Document ID: FDA-2013-N-0035-0001
Agency: fda
Document Type: Notice
Title: Considerations Regarding Food and Drug Administration Review and Regulation of Drugs for the Treatment of Amyotrophic Lateral Sclerosis; Public Hearing
Posted Date: 2013-01-31T05:00Z

[Federal Register Volume 78, Number 21 (Thursday, January 31, 2013)]
[Notices]
[Pages 6824-6825]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2013-02088]

[[Page 6824]]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2013-N-0035]

Considerations Regarding Food and Drug Administration Review and 
Regulation of Drugs for the Treatment of Amyotrophic Lateral Sclerosis; 
Public Hearing

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public hearing; request for comments.

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SUMMARY: The Food and Drug Administration (FDA or the Agency) is 
announcing a public hearing to obtain input on the Agency's regulation 
of drugs for the treatment and/or management of amyotrophic lateral 
sclerosis (ALS). FDA is holding this public hearing to allow patients, 
caregivers, advocates, health care providers, academia, industry, and 
other interested persons to give their perspectives on various aspects 
of the development of drugs for the treatment or management of ALS. The 
input from this public hearing will help inform the work of FDA offices 
that review applications for drugs for the treatment of ALS.

DATES: Public Hearing: The public hearing will be held on February 25, 
2013, from 9 a.m. to 5 p.m. However, depending on the level of public 
participation, the meeting may be extended or end early.
    Presentations and Comments: Submit either electronic or written 
requests for oral presentations to David Banks or Steve Morin (see FOR 
FURTHER INFORMATION CONTACT) by February 8, 2013. Submit electronic 
comments to http://www.regulations.gov by March 25, 2013. Submit 
written comments to the Division of Dockets Management (see ADDRESSES) 
by March 25, 2013. Either electronic or written comments will be 
accepted after the hearing until March 25, 2013.

ADDRESSES: The public hearing will be held at FDA White Oak Campus, 
10903 New Hampshire Ave., Bldg. 31, Rm. 1503B, Silver Spring, MD 20993-
0002. Additional information on parking and public transportation may 
be accessed at http://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.
    Comments and Transcripts: Submit either electronic or 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 and on the Internet at http://www.regulations.gov 
approximately 45 days after the hearing.

FOR FURTHER INFORMATION CONTACT: David Banks, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 32, Rm. 5365, Silver Spring, MD 20993-0002, 301-
796-8459, Email: david.banks@fda.hhs.gov, or Steve Morin, Center for 
Drug Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg 32, Rm. 5343, Silver Spring, MD 20993-0002, 301-
796-0161, Email: steve.morin@fda.hhs.gov.

SUPPLEMENTARY INFORMATION:

I. Background

    ALS, sometimes called Lou Gehrig's disease, is a rapidly 
progressive, invariably fatal neurological disease that attacks the 
nerve cells (neurons) responsible for controlling voluntary muscles. 
Messages from motor neurons in the brain (called ``upper motor 
neurons'') are transmitted to motor neurons in the spinal cord (called 
``lower motor neurons'') and from them to particular muscles. In ALS, 
both the upper motor neurons and the lower motor neurons degenerate or 
die, ceasing to send messages to muscles. Unable to function, the 
muscles gradually weaken, waste away (atrophy), and twitch 
(fasciculations). Eventually, the ability of the brain to start and 
control voluntary movement is lost.
    ALS causes weakness with a wide range of disabilities. Eventually, 
all muscles under voluntary control are affected, and patients lose 
their strength and the ability to move their arms, legs, and body. When 
muscles in the diaphragm and chest wall fail, patients lose the ability 
to breathe without ventilatory support. Most people with ALS die from 
respiratory failure, usually within 3 to 5 years from the onset of 
symptoms. However, about 10 percent of ALS patients survive for 10 or 
more years.
    As many as 20,000 people in the United States have ALS, and an 
estimated 5,000 people in the United States are diagnosed with the 
disease each year. People of all races and ethnic backgrounds are 
affected. ALS most commonly strikes people between 40 and 60 years of 
age, but younger and older people also can develop the disease.
    In 90 to 95 percent of all ALS cases, the disease occurs apparently 
at random with no clearly associated risk factors. Patients do not have 
a family history of the disease, and their family members are not 
considered to be at increased risk for developing ALS.
    No cure has yet been found for ALS. However, several years ago FDA 
approved the first drug treatment for the disease, RILUTEK (riluzole). 
Clinical trials with ALS patients showed that RILUTEK prolongs survival 
by several months. Patients taking RILUTEK must be monitored for liver 
damage and other possible side effects. However, this first disease-
specific therapy offers hope that new medications or combinations of 
drugs may one day slow the progression of ALS.
    Profound unmet medical needs remain for patients with ALS. Patients 
need new treatments to provide symptomatic relief, and to slow, halt, 
reverse, or prevent ALS. In addition to fulfilling responsibilities to 
regulate clinical testing and marketing of new treatments for ALS, FDA 
facilitates the work of researchers and medical product manufacturers 
by providing expert technical assistance.
    The purpose of this meeting is for FDA to hear from stakeholders 
regarding the needs and preferences of patients, as well as suggestions 
regarding how best to be responsive to those needs and preferences.

II. Purpose and Scope of the Hearing

    FDA is holding this hearing to seek input from ALS patients, 
caregivers, advocates, academia, health care providers, the 
pharmaceutical industry, and other interested parties on their 
experience with, concerns about, and suggestions for, the way FDA 
regulates the scientific evaluation of, marketing authorization for, 
and postmarketing surveillance of, drugs for treatment of ALS. The 
scope of the presentations may include, but are not limited to, 
nonclinical testing, clinical trials, and decisions regarding marketing 
authorization and postmarketing surveillance of products for the 
diagnosis or treatment of this disease.

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. Given the debilitating effects of 
ALS, FDA will employ all available measures to enable participation of 
people who are mobility-limited.

[[Page 6825]]

    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 February 8, 2013, to David Banks or Steve Morin (see FOR 
FURTHER INFORMATION CONTACT). You must provide your name, title, 
business affiliation (if applicable), address, telephone and fax 
numbers, email 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 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 David 
Banks or Steve Morin (see FOR FURTHER INFORMATION CONTACT). 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, email, 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 David Banks or Steve Morin (see FOR FURTHER INFORMATION 
CONTACT) 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, and the Center 
for Drug Evaluation and Research.
    Under 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 (part 10 (21 CFR part 10), subpart C) (Sec.  10.203(a)). 
Under Sec.  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.
    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 either electronic comments for 
consideration to http://www.regulations.gov or written comments 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 topics to which 
they refer. 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. Received comments may be seen in the Division of Dockets 
Management between 9 a.m. and 4 p.m., Monday through Friday.

VI. Transcripts

    The hearing will be transcribed as stipulated in Sec.  15.30(b). 
Transcripts of the hearing will be available for review at the Division 
of Dockets Management (see ADDRESSES) and on the Internet at http://www.regulations.gov approximately 45 days after the hearing. A 
transcript will also be available in either hard copy or on a 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., Element Bldg., 
Rockville, MD 20857.

    Dated: January 24, 2013.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2013-02088 Filed 1-30-13; 8:45 am]
BILLING CODE 4160-01-P