Notice2024-14717
M14 General Principles on Plan, Design, and Analysis of Pharmacoepidemiological Studies That Utilize Real-World Data for Safety Assessment of Medicines; International Council for Harmonisation; Draft Guidance for Industry; Availability
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Published
July 5, 2024
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled "M14 General Principles on Plan, Design, and Analysis of Pharmacoepidemiological Studies That Utilize Real-World Data for Safety Assessment of Medicines." The draft guidance was prepared under the auspices of the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH). The draft guidance outlines general principles on planning, designing, and analyzing observational (noninterventional) pharmacoepidemiological studies that utilize fit-for-purpose data for safety assessment of medicines (drugs, vaccines, and other biological products). The draft guidance includes recommendations and high-level best practices for the conduct of these studies. The draft guidance is intended to streamline the development and regulatory assessment of postmarketing pharmacoepidemiological safety studies that include Real-World Data. This guidance also seeks to improve the ability of the study protocol and/or results to be accepted across health authorities and support decision making in response to study results.
Full Text
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<title>Federal Register, Volume 89 Issue 129 (Friday, July 5, 2024)</title>
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[Federal Register Volume 89, Number 129 (Friday, July 5, 2024)]
[Notices]
[Pages 55613-55615]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2024-14717]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2024-D-2754]
M14 General Principles on Plan, Design, and Analysis of
Pharmacoepidemiological Studies That Utilize Real-World Data for Safety
Assessment of Medicines; International Council for Harmonisation; Draft
Guidance for Industry; Availability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 ``M14
General Principles on Plan, Design, and Analysis of
Pharmacoepidemiological Studies That Utilize Real-World Data for Safety
Assessment of Medicines.'' The draft guidance was prepared under the
auspices of the International Council for Harmonisation of Technical
Requirements for Pharmaceuticals for Human Use (ICH). The draft
guidance outlines general principles on planning, designing, and
analyzing observational (noninterventional) pharmacoepidemiological
studies that utilize fit-for-purpose data for safety assessment of
medicines (drugs, vaccines, and other biological products). The draft
guidance includes recommendations and high-level best practices for the
conduct of these studies. The draft guidance is intended to streamline
the development and regulatory assessment of postmarketing
pharmacoepidemiological safety studies that include Real-World Data.
This guidance also seeks to improve the ability of the study protocol
and/or results to be accepted across health authorities and support
decision making in response to study results.
DATES: Submit either electronic or written comments on the draft
guidance by September 3, 2024 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:
<bullet> Federal eRulemaking Portal: <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
Follow the instructions for submitting comments. Comments submitted
electronically, including attachments, to <a href="https://www.regulations.gov">https://www.regulations.gov</a>
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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
<bullet> 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:
<bullet> 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.
<bullet> 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-2754 for ``M14 General Principles on Plan, Design, and
Analysis of Pharmacoepidemiological Studies That Utilize Real-World
Data for Safety Assessment of Medicines.'' Received comments will be
placed in the docket and, except for those submitted as ``Confidential
Submissions,'' publicly viewable at <a href="https://www.regulations.gov">https://www.regulations.gov</a> or at
the Dockets Management Staff between 9 a.m. and 4 p.m., Monday through
Friday, 240-402-7500.
<bullet> 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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
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: <a href="https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf">https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf</a>.
Docket: For access to the docket to read background documents or
the electronic and written/paper comments
[[Page 55614]]
received, go to <a href="https://www.regulations.gov">https://www.regulations.gov</a> 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 (CBER), 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. The guidance may also be obtained by mail by calling
CBER at 1-800-835-4709 or 240-402-8010. See the SUPPLEMENTARY
INFORMATION section for electronic access to the draft guidance
document.
FOR FURTHER INFORMATION CONTACT:
Regarding the guidance: Wei Hua, Center for Drug Evaluation and
Research, Food and Drug Administration, 10903 New Hampshire Ave. Bldg.
22, Rm. 2414, Silver Spring, MD 20993-0002, 240-402-8658,
<a href="/cdn-cgi/l/email-protection#baf5e9ff94eaf7f1eee8fffde9fadcdedb94d2d2c994ddd5cc"><span class="__cf_email__" data-cfemail="0748544229574a4c535542405447616366296f6f7429606871">[email protected]</span></a>; 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.
Regarding the ICH: Jill Adleberg, Center for Drug Evaluation and
Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg.
