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Developing Patient-Reported and Relevant Outcome Measures

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Patient Involvement in Health Technology Assessment

Abstract

This chapter will examine good practice guidance for patient-centred approaches towards PROM development. During the last decade, we have witnessed a paradigm shift in how outcomes are measured from a more clinical, physician-oriented perspective to a more patient-focused perspective, which has led to the emergence of the notion of patient-reported outcome (PRO). The concept of PRO seeks to understand how patients feel, function and live their lives in relation to health challenges and associated healthcare and is more encompassing than earlier terms, such as patient global assessment, health status, quality of life or symptom checklists. In this chapter, we argue that well-developed questionnaires, or PRO measures (PROMs), which reflect patients’ perspectives, have the potential to provide valuable patient-based evidence in HTA. PROM development should engage with patients as participants (US Food and Drug 2009) and increasingly as research partners (Staniszewska et al. 2012; de Wit et al. 2013; Chap. 8) through all stages of development. This promotes patients as the determinants of the key constructs underpinning the PROM. This approach will support a transparent and auditable approach towards capturing patients’ contributions to the measurement of relevant outcomes, thereby enhancing the face and content validity, relevance and acceptability of measures. In this chapter, we describe eight key stages in PROM development and reflect on how patients can participate in this process.

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Notes

  1. 1.

    Consensus-based standards for the selection of health measurement instruments

References

  • Andrich D. Rating scales and Rasch measurement. Expert Rev Pharmacoecon Outcomes Res. 2011;11:571–85.

    Article  PubMed  Google Scholar 

  • Bartlett SJ, Hewlett S, Bingham III CO, Woodworth TG, Alten R, Pohl C, OMERACT RA Flare Working Group, et al. Identifying core domains to assess flare in rheumatoid arthritis: an OMERACT international patient and provider combined Delphi consensus. Ann Rheum Dis. 2012;71:1855–60.

    Article  PubMed  Google Scholar 

  • BrĂ©dart A, Marrel A, Abetz-Webb L, Lasch K, Acquadro C. Interviewing to develop patient-reported outcome (PRO) measures for clinical research: eliciting patients’ experience. Health Qual Life Outcomes. 2014;5:12–5.

    Google Scholar 

  • Bjorner JB, Kosinski M, Ware Jr JE. Calibration of an item pool for assessing the burden of headaches: an application of item response theory to the headache impact test (HIT). Qual Life Res. 2003;12:913–33.

    Article  PubMed  Google Scholar 

  • Blazeby J, Sprangers MA, Cull A, Groenvold M, Bottomley A. EORTC quality of life group: guidelines for developing questionnaire modules. 3rd ed. Brussels: EORTC Quality of Life Group Publication ; 2002.2-930064-24-2

    Google Scholar 

  • Crosby RD, Kolotkin RL, Williams GR. Defining clinically meaningful change in health-related quality of life. J Clin Epidemiol. 2003;56:395–407.

    Article  PubMed  Google Scholar 

  • Christodoulou C, Junghaenel DU, DeWalt DA, Rothrock N, Stone AA. Cognitive interviewing in the evaluation of fatigue items: results from the patient-reported outcomes measurement information system (PROMIS). Qual Life Res. 2008;17:1239–46.

    Article  PubMed  PubMed Central  Google Scholar 

  • de Vet HC, Terwee CB, Ostelo RW, Beckerman H, Knol DL, Bouter LM. Minimal changes in health status questionnaires: distinction between minimally detectable change and minimally important change. Health Qual Life Outcomes. 2006;22:54.

    Article  Google Scholar 

  • de Wit M, Abma T, Koelewijn-van Loon M, Collins S, Kirwan J. Involving patient research partners has a significant impact on outcomes research: a responsive evaluation of the international OMERACT conferences. BMJ Open. 2013;3(5):e002241.

    Article  PubMed  PubMed Central  Google Scholar 

  • Golics CJ, Basra MK, Finlay AY, Salek S. The development and validation of the Family Reported Outcome Measure (FROM-16)© to assess the impact of disease on the partner or family member. Qual Life Res. 2014;23:317–26.

