC of 95.6 to 98.1% compared to ‘non-adherent’ varying between 43.1 and 50.7%. Older age, female gender, higher CHA2 DS2 -VASc score, prior stroke and treatment for chronic cardiovascular disease drugs were associated with high adherence levels. CONCLUSIONS: The uptake of DOACs increased overtime, accounting for 86% in 2017. In our study, 25% of new OAC users presented a low adherence level. Adherence to OACs remains a significant challenge in patients with AF.
METHODOLOGICAL AND CONCEPTUAL STUDIES
CP1: THE IMPACT OF USING AGGREGATE DATA FOR MULTISTATE MODELLING PURPOSES IN ECONOMIC EVALUATIONS
5:00PM - 5:15PM
Wigfield P 1 , Ouwens DM2 , Vincken T1 , Postma M3 , Heeg B1 1 Ingress-health, Rotterdam, ZH, Netherlands, 2 AstraZeneca, Gothenburg, Sweden, 3 University of Groningen, University Medical Center Groningen, Groningen, Netherlands
Background: Multistate modelling (MSM) is an alternative to partitioned survival modelling. For standard oncology models, MSMs separate individual patient event times into three transitions: (1) time to progression (TTP), (2) TTP to death, and (3) time to death after progression. Williams et al. suggests that MSMs can be created from aggregated data, though assumptions are required when creating individual patient-level data (IPD) with PFS and corresponding OS time per patient. The aim was to assess the challenges of creating an MSM using aggregated data. Methods: Based on simulated and subsequently aggregated data, OS and PFS Kaplan-Meier curves were used to obtain IPD for OS and PFS using a validated algorithm by Guyot et al. An assumption that the shortest PFS time corresponds to the shortest OS time was used to create IPD with PFS and OS time per patient. Parametric distributions were fitted and combined to estimate PFS and OS. Results: Due to the assumption of linking shortest PFS to shortest OS, the MSM predicted that all PFS events are progression events, which implied that there are no death events before progression. Also, an artificial correlation was introduced between timing of progression and risk of death. Nevertheless, the MSM predicted PFS accurately, but overestimated OS for the initial months for both treatments, and fitted OS relatively well thereafter. Conclusions: The challenge of using MSMs with aggregated data is that the time each patient contributes to each transition relies on assumptions which affect the plausibility of extrapolations. OS is likely overestimated due to the artificial correlation not accounted for when estimating the time to death after progression predictions. If the number of PFS deaths are reported, this should be considered when linking OS and PFS time per patient. More research and guidance are needed on estimating MSMs based on aggregated data.
CP3: DEVELOPING PROCESSES AND METHODS FOR A EUROPEAN COLLABORATION ON HTA- ACHIEVEMENTS, CHALLENGES AND LESSONS LEARNT
5:30PM - 5:45PM
Fathollah-Nejad R 1 , Rehrmann M1 , Chalon PX2 , Fujita-Rohwerder N1 , Luhnen M1 1 Institute for Quality and Efficiency in Healthcare (IQWiG), Cologne, NW, Germany, 2 KCE - Belgian Health Care Knowledge Centre, Brussels, Belgium
EUnetHTA (European Network for Health Technology Assessment) Joint Action 3 (JA3) is a collaborative network project consisting of 83 organisations from 30 European countries. One of the main objectives is to lay the foundation for a sustainable model of European collaboration on Health technology Assessment (HTA). A central part of this is the establishment of a quality management system (QMS) for the production of high-quality HTA-reports. Since the start of JA3 in May 2016, 27 partner organisations from different European countries and regions have been jointly developing, elaborating and maintaining around 40 standard operating procedures (SOPs) and other parts of the QMS. Previously developed methodological guidelines, templates and tools have undergone required updates; two new guidelines are currently in production. Thereby, this collaboration of a broad variety of partners has offered opportunities and entailed challenges. The wide heterogeneity of given regional and national health care systems throughout the continent with different conditions for reimbursement, HTA-procedures and resource endowments remarkably influence how processes and methods, which are designed for the European context, are understood and formulated. Examples include the data requirements and the mode of stakeholder involvement. To overcome these barriers, remedies have been created such as an information specialist network, a consensus procedure in case of unresolvable disagreements and trainings on practical aspects. The work results benefited from the exchange of different views and from a broad spectrum of experiences and expertise. The concentrated scientific and practical knowledge is one of the major virtues of European consortium projects. The piloting and continuous evaluation of the established QMS helps to improve the mode of collaboration by identifying gaps and shortcomings of the newly developed processes, methods and products. However, to find a balance between adequate processes and given restrictions of national settings will be an ongoing task.
CP2: DO SOCIAL VALUES AND INSTITUTIONAL CONTEXT IMPACT THE USE OF ECONOMIC EVALUATION IN PRIORITY SETTING- EMPIRICAL EVIDENCE FROM OECD COUNTRIES
5:15PM - 5:30PM
Torbica A 1 , Fornaro G2 , Tarricone R2 , Drummond M3 1 Bocconi University, Milan, Italy, 2 SDA Bocconi School of Management, Milan, Italy, 3 University of York, York, UK
OBJECTIVES: To empirically investigate if and how the use of economic evaluation in healthcare priority setting is influenced by social values and institutional context in a given country. METHODS: We developed and tested a conceptual framework in the 36 OECD countries, divided in two groups based on their extent of use of economic evaluation in decision-making. The key social values were efficiency, equity and personal responsibility, measured in a pan-European survey. Countries were classified based on their institutional context in terms of both general paradigm of welfare system, type of healthcare system and according to the administrative tradition they belong to (Anglo-American, Germanic, Napoleonic, Scandinavian, Latin-American, Post-colonial, and Asian). We conducted a series of correlation and similarity tests to analyze association between the use of economic evaluation and other variables. RESULTS: There was significant correlation between Anglo-American administrative tradition and high use of economic evaluation (r=0.4077, p=0.014) while Social Health Insurance was significantly associated with low economic evaluation use (r=-0.3797, =0.032). Napoleonic countries reject personal responsibility in health (r=-0.459, p=0.021) while Germanic countries embrace it (r= 0.473, p= 0.017). Anglo-American countries exhibit a significant preference towards efficiency (r= 0.393, p= 0.052). No significant association was found between social values and use of economic evaluation. CONCLUSIONS: Preliminary results suggest that social values and institutional context do matter greatly when it comes to shaping the attitude towards the use of economic evaluation analysis in healthcare priority setting. While institutional context appears to have a direct influence, role of social values is more mediated through the administrative traditions present in different countries. These findings can help us better understand differences across countries and inform jurisdictions interested in expanding the use economic evaluation in decision making.
CP4: META-ANALYSIS POWERED BY ARTIFICIAL INTELLIGENCE.
5:45PM - 6:00PM
Joshi S, Hvingelby R, Holm-Larsen T A-Evidence, Copenhagen, Denmark
OBJECTIVES: Meta-analysis is a statistical approach to develop a systematic overview of the effects of a therapeutic treatment. It is the highest type of evidence for medical guidelines and health economic prioritization. It is time consuming and only provides a static ‘here and now’ overview, which must be repeated. We wanted to explore how many parts of the meta-analysis could be optimized with an improved information technology (IT) system. METHODS: The process of meta-analysis was divided into 4 phases: Data import, Screening of articles, Extraction of data and Statistical analysis. For each phase we tested which parts of the process could be eliminated through improved IT software and Artificial intelligence (AI). RESULTS: In the data import phase we could directly connect to literature search engines such as Pubmed and Embase. The Screening phase was optimized with dynamic back loops, so the search string would automatically be updated. In the extraction phase, AI was used to extract data directly from the articles. For statistical analysis, the system was connected directly to the statistical software ‘R’. By incorporating all phases into one IT-system i.e. linking the calculation of the statistical forest plot to the search string, we discovered an additional optimization of the meta-analysis process. It was possible to play around with the forest plot without losing track of the initial search string, e.g. it opened for exploration of the direct impact on the forest-plot of deleting an article or of limiting the analysis by age or gender. CONCLUSIONS: We optimized all 4 phases of a meta-analysis. The time reduced on developing meta-analysis will ensure better decision making and prioritization. However, the essential optimization is perhaps the connectivity with the search engines, changing the work with meta-analysis from a static picture of effect and side-effect to constantly updated evidence.
CONSIDERATIONS FOR ANALYZING REAL WORLD GENOMICS DATA – THE VALUE, RISKS & PRECAUTIONS
ISSUE: Genomics data is increasingly incorporated into non-interventional studies (NIS). When collected and analyzed in a rigorous manner, genomics data has the potential to enhance clinical decision making and improve patient outcomes. Issues to consider when conducting RW genomics studies include: type of sample and collection method, sample size, and generalizability of the data. Therefore, it’s critical to debate the risks and benefits of using RW genomics data in NIS to improve this field of work.
OVERVIEW: This panel will debate the value and risks of including genomics data in NIS and implications this may have on clinical decision making. Emilie will begin with an overview of the landscape of genomics data used in NIS studies and pose questions for the panelists to debate: What’s the value of genomics data in research and clinical practice? What are common statistical pitfalls that could be encountered, and prevented, when analyzing RW genomics data? Are there ethical considerations for conducting such studies? Katarzyna will discuss the availability of genomics data within Genomics England and how this data can be linked to clinical data and discuss the value this brings to NIS. Michael will discuss the everyday use such data could have in treating patients and the potential risks of inaccurate data. Jim will highlight the concerns of poor-quality methods when analyzing RW genomics data and how some issues can be mitigated by using robust statistical analysis plans. Each panelist will briefly answer the question around ethical considerations of using genomics data. The discussion will conclude with an audience Q&A. This panel will be valuable for a diverse group of stakeholders –researchers conducting NIS incorporating genomic data; clinicians keen to learn how to interpret such publications; and patients who can benefit from future research that will be both innovative yet adheres to rigorous methodology.
Moderators
Emilie Scherrer, MSc
MSD, Kenilworth, NJ, USA
Panelists
James Anderson, PhD
MSD, North Wales, PA, USA
Michael Weichenthal, MD
Christian-Albrechts-Universitat zu Kiel, Kiel, NJ, Germany
Kate Witkowska, PhD
Genomics England, London, LON, United Kingdom
SURVIVAL EXTRAPOLATION APPROACHES- NEW ERA, NEW METHODS?
ISSUE: Partitioned survival analysis (PartSA) with standard parametric distributions is widely used in disease areas such as oncology, as it is perceived to provide a transparent way of extrapolating and quantifying survival benefits beyond the trial duration. A myriad of methods are available for extrapolating survival curves within a PartSA framework, but all are based on the same structural assumptions. With the introduction of immunotherapies such as checkpoint inhibitors and CAR T-Cell therapies, a new era of oncology therapies is upon us, bringing the potential for prolonged survival and possibly even cure. The question is, do these new therapies warrant new methods for extrapolating survival? Alternative methods such as mixture cure models, splines and responder-based approaches may provide benefits over the traditional PartSA approach, but also introduce new limitations. Furthermore, and perhaps most importantly, what is the acceptability of these new methods for different stakeholders such as policy makers and payers?
OVERVIEW: This panel will debate the application of different survival extrapolation approaches. The debate will be focussed around three different perspectives: a methodological perspective, industry perspective, and policy maker perspective. After 10-minute presentations introducing each of the different perspectives, the panellists will have a plenary discussion in two rounds, covering: merits and limitations of different survival extrapolation approaches, and adoption of new approaches. Each round will be governed by a central question, on which the audience will be polled both at the start and end of the round. Participation of the audience in the discussion, either in-person or via the app, is highly encouraged. The discussion is not meant to involve a technical deep-dive but rather focus on the implications of using the different extrapolation approaches and the adoption of new approaches. The issue panel will therefore be relevant for data analysts, industry representatives, and decision makers alike.
Moderators
Elisabeth Fenwick, PhD
Pharmerit International, Oxford, OXF, United Kingdom
Elisabeth Fenwick is a senior director in the Modeling and Meta-Analysis team at Pharmerit International, based in Oxford in the UK.
Liz provides scientific and strategic support to HE projects globally. She has extensive experience in economic evaluation and health economic modeling having worked in the field for over 20 years. She has worked on a variety of projects in a wide range of disease areas including oncology, respiratory, infectious diseases, cardiology, ophthalmology, and orphan diseases.
Liz has also contributed to methods in the field, in particular relating to decision analytic modeling and simulation methods, probabilistic decision analytic modeling and value of information analysis. Liz was a member of the ISPOR joint task force on good research practices in modeling and a co-author on the joint taskforce paper on uncertainty and is currently co-chairing the ISPOR task force assessing emerging good practice in value of information analysis for research decisions. Liz is also a member of the editorial board for Pharmacoeconomics.
Liz has a PhD and MSc in Health Economics as well as an MSc in Operations Research and joined Pharmerit from ICON plc where she led the modeling team for the global HE group. Prior to her consultancy career, Liz spent over 15 years as an academic working at University of York, McMaster University, and most recently University of Glasgow.
Panelists
Sven Klijn, MSc
Pharmerit International, Rotterdam, ZH, Netherlands
Stephen Palmer, Prof, PhD
University of York, Heslington, York, United Kingdom
John Whalen, BSc, MBA
Ipsen Biopharm Ltd, United Kingdom, United Kingdom
USING PATIENT PREFERENCE DATA TO SUPPORT REIMBURSEMENT AND PRICING DECISIONS- CURRENT PRACTICE, OPPORTUNITIES AND CHALLENGES (Advanced Workshop)
PURPOSE
: To identify and discuss the opportunities and challenges incorporating patient preference data into reimbursement and pricing decisions. This workshop will benefit decision makers and sponsors considering how to make best use of patient preference data.
DESCRIPTION
:
Background: Health technology assessment (HTA) requires value judgments about the trade-offs between benefits, risks and costs. A recent ISPOR working group identified widespread use of preference research to support decision makers with these value judgements, including the use of data on patient preferences. This use of such data is still exploratory and emerging, with a lack of clarity on the when and how these data might be useful and which methods might be useful to collect this data. Many HTA agencies are still initiating such methods to help understand how they might be used.
Leaders : Using the case study of NICE, Kevin Marsh will illustrate how patient preference data have been used to inform reimbursement and pricing decisions and outline the normative challenges to using patient preference data to estimate economic value and how these might be overcome. Salah Ghabri and Douglas Lundin will provide HTA agency/payer perspectives. Douglas will summarize the role of patient preferences at Sweden’s TLV, including when the reliance on QALYs is inappropriate, and whether patient preference data can overcome some of the limitations of the QALY, and the extent to which sponsors have opted to submit this data. Salah will describe the current use of patient preference data at HAS and the opportunities to involve health system users in HTA. Axel Mühlbacher will introduce the ISPOR working group on health preference research and chair and facilitate discussions between workshop leaders and with the audience on the current and future role of patient preference data in European reimbursement decisions
Discussion Leader
Salah Ghabri, PhD
French National Authority for Health (HAS), Saint-Denis La Plaine, France
Douglas Lundin, PhD
Tandvårds- och läkemedelsförmånsverket (TLV), Stockholm, Sweden
Kevin Marsh, PhD
Evidera Ltd, 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.
Axel Mühlbacher, PhD, MBA
Hochschule Neubrandenburg, Neubrandenburg, BW, Germany
IMPROVING EFFICIENCY IN HTA- THE ROLE OF OPEN SOURCE MODELS AND MORE ADVANCED SOFTWARE CHOICE
PURPOSE
: To demonstrate the need to improve efficiency of HTA through increased openness and the use of advanced software; we will work with the audience to understand barriers and devise solutions.
DESCRIPTION
: The uptake of findings from HTA has increased considerably with rising global healthcare costs and the costs of innovation. This increased role in decision-making comes with requests for greater transparency and sharing. Many have argued that openness can improve efficiency of HTA processes by reducing recreation of already developed economic models. However, openness produces concerns about intellectual property and scholarly credit. The ISPOR Open Source Models Special Interest Group (SIG) was formed to address these concerns. At the same time, shifting regulatory and HTA timelines and methodological developments have resulted in more complex analyses being required in shorter timeframes, stretching the limits of Excel to breaking point. This workshop will offer participants the most recent efforts to improve efficiency in HTA. Discussion leaders will draw from their own diverse experiences as collaborators with HTA bodies. Brett McQueen will begin with an overview of HTA goals in the context of openness and new software options for US HTA submissions. Raquel Aguiar-Ibáñez will discuss openness and use of efficient software to increase automation of the more repetitive aspects of economic analysis, from an industry perspective. Dawn Lee will share learnings from model development in more advanced software such as R-Shiny. Gianluca Baio, as a member of ISPOR’s Open Source Models SIG, will discuss some of the barriers to adoption and provide solutions.
Discussion Leader
Raquel Aguiar-Ibáñez, MSc
Merck Sharp & Dohme Ltd, Haarlem, Netherlands
Gianluca Baio, PhD
University College London, London, United Kingdom
Dawn Lee, MMath, MSc
BresMed Health Solutions Ltd., Sheffield, DBY, United Kingdom
R. Brett McQueen, PhD
University of Colorado Anschutz Medical Campus, Denver, CO, USA
R. Brett McQueen is an Assistant Professor at the University of Colorado (CU) Skaggs School of Pharmacy and Pharmaceutical Sciences, and member in the Center for Pharmaceutical Outcomes Research. His research interests include decision-analytic modeling applications and methodology, applied microeconometrics in health, and novel value assessment methods. Brett has current funding in micro-costing health interventions, evaluating performance-based risk sharing agreements, estimating patient and payer preferences for various pharmaceuticals, and novel value assessment methods. He is the course director for “Pharmaceutical Economics and Policy Analysis” in the Pharmaceutical Outcomes Research PhD program at CU.
18:00 - 19:00
POSTER AUTHOR DISCUSSION HOUR - SESSION 2
Attendees have the opportunity to interact with poster presenters during this hour-long session.
18:00 - 19:30
WELCOME RECEPTION IN THE EXHIBIT HALL
18:00 - 21:00
"DINE-AROUND" WITH WOMEN IN HEOR (offsite)
Enjoy the perfect opportunity to sample some of Copenhagen’s delicious foods while engaging in conversations with your colleagues from the Women in Health Economics and Outcomes Research (HEOR) Initiative. We are offering dine-around options during the conference. This special dining option is limited to groups of no more than 10 attendees and features a variety of Copenhagen’s restaurants. Each group has a designated dinner host(s) who is coordinating the event at that restaurant venue. Participants will cover their own cocktails and dinner. Tables fill up quickly, so please be sure to visit the ISPOR Women in HEOR Initiative webpage (
https://www.ispor.org/strategic-initiatives/more/women-in-heor ) to reserve your seat!
18:15 - 19:15
EDUCATIONAL SYMPOSIUM
HOW CAN VALUE ASSESSMENT PRINCIPLES BE APPLIED TO SUPPORT ACCESS TO INNOVATIVE MEDICINES IN MIDDLE EAST AND NORTH AFRICA (MENA)REGION?
The MENA region is characterised by diversity in its demand for health care and its fragmented healthcare systems. Despite universal health coverage pledges by most countries, gaps in health insurance coverage of pharmaceuticals remain. Even though there are no formal HTA systems in place in the region, there is an interest in HTA and a willingness to explore how a shift toward a value driven system could materialise with the adoption of a value assessment framework in the decision-making process.
This symposium outlines the various HTA models and their relevance and applicability to different healthcare systems; it discusses how different HTA models and the associated principles could be applied in MENA region. In the majority of MENA countries, the system is dominated by price control predominantly through External Reference Pricing (ERP) and without explicit consideration of the value of innovation. This often leads to significant delays in the access to innovation. The session will discuss how the MENA region could transition from ERP to a value assessment system in the coming years which incorporates clinical and economic evidence in an effort to improve efficiency in resource allocation and quality of care. The attendees will be introduced to the first steps put in place in the Kingdom of Saudi Arabia to implement health technology assessment in the context of Saudi Arabia’s Vision 2030 and the barriers to overcome to build a sustainable and efficient health care system delivering high-quality care.
Sponsor
Sanofi
Moderators
Chris Chinn, MSc,ACA
sanofi, Weybridge, SRY, United Kingdom
Speakers
Abdulaziz H. Al-Saggabi, BSc,MSc, PharmD
Ministry of National Guard Health Affairs, Past-Chair ISPOR Saudi Arabia Chapter, Chairman ISPOR Arabic Network Executive Committee, Riyadh, Saudi Arabia
Panos Kanavos, PhD
London School of Economics and Political Science, London, United Kingdom
Adrian Towse, MA, MPhil
Office of Health Economics, London, United Kingdom
Professor Adrian Towse is director emeritus and senior research fellow of the Office of Health Economics in the UK. Adrian’s current research includes incentives for new drugs and vaccines to tackle Antimicrobial Resistance, the use of 'risk-sharing' arrangements between healthcare payers and pharmaceutical companies, including value-based pricing approaches; the economics of pharmacogenetics for healthcare payers and the pharmaceutical industry; economic issues that affect both R&D for and access to treatments for diseases prevalent in the developing world; the economics of medical negligence; and measuring productivity in healthcare.
A visiting professor at the London School of Economics and a senior researcher at the Nuffield Department of Population Health at the University of Oxford, Adrian also has been a visiting professor at the University of York. For ten years, he served as the non-executive director of the Oxford Radcliffe Hospitals NHS Trust, one of the UK’s largest hospitals. Adrian was president of ISPOR, for the 2014-15 term.
Adrian joined the OHE in 1993 and served as director for 25 years. He holds an MA (Hons) in Politics, Philosophy and Economics from Keble College, Oxford; an MPhil in Management Studies from Nuffield College, Oxford, and the Oxford Centre for Management Studies; and is a member of the Chartered Institute of Management Accountants.
19:30 - 21:00
ISPOR CENTRAL & EASTERN EUROPE (CEE) CONSORTIUM RECEPTION
The CEE Consortium and Regional Chapters in Europe are hosting the event and welcome you to connect with regional HEOR experts! The reception’s informal atmosphere offers excellent opportunities to share ideas, learn about the activities and initiatives of the CEE Consortium, what is trending in the CEE HEOR community, and expand your network of contacts in the CEE region. To learn about the CEE Consortium, visit www.ispor.org /Global Groups/Central and Eastern Europe Consortium.
STUDENT & FACULTY NETWORKING RECEPTION
All students and faculty are welcome to attend the reception where ISPOR will be distributing prizes and students, and faculty can network with their peers in a relaxed environment.
Tue 5 Nov
7:00 - 8:15
ISPOR FORUMS
ISPOR DIGITAL HEALTH SPECIAL INTEREST GROUP- CATEGORIZATION AND DEFINITIONS OF DIGITAL HEALTH INTERVENTIONS
The digitalization of healthcare has recently experienced exponential and continuous growth, which offers both great opportunity and significant challenges. The myriad of digital health interventions (DHIs) may be classified/categorized in different ways by different organizations or across global regions or not classified/categorized at all. This inconsistency leads to confusion and can inhibit assessments for regulatory, market access, pricing, and reimbursement decision-making. As such, there is a need to provide a common approach towards the categorization and definition of DHIs. This forum will outline the above challenges and discuss the ISPOR Digital Health Special Interest Group’s approach to solving these issues. The panel will address recommendations, ongoing projects, and future plans for the categorization and definition of DHI terms to further our understanding of digital health technologies to better solve healthcare problems. During these presentations, attendees will be asked to respond to specific questions relating to DHI definitions and categorization.
Moderators
Carl V. Asche, PhD
University of Illinois College of Medicine at Peoria, Peoria, IL, USA
Speakers
Anita Burrell, MA, MBA
Anita Burrell Consulting LLC, Flemington, NJ, USA
Katarzyna Kolasa, PhD
Kozminski University, Warszawa, Poland
Katarzyna Kolasa has more than 20 years of academic and business experience in the healthcare sector. She holds a PhD degree in health economics and is an author of more than 60 publications. Katarzyna Kolasa reviews manuscripts submitted to Health Policy, Value in Health, Expert Review of Pharmacoeconomics & Outcomes Research, International Journal for Equity in Health and International Journal of Technology As-sessment.
Katarzyna Kolasa heads the Health Economics & Healthcare Management Division (HeM) at the Kozminski University. She is the leader of the International Master Pro-gram Health Economics & Big Data at the Kozminski University financed by the re-search grant of the National Center for Research and Development which was listed as the best project in the POWER 2018 call.
In the past, she worked at both global and regional Health Economics & Outcomes Re-search (HEOR) functions at AstraZeneca and BiogenIdec being based in Sweden and Switzerland respectively. At Bristol Myers Squibb and Lundbeck she was leading Mar-ket Access teams in Central Eastern European (CEE) and Nordic Region. Her practical skills in the field of health economics were developed at the Kalmar County Council in Sweden as well.
For the last five years, she has been developing experience with the pricing &reimbursement challenges in the field of medical devices. Since 2016, Katarzyna has been representing Straub Medical as Global Market Access Lead being the External Principal Consultant for that Swiss family owned company. Before that, Katarzyna was employed as a Senior HEOR Director at GE Healthcare.
Her extensive knowledge in the field of health economics was acquired at University of York, University of Lund, and University of Bergen as well as during International Doc-toral Courses in Health Economics and Policy organized by the Swiss School of Public Health.
Katarzyna was nominated by the Ministry of Science to represent Poland in the COST Action CA17117, COST Association „Towards an International Network for Evidence-based Research in Clinical Health Research”. Since January 2019, she is working on the establishment of Global Special Interest Group for Digital Health at ISPOR.
Ken Redekop, PhD
Erasmus University Rotterdam, Rotterdam, Netherlands
ANIMAL HEALTH – NEW KID ON THE HEOR BLOCK?
HEOR in Animal Health has historically focused on economics of interventions in livestock. However, the increased appreciation of the importance of emotional and physical effects of pet-ownership, as well as welfare and environmental concerns towards intensive food animal production, demand a wider understanding of value. One Health, which recognizes that Human Health is tightly connected to animals and the environment, has gained traction given the above, as well as the recognition that 70% of all emerging infectious diseases in humans (H1N1, SARS, yellow fever) have an animal origin. Join us for a review of HEOR applications in Animal and One Health. We will compare the underdevelopment of HEOR in these two domains, relative to that in Human Health, discuss opportunities and promising markets, and present our next steps for your feedback and active engagement. Come and help us shape the future of HEOR in Animal and One Health!
Moderators
Isaac Odeyemi, DVM, MSc, MBA, PhD
Zoetis International Operations, Dublin, Ireland
Speakers
Elaine Brohan, PhD
Adelphi Values, London, United Kingdom
Johanne Ellis-Iversen, PhD, MSc, DVM
Technical University of Denmark, Copenhagen, Denmark
Colette Mankowski, BSc, MSc, PhD
Astellas Pharma EMEA, London, SRY, United Kingdom
Jacky Reid, BVMS, PhD, DVA
NewMetrica, Glasgow, United Kingdom
ISPOR RARE DISEASE SPECIAL INTEREST GROUP- SMALL NUMBERS, BIG ISSUES
In recent years, rare diseases have attracted significant interest by drug manufacturers. Legislative and financial incentives, combined with scientific and technological advances, have stimulated the development of novel rare disease treatments. Today, almost 600 orphan drugs are approved in over 700 indication. However, most of the estimated 8,000 rare diseases, cumulatively effecting approximately 350 million people globally, remain without effective treatments. The pathway from identifying a rare disease to sustainable patient access to an effective treatment is still fraught with significant challenges, some of which are unique to rare diseases. The Rare Disease Special Interest Group offers a platform for ISPOR members to address these challenges in support of patients, clinicians, researchers and drug developers concerned with rare diseases. Join us for a brief presentation of the Special Interest Group and the opportunity to propose and discuss topics that the group should advance or volunteer for any future activities.
Moderators
Sandra Nestler-Parr, PhD, MPhil, MSc
Rare Access, London, United Kingdom
Speakers
Laura Crippa, MPharm
RAReg, Milan, Italy
Mariangela Prada, MSc
INTEXO Srl, Rome, Italy
7:30 - 8:30
EDUCATIONAL SYMPOSIUM
“GO WHERE THE MONEY IS” CAPTURING VALUE ACROSS THE HEALTH CARE SYSTEM
The aim of this educational symposium is to discuss why we should seek value across the health care system and how we can apply existing research methods to measure the value of services. While considerable political attention in developed countries continues to be focused on drug spending, there is also growing awareness of the significant contribution of non-drug components of health care (e.g., hospital services and inefficient care delivery) to overall spending growth and patient affordability. At the same time, there is growing interest in making greater use of value assessment and value-based payment to control spending and better align it with care quality. In order to promote greater value, and to do so in ways that respond to the needs of payers and patients, it is essential to assess value across both drug- and non-drug interventions and health care services. This panel will offer expert viewpoints to identify and discuss gaps in value information, rationale and approaches to track and reduce system-wide low value care, and research methods for how to measure health care services.
Sponsor
Office of Health Economics
Moderators
Bruce Stuart, PhD
University of Maryland School of Pharmacy, Baltimore, MD, USA
Speakers
Beth M Beaudin-Seiler, PhD
Altarum, Ann Arbor, MI, USA
William Padula, PhD
University of Southern California, Los Angeles, CA, USA
Adrian Towse, MA, MPhil
Office of Health Economics, London, United Kingdom
Professor Adrian Towse is director emeritus and senior research fellow of the Office of Health Economics in the UK. Adrian’s current research includes incentives for new drugs and vaccines to tackle Antimicrobial Resistance, the use of 'risk-sharing' arrangements between healthcare payers and pharmaceutical companies, including value-based pricing approaches; the economics of pharmacogenetics for healthcare payers and the pharmaceutical industry; economic issues that affect both R&D for and access to treatments for diseases prevalent in the developing world; the economics of medical negligence; and measuring productivity in healthcare.
A visiting professor at the London School of Economics and a senior researcher at the Nuffield Department of Population Health at the University of Oxford, Adrian also has been a visiting professor at the University of York. For ten years, he served as the non-executive director of the Oxford Radcliffe Hospitals NHS Trust, one of the UK’s largest hospitals. Adrian was president of ISPOR, for the 2014-15 term.
Adrian joined the OHE in 1993 and served as director for 25 years. He holds an MA (Hons) in Politics, Philosophy and Economics from Keble College, Oxford; an MPhil in Management Studies from Nuffield College, Oxford, and the Oxford Centre for Management Studies; and is a member of the Chartered Institute of Management Accountants.
CHALLENGES AND PROMISES OF EARLY CANCER TREATMENT – VALIDATING AND UTILIZING SURROGATE ENDPOINTS IN HEALTH TECHNOLOGY ASSESSMENT
A range of novel therapies to treat early stage cancer have emerged in recent years. While these hold promise for patients, uncertainty about the relationship between surrogate endpoints and long-term survival benefit remains a key barrier in appropriate health technology evaluations and prompt access to these therapies.
This symposium will explore the challenges and opportunities posed by Health Technology Assessment (HTA) based on surrogate endpoints. Speakers will discuss the clinical relevance of surrogate endpoints and their co-relation with long-term outcomes, as well as communication of the meaning and interpretation of surrogate endpoint-based results to patients. Methods and evidence required by HTA agencies and payers to assess the value of early-stage cancer treatment will also be discussed. This will be followed by a demonstration of methodological research aiming to overcome HTA barriers, such as methods of surrogate validation and early prediction of long-term outcomes based on surrogate endpoints. Audience input will be sought to bring forward innovative perspectives and concepts.
Sponsor
MSD
Speaker
Sarah Breen, MA, MSc
MSD, Hoddesdon, United Kingdom
Andrew Briggs, DPhil
University of Glasgow, Glasgow, 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.
