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Studies That Utilize Real-World Data for Safety Assessment of Medicines; International Council for Harmonisation; Draft Guidance for Industry; Availability, 55613-55615 [2024-14717]

Download as PDF Federal Register / Vol. 89, No. 129 / Friday, July 5, 2024 / Notices In its application for patent extension, this applicant seeks 766 or 825 days of patent term extension. III. Petitions Anyone with knowledge that any of the dates as published are incorrect may submit either electronic or written comments and, under 21 CFR 60.24, ask for a redetermination (see DATES). Furthermore, as specified in § 60.30 (21 CFR 60.30), any interested person may petition FDA for a determination regarding whether the applicant for extension acted with due diligence during the regulatory review period. To meet its burden, the petition must comply with all the requirements of § 60.30, including but not limited to: must be timely (see DATES), must be filed in accordance with § 10.20, must contain sufficient facts to merit an FDA investigation, and must certify that a true and complete copy of the petition has been served upon the patent applicant. (See H. Rept. 857, part 1, 98th Cong., 2d sess., pp. 41–42, 1984.) Petitions should be in the format specified in 21 CFR 10.30. Submit petitions electronically to https://www.regulations.gov at Docket Nos. FDA–2013–S–0610. Submit written petitions (two copies are required) to the Dockets Management Staff (HFA–305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852. Dated: July 1, 2024. Lauren K. Roth, Associate Commissioner for Policy. [FR Doc. 2024–14729 Filed 7–3–24; 8:45 am] BILLING CODE 4164–01–P 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Studies That Utilize Real-World Data for Safety Assessment of Medicines; International Council for Harmonisation; Draft Guidance for Industry; Availability ddrumheller on DSK120RN23PROD with NOTICES1 AGENCY: Food and Drug Administration, HHS. ACTION: Notice of availability. The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ‘‘M14 General Principles on Plan, Design, and Analysis of Pharmacoepidemiological SUMMARY: VerDate Sep<11>2014 17:16 Jul 03, 2024 Jkt 262001 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. 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. DATES: You may submit comments on any guidance at any time as follows: ADDRESSES: 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’’). PO 00000 Frm 00066 Fmt 4703 Sfmt 4703 55613 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–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 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-201509-18/pdf/2015-23389.pdf. Docket: For access to the docket to read background documents or the electronic and written/paper comments E:\FR\FM\05JYN1.SGM 05JYN1 55614 Federal Register / Vol. 89, No. 129 / Friday, July 5, 2024 / Notices ddrumheller on DSK120RN23PROD with NOTICES1 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 (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, OSE.PMKTREGS@fda.hhs.gov; 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, Jill.Adleberg@fda.hhs.gov. 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. VerDate Sep<11>2014 17:16 Jul 03, 2024 Jkt 262001 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 https://www.ich.org/). ICH works by engaging global regulatory and industry experts in a detailed, science-based, and consensusdriven 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 PO 00000 Frm 00067 Fmt 4703 Sfmt 4703 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 §§ 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. E:\FR\FM\05JYN1.SGM 05JYN1 Federal Register / Vol. 89, No. 129 / Friday, July 5, 2024 / Notices III. Electronic Access Persons with access to the internet may obtain the draft guidance at https:// www.regulations.gov, https:// www.fda.gov/drugs/guidancecompliance-regulatory-information/ guidances-drugs, https://www.fda.gov/ vaccines-blood-biologics/guidancecompliance-regulatory-informationbiologics/biologics-guidances, or https:// www.fda.gov/regulatory-information/ search-fda-guidance-documents. 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 DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2023–N–0249 ] Authorization of Emergency Use of an In Vitro Diagnostic Device in Response to an Outbreak of Mpox; Availability AGENCY: Food and Drug Administration, HHS. ACTION: Notice. The Food and Drug Administration (FDA) is announcing the issuance of an Emergency Use Authorization (EUA) (the Authorization) under the Federal Food, Drug, and Cosmetic Act (FD&C Act) in response to an outbreak of Mpox. FDA has issued an Authorization for an in vitro diagnostic device, Non-variola Orthopoxvirus Realtime PCR Primer and Probe Set as requested by the Centers for Disease Control and Prevention (CDC). The Authorization contains, among other things, conditions on the emergency use of the authorized product. The Authorization follows the August 9, 2022, determination by the Secretary of Health and Human Services (HHS) that there is a public health emergency, or a significant potential for a public health emergency, that affects, or has a significant potential to affect, national security or the health and security of U.S. citizens living abroad, and that involves monkeypox virus. On the basis of such determination, the Secretary of HHS declared, on September 7, 2022, that circumstances exist justifying the authorization of emergency use of in vitro diagnostics for detection and/or diagnosis of infection with the monkeypox virus, including in vitro diagnostics that detect and/or diagnose infection with non-variola Orthopoxvirus, pursuant to the FD&C ddrumheller on DSK120RN23PROD with NOTICES1 SUMMARY: VerDate Sep<11>2014 17:16 Jul 03, 2024 Jkt 262001 Act, subject to terms of any authorization issued under that section. The Authorization, which includes an explanation of the reasons for issuance, and can be accessed on FDA’s website from the