The Best Pharmaceuticals for Children Act (BPCA) Priority List of Needs in Pediatric Therapeutics, 17203-17205 [E9-8477]
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Federal Register / Vol. 74, No. 70 / Tuesday, April 14, 2009 / Notices
findings, and report the findings
generically to all interested parties.
DATES: Letter of interest must be
received within 90 calendar days of
publication in the Federal Register.
ADDRESSES: Mining companies able to
provide NIOSH with mine sites for this
research should submit a letter of
interest to the NIOSH Pittsburgh
Research Laboratory (PRL) Director. The
letter should provide the name of the
mine and a brief description of the
anticipated sealing plans. Any questions
should be addressed by phone or e-mail.
¨
Please send letter of interest to: R. Guner
¨
Gurtunca, PhD, NIOSH Pittsburgh
Research Laboratory (PRL), 626
Cochrans Mill Road, Post Office Box
18070, Pittsburgh, PA 15236, telephone
(412) 386–6601, E-mail
GGurtunca@cdc.gov.
Background: Recent research reports
published by NIOSH and the U.S. Army
Corps of Engineers describe the
potential for explosive methane
mixtures to develop within sealed areas
of underground coal mines. The
composition and behavior of the
atmosphere within sealed areas are not
scientifically well-understood. Areas of
interest include the extent and nature of
explosive mixtures of gases, how the
composition of these mixtures change
over time, whether methane layering
exists, the homogeneity of the
atmosphere, and how barometric
pressure changes impact the atmosphere
behind seals.
Description: To conduct these
measurements, NIOSH will deploy a
tube bundle system (TBS) at the mine
site for a period of 2 to 5 months
(usually not more than 3 months). A
TBS is a mechanical system for
collecting and analyzing atmospheric
samples continuously from anywhere in
a mine. The TBS that NIOSH plans to
use is a system that is currently being
successfully deployed in many
Australian underground coal mines.
NIOSH seeks three to four underground
coal mines throughout the U.S. to
cooperate in this study. Underground
coal mines covering at least one square
mile and producing a medium to high
volume of methane are needed.
Sampling will be conducted one mine at
a time. Either longwall or room-andpillar mines are acceptable. NIOSH
wants to deploy the system in a variety
of geological conditions. A soon-to-beabandoned coal mine is another option
for deployment of the TBS.
Prior to sealing, NIOSH will install
plastic sample tubing throughout the
mine and the future sealed area. This
should require a few days to accomplish
and will require minimal effort from the
VerDate Nov<24>2008
16:39 Apr 13, 2009
Jkt 217001
cooperating mine. NIOSH will need to
be present during the sealing process to
insure that the tubing is properly
installed through the seals. After
sealing, NIOSH will monitor the
composition of the atmosphere
throughout the sealed area during the
initial methane-accumulation phase and
for several months thereafter until
stability of the sealed atmosphere
develops. Collected data will not be
analyzed on a real time basis other than
to insure that the system is properly
working.
NIOSH will require the following
assistance from mining company
personnel:
• Site-specific guidance concerning
the area to be sealed and how to most
efficiently run the sampling tube out of
the mine to the sampling analysis
location.
• Transportation to and from the
sealed area during the installation phase
of the TBS and to occasionally check the
status of the TBS underground.
• A surface location to locate the
sampling trailer.
• For a mine site to be acceptable to
NIOSH for this testing, the cooperating
mine must be installing 120 psi seals
that meet the current design standard.
• After installation, NIOSH will
require little assistance from mining
company personnel until NIOSH is
ready to remove the system from the
mine when some transportation
assistance will be needed.
After the data is analyzed, the
cooperating mine will be provided the
data pertaining to its mine. NIOSH will
present and/or publish data in a manner
that does not identify the cooperating
mines. Cooperating mines will have the
opportunity to review publications and
presentations by NIOSH prior to their
release. While NIOSH will not identify
the mines in its publications, the
identity of cooperating mines may be
subject to release in response to a
request for documents made under the
Freedom of Information Act. This
announcement does not obligate NIOSH
to enter into an agreement with any
respondent.
FOR FURTHER INFORMATION CONTACT: R.
¨
¨
Guner Gurtunca, PhD, NIOSH
Pittsburgh Research Laboratory (PRL),
626 Cochrans Mill Road, Post Office Box
18070, Pittsburgh PA, 15236, telephone
(412) 386–6601, e-mail
GGurtunca@cdc.gov.
