Nonalcoholic Steatohepatitis With Compensated Cirrhosis: Developing Drugs for Treatment; Draft Guidance for Industry; Availability, 26685-26687 [2019-11951]
Download as PDF
26685
Federal Register / Vol. 84, No. 110 / Friday, June 7, 2019 / Notices
review the information and assess
potential risk of bias from both
published and unpublished studies and
its impact on the EPC Program’s
findings. This study is being conducted
by AHRQ, pursuant to AHRQ’s statutory
authority to conduct and support
research on health care and on systems
for the delivery of such care, including
activities with respect to the quality,
effectiveness, efficiency,
appropriateness and value of healthcare
services. 42 U.S.C. 299a(a)(1).
Method of Collection
To achieve the goals of this project the
following data collections will be
implemented:
• Online Submission Form
Instrument. This information is
collected for the purposes of providing
supplemental evidence and data for
systematic reviews (SEADS). The
purpose of SEADS requests is not to
collect generalizable data, but to
supplement the published and grey
literature searches EPC investigators are
conducting. The online submission form
(OSF) collects data from respondents on
their name and the information packet.
This happens following notification of
opportunity to submit via email listserv
and/or Federal Register notice as
needed, with one request per topic. For
the purposes of meta-analyses, trial
summary data from missing and
unidentified studies are sought. For the
purposes of constructing evidence tables
and quality ratings (e.g., on public
reporting of cost measures or health
information exchange), data can vary
(e.g., URLs, study designs, and
consumer-mediated exchange forms).
Submitters are informed of the types of
information that would be most helpful
to include in the information packet,
which includes a list of all sponsored
but unpublished studies (both
completed and ongoing), as well as
comment on the completeness of
information provided.
The EPC Program currently uses a
broad-based email announcement via
email listserv and a Federal Register
notice, as needed, to publicize the
opportunity to submit scientific
information about each topic. The
proposed project does not duplicate
other available sources of this
information. Available study registries
and databases may not sufficiently
inform the Program’s research. The EPC
Program does not anticipate more than
15 topics per year with SEADS requests.
Estimated Annual Respondent Burden
Exhibit 1 presents estimates of the
reporting burden hours for the data
collection efforts. Time estimates are
based on pilot testing of materials and
what can reasonably be requested of
respondents. The number of
respondents listed in ‘‘Number of
respondents per SEADS request’’ of
Exhibit 1 reflects a projected 33%
response rate with approximately 1–2
responses per request and assumes
about 15 SEADS requests per year.
EXHIBIT 1—ESTIMATED ANNUALIZED BURDEN HOURS
Form name
Number of
SEADS
requests
Number of
SEADS
request that
receive
response
Number of
responses
per SEADS
request
Annual
number
of SEADS
responses
Hours per
response
Total
burden hours
per annum
Online Submission Form (OSF) ..............
15
5
1.5
7.5
15/60
1.87
EXHIBIT 2—ESTIMATED ANNUALIZED COST BURDEN
Form name
Number of
SEADS
requests
Total
burden hours
per SEADS
Average
hourly
wage rate *
Total
cost burden
OSF ..................................................................................................................
15
1.87
a $61.39
$115.10
* Occupational Employment Statistics, May 2017 National Occupational Employment and Wage Estimates United States, U.S. Department of
Labor, Bureau of Labor Statistics. https://www.bls.gov/oes/current/oes_nat.htm#11-0000.
a Based on the mean wages for Public Relations and Fundraising Managers, 11–2031, the occupational group most likely tasked with completing the OSF.
jbell on DSK3GLQ082PROD with NOTICES
Request for Comments
In accordance with the Paperwork
Reduction Act, comments on AHRQ’s
information collection are requested
with regard to any of the following: (a)
Whether the proposed collection of
information is necessary for the proper
performance of AHRQ’s health care
research and health care information
dissemination functions, including
whether the information will have
practical utility; (b) the accuracy of
AHRQ’s estimate of burden (including
hours and costs) of the proposed
collection(s) of information; (c) ways to
enhance the quality, utility and clarity
of the information to be collected; and
(d) ways to minimize the burden of the
collection of information upon the
VerDate Sep<11>2014
18:13 Jun 06, 2019
Jkt 247001
respondents, including the use of
automated collection techniques or
other forms of information technology.
