Evidence map›Paper›PMID 28804696›Full record

ArticleTherapeutic innovation & regulatory science2017

Neonatal Safety Information Reported to the FDA During Drug Development Studies.

Debbie Avant, Gerri Baer, Jason Moore, Panli Zheng, Alfred Sorbello, Ron Ariagno, Lynne Yao, Gilbert J Burckart, Jian Wang

Open access · bronzeAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Therapeutic innovation & regulatory science, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
1.2field-weighted citation impact, top 17% of its field
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

4 citing papers in PubMed, 7 citations in OpenAlex.

  1. Article
  2. Article
  3. Review
  4. Review
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 at 4 institutions in 1 country.

Debbie AvantOffice of Pediatric Therapeutics, Office of the Commissioner, US Food and Drug Administration, Silver Spring, MD, USA.
Gerri BaerOffice of Pediatric Therapeutics, Office of the Commissioner, US Food and Drug Administration, Silver Spring, MD, USA.
Jason MooreOffice of Clinical Pharmacology, Center for Drug Evaluation and Research (CDER), US Food and Drug Administration, Silver Spring, MD, USA.
Panli ZhengUniversity of North Carolina School of Pharmacy, Chapel Hill, NC, USA.
Alfred SorbelloOffice of Translational Sciences, US Food and Drug Administration, Silver Spring, MD, USA.
Ron AriagnoOffice of Pediatric Therapeutics, Office of the Commissioner, US Food and Drug Administration, Silver Spring, MD, USA.
Lynne YaoDivision of Pediatric and Maternal Health, Office of New Drugs, CDER, US Food and Drug Administration, Silver Spring, MD, USA.
Gilbert J BurckartOffice of Clinical Pharmacology, Center for Drug Evaluation and Research (CDER), US Food and Drug Administration, Silver Spring, MD, USA.
Jian WangOffice of Drug Evaluation IV, CDER, US Food and Drug Administration, Silver Spring, MD, USA.
United States Food and Drug Administration · USCenter for Drug Evaluation and Research · USStanford University · USUniversity of North Carolina at Chapel Hill · US

Funding

CLINICAL PHARMACOLOGY TRAINING PROGRAMT32GM008562 · NIGMS · THOMAS JEFFERSON UNIVERSITY · PI WALTER K KRAFT, Scott A. Waldman · 1995 to 2026
$10.2M
Intramural FDA HHS FD999999NIGMS NIH HHS T32 GM008562
6 · The paper itself

Abstract

backgroundRelatively few neonatal drug development studies have been conducted, but an increase is expected with the enactment of the Food and Drug Administration Safety and Innovation Act (FDASIA). Understanding the safety of drugs studied in neonates is complicated by the unique nature of the population and the level of illness. The objective of this study was to examine neonatal safety data submitted to the FDA in studies pursuant to the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) between 1998 and 2015.

methodsFDA databases were searched for BPCA and/or PREA studies that enrolled neonates. Studies that enrolled a minimum of 3 neonates were analyzed for the presence and content of neonatal safety data.

resultsThe analysis identified 40 drugs that were studied in 3 or more neonates. Of the 40 drugs, 36 drugs received a pediatric labeling change as a result of studies between 1998 and 2015, that included information from studies including neonates. Fourteen drugs were approved for use in neonates. Clinical trials for 20 of the drugs reported serious adverse events (SAEs) in neonates. The SAEs primarily involved cardiovascular events such as bradycardia and/or hypotension or laboratory abnormalities such as anemia, neutropenia, and electrolyte disturbances. Deaths were reported during studies of 9 drugs.

conclusionsOur analysis revealed that SAEs were reported in studies involving 20 of the 40 drugs evaluated in neonates, with deaths identified in 9 of those studies. Patients enrolled in studies were often critically ill, which complicated determination of whether an adverse event was drug-related. We conclude that the traditional means for collecting safety information in drug development trials needs to be adjusted for neonates and will require the collaboration of regulators, industry, and the clinical and research communities to establish appropriate definitions and reporting strategies for the neonatal population.

Indexed as

drug developmentneonatessafetyserious adverse eventsUS FDA

Identifiers

PMID28804696
PMCPMC5549935
OpenAlexW2727787101

What OpenQuestion holds

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Registered trials

None linked

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.