Agency Information Collection Activities; Proposed Collection; Comment Request; Orphan Drugs, 62306-62309 [2020-21843]
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62306
Federal Register / Vol. 85, No. 192 / Friday, October 2, 2020 / Notices
jbell on DSKJLSW7X2PROD with NOTICES
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–
2018–D–2382 for ‘‘Opioid Use Disorder:
Endpoints for Demonstrating
Effectiveness of Drugs for Treatment.’’
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, 240–402–7500.
• 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
VerDate Sep<11>2014
19:38 Oct 01, 2020
Jkt 253001
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/201523389.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, 240–402–7500.
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 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 guidance document.
FOR FURTHER INFORMATION CONTACT:
Silvana Borges, Center for Drug
Evaluation and Research, Food and
Drug Administration, 10903 New
Hampshire Ave., Bldg. 22, Rm. 3200,
Silver Spring, MD 20993–0002, 301–
796–0963.
SUPPLEMENTARY INFORMATION:
FDA is announcing the availability of
a final guidance for industry entitled
‘‘Opioid Use Disorder: Endpoints for
Demonstrating Effectiveness of Drugs for
Treatment.’’ This guidance addresses
clinical endpoints acceptable to
demonstrate effectiveness of drugs for
treatment of opioid use disorder.
This guidance finalizes the draft
guidance of the same name issued
August 7, 2018 (83 FR 38699). All the
public comments received on the draft
guidance have been considered and the
guidance has been revised as
appropriate in response to such
comments along with a few editorial
changes.
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 ‘‘Opioid Use
Disorder: Endpoints for Demonstrating
Effectiveness of Drugs for Treatment.’’ It
does not establish any rights for any
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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. 3501–3521). 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 guidance at either
https://www.fda.gov/drugs/guidancecompliance-regulatory-information/
guidances-drugs or https://
www.regulations.gov.
Dated: September 29, 2020.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2020–21826 Filed 10–1–20; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2014–N–0386]
Agency Information Collection
Activities; Proposed Collection;
Comment Request; Orphan Drugs
AGENCY:
Food and Drug Administration,
HHS.
I. Background
PO 00000
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.
Sfmt 4703
ACTION:
Notice.
The Food and Drug
Administration (FDA or Agency) is
announcing an opportunity for public
comment on the proposed collection of
certain information by the Agency.
Under the Paperwork Reduction Act of
1995 (PRA), Federal Agencies are
required to publish notice in the
Federal Register concerning each
proposed collection of information,
including each proposed extension of an
existing collection of information, and
to allow 60 days for public comment in
response to the notice. This notice
solicits comments on information
collection associated with Orphan Drug
provisions of the Federal Food, Drug,
and Cosmetic Act (FD&C Act) and
implementing regulations.
DATES: Submit either electronic or
written comments on the collection of
information by December 1, 2020.
ADDRESSES: You may submit comments
as follows. Please note that late,
SUMMARY:
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Federal Register / Vol. 85, No. 192 / Friday, October 2, 2020 / Notices
untimely filed comments will not be
considered. Electronic comments must
be submitted on or before December 1,
2020. The https://www.regulations.gov
electronic filing system will accept
comments until 11:59 p.m. Eastern Time
at the end of December 1, 2020.
Comments received by mail/hand
delivery/courier (for written/paper
submissions) will be considered timely
if they are postmarked or the delivery
service acceptance receipt is on or
before that date.
jbell on DSKJLSW7X2PROD with NOTICES
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–
2014–N–0386 for ‘‘Agency Information
Collection Activities; Proposed
Collection; Comment Request; Orphan
Drugs.’’ Received comments, those filed
in a timely manner (see ADDRESSES),
will be placed in the docket and, except
VerDate Sep<11>2014
19:38 Oct 01, 2020
Jkt 253001
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, 240–402–7500.
• 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.govinfo.gov/content/pkg/FR-201509-18/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, 240–402–7500.
