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Published in: BMC Medicine 1/2011

Open Access 01-12-2011 | Opinion

Measuring patient-reported outcomes: moving beyond misplaced common sense to hard science

Author: Stephen P McKenna

Published in: BMC Medicine | Issue 1/2011

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Abstract

Interest in the patient's views of his or her illness and treatment has increased dramatically. However, our ability to appropriately measure such issues lags far behind the level of interest and need. Too often such measurement is considered to be a simple and trivial activity that merely requires the application of common sense. However, good quality measurement of patient-reported outcomes is a complex activity requiring considerable expertise and experience. This review considers the most important issues related to such measurement in the context of chronic disease and details how instruments should be developed, validated and adapted for use in additional languages. While there is often consensus on how best to undertake these activities, there is generally little evidence to support such accord. The present article questions these orthodox views and suggests alternative approaches that have been shown to be effective.
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Literature
1.
go back to reference World Health Organization: International Classification of Impairments, Disabilities and Handicaps: A Manual of Classification Relating to Consequences of Disease. 1980, Geneva: World Health Organization World Health Organization: International Classification of Impairments, Disabilities and Handicaps: A Manual of Classification Relating to Consequences of Disease. 1980, Geneva: World Health Organization
2.
go back to reference World Health Organization: International Classification of Functioning, Disability and Health. 2001, Geneva: World Health Organization World Health Organization: International Classification of Functioning, Disability and Health. 2001, Geneva: World Health Organization
3.
go back to reference Acquadro C, Berzon R, Dubois D, Leidy NK, Marquis P, Revicki D, Rothman M, PRO Harmonization Group: Incorporating the patient's perspective into drug development and communication: an ad hoc task force report on the patient-reported outcomes (PRO) harmonization group meeting at the Food and Drug Administration, February 16, 2001. Value Health. 2003, 5: 522-531.CrossRef Acquadro C, Berzon R, Dubois D, Leidy NK, Marquis P, Revicki D, Rothman M, PRO Harmonization Group: Incorporating the patient's perspective into drug development and communication: an ad hoc task force report on the patient-reported outcomes (PRO) harmonization group meeting at the Food and Drug Administration, February 16, 2001. Value Health. 2003, 5: 522-531.CrossRef
4.
go back to reference Burke LB, Kennedy DL, Miskala PH, Papadopoulos EJ, Trentacosti AM: The use of patient-reported outcome measures in the evaluation of medical products for regulatory approval. Clin Pharmacol Ther. 2008, 84: 281-283. 10.1038/clpt.2008.128.CrossRefPubMed Burke LB, Kennedy DL, Miskala PH, Papadopoulos EJ, Trentacosti AM: The use of patient-reported outcome measures in the evaluation of medical products for regulatory approval. Clin Pharmacol Ther. 2008, 84: 281-283. 10.1038/clpt.2008.128.CrossRefPubMed
6.
7.
go back to reference Burke L: Acceptable evidence for pharmaceutical advertising and labeling. DIA Workshop on Pharmacoeconomics and Quality of Life Labeling and Marketing Claims, October 3, 2000, New Orleans, LA. 2000, Horsham, PA: Drug Information Association Burke L: Acceptable evidence for pharmaceutical advertising and labeling. DIA Workshop on Pharmacoeconomics and Quality of Life Labeling and Marketing Claims, October 3, 2000, New Orleans, LA. 2000, Horsham, PA: Drug Information Association
8.
go back to reference Doward LC, McKenna SP: Evolution of quality of life assessment. Care Management of Skin Diseases: Life Quality and Economic Impact. Edited by: Rajagopalan R, Sheretz EF, Anderson RT. 1997, New York: Marcel Dekker, 9-33. Doward LC, McKenna SP: Evolution of quality of life assessment. Care Management of Skin Diseases: Life Quality and Economic Impact. Edited by: Rajagopalan R, Sheretz EF, Anderson RT. 1997, New York: Marcel Dekker, 9-33.
