Patient-reported outcomes in cancer: A review of recent research and policy initiatives

CA Cancer Journal for Clinicians

Volumn 57, Issue 5, 2007, Pages 278-300

  Lipscomb, Joseph ^a   Gotay, Carolyn C ^b   Snyder, Claire F ^c  

^a EMORY UNIVERSITY   (United States)

^b UNIVERSITY OF HAWAII CANCER CENTER   (United States)

^c JOHNS HOPKINS UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CANCER CENTER; CANCER DIAGNOSIS; CANCER RADIOTHERAPY; CANCER RESEARCH; CANCER SURVIVAL; COLORECTAL CANCER; FUNCTIONAL ASSESSMENT; HEALTH CARE; HEALTH CARE POLICY; HEALTH SERVICE; HEALTH STATUS; HEALTH SURVEY; HUMAN; LUNG CANCER; MEDICAL DECISION MAKING; OUTCOME ASSESSMENT; PATIENT ASSESSMENT; PATIENT CARE; PRIORITY JOURNAL; PROSTATE CANCER; PROSTATECTOMY; QUALITY OF LIFE; REVIEW;

DECISION MAKING; DISEASE-FREE SURVIVAL; HEALTH SERVICES RESEARCH; HEALTH STATUS; HUMANS; NEOPLASMS; OUTCOME ASSESSMENT (HEALTH CARE); PAIN MEASUREMENT; PATIENT SATISFACTION; POLICY MAKING; POPULATION SURVEILLANCE; PRACTICE GUIDELINES; PRIMARY HEALTH CARE; QUALITY OF LIFE; SURVIVAL ANALYSIS; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 35449006752     PISSN: 00079235     EISSN: 15424863     Source Type: Journal    
DOI: 10.3322/CA.57.5.278     Document Type: Review

References (105)

1. National Cancer Institute. The Nation's Investment in Cancer Research: A Plan and Budget Proposal for Fiscal Year 2008. NIH Publication No. 06-6090. Available at: http://plan.cancer.gov/plan_overview.shtml. Accessed May 26, 2007.
2. American Cancer Society. 2015 Goals of the American Cancer Society. Available at: http://www.cancer.org/docroot/COM/content/div_PA/ COM_4_2X_Volunteer_11087.asp. Accessed March 16, 2007.
3. Johnson JR, Williams G, Pazdur R. End points and United States Food and Drug Administration approval of oncology drugs. J Clin Oncol 2003;21:1404-1411.
4. U.S. Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER), Center for Devices and Radiological Health (CDRH). Guidance for Industry-Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. Available at: http://www.fda.gov/cber/gdlns/prolbl.pdf. Accessed March 20, 2007.
5. Lipscomb J, Gotay CC, Snyder C. Outcomes Assessment in Cancer: Measures, Methods, and Applications. Cambridge, UK: Cambridge University Press; 2005.
6. National Cancer Institute. Patient-Reported Outcomes in Cancer Trials: Evaluating and Enhancing the Payoff to Decision Making. Available at: http://www.scgcorp.com/dccpspatient/index.asp. Accessed March 22, 2007.
7. Doroshow JH. Restructuring the National Cancer Clinical Trials Enterprise: Clinical Trials Working Group Implementation Update. Presented at: Patient-Reported Outcomes Assessment in Cancer Trials: Evaluating and Enhancing the Payoff to Decision Making; September 20-21, 2006; Bethesda, MD.
8. Sloan JA, Cella D, Frost M, et al.; Clinical Significance Consensus Meeting Group. Assessing clinical significance in measuring oncology patient quality of life: introduction to the symposium, content overview, and definition of terms. Mayo Clin Proc 2002;77:367-370.
9. Mayo Clinic. Quality of Life Conference - Scottsdale, AZ. Available at: http://www.mayo.edu/webcasts/arch_qol.html. Accessed March 19, 2007.
10. Centers for Medicare & Medicaid Services. Medicare Launches Efforts to Improve Care for Cancer Patients: New Coverage, Better Evidence, and New Support for Improving Quality of Care. Available at: http://www.cms.hhs.gov/ media/press/release.asp?Counter=1244. Accessed March 21, 2007.
