Using qualitative methods to inform the trade-off between content validity and consistency in utility assessment: the example of type 2 diabetes and Alzheimer's Disease

<p>Abstract</p> <p>Background</p> <p>Key stakeholders regard generic utility instruments as suitable tools to inform health technology assessment decision-making regarding allocation of resources across competing interventions. These instruments require a 'descriptor', a 'valuation' and a 'perspective' of the economic evaluation. There are various approaches that can be taken for each of these, offering a potential lack of consistency between instruments (a basic requirement for comparisons across diseases). The 'reference method' has been proposed as a way to address the limitations of the Quality-Adjusted Life Year (QALY). However, the degree to which generic measures can assess patients' specific experiences with their disease would remain unresolved. This has been neglected in the discussions on methods development and its impact on the QALY values obtained and resulting cost per QALY estimate underestimated. This study explored the content of utility instruments relevant to type 2 diabetes and Alzheimer's disease (AD) as examples, and the role of qualitative research in informing the trade-off between content coverage and consistency.</p> <p>Method</p> <p>A literature review was performed to identify qualitative and quantitative studies regarding patients' experiences with type 2 diabetes or AD, and associated treatments. Conceptual models for each indication were developed. Generic- and disease-specific instruments were mapped to the conceptual models.</p> <p>Results</p> <p>Findings showed that published descriptions of relevant concepts important to patients with type 2 diabetes or AD are available for consideration in deciding on the most comprehensive approach to utility assessment. While the 15-dimensional health related quality of life measure (15D) seemed the most comprehensive measure for both diseases, the Health Utilities Index 3 (HUI 3) seemed to have the least coverage for type 2 diabetes and the EuroQol-5 Dimensions (EQ-5D) for AD. Furthermore, some of the utility instruments contained items that could not be mapped onto either of the proposed conceptual models.</p> <p>Conclusions</p> <p>Content of the utility measure has a significant impact on the treatment effects that can be observed. This varies from one disease to the next and as such contributes to lack of consistency in observable utility effects and incremental utility scores. This observation appears to have been omitted from the method development considerations such as reference methods. As a result, we recommend that patients' perspectives obtained via qualitative methods are taken into consideration in the ongoing methods development in health state descriptions for generic utility instruments. Also, as a more immediate contribution to improving decision making, we propose that a content map of the chosen utility measure with patient-reported domains be provided as standard reporting in utility measurement in order to improve the transparency of the trade-offs in relation to patient relevance and consistency.</p

Towards core outcome set (COS) development: a follow-up descriptive survey of outcomes in Cochrane reviews

BACKGROUND: A core outcome set (COS) can address problems of outcome heterogeneity and outcome reporting bias in trials and systematic reviews, including Cochrane reviews, helping to reduce waste. One of the aims of the international Core Outcome Measures in Effectiveness Trials (COMET) Initiative is to link the development and use of COS with the outcomes specified and reported in Cochrane reviews, including the outcomes listed in the summary of findings (SoF) tables. As part of this work, an earlier exploratory survey of the outcomes of newly published 2007 and 2011 Cochrane reviews was performed. This survey examined the use of COS, the variety of specified outcomes, and outcome reporting in Cochrane reviews by Cochrane Review Group (CRG). To examine changes over time and to explore outcomes that were repeatedly specified over time in Cochrane reviews by CRG, we conducted a follow-up survey of outcomes in 2013 Cochrane reviews. METHODS: A descriptive survey of outcomes in Cochrane reviews that were first published in 2013. Outcomes specified in the methods sections and reported in the results section of the Cochrane reviews were examined by CRG. We also explored the uptake of SoF tables, the number of outcomes included in these, and the quality of the evidence for the outcomes. RESULTS: Across the 50 CRGs, 375 Cochrane reviews that included at least one study specified a total of 3142 outcomes. Of these outcomes, 32 % (1008) were not reported in the results section of these reviews. For 23 % (233) of these non-reported outcomes, we did not find any reason in the text of the review for this non-report. Fifty-seven percent (216/375) of reviews included a SoF table. CONCLUSIONS: The proportion of specified outcomes that were reported in Cochrane reviews had increased in 2013 (68 %) compared to 2007 (61 %) and 2011 (65 %). Importantly, 2013 Cochrane reviews that did not report specified outcomes were twice as likely to provide an explanation for why the outcome was not reported. There has been an increased uptake of SoF tables in Cochrane reviews. Outcomes that were repeatedly specified in Cochrane reviews by CRG in 2007, 2011, and 2013 may assist COS development. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (doi:10.1186/s13643-015-0060-0) contains supplementary material, which is available to authorized users

