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Quantitative challenges facing patient-centered outcomes research

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References

  • Washington EA, Lipstein SH. The patient-centered outcomes research institute – promoting better information, decisions, and health. N Engl J Med 2011;365:e31
  • Lee EO, Emanuel EJ. Shared decision making to improve care and reduce costs. N Engl J Med 2013;368:6-8
  • Fleurence R, Selby JV, Odom-Walker K, et al. How the patient-centered outcomes research institute is engaging patients and others in shaping its research agenda. Health Aff 2013;32:2393-400
  • Ridgeway JL, Beebe TJ, Chute CG, et al. A brief patient-reported outcomes quality of life (PROQOL) instrument to improve patient care. PloS Med 2013;10(11):e1001548
  • Oliver A, Greenberg CC. Measuring outcomes in oncology treatment: the importance of patient-centered outcomes. Surg Clin North Am 2009;89(1):17-25, vii
  • Feurer ID, Russell RT, Pinson CW. Incorporating quality of life and patient satisfaction measures into a transplant outcomes assessment program: technical and practical considerations. Prog Transplant 2007;17(2):121-8
  • Patient-Centered Outcomes Research Institute Methodology Committee. 2013.The PCORI Methodology Report. Available from: pcori.org/research-we-support/research-methodology-standards [Last accessed 20 March 2014]
  • US FDA. Guidance for industry. Patient-reported outcome measures: use in medical product development to support labeling claims. (December 2009) Available from: www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM193282.pdf [Last accessed 4 June 2013]
  • Ahmed S, Berzon RA, Revicki DA, International Society for Quality of Life Research. The use of patient-reported outcomes (PRO) within comparative effectiveness research: implications for clinical practice and health care policy. Med Care 2012;50(12):1060-70
  • Patrick DL, Burke LB, Gwaltney CJ, et al. Content validity – establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO Good Research Practices Task Force report: part 1 – eliciting concepts for a new PRO instrument. Value Health 2011;4:967-77
  • Patrick DL, Burke LB, Gwaltney CJ, et al. Content validity – establishing and reporting the evidence in newly developed patient-reported outcomes (PRO) instruments for medical product evaluation: ISPOR PRO Good Research Practices Task Force report: part 2 – assessing respondent understanding. Value Health 2011;14:978-88
  • Reeve BB, Wyrwich KW, Wu AW, et al. ISOQOL recommends minimum standards for patient-reported outcome measures used in patient-centered outcomes and comparative effectiveness research. Qual Life Res 2013;22(8):1889-905
  • Rabin R, de Charro F. EQ-5D: a measure of health status from the EuroQol Group. Ann Med 2001;33(5):337-43
  • Ware JE, Kosinski M, Dewey JE. In: Lincoln RI, editor. How to score version two of the SF-36 Health Survey. QualityMetric, Incorporated; 2000
  • Krueger RF. The structure of common mental disorders. Arch Gen Psychiatry 1999;56:921-6
  • Reeve B, Hays RD, Bjorner J, et al. the PROMIS cooperative group. Psychometric evaluation and calibration of health–related quality of life item banks: plans for the patient–reported outcome measurement information system (PROMIS). Med Care 2007;45(5):S22-31
  • Revicki DA, Cook KF, Amtmann D, et al. Exploratory and confirmatory factor analysis of the PROMIS pain quality item bank. Qual Life Res 2014;23(1):245-55
  • Slade T, Watson D. The structure of common DSM-IV and ICD-10 mental disorders in the Australian general population. Psychol Med 2006;36:1593-600
  • Gibbons RD, Hedeker D. Full-information item bi-factor analysis. Psychometrika 1992;57:423-36
  • Reise S, Morizot J, Hays R. The role of the bifactor model in resolving dimensionality issues in health outcomes measures. Qual Life Res 2007;16:19-31
  • Leidy NK, Sexton CC, Jones P, et al. Measuring respiratory symptoms in clinical trials of COPD: reliability and validity of a daily diary. Thorax 2014. [Epub ahead of print]
  • Cai L. High-dimensional exploratory item factor analysis by a Metropolis-Hastings Robbins-Monro algorithm. Psychometrika 2010;75:33-57
  • Hao Y. Patient-reported outcomes in support of oncology product labeling claims: regulatory context and challenges. Expert Rev Pharmacoecon Outcomes Res 2010;10(4):407-20
