ReviewJournal of clinical pharmacology2018
Innovative Study Designs Optimizing Clinical Pharmacology Research in Infants and Children.
Review in Journal of clinical pharmacology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 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
17 citing papers in PubMed.
- Randomized controlled trial data for successful new drug application for rare diseases in the United States.Orphanet journal of rare diseases · 2023Trial
- Equitable access to monoclonal antibodies targeting parasites.Trends in parasitology · 2026Review
- Application of a Stochastic Simulation-estimation Approach to Optimize Pharmacokinetic Study Design in the Context of Paediatric Extrapolation: A Tool to Support Decision-making by Drug Sponsors and Regulators.Pharmaceutical research · 2026Article
- Qualitative analysis of pharmacogenetic applications in pediatric clinical trials registered on ClinicalTrials.gov.Frontiers in pharmacology · 2026Article
- Practical Pharmacokinetic-Pharmacodynamic Models in Oncology.Pharmaceutics · 2025Review
- Use of lorazepam for analgosedation during mechanical ventilation in pediatric intensive care.Frontiers in medicine · 2025Article
- Advances in pediatric acute kidney injury pharmacology and nutrition: a report from the 26th Acute Disease Quality Initiative (ADQI) consensus conference.Pediatric nephrology (Berlin, Germany) · 2024Article
- Early brain activity: Translations between bedside and laboratory.Progress in neurobiology · 2022Review
- Improving clinical paediatric research and learning from COVID-19: recommendations by the Conect4Children expert advice group.Pediatric research · 2022Article
- Review of Scavenged Sampling for Sustainable Therapeutic Drug Monitoring: Do More With Less.Therapeutic drug monitoring · 2022Review
- Pregabalin Population Pharmacokinetic and Exposure-Response Analyses for Focal Onset Seizures in Children (4-16 years) and Adults, to Support Dose Recommendations in Children.Clinical pharmacology and therapeutics · 2021Article
- Clinical pharmacology and dosing regimen optimization of neonatal opioid withdrawal syndrome treatments.Clinical and translational science · 2021Review
- Drug Safety in Translational Paediatric Research: Practical Points to Consider for Paediatric Safety Profiling and Protocol Development: A Scoping Review.Pharmaceutics · 2021Article
- Article
- Pharmacometric Evaluation of Umbilical Cord Blood Concentration-Based Early Initiation of Treatment in Methadone-Exposed Preterm Neonates.Children (Basel, Switzerland) · 2021Article
- Methodologic Progress Note: Opportunistic Sampling for Pharmacology Studies in Hospitalized Children.Journal of hospital medicine · 2021Article
- Pediatric Clinical Endpoint and Pharmacodynamic Biomarkers: Limitations and Opportunities.Paediatric drugs · 2020Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
Abstract
Almost half of recent pediatric trials failed to achieve labeling indications, in large part because of inadequate study design. Therefore, innovative study methods are crucial to optimizing trial design while also reducing the potential harms inherent with drug investigation. Several methods exist to optimize the amount of pharmacokinetic data collected from the smallest possible volume and with the fewest number of procedures, including the use of opportunistic and sparse sampling, alternative and noninvasive matrices, and microvolume assays. In addition, large research networks using master protocols promote collaboration, reduce regulatory burden, and increase trial efficiency for both early- and late-phase trials. Large pragmatic trials that leverage electronic health records can capitalize on central management strategies to reduce costs, enroll patients with rare diseases on a large scale, and augment study generalizability. Further, trial efficiency and safety can be optimized through Bayesian adaptive techniques that permit planned protocol changes based on analyses of prior and accumulated data. In addition to these trial design features, advances in modeling and simulation have paved the way for systems-based and physiologically based models that individualize pediatric dosing recommendations and support drug approval. Last, given the low prevalence of many pediatric diseases, collecting deidentified genetic and clinical data on a large scale is a potentially transformative way to augment clinical pharmacology research in children.
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.