[Federal Register Volume 83, Number 145 (Friday, July 27, 2018)]
[Notices]
[Pages 35655-35657]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2018-16027]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2017-D-6380]


Clarification of Orphan Designation of Drugs and Biologics for 
Pediatric Subpopulations of Common Diseases; 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 
``Clarification of Orphan Designation of Drugs and Biologics for 
Pediatric Subpopulations of Common Diseases.'' FDA does not expect to 
grant any additional orphan-drug designation to drugs for pediatric 
subpopulations of common diseases (i.e., diseases or conditions with an 
overall prevalence of 200,000 or greater). This will help resolve an 
unintended loophole in the Pediatric Research Equity Act (PREA) orphan 
exemption process where a sponsor holding a pediatric-subpopulation 
designation can submit a marketing application for use of its drug

[[Page 35656]]

in the non-orphan adult population of that disease, get a pediatric-
subpopulation designation for the pediatric subset of the disease, and, 
due to this designation, be exempt from conducting the pediatric 
studies normally required under PREA when seeking approval of the adult 
indication.

DATES: The announcement of the guidance is published in the Federal 
Register on July 27, 2018.

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:
    <bullet> 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.
    <bullet> 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:
    <bullet> 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.
    <bullet> 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-2017-D-6380 for ``Clarification of Orphan Designation of Drugs and 
Biologics for Pediatric Subpopulations of Common Diseases.'' 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.
    <bullet> 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 
Office of Orphan Products Development, Office of the Commissioner, Food 
and Drug Administration, 10903 New Hampshire Ave., Bldg. 32, Rm. 5295, 
Silver Spring, MD 20993. 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: Aaron Friedman, Office of Orphan 
Products Development, Food and Drug Administration, 10903 New Hampshire 
Ave., Bldg. 32, Rm. 5209, Silver Spring, MD 20993, 301-796-2989.

SUPPLEMENTARY INFORMATION: 

I. Background

    FDA is announcing the availability of a final guidance for industry 
entitled ``Clarification of Orphan Designation of Drugs and Biologics 
for Pediatric Subpopulations of Common Diseases.'' In the Federal 
Register of December 20, 2017 (82 FR 60402), FDA published a notice of 
availability for the draft guidance entitled ``Clarification of Orphan 
Designation of Drugs and Biologics for Pediatric Subpopulations of 
Common Diseases,'' announcing that FDA does not expect to grant any 
additional orphan drug designation to drugs for pediatric 
subpopulations of common diseases (i.e., diseases or conditions with an 
overall prevalence of over 200,000 in the United States). In the 
Federal Register of January 12, 2018 (83 FR 1619), FDA announced that 
it was extending the comment period for this draft guidance for an 
additional 30 days. FDA received several comments on the draft guidance 
and those comments were considered as the guidance was finalized. The 
guidance announced in this notice finalizes the draft guidance dated 
December 2017. FDA does not expect to grant any additional orphan-drug 
designation to drugs for pediatric subpopulations of common diseases 
(i.e., diseases or conditions with an overall prevalence of 200,000 or 
greater). This will help resolve an unintended loophole in the 
Pediatric Research Equity Act (PREA) orphan exemption process where a 
sponsor holding a pediatric-subpopulation designation can submit a 
marketing application for use of its drug in the non-orphan adult 
population of that disease, get a pediatric-subpopulation designation 
for the pediatric subset of the disease, and, due to this designation, 
be exempt from conducting the pediatric studies normally required under 
PREA when seeking approval of the adult indication.
    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 orphan designation of drugs and biologics 
for pediatric subpopulations of common diseases. It does not establish 
any rights for any person and is not binding on FDA or the

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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/Orphan or https://www.regulations.gov.

    Dated: July 23, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-16027 Filed 7-26-18; 8:45 am]
 BILLING CODE 4164-01-P