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Patient-Reported Outcomes: The Example of Health-Related Quality of Life—a European Guidance Document for the Improved Integration of Health-Related Quality of Life Assessment in the Drug Regulatory Process

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Abstract

The added value for assessing the health-related quality of life (HRQOL) in chronic conditions is now well documented for evaluation of treatment effectiveness in clinical trials and as a criterion for licensing new medications and in policy decisions. However, European standards still need to be developed for the measurement and reporting of HRQOL in clinical trials. This is one of the objectives of the European Regulatory Issues on Quality of Life Assessment (ERIQA) Working Group. This document reviews the major issues arising from the selection of an HRQOL instrument; the integration of HRQOL assessment into the research protocol (methodological design, practicalities of HRQOL administration and collection, prevention and handling of missing data); the statistical analysis plan; and the presentation and interpretation of the results. Finally, to gain wider acceptance, whether HRQOL is considered as a primary or secondary endpoint, the scientific principles of clinical trial design should apply to HRQOL.

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References

  1. Johnson JR, Temple R. Food and Drug Administration requirements for approval of new anticancer drugs. Cancer Treat Rep 1985;69:1155–1159.

    CAS  PubMed  Google Scholar 

  2. Lohr KN, Aaronson NK, Alonso J, Bumam MA, Patrick DL, Perrin EB, Roberts JS. Evaluating quality-of-life and health status instruments: Development of scientific review criteria. Clin Ther 1996;18:979–992.

    Article  CAS  PubMed  Google Scholar 

  3. Scientific Advisory Committee. Instrument Review Criteria. Med Outcomes Trust Bull 1995;3:I–IV.

    Google Scholar 

  4. Staquet M, Berzon R, Osoba D, Machin D. Guidelines for reporting results of quality of life assessments in clinical trials. Qual Life Res 1996;5:496–500.

    Article  CAS  PubMed  Google Scholar 

  5. Fayers PM, Hopwood P, Harvey A, Girling DJ, Machin D, Stephens R, on Behalf of the MR. Cancer Trials Office. Quality of Life Assessment in Clinical Trials—Guidelines and a Checklist for Protocol Writers: The U. Medical Research Council Experience. Eur J Cancer 1997;33:20–28.

    Article  CAS  PubMed  Google Scholar 

  6. Revicki DA, Leidy NK, Geneste B. Recommendations for evaluating the validity of quality of life claims for labeling and promotion. Value Health 1999;2:113–127.

    Article  PubMed  Google Scholar 

  7. Smith N. Quality of life studies from the perspective of an FD. reviewing statistician. Drug Inf J 1993;27:617–623.

    Article  Google Scholar 

  8. Sanders C, Egger M, Donovan J, Tallon D, Frankel S. Reporting on quality of life in randomised controlled trials: Bibliographic study. Br Med J 1998;317:1191–1194.

    Article  CAS  Google Scholar 

  9. Chassany O, Bergmann JF, Caulin C. Reporting on quality of life in randomised controlled trials. Br Med J 1999:318:1142.

    Article  CAS  Google Scholar 

  10. Kong SX, Gandhi SK. Methodologic assessments of quality of life measures in clinical trials. Ann Pharmacother 1997;31:830–836.

    Article  CAS  PubMed  Google Scholar 

  11. Brown RS. Strategies and pitfalls in quality of life research. Hepatology 1999;29(Suppl. 1):9S–12S.

    Google Scholar 

  12. de Haes J, Curran D, Young T, Bottomley A, Flechtner H, Aaronson N, Blazeby J, Bjordal K, Brandberg Y, Greimel E, Maher J, Sprangers M, Cull A. Quality of life evaluation in oncological clinical trials—the EORTC model. The EORTC quality of life study group. Eur J Cancer 2000;36:821–825.

    Article  PubMed  Google Scholar 

  13. Chassany O. Does dehydroepiandrosterone improve well-being? Presse Med. 2000;29:1354–1355.

    CAS  PubMed  Google Scholar 

  14. Schipper H, Clinch J, Powell V. Definition and Conceptual Issues. In: Spilker B, ed. Quality of Life Assessments in Clinical Trials. New York, NY: Raven Press; 1990:11–24.

    Google Scholar 

  15. Spitzer WO. State of science 1986: Quality of life and functional status as target variables for research. J Chronic Diseases 1987;6:465–471.

