83_FR_28357 83 FR 28240 - Epidermolysis Bullosa: Developing Drugs for Treatment of Cutaneous Manifestations; Draft Guidance for Industry; Availability

83 FR 28240 - Epidermolysis Bullosa: Developing Drugs for Treatment of Cutaneous Manifestations; Draft Guidance for Industry; Availability

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 83, Issue 117 (June 18, 2018)

Page Range28240-28241
FR Document2018-12976

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled ``Epidermolysis Bullosa: Developing Drugs for Treatment of Cutaneous Manifestations.'' The purpose of this draft guidance is to assist sponsors with the development of drugs for treatment or prevention of the serious cutaneous manifestations of the heterogeneous group of disorders collectively known as epidermolysis bullosa (EB). There is an unmet medical need for EB patients due to the paucity of effective treatment options.

Federal Register, Volume 83 Issue 117 (Monday, June 18, 2018)
[Federal Register Volume 83, Number 117 (Monday, June 18, 2018)]
[Notices]
[Pages 28240-28241]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2018-12976]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2018-D-2016]


Epidermolysis Bullosa: Developing Drugs for Treatment of 
Cutaneous Manifestations; Draft Guidance for Industry; Availability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the availability of a draft guidance for industry entitled 
``Epidermolysis Bullosa: Developing Drugs for Treatment of Cutaneous 
Manifestations.'' The purpose of this draft guidance is to assist 
sponsors with the development of drugs for treatment or prevention of 
the serious cutaneous manifestations of the heterogeneous group of 
disorders collectively known as epidermolysis bullosa (EB). There is an 
unmet medical need for EB patients due to the paucity of effective 
treatment options.

DATES: Submit either electronic or written comments on the draft 
guidance by August 17, 2018 to ensure that the Agency considers your 
comment on this draft guidance before it begins work on the final 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

[[Page 28241]]

identified, as confidential, if submitted as detailed in 
``Instructions.''
    Instructions: All submissions received must include the Docket No. 
FDA-2018-D-2016 for ``Epidermolysis Bullosa: Developing Drugs for 
Treatment of Cutaneous Manifestations; 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.thefederalregister.org/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, or 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 draft guidance document.

FOR FURTHER INFORMATION CONTACT: Barbara Gould, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 22, Rm. 5166, Silver Spring, MD 20993-0002, 301-
796-4224; 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 draft guidance for industry 
entitled ``Epidermolysis Bullosa: Developing Drugs for Treatment of 
Cutaneous Manifestations.'' The purpose of this draft guidance is to 
assist sponsors with the development of drugs for treatment or 
prevention of the serious cutaneous manifestations of the heterogeneous 
group of disorders collectively known as EB. There is an unmet medical 
need for EB patients due to the paucity of effective treatment options.
    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 
``Epidermolysis Bullosa: Developing Drugs for Treatment of Cutaneous 
Manifestations.'' 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 draft 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 part 312 have been 
approved under OMB control number 0910-0014.

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/default.htm, or https://www.regulations.gov.

    Dated: June 12, 2018.
Leslie Kux,
Associate Commissioner for Policy.
[FR Doc. 2018-12976 Filed 6-15-18; 8:45 am]
 BILLING CODE 4164-01-P



                                              28240                          Federal Register / Vol. 83, No. 117 / Monday, June 18, 2018 / Notices

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                                              July 12, 2018,      Food and Drug Ad-         July 5, 2018:          https://www.fda.gov/     June 28, 2018   June 28, 2018: Clos-    Submit Comments by September
                                                from 8:30 a.m.      ministration, Center      Closing date            Food/NewsEvents/                        ing date to request     25,     2018    to:    https://
                                                until 3 p.m. EDT.   for Food Safety and       for registration.       WorkshopsMeetings                       special accom-          www.regulations.gov, or Dock-
                                                                    Applied Nutrition,                                Conferences/de-                         modations due to a      ets Management Staff (HFA–
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                                                                    Drive, College Park,                                                                                              Rm. 1061, Rockville, MD
                                                                    MD 20740.                                                                                                         20852.



