Preliminary List of Drugs for Which Pediatric Studies Are Needed

ACTION:

Notice.

SUMMARY:

The National Institutes of Health (NIH) is providing notice of a “Preliminary 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 identifies 23 drugs that will be reviewed at a scientific meeting on October 25 and 26, 2004, in Bethesda, Maryland. At that time, the drugs will be discussed by the NIH, FDA, and a group of scientific experts to help identify those in most urgent need of study. It is anticipated that the final listing of drugs most in need of study for use by children to ensure their safety and efficacy will be selected from this preliminary listing and will be published in the Federal Register in January 2005. This will be the third annual list published by NIH. NIH will continue to 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. Tamar Lasky, National Institute of Child Health and Human Development, 6100 Executive Boulevard, Suite 5C01G, Bethesda, MD 20892-7510, e-mail <BestPharmaceuticals@mail.nih.gov>, telephone 301-594-8670 (not a toll-free number).

SUPPLEMENTARY INFORMATION:

The NIH is providing notice of a “Preliminary 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 pediatric populations; (2) whether additional information is needed; (3) whether new pediatric studies concerning the drug may produce health benefits in pediatric populations; and (4) whether reformulation of the drug is necessary. In developing this list, the NIH consulted with the FDA and 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 then 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 are the drugs and indications that will be reviewed at a scientific meeting on October 25 and 26, 2004, to select drugs and indications to add to the list for which pediatric studies are most urgently needed:

Acetylcysteine—acetaminophen poisoning

Aclometasone diproprionate cream—dermatitis

Acyclovir—herpetic infections

Albendazole—Giardia infection

Amantadine—influenza

Cefuroxime—infections in children with sickle cell anemia

Cephalexin—acute, oral infections

Chlorothiazide—hypertension

Clarithromycin—oral infections in dental patients

Clonidine—autism, attention deficit disorder

Cyclosporine—heart transplant patients

Desonide ointment—dermatitis

Ethambutol—tuberculosis

Flecainide—life threatening ventricular arrhythmias

Griseofulvin—tinea capitis

Hydrochlorothiazide—hypertension

Hydrocortisone valerate ointment and cream—dermatitis

Hydroxychloroquine—lupus

Ivermectin—scabies

Malathion—lice

Methadone—opiate addicted neonates

Rimantidine—influenza

Sulfasalazine—juvenile rheumatoid arthritis

Twelve additional drugs have been identified as having a sizeable number of studies published since 1990. These twelve drugs will receive extensive systematic literature reviews and meta-analysis to assess the safety and efficacy questions that remain unstudied. The twelve drugs are:

Amoxicillin

Amoxicillin clavulanate potassium

Cefixime

Chloral Hydrate

Dexamethasone

Epinephrine

Fluconazole

Mebendazole

Methylprednisolone

Prednisolone

Prednisone

Trimethoprim

The Foundation for the NIH, Inc., has referred four on-patent drugs to NIH. The feasibility and public health importance of studying these drugs will be reviewed at the scientific meeting on October 25 and 26, 2004. The four on-patent drugs that have been referred by the Foundation for the NIH, Inc., for consideration for study are:

Bupropion

Morphine

Sevelamer

Zonisamide

Individuals or organizations with comments, information, and current data regarding these drugs are requested to contact Dr. Tamar Lasky at NICHD (contact information above).