Prescription Drug User Fee Act Patient-Focused Drug Development; Public Meeting and Request for Comments, 58849-58851 [2012-23454]
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TKELLEY on DSK3SPTVN1PROD with NOTICES
Federal Register / Vol. 77, No. 185 / Monday, September 24, 2012 / Notices
decisionmaking. A key part of
regulatory decisionmaking is
establishing the context in which the
particular decision is made. In drug
regulation, this context includes a
thorough understanding of the severity
of the treated condition and the
adequacy of the existing treatment
options. Patients who live with a
disease have a direct stake in the
outcome of the review process and are
in a unique position to contribute to
weighing benefit-risk considerations
that can occur throughout the medical
product development process. Though
several programs exist to facilitate
patient representation, there are
currently few venues in which the
patient perspective is discussed outside
of a specific product’s marketing
application review. The human drug
and biologic review process could
benefit from a more systematic and
expansive approach to obtaining input
from patients who are experiencing a
particular disease or condition.
FDA is committed to obtaining input
from patients and, as set out in the
commitment letter, will conduct public
meetings to consider 20 different
disease areas over the 5-year
authorization of the program. For each
disease area, FDA will conduct a public
meeting to discuss such topics as the
impact of the disease on patients, the
spectrum of severity for those who have
the disease, the measures of benefit that
matter most to patients, and the
adequacy of the existing treatment
options for patients. In a separate notice
published elsewhere in this issue of the
Federal Register, FDA is seeking
comment on a proposed list of disease
areas for consideration at these
meetings.
FDA recognizes that there is
significant interest in patient-focused
drug development within the patient
community. To ensure that patient
stakeholders have an additional
opportunity to engage in a discussion of
key process considerations as this
initiative moves forward in PDUFA V,
FDA is convening a series of periodic
consultation meetings with patient
stakeholders to address key process
questions for patient-focused drug
development. These periodic
consultation meetings will be separate
from the disease-specific public
meetings that are part of FDA’s
commitments in PDUFA V; however,
the process consultation discussions
may help inform the best strategies for
conducting future disease-specific
meetings. FDA anticipates that the
periodic consultation meetings will be
focused on process questions for
consideration by FDA and patient
VerDate Mar<15>2010
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Jkt 226001
stakeholders. Examples of potential
process topics include the following:
1. Given the limits of FDA staff
resources and time available, how to
prioritize and balance different disease
areas identified by different patient
stakeholders.
2. How to approach issues when
patient stakeholders for the same
disease area have different and
potentially conflicting views.
3. How to balance access to FDA for
patient stakeholders who are local to
FDA headquarters versus those in other
locations who have less physical access.
4. How to support engagement of
patients in disease areas for which no
formal advocacy organizations exist.
What role, if any, might already
organized groups play?
Patient stakeholders provided critical
input in the development of the patientfocused drug development proposal
during the PDUFA V discussions. FDA
expects that there will be continued
interest among patient stakeholders as
this PDUFA V enhancement is
implemented. FDA is publishing this
Federal Register notice to request that
patient stakeholders notify the Agency
of their intention to participate in this
series of process consultation meetings
on patient-focused drug development.
FDA believes that consistent patient
stakeholder representation at these
meetings will be important for ensuring
progress in these discussions.
II. Notification of Intention To
Participate in Periodic Consultation
Meetings
If you are an individual patient
stakeholder who intends to participate
in periodic consultation meetings
regarding FDA’s implementation of the
patient-focused drug development
initiative, please provide notification by
email to PatientFocused@fda.hhs.gov by
October 3, 2012. If you represent an
organization that intends to participate
in these meetings, please designate one
or more representatives from your
organization who will commit to
attending these meetings and preparing
for the discussions as needed and
provide notification by email to
PatientFocused@fda.hhs.gov by October
3, 2012. All notification emails should
contain complete contact information,
including name, title, affiliation,
address, email address, phone number,
and notice of any special
accommodations required because of
disability. Seating will be limited, so
early notification is encouraged. FDA
may limit the number of participants
from each organization based on space
limitations. Patient stakeholders will
receive confirmation and additional
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58849
information about the first meeting once
FDA receives their notification and will
be included in future communications
from FDA about implementing patientfocused drug development. If
stakeholders decide to participate at a
later time, they may notify FDA of their
intent to participate in future meetings
as described previously in this
document (see ADDRESSES). FDA intends
to post summary meeting minutes on its
Web site after each meeting has
concluded.