                                                Dated: June 8, 2018.                                   or prevention of the serious cutaneous                   such as medical information, your or
                                              Leslie Kux,                                              manifestations of the heterogeneous                      anyone else’s Social Security number, or
                                              Associate Commissioner for Policy.                       group of disorders collectively known as                 confidential business information, such
                                              [FR Doc. 2018–12939 Filed 6–15–18; 8:45 am]              epidermolysis bullosa (EB). There is an                  as a manufacturing process. Please note
                                              BILLING CODE 4164–01–P
                                                                                                       unmet medical need for EB patients due                   that if you include your name, contact
                                                                                                       to the paucity of effective treatment                    information, or other information that
                                                                                                       options.                                                 identifies you in the body of your
                                              DEPARTMENT OF HEALTH AND                                 DATES: Submit either electronic or                       comments, that information will be
                                              HUMAN SERVICES                                           written comments on the draft guidance                   posted on https://www.regulations.gov.
                                                                                                       by August 17, 2018 to ensure that the                      • If you want to submit a comment
                                              Food and Drug Administration                             Agency considers your comment on this                    with confidential information that you
                                                                                                       draft guidance before it begins work on                  do not wish to be made available to the
                                              [Docket No. FDA–2018–D–2016]
                                                                                                       the final version of the guidance.                       public, submit the comment as a
                                              Epidermolysis Bullosa: Developing                        ADDRESSES: You may submit comments                       written/paper submission and in the
                                              Drugs for Treatment of Cutaneous                         on any guidance at any time as follows:                  manner detailed (see ‘‘Written/Paper
                                              Manifestations; Draft Guidance for                                                                                Submissions’’ and ‘‘Instructions’’).
                                                                                                       Electronic Submissions
                                              Industry; Availability                                                                                            Written/Paper Submissions
                                                                                                         Submit electronic comments in the
                                              AGENCY:    Food and Drug Administration,                 following way:                                             Submit written/paper submissions as
                                              HHS.                                                       • Federal eRulemaking Portal:                          follows:
                                              ACTION:   Notice of availability.                        https://www.regulations.gov. Follow the
                                                                                                       instructions for submitting comments.                      • Mail/Hand delivery/Courier (for
                                              SUMMARY:  The Food and Drug                              Comments submitted electronically,                       written/paper submissions): Dockets
                                              Administration (FDA or Agency) is                        including attachments, to https://                       Management Staff (HFA–305), Food and
amozie on DSK3GDR082PROD with NOTICES1




                                              announcing the availability of a draft                   www.regulations.gov will be posted to                    Drug Administration, 5630 Fishers
                                              guidance for industry entitled                           the docket unchanged. Because your                       Lane, Rm. 1061, Rockville, MD 20852.
                                              ‘‘Epidermolysis Bullosa: Developing                      comment will be made public, you are                       • For written/paper comments
                                              Drugs for Treatment of Cutaneous                         solely responsible for ensuring that your                submitted to the Dockets Management
                                              Manifestations.’’ The purpose of this                    comment does not include any                             Staff, FDA will post your comment, as
                                              draft guidance is to assist sponsors with                confidential information that you or a                   well as any attachments, except for
                                              the development of drugs for treatment                   third party may not wish to be posted,                   information submitted, marked and


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                                                                             Federal Register / Vol. 83, No. 117 / Monday, June 18, 2018 / Notices                                                   28241

