ArticleNature communications2022
The design and evaluation of hybrid controlled trials that leverage external data and randomization.
Article in Nature communications, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 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
20 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
- Statistical Guardrails for Hybrid-Controlled Trials: Robust to Confounding and Between-Study Heterogeneity.Therapeutic innovation & regulatory science · 2026Article
- Artificial intelligence-generated synthetic data for cancer research and clinical trials.Nature reviews. Cancer · 2026Review
- An E-value-Informed Sensitivity Analysis Framework for Hybrid Controlled Trials.medRxiv : the preprint server for health sciences · 2026Article
- Distress as a bridge to suicidality in schizophrenia spectrum disorders: a network-based intervention simulation study.Research square · 2026Article
- Leveraging External Data for Testing Experimental Therapies with Biomarker Interactions in Randomized Clinical Trials.Biometrika · 2026Article
- Real-World Studies and Randomized Controlled Trials: A Podcast Discussion of the Relative Strengths and Limitations of These Complementary Designs for Cancer Research.Oncology and therapy · 2025Article
- 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
- A Pharmacometrics-Informed Trial Simulation Framework for Optimizing Study Designs for Disease-Modifying Treatments in Rare Neurological Disorders.CPT: pharmacometrics & systems pharmacology · 2025Article
- Master Protocol Design With Hybrid Control for Efficient Early-Phase Trial Consolidation.JCO precision oncology · 2025Article
- Identifying appropriate external control datasets in support of future glioblastoma clinical trials leveraging external data.Neuro-oncology · 2025Article
- Advances and critical aspects in cancer treatment development using digital twins.Briefings in bioinformatics · 2025Review
- Keep asking: What do I want? What do I have? What do I do? : An approach for combining information to learn about a target population.European journal of epidemiology · 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
- Leveraging external control data in the design and analysis of neuro-oncology trials: Pearls and perils.Neuro-oncology · 2024Review
- Clinical development of new drugs for adults and children with cancer, 2010-2020.Journal of the National Cancer Institute · 2023Article
- Validation of Predictive Analyses for Interim Decisions in Clinical Trials.JCO precision oncology · 2023Review
- A causal roadmap for generating high-quality real-world evidence.Journal of clinical and translational science · 2023Review
- Novel Clinical Trial Designs in Neuro-Oncology.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2022Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors.
Funding
Abstract
Patient-level data from completed clinical studies or electronic health records can be used in the design and analysis of clinical trials. However, these external data can bias the evaluation of the experimental treatment when the statistical design does not appropriately account for potential confounders. In this work, we introduce a hybrid clinical trial design that combines the use of external control datasets and randomization to experimental and control arms, with the aim of producing efficient inference on the experimental treatment effects. Our analysis of the hybrid trial design includes scenarios where the distributions of measured and unmeasured prognostic patient characteristics differ across studies. Using simulations and datasets from clinical studies in extensive-stage small cell lung cancer and glioblastoma, we illustrate the potential advantages of hybrid trial designs compared to externally controlled trials and randomized trial designs.
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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.