[Federal Register Volume 79, Number 91 (Monday, May 12, 2014)]
[Notices]
[Pages 26974-26975]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2014-10787]
[[Page 26974]]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2014-N-0505]
Proteomics in the Clinic; Public Workshop; Request for Comments
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of public workshop; request for comments.
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The Food and Drug Administration (FDA) is announcing the following
public workshop entitled ``Proteomics in the Clinic.'' FDA seeks input
from interested stakeholders on how best to develop a regulatory
framework targeted toward the complex issues involved in transforming
research-level assays into validated in vitro diagnostics (IVDs) that
can be used with patients. The topic to be discussed is the state of
the art and challenges surrounding validation of proteomic
methodologies for IVD tests.
Date and Time: The public workshop will be held on June 13, 2014, from
8:30 a.m. to 5 p.m.
Location: 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.
Contact Person: Julia Tait Lathrop, Center for Devices and
Radiological Health, Food and Drug Administration, 10903 New Hampshire
Ave., Bldg. 66, Rm. 5564, Silver Spring, MD 20993-0002, 240-402-5034,
email: [email protected].
Registration: Registration is free and available on a first-come,
first-served basis. Persons interested in attending this public
workshop must register online by 4 p.m. on June 4, 2014. Early
registration is recommended because facilities are limited and,
therefore, FDA may limit the number of participants from each
organization. If time and space permits, onsite registration on the day
of the public workshop will be provided beginning at 7:30 a.m.
If you need special accommodations due to a disability, please
contact Susan Monahan, Center for Devices for Radiological Health, Food
and Drug Administration, 10903 New Hampshire Ave. Bldg. 66, Rm. 4321,
Silver Spring, MD 20993-0002, 301-796-5661, email:
[email protected] no later than 4 p.m. on May 30, 2014.
To register for the public workshop, please visit FDA's Medical
Devices News & Events--Workshops & Conferences calendar at http://www.fda.gov/MedicalDevices/NewsEvents/WorkshopsConferences/default.htm.
(Select this public workshop from the posted events list.) Please
provide complete contact information for each attendee, including name,
title, affiliation, email, and telephone number. Those without Internet
access should contact Julia Tait Lathrop to register (see Contact
Person). Registrants will receive confirmation after they have been
accepted. You will be notified if you are on a waiting list.
Streaming Webcast of the Public Workshop: This public workshop will
also be Webcast. Persons interested in viewing the Webcast must
register online by 4 p.m. on June 4, 2014. Early registration is
recommended because Webcast connections are limited. Organizations are
requested to register all participants, but to view using one
connection per location. Webcast participants will be sent technical
system requirements after registration and will be sent connection
access information after June 9, 2014. If you have never attended a
Connect Pro event before, test your connection at https://collaboration.fda.gov/common/help/en/support/meeting_test.htm. To get
a quick overview of the Connect Pro program, visit http://www.adobe.com/go/connectpro_overview. (FDA has verified the Web site
addresses in this document, but FDA is not responsible for any
subsequent changes to the Web sites after this document publishes in
the Federal Register.)
Comments: FDA is holding this public workshop to discuss with
interested stakeholders the issues surrounding validation of proteomic
methodologies for IVD tests. In order to permit the widest possible
opportunity to obtain public comment, FDA is soliciting either
electronic or written comments on all aspects of the workshop topics.
The deadline for submitting comments related to this public workshop is
July 11, 2014.
Regardless of attendance at the public workshop, interested persons
may submit either electronic comments regarding this document to http://www.regulations.gov or written comments to the Division of Dockets
Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane,
Rm. 1061, Rockville, MD 20852. It is only necessary to send one set of
comments. Identify comments with the docket number found in brackets in
the heading of this document. In addition, when responding to specific
questions as outlined in section II of this document, please identify
the question you are addressing. Received comments may be seen in the
Division of Dockets Management between 9 a.m. and 4 p.m., Monday
through Friday, and will be posted to the docket at http://www.regulations.gov.
Transcripts: Please be advised that as soon as a transcript is
available, it will be accessible at http://www.regulations.gov. It may
be viewed at the Division of Dockets Management (see Comments). A
transcript will also be available in either hardcopy or on CD-ROM,
after submission of a Freedom of Information request. Written requests
are to be sent to the Division of Freedom of Information (ELEM-1029),
Food and Drug Administration, 12420 Parklawn Dr., Element Bldg.,
Rockville, MD 20857. A link to the transcripts will also be available
approximately 45 days after the public workshop on the Internet at
http://www.fda.gov/MedicalDevices/NewsEvents/WorkshopsConferences/default.htm. (Select this public workshop from the posted events list).
