[Federal Register Volume 85, Number 51 (Monday, March 16, 2020)]
[Notices]
[Pages 14949-14951]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-05335]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2018-D-2456]
Slowly Progressive, Low-Prevalence Rare Diseases With Substrate
Deposition That Results From Single Enzyme Defects: Providing Evidence
of Effectiveness for Replacement or Corrective Therapies; 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 final guidance for industry entitled ``Slowly
Progressive, Low-Prevalence Rare Diseases With Substrate Deposition
That Results From Single Enzyme Defects: Providing Evidence of
Effectiveness for Replacement or Corrective Therapies.'' This document
provides guidance to sponsors on the evidence necessary to demonstrate
the effectiveness of investigational new drugs or new drug uses
intended for slowly progressive, low-prevalence rare diseases that are
associated with substrate deposition and are caused by single enzyme
defects.
This guidance applies only to those low-prevalence rare diseases
with a well-characterized pathophysiology and in which changes in
substrate deposition can be readily measured in relevant tissue or
tissues. This guidance incorporates the comments received for and
finalizes the draft guidance of the same name issued on July 27, 2018.
DATES: The announcement of the guidance is published in the Federal
Register on March 16, 2020.
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
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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-2018-D-2456 for ``Slowly Progressive, Low-Prevalence Rare Diseases
With Substrate Deposition That Results From Single Enzyme Defects:
Providing Evidence of Effectiveness for Replacement or Corrective
Therapies.'' 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.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf.
Docket: For access to the docket to read background documents or
the electronic and written/paper comments received, go to https://www.regulations.gov and insert the docket number, found in brackets in
the heading of this document, into the ``Search'' box and follow the
prompts and/or go to the Dockets Management Staff, 5630 Fishers Lane,
Rm. 1061, Rockville, MD 20852.
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: Hylton Joffe, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 22, Rm. 6300, Silver Spring, MD 20993-0002, 301-
796-1954; 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 ``Slowly Progressive, Low-Prevalence Rare Diseases With
Substrate Deposition That Results From Single Enzyme Defects: Providing
Evidence of Effectiveness for Replacement or Corrective Therapies.''
This document provides guidance to sponsors on the evidence necessary
to demonstrate the effectiveness of investigational new drugs or new
drug uses intended for slowly progressive, low-prevalence rare diseases
that are associated with substrate deposition and are caused by single
enzyme defects. This guidance applies only to those low-prevalence rare
diseases with a well-characterized pathophysiology and in which changes
in substrate deposition can be readily measured in relevant tissue or
tissues.
This guidance finalizes the draft guidance of the same name issued
on July 27, 2018 (83 FR 35653). FDA considered comments received on the
draft guidance in devising this final guidance. Changes from the draft
to the final guidance include the following: clarification that a ``low
prevalence'' condition may be defined as one affecting a very small
population (e.g., approximately a few thousand persons or fewer in the
United States); clarification that, in the absence of nonhuman data to
guide a potentially efficacious dose, animal toxicology data can inform
a safe starting human dose; and removal of language regarding assay
versus intrasubject variability--approaches to manage intrasubject
variability within specific drug development programs can be addressed
via formal sponsor meetings with the relevant division at FDA.
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 ``Slowly Progressive, Low-Prevalence Rare
Diseases With Substrate Deposition That Results From Single Enzyme
Defects: Providing Evidence of Effectiveness for Replacement or
Corrective Therapies.'' It does not establish any rights for any person
and is not binding on FDA or the public. You can use an alternative
approach if it satisfies the requirements of the applicable statutes
and regulations.
II. Paperwork Reduction Act of 1995
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-
3521). 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 50 have been approved under OMB control
number 0910-0755. The collections of information for expedited programs
in the guidance for industry entitled ``Expedited Programs for Serious
[[Page 14951]]
Conditions--Drugs and Biologics'' (available at https://www.fda.gov/media/86377/download) have been approved under OMB control number 0910-
0765.
III. Electronic Access
Persons with access to the internet may obtain the guidance at
https://www.fda.gov/drugs/guidance-compliance-regulatory-information/guidances-drugs, https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics, or https://www.regulations.gov.
Dated: March 10, 2020.
Lowell J. Schiller,
Principal Associate Commissioner for Policy.
[FR Doc. 2020-05335 Filed 3-13-20; 8:45 am]
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