Sat May 18
8:00 AM - 12:00 PM
SHORT COURSE MORNING SESSION
Introduction to the Design & Database Analysis of Observational Studies of Treatment Effects Using Retrospective Data Sources
Level: Introductory
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
Benjamin M. Craig, PhD
Moffitt Cancer Center, Tampa, FL, USA
Benjamin M. Craig, PhD, is an Associate Professor of Economics at the University of South Florida. He received his MS in Economics at the University of Texas at Austin in 1999 and his PhD in Population Health from the University of Wisconsin in 2003. Trained as an econometrician, his research focuses on health preference research and the economics of cancer prevention, detection, and control. He is an active member of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), the International Society for Quality of Life Research (ISOQOL), the International Academy of Health Preference Research (IAHPR), and the EuroQol Group.
Bradley Martin, PharmD, PhD, RPh
University of Arkansas for Medical Sciences College of Pharmacy, 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
Level: Introductory
Track: Patient-Centered Research
Patient-reported outcome (PRO) assessments are increasingly being used to measure/assess patients’ experiences (e.g., treatment benefit, tolerability, preferences, perspectives of benefit and risks of treatment) in research. Conceptual, methodological, and practical methods for determining what concepts to measure and how to measure them will be presented. Theoretical frameworks, reliability, validity, responsiveness, methods of administration, respondent and administrative burdens, and issues of analysis and interpretation of PRO data will be discussed, using real-world examples. Key considerations related to the selection, modification, and/or development of PRO instruments, will also be discussed. This course is intended for those with little experience with these methodologies.
Faculty Member
Alexandra I. Barsdorf, PhD
Clinical Outcomes Solutions, Chicago, IL, USA
Alex recently joined Clinical Outcomes Solutions (COS) as Director of Clinical Outcome Assessments (COA). Alex has 10 years pharmaceutical industry outcomes research experience at Pfizer Inc. with a focus on clinical outcome assessments (COAs) and health economics outcomes research (HEOR). She worked for five years in the PRO Center at Pfizer where she was responsible for the selection, development, validation, implementation, analysis, and interpretation of COAs across the product lifecycle across various therapeutic areas including urology, pain, diabetes, oncology, and women’s health. In her HEOR role, she was responsible for the strategic development and delivery of a portfolio of economic and quality of life evidence in support of regulatory submissions and commercialization mainly within rare diseases, e.g., Duchenne muscular dystrophy and sickle cell disease. Alex has a special interest in electronic clinical outcome assessments (eCOA) and has served as Co-Chair of the Critical Path Institute’s (C-Path) ePRO subcommittee.
Prior to joining Pfizer, Alex worked for over 13 years conducting industry and National Institutes of Health (NIH) sponsored clinical research at several academic medical institutions in New York City including New York Presbyterian Hospital-Weill Medical College of Cornell University, Mount Sinai School of Medicine-Brookdale Department of Geriatrics and Adult Development, and Columbia University Medical. In these positions she gained extensive experience in clinical interviewing, neuropsychological testing, and the design, administration, scoring, and interpretation of clinical, neuropsychological, and patient-reported outcomes with a variety of subject populations in various settings and across several disease areas. She has worked with children, adults, and elderly patients as well as physicians and nurses. She worked in inpatient, outpatient, and nursing home settings and has experience in the areas of psychiatry, neurology, and rheumatology. She also served as Associate Professor at Fordham College of Liberal Studies.
Alex holds a doctorate in Developmental Psychology from Fordham University.
Elizabeth (Nicki) Bush, MHS
Eli Lilly and Company, Indianapolis, IN, USA
Elizabeth (Nicki) Bush is the Director and Global Head of the Patient-Focused Outcomes Center of Expertise at Eli Lilly and Company, responsible for the design and implementation of patient-centered measurement strategies to support drug development efforts across Lilly’s portfolio. In this capacity, she and her team advise drug development teams in the selection, modification and development of clinical outcome assessments (COAs) for use in clinical trials and other research studies, and utilize mixed methods approaches to evaluate the measurement properties of COAs and aide in interpretation of the resultant data. Nicki has held research and leadership positions in the patient-centered measurement field in both consultancy and industry for over ten years. During this time, she has collaborated with partners in industry, regulatory agencies, and academia and held the position of Industry Co-Director of the PRO Consortium from 2016-2018. Her previous experience includes behavioral research and program evaluation. Ms. Bush earned her BA in the liberal arts (philosophy and history of mathematics and science) from St. John’s College and her master’s degree in epidemiology from the Johns Hopkins Bloomberg School of Public Health.
Tara Symonds, PhD
Clinical Outcomes Solutions, Folkestone, Kent, United Kingdom
Tara Symonds is the Clinical Outcomes Assessment (COA) Strategic Lead & Chief Science Officer at Clinical Outcomes Solutions providing advice on COA strategy from development to dissemination. Tara has more than 20 years of experience in the development and implementation of COAs for use in clinical research. Her areas of interest and expertise are in qualitative research, ePRO/BYOD (Bring Your Own Device) and application of PRO measures in comparative effectiveness research by incorporation into electronic medical records and registries. Many of the questionnaires and screening tools she has worked on can be accessed at: www.pfizerpatientreportedoutcomes.com. She has worked across numerous therapeutic areas and has extensive expertise in Sexual Health, Women’s Health, Pain, Respiratory, and Rare Diseases. She worked in industry for 16 years, her final assignment was to set-up and lead the PRO Center of Excellence at Pfizer. Tara has much experience in negotiating COA labelling at both FDA and EMA and in formulating best positioning of COA data for maximising reimbursement with EU and US payers. Tara has also previously worked in academia teaching undergraduate and postgraduate psychology. She has published more than 60 peer reviewed articles and is co-author of the following book: Cappelleri et al (2014) Patient Reported Outcomes: Measurement, Implementation and Interpretation, CRC Press. Tara is a Chartered Psychologist and holds a Doctorate in Health Psychology from the University of Huddersfield, UK.
Introduction to Modeling Methods
Level: Introductory
Track: Methodological & Statistical Research
Decision analysis is a tool that uses an explicit, quantitative structure to describe and analyze complex health care decisions. This course will provide an introduction to the principles and practice of decision analysis. Upon completion of the course, participants will be able to evaluate the appropriateness of decision analysis in different settings, construct simple decision trees, understand the basic mechanics of tree evaluation and sensitivity analysis, and acquire skill in the interpretation of a published decision analysis. Extension of basic techniques, such as cost-effectiveness analysis and the assessment of patient preferences, will be briefly discussed. Class exercises will be used to illustrate these principles. This course is suitable for those with little experience with decision analysis.
Faculty Member
Mark S. Roberts, MD, MPP
University of Pittsburgh, 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.