9.
go back to reference Hunt SM, McKenna SP: The QLDS: A scale for the measurement of quality of life in depression. Health Policy. 1992, 22: 307-319. 10.1016/0168-8510(92)90004-U.CrossRefPubMed Hunt SM, McKenna SP: The QLDS: A scale for the measurement of quality of life in depression. Health Policy. 1992, 22: 307-319. 10.1016/0168-8510(92)90004-U.CrossRefPubMed
10.
11.
go back to reference Hyde M, Wiggins RD, Higgs P, Blane DB: A measure of quality of life in early old age: the theory, development and properties of a needs satisfaction model (CASP-19). Aging Ment Health. 2003, 7: 186-194. 10.1080/1360786031000101157.CrossRefPubMed Hyde M, Wiggins RD, Higgs P, Blane DB: A measure of quality of life in early old age: the theory, development and properties of a needs satisfaction model (CASP-19). Aging Ment Health. 2003, 7: 186-194. 10.1080/1360786031000101157.CrossRefPubMed
12.
go back to reference Hornquist JO: The concept of quality of life. Scand J Soc Med. 1982, 10: 57-61.PubMed Hornquist JO: The concept of quality of life. Scand J Soc Med. 1982, 10: 57-61.PubMed
13.
go back to reference Gilworth G, Chamberlain MA, Bhakta B, Haskard D, Silman A, Tennant A: Development of the BD-QoL: a quality of life measure specific to Behçet's disease. J Rheumatol. 2004, 31: 931-937.PubMed Gilworth G, Chamberlain MA, Bhakta B, Haskard D, Silman A, Tennant A: Development of the BD-QoL: a quality of life measure specific to Behçet's disease. J Rheumatol. 2004, 31: 931-937.PubMed
14.
go back to reference Rabin R, de Charro F: EQ-5D: A measure of health status from the EuroQol Group. Ann Med. 2001, 33: 337-343. 10.3109/07853890109002087.CrossRefPubMed Rabin R, de Charro F: EQ-5D: A measure of health status from the EuroQol Group. Ann Med. 2001, 33: 337-343. 10.3109/07853890109002087.CrossRefPubMed
15.
go back to reference Macran S, Weatherly H, Kind P: Measuring population health: a comparison of three generic health status measures. Med Care. 2003, 41: 218-231.PubMed Macran S, Weatherly H, Kind P: Measuring population health: a comparison of three generic health status measures. Med Care. 2003, 41: 218-231.PubMed
16.
go back to reference Doward LC, McKenna SP, Meads DM, Ratcliffe J, Whalley D, Langley PC: Investigation into the feasibility of deriving relative and absolute utility from the Recurrent Genital Herpes Quality of Life Questionnaire (RGHQoL). Value Health. 2002, 5: 573-574.CrossRef Doward LC, McKenna SP, Meads DM, Ratcliffe J, Whalley D, Langley PC: Investigation into the feasibility of deriving relative and absolute utility from the Recurrent Genital Herpes Quality of Life Questionnaire (RGHQoL). Value Health. 2002, 5: 573-574.CrossRef
17.
go back to reference McKenna SP, Ratcliffe J, Meads DM, Brazier JE: Development and validation of a preference based measure derived from the Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) for use in cost utility analyses. Health Qual Life Outcomes. 2008, 6: 65-10.1186/1477-7525-6-65.CrossRefPubMedPubMedCentral McKenna SP, Ratcliffe J, Meads DM, Brazier JE: Development and validation of a preference based measure derived from the Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) for use in cost utility analyses. Health Qual Life Outcomes. 2008, 6: 65-10.1186/1477-7525-6-65.CrossRefPubMedPubMedCentral
18.
go back to reference Meads DM, McKenna SP, Doughty N, Das C, Gin-Sing W, Langley J, Pepke-Zaba J: The responsiveness and validity of the CAMPHOR Utility Index. Eur Resp J. 2008, 32: 1513-1519. 10.1183/09031936.00069708.CrossRef Meads DM, McKenna SP, Doughty N, Das C, Gin-Sing W, Langley J, Pepke-Zaba J: The responsiveness and validity of the CAMPHOR Utility Index. Eur Resp J. 2008, 32: 1513-1519. 10.1183/09031936.00069708.CrossRef
19.
