ReviewThe Journal of pharmacy and pharmacology2017
How to optimise drug study design: pharmacokinetics and pharmacodynamics studies introduced to paediatricians.
Review in The Journal of pharmacy and pharmacology, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers, 1 of them a synthesis that pooled it.
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
12 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Animal Pharmacokinetic/Pharmacodynamic Studies (APPS) Reporting Guidelines.European journal of drug metabolism and pharmacokinetics · 2018Guideline
- Brief Report: Intranasal Ketamine in Adolescents and Young Adults with Autism Spectrum Disorder-Initial Results of a Randomized, Controlled, Crossover, Pilot Study.Journal of autism and developmental disorders · 2021Trial
- Empagliflozin in paediatric heart failure: model-based optimisation of a pharmacokinetic bridging study.Frontiers in medicine · 2025Article
- Use of lorazepam for analgosedation during mechanical ventilation in pediatric intensive care.Frontiers in medicine · 2025Article
- Advancing Precision Medicine: A Review of Innovative In Silico Approaches for Drug Development, Clinical Pharmacology and Personalized Healthcare.Pharmaceutics · 2024Review
- Pharmacokinetic-Pharmacodynamic Modeling of Midazolam in Pediatric Surgery.Pharmaceutics · 2023Article
- Dose rationale for gabapentin and tramadol in pediatric patients with chronic pain.Pharmacology research & perspectives · 2023Article
- Challenges and Pitfalls: Performing Clinical Trials in Patients With Congenital Diaphragmatic Hernia.Frontiers in pediatrics · 2022Review
- Age-Related Changes in Pediatric Physiology: Quantitative Analysis of Organ Weights and Blood Flows : Age-Related Changes in Pediatric Physiology.The AAPS journal · 2021Article
- Innovative Study Designs Optimizing Clinical Pharmacology Research in Infants and Children.Journal of clinical pharmacology · 2018Review
- Pharmacokinetic studies in children: recommendations for practice and research.Archives of disease in childhood · 2018Review
- Useful pharmacodynamic endpoints in children: selection, measurement, and next steps.Pediatric research · 2018Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectivesIn children, there is often lack of sufficient information concerning the pharmacokinetics (PK) and pharmacodynamics (PD) of a study drug to support dose selection and effective evaluation of efficacy in a randomised clinical trial (RCT). Therefore, one should consider the relevance of relatively small PKPD studies, which can provide the appropriate data to optimise the design of an RCT.
methodsBased on the experience of experts collaborating in the EU-funded Global Research in Paediatrics consortium, we aimed to inform clinician-scientists working with children on the design of investigator-initiated PKPD studies. KEY
findingsThe importance of the identification of an optimal dose for the paediatric population is explained, followed by the differences and similarities of dose-ranging and efficacy studies. The input of clinical pharmacologists with modelling expertise is essential for an efficient dose-finding study.
conclusionsThe emergence of new laboratory techniques and statistical tools allows for the collection and analysis of sparse and unbalanced data, enabling the implementation of (observational) PKPD studies in the paediatric clinic. Understanding of the principles and methods discussed in this study is essential to improve the quality of paediatric PKPD investigations, and to prevent the conduct of paediatric RCTs that fail because of inadequate dosing.
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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.