[Federal Register Volume 74, Number 27 (Wednesday, February 11, 2009)]
[Notices]
[Page 6903]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: E9-2905]


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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: [email protected].
    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