[Federal Register Volume 77, Number 247 (Wednesday, December 26, 2012)]
[Notices]
[Pages 76050-76051]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2012-31043]


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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 an efficacy/response biomarker in evaluating 
new drugs for the treatment of acute myeloid leukemia (AML). Our 
objective is for the workshop to provide a venue for an in-depth 
discussion of potential endpoints for trials intended to support the 
approval of new drugs or biologics for treatment of AML. Participants 
in the workshop will examine if any currently used biomarker can be 
used as a surrogate endpoint, identify the preferred technology 
platform and performance characteristics for the assay of the 
biomarker, discuss any issues regarding ongoing deficiencies in 
methodological standardization for the biomarker, and determine the 
need for additional FDA-approved in-vitro diagnostics for AML drug 
development. The primary focus will be on the biomarkers that are or 
will soon be ready for incorporation into clinical trials, and the 
technical and regulatory challenges for use of these markers.

[[Page 76051]]


DATES: The public workshop will be held on March 4, 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-
796-2340, email: [email protected].

SUPPLEMENTARY INFORMATION:

I. Background

    Complete remission, relapse-free survival, and overall survival are 
frequently used as endpoints in clinical trials of new therapeutics for 
AML. These endpoints have some limitations, especially in the context 
of minimal residual disease. Use of morphological complete remission 
may miss individuals with clinically significant residual disease who 
are not truly in remission. For those being followed after remission 
induction, new evidence of submorphological disease may prompt therapy 
before morphological relapse. Additionally, for patients with good 
prognosis, the length of the clinical trial followup may be very long 
when survival is the outcome measured, raising logistical and financial 
challenges for study conduct. More information is needed on whether MRD 
in AML can be qualified as a response biomarker and then used as a 
clinical trial endpoint and what the challenges would be to implement 
use of such an endpoint.
    This Public Workshop on Minimal Residual Disease in AML 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 has explored, or plans to explore, 
the potential utility of MRD as a surrogate endpoint in acute 
lymphoblastic leukemia (ALL) (including the relapsed setting), chronic 
lymphocytic leukemia (CLL), and 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, and the CLL 
workshop will be held on February 27, 2013.

II. Structure and Scope of the Workshop

    The workshop's scope will include discussions of the use of flow 
cytometry and molecular methods used to detect and measure minimal 
residual disease in patients being treated for AML. The workshop will 
consist of formal presentations examining the regulatory, scientific, 
and clinical basis for use of biomarkers as potential clinical trial 
endpoints in AML interspersed with discussions on issues associated 
with these endpoints.

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/WEB22GPAXN9NQB (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. 2012-31043 Filed 12-21-12; 4:15 pm]
BILLING CODE 4160-01-P