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  • Page extent: 676 pages
  • Size: 247 x 174 mm
  • Weight: 1.663 kg


 (ISBN-13: 9780521838900 | ISBN-10: 0521838908)

Outcomes Assessment in Cancer
Measures, Methods, and Applications

Cancer touches the lives of millions worldwide each year. This is reflected not only in well-publicized mortality statistics but also in the profound – though much more difficult to measure – effects of cancer on the health-related quality of life, economic status, and overall well-being of patients and their families. In 2001, the US National Cancer Institute established the Cancer Outcomes Measurement Working Group to evaluate the state of the science in meas- uring the important and diverse impacts of this disease on individuals and populations. The findings and recommendations of the working group’s 35 internationally recognized members are reported in Outcomes Assessment in Cancer, lucidly written and accessible to both researchers and policy makers in academia, government, and industry. This volume provides the most penetrating yet practical discussion to date of alternative approaches for comprehensively measuring the burden of cancer and the effectiveness of preventive and therapeutic interventions.

Joseph Lipscomb is Professor of Public Health at Emory University (Atlanta, GA, USA) and formerly Chief of the Outcomes Research Branch within the Applied Research Program of the Division of Cancer Control and Population Sciences at the National Cancer Institute (Bethesda, MD, USA).

Carolyn C. Gotay is a professor within the Cancer Research Center of Hawai’i at the University of Hawai’i (Honolulu, HI, USA).

Claire Snyder is an expert in outcomes research in the Outcomes Research Branch within the Applied Research Program of the Division of Cancer Control and Population Sciences at the National Cancer Institute (Bethesda, MD, USA).

Outcomes Assessment in Cancer

Measures, Methods, and Applications


Joseph Lipscomb
Emory University, Atlanta, GA, USA and formerly National Cancer Institute, Bethesda, MD, USA

Carolyn C. Gotay
Cancer Research Center of Hawai’i, University of Hawaii, Honolulu, HI, USA

Claire Snyder
National Cancer Institute, Bethesda, MD, USA

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© Cambridge University Press 2005

Parts of this book are a work of the United States Government
and are not protected by copyright in the United States

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exception and to the provision of relevant collective licensing agreements,
no reproduction of any part may take place without
written permission from Cambridge University Press.

First published 2005

Printed in the United Kingdom at the University Press, Cambridge

Typefaces Utopia 8.5/12 pt. and Dax     System LATEX 2e   [TB]

A catalog record for this book is available from the British Library
Library of Congress Cataloging in Publication data
Outcomes assessment in cancer / edited by Joseph Lipscomb, Carolyn C. Gotay, Claire Snyder.
p.;   cm.
Includes bibliographical references and index.
ISBN (invalid) 052183890 (hardback : alk. paper)
1. Cancer – Treatment – Evaluation. 2. Outcome assessment (Medical care) I. Lipscomb, Joseph, 1948– II. Gotay, Carolyn C., 1951– III. Snyder, Claire, 1973–
[DNLM: 1. Neoplasms – therapy. 2. Health Status Indicators. 3. Quality of Life. 4. Treatment Outcome. QZ 266 O94 2005]
RC270.8.O93 2005
616.99′406 – dc22 2004054539

ISBN 0 521 83890 8 hardback

The publisher has used its best endeavors to ensure that the URLs for external websites referred to in this book are correct and active at the time of going to press. However, the publisher has no responsibility for the websites and can make no guarantee that a site will remain live or that the content is or will remain appropriate.

   Every effort has been made in preparing this book to provide accurate and up-to-date information that is in accord with accepted standards and practice at the time of publication. Nevertheless, the authors, editors, and publisher can make no warranties that the information contained herein is totally free from error, not least because clinical standards are constantly changing through research and regulation. The authors, editors, and publisher therefore disclaim all liability for direct or consequential damages resulting from the use of material contained in this book. Readers are strongly advised to pay careful attention to information provided by the manufacturer of any drugs or equipment that they plan to use.


