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Getting it wrong most of the time? Comparing trialists’ choice of primary outcome with what patients and health professionals want
BACKGROUND: Randomised trials support improved decision-making through the data they collect. One important piece of data is the primary outcome — so called because it is what the investigators decide is the most important. Secondary outcomes provide additional information to support decision-making...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9235090/ https://www.ncbi.nlm.nih.gov/pubmed/35761293 http://dx.doi.org/10.1186/s13063-022-06348-z |
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author | Treweek, Shaun Miyakoda, Viviane Burke, Dylan Shiely, Frances |
author_facet | Treweek, Shaun Miyakoda, Viviane Burke, Dylan Shiely, Frances |
author_sort | Treweek, Shaun |
collection | PubMed |
description | BACKGROUND: Randomised trials support improved decision-making through the data they collect. One important piece of data is the primary outcome — so called because it is what the investigators decide is the most important. Secondary outcomes provide additional information to support decision-making. We were interested in knowing how important patients and healthcare professionals consider the outcomes (especially the primary outcome) measured in a selection of published trials. METHODS: The work had three stages: (1) We identified a body of late-stage trials in two clinical areas, breast cancer management and nephrology. (2) We identified the primary and secondary outcomes for these trials. (3) We randomly ordered these outcomes and presented them to patients and healthcare professionals (with experience of the clinical area), and we asked them to rank the importance of the outcomes. They were not told which outcomes trial authors considered primary and secondary. RESULTS: In our sample of 44 trials with 46 primary outcomes, 29 patients, one patient representative and 12 healthcare professionals together ranked the primary outcome as the most important outcome 13/46 times or 28%. Breast cancer patients and healthcare professionals considered the primary outcome to be the most important outcome for 8/21 primary outcomes chosen by trialists. For nephrology, the equivalent figure was 5/25. The primary outcome appeared in a respondent’s top 5 ranked outcomes 151/178 (85%) times for breast cancer and 225/259 (87%) times for nephrology even if the primary was not considered the most important outcome. CONCLUSIONS: The primary outcome in a trial is the most important piece of data collected. It is used to determine how many participants are required, and it is the main piece of information used to judge whether the intervention is effective or not. In our study, patients and healthcare professionals agreed with the choice of the primary outcome made by trial teams doing late-stage trials in breast cancer management and nephrology 28% of the time. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1186/s13063-022-06348-z. |
format | Online Article Text |
id | pubmed-9235090 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | BioMed Central |
record_format | MEDLINE/PubMed |
spelling | pubmed-92350902022-06-28 Getting it wrong most of the time? Comparing trialists’ choice of primary outcome with what patients and health professionals want Treweek, Shaun Miyakoda, Viviane Burke, Dylan Shiely, Frances Trials Methodology BACKGROUND: Randomised trials support improved decision-making through the data they collect. One important piece of data is the primary outcome — so called because it is what the investigators decide is the most important. Secondary outcomes provide additional information to support decision-making. We were interested in knowing how important patients and healthcare professionals consider the outcomes (especially the primary outcome) measured in a selection of published trials. METHODS: The work had three stages: (1) We identified a body of late-stage trials in two clinical areas, breast cancer management and nephrology. (2) We identified the primary and secondary outcomes for these trials. (3) We randomly ordered these outcomes and presented them to patients and healthcare professionals (with experience of the clinical area), and we asked them to rank the importance of the outcomes. They were not told which outcomes trial authors considered primary and secondary. RESULTS: In our sample of 44 trials with 46 primary outcomes, 29 patients, one patient representative and 12 healthcare professionals together ranked the primary outcome as the most important outcome 13/46 times or 28%. Breast cancer patients and healthcare professionals considered the primary outcome to be the most important outcome for 8/21 primary outcomes chosen by trialists. For nephrology, the equivalent figure was 5/25. The primary outcome appeared in a respondent’s top 5 ranked outcomes 151/178 (85%) times for breast cancer and 225/259 (87%) times for nephrology even if the primary was not considered the most important outcome. CONCLUSIONS: The primary outcome in a trial is the most important piece of data collected. It is used to determine how many participants are required, and it is the main piece of information used to judge whether the intervention is effective or not. In our study, patients and healthcare professionals agreed with the choice of the primary outcome made by trial teams doing late-stage trials in breast cancer management and nephrology 28% of the time. SUPPLEMENTARY INFORMATION: The online version contains supplementary material available at 10.1186/s13063-022-06348-z. BioMed Central 2022-06-27 /pmc/articles/PMC9235090/ /pubmed/35761293 http://dx.doi.org/10.1186/s13063-022-06348-z Text en © The Author(s) 2022 https://creativecommons.org/licenses/by/4.0/Open AccessThis article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) . The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/ (https://creativecommons.org/publicdomain/zero/1.0/) ) applies to the data made available in this article, unless otherwise stated in a credit line to the data. |
spellingShingle | Methodology Treweek, Shaun Miyakoda, Viviane Burke, Dylan Shiely, Frances Getting it wrong most of the time? Comparing trialists’ choice of primary outcome with what patients and health professionals want |
title | Getting it wrong most of the time? Comparing trialists’ choice of primary outcome with what patients and health professionals want |
title_full | Getting it wrong most of the time? Comparing trialists’ choice of primary outcome with what patients and health professionals want |
title_fullStr | Getting it wrong most of the time? Comparing trialists’ choice of primary outcome with what patients and health professionals want |
title_full_unstemmed | Getting it wrong most of the time? Comparing trialists’ choice of primary outcome with what patients and health professionals want |
title_short | Getting it wrong most of the time? Comparing trialists’ choice of primary outcome with what patients and health professionals want |
title_sort | getting it wrong most of the time? comparing trialists’ choice of primary outcome with what patients and health professionals want |
topic | Methodology |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9235090/ https://www.ncbi.nlm.nih.gov/pubmed/35761293 http://dx.doi.org/10.1186/s13063-022-06348-z |
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