Dated: September 14, 2012.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2012–23453 Filed 9–21–12; 8:45 am]
BILLING CODE 4160–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2012–N–0967]
Prescription Drug User Fee Act
Patient-Focused Drug Development;
Public Meeting and Request for
Comments
AGENCY:
Food and Drug Administration,
HHS.
Notice of public meeting;
request for comments.
ACTION:
The Food and Drug
Administration (FDA) is announcing a
public meeting and an opportunity for
public comment related to FDA’s
patient-focused drug development
initiative. This initiative is being
conducted to fulfill FDA performance
commitments made as part of the fifth
authorization of the Prescription Drug
User Fee Act (PDUFA V). This effort
provides for a more systematic approach
under PDUFA V for obtaining patient
perspective on the disease severity and
the currently available treatments for a
set of disease areas. FDA is publishing
a preliminary list of nominated disease
areas for the patient-focused drug
development initiative and the criteria
used for nomination. The public is
invited to comment on this preliminary
list through a public docket and at a
public meeting where FDA will provide
an overview of the patient-focused drug
development initiative with discussion
of the nominated disease areas.
DATES: Submit either electronic or
written comments by November 1, 2012.
The public meeting will be held on
October 25, 2012, from 9 a.m. to 12:30
p.m. Registration to attend the meeting
must be received by October 18, 2012.
See the SUPPLEMENTARY INFORMATION
SUMMARY:
E:\FR\FM\24SEN1.SGM
24SEN1
58850
Federal Register / Vol. 77, No. 185 / Monday, September 24, 2012 / Notices
TKELLEY on DSK3SPTVN1PROD with NOTICES
section for information on how to
register for the meeting.
ADDRESSES: The meeting will be held at
the FDA White Oak Campus, 10903
New Hampshire Ave., Bldg. 31
Conference Center, the Great Room (Rm.
1503), Silver Spring, MD 20993–0002.
Entrance for the public meeting
participants (non-FDA employees) is
through Building 1 where routine
security check procedures will be
performed. For parking and security
information, please refer to https://
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.
FOR FURTHER INFORMATION CONTACT:
Andrea Tan, Center for Drug Evaluation
and Research, Food and Drug
Administration, 10903 New
Hampshire Ave., Bldg. 51, Rm. 1168,
Silver Spring, MD 20993, 301–796–
7641, FAX: 301–847–8443,
Andrea.Tan@fda.hhs.gov,
or
Robert Yetter, Center for Biologics
Evaluation and Research (HFM–25),
1401 Rockville Pike, Suite 200N,
Rockville, MD 20852–1448, 301–827–
0373.
SUPPLEMENTARY INFORMATION:
I. Background
On July 9, 2012, the President signed
into law the Food and Drug
Administration Safety and Innovation
Act of 2012 (FDASIA). Title I of FDASIA
reauthorizes the Prescription Drug User
Fee Act (PDUFA) that provides FDA
with the necessary user fee resources to
maintain an efficient review process for
human drug and biologic products. The
reauthorization of PDUFA includes
performance goals and procedures that
represent FDA’s commitments during
fiscal years (FY) 2013–2017. These
commitments are referred to in section
101 of FDASIA and are available on the
FDA Web site at https://www.fda.gov/
downloads/ForIndustry/UserFees/
PrescriptionDrugUserFee/
UCM270412.pdf.
Section X of these commitments
relates to enhancing benefit-risk
assessment in regulatory
decisionmaking. A key part of
regulatory decisionmaking is
establishing the context in which the
VerDate Mar<15>2010
18:54 Sep 21, 2012
Jkt 226001
particular decision is made. In drug
regulation, this context includes a
thorough understanding of the severity
of the treated condition and the
adequacy of the existing treatment
options. Patients who live with a
disease have a direct stake in the
outcome of the review process and are
in a unique position to contribute to
weighing benefit-risk considerations
that can occur throughout the medical
product development process. Though
several programs exist to facilitate
patient representation, there are
currently few venues in which the
patient perspective is discussed outside
of a specific product’s marketing
application review. The human drug
and biologic review process could
benefit from a more systematic and
expansive approach to obtaining input
from patients who experience a
particular disease or condition.