Elements of Pharmaceutical/Biotech Pricing 1 - Introduction
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 pharmacoeconomics, 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 covers 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.
Utility Measures
Level: Introductory
Track: Patient-Centered Research
This course is designed to provide an introduction and overview of utility measures to support economic evaluations. The concepts of health-related quality of life and utility will be introduced and discussed in terms of their differences and similarities. Faculty will describe how these data can be combined with survival to estimate quality-adjusted life years. Some issues for debate will be introduced. In the second section, course participants will explore the methods that are used to capture utilities such as standard gamble, time trade off, and rating scales. Building on this will be a presentation of the different generic instruments that have been developed for measuring quality of life such as the EQ-5D, Health Utilities Index, and SF-36. Estimating utilities from a condition-specific measure will also be discussed. In the third section we will describe approaches that can be used when utility data from trials are not available. The development of mapping functions and other crosswalks will be described from disease-specific measures to generic HRQL measures. The pros and cons of the different main approaches will be discussed. Other approaches to addressing a lack of utility data will also be described including prospective observational studies, systematic reviews, critical appraisal of published values, and the valuation of vignette type descriptions of health. In the final section we will describe the requirements and preferences of different reimbursement agencies around the world including UK/Australia/Canada; US agencies; other EU markets such as Sweden/Belgium/Netherlands/Germany; Asia; and Latin America. The course will be interactive with break-out sessions and group discussion. No prior knowledge of utilities or health-related quality of life is assumed.
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/
Brendan Mulhern, MRes
Univeristy of Technology Sydney, Sydney, Australia
Brendan Mulhern (MRes) is a Senior Research Fellow in Health Economics and Outcomes Measurement at the Centre for Health Economics Research and Evaluation (CHERE), University of Technology Sydney, Australia. He previously worked at the School of Health and Related Research at the University of Sheffield, UK. His research interests include the development of both generic and condition specific preference based measures for use in economic evaluation. Example studies include the development of the EQ-5D-5L value set for England/UK, and (along with Prof. Brazier) a large international study developing version 2 of the SF-6D (SF-6Dv2).
He is also involved in research developing and testing of new and innovative methods for valuing health. He is also interested in developing non preference based measures of health and quality of life, and testing and comparing the psychometric performance of existing instruments using Item Response Theory methods.
He is a member of the EuroQol Group, the International Society for Quality of Life Research (ISOQOL), the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), the International Academy for Health Preference Research (IAHPR) and the Australian Health Economics Society (AHES). He is a member of the ISPOR Australia Committee.
New! 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.
Carrie Savage Bennette, MPH, PhD
University of Washington, Seattle, WA, USA
Carrie Bennette is a principal quantitative scientist at Flatiron Health and an affiliate assistant professor at the University of Washington. She holds a PhD in health economics and outcomes research from the University of Washington, an MPH from Columbia University and has over 100 peer-reviewed publications in health economics and outcomes research. Carrie's current research focuses on developing and applying statistical methods that leverage real-world data to accelerate clinical research in oncology. At Flatiron Health, Carrie is focused on building and validating external control arms using real-world data to support regulatory and coverage decisions.
Joshua Kraut, MS
Flatiron Health, Seattle, USA
Josh Kraut is a Senior Data Insights Engineer at Flatiron Health. He spent the past two years building analytic software to improve the delivery of care at academic medical centers and is currently focused on building tools to improve the research process for scientists using real-world data. Previously, Josh worked at Google and Palantir Technologies and studied applied statistics at Columbia University.
8:00 AM - 5:00 PM
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 economics 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. This course is suitable for those with little or no experience with health economics.
Faculty Member
Lorne Basskin, PharmD
School of Pharmacy, 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.
Bayesian Analysis – Overview and Applications
Level: Introductory
Track: Methodological & Statistical Research
The first portion of this course is designed to provide an overview of the Bayesian approach and its applications to health economics and outcomes research. The course will cover basic elements of Bayesian statistics, contrasting briefly with classical (frequentist) statistics, and introduce available statistical packages. The second part of the course is a "hands-on" workshop where participants will be led through live examples using JAGS and R. Attendees will have the chance to apply the principles they have learned in the morning session to challenging data analysis problems, including the use of Bayesian generalized linear models (GLM) to analyze cost and outcomes data. This course is designed for those with a limited understanding of Bayesian statistical concepts or for those who want a refresher and more practical experience. Participants who wish to gain hands-on experience are required to bring their laptops with Jags and R installed.
Faculty Member
Christopher S. Hollenbeak, PhD
The Pennsylvania State University, Hershey, PA, USA
Christopher S. Hollenbeak, PhD is Professor and Head of the Department of Health Policy and Administration at the Pennsylvania State University. Dr. Hollenbeak has broad interests in health economics and health outcomes research, with a particular focus on economic issues in surgery. In previous research he has studied modifications to the kidney allocation mechanism, estimated the cost-effectiveness of screening for head and neck cancer, and estimated the burden of surgical site infections after major abdominal and cardiothoracic surgical procedures. Dr. Hollenbeak received his doctorate in Economics from Washington University in St. Louis, where he studied Bayesian econometrics under Ed Greenberg and Siddhartha Chib. He uses Bayesian methods in meta-analysis and to fit nonlinear risk adjustment models for continuous hospital performance measures.
David Vanness, PhD
The Pennsylvania State University, Hershey, PA, USA
1:00 PM - 5:00 PM
SHORT COURSE AFTERNOON SESSION
New! Health State Utility (HSU) Recommendations for Identification and Use of Hsu Data in Cost-Effectiveness Modelling
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. This short course will be presented by Drs. John Brazier, Jon Karnon and Andrew Lloyd and is timed to support the dissemination of the TF report and requires some knowledge of utilities. 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.
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/
Jonathan Karnon, PhD
Flinders University, Adelaide, Australia
Jonathan is professor of health economics at Flinders University, South Australia. He has been undertaking applied economic evaluations of health care technologies and services for over 20 years. He has particular expertise in the use of cost-effectiveness models and was a member of the most recent ISPOR taskforce on good modelling practice, chairing the discrete event simulation working group. In Australia, he has sat on the Economic Sub-Committee of the Pharmaceuticals Benefits Advisory Committee since 2009.
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.
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 A. Rassen, ScD
Aetion, Inc., New York, 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).
Case Studies in Pharmaceutical / Biotech Pricing 2 – Advanced
Level: Intermediate
Track: Health Policy & Regulatory
Case studies will be employed to lead participants through the key steps of new product pricing, with focus on the need to thoroughly analyze the business environment and its constraints and opportunities, and the need to closely integrate the pricing, reimbursement, and pharmacoeconomic strategy for the new product with the clinical development and marketing strategies. Practical exercises will allow participants to consolidate the concepts delivered in the “Elements” introductory session and expanded here. Areas covered will include the post-launch issues of reimbursement and pricing maintenance as a part of life-cycle management in a global environment. This course is designed for those with limited experience in the area of pharmaceutical pricing and covers 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.
