[Federal Register Volume 72, Number 59 (Wednesday, March 28, 2007)]
[Notices]
[Pages 14588-14589]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: E7-5673]


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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 
the ``Priority List of Drugs for Which Pediatric Studies Are Needed.'' 
The NIH develops the list in consultation with the Food and Drug 
Administration (FDA) and pediatric experts, as mandated by the Best 
Pharmaceuticals for Children Act. This list prioritizes certain drugs 
that are 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. Perdita Taylor-Zapata, National 
Institute of Child Health and Human Development (NICHD), 6100 Executive 
Boulevard, Suite 4A-01, Bethesda, MD 20892-7510, e-mail 
[email protected] or [email protected], telephone 
301-496-9584 (not a toll-free number).

SUPPLEMENTARY INFORMATION: The NIH is providing notice of the ``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

[[Page 14589]]

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. For this year, we are providing an update and a 
summary of the progress made by the prioritization working group from 
last year's notice until now, as well as a summary of the annual 
scientific prioritization meeting held with pediatric experts on 
December 5-6, 2006.
    We have updated the complete list of drugs, listed previously in 
the April 2006 Federal Register notice, and post it on the BPCA Web 
site http://bpca.nichd.nih.gov/index.cfm. We will continue to 
reevaluate this list throughout the year and will provide updates as 
required, based upon the reauthorization of the BPCA.
    In 2005, and with the suggestion of pediatric experts, NIH changed 
the listing system from a focus on individual off-patent drugs to a 
therapeutic class-based approach. Pediatric experts indicated that this 
approach will allow us to compare drugs within a therapeutic class (on 
and off patent) and give a broader description of the use of these 
drugs in children. This approach will also allow us to obtain focused 
expertise in therapeutic areas that will subsequently give us more 
insight into scientific gaps in treatments of the proposed conditions, 
as well as feasibility and study designs. Based on expert opinion 
obtained throughout the year as part of our regular outreach program, a 
preliminary list of conditions and suggested drugs was drafted and 
categorized for the 2007 prioritization based on this approach.
    The following are the conditions and the drugs discussed in our 
December 5-6, 2006 scientific meeting with experts in pediatric 
research: Infectious Diseases, with a focus on Methicillin-resistant 
Staphylococcus aureus (MRSA) infections; Pediatric Cancer, specifically 
Neuroblastoma; Neonatal Pain; and Asthma. The gaps in scientific 
knowledge as well as specific drugs thought to be effective for 
treatment in each of these conditions were then discussed based on off-
patent status, gaps in pediatric labeling, and the potential for 
providing a health benefit in the general pediatric population. We also 
provided updates on our current work in the areas of Pediatric 
Hypertension, Sickle Cell Anemia, and Attention Deficit Hyperactivity 
Disorder during this meeting. There was also a brief discussion on 
future areas of consideration, pending the reauthorization of the BPCA, 
that include topics such as childhood obesity, counter-terrorism 
research, and Fragile X Syndrome.
    Following below are the conditions and drugs we discussed in the 
December 5-6, 2006, scientific meeting with experts in pediatric 
research. We will add these conditions and drugs, and their indications 
for use, to the Priority List for 2007 for which pediatric studies are 
most urgently needed.

Treatment of Pediatric Cancers: 13-Cis-Retinoic Acid

    There is a need for information regarding the pharmacokinetics, 
safety, and efficacy of 13-Cis-Retinoic Acid in the treatment of 
neuroblastoma.

Treatment of Pediatric MRSA: Clindamycin, Tetracycline, Doxycycline and 
Trimethoprim-Sulfamethoxazole

    There is a need for further pharmacokinetic and safety data in the 
use of these drugs to treat children with MRSA infections.
    In addition to the above conditions and their associated drugs for 
consideration, the following are conditions that have been identified 
as needing improvements in the treatment strategies and/or assessments 
in pediatrics.

Pediatric Hypertension

    Data from the medical literature, clinical trials, and experience 
were presented and discussed by experts in the field of Pediatric 
Hypertension. Gaps in knowledge in this field include standardization 
of blood pressure measurements in children as well as the sequence of 
drugs for hypertension treatment in children.

Asthma

    Data from the medical literature, clinical trials, and experience 
were presented and discussed by experts in the field of Pediatric 
Asthma. Gaps in knowledge in this field include gaps in measuring 
efficacy and safety of treatments and drug delivery systems, especially 
in young children. There is also a need for the development of new 
tools to identify symptom measures, pulmonary function tests, 
biomarkers, and genetics.

Neonatal Research

    There are many areas in the field of neonatal medicine that can 
benefit from advances in neonatal research. Such gaps in research 
include areas such as determining feasibility of studying specific 
drugs in low-birth-weight infants based on current use; the development 
of novel study designs that take into account the small number of 
patients available due to either ethical limitations and/or feasibility 
issues; and the performance of clinical studies in areas such as the 
treatment of pain, neonatal seizures, and bronchopulmonary dysplasia, 
based on templates that are being developed by experts in research such 
as the working groups of the Newborn Drug Development Initiative.
    For the coming year, NICHD is planning a series of discussions with 
experts in the fields listed above and plans to identify and work with 
experts in these respective fields along with our continuing 
discussions with the other NIH Institutes and Centers. The goal of all 
of these discussions will be to specifically identify current gaps in 
scientific knowledge regarding research and treatment of these various 
pediatric conditions with the ultimate goal of determining future 
approved drugs for which pediatric studies are needed.

    Dated: March 15, 2007.
Raynard S. Kington,
Deputy Director, National Institutes of Health.
[FR Doc. E7-5673 Filed 3-27-07; 8:45 am]
BILLING CODE 4140-01-P