Patient and family engagement in the development of core outcome sets for two rare chronic diseases in children

Abstract

Background Core outcome sets (COS) are lists of consensus-determined outcomes to be measured and reported in all clinical research studies within a disease area. While including patients and families in COS development to improve their relevance and applicability to patient values is key, there is limited literature documenting practical barriers and facilitators to successful patient engagement in COS development. In this paper, as researchers and patient partners, we provide a resource for COS developers to meaningfully and effectively engage patients and families. Main body To establish a consensus-based COS for children with two inherited metabolic diseases (medium-chain acyl-CoA dehydrogenase deficiency and phenylketonuria), we conducted an evidence review, Delphi survey, and workshop. Two adult patient partner co-investigators co-developed the study protocol, co-designed strategies to address challenges with incorporating patient perspectives, and led all patient engagement activities, including communication with a group of family advisors. Seven adult family advisors received training about COS development and subsequently contributed to Delphi survey development, outcome definitions, the consensus workshop, and selection of outcome measurement instruments. Patient partner co-investigators and family advisors were essential to the successful design, conduct, and completion of the two COS. Patient partner co-investigators supported the understanding, inclusion and engagement of family advisors, and helped develop accessible tools to determine patient-oriented outcome measurement instruments. Patient partner co-investigators and family advisors collaborated with the study team to co-develop surveys, modify technical language, and recruit participants to the study. Together, we addressed challenges to patient engagement in COS development such as unfamiliarity with study methods, comprehensibility of materials and ongoing engagement, and power imbalances between team members. Conclusion Our approach to patient and family engagement in COS development for two rare conditions for children was feasible and considered valuable by all study team members, including patients and family members, in improving the relevance of the deliverable to patients. This approach to patient engagement in developing COS can be applied to other paediatric disease contexts, allowing patient and family perspectives to influence the direction of future studies to develop COS.

Development of minimum reporting sets of patient characteristics in epidemiological research: a methodological systematic review

“…However, embracing patients’ perspective on certain variables in the final set could be helpful, especially when these variables are private information of patients such as socioeconomic status, income, family history etc. Methods for patient engagement has recently been proposed for core outcome set, which could be further adjusted for the development of CPCS (45, 46). Besides, many CPCS studies do not report how the representativeness of participants is ensured.…”

Development of minimum reporting sets of patient characteristics in epidemiological research: A methodological systematic review

“…Methods for patient engagement have recently been proposed for core outcome sets, which could be further adjusted for the development of CPCSs. 52,53 Besides, many CPCS studies do not report how the representativeness of participants is ensured. Such information is important to determine the quality of the obtained CPCS and its uptake, hence should be better reported in future practice.…”

Patient Engagement in a Multi-Stakeholder Workshop to Plan the Collection of Patient-Oriented Outcomes for Children with Inherited Metabolic Diseases

“…This workshop followed the completion of a COS study that incorporated a comprehensive patient engagement strategy (Vanderhout et al 2021). This facilitated the co-designing of the workshop with the patient partners who had been investigators on that study and contributions from some of the same family advisors.…”

“…Our patient engagement strategy for the COS study has also been described in another study (Vanderhout et al 2021). Briefly, two patient or family member partners were engaged as co-investigators throughout the study and a Family Advisory Forum (FAF), which included seven parents of children with IMDs, were engaged at key stages.…”