[Federal Register Volume 84, Number 57 (Monday, March 25, 2019)]
[Notices]
[Pages 11110-11112]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2019-05655]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2019-D-0481]
Rare Diseases: Natural History Studies for Drug Development;
Draft Guidance for Industry; Availability
AGENCY: Food and Drug Administration, HHS.
[[Page 11111]]
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the availability of a draft guidance for industry entitled ``Rare
Diseases: Natural History Studies for Drug Development.'' FDA is
publishing this draft guidance to help inform the design and
implementation of natural history studies that can be used to support
the development of safe and effective drugs and biological products for
rare diseases. A natural history study collects information about the
natural history of a disease in the absence of an intervention, from
the disease's onset until either its resolution or the individual's
death. Although knowledge of a disease's natural history can benefit
drug development for many disorders and conditions, natural history
information is usually not available or is incomplete for most rare
diseases; therefore, natural history information is particularly needed
for these diseases.
DATES: Submit either electronic or written comments on the draft
guidance by May 24, 2019 to ensure that the Agency considers your
comment on this draft guidance before it begins work on the final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-0481 for ``Rare Diseases: Natural History Studies for Drug
Development; Draft Guidance for Industry.'' 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; 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; or
the Office of Orphan Products Development, Office of Special Medical
Programs, Office of the Commissioner, Food and Drug Administration,
10903 New Hampshire Ave., Bldg. 32, Rm. 5295, 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: Lucas Kempf, Center for Drug
Evaluation and Research, Food and Drug Administration, 10903 New
Hampshire Ave., Bldg. 22, Rm. 6460, Silver Spring, MD 20993, 301-796-
1140; 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; or Aaron Friedman,
Office of Orphan Products Development, Office of Special Medical
Programs, Office of the Commissioner, Food and Drug Administration,
10903 New Hampshire Ave., Bldg. 32, Rm. 5295, Silver Spring, MD 20993-
0002, 301-796-8660.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a draft guidance for industry
entitled ``Rare Diseases: Natural History Studies for Drug
Development.'' This draft guidance is intended to help inform the
design and implementation of natural history studies that can be used
to support the development of safe and effective drugs and biological
products for rare diseases. Although FDA has published guidance
concerning common issues encountered in drug
[[Page 11112]]
development for rare diseases, this draft guidance expands on the topic
of natural history studies specifically.
There are approximately 7,000 recognized rare diseases.
Individually, rare diseases affect a small number of people, but
collectively rare diseases affect about 1 in 10 people in the United
States. Most rare diseases have no approved therapies and thus present
a significant unmet public health need. Although knowledge of a
disease's natural history can benefit drug development for many
disorders and conditions, natural history information is usually not
available or is incomplete for most rare diseases; therefore, natural
history information is particularly needed for these diseases.
This draft guidance describes the potential uses of a natural
history study in all phases of drug development and in the
postmarketing period, the strengths and weaknesses of various types of
natural history studies that might be conducted to support drug
development, data elements and research plans, and a practical
framework for the conduct of a natural history study. The draft
guidance also discusses patient confidentiality and data protection
issues in natural history studies and the potential nature of
interactions with FDA related to these studies.
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 ``Rare
Diseases: Natural History Studies for Drug Development.'' 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 parts 312 and 314 have
been approved under OMB control numbers 0910-0014 and 0910-0001,
respectively. The collections of information in 21 CFR parts 50 and 56
(Protection of Human Subjects: Informed Consent; Institutional Review
Boards) have been approved under OMB control number 0910-0755.
III. Electronic Access
Persons with access to the internet may obtain the draft guidance
at https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, https://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, https://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/default.htm, or https://www.regulations.gov.
Dated: March 20, 2019.
Lowell J. Schiller,
Acting Associate Commissioner for Policy.
[FR Doc. 2019-05655 Filed 3-22-19; 8:45 am]
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