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Short Courses

The ISPOR Short Course Program is offered in conjunction with ISPOR conferences around the world as a series of 4- and 8-hour training courses, designed to enhance your knowledge and technique in 7 key topic areas (“Tracks”) related to health economics and outcomes research (HEOR).  Short courses range in skill level from Introductory to Experienced.

For more information on ISPOR Short Courses, please visit this page or for questions related to this or any other ISPOR Short Course Program, please contact shortcourse@ispor.org.

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May 18, 2019

8:00 AM - 12:00 PM

Elements of Pharmaceutical/Biotech Pricing I - Introduction

Level

Introductory

Track

Health Policy & Regulatory


  • Description
  • 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. 



May 18, 2019

8:00 AM - 12:00 PM

Utility Measures

Level

Introductory

Track

Patient-Centered Research


  • Description
  • 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. 



May 18, 2019

8:00 AM - 12:00 PM

New! Tools for Reproducible Real-World Data Analysis

Level

Intermediate

Track

Real World Data & Information Systems


  • Description
  • 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.



May 18, 2019

8:00 AM - 5:00 PM

Bayesian Analysis: Overview and Applications

Level

Introductory

Track

Methodological & Statistical Research


  • Description
  • 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.



May 18, 2019

8:00 AM - 12:00 PM

Introduction to Modeling Methods

Level

Introductory

Track

Methodological & Statistical Research


  • Description
  • 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.



May 18, 2019

8:00 AM - 12:00 PM

Introduction to the Design & Database Analysis of Observational Studies of Treatment Effects Using Retrospective Data Sources

Level

Introductory

Track

Study Approaches


  • Description
  • 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.



May 18, 2019

8:00 AM - 12:00 PM

Introduction to Patient-Reported Outcomes

Level

Introductory

Track

Patient-Centered Research


  • Description
  • 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. 



May 18, 2019

8:00 AM - 5:00 PM

Introduction to Health Economics and Outcomes Research

Level

Introductory

Track

Economic Evaluation


  • Description
  • 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. 



May 18, 2019

1:00 PM - 5:00 PM

New! Health State Utility (HSU) Recommendations for Identification and Use of HSU Data in Cost-Effectiveness Modelling

Level

Introductory

Track

Patient-Centered Research


  • Description
  • 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.



May 18, 2019

1:00 PM - 5:00 PM

Use of Propensity Scores in Observational Studies of Treatment Effects

Level

Intermediate

Track

Study Approaches


  • Description
  • Previous attendance at the short course “Introduction to the Design & Analysis of Observational Studies of Treatment Effects Using Retrospective Data Sources,” or equivalent knowledge, is recommended. 

    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. 



May 18, 2019

1:00 PM - 5:00 PM

Case Studies in Pharmaceutical/Biotech Pricing II - Advanced

Level

Intermediate

Track

Health Policy & Regulatory


  • Description
  • Participation in the ISPOR Short Course, “Elements of Pharmaceutical / Biotech Pricing I – Introduction,” or equivalent knowledge, is required. 

    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. 



May 18, 2019

1:00 PM - 5:00 PM

Meta-Analysis and Systematic Reviews in Comparative Effectiveness Research

Level

Introductory

Track

Study Approaches


  • Description
  • 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. 



May 18, 2019

1:00 PM - 5:00 PM

Value of Information: Active Learning, Modeling Tools, and Applications

Level

Intermediate

Track

Economic Evaluation


  • Description
  • Participation in the ISPOR short course “Introduction to Modeling Methods,” or equivalent knowledge, is required. 

    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.



May 18, 2019

1:00 PM - 5:00 PM

New! US Healthcare System and its Approach to Value and Affordability™

Level

Introductory

Track

Health Policy & Regulatory


  • Description
  • 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. 



May 18, 2019

1:00 PM - 5:00 PM

Applications in Using Large Databases

Level

Intermediate

Track

Study Approaches


  • Description
  • Previous attendance at the ISPOR Short Course, “Introduction to Design &Analysis of Observational Studies of Treatment Effects Using Retrospective Data Sources,” or equivalent knowledge, is recommended. 

    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. 



May 18, 2019

1:00 PM - 5:00 PM

Modeling: Design and Structure of a Model

Level

Intermediate

Track

Methodological & Statistical Research


  • Description
  • Participation in the short course “Introduction to Modeling Methods,” or equivalent knowledge, is required. 

    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.



May 19, 2019

8:00 AM - 12:00 PM

Introduction to Health Technology Assessment

Level

Introductory

Track

Health Technology Assessment


  • Description
  • 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. 



May 19, 2019

8:00 AM - 12:00 PM

Statistical Methods in Economic Evaluations

Level

Intermediate

Track

Economic Evaluation


  • Description
  • 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



May 19, 2019

8:00 AM - 12:00 PM

Cost-Effectiveness Analysis alongside Clinical Trials

Level

Introductory

Track

Economic Evaluation


  • Description
  • 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. 



May 19, 2019

8:00 AM - 12:00 PM

Stated Preference Methods - Part 1

Level

Introductory

Track

Patient-Centered Research


  • Description
  • 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.