    Article  PubMed  Google Scholar 

  • Gorecki C, Lamping DL, Brown JM, Madill A, Firth J, Nixon J. Development of a conceptual framework of health-related quality of life in pressure ulcers: a patient-focused approach. Int J Nurs Stud. 2010;47:1525–34.

    Article  PubMed  Google Scholar 

  • Gorecki C, Lamping DL, Nixon J, Brown JM, Cano S. Applying mixed methods to pretest the Pressure Ulcer Quality of Life (PU-QOL) instrument. Qual Life Res. 2012;21:441–51.

    Article  CAS  PubMed  Google Scholar 

  • Gorecki C, Brown JM, Cano S, Lamping DL, Briggs M, Coleman S, et al. Development and validation of a new patient-reported outcome measure for patients with pressure ulcers: the PU-QOL instrument. Health Qual Life Outcomes. 2013;11:95.

    Article  PubMed  PubMed Central  Google Scholar 

  • Gossec L, de Wit M, Kiltz U, Braun J, Kalyoncu U, Scrivo R, EULAR PsAID Taskforce, et al. A patient-derived and patient-reported outcome measure for assessing psoriatic arthritis: elaboration and preliminary validation of the Psoriatic Arthritis Impact of Disease (PsAID) questionnaire, a 13-country EULAR initiative. Ann Rheum Dis. 2014;73:1012–9.

    Article  PubMed  Google Scholar 

  • Hay JL, Atkinson TM, Reeve BB, Mitchell SA, Mendoza TR, Willis G, NCI PRO-CTCAE Study Group, et al. Cognitive interviewing of the US National Cancer Institute’s patient-reported outcomes version of the common terminology criteria for adverse events (PRO-CTCAE). Qual Life Res. 2014;23:257–69.

    Article  PubMed  Google Scholar 

  • Haywood KL, Garratt AM, Jordan K, Healey EL, Packham JC. Evaluation of ankylosing spondylitis quality of life (EASi-QoL): reliability and validity of a new patient-reported outcome measure. J Rheumatol. 2010;37:2100–9.

    Article  PubMed  Google Scholar 

  • Haywood KL, Staniszewska S, Chapman S. Quality and acceptability of patient reported outcome measures in chronic fatigue syndrome/Myalgic encephalitis (CFS/ME): a structured review. Qual Life Res. 2012;21:35–52.

    Article  PubMed  Google Scholar 

  • Haywood KL, Collins S, Crawley E. Assessing severity of illness and outcomes of treatment in children with Chronic Fatigue Syndrome/Myalgic Encephalitis (CFS/ME): a systematic review of patient-reported outcome measures. Child Care Health Dev. 2014a;40:806–24.

    Article  CAS  PubMed  Google Scholar 

  • Haywood KL, Whitehead L, Perkins GD. The psychosocial outcomes of cardiac arrest: relevant and robust patient-centred assessment is essential. Resuscitation. 2014b;85:718–9. doi:10.1016/j.resuscitation.2014.03.305.

    Article  PubMed  Google Scholar 

  • Haywood KL, Wilson R, Staniszewska S, Salek S. Using PROMs in healthcare: who should be in the driving seat–policy makers, health professionals, methodologists or patients? Patient. 2016;9(6):495–8.

    Article  PubMed  Google Scholar 

  • Health Measures. Applications in reserach. 2016. http://www.nihpromis.org/researchers/researchershome. Accessed 21 Dec 2016.

  • Hobart J, Cano S. Improving the evaluation of therapeutic interventions in multiple sclerosis: the role of new psychometric methods. Health Technol Assess. 2009;13:1–177.

    Article  Google Scholar 

  • Hongbo Y, Thomas CL, Harrison MA, Salek MS, Finlay AY. Translating the science of quality of life into practice: what do dermatology life quality index scores mean? J Invest Dermatol. 2005;125:659–6.

