Clinical Trials最新文献

{"title":"Sample size estimation for the averted events ratio.","authors":"David T Dunn, Oliver T Stirrup, David V Glidden","doi":"10.1177/17407745251377435","DOIUrl":"10.1177/17407745251377435","url":null,"abstract":"<p><strong>Background: </strong>The averted events ratio (AER) is a recently developed estimand for non-inferiority active-control prevention trials with a time-to-event outcome. In contrast to the traditional rate ratio or rate difference, the AER is based on the number of events <i>averted</i> by each of the two treatments rather than the observed events. The AER requires an assumption about either the background event rate (the counterfactual placebo incidence) or the counterfactual effectiveness of the control treatment. We develop and present sample size formulae for trials in which the AER is defined as the primary estimand, and draw comparisons with the conventional 95-95 method based on the rate ratio.</p><p><strong>Methods: </strong>We express sample size in terms of the expected number of events and required person-years follow-up in the control and experimental arms. Sample size formulae were based on Wald confidence intervals on a logarithmic scale, assuming the active and control treatments to be equally effective. Using the AER, sample size depends on whether the analysis will be based on the counterfactual placebo incidence or the counterfactual treatment effectiveness. For both approaches, and the 95-95 method, sample size is a function of the background event rate, the effectiveness of the control treatment, the preservation-of-effect size (non-inferiority margin), the confidence limit for inferring non-inferiority, and the desired statistical power to demonstrate non-inferiority.</p><p><strong>Results: </strong>The smallest sample size is obtained using the AER based on the counterfactual placebo incidence. The advantage is greater the higher the value of the control treatment effectiveness. For example, compared with the 95-95 method, it allows between a 2.6-fold and 4.0-fold reduction in sample size for 50% treatment effectiveness (depending of the non-inferiority margin), and between a 7.7-fold and 11.9-fold reduction for 80% treatment effectiveness. The AER based on the control treatment effectiveness is less efficient but still requires smaller sample sizes than the 95-95 method: between a 1.5-fold and 2.9-fold reduction for 50% treatment effectiveness, and between a 2.3-fold and 6.4-fold reduction for 80% treatment effectiveness. Sample size is highly sensitive to the non-inferiority margin: increasing the preservation-of-effect size from 50% to 60% implies a 1.84-fold increase in the sample size; from 60% to 70%, an increase of 2.15-fold; and from 70% to 80%, an increase of 2.55-fold.</p><p><strong>Conclusion: </strong>As well as having important advantages of interpretation, using the AER as the primary estimand in active-control non-inferiority trials permits smaller and more cost-effective studies. Ideally, the AER should be derived via the counterfactual placebo incidence when this is practicable.</p>","PeriodicalId":10685,"journal":{"name":"Clinical Trials","volume":" ","pages":"5-12"},"PeriodicalIF":2.4,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12614357/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145344025","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Mild-to-moderate depressive symptoms impact on self-reported outcome measures in clinical trials for neurodegenerative diseases.","authors":"Christine Girges, Nirosen Vijiaratnam, Alexa King, Grace Auld, Rachel McComish, Kashfia Chowdhury, Gareth Ambler, Kate Maclagan, Patricia Limousin, Dilan Athauda, Huw R Morris, Vincenzo Libri, Thomas Foltynie","doi":"10.1177/17407745251387571","DOIUrl":"10.1177/17407745251387571","url":null,"abstract":"<p><strong>Introduction: </strong>Depression can be an intrinsic part of neurodegeneration, or a reaction to the onset of motor or non-motor disability. Depression can also adversely influence an individual's perception of their disease, independently of its severity or impact on function. Participant-reported outcome measures are recognised as an important adjunct to objective clinical trial data. Although severe depression is a frequent contraindication for trial participation, mild-to-moderate depressive symptoms could potentially influence the outcome of such questionnaires. We aimed to explore this within two interventional trials for Parkinson's disease (Exenatide PD3; NCT04232969) and multiple system atrophy (Exenatide MSA; NCT04431713).</p><p><strong>Methods: </strong>Prior to investigational drug exposure, participants completed either the Patient Health Questionnaire or Beck Depression Inventory-II, which allowed us to dichotomise them into two groups (normal or mild-to-moderately elevated burden of depressive symptoms). Participants with Parkinson's self-completed the Movement Disorder Society Sponsored Revision of the Unified Parkinson's disease Rating Scale Parts 1b and 2, Parkinson's Disease Questionnaire-39, Non-motor Symptoms Scale and EQ-5D-5L, while participants with multiple system atrophy self-completed a Quality of Life scale and had assistance with completing the Unified Multiple System Atrophy Rating Scale Part 1. The Movement Disorder Society Sponsored Revision of the Unified Parkinson's Disease Rating Scale Part 3 and Unified Multiple System Atrophy Rating Scale Part 2 provided objective, clinician-rated measures of motor severity.