ArticleJournal of clinical pharmacology2018
Primary Endpoints in Pediatric Efficacy Trials Submitted to the US FDA.
Article in Journal of clinical pharmacology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 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
12 citing papers in PubMed.
- A Review of Trials for Unsuccessful Pediatric Drug Development Programs Submitted to the US Food and Drug Administration 2015-2022.Therapeutic innovation & regulatory science · 2026Review
- Challenges, approaches and enablers: effectively triangulating towards dose selection in pediatric rare diseases.Journal of pharmacokinetics and pharmacodynamics · 2023Review
- Variation in Endpoints in FDA Medication Approvals: A Review of Acute and Preventive Migraine Medications.Neurology · 2023Review
- Assessing disease activity using the pediatric Crohn's disease activity index: Can we use subjective or objective parameters alone?World journal of gastroenterology · 2021Article
- FutureTox IV Workshop Summary: Predictive Toxicology for Healthy Children.Toxicological sciences : an official journal of the Society of Toxicology · 2021Review
- Combined Pediatric and Adult Trials Submitted to the US Food and Drug Administration 2012-2018.Clinical pharmacology and therapeutics · 2020Article
- Prevention and Treatment of Acute Graft-versus-Host Disease in Children, Adolescents, and Young Adults.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2020Review
- End Points, Collection, Processing, and Time: Four Key Elements to Consider When Planning for Use of Handheld Devices in a Drug Development Setting.Clinical pharmacology and therapeutics · 2020Article
- The Revolution in Pediatric Drug Development and Drug Use: Therapeutic Orphans No More.The journal of pediatric pharmacology and therapeutics : JPPT : the official journal of PPAG · 2020Article
- Developmental Pharmacodynamics and Modeling in Pediatric Drug Development.Journal of clinical pharmacology · 2019Review
- Paediatric extrapolation: the panacea for paediatric drug development?British journal of clinical pharmacology · 2019Article
- Surrogate Endpoints in Pediatric Studies Submitted to the US FDA.Clinical pharmacology and therapeutics · 2019Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
10 authors.
Funding
Abstract
The selection of appropriate endpoints in pediatric drug development trials is a critical aspect of trial design. Given the high pediatric trial failure rate, selecting optimal trial design elements, such as the primary efficacy endpoint, is essential to ensuring increased potential for trial success. The objectives of this study were to identify the primary efficacy endpoints measured in pediatric drug development trials submitted to the US Food and Drug Administration and to relate endpoint attributes to trial and label outcome. The analysis included pediatric pivotal efficacy studies submitted from September 2007 to July 2016 for which there was a corresponding adult trial for the same indication. Two hundred and thirty-four efficacy trials on 138 unique products studied in pediatric patients were assessed. The adult and pediatric endpoints were the same in 141 of the 234 trials (60.3%), and these trials succeeded in meeting their primary endpoint more often (122 of 141 [86.5%]) than when the adult and pediatric endpoints differed (57 of 93 [61.3%]; odds ratio, 4.03; 95%CI, 2.10-7.80). Trials that included both pediatric and adult patients succeeded more frequently than those trials that did not combine pediatric and adult patients (85 of 95 versus 94 of 139, respectively; odds ratio, 4.05; 95%CI, 1.94-9.31). No differences were observed in pediatric trial success between those using subjective and objective endpoints. Using the same endpoint in the pediatric trial as was measured in the corresponding adult trial and enrolling pediatric and adult patients in the same trial were attributes associated with trial success.
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What 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.