Skip to main content
Log in

Increasing the Patient-Centeredness of Health Economics and Outcomes Research Through Patient Engagement in Core Outcome Set Development

  • Practical Application
  • Published:
The Patient - Patient-Centered Outcomes Research Aims and scope Submit manuscript

Abstract

Core outcome sets (COS) are becoming increasingly popular in clinical research and can provide important inputs for further health economics and outcomes research (HEOR) studies. Use of standard, consistently reported outcomes can demonstrate and allow differentiation of the effectiveness and value of different treatments. Incorporating patient values during COS development increases the patient centeredness of evidence available across decision-making contexts. However, the approach to meaningful patient engagement in the COS process is evolving and poses both unique challenges and opportunities. We describe an approach to patient-centered COS development and discuss challenges and adaptations to improve engagement across COS projects. We provide examples from our experience in patient engagement for COS development using three completed COS projects. This approach includes patient engagement in terms of partnering with patient organizations, orientation and training, and the consensus process. Including COS in clinical development programs and HEOR will ensure that relevant, consistent outcomes are available for healthcare decision making and should result in faster access to high-value and novel therapies for patients. Patient-centered COS development increases the likelihood that further HEOR studies and decisions made using the COS are relevant to patients.

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

Access this article

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

Instant access to the full article PDF.

Institutional subscriptions

Fig. 1

Similar content being viewed by others

References

  1. Clarke M. Standardising outcomes for clinical trials and systematic reviews. Trials. 2007;8(1):39.

    Article  Google Scholar 

  2. Skinner M, Clearfield E, Iorio A, Tunis SR. Comparing outcomes across clinical trials: core outcome set for hemophilia gene therapy as a model for other diseases. DIA Global Forum. 2017;9(5):16–7.

    Google Scholar 

  3. Williamson PR, Altman DG, Blazeby JM, Clarke M, Devane D, Gargon E, Tugwell P. Developing core outcome sets for clinical trials: issues to consider. Trials. 2012;13(1):132.

    Article  Google Scholar 

  4. Kirkham JJ, Gargon E, Clarke M, Williamson PR. Can a core outcome set improve the quality of systematic reviews? A survey of the Co-ordinating Editors of Cochrane Review Groups. Trials. 2013;14(1):21.

    Article  Google Scholar 

  5. RFA-FD-19-006 Development of Standard Core Clinical Outcomes Assessments (COAs) and Endpoints (UG3/UH3 Clinical Trial Optional). 2019. https://grants.nih.gov/grants/guide/rfa-files/RFA-FD-19-006.html. Accessed 30 Jan 2020.

  6. Gargon E, Gorst SL, Williamson PR. Choosing important health outcomes for comparative effectiveness research: 5th annual update to a systematic review of core outcome sets for research. PLoS One. 2019;14(12):e0225980.

    Article  CAS  Google Scholar 

  7. Gottlieb S. Implementation of the 21st century cures act: progress and the path forward for medical innovation. Testimony of Scott Gottleib, M.D. Commissioner of Food and Drugs Food and Drug Administration Before the Committee on Health, Education, Labor & Pensions, U.S. Senate. 2017. https://www.fda.gov/news-events/congressional-testimony/implementation-21st-century-cures-act-progress-and-path-forward-medical-innovation-12062017-12062017. Accessed 30 Jan 2020.

  8. Lowe MM, Blaser DA, Cone L, Arcona S, Ko J, Sasane R, Wicks P. Increasing patient involvement in drug development. Value Health. 2016;19(6):869–78.

    Article  Google Scholar 

  9. Frank L, Forsythe L, Ellis L, Schrandt S, Sheridan S, Gerson J, Konopka K, Daugherty S. Conceptual and practical foundations of patient engagement in research at the patient-centered outcomes research institute. Qual Life Res. 2015;24(5):1033–41.

    Article  Google Scholar 

  10. Gargon E, Gurung B, Medley N, Altman D, Blazeby J, Clarke M, Williamson P. Choosing important health outcomes for comparative effectiveness research: a systematic review. Value Health. 2014;17(7):A435.

    Article  CAS  Google Scholar 

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

    PubMed  Google Scholar 

  12. Mease PJ, Arnold LM, Crofford LJ, Williams DA, Russell IJ, Humphrey L, Abetz L, Martin SA. Identifying the clinical domains of fibromyalgia: contributions from clinician and patient Delphi exercises. Arthritis Rheum. 2008;59(7):952–60.

    Article  Google Scholar 

  13. Biggane AM, Brading L, Ravaud P, Young B, Williamson PR. Survey indicated that core outcome set development is increasingly including patients, being conducted internationally and using Delphi surveys. Trials. 2018;19(1):113.