</p><p><strong>Results: </strong>A mild-to-moderately elevated burden of depressive symptoms was identified in 32.5% (63/194) and 42.0% (21/50) of Parkinson's and multiple system atrophy participants, respectively. Despite the normal and elevated groups being comparable in terms of disease duration and objective motor severity, those with a mild-to-moderately elevated burden of depressive symptoms self-reported worse disease impact. However, measures which involved clinician and/or carer input (Unified Multiple System Atrophy Rating Scale Part 1) were not influenced by co-existing depressive symptoms.</p><p><strong>Conclusions: </strong>Mild-to-moderate depressive symptoms, below the threshold for diagnosing a depressive disorder, are associated with negative self-reporting, and such findings should be carefully considered when planning the design and analysis of trials in neurodegenerative diseases.</p>","PeriodicalId":10685,"journal":{"name":"Clinical Trials","volume":" ","pages":"54-64"},"PeriodicalIF":2.2,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12909607/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145647471","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Patient notification about pragmatic clinical trials conducted with a waiver of consent: A qualitative study.","authors":"Stephanie R Morain, Abigail Brickler, Matthew W Semler, Jonathan D Casey","doi":"10.1177/17407745251377730","DOIUrl":"10.1177/17407745251377730","url":null,"abstract":"<p><strong>Background: </strong>Some scholars have proposed that investigators and health systems should notify patients about their enrollment in pragmatic clinical trials conducted with a waiver of consent. However, others argue that decision-making about notification requires judgment, and reports suggest considerable heterogeneity about whether, when, and how individuals enrolled in pragmatic clinical trials with a waiver of consent are notified about that enrollment. Empirical data can inform this decision-making.</p><p><strong>Methods: </strong>We conducted semi-structured interviews with knowledgeable stakeholders involved in conducting and/or overseeing pragmatic clinical trials conducted with waivers of consent, including investigators, those charged with the oversight of human subjects research, and operational leadership. Interviews were conducted via video conference from September to December 2024 and were audio-recorded and professionally transcribed. Data were qualitatively analyzed using an integrated approach, including both a priori codes drawn from the interview guide and emergent, inductive codes.</p><p><strong>Results: </strong>Twenty-three of 28 experts invited to participate completed interviews. Respondents described rationales both for and against notification. Rationales for notification included both appeals to moral values (respect for persons, respect for autonomy, and transparency), as well as instrumental goals (promoting understanding of and/or support for research, avoiding downstream surprise, and supporting buy-in). Rationales against notification included preserving scientific validity, perceiving notification to lack value, and concerns that notification might be burdensome for patient-subjects or undermine trust and/or clinical or public health goals. Decision-making about notification was context-specific and reflected features related to the study design, the health system setting, the patient population, the clinical condition, and the intervention(s) being evaluated. While some factors were consistently described as weighing against notification, including scientific validity or decisions for which a patient would not be offered a choice outside the research context, other factors resulted in divergent decisions across different pragmatic clinical trials (or even across different sites for the same trial).</p><p><strong>Conclusions: </strong>While several rationales support notification about enrollment in pragmatic clinical trials conducted with waivers of consent, the relative value and practicability of notification is context-dependent. Some features, such as the need to preserve scientific validity, may appropriately weigh in favor of forgoing notification. However, evidence of divergent decision-making for similar trials suggests the need for a framework to guide future notification decisions. These data can be an important input to inform future framework development.</p>","PeriodicalId":10685,"journal":{"name":"Clinical Trials","volume":" ","pages":"65-74"},"PeriodicalIF":2.2,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12614351/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145298955","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Electronic patient-reported adverse event monitoring in academic early-phase clinical trials: A feasibility study.","authors":"Leanne Shearsmith, Sarah Danson, Sarah Gelcich, Andrea Gibson, Kathryn Gordon, Fiona Collinson, Julie Croft, Emma Griffiths, Zoe Rogers, Robert Carter, Julia Brown, Galina Velikova, Fiona Kennedy","doi":"10.1177/17407745251378668","DOIUrl":"10.1177/17407745251378668","url":null,"abstract":"<p><strong>Background: </strong>Adverse event monitoring is essential to monitor safety for oncology patients on early-phase clinical trials. Previous research considers that electronic patient-reported adverse events reporting is feasible and complementary to traditional clinician-led recording. An electronic patient-reported adverse event system was developed to explore the feasibility of this in early trials patients.</p><p><strong>Methods: </strong>A prospective single-arm feasibility study was undertaken at two recruiting hospitals. Participants were adult oncology patients who had recently (<1 month) started receiving a novel anticancer treatment on an academic early-phase trial and had access to the Internet. For a 12-week period, weekly reminders were sent to participants to complete an electronic patient-reported adverse event questionnaire remotely covering symptoms identified as relevant to the recruiting trials. The primary outcome was compliance (proportion of completed questionnaires/expected completions). Secondary outcomes included recruitment rates, attrition, electronic patient-reported outcome versus clinician-recorded adverse events, number of notifications, issues recorded, and patient acceptability.</p><p><strong>Results: </strong>Twenty-three participants consented (76.7% consent rate), 18 remained on study at 12 weeks (4 were withdrawn due to toxicity and 1 patient choice). Compliance with weekly electronic patient-reported adverse event was high, with a cumulative of 85.1% across the 12 weeks. Comparison with clinician-recorded adverse events showed electronic patient-reported adverse event resulted in wider coverage of adverse events: three times as many symptoms reported on electronic patient-reported adverse event (n = 174 last assessment) than recorded in the medical charts (n = 50 last record). End-of-study feedback indicated most patients reflected positively on their time on the study.</p><p><strong>Conclusions: </strong>Remote electronic patient-reported adverse event reporting by patients in early-phase trials is feasible and acceptable. The study highlights some logistical challenges that require consideration in future electronic patient-reported outcome work to ensure adverse events are fully captured and recorded.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov ID: NCT03461939 (first registered: 05/03/2018).</p>","PeriodicalId":10685,"journal":{"name":"Clinical Trials","volume":" ","pages":"113-120"},"PeriodicalIF":2.4,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12909613/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145372430","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Salvaging information from paused or stopped clinical studies.","authors":"Davey Smith, Thomas Fleming, Sara Gianella, Elizabeth Halloran, Sharon Hillier, Ira Longini, Laura Smeaton, Victor DeGruttola","doi":"10.1177/17407745251353429","DOIUrl":"10.1177/17407745251353429","url":null,"abstract":"","PeriodicalId":10685,"journal":{"name":"Clinical Trials","volume":" ","pages":"761-762"},"PeriodicalIF":2.4,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12331140/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144616591","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Incorporating data from multiple ongoing trials for Bayesian two-stage phase II single-arm studies.","authors":"Susan Halabi, Taehwa Choi, Elizabeth Garrett-Mayer, Richard L Schilsky, Lorenzo Trippa","doi":"10.1177/17407745251358233","DOIUrl":"10.1177/17407745251358233","url":null,"abstract":"<p><strong>Background/aim: </strong>Basket designs have been utilized in recent oncology clinical trials due to an increased interest in precision medicine. One current successful basket trial is the American Society for Clinical Oncology Targeted Agent and Profiling Utilization Registry (TAPUR) study, a pragmatic phase II trial where patients are matched based on their tumor genomic profile to treatments that target specific genomic alterations. Despite its success, recruiting patients with rare genomic alterations remains challenging. This study aims to introduce and evaluate a Bayesian approach for integrating data from ongoing independent basket trials that share similar primary aims to improve interim decisions and final analyses and reduce necessary to evaluate treatments.</p><p><strong>Methods: </strong>We introduce a Bayesian two-stage phase II single-arm trial specifically for rare cancers utilizing a hierarchical Bayesian random effects model that incorporate data from ongoing trials. We compare this approach with the standard Simon two-stage design through extensive numerical simulations and apply it to real-world scenarios.</p><p><strong>Results: </strong>Simulation results demonstrate that in rare populations our Bayesian approach has attractive operating characteristics. The simulations show that our approach performs well across a broad set of scenarios with fixed and variable numbers of trials.