Developing a Consolidated Pediatric Rheumatology Observational Registry; Public Workshop, 13215-13216 [E9-6709]
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Federal Register / Vol. 74, No. 57 / Thursday, March 26, 2009 / Notices
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Dated: March 19, 2009.
Randall W. Lutter,
Deputy Commissioner for Policy.
[FR Doc. E9–6796 Filed 3–25–09; 8:45 am]
BILLING CODE 4160–01–S
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2009–N–0145]
Developing a Consolidated Pediatric
Rheumatology Observational Registry;
Public Workshop
AGENCY:
Food and Drug Administration,
HHS.
ACTION: Notice of public workshop;
request for comments.
SUMMARY: The Food and Drug
Administration (FDA) is announcing a
public workshop entitled ‘‘Developing a
Consolidated Pediatric Rheumatology
Observational Registry.’’ This 2-day
public workshop is intended to seek
VerDate Nov<24>2008
20:28 Mar 25, 2009
Jkt 217001
constructive input from key
stakeholders in the pediatric
rheumatology community, the
pharmaceutical industry and the public
to explore the value and feasibility of
developing a consolidated pediatric
rheumatology observational registry.
DATES: The public workshop will be
held on May 12, 2009, from 8:30 a.m. to
5 p.m. and on May 13, 2009, from 8:30
a.m. to noon. Register by April 21, 2009,
to make a presentation at the workshop.
See section III of this document for
information on how to attend or present
at the workshop. We are opening a
docket to receive your written or
electronic comments. Written or
electronic comments must be submitted
to the docket by July 14, 2009.
ADDRESSES: The public workshop will
be held at the Hilton Washington DC/
Silver Spring, The Ballrooms, 8727
Colesville Rd., Silver Spring, MD 20910
(Metro: Silver Spring Station on the Red
Line). Submit written or electronic
requests to make a presentation to Diane
Ehrlich (see FOR FURTHER INFORMATION
CONTACT).
Submit written comments to the
Division of Dockets Management (HFA–
305), Food and Drug Administration,
5630 Fishers Lane, rm. 1061. Rockville,
MD 20852. Submit electronic comments
to https://www.regulations.gov. All
comments should be identified with the
docket number found in brackets in the
heading of this document.
Transcripts of the hearing will be
available for review at the Division of
Dockets Management and on the
Internet at https://www.regulations.gov
approximately 30 days after the
workshop.
FOR FURTHER INFORMATION CONTACT:
Diane Ehrlich, Center for Drug
Evaluation and Research, Food and
Drug Administration, 10903 New
Hampshire Ave., Bldg. 51, rm. 6190,
Silver Spring, MD 20993–0002, 301–
796–3452, FAX: 301–847–8753, e-mail:
Diane.Ehrlich@fda.hhs.gov.
SUPPLEMENTARY INFORMATION:
I. Background
Currently, approved drug and
therapeutic biological products for
patients with juvenile idiopathic
arthritis (JIA) (or juvenile rheumatoid
arthritis (JRA)) are monitored for longterm safety beyond the information
available at the time of approval on a
product-by-product basis using
registries mandated by FDA’s
postmarketing requirements. FDA is
addressing concerns raised by
individuals in the pediatric
rheumatology community about the
current approach of using product-
PO 00000
Frm 00043
Fmt 4703
Sfmt 4703
13215
specific pediatric rheumatology
observational safety registries. Some of
the concerns expressed include the
following:
1. It is difficult to capture important
information from children and
adolescents whose medication is
switched over time because long-term
data on these patients will not be
available under the product specific
registries. Patients on ‘‘real-life’’
combinations of medications and/or
nonstandard doses are often not
included in product-specific registries.
2. Current registries do not always
provide an adequate control group to
assess background rates of important
adverse events.
3. The limited number of patients
with JIA will make adequate enrollment
in product-specific observational
registries more difficult as the number
of approved drug and biological
products increases.
4. A nonproprietary registry rather
than a proprietary registry would allow
wider access to the safety data that is
collected.