    Article  Google Scholar 

  16. Sullivan M, Karlsson J, Taft C. How to Assess Quality of Life in Medicine: Rationale and Methods. In: B Guy-Grand, G Ailhaud, eds. Progress in Obesity Research:8. London: Libbey; 1999:749–755.

    Google Scholar 

  17. World Health Organization. World Health Organization Constitution. Basic Documents. Geneva, Switzerland: World Health Organization; 1948.

    Google Scholar 

  18. Patrick DL, Erickson P. Health Status and Health Policy: Allocating Resources to Health Care. New York, NY: Oxford University Press; 1993.

    Google Scholar 

  19. Burke L. Acceptable evidence for pharmaceutical advertising and labeling, DIA Workshop on Pharmacoeconomic and Quality of Life Labeling and Marketing Claims, October 3, 2000, New Orleans, LA.

    Google Scholar 

  20. Aaronson NK. Quality of life assessment in clinical trials: Methodologic issues. Control Clin Trials 1989;10:S195–S208.

    Article  Google Scholar 

  21. Testa MA, Simonson DC. Assessment of quality of life outcomes. N Engl J Med 1996:334:835–840.

    Article  CAS  Google Scholar 

  22. Revicki DA, Ehreth JL. Health-related quality-of-life assessment and planning for the pharmaceutical industry. Clin Ther 1997;19:1101–1115.

    Article  CAS  PubMed  Google Scholar 

  23. Feinstein AR. Clinimetric perspectives. J Chronic Diseases 1987;40:635–640.

    Article  CAS  Google Scholar 

  24. Bulpitt CJ, Fletcher AE. The measurement of quality of life in hypertensive patients: A practical approach. Br J Clin Pharmacol 1990;30:353–364.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  25. Wiklund I, Dimenäs E, Wahl M. Factors of importance when evaluating quality of life in clinical trials. Control Clin Trials 1990;11:169–179.

    Article  CAS  PubMed  Google Scholar 

  26. Wiklund I. Aspects of quality of life in gastrointestinal disease: Some methodological issues. Scand J Gastroenterol 1995;208:129–132.

    Article  CAS  Google Scholar 

  27. Trus TL, Laycock WS, Branum GD, Waring JP, Hunter JG. Quality of life scores correlate poorly with subjective and objective measurements of gastroesophageal reflux. Gastroenterol 1997;112:A1480.

    Google Scholar 

  28. Guyatt GH, Thompson PJ, Berman LB, Sullivan MJ, Townsend M, Jones NL, Pugsley SO. How should we measure function in patients with chronic heart and lung disease? J Chronic Diseases. 1985;38:517–524.

    Article  CAS  Google Scholar 

  29. Rector TS, Kubo SH, Cohn JN. Patients’ Self-assessment of their cognitive heart failure: II: Content, reliability and validity of a new measure. The Minnesota Living with Heart Failure Questionnaire. Heart Failure 1987;3:198–209.

    Google Scholar 

  30. Juniper EF, Guyatt GH, Ferrie PJ, Griffith LE. Measuring quality of life in asthma. Am Rev Respir Dis 1993;147:832–838.

    Article  CAS  PubMed  Google Scholar 

  31. Wiklund I, Comerford MB, Dimenas E. The relationship between exercise tolerance and quality of life in angina pectoris. Clin Cardiol 1991;14:204–208.

    Article  CAS  PubMed  Google Scholar 

  32. Guyatt GH, Feeny DH, Patrick DL. Measuring Health-related quality of life. Ann Int Med 1993;118:622–629.

    Article  CAS  PubMed  Google Scholar 

  33. Stewart AL, Greenfield S, Hays RD, Wells K, Rogers WH, Berry SD, McGlynn EA, Ware JE. Functional status and well-being of patients with chronic conditions. Results from the Medical Outcomes Study. JAMA 1989;262:907–913.

    Article  CAS  PubMed  Google Scholar 

  34. Bayliss MS, Gandek B, Bungay KM, Sugano D, Hsu MA, Ware JE. A questionnaire to assess the generic and disease-specific health outcomes of patients with chronic hepatitis C. Qual Life Res 1998;7:39–55.

    Article  CAS  PubMed  Google Scholar 

  35. Ganz PA, Coscarelli A, Fred C, Kahn B, Plinski ML, Petersen L. Breast cancer survivors: Psychological concerns and quality of life. Breast Cancer Res Treat 1996;38:183–199.

