Document ID: FDA-2014-N-0001-0097
Agency: fda
Document Type: Notice
Title: Developing and Using Precision Therapies in the ‘‘Omics’’ Era:
Generating and Interpreting Evidence for Rare Subsets; Public Workshop
Posted Date: 2014-11-21T05:00Z

[Federal Register Volume 79, Number 225 (Friday, November 21, 2014)]
[Notices]
[Pages 69497-69498]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2014-27523]

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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2014-N-0001]

Developing and Using Precision Therapies in the ``Omics'' Era: 
Generating and Interpreting Evidence for Rare Subsets; Public Workshop

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public workshop.

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SUMMARY: The Food and Drug Administration (FDA or the Agency) is 
announcing a public workshop entitled ``Developing and Using Precision 
Therapies in the `Omics' Era: Generating and Interpreting Evidence for 
Rare Subsets.'' This public workshop is being cosponsored with the 
Center for Translational and Regulatory Sciences at the University of 
Virginia (UVA). The goals of this public workshop are to facilitate 
discussion on current scientific approaches using rare subsets during 
drug development programs and to further seek input from multiple 
stakeholders on approaches to obtain evidence that inform the 
regulatory evaluation of therapeutic products in rare subsets of 
patients identified through in-vitro diagnostic testing when specific, 
controlled trials are not feasible.

DATES: The public workshop will be held on December 12, 2014, from 9 
a.m. to 5 p.m. Individuals who wish to attend the public workshop in 
person or via a live Webcast must register online by December 1, 2014, 
at: https://www.signup4.net/Public/ap.aspx?OID=130&EID=DEVE96E. Section 
II of this document provides attendance and registration information.

ADDRESSES: The public workshop will be held at the FDA White Oak 
Campus, 10903 New Hampshire Ave., Bldg. 31 Conference Center, the Great 
Room (Rm. 1503A), 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: Padmaja Mummaneni, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 2164, Silver Spring, MD 20993-0002, 301-
796-2027, email: padmaja.mummaneni@fda.hhs.gov.

SUPPLEMENTARY INFORMATION:

I. Background

    Therapeutic products are increasingly targeted to patients who have 
molecular characteristics that are diagnostic of a particular subtype 
of disease, prognostic for better or worse outcomes, or predictive of 
treatment response. The advent of next-generation sequencing and other 
high throughput technologies has enabled the development of in-vitro 
diagnostic tests that are able to detect rare molecular variations, 
specifically in the patient, tumor, or microbial DNA sequence. FDA and 
UVA are cosponsoring an open public workshop among stakeholders in the 
pharmaceutical industry, representatives from academia, regulatory 
scientists, and other interested parties on the development and usage 
of diagnostic and therapeutic products that respectively have the 
potential to identify and treat patients with rare molecular 
characteristics. It is important for regulatory agencies, 
pharmaceutical and diagnostic industries, and the medical community, 
including payers, to have a mutual

[[Page 69498]]

understanding of various forms of evidence that could inform regulatory 
and medical decision making. The public workshop will help identify key 
components of such an evidence framework when therapeutic effectiveness 
is being evaluated in patients with molecular characteristics that are 
rare or have not been studied in clinical trials.

II. Attendance and Registration

    The FDA Conference Center at the White Oak location is a Federal 
facility with security procedures and limited seating. Individuals who 
wish to attend the public workshop must register on or before December 
1, 2014, by visiting: https://www.signup4.net/Public/ap.aspx?OID=130&EID=DEVE96E.
    Early registration is recommended. Registration is free and will be 
on a first-come, first-served basis. However, FDA may limit the number 
of participants from each organization based on space limitations. On-
site registration on the day of the public workshop will be based on 
space availability.
    FDA will provide additional background information at the time the 
Federal Register notice is published and an agenda approximately 2 
weeks before the public workshop at the FDA Meeting Information page, 
which is available online at:  http://wcms.fda.gov/FDAgov/Drugs/NewsEvents/ucm416622.htm?SSContributor=true.
    If you need special accommodations because of disability, please 
contact Padmaja Mummaneni (see FOR FURTHER INFORMATION CONTACT) at 
least 7 days before the public workshop.
    A live Webcast of this public workshop will be viewable on Adobe 
Connect at https://collaboration.fda.gov/rsw2014/ on the day of the 
public workshop.

    Dated: November 17, 2014.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2014-27523 Filed 11-20-14; 8:45 am]
BILLING CODE 4164-01-P