82 FR 60402 - Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases; Draft Guidance for Industry; Availability
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 82, Issue 243 (December 20, 2017)
Food and Drug Administration
Federal Register Volume 82, Issue 243 (December 20, 2017)
| Page Range | 60402-60403 | |
| FR Document | 2017-27435 |
[Federal Register Volume 82, Number 243 (Wednesday, December 20, 2017)] [Notices] [Pages 60402-60403] From the Federal Register Online [www.thefederalregister.org] [FR Doc No: 2017-27435] ----------------------------------------------------------------------- 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Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases; Draft Guidance for Industry; Availability AGENCY: Food and Drug Administration, HHS. ACTION: Notice of availability. ----------------------------------------------------------------------- SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ``Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases.'' FDA intends to no longer grant 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), unless the use of the drug in the pediatric subpopulation meets the regulatory criteria for an orphan subset, or unless the disease in the pediatric subpopulation is considered a different disease from the disease in the adult population. 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. DATES: Submit either electronic or written comments on the draft guidance by January 19, 2018 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance.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 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. [[Page 60403]] 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.thefederalregister.org/fdsys/pkg/FR-201-09-18/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: Aaron Friedman, Office of Orphan Products Development, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 32, Rm. 5295, Silver Spring, MD 20993, 301-796-8660. SUPPLEMENTARY INFORMATION: I. Background FDA is announcing the availability of a draft guidance for industry entitled ``Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases.'' FDA intends to no longer grant 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), unless the use of the drug in the pediatric subpopulation meets the regulatory criteria for an orphan subset, or unless the disease in the pediatric subpopulation is considered a different disease from the disease in the adult population. This will help resolve an unintended loophole in the 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. FDA expects to implement this policy upon publication of the final version of this guidance dependent upon comments received. In the interim, FDA will refrain from issuing final decisions on requests for pediatric-subpopulation designation until the guidance is finalized. 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 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 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/Orphan or https://www.regulations.gov. Dated: December 14, 2017. Leslie Kux, Associate Commissioner for Policy. [FR Doc. 2017-27435 Filed 12-19-17; 8:45 am] BILLING CODE 4164-01-P
![60402 Federal Register / Vol. 82, No. 243 / Wednesday, December 20, 2017 / Notices
be sent electronically to West Virginia, 26505, (304) 285–5951; DATES: Submit either electronic or
Comments.applications@stls.frb.org: mgoldcamp@cdc.gov. written comments on the draft guidance
1. David Armbrust, Salem, Illinois; to The Director, Management Analysis by January 19, 2018 to ensure that the
acquire voting shares of Iuka and Services Office, has been delegated Agency considers your comment on this
Bancshares, Inc., Salem, Illinois, and the authority to sign Federal Register draft guidance before it begins work on
thereby indirectly acquire shares of Iuka notices pertaining to announcements of the final version of the guidance.
State Bank, Iuka, Illinois. meetings and other committee ADDRESSES: You may submit comments
B. Federal Reserve Bank of Kansas management activities, for both the on any guidance at any time as follows:
City (Dennis Denney, Assistant Vice Centers for Disease Control and
President) 1 Memorial Drive, Kansas Electronic Submissions
Prevention and the Agency for Toxic
City, Missouri 64198–0001: Substances and Disease Registry. Submit electronic comments in the
1. David Riordan, Abilene, Kansas; following way:
Robert Riordan, Solomon, Kansas;
Elaine L. Baker, • Federal eRulemaking Portal: https://
Dennis Riordan, Salina, Kansas; Director, Management Analysis and Services www.regulations.gov. Follow the
Office; Centers for Disease Control and instructions for submitting comments.
