[Federal Register: July 6, 2004 (Volume 69, Number 128)]
[Page 40639-40640]
From the Federal Register Online via GPO Access [wais.access.gpo.gov]



Food and Drug Administration

[Docket No. 2004N-0033]

Establishing a Docket for the Factor VIII Inhibitor Public 
Workshop; Availability

AGENCY:  Food and Drug Administration, HHS.

ACTION:  Notice.


SUMMARY:  The Food and Drug Administration (FDA) is announcing the 
opening of a docket to receive information and comments on the November 
21, 2003, public workshop entitled ``Factor VIII Inhibitors'' (the 
workshop). We are opening the docket because there was insufficient 
time available during the workshop for a full discussion of the many 
important topics covered at the workshop.

DATES:  Submit written or electronic comments on the workshop, related 
regulatory and scientific issues, and comments on information submitted 
to the docket by other interested parties by January 6, 2006.

ADDRESSES:  Submit written comments and information related to the 
workshop to the Division of Dockets Management (HFA-305), Food and Drug 
Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD 20852-1448. 
Submit electronic comments or information to http://frwebgate.access.gpo.gov/cgi-bin/leaving.cgi?from=leavingFR.html&log=linklog&to=http://www.fda.gov/dockets/ecomments.

electronic and other access to the slide presentations and transcript 
from the workshop.

FOR FURTHER INFORMATION CONTACT: Sharon Carayiannis, Center for 
Biologics Evaluation and Research (HFM-17), Food and Drug 
Administration, 1401 Rockville Pike, Rockville, MD 20852-1448, 301-827-


I. Background

    In the Federal Register of October 20, 2003 (68 FR 59942), we 
published a notice to announce a public workshop entitled ``Factor VIII 
Inhibitors.'' On November 21, 2003, we, in cosponsorship with the 
International Association for Biologicals, held the workshop to address 
regulatory and scientific concerns about inhibitors to Factor VIII 
induced by Antihemophilic Factor (Factor VIII) products. These 
inhibitors arise in a significant minority of patients with hemophilia 
and make replacement therapy problematic. The workshop covered a broad 
range of

[[Page 40640]]

topics. The workshop provided valuable information, but additional time 
was needed at the close of the meeting for continued dialogue on 
important topics. At the end of the workshop, we invited written 
comments to provide an opportunity for a full discussion of issues.
    We have established this docket to encourage interested parties to 
continue to provide information about Factor VIII inhibitors, comments 
on the workshop, and comments on information submitted to the docket by 
other interested parties. We also request that those who have already 
submitted written comments and information to FDA resubmit the same 
comments to the docket to ensure their adequate consideration since 
this information was not previously submitted to the docket. We also 
posted this request for comments and information at http://frwebgate.access.gpo.gov/cgi-bin/leaving.cgi?from=leavingFR.html&log=linklog&to=http://www.fda.gov/cber/meetings/fctrvIII112103L.htm

    Comments submitted to the docket will assist us in determining the 
need for and feasibility of establishing new inhibitor assay standards 
and methodologies, stakeholders' opinions about current upper and lower 
limits of acceptable inhibitor formation in clinical trials, and the 
use of plasma-derived versus recombinant Factor VIII controls in 
pharmacokinetic trials, among other issues. We may also consider the 
information in preparing any future guidance on clinical trials to 
evaluate potential inhibitor formation from Factor VIII products.

II. Comments

    Interested persons may submit to the Division of Dockets Management 
(see ADDRESSES) written or electronic comments regarding the workshop. 
Submit a single copy of electronic comments or two paper copies of any 
mailed comments, except that individuals may submit one paper copy. 
Comments are to be identified with the docket number found in brackets 
in the heading of this document. A copy of this notice, the slide 
presentations and transcript from the workshop, and received comments 
are available for public examination in the Division of Dockets 
Management between 9 a.m. and 4 p.m., Monday through Friday.

III. Electronic Access

    Persons with access to the Internet may obtain the slide 
presentations at http://frwebgate.access.gpo.gov/cgi-bin/leaving.cgi?from=leavingFR.html&log=linklog&to=http://www.fda.gov/cber/summaries.htm and the transcript of the workshop at http://frwebgate.access.gpo.gov/cgi-bin/leaving.cgi?from=leavingFR.html&log=linklog&to=http://www.fda.gov/cber/minutes/workshop-min.htm.

    Dated: June 24, 2004.
Jeffery Shuren,
Assistant Commissioner for Policy.
[FR Doc. 04-15135 Filed 7-2-04; 8:45 am]