Introduction to Pharmacoeconomics
SATURDAY, MAY 15, 8:00 AM - 5:00 PM
Course Description: This course is designed to teach clinicians and new researchers how to incorporate pharmacoeconomics into study design and data analysis. Participants will learn 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. The development of economic protocols and data collection sheets will be discussed. Different pharmacoeconomic models and techniques will be demonstrated and practiced in lectures and case studies. These include cost-minimization, cost-of-illness, cost-effectiveness, cost-benefit, and cost-utility analysis. Decision analysis, sensitivity analysis, and discounting, will all be demonstrated and practiced. Participants will also learn to compare and evaluate interventions such as drugs, devices and clinical services. This course is suitable for those with little or no experience with pharmacoeconomics.

Cost-Effectiveness Analysis alongside Clinical Trials
SATURDAY, MAY 15, 8:00 AM - 12:00 PM
Course 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, the Good Research Practices for Cost-Effectiveness Analysis alongside Clinical Trials: the ISPOR RCT-CEA Task Force Report. Trial design, selecting data elements, database design and management, analysis, and reporting of results will be presented. Trials designed to evaluate effectiveness (rather than efficacy), as well as clinical outcome measures will be discussed. How to obtain health resource use and health state utilities directly from study subjects and economic data collection fully integrated into the study will also be discussed. Analyses guided by an analysis plan and hypotheses, an incremental analysis using an intention to treat approach, characterization of uncertainty, and standards for reporting results will be presented. This course is an introductory/intermediate level. Familiarity with economic evaluations will be helpful.

Financial Impact / Cost of Illness
SATURDAY, MAY 15, 1:00 PM - 5:00 PM
Course Description: This course will describe methods to determine the costs associated with a health condition and the budget impact of new technologies for that condition. The course will present incidence- and prevalence-based costing strategies. Treatment algorithms and event-based approaches will be demonstrated for disease-specific costs from different decision-maker perspectives. Both static and dynamic methods for estimating the budget impact of adding a new drug to a health plan formulary will be presented. Issues related to imputing missing data will also be discussed. This course is designed for those with some experience with pharmacoeconomic analysis.

Statistical Considerations in Health Economic Evaluations
Sunday, MAY 16, 8:00 AM - 12:00 PM
Course Description: Adoption and diffusion of new medical treatments depend increasingly on robust analysis of costs and cost-effectiveness. During this course, we discuss design issues for the collection of primary economic data as well as statistical considerations, including the effect of distributional assumptions, univariate and multivariable analyses of data, sample size and power calculations, and estimation of sampling uncertainty for cost-effectiveness analysis. Examples will be provided to illustrate concepts.  Participants should have basic knowledge of economic evaluations and statistics.

NEW! Risk-Sharing/Performance-Based Arrangements for Drugs and Other Medical Products
Sunday, MAY 16, 1:00 PM - 5:00 PM
Course Description: 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.  The 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.  It would be helpful for individuals taking this course 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.Considerations in Economic Evaluations, is a strong prerequisite for this course.

Applications of Statistical Considerations in Health Economic Evaluations
Sunday, MAY 16, 1:00 PM - 5:00 PM
Course Description: This course will provide applications of statistical considerations in economic analysis. Specific exercises will be conducted to illustrate univariate & multivariable analysis of costs (including the effect of  distributional assumptions), sample size and power calculations for cost-effectiveness analysis, and estimation of confidence intervals for cost-effectiveness ratios and net monetary benefits as well as acceptability curves.  Participants are encouraged to have hands-on experience and bring their laptops. Statistical analysis will be done by use of STATA.  A trial version of the software will be distributed for those who do not have the software. The text “Economic Evaluation in Clinical Trials” (Oxford: OUP, 2007) is recommended reading for this course. The course, Statistical Considerations in Economic Evaluations, is a strong prerequisite for this course.

Introduction to Modeling (Introduction to Decision Analysis)
Saturday, MAY 15, 8:00 AM - 12:00 PM
Course 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.

Modeling: Design and Structure of a Model
Saturday, MAY 15, 1:00 PM - 5:00 PM
Course Description: This course will include a review of modeling techniques (Markov models, discrete event simulations, and Monte Carlo techniques) including a discussion of the ISPOR Principles of Good Practice for Decision Analytic Modeling in Health Care Evaluations. Markov models and first and second order Monte Carlo simulations including data identification, data modeling, and data incorporation will be demonstrated. Using a series of examples, the course will carefully review the practical steps involved in developing and using these kinds of models. Examples will be presented using Microsoft Excel, supplemented with add on simulation software. This course will cover the practical steps involved in the selection of models and options in modeling of data inputs. Participants who wish to have hands-on experience should bring their laptops.  Participants should have a basic understanding of decision analysis.  Familiarity with the short course Introduction to Decision Analysis (or equivalent knowledge) is recommended.

Bayesian Analysis – Overview and Applications
Saturday, MAY 15, 8:00 AM - 5:00 PM
Course 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 the free Markov Chain Monte Carlo package WinBUGS.  Attendees will have the chance to apply the principles they have learned in part one 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.

