80 FR 51827 - Best Pharmaceuticals for Children Act (BPCA) Priority List of Needs in Pediatric Therapeutics
DEPARTMENT OF HEALTH AND HUMAN SERVICES
National Institutes of Health Federal Register Volume 80, Issue 165 (August 26, 2015)
National Institutes of Health
Federal Register Volume 80, Issue 165 (August 26, 2015)
| Page Range | 51827-51828 | |
| FR Document | 2015-21155 |
[Federal Register Volume 80, Number 165 (Wednesday, August 26, 2015)] [Notices] [Pages 51827-51828] From the Federal Register Online [www.thefederalregister.org] [FR Doc No: 2015-21155] ----------------------------------------------------------------------- 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. ----------------------------------------------------------------------- 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 [[Page 51828]] 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
![Federal Register / Vol. 80, No. 165 / Wednesday, August 26, 2015 / Notices 51827
DEPARTMENT OF HEALTH AND DEPARTMENT OF HEALTH AND an increase in evidence-based data
HUMAN SERVICES HUMAN SERVICES about medications used in children.
National Institutes of Health National Institutes of Health Update on BPCA Prioritization
The BPCA requires that the NIH, in
Center for Scientific Review; Notice of Best Pharmaceuticals for Children Act consultation with the Food and Drug
Closed Meetings (BPCA) Priority List of Needs in Administration and experts in pediatric
Pediatric Therapeutics research, identify the drugs and
Pursuant to section 10(d) of the
therapeutic areas of highest priority for
Federal Advisory Committee Act, as AGENCY: National Institutes of Health, study in pediatric populations. The
amended (5 U.S.C. App.), notice is The Eunice Kennedy Shriver National NIH’s authority and responsibility
hereby given of the following meetings. Institute of Child Health and Human
The meetings will be closed to the outlined in the BPCA legislation is to
Development (NICHD). establish a program for pediatric drug
public in accordance with the ACTION: Notice.
provisions set forth in sections testing and development and to publish
552b(c)(4) and 552b(c)(6), title 5 U.S.C., a list of information needs regarding
SUMMARY: The National Institutes of
as amended. The grant applications and drugs used to treat children. The drug
Health (NIH) hereby announces the Best
development program consists of a
the discussions could disclose Pharmaceuticals for Children Act
series of clinical trials in various
confidential trade secrets or commercial (BPCA) Priority List of Needs in therapeutic areas, which may be based
property such as patentable material, Pediatric Therapeutics for 2015. The on proposed pediatric study requests
and personal information concerning Best Pharmaceuticals for Children Act (PPSRs) submitted to FDA and/or from
individuals associated with the grant (BPCA) seeks to improve the level of written requests (WR) received from the
applications, the disclosure of which information on the safe and effective use FDA. The BPCA Priority List consists of
would constitute a clearly unwarranted of pharmaceuticals used to treat key therapeutic needs in the medical
invasion of personal privacy. children. The BPCA requires that the treatment of children and adolescents
Name of Committee: Center for Scientific NIH identify the drugs of highest identified for further study; it is
Review Special Emphasis Panel PAR14–262: priority for study in pediatric organized by therapeutic area, which
Weight and Sleep Outcomes After Bariatric populations, and publish a list of drugs/ can be a group of conditions, a subgroup
Surgery. needs in pediatric therapeutics. This
Date: September 17, 2015.
of the population, or a setting of care.
notice fulfills the requirement to The first priority list of off-patent drugs
Time: 2:00 p.m. to 5:00 p.m. publish that list.
Agenda: To review and evaluate grant needing further study under the 2002
applications. ADDRESSES: All nominations of pediatric BPCA legislation was published in
Place: National Institutes of Health, 6701 therapeutics for future consideration by January 2003 in the Federal Register
Rockledge Drive, Bethesda, MD 20892, NICHD should be submitted to Dr. (FR Vol. 68, No. 13; Tuesday, January
(Telephone Conference Call). Perdita Taylor-Zapata at taylorpe@ 21, 2003: 2789–2790). The most recent
Contact Person: Ellen K Schwartz, EDD., mail.nih.gov. priority list was published August 25,
Scientific Review Officer, Center for 2014; all Federal Register notices can be
Scientific Review, National Institutes of FOR FURTHER INFORMATION CONTACT: Dr.
Health, 6701 Rockledge Drive, Room 3144, Perdita Taylor-Zapata via email at found on the BPCA Web site: http://
MSC 7770, Bethesda, MD 20892, 301–828– taylorpe@mail.nih.gov; by phone at bpca.nichd.nih.gov/prioritization/
6146, schwarel@mail.nih.gov. 301–496–9584; or by fax at 301–480– status.cfm. The BPCA legislation
Name of Committee: Center for Scientific 2897. requires the NIH to update the priority
Review Special Emphasis Panel PAR Panel: list every three years. This publication
Targeting Temporal Dynamics of the Brain SUPPLEMENTARY INFORMATION: The serves as an update to the BPCA priority
Activity for the Treatment of Cognitive pediatric medical community, the list of needs in pediatric therapeutics.
