ReviewBritish journal of clinical pharmacology2025
A review on the role of extrapolation as basis for paediatric marketing authorization applications of medicines in the EU.
Review in British journal of clinical pharmacology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
What it found
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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
3 citing papers in PubMed.
- Strategies for pediatric dose derivation from population pharmacokinetic models.Journal of pharmacokinetics and pharmacodynamics · 2026Article
- Utilization of Child-Appropriate Medicines: Use of Pediatric Use Marketing Authorisation (PUMA) Products in Croatia.Journal of clinical medicine · 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
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
8 authors.
Funding
Abstract
aimsFor new medicines, drug companies obtain regulatory approval on the strategy to generate evidence in the paediatric population, which can be supported by extrapolation of evidence obtained in a reference population. This study investigated whether paediatric marketing authorization applications (PMAAs) supported by extrapolation based on exposure-matching were more successful-i.e. approval of the targeted paediatric population-and efficient-i.e. duration of the drug development-compared to PMAAs not supported by extrapolation.
methodsData was extracted from completed paediatric investigation plans (PIPs), associated drug labels and public assessment reports published on the European Medicines Agency website. Assessment reports were evaluated to assess whether PMAAs were supported by extrapolation based on exposure-matching. Wilcoxon rank-sum tests were used to compare PMAAs supported and not supported by extrapolation based on exposure-matching for outcomes of interest.
resultsExposure-matching supported the benefit/risk assessment of 39.6% of the PMAAs. Targeted and approved minimum age of the paediatric population were comparable for PMAAs where extrapolation based on exposure-matching supported the benefit/risk assessment (2.0 vs. 2.0 years, P-value = .72), but not for PMAAs not supported by extrapolation (0.2 years vs. 0.5 years, P-value = .05). Completion of drug development was 5.4 years vs. 4.3 years (P = .04) in PMAAs supported by extrapolation based on exposure-matching compared to those not supported by extrapolation, respectively.
conclusionsPMAAs supported by extrapolation based on exposure-matching succeeded more often in obtaining marketing approval in the targeted paediatric population than PMAAs not supported by exposure-matching, but were also less efficient.
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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.