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Objectives: The objective of this study was to determine the proportion of all published core outcome set (COS) studies that include an adverse event or harm outcome, to determine the proportion of individual vs pooled harms, and to investigate characteristics that influence their inclusion., Methods: We examined the extent to which a sample of 100 published COS studies (from January 2021 to January 2023) include both pooled and individual harms in the final COS. One investigator extracted the information from the COS studies, which was cross-checked against previous COS investigational research, and where possible verified with COS authors or a pharmacologist. Using Qualtrics™, we conducted a personalized online survey of developers of the 100 COS to ask them about the importance, their experiences, and methodological approaches for dealing with harms within their COS development studies., Results: One hundred COS were identified from 91 separate COS studies, the majority of which considered most of the minimum standards for development. Two-thirds (65%) of the COS included at least 1 harm outcome. In total, 1104 core outcomes were identified across the 100 COS, of which 184 (17%) were harm outcomes (154 individual vs 56 pooled). Individual harms were more likely to be included in a final COS if they were developed for single treatment interventions (50%) compared to those being developed for multitreatment modalities (39%). Some COS developers adopted outcome frameworks as part of their COS development process to facilitate the inclusion of harm outcomes in their final COS. A third (33%) of respondents felt that harm outcomes should be included in all COS but over half (56%) thought this would be dependent on some aspect of the scope of the COS and improved methodology and awareness of how to deal with harm outcomes in the COS development process., Conclusion: Harm outcomes are already included in many COS either as individual or pooled harms. It is evident that there are some challenges with regards to both the methodology and necessity to include harms within a COS (pooled or individual. COS developers should carefully consider the need to include important harms outcomes in relation to the scope of the COS that they are developing., Competing Interests: Declaration of competing interest J.J.K and J.B. are members of the Core Outcome Measures in Effectiveness Trials Initiative. There are no competing interests for any other author., (Crown Copyright © 2024. Published by Elsevier Inc. All rights reserved.)

Introduction: Core outcome sets (COSs) are agreed outcomes (domains (subdomains) and instruments) that should be measured as a minimum in clinical trials or practice in certain diseases or clinical fields. Worldwide, the number of COSs is increasing and there might be conceptual overlaps of domains (subdomains) and instruments within disciplines. The aim of this scoping review is to map and to classify all outcomes identified with COS projects relating to skin diseases., Methods and Analysis: We will conduct a scoping review of outcomes of skin disease-related COS initiatives to identify all concepts and their definitions. We will search PubMed, Embase and Cochrane library. The search dates will be 1 January 2010 (the point at which Core Outcome Measures in Effectiveness Trials (COMET) was established) to 1 January 2024. We will also review the COMET database and C3 website to identify parts of COSs (domains and/or instruments) that are being developed and published. This review will be supplemented by querying relevant stakeholders from COS organisations, dermatology organisations and patient organisations for additional COSs that were developed. The resulting long lists of outcomes will then be mapped into conceptually similar concepts., Ethics and Dissemination: This study was supported by departmental research funds from the Department of Dermatology at Northwestern University. An ethics committee review was waived since this protocol was done by staff researchers with no involvement of patient care. Conflicts of interests, if any, will be addressed by replacing participants with relevant conflicts or reassigning them. The results will be disseminated through publication in peer-reviewed journals, social media posts and promotion by COS organisations., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Objectives: A core outcome set (COS) is an agreed standardised minimum collection of outcomes that should be measured and reported in research in a specific area of health. Cochrane systematic reviews ('reviews') are rigorous reviews on health-related topics conducted under the auspices of Cochrane. This study examines the use of existing COS to inform the choice of outcomes in Cochrane systematic reviews ('reviews') and investigates the views of the coordinating editors of Cochrane Review Groups (CRGs) on this topic., Methods: A cohort of 100 recently published or updated Cochrane reviews were assessed for reference to a COS being used to inform the choice of outcomes for the review. Existing COS, published 2 or more years before the review publication, were then identified to assess how often a reviewer could have used a relevant COS if it was available. We asked 52 CRG coordinating editors about their involvement in COS development, how outcomes are selected for reviews in their CRG and their views of the advantages and challenges surrounding the standardisation of outcomes within their CRG., Results: In the cohort of reviews from 2019, 40% (40/100) of reviewers noted problems due to outcome inconsistency across the included studies. In 7% (7/100) of reviews, a COS was referenced in relation to the choice of outcomes for the review. Relevant existing COS could be considered for a review update in 35% of the others (33/93). Most editors who responded (31/36, 86%) thought that COS should definitely or possibly be used to inform the choice of outcomes in a review., Conclusions: Systematic reviewers are continuing to note outcome heterogeneity but are starting to use COS to inform their reviews. There is potential for greater uptake of COS in Cochrane reviews., Competing Interests: Competing interests: PRW and MC are members of the COMET (Core Outcome Measures in Effectiveness Trials) Management Group. JJK and JS are members of the CS-COUSIN (Cochrane Skin—Core Outcome Set Initiative) methods group. The remaining authors declare no competing interests., (© Author(s) (or their employer(s)) 2020. Re-use permitted under CC BY. Published by BMJ.)

