Blenkinsop, A;
Choodari-Oskooei, B;
(2019)
Multiarm, multistage randomized controlled trials with stopping boundaries for efficacy and lack of benefit: An update to nstage.
The Stata Journal
, 19
(4)
pp. 782-802.
10.1177/1536867X19893616.
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Abstract
Royston et al.’s (2011, Trials 12: 81) multiarm, multistage (MAMS) framework for the design of randomized clinical trials uses intermediate outcomes to drop research arms early for lack of benefit at interim stages, increasing efficiency in multiarm designs. However, additionally permitting interim evaluation of efficacy on the primary outcome measure could increase adoption of the design and result in practical benefits, such as savings in patient numbers and cost, should any efficacious arm be identified early. The nstage command, which aids the design of MAMS trial designs, has been updated to support this methodological extension. Operating characteristics can now be calculated for a design with binding or nonbinding stopping rules for lack of benefit and with efficacy stopping boundaries. An additional option searches for a design that strongly controls the familywise error rate at the desired level. We illustrate how the new features can be used to design a trial with the drop-down menu, using the original comparisons from the MAMS trial STAMPEDE as an example. The new functionality of the command will serve a broader range of trial objectives and increase efficiency of the design and thus increase uptake of the MAMS design in practice.
| Type: | Article |
|---|---|
| Title: | Multiarm, multistage randomized controlled trials with stopping boundaries for efficacy and lack of benefit: An update to nstage |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1177/1536867X19893616 |
| Publisher version: | https://doi.org/10.1177/1536867X19893616 |
| Language: | English |
| Additional information: | This article is distributed under the terms of the Creative Commons Attribution 4.0 License (https://creativecommons.org/licenses/by/4.0/) which permits any use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access pages (https://us.sagepub.com/en-us/nam/open-access-at-sage). |
| Keywords: | st0175 2, nstage, nstagemenu, multiarm multistage, familywise error rate, efficacy stopping boundaries, adaptive designs |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Inst of Clinical Trials and Methodology UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Inst of Clinical Trials and Methodology > MRC Clinical Trials Unit at UCL |
| URI: | https://discovery-pp.ucl.ac.uk/id/eprint/10083247 |
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