Meta-Analysis and Systematic Reviews in Comparative Effectiveness Research
Level: Introductory
Track: Study Approaches
Comparative effectiveness research is a rigorous evaluation of the impact of different options that are available for treating a given medical condition for a particular set of patients. Its purpose is to assist consumers, clinicians, purchasers, and policy makers to make the informed decisions that will improve health care at both the individual and population levels. Systematic reviews are considered the standard practice to inform evidence-based decision making of medical technology. A systematic literature review includes the identification, selection, appraisal, and summary of evidence that can answer a particular research question. Results of several similar studies identified with a systematic literature review can be quantitatively synthesized by means of meta-analysis to obtain a pooled estimate of the outcome of interest and the evaluation of heterogeneity. In its basic form, a meta-analysis typically involves comparisons of two interventions for one particular endpoint, but can be expanded with multiple treatment comparisons or outcomes. This course highlights and expounds upon six key and interrelated areas: 1) comparative effectiveness research, 2) impetus for systematic reviews and meta-analysis, 3) basic steps to perform a systematic literature review, 4) statistical methods of combining data, 5) reporting of results, and 6) appraisal and use of meta-analytic reports. The material is motivated by instructive and real examples. Interactive exercises are an integral part of this short course. This course is designed for those having little experience with meta-analysis, or as a refresher and update for those with more experience.
Faculty Member
Joseph C. Cappelleri, PhD, MPH, MS
Pfizer Inc, Groton, CT, USA
Joseph C. Cappelleri earned his M.S. in statistics from the City University of New York (Baruch College), Ph.D. in psychometrics from Cornell University, and M.P.H. in epidemiology from Harvard University. In June 1996, Dr. Cappelleri joined Pfizer Inc as a statistical scientist collaborating with Outcomes Research and is an executive director of biostatistics at Pfizer. As an adjunct professor, he has served on the faculties at Brown University, Tufts Medical Center, and the University of Connecticut. Dr. Cappelleri has delivered numerous conference presentations and has published extensively on clinical and methodological topics, including regression-discontinuity designs, health measurement scales, and meta-analysis. He has been involved with research and applications on meta-analysis for 25 years and is an associate editor for the journal Research Synthesis Methods. He is lead author of the book “Patient-Reported Outcomes: Measurement, Implementation and Interpretation.” Dr. Cappelleri is a Fellow of the American Statistical Association.
Jeroen P Jansen, PhD
Precision Xtract, Oakland, CA, USA
Jeroen P Jansen is a methodologist working at the intersection of evidence synthesis, biostatistics, and health economics. Currently, he is chief scientist Health Economics & Outcomes Research at the Precision Medicine Group and an adjunct professor of Health Research & Policy (Epidemiology) at Stanford University. Dr. Jansen also acts as scientific advisor for the Innovation & Value Initiative Foundation.
Dr. Jansen has 15 years of research experience in the areas of epidemiology, evidence synthesis, outcomes research, and economic evaluations to understand the value of healthcare interventions. The numerous research projects he led were frequently performed in the context of biopharmaceutical development and manufacturer submissions to health technology assessment agencies. Prompted by the challenges encountered in these projects, he has performed independent methodological research. Notable contributions are the development of novel statistical methods to overcome the typical challenges in model-based cost-effectiveness evaluations characterized by gaps in the evidence base and complex evidence structures. Furthermore, Dr. Jansen led initiatives to develop guidance for consumers and producers of network meta-analysis studies. His current research interests are statistical methods for evidence synthesis and the development of open-source health economic models.
He has published extensively in his areas of expertise and is widely cited. He is co-author of a textbook on network meta-analysis for decision-making and was associate editor for the Journal for Research Synthesis Methods. Dr. Jansen has a PhD in epidemiology from the Erasmus University in the Netherlands.
Value of Information- Active Learning, Modeling Tools and Applications
Level: Intermediate
Track: Economic Evaluation
When the aim of a study is to aid decision makers about acquiring additional information to reduce uncertainty, the ISPOR-SMDM Modeling Good Research Practices Task Force recommends the results of pharmacoeconomic simulation models be presented using Expected Value of Perfect Information (EVPI) and Expected Value of Partial Perfect Information (EVPPI). EVPI and EVPPI estimate the monetary or health value of resolving all of the uncertainty in a decision problem related to all parameters (EVPI) or a subset of parameters (EVPPI). While the use and presentation of EVPI and EVPPI estimates in the literature base has steadily increased, the concept remains complex, especially to consumers of pharmacoeconomic research. This course will provide an overview of probabilistic sensitivity analysis as a building block and prerequisite of EVPI and EVPPI. Through active learning, audience members will calculate EVPI using a one-page simplified exercise. The course will expand on this simplified exercise by demonstrating the calculation and graphical presentation of an EVPI curve and EVPPI analysis from an example Excel-based modeling tool. This value of information modeling tool will be provided as a resource to all course participants. Computers are recommended so participants may follow along with Excel demonstrations. Discussion leaders will present examples of how EVPI and EVPPI can be used to inform reimbursement and research funding decisions and include a policy discussion session for participants to engage and share potential barriers of applying these methods. For this course, faculty strongly recommend that participants bring a fully charged personal laptop equipped with Microsoft Excel 2011 or later to complete all course exercises.
Faculty Member
Jonathan D Campbell, PhD
University of Colorado Denver, Aurora, USA
Jonathan (Jon) Campbell, PhD, is an Associate Professor with tenure in the Department of Clinical Pharmacy at the University of Colorado Anschutz Medical Campus. He recently took on the role as Director of the Center for Pharmaceutical Outcomes Research (CePOR). Coming from a mathematics and biostatistics upbringing, Jon likes to work with numbers and solve problems. His scholarship and teaching is in the field of pharmaceutical outcomes research and in particular, value assessment research applications, methods, and measurement. Nearing Jon’s ten-year anniversary as a University of Colorado faculty member, he has led or coauthored over 90 peer-reviewed manuscripts. He has active funding from federal and non-profit foundations in discovering the value of pharmaceuticals and other medical interventions. One line of research collaboration is with the Institute for Clinical and Economic Review. He recently established The University of Colorado Pharmaceutical Value (pValue) initiative with a Value Assessment Center of Excellence Award from the PhRMA Foundation. The foundation grant was awarded to Jon, R. Brett McQueen, PhD, and Melanie Whittington, PhD and will be housed within CePOR with close collaborative ties to the Data Science to Patient Value initiative supported by the Dean’s Office of the University of Colorado School of Medicine. The mission of pValue is to apply and test novel US healthcare value assessment methods to guide coverage and reimbursement decision making. Dr. Campbell is active in teaching in both the professional PharmD and Pharmaceutical Outcomes Research PhD programs. He is the lead instructor in a PhD course titled, “Applied Cost-Effectiveness Analysis,” where each student conducts their own cost-effectiveness study.
