04-3179. List of Drugs for Which Pediatric Studies Are Needed

ACTION:

Notice.

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 [e-mail], telephone [phone] (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.

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