Discrete Event Simulation for Economic Analyses
Sunday, MAY 16, 8:00 AM - 5:00 PM
Course Description: This course will provide a basic understanding of the key concepts of discrete event simulation. The focus will be on the use of these simulation models to address pharmacoeconomic (and device-related) problems. The course will be structured around practical exercises. Topics to be covered are: Why DES? Dynamic simulation as a tool; Components of a DES; How do you build a model? Modeling of processes and resource use; Modeling of variables and decisions. Simple animation will be demonstrated. We will use ARENA to build simple models. Instructors will distribute training versions of Arena. Participants who wish to have hands-on experience should bring their personal laptops.  This course is designed for those with some experience with modeling.

Bayesian Analysis – Advanced
Sunday, MAY 16, 8:00 AM - 12:00 PM
Course Description: This course introduces the use of Bayesian methods in evidence synthesis (including meta-analysis) and allows participants to gain hands-on experience using such modeling techniques within WinBUGS. Methodological issues considered in the course include: fixed & random effects models, choice of prior distributions, subgroups, meta-regression and adjusting for baseline risk, together with indirect and mixed treatment comparisons. Further meta-analysis topics for which a Bayesian approach can be of benefit will also be highlighted. Participants will be expected to be familiar with the use of WinBUGS and will be responsible for bringing a laptop with the latest, unrestricted version of WinBUGS pre-installed [Details at www.mrc-bsu.cam.ac.uk/bugs ]. This course is a follow-up to the course: Bayesian Analysis – Overview and Applications. Basic knowledge of Bayesian approach and use of WinBUGS (equivalent to attendance at Bayesian Analysis-Applications) will be assumed.

Advanced Decision Modeling for Health Economic Evaluations
Sunday, MAY 16, 1:00 PM - 5:00 PM
Course Description: 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 will be presented. 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 including Excel programming will be used to illustrate concepts. Participants should have a basic understanding of decision analysis. The course, Modeling: Design and Structure of a Model, is a strong prerequisite for this course.

Retrospective Database Analysis
Saturday, MAY 15, 8:00 AM - 5:00 PM
Course Description: Large administrative claims databases provide a unique opportunity to examine retrospectively the effects of drug use on clinical and economic outcomes in "real world" settings. This course will cover a discussion of the ISPOR Checklist for Retrospective Database Studies - Report of the ISPOR Task Force on Retrospective Databases and selected topics related to estimators and sampling distributions, properties of sampling distributions (unbiasedness, efficiency, mean square error), and ordinary least squares (OLS) regression. OLS model assumptions and the implications of violations (e.g., heteroscedasticity, multicollinearity, autocorrelation) will also be discussed. More complex topics beginning with the problem of endogeneity, identification, instrumental variables, sample selection models, and propensity score models, maximum likelihood methods and the estimation of limited dependent variables models including logit, multinomial logit, count models, and survival models will be discussed. This course will assume participants have knowledge of statistical methods through OLS regression and experience in the analysis of administrative claims databases.

Instrumental Variables in Addressing Selection Bias in Observational Studies
Saturday, MAY 15, 1:00 PM - 5:00 PM
Course Description: 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, 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.  (For those who have STATA loaded on their laptops, you are encouraged to bring your laptop).  This course is suitable for those with some knowledge of econometrics.

Applications in Using Large Databases
Sunday, MAY 16, 8:00 AM - 12:00 PM
Course Description: This course will provide a review of three health care databases – GPRD (UK database), GE Centricity electronic medical record (USA database) and Medicaid (USA database).  Each database will be discussed in-depth including directions on how to access the information and how researchers utilize this information.  Instructors will distinguish the important differences between these databases including the limitations and strategies to maximize their value through the use of an interactive format with interactive examples, Discussion will include a reference to the ISPOR Classification of Databases Working Group / Retrospective Database Special Interest Group and its digest of international databases. Participants must have some knowledge of administrative health care database analysis.

Patient Registries
Sunday, MAY 16, 8:00 AM - 12:00 PM
Course Description: This course is designed to provide an overview of patient registries and their applications in identifying 'real world' clinical, safety, and patient-perspective issues. The pros and cons of registry data compared to other ‘real world’ and clinical trial data collection will be presented.  How registry information can be used to support other health economics /outcomes research initiatives and health care decision making will be addressed.  Registry strategy, design, operations and measures of program success will be discussed.  In addition, regulatory trends and requirements, including the Agency for Healthcare Research & Quality’s (AHRQ) May 2007 publication: Registries for Evaluating Patient Outcomes: A User's Guide, will be examined.  This course is designed for those with little experience with patient registries.

