[Federal Register Volume 77, Number 206 (Wednesday, October 24, 2012)]
[Notices]
[Pages 65002-65004]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2012-26214]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

National Institutes of Health


Best Pharmaceuticals for Children Act (BPCA) Priority List of 
Needs in Pediatric Therapeutics

AGENCY: National Institutes of Health, the Eunice Kennedy Shriver 
National Institute of Child Health and Human Development.

ACTION: Notice.

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SUMMARY: The National Institutes of Health (NIH) hereby announces the 
Best Pharmaceuticals for Children Act (BPCA) Priority List of Needs in 
Pediatric Therapeutics for 2012. The BPCA seeks to improve the level of 
information on the safe and effective use of pharmaceuticals used to 
treat children. It requires that the NIH identify the drugs of highest 
priority for study in pediatric populations and publish a list of 
drugs/needs in pediatric therapeutics. This notice fulfills the 
requirement to publish that list.

SUPPLEMENTARY INFORMATION: The pediatric medical community, the public 
health community, and government agencies have recognized multiple gaps 
in knowledge regarding the use of therapeutics in children, including 
the correct dose, appropriate indications, side effects, and safety 
concerns of pharmaceuticals in the short- and long-term. These gaps 
have frequently resulted in inadequate labeling for pediatric use and 
in widespread off-label use of prescription drugs in children. Off-
label use of a drug substantially limits the ability to gain clinical 
information of the drug product, such as appropriate dosing of a drug, 
changes in drug metabolism and response during growth and development, 
and important short- and long-term effects. Contributing factors to 
extensive off-label product use include limited access to patient 
populations for study, lack of knowledge related to the ethical conduct 
of clinical trials in

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children, the absence of sufficient evidence-based information about 
medication use in children, and a general lack of long-term safety data 
on the medications that are used. All of these factors contribute to 
the lack of adequately collected pharmacokinetic, pharmacodynamic, 
safety, and efficacy data in children and can increase a child's risk 
for unknown and/or adverse effects.
    The Eunice Kennedy Shriver National Institute of Child Health and 
Human Development (NICHD), the Food and Drug Administration (FDA), 
other federal agencies, and various non-profit and commercial 
organizations have taken steps to address the knowledge gaps that exist 
in pediatric therapeutics.

The 2002 BPCA Legislation

    The initial BPCA legislation reauthorized an incentive program for 
on-patent drugs that met certain criteria that were first authorized in 
the FDA Modernization Act (FDAMA). The BPCA also contains provisions 
for off-patent drugs and general support for pediatric product 
development that were not included in the FDAMA. The legislation, as it 
applies to the NIH, authorizes a research program through the 
Department of Health and Human Services (HHS), with implementation 
through the NIH, specifically by the NICHD. The NICHD is responsible 
for the development of: (1) A priority list of needs in pediatric 
therapeutics, in consultation with the FDA and experts in pediatrics; 
(2) sponsorship of relevant pediatric clinical trials; and 3) 
submission of resulting clinical trial data to FDA for pediatric 
labeling changes.

The Updated BPCA Legislation of 2007

    Title V of Public Law 110-85, the Best Pharmaceuticals for Children 
Act of 2007, was enacted on September 27, 2007, as part of the Food and 
Drug Administration Amendments Act of 2007. This legislation, which 
reauthorizes the BPCA (Section 409I of the Public Health Service Act), 
extends the 6-month patent exclusivity provision for currently on-
patent drugs being studied for pediatric use, and also extends and 
expands the NIH research program that was established in the earlier 
law. The priority list procedure was revised to emphasize knowledge 
gaps in therapeutic areas in contrast to knowledge gaps about specific 
drug products.