R. Brett McQueen, PhD
Johns Hopkins Bloomberg School of Public Health, Denver, USA
R. Brett McQueen is an Assistant Professor at the University of Colorado (CU) Skaggs School of Pharmacy and Pharmaceutical Sciences, and member in the Center for Pharmaceutical Outcomes Research. His research interests include decision-analytic modeling applications and methodology, applied microeconometrics in health, and novel value assessment methods. Brett has current funding in micro-costing health interventions, evaluating performance-based risk sharing agreements, estimating patient and payer preferences for various pharmaceuticals, and novel value assessment methods. He is the course director for “Pharmaceutical Economics and Policy Analysis” in the Pharmaceutical Outcomes Research PhD program at CU.
Applications in Using Large Databases
Level: Intermediate
Track: Study Approaches
This course will provide an overview of various three health care databases including the Quintiles EMR database, the VA database, the CPRD (UK database) and assorted claims and survey based databases. Each database will be discussed in-depth including directions on how to access the information and how researchers utilize this data to generate information for health care decision making. Instructors will distinguish the important differences between these databases including the limitations and strategies to maximize their value through the use of an interactive assignments and workshops with the audience. The ISPOR International Digest of Databases and its use in identifying health care databases around the globe will be briefly discussed. Participants must have some knowledge of administrative health care database analysis.
Faculty Member
Brandon K Bellows, PharmD, MS
Columbia University, New York, NY, USA
Brandon Bellows received his PharmD and MS from the University of Utah, where he also completed a two-year post-doctoral fellowship in health economics and outcomes research. His research is focused on using computer simulations to estimate the clinical and cost effectiveness of healthcare interventions, particularly in hypertension and cardiovascular disease prevention, and inform implementation decisions.
Joanne LaFleur, PharmD, MPH
George E. Whalen Veterans Health Administration IDEAS Center, Salt Lake City, UT, USA
Joanne LaFleur is an Assistant Professor in the Department of Pharmacotherapy within the University of Utah College of Pharmacy. She has a joint appointment as an Investigator in the Salt Lake City Veterans Affairs Informatics, Decision Enhancement, and Surveillance (IDEAS) Center and is Director of the Data-driven Collaborative of Informatics, Pharmacoepidemiology, and Health Economics Researchers (DeCIPHER).
She received her PharmD degree from the University of Utah in 2003 and went on to complete a Fellowship in Pharmacoeconomics and Outcomes Research as well as a Master’s degree in Public Health in 2005, both at the University of Utah. Dr. LaFleur’s research interests lie primarily in the area of pharmacoepidemiology, epidemiology, pharmacoeconomics, and medical informatics. The focus of her K award from AHRQ, entitled “Knowledge Engineering for Decision Support in Osteoporosis,” is on the development of clinical decision support tools for osteoporotic fracture prevention.
Modeling- Design and Structure of a Model
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 should have a basic understanding of decision analysis. 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, 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.
New! US Healthcare System and Its Approach to Value and Affordability™
Level: Introductory
Track: Health Policy & Regulatory
The US healthcare system is a hybrid system that provides healthcare and/or access to it via various means. This hybrid system can be categorized as: governmental systems (Medicare, Medicaid, Veterans Health Administration, and Department of Defense, etc), private markets (regional and national health plans), and providers assuming more responsibility for access and coverage via mechanisms such as Provider Networks, Integrated Delivery Networks (IDNs), Accountable Care Organizations (ACOs), etc. These different parts of the overall US system are quite heterogeneous and characterized by different coverage and access mechanisms. The course will cover the roots of the current US system, its evolutionary process, and the consequent independence of the different parts of the system. The focus will be on value and affordability as defined by cost, access and quality. While this dynamic US healthcare environment espouses value and affordability, these different participants take various perspectives that may define those goals differently resulting 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 the system. 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 competency needed to work within 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. 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. 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.
Faculty Member
Finn Børlum Kristensen, MD, PhD
Science & Policy, 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.
James F. Murray, PhD
Eli Lilly and Company, Carmel, IN, USA
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.
Karen Worley, PhD
Humana, Cincinnati, OH, USA
5:30 PM - 6:30 PM
EDUCATIONAL SYMPOSIUM
DE-RISKING RISK SHARING- ENABLING PAYER-PHARMA INTERACTIONS AND VALUE-BASED CONTRACTING WITH RWE
Engaging in value-based contracts continues to be a strategic priority among both payers and biopharmaceutical manufacturers seeking to improve health outcomes while curbing healthcare spending. However, implementation challenges exist to constructing risk-sharing agreements including (a) establishing causality between the biopharmaceutical intervention and relevant outcome (b) transparency and alignment in defining and measuring outcomes (c) sufficient data infrastructure to execute and track outcomes (d) assessing upfront risk due to uncertainty of real-world performance. This education symposium brings together key stakeholders — from health insurers and manufacturers to real-world data analytics providers — to describe the benefits and barriers of value-based payment models and the promising role of rapid-cycle analytics of real-world data to address these challenges. Learning objectives will be supported by illustrative case studies.
Sponsor
Aetion
Speaker
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.
Sebastian Schneeweiss, MD, ScD
Brigham and Women's Hospital and Harvard Medical School, Boston, USA
Sharash Shetty, PhD
Boehringer Ingelheim, Ridgefield, CT, USA
Adrian Towse, MA, MPhil
Office of Health Economics, London, United Kingdom
Adrian Towse, MA, MPhil, is the Director of the Office of Health Economics (OHE). OHE is a charitable research organisation (registration number 1170829) which also has a consulting arm (OHE Consulting Ltd). OHE works in the related fields of the economics of HTA, the economics of health care systems and the economics of the life sciences industry. Adrian Towse is a Visiting Professor at the London School of Economics and a Visiting Senior Researcher at the Health Economics Research Centre, Nuffield Department of Population Health at the University of Oxford. Adrian also has been a visiting Professor at the University of York, and for ten years he served as a Non-Executive Director of the Oxford University Hospitals NHS Trust, one of the UK's largest teaching and research hospitals. He chaired the Governance Committee overseeing arrangements for delivering clinical quality.
Adrian has published on 'risk-sharing' arrangements between payers and manufacturers; value-based pricing; HTA development in emerging markets; the economics of stratified (personalized) medicine for payers and the industry; differential pricing, the use of ICERs to arrive at optimal pricing across countries; economic issues that affect both R&D for and access to treatments for diseases prevalent in the developing world; and the economics of medical negligence. Adrian was educated at Keble College, University of Oxford, Nuffield College, University of Oxford, and the Oxford Centre for Management Studies (now the Said Business School). He is also a Member of the Chartered Institute of Management Accountants.
