E17 General Principles for Planning and Design of Multiregional Clinical Trials; International Council for Harmonisation; Guidance for Industry; Availability, 34139-34140 [2018-15395]
Download as PDF
<![CDATA[
Federal Register / Vol. 83, No. 139 / Thursday, July 19, 2018 / Notices
‘‘Drugs@FDA’’ website at https://
www.accessdata.fda.gov/scripts/cder/
daf/.
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2017–N–0809]
Issuance of Priority Review Voucher;
Rare Pediatric Disease Product
AGENCY:
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Notice.
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 EPIDIOLEX
(cannabidiol oral solution)
manufactured by GW Research Ltd.,
meets the criteria for a priority review
voucher.
FOR FURTHER INFORMATION CONTACT:
Althea Cuff, Center for Drug Evaluation
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 EPIDIOLEX
(cannabidiol oral solution)
manufactured by GW Research Ltd.,
meets the criteria for a priority review
voucher. EPIDIOLEX (cannabidiol oral
solution) is indicated for the treatment
of seizures associated with Dravet
Syndrome or Lennox-Gastaut Syndrome
in patients 2 years of age and older.
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/
DevelopingProductsforRareDiseases
Conditions/RarePediatricDiseasePriority
VoucherProgram/default.htm. For
further information about EPIDIOLEX
(cannabidiol oral solution), go to the
SUMMARY:
sradovich on DSK3GMQ082PROD with NOTICES
[FR Doc. 2018–15393 Filed 7–18–18; 8:45 am]
Food and Drug Administration,
HHS.
ACTION:
Dated: July 13, 2018.
Leslie Kux,
Associate Commissioner for Policy.
VerDate Sep<11>2014
17:34 Jul 18, 2018
Jkt 244001
Food and Drug Administration
[Docket No. FDA–2016–D–2567]
E17 General Principles for Planning
and Design of Multiregional Clinical
Trials; International Council for
Harmonisation; 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 entitled ‘‘E17 General
Principles for Planning and Design of
Multiregional Clinical Trials.’’ The
guidance was prepared under the
auspices of the International Council for
Harmonisation (ICH), formerly the
International Conference on
Harmonisation. The guidance describes
general principles for planning and
designing multiregional clinical trials
(MRCTs). The guidance is intended to
increase the acceptability of data from
MRCTs as the primary source of
evidence supporting marketing approval
in global regulatory submissions and
thereby facilitate more efficient drug
development and earlier access to
medicines.
DATES: The announcement of the
guidance is published in the Federal
Register on July 19, 2018.
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
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
PO 00000
Frm 00042
Fmt 4703
Sfmt 4703
34139
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–
2016–D–2567 for ‘‘E17 General
Principles for Planning and Design of
Multiregional Clinical Trials;
International Council for
Harmonisation.’’ 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 office
between 9 a.m. and 4 p.m., Monday
through Friday.
• 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
E:\FR\FM\19JYN1.SGM
19JYN1
34140
Federal Register / Vol. 83, No. 139 / Thursday, July 19, 2018 / Notices
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.gpo.gov/
fdsys/pkg/FR-2015-09-18/pdf/201523389.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.
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
(CBER), Food and Drug Administration,
10903 New Hampshire Ave., Bldg. 71,
Rm. 3128, Silver Spring, MD 20993–
0002. Send one self-addressed adhesive
label to assist that office in processing
your requests. The guidance may also be
obtained by mail by calling CBER at 1–
800–835–4709 or 240–402–8010. See
the SUPPLEMENTARY INFORMATION section
for electronic access to the guidance
document.
sradovich on DSK3GMQ082PROD with NOTICES
FOR FURTHER INFORMATION CONTACT:
Regarding the guidance: Aloka
Chakravarty, Center for Drug Evaluation
and Research, Food and Drug
Administration, 10903 New Hampshire
Ave, Bldg. 21, Rm. 3514, Silver Spring,
MD 20993–0002, 301–796–1655; or R.
Douglas Pratt, Center for Biologics
Evaluation and Research, Food and
Drug Administration, 10903 New
Hampshire Ave., Bldg. 71, Rm. G112,
Silver Spring, MD 20993–0002, 301–
796–2640.
Regarding the ICH: Amanda Roache,
Center for Drug Evaluation and
Research, Food and Drug
Administration, 10903 New Hampshire
VerDate Sep<11>2014
17:34 Jul 18, 2018
Jkt 244001
Ave., Bldg. 51, Rm. 1176, Silver Spring,
MD 20993–0002, 301–796–4548.