11. Ayanian JZ, Chrischilles EA, Fletcher RH, et al. Understanding cancer treatment and outcomes: the Cancer Care Outcomes Research and Surveillance Consortium. J Clin Oncol 2004;22:2992-2996.
12. Schneider EC, Malin JL, Kahn KL, et al. Developing a system to assess the quality of cancer care: ASCO's national initiative on cancer care quality. J Clin Oncol 2004:22:2985-2991.
13. IMMPACT. Initiative on Methods, Measurement, and P ain Assessment in Clinical Trials. Available at: http://www.immpact.org/index.html. Accessed March 21, 2007.
14. National Institutes of Health. Patient-Reported Outcomes Measurement Information System: Dynamic Tools to Measure Health Outcomes From the Patient Perspective. Available at: http://www.nihPROMIS.org. Accessed March 22, 2007.
15. Acquadro C, Berzon R, Dubois D, et al. Incorporating the patient's perspective into drug development and communication: an ad hoc task force report of the Patient-Reported Outcomes (PRO) Harmonization Group meeting at the Food and Drug Administration, February 16, 2001. Value Health 2003;6:522-531.
16. Aaronson NK, Ahmedzai S, Bergman B, et al. The European Organization for Research and Treatment of Cancer QLQ-C30: a quality-of-life instrument for use in international clinical trials in oncology. J Natl Cancer Inst 1993;85:365-376.
17. Ferrans CE. Definitions and conceptual models of quality of life, in Lipscomb J, Gotay CC, Snyder C (eds). Outcomes Assessment in Cancer: Measures, Methods, and Applications. Cambridge, UK: Cambridge University Press; 2005:14-30.
18. Cella DF. Measuring quality of life in palliative care. Semin Oncol 1995;22:73-81.
19. Lipscomb J, Gotay CC, Snyder C. Introduction to outcomes assessment in cancer, in Lipscomb J, Gotay CC, Snyder C (eds). Outcomes Assessment in Cancer: Measures, Methods, and Applications. Cambridge, UK: Cambridge University Press; 2005:1-14.
20. de Haes JC, van Knippenberg FC, Neijt JP. Measuring psychological and physical distress in cancer patients: structure and application of the Rotterdam Symptom Checklist. Br J Cancer 1990;62:1034-1038.
21. Cleeland CS, Ryan KM. Pain assessment: global use of the Brief Pain Inventory. Ann Acad Med Singapore 1994;23:129-138.
22. National Cancer Institute. Common Terminology Criteria for Adverse Events v.3.0 (CTCAE). Available at: http://ctep.cancer.gov/reporting/ctc.html. Accessed July 15, 2007.
23. Mystakidou K, Tsilika E, Parpa E, et al. Beck Depression Inventory: exploring its psychometric properties in a palliative care population of advanced cancer patients. Eur J Cancer Care 2007;16:244-250.
24. Cella DF, Tulsky DS, Gray G, et al. The Functional Assessment of Cancer Therapy scale: development and validation of the general measure. J Clin Oncol 1993;11:570-579.
25. Feeny D, Furlong W, Torrance GW, et al. Multiattribute and single-attribute utility functions for the health utilities index mark 3 system. Med Care 2002:40:113-128.
26. Luo N, Johnson JA, Shaw JW, et al. Self-reported health status of the general adult U.S. population as assessed by the EQ-5D and Health Utilities Index. Med Care 2005;43:1078-1086.
27. Feeny DH. The roles for preference-based measures in support of cancer research and policy, in Lipscomb J, Gotay CC, Snyder C (eds). Outcomes Assessment in Cancer: Measures, Methods, and Applications. Cambridge, UK: Cambridge University Press; 2005:69-93.
28. Ware JE, Bayliss MS, Mannocchia M, Davis GL. Health-related quality of life in chronic hepatitis C: impact of disease and treatment response. The Interventional Therapy Group. Hepatology 1999;30:550-555.
29. Ware JE. SF-36® Health Survey Update. Available at: http://www.sf-36.org/tools/sf36.shtml#LIT. Accessed March 22, 2007.