Adrian served on the ISPOR Board of Directors 1999-2001 and subsequently on the ViH Management Board. He has played an active role in the scientific life of ISPOR, presenting at ISPOR Plenary sessions in the US and Europe as well as many Issues Panels and Workshops. He was a member of the Task Force on Prospective Observational Studies and the co-Chair of the Task Force on Performance Based Risk Sharing Agreements (PBRSAs). He has given pre-conference training sessions on PBRSAs with colleagues from the University Of Washington School Of Pharmacy for the last nine years. Adrian served as President of ISPOR in the year 2014-2015.
Sun May 19
8:00 AM - 12:00 PM
SHORT COURSE MORNING SESSION
Advanced Patient-Reported Outcomes
Level: Experienced
Track: Patient-Centered Research
This course provides an in-depth discussion of qualitative and psychometric methods used to develop, evaluate, and interpret patient-reported outcome instruments. Participants will become familiar with common qualitative interview methodology, as well as learn a range of methods for evaluating the reliability, validity, and responsiveness of scores, and set thresholds for interpreting scores and score changes. While instrument development jargon can be daunting, this course will take an accessible approach to understanding the goals of each method, and where it fits into the instrument development process. A variety of methods will be presented, and the course will emphasize that different paths can be taken to achieve similar solutions. Course participants will engage in an interactive exercise throughout the session to gain experience in making instrument development and interpretation decisions based on the integration of qualitative and quantitative information. This course will not utilize a dataset and no programming will be conducted.
Faculty Member
Cheryl Coon, PhD
Outcometrix, Tucson, AZ, USA
Cheryl D. Coon, PhD is a psychometrician with 15 years of experience as a consultant in the pharmaceutical field. She applies qualitative and quantitative methods to the development and evaluation of PRO instruments. During her years in the PRO field, she has been involved in the creation and evaluation of PRO instruments in all stages of the development process, packaging evidence into PRO dossiers and defending the use of PROs to construct endpoints during regulatory interactions. Additionally, she has led the statistical analysis of PRO endpoint data in phase 2 and 3 clinical trials, with particular understanding of the interpretation of PRO scores. She takes a pragmatic approach to PRO instrument development and seeks to make instruments more interpretable and data from them more actionable. Dr. Coon earned her PhD degree in Quantitative Psychology at the University of North Carolina at Chapel Hill, where her research focused on item response theory and where she was involved in the early work on the Patient-Reported Outcomes Measurement Information System.
Jason Lundy, PhD
Outcometrix, Tucson, AZ, USA
J. Jason Lundy, PhD is a measurement scientist specializing in the development and analysis of clinical outcome assessments. Jason has worked on numerous PRO instrument qualification projects in collaboration with FDA and industry colleagues. Over the past ten years, he has written extensively about measurement equivalence and best practices for the electronic implementation of PRO measures in clinical studies. Dr. Lundy has developed PRO measures in a variety of therapeutic areas, and he has helped to support the use of PRO instruments as endpoints in discussions with regulatory agencies. Dr. Lundy earned his PhD in Pharmaceutical Economics, Policy, and Outcomes Research from the University of Arizona.
Use of Instrumental Variables in Observational Studies of Treatment Effects
Level: Intermediate
Track: Study Approaches
In any non-randomized study, selection bias is a potential threat to the validity of conclusions reached. Failure to account for sample selection bias can lead to conclusions about treatment effectiveness or treatment cost that are not really due to the treatment at all, but rather to the unobserved factors that are correlated with both treatment and outcomes. Sample selection models provide a test for the presence of selection bias. These models also provide a correction for selection bias, enabling an investigator to obtain unbiased estimates of treatment effects. This course will discuss the various models and their applications and, in particular, will address instrument variables (two-stage least squares, intuition, and RCTs), including an overview of examples from the current literature. Participants will benefit from interactive exercises using instrumental variables and sample selection techniques using STATA. This course is suitable for those with some knowledge of econometrics. For those who have STATA loaded on their laptops, you are encouraged to bring your laptop.
Faculty Member
Benjamin M. Craig, PhD
Moffitt Cancer Center, Tampa, FL, USA
Benjamin M. Craig, PhD, is an Associate Professor of Economics at the University of South Florida. He received his MS in Economics at the University of Texas at Austin in 1999 and his PhD in Population Health from the University of Wisconsin in 2003. Trained as an econometrician, his research focuses on health preference research and the economics of cancer prevention, detection, and control. He is an active member of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), the International Society for Quality of Life Research (ISOQOL), the International Academy of Health Preference Research (IAHPR), and the EuroQol Group.
Antoine C. El Khoury, PhD, MS
Janssen Pharmaceutical, Raritan, NJ, USA
Antoine C. El Khoury obtained his doctorate degree in economics from Oklahoma State University in 2004 and completed a two year post-doctoral fellowship in pharmacoeconomics and outcomes research at the University of Maryland School of Pharmacy. During his fellowship, he was involved in studies related to adherence to pharmacotherapy, cost-effectiveness of drugs, and cost of disease management. His research focused on the application of statistical and econometric modeling to help in formulary and pharmaceutical decision makings. He worked at Merck for about six years within the Global Health Outcomes Department where he was involved in economic modeling and outcomes research studies for vaccines and infectious disease drugs. Currently he is employed by Johnson and Johnson as Senior Director, Global Access and Policy for Vaccines and serves as an adjunct assistant professor at the University of Arkansas for Medical Sciences' College of Pharmacy. His research has been published in international scientific peer reviewed journals. He is a member of ISPOR.
Bradley Martin, PharmD, PhD, RPh
University of Arkansas for Medical Sciences College of Pharmacy, 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.
Budget Impact Analysis 1 – 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
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.
Josephine Mauskopf, PhD
RTI Health Solutions, Research Triangle Park, NC, USA
Josephine Mauskopf, PhD, MHA, is Vice President of Health Economics at RTI‑HS. She has extensive experience both as a consultant and within the pharmaceutical industry designing and implementing pharmacoeconomic research strategies. She has designed pharmacoeconomic research programs for drugs for bacterial infections, viral infections, psychiatric illness, and neurologic diseases. Dr. Mauskopf has estimated budget impacts for new products for schizophrenia, multiple sclerosis, bipolar disease, breast cancer, hepatitis C and HIV infection and vaccines for infectious diseases. She has estimated the cost effectiveness of antiretroviral drugs, as well as drugs for treating Alzheimer’s disease, multiple sclerosis, fungal infections, herpes zoster, epilepsy, neonatal respiratory distress syndrome, digoxin toxicity, community-acquired pneumonia, intra-abdominal infections, influenza vaccination, and primary pulmonary hypertension. Dr. Mauskopf also has estimated the impact of an antidepressant on work and social disability. Dr. Mauskopf has performed many strategic literature reviews that have been published in peer-reviewed journals, including reviews of cost of care for Alzheimer’s disease, cost of care for treatment-resistant depression, a review of the methods used to estimate the cost-effectiveness of vaccine programs using dynamic transmission models, cost-effectiveness of tiotropium for chronic obstructive pulmonary disease, and treatments for uterine fibroids. Dr. Mauskopf has developed Markov models of disease progression for lung cancer and HIV infection and has developed simulation models of time spent in the operating and recovery rooms and of disease progression for HIV infection. Dr. Mauskopf has used multi-criteria decision analysis (MCDA) to estimate the likelihood of reimbursement for products with different attributes and to the impact of benefits and risks on drug prescribing decisions.