Update on BPCA Prioritization

    The BPCA requires that the NIH, in consultation with the Food and 
Drug Administration and experts in pediatric research, identify the 
drugs and therapeutic areas of highest priority for study in pediatric 
populations. Part of fulfilling the NIH's authority and responsibility 
outlined in the BPCA legislation is to establish a program for 
pediatric drug testing and development and to publish a list of drugs/
needs in pediatric therapeutics. The BPCA Priority List consists of key 
therapeutic needs in the medical treatment of children and adolescents; 
it is organized by therapeutic area, which can be a group of 
conditions, a subgroup of the population, or a setting of care. The 
first priority list of off-patent drugs needing further study under the 
2002 BPCA legislation was published in January 2003 in the Federal 
Register (FR Vol. 68, No. 13; Tuesday, January 21, 2003: 2789-2790). 
The most recent priority list was published April 1, 2011; all Federal 
Register notices can be found on the BPCA Web site: http://bpca.nichd.nih.gov/prioritization/status.cfm. NIH is required by BPCA 
to update the priority list every three years. This publication serves 
as an update to the BPCA priority list of needs in pediatric 
therapeutics.
    The Obstetric and Pediatric Pharmacology Branch of the NICHD has 
developed a prioritization process for determination of the needs in 
pediatric therapeutics. There are two main phases in the prioritization 
process. Phase I entails identifying therapeutic areas, which are 
general categories of conditions, diseases, settings of care, or 
populations with multiple therapeutic needs. Phase II involves 
determining more specific pediatric needs, including research 
associated with a particular drug, biologic, or device. Please visit 
the BPCA Web site for more details (http://bpca.nichd.nih.gov/prioritization/priority_list.cfm). Factors incorporated in the process 
include the following:
    [dec221] Availability of information concerning the safe and 
effective use of a drug in the pediatric population and the need for 
additional information;
    [dec221] Potential health benefits in the pediatric population 
resulting from new studies;
    [dec221] Possible need for reformulation of existing products;
    [dec221] Therapeutic gaps in pediatrics that may include 
developmental pharmacology, pharmacogenetic determinants of drug 
response, metabolism of drugs and biologics in children, and pediatric 
clinical trials;
[dec221] Particular pediatric diseases, disorders, or conditions where 
more complex knowledge and testing of therapeutics, including drugs and 
biologics, may be beneficial in pediatric populations; and
[dec221] The adequacy of necessary infrastructure to conduct pediatric 
pharmacological research, including research networks and trained 
pediatric investigators.
    The NICHD evaluates the current list of needs in pediatric 
therapeutics regularly to determine target areas for the coming 
calendar year. The NICHD sponsored the BPCA Annual Prioritization 
Meeting, held December 9-10, 2011, with stakeholders from the NIH, the 
FDA, and the American Academy of Pediatrics (AAP), as well as other 
pediatric organizations, societies, and patient advocates. The meeting 
allowed all stakeholders to review the present progress from ongoing 
research, to discuss lessons learned since the implementation of the 
BPCA legislation, and to discuss the proposed therapeutic areas from 
the 2011 recommendations for future study under the BPCA. Meeting 
minutes can be found on the BPCA Web site: http://bpca.nichd.nih.gov/prioritization/meeting_summary.cfm.
    Below is an updated list of therapeutic areas and drugs that have 
been prioritized for study since the inception of the BPCA, which 
includes new areas of prioritization from the 2010 outreach 
nominations, recommendations from the 2011 working groups, and a 
summary of the NICHD's plans and progress in all of these areas. The 
NICHD also solicits input from the pediatric medical community on 
additional gaps in pediatric therapeutics for future consideration. All 
nominations should be submitted to Dr. Perdita Taylor-Zapata at the 
contact information below.

Priority List of Needs in Pediatric Therapeutics 2012

    In accordance with the BPCA legislation, the list outlines priority 
needs in pediatric therapeutics for multiple therapeutic areas listed 
below. The complete list can be found on the BPCA Web site at the 
following address: http://bpca.nichd.nih.gov.
[dec221] Table 1: Infectious Disease Priorities
[dec221] Table 2: Cardiovascular Disease Priorities
[dec221] Table 3: Respiratory Disease Priorities
[dec221] Table 4: Intensive Care Priorities
[dec221] Table 5: Bio-defense Research Priorities
[dec221] Table 6: Pediatric Cancer Priorities
[dec221] Table 7: Psychiatric Disorder Priorities
[dec221] Table 8: Neurological Disease Priorities
[dec221] Table 9: Neonatal Research Priorities
[dec221] Table 10: Adolescent Research Priorities

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[dec221] Table 11: Hematologic Disease Priorities
[dec221] Table 12: Endocrine Disease Priorities and Diseases with 
Limited Alternative Therapies
[dec221] Table 13: Dermatologic Disease Priorities
[dec221] Table 14: Gastrointestinal Disease Priorities
[dec221] Table 15: Renal Disease Priorities
[dec221] Table 16: Rheumatologic Disease Priorities
[dec221] Table 17: Special Considerations.

FOR FURTHER INFORMATION CONTACT: Dr. Perdita Taylor-Zapata via email at 
[email protected]; by phone at 301-496-9584; or by fax at 301-480-
2897.

    Dated: October 17, 2012.
Francis S. Collins,
Director, National Institutes of Health.
[FR Doc. 2012-26214 Filed 10-23-12; 8:45 am]
BILLING CODE 4140-01-P