
[Federal Register Volume 81, Number 86 (Wednesday, May 4, 2016)]
[Notices]
[Pages 26799-26800]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2016-10391]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2016-D-1174]


Special Protocol Assessment; Draft Guidance for Industry; 
Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice.

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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing 
the availability of a draft guidance for industry entitled ``Special 
Protocol Assessment.'' This draft guidance provides information about 
the procedures and general policies adopted by the Center for Drug 
Evaluation and Research (CDER) and the Center for Biologics Evaluation 
and Research (CBER) for special protocol assessment (SPA). This draft 
guidance is intended to improve the quality of Requests for SPAs and 
accompanying submission materials, and the quality of the resulting 
interaction between sponsors and FDA. This draft guidance revises the 
guidance for industry entitled ``Special Protocol Assessment'' issued 
May 17, 2002.

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draft guidance before it begins work on the final version of the 
guidance, submit either electronic or written comments on the draft 
guidance by July 5, 2016.

ADDRESSES: You may submit comments as follows:

Electronic Submissions

    Submit electronic comments in the following way:
     Federal eRulemaking Portal: http://www.regulations.gov. 
Follow the instructions for submitting comments. Comments submitted 
electronically, including attachments, to http://www.regulations.gov 
will be posted to the docket unchanged. Because your comment will be 
made public, you are solely responsible for ensuring that your comment 
does not include any confidential information that you or a third party 
may not wish to be posted, such as medical information, your or anyone 
else's Social Security number, or confidential business information, 
such as a manufacturing process. Please note that if you include your 
name, contact information, or other information that identifies you in 
the body of your comments, that information will be posted on http://www.regulations.gov.
     If you want to submit a comment with confidential 
information that you do not wish to be made available to the public, 
submit the comment as a written/paper submission and in the manner 
detailed (see ``Written/Paper Submissions'' and ``Instructions'').

Written/Paper Submissions

    Submit written/paper submissions as follows:
     Mail/Hand delivery/Courier (for written/paper 
submissions): Division of Dockets Management (HFA-305), Food and Drug 
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
     For written/paper comments submitted to the Division of 
Dockets Management, FDA will post your comment, as well as any 
attachments, except for information submitted, marked and identified, 
as confidential, if submitted as detailed in ``Instructions.''
    Instructions: All submissions received must include the Docket No. 
FDA-2016-D-1174 for ``Special Protocol Assessment; Draft Guidance for 
Industry; Availability.'' Received comments will be placed in the 
docket and, except for those submitted as ``Confidential Submissions,'' 
publicly viewable at http://www.regulations.gov or at the Division of 
Dockets Management between 9 a.m. and 4 p.m., Monday through Friday.
     Confidential Submissions--To submit a comment with 
confidential information that you do not wish to be made publicly 
available, submit your comments only as a written/paper submission. You 
should submit two copies total. One copy will include the information 
you claim to be confidential with a heading or cover note that states 
``THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.'' The Agency will 
review this copy, including the claimed confidential information, in 
its consideration of comments. The second copy, which will have the 
claimed confidential information redacted/blacked out, will be 
available for public viewing and posted on http://www.regulations.gov. 
Submit both copies to the Division of Dockets Management. If you do not 
wish your name and contact information to be made publicly available, 
you can provide this information on the cover sheet and not in the body 
of your comments and you must identify this information as 
``confidential.'' Any information marked as ``confidential'' will not 
be disclosed except in accordance with 21 CFR 10.20 and other 
applicable disclosure law. For more information about FDA's posting of 
comments to public dockets, see 80 FR

[[Page 26800]]

56469, September 18, 2015, or access the information at: http://www.fda.gov/regulatoryinformation/dockets/default.htm.
    Docket: For access to the docket to read background documents or 
the electronic and written/paper comments received, go to http://www.regulations.gov and insert the docket number, found in brackets in 
the heading of this document, into the ``Search'' box and follow the 
prompts and/or go to the Division of Dockets Management, 5630 Fishers 
Lane, Rm. 1061, Rockville, MD 20852.
    Submit written requests for single copies of the draft guidance to 
the Division of Drug Information, Center for Drug Evaluation and 
Research, Food and Drug Administration, 10001 New Hampshire Ave., 
Hillandale Building., 4th Floor, Silver Spring, MD 20993-0002, or 
Office of Communication, Outreach, and Development, Center for 
Biologics Evaluation and Research, 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 draft guidance document.

