03-1250. 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 annually until the Act expires on October 1, 2007.

DATES:

The list is effective upon publication.

FOR FURTHER INFORMATION CONTACT:

Dr. Donald Mattison, National Institute of Child Health and Human Development, 6100 Executive Boulevard, Room 4B-100, Rockville, MD 20892, 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, Pub. L. 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 initial list, the NIH consulted with the FDA, the American Academy of Pediatrics, the United States Pharmacopoeia and other experts in pediatric research. A preliminary list of off-patent 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.

Following is the list of drugs for which pediatric studies are most urgently needed:

Azithromycin

Baclofen

Bumetanide

Dobutamine

Dopamine

Furosemide

Heparin

Lithium

Lorazepam

Rifampin

Sodium Nitroprusside

Spironolactone

Dated: January 9, 2003.

Ruth L. Kirschstein,

Deputy Director, NIH.