Peter Fasching, MD, PhD
University Hospital Erlangen, Friedrich-Alexander University Erlangen-Nuremberg,, Erlangen, Germany
Amin Haiderali, MBBS, MBA, MPH
MSD, North Wales, PA, USA
Min Huang, PhD
MSD, North Wales, PA, USA
8:45 - 10:30
WELCOME AND SECOND PLENARY
UPDATE FROM ISPOR CHIEF SCIENCE OFFICER
Speaker
Richard Willke, PhD
ISPOR, Lawrenceville, NJ, USA
Richard J. Willke, PhD is chief science officer of ISPOR, the leading global professional society for health economics and outcomes research. Dr Willke has more than 25 years of experience in the life sciences arena and has specialized in outcomes research in a succession of group leadership roles with Pfizer and its legacy companies.
At ISPOR, Dr Willke is responsible for designing and implementing strategic initiatives related to scientific research and content priorities that will advance the Society’s mission of promoting health economics and outcomes research excellence to improve decision making for health globally.
Previously, Dr Willke was vice president, Outcomes and Evidence Cluster Lead at Pfizer for its Global Health and Value division. He has also served in a number of leadership roles with affiliated organizations, including the Chair of ISPOR Institutional Council (2010), ISPOR Board of Directors (2007-2009), and Chair of the PhRMA Health Outcomes Committee (2002-2004).
Prior to joining industry, Dr Willke served as department director in the Center of Health Policy Research at the American Medical Association and held research and teaching positions at The Ohio State University.
Dr Willke earned a PhD and MA in economics from Johns Hopkins University. He has authored more than 80 scholarly publications that examine the science and methodologies of health economics and outcomes research.
SECOND PLENARY SESSION- SHAPING THE DIGITAL HEALTHCARE SYSTEM
It has been anticipated that within 20 years, 90% of all healthcare professionals will require some element of digital and genomics skills in order to work within a transformed, data-driven healthcare environment. The rapidly changing environment requires that transformation of healthcare structures, processes, policies, practices is thought through and implemented already now in order to ensure the quality, sustainability, and fairness of the healthcare system. In this session we will explore how the key ‘actors’ in healthcare systems are adapting to the technological developments in healthcare (e.g. genomics, artificial intelligence, digital and real-time medicine, robotics) to deliver more productive, more effective and more personal care for patients. Each speaker will outline the new policies, processes and skill sets they are developing to deliver this digital healthcare system change. The panel will then debate what it takes to deliver the changes required at pace and at scale for the benefit of patients.
Moderators
Petra Wilson
Health Connect Partners, FTI Consulting, Brussels, Belgium
Health Connect Partners helps clients understand the role European health policy environment for the development and roll out of digital health solutions. In addition to managing Health Connect Partners, Petra is engaged as EU Programme Director for Personal Connected Health Alliance, and also acts as senior advisor on digital health for FTI Consulting.
Before setting up Health Connect Partners Petra served as CEO of the International Diabetes Federation; and her prior experience includes eight years in the European Commission in the Digital Health Unit, seven years as Senior Director of Connected Health at Cisco. Prior to these roles she taught healthcare law at Nottingham University, UK
Speakers
Pekka Kahri, MSc
Helsinki University Hopsital, Helsinki, Finland
Pekka Kahri is technology officer at HUS Helsinki University Hospital, which is the biggest public healthcare provider and academic medical center in Finland and one of the leading hospitals in digitalization in Europe. Formerly he has worked as Director of Information Services at THL – National Institute for Health and Welfare, with extensive experience in national health registries and data resources as well as Finland’s national eHealth infrastructure and Kanta services.
Suzanne Schrandt
ExPPect
Thomas Senderovitz
Danish Medicines Agency, HMA Management Group, Copenhagen, Denmark
Dr. Thomas Senderovitz is Director General of the Danish Medicines Agency.
He holds several posts in the EU Medicines Regulatory Network, e.g. chair of the Heads of Medicines Agencies Management Group and member of the European Medicines Agency’s Management Board as well as a member of the EU Telematics Management Board.
In Denmark, he is member of the Steering Committee of the Center for Regulatory Science, University of Copenhagen, of the Reference Group for the Center of Public Leadership, University of Aarhus, of the Board of the Danish Strategy for Personalized Medicine and of the newly established Data Ethics Council.
Thomas is a M.D. from the Faculty of Medicine, University of Copenhagen with more than 26 years’ experience from healthcare, the pharmaceutical industry, biotech, CRO and regulatory authorities. He has held several senior management positions within R&D in PAREXEL International, Grünenthal, UCB and Ferring – in addition, he has founded two biotech companies.
Tristan van Doormaal, MD, PhD
Universitätsspital Zürich, Zurich, Switzerland
Tristan van Doormaal is a vascular and skull base neurosurgeon at the University Hospital Zurich and University Hospital Utrecht. His research focusses on bench to bedside development of new techniques to improve patient outcomes. He is co-inventor of the SELANA technique to perform safe bypass surgeries in the brain and Liqoseal, a patch to prevent cerebrospinal fluid leakage after surgery. He is co-founder of AugmedIT, a startup company aimed at providing surgeon with holograms to better prepare and perform complex surgeries.
WELCOME FROM ISPOR PRESIDENT AND PRESENTATION OF ISPOR BEST CHAPTER AWARDS
Speaker
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.
10:30 - 14:00
RESEARCH POSTER SESSION 3
10:30 - 19:30
EXHIBIT & POSTER HALL HOURS
10:45 - 11:30
ISPOR BOOTH EVENT - ISPOR GLOBAL NETWORKS —MEET THE 2019 ISPOR OUTSTANDING CHAPTERS!
. . .
11:00 - 12:00
BREAKOUT SESSION 5
INFECTIOUS DISEASE STUDIES
IN3: THE HEALTH SYSTEM BURDEN OF SELECTED VACCINE-PREVENTABLE ILLNESSES ON SECONDARY CARE IN ENGLAND- A FIVE-YEAR STUDY USING AN ADMINISTRATIVE HEALTHCARE DATASET
11:30AM - 11:45AM
Rabe AP 1 , Clark-Wright J2 , Carroll S2 , Gray I3 1 Imperial College London, London, LON, UK, 2 Sanofi Pasteur, UK & Ireland, Maidenhead, Berkshire, UK, 3 Sanofi Pasteur, UK & Ireland, Maidenhead, UK
BACKGROUND Vaccines and vaccination have been essential in the control of vaccine-preventable infectious diseases worldwide. However, the legacy of vaccine success is accompanied with a lack of historical memory of the impact of vaccine-preventable diseases, which when coupled with misinformation about vaccines, has the potential to jeopardise public health. OBJECTIVES : This study primarily aims to analyse the health care resource use (HCRU) burden of selected vaccine-preventable diseases on the secondary healthcare system in England. METHODS : We utilised Hospital Episode Statistics (HES), an administrative healthcare dataset covering all secondary care interactions within NHS England, to identify and track the HCRU of patients who have any of the following diagnoses in their record: pertussis, haemophilus influenzae type b (Hib), hepatitis B, diphtheria, poliomyelitis, or tetanus. The index date was set at the first documented diagnosis during the study period (01/04/2013 to 31/12/2018). RESULTS : The study included 75,553 patients with a total of 57,734,216 patient-days as follow-up. Among the selected conditions, Hib and Hep B contributed the most to the overall cost burden in excess of £490m with £312.6m and £135.3m respectively. In the 0-9 year of age cohort (n=8,018), patients that acquired pertussis (n=1,743) and Hib (6,180) were the most common. The majority of patients who acquired poliomyelitis were observed in the 60-89 year of age category (4,272/5,481).Patients that acquired Diphtheria, although small in number (496/75,553), had the highest per patient cost (£11,058) followed by Hib (£8,578). CONCLUSIONS : Our study has demonstrated that although patients that present with a vaccine-preventable disease continue to impose a large burden on NHS resources, there is potential to reduce this cost burden. This could be achieved by optimising vaccination schedules and ensuring that high vaccine coverage rates are maintained to improve public health and reduce NHS burden.
IN1: BREAK THE BRAKES- BRAKES AND INCITEMENTS TO INFLUENZA VACCINATION, A MEDICAL RESIDENTS-CENTERED SURVEY
11:00AM - 11:15AM
Colrat F1 , Abad L2 , Bertin L 3 1 Hospices Civils de Lyon, Lyon 1 University, Lyon, 69, France, 2 Hospices Civils de Lyon, Centre international de recherche en infectiologie, INSERM, Lyon, 69, France, 3 Paris-Saclay University, Chatenay-Malabry, France
OBJECTIVES : Influenza attack rate is commonly accepted to be lower than 2 in community settings, but it has been shown that it could be much higher in hospital settings. This is why vaccination of healthcare workers (HCW) is essential to prevent nosocomial epidemics. The aim of this study was to determine the reasons of such low influenza vaccine coverage rates (VCR) among HCW in France, and to determine its brakes and incitements in the subgroup of medical and pharmacy residents. METHODS : We shared a self-administered 18-item survey to residents in France from 01/25/2019 to 05/31/2019 through professional networks. The inclusion criteria was to be resident in France. We compared the incitements and brakes (both closed and open questions) among vaccinated (VAX) and non-vaccinated (nVAX) residents. RESULTS : Among the 1571 answers, 1541 were included. The VCR was 72.4% (1115/1541). VAX were not more likely to trust vaccine effectiveness (p=0.24) nor safety (p=0.24) than the nVAX. No difference of willingness in being vaccinated was found between the two groups, who were both willing to vaccinate for themselves (VAX; nVAX respectively: 89%, n=994; 80%, n=342), for their immediate social circle (80%, n=891; 80%, n=342) and for patients (90%, n=1003; 72%, n=307). However, we found differences for the brakes: lack of time (15%, n=162, 37%, n=159, p<0.05) and omission (10%, n=108; 33%, n=139, p<0.05) respectively for VAX and nVAX. CONCLUSIONS : As lack of time and omission are the main brakes in the nVAX group it could be very useful to promote vaccination directly in the healthcare services repeatedly in the same season. As the VCR is high for this subgroup, these brakes should be compared to those of HCW with lower VCR in order to increase their VCR. This study is the first to explore VCR in medical and pharmacy residents population in France.
IN2: IMPACT OF TESTING & RAPID INITIATION ON HIV INCIDENCE IN FRANCE- RESULTS OF A TRANSMISSION MODEL
11:15AM - 11:30AM
Pialoux G1 , Delaugerre C1 , Gallien S2 , Gras G3 , Massetti M4 , Leleu H 5 , Blachier M5 , Rodriguez I6 1 Hôpital Tenon APHP, Paris, France, 2 CHU Henri Mondor, APHP, Créteil, France, 3 University Hospital of Tours, Tours, France, 4 Public Health Expertise, PARIS, France, 5 Public Health Expertise, Paris, France, 6 Gilead Sciences, BOULOGNE BILLANCOURT, France
OBJECTIVES : Test & Treat (T&T) policy (WHO) recommends universal cART initiation regardless of CD4 T cell count. We modelled the impact of RI and T&T on the French HIV epidemiology, accounting for faster treatment initiation and virological control as well as reinforced treatment adherence and retention to care. METHODS : We developed a Markov transmission model that simulates the HIV-infected population at a cohort level. Different subgroups of interest have been defined: MSM, heterosexual French and non-French, PWID. Each year, infectious patients with detectable viral loads transmit HIV a number of new subjects based on a “propagation factor” calibrated on the number of new infections every year, thus reflecting the number of at risk acts per infectious patient, condom use and efficacy and HIV status of partners. Newly infected and undiagnosed patients then transit between states based on the time interval in the cascade of HIV care observed in France. Mortality is applied each year by sub-group. Three scenarios based on the RAPID study results were tested: S1 accounts for RI of ART (day 1 vs. 33), S2 accounts for RI of ART and impact on lost-to-follow-up rates (RR=0.69, Rapid Start Study), S3 adds optimized testing and diagnosis of the hidden epidemic (90% by 2020) to these two effects. RESULTS : The current situation (CS) results in a stable incidence of HIV infections between 5,655 and 5,684 new cases annually (years 2018 and 2030, respectively). S1 led to 5,573 new infections in 2030 (-111 [-2.0%] vs. CS). S2 led to 5,470 new infections in 2030 (-214 [-3.8%] vs. CS). S3 led to 3,908 new infections in 2030 (-1,776 [-31.2%] vs. CS). CONCLUSIONS : By reducing the time during which HIV-positive patients are infectious and improving treatment initiation and adherence rates, rapid initiation of ART could prevent over 31% of new HIV infections in France by 2030.
CLINICAL OUTCOMES ASSESSMENT STUDIES
CL2: SYSTEMATIC REVIEW AND METANALYSIS ON THE IMPACT OF MULTI-DRUG RESISTANT BACTERIA ON ECONOMIC AND CLINICAL OUTCOMES OF HEALTHCARE ACQUIRED INFECTIONS
11:15AM - 11:30AM
Serra-Burriel M Centre for Research in Health and Economics - Pompeu Fabra University, Barcelona, Spain
OBJECTIVES: Multidrug resistant (MDR) bacterial infections in hospital settings have substantial implications in terms of clinical and economic outcomes. However, due to the heterogeneity in resistance patterns, findings about the relationships between MDR infections and economic or clinical outcomes are currently under debate. We performed a systematic review and meta-analysis on the impact of MDR on clinical and economic outcomes, considering differences in the specific application, study design, and methodology. METHODS : A literature search was conducted in PubMed/MEDLINE library database to select studies that evaluated the impact of antimicrobial resistance on economic and clinical outcomes, published in between 1980 and 2018. Next, a meta-analysis was performed to establish the adjusted association of MDR hospital acquired infection (HAI) with direct cost of care, prolonged length of stay and mortality. RESULTS : Overall, 23 articles were included in the systematic review of which 12 articles assessed cost, 13 articles assessed length of stay, and 12 assessed mortality. The pooled mean ratios with 95% confidence intervals (95%CI) were 1.55 (95%CI=1.49; 1.62) for direct cost of care was and 1.23 (95%CI=1.20; 1.27) for length of stay, respectively. The hazard rate for mortality was 2.27 (95%CI=2.13; 2.41). Statistical and methodological heterogeneity among the included studies was found to be present. CONCLUSIONS : MDR HAI appears to be strongly associated with increases in direct cost, prolonged length of stay and increased mortality. Further comprehensive studies in this setting are warranted.
CL1: ASSOCIATION BETWEEN DIABETES MEDICATION USE AND CARDIOVASCULAR EVENTS AMONG TYPE 2 DIABETES PATIENTS WITH ADVANCED CHRONIC KIDNEY DISEASE- A NATIONWIDE COHORT STUDY.
11:00AM - 11:15AM
Chang HC 1 , Cheng HM2 , Chang PY3 , Chiang CE2 , Tsai YW4 1 National Yang-Ming University, Taipei, Taiwan, 2 Taipei Veterans General Hospital, Taipei, Taiwan, 3 Stanford University, Stanford, Taiwan, 4 Institute of Health and Welfare Policy, National Yang-Ming University, Taipei, Taiwan
OBJECTIVES : Type 2 Diabetes (T2D) increases the risk of cardiovascular diseases (CVD). However, limited studies demonstrated the risk of CVD related to anti-diabetes medications in adults with T2D and with advanced chronic kidney disease (CKD). This study aimed to compare the risk of CVD and all-cause mortality in adults with T2D and with advanced CKD who received sulfonylurea, dipeptidyl peptidase-4 (DPP-4), thiazolidinedione, α-glucosidase inhibitor or glinide. METHODS : We prospectively studied adults with T2D who used erythropoietin, as an indicator for advanced CKD, from January 1, 2009 to December 31, 2012, in Taiwan’s National Health Insurance Research Database. Follow-up ended on December 31, 2013. We identified 5,415 adults with T2D who received prescriptions of single anti-diabetes medication within 90 days of the first EPO prescription, excluding those with coronary heart disease, stroke, amputation, heart failure, or dialysis in 30 days before the prescription of anti-diabetes medication. The composite primary outcome included CVD (coronary heart disease, stroke, amputation, heart failure) and all-cause mortality. Cox regression estimated hazard ratio with adjustment for age, gender, T2D duration, CVD, Comorbid condition, Antidiabetic medication, Antihypertensive medication, antiplatelet and Statin use . RESULTS : After 1:4 ration propensity score matching, 302 sulfonylurea users, 23 thiazolidinedione users, 61 DPP-4 users and 37 𝛂-glucosidase inhibitor users were matched to 663, 41, 105 and 72 glinide users, respectively. These 2589 matched samples (mean age, 64.5 years; T2D duration, 8.4 years) contributed to an average of 8.5 months (SD=9.8) of follow-up. Multivariable-adjusted hazard ratio for the primary outcome were 0.89 (p=0.11) comparing sulfonylurea with glinide, 0.57 (p=0.17) comparing thiazolidinedione with glinide, 1.17 (p=0.42) for DPP-4 versus glinide, and 1.10 (p=0.68) for 𝛂-glucosidase inhibitor versus glinide. CONCLUSIONS : In adults with T2D and with advance CKD, sulfonylurea, thiazolidinedione, DPP-4 and 𝛂-glucosidase inhibitor seemed to not increase the risk of CVD as compared with glinides.
CL3: THE ASSOCIATION BETWEEN TREATMENT INTENSITY AND ADHERENCE TO LIPID-LOWERING THERAPIES AND CARDIOVASCULAR OUTCOMES IN PATIENTS WITH ATHEROSCLEROTIC CARDIOVASCULAR DISEASE IN PRIMARY AND SECONDARY CARE- A SWEDISH REGISTRY-BASED STUDY
11:30AM - 11:45AM
Leosdottir M1 , Sorio Vilela F 2 , Eriksson Svensson M3 , Villa G2 , Banefelt J4 , Lindh M4 , Rieem Dun A5 , Norhammar A6 1 Skane University Hospital, Malmo, Sweden, 2 Amgen (Europe) GmbH, Rotkreuz, ZG, Switzerland, 3 Uppsala University, Uppsala, Sweden, 4 Quantify Research, Stockholm, Sweden, 5 Quantify Research, Stockholm, AB, Sweden, 6 Karolinska Institutet, Stockholm, Sweden
OBJECTIVES : Assess the association between a combined measure of treatment intensity and adherence to lipid lowering therapies (LLT) and cardiovascular (CV) outcomes in patients with atherosclerotic CV disease (ASCVD) in primary and secondary (specialist) care in Sweden. METHODS : Retrospective observational registry-based study including patients with ASCVD who initiated treatment with a statin and/or ezetimibe between 2001 and 2017 (n=142,529). Patients were allocated to two cohorts depending on the setting of the prescribing physician (primary or secondary care). The exposure variable was a combined measure of treatment intensity and adherence, constructed by multiplying treatment intensity , measured as the expected reduction (%) in low-density lipoprotein cholesterol based on the prescribed LLT, and adherence , measured as proportion of days covered by medication. A Cox proportional hazards model was used to assess the association between the combined measure and a composite endpoint of CV events (myocardial infarction, unstable angina, stroke, revascularization, heart failure and CV-death). RESULTS : The hazard ratios (HR) for a 10% increase in the combined measure were 0.88 (95% CI 0.85-0.91) in primary and 0.88 (0.86-0.90) in secondary care settings; showing a 10-15% reduction in CV event risk for each 10% increase in the combined measure. HR for groups based on the combination of two adherence (adherent, non-adherent) and three intensity categories (high, medium, low) in primary [and secondary] care as compared to a no-treatment group were as follows: high-intensity/adherent 0.55, 0.47-0.64 [0.54, 0.48-0.61], medium-intensity/adherent 0.66, 0.59-0.74 [0.65, 0.59-0.70], high-intensity/non-adherent 0.71, 0.65-0.78 [0.71, 0.60-0.75], low-intensity/adherent 0.73, 0.67-0.80 [0.73, 0.67-0.77], medium-intensity/non-adherent 0.79, 0.74-0.84 [0.79, 0.75-0.82] and low-intensity/non-adherent 0.84, 0.80-0.88 [0.85, 0.82-0.88]. CONCLUSIONS : A combined measure of intensity and adherence to LLT showed a strong and consistent association with CV outcomes reduction both in primary and secondary care settings. Adherence appears to be a key element to reduce CV outcomes beyond treatment intensity.
CL4: MATCHING-ADJUSTED INDIRECT COMPARISON (MAIC) OF THE EFFICACY AND TOLERABILITY OF APALUTAMIDE AND DAROLUTAMIDE FOR THE TREATMENT OF NONMETASTATIC CASTRATION-RESISTANT PROSTATE CANCER (NMCRPC)
11:45AM - 12:00PM
Chowdhury S1 , Oudard S2 , Uemura H3 , Joniau S4 , Liu J5 , Dearden L6 , Sermon J7 , Van Sanden S 7 , Diels J7 , Hadaschik BA8 1 Guy's, King's, and St. Thomas' Hospitals, London, UK, 2 Georges Pompidou Hospital, Paris, France, 3 Yokohama City University Medical Center, Yokohama, Japan, 4 University Hospitals Leuven, Leuven, Belgium, 5 Janssen Scientific Affairs, LLC, Horsham, PA, USA, 6 Janssen Global Services, Raritan, NJ, USA, 7 Janssen EMEA, Beerse, Belgium, 8 University of Duisburg-Essen, and German Cancer Consortium (DKTK), partner site University Hospital Essen, Essen, Germany
OBJECTIVES: Apalutamide (APA) and darolutamide (DARO), both combined with androgen deprivation therapy (ADT), have been compared with ADT in the respective SPARTAN and ARAMIS randomised trials. In the absence of head-to-head trials, both agents are compared indirectly. METHODS: Patient-level data from SPARTAN (first interim analysis [IA1]) and published data from ARAMIS (IA1) were utilized. SPARTAN patients were weighted to match baseline characteristics as reported for ARAMIS in an anchored MAIC, using ADT as the common treatment arm. First, hazard ratios (HRs) for efficacy end-points metastasis-free survival (MFS), progression-free survival (PFS), prostate-specific antigen (PSA) progression and overall survival (OS), and odds ratios for tolerability were re-estimated for SPARTAN using weighted Cox proportional hazards and logistic regression models. Next, the reweighted SPARTAN results were indirectly compared with reported HRs and ORs from ARAMIS using a Bayesian framework. RESULTS: A total of 1,150 SPARTAN patients were included and assigned weights to match the ARAMIS population (N=1,509). MAIC-based HRs (95% confidence interval) for SPARTAN were significant in favor of APA+ADT vs ADT at 0.29 (0.22, 0.38) for MFS, 0.30 (0.23, 0.39) for PFS, 0.06 (0.05, 0.08) for PSA progression, and indicated a favorable trend for OS (HR = 0.75 [0.45, 1.23]). MAIC-based HRs (95% confidence interval) for APA+ADT vs DARO+ADT were 0.70 (0.51, 0.98) for MFS, 0.79 (0.59, 1.08) for PFS, 0.46 (0.33, 0.64) for PSA progression, and 1.05 (0.58, 1.93) for OS. The Bayesian probabilities of APA+ADT being more effective than DARO+ADT were 98.3% for MFS, 93.1% for PFS, ~100% for PSA progression, and 43.5% for OS. The MAIC results indicated comparable tolerability profiles for APA and DARO. CONCLUSIONS: Based on available data and Bayesian probabilities, results suggest that nmCRPC patients treated with APA+ADT had more favorable MFS, PFS, and PSA progression than those who received DARO+ADT with comparable tolerability of the treatments.
PATIENT ENGAGEMENT IN HEALTH RESEARCH- VALUE OF LESSONS LEARNED IN HTA AND POSSIBILITIES FOR CROSS CONTEXT TRANSFER OF EXPERIENCE
PURPOSE: Can the rest of the world replicate the European Patients Academy (EUPATI) approaches to HTA, pioneered in European Union to educate patients in healthcare research and development as we roll out universal health coverage for the rest of the world. Is HTA definition universal and can that be adapted for all healthcare settings?Is this guidance specific to the institutional, legal, policy, practice and standards infrastructural arrangement of the EU or can it be adopted anywhere globally?
DESCRIPTION: The discussants will describe if there are cultural and structural dimensions which support or restrict patient engagement in the health technology assessment process in low- and middle-income countries. Specific issues to be discussed will be:
Consultations with patient organizations: Is there a barrier when inviting and involving patient organizations? Is there a difference in how European and LMIC patients react when invited for written evidence base submissions? Can we ensure equity and inclusiveness when inviting oral submissions at committee meetings? Providing access and support: How can health policy makers and researchers provide support and encourage uptake of templates, guidance documents, and preparing to act as patient experts at meetings; provide language and health literacy that is sensitive & easy to read summaries of documentation sent out ahead of individual HTAs? Improve LMIC free access for patients to any original publications that will form part of the HTA evidence? Identifying and prioritizing which technologies to assess for developing a system for patients to nominate technologies for HTA. In addition, the panel will invite discussion on: How can we optimise and summarize patient input in HTA outcome documents, and how it was used in reaching the final recommendation? Is there a need to develop and disseminate a clear system for patients to appeal HTA decisions and involve patients in the review of patient involvement processes?
Discussion Leader
Tamás Bereczky, PhD Social Psychology
European Patients Academy for Therapeutic Innovation (EUPATI), Berlin, Germany
Neda Milevska Kostova, PhD
CRPRC Studiorum, Skopje, Macedonia
Kawaldip Sehmi, Msc. Public Health, MBA LLB
International Alliance of patients' Organisations, London, United Kingdom
Wendy Wiedner, BA, MA
Alzheimer’s Disease International, London, United Kingdom
ESTIMATING THE OPPORTUNITY COST BASED COST EFFECTIVENESS THRESHOLD- EMPIRICAL APPROACHES AND THE POLICY CHALLENGES FOR PAYERS
PURPOSE
: 1) Illustrate how empirical work can inform cost-effectiveness thresholds based on opportunity cost 2) Discuss the challenges for HTA bodies and payers in selecting a threshold
DESCRIPTION
: Cost-effectiveness thresholds are a key element of decision making in many countries. It is now recognised that these thresholds need to be grounded in evidence about the improvements in health that could be achieved by using resources for other activities in the health system (opportunity costs), and research is underway in several countries to provide that evidence. We will reflect on whether there is any emerging consensus on good practice in empirical work of this kind, and consider the views of HTA and payer bodies about what evidence they think should inform the choice of a threshold. Professor Nancy Devlin – why is this important? (10 mins) – Nancy will emphasise the importance of exploring the empirical basis for cost-effectiveness thresholds based on opportunity cost and overview approaches that might be taken to inform this kind of work, highlighting challenges and limitations. Dr James Lomas – methods for estimation (10 mins) – James will draw good practice lessons for using econometric analysis to estimate health opportunity costs. Dr Martin Henriksson – estimation and impact in Sweden (10 mins) – as both a researcher and a decision maker on behalf of TLV, Martin will explore lessons from work in this area in Sweden. He will focus on how the opportunity cost work can inform trade-offs concerning severity of disease and rarity, both explicitly considered in Swedish healthcare prioritisation. Danny Palnoch – how is this evidence used? (15 mins) – as Head of Medicines Analysis for NHS England, Danny directly inputs into decisions concerning the ~£17.4 billion spent on medicines in England. Danny will highlight challenges associated with policy using this evidence and suggest future research.
Discussion Leader
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.
Martin Henriksson, PhD
Linköping University, Linköping, Sweden
James Lomas, PhD
University of York, Heslington, York, United Kingdom
Danny Palnoch, BSc
NHS England, London, United Kingdom
CROSS-BORDER COLLABORATION ON AVAILABILITY OF PHARMACEUTICALS IN EUROPE – WHAT, WHY AND WHAT IF?
Cross-border collaboration on joint HTA or joint pricing negotiations are not new initiatives, however in order to truly embrace the concept, and succeed with the proposed transition to a new practice, one needs to have a clear understanding of the path to ultimate outcome and the envisioned impact of the change efforts. The current European cross-border collaboration initiatives claim to offer better access to pharmaceuticals for European patients, and have gathered a wealth of practical experience with individual projects. The participants will update and reflect on recent developments in the cross-border initiatives in the context of the transformation of healthcare systems that is accelerated by digital technologies. The session will also address the following questions: What objectives of cross-border collaboration are shared by all affected? What will cross-border collaboration require individual countries to give up? For whom and why the proposed change in the current practice of HTA, price negotiation, procurement is necessary? What will happen if we stick to the status quo in the European countries’ individual practices? What is needed to move beyond joint discussions and “agreement to agree/disagree”?
Moderators
Sarah Garner, PhD
World Health Organization (WHO), Copenhagen, Denmark
Panelists
Francis Arickx, Pharm.D
National Institute for Health and Disability Insurance (RIZIV-INAMI), Brussels, Belgium
Dominik Dziurda, MBA
Agency for Health Technology Assessment and Tariff System (AOTMiT), Warsaw, Poland
Edith Frénoy
EFPIA, Brussels, Belgium
Flora Giorgio, PhD
European Commission, Brussels, Belgium
Niklas Hedberg, MSc, Pharmacy
The Dental and Pharmaceutical Benefits Agency (TLV), Stockholm, Sweden
TRANSPARENCY IN RWE - CAN WE NAVIGATE THE KEY CHALLENGES?
This session will provide a brief overview of the RWE Transparency Initiative and then focus on several of the key challenges in moving it forward. These challenges include: establishing a protocol template this inclusive enough to be clearly specify the hypothesis(es) to be tested and the analytics to be used; the conditions needed to ensure trust that any prior analyses of the data in question did not provide prior information about the hypotheses being tested; and the external incentives that could help lead to registration of RWD study protocols, and the posting of their results, becoming common practice.
Moderators
Richard Willke, PhD
ISPOR, Lawrenceville, NJ, USA
Richard J. Willke, PhD is chief science officer of ISPOR, the leading global professional society for health economics and outcomes research. Dr Willke has more than 25 years of experience in the life sciences arena and has specialized in outcomes research in a succession of group leadership roles with Pfizer and its legacy companies.
At ISPOR, Dr Willke is responsible for designing and implementing strategic initiatives related to scientific research and content priorities that will advance the Society’s mission of promoting health economics and outcomes research excellence to improve decision making for health globally.
Previously, Dr Willke was vice president, Outcomes and Evidence Cluster Lead at Pfizer for its Global Health and Value division. He has also served in a number of leadership roles with affiliated organizations, including the Chair of ISPOR Institutional Council (2010), ISPOR Board of Directors (2007-2009), and Chair of the PhRMA Health Outcomes Committee (2002-2004).
Prior to joining industry, Dr Willke served as department director in the Center of Health Policy Research at the American Medical Association and held research and teaching positions at The Ohio State University.
Dr Willke earned a PhD and MA in economics from Johns Hopkins University. He has authored more than 80 scholarly publications that examine the science and methodologies of health economics and outcomes research.
Panelists
Andrew Bate, phD
Pfizer, Dorking, Surrey, United Kingdom
Marc Berger, MD
Pfizer, Inc., New York, NY, USA
C. Daniel Mullins, PhD
University of Maryland, School of Pharmacy, Baltimore, MD, USA
C. Daniel Mullins, PhD is a professor and chair of the Pharmaceutical Health Services Research Department at the University of Maryland School of Pharmacy. He is founder and executive director of the University of Maryland PATient-centered Involvement in Evaluating effectiveNess of TreatmentS (PATIENTS) Program, an infrastructure to support patient-centered outcomes research and related training activities. He also serves as director of the Community and Collaboration Core within the university’s Institute for Clinical and Translational Research. Dr. Mullins has received funding as a principal investigator from the NIH/NIA, NIH/NHLBI, AHRQ, the Patient-Centered Outcomes Research Institute (PCORI), and various patient advocacy and industry organizations. He has served as a regular member of AHRQ and NCI Study Sections and has chaired PCORI Study Sections. Professor Mullins is one of two editors-in-chief for Value in Health and is author/co-author of more than 225 peer-reviewed articles. He has received an Outstanding Service Award from the Drug Information Association (DIA) and two Service Awards from ISPOR. In 2007, he received the Dr. Patricia Sokolove Outstanding Mentor Award from the University of Maryland Baltimore campus-wide Graduate Student Association. In 2013, he was the recipient of the Dr. Daniel D. Savage Memorial Science Award, the Association of Black Cardiologists’ most prestigious annual award. Also in 2013, he was awarded a University System of Maryland Wilson H. Elkins Professorship. He was named Researcher of the Year in 2014 and received the Martin Luther King Faculty Diversity Award in 2017 for the University of Maryland Baltimore campus and the ISPOR Marilyn Dix Smith Leadership Award.
