ArticleBMC medical research methodology2020
Including non-concurrent control patients in the analysis of platform trials: is it worth it?
Article in BMC medical research methodology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers, 1 of them a synthesis that pooled it.
What it found
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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
26 citing papers in PubMed, 1 synthesis or guideline 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
- A diagnostic phase III/IV seamless design to investigate the diagnostic accuracy and clinical effectiveness using the example of HEDOS and HEDOS II.Statistical methods in medical research · 2024Trial
- On the Inclusion of Non-Concurrent Controls in Platform Trials With an Interim Analysis.Statistics in medicine · 2026Article
- Clinical Trial Simulation: Planning With the OCTAVE Framework, Implementation and Validation Principles.Statistics in medicine · 2026Article
- Randomization in the age of platform trials: unexplored challenges and some potential solutions.BMC medical research methodology · 2025Review
- Statistical Modeling to Adjust for Time Trends in Adaptive Platform Trials Utilizing Non-Concurrent Controls.Biometrical journal. Biometrische Zeitschrift · 2025Article
- Operating characteristics of unequal allocation ratios in platform trials with the staggered addition of drugs using binary endpoints.Contemporary clinical trials communications · 2025Article
- Identification and Estimation of Causal Effects Using Non-Concurrent Controls in Platform Trials.Statistics in medicine · 2025Article
- Review article: Primer for clinical researchers on innovative trial designs for emergency medicine.Emergency medicine Australasia : EMA · 2025Review
- A Preplanned Multi-Stage Platform Trial for Discovering Multiple Superior Treatments With Control of FWER and Power.Biometrical journal. Biometrische Zeitschrift · 2025Article
- Why and how should we simulate platform trials? Learnings from EU-PEARL.BMC medical research methodology · 2025Article
- Evaluation of the Fill-it-up-design to use historical control data in randomized clinical trials with two arm parallel group design.BMC medical research methodology · 2024Article
- The impact of heterogeneity on the analysis of platform trials with normally distributed outcomes.BMC medical research methodology · 2024Article
- Treatment Comparisons in Adaptive Platform Trials Adjusting for Temporal Drift.Statistics in biopharmaceutical research · 2024Article
- Improved efficiency for cross-arm comparisons via platform designs.Biostatistics (Oxford, England) · 2023Article
- Article
- Borrowing Concurrent Information from Non-Concurrent Control to Enhance Statistical Efficiency in Platform Trials.Current oncology (Toronto, Ont.) · 2023Article
- On the use of historical estimates.Statistical papers (Berlin, Germany) · 2023Article
- Estimands and Complex Innovative Designs.Clinical pharmacology and therapeutics · 2022Review
- Commentary: Two approaches to analyze platform trials incorporating non-concurrent controls with a common assumption.Clinical trials (London, England) · 2022Article
Corrections and comments
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Authors and funding
2 authors.
Funding
Abstract
backgroundPlatform trials allow adding new experimental treatments to an on-going trial. This feature is attractive to practitioners due to improved efficiency. Nevertheless, the operating characteristics of a trial that adds arms have not been well-studied. One controversy is whether just the concurrent control data (i.e. of patients who are recruited after a new arm is added) should be used in the analysis of the newly added treatment(s), or all control data (i.e. non-concurrent and concurrent).
methodsWe investigate the benefits and drawbacks of using non-concurrent control data within a two-stage setting. We perform simulation studies to explore the impact of a linear and a step trend on the inference of the trial. We compare several analysis approaches when one includes all the control data or only concurrent control data in the analysis of the newly added treatment.
resultsWhen there is a positive trend and all the control data are used, the marginal power of rejecting the corresponding hypothesis and the type one error rate can be higher than the nominal value. A model-based approach adjusting for a stage effect is equivalent to using concurrent control data; an adjustment with a linear term may not guarantee valid inference when there is a non-linear trend.
conclusionsIf strict error rate control is required then non-concurrent control data should not be used; otherwise it may be beneficial if the trend is sufficiently small. On the other hand, the root mean squared error of the estimated treatment effect can be improved through using non-concurrent control data.
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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.