Doctor: Alice Biggane
Title: Prioritising health outcomes with patients and members of the public in the context of core outcome sets and National Institute for Health and Care Excellence guidance
Supervisors: Paula Williamson and Philippe Ravaud
Doctoral school: ED 393 Epidemiology and Biomedical Information Sciences, Université Paris Cité
Date of thesis defense: 30/06/2020
Jury: Declan Devane, Bruno Falissard, Veronique Sebille, Paula Williamson, Philippe Ravaud
Core outcome sets (COS) are agreed minimum sets of health outcomes that should be measured and reported in all relevant trials. COS development with patient and public input can help ensure the resulting COS reflects their needs and priorities. Similarly, patient and public input in health outcome selection of clinical guideline development can help ensure the resulting guidance is relevant to patients. This thesis investigated methods and perspectives surrounding patient and public input in COS and clinical guideline development and identified pointers to support future research in this area.
A survey of COS developers mapped commonly used methods of patient participation. A qualitative interview study explored participant experiences of the COS development methods. An ethnographic study investigated patient and public influence on health outcome selection in clinical guideline development. Discussion with a range of early stage researchers (ESRs) and European consultants enabled reflection on the roles of patients and members of the public in health research.
Survey responses indicated that patient participants were included in 87% (141/162) of published, completed or ongoing COS. The Delphi survey was used singularly or in combination with other methods in 85% (119/140) of projects. The survey findings also highlighted the increasingly global nature of COS development. I interviewed 24 patients and health professionals about their experiences of participation in COS Delphi studies. Some interviewees struggled to understand the purpose of COS and aspects of the Delphi survey. Interviewees differed in how they interpreted and subsequently used the written documentation provided to COS participants. They wanted guidance regarding the use of the scoring system and stakeholder feedback. My ethnography included 230 hours of observations and 18 interviews. This identified the need for continued support and guidance for patients and the public by the committee, specifically, the chairperson, during guideline development. Specific recommendations include the use of plain language, specifically inviting patient and public input, and alternative methods of facilitating involvement including the use of COS previously developed with patient input. Discussion with ESRs and European consultants in combination with the other data in this study identified different perspectives including perceived challenges surrounding the role of patients in methodological health research and health outcome prioritisation. Further international conversation, collaboration and training in identifying and facilitating the various roles patients have in health research is needed.
There has been an increase in patient and public input in COS development, but a lack of parallel increased focus on how to optimise such patient and public input, internationally and across other methodological health research. The findings of this thesis will inform the development of guidance and research in these areas and help to improve methods. International collaboration is also needed to progress patient and public input in health research generally.