Choosing Important Health Outcomes for Comparative Effectiveness Research: A Systematic Review

Abstract

BackgroundA core outcome set (COS) is a standardised set of outcomes which should be measured and reported, as a minimum, in all effectiveness trials for a specific health area. This will allow results of studies to be compared, contrasted and combined as appropriate, as well as ensuring that all trials contribute usable information. The COMET (Core Outcome Measures for Effectiveness Trials) Initiative aims to support the development, reporting and adoption of COS. Central to this is a publically accessible online resource, populated with all available COS. The aim of the review we report here was to identify studies that sought to determine which outcomes or domains to measure in all clinical trials in a specific condition and to describe the methodological techniques used in these studies.MethodsWe developed a multi-faceted search strategy for electronic databases (MEDLINE, SCOPUS, and Cochrane Methodology Register). We included studies that sought to determine which outcomes/domains to measure in all clinical trials in a specific condition.ResultsA total of 250 reports relating to 198 studies were judged eligible for inclusion in the review. Studies covered various areas of health, most commonly cancer, rheumatology, neurology, heart and circulation, and dentistry and oral health. A variety of methods have been used to develop COS, including semi-structured discussion, unstructured group discussion, the Delphi Technique, Consensus Development Conference, surveys and Nominal Group Technique. The most common groups involved were clinical experts and non-clinical research experts. Thirty-one (16%) studies reported that the public had been involved in the process. The geographic locations of participants were predominantly North America (n = 164; 83%) and Europe (n = 150; 76%).ConclusionsThis systematic review identified many health areas where a COS has been developed, but also highlights important gaps. It is a further step towards a comprehensive, up-to-date database of COS. In addition, it shows the need for methodological guidance, including how to engage key stakeholder groups, particularly members of the public.

Perspectives of survivors, families and researchers on key outcomes for research in acute respiratory failure

“…Moreover, findings of this study emphasise the importance of patient and family input to decision making regarding core outcome domains, which has been under-represented in prior projects aimed at creating core outcome sets 44. Incorporating patient perspectives is important to ensure that the outcomes measured by researchers are patient centred 45 46.…”

Core Outcome Sets

“…These include primary qualitative research—in-depth interviews or focus groups15—undertaken for the purpose of the Core Outcome Set, and systematic reviews of published qualitative research 16. Such approaches are required because clinical trials have been repeatedly shown to miss outcomes considered important by these other stakeholder groups 17 18…”

“…Different techniques have been used to reach consensus regarding which outcomes are ‘core.’ These range from relatively informal methods, such as semistructured group discussion at a workshop or meeting, to more formal group work, such as use of the Nominal Group technique 15. Currently the most common method used is modified Delphi technique13 ; this allows the views of a large number of geographically diverse stakeholders to be integrated quickly and efficiently.…”

The impact of treatment on quality of life of patients with head and neck cancer and its association with prognosis

“…1e3 At present, there is a growing interest for the inclusion of HRQL assessment in clinical trials in Oncology, allowing for comparison, contrast, or combining as appropriate, in order to ensure the production of high-quality evidence. 3 Recent reports have evaluated the association of HRQL and prognosis in cancer, 4,5 and specifically in Head and Neck (H&N) squamous cell carcinoma. 6,7 However, there is scarce information that explores improvement in HRQL as a result of adequate treatments in the prognosis of H&N cancers, although this interesting association has been reported in other neoplasms.…”