May 19, 2019

8:00 AM - 12:00 PM

Risk-Sharing/Performance-Based Arrangements for Drugs and other Medical Products

Level

Intermediate

Track

Health Policy & Regulatory


  • Description
  • It will be helpful for individuals to have completed the short course, “Elements of Pharmaceutical / Biotech Pricing I – Introduction,” or to be familiar with both the key determinants of pharmaceutical pricing and the main international health systems. 

    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.



May 19, 2019

8:00 AM - 12:00 PM

New! Market Access & Value Assessment of Medical Devices

Level

Intermediate

Track

Health Policy & Regulatory


  • Description
  • 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.



May 19, 2019

8:00 AM - 12:00 PM

Use of Instrumental Variables in Observational Studies of Treatment Effects

Level

Intermediate

Track

Study Approaches


  • Description
  • Previous attendance at the ISPOR Short Course, “Introduction to the Design & Analysis of Observational Studies of Treatment Effects Using Retrospective Data Sources,” or equivalent knowledge, is recommended. 

    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.




May 19, 2019

8:00 AM - 12:00 PM

Budget Impact Analysis I - A 6-Step Approach

Level

Intermediate

Track

Economic Evaluation


  • Description
  • 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. 



May 19, 2019

8:00 AM - 12:00 PM

Advanced Patient-Reported Outcomes

Level

Intermediate

Track

Patient-Centered Research


  • Description
  • 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.



May 19, 2019

8:00 AM - 12:00 PM

Using DICE Simulation for Health Economic Analyses

Level

Introductory

Track

Methodological & Statistical Research


  • Description
  • 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.



May 19, 2019

1:00 PM - 5:00 PM

Patient-Reported Outcomes: Item Response Theory

Level

Intermediate

Track

Patient-Centered Research


  • Description
  • There is a great need in health outcomes research to develop instruments that accurately measure a person's health status with minimal response burden. This need for psychometrically sound and clinically meaningful measures calls for better analytical tools beyond the methods available from traditional measurement theory. Applications of item response theory (IRT) modeling have increased considerably because of its utility for instrument development and evaluation, assessment of measurement equivalence, instrument linking, and computerized adaptive testing. IRT models the relationship, in probabilistic terms, between a person's response to a survey question and their standing on a health construct such as fatigue or depression. This information allows instrument developers to develop reliable and efficient quality of life measures tailored for an individual or group. This introductory course will discuss the basics of IRT models and applications of these models to improve health outcomes measurement. Illustrations that focus on measuring key health-related quality of life domains in different disease populations will be used throughout the presentation. The NIH Patient-Reported Outcomes Measurement Information System (PROMIS) project will also be discussed for its relevance for assessing patient-reported outcomes using modern psychometric methods. This course is designed for those with little to no experience with IRT. 



May 19, 2019

1:00 PM - 5:00 PM

Causal Inference and Causal Diagrams in Big, Real-World Observational Data and Pragmatic Trials

Level

Experienced

Track

Real World Data & Information Systems


  • Description
  • Previous attendance at the ISPOR short course, “Introduction to the Design & Analysis of Observational Studies of Treatment Effects Using Retrospective Data Sources”, or equivalent knowledge, is recommended. 

    Innovative causal inference methods are needed for the design and analysis of big real world observational data and pragmatic trials. This course will provide an introduction to the principles of causation in comparative effectiveness research, the use of causal diagrams (directed acyclic graphs; DAGs), and focus on causal inference methods for time-independent confounding (multivariate regression, propensity scores) and time-dependent confounding (g-formula, marginal structural models with inverse probability of treatment weighting, and structural nested models with g-estimation). The “target trial” concept and a counterfactual approach with “replicates” will be used to apply causal methods to big real-world datasets with case examples from oncology, cardiovascular disease, HIV, nutrition and obstetrics. The course will consist of lectures, exercises drawn from the published literature and interactive discussion. The intended audience includes researchers from all substance matter fields, statisticians, epidemiologists, outcome researchers, health economists and health policy decision makers interested either in methods of causal analysis or causal interpretation of results based on the underlying method.



May 19, 2019

1:00 PM - 5:00 PM

Stated Preference Methods - Part II

Level

Intermediate

Track

Patient-Centered Research


  • Description
  • The need to understand patient preferences for health and healthcare has become well established and demand for stated preference studies – especially conjoint analysis and discrete-choice experiments – has grown exponentially in recent years. This course will provide an in-depth overview of methods for conducting stated-preference studies including designing a survey, developing an experimental design, analyzing data, and presenting results. The course will review good research practices for conducting studies using conjoint analysis and discrete-choice experiments and review recent practical and methodological advances in this developing field. The course will include hands-on exercises and detailed case studies of recent empirical examples to illustrate concepts.



