Skip to main content
SpringerLink
Log in

Patient-Reported Outcome Data

  • Chapter
  • First Online:
Clinical Research Informatics

Part of the book series: Health Informatics ((HI))

  • 589 Accesses

Abstract

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 healthcare 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” are 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.

This is a preview of subscription content, log in via an institution to check access.

Access this chapter

Log in via an institution
eBook
USD 16.99
Price excludes VAT (USA)
  • Available as EPUB and PDF
  • Read on any device
  • Instant download
  • Own it forever
Hardcover Book
USD 109.99
Price excludes VAT (USA)
  • Durable hardcover edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info

Tax calculation will be finalised at checkout

Purchases are for personal use only

Institutional subscriptions

References

  1. FDA. Guidance for industry: patient-reported outcome measures; use in medical product development to support labeling claims. Silver Spring: U.S. Department of Health and Human Services; 2009.

    Google Scholar 

  2. McKenna P, Doward L. Integrating patient reported outcomes. Value Health. 2004;7:S9–12.

    Article  PubMed  Google Scholar 

  3. Garratt A. Patient reported outcome measures in trials. BMJ. 2009;338:a2597.

    Article  PubMed  Google Scholar 

  4. 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 

  5. Fayers PM, Machin D. Quality of life: the assessment, analysis and interpretation of patient-reported outcomes. Chichester: Wiley; 2013.

    Google Scholar 

  6. Atkinson MJ, Lennox RD. Extending basic principles of measurement models to the design and validation of patient reported outcomes. Health Qual Life Outcomes. 2006;4(1):65.

    Article  PubMed  PubMed Central  Google Scholar 

  7. Frost MH, Reeve BB, Liepa AM, Stauffer JW, Hays RD. Mayo/FDA patient-reported outcomes consensus meeting group. What is sufficient evidence for the reliability and validity of patient-reported outcome measures? Value Health. 2007a;10:S94–S105.

    Article  PubMed  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 

  9. Valderas J, Alonso J. Patient reported outcome measures: a model-based classification system for research and clinical practice. Qual Life Res. 2008;17:1125–35.

    Article  PubMed  Google Scholar 

  10. Calvert M, Blazeby J, Altman DG, Revicki DA, Moher D, Brundage MD, CONSORT PRO Group. Reporting of patient-reported outcomes in randomized trials: the CONSORT PRO extension. JAMA. 2013;309(8):814–22.

    Article  CAS  PubMed  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 

  13. Morgan R, Teal C, et al. Measurement in VA health services research: veterans as a special population. Health Serv Res. 2005;40:1573–83.

    Article  PubMed  PubMed Central  Google Scholar 

  14. Frost MH, Reeve BB, Liepa AM, Stauffer JW, Hays RD, the Mayo/FDA Patient-Reported Outcomes Consensus Meeting Group. What is sufficient evidence for the reliability and validity of patient-reported outcome measures? Value Health. 2007b;10(S2):S94–S105.

    Article  PubMed  Google Scholar 

  15. DeVellis RF. Scale development: theory and applications. 3rd ed. Thousand Oaks, CA: Sage; 2012.

    Google Scholar 

  16. Vogt W. Dictionary of statistics and methodology: a nontechnical guide for the social sciences. 2nd ed. Thousand Oaks, CA: Sage Publications; 1999.

    Google Scholar 

  17. Deng L, Chan W. Testing the difference between reliability coefficients alpha and omega. Educ Psychol Meas. 2017;77(2):185–203. https://doi.org/10.1177/0013164416658325.

    Article  PubMed  Google Scholar 

  18. Aday L, Cornelius L. Designing and conducting health surveys: a comprehensive guide. 3rd ed. San Francisco, CA: Jossey-Bass; 2006.

    Google Scholar 

  19. McDonald RP. Test theory: a unified treatment. New York: Psychology Press; 2013.

    Book  Google Scholar 

  20. McDowell I. Measuring health: a guide to rating scales and questionnaires. 3rd ed. New York: Oxford University Press; 2006.

    Book  Google Scholar 

  21. 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(2):102–9.

    Article  PubMed  Google Scholar 

  22. Bennett AV, Dueck AC, Mitchell SA, Mendoza TR, Reeve BB, Atkinson TM, et al. Mode equivalence and acceptability of tablet computer-, interactive voice response system-, and paper-based administration of the US National Cancer Institute’s Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE). Health Qual Life Outcomes. 2016;14(1):1–12.

    Article  Google Scholar 

  23. Miele G, Straccia G, Moccia M, Leocani L, Tedeschi G, Bonavita S, Lavorgna L. Digital Technologies, Web and social media study Group of the Italian Society of neurology. Telemedicine in Parkinson’s disease: how to ensure patient needs and continuity of care at the time of COVID-19 pandemic. Telemed J E Health. 2020;26(12):1533–6.

