The Ceiling Effect, the Floor Effect, and the Importance of Active and Placebo Control Arms in Randomized Controlled Trials of an Investigational Drug

Randomized controlled trials (RCTs) of a new investigational drug often include active as well as placebo control arms. The active arm, comprising an approved treatment for the indication under study, along with the placebo arm, are together required to establish assay sensitivity; if the active tre...

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Autor principal: Andrade, Chittaranjan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: SAGE Publications 2021
Materias:
Learning Curve
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8327873/
https://www.ncbi.nlm.nih.gov/pubmed/34385732
http://dx.doi.org/10.1177/02537176211021280
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author Andrade, Chittaranjan
author_facet Andrade, Chittaranjan
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description Randomized controlled trials (RCTs) of a new investigational drug often include active as well as placebo control arms. The active arm, comprising an approved treatment for the indication under study, along with the placebo arm, are together required to establish assay sensitivity; if the active treatment outperforms placebo, as expected, the results of the RCT can be further interpreted, but if the active treatment is no better than placebo (such as because of ceiling or floor effects), the RCT is a failed trial. The concepts involved are explained from scientific and ethical perspectives.
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spelling pubmed-83278732021-08-11 The Ceiling Effect, the Floor Effect, and the Importance of Active and Placebo Control Arms in Randomized Controlled Trials of an Investigational Drug Andrade, Chittaranjan Indian J Psychol Med Learning Curve Randomized controlled trials (RCTs) of a new investigational drug often include active as well as placebo control arms. The active arm, comprising an approved treatment for the indication under study, along with the placebo arm, are together required to establish assay sensitivity; if the active treatment outperforms placebo, as expected, the results of the RCT can be further interpreted, but if the active treatment is no better than placebo (such as because of ceiling or floor effects), the RCT is a failed trial. The concepts involved are explained from scientific and ethical perspectives. SAGE Publications 2021-06-26 2021-07 /pmc/articles/PMC8327873/ /pubmed/34385732 http://dx.doi.org/10.1177/02537176211021280 Text en © 2021 Indian Psychiatric Society - South Zonal Branch https://creativecommons.org/licenses/by-nc/4.0/This article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 License (https://creativecommons.org/licenses/by-nc/4.0/) which permits non-commercial use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access page (https://us.sagepub.com/en-us/nam/open-access-at-sage).
spellingShingle Learning Curve
Andrade, Chittaranjan
The Ceiling Effect, the Floor Effect, and the Importance of Active and Placebo Control Arms in Randomized Controlled Trials of an Investigational Drug
title The Ceiling Effect, the Floor Effect, and the Importance of Active and Placebo Control Arms in Randomized Controlled Trials of an Investigational Drug
title_full The Ceiling Effect, the Floor Effect, and the Importance of Active and Placebo Control Arms in Randomized Controlled Trials of an Investigational Drug
title_fullStr The Ceiling Effect, the Floor Effect, and the Importance of Active and Placebo Control Arms in Randomized Controlled Trials of an Investigational Drug
title_full_unstemmed The Ceiling Effect, the Floor Effect, and the Importance of Active and Placebo Control Arms in Randomized Controlled Trials of an Investigational Drug
title_short The Ceiling Effect, the Floor Effect, and the Importance of Active and Placebo Control Arms in Randomized Controlled Trials of an Investigational Drug
title_sort ceiling effect, the floor effect, and the importance of active and placebo control arms in randomized controlled trials of an investigational drug
topic Learning Curve
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8327873/
https://www.ncbi.nlm.nih.gov/pubmed/34385732
http://dx.doi.org/10.1177/02537176211021280
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