ArticlePediatric research2017
Enhancing pediatric clinical trial feasibility through the use of Bayesian statistics.
Article in Pediatric research, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
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
12 citing papers in PubMed.
- A study on pharmacokinetics, pharmacodynamics and safety of lixisenatide in children and adolescents with type 2 diabetes.Pediatric diabetes · 2022Trial
- Drug Development in Pediatric Chronic Kidney Disease: A Review of Promising Treatments, Old Challenges, and New Strategies.Paediatric drugs · 2025Review
- Use of Bayesian approaches in oncology clinical trials: A cross-sectional analysis.Frontiers in pharmacology · 2025Article
- The Next Horizon of Drug Development: External Control Arms and Innovative Tools to Enrich Clinical Trial Data.Therapeutic innovation & regulatory science · 2024Review
- Analysis of the first ten years of FDA's rare pediatric disease priority review voucher program: designations, diseases, and drug development.Orphanet journal of rare diseases · 2024Review
- Bayesian adaptive design for pediatric clinical trials incorporating a community of prior beliefs.BMC medical research methodology · 2022Article
- Clindamycin adjunctive therapy for severeJAC-antimicrobial resistance · 2022Article
- Handling Poor Accrual in Pediatric Trials: A Simulation Study Using a Bayesian Approach.International journal of environmental research and public health · 2021Article
- Scientific considerations for global drug development.Science translational medicine · 2020Review
- The impact of the Orphan Drug Act on Food and Drug Administration-approved therapies for rare skin diseases and skin-related cancers.Journal of the American Academy of Dermatology · 2019Article
- Optimizing Clinical Trial Design to Maximize Evidence Generation in Pediatric HIV.Journal of acquired immune deficiency syndromes (1999) · 2018Article
- A SuperLearner-enforced approach for the estimation of treatment effect in pediatric trials.Digital healthArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
BackgroundPediatric clinical trials commonly experience recruitment challenges including limited number of patients and investigators, inclusion/exclusion criteria that further reduce the patient pool, and a competitive research landscape created by pediatric regulatory commitments. To overcome these challenges, innovative approaches are needed.MethodsThis article explores the use of Bayesian statistics to improve pediatric trial feasibility, using pediatric Type-2 diabetes as an example. Data for six therapies approved for adults were used to perform simulations to determine the impact on pediatric trial size.ResultsWhen the number of adult patients contributing to the simulation was assumed to be the same as the number of patients to be enrolled in the pediatric trial, the pediatric trial size was reduced by 75-78% when compared with a frequentist statistical approach, but was associated with a 34-45% false-positive rate. In subsequent simulations, greater control was exerted over the false-positive rate by decreasing the contribution of the adult data. A 30-33% reduction in trial size was achieved when false-positives were held to less than 10%.ConclusionReducing the trial size through the use of Bayesian statistics would facilitate completion of pediatric trials, enabling drugs to be labeled appropriately for children.
Indexed as
Identifiers
28700566What 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.