</p><p><strong>Conclusion: </strong>Our proposed Bayesian two-stage approach effectively integrates data from multiple ongoing basket trials, enhancing the ability to recruit and analyze patients with rare genomic alterations. This approach improves the timing of interim decision-making and final analysis, making it a valuable tool for trials with slow accrual rates.</p>","PeriodicalId":10685,"journal":{"name":"Clinical Trials","volume":" ","pages":"667-675"},"PeriodicalIF":2.2,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12373003/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144945745","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Automated data collection from an electronic medical record for a prospective real-world study in patients with retinal disease (VOYAGER).","authors":"Clare Bailey, Ian Pearce, Christiana Dinah, Melanie Dodds, Laia Vidal-Brime, Adam Wilson, Juliet Ellis, Jason Hall, Richard Pohler, Beijue Shi, Dimitar Toshev, Robyn Guymer","doi":"10.1177/17407745251358235","DOIUrl":"10.1177/17407745251358235","url":null,"abstract":"&lt;p&gt;&lt;p&gt;Background/AimsVOYAGER is a prospective, real-world study of treatment patterns and outcomes in retinal diseases. Data collection often requires double entry of routinely captured clinical data, into both site electronic medical records and VOYAGER electronic Case Report Forms (eCRFs), posing a significant time and resource burden and risk of transcription errors. To overcome these challenges, an electronic medical record-to-electronic data capture solution (EMR-to-EDC) was implemented to automate the direct transfer of electronic medical record data into the VOYAGER electronic data capture. This pilot study aimed to establish whether EMR-to-EDC could reduce data entry burden and improve data accuracy.MethodsEMR-to-EDC automatically retrieved study-specific data variables from patients in the mediSIGHT EMR (Medisoft) to pre-populate corresponding eCRF fields within the VOYAGER electronic data capture. Once pre-population of a visit was completed, site staff reviewed the eCRFs and, if required, edited erroneous fields and manually filled in fields that were not pre-populated. This study analyzed eCRF data from two UK VOYAGER sites, collected from patients for whom data were entered manually and patients for whom data were collected using EMR-to-EDC for ~6 months. Outcomes to assess the impact of EMR-to-EDC on data entry burden and accuracy were proportions of eCRF fields which were pre-populated and manually entered for pre-populated visits, and proportion of pre-populated fields overwritten by site staff. Site staff completed surveys to evaluate end-user satisfaction and acceptance of EMR-to-EDC.ResultsOverall, 49 baseline and 143 follow-up visits were registered, of which 146 (baseline: 39; follow-up: 107) were pre-populated by EMR-to-EDC, encompassing 5,017 baseline and 7,371 follow-up visit eCRF fields in total. Of these, 27.9% baseline and 20.5% follow-up visit fields were pre-populated by EMR-to-EDC. A low proportion of pre-populated baseline (8.1%) and follow-up (1.6%) fields were overwritten by site staff. Mean number of queries generated by the electronic data capture per visit was lower for pre-populated patients versus patients whose data were entered manually (baseline: 17.1 versus 22.0 (p = 0.22); follow-up: 4.1 versus 7.1 (p &lt; 0.05)). Survey results demonstrated that site staff generally agreed that EMR-to-EDC helped reduce study data entry burden and collect high quality data. Most staff estimated that EMR-to-EDC saved 11-20 min and 0-10 min per patient for baseline and follow-up visit data entry, respectively, by the end of the study. Main reported benefits of EMR-to-EDC were time-saving and quality data collection; main challenges were high number of system queries generated and pull-through of study-irrelevant data.ConclusionThese results support EMR-to-EDC as an innovative tool to efficiently transfer large amounts of electronic medical record data into study databases while maintaining data quality, with potential to faci","PeriodicalId":10685,"journal":{"name":"Clinical Trials","volume":" ","pages":"637-648"},"PeriodicalIF":2.2,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12647373/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144945709","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Some drawbacks of variable-weight composite endpoints.","authors":"Greg Hather, Polyna Khudyakov","doi":"10.1177/17407745251358820","DOIUrl":"https://doi.org/10.1177/17407745251358820","url":null,"abstract":"","PeriodicalId":10685,"journal":{"name":"Clinical Trials","volume":"22 6","pages":"763-764"},"PeriodicalIF":2.2,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145654054","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Optimising adherence to inpatient rehabilitation trial protocols: A mixed-methods systematic review.","authors":"Angela Logan, Jonathan Marsden, Jennifer Freeman, Emma Cork, Bridie Kent","doi":"10.1177/17407745251358262","DOIUrl":"10.1177/17407745251358262","url":null,"abstract":"<p><strong>Background: </strong>The results of rehabilitation clinical trials can be negatively affected by adherence to trial protocols. Adherence is multi-factorial, but studies often look at adherence factors separately. Therefore, a systematic review to appraise and synthesise the evidence is warranted to determine the barriers, facilitators and predictors associated with adherence to inpatient rehabilitation trial protocols, whether and how factors interact with one another, and how adherence to rehabilitation protocols can be optimised.