Objectives: Billions of dollars have been invested in Health Information Technologies (HITs), and randomized controlled trials (RCTs) have been conducted to identify the effects of these interventions. Our objective was to identify the types of outcomes that were measured and reported in these RCTs., Study Design and Setting: We completed a systematic review (Medline, EMBASE, and CENTRAL databases) of RCTs involving children (<18 years) and utilizing HIT interventions., Results: We identified 45 RCTs involving 323,945 children. Most studies reported process outcomes (n = 40/45 (88.9%)) but did not include patient-focused outcomes such as patient/carer functioning (n = 12/45 (26.7%)), clinical/physiological health (n = 10/45, 22.2%), quality of life (n = 3/45, 6.7%), or mortality (n = 1/45, 2.2%). Only 3 of 45 (6.7%) studies reported an evaluation of adverse events. In only 14 of 45 (31.1%) studies was it clear that all outcomes that were measured were reported., Conclusion: It is difficult to use RCTs to fully evaluate the benefits and risks of using HIT interventions in pediatric health care settings because patient-focused outcomes and adverse events are rarely reported. Measures to improve the quality of future trials may include the publication of study protocols and the development of an outcome reporting framework or core outcome set., (Copyright © 2019 Elsevier Inc. All rights reserved.)

Objective: Establishing a research agenda on standardizing pain measurement in clinical trials in rheumatic and musculoskeletal diseases (RMD)., Methods: Discussion during a meeting at the Outcome Measures in Rheumatology (OMERACT) 2018, prepared by a systematic review of existing core outcome sets and a patient online survey., Results: Several key questions were debated: Is pain a symptom or a disease? Are pain core (sub)domains consistent across RMD? How to account for pain mechanistic descriptors (e.g., central sensitization) in pain measurement?, Conclusion: Characterizing and assessing the spectrum of pain experience across RMD in a standardized fashion is the objective of the OMERACT Pain Working Group.

Background: Core outcome sets (COS) have the potential to reduce waste in research by improving the consistency of outcomes measured in trials of the same health condition. However, this reduction in waste will only be realised through the uptake of COS by clinical trialists. Without uptake, the continued development of COS that are not implemented may add to waste in research. Funders of clinical trials have the potential to have an impact on COS uptake by recommending their use to those applying for funding. The aim of our study was to assess the extent to which applicants followed the National Institute for Health Research Health Technology Assessment (NIHR HTA) programme's recommendation to search for a COS to include in their clinical trial., Methods and Findings: We examined the outcomes section and detailed project descriptions of all 95 researcher-led primary research applications submitted to the NIHR HTA between January 2012, when the recommendation to search for a COS was included in the guidance for applicants, and December 2015 for evidence that a search for a COS had taken place and rationale for outcome choice in the absence of COS. A survey of applicants was conducted to further explore their use of COS and choice of outcomes with a response rate of 49%. Nine out of 95 applicants (10%) stated in their application that they had searched the COMET (Core Outcome Measures for Effectiveness Trials) Initiative database for a COS and another nine referred to searching for a COS using another method, e.g. a review of the literature. Of the 77 (81%) applicants that did not mention COMET or COS in their application, eight stated in the survey that they had searched the COMET database and ten carried out a search using another method. Some applicants who did not search for a COS gave reasons for their choice of outcomes including taking advice from patients and the public and choosing outcomes used in previous trials., Conclusion: A funding body can have an impact on COS uptake by encouraging trialists to search for a COS. Funders could take further steps by putting processes in place to prompt applicants to be explicit about searching for COS in their application and notifying the funding board if a search has not taken place. The sources of information used by trialists to make decisions about outcomes in the absence of COS may suggest methods of dissemination for COS., Competing Interests: I have read the journal’s policy and the authors of this manuscript have the following competing interests: MC and PRW are members of the COMET Management Group. KLH and JJK declare that no competing interests exist. This does not alter our adherence to PLOS ONE policies on sharing data and materials.