  • Alemayehu D, Sanchez R, Cappelleri JC. Considerations on the use of patient-reported outcomes in comparative effectiveness research. J Manag Care Pharm 2011;17(9-a):S27-33
  • EMA Committee for Medicinal Products for Human Use (CHMP). Reflection paper on the regulatory guidance for the use of health related quality of life (HRQL) measures in the evaluation of medicinal products. Doc Ref EMA/CHMP/EWP/139391/2004 EMA; London, UK: 2005
  • Committee for Proprietary Medicinal Products (CPMP). Points to consider on multiplicity issues in clinical trials 2010. Available from: www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003640.pdf [Last accessed 21 March 2014]
  • Nunnally JC, Bernstein IH. Psychometric theory. 3rd edition McGraw-Hill; NY, USA: 1994
  • Sébille V, Hardouin J, Le Néel T, et al. Methodological issues regarding power of classical test theory (CTT) and item response theory (IRT)-based approaches for the comparison of patient-reported outcomes in two groups of patients–a simulation study. BMC Med Res Methodol 2010;10:24
  • Blanchin M, Hardouin J-B, Guillemin F, et al. Power and sample size determination for the Group Comparison of Patient-Reported outcomes with rasch family models. PLoS One 2013;8:e57279
  • MacCallum RC, Widaman KF, Zhang S, Hong S. Sample size in factor analysis. Psychological Methods 1999;4:84-99
  • Chen WH, Lenderking W, Jin Y, et al. Is Rasch model analysis applicable in small sample size pilot studies for assessing item characteristics? An example using PROMIS pain behavior item bank data. Qual Life Res 2014;23(2):485-93
  • Andrich D, Luo G, Sheridan B. Interpreting RUMM2020. RUMM Laboratory; Perth, WA, USA: 2004
  • Chow S. Sample size calculations for clinical trials. In: Interdisciplinary reviews: computational statistics. John Wiley & Sons; NY, USA: 2011. p. 414-27
  • Röhrig B, du Prel JB, Wachtlin D, et al. Sample size calculation in clinical trials: part 13 of a series on evaluation of scientific publications. Dtsch Arztebl Int 2010;107(31-32):552-6
  • National Research Council. The prevention and treatment of missing data in clinical trials. The National Academies Press; Washington DC, UK: 2010
  • Rubin DB. Interference and missing data. Biometrika 1976;63(3):581-92
  • Little RJA, Rubin DB. Statistical analysis with missing Data. 2nd edition John Wiley; NY, USA: 2002
  • Rubin DB. Multiple Imputation for Nonresponse in Surveys. John Wiley & Sons, Inc; NY, USA: 1987
  • Schafer JL. Analysis of incomplete multivariate data. Chapman and Hall; London, UK: 1997
  • Diggle P, Kenward MG. Informative dropout in longitudinal analysis. Appl Statist 1994;43(1):49-93
  • Robins JM, Rotnitzky A, Zhao LP. Analysis of semiparametric regression models for repeated outcomes in the presence of missing data. J Am Statist Assoc 1995;90:106-21
  • Rotnitzky A, Robins JM, Scharfstein DO. Semiparametric regression for repeated outcomes with nonignorable nonresponse. J Am Statist Assoc 1998;93:1321-39
  • Little RJA. Pattern-mixture models for multivariate incomplete data. J Am Statist Assoc 1993. 88:125-34
  • Little RJA. A class of pattern mixture models for normal missing data. Biometrika 1994;81:471-83
  • Eekhout I, de Boer R, Twisk JW, et al. Missing data: a systematic review of how they are reported and handled. Epidemiology 2012;23:729e32
  • Eekhout I, de Vet HC, Twisk JW, et al. Missing data in a multi-item instrument were best handled by multiple imputation at the item score level. J Clin Epidemiol 2014;67(3):335-42
  • Fayers PM, Curran D, Machin D. Incomplete quality of life data in randomized trials: missing items. Stat Med 1998;17(5-7):679-96
  • Shrive FM, Stuart H, Quan H, Ghali WA. Dealing with missing data in a multi-question depression scale: a comparison of imputation methods. BMC Med Res Method 2006;6:57
  • Frost MH, Reeve BB, Liepa AM, Mayo/FDA Patient-Reported Outcomes Consensus Meeting Group. What is sufficient evidence for the reliability and validity of patient-reported outcome measures? Value Health 2007;10(Suppl 2)):S94-S105
  • Fitzpatrick R, Davey C, Buxton MJ, Jones DR. Evaluating patient-based outcome measures for use in clinical trials. Health Technol Assess 1998;2:14
