To add or not to add a new treatment arm to a multiarm study: A decision-theoretic framework.
Statistics in medicine
John Wiley & Sons Inc.
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Lee, K., Wason, J., & Stallard, N. (2019). To add or not to add a new treatment arm to a multiarm study: A decision-theoretic framework.. Statistics in medicine, 38 (18), 3305-3321. https://doi.org/10.1002/sim.8194
Multi-arm clinical trials, which compare several experimental treatments against control, are frequently recommended due to their efficiency gain. In practice, all potential treatments may not be ready to be tested in a phase II/III trial at the same time. It has become appealing to allow new treatment arms to be added into ongoing clinical trials using a ‘platform’ trial approach. To the best of our knowledge, many aspects of when to add arms to an existing trial have not been explored in the literature. Most work on adding arm(s) assume that a new arm is opened whenever a new treatment becomes available. This strategy may prolong the overall duration of a study or cause reduction in marginal power for each hypothesis if the adaptation is not well accommodated. Within a two-stage trial setting, we propose a decision-theoretic framework to investigate when to add or not to add a new treatment arm based on the observed stage one treatment responses. To account for different prospect of multi-arm studies, we define utility in two different ways; one for a trial that aims to maximize the number of rejected hypotheses; the other for a trial that would declare a success when at least one hypothesis is rejected from the study. Our framework shows that it is not always optimal to add a new treatment arm to an existing trial. We illustrate a case study by considering a completed trial on knee osteoarthritis.
Humans, Osteoarthritis, Knee, Clinical Protocols, Cryotherapy, Nerve Block, Multivariate Analysis, Decision Theory, Controlled Clinical Trials as Topic, Biostatistics, Adaptive Clinical Trials as Topic
External DOI: https://doi.org/10.1002/sim.8194
This record's URL: https://www.repository.cam.ac.uk/handle/1810/292079
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