Journal of Clinical Epidemiology最新文献

{"title":"When and how to establish a new reference standard for medical tests: a scoping review identifying methodological priorities.","authors":"Samuel J White, Miranda W Langendam, Bada Yang, Yasaman Vali, Yaxin Chen, Elio Arruzza, Minh Chau, Mason Crossman, Zachary Munn, Tracy Merlin, Timothy H Barker, Holger J Schünemann","doi":"10.1016/j.jclinepi.2026.112306","DOIUrl":"https://doi.org/10.1016/j.jclinepi.2026.112306","url":null,"abstract":"<p><strong>Introduction: </strong>The reference standard, defined as the best available test or test strategy for diagnosing a target disease/condition in a specific clinical population, is rarely perfectly accurate. This imperfection raises the question of when (and how) to establish a new test as the reference standard test. The objective of this scoping review was to assess methodological priorities for establishing a new reference standard for medical tests.</p><p><strong>Methods: </strong>This scoping review was performed using JBI methodology and reported in accordance with the PRISMA-ScR reporting guideline. We included sources describing methodological approaches to establishing a new reference standard across all healthcare populations and settings. Data were analysed using a combination of descriptive and qualitative content and thematic analyses.</p><p><strong>Results: </strong>We screened 5,396 studies and included 13 in our final analysis (7 methodological studies and 6 applied studies). Three main methodological themes emerged: 1) demonstrating the imperfectness of the current reference standard, 2) comparing candidate reference standard tests, and 3) decision principles for accepting a new reference standard. Amongst the six applied studies that established reference standards for specific conditions, five used composite reference standards.</p><p><strong>Conclusion: </strong>Based on the findings of our review, we define a potential workflow for structuring decision-making relating to whether to accept a new test as the reference standard. Development of formal methodological guidance for when and how to establish or replace a reference standard would likely benefit guideline developers, clinicians, technology assessors and, ultimately, patients.</p>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":" ","pages":"112306"},"PeriodicalIF":5.2,"publicationDate":"2026-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147857582","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Understanding What Works in Disseminating Clinical Practice Guidelines - A Qualitative Evidence Synthesis.","authors":"Sumanth Kumbargere Nagraj, Tandekile Lubelwana Hafver, Valerie Reinthaler, Stijn Van de Velde, Carlos Zaror, YanJiao Shen, Susan Banda, Talitha Mpando, Gertrude Kunje, Suzgika Lakudzala, Ameer Hohlfeld, Emmanuel Effa, Denny Mabetha, Thomas Agoritsas, Per Olav Vandvik, Nicolas Delvaux","doi":"10.1016/j.jclinepi.2026.112313","DOIUrl":"https://doi.org/10.1016/j.jclinepi.2026.112313","url":null,"abstract":"<p><strong>Aim of the review: </strong>To identify the barriers and facilitators that influence the dissemination of clinical practice guidelines, and to explore the perceived usefulness, practical utility, expectations and unmet needs of end-users in relation to current dissemination strategies.</p><p><strong>Methods: </strong>This qualitative evidence synthesis is a part of mixed-methods evidence synthesis. We searched literature from MEDLINE, Embase, CINAHL, Web of Science, Scopus, Epistemonikos, Agency for Healthcare Research and Quality and Medical Guidelines Clearing house on 30th January 2025. Screening, data extraction and quality appraisal of the included studies were done by at least two authors independently and in duplicate. We performed reflexive thematic analysis of the data related to barriers, facilitators, usefulness, utility and unmet needs of dissemination products and channels. We assessed the confidence of evidence following the GRADE CERQual methods.</p><p><strong>Results: </strong>We screened 31,853 titles and abstracts and found 40 eligible studies to be included in this qualitative evidence synthesis. We identified themes related to barriers and facilitators, practical utility and usefulness, unmet needs of dissemination products and unmet needs of dissemination channels. Key barriers were weak institutional support, language barriers, scepticism, low awareness and lack of training programmes. Main facilitators were related to leadership and institutional support, consumer and stakeholder involvement, training and multichannel dissemination. We identified the dissemination strategies that were perceived as useful when focused on improving clinical decision making and encouraging organisation adoption. Key aspects related to practical utility were the improved communication between healthcare professionals and patients or carers and better understandability of the guidelines when presented using simple layouts, visual features and multiple channels including social media. Unmet needs related to dissemination products included a demand for cultural and linguistic inclusivity, active promotion of guidelines, easier navigation through digital formats and training support for implementation. The key unmet need related to dissemination channels was the visibility and awareness of channels.