SynthesisEuropean journal of clinical pharmacology2011
Innovative study design for paediatric clinical trials.
Synthesis in European journal of clinical pharmacology, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 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
19 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Sequential analysis in neonatal research-systematic review.European journal of pediatrics · 2018Pooled it
- Safety and efficacy of pregabalin in adolescents with fibromyalgia: a randomized, double-blind, placebo-controlled trial and a 6-month open-label extension study.Pediatric rheumatology online journal · 2016Trial
- Preliminary experience using milnacipran in patients with juvenile fibromyalgia: lessons from a clinical trial program.Pediatric rheumatology online journal · 2015Trial
- Peritoneal dialysis does not adversely affect kidney function recovery after congenital heart surgery.The International journal of artificial organs · 2014Trial
- Consensus for the most suitable trial design to assess therapy for rare vascular malformations: a Delphi study.Scientific reports · 2026Article
- Innovative research methodologies in the EU regulatory framework: an analysis of EMA qualification procedures from a pediatric perspective.Frontiers in medicine · 2024Article
- Designs used in published therapeutic studies of rare superficial vascular anomalies: a systematic literature search.BMC medical research methodology · 2023Review
- Article
- Challenges in conducting paediatric trials with off-patent drugs.Contemporary clinical trials communications · 2021Article
- Navigating the Regulatory Landscape to Develop Pediatric Oncology Drugs: Expert Opinion Recommendations.Paediatric drugs · 2021Review
- Handling Poor Accrual in Pediatric Trials: A Simulation Study Using a Bayesian Approach.International journal of environmental research and public health · 2021Article
- The world is waiting, use sequential analysis and get us the evidence-based treatment we need for COVID-19.The Libyan journal of medicine · 2020Review
- Optimizing Clinical Trial Design to Maximize Evidence Generation in Pediatric HIV.Journal of acquired immune deficiency syndromes (1999) · 2018Article
- Validity of mobile electronic data capture in clinical studies: a pilot study in a pediatric population.BMC medical research methodology · 2017Article
- Optimizing Research to Speed Up Availability of Pediatric Antiretroviral Drugs and Formulations.Clinical infectious diseases : an official publication of the Infectious Diseases Society of America · 2017Review
- Model-Based Assessment of Alternative Study Designs in Pediatric Trials. Part I: Frequentist Approaches.CPT: pharmacometrics & systems pharmacology · 2016Article
- Successful private-public funding of paediatric medicines research: lessons from the EU programme to fund research into off-patent medicines.European journal of pediatrics · 2015Article
- Paediatric pharmacokinetics: key considerations.British journal of clinical pharmacology · 2015Review
- 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
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
purposeDespite representing a fundamental step towards the efficacious and safe utilisation of drugs in the paediatric population, the conduct of clinical trials in children poses several problems. Methodological issues and ethical concerns represent the major obstacles that have traditionally limited paediatric research. The randomised clinical trial, mainstay of clinical studies to assess the effects of any therapeutic intervention, shows some weaknesses that make it scarcely applicable to the paediatric population. Alternative and innovative approaches to clinical trial design in small populations have been developed in the last few decades with the aim of overcoming the limits related to small samples and to the acceptability of the trial.
methodsThis systematic review describes a variety of alternative designs to assess efficacy and safety in the paediatric population, including their applicability, advantages, disadvantages and real case examples. Approaches include sequential and adaptive designs, Bayesian methods and other innovative approaches.
resultsBy limiting the sample size and increasing acceptability, these methods may rationally limit the amount of experimentation in children to what is achievable, necessary and ethical
conclusionThanks to their features, these methods represent a reliable way of ultimately improving paediatric care.
Indexed as
Identifiers
21301825What 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.