Faculty: Scott Ramsey MD, PhD, Associate Member, Fred Hutchinson Cancer Research Center, Seattle, WA, USA; Richard Willke PhD, Senior Director, Group Leader, Global Outcome Research, Worldwide Outcomes Research, US Development Sites Pfizer, Inc., Bridgewater, NJ, USA

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, and 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
 

Faculty: Diana Brixner PhD, RPh, Associate Professor and Department Chair, College of Pharmacy, University of Utah, Salt Lake City, UT; Gregory de Lissovoy, MPH, Senior Research Scientist, Center for Health Economics and Policy, United BioSource Corporation, Bethesda, MD,USA; Daniel M. Huse MA, Practice Leader, Information Products, Thomson Medstat Inc., Cambridge, MA

Course Description: ‘Real world’ data is defined as information (data) collected beyond that which is normally collected in Phase III clinical trials that focus on efficacy. This course will address the issues and framework for analysis of ‘real world’ data in outcomes research. The types of ‘real world’ data (piggy-back information from Phase III clinical trials, large simple trials, registries, administrative claims databases, surveys) and benefits and challenges of these data, as well as evidence hierarchies and their usefulness will be discussed. Examples of the use of ‘real world’ data for different types of outcomes (clinical outcomes, economic outcomes, and quality of life / patient-reported outcomes) will be presented. Health care payer’s perspectives will also be addressed. This course is designed for those with little experience with ‘real world’ data assessment and use in health care decisions.
 

Faculty:  Bryan Luce MBA, PhD, Senior Vice President, Science Policy, United BioSource Corporation, Bethesda, MD, USA; Christopher S. Hollenbeak PhD, Surgery and Health Evaluation Sciences, Penn State College of Medicine, Hershey, PA, USA; David Vanness PhD, Assistant Professor of Population Health Sciences, University of Wisconsin Medical School, Madison, WI, USA; Keith R. Abrams PhD, Department of Health Sciences, University of Leicester, England, U.K.

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 modelling 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 courses: Bayesian Analysis-Overview and Bayesian Analysis-Applications. Basic knowledge of Bayesian approach and use of WinBUGS (equivalent to attendance at Bayesian Analysis-Applications) will be assumed.
 

Faculty: J. Jaime Caro MDCM, FCRPC, FACP, Adjunct Professor of Medicine, Adjunct Professor of Epidemiology and Biostatistics, McGill University, Montreal PQ and Scientific Director, Caro Research Institute, Concord, MA, USA; Jörgen Möller MSc Mech Eng, Simulation Specialist, Caro Research Institute, Concord, MA, USA

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. If time permits, simple animation will be demonstrated. We will use ARENA to build simple models. Participants who wish to have hands-on experience should bring their laptops. Instructors will distribute training versions of Arena. This course is designed for those with some experience with modeling.
 

Faculty: Bruce Crawford MPH, MA, Director, Patient Reported Outcomes and Regulatory Consulting-Operations Director, Mapi Values, Boston, MA, USA; Kathleen Rosa, PhD, Director of Psychometrics and Statistics, Mapi Values, Boston, MA, USA

Course Description: This course will provide an in-depth discussion of operating characteristics, validity testing, analysis and interpretation with examples of each. It will provide a range of methods that may help to solve common problems encountered with quality of life / patient-reported outcomes. These include an overview of psychometric validation methods including: a brief overview of Rasch analysis, pragmatic issues in validating a PRO from clinical trial data, ePRO validation, methods of estimation of minimally clinically important differences and alternatives to provide information on interpretation Clinical trial analysis will include missing data analysis techniques and mixed modelling appropriate to PRO data and study design, There will be a focus on addressing these issues within the framework provided by the PRO guidance recently released by the SEALD group at the FDA. Specific examples will be used throughout the course and participants will be asked to complete a short exercise. This course is designed for those with intermediate experience in health-related quality-of-life assessment.
 

Faculty: Jack Mycka, President, Optimar Strategic Consulting LLC, Montclair, NJ, USA;
Renato Dellamano PhD, President, ValueVector (Value Added Business Strategies), Milan, Italy

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.
 

Faculty: Henry Glick PhD, University of Pennsylvania, School of Medicine, Philadelphia, PA, USA;
Jalpa Doshi PhD, University of Pennsylvania, School of Medicine, Philadelphia, PA, USA;
Daniel Polsky PhD, University of Pennsylvania, School of Medicine, Philadelphia, PA, USA

Course Description: The adoption and diffusion of new medical treatments depend increasingly on evidence of costs and cost effectiveness. This evidence is increasingly being generated from patient level data in randomized study designs. This course will discuss design and analysis issues that arise when conducting such analyses. Specifically, we will address topics on strategic issues in the design of economic assessments, sample size and power calculations, analysis of costs and how it is affected by distributional assumptions, and assessing stochastic uncertainty. The course will be practical in orientation and will routinely provide examples to illustrate the "how-to's". This course is an introductory/intermediate level. Familiarity with economics and statistics will be helpful.
 

