
[Federal Register Volume 88, Number 168 (Thursday, August 31, 2023)]
[Notices]
[Pages 60215-60217]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2023-18841]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2021-D-1214]


Considerations for the Use of Real-World Data and Real-World 
Evidence To Support Regulatory Decision-Making for Drug and Biological 
Products; 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 final guidance for industry entitled 
``Considerations for the Use of Real-World Data and Real-World Evidence 
to Support Regulatory Decision-Making for Drug and Biological 
Products.'' FDA is issuing this guidance as part of its Real-World 
Evidence (RWE) Program for drugs and to satisfy, in part, the mandate 
under the Federal Food, Drug, and Cosmetic Act (FD&C Act) to issue 
guidance about the use of RWE in regulatory decision making. This 
guidance discusses the applicability of FDA's investigational new drug 
application (IND) regulations to various clinical study designs that 
utilize real-world data (RWD) and clarifies the Agency's expectations 
regarding clinical studies using RWD submitted to FDA in support of a 
regulatory decision regarding the effectiveness or safety of a drug 
that are not subject to the IND regulations. This guidance finalizes 
the draft guidance of the same title issued on December 9, 2021.

DATES: The announcement of the guidance is published in the Federal 
Register on August 31, 2023.

ADDRESSES: You may submit either electronic or written comments on 
Agency guidances 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-1214 for ``Considerations for the Use of Real-World Data and 
Real-World Evidence to Support Regulatory Decision-Making for Drug and 
Biological Products.'' 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

[[Page 60216]]

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 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 this 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 to 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 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. 3226, Silver Spring, MD 20993-0002, 301-
796-3161, [email protected]; or Anne Taylor, Center for 
Biologics Evaluation and Research, Food and Drug Administration, 10903 
New Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993-0002, 
240-402-7911, [email protected].

SUPPLEMENTARY INFORMATION: 

I. Background

    FDA is announcing the availability of a guidance for industry 
entitled ``Considerations for the Use of Real-World Data and Real-World 
Evidence to Support Regulatory Decision-Making for Drug and Biological 
Products.'' The guidance discusses the following major topics: (1) 
applicability of 21 CFR part 312 to studies using RWD and (2) 
regulatory considerations for non-interventional (observational) 
studies involving the use of RWD. Regulatory considerations addressed 
by the guidance include the following: (1) transparency for data 
collection and analysis, (2) access to RWD, (3) study monitoring, (4) 
safety reporting, (5) other sponsor responsibilities, and (6) the 
analysis of RWD generated from the use of a product under an emergency 
use authorization (EUA) in routine practice.
    Section 3022 of the 21st Century Cures Act (Cures Act) of 2016 
amended the FD&C Act to add section 505F, Utilizing Real World Evidence 
(21 U.S.C. 355g), which requires FDA to issue guidance about the use of 
RWE in regulatory decision-making. In addition, under the Prescription 
Drug User Fee Amendments of 2017 (PDUFA VI), FDA is committed to 
publish draft guidance on how RWE can contribute to the assessment of 
safety and effectiveness in regulatory submissions. In 2018, FDA 
created an RWE Framework and an RWE Program to evaluate the potential 
use of RWE to help support the approval of a new indication for a drug 
already approved under the FD&C Act or to help support or satisfy 
postapproval study requirements. In late 2021, FDA utilized the RWE 
Program to issue draft guidances outlining considerations for the use 
of RWD and RWE in regulatory decision making to satisfy the Cures Act 
mandate and the PDUFA VI commitment.
    This guidance finalizes the draft guidance of the same title issued 
on December 9, 2021 (86 FR 70131). FDA considered comments received on 
the draft guidance as the guidance was finalized. Changes from the 
draft to the final guidance include transfer of relevant definitions 
from the glossary to the text as well as additional language about data 
generated in clinical practice for products used under an EUA 
consistent with a mandate under the Food and Drug Omnibus Reform Act of 
2022. Clarifying information was also added, including the use of 
existing regulatory pathways for third parties to provide patient-level 
data to FDA when a sponsor cannot submit such data to FDA through 
traditional channels. In addition, editorial changes were made to 
improve clarity.
    This guidance is being issued consistent with FDA's good guidance 
practices regulation (21 CFR 10.115). The guidance represents the 
current thinking of FDA on ``Considerations for the Use of Real-World 
Data and Real-World Evidence to Support Regulatory Decision-Making for 
Drug and Biological Products.'' 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. The 
previously approved collections of information are subject to review by 
Office of Management and Budget (OMB) under the Paperwork Reduction Act 
of 1995 (PRA) (44 U.S.C. 3501-3521). The collections of information in 
21 CFR part 11 pertaining to electronic records and signatures have 
been approved under OMB control number 0910-0303. The collections of 
information in 21 CFR parts 50 and 56 relating to protection of human 
subjects and Institutional Review Boards have been approved under OMB 
control number 0910-0130. The collections of information in 21 CFR part 
310 relating to postmarketing adverse drug experience reporting have 
been approved under OMB control number 0910-0230. The collections of 
information in 21 CFR part 600 for general records and postmarketing 
adverse experience reporting pertaining to biological products have 
been approved under OMB control number 0910-0308. The collections of 
information in 21 CFR parts 310, 314, and 600 pertaining to adverse 
event and product experience reporting have been approved under OMB 
control number 0910-0291. The collections of information in 21 CFR part 
312 have been approved under OMB control number 0910-0014. The 
collections of information in 21 CFR part 314 have been approved under 
OMB control number 0910-0001. The collections of information in 21 CFR 
part 601 have been approved under OMB control number 0910-0338. The 
collections of information in 21 CFR part 600 have been approved under 
OMB control number 0910-0458.

III. Electronic Access

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


[[Page 60217]]


    Dated: August 28, 2023.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2023-18841 Filed 8-30-23; 8:45 am]
BILLING CODE 4164-01-P