PO 00000
Frm 00061
Fmt 4703
Sfmt 4703
17203
Dated: April 6, 2009.
Christine M. Branche,
Acting Director, National Institute for
Occupational Safety and Health, Centers for
Disease Control and Prevention.
[FR Doc. E9–8462 Filed 4–13–09; 8:45 am]
BILLING CODE 4163–19–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
National Institutes of Health
The Best Pharmaceuticals for Children
Act (BPCA) Priority List of Needs in
Pediatric Therapeutics
ACTION:
Notice.
SUMMARY: For many decades, the
pediatric medical community, the
public health community, and
government agencies have recognized a
range of questions regarding the use of
therapeutics in children, including the
shortage of clinical studies of drugs in
children resulting in inadequate
labeling for pediatric use. The lack of
appropriate labeling results in off-label
use of prescription drugs in many
children and for many conditions.
Contributing factors to this frequent offlabel use of drugs in pediatrics include
the rarity of some conditions in children
with limited patient availability, the
ethical concerns regarding the conduct
of clinical trials in children, the lack of
accurate information about which drugs
are used by children, and the lack of
long-term data on the medications that
are frequently used.
Several steps have been taken in
response to the growing awareness of
the knowledge gaps that exist in
pediatric therapeutics. The BPCA was
originally enacted in January 2002 and
reauthorized in September 2007, with
the overall purpose of improving the
level of information about
pharmaceuticals used to treat children
(https://www.fda.gov/opacom/laws/
pharmkids/contents.html). The BPCA
outlines a number of goals, including
the identification and prioritization of
therapeutic needs in pediatrics,
especially drugs, biologics, or
indications that require study. The
legislation also calls for the conduct of
pediatric research to learn more about
the efficacy and safety of drugs in
children as well as the training of
experts needed to address the
knowledge gaps in pediatric
pharmacology. To identify drugs in
need of further study, the BPCA
mandates that the National Institutes of
Health (NIH), in consultation with the
U.S. Food and Drug Administration
(FDA) and experts in pediatrics, develop
E:\FR\FM\14APN1.SGM
14APN1
17204
Federal Register / Vol. 74, No. 70 / Tuesday, April 14, 2009 / Notices
a process for prioritizing needs in
pediatric therapeutics and publish a
priority list at least every 3 years,
starting in September 2008. In this
notice, we will summarize past efforts to
prioritize off-patent drugs that need
further study as mandated by the BPCA
2002 and describe the plans for
identifying needs in pediatric
therapeutics as authorized by the BPCA
2007.
DATES: The list is effective upon
publication.
FOR FURTHER INFORMATION CONTACT: Dr.
Perdita Taylor-Zapata, Eunice Kennedy
Shriver National Institute of Child
Health and Human Development
(NICHD), 6100 Executive Boulevard,
Suite 4A–01, Bethesda, MD 20892–
7510, e-mail taylorpe@mail.nih.gov or
BestPharmaceuticals@mail.nih.gov,
telephone 301–496–9584 (not a toll-free
number).
In the
Federal Register Notice of January 21,
2003 (Vol. 68, No. 13), the NICHD
announced the first list of off-patent
drugs to be considered for study and
described the process used in
developing this list. Prioritization of
drugs on the list was based in general
on three major factors: (1) Frequency of
use in the pediatric population, (2)
severity of the condition being treated,
and (3) potential for providing a health
benefit in the pediatric population.
These factors follow from the original
BPCA legislation, which required NIH
to consider (among other criteria) for
each drug ‘‘whether new pediatric
studies concerning the drug may
produce health benefits in the pediatric
population.’’
During the initial years of the BPCA
prioritization process (2003–2005), the
NICHD identified many individual
drugs and indications that required
further dosing, efficacy, and safety
information. In 2005, based on the input
of pediatric experts, an alternative
approach was proposed that included
identifying and prioritizing pediatric
conditions and therapeutic approaches
for those conditions. This proposed
condition-based approach would allow
us to identify gaps in scientific
knowledge, determine key research
agendas in pediatric medicine, evaluate
the treatments of these conditions, and
compare the use of drugs within a
therapeutic class (both on- and offpatent). This approach would also allow
us to obtain focused expertise in
specific therapeutic areas that would
help elucidate the scientific gaps within
the prioritized area.
