Clustering by health professional in individually randomised trials.
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ABSTRACT:
SUBMITTER: Lee KJ
PROVIDER: S-EPMC544437 | biostudies-literature | 2005 Jan
REPOSITORIES: biostudies-literature
Publications
Clustering by health professional in individually randomised trials.
Lee Katherine J KJ Thompson Simon G SG
BMJ (Clinical research ed.) 20050101 7483
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Project description:BackgroundIt has long been advised to account for baseline covariates in the analysis of confirmatory randomised trials, with the main statistical justifications being that this increases power and, when a randomisation scheme balanced covariates, permits a valid estimate of experimental error. There are various methods available to account for covariates but it is not clear how to choose among them.MethodsTaking the perspective of writing a statistical analysis plan, we consider how to choose between the three most promising broad approaches: direct adjustment, standardisation and inverse-probability-of-treatment weighting.ResultsThe three approaches are similar in being asymptotically efficient, in losing efficiency with mis-specified covariate functions and in handling designed balance. If a marginal estimand is targeted (for example, a risk difference or survival difference), then direct adjustment should be avoided because it involves fitting non-standard models that are subject to convergence issues. Convergence is most likely with IPTW. Robust standard errors used by IPTW are anti-conservative at small sample sizes. All approaches can use similar methods to handle missing covariate data. With missing outcome data, each method has its own way to estimate a treatment effect in the all-randomised population. We illustrate some issues in a reanalysis of GetTested, a randomised trial designed to assess the effectiveness of an electonic sexually transmitted infection testing and results service.ConclusionsNo single approach is always best: the choice will depend on the trial context. We encourage trialists to consider all three methods more routinely.
| S-EPMC9014627 | biostudies-literature
Project description:BackgroundThe randomized controlled trial (RCT) is considered the gold standard study design to inform decisions about the effectiveness of interventions. However, a common limitation is inadequate reporting of the applicability of the intervention and trial results for people who are "socially disadvantaged" and this can affect policy-makers' decisions. We previously developed a framework for identifying health-equity-relevant trials, along with a reporting guideline for transparent reporting. In this study, we provide a descriptive assessment of health-equity considerations in 200 randomly sampled equity-relevant trials.MethodsWe developed a search strategy to identify health-equity-relevant trials published between 2013 and 2015. We randomly sorted the 4316 records identified by the search and screened studies until 100 individually randomized (RCTs) and 100 cluster randomized controlled trials (CRTs) were identified. We developed and pilot-tested a data extraction form based on our initial work, to inform the development of our reporting guideline for equity-relevant randomized trials.ResultsIn total, 39 trials (20%) were conducted in a low- and middle-income country and 157 trials (79%) in a high-income country focused on socially disadvantaged populations (78% CRTs, 79% RCTs). Seventy-four trials (37%) reported a subgroup analysis across a population characteristic associated with disadvantage (25% CRT, 49% RCTs), with 19% of included studies reporting subgroup analyses across sex, 9% across race/ethnicity/culture, and 4% across socioeconomic status. No subgroup analyses were reported for place of residence, occupation, religion, education, or social capital. One hundred and forty-one trials (71%) discussed the applicability of their results to one or more socially disadvantaged populations (68% of CRT, 73% of RCT).DiscussionIn this set of trials, selected for their relevance to health equity, data that were disaggregated for socially disadvantaged populations were rarely reported. We found that even when the data are available, opportunities to analyze health-equity considerations are frequently missed. The recently published equity extension of the Consolidated Reporting Standards for Randomized Trials (CONSORT-Equity) may help improve delineation of hypotheses related to socially disadvantaged populations, and transparency and completeness of reporting of health-equity considerations in RCTs. This study can serve as a baseline assessment of the reporting of equity considerations.
| S-EPMC7118943 | biostudies-literature
Project description:BackgroundIn randomised controlled trials, the assumption of independence of individual observations is fundamental to the design, analysis and interpretation of studies. However, in individually randomised trials in primary care, this assumption may be violated because patients are naturally clustered within primary care practices. Ignoring clustering may lead to a loss of power or, in some cases, type I error.MethodsClustering can be quantified by intra-cluster correlation (ICC), a measure of the similarity between individuals within a cluster with respect to a particular outcome. We reviewed 17 trials undertaken by the Department of Primary Care at the University of Southampton over the last ten years. We calculated the ICC for the primary and secondary outcomes in each trial at the practice level and determined whether ignoring practice-level clustering still gave valid inferences. Where multiple studies collected the same outcome measure, the median ICC was calculated for that outcome.ResultsThe median intra-cluster correlation (ICC) for all outcomes was 0.016, with interquartile range 0.00-0.03. The median ICC for symptom severity was 0.02 (interquartile range (IQR) 0.01 to 0.07) and for reconsultation with new or worsening symptoms was 0.01 (IQR 0.00, 0.07). For HADS anxiety the ICC was 0.04 (IQR 0.02, 0.05) and for HADS depression was 0.02 (IQR 0.00, 0.05). The median ICC for EQ. 5D-3 L was 0.01 (IQR 0.01, 0.04).ConclusionsThere is evidence of clustering in individually randomised trials primary care. The non-zero ICC suggests that, depending on study design, clustering may not be ignorable. It is important that this is fully considered at the study design phase.