30. Erickson P. Assessing health status and quality of life of cancer patients: the use of general instruments, in Lipscomb J, Gotay CC, Snyder C (eds). Outcomes Assessment in Cancer: Measures, Methods, and Applications. Cambridge, UK: Cambridge University Press; 2005:21-69.
31. Sprangers MA, Groenvold M, Arraras JI, et al. The European Organization for Research and Treatment of Cancer breast cancer-specific quality-of-life questionnaire module: first results from a three-country field study. J Clin Oncol 1996;14:2756-2768.
32. Calhoun EA, Change C-H, Welshman EE, et al. A neutropenia-specific quality of life instrument: rationale for the development of the FACT-N [abstract]. Proceedings of the American Society for Clinical Oncology 2002;21:375a. Abstract 1498.
33. Bacik J, Mazumdar M, Murphy BA, et al. The functional assessment of cancer therapy-BRM (FACT-BRM): a new tool for the assessment of quality of life in patients treated with biologic response modifiers. Qual Life Res 2004;13:137-154.
34. Lipscomb J, Donaldson MS, Hiatt RA. Cancer outcomes research and the arenas of application. J Natl Cancer Inst Monogr 2004;33:1-7.
35. Ward WL, Hahn EA, Mo F, et al. Reliability and validity of the Functional Assessment of Cancer Therapy-Colorectal (FACT-C) quality of life instrument. Qual Life Res 1999;8:181-195.
36. Shaw JW, Johnson JA, Coons SJ. US valuation of the EQ-5D health states: development and testing of the D1 valuation model. Med Care 2005;43:203-220.
37. Juniper EF, Guyatt GH, Jaeschke R. How to develop and validate a new health-related quality of life instrument, in Spilker B. Quality of Life and Pharmacoeconomics in Clinical Trials. Philadelphia, PA: Lippincott-Raven Publishers; 1996:49-56.
38. Kaplan RM, Anderson JP. The General Health Policy Model: an integrated approach, in Spilker B. Quality of Life and Pharmacoeconomics in Clinical Trials. Philadelphia, PA: Lippincott-Raven Publishers; 1996:309-322.
39. Scientific Advisory Committee of the Medical Outcomes Trust. Assessing health status and quality-of-life instruments: attributes and review criteria. Qual Life Res 2002;11:193-205.
40. Lipscomb J, Snyder CF, Gotay CC. Cancer outcomes measurement: Through the lens of the Medical Outcomes Trust framework. Qual Life Res 2007;16:143-164.
41. Snyder C, Gotay CC, Lipscomb J. The Cancer Outcomes Measurement Working Group: rationale, overview, and a look to the future. Expert Rev Pharmacoeconomics Outcomes Res 2006;6:407-416.
42. Gotay CC, Lipscomb J, Snyder CF. Reflections on findings of the Cancer Outcomes Measurement Working Group: moving to the next phase. J Natl Cancer Inst 2005;97:1568-1574.
43. Fairclough DL. Practical considerations in outcomes assessment for clinical trials, in Lipscomb J, Gotay CC, Snyder C (eds). Outcomes Assessment in Cancer: Measures, Methods, and Applications. Cambridge, UK: Cambridge University Press; 2005:346-362.
44. Ganz PA, Goodwin PJ. Quality of life in breast cancer - what have we learned and where do we go from here, in Lipscomb J, Gotay CC, Snyder C (eds). Outcomes Assessment in Cancer: Measures, Methods, and Applications. Cambridge, UK: Cambridge University Press; 2005:93-126.
45. Litwin MS, Talcott JA. Measur ing quality of life in prostate cancer: progress and challenges, in Lipscomb J, Gotay CC, Snyder C (eds). Outcomes Assessment in Cancer: Measures, Methods, and Applications. Cambridge, UK: Cambridge University Press; 2005:126-160.
46. Earle CC, Weeks JC. The science of quality-of-life measurement in lung cancer, in Lipscomb J, Gotay CC, Snyder C (eds). Outcomes Assessment in Cancer: Measures, Methods, and Applications. Cambridge, UK: Cambridge University Press; 2005:160-178.
