Skip to main content
SpringerLink
Log in

Item Development and Face Validity of the Rheumatoid Arthritis Patient Priorities in Pharmacological Interventions Outcome Measures

  • Original Research Article
  • Published:
The Patient - Patient-Centered Outcomes Research Aims and scope Submit manuscript

Abstract

Background

The assessment of rheumatoid arthritis (RA) is dominated by core sets and indices that have been developed by RA professionals. Previous research developed a set of eight priority treatment outcomes generated by patients to complement the professionally developed core sets for RA.

Objective

This study aimed to facilitate quantitative measurement of these outcomes.

Methods

Two consultation meetings with patient research partners diagnosed with RA (n = 18) were held to identify face validity in existing instruments (Phase 1) at the Bristol Royal Infirmary. Where validated measures did not exist, new numerical rating scales (NRS) were constructed and discussed at two focus groups with patients diagnosed with RA (n = 8) at the Bristol Royal Infirmary and the Royal National Hospital for Rheumatic Diseases (Phase 2). Feedback on the stem question, time frame, anchors and layout was recorded and transcribed verbatim.

Results

Of the eight priorities, existing NRS for pain, activities of daily living and fatigue were voted as acceptable (Phase 1), but new NRS were required for five priorities. The partners strongly recommended that the three separate domains of severity, effect and ability to cope in each measurement area be assessed, as in the existing validated fatigue NRS. Focus group participants (Phase 2) made significant contributions to the phrasing of questions, for example how to ensure ‘mobility’ could be uniformly understood and how changes in valued activities be judged appropriately.

Conclusion

Through extensive patient feedback, 24 NRS were constructed based on priorities identified by patients and encompassing domains where existing questionnaires contain many more items and do not address three important concepts endorsed by patients: severity, effect and coping. The Rheumatoid Arthritis Patient Priorities in Pharmacological Interventions patient-reported outcome measures are now ready for the evaluation of comprehension, construct validity and sensitivity through an observational study.

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

Access this article

Log in via an institution

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Fig. 1

Similar content being viewed by others

References

  1. Boers M, Tugwell P, Felson DT, van Reil PLCM, Kirwan JR, et al. World Health Organisation (WHO) and International League Against Rheumatism (ILAR) core endpoints for symptom-modifying antirheumatic drugs in rheumatoid arthritis clinical trials. J Rheumatol. 1994;21(41):86–9.

    Google Scholar 

  2. Felson D, Anderson J, Boers M. The American College of Rheumatology preliminary cores set of disease activity measures for rheumatoid arthritis clinical trials. Arthritis Rheum. 1993;36(6):729–40.

    Article  CAS  PubMed  Google Scholar 

  3. Van der Heijde D, Hof M, Piet L, Putte L. Development of a disease activity score based on judgement in clinical practice by rheumatologists. J Rheumatol. 1993;20(3):579–81.

    PubMed  Google Scholar 

  4. French T, Hewlett S, Kirwan J, Sanderson T. Different wording of the Patient Global Visual Analogue Scale (PG-VAS) affects rheumatoid arthritis patients’ scoring and the overall Disease Activity Score (DAS28): a cross-sectional study. Musculoskeletal Care. 2013;11(4):229–37.

    Article  PubMed  Google Scholar 

  5. Fries JF, Spitz PW, Kraines RG, Holman HR. Measurement of patient outcome in arthritis. Arthritis Rheum. 1980;23:137–45.

    Article  CAS  PubMed  Google Scholar 

  6. NICE. Adalimumab, etanercept and infliximab for the treatment of rheumatoid arthritis after failure of a TNF-alpha inhibitor. Appraisal consultation document. London: NICE; 2008.

  7. Stucki G, Cieza A, Geyh S, Battistella L, Lloyd J, Symmons D, Kostanjsek N, Schouten J. ICF core sets for rheumatoid arthritis. J Rehabil Med. 2004;Suppl(44):87-93.

  8. Coenen M, Stamm T, Cieza A, Amann E, Kollerits B, Stucki G. Validation of the comprehensive ICF core set for rheumatoid arthritis from the patients’ perspective using focus groups. Arthritis Res Ther. 2006;8:R84.

    Article  PubMed  PubMed Central  Google Scholar 

  9. Kirwan J, Minnock P, Adebajo A, Bresnihan B, Choy E, De Witt M, et al. Patient perspective workshop: fatigue as a recommended patient-centred outcome measure in rheumatoid arthritis. J Rheumatol. 2007;34:1174–82.