SUPPLEMENTARY INFORMATION:
I. Background
In recent years, regulatory authorities
and industry associations from around
the world have participated in many
important initiatives to promote
international harmonization of
regulatory requirements under the ICH.
FDA has participated in several ICH
meetings designed to enhance
harmonization and FDA is committed to
seeking scientifically based harmonized
technical procedures for pharmaceutical
development. One of the goals of
harmonization is to identify and reduce
differences in technical requirements for
drug development among regulatory
agencies.
ICH was established to provide an
opportunity for harmonization
initiatives to be developed with input
from both regulatory and industry
representatives. FDA also seeks input
from consumer representatives and
others. ICH is concerned with
harmonization of technical
requirements for the registration of
pharmaceutical products for human use
among regulators around the world. The
six founding members of the ICH are the
European Commission; the European
Federation of Pharmaceutical Industries
Associations; the FDA; the Japanese
Ministry of Health, Labour, and Welfare;
the Japanese Pharmaceutical
Manufacturers Association; and the
Pharmaceutical Research and
Manufacturers of America. The
Standing Members of the ICH
Association include Health Canada and
Swissmedic. Any party eligible as a
Member in accordance with the ICH
Articles of Association can apply for
membership in writing to the ICH
Secretariat. The ICH Secretariat, which
coordinates the preparation of
documentation, operates as an
international nonprofit organization and
is funded by the Members of the ICH
Association.
The ICH Assembly is the overarching
body of the Association and includes
representatives from each of the ICH
members and observers. The Assembly
is responsible for the endorsement of
draft guidelines and adoption of final
guidelines. FDA publishes ICH
guidelines as FDA guidance.
In the Federal Register of September
9, 2016 (81 FR 62506), FDA published
a notice announcing the availability of
a draft guidance entitled ‘‘E17 General
Principles for Planning and Design of
Multi-Regional Clinical Trials.’’ The
notice gave interested persons an
PO 00000
Frm 00043
Fmt 4703
Sfmt 9990
opportunity to submit comments by
November 8, 2016.
After consideration of the comments
received and revisions to the guideline,
a final draft of the guideline was
submitted to the ICH Assembly and
endorsed by the regulatory agencies in
November 2017.
The guidance provides guidance on
general principles for planning and
designing MRCTs. MRCTs conducted
according to the guidance will
investigate treatment effects in overall
populations with multiple ethnic factors
(intrinsic and extrinsic factors as
described in Appendix A of the ICH
guidance entitled ‘‘E5 Ethnic Factors in
the Acceptability of Foreign Clinical
Data’’) and evaluate the consistency of
treatment effects across populations.
The guidance explicitly states that
MRCTs are planned under the
assumption that the treatment effect
applies to the entire target population,
particularly to the regions included in
the trial. The concept of ‘‘consistency of
treatment effect’’ across regions is
defined in the text and in the glossary,
and the terms ‘‘pooled populations’’ and
‘‘pooled regions’’ are also added to the
glossary. The guidance further clarifies
that prespecified strategies for pooling
regions and/or subpopulations provide
flexibility in sample-size allocation to
regions, and that the strategies facilitate
the assessment of consistency in
treatment effects across regions.
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 ‘‘E17 General
Principles for Planning and Design of
Multiregional Clinical Trials.’’ 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. This
guidance is not subject to Executive
Order 12866.
II. Electronic Access
Persons with access to the internet
may obtain the document at https://
www.regulations.gov, https://
www.fda.gov/Drugs/Guidance
ComplianceRegulatoryInformation/
Guidances/default.htm, or https://
www.fda.gov/BiologicsBloodVaccines/
GuidanceComplianceRegulatory
Information/Guidances/default.htm.
Dated: July 13, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018–15395 Filed 7–18–18; 8:45 am]
BILLING CODE 4164–01–P
E:\FR\FM\19JYN1.SGM
19JYN1
]]>Agencies
[Federal Register Volume 83, Number 139 (Thursday, July 19, 2018)]
[Notices]
[Pages 34139-34140]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2018-15395]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2016-D-2567]
E17 General Principles for Planning and Design of Multiregional
Clinical Trials; International Council for Harmonisation; 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 entitled ``E17 General Principles
for Planning and Design of Multiregional Clinical Trials.'' The
guidance was prepared under the auspices of the International Council
for Harmonisation (ICH), formerly the International Conference on
Harmonisation. The guidance describes general principles for planning
and designing multiregional clinical trials (MRCTs). The guidance is
intended to increase the acceptability of data from MRCTs as the
primary source of evidence supporting marketing approval in global
regulatory submissions and thereby facilitate more efficient drug
development and earlier access to medicines.