5. A consolidated pediatric
rheumatology observational registry
may allow more efficient identification
of longer term safety issues in this
population.
II. Scope of Public Workshop
At the public workshop, FDA will
present its current thinking on the use
of product-specific postmarketing
registries to capture long-term safety
data of drug and biological products
administered to patients with JIA.
Product-specific registries will be
compared with a consolidated pediatric
rheumatology observational registry that
could meet the regulatory postmarketing
requirements of FDA and also collect
other safety information and support
potential research initiatives.
A. Objectives of the Workshop
The workshop objectives are as
follows:
1. Discuss potential registry models,
taking into account existing registries
for other diseases and in other
countries.
2. Discuss the advantages and
disadvantages of a common
consolidated registry for JIA, taking into
account:
∑ The pediatric rheumatology
perspective.
∑ The pharmaceutical company
perspective.
3. Discuss methods to capture
information regarding safety signals in
rare diseases.
4. Discuss the value of working
through existing large pediatric
E:\FR\FM\26MRN1.SGM
26MRN1
13216
Federal Register / Vol. 74, No. 57 / Thursday, March 26, 2009 / Notices
rheumatology collaborative networks,
such as the Childhood Arthritis and
Rheumatology Research Alliance or the
Pediatric Rheumatology Collaborative
Study Group.
5. Define, for phase 4 studies in JIA
patients, the database standards and
elements of data collection (e.g., data
quality, monitoring) that are necessary
and sufficient to meet FDA regulatory
requirements.
6. Discuss how pertinent research
initiatives can be accomplished in the
framework of a consolidated JIA
registry, including:
∑ Ethical considerations.
∑ Data sharing considerations.
7. Discuss the options for funding a
consolidated JIA registry.
B. Issues for Comment
FDA is interested in obtaining public
comment on the following issues
relating to development of a
consolidated pediatric rheumatology
observational registry:
1. Should we transition from productspecific registries to a consolidated
pediatric rheumatology observational
registry?
2. Currently, the product-specific
registries are conducted by the
individual sponsors of the approved
drugs and/or biological products with
the safety data submitted to FDA.
∑ How should a consolidated
pediatric rheumatology observational
registry be structured to collect data and
conduct analyses to meet the standards
for postmarketing requirements set by
FDA and provide information about
long-term safety?
∑ What hurdles must be overcome to
transition from product-specific
registries to a consolidated pediatric
rheumatology observational registry
(e.g., industry concerns, pediatric
rheumatology community concerns,
proprietary issues of longer term data
and informed consent, fulfilling FDA
regulatory requirements, challenges of
registry funding, management and
ownership or sharing of data)?
3. What data should be collected in a
consolidated pediatric rheumatology
observational registry? Consider the
following topics:
Database standards and terminology
(e.g., compatibility with large
databases).
Necessary and sufficient data
elements (e.g., safety, effectiveness,
growth and development, comorbidities,
tracking medication switches over time,
as well as concurrent medication).
Length of individual patients’
participation and overall duration of the
consolidated pediatric rheumatology
observational registry (e.g., managing
VerDate Nov<24>2008
20:28 Mar 25, 2009
Jkt 217001
pediatric data through and beyond the
age of consent).
4. What are the optimal methods to
analyze data from a consolidated
pediatric rheumatology observational
registry to identify safety signals? For
example, should the methods define
risk windows for attribution to a drug or
biological product; internal controls;
and/or analyses of confounding by
indication, switches in medication, and
multiple concurrent medications?
5. What are the opportunities for
research initiatives within a
consolidated observational
rheumatology registry?
III. Attendance and Registration to
Speak
There is no fee to attend the
workshop, and attendees who do not
wish to make an oral presentation do
not need to register. Seating will be on
a first-come, first-served basis.
If you would like to make an oral
presentation during the open public
session on day one of the workshop, you
must register and provide an abstract of
your presentation by close of business
on April 21, 2009. To speak, submit
your name, title, business affiliation (if
applicable), address, telephone and fax
numbers, and e-mail address to Diane
Ehrlich (see FOR FURTHER INFORMATION
CONTACT). FDA has included questions
for comment in section II of this
document. You should also identify by
number each question you wish to
address in your presentation, and the
approximate time requested for your
presentation. FDA will do its best to
accommodate requests to speak.
