[Federal Register Volume 78, Number 47 (Monday, March 11, 2013)]
[Notices]
[Pages 15371-15373]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2013-05562]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2012-N-0962]
Drug Development for Chronic Fatigue Syndrome and Myalgic
Encephalomyelitis; Public Workshop
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of public workshop; request for comments.
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The Food and Drug Administration (FDA), Center for Drug Evaluation
and Research, is announcing a public workshop to discuss how best to
facilitate and expedite the development of safe and effective drug
therapies to treat signs and symptoms related to chronic fatigue
syndrome (CFS) and myalgic encephalomyelitis (ME). FDA has determined
that CFS and ME are serious conditions for which there are no approved
drug treatments. On April 25, 2013, as part of FDA's Patient-Focused
Drug Development initiative, patients will provide feedback on disease
impact on quality of life and individual experience with current
treatment regimens. On April 26, 2013, there will be discussions with
academic and Government experts, patient advocates, patients, and
clinicians on how to identify sound, quantitative outcome measures that
can be used in clinical trials to determine whether disease symptoms
improve with specific drug interventions.
Date and Time: The public workshop will be held on April 25, 2013,
from 1 p.m. to 5 p.m., and on April 26, 2013, from 8:30 a.m. to 5 p.m.
[[Page 15372]]
Location: The public workshop will be held at the Bethesda
Marriott, 5151 Pooks Hill Rd., Bethesda, MD 20814, 301-897-9400, Fax:
301-897-0192.
Contact Persons:
Mary Gross, Center for Drug Evaluation and Research, Food and Drug
Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993-0002,
301-796-3519, [email protected];
Randi Clark, Center for Drug Evaluation and Research, Food and Drug
Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993-0002,
301-796-4287, [email protected]; or
Sara Eggers, Center for Drug Evaluation and Research, Food and Drug
Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993-0002,
301-796-4904, [email protected].
Registration and Requests to Participate in Panel Discussions: If
you wish to attend the public workshop or participate in a panel
discussion, you must register by submitting an electronic or written
request by 5 p.m. on April 8, 2013. Submit electronic requests to
http://mecfsmeeting.eventbrite.com. Submit written requests to Mary
Gross, Randi Clark, or Sara Eggers (see Contact Persons). You must
provide your name and business, organization, or personal affiliation
as applies (e.g., industry, government, patient). Patients who are
interested in presenting comments as part of the initial panel
discussions may indicate which topic(s) they wish to address (see
section II of this document).
The public workshop is free and seating will be on a first-come,
first-served basis. We recommend that you register early because
seating is limited. FDA may limit both the number of participants from
individual organizations and the total number of attendees, based on
space limitations. Registrants will receive confirmation once they have
been accepted to attend the meeting. For those who cannot attend in
person, a live Webcast of the meeting will be located at http://mecfsmeeting.eventbrite.com. For information about joining the meeting
via Webcast, please go to http://www.fda.gov/Drugs/NewsEvents/ucm319188.htm.
FDA will post an agenda of the public workshop and other background
material 5 days before the workshop at http://www.fda.gov/Drugs/NewsEvents/ucm319188.htm.
You may submit questions about the public workshop to [email protected] prior to the April 25 and 26 workshop dates.
If you need special accommodations because of a disability, contact
Mary Gross, Randi Clark, or Sara Eggers (see Contact Persons) at least
7 days in advance.
Comments: Submit either electronic or written comments by April 8,
2013, to receive consideration. Submit electronic comments to
www.regulations.gov. Submit 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. 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. Electronic or written comments will be accepted
after the meeting until August 2, 2013.
FDA will also hold an open public comment period on April 25 to
give the public an opportunity to comment on topics that may not have
been addressed in the discussion of topics 1 and 2 (see section II of
this document). Workshop participants should register to participate in
the open public comment period by April 8, 2013, and will be asked to
provide a brief summary of their comments.
SUPPLEMENTARY INFORMATION
I. Background
The Food and Drug Administration, Center for Drug Evaluation and
Research, is announcing a scientific workshop to discuss how best to
facilitate and expedite the development of safe and effective drug
therapies to treat signs and symptoms related to CFS and ME. FDA has
determined that CFS and ME are serious conditions for which there are
no approved drug treatments. On April 25, 2013, patients will give
feedback on disease impact on quality of life and their experiences
with current treatment regimens. On April 26, 2013, there will be
discussions with academic and Government experts, patient advocates,
patients, and clinicians on how to identify sound, quantitative outcome
measures to determine whether disease symptoms improve with specific
interventions. For purposes of this workshop, the terms ``CFS'' and
``ME'' have been used interchangeably in describing the conditions.
