[Federal Register Volume 85, Number 218 (Tuesday, November 10, 2020)]
[Notices]
[Pages 71654-71656]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-24881]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2019-D-1264]


Enhancing the Diversity of Clinical Trial Populations--
Eligibility Criteria, Enrollment Practices, and Trial Designs; 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 final guidance for industry entitled ``Enhancing 
the Diversity of Clinical Trial Populations--Eligibility Criteria, 
Enrollment Practices, and Trial Designs.'' This guidance recommends 
approaches that sponsors of clinical trials intended to support a new 
drug application or a biologics license application can take to 
increase enrollment of underrepresented populations in their clinical 
trials. This guidance is being issued, in part, to satisfy the mandates 
of the FDA Reauthorization Act of 2017 (FDARA). This guidance finalizes 
the draft guidance of the same title issued on June 7, 2019.

DATES: The announcement of the guidance is published in the Federal 
Register on November 10, 2020.

ADDRESSES: You may submit either electronic or written comments on 
Agency guidances at any time as follows:

Electronic Submissions

    Submit electronic comments in the following way:
     Federal eRulemaking Portal: https://www.regulations.gov. 
Follow the

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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-2019-D-1264 for ``Enhancing the Diversity of Clinical Trial 
Populations--Eligibility Criteria, Enrollment Practices, and Trial 
Designs.'' 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 this 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 guidance document.

FOR FURTHER INFORMATION CONTACT: Dat Doan, Center for Drug Evaluation 
and Research, Food and Drug Administration, 10903 New Hampshire Ave., 
Bldg. 51, Rm. 3334, Silver Spring, MD 20993, 240-402-8926, 
[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 guidance for industry 
entitled ``Enhancing the Diversity of Clinical Trial Populations--
Eligibility Criteria, Enrollment Practices, and Trial Designs.'' In 
issuing this guidance, FDA is satisfying the mandates under section 
610(a)(3) of FDARA (Pub. L. 115-52).
    One objective of eligibility criteria is to help protect 
participants by excluding people for whom the risk of an adverse event 
from participation is not likely to be reasonable in relation to any 
potential benefit and the importance of the knowledge that may be 
expected to result. FDA recognizes that certain exclusions are 
appropriate when necessary to help protect these individuals. For 
example, patients with varying degrees of kidney or liver impairment 
are often excluded early in drug development programs because adequate 
information is not available on how to adjust doses for such patients 
or whether these patients could be more vulnerable to certain risks. 
Medically complex patients with certain concomitant illnesses or those 
taking particular drugs may also be excluded from drug development 
programs. As data on excretory and metabolic pathways and drug-drug 
interactions become available during the drug development program, 
allowing appropriate dose adjustments, exclusions related to 
concomitant medications or comorbidities should be narrowed. Similarly, 
as the safety experience with a product increases, eligibility criteria 
should be broadened to include more medically complex participants; any 
remaining exclusions should be justified. This guidance provides 
recommendations on broadening eligibility criteria in clinical trials 
through inclusive trial practices, trial designs, and methodological 
approaches.
    Beyond the limitations in participation imposed by narrow 
eligibility criteria, potential participants may face additional 
challenges to enrolling in clinical trials. A trial requiring 
participants to make frequent visits to specific sites may result in an 
added burden for participants, especially the elderly, children, 
disabled, and cognitively impaired individuals who require 
transportation or caregiver assistance, or participants who live far 
from research facilities, such as those in rural or remote locations. 
Financial costs (e.g., travel, missing work, dependent care) may also 
impede participation, and study visits may interfere with jobs and/or 
family and community obligations. Moreover,

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for individuals under current clinical care on a regularly scheduled 
basis (e.g., individuals with multiple chronic conditions), additional 
clinical trial study visits may be psychologically, physically, and 
financially burdensome and a disincentive for enrollment. This guidance 
provides recommendations on how sponsors can improve the diversity of 
enrolled participants by accounting for logistical and other 
participant-related factors that could limit participation in clinical 
trials.
    Clinical trials of investigational drugs intended to treat rare 
diseases or conditions present a unique set of challenges. Because of 
the limited numbers of patients, maximum participation in clinical 
trials is essential for successful trial completion and interpretation. 
Rare diseases often affect small, geographically dispersed patient 
populations with disease-related travel limitations, so special efforts 
may be necessary to enroll and retain these participants to ensure that 
a broad spectrum of the patient population is represented. This 
guidance provides recommendations on broadening clinical trial 
eligibility criteria for clinical trials of investigational drugs 
intended to treat rare diseases and recommendations on improving the 
enrollment and retention of participants with rare diseases.
    This guidance finalizes the draft guidance of the same title issued 
on June 7, 2019 (84 FR 26687). FDA considered comments received on the 
draft guidance as the guidance was finalized. Changes to the guidance 
include additional recommendations on broadening eligibility criteria, 
such as the use of real-world data to find trial participants and the 
use of mobile medical professionals to visit participants at their 
locations instead of requiring participants to visit distant clinical 
trial sites. FDA added information on the inclusion of racial and 
ethnic minorities, with recommendations included from FDA's draft 
guidance entitled ``Collection of Race and Ethnicity Data in Clinical 
Trials.'' FDA also added recommendations on fostering community 
engagement and making recruitment events more accessible as well as 
information on how to reach participants with little or no internet 
access. In addition, editorial changes were made to improve clarity.
    This guidance is being issued consistent with FDA's good guidance 
practices regulation (21 CFR 10.115). The guidance represents the 
current thinking of FDA on ``Enhancing the Diversity of Clinical Trial 
Populations--Eligibility Criteria, Enrollment Practices, and Trial 
Designs.'' 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 312 have been approved under OMB control 
number 0910-0014.

III. Electronic Access

    Persons with access to the internet may obtain the 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, or 
https://www.regulations.gov.

    Dated: November 4, 2020.
Lauren K. Roth,
Acting Principal Associate Commissioner for Policy.
[FR Doc. 2020-24881 Filed 11-9-20; 8:45 am]
BILLING CODE 4164-01-P