Individuals and organizations with
common interests are urged to
consolidate or coordinate their
presentations, and to request time for a
joint presentation. FDA will determine
the amount of time allotted to each
presenter and the approximate time that
each oral presentation is scheduled to
begin. Persons registered to make an
oral presentation should check in before
the workshop.
Ample time will be allowed during
the scheduled agenda for attendees to
ask questions of panelists. In addition,
we strongly encourage written
comments to the docket. Written or
electronic comments will be accepted
until July 14, 2009.
If you need special accommodations
because of disability, please contact
Diane Ehrlich (see FOR FURTHER
INFORMATION CONTACT) at least 7 days
before the workshop.
IV. Comments
Regardless of attendance at the public
workshop, interested persons may
PO 00000
Frm 00044
Fmt 4703
Sfmt 4703
submit written or electronic comments
to the Division of Dockets Management
(see ADDRESSES). Submit a single copy
of electronic comments or two paper
copies of any mailed comments, except
that individuals may submit one paper
copy. Comments should be identified
with the docket number found in
brackets in the heading of this
document. To ensure consideration,
submit comments by July 14, 2009 (see
DATES). Received comments may be seen
in the Division of Dockets Management
between 9 a.m. and 4 p.m., Monday
through Friday.
V. Transcript
Please be advised that as soon as a
transcript is available, it will be
accessible at https://
www.regulations.gov. It may be viewed
at the Division of Dockets Management
(see ADDRESSES). A transcript will also
be available in either hardcopy or on
CD–ROM, after submission of a
Freedom of Information request. Written
requests are to be sent to Division of
Freedom of Information (HFI–35), Office
of Management Programs, Food and
Drug Administration, 5600 Fishers
Lane, rm. 6–30, Rockville, MD 20857.
Dated: March 19, 2009.
Jeffrey Shuren,
Associate Commissioner for Policy and
Planning.
[FR Doc. E9–6709 Filed 3–25–09; 8:45 am]
BILLING CODE 4160–01–S
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
National Institutes of Health
Eunice Kennedy Shriver National
Institute of Child Health & Human
Development; Notice of Meeting
Pursuant to section 10(a) of the
Federal Advisory Committee Act, as
amended (5 U.S.C. Appendix 2), notice
is hereby given of the following
meeting.
The meeting will be open to the
public, with attendance limited to space
available. Individuals who plan to
attend and need special assistance, such
as sign language interpretation or other
reasonable accommodations, should
notify the Contact Person listed below
in advance of the meeting.
Name of Committee: National Institute of
Child Health and Human Development
Special Emphasis Panel; Gestational Diabetes
Life-Course Study.
Date: April 20, 2009.
Time: 2 p.m. to 3:30 p.m.
Agenda: To provide concept review of
proposed concept review.
E:\FR\FM\26MRN1.SGM
26MRN1
]]>Agencies
[Federal Register Volume 74, Number 57 (Thursday, March 26, 2009)]
[Notices]
[Pages 13215-13216]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: E9-6709]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2009-N-0145]
Developing a Consolidated Pediatric Rheumatology Observational
Registry; Public Workshop
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of public workshop; request for comments.
-----------------------------------------------------------------------
SUMMARY: The Food and Drug Administration (FDA) is announcing a public
workshop entitled ``Developing a Consolidated Pediatric Rheumatology
Observational Registry.'' This 2-day public workshop is intended to
seek constructive input from key stakeholders in the pediatric
rheumatology community, the pharmaceutical industry and the public to
explore the value and feasibility of developing a consolidated
pediatric rheumatology observational registry.
