[Federal Register Volume 79, Number 164 (Monday, August 25, 2014)]
[Pages 50679-50680]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2014-20156]



National Institutes of Health

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

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

ACTION: Notice.


SUMMARY: The National Institutes of Health (NIH) hereby announces the 
Best Pharmaceuticals for Children Act (BPCA) Priority List of Needs in 
Pediatric Therapeutics for 2014. The BPCA, reauthorized in 2012 as part 
of the Food and Drug Safety and Innovation Act, 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.

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 
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 is working with the Food and Drug Administration (FDA), 
other federal agencies, and various nonprofit and commercial 
organizations to address the 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 FDA and 
experts in pediatric research, identify the drugs and therapeutic areas 
of highest priority for study in pediatric populations. The BPCA 
authorizes the NIH to establish a program for pediatric drug testing 
and development and to publish a list of drugs/needs in pediatric 
therapeutics. The BPCA program consists of a series of clinical trials 
in various therapeutic areas, which may be based on proposed pediatric 
study requests (PPSRs) submitted to the FDA and/or on written requests 
(WR) received from the FDA. The BPCA Priority List consists of key 
therapeutic needs in the medical treatment of children and adolescents 
that need further study. It is organized by therapeutic areas, which 
are general categories of conditions, diseases, settings of care, or 
populations with multiple therapeutic needs. 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 October 24, 2012; all Federal Register Notices can 
be found on the BPCA Web site: http://bpca.nichd.nih.gov/

[[Page 50680]]

status/Pages/status.aspx. The NIH is required by the 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 and Therapeutics Branch of 
the NICHD developed a prioritization process for the determination of 
the needs in pediatric therapeutics. There are two main phases in this 
process. 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 the pediatric research, 
general pediatric and subspecialty care, and patient advocacy groups to 
determine these therapeutic areas that need further study. At the 
beginning of every year, the NICHD revisits the current list of needs 
in pediatric therapeutics, prioritizes three therapeutic areas of 
interest for that calendar year, and develops working groups in the 
prioritized areas. Recommendations from the therapeutic area working 
groups are then presented to the NICHD and the FDA at the end of the 
calendar year at the Institute's annual BPCA meeting. Previous meeting 
minutes can be found on the BPCA Web site: http://bpca.nichd.nih.gov/prioritization/meetings/Pages/index.aspx.
    Phase II of the prioritization process entails an extensive review 
and ranking of all nominations received based on key criteria for 
prioritization, such as relevance, label gaps, affected population, and 
feasibility. Please visit the BPCA Web site for more details: (http://bpca.nichd.nih.gov/prioritization/priority_list/Pages/priority_list.aspx).
    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 (contact 
information below).

Priority List of Needs in Pediatric Therapeutics 2014

    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

    We would like for you to save the date of December 10, 2014, for 
our annual BPCA stakeholders meeting. The meeting will be held at the 
Natcher Auditorium on the NIH campus in Bethesda, Maryland, and will be 
streamed live via videocast for all non-local attendees.

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-

    Dated: August 18, 2014.
Francis S. Collins,
Director, National Institutes of Health.
[FR Doc. 2014-20156 Filed 8-22-14; 8:45 am]