Drug Development for Chronic Fatigue Syndrome and Myalgic Encephalomyelitis; Public Workshop, 15371-15373 [2013-05562]
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Federal Register / Vol. 78, No. 47 / Monday, March 11, 2013 / Notices
Administration, 10903 New Hampshire
Ave., Bldg. 66, Rm. G120, Silver Spring,
MD 20993–0002, 301–796–6530,
Michael.Bailey@fda.hhs.gov.
SUPPLEMENTARY INFORMATION:
I. Background
Contact with devices containing
natural rubber has been associated with
anaphylaxis in individuals allergic to
natural rubber latex proteins. FDA
medical device regulations include
provisions that require certain labeling
statements on medical devices if the
device or device packaging is composed
of or contains natural rubber that
contacts humans. (See 21 CFR 801.437.)
The biological products regulations
require that the package label or package
insert declare the presence of known
sensitizing substances, but do not
specifically mention natural rubber
latex (21 CFR 610.61(l)). Specific
regulations for labeling of natural rubber
latex content in medical products or
their containers do not exist for drugs or
veterinary products.
At this time, there are no regulations
requiring the labeling of a medical
product to state that natural rubber latex
was not used as a material in the
manufacture of a medical product or
medical product container. However,
some manufacturers have included the
promotional statements ‘‘latex-free’’ or
‘‘does not contain latex’’ in medical
product labeling to inform users that
natural rubber latex, dry natural rubber,
or synthetic derivatives of natural
rubber latex were not used. These
labeling statements are not sufficiently
specific, not necessarily scientifically
accurate and may be misunderstood or
applied too widely, and therefore, it is
inappropriate to include such
statements in medical product labeling.
Use of these terms may give users
allergic to natural rubber latex a false
sense of security when using a medical
product. The draft guidance provides
recommendations for scientifically
accurate labeling that can be used by
manufacturers who wish to convey that
natural rubber latex was not used as a
material in the manufacture of a medical
product or medical product container.
mstockstill on DSK4VPTVN1PROD with NOTICES
II. Significance of Guidance
This draft guidance document is being
issued consistent with FDA’s good
guidance practices regulation (21 CFR
10.115). The draft guidance, when
finalized, will represent the Agency’s
current thinking on labeling medical
products to inform users that a product
or product container was not made with
natural rubber latex. It does not create
or confer any rights for or on any person
VerDate Mar<15>2010
16:19 Mar 08, 2013
Jkt 229001
and does not operate to bind FDA or the
public. An alternative approach may be
used if such approach satisfies the
requirements of the applicable statute
and regulations.
III. Electronic Access
Persons interested in obtaining a copy
of the draft guidance may do so by using
the Internet. A search capability for all
CDRH guidance documents is available
at https://www.fda.gov/MedicalDevices/
DeviceRegulationandGuidance/
GuidanceDocuments/default.htm.
Guidance documents also are available
at https://www.regulations.gov. To
receive ‘‘Draft Guidance for Industry
and FDA Staff: Recommendations for
Labeling Medical Products to Inform
Users That the Product or Product
Container Is Not Made With Natural
Rubber Latex,’’ you may either send an
email request to dsmica@fda.hhs.gov for
an electronic copy of the document or
send a fax request to 301–847–8149 to
receive a hard copy. Please use the
document number 1768 to identify the
guidance you are requesting.
IV. Paperwork Reduction Act of 1995
This draft guidance refers to currently
approved collections of information
found in FDA regulations. These
collections of information are subject to
review by the Office of Management and
Budget (OMB) under the Paperwork
Reduction Act of 1995 (44 U.S.C. 3501–
3520) (the PRA). The collections of
information in 21 CFR part 801 are
approved under OMB control number
0910–0485 and the collections of
information in 21 CFR part 610 subpart
G are approved under OMB control
number 0910–0338.
The labeling provisions recommended
in this draft guidance are not subject to
review by OMB because they do not
constitute a ‘‘collection of information’’
under the PRA. Rather, the
recommended labeling is a ‘‘public
disclosure of information originally
supplied by the Federal government to
the recipient for the purpose of
disclosure to the public’’ (5 CFR
1320.3(c)(2)).
V. Comments
Interested persons may submit either
electronic comments regarding this
document to https://www.regulations.gov
or written comments to the Division of
Dockets Management (see ADDRESSES). 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
PO 00000
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15371
will be posted to the docket at https://
www.regulations.gov.
VI. Reference
The following reference has been
placed on display in the Division of
Dockets Management (see ADDRESSES)
and may be seen by interested persons
between 9 a.m. and 4 p.m., Monday
through Friday, and are available
electronically at https://
www.regulations.gov.
1. Ahmed, S.M., T.C. Aw, and A.
Adisesh, ‘‘Toxicological and
Immunological Aspects of Occupational
Latex Allergy,’’ Toxicological Reviews,
vol. 23, pp. 123–134, 2004.
