[Federal Register Volume 78, Number 49 (Wednesday, March 13, 2013)]
[Notices]
[Pages 15953-15955]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2013-05726]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2013-N-0010]


Cooperative Agreement To Support Regulatory Research Related to 
Food and Drug Administration Commitments Under the 2012 Prescription 
Drug User Fee Act

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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SUMMARY: The Food and Drug Administration (FDA) announces its intention 
to accept and consider a single source application for award of a 
cooperative agreement to the Brookings Institution's Engelberg Center 
for Health Care Reform (ECHCR) in support of efforts to inform major 
initiatives for process improvement and regulatory science related to 
FDA commitments under the 2012 reauthorization of the Prescription Drug 
User Fee Act (PDUFA V).

DATES: Important dates are as follows:
    1. The application due date is April 15, 2013.
    2. The anticipated start date is June 1, 2013.
    3. The expiration date is April 16, 2013.
    For Further Information and Additional Requirements Contact:
Adam Kroetsch, Office of Planning and Analysis, Food and Drug 
Administration, 10903 New Hampshire Ave., Bldg. 51, Rm. 1192, Silver 
Spring, MD 20993, 301-796-3842, [email protected];

or

Yemisi Akinneye, Office of Acquisitions and Grants Services, Food and 
Drug Administration, 5630 Fishers Lane, HFA 500, Rm. 2037, Rockville, 
MD 20857, 301-827-0079, [email protected].

    For more information on this funding opportunity announcement (FOA) 
and to obtain detailed requirements, please refer to the full FOA 
located at http://grants2.nih.gov/grants/guide/and/or http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm093567.htm.

SUPPLEMENTARY INFORMATION:

I. Funding Opportunity Description

RFA-FD-13-005; 93.103.

A. Background

    The FDA Center for Drug Evaluation and Research (CDER) seeks to 
support efforts to research, identify key issues, and convene 
appropriate subject matter experts to help inform major initiatives for 
process improvement and regulatory

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science related to FDA commitments under PDUFA V. PDUFA, first enacted 
in 1992, has provided FDA with the resources and process enhancements 
to enable a transformation of the human drug review process, increasing 
the quality, number, and timely access to new drugs for U.S. patients.
    The 2012 reauthorization of PDUFA initiated a set of performance 
goals and procedures for FDA through fiscal year 2017. These 
performance goals represent a series of commitments which were 
established in consultation with drug industry representatives, patient 
and consumer advocates, and health care professionals. Specific PDUFA 
commitments include public meetings, staff training procedures, and 
efficiency standards on a variety of issues. More information about 
FDA's commitments under PDUFA V can be found at the following Web site: 
http://www.fda.gov/downloads/forindustry/userfees/prescriptiondruguserfee/ucm270412.pdf.

B. Research Objectives

    In the most recent reauthorization of PDUFA, FDA has committed to 
build on a record of continuing improvement through a wide range of new 
innovative initiatives related to virtually every aspect of the new 
drug life cycle, each of which represent specific areas of research 
interest. These initiatives may include, but not be limited to, the 
following:
     Enhancing regulatory science and expediting drug 
development;
     Advancing metaanalysis methods;
     Advancing the use of biomarkers and pharmacogenomics;
     Developing and enhancing patient-reported outcomes to 
support patient-focused drug development;
     Facilitating rare disease drug development;
     Structured approaches to enhancing FDA's assessment of 
benefits and risks in human drugs;
     Improving evaluation, standardization, and integration of 
Risk Evaluation and Mitigation Strategies (REMS);
     Exploring the use of Sentinel as a tool for evaluating 
drug safety issues; and
     Requiring electronic submissions and standardization of 
electronic application data.
    Several key areas of research interest are described in greater 
detail below:
1. Developing and Enhancing Patient-Reported Outcomes To Support 
Patient-Focused Drug Development
    The advancement of patient-reported outcome measures (PROs) is 
designed to promote patient engagement throughout the drug development 
process. FDA has dedicated steps toward the development of these tools 
by expanding clinical and statistical staff capacity, providing 
qualification consultations, and promoting best practices for the use 
of outcome assessment tools. FDA seeks to identify the challenges and 
opportunities within the current review and qualification of PROs, to 
address key issues with PRO evidentiary standards, develop new methods 
for communication between the multiple stakeholders involved in PROs, 
and identify best practices for evaluation and statistical analysis and 
design of PROs.
2. Structured Approaches to Enhancing FDA's Assessment of Benefits and 
Risks in Human Drugs
    FDA recognizes that the Agency's efforts to develop a more 
structured approach to benefit-risk assessment could be complemented by 
further engagement of stakeholders and other parties. This engagement 
seeks to focus on the current efforts and methods that have been 
applied to structure and communicate regulatory decisions, including 
the relevance to the work of a regulator and how well such approaches 
integrate with how regulators think about their decisions. FDA expects 
that these discussions would focus on the results of implementing 
frameworks at regulatory agencies both in premarket application review 
as well as post-market safety review, providing an opportunity to share 
challenges and lessons learned in applying a more structured approach 
to regulatory decision-making.
3. Improving Evaluation, Standardization, and Integration of REMS
    FDA seeks stakeholder and expert feedback on approaches to 
standardizing of REMS and integrating them into the health care 
delivery system. Areas for research include the following:
     A standardized methodology for selecting appropriate risk 
management interventions when a REMS is deemed necessary. Such a 
methodology should allow FDA and sponsors to proactively identify and 
address the underlying causes of patient harm, and evaluate and 
prioritize risk management interventions based on evidence of their 
effectiveness and burden on the health care delivery system.
     Standard approaches and best practices for implementing 
REMS and integrating them into the existing health care delivery 
system. These approaches may include the use of improved methods for 
communicating with and training REMS stakeholders and the use of 
information technology to facilitate REMS implementation.
     Standard methods to evaluate REMS, including methods to 
assess REMS effectiveness, impact on patient access, and burden on the 
health care delivery system.