    Article  Google Scholar 

  14. Williamson PR, Altman DG, Bagley H, Barnes KL, Blazeby JM, Brookes ST, Clarke M, Gargon E, Gorst S, Harman N, Kirkham JJ, McNair A, Prinsen CAC, Schmitt J, Terwee CB, Young B. The COMET Handbook: version 1.0. Trials. 2017;18(Suppl 3):280.

    Article  Google Scholar 

  15. Sinha IP, Smyth RL, Williamson PR. Using the Delphi technique to determine which outcomes to measure in clinical trials: recommendations for the future based on a systematic review of existing studies. PLoS Med. 2011;8(1):e1000393.

    Article  Google Scholar 

  16. Gargon E, Williamson PR, Young B. Improving core outcome set development: qualitative interviews with developers provided pointers to inform guidance. J Clin Epidemiol. 2017;86:140–52.

    Article  Google Scholar 

  17. Young B, Bagley H. Including patients in core outcome set development: issues to consider based on three workshops with around 100 international delegates. Res Involv Engagem. 2016;2(1):25.

    Article  Google Scholar 

  18. Tambor E, Shalowitz M, Harrington JM, Hull K, Watson N, Sital S, Al Naber J, Miller D. Engaging patients, clinicians, and the community in a clinical data research network: lessons learned from the CAPriCORN CDRN. Learn Health Syst. 2019;3(2):e10079.

    Article  Google Scholar 

  19. Lavallee DC, Williams CJ, Tambor ES, Deverka PA. Stakeholder engagement in comparative effectiveness research: how will we measure success? J Comp Eff Res. 2012;1(5):397–407.

    Article  Google Scholar 

  20. Hoffman A, Montgomery R, Aubry W, Tunis SR. How best to engage patients, doctors, and other stakeholders in designing comparative effectiveness studies. Health Aff. 2010;29(10):1834–41.

    Article  Google Scholar 

  21. Kirkham JJ, Davis K, Altman DG, Blazeby JM, Clarke M, Tunis S, Williamson PR. Core outcome set-STAndards for development: the COS-STAD recommendations. PLoS Med. 2017;14(11):e1002447.

    Article  Google Scholar 

  22. Kirkham JJ, Gorst S, Altman DG, Blazeby JM, Clarke M, Tunis S, Williamson PR, COS-STAP Group. Core outcome set-STAndardised protocol items: the COS-STAP statement. Trials. 2019;20(1):116.

    Article  Google Scholar 

  23. Kirkham JJ, Gorst S, Altman DG, Blazeby JM, Clarke M, Devane D, Gargon E, Moher D, Schmitt J, Tugwell P, Tunis S, Williamson PR. Core outcome Set-STAndards for reporting: the COS-STAR statement. PLoS Med. 2016;13(10):e1002148.

    Article  Google Scholar 

  24. Iorio A, Skinner MW, Clearfield E, Messner D, Pierce GF, Witkop M, Tunis S, coreHEM panel. Core outcome set for gene therapy in haemophilia: results of the coreHEM multistakeholder project. Haemophilia. 2018;24(4):e167–e172172.

    Article  CAS  Google Scholar 

  25. Nadglowski J, Clearfield E, Al Naber J, Greene KN, Miller V, Messner D. coreNASH: a core outcome set for nonalcoholic steatohepatitis. 2018. https://2018.obesityweek.com/abstract/corenash-a-core-outcome-set-for-nonalcoholic-steatohepatitis/index.html. Accessed 30 Jan 2020.

  26. Pierce GF, Ragni MV, van den Berg HM, Weill A, O'Mahony B, Skinner MW, Pipe SW. Establishing the appropriate primary endpoint in haemophilia gene therapy pivotal studies. Haemophilia. 2017;23(5):643–4.

    Article  CAS  Google Scholar 

  27. Riva JJ, Malik KM, Burnie SJ, Endicott AR, Busse JW. What is your research question? An introduction to the PICOT format for clinicians. J Can Chiropr Assoc. 2012;56(3):167–71.

    PubMed  PubMed Central  Google Scholar 

  28. Perfetto EM, Burke L, Oehrlein EM, Epstein RS. Patient-focused drug development: a new direction for collaboration. Med Care. 2015;53(1):9–17.

    Article  Google Scholar 

  29. Carlton J, Peasgood T, Khan S, Barber R, Bostock J, Keetharuth AD. An emerging framework for fully incorporating public involvement (PI) into patient-reported outcome measures (PROMs). J Patient Rep Outcomes. 2020;4(1):4.

