Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment; Guidance for Industry; Availability, 50045-50047 [2019-20629]
Download as PDF
Federal Register / Vol. 84, No. 185 / Tuesday, September 24, 2019 / Notices
khammond on DSKJM1Z7X2PROD with NOTICES
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 to 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:
Sarah Venti, Center for Drug Evaluation
and Research, Food and Drug
Administration, 10903 New Hampshire
Ave., Silver Spring, MD 20993–0002,
301–796–3130, drugtrackandtrace@
fda.hhs.gov.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of
a guidance for industry entitled
‘‘Wholesale Distributor Verification
Requirement for Saleable Returned Drug
Product—Compliance Policy.’’ FDA is
issuing this guidance consistent with
the good guidance practices regulation
(21 CFR 10.115). FDA is implementing
this guidance without prior public
comment because the Agency has
determined that prior public
participation is not feasible or
appropriate (21 CFR 10.115(g)(2)). FDA
made this determination because this
guidance document provides
information pertaining to the statutory
requirement that takes effect November
27, 2019, for wholesale distributors to
verify saleable returned drug products
prior to redistribution under section
582(c)(4)(D) of the Federal Food, Drug,
and Cosmetic Act (FD&C Act) (21 U.S.C.
360eee–1(c)(4)(D)). It is important that
FDA provide this information before
that date. Although this guidance
document is immediately in effect, it
remains subject to comment in
accordance with the Agency’s good
guidance practices (21 CFR
10.115(g)(3)).
The DSCSA (Title II of Pub. L. 113–
54) was signed into law on November
27, 2013. Section 202 of the DSCSA
added section 582 to the FD&C Act. This
section established product tracing,
product identifier, authorized trading
partner, and verification requirements
for manufacturers, wholesale
distributors, repackagers, and
VerDate Sep<11>2014
17:37 Sep 23, 2019
Jkt 247001
dispensers to facilitate the tracing of a
product through the pharmaceutical
distribution supply chain. Failure to
comply with the requirements of section
582 is prohibited under section 301(t) of
the FD&C Act (21 U.S.C. 331(t)) and
subject to enforcement action under the
FD&C Act.
Beginning November 27, 2019,
wholesale distributors are required,
under section 582(c)(4)(D) of the FD&C
Act, to verify the product identifier,
including the standardized numerical
identifier, on each sealed homogeneous
case of saleable returned product, or, if
such product is not in a sealed
homogeneous case, on each package of
saleable returned product, prior to
further distributing such returned
product.
As described in the guidance, FDA
has received comments and feedback
from wholesale distributors as well as
other trading partners and stakeholders
expressing concern with industry-wide
readiness for implementation of the
verification of saleable returned product
requirement for wholesale distributors
and the challenges stakeholders face
with developing interoperable,
electronic systems to enable such
verification and achieve interoperability
between networks. Given the concerns
expressed, FDA recognizes that some
wholesale distributors may need
additional time beyond November 27,
2019, before they can begin verifying the
product identifier on returned products
prior to further distribution in an
efficient, secure, and timely manner.
To minimize possible disruptions in
the distribution of prescription drugs in
the United States, FDA has adopted the
compliance policy described in this
guidance. Under this compliance policy,
FDA does not intend to take action
before November 27, 2020, against
wholesale distributors who do not verify
a product identifier prior to further
distribution of a package or sealed
homogenous case of product as required
by section 582(c)(4)(D) of the FD&C Act.
Additionally, section 582 of the FD&C
Act requires certain trading partners
(manufacturers, repackagers, wholesale
distributors, and dispensers) to
exchange transaction information,
transaction history, and a transaction
statement when engaging in transactions
involving certain prescription drugs.
Section 581(27)(E) of the FD&C Act (21
U.S.C. 360eee(27)(E)) requires that the
transaction statement include a
statement that the entity transferring
ownership in a transaction had systems
and processes in place to comply with
verification requirements under section
582. This guidance also explains that,
prior to November 27, 2020, FDA does
PO 00000
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Fmt 4703
Sfmt 4703
50045
not intend to take action against a
wholesale distributor for providing a
transaction statement to a subsequent
purchaser of product on the basis that
such wholesale distributor does not yet
have systems and processes in place to
comply with the saleable return
verification requirements under section
582(c)(4)(D). The guidance document
explains the scope of the compliance
policy in further detail.
This guidance is being issued
consistent with FDA’s good guidance
practices regulation (21 CFR 10.115).
This guidance represents the current
thinking of FDA on ‘‘Wholesale
Distributor Verification Requirement for
Saleable Returned Drug Product—
Compliance Policy.’’ 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 guidance at either
https://www.fda.gov/Drugs/Guidance
ComplianceRegulatoryInformation/
Guidances/default.htm, https://
www.fda.gov/vaccines-blood-biologics/
guidance-compliance-regulatoryinformation-biologics, or https://
www.regulations.gov.
