Evidence map›Paper›PMID 40066584›Full record

ArticleJournal of clinical pharmacology2025

Dose Ranging in Pediatric Drug Development Trials Submitted to the US FDA 2012-2020.

Carlton K K Lee, Eugenia Kwon, Francis G Green, Connor M I Kelley, Jennifer Pham, Gelareh Abulwerdi, Sherbet Samuels, Dionna J Green, Gilbert J Burckart

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in 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 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from 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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors.

Carlton K K LeeDepartments of Pediatrics and Pharmacy, Johns Hopkins Medical University, Baltimore, MD, USA.
Eugenia KwonAlfred E. Mann School of Pharmacy and Pharmaceutical Sciences, University of Southern California, Los Angeles, CA, USA.
Francis G GreenOffice of Clinical Pharmacology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA.
Connor M I KelleyHarrison College of Pharmacy, Auburn University, Auburn, AL, USA.
Jennifer PhamSkaggs School of Pharmacy and Pharmaceutical Sciences, University of California San Diego, San Diego, CA, USA.
Gelareh AbulwerdiOffice of Clinical Pharmacology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA.
Sherbet SamuelsOffice of Clinical Pharmacology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA.
Dionna J GreenOffice of Pediatric Therapeutics, Office of the Commissioner, US Food and Drug Administration, Silver Spring, MD, USA.
Gilbert J BurckartOffice of Clinical Pharmacology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA.ORCID 0000-0003-2005-9301

Funding

Intramural FDA HHS FD999999
6 · The paper itself

Abstract

Dose selection is a critical process within pediatric drug development and dose-ranging studies are integral to establish a reasonable dose. The objective of this analysis was to examine the dose-ranging trials utilized in pediatric drug development and to determine (1) the dose-ranging strategies that were used in all available pediatric dose-ranging studies, (2) the success of achieving pediatric labeling in those submissions to the US Food and Drug Administration, and (3) ethical aspects of providing a prospect of direct benefit to pediatric patients in dose-ranging studies. Of the 275 programs that previously surveyed pediatric drug development programs from 2012 to 2020, it was determined that dose-ranging studies were used for 97 (35.3%) programs. The three categorizations of these 97 programs included the parallel dose design (n = 66; 68%), the dose-escalation design (n = 18; 18.6%), and the crossover design (n = 13; 13.4%). In the 66 that used a parallel design, 41 of these products were approved for use in pediatric patients. In 13 out of the 41 drugs (31.7%) approved for pediatric use using parallel dose ranging, the lowest parallel dose (patient on the dose for the entire study) was lower than the approved dose. Dose ranging remains an important strategy for optimizing dosing, but ethical considerations and the need to optimize benefit for individual patients should drive decisions about dosing approaches in pediatric patients. The inclusion of adaptive designs is one possible approach to optimizing dose-ranging studies for pediatric patients.

Indexed as

Clinical Trials as TopicDrug DevelopmentChildDose-Response Relationship, DrugDrug ApprovalHumansPediatricsPharmaceutical PreparationsResearch DesignUnited StatesUnited States Food and Drug AdministrationPharmaceutical Preparationsclinical trials (CTR)dose rangingdrug developmentpediatrics (PED)pharmacology (PHA)

Identifiers

PMID40066584
PMCPMC12310368

What OpenQuestion holds

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Registered trials

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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.