go back to reference Mavranezouli I, Brazier JE, Young TA, Barkham M: Using Rasch analysis to form plausible health states amenable to valuation: the development of CORE-6D from a measure of common mental health problems (CORE-OM). Qual Life Res. 2011, 20: 321-333. 10.1007/s11136-010-9768-4.CrossRefPubMed Mavranezouli I, Brazier JE, Young TA, Barkham M: Using Rasch analysis to form plausible health states amenable to valuation: the development of CORE-6D from a measure of common mental health problems (CORE-OM). Qual Life Res. 2011, 20: 321-333. 10.1007/s11136-010-9768-4.CrossRefPubMed
20.
go back to reference McTaggart-Cowan HM, Marra CA, Yang Y, Brazier JE, Kopec JA, FitzGerald JM, Anis AH, Lynd LD: The validity of generic and condition-specific preference-based instruments: the ability to discriminate asthma control status. Qual Life Res. 2008, 17: 453-462. 10.1007/s11136-008-9309-6.CrossRefPubMed McTaggart-Cowan HM, Marra CA, Yang Y, Brazier JE, Kopec JA, FitzGerald JM, Anis AH, Lynd LD: The validity of generic and condition-specific preference-based instruments: the ability to discriminate asthma control status. Qual Life Res. 2008, 17: 453-462. 10.1007/s11136-008-9309-6.CrossRefPubMed
21.
go back to reference Bergner M, Bobbit RA, Kressel S, Pollard WE, Gilson BS, Morris JR: The Sickness Impact Profile: conceptual formulation and methodology for the development of a health status measure. Int J Health Serv. 1976, 6: 393-415. 10.2190/RHE0-GGH4-410W-LA17.CrossRefPubMed Bergner M, Bobbit RA, Kressel S, Pollard WE, Gilson BS, Morris JR: The Sickness Impact Profile: conceptual formulation and methodology for the development of a health status measure. Int J Health Serv. 1976, 6: 393-415. 10.2190/RHE0-GGH4-410W-LA17.CrossRefPubMed
22.
go back to reference Hunt SM, McEwen J, McKenna SP: Measuring Health Status. 1986, London: Croom Helm Hunt SM, McEwen J, McKenna SP: Measuring Health Status. 1986, London: Croom Helm
23.
go back to reference Ware JE, Sherbourne CD: The MOS 36-Item Short-Form Health Survey (SF-36). Med Care. 1992, 30: 474-483. Ware JE, Sherbourne CD: The MOS 36-Item Short-Form Health Survey (SF-36). Med Care. 1992, 30: 474-483.
24.
go back to reference Nijsten T, Meads DM, McKenna SP: Dimensionality of the Dermatology Life Quality Index (DLQI): a commentary. Acta Dermato-Venereologica. 2006, 86: 284-285. 10.2340/00015555-0075.CrossRefPubMed Nijsten T, Meads DM, McKenna SP: Dimensionality of the Dermatology Life Quality Index (DLQI): a commentary. Acta Dermato-Venereologica. 2006, 86: 284-285. 10.2340/00015555-0075.CrossRefPubMed
25.
go back to reference Tennant A, Penta M, Tesio L, Grimby G, Thonnard JL, Slade A, Lawton G, Simone A, Carter J, Lundgren-Nilsson A, Tripolski M, Ring H, Biering-Sørensen F, Marincek C, Burger H, Phillips S: Assessing and adjusting for cross-cultural validity of impairment and activity limitation scales through differential item functioning within the framework of the Rasch model: the PRO-ESOR project. Med Care. 2004, 42 (1 Suppl): I37-I48.PubMed Tennant A, Penta M, Tesio L, Grimby G, Thonnard JL, Slade A, Lawton G, Simone A, Carter J, Lundgren-Nilsson A, Tripolski M, Ring H, Biering-Sørensen F, Marincek C, Burger H, Phillips S: Assessing and adjusting for cross-cultural validity of impairment and activity limitation scales through differential item functioning within the framework of the Rasch model: the PRO-ESOR project. Med Care. 2004, 42 (1 Suppl): I37-I48.PubMed
26.
