PHARMACOECONOMIC / ECONOMIC METHODS

Pharmacoeconomics for Decision-Makers

Faculty: Lorne Basskin PhD, Director, Pharmacy Services, Healthsouth Sunrise Rehab Hospital, Cooper City, FL, USA

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.

MODELING METHODS

Bayesian Analysis: Overview & Applications

Faculty: Bryan Luce MBA, PhD, Senior Vice President, Science Policy, United BioSource Corporation, Bethesda, MD, USA; Christopher S. Hollenbeak PhD, Assistant Professor, 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

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 will introduce available statistical packages. The second portion of this course will focus on the Bayesian “informative prior.” Several example vignettes of how a Bayesian analysis can be used within outcomes modeling problems will be presented. Participants will learn how a Bayesian approach is different, why it is useful for their work and what tools are available to them. Participants of this course should be prepared to use their own laptops as the exercises presented use interactive software. This course is designed for those with a limited understanding of Bayesian statistical concepts.

PHARMACOECONOMIC / ECONOMIC METHODS

Finding and Extracting Cost Data

Faculty: Gregory de Lissovoy MPH, PhD, Vice-President, United BioSource Corporation, Bethesda, MD, USA; L. Clark Paramore MSPH, Executive Director and Research Scientist, Center for Health Economics, Epidemiology & Science Policy, United BioSource Corporation, Medford, MA, USA

Course Description: This course will focus on practical aspects of cost development for pharmacoeconomic studies. The objective is to help the participant bridge the gap between understanding pharmacoeconomic theory and the practice of developing cost estimates. Factors to consider when costing pharmacoeconomic analyses, such as perspective, data sources, data classification systems, developing resource use profiles, obtaining unit costs, and making cost adjustments will be presented. Examples of issues encountered when identifying and extracting cost data will be discussed. This course is designed for those with some experience with pharmacoeconomic analysis.

MODELING METHODS

Modeling: Design and Structure of a Model

Faculty: Marc Botteman MA, Managing Partner and Director of Health Economics, PharMerit North America LLC, Bethesda, MD, USA; Ben van Hout PhD, Scientific Director, PharMerit, Rotterdam, The Netherlands and Professor in Medical Technology Assessment, Julius Centre for Health Sciences and Primary Care, University Medical Centre Utrecht, Utrecht, The Netherlands

Course Description: This course will include a review of Markov models, discrete event models, and other modeling techniques and their appro­priate usage. Using a series of related examples, the course will carefully review the practical steps involved in developing and using these kinds of models. This intermediate course requires a basic understanding of decision analysis.

REAL WORLD DATA METHODS

Applications in Using Large Databases

Faculty: Diana Brixner PhD, RPh, Professor and Chair, University of Utah, College of Pharmacy, Department of Pharmacotherapy, Salt Lake City, UT, USA; John Parkinson PhD, Head of GPRD, London, UK; Michael Eaddy PhD, PharmD, Senior Director, Xcenda, Palm Harbor, FL, USA

Course Description:This course will provide a review of 3 health care databases – GPRD (UK database), GE Centricity electronic medical record and Medicare (USA databases).  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 Database Working Group / Retrospective Database Special Interest Group and its digest of International Databases. Participants must have some knowledge of administrative health care database analysis.

SATURDAY, MAY 3, 2008 (Afternoon Courses)
1:00 PM - 5:00 PM

PHARMACOECONOMIC / ECONOMIC METHODS

Financial Impact / Cost of Illness

Faculty: Josephine Mauskopf PhD, Vice President, Health Economics, RTI Health Solutions, Research Triangle Park, NC, USA; C. Daniel Mullins PhD, Professor and Chair, Pharmaceutical Health Services Research, University of Maryland, School of Pharmacy, Baltimore, MD, USA

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.

Cost-Effectiveness Analysis Alongside Clinical Trials

Faculty: Scott Ramsey MD, PhD, Full Member and Professor, Fred Hutchinson Cancer Research Center, Seattle, WA, USA; Richard Willke PhD, Senior Director, Group Leader, Global Outcomes Research, Pfizer, Inc., Bridgewater, NJ, USA; Sean D. 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, characterization of uncertainty and standards for reporting results will be presented. This is an introductory/intermediate level course. Familiarity with economic evaluations will be helpful.

QUALITY OF LIFE / PATIENT-REPORTED OUTCOMES METHODS

Advanced Quantitative Methods for Quality of Life / Patient-Reported Outcomes

Faculty: Bruce Crawford MA, MPH, General Manager, Asia, Mapi Values, Meguro, Tokyo, Japan; Kathleen Rosa MS, PhD, Director, Psychometrics and Statistics, Mapi Values, Boston, MA, USA; Jeffrey McDonald MS, Statistician, 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 modeling 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.

Instrumental Variables in Addressing Selection Bias in Observational Studies

Faculty: Benjamin M Craig PhD, Assistant Professor, Oncologic Sciences and Economics, University Of South Florida & Assistant Member, Moffitt Cancer Center, Tampa, FL, USA; Antoine C. El Khoury, PhD, Manager, Outcomes Research, Merck & Co Inc., West Point, PA, USA, & Adjunct Assistant Professor, Division of Pharmaceutical Evaluation and Policy, University of Arkansas for Medical Sciences College of Pharmacy, Little Rock, AR, USA; Bradley Martin PhD, RPh, PharmD, Professor and Division Chair, College of Pharmacy, University of Arkansas for Medical Sciences, Department of Pharmacy Practice, Little Rock, AR, USA

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.

USE OF PHARMACOECONOMICS/ ECONOMIC / OUTCOMES RESEARCH INFORMATION

Elements of Pharmaceutical/Biotech Pricing I - Introduction

Faculty: Jack Mycka, President and Partner, MME 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.