Enhancing the Diversity of Clinical Trial Populations-Eligibility Criteria, Enrollment Practices, and Trial Designs; Guidance for Industry; Availability, 71654-71656 [2020-24881]
Download as PDF
<![CDATA[
jbell on DSKJLSW7X2PROD with NOTICES
71654
Federal Register / Vol. 85, No. 218 / Tuesday, November 10, 2020 / Notices
information from the public. Under the
Paperwork Reduction Act of 1995 (the
PRA), Federal agencies are required to
publish notice in the Federal Register
concerning each proposed collection of
information (including each proposed
extension or reinstatement of an existing
collection of information) and to allow
60 days for public comment on the
proposed action. Interested persons are
invited to send comments regarding our
burden estimates or any other aspect of
this collection of information, including
the necessity and utility of the proposed
information collection for the proper
performance of the agency’s functions,
the accuracy of the estimated burden,
ways to enhance the quality, utility, and
clarity of the information to be
collected, and the use of automated
collection techniques or other forms of
information technology to minimize the
information collection burden.
DATES: Comments must be received by
January 11, 2021.
ADDRESSES: When commenting, please
reference the document identifier or
OMB control number. To be assured
consideration, comments and
recommendations must be submitted in
any one of the following ways:
1. Electronically. You may send your
comments electronically to https://
www.regulations.gov. Follow the
instructions for ‘‘Comment or
Submission’’ or ‘‘More Search Options’’
to find the information collection
document(s) that are accepting
comments.
2. By regular mail. You may mail
written comments to the following
address: CMS, Office of Strategic
Operations and Regulatory Affairs,
Division of Regulations Development,
Attention: Document Identifier/OMB
Control Number ll, Room C4–26–05,
7500 Security Boulevard, Baltimore,
Maryland 21244–1850.
To obtain copies of a supporting
statement and any related forms for the
proposed collection(s) summarized in
this notice, you may make your request
using one of following:
1. Access CMS’ website address at
website address at https://www.cms.gov/
Regulations-and-Guidance/Legislation/
PaperworkReductionActof1995/PRAListing.html.
2. Call the Reports Clearance Office at
(410) 786–1326.
FOR FURTHER INFORMATION CONTACT:
William N. Parham at (410) 786–4669.
SUPPLEMENTARY INFORMATION:
Contents
This notice sets out a summary of the
use and burden associated with the
following information collections. More
VerDate Sep<11>2014
17:21 Nov 09, 2020
Jkt 253001
detailed information can be found in
each collection’s supporting statement
and associated materials (see
ADDRESSES).
CMS–2552–10 Hospital and Health
Health Care Complex Cost Report
Under the PRA (44 U.S.C. 3501–
3520), federal agencies must obtain
approval from the Office of Management
and Budget (OMB) for each collection of
information they conduct or sponsor.
The term ‘‘collection of information’’ is
defined in 44 U.S.C. 3502(3) and 5 CFR
1320.3(c) and includes agency requests
or requirements that members of the
public submit reports, keep records, or
provide information to a third party.
Section 3506(c)(2)(A) of the PRA
requires federal agencies to publish a
60-day notice in the Federal Register
concerning each proposed collection of
information, including each proposed
extension or reinstatement of an existing
collection of information, before
submitting the collection to OMB for
approval. To comply with this
requirement, CMS is publishing this
notice.
Information Collection
1. Type of Information Collection
Request: Revision of a currently
approved collection; Title of
Information Collection: Hospital and
Health Health Care Complex Cost
Report; Use: CMS requires the Form
CMS–2552–10 to determine a hospital’s
reasonable cost incurred in furnishing
medical services to Medicare
beneficiaries and calculate the hospital
reimbursement. Hospitals paid under a
prospective payment system (PPS) may
receive reimbursement in addition to
the PPS for hospital-specific
adjustments such as Medicare
reimbursable bad debts,
disproportionate share, uncompensated
care, direct and indirect medical
education costs, and organ acquisition
costs.
CMS uses the Form CMS–2552–10 for
rate setting; payment refinement
activities, including developing a
hospital market basket; and Medicare
Trust Fund projections; and to support
program operations. Additionally, the
Medicare Payment Advisory
Commission (MedPAC) uses the
hospital cost report data to calculate
Medicare margins (a measure of the
relationship between Medicare’s
payments and providers’ Medicare
costs) and analyze data to formulate
Medicare Program recommendations to
Congress.
We welcome comments on our
burden estimates for the information
collection request. Form Number: CMS–
PO 00000
Frm 00052
Fmt 4703
Sfmt 4703
2552–10 (OMB control number: 0938–
0050); Frequency: Occasionally;
Affected Public: Private Sector; Business
or other for-profit and not-for-profit
institutions; Number of Respondents:
6.013; Total Annual Responses: 6,013;
Total Annual Hours: 4,173,022. (For
policy questions regarding this
collection contact Gail Duncan at 410–
786–7278.)
Dated: November 4, 2020.
William N. Parham, III,
Director, Paperwork Reduction Staff, Office
of Strategic Operations and Regulatory
Affairs.
[FR Doc. 2020–24948 Filed 11–9–20; 8:45 am]
BILLING CODE 4120–01–P
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.
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:
SUMMARY:
Electronic Submissions
Submit electronic comments in the
following way:
• Federal eRulemaking Portal:
https://www.regulations.gov. Follow the
E:\FR\FM\10NON1.SGM
10NON1
Federal Register / Vol. 85, No. 218 / Tuesday, November 10, 2020 / Notices
jbell on DSKJLSW7X2PROD with NOTICES
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
VerDate Sep<11>2014
17:21 Nov 09, 2020
Jkt 253001
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
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.
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, Dat.Doan@
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.
FOR FURTHER INFORMATION CONTACT:
SUPPLEMENTARY INFORMATION:
PO 00000
Frm 00053
Fmt 4703
Sfmt 4703
71655
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,
E:\FR\FM\10NON1.SGM
10NON1
jbell on DSKJLSW7X2PROD with NOTICES
71656
Federal Register / Vol. 85, No. 218 / Tuesday, November 10, 2020 / Notices
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 participantrelated 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,
VerDate Sep<11>2014
17:21 Nov 09, 2020
Jkt 253001
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/guidancecompliance-regulatory-information/
guidances-drugs, https://www.fda.gov/
vaccines-blood-biologics/guidancecompliance-regulatory-informationbiologics/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
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2017–D–4792]
Regulatory Considerations for
Microneedling Products; Guidance for
Industry and Food and Drug
Administration Staff; Availability
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice of availability.
The Food and Drug
Administration (FDA or Agency) is
announcing the availability of a final
guidance entitled ‘‘Regulatory
Considerations for Microneedling
Products.’’ This guidance is being
issued to assist industry in
understanding when a microneedling
product is a device as defined in the
Federal Food, Drug, and Cosmetic Act
(FD&C Act). This document also
provides information on the regulatory
SUMMARY:
PO 00000
Frm 00054
Fmt 4703
Sfmt 4703
pathway to market for microneedling
devices for aesthetic use.
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
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–
2017–D–4792 for ‘‘Regulatory
Considerations for Microneedling
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.
E:\FR\FM\10NON1.SGM
10NON1
]]>Agencies
[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]
-----------------------------------------------------------------------
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.
-----------------------------------------------------------------------
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
[[Page 71655]]
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,
[[Page 71656]]
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