47. Moinpour CM, Provenzale D. Treatment for colorectal cancer: impact on health-related quality of life, in Lipscomb J, Gotay CC, Snyder C (eds). Outcomes Assessment in Cancer: Measures, Methods, and Applications. Cambridge, UK: Cambridge University Press; 2005:178-201.
48. Mandelblatt JS, Selby JV. Short-term outcomes of chemoprevention, genetic susceptibility testing and screening interventions: What are they? How are they measured? When should they be measured?, in Lipscomb J, Gotay CC, Snyder C (eds). Outcomes Assessment in Cancer: Measures, Methods, and Applications. Cambridge, UK: Cambridge University Press; 2005:216-241.
49. Zebrack B, Cella D. Evaluating quality of life in cancer survivors, in Lipscomb J, Gotay CC, Snyder C (eds). Outcomes Assessment in Cancer: Measures, Methods, and Applications. Cambridge, UK: Cambridge University Press; 2005:241-264.
50. Ferrell BR. Assessing health-related quality of life at end of life, in Lipscomb J, Gotay CC, Snyder C (eds). Outcomes Assessment in Cancer: Measures, Methods, and Applications. Cambridge, UK: Cambridge University Press; 2005:264-286.
51. Snyder C. Assessing the subjective impact of caregiving on informal caregivers of cancer patients, in Lipscomb J, Gotay CC, Snyder C (eds). Outcomes Assessment in Cancer: Measures, Methods, and Applications. Cambridge, UK: Cambridge University Press, 2005:329-346.
52. 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;23:591-598.
53. Rock EP, Scott JA, Kennedy DL, et al. Challenges to use of health-related quality of life for FDA approval of anti-cancer drugs. J Natl Cancer Inst. In press.
54. Mayo Clinic. Experts to Address Use of Patient-Reported Outcomes in Clinical Trials at Joint Mayo-FDA Forum. Available at: http://www.mayoclinic. org/news2006-rst/3235.html. Accessed March 21, 2007.
55. Szende A, Leidy NK, Revicki D. Health-related quality of life and other patient-reported outcomes in the European centralized drug regulatory process: a review of guidance documents and performed authorizations of medicinal products 1995 to 2003. Value Health 2005;8:534-548.
56. Trimble EL, Rowland J, Varricchio C, Gore-Langton RE. Clinical trials referral resource. Health-related quality of life in cancer clinical trials. Oncology (Williston Park) 2001;15:456-458, 461-466, 601-603, 606-608, 611.
57. Gotay CC, Korn EL, McCabe MS, et al. Quality-of-life assessment in cancer treatment protocols: research issues in protocol development. J Natl Cancer Inst 1992;84:575-579.
58. Gotay CC, Korn EL, McCabe MS, et al. Building quality of life assessment into cancer treatment studies. Oncology (Williston Park) 1992;6:25-28.
59. National Cancer Institute. Report of the Clinical Trials Working Group of the National Cancer Advisory Board: Restructuring the National Cancer Clinical Trials Enter prise. Available at: http://integratedtrials.nci.nih.gov/ict/ CTWG_report_June2005.pdf. Accessed March 22, 2007.
60. Turk DC, Dworkin RH, Allen RR, et al. Core outcome domains for chronic pain clinical tr ials: IMMPACT recommendations. Pain 2003;106:337-345.
61. Dworkin RH, Turk DC, Farrar JT, et al. Core outcome measures for chronic pain clinical trials: IMMPACT recommendations. Pain 2005;113:9-19.
62. Lipscomb J, Donaldson MS, Arora NK, et al. Cancer outcomes research. J Natl Cancer Inst Monogr 2004;33:178-197.
63. Potosky AL, Harlan LC, Stanford JL, et al. Prostate cancer practice patterns and quality of life: the Prostate Cancer Outcomes Study. J Natl Cancer Inst 1999;91:1719-1724.
64. National Cancer Institute. History & Goals of the Prostate Cancer Outcomes Study. Available at: http://healthservices.cancer.gov/pcos/. Accessed March 21, 2007.
65. Penson DF, Feng Z, Kuniyuki A, et al. General quality of life 2 years following treatment for prostate cancer: what influences outcomes? Results from the prostate cancer outcomes study. J Clin Oncol 2003;21:1147-1154.
