ArticleClinical pharmacology and therapeutics2021
Benefit-Risk Assessment of Off-Label Drug Use in Children: The Bravo Framework.
Article in Clinical pharmacology and therapeutics, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 33 papers, 3 of them syntheses that pooled it.
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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.
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Who cites it
33 citing papers in PubMed, 3 syntheses or guidelines pooled it, 56 citations in OpenAlex.
- Off-Label Medicine Use in Pediatric Anesthesia-Recommendations of the World Federation of Societies of Anaesthesiologists (WFSA) Paediatric Committee.Paediatric anaesthesia · 2026Guideline
- Effects of repetitive transcranial magnetic stimulation in children and young people with psychiatric disorders: a systematic review.European child & adolescent psychiatry · 2025Pooled it
- Guideline for the management of pediatric off-label use of drugs in China (2021).BMC pediatrics · 2022Guideline
- Stewardship Challenges, PK/PD Evidence, and Prescribing Appropriateness for Recently Approved Antibiotics in Pediatric Practice.Antibiotics (Basel, Switzerland) · 2026Review
- Promises, Pitfalls, and Paths Forward for the Pediatric Research Equity Act.JAMA health forum · 2026Article
- Adverse drug events from paediatric use of extemporaneously compounded medicines in community pharmacy settings: a scoping review.BMJ paediatrics open · 2026Article
- Pediatric studies and labeling additions required by the U.S. FDA for novel drugs approved from 2011 to 2023: A retrospective cohort study.PLoS medicine · 2025Article
- Off-label prescribing in Italian pediatric practice: insights from a nationwide survey.European journal of pediatrics · 2025Article
- Paediatric rare diseases: Can large language models assist off-label prescribing?British journal of clinical pharmacology · 2025Article
- Lack of children in public medical imaging data points to growing age bias in biomedical AI.Research square · 2025Article
- Off-label use of Avastin in Pakistan: Lessons from the 2023 Punjab outbreak.Journal of public health research · 2025Article
- Blueprint for clinical N-of-1 strategies with off-label precision treatments in monogenic epilepsies.Orphanet journal of rare diseases · 2025Review
- Lack of children in public medical imaging data points to growing age bias in biomedical AI.medRxiv : the preprint server for health sciences · 2025Article
- A review on the role of extrapolation as basis for paediatric marketing authorization applications of medicines in the EU.British journal of clinical pharmacology · 2025Review
- A User-Driven Framework for Dose Selection in Pregnancy: Proof of Concept for Sertraline.Clinical pharmacology and therapeutics · 2025Article
- Development of a novel tool for individual treatment trials in mucopolysaccharidosis.Journal of inherited metabolic disease · 2025Article
- Digital monitoring of medication safety in children: an investigation of ADR signalling techniques in Malaysia.BMC medical informatics and decision making · 2024Article
- Oral Gels as an Alternative to Liquid Pediatric Suspensions Compounded from Commercial Tablets.Pharmaceutics · 2024Article
- Article
- The real-world safety of oseltamivir and baloxavir marboxil in children: a disproportionality analysis of the FDA adverse event reporting system.Frontiers in pharmacology · 2024Article
Corrections and comments
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Authors and funding
11 authors at 9 institutions in 4 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
A drug is granted a license for use after a thorough assessment of risks and benefits based on high-quality scientific proof of its efficacy and safety. Many drugs that are relevant to children are not licensed for use in this population implying that a thorough assessment of risks and benefits in the pediatric population has not been made at all, implying a negative risk-benefit balance in children, or implying insufficient information to establish the risk-benefit balance. Use of drugs without positive assessment of risks and benefits exposes children to potential lack of efficacy, unknown toxicity, and harm. To aid guideline committees and individual prescribers, we here present a tutorial of the Benefit and Risk Assessment for Off-label use (BRAvO) decision framework. This pragmatic framework offers a structured assessment of benefits and risks of off-label drug use, including a clinical pharmacological based approach to age-appropriate dose selection. As proof of concept and to illustrate the practical use, we have applied the framework to assess benefits and risks of off-label use of ondansetron for gastroenteritis-induced nausea and vomiting. The framework could also guide decisions on off-label use in other special populations (e.g., pregnant women, elderly, obese, or critically ill patients) where off-label drug use is frequent, thereby contributing to effective and safe pharmacotherapy.
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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.