Best Pharmaceuticals for Children Act (BPCA) Priority List of Needs in Pediatric Therapeutics, 65002-65004 [2012-26214]
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Federal Register / Vol. 77, No. 206 / Wednesday, October 24, 2012 / Notices
dysfunction, progression of carotid
atherosclerosis and left ventricular
hypertrophy, and responses to stress,
racism, and discrimination as well as
new components such as renal disease,
body fat distribution and body
composition, and metabolic
consequences of obesity. The JHS
Community Health Advisor Networks
(CHANs) comprise another component
of the study. The JHS data shows high
prevalences of risk factors: 73% of
recruited participants are hypertensive,
29% are diabetic, 56% are obese (BMI
> 30kg/m2), and 30% have the
metabolic syndrome. Exploration of the
impact on and interaction of high risk
factor levels with other measures of
clinical and subclinical disease will
help identify unique approaches
through epidemiology and prevention
research to reduce the disproportionate
burden of CVD in African-Americans. .
The JHS CHANs play an important role
to address CVD prevention by providing
training to community members to
spread health promotion and prevention
messages within the Jackson
community. The JHS Community Health
Advisors (CHAs) are trained and
certified to organize and conduct
various outreach activities in five
Jackson-area communities. Data on the
JHS CHAs will be collected. Frequency
of Response: One-time. Affected Public:
Individuals or households; Businesses
or other for profit; not-for-profit
institutions. Type of Respondents:
Middle aged and elderly adults; doctors
and staff of hospitals and nursing
homes. The annual reporting burden is
as follows: Estimated Number of
Respondents: 478; Estimated Number of
Responses per Respondent: 1.0; Average
Burden Hours Per Response: 2.47); and
Estimated Total Annual Burden Hours
Requested: 1253. The annualized cost to
respondents is estimated at $24,206.
There are no Capital Costs to report.
There are no Operating or Maintenance
Costs to report.
ESTIMATE OF ANNUAL HOUR BURDEN
Number of
respondents
Type of respondents
Frequency of
responses
Average time
per response
Annual hour
burden
Families ............................................................................................................
Physicians ........................................................................................................
Communities:
Bolton ........................................................................................................
Canton ......................................................................................................
Clinton .......................................................................................................
Jackson .....................................................................................................
Rankin .......................................................................................................
200
200
1
1
1/6
15/60
331⁄3
50
16
14
13
15
20
10
10
10
10
10
90/60
90/60
90/60
90/60
90/60
240
210
195
225
300
Total ...................................................................................................
478
........................
........................
1253 1⁄3
Request for Comments: Written
comments and/or suggestions from the
public and affected agencies are invited
on one or more of the following points:
(1) Whether the proposed collection of
information is necessary for the proper
performance of the function of the
agency, including whether the
information will have practical utility;
(2) The accuracy of the agency’s
estimate of the burden of the proposed
collection of information, including the
validity of the methodology and
assumptions used; (3) Ways to enhance
the quality, utility, and clarity of the
information to be collected; and (4)
Ways to minimize the burden of the
collection of information on those who
are to respond, including the use of
appropriate automated, electronic,
mechanical, or other technological
collection techniques or other forms of
information technology.
To
request more information on the
proposed project or to obtain a copy of
the data collection plans and
instruments, contact Ms. Cheryl Nelson,
Project Officer, NIH, NHLBI, 6701
Rockledge Drive, MSC 7934, Bethesda,
MD 20892–7934, or call non-toll-free
number 301- 435–0451 or Email your
request, including your address to:
NelsonC@nhlbi.nih.gov.
wreier-aviles on DSK5TPTVN1PROD with
FOR FURTHER INFORMATION CONTACT:
VerDate Mar<15>2010
14:21 Oct 23, 2012
Jkt 229001
Comments Due Date: Comments
regarding this information collection are
best assured of having their full effect if
received within 60-days of the date of
this publication.
Dated: October 18, 2012.
Lynn Susulske,
NHLBI Project Clearance Liaison, National
Institutes of Health.
Michael Lauer,
Director, DCVS, National Institutes of Health.
[FR Doc. 2012–26226 Filed 10–23–12; 8:45 am]
BILLING CODE 4140–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
National Institutes of Health
Best Pharmaceuticals for Children Act
(BPCA) Priority List of Needs in
Pediatric Therapeutics
National Institutes of Health,
the Eunice Kennedy Shriver National
Institute of Child Health and Human
Development.
ACTION: Notice.
AGENCY:
The National Institutes of
Health (NIH) hereby announces the Best
Pharmaceuticals for Children Act
(BPCA) Priority List of Needs in
Pediatric Therapeutics for 2012. The
SUMMARY:
PO 00000
Frm 00053
Fmt 4703
Sfmt 4703
BPCA seeks to improve the level of
information on the safe and effective use
of pharmaceuticals used to treat
children. It 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.
