ArticleTrials2021
Statistical consideration when adding new arms to ongoing clinical trials: the potentials and the caveats.
Article in Trials, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
18 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- The use of master protocols for efficient trial design to evaluate radiotherapy interventions: a systematic review.Journal of the National Cancer Institute · 2024Pooled it
- Design and analysis of umbrella trials: Where do we stand?Frontiers in medicine · 2022Pooled it
- Trial
- Randomization in the age of platform trials: unexplored challenges and some potential solutions.BMC medical research methodology · 2025Review
- Platform trials: key features, when to use them and methodological challenges.The Medical journal of Australia · 2025Article
- Statistical Modeling to Adjust for Time Trends in Adaptive Platform Trials Utilizing Non-Concurrent Controls.Biometrical journal. Biometrische Zeitschrift · 2025Article
- Article
- A Preplanned Multi-Stage Platform Trial for Discovering Multiple Superior Treatments With Control of FWER and Power.Biometrical journal. Biometrische Zeitschrift · 2025Article
- The impact of heterogeneity on the analysis of platform trials with normally distributed outcomes.BMC medical research methodology · 2024Article
- Optimal allocation strategies in platform trials with continuous endpoints.Statistical methods in medical research · 2024Article
- Improved efficiency for cross-arm comparisons via platform designs.Biostatistics (Oxford, England) · 2023Article
- Online error rate control for platform trials.Statistics in medicine · 2023Article
- Estimands and Complex Innovative Designs.Clinical pharmacology and therapeutics · 2022Review
- On model-based time trend adjustments in platform trials with non-concurrent controls.BMC medical research methodology · 2022Article
- Combining factorial and multi-arm multi-stage platform designs to evaluate multiple interventions efficiently.Clinical trials (London, England) · 2022Article
- Resting state electroencephalographic rhythms are affected by immediately preceding memory demands in cognitively unimpaired elderly and patients with mild cognitive impairment.Frontiers in aging neuroscience · 2022Article
- The design of a Bayesian adaptive clinical trial of tranexamic acid in severely injured children.Trials · 2021Article
- Moving forward in clinical research with master protocols.Contemporary clinical trials · 2021Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
Abstract
backgroundPlatform 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.
conclusionPlatform 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.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.