FDA is committed to obtaining input
from patients and, as set out in the
commitment letter, will conduct public
meetings to consider 20 different
disease areas over the 5-year
authorization of the program. For each
disease area, FDA will conduct a public
meeting to discuss such topics as the
impact of the disease on patients, the
spectrum of severity for those who have
the disease, the measures of benefit that
matter most to patients, and the
adequacy of the existing treatment
options for patients. These meetings
will include participation of FDA
review divisions, the relevant patient
advocacy community, and other
interested stakeholders. FDA seeks
public comment on the set of disease
areas that will be discussed at these
meetings throughout PDUFA V. A
preliminary list of possible disease areas
and the criteria used to identify these
disease areas are published in this
notice for public comment.
FDA recognizes that there is
significant interest in patient-focused
drug development within the patient
community. To ensure that patient
stakeholders have an opportunity to
contribute as this initiative moves
forward in PDUFA V, FDA also is
convening an additional series of
patient consultation meetings with
patient stakeholders to discuss key
process questions for patient-focused
drug development. These consultation
meetings will be separate from the
disease-specific meetings that are part of
FDA’s commitments in PDUFA V. FDA
has published a separate notice
elsewhere in this issue of the Federal
Register requesting that patient
stakeholders notify FDA if they intend
to participate in the patient consultation
meetings.
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Fmt 4703
Sfmt 4703
II. Disease Area Nomination
FDA is nominating the following
disease areas as potential candidates for
the focus of one of the 20 future public
meetings and invites public comment
on this preliminary list. In your
comments, please identify the disease
areas that you consider to be of greatest
priority and explain the rationale for
your recommendation.
• Pulmonary arterial hypertension.
• Heart failure.
• Primary glomerular diseases.
• Narcolepsy.
• Huntington’s Disease.
• Depression.
• Autism.
• Peripheral neuropathy.
• Fibromyalgia.
• Obesity.
• Nocturia.
• Chronic fatigue syndrome.
• Irritable bowel syndrome.
• Inflammatory bowel disease.
• Alopecia areata.
• Diabetic ulcers.
• Female sexual dysfunction.
• Interstitial cystitis/painful bladder
syndrome.
• Fracture healing.
• Diabetic foot infections.
• Hepatitis C.
• HIV.
• Patients who have experienced an
organ transplant.
• Sickle cell disease.
• Chronic graft versus host disease.
• Amyloidosis.
• Aplastic anemia.
• Melanoma.
• Lung cancer.
• Cancer and young patients.
• Cancer treatment in pregnancy.
• Cancer and sexual dysfunction.
• Cancer and depression.
• Clotting disorders (e.g., hemophilia
A (factor VIII deficiency) and von
Willebrand disease).
• Thrombotic disorders (e.g.,
antithrombin deficiency and protein C
deficiency).
• Primary humoral immune
deficiencies (e.g., common variable
immune deficiency).
• Neurologic disorders treated with
immune globulins (e.g., chronic
inflammatory demyelinating
polyneuropathy).
• Hereditary angioedema.
• Alpha-1 antitrypsin deficiency.
FDA is also interested in public
comment on disease areas that are not
represented on this preliminary list. The
Agency used several criteria to develop
the preliminary list of potential disease
areas. FDA requests that when
proposing additional disease areas for
consideration, please describe how you
E:\FR\FM\24SEN1.SGM
24SEN1
Federal Register / Vol. 77, No. 185 / Monday, September 24, 2012 / Notices
TKELLEY on DSK3SPTVN1PROD with NOTICES
applied the identified criteria in making
recommendations for additional disease
areas to consider.
FDA also welcomes public comment
on the criteria for disease area selection.
These criteria include the following:
• Disease areas that are chronic,
symptomatic, or affect functioning and
activities of daily living;
• Disease areas that reflect a range of
severity;
• Disease areas for which aspects of
the disease are not formally captured in
clinical trials;
• Disease areas that have a severe
impact on identifiable subpopulations
(such as children or the elderly);
• Disease areas that represent a broad
range in terms of size of the affected
population; or
• Disease areas for which there are
currently no therapies or very few
therapies, or the available therapies do
not directly affect how a patient feels,
functions, or survives.
FDA will consider the public
comments received at the public
meeting and through the docket and
post the set of disease areas for FY
2013–2015 on the FDA Web site. By the
end of FY 2015, FDA will initiate a
public process for determining the list
of disease areas for FY 2016–2017.
III. Public Meeting
FDA is holding a public meeting that
will begin FDA’s patient-focused drug
development initiative in PDUFA V.
