The Relevance of Core Outcome Sets to Improving Healthcare Decision Making

Author(s)

Paula Williamson, Sarah Gorst, PhD, Susanna Dodd, PhD.
University of Liverpool, Liverpool, United Kingdom.
OBJECTIVES: Research shows that outcomes measured in clinical trials are not always those most important to healthcare decision-makers, including patients. A core outcome set (COS), agreed by key groups, could overcome these problems, being the minimum to be measured, and reported, in all clinical trials for a specific health condition. Core Outcome Sets (COS) have been endorsed internationally by trialist groups, funders, trial registries, regulatory bodies and industry, https://www.comet-initiative.org/COSEndorsement. In 2024, consideration of COS was endorsed in the World Health Organisation Guidance for Best Practices for Clinical Trials and for health technology assessment by the EC HTA Coordination Group. The objectives of this study were to assess COS development methods and describe the outcomes deemed core across health conditions.
METHODS: Studies reporting the development of a COS, published or indexed by 2024, were identified using established systematic review methods. Adherence to a set of internationally agreed consensus-based standards for COS development was assessed. An existing outcome taxonomy was used to classify the core outcomes from all studies.
RESULTS: 752 COS have been published by 2024, with a noticeable increase in methodological rigour over time. Patient participation continues to improve, from 15% upto 2013, 48% by 2021, to 87% in 2024. The trend of COS being developed for use in both clinical research and practice has continued, increasing from 10% upto 2013, 28% in 2021, to 59% in 2024. COS involving patients or their representatives more often included life impact outcomes (374/422, 89%) than COS without patient participation (207/330, 63%). Common core outcome domains include physical functioning and global quality of life, each included in over 40% COS.
CONCLUSIONS: Improving adherence to recognised COS development standards is evident, reflecting a meaningful shift toward more inclusive, patient-centred outcome selection. The broader application of COS, including in HTA, signals a maturing field responding to global policy endorsement.

Conference/Value in Health Info

2025-11, ISPOR Europe 2025, Glasgow, Scotland

Value in Health, Volume 28, Issue S2

Code

PCR240

Topic

Clinical Outcomes, Health Technology Assessment, Patient-Centered Research

Topic Subcategory

Patient Engagement

Disease

No Additional Disease & Conditions/Specialized Treatment Areas

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