Published by Oxford University Press 2007.
Introduction
Affiliation of authors: Community Oncology and Prevention Trials Research Group, Division of Cancer Prevention, National Cancer Institute, National Institutes of Health, Bethesda, MD
Correspondence to: Lori Minasian, MD, National Cancer Institute, 6130 Executive Blvd, MSC-7340, Bethesda, MD 20892-7340 (e-mail: minasilo{at}mail.nih.gov).
 |
INTRODUCTION |
|---|
 Top Introduction Assessing Quality of Life...NotesReferences |
|---|
Over the past 30 years, National Cancer Institute (NCI) has supported quality of life (QOL) assessments in conjunction with its clinical trials and convened periodic workshops to review the progress of its inclusion as an endpoint (1,2). In 2003, the NCI staff initiated a systematic review of health-related quality of life (HRQL) assessments in the symptom management clinical trials conducted through the Community Clinical Oncology Program (CCOP). The review evaluated whether assessing QOL contributed additional value to the results of the trials beyond the effect of the intervention on the designated symptom (3). In practice, a clinician would treat a patient's symptoms even if the successful resolution of those symptoms did not result in an improvement in the patient's overall QOL (4).
The CCOP program is an NCI-funded clinical trials network and the primary mechanism to support NCI-sponsored symptom management clinical trials. From 1995 through 2005, more than 100 of these types of clinical trials were conducted. Almost all are randomized, placebo-controlled clinical trials with an assessment of the designated symptom as the primary endpoint.
HRQL assessments have been encouraged as a means to further describe the patient's experience in conjunction with his/her cancer treatment. The typical endpoints in cancer treatment clinical trials have been survival, response to therapy, risk of recurrence, and toxicities. These endpoints include both objective measures and physician-reported endpoints.
For clinical trials that are designed as toxicity reduction studies, the typical primary endpoint has been a reduction from a high to a lower grade of toxicity, as measured by the Common Terminology Criteria for Adverse Events (CTCAE, formerly Common Toxicity Criteria). For example, a study to evaluate an agent hypothesized to mitigate radiation-induced dermatitis would look at the reduction in grade 3 or higher skin toxicity as evaluated by CTCAE (5). However, not all toxicities are amenable to CTCAE, which is a clinician-based reporting tool. For subjective or symptomatic toxicities, the CTCAE might not accurately reflect the patient's perception of that toxicity or its burden (6). Thus, it becomes important to capture the patient's own assessment of improvement in these symptoms.
With a more precise assessment of the specific symptom, is it possible to also capture a sense of the impact of the symptom on the patient's overall HRQL? During the process of reviewing the assessment tools being used for the primary and secondary endpoints in the symptom management trials, several key issues emerged (3). Investigators did not routinely describe their underlying rationale for including HRQL assessments as secondary endpoints nor delineate the hypothesized relationship between the improvement of a symptom and a subsequent improved QOL. Finally, it became clear that the specific questions asked in the assessment tools may not always address the concerns or the issues that the investigators are most interested in exploring. For example, FACT-N, a seven-item instrument assessment tool to measure peripheral neuropathy, has one question specific to ringing in the ears (7). Although this is an important question to help capture cisplatin-induced neuropathy, it may not be relevant in the neuropathies that develop with some of the newer targeted agents. Thus, using this specific tool may not be the most suitable for capturing the patients' perceptions of neuropathy with some of the newer agents.
One of the recommendations from the NCI review was to convene a workshop that would explore the role of assessing QOL of patients enrolled on the symptom management clinical trials done through the NCI CCOP program. Convened on October 24–25, 2005, the workshop focused on three areas: 1) the rationale for QOL inclusion in symptom management trials, 2) the conceptual framework for the QOL assessment, and 3) the instruments, measurements, and analysis. The workshop included HRQL investigators within the CCOP program, HRQL investigators funded by NCI but not involved in the CCOP program, and NCI and Food and Drug Administration (FDA) colleagues. The FDA was invited to provide the regulatory perspective of incorporating QOL or patient-reported outcomes into symptom management trials. Additionally, the FDA was soon to release a draft guidance for the role of patient reported outcomes in the drug approval process (8).