Michael Riordan, St. Charles, Missouri; Prevention.
and Kirk Berneking, Salina, Kansas; to Comments submitted electronically,
[FR Doc. 2017–27324 Filed 12–19–17; 8:45 am] including attachments, to https://
retain voting shares of Solomon
Bancshares, Inc., Solomon, Kansas, and BILLING CODE 4163–18–P www.regulations.gov will be posted to
thereby indirectly retain shares of the docket unchanged. Because your
Solomon State Bank, Solomon, Kansas. comment will be made public, you are
DEPARTMENT OF HEALTH AND solely responsible for ensuring that your
Board of Governors of the Federal Reserve HUMAN SERVICES comment does not include any
System, December 15, 2017.
confidential information that you or a
Ann E. Misback, Food and Drug Administration
third party may not wish to be posted,
Secretary of the Board. such as medical information, your or
[Docket No. FDA–2017–D–6380]
[FR Doc. 2017–27408 Filed 12–19–17; 8:45 am] anyone else’s Social Security number, or
BILLING CODE 6210–01–P Clarification of Orphan Designation of confidential business information, such
Drugs and Biologics for Pediatric as a manufacturing process. Please note
Subpopulations of Common Diseases; that if you include your name, contact
DEPARTMENT OF HEALTH AND Draft Guidance for Industry; information, or other information that
HUMAN SERVICES Availability identifies you in the body of your
comments, that information will be
Centers for Disease Control and AGENCY: Food and Drug Administration, posted on https://www.regulations.gov.
Prevention HHS. • If you want to submit a comment
ACTION: Notice of availability. with confidential information that you
Notice of Closed Meeting do not wish to be made available to the
SUMMARY: The Food and Drug public, submit the comment as a
In accordance with the Federal Administration (FDA or Agency) is
Advisory Committee Act, the Centers for written/paper submission and in the
announcing the availability of a draft manner detailed (see ‘‘Written/Paper
Disease Control and Prevention (CDC) guidance for industry entitled
announces the following meeting. Submissions’’ and ‘‘Instructions’’).
‘‘Clarification of Orphan Designation of
The meeting will be closed to the Drugs and Biologics for Pediatric Written/Paper Submissions
public in accordance with provisions set Subpopulations of Common Diseases.’’
forth in Section 552b(c)(4) and (6), Title Submit written/paper submissions as
FDA intends to no longer grant orphan follows:
5 U.S.C., and the Determination of the drug designation to drugs for pediatric • Mail/Hand delivery/Courier (for
Director, Management Analysis and subpopulations of common diseases written/paper submissions): Dockets
Services Office, CDC, pursuant to Public (i.e., diseases or conditions with an Management Staff (HFA–305), Food and
Law 92–463. overall prevalence of over 200,000 in Drug Administration, 5630 Fishers
Name of Committee: Disease, the United States), unless the use of the Lane, Rm. 1061, Rockville, MD 20852.
Disability, and Injury Prevention and drug in the pediatric subpopulation • For written/paper comments
Control Special Emphasis Panel (SEP)– meets the regulatory criteria for an submitted to the Dockets Management
PAR15–303, Occupational Safety and orphan subset, or unless the disease in Staff, FDA will post your comment, as
Health Education Research Centers the pediatric subpopulation is well as any attachments, except for
(ERC). considered a different disease from the information submitted, marked and
Date: February 21–23, 2018. disease in the adult population. This identified, as confidential, if submitted
Time: 8:30 a.m.–6:00 p.m., EST. will help resolve an unintended as detailed in ‘‘Instructions.’’