| S-EPMC7158044 | biostudies-literature
Project description:Health Professional Follow-Up Study
| PRJNA736488 | ENA
Project description:Health Professional Follow-Up Study
| PRJNA736489 | ENA
Project description:BackgroundTrial participant recruitment is an interactional process between health care professionals, patients and carers. Little is known about how clinicians carry out this role in palliative care trials and the reasons why they do or do not recruit participants.AimsTo explore how clinicians recruit to palliative care trials, why they choose to implement particular recruitment strategies, and the factors that influence their choices.DesignA qualitative multiple case study of three UK palliative care trials. Data collection included interviews and study documentation. Analysis involved developing and refining theoretical propositions, guided by the '6Ps' of the 'Social Marketing Mix Framework' as an a priori framework (identifying participants, product, price, place, promotion and working with partners). Framework Analysis guided within and then cross-case analysis.Settings/participantsStudy investigators and research staff (n = 3, 9, 7) from trial coordinating centres and recruitment sites (hospice and hospital).ResultsCross-case analysis suggests the 'Social Marketing Mix Framework' is useful for understanding recruitment processes but wider contextual issues need to be incorporated. These include the 'emotional labour' of diagnosing dying and communicating palliative and end-of-life care to potential participants and how the recruitment process is influenced by the power relationships and hierarchies that exist among professional groups. These factors can lead to and support paternalistic practices.ConclusionsThose planning trials need to ensure that trial recruiters, depending on their experience and trial characteristics, have access to training and support to address the 'emotional labour' of recruitment. The type of training required requires further research.
| S-EPMC10657513 | biostudies-literature
Project description:BackgroundImplementation of Professional Pharmacy Services (PPSs) requires a demonstration of the service's impact (efficacy) and its effectiveness. Several systematic reviews and randomised controlled trials (RCT) have shown the efficacy of PPSs in patient's outcomes in community pharmacy. There is, however, a need to determine the level of evidence on the effectiveness of PPSs in daily practice by means of pragmatic trials. To identify and analyse pragmatic RCTs that measure the effectiveness of PPSs in clinical, economic and humanistic outcomes in the community pharmacy setting.MethodsA systematic search was undertaken in MEDLINE, EMBASE, the Cochrane Library and SCIELO. The search was performed on January 31, 2020. Papers were assessed against the following inclusion criteria (1) The intervention could be defined as a PPS; (2) Undertaken in a community pharmacy setting; (3) Was an original paper; (4) Reported quantitative measures of at least one health outcome indicator (ECHO model); (5) The design was considered as a pragmatic RCT, that is, it fulfilled 3 predefined attributes. External validity was analyzed with PRECIS- 2 tool.ResultsThe search strategy retrieved 1,587 papers. A total of 12 pragmatic RCTs assessing 5 different types of PPSs were included. Nine out of the 12 papers showed positive statistically significant differences in one or more of the primary outcomes (clinical, economic or humanistic) that could be associated with the following PPS: Smoking cessation, Dispensing/Adherence service, Independent prescribing and MTM. No paper reported on cost-effectiveness outcomes.ConclusionsThere is limited available evidence on the effectiveness of community-based PPS. Pragmatic RCTs to evaluate clinical, humanistic and economic outcomes of PPS are needed.
| S-EPMC7890900 | biostudies-literature
Project description:Participants in trials may be randomized either individually or in groups and may receive their treatment either entirely individually, entirely in groups, or partially individually and partially in groups. This paper concerns cases in which participants receive their treatment either entirely or partially in groups, regardless of how they were randomized. Participants in group-randomized trials are randomized in groups, and participants in individually randomized group treatment trials are individually randomized, but participants in both types of trials receive part or all of their treatment in groups or through common change agents. Participants who receive part or all of their treatment in a group are expected to have positively correlated outcome measurements. This paper addresses a situation that occurs in group-randomized trials and individually randomized group treatment trials-participants receive treatment through more than one group. As motivation, we consider trials in The Childhood Obesity Prevention and Treatment Research Consortium, in which each child participant receives treatment in at least two groups. In simulation studies, we considered several possible analytic approaches over a variety of possible group structures. A mixed model with random effects for both groups provided the only consistent protection against inflated type I error rates and did so at the cost of only moderate loss of power when intraclass correlations were not large. We recommend constraining variance estimates to be positive and using the Kenward-Roger adjustment for degrees of freedom; this combination provided additional power but maintained type I error rates at the nominal level.