  List of contributors page ix
  Acknowledgments xiii
1   Introduction to Outcomes Assessment in Cancer 1
  Joseph Lipscomb, Ph.D., Carolyn C. Gotay, Ph.D., and Claire Snyder, M.H.S. (The Editors)
Health-related quality of life in cancer: general concepts and generic measures
2   Definitions and conceptual models of quality of life 14
  Carol Estwing Ferrans, Ph.D., R.N., F.A.A.N.
3   Assessing health status and quality of life of cancer patients: the use of general instruments 31
  Pennifer Erickson, Ph.D.
4   The roles for preference-based measures in support of cancer research and policy 69
  David H. Feeny, Ph.D.
Assessing health-related quality of life during treatment
5   Quality of life in breast cancer: what have we learned and where do we go from here? 93
  Patricia A. Ganz, M.D., and Pamela J. Goodwin, M.D., M.Sc.
6   Measuring quality of life in prostate cancer: progress and challenges 126
  Mark S. Litwin, M.D., M.P.H., and James A. Talcott, M.D., S.M.
7   The science of quality-of-life measurement in lung cancer 160
  Craig C. Earle, M.D., M.Sc., and Jane C. Weeks, M.D.
8   Treatment for colorectal cancer: impact on health-related quality of life 178
  Carol M. Moinpour, Ph.D., and Dawn Provenzale, M.D., M.S.
9   Instruments to measure the specific health impact of surgery, radiation, and chemotherapy on cancer patients 201
  Michael J. Barry, M.D., and Janet E. Dancey, M.D.
Assessing health-related quality of life across the cancer continuum
10   Short-term outcomes of chemoprevention, genetic susceptibility testing, and screening interventions: What are they? How are they measured? When should they be measured? 216
  Jeanne S. Mandelblatt, M.D., M.P.H., and Joe V. Selby, M.D., M.P.H.
11   Evaluating quality of life in cancer survivors 241
  Brad Zebrack, Ph.D., M.S.W., and David Cella, Ph.D.
12   Assessing health-related quality of life at end of life 264
  Betty R. Ferrell, Ph.D., F.A.A.N.
Measuring the experience and needs of cancer patients and caregivers
13   Patient advocate perspective on health-related quality of life issues with prostate cancer survivors 286
  (Col. Ret.) James E. Williams, Jr.
14   Measuring the patient’s perspective on the interpersonal aspects of cancer care 290
  Charles Darby
15   Needs assessment in cancer 305
  David H. Gustafson, Ph.D.
16   Assessing the subjective impact of caregiving on informal caregivers of cancer patients 329
  Claire Snyder, M.H.S.
Methodological considerations in applications to cancer outcomes research
17   Practical considerations in outcomes assessment for clinical trials 346
  Diane L. Fairclough, Dr.P.H.
18   Statistical issues in the application of cancer outcome measures 362
  Jeff A. Sloan, Ph.D.
19   The clinical value and meaning of health-related quality-of-life outcomes in oncology 386
  David Osoba, B.Sc., M.D., F.R.C.P.C.
20   Cross-cultural use of health-related quality of life assessments in clinical oncology 406
  Neil K. Aaronson, Ph.D.
Modern psychometric theory in cancer outcomes research
21   Item response theory and its applications for cancer outcomes measurement 425
  Steven P. Reise, Ph.D.
22   Applications of item response theory to improve health outcomes assessment: developing item banks, linking instruments, and computer-adaptive testing 445
  Ronald K. Hambleton, Ph.D.
23   Subscales and summary scales: issues in health-related outcomes 465
  Mark Wilson, Ph.D.
Assessing the economic impact of cancer
24   On the definition and measurement of the economic burden of cancer 480
  Mark C. Hornbrook, Ph.D.
25   Cost-effectiveness analysis in cancer: toward an iterative framework for integration of evidence from trials and models 503
  Bernie J. O’Brien, Ph.D.
Research and policy implications
26   Data for cancer outcomes research: identifying and strengthening the empirical base 522
  Carolyn C. Gotay, Ph.D., and Joseph Lipscomb, Ph.D.
27   Use of health-related quality of life measures by industry and regulatory agencies in evaluating oncology therapies 550
  Dennis A. Revicki, Ph.D.
28   Reflections on COMWG findings and moving to the next phase 568
  Carolyn C. Gotay, Ph.D., Joseph Lipscomb, Ph.D., and Claire Snyder, M.H.S.
Invited papers
  Invited Paper A The world of outcomes research: yesterday, today, and tomorrow 584
  Bert Spilker, Ph.D., M.D.
  Invited Paper B The ten Ds of health outcomes measurement for the twenty-first century 590
  Colleen A. McHorney, Ph.D., and Karon F. Cook, Ph.D.
  Invited Paper C The use of cognitive interviewing techniques in quality of life and patient-reported outcomes assessment 610
  Gordon B. Willis, Ph.D., Bryce B. Reeve, Ph.D., and Ivan Barofsky, Ph.D.
  Invited Paper D Industry perspective regarding outcomes research in oncology 623
  Kati Copley-Merriman, M.S., M.B.A., Joseph Jackson, Ph.D., J. Gregory Boyer, Ph.D., Joseph C. Cappelleri, Ph.D., M.P.H., Robert DeMarinis, Ph.D., Joseph DiCesare, M.P.H., R.Ph., M. Haim Erder, Ph.D., Jean Paul Gagnon, Ph.D., Lou Garrison, Ph.D., Kathleen Gondek, Ph.D., Kim A. Heithoff, Sc.D., Tom Hughes, Ph.D., David Miller, Ph.D., Margaret Rothman, Ph.D., Nancy Santanello, M.D., M.S., Richard Willke, Ph.D., and Bruce Wong, M.D.
  Index 639