    Article  CAS  PubMed  Google Scholar 

  36. Fletcher A, Gore S, Jones D, Fiztpatrick R, Spiegelhalter D, Cox D. Quality of life measures in health care. II: Design, analysis, and interpretation. Br Med J 1992;305:1145–1148.

    Article  CAS  Google Scholar 

  37. Gill TM, Feinstein AR. A critical appraisal of the quality of quality of life measurements. JAMA 1994;272:619–626.

    Article  CAS  PubMed  Google Scholar 

  38. Patrick DL, Deyo RA. Generic and disease-specific measures in assessing health status and quality of life. Med Care 1989;27(Suppl 3):S217–S232.

    Article  Google Scholar 

  39. Goldstein RS, Gort EH, Guyatt GH, Stubbing D, Avendano MA. Prospective randomized controlled trial of respiratory rehabilitation. Lancet 1994;344:1394–1397.

    Article  CAS  PubMed  Google Scholar 

  40. Tandon PK, Stander H, Schwarz RPJ. Analysis of quality of life data from a randomized, placebo controlled heart-failure trial. J Clin Epidemiol 1989;42:955–962.

    Article  CAS  PubMed  Google Scholar 

  41. Laupacis A, Wong C, Churchill D. The use of generic and specific quality-of-life measures in hemodialysis patients treated with Erythropoietin. Control Clin Trials 1991;121:68S–79S.

    Google Scholar 

  42. Galmiche JP, Barthelemy P, Hamelin B. Treating the symptoms of gastro-oesophageal reflux disease: A double-blind comparison of Omeprazole and Cisapride. Aliment Pharmacol Ther 1997;11:765–773.

    Article  CAS  PubMed  Google Scholar 

  43. Tarter RE, Switala J, Arria A, Plail J, Van Thiel D. Quality of life before and after orthotopic hepatic transplantation. Arch Int Med 1991;151:1521–1526.

    Article  CAS  Google Scholar 

  44. Chassany O, Marquis P, Scherrer B, Read NW, Finger T, Bergmann JF, Fraitag B, Geneve J, Caulin C. Validation of a specific quality of life questionnaire in Functional Digestive Disorders (FDDQL). Gut 1999;44:527–533.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  45. Bergner M, Rofhman ML. Health status measures: an overview and guide for selection. Ann Rev Public Health 1987;8:191–210.

    Article  CAS  Google Scholar 

  46. Wiklund I. Quality of life and regulatory issues. Scand J Gastroenterol 1996;31(Suppl. 221):37–38.

    Article  Google Scholar 

  47. Testa MA, Nackley JF. Methods for quality-of-life studies. Ann Rev Public Health 1994;15:535–559.

    Article  CAS  Google Scholar 

  48. Guyatt GH, Kirshner B, Jaeschke R. Measuring health status: What are the necessary measurement properties? J Clin Epidemiol. 1992;45:1341–1345.

    Article  CAS  PubMed  Google Scholar 

  49. Hays R, Anderson R, Revicki DA. Assessing Reliability and Validity of Measurement in Clinical Trials. In: Staquet MJ, Hays RD, Fayers PM, eds. Quality of Life Assessment in Clinical Trials. New York, NY: Oxford University Press; 1998: 169–182.

    Google Scholar 

  50. Fletcher A. Quality-of-life measurements in the evaluation of treatment: Proposed guidelines. Br J Clin Pharmacother 1995;39:217–222.

    Article  CAS  Google Scholar 

  51. Marquis P, Fayol C, Joire JE, Leplège A. Psychometric properties of a specific quality of life questionnaire in angina pectoris patients. Qual Life Res 1995;4:540–546.

    Article  CAS  PubMed  Google Scholar 

  52. Jenkinson C, Ziebland S, Fitzpatrick R, Mowat A. Sensitivity to change of weighted and unweighted versions of two health status measures. Intl J Health Sciences 1991;2:189–194.

    Google Scholar 

  53. Zimmerman M. Weighted versus unweighted life event scores: Is there a difference? J Human Stress. 1983;9:30–35.

    Article  CAS  PubMed  Google Scholar 

  54. Fitzpatrick R, Fletcher A, Gore S, Jones D, Spiegel-halter D, Cox D. Quality of life measures in health care. I: Applications and issues in assessment. Br Med J 1992;305:1074–1077.

