[Federal Register Volume 69, Number 30 (Friday, February 13, 2004)]
[Notices]
[Pages 7243-7244]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 04-3179]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

National Institutes of Health


List of Drugs for Which Pediatric Studies Are Needed

ACTION: Notice.

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SUMMARY: 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.

DATES: The list is effective upon publication.

FOR FURTHER INFORMATION CONTACT: Dr. Anne Zajicek, National Institute 
of Child Health and Human Development, 6100 Executive Boulevard, Suite 
4B-11, Bethesda, MD 20892-7510, e-mail 
[email protected],

[[Page 7244]]

telephone 301-435-6865 (not a toll-free number).

SUPPLEMENTARY 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:

Ampicillin--infections;
Ketamine--sedation;
Vincristine--malignancies;
Dactinomycin--malignancies;
Metolazone--diuresis.

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

------------------------------------------------------------------------
                                   Indications for pediatric
  Drug needing pediatric study                use               Status
------------------------------------------------------------------------
Lorazepam.......................  Sedation in the Intensive           1
                                   Care Unit.
                                  Treatment of status                 1
                                   epilepticus.
Nitroprusside...................  Reduction of blood                  1
                                   pressure.
Baclofen........................  Oral treatment of                   1
                                   spasticity of cerebral
                                   palsy.
Azithromycin....................  Prevention of                       1
                                   bronchopulmonary
                                   dysplasia in neonates
                                   colonized with U.
                                   urealyticum.
                                  Treatment of Chlamydia              2
                                   pneumonia, prevention of
                                   Chlamydia conjunctivitis
                                   and pneumonia.
Lithium.........................  Treatment of mania in               1
                                   bipolar disorder.
Ampicillin/sulbactam............  Pediatric infections......          2
Diazoxide.......................  Hypoglycemia..............          2
Isoflurane......................  Maintenance of general              2
                                   anesthesia.
Meropenem.......................  Pediatric infections......          2
Metoclopramide..................  Gastroesophageal reflux...          2
Piperacillin/tazobactam.........  Pediatric infections......          2
Promethazine....................  Nausea/vomiting...........          2
Rifampin........................  Staphylococcus                      2
                                   endocarditis.
                                  CNS shunt infections......          2
Lindane.........................  2nd line treatment of               2
                                   scabies.
Heparin.........................  Anticoagulant.............        \1\
Bumetanide......................  Diuresis..................          3
Furosemide......................  Diuresis..................          3
Dobutamine......................  Increase cardiac output...          3
Dopamine........................  Increase cardiac output...          3
Spironolactone..................  Diuresis..................         3
------------------------------------------------------------------------
\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.


    Dated: February 6, 2004.
Elias A. Zerhouni,
Director, National Institutes of Health.
[FR Doc. 04-3179 Filed 2-12-04; 8:45 am]
BILLING CODE 4140-01-P