[Federal Register Volume 78, Number 184 (Monday, September 23, 2013)]
[Notices]
[Pages 58311-58313]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2013-22959]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2013-N-0985]
Complex Issues in Developing Drug and Biological Products for
Rare Diseases; Public Workshop; Request for Comments
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of public workshop; request for comments.
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The Food and Drug Administration (FDA) is announcing the following
public workshop entitled ``Complex Issues in Developing Drug and
Biological Products for Rare Diseases.'' The purpose of the public
workshop is twofold: To discuss complex issues in clinical trials for
developing drug and biological products (``drugs'') for rare diseases,
including endpoint development and selection, use of surrogate
endpoints and the accelerated approval pathway, clinical trial design,
conduct and analysis, safety considerations, and dose selection; and to
discuss ways to encourage and accelerate the development of new
therapies for pediatric rare diseases. FDA is seeking input on these
topics from academic, clinical, and treating communities; patients and
advocacy groups; industry; and governmental agencies. Input from this
public workshop will help develop a strategic plan to encourage and
accelerate the development of new therapies for rare diseases.
Date and Time: The public workshop will be held on January 6, 2014,
from 8 a.m. to 5 p.m. and on January 7, 2014, from 8 a.m. to 4:45 p.m.
Location: The public workshop will be held at FDA's White Oak
Campus, 10903 New Hampshire Ave., Building 31 Conference Center, the
Great Room (Rm. 1503), Silver Spring, MD 20993-0002. Entrance for the
public meeting participants (non-FDA employees) is through Building 1
where routine security check procedures will be performed. For parking
and security information, please refer to http://
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www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/
WhiteOakCampusInformation/ucm241740.htm.
Contact Person: Tomeka Arnett, Center for Drug Evaluation and
Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg.
51, Rm. 6331, Silver Spring, MD 20993-0002, 301-796-2500, FAX: 301-847-
3529, email: [email protected].
Registration: Registration is free and available on a first-come,
first-served basis. Persons interested in attending the public workshop
must register online by December 20, 2013. Early registration is
recommended because facilities are limited and, therefore, FDA may
limit the number of participants from each organization. If time and
space permits, onsite registration on the day of the public workshop
will be provided beginning at 7:30 a.m. Seating will be available on a
first-come, first-served basis.
If you need special accommodations due to a disability, please
contact Tomeka Arnett (see Contact Person) no later than 7 days in
advance.
To register for the public workshop, please visit FDA's Drugs News
& Events--Meetings, Conferences & Workshops calendar at http://www.fda.gov/Drugs/NewsEvents/ucm132703.htm. (Select this public
workshop from the posted events list.) Please provide complete contact
information for each attendee, including name, title, affiliation,
address, email, and telephone number. Those without Internet access
should contact Tomeka Arnett to register (see Contact Person).
Registrants will receive confirmation after they have been accepted.
You will be notified if you are on a waiting list.
Streaming Webcast of the Public Workshop: This public workshop will
also be Webcast. Persons interested in viewing the Webcast may visit
FDA's Drugs News & Events--Meetings, Conferences & Workshops calendar
at http://www.fda.gov/Drugs/NewsEvents/ucm132703.htm. (Select this
public workshop from the posted events list.) Select https://collaboration.fda.gov/drugbiord/ to view the Webcast. If you have never
attended a Connect Pro event before, test your connection at https://collaboration.fda.gov/common/help/en/support/meeting_test.htm. (FDA
has verified the Web site addresses in this document, but FDA is not
responsible for any subsequent changes to the Web sites after this
document publishes in the Federal Register.)
Comments: FDA is holding this public workshop to obtain information
about complex issues in clinical trials for developing drugs for rare
diseases and to discuss ways to encourage and accelerate the
development of new therapies for pediatric rare diseases. In order to
permit the widest possible opportunity to obtain public comment, FDA is
soliciting either electronic or written comments on all aspects of the
public workshop. The deadline for submitting comments regarding this
public workshop is March 10, 2014.
