E SSENTIALS OF P HARMACOECONOMICS Karen L. Rascati, PhD Eckerd/Turley Centennial Professor of Pharmacy Administration University of Texas College of Pharmacy Austin, Texas Acquisitions Editor:Barrett Koger Managing Editor: Meredith L. Brittain Marketing Manager: Christen D. Murphy Production Editor: Sally Anne Glover Designer: Terry Mallon Compositor: International Typesetting and Composition Copyright © 2009 Lippincott Williams & Wilkins, a Wolters Kluwer business. 351 West Camden Street 530 Walnut Street Baltimore, MD 21201 Philadelphia, PA 19106 Printed in The United States of America. All rights reserved. This book is protected by copyright. No part of this book may be repro- duced or transmitted in any form or by any means, including as photocopies or scanned-in or other electronic copies, or utilized by any information storage and retrieval system without written permission from the copyright owner, except for brief quotations embodied in critical articles and reviews. Materials appearing in this book prepared by individuals as part of their official duties as U.S. government employees are not covered by the above-mentioned copy- right. To request permission, please contact Lippincott Williams & Wilkins at 530 Walnut Street, Philadelphia, PA 19106, via email at permissions 9 8 7 6 5 4 3 2 1 Library of Congress Cataloging-in-Publication Data Rascati, Karen L. Essentials of pharmacoeconomics / Karen L. Rascati. p. ; cm. Includes bibliographical references. ISBN-13: 978-0-7817-6544-2 ISBN-10: 0-7817-6544-7 1. Chemotherapy—Economic aspects. 2. Drugs—Cost effectiveness. 3. Pharmacy—Economic aspects. I. Title. [DNLM: 1. Economics, Pharmaceutical. 2. Costs and Cost Analysis—methods. 3. Models, Economic. 4. Pharmaceutical Preparations—economics. 5. Pharmaceutical Services—economics. 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Visit Lippincott Williams & Wilkins on the Internet: http://www.lww.com Lippincott Williams & Wilkins customer service representatives are available from 8:30 am to 6:00 pm, EST. I dedicate this book to my mother, Glenna Rupp Lewis, whose lifelong support and belief in me helped shape my career choices, first as a pharmacist and then as an academician. Although she is officially retired from her nursing career (and from raising five children), she recently won an award for her work as a hospice volunteer. During the time I was working on this book, she was genuinely interested in its progress, and her encouragement was appreciated more than I can say. I would also like to thank my husband, Joe, and my daughters, Maria and Michelle, for their continued patience and understanding during my preoccupation with this project. P REFACE Need for This Textbook The term “pharmacoeconomics” first appeared in the literature in the mid-1980s. Pharmacoeconomics incorporates methods from more established disciplines to help estimate the value of pharmacy products and services by comparing costs and out- comes. Pharmacoeconomics is included in the curricula of more than 90% of US col- leges of pharmacy, although often as part of a course rather than as a whole course. The Accreditation Council for Pharmacy Education (ACPE) has developed standards and guidelines for professional programs in pharmacy leading to a PharmD degree (adopted in January 2006 and effective as of July 2007); these include an outline of the subject matter that should be included in pharmacy school curricula. The fol- lowing topics are listed in the ACPE document (see http://www.acpe-accredit. org/pdf/ACPE_Revised_PharmD_Standards_Adopted_Jan152006.pdf) under the Economics/Pharmacoeconomics heading: economic principles in relation to pharma- coeconomic analysis, concepts of pharmacoeconomics in relation to patient care, and applications of economic theories and health-related quality of life concepts to improve allocation of limited health care resources. This book covers the application of economic-based evaluation methods to pharmaceutical products and services and includes examples of how pharmacoeconomic evaluations relate to decisions that affect patient care and how health-related quality of life (HRQoL) is assessed and val- ued. Understanding these principles helps providers and decision makers improve clinical and humanistic outcomes based on available resources. Intended Audience Essentials of Pharmacoeconomics is designed for the true beginner—a student or practitioner who may not have even heard of the term “pharmacoeconomics.” The purpose of the book is to introduce the fundamental topics, define the terminology used in pharmacoeconomic research, and give many examples in evaluating pub- lished research so that readers can evaluate literature relevant to their future or cur- rent practice. When readers have completed the book, they should be able to understand, interpret, and determine the usefulness of pharmacoeconomic research articles. This textbook can be used for PharmD students at any level of their professional education. The distinction of this textbook, compared with other textbooks on the subject, is that it is written at an introductory level and provides many practical examples. Another advantage of the book is that it was written by one author. This helps ensure a clear flow in providing the information and an elimination of the unin- tended redundancy sometimes seen in textbooks with multiple authors. Please note that this is not intended to be an economics textbook, nor is it intended for graduate students who would need to know more about the theory of economics v vi PREFACE and the statistics or mathematical computations involved in the analyses. Moreover, the purpose is not to train a future pharmacoeconomics researcher but to give a cur- rent or future practitioner the knowledge and skills needed to understand and use pharmacoeconomics research (conducted by someone else) in his or her decision making. Organizational Philosophy This book follows the outline of a course taught to PharmD students by the author for more than 10 years (and updated annually). Part I (Chapters 1 through 8) cov- ers the basics of pharmacoeconomics. The author recognizes that not all colleges of pharmacy