    Article  CAS  PubMed  Google Scholar 

  24. Aiyegbusi OL, Calvert MJ. Patient-reported outcomes: central to the management of COVID-19. Lancet. 2020;396(10250):531.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  25. Bowling A. Mode of questionnaire administration can have serious effects on data quality. J Public Health. 2005;27:281–91.

    Article  Google Scholar 

  26. Dillman DA. Internet, mail and mixed-mode surveys: the tailored design method. 4th ed. New York: Wiley; 2014.

    Google Scholar 

  27. Snyder CF, Blackford AL, Wolff AC, Carducci MA, Herman JM, Wu AW, the PatientViewpoint Scientific Advisory Board. Feasibility and value of PatientViewpoint: a web system for patient-reported outcomes assessment in clinical practice. Psycho-Oncology. 2013;22(4):895–901.

    Article  PubMed  Google Scholar 

  28. Juanjuan L, Santa-Maria CA, Hongfang F, Lingcheng W, Pengcheng Z, Yuanbing X, et al. Patient-reported outcomes of patients with breast cancer during the COVID-19 outbreak in the epicenter of China: a cross-sectional survey study. Clin Breast Cancer. 2020;20(5):e651–62.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  29. Gwaltney CJ, Shields AL, Shiffman S. Equivalence of electronic and paper-and-pencil administration of patient-reported outcome measures: a meta-analytic review. Value Health. 2008;11(2):322–33.

    Article  PubMed  Google Scholar 

  30. Gold HT, Karia RJ, Link A, Lebwohl R, Zuckerman JD, Errico TJ, et al. Implementation and early adaptation of patient-reported outcome measures into an electronic health record: a technical report. Health Informatics J. 2020;26(1):129–40.

    Article  PubMed  Google Scholar 

  31. Coons S, Gwaltney C, et al. 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;12:419–29.

    Article  PubMed  Google Scholar 

  32. Coons SJ, Eremenco S, Lundy JJ, O’Donohoe P, O’Gorman H, Malizia W. Capturing patient-reported outcome (PRO) data electronically: the past, present, and promise of ePRO measurement in clinical trials. Patient. 2015;8(4):301–9.

    Article  PubMed  Google Scholar 

  33. Castor. Electronic Data Capture (EDC) System for Clinical Research Trials. Castor. [Online]. https://www.castoredc.com/electronic-data-capture-system/. Accessed 14 Apr 2022.

  34. AssisTek. AssisTek. [Online]. https://assistek.com/. Accessed 14 Apr 2022.

  35. Medidata. Medidata Patient Cloud: Patient Centric Clinical Trials. Medidata. [Online]. https://www.medidata.com/en/clinical-trial-products/patient-centric-clinical-trials. Accessed 14 Apr 2022.

  36. Peeters M, Van Dam P, Rasschaert MA, Vulsteke C, De Keersmaecker S, Croes L, et al. Prescreening for COVID-19 in patients receiving cancer treatment using a patient-reported outcome platform. ESMO Open. 2020;5(3):e000817.

    Article  PubMed  PubMed Central  Google Scholar 

  37. Elkan M, Dvir A, Zaidenstein R, Keller M, Kagansky D, Hochman C, Koren R. Patient-reported outcome measures after hospitalization during the COVID-19 pandemic: a survey among COVID-19 and non-COVID-19 patients. Int J Gen Med. 2021;14:4829–36.

    Article  PubMed  PubMed Central  Google Scholar 

  38. Hamilton SN, Chau N, Berthelet E, Wu J, Tran E, Chevrier M, et al. Patient-reported outcomes and complications during head and neck cancer radiotherapy before versus during the COVID-19 pandemic. Support Care Cancer. 2022;30(3):2745–53.

    Article  PubMed  Google Scholar 

  39. Collins R, Kashdan T, et al. The feasibility of using cellular phones to collect ecological momentary assessment data: application to alcohol consumption. Exp Clin Psychopharmacol. 2003;11:73–8.

    Article  PubMed  Google Scholar 

  40. Freedman M, Lester K, et al. Cell phones for ecological momentary assessment with cocaine-addicted homeless patients in treatment. J Subst Abus Treat. 2006;30:105–11.

    Article  Google Scholar 

  41. Reid S, Kauer S, et al. A mobile phone program to track young people’s experiences of mood, stress and coping. Soc Psychiatry Psychiatr Epidemiol. 2009;44(6):501–7.

    Article  PubMed  Google Scholar 

  42. 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.

    Article  PubMed  PubMed Central  Google Scholar 

  43. 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.

    Article  PubMed  Google Scholar 

  44. 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.

    Article  PubMed  Google Scholar 

  45. 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.

    Article  PubMed  Google Scholar 

  46. Reeve BB, Hays RD, Bjorner JB, Cook KF, Crane PK, Teresi JA, et al. Psychometric evaluation and calibration of health-related quality of life item banks: plans for the patient-reported outcomes measurement information system (PROMIS). Med Care. 2007;45(5):S22–31.

