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Multiply efficient trials: combining multiple trial arms and critical secondary questions increases trial efficiency

Abstract:
Designing explanatory trials to answer additional questions such as how and for whom treatments work should be a priority for improving trial efficiency. Multiple arm trials are also more efficient, as they provide more information about treatments over a shorter time span [1]. We studied the benefits of multiple trial arms and explanatory design using the Pacing, Graded Activity, and Cognitive Behaviour Therapy: A Randomised Evaluation (PACE) trial as an example. This trial studied three complex therapies and a specialised medical care comparison arm for the treatment of chronic fatigue syndrome. The study of how the treatments worked - mediation analysis - was built into the trial design. In terms of mediation, one interest was whether different treatments with some disparate components might vary in mechanism. In other words, might the effects of different treatments on a mediator (a paths or action theories) be associated with different mediator-outcome relationships (b paths or conceptual theories)?
Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.1186/s13063-017-1902-y

Authors

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Institution:
University of Oxford
Division:
MSD
Department:
Psychiatry
Oxford college:
St Cross College
Role:
Author


Publisher:
BioMed Central
Journal:
4th International Clinical Trials Methodology Conference (ICTMC 2017) More from this journal
Publication date:
2017-05-01
Acceptance date:
2016-11-10
DOI:
ISSN:
1745-6215


Pubs id:
pubs:734960
UUID:
uuid:ceac9018-35b2-4342-8527-2f963b18261a
Local pid:
pubs:734960
Source identifiers:
734960
Deposit date:
2017-11-01
ARK identifier:

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