Shirley Wang, PhD
Brigham and Women's Hospital and Harvard Medical School, Boston, MA, USA
HOW CAN WE CREATE HEADROOM FOR HIGH-VALUE CARE BY INCREASING EFFICIENCY IN HEALTH SYSTEMS?
ISSUE
: The OECD reports up to one-fifth of healthcare spending is wasted (such as through unnecessary treatments, delayed discharges or errors), making no meaningful contribution to health outcomes. If these resources could be identified, released and reallocated, greater gain could be achieved from our over-stretched health care budgets. This would allow greater improvements in health and quality of life, as well as improvements in quality of health care, without increases in expenditure.
Increased efficiency would also create headroom for innovation and high-value interventions, including from innovative medical technologies and services, which bring real value for patients.
OVERVIEW
: Short innovative case studies that cover sources of inefficiency across different health systems in Europe will be presented. Case studies will include screening, procurement and use of biosimilars, amongst others, and will demonstrate how inefficiencies can arise, explore various potential solutions and suggest the magnitude of benefit that could be achieved through reductions in inefficiency. The case studies are highly topical as policymakers are beginning to pay closer attention to the challenge of inefficiency. Implementation narratives will also be presented, through which the potential for implementation of the solutions have been explored with stakeholders across five European countries. Recommendations and next steps will be provided. The panel is designed to be beneficial to a wide variety of stakeholders by reporting on and debating multi-stakeholder issues and solutions. Indeed, all stakeholder groups will be required to work together to tackle the system-wide challenge of reducing inefficiency. The overview of the case studies and implementation narratives will be approximately 10 minutes, with 30 minutes for panellists’ perspectives and 20 minutes for audience discussion.
Moderators
Maria Errea, PhD
The Office of Health Economics, London, LON, United Kingdom
Panelists
Stefan Gijssels, M.A.
Digestive Cancers Europe, Brussels, Belgium
Douglas Gregory, MBA
Amgen, Brussels, Belgium
Carlos Mur, MD, PhD
SEDISA Hospital Universitario de Fuenlabrada, Madrid, Spain
12:00 - 13:30
LUNCH IN THE POSTER & EXHIBIT HALL
12:30 - 13:30
EDUCATIONAL SYMPOSIUM
CAN WE AFFORD CURATIVE THERAPIES WITHOUT SHARING RISKS? PERSPECTIVES ON HTA AND AFFORDABILITY IN THIS NEW INNOVATION ERA
Gene therapy, ATMPs (advanced therapy medicinal products), or personalized medicine—all these buzz words are now part of the day-to-day reality in our industry. With several recent regulatory approvals across the globe, discussion has ensued in earnest on how to assess their value and whether traditional reimbursement systems and health economic methods are fit-for-purpose to meet the unique challenges being encountered. These challenges center less on whether the innovations provide value, and more on affordability in our current short-term global financing systems. To date, manufacturers have proactively addressed these topics with payers in various markets through pay-for-performance arrangements, milestone payments over various durations, and other novel payment schemes. All these developments over the short- and mid-term will lead to significant changes in how health care systems assess innovations during Health Technology Assessment pathways. Further, they will impact how financing systems evolve, especially with respect to risk sharing on potential outcomes. This symposium aims to provide insights on how payers and manufacturers across a number of important markets are currently dealing with these challenges.
Sponsor
Xcenda
Moderators
Jay Jackson, PharmD, MPH
Xcenda, Palm Harbor, FL, USA
Speakers
Stève Bénard, PharmD
Stève Consultants, Rhône-Alpes, France
Christian A Hill, BSc
MAP BioPharma, Cambridge, United Kingdom
Paola Lanati
MA Provider, Milan, Italy
Thomas Mittendorf, PhD
Xcenda GmbH, Hannover, Germany
12:30 - 13:45
ISPOR FORUMS
ISPOR HEALTH PREFERENCE RESEARCH SPECIAL INTEREST GROUP - CHOICE DEFINES VALUE- DECISIONS, STEPS, OBSTACLES AND OUTREACH IN HEALTH PREFERENCE RESEARCH
A concern of patients, clinicians, and regulatory agencies alike is understanding, informing, and supporting decisions about health care interventions. Health preference studies use observational and experimental methods to collect empirical evidence on preferential choice behaviors related to health. Therefore, health preference research (HPR) refers to any investigation dedicated to understanding the value of health and health-related alternatives using observational or experimental methods. The HPR Special Interest Group (HPR SIG) at ISPOR is actively promoting good research practices as part of broader initiatives. Outreach is the primary bottleneck in HPR and the purpose of this Forum. This Forum will start with a how-to guide of the major decisions, crucial steps, and obstacles involved in developing and conducting a health preference study. Using this foundation, the Forum will engage the participants regarding ongoing endeavors at ISPOR toward enhancing good practices, such as short courses, networking and checklists, in HPR.
Moderators
Karin Groothuis-Oudshoorn, Phd
Faculty of Behavioral, Sciences, University of Twente, Enschede, Netherlands
Speakers
Benjamin M Craig, PhD
University of South Florida, Tampa, FL, USA
Axel Mühlbacher, PhD, MBA
Hochschule Neubrandenburg, Neubrandenburg, BW, Germany
Juan Manuel Ramos-Goni, MSc, PhD
Axentiva Solutions, Tacoronte, Spain
Oliver Rivero-Arias, PhD, MSc
University of Oxford, Oxford, United Kingdom
ISPOR STATISTICAL METHODS IN HEALTH ECONOMICS AND OUTCOMES RESEARCH SPECIAL INTEREST GROUP- WHERE HAVE WE BEEN? WHERE ARE WE GOING?
This session is open to all attendees interested in statistical methods used in the health economics and outcomes research (HEOR) field. Grab your lunch and join us for a brief presentation on the special interest group’s (SIG) activities and ways to get involved. We will discuss the findings from the current project on missing data in HEOR, review topics for upcoming webinars, and discuss opportunities to contribute to the SIG’s member engagement projects such as the update to the ISPOR Book of Terms. This is intended to be an informal session to encourage interaction and open discussion between SIG members.
Moderators
Rita M. Kristy, MS
Astellas Pharma Global Development, Northbrook, IL, USA
Speakers
Gianluca Baio, PhD
University College London, London, United Kingdom
Necdet Gunsoy, PhD
GSK, Uxbridge, BKM, United Kingdom
ISPOR GLOBAL GROUPS- USE OF MCDA IN HTA, COVERAGE AND REIMBURSEMENT DECISION-MAKING- EXPERIENCE AND INSIGHTS FROM EMEA, LATIN AMERICA AND ASIA-PACIFIC
MCDA improves objective decision-making by evaluating multiple, sometimes conflicting criteria. Its potential applications in health care decisions have been discussed widely, especially to support health technology assessment (HTA) and appraisal processes. A growing interest in the use of MCDA in HTA and supporting health coverage and reimbursement decisions has been observed across ISPOR global regions. In this session, the expert speakers from Latin America, Asia-Pacific and EMEA will share experiences and insights of MCDA application in HTA and coverage decision making by presenting case studies. They will also discuss key issues, challenges, lessons learned, and future potential of MCDA across ISPOR global regions.
Moderators
Zoltan Kalo, PhD
Semmelweis University and Syreon Research Institute, Budapest, 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.
Speakers
Ramiro Gilardino, MD,MSc
ISPOR, Lawrenceville, NJ, USA
Ataru Igarashi, PhD
Yokohama City University School of Medicine, Yokohama, Japan
Ivett Jakab, MSc
Syreon Research Institute, Budapest, Hungary
ISPOR REAL WORLD EVIDENCE TRANSPARENCY INITIATIVE PARTNERSHIP- TRANSPARENCY IN RWE - MOVING FORWARD
This panel will briefly review the Real World Transparency Initiative and the issues discussed in the prior Spotlight and Issue Panel sessions focused on this Initiative. It will focus on the recommendations of the white paper, especially the near-term, medium-term and long term goals and the efforts needed to achieve them, as well as on the major areas of comments received on the white paper. Significant time will be allotted for audience comments and discussions.
Speaker
Bart Barefoot, JD
GlaxoSmithKline, Brentford, United Kingdom
William Crown, PhD
Optum Labs, Cambridge, MA, USA
Pall Jonsson, BS, MRes, PhD
National Institute for Health and Care Excellence, Manchester, United Kingdom
Nirosha Mahendraratnam, PhD, MSPH
Duke-Robert J. Margolis Center for Health Policy, Washington, DC, USA
ISPOR BIOSIMILARS SPECIAL INTEREST GROUP AND ISPOR CENTRAL AND EASTERN EUROPE CONSORTIUM- BIOSIMILARS- AN OPPORTUNITY FOR COUNTRIES WITH RESTRICTED RESOURCES TO IMPROVE PATIENT ACCESS?
PURPOSE: To present an overview of and discuss the goals, barriers and facilitators of biosimilar adoption in countries with limited resources, from HTA and policy perspectives. DESCRIPTION: Value propositions of biosimilars are often approached from a perspective of price erosion, but they can also generate health gain. Utilising biosimilars in different decision settings necessitates scientifically rigorous yet flexible value assessment methods. Sustainable policies are also required to translate value propositions of biosimilars to real-world cost savings or health gain. Discussions will include: Dr. Inotai - current challenges facing biosimilars’ uptake from a CEE perspective; Ms. Moorkens - existing European biosimilar policies; Dr. Lipska - CEE HTA/Payer perspective and challenges in pricing negotiations on biosimilars; Dr. Dawoud - how the outputs of the SIG can provide guidance for biosimilars’ HTA and policy decision-making. Live poll and interactive discussions during the presentations and open discussion at the end will facilitate audience participation.
Moderators
Vera Pataki, MD, MBA
Medicines for Europe, Brussels, Belgium
Speakers
Dalia Dawoud, MSc, PhD
NICE, London, LON, United Kingdom
Andras Inotai, PharmD, PhD, DrHabil
1) Center for Health Technology Assessment, Semmelweis University; 2) SYREON Research Institute, Budapest, Hungary
Iga Lipska, MD, PhD
National Health Fund, Warsaw, Poland
Evelien Moorkens, MSc
University of Leuven, Leuven, Belgium
ISPOR PERSONALIZED PRECISION MEDICINE SPECIAL INTEREST GROUP- TRANSLATING GENOMIC TECHNOLOGIES INTO CLINICAL PRACTICE – ARE WE FALLING SHORT? – WHAT ARE THE CHALLENGES? OVER-COMING THE CHALLENGES? – ARE WE INADVERTENTLY CREATING DISPARITIES ...
Next-generation sequencing, targeted cancer products, even commercial companies will provide genetic profiles for consumers. The availability of genomic data and health information through the digital technologies has the potential to greatly benefit health. However, how do we optimize the integration of this information into clinical practice? Are we inadvertently creating disparities and inequities in health care? We have four presenters that will discuss these questions from a practical standpoint, each from a different perspective including a community-based, safety net hospital system with a large rural population, a market access consultant perspective, and a patient perspective. Additionally, the panel will discuss recent research on patient preferences. During the meeting, attendees will have the opportunity to share their perspectives and challenges in translating genomic information into clinical practice.
Moderators
Emily Reese, PhD, MPH
Levine Cancer Institute, Atrium Health, Charlotte, NC, USA
Speakers
Jan Geissler, MBA
Co-founder and committee member, Acute Leukemia Advocates Network and CML Advocates Network, Switzerland, Germany
Gavin Outteridge, MA
AESARA, London, United Kingdom
Eline van Overbeeke, MSc
KU Leuven, Leuven, Belgium
13:00 - 14:00
POSTER AUTHOR DISCUSSION HOUR - SESSION 3
Attendees have the opportunity to interact with poster presenters during this hour-long session.
14:15 - 15:15
BREAKOUT SESSION 6
LOOKING BEYOND STATISTICAL ADJUSTMENT TO UNTANGLE THE EFFECTS OF SUBSEQUENT TREATMENTS SELECTED BY INVESTIGATORS IN ONCOLOGY TRIALS (Advanced Workshop)
PURPOSE: To discuss potential solutions to confounding due to imbalances in subsequent therapies selected by investigators after discontinuation of study treatments, focusing on the need to look beyond statistical adjustment and considering complimentary approaches like disease modelling and innovative trial design.
DESCRIPTION: Initiation of new lines of therapies after discontinuation of study treatments is necessary for most patients in cancer trials. The choice of subsequent treatments is usually made by investigators based on their prognosis of the patient from locally available choices. It is, therefore, not unusual that subsequent treatments differ within and between studies, leading to confounded comparisons of OS. Statistical methods are available to adjust for this bias, but applications of these have often resulted in no or minor changes in hazard ratios, even in cases with evidence to the contrary. Furthermore, the applicability of adjusted results for decision making requires that the analyses allows the adjustment of subsequent treatment patterns to reflect local treatment guidelines and apply long-term. Reliance on these assumptions can be reduced in a sequential modelling framework by explicitly tracking treatment sequences to reconcile treatment patterns in trials and reflect clinical practice. Data requirements for such detailed modeling are substantial, ideally tracking many patients through each line of treatment to capture their continuous sequence, including delays in initiation of new treatments and accounting for the role of prior treatments. Challenges with either approach relate back to the design of clinical trials, which seem poorly suited for assessment of OS in a context where switching is inevitable. Innovative trial designs like trials nested within cohorts (TwiCs) are available to offer potential solutions.
Discussion Leader
Keith Abrams, PhD, MSc
University of Leicester, Leicester, United Kingdom
K. Jack Ishak, PhD
Evidera, Montreal, QC, Canada
Noemi Muszbek, MSc
Visible Analytics, Reading, RDG, United Kingdom
STATED PREFERENCE & PATIENT SATISFACTION RESEARCH
SP3: PATIENTS′ PREFERENCES FOR BREAST CANCER TREATMENTS- RESULTS OF A DISCRETE CHOICE EXPERIMENT (DCE) SURVEY FROM SPAIN, FRANCE, POLAND AND IRELAND
2:45PM - 3:00PM
Konstantopoulou T , Pacheco R Novartis Oncology Region Europe, Origgio (VA), Italy
OBJECTIVES : Patients′ involvement is becoming increasingly important by regulatory and Health Technology Assessment Organisations around Europe. The survey objectives′ were to understand breast cancer patients′ perspective when choosing their treatment, highlight treatment characteristics valued as most important by patients and gain information on patients′ trade-offs between available treatments. METHODS : A DCE survey of breast cancer patients was conducted in Spain, Poland, France and Ireland. The survey design included: literature review, qualitative research, cognitive debriefing, data collection and analysis. A panel of experts from participating countries (physicians, patient group representatives, ex-payers) selected the survey treatment attributes: Progression free survival (PFS), Febrile neutropenia (FN), Pain, Functional well-being (FWB) and Out-of-pocket payment (OPP). Patients selected (or opted-out) between two unlabeled hypothetical treatments containing the previously mentioned five attributes. Panels of breast cancer patients were recruited as respondents and the questionnaire was deployed through a website platform. A D-efficient experimental design with 16 choice-sets was constructed and a conditional logistic model was used to estimate patients’ preferences. Marginal rates of substitution (MRS) was used to estimate out-of-pocket payments. RESULTS : 371 breast cancer patients completed the survey. FWB and pain were the most important attributes chosen by patients from all countries. More specifically, Spanish, Polish and Irish patients were willing to offer €34,753, €45,712 and €32,222 respectively as out-of-pocket payments for moving from severe to no impairment in FWB. Spanish patients would pay €813 annually for one additional PFS month while Irish patients would pay €1,219.50 and Polish patients €2.575 respectively. French patients’ strongest preference was no pain and they were willing to pay €15,017 for moving from severe to no pain level. CONCLUSIONS : Breast cancer treatments that improve FWB, pain and prolong PFS can be considered preferred treatments from patients’ perspective. Patients’ preferences should be incorporated in regulatory, HTA and industry decision-making processes.
SP1: COMPARISON OF GENERIC AND CONDITION-SPECIFIC PREFERENCE MEASURES TO DERIVE HEALTH UTILITIES- A RETROSPECTIVE ANALYSIS OF NIVOLUMAB TRIALS IN SOLID TUMOURS
2:15PM - 2:30PM
Shaw JW 1 , Bennett B2 , DeRosa M3 , Taylor F3 , Kiff C2 , Ntais D2 , Noon K2 , King MT4 , Cocks K5 1 Bristol-Myers Squibb, Lawrenceville, NJ, USA, 2 Bristol-Myers Squibb Pharmaceuticals Ltd, Uxbridge, UK, 3 Adelphi Values, Boston, MA, USA, 4 University of Sydney, Sydney, Australia, 5 Adelphi Values Ltd, Bollington, UK
OBJECTIVES: There is growing interest in using condition-specific preference measures, including the European Organization for Research and Treatment of Cancer Quality of Life Utility Measure-Core 10 Dimensions (EORTC QLU-C10D), to inform economic evaluations of cancer treatments. This research assessed the implications of using utility indices based on the 3-level EQ-5D (EQ-5D-3L), a mapping of the EQ-5D-3L to the 5-level EQ-5D (EQ-5D-5L), and the QLU-C10D and compared the psychometric properties of the indices. METHODS: Utilities for progression-related states were estimated for 8 phase 3 trials of nivolumab±ipilimumab using the EQ-5D-3L UK and US value sets, mapped EQ-5D-5L English value set, and QLU-C10D UK and Australian value sets. These were entered into UK cost-effectiveness models to derive quality-adjusted life years (QALYs) for treatments. Psychometric properties of the indices were assessed using pooled trial data. RESULTS: The mapped EQ-5D-5L English index yielded greater incremental QALYs for nivolumab±ipilimumab vs comparators than the EQ-5D-3L UK and QLU-C10D UK indices. Absolute differences in incremental QALYs ranged from 0.01-0.16 (mapped EQ-5D-5L English vs EQ-5D-3L UK), 0.04-0.31 (mapped EQ-5D-5L English vs QLU-C10D UK), and 0.02-0.15 (EQ-5D-3L UK vs QLU-C10D UK). All indices differentiated groups defined by baseline performance status (PS), EQ-5D visual analogue scale (VAS) score, and cancer stage. Similarly, all indices detected meaningful change in tumour response, VAS score, and PS. Effect sizes were generally similar in interpretation, though the QLU-C10D UK index was more sensitive to worsened EQ-5D VAS score and PS, while the mapped EQ-5D-5L English index was less sensitive to improved tumour response. CONCLUSIONS: On average, the mapped EQ-5D-5L English index and QLU-C10D UK index yielded 6% more and 2% fewer incremental QALYs, respectively, than the EQ-5D-3L UK index. The lower QALY yields of the QLU-C10D were balanced by slightly greater validity and responsiveness. The implications of these findings for use of the EQ-5D-5L and QLU-C10D are discussed.
SP4: EXPLORING HETEROGENEITY IN PREFERENCES FOR ATTRIBUTES OF PHARMACEUTICAL TREATMENTS FOR OSTEOARTHRITIS AND CHRONIC LOW BACK PAIN IN THE UNITED KINGDOM- A LATENT CLASS APPROACH
3:00PM - 3:15PM
Boeri M 1 , Abraham L2 , Hauber B3 , Atkinson J2 , Bushmakin AG4 , Cappelleri JC5 , Russo L6 , Viktrup L7 , Walsh D8 , Turk D9 1 RTI Health Solutions, Belfast, UK, 2 Pfizer, LTD, Surrey, UK, 3 RTI Health Solutions, Research Triangle Park, NC, USA, 4 Pfizer Inc, Groton, CT, USA, 5 Pfizer, Inc, Groton, CT, USA, 6 Pfizer, Inc, Collegeville, PA, USA, 7 Eli Lilly and Company, Indianapolis, IN, USA, 8 University of Nottingham, Nottingham, UK, 9 University of Washington, Seattle, WA, USA
OBJECTIVES : Our primary objective was to quantify patients’ preferences for attributes of pharmaceutical treatments for chronic pain (i.e., associated with osteoarthritis [OA] of the hip or knee and chronic low back pain [CLBP]) in the United Kingdom. Our secondary objective was to explore preference heterogeneity. METHODS : A discrete-choice experiment was administered online to respondents with a self-reported physician diagnosis of OA and/or CLBP and moderate-to-severe pain. Respondents were presented with a series of choices between two hypothetical treatments, defined by six attributes: effectiveness (pain and symptom control); associated risks (i.e., rapidly progressive osteoarthritis [RPOA], heart attack, physical dependence); mode (pill vs. injection) and frequency of administration; and cost. Latent class analysis was used to segment preferences in the sample, comparing them with results for the average respondent. RESULTS : The survey was completed by 437 respondents: 171 with OA, 188 with CLBP, and 78 with OA and CLBP. Mean (SD) age was 54.4 (14.3) years. On average, results suggested the following attribute relative importance (mean [SE]): cost (2.6 [0.2]), pain and symptoms control (2.0 [0.2]), risk of physical dependence (1.2 [0.2]), risk of heart attack (0.4 [0.1]), risk of RPOA (0.4 [0.1]), and mode and frequency of administration (0.2 [0.1]). The latent class analysis identified four classes: one class focused on efficacy (33.7%), one on cost (29.4%), one on avoiding risk of physical dependency (19.6%), and one on mode and frequency of administration (17.3%). CONCLUSIONS : On average, treatment choices were primarily driven by cost, improving efficacy, and avoiding physical dependence. However, we found evidence of preference heterogeneity; that is, different patients focused on different treatment characteristics, suggesting that patient groups may have different priorities when considering treatments for chronic pain. Discussions between doctor and patient may be particularly important to provide information about a treatment aligned with the patient’s preferences.
SP2: DISCREPANCY IN HEALTH STATE UTILITY VALUES BETWEEN COST-UTILITY ANALYSIS AND REFERENCED ORIGINAL HEALTH UTILITY STUDIES IN CARDIOVASCULAR DISEASE- A REGISTRY-BASED ANALYSIS
2:30PM - 2:45PM
Zhou T1 , Chen Z1 , Li H2 , Xie F 1 1 McMaster University, Hamilton, ON, Canada, 2 China Pharmaceutical University, Nanjing, China
OBJECTIVES: Cardiovascular disease (CVD) is the leading cause of noncommunicable disease deaths worldwide. CVD is associated with significant financial and humanistic burden. Cost-utility analysis (CUA) uses quality-adjusted life (QALY) as the outcome measure and is commonly used in CVD. Health state utility values (HSUVs) used in calculating QALYs play an important role in determining cost effectiveness of CVD treatments. The objective was to compare HSUVs between CUAs and referenced original HSUV studies in CVD. METHODS: Tufts Cost-Effectiveness Analysis Registry is a comprehensive database including more than 7,000 CUAs studies published since 1976. We used the primary disease filter to identify all CUA studies focusing on CVD included in the registry. We reviewed and extracted original HSUV studies cited in the CUAs whenever available. The descriptions and values of health states used in the CUA were compared with those reported in the original HSUV studies to evaluate the consistency in using published HSUVs. RESULTS: A total of 577 CVD CUAs published between 1982 and 2016 were included in the analysis. Out of 3358 health states extracted, 642 were collected as part of the CUA and 2278 obtained from external sources. The EQ-5D (50.8%) and time trade-off (TTO) (29.1%) were the most frequently used indirect and direct methods to measure HSUVs, respectively. For the 2278 health states, only 574 (25.2%) had the same health state description and value as those in the original sources. 990 health states (43.5%) differed in both description and value for which the majority did not provide any explanation for the discrepancies. CONCLUSIONS: Discrepancy in the description and value for the health states between the CUA and referenced health utility studies remain an important issue in CVD. Any such discrepancy, if not justified, could affect the validity of cost effectiveness results.
A FORMAL SYSTEM FOR USING REAL-WORLD EVIDENCE TO REVISIT THE HTA DECISION- MORE TROUBLE THAN IT’S WORTH?
ISSUE
: Many European health technology assessment (HTA) processes make decisions for pharmacological therapies based on short-term randomised controlled trial (RCT) evidence, which is assumed to be generalisable to real-world settings and extrapolated under uncertainty to provide long-term estimates. The increasing availability of real-world evidence (RWE) provides opportunities to use such evidence to test these assumptions and uncertainties in a formal post-adoption HTA process, yet currently RWE collected post-adoption rarely informs subsequent HTAs – the Cancer Drugs Fund in England potentially being one example exception. A world can be imagined where RWE is routinely utilised to inform formal post-adoption HTA, to confirm initial decisions taken under uncertainty or in other ways (for example to inform dynamic adjustment of value-based prices). However, questions remain over the desirability, structure and practicality of such processes, and stakeholders may have divergent views on the merits, opportunities and challenges of moving to a formal system of post-adoption HTA based on RWE. With RWE representing one of ISPOR’s top 10 HEOR trends, now is the time for this discussion.
OVERVIEW
: Matt Griffiths will moderate discussion and challenge the panellists to discuss whether HTA processes should be adjusted to formally use RWE for post-adoption re-appraisal and present their vision for how such a system might look. Adrian Towse will provide an academic perspective, outlining the theoretical pros and cons of a formal system of post-adoption HTA informed by RWE in the context of the European HTA paradigm that includes national HTA bodies and EUnetHTA. Jeanette Kusel and Anna Halliday will provide the HTA and industry perspective, respectively, on the desirability of such a system, the opportunities offered and the practical and political barriers to its implementation. Following presentations, audience members will have opportunity to vote on the issue panel title question and put their questions to the panellists.
Moderators
Craig Brooks Rooney, MA
Costello Medical Singapore Pte Ltd, Singapore, Singapore
Panelists
Anna Halliday, MA (Oxon), MSc
Novartis Pharmaceuticals UK Ltd, Camberley, Surrey, United Kingdom
Jeanette Kusel, MSci, MSc
National Institute for Health and Care Excellence, London, United Kingdom
Adrian Towse, MA, MPhil
Office of Health Economics, London, United Kingdom
Professor Adrian Towse is director emeritus and senior research fellow of the Office of Health Economics in the UK. Adrian’s current research includes incentives for new drugs and vaccines to tackle Antimicrobial Resistance, the use of 'risk-sharing' arrangements between healthcare payers and pharmaceutical companies, including value-based pricing approaches; the economics of pharmacogenetics for healthcare payers and the pharmaceutical industry; economic issues that affect both R&D for and access to treatments for diseases prevalent in the developing world; the economics of medical negligence; and measuring productivity in healthcare.
A visiting professor at the London School of Economics and a senior researcher at the Nuffield Department of Population Health at the University of Oxford, Adrian also has been a visiting professor at the University of York. For ten years, he served as the non-executive director of the Oxford Radcliffe Hospitals NHS Trust, one of the UK’s largest hospitals. Adrian was president of ISPOR, for the 2014-15 term.
Adrian joined the OHE in 1993 and served as director for 25 years. He holds an MA (Hons) in Politics, Philosophy and Economics from Keble College, Oxford; an MPhil in Management Studies from Nuffield College, Oxford, and the Oxford Centre for Management Studies; and is a member of the Chartered Institute of Management Accountants.
SHOULD GENE THERAPIES BE EXEMPT FROM HTA SCRUTINY?
ISSUE
: Gene therapies are the new kids on the block, with potential for substantial patient/caregiver benefit, disease modification or cure, often in rare severely disabling and life-threatening diseases. However, they have a high upfront price tag meaning demonstrating cost-effectiveness is difficult and highly uncertain to the point that conventional HTA, whilst feasible to perform, is itself cumbersome and arguably of limited value for reliable decision-making. An alternative proposal is to bring key stakeholders together (payers, clinicians, patients, manufacturers) in order to negotiate a fair, affordable and sustainable price, and a payment plan that respects the constraints of the healthcare system. Alternative approaches need urgent discussion before more gene therapies hit the market.
OVERVIEW
: The panel will debate the merits and drawbacks of performing HTA on the relative clinical and cost-effectiveness of innovative gene therapies. The moderator will set up the debate on whether gene therapies represent a special case for exemption/modification from conventional HTA. Maarten Posta will present a case that all gene therapies should undergo rigorous HTA to assess value and quantify key areas of uncertainty for informing meaningful realworld outcomes data collection. Nevertheless, it maybe argued that the challenges and data uncertainties with gene therapies are clear without need for full HTA. Josie Godfrey will present the case for the patient organisation to be a key stakeholder in these negotiations in order to agree an equitable access plan that puts the needs of the patients and their carers at the centre of decision making. The panelists will have 10 minutes each to present their case followed by audience Q&A/discussion. This panel will be of interest to all HTA stakeholders and decision-makers, including healthcare payers.
Moderators
Keith Tolley, BA, MPhil
Tolley Health Economics Ltd., Buxton, DBY, Great Britain
Panelists
Josie Godfrey, BA MA MA
JG Zebra Consulting, London, United Kingdom
Maarten Postma, PhD
University of Groningen, University Medical Center Groningen, Groningen, Netherlands
Darren Walsh
Orchard Therapeutics, Peterborough, United Kingdom
14:15 - 15:30
ISPOR IDEAS LAB
New Insights to Improve Decision Making for Health Globally
ISPOR’s Ideas Lab - an intensive, interactive and free-thinking environment where a diverse group of HEOR stakeholders will discover and discuss new insights to improve decision making for health globally. Participants are encouraged to come together to explore, challenge, and co-create novel and potentially transformative approaches (“aha!” moments) to HEOR. Topics for discussion will include new thinking on “social determinants” and alternative perspective on the “O” and the “E” in HEOR, sustainability index to future proof healthcare, and next generation HTA for complex and personalized combinations of health technologies. The session is designed to fuel original views and fresh perspectives and to trigger and strengthen innovative thinking and the spirit of discovery in the HEOR community.
Moderator: Julia Chamova, MBA, Senior Director, Global Networks, ISPOR, Lawrenceville, NJ, USA
Topic 1 : Ecosystems of health value: A value design framework
Speaker:
Chris Lawer, CEO/Founder, Umio, Oxford, UK and Senior Fellow - UCLA, Center for Healthier Children, Families and Communities, Los Angeles, California, USA
Topic 2: FutureProofing Healthcare: Improving the sustainability of healthcare systems through data
Speakers:
Panos Kanavos, Associate Professor, London School of Economics, London, UK,
Mary Harney, Adviser, Former Tánaiste (Deputy Prime Minister) and Minister for Health of Ireland
Topic 3 HTx --- Next Generation Health Technology Assessment
Speaker:
Wim Goettsch, Associate Professor for HTA, Utrecht University, Utrecht, Netherlands
14:30 - 15:00
ISPOR 2019 EXHIBITOR HEOR THEATER
Linked Data Breakthrough Delivers Major Advancement for Pro and Clinical Research
Presenters:
Tom Haskell , Global Head of Data Analytics, Kantar
Patricia Medina , Medical Intelligence Director, Kantar
To address the needs of patients and their product portfolios, life sciences companies need highly-accurate and timely information from both the physician’s and patient’s perspective. Previously, acquiring this information often required companies to design and conduct large primary research studies that were often expensive and time-consuming. While claims and EHR data analysis by itself is relatively less expensive and quicker than primary research, the results focus on the physician’s perspective and do not take into account outcomes from the patient’s perspective.
To advance research related to linking disparate data sources, Kantar has developed a validated method to link Patient Reported Outcomes (PROs) data with clinical data at the patient level, providing an opportunity to examine the clinical view alongside the patient’s unique perspective, thus delivering significant benefits in both insights and efficiencies.