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 received his BS in Economics from M.I.T. and his PhD in Economics from Duke University. His research and teaching focus on comparative effectiveness research (CER), patient-centered outcomes research (PCOR), pharmacoeconomics, and health disparities research. He directs the University of Maryland PATient-centered Involvement in Evaluating effectiveNess of TreatmentS (PATIENTS) Program, which received a University of Maryland Baltimore Champion of Excellence Award. He has received funding as a Principal Investigator from the NIH/NIA, NIH/NHLBI, AHRQ, and the Patient-Centered Outcomes Research Institute (PCORI) and was the Shared Resources Core Director for the NIH-sponsored University of Maryland Center for Health Disparities Research, Training, and Outreach. He previously served as a Regular Member of the AHRQ HSR and the NCI-J Study Sections and currently serves as a Regular Member for the AHRQ HCRT Study Section. He also has chaired PCORI Study Sections. In addition to his work on federal grants, Professor Mullins is co-Editor-in-Chief for Value in Health and is author/co-author of approximately 200 peer-reviewed articles and book chapters on pharmacoeconomics, outcomes research, and pharmaceutical policy and health disparities research in journals such as The American Journal of Managed Care, The American Journal of Public Health, Cancer, Chest, Health Affairs, Health Services Research, JAMA, Journal of Clinical Oncology, Pharmacoeconomics, and Social Science & Medicine. He has received an Outstanding Service Award from the Drug Information Association (DIA) and two Service Awards from the International Society for Pharmacoeconomics and Outcomes Research (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. In 2014, he and Robin Newhouse were named co-researchers of the year for the University of Maryland Baltimore campus.
Using DICE Simulation for Health Economic Analyses
Level: Introductory
Track: Methodological & Statistical Research
This course will provide a basic understanding of the concepts of discretely-integrated condition event (DICE) simulation as it is applied in health technology assessment (HTA). Topics to be covered are: what is the basic idea of DICE; what are its components; how does it work; how is it conceptualized; how are outcomes obtained; how to implement a DICE in EXCEL (including both discrete event simulation and Markov models, and their combination in a single structure); how to do structural sensitivity analyses; what are the advantages and disadvantages of DICE. For this course, participants are required to bring a fully charged personal laptop equipped with Microsoft Excel installed, a version 2007 or later. It needs to be a Windows version so if it is on a Mac, a Windows emulator and Windows Excel is needed.
Faculty Member
J. Jaime Caro, MDCM, FRCPC
Evidera, Waltham, MA, EUA y Profesor de Epidemiología y Medicina y Bioestadisticas, Universidad McGill, Montreal, QC, Canada
J. Jaime Caro es el jefe científico de Evidera y profesor de epidemiología y medicina en la Universidad McGill y profesor de la Escuela de Economía de Londres. Él fue pionero en el uso de SED, desarrolló el método de comparación de tratamientos por vía de simulación y propuso la frontera de eficiencia como una alternativa viable al uso de costo por AVAC. Recientemente, ha desarrollado un nuevo método para modelos, la simulación DICE, apto para los problemas de evaluación de las tecnologías en salud.
Jorgen Moller, MSc
Lund University, Lund, Sweden
Jörgen Möller, MSc, is Vice President, Modeling Technologies with Evidera in London, UK. He specializes in simulating complex systems and played a key role in simulating the re-design of the Swedish national postal distribution system, the extension of Stockholm-Arlanda Airport, and the utilization of surgical theatres at Eskilstuna Hospital. Intrigued by the dearth of simulation in pharmacoeconomics and outcomes research, Mr. Möller joined Caro Research in 2003 as a specialist in discrete event simulation (DES) and has been instrumental in implementing DES as a modeling tool in medicine. His focus has been on translating methods from operations research to pharmacoeconomics and on developing guidelines for this type of modeling. Mr. Möller has created more than a dozen DES models in the areas of devices and pharmaceuticals. He also conducts advanced training courses in DES and the ARENA-software. Mr. Möller received an MSc-degree in mechanical engineering at Lund University, Sweden.
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
Brian W. Bresnahan, PhD
University of Washington and Harborview Medical Center, Seattle, WA, USA
Michael Drummond, MCom, DPhil
University of York, York, United Kingdom
Michael Drummond, BSc, MCom, DPhil is Professor of Health Economics and former Director of the Centre for Health Economics at the University of York. His particular field of interest is in the economic evaluation of health care treatments and programmes. He has undertaken evaluations in a wide range of medical fields including care of the elderly, neonatal intensive care, immunization programmes, services for people with AIDS, eye health care and pharmaceuticals. He is the author of two major textbooks and more than 650 scientific papers, and has acted as a consultant to the World Health Organization and the European Union. He has been President of the International Society of Technology Assessment in Health Care, and the International Society for Pharmacoeconomics and Outcomes Research. In October 2010 he was made a member of the National Academy of Medicine in the USA. He has advised several governments on the assessment of health technologies and chaired one of the Guideline Review Panels for the National Institute for Health and Care Excellence (NICE) in the UK. He is currently Co-Editor-in-Chief of Value in Health and has been awarded 3 honorary doctorates, from City University (London), Erasmus University (Rotterdam) and the University of Lisbon.
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.
Belinda A. Mohr, Ph.D.
W. L. Gore & Associates, Inc., Phoenix, AZ, USA
Belinda A. Mohr is the Lead Health Economist on the Global Health Economics and Outcomes Research Team at W. L. Gore & Associates in Phoenix, AZ. She has conducted several economic evaluations to demonstrate the value of Gore medical devices to multiple stakeholders. Her work has been disseminated in peer-reviewed publications, podium and poster presentations, reimbursement submissions, and sales and marketing tools. Prior to joining Gore, Dr. Mohr was an Economist for the U.S. Food and Drug Administration working on cost-benefit analyses of proposed FDA regulations. She focused on estimating the public health benefits and the costs to FDA regulated industries of implementing the regulations. She holds a Ph.D. and M.A. in Economics from the University of California, Santa Barbara.
