Response adaptive intervention allocation in stepped-wedge cluster randomized trials.
Stat Med
; 41(6): 1081-1099, 2022 03 15.
Article
em En
| MEDLINE
| ID: mdl-35064595
ABSTRACT
BACKGROUND:
Stepped-wedge cluster randomized trial (SW-CRT) designs are often used when there is a desire to provide an intervention to all enrolled clusters, because of a belief that it will be effective. However, given there should be equipoise at trial commencement, there has been discussion around whether a pre-trial decision to provide the intervention to all clusters is appropriate. In pharmaceutical drug development, a solution to a similar desire to provide more patients with an effective treatment is to use a response adaptive (RA) design.METHODS:
We introduce a way in which RA design could be incorporated in an SW-CRT, permitting modification of the intervention allocation during the trial. The proposed framework explicitly permits a balance to be sought between power and patient benefit considerations. A simulation study evaluates the methodology.RESULTS:
In one scenario, for one particular RA design, the proportion of cluster-periods spent in the intervention condition was observed to increase from 32.2% to 67.9% as the intervention effect was increased. A cost of this was a 6.2% power drop compared to a design that maximized power by fixing the proportion of time in the intervention condition at 45.0%, regardless of the intervention effect.CONCLUSIONS:
An RA approach may be most applicable to settings for which the intervention has substantial individual or societal benefit considerations, potentially in combination with notable safety concerns. In such a setting, the proposed methodology may routinely provide the desired adaptability of the roll-out speed, with only a small cost to the study's power.Palavras-chave
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MEDLINE
Assunto principal:
Projetos de Pesquisa
Idioma:
En
Ano de publicação:
2022
Tipo de documento:
Article