FOR FURTHER INFORMATION CONTACT: Amalia Himaya, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 22, Rm. 6439, Silver Spring, MD 20993-0002, 301-
796-0700; or Stephen Ripley, Center for Biologics Evaluation and 
Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 
71, Rm. 3128, Silver Spring, MD 20993-0002, 240-402-7911.

SUPPLEMENTARY INFORMATION: 

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``Special Protocol Assessment.'' SPA is a process by which 
sponsors may request to meet with FDA to reach agreement on the design 
and size of certain trials, clinical studies, or animal trials to 
determine if they adequately address scientific and regulatory 
requirements. After completing the SPA review, FDA issues a letter 
including an assessment of the protocol, agreement or nonagreement with 
the proposed protocol, and answers to the sponsor's relevant questions. 
Section 119 of the Food and Drug Administration Modernization Act of 
1997 amended section 505(b) of the Federal Food, Drug, and Cosmetic Act 
(the FD&C Act) (21 U.S.C. 355(b)) and directed FDA to meet with 
sponsors who request to meet, provided certain conditions are met, to 
reach agreement on the design and size of the well-controlled clinical 
trials intended to form the primary basis for a demonstration of 
effectiveness in a marketing application submitted under section 505(b) 
of the FD&C Act or section 351 of the Public Health Service (PHS) Act 
(42 U.S.C. 262). These provisions subsequently were amended in section 
7002(d)(1) of the Biologics Price Competition and Innovation Act of 
2009 to include any necessary clinical study or studies for biosimilar 
biological product applications under section 351(k) of the PHS Act. In 
2013, the Pandemic and All Hazards Preparedness Reauthorization Act of 
2013 (Pub. L. 113-5) further amended the SPA provisions to provide for 
SPA agreements regarding animal and associated clinical trials 
conducted in support of applications for products developed under 21 
CFR part 314 subpart I, and 21 CFR part 601 subpart H (the animal 
rule). Such marketing applications include new drug applications 
(NDAs), biologics license applications (BLAs), and efficacy supplements 
to approved NDAs and BLAs.
    In conjunction with the Prescription Drug User Fee Amendments of 
2012 (PDUFA V), enacted as part of the Food and Drug Administration 
Safety and Innovation Act (FDASIA), and with the Biosimilar User Fee 
Act of 2012 (BsUFA), enacted as part of FDASIA, FDA agreed to specific 
performance goals (PDUFA V goals and BsUFA goals, respectively) for 
SPA. Per section 505(b)(5)(B) of the FD&C Act, the PDUFA V goals, and 
the BsUFA goals, the following protocols are eligible for SPA: (1) 
Animal carcinogenicity protocols; (2) drug substance and drug product 
stability protocols; (3) animal efficacy protocols for studies intended 
to provide primary evidence of effectiveness required for approval or 
for licensure for products developed under the animal rule; (4) 
protocols for clinical trials or studies intended to form the primary 
basis of an efficacy claim; and (5) protocols for clinical studies 
necessary to prove biosimilarity and/or interchangeability.
    This draft guidance revises the guidance of the same name issued in 
May 2002. After it has been finalized, this guidance will replace the 
May 2002 guidance. Significant changes from the 2002 version include 
the following: (1) Clarifying which protocols are eligible for SPA; (2) 
adding animal rule efficacy protocols intended to support approval 
under part 314 subpart I, and part 601 subpart H, for drugs and 
biological products, respectively; (3) adding protocols intended to 
support approval of a biosimilar biological product; (4) providing 
greater detail about the content of an SPA submission; and (5) 
clarifying the process for rescinding an SPA agreement. FDA seeks 
comments to aid in finalizing this draft guidance.
    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 current thinking of FDA on the procedural 
aspects of SPA. It does not establish any rights for any person and is 
not binding on FDA or the public. You can use an alternative approach 
if it satisfies the requirements of the applicable statutes and 
regulations.

II. The Paperwork Reduction Act of 1995

    This draft guidance refers to previously approved collections of 
information 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 referred to in the guidance 
entitled ``Special Protocol Assessment'' have been approved under OMB 
control number 0910-0470. The collections of information for FDA Form 
1571 have been approved under OMB control number 0910-0014.

III. Electronic Access

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

    Dated: April 28, 2016.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2016-10391 Filed 5-3-16; 8:45 am]
 BILLING CODE 4164-01-P