Stated Preference Methods – Part 1
Level: Introductory
Track: Patient-Centered Research
The need to understand patient preferences for health and healthcare has become well established and demand for stated preference studies has grown exponentially in recent years. This course is designed as an introduction to a range of stated-preference methods and the application of these methods in health. The course will also provide an overview of good research practices and principles that are broadly applicable to all stated-preference methods and describe how good research practices can be applied to discrete choice experiments and several other stated-preference methods. This course will include hands-on exercises and detailed case studies of recent empirical examples to illustrate concepts.
Faculty Member
John F. P. Bridges, PhD
The Ohio State University College of Medicine, Columbus, OH, USA
John F P Bridges PhD is a professor in the Departments of Biomedical Informatics and Surgery within the Ohio State University College of Medicine. He is core faculty within the newly created Center for the Advancement of Team Science, Analytics and Systems Thinking in Health Systems Research and Implementation Science (CATALYST) and the Center for Surgical Health Assessment, Research and Policy (SHARP).
John’s research promotes the translation and implementation of advances in medicine through informed decision making. He is an international leader in advancing and applying methods to promote a greater understanding of the preferences of patients and other stakeholders in medicine. Through community-centered research conducted in partnership with several patient organizations, PCORI, and the FDA, John has demonstrated the importance of incorporating the perspectives of patients and caregivers in the assessment of the benefits and risk of treatments. This work has helped advance the science of patient engagement, especially as it relates to regulatory science.
In 2008 John founded a new journal, The Patient – Patient Centered Outcomes Research, as one of the first academic venues for research focused on understanding the patient’s perspective in medicine. Within the International Society of Pharmacoeconomics and Outcomes Research (ISPOR) he was the founding chair of the Conjoint Analysis Working Group and the Conjoint Analysis Task Force that produced several reports on good research practices for stated-preference methods. In 2006 he received ISPOR’s Bernie O’Brien New Investigator Award and in 2011 received an ISPOR Distinguished Service Award for his contributions to the field. He is the author of over 200 articles and a frequent speaker on the art and science of studying the patient’s perspective.
John received his PhD in Economics from the City University of New York Graduate Center. Prior to joining OSU he was on the faculty of the Johns Hopkins Bloomberg School of Public Health, the Department of Tropical Hygiene and Public Health within University of Heidelberg School of Medicine and the Department of Epidemiology and Biostatistics within the Case Western Reserve University School of Medicine. He has previously held positions within Department of Economics within the Weatherhead School of Management at Case Western Reserve University, the National Bureau of Economic Research (NBER), and the Center for Health Economics, Research and Evaluation (CHERE) in Australia.
Brett Hauber, PhD
RTI Health Solutions, Research Triangle Park, NC, USA
Brett Hauber, PhD, is a Senior Economist and the Vice President of Health Preference Assessment at RTI‑HS and an Affiliate Associate Professor in the School of Pharmacy at the University of Washington. He has more than 20 years of academic, research, and government experience in health and environmental economics. His primary area of specialization is in quantifying preferences for health interventions and health outcomes. He also has extensive experience in conducting benefit-risk analysis with patients and other health care decision makers. He has studied the theoretical and empirical relationships among various health utility measures. His most recent applied work has included the use of multiple stated preference techniques to elicit patient and physician benefit-risk preferences for treatments for conditions in numerous therapeutic areas. Dr. Hauber regularly teaches courses on conjoint analysis and discrete-choice experiments. He was a member of to the Patient-Centered Benefit-Risk Steering Committee of the Medical Device Innovation Consortium (MDIC) and was the principal investigator for developing the Catalog of Methods for Assessing Patient Preferences for Benefits and Harms of Medical Technologies for MDIC. He is currently a member of the scientific advisory board for the IMI-PREFER project and an advisor to a number of initiatives led by industry and patient-advocacy organizations to incorporate patient preferences in regulatory and reimbursement decision making in multiple disease areas. He was the chair of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Conjoint Analysis–Statistical Analysis, Reporting, and Conclusions (CA-SARC) task force and was previously a member of the ISPOR task force that developed the ISPOR Checklist for Good Research Practices in Conjoint Analysis. Dr. Hauber's research has been published in numerous health outcomes and medical journals.
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.
Cost-Effectiveness Analysis Alongside Clinical Trials
Level: Introductory
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." Familiarity with economic evaluations will be helpful.
Faculty Member
Scott D. Ramsey, MD, PhD
Fred Hutchinson Cancer Research Center and University of Washington, Seattle, WA, USA
Dr. Ramsey is a general internist and health economist. He is a Full Member in the Cancer Prevention Program, Public Health Sciences Division at the Fred Hutchinson Cancer Research Center, where he directs Hutchinson Institute for Cancer Outcomes Research, a multidisciplinary team devoted to clinical and economic evaluations of new and existing cancer prevention, screening and treatment technologies. In addition, Dr. Ramsey is a Professor in the Schools of Medicine and Pharmacy at the University of Washington. Trained in Medicine and economics, Dr. Ramsey’s research focuses on economic evaluations in cancer. He has published widely on patterns of care, costs, and cost-effectiveness of treatments for lung, colorectal, and prostate cancer. His research portfolio and interests include: large scale SEER-Medicare/Cancer Registry data linkages, patient reported outcomes, economic modeling of health care interventions, cost-effectiveness analysis, quality of life assessment, patterns of care, health care utilization, economic burden of disease for patients and society, pragmatic trial design, early technology assessment, and stakeholder engagement. Dr. Ramsey is co-Chair of the Outcomes and Comparative Effectiveness Committee of the Southwest Oncology Group, past President of the International Society of Pharmacoeconomics and Outcomes Research (ISPOR), and has served on the IOM Cancer Policy Forum.
Richard Willke, PhD
ISPOR, Lawrenceville, NJ, USA
Richard J. Willke, PhD is chief science officer of ISPOR, the leading global professional society for health economics and outcomes research. Dr Willke has more than 25 years of experience in the life sciences arena and has specialized in outcomes research in a succession of group leadership roles with Pfizer and its legacy companies.
At ISPOR, Dr Willke is responsible for designing and implementing strategic initiatives related to scientific research and content priorities that will advance the Society’s mission of promoting health economics and outcomes research excellence to improve decision making for health globally.
Previously, Dr Willke was vice president, Outcomes and Evidence Cluster Lead at Pfizer for its Global Health and Value division. He has also served in a number of leadership roles with affiliated organizations, including the Chair of ISPOR Institutional Council (2010), ISPOR Board of Directors (2007-2009), and Chair of the PhRMA Health Outcomes Committee (2002-2004).
