[Federal Register Volume 85, Number 84 (Thursday, April 30, 2020)]
[Notices]
[Pages 23981-23982]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-09136]


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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 updates to the Best Pharmaceuticals for 
Children Act (BPCA) Program priorities for 2020-2021. 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, publish a list of 
drugs/needs in pediatric therapeutics, and fund studies in the 
prioritized areas. This notice will provide a brief update on the 
current progress of the BPCA Program and provide the current Priority 
List of Needs in Pediatric Therapeutics.

DATES: The next BPCA stakeholders meeting will take place on December 
14-15, 2020. More information will be provided on the BPCA website as 
it becomes available.

ADDRESSES: The complete Priority List of Needs in Pediatric 
Therapeutics 2020-2021 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 one-hundred and fifty (150) 
drugs and therapeutic areas, funded more than forty (40) clinical 
studies, and improved the labeling to date of eleven (11) drugs and two 
(2) devices 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 knowledge still 
remain regarding the use of therapeutics in children including the 
correct dosage, appropriate indications, side effects, and safety 
concerns 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 metabolic pathways 
and response during growth and development, identifying precision-based 
treatments (i.e., impact of genotype and phenotype of medication 
responses), and determining short- and long-term effects of 
medications. The mandate of the NIH BPCA Program is to identify 
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 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 Public Law 107-109, 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 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 BPCA authorizing legislation 
requires the NIH to hold meetings with stakeholders for input into the 
prioritization process and to update the priority list every three 
years.
    The most recent BPCA stakeholders meeting was held in Bethesda, 
Maryland on March 22, 2019. Please save the date for December 14-15, 
2020 for the next stakeholders meeting. 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. This Notice serves as an update to the BPCA priority 
list of needs in pediatric therapeutics.

Priority List of Needs in Pediatric Therapeutics 2020-2021

    In accordance with the BPCA legislation, the list outlines priority 
needs in pediatric therapeutics for multiple therapeutic areas listed 
below. The complete 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, 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

[[Page 23982]]

[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: April 22, 2020.
Lawrence A. Tabak,
Principal Deputy Director, National Institutes of Health.
[FR Doc. 2020-09136 Filed 4-29-20; 8:45 am]
 BILLING CODE 4140-01-P