Dated: September 18, 2019.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2019–20651 Filed 9–23–19; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2013–N–0035]
Amyotrophic Lateral Sclerosis:
Developing Drugs for Treatment;
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
‘‘Amyotrophic Lateral Sclerosis:
Developing Drugs for Treatment.’’ The
guidance provides FDA’s current
thinking on the clinical development
program and clinical trial designs for
drugs to support an indication for the
treatment of amyotrophic lateral
SUMMARY:
E:\FR\FM\24SEN1.SGM
24SEN1
50046
Federal Register / Vol. 84, No. 185 / Tuesday, September 24, 2019 / Notices
sclerosis (ALS). The guidance addresses
the clinical development of drugs
intended to treat the main motor aspects
of ALS, i.e., muscle weakness and its
direct consequences, including
shortened life expectancy. It does not
address in detail the development of
drugs to treat other symptoms that may
arise in ALS, such as muscle cramps,
spasticity, sialorrhea, pseudobulbar
affect, and others. This guidance
finalizes the draft guidance of the same
name issued on February 16, 2018.
DATES: The announcement of the
guidance is published in the Federal
Register on September 24, 2019.
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’’).
khammond on DSKJM1Z7X2PROD 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
17:37 Sep 23, 2019
Jkt 247001
Instructions: All submissions received
must include the Docket No. FDA–
2013–N–0035 for ‘‘Amyotrophic Lateral
Sclerosis: 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 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–
PO 00000
Frm 00068
Fmt 4703
Sfmt 4703
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:
Billy Dunn, Center for Drug Evaluation
and Research, Food and Drug
Administration, 10903 New Hampshire
Ave., Bldg. 22, Rm. 4332, Silver Spring,
MD 20993–0002, 301–796–2250; 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 final guidance for industry entitled
‘‘Amyotrophic Lateral Sclerosis:
Developing Drugs for Treatment.’’ ALS
is a progressive neurodegenerative
disease that primarily affects motor
neurons in the cerebral motor cortex,
brainstem, and spinal cord, leading to
loss of voluntary movement and the
development of difficulty in
swallowing, speaking, and breathing.
The purpose of this guidance is to assist
sponsors in the clinical development of
drugs for the treatment of ALS.
The guidance addresses the clinical
development of drugs intended to treat
the main motor aspects of ALS (i.e.,
muscle weakness and its direct
consequences, including shortened
survival). It does not address in detail
the development of drugs to treat other
symptoms that may arise in ALS, such
as muscle cramps, spasticity, sialorrhea,
and pseudobulbar affect.
The guidance covers considerations
for early phase clinical development,
drug development population, clinical
efficacy study design and endpoints,
and risk-benefit, as well as other factors,
such as relevant nonclinical safety and
pharmacokinetic/pharmacodynamic
considerations. This guidance finalizes
the draft guidance of the same name
issued on February 16, 2018 (83 FR
7047). Changes made to the guidance
took into consideration written and
verbal comments received. Information
was added about strategies to expedite
clinical trials in ALS and minimize
exposure to placebo, about the
importance of broad inclusion criteria in
ALS clinical trials, encouraging the use
E:\FR\FM\24SEN1.SGM
24SEN1
Federal Register / Vol. 84, No. 185 / Tuesday, September 24, 2019 / Notices
of patient input and experience in the
development of new outcome measures
that are capable of measuring clinically
meaningful effects in patients, and
encouraging the incorporation of
exploratory biomarkers in ALS
development programs. Language was
also added to provide greater context on
the use of historical control data in ALS
clinical trials, to suggest approaches to
minimize patient burden during clinical
trials, and to clarify safety data
expectations for new ALS treatments.
Finally, additions emphasize FDA’s
willingness to exercise flexibility in
applying the statutory standards for
approval of drugs for the treatment of
serious diseases with unmet medical
needs, while preserving appropriate
assurances for safety and effectiveness.
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 ‘‘Amyotrophic
Lateral Sclerosis: 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 guidance refers to previously
approved collections of information that
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 in 21 CFR part 312 have
been approved under OMB control
number 0910–0014, the collections of
information in 21 CFR part 314 have
been approved under OMB control
number 0910–0001, and the collections
of information referred to in the
guidance for industry entitled
‘‘Establishment and Operation of
Clinical Trial Data Monitoring
Committees’’ (available at https://
www.fda.gov/media/75398/download)
have been approved under OMB control
number 0910–0581.
khammond on DSKJM1Z7X2PROD with NOTICES
III. Electronic Access
Persons with access to the internet
may obtain the guidance at either
https://www.fda.gov/Drugs/
GuidanceCompliance
RegulatoryInformation/Guidances/
default.htm, https://www.fda.gov/
vaccines-blood-biologics/guidancecompliance-regulatory-informationbiologics, or https://
www.regulations.gov.
VerDate Sep<11>2014
17:37 Sep 23, 2019
Jkt 247001
Dated: September 18, 2019.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2019–20629 Filed 9–23–19; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2019–D–3846]
Patient Engagement in the Design and
Conduct of Medical Device Clinical
Investigations; Draft Guidance for
Industry, Food and Drug
Administration Staff, and Other
Stakeholders; Availability
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice of availability.
