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Measurement of Patient-Reported Outcomes of Health Services

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Methods in Health Services Research

Part of the book series: Health Services Research ((HEALTHSR))

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Abstract

A patient-reported outcome (PRO) is any report on the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else. The measurement of PROs should address key protocol elements that include the rationale for the specific aspect of PRO being measured, explicit research objectives and endpoints, strategies for minimizing the exclusion of subjects from the study, rationale for timing of assessments and off-study rules, rationale for instruction selection, details for administration of PRO assessments to minimize bias and missing data, sample size estimation, and analytic plan. Another key element involves the measurement properties of a PRO. These protocol elements are central to this chapter as they relate to the design and measurement of PROs. These elements are discussed and framed within the five characteristics that tend to be associated with PROs: missing and incomplete data, psychometric validation, interpretation, multiple testing, and longitudinal analysis. Special consideration is given for developing a PRO measurement strategy in a regulatory context where the intent is to have a labeling claim on a PRO.

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References

  • Alcion L, Petersen JL, Temple S, Arndt S. Probabilistic index: an intuitive non-parametric approach to measuring the size of treatment effects. Stat Med. 2006;25:591–602.

    Article  Google Scholar 

  • Brooks MM, Jenkins LS, Schron EB, Steinberg JS, Cross JA, Paeth DS. Quality of life at baseline: is assessment after randomization valid? Med Care. 1998;26:1515–9.

    Article  Google Scholar 

  • Brown TA. Confirmatory factor analysis for applied research. New York: The Guilford Press; 2006.

    Google Scholar 

  • Cappelleri JC, Gerber RA. Exploratory factor analysis. In: Chow S-C, editor. Encyclopedia of biopharmaceutical statistics.3rd ed., revised and expanded. New York: Informa Healthcare; 2010. p. 480–5.

    Google Scholar 

  • Cella D, Li JZ, Cappelleri JC, Bushmakin A, Charbonneau C, Kim ST, Chen I, Michaelson MD, Motzer RJ. Quality of life in patients with metastatic renal cell carcinoma treated with sunitinib versus interferon-alfa: Results from a phase III randomized trial. J Clin Oncol. 2008;26:3763–9.

    Article  CAS  PubMed  Google Scholar 

  • Fairclough DL. Patient reported outcomes as endpoints in medical research. Stat Methods Med Res. 2004;13:115–38.

    Article  PubMed  Google Scholar 

  • Fairclough DL. Analysing longitudinal studies of QoL. In: Fayers P, Hayes R, editors. Assessing quality of life in clinical trials. Oxford: Oxford University Press; 2005. p. 149–65.

    Google Scholar 

  • Fairclough DL. Design and analysis of quality of life studies in clinical trials. 2nd ed. Boca Raton: Chapman & Hall/CRC; 2010.

    Google Scholar 

  • Fayers FM, Machin D. Quality of life: the assessment, analysis and interpretation of patient-reported outcomes. 2nd ed. Chichester: Wiley; 2007.

    Book  Google Scholar 

  • Fayers PM, Aaronson NK, Bjordal K, Groenvold M, Curran D, Bottomley A. On behalf of the EORTC quality of life group. In: EORTC QLQ-C30 scoring manual. 3rd ed. Brussels: EORTC; 2001.

    Google Scholar 

  • Fetting JJ, Gray R, Fairclough DL, Smith TJ, Margolin KA, Citron ML, Grove-Conrad M, Cella D, Pandya K, Robert N, Henderson C, Osborne K, Abeloff MD. A 16-week multidrug regimen versus cyclophosphamide, doxorubicin and 5-flurouracil as adjuvant therapy for node-positive, receptor negative breast cancer: an intergroup study. J Clin Oncol. 1998;16:2382–91.

    Article  CAS  PubMed  Google Scholar 

  • Fitzmaurice GH, Laird NM, Ware JH. Applied longitudinal analysis. 2nd ed. Hoboken: Wiley; 2011.

    Google Scholar 

  • Food and Drug Administration. Guidance for industry on patient-reported outcome measures: Use in medical product development to support labeling claims. Fed Regist. 2009;74(235):65132–3.

    Google Scholar 

  • Gotay CC, Korn EL, McCabe MS, Moore TD, Cheson BD. Building quality of life assessment into cancer treatment studies. Oncology. 1992;6:25–8.

