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Patient and family engagement in the development of core outcome sets for two rare chronic diseases in children Vanderhout, Shelley M.; Smith, Maureen; Pallone, Nicole; Tingley, Kylie; Pugliese, Michael; Chakraborty, Pranesh; Stockler, Sylvia; Offringa, Martin; Butcher, Nancy; Nicholls, Stuart G.; et al.
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.
Item Metadata
Title |
Patient and family engagement in the development of core outcome sets for two rare chronic diseases in children
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Creator | |
Contributor | |
Publisher |
BioMed Central
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Date Issued |
2021-09-14
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Description |
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.
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Subject | |
Genre | |
Type | |
Language |
eng
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Date Available |
2021-10-08
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Provider |
Vancouver : University of British Columbia Library
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Rights |
Attribution 4.0 International (CC BY 4.0)
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DOI |
10.14288/1.0402479
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URI | |
Affiliation | |
Citation |
Research Involvement and Engagement. 2021 Sep 14;7(1):66
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Publisher DOI |
10.1186/s40900-021-00304-y
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Peer Review Status |
Reviewed
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Scholarly Level |
Faculty; Researcher
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Copyright Holder |
The Author(s)
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Rights URI | |
Aggregated Source Repository |
DSpace
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Item Media
Item Citations and Data
Rights
Attribution 4.0 International (CC BY 4.0)