[Federal Register Volume 78, Number 215 (Wednesday, November 6, 2013)]
[Notices]
[Pages 66747-66748]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2013-26548]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-N-2013-1328]
Sickle Cell Disease Public Meeting on Patient-Focused Drug
Development
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of public meeting; request for comments.
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SUMMARY: The Food and Drug Administration (FDA) is announcing a public
meeting and an opportunity for public comment on Patient-Focused Drug
Development for sickle cell disease. Patient-Focused Drug Development
is part of FDA's performance commitments in the fifth authorization of
the Prescription Drug User Fee Act (PDUFA V). The public meeting is
intended to allow FDA to obtain patients' perspectives on the impact of
sickle cell disease on daily life and on available therapies for sickle
cell disease.
DATES: The public meeting will be held on February 7, 2014; from 10
a.m. to 4 p.m. Registration to attend the meeting must be received by
January 27, 2014. See the SUPPLEMENTARY INFORMATION section for
information on how to register for the meeting. Submit electronic or
written comments by April 8, 2014.
ADDRESSES: The meeting will be held at the FDA White Oak Campus, 10903
New Hampshire Ave., Bldg. 31 Conference Center, in Sections B and C of
the Great Room (Rm. 1503), Silver Spring, MD 20993. Entrance for the
public meeting participants is through Building 1, where routine
security check procedures will be performed. For more information on
parking and security procedures, please refer to http://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.
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. All
comments should be identified with the docket number found in brackets
in the heading of this document.
FDA will post the agenda approximately 5 days before the meeting
at: http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm370867.htm.
FOR FURTHER INFORMATION CONTACT: Graham Thompson, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 51, Rm. 1199, Silver Spring, MD 20993, 301-796-
5003, Fax: 301-847-8443, email: [email protected].
SUPPLEMENTARY INFORMATION:
I. Background on Patient-Focused Drug Development
FDA has selected sickle cell disease to be the focus of a meeting
under Patient-Focused Drug Development, an initiative that involves
obtaining a better understanding of patients' perspectives on the
severity of the disease and the available therapies for the condition.
Patient-Focused Drug Development is being conducted to fulfill FDA's
performance commitments made as part of the authorization of PDUFA V
under Title I of the Food and Drug Safety and Innovation Act (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.
FDA has committed to obtain the patient perspective in 20 disease
areas during the course of PDUFA V. For each disease area, the Agency
will conduct a public meeting to discuss the disease and its impact on
patients' daily lives, the types of treatment benefit that matter most
to patients, and patients' perspectives on available therapies for
sickle cell disease. These meetings will include participation of FDA
review divisions, the relevant patient community, and other interested
stakeholders.
On April 11, 2013, FDA published a notice (78 FR 21613) in the
Federal Register announcing the disease areas for meetings in fiscal
years (FYs) 2013 through 2015, the first 3 years of the 5-year PDUFA V
timeframe. To develop the list of disease areas, the Agency used
several criteria that were outlined in the April 2013 notice. The
Agency obtained public comment on these criteria and potential disease
areas through a notice for public comment published in the Federal
Register on September 24, 2012 (77 FR 58849), and through a public
meeting held on October 25, 2012. In selecting the disease areas, FDA
carefully considered the public comments received and the perspectives
of its review divisions. By the end of FY 2015, FDA will initiate
another public process for determining the disease areas for FYs 2016
and 2017. More information, including the list of disease areas and a
general schedule of meetings, is posted on FDA's Web site at http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm326192.htm.
II. Public Meeting Information
A. Purpose and Scope of the Meeting
As part of Patient-Focused Drug Development, FDA will obtain
patient and patient stakeholder input on sickle cell disease and on
current approaches to treatment. Approximately 100,000 people in the
United States, and millions of people worldwide, have sickle cell
disease. Sickle cell disease is an inherited red blood cell disorder
resulting from a mutation in the beta globin gene. Red blood cells are
more prone to an abnormal shape and rigidity, causing multi-organ
damage over time. Some of the effects of sickle cell disease are
painful crises, increased risk of infections, stroke, pulmonary
hypertension, acute chest syndrome, recurrent priapism, gallstones, and
kidney dysfunction.
Therapies to prevent the complications of sickle cell disease are
limited and can include prescription medications and blood
transfusions. Bone marrow transplantation is an option for some
patients. Other therapies, such as pain medications, antibiotics,
supplemental oxygen, and vitamin supplements, are used to manage
specific health effects of the disease. New approaches to treating
sickle cell disease or preventing its complications are being explored,
including new medications, advances in transplantation, and gene
therapies. FDA is interested in obtaining a better understanding of
patients' perspectives on sickle cell disease, including the symptoms
that matter most to patients, limitations to current treatment
approaches, opportunities for new treatment approaches, and specific
considerations regarding sickle cell disease in pediatric patients.