    Article  PubMed  Google Scholar 

  47. Cella D, Yount S, Rothrock N, Gershon R, Cook K, Reeve B, et al. The patient-reported outcomes measurement information system (PROMIS): progress of an NIH roadmap cooperative group during its first two years. Med Care. 2007;45(5 Suppl 1):S3.

    Article  PubMed  PubMed Central  Google Scholar 

  48. Cella D, Riley W, Stone A, Rothrock N, Reeve B, Yount S, et al. The patient-reported outcomes measurement information system (PROMIS) developed and tested its first wave of adult self-reported health outcome item banks: 2005–2008. J Clin Epidemiol. 2010;63(11):1179–94.

    Article  PubMed  PubMed Central  Google Scholar 

  49. Harniss M, Amtmann D, et al. Considerations for developing interfaces for collecting patient-reported outcomes that allow the inclusion of individuals with disabilities. Med Care. 2007;45:S48–54.

    Article  PubMed  PubMed Central  Google Scholar 

  50. https://www.fda.gov/about-fda/oncology-center-excellence/project-patient-voice

  51. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/core-patient-reported-outcomes-cancer-clinical-trials

  52. Patient-Focused Drug Development: Methods to Identify What Is Important to Patients Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders. https://www.fda.gov/media/131230/download

  53. Pugh SL, Rodgers JP, Yeager KA, Chen RC, Movsas B, Bonanni R, et al. Characteristics of participation in patient-reported outcomes and electronic data capture components of NRG oncology clinical trials. Int J Radiat Oncol Biol Phys. 2020;108(4):950–9.

    Article  PubMed  PubMed Central  Google Scholar 

  54. Kunonga TP, Spiers GF, Beyer FR, Hanratty B, Boulton E, Hall A, Bower P, Todd C, Craig D. Effects of digital technologies on older people’s access to health and social care: umbrella review. J Med Internet Res. 2021;23(11):e25887.

    Article  PubMed  PubMed Central  Google Scholar 

  55. Basford JR, Cheville AL. Advancing patient-centered rehabilitation with electronic and computerized patient reported outcomes measures. Phys Med Rehabil. 2022;103(5):S1–S168.

    Article  Google Scholar 

  56. Austin EJ, LeRouge C, Lee JR, Segal C, Sangameswaran S, Heim J, Lober WB, Hartzler AL, Lavallee DC. A learning health systems approach to integrating electronic patient-reported outcomes across the health care organization. Learn Health Syst. 2021;5(4):e10263. https://doi.org/10.1002/lrh2.10263. PMID: 34667879; PMCID: PMC8512814

    Article  PubMed  PubMed Central  Google Scholar 

  57. Bakken S, Cimino JJ, Haskell R, Kukafka R, Matsumoto C, Chan GK, Huff SM. Evaluation of the clinical LOINC (logical observation identifiers, names, and codes) semantic structure as a terminology model for standardized assessment measures. J Am Med Inform Assoc. 2000;7(6):529–38. https://doi.org/10.1136/jamia.2000.0070529. PMID: 11062226; PMCID: PMC129661

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  58. Office of the National Coordinator for Health IT. Interoperability Standards Advisory - Collection and Exchange of Patient Reported Outcomes. 2021. https://www.healthit.gov/isa/collection-and-exchange-patient-reported-outcomes. Accessed 31 Aug 2022.

Download references

Author information

Authors and Affiliations

  1. Department of Management, Policy and Community Health, University of Texas Health Science Center at Houston School of Public Health, Houston, TX, USA

    Robert O. Morgan & Laura E. Witte

  2. Health Economics and Outcomes Research, AbbVie Pharmaceuticals, North Chicago, IL, USA

    Kavita R. Sail

Authors
  1. Robert O. Morgan
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Kavita R. Sail
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. Laura E. Witte
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to Robert O. Morgan .

Editor information

Editors and Affiliations

  1. Learning Health Sciences, University of Michigan School of Medicin, Ann Arbor, MI, USA

    Rachel L. Richesson

  2. School of Information, University of South Florida, Tampa, FL, USA

    James E. Andrews

  3. Medical Informatics and Clinical Epidemiology, Oregon Health and Science University, Portland, OR, USA

    Kate Fultz Hollis

Rights and permissions

Reprints and permissions

Copyright information

© 2023 The Author(s), under exclusive license to Springer Nature Switzerland AG

About this chapter

Check for updates. Verify currency and authenticity via CrossMark

Cite this chapter

Morgan, R.O., Sail, K.R., Witte, L.E. (2023). Patient-Reported Outcome Data. In: Richesson, R.L., Andrews, J.E., Fultz Hollis, K. (eds) Clinical Research Informatics. Health Informatics. Springer, Cham. https://doi.org/10.1007/978-3-031-27173-1_15

Download citation

  • DOI: https://doi.org/10.1007/978-3-031-27173-1_15

  • Published:

  • Publisher Name: Springer, Cham

  • Print ISBN: 978-3-031-27172-4

  • Online ISBN: 978-3-031-27173-1

  • eBook Packages: MedicineMedicine (R0)

Publish with us

Policies and ethics

Search

Navigation