SUPPLEMENTARY INFORMATION:
VerDate Nov<24>2008
16:39 Apr 13, 2009
Jkt 217001
Update on BPCA Conditions/
Therapeutic Areas
In 2006, the NICHD and the FDA, in
collaboration with pediatric experts,
considered an alternative approach for
prioritization—from a drug/indication
approach to a condition-based
approach. Please refer to the Federal
Register Notice of April 25, 2006
(Volume 71, No. 79), and the Federal
Register Notice of March 28, 2007
(Volume 72, No. 59), for a complete
review of the previous prioritization
process and therapeutic categories
considered under the 2002 BPCA
legislation. In addition, an update on
the status of all drugs previously listed
under BPCA 2002 is provided at
https://bpca.nichd.nih.gov. The
following conditions have been listed to
date, with brief updates on progress
and/or current NICHD commitments.
• 2006
—Oncology: Four clinical trials are
under way in collaboration with the
National Cancer Institute (NCI) and
the Children’s Oncology Group (COG)
to evaluate the pharmacokinetics
(PK)/pharmacodynamics (PD), safety,
and efficacy of chemotherapeutic
agents used in children with cancer.
The drugs under study are
methotrexate, vincristine,
daunomycin, and actinomycin-D.
—Sickle Cell Disease (SCD): A clinical
trial of PK, efficacy, and safety of
hydroxyurea to treat infants and
young children with SCD is under
way, with a planned safety follow-up.
—Attention Deficit Hyperactivity
Disorder: The NICHD is funding basic
and clinical research to evaluate the
potential toxicity of methylphenidate.
—Organophosphate poisonings:
Existing data on the use of
pralidoxime for this indication is
under review.
• 2007
—Oncology: The NICHD consulted with
experts in pediatric oncology to
discuss the use of 13-cis-retinoic acid
for the indication of neuroblastoma
and to develop a pediatric
formulation for this indication.
—Methicillin-resistant Staphylococcus
aureus (MRSA) infections: The
NICHD consulted with experts in
infectious disease to discuss the need
for PK, safety, and efficacy studies of
clindamycin, doxycycline,
tetracycline, and trimethoprimsulfamethoxazole for the treatment of
MRSA infections.
—Asthma: The NICHD has pursued
potential collaborations with research
networks within the NIH that are
conducting clinical trials and other
PO 00000
Frm 00062
Fmt 4703
Sfmt 4703
research in pediatric asthma,
specifically networks supported by
the National Heart, Lung, and Blood
Institute and the National Institute of
Allergy and Infectious Diseases.
—Hypertension: A written request for
the study of hydrochlorothiazide in
hypertension has been received by the
NICHD. The NICHD has conducted a
third working group meeting with
experts in the field of pediatric
hypertension to discuss studies
needed in this area. Future clinical
trials are being considered.
• Other areas of continued or future
consideration for study under the BPCA
discussed in previous Federal Register
Notices and/or BPCA scientific meetings
include:
—Obesity: The NICHD is consulting
with experts in the field on the
treatment of the metabolic syndrome
and obesity-related Type-2 diabetes
and hypertension.
—Counterterrorism research: The
NICHD has developed a working
group to discuss the needs in
pediatric therapeutics for the
treatment of chemical, biologic,
radiologic, nuclear, and explosive
(CBRNE) exposure. The NICHD is
collaborating with the National
Institute of Neurological Disorders
and Stroke on its Counter-Act
initiative to develop new and
improved medical counter-measures
against chemical threats in children
and adults.
—Influenza and parasitic diseases: The
NICHD held preliminary discussions
with international experts on global
pediatric pharmacology issues.
Influenza and parasitic diseases are
potential prototypes for future
collaborations.
—Fragile X syndrome: The NICHD
continues to consult with experts in
the field, including the National
Institute of Mental Health, to consider
selected drugs and clinical outcome
measures for evaluation and/or study.
—Depression: The NICHD has consulted
with experts in the field to consider
the approaches and design of safety
studies of psychotropic medications
in children, including antidepressants
and other psychotropic medications.
Throughout 2007 and 2008, the
NICHD continued its outreach to
pediatric organizations and other NIH
Institutes and Centers. The goal of these
discussions was specifically to identify
current gaps in scientific knowledge
regarding research and treatment of
pediatric conditions with the ultimate
goal of determining approved drugs for
which future pediatric studies are
needed. Minutes of all working group
E:\FR\FM\14APN1.SGM
14APN1
Federal Register / Vol. 74, No. 70 / Tuesday, April 14, 2009 / Notices
meetings conducted under the BPCA
can be found on the BPCA Web site
listed above.