    PubMed  Google Scholar 

  10. US Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research, Center for Biologics Evaluation and Research, Center for Devices and Radiological Health. Guidance for industry: patient-reported outcome measures. Use in medical product development to support labeling claims; 2009. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM193282.pdf. Accessed 4 Jan 2014.

  11. Linde L, Sorensen J, Ostergaard M, Horslev-Pedersen K, Lund Hetland M. Health-related quality of life: validity, reliability, and responsiveness of SF-36, 15D, EQ-5D, RAQoL, and HAQ in patients with rheumatoid arthritis. J Rheumatol. 2008;35:1528–37.

    PubMed  Google Scholar 

  12. Patrick DL, Burke LB, Powers JH, Scott JA, Rock EP, Dawisha S, et al. Patient-reported outcomes to support medical product labeling claims: FDA perspective. Value Health. 2007;10(Suppl 2):S125–37.

    Article  PubMed  Google Scholar 

  13. Frost MF, Reeve BB, Liepa AM, Stauffer JW, Hays RD. What is sufficient evidence for the reliability and validity of patient-reported outcome measures? Value Health. 2007;2:S94–105.

    Article  Google Scholar 

  14. Hewlett S. Patients and clinicians have different perspectives on outcomes in arthritis. J Rheumatol. 2003;30(4):877–9.

    PubMed  Google Scholar 

  15. Hewlett S, Carr M, Ryan S, Kirwan J, Richards P, Carr A, et al. Outcomes generated by patients with rheumatoid arthritis: how important are they? Musculoskeletal Care. 2005;3(3):131–42.

    Article  PubMed  Google Scholar 

  16. Gossec L, Patternotte S, Aanerud GJ, Balanescu A, Boumpas DT, Carmona L, et al. Finalisation and validation of the rheumatoid arthritis impact of disease score, a patient-derived composite measure of impact of rheumatoid arthritis: a EULAR initiative. Ann Rheum Dis. 2011;70:935–42.

    Article  CAS  PubMed  Google Scholar 

  17. Sanderson T, Morris M, Calnan M, Richards P, Hewlett S. What outcomes from pharmacological treatments are important to people with rheumatoid arthritis? Creating the basis of a patient core set. Arthritis Care Res. 2010;62(5):640–6.

    Article  CAS  Google Scholar 

  18. Marshall NJ, Wilson G, Lapworth K, Kay LJ. Patients’ perceptions of treatment with anti-TNF therapy for rheumatoid arthritis: a qualitative study. Rheumatology. 2004;43:1034–8.

    Article  CAS  PubMed  Google Scholar 

  19. Ahlmen M, Nordenskiold U, Archenholtz B, Thyberg I, Ronnqvist R, Linden L, et al. Rheumatology outcomes: the patient’s perspective. A multicentre focus group interview study of Swedish rheumatoid arthritis patients. Rheumatology. 2005;44(1):105–10.

    Article  CAS  PubMed  Google Scholar 

  20. Sanderson T, Morris M, Calnan M, Richards P, Hewlett S. Patient perspective of measuring treatment efficacy: the Rheumatoid Arthritis Patient Priorities for Pharmacological Interventions (RAPP-PI) outcomes. Arthritis Care Res. 2010;62(5):647–56.

    Article  CAS  Google Scholar 

  21. Boers M, Kirwan JR, Wells G, Beaton D, Gossec L, D’agostino M-A, et al. Developing core outcome measurement sets for clinical trials: OMERACT Filter 2.0. J Clin Epidemiol. 2014;67:745–53.

    Article  PubMed  Google Scholar 

  22. Sanderson T. Well-being and the prioritisation of treatment outcomes in rheumatoid arthritis. Doctoral thesis; 2009.

  23. Sanderson T, Kirwan J, Almeida C, Morris M, Watts A, Noddings R, Hewlett S. Feasibility, and preliminary validity and sensitivity of the Rheumatoid Arthritis Patient Priorities in Pharmacological Interventions (RAPP-PI) outcome measures (awaiting submission).

  24. Arnett FC, Edworthy SM, Bloch DA, McShane DJF, Cooper NS, Healey LA, et al. The American Rheumatism Association 1987 revised criteria for the classification of rheumatoid arthritis. Arthritis Rheum. 1988;31:315–24.

