[Federal Register Volume 75, Number 83 (Friday, April 30, 2010)]
[Notices]
[Pages 22819-22821]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2010-10079]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2010-N-0218]


Considerations Regarding Food and Drug Administration Review and 
Regulation of Articles for the Treatment of Rare Diseases; Public 
Hearing

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public hearing; request for comment.

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SUMMARY: The Food and Drug Administration (FDA) is announcing a public 
hearing regarding the Agency's regulation of drugs, biological 
products, and devices (e.g., therapies and diagnostics) for the 
treatment, diagnosis, and/or management of rare diseases. This public 
hearing is intended to gain from health care providers, academia, 
industry, patients, and other interested persons their perspectives on 
various aspects of the development of medical products for the 
diagnosis, treatment, or management of rare diseases. The input from 
this public hearing will help inform the work of FDA's committee for 
rare diseases. To help solicit such information and views, FDA is 
seeking responses to specific questions.

[[Page 22820]]


DATES:  The public hearing will be held on June 29 and 30, 2010, from 9 
a.m. to 5 p.m. However, depending on the level of public participation, 
the meeting may be extended or may end early. Submit written or 
electronic requests for oral presentations to Paras M. Patel (see FOR 
FURTHER INFORMATION CONTACT) by May 31, 2010. Submit written comments 
to the Division of Dockets Management by May 31, 2010. Submit 
electronic comments to http://www.regulations.gov by May 31, 2010. 
Written or electronic comments will be accepted after the hearing until 
August 31, 2010.

ADDRESSES: The public hearing will be held at 10903 New Hampshire Ave., 
Bldg. 31, rm. 1503, Silver Spring, MD 20993. Additional information on 
parking and public transportation may be accessed at http://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm058421.htm. (FDA has verified the Web site 
addresses throughout this document, but FDA is not responsible for any 
subsequent changes to the Web sites after this document publishes in 
the Federal Register.)
    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. Transcripts of the hearing will be available for 
review at the Division of Dockets Management at http://www.regulations.gov approximately 45 days after the hearing.

FOR FURTHER INFORMATION CONTACT: Paras M. Patel, Food and Drug 
Administration, 10903 New Hampshire Ave., Bldg. 32, rm. 5271, Silver 
Spring, MD 20993-0002, 301-796-8660, FAX: 301-847-8621, e-mail: 
[email protected].

SUPPLEMENTARY INFORMATION:

I. Background

    The development of therapies and diagnostics for people with rare 
diseases, defined as those conditions which affect fewer than 200,000 
people in the United States, presents economic and scientific 
challenges. Prior to the 1983 passage of (and subsequent amendments to) 
the Orphan Drug Act (ODA), the high development cost for therapies 
targeting few patients was often a prohibitive economic barrier; from 
1973-1982 only 12 new drugs for rare diseases were approved by FDA. 
Since the ODA's passage, 357 drugs and biological products with Orphan 
Designation have received FDA marketing approval. More modest advances 
have been made in medical devices for people with rare diseases through 
the humanitarian use device (HUD) and humanitarian device exemption 
(HDE) programs. Nevertheless for most of the estimated 7,000 rare 
diseases that affect an estimated 30 million Americans, no approved 
therapies exist.
    To optimize the means by which FDA considers articles for people 
with rare diseases, a recent public law (Agriculture, Rural 
Development, Food and Drug Administration, and Related Agencies 
Appropriations Act, 2010, Public Law 111-80, section 740) calls for the 
establishment of a committee of FDA employees to consider the means by 
which the Agency reviews the data from non-clinical studies and 
clinical trials, and makes decisions about marketing authorization and 
postmarketing surveillance for these patient populations. This 
committee, which was established March 11, 2010, is seeking public 
input to benefit from a better understanding of the opinions and 
suggestions of external stakeholders.