    Article  CAS  Google Scholar 

  55. Kirschner B, Guyatt GH. A methodologic framework for assessing health indices. J Chronic Diseases 1985;38:27–36.

    Article  Google Scholar 

  56. Guyatt G, Mitchell A, Irvine EJ, Singer J, Williams N, Goodacre R, Tompkins C. A new measure of health status for clinical trials in inflammatory bowel disease. Gastroenterol 1989;96:804–810.

    Article  CAS  Google Scholar 

  57. Eypasch E, Williams JI, Wood-Dauphinee S, Ure BM, Schmiilling C, Neugebauer E, Troidl H. Gastrointestinal Quality of Life Index: Development, validation and application of a new instrument. Br J Surg 1995;82:216–222.

    Article  CAS  PubMed  Google Scholar 

  58. Scharloo M, Kaptein AA, Weinman JA, Willems LNA, Rooijmans HGM. Physical and psychological correlates of functioning in patients with chronic obstructive pulmonary disease. J Asthma 2000;37:17–29.

    Article  CAS  PubMed  Google Scholar 

  59. Faller H, Bulzebruck H, Drings P, Lang H. Coping, distress, and survival among patients with lung cancer. Arch Gen Psychiatry 1999;56:756–762.

    Article  CAS  PubMed  Google Scholar 

  60. Stockier MR, Osoba D, Corey P, Goodwin PJ, Tannock IF. Convergent, discriminative, and predictive validity of the Prostate Cancer Specific Quality of Life Instrument (PROSQOLI) assessment and comparison with Analogous Scales Form the EORTC QLQ-C30 and a trial-specific module. J Clin Epidemiol 1999;52:653–666.

    Article  Google Scholar 

  61. Feagan BG, McDonald JW, Rochon J, Laupacis A, Fedorak RN, Kinnear D, Saibil F, Groll A, Archambault A, Gillies R, Valberg B, Irvine JE, for the Canadian Crohn’s Relapse Prevention Trial Investigators. Low-dose Cyclosporine for the treatment of Crohn’s Disease. N Engl J Med 1994;330:1846–1851.

    Article  CAS  PubMed  Google Scholar 

  62. Guyatt GH, Walter S, Norman G. Measuring health status over time. Assessing the usefulness of evaluative instruments. J Chronic Diseases 1987;40:171–178.

    Article  CAS  Google Scholar 

  63. Kazis LE, Anderson JJ, Meenan RF. Effect sizes for interpreting changes in health status. Med Care 1989;27(Suppl. 3):S178–S189.

    Article  CAS  PubMed  Google Scholar 

  64. Deyo RA, Diehr P, Patrick DL. Reproducibility and responsiveness of health status measures. Statistics and strategies for evaluation. Control Clin Trials 1991;12:S142–SI58.

    Article  Google Scholar 

  65. Acquadro C, Jambon B, Ellis D, Marquis P. Languages and Translation Issues. In: Spilker B, ed. Quality of Life and Pharmacoeconomics in Clinical Trials. Second edition. Philadelphia, PA: Lippincott-Raven Publishers; 1996:575–585.

    Google Scholar 

  66. Bullinger M, Anderson R, Cella D, Aaronson N. Developing and evaluating cross-cultural instruments from minimum requirements to optimal models. Qual Life Res 1993;2:451–459.

    Article  CAS  PubMed  Google Scholar 

  67. Hunt SM. Cross-cultural Issues in the Use of Quality of Life Measures in Randomized Clinical Trials. In: Staquet MJ, Hays RD, Fayers PM, eds. Quality of Life Assessment in Clinical Trials: Methods and Practice. New York, NY: Oxford University Press; 1998: 51–67.

    Google Scholar 

  68. Bullinger M, Alonso J, Apolone G, Leplege A, Sullivan M, Wood-Dauphinee S, Gandek B, Wagner A, Aaronson N, Bech P, Fukuhara S, Kaasa S, Ware J. Jr. Translating health status questionnaires and evaluating their quality: the IQOLA project approach. International Quality of Life Assessment. J Clin Epidemiol 1998;51:913–923.

    Article  CAS  PubMed  Google Scholar 

  69. Guillemin F, Bombardier C, Beaton D. Cross-cultural adaptation of health-related quality of life measures: Literature review and proposed guidelines. J Clin Epidemiol 1993;46:1417–1432.

