SUNDAY, 21 OCTOBER 2007 - MORNING COURSES (8:00 - 12:00)

Case Studies in Pharmaceutical/Biotech Pricing II - Advanced
Faculty: Jack Mycka, President & Partner, MME 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.

Health Care and Reimbursement Systems in Europe
Faculty: Christian Gericke MD, MPH, MSc, Professor and Chair in Public Health Policy, The University of Adelaide, Adelaide, Australia
Course Description: This course will describe and compare various European health care systems, and will include information on health care financing and resource allocation mechanisms. Various issues encountered when defining benefit packages or introducing new healthcare technology will be discussed. The course will also cover newer developments in European health care system organization and management, such as provision and purchaser-provider splits. This includes a discussion of the current role of health technology assessment and health economic analysis for reimbursement decisions in Europe. Participants will have the possibility to apply the taught course contents in a country-specific case study. This course is designed for those with little experience with health care systems and reimbursement in Europe.

Transferability of Cost-Effectiveness Data between Countries
Faculty: Michael Drummond PhD, Professor of Health Economics, University of York, York, UK; Andrea Manca PhD, MSc, Wellcome Trust Fellow in Health Services Research, Centre for Health Economics, University of York, York, UK; Marco Barbieri MSc, Associate Lecturer and Research Associate, University Pompeu Fabra and i3 innovus Research (UK) Ltd, Barcelona, Spain.
Course Description: Although the number of countries requiring an economic dossier as part of the submission dossier for public reimbursement of new drugs is growing, the pharmaceutical industry cannot conduct economic evaluations in every potential market. Clinical trials are increasingly done in international settings in order to quickly recruit a sufficient number of patients and to have at least some economic data from multiple countries. However, national decision makers require country-specific or region-specific data on health care costs and are only willing to accept foreign data or international data when they are translated to their own specific setting. But little guidance on how to do this exists. This course starts with a discussion of factors that make economic data more difficult to transfer from one country to other countries than clinical data, and the evidence on the variability of cost-effectiveness results across countries. Then we will review the methods that have been presented to offer a solution to this problem and their pros and cons. Finally, we will discuss the emerging international guidance for dealing with issues of transferability, including the suggestions made by the ISPOR Good Practices Task Force on this topic. The methods to be discussed include various types of regression based approaches and Markov models. This course is for those with basic understanding of cost calculation and modeling.

Cost-Effectiveness Analysis alongside Clinical Trial
Faculty: Scott Ramsey MD, PhD, Full Member & Professor, 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; Sean Sullivan PhD, RPh, MS, Professor and Director, University of Washington, Pharmaceutical Outcomes Research and Policy Program, Seattle, WA, 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.

Pharmacoeconomic Modeling - Advanced
Faculty: Uwe Siebert MD, MPH, MSc, ScD, Professor of Public Health; Head of the Department of Public Health, Medical Decision Making and Health Technology Assessment, University of Health Sciences, Medical Informatics and Technology, Hall/Innsbruck, Austria, and Associate Professor of Radiology, Harvard Medical School, Director of the Cardiovascular Research Program, Institute for Technology Assessment, Massachusetts General Hospital, Boston, MA, USA; Alexander Göhler MD, MSc, MPH, Instructor, MGH Institute for Technology Assessment, Harvard Medical School, Boston, MA, USA; UMIT, Hall/Innsbruck, Austria.
Course Description: This course will provide an in-depth look at modeling techniques and their application in a “real world” setting considering the ISPOR Principles of Good Practice for Decision Analytic Modeling in Health Care Evaluations. Using a series of related examples, we will carefully review the practical steps involved in developing and using pharmacoeconomic models. This course will cover the selection and modeling of data inputs and practical aspects related to the determination of when, why and how to use deterministic, first order and second order Monte Carlo simulation techniques. We will give an introduction to Value of Information (VOI) Analysis and discuss important biases in decision modeling. Participants will have the opportunity to apply the discussed methods in interactive discussions and exercises. This course is intended as a follow-up to the short course, “Pharmacoeconomic Modeling”, and is designed for those with intermediate to advanced knowledge of modeling methods.

Decision Analytic Modeling
Faculty: Donald Chalfin MD, MS, Chief Medical Officer, Analytica International & Associate Professor of Medicine, Epidemiology, and Population Medicine, Albert Einstein College of Medicine & Attending Intensivist/Director CCM Outcomes Research, Montefiore Medical Center, New York, NY, USA
Course Description: This course will provide a forum for hands-on application of modeling methods through the application of various software programs. Computer-assisted decision analytic modeling including design of decision trees, sensitivity analysis, Markov, Bayesian, and run-time models will be presented and discussed in an interactive environment. This course is designed for individuals with some familiarity with basic decision analytic modeling. Students should come prepared to use their personal laptops.

Analysis and Interpretation of Quality of Life / Patient-Reported Outcomes
Faculty: Bruce Crawford MPH, MA, Operations Director - PRO and Regulatory, Mapi Values, Boston, MA, USA; Kathleen Rosa MS, PhD, Director, Psychometrics and Statistics, Mapi Values, Boston, MA, USA
Course Description: This course is designed to provide a range of methods that may help to solve common problems encountered with patient-reported outcomes (PROs). Often, many of those charged with interpretation of PRO results have little experience in doing so. This course will provide discussion of 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 PRO data. Topics discussed will include: an overview of psychometric validation, pragmatic issues in validating a PRO within a clinical trial, ePRO validation, methods of estimation of minimally clinically important differences and alternatives to provide information on interpretation, missing data analysis techniques and mixed modeling appropriate to PRO data and study design. There will be 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. This course is designed for those with limited to intermediate experience with PROs.