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Form follows function: pragmatic controlled trials (PCTs) have to answer different questions and require different designs than randomized controlled trials (RCTs)

AIM: Rising concern for demonstrated real world comparative effectiveness has heightened interest in “pragmatic trials” design. Pragmatic trials investigate whether the efficacy, presumed or found in explanatory trials under ideal conditions, can also be detected under real world conditions, i.e. ef...

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Detalles Bibliográficos
Autores principales: Porzsolt, Franz, Eisemann, Martin, Habs, Michael, Wyer, Peter
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer-Verlag 2012
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3655212/
https://www.ncbi.nlm.nih.gov/pubmed/23687408
http://dx.doi.org/10.1007/s10389-012-0544-5
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author Porzsolt, Franz
Eisemann, Martin
Habs, Michael
Wyer, Peter
author_facet Porzsolt, Franz
Eisemann, Martin
Habs, Michael
Wyer, Peter
author_sort Porzsolt, Franz
collection PubMed
description AIM: Rising concern for demonstrated real world comparative effectiveness has heightened interest in “pragmatic trials” design. Pragmatic trials investigate whether the efficacy, presumed or found in explanatory trials under ideal conditions, can also be detected under real world conditions, i.e. effectiveness. It is also recognized that ‘real world’ effects which are usually addressed in public health research gain growing interest in confirming the ‘road capability’ of results obtained under ideal study conditions. This paper demonstrates that studies under ideal or real world conditions use different methods, generate different information and cannot replace each other. SUBJECTS AND METHODS: The PCT design meets four requirements of public health and of effectiveness research. It includes all individuals who presented with the selected condition. It classifies the included individuals according to baseline risks. It enables plausibility controls. Finally, it compares the outcomes resulting from specified and not-specified interventions or treatments. PROPOSAL: We propose a pragmatic controlled trial (PCT) design in which patient preference and other co-factors crucial in determining the actual effectiveness of interventional options will not be neutralized by concealed randomization and blinding. This design is applicable to record the selected interventions and generated outcomes in day-to-day health care and is capable of incorporating preference and other participative factors into assessment of effectiveness. CONCLUSIONS: The PCT design is useful for public health research, e.g. the effectiveness of interventions to change smoking habits or to prevent death from breast cancer, as well as for comparative effectiveness research where it will supplement the traditional randomized controlled trial (RCT).
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spelling pubmed-36552122013-05-16 Form follows function: pragmatic controlled trials (PCTs) have to answer different questions and require different designs than randomized controlled trials (RCTs) Porzsolt, Franz Eisemann, Martin Habs, Michael Wyer, Peter Z Gesundh Wiss Overview AIM: Rising concern for demonstrated real world comparative effectiveness has heightened interest in “pragmatic trials” design. Pragmatic trials investigate whether the efficacy, presumed or found in explanatory trials under ideal conditions, can also be detected under real world conditions, i.e. effectiveness. It is also recognized that ‘real world’ effects which are usually addressed in public health research gain growing interest in confirming the ‘road capability’ of results obtained under ideal study conditions. This paper demonstrates that studies under ideal or real world conditions use different methods, generate different information and cannot replace each other. SUBJECTS AND METHODS: The PCT design meets four requirements of public health and of effectiveness research. It includes all individuals who presented with the selected condition. It classifies the included individuals according to baseline risks. It enables plausibility controls. Finally, it compares the outcomes resulting from specified and not-specified interventions or treatments. PROPOSAL: We propose a pragmatic controlled trial (PCT) design in which patient preference and other co-factors crucial in determining the actual effectiveness of interventional options will not be neutralized by concealed randomization and blinding. This design is applicable to record the selected interventions and generated outcomes in day-to-day health care and is capable of incorporating preference and other participative factors into assessment of effectiveness. CONCLUSIONS: The PCT design is useful for public health research, e.g. the effectiveness of interventions to change smoking habits or to prevent death from breast cancer, as well as for comparative effectiveness research where it will supplement the traditional randomized controlled trial (RCT). Springer-Verlag 2012-11-07 2013 /pmc/articles/PMC3655212/ /pubmed/23687408 http://dx.doi.org/10.1007/s10389-012-0544-5 Text en © The Author(s) 2012 https://creativecommons.org/licenses/by/4.0/ This article is distributed under the terms of the Creative Commons Attribution License which permits any use, distribution, and reproduction in any medium, provided the original author(s) and the source are credited.
spellingShingle Overview
Porzsolt, Franz
Eisemann, Martin
Habs, Michael
Wyer, Peter
Form follows function: pragmatic controlled trials (PCTs) have to answer different questions and require different designs than randomized controlled trials (RCTs)
title Form follows function: pragmatic controlled trials (PCTs) have to answer different questions and require different designs than randomized controlled trials (RCTs)
title_full Form follows function: pragmatic controlled trials (PCTs) have to answer different questions and require different designs than randomized controlled trials (RCTs)
title_fullStr Form follows function: pragmatic controlled trials (PCTs) have to answer different questions and require different designs than randomized controlled trials (RCTs)
title_full_unstemmed Form follows function: pragmatic controlled trials (PCTs) have to answer different questions and require different designs than randomized controlled trials (RCTs)
title_short Form follows function: pragmatic controlled trials (PCTs) have to answer different questions and require different designs than randomized controlled trials (RCTs)
title_sort form follows function: pragmatic controlled trials (pcts) have to answer different questions and require different designs than randomized controlled trials (rcts)
topic Overview
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3655212/
https://www.ncbi.nlm.nih.gov/pubmed/23687408
http://dx.doi.org/10.1007/s10389-012-0544-5
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