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Real-world evidence: How pragmatic are randomized controlled trials labeled as pragmatic?
INTRODUCTION: Pragmatic randomized controlled trials (RCTs) mimic usual clinical practice and they are critical to inform decision-making by patients, clinicians and policy-makers in real-world settings. Pragmatic RCTs assess effectiveness of available medicines, while explanatory RCTs assess effica...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2018
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5883397/ https://www.ncbi.nlm.nih.gov/pubmed/29615035 http://dx.doi.org/10.1186/s12916-018-1038-2 |
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author | Dal-Ré, Rafael Janiaud, Perrine Ioannidis, John P. A. |
author_facet | Dal-Ré, Rafael Janiaud, Perrine Ioannidis, John P. A. |
author_sort | Dal-Ré, Rafael |
collection | PubMed |
description | INTRODUCTION: Pragmatic randomized controlled trials (RCTs) mimic usual clinical practice and they are critical to inform decision-making by patients, clinicians and policy-makers in real-world settings. Pragmatic RCTs assess effectiveness of available medicines, while explanatory RCTs assess efficacy of investigational medicines. Explanatory and pragmatic are the extremes of a continuum. This debate article seeks to evaluate and provide recommendation on how to characterize pragmatic RCTs in light of the current landscape of RCTs. It is supported by findings from a PubMed search conducted in August 2017, which retrieved 615 RCTs self-labeled in their titles as “pragmatic” or “naturalistic”. We focused on 89 of these trials that assessed medicines (drugs or biologics). DISCUSSION: 36% of these 89 trials were placebo-controlled, performed before licensing of the medicine, or done in a single-center. In our opinion, such RCTs overtly deviate from usual care and pragmatism. It follows, that the use of the term ‘pragmatic’ to describe them, conveys a misleading message to patients and clinicians. Furthermore, many other trials among the 615 coined as ‘pragmatic’ and assessing other types of intervention are plausibly not very pragmatic; however, this is impossible for a reader to tell without access to the full protocol and insider knowledge of the trial conduct. The degree of pragmatism should be evaluated by the trial investigators themselves using the PRECIS-2 tool, a tool that comprises 9 domains, each scored from 1 (very explanatory) to 5 (very pragmatic). CONCLUSIONS: To allow for a more appropriate characterization of the degree of pragmatism in clinical research, submissions of RCTs to funders, research ethics committees and to peer-reviewed journals should include a PRECIS-2 tool assessment done by the trial investigators. Clarity and accuracy on the extent to which a RCT is pragmatic will help understand how much it is relevant to real-world practice. |
format | Online Article Text |
id | pubmed-5883397 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2018 |
publisher | BioMed Central |
record_format | MEDLINE/PubMed |
spelling | pubmed-58833972018-04-10 Real-world evidence: How pragmatic are randomized controlled trials labeled as pragmatic? Dal-Ré, Rafael Janiaud, Perrine Ioannidis, John P. A. BMC Med Debate INTRODUCTION: Pragmatic randomized controlled trials (RCTs) mimic usual clinical practice and they are critical to inform decision-making by patients, clinicians and policy-makers in real-world settings. Pragmatic RCTs assess effectiveness of available medicines, while explanatory RCTs assess efficacy of investigational medicines. Explanatory and pragmatic are the extremes of a continuum. This debate article seeks to evaluate and provide recommendation on how to characterize pragmatic RCTs in light of the current landscape of RCTs. It is supported by findings from a PubMed search conducted in August 2017, which retrieved 615 RCTs self-labeled in their titles as “pragmatic” or “naturalistic”. We focused on 89 of these trials that assessed medicines (drugs or biologics). DISCUSSION: 36% of these 89 trials were placebo-controlled, performed before licensing of the medicine, or done in a single-center. In our opinion, such RCTs overtly deviate from usual care and pragmatism. It follows, that the use of the term ‘pragmatic’ to describe them, conveys a misleading message to patients and clinicians. Furthermore, many other trials among the 615 coined as ‘pragmatic’ and assessing other types of intervention are plausibly not very pragmatic; however, this is impossible for a reader to tell without access to the full protocol and insider knowledge of the trial conduct. The degree of pragmatism should be evaluated by the trial investigators themselves using the PRECIS-2 tool, a tool that comprises 9 domains, each scored from 1 (very explanatory) to 5 (very pragmatic). CONCLUSIONS: To allow for a more appropriate characterization of the degree of pragmatism in clinical research, submissions of RCTs to funders, research ethics committees and to peer-reviewed journals should include a PRECIS-2 tool assessment done by the trial investigators. Clarity and accuracy on the extent to which a RCT is pragmatic will help understand how much it is relevant to real-world practice. BioMed Central 2018-04-03 /pmc/articles/PMC5883397/ /pubmed/29615035 http://dx.doi.org/10.1186/s12916-018-1038-2 Text en © The Author(s). 2018 Open AccessThis article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated. |
spellingShingle | Debate Dal-Ré, Rafael Janiaud, Perrine Ioannidis, John P. A. Real-world evidence: How pragmatic are randomized controlled trials labeled as pragmatic? |
title | Real-world evidence: How pragmatic are randomized controlled trials labeled as pragmatic? |
title_full | Real-world evidence: How pragmatic are randomized controlled trials labeled as pragmatic? |
title_fullStr | Real-world evidence: How pragmatic are randomized controlled trials labeled as pragmatic? |
title_full_unstemmed | Real-world evidence: How pragmatic are randomized controlled trials labeled as pragmatic? |
title_short | Real-world evidence: How pragmatic are randomized controlled trials labeled as pragmatic? |
title_sort | real-world evidence: how pragmatic are randomized controlled trials labeled as pragmatic? |
topic | Debate |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5883397/ https://www.ncbi.nlm.nih.gov/pubmed/29615035 http://dx.doi.org/10.1186/s12916-018-1038-2 |
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