May 19, 2019

1:00 PM - 5:00 PM

Network Meta-Analysis

Level

Intermediate

Track

Study Approaches


  • Description
  • For several medical questions of interest, many treatment options exist for the same indication. These treatments may have been compared against placebo or against each other in clinical trials. Knowing whether one specific treatment is better than placebo or some other specific comparator is only a fragment of the big picture, which should incorporate all available information. Ideally, one would know how all the treatment options rank against each other and the level of differences in treatment effects between all the available options. Network meta-analysis provides an integrated and unified method that incorporates all direct and indirect comparative evidence about treatments. Based in part on the ISPOR Task Force Reports on Indirect Treatment Comparisons, the fundamentals and concepts of network meta-analysis will be presented. The evaluation of networks presents special challenges and caveats, which will also be highlighted in this course. The material is motivated by instructive and concrete examples. The ISPOR-AMCP-NPC questionnaire for assessing the credibility of a network meta-analysis will also be introduced. This course requires at least a basic knowledge of meta-analysis and statistics. 



May 19, 2019

1:00 PM - 5:00 PM

Budget Impact Analysis II - Applications and Design Issues

Level

Intermediate

Track

Economic Evaluation


  • Description
  • Participation in the ISPOR short course, “Budget Impact Analysis I: A 6-Step Approach,” or equivalent knowledge, is recommended. 

    This course provides an opportunity for participants to engage with concrete applications of the six-step approach for developing budget impact analyses and to participate in hands-on learning with two different budget impact models programmed in Excel. The course will first review the basics of budget impact analysis, interpretation of results, critical questions to consider when using a budget impact analysis, and how such analyses are used by payers and other decision makers. The course will cover technical topics such as use of static versus dynamic budget impact models, considerations for budget impact analyses of device and diagnostic technologies, and how to handle important issues such as patient copayments, adherence, and use of generics. To help participants engage with the course content, the instructors will walk through two different budget impact analyses programmed in Excel (one static and one dynamic), work with participants on hands-on exercises to modify and complete these models, present conceptual content, and lead discussion on various topics. The instructors will also discuss good practices for building budget impact models and provide a number of Excel tips. The Excel-based budget impact models used for the course will be provided to participants in advance of the conference. This course is designed for those who have basic knowledge of budget impact analyses and desire exposure to these analyses in Excel. Participants who wish to gain hands-on experience must bring their laptops with Microsoft Excel for Windows installed.



May 19, 2019

1:00 PM - 5:00 PM

Preference Data for Patient-Centric Benefit Risk Analysis

Level

Experienced

Track

Patient-Centered Research


  • Description
  • Previous attendance at the ISPOR short course, “Conjoint Analysis – Theory and Methods”, or equivalent knowledge of stated-preference methods, is required. 

    Course participants will learn how to design and apply effective stated-preference studies for use in benefit-risk assessments, the study requirements to correctly quantify preference weights for beneficial and harmful outcomes in accordance with published FDA guidance, the role and interpretation of risk-tolerance measures conditioned on treatment benefits, and how to use the results in structured benefit-risk frameworks. The course will include example applications to drug-development and regulatory decision making.



May 19, 2019

1:00 PM - 5:00 PM

A Health Economics Approach to US Value Assessment

Level

Introductory

Track

Health Technology Assessment


  • Description
  • This short course will focus on the recent ISPOR Special Task Force Report, “A Health Economics Approach to US Value Frameworks.” It will begin with an overview of recent US value assessment frameworks, with emphasis on the importance of perspective and decision context in the construction and use of value frameworks. It will then review how a health economics approach from a societal or health plan perspective leads to use of cost-effectiveness analysis (CEA) to help guide efficient resource allocation. There will be in-depth discussion of how measuring some aspects of the value of health benefits could augment the standard cost-per-quality-adjusted-life-year metric for CEA. Elements such as value of insurance, value of “hope,” real option value, severity of illness, and several others, have the potential to better capture how patients and/or society value the benefits of some treatments; each one is based on some research findings and some case examples will be shown. The course will then review how budget considerations, cost-effectiveness thresholds, and opportunity costs enter CEA-based decision-making. Next faculty will review broader approaches to cost-benefit aggregation and value-based decision-making, including extended CEA, augmented CEA (introduced by this Report), and multi-criteria decision analysis (MCDA), with an overview of issues and new approaches to MCDA. It then discusses the strengths and weaknesses of recent US value assessment frameworks from this health economic perspective, and closes with a review of the high-level recommendations of this Special Task Force.



May 19, 2019

1:00 PM - 5:00 PM

Advanced Decision Modeling for Health Economic Evaluations

Level

Experienced

Track

Methodological & Statistical Research


  • Description
  • Previous attendance at the short course “Modeling: Design and Structure of a Model,” or equivalent knowledge, is required. 

    During this course, the key aspects and new developments of decision modeling for economic analysis will be considered. How models can be made probabilistic to capture parameter uncertainty (including rationale, choosing parameter distributions, and types of uncertainty) will be covered. How to analyze and present the results of probabilistic models, how the results of probabilistic decision modeling should be interpreted, and how decisions should be made (including decisions with uncertainty and expected value of perfect information [EVPI]), will be presented. Specific examples using Excel programming will be used to illustrate concepts. Participants should have an understanding of decision analysis.