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Drug labeling and exposure in neonates
Matthew M Laughon1, Debbie Avant2, Nidhi Tripathi3
1Department of Pediatrics, University of North Carolina at Chapel Hill, Chapel Hill.
Insights
Pediatric legislation increased drug studies, but neonatal drug information remains scarce. Most studied drugs are rarely used in neonatal intensive care units (NICUs), highlighting a need for better neonatal drug research strategies.
Area of Science:
- Neonatal pharmacology
- Pediatric drug development
- Regulatory science
Background:
- Federal legislation has spurred pediatric drug studies, improving product labeling.
- Despite progress, about 50% of drug labels lack adequate pediatric information.
- Neonatal drug labeling is particularly limited due to study challenges and vulnerable populations.
Purpose of the Study:
- To assess the impact of recent legislation on neonatal drug studies.
- To quantify improvements in neonatal drug information within product labeling.
Main Methods:
- Identified neonatal drug studies from 1997-2010 linked to pediatric legislation using FDA data.
- Determined study publication status and resulting labeling changes.
- Analyzed drug usage in a cohort of 446,335 infants admitted to US neonatal intensive care units (NICUs) from 2005-2010.
Main Results:
- 28 drugs were studied in neonates, leading to 24 labeling changes; 46% established safety and efficacy.
- 13 of the 28 studied drugs were not used in NICUs, and 8 were used in fewer than 60 neonates.
- Ranitidine was the most frequently used studied drug in neonates within NICUs.
Conclusions:
- Legislation has yielded limited improvements in neonatal drug labeling.
- Many drugs studied in neonates are infrequently used in clinical practice.
- Novel strategies are required to enhance the study and use of safe, effective neonatal drugs.
Importance:
Federal legislation has led to a notable increase in pediatric studies submitted to the Food and Drug Administration (FDA), resulting in new pediatric information in product labeling. However, approximately 50% of drug labels still have insufficient information on safety, efficacy, or dosing in children. Neonatal information in labeling is even scarcer because neonates comprise a vulnerable subpopulation for which end-point development is lagging and studies are more challenging.
Objective:
To quantify progress made in neonatal studies and neonatal information in product labeling as a result of recent legislation.
Design, Setting, And Participants:
We identified a cohort of drug studies between 1997 and 2010 that included neonates as a result of pediatric legislation using information available on the FDA website. We determined what studies were published in the medical literature, the legislation responsible for the studies, and the resulting neonatal labeling changes. We then examined the use of these drugs in a cohort of neonates admitted to 290 neonatal intensive care units (NICUs) (the Pediatrix Data Warehouse) in the United States from 2005 to 2010.
Exposure:
Infants exposed to a drug studied in neonates as identified by the FDA website.
Main Outcomes And Measures:
Number of drug studies with neonates and rate of exposure per 1000 admissions among infants admitted to an NICU.
Results:
In a review of the FDA databases, we identified 28 drugs studied in neonates and 24 related labeling changes. Forty-one studies encompassed the 28 drugs, and 31 (76%) of these were published. Eleven (46%) of the 24 neonatal labeling changes established safety and effectiveness. In a review of a cohort of 446,335 hospitalized infants, we identified 399 drugs used and 1,525,739 drug exposures in the first 28 postnatal days. Thirteen (46%) of the 28 drugs studied in neonates were not used in NICUs; 8 (29%) were used in fewer than 60 neonates. Of the drugs studied, ranitidine was used most often (15,627 neonates, 35 exposures per 1000 admissions).
Conclusions And Relevance:
Few drug labeling changes made under pediatric legislation include neonates. Most drugs studied are either not used or rarely used in US NICUs. Strategies to increase the study of safe and effective drugs for neonates are needed.
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