The purpose of this meeting will be to
obtain public comment on the
preliminary list of potential disease
areas and the criteria used to develop
the list. In addition, recognizing that
there are many more disease areas than
can be addressed in the 20 planned FDA
meetings for PDUFA V, FDA will also
discuss strategies that have already been
pursued by patient and other public
stakeholder collaborations outside of
FDA to address the types of questions
being considered under the PDUFA
patient-focused drug development
effort, to review lessons learned and
identify a roadmap that could be used
by patient-focused private
collaborations going forward.
If you wish to attend this meeting,
please register by email to
PatientFocused@fda.hhs.gov by October
18, 2012. Your email should contain
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.
VerDate Mar<15>2010
18:54 Sep 21, 2012
Jkt 226001
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
Andrea Tan (see FOR FURTHER
INFORMATION CONTACT) at least 7 days
before the meeting.
Dated: September 14, 2012.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2012–23454 Filed 9–21–12; 8:45 am]
BILLING CODE 4160–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
National Institutes of Health
National Institute of Allergy and
Infectious Diseases; 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 a meeting of the
Division of Intramural Research Board
of Scientific Counselors, NIAID.
The meeting will be closed to the
public as indicated below in accordance
with the provisions set forth in section
552b(c)(6), Title 5 U.S.C., as amended
for the review, discussion, and
evaluation of individual intramural
programs and projects conducted by the
National Institute of Allergy and
Infectious Diseases, including
consideration of personnel
qualifications and performance, and the
competence of individual investigators,
the disclosure of which would
constitute a clearly unwarranted
invasion of personal privacy.
Name of Committee: Division of Intramural
Research Board of Scientific Counselors,
NIAID.
Date: December 10–12, 2012.
Time: December 10, 2012, 7:45 a.m. to 6:25
p.m.
Agenda: To review and evaluate personal
qualifications and performance, and
competence of individual investigators.
Place: National Institutes of Health,
Building 50, 50 Center Drive, Room 1227/
1233, Bethesda, MD 20892.
Time: December 11, 2012, 7 a.m. to 6:25
p.m.
Agenda: To review and evaluate personal
qualifications and performance, and
competence of individual investigators.
Place: National Institutes of Health,
Building 50, 50 Center Drive, Room 1227/
1233, Bethesda, MD 20892.
Time: December 12, 2012, 7 a.m. to 11 a.m.
Agenda: To review and evaluate personal
qualifications and performance, and
competence of individual investigators.
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58851
Place: National Institutes of Health,
Building 50, 50 Center Drive, Room 1227/
1233, Bethesda, MD 20892.
Contact Person: Kathryn C. Zoon, Ph.D.,
Director, Division of Intramural Research,
National Institute of Allergy, and Infectious
Diseases, NIH, Building 31, Room 4A30,
Bethesda, MD 20892, 301–496–3006,
kzoon@niaid.nih.gov.
In the interest of security, NIH has
instituted stringent procedures for entrance
onto the NIH campus. All visitor vehicles,
including taxicabs, hotel, and airport shuttles
will be inspected before being allowed on
campus. Visitors will be asked to show one
form of identification (for example, a
government-issued photo ID, driver’s license,
or passport) and to state the purpose of their
visit.
(Catalogue of Federal Domestic Assistance
Program Nos. 93.855, Allergy, Immunology,
and Transplantation Research; 93.856,
Microbiology and Infectious Diseases
Research, National Institutes of Health, HHS)
Dated: September 18, 2012.
David Clary,
Program Analyst, Office of Federal Advisory
Committee Policy.
[FR Doc. 2012–23379 Filed 9–21–12; 8:45 am]
BILLING CODE 4140–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
National Institutes of Health
National Cancer Institute; Notice of
Meeting
Pursuant to section 10(a) of the
Federal Advisory Committee Act, as
amended (5 U.S.C. App.), notice is
hereby given of a meeting of the
National Cancer Institute Director’s
Consumer Liaison Group.
The meeting will be open to the
public, with attendance limited to space
available. Individuals who plan to
attend and need special assistance, such
as sign language interpretation or other
reasonable accommodations, should
notify the Contact Person listed below
in advance of the meeting.
Name of Committee: National Cancer
Institute Director’s Consumer Liaison Group.
Date: October 25–26, 2012.
Time: 9 a.m. to 1 p.m.
Agenda: October 25, 2012—Improving
Uptake of Research Findings; October 26,
2012—NCI Update.
Place: National Institutes of Health,
Building 31, 31 Center Drive, C Wing, 6th
Floor, Conference Room 10, Bethesda, MD
20892.
