Some recommendations for multi-arm multi-stage trials
Multi-arm multi-stage designs can improve the efficiency of the drug-development process by evaluating multiple experimental arms against a common control within one trial. This reduces the number of patients required compared to a series of trials testing each experimental arm separately against co...
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| Format: | Online |
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SAGE Publications
2012
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| Online Access: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4843088/ |
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pubmed-48430882016-05-24 Some recommendations for multi-arm multi-stage trials Wason, James Magirr, Dominic Law, Martin Jaki, Thomas Articles Multi-arm multi-stage designs can improve the efficiency of the drug-development process by evaluating multiple experimental arms against a common control within one trial. This reduces the number of patients required compared to a series of trials testing each experimental arm separately against control. By allowing for multiple stages experimental treatments can be eliminated early from the study if they are unlikely to be significantly better than control. Using the TAILoR trial as a motivating example, we explore a broad range of statistical issues related to multi-arm multi-stage trials including a comparison of different ways to power a multi-arm multi-stage trial; choosing the allocation ratio to the control group compared to other experimental arms; the consequences of adding additional experimental arms during a multi-arm multi-stage trial, and how one might control the type-I error rate when this is necessary; and modifying the stopping boundaries of a multi-arm multi-stage design to account for unknown variance in the treatment outcome. Multi-arm multi-stage trials represent a large financial investment, and so considering their design carefully is important to ensure efficiency and that they have a good chance of succeeding. SAGE Publications 2012-12-12 2016-04 /pmc/articles/PMC4843088/ /pubmed/23242385 http://dx.doi.org/10.1177/0962280212465498 Text en © The Author(s) 2012 http://creativecommons.org/licenses/by-nc/3.0/ This article is distributed under the terms of the Creative Commons Attribution-NonCommercial 3.0 License (http://www.creativecommons.org/licenses/by-nc/3.0/) 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). |
| repository_type |
Open Access Journal |
| institution_category |
Foreign Institution |
| institution |
US National Center for Biotechnology Information |
| building |
NCBI PubMed |
| collection |
Online Access |
| language |
English |
| format |
Online |
| author |
Wason, James Magirr, Dominic Law, Martin Jaki, Thomas |
| spellingShingle |
Wason, James Magirr, Dominic Law, Martin Jaki, Thomas Some recommendations for multi-arm multi-stage trials |
| author_facet |
Wason, James Magirr, Dominic Law, Martin Jaki, Thomas |
| author_sort |
Wason, James |
| title |
Some recommendations for multi-arm multi-stage trials |
| title_short |
Some recommendations for multi-arm multi-stage trials |
| title_full |
Some recommendations for multi-arm multi-stage trials |
| title_fullStr |
Some recommendations for multi-arm multi-stage trials |
| title_full_unstemmed |
Some recommendations for multi-arm multi-stage trials |
| title_sort |
some recommendations for multi-arm multi-stage trials |
| description |
Multi-arm multi-stage designs can improve the efficiency of the drug-development process by evaluating multiple experimental arms against a common control within one trial. This reduces the number of patients required compared to a series of trials testing each experimental arm separately against control. By allowing for multiple stages experimental treatments can be eliminated early from the study if they are unlikely to be significantly better than control. Using the TAILoR trial as a motivating example, we explore a broad range of statistical issues related to multi-arm multi-stage trials including a comparison of different ways to power a multi-arm multi-stage trial; choosing the allocation ratio to the control group compared to other experimental arms; the consequences of adding additional experimental arms during a multi-arm multi-stage trial, and how one might control the type-I error rate when this is necessary; and modifying the stopping boundaries of a multi-arm multi-stage design to account for unknown variance in the treatment outcome. Multi-arm multi-stage trials represent a large financial investment, and so considering their design carefully is important to ensure efficiency and that they have a good chance of succeeding. |
| publisher |
SAGE Publications |
| publishDate |
2012 |
| url |
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4843088/ |
| _version_ |
1613570299625734144 |