go back to reference Bjorner JB, Pejtersen JH: Evaluating construct validity of the second version of the Copenhagen Psychosocial Questionnaire through analysis of differential item functioning and differential item effect. Scand J Public Health. 2010, 38 (3 Suppl): 90-105. 10.1177/1403494809352533.CrossRefPubMed Bjorner JB, Pejtersen JH: Evaluating construct validity of the second version of the Copenhagen Psychosocial Questionnaire through analysis of differential item functioning and differential item effect. Scand J Public Health. 2010, 38 (3 Suppl): 90-105. 10.1177/1403494809352533.CrossRefPubMed
27.
go back to reference Chien CW, Brown T, McDonald R: Rasch analysis of the assessment of children's hand skills in children with and without disabilities. Res Dev Disabil. 2011, 32: 253-261. 10.1016/j.ridd.2010.09.022.CrossRefPubMed Chien CW, Brown T, McDonald R: Rasch analysis of the assessment of children's hand skills in children with and without disabilities. Res Dev Disabil. 2011, 32: 253-261. 10.1016/j.ridd.2010.09.022.CrossRefPubMed
28.
go back to reference Keenan AM, McKenna SP, Doward LC, Conaghan PG, Emery P, Tennant A: OAQoL: the development and validation of a quality of life instrument for osteoarthritis. Arthritis Rheum. 2008, 15: 841-848.CrossRef Keenan AM, McKenna SP, Doward LC, Conaghan PG, Emery P, Tennant A: OAQoL: the development and validation of a quality of life instrument for osteoarthritis. Arthritis Rheum. 2008, 15: 841-848.CrossRef
29.
go back to reference Bradley C: Importance of differentiating health status from quality of life. Lancet. 2001, 357: 7-8. 10.1016/S0140-6736(00)03562-5.CrossRefPubMed Bradley C: Importance of differentiating health status from quality of life. Lancet. 2001, 357: 7-8. 10.1016/S0140-6736(00)03562-5.CrossRefPubMed
30.
go back to reference Jahoda M: Employment and Unemployment: A Social Psychological Analysis. 1982, Cambridge, UK: Cambridge University Press Jahoda M: Employment and Unemployment: A Social Psychological Analysis. 1982, Cambridge, UK: Cambridge University Press
31.
go back to reference Fryer DM, McKenna SP: The laying off of hands. Unemployment: Personal and Social Consequences. Edited by: Fineman S. 1987, London: Tavistock, 47-73. Fryer DM, McKenna SP: The laying off of hands. Unemployment: Personal and Social Consequences. Edited by: Fineman S. 1987, London: Tavistock, 47-73.
32.
go back to reference Erickson P, Taeuber RC, Scott J: Operational aspects of quality-of-life assessment: choosing the right instrument. Pharmacoeconomics. 1995, 7: 39-48. 10.2165/00019053-199507010-00005.CrossRefPubMed Erickson P, Taeuber RC, Scott J: Operational aspects of quality-of-life assessment: choosing the right instrument. Pharmacoeconomics. 1995, 7: 39-48. 10.2165/00019053-199507010-00005.CrossRefPubMed
33.
go back to reference Emery MP, Perrier LL, Acquadro C: Patient-Reported Outcome and Quality of Life Instruments Database (PROQOLID): frequently asked questions. Health Qual Life Outcomes. 2005, 3: 12-10.1186/1477-7525-3-12.CrossRefPubMedPubMedCentral Emery MP, Perrier LL, Acquadro C: Patient-Reported Outcome and Quality of Life Instruments Database (PROQOLID): frequently asked questions. Health Qual Life Outcomes. 2005, 3: 12-10.1186/1477-7525-3-12.CrossRefPubMedPubMedCentral
34.
go back to reference Patrick DL, Burke LB, Powers JH, Scott JA, Rock EP, Dawisha S, O'Neill R, Kennedy DL: Patient-reported outcomes to support medical product labeling claims: FDA perspective. Value Health. 2007, 10 (Suppl 2): S125-S137.CrossRefPubMed Patrick DL, Burke LB, Powers JH, Scott JA, Rock EP, Dawisha S, O'Neill R, Kennedy DL: Patient-reported outcomes to support medical product labeling claims: FDA perspective. Value Health. 2007, 10 (Suppl 2): S125-S137.CrossRefPubMed
35.