Contact Person: Kelli Marciel, Director,
Office of Advocacy Relations, National
Cancer Institute, National Institutes of
Health, 31 Center Drive, Building 31, Room
10A28, Bethesda, MD 20892, 301–594–3194.
Any interested person may file written
comments with the committee by forwarding
E:\FR\FM\24SEN1.SGM
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]]>Agencies
[Federal Register Volume 77, Number 185 (Monday, September 24, 2012)]
[Notices]
[Pages 58849-58851]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2012-23454]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2012-N-0967]
Prescription Drug User Fee Act Patient-Focused Drug Development;
Public Meeting and Request for Comments
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of public meeting; request for comments.
-----------------------------------------------------------------------
SUMMARY: The Food and Drug Administration (FDA) is announcing a public
meeting and an opportunity for public comment related to FDA's patient-
focused drug development initiative. This initiative is being conducted
to fulfill FDA performance commitments made as part of the fifth
authorization of the Prescription Drug User Fee Act (PDUFA V). This
effort provides for a more systematic approach under PDUFA V for
obtaining patient perspective on the disease severity and the currently
available treatments for a set of disease areas. FDA is publishing a
preliminary list of nominated disease areas for the patient-focused
drug development initiative and the criteria used for nomination. The
public is invited to comment on this preliminary list through a public
docket and at a public meeting where FDA will provide an overview of
the patient-focused drug development initiative with discussion of the
nominated disease areas.
DATES: Submit either electronic or written comments by November 1,
2012. The public meeting will be held on October 25, 2012, from 9 a.m.
to 12:30 p.m. Registration to attend the meeting must be received by
October 18, 2012. See the SUPPLEMENTARY INFORMATION
[[Page 58850]]
section for information on how to register for the meeting.
ADDRESSES: The meeting will be held at the FDA White Oak Campus, 10903
New Hampshire Ave., Bldg. 31 Conference Center, the Great Room (Rm.
1503), Silver Spring, MD 20993-0002. Entrance for the public meeting
participants (non-FDA employees) is through Building 1 where routine
security check procedures will be performed. For parking and security
information, please refer to https://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.
FOR FURTHER INFORMATION CONTACT:
Andrea Tan, Center for Drug Evaluation and Research, Food and Drug
Administration, 10903 New Hampshire Ave., Bldg. 51, Rm. 1168, Silver
Spring, MD 20993, 301-796-7641, FAX: 301-847-8443,
Andrea.Tan@fda.hhs.gov,
or
Robert Yetter, Center for Biologics Evaluation and Research (HFM-25),
1401 Rockville Pike, Suite 200N, Rockville, MD 20852-1448, 301-827-
0373.
SUPPLEMENTARY INFORMATION:
I. Background
On July 9, 2012, the President signed into law the Food and Drug
Administration Safety and Innovation Act of 2012 (FDASIA). Title I of
FDASIA reauthorizes the Prescription Drug User Fee Act (PDUFA) that
provides FDA with the necessary user fee resources to maintain an
efficient review process for human drug and biologic products. The
reauthorization of PDUFA includes performance goals and procedures that
represent FDA's commitments during fiscal years (FY) 2013-2017. These
commitments are referred to in section 101 of FDASIA and are available
on the FDA Web site at https://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM270412.pdf.
Section X of these commitments relates to enhancing benefit-risk
assessment in regulatory decisionmaking. A key part of regulatory
decisionmaking is establishing the context in which the particular
decision is made. In drug regulation, this context includes a thorough
understanding of the severity of the treated condition and the adequacy
of the existing treatment options. Patients who live with a disease
have a direct stake in the outcome of the review process and are in a
unique position to contribute to weighing benefit-risk considerations
that can occur throughout the medical product development process.
Though several programs exist to facilitate patient representation,
there are currently few venues in which the patient perspective is
discussed outside of a specific product's marketing application review.
The human drug and biologic review process could benefit from a more
systematic and expansive approach to obtaining input from patients who
experience a particular disease or condition.
FDA is committed to obtaining input from patients and, as set out
in the commitment letter, will conduct public meetings to consider 20
different disease areas over the 5-year authorization of the program.
For each disease area, FDA will conduct a public meeting to discuss
such topics as the impact of the disease on patients, the spectrum of
severity for those who have the disease, the measures of benefit that
matter most to patients, and the adequacy of the existing treatment
options for patients. These meetings will include participation of FDA
review divisions, the relevant patient advocacy community, and other
interested stakeholders. FDA seeks public comment on the set of disease
areas that will be discussed at these meetings throughout PDUFA V. A
preliminary list of possible disease areas and the criteria used to
identify these disease areas are published in this notice for public
comment.
