A pragmatic–explanatory continuum indicator summary (PRECIS): a tool to help trial designers

Thorpe, Kevin E.; Zwarenstein, Merrick; Oxman, Andrew D; Treweek, Shaun; Furberg, Curt D.; Altman, Douglas G.; Tunis, Sean; Bergel, Eduardo; Harvey, Ian; Magid, David J.; Chalkidou, Kalipso

doi:10.1016/j.jclinepi.2008.12.011

Cited by 1,052 publications

(811 citation statements)

References 12 publications

(35 reference statements)

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“…Finally, we have not examined other factors that can affect applicability more broadly, such as provider characteristics and healthcare environment. 51 In summary, we have proposed quantitative methods for assessing the applicability of trial evidence to a target sample. The proposed methods exploit the advantages of a trial with respect to valid treatment effects and the advantages of observational data regarding a broad representation of target population.…”

Section: Discussionmentioning

confidence: 99%

Assessing the applicability of trial evidence to a target sample in the presence of heterogeneity of treatment effect

Weiss

Segal

Varadhan

2012

Pharmacoepidemiology and Drug

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Purpose To propose methods for the quantitative assessment of the applicability of evidence from a trial to a target sample using individual data. Methods Demonstration was with a trial of drug therapy to prevent mortality and an accompanying registry of people with heart failure. Principal components analysis with biplots did not identify measurement discrepancies. Multiple imputation with chained equations addressed missing predictor values. A proportional hazards model with interaction term, including graphical interpretation and a multivariate interaction test, identified heterogeneity of treatment effect. An interval of homogeneity of treatment effect was the interval of the baseline risk of outcome in which no two treatment effects were statistically significantly different. Absolute risk reduction for individuals was estimated for both benefit and harm outcomes and presented in a bivariate treatment effects scatterplot. Results Overall, the trial evidence applied to most of the registry according to overlapping distributions of estimated benefit and harm. However, 52% of trial and 33% of registry participants were estimated to have net benefit, and 14% of trial and 36% of registry participants were estimated to have strong net harmful treatment effect, that is, the individual estimate of harm was more than twice the estimate of benefit. Conclusions The proposed methods provide quantitative assessment of the applicability of trial evidence to a target sample. They combine the strengths of different study designs, namely, unbiased effects estimation from trials and representation in observational studies, while addressing the practical challenges of combining information, namely, measurement discrepancies and missing data.

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Section: Discussionmentioning

confidence: 99%

Assessing the applicability of trial evidence to a target sample in the presence of heterogeneity of treatment effect

Weiss

Segal

Varadhan

2012

Pharmacoepidemiology and Drug

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“…Two were considered not feasible: one on pragmatic-explanatory continuum indicator summary (PRECIS), 48 the other on complex interventions. The PRECIS question has 10 headings with up to six subheadings requiring approximately 68 data fields, or around one-third of the total fields for this theme.…”

Section: Methodsmentioning

confidence: 99%

“…Given the lack of data on basic aspects of the HTA RCTs, such as the number and types of interventions, more detailed work such as that required by PRECIS 48 or complex interventions seemed a task for further work.…”

Section: Methodsmentioning

confidence: 99%

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Clinical trial metadata: defining and extracting metadata on the design, conduct, results and costs of 125 randomised clinical trials funded by the National Institute for Health Research Health Technology Assessment programme

Raftery

Young

Stanton

et al. 2015

Health Technology Assessment

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BackgroundBy 2011, the Health Technology Assessment (HTA) programme had published the results of over 100 trials with another 220 in progress. The aim of the project was to develop and pilot ‘metadata’ on clinical trials funded by the HTA programme.ObjectivesThe aim of the project was to develop and pilot questions describing clinical trials funded by the HTA programme in terms of it meeting the needs of the NHS with scientifically robust studies. The objectives were to develop relevant classification systems and definitions for use in answering relevant questions and to assess their utility.Data sourcesPublished monographs and internal HTA documents.Review methodsA database was developed, ‘populated’ using retrospective data and used to answer questions under six prespecified themes. Questions were screened for feasibility in terms of data availability and/or ease of extraction. Answers were assessed by the authors in terms of completeness, success of the classification system used and resources required. Each question was scored to be retained, amended or dropped.ResultsOne hundred and twenty-five randomised trials were included in the database from 109 monographs. Neither the International Standard Randomised Controlled Trial Number nor the term ‘randomised trial’ in the title proved a reliable way of identifying randomised trials. Only limited data were available on how the trials aimed to meet the needs of the NHS. Most trials were shown to follow their protocols but updates were often necessary as hardly any trials recruited as planned. Details were often lacking on planned statistical analyses, but we did not have access to the relevant statistical plans. Almost all the trials reported on cost-effectiveness, often in terms of both the primary outcome and quality-adjusted life-years. The cost of trials was shown to depend on the number of centres and the duration of the trial. Of the 78 questions explored, 61 were well answered, 33 fully with 28 requiring amendment were the analysis updated. The other 17 could not be answered with readily available data.LimitationsThe study was limited by being confined to 125 randomised trials by one funder.ConclusionsMetadata on randomised controlled trials can be expanded to include aspects of design, performance, results and costs. The HTA programme should continue and extend the work reported here.FundingThe National Institute for Health Research HTA programme.

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“…To align with the principles of pragmatic trials, the usual care received was not standardized among participants [22].…”

Section: Interventionmentioning

confidence: 99%

Mobile App for Improved Self-Management of Type 2 Diabetes: Multicenter Pragmatic Randomized Controlled Trial (Preprint)

Agarwal¹,

Mukerji²,

Desveaux³

et al. 2018

Preprint

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Background: A growing number of mobile apps are available to support self-management of diabetes. One such app, called BlueStar™, has been shown to significantly reduce glycemic control in small trials. While the efficacy of the app led to become the first app in the USA to receive FDA approval as a Mobile Prescription Therapy, its effectiveness across a range of usual care settings remains unclear.

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A pragmatic–explanatory continuum indicator summary (PRECIS): a tool to help trial designers

Cited by 1,052 publications

References 12 publications

Assessing the applicability of trial evidence to a target sample in the presence of heterogeneity of treatment effect

Assessing the applicability of trial evidence to a target sample in the presence of heterogeneity of treatment effect

Clinical trial metadata: defining and extracting metadata on the design, conduct, results and costs of 125 randomised clinical trials funded by the National Institute for Health Research Health Technology Assessment programme

Mobile App for Improved Self-Management of Type 2 Diabetes: Multicenter Pragmatic Randomized Controlled Trial (Preprint)

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