Improving Endpoints, Improving Care: Alpha-1 Antitrypsin Augmentation Therapy and Clinical Trials; Public Workshop, 6903 [E9-2905]
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Federal Register / Vol. 74, No. 27 / Wednesday, February 11, 2009 / Notices
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Jeffrey Shuren,
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[FR Doc. E9–2802 Filed 2–6–09; 12:00 pm]
BILLING CODE 4160–01–S
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
mstockstill on PROD1PC66 with NOTICES
[Docket No. FDA–2009–N–0664]
Improving Endpoints, Improving Care:
Alpha-1 Antitrypsin Augmentation
Therapy and Clinical Trials; Public
Workshop
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice of public workshop.
VerDate Nov<24>2008
17:58 Feb 10, 2009
Jkt 217001
The Food and Drug Administration
(FDA) is announcing a public workshop
entitled: Improving Endpoints,
Improving Care: Alpha-1 Antitrypsin
Augmentation Therapy and Clinical
Trials. The purpose of the public
workshop is to identify the most useful
clinical trial endpoints and surrogate
markers for Alpha-1 antitrypsin (AAT)
augmentation therapy. FDA, Alpha-1
Foundation, and the Department of
Health and Human Services, Office of
Public Health and Science are
convening this workshop to facilitate
the design of future clinical trials
intended to establish clinical efficacy of
AAT products. The public workshop
will feature presentations and panel
discussions led by experts from
academic institutions, government, and
industry.
Date and Time: The public workshop
will be held on March 23, 2009, from
8:30 a.m. to 5:30 p.m. and March 24,
2009, from 8:30 a.m. to 5 p.m.
Location: The public workshop will
be held at the Lister Hill Center
Auditorium, Bldg. 38A, National
Institutes of Health, 8800 Rockville
Pike, Bethesda, MD 20894.
Contact Person: Rhonda Dawson,
Center for Biologics Evaluation and
Research (HFM–302), Food and Drug
Administration, 1401 Rockville Pike,
suite 200N, Rockville, MD 20852–1448,
301–827–6129, FAX: 301–827–2843, email: rhonda.dawson@fda.hhs.gov.
Registration: Mail, fax, or e-mail your
registration information (including
name, title, firm name, address,
telephone, and fax numbers) to the
contact person by March 6, 2009. There
is no registration fee for the public
workshop. Early registration is
recommended because seating is limited
to 175 attendees. Registration on the day
of the public workshop will be provided
on a space available basis beginning at
7:30 a.m.
If you need special accommodations
due to a disability, please contact
Rhonda Dawson (see Contact Person) at
least 7 days in advance.
SUPPLEMENTARY INFORMATION: AAT
deficiency is a genetic condition that
leads to decreased levels of alpha-1
antitrypsin in the blood and
significantly increases the risk of serious
lung disease in adults and liver disease
in infants, children, and adults.
Intravenous augmentation therapy with
FDA-licensed, plasma-derived AAT
products has become the standard of
care for treatment in the subset of
patients with AAT deficiency who have
moderate pulmonary disease. Since the
original product approvals, additional
data collection and advances in
PO 00000
Frm 00049
Fmt 4703
Sfmt 4703
6903
understanding of AAT deficiency
suggest the need to revisit and improve
clinical trial efficacy endpoints.
The public workshop will facilitate
scientific discussions to identify the
most relevant and feasible, currently
available and future clinical trial
efficacy endpoints for AAT
augmentation therapy and further
evaluate its usefulness to a broader
patient population. Topics to be
discussed include: (1) AAT deficiency
disease characteristics, progression and
pulmonary pathophysiology; (2) patient
selection for clinical trials; (3) current
challenges to the development of
endpoints for clinical trials; and (4)
currently available and future clinical
trial endpoints, including functional
markers of disease progression, and
radiological and biochemical endpoints.
Transcripts: Transcripts of the public
workshop may be requested in writing
from the Freedom of Information Office
(HFI–35), Food and Drug
Administration, 5600 Fishers Lane, rm.
6–30, Rockville, MD 20857,
approximately 15 working days after the
public workshop at a cost of 10 cents
per page. A transcript of the public
workshop will be available on the
Internet at https://www.fda.gov/cber/
minutes/workshop-min.htm.
Dated: February 6, 2009.
Jeffrey Shuren,
Associate Commissioner for Policy and
Planning.
[FR Doc. E9–2905 Filed 2–10–09; 8:45 am]
BILLING CODE 4160–01–S
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2009–N–0664]
Cardiovascular and Renal Drugs
Advisory Committee; Notice of Meeting
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice.
