SynthesisBMC medical research methodology2026
Basket and umbrella trials in pediatric precision medicine: a systematic review of designs, opportunities, and challenges.
Synthesis in BMC medical research methodology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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.
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Who cites it
0 citing papers in PubMed.
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Corrections and comments
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Authors and funding
8 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundPersonalized medicine, driven by genomic insights, has catalyzed the emergence of innovative clinical trial designs such as basket and umbrella trials. These designs are particularly suited for evaluating targeted therapies in biomarker-defined subgroups and rare pediatric conditions where traditional trials face challenges of small sample sizes and disease heterogeneity.
objectivesThis systematic review aimed to characterize and synthesize the current literature on basket and umbrella trial designs in pediatric drug development, with a focus on their methodological, regulatory, and statistical aspects.
methodsA systematic search was conducted in the electronic databases PubMed, Scopus, and Web of Science to identify all literature related to basket and umbrella trials. A text mining analysis using unsupervised machine learning technique was performed with relevant articles to automatically identify the primary topics within publications on basket and umbrella trials. A systematic search of PubMed, Scopus, and Web of Science identified 1867 records. After screening and eligibility assessment, 28 studies were included in the final review. Topic modelling using Latent Dirichlet Allocation (LDA) was performed on 76 pertinent articles to identify dominant themes. Statistical convergence, topic coherence, and classification accuracy (> 85%) were validated.
resultsA systematic search of PubMed, Scopus, and Web of Science identified 1867 records. After screening and eligibility assessment, 28 studies were included in the final review. Basket trial designs were more prevalent than umbrella trials, particularly in early-phase oncology and rare disease research. Topic modelling using Latent Dirichlet Allocation (LDA) was performed on 76 relevant articles to identify dominant themes. Statistical convergence, topic coherence, and classification accuracy (> 85%) were validated.
conclusionsBasket and umbrella trials offer substantial advantages for pediatric drug development by increasing trial efficiency, enabling precision targeting, and supporting adaptive decision-making. Their success depends on robust statistical planning, careful use of Bayesian methods, and attention to regulatory guidance.
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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.