The ‘‘New’’ BPCA
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
provision of additional patent
exclusivity for currently on-patent drugs
that are being tested for pediatric use.
This legislation also extends and
expands the research program at the
NIH established in the earlier law. The
NICHD administers the research
program through its Obstetric and
Pediatric Pharmacology Branch,
working in cooperation with the other
NIH Institutes and Centers with
significant pediatric research portfolios.
Important changes to the 2002 BPCA
legislation for the NIH include the
following:
• Focus on condition-based approach.
• More flexible funding mechanisms.
• Development of Proposed Pediatric
Study Requests (PPSR).
• Feasibility study for the
development of a pediatric formulary.
The NICHD will prioritize all
therapeutic areas over the upcoming
years based on the following
considerations:
• Building upon the current
foundation established by the 2002
BPCA implementation;
• Evaluating all currently listed drugs
and therapeutic areas for feasibility and
identification of additional or new
scientific and therapeutic gaps;
• Changing the listing process from
an individual drug/indication approach
to listing needs in pediatric therapeutic
areas;
• Determining new areas of need
based on consultation with other NIH
Institutes and Centers, as well as experts
in pediatric therapeutics and the
pediatric medical community.
The overall goal of the NIH for
implementing the provisions of the
BPCA is to improve pediatric
therapeutics through scientific
advancements and labeling changes that
will have an impact on the safe and
effective use of drugs in children. This
can be accomplished through the
following:
• Data gathering
—Using the principles of
pharmacoepidemiology research to
quantify adverse drug reactions, drug
efficacy, and patterns of drug use in
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16:39 Apr 13, 2009
Jkt 217001
large populations to elucidate health
services utilization.
—Bringing together multidisciplinary
teams to provide input on needs in
pediatric therapeutics through
outreach to experts in pediatric
research in academic institutions;
other NIH Institutes and Centers; and
pediatric organizations, societies,
advocacy groups, and industry.
• Clinical trials
—Phase 1, 2, and 3 clinical trials to
increase the knowledge of PK, safety,
and efficacy of medicines used in
children.
• Basic and translational research
—To inform such areas as
developmental pharmacology,
pharmacogenomics, and pediatric
clinical trial design.
There will be an open scientific
meeting annually, starting in 2008, to
review and discuss the proposed
therapeutic areas, to present progress
from ongoing research, and to provide
an opportunity for the medical
community to provide input into the
future therapeutic areas to be studied
under the BPCA. Stakeholders will
include the NIH, the FDA, and members
of the American Academy of Pediatrics,
and other pediatric organizations and
societies. There will be a report to
Congress at least every 3 years starting
in 2008. Throughout the year, there will
also be smaller group meetings with
expert panels within prioritized
therapeutic areas under the BPCA. The
goals of the working group meetings
will be to evaluate and discuss the gaps
in scientific knowledge (whether
necessary data are available or
unavailable) as well as to determine
gaps in the treatments of these
conditions; for example, to determine
what may be needed to enhance the
treatment of these conditions in
children. These consultations will assist
the NICHD in the development of future
proposed areas of study encompassing
multiple therapeutic categories and/or
addressing multiple questions within a
therapeutic category.
A scientific prioritization meeting was
held in Rockville, Maryland, from June
30 to July 1, 2008, to determine needs
in pediatric therapeutics as mandated
by the BPCA 2007 legislation. The final
BPCA List of Needs in Pediatric
Therapeutics, and information on the
prioritization process, will be posted on
the BPCA Web site https://
bpca.nichd.nih.gov.
PO 00000
Frm 00063
Fmt 4703
Sfmt 4703
17205
Dated: April 7, 2009.
Raynard S. Kington,
Acting Director, National Institutes of Health.
[FR Doc. E9–8477 Filed 4–13–09; 8:45 am]
BILLING CODE 4140–01–P
DEPARTMENT OF HOMELAND
SECURITY
[Docket No. DHS–2009–0008]
The National Infrastructure Advisory
Council
AGENCY: Directorate for National
Protection and Programs, Department of
Homeland Security.
ACTION: Committee Management; Notice
of cancellation for Federal Advisory
Committee Meeting.
