
[Federal Register Volume 78, Number 123 (Wednesday, June 26, 2013)]
[Notices]
[Pages 38349-38351]
From the Federal Register Online via the Government Printing Office [www.gpo.gov]
[FR Doc No: 2013-15250]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2013-D-0575]


Draft Guidance for Industry on Expedited Programs for Serious 
Conditions--Drugs and Biologics; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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SUMMARY: The Food and Drug Administration (FDA) is announcing the 
availability of a draft guidance for industry entitled ``Expedited 
Programs for Serious Conditions--Drugs and Biologics.'' The purpose of 
this draft guidance is to provide a single resource for information on 
FDA's policies and procedures related to expedited drug development and 
review programs. The following programs are intended to facilitate and 
expedite development and review of new drugs to address unmet medical 
need in the treatment of serious or life-threatening conditions 
(expedited programs): Fast track designation, breakthrough therapy 
designation, accelerated approval, and priority review designation.

DATES: Although you can comment on any guidance at any time (see 21 CFR 
10.115(g)(5)), to ensure that the Agency considers your comment on this 
draft guidance before it begins work on the final version of the 
guidance, submit either electronic or written comments on the draft 
guidance by August 26, 2013. Submit either electronic or written 
comments concerning the proposed collection of information by August 
26, 2013.

ADDRESSES: Submit written requests for single copies of the draft 
guidance to the Division of Drug Information, Center for Drug 
Evaluation and Research (CDER), Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 2201, Silver Spring, MD 20993-0002; or 
the Office of Communication, Outreach, and Development (HFM-40), Center 
for Biologics Evaluation and Research (CBER), Food and Drug 
Administration, 1401 Rockville Pike, Suite 200N, Rockville, MD 20852-
1448. Send one self-addressed adhesive label to assist that office in 
processing your requests. See the SUPPLEMENTARY INFORMATION section for 
electronic access to the draft guidance document.

[[Page 38350]]

    Submit electronic comments on the draft guidance to http://www.regulations.gov.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.

FOR FURTHER INFORMATION CONTACT: Melissa Robb, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 6360, Silver Spring MD 20993-0002, 301-
796-2500; 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

    FDA is announcing the availability of a draft guidance for industry 
entitled ``Expedited Programs for Serious Conditions--Drugs and 
Biologics.'' This draft guidance provides a single resource for 
information on FDA's policies and procedures related to the following 
expedited programs for serious conditions: (1) Fast track designation, 
(2) breakthrough therapy designation, (3) accelerated approval, and (4) 
priority review designation. The draft guidance describes threshold 
criteria generally applicable to expedited programs, including what is 
meant by serious condition, unmet medical need, and available therapy. 
This draft guidance also discusses considerations for expedited 
development and review such as manufacturing scale-up and inspections, 
long-term nonclinical toxicity studies, and review cycle clinical 
inspections. In addition, this guidance aligns CDER's criteria for 
priority review designation with CBER's criteria. Only products 
intended to treat a serious condition are eligible for priority review 
(unless otherwise eligible under specific statutory provisions).
    For over 30 years, expediting the availability of promising 
therapies to patients with serious conditions has been a priority for 
FDA. With the passage of the Food and Drug Administration Safety and 
Innovations Act (FDASIA), FDA is expanding its efforts to expedite 
development and review of therapies intended to treat patients with 
serious conditions. This draft guidance is intended to satisfy the 
statutory requirements of sections 901(c)(1) and 902(b)(1)(A) of 
FDASIA.
    Section 901(c)(1) of FDASIA requires FDA to issue draft guidance to 
implement amendments to the Federal Food, Drug, and Cosmetic Act (the 
FD&C Act) (Enhancement of Accelerated Approval Access to New Medical 
Treatments) within 1 year of the date of enactment. The fast track 
designation, accelerated approval, and other relevant provisions of 
this draft guidance are intended to fulfill this requirement.
    Section 902(b)(1)(A) of FDASIA requires FDA to issue draft guidance 
to implement requirements of section 902 (Breakthrough Therapies) 
within 18 months of the date of enactment. The breakthrough therapy and 
other relevant provisions of this draft guidance are intended to 
fulfill this requirement.
    The provisions of this draft guidance relating to fast track 
development and other issues such as serious condition and unmet 
medical need, when finalized, will replace the current guidance for 
industry entitled ``Fast Track Drug Development Programs--Designation, 
Development, and Application Review.'' The provisions of this draft 
guidance pertaining to available therapy, when finalized, will replace 
the current guidance for industry entitled ``Available Therapy.''
    This draft guidance is being issued consistent with FDA's good 
guidance practices regulation (21 CFR 10.115). The draft guidance, when 
finalized, will represent the Agency's current thinking on expedited 
programs for serious conditions--drugs and biologics. It does not 
create or confer any rights for or on any person and does not operate 
to bind FDA or the public. An alternative approach may be used if such 
approach satisfies the requirements of the applicable statutes and 
regulations.