The questions that will be asked of patients and patient
stakeholders at the meeting are listed in this section, organized by
topic. For each topic, a brief patient panel discussion will begin the
dialogue, followed by a facilitated discussion inviting comments from
other patient and patient stakeholder participants. In addition to
input generated through this public meeting, FDA is interested in
receiving patient input addressing these questions
[[Page 66748]]
through written comments that can be submitted to the public docket
(see ADDRESSES). When submitting comments to the docket, please provide
some context to your comment by indicating whether you are an
adolescent or young adult, or older adult. If you are commenting on
behalf of a child or other loved one who has sickle cell disease,
please indicate that and answer the following questions as much as
possible from the patient's perspective.
Topic 1: The Effects of Sickle Cell Disease That Matter Most to You
1. Of all of the ways that sickle cell disease affects your health,
which one to three effects have the greatest impact on your life?
(Examples may include pain crises, breathing problems, difficulty
concentrating, tiredness, infections, and others.)
2. How does sickle cell disease affect your life on an ``average''
day?
a. Are there activities that you cannot do at all or as well as you
would like on these ``average'' days? Please describe, using specific
examples. (Examples may include sleeping through the night,
concentrating at work or at school, participating in physical
activities, and others.)
3. How does sickle cell disease affect your life on the ``worst''
days, such as days when you have a pain crisis or have to be
hospitalized for some reason?
a. Are there activities that you cannot do at all or as well as you
would like on these ``worst'' days? Please describe, using specific
examples.
4. What worries you most about how sickle cell disease could affect
your health in the future?
5. What specific concerns do you have about sickle cell disease:
a. In infants and young children?
b. In adolescents and young adults?
c. In older adults?
Topic 2: Perspectives on Treatments for Sickle Cell Disease
1. Are you currently using any prescription medicines or medical
treatments to prevent or treat any negative effects of your sickle cell
disease? Please describe these treatments, which may include blood
transfusions, supplemental oxygen and prescription medications such as
hydroxyurea, antibiotics, pain medications, and others.
a. How well do these treatments work for you? For example, how well
do they reduce your number of pain crises, hospitalizations, or
strokes? How well do they help you manage your pain, breathing
difficulties, or other health effects?
b. What are the biggest problems with these treatments? (Examples
may include side effects of medicine, going to the hospital for
treatment, frequent blood tests, etc.) How do these problems affect
your daily life?
2. Besides prescription medications, what else do you do to prevent
or treat any negative effects of your sickle cell disease? Please
describe any medications purchased at a store without a prescription,
home remedies, diet changes, massages, or other therapies.
a. What specific parts of your sickle cell disease do these
treatments address?
b. How well do these treatments work for you?
c. What are the biggest problems with these treatments?
3. What parts of your sickle cell disease do your current
treatments not treat at all or not as well as you would like?
4. Assuming that there is no cure for sickle cell disease, what
specific things would you look for in an ideal treatment?
5. If you had the opportunity to consider participating in a
clinical trial studying experimental treatments for sickle cell
disease, what things would you consider when deciding whether or not to
participate? Examples may include how severe your sickle cell disease
is, how well current treatments are working for you, your concern about
serious risks, and other things.
B. Meeting Attendance and/or Participation
If you wish to attend this meeting, visit https://patientfocusedsicklecell.eventbrite.com. Please register by January 27,
2014. Those who are unable to attend the meeting in person can register
to participate in a live Webcast of the meeting. You will be asked to
indicate in your registration whether you plan to attend in person or
via the Webcast. Your registration should also contain your complete
contact information, including name, title, affiliation, address, email
address, and phone number.
Seating will be limited, so early registration is recommended.
Registration is free and will be on a first-come, first-served basis.
However, FDA may limit the number of participants from each
organization based on space limitations. Registrants will receive
confirmation once they have been accepted. Onsite registration on the
day of the meeting will be based on space availability. If you need
special accommodations because of disability, please contact Graham
Thompson (see FOR FURTHER INFORMATION CONTACT) at least 7 days before
the meeting.
Patients who are interested in presenting comments as part of the
initial panel discussions will be asked to indicate in their
registration which topic(s) they wish to address. They will also be
asked to send a brief summary of responses to the topic questions to
[email protected]. Panelists will be notified of their
selection soon after the close of registration on January 27, 2014. FDA
will try to accommodate all patients and patient stakeholders who wish
to speak, either through the panel discussion or audience
participation; however, the duration of comments may be limited by time
constraints.
Interested members of the public, including those who attend the
meeting in person or through the Webcast, are invited to provide
electronic or written responses to the questions pertaining to Topics 1
and 2 to the public docket (see ADDRESSES). Comments may be submitted
until April 8, 2014.
Dated: October 31, 2013.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2013-26548 Filed 11-5-13; 8:45 am]
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