In this presentation, Kantar will share our approach for linking PRO data with more traditional Real-World Data, including medical and prescription claims, Electronic Health Records (EHRs), and outside labs, and present specific case studies to support the research. Multiple case studies will be presented.
Finally, Kantar will focus on approaches that will allow patient-level linking of PROs and clinical data in Europe to occur. We will focus on the methodology and how to assure that the solution is GDPR compliant.
Sponsored by Kantar
The HEOR Theater presentations are not an official educational offering of ISPOR Europe 2019 and are not sponsored, endorsed or accredited by ISPOR.
15:00 - 16:00
ISPOR BOOTH EVENT - TIPS AND ADVICE FOR NEW HEOR PROFESSIONALS- MEET THE NEW PROFESSIONAL STEERING COMMITTEE CHAIR ELISABETH OEHRLEIN
. . .
15:45 - 16:45
BREAKOUT SESSION 7
DO WE STILL NEED RANDOMIZED CONTROLLED TRIALS?
ISSUE: Both randomized controlled trials and observational studies provide evidence regarding pharmaceutical efficacy or effectiveness and safety. Can high-quality observational studies replace randomized controlled trials to determine relative treatment effects?
OVERVIEW: Traditional randomized controlled trial (RCT) evidence has long been considered the gold standard to determine relative treatment effects and inform clinical decision-making, regulatory approval, and value assessment. However, there is growing interest in using real-world observational evidence to evaluate relative treatment effects, both in a regulatory and a health technology assessment (HTA) context. For example, we see conditional and accelerated regulatory approvals based on single arm trials compared with historical controls based on observational data. Yet, many would argue that the risk of bias, incomplete data, possibility for data dredging or p-hacking, and data silos make it difficult or impossible to trust the results of analyses involving observational data. This panel will discuss the tension between the need for credible evidence and the demand for faster access to promising new treatment alternatives, and the current and potential future role of observational evidence. Lucinda Orsini will provide an overview of efforts and initiatives where RCTs have been compared with observational evidence. Bill Crown will argue that high quality real-world evidence is a robust alternative to RCTs and can be used to understand causal inferences of treatment effects in a more efficient and timely manner. Leanne Larson will outline the spectrum of pragmatic approaches to study design and why each is important. Jeroen Jansen will illustrate how randomized and observational study findings can be formally integrated to make optimal use of the available evidence base.
Moderators
Lucinda Orsini, DPM, MPH
ISPOR, Lawrenceville, NJ, USA
Lucinda Orsini is currently Associate Chief Science Officer at ISPOR, the leading society for health economics and outcome research. The science office at ISPOR develops, leads, and supports strategic initiatives related to research, scientific, and content priorities. While Lucinda holds a professional medical degree, she has spent most of her ~20-year career in health economics and outcomes research. Starting at The Medstat Group (Truven Health Analytics) Lucinda managed projects using health care claims data for external clients. She has worked in the pharmaceutical industry at Bristol-Myers Squibb and subsidiaries in Global HEOR focused on oncology and immunotherapy leading and publishing on patient reported outcomes, health economic modeling and real-world evidence studies – both prospective and retrospective in many different tumor types. Lucinda has recently led HEOR efforts at PAREXEL a full-service contract research group prior to coming to ISPOR.
Panelists
William Crown, PhD
Optum Labs, Cambridge, MA, USA
Jeroen P Jansen, PhD
Precision Xtract, Oakland, CA, USA
Jeroen P. Jansen is the chief scientist – Evidence Synthesis and Decision Modeling at Precision Xtract and adjunct professor of Health Research & Policy (Epidemiology) at Stanford University. In addition, he is lead scientific advisor - Open Source Value Project (OSVP) at the Innovation and Value Initiative (IVI) where he oversees the development of open-source cost-effectiveness models. He has 15 years of research experience in the areas of evidence synthesis and economic modeling. He was an associate editor of the Research Synthesis Methods Journal; and is co-author of a textbook on network meta-analysis for decision-making. Dr. Jansen has published extensively on both applied and methods research in his areas of expertise. Notable contributions are the development of guidance for consumers and producers of network meta-analysis studies (ISPOR, PRISMA), as well as the development of methods for network meta-analysis of time-to-event data, repeated measures, and integration of patient level data with study level data. Previously, he was a founder of Redwood Outcomes, a health economics and outcomes research firm that was acquired by the Precision Medicine Group.
Leanne Larson, MHA
PAREXEL International, Waltham, MA, USA
IS THERE A SYSTEMATIC METHODOLOGY FOR CHOOSING TRIAL ENDPOINTS THAT INCORPORATES PATIENTS’ PERSPECTIVES? WHAT MIGHT IT LOOK LIKE?
PURPOSE
: To discuss strategies by which trial endpoints might be selected and, ultimately, translated into meaningful and patient-focused treatment claims.
DESCRIPTION
: Trial endpoints
should reflect treatment benefits and harms to patients based on how they feel, function, and/or survive. Selecting endpoints raises challenges in that they summarize concepts, how those concepts are measured (e.g., tools used, and how tools are administered), and data analytic strategies (e.g., group vs. within person comparisons, time to event vs fixed time point). There is a lack of clarity about the methods by which endpoints are developed and defined, and we make the case that several stages in the process are not incorporating patient perspectives. To frame the workshop, Dr. Shields (10 minutes) will articulate a model emphasizing how patient and other stakeholder input could be used to guide endpoint selection decisions. This will be followed by gauging audience experience in selecting trial endpoints. Next, Dr. Powers (15 minutes) will engage the audience to reflect these participant experiences, compare them to his own experiences defining endpoints as an FDA reviewer, physician, and outcomes scientist, and provide a summary of “things to think about” for endpoints. He will highlight particular facets of how and where patient perspectives could have informed endpoint development. Next, Dr. Stone (15 minutes) will present several new pain indices derived from momentary, real-time research; how stakeholders evaluated these indices; how the indices performed in trials; and how the indices relate to performance outcomes. These findings highlight quandaries in the selection of trial endpoints, especially how patient perspectives may not align with other data. The final 15 minutes of the workshop will be spent eliciting and integrating audience perspectives and suggestions to reflect how principles, guidelines, and data from multiple sources may inform clinical trial endpoints in a more systematic and transparent process.
Discussion Leader
John H Powers, MD
George Washington University School of Medicine, Washington, DC, USA
Alan Shields, PhD
Adelphi Values, Boston, MA, USA
Arthur A. Stone, PhD
University of Southern California, Los Angeles, CA, USA
APPLICATION OF EXISTING AND NOVEL NETWORK META-ANALYSIS METHODOLOGIES- APPROPRIATENESS FOR HTA PURPOSES (Advanced Workshop)
PURPOSE: Network meta-analyses (NMA) and survival extrapolations are needed for HTA purposes. Often NMAs are performed based on hazard ratios (HRs). In case the proportional hazard assumption is violated, an NMA on HRs is no longer appropriate. For recently introducted immune-oncology therapies in indications such as non-small cell lung cancer or melanoma the proportional hazards assumption is usually violated. The objective of this workshop is to present advanced novel and existing NMA approaches for survival endpoints and discuss these in light of their appropriateness for HTA purposes.
DESCRIPTION: First, a brief introduction to NMAs relying on HRs is provided by discussing the proportional hazard assumption and the implications of implementing the results in health economic models. The implications will be illustrated by presenting two evidence networks: 1) a two-trial network covering three therapies (Nivolumab/Dacarbazine/Ipilimumab plus Dacarbacine) in advanced previously untreated melanoma, and 2) a three-trial network covering four therapies (Nivolumab/Pembrolizumab 2mg/Pembrolizumab 10 mg/Docetaxel) in second-line non-small cell lung cancer.
Second, considering violation of the proportional hazard assumption, the parametric NMA will be applied to both networks, which uses parametric distributions for estimating the relative treatment effect over time. Third, other advanced methods allowing for fluctuating hazards, proportional hazard assumption violations and/or cure will be presented. These include NMAs based on splines, fractional polynomials and mixture-cure models. Additionally, it will be demonstrated how external survival data with longer follow-up time can be used to reduce uncertainty of NMA predicted survival data relying on immature survival data by using prior information. Finally, an overview of the application and acceptability of different existing methodologies in HTA submissions will be presented. Guidance will be provided on choosing the most appropriate NMA approach for HTA purposes. Advantages and disadvantages of each approach will be discussed.
Discussion Leader
Bart Heeg, PhD
Ingress-Health, Rotterdam, ZH, Netherlands
Dr. Mario Ouwens, PhD
AstraZeneca, Gothenburg, Sweden
Maarten Postma, PhD
University of Groningen, University Medical Center Groningen, Groningen, Netherlands
Sophie van Beekhuizen, MSc MPhil
Ingress-Health, Rotterdam, Netherlands
SPOTLIGHT SESSION 3
THE EVOLVING DEMANDS FOR MEDICAL DEVICE EVIDENCE DEVELOPMENT- WHAT THE FUTURE HOLDS
This expert panel will review the latest issues in medical device evidence requirements, with discussion of several case study examples. Key issues to be addressed include the evolution of EUnetHTA joint assessments and how they are applied by EU member countries, the impact of new CE mark-related evidence requirements, and how medical device firms should work to bridge the divide between country level requirements for Health Technology Assessment and regulatory study requirements. Panel will include HTA experts from national bodies, along with industry experts.
Moderators
Meike Bomhof
Global Headquarters, Rockland, MA, USA
Panelists
Rossana Alessandrello, MSc
Agency for Health Quality and Assessment of Catalonia (AQuAS), Barcelona, Spain
Rossana Alessandrello is currently the Value Based Public Procurement Coordinator at AQuAS, the Agency for Health Quality and Assessment of Catalonia. She also serves as Member Board Of Directors of the VPH Institute, as External Expert of the European Union and as invited lecturer at several trainings, courses and symposia about digital health transformation projects, pre-commercial procurement, public procurement of innovation and value base procurement. Rossana earned her MSc in Electronic Engineering, with specialization in bioengineering, at the Politecnico di Milano (Italy) and her Certificate in Business Administration at the Manchester Business School (UK). She has developed her career working for international SMEs, R&D centres, public administration and as external consultant enabling her to gain experience and knowledge on digital innovation and transformation (from needs identification to procurement design, providers selection and management; from innovative services design to their development and deployment). She coordinated DECIPHER, the very first cross-border pre-commercial procurement in mHealth, she was AQuAS IP of INSPIRE, a coordinated support action aimed to evangelize public procurers, industry players, policy makers and investors on pre-commercial procurement/ public procurement of innovation and is now actively contributing to ANTI-SUPERBUGS pre-commercial procurement, RITMOCORE public procurement of innovation and PiPPi, a community of practice around public procurement of innovation and led by the European University Hospital Alliance (DECIPHER, INSPIRE, ANTI-SUPERBUGS, RITMOCORE and PiPPi all received or are receiving EC funding). Her main interest is improving the current way of doing, bringing value to the healthcare systems and to the health outcomes, through the adoption of the public procurement of innovation aimed at satisfying felt unmet needs.
Lindsay Bockstedt, PhD
Medtronic, Minneapolis, MN, USA
Rachele Busca, PharmD, MSc, MBA
W.L GORE, Verona, Italy
Graduated in Chemistry & Pharmaceutical Technology at Bologna University and completed education in Health Economics & Outcomes Research with a Master at the University of Milan and an MBA at the Bologna Business School.
Market Access & Health Economics & Outcomes Research expert / Director level professional with over 10 years of experience in Medical Device, including Health Economics & Outcomes Research, Reimbursement & Access and Value strategies, delivering data and tools to effect reimbursement, funding and Health Technology Assessments at International level.
Currently EMEA Leader Health Economics at W.L GORE – a Global Devices manufacturer known for innovation and a modern, distinctive company culture.
Previously served for over 7 years the Italian Pharmaceutical business in Bayer, Recordati and consultancy group Pbe. Most recently as Director Pricing and Reimbursement Strategy at BTG and Principal Manager at Medtronic assisting several divisions: interventional pulmonology, cardiology, cardiac disease management, structural heart, peripheral products.
Specialties: market access strategies, pricing, HEOR, incorporation of market access requirements into clinical, medical & commercial strategies, new market assessment, due diligence.
Hedi Schelleman, PhD
The National Healthcare Institute (ZIN), Diemen, Netherlands
Hedi Schelleman is a scientific coordinator of the Research Programme Potentially Promising Care, a position she held since January 2019. She has a PhD in epidemiology and started her career as postdoctoral researcher at the Center for Clinical Epidemiology and Biostatics at the University of Pennsylvania. In 2010 Hedi joined The National Healthcare Institute. There she worked for seven years as an HTA assessor of drugs and medical devices. In the last 4 years she was the scientific coordinator of Research Programs involving medical Devices, medical Aids and pharmaceuticals. In this role she follows the developments of the new MDR-regulation. She is also a member of the European Network of Health Technology Assessments (EUnetHTA).
BREAKOUT SESSION 7
CARDIOVASCULAR & DIABETES DISORDER STUDIES
CV1: DIABETES TREATMENT IN DENMARK- HOSPITAL- VS. GP-MONITORING
3:45PM - 4:00PM
Pulleyblank R , Laudicella M, Olsen KR University of Southern Denmark, Odense, 83, Denmark
OBJECTIVES : Government policy in Denmark has been to push monitoring of diabetes patients away from specialized hospital clinics into general practice settings. This study investigates composition of hospital- and GP-monitored diabetes patients, and treatment cost-efficiency implications of moving monitoring into primary care. METHODS : Using administrative databases of hospitalizations, primary care services, and prescriptions, we algorithmically identified diabetes patients, and whether they were hospital-monitored. Socio-demographics, comorbidities, and annual treatment cost were compared across monitoring settings. Propensity score matching (PSM) was used to estimate impacts on annual healthcare costs of being monitored in hospital (Average Treatment Effect on the Treated - ATET). RESULTS : In 2015, 288578 diabetes patients were identified, and 41976 were identified as being regularly monitored in hospital. Hospital-monitored patients: had significantly higher Charlson Comorbidity Index scores (3.4 vs. 1.9); were younger (58 vs 63), but with longer histories of diabetes (14 vs. 8); more likely to be male (58% vs. 51%); had higher levels of education; and were less likely to be immigrants. Hospital-based patients had higher annual costs for medications, hospital inpatient and outpatient admissions, and non-hospital-based specialists; only general practice costs were lower. Estimated ATET on total costs for hospital-monitored patients was 7873DKK; positively driven by outpatient cost (9120DKK) and prescriptions (1266DKK); and negatively driven by ATET estimates for general practice (-520DKK) and inpatient costs (-2047DKK). Estimated ATET for costs of hospitalizations originating as emergency visits were negative (-1103DKK). No significant impact on costs for non-hospital-based specialist services were identified. CONCLUSIONS : This study finds evidence that average hospital-monitored diabetes patients in Denmark are in poorer health than GP-monitored patients, and the quality of healthcare delivered to hospital-monitored patients is higher. However, PSM results suggest that there are possible net cost savings to be realized by moving care of some hospital-monitored patients to primary care.
CV4: HOSPITALIZED WITH STROKE AT THE WEEKEND- ARE THERE HIGHER RISK OF EARLY DEATH AND HIGHER COSTS?
4:30PM - 4:45PM
Ghiani M 1 , Wilke T1 , Maywald U2 1 IPAM, University of Wismar, Wismar, Germany, 2 AOK PLUS, Dresden, Germany
OBJECTIVES Previous studies documented poorer health outcomes and mortality associated with weekend hospitalizations (“weekend effect”). However, few studies adjusted for disease severity and little is known on costs. This work investigates the weekend effect and its costs for patients with cerebral infarction (CI) in Germany, adjusting for patient characteristics and proxies of stroke severity. METHODS Adult patients with a first-time CI hospitalization (ICD-10: I63) between 01/01/2014 and 30/06/2017 were included from German health claims. Propensity score matching (PSM) was used to match patients hospitalized on weekends or on public holidays (weekend group) with patients hospitalized during the working week (workday group), based on baseline characteristics and proxies for disease severity such as concomitant diagnoses of aphasia, ataxia, and coma, or peg tube at index hospitalization. Matched cohorts were compared in terms of in-hospital mortality, 7-day mortality, and risk and costs of stroke and rehabilitation stays in the year after first stroke. RESULTS Of 32,311 patients hospitalized for a CI between 01/01/2014 and 30/06/2017, 8,409 were in the weekend group, and 23,902 in the workday group. After PSM, 16,730 patients were included (8,365 per group). Matched cohorts did not differ in baseline characteristics or stroke severity. In the weekend group, the risk of in-hospital death (11.2%) and the 7-day mortality rate (6.8%) were 13.1% and 17.2% higher than in the workday group, respectively (both p<0.01). The risks of subsequent stroke hospitalization and rehabilitation stays for a stroke were 8.4% higher and 5.5% higher in the weekend group (both p=0.02). As a result, the stroke-related hospitalization and rehabilitation costs per patient year were, respectively, 5.6% and 8.0% higher in the weekend group (both p=0.01). CONCLUSIONS A significant weekend effect emerged after controlling for a wealth of patient characteristics and proxies of stroke severity, resulting in higher costs for patients admitted on weekends.
CV3: MECHANICALLY EXPANDED VERSUS SELF-EXPANDING TRANSCATHETER AORTIC VALVE REPLACEMENT IN HIGH-RISK PATIENTS WITH AORTIC STENOSIS- A BUDGET IMPACT ANALYSIS
4:15PM - 4:30PM
Neeser K 1 , Rizik DG2 , Amorosi SL3 , Dlotko E1 , Agrawal M1 , Rojanasarot S3 , Feng C4 , Ihlberg L3 , Reardon MJ5 1 Certara, Loerrach, BW, Germany, 2 HonorHealth Heart Group, Scottsdale, AZ, USA, 3 Boston Scientific, Marlborough, MA, USA, 4 Boston Scientific, Maple Grove, MN, USA, 5 Houston Methodist DeBakey Heart & Vascular Center, Houston, TX, USA
OBJECTIVES: Each transcatheter aortic valve replacement (TAVR) technology is associated with device-specific benefits and risks. This study evaluated Medicare costs of TAVR with a mechanically expanded valve (MEV) versus a self-expanding valve (SEV) in high-risk patients with severe aortic stenosis. METHODS: A 1-year budget impact model was developed from a Medicare perspective. MEV and SEV clinical event rates were obtained from the REPRISE III randomized controlled trial at 12 months of follow-up. The model included all relevant procedural complications, including non-disabling and disabling stroke, repeat TAVR procedure, pacemaker implant, surgical aortic valve replacement, hospitalizations for worsening heart failure (HF), major vascular complications, life-threatening bleeds and acute kidney injury. Patient-level costs were assessed for the index TAVR admission (days 0-7) and post-TAVR short- (days 8-30) and long-term (days 31-365) complications. Acute care costs were obtained from 2019 Medicare MS-DRG rates. Long-term care costs for disabling stroke and worsening HF were derived from REPRISE III and published Medicare analyses. One-way sensitivity analysis (OWSA) was performed. RESULTS: At 1 year, disabling stroke, repeat procedure and HF hospitalization rates were lower for MEV compared to SEV; pacemaker rates were higher for MEV. Other complication rates in the model were similar. Total Medicare costs for MEV were lower than SEV ($45,030 versus $47,006). This difference was attributable to the lower costs of disabling stroke (-$1,370), repeat procedure (-$574) and worsening HF (-$314). Pacemaker costs were higher for MEV ($774) than SEV ($477). OWSA showed TAVR procedure costs and the occurrence rates of disabling stroke, repeat procedures and hospitalizations for worsening HF had the greatest influence on model results. CONCLUSIONS: MEV is a less costly alternative to SEV at 1 year from a Medicare perspective. Continued technological innovation in TAVR aimed at reducing complications may result in greater savings for payers.
CV2: NATIONAL SAVINGS FROM TELEHEALTH CARE IN HEART FAILURE RESULTS FROM THE DANISH TELECARE NORTH TRIAL
4:00PM - 4:15PM
Ehlers L , Hansen L, Jensen MB, Sørensen SS, Sørensen AS Aalborg University, Aalborg University, Denmark
OBJECTIVES: To estimate the national budget impact (BI) on a Danish health plan of introducing telehealth care in heart failure patients. METHODS: BI analysis was conducted from a national (public) payer perspective. Data was taken from the TeleCare North randomized-controlled trial and literature. Patients in the intervention group were provided with a Telekit consisting of a tablet, a digital blood pressure monitor, and a scale, and were instructed to perform measurements 1-2 times a week. Micro-costing and patient-specific register data from 274 patients were analyzed. RESULTS: For an expected incidence of 9.000 newly diagnosed heart failure patients in Denmark, the 2018 direct start-up investments in equipment, education of patients and health professionals, software developments, and other investments were £5.9 million to be annuitized over 3-5 years. The between-group adjusted difference in mean annual costs per patient was -£5096 [95%CI: -£8736;-£1456]. The one-year adjusted QALY difference was 0.0034 [95% CI: -0.0711; 0.0780] thus, providing a positive incremental NMB of £5164. Total annual healthcare savings in Denmark was £45.9 million, of which approximately 67% could be referred to the reduced costs of hospitalizations. On other health care services, there were savings on outpatient hospital services (17%), community-based health care (10%), and other (5%). Sensitivity analyses revealed limited explanation of what components of the intervention were effective or whether the effect is contingent on the intervention in its entirety. CONCLUSIONS: Total cost savings of introducing telehealth care to heart failure patients in a national health-care plan accrued from the lower hospitalizations that outweigh investments and increased costs of telecommunication and home monitoring. Because of the lack of detailed understanding of the mechanisms that lead to cost savings, the national rollout of the telehealth service should be accompanied by research and careful monitoring of the implementation in each of the five Danish regions.
15:45 - 19:00
RESEARCH POSTER SESSION 4
16:00 - 16:30
ISPOR 2019 EXHIBITOR HEOR THEATER
Communicating Value to Decision-Makers: What’s Your Prognosis?
Presenters:
Walter Bouwmeester , PhD, Center of Excellence lead, Associate Director, Pharmerit
Elisabeth Fenwick , PhD, Senior Director, Pharmerit
When considering whether to prescribe a new agent, clinicians face uncertainty in determining which patients would benefit most from treatment. Policymakers face the same challenge when making funding allocation decisions. As such, there is a requirement for information that can help to identify the appropriate target population for treatment. In this presentation, we will look at how to incorporate commonly used risk factors and prognostic scores into decision-making tools that can enable payers and clinicians to predict treatment success and determine the value of novel agents. The approach will be illustrated using a case study.
Sponsored by Pharmerit International
The HEOR Theater presentations are not an official educational offering of ISPOR Europe 2019 and are not sponsored, endorsed or accredited by ISPOR.
16:00 - 17:00
ISPOR BOOTH EVENT - ARE YOU A NEW ISPOR MEMBER? LEARN HOW TO GET INVOLVED.
. . .
17:00 - 18:00
ISPOR BOOTH EVENT - EXPLORE WAYS TO ENGAGE WITH THE ISPOR STUDENT NETWORK- MEET THE STUDENT NETWORK CHAIR AAKASH GANDHI
. . .
BREAKOUT SESSION 8
COST-EFFECTIVENESS WITH BINDING BUDGETS- BAD MEDICINE, BAD ECONOMICS, NEITHER OR BOTH? (Advanced Workshop)
PURPOSE:
To help choose among ways to establish proper Willingness-to-pay (WTP) cost-effectiveness cutoffs (K) and to assess choices when technologies funded using that cutoff exceed available health system budgets.
DESCRIPTION:
SECTION 1: Two primary approaches exist to choose maximum WTP cutoffs (K). Welfare economics-based approaches say to use population-based preferences. We will compare various approaches to such methods—league tables, wage premiums for risky occupations, and method based on economic utility theory. Each approach uses different observed economic parameters to estimate optimal cutoffs. Alternative approaches use health system budgets to infer implicit optimal cutoffs, stating that these "supply side" cutoffs are the relevant measure by which health technologies should be measured. This “opportunity cost” school argues that applying larger cutoffs than implied by budgets will consume resources inefficiently, thus harming population health. We will summarize methods used to estimate this cutoff. We will also discuss an apparent “regularity”—that single-payer systems commonly adopt budgets with a low cutoffs vis-a-vis demand-side models, while competitive systems (e.g. USA) have few restrictions on technology, potentially stressing operating budgets and/or increasing premiums or taxes. SECTION 2: We will use economic concepts of short-run and long run equilibria (in relation to what is variable) to illuminate these issues and help determine both both short and long-run operational methods for evaluating health technologies using cost-effectiveness frameworks. This discussion will separate two relevant questions – how to choose the optimal cutoff and what to do when choices implied by the cutoff are unaffordable. We will assess whether (and if so, how) WTP methods can inform single-payer system choices, and discuss other key decisions affecting optimal cutoffs, including whether or not out-of-system “supplementation” is allowed and whether K is determined by popular vote (median-voter model), constrained welfare-maximizing models of some sort, or other alternatives.
Discussion Leader
Charles E. Phelps, PhD, MBA
University of Rochester, Gualala, CA, USA
Charles E Phelps, PhD, University Professor and Provost Emeritus of the University of Rochester (Rochester, NY USA), health economist, has published major theoretical and applied research in health economics and particularly in cost-effectiveness analysis since beginning his career at the RAND Corporation, including a founding role in the RAND Health Insurance Experiment. During his time on the active faculty of the University of Rochester (1984 – 2010), he served for five years as chair of the (currently named) Department of Public Health Sciences, and then (for 13 years) as Provost of the University of Rochester. His more recent work has focused on expanding cost-effectiveness analysis using multi-criteria decision analysis (MCDA) models, voting theory (to improve use of MCDA models in group settings), and systems approaches to evaluating health care. His textbook, Health Economics, is in its sixth edition (2017) and (with Stephen T. Parente) published in 2017 a new book entitled “The Economics of US Health Care Policy.” He was elected to the Institute of Medicine (now the National Academy of Medicine) in 1991. Professor Phelps served as a member of the ISPOR Task Force on Value in Health in 2016-2017.
Adrian Towse, MA, MPhil
Office of Health Economics, London, United Kingdom
Professor Adrian Towse is director emeritus and senior research fellow of the Office of Health Economics in the UK. Adrian’s current research includes incentives for new drugs and vaccines to tackle Antimicrobial Resistance, the use of 'risk-sharing' arrangements between healthcare payers and pharmaceutical companies, including value-based pricing approaches; the economics of pharmacogenetics for healthcare payers and the pharmaceutical industry; economic issues that affect both R&D for and access to treatments for diseases prevalent in the developing world; the economics of medical negligence; and measuring productivity in healthcare.
A visiting professor at the London School of Economics and a senior researcher at the Nuffield Department of Population Health at the University of Oxford, Adrian also has been a visiting professor at the University of York. For ten years, he served as the non-executive director of the Oxford Radcliffe Hospitals NHS Trust, one of the UK’s largest hospitals. Adrian was president of ISPOR, for the 2014-15 term.
Adrian joined the OHE in 1993 and served as director for 25 years. He holds an MA (Hons) in Politics, Philosophy and Economics from Keble College, Oxford; an MPhil in Management Studies from Nuffield College, Oxford, and the Oxford Centre for Management Studies; and is a member of the Chartered Institute of Management Accountants.
A NOVEL APPROACH TO IMPROVING OUR UNDERSTANDING OF THE BURDEN OF A RARE DISEASE- THE USE OF OPPORTUNISTIC RETROSPECTIVE QUALITATIVE ANALYSIS OF PUBLIC RESPONSES TO HIGHLY SPECIALISED TECHNOLOGY APPRAISAL
PURPOSE: To describe how researchers may improve their understanding of rare diseases, such as XLH, through the use of novel approaches. We will describe the National Institute for Health and Care Excellence (NICE) Highly Specialised Technology (HST) appraisal process and how the patient voice may bring value to this process. We will describe how a thematic analysis of patient submissions made during the HST process have increased understanding of the burden of disease.
DESCRIPTION: The proposed workshop will provide an overview of HST, XLH and its burden, and the process of thematic analysis. We will describe how in combination with NICE, patient groups and physicians we enabled the patient experience of XLH to be described to a wider audience.
NICE HST Process (10 minutes, NICE):
An overview of the HST process, including a description of timelines, how patients and physicians input into the process, and how learning about the burden of disease direct from the patient can inform the discussion XLH (10 minutes, Kyowa Kirin International):
Extracts of video footage on XLH disease burden from a patient Summary of available data, or lack of data, on burden of disease in XLH Experience of the value of using novel approaches including retrospective data sources in qualitative analysis Thematic Analysis (15 minutes, MAP BioPharma):
We will provide a working example on the use of thematic analysis in eliciting patient preference in XLH Compare findings to published literature and physician testimony, to understand the added value of the analysis Discussion (25 minutes)
The use of novel innovative approaches to generating information using opportunistic data: utilising all available evidence in rare diseases Giving patients a bigger platform: respecting their efforts in contributing to the process and maximising the usefulness of their submissions, to increase understanding of XLH Collaboration with patients, physicians and the NICE was key
Discussion Leader
Paul Connor, BSc
Kyowa Kirin, Galashiels, United Kingdom
Jade D Marshall, MEng, MSc
MAP BioPharma Limited, Cambridge, United Kingdom
Sheela Upadhyaya, MSc
Kyowa Kirin, London, United Kingdom
VALUE ASSESSMENT OF MEDICAL TECHNOLOGY
MT2: UNDERSTANDING POTENTIAL CAPACITY CONSTRAINTS RESTRICTING TIME-TO-DIAGNOSIS IN A MULTI-DISCIPLINARY DIAGNOSTIC CENTRE (MDC) FOR PEOPLE WITH NON-SPECIFIC SYMPTOMS OF CANCER- A DISCRETE EVENT SIMULATION
5:15PM - 5:30PM
Jones C 1 , Youn JH2 , Payne K1 1 The University of Manchester, Manchester, UK, 2 University of Manchester, Manchester, UK
OBJECTIVES : Multi-disciplinary diagnostic centres (MDC) perform a series of diagnostic tests with the aim of providing patients with a timely and accurate diagnosis of whether they have cancer within one day. This study aimed to estimate the: 1) resource use and associated cost of the time taken to reach a diagnosis of cancer in a MDC compared with the existing two-week-wait (2WW) referral pathway in England; 2) potential for capacity constraints in the system affecting the flow of patients through the MDC pathway. METHODS : A discrete event simulation, assuming the healthcare system perspective, was developed to represent the referral pathways to reach a cancer diagnosis for the MDC and 2WW. The time horizon was from initial GP referral to definitive diagnosis. Expert opinion (radiologist, general practitioner, cancer programme manager) informed staffing levels and the mean time taken for each consultation and diagnostic test. Unit cost data (£; 2017) was identified using published literature. Three scenarios were developed to assess capacity constraints: 1) one extra computerised tomography (CT) scanner; 2) one extra CT scanner and required imaging staff; and 3) twenty extra patients per clinic. RESULTS : Estimated mean time-to-diagnosis (and associated cost) was 0.94 (0.94 to 0.95) days (£475) and 10.57 (10.54 to 10.62) days (£696) for MDC and 2WW, respectively. The number of CT scanners available and imaging staff were confirmed to be capacity constraints. When compared with the base-case scenario: scenario 1 increased overall waiting times by 2%; scenario 2 decreased waiting times by 49%; scenario 3 decreased waiting times by 31%. CONCLUSIONS : Indicative results suggest the MDC is able to diagnose patients faster and at lower cost compared with 2WW. Implementation of MDCs must take into account the number of CT scanners and imaging staff available to facilitate an accurate diagnosis in one day.