Prior to joining industry, Dr Willke served as department director in the Center of Health Policy Research at the American Medical Association and held research and teaching positions at The Ohio State University.
Dr Willke earned a PhD and MA in economics from Johns Hopkins University. He has authored more than 80 scholarly publications that examine the science and methodologies of health economics and outcomes research.
Statistical Methods in Economic Evaluations
Level: Intermediate
Track: Economic Evaluation
Economic evaluations play an integral role in informing health care policy decisions. These studies often rely on data from clinical trials, prospective registries, and secondary data. The availability of patient-level data allows analysts to apply conventional and innovative statistical methods to patient-level data. In this course, faculty will examine statistical approaches that address common features of resource use and cost data, including distributional characteristics, censoring, hierarchical data structures, and potential confounding. Faculty will also examine additional statistical issues that arise when combining patient-level estimates of costs and effectiveness. Throughout the course, faculty will include examples of statistical analyses. Participants should have basic knowledge of economic evaluations and statistics
Faculty Member
Brad Hammill, DrPh, MS
Duke University, Durham, NC, USA
Brad Hammill is Associate Professor in the Department of Population Health Sciences and faculty at the Duke Clinical Research Institute at Duke University. His primary expertise and experience is in outcomes research and observational data analysis. He has used administrative data, including Medicare claims, extensively for many different types of research, including comparative effectiveness studies, cost analysis studies, and incidence/prevalence studies. With his strong background in complex statistical programming, he has also implemented a method to link these administrative data to clinical registry data.
Shelby Reed, PhD, RPh
Duke University, Durham, USA
Shelby D. Reed, PhD, RPh, is Professor in the Departments of Population Health Sciences and Medicine at the Duke Clinical Research Institute in Durham, NC. Dr. Reed received her pharmacy and doctoral degrees from the School of Pharmacy at the University of Maryland and completed her training in the Pharmaceutical Outcomes Research and Policy Program at the University of Washington. 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 health care 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.
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.
Faculty Member
Josh Carlson, MPH, PhD
University of Washington, Seattle, WA, USA
Dr. Carlson is an Associate Professor in the University of Washington Pharmaceutical Outcomes Research and Policy Program (PORPP) and an Affiliate Investigator in the Cancer Prevention Program at the Fred Hutchinson Cancer Research Center. Dr. Carlson's current research interests and work to date has primarily focused on the intersection of three areas: 1) genomics and emerging technologies in the field of personalized medicine; 2) uncertainty both in our decision-making processes and as the concept applies to the application of medical technologies in "real world" settings (i.e. outside of clinical trials) including comparative effectiveness research; and 3) economic and policy options to address these uncertainties as we seek to improve our health care system and the health of our population. Dr. Josh Carlson graduated with his PhD from the Institute for Public Health Genetics in the School of Public Health and Community Medicine at the University of Washington in 2007 and received his MPH from the same department in 2004. Dr. Carlson conducted his postdoctoral training in pharmacoeconomics with the University of Washington from 2007-2009.
Louis P. Garrison, PhD
University of Washington, Seattle, WA, USA
Louis P. Garrison, Jr., PhD, is Professor Emeritus in the Pharmaceutical Outcomes Research & Policy Program in the School of Pharmacy, and has been Adjunct Professor in the Departments of Global Health and Health Services at the University of Washington, where he joined the faculty in 2004. In October 2016, he became Senior Visiting Fellow at the Office of Health Economics, London, UK. For the first 13 years of his career, Dr. Garrison worked in non-profit health policy research first at the Battelle Human Affairs Research Centers (Seattle), and then at the Project HOPE Center for Health Affairs (Virginia), 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 140 publications in peer-reviewed journals. His research interests include national and international health policy issues related to personalized medicine, benefit-risk analysis, insurance, pricing, reimbursement, and risk-sharing agreements, as well as the economic evaluation of pharmaceuticals, diagnostics, devices, surgical procedures, and vaccines, particularly as related to organ transplantation, influenza, measles, obesity, and cancer. From 2007-2009, Dr. Garrison served on the ISPOR Board of Directors. He co-chaired two ISPOR Good Practice Task Forces—on Real-World Data and on Performance-Based Risk-Sharing Arrangements—and he chaired the ISPOR Health Science Policy Council from 2012 to 2015. He is currently co-chair of the ISPOR Special Task Force on U.S Value Frameworks, and faculty advisor for the UW ISPOR Student Chapter. He was elected as ISPOR President for July 2016-June 2017, and is currently serving on the Board for one year as Immediate Past President and as chair of the Past Presidents’ Council.
Adrian Towse, MA, MPhil
Office of Health Economics, London, United Kingdom
Adrian Towse, MA, MPhil, is the Director of the Office of Health Economics (OHE). OHE is a charitable research organisation (registration number 1170829) which also has a consulting arm (OHE Consulting Ltd). OHE works in the related fields of the economics of HTA, the economics of health care systems and the economics of the life sciences industry. Adrian Towse is a Visiting Professor at the London School of Economics and a Visiting Senior Researcher at the Health Economics Research Centre, Nuffield Department of Population Health at the University of Oxford. Adrian also has been a visiting Professor at the University of York, and for ten years he served as a Non-Executive Director of the Oxford University Hospitals NHS Trust, one of the UK's largest teaching and research hospitals. He chaired the Governance Committee overseeing arrangements for delivering clinical quality.
Adrian has published on 'risk-sharing' arrangements between payers and manufacturers; value-based pricing; HTA development in emerging markets; the economics of stratified (personalized) medicine for payers and the industry; differential pricing, the use of ICERs to arrive at optimal pricing across countries; economic issues that affect both R&D for and access to treatments for diseases prevalent in the developing world; and the economics of medical negligence. Adrian was educated at Keble College, University of Oxford, Nuffield College, University of Oxford, and the Oxford Centre for Management Studies (now the Said Business School). He is also a Member of the Chartered Institute of Management Accountants.
Adrian served on the ISPOR Board of Directors 1999-2001 and subsequently on the ViH Management Board. He has played an active role in the scientific life of ISPOR, presenting at ISPOR Plenary sessions in the US and Europe as well as many Issues Panels and Workshops. He was a member of the Task Force on Prospective Observational Studies and the co-Chair of the Task Force on Performance Based Risk Sharing Agreements (PBRSAs). He has given pre-conference training sessions on PBRSAs with colleagues from the University Of Washington School Of Pharmacy for the last nine years. Adrian served as President of ISPOR in the year 2014-2015.
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, 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 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 teaches courses in decision-analytic modeling, HTA, economic evaluation, analysis of big data, and advanced causal epidemiologic methods at several universities and for industry in Europe, USA, South America, and Asia.
Prof. Siebert is the President of the Society for Medical Decision Making (SMDM), a member of the Latin America Consortium Advisory Committee of the International So
