
[Federal Register Volume 78, Number 184 (Monday, September 23, 2013)]
[Notices]
[Pages 58311-58313]
From the Federal Register Online via the Government Printing 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: Tomeka.Arnett@fda.hhs.gov.
    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]
BILLING CODE 4160-01-P


