Literature DB >> 31115078

To add or not to add a new treatment arm to a multiarm study: A decision-theoretic framework.

Kim May Lee1, James Wason1,2, Nigel Stallard3.   

Abstract

Multiarm clinical trials, which compare several experimental treatments against control, are frequently recommended due to their efficiency gain. In practise, 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 on-going 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 works 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 multiarm studies, we define utility in two different ways; one for a trial that aims to maximise 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.
© 2019 The Authors. Statistics in Medicine Published by John Wiley & Sons Ltd.

Entities:  

Keywords:  adding-arm; disjunctive power; multiarm; number of rejected hypotheses

Mesh:

Year:  2019        PMID: 31115078      PMCID: PMC6619445          DOI: 10.1002/sim.8194

Source DB:  PubMed          Journal:  Stat Med        ISSN: 0277-6715            Impact factor:   2.373


  30 in total

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3.  One-sided sequential stopping boundaries for clinical trials: a decision-theoretic approach.

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Journal:  Biometrics       Date:  1988-03       Impact factor: 2.571

4.  To add or not to add a new treatment arm to a multiarm study: A decision-theoretic framework.

Authors:  Kim May Lee; James Wason; Nigel Stallard
Journal:  Stat Med       Date:  2019-05-21       Impact factor: 2.373

5.  Flexible analytical methods for adding a treatment arm mid-study to an ongoing clinical trial.

Authors:  Jordan J Elm; Yuko Y Palesch; Gary G Koch; Vanessa Hinson; Bernard Ravina; Wenle Zhao
Journal:  J Biopharm Stat       Date:  2012       Impact factor: 1.051

6.  MIDAS: a practical Bayesian design for platform trials with molecularly targeted agents.

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Journal:  Stat Med       Date:  2016-04-25       Impact factor: 2.373

7.  A comparison of methods for constructing confidence intervals after phase II/III clinical trials.

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8.  Identifying combined design and analysis procedures in two-stage trials with a binary end point.

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Review 9.  Adding a treatment arm to an ongoing clinical trial: a review of methodology and practice.

Authors:  Dena R Cohen; Susan Todd; Walter M Gregory; Julia M Brown
Journal:  Trials       Date:  2015-04-22       Impact factor: 2.279

Review 10.  Correcting for multiple-testing in multi-arm trials: is it necessary and is it done?

Authors:  James M S Wason; Lynne Stecher; Adrian P Mander
Journal:  Trials       Date:  2014-09-17       Impact factor: 2.279

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  4 in total

1.  To add or not to add a new treatment arm to a multiarm study: A decision-theoretic framework.

Authors:  Kim May Lee; James Wason; Nigel Stallard
Journal:  Stat Med       Date:  2019-05-21       Impact factor: 2.373

2.  Including non-concurrent control patients in the analysis of platform trials: is it worth it?

Authors:  Kim May Lee; James Wason
Journal:  BMC Med Res Methodol       Date:  2020-06-24       Impact factor: 4.615

3.  Statistical consideration when adding new arms to ongoing clinical trials: the potentials and the caveats.

Authors:  Kim May Lee; Louise C Brown; Thomas Jaki; Nigel Stallard; James Wason
Journal:  Trials       Date:  2021-03-10       Impact factor: 2.279

4.  Uptake of the multi-arm multi-stage (MAMS) adaptive platform approach: a trial-registry review of late-phase randomised clinical trials.

Authors:  Nurulamin M Noor; Sharon B Love; Talia Isaacs; Richard Kaplan; Mahesh K B Parmar; Matthew R Sydes
Journal:  BMJ Open       Date:  2022-03-10       Impact factor: 2.692

  4 in total

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