</p><p><strong>Conclusion: </strong>This qualitative evidence synthesis identified key barriers and facilitators of dissemination strategies including unmet needs regarding the products and channels used in these strategies. The results can further inform organisations that develop and disseminate clinical practice guidelines.</p>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":" ","pages":"112313"},"PeriodicalIF":5.2,"publicationDate":"2026-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147857605","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Enhancing trial recruitment through a social media influencer campaign in the MeMaF study: a mixed methods approach.","authors":"Stefanie Lysk, Claudia M Witt, Claudia Canella, Sabine Wiegmann, Rebecca Paprott, Hannah Neumayer, Thomas Gärtner, Stefan Konigorski, Daniel Pach","doi":"10.1016/j.jclinepi.2026.112310","DOIUrl":"https://doi.org/10.1016/j.jclinepi.2026.112310","url":null,"abstract":"<p><strong>Objectives: </strong>Recruitment remains a major challenge in clinical research, particularly among young populations. This study evaluated the effectiveness of a paid social media influencer campaign as a recruitment strategy for the MeMaF study (Menstrual Health and Hybrid Care Model for All Young Females), which targets young women with severe menstrual pain in Germany.</p><p><strong>Study design and setting: </strong>This mixed methods study was nested within the MeMaF single-arm trial. Recruitment data were collected via the study app, and three recruitment periods were defined retrospectively: (1) basic recruitment period (March 8, 2024-January 8, 2025), (2) social media influencer campaign (January 9-31, 2025), and (3) post-campaign period (February 1-28, 2025). Quantitative analyses included descriptive statistics of daily enrollments, app downloads, and download-to-enrollment ratios across the three recruitment periods. Qualitative data consisted of anonymized social media comments from Instagram and TikTok related to the influencer postings. A thematic content analysis was conducted using MAXQDA, supported by large language models integrated into the software.</p><p><strong>Results: </strong>Over the entire recruitment period, 3,496 participants were enrolled, thereby meeting the recruitment goal. The mean daily enrollment rate during the social media influencer campaign (45.5 participants/day) was six times higher than during the basic recruitment period (6.8 participants/day) and remained elevated in the post-campaign period (13.4 participants/day). Posts from six female influencers accumulated 2.8 million views and 114,600 likes on Instagram, and 865,000 views and 100,000 likes on TikTok. Qualitative analysis of 501 comments revealed strong audience engagement, including discussion of the study's inclusion criteria, often expressing criticism of eligibility restrictions alongside appreciation for the study initiative. Comment sections also evolved into a space for sharing personal experiences with menstrual pain and healthcare access.</p><p><strong>Conclusion: </strong>A paid influencer campaign can substantially enhance study recruitment among young populations. Collaboration with influencers represents a feasible and effective strategy to broaden reach, strengthen engagement, and complement traditional recruitment methods. Future studies should evaluate cost-effectiveness and transferability to other populations and platforms.</p>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":" ","pages":"112310"},"PeriodicalIF":5.2,"publicationDate":"2026-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147857607","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Response to: Swieczkowski D, Kwaśny A, Cubała WJ. Regulatory context for protocol amendments and public reporting in psychedelic trials.","authors":"Marija Franka Žuljević, Antonija Mijatović, Renata Orhanović, Glenn Goasdoué, Diana Gujinović","doi":"10.1016/j.jclinepi.2026.112312","DOIUrl":"https://doi.org/10.1016/j.jclinepi.2026.112312","url":null,"abstract":"","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":" ","pages":"112312"},"PeriodicalIF":5.2,"publicationDate":"2026-05-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147845711","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Regulatory context for protocol amendments and public reporting in psychedelic trials.","authors":"Damian Swieczkowski, Aleksander Kwaśny, Wiesław Jerzy Cubała","doi":"10.1016/j.jclinepi.2026.112311","DOIUrl":"https://doi.org/10.1016/j.jclinepi.2026.112311","url":null,"abstract":"","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":" ","pages":"112311"},"PeriodicalIF":5.2,"publicationDate":"2026-05-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147845421","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Identifiability and Selection Bias in Indirect Comparisons Using Real-World Controls: A Systematic Review of Methods and Reporting.","authors":"Matylde Diouf, Jerome Lambert, Sylvie Chevret","doi":"10.1016/j.jclinepi.2026.112301","DOIUrl":"https://doi.org/10.1016/j.jclinepi.2026.112301","url":null,"abstract":"<p><strong>Objective: </strong>Recently, the integration of external controls to construct an externally controlled trial has gained attraction as a way to strengthen the level of evidence of single-arm trials. We aimed to systematically evaluate the reporting of key methodological features of indirect comparisons that use real-world controls to supplement single-arm drug trials.