Evidence map›Paper›PMID 27774353›Full record

ArticleTherapeutic innovation & regulatory science2016

Stratification, Hypothesis Testing, and Clinical Trial Simulation in Pediatric Drug Development.

Ann W McMahon, Kevin Watt, Jian Wang, Dionna Green, Ram Tiwari, Gilbert J Burckart

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Therapeutic innovation & regulatory science, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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

2 citing papers in PubMed.

  1. Article
  2. 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

6 authors.

Ann W McMahonOffice of Pediatric Therapeutics, Office of the Commissioner, Food and Drug Administration, Silver Spring, MD, USA.
Kevin WattDuke University Medical Center, Durham, NC, USA.
Jian WangOffice of Clinical Pharmacology, Office of Translational Sciences, Food and Drug Administration, Silver Spring, MD, USA.
Dionna GreenOffice of Clinical Pharmacology, Office of Translational Sciences, Food and Drug Administration, Silver Spring, MD, USA.
Ram TiwariOffice of Biostatistics, Office of Translational Sciences, Food and Drug Administration, Silver Spring, MD, USA.
Gilbert J BurckartOffice of Clinical Pharmacology, Office of Translational Sciences, Food and Drug Administration, Silver Spring, MD, USA.

Funding

Physiologically Based Pharmacokinetics in Critically Ill ChildrenK23HD075891 · NICHD · DUKE UNIVERSITY · PI WATT, KEVIN M · 2014 to 2018
$638k
Intramural FDA HHS FD999999NICHD NIH HHS K23 HD075891
6 · The paper itself

Abstract

backgroundPediatric drug development is plagued by small sample sizes, unvalidated clinical endpoints, and limited studies.

objectivesThe objective of this study was to determine whether age stratification within the pediatric population could be used to (1) assess response to a pharmacologic intervention and to (2) design future trials based upon published stratified disease data using clinical trial simulation (CTS).

methodsData available from the literature for Kawasaki disease (KD) was used in the model. Age-stratified CTS for a theoretical new drug was conducted.

resultsPopulation-specific differences due to age might affect trial success if not taken into account. CTS predicted inflammatory indices, and inclusion cutoff significantly altered the trial outcome. Finally, altered pharmacokinetics/pharmacodynamics in varying age groups of KD patients may alter drug exposure and response.

conclusionsIf assumptions regarding a pediatric disease process, such as KD, do not include age stratification with inclusion or response, then the wrong decision could result with regard to age-appropriateness or approval of a drug.

Indexed as

age groupsKawasaki diseasemodelingpharmacokinetics pharmacodynamics

Identifiers

PMID27774353
PMCPMC5069699

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.