Members of the Cancer Outcomes Measurement Working Group (2001–2004)


Joseph Lipscomb, Ph.D.
Chief, Outcomes Research Branch
Applied Research Program
Division of Cancer Control and Population Sciences
National Cancer Institute

Carolyn C. Gotay, Ph.D.
Professor, Cancer Research Center of Hawai’i
University of Hawaii

Working Group Initiator

Claire Snyder, M.H.S
Expert, Outcomes Research Branch
Applied Research Program
Division of Cancer Control and Population Sciences
National Cancer Institute

Working Group Participants

Neil K. Aaronson, Ph.D.
Head, Division of Psychosocial Research &
The Netherlands Cancer Institute
Professor, Faculty of Medicine
Vrije Universiteit

Michael J. Barry, M.D.
Chief, General Medicine Unit
Massachusetts General Hospital

David Cella, Ph.D.
Professor of Psychiatry and Behavioral Science
Northwestern University Feinberg School
of Medicine
Director, Center on Outcomes Research
and Education
Evanston Northwestern Healthcare

Janet E. Dancey, M.D.
Senior Clinical Investigator, Investigational Drug
Branch, Cancer Therapy Evaluation Program
Division of Cancer Treatment and Diagnosis
National Cancer Institute

Charles Darby
Social Science Administrator
Agency for Healthcare Research and Quality

Craig C. Earle, M.D., M.Sc.
Assistant Professor of Medicine, Harvard
Medical School
Dana-Farber Cancer Institute

Pennifer Erickson, Ph.D.
Associate Professor, Departments of Biobehavioral
Health and Health Evaluation Sciences
Pennsylvania State University

Diane L. Fairclough, Dr.P.H.
Professor, Colorado Health Outcomes Center and
Department of Preventive Medicine and Biometry
University of Colorado Health Sciences Center

David H. Feeny, Ph.D.
Professor of Pharmacy and Pharmaceutical
Institute for Health Care Economics
and Departments of Economics
and Public Health Sciences
University of Alberta

Carol Estwing Ferrans, Ph.D., R.N., F.A.A.N.
Professor, College of Nursing
University of Illinois at Chicago

Betty R. Ferrell, Ph.D., F.A.A.N.
Research Scientist
City of Hope Medical Center

Patricia A. Ganz, M.D.
Professor, Schools of Medicine and Public Health
Director, Division of Cancer Prevention
and Control Research
Jonsson Comprehensive Cancer Center
University of California, Los Angeles

Pamela J. Goodwin, M.D., M.Sc., F.R.C.P. (C)
Senior Scientist, Samuel Lunenfeld Research
Institute, Mount Sinai Hospital
Professor of Medicine, University of Toronto

David H. Gustafson, Ph.D.
Robert Ratner Professor of Industrial Engineering
Director, Center of Excellence in Cancer
Communications Research
University of Wisconsin, Madison

Ronald K. Hambleton, Ph.D.
Distinguished University Professor, School
of Education
University of Massachusetts

Mark C. Hornbrook, Ph.D.
Chief Scientist, Center for Health Research,
Northwest and Hawaii
Kaiser Permanente, Northwest Region

Mark S. Litwin, M.D., M.P.H.
Professor of Urology and Health Services
Schools of Medicine and Public Health
University of California, Los Angeles

Jeanne S. Mandelblatt, M.D., M.P.H.
Director, Cancer & Aging Research
Lombardi Comprehensive Cancer Center
and Departments of Oncology and Medicine
Georgetown University Medical Center

