[Federal Register Volume 74, Number 57 (Thursday, March 26, 2009)]
[Pages 13215-13216]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: E9-6709]



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 
    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 http://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 http://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.


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 
    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 
    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 http://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]