Pharmacokinetics in Patients With Impaired Renal Function-Study Design, Data Analysis, and Impact on Dosing; Draft Guidance for Industry; Availability, 55303-55304 [2020-19597]
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Federal Register / Vol. 85, No. 173 / Friday, September 4, 2020 / Notices
public conduct during advisory
committee meetings.
Notice of this meeting is given under
the Federal Advisory Committee Act (5
U.S.C. app. 2).
Dated: August 26, 2020.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2020–19562 Filed 9–3–20; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2010–D–0133]
Pharmacokinetics in Patients With
Impaired Renal Function—Study
Design, Data Analysis, and Impact on
Dosing; Draft Guidance for Industry;
Availability
Food and Drug Administration,
Health and Human Services (HHS).
ACTION: Notice of availability.
AGENCY:
The Food and Drug
Administration (FDA or Agency) is
announcing the availability of a draft
guidance for industry entitled
‘‘Pharmacokinetics in Patients with
Impaired Renal Function—Study
Design, Data Analysis, and Impact on
Dosing.’’ In general, drug development
programs should be conducted so that
when products are approved, the
labeling provides appropriate dosing
recommendations for patients with
renal impairment. This draft guidance
revises and replaces the draft guidance
entitled ‘‘Pharmacokinetics in Patients
with Impaired Renal Function—Study
Design, Data Analysis, and Impact on
Dosing and Labeling’’ (March 2010) and
is meant to assist sponsors in the design
and analysis of studies that assess the
influence of impaired renal function on
the pharmacokinetics (PK) and/or
pharmacodynamics of an investigational
drug and how such information can
impact dosing.
DATES: Submit either electronic or
written comments on the draft guidance
by December 3, 2020 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draft guidance before it begins work on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:
jbell on DSKJLSW7X2PROD with NOTICES
SUMMARY:
Electronic Submissions
Submit electronic comments in the
following way:
• Federal eRulemaking Portal:
https://www.regulations.gov. Follow the
instructions for submitting comments.
VerDate Sep<11>2014
16:42 Sep 03, 2020
Jkt 250001
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–
2010–D–0133 for ‘‘Pharmacokinetics in
Patients with Impaired Renal
Function—Study Design, Data Analysis,
and Impact on Dosing.’’ 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
PO 00000
Frm 00052
Fmt 4703
Sfmt 4703
55303
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 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. 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:
Lauren Milligan, Center for Drug
Evaluation and Research, Food and
Drug Administration, 10903 New
Hampshire Ave., Bldg. 51, Rm. 3159,
Silver Spring, MD 20903–0002, 301–
796–5008, or OCP@fda.hhs.gov.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of
a draft guidance for industry entitled
‘‘Pharmacokinetics in Patients with
Impaired Renal Function—Study
Design, Data Analysis, and Impact on
Dosing.’’ Drugs are eliminated from the
body by a variety of mechanisms. Most
drugs are cleared by a combination of
some or all of the following pathways:
Metabolism and transport in the small
intestine, metabolism and transport in
E:\FR\FM\04SEN1.SGM
04SEN1
jbell on DSKJLSW7X2PROD with NOTICES
55304
Federal Register / Vol. 85, No. 173 / Friday, September 4, 2020 / Notices
the liver, and glomerular filtration and
tubular secretion of unchanged drug by
the kidneys (i.e., renal excretion). If a
drug is eliminated primarily through
renal excretion, then impaired renal
function usually alters the drug’s PK to
an extent that the dosage regimen may
need to be changed from that used in
patients with normal renal function. For
most drugs that are likely to be
administered to patients with impaired
renal function, it is important to
characterize PK in subjects with
impaired renal function to provide
appropriate dosing recommendations.
The safety and efficacy of a drug are
generally established for a particular
dosage regimen (or range of dosage
regimens) in late-phase clinical trials
that enroll patients from the target
patient population. Frequently,
however, individuals with advanced
kidney disease are explicitly excluded
from participation in these studies,
hindering the assessment of the effects
of severely impaired kidney function on
the PK of a drug or the patient’s clinical
response. A well-planned drug
development program can enable
prospective dosage adjustment based on
the observed or expected changes in the
PK of a drug due to impaired renal
function prior to initiating phase 2 or
phase 3 trials.