These terms are used as a frame of reference only. The terms are
intended to be inclusive and make no judgment on the cause of different
symptom complexes. Drug development focuses on quantitative measures of
benefit (e.g., symptom improvement) in either the entire population or
in a defined subset, not on the name of the disease. In some cases,
evaluating symptoms individually may be the optimal approach, while in
others, evaluating a constellation of symptoms may be better.
II. Purpose and Scope of the Public Workshop
FDA has selected CFS and ME to be the focus for a workshop under
the Patient-Focused Drug Development initiative, an effort that
involves obtaining a better understanding of patients' perspectives on
the severity of the disease and assessment of currently available
treatment options. Patient-Focused Drug Development is being conducted
to fulfill FDA performance commitments made as part of the
authorization of the Prescription Drug User Fee Act under Title I of
the Food and Drug Safety and Innovation Act (FDASIA) (Pub. L. 112-144).
The full set of performance commitments is available on the FDA Web
site at http://www.fda.gov/downloads/forindustry/userfees/prescriptiondruguserfee/ucm270412.pdf.
On Day 1 of the workshop (April 25, 2013) FDA will gather patients'
perspectives on CFS and ME as part of the Patient-Focused Drug
Development initiative. Day 1 will focus on two main topics: (1)
Disease symptoms and daily impacts that matter most to patients; and
(2) Patients' perspectives on current approaches to treating CFS and
ME. Discussion questions for topics 1 and 2 are as follows:
Topic 1: Disease Symptoms and Daily Impacts That Matter Most to
Patients
1. What are the most significant symptoms that you experience
resulting from your condition? (Examples may include prolonged
exhaustion, confusion, muscle pain, heat or cold intolerance.)
2. What are the most negative impacts on your daily life that
result from your condition and its symptoms? (Examples may include
difficulty with specific activities, such as sleeping through the
night.)
a. How does the condition affect your daily life on the best days
and worst days?
b. What changes have you had to make in your life because of your
condition?
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Topic 2: Patients' Perspectives on Current Approaches To Treating CFS
and ME
1. What treatments are you currently using to help treat your
condition or its symptoms? (Examples may include FDA-approved
medicines, over-the-counter products, and other therapies, including
non-drug therapies such as activity limitations.)
a. What specific symptoms do your treatments address?
b. How has your treatment regimen changed over time and why?
2. How well does your current treatment regimen treat the most
significant symptoms of your disease?
a. Have these treatments improved your daily life (for example,
improving your ability to do specific activities)? Please explain.
b. How well have these treatments worked for you as your condition
has changed over time?
c. What are the most significant downsides of these treatments (for
example, specific side effects)?
For each of these topics, a brief initial patient panel discussion
will begin the dialogue, followed by a facilitated discussion inviting
comments from other patient participants. FDA has not yet identified
the panel participants. As part of the meeting registration, patients
who are interested in presenting comments as part of the initial panel
discussions may indicate which topic(s) they wish to address and will
be asked to provide a brief summary of responses to the questions
listed below. FDA will confirm with patients who have been identified
to provide comments as part of the opening panel discussion in advance
of the workshop.
FDA will try to accommodate all participants who wish to speak on
Day 1, either through the panel discussions, audience participation, or
the open public comment period; however, the duration of comments may
be limited by time constraints. Those who are unable to attend the
meeting in person, but who would like to provide their perspective on
the discussion questions for topics 1 and 2 are invited to submit
electronic or written comments to the Division of Docket Management
(see Comments).
Day 2 of the workshop (April 26, 2013), will include a scientific
discussion on how best to facilitate and expedite the development of
safe and effective drug therapies for signs and symptoms related to CFS
and ME. Presentations and panel discussions will include the following:
Lessons learned from previous studies;
The role of drug repurposing;
Pathways to expediting drug therapies;
Appropriate clinical trial design in CFS and ME;
Outcome measures to assess efficacy; and
Potential valid endpoint measurements of symptom
improvement.
III. Transcripts
Please be advised that a transcript of the workshop will be
available for review at the Division of Dockets Management (see
Comments) and on the Internet at http://www.regulations.gov. The
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., Element Bldg.,
Rockville, MD 20857.
Dated: March 6, 2013.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2013-05562 Filed 3-8-13; 8:45 am]
BILLING CODE 4160-01-P