Objectives: Visual impairment following stroke is common with a reported incidence of visual impairment in 60% of stroke survivors. Screening for visual impairment is neither routine nor standardised. This results in a health inequality where some stroke survivors receive comprehensive vision assessment to identify any existent visual problems while others receive no vision assessment leaving them with unmet needs from undiagnosed visual problems. The aim of this study was to define two core outcome sets (COS), one for vision screening and one for full visual assessment of stroke survivors., Design: A list of potentially relevant visual assessments was created from a review of the literature. The consensus process consisted of an online 3-round Delphi survey followed by a consensus meeting of the key stakeholders., Participants: Stakeholders included orthoptists, occupational therapists, ophthalmologists, stroke survivors and COS users such as researchers, journal editors and guideline developers., Setting: University., Outcome Measures: COS., Results: Following the consensus process we recommend the following nine assessments for vision screening: case history, clinical observations of visual signs, visual acuity, eye alignment position, eye movement assessment, visual field assessment, visual neglect assessment, functional vision assessment and reading assessment. We recommend the following 11 assessments for full vision assessment: case history, observations, visual acuity, eye alignment position, eye movement assessment, binocular vision assessment, eye position measurement, visual field assessment, visual neglect assessment, functional vision assessment, reading assessment and quality of life questionnaires., Conclusions: COS are defined for vision screening and full vision assessment for stroke survivors. There is potential for their use in reducing heterogeneity in routine clinical practice and for improving standardisation and accuracy of vision assessment. Future research is required to evaluate the use of these COS and for further exploration of core outcome measures., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2019. Re-use permitted under CC BY. Published by BMJ.)

Objective: The Core Outcome Set-STAndards for Development (COS-STAD) contains 11 standards (12 criteria) that are deemed to be the minimum design recommendations for all core outcome set (COS) development projects. Cancer is currently the disease area with the highest number of published COSs and is a major cause of worldwide morbidity and mortality. The aim of this study was to provide a baseline of cancer COS standards., Study Design and Setting: Systematic reviews of COSs have identified 307 published COS studies. Cancer COSs were eligible for inclusion. Two reviewers independently assessed each of the COSs against the 12 criteria., Results: Forty-nine cancer COSs were included; none met all 12 criteria representing the 11 minimum standards assessed in this study (range = 4-11 criteria, median = 6 criteria). All studies met the four scope standards, eight (16%) met all three standards for stakeholders involved, and two (4%) met all four standards for consensus process standards., Conclusion: With the exception of "scope" specification, there is much need for improvement. Poor reporting often made it challenging to assess whether minimum standards were met. The consensus process criteria were most difficult to assess, particularly those that required an assessment of being a priori. This is the first application of COS-STAD criteria to studies that have developed COSs and provides a baseline of cancer COS standards of development., (Copyright © 2019 The Authors. Published by Elsevier Inc. All rights reserved.)

