Journal of Clinical Epidemiology最新文献

Objectives: To review and synthesise available evidence on carbon emissions associated with clinical trials to inform future research on design and delivery of greener trials.

Study design and setting: We performed a scoping review by following the Joanna Briggs Institute (JBI) guidance and the Preferred Reporting Items for Systematic Reviews and Meta-Analyses Extension for Scoping Reviews (PRISMA-ScR). A systematic search was conducted on MEDLINE (Ovid) from 1 January 2007 to 15 April 2024 with no geographic and language restrictions complemented by forward and backward citation analysis (snowballing). We included all types of research literature within the context of clinical trials reporting any aspect related to trial specific carbon emissions.

Results: Twenty-two articles were identified as eligible and included in the review. Most included studies (n=17, 77%) were published between 2020 and 2024. Over half of the included studies (n=13, 59%) were primary research articles with the majority reporting carbon audits of trials and their associated processes. The remaining literature comprised secondary studies (n=3, 14%) and opinion pieces (n=6, 27%). Diverse and evolving approaches to studying trial-related carbon emissions were identified alongside several carbon hotspots including those associated with trial-related travel, trial facilities, and sample lifecycle.

Conclusion: The literature on carbon emissions associated with clinical trials has focused on studies reporting carbon audits of trials and their associated processes. Efforts have been made to quantify the trial carbon output with variability in methods and carbon output. Despite the development and evolution of carbon measurement tools, strategies to mitigate trial specific carbon emissions are still much in need.

Background and objective: Multi-centre randomised controlled trials (RCTs) provide vital information about healthcare interventions. Reporting on country-level participation is important for understanding the context of multi-centre RCTs. This study aimed to examine multi-centre RCT reporting of country-level participation, using Ireland as a case study.

Study design and setting: This is meta-research study included RCTs identified in a previous study of Irish RCTs. The previous study involved searching six databases (inception-2018) for RCTs with participants recruited in Ireland: PubMed, Embase, Scopus, CINAHL, PsychINFO and the Cochrane Register of Controlled Trials. This current study focuses on multi-centre RCTs conducted on humans in healthcare settings with <80% of participants recruited in Ireland. Outcome variables were trial characteristics and reporting rates for several variables, including: number of Irish centres, number of participants recruited in Ireland, and reporting use of relevant reporting guidelines. Descriptive statistics were used for analysis.

Results: Overall, 239 RCTs were included. The most common intervention was a drug (74.9% of RCTs). The most common clinical domain was the cardiovascular system (18.0%). Number of Irish centres was reported in 75.3% of RCTs, and number of participants recruited in Ireland in 27.2%. Among RCTs published after the CONSORT reporting guideline was published, 8.3% reported using a relevant reporting guideline.

Conclusion: Our findings show deficits in reporting for multi-centre RCTs, particularly in reporting number of participants in Ireland and reporting use of relevant reporting guidelines. The development of a multi-centre trial extension to existing reporting guidelines may partly address country-level reporting issues.

Objective: Genetic and genomic tests are the cornerstone of personalized preventive approaches. Inconsistency in evaluating their clinical utility is often cited as a reason for their limited implementation in clinical practice, Previous reviews have primarily focused on theoretical frameworks used for clinical utility evaluations of genetic tests, rather than actual assessments, and examined dimensions, rather than specific indicators within these dimensions. We aimed to review the dimensions and the specific indicators measured in published assessment reports of genetic or genomic tests.

Design and setting: We conducted a scoping review of assessment reports of genetic and genomic tests used for prevention, searching through PubMed, Web of Science, Scopus, the websites of 20 different organizations, Google, and Google Scholar. From the included assessments, we extracted the reported indicators of clinical utility, compiling a list of disease-specific indicators that detailed their numerator, denominator, and calculation methods. We analyzed the extracted indicators by stratifying them according to ten comprehensive dimensions of clinical utility, the assessment framework used, and the type of indicator (categorized as quantitative, qualitative, reference, no evidence reported). From these indicators, we then distilled a list of general indicators.

Results: We reviewed 3054 unique references and 12000 results from grey literature searches, ultimately selecting 57 assessment reports. The reference frameworks used were HTA (42%), EGAPP (25%), ACCE (21%), and others (12%). We identified 951 disease-specific indicators. The dimensions most frequently evaluated (i.e., had at least one indicator) were analytic validity (60%), clinical validity (79%), clinical efficacy (79%), and economic impact (58%). Only 12 assessments compared health outcomes between tested and untested groups, and fewer than 15% of the assessments addressed equity, acceptability, legitimacy, and personal value.