Dated: March 5, 2013.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2013–05554 Filed 3–8–13; 8:45 am]
BILLING CODE 4160–01–P
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.
Notice of public workshop;
request for comments.
ACTION:
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 PatientFocused 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.
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15372
Federal Register / Vol. 78, No. 47 / Monday, March 11, 2013 / Notices
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 https://
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, firstserved 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 https://
mecfsmeeting.eventbrite.com. For
information about joining the meeting
via Webcast, please go to https://
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
https://www.fda.gov/Drugs/NewsEvents/
ucm319188.htm.
You may submit questions about the
public workshop to ME-CFS-
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16:19 Mar 08, 2013
Jkt 229001
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 https://
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
PO 00000
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Fmt 4703
Sfmt 4703
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. PatientFocused 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 https://
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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Federal Register / Vol. 78, No. 47 / Monday, March 11, 2013 / Notices
mstockstill on DSK4VPTVN1PROD with NOTICES
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-thecounter 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;
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16:19 Mar 08, 2013
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• 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 https://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
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
National Institutes of Health
Center for Scientific Review; Notice of
Closed Meeting
Pursuant to section 10(d) of the
Federal Advisory Committee Act, as
amended (5 U.S.C. App.), notice is
hereby given of the following meeting.
The meeting will be closed to the
public in accordance with the
provisions set forth in sections
552b(c)(4) and 552b(c)(6), Title 5 U.S.C.,
as amended. The grant applications and
the discussions could disclose
confidential trade secrets or commercial
property such as patentable material,
and personal information concerning
individuals associated with the grant
applications, the disclosure of which
would constitute a clearly unwarranted
invasion of personal privacy.
Name of Committee: Center for Scientific
Review Special Emphasis Panel; ODCS Small
Business.
Date: March 13–14, 2013.
Time: 8:00 a.m. to 5:00 p.m.
Agenda: To review and evaluate grant
applications.
Place: National Institutes of Health, 6701
Rockledge Drive, Bethesda, MD 20892,
(Virtual Meeting).
Contact Person: Yi-Hsin Liu, Ph.D.,
Scientific Review Officer, Center for
Scientific Review, National Institutes of
PO 00000
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Fmt 4703
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15373
Health, 6701 Rockledge Drive, Room 4214,
MSC 7814, Bethesda, MD 20892, 301–435–
1781, liuyh@csr.nih.gov.
This notice is being published less than 15
days prior to the meeting due to the timing
limitations imposed by the review and
funding cycle.
(Catalogue of Federal Domestic Assistance
Program Nos. 93.306, Comparative Medicine;
93.333, Clinical Research, 93.306, 93.333,
93.337, 93.393–93.396, 93.837–93.844,
93.846–93.878, 93.892, 93.893, National
Institutes of Health, HHS)
Dated: March 5, 2013.
Michelle Trout,
Program Analyst, Office of Federal Advisory
Committee Policy.
[FR Doc. 2013–05511 Filed 3–8–13; 8:45 am]
BILLING CODE 4140–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
National Institutes of Health
Center for Scientific Review; Notice of
Closed Meetings
Pursuant to section 10(d) of the
Federal Advisory Committee Act, as
amended (5 U.S.C. App.), notice is
hereby given of the following meetings.
The meetings will be closed to the
public in accordance with the
provisions set forth in sections
552b(c)(4) and 552b(c)(6), Title 5 U.S.C.,
as amended. The grant applications and
the discussions could disclose
confidential trade secrets or commercial
property such as patentable material,
and personal information concerning
individuals associated with the grant
applications, the disclosure of which
would constitute a clearly unwarranted
invasion of personal privacy.
Name of Committee: Center for Scientific
Review Special Emphasis Panel; Member
Conflict: Hematology and Vascular
Pathobiology.
Date: April 1–2, 2013.
Time: 10:00 a.m. to 7:00 p.m.
Agenda: To review and evaluate grant
applications.
Place: National Institutes of Health, 6701
Rockledge Drive, Bethesda, MD 20892
(Virtual Meeting).
Contact Person: Ai-Ping Zou, MD, Ph.D.,
Scientific Review Officer, Center for
Scientific Review, National Institutes of
Health, 6701 Rockledge Drive, Room 4118,
MSC 7814, Bethesda, MD 20892, 301–408–
9497, zouai@csr.nih.gov.
Name of Committee: Center for Scientific
Review Special Emphasis Panel; Member
Conflict: AIDS and AIDS Related Research.
Date: April 1, 2013.
Time: 12:00 p.m. to 3:00 p.m.
Agenda: To review and evaluate grant
applications.
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]]>Agencies
[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
https://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 https://mecfsmeeting.eventbrite.com. For information about joining the meeting
via Webcast, please go to https://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 https://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 https://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 https://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 https://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
