Representation of published core outcome sets for research in regulatory guidance: protocol

Dodd, Susanna ORCID: 0000-0003-2851-3337, Fish, Rebecca, Gorst, Sarah ORCID: 0000-0002-7818-9646, Hall, Deborah ORCID: 0000-0002-3804-1452, Jacobsen, Pamela ORCID: 0000-0001-8847-7775, Kirkham, Jamie, Main, Barry, Matvienko-Sikar, Karen ORCID: 0000-0003-2777-6581, Saldanha, Ian, Trépel, Dominic
et al (show 1 more authors) (2021) Representation of published core outcome sets for research in regulatory guidance: protocol. HRB Open Research, 4. p. 45.

Access the full-text of this item by clicking on the Open Access link.


<h4>Background: </h4>   The COMET Initiative promotes the development and use of ‘core outcome sets’ (COS), agreed standardised sets of outcomes that should be measured and reported in all studies in a particular clinical condition. COS are determined by consensus amongst key stakeholders, including health professionals, policymakers and patients, ensuring that the priorities and expertise of these representatives inform the choice of the most important outcomes to measure for a given condition. There is increased recognition of the need to integrate COS across the healthcare system and with existing regulatory apparatus, to ensure that outcomes being recorded are those of key relevance to important stakeholders. The aim of this study is to assess the degree of concordance between outcomes recommended in COS for research and in guidance provided by two key regulators: US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). <h4>Methods: </h4>:  COS for research published during 2015-2019 with patient involvement and covering drug or device interventions will be compared against relevant regulatory guidelines, matched by condition. Guidance documents matching in scope (relating to intervention and population) to a COS for research will be scrutinised to identify all suggested outcomes for comparison against the core outcomes in the corresponding COS. <h4>Discussion: </h4>   This study will identify variation between outcomes suggested in FDA and EMA regulatory guidance relative to outcomes included in published COS for research, thus demonstrating the degree of representation of COS in regulatory guidance and vice versa. We will share the study findings (in particular, highlighting any lack of concordance between COS and regulatory guidance overall or for particular disease areas) and will invite feedback from FDA and EMA; we will seek to highlight where findings support the recommendations towards using well-developed COS or will make recommendations to COS developers on outcomes of importance to these key regulators.

Item Type: Article
Divisions: Faculty of Health and Life Sciences
Faculty of Health and Life Sciences > Institute of Population Health
Depositing User: Symplectic Admin
Date Deposited: 10 Dec 2021 11:34
Last Modified: 18 Jan 2023 21:23
DOI: 10.12688/hrbopenres.13139.3
Open Access URL:
Related URLs: