Observational research, randomised trials, and two views of medical science.
Ontology highlight
ABSTRACT:
SUBMITTER: Vandenbroucke JP
PROVIDER: S-EPMC2265762 | biostudies-literature | 2008 Mar
REPOSITORIES: biostudies-literature
Publications
Observational research, randomised trials, and two views of medical science.
PLoS medicine 20080301 3
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Project description:BackgroundSeveral reviews of published cluster randomised trials have reported that about half did not take clustering into account in the analysis, which was thus incorrect and potentially misleading. In this paper I ask whether cluster randomised trials are increasing in both number and quality of reporting.MethodsComputer search for papers on cluster randomised trials since 1980, hand search of trial reports published in selected volumes of the British Medical Journal over 20 years.ResultsThere has been a large increase in the numbers of methodological papers and of trial reports using the term 'cluster random' in recent years, with about equal numbers of each type of paper. The British Medical Journal contained more such reports than any other journal. In this journal there was a corresponding increase over time in the number of trials where subjects were randomised in clusters. In 2003 all reports showed awareness of the need to allow for clustering in the analysis. In 1993 and before clustering was ignored in most such trials.ConclusionCluster trials are becoming more frequent and reporting is of higher quality. Perhaps statistician pressure works.
| S-EPMC515302 | biostudies-literature
Project description:Since the latter part of the 1990's, the English-speaking medical education community has been engaged in a debate concerning the types of research that should have priority. To shed light on this debate and to better understand its implications for the practice of research, 23 semi-structured interviews were conducted with "influential figures" from the community. The results were analyzed using the concept of "field" developed by the sociologist Pierre Bourdieu. The results reveal that a large majority of these influential figures believe that research in medical education continues to be of insufficient quality despite the progress that has taken place over the past 2 decades. According to this group, studies tend to be both redundant and opportunistic, and researchers tend to have limited understanding of both theory and methodological practice from the social sciences. Three factors were identified by the participants to explain the current problems in research: the working conditions of researchers, budgetary restraints in financing research in medical education, and the conception of research in the medical environment. Two principal means for improving research are presented: intensifying collaboration between PhD's and clinicians, and encouraging the diversification of perspectives brought to bear on research in medical education.
| S-EPMC2779425 | biostudies-other
Project description:BackgroundDespite efforts to improve the accuracy and transparency of the design, conduct, and reporting of randomised controlled trials (RCTs), deficiencies remain. Such deficiencies contribute to significant, avoidable waste of health research investment and impede reproducibility. This study aimed to synthesise and critically analyse changes over time in the conduct and reporting of internationally published evidence on patient and/or population health-oriented RCTs conducted in one country.MethodsThis observational study drew on systematic review methods. We searched six databases for published RCTs (database inception to December 2018) where ≥ 80% of participants were recruited in the Republic of Ireland. RCTs of interventions targeted at patients, providers and/or policy makers intended to improve health, healthcare or health research were included. For each study, screening, data extraction and methodological quality appraisal were conducted by one member of the author team.ResultsFrom 17,560 titles and abstracts, 752 unique RCTs were published in 745 papers between 1968 and 2018, with a steady year-on-year increase since 1968. The number of participants was in the range of 2-8628. The majority were parallel design (86%) and classified as treatment evaluation. Of the 418 RCTs published since the introduction of mandatory clinical trial registration by the International Committee of Medical Journal Editors in 2005, 32% (n = 134) provided a trial registration number. This increased to 47% when taking studies published between 2013 and 2018 (n = 232). Since the 1996 publication of the CONSORT statement, 16% of included RCTs made specific reference to a standardised reporting guideline and this increased to 31% for more recent studies published between 2013 and 2018. Overall, 7% (n = 53) of studies referred to a published study protocol, increasing to 20% for studies published between 2013 and 2018.ConclusionEvidence from this single-country study of RCTs published in the international literature suggests that both the number overall, the number registered and the number referencing reporting guidelines have increased steadily over time. Despite widespread endorsement of reporting standards, reporting of RCTs remains suboptimal in domains such as compliance with the CONSORT statement and prospective trial registration. Researchers, funders and journal editors, nationally and internationally, should continue to focus on improving reporting and examining avoidable waste of health research investment.
| S-EPMC7278139 | biostudies-literature
Project description:BackgroundMedical students engage in curricular and extracurricular activities, including undergraduate research (UR). The advantages, difficulties and motivations for medical students pursuing research activities during their studies have rarely been addressed. In Brazil, some medical schools have included undergraduate research into their curriculum. The present study aimed to understand the reality of scientific practice among medical students at a well-established Brazilian medical school, analyzing this context from the students' viewpoint.MethodsA cross-sectional survey based on a questionnaire applied to students from years one to six enrolled in an established Brazilian medical school that currently has no curricular UR program.ResultsThe questionnaire was answered by 415 students, 47.2% of whom were involved in research activities, with greater participation in UR in the second half of the course. Independent of student involvement in research activities, time constraints were cited as the main obstacle to participation. Among students not involved in UR, 91.1% said they favored its inclusion in the curriculum, since this would facilitate the development of such activity. This approach could signify an approximation between the axes of teaching and research. Among students who had completed at least one UR project, 87.7% said they would recommend the activity to students entering the course.ConclusionEven without an undergraduate research program, students of this medical school report strong involvement in research activities, but discussion of the difficulties inherent in its practice is important to future developments.
| S-EPMC4021277 | biostudies-literature
Project description:ObjectivesTo explore stakeholders' perceptions of decision aids designed to support the informed consent decision-making process for randomised controlled trials.DesignQualitative semistructured interviews. Participants were provided with prototype trial decision aids in advance to stimulate discussion. Interviews were analysed using an established interpretive approach.Participants23 stakeholders: Trial Managers (n=5); Research Nurses (n=5); Ethics Committee Chairs (n=5); patients (n=4) and Clinical Principal Investigators (n=4).SettingEmbedded within two ongoing randomised controlled trials. All interviews conducted with UK-based participants.ResultsCertain key aspects (eg, values clarification exercises, presentation of probabilities, experiences of others and balance of options) in the prototype decision aids were perceived by all stakeholders as having a significant advantage (over existing patient information leaflets) in terms of supporting well informed appropriate decisions. However, there were some important differences between the stakeholder groups on specific content (eg, language used in the section on positive and negative features of taking part in a trial and the overall length of the trial decision aids). Generally the stakeholders believed trial decision aids have the potential to better engage potential participants in the decision-making process and allow them to make more personally relevant decisions about their participation.ConclusionsCompared to existing patient information leaflets, stakeholders perceived decision aids for trial participation to have the potential to promote a more 'informed' decision-making process. Further efforts to develop, refine and formally evaluate trial decision aids should be explored.
| S-EPMC4139633 | biostudies-literature
Project description:BackgroundRecruitment of patients is one of the main challenges when designing and conducting randomised controlled trials (RCTs). Trials of rare injuries or those that include surgical interventions pose added challenges due to the small number of potentially eligible patients and issues with patient preferences and surgeon equipoise. We explore key issues to consider when recruiting to orthopaedic surgical trials from the perspective of staff and patients with the aim of informing the development of strategies to improve recruitment in future research.DesignTwo qualitative process evaluations of a UK-wide orthopaedic surgical RCT (ACTIVE) and mixed methods randomised feasibility study (PRESTO). Qualitative semi-structured interviews were conducted and data was analysed thematically.SettingNHS secondary care organisations throughout the UK. Interviews were undertaken via telephone.ParticipantsThirty-seven health professionals including UK-based spinal and orthopaedic surgeons and individuals involved in recruitment to the ACTIVE and PRESTO studies (e.g. research nurses, surgeons, physiotherapists). Twenty-two patients including patients who agreed to participate in the ACTIVE and PRESTO studies (n=15) and patients that declined participation in the ACTIVE study (n=7) were interviewed.ResultsWe used a mixed methods systematic review of recruiting patients to randomised controlled trials as a framework for reporting and analysing our findings. Our findings mapped onto those identified in the systematic review and highlighted the importance of equipoise, randomisation, communication, patient's circumstances, altruism and trust in clinical and research teams. Our findings also emphasised the importance of considering how eligibility criteria are operationalised and the impact of complex patient pathways when recruiting to surgical trials. In particular, the influence of health professionals, who are not involved in trial recruitment, on patients' treatment preferences by suggesting they would receive a certain treatment ahead of recruitment consultations should not be underestimated.ConclusionsA wealth of evidence exploring factors affecting recruitment to randomised controlled trials exists. A methodological shift is now required to ensure that this evidence is used by all those involved in recruitment and to ensure that existing knowledge is translated into methods for optimising recruitment to future trials.Trial registrationACTIVE: ( ISRCTN98152560 ). Registered on 06/03/2018. PRESTO: ( ISRCTN12094890 ). Registered on 22/02/2018.
| S-EPMC8285860 | biostudies-literature
Project description:BackgroundIn sepsis and acute respiratory distress syndrome (ARDS), heterogeneity has contributed to difficulty identifying effective pharmacotherapies. In ARDS, two molecular phenotypes (hypoinflammatory and hyperinflammatory) have consistently been identified, with divergent outcomes and treatment responses. In this study, we sought to derive molecular phenotypes in critically ill adults with sepsis, determine their overlap with previous ARDS phenotypes, and evaluate whether they respond differently to treatment in completed sepsis trials.MethodsWe used clinical data and plasma biomarkers from two prospective sepsis cohorts, the Validating Acute Lung Injury biomarkers for Diagnosis (VALID) study (N=1140) and the Early Assessment of Renal and Lung Injury (EARLI) study (N=818), in latent class analysis (LCA) to identify the optimal number of classes in each cohort independently. We used validated models trained to classify ARDS phenotypes to evaluate concordance of sepsis and ARDS phenotypes. We applied these models retrospectively to the previously published Prospective Recombinant Human Activated Protein C Worldwide Evaluation in Severe Sepsis and Septic Shock (PROWESS-SHOCK) trial and Vasopressin and Septic Shock Trial (VASST) to assign phenotypes and evaluate heterogeneity of treatment effect.FindingsA two-class model best fit both VALID and EARLI (p<0·0001). In VALID, 804 (70·5%) of the 1140 patients were classified as hypoinflammatory and 336 (29·5%) as hyperinflammatory; in EARLI, 530 (64·8%) of 818 were hypoinflammatory and 288 (35·2%) hyperinflammatory. We observed higher plasma pro-inflammatory cytokines, more vasopressor use, more bacteraemia, lower protein C, and higher mortality in the hyperinflammatory than in the hypoinflammatory phenotype (p<0·0001 for all). Classifier models indicated strong concordance between sepsis phenotypes and previously identified ARDS phenotypes (area under the curve 0·87-0·96, depending on the model). Findings were similar excluding participants with both sepsis and ARDS. In PROWESS-SHOCK, 1142 (68·0%) of 1680 patients had the hypoinflammatory phenotype and 538 (32·0%) had the hyperinflammatory phenotype, and response to activated protein C differed by phenotype (p=0·0043). In VASST, phenotype proportions were similar to other cohorts; however, no treatment interaction with the type of vasopressor was observed (p=0·72).InterpretationMolecular phenotypes previously identified in ARDS are also identifiable in multiple sepsis cohorts and respond differently to activated protein C. Molecular phenotypes could represent a treatable trait in critical illness beyond the patient's syndromic diagnosis.FundingUS National Institutes of Health.
| S-EPMC10841178 | biostudies-literature
Project description:IntroductionThe American University of Beirut Medical Center (AUBMC) developed the Fellowship and Residency Research Program (FRRP) to provide residents and clinical fellows with a supervised and structured research experience. The objective of this study was to explore the views of FRRP participants about the training program and how to enhance it.MethodsIn 2018, we conducted a qualitative study targeting residents where we invited potential participants through email and via snowball sampling. We continued the data collection until we reached data saturation with 21 participants (N = 21) and used thematic analysis to identify emerging themes.ResultsThematic analysis generated four emerging themes: one related to the expectations of residents, while the remaining three themes related to how the program is working to meet those expectations, specifically: coordination, mentorship, and capacity building. For these three latter themes, we discussed the strengths and challenges related to each. In terms of coordination, few residents complained that the deadlines to submit deliverables are not evenly distributed across the years. In terms of mentorship, participants appreciated the flexibility in choosing the mentor. In terms of capacity building activities, participants found the lecture series, both real time and virtual, to be helpful. Finally, participants pointed out that the FRRP program should be mandatory.DiscussionTo provide residents and clinical fellows with a supervised and structured research experience, we have built on our findings to introduce several changes to our program such as ensuring the availability and commitment of faculty mentors, and providing capacity building activities to the program participants.
| S-EPMC8782500 | biostudies-literature
Project description:BackgroundOpen Science Practices (OSPs) are essential when assessing research integrity and quality of Randomised Clinical Trials (RCTs). As dental caries represents a significant oral health burden, our objective was to identify and analyse the adoption of OSPs within RCTs focused on addressing this disease.MethodsWe searched PubMed to retrieve RCTs related to dental caries published from January 2000 to March 2022. Two independent researchers assessed a random sample of these articles to evaluate their eligibility until reaching the minimum sample size. Then, the same examiners reviewed the included texts regarding the OSPs adopted in the articles. The collected variables related to OSPs were reporting guidelines, protocol registration, detailed methodology available, open-source software, statistical analysis code sharing, statistical analysis plan, data sharing, open peer review, and open access. Association analyses using logistic regression were conducted considering the publication year, the continent of the first author, impact factor and open-access policy of the journals (explanatory variables), and adoption of at least one OSP or one OSP other than open access (outcomes). The recommendations for adopting OSPs were assessed by reviewing the "Instructions for Authors" section of the most frequently used journals where the included papers were published.Results64.8% of the articles (95% Confidence Interval = 59.3-70.1%) adopted at least one OSP. However, no individual OSP was adopted by more than 50% of the articles. The most adopted practices were protocol registration (37.1%), the use of reporting guidelines (33.1%) and publishing open access (37.3%). These are also the OSPs most often recommended by journals in the Instructions for Authors. A few articles adopted other practices. Older articles presented a lower frequency of adopting these practices, and articles published in higher impact factor journals were positively associated with both outcomes.ConclusionThe RCTs published on dental caries demonstrate a low frequency of adoption of most OSPs. However, a trend toward increased adoption of these practices has been notable in recent years.
| S-EPMC11585932 | biostudies-literature
Project description:We established patient-derived organoids and monolayer culture cells from the salivary gland cancer cases. To compare the RNA profiles of primary culture cells (Organoids and monolayer culture cells) and their parental tumors, we isolated total RNA from 2 cases of the salivary gland cancer and performed transcriptome sequencing for the organoids, monolayer culture cells, and their parental tumors of both cases. Case 6 is a case of adenoid cystic carcinoma and Case 11 is a case of salivary duct carcinoma.
2023-04-27 | GSE223554 | GEO