As patients live longer both as cancer patients and as cancer survivors, there is a growing need to manage both short-term side effects and long-term sequelae of treatment. It becomes important to design and develop clinical trials in a manner that will provide useful information to physicians and patients. This monograph provides more fully developed versions of the papers that were presented at the workshop and a summary conclusion by Drs Ganz and Goodwin. The goal is to highlight critical issues to advance the state of the science in future research on HRQL assessment in cancer symptom management clinical trials.
|
Assessing Quality of Life (QOL) in Cancer Symptom Management Trials Workshop |
|---|
|
TopIntroductionAssessing Quality of Life...NotesReferences |
|---|
Participants List
Claudia Aguado, MPH
Research Coordinator
Pediatrics Epidemiology Center
Community Clinical Oncology Program
H. Lee Moffitt Cancer Center at the University of South Florida
Tampa, FL
Noreen Aziz, MD, PhD, MPH
Senior Program Director
Office of Cancer Survivorship
Division of Cancer Control and Population Sciences
National Cancer Institute
National Institutes of Health
Bethesda, MD
Andrea Barsevick, DNSc, RN, AOCN
Director of Nursing Research
Department of Population Science
Fox Chase Cancer Center
Cheltenham, PA
Peter Bross, MD
Clinical Team Leader
Division of Clinical Evaluation
Center for Biologics Evaluation and Research
Food and Drug Administration
Rockville, MD
David Buchanan, DrPH
Research Fellow
Division of Cancer Prevention
National Cancer Institute
National Institutes of Health
Bethesda, MD
Robert Croyle, PhD
Director
Division of Cancer Control and Population Sciences
National Cancer Institute
National Institutes of Health
Bethesda, MD
Andrea Denicoff, RN, MS, ANP
Nurse Consultant
Clinical Investigations Branch
Cancer Therapy Evaluation Program
Division of Cancer Treatment and Diagnosis
National Cancer Institute
National Institutes of Health
Bethesda, MD
Michelle DeSilvio, PhD
Senior Biostatistician
Department of Statistics
American College of Radiology
Philadelphia, PA
Raymond Dionne, PhD, DDS
Scientific Director
National Institute of Nursing Research
National Institutes of Health
Bethesda, MD
Molla Donaldson, DrPH
Senior Scientist
Quality of Care Research and Policy Outcomes Research Branch
Applied Research Program
Division of Cancer Control and Population Sciences
National Cancer Institute
National Institutes of Health
Bethesda, MD
Amylou Dueck, PhD
Research Associate
Division of Biostatistics
Mayo Clinic College of Medicine
Rochester, MN
Sara Dapen, PhD
Research Associate
Department of Biostatistics and Computational Biology
Eastern Cooperative Oncology Group Statistical Center
Dana-Farber Cancer Institute
Boston, MA
Ann Farrell, MD
Clinical Team Leader
Division of Drug Oncology Products
Food and Drug Administration
Rockville, MD
Michael Fisch, MD, MPH
Medical Director
Community Clinical Oncology Program
The University of Texas M. D. Anderson Cancer Center
Houston, TX
Carolyn Gotay, PhD
Professor, Program Director
Prevention and Control Program
Cancer Research Center of Hawaii
Honolulu, HI
Martha Hare, PhD, RN
Program Director
National Institute of Nursing Research
National Institutes of Health
Bethesda, MD
Pamela Hinds, PhD, RN
Director
Division of Nursing Research
St. Jude's Children's Research Hospital
Memphis, TN
Joseph Kelaghan, MD
Program Director
Community Oncology and Prevention Trials Research Group
National Cancer Institute
National Institutes of Health
Bethesda, MD
Alice Kornblith, PhD
Senior Research Scientist
Department of Medical Oncology
Women's Cancer Program
Dana-Farber Cancer Institute
Boston, MA
Virginia Kwitowski, MS, RN, CRNP
Senior Nurse Practitioner
Medical Oncology Branch
Center for Cancer Research
National Cancer Institute
National Institutes of Health
Bethesda, MD
Joseph Lipscomb, PhD
Professor of Public Health
Health Policy and Management
Rollins School of Public Health
Emory University
Atlanta, GA
Susan Marden, PhD
Clinical Nurse Scientist
National Institute of Nursing Research
National Institutes of Health
Bethesda, MD
Lori Minasian, MD
Chief
Community Oncology and Prevention Trials Research Group
Division of Cancer Prevention
National Cancer Institute
National Institutes of Health
Bethesda, MD
Gary Morrow, PhD, MS
Professor of Radiation Oncology
Professor of Psychiatry
James P. Wilmot Cancer Center
University of Rochester Medical Center
Rochester, NY
Michelle Naughton, PhD
Associate Professor