Deficits. public health community, and The Obstetric and Pediatric
Date: September 29, 2015. government agencies have long Pharmacology and Therapeutics Branch
Time: 10:30 a.m. to 12:30 p.m. recognized multiple gaps in knowledge of the Eunice Kennedy Shriver National
Agenda: To review and evaluate grant regarding the use of therapeutics in Institute of Child Health and Human
applications. children including the correct dosage, Development (NICHD), NIH, has
Place: National Institutes of Health, 6701
appropriate indications, side effects, developed a prioritization process for
Rockledge Drive, Bethesda, MD 20892,
(Virtual Meeting). and safety concerns of pharmaceuticals the determination of the needs in
Contact Person: Wei-Qin Zhao, Ph.D., in the short- and long term. These gaps pediatric therapeutics. There are two
Scientific Review Officer, Center for have frequently resulted in inadequate main phases: Phase I of the
Scientific Review, National Institutes of labeling for pediatric use and in wide- prioritization process entails identifying
Health, 6701 Rockledge Drive, Room 5181 spread off-label use of prescription therapeutic areas, which are general
MSC 7846, Bethesda, MD 20892–7846, 301– drugs in children. Off-label use of a drug categories of conditions, diseases,
435–1236, zhaow@csr.nih.gov. substantially limits the ability to gain settings of care, or populations with
(Catalogue of Federal Domestic Assistance clinical information of the drug product multiple therapeutic needs. The NICHD
Program Nos. 93.306, Comparative Medicine; such as appropriate dosing of a drug, solicits input from experts in pediatric
93.333, Clinical Research, 93.306, 93.333, changes in drug metabolism and research, general pediatric and
93.337, 93.393–93.396, 93.837–93.844,
response during growth and subspecialty care, organizations focused
93.846–93.878, 93.892, 93.893, National
development, and important short- and on specific conditions, and professional
rmajette on DSK7SPTVN1PROD with NOTICES
Institutes of Health, HHS)
long-term effects. societies to determine these therapeutic
Dated: August 21, 2015. The NICHD, the Food and Drug areas that need further study. Each year,
Carolyn Baum, Administration (FDA), other federal the NICHD revisits the current list of
Program Analyst, Office of Federal Advisory agencies, and various non-profit and needs in pediatric therapeutics,
Committee Policy. commercial organizations are taking prioritizes three therapeutic areas of
[FR Doc. 2015–21153 Filed 8–25–15; 8:45 am] steps to fill knowledge gaps that exist in interests for that calendar year, and
BILLING CODE 4140–01–P pediatric therapeutics and to promote develops working groups in the
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![51828 Federal Register / Vol. 80, No. 165 / Wednesday, August 26, 2015 / Notices
prioritized areas. Recommendations D Table 17: Special Considerations. • Email: NIAC@hq.dhs.gov. Include
from these working groups are then Dated: August 19, 2015. the docket number in the subject line of
presented to the NICHD and the FDA at Francis S. Collins, M.D.,
the message.
the annual BPCA meeting. Previous • Fax: (703) 235–9707.
Director, National Institutes of Health. • Mail: Nancy Wong, National
meeting minutes can be found on the
[FR Doc. 2015–21155 Filed 8–25–15; 8:45 am] Protection and Programs Directorate,
BPCA Web site: http://
bpca.nichd.nih.gov/prioritization/ BILLING CODE 4140–01–P Department of Homeland Security, 245
meeting_summary.cfm. Phase II of the Murray Lane SW., Mail Stop 0612,
prioritization process includes an Washington, DC 20598–0607.
extensive review and ranking of all DEPARTMENT OF HOMELAND Instructions: All written submissions
stakeholder nominations received based SECURITY received must include the words
on key criteria for prioritization, such as ‘‘Department of Homeland Security’’
[Docket No. DHS–2015–0042]
relevance, gaps in labeling information, and the docket number for this action.
affected populations and feasibility. National Infrastructure Advisory Written comments received will be
Please visit the BPCA Web site for more Council posted without alteration at
details (http://bpca.nichd.nih.gov/ www.regulations.gov, including any
prioritization/priority_list.cfm). AGENCY: National Protection and personal information provided.