II. Purpose and Scope of the Hearing

    This hearing is intended to provide advocates for patients with 
rare diseases, academics, health care providers, the pharmaceutical 
industry, and other interested parties an opportunity to relate their 
experience with, concerns about, and suggestions for the way FDA 
regulates the scientific evaluation of, marketing authorization for, 
and postmarket surveillance of, articles for rare diseases. The scope 
of such presentations may include non-clinical testing, clinical 
trials, and decisions regarding marketing authorization and 
postmarketing surveillance of products for the diagnosis or treatment 
of rare diseases. FDA invites public comment from interested parties on 
the following questions/issues:
    1. Orphan drug marketing applications are reviewed under the same 
review process and statutory standards regarding demonstration of 
safety, effectiveness, and product quality as drugs for patients with 
non-orphan diseases or conditions. FDA is sensitive to the unique needs 
of patients with rare diseases as it makes approval decisions regarding 
the overall risk-benefit profile of therapies for the particular 
patient population for which they are being considered. Please comment 
on whether this practice has adequately addressed the needs of patients 
with rare diseases. If improvements are suggested, please provide 
specific examples/suggestions for any recommended changes.
    2. FDA designates a medical device as an HUD designed to treat or 
diagnose a rare disease--defined in this instance as a disease 
affecting or manifesting in fewer than 4,000 patients per year. Please 
comment on whether this practice has adequately addressed the needs of 
patients with rare diseases. Please also comment and provide your 
rationale on whether 4,000 patients constitutes an appropriate 
population size for an HUD determination. If improvements are 
suggested, please provide specific examples/suggestions for any 
recommended changes.
    3. Current regulations for the approval of an HUD through the HDE 
pathway require that the application have a ``description of the device 
and a discussion of the scientific rationale for the use of the device 
for the rare disease or condition'' and ``an explanation of why the 
probable benefit to health from the use of the device outweighs the 
risk of injury or illness from its use, taking into account the 
probable risks and benefits of currently available devices or 
alternative forms of treatment'' (21 CFR 814.102 and 814.104). Please 
comment if you believe that these standards remain appropriate for the 
approval of devices for rare diseases under the HDE mechanism; please 
also comment whether a more precise definition of probable benefit is 
needed.
    4. Have current processes for rare disease stakeholders to 
communicate with FDA regarding rare disease article development been 
useful? How could these processes be improved? Please provide specific 
examples/suggestions for any recommended changes.

III. Attendance and/or Participation in the Public Hearing

    The public hearing is free and seating will be on a first-come, 
first-served basis. Attendees who do not wish to make an oral 
presentation do not need to register.
    If you wish to make an oral presentation during the hearing, you 
must register by submitting a written or electronic request by close of 
business on May 31, 2010, to Paras M. Patel (see FOR FURTHER 
INFORMATION CONTACT). You must provide your name, title, business 
affiliation (if applicable), address, telephone and fax numbers, e-mail 
address, and type of organization you represent (e.g., industry, 
consumer organization). You also should submit a brief summary of the 
presentation, including the discussion topic(s) that will be addressed 
and the approximate time requested for your presentation. We encourage 
individuals and

[[Page 22821]]

organizations with common interests to consolidate or coordinate their 
presentations to allow adequate time for each request for presentation. 
Persons registered to make an oral presentation should check in before 
the hearing.
    Participants should submit a copy of each presentation to Paras M. 
Patel. We will file the hearing schedule, indicating the order of 
presentation and the time allotted to each person, with the Division of 
Dockets Management (see ADDRESSES). We will mail, e-mail, or fax the 
schedule to each participant before the hearing. Participants are 
encouraged to arrive early to ensure the designated order of 
presentation.
    If you need special accommodations due to a disability, please 
contact Paras M. Patel at least 14 days in advance.

IV. Notice of Hearing Under 21 CFR Part 15

    The Commissioner of Food and Drugs is announcing that the public 
hearing will be held in accordance with part 15 (21 CFR part 15). The 
hearing will be conducted by a presiding officer, accompanied by FDA 
senior management from the Office of the Commissioner, the Office of 
Orphan Products Development, as well as representatives from the 
committee established by section 740 of the Agriculture, Rural 
Development, Food and Drug Administration, and Related Agencies 
Appropriations Act, 2010.
    Under paragraph Sec.  15.30(f), the hearing is informal, and the 
rules of evidence do not apply. No participant may interrupt the 
presentation of another participant. Only the presiding officer and 
panel members may question any person during or at the conclusion of 
each presentation (Sec.  15.30(e)).
    Public hearings under part 15 are subject to FDA's policy and 
procedures for electronic media coverage of FDA's public administrative 
proceedings (21 CFR 10.203(a)). Under 21 CFR 10.205, representatives of 
the electronic media may be permitted, subject to certain limitations, 
to videotape, film, or otherwise record FDA's public administrative 
proceedings, including presentations by participants.
    The hearing will be transcribed as stipulated in paragraph Sec.  
15.30(b). 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 45 days after the hearing. A 
transcript will also be available in either hardcopy or on a CD-ROM 
after submission of a Freedom of Information request. Submit written 
requests to the Division of Freedom of Information (HFI-35), Office of 
Management Programs, Food and Drug Administration, 5600 Fishers Lane, 
rm. 6-30, Rockville, MD 20857.
    To the extent that the conditions for the hearing, as described in 
this document, conflict with any provisions set out in part 15, this 
notice acts as a waiver of those provisions as specified in Sec.  
15.30(h).

V. Request for Comments

    Interested persons may submit written or electronic comments for 
consideration to the Division of Dockets Management (see ADDRESSES). 
Persons who wish to provide additional materials for consideration 
should file these materials with the Division of Dockets Management. 
You should annotate and organize your comments to identify the specific 
questions identified by topic to which they refer. 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. Received comments may be seen in the Division of Dockets 
Management between 9 a.m. and 4 p.m. Monday through Friday.

    Dated: April 26, 2010.
Leslie Kux,
Acting Assistant Commissioner for Policy.
[FR Doc. 2010-10079 Filed 4-29-10; 8:45 am]
BILLING CODE 4160-01-S