ArticleNeuro-oncology2019
To randomize, or not to randomize, that is the question: using data from prior clinical trials to guide future designs.
Article in Neuro-oncology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 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
14 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
- Accelerating discovery: Transformative clinical trial models in neuro-oncology.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2026Review
- Glioblastoma in adults: A Society for Neuro-Oncology (SNO) and European Society of Neuro-Oncology (EANO) consensus review on current management and future directions.Neuro-oncology · 2025Review
- Leveraging external control data in the design and analysis of neuro-oncology trials: Pearls and perils.Neuro-oncology · 2024Review
- Looking ahead in early-phase trial design to improve the drug development process: examples in oncology.BMC medical research methodology · 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
- Balancing Risk and Efficiency in Drug Development for Rare and Challenging Tumors: A New Paradigm for Glioma.Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2022Review
- The design and evaluation of hybrid controlled trials that leverage external data and randomization.Nature communications · 2022Article
- Novel Clinical Trial Designs in Neuro-Oncology.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2022Review
- Review
- Glioblastoma Clinical Trials: Current Landscape and Opportunities for Improvement.Clinical cancer research : an official journal of the American Association for Cancer Research · 2022Review
- Leveraging external data in the design and analysis of clinical trials in neuro-oncology.The Lancet. Oncology · 2021Review
- Review
- World Cancer Day 2021 - Perspectives in Pediatric and Adult Neuro-Oncology.Frontiers in oncology · 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
8 authors.
Funding
Abstract
backgroundUnderstanding the value of randomization is critical in designing clinical trials. Here, we introduce a simple and interpretable quantitative method to compare randomized designs versus single-arm designs using indication-specific parameters derived from the literature. We demonstrate the approach through application to phase II trials in newly diagnosed glioblastoma (ndGBM).
methodsWe abstracted data from prior ndGBM trials and derived relevant parameters to compare phase II randomized controlled trials (RCTs) and single-arm designs within a quantitative framework. Parameters included in our model were (i) the variability of the primary endpoint distributions across studies, (ii) potential for incorrectly specifying the single-arm trial's benchmark, and (iii) the hypothesized effect size. Strengths and weaknesses of RCT and single-arm designs were quantified by various metrics, including power and false positive error rates.
resultsWe applied our method to show that RCTs should be preferred to single-arm trials for evaluating overall survival in ndGBM patients based on parameters estimated from prior trials. More generally, for a given effect size, the utility of randomization compared with single-arm designs is highly dependent on (i) interstudy variability of the outcome distributions and (ii) potential errors in selecting standard of care efficacy estimates for single-arm studies.
conclusionsA quantitative framework using historical data is useful in understanding the utility of randomization in designing prospective trials. For typical phase II ndGBM trials using overall survival as the primary endpoint, randomization should be preferred over single-arm designs.
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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.