C. Approach

    In order to achieve these research objectives as part of its PDUFA 
V commitments, FDA has committed to seek input from relevant external 
subject matter experts and other interested public stakeholders. In 
addition, this input process should be conducted so as to be timely, 
well-informed, candid, thoughtful, thorough, and well-documented.
    FDA has a limited capacity to conduct the needed research to fully 
inform and undertake these external expert engagements to ensure the 
successful accomplishment of these PDUFA V commitments. FDA is 
therefore seeking to establish a cooperative agreement with the 
Brookings Institution's ECHCR for its unique qualifications and 
experience in the conduct of the needed research, workshops and other 
meetings, and related work.
    The goal of this collaboration is to support the implementation of 
PDUFA V performance goals by convening stakeholders with diverse 
expertise. Through a series of meetings, workshops, webinars, and/or 
workgroups, ECHCR would provide effective opportunities for engagement 
of these stakeholders to inform implementation of the PDUFA V goals. In 
addition to gathering input from selected stakeholder groups, ECHCR may 
conduct background research prior to expert engagement, and to 
communicate updates on the progress of PDUFA implementation to broader 
audiences. Specific objectives of this collaboration would include:
     Working collaboratively with FDA to identify and 
prioritize pressing issues related to the implementation of PDUFA 
reauthorization performance goals and procedures;
     Conducting research and reviews of relevant literature to 
plan the focus of sessions in which experts are convened to provide 
critical input to FDA regulatory enhancement discussions;
     Convening expert stakeholders in focused, substantive 
discussions of these issues, and identify and explore potential 
strategies for resolving them; and
     Developing reports that summarize the background research 
and discussion

[[Page 15955]]

at each meeting and post these reports for public access.

D. Eligibility Information

    The following organization is eligible to apply: ECHCR. Within the 
Brookings Institution, the mission of the ECHCR is to provide practical 
solutions to achieve high-quality, innovative, affordable health care 
with particular emphasis on identifying opportunities on the national, 
State, and local levels. Leveraging its status as a neutral, nonprofit, 
research-focused institution with deep health care policy and technical 
expertise, ECHCR frequently serves as a convener of discussions, 
workshops, and symposia on complex policy and science topics. The 
Center has developed a reputation as an ``honest broker'' with the 
ability to identify practical solutions that reflect the best available 
science and input from all stakeholders. The performance goals and 
procedures outlined within PDUFA V will require a high degree of 
leadership, research, outreach, and involvement from a broad range of 
stakeholders across the health care system. ECHCR is uniquely qualified 
to conduct the background research and act as a convener for engaging 
critical stakeholders, raising awareness, and identifying practical 
solutions that identify and overcome potential challenges and help 
determine a clear path forward.

II. Award Information/Funds Available

A. Award Amount

    FDA intends to fund one award, corresponding to a total of 
$700,000, for fiscal year (FY) 2013. Future year amounts will depend on 
annual appropriations. CDER anticipates providing in FY2013 up to 
$700,000 (total costs include direct and indirect costs) for one award 
subject to availability of funds in support of this project. The 
possibility of four additional years of support is contingent upon 
successful performance and the availability of funds, and would provide 
funds up to following amounts:

FY 2014: $721,000
FY 2015: $743,000
FY 2016: $765,000
FY 2017: $788,000

B. Length of Support

    The support will be 1 year with the possibility of an additional 4 
years of noncompetitive support. Continuation beyond the first year 
will be based on satisfactory performance during the preceding year, 
receipt of a noncompeting continuation application and available 
Federal FY appropriations.

III. Paper Application, Registration, and Submission Information

    To submit a paper application in response to this FOA, applicants 
should first review the full announcement located at http://grants2.nih.gov/grants/guide and/or http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm093567.htm. (FDA has verified the 
Web site addresses throughout this document, but FDA is not responsible 
for any subsequent changes to the Web sites after this document 
publishes in the Federal Register.) Persons interested in applying for 
a grant may obtain an application at http://grants.nih.gov/grants/forms.htm. For all paper application submissions, the following steps 
are required:
     Step 1: Obtain a Dun and Bradstreet (DUNS) Number
     Step 2: Register With System for Award Management
    Steps 1 and 2, in detail, can be found at http://www07.grants.gov/applicants/organization_registration.jsp. After you have followed 
these steps, submit paper applications to: Yemisi Akinneye, Grants 
Management, 5630 Fishers Lane, HFA-500, rm. 2037, Rockville, MD.

    Dated: March 8, 2013.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2013-05726 Filed 3-12-13; 8:45 am]
BILLING CODE 4160-01-P