[Federal Register Volume 80, Number 165 (Wednesday, August 26, 2015)]
[Notices]
[Pages 51827-51828]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2015-21155]


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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 (NICHD).

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 2015. The Best Pharmaceuticals for Children 
Act (BPCA) seeks to improve the level of information on the safe and 
effective use of pharmaceuticals used to treat children. The BPCA 
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.

ADDRESSES: All nominations of pediatric therapeutics for future 
consideration by NICHD should be submitted to Dr. Perdita Taylor-Zapata 
at [email protected].

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.

SUPPLEMENTARY INFORMATION: The pediatric medical community, the public 
health community, and government agencies have long recognized multiple 
gaps in knowledge regarding the use of therapeutics in children 
including the correct dosage, 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 wide-spread 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.
    The NICHD, the Food and Drug Administration (FDA), other federal 
agencies, and various non-profit and commercial organizations are 
taking steps to fill knowledge gaps that exist in pediatric 
therapeutics and to promote an increase in evidence-based data about 
medications used in children.

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. 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 information needs regarding 
drugs used to treat children. The drug development program consists of 
a series of clinical trials in various therapeutic areas, which may be 
based on proposed pediatric study requests (PPSRs) submitted to FDA 
and/or from written requests (WR) received from the FDA. The BPCA 
Priority List consists of key therapeutic needs in the medical 
treatment of children and adolescents identified for further study; 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 August 25, 2014; all Federal Register 
notices can be found on the BPCA Web site: http://bpca.nichd.nih.gov/prioritization/status.cfm. The BPCA legislation requires the NIH 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 and Therapeutics Branch of 
the Eunice Kennedy Shriver National Institute of Child Health and Human 
Development (NICHD), NIH, has developed a prioritization process for 
the determination of the needs in pediatric therapeutics. There are two 
main phases: Phase I of the prioritization process entails identifying 
therapeutic areas, which are general categories of conditions, 
diseases, settings of care, or populations with multiple therapeutic 
needs. The NICHD solicits input from experts in pediatric research, 
general pediatric and subspecialty care, organizations focused on 
specific conditions, and professional societies to determine these 
therapeutic areas that need further study. Each year, the NICHD 
revisits the current list of needs in pediatric therapeutics, 
prioritizes three therapeutic areas of interests for that calendar 
year, and develops working groups in the

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prioritized areas. Recommendations from these working groups are then 
presented to the NICHD and the FDA at the annual BPCA meeting. Previous 
meeting minutes can be found on the BPCA Web site: http://bpca.nichd.nih.gov/prioritization/meeting_summary.cfm. Phase II of the 
prioritization process includes an extensive review and ranking of all 
stakeholder nominations received based on key criteria for 
prioritization, such as relevance, gaps in labeling information, 
affected populations and feasibility. Please visit the BPCA Web site 
for more details (http://bpca.nichd.nih.gov/prioritization/priority_list.cfm).
    Below is an updated list of therapeutic areas and drugs that have 
been prioritized for study since the inception of the BPCA and a 
summary of the NICHD's plans and progress in all of these areas to 
date. The NICHD welcomes 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 
address in the ADDRESSES section of this notice.

Priority List of Needs in Pediatric Therapeutics 2015

    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.
    [ssquf] Table 1: Infectious Disease Priorities
    [ssquf] Table 2: Cardiovascular Disease Priorities
    [ssquf] Table 3: Respiratory Disease Priorities
    [ssquf] Table 4: Intensive Care Priorities
    [ssquf] Table 5: Bio-defense Research Priorities
    [ssquf] Table 6: Pediatric Cancer Priorities
    [ssquf] Table 7: Psychiatric Disorder Priorities
    [ssquf] Table 8: Neurological Disease Priorities
    [ssquf] Table 9: Neonatal Research Priorities
    [ssquf] Table 10: Adolescent Research Priorities
    [ssquf] Table 11: Hematologic Disease Priorities
    [ssquf] Table 12: Endocrine Disease Priorities and Diseases with 
Limited Alternative Therapies
    [ssquf] Table 13: Dermatologic Disease Priorities
    [ssquf] Table 14: Gastrointestinal Disease Priorities
    [ssquf] Table 15: Renal Disease Priorities
    [ssquf] Table 16: Rheumatologic Disease Priorities
    [ssquf] Table 17: Special Considerations.

    Dated: August 19, 2015.
Francis S. Collins, M.D.,
Director, National Institutes of Health.
[FR Doc. 2015-21155 Filed 8-25-15; 8:45 am]
BILLING CODE 4140-01-P