The Food and Drug
Administration (FDA or Agency) is
announcing the availability of the draft
guidance entitled ‘‘Patient Engagement
in the Design and Conduct of Medical
Device Clinical Investigations.’’ The
Patient Engagement Advisory
Committee (PEAC) recommended that
FDA and industry develop some type of
framework to clarify how patient
advisors can engage in the clinical
investigation process. This draft
guidance focuses on the applications,
perceived barriers, and common
challenges of patient engagement in the
design and conduct of medical device
clinical investigations. This draft
guidance is not final nor is it in effect
at this time.
DATES: Submit either electronic or
written comments on the draft guidance
by November 25, 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:
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
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
PO 00000
Frm 00069
Fmt 4703
Sfmt 4703
50047
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–3846 for ‘‘Patient Engagement
in the Design and Conduct of Medical
Device Clinical Investigations.’’
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
E:\FR\FM\24SEN1.SGM
24SEN1
Agencies
[Federal Register Volume 84, Number 185 (Tuesday, September 24, 2019)]
[Notices]
[Pages 50045-50047]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2019-20629]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2013-N-0035]
Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment;
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
``Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment.'' The
guidance provides FDA's current thinking on the clinical development
program and clinical trial designs for drugs to support an indication
for the treatment of amyotrophic lateral
[[Page 50046]]
sclerosis (ALS). The guidance addresses the clinical development of
drugs intended to treat the main motor aspects of ALS, i.e., muscle
weakness and its direct consequences, including shortened life
expectancy. It does not address in detail the development of drugs to
treat other symptoms that may arise in ALS, such as muscle cramps,
spasticity, sialorrhea, pseudobulbar affect, and others. This guidance
finalizes the draft guidance of the same name issued on February 16,
2018.
DATES: The announcement of the guidance is published in the Federal
Register on September 24, 2019.
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-2013-N-0035 for ``Amyotrophic Lateral Sclerosis: 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 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: Billy Dunn, Center for Drug Evaluation
and Research, Food and Drug Administration, 10903 New Hampshire Ave.,
Bldg. 22, Rm. 4332, Silver Spring, MD 20993-0002, 301-796-2250; 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 final guidance for industry
entitled ``Amyotrophic Lateral Sclerosis: Developing Drugs for
Treatment.'' ALS is a progressive neurodegenerative disease that
primarily affects motor neurons in the cerebral motor cortex,
brainstem, and spinal cord, leading to loss of voluntary movement and
the development of difficulty in swallowing, speaking, and breathing.
The purpose of this guidance is to assist sponsors in the clinical
development of drugs for the treatment of ALS.
The guidance addresses the clinical development of drugs intended
to treat the main motor aspects of ALS (i.e., muscle weakness and its
direct consequences, including shortened survival). It does not address
in detail the development of drugs to treat other symptoms that may
arise in ALS, such as muscle cramps, spasticity, sialorrhea, and
pseudobulbar affect.
The guidance covers considerations for early phase clinical
development, drug development population, clinical efficacy study
design and endpoints, and risk-benefit, as well as other factors, such
as relevant nonclinical safety and pharmacokinetic/pharmacodynamic
considerations. This guidance finalizes the draft guidance of the same
name issued on February 16, 2018 (83 FR 7047). Changes made to the
guidance took into consideration written and verbal comments received.
Information was added about strategies to expedite clinical trials in
ALS and minimize exposure to placebo, about the importance of broad
inclusion criteria in ALS clinical trials, encouraging the use
[[Page 50047]]
of patient input and experience in the development of new outcome
measures that are capable of measuring clinically meaningful effects in
patients, and encouraging the incorporation of exploratory biomarkers
in ALS development programs. Language was also added to provide greater
context on the use of historical control data in ALS clinical trials,
to suggest approaches to minimize patient burden during clinical
trials, and to clarify safety data expectations for new ALS treatments.
Finally, additions emphasize FDA's willingness to exercise flexibility
in applying the statutory standards for approval of drugs for the
treatment of serious diseases with unmet medical needs, while
preserving appropriate assurances for safety and effectiveness.
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 ``Amyotrophic Lateral Sclerosis: 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 guidance refers to previously approved collections of
information that 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 in 21 CFR part 312 have been
approved under OMB control number 0910-0014, the collections of
information in 21 CFR part 314 have been approved under OMB control
number 0910-0001, and the collections of information referred to in the
guidance for industry entitled ``Establishment and Operation of
Clinical Trial Data Monitoring Committees'' (available at https://www.fda.gov/media/75398/download) have been approved under OMB control
number 0910-0581.
III. Electronic Access
Persons with access to the internet may obtain the guidance at
either https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics, or https://www.regulations.gov.
Dated: September 18, 2019.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2019-20629 Filed 9-23-19; 8:45 am]
BILLING CODE 4164-01-P