DATES: The public workshop will be held on May 12, 2009, from 8:30 a.m.
to 5 p.m. and on May 13, 2009, from 8:30 a.m. to noon. Register by
April 21, 2009, to make a presentation at the workshop. See section III
of this document for information on how to attend or present at the
workshop. We are opening a docket to receive your written or electronic
comments. Written or electronic comments must be submitted to the
docket by July 14, 2009.
ADDRESSES: The public workshop will be held at the Hilton Washington
DC/Silver Spring, The Ballrooms, 8727 Colesville Rd., Silver Spring, MD
20910 (Metro: Silver Spring Station on the Red Line). Submit written or
electronic requests to make a presentation to Diane Ehrlich (see FOR
FURTHER INFORMATION CONTACT).
Submit written comments to the Division of Dockets Management (HFA-
305), Food and Drug Administration, 5630 Fishers Lane, rm. 1061.
Rockville, MD 20852. Submit electronic comments to https://www.regulations.gov. All comments should be identified with the docket
number found in brackets in the heading of this document.
Transcripts of the hearing will be available for review at the
Division of Dockets Management and on the Internet at https://www.regulations.gov approximately 30 days after the workshop.
FOR FURTHER INFORMATION CONTACT: Diane Ehrlich, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 51, rm. 6190, Silver Spring, MD 20993-0002, 301-
796-3452, FAX: 301-847-8753, e-mail: Diane.Ehrlich@fda.hhs.gov.
SUPPLEMENTARY INFORMATION:
I. Background
Currently, approved drug and therapeutic biological products for
patients with juvenile idiopathic arthritis (JIA) (or juvenile
rheumatoid arthritis (JRA)) are monitored for long-term safety beyond
the information available at the time of approval on a product-by-
product basis using registries mandated by FDA's postmarketing
requirements. FDA is addressing concerns raised by individuals in the
pediatric rheumatology community about the current approach of using
product-specific pediatric rheumatology observational safety
registries. Some of the concerns expressed include the following:
1. It is difficult to capture important information from children
and adolescents whose medication is switched over time because long-
term data on these patients will not be available under the product
specific registries. Patients on ``real-life'' combinations of
medications and/or nonstandard doses are often not included in product-
specific registries.
2. Current registries do not always provide an adequate control
group to assess background rates of important adverse events.
3. The limited number of patients with JIA will make adequate
enrollment in product-specific observational registries more difficult
as the number of approved drug and biological products increases.
4. A nonproprietary registry rather than a proprietary registry
would allow wider access to the safety data that is collected.
5. A consolidated pediatric rheumatology observational registry may
allow more efficient identification of longer term safety issues in
this population.
II. Scope of Public Workshop
At the public workshop, FDA will present its current thinking on
the use of product-specific postmarketing registries to capture long-
term safety data of drug and biological products administered to
patients with JIA. Product-specific registries will be compared with a
consolidated pediatric rheumatology observational registry that could
meet the regulatory postmarketing requirements of FDA and also collect
other safety information and support potential research initiatives.
A. Objectives of the Workshop
The workshop objectives are as follows:
1. Discuss potential registry models, taking into account existing
registries for other diseases and in other countries.
2. Discuss the advantages and disadvantages of a common
consolidated registry for JIA, taking into account:
The pediatric rheumatology perspective.
The pharmaceutical company perspective.
3. Discuss methods to capture information regarding safety signals
in rare diseases.
4. Discuss the value of working through existing large pediatric
[[Page 13216]]
rheumatology collaborative networks, such as the Childhood Arthritis
and Rheumatology Research Alliance or the Pediatric Rheumatology
Collaborative Study Group.
5. Define, for phase 4 studies in JIA patients, the database
standards and elements of data collection (e.g., data quality,
monitoring) that are necessary and sufficient to meet FDA regulatory
requirements.
6. Discuss how pertinent research initiatives can be accomplished
in the framework of a consolidated JIA registry, including:
Ethical considerations.
Data sharing considerations.
7. Discuss the options for funding a consolidated JIA registry.
B. Issues for Comment
FDA is interested in obtaining public comment on the following
issues relating to development of a consolidated pediatric rheumatology
observational registry:
1. Should we transition from product-specific registries to a
consolidated pediatric rheumatology observational registry?