This guidance replaces the 2010
version and provides updated
recommendations on the following
topics:
(1) When a dedicated study of a drug’s
PK in subjects with impaired renal
function is recommended and when it
may not be needed;
(2) The design and conduct of
pharmacokinetic studies in subjects
with impaired renal function;
(3) Considerations for characterizing a
drug’s PK in patients undergoing
intermittent or continuous dialytic
therapies;
(4) The use of pharmacokinetic
information from phase 2 and 3 studies
to inform dosing recommendations for
patients with renal impairment; and
(5) The analysis and reporting of the
results of studies that characterize the
impact of renal impairment and how
these data inform dosing.
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 ‘‘Pharmacokinetics in Patients with
Impaired Renal Function—Study
Design, Data Analysis, and Impact on
Dosing.’’ 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
VerDate Sep<11>2014
16:42 Sep 03, 2020
Jkt 250001
requirements of the applicable statutes
and regulations.
II. Paperwork Reduction Act of 1995
FDA tentatively concludes that this
draft guidance contains no collection 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.
However, this draft guidance refers to
previously approved FDA collections of
information. These collections of
information are subject to review by
OMB under the PRA. The collection of
information in 21 CFR 201.57 has been
approved under OMB control number
0910–0572.
III. Electronic Access
Persons with access to the internet
may obtain the draft guidance at either
https://www.fda.gov/drugs/guidancecompliance-regulatory-information/
guidances-drugs or https://
www.regulations.gov.
Dated: August 31, 2020.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2020–19597 Filed 9–3–20; 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
Food and Drug Administration,
Health and Human Services (HHS).
ACTION: Notice.
AGENCY:
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 (the FD&C Act),
as amended by the Food and Drug
Administration Safety and Innovation
Act (FDASIA), 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 VILTEPSO
(viltolarsen) manufactured by Nippon
Shinyaku Co., Ltd. (NS Pharma Inc.,
U.S. Agent), meets the criteria for a
priority review voucher.
FOR FURTHER INFORMATION CONTACT:
Althea Cuff, Center for Drug Evaluation
SUMMARY:
PO 00000
Frm 00053
Fmt 4703
Sfmt 4703
and Research, Food and Drug
Administration, 10903 New Hampshire
Ave., Silver Spring, MD 20993–0002,
301–796–4061, Fax: 301–796–9856,
email: althea.cuff@fda.hhs.gov.
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), which was
added by FDASIA, FDA will award
priority review vouchers to sponsors of
approved rare pediatric disease product
applications that meet certain criteria.
FDA has determined that VILTEPSO
(viltolarsen) manufactured by Nippon
Shinyaku Co., Ltd. (NS Pharma Inc.,
U.S. Agent), meets the criteria for a
priority review voucher.
VILTEPSO (viltolarsen) is indicated
for the treatment of Duchenne Muscular
Dystrophy in patients amenable to Exon
53 Skipping.
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/ForIndustry/
DevelopingProductsfor
RareDiseasesConditions/
RarePediatricDiseasePriorityVoucher
Program/default.htm. FOR FURTHER
INFORMATION about VILTEPSO
(viltolarsen) go to the ‘‘Drugs@FDA’’
website at https://
www.accessdata.fda.gov/scripts/cder/
daf/.
Dated: August 31, 2020.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2020–19604 Filed 9–3–20; 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 Organ Transplantation
Health Resources and Services
Administration (HRSA), Department of
Health and Human Services (HHS).
ACTION: Notice.
AGENCY:
In accordance with the
Federal Advisory Committee Act
(FACA), HHS is hereby giving notice
that the Advisory Committee on Organ
Transplantation (ACOT) has been
renewed. The effective date of the
renewed charter is August 31, 2020.