Place: The Alexandrian Hotel, 480 loophole in the Pediatric Research Instructions: All submissions received
King Street Alexandria, VA 22314, 703– Equity Act (PREA) orphan exemption must include the Docket No. FDA–
549–6080. process where a sponsor holding a 2017–D–6380 for ‘‘Clarification of
Agenda: The meeting will include the pediatric-subpopulation designation can Orphan Designation of Drugs and
initial review, discussion, and submit a marketing application for use Biologics for Pediatric Subpopulations
evaluation of applications received in of its drug in the non-orphan adult of Common Diseases.’’ Received
sradovich on DSK3GMQ082PROD with NOTICES
response to PAR15–303, Occupational population of that disease, get a comments will be placed in the docket
Safety and Health Education Research pediatric-subpopulation designation for and, except for those submitted as
Centers (ERC). the pediatric subset of the disease, and, ‘‘Confidential Submissions,’’ publicly
FOR FURTHER INFORMATION CONTACT: due to this designation, be exempt from viewable at https://www.regulations.gov
Michael Goldcamp, Ph.D., Scientific conducting the pediatric studies or at the Dockets Management Staff
Review Officer/CDC, 1095 Willowdale normally required under PREA when between 9 a.m. and 4 p.m., Monday
Road, Mailstop H1808, Morgantown, seeking approval of the adult indication. through Friday.
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![Federal Register / Vol. 82, No. 243 / Wednesday, December 20, 2017 / Notices 60403
• Confidential Submissions—To SUPPLEMENTARY INFORMATION: DEPARTMENT OF HEALTH AND
submit a comment with confidential HUMAN SERVICES
information that you do not wish to be I. Background
made publicly available, submit your FDA is announcing the availability of Food and Drug Administration
comments only as a written/paper a draft guidance for industry entitled [Docket No. FDA–2017–D–6580]
submission. You should submit two ‘‘Clarification of Orphan Designation of
copies total. One copy will include the Drugs and Biologics for Pediatric Drug Products Labeled as
information you claim to be confidential Subpopulations of Common Diseases.’’ Homeopathic; Draft Guidance for Food
with a heading or cover note that states FDA intends to no longer grant orphan and Drug Administration Staff and
‘‘THIS DOCUMENT CONTAINS drug designation to drugs for pediatric Industry; Availability
CONFIDENTIAL INFORMATION.’’ The subpopulations of common diseases
Agency will review this copy, including (i.e., diseases or conditions with an AGENCY: Food and Drug Administration,
the claimed confidential information, in overall prevalence of over 200,000 in HHS.
its consideration of comments. The the United States), unless the use of the ACTION: Notice of availability.
second copy, which will have the drug in the pediatric subpopulation
claimed confidential information meets the regulatory criteria for an SUMMARY: The Food and Drug
redacted/blacked out, will be available orphan subset, or unless the disease in Administration (FDA or Agency) is
for public viewing and posted on the pediatric subpopulation is announcing the availability of a draft
https://www.regulations.gov. Submit considered a different disease from the guidance for FDA staff and industry
both copies to the Dockets Management disease in the adult population. This entitled ‘‘Drug Products Labeled as
Staff. If you do not wish your name and will help resolve an unintended Homeopathic.’’ This draft guidance
contact information to be made publicly loophole in the PREA orphan exemption describes how FDA intends to prioritize
available, you can provide this process where a sponsor holding a enforcement and regulatory action with
information on the cover sheet and not pediatric-subpopulation designation can regard to drug products, including
in the body of your comments and you submit a marketing application for use biological products, labeled as
must identify this information as of its drug in the non-orphan adult homeopathic and marketed in the
‘‘confidential.’’ Any information marked population of that disease, get a United States without the required FDA
as ‘‘confidential’’ will not be disclosed pediatric-subpopulation designation for approval.
except in accordance with 21 CFR 10.20 the pediatric subset of the disease, and, DATES: Submit either electronic or
and other applicable disclosure law. For due to this designation, be exempt from written comments on the draft guidance
more information about FDA’s posting conducting the pediatric studies by March 20, 2018 to ensure that the
of comments to public dockets, see 80 normally required under PREA when Agency considers your comment on this
FR 56469, September 18, 2015, or access seeking approval of the adult indication. draft guidance before it begins work on
the information at: https://www.gpo.gov/ the final version of the guidance.
fdsys/pkg/FR-201-09-18/pdf/2015- FDA expects to implement this policy
upon publication of the final version of ADDRESSES: You may submit comments
23389.pdf. on any guidance at any time as follows:
Docket: For access to the docket to this guidance dependent upon
read background documents or the comments received. In the interim, FDA Electronic Submissions
electronic and written/paper comments will refrain from issuing final decisions
on requests for pediatric-subpopulation Submit electronic comments in the
received, go to https:// following way:
designation until the guidance is
www.regulations.gov and insert the
finalized. • Federal eRulemaking Portal:
docket number, found in brackets in the https://www.regulations.gov. Follow the
heading of this document, into the This draft guidance is being issued instructions for submitting comments.