go back to reference US Department of Health and Human Services, US Food and Drug Administration: Guidance for Industry: Patient-Reported Outcome Measures. Use in Medical Product Development to Support Labeling Claims. 2009, Washington, DC: US Food and Drug Administration US Department of Health and Human Services, US Food and Drug Administration: Guidance for Industry: Patient-Reported Outcome Measures. Use in Medical Product Development to Support Labeling Claims. 2009, Washington, DC: US Food and Drug Administration
36.
go back to reference European Medicines Agency, Committee for Medicinal Products for Human Use (CMHP): Reflection Paper on the Regulatory Guidance for the Use of Health-Related Quality of Life (HRQL) Measures in the Evaluation of Medicinal Products. 2005, London: European Medicines Agency European Medicines Agency, Committee for Medicinal Products for Human Use (CMHP): Reflection Paper on the Regulatory Guidance for the Use of Health-Related Quality of Life (HRQL) Measures in the Evaluation of Medicinal Products. 2005, London: European Medicines Agency
37.
go back to reference Güthlin C, Walach H: Structural equation modeling to test the construct validity of the second-order factor structure. Eur J Psychol Assess. 2007, 23: 15-23. 10.1027/1015-5759.23.1.15.CrossRef Güthlin C, Walach H: Structural equation modeling to test the construct validity of the second-order factor structure. Eur J Psychol Assess. 2007, 23: 15-23. 10.1027/1015-5759.23.1.15.CrossRef
38.
go back to reference Horner-Johnson W, Krahn GL, Suzuki R, Peterson JJ, Roid G, Hall T, RRTC Expert Panel on Health Measurement: Differential performance of SF-36 items in healthy adults with and without functional limitations. Arch Phys Med Rehabil. 2010, 91: 570-575. 10.1016/j.apmr.2009.12.015.CrossRefPubMed Horner-Johnson W, Krahn GL, Suzuki R, Peterson JJ, Roid G, Hall T, RRTC Expert Panel on Health Measurement: Differential performance of SF-36 items in healthy adults with and without functional limitations. Arch Phys Med Rehabil. 2010, 91: 570-575. 10.1016/j.apmr.2009.12.015.CrossRefPubMed
39.
go back to reference Dixon P, Heaton J, Long A, Warburton A: Reviewing and applying the SF-36. Outcomes Briefing. 1994, 4: 3-25. Dixon P, Heaton J, Long A, Warburton A: Reviewing and applying the SF-36. Outcomes Briefing. 1994, 4: 3-25.
40.
go back to reference European Medicines Agency, Committee for Medicinal Products for Human Use (CMHP): Draft Biomarkers Qualification: Guidance to Applicants. 2008, London: European Medicines Agency European Medicines Agency, Committee for Medicinal Products for Human Use (CMHP): Draft Biomarkers Qualification: Guidance to Applicants. 2008, London: European Medicines Agency
42.
go back to reference Tennant A, Gonaghan PG: The Rasch measurement model in rheumatology: What is it and why use it? When should it be applied, and what should one look for in a Rasch paper?. Arthritis Rheum. 2007, 57: 1358-1362. 10.1002/art.23108.CrossRefPubMed Tennant A, Gonaghan PG: The Rasch measurement model in rheumatology: What is it and why use it? When should it be applied, and what should one look for in a Rasch paper?. Arthritis Rheum. 2007, 57: 1358-1362. 10.1002/art.23108.CrossRefPubMed
43.
go back to reference Pallant JF, Tennant A: An introduction to the Rasch measurement model: An example using the Hospital Anxiety and Depression Scale (HADS). Br J Clin Psychol. 2007, 46: 1-18. 10.1348/014466506X96931.CrossRefPubMed Pallant JF, Tennant A: An introduction to the Rasch measurement model: An example using the Hospital Anxiety and Depression Scale (HADS). Br J Clin Psychol. 2007, 46: 1-18. 10.1348/014466506X96931.CrossRefPubMed
44.