MT4: COST ANALYSIS OF INTEGRATED VERSUS CONVENTIONAL OPERATING ROOMS
5:45PM - 6:00PM
Risør B , Carstensen K, Kejser Jensen E, Loevschall C, Ladehoff Thomsen AM, Tayyari Dehbarez N DEFACTUM, Central Denmark Region, Aarhus N, 82, Denmark
OBJECTIVES
Integrated operating rooms allow for surgical staff to control all relevant equipment from the sterile field as different systems/technologies are functionally linked through a single touchpad. Thereby it potentially reduces inappropriate workflow and saves time. This study compared resource use and costs of the integrated operating room (IOR) and conventional operating room (COR).
METHODS
Surgical departments with an IOR and COR that performed comparable surgeries were identified to be included in the study. A hospital perspective was applied to compare resource use and costs of the two operating rooms (OR). Field studies and unstructured interviews were conducted to identify activities and resources that varied in the OR set-up. Time consumption during identified procedures was extracted from the clinical system at the hospital. Independent t-test was used to estimate the statistical difference in mean time between the two alternatives. Technology costs were estimated based on procurement data for the past 3 years at the hospital. Costs common to both IOR and the COR were not included.
RESULTS
The department of heart, lung and vascular surgery and the department of gastrointestinal surgery at Aarhus University Hospital in Central Denmark Region were included. During the study period a total of 1,192 surgeries of selected types were performed in the ORs. We found no statistically significant differences in procedure time or staff cost between IOR and COR. The cost analysis revealed incremental costs of an IOR varying from investment costs of 93,290 EUR corresponding to an equivalent annual cost of 11,909 EUR for the simplest solution to investment costs of 151,007 EUR corresponding to an equivalent annual cost of 19,483 EUR for a more comprehensive solution.
CONCLUSIONS
The study found no difference in procedure time or staff costs between the IOR and the COR. The IOR was associated with varying incremental costs dependent of the integrated solution.
MT3: OUTCOMES AND HOSPITAL UTILIZATION OF SLEEP APNEA PATIENTS TREATED WITH POSITIVE AIRWAY PRESSURE DURING AND AFTER ONSET OF HEART FAILURE
5:30PM - 5:45PM
Huang F 1 , Goldman M1 , Lotz G2 1 Philips, Cambridge, MA, USA, 2 Philips, Monroeville, PA, USA
OBJECTIVES As a few recent studies failed to show beneficial effect of Positive Airway Pressure (PAP) on the sleep apnea patients with cardiovascular comorbidities, we aimed to use large administrative database to investigate the association of PAP usage and the health economics outcomes of the sleep apnea patients with a new onset of heart failure (HF). METHODS Adult sleep apnea patients with a new onset of HF were selected in the 5% Medicare data during June 2013-December 2014. The remaining patients were further divided by whether they had used PAP within 6 months prior to the HF onset. One-year clinical outcomes and medical utilization were compared between the groups. Propensity-score matching by patient/hospital characteristics and comorbidities were performed to adjust baseline differences. T-test or Chi-square test was used to determine statistical significance and COX Survival analysis was used to determine the hazard ratio (HR) of the mortality. RESULTS Our study identified 15,511 sleep apnea patients with a new onset of heart failure, of whom, a total of 4,376 (28.2%) were identified as PAP users. After propensity-score matching, the patients who had used PAP prior to the heart failure had less hospital charge (56,250 USD vs. 60,515 USD, p=0.02), shorter hospital length of stay (5.45 vs. 5.89 days, p=0.0005), shorter ICU length of stay (4.64 vs. 5.07 days, p=0.03) during the index visit. Further, PAP users had significantly decreased 30-day readmission (18.5% vs. 20.4%, p=0.04) and lower mortality with the hazard ratio of 0.85 compared to non-PAP users (p<0.0001) during the one-year follow-up. CONCLUSIONS We have found a significant association of the PAP usage with higher one-year survival and lower hospital charge among sleep apnea patients with a new onset of heart failure (HF). Therefore, effective PAP treatment for sleep apnea among high-risk heart failure patients is strongly recommended.
MT1: LONG BONE FRACTURES- OVERVIEW OF SURGICAL APPROACHES BY FRACTURE TYPES AND PATIENT PROFILE
5:00PM - 5:15PM
Chitnis AS 1 , Ruppenkamp J2 , Vanderkarr M3 , Sparks C4 , Grebenyuk Y4 , Holy CE5 1 Johnson & Johnson, New Brunswick, NJ, USA, 2 Johnson & Johnson, Garner, NC, USA, 3 DePuy Synthes, Inc., Bay Village, OH, USA, 4 DePuy Synthes, West Chester, PA, USA, 5 Johnson & Johnson, Somerville, MA, USA
OBJECTIVES : Surgical treatment for long-bone fractures may require medical devices such as pins/plates, external fixators or intramedullary nails (IMN). The utilization of each type of device by fracture anatomy and patient presentation is characterized in this study. METHODS : A retrospective database analysis was conducted in the IBM® MarketScan® Commercial Database. Patients with femoral, tibial or humeral fractures coded during an inpatient admission between 2008-2017 were identified. Patient demographics, comorbidities at time of index repair procedure (31 Elixhauser comorbidity indices), fracture type (open vs closed), precise fracture anatomy and repair procedure (IMN vs plating vs external fixation vs no device) were queried. The prevalence and patient/fracture characteristics associated with each repair procedure were analyzed. RESULTS : 165,554 patients were identified with 37,653 humerus, 58,790 tibial and 69,111 femoral fractures, of which 67%, 86% and 69% required surgical repair procedures, respectively. Within the surgical cohort, IMN was used for 46% femoral, 30% tibial and 5% humeral fracture repairs. For patients with femoral fractures, there was a significant association in use of IMN with shaft fractures (55.3% of IMN cohort vs 24.0% of the non-IMN cohort), trochanteric fractures (48.7% IMN vs 21.7% non-IMN) and displaced fractures (15.2% IMN vs 7.4% non-IMN). For patients with tibial fractures, there was also a significant association in use of IMN with open (31.5% IMN vs 17.5% non-IMN) and shaft fractures (94.5% IMN vs 32.7% non-IMN). For humeral fractures, shaft and displaced fractures were mostly associated with use of IMN. Use of IMN increased for all anatomies in the 10-years considered, from 6.0-7.3%, 28.3%-29.3% and 44.5%-56.4% for humeral, tibial and femur fractures requiring surgery. CONCLUSIONS : A significant proportion of long bone fractures require surgical repair. In the past 10 years, utilization of IMN for these fractures increased consistently and now represent the predominant device used for specific fractures such as shaft fractures.
NEUROLOGICAL DISORDERS STUDIES
ND3: SHORT- AND LONG-TERM DISABILITY BURDEN AMONG EMPLOYED PATIENTS RECEIVING DISEASE-MODIFYING TREATMENT FOR MULTIPLE SCLEROSIS
5:30PM - 5:45PM
Bonafede M 1 , Mehta R2 , Kim G3 , Sruti I3 , Tian M2 , Pelletier C2 , Goldfarb NI4 1 IBM Watson Health, Brentwood, NH, USA, 2 Celgene Corporation, Summit, NJ, USA, 3 IBM Watson Health, Cambridge, MA, USA, 4 Greater Philadelphia Business Coalition on Health, Philadelphia, PA, USA
OBJECTIVES : This study examined the short-term disability (STD) and long-term disability (LTD) burden (as measures of severe productivity loss) among employees with multiple sclerosis (MS) initiating disease-modifying treatments (DMTs). METHODS : Using the IBM MarketScan Commercial Claims and Health and Productivity Management Database, patients with ≥2 new MS claims (≥1 and <365 days apart) between January 1, 2009, and January 1, 2017, and 1 subsequent claim for a DMT (index date) were identified. Patients had ≥12 months of continuous enrollment pre-index, ≥1 full calendar year of STD and/or LTD eligibility (accessible to active employees only), and no evidence of pregnancy or malignancy during the study period. The number and duration of all STD and/or LTD claims were captured during all calendar years of follow-up. RESULTS : A total of 3,023 patients initiating orals (25.5%), injectables (64.1%), or infusions (6.1%) were identified (mean age: 41.4 years; female: 62.1%). In the first year, the percentage of patients with STD claims was generally similar across routes of administration (total: 23.2%; orals: 21.7%; injectables: 23.4%; infusions: 27.7%), as were STD days per patient with a claim (68.8, 69.9, 68.8, and 64.6 days, respectively). Percentages of patients with LTD claims were low (total: 3.1%; orals: 3.7%; injectables: 2.9%; infusions: 2.8%). Oral DMT initiators had the lowest number of LTD days per patient (31.7 days), with a numerically lower number of days compared with infusions (45.8 days) and a significantly lower difference compared with injectables (76.3 days; P =0.0307). CONCLUSIONS : Productivity loss burden was high in the first year in MS patients. Oral DMT initiators had the lowest percentage of short-term disability claims and days per long-term disability claim, suggesting productivity benefits in severe productivity loss with early access to orals. Additional research is needed to better quantify this benefit.
ND4: WHO BENEFITS MOST FROM COLLABORATIVE DEMENTIA CARE? A POST-HOC SUBGROUP COST-EFFECTIVENESS ANALYSIS
5:45PM - 6:00PM
Rädke A 1 , Hoffmann W2 , Michalowsky B2 1 German Center for Neurodegenerative Diseases (DZNE), Greifswald, Germany, 2 German Center for Neurodegenerative Diseases, Greifswald, Germany
OBJECTIVES: Collaborative dementia care aim to provide optimal treatment for people with dementia (PwD). Treatment and care needs are dependent on patients’ sociodemographic and clinical characteristics. We conduct a cost-effectiveness analysis under consideration of such characteristics to detect subgroups that benefits most from the intervention and for those a significant cost-effectiveness could be achieved. METHODS: Data were retrieved from the DelpHi-trial and analysis was based on 444 participants. Healthcare resource use, costs, QALY and incremental cost per QALY gained were measured over 24-month. Subgroup cost-effectiveness analysis was used to assess differences in variables. The probability of cost-effective was calculated at a wide range of willingness-to-pay margins and displayed using cost-effectiveness acceptability curves. The impact of dementia care management (DCM) on costs, QALYs and individual net-monetary-benefit was assessed by using multivariate regression models and interaction terms between PwD characteristics and intervention. RESULTS: The probability of DCM being cost-effective for the entire sample was 86% at the willingness-to-pay threshold of 40,000€/QALY gained after 24 month. The probability of cost-effectiveness was higher in older PwD than in younger PwD (87% vs. 48%), in females than in males (96% vs. 16%) and in those living alone compared to those living with a caregiver (96% vs. 26%). The probability of the DCM being cost-effectiveness was much higher for PwD with a high functional (97%) and cognitive impairment (99%) compared to mild functional or cognitive deficits (69% and 76%) as well as in those having a high compared to a low comorbidity (96% vs. 27%). CONCLUSIONS: The significant interactions indicated that for some subgroups a collaborative DCM is highly cost-effective. Especially, patients being female and living alone benefits most of such approaches. For those PwDs, healthcare payers could gain the highest cost savings and the highest effects by improving QALYs when such approaches will be implemented.
ND2: THE IMPACT OF HEREDITARY TRANSTHYRETIN AMYLOIDOSIS ON WORK-RELATED OUTCOMES- A MIXED METHODS APPROACH
5:15PM - 5:30PM
Sikora Kessler J 1 , Pollock M2 , Guthrie S3 , White MK4 , Raymond K4 1 Optum, San Antonio, TX, USA, 2 Akcea Therapeutics, Carlsbad, CA, USA, 3 Biopharma Strategic Consulting, LLC, San Francisco, CA, USA, 4 Optum, Johnston, RI, USA
OBJECTIVES : To examine the impact of hereditary transthyretin amyloidosis (hATTR) on patients’ work-related outcomes through evidence from observational survey data and qualitative interviews. METHODS : A mixed methods embedded design was used to examine the impact of hATTR on adults in the US. Quantitative data based on 64 completed surveys, collected in an observational, online study, included the Work Productivity and Activity Impairment Questionnaire (WPAI) and additional employment-related questions. Qualitative data were based on 11 concept elicitation interviews with hATTR patients who were in the workforce at symptom onset. RESULTS : In the observational study, 20 of the 64 patients were employed in the 7 days prior to completing the WPAI and were absent an average of 13% of their work week, experienced 24% work impairment, and 30% work productivity loss due to hATTR. Across all 64 patients, the average activity impairment due to hATTR was 49%. During qualitative interviews, over half (54%) described feeling less productive and effective at work due to hATTR, and 73% of patients expressed emotional loss and frustration from leaving the workforce prematurely due to hATTR symptoms. Patient reported hATTR symptoms, which caused them to leave the workforce prematurely, included: problems with mobility, manual dexterity, pain, fatigue, GI issues, and cognitive difficulties – such as trouble focusing, difficulty finding words, memory issues, and “feeling loopy.” Half (50%) attributed both treatment burden and symptom burden to early workforce exit. CONCLUSIONS : Patients with hATTR experienced high levels of employment interference and disruption. Symptoms and treatment burden of hATTR led to early transitions out of the workforce and serious negative impacts on patients’ overall independence and emotional well-being. This mixed methods design allowed data collection on a large sample in a rare condition to be complemented with in-depth narrative data from qualitative interviews, providing context on how early workforce exits impact patients’ lives.
ND1: HEALTHCARE RESOURCE USE IN HEADACHE SPECIALIZED CENTERS TO MANAGE MIGRAINE PORTUGUESE PATIENTS WHO HAVE FAILED PREVIOUS PROPHYLACTIC TREATMENT
5:00PM - 5:15PM
Silva C 1 , Laires P2 , Ritter S3 , Snellman J4 1 Novartis Farma, Produtos Farmacêuticos SA, Setúbal, 11, Portugal, 2 Novartis Farma, Produtos Farmacêuticos SA, PORTO SALVO, 11, Portugal, 3 Novartis Pharma AG, CRANFORD, NJ, USA, 4 Novartis Pharma AG, Basel, Switzerland
OBJECTIVES: To characterize migraine-related healthcare resource use (HCRU) in patients who have failed at least one migraine prophylactic treatment in headache specialized centers in Portugal. METHODS: BECOME observational study was conducted across 17 European countries and Israel between Nov2017-Aug2018 entailing part 1 (cumulative-hospital data) and part 2 (patient-level data). Part 2 included patients aged 18-65 years, attending headache specialized centers as outpatients/inpatients, migraine diagnosis, > 4 monthly migraine days (MMD) and evidence of > 1 prophylactic treatment failure (TF) in the past 5 years. Annualized self-reported HCRU (except medication) were calculated for the part 2 Portuguese sample. RESULTS: The Portuguese sample (n=103) had 99% females, mean age of 43.8 (SD=11.4), mean MMD of 10.4 (SD=6.1), 40% TF1, 29% TF2 and 31% TF3+. Patients attended a mean of 3.5 (SD=4.8) neurologist visits/year, 1.8 (SD=8.9) hospital non-medical visits/year, 1.1 (SD=1.9) emergency admissions/year and 0.1 (SD=0.5) hospitalizations/year and performed 0.3 (SD=0.8) CT scans/year and 0.1 (SD=0.3) MRI/year. TF1/TF2+/TF3+ patients averaged 2.6/4.1/5.9 neurologist visits/year, 2.1/1.6/3.1 hospital non-medical visits, 1.0/1.1/1.4 emergency admissions/year, 0.0/0.1/0.2 hospitalizations/year, 0.5/0.2/0.3 CT scans/year and 0.1/0.1/0.1 MRI/year. CONCLUSIONS: Our results shows that number of neurologist visits, emergency admissions and hospitalizations seems to increase with number of prophylactic TFs, which highlights a higher healthcare need in more difficult-to-treat patients.
IS DIGITAL HEALTH INTEROPERABILITY ENSURED FOR RESEARCH PURPOSES?
ISSUE
: Healthcare systems stakeholders are looking to reduce inefficiencies, remove redundancies, personalize treatments, improve healthcare quality, and patient access, whilst trying to reduce healthcare costs. These changes are under way and digital health solutions are in a unique position to help enable this process. Lack of data interoperability among different types of digital health solutions remains an issue. The exchange of data across different types of digital health solutions will continue to be debated. To ensure value for money, end users (patients) and producers are required to provide all of their tracking data. The question remains as to how we can ensure data interoperability for meaningful research while pooling the data into analysis.
OVERVIEW
:
This panel will debate the pros and cons of research data and steps needed to ensure digital health data interoperability. Carl Asche will moderate and set the stage with an overview of our Digital Health definition and the types of changes that the evolution of digital health is producing. He will question panelists to debate on: Can we ensure the interoperability between digital health data types? Is there a difference in terms of digital health type data collected and risk of interoperability implementation gone wrong at the stakeholder level? Real life examples will be used. Ken will argue that we have to deal with the data compatibility contained in different databases, Vlad will argue that each digital health type data sole extraction is a viable option and Katarzyna will argue that there are variations between the two approaches. All three panelists and the moderator work globally and will address different geographic region perspectives. Academia, researcher, payer, industry and patient¹s views will be provided. Q&A part of the session will be 15 minutes long and is allotted for interactive audience participation.
Moderators
Carl V. Asche, PhD
University of Illinois College of Medicine at Peoria, Peoria, IL, USA
Panelists
Katarzyna Kolasa, PhD
Kozminski University, Warszawa, Poland
Katarzyna Kolasa has more than 20 years of academic and business experience in the healthcare sector. She holds a PhD degree in health economics and is an author of more than 60 publications. Katarzyna Kolasa reviews manuscripts submitted to Health Policy, Value in Health, Expert Review of Pharmacoeconomics & Outcomes Research, International Journal for Equity in Health and International Journal of Technology As-sessment.
Katarzyna Kolasa heads the Health Economics & Healthcare Management Division (HeM) at the Kozminski University. She is the leader of the International Master Pro-gram Health Economics & Big Data at the Kozminski University financed by the re-search grant of the National Center for Research and Development which was listed as the best project in the POWER 2018 call.
In the past, she worked at both global and regional Health Economics & Outcomes Re-search (HEOR) functions at AstraZeneca and BiogenIdec being based in Sweden and Switzerland respectively. At Bristol Myers Squibb and Lundbeck she was leading Mar-ket Access teams in Central Eastern European (CEE) and Nordic Region. Her practical skills in the field of health economics were developed at the Kalmar County Council in Sweden as well.
For the last five years, she has been developing experience with the pricing &reimbursement challenges in the field of medical devices. Since 2016, Katarzyna has been representing Straub Medical as Global Market Access Lead being the External Principal Consultant for that Swiss family owned company. Before that, Katarzyna was employed as a Senior HEOR Director at GE Healthcare.
Her extensive knowledge in the field of health economics was acquired at University of York, University of Lund, and University of Bergen as well as during International Doc-toral Courses in Health Economics and Policy organized by the Swiss School of Public Health.
Katarzyna was nominated by the Ministry of Science to represent Poland in the COST Action CA17117, COST Association „Towards an International Network for Evidence-based Research in Clinical Health Research”. Since January 2019, she is working on the establishment of Global Special Interest Group for Digital Health at ISPOR.
Licínio Kustra Mano
European Commission, DG SANTE, Brussels, Belgium
Ken Redekop, PhD
Erasmus University Rotterdam, Rotterdam, Netherlands
Vladimir Zah, DPhil
ZRx Outcomes Research Inc., Mississauga, ON, Canada
CAN AND SHOULD EVIDENCE / PREFERENCES COLLECTED FROM PATIENTS TILT THE BOARD IN FAVOUR OF ACCEPTING A NEW TECHNOLOGY?
ISSUE
: The panel will explore challenges and opportunities from a multi-stakeholder perspective in incorporating patient insights into HTA deliberations, and discuss the ‘weight’ of the patient voice in decision-making. The debate will be enlivened and made relevant by focusing on a real-life case study.
OVERVIEW
: The case study (ultrasound-guided ablation techniques in nodular thyroid disease) will be used to discuss the value, methodology and approach(es) for integrating patient preference/evidence into HTA process. This new non-invasive technique can be used to remove a range of benign thyroid nodules without surgery and is associated with improved QoL for patients since no surgery is involved. However, since costs are the same there is no incentive for hospitals to introduce ablation, change supplies and train staff but patients prefer it on the basis of better QoL. Questions for the panel include:
What is the value of incorporating patient evidence/preference into HTAs? Do we have metrics/evidence? Is there economic value? What is the potential consequence of NOT incorporating patient evidence/preference? What do HTA bodies consider robust patient evidence? What methodology should be used? Does patient evidence/preference carry equal weight as other ‘traditional’ evidence – if not – why not? Are different HTA bodies aligned in incorporation/use of patient evidence? Is better alignment/standardization required? What tools or guidance is there/are needed? Could guidance from other PE initiatives be tailored for HTA use? If so how and by who - through co-creation? The session will provide interactive opportunities for audience participation in debate of key issues. Attendance will benefit: clinicians (understanding the value of patient involvement); payors, statisticians and patient organization representatives (understand that approaching an issue from an economic perspective can lead to important outcomes for patients); and economists (insights on issues that impact meaningful patient involvement in HTA decisions).
Moderators
Elisabeth Oehrlein, PhD, MS
National Health Council, Washington, DC, USA
Panelists
Paola Kruger, MA
Accademia dei Pazienti/EUPATI Italia, Rome, Italy
Andrea Marcellusi, PhD
Faculty of Economics, Centre for Economic and International Studies (CEIS)-Economic Evaluation and HTA (EEHTA), University of Rome Tor Vergata, Rome, Italy
Giovanni Mauri
European Institute of Oncology, IRCCS, Milan, Italy
REDUCTION OF BIAS OR A BURDEN? THE USE OF INDIVIDUAL-PATIENT MODELS FOR SUBMISSION TO HTA AUTHORITIES
ISSUE
: Decision analytic models are pivotal to informing healthcare decisions at HTA authorities (e.g. NICE). Compared with commonly used cohort models, individual patient models can offer added value by adding flexibility to incorporate patient heterogeneity and/or non-linearity between model parameters and outcomes (i.e. “Jensen’s inequality”). Ignoring this can lead to bias (e.g. non-linearity is a feature that is present in many decision analytic models). Despite these potential benefits, individual-patient modelling is not the default approach both in the scientific literature or in submissions to HTA authorities. Barriers that are typically mentioned (in the literature) include that individual-patient models are generally more complex to develop, debug and communicate to non-experts, and increase the computational burden as well as data requirements.
OVERVIEW
: This issue panel will explore the current and future role of individual-patient models for submissions to HTA authorities and will be of interest to all stakeholders involved in submissions to HTA authorities (e.g. decision-makers, analysts and companies). Manuela Joore, the panel moderator, will provide an overview of pros and cons of individual-patient models compared with cohort models and will raise key questions for the panellists to debate. Subsequently, the pros and cons of individual-patient models as well as barriers and facilitators to their use will be discussed from different perspectives. This includes 1) the consultant perspective (Jaime Caro) related to the preparation of the economic model submitted to HTA authorities; 2) Evidence Review Group (ERG) experiences (Bram Ramaekers) related to the independent review of individual-patient models and; 3) the NICE Appraisal Committee perspective (Paul Tappenden) related to the appraisal of individual-patient models.
Moderators
Manuela Joore, PhD
Maastricht University Medical Centre+, Maastricht, Netherlands
Manuela A. Joore graduated from Health Policy, Economics and Management (Maastricht University) in 1996. Following her graduation she has worked as a researcher in the field of Health Technology Assessment at the Maastricht University Medical Center and Maastricht University. She got her PhD in Economics in 2002 (Maastricht University) on the topic of health economic evaluation. Since 2006 Manuela has been teaching methods of Health Technology Assessment at Maastricht University and at other organizations. She is the developer and coordinator of the 'Cost-effectiveness modelling methods' course of the Health Sciences Research Master. She is a member of the 'Scientific Advisory Committee' of the National Health Care Institute. She is also a member of the 'Rational Pharmacotherapy' committees of the Netherlands Organisation for Health Research and Development (ZonMw). She is on the HTA board of the Dutch Centre for Personalised Cancer Treatment. Manuela leads a health economics evidence review group for the National Institute for Health and Care Excellence in the United Kingdom.
She holds a chair on 'Health Technology Assessment and decision-making' at Maastricht University. Her research focus is on (mostly model-based) health economic evaluation in a large variety of clinical areas. She uses economic evaluation to inform decision making regarding the (further) development, reimbursement, and use of healthcare technologies. She is (co-)author of over 120 peer-reviewed papers and is/was supervisor of 16 PhD students. She is involved in numerous Health Technology Assessment studies as an HTA-project leader and promotor. The total number of national and international grants obtained as a (co-)applicant adds up to more than 30 with a total sum of more than 13 million Euros.
Panelists
J. Jaime Caro, MDCM, FRCPC
Evidera, Waltham, MA, USA and University McGill, Canada, Waltham, MA, USA
J. Jaime Caro, MDCM, FRCPC, FACP, is chief scientist at Evidera where he advances Evidera’s leadership in developing and applying novel techniques in modeling, health economics, comparative effectiveness, epidemiology, and outcomes research. Dr. Caro is also adjunct professor of Medicine, Epidemiology and Biostatistics at McGill University, and professor in Practice at the London School of Economics. He also lends his teaching ability to other academic institutions such as Thomas Jefferson University School of Population Health.
Dr. Caro continues to pioneer new methodologies. In an effort to provide an alternative to the well-known cost per QALY technique and avoid many of the latter’s problems, he is working on a broader approach to valuing health benefits. He is also further developing DICE, the unified approach to health economic modeling that he has created. Working with health technology assessment agencies and academic groups, he is formalizing this innovation to enable rapid, standardized and less error-prone development of decision-analytic model. Previously, Dr. Caro adapted an engineering technique – discrete event simulation – to model diseases and their treatment and extended it further to simulate the design of clinical trials and other types of studies, something which has been particularly effective in helping design pragmatic clinical trials. He has also applied the technique to provide comparative effectiveness information in the absence of head-to-head trials in a new method called Simulated Treatment Comparison. In response to increasingly frequent requests by authorities to provide data to support the value of new interventions, Dr. Caro has applied simulation techniques to make post-marketing registries more feasible and efficient – an approach called SAVES.
On behalf of the German health technology assessment agency, he proposed an innovative approach to the assessment of health technologies, involving the efficiency frontier. As part of his work with governments, he has helped the World Bank Institute and the InterAmerican Court for Human Rights address the growing problem of Supreme Courts overriding health care system decisions and ordering them to provide treatments that had been considered unwarranted. After leading the Quality Assurance for Modeling Studies Task Force, jointly sponsored by ISPOR, Academy of Managed Care Pharmacy and the National Pharmaceutical Council, and the ISPOR-SMDM Good Modeling Practices Task Force, endorsed by the Society for Medical Decision Making, Dr. Caro was recently named co-chair of the ISPOR Science and Research Committee and has been awarded the Marilyn Dix Smith Leadership Award.
Bram Ramaekers, PhD
Maastricht University Medical Centre+, Maastricht, Netherlands
Paul Tappenden, PhD, MSc
ScHARR - University of Sheffield, Sheffield, United Kingdom
18:00 - 19:00
POSTER AUTHOR DISCUSSION HOUR - SESSION 4
Attendees have the opportunity to interact with poster presenters during this hour-long session.
18:00 - 19:30
ISPOR BOOTH EVENT - WOMEN IN HEOR NETWORKING RECEPTION
Join us for the ISPOR Women in Health Economics and Outcomes Research (HEOR) reception in the Exhibit Hall (Booth #C3-052). Meet the speakers from the Women in HEOR Forum and network with others who support the advancement of women in the field of HEOR.
NETWORKING RECEPTION IN THE EXHIBIT HALL
19:30 - 21:00
"DINE-AROUND" WITH WOMEN IN HEOR (offsite)
Enjoy the perfect opportunity to sample some of Copenhagen’s delicious foods while engaging in conversations with your colleagues from the Women in Health Economics and Outcomes Research (HEOR) Initiative. We are offering dine-around options during the conference. This special dining option is limited to groups of no more than 10 attendees and features a variety of Copenhagen’s restaurants. Each group has a designated dinner host(s) who is coordinating the event at that restaurant venue. Participants will cover their own cocktails and dinner. Tables fill up quickly, so please be sure to visit the ISPOR Women in HEOR Initiative webpage (
https://www.ispor.org/strategic-initiatives/more/women-in-heor ) to reserve your seat!
Wed 6 Nov
7:30 - 10:30
8:30 - 9:30
BREAKOUT SESSION 9
COST-EFFECTIVENESS STUDIES
CC2: ECONOMIC ASSESSMENT OF A HIGH DOSE VERSUS A STANDARD DOSE INFLUENZA VACCINE IN THE US VETERAN POPULATION- ESTIMATING THE IMPACT ON HOSPITALIZATION COST FOR CARDIOVASCULAR AND RESPIRATORY DISEASE
8:45AM - 9:00AM
van Aalst R 1 , Russo E2 , Neupane N2 , Mahmud S3 , Mor V4 , Wilschut J5 , Samson S6 , Chit A1 , Postma M5 , Young-Xu Y2 1 Sanofi Pasteur, Swiftwater, PA, USA, 2 Veterans Affairs Medical Center, White River Junction, VT, USA, 3 University of Manitoba, Winnipeg, PA, USA, 4 Brown University School of Public Health, Providence, PA, USA, 5 University of Groningen, Groningen, Netherlands, 6 Sanofi, Bridgewater, NJ, USA
OBJECTIVES Cost savings associated with high-dose (HD) as compared to standard-dose (SD) influenza vaccination in the United States (US) Veteran’s Health Administration (VHA) population has been reported to be due to the additional prevention of hospitalizations for cardio-respiratory disease; however, the stratification of this outcome into cardiovascular and respiratory disease remains to be explored. METHODS Leveraging a relative vaccine effectiveness study of HD versus SD over five influenza seasons (2010/11 through 2014/15) in the VHA, we collected cost data for healthcare provided to these subjects at VHA as well as through Medicare services. Our economic assessment compared the costs of vaccination and hospital care for patients with cardiovascular and respiratory disease separately and combined. We calculated the instrumental variable (IV) adjusted relative vaccine effectiveness (rVE) for each of the outcomes to estimate the net savings adjusted for measured and unmeasured confounding factors. RESULTS We analyzed 3.5 million SD and 158,636 HD person-seasons. The average cost of HD and SD vaccination were $23.48 (95% CI: $21.29 - $25.85) and $12.21 (95% CI: $11.49 - $13.00) per recipient, respectively. The IV adjusted rVEs were 14% (95% CI: 7% - 20%) for cardiovascular; 15% (95% CI: 5% - 25%) for respiratory; and 14% (8% - 19%) for cardio-respiratory hospitalizations. Net cost-savings per HD vaccinated Veteran were $138 (95% CI: $66 - $200) for cardiovascular; $62 (95% CI: $10 - $107) for respiratory; and $202 (95% CI: $115 - $280) for cardio-respiratory hospitalizations. CONCLUSIONS For the five-season period of 2010/11 through 2014/15, the estimated reduction of hospitalizations for cardiovascular disease contributed substantially more than those for respiratory disease to the association between HD vaccination and net-cost savings for cardio-respiratory disease as compared to SD vaccination in the US VHA population. This study was funded by Sanofi Pasteur.
