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