Section on Social Sciences and Health Policy
Department of Public Health Sciences
School of Medicine
Wake Forest University
Winston-Salem, NC
Ann O'Mara, PhD, RN
Program Director
Community Clinical Oncology Program
Division of Cancer Prevention
National Cancer Institute
National Institutes of Health
Bethesda, MD
J. Lynn Palmer, PhD
Associate Professor of Biostatistics
Palliative Care and Rehabilitation Medicine
The University of Texas M. D. Anderson Cancer Center
Houston, TX
Bryce Reeve, PhD
Psychometrician
Outcomes Research Branch
Applied Research Program
Division of Cancer Control and Population Sciences
National Cancer Institute
National Institutes of Health
Bethesda, MD
Ellen Richmond, RN, MS, GNP
Nurse Specialist (Clinical Trials)
Division of Cancer Prevention
National Cancer Institute
National Institutes of Health
Bethesda, MD
Julia Rowland, PhD
Director
Office of Cancer Survivorship
Division of Cancer Control and Population Sciences
National Cancer Institute
National Institutes of Health
Bethesda, MD
Jane Scott, PhD
Endpoint Reviewer
Study Endpoint and Development Team
Office of New Drugs
Center for Drug Evaluation and Research
U.S. Food and Drug Administration
Silver Spring, MD
Maria Sgambati, MD
Program Director
Community Oncology Program
Division of Cancer Prevention
National Cancer Institute
National Institutes of Health
Bethesda, MD
Bonnie Teschendorf, PhD
Director
Quality of Life Science
Cancer Control and Population Science
American Cancer Society
Atlanta, GA
Ted Trimble, MD, MPH
Head
Quality of Cancer Care Therapeutics
Clinical Investigations Branch
Cancer Therapy Evaluation Program
Division of Cancer Treatment and Diagnosis
National Cancer Institute
Bethesda, MD
Lynne Wagner, PhD
Clinical Research Scientist
The Center on Outcomes, Research and Education
Evanston Northwestern Healthcare
Northwestern University Medical School
Chicago, IL
Lari Wenzel, PhD
Associate Professor
General Internal Medicine
University of California, Irvine
Irvine, CA
Linda Wong
Extramural Support Assistant
Community Oncology and Prevention Trials Research Group
Division of Cancer Prevention
National Cancer Institute
National Institutes of Health
Bethesda, MD
|
NOTES |
|---|
Workshop entitled "Assessing Quality of Life (QOL) in Cancer Symptom Management Trials" was supported by the Division of Cancer Prevention, National Cancer Institute, National Institutes of Health, Department of Health and Human Services.
|
REFERENCES |
|---|
|
TopIntroductionAssessing Quality of Life...NotesReferences |
|---|
(1) Nayfield SG, Hailey BJ, McCabe M. Quality of life assessment in cancer clinical trials. Report of the Workshop on Quality of Life Research in Cancer Clinical Trials; July 16–17, 1990; Bethesda (MD):US DHHS; 1991.
(2) Varricchio CG, McCabe MS, Trimble E, Korn EL. Quality of life in clinical cancer trials. J Natl Cancer Inst Monogr (1996) 20:vii–viii.
(3) Buchanan DR, O'Mara AM, Kelaghan JW, Minasian LM. Quality-of-life assessment in the symptom management trials of the National Cancer Institute-supported Community Clinical Oncology Program. J Clin Oncol (2005) 23:591–8.
(4) Jatoi A, Kumar S, Sloan JA, Nguyen PL. On appetite and its loss. J Clin Oncol (2000) 18:2930–2.
(5) Elliot EA, Wright JR, Swann RS, Nguyen-Tân F, Takita C, Bucci MK, et al. Phase III trial of an emulsion containing trolamine for the prevention of radiation dermatitis in patients with advanced squamous cell carcinoma of the head and neck: results of Radiation Therapy Oncology Group Trial 99-13. J Clin Oncol (2006) 24:2092–7.
(6) Basch E, Iasonos A, McDonough T, Barz A, Culkin A, Kris MG, et al. Patient versus clinician symptom reporting using the National Cancer Institute Common Terminology Criteria for Adverse Events: results of a questionnaire-based study. Lancet Oncol (2006) 7:903–9.[CrossRef][Web of Science][Medline]
(7) Calhoun EA, Welshman EE, Chang CH, Lurain JR, Fishman DA, Hunt TL, et al. Psychometric evaluation of the Functional Assessment of Cancer Therapy/Gynecologic Oncology Group-Neurotoxicity (Fact/GOG-Ntx) questionnaire for patients receiving systemic chemotherapy. Int J Gynecol Cancer (2003) 13:741–8.[CrossRef][Web of Science][Medline]
(8) Guidance for industry patient-reported outcome measures: use in medical product development to support labeling claims. Available at: http://www.fda.gov/cder/guidance/5460dft.htm. [Last accessed: March 23, 2007.].
CiteULike 
Connotea 
Del.icio.us What's this?
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||