[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]
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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.
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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,
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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,
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
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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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\
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Annual
21 CFR section Number of frequency per Total annual Hours per Total hours
respondents response responses response
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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....
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Total........................... .............. .............. .............. .............. 123,623
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\1\ There are no capital costs or operating and maintenance costs associated with this collection of
information.
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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