Choosing Important Health Outcomes for Comparative Effectiveness Research: An Updated Review and Identification of Gaps

<div><p>Background</p><p>The COMET (Core Outcome Measures in Effectiveness Trials) Initiative promotes the development and application of core outcome sets (COS), including relevant studies in an online database. In order to keep the database current, an annual search of the literature is undertaken. This study aimed to update a previous systematic review, in order to identify any further studies where a COS has been developed. Furthermore, no prioritization for COS development has previously been undertaken, therefore this study also aimed to identify COS relevant to the world’s most prevalent health conditions.</p><p>Methods</p><p>The methods used in this updated review followed the same approach used in the original review and the previous update. A survey was also sent to the corresponding authors of COS identified for inclusion in this review, to ascertain what lessons they had learnt from developing their COS. Additionally, the COMET database was searched to identify COS that might be relevant to the conditions with the highest global prevalence.</p><p>Results</p><p>Twenty-five reports relating to 22 new studies were eligible for inclusion in the review. Further improvements were identified in relation to the description of the scope of the COS, use of the Delphi technique, and the inclusion of patient participants within the development process. Additionally, 33 published and ongoing COS were identified for 13 of the world’s most prevalent conditions.</p><p>Conclusion</p><p>The development of a reporting guideline and minimum standards should contribute towards future improvements in development and reporting of COS. This study has also described a first approach to identifying gaps in existing COS, and to priority setting in this area. Important gaps have been identified, on the basis of global burden of disease, and the development and application of COS in these areas should be considered a priority.</p></div

Parodie et carnavalisation : l’exemple de Hubert Aquin

One of the defining features of patient-centered outcomes research (PCOR) is the emphasis on reporting outcomes that are meaningful to patients. Accelerating progress toward this objective could be achieved through increased development and uptake of core outcome sets (COS), which are intended to represent a standardized minimum set of outcomes that should bemeasured and reported in all clinical trials in a specific condition. The level of activity around COS has increased significantly over recent years, however there are many important clinical conditions for which high quality COS havenot been developed. We believe that meaningful progress toward the goals behind the significant investments in PCOR will depend on sustained attention to the challenges of COS development and uptake

A systematic review of studies reporting the development of Core Outcome Sets for use in routine care.

ObjectivesCore outcome sets (COS) represent the minimum health outcomes to be measured for a given health condition. Interest is growing in using COS within routine care to support delivery of patient-focussed care. This review aims to systematically map COS developed for routine care to understand their scope, stakeholder involvement and development methods.MethodsMedline (OVID), Scopus and Web of Science Core collection were searched for studies reporting development of COS for routine care. Data on scope, methods and stakeholder groups were analysed in subgroups defined by setting.ResultsScreening 25,301 records identified 262 COS: 164 for routine care only; 98 for routine care and research. Nearly half of the COS (112/254, 44%) were developed with patients, alongside input from experts in: registries; insurance; legal; outcomes measurement; performance management. Research publications were often searched to generate an initial list of outcomes (115/198, 58%) with few searching routine health records (47/198, 24%).ConclusionAn increasing number of COS are being developed for routine care. Although involvement of patient stakeholders has increased in recent years further improvements are needed. Methodology and scope are broadly similar to COS for research but implementation of the final set is a greater consideration during development

Core Outcome Set-STAndards for Reporting The COS-STAR Statement

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

Pain measurement in rheumatic and musculoskeletal diseases: where to go from here? Report from a Special Interest Group at OMERACT 2018

Objective: Establishing a research agenda on standardizing pain measurement in clinical trials in rheumatic and musculoskeletal diseases (RMDs). Methods: Discussion during a meeting at 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 RMDs? how to account for pain mechanistic descriptors (e.g. central sensitization) in pain measurement? Conclusion: Characterizing and assessing the spectrum of pain experience across RMDs in a standardized fashion is the future objective of the OMERACT pain working group