CC1: COST-EFFECTIVENESS OF SCREENING AND TREATING CHILDREN WITH FAMILIAL HYPERCHOLESTEROLEMIA EARLY IN LIFE FROM A DUTCH HEALTHCARE PERSPECTIVE
8:30AM - 8:45AM
Ademi Z 1 , Norman R2 , Pang J3 , Ference B4 , Liew D5 , Sijbrands E6 , Watts G3 , Wiegman A7 1 Monash University, Melbourne, VIC, Australia, 2 Curtin University, Perth, Australia, 3 University of Western Australia, Perth, Australia, 4 University of Cambridge, Cambridge, UK, 5 Monash University, Melbourne, Australia, 6 Erasmus University, Rotterdam, Netherlands, 7 Academisch Medisch Centrum Universiteit van Amsterdam, Amsterdam, Netherlands
OBJECTIVES : Studies have shown that atherosclerosis in heterozygous familial hypercholesterolemia (HeFH) starts at birth and is reversible in childhood. Hitherto no economic evaluation has shown the impact of screening and early treatment in children with HeFH. We investigated the impact and cost-effectiveness of offering screening and preventive treatment to children with HeFH from the perspective of the Dutch healthcare. METHODS : Screening and preventive treatment with statins was modelled to simulate the progression of ten-year olds suspected of having HeFH over a lifetime. The model consisted of three health states (alive without coronary heart disease (CHD), alive with CHD, and dead). The prevalence of HeFH in this target population was 54.13%, and the sensitivity and specificity of testing was 100%. The decision tree consisted of confirmation and immediate treatment with statins, and the comparator was usual care. Mendellian-Randomisation-Analysis data was used to understand the lifelong exposure to elevated low-density-lipoprotein- cholesterol. Costs and outcome data was sourced from the Dutch registry and other published sources. Cost-effectiveness was defined as below €20,000/QALY (quality-adjusted life years) gained, using incremental cost-effectiveness ratios (ICERs), compared with usual care. All future benefits and costs were discounted annually by 1.5% and 4% respectively. RESULTS : Screening and early treatment at age 10 years compared with usual care would save 4.22 life years gained (LYG) and 4.13 QALYs per person (undiscounted). Discounted results showed that an initiation of treatment at age 10 years compared with usual care was predicted to achieve 2.18 LYG and 2.22 QALYs gained per person, at an additional cost of €8,617. These equated to ICERs of €3957 per LYG gained and €3880 per QALY gained. CONCLUSIONS : Screening and initiation of statins in children with HeHF from age 10 years is highly cost-effective over a lifetime. Our findings and conclusion are conditional on the assumptions inherent in our health economic model.
CC4: COST-EFFECTIVENESS ANALYSIS OF PERTUZUMAB WITH TRASTUZUMAB AND CHEMOTHERAPY COMPARED TO TRASTUZUMAB AND CHEMOTHERAPY IN THE ADJUVANT TREATMENT FOR PATIENTS WITH HER2-POSITIVE EARLY BREAST CANCER AT HIGH RISK OF RECURRENCE IN CHINA
9:15AM - 9:30AM
Guan X 1 , Li H1 , Chen Q2 , Hao C3 , Li J4 , Wang Y5 , Zhang J4 , Xu H6 , Liu C7 , Chu Y8 , Ma A1 1 China Pharmaceutical University, Nanjing, China, 2 Guangdong Traditional Chinese Medical Hospital, Guangzhou, China, 3 Tianjin Medical University Cancer Hospital, Tianjin, China, 4 Fudan University Shanghai Cancer Center, Shanghai, China, 5 Shandong Cancer Hospital, Jinan, China, 6 China Pharmaceutical University, Nanjing, 32, China, 7 Shanghai Roche Pharmaceuticals Ltd, Shanghai, China, 8 Shanghai Roche Pharmaceuticals Ltd, Beijing, 11, China
OBJECTIVES: To estimate the cost-effectiveness of pertuzumab with trastuzumab and chemotherapy (PHT) vs trastuzumab and chemotherapy (HT) as the adjuvant treatment for Chinese patients with HER2-positive early breast cancer at high risk of recurrence (HR-negative or node-positive). METHODS: A 6-state Markov model with monthly cycle was constructed to estimate the lifetime incremental cost-effectiveness ratio (ICER). Main clinical input was the time spent in invasive Disease Free Survival state, estimated by parametric extrapolations to the Chinese subgroup data observed in the clinical trial APHINITY. Utilities were calculated from EQ-5D of Chinese patients in APHINITY with Chinese Tariffs and the published literatures. The modelled adjuvant cost comprised drugs, administration, adverse events management costs, follow-up and therapeutic cost. Furthermore, indirect costs were included when analyzed from the society perspective. All costs were mainly obtained from real world data and local published resources. Costs and outcomes were both discounted at 5%. Sensitivity analysis were conducted to verify the robustness of the results. RESULTS: PHT provided 1.02 more QALYs with additional costs than HT. From healthcare system perspective, the ICER was CNY 115,329/QALY. Further 26% lower indirect costs in PHT group resulting an ICER of CNY 107,400/QALY from the society perspective. Both ICERs were between 1~2 times GDP per capita, far below the local threshold of 3 times GDP per capita in 2018 (CNY 193,932). Acquisition cost of pertuzumab is partially offset by the prevention of disease recurrences over the time. Cost for managing recurrences in PHT was 42% lower than that in HT group. Probabilistic sensitivity analysis showed that PHT was more cost-effective in more than 70% simulations at local threshold regardless of the perspective. CONCLUSIONS: Compared to HT, PHT is more cost-effective in the adjuvant treatment for patients with HER2-positive early breast cancer at high risk of recurrence in China.
CC3: COST-EFFECTIVENESS ANALYSIS OF VORETIGENE NEPARVOVEC VERSUS BEST SUPPORTIVE CARE IN PATIENTS WITH RPE65-MEDIATED INHERITED RETINAL DYSTROPHY- A FRENCH HEALTHCARE SYSTEM PERSPECTIVE
9:00AM - 9:15AM
Cariou C 1 , Baba J1 , Gherardi A2 , Roze S2 , Viriato D3 1 Novartis Pharmaceuticals, Rueil-Malmaison, 75, France, 2 HEVA HEOR Sarl, Lyon, France, 3 Novartis Pharma AG, Porto Salvo, 11, Portugal
OBJECTIVES : An economic model was developed to evaluate the cost-effectiveness of voretigene neparvovec (VN) compared to best supportive care (BSC) in individuals with RPE65-mediated inherited retinal dystrophy (IRD), from a French healthcare system perspective. Methods were based on HAS guidelines and international good research practices for modelling. METHODS : A Markov model was used and included six health states (HS) based on AMA visual deficiencies classification: Moderate Visual Impairment (VI), Severe VI, Profound VI, vision limited to “counting fingers”, vision limited to “hand motion” to “no light perception” and death. A lifetime horizon was used. Transition probabilities were calculated based on the results from the VN phase III trial and a natural history study. The economic endpoints used in the model were blindness-free years (BFY) and quality-adjusted life years (QALYs). Utility data from a vignette study were used. Resource utilization and costs included: disease-related costs (diagnosis, mutation testing, paramedical care and follow-up), drug costs (acquisition, administration and adverse events), costs of medical transport and accommodation and death. RESULTS : Patients treated with VN stayed longer in better HS while patients in the BSC arm progressed more quickly to worse HS. The treatment with VN results in a gain of 11.7 BFY and 4.5 QALY versus BSC. The ICER for VN versus BSC was €51, 552 per BFY and €132, 607 per QALY. Deterministic and probabilistic sensitivity analyses generally showed consistency with base case findings. When additional scenarios were explored, ICERs were most sensitive to variations in the multi-state model parameters and duration of treatment effect. CONCLUSIONS : VN is the first gene therapy approved in RPE65-mediated IRD and represents a clinically significant advancement in the management of this disease. VN was associated with an important gain of QALYs versus BSC and can be considered a cost-effective therapy in this orphan disease.
NEW FRONTIERS IN HEALTH TECHNOLOGY ASSESSMENT RESEARCH
HT2: IS THERE POTENTIAL FOR JOINT HEALTH TECHNOLOGY ASSESSMENT (HTA) IN EUROPE? AN EVALUATION OF EUNETHTA JOINT CLINICAL ASSESSMENTS VS. NATIONAL APPRAISALS IN ONCOLOGY
8:45AM - 9:00AM
Jose R 1 , Ostawal A2 , Arca E3 , Hartl K4 1 Pharmerit International, Rotterdam, ZH, Netherlands, 2 Pharmerit International, Berlin, Germany, 3 Pharmerit International, Rotterdam, Netherlands, 4 Pharmerit International, Berlin, BE, Germany
Background Europe is striving towards greater cooperation in Health Technology Assessment (HTA). While there is increasing focus on the uptake of EUnetHTA joint clinical assessments (JCA), challenges need to be identified and addressed, before current efforts can translate into seamless and efficient HTA harmonization in Europe. Objective: To evaluate the outcomes assessed and methods employed in the JCA (within the joint action 3 [JA3] framework) and the appraisals performed by the national HTA bodies (NICE, G-BA and HAS) in oncology. Methods: We conducted a qualitative evaluation of the EUnetHTA JCA and national-level HTA appraisals from the HAS, G-BA/IQWIG and NICE. For reasons of consistency and to minimize variability, we chose the same three products and indications across all agencies – namely, alectinib in ALK+ advanced non-small cell lung cancer (NSCLC), midostaurin in acute myeloid leukemia (AML), and regorafenib in hepatocellular carcinoma (HCC). Results: Across the three JCAs, there seems to be consistent focus on survival and health-related quality of life outcomes, and an increasing body of evidence has been included over time, to enable indirect treatment comparisons. JCAs have, at the very least, included comparators as deemed relevant by HAS, G-BA/IQWIG and NICE. There is heterogeneity in the use of safety endpoints as well as in the use of indirect treatment comparisons and patient engagement considered by the JCA and those used for decision-making by national HTAs. Conclusion: The adoption of JCA, especially when operationalized through the European Commission (EC) regulation could potentially lead to a more agile system which can allow EU member states to re-allocate resources for HTA. This is however, dependent on the development of clear guidance on what constitutes appropriate quality of evidence and the creation of a framework outlining an optimal process that would deliver robust evidence while maximizing efficiency in HTA across the EU.
HT4: INCLUSION OF PATIENT INPUT IN VALUE AND HEALTH TECHNOLOGY ASSESSMENT
9:15AM - 9:30AM
Desai B 1 , Mattingly TJ1 , Subedi P2 , Pham NN3 , Frailer M1 , Yang J1 , Perfetto EM4 1 University of Maryland School of Pharmacy, Baltimore, MD, USA, 2 Pfizer, New York, NY, USA, 3 University of Washington, Seattle, WA, USA, 4 National Health Council, Washington, DC, USA
OBJECTIVES : Inclusion of patient input in value and health technology assessment (V/HTA) processes is growing and evolving. However, it is unclear how solicitation and implementation of patient input is taking place across various V/HTA organizations worldwide. As healthcare globally shifts towards a patient-centered system, identifying and benchmarking current practices in V/HTA patient engagement could help to enhance improvement those practices. Our objective was to evaluate V/HTA organizations’ requirements for and use of patient input, comparing practices across U.S. value assessment organizations and ex-U.S. health technology assessment bodies. METHODS : Methods documents from 11 V/HTA organizations were screened and systematically abstracted for mentions of or requirements for patient involvement and/or input. Two reviewers evaluated each document and a third reviewer acted as tie-breaker when needed. Up to three 2018 reports for each organization were reviewed to assess actual elicitation or use of patient input and corroboration with methods documentation. RESULTS : Of 7 U.S. organizations evaluated, 2 stated patient stakeholder input was accepted and 1 provided details in a report on methods used to solicit patient input. All 4 ex-U.S. organizations stated input from patient stakeholders was accepted; however, the methods for solicitation of input were often not clear in reports. Use of patient input in V/HTA reports varied across organizations. While ICER and CADTH report using patient input to inform economic model inputs, whereas IQWIG utilizes patient input to assist with specification of patient-relevant outcomes and subgroups in the early phases of an assessment. CONCLUSIONS : It appears that, in general, U.S. value assessment organizations are lagging behind their HTA counterparts in Canada and Europe in soliciting and incorporating patient input. Good practice in V/HTA patient engagement should be identified and shared among V/HTA organizations.
HT3: WHAT INFLUENCES THE PROBABILITY TO BE SUCCESSFUL IN AN ONCOLOGY HTA? AN ANALYSIS OF ONCOLOGY HTA DECISIONS IN GERMANY, SPAIN AND THE UK
9:00AM - 9:15AM
Hardtstock F 1 , Kocaata Z1 , Wilke T2 , Rebollo P3 , Garcia A4 , Heeg B5 1 Ingress-Health HWM GmbH, Wismar, MV, Germany, 2 Ingress-Health HWM GmbH, Wismar, Germany, 3 Ingress-Health Spain S.L., Oviedo, O, Spain, 4 Ingress-Health Nederland B.V., rotterdam, Netherlands, 5 Ingress-Health, Rotterdam, Netherlands
OBJECTIVES This study aimed to investigate the factors affecting decisions on oncology-related health technology assessments (HTAs) in Germany, Spain and the UK. METHODS HTA dossiers submitted to German G-BA and UK NICE and therapeutic positioning reports issued by Spanish AEMPS until April 2019 were analyzed. Assessments with added benefit in Germany, inclusion in the national reimbursement list in Spain and reimbursement recommendation with/without restrictions in the UK were interpreted as positive decisions. Decisions were regressed on assessments’ general characteristics and type of evidence presented in dossiers in a logistic regression framework. RESULTS The study included 436 assessments (75.2% positive) on 122 agents [GER: 153 assessments/73.9% positive; ESP: 72/65.3%; UK: 221/79.6%]. Controlling for all general characteristics such as indication type and assessment year, the following characteristics were associated with a higher probability of a positive decision: orphan status (OR: 3.19, p<0.01), presented RCT evidence (OR: 2.24, p=0.04), assessment in the UK (OR: 2.14, p=0.02, reference group: GER), whereas first-line treatment (compared to further treatment lines; OR: 0.63, p=0.07) and inclusion of evidence from indirect treatment comparisons (ITC; OR: 0.56, p=0.06) was associated with a negative success probability. CONCLUSIONS Evidence from RCTs and orphan status increase the probability of a successful HTA. Obviously, presentation of ITC evidence, especially in GER and ESP, is an indicator for weaker head-to-head evidence, which might explain the negative association between ITC evidence and HTA success.
HT1: DOES HIV COINFECTION AFFECT HCV REIMBURSEMENT DECISIONS?
8:30AM - 8:45AM
Gizaw N 1 , Rubinstein J2 , Adler B3 , Guill EW4 1 Context Matters (a Decision Resources Group company), Jersey City, NJ, USA, 2 Context Matters (a Decision Resources Group company), New York, NY, USA, 3 Context Matters (a Decision Resources Group company), Dallas, TX, USA, 4 Context Matters (A Decision Resources Group Company), New York, NY, USA
OBJECTIVES : The aim of this analysis is to assess whether including human immunodeficiency virus (HIV) coinfected patients plays a role in reimbursement decisions for hepatitis C virus (HCV) treatments for adult patients in Scotland, the United Kingdom (UK), Canada, Australia, France, and Germany. METHODS : The analysis used Scottish Medicines Consortium (SMC), National Institute for Health and Care Excellence (NICE), Canadian Agency for Drugs and Technologies in Health (CADTH), Pharmaceutical Benefits Advisory Committee (PBAC), Haute Autorité de Santé (HAS), and Gemeinsamer Bundesausschuss (G-BA) reimbursement decision data between 2009 and 2018. Non-submissions, differed, and no decisions were excluded. Statistical analyses were performed. RESULTS : A total of 288 reimbursement decisions were included in the model of which, 84.72% (n=244) were positive. Out of all the agencies, G-BA gave the highest positive decisions, followed by HAS, with corresponding rates of 100% (n=70) and 97.6% (n=40), respectively. Among all the HCV therapies HAS approved for reimbursement, 95.12% (n=39) gave substantial improvement of which, all (n=2) treated HIV-coinfected patients. Conversely, the distribution of G-BA’s benefit scores was sporadic: 83% (n=6) of HCV therapies with HIV coinfection provided either a minor or unquantifiable benefit over comparators versus 35.72% (n=25) of all positive decisions combined. Approximately 45.71% (n=32) of HCV treatments and 16.67% (n=1) of HCV therapies for HIV-coinfected patients provided no additional benefit. HCV treatments for patients with HIV coinfection accounted for 4.51% (n=11) of positive decisions whereas HCV therapies alone constituted 95.49% (n=233) of the recommendations. Fisher’s exact test demonstrated that there is no significant relationship between receiving positive reimbursement decisions and submissions for HCV therapies for adult patients who have HIV coinfection (p=0.1558). CONCLUSIONS : All HCV therapies for patients coinfected with HIV were recommended. However, due to the small number of decisions for this population, it's unclear whether including an HIV-coinfected subgroup increases the rate of receiving positive decisions.
USING PATIENT PREFERENCE DATA TO SUPPORT CLINICAL TRIAL DESIGN- CURRENT PRACTICE, OPPORTUNITIES AND CHALLENGES
PURPOSE
: To identify, illustrate and debate the opportunities and challenges associated with using patient preference data to improve clinical trial design. The workshop will benefit those responsible for designing or assessing the design of trials, and health economists inputting into the design of trials.
DESCRIPTION
:
Background : The last 10 years have seen trial designs becomes more complex. Consequently, greater numbers of trials are failing to meet their recruitment targets. Initiatives to stimulate patient focused drug development have encouraged engagement with patients to inform trial design, including informing endpoint selection, the content of outreach material, and the location, schedule, timing and invasiveness of assessments. The evidence suggests that patient centred trial design increases enrolment rate and improves time to and probability of launch. Patient insight on trial design has conventionally taken the form of qualitative data. More recently it has been proposed that quantitative patient preference data be used to optimize trial designs. A recent high-profile example being NICE’s scientific advice on the use of a discrete choice experiment to inform the selection of trial endpoints.
Leaders : Elisabeth Oehrlein will provide a patient advocacy group perspective, overview patient engagement in trial design, using case studies to illustrate the ways that patient centered research has informed trial design, summarizing existing good practice, and identifying ways that quantitative patient preference data could complement qualitative patient insight on design trials. Deborah Morrison will provide a HTA agency perspective on how patient centred trial design might impact product evaluation, and the role of scientific advice in patient preference study design. Sebastian Heidenreich will provide a methodological perspective, highlighting some of the challenges that will need to be addressed if patient preference data is to effectively influence trial design. Kevin Marsh will chair and facilitate discussions between workshop leaders and with the audience.
Discussion Leader
Sebastian Heidenreich, PhD
Evidera, Aberdeen, ABE, United Kingdom
Kevin Marsh, PhD
Evidera Ltd, 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.
Deborah Morrison, MSc, BSc (Pharmacy)
National Institute for Health and Care Excellence, Manchester, United Kingdom
Elisabeth Oehrlein, PhD, MS
National Health Council, Washington, DC, USA
INCLUDING CAREGIVER / FAMILY MEMBER QUALITY OF LIFE IN ECONOMIC EVALUATIONS – YOUR QUESTIONS ANSWERED
PURPOSE: Many health conditions have profound impacts on the quality of life of informal caregivers and family members which may be alleviated by effective treatment. However, our ability to capture these benefits in economic evaluations is hindered by uncertainty about decision-makers’ attitudes toward their inclusion, issues related to how they may be incorporated into economic models, and the availability of suitable utility data and measures for caregivers / family members. This workshop aims to answer common questions related to these issues and discuss questions and experience from the audience, as well as to outline ongoing research in the field.
DESCRIPTION: The Discussion Leaders’ presentations will consider common questions, for example:
Dr Knight of NICE will discuss whether decision-makers allow such benefits to be included in cost-utility analyses, and whether they can they be included in the basecase analysis or only in scenario analysis. Professor Brazier will consider how caregiver / family member utility can be measured, and whether preference-based measures designed for patients or existing caregiver quality of life measures are appropriate for measurement of caregiver/family member utility Dr Wolowacz will consider how utility estimates for caregivers / family members can be incorporated into economic evaluations. This workshop will be of value for researchers interested in quality of life research, health utility estimation, economic evaluation and/or health technology assessment.
Discussion Leader
John E. Brazier, MSc PhD
University of Sheffield, Sheffield, United Kingdom
John Brazier is Professor of Health Economics and Dean of the School of Health and related Research (ScHARR) at the University of Sheffield. He has more than 25 years’ experience of conducting economic evaluations of health care interventions for policy maker and published over 200 peered reviewed papers. He has a particular interest in the measurement and valuation of health for economic evaluation where he has published widely. He is perhaps best known for his work in developing a preference-based measure of health for the SF-36 (SF-6D), but with colleagues has further developed and extended these methods to a number of specific condition including measures in mental health (ReQoL), asthma, cancer, overactive bladder, dementia and epilepsy. His research has also examined issues including methods of reviewing measures, mapping between measures, valuation methods (including the use of DCE) and more recently he has been developing ways to incorporate equity concerns such as burden of disease into the weights applied to QALYs. He has been an adviser to NICE on HTA methods and was a member of the NICE Technology Assessment Committee. He is a member of the Euroqol Executive Group.
Currently he is leading a project to develop a new broader generic measure of quality of life for use in economic evaluation. It is funded by the UK MRC and the EuroQoL Research Foundation in collaboration with colleagues at the Universities of Sheffield and Kent, the Office for Health Economics and NICE, together with colleagues in 5 other countries (Australia, Argentina, Germany, USA and Singapore). For more information about the ‘Extending the QALY’ project see: https://scharr.dept.shef.ac.uk/e-qaly/welcome/
Helen Knight, PhD
NICE, Manchester, United Kingdom
Sorrel Wolowacz, PhD
RTI Health Solutions, Manchester, United Kingdom
SUBSCRIPTION-BASED MODEL FOR DRUG REIMBURSEMENT – A FAD OR THE FUTURE?
ISSUE: Subscription-based reimbursement models are where manufacturers are paid a lump sum to provide an unlimited supply of drugs for a given population for a given timed period (as opposed to revenues being linked to the volume of drugs sold). These are now being used in the reimbursement of hepatitis C therapies in Australia as well as for Medicaid recipients in Louisiana and Washington. Further, in 2018, NHS England offered Vertex an unprecedented pricing agreement whereby, in return for a guaranteed £1 billion over the next 10 years, NHSE would secure unlimited access for its existing and future cystic fibrosis therapies. The potential growth of such a new model of reimbursement offers new opportunities to meet access challenges, in terms of providing payers a more affordable option and manufacturers guaranteed revenues. However, this also poses some significant implementation, including integrating this into the traditional pricing, reimbursement and HTA processes, which this panel will discuss.
OVERVIEW: Our issue panel aims to present and discuss the differing perspectives of a UK payer (Andrew Walker), a local budget holder (Diar Fattah), and a Australian payer (Erika Turkstra) on the opportunities and challenges associated with the subscription-based pricing model. Each panelist will give a short 10-minute presentation (after a 10-minute situation overview from the moderator, Richard Macaulay) of their views on the topic of subscription-based reimbursement models before a wider 20-minute panel discussion and audience Q&A.
Moderators
Richard Macaulay, PhD
Parexel International, London, HRT, United Kingdom
Panelists
Diar Fattah, MSc MPharm
NHS DGS and Swale CCG, Kent, United Kingdom
Erika Turkstra, PhD
Parexel International, London, United Kingdom
Andrew Walker, PhD
University of Glasgow, Glasgow, United Kingdom
HOW EFFECTIVE ARE MANAGED ACCESS AGREEMENTS AS A VEHICLE FOR EARLIER REIMBURSEMENT IN THE PRESENCE OF UNCERTAINTY? LESSONS FROM THE NEW CANCER DRUGS FUND.
ISSUE
: The Cancer Drugs Fund (CDF), re-established in 2016, provides patients with earlier access to new cancer drugs and a mechanism for managed access for promising new treatments where significant clinical uncertainties exist. A key component of Managed Access Agreements (MAAs) is further data collection with the objective of addressing uncertainties identified throughout the NICE Technology Appraisal process. With a ring-fenced budget, high-cost medicines and increasing political pressure to provide earlier access, it is yet to be determined how sustainable the continued growth of the CDF will be, and how effective data collection arrangements are in resolving uncertainty at the time of reappraisal.
OVERVIEW
: This panel will debate the pros and cons of the current CDF system and explore alternative approaches to how uncertainty can be handled throughout the HTA process. Prof. Akehurst will begin the session by providing an overview of the revised CDF and corresponding MAAs including the relevant implications for future decision making. Case studies will be provided, drawing upon the findings of a detailed review of CDF recommendations made since the formulation of the revised system in June 2016. The case studies will comment on the degree with which the uncertainties identified during appraisal process correspond to data collection targeted by the CDF. Prof. Akehurst will then pose key questions for the panellists to debate:
Does the current CDF system provide an effective approach to managing uncertainties? What lessons can be drawn from how other countries implement MAAs? Are there alternative processes or mechanisms that could be developed to help improve managed access in England and more widely? These questions will enable an informed, interactive 15-minute discussion with the audience at the end of the issue panel, relating to practical steps that can be taken to improve MAAs in the future.
Moderators
Ronald L Akehurst, DSc, Hon MFPHM
BresMed Health Solutions, Sheffield, United Kingdom
Panelists
Jennifer M Lee, MBA
Janssen UK, High Wycombe, BKM, Great Britain
Stephen Palmer, Prof, PhD
University of York, Heslington, York, United Kingdom
Steve Williamson, MSc, BPharm
NHS England, London, United Kingdom
VALUE VS VALUE TRAPS- HOW TO CONTROL PRICES FOR HIGHLY EFFECTIVE BUT HIGH-PRICED THERAPIES?
ISSUE: Recent advancements in drug discovery have led to the development of innovative therapies that provide otherwise incomparable or unavailable therapeutic benefits in treating serious and life-threatening conditions. However, these drugs are very expensive. Harnessing drug innovation while controlling drug spending is one of the most challenging tasks in ensuring the sustainability of healthcare systems worldwide.
In response to growing concerns about healthcare spending, attention is turning to drug pricing as a means to manage healthcare expenditures. The use of cost-effectiveness analysis and other value-based frameworks have been proposed yet the price of new drugs continues to rise. This begs the question why existing approaches have not been effective in controlling drug prices and if there is an alternative. This will be the focus of debate for the issue panel.
OVERVIEW: Brittany Humphries will moderate the panel. She will provide the audience with a 10-minute overview of global drug spending and recently approved highly effective high-priced therapies. Each speaker will then have 10 minutes to present their perspective.
Dr. Michael Drummond will review the use of economic evaluations and value-based frameworks in drug pricing. He will discuss their strengths and limitations when applied to highly effective and high-priced therapies. Following this,
Dr. Feng Xie will introduce alternative approaches (external reference pricing and outcomes-based pricing) to control drug prices. He will discuss their limitations and offer insights into what could be a promising approach to balance affordability and incentivise drug innovation. Finally,
Dr. Nicola Magrini will present a case study of different high-priced medicines evaluated for listing in the World Health Organization Essential Medicine List that provides the basis for Universal Health Coverage programs. At the end of the discussion, the audience will have 20-minutes to ask questions. Stakeholders who would benefit from attending include HEOR researchers, payers, manufacturers, and regulators.
Moderators
Brittany Humphries, BA, MSc, PhD(c)
McMaster University, Toronto, Canada
Panelists
Michael Drummond, MCom, DPhil
University of York, York, 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.
Nicola Magrini, MD
WHO Expert Committee on the Selection and Use of Essential Medicines, Geneva, Switzerland
Feng Xie, PhD
McMaster University, Hamilton, ON, Canada
9:30 - 14:00
RESEARCH POSTER SESSION 5
EXHIBIT & POSTER HALL HOURS
9:45 - 10:45
BREAKOUT SESSION 10
EXPANDING HORIZON OF OUTCOME STUDIES IN ONCOLOGY
ON5: DEVELOPMENT AND TESTING OF PATIENT-REPORTED OUTCOME PERFORMANCE MEASURES (PRO-PMS) FOR COMPARING ONCOLOGY PRACTICES
9:45AM - 10:00AM
Stover A1 , Urick B 1 , Deal A2 , Jansen J2 , Henson S2 , Miller R3 , Smith T4 , Scholle S5 , Chiang A6 , Cleeland C7 , Deutsch Y8 , Zylla D9 , Pitzen C10 , Snyder C11 , McNiff K12 , Krzyzanowska M13 , Spears P14 , Smith ML15 , Geoghegan C16 , Basch EM1 1 University of North Carolina at Chapel Hill, Chapel Hill, NC, USA, 2 UNC Lineberger Comprehensive Cancer Center, Chapel Hill, NC, USA, 3 American Society for Clinical Oncology, Alexandria, VA, USA, 4 American Cancer Society, Atlanta, GA, USA, 5 National Committee for Quality Assurance, Washington, DC, USA, 6 Yale University, New Haven, CT, USA, 7 University of Texas MD Anderson Cancer Center, Houston, TX, USA, 8 Memorial Healthcare System, Pembroke Pines, FL, USA, 9 Frauenshuh Cancer Center, St. Louis Park, MN, USA, 10 MN Community Measurement, Minneapolis, MN, USA, 11 Johns Hopkins School of Medicine, Baltimore, MD, USA, 12 KM Healthcare Consulting, Boston, MA, USA, 13 Princess Margaret Cancer Centre, Toronto, ON, Canada, 14 UNC-Chapel Hill, Chapel Hill, NC, USA, 15 Research Advocacy Network, Naperville, IL, USA, 16 Patients and Partners LLC, New Haven, CT, USA
OBJECTIVES. Symptom management is a cornerstone of quality oncology practice. The American Society for Clinical Oncology (ASCO) established a Working Group to develop patient-reported outcome performance measures (PRO-PMs) for assessing symptom management during chemotherapy. We describe multicenter testing funded by PCORI. METHODS. Multi-stakeholder consensus and literature review identified 12 symptoms to test as potential PRO-PMs: gastrointestinal (nausea, vomiting, diarrhea, constipation, eating and drinking decreases), pain, insomnia, fatigue, dyspnea, neuropathy, depression, and anxiety. For these symptoms, questions from PRO-CTCAE were administered at 6 US academic and community oncology practices. Patients across cancer types completed questions electronically on days 5-15 of chemotherapy cycles. PRO-CTCAE scores were dichotomized to delineate clinically meaningful thresholds (0-1 vs >=2), and rates were tabulated between practices. Symptoms were selected for model testing if they were clinically actionable and had a prevalence ≥20%; between-practice variation was evaluated using χ2 . Twelve candidate sociodemographic and clinical risk adjustment variables were evaluated via Akaike information criterion testing. Risk-adjusted PRO-PM rates were calculated using observed:expected ratios via generalized linear mixed modeling. RESULTS: Among 653 enrolled patients, 607 (93%) completed questionnaires. Four of 12 symptoms met criteria for PRO-PM development: nausea, constipation, insomnia, pain. Unadjusted scores across practices ranged from 13-30% for uncontrolled nausea (χ2 p = 0.06), 19-37% for uncontrolled constipation (χ2 p = 0.12), 31-54% for uncontrolled insomnia (χ2 p = 0.04), and 36-50% for uncontrolled pain (χ 2 p = 0.30). Four risk adjustment variables met inclusion criteria: age, gender, cancer type, insurance type. Risk-adjustment yielded a modest impact on practice scores, with changes around 1-4%. CONCLUSIONS: Oncology PRO-PMs have been developed to quantify the burden of actionable symptoms at the practice level. Collecting PROs from patients at home on days 5-15 of chemotherapy cycles is feasible. Further refinement and validation is underway prior to submission for endorsement by the U.S. National Quality Forum.
