
[Federal Register Volume 77, Number 130 (Friday, July 6, 2012)]
[Notices]
[Pages 40072-40073]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2012-16529]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2012-N-0603]


Assessment of the Program for Enhanced Review Transparency and 
Communication for New Molecular Entity New Drug Applications and 
Original Biologics License Applications in Prescription Drug User Fee 
Act V; Request for Comments

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice; request for comments.

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SUMMARY: The Food and Drug Administration (FDA) is announcing an 
opportunity for public comment on the statement of work for an 
assessment of the Program for Enhanced Review Transparency and 
Communication for New Molecular Entity (NME) New Drug Applications 
(NDAs) and Original Biologics License Applications (BLAs) (the 
Program). The Program is part of the FDA performance commitments under 
the proposed fifth authorization of the Prescription Drug User Fee Act 
(PDUFA), which, if enacted into law, will allow FDA to collect user 
fees for the review of human drug and biologics applications for fiscal 
years (FYs) 2013-2017. The Program is described in detail in section 
II.B of the document entitled ``PDUFA Reauthorization Performance Goals 
and Procedures Fiscal Years 2013 through 2017.'' \1\ The Program will 
be evaluated by an independent contractor in an interim and final 
assessment. As part of the FDA performance commitment, FDA is providing 
a period of 30 days for public comment on the statement of work before 
letting the contract for the assessment.
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    \1\ The ``PDUFA Reauthorization Performance Goals and Procedures 
Fiscal Years 2013 through 2017'' is available on the Internet at 
http://www.fda.gov/downloads/For Industry/UserFees/
PrescriptionDrugUserFee/UCM270412.pdf.

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DATES: Submit electronic or written comments by August 6, 2012.

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

FOR FURTHER INFORMATION CONTACT: Andrea Tan, Center for Drug Evaluation 
and Research, Food and Drug Administration, 10903 New Hampshire Ave., 
Bldg. 51, Rm. 1173, Silver Spring, MD 20993-0002, 301-796-7641, 
Andrea.Tan@fda.hhs.gov.

SUPPLEMENTARY INFORMATION:

I. Background

    The timely review of the safety and effectiveness of new drugs and 
biologics is central to FDA's mission to protect and promote the public 
health. Before the enactment of PDUFA in 1992, FDA's drug review 
process was relatively slow and not very predictable compared to other 
countries. As a result of concerns expressed by industry, patients, and 
other stakeholders at the time, Congress enacted PDUFA, which provided 
the added funds through user fees that enabled FDA to hire additional 
reviewers and support staff and upgrade its information technology 
systems. In return for these additional resources, FDA agreed to 
certain review performance goals, such as completing reviews of NDAs 
and BLAs and taking regulatory actions on them in predictable 
timeframes. These changes revolutionized the drug approval process in 
the United States and enabled FDA to speed the application review 
process for new drugs and biologics without compromising the Agency's 
high standards for demonstration of safety, efficacy, and quality of 
new drugs and biologics prior to approval.
    PDUFA provides FDA with a source of stable, consistent funding that 
has made possible our efforts to focus on

[[Page 40073]]

promoting innovative therapies and helping to bring to market critical 
products for patients. When PDUFA was originally authorized in 1992, it 
had a 5-year term. The PDUFA program has been reauthorized every 5 
years, with the most recent reauthorization occurring in 2007 for FYs 
2008-2012. As directed by Congress in preparing for reauthorization of 
PDUFA for a new 5-year period, FDA conducted negotiations with 
regulated industry and conducted regular consultations with public 
stakeholders, including patient advocates, consumer advocates, and 
health care professionals between July 2010 and May 2011. Following 
these discussions, related public meetings, and Agency requests for 
public comment, FDA transmitted proposed PDUFA V recommendations to 
Congress for FYs 2013-2017 on January 13, 2012. If enacted into law, 
FDA's proposed PDUFA V recommendations will include an FDA commitment 
to implement a new review program for NME NDAs and original BLAs to 
enhance review transparency and communication between FDA and 
applicants on these complex applications.

II. PDUFA V NME NDA and Original BLA Review Program

    FDA's existing review performance goals for priority and standard 
applications, 6 and 10 months respectively, were established more than 
15 years ago. Since that time, additional requirements in the drug 
review process and scientific advances in drug development have made 
those goals increasingly challenging to meet, particularly for more 
complex applications like NME NDAs and original BLAs that generally are 
discussed in an FDA advisory committee meeting. FDA further recognizes 
that increasing communication between the Agency and applicants during 
FDA's review has the potential to increase efficiency in the review 
process.
    To promote greater transparency and improve communication between 
the FDA review team and the applicant, FDA has proposed a new review 
model for NME NDAs and original BLAs in PDUFA V. The Program provides 
opportunities for increased communication by building in mid-cycle 
communications and late-cycle meetings between FDA and applicants. To 
accommodate this increased interaction during regulatory review and to 
address the need for additional time to review these complex 
applications, FDA's review clock will begin after the 60-day 
administrative filing review period for applications reviewed under the 
Program. The Program will apply to all NME NDAs and original BLAs 
received from October 1, 2012, through September 30, 2017. The goal of 
the Program is to improve the efficiency and effectiveness of the 
first-cycle review process by increasing communication with sponsors 
before application submission to improve the quality and completeness 
of submissions, and by increasing communications during application 
review. This will provide sponsors with opportunities to clarify 
previous submissions and provide additional data and analyses that are 
readily available, potentially avoiding the need for an additional 
review cycle when FDA's concerns about an application can be promptly 
resolved, but without compromising FDA's traditional high standards for 
approval. An efficient and effective review process that allows for 
timely responses to FDA questions can help ensure timely patient access 
to safe, effective, and high quality new drugs and biologics. To 
understand the Program's effect on the review of these applications, 
interim and final assessments by an independent contractor are key 
components of the Program. The performance commitments state that the 
statement of work for this effort will be published for public comment 
before beginning the assessment (section II.B). Because the assessment 
needs to commence at the beginning of PDUFA V on October 1, 2012, if 
the program is reauthorized, FDA must publish the statement of work for 
public comment in advance of that reauthorization to be able to begin 
the assessment on October 1, 2012. Accordingly, FDA is seeking public 
comment now on the proposed statement of work for the assessment of the 
Program, available at http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm304793.pdf.

III. Comments

    Interested persons may submit to the Division of Dockets Management 
(see ADDRESSES) either electronic or written comments regarding this 
document. 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. Received comments may be seen in the Division of Dockets 
Management between 9 a.m. and 4 p.m., Monday through Friday.

    Dated: June 29, 2012.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2012-16529 Filed 7-5-12; 8:45 am]
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