‘‘Search’’ box and follow the prompts consistent with FDA’s good guidance Comments submitted electronically,
and/or go to the Dockets Management practices regulation (21 CFR 10.115). including attachments, to https://
Staff, 5630 Fishers Lane, Rm. 1061, The draft guidance, when finalized, will www.regulations.gov will be posted to
Rockville, MD 20852. represent the current thinking of FDA the docket unchanged. Because your
You may submit comments on any on orphan designation of drugs and comment will be made public, you are
guidance at any time (see 21 CFR biologics for pediatric subpopulations of solely responsible for ensuring that your
10.115(g)(5)). common diseases. It does not establish comment does not include any
Submit written requests for single any rights for any person and is not confidential information that you or a
copies of the draft guidance to the binding on FDA or the public. You can third party may not wish to be posted,
Division of Drug Information, Center for use an alternative approach if it satisfies such as medical information, your or
Drug Evaluation and Research, Food the requirements of the applicable anyone else’s Social Security number, or
and Drug Administration, 10001 New statutes and regulations. This guidance confidential business information, such
Hampshire Ave., Hillandale Building, is not subject to Executive Order 12866. as a manufacturing process. Please note
4th Floor, Silver Spring, MD 20993– that if you include your name, contact
II. Electronic Access
0002. Send one self-addressed adhesive information, or other information that
label to assist that office in processing Persons with access to the internet identifies you in the body of your
your requests. See the SUPPLEMENTARY may obtain the draft guidance at either comments, that information will be
INFORMATION section for electronic https://www.fda.gov/Orphan or https:// posted on https://www.regulations.gov.
sradovich on DSK3GMQ082PROD with NOTICES
access to the draft guidance document. www.regulations.gov. • If you want to submit a comment
FOR FURTHER INFORMATION CONTACT:
Dated: December 14, 2017. with confidential information that you
Aaron Friedman, Office of Orphan do not wish to be made available to the
Leslie Kux,
Products Development, Food and Drug public, submit the comment as a
Administration, 10903 New Hampshire Associate Commissioner for Policy. written/paper submission and in the
Ave., Bldg. 32, Rm. 5295, Silver Spring, [FR Doc. 2017–27435 Filed 12–19–17; 8:45 am] manner detailed (see ‘‘Written/Paper
MD 20993, 301–796–8660. BILLING CODE 4164–01–P Submissions’’ and ‘‘Instructions’’).
VerDate Sep<11>2014 21:36 Dec 19, 2017 Jkt 244001 PO 00000 Frm 00035 Fmt 4703 Sfmt 4703 E:\FR\FM\20DEN1.SGM 20DEN1](https://thefederalregister.org/doc/82_FR_60645/page/2.svg)
Document Created: 2018-10-25 10:55:48
Document Modified: 2018-10-25 10:55:48
| Category | Regulatory Information | |
| Collection | Federal Register | |
| sudoc Class | AE 2.7: GS 4.107: AE 2.106: | |
| Publisher | Office of the Federal Register, National Archives and Records Administration | |
| Section | Notices | |
| Action | Notice of availability. | |
| Dates | Submit either electronic or written comments on the draft guidance by January 19, 2018 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance. | |
| Contact | Aaron Friedman, Office of Orphan Products Development, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 32, Rm. 5295, Silver Spring, MD 20993, 301-796-8660. | |
| FR Citation | 82 FR 60402 |
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