</p><p><strong>Study design and setting: </strong>We conducted a systematic review to critically appraise the methodological features of these designs and identify key omissions. We compared studies based on methods that address selection bias by targeting covariate balance (e.g., matching, weighting, g-computation or doubly robust estimators) (\"balancing-based methods\") with those which do not explicitly target imbalances, using outcome-regression or unadjusted analyses for group adjustment (\"other methods\").</p><p><strong>Results: </strong>Our search identified 55 single-arm drug trials published from 1987 to 2024. Of these, 29 were described as using balancing-based methods and 26 used other methods. Over half of the trials were in oncology, with hematologic-oncology being the most represented pathology. Only 29% of studies provided a rationale for using an indirect comparison of the single-arm trial to real-world data. Real-world control patients were recruited over longer and earlier time periods than patients in the single-arm trial, compromising positivity assumption. Balancing-based methods were more recent compared to other methods (median of publication time, 2023 vs 2019), with the indirect comparison often following a previous publication of the trial (66% vs 8%). In the balancing-based group, sample sizes in both the experimental (median 77 vs 31) and control (median 97 vs 40) groups were higher, as well as the number of participating centers (with monocentric studies in 30% vs 57%). Assessment of post-adjustment balance was reported more frequently in the balancing-based group, though often only based on p-values (34% vs 62%), improved for those using weighting compared to matching approaches.</p><p><strong>Conclusion: </strong>To ensure the validity of unanchored indirect comparisons with RWD controls, future studies should adopt and transparently report on robust methodological frameworks based on the principles of causal inference.</p>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":" ","pages":"112301"},"PeriodicalIF":5.2,"publicationDate":"2026-05-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147845405","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Advancing Health Equity Through Observational Research: Dissemination and Uptake of the STROBE-Equity Extension.","authors":"Omar Dewidar, Larissa Shamseer, Lucy C Barker, Ebenezer Owusu-Addo, Janice Tufte, Lawrence Mbuagbaw, Sarah Funnell, Janet Jull, Vivian Welch","doi":"10.1016/j.jclinepi.2026.112302","DOIUrl":"https://doi.org/10.1016/j.jclinepi.2026.112302","url":null,"abstract":"<p><p>Health equity is achieved when all individuals have the opportunity to attain their full health potential. The STROBE (Strengthening the Reporting of Observational Studies in Epidemiology) guidelines were developed to enhance the transparency of reporting in observational research, enabling readers to assess methodological rigor, and limitations. The STROBE-Equity extension builds on this by promoting comprehensive and transparent reporting of health equity considerations to improve the usability of observational evidence in health equity decision-making. For STROBE-Equity to achieve meaningful impact, coordinated efforts from diverse interest-holders are required. Using established frameworks and iterative discussions within the STROBE-Equity KT (Knowledge Translation) Task Force, we identified key interest-holder groups for whom the guideline is most relevant and proposed low-risk, actionable strategies to support its uptake and dissemination. Tailored KT strategies are still needed to align the implementation of the guidance with the roles, capacities and motivations of different interest-holder groups.</p>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":" ","pages":"112302"},"PeriodicalIF":5.2,"publicationDate":"2026-05-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147845363","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Results Reporting in Antimicrobial Agent Clinical Trials: An analysis of Compliance and Timeliness Under the 2017 Final FDAAA Rule.","authors":"Megan Curtin, Allisun Wiltshire, Gustav Nilsonne, Maximilian Siebert","doi":"10.1016/j.jclinepi.2026.112307","DOIUrl":"https://doi.org/10.1016/j.jclinepi.2026.112307","url":null,"abstract":"<p><strong>Objective: </strong>Antimicrobial resistance (AMR) is an urgent global health threat. Timely and complete antimicrobial agent clinical trial results reporting is essential to evaluate the safety and efficacy of investigational therapies. The Food and Drug Administration Amendments Act (FDAAA) of 2007 mandated results reporting to ClinicalTrials.gov. After nearly ten years of underreporting, the U.S. Department of Health and Human Services issued the Final Rule, requiring a designated responsible party to submit results to ClinicalTrials.gov. The Final Rule clarified that trials meeting \"applicable clinical trial\" (ACT) criteria must adhere to the federal reporting requirements. ACTs and probable ACTs (pACTs) are interventional studies regulated by the FDA with at least one site based in the United States. However, pACTs were initiated prior to January 2017, thus the Final Rule requirements do not apply to these trials. This study investigates the effectiveness of the Final Rule by analyzing compliance and timeliness of results reporting of ACTs and pACTs for antimicrobial agents.