FOR FURTHER INFORMATION CONTACT:
Jonna Capezzuto, Office of Operations,
Food and Drug Administration, Three
White Flint North, 10A–12M, 11601
Landsdown St., North Bethesda, MD
20852, 301–796–3794, PRAStaff@
fda.hhs.gov.
SUPPLEMENTARY INFORMATION: Under the
PRA (44 U.S.C. 3501–3521), Federal
Agencies must obtain approval from the
Office of Management and Budget
(OMB) for each collection of
information they conduct or sponsor.
‘‘Collection of information’’ is defined
in 44 U.S.C. 3502(3) and 5 CFR
PO 00000
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62307
1320.3(c) and includes Agency requests
or requirements that members of the
public submit reports, keep records, or
provide information to a third party.
Section 3506(c)(2)(A) of the PRA (44
U.S.C. 3506(c)(2)(A)) requires Federal
Agencies to provide a 60-day notice in
the Federal Register concerning each
proposed collection of information,
including each proposed extension of an
existing collection of information,
before submitting the collection to OMB
for approval. To comply with this
requirement, FDA is publishing notice
of the proposed collection of
information set forth in this document.
With respect to the following
collection of information, FDA invites
comments on these topics: (1) Whether
the proposed collection of information
is necessary for the proper performance
of FDA’s functions, including whether
the information will have practical
utility; (2) the accuracy of FDA’s
estimate of the burden of the proposed
collection of information, including the
validity of the methodology and
assumptions used; (3) ways to enhance
the quality, utility, and clarity of the
information to be collected; and (4)
ways to minimize the burden of the
collection of information on
respondents, including through the use
of automated collection techniques,
when appropriate, and other forms of
information technology.
Orphan Drugs; 21 CFR Part 316
OMB Control Number 0910–0167—
Revision
This information collection supports
FDA regulations implementing sections
525, 526, 527, and 528 of the FD&C Act,
as well as related guidance. Sections
525, 526, 527, and 528 pertain to the
development of drugs for rare diseases
or conditions, including biological
products and antibiotics, otherwise
known or referred to as ‘‘Orphan
Drugs.’’ Specifically, section 525 of the
FD&C Act (21 U.S.C. 360aa) requires
written recommendations on studies
required for approval of a marketing
application for a drug for a rare disease
or condition. The information collection
in 21 CFR 316.10, 316.12, and 316.14 is
approved under OMB control numbers
0910–0001 and 0910–0014. Section 526
of the FD&C Act (21 U.S.C. 360bb)
provides for designation of drugs as
orphan drugs when certain conditions
are met; section 527 (21 U.S.C. 360cc)
provides conditions under which a
sponsor of an approved orphan drug
enjoys exclusive FDA marketing
approval for that drug for the orphan
indication for a period of 7 years; and
finally, section 528 (21 U.S.C. 360dd) is
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Federal Register / Vol. 85, No. 192 / Friday, October 2, 2020 / Notices
intended to encourage sponsors to make
investigational orphan drugs available
for treatment of persons in need on an
open protocol basis before the drug has
been approved for general marketing.
Open protocols may permit patients
who are not part of the formal clinical
investigation to obtain treatment where
adequate supplies exist and no
alternative effective therapy is available.