Comments submitted in response to
this notice will be summarized and
included in the Agency’s subsequent
request for OMB approval of the
proposed information collection. All
comments will become a matter of
public record.
Nonalcoholic Steatohepatitis With
Compensated Cirrhosis: Developing
Drugs for Treatment; Draft Guidance
for Industry; Availability
Food and Drug Administration,
ACTION:
Notice of availability.
The Food and Drug
Administration (FDA or Agency) is
announcing the availability of a draft
guidance for industry entitled
‘‘Nonalcoholic Steatohepatitis with
SUMMARY:
BILLING CODE 4160–90–P
Fmt 4703
[Docket No. FDA–2019–D–1516]
HHS.
[FR Doc. 2019–11949 Filed 6–6–19; 8:45 am]
Frm 00045
Food and Drug Administration
AGENCY:
Virginia L. Mackay-Smith,
Associate Director.
PO 00000
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Sfmt 4703
E:\FR\FM\07JNN1.SGM
07JNN1
26686
Federal Register / Vol. 84, No. 110 / Friday, June 7, 2019 / Notices
Compensated Cirrhosis: Developing
Drugs for Treatment.’’ The purpose of
this draft guidance is to provide the
Agency’s current recommendations
regarding the important components of
a drug development program for
nonalcoholic steatohepatitis (NASH)
with compensated cirrhosis. This draft
guidance focuses on the enrollment
criteria, trial design, efficacy endpoints,
and safety considerations for phase 3
trials.
Submit either electronic or
written comments on the draft guidance
by August 6, 2019 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:
DATES:
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’’).
jbell on DSK3GLQ082PROD with NOTICES
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.’’
VerDate Sep<11>2014
18:13 Jun 06, 2019
Jkt 247001
Instructions: All submissions received
must include the Docket No. FDA–
2019–D–1516 for ‘‘Nonalcoholic
Steatohepatitis with Compensated
Cirrhosis: Developing Drugs for
Treatment.’’ 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.
• 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.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 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,
PO 00000
Frm 00046
Fmt 4703
Sfmt 4703
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:
Frank A. Anania, Center for Drug
Evaluation and Research, Food and
Drug Administration, 10903 New
Hampshire Ave., Bldg. 22, Rm. 5387,
Silver Spring, MD 20993, 240–402–
9725.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of
a draft guidance for industry entitled
‘‘Nonalcoholic Steatohepatitis with
Compensated Cirrhosis: Developing
Drugs for Treatment.’’
This draft guidance focuses on the
design of clinical trials to study patients
who have compensated cirrhosis
secondary to NASH. This draft guidance
is in addition to a draft guidance
published in 2018 which discusses the
Agency’s thinking for the design of
clinical trials for patients who have
NASH but do not have cirrhosis (see the
draft guidance for industry entitled
‘‘Noncirrhotic Nonalcoholic
Steatohepatitis with Liver Fibrosis:
Developing Drugs for Treatment’’
available at https://www.fda.gov/ucm/
groups/fdagov-public/@fdagov-drugsgen/documents/document/
ucm627376.pdf).
NASH is the hepatic manifestation of
insulin resistance syndrome and is
associated with type 2 diabetes,
hypertension, hypertriglyceridemia, and
obesity, among other diseases. NASHrelated cirrhosis is becoming a major
public health problem and is
anticipated to be the leading indication
for orthotopic liver transplantation
within a decade.
This draft guidance applies only to
compensated cirrhosis and specifically
excludes patients with decompensated
cirrhosis, i.e. patients who have already
experienced any of several clinical
events (e.g., variceal bleeding, ascites,
hepatic encephalopathy) that are
associated with high morbidity and
significantly reduce their life
expectancies. This draft guidance
describes the criteria for enrolling
patients with compensated cirrhosis in
clinical trials, including the histologic
criteria to establish the pathological
diagnosis of cirrhosis.
