[Federal Register Volume 79, Number 7 (Friday, January 10, 2014)]
[Notices]
[Pages 1877-1879]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2014-00258]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2013-N-1658]


Characterizing and Communicating Uncertainty in the Assessment of 
Benefits and Risks in Drug Regulatory Decision-Making; Public Workshop; 
Request for Comments

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of Public Workshop; request for public comments.

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SUMMARY: The Food and Drug Administration (FDA or the Agency) is 
announcing the following workshop convened by the Institute of Medicine 
(IOM): ``Characterizing and Communicating Uncertainty in the Assessment 
of Benefits and Risks in Drug Regulatory Decision-Making.'' The purpose 
of the workshop is twofold: To explore potential approaches to 
addressing and communicating uncertainty and to identify key 
considerations on developing, evaluating, and incorporating potential 
approaches for addressing uncertainty into the assessment of benefits 
and risks in the human drug review process. The format of the meeting 
consists of a series of presentations on topics related to uncertainty 
in the assessment of benefits

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and risks, followed by a discussion on those topics with invited 
panelists and audience members. This workshop satisfies an FDA 
commitment that is part of the fifth authorization of the Prescription 
Drug User Fee Act (PDUFA V).

DATES: The public workshop will be held on February 12, 2014, from 8:30 
a.m. to 4:30 p.m. and February 13, 2014, from 8:30 a.m. to 3:30 p.m. 
Registration to attend the public workshop must be received by January 
31, 2014. See the SUPPLEMENTARY INFORMATION section for information on 
how to register for the workshop. Submit either electronic or written 
comments by March 14, 2014.

ADDRESSES: The public workshop will be held at FDA's White Oak Campus, 
10903 New Hampshire Ave., Building 31 Conference Center, Rm. 1503A, 
Silver Spring, MD 20993-0002. Entrance for public workshop attendees 
(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.
    Submit electronic comments to http://www.regulations.gov. Submit 
written comments to the Division of Dockets Management (HFA-305), Food 
and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 
20852. Identify all comments with the docket number found in brackets 
in the heading of this document.

FOR FURTHER INFORMATION CONTACT: Sara Eggers, Food and Drug 
Administration, Center for Drug Evaluation and Research, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 1166, Silver Spring, MD 20993-0002, 301-
796-4904, FAX: 301-847-8443, email: sara.eggers@fda.hhs.gov.

SUPPLEMENTARY INFORMATION:

I. Background

    On July 9, 2012, the President signed into law the Food and Drug 
Administration Safety and Innovation Act (FDASIA) (Pub. L. 112-144). 
Title I of FDASIA reauthorizes PDUFA and provides FDA with the user fee 
resources necessary to maintain an efficient review process for human 
drug and biological products. The reauthorization of PDUFA includes 
performance goals and procedures for the Agency that represents FDA's 
commitments during fiscal years 2013-2017. These commitments are fully 
described in the document entitled ``PDUFA Reauthorization Performance 
Goals and Procedures Fiscal Years 2013 through 2017'' (PDUFA Goals 
Letter), available on FDA's Web site at http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM270412.pdf.
    Section X of the PDUFA Goals Letter, entitled ``Enhancing Benefit-
Risk Assessment in Regulatory Decision-Making,'' includes development 
of a plan to further develop and implement a structured approach to 
benefit-risk assessment in the human drug review process. As part of 
this enhancement, FDA committed to holding two public workshops on 
benefit-risk considerations from the regulator's perspective that will 
begin by the first quarter of fiscal year 2014. The public workshop 
announced in this notice will fulfill the first of the two workshop 
commitments.
    As part of its commitment, FDA has published the ``Structured 
Approach to Benefit-Risk Assessment in Drug Regulatory Decision-Making: 
Draft PDUFA V Implementation Plan,'' available on FDA's Web site at 
http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM329758.pdf. In this plan, FDA identified as 
an area of further development the exploration of structured approaches 
to evaluate and communicate the uncertainty in the assessment of 
benefits and risks. FDA's human drug regulatory decisions are informed 
by an extensive body of evidence on the safety and efficacy of a drug 
product. In many cases, this evidence is subject to uncertainty arising 
from many sources. One example is the uncertainty in the degree to 
which premarket clinical trial data translates to the postmarket 
setting after the drug is approved and used in a much wider patient 
population. Another example is uncertainty about a potential safety 
signal that emerges in the postmarket setting, where the basis for the 
finding comes from multiple evidence sources of varying levels of 
rigor. Drawing conclusions in the face of uncertainty can be a complex 
and challenging task. Furthermore, being explicit about the impact of 
uncertainty on decision-making is an important part of communicating 
regulatory decisions.

II. Purpose and Scope of the Workshop

    This 2-day public workshop is being convened by IOM. The public 
workshop will explore more systematic and structured approaches to 
evaluate and communicate: (1) The sources of uncertainty in the 
assessment of benefits and risks and (2) their implications on human 
drug regulatory decisions. Specifically, the workshop will explore 
potential analytical and communication approaches to addressing and 
communicating uncertainty and identify key considerations on 
developing, evaluating, and incorporating potential approaches for 
addressing uncertainty into the assessment of benefits and risks in the 
human drug review process. This public workshop will consider the 
entire drug development life cycle, including premarket drug review and 
postmarket safety surveillance. The format of the meeting consists of a 
series of presentations on topics related to uncertainty in the 
assessment of benefits and risks, followed by a discussion on those 
topics with invited panelists and audience members.

III. Attendance and Registration

    FDA's Conference Center at the White Oak location is a Federal 
facility with security procedures and limited seating. Persons 
interested in attending the public workshop must register online by 
January 31, 2014. To register for the public workshop, please visit 
FDA's workshop Web site at http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm378861.htm. Early registration is 
recommended. Registration is free and will be on a first-come, first-
served basis. However, the number of participants from each 
organization may be limited based on space limitations. Registrants 
will receive confirmation once they have been accepted. Onsite 
registration on the day of the workshop will be based on space 
availability. If you need special accommodations because of disability, 
please contact Sara Eggers (see FOR FURTHER INFORMATION CONTACT) at 
least 7 days before the workshop. More information will be made 
available on FDA's workshop Web site at least 5 days before the 
workshop date.
    A live Webcast of this workshop will be viewable on FDA's workshop 
Web site at http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm378861.htm on the day of the workshop. A 
video recording of the workshop will be available on FDA's workshop Web 
site approximately 1 week following the workshop. IOM will 
independently prepare a summary of the workshop, and the summary will 
be available on FDA's workshop Web site approximately 10 months 
following the workshop.

IV. Comments

    Regardless of attendance at the public workshop, interested persons 
may submit either electronic comments or written comments to the 
Division of Dockets Management (see ADDRESSES). It is only necessary to 
send one set of

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comments. Identify comments with the docket number found in brackets in 
the heading of this document. To ensure consideration, submit comments 
by March 14, 2014. 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.

    Dated: January 6, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014-00258 Filed 1-9-14; 8:45 am]
BILLING CODE 4160-01-P