[Federal Register Volume 86, Number 202 (Friday, October 22, 2021)]
[Notices]
[Pages 58672-58674]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2021-23081]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2021-D-0548]


Data Standards for Drug and Biological Product Submissions 
Containing Real-World Data; Draft Guidance for Industry; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of availability.

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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing 
the availability of a draft guidance for industry entitled ``Data 
Standards for Drug and Biological Product Submissions Containing Real-
World Data.'' This guidance provides recommendations to sponsors to 
help support compliance with the Federal Food, Drug, and Cosmetic Act 
(FD&C Act) when submitting study data derived from real-world data 
(RWD) sources in applicable regulatory submissions using standards 
specified in the Data Standards Catalog (Catalog). FDA is publishing 
this draft guidance as part of a series of guidance documents under its 
program to evaluate the use of real-world evidence (RWE) in regulatory 
decision making.

DATES: Submit either electronic or written comments on the draft 
guidance by December 21, 2021 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 
version of the guidance.

ADDRESSES: You may submit comments on any guidance at any time as 
follows:

Electronic Submissions

    Submit electronic comments in the following way:
     Federal eRulemaking Portal: https://www.regulations.gov. 
Follow the instructions for submitting comments. Comments submitted 
electronically, including attachments, to https://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 https://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): Dockets Management Staff (HFA-305), Food and Drug 
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
     For written/paper comments submitted to the Dockets 
Management Staff, 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-2021-D-0548 for ``Data Standards for Drug and Biological Product 
Submissions Containing Real-World Data.'' Received comments will be 
placed in the docket and, except for those submitted as ``Confidential 
Submissions,'' publicly viewable at https://www.regulations.gov or at 
the Dockets Management Staff between 9 a.m. and 4 p.m., Monday through 
Friday, 240-402-7500.
     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 https://www.regulations.gov. 
Submit both copies to the Dockets Management Staff. 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

[[Page 58673]]

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 56469, 
September 18, 2015, or access the information at: https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf.
    Docket: For access to the docket to read background documents or 
the electronic and written/paper comments received, go to https://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 Dockets Management Staff, 5630 Fishers Lane, 
Rm. 1061, Rockville, MD 20852, 240-402-7500.
    You may submit comments on any guidance at any time (see 21 CFR 
10.115(g)(5)).
    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 the 
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: Dianne Paraoan, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave, Bldg. 51, Rm. 3326, Silver Spring, MD 20993-0002, 301-
796-3161, [email protected], or Stephen Ripley, Center for 
Biologics Evaluation and Research, Food and Drug Administration, Bldg. 
71, Rm. 7301, Silver Spring, MD 20993-0002, 240-402-7911.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``Data Standards for Drug and Biological Product Submissions 
Containing Real-World Data.'' Under section 745A(a) of the FD&C Act (21 
U.S.C. 379k-1(a)) and the guidance for industry entitled ``Providing 
Regulatory Submissions in Electronic Format--Standardized Study Data'' 
(Study Data Guidance), clinical or nonclinical study data contained in 
new drug applications (NDAs), abbreviated new drug applications 
(ANDAs), certain biologics license applications (BLAs), and certain 
investigational new drug applications (INDs) must be in an electronic 
format that the Agency can process, review, and archive, unless such 
submission is exempt from the electronic submission requirements or if 
FDA has granted a waiver. This guidance clarifies that RWD submitted as 
study data in NDAs, ANDAs, certain BLAs, and certain INDs are subject 
to the requirements in section 745A(a) of the FD&C Act (21 U.S.C. 379k-
1(a)) and the Study Data Guidance. Currently, as stated in the Study 
Data Guidance, the Agency can process, review, and archive electronic 
submissions of clinical and nonclinical study data (including data 
derived from RWD sources) that use the standards specified in the 
Catalog posted to FDA's Study Data Standards Resources web page 
(https://www.fda.gov/industry/fda-resources-data-standards/study-data-standards-resources). Therefore, submissions subject to section 745A(a) 
of the FD&C Act that contain study data derived from RWD sources must 
be in electronic format using the study data standards currently 
supported by FDA as specified in the Catalog. This guidance provides 
recommendations to sponsors for complying with section 745A(a) of the 
FD&C Act when submitting study data derived from RWD sources in an 
applicable regulatory submission using standards specified in the 
Catalog.
    Section 3022 of the 21st Century Cures Act (Cures Act) amended the 
FD&C Act to add section 505F, Utilizing Real World Evidence (21 U.S.C. 
355g). This section requires the establishment of a program to evaluate 
the potential use of RWE to help support the approval of a new 
indication for a drug approved under section 505(c) of the FD&C Act (21 
U.S.C. 355(c)) and to help support or satisfy postapproval study 
requirements. This section also requires that FDA use the program to 
inform guidance for industry on the circumstances under which sponsors 
of drugs may rely on RWE and the appropriate standards and 
methodologies for collection and analysis of RWE submitted to evaluate 
the potential use of RWE for those purposes. Further, under the 
Prescription Drug User Fee Amendments of 2017 (PDUFA VI), FDA committed 
to publishing draft guidance on how RWE can contribute to the 
assessment of safety and effectiveness in regulatory submissions. FDA 
is issuing the draft guidance entitled ``Data Standards for Drug and 
Biological Product Submissions Containing Real-World Data'' as part of 
a series of guidance documents to satisfy the Cures Act mandate and the 
PDUFA VI commitment.
    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 ``Data 
Standards for Drug and Biological Product Submissions Containing Real-
World Data.'' 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. Paperwork Reduction Act of 1995

    While this guidance contains no collection of information, it does 
refer to previously approved FDA collections of information. Therefore, 
clearance by the Office of Management and Budget (OMB) under the 
Paperwork Reduction Act of 1995 (PRA) (44 U.S.C. 3501-3521) is not 
required for this guidance. The previously approved collections of 
information are subject to review by OMB under the PRA. The collections 
of information in 21 CFR part 314 (Applications for FDA Approval to 
Market a New Drug) have been approved under OMB control number 0910-
0001; the collections of information in 21 CFR part 312 
(Investigational New Drug Regulations) have been approved under OMB 
control number 0910-0014; the collections of information in 21 CFR part 
58 (Good Laboratory Practice Regulations for Nonclinical Laboratory 
Studies) have been approved under OMB control number 0910-0119; and the 
collections of information in 21 CFR part 601 (General Licensing 
Provisions: Biologics License Application, Changes to an Approved 
Application, Labeling, Revocation and Suspension) have been approved 
under OMB control number 0910-0338.

III. Electronic Access

    Persons with access to the internet may obtain the draft guidance 
at https://www.fda.gov/regulatory-information/search-fda-guidance-documents, https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances, or https://www.regulations.gov.


[[Page 58674]]


    Dated: October 8, 2021.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2021-23081 Filed 10-21-21; 8:45 am]
BILLING CODE 4164-01-P