[Federal Register Volume 86, Number 234 (Thursday, December 9, 2021)]
[Notices]
[Pages 70131-70132]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2021-26640]


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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Products; Draft Guidance for Industry; Availability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the availability of a draft guidance for industry entitled 
``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 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. FDA created a framework 
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 to support or satisfy postapproval study requirements. 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 
(e.g., as part of a new drug application or a biologics license 
application) that are not subject to the IND regulations.

DATES: Submit either electronic or written comments on the draft 
guidance by March 9, 2022 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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-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 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 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

[[Page 70132]]

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, 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 draft 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 draft guidance discusses the following major topics: 
(1) Applicability of part 312 (21 CFR part 312) to studies using RWD 
and (2) regulatory considerations for non-interventional 
(observational) studies using RWD. Topics covered under regulatory 
considerations include the following: (1) Transparency for data 
collection and analysis, (2) data access, (3) study monitoring, (4) 
safety reporting, and (5) sponsor responsibilities.
    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 utilize 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 is 
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 ``Considerations for the Use of 
Real-World Data and Real-World Evidence to Support Regulatory Decision-
Making for Drug and Biological Products'' 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 
``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. 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 11 have been approved under OMB control 
number 0910-0303. The collections of information in 21 CFR parts 50 and 
56 have been approved under OMB control number 0910-0130. The 
collections of information in 21 CFR part 54 have been approved under 
OMB control number 0910-0396. The collections of information in 21 CFR 
part 310 have been approved under OMB control number 0910-0230. The 
collections of information in 21 CFR parts 310, 314, and 600 have been 
approved under OMB control number 0910-0645. The collections of 
information in 21 CFR parts 310, 314, and 600 have been approved under 
OMB control number 0910-0291. The collections of information in 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. The collections of information in FDA's 
guidance for industry entitled ``Oversight of Clinical Investigations: 
A Risk-Based Approach to Monitoring'' have been approved under OMB 
control number 0910-0733. The collections of information in FDA's 
guidance for industry entitled ``Formal Meetings with Sponsors and 
Applicants for PDUFA Products'' have been approved under OMB control 
number 0910-0429.

III. Electronic Access

    Persons with access to the internet may obtain the draft 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.

    Dated: December 2, 2021.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2021-26640 Filed 12-8-21; 8:45 am]
BILLING CODE 4164-01-P