  • Hays RD, Revicki DA. Assessing reliability and validity of measurement in clinical trials. In: Fayers P, Hays R, editors. Assessing quality of life in clinical trials. Oxford University Press; NY, USA: 2005. p. 25-40
  • Revicki D, Hays RD, Cella D, et al. Recommended methods for determining responsiveness and minimally important differences for patient-reported outcomes. J Clin Epidemiol 2008;61:102-9
  • McLeod LD, Coon CD, Martin SA, et al. Interpreting patient-reported outcome results: US FDA guidance and emerging methods. Exp Rev Pharmacoecon Outcomes Res 2011;11(2):163-9
  • Jaeschke R, Singer J, Guyatt GH. Measurement of health status. Ascertaining the minimal clinically important difference. Control Clin Trials 1989;10:407-15
  • 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;47:81-7
  • Osoba D, King MT. Interpreting quality of life in individuals and groups: meaningful differences. In: Fayers P, Hays R, editors. Assessing quality of life in clinical trials. Oxford University Press; NY, USA: 2005. p. 243-57
  • Terwee CB, Roorda LD, Knol DL, et al. Linking measurement error to minimal important change of patient-reported outcomes. J Clin Epidemiol 2009;62(10):1062-7
  • Wyrwich KW, Norquist JM, Lenderking WR, Acaster S; Industry Advisory Committee of International Society for Quality of Life Research (ISOQOL). Methods for interpreting change over time in patient-reported outcome measures. Qual Life Res 2013;22(3):475-83
  • Cappelleri JC, Hwang LJ, Mardekian J, et al. Cumulative distribution functions of sildenafil citrate on exercise capacity and hemodynamic function in children with pulmonary arterial hypertension. J Pulmon Resp Med 2013;S4:002
  • FDA. Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims. Draft guidance. (February 2006). Available from: www.ispor.org/workpaper/FDAPROGuidance2006.pdf [Last accessed 25 January 2014]
  • Wyrwich KW, Tierney WM, Wolinsky FD. Further evidence supporting an SEM-based criterion for identifying meaningful intra-individual changes in health-related quality of life. J Clin Epidemiol 1999;52(9):861-73
  • Leidy NK, Wyrwich KW. Bridging the gap: using triangulation methodology to estimate minimal clinically important differences (MCIDs). COPD 2005;2(1):157-65
  • Ljungberg AK, Fossum B, Fürst CJ, Hagelin CL. Translation and cultural adaptation of research instruments – guidelines and challenges: an example in FAMCARE-2 for use in Sweden. Inform Health Soc Care 2014. [Epub ahead of print]
  • 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;46:1417-32
  • Marquis P, Keininger D, Acquadro C, de la Loge C. Translating and evaluating questionnaires: cultural issues for international research. In: Fayers P, Hays R, editors. Assessing Quality of Life in Clinical Trials. Oxford University Press; NY, USA: 2005. p. 77-93
  • Wild D, Grove A, Martin M, ISPOR Task Force for Translation and Cultural Adaptation. 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 20058(2):94-104
  • Zbrozek A, Hebert J, Gogates G, et al. Validation of electronic systems to collect patient-reported outcome (PRO) data - Recommendations for clinical trial teams: report of the ISPOR ePRO systems validation good research practices task force. Value Health 2013;16:480-9
  • Coons SJ, Gwaltney CJ, Hays RD, ISPOR ePRO Task Force. Recommendations on evidence needed to support measurement equivalence between electronic and paper-based patient-reported outcome (PRO) measures: ISPOR ePRO Good Research Practices Task Force report. Value Health 2009;12(4):419-29
  • Dalal AA, Nelson L, Gilligan T, et al. Evaluating patient-reported outcome measurement comparability between paper and alternate versions, using the lung function questionnaire as an example. Value Health 2011;14(5):712-20
  • Brundage M, Blazeby J, Revicki D, et al. Patient-reported outcomes in randomized clinical trials: development of ISOQOL reporting standards. Qual Life Res 2013;22(6):1161-75
  • Calvert M, Blazeby J, Revicki D, et al. Improving health-related quality of life reporting in clinical trials: why do we need a CONSORT extension? Lancet 2011;378(9804):1684-5
  • Calvert M, Blazeby J, Altman DG, et al. Group. Reporting of Patient-Reported Outcomes in Randomized Trials: the CONSORT PRO Extension. JAMA 2013;309(8):814-22

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