link indicated in Section III. Authorization. DATES: The Authorization is effective as of March 22, 2024. ADDRESSES: Submit written requests for single copies of an EUA to the Office of Policy, Center for Devices and Radiological Health, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 66, Rm. 5431, Silver Spring, MD 20993–0002. Send one selfaddressed adhesive label to assist that office in processing your request or include a fax number to which the Authorization may be sent. See the SUPPLEMENTARY INFORMATION section for electronic access to the Authorization. FOR FURTHER INFORMATION CONTACT: Kim Sapsford-Medintz, Office of Product Evaluation and Quality, Center for Devices and Radiological Health, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 66, Rm. 3216, Silver Spring, MD 20993–0002, 301– 796–0311 (this is not a toll-free number). SUPPLEMENTARY INFORMATION: I. Background Section 564 of the FD&C Act (21 U.S.C. 360bbb–3) allows FDA to strengthen public health protections against biological, chemical, radiological, or nuclear agent or agents. Among other things, section 564 of the FD&C Act allows FDA to authorize the use of an unapproved medical product or an unapproved use of an approved medical product in certain situations. With this EUA authority, FDA can help ensure that medical countermeasures may be used in emergencies to diagnose, treat, or prevent serious or lifethreatening diseases or conditions caused by biological, chemical, radiological, or nuclear agent or agents when there are no adequate, approved, and available alternatives (among other criteria). II. Criteria for EUA Authorization Section 564(b)(1) of the FD&C Act provides that, before an EUA may be issued, the Secretary of HHS must declare that circumstances exist justifying the authorization based on one of the following grounds: (1) a determination by the Secretary of Homeland Security that there is a domestic emergency, or a significant potential for a domestic emergency, involving a heightened risk of attack with a biological, chemical, radiological, PO 00000 Frm 00068 Fmt 4703 Sfmt 4703 55615 or nuclear agent or agents; (2) a determination by the Secretary of Defense that there is a military emergency, or a significant potential for a military emergency, involving a heightened risk to U.S. military forces, including personnel operating under the authority of title 10 or title 50, U.S. Code, of attack with (A) a biological, chemical, radiological, or nuclear agent or agents or (B) an agent or agents that may cause, or are otherwise associated with, an imminently life-threatening and specific risk to U.S. military forces; 1 (3) a determination by the Secretary of HHS that there is a public health emergency, or a significant potential for a public health emergency, that affects, or has a significant potential to affect, national security or the health and security of U.S. citizens living abroad, and that involves a biological, chemical, radiological, or nuclear agent or agents, or a disease or condition that may be attributable to such agent or agents; or (4) the identification of a material threat by the Secretary of Homeland Security pursuant to section 319F–2 of the Public Health Service (PHS) Act (42 U.S.C. 247d–6b) sufficient to affect national security or the health and security of U.S. citizens living abroad. Once the Secretary of HHS has declared that circumstances exist justifying an authorization under section 564 of the FD&C Act, FDA may authorize the emergency use of a drug, device, or biological product if the Agency concludes that the statutory criteria are satisfied. Under section 564(h)(1) of the FD&C Act, FDA is required to publish in the Federal Register a notice of each authorization, and each termination or revocation of an authorization, and an explanation of the reasons for the action. Under section 564(h)(1) of the FD&C Act, revisions to an authorization shall be made available on the internet website of FDA. Section 564 of the FD&C Act permits FDA to authorize the introduction into interstate commerce of a drug, device, or biological product intended for use in an actual or potential emergency when the Secretary of HHS has declared that circumstances exist justifying the authorization of emergency use. Products appropriate for emergency use may include products and uses that are not approved, cleared, or licensed under sections 505, 510(k), 512, or 515 of the FD&C Act (21 U.S.C. 355, 360(k), 360b, 1 In the case of a determination by the Secretary of Defense, the Secretary of HHS shall determine within 45 calendar days of such determination, whether to make a declaration under section 564(b)(1) of the FD&C Act, and, if appropriate, shall promptly make such a declaration. E:\FR\FM\05JYN1.SGM 05JYN1

Agencies

[Federal Register Volume 89, Number 129 (Friday, July 5, 2024)]
[Notices]
[Pages 55613-55615]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2024-14717]


-----------------------------------------------------------------------

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.

-----------------------------------------------------------------------

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:
     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-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 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

[[Page 55614]]

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 (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, 
[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.
    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, 
[email protected].

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 https://www.ich.org/).
    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 https://www.regulations.gov, 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, or https://www.fda.gov/regulatory-information/search-fda-guidance-documents.

    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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