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
widespread 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. Contributing factors to
extensive off-label product use include
limited access to patient populations for
study, lack of knowledge related to the
ethical conduct of clinical trials in
SUPPLEMENTARY INFORMATION:
E:\FR\FM\24OCN1.SGM
24OCN1
Federal Register / Vol. 77, No. 206 / Wednesday, October 24, 2012 / Notices
children, the absence of sufficient
evidence-based information about
medication use in children, and a
general lack of long-term safety data on
the medications that are used. All of
these factors contribute to the lack of
adequately collected pharmacokinetic,
pharmacodynamic, safety, and efficacy
data in children and can increase a
child’s risk for unknown and/or adverse
effects.
The Eunice Kennedy Shriver National
Institute of Child Health and Human
Development (NICHD), the Food and
Drug Administration (FDA), other
federal agencies, and various non-profit
and commercial organizations have
taken steps to address the knowledge
gaps that exist in pediatric therapeutics.
wreier-aviles on DSK5TPTVN1PROD with
The 2002 BPCA Legislation
The initial BPCA legislation
reauthorized an incentive program for
on-patent drugs that met certain criteria
that were first authorized in the FDA
Modernization Act (FDAMA). The
BPCA also contains provisions for offpatent drugs and general support for
pediatric product development that
were not included in the FDAMA. The
legislation, as it applies to the NIH,
authorizes a research program through
the Department of Health and Human
Services (HHS), with implementation
through the NIH, specifically by the
NICHD. The NICHD is responsible for
the development of: (1) A priority list of
needs in pediatric therapeutics, in
consultation with the FDA and experts
in pediatrics; (2) sponsorship of relevant
pediatric clinical trials; and 3)
submission of resulting clinical trial
data to FDA for pediatric labeling
changes.
The Updated BPCA Legislation of 2007
Title V of Public Law 110–85, the Best
Pharmaceuticals for Children Act of
2007, was enacted on September 27,
2007, as part of the Food and Drug
Administration Amendments Act of
2007. This legislation, which
reauthorizes the BPCA (Section 409I of
the Public Health Service Act), extends
the 6-month patent exclusivity
provision for currently on-patent drugs
being studied for pediatric use, and also
extends and expands the NIH research
program that was established in the
earlier law. The priority list procedure
was revised to emphasize knowledge
gaps in therapeutic areas in contrast to
knowledge gaps about specific drug
products.
Update on BPCA Prioritization
The BPCA requires that the NIH, in
consultation with the Food and Drug
Administration and experts in pediatric
VerDate Mar<15>2010
14:21 Oct 23, 2012
Jkt 229001
research, identify the drugs and
therapeutic areas of highest priority for
study in pediatric populations. Part of
fulfilling 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 drugs/needs in
pediatric therapeutics. The BPCA
Priority List consists of key therapeutic
needs in the medical treatment of
children and adolescents; 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 April 1, 2011; all
Federal Register notices can be found
on the BPCA Web site: https://
bpca.nichd.nih.gov/prioritization/
status.cfm. NIH is required by 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 Branch of the NICHD has
developed a prioritization process for
determination of the needs in pediatric
therapeutics. There are two main phases
in the prioritization process. Phase I
entails identifying therapeutic areas,
which are general categories of
conditions, diseases, settings of care, or
populations with multiple therapeutic
needs. Phase II involves determining
more specific pediatric needs, including
research associated with a particular
drug, biologic, or device. Please visit the
BPCA Web site for more details (https://
bpca.nichd.nih.gov/prioritization/
priority_list.cfm). Factors incorporated
in the process include the following:
› Availability of information
concerning the safe and effective use of
a drug in the pediatric population and
the need for additional information;
› Potential health benefits in the
pediatric population resulting from new
studies;
› Possible need for reformulation of
existing products;
› Therapeutic gaps in pediatrics that
may include developmental
pharmacology, pharmacogenetic
determinants of drug response,
metabolism of drugs and biologics in
children, and pediatric clinical trials;
› Particular pediatric diseases,
disorders, or conditions where more
complex knowledge and testing of
therapeutics, including drugs and
biologics, may be beneficial in
pediatric populations; and
PO 00000
Frm 00054
Fmt 4703
Sfmt 4703
65003
› The adequacy of necessary
infrastructure to conduct pediatric
pharmacological research, including
research networks and trained
pediatric investigators.
The NICHD evaluates the current list
of needs in pediatric therapeutics
regularly to determine target areas for
the coming calendar year. The NICHD
sponsored the BPCA Annual
Prioritization Meeting, held December
9–10, 2011, with stakeholders from the
NIH, the FDA, and the American
Academy of Pediatrics (AAP), as well as
other pediatric organizations, societies,
and patient advocates. The meeting
allowed all stakeholders to review the
present progress from ongoing research,
to discuss lessons learned since the
implementation of the BPCA legislation,
and to discuss the proposed therapeutic
areas from the 2011 recommendations
for future study under the BPCA.
