[Federal Register Volume 84, Number 78 (Tuesday, April 23, 2019)]
[Notices]
[Pages 16872-16874]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2019-08167]


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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, HHS.

ACTION: Notice.

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SUMMARY: The National Institutes of Health (NIH), Eunice Kennedy 
Shriver National Institute of Child Health and Human Development 
(NICHD) hereby announces the renewal of the Best Pharmaceuticals for 
Children Act (BPCA) Program. The Best Pharmaceuticals for Children Act 
(BPCA) seeks to improve the level of

[[Page 16873]]

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, publish a list of 
drugs/needs in pediatric therapeutics, and fund studies in the 
prioritized areas. This notice will provide a brief summary of recent 
changes in the legislation, a brief update on the current progress of 
the BPCA Program and provide the current Priority List of Needs in 
Pediatric Therapeutics.

ADDRESSES: The complete Priority List of Needs in Pediatric 
Therapeutics 2018-2019 can be found on the BPCA website at the 
following address: https://www.nichd.nih.gov/research/supported/bpca/activities.

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

SUPPLEMENTARY INFORMATION: 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 BPCA Program has 
been in existence since 2004 and is overseen by the Obstetric and 
Pediatric Pharmacology and Therapeutics Branch (OPPTB) of the NICHD. To 
date, the BPCA Program has prioritized over 150 drugs and therapeutic 
areas, funded more than 25 clinical studies, and improved the labeling 
to date of eight drugs and one device in the ongoing effort of 
advancing the knowledge of dosing, safety and effectiveness of 
medicines used in children. However, despite these and many other 
efforts, many gaps in our knowledge still remain 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 result in inadequate labeling and/
or wide-spread off-label use of prescription drugs in children. Off-
label use of a drug substantially limits the ability to obtain 
important clinical information for more generalized use of a drug 
product, such as characterizing changes in drug metabolism and response 
during growth and development, identifying precision-based responses 
(i.e., impact of genotype and phenotype of medication responses, the 
impact of obesity on dosing), and determining short- and long-term 
effects. The mandate of the NIH BPCA Program is to fill knowledge gaps 
that exist in pediatric therapeutics and to promote an increase in 
evidence-based data about medications used in children. Please see the 
BPCA website for more information: https://www.nichd.nih.gov/research/supported/bpca/about.

Update on the BPCA Legislation

    First authorized in 2002, the Best Pharmaceuticals for Children Act 
(BPCA) has been reauthorized as part of larger Food and Drug 
Administration (FDA) user fee legislation three additional times: 2007, 
2012, and now 2017. The overall mandate for the implementation of the 
research program at NIH has remained the same throughout, but with 
clarifications each time: To prioritize testing of pediatric 
therapeutics that do not have labeling for pediatric use, to sponsor 
clinical trials and other research to provide the necessary data, and 
to submit those data to the FDA to begin the process of obtaining label 
changes and provide clinicians with the appropriate information on 
appropriate pediatric use and dosing. In August of 2017, the BPCA 
legislation was reauthorized by Congress, which renewed the NIH BPCA 
Program for five years (the FDA portion of the program is permanently 
authorized). The new legislation also permits the NIH to prioritize 
research on the identification of biomarkers for pediatric diseases and 
conditions. In addition, a new provision specifically allows the NIH to 
post the data from the pediatric studies it funded on its public 
website when it submits the report to the FDA, as required for 
potential label changes.

Update on BPCA Prioritization

    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 has been published to the BPCA website; 
more information on the prioritization process, all BPCA priority 
lists, and all Federal Register Notices can be found on the BPCA 
website: https://www.nichd.nih.gov/research/supported/bpca/prioritizing-pediatric-therapies. The BPCA authorizing legislation 
requires the NIH to update the priority list every three years. This 
Notice serves as an update to the BPCA priority list of needs in 
pediatric therapeutics.
    Each year, the NICHD revisits the current list of needs in 
pediatric therapeutics and seeks input from experts in pediatric 
research and medicine to determine if previous needs still exist and if 
new areas of needs have developed.
    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. In 2017, the NIH BPCA Program focused on the following areas: 
Treatment options in Pediatric Hypertension, Biomarkers in Pediatric 
Research (various subspecialties), and Treatment strategies in several 
neonatal conditions (including Neonatal Opioid Withdrawal Syndrome, 
also known as Neonatal Abstinence Syndrome). Meeting minutes for 
workshops and lectures on the above topics can be found on the BPCA 
website https://www.nichd.nih.gov/research/supported/bpca/research-initiatives-collaborations.
    For 2018, the NIH BPCA Program's priorities have included: Heart 
failure in children, Kidney diseases, and Lactation (in particular, 
neonatal and infant medication exposure). The NICHD welcomes input from 
the pediatric medical community on additional gaps in pediatric 
therapeutics for future consideration. The most recent BPCA 
stakeholders meeting was held in Bethesda, Maryland on March 22, 2019. 
More information will be provided on the BPCA website as it becomes 
available. All inquiries should be submitted to Dr. Perdita Taylor-
Zapata at the contact information above.

Priority List of Needs in Pediatric Therapeutics 2018-2019

    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 website at the 
following address: https://www.nichd.nih.gov/research/supported/bpca/activities.

[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

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[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: April 17, 2019.
Francis S. Collins,
Director, National Institutes of Health.
[FR Doc. 2019-08167 Filed 4-22-19; 8:45 am]
BILLING CODE 4140-01-P