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 surve

SUBMITTER: Vanderhout SM 

PROVIDER: S-EPMC8439069 | biostudies-literature | 2021 Sep

REPOSITORIES: biostudies-literature