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Statistical consideration when adding new arms to ongoing clinical trials : the potentials and the caveats
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Lee, Kim May, Brown, Louise C., Jaki, Thomas, Stallard, Nigel and Wason, James (2021) Statistical consideration when adding new arms to ongoing clinical trials : the potentials and the caveats. Trials, 22 (1). 203. doi:10.1186/s13063-021-05150-7
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WRAP-Statistical-consideration-new-arms-clinical-trials-potentials-caveats-2021.pdf - Published Version - Requires a PDF viewer. Available under License Creative Commons Attribution 4.0. Download (402Kb) | Preview |
Official URL: http://dx.doi.org/10.1186/s13063-021-05150-7
Abstract
Background
Platform trials improve the efficiency of the drug development process through flexible features such as adding and dropping arms as evidence emerges. The benefits and practical challenges of implementing novel trial designs have been discussed widely in the literature, yet less consideration has been given to the statistical implications of adding arms.
Main
We explain different statistical considerations that arise from allowing new research interventions to be added in for ongoing studies. We present recent methodology development on addressing these issues and illustrate design and analysis approaches that might be enhanced to provide robust inference from platform trials. We also discuss the implication of changing the control arm, how patient eligibility for different arms may complicate the trial design and analysis, and how operational bias may arise when revealing some results of the trials. Lastly, we comment on the appropriateness and the application of platform trials in phase II and phase III settings, as well as publicly versus industry-funded trials.
Conclusion
Platform trials provide great opportunities for improving the efficiency of evaluating interventions. Although several statistical issues are present, there are a range of methods available that allow robust and efficient design and analysis of these trials.
| Item Type: | Journal Article | |||||||||
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| Subjects: | R Medicine > R Medicine (General) | |||||||||
| Divisions: | Faculty of Medicine > Warwick Medical School > Health Sciences Faculty of Medicine > Warwick Medical School > Health Sciences > Statistics and Epidemiology Faculty of Medicine > Warwick Medical School |
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| Library of Congress Subject Headings (LCSH): | Clinical trials , Clinical medicine -- Research -- Statistical methods | |||||||||
| Journal or Publication Title: | Trials | |||||||||
| Publisher: | Biomed Central | |||||||||
| ISSN: | 1745-6215 | |||||||||
| Official Date: | 10 March 2021 | |||||||||
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| Volume: | 22 | |||||||||
| Number: | 1 | |||||||||
| Article Number: | 203 | |||||||||
| DOI: | 10.1186/s13063-021-05150-7 | |||||||||
| Status: | Peer Reviewed | |||||||||
| Publication Status: | Published | |||||||||
| Access rights to Published version: | Open Access | |||||||||
| RIOXX Funder/Project Grant: |
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