[Federal Register Volume 62, Number 167 (Thursday, August 28, 1997)]
[Notices]
[Pages 45670-45671]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 97-22982]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration


Potency and Dosage of Von Willebrand Factor Concentrates; Public 
Workshop

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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    The Food and Drug Administration (FDA) is announcing a public 
workshop entitled ``FDA Sponsored Workshop on Potency and Dosage of von 
Willebrand Factor Concentrates (vWF).'' The topics to be discussed 
include potency assays and standards for vWF concentrates; 
pharmacokinetic studies and clinical trials of vWF concentrates; the 
correlation of dosage regimens with clinical outcome; and labeling of 
vWF concentrates.
    Date and Time: The workshop will be held on September 26, 1997, 8 
a.m. to 5 p.m.
    Location: The workshop will be held at Jack Masur Auditorium, 
National Institute of Health, 8800 Rockville Pike, Bldg. 10, Bethesda, 
MD 20892.
    Contact: Joseph Wilczek, Center for Biologics Evaluation and 
Research (HFM-350), Food and Drug Administration, 1401 Rockville Pike, 
Rockville, MD 20852-1448, 301-827-3514, FAX 301-827-2843.
SUPPLEMENTARY INFORMATION:
    FDA has the responsibility of ensuring that product labeling 
provides information about product potency and dosage. In the case of 
replacement therapy for deficiencies in coagulation factor activity, 
this has been done by assessing the potency of a product relative to a 
defined standard, and by measuring the pharmacokinetics of the product. 
This information has been used to establish a dosage that will raise 
the concentration of circulating coagulation activity to a targeted 
level for a known period of time. Clinical trials establish the 
clinical benefit of a given dosage regimen. This model has been 
difficult to apply to products submitted to FDA for licensure for the 
treatment of vWF because there is no standardized in vitro test for vWF 
potency; there is no vWF

[[Page 45671]]

concentrate standard, and assays based on vWF plasma standards may not 
be appropriate to measure the potency of concentrates; and published 
clinical trials have not correlated the dosage of specific products 
with clinical outcome. The main goal of this workshop is to address 
these concerns through exchange of information about each of these 
issues, through the participation of the patient, industrial, medical, 
scientific, and regulatory communities. Workshop participants are asked 
to present their positions, rationales, and/or experiences regarding: 
(1) The benefits and liabilities of using ristocetin cofactor activity, 
or other tests, to measure vWF activity; (2) proposals for 
standardizing the potency and dosage of vWF concentrates; and (3) 
clinical trials to relate given dosage regimen to clinical benefit. 
Information presented at this workshop will assist in product 
development and facilitate licensure of safe and effective vWF 
products.
    Registration and Requests for Oral Presentations: Fax registration 
information (including name, title, firm name, address, telephone, and 
fax number), and written material and requests to make oral 
presentations, to the contact person by September 19, 1997. 
Registration at the site will be done on a space available basis on the 
day of the workshop beginning at 7:30 a.m. There is no registration fee 
for the workshop.
    If you need special accommodations due to a disability, please 
contact Joseph Wilczek at least 7 days in advance.
    Transcripts: Transcripts of the workshop may be requested in 
writing from the Freedom of Information Office (HFI-35), Food and Drug 
Administration, rm. 12A-16, 5600 Fishers Lane, Rockville, MD 20857, 
approximately 15 working days after the workshop at a cost of 10 cents 
per page.

    Dated: August 22, 1997.
William B. Schultz,
Deputy Commissioner for Policy.
[FR Doc. 97-22982 Filed 8-27-97; 8:45 am]
BILLING CODE 4160-01-F