Systematic Review of the “Pragmatism” of Pragmatic Critical Care Trials

Palakshappa, Jessica A.; Gibbs, Kevin W.; Lannan, M.; Cranford, A.; Taylor, Stephanie Parks

doi:10.1097/cce.0000000000000738

Cited by 8 publications

(2 citation statements)

References 41 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…2,13 These have served to underline the importance and subtlety of the distinction made by Schwartz and Lellouch over half a century ago, the implications of which remain a focus of active research, such as surveys of how trials labelled as ‘pragmatic’ differ from others. 14,15…”

Section: Introductionmentioning

confidence: 99%

The differences and overlaps between ‘explanatory’ and ‘pragmatic’ controlled trials: a historical perspective

Glasziou,

Matthews,

Boutron

et al. 2023

J R Soc Med

View full text Add to dashboard Cite

In designing a controlled trial, a potential tension may exist between (1) providing insights into therapeutic mechanisms and (2) guiding decisions about 'what works' in everyday practice. In their 1967 paper in the Journal of Chronic Diseases, Schwartz and Lellouch 1 designated these aims as 'explanatory' and 'pragmatic', illustrating the potential tension with the different trial designs needed to assess the effect of an agent hypothesised to increase responsiveness to radiotherapy (a 'radiosensitiser') with immediate initiation of radiotherapy in the 'pragmatic' design versus a 30-day delay in initiating radiotherapy for the 'explanatory' design. This dilemma is manifest in real clinical situations, such as chemotherapy or radiotherapy prior to cancer surgery ('neoadjuvant therapy'), 'pre-habilitation' to prepare patients for joint surgery and whether to use a placebo when comparing two versus four times daily drug dosing.Most study design choices are not so clearly and unidimensionally 'pragmatic' or 'explanatory'. The PRagmatic-Explanatory Continuum Indicator Summary (PRECIS) tool 2 was developed to provide a graphical assessment of this spectrum for 10 'domains' that include eligibility criteria, flexibility of interventions, practitioner expertise, intensity of follow-up, compliance with treatments and scope of the analysis.The 'explanatory' versus 'pragmatic' tensions can sometimes be resolved. We suggest three categories:(1) sometimes both 'explanatory' and 'pragmatic' objectives of a trial can be achieved by aligning the design to address both; (2) sometimes there are possible compromises, for example, using an 'explanatory' sub-study within the framework of a 'pragmatic' main trial; and (3) rarely, there are incompatible objectives as illustrated by Schwartz and Lellouch. 1 Both the 'explanatory' and 'pragmatic' aims of trials are important. In general, as research moves from an understanding of the mode of therapeutic action towards clinical application, studies move increasingly from the 'explanatory' mode to the 'pragmatic' mode. Recognising when and how to focus on 'explanatory' versus 'pragmatic' reasons for research will remain an important element in that judgement.

show abstract

Section: Introductionmentioning

confidence: 99%

The differences and overlaps between ‘explanatory’ and ‘pragmatic’ controlled trials: a historical perspective

Glasziou,

Matthews,

Boutron

et al. 2023

J R Soc Med

View full text Add to dashboard Cite

show abstract

“…This is also the case for other traditional medicine practices [30], and even if randomized clinical trials conform and can be pooled, there are methodological features of these approaches which may lead to statistical heterogeneity, and do not reflect real world clinical practice [31]. Pragmatic studies are increasing in every field of medicine [32]. It is now recognized that the complexity of care must be addressed in order for an intervention to be applicable to the real world, and studies and consequent data synthesis requires a different methodology that resembles routine practice.…”

mentioning

confidence: 99%

Best Evidence for Best Care

Robinson

2023

PIM

View full text Add to dashboard Cite

Averaged versus individualized: pragmatic N-of-1 design as a method to investigate individual treatment response

Serpico,

Maziarz

2023

Euro Jnl Phil Sci

View full text Add to dashboard Cite

Heterogeneous treatment effects represent a major issue for medicine as they undermine reliable inference and clinical decision-making. To overcome the issue, the current vision of precision and personalized medicine acknowledges the need to control individual variability in response to treatment. In this paper, we argue that gene-treatment-environment interactions (G × T × E) undermine inferences about individual treatment effects from the results of both genomics-based methodologies—such as genome-wide association studies (GWAS) and genome-wide interaction studies (GWIS)—and randomized controlled trials (RCTs). Then, we argue that N-of-1 trials can be a solution to overcome difficulties in handling individual variability in treatment response. Although this type of trial has been suggested as a promising strategy to assess individual treatment effects, it nonetheless has limitations that limit its use in everyday clinical practice. We analyze the existing variability within the designs of N-of-1 trials in terms of a continuum where each design prioritizes epistemic and pragmatic considerations. We then support wider use of the designs located at the pragmatic end of the explanatory-pragmatic continuum.

show abstract

Systematic Review of the “Pragmatism” of Pragmatic Critical Care Trials

Abstract: To assess the pragmatism of published critical care randomized controlled trials self-described as pragmatic using a validated tool.

Cited by 8 publications

References 41 publications

The differences and overlaps between ‘explanatory’ and ‘pragmatic’ controlled trials: a historical perspective

The differences and overlaps between ‘explanatory’ and ‘pragmatic’ controlled trials: a historical perspective

Best Evidence for Best Care

Averaged versus individualized: pragmatic N-of-1 design as a method to investigate individual treatment response

Contact Info

Product

Resources

About