Mary S. McCabe, R.N., M.A.
Director, Office of Education and Special
National Cancer Institute

Carol M. Moinpour, Ph.D.
Behavioral Scientist
Southwest Oncology Group Statistical Center
Associate Member, Division of Public
Health Sciences
Fred Hutchinson Cancer Research Center

Bernie J. O'Brien, Ph.D.
Professor, Department of Clinical Epidemiology
and Biostatistics
McMaster University
Associate Director, Centre for Evaluation of
St. Joseph’s Healthcare

David Osoba, B.Sc., M.D., F.R.C.P.C.
Quality of Life Consultant
QOL Consulting, West Vancouver, BC

Dawn Provenzale, M.D., M.S.
Associate Professor of Medicine and
Director GI Outcomes Research
Duke University Medical Center

Steven P. Reise, Ph.D.
Professor, Department of Psychology
University of California, Los Angeles

Dennis A. Revicki, Ph.D.
Vice President and Director, Center for
Health Outcomes Research
MEDTAP International

Joe V. Selby, M.D., M.P.H.
Director, Division of Research
Kaiser Permanente Northern California

Jeff A. Sloan, Ph.D.
Lead Statistician, Cancer Center Statistics
Mayo Clinic, Rochester (MN)

James A. Talcott, M.D., S.M.
Assistant Professor and Director, Center
for Medical Outcomes
Massachusetts General Hospital

Jane C. Weeks, M.D., M.Sc.
Associate Professor of Medicine
Chief, Division of Population Science
Dana-Farber Cancer Institute

James E. Williams, Jr. (Col. Ret.) USA
Co-Chairman, Pennsylvania Prostate
Cancer Coalition
Vice President, Intercultural Cancer
Council Caucus

Mark Wilson, Ph.D.
Professor, Graduate School of Education
University of California, Berkeley

Brad Zebrack, Ph.D., M.S.W.
Cancer Survivor/Advocate
NCI Director's Consumer Liaison Group
Assistant Professor, School of Social Work
University of Southern California

Authors of invited papers

Ivan Barofsky, Ph.D.
The Quality of Life Institute, East Sandwich, MA

J. Gregory Boyer, Ph.D.
Assistant Executive Director, Accreditation
Evaluation and Research
The Accreditation Council for Pharmacy Education

Joseph C. Cappelleri, Ph.D., M.P.H.
Director, Biostatistics
Pfizer, Inc.

Karon F. Cook, Ph.D.
Associate Director for Research, Parkinson’s Disease, Research & Education Center
Houston Veterans Affairs Medical Center
Assistant Professor, Department of Neurology
Baylor College of Medicine

Kati Copley-Merriman, M.S., M.B.A.
Senior Director/Site Leader, Global Outcomes
Pfizer, Inc.

Robert M. DeMarinis, Ph.D.
Assistant Vice President, Global Health Outcomes Assessment
Wyeth Research

Joseph DiCesare, M.P.H., R.Ph.
Executive Director, Health Economics &
Outcomes Research
Novartis Pharmaceuticals Corporation

M. Haim Erder, Ph.D.
Director, Health Economics & Epidemiology
Amgen Inc.

Jean Paul Gagnon, Ph.D.
Director, Public Policy
Aventis Pharmaceuticals Inc.

Lou Garrison, Ph.D.
Vice President and Head, Health Economics
& Strategic Pricing
F. Hoffmann-La Roche AG.

Kathleen Gondek, Ph.D.
Director, Health Economics and Outcomes
Bayer Corporation

Kim Allen Heithoff, Sc.D.
Director, Health Economics
Schering-Plough Pharmaceuticals

Tom Hughes, Ph.D.
Director, Health Outcomes
Global Economic Affairs
Eli Lilly and Company

Joseph D. Jackson, Ph.D.
Group Director, Outcomes Research
Bristol-Myers Squibb

Colleen A. McHorney, Ph.D.
Professor of Medicine
Indiana University School of Medicine
and Regenstrief Institute for Health Care;
Research Career Scientist and Senior Scientist
Roudebush Veterans Affairs Medical Center

David W. Miller, Ph.D.
Vice President, Global Health Outcomes

Bryce B. Reeve, Ph.D.
Psychometrician, Outcomes Research Branch
Applied Research Program
Division of Cancer Control and Population Sciences
National Cancer Institute

Margaret L. Rothman, Ph.D.
Executive Director, HE&P, PGSM
Johnson & Johnson Pharmaceutical Services, LLC

Nancy Santanello, M.D., M.S.
Executive Director, Epidemiology
Merck Research Laboratories

Bert Spilker, Ph.D., M.D.
Bert Spilker & Associates, LLC

Richard Willke, Ph.D.
Senior Director/Group Leader, OR Product
Development Group B
Worldwide Outcomes Research
Pfizer, Inc.