                                              identified, as confidential, if submitted               and Drug Administration, 10001 New                     III. Electronic Access
                                              as detailed in ‘‘Instructions.’’                        Hampshire Ave., Hillandale Building,                     Persons with access to the internet
                                                 Instructions: All submissions received               4th Floor, Silver Spring, MD 20993–                    may obtain the draft guidance at https://
                                              must include the Docket No. FDA–                        0002, or Office of Communication,                      www.fda.gov/Drugs/
                                              2018–D–2016 for ‘‘Epidermolysis                         Outreach, and Development, Center for                  GuidanceCompliance
                                              Bullosa: Developing Drugs for                           Biologics Evaluation and Research,                     RegulatoryInformation/Guidances/
                                              Treatment of Cutaneous Manifestations;                  Food and Drug Administration, 10903                    default.htm, https://www.fda.gov/
                                              Draft Guidance for Industry.’’ Received                 New Hampshire Ave., Bldg. 71, Rm.                      BiologicsBloodVaccines/
                                              comments will be placed in the docket                   3128, Silver Spring, MD 20993–0002.
                                              and, except for those submitted as                                                                             GuidanceCompliance
                                                                                                      Send one self-addressed adhesive label                 RegulatoryInformation/default.htm, or
                                              ‘‘Confidential Submissions,’’ publicly                  to assist that office in processing your
                                              viewable at https://www.regulations.gov                                                                        https://www.regulations.gov.
                                                                                                      requests. See the SUPPLEMENTARY
                                              or at the Dockets Management Staff                      INFORMATION section for electronic                       Dated: June 12, 2018.
                                              between 9 a.m. and 4 p.m., Monday                       access to the draft guidance document.                 Leslie Kux,
                                              through Friday.                                                                                                Associate Commissioner for Policy.
                                                 • Confidential Submissions—To                        FOR FURTHER INFORMATION CONTACT:
                                                                                                                                                             [FR Doc. 2018–12976 Filed 6–15–18; 8:45 am]
                                              submit a comment with confidential                      Barbara Gould, Center for Drug
                                                                                                      Evaluation and Research, Food and                      BILLING CODE 4164–01–P
                                              information that you do not wish to be
                                              made publicly available, submit your                    Drug Administration, 10903 New
                                              comments only as a written/paper                        Hampshire Ave., Bldg. 22, Rm. 5166,
                                                                                                                                                             DEPARTMENT OF HEALTH AND
                                              submission. You should submit two                       Silver Spring, MD 20993–0002, 301–
                                                                                                                                                             HUMAN SERVICES
                                              copies total. One copy will include the                 796–4224; or Stephen Ripley, Center for
                                              information you claim to be confidential                Biologics Evaluation and Research,                     Food and Drug Administration
                                              with a heading or cover note that states                Food and Drug Administration, 10903
                                              ‘‘THIS DOCUMENT CONTAINS                                New Hampshire Ave., Bldg. 71, Rm.                      [Docket No. FDA–2016–D–2569]
                                              CONFIDENTIAL INFORMATION.’’ The                         7301, Silver Spring, MD 20993–0002,
                                              Agency will review this copy, including                 240–402–7911.                                          S9 Nonclinical Evaluation for
                                              the claimed confidential information, in                                                                       Anticancer Pharmaceuticals—
                                                                                                      SUPPLEMENTARY INFORMATION:
                                              its consideration of comments. The                                                                             Questions and Answers; International
                                              second copy, which will have the                        I. Background                                          Council for Harmonisation; Guidance
                                              claimed confidential information                                                                               for Industry; Availability
                                                                                                         FDA is announcing the availability of
                                              redacted/blacked out, will be available                 a draft guidance for industry entitled                 AGENCY:   Food and Drug Administration,
                                              for public viewing and posted on                        ‘‘Epidermolysis Bullosa: Developing                    HHS.
                                              https://www.regulations.gov. Submit                     Drugs for Treatment of Cutaneous
                                              both copies to the Dockets Management                                                                          ACTION:   Notice of availability.
                                                                                                      Manifestations.’’ The purpose of this
                                              Staff. If you do not wish your name and                 draft guidance is to assist sponsors with              SUMMARY:   The Food and Drug
                                              contact information to be made publicly                 the development of drugs for treatment                 Administration (FDA or Agency) is
                                              available, you can provide this                         or prevention of the serious cutaneous                 announcing the availability of a final
                                              information on the cover sheet and not                  manifestations of the heterogeneous                    guidance for industry entitled ‘‘S9
                                              in the body of your comments and you                    group of disorders collectively known as               Nonclinical Evaluation for Anticancer
                                              must identify this information as                       EB. There is an unmet medical need for                 Pharmaceuticals—Questions and
                                              ‘‘confidential.’’ Any information marked                EB patients due to the paucity of                      Answers.’’ The guidance was prepared
                                              as ‘‘confidential’’ will not be disclosed                                                                      under the auspices of the International
                                                                                                      effective treatment options.
                                              except in accordance with 21 CFR 10.20                                                                         Council for Harmonisation of Technical
                                              and other applicable disclosure law. For                   This draft guidance is being issued
                                                                                                      consistent with FDA’s good guidance                    Requirements for Pharmaceuticals for
                                              more information about FDA’s posting                                                                           Human Use (ICH), formerly the
                                              of comments to public dockets, see 80                   practices regulation (21 CFR 10.115).
                                                                                                      The draft guidance, when finalized, will               International Conference on
                                              FR 56469, September 18, 2015, or access                                                                        Harmonisation of Technical
                                              the information at: https://www.gpo.gov/                represent the current thinking of FDA
                                                                                                      on ‘‘Epidermolysis Bullosa: Developing                 Requirements for Registration of
                                              fdsys/pkg/FR-2015-09-18/pdf/2015-                                                                              Pharmaceuticals for Human Use. The
                                              23389.pdf.                                              Drugs for Treatment of Cutaneous
                                                                                                      Manifestations.’’ It does not establish                guidance consists of questions and
                                                 Docket: For access to the docket to                                                                         answers that were developed to provide
                                              read background documents or the                        any rights for any person and is not
                                                                                                      binding on FDA or the public. You can                  additional clarity about anticancer
                                              electronic and written/paper comments                                                                          pharmaceutical development described
                                              received, go to https://                                use an alternative approach if it satisfies
                                                                                                      the requirements of the applicable                     in the ICH guidance ‘‘S9 Nonclinical
                                              www.regulations.gov and insert the                                                                             Evaluation for Anticancer
                                              docket number, found in brackets in the                 statutes and regulations. This guidance
                                                                                                      is not subject to Executive Order 12866.               Pharmaceuticals’’ that was published in
                                              heading of this document, into the                                                                             2010 (ICH S9 (2010)), as well as to
                                              ‘‘Search’’ box and follow the prompts                   II. Paperwork Reduction Act of 1995                    continue progress in the 3Rs of
                                              and/or go to the Dockets Management                                                                            Reduction, Refinement, and
                                              Staff, 5630 Fishers Lane, Rm. 1061,                       This draft guidance refers to
                                                                                                      previously approved collections of                     Replacement in the use of animals.
                                              Rockville, MD 20852.
amozie on DSK3GDR082PROD with NOTICES1




