ReviewClinical pharmacology and therapeutics2022
Off-Label, but on-Evidence? A Review of the Level of Evidence for Pediatric Pharmacotherapy.
Review in Clinical pharmacology and therapeutics, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 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
24 citing papers in PubMed.
- A Complex Intervention to Prevent Medication-Related Hospital Admissions.Deutsches Arzteblatt international · 2023Trial
- Off-label prescribing in pediatrics: between regulatory gaps and clinical evidence.Italian journal of pediatrics · 2026Article
- [Evidence-based drug information for children and adolescents in Germany-Are we moving in the right direction?]Bundesgesundheitsblatt, Gesundheitsforschung, Gesundheitsschutz · 2026Article
- Facilitators and barriers in obtaining informed consent for neonatal research: a scoping review.European journal of pediatrics · 2026Article
- Stable Overall Volume, Shifting Structure: Nine Years of Paediatric Outpatient Prescribing and Dispensing in Finland, 2017-2025.Clinical epidemiology · 2026Article
- A manufacturer-aware cross-system benchmark for pediatric pharmacovigilance signal concordance.Frontiers in pharmacology · 2026Article
- Paediatric rare diseases: Can large language models assist off-label prescribing?British journal of clinical pharmacology · 2025Article
- Identification and treatment of neurodevelopmental and mental disorders in boys and adults with Duchenne muscular dystrophy: a cohort study.Archives of disease in childhood · 2025Article
- Individualized and innovative gender healthcare for transgender and nonbinary youth.Nature reviews. Endocrinology · 2025Review
- Targeted Therapy for Complex Lymphatic Anomalies in Patients with Noonan Syndrome and Related Disorders.International journal of molecular sciences · 2025Article
- Off-label drug use in palliative medicine: Delphi study for the consensus of evidence-based treatment recommendations.Palliative medicine · 2025Article
- Prenatal and Early Childhood Exposure to Proton Pump Inhibitors and Antibiotics and the Risk of Childhood Cancer: A Nationwide Population-Based Cohort Study.Drug safety · 2025Article
- Trends in Proton Pump Inhibitor Use in Sweden by Sex and Age: A Drug Utilisation Study.Drug safety · 2025Article
- Narrative Review: Revised Principles and Practice Recommendations for Adolescent Substance Use Treatment and Policy.Journal of the American Academy of Child and Adolescent Psychiatry · 2025Review
- Analysis of off-label drug use and its influencing factors in pediatric patients undergoing otolaryngology, head, and neck surgery.Frontiers in pharmacology · 2025Article
- Next-generation pediatric care: nanotechnology-based and AI-driven solutions for cardiovascular, respiratory, and gastrointestinal disorders.World journal of pediatrics : WJP · 2025Review
- The Current and Potential Role of Pediatric Clinical Pharmacologists in Italy: A Review with Evidence Synthesis and Literature Analysis.Current medicinal chemistry · 2025Review
- Analysis of queries to a Swedish drug information centre identifies scientific knowledge gaps.Scientific reports · 2024Article
- Considerations in Paediatric and Adolescent Inflammatory Bowel Disease.Journal of Crohn's & colitis · 2024Review
- Off-Label Prescribing in Pediatric Population-Literature Review for 2012-2022.Pharmaceutics · 2023Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
13 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Many drugs are still prescribed off-label to the pediatric population. Although off-label drug use not supported by high level of evidence is potentially harmful, a comprehensive overview of the quality of the evidence pertaining off-label drug use in children is lacking. The Dutch Pediatric Formulary (DPF) provides best evidence-based dosing guidelines for drugs used in children. For each drug-indication-age group combination-together compiling one record-we scored the highest available level of evidence: labeled use, systematic review or meta-analysis, randomized controlled trial (RCT), comparative research, noncomparative research, or consensus-based expert opinions. For records based on selected guidelines, the original sources were not reviewed. These records were scored as guideline. A total of 774 drugs were analyzed comprising a total of 6,426 records. Of all off-label records (n = 2,718), 14% were supported by high quality evidence (4% meta-analysis or systematic reviews, 10% RCTs of high quality), 20% by comparative research, 14% by noncomparative research, 37% by consensus-based expert opinions, and 15% by selected guidelines. Fifty-eight percent of all records were authorized, increasing with age from 30% in preterm neonates (n = 110) up to 64% in adolescents (n = 1,630). Many have advocated that off-label use is only justified when supported by a high level of evidence. We show that this prerequisite would seriously limit available drug treatment for children as the underlying evidence is low across ages and drug classes. Our data identify the drugs and therapeutic areas for which evidence is clearly missing and could drive the global research agenda.
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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.