This draft guidance provides
recommendations on the selection of
primary efficacy endpoints in clinical
trials intended to study pharmacological
treatments for compensated NASH
E:\FR\FM\07JNN1.SGM
07JNN1
Federal Register / Vol. 84, No. 110 / Friday, June 7, 2019 / Notices
cirrhosis. Finally, the Agency discusses
the rationale for recommending that
sponsors conduct clinical outcome trials
for drugs treating compensated NASH
cirrhosis. The Agency also provides
recommendations to help ensure safety
in patients with hepatic impairment and
strategies to deal with drug-induced
liver injury during a compensated
NASH cirrhosis clinical trial.
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 ‘‘Nonalcoholic Steatohepatitis with
Compensated Cirrhosis: Developing
Drugs for Treatment.’’ 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.
jbell on DSK3GLQ082PROD with NOTICES
II. Paperwork Reduction Act of 1995
This draft guidance refers to
previously approved collections of
information found in FDA regulations.
These collections of information are
subject to review by the Office of
Management and Budget (OMB) under
the Paperwork Reduction Act of 1995
(44 U.S.C. 3501–3520). The collections
of information under 21 CFR part 312
(Investigational New Drug Application)
have been approved under OMB control
number 0910–0014. The collections of
information in 21 CFR parts 50 and 56
(Protection of Human Subjects:
Documentation of Informed Consent;
Institutional Review Boards) have been
approved under OMB control number
0910–0755. The collection of
information under 21 CFR part 314,
including the submission of information
under subpart H (‘‘Accelerated
Approval of New Drugs for Serious or
Life-Threatening Illnesses’’), has been
approved under OMB control number
0910–0001. The collection of
information under the guidance for
industry entitled ‘‘Expedited Programs
for Serious Conditions—Drugs and
Biologics’’ (available at https://
www.fda.gov/ucm/groups/fdagovpublic/@fdagov-drugs-gen/documents/
document/ucm358301.pdf) has been
approved under OMB control number
0910–0765.
III. Electronic Access
Persons with access to the internet
may obtain the draft guidance at either
https://www.fda.gov/Drugs/Guidance
ComplianceRegulatoryInformation/
Guidances/default.htm or https://
www.regulations.gov.
VerDate Sep<11>2014
18:13 Jun 06, 2019
Jkt 247001
Dated: June 3, 2019.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2019–11951 Filed 6–6–19; 8:45 am]
BILLING CODE 4164–01–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; Draft 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 draft
guidance for industry entitled
‘‘Enhancing the Diversity of Clinical
Trial Populations—Eligibility Criteria,
Enrollment Practices, and Trial
Designs.’’ This draft guidance
recommends approaches that sponsors
of clinical trials to support a new drug
application or a biologics license
application can take to broaden
eligibility criteria, when scientifically
and clinically appropriate, and increase
enrollment of underrepresented
populations in their clinical trials. The
draft guidance reflects FDA policy
encouraging inclusion in clinical trials
of participants representative of the
broad population of patients who will
be exposed to a marketed drug and is
being issued to satisfy the FDA
Reauthorization Act of 2017 (FDARA)
mandate.
SUMMARY:
Submit either electronic or
written comments on the draft guidance
by August 6, 2019 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:
DATES:
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
PO 00000
Frm 00047
Fmt 4703
Sfmt 4703
26687
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.
• 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\07JNN1.SGM
07JNN1
Agencies
[Federal Register Volume 84, Number 110 (Friday, June 7, 2019)]
[Notices]
[Pages 26685-26687]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2019-11951]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2019-D-1516]
Nonalcoholic Steatohepatitis With Compensated Cirrhosis:
Developing Drugs for Treatment; Draft 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 draft guidance for industry entitled
``Nonalcoholic Steatohepatitis with
[[Page 26686]]
Compensated Cirrhosis: Developing Drugs for Treatment.'' The purpose of
this draft guidance is to provide the Agency's current recommendations
regarding the important components of a drug development program for
nonalcoholic steatohepatitis (NASH) with compensated cirrhosis. This
draft guidance focuses on the enrollment criteria, trial design,
efficacy endpoints, and safety considerations for phase 3 trials.