ON8: CHEMOTHERAPY INDUCED NAUSEA VOMITING- VALIDATION OF RISK SCORING ALGORITHM IN PATIENTS WITH GYNECOLOGICAL AND GASTROINTESTINAL CANCERS
10:30AM - 10:45AM
Anitha D 1 , Jada H2 , Krishna Murthy M3 , Vinayak VM4 1 Ramaiah University Of Applied Sciences, bangalore, KA, India, 2 Ramaiah University Of Applied Sciences, chittoor, KA, India, 3 Faculty of pharmacy, M.S.Ramaiah University of Applied Sciences, Bangalore, India, 4 Ramaiah hospitals, Bangalore, India
OBJECTIVES The study aims to prospectively evaluate the risk scoring algorithm of Chemotherapy induced nausea-vomiting (CINV) in patients undergoing chemotherapy. METHODS All patients with Gynecological and Gastrointestinal cancers receiving chemotherapy are recruited after obtaining informed-consent. Patients are followed-up on Day-1 and Day-5 to record any evidences of acute and delayed CINV respectively through phone calls. Prior to every cycle of chemotherapy, the scoring-systems are applied to stratify patients into low and high-risk groups. Logistic-regression modelling is applied to compare the risk for grade 2 or greater CINV between patients considered to be at high and low risk. To assess the external-validity of each system, an area under the receiver-operating characteristic curve-(AUROC) analysis is performed. RESULTS : The CINV outcomes data from a total of 104 patients (until March 2019) over 326-cycles of chemotherapy was collected. The incidence of acute and delayed CINV was 42.7% and 76.4% respectively. Major significant risk-factors included younger patient age, platinum/anthracycline-based chemotherapy, low alcohol consumption, previous history of morning sickness and nausea/emetic episodes prior to chemotherapy. Both acute and delayed scoring systems had good predictive accuracy when applied to the external validation sample (acute-AUROC: 0.75; 95% CI: 0.69 to 0.80; delayed-AUROC: 0.71; 95% CI: 0.65 to 0.78). Patients identified by the scoring systems to be at high-risk were 3.8 (p = 0.005) and 6.6 (p = 0.002) times more likely to develop grade-2 or greater acute and delayed-CINV respectively. CONCLUSIONS : The present research establishes that the scoring-systems can precisely classify the patients at high-risk for acute and delayed CINV. Further, the data collected until October 2019 will be analyzed and presented at the conference.
ON7: TIME-TO-TREATMENT DISCONTINUATION (TTD) AS A PRAGMATIC PREDICTOR OF OVERALL SURVIVAL (OS) IN FIRST-LINE ADVANCED NON-SMALL CELL LUNG CANCER (ANSCLC) - REAL-WORLD PERSPECTIVE
10:15AM - 10:30AM
Hashim M 1 , Hardtstock F2 , Cizova D3 , Maywald U4 , Postma M5 , Heeg B1 , Wilke T6 1 Ingress-Health, Rotterdam, Netherlands, 2 IPAM, University of Wismar, Wismar, MV, Germany, 3 Ingress-Health HWM GmbH, Wismar, Germany, 4 AOK PLUS, Dresden, Germany, 5 University of Groningen, University Medical Center Groningen, Groningen, GR, Netherlands, 6 IPAM, University of Wismar, Wismar, Germany
OBJECTIVES: Measuring progression-free survival (PFS) in real-world is challenging; due to the variability in assessments’ timing and missing data. In aNSCLC, patient-level analysis conducted by the FDA showed that TTD may represent a pragmatic alternative endpoint for PFS. To date, the relationship between TTD and OS is yet to be tested in experimental and real-world settings. Therefore, we aimed to assess if TTD can be used as a pragmatic predictor of OS in real-world. METHODS: For this application, we limited our analysis to first-line systemic treatment in a German claims-based dataset for aNSCLC. The correlation between TTD and OS was estimated using Pearson's r, Spearman's rho, and Kendall’s τ. Kendall’s τ was computed using methods for doubly censored data. Landmark analyses were also performed at several timepoints to estimate OS in two groups of patients, conditional on TTD at a specific timepoint: the landmark time. Multivariate and univariate Cox proportional hazard models were fitted to estimate the effect of TTD on OS. Hazard ratios (HRs) together with the 95% confidence interval (CI) were estimated. RESULTS: 1,672 patients were included in the analysis. Median TTD was 5.75 months (95%CI:5.62-5.98) and median OS was 9.90 months (95%CI:9.24-10.59). Correlation between OS and TTD was statistically significant (Pearson's r=0.44 (p<.001), Spearman's rho=0.61 (p<.001) and Kendall’s τ=0.32 (p<.001). TTD was consistently found to be a strong predictor of OS at all timepoints. At a landmark timepoint of 6 months, the HR for death was 0.717 (95%CI:0.623-0.826) for patients who were on treatment compared to those who discontinued treatment. CONCLUSIONS: TTD is strongly correlated with OS in real-world, may predict OS and be used as a patient-relevant endpoint in the first-line aNSCLC. Prior to the usage of TTD in clinical trials as a key endpoint, further investigation of this relationship in different oncology settings is warranted.
ON6: IMPACT OF THE FOLLICULAR LYMPHOMA (FL) TREATMENT CONTINUUM ON A PATIENTS QUALITY OF LIFE (QOL) IN EUROPE (EU5)
10:00AM - 10:15AM
Häfliger B 1 , Massey L2 , Rider A2 , Bailey A3 , Bacon T4 1 Janssen-Cilag AG, Zug, ZG, Switzerland, 2 Adelphi Real World, Bollington, UK, 3 Adelphi Real World, USA, 4 Janssen Sciences Ireland, UC, Dublin, Ireland
OBJECTIVES : This study investigated key predictors that impacted QoL for patients with FL across EU5. METHODS : Real world data were drawn from the FL Disease-specific ProgrammeTM - a point in time study administered to haem-oncologists who completed patient record forms for the next 8 consulting patients with FL in Q2/3 2017 across EU5. QoL analysis was conducted on those that completed a patient self-completion form (PSC). Statistical methods used included Spearman’s rank correlation coefficient, bivariate analysis, and multiple linear regression. RESULTS : Data analysis was conducted on 1,534 patients diagnosed with FL, of whom 591 completed a PSC. Patients that completed a PSC were more likely to be at 1st line (31% vs 23%; p=0.0035), responding to treatment at time of study (21% vs 16%; p=0.0061) and experience fewer comorbidities (1.2 vs 1.3; p=0.0169) than those that didn’t complete a PSC. Characteristics of patients that completed a PSC were: mean age 65.1 years (SD=11.3), patients were most commonly grade 2 and stage III (43%; 41%), on their 2nd line of therapy (59%) and had an ECOG of 1 (53%). Multiple QoL measures (FACT and WPAI), ECOG score and presence of comorbidities were significantly correlated with the EQ5D-5L (p<0.05). Comorbidities and ECOG score were key predictors of the EQ5D-5L. Patients on later lines of therapy had worse QoL (line 1-line 3; p-value), including EQ5D-5L (0.82-0.59; p<0.0001), FACT-G score (67.79-54.87; p<0.0001), and FACT-Lymphoma score (109.29-87.94; p<0.0001). With increasing lines of therapy, patients receiving induction therapy had better QoL compared to those on maintenance therapy (EQ5D-5L: 0.81-0.77; p=0.5739 vs 0.86-0.23; p<0.0001, FACT-G score: 66.42-63.34; p=0.2136 vs 72.05-38.77; p<0.0001, FACT-Lymphoma score: 106.86-104.11; 0.3822 vs 116.80-57.23; p<0.0001). CONCLUSIONS : Patients on later lines of therapy and maintenance therapy, show a deterioration of their QoL, highlighting the need for novel treatment options to maintain QoL in these settings.
COMPARATIVE EFFECTIVENESS OR EFFICACY STUDIES
CE1: EVALUATION OF THE EVOLVING TREATMENT LANDSCAPE IN EARLY-STAGE/LOCALLY-ADVANCED NON-SMALL CELL LUNG CANCER (ES/LA-NSCLC)- A FORWARD-LOOKING NETWORK META-ANALYSIS (NMA) FEASIBILITY STUDY
9:45AM - 10:00AM
Rizzo M 1 , Pollack M2 , Ballew NG2 , Kulp W3 , Wissinger E2 1 Xcenda UK, Dartford, UK, 2 Xcenda LLC, Palm Harbor, FL, USA, 3 Xcenda GmbH, Hannover, Germany
OBJECTIVES: The treatment landscape in ES/LA-NSCLC is rapidly evolving with new treatment options for patients including immunotherapy (IO) and targeted therapies. Since any new treatment preparing for reimbursement may need to demonstrate relative treatment efficacy and safety against new-to-market comparator(s), we conducted a forward-looking NMA feasibility study to gain insight into considerations when developing future indirect treatment comparisons for ongoing trials with results currently unavailable in ES/LA-NSCLC. METHODS: Using systematic literature review (SLR) methodology, we reviewed SLRs/NMAs in ES/LA-NSCLC (stage IB−III) to identify currently-available curative treatment options. We supplemented this with a search via clinicaltrials.gov for ongoing trials. We extracted key information across relevant studies and conducted an NMA feasibility study, with separate networks in resectable and unresectable ES/LA-NSCLC. RESULTS: New IO and targeted therapies are currently under investigation in both resectable and unresectable disease. There are however limited options available to comparatively analyze those treatments targeting actionable alterations due to differences in patient populations and/or choice of comparator(s). For outcome selection in NMAs, the most commonly-reported endpoints in resectable ES/LA-NSCLC include major pathological response rate, disease-free survival and overall survival. A future NMA in ES/LA-NSCLC should also consider an analysis by drug-class to maximize the number of trials available for evaluation, and by specific type of treatment to guide reimbursement decision-making, although the latter approach causes some trials to disconnect from the network. Based on the primary trial completion dates reported on clinicaltrials.gov, in resectable NSCLC there will be a gradual release of data from 2020—2023, steadily increasing options for analysis over time. CONCLUSIONS: The treatment landscape for ES/LA-NSCLC is complex with limited NMAs available. Careful consideration is needed on how to maximize the connections of newer treatments within an NMA, while maintaining the validity of the analysis to guide decision-making.
CE4: COMPARISON OF NETWORK META-ANALYSIS (NMA) USING THE BAYESIAN AND FREQUENTIST APPROACH- CONCRETE EXAMPLE ON THE BASIS OF A PUBLISHED NMA IN RELAPSING REMITTING MULTIPLE SCLEROSIS AND PLAQUE PSORIASIS
10:30AM - 10:45AM
Dasari A 1 , Kommoju UJ2 , Dixit M2 , Sharma S2 , Bergemann R3 1 Evalueserve, Gurgaon, HR, India, 2 Evalueserve, Gurugram, HR, India, 3 Evalueserve, London, UK
OBJECTIVES: The Bayesian and the Frequentist methods are two different approaches for performing a network meta-analysis (NMA). The choice of the method is mostly based on preference of the statistician (Bayesian or Frequentist school) and not on a specific rationale. The objective of this study is to replicate published NMAs that have used one of the two approaches, conducting the NMA with the other approach and determining possible differences in the outcomes. METHODS: Two published NMAs with complete information of the underlying methodology and the statistical package in R and Stata were selected. NMAs selected include one on Relapsing Remitting Multiple Sclerosis (RRMS) with R package (Huisman et al., 2017) and another on Plaque Psoriasis (PP) with Stata package (Sbidian et al., 2017). Outcomes for risk ratio (RR) and rate ratio, p-score and SUCRA were used for the comparison. For the Frequentist method, netmeta package for R was used. Input data was taken from original publications. The Frequentist NMAs were run with R-Studio version 1.1. RESULTS: RRMS NMA: 16 treatments were analyzed in RRMS. For the rate ratios, no differences were observed in 8 treatments, while the difference ranged from 0.025 to 0.006 in the remaining 8 treatments. The comparison between SUCRA and p-score was identical in 15 treatments and the difference was 0.01 in 1 treatment. PP NMA: 20 treatments were assessed for PASI 90 outcome. For RR, no difference was found in 4 treatments with a mean relative difference of -0.1% for all treatment comparisons. Differences for SUCRA vs p-score were similar. CONCLUSIONS: The Bayesian and Frequentist approaches used for NMAs are similar for rate ratios and the ranking. Differences seem more related to the package used (Stata or R) than the underlying methods. Further research is necessary to investigate this.
CE2: EFFICACY OF BRODALUMAB AND GUSELKUMAB IN PATIENTS WITH PLAQUE PSORIASIS WHO ARE INADEQUATE RESPONDERS TO USTEKINUMAB- A MATCHING ADJUSTED INDIRECT COMPARISON (MAIC)
10:00AM - 10:15AM
Borg E 1 , Faurby M2 , Hampton PJ3 1 LEO Pharma, Ballerup, 84, Denmark, 2 Leo Pharma, Hvidovre, Denmark, 3 The Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK
OBJECTIVES: With an increasing number of biologics available for the treatment of plaque psoriasis, data on relative efficacy in different patient populations becomes increasingly important to optimize clinical decision making. Brodalumab and guselkumab are approved for the treatment of moderate to severe plaque psoriasis in adult patients eligible for systemic therapies. In the absence of direct randomized controlled trial data, we have used a matching adjusted indirect comparison (MAIC) to estimate the comparative efficacy in patients with an inadequate response to ustekinumab. METHODS: We used an unanchored MAIC analysis matching pooled individual patient level data (IPD) from the AMAGINE-2 and -3 trials and summary data from NAVIGATE trial, where patients had an inadequate response to ustekinumab (45/90mg). In the AMAGINE-2 & -3 trials inadequate responders switched to brodalumab 210 mg Q2W at week 16 (n=124). In the NAVIGATE trial inadequate responders switched to guselkumab at week 16 (n=135). Differences in baseline and week 16 disease characteristics were adjusted using a propensity score re-weighting method. Adjusted proportions and risk differences were calculated using a multivariate logistic regression model for Psoriasis Area and Severity Index (PASI) 90 and 100 responses, and Physician/Investigator Global Assessment (PGA/IGA) 0/1 response. RESULTS: Overall, brodalumab was associated with higher efficacy response rates compared to guselkumab. Brodalumab treatment resulted in statistically significant higher PASI 100 rates at week 36, after inadequate response to ustekinumab, compared to guselkumab of 40.8% vs. 20.0% (RD: 20.8% [95%CI: 12.0-29.5], p<0.001), and PASI 90 and PGA/IGA 0/1 rates of 64.4% vs. 51.1% (RD: 13.3% [95%CI: 4.7-21.8], p=0.002) and 50.1% vs. 36.3% (RD: 13.8% [95%CI: 1.2-26.5], p=0.031), respectively. CONCLUSIONS: Brodalumab was associated with significantly higher complete clearance rates, PASI 90 and PGA/IGA responder rates compared to guselkumab at week 36 after inadequate response to ustekinumab.
CE3: COMPARATIVE EFFECTIVENESS AMONG SINGLE AGENT CHEMOTHERAPEUTICS IN THE TREATMENT OF TNBC USING TIME TO NEXT TREATMENT AS A SURROGATE FOR EFFICACY
10:15AM - 10:30AM
Feinberg B 1 , Kish J2 , Dokubo I3 , Wojtynek J3 , Lord K4 1 Cardinal Health Specialty Solutions, ATLANTA, GA, USA, 2 Cardinal Health Specialty Solutions, Dublin, OH, USA, 3 Athenex Inc., Schaumburg, IL, USA, 4 Cardinal Health Specialty Solutions, San Antonio, TX, USA
OBJECTIVES There is an extensive list of FDA approved and NCCN recommended single agent chemotherapies (SAC) for the treatment of metastatic triple negative breast cancer (mTNBC) with similar levels of evidence and disease activity based upon clinical trials with highly variable design. The objective of this study was to describe the real-world frequency of SAC by line of therapy (LOT) comparing their effectiveness by measuring time to next treatment (TNT). METHODS Female TNBC patients, identified by excluding all patients who received any HER2 targeted therapy or endocrine therapy, with evidence of continuous medical and/or pharmacy claims were identified in the Symphony Health Integrated Dataverse (SHIDV) database. Inclusion criteria required a minimum of one non-diagnostic claim for breast cancer and one distant metastasis (ICD-9/10 code) and having initiated at least one SAC from 01/01/2013 to 12/31/2017. The proportions of patients receiving each agent in first-line (1L), second-line (2L), and ≥ third-line (3L+) were calculated. TNT was measured using the Kaplan Meier method. RESULTS 1795 mTNBC patients met the study criteria; mean age 59.8 years, 58% commercially insured. The top 3 most frequently prescribed SAC by LOT: (1L) capecitabine (15%), nab paclitaxel (7%), carboplatin (6%); (2L) capecitabine (17%), paclitaxel (15%), eribulin (11%); (3L+) eribulin (17%), capecitabine (10%), gemcitabine (9%). TNT (months): (1L) capecitabine (9.7), carboplatin (6.4) and liposomal doxorubicin (6.4); (2L) capecitabine (8.5), vinorelbine (6.0), carboplatin (5.6); (3L+) carboplatin (6.1), capecitabine (6.0), liposomal doxorubicin (4.7). CONCLUSIONS Our research demonstrates significant variance between the frequencies of individual SAC use and their respective TNT. TNT is a surrogate for progression-free survival and is likely influenced by toxicity and patient choice. Preserved TNT in later LOT and the practice of SAC sequencing may result in no survival difference by respective sequence but research is warranted to test this hypothesis.
HTA’S NEXT TOP MODEL- MEDICINES WITH TUMOR-AGNOSTIC INDICATIONS
ISSUE
:
HTA Agencies and payers will face new challenging access questions regarding clinical trial design, evidence assessment and diagnostics with some of their first tumour-agnostic submissions. Precision oncology has given rise to the development of tumour-agnostic indications; indications defined by the genomic profile rather than histology of origin. The first indications will be for rare genomic alterations found across many disease sites. Are HTA frameworks equipped to handle the evolution in clinical design, comparative effectiveness estimation and testing? Because these genomic alterations are rare across many disease sites, HTA agencies will have to assess a clinical design of a non-comparative basket trial designs enrolling across all histologies. There are, therefore, many relevant standard of care therapies that are not mutually exclusive. A “comparator” of standards of care may require evidence synthesis from a number of published data or a synthesized virtual control arm from real world evidence, for example. How will comparative effectiveness be estimated and interpreted? The shift in histology-defined to genomically-defined cancer elevates the importance of testing. How should testing be incorporated within a drug assessment when testing infrastructure has impact beyond a single drug or indication? Given the challenges in feasible clinical design, comparative effectiveness estimation and testing facing the first HTA submissions for rare, tumour-agnostic indications, novel solutions will be required. OVERVIEW
:
The panel will open up by introducing the rationale for why we may consider that a drug can work across tumour types, tumour-agnostic clinical development programs and the evolution in testing. Panelists will debate how to make use of all the feasible data to be generated for HTA and payer decision making for appropriate access to tumour-agnostic medicines. Finally, panelists will debate on the appropriate access models for tumour-agnostic medicines.
Moderators
Clarissa Higuchi Zerbini, Bachelor
F. Hoffmann-La Roche Ltd, Basel, Switzerland
Panelists
Jacoline Bouvy, PhD
National Institute for Health and Care Excellence (NICE), London, United Kingdom
Rosa Giuliani, MD
The Clatterbridge Cancer Center, Liverpool, United Kingdom
Marc Van Den Bulcke, PhD
Sciensano, Ixelles, Belgium
WHICH IS THE WAY FORWARD FOR HEALTH TECHNOLOGY ASSESSMENT OF INTERVENTIONS WITH EXTERNAL BENEFITS? EXTENDED COST-EFFECTIVENESS ANALYSIS VS. COST-BENEFIT ANALYSIS
ISSUE: Health Technology Assessments increasingly need to evaluate interventions like vaccines that are accompanied, apart from benefits at the individual/patient level, by external socio-economic benefits. Such externalities are rarely included in “third-party payer perspective” economic evaluations – which risks distorting or underestimating the true value of interventions when such socio-economic benefits are not proportional to QALY benefits. Changes to the current HTA economic evaluation paradigm should be explored, that provide a holistic view of benefits from healthcare interventions and assess them in a fair manner.
OVERVIEW: The panel will debate two potential improvements to the typical HTA paradigm in order to include external benefits in assessment decisions. Following a 5-minute introduction by the Moderator regarding the inefficiencies of the current HTA setting in terms of including external benefits and the probable consequences for resource allocation decisions, each Panelist will advocate, for 15 minutes, in favour of a potential solution to this issue and, specifically, whether a Cost-Benefit Analysis or an Extended Cost-Effectiveness Analysis framework could better replace the third-party perspective CEA that is mostly used today for HTA decisions.
Panelists Perspective: Panelists will use vaccines as case study. JP Sevilla will highlight the spectrum of extended benefits that are associated with vaccines and argue that the optimal way to incorporate such benefits is to use a CBA framework, applicable to all healthcare interventions. Maarten Postma will argue that the current CEA paradigm is firmly rooted in HTA processes and that an evolution (rather than a revolution) is necessary - and this can be achieved through the application of an Extended CEA approach. 25 minutes of discussion will be available, for an issue that is of interest to the full spectrum of potential stakeholders due to the nature of its externalities.
Moderators
Kostas Athanasakis, MSc, PhD
Department of Public Health Policies, University of West Attica, Athens, Greece
Panelists
Maarten Postma, PhD
University of Groningen, Groningen, Netherlands
Joseph P Sevilla, Ph.D.
Data for Decisions, LLC, Waltham, MA, USA
NORDIC REGISTRIES IN SAFETY STUDIES AND OUTCOME RESEARCH- USING REAL WORLD EVIDENCE. ADVANTAGES AND CHALLENGES OF POOLING REGISTER DATA FROM DENMARK, SWEDEN, FINLAND AND NORWAY
PURPOSE: To discuss the opportunities and challenges of pooling data from Nordic registers for safety and outcome research drawing on experience with Nordic collaborators.
DESCRIPTION:
Authorities responsible for drug safety (including FDA and EMA) and pricing/reimbursement are increasingly requesting studies based on Real World Evidence (RWE). The workshop will show how the Nordic registers contain rich longitudinal patient data from cradle to grave across diseases, treatments, resource utilization and socio-economic status and facilitates monitoring and quality measurements at the patient level to support early intervention and improved management. The Nordic registries have similar data structure, validity, and potentials for longitudinal follow-up for epidemiological and health economic studies. Statistical power of rare exposures and interventions can be increased by pooling data from the Nordic health registers at the same time heterogeneity between countries can be assessed improving the interpretation of differences of effectiveness of treatment. Comparative forecasting (of disease populations from observed incidence and prevalence) as well as modelling of drug treatment-lines/pathways from prescription data is possible across the Nordic countries and may, combined with data from the abundant clinical quality registers including laboratory data, provide information otherwise difficult to obtain. Despite many similarities the use and pooling of health data from the Nordic countries require in-depth, local knowledge and time-consuming management of how to harmonize and combine data, considering local coding schemes and practices as well as variability in validity aspects. Collaboration across Nordic teams is essential for the process. The workshop is relevant for researchers, sponsors, decision makers and payers and will cover 1) Discussion of opportunities and challenges as outlined above based on 2) Examples of safety and outcome studies combining health registers (i.e. patient, cancer, diabetes, prescription), health-economic and socio-economic (DRG&DAG/National Statistics) and clinical data. 3) The pooling and harmonization of individual data across countries.
Discussion Leader
Kristian Bolin, PhD
University of Gothenburg, Gothenburg, Sweden
Annette Ersbøll, PhD
University of Southern Denmark, Copenhagen, Denmark
Anders Green, MD, PhD, Dr.Med.Sci
University of Southern Denmark, Copenhagen N, Denmark
A QUANTITATIVE BENEFIT-RISK ASSESSMENT BY APPLYING THE PRINCIPLES OF THE EUROPEAN MEDICINES AGENCY BENEFIT RISK ASSESSMENT FRAMEWORK AND THE PUBLICLY AVAILABLE, OPEN SOURCE TOOL ADDIS - A HANDS-ON WORKSHOP
PURPOSE: To familiarize attendees with the analytic steps for quantitative Benefit Risk Assessment by means of a real time, interactive modeling exercise, using the concept of the European Medicines Agency (EMA) Benefit Risk assessment framework and the publicly available, open source tool ADDIS.
DESCRIPTION: Quantitative methods for drug benefit-risk (BR) assessment (qBRA) are gaining interest from experts in regulatory agencies, academia, and industry due to their capability to integrate stakeholder preferences and treatment performance data for a transparent, reproducible analysis. The models account for multiple BR criteria simultaneously, generate composite scores, and provide visual outputs that are easy to interpret and communicate. A range of sensitivity analyses can be performed to assess the robustness of the conclusions in situations with varying degrees of uncertainties around data on both performance and preference. However, routine utilization of such methods in the field is still limited due to lack of familiarity with the practical aspects of conducting a qBRA and supporting software. The feasibility of using a more explicit approach in describing value-judgments in the current EMA BR assessment framework is currently being tested in different pilots by regulatory agency experts using a web-based analytical tool, ADDIS which allows to elicit BR trade-offs using various methods, including swing weighting and matching. The workshop will start with an overview of the underlying EMA BR assessment framework and the theoretical principles of qBRA. Then, a real-world case of a recent regulatory evaluation of will be introduced. The preferences will be fed into separate MCDA models and the ranking of the treatment options based on these preferences will be calculated in real time using ADDIS. The exercise will demonstrate and discuss potential discordance of qBRA conclusions based on different value judgements from different stakeholder groups, and how these differences can be made explicit and transparent using qBRA methods.
Discussion Leader
Quasi Ataher, MHS, PhD
Pfizer Inc, Philadelphia, PA, USA
Hans Hillege, MD, PhD, MSc
Dutch Medicines Evaluation Board, Utrecht, Netherlands
Douwe Postmus, PhD
University of Groningen, University Medical Center Groningen, Groningen, Netherlands
Tommi Tervonen, PhD
Evidera, London, United Kingdom
CONSIDERING THE APPLICABILITY OF GUIDANCE FOR EXPERT ELICITATION IN HEALTHCARE DECISION MAKING
PURPOSE
: To consider how recently developed guidance for structured expert elicitation in healthcare can be applied across decision-making contexts and discuss some of the challenges faced in using these methods.
DESCRIPTION
: Healthcare decision-makers often rely on input from experts to fill in gaps where there is little or no evidence. A quantitative process to capture experts’ beliefs is structured expert elicitation (SEE). Two of the panelists recently published guidance on how to conduct SEE in healthcare decision-making (HCDM). This focused on national-level HTA but acknowledged the complexities when using SEE outside of this setting, some of which may require modifications to the guidance approach. The guidance is intended to be used by HTA agencies, healthcare commissioners and healthcare funders, both national and local, and deviations from the suggested choices should be justified. There is a recognition, however, that some of the choices may depend on context and the complexities in using SEE in particular settings, for example in orphan drugs or in public health. The workshop will consider the potential benefits to HTA agencies of adopting the methodological guidance, such as greater transparency and consistency in methods. We will also discuss the barriers to adoption within HTA, such as pressures on time and resources.
Discussion Leader
Laura Bojke, PhD
University of York, York, United Kingdom
Karen Lee, MA
CADTH, Ottawa, ON, Canada
Rosie Lovett, PhD
National Institute for Health and Care Excellence (NICE), London, United Kingdom
Marta Soares, MSc, PhD
University of York, York, United Kingdom
11:00 - 12:30
WELCOME AND THIRD PLENARY
THIRD PLENARY SESSION- BIG HEALTHCARE DATA- ENDLESS OPPORTUNITIES FOR RESEARCH AND LEARNING
Healthcare data vary from discrete coded data elements to images of diagnostic tests to unstructured clinical notes. Such big data present a tremendous opportunity for the measurement and reporting of quality in healthcare. However, such high volume and high-variety information demand innovative forms of information processing to ensure enhanced insight and decision making. Data analytics applications on predictive modeling, population health, and quality measurement are all moving forward quickly. Predictive modeling can identify the high cost, high risk drivers and allows early intervention and patient management. Continuing quality measurement at the individual patient level can enhance personalized medicine and improve the overall effectiveness of treatment. Big data may also prove a useful tool for supplementing RCT data with real-world data in order to improve our evidence base. Finally, the richness of social media data may provide alternative evidence on life style patterns and mental health issues. The opportunities are vast and the challenges are great and new methods are needed to harness the potential for these data to have real impact. The aim of this plenary session is to inspire the audience with examples from the experts who are using these tools not only for research but driving learning at the healthcare system level.
Moderators
Dorte Gyrd-Hansen, PhD, MSc
University of Southern Denmark, Odense, Denmark
Dorte Gyrd-Hansen is Director of the Danish Centre for Health Economics, DaCHE, and Professor of Health Economics at Department of Public Health, University of Southern Denmark, and at University of Tromsø. Her research interests lie in the intersection of health economics and behavioral economics, with a particular focus on citizens’ preferences for health gains, and patients’ and health professionals’ behavior. She uses register data, survey data an conducts experiments in order to improve our understanding of policy relevant behavioral mechanisms. She is president-elect of the European Health Economics Association, and has previously held professorships at University of Queensland and Copenhagen Business School.
Speakers
Laura Hatfield, PhD
Harvard Medical School, Boston, MA, USA
Dr. Hatfield is an Associate Professor of Health Care Policy (Biostatistics) at Harvard Medical School's Department of Health Care Policy. Her methods research centers on trade-offs among multiple outcomes, with an emphasis on hierarchical Bayesian modeling, and causal inference with observational data, particularly difference-in-differences designs. She is faculty co-director of the Health Policy Data Science Lab and co-PI of the Methods Core of the Healthcare Markets and Regulation Lab. Recent honors include the 2018 ISPOR Health Economics and Outcomes Research - Methodology award for her paper on incorporating loss functions in safety surveillance decisions. Dr. Hatfield is the 2019 Chair-Elect of the Health Policy Statistics Section of the American Statistical Association and is serving a three-year term on ENAR's Regional Committee (RECOM). Dr. Hatfield earned her BS in genetics from Iowa State University and her MS and PhD in biostatistics from the University of Minnesota.
Jeremy Rassen, ScD
Aetion, Inc., New York, NY, USA
Dr. Jeremy A. Rassen, ScD, is co-founder and chief scientific officer at Aetion, Inc., a company that provides software to evaluate the effectiveness, safety, and value of medical treatments. At Aetion, Dr. Rassen leads the scientific effort around designing methodology for obtaining and communicating medical evidence from real-world data.
Dr. Rassen was formerly an assistant professor of Medicine at the Brigham and Women's Hospital and Harvard Medical School, where he focused on methodology for improved validity and reach of pharmacoepidemiology and comparative effectiveness research, including research into propensity score and instrumental variable methods. Before coming to the Brigham and Women’s Hospital, Dr. Rassen worked in Silicon Valley in numerous computer and software companies, including Hewlett-Packard and Epiphany, Inc. His focus was on high-performance software for the creation and analysis of large marketing databases.
Dr. Rassen received his Bachelor’s degree from Harvard College and his Doctorate degree in Epidemiology from the Harvard School of Public Health.
Michal Rosen-Zvi, PhD
IBM Research, The Hebrew University, Jerusalem, Israel
Michal is leading a worldwide team of researchers, expert in AI applied to health data and a local team who focuses on deep learning applied to medical imaging, machine learning and cuasual inference technologies applied to patients data. She joined IBM Research in 2005 and has since led various projects in the area of machine learning and healthcare; Therese are multidisciplinary projects where physicians, data scientists, AI experts and experts from Pharmaceutical companies join forces to analyze post launch patients data. Aside from teaching in various academic forums including courses at Tel-Aviv University and the Hebrew University, Michal has published more than 40 peer-reviewed papers, and serves as a Senior Program Committee member and reviewer at leading machine learning, health informatics, and bioinformatics conferences and journals. Nowadays she is also a visiting professor at the Faculty of Medicine at the Hebrew University. Michal holds a PhD in computational physics and completed her postdoctoral studies at UC Berkeley, UC Irvine, and the Hebrew University in the area of Machine Learning. She serves at various boards such as the committees such as the Israeli national digital health committee and the Impact Advisory Committee of the 8400 health network.
WELCOME FROM ISPOR PRESIDENT AND PRESENTATION OF ISPOR SERVICE AWARDS
Speaker
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.
