Document ID: FDA-2009-N-0664-0009
Agency: fda
Document Type: Notice
Title: Improving Endpoints, Improving Care; Alpha-1 Antitrypsin Augmentation Therapy and Clinical Trials; Public Workshop
Posted Date: 2009-02-11T05:00Z

[Federal Register: February 11, 2009 (Volume 74, Number 27)]
[Notices]
[Page 6903]
From the Federal Register Online via GPO Access [wais.access.gpo.gov]
[DOCID:fr11fe09-62]

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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.

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    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 http://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