[Federal Register Volume 77, Number 247 (Wednesday, December 26, 2012)]
[Notices]
[Pages 76051-76052]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2012-31044]


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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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SUMMARY: The Food and Drug Administration (FDA), in cosponsorship with 
the American Society of Clinical Oncology, is announcing a public 
workshop that will provide a forum for discussion of extending the 
qualification of minimal residual disease (MRD) detection as a 
prognostic biomarker to that of an efficacy/response biomarker in 
evaluating new drugs for the treatment of chronic lymphocytic leukemia 
(CLL). Our objective for the workshop is to provide a venue for an in-
depth discussion of potential surrogate endpoints for trials intended 
to support the approval of new drugs or biologics for the treatment of 
CLL. Participants in the workshop will examine the advantages and 
disadvantages of MRD as a surrogate endpoint for approval, identify the 
preferred technology platform and performance characteristics for the 
assay of this biomarker, and discuss any issues regarding 
methodological standardization for the biomarker. The primary focus 
will be on the biomarkers that are ready for incorporation into 
clinical trials and the technical and regulatory challenges for use of 
these markers.

DATES: The public workshop will be held on February 27, 2013, from 8 
a.m. to 4 p.m.

ADDRESSES: The public workshop will be held at the FDA White Oak 
Campus, 10903 New Hampshire Ave., Building 31 Conference Center, the 
Great Room (rm. 1503), Silver Spring, MD 20993-0002. Entrance for the 
public workshop participants (non-FDA employees) is through Building 1 
where routine security check procedures will be performed. For parking 
and security information please refer to http://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.

FOR FURTHER INFORMATION CONTACT: 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-

[[Page 76052]]

796-2340, email: [email protected].

SUPPLEMENTARY INFORMATION:

I. Background

    The Public Workshop on Minimal Residual Disease will be one of a 
series of FDA workshops to establish processes and procedures necessary 
to qualify a prognostic biomarker, MRD, as a possible response or 
efficacy biomarker in a group of hematological malignancies. Evaluation 
of clinical data suggests that MRD can be established as a potential 
surrogate endpoint for pivotal clinical trials and drug approval given 
its prominent role as a prognostic indicator in certain subtypes of 
acute and chronic leukemia. The Office of Hematology and Oncology 
Products plans to explore the potential utility of MRD as a surrogate 
endpoint in acute lymphoblastic leukemia (ALL) (including the relapsed 
setting), CLL, and acute myeloid leukemia (AML). Given the diverse 
pathophysiology and natural history of these diseases, and current 
practice standards, individualized consideration of MRD as a surrogate 
endpoint is warranted. The ALL workshop was held on April 18, 2012. The 
CLL and AML workshops are scheduled for February 27, 2013, and March 4, 
2013, respectively.

II. Structure and Scope of the Workshop

    The workshop's scope will extend to the use of flow cytometry and 
the molecular methods used to measure minimal residual disease in 
patients being treated for CLL. The workshop will consist of formal 
presentations examining the regulatory, scientific, and clinical basis 
for use of biomarkers as potential clinical trial endpoints in CLL 
followed by discussions on issues associated with use of an MRD 
endpoint.

III. 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. There is no 
registration fee for the public workshop. To register electronically, 
please use the following Web site: http://www.zoomerang.com/Survey/WEB22GPA3U95QX (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.''

    Dated: December 20, 2012.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2012-31044 Filed 12-21-12; 4:15 pm]
BILLING CODE 4160-01-P