Real-World Data: Assessing Electronic Health Records and Medical Claims Data To Support Regulatory Decision-Making for Drug and Biological Products; Draft Guidance for Industry; Availability, 54219-54221 [2021-21315]

Download as PDF LOTTER on DSK11XQN23PROD with NOTICES1 Federal Register / Vol. 86, No. 187 / Thursday, September 30, 2021 / Notices wire transfer, check, or online payments. As noted in section II, if a sponsor uses a rare pediatric disease priority review voucher for a human drug application, the sponsor would incur the rare pediatric disease priority review voucher fee in addition to any PDUFA fee that is required for the application. The sponsor would need to follow FDA’s normal procedures for timely payment of the PDUFA fee for the human drug application. Payment must be made in U.S. currency by electronic check, check, bank draft, wire transfer, credit card, or U.S. postal money order payable to the order of the Food and Drug Administration. The preferred payment method is online using electronic check (Automated Clearing House (ACH) also known as eCheck). Secure electronic payments can be submitted using the User Fees Payment Portal at https:// userfees.fda.gov/pay (Note: Only full payments are accepted. No partial payments can be made online). Once you search for your invoice, select ‘‘Pay Now’’ to be redirected to Pay.gov. Note that electronic payment options are based on the balance due. Payment by credit card is available for balances that are less than $25,000. If the balance exceeds this amount, only the ACH option is available. Payments must be made using U.S bank accounts as well as U.S. credit cards. If paying by paper check the invoice number should be included on the check, followed by the words ‘‘Rare Pediatric Disease Priority Review.’’ All paper checks must be in U.S. currency from a U.S. bank made payable and mailed to: Food and Drug Administration, P.O. Box 979107, St. Louis, MO 63197–9000. If checks are sent by a courier that requests a street address, the courier can deliver the checks to: U.S. Bank, Attention: Government Lockbox 979107, 1005 Convention Plaza, St. Louis, MO 63101. (Note: This U.S. Bank address is for courier delivery only. If you have any questions concerning courier delivery, contact the U.S. Bank at 314– 418–4013. This telephone number is only for questions about courier delivery). The FDA post office box number (P.O. Box 979107) must be written on the check. If needed, FDA’s tax identification number is 53– 0196965. If paying by wire transfer, please reference your invoice number when completing your transfer. The originating financial institution may charge a wire transfer fee. If the financial institution charges a wire transfer fee, it is required to add that VerDate Sep<11>2014 18:15 Sep 29, 2021 Jkt 253001 amount to the payment to ensure that the invoice is paid in full. The account information is as follows: U.S. Dept. of the Treasury, TREAS NYC, 33 Liberty St., New York, NY 10045, Account Number: 75060099, Routing Number: 021030004, SWIFT: FRNYUS33. V. Reference The following reference is on display at the Dockets Management Staff (HFA– 305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852, 240–402–7500, and is available for viewing by interested persons between 9 a.m. and 4 p.m., Monday through Friday; it is not available electronically at https:// www.regulations.gov as this reference is copyright protected. FDA has verified the website address, as of the date this document publishes in the Federal Register, but websites are subject to change over time. 1. Ridley, D.B., H.G. Grabowski, and J.L. Moe, ‘‘Developing Drugs for Developing Countries,’’ Health Affairs, vol. 25, no. 2, pp. 313–324, 2006, available at: https:// www.healthaffairs.org/doi/full/10.1377/ hlthaff.25.2.313. Dated: September 27, 2021. Lauren K. Roth, Acting Principal Associate Commissioner for Policy. [FR Doc. 2021–21329 Filed 9–29–21; 8:45 am] BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2020–N–0026] Issuance of Priority Review Voucher; Rare Pediatric Disease Product AGENCY: Food and Drug Administration, HHS. ACTION: The Food and Drug Administration (FDA) is announcing the issuance of a priority review voucher to the sponsor of a rare pediatric disease product application. The Federal Food, Drug, and Cosmetic Act (FD&C Act) authorizes FDA to award priority review vouchers to sponsors of approved rare pediatric disease product applications that meet certain criteria. FDA is required to publish notice of the award of the priority review voucher. FDA has determined that RETHYMIC (allogeneic processed thymus tissue-agdc), manufactured by Enzyvant Therapeutics, GmbH, meets the criteria for a priority review voucher. Frm 00071 Fmt 4703 FOR FURTHER INFORMATION CONTACT: Myrna Hanna, 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: FDA is announcing the issuance of a priority review voucher to the sponsor of an approved rare pediatric disease product application. Under section 529 of the FD&C Act (21 U.S.C. 360ff), FDA will award priority review vouchers to sponsors of approved rare pediatric disease product applications that meet certain criteria. FDA has determined that RETHYMIC (allogeneic processed thymus tissue-agdc), manufactured by Enzyvant Therapeutics, GmbH, meets the criteria for a priority review voucher. RETHYMIC (allogeneic processed thymus tissue-agdc) is indicated for immune reconstitution in pediatric patients with congenital athymia. For further information about the Rare Pediatric Disease Priority Review Voucher Program and for a link to the full text of section 529 of the FD&C Act, go to https://www.fda.gov/industry/ developing-products-rare-diseasesconditions/rare-pediatric-disease-rpddesignation-and-voucher-programs. For further information about RETHYMIC (allogeneic processed thymus tissueagdc), go to the Center for Biologics Evaluation and Research Cellular and Gene Therapy Products website at https://www.fda.gov/vaccines-bloodbiologics/cellular-gene-therapyproducts/approved-cellular-and-genetherapy-products. Dated: September 21, 2021. Lauren K. Roth, Acting Principal Associate Commissioner for Policy. [FR Doc. 2021–21311 Filed 9–29–21; 8:45 am] BILLING CODE 4164–01–P Notice. SUMMARY: PO 00000 54219 Sfmt 4703 DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2020–D–2307] Real-World Data: Assessing Electronic Health Records and Medical Claims Data To Support Regulatory DecisionMaking for Drug and Biological Products; Draft Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS. ACTION: E:\FR\FM\30SEN1.SGM Notice of availability. 30SEN1 54220 Federal Register / Vol. 86, No. 187 / Thursday, September 30, 2021 / Notices The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ‘‘RealWorld Data: Assessing Electronic Health Records and Medical Claims Data to Support Regulatory Decision-Making for Drug and Biological Products.’’ FDA is issuing 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 (FD&C Act) to issue guidance about the use of RWE in regulatory decision making. This draft guidance is intended to provide sponsors, researchers, and other interested stakeholders with considerations when proposing to use electronic health records (EHRs) or medical claims data in clinical studies to support a regulatory decision for effectiveness or safety. DATES: Submit either electronic or written comments on the draft guidance by November 29, 2021 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: LOTTER on DSK11XQN23PROD with NOTICES1 SUMMARY: 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 VerDate Sep<11>2014 18:15 Sep 29, 2021 Jkt 253001 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– 2020–D–2307 for ‘‘Real-World Data: Assessing Electronic Health Records and Medical Claims Data 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-201509-18/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 PO 00000 Frm 00072 Fmt 4703 Sfmt 4703 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 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 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– 796–2500, dianne.paraoan@fda.hhs.gov, 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. SUPPLEMENTARY INFORMATION: I. Background FDA is announcing the availability of a draft guidance for industry entitled ‘‘Real-World Data: Assessing Electronic Health Records and Medical Claims Data to Support Regulatory DecisionMaking for Drug and Biological Products.’’ This guidance discusses the following topics related to the potential use of EHRs and medical claims in clinical studies to support regulatory decisions: Selection of data sources that appropriately address the study question and sufficiently capture study populations, exposure, outcomes of interest, and key covariates; development and validation of definitions for study design elements (e.g., exposure, outcomes, covariates); and data provenance and quality during data accrual, data curation, and data transformation into the final studyspecific dataset. 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 E:\FR\FM\30SEN1.SGM 30SEN1 Federal Register / Vol. 86, No. 187 / Thursday, September 30, 2021 / Notices use of RWE to: (1) Help to 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 (2) help to support or satisfy postapproval study requirements. This section also requires that FDA use 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 the 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 committed to the goal of publishing draft guidance on how RWE can contribute to the assessment of safety and effectiveness in regulatory submissions. FDA is issuing the draft guidance as part of a series of guidance documents to satisfy the Cures Act mandate and the PDUFA VI goal. The RWE Program will cover clinical studies that use realworld data sources, such as information from routine clinical practice, to derive RWE. 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 ‘‘Real-World Data: Assessing Electronic Health Records and Medical Claims Data 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. LOTTER on DSK11XQN23PROD with NOTICES1 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 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 VerDate Sep<11>2014 18:15 Sep 29, 2021 Jkt 253001 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/guidancecompliance-regulatory-information/ guidances-drugs, https://www.fda.gov/ vaccines-blood-biologics/guidancecompliance-regulatory-informationbiologics/biologics-guidances, https:// www.fda.gov/regulatory-information/ search-fda-guidance-documents, or https://www.regulations.gov. Dated: September 27, 2021. Lauren K. Roth, Acting Principal Associate Commissioner for Policy. [FR Doc. 2021–21315 Filed 9–29–21; 8:45 am] BILLING CODE 4164–01–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Health Resources and Services Administration Charter Renewal for the Advisory Committee on Infant and Maternal Mortality (Formerly the Advisory Committee on Infant Mortality) Health Resources and Services Administration (HRSA), Department of Health and Human Services (HHS). ACTION: Notice. AGENCY: In accordance with the Federal Advisory Committee Act, HHS is hereby giving notice that the Advisory Committee on Infant Mortality has been renamed the Advisory Committee on Infant and Maternal Mortality (ACIMM) and has been renewed. DATES: The effective date of the charter renewal is September 30, 2021. FOR FURTHER INFORMATION CONTACT: Vanessa Lee, MPH, Designated Federal Official, HRSA, Maternal and Child Health Bureau, 5600 Fishers Lane, 18N84, Rockville, Maryland 20857; (301) 443–0543; or VLee1@hrsa.gov. SUPPLEMENTARY INFORMATION: ACIMM is authorized by section 222 of the Public Health Service Act (42 U.S.C. 217a), as amended. The Committee is governed by provisions of Public Law 92–463, as amended, (5 U.S.C. App. 2), which sets forth standards for the formation and use of Advisory Committees. ACIMM advises the Secretary of HHS on department activities, partnerships, policies, and programs directed at reducing infant mortality, maternal mortality and severe maternal morbidity, and improving the health status of infants and women before, SUMMARY: PO 00000 Frm 00073 Fmt 4703 Sfmt 4703 54221 during, and after pregnancy. The Committee provides advice on how best to coordinate federal, state, local, tribal, and territorial governmental efforts designed to improve infant mortality, related adverse birth outcomes, and maternal health, as well as influence similar efforts in the private and voluntary sectors. ACIMM provides guidance and recommendations on the policies, programs, and resources required to address the disparities and inequities in infant mortality, related adverse birth outcomes and maternal health outcomes, including maternal mortality and severe maternal morbidity. With its focus on underlying causes of the disparities and inequities seen in birth outcomes for women and infants, the Committee advises the Secretary on the health, social, economic, and environmental factors contributing to the inequities and proposes structural, policy, and/or systems level changes. The charter renewal and name change for ACIMM was approved on September 30, 2021, which will also stand as the filing date. Renewal of the ACIMM charter gives authorization for the ACIMM committee to operate until September 30, 2023. A copy of the ACIMM charter is available on the ACIMM website at https://www.hrsa.gov/advisorycommittees/infant-mortality/. A copy of the charter also can be obtained by accessing the FACA database that is maintained by the Committee Management Secretariat under the General Services Administration. The website address for the FACA database is https:// www.facadatabase.gov/. Maria G. Button, Director, Executive Secretariat. [FR Doc. 2021–21277 Filed 9–29–21; 8:45 am] BILLING CODE 4165–15–P DEPARTMENT OF HEALTH AND HUMAN SERVICES Health Resources and Services Administration Agency Information Collection Activities: Proposed Collection: Public Comment Request Information Collection Request Title: The HRSA Community-Based Outreach Reporting Module, OMB #0906–0064, Revision Health Resources and Services Administration (HRSA), Department of Health and Human Services. AGENCY: ACTION: E:\FR\FM\30SEN1.SGM Notice. 30SEN1