Meeting minutes can be found on the
BPCA Web site: https://
bpca.nichd.nih.gov/prioritization/
meeting_summary.cfm.
Below is an updated list of
therapeutic areas and drugs that have
been prioritized for study since the
inception of the BPCA, which includes
new areas of prioritization from the
2010 outreach nominations,
recommendations from the 2011
working groups, and a summary of the
NICHD’s plans and progress in all of
these areas. The NICHD also solicits
input from the pediatric medical
community on additional gaps in
pediatric therapeutics for future
consideration. All nominations should
be submitted to Dr. Perdita TaylorZapata at the contact information below.
Priority List of Needs in Pediatric
Therapeutics 2012
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:
https://bpca.nichd.nih.gov.
› Table 1: Infectious Disease Priorities
› Table 2: Cardiovascular Disease
Priorities
› Table 3: Respiratory Disease
Priorities
› Table 4: Intensive Care Priorities
› Table 5: Bio-defense Research
Priorities
› Table 6: Pediatric Cancer Priorities
› Table 7: Psychiatric Disorder
Priorities
› Table 8: Neurological Disease
Priorities
› Table 9: Neonatal Research Priorities
› Table 10: Adolescent Research
Priorities
E:\FR\FM\24OCN1.SGM
24OCN1
65004
Federal Register / Vol. 77, No. 206 / Wednesday, October 24, 2012 / Notices
› Table 11: Hematologic Disease
Priorities
› Table 12: Endocrine Disease
Priorities and Diseases with Limited
Alternative Therapies
› Table 13: Dermatologic Disease
Priorities
› Table 14: Gastrointestinal Disease
Priorities
› Table 15: Renal Disease Priorities
› Table 16: Rheumatologic Disease
Priorities
› Table 17: Special Considerations.
FOR FURTHER INFORMATION CONTACT: Dr.
Perdita Taylor-Zapata via email at
taylorpe@mail.nih.gov; by phone at
301–496–9584; or by fax at 301–480–
2897.
Dated: October 17, 2012.
Francis S. Collins,
Director, National Institutes of Health.
[FR Doc. 2012–26214 Filed 10–23–12; 8:45 am]
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Dated: October 18, 2012.
Melanie J. Gray,
Program Analyst, Office of Federal Advisory
Committee Policy.
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
National Institutes of Health
[FR Doc. 2012–26097 Filed 10–23–12; 8:45 am]
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Date: November 14, 2012.
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Agenda: To review and evaluate personal
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Place: National Institutes of Health,
Building 31, Conference Room 6, 31 Center
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Contact Person: Florence E. Farber, Ph.D.,
Executive Secretary, Office of the Director,
National Cancer Institute, National Institutes
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E:\FR\FM\24OCN1.SGM
24OCN1
]]>Agencies
[Federal Register Volume 77, Number 206 (Wednesday, October 24, 2012)]
[Notices]
[Pages 65002-65004]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2012-26214]
-----------------------------------------------------------------------
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.
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 2012. The BPCA seeks to improve the level of
information on the safe and effective use of pharmaceuticals used to
treat children. It 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 widespread 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. Contributing factors to
extensive off-label product use include limited access to patient
populations for study, lack of knowledge related to the ethical conduct
of clinical trials in
[[Page 65003]]
children, the absence of sufficient evidence-based information about
medication use in children, and a general lack of long-term safety data
on the medications that are used. All of these factors contribute to
the lack of adequately collected pharmacokinetic, pharmacodynamic,
safety, and efficacy data in children and can increase a child's risk
for unknown and/or adverse effects.
The Eunice Kennedy Shriver National Institute of Child Health and
Human Development (NICHD), the Food and Drug Administration (FDA),
other federal agencies, and various non-profit and commercial
organizations have taken steps to address the knowledge gaps that exist
in pediatric therapeutics.
The 2002 BPCA Legislation
The initial BPCA legislation reauthorized an incentive program for
on-patent drugs that met certain criteria that were first authorized in
the FDA Modernization Act (FDAMA). The BPCA also contains provisions
for off-patent drugs and general support for pediatric product
development that were not included in the FDAMA. The legislation, as it
applies to the NIH, authorizes a research program through the
Department of Health and Human Services (HHS), with implementation
through the NIH, specifically by the NICHD. The NICHD is responsible
for the development of: (1) A priority list of needs in pediatric
therapeutics, in consultation with the FDA and experts in pediatrics;
(2) sponsorship of relevant pediatric clinical trials; and 3)
submission of resulting clinical trial data to FDA for pediatric
labeling changes.