Background: The use of core outcome sets (COS) ensures that researchers measure and report those outcomes that are most likely to be relevant to users of their research. Several hundred COS projects have been systematically identified to date, but there has been no formal quality assessment of these studies. The Core Outcome Set-STAndards for Development (COS-STAD) project aimed to identify minimum standards for the design of a COS study agreed upon by an international group, while other specific guidance exists for the final reporting of COS development studies (Core Outcome Set-STAndards for Reporting [COS-STAR])., Methods and Findings: An international group of experienced COS developers, methodologists, journal editors, potential users of COS (clinical trialists, systematic reviewers, and clinical guideline developers), and patient representatives produced the COS-STAD recommendations to help improve the quality of COS development and support the assessment of whether a COS had been developed using a reasonable approach. An open survey of experts generated an initial list of items, which was refined by a 2-round Delphi survey involving nearly 250 participants representing key stakeholder groups. Participants assigned importance ratings for each item using a 1-9 scale. Consensus that an item should be included in the set of minimum standards was defined as at least 70% of the voting participants from each stakeholder group providing a score between 7 and 9. The Delphi survey was followed by a consensus discussion with the study management group representing multiple stakeholder groups. COS-STAD contains 11 minimum standards that are the minimum design recommendations for all COS development projects. The recommendations focus on 3 key domains: the scope, the stakeholders, and the consensus process., Conclusions: The COS-STAD project has established 11 minimum standards to be followed by COS developers when planning their projects and by users when deciding whether a COS has been developed using reasonable methods.

Objectives: The aim of the study was to evaluate citation analysis as an approach to measuring core outcome set (COS) uptake, by assessing whether the number of citations for a COS report could be used as a surrogate measure of uptake of the COS by clinical trialists., Study Design and Setting: Citation data were obtained for COS reports published before 2010 in five disease areas (systemic sclerosis, rheumatoid arthritis, eczema, sepsis and critical care, and female sexual dysfunction). Those publications identified as a report of a clinical trial were examined to identify whether or not all outcomes in the COS were measured in the trial., Results: Clinical trials measuring the relevant COS made up a small proportion of the total number of citations for COS reports. Not all trials citing a COS report measured all the recommended outcomes. Some trials cited the COS reports for other reasons, including the definition of a condition or other trial design issues addressed by the COS report., Conclusion: Although citation data can be readily accessed, it should not be assumed that the citing of a COS report indicates that a trial has measured the recommended COS. Alternative methods for assessing COS uptake are needed., (Copyright © 2017 The Authors. Published by Elsevier Inc. All rights reserved.)

Background: Core outcome sets (COS) can enhance the relevance of research by ensuring that outcomes of importance to health service users and other people making choices about health care in a particular topic area are measured routinely. Over 200 COS to date have been developed, but the clarity of these reports is suboptimal. COS studies will not achieve their goal if reports of COS are not complete and transparent., Methods and Findings: In recognition of these issues, an international group that included experienced COS developers, methodologists, journal editors, potential users of COS (clinical trialists, systematic reviewers, and clinical guideline developers), and patient representatives developed the Core Outcome Set-STAndards for Reporting (COS-STAR) Statement as a reporting guideline for COS studies. The developmental process consisted of an initial reporting item generation stage and a two-round Delphi survey involving nearly 200 participants representing key stakeholder groups, followed by a consensus meeting. The COS-STAR Statement consists of a checklist of 18 items considered essential for transparent and complete reporting in all COS studies. The checklist items focus on the introduction, methods, results, and discussion section of a manuscript describing the development of a particular COS. A limitation of the COS-STAR Statement is that it was developed without representative views of low- and middle-income countries. COS have equal relevance to studies conducted in these areas, and, subsequently, this guideline may need to evolve over time to encompass any additional challenges from developing COS in these areas., Conclusions: With many ongoing COS studies underway, the COS-STAR Statement should be a helpful resource to improve the reporting of COS studies for the benefit of all COS users., Competing Interests: I have read the journal's policy and have the following conflicts: DGA, JMB, MC, EG, PRW, and ST are members of the COMET Management Group. DM is a member of the Editorial Board of PLOS Medicine. DD, SG, JJK, JS, and PT declare no competing interests.

Objectives: Outcome reporting bias (ORB) is a threat to validity of systematic reviews. Multivariate meta-analysis (MVMA) can potentially reduce the impact of ORB when outcomes are correlated. The aim of this study was to assess ORB in Cochrane systematic reviews of rheumatoid arthritis and to demonstrate how MVMA may examine its impact., Study Design and Setting: Reviews were assessed for ORB in relation to eight outcomes for rheumatoid arthritis using a nine-point classification system. Impact of ORB was assessed by comparing estimates from univariate meta-analysis and MVMA models., Results: ORB assessment was applied in 21 included reviews, and all contained missing data on at least one of the eight outcomes. ORB was highly suspected in 247 (22%) of the 1,118 evaluable outcomes from 155 assessable trials. MVMA and univariate results sometimes differed importantly. The maximum change in treatment effect estimate between MVMA and univariate meta-analysis approach was found to be 176% for one of the outcome considered., Conclusion: ORB has the potential to affect the conclusions in meta-analyses. This could be avoided if trialists reported on all measured outcomes in full. If missing outcome data are unobtainable, MVMA is useful to examine the impact of missing outcomes and ORB on conclusions., (Copyright © 2015 Elsevier Inc. All rights reserved.)

