Patient-Reported Outcome Data
- 702 Downloads
This chapter provides a brief introduction to patient-reported outcome measures (PROs), with an emphasis on measure characteristics and the implications for informatics of the use of PROs in clinical research. Because of increased appreciation on behalf of health-care funders and regulatory agencies for actual patient experience, PROs have become recognized as legitimate and attractive endpoints for clinical studies and for comparative effectiveness research. “Patient-reported outcomes” is an internationally recognized umbrella term that includes both single dimension and multidimension measures of symptoms, with the defining characteristic that all information is provided directly by the patient. PROs can be administered in a variety of formats and settings, ranging from face-to-face interaction in clinics to web interfaces to mobile devices (e.g., smart phones). PRO instruments measure one or more aspects of patients’ health status and are especially important when more objective measures of disease outcome are not available. PROs can be used to measure a broad array of health status indicators within the context of widely varying study designs exploring a multitude of diseases. As a result, they need to be well characterized so that they can be identified and used appropriately. The standardization, indexing, access, and implementation of PROs are issues that are particularly relevant to clinical research informatics. In this chapter, we discuss design characteristics of PROs, measurement issues relating to the use of PROs, modes of administration, item and scale development, scale repositories, and item banking.
KeywordsPatient-reported outcome data Outcome data by patient report Scales Assessment methods Reliability Validity Electronic data collection devices The patient-reported outcome measurement information system
- 1.FDA. Guidance for industry: patient-reported outcome measures; use in medical product development to support labeling claims. Silver Spring: U. S. D. o. H. a. H. Services; 2009.Google Scholar
- 5.Fayers PM, Machin D. Quality of life: the assessment, analysis and interpretation of patient-reported outcomes. Chichester: Wiley; 2013.Google Scholar
- 8.Shields A, Gwaltney C, Tiplady B, et al. Grasping the FDA’s PRO guidance: what the agency requires to support the selection of patient reported outcome instruments. Appl Clin Trials. 2006;15:69–83.Google Scholar
- 11.Skinner J, Teresi J, et al. Measurement in older ethnically diverse populations: overview of the volume. J Ment Health Aging. 2001;7:5–8.Google Scholar
- 12.Anastasi A. Psychological testing. 6th ed. New York: Macmillan Publishing Company; 1998.Google Scholar
- 15.DeVellis RF. Scale development: theory and applications. 3rd ed. Thousand Oaks: Sage; 2012.Google Scholar
- 16.Vogt W. Dictionary of statistics and methodology: a nontechnical guide for the social sciences. 2nd ed. Thousand Oaks: Sage Publications; 1999.Google Scholar
- 17.Aday L, Cornelius L. Designing and conducting health surveys: a comprehensive guide. 3rd ed. San Francisco: Jossey-Bass; 2006.Google Scholar
- 21.Dillman DA. Internet, mail and mixed-mode surveys: the tailored design method. 4th ed. New York: Wiley; 2014.Google Scholar
- 24.Electronic Patient Reported Outcomes. PAREXEL. https://www.parexel.com/solutions/infor-matics/clinical-outcome-assessments/epro. Accessed 2 Feb 2018.
- 25.Electronic Patient Reported Outcomes (ePRO). ICON plc. http://www.iconplc.com/jp/tech-nology/application-areas/electronic-patient-report/. Accessed 2 Feb 2018.
- 26.Patient Engagement|ePRO. IBM clinical development. https://www.ibmclinicaldevelop-ment.com/en/ibm-clinical-development-epro. Accessed 2 Feb 2018.
- 27.Patient Reported Outcomes. VitalHealth Software. https://www.vitalhealthsoftware.com/prod-ucts/patient-health-questionnaires/patient-reported-outcomes. Accessed 2 Feb 2018.
- 28.e-Patient Reported Outcomes.Acceliant. http://www.acceliant.com/products/e-patient-reported-outcomes. Accessed 2 Feb 2018.
- 29.Rave eCOA/ePRO. Medidata. https://www.mdsol.com/en/products/rave/ecoa-epro. Accessed 2 Feb 2018.
- 34.Muehlhausen W, Doll H, Quadri N, Fordham B, O’Donohoe P, Dogar N, Wild DJ. Equivalence of electronic and paper administration of patient-reported outcome measures: a systematic review and meta-analysis of studies conducted between 2007 and 2013. Health Qual Life Outcomes. 2015;13:167. https://doi.org/10.1186/s12955-015-0362-x.CrossRefPubMedPubMedCentralGoogle Scholar
- 35.Rothman M, Burke L, Erickson P, Leidy NK, Patrick DL, Petrie CD. Use of existing patient-reported outcome (PRO) instruments and their modification: the ISPOR good research practices for evaluating and documenting content validity for the use of existing instruments and their modification PRO task force report. Value Health. 2009;12(8):1075–83.CrossRefGoogle Scholar
- 36.Patrick DL, Burke LB, Gwaltney CJ, Leidy NK, Martin ML, Molsen E, Ring L. 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;14(8):967–77.CrossRefGoogle Scholar
- 37.Patrick DL, Burke LB, Gwaltney CJ, Leidy NK, Martin ML, Molsen E, Ring L. 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(8):978–88.CrossRefGoogle Scholar