Use of core outcome sets was low in clinical trials published in major medical journals

Matvienko‐Sikar, Karen; Avery, Kerry; Blazeby, Jane M; Devane, Declan; Dodd, Susanna; Egan, Aoife M.; Gorst, Sarah L.; Hughes, Karen; Jacobsen, Pamela; Kirkham, Jamie J; Kottner, Jan; Mellor, Katie; Millward, Christopher P; Patel, Smitaa; Quirke, Fiona; Saldanha, Ian J.; Smith, Valerie; Terwee, Caroline B.; Young, Amber; Williamson, Paula

doi:10.1016/j.jclinepi.2021.10.012

Cited by 45 publications

(36 citation statements)

References 23 publications

(43 reference statements)

Supporting

Mentioning

Contrasting

Order By: Relevance

“…It may still be necessary to decide which outcome in a core outcome set is most important but the people to decide that are patients/patient representatives and healthcare professionals, not researchers. Despite the availability of core outcome sets, 98% of trials do not use them, even when a relevant core outcome set exists [ 12 ]. Matvienko-Sikar and colleagues found that the most common barrier to the use of a core outcome set was trial team’s own outcome preferences [ 12 ] and as our work shows, those preferences do not always align with those of patients and healthcare professionals.…”

Section: Discussionmentioning

confidence: 99%

“…Despite the availability of core outcome sets, 98% of trials do not use them, even when a relevant core outcome set exists [ 12 ]. Matvienko-Sikar and colleagues found that the most common barrier to the use of a core outcome set was trial team’s own outcome preferences [ 12 ] and as our work shows, those preferences do not always align with those of patients and healthcare professionals.…”

Section: Discussionmentioning

confidence: 99%

See 1 more Smart Citation

Getting it wrong most of the time? Comparing trialists’ choice of primary outcome with what patients and health professionals want

Treweek

Miyakoda²,

Burke

et al. 2022

Trials

View full text Add to dashboard Cite

Background Randomised trials support improved decision-making through the data they collect. One important piece of data is the primary outcome — so called because it is what the investigators decide is the most important. Secondary outcomes provide additional information to support decision-making. We were interested in knowing how important patients and healthcare professionals consider the outcomes (especially the primary outcome) measured in a selection of published trials. Methods The work had three stages: (1) We identified a body of late-stage trials in two clinical areas, breast cancer management and nephrology. (2) We identified the primary and secondary outcomes for these trials. (3) We randomly ordered these outcomes and presented them to patients and healthcare professionals (with experience of the clinical area), and we asked them to rank the importance of the outcomes. They were not told which outcomes trial authors considered primary and secondary. Results In our sample of 44 trials with 46 primary outcomes, 29 patients, one patient representative and 12 healthcare professionals together ranked the primary outcome as the most important outcome 13/46 times or 28%. Breast cancer patients and healthcare professionals considered the primary outcome to be the most important outcome for 8/21 primary outcomes chosen by trialists. For nephrology, the equivalent figure was 5/25. The primary outcome appeared in a respondent’s top 5 ranked outcomes 151/178 (85%) times for breast cancer and 225/259 (87%) times for nephrology even if the primary was not considered the most important outcome. Conclusions The primary outcome in a trial is the most important piece of data collected. It is used to determine how many participants are required, and it is the main piece of information used to judge whether the intervention is effective or not. In our study, patients and healthcare professionals agreed with the choice of the primary outcome made by trial teams doing late-stage trials in breast cancer management and nephrology 28% of the time.

show abstract

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Getting it wrong most of the time? Comparing trialists’ choice of primary outcome with what patients and health professionals want

Treweek

Miyakoda²,

Burke

et al. 2022

Trials

View full text Add to dashboard Cite

show abstract

“…Core outcome sets remain a minority choice, only 2% of all trials use them, 22 although use in rheumatoid arthritis, the area we chose, is much higher with 82% of rheumatoid arthritis trials found to use a core outcome set in a recent systematic review 23 . Increased data collection workload does not appear to be an argument against using core outcome sets.…”

Section: Discussionmentioning

confidence: 99%

A good use of time? Providing evidence for how effort is invested in primary and secondary outcome data collection in trials

