Explanatory and Pragmatic Attitudes in Therapeutical Trials

Schwartz, D.; Lellouch, J

doi:10.1016/j.jclinepi.2009.01.012

Cited by 712 publications

(321 citation statements)

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“…Thus, although the effects were likely partly due to bias, the effects were not just bias. When blinding is not possible, such as in a dietary pattern intervention, it is often more justifiable to administer the intervention in its full context, so-called pragmatic trials (Schwartz & Lellouch, 2009). In this case, expectations about the effects of certain dietary advises, apparently are part of that context.…”

Section: Prior Expectations and Biasmentioning

confidence: 99%

“…In this case, expectations about the effects of certain dietary advises, apparently are part of that context. When it comes to answering the question which of two dietary advises should be preferred, pragmatic approaches are often more justifiable than approaches that are aimed at understanding (Schwartz & Lellouch, 2009). Nevertheless, in future studies, it would be interesting to include relevant biomarkers to investigate if those individuals with greatest changes in biomarker values, are also the ones with greatest changes in symptoms.…”

Section: Prior Expectations and Biasmentioning

confidence: 99%

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Feasibility of a Web-Based Cross-Over Paleolithic Diet Intervention in the General Population

Nederhof¹

2017

jevohealth

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Section: Prior Expectations and Biasmentioning

confidence: 99%

Section: Prior Expectations and Biasmentioning

confidence: 99%

Feasibility of a Web-Based Cross-Over Paleolithic Diet Intervention in the General Population

Nederhof¹

2017

jevohealth

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“…In a classic text from 1967, Schwartz and Lellouch coined the terms "pragmatic" and "explanatory" trials in order to distinguish between trials designed to answer two different research questions. While the pragmatic trial is well suited to establish knowledge that can help choose between options for care, the explanatory trial is well suited for testing causal research hypotheses, for example that a particular intervention causes a particular biological change (Schwartz & Lellouch, 2009;Zwarenstein et al, 2008). In other words, the explanatory trial aims to test a hypothesis of the effect of a particular anticipated active ingredient in order to contribute to the understanding and explanation of the treatment efficacy, while pragmatic trials concern whether the therapy is effective in practice as usual without considering the active ingredients or working mechanisms (Zwarenstein et al 2008).…”

Section: Pragmatic Trials As a Starting Point For Individualized Treamentioning

confidence: 99%

“…However, in order distinct types of design for the evaluation of the effect of an intervention (Gartlehner, Hansen, Nissman, Lohr, & Carey, 2006;Schwartz & Lellouch, 2009), it has been argued, that they should be seen as ideal types of design, and that actual trials most often will be positioned in between the two (Thorpe et al, 2009). In this perspective, the two types of design are not seen as distinct (and clean) trials, but merely as two ends of a continuum in which actual trials will be positioned according to the choices within ten domain (see Table 1).…”

Section: A Pragmatic-explanatory Continuum Indicatormentioning

confidence: 99%

Designing effectiveness studies of individualized treatments with personalized outcomes.

Johannessen¹

2015

MRAJ

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Background: Patients with chronic or severe health problems or involved in rehabilitation programs often have needs that vary greatly. As a response to this, the health interventions are most often individualized and aimed at personalized outcomes. Aim:The aim of this paper is to investigate how to design clinical trials that can measure the effectiveness of individualized interventions with personalized outcomes. Methods:With departure in a discussion of pragmatic trials designed to assess outcomes of individualized treatments, further developments for acknowledging patients' personal priorities in outcomes are explored, including a discussion of randomization vs self-selection and presentation of flexible tools for the selection of personalized primary outcomes. Finally, the relevance of a cross-disciplinary approach is suggested. Conclusion:Trials investigating the effectiveness of individualized interventions for personalized outcomes can be based on the design of pragmatic trials, with a few additional extensions. Tools for outcome must allow for personalized preferences and may include interviews, questionnaires with open formulation of concerns, or personal prioritizing of issues covered by validated questionnaires. In order to encompass patients' personal choice of outcomes, a cross-disciplinary methodology may be necessary.

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“…Pragmatic trials have been defined as those with broad inclusion criteria, carried out in many centres and with patient-relevant outcomes. 32 To employ a term given prominence in the Cooksey Report (2006), 33 NHS-funded research had to be restricted to public interest or market failure research, that is, work that the private sector would not be interested in carrying out. This is often due to the inability to patent that which is being tested (difficult outside new drugs or, in particular, interventions made up of services rather than tightly defined products).…”

mentioning

confidence: 99%

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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Explanatory and Pragmatic Attitudes in Therapeutical Trials

Cited by 712 publications

References 4 publications

Feasibility of a Web-Based Cross-Over Paleolithic Diet Intervention in the General Population

Feasibility of a Web-Based Cross-Over Paleolithic Diet Intervention in the General Population

Designing effectiveness studies of individualized treatments with personalized outcomes.

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

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