[Federal Register Volume 78, Number 46 (Friday, March 8, 2013)]
[Notices]
[Pages 15019-15020]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2013-05471]



[[Page 15019]]

-----------------------------------------------------------------------

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2013-N-0196]


Food and Drug Administration Prescription Drug User Fee Act V 
Benefit-Risk Plan; Request for Comments

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice, request for comments.

-----------------------------------------------------------------------

SUMMARY: The Food and Drug Administration (FDA or the Agency) is 
announcing the availability of a draft 5-year plan describing the 
Agency's approach to further developing and implementing a structured 
framework for benefit-risk assessment in the human drug and biologic 
review process and the opportunity for public comment on the draft 
plan. This plan is part of FDA's commitments that were made as part of 
the fifth authorization of the Prescription Drug User Fee Act (PDUFA 
V). FDA has published the draft plan on its Web site at http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM329758.pdf.

DATES: Submit either electronic or written comments by May 7, 2013.

ADDRESSES: Submit electronic comments to 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. All comments should be identified with the docket number found 
in brackets in the heading of this document.

FOR FURTHER INFORMATION CONTACT: Graham Thompson, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, rm. 1199, Silver Spring, MD 20993, 301-796-
5003, FAX: 301-847-8443, Email: [email protected]; or Stephen 
Ripley, Center for Biologics Evaluation and Research (HFM-17), Food and 
Drug Administration, 1401 Rockville Pike, suite 200N, Rockville, MD 
20852-1448, 301-827-6210.

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). 
Section 905 of FDASIA amends section 505(d) of the Federal Food, Drug, 
and Cosmetic Act (FD&C Act) by requiring FDA to ``implement a 
structured risk-benefit assessment framework in the new drug approval 
process to facilitate the balanced consideration of benefits and risks, 
a consistent and systematic approach to the discussion and regulatory 
decisionmaking, and the communication of the benefits and risks of new 
drugs.'' 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 represent 
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 Document''), available on FDA's Web site at http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM270412.pdf. Section X of the PDUFA Goals Document, titled 
``Enhancing Benefit-Risk Assessment in Regulatory Decisionmaking,'' 
addresses the development of a 5-year plan that describes the Agency's 
approach to further develop and implement a structured benefit-risk 
framework in its human drug and biologic review process. The 
publication and implementation of this plan are intended to fulfill the 
requirement in section 905 of FDASIA and the commitments described in 
Section X of the PDUFA Goals Document.

II. Draft Plan Describing Structured Approach to Benefit-Risk 
Assessment

    Ensuring the safety, effectiveness, and quality of human drugs is a 
complicated regulatory task, requiring FDA's consideration of a 
multitude of complex factors. FDA's regulatory decision making process 
takes into consideration not only the data submitted in a marketing 
application, but also a broad set of additional factors, including the 
clinical context for the proposed product (such as the nature and 
severity of the disease or condition that the proposed product is 
intended to treat or prevent and the benefits and risks of other 
available therapies for that disease or condition) and any risk 
management tools that might be necessary to ensure that the benefits of 
the proposed product outweigh its risks.
    FDA believes that implementing a standardized structure for the 
analysis of the various benefit and risk considerations that make up a 
regulatory decision will help to facilitate balanced and consistent 
consideration of the benefit and risk factors during the review process 
and to enhance the transparency of regulatory review. FDA therefore has 
developed a draft plan describing a benefit-risk assessment framework 
that is designed to make explicit the consideration of the various 
benefit-risk factors and the role of those factors in the regulatory 
decision-making process for human drug and biological product marketing 
applications. It is important to note that, as specified in section 905 
of FDASIA, this framework does not change the criteria for approval of 
a drug or biological product. All new drug applications and biological 
license applications must meet the requirements for approval under the 
FD&C Act and the Public Health Service Act, respectively.
    By clearly articulating FDA's key considerations in a standard 
structure, this framework can serve as an important tool for the 
analysis and discussion of the relevant benefit and risk considerations 
during the review process. A second and equally important purpose of 
the benefit-risk framework is that it can serve as a tool to 
communicate the reasoning of FDA's regulatory decisions to the public. 
When FDA approves a new drug or biological product, it generally posts 
decisional memos on the Agency's Web site. These documents may be 
highly technical and may not be easily understandable to a broad 
audience with varying backgrounds. The benefit-risk framework aims to 
enhance FDA's communication of its decisions by making clear the 
important considerations in the Agency's decision-making process, and 
how they affected the final regulatory decision, in a clear, succinct 
summary.
    With this notice, FDA is announcing the availability of a draft 5-
year plan describing the Agency's approach to further developing and 
implementing the benefit-risk framework and the opportunity for the 
public to comment on the plan. FDA has published the plan on the 
Agency's Web site at http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM329758.pdf. The comment period will remain 
open for 60 days following the publication of this notice. After 
consideration of public comments, FDA will finalize the plan. 
Throughout PDUFA V, the Agency will update the plan as necessary and 
post all updates on the FDA's Web site.


[[Page 15020]]


    Dated: March 5, 2013.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2013-05471 Filed 3-7-13; 8:45 am]
BILLING CODE 4160-01-P