    Article  CAS  PubMed  Google Scholar 

  • Jaeschke R, Singer J, Guyatt G. Measurement of health status. Ascertaining the minimal clinically important difference. Control Clin Trials. 1989;10:407–15.

    Article  CAS  PubMed  Google Scholar 

  • Kamudoni P, Mueller B, Salek MS. The development and validation of a disease-specific quality of life measure in hyperhidrosis: the hyperhidrosis quality of life index (HidroQOL©). Qual Life Res. 2015;24:1017–27.

    Article  CAS  PubMed  Google Scholar 

  • Kosinski M, Bayliss MS, Bjorner JB, Ware Jr JE, Garber WH, Batenhorst A, et al. A six-item short-form survey for measuring headache impact: the HIT-6. Qual Life Res. 2003;12:963–74.

    Article  CAS  PubMed  Google Scholar 

  • Leidy N, Vernon M. Perspectives on patient-reported outcomes. Content validity and qualitative research in a changing clinical trial environment. PharmacoEconomics. 2008;26:363–70.

    Article  PubMed  Google Scholar 

  • Longworth L, Rowen D. Mapping to obtain EQ-5D utility values for use in NICE health technology assessments. Value Health. 2013;16:202–10.

    Article  PubMed  Google Scholar 

  • Mokkink LB, Terwee CB, Patrick DL, Alonso J, Stratford PW, Knol DL, et al. The COSMIN study reached international consensus on taxonomy, terminology, and definitions of measurement properties for health-related patient-reported outcomes. J Clin Epidemiol. 2010;63:737–45.

    Article  PubMed  Google Scholar 

  • Parslow R, Patel A, Beasant L, Haywood KL, Johnson D, Crawley E. What matters to children with CFS/ME? A conceptual model as the first stage in developing a PROM. Arch Dis Child. 2015;100:1141–7. doi:10.1136/archdischild-2015-308831. Epub 2015 Oct 9

    Article  PubMed  PubMed Central  Google Scholar 

  • Parslow R, Harris S, Broughton J, Alattas A, Crawley E, Haywood K, et al. Children’s experiences of Chronic Fatigue Syndrome/Myalgic Encephalomyelitis (CFS/ME): A systematic review and meta-ethnography of qualitative studies. BMJ Open. 2016;7(1):e012633.

    Article  Google Scholar 

  • Parslow RM. (2016). Developing a Patient Reported Outcome Measure (PROM) for Children with Chronic Fatigue Syndrome/Myalgic Encephalomyelitis (CFS/ME). Thesis submitted to the University of Bristol.

    Google Scholar 

  • Patrick DL, Burke LB, Gwaltney CJ, Leidy NK, Martin ML, Molsen E, 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. 2011a;14:967–77.

    Article  PubMed  Google Scholar 

  • Patrick DL, Burke LB, Gwaltney CJ, Leidy NK, Martin ML, Molsen E, 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. 2011b;14:978–88.

    Article  PubMed  Google Scholar 

  • Petkovic J, Epstein J, Buchbinder R, Welch V, Rader T, Lyddiatt A, et al. Toward ensuring health equity: readability and cultural equivalence of OMERACT patient-reported outcome measures. J Rheumatol. 2015;42:2448–59.

    Article  PubMed  PubMed Central  Google Scholar 

  • Reeve BB, Hays RD, Bjorner JB, Cook KF, Crane PK, Teresi JA, PROMIS Cooperative Group, 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 Suppl 1):S22–31.

    Article  PubMed  Google Scholar 

  • 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;12:1075–83.

    Article  PubMed  Google Scholar 

  • Salaffi F, Carotti M, Gasparini S, Intorcia M, Grassi W. The health-related quality of life in rheumatoid arthritis, ankylosing spondylitis, and psoriatic arthritis: a comparison with a selected sample of healthy people. Health Qual Life Outcomes. 2009;7:25.

    Article  PubMed  PubMed Central  Google Scholar 

  • Salek S, Kamudoni P, Oliva E, Ionova T. Quality of life issues important to patients with haematological malignancies. Value Health. 2016;18:A709.