FOR FURTHER INFORMATION CONTACT:
Robert Walsh, Designated Federal
Officer, HRSA Division of
SUMMARY:
E:\FR\FM\04SEN1.SGM
04SEN1
]]>Agencies
[Federal Register Volume 85, Number 173 (Friday, September 4, 2020)]
[Notices]
[Pages 55303-55304]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-19597]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2010-D-0133]
Pharmacokinetics in Patients With Impaired Renal Function--Study
Design, Data Analysis, and Impact on Dosing; Draft Guidance for
Industry; Availability
AGENCY: Food and Drug Administration, Health and Human Services (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
``Pharmacokinetics in Patients with Impaired Renal Function--Study
Design, Data Analysis, and Impact on Dosing.'' In general, drug
development programs should be conducted so that when products are
approved, the labeling provides appropriate dosing recommendations for
patients with renal impairment. This draft guidance revises and
replaces the draft guidance entitled ``Pharmacokinetics in Patients
with Impaired Renal Function--Study Design, Data Analysis, and Impact
on Dosing and Labeling'' (March 2010) and is meant to assist sponsors
in the design and analysis of studies that assess the influence of
impaired renal function on the pharmacokinetics (PK) and/or
pharmacodynamics of an investigational drug and how such information
can impact dosing.
DATES: Submit either electronic or written comments on the draft
guidance by December 3, 2020 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-2010-D-0133 for ``Pharmacokinetics in Patients with Impaired Renal
Function--Study Design, Data Analysis, and Impact on Dosing.'' 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. 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: Lauren Milligan, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 51, Rm. 3159, Silver Spring, MD 20903-0002, 301-
796-5008, or [email protected].
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a draft guidance for industry
entitled ``Pharmacokinetics in Patients with Impaired Renal Function--
Study Design, Data Analysis, and Impact on Dosing.'' Drugs are
eliminated from the body by a variety of mechanisms. Most drugs are
cleared by a combination of some or all of the following pathways:
Metabolism and transport in the small intestine, metabolism and
transport in
[[Page 55304]]
the liver, and glomerular filtration and tubular secretion of unchanged
drug by the kidneys (i.e., renal excretion). If a drug is eliminated
primarily through renal excretion, then impaired renal function usually
alters the drug's PK to an extent that the dosage regimen may need to
be changed from that used in patients with normal renal function. For
most drugs that are likely to be administered to patients with impaired
renal function, it is important to characterize PK in subjects with
impaired renal function to provide appropriate dosing recommendations.
The safety and efficacy of a drug are generally established for a
particular dosage regimen (or range of dosage regimens) in late-phase
clinical trials that enroll patients from the target patient
population. Frequently, however, individuals with advanced kidney
disease are explicitly excluded from participation in these studies,
hindering the assessment of the effects of severely impaired kidney
function on the PK of a drug or the patient's clinical response. A
well-planned drug development program can enable prospective dosage
adjustment based on the observed or expected changes in the PK of a
drug due to impaired renal function prior to initiating phase 2 or
phase 3 trials.
This guidance replaces the 2010 version and provides updated
recommendations on the following topics:
(1) When a dedicated study of a drug's PK in subjects with impaired
renal function is recommended and when it may not be needed;
(2) The design and conduct of pharmacokinetic studies in subjects
with impaired renal function;
(3) Considerations for characterizing a drug's PK in patients
undergoing intermittent or continuous dialytic therapies;
(4) The use of pharmacokinetic information from phase 2 and 3
studies to inform dosing recommendations for patients with renal
impairment; and
(5) The analysis and reporting of the results of studies that
characterize the impact of renal impairment and how these data inform
dosing.
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
``Pharmacokinetics in Patients with Impaired Renal Function--Study
Design, Data Analysis, and Impact on Dosing.'' 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
FDA tentatively concludes that this draft guidance contains no
collection 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.
However, this draft guidance refers to previously approved FDA
collections of information. These collections of information are
subject to review by OMB under the PRA. The collection of information
in 21 CFR 201.57 has been approved under OMB control number 0910-0572.
III. Electronic Access
Persons with access to the internet may obtain the draft guidance
at either https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs or https://www.regulations.gov.
Dated: August 31, 2020.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2020-19597 Filed 9-3-20; 8:45 am]
BILLING CODE 4164-01-P