Gordon B. Willis, Ph.D.
Cognitive Psychologist, Applied Research Program
Division of Cancer Control and Population Sciences
National Cancer Institute

Bruce Wong, M.D.
Vice President, Global Outcomes Research
Bristol-Myers Squibb


This volume reflects the contributions of a host of individuals who devoted time, intellectual energy, and substantive expertise towards the common goal of better understanding how to assess the outcomes of cancer and cancer care. While we mention only a few names below, we are extremely grateful to everyone who provided support and scientific guidance to the Cancer Outcomes Measurement Working Group (COMWG), established in early 2001 by the US National Cancer Institute (NCI) and whose work continued well into 2004.

   First and foremost, we salute the members of the COMWG. Outcomes Assessment in Cancer is the product of their hard work, always under tight deadlines and sometimes under difficult circumstances. We also heartily thank the authors of the invited chapters, whose contributions fill important gaps in the working group’s coverage of topics to create a stronger overall product.

   We note that the work of each COMWG member who was not a US government employee was supported under an NCI contract; participation by Federal members was regarded as an “official duty” activity. The National Cancer Institute also provided limited financial support for research assistance for any COMWG member requesting it. Non-Federal authors of the invited chapters were likewise supported under NCI contract, except the members of the PhRMA Health Outcomes Committee, whose efforts were contributed. Consistent with the operating principles of an NCI-approved working group (see Chapter 1), the views expressed in each chapter are solely those of the author(s) and are not intended to reflect working group opinions or consensus positions. Chapters authored by one or more Federal employees were subject to internal clearance review by each author’s agency to evaluate factual accuracy and editorial quality. These internal review policies applied to both working group and invited chapters.

   Staff scientists in NCI’s Outcomes Research Branch supported the working group’s operations in a number of important ways. Most especially, we acknowledge the invaluable contributions of Bryce Reeve, Ph.D., a psychometrician who provided expert review and commentary on chapters dealing with application of modern measurement concepts like item response theory to improve cancer outcomes assessment. Similarly, Neeraj Arora, Ph.D., offered important perspectives on chapters examining individuals’ perceptions about cancer care and approaches to assessing patient needs.

   Created as one component of the NCI-wide initiative to improve the quality of cancer care, the COMWG’s administrative home was NCI’s Division of Cancer Control and Population Sciences (DCCPS). The project began under the leadership of DCCPS Director Barbara Rimer, Dr.P.H., and Deputy Director Robert Hiatt, M.D., Ph.D., and continued to receive unfailing support and sound guidance from Robert Croyle, Ph.D., when he became DCCPS Director in 2003. Throughout, valuable substantive direction and editorial advice was provided by Rachel Ballard-Barbash, M.D., Associate Director Applied Research, DCCPS.

   In the course of the COMWG’s three on-site meetings, there were stimulating presentations by Robert Wittes, M.D., NCI Deputy Director; Laurie B. Burke, R.Ph., M.P.H., Director, Endpoints and Labeling Development at the US Food and Drug Administration; Julia Rowland, Ph.D., Director of the NCI Office of Cancer Survivorship; and also members of the working group and invited chapter authors.

   Two contractors provided critically important assistance. The late Ellen Tobin, President of Cancer Care Strategies, conducted a number of focus groups to deepen the COMWG’s understanding of how cancer patients and survivors perceive and cope with this disease and its aftermath. Jane Kolimaga worked rapidly, tirelessly, and with unerring accuracy in copy-editing the book’s chapters.

   We note with great sadness the untimely passing in early 2004 of COMWG member Bernie O’Brien, Ph.D., an internationally recognized health economist whose contributions to the profession are well exemplified by, but extend far beyond, the superb chapter he produced for this volume.

   Over the months since the COMWG began, cancer touched our lives through family, friends, and colleagues. We have seen first-hand the impact on patients and their loved ones. It is our sincere hope that the work presented here will stimulate additional research and application to improve the lives of all those affected by cancer

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