</p><p><strong>Methods: </strong>A mixed-methods systematic review was conducted and reported in accordance with Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA). Databases searched were PubMed (Ovid), EMBASE (Ovid), MEDLINE (Ovid), CINAHL (Ovid), PsycINFO (Ovid), Cochrane Library, Health Technology Assessment Database, Web of Science and grey literature up to April 2024. A cohesive, integrated methodology was employed, leveraging the Consolidated Framework for Implementation Research (CFIR) 2.0, to transform, synthesise and integrate data from various methodologies to address the review objectives.</p><p><strong>Results: </strong>Twenty-seven studies met the inclusion criteria (randomised controlled trials, qualitative studies related to randomised controlled trials or mixed-methods). Most of the studies were in stroke (n = 17), but other studies included neurological, respiratory, cardiovascular, post-surgical, osteoarthritis and elderly medical. Multiple factors affecting adherence protocols were identified. Adherence was measured in various ways, and setting pre-specified adherence levels was uncommon.</p><p><strong>Conclusion: </strong>Adherence to inpatient rehabilitation trial protocols is multi-dimensional and multi-factorial. Consensus of adherence measurement and interpretation of adherence levels is needed to make meaningful comparisons between trials. A standardised approach, including adopting a traffic light system, would enable trialists to implement changes mid-trial or stop the trial to avoid research waste. Adopting approaches from behavioural science in the design and conduct of inpatient rehabilitation trials may overcome some of the behavioural barriers identified and optimise adherence for those delivering and receiving the intervention.</p><p><strong>Review registration: </strong>Prospective Register of Systematic Reviews, registration number CRD42021270121.</p>","PeriodicalId":10685,"journal":{"name":"Clinical Trials","volume":" ","pages":"728-744"},"PeriodicalIF":2.2,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12819895/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145451055","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Adjudication of cause of death in older adults: Learnings for death certification from the ASPirin in Reducing Events in the Elderly study.","authors":"Mark R Nelson, John J McNeil, Nigel Stocks, Sameer Panjwani, Raj C Shah","doi":"10.1177/17407745251368001","DOIUrl":"10.1177/17407745251368001","url":null,"abstract":"<p><strong>Background: </strong>Death certificates are a legal requirement for a body to be buried or cremated. Many randomised clinical trials utilise disease-specific causes of death as key outcomes informed by these documents. Determination of cause of death is commonly assigned to an adjudication committee, a time- and resource-intensive process which becomes more difficult in trials of older individuals. We sought to assess whether a simple transcription from a death certificate would be adequate to meet trial requirements and whether the certification process itself can be improved to meet public health and research requirements.</p><p><strong>Methods: </strong>Random audit of 100 death certificates from ASpirin in Reducing Endpoints in the Elderly, a randomised controlled trial conducted in Australian general practice and US community research centres. Participants were Australians (aged 70+ years) and Americans (65+) living in the community and without life-limiting medical conditions at baseline, recruited between 1 March 2010 and 31 December 2014 for the ASpirin in Reducing Endpoints in the Elderly trial. Outcome of interest was misclassification of death rate.</p><p><strong>Results: </strong>There were 2757 deaths in the 19,114 study population. In a random sample of 100 of these deaths, misclassification of cause of death was identified through the trial adjudication process in 9% of death certificates.</p><p><strong>Conclusion: </strong>In trials without the resources of ASpirin in Reducing Endpoints in the Elderly, a coding method can be accepted. Based on our experience, we recommend changes to the structure and process of death certification to better serve both clinical research and public health needs, particularly in older, multimorbid populations.</p>","PeriodicalId":10685,"journal":{"name":"Clinical Trials","volume":" ","pages":"695-700"},"PeriodicalIF":2.4,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12614313/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145224993","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}