    Article  CAS  PubMed  Google Scholar 

  25. Herr KA, Spratt K, Mobily PR, Richardson G. Pain intensity assessment in older adults: use of experimental pain to compare psychometric properties and usability of selected pain scales with younger adults. Clin J Pain. 2004;20(4):207–19.

    Article  PubMed  Google Scholar 

  26. Anderson D. Development of an instrument to measure pain in rheumatoid arthritis: Rheumatoid Arthritis Pain Scale (RAPS). Arthritis Care Res. 2001;45:317–23.

    Article  CAS  Google Scholar 

  27. Pincus T, Swearingen C, Wolfe F. Toward a multidimensional Health Assessment Questionnaire (MDHAQ): assessment of advanced activities of daily living and psychological status in the patient-friendly health assessment questionnaire format. Arthritis Rheum. 1999;42:2220–30.

    Article  CAS  PubMed  Google Scholar 

  28. Wolfe F, Michaud K, Pincus T. Preliminary evaluation of a visual analog function scale for use in rheumatoid arthritis. J Rheumatol. 2005;32:1261–6.

    PubMed  Google Scholar 

  29. Bellamy N, Buchanan WW, Goldsmith CH, Campbell J, Stitt LW. Validation study of WOMAC: a health status instrument for measuring clinically important patient relevant outcomes to antirheumatic drug therapy in patients with osteoarthritis of the hip or knee. J Rheumatol. 1988;15:1833–40.

    CAS  PubMed  Google Scholar 

  30. WHOQOL. The World Health Organisation Quality of Life (WHOQOL)-BREF UK version. WHO: Geneva; 2004.

  31. WHOQOL. The World Health Organisation Quality of Life (WHOQOL)—100. WHO: Geneva; 1995.

  32. Meenan RF, Mason JH, Anderson JJ, Guccione AA, Kazis LE. AIMS2: the content and properties of a revised and expanded Arthritis Impact Measurement Scales health status questionnaire. Arthritis Rheum. 1992;35:1–10.

    Article  CAS  PubMed  Google Scholar 

  33. Diener E, Emmons RA, Larsen RJ, Griffin S. The satisfaction with life scale. J Pers Assess. 1985;49(1):71–5.

    Article  CAS  PubMed  Google Scholar 

  34. Stuifbergen AK, Seraphine A, Roberts G. An explanatory model of health promotion and quality of life in chronic disabling conditions. Nurs Res. 2000;49(3):122–9.

    Article  CAS  PubMed  Google Scholar 

  35. de Jong Z, van der Heijde D, McKenna SP, Whalley D. The reliability and construct validity of the RAQoL: a rheumatoid arthritis-specific quality of life instrument. Br J Rheumatol. 1997;36:878–83.

    Article  PubMed  Google Scholar 

  36. Nicassio PM, Wallston KA, Callahan LF, Herbert M, Pincus P. The measurement of helplessness in rheumatoid arthritis: the development of the Arthritis Helplessness Index. J Rheumatol. 1985;12:462–7.

    CAS  PubMed  Google Scholar 

  37. Nicklin J, Cramp F, Kirwan J, Greenwood R, Urban M, Hewlett S. Measuring fatigue in rheumatoid arthritis: a cross-sectional study to evaluate the Bristol Rheumatoid Arthritis Fatigue Multi-Dimensional questionnaire, visual analog scales, and numerical rating scales. Arthritis Care Res. 2010;62(11):1559–68.

    Article  Google Scholar 

  38. Khanna D, Pope JE, Khanna PP, Maloney M, Samedi N, Norrie D, Ouimet G, Hays R. The minimally important difference for the fatigue visual analog scale in patients with rheumatoid arthritis followed in an academic clinical practice. J Rheumatol. 2008;35(12):2335–43.

    Article  Google Scholar 

  39. Westhovens R, Bole JC, Li T, Martin M, MacLean R, Lin P, Blaisdell B, Wallenstein GV, Aranda R, Sherrer Y. Improved health-related quality of life for rheumatoid arthritis patients treated with abatercept who have inadequate response to anti-TNF therapy in a double-blind, placebo-controlled, multicentre randomized clinical trial. Rheumatology. 2006;45(10):1238–46.