II. Paperwork Reduction Act of 1995

    Under the Paperwork Reduction Act of 1995 (the PRA) (44 U.S.C. 
3501-3520), Federal Agencies must obtain approval from the Office of 
Management and Budget (OMB) for each collection of information that 
they conduct or sponsor. ``Collection of information'' is defined in 44 
U.S.C. 3502(3) and 5 CFR 1320.3(c) and includes Agency requests or 
requirements that members of the public submit reports, keep records, 
or provide information to a third party. Section 3506(c)(2)(A) of the 
PRA (44 U.S.C. 3506(c)(2)(A)) requires Federal Agencies to provide a 
60-day notice in the Federal Register for each proposed collection of 
information before submitting the collection to OMB for approval. To 
comply with this requirement, FDA is publishing this notice of the 
proposed collection of information set forth in this document.
    With respect to the collection of information associated with this 
draft guidance, FDA invites comments on the following topics: (1) 
Whether the proposed information collected is necessary for the proper 
performance of FDA's functions, including whether the information will 
have practical utility; (2) the accuracy of FDA's estimated burden of 
the proposed information collected, including the validity of the 
methodology and assumptions used; (3) ways to enhance the quality, 
utility, and clarity of the information collected; and (4) ways to 
minimize the burden of information collected on the respondents, 
including through the use of automated collection techniques, when 
appropriate, and other forms of information technology.
    Title: Submission of Information Related to Expedited Programs for 
Serious Conditions--Drugs and Biologics.
    Description of Respondents: Respondents to this collection of 
information are sponsors that develop drugs and biological products.
    Burden Estimate: This draft guidance outlines FDA's policies and 
procedures related to the following expedited programs for serious 
conditions: (1) Fast track designation, including rolling review, (2) 
breakthrough therapy designation, (3) accelerated approval, and (4) 
priority review designation. In addition, this draft guidance describes 
threshold criteria generally applicable to expedited programs.
    This draft guidance also refers to previously approved collections 
of information found in FDA regulations. The collections of information 
in 21 CFR 202.1, parts 314 and 601 (21 CFR parts 314 and 601), and 
sections 505(a), 506(a)(1), 735, and 736 of the FD&C Act (21 U.S.C. 
355(a), 356(a)(1), 379g, and 379h) have been approved under OMB control 
numbers 0910-0686, 0910-0001, 0910-0338, 0910-0014, and 0910-0297.
    This draft guidance proposes the following new collections of 
information:
    Priority Review Designation Request. The draft guidance describes 
that a sponsor may expressly request priority review of an application. 
Based on information from FDA's databases and information available to 
FDA, we estimate that approximately 47 sponsors will prepare and submit 
approximately 1 priority review designation submission in accordance 
with the draft guidance and that the added burden for each submission 
will be approximately 30 hours to develop and submit to FDA as part of 
the application (totaling 1,410 hours).
    Breakthrough Therapy Designation Request. The draft guidance 
describes the process for sponsors to request breakthrough therapy 
designation in an

[[Page 38351]]

application. Based on information available to FDA, we estimate that 
approximately 24 sponsors will prepare approximately 1 breakthrough 
therapy designation submission in accordance with the draft guidance 
and that the added burden for each submission will be approximately 70 
hours to prepare and submit (totaling 1,680 hours).
    Promotional Materials for Accelerated Approval Under Part 314. The 
draft guidance describes section 506(c)(2)(B) of the FD&C Act and FDA's 
accelerated approval regulations (Sec. Sec.  314.550 and 601.45). These 
provisions authorize FDA to require sponsors to submit copies of all 
promotional materials to the Agency for consideration prior to their 
dissemination. The regulations provide that copies of all promotional 
materials including promotional labeling as well as advertisements 
intended for dissemination or publication within 120 days following 
marketing approval must be submitted to FDA during the preapproval 
period. The regulations further provide that after 120 days following 
marketing approval, unless otherwise informed by the Agency, the 
applicant must submit promotional materials at least 30 days prior to 
the intended time of initial dissemination of the labeling or initial 
publication of the advertisement. Currently, FDA has OMB approval for 
the submission of copies of all promotional materials under part 601 
(OMB control number 0910-0338) but does not have approval for the 
submission of copies of all promotional materials under part 314.
    Based on information from FDA's databases and information available 
to FDA, we estimate that approximately 20 sponsors will submit 
promotional materials for accelerated approval 7 times annually in 
accordance with Sec.  314.550 and that the burden for each submission 
will be approximately 120 hours (a total of 16,800 hours).
    FDA estimates the burden of this collection of information as 
follows:

                                 Table 1--Estimated Annual Reporting Burden \1\
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                                                     Number of
   Draft guidance on expedited       Number of     responses per   Total annual   Average burden    Total hours
            programs                respondents     respondent       responses     per response
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Priority Review Designation                   47               1              47              30           1,410
 Request........................
Breakthrough Therapy Designation              24               1              24              70           1,680
 Request........................
Promotional Materials for                     20               7             140             120          16,800
 Accelerated Approval Under Sec.
   314.550......................
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    Total.......................  ..............  ..............  ..............  ..............          19,890
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\1\ There are no capital costs or operating and maintenance costs associated with this collection of
  information.

III. Comments

    Interested persons may submit either electronic comments regarding 
this document to http://www.regulations.gov or written comments to the 
Division of Dockets Management (see ADDRESSES). 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, and will be posted to the docket at http://www.regulations.gov.

IV. Electronic Access

    Persons with access to the Internet may obtain the document at 
either http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/default.htm, or http://www.regulations.gov.

    Dated: June 20, 2013.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2013-15250 Filed 6-25-13; 8:45 am]
BILLING CODE 4160-01-P