The Updated BPCA Legislation of 2007
Title V of Public Law 110-85, the Best Pharmaceuticals for Children
Act of 2007, was enacted on September 27, 2007, as part of the Food and
Drug Administration Amendments Act of 2007. This legislation, which
reauthorizes the BPCA (Section 409I of the Public Health Service Act),
extends the 6-month patent exclusivity provision for currently on-
patent drugs being studied for pediatric use, and also extends and
expands the NIH research program that was established in the earlier
law. The priority list procedure was revised to emphasize knowledge
gaps in therapeutic areas in contrast to knowledge gaps about specific
drug products.
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. Part of fulfilling 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 drugs/
needs in pediatric therapeutics. The BPCA Priority List consists of key
therapeutic needs in the medical treatment of children and adolescents;
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 April 1, 2011; all Federal
Register notices can be found on the BPCA Web site: https://bpca.nichd.nih.gov/prioritization/status.cfm. NIH is required by 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 Branch of the NICHD has
developed a prioritization process for determination of the needs in
pediatric therapeutics. There are two main phases in the prioritization
process. Phase I entails identifying therapeutic areas, which are
general categories of conditions, diseases, settings of care, or
populations with multiple therapeutic needs. Phase II involves
determining more specific pediatric needs, including research
associated with a particular drug, biologic, or device. Please visit
the BPCA Web site for more details (https://bpca.nichd.nih.gov/prioritization/priority_list.cfm). Factors incorporated in the process
include the following:
[dec221] Availability of information concerning the safe and
effective use of a drug in the pediatric population and the need for
additional information;
[dec221] Potential health benefits in the pediatric population
resulting from new studies;
[dec221] Possible need for reformulation of existing products;
[dec221] Therapeutic gaps in pediatrics that may include
developmental pharmacology, pharmacogenetic determinants of drug
response, metabolism of drugs and biologics in children, and pediatric
clinical trials;
[dec221] Particular pediatric diseases, disorders, or conditions where
more complex knowledge and testing of therapeutics, including drugs and
biologics, may be beneficial in pediatric populations; and
[dec221] The adequacy of necessary infrastructure to conduct pediatric
pharmacological research, including research networks and trained
pediatric investigators.
The NICHD evaluates the current list of needs in pediatric
therapeutics regularly to determine target areas for the coming
calendar year. The NICHD sponsored the BPCA Annual Prioritization
Meeting, held December 9-10, 2011, with stakeholders from the NIH, the
FDA, and the American Academy of Pediatrics (AAP), as well as other
pediatric organizations, societies, and patient advocates. The meeting
allowed all stakeholders to review the present progress from ongoing
research, to discuss lessons learned since the implementation of the
BPCA legislation, and to discuss the proposed therapeutic areas from
the 2011 recommendations for future study under the BPCA. Meeting
minutes can be found on the BPCA Web site: https://bpca.nichd.nih.gov/prioritization/meeting_summary.cfm.
Below is an updated list of therapeutic areas and drugs that have
been prioritized for study since the inception of the BPCA, which
includes new areas of prioritization from the 2010 outreach
nominations, recommendations from the 2011 working groups, and a
summary of the NICHD's plans and progress in all of these areas. The
NICHD also solicits 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
contact information below.
Priority List of Needs in Pediatric Therapeutics 2012
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: https://bpca.nichd.nih.gov.
[dec221] Table 1: Infectious Disease Priorities
[dec221] Table 2: Cardiovascular Disease Priorities
[dec221] Table 3: Respiratory Disease Priorities
[dec221] Table 4: Intensive Care Priorities
[dec221] Table 5: Bio-defense Research Priorities
[dec221] Table 6: Pediatric Cancer Priorities
[dec221] Table 7: Psychiatric Disorder Priorities
[dec221] Table 8: Neurological Disease Priorities
[dec221] Table 9: Neonatal Research Priorities
[dec221] Table 10: Adolescent Research Priorities
[[Page 65004]]
[dec221] Table 11: Hematologic Disease Priorities
[dec221] Table 12: Endocrine Disease Priorities and Diseases with
Limited Alternative Therapies
[dec221] Table 13: Dermatologic Disease Priorities
[dec221] Table 14: Gastrointestinal Disease Priorities
[dec221] Table 15: Renal Disease Priorities
[dec221] Table 16: Rheumatologic Disease Priorities
[dec221] Table 17: Special Considerations.
FOR FURTHER INFORMATION CONTACT: Dr. Perdita Taylor-Zapata via email at
taylorpe@mail.nih.gov; by phone at 301-496-9584; or by fax at 301-480-
2897.
Dated: October 17, 2012.
Francis S. Collins,
Director, National Institutes of Health.
[FR Doc. 2012-26214 Filed 10-23-12; 8:45 am]
BILLING CODE 4140-01-P