66. Potosky AL, Davis WW, Hoffman RM, et al. Five-year outcomes after prostatectomy or radiotherapy for prostate cancer: the prostate cancer outcomes study. J Natl Cancer Inst 2004;96:1358-1367.
67. Hoffman RM, Hunt WC, Gilliland FD, et al. Patient satisfaction with treatment decisions for clinically localized prostate carcinoma. Results from the Prostate Cancer Outcomes Study. Cancer 2003;97:1653-1662.
68. The Cancer Care Outcomes Research and Surveillance Consortium (CanCORS). The Cancer Care Outcomes Research and Surveillance Consortium: Share Thoughts on Care. Available at: http://www.cancors.org/public/. Accessed March 22, 2007.
69. Malin JL, Schneider EC, Epstein AM, et al. Results of the National Initiative for Cancer Care Quality: how can we improve the quality of cancer care in the United States? J Clin Oncol 2006;24:626-634.
70. Donaldson MS. Taking stock of health-related quality-of-life measurement in oncology practice in the United States. J Natl Cancer Inst Monogr 2004;33:155-167.
71. Donaldson MS. Using patient-reported outcomes in clinical oncology practice: benefits, challenges and next steps. Expert Rev Pharmacoeconomics Outcomes Res 2006;6:87-95.
72. Ganz PA. What outcomes matter to patients: a physician-researcher point of view. Med Care 2002;40(suppl):III11-III19.
73. Hewitt M, Greenfield S, Stovall E (eds). From Cancer Patient to Cancer Survivor: Lost in Transition. Washington, DC: National Academy Press; 2005.
74. Detmar SB, Muller MJ, Schornagel JH, et al. Health-related quality-of-life assessments and patient-physician communication: a randomized controlled trial. JAMA 2002;288:3027-3034.
75. Velikova G, Booth L, Smith AB, et al. Measuring quality of life in routine oncology practice improves communication and patient well-being: a randomized controlled trial. J Clin Oncol 2004;22:714-724.
76. Gotay CC, Bottomley A, Kawamoto C, et al. Quality of life as a prognostic factor in cancer. Presented at: 12th Annual Conference of the International Society for Quality of Life Research (ISOQOL); October 19-22, 2005; San Francisco, CA.
77. Efficace F, Bottomley A, Coens C, et al. Does a patient's self-reported health-related quality of life predict survival beyond key biomedical data in advanced colorectal cancer? Eur J Cancer 2006;42:42-49.
78. Office of the Inspector General, US Department of Health and Human Services. Cost and Performance of Medicare's 2005 Chemotherapy Demonstration Project. Available at: http://oig.hhs.gov/oei-09-05-00171.pdf. Accessed July 15, 2007.
79. U.S. Senate Committee on Finance. Grassley says government needs to find a better way to improve care for cancer patients. Available at: http://finance.senate.gov/press/Gpress/2005/prg083006a.pdf. Accessed March 22, 2007.
80. CMS (Centers for Medicare & Medicaid Services). 2006 Oncology Demonstration Program: Improved Quality of Care for Cancer Patients Through More Effective Payments and Evidence-Based Care. Available at: http://www.cms.hhs. gov/media/press/release.asp?Counter=1717. Accessed March 22, 2007.
81. International Society for Quality of Life Research. Patient-Reported Outcomes in Clinical Practice; June 24-26, 2007; Budapest, Hungary. Available at: http://www.isoqol.org. Accessed June 21, 2007.
82. American Cancer Society. Cancer Facts & Figures 2006. Available at: http://www.cancer.org/downloads/STT/CAFF2006PWSecured.pdf. Accessed March 22, 2007.
83. American Cancer Society. Challenge Goals, Principles, & Nationwide Objectives. Available at: www.facs.org/cancer/coc/acschallengegoals.pdf. Accessed June 21, 2007.
84. Institute of Medicine National Research Council. Enhancing data systems to improve the quality of cancer care. Washington, DC: National Academy Press; 2000.
85. Clauser SB. Use of cancer performance measures in population health: a macro-level perspective. J Natl Cancer Inst Monogr 2004;33:142-154.