go back to reference De Jong Z, van der Heijde D, McKenna SP, Whalley D: The reliability and construct validity of the RAQoL: A rheumatoid arthritis-specific quality of life instrument. Br J Rheumatol. 1997, 36: 878-883. 10.1093/rheumatology/36.8.878.CrossRefPubMed De Jong Z, van der Heijde D, McKenna SP, Whalley D: The reliability and construct validity of the RAQoL: A rheumatoid arthritis-specific quality of life instrument. Br J Rheumatol. 1997, 36: 878-883. 10.1093/rheumatology/36.8.878.CrossRefPubMed
45.
go back to reference Doward LC, McKenna SP, Kohlmann T, Niero M, Patrick D, Spencer B, Thorsen H: The international development of the RGHQoL: a quality of life measure for recurrent genital herpes. Qual Life Res. 1998, 7: 143-153. 10.1023/A:1008857426633.CrossRefPubMed Doward LC, McKenna SP, Kohlmann T, Niero M, Patrick D, Spencer B, Thorsen H: The international development of the RGHQoL: a quality of life measure for recurrent genital herpes. Qual Life Res. 1998, 7: 143-153. 10.1023/A:1008857426633.CrossRefPubMed
46.
go back to reference McKenna SP, Doward LC, Alonso J, Kohlmann T, Niero M, Prieto L, Wíren L: The QoL-AGHDA: an instrument for the assessment of quality of life in adults with growth hormone deficiency. Qual Life Res. 1999, 8: 373-383. 10.1023/A:1008987922774.CrossRefPubMed McKenna SP, Doward LC, Alonso J, Kohlmann T, Niero M, Prieto L, Wíren L: The QoL-AGHDA: an instrument for the assessment of quality of life in adults with growth hormone deficiency. Qual Life Res. 1999, 8: 373-383. 10.1023/A:1008987922774.CrossRefPubMed
47.
go back to reference Wild D, Grove A, Martin M, Eremenco S, McElroy S, Verjee-Lorenz A, Erikson P: Principles of good practice for the translation and cultural adaptation process for patient reported outcomes (PRO) measures: report of the ISPOR task force for translation and cultural adaptation. Value Health. 2005, 8: 94-104. 10.1111/j.1524-4733.2005.04054.x.CrossRefPubMed Wild D, Grove A, Martin M, Eremenco S, McElroy S, Verjee-Lorenz A, Erikson P: Principles of good practice for the translation and cultural adaptation process for patient reported outcomes (PRO) measures: report of the ISPOR task force for translation and cultural adaptation. Value Health. 2005, 8: 94-104. 10.1111/j.1524-4733.2005.04054.x.CrossRefPubMed
48.
go back to reference Hagell P, Hedin PJ, Meads DM, Nyberg L, McKenna SP: Effects of method of translation of patient-reported health outcome questionnaires: a randomized study of the translation of the Rheumatoid Arthritis Quality of Life (RAQoL) instrument for Sweden. Value Health. 2010, 13: 424-430. 10.1111/j.1524-4733.2009.00677.x.CrossRefPubMed Hagell P, Hedin PJ, Meads DM, Nyberg L, McKenna SP: Effects of method of translation of patient-reported health outcome questionnaires: a randomized study of the translation of the Rheumatoid Arthritis Quality of Life (RAQoL) instrument for Sweden. Value Health. 2010, 13: 424-430. 10.1111/j.1524-4733.2009.00677.x.CrossRefPubMed
49.
go back to reference McKenna SP, Doward LC, Meads DM, Patrick D, Tennant A: Summary of needs-based quality of life instruments. Value Health. 2004, 7 (Suppl 1): S39-S40.CrossRef McKenna SP, Doward LC, Meads DM, Patrick D, Tennant A: Summary of needs-based quality of life instruments. Value Health. 2004, 7 (Suppl 1): S39-S40.CrossRef
Metadata
Title
Measuring patient-reported outcomes: moving beyond misplaced common sense to hard science
Author
Stephen P McKenna
Publication date
01-12-2011
Publisher
BioMed Central
Published in
BMC Medicine / Issue 1/2011
Electronic ISSN: 1741-7015
DOI
https://doi.org/10.1186/1741-7015-9-86

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