This study aims to investigate how well the Assessment of SpondyloArthritis international Society (ASAS)/Outcome Measures in Rheumatology Clinical Trials (OMERACT) core set and response criteria for ankylosing spondylitis (AS) have been implemented in randomized controlled trials (RCTs) testing pharmacological and non-pharmacological interventions. A systematic literature search was performed up to June 2013 looking for RCTs in patients with axial spondyloarthritis (SpA) (AS and non-radiographic axial SpA). The assessed domains and instruments belonging to the core sets for disease-controlling anti-rheumatic therapy (DC-ART) and symptom-modifying anti-rheumatic drugs (SMARDs) were extracted. Results were reported separately for those trials published until 2 years after the publication of the core set (1 April 2001; 'control trials') and those trials published at least 2 years after the publication date ('implementation trials'). One hundred twenty-three articles from 99 RCTs were included in the analysis, comparing 48 'control trials' and 51 'implementation trials'. Regarding DC-ART core set, the following domains were significantly more frequently assessed in the 'implementation group' in comparison to the 'control group': 'physical function' (100 vs 41.7 %; p ≤ 0.001), 'peripheral joints/entheses' (100 vs 33.3 %; p ≤ 0.001) and 'fatigue' (100 vs 0 %; p ≤ 0.001). Three instruments were significantly more used in the 'implementation group': Bath Ankylosing Spondylitis Functional Index (BASFI) (100 vs 8.3 %; p = ≤ 0.001), CRP (92.3 vs 58.3 %; p = 0.01) and Bath Ankylosing Spondylitis Metrology Index (BASMI) (53.8 vs 0 %; p = 0.001). Regarding SMARD core set domains, physical function (92 vs 23 %; p ≤ 0.001) and fatigue (84 vs 17 %; p ≤ 0.001), as well as the instruments BASFI (88 vs 14 %; p ≤ 0.001) and BASMI (52 vs 0 %; p ≤ 0.001), increased significantly in the 'implementation group'. Twenty per cent of trials from the 'implementation group' but none from the 'control group' included all domains of the core set. In conclusion, this study provides evidence for the implementation of the ASAS/OMERACT core set in RCTs of both DC-ART and SMARD. This applies to the use of the domains and, to a lesser extent, to the specific instruments.

Background: The development and application of standardised sets of outcomes to be measured and reported in clinical trials have the potential to increase the efficiency and value of research. One of the most notable of the current outcome sets began nearly 20 years ago: the World Health Organization and International League of Associations for Rheumatology core set of outcomes for rheumatoid arthritis clinical trials, originating from the OMERACT (Outcome Measures in Rheumatology) Initiative. This study assesses the use of this core outcome set by randomised trials in rheumatology., Methods: An observational review was carried out of 350 randomised trials for the treatment of rheumatoid arthritis identified through The Cochrane Library (up to and including September 2012 issue). Reports of these trials were evaluated to determine whether or not there were trends in the proportion of trials reporting on the full set of core outcomes over time. Researchers who conducted trials after the publication of the core set were contacted to assess their awareness of it and to collect reasons for non-inclusion of the full core set of outcomes in the study., Results: Since the introduction of the core set of outcomes for rheumatoid arthritis, the consistency of measurement of the core set of outcomes has improved, although variation in the choice of measurement instrument remains. The majority of trialists who responded said that they would consider using the core outcome set in the design of a new trial., Conclusions: This observational review suggests that a higher percentage of trialists conducting trials in rheumatoid arthritis are now measuring the rheumatoid arthritis core outcome set. Core outcome sets have the potential to improve the evidence base for health care, but consideration must be given to the methods for disseminating their availability amongst the relevant communities.

