
[Federal Register Volume 78, Number 47 (Monday, March 11, 2013)]
[Notices]
[Pages 15371-15373]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2013-05562]


-----------------------------------------------------------------------

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.

-----------------------------------------------------------------------

    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, Mary.Gross@fda.hhs.gov;
    Randi Clark, Center for Drug Evaluation and Research, Food and Drug 
Administration, 10903 New Hampshire Ave., Silver Spring, MD 20993-0002, 
301-796-4287, Randi.Clark@fda.hhs.gov; 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, Sara.Eggers@fda.hhs.gov.
    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 ME-CFS-Meeting@fda.hhs.gov 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?

[[Page 15373]]

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