Faculty: Fadia Shaya MPH, PhD, Assistant Professor/Associate Director, University of Maryland School of Pharmacy, Center on Drugs and Public Policy, Baltimore, MD, USA

Course Description: A large part of the evidence about the effectiveness of different treatments is based on retrospective studies. Issues of bias and confounding relate to the non-random assignment of subjects and co-morbidity burden. This course will outline the concerns about bias and explain the methods for causal inference in observational studies, where researchers have no control over the treatment assignment. A lack of balance in the covariates between the treatment and control groups can produce biased estimates of the treatment effects. We will explain how propensity scores can be used to reduce bias, through stratification, matching or regression. Confounding and the pros and cons of standard adjustment, propensity scoring methodology (sub classification on one confounding variable, overlap in treatment groups, variable selection) will be discussed. In the second part, we will elaborate on risk adjustment models, focusing on morbidity indices, e.g the Charlson Comorbidity Index, and Chronic Disease Scores. Examples using a step by step approach will be presented. This is an introductory course, designed for those with little experience with this methodology but some knowledge of observational databases.
 

Faculty: Jeff Trotter MBA, President, Ovation Research Group, Highland Park, IL, USA

Course Description: This course is designed to provide an overview of patient registries and its applications in identifying 'real world' clinical, safety, and patient-perspective issues. The advantages and disadvantages of patient registry versus other “real world’ data collection will be presented. The course will address safety and clinical objectives, as well as regulatory trends and requirements. Key operational components and challenges, and measures of program success will be discussed. Management issues, including creating effective partnerships with patient-oriented organizations and facilitating long-term program operations within a changing organizational structure will be addressed. This course is designed for those with some or no experience with patient registries.
 

Faculty: F. Reed Johnson PhD, Senior Fellow and Principal Economist, RTI Health Solutions, Research Triangle Park, NC, USA; A. Brett Hauber PhD, Senior Director of Health Economics, RTI Health Solutions, Research Triangle Park, NC, USA

Course Description: Course participants will learn the conceptual and empirical features of various health-utility measures and their relative advantages for different health care decisions. This course evaluates new methods for bridging the gap between ordinal and cardinal utility measures. Newer methods allow analysts to estimate “super QALY” values using time or other non-monetary tradeoffs that do not require the restrictive assumptions of conventional cardinal-utility methods. The course focuses particularly on how to derive utility estimates from surveys, including developing valid and reliable tradeoff surveys and analyzing the resulting data. The uses of utility assessment involving individual decisions versus population resource allocations will be compared. This course is designed for those with some experience with psychometric measures.
 

Faculty: Seema Sonnad PhD, Associate Professor, Department of Surgery, University of Pennsylvania, Philadelphia, PA, USA; Stacey Ackerman, MSE, PhD,  Vice President, Covance Market Access Services, San Diego, CA, USA

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 & 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.
 

Faculty: Jack Mycka, President, Optimar Strategic Consulting LLC, Montclair, NJ, USA;
Renato Dellamano PhD, President, ValueVector (Value Added Business Strategies), Milan, Italy

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 PE 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.
 

Risk Assessment: Analysis and Management

Faculty: Dennis W. Raisch, PhD, Associate Center Director, Scientific Affairs, VA Cooperative Studies Program, Clinical Research Pharmacy, Albuquerque, NM, Anthony Lockett, MD, PhD, MBA, Medical Director, ICO, Leeds, United Kingdom, Suellen Curkendall, PhD, Principal Investigator, Cerner Health Insights, Vienna, VA

Course Description: This course will provide an overview of risk management for pharmaceuticals and devices. The risk/benefit assessment process will be described in regards to stage of product development, from pre-marketing through post-marketing. Risk mitigation includes the various strategies employed by manufacturers, regulators, and health care providers, with an emphasis on international differences in risk mitigation and decision making. Risk/benefit communication processes will be described, focusing upon how decisions regarding risk of pharmaceuticals and devices are communicated to health care providers and the public. This includes direct mailing, direct-to-consumer marketing, and labeling. Real-world exercises will allow participants to discuss key topics and propose implementation strategies for risk management. This course is designed for those with a basic understanding of pharmacoepidemiology principles.