    Article  CAS  PubMed  Google Scholar 

  70. Gandek B, Ware J. Jr, Aaronson NK, Alonso J, Apolone G, Bjorner JB, Brazier J, Bullinger M, Fukuhara S, Kaasa S, Leplege A, Sullivan M. Tests of data quality, scaling assumptions, and reliability of the SF-36 in eleven countries: Results from the IQOLA project. J Clin Epidemiol 1998;51:1149–1158.

    Article  CAS  PubMed  Google Scholar 

  71. Spilker B. Quality of Life Trials. In: Spilker B, ed. Guide to Clinical Trials. New York, NY: Raven Press; 1991:370–378.

    Google Scholar 

  72. Bernhard J, Cella DF, Coates CA, Fallowfield L, Ganz PA, Moinpour CM, Mosconi P, Osoba D, Simes J, Hürny C. Missing quality data in cancer clinical trials: Serious problems and challenges. Stat Med 1998;17:517–532.

    Article  CAS  PubMed  Google Scholar 

  73. Osoba D. Rationale for the timing of health-related quality-of-life assessments in oncological palliative therapy. Cancer Treat Rev 1996;22(Suppl.A):69–73.

    Article  PubMed  Google Scholar 

  74. Rothman ML, Hedrick SC, Bulcroft KA, Hickam DH, Rubenstein LZ. The validity of proxy-generated scores as measures of patient health status. Med Care 1991;29:115–124.

    Article  CAS  PubMed  Google Scholar 

  75. Sneeuw KCA, Aaronson NK, Osoba D, Muller MJ, Hsu MA, Yung WK, Brada M, Newlands ES. The use of significant others as proxy raters of the quality of life of patients with brain cancer. Med Care 1997;35:490–506.

    Article  CAS  PubMed  Google Scholar 

  76. Sprangers MAG, Aaronson NK. The role of health care providers and significant others in evaluating the quality of life of patients with chronic disease: a review. J Clin Epidemiol 1992;45:743–760.

    Article  CAS  PubMed  Google Scholar 

  77. Testa MA, Hollenberg NK, Anderson RB, Williams GH. Assessment of quality of life by patient and spouse during antihypertensive therapy with Atenolol and Nifedipine gastrointestinal therapeutic system. Am J Hypertens 1991;4:363–373.

    Article  CAS  PubMed  Google Scholar 

  78. Mathias SD, Bates MM, Pasta DJ, Cisternas MG, Feeny D, Patrick DL. Use of the health utilities index with stroke patients and their caregivers. Stroke 1997;28:1888–1894.

    Article  CAS  PubMed  Google Scholar 

  79. Sneeuw KCA, Aaronson NK, Sprangers MAG, Detmar SB, Wever LDV, Schornagel JH. Value of caregiver ratings in evaluating the quality of life of patients with cancer. J Clin Oncol 1997;15:1206–1217.

    Article  CAS  PubMed  Google Scholar 

  80. Sneeuw KCA, Aaronson NK, de Haan RJ, Limburg M. Assessing quality of life after stroke. The value and limitations of proxy ratings. Stroke 1997;28:1541–1549.

    Article  CAS  PubMed  Google Scholar 

  81. Stephens RJ, Hopwood P, Girling DJ, Machin D. Randomized trials with quality of life endpoints. Are doctor’s ratings of patients’ physical symptoms interchangeable with patients’ self-ratings? Qual Life Res. 1997;6:225–236.

    Article  CAS  PubMed  Google Scholar 

  82. Simes RJ, Greatorex V, Gebski VJ. Practical approaches to minimize problems with missing quality of life data. Stat Med 1998;17:725–737.

    Article  CAS  PubMed  Google Scholar 

  83. Statistical Principles for Clinical Trials. ICH Harmonised Tripartite Guideline. CPMP/ICH/363/96. http://www.eudra.org/humandocs/humans/ICH.htm.

  84. Lydick E, Epstein RS. Clinical Significance of Quality of Life Data. In: Spilker B, nted. Quality of Life and Pharmacoeconomics in Clinical Trials. Philadelphia, PA: Lippincott-Raven Publishers; 1996: 461–465.

