[Federal Register Volume 89, Number 181 (Wednesday, September 18, 2024)]
[Notices]
[Pages 76482-76484]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2024-21077]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2024-D-2052]


Integrating Randomized Controlled Trials for Drug and Biological 
Products Into Routine Clinical Practice; Draft Guidance for Industry; 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the availability of a draft guidance for industry entitled 
``Integrating Randomized Controlled Trials for Drug and Biological 
Products Into Routine Clinical Practice.'' FDA is publishing this draft 
guidance as part of a series of guidance documents under its Real-World 
Evidence (RWE) Program and to satisfy, in part, a mandate under the 
Federal Food, Drug, and Cosmetic Act to issue guidance about the use of 
RWE in regulatory decision-making. This draft guidance is intended to 
support the conduct of randomized controlled drug trials with 
streamlined protocols and procedures that can integrate research into 
routine clinical practice.

DATES: Submit either electronic or written comments on the draft 
guidance by December 17, 2024 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 
version of the guidance.

[[Page 76483]]


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-2024-D-2052 for ``Integrating Randomized Controlled Trials for Drug 
and Biological Products Into Routine Clinical Practice.'' 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 INFORMATION section for electronic 
access to the draft guidance document.

FOR FURTHER INFORMATION CONTACT: Heather Stone, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, Rm. 3348, Silver Spring, MD 20993-0002, 301-
796-2274, [email protected]; or James Myers, 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.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``Integrating Randomized Controlled Trials for Drug and 
Biological Products Into Routine Clinical Practice.'' This draft 
guidance is intended to support the conduct of randomized controlled 
drug trials with streamlined protocols and procedures that focus on 
essential data, allowing integration of research into routine clinical 
practice. Depending on the condition and the intervention to be 
studied, the spectrum of trial designs may range from those that are 
almost completely reliant on data acquired by the participant's local 
healthcare providers during routine clinical practice visits to those 
that require significant supplementation with dedicated, research-
specific activities for data collection conducted by trial staff.
    Traditional randomized controlled drug trials typically capture a 
large amount of protocol-specified patient information (e.g., patient 
characteristics, medical history, concomitant medications, vital signs, 
adverse events, laboratory results, measures of drug response, clinical 
status) at baseline and over the course of the trial. Some of these 
data are also collected in routine clinical practice, although the 
specific procedures and methods, timing of collection, and 
documentation formats typically differ from those in a clinical trial. 
Given the potential overlap in information collected, data for clinical 
research can, under appropriate circumstances, be obtained from routine 
clinical practice interactions, reducing the need for dedicated trial 
sites.
    This draft guidance applies to studies involving FDA-approved drugs 
being studied for new indications, populations, or doses; drug safety 
studies for FDA-approved drugs; other postmarketing studies; 
comparative effectiveness studies; and trials of unapproved drugs in 
later development when the safety profile is sufficiently characterized 
and the drug is appropriate to be administered and managed in the 
setting of routine clinical practice. This guidance does not address 
non-interventional (observational) studies.
    Among other things, the draft guidance provides considerations

[[Page 76484]]

regarding the roles and responsibilities of sponsors, investigators, 
healthcare institutions, and local healthcare providers and addresses 
using a quality by design approach to facilitate the conduct of trials 
in the clinical practice setting.
    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 ``Integrating 
Randomized Controlled Trials for Drug and Biological Products Into 
Routine Clinical Practice.'' 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 
the 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 parts 50 and 56 have been approved under OMB control number 
0910-0130; 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; and the collections of information in 21 CFR part 601 
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/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: September 12, 2024.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2024-21077 Filed 9-17-24; 8:45 am]
BILLING CODE 4164-01-P