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