Gardner

Feky

Pickles

et al. 2022

Preprint

View full text Add to dashboard Cite

BackgroundData collection is a substantial part of trial workload for participants and staff alike. How these hours of work are spent is important because stakeholders are more interested in some outcomes than others. The ORINOCO study compared the time spent collecting primary outcome data to the time spent collecting secondary outcome data in a cohort of trials.MethodsWe searched PubMed for phase III trials indexed between 2015 and 2019. From these, we randomly selected 120 trials evaluating a therapeutic intervention plus an additional random selection of 20 trials evaluating a public health intervention. We also added eligible trials from a cohort of 189 trials in rheumatology that had used the same core outcome set.We then obtained the time taken to collect primary and secondary outcomes in each trial. We used a hierarchy of methods that included data in trial reports, contacting the trial team, and approaching individuals with experience of using the identified outcome measures. We calculated the primary:secondary data collection time ratio and notional data collection cost for each included trial.ResultsWe included 161 trials (120 Phase III; 21 Core outcome set; 20 Public health), which together collected 230 primary and 688 secondary outcomes. Full primary and secondary timing data were obtained for 134 trials. The median time spent on primaries was 56 hours (range 0.0 – 10,747) and the median time spent on secondaries was 191 hours (range 0.0 – 1,356,833). The median primary:secondary data collection time ratio was 1:3.0 (i.e. for every minute spent on primary outcomes, 3.0 were spent on secondaries. The ratio varied by trial type: Phase III trials were 1: 3.1, Core outcome set 1:3.4 and Public health trials 1:2.2. The median notional overall data collection cost was £8,016 (range £53 – £31,899,141).ConclusionsDepending on trial type, between two and three times as much time is spent collecting secondary outcome data than collecting primary outcome data. Trial teams should explicitly consider how long it will take to collect the data for an outcome and decide whether that time is worth it given importance of the outcome to the trial.

show abstract

“…Standardization of critical and important outcomes is largely available in Core Outcome Set (COS), an agreed standardized set of outcomes that should be measured and reported, as a minimum, in all clinical trials in specific health areas [ 18 , 24 ]. However, the uptake of COS in both Cochrane reviews [ 23 , 25 ] and clinical trials is are not yet consistent [ 26 , 27 ].…”

Section: Introductionmentioning

confidence: 99%

Usefulness of Cochrane Reviews in Clinical Guideline Development—A Survey of 585 Recommendations

Korfitsen

Mikkelsen

Ussing

et al. 2022

IJERPH

View full text Add to dashboard Cite

The Danish Health Authority develops clinical practice guidelines to support clinical decision-making based on the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) system and prioritizes using Cochrane reviews. The objective of this study was to explore the usefulness of Cochrane reviews as a source of evidence in the development of clinical recommendations. Evidence-based recommendations in guidelines published by the Danish Health Authority between 2014 and 2021 were reviewed. For each recommendation, it was noted if and how Cochrane reviews were utilized. In total, 374 evidence-based recommendations and 211 expert consensus recommendations were published between 2014 and 2021. Of the 374 evidence-based recommendations, 106 included evidence from Cochrane reviews. In 28 recommendations, all critical and important outcomes included evidence from Cochrane reviews. In 36 recommendations, a minimum of all critical outcomes included evidence from Cochrane reviews, but not all important outcomes. In 33 recommendations, some but not all critical outcomes included evidence from Cochrane reviews. Finally, in nine recommendations, some of the important outcomes included evidence from Cochrane reviews. In almost one-third of the evidence-based recommendations, Cochrane reviews were used to inform clinical recommendations. This evaluation should inform future evaluations of Cochrane review uptake in clinical practice guidelines concerning outcomes important for clinical decision-making.

show abstract

Use of core outcome sets was low in clinical trials published in major medical journals

Cited by 45 publications

References 23 publications

Getting it wrong most of the time? Comparing trialists’ choice of primary outcome with what patients and health professionals want

Getting it wrong most of the time? Comparing trialists’ choice of primary outcome with what patients and health professionals want

A good use of time? Providing evidence for how effort is invested in primary and secondary outcome data collection in trials

Usefulness of Cochrane Reviews in Clinical Guideline Development—A Survey of 585 Recommendations

Contact Info

Product

Resources

About