DATES: The announcement of the guidance is published in the Federal
Register on July 19, 2018.
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-2016-D-2567 for ``E17 General Principles for Planning and Design of
Multiregional Clinical Trials; International Council for
Harmonisation.'' 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 office between 9 a.m. and 4 p.m., Monday through Friday.
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
[[Page 34140]]
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.gpo.gov/fdsys/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.
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 (CBER), Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 71, Rm. 3128, Silver Spring, MD 20993-0002. Send
one self-addressed adhesive label to assist that office in processing
your requests. The guidance may also be obtained by mail by calling
CBER at 1-800-835-4709 or 240-402-8010. See the SUPPLEMENTARY
INFORMATION section for electronic access to the guidance document.
FOR FURTHER INFORMATION CONTACT:
Regarding the guidance: Aloka Chakravarty, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave, Bldg. 21, Rm. 3514, Silver Spring, MD 20993-0002, 301-
796-1655; or R. Douglas Pratt, Center for Biologics Evaluation and
Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg.
71, Rm. G112, Silver Spring, MD 20993-0002, 301-796-2640.
Regarding the ICH: Amanda Roache, Center for Drug Evaluation and
Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg.
51, Rm. 1176, Silver Spring, MD 20993-0002, 301-796-4548.
SUPPLEMENTARY INFORMATION:
I. Background
In recent years, regulatory authorities and industry associations
from around the world have participated in many important initiatives
to promote international harmonization of regulatory requirements under
the ICH. FDA has participated in several ICH meetings designed to
enhance harmonization and FDA is committed to seeking scientifically
based harmonized technical procedures for pharmaceutical development.
One of the goals of harmonization is to identify and reduce differences
in technical requirements for drug development among regulatory
agencies.
ICH was established to provide an opportunity for harmonization
initiatives to be developed with input from both regulatory and
industry representatives. FDA also seeks input from consumer
representatives and others. ICH is concerned with harmonization of
technical requirements for the registration of pharmaceutical products
for human use among regulators around the world. The six founding
members of the ICH are the European Commission; the European Federation
of Pharmaceutical Industries Associations; the FDA; the Japanese
Ministry of Health, Labour, and Welfare; the Japanese Pharmaceutical
Manufacturers Association; and the Pharmaceutical Research and
Manufacturers of America. The Standing Members of the ICH Association
include Health Canada and Swissmedic. Any party eligible as a Member in
accordance with the ICH Articles of Association can apply for
membership in writing to the ICH Secretariat. The ICH Secretariat,
which coordinates the preparation of documentation, operates as an
international nonprofit organization and is funded by the Members of
the ICH Association.
The ICH Assembly is the overarching body of the Association and
includes representatives from each of the ICH members and observers.
The Assembly is responsible for the endorsement of draft guidelines and
adoption of final guidelines. FDA publishes ICH guidelines as FDA
guidance.
In the Federal Register of September 9, 2016 (81 FR 62506), FDA
published a notice announcing the availability of a draft guidance
entitled ``E17 General Principles for Planning and Design of Multi-
Regional Clinical Trials.'' The notice gave interested persons an
opportunity to submit comments by November 8, 2016.
After consideration of the comments received and revisions to the
guideline, a final draft of the guideline was submitted to the ICH
Assembly and endorsed by the regulatory agencies in November 2017.
The guidance provides guidance on general principles for planning
and designing MRCTs. MRCTs conducted according to the guidance will
investigate treatment effects in overall populations with multiple
ethnic factors (intrinsic and extrinsic factors as described in
Appendix A of the ICH guidance entitled ``E5 Ethnic Factors in the
Acceptability of Foreign Clinical Data'') and evaluate the consistency
of treatment effects across populations. The guidance explicitly states
that MRCTs are planned under the assumption that the treatment effect
applies to the entire target population, particularly to the regions
included in the trial. The concept of ``consistency of treatment
effect'' across regions is defined in the text and in the glossary, and
the terms ``pooled populations'' and ``pooled regions'' are also added
to the glossary. The guidance further clarifies that prespecified
strategies for pooling regions and/or subpopulations provide
flexibility in sample-size allocation to regions, and that the
strategies facilitate the assessment of consistency in treatment
effects across regions.
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 ``E17 General Principles for Planning and
Design of Multiregional Clinical Trials.'' 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. This guidance is not subject to
Executive Order 12866.
II. Electronic Access
Persons with access to the internet may obtain the document at
https://www.regulations.gov, https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, or
https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm.
Dated: July 13, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-15395 Filed 7-18-18; 8:45 am]
BILLING CODE 4164-01-P