    Article  CAS  Google Scholar 

  30. Kreis J, Puhan MA, Schunemann HJ, Dickersin K. Consumer involvement in systematic reviews of comparative effectiveness research. Health Expect. 2013;16(4):323–37.

    Article  Google Scholar 

  31. Khodyakov D, Grant S, Denger B, Kinnett K, Martin A, Peay H, Coulter I. Practical considerations in using online modified-Delphi approaches to engage patients and other stakeholders in clinical practice guideline development. Patient. 2020;13(1):11–21.

    Article  Google Scholar 

  32. Wiering B, de Boer D, Delnoij D. Patient involvement in the development of patient-reported outcome measures: the developers' perspective. BMC Health Serv Res. 2017;17(1):635.

    Article  Google Scholar 

  33. Vass C, Rigby D, Payne K. The role of qualitative research methods in discrete choice experiments. Med Decis Mak. 2017;37(3):298–313.

    Article  Google Scholar 

  34. Carroll SL, Embuldeniya G, Abelson J, McGillion M, Berkesse A, Healey JS. Questioning patient engagement: research scientists' perceptions of the challenges of patient engagement in a cardiovascular research network. Patient Prefer Adherence. 2017;11:1573.

    Article  Google Scholar 

  35. Harrison JD, Anderson WG, Fagan M, Robinson E, Schnipper J, Symczak G, Hanson C, Carnie MB, Banta J, Chen S, Duong J, Wong C, Auerbach AD. Patient and Family Advisory Councils (PFACs): identifying challenges and solutions to support engagement in research. Patient. 2018;11(4):413–23.

    Article  Google Scholar 

  36. Janssen EM, Segal JB, Bridges JF. A framework for instrument development of a choice experiment: an application to type 2 diabetes. Patient. 2016;9(5):465–79.

    Article  Google Scholar 

  37. Trail M, Nelson N, Van JN, Appel SH, Lai EC. Major stressors facing patients with amyotrophic lateral sclerosis (ALS): a survey to identify their concerns and to compare with those of their caregivers. Amyotroph Lateral Scler Other Motor Neuron Disord. 2004;5(1):40–5.

    Article  Google Scholar 

  38. Labott SM, Johnson TP, Fendrich M, Feeny NC. Emotional risks to respondents in survey research: some empirical evidence. J Empir Res Hum Res Ethics. 2013;8(4):53–66.

    Article  Google Scholar 

  39. Oehrlein EM, Love TR, Anyanwu C, Hanna ML, Kraska J, Perfetto EM. Multi-method patient-engagement approach: a case example from a PCORI-funded training project. Patient. 2019;12(2):277–80.

    Article  Google Scholar 

  40. Hamilton CB, Hoens AM, McQuitty S, McKinnon AM, English K, Backman CL, Azimi T, Khodarahmi N, Li LC. Development and pre-testing of the Patient Engagement In Research Scale (PEIRS) to assess the quality of engagement from a patient perspective. PLoS One. 2018;13(11):e0206588.

    Article  Google Scholar 

Download references

Acknowledgements

The authors sincerely thank all advisory board members and Delphi voting members of coreHEM, coreNASH, and coreSCD for their time and contributions to these studies.

Author information

Authors and Affiliations

Authors

Contributions

Ms. EC and Ms. ET contributed to the study design, conceptualization, data acquisition, analysis, and manuscript preparation. Dr. EMJ contributed to conceptualization, data analysis, and manuscript preparation. Dr. DAM contributed to study design, conceptualization, and manuscript preparation.

Corresponding author

Correspondence to Ellen M. Janssen.

Ethics declarations

Funding

coreHEM, coreNASH, and coreSCD were funded by a pre-competitive consortium of life science industry companies, academic gene therapy groups, and patient advocacy organizations. No funding was received for the preparation of this manuscript.

Conflict of Interest

Ms. Clearfield, Ms. Tambor, Dr. Janssen, and Dr. Messner have no competing financial or nonfinancial interests that are directly relevant to the content of this article.

Ethical Approval

This research was conducted in accordance with the Declaration of Helsinki. The coreHEM, coreNASH, and coreSCD studies were reviewed by Chesapeake Institutional Review Board (now Advarra) and found to be exempt from human subjects’ research requirements.

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Clearfield, E., Tambor, E., Janssen, E.M. et al. Increasing the Patient-Centeredness of Health Economics and Outcomes Research Through Patient Engagement in Core Outcome Set Development. Patient 14, 413–420 (2021). https://doi.org/10.1007/s40271-020-00424-9

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1007/s40271-020-00424-9

Navigation