FDA recognizes that there is significant interest in patient-
focused drug development within the patient community. To ensure that
patient stakeholders have an opportunity to contribute as this
initiative moves forward in PDUFA V, FDA also is convening an
additional series of patient consultation meetings with patient
stakeholders to discuss key process questions for patient-focused drug
development. These consultation meetings will be separate from the
disease-specific meetings that are part of FDA's commitments in PDUFA
V. FDA has published a separate notice elsewhere in this issue of the
Federal Register requesting that patient stakeholders notify FDA if
they intend to participate in the patient consultation meetings.
II. Disease Area Nomination
FDA is nominating the following disease areas as potential
candidates for the focus of one of the 20 future public meetings and
invites public comment on this preliminary list. In your comments,
please identify the disease areas that you consider to be of greatest
priority and explain the rationale for your recommendation.
Pulmonary arterial hypertension.
Heart failure.
Primary glomerular diseases.
Narcolepsy.
Huntington's Disease.
Depression.
Autism.
Peripheral neuropathy.
Fibromyalgia.
Obesity.
Nocturia.
Chronic fatigue syndrome.
Irritable bowel syndrome.
Inflammatory bowel disease.
Alopecia areata.
Diabetic ulcers.
Female sexual dysfunction.
Interstitial cystitis/painful bladder syndrome.
Fracture healing.
Diabetic foot infections.
Hepatitis C.
HIV.
Patients who have experienced an organ transplant.
Sickle cell disease.
Chronic graft versus host disease.
Amyloidosis.
Aplastic anemia.
Melanoma.
Lung cancer.
Cancer and young patients.
Cancer treatment in pregnancy.
Cancer and sexual dysfunction.
Cancer and depression.
Clotting disorders (e.g., hemophilia A (factor VIII
deficiency) and von Willebrand disease).
Thrombotic disorders (e.g., antithrombin deficiency and
protein C deficiency).
Primary humoral immune deficiencies (e.g., common variable
immune deficiency).
Neurologic disorders treated with immune globulins (e.g.,
chronic inflammatory demyelinating polyneuropathy).
Hereditary angioedema.
Alpha-1 antitrypsin deficiency.
FDA is also interested in public comment on disease areas that are
not represented on this preliminary list. The Agency used several
criteria to develop the preliminary list of potential disease areas.
FDA requests that when proposing additional disease areas for
consideration, please describe how you
[[Page 58851]]
applied the identified criteria in making recommendations for
additional disease areas to consider.
FDA also welcomes public comment on the criteria for disease area
selection. These criteria include the following:
Disease areas that are chronic, symptomatic, or affect
functioning and activities of daily living;
Disease areas that reflect a range of severity;
Disease areas for which aspects of the disease are not
formally captured in clinical trials;
Disease areas that have a severe impact on identifiable
subpopulations (such as children or the elderly);
Disease areas that represent a broad range in terms of
size of the affected population; or
Disease areas for which there are currently no therapies
or very few therapies, or the available therapies do not directly
affect how a patient feels, functions, or survives.
FDA will consider the public comments received at the public
meeting and through the docket and post the set of disease areas for FY
2013-2015 on the FDA Web site. By the end of FY 2015, FDA will initiate
a public process for determining the list of disease areas for FY 2016-
2017.
III. Public Meeting
FDA is holding a public meeting that will begin FDA's patient-
focused drug development initiative in PDUFA V. The purpose of this
meeting will be to obtain public comment on the preliminary list of
potential disease areas and the criteria used to develop the list. In
addition, recognizing that there are many more disease areas than can
be addressed in the 20 planned FDA meetings for PDUFA V, FDA will also
discuss strategies that have already been pursued by patient and other
public stakeholder collaborations outside of FDA to address the types
of questions being considered under the PDUFA patient-focused drug
development effort, to review lessons learned and identify a roadmap
that could be used by patient-focused private collaborations going
forward.
If you wish to attend this meeting, please register by email to
PatientFocused@fda.hhs.gov by October 18, 2012. Your email should
contain 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 Andrea Tan (see FOR FURTHER INFORMATION
CONTACT) at least 7 days before the meeting.
Dated: September 14, 2012.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2012-23454 Filed 9-21-12; 8:45 am]
BILLING CODE 4160-01-P