We have issued regulations in part
316 (21 CFR part 316) to implement the
Orphan Drug provisions of the FD&C
Act and to set forth procedures and
requirements related to requesting
recommendations for investigations of
drugs for rare diseases or conditions;
requesting designation of a drug for a
rare disease or condition; or requesting
exclusive approval for a drug for a rare
disease or condition. To assist
respondents and consistent with 21 CFR
part 316.50, our Office of Orphan
Products Development (OOPD)
maintains and makes publicly available
guidance documents that apply to the
Orphan Drug provisions of the FD&C
Act and regulations in part 316. The list
is maintained on the internet and
guidance documents are issued in
accordance with our Good Guidance
Practice regulations in 21 CFR part
10.115 which provide for public
comment at any time. Accordingly, we
are revising the information collection
to include Agency guidance. The
document entitled, ‘‘Guidance for
Industry, Researchers, Patient Groups,
and Food and Drug Administration Staff
on Meetings With the Office of Orphan
Products Development,’’ provides
recommendations to industry,
researchers, patient groups, and other
stakeholders interested in requesting a
meeting, including a teleconference,
with OOPD on issues related to orphan
drug designation requests, humanitarian
use device designation requests, rare
pediatric disease designation requests,
funding opportunities through the
Orphan Products Grants Program and
the Pediatric Device Consortia Grants
Program, and orphan product patient
related topics of concern. It is also
intended to assist OOPD staff in
addressing such meeting requests. This
guidance describes procedures for
requesting, preparing, scheduling,
conducting, and documenting such
meetings, and discusses background
information we recommend be included
in such requests. Information collection
attendant to recommendations in the
guidance are currently approved under
OMB control number 0910–0787,
however for efficiency of Agency
operations we are consolidating it into
this related information collection. The
guidance is available at https://
www.fda.gov/media/92815/download.
The FDA Orphan Drug Designation
Request Form (Form FDA 4035) is
intended to benefit sponsors who desire
to seek orphan designation of drugs
intended for rare diseases or conditions
from only FDA. The form is a simplified
method for sponsors to provide only
information required by 21 CFR 316.20
for FDA to make a decision.
During this public health emergency
associated with the COVID–19
pandemic, the OOPD is providing
sponsors with increased flexibility for
submission of orphan drug designation
requests and related submissions
(amendments, annual reports, etc.).
During this public health emergency,
orphan drug designation, humanitarian
use device designation, and rare
pediatric disease designation requests
and submissions may be submitted
electronically by email to the OOPD.
When transmitting information to the
Orphan Drug Designation Program via
email, please utilize the mailbox
orphan@fda.hhs.gov. The use of
automated read receipt is recommended
to avoid the need to call to verify receipt
of the email. Sponsors and others who
plan to email information to FDA that
is considered to be private, sensitive,
proprietary, or commercial confidential
are strongly encouraged to send it from
an FDA secured email address so the
transmission is encrypted. The OOPD
will assume that the addresses of emails
received or email addresses provided as
a point of contact are FDA secure when
responding to those email addresses.
We estimate the burden of the
information collection as follows:
TABLE 1—ESTIMATED ANNUAL REPORTING BURDEN 1
Content and format of a request for designation; request
for verification of status; amendment to designation .......
§§ 316.20, 316.21, 316.26 (Form FDA 4035) ......................
§ 316.22; Notifications of changes in agents .......................
§ 316.24(a); Deficiency letters and granting orphan-drug
designation .......................................................................
§ 316.27; Submissions to change ownership of orphandrug designation ...............................................................
§ 316.30; Annual reports ......................................................
§ 316.36; Assurance of the availability of sufficient quantities of the orphan drug; holder’s consent for the approval of other marketing applications for the same drug
Guidance Recommendations: Meeting requests to OOPD
and related submission packages ....................................
Total .....................................................................................
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1 There
Annual
frequency per
response
Number of
respondents
21 CFR section
Total annual
responses
Hours per
response
534
534
132
1.25
1.25
1
668
668
70
135
32
2
90,180
21,376
264
20
1
20
2
40
104
744
1
1
104
744
5
3
520
2,232
1
3
3
15
45
2,508
1
2,508
3.595
9,016
........................
........................
........................
........................
123,623
are no capital costs or operating and maintenance costs associated with this collection of information.
VerDate Sep<11>2014
19:38 Oct 01, 2020
Jkt 253001
PO 00000
Total hours
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Federal Register / Vol. 85, No. 192 / Friday, October 2, 2020 / Notices
Based on our evaluation, we have
adjusted the currently approved burden
estimate we attribute to information
collection activities associated with our
Orphan Drug program to reflect an
increase in submissions.