ISPOR EUROPE 2020 ANNOUNCEMENT
Speaker
Nancy S. Berg
ISPOR, Lawrenceville, NJ, USA
Nancy S. Berg is chief executive officer (CEO) and executive director of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), the leading global scientific and educational organization for health economics and outcomes research. Ms. Berg has over 30 years of experience in health care and scientific/technical association leadership, and has been an entrepreneur and business consultant to both commercial and nonprofit organizations. At ISPOR, Ms. Berg is responsible for the Society’s global strategic direction and leadership of the association. She has led the organization, in concert with the Board of Directors, to design and implement a new strategic plan, mission, and vision to guide the Society into the future.
Previously, Ms. Berg led the International Society for Pharmaceutical Engineering (ISPE) as president and CEO. ISPE is the world's largest professional society fostering dialogue and education on technical, scientific, and regulatory best practices in the pharmaceutical and biotech industries. During her tenure with ISPE, Ms. Berg was the architect of the Society's Strategic Plan, focusing ISPE's energies and expertise on high-value initiatives related to drug shortages, quality metrics, global manufacturing quality, facility and supply chain issues, and patient experiences in clinical trials. Prior to her role at ISPE, Ms. Berg served as executive director and CEO of the Society of Manufacturing Engineers (SME). She also previously founded a strategic and business development consultancy company.
Ms. Berg has been engaged in education and business issues at national and international levels. She has served on many government initiatives involving business, community, development, growth, revitalization, and labor issues. She is a supporter of the Leukemia and Lymphoma Society’s Team in Training Program cycling events and a patron of many other charities. Ms. Berg is a graduate of the University of Michigan-Flint and is married to Timothy Jackson.
12:30 - 13:45
EXHIBITS & RESEARCH POSTER PRESENTATIONS VIEWING - SESSION 5
12:30 - 14:00
LUNCH IN THE POSTER & EXHIBIT HALL
12:45 - 13:45
POSTER AUTHOR DISCUSSION HOUR - SESSION 5
Attendees have the opportunity to interact with poster presenters during this hour-long session.
EDUCATIONAL SYMPOSIUM
VALUE BASED RENAL CARE IN EUROPE
Although other regions of the world are experiencing higher growth, chronic kidney disease (CKD) is an increasingly important issue in Europe. There is significant variation in CKD prevalence across Europe, and also in average healthcare costs per patient. CKD poses a risk to the future sustainability of health systems in Europe if improvements in shared decision-making and patient-centred care are not attained.
Our research programme will analyse current healthcare pathways, the role of patients in decision-making, barriers to better outcomes and innovative approaches in CKD. Attendees will be introduced to the preliminary findings, which will include comparisons across European countries in patient care, use of value principles and best practices in renal care. The final results will be used to highlight opportunities for better policies and initiatives around CKD in European health systems.
Sponsor
Baxter
Speaker
Rolf Dieter Bach, MD
KfH Kuratorium für Dialyse und Nierentransplantation e. V., Neu-Isenburg, Germany
Chrissy Bishop, Dr
The Economist Intelligence Unit, London, United Kingdom
Corinne Isnard Bagnis, PhD, MD
Hopital Pitié Salpetrière, Paris, France
Anna Marti Monros, RN, Master University Teacher
Consorcio Hospital General Universitario Valencia, Valencia, Spain
14:00 - 15:00
BREAKOUT SESSION 11
HEALTH TECHNOLOGY APPRAISAL METHODS AND PROCESS FOR ANTIBIOTICS- DEVELOPMENTS IN THE UK AND IMPLICATIONS FOR EUROPE
PURPOSE: To explain recent methods and process developments in the UK relating to the Health Technology Assessment (HTA) of new antibiotics and assess how these initiatives could be adapted for other settings in Europe.
DESCRIPTION: Incentivising the development of new antimicrobials is one of the major challenges in health policy globally given the risk of multi-drug resistance. Existing HTA processes have struggled to conduct appropriate evaluations of antibiotics due to the complex ways in which these products deliver value to treated patients and the wider population over the long term. Ensuring that the benefits of antibiotics are reflected in a value-based payment to drug manufacturers is essential to incentivise the development of new antibiotics that treat high-priority resistant infections. Recent work has shown how the long term benefits and costs of new antibiotics can be reflected in QALY-based systems, and linked to novel payment arrangements designed to maximise incentives for development and appropriate usage (e.g. payments ‘delinked’ from drug sales volumes). Based on this work the National Institute for Health and Care Excellence (NICE), NHS England and the Department of Health and Social Care (DHSC) have initiated a project to develop and test new models for the evaluation and purchasing of antimicrobials.
Beth Woods will discuss how the benefits and costs of antibiotics can be evaluated within a QALY-based system and how these analyses can inform delinked payments (12 minutes). Nick Crabb will provide an overview of the NICE, NHS England and DHSC project, including how the evaluation will inform the negotiation of payments based on the value to the NHS as opposed to volumes used (12 minutes). Angela Blake will provide a manufacturer’s perspective on the challenges of demonstrating the value of antibiotics and the current UK policy developments (12 minutes). Mark Sculpher will chair and lead the audience interactive element.
Discussion Leader
Angela Blake, MSc
Pfizer Ltd, Walton Oaks, United Kingdom
Nicholas Crabb, PhD
NICE, Manchester, United Kingdom
Mark Sculpher, PhD
University of York, York, NYK, 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 modelling 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 Modelling 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 the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) (2011-12).
Beth Woods, MSc
University of York, York, United Kingdom
QUALITATIVE RESEARCH FOR DISCRETE CHOICE EXPERIMENTS (DCE)- TOWARDS DEFINING GOLD STANDARDS
PURPOSE: The FDA has released a priority list of patient-preference-sensitive areas, and preference studies are of increasing interest to regulatory authorities and payers globally. Current guidelines document the importance of qualitative research for DCEs but give no further guidance on the standards, methods or different applications of qualitative research in this context. The objective of this workshop is to propose and discuss standards and encourage systematic reporting of qualitative methods in DCEs; identify best practice and further the application of gold standard methods for qualitative approaches in DCEs.
DESCRIPTION: We will present a recent literature review showing that a large proportion of published DCEs do not report details on the qualitative methods used. In those that do, a great heterogeneity in methods and applications is noted. Examples of qualitative approaches and their challenges for DCEs will be presented and discussed. Shared experience will be used to define key learnings. The applicability of elements of PRO development standards to methods used for DCEs will be presented, with specific examples and case studies from both PRO and DCE studies. Finally, a list of standards necessary for qualitative research in DCEs, based on common experience will be discussed and drawn up. This workshop will give participants the opportunity to gain insight into necessary standards for qualitative research for DCEs, and how best practice guidelines could support these efforts. Discussion leaders will present in 2 20-minute sessions, with 2 10-minute audience interactive sessions.
Discussion Leader
Samuel Aballea, PhD
Creativ-Ceutical, Rotterdam, Netherlands
Linda Abetz-Webb, MA
Patient-Centred Outcomes Assessments, Bollington, United Kingdom
Nicola Germain, MA
Creativ-Ceutical, Paris, France
EARLY HTA ADVICE- A REVIEW OF THE REQUIREMENTS AND OPPORTUNITIES TO ACHIEVE COLLABORATIVE AND EFFICIENT DIALOGUE
PURPOSE
: The objective of this workshop is to explore the early HTA process in key EU markets (UK, FRA, GER) and the rationale to seek early HTA advice from both the payer and manufacturer perspective.
DESCRIPTION
: Early HTA advice is an opportunity for manufacturers and HTA bodies to align on clinical development programs and ensure appropriate access for patients. Key aspects of healthcare system evolution increasing the value of early advice include growing HTA scrutiny on trial design and local data requirements, requests to launch with immature data due to difficulties with time required to show survival data, and divergence in trial endpoint acceptability between regulatory and payer bodies. Early dialogue discussions with HTA bodies allow for valuable input into early trial development at Phase 1b / 2. Additionally, early HTA advice provides important insights and feedback on the evidence package at Phase 3, thus, addressing key HTA needs to create an impactful submission. This workshop will outline the expectations HTA bodies have for manufacturers regarding early advice request submission and post-submission to set up for an impactful early dialogue discussion. The discussion will then turn to understanding the advantages of HTA early advice output, including the benefit to the HTA body; e.g., are final submissions more aligned with country-specific requirements and do they result in improved patient access? Discussion leaders will invite the audience to share their experiences and opinions on how to make early HTA advice effective for both manufacturers and payers, and how expectations may differ across EU markets.
Discussion Leader
Richard Grieve, PhD
London School of Hygiene and Tropical Medicine (LSHTM), London, United Kingdom
Richard Grieve is a professor of Health Economics Methodology at the London School of Hygiene and Tropical Medicine (LSHTM). He holds a senior research fellowship from the National Institute of Health Research (NIHR), which aims to improve analytical methods for decision-making.
Since 2002, Richard has led a research programme on the design and analysis of observational studies for establishing relative effectiveness and cost-effectiveness. Richard has provided scientific advice to NICE and has contributed to Decision Support Unit (DSU) Technical Support Documents on the appropriate use of observational data. Richard also advises the Department of Health in England on how to improve the use of evidence from observational studies for policy-making purposes. Richard has published more than 90 articles in peer-reviewed journals, and secured about £25 million in research grants from the research councils, NIHR, and the DoH.
Maximilian Hunt, BSc
CBPartners, New York, NY, USA
Cornelia Lechner, PharmD
Amgen, Munich, Germany
Tanja Podkonjak, MBA
Takeda UK Ltd, Wooburn Green, United Kingdom
VALUING HEALTH IN CHILDREN – WHERE ARE WE NOW, AND WHAT FURTHER WORK IS NEEDED?
ISSUE:
To facilitate the HTA of interventions for younger patients, utilities are needed that reflect the value of health in children and adolescents. However valuing health in children is challenging and raises questions about methodology, ethics and social values. For example: Which preference elicitation techniques should be used? Whose preferences should count (e.g. adults, parents, adolescents)? Under what perspective should those preferences be elicited (e.g. adults valuing health states for themselves or for a child)? Should we aim for consistency between child and adult health valuation, in terms of both methods and values? Recent research has provided insights on some of these issues, but uncertainties and disagreements remain about the best way forward.
OVERVIEW:
In this issue panel, Koonal Shah will moderate and summarise emerging international research that addresses some of the key challenges. Elly Stolk will describe the development of the first international protocol for valuing the EQ-5D-Y, the youth version of the EQ-5D, and its recent application in Japan. This protocol combines time trade-off (TTO) and discrete-choice experiments to elicit adult preferences, but differs from the protocol for valuing the adult EQ-5D-5L. Donna Rowen will explain her concerns about the use of TTO in this setting and will examine what alternative approaches are possible, focusing on how other measures such as the CHU9D have been valued. She will also highlight current research gaps. Rosie Lovett will provide the perspective of an HTA agency exploring whether to issue guidance to companies about measuring and valuing child health. She will focus on questions about consistency and outline what further work would help agencies like NICE to make methodological recommendations. Following the brief presentations, 30 minutes will be reserved for panel and audience discussion, with questions and suggestions on how to reach consensus on issues requiring scientific and social value judgements particularly welcomed.
Moderators
Koonal Shah, PhD
Office of Health Economics, London, United Kingdom
Panelists
Rosie Lovett, PhD
National Institute for Health and Care Excellence (NICE), London, United Kingdom
Donna Rowen, PhD
University of Sheffield, Sheffield, United Kingdom
Elly A. Stolk, PhD
EuroQol Research Foundation, Rotterdam, Netherlands
COST UTILITY ANALYSIS OR COST BENEFIT ANALYSIS FOR THE ECONOMIC EVALUATION OF NUTRITION INTERVENTIONS?
ISSUE: There is increasing recognition of the importance of nutrition interventions in contributing to nations’ health. However, these interventions are quite diverse, ranging from medical nutrition to broad public health programmes. There are challenges in the economic evaluations of public health interventions in general and nutrition programs in particular. First, causal effects may be difficult to ascertain because studies such as randomized controlled studies are often too costly, poorly generalizable, or simply unfeasible in these contexts. Nutrition is complicated by requiring multi-component, complex interventions that dynamically change with time and interact with the surrounding environment. Furthermore, the impact of these interventions may go beyond the direct health effects on the targeted individuals, to include broader consequences at the societal level. They may generate interlinked costs and consequences between different public sectors, the general public or the economy at large. Issues of consumer choice, preference, culture, community, habits and lifestyle have greater prominence than in pharmaceutical treatments. Lastly, equity considerations may be even more relevant, given their potential impact on the distribution of health across different population subgroups. There is a need to explore appropriate methods for economic evaluation in nutrition interventions.
OVERVIEW:
The panel will explore the respective merits of cost utility analysis versus cost-benefit analysis for measuring and valuing the key impacts in nutrition policies in different contexts. Each speaker will speak for 12 minutes, to leave 20 minutes for interactive audience questions, comment and debate. The session will be of particular interest to researchers and policy makers active in the fields of health technology assessment, economic evaluation, public health, nutrition and lifestyle.
Moderators
Carlo Federici, MSc
SDA Bocconi School of Management, MILANO, Italy
Panelists
Michael Drummond, MCom, DPhil
University of York, York, 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.
David Epstein, Phd
University of Granada, Granada, Spain
Karen Freyer, PhD, RDN
Maastricht University, Maastricht, Netherlands
REAL WORLD DATA & INFORMATION SYSTEMS
RW4: APPLICATION OF ARTIFICIAL INTELLIGENCE AND MEDICAL BIG-DATA IN REAL WORLD EVIDENCE GENERATION- A LITERATURE REVIEW OF RESEARCH PRACTICE IN CHINA
2:45PM - 3:00PM
Xiong T , Li J, Hao Y, Xu J Tianjin Happy Life Technology Co. Ltd., Beijing, China
OBJECTIVES : Combination of artificial intelligence (AI) and medical big-data is considered as one of the most valuable technique in the 21st century, which was expected to be assisting in medical innovation of healthcare. To apply AI and patient level data in clinical research, a traceable and regulated Data Process & Application Platform (DPAP) was developed. In this study, all published peer reviewed scientific papers based on the AI technology and real-world data (RWD) derived from DPAP were scrutinized, to examine the application of AI and RWD in real-world evidence generation in China. METHODS : Thirty-eight papers were retrieved by searching literature databases with inclusion criteria of usage of AI technology, RWD and DPAP. All the papers were published in English. Study types and related disease areas were categorized and analyzed. RESULTS : Among the 38 papers, 24 were medical research, 14 belong to computer science covered topics in natural language processing (NLP) and data standardization, etc. Publications were all indexed by SCI or SCIE, and the impact factors ranged from 1.80 to 36.42. All papers were published in the past 3 years, indicating the RWD and AI technology have been increasingly contributed to improved health outcomes. The clinical research focused on cancers (15 papers), pneumonia (3 papers), diabetes (2 papers), depression, hand, food and mouth disease, and Kawasaki disease. There were 4 retrospective cohort studies and 20 cross-sectional studies. Eight papers reported multi-center clinical studies. CONCLUSIONS : The big-data and intelligence database platform accelerate and simplified the process of data extraction, analyses, and management. It could rapidly transform clinical data into research conclusions through proper compliance procedures, which has been well recognized by the pharmaceutical industry, reflecting the demand of demonstrating the medical product values in real world settings in China.
RW3: HUMAN PAPILLOMA VIRUS IN ITALY- RETROSPECTIVE COHORT ANALYSIS AND VACCINATION EFFECT FROM REAL-WORLD DATA.
2:30PM - 2:45PM
Marcellusi A 1 , Fabiano G2 , Sciattella P1 , Favato G2 , Mennini FS3 1 Faculty of Economics, Centre for Economic and International Studies (CEIS)-Economic Evaluation and HTA (EEHTA), University of Rome Tor Vergata, Rome, Italy, 2 Institute for Leadership and Management in Health, Kingston University, London, SRY, UK, 3 Faculty of Economics, Centre for Economic and International Studies (CEIS)-Economic Evaluation and HTA (EEHTA), University of Rome Tor Vergata, Rome, RM, Italy
OBJECTIVES : The objective of this study is to estimate the lifetime risk of hospitalization associated with Human Papilloma Virus (HPV)-related disease in Italy. METHODS : A retrospective,non-randomized,observational study was developed based on patients hospitalized between 2008 and 2016 in Italy. All hospitalisations were identified through administrative archives, according to the International Classification of Diseases (ICD-9 CM). Information related to the hospital discharges of all accredited public and private hospitals, both for ordinary and daycare regimes, was taken into account. We included hospitalizations related to resident patients presenting one of the ICD9-CM codes as primary or secondary diagnosis: Genital warts (GW): ‘Condyloma acuminatum’ (078.11);‘Anal cancers’(AC)(154.2–154.8);Oropharyngeal cancers(OC): ‘Oropharyngeal cancer’(146.0–146.9) and ‘Head, face and neck cancers’(171.0);Genital cancers(GC):‘Penis cancer’(187.1–187.9) and ‘Cervical cancer’(180.0–180.9).Data was stratified by birth years and divided into two groups:a) cohort born before 1996(Not vaccinable) and b)cohort born after 1997 (Vaccinable–first cohort that could be vaccinated at the beginning of immunization schedule in girls since 2008 in Italy). Hospitalisation risks for both groups were estimated by year and age. RESULTS : Epidemiological data demonstrate that the peak of hospitalization risk was occurring at: 24-26 years of age for GW (both male and female), 33 – 41 and 47 – 54 years for AC male and female respectively, 53 – 59 and 52 – 58 years for OC male and female respectively and 54 – 60 and 39 – 46 years for GC male and female respectively. Focusing on GW and GC, Vaccinable female demonstrate a significant reduction on hospitalization risks (-54% on average) compared to Not vaccinable female until the 20 years of age (maximum follow-up available for girls born after 1997). Comparing the same birth cohort of male, no differences in the hospitalization risk were found. CONCLUSIONS : This study represents the first analysis that estimates the effects of anti-HPV vaccination strategies in Italy based on real-world data.
RW2: METHODOLOGICAL APPROACHES FOR INCORPORATING REAL-WORLD DATA (RWD) FOR OVERALL SURVIVAL (OS) INTO LONG-TERM SURVIVAL ESTIMATES- A CASE-STUDY NICE TECHNOLOGY APPRAISAL IN EXTENSIVE-STAGE SMALL-CELL LUNG CANCER (ES-SCLC).
2:15PM - 2:30PM
Connor S 1 , Orfanos P2 , Flahavan E3 , Patterson K4 , Lee D5 1 Roche Products Ltd, Herts, UK, 2 F. Hoffmann La Roche, Basel, Switzerland, 3 F. Hoffmann La Roche, Welwyn Garden City, HRT, UK, 4 BresMed, Sheffield, UK, 5 BresMed Health Solutions Ltd., Sheffield, DBY, UK
OBJECTIVES Increase plausibility of long-term survival estimates for ES-SCLC patients via incorporation of RWD. METHODS The IMpower133 (NCT02763579) trial follow-up was insufficient to describe the proportion of ES-SCLC patients expected to survive long-term, when treated with platinum-etoposide. To improve estimates of long-term survival for ES-SCLC patients, we identified a cohort from the US Flatiron Health electronic health record-derived database. In line with the trial, patients with 0-1 ECOG performance status, and platinum-etoposide treatment in first-line ES-SCLC were selected (N=860) and survival curves generated. Different methodologies were applied to incorporate RWD into the CEM projections. Firstly, RWD could be incorporated from different time points, e.g. at time zero or when 90% of trial observed events occurred. Secondly, either the RWD hazard of death was applied alone, or in combination with the clinical trial hazard of death, assuming a fixed shape over time. These combined into 4 different approaches for estimating long-term survival, with standard parametric extrapolations applied. The intervention arm assumed a constant effect beyond the trial, with alternative assumptions tested in scenarios. RESULTS With the log-logistic extrapolation in the base case, incorporating RWD into the survival model along with the trial data lowered the total life years gained, compared to applying trial data alone: 1.21 versus 1.00-1.11 incorporating the RWD. These lower, life years estimates with RWD were considered by clinical experts to more accurately reflect long-term survival in the control arm and gave improved cost-effectiveness estimates. CONCLUSIONS Incorporating real-world OS data can improve the clinical plausibility of long-term survival estimates, impacting the incremental difference estimated between therapies, and therefore ICER calculations. Further research is needed into the most appropriate methods to use in different circumstances. The methodology described here is one of the first approaches to formally incorporate RWD into a NICE appraisal.
RW1: HOSPITAL COSTS AND OUTCOMES OF UNICOMPARTEMENTAL COMPARED TO TOTAL KNEE REPLACEMENT FOR PATIENTS WITH MULTIPLE COMORBIDITIES- A POPULATION-BASED STUDY
2:00PM - 2:15PM
Kolovos S , Strauss VY, Prats-Uribe A, Prieto-Alhambra D, Pinedo-Villanueva R University of Oxford, Oxford, UK
OBJECTIVES: A randomised clinical trial (TOPKAT) recently examined the cost-utility of unicompartemental (UKR) compared to total knee replacement (TKR). However, TOPKAT excluded patients with multiple comorbidities (ASA≥3). Various analytical methods minimising confounding were examined to replicate trial results using observational data, with inverse probability weighting (IPW) and propensity score stratification (PSS) accurately reproducing trial results. The objective of the current study was to estimate the hospital costs and outcomes (revision rates and utility estimates) of UKR compared to TKR for complex patients with comorbidities ineligible for the TOPKAT trial in order to assess their impact and ultimately estimate the cost-effectiveness of UKR compared to TKR for this population. METHODS: We used data from the National Joint Registry linked to hospital records from the English National Health Service including quality of life (EQ-5D-3L) from the national PROMs database. Patients who underwent UKR or TKR between 2009 and 2016, with an ASA≥3 and completed EQ-5D-3L questionnaires were included. Costs for primary/revision surgery and complications were estimated based on national reference costs before applying IPW or PSS. Estimates will subsequently be adjusted using these methods. RESULTS: A total of 21,160 patients were included, 146 (0.6%) UKR and 24,014 (99.4%) TKR. Four (2.7%) UKR patients and 339 (1.4%) TKR patients had a revision surgery. Preliminary analysis showed that the mean post-operative utility estimate was 0.71 (SD= 0.29) for UKR and 0.67 (SD= 0.28) for TKR. The mean healthcare costs were £6,416 (SD= 1,274) for UKR and £6,803 (SD= 2,023) for TKR. CONCLUSIONS: A large number of patients undergoing UKR or TKR with comorbidities are not eligible for clinical trials but estimates of cost-effectiveness can be produced by applying analytical methods previously validated in population-level data. Unadjusted findings suggest UKR may lead to higher utility scores, higher revision rates, and lower costs than TKR for multimorbid patients.
TREATMENT PATTERNS & GUIDELINES STUDIES
TP4: USE OF EPISODIC THERAPY IN MODERATE/SEVERE HEMOPHILIA A PATIENTS ACROSS THE EU5
2:45PM - 3:00PM
Kyle A1 , Brown S 2 , Yaacob NB3 , Rosli A4 , Eryse S3 1 Ipsos Healthcare, London, LON, UK, 2 Ipsos Healthcare, London, UK, 3 Ipsos, Kuala Lumpur, Malaysia, 4 Ipsos, London, UK
OBJECTIVES : Despite prophylaxis therapy being a preferred treatment approach for moderate/severe Hemophilia A, some patients remain on episodic treatment only. This paper examines the usage of episodic treatment across the EU5 and explores the reasons why these patients are not receiving prophylaxis therapy. METHODS : Results are from the Q1 2019 Ipsos Healthcare Haemophilia Therapy Monitor, an online study where physicians provided data on their Hemophilia A/B patients. This paper focuses on moderate/severe Hemophilia A patients in France, Germany, Italy, Spain and UK aged 13–90 who had been on their current treatment for >6 months and received no previous treatment (EU5: n=542; France: n=114; Germany: n=99; Italy: n=134; Spain: n=100; UK: n=95). RESULTS : Differences were observed in the percentage of patients receiving episodic vs prophylaxis treatment for their moderate/severe Hemophilia A across the EU5 markets. Germany had the highest percentage of patients receiving episodic treatment only (47%), with 40% of patients in France also exclusively receiving episodic therapy. In contrast, only 15% of patients in the UK were currently prescribed episodic treatment only, below the EU5 average of 35%. Across all countries, the main reason why episodic patients were not prescribed prophylaxis therapy was that the patient’s disease severity did not require it (74% of EU5 episodic patients). ‘Patient/caregiver request/preference’ was found to be the second most common reason (19% of EU5 episodic patients). Reasons such as ‘inconvenience of prophylaxis dosing schedule’ or ‘expense of prophylaxis therapy’ were less prevalent, but this varied between countries, with 35% of patients in Spain not receiving prophylaxis treatment due to the dosing schedule vs 7% of patients in France. CONCLUSIONS : This study highlights country differences in the percentage of moderate/severe Hemophilia A patients who are receiving episodic therapy, along with differences in the reasons why these patients are not receiving prophylaxis treatment.
TP3: CHARACTERIZING AND COMPARING US COMMERCIALLY-INSURED PATIENTS WITH OPIOID USE DISORDER (OUD) BY RECEIPT OF MEDICATION-ASSISTED TREATMENT (MAT)
2:30PM - 2:45PM
Perry A 1 , Manjelievskaia J2 1 IBM Watson Health, Boston, MA, USA, 2 IBM Watson Health, Cambridge, MA, USA
OBJECTIVES : Less than half of patients with opioid use disorder (OUD) are treated with medication-assisted treatments (MATs): Buprenorphine, Naltrexone, Methadone. Barriers to MAT are not well understood. This study characterizes and compares OUD patients based on receipt of MAT.
METHODS : In IBM MarketScan® Commercial Claims and Encounters Database, patients were selected with initial OUD diagnosis between January 1, 2012 and December 31, 2017, ages 12-64, and enrolled for 12 months prior to, and 6 months following, diagnosis. Demographic and clinical characteristics were described and compared between MAT-treated versus MAT-naïve patients.
RESULTS : This study included 96,380 OUD patients (mean age: 38.29, 55% male; 31% child/other of insured employee; 42% South region). Overall, 39% received MAT, with 30%, 8%, and 1% receiving Buprenorphine, Naltrexone and Methadone, respectively. Compared to MAT-naïve patients, MAT-treated patients were, on average, 7.3 years younger (95% CI 7.1, 7.4), and a majority were male (63%). MAT-treated patients had higher rates of ADHD (10% difference; 95% CI 9.6, 10.4) and lower rates of chronic pain (21% difference; 95% CI 20.4, 21.6). Most OUD patients were urban residents (>85% in both groups), with a higher proportion of MAT-naïve patients in the South (7% difference; 95% CI 6.4, 7.6). A higher proportion of MAT-treated patients had mental health/substance abuse coverage (33% difference; 95% CI 32.5, 33.5). Within six months following OUD diagnosis, MAT-treated patients had higher rates of psychotherapy (11% difference; 95% CI 10.4, 11.6) and lower rates of emergency department visits (9% difference; 95% CI 8.4, 9.6).
CONCLUSIONS : This study provides real-world evidence highlighting demographic and clinical differences in OUD patients by MAT receipt. Receipt of MAT may be associated with higher engagement in OUD- and mental-health-related services and decreased healthcare utilization. Further research focusing on barriers to receipt of MAT and other OUD-related services is warranted.
TP1: TREATMENT PATTERNS IN RELAPSED/REFRACTORY MULTIPLE MYELOMA PATIENTS IN GERMANY
2:00PM - 2:15PM
Patel V1 , Kap EJ2 , Kutikova L 3 , Lebioda A4 , Schoehl M4 , Kostev K5 1 Amgen (Europe) GmbH, London, UK, 2 IQVIA, Frankfurt am Main, Germany, 3 Amgen (Europe) GmbH, Rotkreuz, ZG, Switzerland, 4 Amgen GmbH, Munich, Germany, 5 IQVIA Commercial GmbH & Co. OHG, Frankfurt am Main, Germany
OBJECTIVES To describe real-world use of carfilzomib (K), bortezomib (V), lenalidomide (R), pomalidomide (P), ixazomib (I), daratumumab (D) and elotuzumab (E) in relapsed/refractory (RR) multiple myeloma (MM) patients in Germany. METHODS This retrospective analysis was based on 2015 to February 2019 LRx database, which includes nationwide data on 80% of prescriptions (full product information and prescription data) reimbursed by statutory health insurance funds in Germany. We analyzed demographic and treatment characteristics by regimen and line (2 and 3L+) in mutually exclusive categories. A validated algorithm was used to determine treatment regimens and end of treatment line (Palmaro 2017). RESULTS This analysis included 4,533 RRMM patients, with most recent regimen in 2L 3,537 and 996 in 3L+. 2L patients were treated with V 31% (triplet 22%, non-triplet 9%), R non-triplet 21% and K 19% (non-triplet 14%, triplet 5%), D 16% (non-triplets 8%, triplets 8%), P 6%, E and I 3-4% each. Where known, previous treatment included V (62%), R non-triplet (28%) or regimens based on melphalan or cytostatic drugs (10%). 3L+ patients were treated with D 35% (non-triplet 17%, triplet 18%), K 22% (non-triplet 12%, K triplet 10%), V, P and R regimens 8-12%. Previous treatment included V (24%), D (17%), R (16%), K (25%), P, E, and I (2-8%). In 2L, mean age ranged from 65-74 years. Older patients and those with higher estimated Charlson comorbidity index (CCI > 1), more often received regimens containing V and/or R and non-triplet regimens In 3+L, more patients with higher estimated CCIs (>1) more often received treatment containing D, K, I, E or P. CONCLUSIONS This real-world database analysis shows that majority of patients in 2L are treated with older R or V-based regimens. This reflects lower use of more potent novel and combination therapies than expected. Trends by year will be investigated.
TP2: TREATMENT PATTERNS IN SPINAL MUSCULAR ATROPHY- INTERVIEWS WITH HEALTHCARE PROFESSIONALS IN THE UNITED KINGDOM
2:15PM - 2:30PM
Dabbous O 1 , Germain N2 , Maru B1 , Aballéa S3 , Desroches M2 , Toumi M2 , Arjunji R1 1 AveXis, Inc., Bannockburn, IL, USA, 2 Creativ-Ceutical, Paris, France, 3 Creativ-Ceutical, Rotterdam, Netherlands
OBJECTIVES: Spinal muscular atrophy (SMA) is a rare genetic disorder characterized by weakness and atrophy in skeletal muscles. Excluding the prenatal onset form (type 0), SMA is classified into 4 phenotypes based on severity, with type 1 being the most severe. Emerging treatments are shifting the treatment paradigm, but little is known about current treatment patterns. The objective is to collect data on treatment patterns in SMA types 1, 2 and 3 in the UK through health care professional (HCP) interviews. METHODS: HCPs, who had seen at least 2 patients with SMA in the past 12 months, were recruited through contacts in clinical centers in the UK. Informed consent was obtained from all respondents with subsequent telephone interviews. RESULTS: Interviews were conducted with 16 HCPs from 8 UK regions: nine neurologists, three pulmonologists, two physiotherapists, one nurse, and one health visitor. HCPs reported that paths to diagnosis depend on symptom timing, and HCPs involved. Time to diagnosis was estimated at up to 3 months for SMA 1, and up to 12 months for SMA 2 and 3. HCPs agreed on key approaches to management and care coordination. Patients’ geographic proximity to hospital often determines who coordinates care. The existence and characteristics of phases in SMA was divisive, although focus was on ventilation, nutrition, and mobility. Many HCPs are involved in patient care, with community support being essential, however, HCPs were not able to report on palliative care and personal social services. The challenges faced by HCPs in SMA are numerous and include lack of resources and lack of effective treatment. CONCLUSIONS: HCP reports underline the complexity of treatment coordination for SMA, between hospitals and communities. Management is focused on ventilation, feeding and mobility, with involvement of a large number of HCPs. A comprehensive SMA care model is needed.