    Article  Google Scholar 

  • Staniszewska S, Haywood KL, Brett J, Tutton L. Patient and public involvement in patient-reported outcome measures: evolution not revolution. Patient. 2012;5:79–87.

    Article  PubMed  Google Scholar 

  • Streiner DL, Norman GR, Cairney J. Health measurement scales: a practical guide to their development and use. 5th ed. UK: Oxford University Press; 2014.

    Google Scholar 

  • Terwee CB, Bot SD, de Boer MR, van der Windt DA, Knol DL, Dekker J, et al. Quality criteria were proposed for measurement properties of health status questionnaires. J Clin Epidemiol. 2007;60:34–42.

    Article  PubMed  Google Scholar 

  • Terwee CB, Mokkink LB, Knol DL, Ostelo RW, Bouter LM, de Vet HC. Rating the methodological quality in systematic reviews of studies on measurement properties: a scoring system for the COSMIN checklist. Qual Life Res. 2012;21:651–7.

    Article  PubMed  Google Scholar 

  • Tourangeau R. Cognitive science and survey methods. In: Jabine T, Straf M, Tanur J, & Tourangeau R (Eds.). Cognitive aspects of survey methodology: Building a bridge between disciplines. Washington, DC: National Academy Press. 1984. pp. 73–100.

    Google Scholar 

  • US Food and Drug. Administration guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims. Rockville: Department of Health and Human Services, Food and Drug Administration, Centre for Drug Evaluation and Research; 2009. http://www.fda.gov/downloads/Drugs/Guidances/UCM193282.pdf. Accessed 30 Jan 2016

    Google Scholar 

  • Victorson DE, Cella D, Grund H, Judson MA. A conceptual model of health-related quality of life in sarcoidosis. Qual Life Res. 2014;23:89–101.

    Article  PubMed  Google Scholar 

  • Zraick RI, Atcherson SR. Readability of patient-reported outcome questionnaires for use with persons with dysphonia. J Voice. 2012;26:635–41.

    Article  PubMed  Google Scholar 

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Acknowledgements

The authors would like to acknowledge comments received from Dr. Stefan Cano whilst revising the original transcript. All views expressed and any errors are entirely the responsibility of the authors.

All authors declare no conflict of interest.

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Authors and Affiliations

  1. Royal College of Nursing Research Institute, Department of Health Sciences, Warwick Medical School, The University of Warwick, Gibbet Hill, Coventry, CV4 7AL, UK

    Kirstie L Haywood & Sophie Staniszewska

  2. Department of Medical Humanities, VU University Medical Centre, Amsterdam, The Netherlands

    Maarten de Wit

  3. UCB Biopharma, Brussels, Belgium

    Thomas Morel

  4. School of Life and Medical Sciences, School of Pharmacy, Pharmacology and Postgraduate Medicine, University of Hertfordshire, College Lane, Hatfield, AL10 9AB, UK

    Sam Salek

Authors
  1. Kirstie L Haywood
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  2. Maarten de Wit
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  3. Sophie Staniszewska
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  4. Thomas Morel
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  5. Sam Salek
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Corresponding author

Correspondence to Kirstie L Haywood .

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Editors and Affiliations

  1. No. 9 Bioquarter, Usher Institute of Population Health Sciences and Informatics, University of Edinburgh, Edinburgh, United Kingdom

    Karen M. Facey

  2. Department of Public Health, Research Unit of General Practice, University of Southern Denmark, Institute of Public Health, Odense, Denmark

    Helle Ploug Hansen

  3. Patient and Citizen Involvement Interest, HTAi, Ashgrove, Queensland, Australia

    Ann N.V. Single

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Haywood, K.L., de Wit, M., Staniszewska, S., Morel, T., Salek, S. (2017). Developing Patient-Reported and Relevant Outcome Measures. In: Facey, K., Ploug Hansen, H., Single, A. (eds) Patient Involvement in Health Technology Assessment. Adis, Singapore. https://doi.org/10.1007/978-981-10-4068-9_9

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  • DOI: https://doi.org/10.1007/978-981-10-4068-9_9

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