[Federal Register Volume 74, Number 48 (Friday, March 13, 2009)]
[Page 10944]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: E9-5496]



Food and Drug Administration

[Docket No. FDA-2009-N-0664]

Clinical Trials Endpoints for Acute Graft-Versus-Host Disease 
After Allogeneic Hematopoietic Stem Cell Transplantation; Public 

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public workshop.


    The Food and Drug Administration (FDA) and National Institutes of 
Health (NIH) in co-sponsorship with the Center for International Blood 
and Marrow Transplantation Research (CIBMTR) and the American Society 
for Blood and Marrow Transplantation (ASBMT) are announcing a public 
workshop entitled ``Clinical Trials Endpoints for Acute Graft-Versus-
Host Disease (GVHD) After Allogeneic Hematopoietic Stem Cell 
Transplantation.'' This is a 1-day workshop for academics, government 
researchers, clinical trial experts, government regulators, and 
industry representatives. The purpose of the public workshop is to 
review the data that will serve as the foundation for protocol design 
and clinical trial evidence-based endpoints intended to support the 
approval of new drugs or biologics to prevent or treat acute GVHD. The 
public workshop also will inform FDA and assist investigators in 
facilitating clinical development programs for products to prevent or 
treat acute GVHD indications.
    Date and Time: The public workshop will be held on May 19, 2009, 
from 8:30 a.m. to 5 p.m.
    Location: The public workshop will be held at the Hilton Washington 
DC/Rockville Executive Meeting Center, 1750 Rockville Pike, Rockville, 
MD 20852.
    Overnight accommodations can be booked at the Hilton under group 
code ``MCW'' for the conference rate by calling 1-800-445-8667 or by 
using the Reservation Web site athttp://www.hilton.com/en/hi/groups/personalized/IADMRHF-MCW-20090518/index.jhtml. Accommodation agreement 
courtesy of CIBMTR. (FDA has verified the Web site address, but is not 
responsible for subsequent changes to the Web site after this document 
publishes in the Federal Register).
    Contact Person: Leslie Haynes, Center for Biologics Evaluation and 
Research (HFM-43), Food and Drug Administration, 1401 Rockville Pike, 
suite 200N, Rockville, MD 20852-1448, 301-827-2000, FAX: 301-827-3079; 
e-mail: [email protected] (Subject line: Acute GVHD Workshop).
    Registration: Mail or fax your registration information (including 
name, title, firm name, address, telephone and fax numbers) to the 
Contact Person by April 18, 2009. There is no registration fee for the 
public workshop. Early registration is recommended because seating is 
limited. Registration on the day of the public workshop will be 
provided on a space available basis beginning at 8:15 a.m.
    If you need special accommodations due to a disability, please 
contact Leslie Haynes at least 7 days in advance.

SUPPLEMENTARY INFORMATION: At the present time, there are no drugs or 
biologics approved for prevention or treatment of acute GVHD. 
Development of products to prevent or treat acute GVHD poses several 
challenges. First, the market is not very big, so there is little 
incentive for investment if the process is cumbersome; second, analyses 
of these studies are complicated by confounding factors; and third, 
there is a lack of evidence-based endpoints that can be used to 
demonstrate a clinically meaningful benefit of any therapy.
    The Center for Biologics Evaluation and Research is the FDA Center 
with regulatory responsibility for vaccines, blood and blood products, 
allergenic products, and therapies involving cells, tissues, and genes. 
The mission of FDA is to protect and enhance the public health 
including the safety and purity of medical products and the Nation's 
blood supply. The purpose of this event is to review the data that can 
be used to develop evidence-based endpoints for clinical trials 
targeting acute GVHD.
    ASBMT is a professional organization that promotes advancement of 
the field of blood and bone marrow transplantation. Its members are 
both in clinical practice and in research.
    CIBMTR is a research network comprised of the National Marrow Donor 
Program[sscopy] and the International Bone Marrow Transplant Registry 
and Autologous Blood and Marrow Transplant Registry. Its activities 
include support for the National Heart, Lung and Blood Institute 
(NHLBI)-funded Blood and Marrow Transplantation Clinical Trials Network 
and Health Resources and Services Administration's C.W. Bill Young Cell 
Transplantation Program. The goals of the CIBMTR include defining key 
areas for future research in collaboration with leading scientists, 
physicians, and others in the blood and marrow transplant community; 
the design and implementation of clinical studies; and making available 
research resources including a clinical database of related blood and 
marrow transplants, along with repositories of matched tissue samples 
from transplant recipients and their donors.
    The NHLBI, National Institute of Allergy and Infectious Diseases 
(NIAID), National Cancer Institute (NCI), and Office of Rare Diseases 
(ORD) are at the National Institutes of Health (NIH), the primary 
Federal agency for conducting and supporting medical research. NIH's 
mission is science in pursuit of fundamental knowledge about the nature 
and behavior of living systems and the application of that knowledge to 
extend healthy life and reduce the burdens of illness and disability.
    The public workshop will feature presentations by FDA, CIBMTR, and 
members of ASBMT. The topics to be discussed include the following: (1) 
Regulatory requirements for clinical trials, (2) extant data which 
support the endpoints currently used in clinical trials, (3) data 
analyses to support the validity of the proposed endpoints, (4) 
statistical approaches to minimize confounding factors in stem cell 
transplantation study analysis, (5) biomarkers for acute GVHD, and (6) 
patient-reported outcomes for acute GVHD prevention and treatment 
    Presentations: Presentations from the public workshop will be 
maintained on the CIBMTR's Web site for at least 1 year.

    Dated: March 6, 2009.
Jeffrey Shuren,
Associate Commissioner for Policy and Planning.
[FR Doc. E9-5496 Filed 3-12-09; 8:45 am]