    Google Scholar 

  85. Chassany O, Duracinsky M. Ethics and clinical trials. Fundam Clin Pharmacol 1999;13:437–444.

    Article  CAS  PubMed  Google Scholar 

  86. Rush DR, Stelmach J, Young TL, Kirchdoefer LJ, Scott-Lennox J, Holverson HE, Sabesin SM, Nicholas TA. Clinical effectiveness and quality of life with ranitidine vs placebo in gastroesophageal reflux disease patients: a clinical experience network (CEN) Study. J Fam Pract 1995;41:126–136.

    CAS  PubMed  Google Scholar 

  87. Juniper EF. Quality of life questionnaires: Does statistically significant = clinically important? J Allergy Clin Immunol. 1998;102:16–17.

    Article  CAS  PubMed  Google Scholar 

  88. Lydick E, Epstein RS. Interpretation of quality of life changes. Qual Life Res 1993;2:221–226.

    Article  CAS  PubMed  Google Scholar 

  89. Offerhaus L. Measurement of the quality of life in clinical trials: in pursuit of the unapproachable. Eur J Clin Pharmacol 1991;40:205–208.

    Article  CAS  PubMed  Google Scholar 

  90. Curran D, Fayers PM, Molengerghs G, Machin D. Analysis of Incomplete Quality of Life Data in Clinical Trials. In: Staquet MJ, Hays RD, Fayers PM, eds. Quality of Life Assessment in Clinical Trials. New York, NY: Oxford University Press; 1998:249–280.

    Google Scholar 

  91. Fairclough D, Peterson H, Cella D, Bonomi P. Comparison of several model-based methods for analyzing incomplete quality of life data in cancer clinical trials. Stat Med 1998;17:781–796.

    Article  CAS  PubMed  Google Scholar 

  92. Hayden KA, Moinpour CM, Metch B, Feigl P, O’Brian RM, Green S, Osborne CK. Pitfalls in quality of life assessment: Lessons from a Southwest Oncology Group breast cancer clinical trial. Oncol Nurs Forum 1993;20:1415–1419.

    CAS  PubMed  Google Scholar 

  93. Olschewski M. Compliance with QoL assessment in multicentre German breast cancer trials. Stat Med 1998;17:571–575.

    Article  CAS  PubMed  Google Scholar 

  94. Wiklund I, Junghard O, Grace E, Talley NJ, Kamm M, Veldhuyzen van Zanten S, Paré P, Chiba N, Leddin DS, Bigard MA, Colin R, Schoenfeld P. Quality of life in reflux and dyspepsia patients. Psychometric documentation of a new disease-specific questionnaire (QOLRAD). Eur J Surg. 1998:83(Suppl.583):41–49.

    Google Scholar 

  95. Machin D, Weeden S. Suggestions for the presentation of quality of life data from clinical trials. Stat Med 1998;17:711–724.

    Article  CAS  PubMed  Google Scholar 

  96. Troxel AB, Fairclough DL, Curran D, Hahn EA. Statistical analysis of quality of life with missing data in cancer clinical trials. Stat Med 1998;17:517–532.

    Article  Google Scholar 

  97. Fairclough DL, Gelber RD. Quality of Life: Statistical Issues and Analysis. In: Spilker B, ed. Quality of Life and Pharmacoeconomics in Clinical Trials. Philadelphia, PA: Lippincott-Raven Publishers; 1996:427–435.

    Google Scholar 

  98. Guyatt GH, Juniper EF, Walter SD, Griffith LE, Goldstein RS. Interpreting treatment effects in randomised trials. Br Med J 1998;316:690–693.

    Article  CAS  Google Scholar 

  99. Jaeschke R, Singer J, Guyatt GH. Measurement of Health Status: Ascertaining the Minimal Clinically Important Difference. Control Clin Trials 1989;10:407–415.

    Article  CAS  PubMed  Google Scholar 

  100. Osoba D, Rodrigues G, Myles J, Zee B, Pater J. Interpreting the significance of changes in health-related quality-of-life scores. J Clin Oncol 1998;16:139–144.

    Article  CAS  PubMed  Google Scholar 

  101. Juniper EF, Guyatt GH, Willan A, Griffith LE. Determining a minimal important change in a disease-specific quality of life questionnaire. J Clin Epidemiol 1994;47:81–87.

    Article  CAS  PubMed  Google Scholar 

  102. Mahajan P, Pearlman D, Okamoto L. The effect of Fluticasone Propionate on functional status and sleep in children with asthma and on the quality of life of their parents. J Allergy Clin Immunol 1998;102:19–23.

