ArticleNeuro-oncology2022
The use of external control data for predictions and futility interim analyses in clinical trials.
Article in Neuro-oncology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers.
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
26 citing papers in PubMed.
- Treatment Effect Reanalysis of the Randomized Individual Screening Trial of Innovative Glioblastoma Therapy in Newly Diagnosed Glioblastoma With External Control Data.Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2026Trial
- Impact of hydrocolloid dressings in the prevention of pressure ulcers in high-risk patients: a randomized controlled trial (PENFUP).Scientific reports · 2023Trial
- Prospectively shared control data across concurrent randomised clinical trials.European journal of cancer (Oxford, England : 1990) · 2023Trial
- Harmonized Estimation of Subgroup-Specific Treatment Effects in Randomized Trials: The Use of External Control Data.Journal of the Royal Statistical Society. Series B, Statistical methodology · 2026Article
- Leveraging External Data for Testing Experimental Therapies with Biomarker Interactions in Randomized Clinical Trials.Biometrika · 2026Article
- Identifying appropriate external control datasets in support of future glioblastoma clinical trials leveraging external data.Neuro-oncology · 2025Article
- Informative censoring in externally controlled clinical trials: a potential source of bias.ESMO open · 2025Article
- Design and conduct of theranostic trials in neuro-oncology: Challenges and opportunities.Neuro-oncology · 2024Review
- Linking clinical trial participants to their U.S. real-world data through tokenization: A practical guide.Contemporary clinical trials communications · 2024Article
- Examining external control arms in oncology: A scoping review of applications to date.Cancer medicine · 2024Article
- Leveraging external control data in the design and analysis of neuro-oncology trials: Pearls and perils.Neuro-oncology · 2024Review
- Improvement of an External Predictive Model Based on New Information Using a Synthetic Data Approach: Application to CADASIL.Neurology. Genetics · 2023Article
- Machine Learning in Clinical Trials: A Primer with Applications to Neurology.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2023Review
- Looking ahead in early-phase trial design to improve the drug development process: examples in oncology.BMC medical research methodology · 2023Article
- Accessible Data Collections for Improved Decision Making in Neuro-Oncology Clinical Trials.Clinical cancer research : an official journal of the American Association for Cancer Research · 2023Article
- Advances in Treatment of Isocitrate Dehydrogenase (IDH)-Wildtype Glioblastomas.Current neurology and neuroscience reports · 2023Review
- Dynamic use of historical controls in clinical trials for rare disease research: A re-evaluation of the MILES trial.Clinical trials (London, England) · 2023Article
- Clinical Trials in the Brain Tumour Population: Challenges and Strategies for the Future.Current oncology reports · 2023Review
- Comment: Advancing Clinical Trials with Novel Designs and Implementations.Statistical science : a review journal of the Institute of Mathematical Statistics · 2023Article
- Current drug development and trial designs in neuro-oncology: report from the first American Society of Clinical Oncology and Society for Neuro-Oncology Clinical Trials Conference.The Lancet. Oncology · 2023Review
Corrections and comments
- Commented on by
Authors and funding
7 authors.
Funding
Abstract
backgroundExternal control (EC) data from completed clinical trials and electronic health records can be valuable for the design and analysis of future clinical trials. We discuss the use of EC data for early stopping decisions in randomized clinical trials (RCTs).
methodsWe specify interim analyses (IAs) approaches for RCTs, which allow investigators to integrate external data into early futility stopping decisions. IAs utilize predictions based on early data from the RCT, possibly combined with external data. These predictions at IAs express the probability that the trial will generate significant evidence of positive treatment effects. The trial is discontinued if this predictive probability becomes smaller than a prespecified threshold. We quantify efficiency gains and risks associated with the integration of external data into interim decisions. We then analyze a collection of glioblastoma (GBM) data sets, to investigate if the balance of efficiency gains and risks justify the integration of external data into the IAs of future GBM RCTs.
resultsOur analyses illustrate the importance of accounting for potential differences between the distributions of prognostic variables in the RCT and in the external data to effectively leverage external data for interim decisions. Using GBM data sets, we estimate that the integration of external data increases the probability of early stopping of ineffective experimental treatments by up to 25% compared to IAs that do not leverage external data. Additionally, we observe a reduction of the probability of early discontinuation for effective experimental treatments, which improves the RCT power.
conclusionLeveraging external data for IAs in RCTs can support early stopping decisions and reduce the number of enrolled patients when the experimental treatment is ineffective.
Indexed as
Identifiers
What OpenQuestion holds
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.