[Federal Register Volume 83, Number 2 (Wednesday, January 3, 2018)]
[Notices]
[Pages 383-384]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2017-28337]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2017-D-6759]
Establishing Effectiveness for Drugs Intended To Treat Male
Hypogonadotropic Hypogonadism Attributed to Non-Structural Disorders;
Draft Guidance for Industry; Availability
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice of availability.
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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing
the availability of a draft guidance for industry entitled
``Establishing Effectiveness for Drugs Intended to Treat Male
Hypogonadotropic Hypogonadism Attributed to Non-Structural Disorders.''
This draft guidance provides key design considerations, including
recommendations for patient enrollment criteria and efficacy endpoints,
for clinical trials to establish effectiveness for drugs intended to
treat male hypogonadotropic hypogonadism associated with obesity and
other conditions that do not cause intrinsic damage to the hypothalamus
or pituitary gland. This draft guidance is consistent with
recommendations FDA received at the December 2014 advisory committee
meeting on the appropriate indicated population for testosterone
replacement therapy, and the December 2016 advisory committee meeting
on hypogonadotropic hypogonadism.
DATES: Submit either electronic or written comments on the draft
guidance by March 5, 2018 to ensure that the Agency considers your
comment on this draft guidance before it begins work on the final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
[[Page 384]]
identified, as confidential, if submitted as detailed in
``Instructions.''
Instructions: All submissions received must include the Docket No.
FDA-2017-D-6759 for ``Establishing Effectiveness for Drugs Intended to
Treat Male Hypogonadotropic Hypogonadism Attributed to Non-Structural
Disorders.'' 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 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: Jeannie Roule, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 22, Rm. 5332, Silver Spring, MD 20993-0002, 301-
796-3993.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a draft guidance for industry
entitled ``Establishing Effectiveness for Drugs Intended to Treat Male
Hypogonadotropic Hypogonadism Attributed to Non-Structural Disorders.''
This draft guidance is intended to assist sponsors in designing drug
development programs to demonstrate effectiveness of drugs intended to
treat male hypogonadotropic hypogonadism associated with obesity and
other conditions that do not cause intrinsic damage to the hypothalamus
or pituitary gland.
Male hypogonadism is characterized by serum testosterone
concentrations below the lower limit of the normal range for young,
healthy men with associated symptoms (e.g., reduced libido) or signs
(e.g., loss of muscle mass with reduced muscle strength). Some men who
have had normal puberty and sexual development are subsequently
diagnosed with hypogonadotropic hypogonadism associated with obesity or
other acquired conditions in the absence of intrinsic damage to the
hypothalamus or pituitary. Although these men have serum testosterone
concentrations below the lower limit of the normal range for young,
healthy men, the associated symptoms often experienced in this
population (e.g., low energy, depressed mood) are nonspecific and
cannot definitively be attributed to the low testosterone
concentrations. In addition, it is unclear whether these testosterone
concentrations--in the absence of intrinsic damage to the hypothalamus
and pituitary gland--are inappropriately low and whether increasing
testosterone concentrations in these men confers clinical benefit.
For these reasons, serum testosterone is not a validated surrogate
endpoint for establishing efficacy in these patients, and sponsors
would need to show that an increase in serum testosterone translates
into improvement in how patients feel, function, or survive.
This draft guidance addresses the following topics in establishing
effectiveness of drugs for this population:
Identification of patients for inclusion in clinical trials
and
Efficacy endpoints.
This draft guidance is consistent with recommendations FDA received
at the December 2014 advisory committee meeting on the appropriate
indicated population for testosterone replacement therapy, and the
December 2016 advisory committee meeting on hypogonadotropic
hypogonadism.
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 establishing
effectiveness for drugs intended to treat male hypogonadotropic
hypogonadism attributed to non-structural disorders. 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 draft guidance
at either https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, or
https://www.regulations.gov.
Dated: December 27, 2017.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2017-28337 Filed 1-2-18; 8:45 am]
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