</p><p><strong>Design: </strong>We extracted data from ClinicalTrials.gov for trials involving antimicrobial agents with primary completion dates between May 1, 2013, and May 1, 2023. We analyzed the time from primary completion to results reporting and estimated the hazard ratio to compare timeliness between ACTs and pACTs. Additionally, we assessed delays in reporting across different study types and funding sources.</p><p><strong>Results: </strong>Our search resulted in 2629 NCT records. After exclusion of ineligible trials, we included 2525 trials. We found 1769 pACTs (70.1%; 95% CI, 68.2%-71.8%) and 756 ACTs (29.9%; 95% CI, 28.2%-31.8%). Among the 2525 eligible trials, 2288 trials (90.6%; 95% CI, 89.5%-91.7%) were reported on ClinicalTrials.gov and 74 out of the 237 (31.2%) missing records were found in journals. Overall, 79.4% (95% CI, 77.7%-81.0%) of trials were reported late (71.3% of ACTs vs 82.6% of pACTs). ACTs were more likely to report results earlier than pACTs, with a hazard ratio of 1.3 (95% CI, 1.2-1.4).</p><p><strong>Conclusions: </strong>ACTs demonstrated greater reporting compliance and shorter delays in the reporting of overdue results. While this analysis provides initial insights, limitations related to timeline and sample scope suggest that broader investigations are needed to fully evaluate the impact of the Final Rule.</p>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":" ","pages":"112307"},"PeriodicalIF":5.2,"publicationDate":"2026-05-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147845665","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Conditions for unbiased estimation of sensitivity and specificity when using a probabilistic reference standard could be identified.","authors":"Werner Vach","doi":"10.1016/j.jclinepi.2026.112308","DOIUrl":"https://doi.org/10.1016/j.jclinepi.2026.112308","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background and objectives: &lt;/strong&gt;If a reference standard is affected by uncertainty in some patients, it can be considered to assign a probability for the presence of the target condition in these patients. Such probabilistic reference standards have been suggested or can be imagined in various settings. This paper aims at identifying conditions for unbiased estimation of sensitivity and specificity when a probabilistic reference standard is used in a diagnostic accuracy study.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;The conditional distribution of the index test given the probabilistic reference standard carries the information on sensitivity and specificity. An explicit expression for this distribution is derived. It includes three different model components reflecting a potential association of sensitivity with the probabilistic reference standard, a potential association of specificity with the probabilistic reference standard, and a potential mean miscalibration of the probabilistic reference standard. Simple parametrizations of the three different model components are suggested. The dependence of the conditional distribution on the parameter values is investigated, and the bias of model-based and model-free estimates is investigated in a simulation study.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Due to identifiability issues, it can only be expected to be able to include one of the three components in a model-based estimation. If absence of the other two components can be assumed, model-based estimation allows estimates with negligible bias. Model-free estimates show a high risk of bias.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusion: &lt;/strong&gt;The use of probabilistic reference standards in diagnostic accuracy studies is challenging. Unbiased estimation of sensitivity and specificity can only be expected if two out of the following three conditions can be ruled out: 1) Association of sensitivity with the probabilistic reference standard. 2) Association of specificity with the probabilistic reference standard. 3) Insufficient mean calibration of the probabilistic reference standard. If this is the case, adequate statistical methods allow estimation with negligible bias. Arguing for absence of two conditions requires corresponding reasoning.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Plain language summary: &lt;/strong&gt;Evaluating the accuracy of a diagnostic test requires a systematic comparison of the test results with the true status in a series of patients. Determining the true status by a so-called reference test can be challenging in some patients. It has been suggested that the reference test should then result in a probability instead of a definite decision about the true status. It is unknown how to make use of such a probabilistic reference standard in analysing the diagnostic accuracy of the index test - i.e. the test of interest. This paper investigates how to estimate two key accuracy parameters - sensitivity and specificity - based on a probabilistic reference standard. Three condition","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":" ","pages":"112308"},"PeriodicalIF":5.2,"publicationDate":"2026-05-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147845413","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Editors' Choice: May 2026.","authors":"Andrea C Tricco, David Tovey","doi":"10.1016/j.jclinepi.2026.112277","DOIUrl":"https://doi.org/10.1016/j.jclinepi.2026.112277","url":null,"abstract":"","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"193 ","pages":"112277"},"PeriodicalIF":5.2,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147845722","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}