have a whole course devoted to pharmacoeconomics. If this book is used in a course that includes other topics and time allotted to pharmacoeconomics is limited, Part I of the book could stand alone as an introduction that covers the essential topics outlined by ACPE. Part II (Chapters 9 through 14) includes addi- tional topics that are more complex (e.g., modeling) or may be of further interest to the reader (e.g., international use of pharmacoeconomics). Part I: Basic Topics Chapter 1 serves as an introduction, stressing the importance of the use of pharma- coeconomics to compare costs and outcomes of pharmacy interventions. The chap- ter discusses four basic types of pharmacoeconomic research: cost-minimization analysis (CMA), cost-effectiveness analysis (CEA), cost-utility analysis (CUA), and cost-benefit analysis (CBA). All four analyses include the measurement of costs but differ in the methods used to measure outcomes. Chapter 2 covers the measure- ment of costs, and Chapter 3 outlines basic questions readers should ask when evaluating any pharmacoeconomic study. Chapters 4 through 7 expand on each of the four types of studies (CMA, CEA, CUA, and CBA) by reviewing the methods used to measure outcomes for each particular type. Chapter 8 provides an overview of health-related quality of life (HRQoL) measures. Part II: Advanced Topics Chapter 9 outlines the use of decision analysis methods to model costs and outcomes that can be summarized in a single cycle. Chapter 10 explains Markov modeling, which is used for analyzing costs and outcomes that are estimated using multiple cycles. Chapter 11 discusses the advantages and disadvantages of using retrospective databases. Chapter 12 reviews the unique issues involved in evaluating the costs and outcomes associated with pharmacy services. The last two chapters address the use of pharmacoeconomic research in decision making. Chapter 13 summarizes the use of pharmacoeconomic evaluations by decision makers outside of the United States, and Chapter 14 outlines the extent to which pharmacoeconomic data are used to make decisions in the United States, includes barriers to its use, and discusses future issues. Features Essentials of Pharmacoeconomics contains the following elements that are geared toward the pharmacy student: PREFACE vii • Composite research articles that incorporate the positive and negative aspects found in a mix of published research articles. Most chapters contain a compos- ite article. Depending on the classroom time allotted to pharmacoeconomics, these composite articles can be included as part of the didactic portion of the course, or they can be incorporated into small group discussion sections or assigned as homework. (Although topics are taken from actual articles, methods and data have been changed to illustrate points made in the chapter. Medication names in these composite articles are fictitious.) • Key terms are bolded throughout the textbook, and definitions of these terms are summarized in a glossary. Pharmacoeconomics research uses terminology derived from other disciplines, such as economics, that might not be familiar to all readers. • Examplesprovide added information or instances from the literature about the chapter topic and reinforce chapter concepts. Most chapters have at least one example. • Equations readers will need to know are explained using multiple example calculations. • Summaries highlight and reinforce the main points of each chapter. • Questions/Exercisesappear at the end of each chapter so that readers can assess their understanding of key concepts. Instructors can find answers to these prob- lems on the book’s companion website. • References andSuggested Readingsat the end of each chapter provide resources available to the reader for further study on the chapter topic. Student and Instructor Resources Student Resources A Student Resource Center at http://thePoint.lww.com/rascati includes the follow- ing materials: • An Image Bank that contains the figures and tables from the textbook • A sample worksheet that includes the 14 questions you should use when critiquing a research article Instructor Resources In addition to the student resources just listed, an Instructor’s Resource Center at http://thePoint.lww.com/rascati includes the following: • Answers to the questions/exercises found in the text Acknowledgments Three of the chapters in the book are based on previous chapters cowritten with colleagues: Dr. James Wilson (Chapter 1), Dr. Jamie Barner (Chapter 7), and Dr. Gábor Vincze (Ch. 8). Another colleague and good friend, Dr. Carolyn Brown, served as my “sounding board” for some of the ideas for this book. My major advi- sor, Dr. Carole Kimberlin, has long served as one of my role models. Her author- ship of a textbook inspired me to consider this endeavor. viii PREFACE I would like to thank both the professional (PharmD) and graduate students at the University of Texas. Their intelligence and inquisitiveness, plus their insightful questions, keep me “on my toes” and current in the field. I would also like to thank my able Administative Associate, Iris Jennings. I would like to thank Meredith Hodges, Dr. Kenneth Lawson, and Dr. Esmond Nwokeji for proofreading various parts of this book. Their comments were especially helpful. I would also like to thank the people I have met and worked with through the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Dr. Marilyn Dix-Smith has guided this organization to international prominence. When I participate on ISPOR committees or attend ISPOR meetings, I am re-ener- gized by other people with an interest in and a passion for this field of study. And last, but definitely not least, I am indebted to the reviewers for their excel- lent advice on ways to improve on the first drafts and to the editors and staff at Lippincott Williams & Wilkins/Wolters Kluwer. To David Troy, who finally con- vinced me to write this book; to Meredith Brittain, for her gentle prodding that kept me on schedule; to Barrett Koger, who kept the project on track; and to the staff, who transformed my ideas into this polished result.
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