| S-EPMC4013262 | biostudies-other
Project description:Focusing on the case of time-lapse imaging (TLI), this paper analyses how medical professionals negotiate the use of new 'add-on' fertility treatments in light of the limited evidence available. The data produced by TLI technologies is meant to help professionals identify the best embryo to be implanted. Embryo selection is essential in IVF practice for increasing pregnancy rates and reducing the negative effects of repeated failures. More than 5 years after the introduction of TLI in IVF labs, however, there has been no conclusive randomised control trial (RCT) evidence to show that the tools do indeed have a significant impact on pregnancy rates. Nonetheless, many public clinics in the UK have adopted such technologies. Consequently, our research asks: How is the use of TLI tools legitimised by professionals, in light of contradictory evidence? Focusing on 25 semi-structured staff interviews, we argue that professionals use several strategies to legitimise the use of TLI in the clinic without, however, challenging the tenets of evidence-based medicine (EBM) and the value it places on RCTs. Rather, professionals emphasise various advantages that TLI offers, including its use as a lab tool, its potential for knowledge production in embryology, and the role it plays in the management of patient expectations and course of treatment. This paper contributes to debates on the role of EBM in modern medicine and fertility care specifically - an area where this inter-relationship has been underexplored. We conclude by suggesting avenues towards a more nuanced understanding of EBM as it relates to IVF treatment and a rapidly changing biotechnology context.
| S-EPMC7369645 | biostudies-literature
Project description:BackgroundThe COVID-19 pandemic has had adverse impacts on mental health and substance use worldwide. Systematic reviews suggest eHealth interventions can be effective at addressing these problems. However, strong positive eHealth outcomes are often tied to the intensity of web-based therapist guidance, which has time and cost implications that can make the population scale-up of more effective interventions difficult. A way to offset cost while maintaining the intensity of therapist guidance is to offer eHealth programs to groups rather than more standard one-on-one formats.ObjectiveThis systematic review aims to assess experimental evidence for the effectiveness of live health professional-led group eHealth interventions on mental health, substance use, or bereavement among community-dwelling adults. Within the articles selected for our primary aim, we also seek to examine the impact of interventions that encourage physical activity compared with those that do not.MethodsOverall, 4 databases (MEDLINE, CINAHL, PsycINFO, and the Cochrane Library) were searched in July 2020. Eligible studies were randomized controlled trials (RCTs) of eHealth interventions led by health professionals and delivered entirely to adult groups by videoconference, teleconference, or webchat. Eligible studies reported mental health, substance use, or bereavement as primary outcomes. The results were examined by outcome, eHealth platform, and intervention length. Postintervention data were used to calculate effect size by study. The findings were summarized using the Synthesis Without Meta-Analysis guidelines. Risk of bias was assessed using the Cochrane Collaboration Tool.ResultsOf the 4099 identified studies, 21 (0.51%) RCTs representing 20 interventions met the inclusion criteria. These studies examined mental health outcomes among 2438 participants (sample size range: 47-361 participants per study) across 7 countries. When effect sizes were pooled, live health professional-led group eHealth interventions had a medium effect on reducing anxiety compared with inactive (Cohen d=0.57) or active control (Cohen d=0.48), a medium to small effect on reducing depression compared with inactive (Cohen d=0.61) or active control (Cohen d=0.21), and mixed effects on mental distress and coping. Interventions led by videoconference, and those that provided 8-12 hours of live health professional-led group contact had more robust effects on adult mental health. Risk of bias was high in 91% (19/21) of the studies. Heterogeneity across interventions was significant, resulting in low to very low quality of evidence. No eligible RCT was found that examined substance use, bereavement, or physical activity.ConclusionsLive eHealth group interventions led by health professionals can foster moderate improvements in anxiety and moderate to small improvements in depression among community-based adults, particularly those delivered by videoconference and those providing 8-12 hours of synchronous engagement.Trial registrationPROSPERO CRD42020187551; https://www.crd.york.ac.uk/prospero/display_record.php?RecordID=187551.International registered report identifier (irrid)RR2-10.1186/s13643-020-01479-3.
| S-EPMC8790691 | biostudies-literature