This notice announces a forthcoming
meeting of a public advisory committee
of the Food and Drug Administration
(FDA). The meeting will be open to the
public.
Name of Committee: Cardiovascular
and Renal Drugs Advisory Committee.
General Function of the Committee:
To provide advice and
recommendations to the agency on
FDA’s regulatory issues.
Date and Time: The meeting will be
held on March 18, 2009, from 8 a.m. to
5 p.m.
E:\FR\FM\11FEN1.SGM
11FEN1
]]>Agencies
[Federal Register Volume 74, Number 27 (Wednesday, February 11, 2009)]
[Notices]
[Page 6903]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: E9-2905]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2009-N-0664]
Improving Endpoints, Improving Care: Alpha-1 Antitrypsin
Augmentation Therapy and Clinical Trials; Public Workshop
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of public workshop.
-----------------------------------------------------------------------
The Food and Drug Administration (FDA) is announcing a public
workshop entitled: Improving Endpoints, Improving Care: Alpha-1
Antitrypsin Augmentation Therapy and Clinical Trials. The purpose of
the public workshop is to identify the most useful clinical trial
endpoints and surrogate markers for Alpha-1 antitrypsin (AAT)
augmentation therapy. FDA, Alpha-1 Foundation, and the Department of
Health and Human Services, Office of Public Health and Science are
convening this workshop to facilitate the design of future clinical
trials intended to establish clinical efficacy of AAT products. The
public workshop will feature presentations and panel discussions led by
experts from academic institutions, government, and industry.
Date and Time: The public workshop will be held on March 23, 2009,
from 8:30 a.m. to 5:30 p.m. and March 24, 2009, from 8:30 a.m. to 5
p.m.
Location: The public workshop will be held at the Lister Hill
Center Auditorium, Bldg. 38A, National Institutes of Health, 8800
Rockville Pike, Bethesda, MD 20894.
Contact Person: Rhonda Dawson, Center for Biologics Evaluation and
Research (HFM-302), Food and Drug Administration, 1401 Rockville Pike,
suite 200N, Rockville, MD 20852-1448, 301-827-6129, FAX: 301-827-2843,
e-mail: rhonda.dawson@fda.hhs.gov.
Registration: Mail, fax, or e-mail your registration information
(including name, title, firm name, address, telephone, and fax numbers)
to the contact person by March 6, 2009. There is no registration fee
for the public workshop. Early registration is recommended because
seating is limited to 175 attendees. Registration on the day of the
public workshop will be provided on a space available basis beginning
at 7:30 a.m.
If you need special accommodations due to a disability, please
contact Rhonda Dawson (see Contact Person) at least 7 days in advance.
SUPPLEMENTARY INFORMATION: AAT deficiency is a genetic condition that
leads to decreased levels of alpha-1 antitrypsin in the blood and
significantly increases the risk of serious lung disease in adults and
liver disease in infants, children, and adults. Intravenous
augmentation therapy with FDA-licensed, plasma-derived AAT products has
become the standard of care for treatment in the subset of patients
with AAT deficiency who have moderate pulmonary disease. Since the
original product approvals, additional data collection and advances in
understanding of AAT deficiency suggest the need to revisit and improve
clinical trial efficacy endpoints.
The public workshop will facilitate scientific discussions to
identify the most relevant and feasible, currently available and future
clinical trial efficacy endpoints for AAT augmentation therapy and
further evaluate its usefulness to a broader patient population. Topics
to be discussed include: (1) AAT deficiency disease characteristics,
progression and pulmonary pathophysiology; (2) patient selection for
clinical trials; (3) current challenges to the development of endpoints
for clinical trials; and (4) currently available and future clinical
trial endpoints, including functional markers of disease progression,
and radiological and biochemical endpoints.
Transcripts: Transcripts of the public workshop may be requested in
writing from the Freedom of Information Office (HFI-35), Food and Drug
Administration, 5600 Fishers Lane, rm. 6-30, Rockville, MD 20857,
approximately 15 working days after the public workshop at a cost of 10
cents per page. A transcript of the public workshop will be available
on the Internet at https://www.fda.gov/cber/minutes/workshop-min.htm.
Dated: February 6, 2009.
Jeffrey Shuren,
Associate Commissioner for Policy and Planning.
[FR Doc. E9-2905 Filed 2-10-09; 8:45 am]
BILLING CODE 4160-01-S