Conclusions: Our study illustrates that, although dimensions such as equity and acceptability, are significantly emphasized in traditional evaluation frameworks, these are often not considered in the assessments. Additionally, our study has underscored a significant dearth of reported primary evidence concerning the clinical efficacy of these tests.

Objective: To review current methodological guidance for handling and reporting of multiple outcomes (MOCs) in randomised controlled trials (RCTs).

Study design and setting: A scoping review with bibliographic database searches including Embase, PubMed, and Web of Science up to 22 July 2022 was conducted. Inclusion criteria were articles that: (1) provide advice on design, analysis, or reporting of RCTs using MOCs; and/or (2) discuss statistical approaches for handling MOCs in RCTs. Six specific websites were also checked for formal and reporting guidelines. Included articles were summarised using thematic analysis.

Results: Searches retrieved 1716 articles of which 123 were included. Eight additional articles were identified by the specific websites search. Six main subthemes on methodological recommendations for using MOCs were identified from 74 of 123 articles (60%): (1) need to pre-specify outcomes and analysis, (2) multiplicity adjustment, (3) power and sample size implications, (4) secondary outcomes multiplicity, (5) considerations of MOCs correlation, and (6) specific applications of MOCs. Recommendations on co-primary and composite outcomes were also identified, including their features, analyses methods, reporting, and challenges. Statistical methods for analysing MOCs were discussed in 53 of 123 articles (43%), with the majority describing modifications of pre-existing statistical approaches.

Conclusion: Current recommendations on using MOCs in RCTs focus primarily on statistical considerations and trials of licensing drugs or medical devices. Areas for further methodological research and guidance include reporting of the rationale for the use and selection of MOCs in RCTs and considerations for trials undertaken in non-regulatory setting, including complex interventions.

Objectives: Better engagement of diverse groups of interest-holders in the development of health guidelines has been proposed to improve their usefulness, implementability, and acceptability. Guidelines shape clinical- or public-health practice decision-making. Trustworthy guidelines are systematically developed documents that include actionable statements based on evidence and a formal, structured and transparent decision process. This paper describes the GIN-McMaster GDC Extension for Engagement to assist developers with engaging multiple interest-holders throughout all topics of guideline development.

Study design and setting: To produce this checklist extension, we conducted a three-phase mixed methods study. First, we utilized 10 groups of interest-holders to be engaged in health guideline development: patients, the public, providers, program managers, principal investigators, payers/purchasers of health services, payers/funders of health research, policymakers, peer-reviewed journal editors, and product makers identified in previous work and recruited co-leads to represent these groups (n=26 total).

Results: We conducted a series of reviews to identify existing methods and barriers/facilitators for engagement, approaches to managing conflicts of interest, and describing the impacts of engagement on the guideline development process. The results of these reviews informed the development of an online survey for which we received 195 responses. We clarified these results through 43 key informant interviews with interest-holders. The final GDC extension checklist was determined based on consensus methods with our co-leads.

Conclusion: This paper presents the GIN-McMaster GDC Extension for Engagement. This checklist provides interest-holder-informed recommendations for providing advice/feedback or participating in decision-making in guideline development.

Background: The guideline for the content of Statistical Analysis Plans (SAPs) outlines recommendations for items to be included in statistical analysis plans. As yet there is no specific tailoring of this guideline for Cluster Randomized Trials (CRTs). There has also been no assessment of reporting quality of SAPs against this guideline.

Objectives: Our intention is to identify how well a sample of SAPs for CRTs are adhering to the reporting of key items in the current guidelines, as well as additional analysis aspects considered to be important in CRTs.

Methods: We include (i) fully published standalone SAPs identified via Ovid-MEDLINE and (ii) SAPs published as supplementary material or appendices to the final published report identified by searching an existing database of nearly 800 CRTs.

Results: The search identified 85 unique SAPs: 26 were published in standalone format and 59 were supplementary material to the full trial report. There was mixed clarity in reporting of items related to the current guideline (e.g., most (61/85, 72%) reported what covariates will be included in any adjustment; but fewer (26/85, 31%) reported what method will be used to estimate the absolute measure of effect). Considering additional aspects important for CRTs the majority (79/85, 93%) included a plan to allow for clustering in the analysis; but fewer (10/40, 25%) reported how a small number of clusters would be accommodated (this was only considered relevant for the subset of CRTs with fewer than 40 clusters). Few (5/85, 6%) reported how the intra-cluster correlation would be estimated. Few clearly reported statistical targets of inference: in only two SAPs (2/85, 2%) was it clear whether the objectives related to the individual or cluster-level average; in trials where relevant, only three (3/70, 4%) clearly reported whether the objectives related to the marginal or cluster-specific effect.

Conclusions: This review has identified specific areas of poor quality of reporting that might need additional consideration when developing the guidance for the reporting of SAPs for CRTs.