SUMMARY: The meeting of the National
Infrastructure Advisory Council (NIAC)
scheduled for Tuesday April 14, 2009 at
the J.W. Marriott, 1331 Pennsylvania
Avenue, Washington, DC announced in
the Federal Register on February 17,
2009 (73 FR 7456), will not be held.
FOR FURTHER INFORMATION CONTACT:
Contact Matthew Sickbert by phone at
703–235–2888 or by e-mail at
Matthew.Sickbert@associates.dhs.gov.
Dated: April 9, 2009.
Nancy J. Wong,
Designated Federal Officer for the NIAC.
[FR Doc. E9–8541 Filed 4–10–09; 11:15 am]
BILLING CODE 4410–10–P
DEPARTMENT OF HOMELAND
SECURITY
United States Immigration and
Customs Enforcement
60-Day Notice of New Information
Collection; Form 70–005, ICE Secure
Communities Stakeholder ID
Assessment Questionnaire; Agency
Information Collection Activities: New
Information Collection; Comment
Request
ACTION: 60-Day Notice of New
Information Collection; Form 70–005,
ICE Secure Communities Stakeholder ID
Assessment Questionnaire.
The Department of Homeland
Security, U.S. Immigration and Customs
Enforcement (USICE), has submitted the
following information collection request
for review and clearance in accordance
with the Paperwork Reduction Act of
1995. The information collection is
published to obtain comments from the
public and affected agencies. Comments
are encouraged and will be accepted for
sixty days until June 15, 2009.
E:\FR\FM\14APN1.SGM
14APN1
Agencies
[Federal Register Volume 74, Number 70 (Tuesday, April 14, 2009)]
[Notices]
[Pages 17203-17205]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: E9-8477]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
National Institutes of Health
The Best Pharmaceuticals for Children Act (BPCA) Priority List of
Needs in Pediatric Therapeutics
ACTION: Notice.
-----------------------------------------------------------------------
SUMMARY: For many decades, the pediatric medical community, the public
health community, and government agencies have recognized a range of
questions regarding the use of therapeutics in children, including the
shortage of clinical studies of drugs in children resulting in
inadequate labeling for pediatric use. The lack of appropriate labeling
results in off-label use of prescription drugs in many children and for
many conditions. Contributing factors to this frequent off-label use of
drugs in pediatrics include the rarity of some conditions in children
with limited patient availability, the ethical concerns regarding the
conduct of clinical trials in children, the lack of accurate
information about which drugs are used by children, and the lack of
long-term data on the medications that are frequently used.
Several steps have been taken in response to the growing awareness
of the knowledge gaps that exist in pediatric therapeutics. The BPCA
was originally enacted in January 2002 and reauthorized in September
2007, with the overall purpose of improving the level of information
about pharmaceuticals used to treat children (https://www.fda.gov/opacom/laws/pharmkids/contents.html). The BPCA outlines a number of
goals, including the identification and prioritization of therapeutic
needs in pediatrics, especially drugs, biologics, or indications that
require study. The legislation also calls for the conduct of pediatric
research to learn more about the efficacy and safety of drugs in
children as well as the training of experts needed to address the
knowledge gaps in pediatric pharmacology. To identify drugs in need of
further study, the BPCA mandates that the National Institutes of Health
(NIH), in consultation with the U.S. Food and Drug Administration (FDA)
and experts in pediatrics, develop
[[Page 17204]]
a process for prioritizing needs in pediatric therapeutics and publish
a priority list at least every 3 years, starting in September 2008. In
this notice, we will summarize past efforts to prioritize off-patent
drugs that need further study as mandated by the BPCA 2002 and describe
the plans for identifying needs in pediatric therapeutics as authorized
by the BPCA 2007.
DATES: The list is effective upon publication.
FOR FURTHER INFORMATION CONTACT: Dr. Perdita Taylor-Zapata, Eunice
Kennedy Shriver National Institute of Child Health and Human
Development (NICHD), 6100 Executive Boulevard, Suite 4A-01, Bethesda,
MD 20892-7510, e-mail taylorpe@mail.nih.gov or
BestPharmaceuticals@mail.nih.gov, telephone 301-496-9584 (not a toll-
free number).