Background: The increased use of meta-analysis in systematic reviews of healthcare interventions has highlighted several types of bias that can arise during the completion of a randomised controlled trial. Study publication bias and outcome reporting bias have been recognised as a potential threat to the validity of meta-analysis and can make the readily available evidence unreliable for decision making., Methodology/principal Findings: In this update, we review and summarise the evidence from cohort studies that have assessed study publication bias or outcome reporting bias in randomised controlled trials. Twenty studies were eligible of which four were newly identified in this update. Only two followed the cohort all the way through from protocol approval to information regarding publication of outcomes. Fifteen of the studies investigated study publication bias and five investigated outcome reporting bias. Three studies have found that statistically significant outcomes had a higher odds of being fully reported compared to non-significant outcomes (range of odds ratios: 2.2 to 4.7). In comparing trial publications to protocols, we found that 40-62% of studies had at least one primary outcome that was changed, introduced, or omitted. We decided not to undertake meta-analysis due to the differences between studies., Conclusions: This update does not change the conclusions of the review in which 16 studies were included. Direct empirical evidence for the existence of study publication bias and outcome reporting bias is shown. There is strong evidence of an association between significant results and publication; studies that report positive or significant results are more likely to be published and outcomes that are statistically significant have higher odds of being fully reported. Publications have been found to be inconsistent with their protocols. Researchers need to be aware of the problems of both types of bias and efforts should be concentrated on improving the reporting of trials.

Background: Missing outcome data or the inconsistent reporting of outcome data in clinical research can affect the quality of evidence within a systematic review. A potential solution is an agreed standardized set of outcomes known as a core outcome set (COS) to be measured in all studies for a specific condition. We investigated the amount of missing patient data for primary outcomes in Cochrane systematic reviews, and surveyed the Co-ordinating Editors of Cochrane Review Groups (CRGs) on issues related to the standardization of outcomes in their CRG's reviews. These groups are responsible for the more than 7,000 protocols and full versions of Cochrane Reviews that are currently available, and the several hundred new reviews published each year, presenting the world's largest collection of standardized systematic reviews in health care., Methods: Using an unselected cohort of Cochrane Reviews, we calculated and presented the percentage of missing patient data for the primary outcome measure chosen for each review published by each CRG. We also surveyed the CRG Co-ordinating Editors to see what their policies are with regards to outcome selection and outcomes to include in the Summary of Finding (SoF) tables in their Cochrane Reviews. They were also asked to list the main advantages and challenges of standardizing outcomes across all reviews within their CRG., Results: In one fifth of the 283 reviews in the sample, more than 50% of the patient data for the primary outcome was missing. Responses to the survey were received from 90% of Co-ordinating Editors. Thirty-six percent of CRGs have a centralized policy regarding which outcomes to include in the SoF table and 73% of Co-ordinating Editors thought that a COS for effectiveness trials should be used routinely for a SoF table., Conclusions: The reliability of systematic reviews, in particular meta-analyses they contain, can be improved if more attention is paid to missing outcome data. The availability of COSs for specific health conditions might help with this and the concept has support from the majority of Co-ordinating Editors in CRGs.

Trauma is a term used in medicine for describing physical injury. The prospective evaluation of the care of injured patients aims to improve the management of a trauma system and acts as an ongoing audit of trauma care. One of the principal techniques used to evaluate the effectiveness of trauma care at different hospitals is through a comparative outcome analysis. In such an analysis, a national 'league table' can be compiled to determine which hospitals are better at managing trauma care. One of the problems with the conventional analysis is that key covariates for measuring physiological injury can often be missing. It is also hypothesized that this missingness is not missing at random (NMAR). We describe the methods used to assess the performance of hospitals in a trauma setting and implement the method of weights for generalized linear models to account for the missing covariate data, when we suspect the missing data mechanism is NMAR using a Monte Carlo EM algorithm. Through simulation work and application to the trauma data we demonstrate the affect the missing covariate data can have on the performance of hospitals and how the conclusions we draw from the analysis can differ. We highlight the differences in hospital performance and the ranking of hospitals., (Copyright (c) 2008 John Wiley & Sons, Ltd.)