Skip to Content


List of Drugs for Which Pediatric Studies Are Needed

Document Details

Information about this document as published in the Federal Register.

Document Statistics
Document page views are updated periodically throughout the day and are cumulative counts for this document including its time on Public Inspection. Counts are subject to sampling, reprocessing and revision (up or down) throughout the day.
Published Document

This document has been published in the Federal Register. Use the PDF linked in the document sidebar for the official electronic format.

Start Preamble




The National Institutes of Health (NIH) is providing notice of a “List of Drugs for Which Pediatric Studies Are Needed.” The NIH developed the list in consultation with the Food and Drug Administration (FDA) and pediatric experts, as mandated by the Best Pharmaceuticals for Children Act (BPCA). This list prioritizes certain drugs most in need of study for use by children to ensure their safety and efficacy. The NIH will update the list at least annually until the Act expires on October 1, 2007.


The list is effective upon publication.

Start Further Info


Dr. Anne Zajicek, National Institute of Child Health and Human Development, 6100 Executive Boulevard, Suite 4B-11, Bethesda, MD 20892-7510, e-mail, Start Printed Page 7244telephone 301-435-6865 (not a toll-free number).

End Further Info End Preamble Start Supplemental Information


The NIH is providing notice of a “List of Drugs for Which Pediatric Studies Are Needed,” as authorized under section 3, Public Law 107-109 (42 U.S.C. 409I). On January 4, 2002, President Bush signed into law the Best Pharmaceuticals for Children Act (BPCA). The BPCA mandates that not later than one year after the date of enactment, the NIH in consultation with the FDA and experts in pediatric research shall develop, prioritize, and publish an annual list of certain approved drugs for which pediatric studies are needed. For inclusion on the list, an approved drug must meet the following criteria: (1) There is an approved application under section 505(j) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 355(j)); (2) there is a submitted application that could be approved under the criteria of section 505(j) of the Federal Food, Drug, and Cosmetic Act; (3) there is no patent protection or market exclusivity protection under the Federal Food, Drug, and Cosmetic Act; or (4) there is a referral for inclusion on the list under section 505A(d)(4)(c); and additional studies are needed to assess the safety and effectiveness of the use of the drug in the pediatric population. The BPCA further stipulates that in developing and prioritizing the list, the NIH shall consider for each drug on the list: (1) The availability of information concerning the safe and effective use of the drug in the pediatric population; (2) whether additional information is needed; (3) whether new pediatric studies concerning the drug may produce health benefits in the pediatric population; and (4) whether reformulation of the drug is necessary. In developing this list, the NIH consulted with the FDA, the American Academy of Pediatrics, and other experts in pediatric research and practice. A preliminary list of drugs was drafted and categorized as a function of indication and use. The drugs were than prioritized based on frequency of use in the pediatric population, severity of the condition being treated, and potential for providing a health benefit in the pediatric population.

The following are the drugs newly added to the list for which pediatric studies are most urgently needed and their indications for use:






Drugs that were previously listed as urgently needing studies, their indications for use, and their current status, are described in the table.

Drug needing pediatric studyIndications for pediatric useStatus
LorazepamSedation in the Intensive Care Unit1
Treatment of status epilepticus1
NitroprussideReduction of blood pressure1
BaclofenOral treatment of spasticity of cerebral palsy1
AzithromycinPrevention of bronchopulmonary dysplasia in neonates colonized with U. urealyticum1
Treatment of Chlamydia pneumonia, prevention of Chlamydia conjunctivitis and pneumonia2
LithiumTreatment of mania in bipolar disorder1
Ampicillin/sulbactamPediatric infections2
IsofluraneMaintenance of general anesthesia2
MeropenemPediatric infections2
MetoclopramideGastroesophageal reflux2
Piperacillin/tazobactamPediatric infections2
RifampinStaphylococcus endocarditis2
CNS shunt infections2
Lindane2nd line treatment of scabies2
DobutamineIncrease cardiac output3
DopamineIncrease cardiac output3
1 Drug labeled for use in children.
> Status: 1 = Contract being developed; 2 = Written Request being developed; 3 = Drug undergoing extensive review by NIH and FDA.
Start Signature

Dated: February 6, 2004.

Elias A. Zerhouni,

Director, National Institutes of Health.

End Signature End Supplemental Information

[FR Doc. 04-3179 Filed 2-12-04; 8:45 am]