Dated: September 28, 2020.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2020–21843 Filed 10–1–20; 8:45 am]
BILLING CODE 4164–01–P
DEPARTMENT OF HEALTH AND
HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA–2020–D–1497]
Bladder Cancer: Developing Drugs and
Biologics for Adjuvant Treatment;
Draft Guidance for Industry;
Availability
AGENCY:
Food and Drug Administration,
HHS.
ACTION:
Notice of availability.
The Food and Drug
Administration (FDA or Agency) is
announcing the availability of a draft
guidance for industry entitled ‘‘Bladder
Cancer: Developing Drugs and Biologics
for Adjuvant Treatment.’’ This draft
guidance provides recommendations to
sponsors regarding the development of
drugs and biologics (referred to as drugs
in this document) for the adjuvant
treatment of muscle-invasive bladder
cancer. The draft guidance includes
recommendations regarding eligibility
criteria, choice of comparator, follow up
imaging assessments, determination of
disease recurrence, analyses of diseasefree survival, and interpretation of trial
results. This draft guidance is intended
to facilitate the development of drugs
for the adjuvant treatment of bladder
cancer.
DATES: Submit either electronic or
written comments on the draft guidance
by December 1, 2020 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:
jbell on DSKJLSW7X2PROD with NOTICES
SUMMARY:
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
VerDate Sep<11>2014
19:38 Oct 01, 2020
Jkt 253001
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–
2020–D–1497 for ‘‘Bladder Cancer:
Developing Drugs and Biologics for
Adjuvant Treatment.’’ 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, 240–402–7500.
• 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
PO 00000
Frm 00036
Fmt 4703
Sfmt 4703
62309
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.govinfo.gov/content/pkg/FR-201509-18/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, 240–402–7500.
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
(CDER), Food and Drug Administration,
10001 New Hampshire Ave., Hillandale
Building, 4th Floor, Silver Spring, MD
20993–0002 or to the Office of
Communication, Outreach and
Development, Center for Biologics
Evaluation and Research (CBER), 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. The draft guidance may also be
obtained by mail by calling CBER at 1–
800–835–4709 or 240–402–8010. See
the SUPPLEMENTARY INFORMATION section
for electronic access to the draft
guidance document.
FOR FURTHER INFORMATION CONTACT: Julia
Beaver, Center for Drug Evaluation and
Research, Food and Drug
Administration, 10903 New Hampshire
Ave., Bldg. 22, Rm. 2100, Silver Spring,
MD 20993–0002, 240–402–0489 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
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]]>Agencies
[Federal Register Volume 85, Number 192 (Friday, October 2, 2020)]
[Notices]
[Pages 62306-62309]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2020-21843]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2014-N-0386]
Agency Information Collection Activities; Proposed Collection;
Comment Request; Orphan Drugs
AGENCY: Food and Drug Administration, HHS.
ACTION: Notice.
-----------------------------------------------------------------------
SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing
an opportunity for public comment on the proposed collection of certain
information by the Agency. Under the Paperwork Reduction Act of 1995
(PRA), Federal Agencies are required to publish notice in the Federal
Register concerning each proposed collection of information, including
each proposed extension of an existing collection of information, and
to allow 60 days for public comment in response to the notice. This
notice solicits comments on information collection associated with
Orphan Drug provisions of the Federal Food, Drug, and Cosmetic Act
(FD&C Act) and implementing regulations.
DATES: Submit either electronic or written comments on the collection
of information by December 1, 2020.
ADDRESSES: You may submit comments as follows. Please note that late,
[[Page 62307]]
untimely filed comments will not be considered. Electronic comments
must be submitted on or before December 1, 2020. The https://www.regulations.gov electronic filing system will accept comments until
11:59 p.m. Eastern Time at the end of December 1, 2020. Comments
received by mail/hand delivery/courier (for written/paper submissions)
will be considered timely if they are postmarked or the delivery
service acceptance receipt is on or before that date.