Outcomes Research for Medical Devices and Diagnostics
Sunday, MAY 16, 1:00 PM - 5:00 PM
Course Description: This course will present outcomes research practices that are specifically tailored for the fast-paced medical device and diagnostics technology environment and address issues related to these health technology assessment methodologies. Outcomes research including clinical outcomes, economic outcomes, and patient-reported outcomes will be discussed. Outcomes research for medical devices and diagnostics will be differentiated from other health care interventions such as drugs. The evidence hierarchy for medical devices and diagnostic procedures including ‘real world’ outcomes research information in coverage and reimbursement decisions will be discussed. This course is designed for those with little experience with outcomes research for medical devices and diagnostic technologies.

NEW! Introduction to Patient-Reported Outcomes
Saturday, MAY 15, 8:00 AM - 12:00 PM
Course Description: Conceptual, methodological, and practical methods for measuring quality of life, health status and other types of health outcomes will be presented. Theoretical frameworks, reliability, validity, responsiveness, methods of administration, respondent and administrative burdens, and issues of analysis and interpretation will be discussed using examples drawn from specific quality-of-life instruments and their applications. A model of selecting appropriate instruments from the many existing generic and disease-specific instruments will be presented.  This is an introductory course intended for those with little experience with these methodologies.

NEW! Introduction to Conjoint Analysis
Saturday, MAY 15, 1:00 PM - 5:00 PM
Course Description: Course participants will learn the conceptual and empirical basis for using conjoint analysis to elicit preferences in outcomes research.  The course will introduce participants to both the conceptual basis for quantifying decision-maker preferences for medical interventions and the practical design and analytical issues that must be addressed in order to obtain valid empirical preference estimates.  The course will be structured following the good research practice guidelines and discussion being prepared by the ISPOR Good Research Practices for the Application of Conjoint Analysis in Health Task Force. The course will include lectures and interactive group exercises and group discussion.  This course is designed for clinicians, policy makers, researchers, patient advocates/researchers with little or no knowledge of conjoint analysis or other stated-preference methods.

Advanced Patient-Reported Outcomes Assessment: Psychometric Methods
Saturday, MAY 15, 1:00 PM - 5:00 PM
Course Description: This course will discuss psychometric analysis and the application of various techniques such as structural equation modeling (SEM), factor analysis (FA) and item response theory (IRT) in testing patient-reported outcomes (PRO) instruments, measures and construct / criterion validity.  Validity indicates how well a measurement tool allows us to infer something about the true nature and value of the object or system being considered.  Instructors will explain and demonstrate how to analyze observed and latent constructs and variables within a model as well as to test the validity of a PRO measure.  Specific examples will be given to highlight how researchers can apply these techniques to test methods, criteria and new measures.  This is an advanced course designed for those with a working knowledge of QoL/PRO methods.

Patient-Reported Outcomes – Item Response Theory
Sunday, MAY 16, 8:00 AM - 12:00 PM
Course 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 workshop will discuss the basics of IRT models and applications of these models to improve health outcomes measurement. Illustrations will be used throughout the presentation that focus on measuring key health-related quality of life domains in different disease populations.  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 introductory course is designed for those with little to no experience with IRT.

Utility Measures
Sunday, MAY 16, 1:00 PM - 5:00 PM
Course 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.  We 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 we will explore the methods that are used to capture utilities such as standard gamble, time trade off and rating scales will be described.  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-6D.  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 and critical appraisal of published values and lastly 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; EU markets such as Sweden/ Belgium/ Netherlands/ Germany; Asia and Latin America.  The course will be interactive with break out sessions and group discussion.  This is an introductory level course; no prior knowledge of utilities or health-related quality of life is assumed.

NEW! Establishing the Content Validity of Patient-Reported Outcome (PRO) Instruments
Sunday, MAY 16, 1:00 PM - 5:00 PM
Course Description: This course will focus on establishing the content validity of patient-reported outcome (PRO) instruments intended for use as the basis for medical product claims in the US and Europe, according to the FDA Guidance for Industry – Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims, and the EMA Reflection Paper on the Regulatory Guidance on the Use of Health-Related Quality of Life (HRQL) Measures in the Evaluation of Medicinal Products.  After this course, participants will be able to: 1) define eight essential requirements for establishing content validity of existing and new PRO instruments; 2) gather evidence to meet these requirements and 3) document the evidence in applications for regulatory approval of desired medical product claims.  Examples will be given throughout on each evidence requirement. Participants will take part in several practical exercises that are part of the iterative process for determining and establishing evidence of content validity for PRO instruments.  Faculty will also reference The ISPOR Good Research Practices for Evaluating and Documenting Content Validity for the Use of Existing Instruments and Their Modifications PRO Task Force Report.  This is an advanced course that assumes attendees have a basic understanding of qualitative interviewing methods and measurement properties of PRO instruments.

Elements of Pharmaceutical/Biotech Pricing I – Introduction
Saturday, MAY 15, 8:00 AM - 12:00 PM
Course 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 will cover topics within a global context.

Case Studies in Pharmaceutical/Biotech Pricing II – Advanced
Sunday, MAY 16, 8:00 AM - 12:00 PM
Course Description: 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 for individuals who have completed Elements of Pharmaceutical Pricing I – Introduction or are familiar with both the key determinants of pharmaceutical pricing and the main international health systems.