DATES: Submit either electronic or written comments on the draft
guidance by August 6, 2019 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-2019-D-1516 for ``Nonalcoholic Steatohepatitis with Compensated
Cirrhosis: Developing Drugs for Treatment.'' 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.
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.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 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: Frank A. Anania, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 22, Rm. 5387, Silver Spring, MD 20993, 240-402-
9725.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a draft guidance for industry
entitled ``Nonalcoholic Steatohepatitis with Compensated Cirrhosis:
Developing Drugs for Treatment.''
This draft guidance focuses on the design of clinical trials to
study patients who have compensated cirrhosis secondary to NASH. This
draft guidance is in addition to a draft guidance published in 2018
which discusses the Agency's thinking for the design of clinical trials
for patients who have NASH but do not have cirrhosis (see the draft
guidance for industry entitled ``Noncirrhotic Nonalcoholic
Steatohepatitis with Liver Fibrosis: Developing Drugs for Treatment''
available at https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm627376.pdf).
NASH is the hepatic manifestation of insulin resistance syndrome
and is associated with type 2 diabetes, hypertension,
hypertriglyceridemia, and obesity, among other diseases. NASH-related
cirrhosis is becoming a major public health problem and is anticipated
to be the leading indication for orthotopic liver transplantation
within a decade.
This draft guidance applies only to compensated cirrhosis and
specifically excludes patients with decompensated cirrhosis, i.e.
patients who have already experienced any of several clinical events
(e.g., variceal bleeding, ascites, hepatic encephalopathy) that are
associated with high morbidity and significantly reduce their life
expectancies. This draft guidance describes the criteria for enrolling
patients with compensated cirrhosis in clinical trials, including the
histologic criteria to establish the pathological diagnosis of
cirrhosis.
This draft guidance provides recommendations on the selection of
primary efficacy endpoints in clinical trials intended to study
pharmacological treatments for compensated NASH
[[Page 26687]]
cirrhosis. Finally, the Agency discusses the rationale for recommending
that sponsors conduct clinical outcome trials for drugs treating
compensated NASH cirrhosis. The Agency also provides recommendations to
help ensure safety in patients with hepatic impairment and strategies
to deal with drug-induced liver injury during a compensated NASH
cirrhosis clinical trial.
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 ``Nonalcoholic
Steatohepatitis with Compensated Cirrhosis: Developing Drugs for
Treatment.'' 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. Paperwork Reduction Act of 1995
This draft guidance refers to previously approved collections of
information found in FDA regulations. These collections of information
are subject to review by the Office of Management and Budget (OMB)
under the Paperwork Reduction Act of 1995 (44 U.S.C. 3501-3520). The
collections of information under 21 CFR part 312 (Investigational New
Drug Application) have been approved under OMB control number 0910-
0014. The collections of information in 21 CFR parts 50 and 56
(Protection of Human Subjects: Documentation of Informed Consent;
Institutional Review Boards) have been approved under OMB control
number 0910-0755. The collection of information under 21 CFR part 314,
including the submission of information under subpart H (``Accelerated
Approval of New Drugs for Serious or Life-Threatening Illnesses''), has
been approved under OMB control number 0910-0001. The collection of
information under the guidance for industry entitled ``Expedited
Programs for Serious Conditions--Drugs and Biologics'' (available at
https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm358301.pdf) has been approved under OMB control
number 0910-0765.
III. Electronic Access
Persons with access to the internet may obtain the draft guidance
at either https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm or
https://www.regulations.gov.
Dated: June 3, 2019.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2019-11951 Filed 6-6-19; 8:45 am]
BILLING CODE 4164-01-P