
[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Draft Guidance for Industry; Availability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the availability of a draft guidance for industry entitled ``Rare 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publishing this draft guidance to help inform the design and 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DATES: Submit either electronic or written comments on the draft 
guidance by May 24, 2019 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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]
 BILLING CODE 4164-01-P