SUPPLEMENTARY INFORMATION: In the Federal Register Notice of January
21, 2003 (Vol. 68, No. 13), the NICHD announced the first list of off-
patent drugs to be considered for study and described the process used
in developing this list. Prioritization of drugs on the list was based
in general on three major factors: (1) Frequency of use in the
pediatric population, (2) severity of the condition being treated, and
(3) potential for providing a health benefit in the pediatric
population. These factors follow from the original BPCA legislation,
which required NIH to consider (among other criteria) for each drug
``whether new pediatric studies concerning the drug may produce health
benefits in the pediatric population.''
During the initial years of the BPCA prioritization process (2003-
2005), the NICHD identified many individual drugs and indications that
required further dosing, efficacy, and safety information. In 2005,
based on the input of pediatric experts, an alternative approach was
proposed that included identifying and prioritizing pediatric
conditions and therapeutic approaches for those conditions. This
proposed condition-based approach would allow us to identify gaps in
scientific knowledge, determine key research agendas in pediatric
medicine, evaluate the treatments of these conditions, and compare the
use of drugs within a therapeutic class (both on- and off-patent). This
approach would also allow us to obtain focused expertise in specific
therapeutic areas that would help elucidate the scientific gaps within
the prioritized area.
Update on BPCA Conditions/Therapeutic Areas
In 2006, the NICHD and the FDA, in collaboration with pediatric
experts, considered an alternative approach for prioritization--from a
drug/indication approach to a condition-based approach. Please refer to
the Federal Register Notice of April 25, 2006 (Volume 71, No. 79), and
the Federal Register Notice of March 28, 2007 (Volume 72, No. 59), for
a complete review of the previous prioritization process and
therapeutic categories considered under the 2002 BPCA legislation. In
addition, an update on the status of all drugs previously listed under
BPCA 2002 is provided at https://bpca.nichd.nih.gov. The following
conditions have been listed to date, with brief updates on progress
and/or current NICHD commitments.
2006
--Oncology: Four clinical trials are under way in collaboration with
the National Cancer Institute (NCI) and the Children's Oncology Group
(COG) to evaluate the pharmacokinetics (PK)/pharmacodynamics (PD),
safety, and efficacy of chemotherapeutic agents used in children with
cancer. The drugs under study are methotrexate, vincristine,
daunomycin, and actinomycin-D.
--Sickle Cell Disease (SCD): A clinical trial of PK, efficacy, and
safety of hydroxyurea to treat infants and young children with SCD is
under way, with a planned safety follow-up.
--Attention Deficit Hyperactivity Disorder: The NICHD is funding basic
and clinical research to evaluate the potential toxicity of
methylphenidate.
--Organophosphate poisonings: Existing data on the use of pralidoxime
for this indication is under review.
2007
--Oncology: The NICHD consulted with experts in pediatric oncology to
discuss the use of 13-cis-retinoic acid for the indication of
neuroblastoma and to develop a pediatric formulation for this
indication.
--Methicillin-resistant Staphylococcus aureus (MRSA) infections: The
NICHD consulted with experts in infectious disease to discuss the need
for PK, safety, and efficacy studies of clindamycin, doxycycline,
tetracycline, and trimethoprim-sulfamethoxazole for the treatment of
MRSA infections.
--Asthma: The NICHD has pursued potential collaborations with research
networks within the NIH that are conducting clinical trials and other
research in pediatric asthma, specifically networks supported by the
National Heart, Lung, and Blood Institute and the National Institute of
Allergy and Infectious Diseases.
--Hypertension: A written request for the study of hydrochlorothiazide
in hypertension has been received by the NICHD. The NICHD has conducted
a third working group meeting with experts in the field of pediatric
hypertension to discuss studies needed in this area. Future clinical
trials are being considered.
Other areas of continued or future consideration for study
under the BPCA discussed in previous Federal Register Notices and/or
BPCA scientific meetings include:
--Obesity: The NICHD is consulting with experts in the field on the
treatment of the metabolic syndrome and obesity-related Type-2 diabetes
and hypertension.
--Counterterrorism research: The NICHD has developed a working group to
discuss the needs in pediatric therapeutics for the treatment of
chemical, biologic, radiologic, nuclear, and explosive (CBRNE)
exposure. The NICHD is collaborating with the National Institute of
Neurological Disorders and Stroke on its Counter-Act initiative to
develop new and improved medical counter-measures against chemical
threats in children and adults.
--Influenza and parasitic diseases: The NICHD held preliminary
discussions with international experts on global pediatric pharmacology
issues. Influenza and parasitic diseases are potential prototypes for
future collaborations.
