[Federal Register Volume 77, Number 49 (Tuesday, March 13, 2012)]
[Notices]
[Pages 14813-14814]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2012-6038]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2012-N-0001]


Public Workshop on Minimal Residual Disease; 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 to provide a forum for discussion of the use of minimal 
residual disease (MRD) as a biomarker for evaluating new drugs for the 
treatment of acute lymphoblastic leukemia (ALL). The meeting is 
cosponsored with the American Society of Clinical Oncology and will be 
the first in a series of workshops intended to bring together 
scientific and advocacy communities and the pharmaceutical and in vitro 
diagnostic device industries to help develop processes and procedures 
to qualify MRD as a biomarker of efficacy and/or response to treatment 
in a group of hematological malignancies.

DATES:  Date and Time: The public workshop will be held on April 18, 
2012, from 8 a.m. to 4 p.m.
    Location: The public workshop will be held at the FDA White Oak 
Campus, 10903 New Hampshire Ave. Bldg. 31 Conference Center, the Great 
Room (rm. 1503), Silver Spring, MD 20993-0002.
    Contact Person: Christine Lincoln, Center for Drug Evaluation and 
Research, Food and Drug Administration, 10903 New Hampshire Ave. Bldg. 
22, rm. 6413, Silver Spring, MD 20993-0002, 301-796-2340.

SUPPLEMENTARY INFORMATION:

I. Background

    Clinical data from patients with certain subtypes of acute and 
chronic leukemia suggest that MRD can be established as a surrogate 
endpoint for clinical trials and drug approval. This public workshop 
will provide a forum for discussion among scientific and advocacy 
communities and the pharmaceutical and in vitro diagnostic device 
industries of issues related to the qualification (validation) of MRD 
as a biomarker (i.e., a measurable characteristic that is predictive of 
disease outcome) that can be used to determine efficacy and/or response 
in evaluation of new drugs for the treatment of ALL. Although the data 
related to the prognostic significance of MRD are most extensive in the 
pediatric population, and are currently used to stratify patients for 
risk-adjusted therapy, MRD may also be pertinent to subtypes of adult 
ALL; hematologists who treat adult patients have been invited to 
participate, as well as hematologists who treat pediatric patients. 
Topics to be discussed at the workshop include: (1) Evaluation of the 
prognostic biomarker data that is currently available to support the 
qualification of MRD as a marker of response and/or efficacy in both 
pediatric and adult ALL; (2) the specificity, sensitivity, and 
comparability of techniques that might be used in a standardized 
fashion to measure MRD; (3) the performance characteristics and 
proficiency assessment of current technology platforms; and (4) the 
design and analysis of the clinical trials needed to establish the use 
of postinduction MRD as an alternative endpoint for approval of new 
drugs to treat ALL.
    This workshop is part of a series in which FDA's Office of 
Hematology and Oncology Products will explore the utility of MRD as a 
surrogate endpoint in ALL (including ALL that has recurred), chronic 
lymphocytic leukemia (CLL), and acute myeloid leukemia (AML). Given the 
diverse etiologies, pathophysiologies, and natural histories of these 
diseases and current practice standards, separate consideration of MRD 
as a surrogate endpoint in each disease is warranted. FDA is seeking 
representation from both North American and European academic 
investigators as well as cooperative groups at the workshops. The 
workshops for CLL and AML are tentatively scheduled for October 10 and 
11, 2012, respectively.

II. Attendance and Registration

    FDA encourages patient advocates, representatives from industry, 
consumer groups, health care professionals, researchers, and other 
interested persons to attend this public workshop.
    Registration: There is no registration fee for the public workshop. 
To register electronically, please use the following Web site: http://
www.zoomerang.com/

[[Page 14814]]

Survey/WEB22EJ4HRZLW9. (FDA has verified the Web site address, but we 
are not responsible for any subsequent changes to the Web site after 
this document publishes in the Federal Register.)
    Seats are limited and conference space will be filled in the order 
in which registrations are received. Onsite registration will be 
available to the extent that space is available on the day of the 
conference.
    Information regarding special accommodations due to a disability, 
visitor parking, and transportation may be accessed at: http://www.fda.gov/AdvisoryCommittees/default.htm; under the heading 
``Resources for You,'' click on ``Public Meetings at the FDA White Oak 
Campus.'' Please note that visitors to the White Oak Campus must enter 
through Bldg. 1.

    Dated: March 8, 2012.
Leslie Kux,
Acting Assistant Commissioner for Policy.
[FR Doc. 2012-6038 Filed 3-12-12; 8:45 am]
BILLING CODE 4160-01-P