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-2014-N-0386 for ``Agency Information Collection Activities;
Proposed Collection; Comment Request; Orphan Drugs.'' Received
comments, those filed in a timely manner (see ADDRESSES), 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, 240-402-7500.
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.govinfo.gov/content/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, 240-402-7500.
FOR FURTHER INFORMATION CONTACT: Jonna Capezzuto, Office of Operations,
Food and Drug Administration, Three White Flint North, 10A-12M, 11601
Landsdown St., North Bethesda, MD 20852, 301-796-3794,
[email protected].
SUPPLEMENTARY INFORMATION: Under the PRA (44 U.S.C. 3501-3521), Federal
Agencies must obtain approval from the Office of Management and Budget
(OMB) for each collection of information they conduct or sponsor.
``Collection of information'' is defined in 44 U.S.C. 3502(3) and 5 CFR
1320.3(c) and includes Agency requests or requirements that members of
the public submit reports, keep records, or provide information to a
third party. Section 3506(c)(2)(A) of the PRA (44 U.S.C. 3506(c)(2)(A))
requires Federal Agencies to provide a 60-day notice in the Federal
Register concerning each proposed collection of information, including
each proposed extension of an existing collection of information,
before submitting the collection to OMB for approval. To comply with
this requirement, FDA is publishing notice of the proposed collection
of information set forth in this document.
With respect to the following collection of information, FDA
invites comments on these topics: (1) Whether the proposed collection
of information is necessary for the proper performance of FDA's
functions, including whether the information will have practical
utility; (2) the accuracy of FDA's estimate of the burden of the
proposed collection of information, including the validity of the
methodology and assumptions used; (3) ways to enhance the quality,
utility, and clarity of the information to be collected; and (4) ways
to minimize the burden of the collection of information on respondents,
including through the use of automated collection techniques, when
appropriate, and other forms of information technology.
Orphan Drugs; 21 CFR Part 316
OMB Control Number 0910-0167--Revision
This information collection supports FDA regulations implementing
sections 525, 526, 527, and 528 of the FD&C Act, as well as related
guidance. Sections 525, 526, 527, and 528 pertain to the development of
drugs for rare diseases or conditions, including biological products
and antibiotics, otherwise known or referred to as ``Orphan Drugs.''
Specifically, section 525 of the FD&C Act (21 U.S.C. 360aa) requires
written recommendations on studies required for approval of a marketing
application for a drug for a rare disease or condition. The information
collection in 21 CFR 316.10, 316.12, and 316.14 is approved under OMB
control numbers 0910-0001 and 0910-0014. Section 526 of the FD&C Act
(21 U.S.C. 360bb) provides for designation of drugs as orphan drugs
when certain conditions are met; section 527 (21 U.S.C. 360cc) provides
conditions under which a sponsor of an approved orphan drug enjoys
exclusive FDA marketing approval for that drug for the orphan
indication for a period of 7 years; and finally, section 528 (21 U.S.C.
360dd) is
[[Page 62308]]
intended to encourage sponsors to make investigational orphan drugs
available for treatment of persons in need on an open protocol basis
before the drug has been approved for general marketing. Open protocols
may permit patients who are not part of the formal clinical
investigation to obtain treatment where adequate supplies exist and no
alternative effective therapy is available.