SUPPLEMENTARY INFORMATION:
I. Background
Basic research in proteomics, the study of all of the proteins and
their interactions in an individual, has led to new understanding of
proteins' contributions to health and disease. It has also driven the
advancement of powerful analytical technologies used to explore these
contributions. Translating these discoveries and technologies into IVD
tests presents expanded opportunities to improve patient care; however,
the complexity of these technologies raises challenging questions on
how to evaluate the safety and effectiveness of these tests. FDA is
holding this public workshop to invite discussion with industry,
academia, government, and the public on how best to adapt current
regulatory strategies to the challenges presented by proteomic
approaches to IVDs, while still accelerating and supporting the
introduction of innovative diagnostic tests into the clinic.
Over the past 20 years, basic proteomic research has spurred
intense innovation in biochemical analytic technologies (e.g., mass
spectrometry, multiplex arrays, bioinformatics). This research has led
to new insights into how proteins interact to maintain health and to
cause disease; however, it is only recently that these technologies
have
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matured to the point that their introduction into the clinic appears
practical and useful. Fundamental to using IVDs in the clinic is the
need to demonstrate that the tests are safe and effective--that the
results claimed are accurate and precise and that the interpretation of
the results are supported by science. FDA's regulatory process is
designed to ensure that intended use claims are supported with
appropriate data. However, the range of variables that can be included
in proteomic IVDs such as technological approaches, variety of sample
types and preparation methods, data capture, and analysis algorithms,
poses unique regulatory challenges, as more complex the information
gathered, more challenging is the validation of results. At this point,
what we need is a regulatory framework, tuned to proteomic
technologies, which will facilitate the introduction of validated IVDs
into the clinic.
The intent of this workshop is not to discuss the limitations and
strengths of the proteomic discovery process. The theoretical
analytical performance of proteomic technologies have been well
demonstrated, and in the past few years a number of initiatives have
been launched to bring standardization and quality control to the
discovery and pre-clinical development of proteomic-based assays.
However, this level of quality control does not ensure that these
assays have been validated for their intended use as IVDs tests that
are used for diagnosis of disease and clinical management of patients;
e.g., assessment of risk, monitoring of disease, prediction of response
to therapy, and selection of treatment.
Strategies are needed that will guide the successful transfer of
research and discovery-level assays into the clinic. This includes
their use in clinical trials, so that the analytical and clinical
validity of the test procedure and outcome are assured. As a general
rule, the requirements for analytical and clinical validation of IVDs
are much greater than the studies that are commonly performed in a
research and development setting. To support the least burdensome
approach to assay development, FDA is willing to discuss unconventional
approaches to IVD validation driven by, for example, the theoretical
precision of multiple reaction monitoring assays. However, theoretical
performance must be balanced by the recognition that there are few, if
any, recognized reference standards for the analytes or the assays with
which to assess the performance of proteomic IVDs. The impact of the
lack of standards may be substantial: Assays that combine the
measurements of several, if not dozens, of individual analytes into a
single, actionable ``score'' may require validation of each individual
analyte separately and in combination. Thus, the objective of this
public workshop is to obtain feedback from academia, government,
industry, clinical laboratories, and other stakeholders on the
development of a regulatory approach that may reduce the burden of
assay validation while assuring that the assays are safe and effective.
II. Topics for Discussion at the Public Workshop
We plan to include the following topics at the public workshop.
State of the art: Current state of proteomic IVD landscape
and FDA's perspectives;
Community initiatives: Overview of community (governmental
and non-governmental) initiatives to help standardize proteomic
technologies and provide quality control to discovery;
Success stories: Description of FDA experience in the
clearance of IVDs that use proteomic technologies, with lessons learned
and challenges discovered in bringing proteomic-based assays to the
clinic; and
Case study open discussion: In an open discussion, FDA
will present a hypothetical case study that includes assay design and
validation issues with which FDA has experience. The goal is to
stimulate discussion with attendees regarding what expectations from
FDA are reasonable, what validation by manufacturers is possible and
other challenges inherent in bringing these tests to the clinic and the
Agency. Possible points of discussion will be solicited from the
attendees, and may include:
[cir] How can or should the FDA use community-developed reference
standards/assays to assess IVD validity?
[cir] How can manufacturers assess accuracy in a multiplex/
multipeak assay without a reference standard for the analytes?
[cir] Are there general rules for assay validation that cannot or
should not be applied to different platforms?
[cir] How can or should late-stage validation considerations be
incorporated into early-stage assay development?
Dated: May 7, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014-10787 Filed 5-9-14; 8:45 am]
BILLING CODE 4160-01-P