86. Gotay CC, Lipscomb J. Data for cancer outcomes research: identifying and strengthening the empirical base, in Lipscomb J, Gotay CC, Snyder C (eds). Outcomes Assessment in Cancer: Measures, Methods, and Applications. Cambridge, UK: Cambridge University Press; 2005:522-549.
87. Wingo PA, Howe HL, Thun MJ, et al. A national framework for cancer surveillance in the United States. Cancer Causes Control 2005;16:151-170.
88. Hays RD, Revicki D, Coyne KS. Application of structural equation modeling to health outcomes research. Eval Health Prof 2005;28:295-309.
89. Guyatt GH, Osoba D, Wu AW, et al.; Clinical Significance Consensus Meeting Group. Methods to explain the clinical significance of health status measures. Mayo Clin Proc 2002;77:371-383.
90. Cella D, Bullinger M, Scott C, et al.; Clinical Significance Consensus Meeting Group. Group vs individual approaches to understanding the clinical significance of differences or changes in quality of life. Mayo Clin Proc 2002;77:384-392.
91. Sloan JA, Aaronson N, Cappelleri JC, et al.; Clinical Significance Consensus Meeting Group. Assessing the clinical significance of single items relative to summated scores. Mayo Clin Proc 2002;77:479-487.
92. Frost MH, Bonomi AE, Ferrans CE, et al.; Clinical Significance Consensus Meeting Group. Patient, clinician, and population perspectives on determining the clinical significance of quality-of-life scores. Mayo Clin Proc 2002;77:488-494.
93. Sprangers MA, Moinpour CM, Moynihan TJ, et al.; Clinical Significance Consensus Meeting Group. Assessing meaningful change in quality of life over time: a users' guide for clinicians. Mayo Clin Proc 2002;77:561-571.
94. Symonds T, Berzon R, Marquis P, et al.; Clinical Significance Consensus Meeting Group. The clinical significance of quality-of-life results: practical considerations for specific audiences. Mayo Clin Proc 2002;77:572-583.
95. Osoba D. The clinical value and meaning of health-related quality of life outcomes in oncology, in Lipscomb J, Gotay CC, Snyder C (eds). Outcomes Assessment in Cancer: Measures, Methods, and Applications. Cambridge, UK: Cambridge University Press; 2005:386-405.
96. Cohen J. Statistical Power Analysis for the Behavioral Sciences. Hillsdale, NJ: Lawrence Erlbaum Associates; 1988.
97. Schwartz CE, Sprangers MA. Adaptation to Changing Health: Response Shift in Quality-of-Life Research. Washington, DC: American Psychological Association; 2000.
98. Sprangers MA, Schwartz CE. Integrating response shift into health-related quality of life research: a theoretical model. Soc Sci Med 1999;48:1507-1515.
99. Reise SP. Item response theory and its applications for cancer outcomes measurement, in Lipscomb J, Gotay CC, Snyder C (eds). Outcomes Assessment in Cancer: Measures, Methods, and Applications. Cambridge, UK: Cambridge University Press; 2005:425-444.
100. McHorney CA, Haley SM, Ware JE Jr. Evaluation of the MOS SF-36 Physical Functioning Scale (PF-10): II. Comparison of relative precision using Likert and Rasch scoring methods. J Clin Epidemiol 1997;50:451-467.
101. Ware JE. Conceptualization and measurement of health-related quality of life: comments on an evolving field. Arch Phys Med Rehabil 2003;84(suppl):S43- S51.
102. Reise SP, Henson JM. A discussion of modern versus traditional psychometrics as applied to personality assessment scales. J Pers Assess 2003;81;93-103.
103. Gotay CC. Quality of life assessment for targeted therapies. Report presented at: The Institute of Medicine; 2003; Washington, DC.
104. Langeveld NE, Stam H, Grootenhuis MA, Last BF. Quality of life in young adult survivors of childhood cancer. Support Care Cancer 2002;10:579-600.
105. Gotay CC, Muraoka MY. Quality of life in long-term survivors of adult-onset cancers. J Natl Cancer Inst 1998;90:656-667.