                                                 You may submit comments on any                       information that are subject to review by              DATES: The announcement of the
                                              guidance at any time (see 21 CFR                        the Office of Management and Budget                    guidance is published in the Federal
                                              10.115(g)(5)).                                          (OMB) under the Paperwork Reduction                    Register on June 18, 2018.
                                                 Submit written requests for single                   Act of 1995 (44 U.S.C. 3501–3520). The                 ADDRESSES: You may submit either
                                              copies of the draft guidance to the                     collections of information in 21 CFR                   electronic or written comments on
                                              Division of Drug Information, Center for                part 312 have been approved under                      Agency guidances at any time as
                                              Drug Evaluation and Research, Food                      OMB control number 0910–0014.                          follows:


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Document Created: 2018-06-18 15:49:56
Document Modified: 2018-06-18 15:49:56
CategoryRegulatory Information
CollectionFederal Register
sudoc ClassAE 2.7:
GS 4.107:
AE 2.106:
PublisherOffice of the Federal Register, National Archives and Records Administration
SectionNotices
ActionNotice of availability.
DatesSubmit either electronic or written comments on the draft guidance by August 17, 2018 to ensure that the Agency considers your comment on this draft guidance before it begins work on the final version of the guidance.
ContactBarbara Gould, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 22, Rm. 5166, Silver Spring, MD 20993-0002, 301- 796-4224; 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.
FR Citation83 FR 28240 

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