Below is an updated list of Programs Directorate, DHS. Docket: For access to the docket to
therapeutic areas and drugs that have ACTION: Committee Management; Notice read background documents or
been prioritized for study since the of an Open Federal Advisory Committee comments received by the National
inception of the BPCA and a summary Meeting. Infrastructure Advisory Council, go to
of the NICHD’s plans and progress in all www.regulations.gov. Enter ‘‘NIAC’’ in
of these areas to date. The NICHD SUMMARY: The National Infrastructure the search line and the Web site will list
welcomes input from the pediatric Advisory Council will meet Friday, all relevant documents for your review.
medical community on additional gaps September 11, 2015, at the Navy League Members of the public will have an
in pediatric therapeutics for future Building, 2300 Wilson Blvd. Arlington, opportunity to provide oral comments
consideration. All nominations should VA 22201. This meeting will be open to on the topics on the meeting agenda
be submitted to Dr. Perdita Taylor- the public. below, and on any previous studies
Zapata at the address in the ADDRESSES DATES: The National Infrastructure issued by the National Infrastructure
section of this notice. Advisory Council will meet on Advisory Council. We request that
September 11, 2015 from 1:30 p.m.–4:30 comments be limited to the issues and
Priority List of Needs in Pediatric p.m. EDT. The meeting may close early studies listed in the meeting agenda and
Therapeutics 2015 if the committee has completed its previous National Infrastructure
In accordance with the BPCA business. For additional information, Advisory Council studies. All previous
legislation, the list outlines priority please consult the National National Infrastructure Advisory
needs in pediatric therapeutics for Infrastructure Advisory Council Web Council studies can be located at
multiple therapeutic areas listed below. site, www.dhs.gov/NIAC, or contact the www.dhs.gov/NIAC. Public comments
The complete list can be found on the National Infrastructure Advisory may be submitted in writing or
BPCA Web site at the following address: Council Secretariat by phone at (703) presented in person for the Council to
http://bpca.nichd.nih.gov. 235–2888 or by email at NIAC@ consider. Comments received by Nancy
D Table 1: Infectious Disease Priorities hq.dhs.gov. Wong after 12:00 p.m. on September 9,
D Table 2: Cardiovascular Disease 2015, will still be accepted and
ADDRESSES: Navy League Building, 2300
Priorities reviewed by the members, but not
Wilson Blvd. Arlington, VA 22201.
D Table 3: Respiratory Disease necessarily by the time of the meeting.
Members of the public will register at
Priorities In-person presentations will be limited
the table at the door to the meeting
D Table 4: Intensive Care Priorities to three minutes per speaker, with no
D Table 5: Bio-defense Research room. For information on facilities or
more than 15 minutes for all speakers.
Priorities services for individuals with
Parties interested in making in-person
D Table 6: Pediatric Cancer Priorities disabilities, or to request special
comments should register on the Public
D Table 7: Psychiatric Disorder assistance at the meeting, contact the
Comment Registration list available at
Priorities person listed under FOR FURTHER
the meeting location no later than 15
D Table 8: Neurological Disease INFORMATION CONTACT below as soon as
minutes prior to the beginning of the
Priorities possible.
meeting.
D Table 9: Neonatal Research To facilitate public participation, we
are inviting public comment on the FOR FURTHER INFORMATION CONTACT:
Priorities
D Table 10: Adolescent Research issues to be considered by the Council Nancy Wong, National Infrastructure
Priorities as listed in the ‘‘Summary’’ section Advisory Council, Designated Federal
D Table 11: Hematologic Disease below. Comments must be submitted in Officer, Department of Homeland
Priorities writing no later than 12:00 p.m. on Security, (703) 235–2888.
D Table 12: Endocrine Disease September 9, 2015, in order to be SUPPLEMENTARY INFORMATION: Notice of
Priorities and Diseases with Limited considered by the council in its this meeting is given under the Federal
Alternative Therapies meeting. The comments must be Advisory Committee Act, 5 U.S.C.
rmajette on DSK7SPTVN1PROD with NOTICES
D Table 13: Dermatologic Disease identified by ‘‘DHS–2015–0042,’’ and Appendix. The National Infrastructure
Priorities may be submitted by any one of the Advisory Council shall provide the
D Table 14: Gastrointestinal Disease following methods: President, through the Secretary of
Priorities • Federal eRulemaking Portal: Homeland Security, with advice on the
D Table 15: Renal Disease Priorities www.regulations.gov. Follow the security and resilience of the Nation’s
D Table 16: Rheumatologic Disease instructions for submitting written critical infrastructure sectors.The NIAC
Priorities comments. will meet to discuss issues relevant to
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Document Created: 2015-12-15 10:59:00
Document Modified: 2015-12-15 10:59:00
| Category | Regulatory Information | |
| Collection | Federal Register | |
| sudoc Class | AE 2.7: GS 4.107: AE 2.106: | |
| Publisher | Office of the Federal Register, National Archives and Records Administration | |
| Section | Notices | |
| Action | Notice. | |
| Contact | Dr. Perdita Taylor-Zapata via email at [email protected]; by phone at 301-496-9584; or by fax at 301-480- 2897. | |
| FR Citation | 80 FR 51827 |
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