    Article  CAS  PubMed  Google Scholar 

  103. Samsa G, Edelman D, Rothman ML, Williams GR, Lipscomb J, Matchar D. Determining clinically important differences in health status measures. PharmacoEconomics 1999;15:141–155.

    Article  CAS  PubMed  Google Scholar 

  104. Wyrwich KW, Nienaber NA, Tierney WM, Wolinsky FD. Linking clinical relevance and statistical significance in evaluating intra-individual changes in health-related quality of life. Med Care 1999;37:469–478.

    Article  CAS  PubMed  Google Scholar 

  105. Ware JE, Snow KK, Kosinski M, Gandek B. SF-36 Health Survey: Manual and Interpretation Guide. Boston, MA: The Health Institute, New England Medical Center; 1993.

    Google Scholar 

  106. Thompson MS, Read JL, Hutchings HC, Paterson M, Harris ED. The cost effectiveness of Auranofin: Results of a randomized clinical trial. J Rheumatol 1988;15:35–42.

    CAS  PubMed  Google Scholar 

  107. Ware JE, Keller SD. Interpreting General Health Measures. In: Spilker B, ed. Quality of Life and Pharmacoeconomics in Clinical Trials. Philadelphia, PA: Lippincott-Raven Publishers; 1996:445–460.

    Google Scholar 

  108. Cook RJ, Sackett DL. The number needed to treat: a clinically useful measure of treatment effect. Br Med J 1995;310:452–454.

    Article  CAS  Google Scholar 

  109. Altman DG. Confidence intervals for the number needed to treat. Br Med J 1998;317:1309–1312.

    Article  CAS  Google Scholar 

  110. Juniper EF. The value of quality of life in asthma. Eur Respir Rev 1997;49:333–337.

    Google Scholar 

  111. Liard F, Benichou AC, Gamand S, Lehert P. The effects of Naftidrofuryl on quality of life. Disease Manage Health Outcomes 1997;2(Suppl.1):71–78.

    Article  Google Scholar 

  112. Altman DG, Bland JM. Absence of evidence is not evidence of absence. Br Med J 1995;311:485–486.

    Article  CAS  Google Scholar 

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Correspondence to Olivier Chassany MD, PhD.

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Members of the European Regulatory Issues on Quality of Life Assessment (ERIQA) Group: Dr. Neil Aaronson, The Netherlands Cancer Institute, The Netherlands; Dr. Catherine Acquadro, Coordinator of the ERIQA Group, Mapi Research Institute, France; Dr. Giovanni Apolone, Mario Negri Institute, Italy; Dr. Harry Burns, Dalian House, Glasgow, UK; Dr. Olivier Chassany, Hopital Saint-Louis, France; Dr. Gianfranco De Carli, GlaxoWellcome, Italy; Dr. Dominique Dubois, Janssen Pharmaceutica N.V., Belgium; Steve Fullerton, Cedars-Sinai Health System/Zynx Health Incorporated, US; Dr. Bernard Geneste, Aventis, France; Dr. Asha Hareendran, Pfizer Central Research, UK; Bernard Jambon, Mapi Group, France; Dr. Patrick Marquis, Mapi Values, US; Dr. Pauline McNulty, ICOM Health Economics, Johnson & Johnson, US; Dr. Caroline Miltenburger, SCHERING AG, Germany; Dr. Annoesjka Novak, NV Organon, The Netherlands; Dr. Margaret L. Rothman, Janssen Research Foundation, US; Dr. Pierre Philippe Sagnier, Bayer plc, Medical Affairs, UK; Dr. François Schubert, GlaxoWellcome Research & Development, UK; Dr. Soren E. Skovlund, Novo Nordisk, Denmark; Dr. Marianne Sullivan, Goteborg University, Sweden; Dr. Marc Tomas, Outcomes Research BMS, Belgium; Dr. Suzanne Wait, Bristol Myers Squibb, UK; Dr. Ingela Wiklund, AstraZeneca R&D, Sweden; and Dr. G. Rhys Williams, Knoll Pharmaceutical Company, US.

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Chassany, O., Sagnier, P., Marquis, P. et al. Patient-Reported Outcomes: The Example of Health-Related Quality of Life—a European Guidance Document for the Improved Integration of Health-Related Quality of Life Assessment in the Drug Regulatory Process. Ther Innov Regul Sci 36, 209–238 (2002). https://doi.org/10.1177/009286150203600127

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