2. Currently, the product-specific registries are conducted by the
individual sponsors of the approved drugs and/or biological products
with the safety data submitted to FDA.
How should a consolidated pediatric rheumatology
observational registry be structured to collect data and conduct
analyses to meet the standards for postmarketing requirements set by
FDA and provide information about long-term safety?
What hurdles must be overcome to transition from product-
specific registries to a consolidated pediatric rheumatology
observational registry (e.g., industry concerns, pediatric rheumatology
community concerns, proprietary issues of longer term data and informed
consent, fulfilling FDA regulatory requirements, challenges of registry
funding, management and ownership or sharing of data)?
3. What data should be collected in a consolidated pediatric
rheumatology observational registry? Consider the following topics:
Database standards and terminology (e.g., compatibility with large
databases).
Necessary and sufficient data elements (e.g., safety,
effectiveness, growth and development, comorbidities, tracking
medication switches over time, as well as concurrent medication).
Length of individual patients' participation and overall duration
of the consolidated pediatric rheumatology observational registry
(e.g., managing pediatric data through and beyond the age of consent).
4. What are the optimal methods to analyze data from a consolidated
pediatric rheumatology observational registry to identify safety
signals? For example, should the methods define risk windows for
attribution to a drug or biological product; internal controls; and/or
analyses of confounding by indication, switches in medication, and
multiple concurrent medications?
5. What are the opportunities for research initiatives within a
consolidated observational rheumatology registry?
III. Attendance and Registration to Speak
There is no fee to attend the workshop, and attendees who do not
wish to make an oral presentation do not need to register. Seating will
be on a first-come, first-served basis.
If you would like to make an oral presentation during the open
public session on day one of the workshop, you must register and
provide an abstract of your presentation by close of business on April
21, 2009. To speak, submit your name, title, business affiliation (if
applicable), address, telephone and fax numbers, and e-mail address to
Diane Ehrlich (see FOR FURTHER INFORMATION CONTACT). FDA has included
questions for comment in section II of this document. You should also
identify by number each question you wish to address in your
presentation, and the approximate time requested for your presentation.
FDA will do its best to accommodate requests to speak. Individuals and
organizations with common interests are urged to consolidate or
coordinate their presentations, and to request time for a joint
presentation. FDA will determine the amount of time allotted to each
presenter and the approximate time that each oral presentation is
scheduled to begin. Persons registered to make an oral presentation
should check in before the workshop.
Ample time will be allowed during the scheduled agenda for
attendees to ask questions of panelists. In addition, we strongly
encourage written comments to the docket. Written or electronic
comments will be accepted until July 14, 2009.
If you need special accommodations because of disability, please
contact Diane Ehrlich (see FOR FURTHER INFORMATION CONTACT) at least 7
days before the workshop.
IV. Comments
Regardless of attendance at the public workshop, interested persons
may submit written or electronic comments to the Division of Dockets
Management (see ADDRESSES). Submit a single copy of electronic comments
or two paper copies of any mailed comments, except that individuals may
submit one paper copy. Comments should be identified with the docket
number found in brackets in the heading of this document. To ensure
consideration, submit comments by July 14, 2009 (see DATES). Received
comments may be seen in the Division of Dockets Management between 9
a.m. and 4 p.m., Monday through Friday.
V. Transcript
Please be advised that as soon as a transcript is available, it
will be accessible at https://www.regulations.gov. It may be viewed at
the Division of Dockets Management (see ADDRESSES). A transcript will
also be available in either hardcopy or on CD-ROM, after submission of
a Freedom of Information request. Written requests are to be sent to
Division of Freedom of Information (HFI-35), Office of Management
Programs, Food and Drug Administration, 5600 Fishers Lane, rm. 6-30,
Rockville, MD 20857.
Dated: March 19, 2009.
Jeffrey Shuren,
Associate Commissioner for Policy and Planning.
[FR Doc. E9-6709 Filed 3-25-09; 8:45 am]
BILLING CODE 4160-01-S