Regardless of attendance at the public workshop, interested persons
may submit either electronic comments regarding this document to http://www.regulations.gov or written comments to the Division of Dockets
Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane,
Rm. 1061, Rockville, MD 20852. It is only necessary to send one set of
comments. Identify comments with the docket number found in brackets in
the heading of this document. In addition, when responding to specific
topics as outlined in section II, please identify the topic you are
addressing. Received comments may be seen in the Division of Dockets
Management between 9 a.m. and 4 p.m., Monday through Friday, and will
be posted to the docket at http://www.regulations.gov.
Transcripts: Please be advised that as soon as a transcript is
available, it will be accessible at http://www.regulations.gov. 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 the Division of Freedom of Information (ELEM-1029),
Food and Drug Administration, 12420 Parklawn Dr., Rockville, MD 20857.
SUPPLEMENTARY INFORMATION:
I. Background
The Orphan Drug Act of 1983 (the Orphan Drug Act) (Pub. L. 97-414),
as amended, defines a ``rare disease or condition'' to include those
that affect less than 200,000 persons in the United States. This
definition is codified in section 526(a)(2) of the Federal Food, Drug,
and Cosmetic Act (21 U.S.C. 360bb(a)(2)). The Orphan Drug Act provides
incentives to reduce the cost and increase the potential reward for
developing products for small numbers of patients; however, it does not
alter the statutory standards for marketing approval. To gain approval,
all drugs must demonstrate substantial evidence of effectiveness,
safety, and product quality for the treatment of the condition in the
identified patient population. FDA acknowledges that certain aspects of
drug development for rare diseases are challenging, and U.S.
regulations allow for flexibility and scientific judgment in applying
approval standards and in determining the kind and quantity of data
required for a particular drug to meet the statutory standards.
This public workshop is being held in response to section 510--
Pediatric rare diseases of the Food and Drug Administration Safety and
Innovation Act (Pub. L. 122-144) (125 Stat. 1050), whereby FDA is
required to hold at least one public meeting to discuss ways to
encourage and accelerate the development of new therapies for pediatric
rare diseases. Additionally, as stated in section IX.E--Enhancing
Regulatory Science and Expediting Drug Development, Advancing
Development of Drugs for Rare Diseases of the Prescription Drug User
Fee Act Reauthorization Performance Goals and Procedures Fiscal Years
2013 through 2017 (available at http://www.fda.gov/downloads/forindustry/userfees/prescriptiondruguserfee/ucm270412.pdf ), FDA will
conduct a public meeting to discuss complex issues in clinical trials
for studying drugs for rare diseases.
This public workshop is being held in conjunction with FDA's Center
for Devices and Radiological Health and Office of Orphan Products
Development public workshop entitled ``Complex Issues in Developing
Medical Devices for Pediatric Patients Affected by Rare Diseases,''
which will be held on January 8, 2014, from 8 a.m. to 5 p.m., announced
in a separate notice publishing elsewhere in this issue of the Federal
Register.
II. Topics for Discussion at the Public Workshop
FDA is announcing a public workshop regarding complex issues in
clinical trials for developing drugs for rare diseases and to discuss
ways to encourage and accelerate the development of new therapies for
pediatric rare diseases. The purpose of this public workshop is to seek
broad input from rare disease experts and stakeholders, including
industry; academic and clinical experts; patients and advocates and
governmental agencies to address complex issues in rare disease product
development.
Topics for discussion on day 1 include: (1) Complex issues for
endpoints, including endpoint selection, use of surrogate endpoints and
the accelerated approval pathway, clinical significance of primary
endpoints, and development of patient-reported outcome instruments; (2)
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complex issues for trial design conduct and analysis; (3) development
of translational and regulatory science to support rare disease drug
development; and (4) safety and dosing considerations, including safety
exposures and assessment of dose selection.
Topics for discussion on day 2 include: (1) Collaborative research
networks for pediatric rare diseases; (2) safety considerations for
pediatric rare diseases; (3) pediatric rare cancers; and (4)
development of gene therapies for rare pediatric disorders. Discussions
will help develop a report that includes a strategic plan to encourage
and accelerate the development of new therapies for pediatric rare
diseases.
FDA encourages individuals, patients, advocates, industry, consumer
groups, health care professionals, researchers and other interested
persons to attend this public workshop.
Dated: September 17, 2013.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2013-22959 Filed 9-20-13; 8:45 am]
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