[Federal Register Volume 79, Number 104 (Friday, May 30, 2014)]
[Notices]
[Pages 31117-31118]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2014-12534]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

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


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 guidance for industry entitled ``Expedited Programs 
for Serious Conditions--Drugs and Biologics.'' The purpose of this 
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 a serious or life-threatening condition 
(expedited programs): Fast track designation, breakthrough therapy 
designation, accelerated approval, and priority review designation. 
This guidance finalizes the draft guidance issued in June 2013.

DATES: Submit either electronic or written comments on Agency guidances 
at any time.

ADDRESSES: Submit written requests for single copies of this 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, Center for Biologics 
Evaluation and Research (CBER), Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 71, Rm. 3128, Silver Spring, MD 20993-0002. Send 
one self-addressed adhesive label to assist that office in processing 
your requests. See the SUPPLEMENTARY INFORMATION section for electronic 
access to the guidance document.
    Submit electronic comments on the 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, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 
71, Silver Spring, MD 20993-0002, 240-402-7911.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a guidance for industry 
entitled ``Expedited Programs for Serious Conditions--Drugs and 
Biologics.'' This 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 guidance describes threshold criteria 
generally applicable to expedited programs, including what is meant by 
serious condition, unmet medical need, and available therapy. This 
guidance also discusses considerations for expedited development and 
review such as manufacturing and product quality, nonclinical studies, 
and 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 
Innovation Act (FDASIA) (Public Law 112-122), FDA is expanding its 
efforts to expedite development and review of therapies intended to 
treat patients with serious conditions. This guidance is intended to 
satisfy the statutory requirements of sections 901(c)(2) and 
902(b)(1)(A) of FDASIA.
    Section 901(c)(2) of FDASIA requires FDA to issue a final guidance 
document to implement amendments to the Federal Food, Drug, and 
Cosmetic Act (the FD&C Act) made by section 901 of

[[Page 31118]]

FDASIA (Enhancement of Accelerated Approval Access to New Medical 
Treatments) within 1 year of the date the draft guidance issues. The 
discussions of accelerated approval, and other more broadly applicable 
provisions in this guidance, are intended to meet this requirement.
    Section 902(b)(1)(A) of FDASIA requires FDA to issue a guidance 
document to implement requirements of section 902 (Breakthrough 
Therapies) within 1 year of the date the comment period closes for the 
draft guidance. The breakthrough therapy discussion and other more 
broadly applicable provisions in this guidance are intended to meet 
this requirement.
    In the Federal Register of June 26, 2013 (78 FR 38349), FDA 
announced the availability of the draft guidance entitled ``Expedited 
Programs for Serious Conditions--Drugs and Biologics.'' The notice gave 
the public an opportunity to comment by August 26, 2013. FDA carefully 
considered all comments received in developing the final guidance. This 
guidance addresses the applicability of expedited programs to rare 
diseases, clarification on available therapy, and additional detail on 
possible flexibility in manufacturing and product quality. The guidance 
also includes clarification on the qualifying criteria for breakthrough 
therapy designation and examples of surrogate endpoints and 
intermediate clinical endpoints used to support accelerated approval. 
This guidance finalizes the draft guidance issued in June 2013.
    The provisions of this guidance relating to fast track development 
and other issues such as serious condition and unmet medical need, 
replace the guidance for industry entitled ``Fast Track Drug 
Development Programs--Designation, Development, and Application 
Review.'' The provisions of this guidance pertaining to available 
therapy replace the guidance for industry entitled ``Available 
Therapy.''
    This guidance is being issued consistent with FDA's good guidance 
practices regulation (21 CFR 10.115). The guidance represents 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

    This guidance contains information collection provisions that are 
subject to review by the Office of Management and Budget (OMB) under 
the Paperwork Reduction Act of 1995 (44 U.S.C. 3501-3520). The 
collections of information in 21 CFR 202.1, certain parts of 21 CFR 
part 314, 21 CFR part 601, and sections 506(b)(1), 735, and 736 of the 
FD&C Act (21 U.S.C. 356(b)(1), 379g, and 379h) have been approved under 
OMB control numbers 0910-0686, 0910-0001, 0910-0338, 0910-0389, 0910-
0297, and 0910-0765.

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: May 23, 2014.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2014-12534 Filed 5-29-14; 8:45 am]
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