    CAS  PubMed  Google Scholar 

  • Johnson JR, Temple R. Food and drug administration requirements for approval of new anticancer drugs. Cancer Treat Rep. 1985;69:1155–9.

    CAS  PubMed  Google Scholar 

  • Luo X, Cappelleri JC. A practical guide on interpreting and evaluating patient-reported outcomes in clinical trials. Clin Res Regul Aff. 2008;25:197–211.

    Article  Google Scholar 

  • Mallinckrodt CH, Lane PW, Schnell D, Peng Y, Mancuso JP. Recommendations for the primary analysis of continuous endpoints in longitudinal clinical trials. Drug Inf J. 2008;42:303–19.

    Google Scholar 

  • National Research Council. The prevention and treatment of missing data in clinical trials. Washington, DC: The National Academies Press; 2010.

    Google Scholar 

  • 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:S125–37.

    Article  PubMed  Google Scholar 

  • Revicki D, Hays RD, Cella D, Sloan J. Recommended methods for determining responsiveness and minimally important differences for patient-reported outcomes. J Clin Epidemiol. 2008;61:102–9.

    Article  PubMed  Google Scholar 

  • Rothman ML, Beltran P, Cappelleri JC, Lipscomb J, Teschendorf B, Mayo/FDA Patient-Reported Outcomes Consensus Meeting Group. Patient-reported outcomes: conceptual issues. Value Health. 2007;10:S66–75.

    Article  PubMed  Google Scholar 

  • Russell IJ, Crofford LJ, Leon T, Cappelleri JC, Bushmakin AG, Whalen E, Barrett JA, Sadosky A. The effects of pregabalin on sleep disturbance symptoms among individuals with fibromyalgia syndrome. Sleep Med. 2009;10:604–10.

    Article  PubMed  Google Scholar 

  • Snyder CF, Watson ME, Jackson JD, Cella D, Halyard MY, Mayo/FDA Patient-Reported Outcomes Consensus Meeting Group. Patient-reported outcomes instruction selection: designing a measurement strategy. Value Health. 2007;10:S76–85.

    Article  PubMed  Google Scholar 

  • Streiner DL, Norman GR. Health measurement scales: a practical guide to their development and use. 4th ed. New York: Oxford University Press; 2008.

    Book  Google Scholar 

  • Wang XS, Fairclough DL, Liao Z, Komaki R, Chang JY, Mobley GM, Cleeland CS. Longitudinal study of the relationship between chemoradiation therapy for non-small-cell lung cancer and patient symptoms. J Clin Oncol. 2006;24:4485–91.

    Article  PubMed  Google Scholar 

  • Wiklund I. Assessment of patient-reported outcomes in clinical trials: the example of health-related quality of life. Fundam Clin Pharmacol. 2004;18:351–63.

    Article  CAS  PubMed  Google Scholar 

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Author information

Authors and Affiliations

  1. Global Product Development, Pfizer Inc, 445 Eastern Point Road (MS 8260-2502), Groton, CT, 06340, USA

    Joseph C. Cappelleri & Andrew G. Bushmakin

Authors
  1. Joseph C. Cappelleri
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  2. Andrew G. Bushmakin
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Corresponding author

Correspondence to Joseph C. Cappelleri .

Editor information

Editors and Affiliations

  1. University of British Columbia Dept. Health Care & Epidemiology, Vancouver, British Columbia, Canada

    Boris Sobolev

  2. Center for Statistical Sciences, Brown University Center for Statistical Sciences, PROVIDENCE, Rhode Island, USA

    Constantine Gatsonis

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Cappelleri, J.C., Bushmakin, A.G. (2017). Measurement of Patient-Reported Outcomes of Health Services. In: Sobolev, B., Gatsonis, C. (eds) Methods in Health Services Research. Health Services Research. Springer, Boston, MA. https://doi.org/10.1007/978-1-4939-6704-9_10-1

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  • DOI: https://doi.org/10.1007/978-1-4939-6704-9_10-1

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  • Publisher Name: Springer, Boston, MA

  • Print ISBN: 978-1-4939-6704-9

  • Online ISBN: 978-1-4939-6704-9

  • eBook Packages: Springer Reference MedicineReference Module Medicine

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