Agencies

[Federal Register Volume 86, Number 187 (Thursday, September 30, 2021)]
[Notices]
[Pages 54219-54221]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2021-21315]


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

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2020-D-2307]


Real-World Data: Assessing Electronic Health Records and Medical 
Claims Data 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.

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

[[Page 54220]]

SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing 
the availability of a draft guidance for industry entitled ``Real-World 
Data: Assessing Electronic Health Records and Medical Claims Data to 
Support Regulatory Decision-Making for Drug and Biological Products.'' 
FDA is issuing 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 (FD&C 
Act) to issue guidance about the use of RWE in regulatory decision 
making. This draft guidance is intended to provide sponsors, 
researchers, and other interested stakeholders with considerations when 
proposing to use electronic health records (EHRs) or medical claims 
data in clinical studies to support a regulatory decision for 
effectiveness or safety.

DATES: Submit either electronic or written comments on the draft 
guidance by November 29, 2021 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-2020-D-2307 for ``Real-World Data: Assessing Electronic Health 
Records and Medical Claims Data 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 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 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-
796-2500, [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.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a draft guidance for industry 
entitled ``Real-World Data: Assessing Electronic Health Records and 
Medical Claims Data to Support Regulatory Decision-Making for Drug and 
Biological Products.'' This guidance discusses the following topics 
related to the potential use of EHRs and medical claims in clinical 
studies to support regulatory decisions: Selection of data sources that 
appropriately address the study question and sufficiently capture study 
populations, exposure, outcomes of interest, and key covariates; 
development and validation of definitions for study design elements 
(e.g., exposure, outcomes, covariates); and data provenance and quality 
during data accrual, data curation, and data transformation into the 
final study-specific dataset.
    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

[[Page 54221]]

use of RWE to: (1) Help to 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 (2) help to support or satisfy postapproval study 
requirements. This section also requires that FDA use 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 the 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 
committed to the goal of publishing draft guidance on how RWE can 
contribute to the assessment of safety and effectiveness in regulatory 
submissions.
    FDA is issuing the draft guidance as part of a series of guidance 
documents to satisfy the Cures Act mandate and the PDUFA VI goal. The 
RWE Program will cover clinical studies that use real-world data 
sources, such as information from routine clinical practice, to derive 
RWE.
    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 ``Real-World 
Data: Assessing Electronic Health Records and Medical Claims Data 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 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 27, 2021.
Lauren K. Roth,
Acting Principal Associate Commissioner for Policy.
[FR Doc. 2021-21315 Filed 9-29-21; 8:45 am]
BILLING CODE 4164-01-P

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