--Fragile X syndrome: The NICHD continues to consult with experts in
the field, including the National Institute of Mental Health, to
consider selected drugs and clinical outcome measures for evaluation
and/or study.
--Depression: The NICHD has consulted with experts in the field to
consider the approaches and design of safety studies of psychotropic
medications in children, including antidepressants and other
psychotropic medications.
Throughout 2007 and 2008, the NICHD continued its outreach to
pediatric organizations and other NIH Institutes and Centers. The goal
of these discussions was specifically to identify current gaps in
scientific knowledge regarding research and treatment of pediatric
conditions with the ultimate goal of determining approved drugs for
which future pediatric studies are needed. Minutes of all working group
[[Page 17205]]
meetings conducted under the BPCA can be found on the BPCA Web site
listed above.
The ``New'' BPCA
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 provision of additional patent
exclusivity for currently on-patent drugs that are being tested for
pediatric use. This legislation also extends and expands the research
program at the NIH established in the earlier law. The NICHD
administers the research program through its Obstetric and Pediatric
Pharmacology Branch, working in cooperation with the other NIH
Institutes and Centers with significant pediatric research portfolios.
Important changes to the 2002 BPCA legislation for the NIH include the
following:
Focus on condition-based approach.
More flexible funding mechanisms.
Development of Proposed Pediatric Study Requests (PPSR).
Feasibility study for the development of a pediatric
formulary.
The NICHD will prioritize all therapeutic areas over the upcoming
years based on the following considerations:
Building upon the current foundation established by the
2002 BPCA implementation;
Evaluating all currently listed drugs and therapeutic
areas for feasibility and identification of additional or new
scientific and therapeutic gaps;
Changing the listing process from an individual drug/
indication approach to listing needs in pediatric therapeutic areas;
Determining new areas of need based on consultation with
other NIH Institutes and Centers, as well as experts in pediatric
therapeutics and the pediatric medical community.
The overall goal of the NIH for implementing the provisions of the
BPCA is to improve pediatric therapeutics through scientific
advancements and labeling changes that will have an impact on the safe
and effective use of drugs in children. This can be accomplished
through the following:
Data gathering
--Using the principles of pharmacoepidemiology research to quantify
adverse drug reactions, drug efficacy, and patterns of drug use in
large populations to elucidate health services utilization.
--Bringing together multidisciplinary teams to provide input on needs
in pediatric therapeutics through outreach to experts in pediatric
research in academic institutions; other NIH Institutes and Centers;
and pediatric organizations, societies, advocacy groups, and industry.
Clinical trials
--Phase 1, 2, and 3 clinical trials to increase the knowledge of PK,
safety, and efficacy of medicines used in children.
Basic and translational research
--To inform such areas as developmental pharmacology, pharmacogenomics,
and pediatric clinical trial design.
There will be an open scientific meeting annually, starting in
2008, to review and discuss the proposed therapeutic areas, to present
progress from ongoing research, and to provide an opportunity for the
medical community to provide input into the future therapeutic areas to
be studied under the BPCA. Stakeholders will include the NIH, the FDA,
and members of the American Academy of Pediatrics, and other pediatric
organizations and societies. There will be a report to Congress at
least every 3 years starting in 2008. Throughout the year, there will
also be smaller group meetings with expert panels within prioritized
therapeutic areas under the BPCA. The goals of the working group
meetings will be to evaluate and discuss the gaps in scientific
knowledge (whether necessary data are available or unavailable) as well
as to determine gaps in the treatments of these conditions; for
example, to determine what may be needed to enhance the treatment of
these conditions in children. These consultations will assist the NICHD
in the development of future proposed areas of study encompassing
multiple therapeutic categories and/or addressing multiple questions
within a therapeutic category.
A scientific prioritization meeting was held in Rockville,
Maryland, from June 30 to July 1, 2008, to determine needs in pediatric
therapeutics as mandated by the BPCA 2007 legislation. The final BPCA
List of Needs in Pediatric Therapeutics, and information on the
prioritization process, will be posted on the BPCA Web site https://bpca.nichd.nih.gov.
Dated: April 7, 2009.
Raynard S. Kington,
Acting Director, National Institutes of Health.
[FR Doc. E9-8477 Filed 4-13-09; 8:45 am]
BILLING CODE 4140-01-P