51, Rm. 6364, Silver Spring, MD 20993-0002, 301-796-5259,
<a href="/cdn-cgi/l/email-protection#d69cbfbabaf897b2bab3b4b3a4b196b0b2b7f8bebea5f8b1b9a0"><span class="__cf_email__" data-cfemail="c983a0a5a5e788ada5acabacbbae89afada8e7a1a1bae7aea6bf">[email protected]</span></a>.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a draft guidance for industry
entitled ``M14 General Principles on Plan, Design, and Analysis of
Pharmacoepidemiological Studies That Utilize Real-World Data for Safety
Assessment of Medicines.'' The draft guidance was prepared under the
auspices of ICH. ICH seeks to achieve greater regulatory harmonization
worldwide to ensure that safe, effective, high-quality medicines are
developed, registered, and maintained in the most resource-efficient
manner.
By harmonizing the regulatory requirements in regions around the
world, ICH guidelines enhance global drug development, improve
manufacturing standards, and increase the availability of medications.
For example, ICH guidelines have substantially reduced duplicative
clinical studies, prevented unnecessary animal studies, standardized
the reporting of important safety information, and standardized
marketing application submissions.
The six Founding Members of the ICH are FDA; the Pharmaceutical
Research and Manufacturers of America; the European Commission; the
European Federation of Pharmaceutical Industries Associations; the
Japanese Ministry of Health, Labour, and Welfare; and the Japanese
Pharmaceutical Manufacturers Association. The Standing Members of the
ICH Association include Health Canada and Swissmedic. ICH membership
continues to expand to include other regulatory authorities and
industry associations from around the world (refer to <a href="https://www.ich.org/">https://www.ich.org/</a>).
ICH works by engaging global regulatory and industry experts in a
detailed, science-based, and consensus-driven process that results in
the development of ICH guidelines. The regulators around the world are
committed to consistently adopting these consensus-based guidelines,
realizing the benefits for patients and for industry.
As a Founding Regulatory Member of ICH, FDA plays a major role in
the development of each of the ICH guidelines, which FDA then adopts
and issues as guidance for industry. FDA's guidance documents do not
establish legally enforceable responsibilities. Instead, they describe
the Agency's current thinking on a topic and should be viewed only as
recommendations, unless specific regulatory or statutory requirements
are cited.
In May 2024, the ICH Assembly endorsed the draft guideline entitled
``M14 General Principles on Plan, Design, and Analysis of
Pharmacoepidemiological Studies That Utilize Real-World Data for Safety
Assessment of Medicines'' and agreed that the guideline should be made
available for public comment. The draft guideline is the product of the
Quality Expert Working Group of the ICH. Comments about this draft will
be considered by FDA and the Quality Expert Working Group.
This draft guidance provides general considerations and
recommendations for use of real-world data for drug, vaccine, and other
biologic product safety assessments. The draft guidance seeks to
harmonize regional recommendations for the design, analysis, and
reporting of postmarketing pharmacoepidemiologic studies that use real-
world data as the number of pharmacoepidemiological studies utilizing
real-world data in a regulatory context have increased globally.
This draft guidance has been left in the original ICH format. The
final guidance will be reformatted and edited to conform with FDA's
good guidance practices regulation (21 CFR 10.115) and style before
publication. The draft guidance, when finalized, will represent the
current thinking of FDA on ``M14 General Principles on Plan, Design,
and Analysis of Pharmacoepidemiologic Studies That Utilize Real-World
Data for Safety Assessment of Medicines.'' 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 (44 U.S.C. 3501-3521). The collections of information
supporting investigational new drug regulations in 21 CFR part 312 have
been approved under OMB control number 0910-0014. The collections of
information supporting FDA approval of new drugs in part 314 (21 CFR
part 314) have been approved under OMB control number 0910-0001. The
collections of information supporting general licensing provisions of
biological products in 21 CFR part 601 have been approved under OMB
control number 0910-0338. The collections of information supporting
adverse experience reporting in 21 CFR 310.305 and 329.100, and
Sec. Sec. 314.80, 314.81, and 314.98, have been approved under OMB
control number 0910-0230. The collections of information supporting
MedWatch safety and adverse event reporting have been approved under
OMB control number 0910-0291. The collections of information supporting
biological products postmarket adverse experience reporting in 21 CFR
part 600 have been approved under OMB control number 0910-0308 and the
collections of information supporting medical device reporting have
been approved under OMB control number 0910-0437.
[[Page 55615]]
III. Electronic Access
Persons with access to the internet may obtain the draft guidance
at <a href="https://www.regulations.gov">https://www.regulations.gov</a>, <a href="https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs">https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs</a>, <a href="https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances">https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances</a>, or <a href="https://www.fda.gov/regulatory-information/search-fda-guidance-documents">https://www.fda.gov/regulatory-information/search-fda-guidance-documents</a>.
Dated: July 1, 2024.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2024-14717 Filed 7-3-24; 8:45 am]
BILLING CODE 4164-01-P
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