    Article  CAS  PubMed  Google Scholar 

  40. Brazier JE, Harper R, Jones NMB, O’Cathain A, Thomas KJ, Usherwood T, Westlake L. Validating the SF-36 health survey questionnaire: new outcome measure for primary care. BMJ. 1992;305:160–4.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  41. Devins GM, Binik YM, Hutchinson TA, Hollomby DJ, Barré PE, Guttmann RD. The emotional impact of end-stage renal disease: importance of patients’ perceptions of intrusiveness and control. Int J Psychiatry Med. 1983;13(4):327–43.

    Article  PubMed  Google Scholar 

  42. Katz PP, Radvanski DC, Allen D, Buyske S, Schiff S, Nadkarni A, Rosenblatt L, Maclean R, Hassett AL. Development and validation of a short form of the valued life activities disability questionnaire for rheumatoid arthritis. Arthritis Care Res (Hoboken). 2011;63(12):1664–71.

    Article  PubMed  PubMed Central  Google Scholar 

  43. Graneheim UH, Lundman B. Qualitative content analysis in nursing research: concepts, procedures and measures to achieve trustworthiness. Nurse Educ Today. 2004;24(2):105–12.

    Article  CAS  PubMed  Google Scholar 

  44. Nicklin J, Cramp F, Kirwan J, Urban M, Hewlett S. Collaboration with patients in the design of patient-reported outcome measures: capturing the experience of fatigue in rheumatoid arthritis. Arthritis Care Res. 2010;62(11):1552–8.

    Article  Google Scholar 

  45. Sanderson T, Hewlett S, Flurey C, Dures E, Richards P, Kirwan J. The impact triad (severity, importance and self-management) as a method of enhancing the measurement of the personal life impact of rheumatic diseases (Editorial). J Rheumatol. 2011;38(2):191–4.

    Article  PubMed  Google Scholar 

  46. Collins D. Pretesting survey instruments: an overview of cognitive methods. Qual Life Res. 2008;12(2):229–38.

    Google Scholar 

  47. Hewlett S, Wit M, Richards P, Quest E, Hughes R, Heiberg T, Kirwan J. Patients and professionals as research partners: challenges, practicalities, and benefits. Arthritis Rheum. 2006;55:676–80.

    Article  PubMed  Google Scholar 

Download references

Acknowledgments

We appreciate the support with recruitment at the Royal National Hospital for Rheumatic Diseases by Dr. Madeleine Piper, Nina Griffith and specialist nurses Sue Brown, Debbie Bond and Nicola Waldron; the support with recruitment at the BRI by Jan Richardson and specialist nurses Tracy French and Sue Taylor; Anne Watts, patient research partner. All authors contributed to the design, funding application, analysis and authorship of this paper. TS and CA conducted the data collection. TS is the guarantor for the study.

Compliance with Ethical Standards

Funding: This study was supported by an Arthritis Research UK feasibility study Grant No. 19434.

Conflicts of interest: Dr. Sanderson, Prof. Kirwan, Ms. Almeida, Dr. Morris, Mr. Noddings and Prof. Hewlett have no conflicts of interest.

Ethical approval: Approval was granted by the London Dulwich Research Ethics Committee (11/LO/1524).

Informed consent: Written informed consent was obtained for participating in the consultation meetings and focus groups, the recording of discussion and the use of anonymised quotations.

Author information

Authors and Affiliations

  1. Department of Anthropology, Durham University, Durham, DH1 3LE, UK

    Tessa Sanderson

  2. Academic Rheumatology Unit, Bristol Royal Infirmary, University of Bristol, Bristol, BS2 8HW, UK

    John Kirwan & Robert Noddings

  3. University of the West of England, Glenside Campus, Blackberry Hill, Bristol, BS16 1DD, UK

    Celia Almeida, Marianne Morris & Sarah Hewlett

Authors
  1. Tessa Sanderson
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. John Kirwan
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. Celia Almeida
    View author publications

    You can also search for this author in PubMed Google Scholar

  4. Marianne Morris
    View author publications

    You can also search for this author in PubMed Google Scholar

  5. Robert Noddings
    View author publications

    You can also search for this author in PubMed Google Scholar

  6. Sarah Hewlett
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to Tessa Sanderson.

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Sanderson, T., Kirwan, J., Almeida, C. et al. Item Development and Face Validity of the Rheumatoid Arthritis Patient Priorities in Pharmacological Interventions Outcome Measures. Patient 9, 103–115 (2016). https://doi.org/10.1007/s40271-015-0130-3

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1007/s40271-015-0130-3

Keywords

Search

Navigation