[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]
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