We have issued regulations in part 316 (21 CFR part 316) to
implement the Orphan Drug provisions of the FD&C Act and to set forth
procedures and requirements related to requesting recommendations for
investigations of drugs for rare diseases or conditions; requesting
designation of a drug for a rare disease or condition; or requesting
exclusive approval for a drug for a rare disease or condition. To
assist respondents and consistent with 21 CFR part 316.50, our Office
of Orphan Products Development (OOPD) maintains and makes publicly
available guidance documents that apply to the Orphan Drug provisions
of the FD&C Act and regulations in part 316. The list is maintained on
the internet and guidance documents are issued in accordance with our
Good Guidance Practice regulations in 21 CFR part 10.115 which provide
for public comment at any time. Accordingly, we are revising the
information collection to include Agency guidance. The document
entitled, ``Guidance for Industry, Researchers, Patient Groups, and
Food and Drug Administration Staff on Meetings With the Office of
Orphan Products Development,'' provides recommendations to industry,
researchers, patient groups, and other stakeholders interested in
requesting a meeting, including a teleconference, with OOPD on issues
related to orphan drug designation requests, humanitarian use device
designation requests, rare pediatric disease designation requests,
funding opportunities through the Orphan Products Grants Program and
the Pediatric Device Consortia Grants Program, and orphan product
patient related topics of concern. It is also intended to assist OOPD
staff in addressing such meeting requests. This guidance describes
procedures for requesting, preparing, scheduling, conducting, and
documenting such meetings, and discusses background information we
recommend be included in such requests. Information collection
attendant to recommendations in the guidance are currently approved
under OMB control number 0910-0787, however for efficiency of Agency
operations we are consolidating it into this related information
collection. The guidance is available at https://www.fda.gov/media/92815/download.
The FDA Orphan Drug Designation Request Form (Form FDA 4035) is
intended to benefit sponsors who desire to seek orphan designation of
drugs intended for rare diseases or conditions from only FDA. The form
is a simplified method for sponsors to provide only information
required by 21 CFR 316.20 for FDA to make a decision.
During this public health emergency associated with the COVID-19
pandemic, the OOPD is providing sponsors with increased flexibility for
submission of orphan drug designation requests and related submissions
(amendments, annual reports, etc.). During this public health
emergency, orphan drug designation, humanitarian use device
designation, and rare pediatric disease designation requests and
submissions may be submitted electronically by email to the OOPD. When
transmitting information to the Orphan Drug Designation Program via
email, please utilize the mailbox [email protected]. The use of
automated read receipt is recommended to avoid the need to call to
verify receipt of the email. Sponsors and others who plan to email
information to FDA that is considered to be private, sensitive,
proprietary, or commercial confidential are strongly encouraged to send
it from an FDA secured email address so the transmission is encrypted.
The OOPD will assume that the addresses of emails received or email
addresses provided as a point of contact are FDA secure when responding
to those email addresses.
We estimate the burden of the information collection as follows:
Table 1--Estimated Annual Reporting Burden \1\
----------------------------------------------------------------------------------------------------------------
Annual
21 CFR section Number of frequency per Total annual Hours per Total hours
respondents response responses response
----------------------------------------------------------------------------------------------------------------
Content and format of a request 534 1.25 668 135 90,180
for designation; request for
verification of status;
amendment to designation.......
Sec. Sec. 316.20, 316.21, 534 1.25 668 32 21,376
316.26 (Form FDA 4035).........
Sec. 316.22; Notifications of 132 1 70 2 264
changes in agents..............
Sec. 316.24(a); Deficiency 20 1 20 2 40
letters and granting orphan-
drug designation...............
Sec. 316.27; Submissions to 104 1 104 5 520
change ownership of orphan-drug
designation....................
Sec. 316.30; Annual reports... 744 1 744 3 2,232
Sec. 316.36; Assurance of the 1 3 3 15 45
availability of sufficient
quantities of the orphan drug;
holder's consent for the
approval of other marketing
applications for the same drug.
Guidance Recommendations: 2,508 1 2,508 3.595 9,016
Meeting requests to OOPD and
related submission packages....
-------------------------------------------------------------------------------
Total........................... .............. .............. .............. .............. 123,623
----------------------------------------------------------------------------------------------------------------
\1\ There are no capital costs or operating and maintenance costs associated with this collection of
information.
[[Page 62309]]
Based on our evaluation, we have adjusted the currently approved
burden estimate we attribute to information collection activities
associated with our Orphan Drug program to reflect an increase in
submissions.
Dated: September 28, 2020.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2020-21843 Filed 10-1-20; 8:45 am]
BILLING CODE 4164-01-P
